2024-511656-41-002AuthorisedAn Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping351-202Duchenne Muscular DystrophySpain:2Italy:2Netherlands:216/03/2026IT: 16/03/2026, ES: 20/03/2026, NL: 16/03/2026Diseases [C] - Musculoskeletal Diseases [C05]Biomarin Pharmaceutical Inc.Pharmaceutical companyTherapeutic exploratory (Phase II)NSAA (total score and selected individual components, i.e., timed 10MWRT and TTR) at baseline and subsequent Q24W visits, Timed 4SC at baseline and subsequent Q24W visits, SV95C (4-week monitoring periods preceding baseline and subsequent Q24W visits), PUL 2.0 at baseline and subsequent Q24W visitsExon 51 specific phosphorothioate oligonucleotide54618-64 years, 0-17 yearsMale3Both5Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, 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publication)PDF2884471.02Spain238732026-03-20T08:58:37.3042026-03-20T08:58:37.3042Italy238722026-03-16T16:27:36.8442026-03-16T16:27:36.8442Netherlands238742026-03-16T10:55:54.6992026-03-16T10:55:54.699223872Italy23873Spain23874Netherlands2024-511656-41-002An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping351-202Duchenne Muscular DystrophySpain:2Italy:2Netherlands:216/03/2026IT: 16/03/2026, ES: 20/03/2026, NL: 16/03/2026Diseases [C] - Musculoskeletal Diseases [C05]Biomarin Pharmaceutical Inc.Pharmaceutical companyTherapeutic exploratory (Phase II)NSAA (total score and selected individual components, i.e., timed 10MWRT and TTR) at baseline and subsequent Q24W visits, Timed 4SC at baseline and subsequent Q24W visits, SV95C (4-week monitoring periods preceding baseline and subsequent Q24W visits), PUL 2.0 at baseline and subsequent Q24W visitsExon 51 specific phosphorothioate oligonucleotide54618-64 years, 0-17 yearsMale35Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiogramNo20/03/202621/03/20262024-511656-41-00Authorised2026-03-16T10:55:54.6992026-03-21T02:36:52.06957214521209858100000000556United Kingdom826GBGBRTrue100000000549Turkey792TRTURTrue501582110564554CONCENTRATE FOR SOLUTION FOR INFUSIONPRD105645541Exon 51 specific phosphorothioate oligonucleotideCONCENTRATE FOR SOLUTION FOR INFUSIONBMN 351BMN 351SUB328513BIOMARIN PHARMACEUTICAL INC.10564554328517BIOMARIN PHARMACEUTICAL INC.Nucleic AcidNucleic AcidBMN 351SUB328513NOTASSIGN-False1Falsemg/kg milligram(s)/kilogram12mg/Kg milligram(s)/kilogram35642972ChemicalPRD10564554BMN 35111INTRAVENOUS INFUSIONFalseExon 51 specific phosphorothioate oligonucleotidebmn 351CONCENTRATE FOR SOLUTION FOR INFUSIONAn Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping9975922548d3d05-c407-42cd-95f3-6f3117de194eEstudio de extensión abierto para evaluar la seguridad y la eficacia a largo plazo de infusiones intravenosas semanales de BMN 351 en participantes con distrofia muscular de Duchenne susceptibles de omisión del exón 517Spanish9975921548d3d05-c407-42cd-95f3-6f3117de194eEen open-label verlengingsonderzoek voor het beoordelen van de veiligheid en werkzaamheid op lange termijn van wekelijkse intraveneuze infusies met BMN 351 bij deelnemers met Duchenne spierdystrofie die mogelijk reageren op exon 51-skipping18DutchAn Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants with Duchenne Muscular Dystrophy9975932011f66da-7da2-4bb9-8106-3cad97aea9faEen open-label verlengingsonderzoek voor het beoordelen van de veiligheid en werkzaamheid op lange termijn van BMN 351 bij deelnemers met Duchenne spierdystrofie18Dutch9975931011f66da-7da2-4bb9-8106-3cad97aea9faEstudio de extensión abierto para evaluar la seguridad y la eficacia a largo plazo de BMN 351 en participantes con distrofia muscular de Duchenne7Spanish351-202495840U1111-1303-9845False42351-202 is a phase 2, open-label extension study of BMN 351 in participants with Duchenne Muscular Dystrophy. In line with EMA guidance on transparency rules, the trial meets the criteria of a Category 2 trial.110929125894Duchenne Muscular Dystrophy99759074fca6846-be65-4ee9-b4f9-cafda6e1fe86Distrofia muscular de Duchenne7Spanish99759084fca6846-be65-4ee9-b4f9-cafda6e1fe86Duchenne spierdystrofie18DutchTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False13Quality of Life3662916366289736629043662925366293To assess the long-term safety and tolerability of BMN 351 in participants with DMD99759303fb44fbe-24bc-40f4-a79c-81b780da0040Het beoordelen van de veiligheid en verdraagbaarheid van BMN 351 op de lange termijn bij deelnemers met DMD18Dutch99759293fb44fbe-24bc-40f4-a79c-81b780da0040Determinar si BMN 351 es seguro, bien tolerado y eficaz para usarlo a largo plazo en participantes con DMD7Spanish4260241To evaluate the effect of BMN 351 on physical function9975933c11ccd46-e766-4276-8907-fdf2b32f3e8aHet beoordelen van het effect van BMN 351 op de lichamelijke functie18Dutch9975934c11ccd46-e766-4276-8907-fdf2b32f3e8aEvaluar el efecto de BMN 351 sobre la función física7Spanish8141821Participants must have completed 351-201 or 351-203 studies without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study9975923467f2a95-d33f-4456-b6ce-833d2f1c7534Los participantes deben haber completado los estudios 351-201 o 351-203 sin una interrupción permanente del medicamento en investigación (PI) ni retirada del estudio7Spanish9975924467f2a95-d33f-4456-b6ce-833d2f1c7534Deelnemers moeten het 351-201- of 351-203-onderzoek hebben voltooid zonder dat het onderzoeksmiddel permanent is stopgezet of dat zij zich uit het onderzoek hebben teruggetrokken18Dutch8141832Currently receiving treatment with oral corticosteroids, on a stable dose regimen during 351-201, and must remain on a consistent dose regimen throughout 351-202 or 351-203 except for modifications to accommodate changes in weight997592683e71cef-f123-4e3c-a619-5685f27a948aOndergaat momenteel een behandeling met orale corticosteroïden, met een stabiele dosering tijdens 351-201, en moet op een consistente dosering blijven gedurende 351-202 of 351-203, met uitzondering van wijzigingen in verband met gewichtsveranderingen18Dutch997592583e71cef-f123-4e3c-a619-5685f27a948aTratamiento actual con corticosteroides orales en una pauta posológica estable durante el estudio 351-201, y mantenimiento de una pauta posológica uniforme durante todo el estudio 351-202 o 351-203, excepto modificaciones para adaptarse a las variaciones del peso.7Spanish8141843Willing to use contraception (sexually mature males) throughout the study and for 90 days after the final dose, if sexually active9975927c45fbe56-4815-4cf4-bf7c-bb651fb6b498Bereid zijn om anticonceptie te gebruiken (geslachtsrijpe mannen) gedurende het onderzoek en gedurende 90 dagen na de laatste dosis, indien seksueel 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Diseases [C05]Satellos Bioscience Inc.Pharmaceutical companyTherapeutic exploratory (Phase II)Changes from baseline in intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in vastus lateralis at Week 12, Changes from baseline in proton muscle transverse relaxation time (T2) in vastus lateralis at Week 12., Changes from baseline in Regeneration Index in open muscle biopsy of the biceps brachii at Week 12, Changes from baseline in function as determined by NSAA assessment at Week 12, Changes from baseline in SV95C at Week 12, Exploratory end point: change from baseline in inflammatory cytokine profile at Week 12, Exploratory end point: change from baseline in creatine kinase at Week 12, Exploratory end point: change from baseline in maximum percent predicted forced vial capacity as measured by spirometry at Week 12, Exploratory end point: change in biceps brachii muscle fiber size and fiber size distribution as determined from histopathology at 12 weeks, Exploratory end point: change in the proportion of embryonic myosin positive fibers as determined from histopathology at 12 weeks., Exploratory end point: change in the number of satellite cells as determined from histopathology at 12 weeks, Exploratory end point: change in endomysial fibrosis and adipose tissue infiltration as determined from histopathology at 12 weeks., Exploratory end point: change in NSAA score over 12 weeks as compared to natural historySAT-3247 10mg tablet, Placebos for SAT-3247 tablets are required for the clinical trial. 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card5822fb3a-23cb-46f2-b1ff-c873b2f87d3d15Subject information and informed consent form (for publication)PDF2816681.01L2_Other subject information material_ES_Pediatrician letter20d795c6-6660-4089-8549-a10f7f410bb715Subject information and informed consent form (for publication)PDF2816681.01L2_Other subject information material_ES_Syde Patient Manualdada15d8-0a8e-4a8e-83f5-5f9ef80e3cee15Subject information and informed consent form (for publication)PDF2816681.21L2_Other subject information material_ES_Syde QuickStartGuide35ca2680-fc6f-45df-afa6-f6b0e6dc0bb615Subject information and informed consent form (for publication)PDF2816681.01L1_parents ICF_ES_redacted07bce20c-1d12-4120-9607-5c7ec42c921c15Subject information and informed consent form (for publication)PDF2816681.21.01Spain554062026-02-17T11:02:40.9652026-02-17T15:08:23.0424Belgium554052026-01-22T16:31:16.0052026-01-30T13:43:54.9092Poland554072026-01-27T13:47:48.7032026-01-30T13:43:54.909255405Belgium55406SpainSTART_OF_TRIAL2026-04-08START_OF_RECRUITMENT2026-04-0855407Poland2025-522522-13-013A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD PatientsSAT-3247-CL-20108/04/2026Duchenne muscular dystrophySpain:4Belgium:2Poland:222/01/2026BE: 30/01/2026, PL: 30/01/2026, ES: 17/02/2026Diseases [C] - Nervous System Diseases [C10]Diseases [C] - Musculoskeletal Diseases [C05]Satellos Bioscience Inc.Pharmaceutical companyTherapeutic exploratory (Phase II)Changes from baseline in intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in vastus lateralis at Week 12, Changes from baseline in proton muscle transverse relaxation time (T2) in vastus lateralis at Week 12., Changes from baseline in Regeneration Index in open muscle biopsy of the biceps brachii at Week 12, Changes from baseline in function as determined by NSAA assessment at Week 12, Changes from baseline in SV95C at Week 12, Exploratory end point: change from baseline in inflammatory cytokine profile at Week 12, Exploratory end point: change from baseline in creatine kinase at Week 12, Exploratory end point: change from baseline in maximum percent predicted forced vial capacity as measured by spirometry at Week 12, Exploratory end point: change in biceps brachii muscle fiber size and fiber size distribution as determined from histopathology at 12 weeks, Exploratory end point: change in the proportion of embryonic myosin positive fibers as determined from histopathology at 12 weeks., Exploratory end point: change in the number of satellite cells as determined from histopathology at 12 weeks, Exploratory end point: change in endomysial fibrosis and adipose tissue infiltration as determined from histopathology at 12 weeks., Exploratory end point: change in NSAA score over 12 weeks as compared to natural historySAT-3247 10mg tablet, Placebos for SAT-3247 tablets are required for the clinical trial. To match the appearance of SAT-3247 active tablets, placebo tablets are formulated by replacing SAT-3247 oxalate DS with lactose monohydrate and microcrystalline cellulose., SAT-3247 50mg tablet0-17 yearsMale332The primary efficacy endpoint is defined as the change from baseline in muscle force measurements as determined by dynamometry at Week 12., Safety endpoints include incidence, severity, and relationship to SAT-3247 of adverse events as well as occurrence of clinically significant changes in physical examination, clinical laboratory measures, vital signs, ECG, and C-SSRSNo17/02/202613/05/20262025-522522-13-01Authorised2026-04-082026-01-22T16:31:16.0052026-05-13T03:33:39.132955084311855419100000000329Australia36AUAUSTrue100000000518Serbia688RSSRBTrue100000000557United States840USUSATrue100000000556United Kingdom826GBGBRTrue491707112753907TABLETPRD127539071SAT-3247 10mg tabletTABLETSAT-3247 OXALATESUB430452SATELLOS BIOSCIENCE INC.12753907430482SATELLOS BIOSCIENCE INC.ChemicalChemicalSAT-3247 OXALATESUB430452False1Falsemg milligram(s)120mg milligram(s)7200122IMP of chemical originPRD12753907SAT-324711ORALTrueSAT-3247 10mg tabletsat-3247 oxalateTABLET4917081N/AN/AN/APlacebos for SAT-3247 tablets are required for the clinical trial. To match the appearance of SAT-3247 active tablets, placebo tablets are formulated by replacing SAT-3247 oxalate DS with lactose monohydrate and microcrystalline cellulose.N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePlacebos for SAT-3247 tablets are required for the clinical trial. To match the appearance of SAT-3247 active tablets, placebo tablets are formulated by replacing SAT-3247 oxalate DS with lactose monohydrate and microcrystalline cellulose.n/aN/A491706112753908TABLETPRD127539081SAT-3247 50mg tabletTABLETSAT-3247 OXALATESUB430452SATELLOS BIOSCIENCE INC.12753908430482SATELLOS BIOSCIENCE INC.ChemicalChemicalSAT-3247 OXALATESUB430452False1Falsemg milligram(s)120mg milligram(s)7200122IMP of chemical originPRD12753908SAT-324711ORALTrueSAT-3247 50mg tabletsat-3247 oxalateTABLETA Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients973732720e3247c-35c5-41fe-a920-e865a37be002Estudio de fase IIa, aleatorizado, doble ciego, controlado con placebo, de comparación de dosis y evaluación exploratoria de la eficacia de SAT-3247 administrado por vía oral a pacientes ambulatorios con distrofia muscular de Duchenne (DMD)7Spanish973732820e3247c-35c5-41fe-a920-e865a37be002Une étude de phase 2a, randomisée, en double aveugle et contrôlée par placebo, pour la comparaison de dose et l’exploration de l’efficacité du SAT-3247 administré par voie orale chez les patients ambulants atteints de DMD33French (Belgium)973732520e3247c-35c5-41fe-a920-e865a37be002Badanie fazy 2a, randomizowane, podwójnie zaślepione, kontrolowane placebo, porównujące dawki oraz eksploracyjne badanie skuteczności doustnie podawanego preparatu SAT-3247 u ambulatoryjnych pacjentów z DMD19Polish973732420e3247c-35c5-41fe-a920-e865a37be002Een fase 2a, gerandomiseerd, dubbelblind, placebogecontroleerd dosisvergelijkend en verkennend onderzoek naar de werkzaamheid van oraal toegediend SAT-3247 bij ambulante DMD-patiënten36Dutch (Belgium)973732620e3247c-35c5-41fe-a920-e865a37be002Eine randomisierte, doppelblinde, placebokontrollierte Dosisvergleichs- und explorative Wirksamkeitsstudie der Phase 2a mit oral verabreichtem SAT-3247 bei gehfähigen DMD-Patienten28German (Belgium)A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients97374037b371a84-6bef-4362-be76-5b56f65b2e37Eine randomisierte, doppelblinde, placebokontrollierte Dosisvergleichs- und explorative Wirksamkeitsstudie der Phase 2a mit oral verabreichtem SAT-3247 bei gehfähigen DMD-Patienten28German (Belgium)97374027b371a84-6bef-4362-be76-5b56f65b2e37Une étude de phase 2a, randomisée, en double aveugle et contrôlée par placebo, pour la comparaison de dose et l’exploration de l’efficacité du SAT-3247 administré par voie orale chez les patients ambulants atteints de DMD33French (Belgium)97374017b371a84-6bef-4362-be76-5b56f65b2e37Badanie fazy 2a, randomizowane, podwójnie zaślepione, kontrolowane placebo, porównujące dawki oraz eksploracyjne badanie skuteczności doustnie podawanego preparatu SAT-3247 u ambulatoryjnych pacjentów z DMD19Polish97374007b371a84-6bef-4362-be76-5b56f65b2e37Een fase 2a, gerandomiseerd, dubbelblind, placebogecontroleerd dosisvergelijkend en verkennend onderzoek naar de werkzaamheid van oraal toegediend SAT-3247 bij ambulante DMD-patiënten36Dutch (Belgium)97373997b371a84-6bef-4362-be76-5b56f65b2e37Estudio de fase IIa, aleatorizado, doble ciego, controlado con placebo, de comparación de dosis y evaluación exploratoria de la eficacia de SAT-3247 administrado por vía oral a pacientes ambulatorios con distrofia muscular de Duchenne (DMD)7SpanishSAT-3247-CL-201False42This is a phase 2a trial108624123323Duchenne muscular dystrophyTrue4358769635877293587715358770Safety: The primary safety objective is to evaluate the safety and tolerability of SAT-3247 in ambulatory DMD patients. Efficacy: The primary efficacy objective is to determine SAT-3247 effects on muscle force as determined by dynamometry at 12 weeks97373964722e808-4398-47d7-bdeb-a16764253ec7Bezpieczeństwo: Pierwszorzędowym celem w zakresie bezpieczeństwa jest ocena bezpieczeństwa i tolerancji preparatu SAT-3247 u ambulatoryjnych pacjentów z DMD Skuteczność: Pierwszorzędowym celem skuteczności jest ocena wpływu preparatu SAT-3247 na siłę mięśni mierzoną za pomocą dynamometrii po 12 tygodniach.19Polish97373954722e808-4398-47d7-bdeb-a16764253ec7Seguridad: El objetivo de seguridad principal es evaluar la seguridad y la tolerabilidad de SAT-3247 en pacientes ambulatorios con DMD Eficacia: El objetivo de eficacia principal es determinar los efectos de SAT-3247 sobre la fuerza muscular determinada por dinamometría al cabo de 12 semanas7Spanish97373944722e808-4398-47d7-bdeb-a16764253ec7Innocuité: L’objectif principal de sécurité est d’évaluer l’innocuité et la tolérabilité du SAT-3247 chez les patients ambulants atteints de DMD Efficacité: L’objectif principal d’efficacité est de déterminer les effets du SAT-3247 sur la force musculaire déterminée par dynamométrie à 12 semaines.33French (Belgium)97373984722e808-4398-47d7-bdeb-a16764253ec7Veiligheid: Het primaire veiligheidsdoel is het evalueren van de veiligheid en verdraagbaarheid van SAT-3247 bij ambulante DMD-patiënten Werkzaamheid: Het primaire werkzaamheidsdoel is het vaststellen van de effecten van SAT-3247 op de spierkracht, zoals bepaald met dynamometrie na 12 weken36Dutch (Belgium)97373974722e808-4398-47d7-bdeb-a16764253ec7Sicherheit: Das primäre Sicherheitsziel ist die Bewertung der Sicherheit und Verträglichkeit von SAT-3247 bei gehfähigen DMD-Patienten Wirksamkeit: Das primäre Wirksamkeitsziel ist die Bestimmung der Auswirkungen von SAT-3247 auf die Muskelkraft, die durch Dynamometrie nach 12 Wochen bestimmt wird28German (Belgium)4167151To determine SAT-3247 effects on intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in vastus lateralis at 12 weeks.9737405b687125d-e5f9-4b68-9b1a-f9d1c10d0e0aBestimmung der Auswirkungen von SAT-3247 auf den intramuskulären Fettanteil in der quantitativen Magnetresonanz (qMR) im Vastus lateralis nach 12 Wochen.28German (Belgium)9737404b687125d-e5f9-4b68-9b1a-f9d1c10d0e0aDéterminer les effets du SAT-3247 sur la fraction de graisse intramusculaire dans la résonance magnétique quantitative (IRMq) du muscle vaste latéral à 12 semaines.33French (Belgium)9737408b687125d-e5f9-4b68-9b1a-f9d1c10d0e0aDeterminar los efectos de SAT-3247 sobre la fracción de grasa intramuscular mediante resonancia magnética cuantitativa (qMR) muscular en el vasto lateral al cabo de 12 semanas.7Spanish9737407b687125d-e5f9-4b68-9b1a-f9d1c10d0e0aHet bepalen van de effecten van SAT-3247 op de kwantitatieve magnetische resonantie (qMR) in de intramusculaire vetfractie in spierweefsel van de vastus lateralis na 12 weken36Dutch (Belgium)9737406b687125d-e5f9-4b68-9b1a-f9d1c10d0e0aOkreślenie wpływu SAT-3247 na frakcję tłuszczu śródmięśniowego w ilościowym rezonansie magnetycznym (qMR) mięśnia obszernego bocznego po 12 tygodniach.19Polish4167162To determine SAT-3247 effects on proton muscle transverse relaxation time (T2) in vastus lateralis at 12 weeks97374093af14c0c-bb41-4a2a-8a06-4b94a35e3a7cHet bepalen van de effecten van SAT-3247 op de transversale relaxatietijd van protonen in spierweefsel (T2) van de vastus lateralis na 12 weken.36Dutch (Belgium)97374113af14c0c-bb41-4a2a-8a06-4b94a35e3a7cDéterminer les effets du SAT-3247 sur le temps de relaxation transversale du muscle protonique (T2) dans le muscle vaste latéral à 12 semaines.33French (Belgium)97374133af14c0c-bb41-4a2a-8a06-4b94a35e3a7cOkreślenie wpływu SAT-3247 na czas relaksacji poprzecznej protonów (T2) w mięśniu obszernym bocznym po 12 tygodniach.19Polish97374123af14c0c-bb41-4a2a-8a06-4b94a35e3a7cBestimmung der Auswirkungen von SAT-3247 auf die transversale Relaxationszeit (T2) der Muskelprotonen im Vastus lateralis nach 12 Wochen.28German (Belgium)97374103af14c0c-bb41-4a2a-8a06-4b94a35e3a7cDeterminar los efectos de SAT-3247 sobre el tiempo de relajación transversal de los protones (T2) en el músculo vasto lateral al cabo de 12 semanas.7Spanish4167173To determine SAT-3247 effects on the Regeneration Index in open muscle biopsy of the biceps brachii at 12 weeks9737415b5508636-ac46-4f0a-b307-50b3b88b5492Bestimmung der Auswirkungen von SAT-3247 auf den Regenerationsindex bei offener Muskelbiopsie des Musculus biceps brachii nach 12 Wochen.28German (Belgium)9737416b5508636-ac46-4f0a-b307-50b3b88b5492Określenie wpływu SAT-3247 na wskaźnik regeneracji w otwartej biopsji mięśnia dwugłowego ramienia po 12 tygodniach.19Polish9737414b5508636-ac46-4f0a-b307-50b3b88b5492Het bepalen van de effecten van SAT-3247 op de regeneratie-index in een open spierbiopt van de biceps brachii na 12 weken.36Dutch (Belgium)9737417b5508636-ac46-4f0a-b307-50b3b88b5492Déterminer les effets du SAT-3247 sur l’indice de régénération dans une biopsie musculaire ouverte du biceps brachii à 12 semaines.33French (Belgium)9737418b5508636-ac46-4f0a-b307-50b3b88b5492Determinar los efectos de SAT-3247 sobre el índice de regeneración en biopsia muscular abierta del bíceps braquial al cabo de 12 semanas.7Spanish4167184To determine population PK of SAT-3247 in ambulatory patients.973742388926545-43ed-4cae-b8f0-8532ab772ea8Déterminer la pharmacocinétique de population du SAT-3247 chez les patients ambulants.33French (Belgium)973741988926545-43ed-4cae-b8f0-8532ab772ea8Określenie farmakokinetyki (PK) SAT-3247 w populacji pacjentów ambulatoryjnych.19Polish973742088926545-43ed-4cae-b8f0-8532ab772ea8Bestimmung der Populations-PK von SAT-3247 bei gehfähigen Patienten.28German (Belgium)973742288926545-43ed-4cae-b8f0-8532ab772ea8Determinar la farmacocinética (PK) poblacional de SAT-3247 en pacientes ambulatorios.7Spanish973742188926545-43ed-4cae-b8f0-8532ab772ea8Het bepalen van de populatie-farmacokinetiek van SAT-3247 bij ambulante patiënten.36Dutch (Belgium)4167195To determine the potential for improvement in muscle function with treatment of SAT-3247 in ambulatory patients97374272c5989a3-b91b-4a91-a969-b337e9b6b839Bestimmung des Potenzials für eine Verbesserung der Muskelfunktion durch die Behandlung mit SAT-3247 bei gehfähigen Patienten.28German (Belgium)97374282c5989a3-b91b-4a91-a969-b337e9b6b839Déterminer le potentiel d’amélioration de la fonction musculaire avec le traitement au SAT-3247 chez les patients ambulants.33French (Belgium)97374242c5989a3-b91b-4a91-a969-b337e9b6b839Określenie potencjału poprawy funkcji mięśni w trakcie leczenia SAT-3247 u pacjentów ambulatoryjnych.19Polish97374262c5989a3-b91b-4a91-a969-b337e9b6b839Determinar el potencial de mejora de la función muscular con el tratamiento de SAT-3247 en pacientes ambulatorios.7Spanish97374252c5989a3-b91b-4a91-a969-b337e9b6b839Het bepalen van het potentieel voor verbetering van de spierfunctie door middel van behandeling met SAT-3247 bij ambulante patiënten.36Dutch (Belgium)4167206Exploratory objective: to determine SAT-3247 effects on respiratory function at 12 weeks973743116826873-e843-4490-98e2-a070b57ec9b5Cel eksploracyjny: Określenie wpływu SAT-3247 na czynność układu oddechowego po 12 tygodniach.19Polish973743216826873-e843-4490-98e2-a070b57ec9b5Objetivo exploratorio: Determinar los efectos de SAT-3247 sobre la función respiratoria al cabo de 12 semanas.7Spanish973743016826873-e843-4490-98e2-a070b57ec9b5Verkennende doestelling: Het bepalen van de effecten van SAT-3247 op de ademhalingsfunctie na 12 weken.36Dutch (Belgium)973743316826873-e843-4490-98e2-a070b57ec9b5Exploratives Ziel: Bestimmung der Auswirkungen von SAT-3247 auf die Atmungsfunktion nach 12 Wochen.28German (Belgium)973742916826873-e843-4490-98e2-a070b57ec9b5Objectif exploratoire: Déterminer les effets du SAT-3247 sur la fonction respiratoire à 12 semaines.33French (Belgium)4167217Exploratory objective: to determine SAT-3247 effects on muscle histopathology of the biceps brachii at 12 weeks9737437f856ccc4-8573-45d9-b531-c90a0accdc99Exploratives Ziel: Bestimmung der Auswirkungen von SAT-3247 auf die Muskelhistopathologie des Musculus biceps brachii nach 12 Wochen.28German (Belgium)9737435f856ccc4-8573-45d9-b531-c90a0accdc99Verkennende doestelling: Het bepalen van de effecten van SAT-3247 op de spierhistopathologie van de biceps brachii na 12 weken.36Dutch (Belgium)9737436f856ccc4-8573-45d9-b531-c90a0accdc99Cel eksploracyjny: Określenie wpływu SAT-3247 na obraz histopatologiczny mięśnia dwugłowego ramienia po 12 tygodniach.19Polish9737434f856ccc4-8573-45d9-b531-c90a0accdc99Objetivo exploratorio: Determinar los efectos de SAT-3247 sobre la histopatología muscular del bíceps braquial al cabo de 12 semanas.7Spanish9737438f856ccc4-8573-45d9-b531-c90a0accdc99Objectif exploratoire: Déterminer les effets du SAT-3247 sur l’histopathologie musculaire du biceps brachial à 12 semaines.33French (Belgium)4167228Exploratory objective: to compare changes in outcomes to natural history973744017c05869-6ce9-4a51-a96e-d4f4e5a12943Objectif exploratoire: Comparer l’évolution des résultats à l’histoire naturelle.33French (Belgium)973743917c05869-6ce9-4a51-a96e-d4f4e5a12943Cel eksploracyjny: Porównanie uzyskanych zmian z naturalnym przebiegiem choroby.19Polish973744317c05869-6ce9-4a51-a96e-d4f4e5a12943Verkennende doestelling: Het vergelijken van veranderingen in resultaten met die bij het natuurlijke beloop van de ziekte.36Dutch (Belgium)973744217c05869-6ce9-4a51-a96e-d4f4e5a12943Objetivo exploratorio: Comparar los cambios en los resultados con la evolución natural de la enfermedad.7Spanish973744117c05869-6ce9-4a51-a96e-d4f4e5a12943Exploratives Ziel: Vergleich der Veränderungen bei den Ergebnissen mit dem natürlichen Verlauf.28German (Belgium)7972531Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene9737330b26cc646-e636-4673-b194-6425c4081f631. Diagnostic définitif de DMD sur la base de résultats cliniques documentés et d’un test génétique préalable confirmant la présence d’une mutation dans le gène DMD.33French (Belgium)9737333b26cc646-e636-4673-b194-6425c4081f631. Definitieve diagnose van DMD op basis van gedocumenteerde klinische bevindingen en eerdere genetische tests met een bevestigde mutatie in het DMD-gen.36Dutch (Belgium)9737331b26cc646-e636-4673-b194-6425c4081f631. Definitive Diagnose von DMD aufgrund dokumentierter klinischer Befunde und vorheriger Gentests mit bestätigter Mutation im DMD-Gen.28German (Belgium)9737332b26cc646-e636-4673-b194-6425c4081f631. El paciente tiene un diagnóstico definitivo de DMD respaldado por resultados clínicos documentados y pruebas genéticas previas con una mutación confirmada en el gen DMD.7Spanish9737329b26cc646-e636-4673-b194-6425c4081f631. Potwierdzona diagnoza dystrofii mięśniowej Duchenne’a (DMD) na podstawie udokumentowanych wyników badań klinicznych oraz wcześniejszych badań genetycznych, z wykrytą mutacją w genie DMD.19Polish79725410If participant is taking (or has taken within 4 weeks prior to enrollment) herbal remedies and supplements which can impact muscle strength and function (e.g., Co-enzyme Q10, creatine, etc.), these are maintained on a stable regimen for the duration of the trial.9737335192a96ba-455c-43cf-8066-0a553295a22d10. Si el participante está tomando (o ha tomado en las 4 semanas anteriores a la inscripción) remedios y complementos a base de hierbas que puedan afectar a la fuerza y a la función muscular (por ejemplo, coenzima Q10, creatina, etc.), estos se mantienen en una pauta estable durante todo el estudio.7Spanish9737336192a96ba-455c-43cf-8066-0a553295a22d10. Si le participant prend (ou a pris dans les 4 semaines précédant l’inscription) des remèdes à base de plantes et des suppléments qui peuvent avoir un impact sur la force et la fonction musculaires (par exemple, coenzyme Q10, créatine, etc.), ceux-ci doivent être maintenus à un régime stable pendant toute la durée de l’étude.33French (Belgium)9737337192a96ba-455c-43cf-8066-0a553295a22d10. Wenn der Teilnehmer pflanzliche Heilmittel und Nahrungsergänzungsmittel einnimmt (oder in den 4 Wochen vor der Aufnahme eingenommen hat), die sich auf die Muskelkraft und -funktion auswirken können (z. B. Co-Enzym Q10, Kreatin usw.), muss für diese für die Dauer der Studie ein stabiler Behandlungsplan beibehalten werden.28German (Belgium)9737334192a96ba-455c-43cf-8066-0a553295a22d10. Als de deelnemer kruidengeneesmiddelen en supplementen gebruikt (of binnen 4 weken voorafgaand aan inschrijving heeft gebruikt) die de spierkracht en -functie kunnen beïnvloeden (bijv. co-enzym Q10, creatine, enz.), worden deze gedurende de duur van het onderzoek op een stabiel regime gehandhaafd.36Dutch (Belgium)9737338192a96ba-455c-43cf-8066-0a553295a22d10. Jeśli uczestnik przyjmuje (lub przyjmował w ciągu 4 tygodni przed włączeniem) preparaty ziołowe i suplementy mogące wpływać na siłę i funkcjonowanie mięśni (np. koenzym Q10, kreatyna itp.), ich stosowanie musi być kontynuowane w stabilnym schemacie przez cały okres trwania badania.19Polish79725511Participants that have previously received delandistrogene moxeparvovec (brand name Elevidys) either in a prior clinical trial or in the commercial setting > 18 months prior to screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible. a. Enrollment of participants previously treated with gene therapy, whether delandistrogene moxeparvovec or another investigational gene therapy product, will be capped at 25% and stratified between cohorts9737343478287ff-5cf7-485d-a2f2-f3900ee636aa11. Les participants qui ont reçu auparavant du delandistrogene moxeparvovec (nom de marque Elevidys) dans le cadre d’une étude clinique antérieure ou dans un contexte commercial > 18 mois avant la sélection et dont les tests de la fonction musculaire se sont stabilisés ou ont montré un déclin ≥ 3 mois avant la sélection, tel que déterminé par l’investigateur et documenté dans les notes du dossier, seront éligibles. a. L’inscription de participants déjà traités par thérapie génique, qu’il s’agisse du delandistrogene moxeparvovec ou d’un autre produit de thérapie génique expérimental, sera plafonné à 25 % et stratifié entre les cohortes.33French (Belgium)9737340478287ff-5cf7-485d-a2f2-f3900ee636aa11. Uczestnicy, którzy wcześniej otrzymali delandistrogen moxeparvovec (nazwa handlowa: Elevidys) w ramach wcześniejszego badania klinicznego lub w warunkach komercyjnych, >18 miesięcy przed wizytą przesiewową, u których testy czynności mięśniowej ustabilizowały się lub wykazały pogorszenie ≥ 3 miesiące przed wizytą przesiewową — zgodnie z oceną badacza i udokumentowane w dokumentacji medycznej — będą kwalifikować się do udziału w badaniu. a. Liczba uczestników wcześniej leczonych terapią genową – zarówno delandistrogenem moxeparvovec, jak i innym badanym produktem terapii genowej – zostanie ograniczona do 25% i zrównoważona pomiędzy kohortami.19Polish9737342478287ff-5cf7-485d-a2f2-f3900ee636aa11. Serán aptos aquellos participantes que hayan recibido previamente moxeparvovec delandistrogénico (nombre comercial Elevidys), ya sea en un estudio previo o en el ámbito comercial > 18 meses antes de la selección, cuyas pruebas de función muscular se hayan estabilizado o hayan presentado un deterioro ≥ 3 meses antes de la selección, según las valoraciones del investigador y lo documentado en la historia clínica. a. Se limitará al 25 % la inscripción de participantes previamente tratados con terapia génica, ya sea delandistrogene moxeparvovec u otro medicamento para terapia génica en investigación, y se estratificará entre cohortes.7Spanish9737339478287ff-5cf7-485d-a2f2-f3900ee636aa11. Deelnemers die > 18 maanden voorafgaand aan screening eerder, in een vorig klinisch onderzoek of op recept, delandistrogene moxeparvovec (merknaam Elevidys) hebben gekregen en wier spierfunctietests ≥ 3 maanden voorafgaand aan screening zijn gestabiliseerd of aangetoond afgenomen, zoals vastgesteld door de onderzoeker en gedocumenteerd in dossiernotities, komen in aanmerking. a. Inschrijving van deelnemers die eerder gentherapie hebben ondergaan, met delandistrogene moxeparvovec of een ander onderzoeksgeneesmiddel voor gentherapie, wordt beperkt tot 25 % en gestratificeerd tussen cohorten.36Dutch (Belgium)9737341478287ff-5cf7-485d-a2f2-f3900ee636aa11. Teilnehmer, die zuvor Delandistrogen Moxeparvovec (Markenname Elevidys) entweder in einer früheren klinischen Studie oder im kommerziellen Rahmen > 18 Monate vor dem Screening erhalten haben und deren Muskelfunktionstests sich stabilisiert haben oder ≥ 3 Monate vor dem Screening eine Verschlechterung aufweisen, wie vom Prüfarzt festgestellt und in den Aufzeichnungen dokumentiert, sind teilnahmeberechtigt. a. Die Aufnahme von Teilnehmern, die zuvor mit einer Gentherapie behandelt wurden, sei es Delandistrogen Moxeparvovec oder ein anderes Gentherapieprodukt, wird auf 25 % begrenzt und zwischen den Kohorten stratifiziert.28German (Belgium)79725612Participants that have previously received an exon skipper > 6 months prior to Screening whose muscle function tests have stabilized or demonstrated declined ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible.97373441abd7c23-3870-467c-8234-cce45c63b32e12. Teilnehmer, die bereits einen Exon-Skipper > 6 Monate vor dem Screening erhalten haben und deren Muskelfunktionstests sich stabilisiert oder ≥ 3 Monate vor dem Screening verschlechtert haben, wie vom Prüfarzt festgestellt und in den Aufzeichnungen dokumentiert, sind teilnahmeberechtigt.28German (Belgium)97373451abd7c23-3870-467c-8234-cce45c63b32e12. Uczestnicy, którzy wcześniej otrzymali lek z grupy exon skipper > 6 miesięcy przed wizytą przesiewową, u których testy funkcji mięśniowej ustabilizowały się lub wykazały pogorszenie ≥ 3 miesiące przed wizytą przesiewową – zgodnie z oceną badacza i potwierdzone w dokumentacji medycznej – będą kwalifikować się do udziału w badaniu.19Polish97373471abd7c23-3870-467c-8234-cce45c63b32e12. Les participants traités antérieurement par saut d’exon > 6 mois avant la sélection et dont les tests de la fonction musculaire se sont stabilisés ou ont montré un déclin ≥ 3 mois avant la sélection, comme déterminé par l’investigateur et documenté dans les notes du dossier, seront éligibles.33French (Belgium)97373461abd7c23-3870-467c-8234-cce45c63b32e12. Serán aptos aquellos participantes que hayan recibido previamente una terapia de salto de exón exonskipping) > 6 meses antes de la selección cuyas pruebas de función muscular se hayan estabilizado o hayan presentado un deterioro ≥ 3 meses antes de la selección, según las valoraciones del investigador y lo documentado en la historia clínica.7Spanish97373481abd7c23-3870-467c-8234-cce45c63b32e12. Deelnemers die eerder > 6 maanden voorafgaand aan screening een exon-scipper hebben gekregen en wier spierfuncties ≥ 3 maanden voorafgaand aan de screening zijn gestabiliseerd of aangetoond afgenomen, zoals vastgesteld door de onderzoeker en gedocumenteerd in dossiernotities, komen in aanmerking.36Dutch (Belgium)79725713If participating in a physical therapy/strength training regimen, must be stable for ≥ 2 months prior to the Screening Visit and for the duration of the trial.9737351e94f456e-aaa7-498e-8843-8082a51b22d213. Deelnemers die meedoen aan een fysiotherapie-/ krachttrainingsregime, moeten stabiel zijn gedurende ≥ 2 maanden voorafgaand aan het Screeningbezoek en voor de duur van het onderzoek.36Dutch (Belgium)9737350e94f456e-aaa7-498e-8843-8082a51b22d213. En cas de participation à un programme de kinésithérapie ou de musculation, celui-ci doit être stable pendant ≥ 2 mois avant la visite de sélection et pendant toute la durée de l’étude.33French (Belgium)9737352e94f456e-aaa7-498e-8843-8082a51b22d213. Si se participa mientras se recibe tratamiento con fisioterapia o entrenamiento de fuerza, dicho tratamiento debe mantenerse sin cambios durante ≥ 2 meses antes de la visita de selección y durante todo el estudio.7Spanish9737349e94f456e-aaa7-498e-8843-8082a51b22d213. Wenn Sie an einer Physiotherapie/Krafttraining teilnehmen, muss dieses ≥ 2 Monate vor der Screening-Visite und während der gesamten Studiendauer stabil sein.28German (Belgium)9737353e94f456e-aaa7-498e-8843-8082a51b22d213. W przypadku uczestniczenia w programie fizjoterapii lub treningu siłowego, musi on być prowadzony w sposób stabilny przez co najmniej 2 miesiące przed wizytą przesiewową i kontynuowany w niezmienionej formie przez cały czas trwania badania.19Polish7972582Male DMD patients who are ambulatory and aged ≥ 7 to < 10 years at the time of screening973735406b12ff3-2292-497d-ba24-e21c6744abd22. Pacientes varones con DMD que sean ambulatorios y tengan una edad comprendida entre ≥ 7 y < 10 años en el momento de la selección.7Spanish973735506b12ff3-2292-497d-ba24-e21c6744abd22. Männliche DMD-Patienten, die gehfähig und zum Zeitpunkt des Screenings ≥ 7 bis < 10 Jahre alt sind.28German (Belgium)973735606b12ff3-2292-497d-ba24-e21c6744abd22. Patients masculins atteints de DMD, ambulants et âgés de ≥ 7 à < 10 ans au moment de la sélection.33French (Belgium)973735706b12ff3-2292-497d-ba24-e21c6744abd22. Mężczyźni z DMD, ambulatoryjni, w wieku od ≥ 7 do < 10 lat w momencie badania przesiewowego.19Polish973735806b12ff3-2292-497d-ba24-e21c6744abd22. DMD-patiënt van het mannelijk geslacht, ambulant en ≥ 7 tot < 10 jaar oud op het moment van screening.36Dutch (Belgium)7972593Completed two four-stair climb assessments at Visit 1 with a mean of 8 seconds or less (≤1 s variance).973736340f3f802-1263-4fc4-9db5-a5076858ecea3. Bei Visite 1 wurden zwei Vier-Stufen-Steigtests mit einer Durchschnittszeit von 8 Sekunden oder weniger (≤ 1 s Varianz) abgeschlossen.28German (Belgium)973736040f3f802-1263-4fc4-9db5-a5076858ecea3. El paciente ha completado dos pruebas consistentes en subir cuatro escalones en la visita 1 con un tiempo medio igual o inferior a 8 segundos (varianza ≤1 segundo).7Spanish973736140f3f802-1263-4fc4-9db5-a5076858ecea3. Ukończenie dwóch prób wspinania się po czterech stopniach podczas wizyty 1 ze średnim czasem ≤ 8 sekund (przy maksymalnej różnicy ≤ 1 sekundy między próbami).19Polish973736240f3f802-1263-4fc4-9db5-a5076858ecea3. Tijdens Bezoek 1 twee vier-treden-klimtests afgerond binnen gemiddeld 8 seconden of minder (variantie ≤ 1 s).36Dutch (Belgium)973735940f3f802-1263-4fc4-9db5-a5076858ecea3. Avoir effectué deux montées chronométrées sur quatre marches lors de la visite 1 avec une moyenne de 8 secondes ou moins (variance ≤1 s).33French (Belgium)7972604Have a time to rise of at least 3 seconds but less than 10 seconds.97373649f7a3d57-ab73-4f05-b91e-1cfecbba61ab4. Czas wstawania z podłogi wynoszący co najmniej 3 sekundy, ale mniej niż 10 sekund.19Polish97373689f7a3d57-ab73-4f05-b91e-1cfecbba61ab4. Avoir un temps de relevé d’au moins 3 secondes mais inférieur à 10 secondes.33French (Belgium)97373669f7a3d57-ab73-4f05-b91e-1cfecbba61ab4. Die Zeit bis zum Aufsteigen muss mindestens 3 Sekunden, aber weniger als 10 Sekunden betragen.28German (Belgium)97373659f7a3d57-ab73-4f05-b91e-1cfecbba61ab4. Overeind kunnen komen binnen 3 tot maximaal 10 seconden.36Dutch (Belgium)97373679f7a3d57-ab73-4f05-b91e-1cfecbba61ab4. El tiempo de subida del paciente es de al menos 3 segundos pero inferior a 10 segundos.7Spanish7972615Healthy, as determined by investigator including medical history, psychiatric history, and no clinically significant findings on physical examination, laboratory tests, and cardiac monitoring.9737372cf85ab00-12c5-4be9-82c2-fb82a5475de25. Stan zdrowia oceniony jako dobry przez badacza, na podstawie wywiadu medycznego, psychiatrycznego oraz braku klinicznie istotnych odchyleń w badaniu fizykalnym, wynikach laboratoryjnych i monitorowaniu kardiologicznym.19Polish9737371cf85ab00-12c5-4be9-82c2-fb82a5475de25. El paciente está en buen estado de salud, según las valoraciones del investigador, incluyendo los antecedentes médicos y psiquiátricos, y sin hallazgos clínicamente relevantes en la exploración física, las pruebas de laboratorio y el seguimiento de la función cardiaca.7Spanish9737373cf85ab00-12c5-4be9-82c2-fb82a5475de25. Gesund, wie vom Prüfarzt unter Berücksichtigung von Anamnese und psychiatrischer Anamnese festgestellt, und keine klinisch signifikanten Befunde bei körperlicher Untersuchung, Labortests und Herzüberwachung.28German (Belgium)9737369cf85ab00-12c5-4be9-82c2-fb82a5475de25. Goede gezondheid, zoals bepaald door de onderzoeker, waarbij tevens gekeken wordt naar de medische en psychiatrische voorgeschiedenis, en geen klinisch significante bevindingen bij lichamelijk onderzoek, laboratoriumtests en hartmonitoring.36Dutch (Belgium)9737370cf85ab00-12c5-4be9-82c2-fb82a5475de25. Être en bonne santé, de l’avis de l’investigateur qui examinera également les antécédents médicaux, les antécédents psychiatriques et l’absence de résultats cliniquement significatifs à l’examen clinique, aux tests de laboratoire et à la surveillance cardiaque.33French (Belgium)7972626Ability for participant and caregiver to communicate satisfactorily with the investigator and to participate in, and comply with the requirements of, the entire trial including scheduled visits, procedures/assessments, questionnaires, laboratory tests, and study restrictions973737897d351f2-ebb6-4e7f-9463-83a053424d046. El participante y el cuidador tienen capacidad para comunicarse de manera satisfactoria con el investigador y para participar y cumplir los requisitos de todo el ensayo, como las visitas programadas, los procedimientos y evaluaciones, los cuestionarios, las pruebas de laboratorio y las restricciones del estudio.7Spanish973737597d351f2-ebb6-4e7f-9463-83a053424d046. Deelnemer en verzorger zijn in staat om op bevredigende wijze met de onderzoeker te communiceren en deel te nemen en te voldoen aan de vereisten van het gehele onderzoek, inclusief geplande bezoeken, procedures/beoordelingen, vragenlijsten, laboratoriumtests en onderzoeksbeperkingen.36Dutch (Belgium)973737797d351f2-ebb6-4e7f-9463-83a053424d046. La capacité du participant et du soignant à communiquer de manière satisfaisante avec l’investigateur, à participer à l’ensemble de l’étude et à en respecter les exigences, y compris les visites programmées, les procédures / évaluations, les questionnaires, les tests de laboratoire et les restrictions de l’étude.33French (Belgium)973737697d351f2-ebb6-4e7f-9463-83a053424d046. Zdolność uczestnika i jego opiekuna do satysfakcjonującej komunikacji z badaczem oraz do uczestniczenia w całym badaniu zgodnie z jego wymaganiami, w tym w zaplanowanych wizytach, procedurach/ocenach, wypełnianiu kwestionariuszy, badaniach laboratoryjnych i przestrzeganiu ograniczeń badania.19Polish973737497d351f2-ebb6-4e7f-9463-83a053424d046. Fähigkeit des Teilnehmers und der Betreuungsperson, mit dem Prüfarzt zufriedenstellend zu kommunizieren und an der gesamten Studie teilzunehmen und deren Anforderungen zu erfüllen, einschließlich geplanter Visiten, Verfahren/Bewertungen, Fragebögen, Labortests und Studieneinschränkungen.28German (Belgium)7972637Participant’s parent(s) or legal guardian(s) have provided written consent to participate after reading the information and consent form, and after having the opportunity to discuss the trial with the investigator or their delegate; participants will be asked to give written or verbal assent according to local requirements.9737379b278e74b-cf3f-45e8-a781-4cbc2f5d235c7. Los padres o tutores legales del participante han dado su consentimiento por escrito para participar tras leer la información y el formulario de consentimiento, y después de haber tenido la oportunidad de hablar sobre el estudio con el investigador o su representante; se pedirá a los participantes que den su consentimiento por escrito o verbal conforme a los requisitos de cada país.7Spanish9737383b278e74b-cf3f-45e8-a781-4cbc2f5d235c7. Le consentement écrit à la participation signé par le(s) parent(s) ou le(s) tuteur(s) légal / légaux du participant, après lecture du document d’information et consentement, et après avoir eu la possibilité de discuter de l’étude avec l’investigateur ou son délégué ; il sera demandé aux participants de donner leur consentement écrit ou verbal conformément aux exigences locales.33French (Belgium)9737381b278e74b-cf3f-45e8-a781-4cbc2f5d235c7. Pisemna zgoda na udział w badaniu udzielona przez rodziców lub opiekunów prawnych po zapoznaniu się z formularzem informacyjnym i zgodą oraz po możliwości przedyskutowania badania z badaczem lub jego przedstawicielem; uczestnicy zostaną poproszeni o wyrażenie pisemnej lub ustnej zgody, zgodnie z lokalnymi przepisami.19Polish9737382b278e74b-cf3f-45e8-a781-4cbc2f5d235c7. Die Eltern oder der/die gesetzliche(n) Vormund(e) des Teilnehmers haben ihr schriftliches Einverständnis zur Teilnahme gegeben, nachdem sie die Informationen und die Einverständniserklärung gelesen haben und die Möglichkeit hatten, die Studie mit dem Prüfarzt oder seinem Beauftragten zu besprechen; die Teilnehmer werden entsprechend den örtlichen Anforderungen um eine schriftliche oder mündliche Zustimmung gebeten.28German (Belgium)9737380b278e74b-cf3f-45e8-a781-4cbc2f5d235c7. De ouder(s) of wettelijke voogd(en) van de deelnemer hebben schriftelijke toestemming voor deelname gegeven na het lezen van het informatie- en toestemmingsformulier en na de gelegenheid te hebben gehad om het onderzoek met de onderzoeker of zijn/haar afgevaardigde te bespreken; deelnemers wordt gevraagd om schriftelijke of mondelinge toestemming te geven volgens lokale vereisten.36Dutch (Belgium)7972648Stable dose of daily systemic glucocorticoids (i.e., prednisolone, deflazacort, or vamorolone) according to the standard of care for ≥ 3 months prior to the Screening Visit and for the duration of the trial. a. Patients who are not receiving glucocorticosteroids are also eligible if stopped ≥ 3 months prior to the Screening Visit.9737387fb6013c2-5e98-4a72-a3fd-9922308715418. Een stabiele dagelijkse dosis systemische glucocorticoïden (prednisolon, deflazacort of vamorolon) volgens de zorgstandaard gedurende ≥ 3 maanden voorafgaand aan het Screeningbezoek en voor de duur van het onderzoek. a. Patiënten die geen glucocorticosteroïden krijgen, komen ook in aanmerking als ze ≥ 3 maanden voorafgaand aan het Screeningbezoek zijn gestopt.36Dutch (Belgium)9737386fb6013c2-5e98-4a72-a3fd-9922308715418. Une dose stable de glucocorticoïdes systémiques quotidiens (c’est-à-dire prednisolone, deflazacort ou vamorolone) conformément à la norme de soins pendant ≥ 3 mois avant la visite de sélection et pendant toute la durée de l’étude. a. Les patients qui ne reçoivent pas de glucocorticoïdes sont également éligibles s’ils ont arrêté ≥ 3 mois avant la visite de sélection33French (Belgium)9737385fb6013c2-5e98-4a72-a3fd-9922308715418. Stabilna dawka ogólnoustrojowych glikokortykosteroidów (np. prednizolon, deflazakort, wamorolon) zgodnie ze standardem leczenia przez co najmniej 3 miesiące przed wizytą przesiewową i przez cały okres trwania badania. a. Pacjenci niestosujący glikokortykosteroidów również mogą zostać włączeni, pod warunkiem że leczenie zostało zakończone co najmniej 3 miesiące przed wizytą przesiewową.19Polish9737384fb6013c2-5e98-4a72-a3fd-9922308715418. Dosis estable de glucocorticoides sistémicos diarios (es decir, prednisolona, deflazacort o vamorolona) según el tratamiento estándar durante ≥ 3 meses antes de la visita de selección y durante todo el estudio. a. Los pacientes que no estén recibiendo glucocorticoides también son aptos si se suspenden ≥ 3 meses antes de la visita de selección.7Spanish9737388fb6013c2-5e98-4a72-a3fd-9922308715418. Stabile Dosis täglicher systemischer Glukokortikoide (d. h. Prednisolon, Deflazacort oder Vamorolon) entsprechend dem Behandlungsstandard für ≥ 3 Monate vor der Screening-Visite und für die Dauer der Studie. a. Patienten, die keine Glukokortikosteroide erhalten, sind ebenfalls teilnahmeberechtigt, wenn sie diese ≥ 3 Monate vor der Screening-Visite abgesetzt haben.28German (Belgium)7972659Stable doses of prescription medicines including ACE inhibitors, β-blockers, and diuretics (excluding glucocorticosteroids) and over-the-counter medicines and/or herbal supplements for supportive care ≥ 1 month prior to the Screening Visit and for the duration of the trial.9737389d4389451-9556-4890-be22-b4b9df66f9d29. Dosis estables de medicamentos de venta con receta, como inhibidores de la ECA, betabloqueantes y diuréticos (excepto glucocorticoides) y medicamentos de venta libre y/o complementos a base de hierbas para cuidados paliativos ≥ 1 mes antes de la visita de selección y durante todo el estudio.7Spanish9737390d4389451-9556-4890-be22-b4b9df66f9d29. Stabilne dawki leków przepisywanych na receptę, w tym inhibitorów ACE, β-blokerów i leków moczopędnych (z wyłączeniem glikokortykosteroidów), a także leków dostępnych bez recepty i/lub suplementów ziołowych stosowanych wspomagająco, przez co najmniej 1 miesiąc przed wizytą przesiewową i przez cały okres trwania badania.19Polish9737391d4389451-9556-4890-be22-b4b9df66f9d29. Een stabiele doses van geneesmiddelen op recept, waaronder ACE-remmers, β-blokkers en diuretica (geen glucocorticosteroïden) en vrij verkrijgbare geneesmiddelen en/of kruidensupplementen voor ondersteunende zorg ≥ 1 maand(en) voorafgaand aan het Screeningbezoek en voor de duur van het onderzoek.36Dutch (Belgium)9737393d4389451-9556-4890-be22-b4b9df66f9d29. Stabile Dosen von verschreibungspflichtigen Medikamenten, einschließlich ACE-Hemmern, β-Blockern und Diuretika (mit Ausnahme von Glukokortikosteroiden) und rezeptfreien Medikamenten und/oder pflanzlichen Nahrungsergänzungsmitteln zur unterstützenden Behandlung ≥ 1 Monat vor der Screening-Visite und während der gesamten Dauer der Studie.28German (Belgium)9737392d4389451-9556-4890-be22-b4b9df66f9d29. Des doses stables de médicaments sur ordonnance, y compris les inhibiteurs de l’ECA, les βbloquants et les diurétiques (à l’exclusion des glucocorticostéroïdes), et de médicaments en vente libre et / ou de suppléments à base de plantes pour les soins de soutien ≥ 1 mois avant la visite de sélection et pendant toute la durée de l’étude.33French (Belgium)13660391Ambulatory patients expected to experience loss of ambulation within ≤ 12 months.9737246f41c01c3-39c6-478f-9b98-a5e02cca48b41. Patients ambulants dont on s’attend à ce qu’ils perdent leur mobilité dans un délai de ≤ 12 mois.33French (Belgium)9737247f41c01c3-39c6-478f-9b98-a5e02cca48b41. Ambulatoryjni pacjenci, u których przewiduje się utratę zdolności do samodzielnego poruszania się w czasie ≤ 12 miesięcy19Polish9737244f41c01c3-39c6-478f-9b98-a5e02cca48b4Pacientes ambulatorios probablemente experimenten pérdida de la deambulación en ≤ 12 meses.7Spanish9737245f41c01c3-39c6-478f-9b98-a5e02cca48b41. Gehfähige Patienten, die voraussichtlich innerhalb von ≤ 12 Monaten ihre Gehfähigkeit verlieren werden.28German (Belgium)9737248f41c01c3-39c6-478f-9b98-a5e02cca48b4Een ambulante patiënt die naar verwachting binnen ≤ 12 maanden verlies van ambulantie zal ervaren.36Dutch (Belgium)136604010Participants for whom MRI or open muscle biopsy are contraindicated.9737249e070ae91-51ab-4184-ab15-be40c255b7954. Teilnehmer, bei denen MRT oder offene Muskelbiopsie kontraindiziert sind.28German (Belgium)9737251e070ae91-51ab-4184-ab15-be40c255b795Participantes en los que estén contraindicadas la IRM o la biopsia muscular abierta.7Spanish9737253e070ae91-51ab-4184-ab15-be40c255b7954. Participants pour lesquels l’IRM ou la biopsie musculaire ouverte sont contre-indiquées.33French (Belgium)9737252e070ae91-51ab-4184-ab15-be40c255b795Een contra-indicatie voor MRI of open spierbiopsie.36Dutch (Belgium)9737250e070ae91-51ab-4184-ab15-be40c255b7954. Przeciwwskazania do wykonania rezonansu magnetycznego (MRI) lub otwartej biopsji mięśnia.19Polish136604111Surgery (e.g., stomach bypass) or medical condition that might affect absorption of medicines.9737255e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9Een operatie (bijv. maagomleiding) of een medische aandoening die de opname van geneesmiddelen kan beïnvloeden.36Dutch (Belgium)9737256e3d679b2-932f-4d9f-899d-6a1a3bc5f1e95. Przebyte zabiegi chirurgiczne (np. ominięcie żołądkowe) lub schorzenia mogące wpływać na wchłanianie leków.19Polish9737258e3d679b2-932f-4d9f-899d-6a1a3bc5f1e95. Intervention chirurgicale (par exemple, pontage gastrique) ou état de santé susceptible d’affecter l’absorption des médicaments.33French (Belgium)9737254e3d679b2-932f-4d9f-899d-6a1a3bc5f1e95. Operationen (z. B. Magenbypass) oder Erkrankungen, die die Aufnahme von Arzneimitteln beeinträchtigen können.28German (Belgium)9737257e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9Cirugía (por ejemplo, bypass gástrico) o enfermedad que pueda afectar a la absorción de los medicamentos.7Spanish136604212Have acute gastrointestinal symptoms (e.g., nausea, vomiting, diarrhea, heartburn) or acute infection (such as influenza) or a significant infection or known inflammatory process at Screening.9737260d31c399b-3374-4d7a-97c4-8f409c8effc56. Ostre objawy ze strony przewodu pokarmowego (np. nudności, wymioty, biegunka, zgaga), ostra infekcja (np. grypa), poważna infekcja lub rozpoznany proces zapalny podczas wizyty przesiewowej.19Polish9737262d31c399b-3374-4d7a-97c4-8f409c8effc56. Akute gastrointestinale Symptome (z. B. Übelkeit, Erbrechen, Durchfall, Sodbrennen) oder eine akute Infektion (z. B. Grippe) oder eine schwerwiegende Infektion oder ein bekannter entzündlicher Prozess beim Screening.28German (Belgium)9737261d31c399b-3374-4d7a-97c4-8f409c8effc56. Présence de symptômes gastro-intestinaux aigus (par exemple, nausées, vomissements, diarrhée, brûlures d’estomac) ou d’une infection aiguë (telle que la grippe) ou d’une infection importante ou d’un processus inflammatoire connu au moment de la sélection.33French (Belgium)9737259d31c399b-3374-4d7a-97c4-8f409c8effc5Pacientes con síntomas gastrointestinales agudos (por ejemplo, náuseas, vómitos, diarrea, acidez) o infección aguda (como gripe) o una infección relevante o un proceso inflamatorio conocido en el momento de la selección.7Spanish9737263d31c399b-3374-4d7a-97c4-8f409c8effc5Acute gastro-intestinale symptomen (zoals misselijkheid, braken, diarree, brandend maagzuur), een acute infectie (zoals influenza) of een ernstige infectie of bekend ontstekingsproces bij screening.36Dutch (Belgium)136604313Impaired cardiac function defined as a left ventricular ejection fraction of < 50% on screening cardiac assessments (echocardiogram or MRI) or evidence of symptomatic cardiomyopathy.973726775c01d77-900e-4472-92da-22a72c5778347. Eingeschränkte Herzfunktion, definiert als linksventrikuläre Auswurffraktion < 50 % bei der kardiologischen Screeninguntersuchung (Echokardiogramm oder MRT) oder Anzeichen einer symptomatischen Kardiomyopathie.28German (Belgium)973726575c01d77-900e-4472-92da-22a72c577834Een verminderde hartfunctie, gedefinieerd als een linkerventrikelejectiefractie van < 50% bij hartonderzoek tijdens screening (echocardiogram of MRI) of tekenen van symptomatische cardiomyopathie.36Dutch (Belgium)973726475c01d77-900e-4472-92da-22a72c5778347. Upośledzona czynność serca, definiowana jako frakcja wyrzutowa lewej komory < 50% w badaniach przesiewowych serca (echokardiografia lub MRI) lub obecność objawowej kardiomiopatii.19Polish973726875c01d77-900e-4472-92da-22a72c577834Función cardíaca deteriorada, entendiendo como tal una fracción de eyección del ventrículo izquierdo de <50 % en las valoraciones cardíacas de selección (ecocardiograma o resonancia magnética) o evidencias de cardiomiopatía sintomática.7Spanish973726675c01d77-900e-4472-92da-22a72c5778347. Altération de la fonction cardiaque définie par une fraction d’éjection du ventricule gauche < 50 % lors des évaluations cardiaques de sélection (échocardiographie ou IRM) ou par des signes de cardiomyopathie symptomatique.33French (Belgium)136604414A forced vital capacity < 60% predicted at the Screening Visit.97372709d4f2469-e7ca-4afe-ac4b-244ca250b85cEen tijdens het Screeningbezoek voorspelde geforceerde vitale capaciteit < 60%.36Dutch (Belgium)97372699d4f2469-e7ca-4afe-ac4b-244ca250b85cUna capacidad vital forzada < 60 % de la prevista en la visita de selección.7Spanish97372739d4f2469-e7ca-4afe-ac4b-244ca250b85c8. Wymuszona pojemność życiowa (FVC) < 60% wartości należnej podczas wizyty przesiewowej.19Polish97372729d4f2469-e7ca-4afe-ac4b-244ca250b85c8. Capacité vitale forcée < 60 % de la valeur prédite lors de la visite de sélection.33French (Belgium)97372719d4f2469-e7ca-4afe-ac4b-244ca250b85c8. Eine forcierte Vitalkapazität < 60 % bei der Screening-Visite.28German (Belgium)136604515Presence or history of severe adverse reaction to any excipients (lactose monohydrate, microcrystalline cellulose, colloidal silicon dioxide, copovidone, crospovidone, sodium strearyl fumarate) in the study medication tablets.97372779a34faa1-c6f8-456a-83c8-6af982b5f84a9. Het bestaan of voorgeschiedenis van ernstige bijwerkingen van hulpstoffen (lactosemonohydraat, microkristallijne cellulose, colloïdaal siliciumdioxide, copovidon, crospovidon, natriumsterearylfumaraat) in de onderzoeksgeneesmiddeltabletten of gekende allergie voor SAT-3247.36Dutch (Belgium)97372759a34faa1-c6f8-456a-83c8-6af982b5f84a9. Vorliegen oder Anamnese einer schweren unerwünschten Reaktion auf einen der Hilfsstoffe (Laktose-Monohydrat, mikrokristalline Cellulose, kolloidales Siliziumdioxid, Copovidon, Crospovidon, Natriumstearylfumarat) in den Tabletten des Prüfpräparats oder bekannte Allergie gegen SAT-3247.28German (Belgium)97372789a34faa1-c6f8-456a-83c8-6af982b5f84a9. Występowanie lub przeszłość ciężkiej reakcji nadwrażliwości na którykolwiek ze składników pomocniczych tabletek badanego leku (monohydrat laktozy, celuloza mikrokrystaliczna, koloidalny dwutlenek krzemu, kopowidon, krospowidon, stearylofumaran sodu) lub znana alergia na SAT-3247.19Polish97372749a34faa1-c6f8-456a-83c8-6af982b5f84a9. Présence ou antécédents de réaction indésirable grave à l’un des excipients (lactose monohydraté, cellulose microcristalline, dioxyde de silicium colloïdal, copovidone, crospovidone, fumarate de stéaryle de sodium) contenus dans les comprimés du médicament à l’étude ou allergie connue au SAT-3247.33French (Belgium)97372769a34faa1-c6f8-456a-83c8-6af982b5f84a9. Presencia o antecedentes de reacciones adversas graves a alguno de los excipientes (lactosa monohidrato, celulosa microcristalina, dióxido de silicio coloidal, copovidona, crospovidona, fumarato de estearilo sódico) presentes en los comprimidos de la medicación del estudio o alergia conocida a SAT-3247 .7Spanish136604616Participants with any known and discernable history of substance abuse and/or dependence.9737282ef67e55a-4f3d-4826-9d38-924d70edcd1516. Participants ayant des antécédents connus et discernables d’abus et/ou de dépendance à des substances33French (Belgium)9737279ef67e55a-4f3d-4826-9d38-924d70edcd1516. Uczestnicy z jakąkolwiek znaną i rozpoznawalną historią nadużywania substancji psychoaktywnych i/lub uzależnienia.19Polish9737283ef67e55a-4f3d-4826-9d38-924d70edcd1516. Deelnemers met om het even welke bekende en herkenbare voorgeschiedenis van middelenmisbruik en/of -afhankelijkheid.36Dutch (Belgium)9737281ef67e55a-4f3d-4826-9d38-924d70edcd1516. Participantes con cualquier historial conocido y discernible de abuso y/o dependencia de sustancias.7Spanish9737280ef67e55a-4f3d-4826-9d38-924d70edcd1516. Teilnehmer mit einer bekannten und erkennbaren Vorgeschichte von Missbrauch und/oder -Abhängigkeit von Substanzen28German (Belgium)13660472Participants maintained on a ten day on/ten day off corticosteroid regimen973728787de9c8e-0315-4fda-ace2-c7301a0b70b4Een aangehouden corticosteroïdregime van tien dagen op/tien dagen af.36Dutch (Belgium)973728687de9c8e-0315-4fda-ace2-c7301a0b70b4Participantes que estén siguiendo una pauta de corticosteroides de diez días con medicación seguida de diez días sin medicación.7Spanish973728587de9c8e-0315-4fda-ace2-c7301a0b70b410. Die Teilnehmer erhielten eine zehntägige Kortikosteroidtherapie mit anschließender zehntägiger Pause.28German (Belgium)973728487de9c8e-0315-4fda-ace2-c7301a0b70b410. Participants maintenus sous un régime de corticostéroïdes de dix jours avec traitement et dix jours sans traitement.33French (Belgium)973728887de9c8e-0315-4fda-ace2-c7301a0b70b410. Uczestnicy pozostający na schemacie leczenia glikokortykosteroidami typu dziesięć dni stosowania / dziesięć dni przerwy.19Polish1366048311 Ongoing participation in any other therapeutic clinical trial or follow-up study for a therapeutic intervention, prior treatment with an investigational gene therapy product (other than delandistrogene moxeparvovec) < 24 months prior to the Screening Visit, or receipt of a stable dose of an approved exon-skipping therapy or any medication indicated for DMD (other than corticosteroids including vamorolone and givinostat in accordance with exception 11b below) within 6 months prior to the Screening Visit. a. Participants that have received a commercially available gene therapy product (i.e., delandistrogene moxeparvovec) > 18 months prior to screening whose muscle function tests have stabilized or demonstrated decline as determined by investigator and documented in the medical record ≥ 3 months prior to screening will be eligible. b. Participants receiving a stable dose of givinostat (brand name Duvyzat) for at least18 months or longer prior to the Screening Visit will be eligible. a. Participants unable to tolerate givinostat who discontinued treatment are eligible to enroll if date of last dose is ≥ 30 days from Screening date. Givinostat should not be discontinued, if tolerated, in order to meet study entry criteria. c. Use of deflazacort (brand name Emflaza) or vamorolone (brand name Agamree) in jurisdictions where these are investigational as they have not received health authority marketing authorization will not be exclusionary; however, simultaneous participation in a clinical trial of deflazacort or vamorolone will be excluded.97372913e4de5d2-2a85-4bad-a556-4ea75d37edeb11. Aktuelle Teilnahme an einer anderen therapeutischen klinischen Studie oder Folgestudie für eine therapeutische Maßnahme, vorherige Behandlung mit einem Gentherapie-Produkt (außer Delandistrogen Moxeparvovec) < 24 Monate vor der Screening-Visite, oder Erhalt einer stabilen Dosis einer zugelassenen Exon-Skipping-Therapie oder eines für DMD indizierten Medikaments (außer Kortikosteroiden einschließlich Vamorolon und Givinostat entsprechend der Ausnahme 11b unten) innerhalb von 6 Monaten vor der Screening-Visite. a. Teilnehmer, die ein kommerziell erhältliches Gentherapieprodukt (z. B. Delandistrogen Moxeparvovec) > 18 Monate vor dem Screening erhalten haben und deren Muskelfunktionstests sich stabilisiert oder verschlechtert haben, wie vom Prüfarzt festgestellt und in der Krankenakte ≥ 3 Monate vor dem Screening dokumentiert, sind teilnahmeberechtigt. b. Teilnehmer, die eine stabile Dosis von Givinostat (Markenname Duvyzat) seit mindestens 18 Monaten oder länger vor der Screening-Visite erhalten, sind teilnahmeberechtigt. a. Teilnehmer, die Givinostat nicht vertragen und die Behandlung abgebrochen haben, können teilnehmen, wenn das Datum der letzten Dosis ≥ 30 Tage nach dem Screening-Datum liegt. Die Einnahme von Givinostat sollte, sofern es vertragen wird, nicht abgebrochen werden, um die Einschlusskriterien der Studie zu erfüllen. c. Die Verwendung von Deflazacort (Markenname Emflaza) oder Vamorolon (Markenname Agamree) in Ländern, in denen diese Arzneimittel noch nicht zugelassen sind, ist kein Ausschlusskriterium; die gleichzeitige Teilnahme an einer klinischen Prüfung von Deflazacort oder Vamorolon ist jedoch ausgeschlossen.28German (Belgium)97372903e4de5d2-2a85-4bad-a556-4ea75d37edeb11. Participation en cours à une autre étude clinique thérapeutique ou à une étude de suivi pour une intervention thérapeutique, traitement antérieur avec un produit de thérapie génique expérimental (autre que le delandistrogene moxeparvovec) < 24 mois avant la visite de sélection, ou réception d’une dose stable d’une thérapie approuvée de saut d’exon ou de tout médicament indiqué pour la DMD (autre que les corticostéroïdes, y compris la vamorolone et le givinostat, conformément à l'exception 11b ci-dessous) dans les 6 mois précédant la visite de sélection. a. Les participants ayant reçu un produit de thérapie génique disponible dans le commerce (c.-à-d. delandistrogene moxeparvovec) > 18 mois avant la sélection et dont les tests de la fonction musculaire se sont stabilisés ou ont montré un déclin tel que déterminé par l’investigateur et documenté dans le dossier médical ≥ 3 mois avant la sélection seront éligibles. b. Les participants recevant une dose stable de givinostat (nom de marque Duvyzat) pendant au moins 18 mois ou plus avant la visite de sélection seront éligibles. a. Les participants qui ne tolèrent pas le givinostat et qui ont interrompu leur traitement sont admissibles à l’inscription si la date de la dernière dose est ≥ 30 jours par rapport à la date de sélection. Le givinostat ne doit pas être arrêté, s’il est bien toléré, afin de satisfaire aux critères d’inclusion dans l’étude c. L’utilisation de deflazacort (nom de marque Emflaza) ou de vamorolone (nom de marque Agamree) dans des juridictions où ces produits sont expérimentaux car ils n’ont pas reçu d’autorisation de mise sur le marché des autorités sanitaires ne sera pas considéré comme un critère d’exclusion ; toutefois, la participation simultanée à une étude clinique de deflazacort ou de vamorolone sera exclue.33French (Belgium)97372893e4de5d2-2a85-4bad-a556-4ea75d37edeb11. Participación en curso en cualquier otro estudio terapéutico o estudio de seguimiento para una intervención terapéutica, tratamiento previo con un medicamento para terapia génica en investigación (que no sea delandistrogene moxeparvovec) < 24 meses antes de la visita de selección, o administración de una dosis estable de una terapia aprobada de salto de exón o cualquier medicación indicada para la DMD (que no sean corticosteroides como la vamorolona y givinostat, de acuerdo con la excepción 11b a continuación) en los 6 meses previos a la visita de selección. a. Serán aptos aquellos participantes que hayan recibido un medicamento para terapia génica ya comercializado (es decir, moxeparvovec delandistrogénico) > 18 meses antes de la selección cuyas pruebas de función muscular se hayan estabilizado o hayan presentado un deterioro según las valoraciones del investigador y lo documentado en la historia clínica ≥ 3 meses antes de la selección. b. Serán aptos aquellos participantes que reciban una dosis estable de givinostat (nombre comercial: Duvyzat) durante al menos 18 meses o más antes de la visita de selección. a. Los participantes que no toleren givinostat y que hayan interrumpido el tratamiento podrán inscribirse si la fecha de la última dosis es ≥ 30 días antes de la fecha de la visita de selección. El tratamiento con givinostat no se debe suspender, si se tolera, para cumplir con los criterios de ingreso al estudio. c. No será un criterio de exclusión el uso de deflazacort (nombre comercial: Emflaza) o vamorolona (nombre comercial: Agamree) en jurisdicciones en las que estén en fase de investigación, ya que no han recibido la autorización de comercialización de las autoridades sanitarias; sin embargo, la participación simultánea en un ensayo clínico de deflazacort o vamorolona sí supondrá un criterio de exclusión.7Spanish97372933e4de5d2-2a85-4bad-a556-4ea75d37edeb11. Bieżący udział w jakimkolwiek innym terapeutycznym badaniu klinicznym lub badaniu obserwacyjnym po interwencji terapeutycznej; wcześniejsze leczenie badanym produktem terapii genowej (innym niż delandistrogene moxeparvovec) < 24 miesięcy przed wizytą przesiewową; lub przyjmowanie stabilnej dawki zatwierdzonej terapii pomijającej egzon bądź jakiegokolwiek innego leku wskazanego w leczeniu DMD (z wyjątkiem kortykosteroidów, w tym wamorolonu i giwinostat zgodnie z wyjątkiem 11b poniżej) w ciągu 6 miesięcy przed wizytą przesiewową. a. Uczestnicy, którzy otrzymali komercyjnie dostępny produkt terapii genowej (tj. delandistrogene moxeparvovec) > 18 miesięcy przed wizytą przesiewową, u których testy czynności mięśniowej ustabilizowały się lub wykazały pogorszenie, zgodnie z oceną badacza i potwierdzone w dokumentacji medycznej ≥ 3 miesiące przed wizytą przesiewową, będą kwalifikować się do udziału w badaniu. b. Uczestnicy przyjmujący stabilną dawkę givinostatu (nazwa handlowa: Duvyzat) przez co najmniej 18 miesięcy przed wizytą przesiewową lub dłużej będą kwalifikować się do udziału. a. Uczestnicy, którzy nie tolerowali givinostatu i przerwali leczenie kwalifikują się do udziału w badaniu, jeżeli od daty przyjęcia ostatniej dawki upłynęło ≥ 30 dni przed wizytą przesiewową. Nie należy przerywać leczenia givinostatem, jeśli jest tolerowane, aby spełnić kryteria włączenia do badania. c. Stosowanie deflazacortu (nazwa handlowa: Emflaza) lub vamorolonu (nazwa handlowa: Agamree) w jurysdykcjach, w których produkty te pozostają w fazie badań i nie uzyskały jeszcze pozwolenia na dopuszczenie do obrotu, nie stanowi kryterium wykluczenia; jednak jednoczesny udział w badaniu klinicznym z zastosowaniem deflazacortu lub vamorolonu stanowi kryterium wykluczenia.19Polish97372923e4de5d2-2a85-4bad-a556-4ea75d37edeb11. Lopende deelname aan een ander klinisch onderzoek naar een therapie of vervolgonderzoek na een therapeutische ingreep, voorafgaande behandeling met een onderzoeksmiddel voor gentherapie (anders dan delandistrogene moxeparvovec) < 24 maanden voorafgaand aan het Screeningbezoek, of het krijgen van een stabiele dosis van een goedgekeurde exon-skipping-therapie of een voor DMD geïndiceerd geneesmiddel (anders dan corticosteroïden, inclusief vamorolon en givinostat, overeenkomstig uitzondering 11b hieronder) binnen 6 maanden voorafgaand aan het Screeningbezoek. a. Personen die > 18 maanden voorafgaand aan screening een in de handel verkrijgbaar middel voor gentherapie (delandistrogene moxeparvovec) hebben ontvangen en wier spierfuncties ≥ 3 maanden voorafgaand aan screening zijn gestabiliseerd of aangetoond afgenomen, zoals vastgesteld door de onderzoeker en gedocumenteerd in het medisch dossier, komen in aanmerking voor deelname. b. Personen die ten minste 18 maanden of langer voorafgaand aan het Screeningbezoek een stabiele dosis givinostat (merknaam Duvyzat) kregen, komen in aanmerking voor deelname. a. Personen die givinostat niet kunnen verdragen en die met de behandeling zijn gestopt, komen in aanmerking voor deelname als de datum van de laatste dosis ≥ 30 dagen vóór die van de screening ligt. Givinostat mag, indien het verdragen wordt, niet stopgezet worden om aan de instapcriteria van de studie te voldoen c. Het gebruik van deflazacort (merknaam Emflaza) of vamorolon (merknaam Agamree) in rechtsgebieden waar hier onderzoek naar wordt gedaan omdat ze geen handelsvergunning van een gezondheidsautoriteit hebben ontvangen, vormen geen reden voor uitsluiting; gelijktijdige deelname aan een klinisch onderzoek met deflazacort of vamorolon is echter uitgesloten.36Dutch (Belgium)13660494Received SAT-3247 in another study.9737297848aa54d-3f96-4b87-b896-9339f34b63eeHet hebben gekregen van SAT-3247 in een ander onderzoek.36Dutch (Belgium)9737295848aa54d-3f96-4b87-b896-9339f34b63ee12. Réception de SAT-3247 dans le cadre d’une autre étude.33French (Belgium)9737294848aa54d-3f96-4b87-b896-9339f34b63ee12. Uprzednie uczestnictwo w badaniu z zastosowaniem SAT-3247.19Polish9737298848aa54d-3f96-4b87-b896-9339f34b63eePacientes que hayan recibido SAT-3247 en otro estudio.7Spanish9737296848aa54d-3f96-4b87-b896-9339f34b63ee12. SAT-3247 wurde in einer anderen Studie erhalten.28German (Belgium)13660505Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator.9737299c24839d9-6102-4c12-a7b2-ed2d8f7f5871Problemas graves cognitivos o de comportamiento que impidan la participación en el estudio, a juicio del investigador.7Spanish9737303c24839d9-6102-4c12-a7b2-ed2d8f7f587113. Schwere Verhaltensstörungen oder kognitive Probleme, die nach Ansicht des Prüfarztes eine Teilnahme an der Studie ausschließen.28German (Belgium)9737300c24839d9-6102-4c12-a7b2-ed2d8f7f587113. Poważne problemy behawioralne lub poznawcze, które w ocenie badacza uniemożliwiają udział w badaniu.19Polish9737302c24839d9-6102-4c12-a7b2-ed2d8f7f5871Ernstige gedrags- of cognitieve problemen die naar de mening van de onderzoeker deelname aan het onderzoek uitsluiten.36Dutch (Belgium)9737301c24839d9-6102-4c12-a7b2-ed2d8f7f587113. Problèmes comportementaux ou cognitifs graves qui empêchent la participation à l’étude, de l’avis de l’investigateur.33French (Belgium)13660516Positive test for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCV) or HIV at Screening Visit.9737305db682db8-30d9-413d-9f9c-ca168c90590aPositief testresultaat voor hepatitis B surface antigen (HBsAg), antistof voor hepatitis C-virus (HCV) of hiv tijdens Screeningbezoek.36Dutch (Belgium)9737304db682db8-30d9-413d-9f9c-ca168c90590a14. Positiver Test auf Hepatitis-B-Oberflächenantigen (HBsAg), Hepatitis-C-Antikörper (HCV) oder HIV bei der Screening-Visite.28German (Belgium)9737308db682db8-30d9-413d-9f9c-ca168c90590a14. Pozytywny wynik testu na obecność antygenu powierzchniowego wirusa zapalenia wątroby typu B (HBsAg), przeciwciał przeciwko wirusowi zapalenia wątroby typu C (HCV) lub wirusowi HIV podczas wizyty przesiewowej.19Polish9737307db682db8-30d9-413d-9f9c-ca168c90590a14. Test positif pour l’antigène de surface de l’hépatite B (HBsAg), l’anticorps de l’hépatite C (HCV) ou le VIH lors de la visite de sélection.33French (Belgium)9737306db682db8-30d9-413d-9f9c-ca168c90590aPrueba positiva del antígeno de superficie de la hepatitis B (HBsAg), del anticuerpo de la hepatitis C (VHC) o del VIH en la visita de selección.7Spanish13660527Employee of the Sponsor, the CRO and/or study site or their relatives97373133e13a4dd-04d0-486d-8843-0280a5ae4c2b15. Mitarbeiter des Sponsors, der CRO und/oder des Studienzentrums oder deren Angehörige.28German (Belgium)97373103e13a4dd-04d0-486d-8843-0280a5ae4c2bWerknemers van de opdrachtgever, de CRO en/of onderzoekscentrum en hun familieleden.36Dutch (Belgium)97373093e13a4dd-04d0-486d-8843-0280a5ae4c2bEmpleado/a del promotor, de la CRO y/o del centro del estudio o sus familiares.7Spanish97373123e13a4dd-04d0-486d-8843-0280a5ae4c2b15. Employés du promoteur, de la CRO et / ou du site de l’étude ou leurs proches.33French (Belgium)97373113e13a4dd-04d0-486d-8843-0280a5ae4c2b15. Pracownik Sponsora, organizacji prowadzącej badanie (CRO) i/lub ośrodka badawczego, lub osoba spokrewniona z którymkolwiek z nich.19Polish13660538Presence of acute or chronic illness or history of chronic illness (other than DMD) sufficient to invalidate participation in the trial or make it unnecessarily hazardous in the judgment of the investigator.97373155ff74ed1-f4a1-405c-9bc3-aed61dd549d4Presencia de enfermedad aguda o crónica o antecedentes de enfermedad crónica (distinta de DMD) suficiente para invalidar la participación en el estudio o hacerla innecesariamente peligrosa a juicio del investigador.7Spanish97373175ff74ed1-f4a1-405c-9bc3-aed61dd549d42. Vorliegen einer akuten oder chronischen Krankheit oder einer chronischen Krankheit in der Anamnese (außer DMD), die nach dem Urteil des Prüfarztes die Teilnahme an der Studie unmöglich macht oder unnötig gefährlich macht.28German (Belgium)97373185ff74ed1-f4a1-405c-9bc3-aed61dd549d4Een acute of chronische ziekte of voorgeschiedenis van een chronische ziekte (anders dan DMD) die naar het oordeel van de onderzoeker ernstig genoeg is om deelname aan het proces onmogelijk of onnodig gevaarlijk te maken.36Dutch (Belgium)97373145ff74ed1-f4a1-405c-9bc3-aed61dd549d42. Występowanie ostrej lub przewlekłej choroby bądź historia przewlekłej choroby (innej niż DMD), która w ocenie badacza mogłaby uniemożliwić udział w badaniu lub uczynić go niepotrzebnie ryzykownym.19Polish97373165ff74ed1-f4a1-405c-9bc3-aed61dd549d42. Présence d’une maladie aiguë ou chronique ou d’antécédents de maladie chronique (autre que la DMD) de nature à invalider la participation à l’étude ou à la rendre inutilement dangereuse, selon le jugement de l’investigateur.33French (Belgium)13660549Prior or ongoing medical condition (e.g., concomitant illness, psychiatric condition, behavioral disorder), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow up would be completed, or could impair the assessment of study results.9737322b7c64826-10ca-4cd7-8ffa-7e033764a6623. Obecność lub przebieg choroby (np. choroby współistniejące, zaburzenia psychiatryczne, zaburzenia zachowania), historia medyczna, wyniki badania fizykalnego, EKG lub nieprawidłowości laboratoryjne, które zdaniem badacza mogłyby negatywnie wpłynąć na bezpieczeństwo uczestnika, utrudnić ukończenie leczenia lub obserwacji, bądź zakłócić ocenę wyników badania.19Polish9737321b7c64826-10ca-4cd7-8ffa-7e033764a662Een voorafgaande of voortdurende medische aandoening (bijv. gelijktijdige ziekte, psychiatrische aandoening, gedragsstoornis), medische voorgeschiedenis, lichamelijke verschijnselen, ecg-bevindingen of afwijkende laboratoriumresultaten die naar het oordeel van de onderzoeker de veiligheid van de deelnemer nadelig zouden kunnen beïnvloeden, het onwaarschijnlijk maken dat de behandeling of follow-up zal worden afgerond of de beoordeling van de onderzoeksresultaten zouden kunnen beïnvloeden.36Dutch (Belgium)9737323b7c64826-10ca-4cd7-8ffa-7e033764a6623. État de santé antérieur ou actuel (par exemple, maladie concomitante, état psychiatrique, trouble du comportement), antécédents médicaux, résultats cliniques, résultats ECG ou anomalies de laboratoire qui, de l’avis de l’investigateur, pourraient nuire à la sécurité du participant, rendre improbable l’achèvement du traitement ou du suivi, ou nuire à l’évaluation des résultats de l’étude.33French (Belgium)9737320b7c64826-10ca-4cd7-8ffa-7e033764a6623. Frühere oder aktuelle Erkrankungen (z. B. Begleiterkrankungen, psychiatrische Erkrankungen, Verhaltensstörungen), Anamnese, körperliche Befunde, EKG-Befunde oder Laboranomalien, die nach Ansicht des Prüfarztes die Sicherheit des Teilnehmers beeinträchtigen könnten, die es unwahrscheinlich machen, dass die Behandlung oder die Nachuntersuchung abgeschlossen werden kann, oder die die Bewertung der Studienergebnisse beeinträchtigen könnten.28German (Belgium)9737319b7c64826-10ca-4cd7-8ffa-7e033764a662Afección médica previa o en curso (por ejemplo, enfermedad concomitante, afección psiquiátrica o trastorno del comportamiento), antecedentes médicos, hallazgos físicos, hallazgos en el ECG o anomalías en las pruebas de laboratorio que, a juicio del investigador, puedan afectar negativamente a la seguridad del participante, dificulten la finalización del tratamiento o el seguimiento, o puedan perjudicar la evaluación de los resultados del estudio.7Spanish9336321The primary efficacy endpoint is defined as the change from baseline in muscle force measurements as determined by dynamometry at Week 12.True973717319c0199a-3aaa-4ad0-a4e8-b09c4e9f76dfLe critère primaire d’évaluation de l’efficacité est défini comme le changement par rapport à la valeur de début d’étude des mesures de la force musculaire déterminées par dynamométrie à la semaine 12.33French (Belgium)973717019c0199a-3aaa-4ad0-a4e8-b09c4e9f76dfPierwszorzędowy punkt końcowy skuteczności zdefiniowano jako zmianę względem wartości wyjściowej w pomiarach siły mięśniowej, określonych za pomocą dynamometrii, w 12. tygodniu.19Polish973717219c0199a-3aaa-4ad0-a4e8-b09c4e9f76dfDer primäre Wirksamkeitsendpunkt ist definiert als die Veränderung der dynamometrisch gemessenen Muskelkraft in Woche 12 im Vergleich zur Baseline.28German (Belgium)973716919c0199a-3aaa-4ad0-a4e8-b09c4e9f76dfEl criterio de valoración principal de la eficacia se define como el cambio respecto al valor basal en las mediciones de la fuerza muscular mediante dinamometría en la semana 12.7Spanish973717119c0199a-3aaa-4ad0-a4e8-b09c4e9f76dfHet primaire eindpunt voor werkzaamheid wordt gedefinieerd als de verandering ten opzichte van de nulmeting voor spierkracht, zoals gemeten met dynamometrie in Week 12.36Dutch (Belgium)9336332Safety endpoints include incidence, severity, and relationship to SAT-3247 of adverse events as well as occurrence of clinically significant changes in physical examination, clinical laboratory measures, vital signs, ECG, and C-SSRSTrue973717770943981-4bb4-4122-af9f-68b6b5f652a6Zu den Sicherheitsendpunkten gehören das Auftreten, der Schweregrad und die Beziehung zu SAT-3247 von unerwünschten Ereignissen sowie das Auftreten klinisch signifikanter Veränderungen bei der körperlichen Untersuchung, bei klinischen Labormessungen, bei den Vitalparametern, beim EKG und beim C-SSRS.28German (Belgium)973717670943981-4bb4-4122-af9f-68b6b5f652a6Los criterios de valoración de la seguridad incluyen la incidencia, la gravedad y la relación con SAT-3247 de los acontecimientos adversos, así como la aparición de cambios clínicamente significativos en la exploración física, las pruebas de laboratorio, las constantes vitales, el ECG y la Columbia-Suicide Severity Rating Scale (C-SSRS).7Spanish973717870943981-4bb4-4122-af9f-68b6b5f652a6Les critères d’évaluation de la sécurité comprennent l’incidence, la gravité et la relation avec le SAT-3247 des événements indésirables ainsi que l’apparition de changements cliniquement significatifs dans l’examen clinique, les mesures cliniques de laboratoire, les signes vitaux, l’ECG et le C-SSRS.33French (Belgium)973717570943981-4bb4-4122-af9f-68b6b5f652a6Punkty końcowe dotyczące bezpieczeństwa obejmują częstość występowania, nasilenie i związek ze stosowaniem SAT-3247 zdarzeń niepożądanych, a także występowanie klinicznie istotnych zmian w badaniu fizykalnym, laboratoryjnych pomiarach klinicznych, parametrach życiowych, EKG oraz skali C-SSRS.19Polish973717470943981-4bb4-4122-af9f-68b6b5f652a6Veiligheidseindpunten zijn onder meer incidentie, ernst en relatie met SAT-3247 van bijwerkingen, evenals het optreden van klinisch significante veranderingen bij lichamelijk onderzoek, klinische laboratoriummetingen, vitale functies, ecg en C-SSRS.36Dutch (Belgium)9336341Changes from baseline in intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in vastus lateralis at Week 12False9737181f0df4176-1260-4cb0-ae8d-98d7a8f5776bVeränderungen des intramuskulären Fettanteils in der quantitativen Magnetresonanz (qMR) im Vastus lateralis in Woche 12 gegenüber der Baseline.28German (Belgium)9737179f0df4176-1260-4cb0-ae8d-98d7a8f5776bVeranderingen ten opzichte van de nulmeting voor de kwantitatieve magnetische resonantie (qMR) in de intramusculaire vetfractie in spierweefsel van de vastus lateralis na 12 weken.36Dutch (Belgium)9737182f0df4176-1260-4cb0-ae8d-98d7a8f5776bVariaciones con respecto al valor basal en la fracción de grasa intramuscular mediante resonancia magnética cuantitativa (qMR) muscular en el vasto lateral en la semana 12.7Spanish9737183f0df4176-1260-4cb0-ae8d-98d7a8f5776bLes changements par rapport à la valeur de début d’étude de la fraction de graisse intramusculaire dans la résonance magnétique quantitative (IRMq) du muscle vaste latéral à la semaine 12.33French (Belgium)9737180f0df4176-1260-4cb0-ae8d-98d7a8f5776bZmiany względem wartości wyjściowej w frakcji tłuszczu śródmięśniowego w ilościowym rezonansie magnetycznym (qMR) mięśnia obszernego bocznego w 12. tygodniu.19Polish9336352Changes from baseline in proton muscle transverse relaxation time (T2) in vastus lateralis at Week 12.False9737184b8d27f0a-5ad5-45c2-9ad2-5943f475c94bVeranderingen ten opzichte van de nulmeting voor transversale relaxatietijd van protonen in spierweefsel (T2) van de vastus lateralis in Week 12.36Dutch (Belgium)9737188b8d27f0a-5ad5-45c2-9ad2-5943f475c94bVariaciones con respecto al valor basal en el tiempo de relajación transversal de los protones (T2) en el músculo vasto lateral en la semana 12.7Spanish9737187b8d27f0a-5ad5-45c2-9ad2-5943f475c94bLes changements par rapport à la valeur de début d’étude dans le temps de relaxation transversale du muscle protonique (T2) dans le muscle vaste latéral à la semaine 12.33French (Belgium)9737186b8d27f0a-5ad5-45c2-9ad2-5943f475c94bZmiany względem wartości wyjściowej w czasie relaksacji poprzecznej protonów (T2) w mięśniu obszernym bocznym w 12. tygodniu.19Polish9737185b8d27f0a-5ad5-45c2-9ad2-5943f475c94bVeränderungen der transversalen Relaxationszeit (T2) der Muskelprotonen im Vastus lateralis in Woche 12 gegenüber der Baseline.28German (Belgium)9336363Changes from baseline in Regeneration Index in open muscle biopsy of the biceps brachii at Week 12False97371918c90e126-35ae-4d72-aa40-1ce65a8e357aZmiany względem wartości wyjściowej w wskaźniku regeneracji w otwartej biopsji mięśnia dwugłowego ramienia w 12. tygodniu19Polish97371908c90e126-35ae-4d72-aa40-1ce65a8e357aVariaciones con respecto al valor basal en el índice de regeneración en biopsia muscular abierta del bíceps braquial en la semana 12.7Spanish97371938c90e126-35ae-4d72-aa40-1ce65a8e357aVeranderingen ten opzichte van de nulmeting voor regeneratie-index in open spierbiopt van de biceps brachii in Week 12.36Dutch (Belgium)97371928c90e126-35ae-4d72-aa40-1ce65a8e357aLes changements par rapport à la valeur de début d’étude de l’indice de régénération dans une biopsie musculaire ouverte du biceps brachial à la semaine 12.33French (Belgium)97371898c90e126-35ae-4d72-aa40-1ce65a8e357aVeränderungen des Regenerationsindex bei offener Muskelbiopsie des Musculus biceps brachii in Woche 12 gegenüber der Baseline.28German (Belgium)9336374Changes from baseline in function as determined by NSAA assessment at Week 12False9737195597000f9-bfbc-4718-8c62-c48b42e23388Veranderingen ten opzichte van de nulmeting voor functionaliteit zoals bepaald met een NSAA-beoordeling in Week 12.36Dutch (Belgium)9737196597000f9-bfbc-4718-8c62-c48b42e23388Zmiany względem wartości wyjściowej w zakresie funkcji, określone na podstawie oceny NSAA, w 12. tygodniu.19Polish9737194597000f9-bfbc-4718-8c62-c48b42e23388Variaciones con respecto al valor basal en la función según la evaluación con la escala North Star Ambulatory Assessment (NSAA) en la semana 12.7Spanish9737197597000f9-bfbc-4718-8c62-c48b42e23388Les changements par rapport à la valeur de début d’étude dans la fonction déterminée par le test NSAA à la semaine 12.33French (Belgium)9737198597000f9-bfbc-4718-8c62-c48b42e23388Veränderungen der Funktion gegenüber der Baseline, ermittelt durch NSAA-Beurteilung in Woche 12.28German (Belgium)9336385Changes from baseline in SV95C at Week 12False9737201c17b2d7f-0a79-4011-967d-3f27212d1e35Veranderingen ten opzichte van de nulmeting voor SV95C in Week 12.36Dutch (Belgium)9737202c17b2d7f-0a79-4011-967d-3f27212d1e35Variaciones respecto al valor basal en SV95C en la semana 12.7Spanish9737203c17b2d7f-0a79-4011-967d-3f27212d1e35Veränderungen des SV95C-Werts gegenüber der Baseline in Woche 12.28German (Belgium)9737200c17b2d7f-0a79-4011-967d-3f27212d1e35Zmiany względem wartości wyjściowej w SV95C w 12. tygodniu.19Polish9737199c17b2d7f-0a79-4011-967d-3f27212d1e35Les changements par rapport à la valeur de début d’étude dans le SV95C à la semaine 12.33French (Belgium)9336396Exploratory end point: change from baseline in inflammatory cytokine profile at Week 12False9737205de2f2e77-efab-4283-be69-dd712635b558Verkennend eindpunt: verandering ten opzichte van de nulmeting voor inflammatoir cytokineprofiel in Week 12.36Dutch (Belgium)9737207de2f2e77-efab-4283-be69-dd712635b558Eksploracyjny punkt końcowy: Zmianę względem wartości wyjściowej w profilu cytokin zapalnych w 12. tygodniu.19Polish9737208de2f2e77-efab-4283-be69-dd712635b558Critère d’évaluation exploratoire: le changement par rapport à la valeur de début d’étude dans le profil des cytokines inflammatoires à la semaine 12.33French (Belgium)9737204de2f2e77-efab-4283-be69-dd712635b558Explorativer Endpunkt: veränderung des entzündlichen Zytokinprofils in Woche 12 gegenüber der Baseline.28German (Belgium)9737206de2f2e77-efab-4283-be69-dd712635b558Criterio de valoración exploratoria: variación respecto al valor basal en perfil de citoquinas inflamatorias en la semana 12.7Spanish9336407Exploratory end point: change from baseline in creatine kinase at Week 12False973720915724a1c-6ff6-4da2-aaad-a3338eba6dbaCriterio de valoración exploratoria: variación respecto al valor basal en la creatina cinasa en la semana 12.7Spanish973721015724a1c-6ff6-4da2-aaad-a3338eba6dbaCritère d’évaluation exploratoire: le changement par rapport à la valeur de début d’étude de la créatine kinase à la semaine 12.33French (Belgium)973721315724a1c-6ff6-4da2-aaad-a3338eba6dbaExplorativer Endpunkt: veränderung der Kreatinkinase in Woche 12 gegenüber der Baseline.28German (Belgium)973721215724a1c-6ff6-4da2-aaad-a3338eba6dbaVerkennend eindpunt: verandering ten opzichte van de nulmeting voor creatinekinase in Week 12.36Dutch (Belgium)973721115724a1c-6ff6-4da2-aaad-a3338eba6dbaEksploracyjny punkt końcowy: Zmianę względem wartości wyjściowej poziomu kinazy kreatynowej w 12. tygodniu.19Polish9336418Exploratory end point: change from baseline in maximum percent predicted forced vial capacity as measured by spirometry at Week 12False97372176345ae9b-4d30-4f9d-92d0-b229ca4f58b9Explorativer Endpunkt: veränderung gegenüber der Baseline der maximalen prognostizierten forcierten Vitalkapazität in Prozent, gemessen durch Spirometrie in Woche 12.28German (Belgium)97372166345ae9b-4d30-4f9d-92d0-b229ca4f58b9Eksploracyjny punkt końcowy: Zmianę względem wartości wyjściowej maksymalnej procentowej przewidywanej wymuszonej pojemności życiowej (FVC), mierzonej za pomocą spirometrii, w 12. tygodniu.19Polish97372146345ae9b-4d30-4f9d-92d0-b229ca4f58b9Verkennend eindpunt: Verandering ten opzichte van de nulmeting voor het maximale percentage voorspelde geforceerde vitale capaciteit zoals gemeten met spirometrie in Week 12.36Dutch (Belgium)97372156345ae9b-4d30-4f9d-92d0-b229ca4f58b9Critère d’évaluation exploratoire: le changement par rapport à la valeur de début d’étude du pourcentage maximal de la capacité respiratoire forcée prédite, mesurée par spirométrie à la semaine 12.33French (Belgium)97372186345ae9b-4d30-4f9d-92d0-b229ca4f58b9Criterio de valoración exploratoria: variación respecto al valor basal en el porcentaje máximo previsto de capacidad vital forzada medido por espirometría en la semana 12.7Spanish9336429Exploratory end point: change in biceps brachii muscle fiber size and fiber size distribution as determined from histopathology at 12 weeksFalse97372198dafafda-e529-4c90-a446-f8425f61fc2fCriterio de valoración exploratoria: variación en el tamaño de las fibras musculares del bíceps braquial y en la distribución del tamaño de las fibras, determinados a partir de la histopatología al cabo de 12 semanas.7Spanish97372218dafafda-e529-4c90-a446-f8425f61fc2fCritère d’évaluation exploratoire: le changement de la taille des fibres musculaires du biceps brachial et de la distribution de la taille des fibres, déterminées par histopathologie à 12 semaines.33French (Belgium)97372228dafafda-e529-4c90-a446-f8425f61fc2fVerkennend eindpunt: verandering in omvang en verdeling van spiervezels van de biceps brachii zoals bepaald op grond van histopathologie na 12 weken.36Dutch (Belgium)97372238dafafda-e529-4c90-a446-f8425f61fc2fExplorativer Endpunkt: veränderung der Muskelfasergröße und Fasergrößenverteilung des Musculus biceps brachii, ermittelt durch Histopathologie nach 12 Wochen.28German (Belgium)97372208dafafda-e529-4c90-a446-f8425f61fc2fEksploracyjny punkt końcowy: Zmianę wielkości włókien mięśniowych mięśnia dwugłowego ramienia oraz rozkładu ich wielkości, określoną na podstawie badania histopatologicznego po 12 tygodniach.19Polish93364310Exploratory end point: change in the proportion of embryonic myosin positive fibers as determined from histopathology at 12 weeks.False97372257f3b32a5-0be3-4ead-acc3-a4c3335716caCriterio de valoración exploratoria: variación en la proporción de fibras positivas a la miosina embrionaria determinado por histopatología al cabo de 12 semanas.7Spanish97372267f3b32a5-0be3-4ead-acc3-a4c3335716caExplorativer Endpunkt: veränderung des Anteils der embryonalen Myosin-positiven Fasern, ermittelt durch Histopathologie nach 12 Wochen.28German (Belgium)97372247f3b32a5-0be3-4ead-acc3-a4c3335716caEksploracyjny punkt końcowy: Zmianę odsetka włókien dodatnich względem zarodkowej miozyny, określoną na podstawie badania histopatologicznego w 12. tygodniu.19Polish97372287f3b32a5-0be3-4ead-acc3-a4c3335716caCritère d’évaluation exploratoire: le changement dans la proportion de fibres embryonnaires positives à la myosine, déterminé par l’histopathologie à 12 semaines.33French (Belgium)97372277f3b32a5-0be3-4ead-acc3-a4c3335716caVerkennend eindpunt: verandering in het aandeel van embryonale myosine-positieve vezels zoals bepaald op grond van histopathologie na 12 weken.36Dutch (Belgium)93364411Exploratory end point: change in the number of satellite cells as determined from histopathology at 12 weeksFalse9737229169f8f53-e9e2-41c4-9ff1-cb42ed222dcbVerkennend eindpunt: verandering in het aantal satellietcellen zoals bepaald op grond van histopathologie na 12 weken.36Dutch (Belgium)9737233169f8f53-e9e2-41c4-9ff1-cb42ed222dcbExplorativer Endpunkt; veränderung der Anzahl der Satellitenzellen, ermittelt durch Histopathologie nach 12 Wochen.28German (Belgium)9737230169f8f53-e9e2-41c4-9ff1-cb42ed222dcbCritère d’évaluation exploratoire: le changement dans le nombre de cellules satellites tel que déterminé par l’histopathologie à 12 semaines.33French (Belgium)9737232169f8f53-e9e2-41c4-9ff1-cb42ed222dcbEksploracyjny punkt końcowy: Zmianę liczby komórek satelitarnych, określoną na podstawie badania histopatologicznego po 12 tygodniach.19Polish9737231169f8f53-e9e2-41c4-9ff1-cb42ed222dcbCriterio de valoración exploratoria: variación en el número de células satélite determinado por histopatología al cabo de 12 semanas.7Spanish93364512Exploratory end point: change in endomysial fibrosis and adipose tissue infiltration as determined from histopathology at 12 weeks.False97372358029651e-b04f-483d-9869-d2b8c257db1dCritère d’évaluation exploratoire: le changement de la fibrose endomysiale et de l’infiltration du tissu adipeux, déterminées par histopathologie à 12 semaines.33French (Belgium)97372368029651e-b04f-483d-9869-d2b8c257db1dExplorativer Endpunkt: veränderung der endomysialen Fibrose und der Fettgewebsinfiltration, ermittelt durch Histopathologie nach 12 Wochen.28German (Belgium)97372378029651e-b04f-483d-9869-d2b8c257db1dCriterio de valoración exploratoria: variación en la fibrosis endomisial y la infiltración de tejido adiposo determinado por histopatología al cabo de 12 semanas.7Spanish97372348029651e-b04f-483d-9869-d2b8c257db1dVerkennend eindpunt: Verandering in endomysiale fibrose en infiltratie van vetweefsel zoals bepaald op grond van histopathologie na 12 weken.36Dutch (Belgium)97372388029651e-b04f-483d-9869-d2b8c257db1dEksploracyjny punkt końcowy: Zmianę stopnia zwłóknienia śródmięśniowego oraz nacieku tkanki tłuszczowej, określoną na podstawie badania histopatologicznego po 12 tygodniach.19Polish93364613Exploratory end point: change in NSAA score over 12 weeks as compared to natural historyFalse97372426f017bf0-6459-4749-871c-3a1058d18206Critère d’évaluation exploratoire: le changement du score NSAA sur 12 semaines par rapport à l’évolution naturelle.33French (Belgium)97372416f017bf0-6459-4749-871c-3a1058d18206Explorativer Endpunkt: veränderung des NSAA-Scores über 12 Wochen im Vergleich zum natürlichen Verlauf.28German (Belgium)97372396f017bf0-6459-4749-871c-3a1058d18206Eksploracyjny punkt końcowy: Zmianę wyniku skali NSAA w ciągu 12 tygodni w porównaniu z historią naturalnego przebiegu choroby.19Polish97372406f017bf0-6459-4749-871c-3a1058d18206Verkennend eindpunt: Verandering in NSAA-score over 12 weken in vergelijking met die bij het natuurlijke beloop van de ziekte.36Dutch (Belgium)97372436f017bf0-6459-4749-871c-3a1058d18206Criterio de valoracación exploratoria: variación en la puntuación de NSAA durante 12 semanas en comparación con la evolución natural de la enfermedad.7Spanish2027-03-312027-03-312025-12-1586458Satellos Bioscience Inc325535222PatientsFalseTrueTrue2Individual participant data will not be shared, as the sponsor does not plan to make the dataset available outside the study team1719431Study period 1 - ScreeningParticipants will be screened to evaluate their eligibility within 28 days before initiating dosing of investigational product at Baseline. During the Screening Visit, participants will be also equipped with a wearable device for assessment of their stride velocity. Participants will be trained on proper usage and will be instructed to wear one sensor on each ankle until Baseline/Visit 2.331719442Study Period 2 – TreatmentStart event: Randomisation (Visit 2) End event: Final study visit (Visit 5) Duration: Approximately 12 weeks During this period participants will complete a Baseline visit (Visit 2), Week 4 (Visit 3), Week 8 (Visit 4), and Week 12 (Visit 5) follow-up visits. Description: Randomised treatment and study assessments During baseline visit (Visit 2), eligible participants will be randomized 1:1:1 to one of three treatment groups: SAT-3247 60 mg SAT-3247 120 mg and/or matched placebo administered once daily, by mouth (PO), for 12 weeks in a blinded manner and stratified by: • Prior DMD treatments (gene therapy, exon skipper, givinostat, or none) • Baseline corticosteroid regimen (either daily or weekend dose regimen) During visit 4 Visit, participants will be also equipped again with a wearable device for assessment of their stride velocity. Participants will be trained on proper usage and will be instructed to wear one sensor on each ankle until Visit 5.21719493Monitor1719474Analyst1719452Investigator1719485Carer1719461SubjectRandomization will be conducted using a computer-generated random sequence schema; randomization assignment will be determined by the programmed Randomization and Trial Supply Management (RTSM) platform. In the event that unblinding is required, (e.g., for a serious adverse event requiring knowledge of treatment assignment), the process is managed through the RTSM platform with notification to and approval from the study medical monitor. Unblinding would be performed on a per-participant basis, preserving the integrity of the overall study blind. All unblinding events are logged within the RTSM and are strictly limited to circumstances in which participant safety is at risk.Participants allocated to 60 mg arm, will take 1 10mg SAT-3247 tablet + 1 50mg SAT-3247 tablet + 1 10mg placebo tablet + 1 50mg placebo tablet for five consecutive days (i.e., weekdays) of each week for 12 weeks and 2 10mg placebo tablets + 2 50mg placebo tablets every weekend day for 12 weeksSAT-3247 60 mg96403Participants allocated to 120 mg arm, will take 2 10mg SAT-3247 tablets + 2 50mg SAT-3247 tablets for five consecutive days (i.e., weekdays) of each week for 12 weeks and 2 10mg placebo tablets + 2 50mg placebo tablets every weekend day for 12 weeksSAT-3247 120 mg96404Participants allocated to placebo will take 2 10mg placebo tablets + 2 50mg placebo tablets fevery day for 12 weeksPlacebo96405150105825824865238Non-EEA National Competent authority17Food And Drug AdministrationFalseTrueORG-100006066Active91625710903 New Hampshire Avenue10903 New Hampshire AvenueSilver Spring20993-0002840United StatesTrueORG-10000606650104825823865237Non-EEA National Competent authority17Medicines And Healthcare Products Regulatory AgencyFalseTrueORG-100003945Active91625610 South Colonnade10 South ColonnadeLondonE14 4PU826United KingdomTrueORG-100003945306902025-522522-13-00Satellos Bioscience Inc.A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD PatientsNON_APPLICABLENON_APPLICABLE24132Falseacceptable2026-01-222026-01-2210Diseases [C] - Nervous System Diseases [C10]5Diseases [C] - Musculoskeletal Diseases [C05]123323Duchenne muscular dystrophyTrue134204True395610PublicProject Managerc.ruiz@innopharma.it+447379634323807121Pharmaceutical company10Satellos Bioscience Inc.TrueTrueORG-100053729Active395611ScientificProject Managerc.ruiz@innopharma.it+447379634323807121Pharmaceutical company10Satellos Bioscience Inc.TrueTrueORG-100053729Active600213832310871821Laboratory/Research/Testing facility9Labor Dr. Wisplinghoff GbRTrueTrueORG-100046123Active924379Horbeller Strasse 18-20, JunkersdorfHorbeller Strasse 18-20JunkersdorfCologne508582013Germany00490221940505d.hoffman@wisplihoff.deTrueORG-10004612393363815lab safety analyses933639400490221940505d.hoffman@wisplihoff.de600220832316871827Hospital/Clinic/Other health care facility8Scout ClinicalFalseTrueORG-100042228Active92438615770 Dallas Pkwy Ste 107515770 Dallas Pkwy Ste 1075Dallas75248-3331840United States+14694802418jamie.harris@scoutclinical.comTrueORG-10004222893365015participants' travel and reimbursement+14694802418jamie.harris@scoutclinical.com600221832311871822Pharmaceutical company10Medassessment Inc.TrueTrueORG-100047373Active92438014 Calle Vista Del Sol14 Calle Vista Del SolSan Clemente92673-6913840United States+19493961483Yelena.Kamenir@MedAssessment.comTrueORG-1000473739336518+19493961483Yelena.Kamenir@MedAssessment.com600224832305871816Pharmaceutical company10Pharma Start LLCTrueTrueORG-100042396Active924374881 Busse Road Suite 310881 Busse Road Suite 310Elk Grove Village60007-2442840United States+12246005664yao.yao@FirmaClinical.comTrueORG-100042396933655109336566+12246005664yao.yao@FirmaClinical.com600223832313871824Laboratory/Research/Testing facility9Rules Based Medicine Inc.TrueTrueORG-100043610Active9243833300 Duval Road3300 Duval RoadAustin78759-3549840United States+15128358026evelyn.rivera@q2labsolutions.comTrueORG-10004361093365315cytokine panel9336544+15128358026evelyn.rivera@q2labsolutions.com600215832319871830Non-Pharmaceutical company11Mednet Solutions Inc.TrueTrueORG-100054456Active924389601 Carlson Parkway Suite 250601 Carlson Parkway Suite 250Minnetonka55305-5236840United States+17632307504abeduhn@mednetsolutions.comTrueORG-10005445693364115site payments93364239336437+17632307504abeduhn@mednetsolutions.com600216832315871826Non-Pharmaceutical company11Medidata Solutions Inc.TrueTrueORG-100016256Active924385350 Hudson Street350 Hudson StreetNew York10014-4504840United States+1maritza.barragan@3ds.comTrueORG-10001625693364415video recording+1maritza.barragan@3ds.com600214832321871832Pharmaceutical company10Clinigen Clinical Supplies Management GmbHTrueTrueORG-100016915Active924391Am Kronberger Hang 3Am Kronberger Hang 3Schwalbach Am Taunus658242013Germany+441932555620kathy.scriven@clinigengroup.comTrueORG-10001691593364014+441932555620kathy.scriven@clinigengroup.com600222832318871829Pharmaceutical company10SysnavTrueTrueORG-100026890Active92438872 Rue Emile Loubet72 Rue Emile LoubetVernon272002012France+33607951097camille.bisson@sysnav.frTrueORG-10002689093365215wearables+33607951097camille.bisson@sysnav.fr600217832307871818Laboratory/Research/Testing facility9LabConnect GmbHTrueTrueORG-100047696Active924376Horbeller Strasse 18-20, JunkersdorfHorbeller Strasse 18-20JunkersdorfCologne508582013Germany+14239002184sberry@labconnect.comTrueORG-1000476969336454+14239002184sberry@labconnect.com600226832301871812Pharmaceutical company10Biospective Inc.TrueTrueORG-100044312Active9243701255 Peel Street Bureau 5601255 Peel Street Bureau 560MontrealH3B 2T9124Canada+15143600141ellen.laurie@biospective.comTrueORG-10004431293366315Imaging+15143600141ellen.laurie@biospective.com600225825860865274Pharmaceutical company10Innopharma S.r.l.TrueTrueORG-100028170Active916311Via Lavoratori Autobianchi 1Via Lavoratori Autobianchi 1Desio208322018Italy+390362573128a.bertani@innopharma.itTrueORG-100028170933657193365811933659129336601393366159336629+390362573128a.bertani@innopharma.it600218832296871807Pharmaceutical company10Agilex Biolabs Pty LimitedTrueTrueORG-100046760Active92436528 Dalgleish Street28 Dalgleish StreetThebarton503136Australia+61883028756Robert.Hammond@agilexbiolabs.comTrueORG-10004676093364615PK9336474+61883028756Robert.Hammond@agilexbiolabs.com600219832298871809Laboratory/Research/Testing facility9Arup Laboratories Inc.TrueTrueORG-100041750Active924367500 South Chipeta Way500 South Chipeta WaySalt Lake City84108-1221840United States+18015832787marcie.traballoni@aruplab.comTrueORG-10004175093364815CK Isoenzymes9336494+18015832787marcie.traballoni@aruplab.com600212832303871814Laboratory/Research/Testing facility9Diverge Translational Science LaboratoryTrueTrueORG-100051693Active924372247 West Freshwater Way Suite 600247 West Freshwater Way Suite 600Milwaukee53204-4117840United States+1mhall@divergetsl.comTrueORG-10005169393363715Biopsy sample histology; Biopsy sample collection; site training+1mhall@divergetsl.com807121Pharmaceutical company10Satellos Bioscience Inc.TrueTrueORG-100053729Active768411807121Pharmaceutical company10Satellos Bioscience Inc.TrueTrueORG-100053729Active847382200 Bay Street Suite 2800200 Bay Street Suite 2800TorontoM5J 2J1124CanadaTrueORG-100053729TrueCommercial2This is a phase 2a trial108690210Diseases [C] - Nervous System Diseases [C10]10869035Diseases [C] - Musculoskeletal Diseases [C05]367942SAT-3247 50mg tablet, SAT-3247 10mg tablet1Test491706112753908TABLETPRD127539081SAT-3247 50mg tabletTABLETSAT-3247 OXALATESUB430452SATELLOS BIOSCIENCE INC.12753908430482SATELLOS BIOSCIENCE INC.ChemicalChemicalSAT-3247 OXALATESUB430452False1Falsemg milligram(s)120mg milligram(s)7200122IMP of chemical originPRD12753908SAT-324711ORALTrueSAT-3247 50mg tabletsat-3247 oxalateTABLET491707112753907TABLETPRD127539071SAT-3247 10mg tabletTABLETSAT-3247 OXALATESUB430452SATELLOS BIOSCIENCE INC.12753907430482SATELLOS BIOSCIENCE INC.ChemicalChemicalSAT-3247 OXALATESUB430452False1Falsemg milligram(s)120mg milligram(s)7200122IMP of chemical originPRD12753907SAT-324711ORALTrueSAT-3247 10mg tabletsat-3247 oxalateTABLET367943Placebos for SAT-3247 tablets are required for the clinical trial. To match the appearance of SAT-3247 active tablets, placebo tablets are formulated by replacing SAT-3247 oxalate DS with lactose monohydrate and microcrystalline cellulose.3Placebo4917081N/AN/AN/APlacebos for SAT-3247 tablets are required for the clinical trial. To match the appearance of SAT-3247 active tablets, placebo tablets are formulated by replacing SAT-3247 oxalate DS with lactose monohydrate and microcrystalline cellulose.N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePlacebos for SAT-3247 tablets are required for the clinical trial. 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19/05/2025, ES: 12/05/2025, BE: 16/05/2025, IT: 15/05/2025Diseases [C] - Musculoskeletal Diseases [C05]Entrada Therapeutics Inc.Pharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansPlasma, muscle, and urine concentration of ENTR-601-45 and its final metabolite (Part A and OL Period), Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A), Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A), Percent change from baseline to End of Part A in exon 45 skipping measured in muscle biopsy (Part A), Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period), Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period), Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period), Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period), Change 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synopsis_IT_2024-517499-39_Entrada_Redacted285ada21-ae70-4176-93c3-a5bec7da8def7Synopsis of the protocol (for publication)PDF1185945.04D1_Protocol Synopsis_ENG_2024-517499-39_Entrada_redacted6e92161b-e0bb-4c68-86c5-222eb23774a47Synopsis of the protocol (for publication)PDF1185945.04D1_Protocol synopsis_Spanish_2024-517584-23_Entrada_redacted99ba403a-838d-4405-9c41-9800d5fdfa117Synopsis of the protocol (for publication)PDF11859443D1_Protocol synopsis_Dutch_2024-517499-39_Entrada_redacted9eef5d29-264a-4610-ae4a-366649dda5887Synopsis of the protocol (for publication)PDF1185945.04D1_Protocol Lay synopsis__ENG_Entrada_redacted0535ae04-c330-4037-bdd9-3c437263c74a7Synopsis of the protocol (for publication)PDF11859433D1_Protocol Lay Synopsis_Dutch_2024-517499-39_Entrada_redacted0a95b78f-47d8-4dd5-943b-d1e83ba459ec7Synopsis of the protocol (for publication)PDF11859433D1_Lay Synopsis_Spanish_2024-517499-39_Entrada_redactedbd844d31-ab5e-4e12-a03c-e64fa1da25677Synopsis of the protocol (for publication)PDF11859433D1_Lay Synopsis_Italian_2024-517499-39_Entrada_Redactedc3f93ae8-7c23-4d38-9fc9-04520a2af12f7Synopsis of the protocol (for publication)PDF1185943.01Netherlands444292025-05-19T16:25:28.6112026-02-11T16:15:01.6673Italy444272025-05-15T10:01:15.252026-02-11T16:15:01.6673Spain444282025-05-12T14:18:23.5672026-02-11T16:15:01.6674Belgium444262025-05-16T14:19:24.3032026-02-11T16:15:01.667444426BelgiumSTART_OF_TRIAL2025-07-31START_OF_RECRUITMENT2025-09-2244427ItalySTART_OF_TRIAL2026-01-0844428SpainSTART_OF_TRIAL2025-07-16START_OF_RECRUITMENT2025-11-1244429NetherlandsSTART_OF_TRIAL2025-11-052024-517499-39-003A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping with an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-45, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)ENTR-601-45-20116/07/2025Duchenne Muscular DystrophyNetherlands:3Italy:3Spain:4Belgium:412/05/2025NL: 19/05/2025, ES: 12/05/2025, BE: 16/05/2025, IT: 15/05/2025Diseases [C] - Musculoskeletal Diseases [C05]Entrada Therapeutics Inc.Pharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansPlasma, muscle, and urine concentration of ENTR-601-45 and its final metabolite (Part A and OL Period), Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A), Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A), Percent change from baseline to End of Part A in exon 45 skipping measured in muscle biopsy (Part A), Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period), Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period), Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period), Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period), Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period), Change from baseline to End of OL Period in North Star Ambulatory Assessment (NSAA) (Part A and OL Period), Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) (Part A and OL Period)SODIUM CHLORIDE, ENTR-601-456540-17 years, 18-64 yearsFemale, Male314Incidence and severity of treatment emergent adverse events (TEAEs) (Part A and OL Period), Changes in vital sign measurements (Part A and OL Period), Changes in clinical laboratory results (Part A and OL Period), Changes in electrocardiogram (ECG) parameters (Part A and OL Period), Changes in physical examination findings (Part A and OL Period)No17/02/202618/02/20262024-517499-39-00Authorised2025-07-162025-05-12T14:18:23.5672026-02-18T02:40:53.008718871311859410100000000556United Kingdom826GBGBRTrue4918593SUB12581MIG-2SODIUM CHLORIDESOLUTION FOR INFUSIONSODIUM CHLORIDESUB12581MIG79225ChemicalSODIUM CHLORIDESUB12581MIG---False3FalseFalsechemicalSUB12581MIG11INTRAVENOUS INFUSIONFalseSODIUM CHLORIDEsodium chloride491860111749346SOLUTION FOR INFUSIONPRD117493461ENTR-601-45SOLUTION FOR INFUSIONENTR-601-45SUB408742ENTRADA THERAPEUTICS, INC.11749346408743ENTRADA THERAPEUTICS, INC.Nucleic AcidNucleic AcidENTR-601-45SUB408742False1FalseConjugate of a DMD exon 44 skipping phosphorodiamidate morpholino oligomer and a cyclic peptidePRD11749346DE_BW_01_MIA_2023_0054/DE_BW_01_Fisher11INTRAVENOUS INFUSIONFalseENTR-601-45entr-601-45SOLUTION FOR INFUSIONA 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping with an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-45, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)97436323869495e-ecac-44d9-940c-284c409a56f7Estudio en dos partes, aleatorizado, doble ciego y controlado con placebo en participantes con distrofia muscular de Duchenne susceptibles de omitir el exón 45 con una dosis ascendente múltiple inicial, parte A, para evaluar la seguridad, la tolerabilidad, la farmacocinética y la farmacodinámica de ENTR-601-45, seguido de una parte B para evaluar la seguridad y la eficacia de ENTR-601-45 (ELEVATE-45)7Spanish97436313869495e-ecac-44d9-940c-284c409a56f7Een 2-delig, gerandomiseerd, dubbelblind, placebogecontroleerd onderzoek bij deelnemers met spierdystrofie van Duchenne die ontvankelijk zijn voor exon 45-skipping met een initiële meervoudige oplopende dosis; deel A om de veiligheid, verdraagbaarheid, farmacokinetiek en farmacodynamiek van ENTR-601-45 te beoordelen, gevolgd door deel B om de veiligheid en werkzaamheid van ENTR-601-45 (ELEVATE-45) te beoordelen18DutchA 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)97436488489f332-c19a-4bbf-90c2-0656aa0fb775Estudio en dos partes, aleatorizado, doble ciego y controlado con placebo en participantes con distrofia muscular de Duchenne susceptibles de omitir el exón 45 para evaluar la seguridad y la eficacia de ENTR-601-45 (ELEVATE-45)7Spanish97436478489f332-c19a-4bbf-90c2-0656aa0fb775Een 2-delig, gerandomiseerd, dubbelblind, placebogecontroleerd onderzoek bij deelnemers met spierdystrofie van Duchenne die ontvankelijk zijn voor exon 45-skipping ter beoordeling van de veiligheid en werkzaamheid van ENTR-601-45 (ELEVATE-45)18DutchENTR-601-45-201488827U1111-1316-6093False72Phase I/II integrated trial108659123368Duchenne Muscular Dystrophy97435800d833443-b494-4c7d-941f-c4ed17a82ac0Distrofia muscular de Duchenne7Spanish97435790d833443-b494-4c7d-941f-c4ed17a82ac0spierdystrofie van Duchenne18DutchTrue10000001254427.1PTDuchenne muscular dystrophy10013801100000004850False10000001204720.1PTDuchenne muscular dystrophy gene carrier10052655100000004850False73588954358892535889363588969358894Part A and OL Period: To evaluate the safety and tolerability of ENTR-601-45 in participants with Duchenne muscular dystrophy (DMD)97436465fd6d5a5-cd82-4464-b82f-05ced56ec826Deel A en OL-periode: Het beoordelen van de veiligheid en verdraagbaarheid van ENTR-601-45 bij deelnemers met spierdystrofie van Duchenne (DMD)18Dutch97436455fd6d5a5-cd82-4464-b82f-05ced56ec826Parte A y período abierto: Evaluar la seguridad y tolerabilidad de ENTR601-45 en participantes con distrofia muscular de Duchenne (DMD).7Spanish4170201To characterize the pharmacokinetics of ENTR-601-45 in participants with DMD in Part A974364949d32b1b-3c3a-4134-b6c1-079cb2de4666Het beschrijven van de farmacokinetiek van ENTR-601-45 bij deelnemers met DMD in Deel A18Dutch974365049d32b1b-3c3a-4134-b6c1-079cb2de4666Definir la farmacocinética de ENTR-601-45 en participantes con DMD en la Parte A7Spanish4170212To characterize the pharmacodynamics of ENTR-601-45 in participants with DMD in Part A9743652586210a4-5d09-464c-b8a4-976dd4770909Het beschrijven van de farmacodynamiek van ENTR-601-45 bij deelnemers met DMD in Deel A18Dutch9743651586210a4-5d09-464c-b8a4-976dd4770909Definir la farmacodinámica de ENTR-601-45 en participantes con DMD en la Parte A7Spanish4170223To evaluate the immune response to ENTR-601-45 in participants with DMD (Part A and OL Period)974365419af0a22-fdc6-43c1-9358-b8a7589f0810Het beoordelen van de immuunreactie op ENTR-601-45 bij deelnemers met DMD in Deel A18Dutch974365319af0a22-fdc6-43c1-9358-b8a7589f0810Evaluar la respuesta inmunitaria a ENTR-601-45 en participantes con DMD (Parte A y período abierto)7Spanish4170234To evaluate the impact of ENTR-601-45 on measures of function in participants with DMD after extended dosing (Part A and OL Period)97436563522c14e-950e-4383-a307-ad538f448974Evaluar el efecto de ENTR-601-45 en las medidas de función en los participantes con DMD tras la administración ampliada (Parte A y período abierto)7Spanish97436553522c14e-950e-4383-a307-ad538f448974Het beoordelen van de impact van ENTR-601-45 op maten van functie bij deelnemers met DMD na lange dosering (Deel A en OL-periode)18Dutch7975611Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene that is amenable to exon 45 skipping as reviewed by a central genetic counselor.97436336509ce2e-a353-4ae3-b822-dfc9f6787e41Genetische diagnose van DMD en bevestigde pathologische variant in het dystrofinegen die geschikt is voor exon 45-skipping zoals beoordeeld door een centrale genetisch consulent.18Dutch97436346509ce2e-a353-4ae3-b822-dfc9f6787e41Diagnóstico genético de DMD y variante patológica confirmada en el gen de la distrofina susceptible de omitir el exón 45, según la revisión de un asesor genético central.7Spanish7975622Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.97436363a0f3b50-ce25-44f1-912e-7ad561b14937Hombre asignado en el nacimiento con signos clínicos compatibles con distrofia muscular de Duchenne, según determine el investigador.7Spanish97436353a0f3b50-ce25-44f1-912e-7ad561b14937Mannelijk bij de geboorte met klinische symptomen die passen bij spierdystrofie van Duchenne, zoals bepaald door de onderzoeker.18Dutch7975633Part A: 4-20 years of age9743638cea22488-e95b-4ad7-b911-c9d848a906dfParte A: 4-20 años de edad7Spanish9743637cea22488-e95b-4ad7-b911-c9d848a906dfDeel A: 4-20 jaar18Dutch7975644Ambulatory Status Part A: ambulatory with a specific Performance of the Upper Limb v2.0 (PUL 2.0) Entry item at Screening974364040313062-f7b9-49bd-ae35-fffc52ea2e62Ambulante status Deel A: ambulant met een beginwaarde van item A ≥ 3 voor Performance of the Upper Limb v2.0 (PUL 2.0) tijdens de screening18Dutch974363940313062-f7b9-49bd-ae35-fffc52ea2e62Estado ambulatorio Parte A: ambulatorio con un ítem de entrada específico en el rendimiento en la función de las extremidades superiores, versión 2.0 (PUL 2.0) en la selección.7Spanish7975655Adequate muscle for obtaining tissue biopsy as assessed by the investigator.9743642ee684bd3-0c08-4c39-8b9d-f67d02d9793dVoldoende spier voor het verkrijgen van een weefselbiopt zoals beoordeeld door de onderzoeker.18Dutch9743641ee684bd3-0c08-4c39-8b9d-f67d02d9793dMúsculo adecuado para obtener la biopsia de tejido según la evaluación del investigador.7Spanish7975666Other protocol-defined criteria apply97436449fa9e234-e328-4164-bca7-a5622bf3aa3aAndere door het protocol gedefinieerde criteria zijn van toepassing18Dutch97436439fa9e234-e328-4164-bca7-a5622bf3aa3aSe aplican otros criterios definidos por el protocolo.7Spanish13665021Any significant concomitant medical condition that interfere with the ability to comply with protocol requirements97436131ca07a79-f4f3-4b3b-a9e5-05d1b5e67ec4Sufre cualquier afección médica concomitante significativa que interfiera en la capacidad de cumplir con los requisitos del protocolo7Spanish97436141ca07a79-f4f3-4b3b-a9e5-05d1b5e67ec4Elke belangrijke bijkomende medische aandoening die de mogelijkheid om te voldoen aan de protocolvereisten belemmert18Dutch13665032Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant’s safety974361541736bcd-3cb7-4bb7-8f44-4eaf0e8d94cdHeeft een acute ziekte binnen vier weken voorafgaand aan de eerste dosis onderzoeksmiddel die de metingen van het onderzoek kan verstoren of de veiligheid van de deelnemer in gevaar kan brengen18Dutch974361641736bcd-3cb7-4bb7-8f44-4eaf0e8d94cdSufre una enfermedad aguda en las 4 semanas anteriores a la primera dosis del fármaco del estudio que pudiera interferir con las mediciones del estudio o poner en peligro la seguridad del participante7Spanish13665043Use of the following medications: a. Prior or current treatment with any exon skipping therapy within the previous 12 months b. Prior or current treatment with any gene therapy c. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents d. Use of immunosuppressants (other than systemic or oral corticosteroids for chronic non-DMD conditions) e. Treatment with a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat9743617cd48463a-bdcf-412e-ad7f-338d6e3ddd37Uso de los siguientes medicamentos: a. Tratamiento previo o actual con cualquier tratamiento de omisión del exón en los 12 meses anteriores. b. Tratamiento previo o actual con cualquier terapia génica. c. Uso de anticoagulantes, antitrombóticos o antiagregantes plaquetarios. d. Uso de inmunosupresores (excepto corticoesteroides sistémicos u orales para afecciones crónicas distintas de la DMD). e. Tratamiento con un inhibidor de la histona-desacetilasa (HDAC), por ejemplo, givinostat.7Spanish9743618cd48463a-bdcf-412e-ad7f-338d6e3ddd37Gebruik van de volgende medicatie: a. Eerdere of huidige behandeling met exon-skippingtherapie binnen de afgelopen twaalf maanden b. Eerdere of huidige behandeling met gentherapie c. Gebruik van antistollingsmiddelen, antitrombotica of antiplaatjesmiddelen d. Gebruik van immuunonderdrukkers (anders dan waaronder systemische of orale corticosteroïden voor chronische niet-DMD-aandoeningen) e. Behandeling met histondeacetylase (HDAC)-remmer, waaronder o.a. givinostat18Dutch13665054Laboratory abnormalities9743620d16e0796-1fe4-47a9-9315-47a436db4d68Anomalías analíticas7Spanish9743619d16e0796-1fe4-47a9-9315-47a436db4d68Afwijkende laboratoriumwaarden18Dutch13665065Daytime ventilator dependence, or any use of invasive mechanical ventilation via tracheostomy.97436228e5fd4b0-906d-4f71-a902-9e68c76619e4Afhankelijkheid van beademing overdag of gebruik van invasieve mechanische beademing via tracheostomie.18Dutch97436218e5fd4b0-906d-4f71-a902-9e68c76619e4Dependencia de un respirador durante el día o cualquier uso de ventilación mecánica invasiva mediante traqueotomía.7Spanish13665076Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and/or a QT interval with Fridericia correction method (QTcF) >450 msec at Screening or prior to the first dose of study drug on Day 1.9743624fdd25465-b0be-4a5c-b3a6-ded782fd9881Heeft een afwijkend elektrocardiogram (ECG) dat door de onderzoeker als klinisch significant wordt beoordeeld, en/of een QT-interval met Fridericia-correctiemethode (QTcF) > 450 msec bij de screening of voor de eerste dosis onderzoeksmiddel op dag 1.18Dutch9743623fdd25465-b0be-4a5c-b3a6-ded782fd9881Presencia de una lectura anómala en el electrocardiograma (ECG) evaluada como clínicamente significativa por el investigador o un intervalo QT con método de corrección de Fridericia (QTcF) >450 ms en la selección o antes de la primera dosis del fármaco del estudio el día 1.7Spanish13665087Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).974362590f5adf6-e445-4e59-84ce-61643479bf66Recepción de cualquier fármaco experimental o en investigación, etc. en los 3 meses anteriores a la primera dosis del fármaco del estudio o en las 5 semividas (lo que sea más largo)7Spanish974362690f5adf6-e445-4e59-84ce-61643479bf66Heeft een experimenteel of onderzoeksmiddel, etc. binnen drie maanden voorafgaand aan de eerste dosis onderzoeksmiddel of binnen vijf halfwaardetijden (wat het langst is)18Dutch13665098Use of any pharmacologic treatment, other than stable corticosteroids, that might have had an effect on muscle strength or function during the study (eg, growth hormone, testosterone).974362748c588e1-e1df-4fe1-bfa9-ccb38926f764Gebruik van een farmacologische behandeling, anders dan stabiele corticosteroïden, die een effect zou kunnen hebben op de spierkracht of -functie tijdens het onderzoek (bijv. groeihormoon, testosteron)18Dutch974362848c588e1-e1df-4fe1-bfa9-ccb38926f764Solo en el caso de la parte B: uso de cualquier tratamiento farmacológico, distinto de corticoesteroides estables, que pudiera tener un efecto sobre la fuerza o la función muscular durante el estudio (p. ej., hormona del crecimiento, testosterona).7Spanish13665109Other protocol-defined criteria apply.9743630a441a961-b52e-42c0-8e6e-7ceaa18f9474Andere door het protocol gedefinieerde criteria zijn van toepassing.18Dutch9743629a441a961-b52e-42c0-8e6e-7ceaa18f9474Se aplican otros criterios definidos por el protocolo.7Spanish9340771Incidence and severity of treatment emergent adverse events (TEAEs) (Part A and OL Period)True9743582e122b74f-78b1-4ffb-bd2c-1eb794e76c7cIncidentie en ernst van tijdens de behandeling ontstane bijwerkingen (TEAE’s) (deel A en OL-periode)18Dutch9743581e122b74f-78b1-4ffb-bd2c-1eb794e76c7cIncidencia e intensidad de los acontecimientos adversos surgidos durante el tratamiento (AAST) (Parte A y período abierto)7Spanish9340782Changes in vital sign measurements (Part A and OL Period)True97435840870f271-93fd-41b7-be13-ffea8826f964Veranderingen in de metingen van de vitale functies (deel A en OL-periode)18Dutch97435830870f271-93fd-41b7-be13-ffea8826f964Variaciones de las determinaciones de constantes vitales (Parte A y período abierto)7Spanish9340793Changes in clinical laboratory results (Part A and OL Period)True9743585a768c455-1732-4ec3-a9b2-8a511f302918Variaciones de los resultados analíticos (Parte A y período abierto)7Spanish9743586a768c455-1732-4ec3-a9b2-8a511f302918Veranderingen in de resultaten van de klinische laboratoriumonderzoeken (deel A en OL-periode)18Dutch9340804Changes in electrocardiogram (ECG) parameters (Part A and OL Period)True97435874c629ce0-10f5-477d-9ae1-039bb1df4b37Variaciones de los parámetros electrocardiográficos (ECG) (Parte A y período abierto)7Spanish97435884c629ce0-10f5-477d-9ae1-039bb1df4b37Veranderingen in de parameters van het elektrocardiogram (ecg) (deel A en OL-periode)18Dutch9340815Changes in physical examination findings (Part A and OL Period)True97435891f64d722-4d53-47c9-a33c-c8cb0a5572c5Veranderingen in de bevindingen uit het lichamelijk onderzoek (deel A en OL-periode)18Dutch97435901f64d722-4d53-47c9-a33c-c8cb0a5572c5Variaciones de los hallazgos de la exploración física (Parte A y período abierto)7Spanish9340821Plasma, muscle, and urine concentration of ENTR-601-45 and its final metabolite (Part A and OL Period)False97435918500c27e-5b72-412d-9a7d-4459b2c058d3De concentratie van ENTR-601-45 en diens uiteindelijke metaboliet in het plasma, de spieren en de urine (Deel A en OL-periode)18Dutch97435928500c27e-5b72-412d-9a7d-4459b2c058d3Concentración plasmática, muscular y en orina de ENTR-601-45 y su metabolito final (Parte A y periodo abierto)7Spanish9340832Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A)False9743594453c1d42-d477-452f-b501-d5575f268c96Verandering van baseline tot het einde van deel A van dystrofine volgens de Western blot op spierbiopsie (Deel A)18Dutch9743593453c1d42-d477-452f-b501-d5575f268c96Variación de la distrofina, determinada mediante inmunotransferencia a partir de una biopsia muscular, entre el momento basal y el final de la Parte A (Parte A)7Spanish9340843Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A)False974359540ff13b5-d993-4997-bc86-21c9088a4615Cambio con respecto al inicio en la expresión y la localización de la distrofina en la biopsia muscular al final del estudio (Parte A)7Spanish974359640ff13b5-d993-4997-bc86-21c9088a4615Verandering van baseline tot het einde van deel A in dystrofine-expressie en -lokalisatie van spierbiopt (Deel A)18Dutch9340854Percent change from baseline to End of Part A in exon 45 skipping measured in muscle biopsy (Part A)False9743598161ee526-453f-4d86-bb70-a73f8a2a22eeProcentuele verandering van baseline tot het einde van deel A in exon 45-skipping gemeten in spierbiopt (Deel A)18Dutch9743597161ee526-453f-4d86-bb70-a73f8a2a22eeVariación porcentual de la omisión del exón 45, medida en una biopsia muscular, entre el momento basal y el final de la Part A (Parte A)7Spanish9340865Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period)False97435996d762faf-355f-469c-8391-a9b407747820Anti-drug-antilichaam (ADA) en anti-dystrofine-antilichaam in serum (Deel A en OL-periode)18Dutch97436006d762faf-355f-469c-8391-a9b407747820Anticuerpo antifármaco (AAF) y anticuerpo antidistrofina en suero (Parte A y periodo abierto)7Spanish9340876Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period)False9743602a4c76d42-13df-41e1-98c9-dc2cb7d5f088Variación en la prueba de caminar/correr 10 metros (10MWR) entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish9743601a4c76d42-13df-41e1-98c9-dc2cb7d5f088Verandering ten opzichte van baseline tot het einde van de OL-periode in 10-meter looptest (10MWR) (deel A en OL-periode)18Dutch9340887Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period)False97436045bc291ee-56cd-4e84-84d1-2ece9afb7410Variación en el tiempo que se tarda en levantarse del suelo entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish97436035bc291ee-56cd-4e84-84d1-2ece9afb7410Verandering ten opzichte van baseline tot het einde van de OL-periode in tijd om recht te staan vanop de vloer (deel A en OL-periode)18Dutch9340898Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period)False9743606e50c6435-8351-4cd2-bd2d-a669c4d948f6Variación en el tiempo que se tarda en subir 4 escalones (4SC) entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish9743605e50c6435-8351-4cd2-bd2d-a669c4d948f6Verandering ten opzichte van baseline tot het einde van de OL-periode in de 4-treden klimmeting op tijd (4SC) (deel A en OL-periode)18Dutch9340909Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period)False97436070979bca5-56a5-4c78-8782-4a2298d0b812Verandering ten opzichte van baseline tot het einde van de OL-periode in de 95th centile Stride Velocity (SV95C) (deel A en 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2.0) (deel A en OL-periode)18Dutch9743612ada2ee7f-3113-4232-81cd-1b638e049501Variación en el funcionamiento de la extremidad superior v2.0 (PUL 2.0) entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish2028-10-302028-10-302025-06-3086487Entrada Therapeutics, Inc.32564322325642333256452626325644252532564624242PatientsTrueTrueTrue250125687171725306Non-EEA National Competent authority17Medicines And Healthcare Products Regulatory AgencyFalseTrueORG-100003945Active750021151 Buckingham Palace Road151 Buckingham Palace RoadLondonSW1W 9SZ826United KingdomTrueORG-10000394550127687166725301EEA National Competent authority16Federal Institute For Drugs And Medical DevicesFalseTrueORG-100003923Active750016Kurt-Georg-Kiesinger-Allee 3, HochkreuzKurt-Georg-Kiesinger-Allee 3HochkreuzBonn531752013GermanyTrueORG-10000392350124687169725304EEA National Competent authority16Swedish Medical Products AgencyFalseTrueORG-100003944Active750019Dag Hammarskjolds Vag 42, Uppsala Science ParkDag Hammarskjolds Vag 42Uppsala Science ParkUppsala752 372028SwedenTrueORG-10000394450126687172725307Non-EEA National Competent authority17Food And Drug AdministrationFalseTrueORG-100006066Active7500225901 Ammendale Road Ste B5901 Ammendale Road Ste BBeltsville20705-1277840United StatesTrueORG-100006066307112024-517584-23-00Entrada Therapeutics Inc.A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping with an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-44, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-44 (ELEVATE-44)NON_APPLICABLENON_APPLICABLE18685Falseacceptable2025-12-262025-12-265Diseases [C] - Musculoskeletal Diseases [C05]123368Duchenne Muscular DystrophyTrue134247True395736PublicRegulatory Affairsclinicaltrials@entradatx.com0602854Pharmaceutical company10Entrada Therapeutics Inc.TrueTrueORG-100052632Active395737ScientificRegulatory Affairsclinicaltrials@entradatx.com0602854Pharmaceutical company10Entrada Therapeutics Inc.TrueTrueORG-100052632Active600417687183725318Pharmaceutical company10Medpace Finland OyTrueTrueORG-100009147Active750034Kaikukatu 4 CKaikukatu 4 CHelsinki005302011Finland+34917900565RS-Advisor-Support@medpace.comTrueORG-10000914793390819339091293391015Imaging9339114933912593391369339148+34917900565RS-Advisor-Support@medpace.com600416687182725317Pharmaceutical company10Agada Biosciences Inc.TrueTrueORG-100051126Active7500331498 Lower Water Street, Mp 11151498 Lower Water StreetMp 1115HalifaxB3J 3R5124Canada0019024424011amackinnon@agadabio.comTrueORG-10005112693390740019024424011amackinnon@agadabio.com600419687179725314Hospital/Clinic/Other health care facility8Illingworth Research Group LimitedFalseTrueORG-100042356Active750030Hazelwood House, Larkwood Way, Tytherington Business ParkHazelwood HouseLarkwood WayTytherington Business ParkMacclesfieldSK10 2XR826United Kingdom+441625617447taylor.ross@syneoshealth.comTrueORG-10004235693391615Home Health Services+441625617447taylor.ross@syneoshealth.com600421687185725320Pharmaceutical company10Flagship Biosciences Inc.TrueTrueORG-100043268Active750036133 Southcenter Court Suite 400133 Southcenter Court Suite 400Morrisville27560-8537840United States0019196535540bfrey@flagshipbio.comTrueORG-10004326893391840019196535540bfrey@flagshipbio.com600418687188725323Pharmaceutical company10PPD Development LPTrueTrueORG-100011560Active7500402244 Dabney Road2244 Dabney RoadRichmond23230-3323840United States0018044010708carrie.mootz@ppd.comTrueORG-10001156093391540018044010708carrie.mootz@ppd.com600420687178725313Pharmaceutical company10Trinds LLCTrueTrueORG-100051849Active7500296425 Living Place6425 Living PlacePittsburgh15206-5122840United States+14697058018sshella@trinds.comTrueORG-10005184993391713+14697058018sshella@trinds.com600422687177725312Non-Pharmaceutical company11ATOM International LimitedTrueTrueORG-100042393Active750028Office 16, Valley House, Kingsway South, Team Valley Trading EstateOffice 16Valley HouseKingsway SouthTeam Valley Trading EstateGatesheadNE11 0SW826United Kingdom+15137069924kyle.haas@atom-international.orgTrueORG-10004239393391915Physical Function Oversight+15137069924kyle.haas@atom-international.org600423687184725319Pharmaceutical company10QPS LLCTrueTrueORG-100012847Active7500353 Innovation Way Suite 2043 Innovation Way Suite 204Newark19711-5456840United States0013024535984John.Kolman@qps.comTrueORG-10001284793392040013024535984John.Kolman@qps.com602854Pharmaceutical company10Entrada Therapeutics Inc.TrueTrueORG-100052632Active570159602854Pharmaceutical company10Entrada Therapeutics Inc.TrueTrueORG-100052632Active6143331 Design Center Place Suite 17-5001 Design Center Place Suite 17-500Boston02210-2349840United StatesTrueORG-100052632TrueCommercial2Phase I/II integrated trial10871995Diseases [C] - Musculoskeletal Diseases 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(for publication)PDF1185945.04D4_Patient facing documents_Blank Document_Entrada8a4f59f8-c19f-4c45-98be-adca5bbd4106104Protocol (for publication)PDF118594N/A1D1_Protocol synopsis_German_2024-517584-23_Entrada_redacted39cddf59-e1bd-4e0c-9c01-55ff3dd24b7e7Synopsis of the protocol (for publication)PDF1185945.04D1_Protocol synopsis_French_2024-517584-23_Entrada_redacted1c72b8ef-cb75-453a-987c-1f754289ecc37Synopsis of the protocol (for publication)PDF1185945.04D1_Protocol synopsis_IT_2024-517499-39_Entrada_Redacted285ada21-ae70-4176-93c3-a5bec7da8def7Synopsis of the protocol (for publication)PDF1185945.04D1_Protocol Synopsis_ENG_2024-517499-39_Entrada_redacted6e92161b-e0bb-4c68-86c5-222eb23774a47Synopsis of the protocol (for publication)PDF1185945.04D1_Protocol synopsis_Spanish_2024-517584-23_Entrada_redacted99ba403a-838d-4405-9c41-9800d5fdfa117Synopsis of the protocol (for publication)PDF11859443D1_Protocol synopsis_Dutch_2024-517499-39_Entrada_redacted9eef5d29-264a-4610-ae4a-366649dda5887Synopsis of the protocol (for publication)PDF1185945.04D1_Protocol Lay synopsis__ENG_Entrada_redacted0535ae04-c330-4037-bdd9-3c437263c74a7Synopsis of the protocol (for publication)PDF11859433D1_Protocol Lay Synopsis_Dutch_2024-517499-39_Entrada_redacted0a95b78f-47d8-4dd5-943b-d1e83ba459ec7Synopsis of the protocol (for publication)PDF11859433D1_Lay Synopsis_Spanish_2024-517499-39_Entrada_redactedbd844d31-ab5e-4e12-a03c-e64fa1da25677Synopsis of the protocol (for publication)PDF11859433D1_Lay Synopsis_Italian_2024-517499-39_Entrada_Redactedc3f93ae8-7c23-4d38-9fc9-04520a2af12f7Synopsis of the protocol (for publication)PDF1185943.01Both3Reverted (2025-07-24)3041CM-IT-00016Dear Applicant, Considering the expiration of the three-year mandate of the members of the National Ethics Committee (CEN) for clinical trials relating to advanced therapies (“ATMP”) and of the National Ethics Committee (CEN) for clinical trials in the pediatric field, appointed by Decree of the Minister of Health - 2 March 2022; Considering the fact that, due to the expiration of the mandate of the members of the aforementioned National Ethics Committee (CEN), for the procedure in subject the assessment of the aspects relating to Part II of the evaluation report pursuant to art. 7 of the aforementioned Regulation (EU) No. 536/2014 has not been carried out, and as a result there is no conclusion of Part II for the EU CT 2024-517499-39-00 procedure (AIFA authorization provision n° 0058222); In compliance with CHAPTER XIII (SUPERVISION BY MEMBER STATES, UNION INSPECTIONS AND CONTROLS) of Regulation 536/2014 with specific reference to Article 77 (Corrective measures to be taken by Member States): 1. Where a Member State concerned has justified grounds for considering that the requirements set out in this Regulation are no longer met, it may take the following measures on its territory: (a) revoke the authorisation of a clinical trial; (b) suspend a clinical trial; (c) require the sponsor to modify any aspect of the clinical trial. A corrective measure is applied suspending the trial. This corrective measure is only applicable to Italy.True44427ItalyItaly2025-07-242025-07-24 2024-517584-23-004AuthorisedA 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping with an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-44, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-44 (ELEVATE-44)ENTR-601-44-201Duchenne Muscular DystrophySpain:4Belgium:4Italy:430/04/2025BE: 30/04/2025, ES: 30/04/2025, IT: 05/05/2025Diseases [C] - Musculoskeletal Diseases [C05]Entrada Therapeutics Inc.Pharmaceutical companyPhase I and Phase II (Integrated)- OtherPlasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite (Part A and OL Period), Change from baseline in dystrophin by Western blot from muscle biopsy (Part A), Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A), Percent change from baseline to End of Part A in exon 44 skipping measured in muscle biopsy (Part A), Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period)), Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period), Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period), Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period), Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period), Change from baseline to End of OL Period in North Star Ambulatory Assessment (NSAA) (Part A and OL Period), Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) (Part A and OL Period)ENTR-601-44, SODIUM CHLORIDE6418-64 years, 0-17 yearsFemale, Male3Both9Incidence and severity of treatment emergent adverse events (TEAEs) (Part A and OL Period)), Changes in vital sign measurements (Part A and OL Period), Changes in clinical laboratory results (Part A and OL Period), Changes in electrocardiogram (ECG) parameters (Part A and OL Period), Changes in physical examination findings (Part A and OL Period)No08/04/202609/04/20262025-04-30T10:09:28.472026-04-09T03:42:38.194580164K1_Recruitment arrangements_BE_Entrada53a7929d-ec12-4d34-b155-4af11d7564a914Recruitment arrangements (for publication)PDF2716831.01K2_Recruitment material_Brochure_French_Entradaf7523ba7-0835-4ef4-9642-a8934a24b14514Recruitment arrangements (for publication)PDF2716831.03K2_Recruitment material_Brochure_Dutch_Entrada6ae20bd8-387e-47bb-b585-7155907b1d8714Recruitment arrangements (for publication)PDF2716831.03K2_Recruitment material_Brochure_English_Entrada7357792c-d525-4110-85ed-9b888129c3e614Recruitment arrangements (for publication)PDF2716831.03K2_Recruitment material_Email blast for Advocacy groups_Dutch_Entrada79d073b9-1869-4c0b-b565-efbf8a63bc0c14Recruitment arrangements (for 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publication)PDF29427511Spain433822025-04-30T10:09:28.472026-01-19T12:36:27.0694Belgium433832025-04-30T10:53:32.6882026-01-19T12:36:27.0694Italy433842025-05-05T16:51:00.8172026-04-08T15:27:55.839443382SpainSTART_OF_TRIAL2025-05-28START_OF_RECRUITMENT2025-06-0343383BelgiumSTART_OF_TRIAL2025-05-16START_OF_RECRUITMENT2025-07-0343384ItalySTART_OF_TRIAL2026-01-08START_OF_RECRUITMENT2026-03-062024-517584-23-004A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping with an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-44, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-44 (ELEVATE-44)ENTR-601-44-20116/05/2025Duchenne Muscular DystrophySpain:4Belgium:4Italy:430/04/2025BE: 30/04/2025, ES: 30/04/2025, IT: 05/05/2025Diseases [C] - Musculoskeletal Diseases [C05]Entrada Therapeutics Inc.Pharmaceutical companyPhase I and Phase II (Integrated)- OtherPlasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite (Part A and OL Period), Change from baseline in dystrophin by Western blot from muscle biopsy (Part A), Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A), Percent change from baseline to End of Part A in exon 44 skipping measured in muscle biopsy (Part A), Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period)), Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period), Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period), Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period), Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period), Change from baseline to End of OL Period in North Star Ambulatory Assessment (NSAA) (Part A and OL Period), Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) (Part A and OL Period)ENTR-601-44, SODIUM CHLORIDE6418-64 years, 0-17 yearsFemale, Male39Incidence and severity of treatment emergent adverse events (TEAEs) (Part A and OL Period)), Changes in vital sign measurements (Part A and OL Period), Changes in clinical laboratory results (Part A and OL Period), Changes in electrocardiogram (ECG) parameters (Part A and OL Period), Changes in physical examination findings (Part A and OL Period)No08/04/202609/04/20262024-517584-23-00Authorised2025-05-162025-04-30T10:09:28.472026-04-09T03:42:38.19458016411799615100000000556United Kingdom826GBGBRTrue489526111749256SOLUTION FOR INFUSIONPRD117492561ENTR-601-44SOLUTION FOR INFUSIONENTR-601-44SUB408740ENTRADA THERAPEUTICS, INC.11749256408741ENTRADA THERAPEUTICS, INC.Nucleic AcidNucleic AcidENTR-601-44SUB408740False1FalseConjugate of a DMD exon 44 skipping phosphorodiamidate morpholino oligomer and a cyclic peptidePRD11749256DE_BB_01_MIA_2024_001511INTRAVENOUS INFUSIONFalseENTR-601-44entr-601-44SOLUTION FOR INFUSION4895273SUB12581MIG-2SODIUM CHLORIDESOLUTION FOR INFUSIONSODIUM CHLORIDESUB12581MIG79225ChemicalSODIUM CHLORIDESUB12581MIG---False3FalseFalseChemicalproduct sourced locally, see test product section for SmPCSUB12581MIG11INTRAVENOUS INFUSIONFalseSODIUM CHLORIDEsodium chlorideA 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping with an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-44, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-44 (ELEVATE-44)96770428bfa132f-db31-4b24-8574-e860ff17f18fEstudio aleatorizado, doble ciego, controlado con placebo y con dos partes en participantes con distrofia muscular de Duchenne susceptible de omisión del exón 44, con una parte A inicial de dosis múltiples ascendentes para evaluar la seguridad, tolerabilidad, farmacocinética y farmacodinámica de ENTR-601-44, seguida de la parte B para evaluar la seguridad y eficacia de ENTR-601-44 (ELEVATE-44)7SpanishA 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44 (ELEVATE-44)967705048660b0e-faf0-4bba-8d9f-166af96deb43Estudio aleatorizado, doble ciego, controlado con placebo y con dos partes en participantes con distrofia muscular de Duchenne susceptible de omisión del exón 44 para evaluar la seguridad y eficacia de ENTR-601-44 (ELEVATE-44)7SpanishENTR-601-44-201487394U1111-1316-5469False92Integrated phase 1/2 trial.108079122678Duchenne Muscular Dystrophy9677017fe6fd5c9-d7a3-4da9-ba10-40829c1c882bDistrofia muscular de Duchenne7SpanishTrue10000001254427.1PTDuchenne muscular dystrophy10013801100000004850False10000001204720.1PTDuchenne muscular dystrophy gene carrier10052655100000004850False73570245357022635702343570259357026Part A and OL Period: To evaluate the safety and tolerability of ENTR-601-44 in participants with Duchenne muscular dystrophy (DMD)96770495422a02a-e774-418b-9455-b4bdf49b0268Parte A y período abierto: Evaluar la seguridad y tolerabilidad de ENTR-601-44 en participantes con distrofia muscular de Duchenne (DMD).7Spanish4145491To characterize the pharmacokinetics of ENTR-601-44 in participants with DMD in Part A96770518ab867c0-b752-414d-8537-bcebdde2dabaDefinir la farmacocinética de ENTR-601-44 en participantes con DMD en Parte A7Spanish4145502To characterize the pharmacodynamics of ENTR-601-44 in participants with DMD in Part A9677052e1718fb4-d488-4fae-a4a4-f8d065efbb4bDefinir la farmacodinámica de ENTR-601-44 en participantes con DMD en Parte A7Spanish4145513To evaluate the immune response to ENTR-601-44 in participants with DMD in Part A9677053bff57c93-c4fb-4917-861f-66433f4f34e3Evaluar la respuesta inmunitaria a ENTR-601-44 en participantes con DMD en Parte A7Spanish4145524To evaluate the impact of ENTR-601-44 on measures of function in participants with DMD after extended dosing (Part A and OL Period)9677054013400cb-4ac0-434c-96c4-6276b679d533Evaluar el efecto de ENTR-601-44 en las medidas de función en los participantes con DMD tras la administración ampliada (Parte A y período abierto)7Spanish7932711Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor.96770432624c36b-3672-43e5-a532-ae489fdfec56Diagnóstico genético de DMD y variante patológica confirmada en el gen de la distrofina susceptible de omisión del exón 44, según la evaluación de un asesor genético central.7Spanish7932722Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.9677044b5dd9359-08c4-40f3-99ee-275d8069c130Sexo masculino asignado al nacer con signos clínicos compatibles con DMD, según lo determinado por el investigador.7Spanish7932733Part A: 4-20 years of age, inclusive9677045d215b009-d955-4e7b-8ffa-2e00db922e4aParte A: edad comprendida entre 4 y 20 años, ambos inclusive7Spanish7932744Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening9677046a4d0b3a9-74fd-40fc-8814-8c95a35eb88fCapacidad deambulatoria: Parte A: presencia de capacidad deambulatoria , con una puntuación según el protocolo de entrada de la escala PUL 2.0 en el período de selección.7Spanish7932755Adequate muscle for obtaining tissue biopsy as assessed by the investigator.9677047d3b10226-7945-4bf2-81a6-d6c317518bd5Músculo adecuado para obtener una biopsia de tejido, según la evaluación del investigador.7Spanish7932766Other protocol-defined criteria apply9677048375cf336-e302-4c6e-b29e-d5b25dabbfc2Se aplican otros criterios definidos por el protocolo7Spanish13589921Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements9677034cff3c5c0-889f-4b66-9a7e-e7d330bd254eCualquier enfermedad concomitante importante que podría dificultar la capacidad de cumplir los requisitos del protocolo7Spanish13589932Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant’s safety967703502ee3c24-6397-4cdb-9f2c-eb3af9455c46Presencia de una enfermedad aguda en las cuatro semanas previas a la primera dosis del fármaco del estudio que podría dificultar las determinaciones del estudio o poner en peligro la seguridad del participante7Spanish13589943Use of the following medications: a. Prior treatment with any exon skipping therapy at any time b. Prior treatment with any gene therapy at any time From at least 30 days prior to the start of the screening period until the end of the study: c. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents d. Use of immunosuppressants (other than oral corticosteroids for DMD conditions) e. Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat96770367329b3a1-a05f-4b9a-87eb-4327d4b4d999Uso de los siguientes medicamentos: a. Tratamiento previo con cualquier terapia de omisión de exones, en cualquier momento. b. Tratamiento previo con cualquier terapia génica, en cualquier momento. Desde al menos 30 días antes del comienzo del período de selección hasta el final del estudio: c. Uso de anticoagulantes, antitrombóticos o antiagregantes plaquetarios d. Uso de inmunodepresores (distintos de los corticoesteroides orales para tratar trastornos relacionados con la DMD) e. Tratamiento previo o actual con un inhibidor de la histona desacetilasa (HDAC), como givinostat, entre otros7Spanish13589954Laboratory abnormalities9677037bf45b488-60e7-4291-a9c2-b61e08742ba5Anomalías analíticas7Spanish13589965Daytime ventilator dependence, or any use of invasive mechanical ventilation via tracheostomy.967703854299cc1-ae77-4954-b233-0acad884af19Dependencia diurna de respirador o cualquier uso de ventilación mecánica invasiva mediante traqueostomía7Spanish13589976Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and/or a QT interval with Fridericia correction method (QTcF) >450 msec at Screening or prior to the first dose of study drug on Day 1.9677039d00fcdf4-4fb1-49a3-bce1-06713ae3a3e0Anomalías en la lectura del electrocardiograma (ECG) que el investigador considere clínicamente significativas o intervalo QT con el método de corrección de Fridericia (QTcF) >450 ms en el período de selección o antes de la primera dosis del fármaco del estudio administrada el día 17Spanish13589987Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).967704048dccdfc-e465-4538-b142-24ec59b82930Recepción de cualquier medicamento experimental o en investigación, etc. en los tres meses previos a la primera dosis del fármaco del estudio o en el período equivalente a cinco semividas (lo que suponga más tiempo)7Spanish13589998Other protocol-defined criteria apply9677041af8ee64e-1d80-4eb8-9ad6-0bdc0bef0c4bSe aplican otros criterios definidos por el protocolo7Spanish9289241Incidence and severity of treatment emergent adverse events (TEAEs) (Part A and OL Period))True9677018641e3e9f-29a6-46bb-92ab-01976f484e5fIncidencia e intensidad de los acontecimientos adversos surgidos durante el tratamiento (AAST) (Parte A y período abierto)7Spanish9289252Changes in vital sign measurements (Part A and OL Period)True967701984684ca2-b81e-4c49-97f7-79139fc41380Variaciones de las determinaciones de constantes vitales (Parte A y período abierto)7Spanish9289263Changes in clinical laboratory results (Part A and OL Period)True9677020ac0d07e6-48a5-46e9-b9b6-e3cf1623fdc5Variaciones de los resultados analíticos (Parte A y período abierto)7Spanish9289274Changes in electrocardiogram (ECG) parameters (Part A and OL Period)True9677021ad004065-4115-4c22-ab43-7e1a2863f414Variaciones de los parámetros electrocardiográficos (ECG) (Parte A y período abierto)7Spanish9289285Changes in physical examination findings (Part A and OL Period)True967702207c8f20c-8c5c-4a44-a3e7-32afb269e844Variaciones de los hallazgos de la exploración física (Parte A y período abierto)7Spanish9289291Plasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite (Part A and OL Period)False96770237f34783e-1ed8-41f0-a468-5f9a8f366e7aConcentración plasmática, muscular y urinaria de ENTR-601-44 y su metabolito final (Parte A y período abierto)7Spanish9289302Change from baseline in dystrophin by Western blot from muscle biopsy (Part A)False9677024a3737c0f-abd2-4a5d-a174-78da782dd9feVariación de la distrofina, determinada mediante inmunotransferencia a partir de una biopsia muscular, entre el momento basal y el final de la Parte A (Parte A)7Spanish9289313Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A)False96770259bcacc8b-240a-48c0-98dd-d18fdf05506cVariación de la expresión y localización de la distrofina a partir de una biopsia muscular entre el momento basal y el final de la Parte A (Parte A)7Spanish9289324Percent change from baseline to End of Part A in exon 44 skipping measured in muscle biopsy (Part A)False9677026a8586be9-bcfa-450f-b8e4-f9b78a339ab6Variación porcentual de la omisión del exón 44, medida en una biopsia muscular, entre el momento basal y el final de la Parte A (Parte A)7Spanish9289335Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period))False9677027694533fa-903f-40f9-8c67-0c67c31a1978Anticuerpos contra el fármaco (ACF) y anticuerpos contra la distrofina en suero (Parte A y período abierto)7Spanish9289346Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period)False96770286c661c7b-056c-4c8b-b669-cc74966d8612Variación en la prueba de caminar/correr 10 metros (10MWR) entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish9289357Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period)False96770291dba255c-00e1-4fb8-8aec-2834fdc96f27Variación en el tiempo que se tarda en levantarse del suelo entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish9289368Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period)False9677030f76c4414-02db-45fd-b449-1e30de910dd5Variación en el tiempo que se tarda en subir 4 escalones (4SC) entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish9289379Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period)False967703191375d7b-6900-40a3-8920-48e3df6b8fc0Variación en el percentil 95 de la velocidad de zancada (SV95C) entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish92893810Change from baseline to End of OL Period in North Star Ambulatory Assessment (NSAA) (Part A and OL Period)False9677032cf1cbbc8-bc4a-4318-89f3-f92270e54b09Variación en la escala North Star Ambulatory Assessment (NSAA) entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish92893911Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) (Part A and OL Period)False96770336afe0a8a-03a9-4a11-af78-093d4760d520Variación en el funcionamiento de la extremidad superior v2.0 (PUL 2.0) entre el momento basal y el final del período abierto (Parte A y período abierto)7Spanish2027-01-042027-01-042025-06-3086030Entrada Therapeutics, Inc.3240123332401122324014262632401324242PatientsTrueTrueTrue349854737804776250Non-EEA National Competent authority17Medicines And Healthcare Products Regulatory AgencyFalseTrueORG-100003945Active810769151 Buckingham Palace Road151 Buckingham Palace RoadLondonSW1W 9SZ826United KingdomTrueORG-10000394549853670163708233EEA National Competent authority16Federal Institute For Drugs And Medical DevicesFalseTrueORG-100003923Active729766Kurt-Georg-Kiesinger-Allee 3, HochkreuzKurt-Georg-Kiesinger-Allee 3HochkreuzBonn531752013GermanyTrueORG-10000392349855673403711482Non-EEA National Competent authority17Food And Drug AdministrationFalseTrueORG-100006066Active73359610903 New Hampshire Avenue10903 New Hampshire AvenueSilver Spring20993-0002840United StatesTrueORG-100006066acceptable5Diseases [C] - Musculoskeletal Diseases [C05]122678Duchenne Muscular DystrophyTrue133529True393610PublicRegulatory Affairsclinicaltrials@entradatx.com0606931Pharmaceutical company10Entrada Therapeutics Inc.TrueTrueORG-100052632Active393611ScientificRegulatory Affairsclinicaltrials@entradatx.com0606931Pharmaceutical company10Entrada Therapeutics Inc.TrueTrueORG-100052632Active596967671465709538Pharmaceutical company10Trinds LLCTrueTrueORG-100051849Active7313006425 Living Place6425 Living PlacePittsburgh15206-5122840United States+14697058018sshella@trinds.comTrueORG-10005184992864013+14697058018sshella@trinds.com596970671428709501Pharmaceutical company10Alliance Pharma Inc.TrueTrueORG-100046000Active73125817 Lee Boulevard17 Lee BoulevardMalvern19355-1234840United States0016102963152mrogers@resolian.comTrueORG-10004600092865040016102963152mrogers@resolian.com596969676165714251Non-Pharmaceutical company11ATOM International LimitedTrueTrueORG-100042393Active736833Office 16, Valley House, Kingsway South, Team Valley Trading EstateOffice 16Valley HouseKingsway SouthTeam Valley Trading EstateGatesheadNE11 0SW826United Kingdom+15137069924kyle.haas@atom-international.orgTrueORG-10004239392864915Physical Function Oversight+15137069924kyle.haas@atom-international.org596973671446709519Pharmaceutical company10Precision For Medicine Inc.TrueTrueORG-100041895Active7312779420 Kirby Drive Suite 1009420 Kirby Drive Suite 100Houston77054-2521840United States+12403064100Haley.Allemand@precisionformedicine.comTrueORG-1000418959286534+12403064100Haley.Allemand@precisionformedicine.com596966671451709524Pharmaceutical company10PPD Development LPTrueTrueORG-100011560Active7312868700 Quioccasin Road8700 Quioccasin RoadRichmond23229-5528840United States0018044010708carrie.mootz@ppd.comTrueORG-10001156092863940018044010708carrie.mootz@ppd.com596968671477709550Pharmaceutical company10Medpace Finland OyTrueTrueORG-100009147Active731318Kaikukatu 4 CKaikukatu 4 CHelsinki005302011Finland+34917900565RS-Advisor-Support@medpace.comTrueORG-1000091479286411928642129286431492864415Imaging9286454928646592864769286488+34917900565RS-Advisor-Support@medpace.com596975671459709532Pharmaceutical company10Flagship Biosciences Inc.TrueTrueORG-100043268Active731294133 Southcenter Court Suite 400133 Southcenter Court Suite 400Morrisville27560-8537840United States0019196535540bfrey@flagshipbio.comTrueORG-10004326892865540019196535540bfrey@flagshipbio.com596974671421709494Pharmaceutical company10Agada Biosciences Inc.TrueTrueORG-100051126Active7312491498 Lower Water Street, Mp 11151498 Lower Water StreetMp 1115HalifaxB3J 3R5124Canada0019024424011amackinnon@agadabio.comTrueORG-10005112692865440019024424011amackinnon@agadabio.com596972676164714250Hospital/Clinic/Other health care facility8Illingworth Research Group LimitedFalseTrueORG-100042356Active736831Hazelwood House, Larkwood Way, Tytherington Business ParkHazelwood HouseLarkwood WayTytherington Business ParkMacclesfieldSK10 2XR826United Kingdom+441625617447taylor.ross@syneoshealth.comTrueORG-10004235692865215Home Health Services+441625617447taylor.ross@syneoshealth.com596971671429709502Pharmaceutical company10QPS LLCTrueTrueORG-100012847Active7312593 Innovation Way Suite 2043 Innovation Way Suite 204Newark19711-5456840United States0013024535984John.Kolman@qps.comTrueORG-10001284792865140013024535984John.Kolman@qps.com606931Pharmaceutical company10Entrada Therapeutics Inc.TrueTrueORG-100052632Active574225606931Pharmaceutical company10Entrada Therapeutics Inc.TrueTrueORG-100052632Active6189011 Design Center Place Suite 17-5001 Design Center Place Suite 17-500Boston02210-2349840United StatesTrueORG-100052632TrueCommercial2Integrated phase 1/2 trial.10835105Diseases [C] - Musculoskeletal Diseases [C05]366323ENTR-601-44DE_BB_01_MIA_2024_00151Test489526111749256SOLUTION FOR INFUSIONPRD117492561ENTR-601-44SOLUTION FOR INFUSIONENTR-601-44SUB408740ENTRADA THERAPEUTICS, 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ICF_Optional_Healthy volunteer_Entrada235bfee3-6824-4d3a-9164-d7f2233db83f15Subject information and informed consent form (for publication)PDF29427511Both3Reverted (2025-07-24)3061CM-IT-00016Dear Applicant, Considering the expiration of the three-year mandate of the members of the National Ethics Committee (CEN) for clinical trials relating to advanced therapies (“ATMP”) and of the National Ethics Committee (CEN) for clinical trials in the pediatric field, appointed by Decree of the Minister of Health - 2 March 2022; Considering the fact that, due to the expiration of the mandate of the members of the aforementioned National Ethics Committee (CEN), for the procedure in subject the assessment of the aspects relating to Part II of the evaluation report pursuant to art. 7 of the aforementioned Regulation (EU) No. 536/2014 has not been carried out, and as a result there is no conclusion of Part II for the EU CT 2024-517584-23-00 procedure (AIFA authorization provision n° 0053041-30/04/2025-AIFA-AIFA_USC-P); In compliance with CHAPTER XIII (SUPERVISION BY MEMBER STATES, UNION INSPECTIONS AND CONTROLS) of Regulation 536/2014 with specific reference to Article 77 (Corrective measures to be taken by Member States): 1. Where a Member State concerned has justified grounds for considering that the requirements set out in this Regulation are no longer met, it may take the following measures on its territory: (a) revoke the authorisation of a clinical trial; (b) suspend a clinical trial; (c) require the sponsor to modify any aspect of the clinical trial. A corrective measure is applied suspending the trial. This corrective measure is only applicable to Italy.True43384ItalyItaly2025-07-242025-07-24 2024-514501-57-004AuthorisedA Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males with Duchenne Muscular DystrophySGT-003-101Duchenne muscular dystrophyItaly:427/01/2025IT: 18/03/2025Diseases [C] - Musculoskeletal Diseases [C05]Solid Biosciences Inc.Pharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansChange from baseline of microdystrophin tissue distribution at Day 90, Change from baseline of microdystrophin tissue distribution at Day 360, Change from baseline of microdystrophin protein levels at Day 360, Change from baseline in time to rise velocity at Day 360 and Day 540, Change from baseline in stride velocity 95th centile (SV95C) at Day 360 and Day 540, Change from baseline in 10-meter walk/run velocity at Day 360 and Day 540, Change from baseline in 4-stair climb velocity at Day 360 and Day 540, Change from baseline in the North Star Ambulatory Assessment (NSAA) score at Day 360 and Day 540, Change from baseline in 6-minute walk test (6MWT) distance at Day 360 and Day 540, Incidence of clinically significant laboratory abnormalities through Day 360 and Day 540, Incidence of clinically significant abnormalities in vital signs through Day 360 and Day 540, Incidence of clinically significant abnormalities in physical examinations through Day 360 and Day 540, Incidence of cardiac abnormalities by electrocardiogram (ECG) or echocardiography (ECHO) through Day 360 and Day 540524630-17 yearsMale3Both8Incidence of treatment-emergent adverse events (AEs) through Day 360, Change from baseline of microdystrophin protein levels at Day 90No05/05/202606/05/20262025-01-27T19:14:30.7432026-05-06T03:37:36.6190782954D1_Protocol 2024-514501-57-00_redacted28081a85-6590-45ac-af3b-7f63cd93b2fd104Protocol (for publication)PDF1281996.04D1_Protocol synopsis_ENG_2024-514501-57-008abd1faa-2693-4622-8c68-643d8830359f7Synopsis of the protocol (for publication)PDF1281996.04D1_Protocol synopsis_ITA_2024-514501-57-0083e40af3-3762-4593-8c41-49d6a93eafba7Synopsis of the protocol (for publication)PDF1281996.04K1_Recruitment arrangementsccff197c-bd6e-47a3-aeee-64d649a0ca6b14Recruitment arrangements (for publication)PDF30277911.01K2_Recruitment material_Recruitment Flyer ITAc8f0cbb7-e908-4fd6-a474-11f98764e03314Recruitment arrangements (for publication)PDF3027794.01L1_SIS and ICF_ITA_Parent_Cohort 1-4_Redacted7c7e9f87-5fe8-4241-9b49-e621e177757915Subject information and informed consent form (for publication)PDF3027794.05L1_SIS and ICF_ITA_Data Protection_Cohorts 1-5_Redactedbce3e912-1c1b-43bd-9094-9d3700ff693515Subject information and informed consent form (for publication)PDF3027792.02L1_SIS and ICF_ITA_Future Research_Redacted2c486f92-cbf8-4807-b27b-4d1eb732e49815Subject information and informed consent form (for publication)PDF3027792.02L1_SIS and ICF_ITA_Assent Child Pre-Screening_Redacted6ba744be-f893-4544-ab47-ba98f9a0d5ad15Subject information and informed consent form (for publication)PDF3027792.02L1_SIS and ICF_ITA_Assent Child_Cohorts 1-4_Redactedae716092-1e38-4d2e-831d-ad18fd681b6d15Subject information and informed consent form (for publication)PDF3027793.03L1_SIS and ICF_ITA_Parent Pre-Screening_Redacted1b7710d3-d404-4713-aedf-9a6144903aea15Subject information and informed consent form (for publication)PDF3027794.04L1_SIS and ICF_ITA_Assent Child_Cohort 5_Redactedd2f2f3ce-20d9-4135-b4fb-c3f818c406cc15Subject information and informed consent form (for publication)PDF3027792.02L1_SIS and ICF_ITA_Parent_Cohort 5_Redacted76414cb1-6242-4c5f-8bc7-10f2e80dfc5a15Subject information and informed consent form (for publication)PDF3027793.03L1_SIS and ICF_ITA_Participant Reaching AoM_Cohort 1-4_Redactedb2471484-ab1f-4cd3-91c2-f620549b8c3615Subject information and informed consent form (for publication)PDF3027793.03L1_SIS and ICF_ITA_Participant Reaching AoM_Cohort 5_Redacted700a3ab2-773d-4b7d-ab15-ca6d7c19977715Subject information and informed consent form (for publication)PDF3027793.03L1_SIS and ICF_ITA_Pregnant Partner_Redacted7c505e28-40df-495c-8df9-90746afb2e4c15Subject information and informed consent form (for publication)PDF3027791.01Italy343612025-01-27T19:14:30.7432026-05-05T10:50:12.672434361ItalySTART_OF_TRIAL2025-06-20START_OF_RECRUITMENT2025-08-072024-514501-57-004A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males with Duchenne Muscular DystrophySGT-003-10120/06/2025Duchenne muscular dystrophyItaly:427/01/2025IT: 18/03/2025Diseases [C] - Musculoskeletal Diseases [C05]Solid Biosciences Inc.Pharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansChange from baseline of microdystrophin tissue distribution at Day 90, Change from baseline of microdystrophin tissue distribution at Day 360, Change from baseline of microdystrophin protein levels at Day 360, Change from baseline in time to rise velocity at Day 360 and Day 540, Change from baseline in stride velocity 95th centile (SV95C) at Day 360 and Day 540, Change from baseline in 10-meter walk/run velocity at Day 360 and Day 540, Change from baseline in 4-stair climb velocity at Day 360 and Day 540, Change from baseline in the North Star Ambulatory Assessment (NSAA) score at Day 360 and Day 540, Change from baseline in 6-minute walk test (6MWT) distance at Day 360 and Day 540, Incidence of clinically significant laboratory abnormalities through Day 360 and Day 540, Incidence of clinically significant abnormalities in vital signs through Day 360 and Day 540, Incidence of clinically significant abnormalities in physical examinations through Day 360 and Day 540, Incidence of cardiac abnormalities by electrocardiogram (ECG) or echocardiography (ECHO) through Day 360 and Day 540524630-17 yearsMale38Incidence of treatment-emergent adverse events (AEs) through Day 360, Change from baseline of microdystrophin protein levels at Day 90No05/05/202606/05/20262024-514501-57-00Authorised2025-06-202025-01-27T19:14:30.7432026-05-06T03:37:36.619078295412819952100000000557United States840USUSATrue100000000354Canada124CACANTrue100000000556United Kingdom826GBGBRTrue532691111621322SUSPENSION FOR IV INFUSIONPRD116213221SUSPENSION FOR IV INFUSIONSGT-003SGT-003SUB396386SOLID BIOSCIENCES, LLC11621322396387SOLID BIOSCIENCES, LLCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherSGT-003SUB396386False1FalseGene therapyPRD11621322SGT-003DE_HE_01_MIA_2022_0026/23.1 (Pl) 18 L 18.01/1113-B67853human microdystrophinadeno-associated virusFalseTrue311INTRAVENOUS USEFalsesgt-003SUSPENSION FOR IV INFUSIONA Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males with Duchenne Muscular DystrophyA Study of SGT-003 Gene Therapy in Duchenne Muscular DystrophySGT-003-101526522NCT06138639False72This is a Phase I/II integrated trial117892133711Duchenne muscular dystrophyTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False33887205388724638872343887217388722To investigate the safety and tolerability of a single intravenous dose of SGT-003 and to investigate the efficacy of a single intravenous dose of SGT-003 by assessing microdystrophin expression in muscle biopsies.4542571To investigate the efficacy of a single intravenous dose of SGT-003 by assessing microdystrophin expression in muscle biopsies.4542582To investigate the efficacy of a single intravenous dose of SGT-003 by assessing changes in ambulatory function in participants who are ambulatory at baseline.4542593To investigate the efficacy of a single intravenous dose of SGT-003 by assessing changes in pulmonary function in participants enrolled at 4 years of age and older.4542604To investigate the safety and tolerability of a single intravenous dose of SGT-003 by assessing changes in upper limb function in participants enrolled in Cohort 4 and Cohort 5.4542615To investigate the safety of a single intravenous dose of SGT-003 by assessing pharmacokinetics, shedding, and immunogenicity.4542626To investigate the safety and tolerability of a single intravenous dose of SGT-003.4542637To investigate the efficacy of a single intravenous dose of SGT-003 by assessing changes in cardiac structure and function in participants with a cardiac magnetic resonance imaging (MRI) at Screening Part A or Rescreening.4542648To investigate the efficacy of a single intravenous dose of SGT-003 by assessing changes in development in participants enrolled up to 30 months of age.4542659To investigate the efficacy of a single intravenous dose of SGT-003 by assessing changes in patient-reported outcomes in participants enrolled at 2 years of age and older.45426610To investigate the efficacy of a single intravenous dose of SGT-003 by assessing changes in biomarkers of muscle structural integrity.8668971Participant age at the time of signing the informed consent form (ICF): Cohort 1: 4 to <7 years Cohort 2: 7 to <12 years Cohort 3: 0 to < 4 years Cohort 4: 12 to < 18 years Cohort 5: 10 to < 18 years86689810Participant is male.86689911Able to understand and comply with all study procedures as appropriate by age and have a parent(s) or legal guardian(s) (i.e., legally authorized representative [LAR]) who is (are) able to understand and comply with the study procedure requirements86690012If participant is of reproductive potential, participant and partner of childbearing potential are willing to use 2 highly effective forms of contraception for 12 months following study drug administration.8669012Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk/run test in < 30 seconds: Cohorts 1, 2, and 4: Ambulatory; Cohort 3: Either ambulatory or non-ambulatory, Cohort 5: Non-ambulatory, but having been previously ambulatory by history8669023Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype, confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion8669034Negative for AAV antibodies8669045Steroid regimen: a. Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice. b. Cohort 3: N/A8669056Meet 10-meter walk/run time criteria8669067Meet time to rise from supine criteria8669078Cohort 5: meet Performance of Upper Limb (PUL) 2.0 criteria8669089Participant has body weight ≤ 90 kg.14824741Any prior or ongoing medical condition, medical history, physical finding that in the Investigator's opinion could adversely affect the safety of the participant, make it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.148247510Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.148247611Major surgery within 3 months prior to recruitment or planned orthopedic surgery for any time during this study which would interfere with the ability to perform outcome measures.148247712Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted to not express exons 1 to 11, exons 42 to 45, or exons 57 to 69 inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.148247813Sponsor employees and their family members are ineligible to participate in this study.148247914Any active infection14824802Abnormal liver function, evidence of active viral hepatitis14824813Abnormal renal function14824824Clinically significant abnormalities of coagulation14824835Impaired cardiovascular function14824846Pulmonary function predictive of (or requiring) the use of daytime ventilatory support14824857Have severe hypersensitivity reactions, including anaphylaxis, to SGT-003 or its components.14824868Treatment with dystrophin modifying drugs within 3 months prior to screening.14824879Current or prior treatment with an approved or investigational gene transfer drug.10149491Incidence of treatment-emergent adverse events (AEs) through Day 360True10149502Change from baseline of microdystrophin protein levels at Day 90True10149511Change from baseline of microdystrophin tissue distribution at Day 90False10149522Change from baseline of microdystrophin tissue distribution at Day 360False10149533Change from baseline of microdystrophin protein levels at Day 360False10149544Change from baseline in time to rise velocity at Day 360 and Day 540False10149555Change from baseline in stride velocity 95th centile (SV95C) at Day 360 and Day 540False10149566Change from baseline in 10-meter walk/run velocity at Day 360 and Day 540False10149577Change from baseline in 4-stair climb velocity at Day 360 and Day 540False10149588Change from baseline in the North Star Ambulatory Assessment (NSAA) score at Day 360 and Day 540False10149599Change from baseline in 6-minute walk test (6MWT) distance at Day 360 and Day 540False101496010Incidence of clinically significant laboratory abnormalities through Day 360 and Day 540False101496111Incidence of clinically significant abnormalities in vital signs through Day 360 and Day 540False101496212Incidence of clinically significant abnormalities in physical examinations through Day 360 and Day 540False101496313Incidence of cardiac abnormalities by electrocardiogram (ECG) or echocardiography (ECHO) through Day 360 and Day 540False2031-05-062031-05-032025-01-0293876Solid Biosciences Inc.35215522352156252535215922223521602424352158262635215723232PatientsFalseTrueTrue1acceptable2026-04-022026-04-025Diseases [C] - Musculoskeletal Diseases [C05]133711Duchenne muscular dystrophyTrue145456True429046PublicKelsey Gendakgenda@solidbio.com+16173374680480222Pharmaceutical company10Solid Biosciences Inc.TrueTrueORG-100011419Active429047ScientificKelsey Gendakgenda@solidbio.com+16173374680480222Pharmaceutical company10Solid Biosciences Inc.TrueTrueORG-100011419Active656344594951627760Laboratory/Research/Testing facility9Diverge Translational Science LaboratoryTrueTrueORG-100051693Active642080247 West Freshwater Way Suite 600247 West Freshwater Way Suite 600Milwaukee53204-4117840United States+19049944038Diverge@stl.comTrueORG-100051693101893315Muscle Pathology, Microsystrophin Expression Analysis+19049944038Diverge@stl.com656335594919627728Pharmaceutical company10Voisin Consulting CH SARLTrueTrueORG-100031396Active642046Innovation Park Batiment F, EpflInnovation Park Batiment FEpflLausanne1015756Switzerland+41215470431brehm@voisinconsulting.comTrueORG-100031396101891712+41215470431brehm@voisinconsulting.com656343594959627768Pharmaceutical company10Charles River Laboratories Inc.TrueTrueORG-100011991Active6420886995 Longley Lane6995 Longley LaneReno89511-1227840United States+17756822298charles@riverlabs.comTrueORG-100011991101893215Viral vector Shedding Analysis+17756822298charles@riverlabs.com656341594953627762Pharmaceutical company10Flagship Biosciences Inc.TrueTrueORG-100043268Active64208211800 Ridge Parkway Suite 45011800 Ridge Parkway Suite 450Broomfield80021-6503840United States+13033255894flagsgip@biosciences.comTrueORG-100043268101892315Microdystrophin Distribution Analysis+13033255894flagsgip@biosciences.com656336597462630285Pharmaceutical company10ViroClinics Biosciences B.V.TrueTrueORG-100046320Active644913Marconistraat 16Marconistraat 16Rotterdam3029 AK2029Netherlands0080060044600cerba@research.comTrueORG-100046320101891815AAV9 Antibody Testing0080060044600cerba@research.com656340594922627731Laboratory/Research/Testing facility9Eurofins Central Laboratory LLCTrueTrueORG-100043608Active6420492430 New Holland Pike2430 New Holland PikeLancaster17601-5964840United States+17176562300eurofins@us.comTrueORG-10004360810189224+17176562300eurofins@us.com656342594917627726Pharmaceutical company10Parexel International (IRL) LimitedTrueTrueORG-100022780Active64204470 Sir John Rogerson's Quay70 Sir John Rogerson's QuayDublin 2D02 R2962017Ireland+35314739500clinicaltrial.enquiries@parexel.comTrueORG-10002278010189241101892510101892611101892715Lab supplies management, Medical Monitoring10189282101892951018930610189318+35314739500clinicaltrial.enquiries@parexel.com656338594949627758Laboratory/Research/Testing facility9Machaon Diagnostics Inc.TrueTrueORG-100050406Active6420782023 8th Street2023 8th StreetBerkeley94710-2026840United States+15108395600machaon@diagnostics.comTrueORG-100050406101892015sC5b-9 Testing+15108395600machaon@diagnostics.com656337594931627740Pharmaceutical company10Eurofins Central Laboratory B.V.TrueTrueORG-100036990Active642058Bergschot 71Bergschot 71Breda4817 PA2029Netherlands+31765737373eurofins@us.comTrueORG-10003699010189194+31765737373eurofins@us.com656345594950627759Pharmaceutical company10Bioagilytix Labs LLCTrueTrueORG-100013030Active6420792300 Englert Drive Suite G2300 Englert Drive Suite GDurham27713-4450840United States+19193816097bio@agilytix.comTrueORG-100013030101893415Anti-SGT-003 antibody testing and cellular immune response testing to microdystrophin+19193816097bio@agilytix.com656339594955627764Pharmaceutical company10Worldwide Clinical Trials Early Phase Services LLCTrueTrueORG-100032461Active6420844509 Freidrich Lane Building 2 Suite 2004509 Freidrich Lane Building 2 Suite 200Austin78744-1866840United States+12106405664clinicaltrials@worldwide.comTrueORG-100032461101892115Microdystrophin Expression Analysis+12106405664clinicaltrials@worldwide.com480222Pharmaceutical company10Solid Biosciences Inc.TrueTrueORG-100011419Active447943480222Pharmaceutical company10Solid Biosciences Inc.TrueTrueORG-100011419Active479815500 Rutherford Avenue500 Rutherford AvenueCharlestown02129-1647840United StatesTrueORG-100011419TrueCommercial2This is a Phase I/II integrated trial11459165Diseases [C] - Musculoskeletal Diseases [C05]397105nullDE_HE_01_MIA_2022_0026/23.1 (Pl) 18 L 18.01/1113-B1Test532691111621322SUSPENSION FOR IV INFUSIONPRD116213221SUSPENSION FOR IV INFUSIONSGT-003SGT-003SUB396386SOLID BIOSCIENCES, LLC11621322396387SOLID BIOSCIENCES, LLCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherSGT-003SUB396386False1FalseGene therapyPRD11621322SGT-003DE_HE_01_MIA_2022_0026/23.1 (Pl) 18 L 18.01/1113-B67853human microdystrophinadeno-associated virusFalseTrue311INTRAVENOUS USEFalsesgt-003SUSPENSION FOR IV INFUSIONFalse3027793436134361153822018Authorised2024-12-162024-12-16TrueItaly2025-03-18Authorised592372025-06-202025-06-23592382025-06-202025-06-23847302025-08-072025-08-11847312025-08-072025-08-11True876102025-06-202025-06-232025-08-07False5408034361Under evaluation2024-09-26T17:00:08.0127481434361Authorised2025-01-27T19:14:30.3131Italyauthorized2025-03-182025-03-1882103902594994627803Hospital/Clinic/Other health care facility8Fondazione Policlinico Universitario Agostino Gemelli IRCCSFalseTrueORG-100014358Active642125Largo Francesco Vito 1Largo Francesco Vito 1Rome001682018Italy+390630157062eugeniomaria.mercuri@policlinicogemelli.itTrueORG-1000143582334899EugenioMercuri+390630157062eugeniomaria.mercuri@policlinicogemelli.it1UOC Neuropsichiatria InfantileAuthorised26173INITIALAuthorised2024-514501-57-00Authorised2024-09-26acceptable2025-01-211665093436134361Italy2018acceptable2025-01-21authorized2025-03-18AuthorisedItalyAuthorised2025-03-18Authorised2025-01-2734361ItalyAuthorisedINFalse551802617334361Italy2025-01-27T19:14:30.743authorizedacceptabledecision16650967895INITIALTrue50948NON SUBSTANTIAL MODIFICATIONAuthorised2024-514501-57-00Authorised2025-03-18acceptable2025-01-211911193436134361Italy2018authorized2025-03-18AuthorisedItalyAuthorised2025-03-18Authorised2025-03-1834361ItalyAuthorisedNSM-2PART_IIFalse626615094834361Italy2025-03-18T13:10:12.475authorizeddecision19111978525NON SUBSTANTIAL MODIFICATIONTrue55113SUBSTANTIAL 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2024-519004-27-002AuthorisedA PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHYDT-DEC01-DMD-CT-1/2Duchenne Muscular DystrophyPoland:220/12/2024PL: 20/12/2024Diseases [C] - Musculoskeletal Diseases [C05]Dystrogen Therapeutics Technology Polska Sp. z o.o.Pharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansPhase 2: Mean changes from the baseline (V0a) recorded by functional assessments (adjusted to the stage of the disease): for ambulatory patients: PUL 2.0, assessment of grip strength by dynamometer; for non-ambulatory patients: assessment of grip strength by dynamometer. At month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Mean changes from the baseline (V0a) in QoL assessed by PODCI at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Evaluation of Overall Treatment Effect at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).DT-DEC014560-17 yearsMale1EEA20Phase 1: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI., Phase 1: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run, for non-ambulatory patients: PUL 2.0, at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 1: Mean changes from the baseline (V0a) in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients, at month 3 (V5), 6 (V6), and 12 (V7)., Phase 2: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI., Phase 2: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: a. for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run b. for non-ambulatory patients: PUL 2.0 at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Mean changes from the baseline in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients at month 3, 6 and 12 .No24/04/202625/04/20262024-12-20T11:01:29.662026-04-25T03:30:58.3996357912D1_Protocol EU CT 2024-519004-27-00 for publication5769c660-163e-4308-9396-e4bb3c4de932104Protocol (for publication)PDF1226741.3.25K1_Recruitment arrangementsfcf77f53-f0d5-4f82-a7b6-31b1f0eea1a114Recruitment arrangements (for publication)PDF2748551.02L1_ICF FSZ DLA RODZICOW ORAZ MAOLETNIEGO ktory ukonczy 13 r-z for publication04e2a19f-5420-4f75-a8dd-d63c3641ee3a15Subject information and informed consent form (for publication)PDF2748551.54L1_ICF FSZ DLA RODZICOW MAOLETNIEGO ponizej 13 r-z20b3089f-7701-44fb-b0e2-1939719b232f15Subject information and informed consent form (for publication)PDF2748551.54L1_ICF FSZ DLA DAWCY for publication910cc194-3b64-4c96-9118-0771da270fee15Subject information and informed consent form (for publication)PDF2748551.34L1_ICF PFI ponizej 13 r-z for publicationa76fb252-cf75-41fd-9270-3230e0d5c08015Subject information and informed consent form (for publication)PDF2748551.54L1_ICF serbski FSZ DLA DAWCY for publication7a6eab57-d126-4acc-9259-453dfd0c9a7615Subject information and informed consent form (for publication)PDF2748551.33L1_ICF serbski FSZ DLA RODZICOW MAOLETNIEGO ponizej 13 r-z for publication00636ed8-e649-499b-aaac-b9b3328bcf5a15Subject information and informed consent form (for publication)PDF2748551.53L1_ICF serbski FSZ DLA RODZICOW i MAOLETNIEGO ktory ukonczy 13 r-z for publicationd658ea4e-03ad-40e3-b5da-0bee7d5debef15Subject information and informed consent form (for publication)PDF2748551.53L1_ICF serbski PFI ponizej 13 r-z for publicationeee498c1-c4c5-42a7-a1a0-c8736b5220b415Subject information and informed consent form (for publication)PDF2748551.53L1_Recruitment advertisement for clinical trial participants v1_0 21Nov2022527001a0-6184-4691-a0dd-6f20274eb38215Subject information and informed consent form (for publication)PDF2748551.01Poland413272024-12-20T11:01:29.662026-04-24T13:05:46.266241327Poland2024-519004-27-002A PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHYDT-DEC01-DMD-CT-1/2Duchenne Muscular DystrophyPoland:220/12/2024PL: 20/12/2024Diseases [C] - Musculoskeletal Diseases [C05]Dystrogen Therapeutics Technology Polska Sp. z o.o.Pharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansPhase 2: Mean changes from the baseline (V0a) recorded by functional assessments (adjusted to the stage of the disease): for ambulatory patients: PUL 2.0, assessment of grip strength by dynamometer; for non-ambulatory patients: assessment of grip strength by dynamometer. At month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Mean changes from the baseline (V0a) in QoL assessed by PODCI at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Evaluation of Overall Treatment Effect at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).DT-DEC014560-17 yearsMale120Phase 1: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI., Phase 1: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run, for non-ambulatory patients: PUL 2.0, at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 1: Mean changes from the baseline (V0a) in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients, at month 3 (V5), 6 (V6), and 12 (V7)., Phase 2: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI., Phase 2: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: a. for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run b. for non-ambulatory patients: PUL 2.0 at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Mean changes from the baseline in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients at month 3, 6 and 12 .No24/04/202625/04/20262024-519004-27-00Authorised2024-12-20T11:01:29.662026-04-25T03:30:58.39963579121226740508850111689940SUSPENSION FOR INJECTIONPRD116899401DT-DEC01SUSPENSION FOR INJECTIONEX VIVO FUSED NORMAL ALLOGENEIC HUMAN MYOBLAST WITH AUTOLOGOUS HUMAN MYOBLAST DERIVED FROM DUCHENNE MUSCULAR DYSTROPHY AFFECTED DONORSUB193214MBN/MBDMD DECDYSTROGEN THERAPEUTICS TECHNOLOGY POLSKA SP. Z O.O.11689940289851DYSTROGEN THERAPEUTICS TECHNOLOGY POLSKA SP. Z O.O.Structurally Diverse Substance - Cell therapyStructurally Diverse Substance - Cell therapyEX VIVO FUSED NORMAL ALLOGENEIC HUMAN MYOBLAST WITH AUTOLOGOUS HUMAN MYOBLAST DERIVED FROM DUCHENNE MUSCULAR DYSTROPHY AFFECTED DONORSUB193214MB(N)/MB(DMD) DECMBN/MBDMD Dystrophin Expressing Chimeric CellFalse1TrueEU/3/18/2089tissue engineered productPRD116899406424EMA/H0005097223Chimeric cells produced by ex vivo fusion of normal allogeneic human myoblast (MBN) with autologous human myoblast derived from Duchenne muscular dystrophy affected donor (MBDMD)11INTRAOSSEOUS USEFalseDT-DEC01ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from duchenne muscular dystrophy affected donorSUSPENSION FOR INJECTIONA PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY10140958519494b6-2a69-4d1c-b35d-7852eb380f34BADANIE KLINICZNE FAZY 1/2 (OTWARTE, EKSPLORACYJNE) OCENIAJĄCE BEZPIECZEŃSTWO I SKUTECZNOŚĆ TERAPII DT-DEC01 U PACJENTÓW Z DYSTROFIĄ MIĘŚNIOWĄ DUCHENNE’A19PolishA PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY10140967039937f0-c441-4350-9521-34d6325e1cfbBADANIE KLINICZNE FAZY 1/2 (OTWARTE, EKSPLORACYJNE) OCENIAJĄCE BEZPIECZEŃSTWO I SKUTECZNOŚĆ TERAPII DT-DEC01 U PACJENTÓW Z DYSTROFIĄ MIĘŚNIOWĄ DUCHENNE’A19PolishDT-DEC01-DMD-CT-1/2False72This is a Phase 1/2 study designed to evaluate the safety and efficacy of DT-DEC01 in DMD subjects of age 5-18 years old assessed for the treatment of Duchenne Muscular Dystrophy. In accordance with EMA guidance on disclosure rules, the study is classified as a Category 2 trial.112550127684Duchenne Muscular DystrophyTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False43715875371588Phase 1 primary objectives include (i) evaluation of safety by clinical observation of the incidence and severity of all adverse events: Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESI) to assess the potential risks (ii) evaluation of efficacy, based on functional assessments of muscle strength and function adjusted to the stage of the disease: (a) for ambulatory patients: Six-Minute Walk Test (6MWT); timed functions of NorthStar Ambulatory Assessment (NSAA): supine to rise and 10-meter walk/run); (b) for non-ambulatory patients: PUL 2.0 and (c) assessments for both ambulatory and non-ambulatory patients: electromyography (EMG) assessment of duration of motor unit potential (MUP) of the selected muscles. Phase 2 primary objectives include evaluation of safety by clinical observation of the incidence and severity of all adverse events: AEs, SAEs and AESI and to assess the potential risks; and evaluation of the efficacy, based on the functional assessments of muscle strength and function adjusted to the stage of the disease including: (a) for ambulatory patients: 6MWT; timed functions of NSAA: supine to rise and 10-meter walk/run), (b) for non-ambulatory patients: PUL 2.0, (c) for both, the ambulatory and non-ambulatory patients: EMG assessment of duration of the MUP of the selected muscles.10140966ade61a1a-1ec7-4b42-99e5-b98675fdb2e6Główne cele fazy 1 obejmują (i) ocenę bezpieczeństwa poprzez kliniczną obserwację częstości występowania i ciężkości wszystkich zdarzeń niepożądanych: zdarzeń niepożądanych (Adverse Events, AE), ciężkich zdarzeń niepożądanych (Serious Adverse Events, SAE) i zdarzeń niepożądanych o szczególnym znaczeniu (Adverse Events of Special Interest, AESI) w celu oceny potencjalnych ryzyk; (ii) ocenę skuteczności na podstawie oceny funkcjonalnej siły i funkcji mięśni, dostosowanej do stadium choroby: (a) dla pacjentów chodzących: 6-Minute Walk Test (6MWT); funkcje czasowe NorthStar Ambulatory Assessment (NSAA): wstanie z pozycji leżącej na plecach oraz 10-metrowy marsz/bieg; (b) dla pacjentów niechodzących: PUL 2.0 oraz (c) zarówno dla pacjentów chodzących, jak i niechodzących: ocena elektromiograficzna (EMG) czasu trwania potencjału jednostek motorycznych (Motor Unit Potential, MUP) wybranych mięśni. Główne cele fazy 2 obejmują ocenę bezpieczeństwa poprzez kliniczną obserwację częstości występowania i ciężkości wszystkich zdarzeń niepożądanych: zdarzeń niepożądanych (Adverse Events, AE), ciężkich zdarzeń niepożądanych (Serious Adverse Events, SAE) i zdarzeń niepożądanych o szczególnym znaczeniu (Adverse Events of Special Interest, AESI) w celu oceny potencjalnych ryzyk; ocenę skuteczności na podstawie oceny funkcjonalnej siły i funkcji mięśni, dostosowanej do stadium choroby: (a) dla pacjentów chodzących: 6-Minute Walk Test (6MWT); funkcje czasowe NorthStar Ambulatory Assessment (NSAA): wstanie z pozycji leżącej na plecach oraz 10-metrowy marsz/bieg; (b) dla pacjentów niechodzących: PUL 2.0 oraz (c) zarówno dla pacjentów chodzących, jak i niechodzących: ocena elektromiograficzna (EMG) czasu trwania potencjału jednostek motorycznych (Motor Unit Potential, MUP) wybranych mięśni.19Polish8264581Subject and his legal representative/parent(s)/legal guardian have understood and accepted to participate in the study according to all study procedures by signing the approved informed consent.101409593b35d593-a777-46c8-b511-2db8d4febe75Uczestnik i jego przedstawiciel ustawowy / rodzic(e) / opiekun prawny zrozumieli i wyrazili zgodę na udział w badaniu zgodnie ze wszystkimi procedurami badania, podpisując zatwierdzony formularz świadomej zgody.19Polish8264592Boys of age 5 to 18 years old (at the time of screening), diagnosed with DMD confirmed by genetic testing.10140960f9ca6a7f-b2c4-4cc0-8e45-1c4d7aa1b914Chłopcy w wieku od 5 do 18 lat (w momencie wizyty przesiewowej), u których zdiagnozowano dystrofię mięśniową Duchenne’a potwierdzoną badaniami genetycznymi.19Polish8264603Subjects must be receiving glucocorticosteroids for a minimum of 6 months prior to the biopsy of muscle tissue.101409618c004b04-b75a-44b6-918e-84e36a5e1944Uczestnicy muszą przyjmować glikokortykosteroidy przez co najmniej 6 miesięcy przed biopsją tkanki mięśniowej.19Polish8264614Subjects with progressive, symmetrical proximal muscle weakness of arms and legs.101409620e91c130-6a9b-4b1d-aed3-87aa1ce64767Uczestnicy wykazują postępującą i symetryczną utratę siły mięśniowej w odcinkach proksymalnych kończyn górnych i dolnych.19Polish8264625Willingness and ability to comply with scheduled visits, tissue biopsy procedure under anesthesia, drug administration plan, laboratory tests, study restrictions, study procedures, and functional testing adapted to the stage of the disease.10140963f4a1536d-69a4-4807-9f4e-3234dd68edf0Uczestnicy wykazują gotowość i zdolność do przestrzegania terminów zaplanowanych wizyt, poddania się procedurze biopsji tkanek w znieczuleniu, przestrzegania planu przyjmowania leków, poddawania się badaniom laboratoryjnym, zastosowania się do ograniczeń spowodowanych udziałem w badaniu oraz zastosowania się do procedur badania i wykonywania badań funkcjonalnych dostosowanych do stopnia zaawansowania choroby.19Polish8264636Willingness to use acceptable forms of contraception if the subject is sexually active.10140964bbc613e4-0832-47c5-bce1-e1a13b121c8fUczestnicy wykazują gotowość do stosowania akceptowalnych metod antykoncepcji, jeśli są aktywni seksualnie.19Polish8264647Patients must be cleared by anesthesiologist for tissue biopsy and DT-DEC01 intraosseous injection procedures which will be performed under anesthesia (local anesthesia / general anesthesia / analgosedation).10140965b5981b9a-4442-498a-a65f-4c73f4d1a4f3Pacjenci muszą pomyślnie przejść kwalifikację anestezjologiczną do pobrania biopsji tkanki mięśniowej i zabiegu doszpikowego podania DT-DEC01, które zostaną wykonane w znieczuleniu (miejscowym/ogólnym/anagosedacji).19Polish14141151Subject was previously exposed to the IMP, any similar experimental therapy with the use of ATMP or any other cell-based product, gene therapy or translarna / ataluren prior to screening.1014094490299eb3-a665-472c-8b9f-efdaf2fc43b6Uczestnika poddano uprzednio leczeniu badanym produktem leczniczym, podobną terapią eksperymentalną z zastosowaniem ATMP lub innym produktem opartym na komórkach, terapią genową lub lekiem translarna/ataluren.19Polish141411610Any injury or procedure which may impact functional testing. Previous injuries must be fully healed prior to consenting. Prior lower limb fractures must be fully healed and at least 3 months should pass from injury date.10140945d8a67b6e-8861-4a7a-8b74-c43c296cb17dU uczestnika wystąpił jakikolwiek uraz lub zabieg mogący utrudnić ocenę funkcjonalną. Przebyte urazy muszą być całkowicie wyleczone przed wyrażeniem zgody. Przebyte złamania kończyn dolnych muszą być całkowicie wyleczone i od daty urazu muszą upłynąć co najmniej 3 miesiące.19Polish141411711Presence or history of other musculoskeletal or neurologic disease or somatic disorder not related to DMD including pulmonary and cardiac disease.10140946cc3ea729-02df-4ce8-a867-417e64caadc0U uczestnika stwierdzono obecność lub historię innych chorób układu mięśniowo-szkieletowego, neurologicznych lub zaburzeń somatycznych niezwiązanych z DMD, w tym chorób płuc i serca.19Polish141411812History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of the investigational product.101409476a83bfb3-bd31-405a-beaa-09330565a87fW przeszłości wystąpiła u uczestnika reakcja alergiczna lub anafilaktyczna na białko terapeutyczne lub diagnostyczne lub na substancje dodatkowe badanego produktu leczniczego.19Polish141411913Ongoing chronic use of any agents with an immunomodulating (activating or suppressing) effect, such as, but not limited to, immunosuppressants or drugs related to immunotherapy (e.g. based on antibodies), chemotherapy or similar therapy affecting cell proliferation.1014094807caa099-d6f0-43b2-b0b4-75439636992cUczestnik obecnie przewlekle stosuje jakiekolwiek środki o działaniu immunomodulującym (aktywującym lub hamującym), takie jak (ale nie wyłącznie) leki immunosupresyjne lub leki związane z immunoterapią (np. oparte na przeciwciałach), chemioterapią lub podobną terapią wpływającą na proliferację komórek.19Polish141412014Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for inclusion into this study.10140949081743c5-7f57-410f-8767-993bc4199de3U uczestnika stwierdzono inny poważny, ostry lub przewlekły stan chorobowy lub psychiczny albo nieprawidłowość w wynikach badań laboratoryjnych, które mogą zwiększać ryzyko związane z uczestnictwem w badaniu lub podawaniem badanego produktu lub mogą wpływać na interpretację wyników badania i, w opinii badacza, sprawiać, że dana osoba nie nadaje się do włączenia do tego badania.19Polish14141212Subject has known history of immune reaction to the administered therapies or history of GvHD.1014095026501da5-9f16-4f43-9fbe-a04e80d36e66W przeszłości wystąpiła u uczestnika reakcja immunologiczna na podany lek lub GvHD.19Polish14141223Subjects with known cognitive impairment or behavioral issues that would impede the ability to follow instructions.1014095158fd6d91-5c23-4a77-9b8c-a96f47c6a06dU uczestnika stwierdzono zaburzenia sfery poznawczej lub problemy z zachowaniem, które upośledzałyby zdolność do wykonywania poleceń.19Polish14141234Subject is positive for human immunodeficiency virus (HIV) 1 or 2, hepatitis B (HBV) or C (HCV*), cytomegalovirus (CMV*), toxoplasmosis* or syphilis at screening visit (V0a). *In case of positive or doubtful result of anti-CMV IgG and anti-Toxo IgG, anti-HCV total the final qualification decision can be made after confirming the negative Nucleic Acid Test (NAT) results in cell culture.101409524d6200e9-c1e5-4be5-ab75-30fc3826b7c9U uczestnika stwierdzono podczas wizyty przesiewowej (wizyta V0a) dodatnie wyniki badań w kierunku zakażenia: ludzkim wirusem niedoboru odporności (HIV) 1 lub 2, wirusem zapalenia wątroby typu B (HBV) lub C (HCV*), wirusem cytomegalii (CMV*), toksoplazmozą* lub kiłą. *W przypadku pozytywnego lub wątpliwego wyniku testu Anty-CMV IgG, Anty- Toxo IgG, Anty-HCV Total decyzja o ostatecznej kwalifikacji tkanki może zostać podjęta po potwierdzeniu ujemnego wyniku testu na obecność kwasu nukleinowego wirusa (NAT) na etapie hodowli komórkowej.19Polish14141245Subject has a history of any autoimmune disease.101409538eedb6da-7215-4c09-a910-063dee4ebe4fW przeszłości wystąpiła u uczestnika choroba o podłożu autoimmunologicznym.19Polish14141256Ongoing participation in any other therapeutic clinical trial.10140954b471eb33-bf21-4019-b350-a8576a148c59Uczestnik aktualnie bierze udział w jakimkolwiek innym badaniu klinicznym o charakterze terapeutycznym.19Polish14141267Presence of pre-existing antibodies in the subject’s serum against the donor lymphocytes.10140955713a920f-0358-459d-b750-b59454b94e3dW surowicy uczestnika wykryto obecność przeciwciał skierowanych przeciwko limfocytom dawcy.19Polish14141278Subject has undergone an organ or bone marrow transplantation.101409563b8e8dcf-1563-4b49-a573-69f842d15efbUczestnik przeszedł przeszczep narządu lub szpiku kostnego.19Polish14141289Change in systemic corticosteroid therapy (e.g. initiation of treatment; cessation of treatment; change in dose, schedule, or type of steroid) within 3 months prior to administration of the investigational product.101409570692df7f-da04-47c9-a3cd-3e88429a9747U uczestnika wprowadzono zmianę w ogólnoustrojowym leczeniu kortykosteroidami (np. rozpoczęcie leczenia, przerwanie leczenia, zmiana dawki, schematu lub rodzaju sterydów) w ciągu 3 miesięcy przed podaniem produktu badanego.19Polish9684471Phase 1: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI.True101409357f451d23-719b-4146-a089-8c5d3b9cb628Faza 1: Częstość występowania i ciężkość wszystkich zdarzeń niepożądanych: zdarzeń niepożądanych (Adverse Events, AE), ciężkich zdarzeń niepożądanych (Serious Adverse Events, SAE) i zdarzeń niepożądanych o szczególnym znaczeniu (Adverse Events of Special Interest, AESI).19Polish9684482Phase 1: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run, for non-ambulatory patients: PUL 2.0, at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).True10140936af8f09cc-cd86-47af-931e-fb696127bfd9Faza 1: Średnie zmiany w stosunku do wartości wyjściowych (V0a) odnotowane w ocenie funkcjonalnej dostosowanej do stopnia zaawansowania choroby: dla pacjentów chodzących: 6MWT, funkcje czasowe NSAA: wstanie z pozycji leżącej na plecach oraz 10-metrowy marsz/bieg, dla pacjentów niechodzących: PUL 2.0, w miesiącu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).19Polish9684493Phase 1: Mean changes from the baseline (V0a) in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients, at month 3 (V5), 6 (V6), and 12 (V7).True1014093707d2d93c-c972-4497-b9f9-34fd784c17afFaza 1: Średnie zmiany w stosunku do wartości wyjściowych (V0a) w ocenie EMG czasu trwania MUP wybranych mięśni kończyn górnych i dolnych u pacjentów chodzących i niechodzących, w miesiącu 3 (V5), 6 (V6) i 12 (V7).19Polish9684504Phase 2: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI.True101409383e65f314-3fd1-4fcb-8ae7-c5d49f19e385Faza 2: Częstość występowania i ciężkość wszystkich zdarzeń niepożądanych: zdarzeń niepożądanych (Adverse Events, AE), ciężkich zdarzeń niepożądanych (Serious Adverse Events, SAE) i zdarzeń niepożądanych o szczególnym znaczeniu (Adverse Events of Special Interest, AESI).19Polish9684515Phase 2: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: a. for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run b. for non-ambulatory patients: PUL 2.0 at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).True101409399d4cbda6-146b-413d-84d5-4388bb8ecb60Faza 2: Średnie zmiany w stosunku do wartości wyjściowych (V0a) odnotowane w ocenie funkcjonalnej dostosowanej do stopnia zaawansowania choroby: dla pacjentów chodzących: 6MWT, funkcje czasowe NSAA: wstanie z pozycji leżącej na plecach oraz 10-metrowy marsz/bieg, dla pacjentów niechodzących: PUL 2.0, w miesiącu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).19Polish9684526Phase 2: Mean changes from the baseline in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients at month 3, 6 and 12 .True101409405a91b7ea-0ead-4af8-9c33-4feaccef941fFaza 2: Średnie zmiany w stosunku do wartości wyjściowych (V0a) w ocenie EMG czasu trwania MUP wybranych mięśni kończyn górnych i dolnych u pacjentów chodzących i niechodzących, w miesiącu 3 (V5), 6 (V6) i 12 (V7).19Polish9684531Phase 2: Mean changes from the baseline (V0a) recorded by functional assessments (adjusted to the stage of the disease): for ambulatory patients: PUL 2.0, assessment of grip strength by dynamometer; for non-ambulatory patients: assessment of grip strength by dynamometer. At month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).False101409410c1dbce8-d34b-422c-8f22-a06175aec546Faza 2: Średnie zmiany w stosunku do wartości wyjściowych (V0a) odnotowane w ocenie funkcjonalnej dostosowanej do stopnia zaawansowania choroby: dla pacjentów chodzących: PUL 2.0, ocena siły uścisku ręki za pomocą dynamometru, dla pacjentów niechodzących: ocena siły uścisku ręki za pomocą dynamometru, w miesiącu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).19Polish9684542Phase 2: Mean changes from the baseline (V0a) in QoL assessed by PODCI at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).False101409428e723114-3642-4e36-a171-914f4392cac2Faza 2: Średnie zmiany w stosunku do wartości wyjściowych (V0a) w jakości życia (Quality of Life, QoL) ocenianej w kwestionariuszu PODCI (Pediatrics Outcomes Data Collection Instrument) w miesiącu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).19Polish9684553Phase 2: Evaluation of Overall Treatment Effect at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).False10140943d0d250b8-7220-4555-9a72-4c3850210452Faza 2: Ocena ogólnego wyniku leczenia za pomocą kwestionariusza Overall Treatment Effect (OTE) w miesiącu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).19Polish2030-12-312027-12-12336935223369382424336937252533693626262PatientsFalseTrueTrue252072902315944409EU Institution/Body/Agency14European Medicines AgencyFalseTrueORG-100013412Active1009021Domenico Scarlattilaan 6Domenico Scarlattilaan 6Amsterdam1083 HS2029NetherlandsTrueORG-10001341210915EMEA-111111-PIP11-11acceptable2026-04-202026-04-205Diseases [C] - Musculoskeletal Diseases [C05]127684Duchenne Muscular DystrophyTrue138987True409716PublicContact Pointsiemiom@dystrogen.com+48530711336664419Pharmaceutical company10Dystrogen Therapeutics Technology Polska Sp. z o.o.TrueTrueORG-100045218Active409717ScientificContact Pointsiemiom@dystrogen.com+48530711336664419Pharmaceutical company10Dystrogen Therapeutics Technology Polska Sp. z o.o.TrueTrueORG-100045218Active664419Pharmaceutical company10Dystrogen Therapeutics Technology Polska Sp. z o.o.TrueTrueORG-100045218Active626513664419Pharmaceutical company10Dystrogen Therapeutics Technology Polska Sp. z o.o.TrueTrueORG-100045218Active677692Ul. Wladyslawa Pytlasinskiego 10/12/14Ul. Wladyslawa Pytlasinskiego 10/12/14Warsaw00-7772031PolandTrueORG-100045218TrueCommercial2This is a Phase 1/2 study designed to evaluate the safety and efficacy of DT-DEC01 in DMD subjects of age 5-18 years old assessed for the treatment of Duchenne Muscular Dystrophy. In accordance with EMA guidance on disclosure rules, the study is classified as a Category 2 trial.11102595Diseases [C] - Musculoskeletal Diseases [C05]380092DT-DEC011Test508850111689940SUSPENSION FOR INJECTIONPRD116899401DT-DEC01SUSPENSION FOR INJECTIONEX VIVO FUSED NORMAL ALLOGENEIC HUMAN MYOBLAST WITH AUTOLOGOUS HUMAN MYOBLAST DERIVED FROM DUCHENNE MUSCULAR DYSTROPHY AFFECTED DONORSUB193214MBN/MBDMD DECDYSTROGEN THERAPEUTICS TECHNOLOGY POLSKA SP. Z O.O.11689940289851DYSTROGEN THERAPEUTICS TECHNOLOGY POLSKA SP. Z O.O.Structurally Diverse Substance - Cell therapyStructurally Diverse Substance - Cell therapyEX VIVO FUSED NORMAL ALLOGENEIC HUMAN MYOBLAST WITH AUTOLOGOUS HUMAN MYOBLAST DERIVED FROM DUCHENNE MUSCULAR DYSTROPHY AFFECTED DONORSUB193214MB(N)/MB(DMD) DECMBN/MBDMD Dystrophin Expressing Chimeric CellFalse1TrueEU/3/18/2089tissue engineered productPRD116899406424EMA/H0005097223Chimeric cells produced by ex vivo fusion of normal allogeneic human myoblast (MBN) with autologous human myoblast derived from Duchenne muscular dystrophy affected donor (MBDMD)11INTRAOSSEOUS USEFalseDT-DEC01ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from duchenne muscular dystrophy affected donorSUSPENSION FOR INJECTIONFalse2748554132741327202142031Authorised2024-11-142024-11-14TruePoland2024-12-20AuthorisedFalseFalse5870641327Under evaluation2024-10-25T10:54:45.967291041327Authorised2024-12-20T11:01:29.3721Poland2027-12-12acceptable2026-03-18authorized2024-12-202024-12-20201884874628053665960Hospital/Clinic/Other health care facility8Med Polonia Sp. z o.o.FalseTrueORG-100041354Active679398Obornicka 262Obornicka 262Poznan60-6932031Poland+48573805308badania.kliniczne@medpolonia.plTrueORG-1000413542094396AnnaUrbańska+48573805308badania.kliniczne@medpolonia.plBadania KliniczneAuthorised38280INITIALAuthorised2024-519004-27-00Authorised2024-10-25acceptable2024-12-171568324132741327Poland2031acceptable2024-12-17authorized2024-12-20AuthorisedPolandAuthorised2024-12-20Authorised2024-12-2041327PolandAuthorisedINFalse519273828041327Poland2024-12-20T11:01:29.66authorizedacceptabledecision15683263376INITIALTrue60405SUBSTANTIAL MODIFICATIONAuthorised2024-519004-27-00Authorised2025-08-08acceptable2025-10-062476124132741327Poland2031acceptable2025-10-31authorized2024-12-20AuthorisedPolandAuthorised2024-12-20Authorised2025-11-0441327PolandAuthorisedSM-1PART_I_AND_PART_IIFalse965746040541327Poland2025-11-04T12:51:32.534authorizedacceptabledecision24761293148SUBSTANTIAL MODIFICATIONTrue75234SUBSTANTIAL MODIFICATIONAuthorised2024-519004-27-00Authorised2026-02-02acceptable2026-04-202748554132741327Poland2031acceptable2026-03-18authorized2024-12-20AuthorisedPolandAuthorised2024-12-20Authorised2026-04-2441327PolandAuthorisedSM-2PART_I_AND_PART_IIFalse1208767523441327Poland2026-04-24T13:05:46.266authorizedacceptabledecision274855122674SUBSTANTIAL MODIFICATIONTruePoland413272024-12-20T11:01:29.662026-04-24T13:05:46.2662True2022-003126-4241327PolandD1_Protocol EU CT 2024-519004-27-00 for publication5769c660-163e-4308-9396-e4bb3c4de932104Protocol (for publication)PDF1226741.3.25K1_Recruitment arrangementsfcf77f53-f0d5-4f82-a7b6-31b1f0eea1a114Recruitment arrangements (for publication)PDF2748551.02L1_ICF FSZ DLA RODZICOW ORAZ MAOLETNIEGO ktory ukonczy 13 r-z for publication04e2a19f-5420-4f75-a8dd-d63c3641ee3a15Subject information and informed consent form (for publication)PDF2748551.54L1_ICF FSZ DLA RODZICOW MAOLETNIEGO ponizej 13 r-z20b3089f-7701-44fb-b0e2-1939719b232f15Subject information and informed consent form (for publication)PDF2748551.54L1_ICF FSZ DLA DAWCY for publication910cc194-3b64-4c96-9118-0771da270fee15Subject information and informed consent form (for publication)PDF2748551.34L1_ICF PFI ponizej 13 r-z for publicationa76fb252-cf75-41fd-9270-3230e0d5c08015Subject information and informed consent form (for publication)PDF2748551.54L1_ICF serbski FSZ DLA DAWCY for publication7a6eab57-d126-4acc-9259-453dfd0c9a7615Subject information and informed consent form (for publication)PDF2748551.33L1_ICF serbski FSZ DLA RODZICOW MAOLETNIEGO ponizej 13 r-z for publication00636ed8-e649-499b-aaac-b9b3328bcf5a15Subject information and informed consent form (for publication)PDF2748551.53L1_ICF serbski FSZ DLA RODZICOW i MAOLETNIEGO ktory ukonczy 13 r-z for publicationd658ea4e-03ad-40e3-b5da-0bee7d5debef15Subject information and informed consent form (for publication)PDF2748551.53L1_ICF serbski PFI ponizej 13 r-z for publicationeee498c1-c4c5-42a7-a1a0-c8736b5220b415Subject information and informed consent form (for publication)PDF2748551.53L1_Recruitment advertisement for clinical trial participants v1_0 21Nov2022527001a0-6184-4691-a0dd-6f20274eb38215Subject information and informed consent form (for publication)PDF2748551.01EEA1 2024-515057-19-008EndedThe efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, placebo controlled studyNBK154/1/2020Duchenne muscular dystrophy; Cardiomyopathy; TachycardiaPoland:816/12/2024PL: 16/12/2024Diseases [C] - Cardiovascular Diseases [C14]Medical University Of GdanskEducational InstitutionTherapeutic confirmatory (Phase III)• Disease Free Survival (DFS) i.e., the time to develop cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method. • prevalence of patients with cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method • Disease Free Survival (DFS) i.e., the time to develop clinically evident Heart Failure (HF)PL1, Betaloc ZOK 100, 95 mg, tabletki o przedłużonym uwalnianiu, Betaloc ZOK 25, 23,75 mg, tabletki o przedłużonym uwalnianiu, PL218-64 years, 0-17 yearsFemale, Male1EEA144The primary endpoint is change in left ventricular ejection fraction (LVEF) by Teichholtz method (echocardiography), compared to baseline at Interim Analysis and Final Analysis.Yes16/12/202409/05/20262024-12-16T11:27:28.3892026-05-09T03:30:41.0299918548Protoko badania NBK154_1_2020592ab968-2aa9-4bde-8984-bb3879964358104Protocol (for publication)PDF612924.01CHPL Betaloc ZOK9ad431eb-cb85-47f1-a1e2-4b6bc2fafd5419Summary of Product Characteristics (SmPC) (for publication)PDF19286611Placeholder_advanced_MeDMDb30de6a0-39a2-4510-97ed-612a59b39ff914Recruitment arrangements (for publication)PDF15249311ICF 8-12ce2e0834-1dbc-471b-b5de-40066c27e8db15Subject information and informed consent form (for publication)PDF1524933.01ICF 13-179f2cead1-9152-4a1b-a9a4-9d80be70fc3715Subject information and informed consent form (for publication)PDF1524933.01ICF 1884b5f835-92fd-4251-8841-9675f1db817415Subject information and informed consent form (for publication)PDF1524932.01ICF Parents-Caretakersf2a237bc-4631-49eb-96e4-deb6b267b14915Subject information and informed consent form (for publication)PDF1524933.01Summary of results091d0b75-b36d-4c6d-9e86-f6cb5eeb7ce2103Summary of results (for publication)PDF606911Layperson Summary4d576842-0cf8-4e76-a6a0-88e6ffa3569f46Laypersons summary of results (for publication)PDF607011Poland325202024-12-16T11:27:28.3892024-12-16T11:27:28.389832520PolandSTART_OF_TRIAL2021-07-12START_OF_RECRUITMENT2021-08-19END_OF_RECRUITMENT2025-10-06EARLY_TERMINATION2025-10-061Sponsor DecisionTrue60692024-515057-19-00Summary of Results – NBK154/1/2020SubmittedSummary of ResultsFinalTrue1331372026-05-08T14:39:04.945842026-05-08T14:39:03.5552026-05-08T14:39:0415971SUM-13313760702024-515057-19-00Layperson Summary of Results – NBK154/1/2020SubmittedLaypersons Summary of ResultsFinalTrue1331382026-05-08T14:40:28.5849682026-05-08T14:40:27.3042026-05-08T14:40:2815971SUM-1331382024-515057-19-008The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, placebo controlled studyNBK154/1/202012/07/202106/10/2025Duchenne muscular dystrophy; Cardiomyopathy; TachycardiaPoland:816/12/2024PL: 16/12/2024Diseases [C] - Cardiovascular Diseases [C14]Medical University Of GdanskEducational InstitutionTherapeutic confirmatory (Phase III)• Disease Free Survival (DFS) i.e., the time to develop cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method. • prevalence of patients with cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method • Disease Free Survival (DFS) i.e., the time to develop clinically evident Heart Failure (HF)PL1, Betaloc ZOK 100, 95 mg, tabletki o przedłużonym uwalnianiu, Betaloc ZOK 25, 23,75 mg, tabletki o przedłużonym uwalnianiu, PL218-64 years, 0-17 yearsFemale, Male1144The primary endpoint is change in left ventricular ejection fraction (LVEF) by Teichholtz method (echocardiography), compared to baseline at Interim Analysis and Final Analysis.Yes16/12/202409/05/20262024-515057-19-00Ended2021-07-122025-10-062024-12-16T11:27:28.3892026-05-09T03:30:41.02999185486129202497141N/AN/AN/APL1N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePL1n/aN/A249713111185030TABLETKI O PRZEDŁUŻONYM UWALNIANIUPRD7378414R/73872Betaloc ZOK 100, 95 mg, tabletki o przedłużonym uwalnianiuPROLONGED-RELEASE TABLETMETOPROLOL TARTRATESUB03275MIGPLRECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.P.A.11185030369706RECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.P.A.ChemicalChemicalMETOPROLOL TARTRATESUB03275MIGC07AB02METOPROLOL5False1Falsemg/kg milligram(s)/kilogram2mg/kg milligram(s)/kilogram3600False603beta blockerPRD7378414PR211ORALTrueBetaloc ZOK 100, 95 mg, tabletki o przedłużonym uwalnianiumetoprolol tartratePROLONGED-RELEASE TABLET249712110473397TABLETKI O PRZEDŁUŻONYM UWALNIANIUPRD737841691682Betaloc ZOK 25, 23,75 mg, tabletki o przedłużonym uwalnianiuPROLONGED-RELEASE TABLETMETOPROLOL TARTRATESUB03275MIGPLRECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.P.A.10473397296142RECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.P.A.ChemicalChemicalMETOPROLOL TARTRATESUB03275MIGC07AB02METOPROLOL5False1Falsemg/kg milligram(s)/kilogram2mg/kg milligram(s)/kilogram3600False603Beta blockerPRD7378416PR111ORALTrueBetaloc ZOK 25, 23,75 mg, tabletki o przedłużonym uwalnianiumetoprolol tartratePROLONGED-RELEASE TABLET2497151N/AN/AN/APL2N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePL2n/aN/AThe efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, placebo controlled study4292168fff57b11-646a-4d0b-a33e-09985d356c36Skuteczność i bezpieczeństwo metoprololu jako leczenie uzupełniające standardową terapię w prewencji rozwoju kardiomiopatii u pacjentów z dystrofią mięśniową Duchenne’a w wieku 8-17 lat. Badanie randomizowane, podwójnie zaślepione, z równoległymi grupami i placebo w grupie kontrolnej19PolishMetoprolol Safety and Efficacy in the Prevention of Cardiomyopathy in Patients with Duchenne Muscular Dystrophy (DMD)42921713e5bbe13-b90b-40bc-bc18-e848b60bb18fBezpieczeństwo i skuteczność metoprololu w zapobieganiu kardiomiopatii u pacjentów z dystrofią mięśniową Duchenne'a (DMD).19PolishNBK154/1/2020289374NCT05066633False52Therapeutic confirmatory (Phase III)5327761328Duchenne muscular dystrophy; Cardiomyopathy; Tachycardia42921648de50b22-9c40-4703-b340-c1bc180942deDystrofia mięśniowa Duchenne'a; Kardiomiopatia; Tachykardia19PolishTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False3176584217658551765834176582The primary objective of this study is to evaluate whether metoprolol succinate in addition to standard of care cardiac treatment introduced before the onset of echocardiography detectable left ventricular dysfunction, can slow the rate of progression left ventricular ejection fraction drop that leads to cardiomyopathy in males with DMD compared to placebo.4292170954af164-cc71-4113-bb8b-73944316e128Głównym celem niniejszego badania jest ocena czy bursztynian metoprololu oprócz standardowego leczenia kardiologicznego wprowadzonego przed wystąpieniem dysfunkcji lewej komory serca wykrywalnej w badaniu echokardiograficznym, może opóźnić początek wystąpienia i / lub spowolnić tempo rozwoju kardiomiopatii u mężczyzn z DMD w porównaniu z placebo.19Polish1985841The secondary objectives of this study are to assess if metoprolol succinate in addition to standard of care treatment introduced before the onset of echocardiography detectable left ventricular dysfunction and compared to placebo: - can delay the occurrence of cardiomyopathy in children with DMD - can delay the occurrence of heart failure in children with DMD - can delay the age of onset and/or slow the rate of progression of myocardial fibrosis assessed by cardiac magnetic resonance (CMR) - can reduce the prevalence of sinus tachycardia assessed by Holter ECG - is safe and well tolerated in children with DMD42921723e7aa102-a0c9-46a1-96c2-2bdd5b37507dDrugorzędowymi celami niniejszego badania jest ocena czy bursztynian metoprololu oprócz standardowego leczenia wprowadzonego przed wystąpieniem rozpoznanej w echokardiografii dysfunkcji lewej komory i porównanie z placebo: - może opóźniać postęp niewydolności serca u dzieci z DMD i / lub zapobiegać rozwojowi niewydolności serca - może opóźnić początek wystąpienia i / lub spowolnić tempo postępu zwłóknienia mięśnia sercowego ocenianego metodą rezonansu magnetycznego serca - może zmniejszyć częstość częstoskurczu zatokowego i poprawić wskaźniki zmienności rytmu serca oceniane za pomocą EKG Holtera - jest bezpieczny i dobrze tolerowany przez dzieci z DMD19Polish38596111. Subject's parent(s) or legal guardian(s) has (have) provided written informed consent, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to requirements (>16 years old) 2. Stated willingness to comply with all study procedures and availability for the duration of the study 3. Male, ≥ 8 years and <17 years of age at time of enrolment in the study 4. Ability to take oral medication and be willing to adhere to the study intervention regimen 5. Subject has confirmed diagnosis of DMD, as defined as clinical picture consistent with typical DMD and: • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, or • Identifiable mutation within the DMD gene (deletion/duplication of one or more exons), where reading frame can be predicted as 'out-offrame' or • Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e., nonsense mutation, deletion/duplication leading to a downstream stop codon) 6. Taking ACEi treatment at minimum required doses as outlined in the protocol for at least 30 days4292169342fbec1-4ac2-44bc-8a08-0b5398405fc51. Rodzice lub opiekunowie prawni uczestnika badania wyrazili pisemną świadomą zgodę, w stosownych przypadkach, przed wykonaniem jakichkolwiek procedur związanych z badaniem; uczestnicy zostaną poproszeni o wyrażenie pisemnej lub ustnej zgody zgodnie z wymaganiami (> 16 lat). 2. Deklarowana gotowość do przestrzegania wszystkich procedur badawczych i dyspozycyjność na czas trwania badania. 3. Mężczyzna w wieku ≥ 8 lat i <17 lat w momencie włączenia do badania. 4. Zdolność do przyjmowania leków doustnych i chęć przestrzegania schematu interwencji w badaniu. 5. U pacjenta potwierdzono rozpoznanie DMD, zdefiniowanej jako obraz kliniczny zgodny z typową DMD oraz: • immunofluorescencja dystrofiny i / lub immunoblot wykazujący całkowity niedobór dystrofiny lub • możliwa do zidentyfikowania mutacja w genie DMD (delecja / duplikacja jednego lub więcej eksonów), gdzie ramka odczytu może być przewidziana jako „poza ramką” lub • pełne sekwencjonowanie genu dystrofiny pokazujące zmianę (mutacja punktowa, duplikacja, inne), która ma uniemożliwić produkcję białka dystrofiny (tj. Nonsensowna mutacja, delecja / duplikacja prowadząca do dalszego kodonu stop). 6. Przyjmowanie ACEi w minimalnych wymaganych dawkach, jak przedstawiono w Tabeli, przez co najmniej 30 dni.19Polish66530411. Current or previous permanent use of any beta-blocker medication 2. Treatment with another investigational drug or other intervention within 3 months prior to screening 3. Clinically significant bradycardia at rest or by Holter ECG, based on age and sex adjusted normal values, atrioventricular block higher than first degree at rest, or second degree Wenckebach at night, pauses longer than 2.5 seconds 4. Presence of pacemaker or ICD 5. Clinical signs or symptoms of heart failure 6. Left ventricular Ejection Fraction (LVEF) <57% (assessed by Teichholtz echocardiography) 7. Inability to obtain adequate quality echocardiography images (necessary to monitor for primary endpoint and safety) 8. Known allergic reactions to components of the IMPs429216736b69ad0-60b3-4c88-80de-ab1e03add72e3. Klinicznie istotna bradykardia w spoczynku lub w EKG Holtera, na podstawie wartości prawidłowych skorygowanych względem wieku i płci, blok przedsionkowo-komorowy wyższy niż pierwszego stopnia w spoczynku lub drugiego stopnia Wenckebacha w nocy, przerwy dłuższe niż 2,5 sekundy. 4. Obecność rozrusznika serca lub ICD. 5. Kliniczne oznaki lub objawy niewydolności serca *. 6. Frakcja wyrzutowa lewej komory * (LVEF) <57% (ocena za pomocą echokardiografii Teichholtza). 7. Brak możliwości uzyskania odpowiedniej jakości obrazów echokardiograficznych (konieczne do monitorowania pierwszorzędowego punktu końcowego i bezpieczeństwa). 8. Znane reakcje alergiczne na składniki IMP.19Polish4462351The primary endpoint is change in left ventricular ejection fraction (LVEF) by Teichholtz method (echocardiography), compared to baseline at Interim Analysis and Final Analysis.True4292165ce644d74-667d-4e90-b787-9fbd83c74d74Pierwszorzędowym punktem końcowym jest zmiana frakcji wyrzutowej lewej komory (LVEF) metodą Teichholtza (echokardiografia) w porównaniu z wartością wyjściową w analizie pośredniej i analizie końcowej.19Polish4462361• Disease Free Survival (DFS) i.e., the time to develop cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method. • prevalence of patients with cardiomyopathy defined as Left Ventricular Ejection Fraction – (LVEF) <55% by echocardiography Teichholtz method • Disease Free Survival (DFS) i.e., the time to develop clinically evident Heart Failure (HF)False4292166ecc45869-f488-4969-841b-0c37cdfe8847• przeżycie wolne od choroby (DFS), tj. czas do wystąpienia kardiomiopatii zdefiniowanej jako frakcja wyrzutowa lewej komory - (LVEF) <55% w echokardiografii metodą Teichholtza. • częstość występowania pacjentów z kardiomiopatią definiowaną jako frakcja wyrzutowa lewej komory - (LVEF) <55% w badaniu echokardiograficznym metodą Teichholtza • częstość występowania pacjentów ze zwłóknieniem mięśnia sercowego ocenianego metodą rezonansu magnetycznego serca (CMR)19Polish2026-03-312021-04-0143136Medical Research Agency (Agencja Badań Medycznych)16533433165333222PatientsTrueTrueTrue3acceptable2024-12-092024-12-0914Diseases [C] - Cardiovascular Diseases [C14]61328Duchenne muscular dystrophy; Cardiomyopathy; TachycardiaTrue65198True193944PublicDirector of Clinical Research Support Centrepiotr.widlak@gumed.edu.pl583492767504113Educational Institution1Medical University Of GdanskTrueTrueORG-100005122Active193945ScientificChief Medical Officer; Principal Investigatorjoannak@gumed.edu.pl0048583492882504113Educational Institution1Medical University Of GdanskTrueTrueORG-100005122Active504113Educational Institution1Medical University Of GdanskTrueTrueORG-100005122Active471737504113Educational Institution1Medical University Of GdanskTrueTrueORG-100005122Active505826Ul. Marii Sklodowskiej-Curie 3aUl. Marii Sklodowskiej-Curie 3aGdansk80-2102031PolandTrueORG-100005122FalseNon-Commercial2Therapeutic confirmatory (Phase III)67321614Diseases [C] - Cardiovascular Diseases [C14]192867Betaloc ZOK 100, 95 mg, tabletki o przedłużonym uwalnianiu1Test249713111185030TABLETKI O PRZEDŁUŻONYM UWALNIANIUPRD7378414R/73872Betaloc ZOK 100, 95 mg, tabletki o przedłużonym uwalnianiuPROLONGED-RELEASE TABLETMETOPROLOL TARTRATESUB03275MIGPLRECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.P.A.11185030369706RECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.P.A.ChemicalChemicalMETOPROLOL TARTRATESUB03275MIGC07AB02METOPROLOL5False1Falsemg/kg milligram(s)/kilogram2mg/kg milligram(s)/kilogram3600False603beta blockerPRD7378414PR211ORALTrueBetaloc ZOK 100, 95 mg, tabletki o przedłużonym uwalnianiumetoprolol tartratePROLONGED-RELEASE TABLET192866Betaloc ZOK 25, 23,75 mg, tabletki o przedłużonym uwalnianiu1Test249712110473397TABLETKI O PRZEDŁUŻONYM UWALNIANIUPRD737841691682Betaloc ZOK 25, 23,75 mg, tabletki o przedłużonym uwalnianiuPROLONGED-RELEASE TABLETMETOPROLOL TARTRATESUB03275MIGPLRECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.P.A.10473397296142RECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.P.A.ChemicalChemicalMETOPROLOL TARTRATESUB03275MIGC07AB02METOPROLOL5False1Falsemg/kg milligram(s)/kilogram2mg/kg milligram(s)/kilogram3600False603Beta blockerPRD7378416PR111ORALTrueBetaloc ZOK 25, 23,75 mg, tabletki o przedłużonym uwalnianiumetoprolol tartratePROLONGED-RELEASE TABLET192868PL13Placebo2497141N/AN/AN/APL1N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePL1n/aN/A192869PL23Placebo2497151N/AN/AN/APL2N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePL2n/aN/AFalse1524933252032520159712031Ended2024-10-302024-10-30TruePoland2024-12-16Ended463042021-07-122024-12-17463052021-07-122024-12-17614022021-08-192024-12-17614032021-08-192024-12-17984282021-08-192025-10-062026-01-20984292021-08-192025-10-062026-01-20True636412021-07-122024-12-172021-08-192025-10-06False5746332520Under evaluation2024-10-16T13:08:49.3827234332520Authorised2024-12-16T11:27:28.17925979132520Ended2026-01-20T10:35:34.8771Polandacceptable2024-12-09authorized2024-12-162024-12-16144991591629250667158Health care2Śląskie Centrum Chorób Serca w ZabrzuFalseFalseORL-000011655Active680751Marii Skłodowskiej-Curie 9Marii Skłodowskiej-Curie 9Zabrze341-8002031Poland+48323733600sek.kdz@sccs.plFalseORL-0000116551107245RolandFiszer+48323733600sek.kdz@sccs.pl2Oddział Wrodzonych Wad Serca i Kardiologii Dziecięcej991592629249667157Hospital/Clinic/Other health care facility8Uniwersyteckie Centrum KliniczneFalseTrueORG-100042500Active680750Ul. Debinki 7Ul. Debinki 7Gdansk80-2112031Poland583492882joannak@gumed.edu.plTrueORG-1000425001107246JoannaKwiatkowska583492882joannak@gumed.edu.pl2Klinika Kardiologii Dziecięcej i Wad Wrodzonych SercaAuthorised27568INITIALAuthorised2024-515057-19-00Ended2024-10-16acceptable2024-12-091524933252032520Poland2031acceptable2024-12-09authorized2024-12-16EndedPolandEnded2024-12-16Authorised2024-12-1632520PolandEndedINFalse511332756832520Poland2024-12-16T11:27:28.389authorizedacceptabledecision15249361292INITIALTrue9ad431eb-cb85-47f1-a1e2-4b6bc2fafd54192866Poland325202024-12-16T11:27:28.3892024-12-16T11:27:28.3898True2020-004901-2932520PolandSTART_OF_TRIAL2021-07-12START_OF_RECRUITMENT2021-08-19END_OF_RECRUITMENT2025-10-06EARLY_TERMINATION2025-10-061Sponsor DecisionTrue60692024-515057-19-00Summary of Results – NBK154/1/2020SubmittedSummary of ResultsFinalTrue1331372026-05-08T14:39:04.945842026-05-08T14:39:03.5552026-05-08T14:39:0415971SUM-13313760702024-515057-19-00Layperson Summary of Results – NBK154/1/2020SubmittedLaypersons Summary of ResultsFinalTrue1331382026-05-08T14:40:28.5849682026-05-08T14:40:27.3042026-05-08T14:40:2815971SUM-133138Protoko badania NBK154_1_2020592ab968-2aa9-4bde-8984-bb3879964358104Protocol (for publication)PDF612924.01CHPL Betaloc ZOK9ad431eb-cb85-47f1-a1e2-4b6bc2fafd5419Summary of Product Characteristics (SmPC) (for publication)PDF19286611Placeholder_advanced_MeDMDb30de6a0-39a2-4510-97ed-612a59b39ff914Recruitment arrangements (for publication)PDF15249311ICF 8-12ce2e0834-1dbc-471b-b5de-40066c27e8db15Subject information and informed consent form (for publication)PDF1524933.01ICF 13-179f2cead1-9152-4a1b-a9a4-9d80be70fc3715Subject information and informed consent form (for publication)PDF1524933.01ICF 1884b5f835-92fd-4251-8841-9675f1db817415Subject information and informed consent form (for publication)PDF1524932.01ICF Parents-Caretakersf2a237bc-4631-49eb-96e4-deb6b267b14915Subject information and informed consent form (for publication)PDF1524933.01Summary of results091d0b75-b36d-4c6d-9e86-f6cb5eeb7ce2103Summary of results (for publication)PDF606911Layperson Summary4d576842-0cf8-4e76-a6a0-88e6ffa3569f46Laypersons summary of results (for publication)PDF607011EEA1 2024-511492-15-005AuthorisedA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping4658-402Duchenne Muscular DystrophyItaly:5Denmark:5Czechia:5Norway:5Hungary:8Netherlands:8Spain:8France:5Germany:5Greece:8Slovenia:8Poland:5Romania:504/11/2024GR: 09/12/2024, DE: 04/11/2024, NL: 04/11/2024, NO: 05/11/2024, HU: 05/11/2024, DK: 04/11/2024, PL: 05/11/2024, ES: 04/11/2024, SI: 08/11/2024, FR: 13/11/2024, IT: 25/11/2024, CZ: 05/11/2024, RO: 06/11/2024Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Diseases [C] - Musculoskeletal Diseases [C05]Sarepta Therapeutics Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)• Change from Baseline at Week 144 in o Time to rise (TTRISE) from floor o 10-meter walk/run time o 6-minute walk test (6MWT) o Timed 4-step stair ascend test o Forced vital capacity percent predicted (FVC%p), • Time to Loss of Ambulation (LOA) through Week 144, • Change from Baseline at Week 24, Week 48, or Week 144 in skeletal muscle dystrophin expression by: o Western blot (quantitation) o Immunohistochemistry (IHC) fiber intensity by Immunofluorescence o Exon skipping quantitation by droplet digital polymerase chain reaction (ddPCR) o Percent dystrophin-positive fibers (PDPF) by Immunofluorescence, PK parameters (plasma and muscle biopsy), • Incidence of AEs • Incidence of AESIs • Incidence of SAEs • Safety laboratory assessments, • ECGs • ECHO • Abnormal changes from Baseline or worsening of vital signs and physical examination findingsETEPLIRSEN (AVI-4658)5460-17 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adm23.01Italy399982024-11-25T00:00:002026-01-27T11:10:31.1285Denmark397612024-11-04T14:17:12.172026-01-26T07:51:18.6655Czechia396102024-11-05T08:18:34.8852026-01-27T13:05:56.8845Norway397082024-11-05T08:55:33.3082026-01-28T16:08:03.0115Hungary396112024-11-05T11:57:22.8222025-07-07T15:31:00.2738Netherlands397412024-11-04T09:39:49.22026-02-02T00:00:008Spain397072024-11-04T10:06:01.7892026-02-02T15:49:10.0098France397622024-11-13T13:24:21.4182026-01-30T19:00:05.8385Germany399972024-11-04T13:15:30.1642026-01-28T10:00:35.715Greece397632024-12-09T17:46:05.5582025-05-13T11:47:31.6758Slovenia397642024-11-08T09:28:41.192026-01-30T09:02:43.0768Poland397652024-11-05T16:21:40.6382026-01-30T14:26:14.1975Romania397672024-11-06T13:30:25.4132026-03-24T09:37:20.831539610CzechiaSTART_OF_TRIAL2022-07-12START_OF_RECRUITMENT2022-08-23END_OF_RECRUITMENT2023-06-2839611HungarySTART_OF_TRIAL2022-10-05END_OF_TRIAL2025-10-08START_OF_RECRUITMENT2022-10-25END_OF_RECRUITMENT2022-11-0339707SpainSTART_OF_TRIAL2021-12-17END_OF_TRIAL2025-12-10START_OF_RECRUITMENT2022-08-29END_OF_RECRUITMENT2023-01-2639708NorwaySTART_OF_TRIAL2022-08-25START_OF_RECRUITMENT2023-08-28END_OF_RECRUITMENT2023-08-2839741NetherlandsSTART_OF_TRIAL2022-11-16END_OF_TRIAL2025-11-25START_OF_RECRUITMENT2023-01-19END_OF_RECRUITMENT2023-01-1939761DenmarkSTART_OF_TRIAL2022-10-24START_OF_RECRUITMENT2022-11-25END_OF_RECRUITMENT2023-06-2639762FranceSTART_OF_TRIAL2021-12-09START_OF_RECRUITMENT2022-06-22END_OF_RECRUITMENT2023-06-0539763GreeceSTART_OF_TRIAL2022-03-10END_OF_TRIAL2025-05-07START_OF_RECRUITMENT2022-05-31END_OF_RECRUITMENT2022-05-3139764SloveniaSTART_OF_TRIAL2022-10-10END_OF_TRIAL2026-01-19START_OF_RECRUITMENT2022-12-12END_OF_RECRUITMENT2022-12-1239765PolandSTART_OF_TRIAL2022-05-17START_OF_RECRUITMENT2022-08-08END_OF_RECRUITMENT2023-07-1139767RomaniaSTART_OF_TRIAL2022-12-13START_OF_RECRUITMENT2023-02-20END_OF_RECRUITMENT2023-09-0139997GermanySTART_OF_TRIAL2022-10-23START_OF_RECRUITMENT2023-05-05END_OF_RECRUITMENT2023-08-2339998ItalySTART_OF_TRIAL2023-07-12START_OF_RECRUITMENT2023-07-27END_OF_RECRUITMENT2023-08-032024-511492-15-005A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping4658-40209/12/2021Duchenne Muscular DystrophyItaly:5Denmark:5Czechia:5Norway:5Hungary:8Netherlands:8Spain:8France:5Germany:5Greece:8Slovenia:8Poland:5Romania:504/11/2024GR: 09/12/2024, DE: 04/11/2024, NL: 04/11/2024, NO: 05/11/2024, HU: 05/11/2024, DK: 04/11/2024, PL: 05/11/2024, ES: 04/11/2024, SI: 08/11/2024, FR: 13/11/2024, IT: 25/11/2024, CZ: 05/11/2024, RO: 06/11/2024Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Diseases [C] - Musculoskeletal Diseases [C05]Sarepta Therapeutics Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)• Change from Baseline at Week 144 in o Time to rise (TTRISE) from floor o 10-meter walk/run time o 6-minute walk test (6MWT) o Timed 4-step stair ascend test o Forced vital capacity percent predicted (FVC%p), • Time to Loss of Ambulation (LOA) through Week 144, • Change from Baseline at Week 24, Week 48, or Week 144 in skeletal muscle dystrophin expression by: o Western blot (quantitation) o Immunohistochemistry (IHC) fiber intensity by Immunofluorescence o Exon skipping quantitation by droplet digital polymerase chain reaction (ddPCR) o Percent dystrophin-positive fibers (PDPF) by Immunofluorescence, PK parameters (plasma and muscle biopsy), • Incidence of AEs • Incidence of AESIs • Incidence of SAEs • Safety laboratory assessments, • ECGs • ECHO • Abnormal changes from Baseline or worsening of vital signs and physical examination findingsETEPLIRSEN (AVI-4658)5460-17 yearsMale337Open-Label Dose Escalation: Incidence of AEs, Incidence of adverse events of special interest (AESIs), Abnormal changes from Baseline or worsening of vitals or physical examination findings, Incidence of SAEs, Safety laboratory assessments, Electrocardiograms (ECGs) and Echocardiograms (ECHO)., Double-Blind Dose Finding and Dose Comparison Part: -Change from Baseline at Week 72 or Week 96 in NSAA total score (for conditional efficacy interim analysis) -Change from Baseline at Week 144 in NSAA total score (for final analysis)No24/03/202625/03/20262024-511492-15-00Authorised2021-12-092024-11-04T09:39:49.22026-03-25T02:43:27.4851263295122488123100000000538Taiwan158TWTWNTrue100000000536Switzerland756CHCHETrue100000000434Jordan400JOJORTrue100000000482New Zealand554NZNZLTrue100000000465Mexico484MXMEXTrue100000000518Serbia688RSSRBTrue100000000556United Kingdom826GBGBRTrue100000000423India356ININDTrue100000000557United States840USUSATrue100000000439Korea, Republic of410KRKORTrue100000000549Turkey792TRTURTrue508040110281667SOLUTION FOR INFUSIONPRD94568671ETEPLIRSEN (AVI-4658)SOLUTION FOR INFUSIONAVI-4658ETEPLIRSENSUB129287ETEPLIRSENSAREPTA THERAPEUTICS INC10281667269047SAREPTA THERAPEUTICS INCNucleic AcidNucleic AcidETEPLIRSENSUB129287False1TrueEU/3/08/586mg/kg milligram(s)/kilogram0mg milligram(s)01142Chemical (Phosphorodiamidate morpholino oligomer for exon 51 skipping)PRD9456867AVI-4658UK MIA 2037711INTRAVENOUSFalseETEPLIRSEN (AVI-4658)eteplirsenSOLUTION FOR INFUSIONA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping10124226fcf362f8-7ae6-416a-9044-204c4cbab511Étude randomisée, en double aveugle, de détermination et de comparaison de doses, visant à évaluer l’innocuité et l’efficacité de doses élevées d’eteplirsen, précédées d’une phase d’augmentation de dose en ouvert, chez des patients atteints de dystrophie musculaire de Duchenne avec mutations par délétion, susceptibles de répondre au saut de l’exon 5110French10124223fcf362f8-7ae6-416a-9044-204c4cbab511Een gerandomiseerd, dubbelblind, dosisbepalend en -vergelijkend onderzoek naar de veiligheid en werkzaamheid van hoge doses Eteplirsen, voorafgegaan door een open-label dosisverhoging bij patiënten met Duchenne spierdystrofie met deletiemutaties, ontvankelijk voor exon 51 skipping37Dutch (Netherlands)10124227fcf362f8-7ae6-416a-9044-204c4cbab511Randomizált, kettős-vak, dóziskereső és dózis-összehasonlító vizsgálat az eteplirszen nagy adagjának biztonságosságának és hatásosságának értékelésére, amelyet nyílt elrendezésű dóziseszkaláció előz meg, Duchenne-féle izomdystrophiában szenvedő, az 51-es exon átugrásával kezelhető deléciós mutációval rendelkező betegeknél13Hungarian10124225fcf362f8-7ae6-416a-9044-204c4cbab511Estudio aleatorizado, doble ciego, de búsqueda de dosis y comparación de la seguridad y la eficacia de dosis altas de eteplirsén, precedida de un aumento de la dosis en régimen abierto, en pacientes con distrofia muscular de Duchenne con mutaciones de deleción susceptibles de omisión del exón 517Spanish10124224fcf362f8-7ae6-416a-9044-204c4cbab511"Μια Τυχαιοποιημένη, Διπλά Τυφλή Μελέτη Προσδιορισμού της Δόσης και Σύγκρισης της Ασφάλειας και Αποτελεσματικότητας Υψηλών Δόσεων του Eteplirsen, των οποίων Προηγείται μια Κλιμάκωση Δόσης Ανοικτής Επισήμανσης, σε Ασθενείς με Μυϊκή Δυστροφία Duchenne με Μεταλλάξεις Διαγραφής που είναι Αποδεκτές για Παράλειψη του Εξονίου 51"32Greek (Greece)10124228fcf362f8-7ae6-416a-9044-204c4cbab511Randomizowane badanie, prowadzone metodą podwójnie ślepej próby, pozwalające na ustalenie i porównanie dawek, oceniające skuteczność i bezpieczeństwo stosowania wysokich dawek Eteplirsenu, poprzedzone zwiększaniem dawki w otwartej próbie, u pacjentów z dystrofią mięśniową Duchenne’a z mutacjami typu delecje podatnymi na pominięcie egzonu 5119PolishA research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy101242445d8daa20-b6cb-4f68-a8c4-937d5cb6984aEstudio de investigación para comparar diferentes dosis de un nuevo medicamento en investigación para el tratamiento de determinados pacientes con distrofia muscular de Duchenne7Spanish101242455d8daa20-b6cb-4f68-a8c4-937d5cb6984aKutatási vizsgálat egy új vizsgálati készítmény különböző dózisainak összehasonlítására egyes Duchenne-izomdisztrófiás betegek kezelésére.13Hungarian101242425d8daa20-b6cb-4f68-a8c4-937d5cb6984aBadanie naukowe mające na celu porównanie różnych dawek nowego badanego produktu leczniczego w leczeniu niektórych pacjentów z dystrofią mięśniową Duchenne’a19Polish101242415d8daa20-b6cb-4f68-a8c4-937d5cb6984aΜία ερευνητική μελέτη για τη σύγκριση διαφορετικών δόσεων ενός νέου ερευνητικού φαρμακευτικού προϊόντος για τη θεραπεία ορισμένων ασθενών με μυϊκή δυστροφία Duchenne32Greek (Greece)101242435d8daa20-b6cb-4f68-a8c4-937d5cb6984aEen onderzoek voor het vergelijken van verschillende doseringen van een nieuw geneesmiddel voor de behandeling van bepaalde patiënten met Duchenne spierdystrofie37Dutch (Netherlands)101242465d8daa20-b6cb-4f68-a8c4-937d5cb6984aÉtude de recherche visant à comparer différentes doses d'un nouveau médicament expérimental pour le traitement de certains patients atteints de la dystrophie musculaire de Duchenne.34French (France)4658-402505193NCT03992430False52According to the "Appendix on transparency rules to the functional specifications of the EU clinical trials portal and database" chapter 4.3.3, Category 2 trials are safety and efficacy trials in patients. The current study meets this requirement, this is a Phase 3b study.112363127490Duchenne Muscular Dystrophy10124167230b573c-4a66-41e3-980a-3321b9056151Dystrophie Musculaire de Duchenne10French10124163230b573c-4a66-41e3-980a-3321b9056151Μυϊκή Δυστροφία Duchenne5Greek10124164230b573c-4a66-41e3-980a-3321b9056151Duchenne-féle izomdystrophia13Hungarian10124165230b573c-4a66-41e3-980a-3321b9056151Duchenne spierdystrofie18Dutch10124168230b573c-4a66-41e3-980a-3321b9056151Distrofia Muscular de Duchenne7Spanish10124166230b573c-4a66-41e3-980a-3321b9056151Dystrofią mięśniowa Duchenne’a19PolishTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False3371058737106013The open-label period of the study evaluates tolerability of higher dose eteplirsen in DMD patients371056537105993710616371057Open-Label Dose Escalation: Evaluate the safety and tolerability of weekly IV doses of 100 and 200 mg/kg of eteplirsen. Double-blind Dose Finding and Dose Comparison Part: • To investigate the effect of high doses of eteplirsen (100 mg/kg and 200 mg/kg) as compared with 30 mg/kg, administered weekly IV, on motor function in ambulant DMD patients with confirmed deletion genotypes amenable to Exon 51 skipping • To evaluate higher doses of eteplirsen (100mg/kg and 200 mg/kg) for dose comparison10124239d30d817c-a7f5-4a81-a5dc-53203a1949b3Augmentation de dose en ouvert Évaluer l’innocuité et la tolérabilité de doses IV hebdomadaires de 100 et 200 mg/kg d’eteplirsen Partie de détermination et comparaison de doses en double aveugle • Examiner l’effet de doses élevées d’eteplirsen (100 mg/kg et 200 mg/kg) par rapport à une dose de 30 mg/kg, administrée une fois par semaine par IV, sur la fonction motrice chez des patients ambulants atteints de dystrophie musculaire de Duchenne (DMD), présentant des génotypes avec délétion confirmée, susceptibles de répondre au saut de l’exon 51 • Évaluer des doses plus élevées d’eteplirsen (100 mg/kg et 200 mg/kg) pour la comparaison de doses34French (France)10124240d30d817c-a7f5-4a81-a5dc-53203a1949b3Aumento escalonado de la dosis en régimen abierto: • Evaluar la seguridad y la tolerabilidad de dosis IV semanales de 100 mg/kg y 200 mg/kg de eteplirsén. Parte en régimen doble ciego de búsqueda y comparación de la dosis: • Investigar el efecto de dosis altas de eteplirsén (100 mg/kg y 200 mg/kg) en comparación con 30 mg/kg, administrada semanalmente por vía IV, sobre la función motora en pacientes con distrofia muscular de Duchenne (DMD) ambulante con genotipos de deleción confirmados susceptibles de omisión del exón 51. • Evaluar dosis más altas de eteplirsén (100 mg/kg y 200 mg/kg) para comparar las dosis.7Spanish10124235d30d817c-a7f5-4a81-a5dc-53203a1949b3Nyílt elrendezésű dóziseszkaláció: Az eteplirszen heti 100 és 200 mg/kg-os iv. adagja biztonságosságának és tolerálhatóságának kiértékelése Kettős-vak dóziskereső és dózis-összehasonlító rész: • Az eteplirszen nagy adagjainak (100 mg/kg vagy 200 mg/kg) a motoros funkcióra kifejtett hatásának tanulmányozása a 30 mg/kg-os adaggal összehasonlítva, hetente egyszeri iv. adagolás mellett, Duchenne-féle izomdystrophiában (DMD) szenvedő, az 51-es exon átugrásával potenciálisan kezelhető delécióval igazoltan rendelkező betegeknél • Az eteplirszen nagyobb adagjainak (100 mg/kg és 200 mg/kg) dózis-összehasonító kiértékelése13Hungarian10124238d30d817c-a7f5-4a81-a5dc-53203a1949b3Eskalacja dawki prowadzona metodą otwartej próby: Ocena bezpieczeństwa i tolerancji cotygodniowych dawek dożylnych 100 mg/kg mc. i 200 mg/kg mc. eteplirsenu Część badania prowadzona metodą podwójnie ślepej próby mająca na celu ustalenie optymalnej dawki oraz porównanie dawek: •Zbadanie wpływu wysokich dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) w porównaniu z 30 mg/kg mc., podawanych co tydzień dożylnie, na funkcje motoryczne u chodzących pacjentów z dystrofią mięśniową Duchenne’a (DMD) z potwierdzonymi genotypami delecji podatnymi na pominięcie egzonu 51 • Ocena wysokich dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) w celu ich porównania19Polish10124237d30d817c-a7f5-4a81-a5dc-53203a1949b3Κλιμάκωσης δόσης ανοικτής επισήμανση: Αξιολόγηση της ασφάλειας και ανεκτικότητας των εβδομαδιαίων ΕΦ δόσεων 100 και 200 mg/kg του eteplirsen Διπλά τυφλό μέρος προσδιορισμού της δόσης και σύγκρισης της δόσης: •Διερεύνηση της επίδρασης υψηλών δόσεων του eteplirsen (100 mg/kg και 200 mg/kg) σε σύγκριση με 30 mg/kg, χορηγούμενης εβδομαδιαίως ΕΦ, στην κινητική λειτουργία σε περιπατητικούς ασθενείς με DMD με επιβεβαιωμένους γονότυπους διαγραφής που είναι αποδεκτοί για παράλειψη του Εξονίου 51 •Αξιολόγηση υψηλότερων δόσεων του eteplirsen (100 mg/kg και 200 mg/kg) για σύγκριση των δόσεων32Greek (Greece)10124236d30d817c-a7f5-4a81-a5dc-53203a1949b3Open-label dosisescalatie: Het beoordelen van de veiligheid en verdraagbaarheid van wekelijkse intraveneuze doses van 100 en 200 mg/kg eteplirsen. Dubbelblind deel voor dosisbepaling en dosisvergelijking: • Het onderzoeken van het effect van hoge doses eteplirsen (100 mg/kg en 200 mg/kg) in vergelijking met 30 mg/kg, wekelijks intraveneus toegediend, op de motorische functie bij ambulante patiënten met Duchenne spierdystrofie (DMD) met bevestigde deletiegenotypes die vatbaar zijn voor exon 51-skipping • Het beoordelen van hogere doses eteplirsen (100 mg/kg en 200 mg/kg) voor dosisvergelijking37Dutch (Netherlands)4316481Double-Blind Dose Finding and Dose Comparison Part : • To evaluate the effect of high doses as compared with 30 mg/kg of eteplirsen, administered weekly IV, on o Ambulatory performance o Pulmonary function o Dystrophin expression • To evaluate the PK and PD of doses (100mg/kg and 200mg/kg) higher than 30 mg/kg of eteplirsen • To evaluate safety and tolerability of doses (100mg/kg and 200mg/kg) higher than 30 mg/kg of eteplirsen administered weekly IV10124247bb3293b1-c950-424d-b0c5-68fff941e457Część badania prowadzona metodą podwójnie ślepej próby mająca na celu ustalenie optymalnej dawki oraz porównanie dawek: •Ocena wpływu wysokich dawek eteplirsenu w porównaniu z dawką 30 mg/kg mc., podawanej dożylnie raz na tydzień na: o sprawność poruszania się o czynność płuc o ekspresję dystrofiny •Ocena farmakokinetyki (PK) i farmakodynamiki (PD) dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) wyższych niż 30 mg/kg mc. •Ocena bezpieczeństwa i tolerancji dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) wyższych niż 30 mg/kg mc. podawanych dożylnie raz na tydzień19Polish10124248bb3293b1-c950-424d-b0c5-68fff941e457Parte en régimen doble ciego de búsqueda y comparación de la dosis: • Evaluar el efecto de dosis altas en comparación con 30 mg/kg de eteplirsén, administrada por vía IV una vez a la semana, sobre los siguientes parámetros: o Rendimiento ambulatorio o Función pulmonar o Expresión de distrofina • Evaluar la FC y la FD de dosis superiores a 30 mg/kg (100 mg/kg y 200 mg/kg) de eteplirsén Evaluar la seguridad y la tolerabilidad de dosis superiores a 30 mg/kg (100 mg/kg y 200 mg/kg) de eteplirsén administradas semanalmente por vía IV7Spanish10124249bb3293b1-c950-424d-b0c5-68fff941e457Partie de détermination et comparaison de doses en double aveugle • Évaluer l’effet de doses élevées par rapport à une dose IV hebdomadaire de 30 mg/kg d’eteplirsen sur les critères suivants: o Performances ambulatoires o Fonction pulmonaire o Expression de la dystrophine • Évaluer la PK et la PD de doses d’eteplirsen (100 mg/kg et 200 mg/kg)supérieures à 30 mg/kg • Évaluer l’innocuité et la tolérabilité de doses d’eteplirsen (100 mg/kg et 200 mg/kg) supérieures à 30 mg/kg administrées par IV une fois par semaine34French (France)10124250bb3293b1-c950-424d-b0c5-68fff941e457Kettős-vak dóziskereső és dózis-összehasonlító rész • Az eteplirszen heti egyszer iv. alkalmazott nagy adagja által a következőkre kifejtett hatás kiértékelése, a 30 mg/kg-os adaggal összehasonlítva: o járás-teljesítőképesség o légzésfunkció o disztrofinexpresszió • Az eteplirszen 30 mg/kg-nál nagyobb adagjai (100 mg/kg és 200 mg/kg) farmakokinetikájának és farmakodinamikájának elemzése. • Az eteplirszen 30 mg/kg-nál nagyobb (100 mg/kg vagy 200 mg/kg), heti egyszeri iv. adagjai biztonságosságának és tolerálhatóságának kiértékelése13Hungarian10124252bb3293b1-c950-424d-b0c5-68fff941e457Dubbelblind deel voor dosisbepaling en dosisvergelijking: • Het beoordelen van het effect van hoge doses in vergelijking met 30 mg/kg eteplirsen, wekelijks intraveneus toegediend, op o Ambulante prestaties o Longfunctie o Dystrofine-expressie • Het beoordelen van de farmacokinetiek en farmacodynamiek van doses (100 mg/kg en 200 mg/kg) hoger dan 30 mg/kg eteplirsen • Het beoordelen van de veiligheid en verdraagbaarheid van doses (100 mg/kg en 200 mg/kg) hoger dan 30 mg/kg eteplirsen, wekelijks intraveneus toegediend37Dutch (Netherlands)10124251bb3293b1-c950-424d-b0c5-68fff941e457Διπλά τυφλό μέρος προσδιορισμού της δόσης και σύγκρισης της δόσης: •Αξιολόγηση της επίδρασης των υψηλών δόσεων σε σύγκριση με 30 mg/kg eteplirsen, χορηγούμενου εβδομαδιαίως ΕΦ, ως προς τα εξής: oΠεριπατητική απόδοση oΠνευμονική λειτουργία oΈκφραση δυστροφίνης •Αξιολόγηση της ΦΚ και ΦΔ δόσεων (100 mg/kg και 200 mg/kg) του eteplirsen που υπερβαίνουν τα 30 mg/kg •Αξιολόγηση της ασφάλειας και της ανεκτικότητας δόσεων (100 mg/kg και 200 mg/kg) του eteplirsen που υπερβαίνουν τα 30 mg/kg, χορηγούμενων ΕΦ εβδομαδιαίως32Greek (Greece)82524411. Be a male with an established clinical diagnosis of DMD and an out-of- frame deletion mutation of the DMD gene amenable to exon 51 skipping (for example, deletions of exons 45-50, 47-50, 48-50, 49-50, 50, 52, and 52-63). 2. Be aged 4 to 13 years, inclusive 3. Ambulatory patient, able to perform TTRISE in 10 seconds or less at the time of screening visit. 4. Able to walk independently without assistive devices. 5. Has intact right and left biceps muscles (the preferred biopsy site) or an alternative upper arm muscle group that will allow for sufficiently sized (1 cm3) muscle biopsies to be obtained prior to and on treatment (for patients in the double-blind part of the study). 6. Has been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization, and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines [Birnkrant 2018, Kinnett 2017]) throughout the study). 7. For ages 7 years and older, has stable pulmonary function (forced vital capacity ≥50% of predicted and no requirement for nocturnal ventilation) that, in the Investigator's opinion, is unlikely to decompensate significantly over the duration of the study. For ages 4 to 6 years , does not require support from ventilator or non- invasive ventilation at time of screening. 8. If sexually active, agree to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose. The sexual partner must also use a medically acceptable form of contraceptive (ie, female oral contraceptives) during this timeframe. Acceptable methods of contraception include combined (estrogen and progesterone containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progesterone- only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intra-uterine hormone-releasing system; bilateral tubal occlusion; vasectomized partner; sexual abstinence (True abstinence: When this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence [such as calendar, ovulation, symptothermal, post-ovulation methods] and withdrawal are not acceptable methods of contraception.); or condom in combination with either cap, diaphragm, or sponge with spermicide (double-barrier contraception). 9. Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements. 10. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide informed consent for the patient to participate in the study.101242307a3047cd-bf3c-49ad-8e8a-4b442b5df1f01. Fiúknak kell lenniük, rendelkezniük kell a DMD klinikai diagnózisával és a DMD gén 51-es exon átugrásával kezelhető (például a 45-50, 47-50, 48-50, 49-50, 50, 52 és 52-63 exonok deléciója), olvasási kereten kívüli (out of frame) deléciós mutációjával; 2. 4 és 13 éves kor között kell lenniük 3. Járóképesnek kell lenniük, képesnek kell lenniük ≤10 másodperc alatt teljesíteni a talajról való felállás tesztet 4. Képes önállóan, segédeszközök nélkül járni. 5. Ép bicepsz vagy alternatív felkar izomcsoporttal kell rendelkezniük, amely lehetővé teszi 1 cm 3-es izombiopszia vételét a kezelés előtt és alatt (a vizsgálat kettős vak részében szereplő betegek esetében) 6. A randomizálást megelőzően legalább 12 hétig stabil dózisban vagy egyenértékű dózisban részesültek orális kortikoszteroid-kezelésben és az adag várhatóan állandó marad (kivéve a nemrégiben közzétett irányelvek [Birnkrant 2018, Kinnett 2017] szerinti, a testsúlyváltozáshoz és a stresszel kapcsolatos szükségletekhez való alkalmazkodáshoz szükséges módosításokat) a vizsgálat során). 7. 7 éves és idősebb korúak esetén stabil tüdőfunkcióval kell rendelkezniük (FVC ≥ a becsült érték 50%-ánál és nem igényelnek éjszakai lélegeztetést), ami a vizsgáló véleménye szerint nem valószínű, hogy jelentősen dekompenzálódik a vizsgálat időtartama alatt. A 4-6 éves korosztály esetében a szűrés időpontjában nem igényelnek légzéstámogatást lélegeztetőgép vagy non-invazív lélegeztetés formájában. 8. Ha szexuálisan aktív, vállalja, hogy a vizsgálat teljes időtartama alatt és az utolsó adagot követő 90 napig férfi óvszert használ. A szexuális partnernek is orvosilag elfogadható fogamzásgátlót (pl. női orális fogamzásgátlót) kell használnia ezen időszak alatt. Az elfogadható fogamzásgátló módszerek közé tartozik a kombinált (ösztrogén- és progeszterontartalmú) hormonális fogamzásgátlás, amely az ovuláció gátlásával jár (orális, intravaginális vagy transzdermális); csak progeszteront tartalmazó hormonális fogamzásgátlás, amely az ovuláció gátlásával jár (orális, injekciós vagy beültethető); méhen belüli eszköz; méhen belüli hormont felszabadító rendszer; kétoldali petevezeték-elzárás; vasektomizált partner; szexuális absztinencia (True abstinence: Ha ez összhangban van a beteg preferált és szokásos életmódjával. Az időszakos absztinencia [mint például a naptári, ovulációs, tüneti, ovuláció utáni módszerek] és a megvonás nem elfogadható fogamzásgátló módszerek); vagy óvszer kombinálva sapkával, pesszáriummal vagy spermiciddel ellátott szivaccsal (kettős gátas fogamzásgátlás). 9. Van(nak) szülő(k) vagy törvényes gyám(ok), aki(k) képes(ek) megérteni és betartani a vizsgálat összes követelményét. 10. Hajlandó tájékozott beleegyezést adni (ha alkalmazható), és rendelkezik (egy) szülővel (szülőkkel) vagy törvényes gyámmal (gyámokkal), aki (akik) hajlandó(ak) tájékozott beleegyezést adni a betegnek a vizsgálatban való részvételéhez.13Hungarian101242297a3047cd-bf3c-49ad-8e8a-4b442b5df1f01. Mężczyzna z ustalonym rozpoznaniem klinicznym dystrofii mięśniowej Duchenne’a (DMD) i mutacją delecyjną w genie DMD z zaburzeniem ramki odczytu (ang. out-of-frame), w przypadku której możliwe jest pominięcie egzonu 51 (na przykład delecje egzonów 45–50, 47–50, 48–50, 49–50, 50, 52 i 52–63). 2. Wiek od 4 do 13 lat włącznie. 3. Pacjent chodzący, zdolny do wykonania testu TTRISE (tj. czas do wstania) w czasie maksymalnie 10 sekund w momencie wizyty przesiewowej. 4. Zdolność samodzielnego chodzenia bez urządzeń wspomagających. 5. Bez zmian dotyczących prawego i lewego mięśnia dwugłowego (preferowane miejsce biopsji) lub alternatywnej grupy mięśni ramienia, co umożliwi pobranie biopsji mięśni o wystarczającej objętości (1 cm3) przed leczeniem i w jego trakcie (dotyczy pacjentów uczestniczących w części badania prowadzonej metodą podwójnie ślepej próby). 6. Przyjmowanie stałej dawki lub dawki równoważnej doustnych kortykosteroidów przez co najmniej 12 tygodni przed randomizacją, przy czym oczekuje się, że dawka pozostanie stała w trakcie całego badania (z wyjątkiem modyfikacji związanych ze zmianą masy ciała i potrzebami związanymi ze stresem, zgodnie z niedawno opublikowanymi wytycznymi [Birnkrant 2018, Kinnett 2017]). 7. Pacjenci w wieku 7 lat i starsi – stabilna czynność płuc (wymuszona pojemność życiowa ≥50% przewidywanej wartości należnej i brak konieczności wspomagania oddychania w nocy), z niewielkim prawdopodobieństwem istotnej dekompensacji w czasie trwania badania w opinii badacza. Pacjenci w wieku od 4 do 6 lat – brak konieczności wspomagania oddychania respiratorem ani stosowania wentylacji nieinwazyjnej w czasie badania przesiewowego. 8. Pacjenci aktywni seksualnie – zgoda na stosowanie prezerwatywy męskiej podczas takiej aktywności przez cały czas trwania badania oraz przez 90 dni od przyjęcia ostatniej dawki. W tym okresie partnerka seksualna musi również stosować medycznie akceptowalną metodę antykoncepcji (tj. doustne środki antykoncepcyjne dla kobiet). Dopuszczalne metody antykoncepcji obejmują złożoną (zawierającą estrogen i progesteron) antykoncepcję hormonalną związaną z hamowaniem owulacji (w postaci doustnej, dopochwowej lub przezskórnej), antykoncepcję hormonalną zawierającą sam progesteron związaną z hamowaniem owulacji (w postaci doustnej, we wstrzyknięciach lub w implantach), wkładkę wewnątrzmaciczną, system wewnątrzmaciczny uwalniający hormony, obustronne zamknięcie jajowodów, partnera po wazektomii, abstynencję seksualną (prawdziwa abstynencja: jeżeli jest zgodna z preferowanym i typowym stylem życia pacjenta. Okresowa abstynencja [taka jak metody kalendarzykowe, owulacyjne, objawowo-termiczne, poowulacyjne] i stosunek przerywany nie są akceptowalnymi metodami antykoncepcji) lub prezerwatywę w połączeniu z kapturkiem antykoncepcyjnym, diafragmą lub gąbką ze środkiem plemnikobójczym (podwójna metoda barierowa). 9. Dostępność rodzica/rodziców lub opiekuna prawnego/opiekunów prawnych, którzy są w stanie zrozumieć wszystkie wymogi związane z badaniem i ich przestrzegać. 10. Chęć wyrażenia świadomej zgody osoby małoletniej (jeśli ma to zastosowanie) i dostępność rodzica/rodziców lub opiekuna prawnego/opiekunów prawnych, którzy są skłonni wyrazić świadomą zgodę na udział pacjenta w badaniu.19Polish101242337a3047cd-bf3c-49ad-8e8a-4b442b5df1f01. Debe ser un varón, con un diagnóstico clínico establecido de DMD y una mutación por deleción fuera del marco del gen DMD susceptible de omisión del exón 51. 2. Tener ente 4 y 13 años, ambos incluidos. 3. Ser capaz de levantarse del suelo en ≤10 segundos o menos, en el momento de la visita de sección 4. Ser capaz de caminar de forma independiente sin dispositivos de ayuda. 5. Tener intactos los bíceps o un grupo muscular alternativo del brazo que permita obtener biopsias musculares de 1 cm3 antes y durante el tratamiento (en los pacientes de la parte doble ciego del estudio). 6. Haber recibido una dosis estable o una dosis equivalente de corticosteroides orales durante un mínimo de 12 semanas antes de la aleatorización; 7. Si tiene ≥7 años de edad, presentar una función pulmonar estable (CVF ≥50 % del valor teórico y sin necesidad de ventilación nocturna) que, en opinión del investigador, es improbable que se descompense significativamente durante el estudio y, en el caso de pacientes de 4 a 6 años de edad, no requerir apoyo con respirador ni ventilación no invasiva en el momento de la selección. 8. Si es sexualmente activo, aceptar el uso del preservativo masculino durante dicha actividad, durante toda la duración del estudio y durante 90 días después de la última dosis. La pareja sexual también debe usar una forma de anticoncepción médicamente aceptable (es decir, anticonceptivos orales femeninos) durante este período de tiempo. Los métodos anticonceptivos aceptables incluyen la anticoncepción hormonal combinada (que contiene estrógeno y progesterona) asociada con la inhibición de la ovulación (oral, intravaginal o transdérmica); anticoncepción hormonal solo con progesterona asociada con la inhibición de la ovulación (oral, inyectable o implantable); dispositivo intrauterino; sistema de liberación de hormonas intrauterinas; oclusión tubárica bilateral; pareja vasectomizada; abstinencia sexual (abstinencia verdadera: cuando esto está en línea con el estilo de vida preferido y habitual del paciente. La abstinencia periódica [como el calendario, la ovulación, los métodos sintotérmicos, los métodos postovulatorios] y el coito interrumpido no son métodos anticonceptivos aceptables). o condón en combinación con capuchón, diafragma o esponja con espermicida (anticoncepción de doble barrera). 9. Debe tener uno o más padres o tutores legales que pueden comprender y cumplir con todos los requisitos del estudio. 10. Debe estar dispuesto a brindar su consentimiento informado (si corresponde) y tener uno o más padres o tutores legales que estén dispuestos a brindar su consentimiento informado para que el paciente participe en el estudio.7Spanish101242327a3047cd-bf3c-49ad-8e8a-4b442b5df1f01. Να είναι άνδρας με τεκμηριωμένη κλινική διάγνωση DMD και μετάλλαξη διαγραφής εκτός πλαισίου του γονιδίου της DMD που είναι αποδεκτό για παράλειψη του Εξονίου 51 (για παράδειγμα, διαγραφές των Εξονίων 45-50, 47-50, 48-50, 49-50, 50, 52 και 52-63). 2. Να είναι ηλικίας 4 έως 13 ετών. 3. Να είναι περιπατητικός ασθενής, ικανός να πετύχει TTRISE σε 10 δευτερόλεπτα ή λιγότερο κατά την επίσκεψη διαλογής. 4. Να έχει δυνατότητα ανεξάρτητης βάδισης χωρίς βοηθητικές συσκευές. 5. Να έχει άθικτο δεξιό και αριστερό δικέφαλο μυ (το προτιμώμενο σημείο βιοψίας) ή μια εναλλακτική ομάδα μυών του άνω μέρους του βραχίονα που θα επιτρέψει τη λήψη μυϊκών βιοψιών επαρκούς μεγέθους (1 cm3) πριν και κατά τη διάρκεια της θεραπείας (για τους ασθενείς στο διπλά τυφλό μέρος της μελέτης). 6. Να λαμβάνει σταθερή δόση ή ισοδύναμη δόση από του στόματος κορτικοστεροειδών για τουλάχιστον 12 εβδομάδες πριν από την τυχαιοποίηση και η δόση αναμένεται να παραμείνει σταθερή (εκτός από τις τροποποιήσεις για την προσαρμογή στις αλλαγές του βάρους και στις ανάγκες που σχετίζονται με το στρες, σύμφωνα με τις πρόσφατα δημοσιευμένες κατευθυντήριες οδηγίες [Birnkrant 2018, Kinnett 2017]) καθ’ όλη τη διάρκεια της μελέτης. 7. Για ηλικίες 7 ετών και άνω, να έχει σταθερή πνευμονική λειτουργία (FVC ≥50% της προβλεπόμενης και καμία απαίτηση για νυκτερινό αερισμό) που, κατά τη γνώμη του Ερευνητή, δεν είναι πιθανό να παρουσιάσει σημαντική αντιρρόπηση κατά τη διάρκεια της μελέτης. Για ηλικίες 4 έως 6 ετών, να μην απαιτεί υποστήριξη από αναπνευστήρα ή μη επεμβατικό αερισμό κατά τη διαλογή. 8. Εάν είναι σεξουαλικά ενεργός, να συμφωνεί να χρησιμοποιεί ανδρικό προφυλακτικό κατά τη διάρκεια της σεξουαλικής δραστηριότητας καθ’ όλη τη διάρκεια της μελέτης και για 90 ημέρες μετά την τελευταία δόση. Η σεξουαλική σύντροφος πρέπει επίσης να χρησιμοποιεί μια ιατρικά αποδεκτή μορφή αντισύλληψης (δηλ. από του στόματος αντισυλληπτικά για γυναίκες) κατά τη διάρκεια αυτού του χρονικού πλαισίου. Οι αποδεκτές μέθοδοι αντισύλληψης περιλαμβάνουν συνδυαστική (περιέχει οιστρογόνα και προγεστερόνη) ορμονική αντισύλληψη που σχετίζεται με την αναστολή της ωορρηξίας (από του στόματος, ενδοκολπική ή διαδερμική), ορμονική αντισύλληψη μόνο με προγεστερόνη που σχετίζεται με αναστολή της ωορρηξίας (από του στόματος, ενέσιμη ή εμφυτεύσιμη), ενδομήτρια συσκευή, ενδομήτριο σύστημα απελευθέρωσης ορμονών, αμφοτερόπλευρη απολίνωση σαλπίγγων, σύντροφο που έχει υποβληθεί σε εκτομή σπερματικού πόρου, σεξουαλική αποχή (Πραγματική αποχή: όταν συνάδει με τον προτιμώμενο και συνήθη τρόπο ζωής του ασθενούς. Η περιοδική αποχή [όπως η ημερολογιακή μέθοδος, η μέθοδος ωορρηξίας, η συμπτωθερμική μέθοδος, η μέθοδος μετά την ωορρηξία] και η διακεκομμένη συνουσία δεν είναι αποδεκτές μέθοδοι αντισύλληψης) ή προφυλακτικό σε συνδυασμό με τραχηλική καλύπτρα, διάφραγμα ή σπόγγο με σπερματοκτόνο (αντισύλληψη διπλού φραγμού). 9. Να έχει γονείς ή νόμιμους κηδεμόνες που είναι σε θέση να κατανοήσουν και να συμμορφωθούν με όλες τις απαιτήσεις της μελέτης. 10. Να είναι πρόθυμος να παράσχει συναίνεση ανηλίκου μετά από ενημέρωση (εάν ισχύει) και να έχει γονείς ή νόμιμους κηδεμόνες που είναι πρόθυμοι να παράσχουν συναίνεση μετά από ενημέρωση για τη συμμετοχή του ασθενούς στη μελέτη.32Greek (Greece)101242317a3047cd-bf3c-49ad-8e8a-4b442b5df1f01. Mannelijke patiënt met een vastgestelde klinische diagnose van DMD en een out-of-frame-deletiemutatie van het DMD-gen die vatbaar is voor exon 51-skipping (bijvoorbeeld deleties van exons 45-50, 47-50, 48-50, 49-50, 50, 52 en 52-63). 2. Van 4 tot en met 13 jaar oud 3. Ambulante patiënt, in staat om TTRISE in 10 seconden of minder uit te voeren op het moment van het screeningbezoek. 4. Kan zelfstandig lopen zonder hulpmiddelen. 5. Heeft intacte linker- en rechterbicepsspieren (de voorkeurslocatie voor een biopsie) of een alternatieve spiergroep in de bovenarm die het mogelijk maakt om voldoende grote (1 cm3) spierbiopsieën te verkrijgen vóór en tijdens de behandeling (voor patiënten in het dubbelblinde deel van het onderzoek). 6. Heeft gedurende ten minste 12 weken voorafgaand aan de randomisatie een stabiele dosis, of een dosisequivalent, orale corticosteroïden gebruikt en de dosis zal naar verwachting gedurende het hele onderzoek constant blijven (met uitzondering van aanpassingen om tegemoet te komen aan veranderingen in gewicht en aan stressgerelateerde behoeften volgens de onlangs gepubliceerde richtlijnen [Birnkrant 2018, Kinnett 2017]). 7. Voor kinderen vanaf 7 jaar: heeft een stabiele longfunctie (geforceerde vitale longcapaciteit ≥ 50% van de voorspelde waarde en geen noodzaak voor nachtelijke beademing) die, naar de mening van de onderzoeker, waarschijnlijk niet significant zal decompenseren tijdens het onderzoek. Voor kinderen van 4 tot 6 jaar: heeft geen ondersteuning van beademingsapparatuur of niet-invasieve beademing nodig op het moment van screening. 8. Indien seksueel actief, stemt ermee in om tijdens een dergelijke activiteit een mannencondoom te gebruiken tijdens het hele onderzoek en 90 dagen na de laatste dosis. De seksuele partner moet ook een medisch aanvaardbare anticonceptiemethode gebruiken (d.w.z. vrouwelijke orale anticonceptiva) in deze periode. Aanvaardbare anticonceptiemethoden omvatten gecombineerde (oestrogeen en progesteron-bevattende) hormonale anticonceptie gericht op remming van de ovulatie (oraal, intravaginaal of transdermaal); alleen progesteron-bevattende hormonale anticonceptie gericht op remming van de ovulatie (oraal, injecteerbaar of implanteerbaar); spiraaltje; hormoonspiraaltje; bilaterale tubale occlusie; partner die een vasectomie heeft ondergaan; seksuele onthouding (volledige onthouding: als dit in lijn is met de voorkeurs- en gebruikelijke levensstijl van de patiënt. Periodieke onthouding [zoals kalender-, ovulatie-, symptothermale, post-ovulatiemethoden] en terugtrekking zijn geen aanvaardbare anticonceptiemethoden.); of een condoom in combinatie met een cervixkapje, pessarium of sponsje met zaaddodend middel (dubbele-barrière-anticonceptie). 9. Heeft (een) ouder(s) of wettelijke voogd(en) die in staat is (zijn) om alle onderzoeksvereisten te begrijpen en eraan te voldoen. 10. Is bereid geïnformeerde instemming te geven (indien van toepassing) en heeft (een) ouder(s) of wettelijke voogd(en) die bereid is (zijn) schriftelijke geïnformeerde toestemming te geven voor de patiënt om aan het onderzoek mee te doen.37Dutch (Netherlands)101242347a3047cd-bf3c-49ad-8e8a-4b442b5df1f01. Être un garçon avec un diagnostic clinique établi de DMD et une mutation par délétion hors cadre du gène DMD, susceptible de répondre au saut de l'exon 51 (par exemple, délétions des exons 45 50, 47 50, 48 50, 49 50, 50, 52 et 52 63). 2.Être âgé de 4 à 13 ans inclus. 3. Patient ambulatoire, capable d'effectuer le TTRISE en 10 secondes ou moins au moment de la visite de sélection. 4. Capable de marcher de façon indépendante, sans dispositifs d’assistance. 5. Présente un biceps ou autre groupe musculaire alternatif intact au niveau du bras supérieur, qui permette l’obtention de biopsies musculaires de 1 cm3 avant et pendant le traitement (pour les patients enrôlés dans la partie en double aveugle de l’étude). 6. Avoir suivi un traitement à dose stable ou dose équivalente de corticoïdes oraux pendant au moins 12 semaines avant la randomisation et la dose devrait rester constante (à l'exception des modifications pour tenir compte des changements de poids et des besoins liés au stress, conformément aux lignes directrices récemment publiées [Birnkrant 2018, Kinnett 2017]) tout au long de l'étude. 7. Pour les plus de 7 ans, avoir une fonction pulmonaire stable (CVF ≥ 50 % de la valeur prédite et aucun recours à la ventilation nocturne) qui, selon l’investigateur, est peu susceptible de décompenser significativement pendant la durée de l’étude ; et pour les âges de 4 à 6 ans, ne pas avoir besoin de l’assistance d’un respirateur ou d’une ventilation non invasive au moment de la sélection. 8. S'il est sexuellement actif, il accepte d'utiliser un préservatif masculin pendant toute la durée de l'étude et pendant 90 jours après la dernière dose. La partenaire sexuelle doit également utiliser une forme de contraception médicalement acceptable (c'est-à-dire des contraceptifs oraux féminins) pendant cette période. Les méthodes de contraception acceptables sont les suivantes : contraception hormonale combinée (contenant des œstrogènes et de la progestérone) associée à une inhibition de l'ovulation (par voie orale, intravaginale ou transdermique) ; contraception hormonale à base de progestérone uniquement associée à une inhibition de l'ovulation (par voie orale, injectable ou implantable) ; dispositif intra-utérin ; système intra-utérin de libération d'hormones ; occlusion tubaire bilatérale ; partenaire vasectomisé ; abstinence sexuelle. (Véritable abstinence : Lorsqu'elle correspond au mode de vie préféré et habituel de la patiente. L'abstinence périodique [telle que les méthodes calendaires, d'ovulation, symptothermiques, post-ovulatoires] et le retrait ne sont pas des méthodes de contraception acceptables) ; ou le préservatif en combinaison avec une cape, un diaphragme ou une éponge avec spermicide (contraception à double barrière). 9. A un (des) parent(s) ou un (des) tuteur(s) légal(aux) qui est (sont) capable(s) de comprendre et de se conformer à toutes les exigences de l'étude. 10.Est disposé à donner son consentement éclairé (le cas échéant) et a un (des) parent(s) ou un (des) tuteur(s) légal(aux) qui est (sont) disposé(s) à donner son (leur) consentement éclairé pour que le patient participe à l'étude.34French (France)141196111. Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization that may have an effect on muscle strength or function. Growth hormone for short stature and testosterone for delayed puberty are permitted if physician has documented the diagnosis and medical necessity of treatment and if the patient has been on a stable dose for at least 24 weeks prior to randomization. 2. Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid administered at least 12 weeks prior to first dose. Drisapersen administered at least 36 weeks prior to first dose. Suvodirsen administered at least 12 weeks prior to first dose. Vamorolone administered at least 12 weeks prior to first dose. Eteplirsen (previous or current use) Tamoxifen administered at least 4 weeks prior to first dose. 3. Major surgery within 3 months prior to randomization or planned surgery for any time during this study, except for allowed protocol- specified surgery, as applicable. 4. Presence of any significant neuromuscular or genetic disease other than DMD (eg, dwarfism). 5. Gamma-glutamyl transpeptidase (GGT) > 3 × the upper limit of normal (ULN) or serum bilirubin > ULN unexplained by Gilbert's Syndrome. 6. Any known impairment of renal function (eg, estimated glomerular filtration rate [eGFR] ≤ 60 mL/min as assessed by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] cystatin C based equation [Inker, 2012, Filler 2012]), or dipstick protein result +2, or persistent and unexplained dipstick protein result +1 7. Platelet count < the lower limit of normal. 8. Presence of other clinically significant illness including significant cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease or malignancy. 9. Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction <50% on the screening ECHO or the Fridericia's correction formula (QTcF) ≥450 milliseconds based on the screening ECGs. 10. Prior or ongoing medical condition that could, in the Investigator's opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results. 11. Known hypersensitivity to eteplirsen or any excipients of eteplirsen. 12. Is, in the Investigator's opinion, unable or unwilling to comply with the study procedures.101242175f2989e9-d92b-4a4e-a7cc-73a708a40b671. Gebruik van een farmacologische behandeling (anders dan corticosteroïden) binnen 12 weken voorafgaand aan randomisatie die een effect kan hebben op spierkracht of -functie. Groeihormoon voor korte gestalte en testosteron voor vertraagde puberteit zijn toegestaan als de arts de diagnose en medische noodzaak van de behandeling heeft gedocumenteerd en als de patiënt gedurende ten minste 24 weken voorafgaand aan randomisatie een stabiele dosis heeft gekregen. 2. Huidige of eerdere behandeling met een andere experimentele farmacologische behandeling voor DMD of eerdere blootstelling aan antisense-oligonucleotide, gentherapie of genbewerking, behalve de volgende: Ezutromid toegediend ten minste 12 weken voorafgaand aan de eerste dosis. Drisapersen toegediend ten minste 36 weken voorafgaand aan de eerste dosis. Suvodirsen toegediend ten minste 12 weken voorafgaand aan de eerste dosis. Vamorolon toegediend ten minste 12 weken voorafgaand aan de eerste dosis. Eteplirsen (eerder of huidig gebruik). Tamoxifen toegediend ten minste 4 weken voorafgaand aan de eerste dosis. 3. Zware operatie binnen 3 maanden voorafgaand aan randomisatie of een geplande operatie op enig moment tijdens dit onderzoek, met uitzondering van toegestane, in het protocol gespecificeerde operaties, indien van toepassing. 4. Aanwezigheid van een andere significante neuromusculaire of genetische ziekte anders dan DMD (bijv. dwerggroei). 5. Gamma-glutamyltranspeptidase (GGT) > 3 × de bovengrens van normaal (ULN) of serumbilirubine > ULN, niet te verklaren door het syndroom van Gilbert. 6. Een bekende nierfunctiestoornis (bijv. geschatte glomerulaire filtratiesnelheid [eGFR] ≤ 60 ml/min, zoals beoordeeld door de op cystatine C-gebaseerde vergelijking van de Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] [Inker, 2012, Filler 2012]) of dipstick-eiwitresultaat +2 of aanhoudend en onverklaard dipstick-eiwitresultaat +1 7. Bloedplaatjestelling < de ondergrens van normaal. 8. Aanwezigheid van andere klinisch significante ziekten, waaronder significante hart-, long-, lever-, nier-, hematologische, immunologische of gedragsziekten of maligniteit. 9. Heeft aanwijzingen voor cardiomyopathie, zoals gedefinieerd door een linkerventrikelejectiefractie van < 50% op het echocardiogram (ECHO) bij screening of de correctieformule van Fridericia (QTcF) ≥ 450 milliseconden, gebaseerd op de elektrocardiogrammen (ECG’s) bij screening. 10. Eerdere of aanhoudende medische aandoeningen die, naar de mening van de onderzoeker, de veiligheid van de patiënt negatief kunnen beïnvloeden, het onwaarschijnlijk maken dat de behandeling wordt voltooid of de beoordeling van de onderzoeksresultaten kunnen verstoren. 11. Bekende overgevoeligheid voor eteplirsen of voor een van de hulpstoffen van eteplirsen. 12. Is naar het oordeel van de onderzoeker niet in staat of niet bereid om zich aan de onderzoeksprocedures te houden.18Dutch101242185f2989e9-d92b-4a4e-a7cc-73a708a40b671. Bármilyen farmakológiai kezelés (a kortikoszteroidok kivételével) alkalmazása a randomizálást megelőző 12 héten belül, amely hatással lehet az izomerőre vagy az izomfunkcióra. Növekedési hormon alacsony termetre és tesztoszteron késleltetett pubertás esetén engedélyezett, ha az orvos dokumentálta a diagnózist és a kezelés orvosi szükségességét, és ha a beteg a randomizálás előtt legalább 24 hétig stabil dózist kapott. 2. Jelenlegi vagy korábbi kezelés bármely más kísérleti farmakológiai kezeléssel a DMD kezelésére, vagy bármilyen korábbi expozíció antisense oligonukleotiddal, génterápiával vagy génszerkesztéssel; kivéve a következőket: Az első adagot megelőzően legalább 12 héttel beadott Ezutromid. Drisapersen, amelyet legalább 36 héttel az első adag előtt adtak be Suvodirsen, amelyet legalább 12 héttel az első adag előtt adtak be. Vamorolon, amelyet legalább 12 héttel az első adag előtt adtak be. Eteplirsen (korábbi vagy jelenlegi alkalmazás). 3. Nagyobb műtét a randomizálást megelőző 3 hónapon belül, vagy a vizsgálat során bármikor tervezett műtét, kivéve a protokoll által meghatározott, engedélyezett műtétet, ha alkalmazható. 4. Bármilyen jelentős neuromuszkuláris vagy genetikai betegség jelenléte a DMD-n kívül (pl. törpeség). 5. Gamma-glutamil-transzpeptidáz (GGT) > 3 × a normálérték felső határa (ULN) vagy szérum bilirubin > ULN, amelyet nem magyaráz meg Gilbert-szindróma. 6. A vesefunkció bármilyen ismert károsodása (pl. a Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] cystatin C alapú egyenlete [Inker, 2012, Filler 2012] alapján becsült glomeruláris filtrációs ráta [eGFR] ≤ 60 ml/perc), vagy mérőszelvény fehérjeeredménye +2, vagy tartós és megmagyarázhatatlan mérőszelvény fehérjeeredménye +1 7. Trombocitaszám < a normálérték alsó határa. 8. Egyéb klinikailag jelentős betegség jelenléte, beleértve a jelentős szív-, tüdő-, máj-, vese-, hematológiai, immunológiai vagy viselkedési betegséget vagy malignitást. 9. Kardiomiopátiára utaló jelek, amelyet a szűrő EKG alapján a bal kamrai ejekciós frakció <50% vagy a Fridericia korrekciós képlet (QTcF) ≥450 milliszekundum alapján a szűrő EKG-ken meghatározott. 10. Korábbi vagy folyamatban lévő egészségügyi állapot, amely a vizsgáló véleménye szerint hátrányosan befolyásolhatja a beteg biztonságát, valószínűtlenné teheti a kezelés befejezését, vagy ronthatja a vizsgálati eredmények értékelését. 11. Ismert túlérzékenység az eteplirszennel vagy az eteplirszén bármely segédanyagával szemben. 12. A vizsgáló véleménye szerint nem képes vagy nem hajlandó megfelelni a vizsgálati eljárásoknak.13Hungarian101242225f2989e9-d92b-4a4e-a7cc-73a708a40b671. Stosowanie w ciągu 12 tygodni przed randomizacją jakiegokolwiek leczenia farmakologicznego (innego niż kortykosteroidy), które może mieć wpływ na siłę lub czynność mięśni. Dopuszczalne jest stosowanie hormonu wzrostu w przypadku niskiego wzrostu oraz testosteronu w przypadku opóźnionego dojrzewania, jeśli lekarz udokumentował rozpoznanie oraz medyczną konieczność leczenia i jeśli pacjent przyjmował stałą dawkę przez co najmniej 24 tygodnie przed randomizacją. 2. Aktualne lub wcześniejsze leczenie DMD z zastosowaniem jakiejkolwiek innej eksperymentalnej terapii farmakologicznej lub jakakolwiek wcześniejsza ekspozycja na oligonukleotyd antysensowny, terapię genową lub edycję genów; wyjątek stanowią następujące leki: Ezutromid podawany co najmniej 12 tygodni przed pierwszą dawką. Drisapersen podawany co najmniej 36 tygodni przed pierwszą dawką. Suvodirsen podawany co najmniej 12 tygodni przed pierwszą dawką. Vamorolone podawany co najmniej 12 tygodni przed pierwszą dawką. Eteplirsen (stosowanie w przeszłości lub obecnie). Tamoksyfen podawany co najmniej 4 tygodnie przed pierwszą dawką. 3. Poważny zabieg chirurgiczny w ciągu 3 miesięcy przed randomizacją lub planowany zabieg chirurgiczny w dowolnym momencie w trakcie tego badania, z wyjątkiem dozwolonych w protokole zabiegów chirurgicznych, jeśli dotyczy. 4. Występowanie jakiejkolwiek istotnej choroby nerwowo-mięśniowej lub genetycznej innej niż DMD (np. karłowatości). 5. Aktywność gamma-glutamylotranspeptydazy (GGTP) > 3 × górnej granicy normy (GGN) lub stężenie bilirubiny w surowicy > GGN, którego nie można wyjaśnić występowaniem zespołu Gilberta. 6. Wszelkie znane zaburzenia czynności nerek (np. szacunkowy wskaźnik filtracji kłębuszkowej [eGFR] ≤ 60 ml/min, obliczony na podstawie wzoru Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI], opartego na stężeniu cystatyny C [Inker, 2012, Filler 2012]) lub wynik testu paskowego na obecność białka wynoszący +2 bądź utrzymujący się i niewyjaśniony wynik testu paskowego na obecność białka wynoszący +1. 7. Liczba płytek krwi < dolnej granicy normy. 8. Występowanie innej, klinicznie istotnej choroby, w tym istotnej choroby serca, płuc, wątroby, nerek, choroby hematologicznej, immunologicznej lub behawioralnej bądź nowotworu złośliwego. 9. Cechy kardiomiopatii, zdefiniowanej jako frakcja wyrzutowa lewej komory wynosząca <50% w przesiewowym badaniu echokardiograficznym (ECHO) lub określonej na podstawie skorygowanego wzoru Fridericii (QTcF) ≥450 milisekund w przesiewowym badaniu elektrokardiograficznym (EKG). 10. Przebyte lub utrzymujące się aktualnie schorzenie, które w opinii badacza mogłoby niekorzystnie wpłynąć na bezpieczeństwo pacjenta, sprawiać, że prawdopodobieństwo ukończenia leczenia byłoby niewielkie lub zakłócać ocenę wyników badania. 11. Stwierdzona nadwrażliwość na eteplirsen lub którąkolwiek substancję pomocniczą eteplirsenu. 12. Brak możliwości lub chęci przestrzegania przez pacjenta procedur badania w opinii badacza.19Polish101242195f2989e9-d92b-4a4e-a7cc-73a708a40b671.Χρήση οποιασδήποτε φαρμακολογικής θεραπείας (εκτός από τα κορτικοστεροειδή) εντός 12 εβδομάδων πριν από την τυχαιοποίηση που μπορεί να έχει επίδραση στη μυϊκή δύναμη ή λειτουργία. Η αυξητική ορμόνη για το βραχύ ανάστημα και η τεστοστερόνη για την καθυστερημένη εφηβεία επιτρέπονται εάν ένας ιατρός έχει τεκμηριώσει τη διάγνωση και την ιατρική αναγκαιότητα της θεραπείας και εάν ο ασθενής λάμβανε σταθερή δόση για τουλάχιστον 24 εβδομάδες πριν από την τυχαιοποίηση. 2.Τρέχουσα ή προηγούμενη θεραπεία με οποιαδήποτε άλλη πειραματική φαρμακολογική θεραπεία για DMD ή οποιαδήποτε προηγούμενη έκθεση σε αντινοηματικό ολιγονουκλεοτίδιο, γονιδιακή θεραπεία ή επεξεργασία γονιδίων, εκτός από τα ακόλουθα: •Ezutromid χορηγούμενο τουλάχιστον 12 εβδομάδες πριν από την πρώτη δόση. •Drisapersen χορηγούμενο τουλάχιστον 36 εβδομάδες πριν από την πρώτη δόση. •Suvodirsen χορηγούμενο τουλάχιστον 12 εβδομάδες πριν από την πρώτη δόση. •Vamorolone χορηγούμενο τουλάχιστον 12 εβδομάδες πριν από την πρώτη δόση. •Eteplirsen (προηγούμενη ή τρέχουσα χρήση). •Ταμοξιφαίνη χορηγούμενη τουλάχιστον 4 εβδομάδες πριν από την πρώτη δόση. 3.Μείζονα χειρουργική επέμβαση εντός 3 μηνών πριν από την τυχαιοποίηση ή προγραμματισμένη χειρουργική επέμβαση οποιαδήποτε στιγμή κατά τη διάρκεια αυτής της μελέτης, εκτός από την επιτρεπόμενη χειρουργική επέμβαση που ορίζεται από το πρωτόκολλο, κατά περίπτωση. 4.Παρουσία οποιασδήποτε σημαντικής νευρομυϊκής ή γενετικής νόσου εκτός της DMD (π.χ. νανισμός). 5.γ-γλουταμυλ-τρανσπεπτιδάση [GGT] >3 × το ανώτατο φυσιολογικό όριο (ULN) ή χολερυθρίνη ορού >ULN η οποία δεν εξηγείται από το σύνδρομο Gilbert. 6.Οποιαδήποτε γνωστή έκπτωση της νεφρικής λειτουργίας (π.χ. εκτιμώμενος ρυθμός σπειραματικής διήθησης [eGFR] ≤60 mL/min, όπως αξιολογείται από την εξίσωση της Συνεργασίας Επιδημιολογίας Χρόνιας Νεφρικής Νόσου [CKD-EPI] βάσει κυστατίνης C [Inker 2012, Filler 2012]) ή αποτέλεσμα πρωτεΐνης +2 με ταινία ούρων (dipstick) ή επίμονο και ανεξήγητο αποτέλεσμα πρωτεΐνης +1 με ταινία ούρων (dipstick). 7.Αριθμός αιμοπεταλίων < το κατώτερο φυσιολογικό όριο. 8.Παρουσία άλλης κλινικά σημαντικής νόσου, συμπεριλαμβανομένης σημαντικής καρδιακής, πνευμονικής, ηπατικής, νεφρικής, αιματολογικής, ανοσολογικής ή συμπεριφορικής νόσου ή κακοήθειας. 9.Έχει ενδείξεις καρδιομυοπάθειας, όπως ορίζεται από κλάσμα εξώθησης αριστερής κοιλίας <50% στο ECHO διαλογής ή στη διόρθωση κατά Fridericia (QTcF) ≥450 χιλιοστά του δευτερολέπτου με βάση τα ΗΚΓ διαλογής. 10.Προηγούμενη ή συνεχιζόμενη ιατρική πάθηση που θα μπορούσε, κατά τη γνώμη του Ερευνητή, να επηρεάσει δυσμενώς την ασφάλεια του ασθενούς, να καταστήσει απίθανο να ολοκληρωθεί η πορεία της θεραπείας ή να επηρεάσει την αξιολόγηση των αποτελεσμάτων της μελέτης. 11.Γνωστή υπερευαισθησία στο eteplirsen ή σε κάποιο από τα έκδοχα του eteplirsen. 12.Είναι, κατά τη γνώμη του Ερευνητή, ανίκανος ή απρόθυμος να συμμορφωθεί με τις διαδικασίες της μελέτης.5Greek101242215f2989e9-d92b-4a4e-a7cc-73a708a40b671. Utilisation de tout traitement pharmacologique (autre que les corticostéroïdes) dans les 12 semaines précédant la randomisation, susceptible d'avoir un effet sur la force ou la fonction musculaire. L'hormone de croissance pour la petite taille et la testostérone pour le retard de puberté sont autorisées si un médecin a documenté le diagnostic et la nécessité médicale du traitement et si le patient a pris une dose stable pendant au moins 24 semaines avant la randomisation. 2.Traitement actuel ou antérieur par tout autre traitement pharmacologique expérimental de la DMD ou toute exposition antérieure à un oligonucléotide antisens, à la thérapie génique ou à l'édition de gènes, à l'exception des cas suivants : • Ezutromid administré au moins 12 semaines avant la première dose. • Drisapersen administré au moins 36 semaines avant la première dose. • Suvodirsen administré au moins 12 semaines avant la première dose. • Vamorolone administré au moins 12 semaines avant la première dose. • Eteplirsen (utilisation antérieure ou actuelle). • Tamoxifène administré au moins 4 semaines avant la première dose. 3. Chirurgie majeure dans les 3 mois précédant la randomisation ou chirurgie planifiée à tout moment au cours de cette étude, à l'exception de la chirurgie autorisée spécifiée par le protocole, le cas échéant. 4.Présence d'une maladie neuromusculaire ou génétique importante autre que la DMD (par exemple, nanisme). 5.Gamma-glutamyl transpeptidase [GGT] > 3 fois la limite supérieure de la normale (LSN) ou bilirubine sérique > LSN inexpliquée par le syndrome de Gilbert. 6.Toute altération connue de la fonction rénale (par exemple, débit de filtration glomérulaire estimé [DFGe] ≤ 60 ml/min, tel qu'évalué par l'équation basée sur la cystatine C de la Chronic Kidney Disease Epidemiology Collaboration [CKD EPI] [Inker 2012, Filler 2012]), ou résultat protéique à la bandelette +2, ou résultat protéique à la bandelette +1 persistant et inexpliqué. 7.Numération plaquettaire < la limite inférieure de la normale 8.Présence d'une autre maladie cliniquement significative, y compris une maladie cardiaque, pulmonaire, hépatique, rénale, hématologique, immunologique, comportementale ou une tumeur maligne. 9.Présence d'une cardiomyopathie, définie par une fraction d'éjection du ventricule gauche < 50 % sur l'ECHO de sélection ou une formule de correction de Fridericia (QTcF) ≥ 450 millisecondes sur la base des ECG de sélection. 10.État de santé antérieur ou actuel qui, de l'avis de l'investigateur, pourrait nuire à la sécurité du patient, rendre improbable l'achèvement du traitement ou compromettre l'évaluation des résultats de l'étude. 11.Hypersensibilité connue à l'eteplirsen ou à l'un des excipients de l'eteplirsen. 12.Est, de l'avis de l'investigateur, incapable ou réticent à se conformer aux procédures de l'étude. Les patients qui ne remplissent pas les critères d'éligibilité peuvent faire l'objet d'une nouvelle sélection à la discrétion de l'investigateur.10French101242205f2989e9-d92b-4a4e-a7cc-73a708a40b671. Uso de cualquier tratamiento farmacológico (excepto corticosteroides) dentro de las 12 semanas previas a la aleatorización que pueda tener un efecto sobre la fuerza o función muscular. Se permite la hormona del crecimiento para la baja estatura y la testosterona para la pubertad tardía si el médico ha documentado el diagnóstico y la necesidad médica del tratamiento y si el paciente ha recibido una dosis estable durante al menos 24 semanas antes de la aleatorización. 2. Tratamiento actual o previo con cualquier otro tratamiento farmacológico experimental para la DMD o cualquier exposición previa a oligonucleótidos antisentido, terapia génica o edición génica; excepto los siguientes: Ezutromid administrado al menos 12 semanas antes de la primera dosis. Drisapersen administrado al menos 36 semanas antes de la primera dosis Suvodirsen administrado al menos 12 semanas antes de la primera dosis. Vamorolone administrado al menos 12 semanas antes de la primera dosis. Eteplirsen (uso previo o actual) Tamoxifeno administrado al menos 4 semanas antes de la primera dosis. 3. Cirugía mayor dentro de los 3 meses previos a la aleatorización o cirugía planificada para cualquier momento durante este estudio, excepto la cirugía permitida especificada en el protocolo, según corresponda. 4. Presencia de cualquier enfermedad neuromuscular o genética significativa distinta de la DMD (p. ej., enanismo). 5. Gamma-glutamil transpeptidasa (GGT) > 3 × el límite superior de lo normal (LSN) o bilirrubina sérica > LSN no explicada por el síndrome de Gilbert. 6. Cualquier deterioro conocido de la función renal (p. ej., tasa de filtración glomerular estimada [TFGe] ≤ 60 ml/min según la ecuación basada en cistatina C de la Colaboración de Epidemiología de la Enfermedad Renal Crónica [CKD-EPI] [Inker, 2012, Filler 2012]), o resultado de proteína en tira reactiva +2, o resultado de proteína en tira reactiva persistente e inexplicable +1 7. Recuento de plaquetas < el límite inferior de lo normal. 8. Presencia de otra enfermedad clínicamente significativa, incluidas enfermedades cardíacas, pulmonares, hepáticas, renales, hematológicas, inmunológicas o conductuales significativas o neoplasias malignas. 9. Tiene evidencia de miocardiopatía, definida por una fracción de eyección del ventrículo izquierdo <50 % en el ecocardiograma de detección o la fórmula de corrección de Fridericia (QTcF) ≥450 milisegundos según los ECG de detección. 10. Condición médica previa o actual que podría, en opinión del investigador, afectar negativamente la seguridad del paciente, hacer improbable que se complete el tratamiento o perjudicar la evaluación de los resultados del estudio. 11. Hipersensibilidad conocida al eteplirsen o a cualquiera de sus excipientes. 12. Es, en opinión del investigador, incapaz o no está dispuesto a cumplir con los procedimientos del estudio.7Spanish9668501Open-Label Dose Escalation: Incidence of AEs, Incidence of adverse events of special interest (AESIs), Abnormal changes from Baseline or worsening of vitals or physical examination findings, Incidence of SAEs, Safety laboratory assessments, Electrocardiograms (ECGs) and Echocardiograms (ECHO).True1012417393f6e994-fe81-42e0-b4c1-4b9f2ac1a18eEskalacja dawki prowadzona metodą otwartej próby: Częstość występowania zdarzeń niepożądanych (AE), Częstość występowania zdarzeń niepożądanych o szczególnym znaczeniu (AESI), Nieprawidłowe zmiany w stosunku do punktu wyjściowego lub pogorszenie parametrów życiowych lub wyników badania fizykalnego, Częstość występowania ciężkich zdarzeń niepożądanych (SAE), Oceny laboratoryjne dotyczące bezpieczeństwa, Badania elektrokardiograficzne (EKG), Echokardiogram (echo serca)19Polish1012416993f6e994-fe81-42e0-b4c1-4b9f2ac1a18eAumento escalonado de la dosis en régimen abierto: Incidencia de EA, Incidencia de eventos adversos de interés especial (AESI) Cambios anormales respecto del valor inicial o empeoramiento de los signos vitales o hallazgos del examen físico, Incidencia de EAG, Evaluaciones de seguridad de laboratorio, Electrocardiogramas (ECG) y Ecocardiogramas (ECO).7Spanish1012417193f6e994-fe81-42e0-b4c1-4b9f2ac1a18eNyílt elrendezésű dóziseszkaláció: A nemkívánatos események (AE-k) gyakorisága, a különös figyelmet érdemlő nemkívánatos események (AESI-k) incidenciája, a kóros eltérések a kiinduláshoz képest, illetve az élettani paraméterek vagy a fizikális vizsgálati leletek rosszabbodása, a súlyos nemkívánatos események (serious adverse events, SAE-k) incidenciája, biztonságossági laboratóriumi vizsgálatok, elektrokardiográfiás vizsgálatok (EKG-k), szív-ultrahangvizsgálatok (echokardiográfia).13Hungarian1012417093f6e994-fe81-42e0-b4c1-4b9f2ac1a18eOpen-label dosisverhoging: Incidentie van bijwerkingen (AE’s), incidentie van bijwerkingen van bijzonder belang (AESI’s), abnormale veranderingen ten opzichte van baseline of verslechtering van de vitale functies of bevindingen bij lichamelijk onderzoek, incidentie van ernstige bijwerkingen (SAE’s), laboratoriumbeoordelingen voor de veiligheid, elektrocardiogrammen (ECG’s) en echocardiogrammen (ECHO).18Dutch1012417493f6e994-fe81-42e0-b4c1-4b9f2ac1a18eAugmentation de dose en ouvert Incidence des événements indésirables (EI), Incidence des événements indésirables d’intérêt spécifique (EIIS), Évolutions anormales par rapport aux valeurs de référence ou aggravation des signes vitaux et des résultats de l’examen physique, Incidence des événements indésirables graves (EIG), Analyses de laboratoire de sécurité, Électrocardiogrammes (ECG), Échocardiogrammes (ECHO).10French1012417293f6e994-fe81-42e0-b4c1-4b9f2ac1a18eΜέρος κλιμάκωσης δόσης ανοικτής επισήμανσης Επίπτωση των ΑΣ, Επίπτωση ανεπιθύμητων συμβάντων ειδικού ενδιαφέροντος (AESI), Παθολογικές μεταβολές από την Έναρξη ή επιδείνωση ζωτικών σημείων ή ευρημάτων κλινικής εξέτασης, Επίπτωση των ΣΑΣ, Εργαστηριακές αξιολογήσεις της ασφάλειας, Ηλεκτροκαρδιογραφήματα (ΗΚΓ), Ηχοκαρδιογραφήματα (ECHO)5Greek9668512Double-Blind Dose Finding and Dose Comparison Part: -Change from Baseline at Week 72 or Week 96 in NSAA total score (for conditional efficacy interim analysis) -Change from Baseline at Week 144 in NSAA total score (for final analysis)True10124179f9258092-d9e7-4cca-878e-484c98ffba06Dubbelblind deel voor dosisbepaling en dosisvergelijking: -Verandering ten opzichte van baseline in week 72 of week 96 in de North Star Ambulatory Assessment (NSAA)-totaalscore (voor voorwaardelijke tussentijdse werkzaamheidsanalyse) -Verandering ten opzichte van baseline in week 144 in de NSAA-totaalscore (voor uiteindelijke analyse)18Dutch10124176f9258092-d9e7-4cca-878e-484c98ffba06Διπλά τυφλό μέρος προσδιορισμού της δόσης και σύγκρισης της δόσης: •Μεταβολή από την Έναρξη την Εβδομάδα 72 ή την Εβδομάδα 96 στη συνολική βαθμολογία NSAA (για ενδιάμεση ανάλυση αποτελεσματικότητας υπό όρους) •Μεταβολή από την Έναρξη την Εβδομάδα 144 στη συνολική βαθμολογία5Greek10124180f9258092-d9e7-4cca-878e-484c98ffba06Kettős-vak dóziskereső és dózis-összehasonlító rész: -A North Star járásvizsgálat (North Star Ambulatory Assessment, NSAA) összpontszámának változása a kiinduláshoz képest a 72. héten és a 96. héten (feltételes időközi hatásossági elemzéshez) -Az NSAA összpontszámának változása a kiindulás és a 144. hét között (a végleges elemzéshez)13Hungarian10124177f9258092-d9e7-4cca-878e-484c98ffba06Część badania prowadzona metodą podwójnie ślepej próby mająca na celu ustalenie optymalnej dawki oraz porównanie dawek: - Zmiana wartości całkowitego wyniku w skali North Star Ambulatory Assessment (NSAA) od punktu wyjściowego w 72. tygodniu lub 96. tygodniu (do warunkowej, śródokresowej analizy skuteczności) - Zmiana wartości całkowitego wyniku w skali NSAA od punktu wyjściowego w 144. tygodniu (do analizy końcowej)19Polish10124175f9258092-d9e7-4cca-878e-484c98ffba06Partie de détermination et comparaison de doses en double aveugle • Évolution par rapport aux valeurs de référence à la semaine 72 ou à la semaine 96 du score global sur l’échelle d’évaluation North Star Ambulatory Assessment (NSAA) (pour l’analyse intermédiaire conditionnelle de l’efficacité) • Évolution par rapport aux valeurs de référence à la semaine 144 du score global NSAA (pour analyse finale)10French10124178f9258092-d9e7-4cca-878e-484c98ffba06Parte en régimen doble ciego de búsqueda y comparación de la dosis: - Cambio con respecto al valor inicial en la semana 72 o la semana 96 en la puntuación total de la NSAA (para el análisis provisional de eficacia condicional) -Cambio con respecto al valor inicial en la semana 144 en la puntuación total de la NSAA (para el análisis final)7Spanish9668521• Change from Baseline at Week 144 in o Time to rise (TTRISE) from floor o 10-meter walk/run time o 6-minute walk test (6MWT) o Timed 4-step stair ascend test o Forced vital capacity percent predicted (FVC%p)False10124183ca98c0a4-b59d-49c1-9463-d2f8b563a29d• Cambio con respecto al valor inicial en la semana 144 en o Tiempo para levantarse (TTRISE) desde el piso o Tiempo para caminar/correr 10 metros o Prueba de caminata de 6 minutos (6MWT) o Prueba de ascenso de escaleras cronometrada de 4 pasos o Porcentaje de capacidad vital forzada prevista (FVC%p)7Spanish10124181ca98c0a4-b59d-49c1-9463-d2f8b563a29d• Verandering ten opzichte van baseline in week 144 in o Tijd om op te staan (TTRISE) van de vloer o 10-meter-loop-/rentijd o 6-minuten-looptest (6MWT) o Getimede 4-treden-traptest o Voorspeld percentage geforceerde vitale longcapaciteit (FVC%p)"18Dutch10124185ca98c0a4-b59d-49c1-9463-d2f8b563a29dÉvolution observée à la semaine 144 par rapport aux valeurs de référence: o Temps pour se relever du sol o Chronométrage de la marche/course sur 10 mètres o Test de marche de 6 minutes(6MWT) o Test chronométré de montée d’escalier sur 4 marches o Taux de déclin annuel de la capacité vitale forcée (CVF) (%) prédite10French10124186ca98c0a4-b59d-49c1-9463-d2f8b563a29dVáltozás a kiindulási értékhez képest a 144. héten: o a talajról történő felálláshoz szükséges idő (TTRISE) o 10 méteres járás/futás ideje o 6 perces járásvizsgálat (6MWT) o 4 lépcsőn fellépdelés időméréses vizsgálata o forszírozott vitálkapacitás, a becsült érték százalékában (FVC%p)13Hungarian10124184ca98c0a4-b59d-49c1-9463-d2f8b563a29d• Zmiana w 144. tygodniu w stosunku do punktu wyjściowego w zakresie poniższych wartości: o Czas potrzebny do wstania (TTRISE) z podłogi o Test 10-metrowego marszu/biegu o Test 6-minutowego marszu (6MWT) o Test wchodzenia po schodach (4 stopnie) z pomiarem czasu o badania procentowej przewidywanej natężonej pojemności życiowej (FVC%p)19Polish10124182ca98c0a4-b59d-49c1-9463-d2f8b563a29d•Μεταβολή από την Έναρξη την Εβδομάδα 144, ως προς τα εξής: oTTRISE από το δάπεδο oΧρόνος κάλυψης απόστασης 10 μέτρων με περπάτημα/τρέξιμο o6MWT oΧρονομετρημένη δοκιμασία ανάβασης 4 σκαλοπατιών oΠροβλεπόμενο ποσοστό βίαιης ζωτικής χωρητικότητας (FVC%p)"5Greek9668532• Time to Loss of Ambulation (LOA) through Week 144False101241894d982e92-5019-49d9-83f4-afcc87f20b29Durée jusqu’à la perte de la marche jusqu’à la semaine 14410French101241904d982e92-5019-49d9-83f4-afcc87f20b29• Tijd tot verlies van loopvermogen (LOA) tot week 14418Dutch101241914d982e92-5019-49d9-83f4-afcc87f20b29• Tiempo transcurrido hasta la pérdida de la deambulación (LOA) hasta la semana 1447Spanish101241874d982e92-5019-49d9-83f4-afcc87f20b29• Czas do utraty zdolności poruszania się (LOA) do tygodnia 144.19Polish101241884d982e92-5019-49d9-83f4-afcc87f20b29A járóképesség elvesztéséig eltelt idő (LOA) a 144. hétig13Hungarian101241924d982e92-5019-49d9-83f4-afcc87f20b29•Χρόνος έως την LOA έως την Εβδομάδα 1445Greek9668543• Change from Baseline at Week 24, Week 48, or Week 144 in skeletal muscle dystrophin expression by: o Western blot (quantitation) o Immunohistochemistry (IHC) fiber intensity by Immunofluorescence o Exon skipping quantitation by droplet digital polymerase chain reaction (ddPCR) o Percent dystrophin-positive fibers (PDPF) by ImmunofluorescenceFalse10124194729283fd-ea51-4eff-83b1-d30d067a8bfcA vázizmok disztrofinexpressziójának változása a kiindulás és a 24., 48. vagy 144. héten, a következő vizsgálatok alapján: o Western blot (mennyiségi meghatározás) o immunhisztokémia (IHC) – rostintenzitás immunfluoreszcenciával o az exonátugrás mennyiségi meghatározása ún. droplet digitális polimeráz láncreakcióval (ddPCR) o A disztrofin-pozitív rostok százalékos aránya (PDPF) immunfluoreszcenciával13Hungarian10124196729283fd-ea51-4eff-83b1-d30d067a8bfc•Μεταβολή από την Έναρξη την Εβδομάδα 24, την Εβδομάδα 48 ή την Εβδομάδα 144 στην έκφραση της δυστροφίνης των σκελετικών μυών μετρημένη με: oΜέθοδο ανοσοαποτύπωσης Western (ποσοτικοποίηση) oΈνταση ινών IHC μέσω δοκιμασίας ανοσοφθορισμού oΠοσοτικοποίηση παράλειψης εξονίων μέσω ddPCR oPDPF μέσω δοκιμασίας ανοσοφθορισμού5Greek10124197729283fd-ea51-4eff-83b1-d30d067a8bfcÉvolution par rapport aux valeurs de référence à la semaine 24, la semaine 48 ou la semaine 144 dans l’expression musculo-squelettique de la dystrophine, selon les méthodes suivantes: o Western blot (quantification) o Immunohistochimie (IHC) (intensité des fibres par immunofluorescence) o Quantification du saut d’exon par réaction en chaîne par polymérase (PCR) o Pourcentage de fibres positives à la dystrophine (PDPF) par immunofluorescence10French10124193729283fd-ea51-4eff-83b1-d30d067a8bfc• Verandering ten opzichte van baseline in week 24, week 48 of week 144 in de expressie van skeletspierdystrofine door: o Western blot (kwantificering) o Immunohistochemie (IHC)-vezelintensiteit door immunofluorescentie o Kwantificering van exon-skipping door middel van droplet digital-polymerasekettingreactie (ddPCR) o Percentage dystrofine-positieve vezels (PDPF) door immunofluorescentie18Dutch10124195729283fd-ea51-4eff-83b1-d30d067a8bfc• Variación entre el momento basal y la semana 24, la semana 48 o la semana 144 de la expresión de distrofina en el músculo esquelético mediante: o Inmunoelectrotransferencia (cuantificación) o Intensidad de las fibras mediante inmunohistoquímica (IHQ) mediante inmunofluorescencia o Cuantificación del salto de exones mediante reacción en cadena de la polimerasa digital en nanogotas (ddPCR) o Porcentaje de fibras positivas para distrofina (PDPF) por inmunofluorescencia7Spanish10124198729283fd-ea51-4eff-83b1-d30d067a8bfc• Zmiana w stosunku do punktu wyjściowego w tygodniu 24., 48. lub 144. w ekspresji dystrofiny w mięśniach szkieletowych na podstawie oznaczenia: o Western blot (oznaczenie ilościowe) o intensywności włókien w badaniu immunohistochemicznym (IHC) w teście immunofluorescencyjnym o Oznaczenie ilościowe z pominięciem egzonu metodą emulsyjnej reakcji łańcuchowej polimerazy (ddPCR) o Odsetek włókien dystrofino-dodatnich (PDPF) w teście immunofluorescencyjnym19Polish9668554PK parameters (plasma and muscle biopsy)False10124200905c40ec-75d7-4984-89df-cd34aca38540• Farmakokinetikai (PK) paraméterek (plazma- és izombiopsziával)13Hungarian10124204905c40ec-75d7-4984-89df-cd34aca38540•Παράμετροι ΦΚ (πλάσματος και με μυϊκή βιοψία)5Greek10124199905c40ec-75d7-4984-89df-cd34aca38540Paramètres PK (plasma et biopsie musculaire)10French10124203905c40ec-75d7-4984-89df-cd34aca38540• Parámetros FC (en plasma y biopsia de músculo)7Spanish10124202905c40ec-75d7-4984-89df-cd34aca38540PK-parameters (plasma- en spierbiopsie)18Dutch10124201905c40ec-75d7-4984-89df-cd34aca38540• Parametry PK (osocze i biopsja tkanki mięśniowej)19Polish9668565• Incidence of AEs • Incidence of AESIs • Incidence of SAEs • Safety laboratory assessmentsFalse10124208e8d5ce8d-375c-4838-98c4-fa444e213cfa•Επίπτωση των ΑΣ •Επίπτωση των AESI •Επίπτωση των ΣΑΣ •Εργαστηριακές αξιολογήσεις της ασφάλειας5Greek10124207e8d5ce8d-375c-4838-98c4-fa444e213cfa• Incidencia de AA • Incidencia de AAIE • Incidencia de AAG • Evaluaciones analíticas de seguridad7Spanish10124209e8d5ce8d-375c-4838-98c4-fa444e213cfa• AE-k előfordulása • Az AESI-k előfordulása • SAE-k előfordulása • Biztonsági laboratóriumi értékelések13Hungarian10124206e8d5ce8d-375c-4838-98c4-fa444e213cfa•Incidence des EI • Incidence des EIIS • Incidence des EIG • Analyses de laboratoire de sécurité10French10124205e8d5ce8d-375c-4838-98c4-fa444e213cfa• Częstość występowania zdarzeń niepożądanych • Częstość występowania zdarzeń niepożądanych o szczególnym znaczeniu (AESI) • Częstość występowania ciężkich zdarzeń niepożądanych (SAE) • Oceny laboratoryjne dotyczące bezpieczeństwa19Polish10124210e8d5ce8d-375c-4838-98c4-fa444e213cfa• Incidentie van AE’s • Incidentie van AESI’s • Incidentie van SAE’s • Laboratoriumbeoordelingen voor de veiligheid18Dutch9668576• ECGs • ECHO • Abnormal changes from Baseline or worsening of vital signs and physical examination findingsFalse101242117b0ecf78-8c4b-4012-b063-876b928eb522• ECG • Ecocardiograma • Variaciones anormales con respecto al inicio o empeoramiento de los hallazgos de las constantes vitales y la exploración física7Spanish101242157b0ecf78-8c4b-4012-b063-876b928eb522•ΗΚΓ •ECHO •Παθολογικές μεταβολές από την Έναρξη ή επιδείνωση ζωτικών σημείων και ευρημάτων κλινικής εξέτασης5Greek101242147b0ecf78-8c4b-4012-b063-876b928eb522• ECG • ECHO • Évolutions anormales par rapport aux valeurs de référence ou aggravation des signes vitaux et des résultats de l’examen physique10French101242127b0ecf78-8c4b-4012-b063-876b928eb522• ECG’s • ECHO • Abnormale veranderingen ten opzichte van baseline of verslechtering van de vitale functies en bevindingen bij lichamelijk onderzoek18Dutch101242167b0ecf78-8c4b-4012-b063-876b928eb522• EKG-k • ECHO • Kóros változások a kiindulási értékhez képest vagy a vitális jelek és a fizikális vizsgálati leletek romlása13Hungarian101242137b0ecf78-8c4b-4012-b063-876b928eb522• EKG • ECHO • Nieprawidłowe zmiany w stosunku do wartości wyjściowej lub pogorszenie parametrów życiowych i wyników badania fizykalnego19Polish2026-10-312026-10-312022-05-3189435Sarepta Therapeutics, Inc.336414223364162552336415244233641726622PatientsFalseTrueTrue2acceptable2026-01-262026-01-2616Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]5Diseases [C] - Musculoskeletal Diseases [C05]127490Duchenne Muscular DystrophyTrue138769True409068PublicPatient RecruitmentSareptAlly@sarepta.com+18887273782360902Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active409069ScientificPatient RecruitmentSareptAlly@sarepta.com+18887273782360902Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active624346603124636004Non-Pharmaceutical company114g Clinical LLCTrueTrueORG-100042775Active651270370 Washington Street370 Washington StreetWellesley02481-6207840United States17816941400ewieland@4Gclinical.comTrueORG-100042775969992317816941400ewieland@4Gclinical.com624342603150636030Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active651296100 Federal Street100 Federal StreetAndover01810-1036840United States+16173018652Xzhang@sarepta.comTrueORG-10000664596998615Muscle biopsy analysis+16173018652Xzhang@sarepta.com624355603147636027Pharmaceutical company10Pharmaceutical Product Development LLCTrueTrueORG-100016999Active6512932 Tesseneer Drive2 Tesseneer DriveHighland Heights41076-9167840United States+18003232996Melissa.north@ppd.comTrueORG-10001699997000615coagulation screening, urinalysis, viral detection, renal function blood test, and liver chemistry9700074+18003232996Melissa.north@ppd.com624336603152636032Hospital/Clinic/Other health care facility8University Of Iowa Hospitals And ClinicsFalseTrueORG-100032405Active651298200 Hawkins Drive, 5239b Roy Carver Pavilion200 Hawkins Drive5239b Roy Carver PavilionIowa City52242-1009840United States193562140Steven-Moore@uiowa.eduTrueORG-1000324059699774193562140Steven-Moore@uiowa.edu624341603139636019Non-Pharmaceutical company11Greenphire LLCTrueTrueORG-100041621Active6512851018 West 9th Avenue Suite 2001018 West 9th Avenue Suite 200King Of Prussia19406-1225840United States9142639106drew.contessa@greenphire.comTrueORG-10004162196998515Patient travel reimbursement9142639106drew.contessa@greenphire.com624350603132636012Laboratory/Research/Testing facility9Elligo Health Research Inc.TrueTrueORG-100044201Active65127811612 Bee Caves Road Building 1 Suite 15011612 Bee Caves Road Building 1 Suite 150Austin78738-5409840United States115125804633kati.duncan@elligodirect.comTrueORG-10004420196999615Patient recruitment and retention115125804633kati.duncan@elligodirect.com624333603137636017Laboratory/Research/Testing facility9Eurofins Central Laboratory Pte LtdTrueTrueORG-100050415Active6512831 International Business Park, The Synergy1 International Business ParkThe SynergySingapore609917702Singapore6565623858aleksandrastophel@eurofinsus.comTrueORG-10005041596997415GLDH Testing6565623858aleksandrastophel@eurofinsus.com624330842967884548Laboratory/Research/Testing facility9Emvenio Clinical Research LLCTrueTrueORG-100044408Active937022500 East 8th Avenue500 East 8th AvenueDenver80203-3716840United States3032537470Jonathan.ernst@pcmtrials.comTrueORG-10004440896997115Home Health Care3032537470Jonathan.ernst@pcmtrials.com624340603146636026Pharmaceutical company10PPD Global Central Labs (S) Pte LtdTrueTrueORG-100041754Active65129261 Science Park Road, #02-12 1561 Science Park Road#02-12 15Singapore117525702Singapore6565946210siteservices.sg@ppd.comTrueORG-10004175496998315coagulation screening, urinalysis, viral detection, renal function blood test, and liver chemistry96998446565946210siteservices.sg@ppd.com624354603144636024Pharmaceutical company10Pharpoint Research Inc.TrueTrueORG-100048095Active6512905003 South Miami Boulevard Suite 1005003 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StreetCambridge02142-1213840United StatesTrueORG-100006645TrueCommercial2According to the "Appendix on transparency rules to the functional specifications of the EU clinical trials portal and database" chapter 4.3.3, Category 2 trials are safety and efficacy trials in patients. The current study meets this requirement, this is a Phase 3b study.110910816Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]11091075Diseases [C] - Musculoskeletal Diseases [C05]379505ETEPLIRSEN (AVI-4658)UK MIA 203771Test508040110281667SOLUTION FOR INFUSIONPRD94568671ETEPLIRSEN (AVI-4658)SOLUTION FOR INFUSIONAVI-4658ETEPLIRSENSUB129287ETEPLIRSENSAREPTA THERAPEUTICS INC10281667269047SAREPTA THERAPEUTICS INCNucleic AcidNucleic AcidETEPLIRSENSUB129287False1TrueEU/3/08/586mg/kg milligram(s)/kilogram0mg milligram(s)01142Chemical (Phosphorodiamidate morpholino oligomer for exon 51 skipping)PRD9456867AVI-4658UK MIA 2037711INTRAVENOUSFalseETEPLIRSEN (AVI-4658)eteplirsenSOLUTION FOR INFUSIONFalse2019403976339763122392014Ended2024-10-232024-10-23FalseunwillingGreece2024-12-09Ended462732022-03-102024-12-17462742022-03-102024-12-17563562022-03-102025-05-072025-05-09621182022-05-312022-05-312024-12-22621192022-05-312022-05-312024-12-22620922022-05-312024-12-22620932022-05-312024-12-22True820312022-03-102025-05-072024-12-17FalseFalseFalseTrue2022-05-312022-05-31False5411039763Under evaluation2024-09-26T18:23:25.7747172739763Authorised2024-12-09T17:46:03.2768259739763Ended2025-05-09T16:50:49.6151Greeceacceptable2025-05-09authorized2024-12-092024-12-0911332349599821632687Hospital/Clinic/Other health care facility8Iaso Private General Obstetrics Gynecological & Pediatric Clinic Diagnostic Therapeutic & Research Center S.A.FalseTrueORG-100046574Active647546Kifissias Leoforos 37-39Kifissias Leoforos 37-39Filothei151 232014Greece+302106383000theon@otenet.grTrueORG-1000465741485126Antigoni E.Papavasileiou+302106383000theon@otenet.gr1Neurological Department of Pediatric ClinicAuthorised2004393999739997122392013Authorised2024-10-232024-10-23FalseunwillingGermany2024-11-04Authorised462872022-10-232024-12-17462882022-10-232024-12-17621382023-05-052023-08-232024-12-22621392023-05-052023-08-232024-12-22621122023-05-052024-12-22621132023-05-052024-12-22True636052022-10-232024-12-172023-05-052023-08-23False5410939997Under evaluation2024-09-26T18:23:25.7555965239997Authorised2024-11-04T13:15:28.0261Germanyacceptable2025-06-30authorized2024-11-042024-11-0431321553603606636487Hospital/Clinic/Other health care facility8Charite Universitaetsmedizin Berlin KöRFalseTrueORG-100008480Active651801Augustenburger Platz 1, WeddingAugustenburger Platz 1WeddingBerlin133532013Germany+4930450666279claudia.weiss@charite.deTrueORG-1000084801473097ClaudiaWeiss+4930450666279claudia.weiss@charite.de1Klinik für Pädiatrie mit Schwerpunkt Neurologie und Sozialpädiatrisches Zentrum der CharitéAuthorised2004463974139741122392029Ended2024-10-232024-10-23FalseunwillingNetherlands2024-11-04Ended462912022-11-162024-12-17462922022-11-162024-12-17713222022-11-162025-11-252025-12-18621242023-01-192023-01-192024-12-22621252023-01-192023-01-192024-12-22621082023-01-192024-12-22621092023-01-192024-12-22True1119132022-11-162025-11-252024-12-17FalseFalseFalseTrue2023-01-192023-01-19False5411639741Under evaluation2024-09-26T18:23:25.8685959639741Authorised2024-11-04T09:39:45.60419572939741Ended2025-12-18T11:54:38.6381Netherlandsacceptable2025-06-10authorized2024-11-042024-11-0411321574597602630426Hospital/Clinic/Other health care facility8Radboud universitair medisch centrum StichtingFalseTrueORG-100023234Active645062Geert Grooteplein Zuid 10Geert Grooteplein Zuid 10Nijmegen6525 GA2029Netherlands+31243614892clinicaltrials.reval@radboudumc.nlTrueORG-1000232341473130SaskiaHouwen+31243614892clinicaltrials.reval@radboudumc.nl1Medical RehabilitationAuthorised2004473970839708122392030Authorised2024-10-232024-10-23FalseunwillingNorway2024-11-05Authorised462812022-08-252024-12-17462822022-08-252024-12-17621162023-08-282024-12-22621172023-08-282024-12-22621402023-08-282023-08-282024-12-22621412023-08-282023-08-282024-12-22True636022022-08-252024-12-172023-08-282023-08-28False5976839708Authorised2024-11-05T08:55:30.5715411739708Under evaluation2024-09-26T18:23:25.8841Norwayacceptable2025-06-19authorized2024-11-052024-11-0511321575598125630950Hospital/Clinic/Other health care facility8Helse Stavanger HFFalseTrueORG-100030100Active645631Gerd-Ragna Bloch Thorsens Gate 8Gerd-Ragna Bloch Thorsens Gate 8Stavanger40112030Norway+4751514700katja.sara.brocke.holmefjord@sus.noTrueORG-1000301001473136Katja SaraHolmefjord+4751514700katja.sara.brocke.holmefjord@sus.no1Barneklinikk, Gerd-Ragna Bloch Thorsens gate 8, 4011 StavangerAuthorised2004503961139611122392015Ended2024-10-232024-10-23FalseunwillingHungary2024-11-05Ended462832022-10-052024-12-17462842022-10-052024-12-17662472022-10-052025-10-082025-10-09621022022-10-252024-12-22621032022-10-252024-12-22621202022-10-252022-11-032024-12-22621212022-10-252022-11-032024-12-22True1014952022-10-052025-10-082024-12-17FalseFalseFalseTrue2022-10-252022-11-03False5411139611Under evaluation2024-09-26T18:23:25.795981239611Authorised2024-11-05T11:57:20.50510197739611Ended2025-10-09T21:10:38.3091Hungaryacceptable2025-07-07authorized2024-11-052024-11-0541321580595279628091Educational Institution1Semmelweis UniversityTrueTrueORG-100023482Active642451Gyulai Pal Utca 2, KeruletGyulai Pal Utca 2KeruletBudapest VIII10852015Hungary+36206632513molnarmj@gmail.comTrueORG-1000234821473146Mária JuditMolnár+36206632513molnarmj@gmail.com2Genomikai Medicina és Ritka Betegségek IntézeteAuthorised2456683976139761122392009Authorised2024-10-232024-10-23TruewillingDenmark2024-11-04Authorised462892022-10-242024-12-17462902022-10-242024-12-17621302022-11-252023-06-262024-12-22621312022-11-252023-06-262024-12-22621042022-11-252024-12-22621052022-11-252024-12-22True636062022-10-242024-12-172022-11-252023-06-26True5410739761Under evaluation2024-09-26T18:23:25.7235967139761Authorised2024-11-04T14:17:10.8061Denmarkauthorized2024-11-042024-11-0431670967599818632684Hospital/Clinic/Other health care facility8RigshospitaletFalseTrueORG-100002431Active647543Blegdamsvej 9Blegdamsvej 9Copenhagen Oe21002009Denmark+4525786197malene.landbo.02.boerresen@regionh.dkTrueORG-1000024311857632Malene LandboBørresen+4525786197malene.landbo.02.boerresen@regionh.dk1Department of Pediatric NeurologyAuthorised2456713976539765122392031Authorised2024-10-232024-10-23FalseunwillingPoland2024-11-05Authorised462752022-05-172024-12-17462762022-05-172024-12-17620962022-08-082024-12-22620972022-08-082024-12-22621342022-08-082023-07-112024-12-22621352022-08-082023-07-112024-12-22True635982022-05-172024-12-172022-08-082023-07-11False5411839765Under evaluation2024-09-26T18:23:25.9025986939765Authorised2024-11-05T16:21:38.611Polandauthorized2024-11-052024-11-0571670971599311632141Hospital/Clinic/Other health care facility8Uniwersyteckie Centrum KliniczneFalseTrueORG-100042500Active646960Ul. Debinki 7Ul. Debinki 7Gdansk80-9522031Poland+48501498492mmazur@gumed.edu.plTrueORG-1000425001857636MariaMazurkiewicz-Bełdzińska+48501498492mmazur@gumed.edu.pl2Klinika Neurologii RozwojowejAuthorised2456703970739707122392027Ended2024-10-232024-10-23FalseunwillingSpain2024-11-04Ended462712021-12-172024-12-17462722021-12-172024-12-17709072021-12-172025-12-092025-12-15743112021-12-172025-12-102026-02-04621002022-08-292024-12-22621012022-08-292024-12-22621262022-08-292023-01-262024-12-22621272022-08-292023-01-262024-12-22True1177442021-12-172025-12-102024-12-17FalseFalseFalseTrue2022-08-292023-01-26False5411539707Under evaluation2024-09-26T18:23:25.8525960039707Authorised2024-11-04T10:05:59.19118613739707Ended2025-12-15T11:45:08.2711Spainauthorized2024-11-042024-11-0431670969596728629544Hospital/Clinic/Other health care facility8Hospital Sant Joan De Deu BarcelonaFalseTrueORG-100023083Active644084Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain34932804000andres.nascimento@sjd.esTrueORG-1000230831857634AndrésNascimento34932804000andres.nascimento@sjd.es1Pediatric Neurology1670970596729629545Hospital/Clinic/Other health care facility8Hospital Universitario Y Politecnico La FeFalseTrueORG-100029610Active644085Avenida Fernando Abril Martorell 106Avenida Fernando Abril Martorell 106Valencia460262027Spain34961244000muelas_nur@gva.esTrueORG-1000296101857635NuriaMuelas34961244000muelas_nur@gva.es1NeurologyAuthorised2456693976439764122392026Ended2024-10-232024-10-23FalseunwillingSlovenia2024-11-08Ended462852022-10-102024-12-17462862022-10-102024-12-17717082022-10-102025-12-222025-12-23739402022-10-102026-01-192026-01-29621222022-12-122022-12-122024-12-22621232022-12-122022-12-122024-12-22621062022-12-122024-12-22621072022-12-122024-12-22True1170772022-10-102026-01-192024-12-17FalseFalseFalseTrue2022-12-122022-12-12False5411439764Under evaluation2024-09-26T18:23:25.8366281939764Authorised2024-11-08T09:28:39.23120779739764Ended2025-12-23T09:21:44.5241Sloveniaauthorized2024-11-082024-11-0811670968599539632369Hospital/Clinic/Other health care facility8University Medical Center LjubljanaFalseTrueORG-100012686Active647220Bohoriceva Ulica 20Bohoriceva Ulica 20Ljubljana10002026Slovenia+38615229272damjan.osredkar@kclj.siTrueORG-1000126861857633DamjanOsredkar+38615229272damjan.osredkar@kclj.si2Department of Pediatric NeurologyAuthorised2483613976239762122392012Authorised2024-10-232024-10-23FalseunwillingFrance2024-11-13Authorised462692021-12-092024-12-17462702021-12-092024-12-17620942022-06-222024-12-22620952022-06-222024-12-22621282022-06-222023-06-052024-12-22621292022-06-222023-06-052024-12-22True635952021-12-092024-12-172022-06-222023-06-05False5410839762Under evaluation2024-09-26T18:23:25.7396907939762Authorised2024-11-13T13:24:18.7071Franceacceptable2025-11-20authorized2024-11-132024-11-1341691267599397632227Hospital/Clinic/Other health care facility8Association Institut De MyologieFalseTrueORG-100046467Active647059Porte 20 2eme Etage, 26 Avenue Du Docteur Arnold NetterPorte 20 2eme Etage26 Avenue Du Docteur Arnold NetterParis750122012France+33171738050a.seferian@institut-myologie.orgTrueORG-1000464671880195AndreeaSEFERIAN+33171738050a.seferian@institut-myologie.org1I-Motion instituteAuthorised2483583999839998122392018Authorised2024-10-232024-10-23FalseunwillingItaly2024-11-25Authorised462952023-07-122024-12-17462962023-07-122024-12-17621362023-07-272023-08-032024-12-22621372023-07-272023-08-032024-12-22621142023-07-272024-12-22621152023-07-272024-12-22True636092023-07-122024-12-172023-07-272023-08-03False5411239998Under evaluation2024-09-26T18:23:25.8057036439998Authorised2024-11-25T23:59:591Italyacceptable2025-11-10authorized2024-11-252024-11-2531691262603415636296Hospital/Clinic/Other health care facility8IRCCS Ospedale Policlinico San MartinoFalseTrueORG-100008531Active651586Largo Rosanna Benzi 10Largo Rosanna Benzi 10Genoa161322018Italy+3901056362912claudiobruno@ospedalegaslini.ge.itTrueORG-1000085311880190ClaudioBruno+3901056362912claudiobruno@ospedalegaslini.ge.it2U.O.S.D. 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BrnoFalseTrueORG-100022981Active643268Cernopolni 9, Cerna PoleCernopolni 9Cerna PoleBrno613 002008Czechia+420532234933Mrazova.Lenka@fnbrno.czTrueORG-1000229811880192LenkaJuříková+420532234933Mrazova.Lenka@fnbrno.cz1Klinika dětské neurologie LF MU a FN Brno Dětská nemocnice1691265596001628815Hospital/Clinic/Other health care facility8Fakultni Nemocnice V MotoleFalseTrueORG-100012719Active643263V Uvalu 84/1, MotolV Uvalu 84/1MotolPrague150 002008Czechia+420224433367jana.haberlova@fnmotol.czTrueORG-1000127191880193JanaHaberlová+420224433367jana.haberlova@fnmotol.cz1Klinika dětské neurologie 2.LF UK a FN MotolAuthorised2901183976739767122392025Authorised2024-10-232024-10-23FalseunwillingRomania2024-11-06Authorised462932022-12-132024-12-17462942022-12-132024-12-17621422023-02-202023-09-012024-12-22621432023-02-202023-09-012024-12-22621102023-02-202024-12-22621112023-02-202024-12-22True636082022-12-132024-12-172023-02-202023-09-01False5411339767Under evaluation2024-09-26T18:23:25.8216000839767Authorised2024-11-06T13:30:23.5831Romaniaauthorized2024-11-062024-11-0631998509598302631130Hospital/Clinic/Other health care facility8Centrul National Clinic De Recuperare Neuropsihomotorie Copii Dr. Nicolae RobanescuFalseTrueORG-100046423Active645830Strada Sold Dumitru Minca 44 Sector 4Strada Sold Dumitru Minca 44 Sector 4Bucharest0414082025Romania+40730758847mada_mada332@yahoo.comTrueORG-1000464232219710Madalina CristinaLeanca+40730758847mada_mada332@yahoo.com1Sectia Neurologie PediatricaAuthorised19606INITIALAuthorised2024-511492-15-00Authorised2024-09-26acceptable2024-10-311500003961039610Czechia2008acceptable2024-10-30authorized2024-11-05AuthorisedCzechiaAuthorised2024-11-05Authorised1500013961139611Hungary2015acceptable2024-10-30authorized2024-11-05EndedHungaryEnded2024-11-05Authorised1500023970739707Spain2027acceptable2024-10-30authorized2024-11-04EndedSpainEnded2024-11-04Authorised1500113999739997Germany2013acceptable2024-10-30authorized2024-11-04AuthorisedGermanyAuthorised2024-11-04Authorised1500103976739767Romania2025acceptable2024-11-01authorized2024-11-06AuthorisedRomaniaAuthorised2024-11-06Authorised1500123999839998Italy2018acceptable2024-11-18authorized2024-11-25AuthorisedItalyAuthorised2024-11-25Authorised1500043974139741Netherlands2029acceptable2024-10-30authorized2024-11-04EndedNetherlandsEnded2024-11-04Authorised1500053976139761Denmark2009acceptable2024-11-04authorized2024-11-04AuthorisedDenmarkAuthorised2024-11-04Authorised1500063976239762France2012acceptable2024-11-07authorized2024-11-13AuthorisedFranceAuthorised2024-11-13Authorised1500073976339763Greece2014acceptable2024-12-05authorized2024-12-09EndedGreeceEnded2024-12-09Authorised1500083976439764Slovenia2026acceptable2024-11-08authorized2024-11-08EndedSloveniaEnded2024-11-08Authorised1500093976539765Poland2031acceptable2024-10-30authorized2024-11-05AuthorisedPolandAuthorised2024-11-05Authorised1500033970839708Norway2030acceptable2024-10-31authorized2024-11-05AuthorisedNorwayAuthorised2024-11-05Authorised2024-11-0439610CzechiaAuthorised39611HungaryEnded39707SpainEnded39997GermanyAuthorised39767RomaniaAuthorised39998ItalyAuthorised39741NetherlandsEnded39761DenmarkAuthorised39762FranceAuthorised39763GreeceEnded39764SloveniaEnded39765PolandAuthorised39708NorwayAuthorisedINFalse482471960639998Italy2024-11-25T00:00:00authorizedacceptabledecision15001260150INITIALFalse448061960639761Denmark2024-11-04T14:17:12.17authorizedacceptabledecision15000560150INITIALTrue449581960639610Czechia2024-11-05T08:18:34.885authorizedacceptabledecision15000060150INITIALFalse449631960639708Norway2024-11-05T08:55:33.308authorizedacceptabledecision15000360150INITIALFalse450391960639611Hungary2024-11-05T11:57:22.822authorizedacceptabledecision15000160150INITIALFalse446951960639741Netherlands2024-11-04T09:39:49.2authorizedacceptabledecision15000460150INITIALFalse447041960639707Spain2024-11-04T10:06:01.789authorizedacceptabledecision15000260150INITIALFalse464981960639762France2024-11-13T13:24:21.418authorizedacceptabledecision15000660150INITIALFalse447791960639997Germany2024-11-04T13:15:30.164authorizedacceptabledecision15001160150INITIALFalse502451960639763Greece2024-12-09T17:46:05.558authorizedacceptabledecision15000760150INITIALFalse455921960639764Slovenia2024-11-08T09:28:41.19authorizedacceptabledecision15000860150INITIALFalse451451960639765Poland2024-11-05T16:21:40.638authorizedacceptabledecision15000960150INITIALFalse453131960639767Romania2024-11-06T13:30:25.413authorizedacceptabledecision15001060150INITIALFalse44119NON SUBSTANTIAL MODIFICATIONAuthorised2024-511492-15-00Authorised2024-12-20acceptable2024-10-312024-12-2039610CzechiaAuthorised39611HungaryEnded39707SpainEnded39997GermanyAuthorised39767RomaniaAuthorised39998ItalyAuthorised39741NetherlandsEnded39761DenmarkAuthorised39762FranceAuthorised39763GreeceEnded39764SloveniaEnded39765PolandAuthorised39708NorwayAuthorisedNSM-1PART_IFalse520334411939610Czechia2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939611Hungary2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939707Spain2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939997Germany2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939767Romania2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939998Italy2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939741Netherlands2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939761Denmark2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONTrue520334411939762France2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939763Greece2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939764Slovenia2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939765Poland2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse520334411939708Norway2024-12-20T15:53:27.28authorizeddecision68135NON SUBSTANTIAL MODIFICATIONFalse47706NON SUBSTANTIAL MODIFICATIONAuthorised2024-511492-15-00Authorised2025-02-18acceptable2024-10-311795003999739997Germany2013authorized2024-11-04AuthorisedGermanyAuthorised2024-11-04Authorised1795013970839708Norway2030authorized2024-11-05AuthorisedNorwayAuthorised2024-11-05Authorised2025-02-1839997GermanyAuthorised39708NorwayAuthorisedNSM-2PART_IIFalse584664770639997Germany2025-02-18T11:00:31.566authorizeddecision17950073598NON SUBSTANTIAL MODIFICATIONFalse584654770639708Norway2025-02-18T11:00:31.566authorizeddecision17950173598NON SUBSTANTIAL MODIFICATIONFalse49234SUBSTANTIAL MODIFICATIONAuthorised2024-511492-15-00Authorised2025-03-03acceptable2019403976339763Greece2014acceptable2025-05-09authorized2024-12-09EndedGreeceEnded2024-12-09Authorised2025-05-1339763GreeceEndedSM-1PART_IIFalse710004923439763Greece2025-05-13T11:47:31.675authorizedacceptabledecision20194075880SUBSTANTIAL MODIFICATIONFalse53326SUBSTANTIAL 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muscular dystrophy (DMD)Netherlands:4Italy:4Belgium:407/10/2024BE: 07/10/2024, NL: 11/10/2024, IT: 21/11/2024Diseases [C] - Musculoskeletal Diseases [C05]Italfarmaco S.p.A.Pharmaceutical companyTherapeutic exploratory (Phase II)CORE PHASE: • Type, incidence, and severity of treatment-emergent adverse events (TEAEs) and SAEs from baseline to Week 48 • Proportion of subjects experiencing TEAEs from baseline to Week 48 • Change from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 48 • Change from baseline electrocardiogram (ECG) to each postbaseline visit up to Week 48, CORE PHASE: • Change in physical function as per the Bayley III Gross Motor scale from baseline to Week 48 for subjects aged ≥2 to <3.5 years of age • Change in physical function as per the North Star Ambulatory Assessment (NSAA) total score from baseline to Week 48 for subjects aged ≥3.5 years of age, CORE PHASE: • Change in Paediatric Outcomes Data Collection Instrument (PODCI) from baseline to Week 48 for subjects aged ≥4 years of age in Cohort 1 only, EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of patients experiencing TEAEs from baseline to Week 144 • Change from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 144 • Change from baseline ECG to each postbaseline up to Week 144ITF23570-17 yearsMale3Both10CORE PHASE: Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Maximum plasma concentration at steady state (Cmax,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Elimination half life (t1/2) assessed after at least 7 days of dosing dosing (at Week 1 and at 6 months), EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of subjects experiencing TEAEs from 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synopsis_2024-511823-32-00_FR-BE7a366564-bfe7-43ca-b5df-06845d4cf03b7Synopsis of the protocol (for publication)PDF788452.02D1_ Protocol synopsis_2024-511823-32-00_DE-BE9e66ff4f-0c2f-4de2-8d6e-64bca51d9e9e7Synopsis of the protocol (for publication)PDF788452.02D1_ Protocol synopsis_2024-511823-32-00_NL-BE501d48fa-7b07-47a2-921e-13d8d9a8f7357Synopsis of the protocol (for publication)PDF788452.02D1_protocol synopsis_2024-511823-32-00_NL_NLacc1907c-a82b-4402-9823-7ae4de0ce5d67Synopsis of the protocol (for publication)PDF788452.02D1_protocol synopsis_2024-511823-32-00_IT-IT0115371e-fddf-420a-bbb5-b7fb48341ab17Synopsis of the protocol (for publication)PDF788452.02D1_ Protocol synopsis_2024-511823-32-00_EN375f8ed8-5d40-4583-8516-eeb390cc911a7Synopsis of the protocol (for publication)PDF788452.02Netherlands296362024-10-11T09:30:00.5922024-10-11T09:30:00.5924Italy296352024-10-08T19:21:33.7172024-11-21T13:08:28.9554Belgium296342024-10-07T15:44:11.1772024-10-07T15:44:11.177429634BelgiumSTART_OF_TRIAL2024-12-04START_OF_RECRUITMENT2025-01-0229635ItalySTART_OF_TRIAL2024-12-18START_OF_RECRUITMENT2025-01-0829636NetherlandsSTART_OF_TRIAL2024-11-06START_OF_RECRUITMENT2025-01-312024-511823-32-004A Phase 2 Open label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years OldDSC/14/2357/5206/11/2024Duchenne muscular dystrophy (DMD)Netherlands:4Italy:4Belgium:407/10/2024BE: 07/10/2024, NL: 11/10/2024, IT: 21/11/2024Diseases [C] - Musculoskeletal Diseases [C05]Italfarmaco S.p.A.Pharmaceutical companyTherapeutic exploratory (Phase II)CORE PHASE: • Type, incidence, and severity of treatment-emergent adverse events (TEAEs) and SAEs from baseline to Week 48 • Proportion of subjects experiencing TEAEs from baseline to Week 48 • Change from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 48 • Change from baseline electrocardiogram (ECG) to each postbaseline visit up to Week 48, CORE PHASE: • Change in physical function as per the Bayley III Gross Motor scale from baseline to Week 48 for subjects aged ≥2 to <3.5 years of age • Change in physical function as per the North Star Ambulatory Assessment (NSAA) total score from baseline to Week 48 for subjects aged ≥3.5 years of age, CORE PHASE: • Change in Paediatric Outcomes Data Collection Instrument (PODCI) from baseline to Week 48 for subjects aged ≥4 years of age in Cohort 1 only, EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of patients experiencing TEAEs from baseline to Week 144 • Change from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 144 • Change from baseline ECG to each postbaseline up to Week 144ITF23570-17 yearsMale310CORE PHASE: Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Maximum plasma concentration at steady state (Cmax,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Elimination half life (t1/2) assessed after at least 7 days of dosing dosing (at Week 1 and at 6 months), EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of subjects experiencing TEAEs from baseline to Week 144No21/03/202514/05/20262024-511823-32-00Authorised2024-11-062024-10-07T15:44:11.1772026-05-14T03:39:38.7761883564788458100000000556United Kingdom826GBGBRTrue322860111133213ORAL SUSPENSIONPRD47976781ITF2357ORAL SUSPENSIONITF2357GIVINOSTATSUB189959GIVINOSTATITALFARMACO SPA11133213337329ITALFARMACO SPAChemicalChemicalGIVINOSTATSUB189959False1TrueEU/3/12/1009mg milligram(s)66.6g gram(s)67.1363histone deacetylase (HDAC) inhibitorPRD4797678ITF235711ORAL USETrueITF2357givinostatORAL SUSPENSIONA Phase 2 Open label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old585659784ed9e8c-b97c-4f47-a094-34a95f3f8481Een open-label fase 2-onderzoek (kernfase plus uitbreidingsfase) met 2 cohorten ter beoordeling van de farmacokinetiek en veiligheid van givinostat bij DMD-patiënten in de leeftijd van ten minste 2 jaar tot minder dan 6 jaar oud37Dutch (Netherlands)A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old58566063ab585e1-e572-4f84-9e53-ebe47aa7f692Een open-label fase 2-onderzoek (kernfase plus uitbreidingsfase) met 2 cohorten ter beoordeling van de farmacokinetiek en veiligheid van givinostat bij DMD-patiënten in de leeftijd van ten minste 2 jaar tot minder dan 6 jaar oud37Dutch (Netherlands)DSC/14/2357/52361377U1111-1308-1552False42Phase II Duchenne Muscular Dystrophy7018180278Duchenne muscular dystrophy (DMD)585656774c64b67-9d89-463d-ba0f-bb4e42a94b2eZiekte van Duchenne (DMD)37Dutch (Netherlands)True623216213Tolerability2321617232164923216532321634232166CORE PHASE: To assess the pharmacokinetic (PK) profile of givinostat at steady state administered in younger DMD boys in the 2 cohorts. EXTENSION PHASE: To assess the safety and tolerability of givinostat long-term administered in younger DMD boys in the 2 cohorts (ie, adverse events [AE])58566059c9f1692-eca0-4abf-aeb8-00a2a4a75924CORE PHASE: Het beoordelen van het farmacokinetische (PK) profiel van givinostat in stationaire toestand toegediend bij jongere jongens met DMD in de 2 cohorten. EXTENSION PHASE: Het beoordelen van de veiligheid en verdraagbaarheid van givinostat op lange termijn toegediend bij jongere jongens met DMD in de 2 cohorten (d.w.z. bijwerkingen [AE])37Dutch (Netherlands)2629251CORE PHASE: To assess the safety and tolerability of givinostat administered in younger DMD boys in the 2 cohorts5856607052db8bf-8adc-4fe4-95c5-b48d2a7f1b5eHet beoordelen van de veiligheid en verdraagbaarheid van givinostat toegediend bij jongere jongens met DMD in de 2 cohorten37Dutch (Netherlands)2629262CORE PHASE: To evaluate the effect of givinostat on muscular functional parameters in the 2 cohorts58566088b8211aa-b74e-4a26-957a-e71680003775Het beoordelen van het effect van givinostat op spierfunctieparameters in de 2 cohorten37Dutch (Netherlands)2629273CORE PHASE: To evaluate the effect of givinostat on functional health-related quality of life (HRQOL) in Cohort 1 (for subjects ≥4 years only)585660961b601dc-1608-4f2d-ae20-8502fd6b604bHet beoordelen van het effect van givinostat op de functionele gezondheidsgerelateerde kwaliteit van leven (HRQOL) in cohort 1 (alleen voor proefpersonen ≥ 4 jaar)37Dutch (Netherlands)2629284EXTENSION PHASE: To further evaluate the safety and tolerability of givinostat long-term administered in younger DMD boys in the 2 cohorts (eg, vital signs, laboratory test values, electrocardiogram [ECG] parameters) cohorts.5856610c2a5ff48-52d8-4ee4-a0ea-423918be7298Het verder beoordelen van de veiligheid en verdraagbaarheid van toediening op lange termijn van givinostat bij jongere jongens met DMD in de 2 cohorten (bijv. vitale functies, laboratoriumtestwaarden, parameters voor elektrocardiogram [ECG])37Dutch (Netherlands)2629295EXTENSION PHASE: To evaluate the effect of long-term administered givinostat on muscular functional parameters in the 2 cohorts5856611bf64c1b3-5072-4305-992e-9e4f86256549Het beoordelen van het effect van toediening op lange termijn van givinostat op spierfunctionele parameters in de 2 cohorten37Dutch (Netherlands)2629306EXTENSION PHASE: To evaluate the effect of long-term administered givinostat on functional HRQOL in Cohort 1 (for subjects >4 years only)58566122e76c2b9-572f-4692-9677-836d6bb6b5b1Het beoordelen van het effect van toediening op lange termijn van givinostat op de functionele HRQOL in cohort 1 (alleen voor proefpersonen > 4 jaar)37Dutch (Netherlands)5097221CORE PHASE: Male children aged ≥2 to <6 years at screening (subjects ≥6 years of age at screening will not be enrolled into the study)5856598b6639473-0fe3-4ea1-8ede-768bcaed5e50Mannelijke kinderen van ≥ 2 tot < 6 jaar bij de screening (proefpersonen ≥ 6 jaar bij de screening worden niet in het onderzoek ingeschreven)37Dutch (Netherlands)5097232CORE PHASE: Written consent provided by parent/legal guardian and subject written assent, if applicable (according to local regulation)58565990de7ae24-695a-4296-b238-28200cde8862Schriftelijke toestemming gegeven door ouder/wettelijke voogd en schriftelijke instemming door de proefpersoon, indien van toepassing (volgens lokale regelgeving)37Dutch (Netherlands)5097243CORE PHASE: A genetic diagnosis of DMD58566008c0be8b4-8a77-445e-be27-123e63e84ff4Een genetische diagnose van DMD hebben37Dutch (Netherlands)5097254CORE PHASE: Corticosteroid treatment considerations: a. For subjects receiving a stable dose of oral systemic corticosteroids: No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 3 months immediately prior to the start of study drug or b. For subjects without current corticosteroids treatment: Must not start corticosteroids during core phase (ie, first 48 weeks)5856601c3ade9c8-fa93-412f-a539-8f94ee13ab44Overwegingen voor behandeling met corticosteroïden: a. Voor proefpersonen die een stabiele dosis orale systemische corticosteroïden krijgen: Geen belangrijke verandering in dosis of doseringsschema (behalve aanpassingen als gevolg van verandering in lichaamsgewicht) gedurende minimaal 3 maanden onmiddellijk voorafgaand aan de start van het onderzoeksmiddel of b. Voor proefpersonen zonder huidige corticosteroïdenbehandeling: Mag niet beginnen met corticosteroïden tijdens de kernfase (d.w.z. eerste 48 weken)37Dutch (Netherlands)5097265EXTENSION PHASE: Must have participated in the Core Phase study (48 weeks) and have attended the End of Treatment Visit (EOT/V12)58566020b82ffef-3996-4424-ade4-42ddedb42a22Moet hebben deelgenomen aan het kernfaseonderzoek (48 weken) en hebben deelgenomen aan het bezoek voor einde van behandeling (EvB/B12)37Dutch (Netherlands)5097276EXTENSION PHASE: Give informed consent and /or assent in writing signed by the parent/legal guardian and/or subject (according to local regulation)585660371181c3f-d4d0-4d85-88ea-a0946bfff725Schriftelijk toestemming en/of instemming geven ondertekend door de ouder/wettelijke vertegenwoordiger en/of proefpersoon (volgens lokale regelgeving)37Dutch (Netherlands)5097287EXTENSION PHASE: In stable oral systemic corticosteroids treatment with no significant change in dose or dosing regimen (except for adjustments due to body weight change). For subjects without corticosteroids during the Core Phase, the treatment can be started based on the Investigator’s clinical medical judgement.5856604b0180ae3-4060-4455-928e-62176446910aBij stabiele behandeling met orale systemische corticosteroïden zonder belangrijke verandering in dosis of doseringsschema (behalve aanpassingen als gevolg van verandering in lichaamsgewicht). Voor proefpersonen zonder corticosteroïden tijdens de kernfase kan de behandeling worden gestart op basis van het klinisch-medisch oordeel van de onderzoeker.37Dutch (Netherlands)8785051CORE PHASE: Exposure to any other investigational drug within 3 months prior to the start of study drug585657422a1cd75-49c9-46f6-b521-d7648e1e3c05Blootstelling aan een ander onderzoeksmiddel binnen 3 maanden voorafgaand aan de start van het onderzoeksmiddel37Dutch (Netherlands)87850610EXTENSION PHASE: Current liver disease or impairment, including but not limited to an elevated total bilirubin (ie, >1.5 × ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert disease585657513df6b28-bebe-4553-a061-93451c9cb19dHuidige leverziekte of -insufficiëntie, inclusief maar niet beperkt tot een verhoogd totaal bilirubine (d.w.z. > 1,5 × ULN), tenzij secundair aan de ziekte van Gilbert of een patroon dat consistent is met de ziekte van Gilbert37Dutch (Netherlands)87850711EXTENSION PHASE: Inadequate renal function, as defined by serum Cystatin C result >2 × ULN5856576ab061dc7-7cfb-431c-b545-4111bae81799Onvoldoende nierfunctie, zoals gedefinieerd met een cystatine C-serumresultaat > 2 × ULN (opmerking: als de waarde > 2 × ULN is, wordt de cystatine C-serumtest eenmaal herhaald; als het herhaalde testresultaat nog steeds > 2 × ULN is, moet de proefpersoon worden uitgesloten)37Dutch (Netherlands)87850812CORE PHASE: Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug5856577259f4fd2-d6f5-41eb-9b3e-31ec719bbedaBlootstelling aan een dystrofine-herstellend product (bijv. ataluren, exon-skipping) binnen 6 maanden voorafgaand aan de start van het onderzoeksmiddel37Dutch (Netherlands)87850913EXTENSION PHASE: Fasting triglycerides >300 mg/dL (3.42 mmol/L)58565785b49230c-8ee5-4a4d-83f4-fa2292b5f189Nuchtere triglyceriden > 300 mg/dl (3,42 mmol/l; NB: als de waarde > 300 mg/dl is, worden de triglyceriden eenmaal herhaald; als het herhaalde testresultaat nog steeds > 300 mg/dl is in nuchtere toestand, moet de proefpersoon worden uitgesloten)37Dutch (Netherlands)87851014EXTENSION PHASE: Have presence of other clinically significant disease, which, in the Investigator’s opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results58565794d17cae1-9fb8-48b0-b0a2-7548ced89066Aanwezigheid van een andere klinisch significante ziekte die, naar het oordeel van de onderzoeker, een negatieve invloed kan hebben op de veiligheid van de proefpersoon, waardoor het onwaarschijnlijk is dat het verloop van de behandeling of de follow-up zou worden voltooid, of die de beoordeling van de onderzoeksresultaten zou kunnen belemmeren37Dutch (Netherlands)87851115EXTENSION PHASE: Evidence of psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on the Investigator’s clinical medical judgement5856580bbd1be31-8ef7-4c31-a708-5109b162481cBewijs van psychiatrische ziekte of sociale omstandigheden waardoor de potentiële proefpersoon niet in staat is om de spierfunctietests en/of de procedures van het onderzoeksprotocol te begrijpen en na te leven, op basis van het klinisch-medische oordeel van de onderzoeker.37Dutch (Netherlands)87851216CORE PHASE: Received any gene therapy (eg, Adeno-associated viruses Micro dystrophin delivery) within 12 months prior to the start of study drug5856581059ac38d-0df5-47dc-9803-833f3111d821Een gentherapie ontvangen (bijv. adeno-gerelateerde virusgemedieerde toediening van micro-dystrofine) binnen 12 maanden voorafgaand aan de start van het onderzoeksmiddel37Dutch (Netherlands)87851317CORE PHASE: Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to the start of study drug (eg, growth hormone)5856582814bbfa1-dcdc-4c83-bcc8-33e722389e02Gebruik van een farmacologische behandeling, anders dan corticosteroïden, dat een effect kan hebben gehad op de spierkracht of -functie binnen 3 maanden voorafgaand aan de start van het onderzoeksmiddel (bijv. groeihormoon). NB: Vitamine D, calcium en andere supplementen zijn toegestaan37Dutch (Netherlands)87851418CORE PHASE: Have had surgery that might have an effect on muscle strength or function within 3 months prior to the start of the study drug or planned surgery at any time during the study58565832732df9d-7808-4c71-b159-77123801d01cHeeft een operatie ondergaan die mogelijk een effect heeft op de spierkracht of -functie binnen 3 maanden voorafgaand aan de start van het onderzoeksmiddel of een geplande operatie op enig moment tijdens het onderzoek37Dutch (Netherlands)87851519CORE PHASE: The presence of other clinically significant disease, which, in the Investigator’s opinion, could adversely affect subject’s safety, making it unlikely to complete the study or to be compliant with study-specific requirements that could impair the assessment of study results585658491a2ea6b-114f-462a-b8db-c0fd46285042De aanwezigheid van een andere klinisch significante ziekte, die naar het oordeel van de onderzoeker de veiligheid van de proefpersoon negatief kan beïnvloeden, waardoor het onwaarschijnlijk is dat het onderzoek wordt voltooid of dat de onderzoekspecifieke vereisten die de beoordeling van de onderzoeksresultaten zouden kunnen belemmeren, worden nageleefd37Dutch (Netherlands)8785162CORE PHASE: Inadequate renal function, as defined by serum Cystatin C result >2 × ULN (Note: if the value is >2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still >2 × ULN, the subject will be excluded)5856585cdcc20f4-fa4f-42e4-8017-b12e558e1a35Onvoldoende nierfunctie, zoals gedefinieerd met een cystatine C-serumresultaat > 2 × ULN (opmerking: als de waarde > 2 × ULN is, wordt de cystatine C-serumtest eenmaal herhaald; als het herhaalde testresultaat nog steeds > 2 × ULN is, moet de proefpersoon worden uitgesloten)37Dutch (Netherlands)87851720CORE PHASE: Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on the Investigator's clinical medical judgement5856586cf903ed3-eda2-431c-9660-3ecd365d1713Diagnose van andere ongecontroleerde neurologische ziekten of aanwezigheid van relevante ongecontroleerde somatische aandoeningen die geen verband houden met DMD, op basis van het klinisch-medische oordeel van de onderzoeker37Dutch (Netherlands)87851821CORE PHASE: Platelet count, white blood cells, and/or haemoglobin < lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results [585658775a2f924-2b30-4309-ae73-454053c7e987Aantal bloedplaatjes, witte bloedcellen en/of hemoglobine < ondergrens van normaal (LLN) bij de screening (NB: voor afwijkende laboratoriumtestresultaten bij de screening [< LLN], worden het aantal bloedplaatjes, witte bloedcellen en hemoglobine eenmaal herhaald; als het herhaalde testresultaat nog steeds < LLN is, moet de proefpersoon worden uitgesloten)37Dutch (Netherlands)87851922CORE PHASE: Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, >1.5 × upper limit of normal [ULN]), unless secondary to Gilbert disease or pattern consistent with Gilbert disease5856588e370a7f6-5613-43b7-9e3c-e3ce8264e285Huidige of voorgeschiedenis van leverziekte of -insufficiëntie, inclusief maar niet beperkt tot een baseline van verhoogd totaal bilirubine (d.w.z. > 1,5 × bovengrens van normaal [ULN]), tenzij secundair aan de ziekte van Gilbert of een patroon dat consistent is met de ziekte van Gilbert37Dutch (Netherlands)87852023CORE PHASE: Body weight <10 kg at screening5856589a535291b-4146-4c69-a8b7-dc58b217cf3bLichaamsgewicht < 10 kg bij screening37Dutch (Netherlands)8785213CORE PHASE: Fasting triglycerides >300 mg/dL (3.42 mmol/L) at screening (Note: if the value is >300 mg/dL, the triglycerides will be repeated once; if the repeated test result is still >300 mg/dL in fasting condition, the subject should be excluded)585659008cc2974-95e3-440f-a164-0050fd03d626Nuchtere triglyceriden > 300 mg/dl (3,42 mmol/l) bij de screening (opmerking: als de waarde > 300 mg/dl is, worden de triglyceriden eenmaal herhaald; als het herhaalde testresultaat nog steeds > 300 mg/dl is in nuchtere toestand, moet de patiënt worden uitgesloten)37Dutch (Netherlands)8785224CORE PHASE: Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening5856591d76fabe1-c17b-4fce-b3a0-4af2c4d3e543Positieve test voor hepatitis B-oppervlakteantigeen, hepatitis C-virus antilichaamserologie of hiv bij de screening37Dutch (Netherlands)8785235CORE PHASE: Baseline corrected QT interval using Fredericia’s formula (QTcF) >450 msec (as the mean of 3 consecutive readings taken 5 minutes apart) or history of additional risk factors for torsades de pointes (eg, heart failure, hypokalaemia, or family history of long QT syndrome)5856592568efb80-ea0a-4ff0-8cf8-8a38b82fc360Gecorrigeerd QT-interval bij de baseline met behulp van Fridericia’s formule (QTcF) > 450 msec (als gemiddelde van 3 opeenvolgende metingen met een tussentijd van 5 minuten) of een voorgeschiedenis van bijkomende risicofactoren voor torsade de pointe (bijv. hartfalen, hypokaliëmie of familiegeschiedenis van lang QT-syndroom)37Dutch (Netherlands)8785246CORE PHASE: Psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on the Investigator’s clinical medical judgement585659370ecfe29-624f-4044-9813-e0620f8e94f4Psychiatrische ziekte of sociale omstandigheden waardoor de potentiële proefpersoon niet in staat is om de spierfunctietests en/of de procedures van het onderzoeksprotocol te begrijpen en na te leven, op basis van het klinisch-medische oordeel van de onderzoeker37Dutch (Netherlands)8785257CORE PHASE: Hypersensitivity to any component of the study drug5856594a8201a2b-de37-4965-894c-e7ee14813c0bOvergevoeligheid voor een bestanddeel van het onderzoeksmiddel37Dutch (Netherlands)8785268CORE PHASE: Sorbitol intolerance or malabsorption, or have the hereditary form of fructose intolerance5856595e4ad30e7-4c9e-4a16-a6f0-b8c578987ec1Intolerantie of malabsorptie van sorbitol, of heeft de erfelijke vorm van fructose-intolerantie37Dutch (Netherlands)8785279EXTENSION PHASE: Platelet count, white blood cells, and/or haemoglobin < LLN at EOT/V125856596119c7a62-786d-48fb-a453-4c3854f1f0b0Aantal bloedplaatjes, witte bloedcellen en/of hemoglobine < LLN bij EvB/B12 (NB: voor afwijkende laboratoriumtestresultaten [< LLN], worden het aantal bloedplaatjes, witte bloedcellen en hemoglobine eenmaal herhaald; als het herhaalde testresultaat nog steeds < LLN is, moet de proefpersoon worden uitgesloten)37Dutch (Netherlands)5939331CORE PHASE: Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Maximum plasma concentration at steady state (Cmax,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Elimination half life (t1/2) assessed after at least 7 days of dosing dosing (at Week 1 and at 6 months)True5856568657a90e2-0d80-4bfe-a5cf-324fa03238a7• Gebied onder de concentratie-tijdcurve van dosering (tijd 0) tot tijd t in stationaire toestand (AUC0-T, ss) na ten minste 7 dagen dosering (na week 1 en na 6 maanden) • Maximale plasmaconcentratie in stationaire toestand (Cmax,ss) na ten minste 7 dagen dosering (na week 1 en na 6 maanden) • Eliminatiehalfwaardetijd (t1/2) beoordeeld na ten minste 7 dagen dosering (na week 1 en na 6 maanden)37Dutch (Netherlands)5939342EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of subjects experiencing TEAEs from baseline to Week 144True5856569f4f82d12-e5f4-4614-b8ea-0751678cd979• Type, incidentie en ernst van TEAE’s en SAE’s vanaf de baseline tot week 144 • Percentage proefpersonen dat TEAE’s ervaart vanaf de baseline tot week 14437Dutch (Netherlands)5939351CORE PHASE: • Type, incidence, and severity of treatment-emergent adverse events (TEAEs) and SAEs from baseline to Week 48 • Proportion of subjects experiencing TEAEs from baseline to Week 48 • Change from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 48 • Change from baseline electrocardiogram (ECG) to each postbaseline visit up to Week 48False585657067f1f75f-5791-4049-bd7f-e959a85b712c• Type, incidentie en ernst van tijdens de behandeling optredende AE’s (TEAE’s) en SAE’s vanaf de baseline tot week 48 • Percentage proefpersonen dat TEAE’s ervaart vanaf de baseline tot week 48 • Verandering ten opzichte van de baseline in vitale functies en klinisch laboratorium onderzoek tot elk bezoek na de baseline tot week 48 • Verandering ten opzichte van het baseline-ECG tot elk bezoek na de baseline tot week 4837Dutch (Netherlands)5939362CORE PHASE: • Change in physical function as per the Bayley III Gross Motor scale from baseline to Week 48 for subjects aged ≥2 to <3.5 years of age • Change in physical function as per the North Star Ambulatory Assessment (NSAA) total score from baseline to Week 48 for subjects aged ≥3.5 years of ageFalse585657166896ffe-9c55-4f8a-9714-6b12a508cb7d• Verandering in lichamelijk functioneren volgens de Bayley III Gross Motor-schaal van baseline tot week 48 voor proefpersonen van ≥ 2 tot < 3,5 jaar oud • Verandering in lichamelijk functioneren volgens de totale score van de North Star Ambulatory Assessment (NSAA) vanaf de baseline tot week 48 voor proefpersonen ≥ 3,5 jaar oud37Dutch (Netherlands)5939373CORE PHASE: • Change in Paediatric Outcomes Data Collection Instrument (PODCI) from baseline to Week 48 for subjects aged ≥4 years of age in Cohort 1 onlyFalse5856572434828ee-1a0c-4829-b093-2af4272f99a6• Verandering in Pediatric Outcomes Data Collection Instrument (PODCI) vanaf baseline tot week 48, alleen voor proefpersonen van ≥ 4 jaar in cohort 137Dutch (Netherlands)5939384EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of patients experiencing TEAEs from baseline to Week 144 • Change from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 144 • Change from baseline ECG to each postbaseline up to Week 144False58565739cef4980-b2ab-46ef-8d7b-b2332ff48e3b• Verandering ten opzichte van de baseline in vitale functies en klinisch laboratorium onderzoek tot week 144 • Verandering ten opzichte van het baseline-ECG tot elk bezoek na de baseline tot week 14437Dutch (Netherlands)2028-12-312028-12-312024-09-01213912222PatientsFalseTrueTrue3NAP1162991Open label, multicentre, multicountry, 2 cohorts to evaluate the PK profile and safety of givinostatSubjects with DMD aged ≥4 to <6 years for Cohort 1 and aged ≥2 to <4 years for Cohort 2. The starting dose for Cohort 2 will be confirmed/adjusted with results of the interim analysis of Cohort 1. The study will consist of 2 phases, a core phase and an extension phase. Two final analysis will be conducted, the first at the end of the Core Phase and the second at the end of the Extension Phase (core and extension data will be combined)3An open label core phase for both cohorts during which subjects will receive givinostat for 48 weeksTest6547336569EMEA-000551-PIP04-21197362023-503521-19-00Italfarmaco S.p.A.Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular DystrophyNON_APPLICABLENON_APPLICABLE3744False361378PMID 27566866PMID 27566866https://pubmed.ncbi.nlm.nih.gov/27566866acceptable5Diseases [C] - Musculoskeletal Diseases [C05]80278Duchenne muscular dystrophy (DMD)True85657True255660PublicPatient Advocacy Representative of Italfarmaco S.p.Apatientadvocacy@italfarmacogroup.com+00000371837Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active255661ScientificNicoletta Coceani – Clinical Scientistn.coceani@italfarmacogroup.com390264432512371837Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active368324456446488750Non-Pharmaceutical company11ATOM International LimitedTrueTrueORG-100042393Active489212Office 16, Valley House, Seventh Avenue, Team Valley Trading EstateOffice 16Valley HouseSeventh AvenueTeam Valley Trading EstateGatesheadNE11 0JW826United Kingdom000000000michelle.eagle@atom-international.orgTrueORG-1000423935775886000000000michelle.eagle@atom-international.org368323454923487226Pharmaceutical company10Alira HealthTrueTrueORG-100030303Active48757012 Rue Charlot12 Rue CharlotParis750032012France000000000lisa.comarella@alirahealth.comTrueORG-100030303577586105775876000000000lisa.comarella@alirahealth.com368325454926487229Hospital/Clinic/Other health care facility8Syneos Health Clinique Inc.FalseTrueORG-100028348Active4875742500 Einstein St2500 Einstein StQuebecG1P 0A2124Canada000000000na@na.comTrueORG-1000283485775894000000000na@na.com368321454907487210Pharmaceutical company10Fortrea Inc.TrueTrueORG-100012602Active4875518 Moore Drive8 Moore DriveDurham27709-0009840United States33147168200submissons@fortea.comTrueORG-10001260257757715775781157757912577580135775811457758225775834577584533147168200submissons@fortea.com368322465360497682Hospital/Clinic/Other health care facility8Blueprint Genetics OyFalseTrueORG-100050758Active498876Keilaranta 16 AKeilaranta 16 AEspoo021502011Finland000000000adriano.saitta@blueprintgenetics.comTrueORG-1000507585775854000000000adriano.saitta@blueprintgenetics.com371837Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active342329371837Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active365638Via Dei Lavoratori 54Via Dei Lavoratori 54Cinisello Balsamo200922018ItalyTrueORG-100000118TrueCommercial2Phase II Duchenne Muscular Dystrophy8102915Diseases [C] - Musculoskeletal Diseases [C05]245918ITF23571Test322860111133213ORAL SUSPENSIONPRD47976781ITF2357ORAL SUSPENSIONITF2357GIVINOSTATSUB189959GIVINOSTATITALFARMACO SPA11133213337329ITALFARMACO SPAChemicalChemicalGIVINOSTATSUB189959False1TrueEU/3/12/1009mg milligram(s)66.6g gram(s)67.1363histone deacetylase (HDAC) inhibitorPRD4797678ITF235711ORAL USETrueITF2357givinostatORAL SUSPENSIONFalse1376302963429634125872002Authorised2024-09-132024-09-13FalseunwillingBelgium2024-10-07Authorised476512024-12-042025-01-09476522024-12-042025-01-09632622025-01-022025-01-09632632025-01-022025-01-09True658672024-12-042025-01-092025-01-02False3931329634Under evaluation2024-06-17T11:08:47.2385558829634Authorised2024-10-07T15:44:10.6891Belgiumacceptable2024-09-25authorized2024-10-072024-10-072898475428580460483Hospital/Clinic/Other health care facility8Association Hospitaliere De Bruxelles Hopital Universitaire Des Enfants Reine FabiolaFalseTrueORG-100045965Active458648Jean Joseph Crocqlaan 15Jean Joseph Crocqlaan 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synopsis_2024-511823-32-00_EN375f8ed8-5d40-4583-8516-eeb390cc911a7Synopsis of the protocol (for publication)PDF788452.02Both3 2024-512828-12-005AuthorisedAn open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study)SNT-IV-VAM-011Duchenne Muscular DystrophyCzechia:5Spain:5Belgium:5Greece:5Ireland:5Netherlands:504/10/2024GR: 15/04/2025, NL: 03/02/2025, CZ: 08/10/2024, BE: 04/10/2024, ES: 07/10/2024, IE: 21/02/2025Diseases [C] - Musculoskeletal Diseases [C05]Santhera Pharmaceuticals (Schweiz) AGPharmaceutical companyTherapeutic use (Phase IV)Time to first vertebral fractures (cumulative incidence), Number of non-vertebral fractures per 1000 person-years based on investigator reporting, Time to first non-vertebral fractures (cumulative incidence), Number of cataracts per 1000 person-years based on ophthalmologist assessment, Number of subjects not reaching Tanner stage 2 by 15 years of age, Frequency of adverse events (AEs) and serious adverse events (SAEs), Change from baseline in body weight, height and body mass index (BMI), Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisol, Change from baseline in Time to Stand Test (TTSTAND) velocity, 6-Minute Walk Test (6MWT) distance, Change from baseline in 6MWT distance, NorthStar Ambulatory Assessment (NSAA) scores, Age at ambulatory and non-ambulatory milestonesAGAMREE 40 mg/ml oral suspension560-17 yearsMale3Both26Number of vertebral fractures per 1000 person-years based on X- ray central readingNo08/01/202609/01/20262024-10-04T16:39:21.6862026-01-09T02:31:24.5787860685K1_Recruitment arrangements_sanitized57d039f5-40ce-4b54-bc1b-38159f63788914Recruitment arrangements (for publication)PDF2363551.01L1_GR_Parent-legal guardian_For publicationdb0dcd2f-63fa-48df-ae19-00a3b0dd192d15Subject 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and informed consent form (for publication)PDF2586062.02L1_IE_SIS and ICF_Parent legal guardian_For publication200da526-3cd0-4d82-97eb-e66f82e58e7b15Subject information and informed consent form (for publication)PDF2586065.05L1_IE_SIS and ICF_Pregnant partner adult form_For publication53d82392-d733-4754-a768-6594746664a115Subject information and informed consent form (for publication)PDF25860611L1_IE_SIS and ICF_Pregnant partner assent form_For publication8eeddb97-0949-4960-bec4-14e4f971c29315Subject information and informed consent form (for publication)PDF25860611Czechia331862024-10-08T15:35:28.772025-09-17T10:44:34.9025Spain331882024-10-07T10:53:22.8442025-09-30T14:46:23.0295Belgium331852024-10-04T16:39:21.6862025-10-24T13:56:33.4615Greece413512025-01-28T11:48:30.132025-10-13T10:46:18.8515Ireland413522025-02-21T13:36:10.3542026-01-08T10:44:19.5475Netherlands413532025-02-03T09:43:33.8622025-10-29T11:35:30.528533185BelgiumSTART_OF_TRIAL2025-01-14START_OF_RECRUITMENT2025-02-05END_OF_RECRUITMENT2025-03-0633186CzechiaSTART_OF_TRIAL2024-12-05START_OF_RECRUITMENT2024-12-11END_OF_RECRUITMENT2025-03-1233188SpainSTART_OF_TRIAL2025-01-07START_OF_RECRUITMENT2025-02-03END_OF_RECRUITMENT2025-02-1941351GreeceSTART_OF_TRIAL2025-07-01START_OF_RECRUITMENT2025-07-04END_OF_RECRUITMENT2025-09-0941352IrelandSTART_OF_TRIAL2025-05-13START_OF_RECRUITMENT2025-05-23END_OF_RECRUITMENT2025-05-2341353NetherlandsSTART_OF_TRIAL2025-03-26START_OF_RECRUITMENT2025-04-10END_OF_RECRUITMENT2025-06-102024-512828-12-005An open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study)SNT-IV-VAM-01105/12/2024Duchenne Muscular DystrophyCzechia:5Spain:5Belgium:5Greece:5Ireland:5Netherlands:504/10/2024GR: 15/04/2025, NL: 03/02/2025, CZ: 08/10/2024, BE: 04/10/2024, ES: 07/10/2024, IE: 21/02/2025Diseases [C] - Musculoskeletal Diseases [C05]Santhera Pharmaceuticals (Schweiz) AGPharmaceutical companyTherapeutic use (Phase IV)Time to first vertebral fractures (cumulative incidence), Number of non-vertebral fractures per 1000 person-years based on investigator reporting, Time to first non-vertebral fractures (cumulative incidence), Number of cataracts per 1000 person-years based on ophthalmologist assessment, Number of subjects not reaching Tanner stage 2 by 15 years of age, Frequency of adverse events (AEs) and serious adverse events (SAEs), Change from baseline in body weight, height and body mass index (BMI), Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisol, Change from baseline in Time to Stand Test (TTSTAND) velocity, 6-Minute Walk Test (6MWT) distance, Change from baseline in 6MWT distance, NorthStar Ambulatory Assessment (NSAA) scores, Age at ambulatory and non-ambulatory milestonesAGAMREE 40 mg/ml oral suspension560-17 yearsMale326Number of vertebral fractures per 1000 person-years based on X- ray central readingNo08/01/202609/01/20262024-512828-12-00Authorised2024-12-052024-10-04T16:39:21.6862026-01-09T02:31:24.578786068510824139100000000556United Kingdom826GBGBRTrue100000000429Israel376ILISRTrue100000000329Australia36AUAUSTrue100000000482New Zealand554NZNZLTrue448629111343078ORAL SUSPENSIONPRD11022268EU/1/23/1776/0012AGAMREE 40 mg/ml oral suspensionORAL SUSPENSIONVAMOROLONESUB188638EUEMEA/H/C/005679SANTHERA PHARMACEUTICALS (DEUTSCHLAND) GMBH11343078387855SANTHERA PHARMACEUTICALS (DEUTSCHLAND) GMBHChemicalChemicalVAMOROLONESUB188638H02ABGLUCOCORTICOIDS4False1TrueEU/3/14/1309mg milligram(s)240mg/kg milligram(s)/kilogram6False483GlucocorticoidsPRD1102226811ORAL USETrueAGAMREE 40 mg/ml oral suspensionvamoroloneORAL SUSPENSIONAn open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study)8738039e49569e0-dc6f-4ea9-8067-16e858844879Een openlabel onderzoek om informatie te verzamelen over de veiligheid en werkzaamheid van de behandeling op de lange termijn met vamorolon bij jongens met Duchenne spierdystrofie (DMD) die eerdere onderzoeken met vamorolon hebben voltooid (het GUARDIAN-onderzoek)37Dutch (Netherlands)8738040e49569e0-dc6f-4ea9-8067-16e858844879Μια μελέτη ανοικτής επισήμανσης για τη συλλογή πληροφοριών σχετικά με την ασφάλεια και την αποτελεσματικότητα της μακροχρόνιας θεραπείας με βαμορολόνη σε αγόρια με μυϊκή δυστροφία Duchenne, που έχουν ολοκληρώσει προηγούμενες μελέτες με βαμορολόνη (Η μελέτη GUARDIAN)5Greek8738041e49569e0-dc6f-4ea9-8067-16e858844879Estudio abierto para recopilar información relativa a seguridad y eficacia del tratamiento prolongado con vamorolona en chicos con distrofia muscular de Duchenne que han completado estudios previos con vamorolona Estudio GUARDIAN7SpanishAn open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study)8738057a8847e9c-15db-4bdf-80c9-52ad28187533Μια μελέτη ανοικτής επισήμανσης για τη συλλογή πληροφοριών σχετικά με την ασφάλεια και την αποτελεσματικότητα της μακροχρόνιας θεραπείας με βαμορολόνη σε αγόρια με μυϊκή δυστροφία Duchenne, που έχουν ολοκληρώσει προηγούμενες μελέτες με βαμορολόνη (Η μελέτη GUARDIAN)5Greek8738058a8847e9c-15db-4bdf-80c9-52ad28187533Een openlabel onderzoek om informatie te verzamelen over de veiligheid en werkzaamheid van de behandeling op de lange termijn met vamorolon bij jongens met Duchenne spierdystrofie (DMD) die eerdere onderzoeken met vamorolon hebben voltooid (het GUARDIAN-onderzoek)37Dutch (Netherlands)8738059a8847e9c-15db-4bdf-80c9-52ad28187533Estudio abierto para recopilar información relativa a seguridad y eficacia del tratamiento prolongado con vamorolona en chicos con distrofia muscular de Duchenne que han completado estudios previos con vamorolona Estudio GUARDIAN7SpanishSNT-IV-VAM-011False63This is a phase IV study, thus under trial category 3. This is an open-label, multi-center trial designed to collect long-term safety and effectiveness data from boys with Duchenne Muscular Dystrophy (DMD) who have participated in studies VBP15-002/003/LTE and VBP15-004 of vamorolone clinical development program.98635112113Duchenne Muscular Dystrophy87379868f38bb37-753c-4552-95f9-f2e20deadff0Μυϊκή δυστροφία Duchenne5Greek87379858f38bb37-753c-4552-95f9-f2e20deadff0Distrofia muscular de Duchenne (DMD)7Spanish87379878f38bb37-753c-4552-95f9-f2e20deadff0Duchenne spierdystrofie37Dutch (Netherlands)True10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False43263795326378To evaluate the safety of long-term treatment with vamorolone in boys with DMD regarding vertebral fractures8738054971dd6fd-fcb5-448f-83ea-d66e8baab4d7Het beoordelen van de veiligheid van de behandeling op de lange termijn met vamorolon bij jongens met DMD met betrekking tot wervelfracturen.37Dutch (Netherlands)8738055971dd6fd-fcb5-448f-83ea-d66e8baab4d7Evaluar la seguridad del tratamiento prolongado con vamorolona en chicos con DMD en lo que respecta a fracturas vertebrales7Spanish8738056971dd6fd-fcb5-448f-83ea-d66e8baab4d7Η αξιολόγηση της ασφάλειας της μακροχρόνιας θεραπείας με βαμορολόνη σε αγόρια με DMD σε σχέση με σπονδυλικά κατάγματα5Greek3761001To evaluate the safety of long-term treatment with vamorolone in boys with DMD on non-vertebral fractures, cataracts, and delayed puberty.8738061d8802a8e-6cfd-44fb-85fa-4b8238b39bfcΗ αξιολόγηση της ασφάλειας της μακροχρόνιας θεραπείας με βαμορολόνη σε αγόρια με DMD σε σχέση με μη σπονδυλικά κατάγματα, καταρράκτη και καθυστερημένη εφηβεία.5Greek8738062d8802a8e-6cfd-44fb-85fa-4b8238b39bfcEvaluar la seguridad del tratamiento prolongado con vamorolona en chicos con DMD en lo que respecta a fracturas no vertebrales, cataratas y retraso de la pubertad7Spanish8738060d8802a8e-6cfd-44fb-85fa-4b8238b39bfcHet beoordelen van de veiligheid van de behandeling op de lange termijn met vamorolon bij jongens met DMD op niet-wervelfracturen, staargevallen en vertraagde puberteit.37Dutch (Netherlands)3761012To evaluate the overall safety of long-term treatment with vamorolone in boys with DMD8738063a939a4d7-d7e3-49e7-8205-790e8a06a37fHet beoordelen van de algehele veiligheid van de behandeling op de lange termijn37Dutch (Netherlands)8738065a939a4d7-d7e3-49e7-8205-790e8a06a37fΗ αξιολόγηση της συνολικής ασφάλειας της μακροχρόνιας θεραπείας με βαμορολόνη σε αγόρια με DMD5Greek8738064a939a4d7-d7e3-49e7-8205-790e8a06a37fEvaluar la seguridad global del tratamiento prolongado con vamorolona en chicos con DMD7Spanish3761023To evaluate the long-term treatment effect of vamorolone on ambulatory and non-ambulatory function8738067a7590339-8aef-4a56-adf6-ab64d738caf5Η αξιολόγηση της μακροχρόνιας θεραπευτικής επίδρασης της βαμορολόνης στην περιπατητική και μη περιπατητική λειτουργία5Greek8738066a7590339-8aef-4a56-adf6-ab64d738caf5Het beoordelen van het behandelingseffect op de lange termijn van vamorolon op ambulante en niet-ambulante functies.37Dutch (Netherlands)8738068a7590339-8aef-4a56-adf6-ab64d738caf5Evaluar el efecto del tratamiento prolongado con vamorolona sobre la función deambulatoria y no deambulatoria7Spanish7218001Subject and/or subject’s parent(s) or legal guardian has provided written informed consent873804308dd0957-d18b-4c00-8b13-7afcad5eadcdEl sujeto y/o sus padres o su tutor legal han otorgado su consentimiento informado por escrito7Spanish873804408dd0957-d18b-4c00-8b13-7afcad5eadcdDe proefpersoon en/of de ouder(s) of wettelijke vertegenwoordiger van de proefpersoon heeft/hebben schriftelijke geïnformeerde toestemming gegeven.37Dutch (Netherlands)873804208dd0957-d18b-4c00-8b13-7afcad5eadcdΟ ασθενής ή/και ο(οι) γονέας(-είς) ή ο νόμιμος κηδεμόνας του ασθενούς έχει παράσχει γραπτή συναίνεση μετά από ενημέρωση5Greek7218012Subject has previously completed either the VBP15-LTE or VBP15-004 study, and transitioned through the CUP, NPP or EAP8738047936541b8-f164-416a-90d2-3d5ccfb837ceΟ ασθενής έχει προηγουμένως ολοκληρώσει είτε τη μελέτη VBP15-LTE είτε τη μελέτη VBP15-004 και έχει μεταβεί μέσω των προγραμμάτων CUP, NPP ή EAP5Greek8738045936541b8-f164-416a-90d2-3d5ccfb837ceEl sujeto ha completado previamente el estudio VBP15-LTE o el estudio VBP15-004 y ha pasado por un PUC, un PPD o un PAE.7Spanish8738046936541b8-f164-416a-90d2-3d5ccfb837ceDe proefpersoon heeft eerder ofwel het VBP15-LTE- ofwel het VBP15-004-onderzoek voltooid, en is overgestapt via het CUP (Compassionate Use Program), NPP (Named Patient Program) of EAP (Extended Access Program).37Dutch (Netherlands)7218023Subject is on vamorolone on the day of enrolment8738049decf361d-17f2-4d3d-8008-5866d57fd27aEl sujeto está recibiendo vamorolona el día del reclutamiento7Spanish8738048decf361d-17f2-4d3d-8008-5866d57fd27aΟ ασθενής λαμβάνει βαμορολόνη την ημέρα της ένταξης5Greek8738050decf361d-17f2-4d3d-8008-5866d57fd27aProefpersoon gebruikt vamorolon op de dag van inschrijving.37Dutch (Netherlands)7218034Subject and parent / legal guardian are willing and able to comply with the protocol schedule, assessments and requirements873805315ffa453-3d1e-4440-af9d-04d3cff22fb0De proefpersoon en zijn ouder/wettelijke vertegenwoordiger zijn bereid en in staat om zich te houden aan het protocolschema, de beoordelingen en de vereisten.37Dutch (Netherlands)873805215ffa453-3d1e-4440-af9d-04d3cff22fb0Ο ασθενής και ο γονέας/νόμιμος κηδεμόνας είναι πρόθυμοι και ικανοί να συμμορφωθούν με το χρονοδιάγραμμα, τις αξιολογήσεις και τις απαιτήσεις του πρωτοκόλλου5Greek873805115ffa453-3d1e-4440-af9d-04d3cff22fb0El sujeto y sus padres/tutor legal pueden y están dispuestos a cumplir el calendario, las evaluaciones y los requisitos del protocolo7Spanish12385621Any medical condition, which in the opinion of the Investigator, would affect study participation, performance or interpretation of study assessments873803146e141c2-33e7-42e8-a2c9-00f021223adaΟποιαδήποτε ιατρική πάθηση, η οποία, κατά τη γνώμη του ερευνητή, θα επηρέαζε τη συμμετοχή στη μελέτη, την απόδοση ή την ερμηνεία των αξιολογήσεων της μελέτης5Greek873803046e141c2-33e7-42e8-a2c9-00f021223adaElke medische aandoening die, naar het oordeel van de onderzoeker, deelname aan of uitvoering van het onderzoek of de interpretatie van de onderzoeksbeoordelingen zou beïnvloeden.37Dutch (Netherlands)873803246e141c2-33e7-42e8-a2c9-00f021223adaCualquier trastorno médico que, en opinión del investigador, pueda afectar a la participación en el estudio o a la realización o interpretación de las evaluaciones del estudio.7Spanish12385632Vamorolone treatment discontinued for ≥6 months within the year prior to enrolment for a non-safety reason, or vamorolone treatment previously discontinued at any time for a safety reason8738035bd111b46-d30a-49c8-895a-23843251279cBehandeling met vamorolon die gedurende ≥ 6 maanden binnen het jaar voorafgaand aan inschrijving werd stopgezet om een andere reden dan vanwege de veiligheid, of behandeling met vamorolon die eerder op enig moment vanwege veiligheidsredenen werd stopgezet.37Dutch (Netherlands)8738033bd111b46-d30a-49c8-895a-23843251279cΗ θεραπεία με βαμορολόνη διακόπηκε για ≥6 μήνες εντός του έτους πριν από την ένταξη για λόγο μη σχετιζόμενο με την ασφάλεια ή η θεραπεία με βαμορολόνη διακόπηκε προηγουμένως οποιαδήποτε στιγμή για λόγο σχετιζόμενο με την ασφάλεια5Greek8738034bd111b46-d30a-49c8-895a-23843251279cSuspensión del tratamiento con vamorolona durante ≥ 6 meses en el año previo al reclutamiento por un motivo no relacionado con la seguridad o suspensión previa del tratamiento con vamorolona en cualquier momento por un motivo de seguridad7Spanish12385643Severe hepatic impairment8738036819d1773-e0d5-480d-8b8f-8d0f402bc9f0Ernstige leverinsufficiëntie.37Dutch (Netherlands)8738037819d1773-e0d5-480d-8b8f-8d0f402bc9f0Insuficiencia hepática grave7Spanish8738038819d1773-e0d5-480d-8b8f-8d0f402bc9f0Σοβαρή ηπατική δυσλειτουργία5Greek8451391Number of vertebral fractures per 1000 person-years based on X- ray central readingTrue8737988ada8ca76-4542-4552-b418-058af6ef89cfNúmero de fracturas vertebrales por 1000 años-persona según la interpretación radiológica centralizada7Spanish8737990ada8ca76-4542-4552-b418-058af6ef89cfΑριθμός σπονδυλικών καταγμάτων ανά 1000 ανθρωποέτη με βάση την κεντρική ανάγνωση των ακτινογραφιών5Greek8737989ada8ca76-4542-4552-b418-058af6ef89cfAantal wervelfracturen per 1000 manjaren op basis van centrale röntgenuitlezing.37Dutch (Netherlands)8451401Time to first vertebral fractures (cumulative incidence)False873799266a8c324-1e05-41f4-9b6a-6f72e40b3436Tiempo hasta la primera fractura vertebral (incidencia acumulada)7Spanish873799366a8c324-1e05-41f4-9b6a-6f72e40b3436Tijd tot eerste wervelfracturen (cumulatieve incidentie).37Dutch (Netherlands)873799166a8c324-1e05-41f4-9b6a-6f72e40b3436Χρόνος έως το πρώτο σπονδυλικό κάταγμα (σωρευτική επίπτωση)5Greek8451412Number of non-vertebral fractures per 1000 person-years based on investigator reportingFalse8737995ee941788-93a1-4a9c-9306-deef1a9dbdc1Αριθμός μη σπονδυλικών καταγμάτων ανά 1000 ανθρωποέτη με βάση την αναφορά του ερευνητή5Greek8737994ee941788-93a1-4a9c-9306-deef1a9dbdc1Aantal niet-wervelfracturen per 1000 manjaren op basis van rapportage door de onderzoeker.37Dutch (Netherlands)8737996ee941788-93a1-4a9c-9306-deef1a9dbdc1Número de fracturas no vertebrales por 1000 años-persona según la evaluación del investigador7Spanish8451423Time to first non-vertebral fractures (cumulative incidence)False873799903ae3fbc-2e93-4e15-b95e-0389736ca096Tijd tot eerste niet-wervelfracturen (cumulatieve incidentie).37Dutch (Netherlands)873799703ae3fbc-2e93-4e15-b95e-0389736ca096Tiempo hasta la primera fractura no vertebral (incidencia acumulada)7Spanish873799803ae3fbc-2e93-4e15-b95e-0389736ca096Χρόνος έως το πρώτο μη σπονδυλικό κάταγμα (σωρευτική επίπτωση)5Greek8451434Number of cataracts per 1000 person-years based on ophthalmologist assessmentFalse8738001ff788f34-2af4-4e49-878c-d21abebd4c36Aantal staargevallen per 1000 manjaren op basis van beoordeling door oogarts.37Dutch (Netherlands)8738000ff788f34-2af4-4e49-878c-d21abebd4c36Número de cataratas por 1000 años-persona según la evaluación del oftalmólogo7Spanish8738002ff788f34-2af4-4e49-878c-d21abebd4c36Αριθμός περιστατικών καταρράκτη ανά 1000 ανθρωποέτη με βάση την οφθαλμολογική αξιολόγηση5Greek8451445Number of subjects not reaching Tanner stage 2 by 15 years of ageFalse873800431e2f304-137f-4094-b80d-78266c2459c1Aantal proefpersonen dat op 15-jarige leeftijd Tanner-stadium 2 niet bereikt.37Dutch (Netherlands)873800331e2f304-137f-4094-b80d-78266c2459c1Número de sujetos que no hayan alcanzado el estadio 2 de Tanner a los 15 años de edad7Spanish873800531e2f304-137f-4094-b80d-78266c2459c1Αριθμός ασθενών που δεν έφτασαν σε στάδιο Tanner 2 έως την ηλικία των 15 ετών5Greek8451456Frequency of adverse events (AEs) and serious adverse events (SAEs)False8738006cb00984f-c064-425f-9c2c-2809e0ea0a40Frecuencia de acontecimientos adversos (AA) y acontecimientos adversos graves (AAG)7Spanish8738007cb00984f-c064-425f-9c2c-2809e0ea0a40Frequentie van bijwerkingen (AE’s) en ernstige bijwerkingen (SAE’s).37Dutch (Netherlands)8738008cb00984f-c064-425f-9c2c-2809e0ea0a40Συχνότητα ανεπιθύμητων συμβάντων (ΑΣ) και σοβαρών ανεπιθύμητων συμβάντων (ΣΑΣ)5Greek8451467Change from baseline in body weight, height and body mass index (BMI)False8738009c99dc9bd-e4a5-4d4d-9aea-6d163814857cVariación con respecto al valor basal del peso corporal, la estatura y el índice de masa corporal (IMC)7Spanish8738011c99dc9bd-e4a5-4d4d-9aea-6d163814857cVerandering ten opzichte van de baseline in lichaamsgewicht, lengte en body mass index (BMI).37Dutch (Netherlands)8738010c99dc9bd-e4a5-4d4d-9aea-6d163814857cΜεταβολή σε σχέση με την αξιολόγηση αναφοράς του σωματικού βάρους, του ύψους και του δείκτη μάζας σώματος (ΔΜΣ)5Greek8451478Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisolFalse8738014ebe6b197-1cc5-4f11-90e5-02f6dd4ce241Αριθμός ασθενών με κλινικά σημαντικές εργαστηριακές ανωμαλίες, συμπεριλαμβανομένης της γλυκοζυλιωμένης αιμοσφαιρίνης (HbA1c) και της πρωινής κορτιζόλης5Greek8738012ebe6b197-1cc5-4f11-90e5-02f6dd4ce241Aantal proefpersonen met klinisch relevante laboratoriumafwijkingen, waaronder geglycosyleerd hemoglobine (HbA1c) en ochtendcortisol.37Dutch (Netherlands)8738013ebe6b197-1cc5-4f11-90e5-02f6dd4ce241Número de sujetos con anomalías analíticas clínicamente relevantes, incluida hemoglobina glucosilada (HbA1c) y cortisol matutino7Spanish8451489Change from baseline in Time to Stand Test (TTSTAND) velocityFalse87380162d209338-e170-4906-85e3-85c56df2f14dVariación con respecto al valor basal de la velocidad en la prueba del tiempo en ponerse de pie (TTSTAND)7Spanish87380152d209338-e170-4906-85e3-85c56df2f14dΜεταβολή σε σχέση με την αξιολόγηση αναφοράς της ταχύτητας εκτέλεσης της δοκιμασίας του χρόνου έως την έγερση σε όρθια θέση (TTSTAND)5Greek87380172d209338-e170-4906-85e3-85c56df2f14dVerandering ten opzichte van de baseline in de snelheid van de tijd-tot-staan-test (TTSTAND).37Dutch (Netherlands)845149106-Minute Walk Test (6MWT) distanceFalse8738020e9336e9d-1748-4cd0-8ed0-e71ebd6b8634Απόσταση της δοκιμασίας βάδισης 6 λεπτών (6MWT)5Greek8738018e9336e9d-1748-4cd0-8ed0-e71ebd6b8634Distancia recorrida en la prueba de marcha de 6 minutos (6MWT)7Spanish8738019e9336e9d-1748-4cd0-8ed0-e71ebd6b86346-Minuten looptest (6MWT) afstand.37Dutch (Netherlands)84515011Change from baseline in 6MWT distanceFalse873802308910413-9e4b-4323-b31c-4c3c8405ea46Μεταβολή σε σχέση με την αξιολόγηση αναφοράς στην απόσταση της δοκιμασίας 6MWT5Greek873802208910413-9e4b-4323-b31c-4c3c8405ea46Variación de la distancia recorrida en la 6MWT con respecto al momento basal7Spanish873802108910413-9e4b-4323-b31c-4c3c8405ea46Verandering ten opzichte van de baseline in 6MWT-afstand.37Dutch (Netherlands)84515112NorthStar Ambulatory Assessment (NSAA) scoresFalse87380254b10eff0-a43f-426f-9a02-1168c9fa2575Βαθμολογίες της περιπατητικής αξιολόγησης NorthStar (NSAA)5Greek87380264b10eff0-a43f-426f-9a02-1168c9fa2575NorthStar Ambulatory Assessment (NSAA) scores.37Dutch (Netherlands)87380244b10eff0-a43f-426f-9a02-1168c9fa2575Puntuaciones NSAA (Evaluación deambulatoria de NorthStar)7Spanish84515213Age at ambulatory and non-ambulatory milestonesFalse8738029b7976fcb-8cc6-4e34-ac11-b9bb90ece44cΗλικία επίτευξης των περιπατητικών και μη περιπατητικών ορόσημων5Greek8738028b7976fcb-8cc6-4e34-ac11-b9bb90ece44cEdad al alcanzar los hitos deambulatorios y no deambulatorios7Spanish8738027b7976fcb-8cc6-4e34-ac11-b9bb90ece44cLeeftijd bij ambulante en niet-ambulante mijlpalen.37Dutch (Netherlands)2028-10-312028-10-312024-10-3178652Santhera Pharmaceuticals (Switzerland) Ltd.29714322297144255229714526622PatientsFalseTrueTrue3acceptable_conditions5Diseases [C] - Musculoskeletal Diseases [C05]112113Duchenne Muscular DystrophyTrue121734True359238PublicSanthera General Inquiriesoffice@santhera.com+41619068950409705Pharmaceutical company10Santhera Pharmaceuticals (Schweiz) AGTrueTrueORG-100004668Active359239ScientificSanthera General Inquiriesoffice@santhera.com+41619068950409705Pharmaceutical company10Santhera Pharmaceuticals (Schweiz) AGTrueTrueORG-100004668Active409705Pharmaceutical company10Santhera Pharmaceuticals (Schweiz) AGTrueTrueORG-100004668Active380074409705Pharmaceutical company10Santhera Pharmaceuticals (Schweiz) AGTrueTrueORG-100004668Active406082Hohenrainstrasse 24Hohenrainstrasse 24Pratteln4133756SwitzerlandTrueORG-100004668TrueCommercial3This is a phase IV study, thus under trial category 3. This is an open-label, multi-center trial designed to collect long-term safety and effectiveness data from boys with Duchenne Muscular Dystrophy (DMD) who have participated in studies VBP15-002/003/LTE and VBP15-004 of vamorolone clinical development program.10160545Diseases [C] - Musculoskeletal Diseases [C05]336513AGAMREE 40 mg/ml oral suspension1Test448629111343078ORAL SUSPENSIONPRD11022268EU/1/23/1776/0012AGAMREE 40 mg/ml oral suspensionORAL SUSPENSIONVAMOROLONESUB188638EUEMEA/H/C/005679SANTHERA PHARMACEUTICALS (DEUTSCHLAND) GMBH11343078387855SANTHERA PHARMACEUTICALS (DEUTSCHLAND) GMBHChemicalChemicalVAMOROLONESUB188638H02ABGLUCOCORTICOIDS4False1TrueEU/3/14/1309mg milligram(s)240mg/kg milligram(s)/kilogram6False483GlucocorticoidsPRD1102226811ORAL USETrueAGAMREE 40 mg/ml oral suspensionvamoroloneORAL SUSPENSIONFalse2363554135141351136382014Authorised2025-01-082025-01-08FalseGreece2025-04-15Authorised599022025-07-012025-07-02599032025-07-012025-07-02815032025-07-042025-07-07815042025-07-042025-07-07869212025-07-042025-09-092025-09-10869222025-07-042025-09-092025-09-10True888582025-07-012025-07-022025-07-042025-09-09False5872141351Under evaluation2024-10-25T11:46:23.3347488241351Authorised2025-01-28T11:48:29.6293Greeceacceptable2025-10-13authorized2025-04-152025-04-1541604043637798675735Hospital/Clinic/Other health care facility8Nosokomeio Paidon I Agia SofiaFalseTrueORG-100040867Active690777Thivon PapadiamantopoulouThivon PapadiamantopoulouAthens115 272014Greece3021320213656mkatsalouli@hotmail.comTrueORG-1000408671783623MarinaKatsalouli3021320213656mkatsalouli@hotmail.comNeurological DepartmentAuthorised2521064135341353136382029Authorised2025-01-272025-01-27FalseNetherlands2025-02-03Authorised540172025-03-262025-04-02540182025-03-262025-04-02734192025-04-102025-04-10734202025-04-102025-04-10831212025-04-102025-06-102025-07-22831222025-04-102025-06-102025-07-22True774722025-03-262025-04-022025-04-102025-06-10False7546141353Authorised2025-02-03T09:43:33.6616953641353Under evaluation2024-11-18T11:06:59.3363Netherlandsacceptable2025-10-29authorized_conditions2025-02-032025-02-0361718997652377690385Hospital/Clinic/Other health care facility8Radboud universitair medisch centrum StichtingFalseTrueORG-100023234Active708355Geert Grooteplein Zuid 10Geert Grooteplein Zuid 10Nijmegen6525 GA2029Netherlands+31243611111saskia.houwen@radboudumc.nlTrueORG-1000232341910830SaskiaHouwen+31243611111saskia.houwen@radboudumc.nl1Rehabilitation1718996652371690379Hospital/Clinic/Other health care facility8Leids Universitair Medisch Centrum (LUMC)FalseTrueORG-100014145Active708348Albinusdreef 2Albinusdreef 2Leiden2333 ZA2029Netherlands+31715262111e.niks@lumc.nlTrueORG-1000141451910829ErikNiks+31715262111e.niks@lumc.nl1NeurologyAuthorised2363603318633186136382008Authorised2024-09-162024-09-16FalseunwillingCzechia2024-10-08Authorised456062024-12-052024-12-11455652024-12-052024-12-11612082024-12-112024-12-17612112024-12-112024-12-17612122024-12-112024-12-17831272024-12-112025-03-122025-07-22831282024-12-112025-03-122025-07-22True623972024-12-052024-12-112024-12-112025-03-12False5588633186Authorised2024-10-08T15:35:28.2514092033186Under evaluation2024-06-27T16:29:35.8251Czechiaacceptable2025-09-16authorized_conditions2024-10-082024-10-0871604089485896518316Hospital/Clinic/Other health care facility8Fakultni Nemocnice V MotoleFalseTrueORG-100012719Active521229V Uvalu 84/1, MotolV Uvalu 84/1MotolPrague150 002008Czechia420224433301Jana.haberlova@fnmotol.czTrueORG-1000127191783675JanaHaberlova420224433301Jana.haberlova@fnmotol.cz1Paediatric Neurology1604088485813518233Hospital/Clinic/Other health care facility8Fakultni Nemocnice BrnoFalseTrueORG-100022981Active521144Cernopolni 9, Cerna PoleCernopolni 9Cerna PoleBrno-Sever613 002008Czechia420532234934jurikova.lenka@fnbrno.czTrueORG-1000229811783674LenkaJurikova420532234934jurikova.lenka@fnbrno.cz1Paediatric NeurologyAuthorised2487613318533185136382002Authorised2024-09-162024-09-16TrueunwillingBelgium2024-10-04Authorised482872025-01-142025-01-17482862025-01-142025-01-17666672025-02-052025-02-07666682025-02-052025-02-07666692025-02-052025-02-07831292025-02-052025-03-062025-07-22831302025-02-052025-03-062025-07-22True670502025-01-142025-01-172025-02-052025-03-06False4091933185Under evaluation2024-06-27T16:29:35.7785533633185Authorised2024-10-04T16:39:21.2561Belgiumacceptable2025-10-23authorized_conditions2024-10-042024-10-0451694105498799531251Educational Institution1Universiteit GentTrueTrueORG-100022735Active535411Corneel Heymanslaan 10Corneel Heymanslaan 10Gent90002002Belgium0473966619nicolas.deconinck@huderf.beTrueORG-1000227351883355NicolasDeconinck0473966619nicolas.deconinck@huderf.be1Neuromusculair referentiecentrum1694104485912518332Hospital/Clinic/Other health care facility8UZ LeuvenFalseTrueORG-100006001Active521246Herestraat 49Herestraat 49Leuven30002002Belgium3216343827Liesbeth.dewaele@uzleuven.beTrueORG-1000060011883354LiesbethDe Waele3216343827Liesbeth.dewaele@uzleuven.be1Pediatric NeurologyAuthorised2363623318833188136382027Authorised2024-09-062024-09-06FalseunwillingSpain2024-10-07Authorised474382025-01-072025-01-08474392025-01-072025-01-08661222025-02-032025-02-04661232025-02-032025-02-04831252025-02-032025-02-192025-07-22831262025-02-032025-02-192025-07-22True655212025-01-072025-01-082025-02-032025-02-19False4092133188Under evaluation2024-06-27T16:29:35.8645544333188Authorised2024-10-07T10:53:22.341Spainacceptable2025-09-30authorized_conditions2024-10-072024-10-0731604098485801518221Hospital/Clinic/Other health care facility8Hospital Universitario Puerta De Hierro De MajadahondaFalseTrueORG-100028532Active521130Calle De Joaquin Rodrigo 2Calle De Joaquin Rodrigo 2Majadahonda282222027Spain34911916437gemma.iglesias@salud.madrid.orgTrueORG-1000285321783688Gemma IglesiasEscalera34911916437gemma.iglesias@salud.madrid.org1Pediatrics and Neonatology1604099485785518205Hospital/Clinic/Other health care facility8Hospital Universitario Y Politecnico La FeFalseTrueORG-100029610Active521109Avenida De Fernando Abril Martorell 106Avenida De Fernando Abril Martorell 106Valencia460262027Spain34687450013pitarch_inmcas@gva.esTrueORG-1000296101783689Immaculada PitarchCastellano34687450013pitarch_inmcas@gva.es1PediatricsAuthorised2586064135241352136382017Authorised2025-01-212025-01-21FalseIreland2025-02-21Authorised583922025-05-132025-06-11583932025-05-132025-06-11831232025-05-232025-05-232025-07-22831242025-05-232025-05-232025-07-22791832025-05-232025-06-11791842025-05-232025-06-11True860832025-05-132025-06-112025-05-232025-05-23False6314141352Under evaluation2024-11-11T16:43:53.1677697841352Authorised2025-02-21T13:36:10.0823Irelandacceptable2025-12-22authorized_conditions2025-02-212025-02-2111766404639384677331Hospital/Clinic/Other health care facility8Children's Health IrelandFalseTrueORG-100022831Active692759Belgard Square North, TallaghtBelgard Square NorthTallaghtDublin 24D24 TN3C2017Ireland00353014142671Denise.Mcdonald@tuh.ieTrueORG-1000228311963445DeniseMcDonald00353014142671Denise.Mcdonald@tuh.ie1Department of Paediatric Neurodisability1790063874605916598Hospital/Clinic/Other health care facility8Children's Health IrelandFalseTrueORG-100022831Active975537Temple StreetTemple StreetDublin 1D01 YC672017Ireland00353014142671denise.mcdonald@tuh.ieTrueORG-1000228311989620DeniseMcDonald00353014142671denise.mcdonald@tuh.ie1Department of Paediatric NeurodisabilityAuthorised22200INITIALAuthorised2024-512828-12-00Authorised2024-06-27acceptable_conditions2024-10-041376973318533185Belgium2002acceptable2024-10-02authorized_conditions2024-10-04AuthorisedBelgiumAuthorised2024-10-04Authorised1377283318633186Czechia2008acceptable2024-10-04authorized_conditions2024-10-08AuthorisedCzechiaAuthorised2024-10-08Authorised1351063318833188Spain2027acceptable2024-10-07authorized_conditions2024-10-07AuthorisedSpainAuthorised2024-10-07Authorised2024-10-0433185BelgiumAuthorised33186CzechiaAuthorised33188SpainAuthorisedINFalse409662220033186Czechia2024-10-08T15:35:28.77authorized_conditionsacceptabledecision13772855080INITIALFalse405772220033188Spain2024-10-07T10:53:22.844authorized_conditionsacceptabledecision13510655080INITIALFalse404962220033185Belgium2024-10-04T16:39:21.686authorized_conditionsacceptabledecision13769755080INITIALTruea0ffd57d-de34-44b1-b3ea-8cce4f82ee1d17277094597fe1-2d27-403e-9fd0-5d19898db112172770ed0e6a3e-bb89-4ee4-9c16-0e2a2b8968ed1727700bf47c8a-1340-435e-b83b-831f6c92339517277038415SUBSEQUENT 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individual skeletal muscles of five non-ambulant patients affected by Duchenne Muscular Dystrophy: a non randomized, open-label, phase I/IIa studyDuchenne Muscular DystrophyItaly:501/10/2024IT: 01/10/2024Diseases [C] - Musculoskeletal Diseases [C05]Ospedale San Raffaele S.r.l., University Of ManchesterHospital/Clinic/Other health care facility, Educational InstitutionPhase I and Phase II (Integrated)- First administration to humansDMD06-MAB60-17 yearsMale1EEA5Safety 1. Incidence and severity of local (foot) and systemic adverse events (any grade) in DMD patients treated with intra- muscular injections of genetically corrected auto-MABS for one year from the injection., Efficacy 2.Presence of dystrophin (≥ to 1:10 dilution of a control muscle for WB; ≥ 10% of gene expression of skipped dystrophin at ddPCR; ≥ 10% of dystrophin positive fibers for IF respect to a control muscle) on muscle biopsy of the EDB muscle transplanted with genetically corrected auto-MABS, in at least two out of the three assays (Quantitative IF , ddPCR, and Western Blot) at two months after injection.No20/12/202424/04/20252024-10-01T00:00:002025-04-24T21:29:11.0452967765Italy323402024-10-01T00:00:002024-12-20T19:26:36.316532340ItalySTART_OF_TRIAL2024-06-27START_OF_RECRUITMENT2024-06-27END_OF_RECRUITMENT2024-12-202024-514860-14-005Cell-mediated skipping of exon 51 for the genetic correction of dystrophin, based upon a single injection of autologous mesoangioblasts (MABs) in individual skeletal muscles of five non-ambulant patients affected by Duchenne Muscular Dystrophy: a non randomized, open-label, phase I/IIa study27/06/2024Duchenne Muscular DystrophyItaly:501/10/2024IT: 01/10/2024Diseases [C] - Musculoskeletal Diseases [C05]Ospedale San Raffaele S.r.l., University Of ManchesterHospital/Clinic/Other health care facility, Educational InstitutionPhase I and Phase II (Integrated)- First administration to humansDMD06-MAB60-17 yearsMale15Safety 1. Incidence and severity of local (foot) and systemic adverse events (any grade) in DMD patients treated with intra- muscular injections of genetically corrected auto-MABS for one year from the injection., Efficacy 2.Presence of dystrophin (≥ to 1:10 dilution of a control muscle for WB; ≥ 10% of gene expression of skipped dystrophin at ddPCR; ≥ 10% of dystrophin positive fibers for IF respect to a control muscle) on muscle biopsy of the EDB muscle transplanted with genetically corrected auto-MABS, in at least two out of the three assays (Quantitative IF , ddPCR, and Western Blot) at two months after injection.No20/12/202424/04/20252024-514860-14-00Authorised2024-06-272024-10-01T00:00:002025-04-24T21:29:11.0452967765685180279512111487538SUSPENSION FOR INJECTIONPRD114875381DMD06-MABSUSPENSION FOR INJECTIONDMD06-MABSUB396896OSPEDALE SAN RAFFAELE S.R.L.11487538396899OSPEDALE SAN RAFFAELE S.R.L.Structurally Diverse Substance - Cell therapyStructurally Diverse Substance - Cell therapyDMD06-MABSUB396896False1FalseATMPPRD114875383196Not applicable31DystrophinTrans-Skip lentiviral vector genetically modified autologous MAB cellsTrueFalseMAB cells311INTRAMUSCULAR USEFalseDMD06-MABdmd06-mabSUSPENSION FOR INJECTIONCell-mediated skipping of exon 51 for the genetic correction of dystrophin, based upon a single injection of autologous mesoangioblasts (MABs) in individual skeletal muscles of five non-ambulant patients affected by Duchenne Muscular Dystrophy: a non randomized, open-label, phase I/IIa studyCell-mediated skipping of exon 51 for the genetic correction of dystrophin, based upon a single injection of autologous mesoangioblasts (MABs) in individual skeletal muscles of five non-ambulant patients affected by Duchenne Muscular Dystrophy: a non randomized, open-label, phase I/IIa studyFalse71Phase I/II6021669092Duchenne Muscular DystrophyFalse41988245198825We will test safety of treatment and efficacy in restoring synthesis of dystrophin in the injected muscle. Safety 1. To assess the incidence of adverse events in DMD patients treated with intra-muscular foot injections of auto-MABS after genetic correction with a lentiviral vector expressing a small nuclear RNA engineered to skip exon 51 of the dystrophin gene. Note: In vitro/in vivo pre-clinical safety assessments will be performed on ATIMP prior to injection, as described in the IMPD. Efficacy 2. To determine dystrophin expression (>= 10% of a healthy subject) after a single intra- muscular injection into the foot medial Extensor Digitorum Brevis (EDB) of genetically corrected auto-MABS.4356981Age between >= 12 and <18 years at time of study entry, provided that participants matching the eligibility criteria can be identified.4356992Non-ambulant at the time of recruitment.4357003Confirmed diagnosis of DMD with documented exon 51 skippable mutations in dystrophin gene.4357014Progression of muscle degeneration =< to 50% reduction of muscle mass as determined by quantitative MRI (grade 2: Kinali et al. 2011).4357025Written informed consent of caregivers of DMD patients and patient’s assent.7504471Positive hepatitis B surface antigen, hepatitis C antibody test, or Human immunodeficiency virus (HIV) test, TPHA test.75044810Presence of severe scoliosis (curve >50°).75044911Presence of significant impairment of renal or hepatic function.7504502Presence of immune deficiency, neoplastic or autoimmune disease (based on clinical history).7504513Bleeding disorder.7504524Any known allergies to products likely to be used in the study.7504535Prior or ongoing medical condition (e.g. concomitant illness, psychiatric condition, behavioural disorder, drug abuse), medical history, physical findings, or laboratory abnormality that, in the investigator’s opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow up would be completed, or could impair the assessment of study results.7504546Ongoing participation in any other therapeutic clinical trial or treatment with exon skipping oligonucleotides. Use of steroids is considered standard care and therefore permitted.7504557LVEF (Left Ventricle Ejection Fraction) < 45% of a healthy subject or ECG finding significant for underlying cardiac impairment.7504568Pulmonary function tests assessed by spirometry (if cooperative) of FEV1 and FVC <30% of the predicted values. If unable, pulse oximetry < 95 % in room air.7504579Change of medication related to DMD within last 3 months with the exception of adjustment based on weight gain of current medications.5067891Safety 1. Incidence and severity of local (foot) and systemic adverse events (any grade) in DMD patients treated with intra- muscular injections of genetically corrected auto-MABS for one year from the injection.True5067902Efficacy 2.Presence of dystrophin (≥ to 1:10 dilution of a control muscle for WB; ≥ 10% of gene expression of skipped dystrophin at ddPCR; ≥ 10% of dystrophin positive fibers for IF respect to a control muscle) on muscle biopsy of the EDB muscle transplanted with genetically corrected auto-MABS, in at least two out of the three assays (Quantitative IF , ddPCR, and Western Blot) at two months after injection.True2025-12-312024-06-271852042218520526262PatientsFalseTrueFalse2acceptable2024-12-202024-12-205Diseases [C] - Musculoskeletal Diseases [C05]69092Duchenne Muscular DystrophyFalse73522True219574PublicStefano Previtaliprevitali.stefano@hsr.it0226432813495951Hospital/Clinic/Other health care facility8Ospedale San Raffaele S.r.l.FalseTrueORG-100006123Active219575ScientificStefano Previtaliprevitali.stefano@hsr.it0226432813495951Hospital/Clinic/Other health care facility8Ospedale San Raffaele S.r.l.FalseTrueORG-100006123ActiveTrueTrueTrue495951Hospital/Clinic/Other health care facility8Ospedale San Raffaele S.r.l.FalseTrueORG-100006123Active463630495951Hospital/Clinic/Other health care facility8Ospedale San Raffaele S.r.l.FalseTrueORG-100006123Active497010Via Olgettina 60Via Olgettina 60Milan201322018ItalyTrueORG-100006123FalseNon-Commercial73523False219576PublicMohammed Zubairclincialtrials@manchester.ac.uk01612752725534812Educational Institution1University Of ManchesterTrueTrueORG-100016549ActiveTrueTrue534812Educational Institution1University Of ManchesterTrueTrueORG-100016549Active502336534812Educational Institution1University Of ManchesterTrueTrueORG-100016549Active5392802nd Floor, Christie Building, Oxford Road2nd FloorChristie BuildingOxford RoadManchesterM13 9PL826United KingdomTrueORG-100016549FalseNon-Commercial1Phase I/II7332905Diseases [C] - Musculoskeletal Diseases [C05]214681DMD06-MAB1Test279512111487538SUSPENSION FOR INJECTIONPRD114875381DMD06-MABSUSPENSION FOR INJECTIONDMD06-MABSUB396896OSPEDALE SAN RAFFAELE S.R.L.11487538396899OSPEDALE SAN RAFFAELE S.R.L.Structurally Diverse Substance - Cell therapyStructurally Diverse Substance - Cell therapyDMD06-MABSUB396896False1FalseATMPPRD114875383196Not applicable31DystrophinTrans-Skip lentiviral vector genetically modified autologous MAB cellsTrueFalseMAB cells311INTRAMUSCULAR USEFalseDMD06-MABdmd06-mabSUSPENSION FOR INJECTIONFalse1683483234032340157662018Authorised2024-05-302024-05-30TrueItaly2024-10-01Authorised516912024-06-272025-03-04516922024-06-272025-03-04691082024-06-272024-12-202025-03-04691092024-06-272024-12-202025-03-04691052024-06-272025-03-04691062024-06-272025-03-04691072024-06-272024-12-202025-03-04True730452024-06-272025-03-042024-06-272024-12-20False5163232340Under evaluation2024-09-09T15:31:02.195481732340Authorised2024-10-01T23:59:591Italyacceptable2024-12-20authorized2024-10-012024-10-0151089149469209501541Hospital/Clinic/Other health care facility8Ospedale San Raffaele S.r.l.FalseTrueORG-100006123Active503068Via Olgettina 60Via Olgettina 60Milan201322018Italy0226432813previtali.stefano@hsr.itTrueORG-1000061231217119StefanoPrevitali0226432813previtali.stefano@hsr.itPediatriaAuthorised27115INITIALAuthorised2024-514860-14-00Authorised2024-09-09acceptable2024-09-241045013234032340Italy2018acceptable2024-09-26authorized2024-10-01AuthorisedItalyAuthorised2024-10-01Authorised2024-10-0132340ItalyAuthorisedINFalse401002711532340Italy2024-10-01T00:00:00authorizedacceptabledecision10450140754INITIALTrue41939SUBSTANTIAL MODIFICATIONAuthorised2024-514860-14-00Authorised2024-11-25acceptable2024-12-201683483234032340Italy2018acceptable2024-12-20authorized2024-10-01AuthorisedItalyAuthorised2024-10-01Authorised2024-12-2032340ItalyAuthorisedSM-1PART_I_AND_PART_IIFalse520744193932340Italy2024-12-20T19:26:36.316authorizedacceptabledecision16834868518SUBSTANTIAL MODIFICATIONTrueItaly323402024-10-01T00:00:002024-12-20T19:26:36.3165True2023-000148-4732340ItalySTART_OF_TRIAL2024-06-27START_OF_RECRUITMENT2024-06-27END_OF_RECRUITMENT2024-12-20EEA1 2024-512626-28-006HaltedA Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)SRP-9001-303Duchenne Muscular DystrophyGermany:7Belgium:6Sweden:6France:7Italy:6Spain:612/09/2024FR: 17/09/2024, BE: 12/09/2024, DE: 17/09/2024, SE: 16/09/2024, ES: 15/10/2024, IT: 30/09/2024Diseases [C] - Musculoskeletal Diseases [C05]Sarepta Therapeutics Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72, Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72, Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot, Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72, Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE), Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72, Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72, Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72.Saline, 0.9% sodium chloride solution IV infusion marketed authorized will be used as the placebo and will be supplied by the site. the SmPC previously authorized are Country specific. for Belgium, Spain and Sweden per local requirements under CTDs no SmPCs were submitted., Delandistrogene moxeparvovec-rokl5618-64 years, 0-17 yearsMale3Both95Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72No18/08/202519/08/20252024-09-12T15:25:29.6032025-08-19T03:32:57.6145738776K1_Recruitment Arrangements Participant Fact Sheet French Public97f8a6ef-3a42-45a9-8b66-24351d6d6fcb14Recruitment arrangements (for publication)PDF1969551.01K1_Recruitment Arrangements Dear Patient Letter French Public619d90a6-a6ff-436d-957a-5de4a4c7a7bc14Recruitment arrangements (for publication)PDF1969552.01K1_Recruitment Flyer Study Information Sheet French Public97d1cac0-984a-4ec4-bce8-e4575bb7ebc314Recruitment arrangements (for publication)PDF1969551.01K1_Recruitment 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consent form (for publication)PDF2142676.01Germany284472024-09-17T10:49:18.5512025-08-13T14:30:52.3117Belgium284452024-09-12T15:25:29.6032025-08-11T09:34:55.6096Sweden284502024-09-16T19:38:40.7882025-08-13T13:53:42.9336France284462024-09-17T10:51:18.6462025-08-18T12:41:56.0897Italy284482024-09-30T00:00:002025-08-12T20:01:48.8386Spain284492024-10-15T17:46:30.8692025-08-14T09:43:41.284628448TH-771732025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.Subject safety reasonsTrue2025-03-31T18:43:06The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3128448ItalyIT28445TH-771742025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulatorsSubject safety reasonsTrue2025-03-31T18:50:02The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3128445BelgiumBE28448TH-513172024-08-232024-11-301Sponsor decisionTrueFalseTrueThe Sponsor, Sarepta Therapeutics, temporarily paused recruitment for the ENVISION study in Italy and Spain on 23Aug2024 due to a pause in AAVrh74 Antibody ELISA sample testing by their Diagnostic Device Partner, Quest Diagnostics, in their clinical performance study (DM-SC-IVD1-PRE 44/2023, CIV-ID: CIV-SE-23-02-042372, no. 44/2023). This pause is not related to changes in the SRP-9001 benefit-risk profile or safety concerns with SRP-9001 or with the Quest ELISA assay. Quest Diagnostics has submitted substantial modifications to Quest’s clinical performance study to the relevant Competent Authorities and Ethics Committees in these EU Countries to inform of an increase in the number of samples Quest plans to test using the AAVrh74 Antibody ELISA. This increase in the number of samples is intended to align with the current screening and enrollment estimates Sarepta has for the SRP-9001-303 ENVISION study in each member state. Sarepta would like to clarify that the total sample size of 148 for the ENVISION study remains unchanged. SRP-9001 is a one-time infusion. To date, 57 subjects have been infused globally, including 12 subjects in Italy and 15 subjects in Spain. Recruitment is ongoing in other countries including Belgium, Japan, the United Kingdom, Australia, Germany, and Taiwan.False2024-10-14T11:12:06Not applicable as the pause in recruitment is not due to any safety concerns or changes to the benefit-risk profile.True2024-10-1428448ItalyIT28450TH-771712025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.Subject safety reasonsTrue2025-03-31T18:36:06The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3128450SwedenSE28449TH-771722025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.Subject safety reasonsTrue2025-03-31T18:38:33The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3128449SpainES28449TH-521282024-08-232024-11-301Sponsor decisionTrueFalseTrueThe Sponsor, Sarepta Therapeutics, temporarily paused recruitment for the ENVISION study in Italy and Spain on 23Aug2024 due to a pause in AAVrh74 Antibody ELISA sample testing by their Diagnostic Device Partner, Quest Diagnostics, in their clinical performance study (DM-SC-IVD1-PRE 44/2023, CIV-ID: CIV-SE-23-02-042372, no. 44/2023). This pause is not related to changes in the SRP-9001 benefit-risk profile or safety concerns with SRP-9001 or with the Quest ELISA assay. Quest Diagnostics has submitted substantial modifications to Quest’s clinical performance study to the relevant Competent Authorities and Ethics Committees in these EU Countries to inform of an increase in the number of samples Quest plans to test using the AAVrh74 Antibody ELISA. This increase in the number of samples is intended to align with the current screening and enrollment estimates Sarepta has for the SRP-9001-303 ENVISION study in each member state. Sarepta would like to clarify that the total sample size of 148 for the ENVISION study remains unchanged. SRP-9001 is a one-time infusion. To date, 57 subjects have been infused globally, including 12 subjects in Italy and 15 subjects in Spain. Recruitment is ongoing in other countries including Belgium, Japan, the United Kingdom, Australia, Germany, and Taiwan.False2024-10-18T14:40:30Not applicable as the pause in recruitment is not due to any safety concerns or changes to the benefit-risk profile.True2024-10-1828449SpainES28445BelgiumSTART_OF_TRIAL2024-03-28START_OF_RECRUITMENT2024-04-09END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3128446France28447GermanySTART_OF_TRIAL2024-09-09START_OF_RECRUITMENT2024-10-0828448ItalyRESTART_OF_TRIAL2024-12-04START_OF_TRIAL2024-03-01START_OF_RECRUITMENT2024-03-05RESTART_OF_RECRUITMENT2025-02-04END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3128449SpainRESTART_OF_TRIAL2024-11-26START_OF_TRIAL2024-02-12START_OF_RECRUITMENT2024-02-19RESTART_OF_RECRUITMENT2024-12-19END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3128450SwedenSTART_OF_TRIAL2024-10-16START_OF_RECRUITMENT2024-11-04END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-312024-512626-28-006A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)SRP-9001-30328/03/2024Duchenne Muscular DystrophyGermany:7Belgium:6Sweden:6France:7Italy:6Spain:612/09/2024FR: 17/09/2024, BE: 12/09/2024, DE: 17/09/2024, SE: 16/09/2024, ES: 15/10/2024, IT: 30/09/2024Diseases [C] - Musculoskeletal Diseases [C05]Sarepta Therapeutics Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72, Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72, Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot, Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72, Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE), Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72, Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72, Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72.Saline, 0.9% sodium chloride solution IV infusion marketed authorized will be used as the placebo and will be supplied by the site. the SmPC previously authorized are Country specific. for Belgium, Spain and Sweden per local requirements under CTDs no SmPCs were submitted., Delandistrogene moxeparvovec-rokl5618-64 years, 0-17 yearsMale395Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72No18/08/202519/08/20252024-512626-28-00Halted2024-03-282024-09-12T15:25:29.6032025-08-19T03:32:57.61457387768816153100000000439Korea, Republic of410KRKORTrue100000000429Israel376ILISRTrue100000000420Hong Kong344HKHKGTrue100000000557United States840USUSATrue100000000329Australia36AUAUSTrue100000000538Taiwan158TWTWNTrue100000000432Japan392JPJPNTrue100000000354Canada124CACANTrue100000000556United Kingdom826GBGBRTrue3619681N/AN/AN/ASaline, 0.9% sodium chloride solution IV infusion marketed authorized will be used as the placebo and will be supplied by the site. the SmPC previously authorized are Country specific. for Belgium, Spain and Sweden per local requirements under CTDs no SmPCs were submitted.N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalseSaline, 0.9% sodium chloride solution IV infusion marketed authorized will be used as the placebo and will be supplied by the site. the SmPC previously authorized are Country specific. for Belgium, Spain and Sweden per local requirements under CTDs no SmPCs were submitted.n/aN/A361969110281347SOLUTION FOR INJECTION/INFUSIONPRD86568511Delandistrogene moxeparvovec-roklSOLUTION FOR INJECTION/INFUSIONSRP-9001DELANDISTROGENE MOXEPARVOVECSUB197789SAREPTA THERAPEUTICS INC10281347235539SAREPTA THERAPEUTICS INCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherDELANDISTROGENE MOXEPARVOVECSUB197789True1TrueEU/3/20/2250vector genomes (vg)/mL13300000000000vector genomes (vg)/mL1330000000000011PRD8656851SRP-9001Catalent Belgium 1779 IMP; Sarepta Ireland IMP13160/00001; Catalent Germany 2021_0095/DE_BW_014352EMA/CAT/113473/20193delandistrogene moxeparvovec dystrophinAAVFalseTrue38INTRAVENOUS USEFalseDelandistrogene moxeparvovec-rokldelandistrogene moxeparvovecSOLUTION FOR INJECTION/INFUSIONA Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)6754190380828ea-3052-46b3-85ac-f0f37a26afb6Étude de phase 3, internationale, randomisée, en double aveugle, contrôlée contre placebo, de thérapie génique systémique, destinée à évaluer la sécurité d'emploi et l'efficacité du SRP9001 chez des patients non ambulatoires et ambulatoires atteints de dystrophie musculaire de Duchenne (ENVISION)34French (France)6754191380828ea-3052-46b3-85ac-f0f37a26afb6Estudio de fase 3, multinacional, aleatorizado, en doble ciego y controlado con placebo, del tratamiento mediante transferencia génica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos, no ambulatorios y ambulatorios, con distrofia muscular de Duchenne (ENVISION)7SpanishA Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)6754207509cc773-50dd-49ec-bb3c-1e23853a50ceEstudio para evaluar la seguridad y la eficacia del sistema de terapia de transferencia génica delandistrogén moxeparvovec (SRP-9001) en participantes en situación ambulatoria y no ambulatoria con distrofia muscular de Duchenne (DMD) (ENVISION)7Spanish6754206509cc773-50dd-49ec-bb3c-1e23853a50ceÉtude de thérapie par transfert de gène visant à évaluer la sécurité d’emploi et l’efficacité du délandistrogène moxéparvovec (SRP-9001) chez des participants ambulatoires et non ambulatoires atteints de dystrophie musculaire de Duchenne (DMD) (ENVISION)34French (France)SRP-9001-303390421NCT05881408390422MOH_2024-04-17_01331ISR Registry Identifier6False52This is a phase 3 clinical trial falling under Category 27923690459Duchenne Muscular Dystrophy6754162840c8a12-580f-42cc-b6d3-6b50264b1f8fDystrophie musculaire de Duchenne34French (France)6754163840c8a12-580f-42cc-b6d3-6b50264b1f8fDistrofia muscular de Duchenne7SpanishTrue10000001204720.1PTDuchenne muscular dystrophy gene carrier10052655100000004850False10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False326183152618304261829Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 7267542040d25734f-9edb-4504-b12d-01602f563d41Parte 1: cambio desde el inicio en la puntuación total de funcionamiento de las extremidades superiores (PUL) (versión 2.0) en la semana 727Spanish67542050d25734f-9edb-4504-b12d-01602f563d41Partie 1 : Variation du score de performance des membres supérieurs (PUL) (version 2.0) entre la référence et la Semaine 7234French (France)2989541Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 726754208d2c000f3-c858-4f02-a3ba-3890411c568cParte 1: cambio desde el inicio en el porcentaje previsto de la capacidad vital forzada (CVF) en la semana 727Spanish6754209d2c000f3-c858-4f02-a3ba-3890411c568cPartie 1 : Variation entre la référence et la semaine 72 du pourcentage de la capacité vitale forcée (CVF) prévue34French (France)2989552Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72675421090402987-7ae0-4a78-a40f-d67533f807d9Parte 1: cambio desde el inicio en el porcentaje previsto de flujo espiratorio máximo (FEM) en la semana 727Spanish675421190402987-7ae0-4a78-a40f-d67533f807d9Partie 1 : Variation entre la référence et la semaine 72 du pourcentage du débit expiratoire de pointe (DEP) prévu34French (France)2989563Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot675421388f63c59-eef7-432b-9fa2-b249a11884baParte 1: cantidad expresada de delandistrogén moxeparvovec distrofina en la semana 12, medida mediante inmunoelectrotransferencia7Spanish675421288f63c59-eef7-432b-9fa2-b249a11884baPartie 1 : Quantité d’expression de la dystrophine dans le délandistrogène moxéparvovec à la semaine 12, mesurée par Western Blot34French (France)2989574Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 726754215fb6baacc-a1b8-4c76-9089-071c72e599b6Partie 1 : Variation du score PROMIS (information sur la mesure des résultats rapportés par les patients) de la fonction des membres supérieurs entre la référence et la semaine 7234French (France)6754214fb6baacc-a1b8-4c76-9089-071c72e599b6Parte 1: cambio desde el inicio en la puntuación del funcionamiento de las extremidades superiores del sistema de información de medición de resultados notificados por el paciente (PROMIS) en la semana 727Spanish2989585Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE), Baseline up to Week 1246754216dd0fe1ed-086e-46ee-a635-d93a70b1ab70Nombre de participants présentant un événement indésirable émergeant du traitement (EIET), un événement indésirable d’intérêt particulier (EIIP) et un événement indésirable grave (EIG), de la référence jusqu’à la semaine 12434French (France)6754217dd0fe1ed-086e-46ee-a635-d93a70b1ab70Número de participantes con algún acontecimiento adverso surgido durante el tratamiento (AAST), acontecimiento adverso de interés especial (AAIE) y acontecimiento adverso grave (AAG) desde el inicio hasta la semana 1247Spanish2989596Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 726754218359ccf38-e40d-454c-bd40-69fd83e22682Parte 1 (solo en relación con la cohorte 2): cambio desde el inicio en la puntuación total de la evaluación ambulatoria North Star (NSAA) en la semana 727Spanish6754219359ccf38-e40d-454c-bd40-69fd83e22682Partie 1 (cohorte 2 uniquement) : Variation du score total de l’évaluation ambulatoire North Star (North Star Ambulatory Assessment, NSAA) entre la référence et la Semaine 7234French (France)2989607Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 7267542215973ec64-a071-4e82-b6ac-279aa18765baPartie 1 : Variation de la souche circonférentielle globale mesurée par IRM cardiaque entre la référence et la Semaine 7234French (France)67542205973ec64-a071-4e82-b6ac-279aa18765baParte 1: cambio desde el inicio en la deformación circunferencial global medida mediante RM cardíaca en la semana 727Spanish2989618Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72675422376efcd89-509e-4c3a-a9b9-f3432b155421Partie 1: Changement par rapport à la ligne de base du score du domaine moyen de la PUL (version 2.0) à la semaine 7234French (France)675422276efcd89-509e-4c3a-a9b9-f3432b155421Parte 1: Cambio en la puntuación por dominio medio de PUL (V2.0) desde el Basal a las 72 semanas.7Spanish5767591Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion in exons 1 to 17 and/or exons 59 to 71.675419304f93479-5443-4fca-a5a2-d3d87be4b7faDiagnóstico definitivo de DMD basado en hallazgos clínicos documentados y pruebas genéticas previas. Una mutación patogénica del marco de lectura (frameshift) o un codón de terminación prematuro en el gen DMD, excepto cualquier deleción en los exones 1 a 17 y/o 59 a 71.7Spanish675419204f93479-5443-4fca-a5a2-d3d87be4b7faDiagnostic définitif de la DMD basé sur des résultats cliniques documentés et des tests génétiques antérieurs. Une mutation pathogène de type frameshift ou un codon stop prématuré dans le gène de la DMD, à l’exception de toute délétion dans les exons 1 à 17 et/ou 59 à 71.34French (France)5767602Cohort 1 only: Non-ambulatory per protocol specified criteria.67541945c3d945c-e83c-433e-9ae0-5dd99ff2c470Solo cohorte 1: ausencia de capacidad ambulatoria según los criterios especificados en el protocolo.7Spanish67541955c3d945c-e83c-433e-9ae0-5dd99ff2c470Cohorte 1 uniquement : Non ambulatoire selon les critères spécifiés per protocole.34French (France)5767613Cohort 2 only: Ambulatory per protocol specified criteria and ≥8 to <18 years of age at the time of Screening.6754197ff2bb1ab-0aee-40a4-8fde-cdefdcfa35e1Cohorte 2 uniquement : Patients ambulatoires selon les critères spécifiés per protocole et âgés de ≥ 8 à < 18 ans au moment de la sélection.34French (France)6754196ff2bb1ab-0aee-40a4-8fde-cdefdcfa35e1Solo cohorte 2: capacidad ambulatoria según los criterios especificados en el protocolo y edad comprendida entre ≥8 y <18 años en el momento de la selección.7Spanish5767624Ability to cooperate with motor assessment testing.675419972c9c41a-329d-4a8a-acf8-0925ace68b89Capacité de coopérer en ce qui concerne les analyses d’évaluation du moteur.34French (France)675419872c9c41a-329d-4a8a-acf8-0925ace68b89Capacidad de cooperar con las pruebas de evaluación motora.7Spanish5767635Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).6754201db5909da-feb3-4cc4-9e2b-ac535e66a2d0Dose quotidienne stable de corticoïdes oraux pendant au moins 12 semaines avant la sélection et la dose devrait rester constante tout au long de l’étude (sauf pour les modifications potentielles afin de s’adapter aux variations du poids).34French (France)6754200db5909da-feb3-4cc4-9e2b-ac535e66a2d0Dosis diaria estable de corticoesteroides por vía oral durante al menos 12 semanas antes de la selección; se espera que dicha dosis permanezca constante (exceptuando posibles modificaciones necesarias para adaptarse a cambios de peso) durante todo el estudio.7Spanish5767646Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.6754203f2d2d72e-a1bf-4a90-8bbf-b624437ca16aLos títulos de anticuerpos contra el virus adenoasociado recombinante de serotipo rh74 (rAAVrh74) no son elevados según los requisitos especificados en el protocolo.7Spanish6754202f2d2d72e-a1bf-4a90-8bbf-b624437ca16aLes titres d’anticorps recombinants de sérotype rh74 (rAAVrh74) du virus adéno-associé ne sont pas élevés conformément aux exigences spécifiées per protocol.34French (France)9919141Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.6754183b57bcad5-8731-415f-b1e8-f09f18c3f5d8Exposición a terapias génicas, medicamentos en investigación o cualquier otro tratamiento diseñado para aumentar la expresión de distrofina dentro de los intervalos de tiempo especificados en el protocolo.7Spanish6754182b57bcad5-8731-415f-b1e8-f09f18c3f5d8Exposition à la thérapie génique, au médicament expérimental ou à tout traitement conçu pour augmenter l’expression de la dystrophine dans les délais spécifiés par le protocole.34French (France)9919152Abnormality in protocol-specified diagnostic evaluations or laboratory tests.675418513984a68-995d-40ca-b124-3ad70d884bf0Anomalías en las evaluaciones diagnósticas o las pruebas analíticas especificadas en el protocolo.7Spanish675418413984a68-995d-40ca-b124-3ad70d884bf0Anomalie dans les évaluations de diagnostic ou les analyses de laboratoire spécifiées dans le protocole.34French (France)9919163Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.6754187a0ad4b5a-d9e7-455b-96d0-e3cf5cdb1d0dPrésence d’une autre maladie cliniquement significative, d’un état pathologique ou d’un besoin de traitement médicamenteux chronique qui, de l’avis de l’investigateur, crée des risques inutiles pour le transfert de gènes.34French (France)6754186a0ad4b5a-d9e7-455b-96d0-e3cf5cdb1d0dPresencia de cualquier otra enfermedad clínicamente significativa, patología o necesidad de tratamiento farmacológico crónico que, en opinión del investigador, crea riesgos innecesarios cuando se usa un sistema de transferencia de genes.7Spanish9919174Other inclusion or exclusion criteria could apply.6754188ec8daa74-3fc2-4a1d-ac1c-cc5005475a70Podrían aplicarse otros criterios de inclusión o exclusión.7Spanish6754189ec8daa74-3fc2-4a1d-ac1c-cc5005475a70D’autres critères d’inclusion ou d’exclusion pourraient s’appliquer.34French (France)6730731Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72True6754165a51dcead-259c-4068-9170-e71857fba91bParte 1: cambio desde el inicio en la puntuación total de funcionamiento de las extremidades superiores (PUL) (versión 2.0) en la semana 727Spanish6754164a51dcead-259c-4068-9170-e71857fba91bPartie 1 : Variation du score de performance des membres supérieurs (PUL) (version 2.0) entre la référence et la Semaine 7234French (France)6730741Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72False675416675557e91-1b38-48a4-9a4e-3981c4a158d8Partie 1 : Variation entre la référence et la semaine 72 du pourcentage de la capacité vitale forcée (CVF) prévue34French (France)675416775557e91-1b38-48a4-9a4e-3981c4a158d8Parte 1: cambio desde el inicio en el porcentaje previsto de la capacidad vital forzada (CVF) en la semana 727Spanish6730752Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72False6754168ff1218e6-a59b-4d2a-8a0c-a1953fe24254Parte 1: cambio desde el inicio en el porcentaje previsto de flujo espiratorio máximo (FEM) en la semana 727Spanish6754169ff1218e6-a59b-4d2a-8a0c-a1953fe24254Partie 1 : Variation entre la référence et la semaine 72 du pourcentage du débit expiratoire de pointe (DEP) prévu34French (France)6730763Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western BlotFalse675417005974abd-9c36-4c2b-bc99-ed097a878014Partie 1 : Quantité d’expression de la dystrophine dans le délandistrogène moxéparvovec à la semaine 12, mesurée par Western Blot34French (France)675417105974abd-9c36-4c2b-bc99-ed097a878014Parte 1: cantidad expresada de delandistrogén moxeparvovec distrofina en la semana 12, medida mediante inmunoelectrotransferencia7Spanish6730774Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72False67541738db88753-ef05-4c67-a93c-5dd08ef2ee47Partie 1 : Variation du score PROMIS (information sur la mesure des résultats rapportés par les patients) de la fonction des membres supérieurs entre la référence et la semaine 7234French (France)67541728db88753-ef05-4c67-a93c-5dd08ef2ee47Parte 1: cambio desde el inicio en la puntuación del funcionamiento de las extremidades superiores del sistema de información de medición de resultados notificados por el paciente (PROMIS) en la semana 727Spanish6730785Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)False67541754ccf0feb-f979-4df5-9458-63ecf3a49dafNúmero de participantes con algún acontecimiento adverso surgido durante el tratamiento (AAST), acontecimiento adverso de interés especial (AAIE) y acontecimiento adverso grave (AAG)7Spanish67541744ccf0feb-f979-4df5-9458-63ecf3a49dafNombre de participants présentant des événements indésirables émergeants du traitement (EIET), des événements indésirables d’intérêt particulier (EIIP) et des événements indésirables grave (EIG)34French (France)6730796Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72False67541772b2f4f45-12ed-4a49-8014-38cfd8aa5dcfParte 1 (solo en relación con la cohorte 2): cambio desde el inicio en la puntuación total de la evaluación ambulatoria North Star (NSAA) en la semana 727Spanish67541762b2f4f45-12ed-4a49-8014-38cfd8aa5dcfPartie 1 (cohorte 2 uniquement) : Variation du score total de l’évaluation ambulatoire North Star (North Star Ambulatory Assessment, NSAA) entre la référence et la Semaine 7234French (France)6730807Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72False6754179b721b43b-6194-414c-9b01-2e38a587a892Parte 1: cambio desde el inicio en la deformación circunferencial global medida mediante RM cardíaca en la semana 727Spanish6754178b721b43b-6194-414c-9b01-2e38a587a892Partie 1 : Variation de la souche circonférentielle globale mesurée par IRM cardiaque entre la référence et la Semaine 7234French (France)6730818Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72.False6754180bb350684-d32f-4168-8b78-1b1101662708Parte 1: Cambio en la puntuación por dominio medio de PUL (V2.0) desde el Basal a las 72 semanas.7Spanish6754181bb350684-d32f-4168-8b78-1b1101662708Partie 1 : Changement par rapport à la ligne de base du score du domaine moyen de la PUL (version 2.0) à la semaine 72.34French (France)2028-06-302028-06-292023-05-2263431Sarepta Therapeutics, Inc.2402003324020122240202255224020326622PatientsFalseTrueTrue235252446643478916EEA National Competent authority16Federal Agency For Medicines And Health ProductsFalseTrueORG-100003913Active478404Galileelaan 5/03Galileelaan 5/03Sint-Joost-Ten-Node12102002BelgiumTrueORG-1000039137520EMEA-002677-PIP01-19221622022-000691-19A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy, Estudio abierto, de dos partes, de administración génica sistémica para evaluar la seguridad y la expresión de RO7494222 (SRP-9001) en sujetos menores de cuatro años con Distrofia Muscular de Duchenne, Uno studio in due parti, in aperto, sulla terapia genica sistemica per valutare la sicurezza e l’espressione di RO7494222 (SRP-9001) in soggetti di età inferiore a quattro anni affetti da distrofia muscolare di Duchenne, Etude en 2 parties, en ouvert, de transfert de gène par voir systémique, évaluant la sécurité et l'expression de RO7494222 (SRP-9001) chez l'enfant âgé de moins de quatre ans présentant une dystrophie musculaire de Duchenne OPTIONALOPTIONALFalse221632019-003374-91A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) , Étude de phase 3, multinationale, randomisée, en double aveugle, contrôlée par placebo, sur le transfert systémique de gènes, visant à évaluer la sécurité d’emploi et l’efficacité du SRP-9001 chez des sujets atteints de dystrophie musculaire de Duchenne (EMBARK), Estudio de fase III multinacional, aleatorizado, doble ciego, controlado con placebo, de liberación génica sistémica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos con distrofia muscular de Duchenne (EMBARK), Studio di fase 3, internazionale, randomizzato, in doppio cieco, controllato verso placebo, sul rilascio sistemico di geni, per valutare la sicurezza e l’efficacia di SRP-9001 in soggetti con distrofia muscolare di Duchenne (EMBARK)OPTIONALOPTIONALFalse221642023-505043-39-00Sarepta Therapeutics Inc.A Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical StudyNON_APPLICABLENON_APPLICABLE5370Falseacceptable_conditions2025-08-112025-08-115Diseases [C] - Musculoskeletal Diseases [C05]90459Duchenne Muscular DystrophyTrue96605True288546PublicPatient RecruitmentSareptAlly@sarepta.com0018887273782402440Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active288547ScientificSarepta Therapeutics Inc.SareptAlly@sarepta.com0018887273782402440Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active421223421054452938Pharmaceutical company10Parexel International (IRL) LimitedTrueTrueORG-100022780Active45040570 Sir John Rogerson's Quay70 Sir John Rogerson's QuayDublin 2D02 R2962017Ireland0035314739500Clinicaltrial.Enquiries@parexel.comTrueORG-100022780660165166016612660167136601681466016915Site selection, Trial supplies and logistics, Study and Vendor management and Patient recruitment and retention6601703660171466017256601736660174766017580035314739500Clinicaltrial.Enquiries@parexel.com402440Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active372839402440Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active398230215 1st Street215 1st StreetCambridge02142-1213840United StatesTrueORG-100006645TrueCommercial2This is a phase 3 clinical trial falling under Category 28783575Diseases [C] - Musculoskeletal Diseases [C05]274257Saline, 0.9% sodium chloride solution IV infusion marketed authorized will be used as the placebo and will be supplied by the site. the SmPC previously authorized are Country specific. for Belgium, Spain and Sweden per local requirements under CTDs no SmPCs were submitted.3Placebo3619681N/AN/AN/ASaline, 0.9% sodium chloride solution IV infusion marketed authorized will be used as the placebo and will be supplied by the site. the SmPC previously authorized are Country specific. for Belgium, Spain and Sweden per local requirements under CTDs no SmPCs were submitted.N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalseSaline, 0.9% sodium chloride solution IV infusion marketed authorized will be used as the placebo and will be supplied by the site. the SmPC previously authorized are Country specific. for Belgium, Spain and Sweden per local requirements under CTDs no SmPCs were submitted.n/aN/A274258Delandistrogene moxeparvovec-roklCatalent Belgium 1779 IMP; Sarepta Ireland IMP13160/00001; Catalent Germany 2021_0095/DE_BW_011Test361969110281347SOLUTION FOR INJECTION/INFUSIONPRD86568511Delandistrogene moxeparvovec-roklSOLUTION FOR INJECTION/INFUSIONSRP-9001DELANDISTROGENE MOXEPARVOVECSUB197789SAREPTA THERAPEUTICS INC10281347235539SAREPTA THERAPEUTICS INCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherDELANDISTROGENE MOXEPARVOVECSUB197789True1TrueEU/3/20/2250vector genomes (vg)/mL13300000000000vector genomes (vg)/mL1330000000000011PRD8656851SRP-9001Catalent Belgium 1779 IMP; Sarepta Ireland IMP13160/00001; Catalent Germany 2021_0095/DE_BW_014352EMA/CAT/113473/20193delandistrogene moxeparvovec dystrophinAAVFalseTrue38INTRAVENOUS USEFalseDelandistrogene moxeparvovec-rokldelandistrogene moxeparvovecSOLUTION FOR INJECTION/INFUSIONFalse1969552844628446134322012Suspended2024-09-042024-09-04FalseunwillingFrance2024-09-17SuspendedFalseFalse5271728446Authorised2024-09-17T10:51:17.884776528446Under evaluation2024-08-09T20:08:25.7747987228446Suspended2025-04-02T17:27:25.4141France2025-08-26acceptable_conditions2025-04-04authorized_conditions2024-09-172024-09-17101295454546762579384Hospital/Clinic/Other health care facility8Hopital Necker Enfants MaladesFalseTrueORG-100023257Active588343149 Rue De Sevres149 Rue De SevresParis750152012France0033144494856isabelle.desguerre@aphp.frTrueORG-1000232571444213IsabelleDesguerre0033144494856isabelle.desguerre@aphp.fr1service de Neuropédiatrie1295456546764579386Hospital/Clinic/Other health care facility8Hospices Civils De LyonFalseTrueORG-100006597Active58834659 Boulevard Pinel59 Boulevard PinelBron695002012France0033472129450carole.vuillerot@chu-lyon.frTrueORG-1000065971444215CaroleVuillerot0033472129450carole.vuillerot@chu-lyon.fr1service de Neuropédiatrie1295455546760579382Hospital/Clinic/Other health care facility8Hopital Des EnfantsFalseTrueORG-100026949Active588341330 Avenue De Grande Bretagne330 Avenue De Grande BretagneToulouse Cedex 9310592012France0033561323562cances.c@chu-toulouse.frTrueORG-1000269491444214ClaudeCancès0033561323562cances.c@chu-toulouse.fr1service de NeuropédiatrieAuthorised2142642844528445134322002Halted2024-09-042024-09-04FalsewillingBelgium2024-09-12Halted376552024-03-282024-10-09376562024-03-282024-10-09492692024-04-092024-10-10492702024-04-092024-10-10722782024-04-092025-03-312025-03-31722792024-04-092025-03-312025-03-31True505462024-03-282024-10-092024-04-092025-03-31True5214728445Authorised2024-09-12T15:25:29.0964776428445Under evaluation2024-08-09T20:08:25.7577968928445Halted2025-03-31T18:50:02.9371Belgium2025-08-26acceptable2025-07-29authorized_conditions2024-09-122024-09-12101428086760411799081Hospital/Clinic/Other health care facility8Centre Hospitalier Regional De La CitadelleFalseTrueORG-100028257Active837997Boulevard Du Douzieme De Ligne 1Boulevard Du Douzieme De Ligne 1Liege40002002Belgium+3243218515aurore.daron@citadelle.beTrueORG-1000282571590316AuroreDaron+3243218515aurore.daron@citadelle.be1446: Laboratoire1428087553983586621Hospital/Clinic/Other health care facility8Universitair Ziekenhuis GentFalseTrueORG-100021542Active596313Corneel Heymanslaan 10Corneel Heymanslaan 10Gent90002002Belgium+0473966619nicolas.deconinck@huderf.beTrueORG-1000215421590317NicolasDeconinck+0473966619nicolas.deconinck@huderf.be2406: Neuromuscular Reference Centre (NMRC)Authorised2142662844728447134322013Suspended2024-09-042024-09-04FalseunwillingGermany2024-09-17Suspended376572024-09-092024-10-09376582024-09-092024-10-09492842024-10-082024-10-10492852024-10-082024-10-10True505472024-09-092024-10-092024-10-08False5271628447Authorised2024-09-17T10:49:17.2324776628447Under evaluation2024-08-09T20:08:25.7887968428447Suspended2025-03-31T18:19:16.0941Germany2025-08-26acceptable2025-07-01authorized_conditions2024-09-172024-09-17151428094760770799443Hospital/Clinic/Other health care facility8Universitaetsklinikum Heidelberg AöRFalseTrueORG-100013733Active838458Im Neuenheimer Feld 430, NeuenheimIm Neuenheimer Feld 430NeuenheimHeidelberg691202013Germany496221564837andreas.ziegler@med.uni-heidelberg.deTrueORG-1000137331590324AndreasZiegler496221564837andreas.ziegler@med.uni-heidelberg.de1470: Zentrum für Kinder und Jugendmedizin1428091547282579906Hospital/Clinic/Other health care facility8University Medical Center Hamburg-EppendorfFalseTrueORG-100008810Active588938Martinistrasse 52, EppendorfMartinistrasse 52EppendorfHamburg202462013Germany004940741055512j.johannsen@uke.deTrueORG-1000088101590321JessikaJohannsen004940741055512j.johannsen@uke.de1462: Nephrology and Rheumatology1428095760762799435Hospital/Clinic/Other health care facility8Charite Universitaetsmedizin Berlin KöRFalseTrueORG-100008480Active838450Augustenburger Platz 1, WeddingAugustenburger Platz 1WeddingBerlin133532013Germany+4930450566279claudia.weiss@charite.deTrueORG-1000084801590325ClaudiaWeiss+4930450566279claudia.weiss@charite.de1448: Department of Neuropaediatrics and Center for Chronically Sick Children1428093547280579904Hospital/Clinic/Other health care facility8Klinikum der Universitaet Muenchen AöRFalseTrueORG-100008479Active588936Lindwurmstrasse 4, Ludwigsvorstadt-IsarvorstadtLindwurmstrasse 4Ludwigsvorstadt-IsarvorstadtMunich803372013Germany4940741055512astrid.blaschek@med.uni-muenchen.deTrueORG-1000084791590323AstridBlaschek4940741055512astrid.blaschek@med.uni-muenchen.de1412: NAP1428092547276579900Hospital/Clinic/Other health care facility8Universitaetsklinikum Essen AöRFalseTrueORG-100009964Active588932Hufelandstrasse 55, HolsterhausenHufelandstrasse 55HolsterhausenEssen451472013Germany004920172383652andrea.gangfuss@uk-essen.deTrueORG-1000099641590322AndreaGangfuss004920172383652andrea.gangfuss@uk-essen.de1409: Klinik für Kinderheilkunde IAuthorised2142692845028450134322028Halted2024-09-042024-09-04FalseunwillingSweden2024-09-16Halted394082024-10-162024-10-22394092024-10-162024-10-22537992024-11-042024-11-05538002024-11-042024-11-05722722024-11-042025-03-312025-03-31722732024-11-042025-03-312025-03-31True530582024-10-162024-10-222024-11-042025-03-31False5266128450Authorised2024-09-16T19:38:40.3134776928450Under evaluation2024-08-09T20:08:25.837968528450Halted2025-03-31T18:36:06.4791Sweden2025-08-26acceptable2025-06-26authorized_conditions2024-09-162024-09-16151428103556423589071Hospital/Clinic/Other health care facility8Karolinska University HospitalFalseTrueORG-100000573Active599055Eugeniavagen 3Eugeniavagen 3Solna171 642028Sweden46851777342thomas.sejersen@ki.seTrueORG-1000005731590333ThomasSejersen46851777342thomas.sejersen@ki.se2NAP1428104556425589073Hospital/Clinic/Other health care facility8Queen Silvia Childrens Hospital - Sahlgrenska University Hospital - Vaestra GoetalandsregionenFalseTrueORG-100031777Active599057Behandlingsvagen 7, HarlandaBehandlingsvagen 7HarlandaGothenburg416 502028Sweden46313434780mar.tulinius@gu.seTrueORG-1000317771590334MarTulinius46313434780mar.tulinius@gu.se2NAPAuthorised2142682844928449134322027Halted2024-09-042024-09-04TrueunwillingSpain2024-10-15Halted387332024-02-122024-10-17387342024-02-122024-10-17451002024-11-262024-12-06451012024-11-262024-12-06511992024-02-192024-08-232024-10-18512002024-02-192024-08-232024-10-18506892024-02-192024-10-17506902024-02-192024-10-17636032024-12-192025-01-13636042024-12-192025-01-13722742024-12-192025-03-312025-03-31722752024-12-192025-03-312025-03-31True521232024-02-122024-10-172024-02-192025-03-31False4776828449Under evaluation2024-08-09T20:08:25.8155734528449Authorised2024-10-15T17:46:30.3995786928449Halted2024-10-18T14:40:30.1177968628449Halted2025-03-31T18:38:33.6377150228449Authorised2024-12-06T07:37:13.3431Spain2025-08-262024-12-19acceptable2025-06-12authorized_conditions2024-10-152024-10-15251428102760040798708Hospital/Clinic/Other health care facility8Hospital Universitario 12 De OctubreFalseTrueORG-100028548Active837555Avenida De Cordoba SnAvenida De Cordoba SnMadrid280412027Spain34913908835acamacho@salud.madrid.orgTrueORG-1000285481590332AnaCamacho Salas34913908835acamacho@salud.madrid.org1Neurología /Sección de Neurología Pediátrica1428100546655579277Hospital/Clinic/Other health care facility8Hospital Sant Joan De Deu BarcelonaFalseTrueORG-100023083Active588224Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain0034932532100andres.nascimento@sjd.esTrueORG-1000230831590330AndresNascimento Osorio0034932532100andres.nascimento@sjd.es1Neuropediatría1428101546653579275Hospital/Clinic/Other health care facility8Hospital Universitario Y Politecnico La FeFalseTrueORG-100029610Active588222Avenida De Fernando Abril Martorell 106Avenida De Fernando Abril Martorell 106Valencia460262027Spain0034961244153muelas_nur@gva.esTrueORG-1000296101590331NuriaMuelas Gómez0034961244153muelas_nur@gva.es1NeurologíaAuthorised2142672844828448134322018Halted2024-09-042024-09-04Falseunwilling_tacitItaly2024-09-30Halted376592024-03-012024-10-09376602024-03-012024-10-09453912024-12-042024-12-10453922024-12-042024-12-10499182024-03-052024-08-232024-10-14499192024-03-052024-08-232024-10-14493002024-03-052024-10-10493012024-03-052024-10-10665242025-02-042025-02-06665252025-02-042025-02-06722762025-02-042025-03-312025-03-31722772025-02-042025-03-312025-03-31True505482024-03-012024-10-092024-03-052025-03-31False5676528448Halted2024-10-14T11:12:06.7025461828448Authorised2024-09-30T23:59:594776728448Under 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MODIFICATIONTrue850305499028446France2025-08-18T12:41:56.089not_authorizedLe promoteur a souhaité ne pas répondre pour l’instant aux considérations émises par le CPP, ce qui n’est plus compatible à ce jour avec les jalons du processus d’évaluation de la partie II.not_acceptabledecision22883388161SUBSTANTIAL MODIFICATIONFalse846515499028450Sweden2025-08-13T13:53:42.933authorized_conditionsacceptabledecision21426988161SUBSTANTIAL MODIFICATIONFalse846685499028447Germany2025-08-13T14:30:52.311authorized_conditionsacceptabledecision21426688161SUBSTANTIAL MODIFICATIONFalse847385499028449Spain2025-08-14T09:43:41.284authorized_conditionsacceptabledecision21426888161SUBSTANTIAL MODIFICATIONFalse845465499028448Italy2025-08-12T20:01:48.838authorized_conditionsacceptabledecision21426788161SUBSTANTIAL MODIFICATIONFalseGermany284472024-09-17T10:49:18.5512025-08-13T14:30:52.3117Belgium284452024-09-12T15:25:29.6032025-08-11T09:34:55.6096Sweden284502024-09-16T19:38:40.7882025-08-13T13:53:42.9336France284462024-09-17T10:51:18.6462025-08-18T12:41:56.0897Italy284482024-09-30T00:00:002025-08-12T20:01:48.8386Spain284492024-10-15T17:46:30.8692025-08-14T09:43:41.2846True2020-002372-1328448TH-771732025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.Subject safety reasonsTrue2025-03-31T18:43:06The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3128448ItalyIT28445TH-771742025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulatorsSubject safety reasonsTrue2025-03-31T18:50:02The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3128445BelgiumBE28448TH-513172024-08-232024-11-301Sponsor decisionTrueFalseTrueThe Sponsor, Sarepta Therapeutics, temporarily paused recruitment for the ENVISION study in Italy and Spain on 23Aug2024 due to a pause in AAVrh74 Antibody ELISA sample testing by their Diagnostic Device Partner, Quest Diagnostics, in their clinical performance study (DM-SC-IVD1-PRE 44/2023, CIV-ID: CIV-SE-23-02-042372, no. 44/2023). This pause is not related to changes in the SRP-9001 benefit-risk profile or safety concerns with SRP-9001 or with the Quest ELISA assay. Quest Diagnostics has submitted substantial modifications to Quest’s clinical performance study to the relevant Competent Authorities and Ethics Committees in these EU Countries to inform of an increase in the number of samples Quest plans to test using the AAVrh74 Antibody ELISA. This increase in the number of samples is intended to align with the current screening and enrollment estimates Sarepta has for the SRP-9001-303 ENVISION study in each member state. Sarepta would like to clarify that the total sample size of 148 for the ENVISION study remains unchanged. SRP-9001 is a one-time infusion. To date, 57 subjects have been infused globally, including 12 subjects in Italy and 15 subjects in Spain. Recruitment is ongoing in other countries including Belgium, Japan, the United Kingdom, Australia, Germany, and Taiwan.False2024-10-14T11:12:06Not applicable as the pause in recruitment is not due to any safety concerns or changes to the benefit-risk profile.True2024-10-1428448ItalyIT28450TH-771712025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.Subject safety reasonsTrue2025-03-31T18:36:06The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3128450SwedenSE28449TH-771722025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.Subject safety reasonsTrue2025-03-31T18:38:33The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3128449SpainES28449TH-521282024-08-232024-11-301Sponsor decisionTrueFalseTrueThe Sponsor, Sarepta Therapeutics, temporarily paused recruitment for the ENVISION study in Italy and Spain on 23Aug2024 due to a pause in AAVrh74 Antibody ELISA sample testing by their Diagnostic Device Partner, Quest Diagnostics, in their clinical performance study (DM-SC-IVD1-PRE 44/2023, CIV-ID: CIV-SE-23-02-042372, no. 44/2023). This pause is not related to changes in the SRP-9001 benefit-risk profile or safety concerns with SRP-9001 or with the Quest ELISA assay. Quest Diagnostics has submitted substantial modifications to Quest’s clinical performance study to the relevant Competent Authorities and Ethics Committees in these EU Countries to inform of an increase in the number of samples Quest plans to test using the AAVrh74 Antibody ELISA. This increase in the number of samples is intended to align with the current screening and enrollment estimates Sarepta has for the SRP-9001-303 ENVISION study in each member state. Sarepta would like to clarify that the total sample size of 148 for the ENVISION study remains unchanged. SRP-9001 is a one-time infusion. To date, 57 subjects have been infused globally, including 12 subjects in Italy and 15 subjects in Spain. Recruitment is ongoing in other countries including Belgium, Japan, the United Kingdom, Australia, Germany, and Taiwan.False2024-10-18T14:40:30Not applicable as the pause in recruitment is not due to any safety concerns or changes to the benefit-risk profile.True2024-10-1828449SpainES28445BelgiumSTART_OF_TRIAL2024-03-28START_OF_RECRUITMENT2024-04-09END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3128446France28447GermanySTART_OF_TRIAL2024-09-09START_OF_RECRUITMENT2024-10-0828448ItalyRESTART_OF_TRIAL2024-12-04START_OF_TRIAL2024-03-01START_OF_RECRUITMENT2024-03-05RESTART_OF_RECRUITMENT2025-02-04END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3128449SpainRESTART_OF_TRIAL2024-11-26START_OF_TRIAL2024-02-12START_OF_RECRUITMENT2024-02-19RESTART_OF_RECRUITMENT2024-12-19END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3128450SwedenSTART_OF_TRIAL2024-10-16START_OF_RECRUITMENT2024-11-04END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-31K1_Recruitment Arrangements Participant Fact Sheet 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Public5a163e98-9733-4346-bdf4-bcd029b5157815Subject information and informed consent form (for publication)PDF2142677.01L1_Country Main ICF Adult English Publice9efde44-541e-47bb-981c-72dbea903bb415Subject information and informed consent form (for publication)PDF2142677.01L1_Country ICF Main Study Parent Englishbce62cb0-70e3-49d8-bda5-a8435ab35c9a15Subject information and informed consent form (for publication)PDF2142677.01L1_Country ICF Assent Child 13-17 English Public392d6755-791b-477b-a7ed-34138e03c8af15Subject information and informed consent form (for publication)PDF2142676.01Both32121CM-DE-000157True28447GermanyGermany2025-03-312131CM-FR-00015After receipt of the Dear Investigator Letter: “Delandistrogene moxeparvovec: fatal case of acute liver failure and intracranial hemorrhage” dated 18 Mar 2025, we ask the Sponsor to immediately put on hold enrollment in France, until analysis of the fatal case has been finalised by the RMSs. The temporay halt was already notified in the other european countries concerned, except France as the clinical trial had not started. The sponsor should confirm that the enrollment, is put on hold in France. Furthermore, the Sponsor has to notify this fatal case through the EU portal without undue delay. Indeed and according to the article 53 of the REGULATION (EU) No 536/2014 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC, the sponsor shall notify the Member States concerned through the EU portal of all unexpected events which affect the benefit-risk balance of the clinical trial, but are not suspected unexpected serious adverse reactions as referred to in Article 42. That notification shall be made without undue delay but no later than 15 days from the date the sponsor became aware of this event.True28446FranceFrance2025-04-02 2023-504520-26-004AuthorisedOpen label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.DSC/14/2357/51Duchenne Muscular Dystrophy (DMD)Germany:4Netherlands:4Spain:4France:4Belgium:4Italy:409/09/2024NL: 10/09/2024, BE: 09/09/2024, ES: 09/09/2024, DE: 09/09/2024, FR: 17/09/2024, IT: 09/10/2024Diseases [C] - Musculoskeletal Diseases [C05]Italfarmaco S.p.A.Pharmaceutical companyTherapeutic confirmatory (Phase III)For ambulant patients: Change from baseline in physical function as measured by 6MWT, NSAA, Time function tests (e.g. time to rise from floor, time to climb 4-stairs, time to 10m walk) (week 48 and then yearly till the end of the study), For ambulant patients: Change from baseline in muscle strength (e.g. knee extension and elbow flexion) as measured by HHM (week 48 and then yearly till the end of the study), For non-ambulant patients: Change in physical function from baseline in the Egen Klassifikation (EK) score (week 48 and then yearly till the end of the study), For non-ambulant patients: Change in patient and/or parent/caregiver reports of activities of daily living as measured by Barthel Index (week 48 and then yearly till the end of the study), For non-ambulant patients: Change in upper limbs muscle strength (week 48 and then yearly till the end of the study) evaluated by handheld myometry (HHM), For all patients: Change from baseline in physical function as measured by the Performance of Upper Limb (PUL) and MFM (week 48 and then yearly till the end of the study), For all patients: Change from baseline in respiratory function (week 48 and then yearly till the end of the study) (e.g. FVC, FEV1, PEF), For all patients: Change in patient and/or parent/caregiver reports of quality of life as 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publication)PDF22422711Germany334392024-09-09T08:30:26.3582025-06-20T09:07:25.7684Netherlands334422024-09-10T08:14:12.8332025-03-10T13:59:58.5114Spain334412024-09-09T08:18:37.2762025-03-12T11:48:08.5434France334382024-09-17T12:02:24.2022025-07-03T10:50:10.8634Belgium334372024-09-09T13:14:53.5472025-03-13T14:26:13.6884Italy334402024-10-09T00:00:002025-07-11T11:27:49.085433437BelgiumSTART_OF_TRIAL2019-06-24START_OF_RECRUITMENT2019-07-2233438FranceSTART_OF_TRIAL2020-01-20START_OF_RECRUITMENT2020-02-1733439GermanySTART_OF_TRIAL2019-12-09START_OF_RECRUITMENT2019-12-1233440ItalySTART_OF_TRIAL2017-10-19START_OF_RECRUITMENT2017-10-2533441SpainSTART_OF_TRIAL2019-06-03START_OF_RECRUITMENT2019-06-0633442NetherlandsSTART_OF_TRIAL2019-10-31START_OF_RECRUITMENT2020-01-072023-504520-26-004Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.DSC/14/2357/5119/10/2017Duchenne Muscular Dystrophy (DMD)Germany:4Netherlands:4Spain:4France:4Belgium:4Italy:409/09/2024NL: 10/09/2024, BE: 09/09/2024, ES: 09/09/2024, DE: 09/09/2024, FR: 17/09/2024, IT: 09/10/2024Diseases [C] - Musculoskeletal Diseases [C05]Italfarmaco S.p.A.Pharmaceutical companyTherapeutic confirmatory (Phase III)For ambulant patients: Change from baseline in physical function as measured by 6MWT, NSAA, Time function tests (e.g. time to rise from floor, time to climb 4-stairs, time to 10m walk) (week 48 and then yearly till the end of the study), For ambulant patients: Change from baseline in muscle strength (e.g. knee extension and elbow flexion) as measured by HHM (week 48 and then yearly till the end of the study), For non-ambulant patients: Change in physical function from baseline in the Egen Klassifikation (EK) score (week 48 and then yearly till the end of the study), For non-ambulant patients: Change in patient and/or parent/caregiver reports of activities of daily living as measured by Barthel Index (week 48 and then yearly till the end of the study), For non-ambulant patients: Change in upper limbs muscle strength (week 48 and then yearly till the end of the study) evaluated by handheld myometry (HHM), For all patients: Change from baseline in physical function as measured by the Performance of Upper Limb (PUL) and MFM (week 48 and then yearly till the end of the study), For all patients: Change from baseline in respiratory function (week 48 and then yearly till the end of the study) (e.g. FVC, FEV1, PEF), For all patients: Change in patient and/or parent/caregiver reports of quality of life as measured by PedsQL for paediatric patients and by SF-36 for adults patients (week 48 and then yearly till the end of the study), For all patients: Age to major disease milestones (e.g. age at loss of ambulation, age at respiratory support needed during the day, age at scoliosis surgery, age at death)ITF2357650-17 years, 18-64 yearsMale3120Type, incidence, and severity of treatment related/not related AEs and SAEsNo11/07/202512/07/20252023-504520-26-00Authorised2017-10-192024-09-09T08:18:37.2762025-07-12T03:35:17.885642217492661102100000000518Serbia688RSSRBTrue100000000429Israel376ILISRTrue100000000557United States840USUSATrue100000000354Canada124CACANTrue100000000556United Kingdom826GBGBRTrue381032111133213ORAL SUSPENSIONPRD47976781ITF2357ORAL SUSPENSIONITF2357GIVINOSTATSUB189959GIVINOSTATITALFARMACO SPA11133213337329ITALFARMACO SPAChemicalChemicalGIVINOSTATSUB189959False1TrueEU/3/12/1009mg milligram(s)140mg milligram(s)0011ChemicalPRD4797678ITF235711ORAL USETrueITF2357givinostatORAL SUSPENSIONOpen label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.7196004052a1424-3974-4684-9c3a-1abb0fa8b5d1Étude en ouvert sur la sécurité d’emploi, la tolérance et l’efficacité à long terme du GIVINOSTAT chez tous les patients atteints de DMD qui ont déjà été traités dans l’une des études sur le GIVINOSTAT10French7196005052a1424-3974-4684-9c3a-1abb0fa8b5d1Estudio en abierto para evaluar la seguridad, la tolerabilidad y la eficacia a largo plazo de GIVINOSTAT en todos los pacientes con DMD que han sido tratados previamente en uno de los estudios de GIVINOSTAT7Spanish7196006052a1424-3974-4684-9c3a-1abb0fa8b5d1Open-label lange termijn onderzoek naar de veiligheid, verdraagbaarheid en werkzaamheid van GIVINOSTAT bij alle DMD-patiënten die al eens zijn behandeld tijdens een van de GIVINOSTAT-onderzoeken37Dutch (Netherlands)Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past71960234d05e17d-d690-482c-b75d-a27903592f0dEstudio en el que todos los pacientes toman el mismo fármaco en investigación con el objetivo de evaluar la seguridad, la tolerabilidad y la eficacia a largo plazo de GIVINOSTAT en todos los pacientes con distrofia muscular de Duchenne que ya han sido tratados en uno de los estudios de GIVINOSTAT en el pasado.7Spanish71960224d05e17d-d690-482c-b75d-a27903592f0dÉtude dans laquelle tous les patients prennent le même médicament expérimental dans le but d’évaluer la sécurité d’emploi, la tolérance et l’efficacité à long terme du GIVINOSTAT chez tous les patients atteints de dystrophie musculaire de Duchenne qui ont déjà été traités dans l’une des études sur le GIVINOSTAT par le passé10French71960244d05e17d-d690-482c-b75d-a27903592f0dOnderzoek waarin alle patiënten hetzelfde onderzoeksmiddel ontvangen met als doel het evalueren van de langetermijnveiligheid, -verdraagbaarheid en -werkzaamheid van GIVINOSTAT bij alle patiënten met Duchenne spierdystrofie die in het verleden al in een van de GIVINOSTAT-onderzoeken zijn behandeld37Dutch (Netherlands)DSC/14/2357/51False52This is a phase III study to investigate long-term safety, tolerability, and efficacy of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.8358895355Duchenne Muscular Dystrophy (DMD)7195932a7e0c782-8b90-4bf7-aede-33869d742f6fDystrophie musculaire de Duchenne (DMD)10French7195933a7e0c782-8b90-4bf7-aede-33869d742f6fDuchenne spierdystrofie (DMD)37Dutch (Netherlands)7195934a7e0c782-8b90-4bf7-aede-33869d742f6fDistrofia muscular de Duchenne (DMD)7SpanishTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False5276197427619913Tollerability276198To assess the long-term safety and tolerability of GIVINOSTAT in patients with DMD following core protocols program and with naïve GIVINOSTAT DMD subjects, i.e. subjects screened in study DSC/14/2357/48 who met: - all the inclusion criteria and none of the exclusion criteria, and - never been randomized because the enrollment in the off-target group was completed.7196020292d7db0-8e45-49bd-939c-31d4b9a53d5bÉvaluer la sécurité d’emploi et la tolérance à long terme du GIVINOSTAT chez des patients atteints de DMD après un programme de protocoles de base et chez des patients atteints de DMD naïfs de GIVINOSTAT, c’est-à-dire des patients sélectionnés dans l’étude DSC/14/2357/48 qui : - ont répondu à la totalité des critères d’inclusion et n’ont répondu à aucun des critères d’exclusion, et - n’ont jamais été randomisés, car l’inclusion dans la cohorte hors cible était terminée.10French7196021292d7db0-8e45-49bd-939c-31d4b9a53d5bHet beoordelen van de langetermijnveiligheid en -verdraagbaarheid van GIVINOSTAT bij patiënten met DMD die het kernprotocolprogramma volgen en bij proefpersonen met DMD die niet met GIVINOSTAT zijn behandeld, d.w.z. proefpersonen die zijn gescreend in onderzoek DSC/14/2357/48 en die voldeden aan: - alle inclusiecriteria en geen van de exclusiecriteria; en - nooit zijn gerandomiseerd, omdat de inschrijving in de off-target groep voltooid was.37Dutch (Netherlands)7196019292d7db0-8e45-49bd-939c-31d4b9a53d5bEvaluar la seguridad y la tolerabilidad a largo plazo de GIVINOSTAT en pacientes con DMD que siguen el programa de los protocolos principales y con participantes con DMD sin tratamiento previo con GIVINOSTAT, es decir, participantes seleccionados en el estudio DSC/14/2357/48 que cumplieron lo siguiente: — cumplieron todos los criterios de inclusión y ninguno de los criterios de exclusión, y — nunca habían sido aleatorizados porque se había completado la inclusión en el grupo no objetivo.7Spanish3163691To evaluate the effects of long-term administration of GIVINOSTAT on muscular function and strength;7196027ad8f6886-5827-4370-bb54-45666340d2a8Evaluar los efectos de la administración a largo plazo de GIVINOSTAT sobre la función y la fuerza muscular7Spanish7196025ad8f6886-5827-4370-bb54-45666340d2a8Het evalueren van de effecten van langetermijntoediening van GIVINOSTAT op de spierfunctie en de spierkracht37Dutch (Netherlands)7196026ad8f6886-5827-4370-bb54-45666340d2a8Évaluer les effets de l’administration à long terme du GIVINOSTAT sur la fonction et la force musculaires10French3163702To evaluate the effects of long-term administration of GIVINOSTAT on respiratory function71960288de26b6c-316c-428d-8d1e-bbb2e69d40d3Het evalueren van de effecten van langetermijntoediening van GIVINOSTAT op de ademhalingsfunctie.37Dutch (Netherlands)71960298de26b6c-316c-428d-8d1e-bbb2e69d40d3Evaluar los efectos de la administración a largo plazo de GIVINOSTAT sobre la función respiratoria.7Spanish71960308de26b6c-316c-428d-8d1e-bbb2e69d40d3Évaluer les effets de l’administration à long terme du GIVINOSTAT sur la fonction respiratoire10French3163713To evaluate the impact on daily activities and quality of life following long-term administration of GIVINOSTAT.7196031ce4c4e1b-96b1-4cff-8867-7f1762397896Évaluer l’impact sur les activités quotidiennes et la qualité de vie après l’administration à long terme du GIVINOSTAT10French7196033ce4c4e1b-96b1-4cff-8867-7f1762397896Evaluar el impacto en las actividades cotidianas y la calidad de vida después de la administración a largo plazo de GIVINOSTAT.7Spanish7196032ce4c4e1b-96b1-4cff-8867-7f1762397896Het evalueren van de impact op de dagelijkse activiteiten en de kwaliteit van leven na langetermijntoediening van GIVINOSTAT37Dutch (Netherlands)6092451Subjects must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in study DSC/14/2357/48 and met: all the inclusion criteria and none of the exclusion criteria, had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or >30%, i.e. included in "off-target" group, never been randomized because the enrollment in the off-target group was completed719600759b9d250-9386-495a-9f9b-f146080469e1Los participantes deben haber participado en uno de los estudios anteriores con GIVINOSTAT en la DMD y haber acudido a la visita de fin del estudio o debían haber sido seleccionados en el estudio DSC/14/2357/48 y cumplir lo siguiente: todos los criterios de inclusión y ninguno de los criterios de exclusión, presentaban una fracción de grasa del músculo vasto externo (vastus lateralis muscle fat fraction, VL MFF) inicial evaluada mediante MRS en el intervalo ≤5 % o >30 %, es decir, haberse incluido en el grupo «no objetivo», nunca haber sido aleatorizado porque la inscripción en el grupo no objetivo se había completado.7Spanish719600959b9d250-9386-495a-9f9b-f146080469e1Proefpersonen moeten hebben deelgenomen aan een van de eerdere onderzoeken met GIVINOSTAT bij DMD en moeten het bezoek aan het einde van het onderzoek hebben bijgewoond of moeten zijn gescreend in onderzoek DSC/14/2357/48 en hebben voldaan aan: alle inclusiecriteria en geen van de exclusiecriteria, het hebben een spiervetfractie van de vastus lateralis (VL MFF) bij de baseline beoordeeld met MRS in het bereik ≤ 5% of > 30%, d.w.z. zijn opgenomen in de "off-target" groep, maar nooit gerandomiseerd, omdat de inschrijving in de off-target groep voltooid was.37Dutch (Netherlands)719600859b9d250-9386-495a-9f9b-f146080469e1Les patients doivent avoir participé à l’une des études précédentes portant sur le GIVINOSTAT dans la DMD et avoir effectué la visite de fin de l’étude, ou doivent avoir été sélectionnés dans l’étude DSC/14/2357/48 et : avoir répondu à la totalité des critères d’inclusion et n’avoir répondu à aucun des critères d’exclusion, avoir présenté une fraction graisseuse du muscle vaste latéral (FG MVL) de référence évaluée par spectrométrie par résonance magnétique (SRM) dans la plage ≤ 5 % ou > 30 %, c’est-à-dire avoir été inclus dans la cohorte « hors cible », ne jamais avoir été randomisés, car l’inclusion dans la cohorte hors cible était terminée10French6092462Subjects aged ≥6 years7196010aaf4cc7b-d817-4286-a7a7-66d84d49bea4Proefpersonen met een leeftijd ≥ 6 jaar.37Dutch (Netherlands)7196012aaf4cc7b-d817-4286-a7a7-66d84d49bea4Participantes con una edad ≥6 años.7Spanish7196011aaf4cc7b-d817-4286-a7a7-66d84d49bea4Patients âgés de ≥ 6 ans10French6092473Subjects are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations)7196015bb1a4229-58ae-400c-a74d-a7ea35542cd5Les patients sont capables de fournir un assentiment et/ou un consentement éclairé par écrit, signé par le patient et/ou son parent/tuteur légal (conformément à la réglementation locale)10French7196013bb1a4229-58ae-400c-a74d-a7ea35542cd5Los participantes son capaces de dar su asentimiento o consentimiento informado por escrito, firmado por el participante o el progenitor/tutor (de acuerdo con la normativa local).7Spanish7196014bb1a4229-58ae-400c-a74d-a7ea35542cd5Proefpersonen zijn in staat om schriftelijke geïnformeerde instemming en/of toestemming te geven, ondertekend door de proefpersoon en/of ouder/wettelijk voogd (volgens plaatselijke regelgeving).37Dutch (Netherlands)6092484Subjects must be willing to use adequate contraception: Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following: -True abstinence (absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception. -Condom with spermicide and the female partner must use an acceptable method of contraception, such as an oral, transdermal, injectable or implanted steroid-based contraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such as for example cervical cap with spermicide jelly.719601843eafb18-1f7e-49e3-99a6-946398e6c0efProefpersonen moeten bereid zijn om adequate anticonceptie te gebruiken: Anticonceptiemethoden moeten zijn toegepast sinds het vorige GIVINOSTAT-onderzoek tot 3 maanden na de laatste dosis onderzoeksmiddel en het volgende omvatten: -Volledige onthouding (afwezigheid van seksuele geslachtsgemeenschap), in overeenstemming met de gewenste en gebruikelijke levensstijl van de proefpersoon. Periodieke onthouding (bijv. kalender-, ovulatie-, sympto-thermale en postovulatiemethode) en terugtrekking zijn geen aanvaardbare anticonceptiemethoden. - Condoom met zaaddodend middel en de vrouwelijke partner moet gebruikmaken van een aanvaardbare anticonceptiemethode, zoals een orale, transdermale, injecteerbare of geïmplanteerde steroïde-bevattende anticonceptiemethode, of een pessarium of een barrièremethode in combinatie met zaaddodende gel, zoals een cervixkapje met zaaddodende gel.37Dutch (Netherlands)719601643eafb18-1f7e-49e3-99a6-946398e6c0efLes patients doivent être disposés à utiliser une contraception adéquate. Les méthodes de contraception doivent être utilisées à partir de la précédente étude sur le GIVINOSTAT et jusqu’à 3 mois après la dernière dose du médicament à l’étude, et comprennent les suivantes : - Abstinence totale (absence de tout rapport sexuel), lorsqu’elle correspond au mode de vie préféré et habituel du patient. L’abstinence périodique (p. ex., méthode du calendrier, méthode de la glaire cervicale, méthode sympto-thermique, méthode des températures) et le retrait ne sont pas des méthodes de contraception acceptables. - Préservatif avec spermicide et utilisation par la partenaire féminine d’une méthode de contraception acceptable, comme un contraceptif oral, transdermique, injectable ou implanté à base de stéroïdes, ou un diaphragme ou une méthode barrière associée à un spermicide, par exemple une cape cervicale avec un gel spermicide.10French719601743eafb18-1f7e-49e3-99a6-946398e6c0efLos participantes deben estar dispuestos a utilizar métodos anticonceptivos adecuados: Los métodos anticonceptivos deben utilizarse desde el estudio de GIVINOSTAT anterior hasta 3 meses después de la última dosis del fármaco del estudio, y son los siguientes: — Abstinencia verdadera (ausencia de relaciones sexuales), si se ajusta al estilo de vida preferido y habitual del participante. No se consideran métodos anticonceptivos aceptables la abstinencia sexual periódica (p. ej., métodos de calendario, ovulación, sintotérmico o posovulación) ni el coito interrumpido. — Preservativo con espermicida, y la pareja de sexo femenino debe usar un método anticonceptivo aceptable, como un anticonceptivo oral, transdérmico, inyectable o implantado con esteroides, o un diafragma o un método anticonceptivo de barrera junto con gel espermicida, como un capuchón cervical con espermicida.7Spanish10472531Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed;7195967045feed2-0630-44cf-ab3b-99f323be8f6dUtilisation de tout traitement pharmacologique, autre que les corticostéroïdes, qui pourrait avoir un effet sur la force ou la fonction musculaire dans les 3 mois précédant l’inclusion dans cette étude (p. ex., hormone de croissance) ; la vitamine D, le calcium et tout autre supplément seront autorisés10French7195965045feed2-0630-44cf-ab3b-99f323be8f6dHet gebruik van een farmacologische behandeling, anders dan corticosteroïden, die mogelijk een effect heeft gehad op de spierkracht of de spierfunctie binnen 3 maanden voorafgaand aan inschrijving in dit onderzoek (bijv. groeihormoon). Vitamine D, calcium en andere supplementen zijn toegestaan.37Dutch (Netherlands)7195966045feed2-0630-44cf-ab3b-99f323be8f6dUso de cualquier tratamiento farmacológico, distinto de los corticoesteroides, que pueda haber tenido un efecto sobre la fuerza o la función muscular en los 3 meses anteriores a la inscripción en este estudio (p. ej., hormona de crecimiento); se permitirá la vitamina D, el calcio y cualquier otro suplemento7Spanish104725410Have a baseline QTcF >450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome)71959709f415ee4-562c-4c60-837e-1f62297e4fa0Présenter un QTcF de référence > 450 ms (moyenne de 3 lectures consécutives espacées de 5 minutes) ou des antécédents de facteurs de risque supplémentaires de torsades de pointes (p. ex., insuffisance cardiaque, hypokaliémie ou antécédents familiaux de syndrome du QT long)10French71959699f415ee4-562c-4c60-837e-1f62297e4fa0Tener un QTcF inicial >450 ms (como la media de 3 lecturas consecutivas con 5 minutos de diferencia) o antecedentes de factores de riesgo adicionales de torsades de pointes (p. ej., insuficiencia cardíaca, hipopotasiemia o antecedentes familiares del síndrome de QT largo).7Spanish71959689f415ee4-562c-4c60-837e-1f62297e4fa0Het hebben van een QTcF > 450 msec bij de baseline (als het gemiddelde van 3 opeenvolgende metingen met tussenpozen van 5 minuten) of een voorgeschiedenis van bijkomende risicofactoren voor torsades de pointes (bijv. hartfalen, hypokaliëmie of een familievoorgeschiedenis van lang QT syndroom).37Dutch (Netherlands)104725511Have a psychiatric illness/social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures71959728e52c6f9-b565-4402-8214-c7ba53f8a7d5Tener una enfermedad psiquiátrica o situaciones sociales que hagan que el posible participante no pueda comprender ni cumplir con las pruebas de función muscular o con los procedimientos del protocolo del estudio.7Spanish71959738e52c6f9-b565-4402-8214-c7ba53f8a7d5Avoir une maladie psychiatrique/une situation sociale rendant le patient potentiel incapable de comprendre les examens de la fonction musculaire et/ou les procédures du protocole de l’étude et de s’y conformer10French71959718e52c6f9-b565-4402-8214-c7ba53f8a7d5Het hebben van een psychiatrische ziekte/sociale situatie die de potentiële proefpersoon belet om de spierfunctietesten en/of de onderzoeksprotocolprocedures te begrijpen en na te leven.37Dutch (Netherlands)104725612Have any hypersensitivity to the components of study medication;719597577539a2d-03d7-4eb0-9967-9b62539b4962Tener hipersensibilidad a los componentes del fármaco del estudio.7Spanish719597677539a2d-03d7-4eb0-9967-9b62539b4962Het hebben van een overgevoeligheid voor de bestanddelen van het onderzoeksmiddel37Dutch (Netherlands)719597477539a2d-03d7-4eb0-9967-9b62539b4962Présenter une hypersensibilité aux composants du médicament à l’étude10French104725713Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance7195978336ea243-860d-49af-be84-cc13ce676313Tener intolerancia al sorbitol o malabsorción de sorbitol o intolerancia hereditaria a la fructosa.7Spanish7195979336ea243-860d-49af-be84-cc13ce676313Présenter une intolérance au sorbitol ou une malabsorption du sorbitol, ou une forme héréditaire d’intolérance au fructose10French7195977336ea243-860d-49af-be84-cc13ce676313Het hebben van een sorbitolintolerantie of sorbitolmalabsorptie of het hebben van de erfelijke vorm van fructose-intolerantie.37Dutch (Netherlands)10472582Use of any current investigational drug other than Givinostat7195981a4158122-f312-4762-b770-40cb68259949Uso de cualquier fármaco en investigación actual distinto de GIVINOSTAT.7Spanish7195982a4158122-f312-4762-b770-40cb68259949Utilisation de tout médicament en cours d’investigation autre que le Givinostat10French7195980a4158122-f312-4762-b770-40cb68259949Huidig gebruik van een experimenteel geneesmiddel anders dan Givinostat.37Dutch (Netherlands)10472593Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results7195985f20da03f-d3a6-46f9-b508-e7cf8afcc1c4Aanwezigheid van een andere klinisch significante ziekte die, naar de mening van de onderzoeker, een negatieve invloed zou kunnen hebben op de veiligheid van de proefpersoon, waardoor het onwaarschijnlijk is dat de behandeling of de follow-up zal worden afgerond of die de beoordeling van de onderzoeksresultaten zou kunnen beïnvloeden.37Dutch (Netherlands)7195983f20da03f-d3a6-46f9-b508-e7cf8afcc1c4Presencia de otra enfermedad clínicamente significativa, que, en opinión del investigador, podría afectar de forma negativa a la seguridad del participante, haciendo improbable que se completase el curso del tratamiento o del seguimiento, o que pueda afectar a la evaluación de los resultados del estudio.7Spanish7195984f20da03f-d3a6-46f9-b508-e7cf8afcc1c4Présenter une autre maladie cliniquement significative qui, de l’avis de l’investigateur, pourrait nuire à la sécurité du patient, rendant peu probable l’achèvement du traitement ou du suivi, ou pourrait nuire à l’évaluation des résultats de l’étude10French10472604Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD7195986a7c15cd5-7994-419d-99f0-bc095da5b943Tener un diagnóstico de otras enfermedades neurológicas no controladas o presencia de trastornos somáticos relevantes no controlados que no estén relacionados con la DMD.7Spanish7195987a7c15cd5-7994-419d-99f0-bc095da5b943Avoir reçu le diagnostic d’autres maladies neurologiques non contrôlées ou présenter des troubles somatiques non contrôlés importants qui ne sont pas liés à la DMD10French7195988a7c15cd5-7994-419d-99f0-bc095da5b943Het hebben van een diagnose van andere ongecontroleerde neurologische ziekten of aanwezigheid van relevante ongecontroleerde somatische aandoeningen die niet gerelateerd zijn aan DMD.37Dutch (Netherlands)10472615Have platelets count, White Blood Cell and Hemoglobin at screening < Lower Limit of Normal (LLN) (for abnormal screening laboratory test results (7195989b30aafcf-42fe-433c-82a1-b29622a29520Présenter une numération plaquettaire, une numération des globules blancs et un taux d’hémoglobine < limite inférieure de la normale (LIN) à la sélection (en cas de résultats anormaux des analyses biologiques de sélection [< LIN], le dosage des plaquettes, des globules blancs et de l’hémoglobine sera répété une fois ; si le résultat des nouveaux dosages est toujours < LIN, cela constituera un critère d’exclusion)10French7195991b30aafcf-42fe-433c-82a1-b29622a29520Het aantal bloedplaatjes, het aantal witte bloedcellen en de concentratie hemoglobine bij de keuring < ondergrens van normaal (LLN - lower limit of normal) (voor afwijkende laboratoriumtestresultaten bij de keuring (37Dutch (Netherlands)7195990b30aafcf-42fe-433c-82a1-b29622a29520Tener unas cifras de trombocitos, leucocitos y hemoglobina en la selección < límite inferior de la normalidad (LIN) (para los resultados anómalos en los análisis clínicos de la selección [7Spanish10472626Have Triglycerides > 300 mg/dL (3.42 mmol/L) in fasting condition at screening visit* (for abnormal screening laboratory test results (>300mg/dl), the triglycerides will be repeated once; if the repeat test result is still >300mg/dl, then exclusionary7195994a019d004-78ea-4373-ad43-6782bc00365bTener triglicéridos >300 mg/dl (3,42 mmol/l) en ayunas en la visita de selección* (para los resultados anómalos en los análisis clínicos de selección (>300 mg/dl); el análisis de los triglicéridos se repetirá una vez; si el resultado de la repetición sigue siendo >300 mg/dl, será excluyente.7Spanish7195993a019d004-78ea-4373-ad43-6782bc00365bDe concentratie triglyceriden > 300 mg/dl (3,42 mmol/l) in nuchtere toestand bij het keuringsbezoek* (voor afwijkende laboratoriumtestresultaten bij de keuring (> 300 mg/dl) wordt de concentratie triglyceriden één keer herhaald; als het testresultaat van de herhaling nog steeds > 300 mg/dl is, dan exclusie).37Dutch (Netherlands)7195992a019d004-78ea-4373-ad43-6782bc00365bPrésenter un taux de triglycérides > 300 mg/dl (3,42 mmol/l) à jeun lors de la visite de sélection* (en cas de résultats anormaux des analyses biologiques de sélection [> 300 mg/dl], le dosage des triglycérides sera répété une fois ; si le résultat du nouveau dosage est toujours > 300 mg/dl, cela constituera un critère d’exclusion)10French10472637Have inadequate renal function, as defined by serum Cystatin C >2 x the upper limit of normal (ULN) at screening visit. If the value is >2 x ULN, the serum Cystatin C will be repeated once; if the repeated test result is still >2 x ULN, the subject should be excluded);7195997fdfcb82a-6919-48ca-a55e-87085c04409bPrésenter une fonction rénale inadéquate, définie par un taux sérique de cystatine C > 2 x la limite supérieure de la normale (LSN) lors de la visite de sélection. Si la valeur est > 2 x LSN, le dosage sérique de la cystatine C sera répété une fois ; si le résultat du nouveau dosage est toujours > 2 x LSN, cela constituera un critère d’exclusion10French7195996fdfcb82a-6919-48ca-a55e-87085c04409bHet hebben van onvoldoende nierfunctie, zoals gedefinieerd door serumcystatine C > 2 x de bovengrens van normaal (ULN, upper limit of normal) bij het keuringsbezoek. Als de waarde > 2 x ULN is, wordt het serumcystatine C eenmaal herhaald; als het testresultaat van de herhaling nog steeds > 2 x ULN is, moet de proefpersoon worden uitgesloten).37Dutch (Netherlands)7195995fdfcb82a-6919-48ca-a55e-87085c04409bPresencia de una función renal insuficiente, definida mediante cistatina C en suero >2 veces el límite superior de la normalidad (LSN) en la visita de selección. Si el valor es >2 veces el LSN, se repetirá el análisis de la cistatina C en suero una vez; si el resultado de la repetición sigue siendo >2 veces el LSN, se deberá excluir al participante).7Spanish10472648Have heart failure (New York Heart Association Class III or IV)7195998ad19ab18-66a8-48f7-800c-078ff67f4da9Présenter une insuffisance cardiaque (de classe III ou IV sur l’échelle de la New York Heart Association)10French7196000ad19ab18-66a8-48f7-800c-078ff67f4da9Het hebben van hartfalen (New York Heart Association klasse III of IV).37Dutch (Netherlands)7195999ad19ab18-66a8-48f7-800c-078ff67f4da9Tener insuficiencia cardíaca (clase III o IV según la Asociación Cardiológica de Nueva York).7Spanish10472659Have a current liver disease or impairment, including but not limited to an elevated total bilirubin(i.e. > 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's7196003ca847bec-46a0-43fb-80d4-96621179e25dTener una enfermedad o deterioro hepático actual, incluidos, entre otros, bilirrubina total elevada (es decir, >1,5 veces el LSN), a menos que sea secundaria a la enfermedad de Gilbert o con un patrón coincidente con la enfermedad de Gilbert.7Spanish7196002ca847bec-46a0-43fb-80d4-96621179e25dPrésenter actuellement une maladie ou une insuffisance hépatique, y compris, mais sans s’y limiter, une bilirubine totale élevée (c’est-à-dire > 1,5 x LSN), à moins qu’elle ne soit secondaire à la maladie de Gilbert ou à un profil compatible avec celui de la maladie de Gilbert10French7196001ca847bec-46a0-43fb-80d4-96621179e25dHet hebben van een huidige leverziekte of leverfunctiestoornis, waaronder, maar niet beperkt tot een verhoogd totaal bilirubine (d.w.z. > 1,5 x ULN), tenzij secundair aan de ziekte van Gilbert of patroon dat overeenkomt met de ziekte van Gilbert.37Dutch (Netherlands)7117621Type, incidence, and severity of treatment related/not related AEs and SAEsTrue7195936f6d7dba4-7c04-4b4d-925f-9401c86259f4Type, incidence et sévérité des EI et des EIG liés ou non liés au traitement10French7195937f6d7dba4-7c04-4b4d-925f-9401c86259f4Type, incidentie en ernst van aan de behandeling gerelateerde/niet aan de behandeling gerelateerde ongewenste voorvallen en ernstige ongewenste voorvallen.37Dutch (Netherlands)7195935f6d7dba4-7c04-4b4d-925f-9401c86259f4Tipo, incidencia e intensidad de los AA y AAG relacionados o no relacionados con el tratamiento.7Spanish7117631For ambulant patients: Change from baseline in physical function as measured by 6MWT, NSAA, Time function tests (e.g. time to rise from floor, time to climb 4-stairs, time to 10m walk) (week 48 and then yearly till the end of the study)False7195940cd23f700-c981-46a9-a54c-d2efbef2eff0Pour les patients capables de se déplacer : • Variation par rapport à la référence de la fonction physique, mesurée par l’épreuve de marche de six minutes, le test NSAA, les tests de fonction temporels (p. ex., temps pour se lever du sol, temps pour monter 4 marches, temps pour marcher 10 m) (semaine 48, puis annuellement jusqu’à la fin de l’étude)10French7195938cd23f700-c981-46a9-a54c-d2efbef2eff0Para pacientes deambulantes: • Cambio con respecto al inicio en la función física medida mediante 6MWT, NSAA, pruebas de función temporal (p. ej. tiempo para levantarse del suelo, tiempo para subir 4 escalones, tiempo para caminar 10 metros) (semana 48 y luego cada año hasta el final del estudio).7Spanish7195939cd23f700-c981-46a9-a54c-d2efbef2eff0Voor poliklinische patiënten: • Verandering ten opzichte van de baseline in lichamelijk functioneren zoals gemeten aan de hand van 6MWT, NSAA, tijdfunctietesten (bijv. tijd om op te staan van de vloer, tijd om 4 trappen op te lopen, tijd om 10 meter te lopen) (week 48 en daarna jaarlijks tot het einde van het onderzoek).37Dutch (Netherlands)7117642For ambulant patients: Change from baseline in muscle strength (e.g. knee extension and elbow flexion) as measured by HHM (week 48 and then yearly till the end of the study)False71959420bdcf54b-d110-41ef-a181-0a2c702fbd22Voor poliklinische patiënten: Verandering ten opzichte van de baseline in de spierkracht (bijv. knie-extensie en elleboogflexie) zoals gemeten aan de hand van HHM (week 48 en daarna jaarlijks tot het einde van het onderzoek).37Dutch (Netherlands)71959410bdcf54b-d110-41ef-a181-0a2c702fbd22Pour les patients capables de se déplacer : Variation par rapport à la référence de la force musculaire (p. ex., extension du genou et flexion du coude), mesurée par myométrie (appareil portatif ; semaine 48, puis annuellement jusqu’à la fin de l’étude)10French71959430bdcf54b-d110-41ef-a181-0a2c702fbd22Para pacientes deambulantes: Cambio con respecto al inicio en la fuerza muscular (p. ej., extensión de la rodilla y flexión del codo) medida mediante miometría manual (handheld myometry, HHM) (semana 48 y luego cada año hasta el final del estudio).7Spanish7117653For non-ambulant patients: Change in physical function from baseline in the Egen Klassifikation (EK) score (week 48 and then yearly till the end of the study)False7195944231965e9-bf33-4dc0-86ef-0faec4905918Pour les patients incapables de se déplacer : • Variation par rapport à la référence de la fonction physique selon le score Egen Klassifikation (EK) (semaine 48, puis annuellement jusqu’à la fin de l’étude)10French7195946231965e9-bf33-4dc0-86ef-0faec4905918Voor niet-poliklinische patiënten: • Verandering ten opzichte van de baseline in lichamelijk functioneren zoals gemeten aan de hand van de Egen Klassifikation (EK)-score (week 48 en daarna jaarlijks tot het einde van het onderzoek).37Dutch (Netherlands)7195945231965e9-bf33-4dc0-86ef-0faec4905918Para pacientes no deambulantes: • Cambio con respecto al inicio en la función física en la puntuación de Egen Klassifikation (EK) (semana 48 y luego cada año hasta el final del estudio).7Spanish7117664For non-ambulant patients: Change in patient and/or parent/caregiver reports of activities of daily living as measured by Barthel Index (week 48 and then yearly till the end of the study)False71959490fb80a3e-c3ed-4a7c-8860-30efc5175049Para pacientes no deambulantes: • Cambios en los informes de los pacientes o progenitores o cuidadores en las actividades cotidianas medidas por el Índice de Barthel (semana 48 y luego cada año hasta el final del estudio).7Spanish71959470fb80a3e-c3ed-4a7c-8860-30efc5175049Voor niet-poliklinische patiënten: • Verandering in door de patiënt en/of ouder/verzorger gedane meldingen betreffende activiteiten van het dagelijks leven zoals gemeten aan de hand van de Barthel-index (week 48 en daarna jaarlijks tot het einde van het onderzoek).37Dutch (Netherlands)71959480fb80a3e-c3ed-4a7c-8860-30efc5175049Pour les patients incapables de se déplacer : • Variation dans les rapports des patients et/ou des parents/aidants sur les activités de la vie quotidienne, mesurée par l’indice de Barthel (semaine 48, puis annuellement jusqu’à la fin de l’étude)10French7117675For non-ambulant patients: Change in upper limbs muscle strength (week 48 and then yearly till the end of the study) evaluated by handheld myometry (HHM)False719595243887e7c-d9c4-4611-91df-9b2a7c54b2ebPara pacientes no deambulantes: • Cambios en la fuerza muscular de las extremidades superiores (semana 48 y luego cada año hasta el final del estudio) evaluada mediante miometría manual (HHM).7Spanish719595143887e7c-d9c4-4611-91df-9b2a7c54b2ebVoor niet-poliklinische patiënten: • Verandering in de spierkracht van de bovenste ledematen (week 48 en daarna jaarlijks tot het einde van het onderzoek) geëvalueerd met HHM (handheld myometrie).37Dutch (Netherlands)719595043887e7c-d9c4-4611-91df-9b2a7c54b2ebPour les patients incapables de se déplacer : • Variation de la force musculaire des membres supérieurs (semaine 48, puis annuellement jusqu’à la fin de l’étude) évaluée par myométrie (appareil portatif)10French7117686For all patients: Change from baseline in physical function as measured by the Performance of Upper Limb (PUL) and MFM (week 48 and then yearly till the end of the study)False7195953e88e01da-29b6-4a85-a8fd-e13b94bd2901Voor alle patiënten: Verandering ten opzichte van de baseline in lichamelijk functioneren zoals gemeten aan de hand van de prestatie van de bovenste ledematen (PUL, Performance of Upper Limb) en MFM (week 48 en daarna jaarlijks tot het einde van het onderzoek).37Dutch (Netherlands)7195955e88e01da-29b6-4a85-a8fd-e13b94bd2901Pour tous les patients : Variation par rapport à la référence de la fonction physique, mesurée par la performance des membres supérieurs (Performance of Upper Limb, PUL) et la MFM (semaine 48, puis annuellement jusqu’à la fin de l’étude)10French7195954e88e01da-29b6-4a85-a8fd-e13b94bd2901Para todos los pacientes: Cambio con respecto al inicio en la función física medida por el rendimiento de la extremidad superior (RES) y MFM (semana 48 y luego cada año hasta el final del estudio).7Spanish7117697For all patients: Change from baseline in respiratory function (week 48 and then yearly till the end of the study) (e.g. FVC, FEV1, PEF)False7195958ce82936c-1add-4215-a943-2e65c8184147Voor alle patiënten: Verandering ten opzichte van de baseline in respiratoire functie (week 48 en daarna jaarlijks tot het einde van het onderzoek) (bijv. FVC, FEV1, PEF).37Dutch (Netherlands)7195956ce82936c-1add-4215-a943-2e65c8184147Para todos los pacientes: Cambio con respecto al inicio en la función respiratoria (semana 48 y luego cada año hasta el final del estudio) (p. ej., CVF, VEM1, FEM).7Spanish7195957ce82936c-1add-4215-a943-2e65c8184147Pour tous les patients : Variation par rapport à la référence de la fonction respiratoire (semaine 48, puis annuellement jusqu’à la fin de l’étude) (p. ex. CVF, VEMS, DEP)10French7117708For all patients: Change in patient and/or parent/caregiver reports of quality of life as measured by PedsQL for paediatric patients and by SF-36 for adults patients (week 48 and then yearly till the end of the study)False7195961bfd2db52-3029-473e-b943-2126bfd4cb60Pour tous les patients : Variation dans les rapports des patients et/ou des parents/aidants sur la qualité de vie mesurée par le PedsQL pour les patients pédiatriques et par le SF-36 pour les patients adultes (semaine 48, puis annuellement jusqu’à la fin de l’étude)10French7195959bfd2db52-3029-473e-b943-2126bfd4cb60Voor alle patiënten: Verandering in door de patiënt en/of ouder/verzorger gedane meldingen betreffende de kwaliteit van leven zoals gemeten aan de hand van PedsQL voor pediatrische patiënten en aan de hand van SF-36 voor volwassen patiënten (week 48 en daarna jaarlijks tot het einde van het onderzoek).37Dutch (Netherlands)7195960bfd2db52-3029-473e-b943-2126bfd4cb60Para todos los pacientes: Cambio en los informes de calidad de vida del paciente o del progenitor o cuidador, medido mediante PedsQL para pacientes pediátricos y mediante SF-36 para pacientes adultos (semana 48 y luego cada año hasta el final del estudio).7Spanish7117719For all patients: Age to major disease milestones (e.g. age at loss of ambulation, age at respiratory support needed during the day, age at scoliosis surgery, age at death)False7195963c9985344-4bbf-4aa2-8869-4c7aa58808dbPara todos los pacientes: Edad en los hitos más importantes de la enfermedad (p. ej., edad en el momento de la pérdida de la deambulación, edad en la que se necesita asistencia respiratoria durante el día, edad en la cirugía de escoliosis, edad en el momento de la muerte).7Spanish7195962c9985344-4bbf-4aa2-8869-4c7aa58808dbPour tous les patients : Âge aux principales étapes de la maladie (p. ex., âge au moment de la perte de la capacité ambulatoire, âge auquel une assistance respiratoire est devenue nécessaire pendant la journée, âge au moment de la chirurgie correctrice de scoliose, âge au décès)10French7195964c9985344-4bbf-4aa2-8869-4c7aa58808dbVoor alle patiënten: Leeftijd op moment van belangrijke mijlpalen van de ziekte (bijv. leeftijd bij verlies van mobiliteit, leeftijd wanneer ademhalingsondersteuning overdag nodig is, leeftijd bij scoliose-operatie, leeftijd bij overlijden).37Dutch (Netherlands)2027-12-032027-12-032017-07-0366918ITALFARMACO S.p.A.2530292225302833253030262625303125252PatientsFalseTrueTrue31354101TreatmentGIVINOSTAT oral suspension (10 mg/mL) has to be administered orally as 2 oral doses daily while the subject is in a fed state. As weight affects GIVINOSTAT exposures, the dosage will be modified based on subject weight according to the rules detailed in the study protocol.337895EMEA-000551-PIP04-21acceptable5Diseases [C] - Musculoskeletal Diseases [C05]95355Duchenne Muscular Dystrophy (DMD)True101950True304577PublicPatient Advocacy Representative og Italfarmaco S.p.Apatientadvocacy@italfarmacogroup.com+390264432529102894Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active304578ScientificNicoletta Coceanin.coceani@italfarmacogroup.com+390264432529102894Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active447289660950698983Hospital/Clinic/Other health care facility8Illingworth Research Group LimitedFalseTrueORG-100042356Active718797Hazelwood House, Larkwood Way, Tytherington Business ParkHazelwood HouseLarkwood WayTytherington Business ParkMacclesfieldSK10 2XR826United Kingdom+441625310020robert.miller@illingworthresearch.comTrueORG-10004235670009415Home Nursing+441625310020robert.miller@illingworthresearch.com447283462561494878Pharmaceutical company10Catalent Germany Schorndorf GmbHTrueTrueORG-100011845Active495875Steinbeisstrasse 1-2Steinbeisstrasse 1-2Schorndorf736142013Germany+49718170000Lauren.Carter@catalent.comTrueORG-10001184570008714+49718170000Lauren.Carter@catalent.com447291660944698977Non-Pharmaceutical company11Fountayn LLCTrueTrueORG-100051582Active7187915900 Landerbrook Drive Suite 1705900 Landerbrook Drive Suite 170Mayfield Heights44124-4085840United States+390458202666valentina.biasi@alirahealth.comTrueORG-1000515827000967+390458202666valentina.biasi@alirahealth.com447292660947698980Non-Pharmaceutical company11ATOM International LimitedTrueTrueORG-100042393Active718794Office 16, Valley House, Seventh Avenue, Team Valley Trading EstateOffice 16Valley HouseSeventh AvenueTeam Valley Trading EstateGatesheadNE11 0JW826United Kingdom000000000michelle.eagle@atom-international.orgTrueORG-10004239370009715physioterapist training and evaluation000000000michelle.eagle@atom-international.org447281471441503816Pharmaceutical company10IQVIA LimitedTrueTrueORG-100008655Active5054943 Forbury Place, 23 Forbury Road3 Forbury Place23 Forbury RoadReadingRG1 3JH826United Kingdom+441184506016eu_clinical_trials_information@iqvia.comTrueORG-100008655700080170008111700082127000831370008457000858+441184506016eu_clinical_trials_information@iqvia.com447294462572494889Laboratory/Research/Testing facility9Acm Global Central Laboratory LimitedTrueTrueORG-100042459Active49588723 Hospital Fields Road, Fulford Industrial Estate23 Hospital Fields RoadFulford Industrial EstateYorkYO10 4DZ826United Kingdom+441904699400ACMYorkEUCTAGroup@acmgloballab.comTrueORG-10004245970009915Primary/ surrogate endpoint test7001004+441904699400ACMYorkEUCTAGroup@acmgloballab.com447288660951698984Hospital/Clinic/Other health care facility8Patient Primary LimitedFalseTrueORG-100052459Active718799Building 329, Doncastle RoadBuilding 329Doncastle RoadBracknellRG12 8PE826United Kingdom+441344577929matthew.newell@patientprimary.comTrueORG-10005245970009315Patient Travel reimbursement+441344577929matthew.newell@patientprimary.com447285660952698985Pharmaceutical company10Qualitymetric Incorporated LLCTrueTrueORG-100044132Active7188011301 Atwood Avenue Suite 216e1301 Atwood Avenue Suite 216eJohnston02919-4933840United States0034690914065Martinezcmartin@qualitymetric.comTrueORG-10004413270008915SF36 questionnaire and proCore software provider0034690914065Martinezcmartin@qualitymetric.com447293660953698986Pharmaceutical company10Trinds LLCTrueTrueORG-100051849Active7188026425 Living Place6425 Living PlacePittsburgh15206-5122840United States000000000mkotlarczyk@trinds.comTrueORG-10005184970009815IDMC services provider000000000mkotlarczyk@trinds.com447279519752552283Pharmaceutical company10Alira HealthTrueTrueORG-100030303Active55854312 Rue Charlot12 Rue CharlotParis750032012France+447917973305hannah.baxter@alirahealth.comTrueORG-100030303700077107000786+447917973305hannah.baxter@alirahealth.com447287660954698987Pharmaceutical company10Transperfect Translations International Inc.TrueTrueORG-100043494Active7188031250 Broadway Fl 32th1250 Broadway Fl 32thNew York10001-3749840United States+34936093273sgomez@transperfect.comTrueORG-10004349470009215Interpretation Services+34936093273sgomez@transperfect.com447280487307519732Health care2MFM Aide Aux Familles D'Enfants Handicapes MoteursFalseFalseORL-000008565Active5227803 rue de la terrasse3 rue de la terrasseLyon2012France0612721608carole.vuillerot@chu-lyon.frFalseORL-00000856570007915MFM physioterapist training and evaluation0612721608carole.vuillerot@chu-lyon.fr447282662091700129Pharmaceutical company10Certara USA Inc.TrueTrueORG-100042611Active7201944 Radnor Corporate Center Suite 3504 Radnor Corporate Center Suite 350Radnor19087-4525840United States+31652260465francesco.bellanti@certara.comTrueORG-10004261170008615Population PK analysis+31652260465francesco.bellanti@certara.com447284462542494859Pharmaceutical company10Endpoint Clinical Inc.TrueTrueORG-100040567Active49585655 Francisco Street Suite 20055 Francisco Street Suite 200San Francisco94133-2122840United States+18478592285support@endpointclinical.comTrueORG-1000405677000883+18478592285support@endpointclinical.com447286519749552280Pharmaceutical company10Eresearchtechnology Inc.TrueTrueORG-100013039Active5585391818 Market Street Ste 26001818 Market Street Ste 2600Philadelphia19103-3600840United States+447824858532Agata.Panas-Adamson@clario.ComTrueORG-10001303970009015ECG analysis/review7000914+447824858532Agata.Panas-Adamson@clario.Com447290660936698969Hospital/Clinic/Other health care facility8Syneos Health Clinique Inc.FalseTrueORG-100028348Active7187832500 Einstein St2500 Einstein StQuebecG1P 0A2124Canada+13673306197sebastien.couraud@syneoshealth.comTrueORG-10002834870009515PK Analysis+13673306197sebastien.couraud@syneoshealth.com102894Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active100100102894Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active105010Via Dei Lavoratori 54Via Dei Lavoratori 54Cinisello Balsamo200922018ItalyTrueLOC-100004430TrueCommercial2This is a phase III study to investigate long-term safety, tolerability, and efficacy of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.9120735Diseases [C] - Musculoskeletal Diseases [C05]288311ITF23571Test381032111133213ORAL SUSPENSIONPRD47976781ITF2357ORAL SUSPENSIONITF2357GIVINOSTATSUB189959GIVINOSTATITALFARMACO SPA11133213337329ITALFARMACO SPAChemicalChemicalGIVINOSTATSUB189959False1TrueEU/3/12/1009mg milligram(s)140mg milligram(s)0011ChemicalPRD4797678ITF235711ORAL USETrueITF2357givinostatORAL SUSPENSIONFalse183903334423344248132029Authorised2024-08-222024-08-22FalseunwillingNetherlands2024-09-10Authorised427912019-10-312024-11-19427922019-10-312024-11-19563462020-01-072024-11-19563472020-01-072024-11-19True581612019-10-312024-11-192020-01-07False4682033442Under evaluation2024-08-05T09:20:26.8615169833442Authorised2024-09-10T08:14:12.1211Netherlandsacceptable2025-03-10authorized_conditions2024-09-102024-09-10241188033517590550116Hospital/Clinic/Other health care facility8Radboud universitair medisch centrum / RADBOUDUMCFalseTrueORG-100031688Active556171Geert Grooteplein Zuid 10Geert Grooteplein Zuid 10Nijmegen6525 GA2029Netherlands+3179908944901saskia.houwen@radboudumc.nlTrueORG-1000316881327828SaskiaHouwen-van Opstal+3179908944901saskia.houwen@radboudumc.nl1Research Facility1188032517585550111Hospital/Clinic/Other health care facility8Leids Universitair Medisch Centrum (LUMC)FalseTrueORG-100014145Active556165Albinusdreef 2Albinusdreef 2Leiden2333 ZA2029Netherlands+31715262197e.h.niks@lumc.nlTrueORG-1000141451327827ErikNiks+31715262197e.h.niks@lumc.nl1Research FacilityAuthorised184546334373343748132002Authorised2024-08-222024-08-22TrueunwillingBelgium2024-09-09Authorised358522019-06-242024-09-25358532019-06-242024-09-25466302019-07-222024-09-25466312019-07-222024-09-25True480672019-06-242024-09-252019-07-22False4681533437Under evaluation2024-08-05T09:20:26.5985158233437Authorised2024-09-09T13:14:52.561Belgiumacceptable2025-03-13authorized_conditions2024-09-092024-09-0991192633500355532819Hospital/Clinic/Other health care facility8UZ LeuvenFalseTrueORG-100006001Active537124Herestraat 49Herestraat 49Leuven30002002Belgium+3216340610liesbeth.dewaele@uzleuven.beTrueORG-1000060011332857LiesbetDe Waele+3216340610liesbeth.dewaele@uzleuven.be1Neurology1192634500356532820Hospital/Clinic/Other health care facility8Centre Hospitalier Regional De La CitadelleFalseTrueORG-100028257Active537125Boulevard Du Douzieme De Ligne 1Boulevard Du Douzieme De Ligne 1Liege40002002Belgium+32142165870l.servais@institut-myologie.orgTrueORG-1000282571332858LaurentServais+32142165870l.servais@institut-myologie.org1CardiologyAuthorised165672334413344148132027Authorised2024-08-222024-08-22FalseunwillingSpain2024-09-09Authorised358502019-06-032024-09-25358512019-06-032024-09-25358492019-06-032024-09-25511502019-06-032025-02-24466282019-06-062024-09-25466292019-06-062024-09-25True480632019-06-032024-09-252019-06-06False4681933441Under evaluation2024-08-05T09:20:26.8115149733441Authorised2024-09-09T08:18:36.531Spainacceptable2025-02-17authorized_conditions2024-09-092024-09-0951072390501939534413Hospital/Clinic/Other health care facility8Hospital Sant Joan De Deu BarcelonaFalseTrueORG-100023083Active538850Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain+34936009733anascimento@sjdhospitalbarcelona.orgTrueORG-1000230831198223AndresNascimento+34936009733anascimento@sjdhospitalbarcelona.org1Neurology1072385501945534419Hospital/Clinic/Other health care facility8University Hospital Virgen Del Rocio S.L.FalseTrueORG-100030783Active538856Avenida De Manuel Siurot S/nAvenida De Manuel Siurot S/nSevilla410132027Spain+34955012431merchilo82@hotmail.comTrueORG-1000307831198218MercedesLopez Lobato+34955012431merchilo82@hotmail.com1Neurology1072389501944534418Hospital/Clinic/Other health care facility8Hospital Universitari Vall D HebronFalseTrueORG-100030781Active538855Passeig De La Vall D'Hebron 119-129Passeig De La Vall D'Hebron 119-129Barcelona080352027Spain+34934893156francina.munell@vhir.orgTrueORG-1000307811198222FrancinaMunell Casadesus+34934893156francina.munell@vhir.org1Neurology1072388501940534414Hospital/Clinic/Other health care facility8Hospital Universitario Y Politecnico La FeFalseTrueORG-100029610Active538851Avenida De Fernando Abril Martorell 106Avenida De Fernando Abril Martorell 106Valencia460262027Spain+34620834854nuriamugo@hotmail.comTrueORG-1000296101198220NuriaMuelas Gomez+34620834854nuriamugo@hotmail.com1NeurologyAuthorised202561334393343948132013Authorised2024-08-222024-08-22FalseunwillingGermany2024-09-09Authorised358772019-12-092024-09-25358782019-12-092024-09-25466522019-12-122024-09-25466532019-12-122024-09-25True480862019-12-092024-09-252019-12-12False4681733439Under evaluation2024-08-05T09:20:26.7125149833439Authorised2024-09-09T08:30:25.5081Germanyacceptable2025-06-19authorized_conditions2024-09-092024-09-09131337635499000531453Hospital/Clinic/Other health care facility8Klinikum der Universitaet Muenchen AöRFalseTrueORG-100008479Active535635Lindwurmstrasse 4, Ludwigsvorstadt-IsarvorstadtLindwurmstrasse 4Ludwigsvorstadt-IsarvorstadtMunich803372013Germany+4989440055110astrid.blaschek@med.uni-muenchen.deTrueORG-1000084791490921AstridBlaschek+4989440055110astrid.blaschek@med.uni-muenchen.de1Neurology1337634499001531454Hospital/Clinic/Other health care facility8Universitaetsklinikum Essen AöRFalseTrueORG-100009964Active535637Hufelandstrasse 55, HolsterhausenHufelandstrasse 55HolsterhausenEssen451472013Germany+492017232508ulrike.schara@uk-essen.deTrueORG-1000099641490920UlrikeSchara-Schmidt+492017232508ulrike.schara@uk-essen.de2Neurology1337633498996531449Hospital/Clinic/Other health care facility8University Medical Center Hamburg-EppendorfFalseTrueORG-100008810Active535631Martinistrasse 52, EppendorfMartinistrasse 52EppendorfHamburg202462013Germany+4940741055512jjohannsen@uke.deTrueORG-1000088101490919JessikaJohannsen+4940741055512jjohannsen@uke.de1Neurology1414992768801807512Hospital/Clinic/Other health care facility8Medical Center - University Of FreiburgFalseTrueORG-100010322Active847866Breisacher Strasse 62, StuehlingerBreisacher Strasse 62StuehlingerFreiburg Im Breisgau791062013Germany+4976127043651janbernd.kirschner@uniklinik-freiburg.deTrueORG-1000103221575776JanberndKirschner+4976127043651janbernd.kirschner@uniklinik-freiburg.de2Department of Neuropediatrics and Muscle Disorders1414993768810807521Hospital/Clinic/Other health care facility8Charite Universitaetsmedizin Berlin KöRFalseTrueORG-100008480Active847876Augustenburger Platz 1, WeddingAugustenburger Platz 1WeddingBerlin133532013Germany+4930450666279joanna.schneider@charite.deTrueORG-1000084801575777JoannaSchneider+4930450666279joanna.schneider@charite.de1NeuropediatryAuthorised202562334383343848132012Authorised2024-09-122024-09-12FalseunwillingFrance2024-09-17Authorised359412020-01-202024-09-25359422020-01-202024-09-25359432020-01-202024-09-25467642020-02-172024-09-25467652020-02-172024-09-25True482032020-01-202024-09-252020-02-17False4681633438Under evaluation2024-08-05T09:20:26.6625273333438Authorised2024-09-17T12:02:23.2651Franceacceptable2025-07-03authorized_conditions2024-09-172024-09-17111337638540054572662Hospital/Clinic/Other health care facility8Trousseau HospitalFalseTrueORG-100031521Active58097926 Avenue Du Docteur Arnold Netter26 Avenue Du Docteur Arnold NetterParis750122012France+33140034170odile.boespflug-tanguy@aphp.frTrueORG-1000315211490927OdileBoespflug-Tanguy+33140034170odile.boespflug-tanguy@aphp.fr2Neuro-Pediatric1337639540056572664Hospital/Clinic/Other health care facility8Centre Hospitalier Universitaire De NantesFalseTrueORG-100007295Active5809811 Place Alexis Ricordeau1 Place Alexis RicordeauNantes440002012France+33240083704yann.pereon@univ-nantes.frTrueORG-1000072951490928YannPereon+33240083704yann.pereon@univ-nantes.fr2Neuro-Pediatric1382097759023797686Hospital/Clinic/Other health care facility8Centre Hospitalier Regional De MarseilleFalseTrueORG-100008698Active836353264 Rue Saint Pierre264 Rue Saint PierreMarseille130052012France0491385605cecile.halbert@ap-hm.frTrueORG-1000086981539824CécileHalbert0491385605cecile.halbert@ap-hm.fr1Neurométabolisme pédiatrique1382096759001797664Hospital/Clinic/Other health care facility8Centre Hospitalier Universitaire De LilleFalseTrueORG-100006742Active836325Rue Emile LaineRue Emile LaineLille Cedex590372012France0320445962jeanbaptiste.davion@chu-lille.frTrueORG-1000067421539823Jean-BaptisteDavion0320445962jeanbaptiste.davion@chu-lille.fr1Neurologie et neurologie pédiatriqueAuthorised224227334403344048132018Authorised2024-08-222024-08-22FalseunwillingItaly2024-10-09Authorised380972017-10-192024-10-11380982017-10-192024-10-11497362017-10-252024-10-11497372017-10-252024-10-11True511642017-10-192024-10-112017-10-25False4681833440Under evaluation2024-08-05T09:20:26.7625614433440Authorised2024-10-09T23:59:591Italyauthorized_conditions2024-10-092024-10-09581509289762781801468Educational Institution1Universita Degli Studi Di 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Antibody Titers After Imlifidase Administration [Time Frame: Up to Hour 120], Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration [Time Frame: Up to Day 7], Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [Time Frame: Up to Week 104]Idefirix 11 mg powder for concentrate for solution for infusion, Delandistrogene moxeparvovec-rokl450-17 yearsMale1EEA7Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content [Time Frame: Baseline, Week 12], Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity [Time Frame: Baseline, Week 12], Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in 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Summary_ENG_Public47521d40-d425-4b5e-b701-08d089bbde8046Laypersons summary of results (for publication)PDF5389n/a1Sarepta_SRP-9001-104__Plain Language Summary_ESP_Publicf8f5f397-30d4-4b92-8ebb-a29215e24f1446Laypersons summary of results (for publication)PDF5389N/A1Sarepta_SRP-9001-104_CT Summary Result_Public6d44bc6f-1b62-41d5-b7c0-e91c062c3b34103Summary of results (for publication)PDF5388N/A1Spain370162024-08-29T08:59:05.3052024-08-29T08:59:05.305837016TH-771652025-03-314Safety related (clinical or pre-clinical results)FalseFalse7Study management relatedFalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.Subject safety reasonsTrue2025-03-31T17:42:28The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3137016SpainES37016SpainSTART_OF_TRIAL2023-12-15START_OF_RECRUITMENT2024-01-17END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-31EARLY_TERMINATION2025-10-101Sponsor DecisionTrue53882024-512624-11-00Sarepta_SRP-9001-104_CT Summary Result_04Mar2026_PublicSubmittedSummary of ResultsFinalTrue1275632026-04-07T09:53:56.6489552026-04-03T21:38:09.9332026-04-07T09:53:5613430SUM-12750753892024-512624-11-00Plain Language SummarySubmittedLaypersons Summary of ResultsFinalTrue1275082026-04-07T09:54:05.3632222026-04-03T12:23:10.9892026-04-07T09:54:0513430SUM-1275082024-512624-11-008An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association with Imlifidase in Subjects with Duchenne Muscular Dystrophy with Pre-existing Antibodies to rAAVrh74SRP-9001-10415/12/202310/10/2025Duchenne muscular dystrophy (DMD)Spain:829/08/2024ES: 29/08/2024Diseases [C] - Musculoskeletal Diseases [C05]Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Sarepta Therapeutics Inc.Pharmaceutical companyHuman Pharmacology (Phase I)- OtherMaximum Observed Plasma Concentration (Cmax) of Imlifidase [Time Frame: Up to Day 7], Total IgG in Serum After Imlifidase Administration [Time Frame: Up to Week 12], rAAVrh74 Antibody Titers After Imlifidase Administration [Time Frame: Up to Hour 120], Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration [Time Frame: Up to Day 7], Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [Time Frame: Up to Week 104]Idefirix 11 mg powder for concentrate for solution for infusion, Delandistrogene moxeparvovec-rokl450-17 yearsMale17Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content [Time Frame: Baseline, Week 12], Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity [Time Frame: Baseline, Week 12], Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF) [Time Frame: Baseline, Week 12], Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration [Time Frame: Week 12]Yes18/03/202508/04/20262024-512624-11-00Ended2023-12-152025-10-102024-08-29T08:59:05.3052026-04-08T03:40:25.3728928998787380322455111507168POWDER FOR CONCENTRATE FOR SOLUTION FOR INFUSIONPRD8297747EU/1/20/1471/0012Idefirix 11 mg powder for concentrate for solution for infusionSOLUTION FOR INFUSIONIMLIFIDASESUB194312EUEMEA/H/C/004849HANSA BIOPHARMA AB11507168352813HANSA BIOPHARMA ABProtein - OtherProtein - OtherIMLIFIDASESUB194312IMMUNOGLOBULIN G DEGRADING ENZYME OF STREPTOCOCCUS PYOGENESHMED-IDESIDESL04AA41-5ImlifidaseFalse1TrueEU/3/16/1826FalseA recombinant cysteine protease and IgG endopeptidase originating from S. pyogenesPRD8297747ImlifidaseIMP00065/0000111INTRAVENOUS USEFalseIdefirix 11 mg powder for concentrate for solution for infusionimlifidaseSOLUTION FOR INFUSION322454110281347SOLUTION FOR INJECTION/INFUSIONPRD86568511Delandistrogene moxeparvovec-roklSOLUTION FOR INJECTION/INFUSIONSRP-9001DELANDISTROGENE MOXEPARVOVECSUB197789SAREPTA THERAPEUTICS INC10281347235539SAREPTA THERAPEUTICS INCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherDELANDISTROGENE MOXEPARVOVECSUB197789False1TrueEU/3/20/2250Adeno-associated virus serotype RH74 containing the human micro-dystrophin genePRD8656851SRP-9001MIA(IMP) 13160/0000137913SRP-9001-dystrophinAAVFalseTrue311INTRAVENOUS USEFalseDelandistrogene moxeparvovec-rokldelandistrogene moxeparvovecSOLUTION FOR INJECTION/INFUSIONAn Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association with Imlifidase in Subjects with Duchenne Muscular Dystrophy with Pre-existing Antibodies to rAAVrh74584544067e79be4-4d63-4f20-af34-5401b3a6f746Estudio abierto de liberación génica sistémica para evaluar la seguridad, la tolerabilidad y la expresión de SRP-9001 en asociación con imlifidasa en sujetos con distrofia muscular de Duchenne con anticuerpos preexistentes contra rAAVrh747SpanishA Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)5845448687c5523-32b3-432b-a36d-a6f1f3396d20Estudio de terapia de transferencia génica para evaluar la seguridad y la eficacia del delandistrogén moxeparvovec (SRP-9001) después de una infusión de imlifidasa en participantes con distrofia muscular de Duchenne (DMD) con presencia confirmada de anticuerpos preexistentes frente al anticuerpos preexistentes frente al virus adenoasociado recombinante, serotipo (rAAVrh74)7SpanishSRP-9001-104361096NCT06241950False31This is a Category 1 (Phase 1 trial)7007480162Duchenne muscular dystrophy (DMD)5845426e238d2b5-41a6-4f17-b051-e2990cb018cbDistrofia Muscular de Duchenne7SpanishTrue10000001204720.1PTDuchenne muscular dystrophy gene carrier10052655100000004850False623176432317665231767102317687231765• Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content [Time Frame: Baseline, Week 12] • Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity [Time Frame: Baseline, Week 12] • Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF) [Time Frame: Baseline, Week 12] • Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration [Time Frame: Week 12]584544773ba93d2-4674-4365-a197-13dc8432283d• Cambio desde el momento basal en el nivel de expresión de la proteína distrofina producida a partir del delandistrogén moxeparvovec, determinado mediante un ensayo de inmunoelectrotransferencia Western blot ajustado por el contenido muscular [plazo: momento basal, semana 12]. • Cambio desde el momento basal en el nivel de expresión de la proteína distrofina producida a partir del delandistrogén moxeparvovec en muestras de biopsia de tejido muscular, determinado según la intensidad de las fibras en un ensayo de inmunofluorescencia [plazo: momento basal, semana 12]. • Cambio desde el momento basal en el nivel de expresión de la proteína distrofina producida a partir del delandistrogén moxeparvovec en muestras de biopsia de tejido muscular, determinado según el porcentaje de fibras con positividad para la distrofina en un ensayo de inmunofluorescencia (PDPF) [plazo: momento basal, semana 12]. • Concentración media de copias de vectores genómicos, determinada mediante una reacción en cadena de la polimerasa con muestras de biopsia de tejido muscular después de la administración de delandistrogén moxeparvovec [plazo: semana 12].7Spanish2625181Maximum Observed Plasma Concentration (Cmax) of Imlifidase [Time Frame: Up to Day 7]5845449b79a0c84-a770-447e-8438-9b22c4214b97Concentración plasmática máxima (Cmáx) observada de imlifidasa [plazo: hasta el día 7].7Spanish2625192Total IgG in Serum After Imlifidase Administration [Time Frame: Up to Week 12]5845450c78e573a-2d46-4f54-89b0-dd1ccaea81f1IgG total en suero después de la administración de imlifidasa [plazo: hasta la semana 12].7Spanish2625203rAAVrh74 Antibody Titers After Imlifidase Administration [Time Frame: Up to Hour 120]5845451a0a2643e-9af0-4b6a-82d4-e8c81b3323c3Valores de anticuerpos contra rAAVrh74 después de la administración de imlifidasa [plazo: hasta la hora 120].7Spanish2625214Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration [Time Frame: Up to Day 7]584545204539dbd-3bdf-4ce7-b8e3-6552c0dc772dConcentración de copias de vectores genómicos, determinada mediante una reacción en cadena de la polimerasa en suero después de la administración de delandistrogén moxeparvovec [plazo: hasta el día 7].7Spanish2625225Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [Time Frame: Up to Week 104]584545362c36ca8-b152-4de6-a015-1e7ef7479a19Número de participantes con un acontecimiento adverso surgido durante el tratamiento (AAST), un acontecimiento adverso de especial interés (AAEI) y un acontecimiento adverso grave (AAG) [plazo: hasta la semana 104].7Spanish5089961Ambulatory per protocol specified criteria.58454415dcde588-edad-4336-b9c0-0131decf2474Ser deambulante, según los criterios especificados en el protocolo.7Spanish5089972Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.58454422104f2e4-2aa0-45ac-982a-4a8ba222a440Tener un diagnóstico definitivo de DMD antes de la selección, en base a hallazgos clínicos documentados y pruebas genéticas confirmatorias.7Spanish5089983Ability to cooperate with motor assessment testing.5845443fadd293b-53d6-46ca-80d3-f6048d7155d8Ser capaz de cooperar durante las pruebas de evaluación motora.7Spanish5089994Has elevated rAAVrh74 antibody titers per protocol-specified requirements.5845444ca9c2d84-cd85-4324-ac03-7c3e0eda8974Tener valores elevados de anticuerpos contra rAAVrh74 según los requisitos especificados en el protocolo.7Spanish5090005A pathogenic frameshift mutation, nonsense mutation or premature stop codon or pathogenic variant in the DMD gene that is expected to lead to absence of dystrophin protein.584544598de0cf3-c7ba-42bd-a2db-ac7eeb18e8b3Tener una mutación patógena de desplazamiento del marco de lectura, una mutación interruptora o un codón finalizador prematuro, u otra variante patógena en el gen DMD que se espera que conlleve la ausencia de proteína distrofina.7Spanish5090016Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).5845446ce32f2dd-7af9-42e0-b5db-88156347e64bHaber recibido una dosis diaria estable de corticoesteroides orales durante un mínimo de 12 semanas antes de la selección y tener previsto que la dosis se mantenga constante durante todo el estudio (excepto posibles modificaciones para adaptarla a los cambios de peso).7Spanish8773021Previous treatment with imlifidase.584543674cdc2c7-2502-4271-8ddb-404c829497eaHaber recibido tratamiento previo con imlifidasa.7Spanish8773032Presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for receiving the study drugs or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.58454372c76d8d1-4e7d-4df5-87b7-b2882a9bced6Presentar cualquier otra enfermedad clínicamente significativa, como enfermedades cardíacas, pulmonares, hepáticas, renales, hematológicas, inmunitarias o del comportamiento; una infección, neoplasia maligna, enfermedad concomitante o necesidad de tratamiento farmacológico crónico que, en opinión del investigador, suponga riesgos innecesarios a la hora de recibir los fármacos del estudio; o una afección médica o circunstancia atenuante que, en opinión del investigador, podría afectar a la capacidad del participante de completar las pruebas o los procedimientos requeridos por el protocolo, o bien comprometer el bienestar, la seguridad o la interpretabilidad clínica del participante y sus resultados.7Spanish8773043Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.584543810ea8d9f-0e35-46c7-b191-af7558e6655cHaber recibido tratamiento génico, alguna medicación en investigación u otro tratamiento especificado en el protocolo dentro de los límites de tiempo especificados en el protocolo.7Spanish8773054Abnormality in protocol-specified diagnostic evaluations or laboratory tests. Note: Other inclusion or exclusion criteria could apply.58454394290b32e-f4f6-46bc-92f1-01e39efa700ePresentar alguna anomalía en las evaluaciones diagnósticas o pruebas analíticas especificadas en el protocolo. Nota: Pueden aplicarse otros criterios de inclusión o exclusión.7Spanish5929751Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content [Time Frame: Baseline, Week 12]True584542797471ffb-a0a9-4746-a39a-b7bc94ff46c9• Cambio desde el momento basal en el nivel de expresión de la proteína distrofina producida a partir del delandistrogén moxeparvovec, determinado mediante un ensayo de inmunoelectrotransferencia Western blot ajustado por el contenido muscular [plazo: momento basal, semana 12].7Spanish5929762Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity [Time Frame: Baseline, Week 12]True58454283f76e64a-bc4a-4797-a0bb-cb098709a6b5• Cambio desde el momento basal en el nivel de expresión de la proteína distrofina producida a partir del delandistrogén moxeparvovec en muestras de biopsia de tejido muscular, determinado según la intensidad de las fibras en un ensayo de inmunofluorescencia [plazo: momento basal, semana 12].7Spanish5929773Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF) [Time Frame: Baseline, Week 12]True5845429447bb0cf-bd36-496f-bfab-e99d14e668b0• Cambio desde el momento basal en el nivel de expresión de la proteína distrofina producida a partir del delandistrogén moxeparvovec en muestras de biopsia de tejido muscular, determinado según el porcentaje de fibras con positividad para la distrofina en un ensayo de inmunofluorescencia (PDPF) [plazo: momento basal, semana 12].7Spanish5929784Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration [Time Frame: Week 12]True58454303fcf5256-912d-4e81-ab25-1705bf8050f9• Concentración media de copias de vectores genómicos, determinada mediante una reacción en cadena de la polimerasa con muestras de biopsia de tejido muscular después de la administración de delandistrogén moxeparvovec [plazo: semana 12].7Spanish5929791Maximum Observed Plasma Concentration (Cmax) of Imlifidase [Time Frame: Up to Day 7]False5845431be42918d-265c-414f-b003-077e7be1b6baConcentración plasmática máxima (Cmáx) observada de imlifidasa [plazo: hasta el día 7].7Spanish5929802Total IgG in Serum After Imlifidase Administration [Time Frame: Up to Week 12]False584543245f61fe4-029b-43f3-a36b-7f873b85211eIgG total en suero después de la administración de imlifidasa [plazo: hasta la semana 12].7Spanish5929813rAAVrh74 Antibody Titers After Imlifidase Administration [Time Frame: Up to Hour 120]False5845433532d58a5-2584-4e4d-ba16-bc86831656f4Valores de anticuerpos contra rAAVrh74 después de la administración de imlifidasa [plazo: hasta la hora 120].7Spanish5929824Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration [Time Frame: Up to Day 7]False58454340b387c7a-2f61-4de3-9647-9e545d49f40eConcentración de copias de vectores genómicos, determinada mediante una reacción en cadena de la polimerasa en suero después de la administración de delandistrogén moxeparvovec [plazo: hasta el día 7].7Spanish5929835Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [Time Frame: Up to Week 104]False584543534266710-58ea-4460-9bf3-e189febd110bNúmero de participantes con un acontecimiento adverso surgido durante el tratamiento (AAST), un acontecimiento adverso de especial interés (AAEI) y un acontecimiento adverso grave (AAG) [plazo: hasta la semana 104].7Spanish2027-06-302024-01-1756140Sarepta Therapeutics, Inc21356322213565242421356425252PatientsFalseTrueTrue230786559911592573EU Institution/Body/Agency14European Medicines AgencyFalseTrueORG-100013412Active602937Domenico Scarlattilaan 6Domenico Scarlattilaan 6Amsterdam1083 HS2029NetherlandsTrueORG-10001341230787559913592575EEA National Competent authority16Federal Agency For Medicines And Health ProductsFalseTrueORG-100003913Active602939Galileelaan 5/03Galileelaan 5/03Sint-Joost-Ten-Node12102002BelgiumTrueORG-100003913196952022-000691-19A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy, Estudio abierto, de dos partes, de administración génica sistémica para evaluar la seguridad y la expresión de RO7494222 (SRP-9001) en sujetos menores de cuatro años con Distrofia Muscular de Duchenne, Uno studio in due parti, in aperto, sulla terapia genica sistemica per valutare la sicurezza e l’espressione di RO7494222 (SRP-9001) in soggetti di età inferiore a quattro anni affetti da distrofia muscolare di Duchenne, Etude en 2 parties, en ouvert, de transfert de gène par voir systémique, évaluant la sécurité et l'expression de RO7494222 (SRP-9001) chez l'enfant âgé de moins de quatre ans présentant une dystrophie musculaire de Duchenne OPTIONALOPTIONALFalse196962020-002372-13A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION) , Étude de phase 3, internationale, randomisée, en double aveugle, contrôlée contre placebo, de thérapie génique systémique, destinée à évaluer la sécurité d’emploi et l’efficacité du SRP9001 chez des patients non ambulatoires et ambulatoires atteints de dystrophie musculaire de Duchenne (ENVISION), Studio di fase 3, internazionale, randomizzato, in doppio cieco, controllato verso placebo, sulla terapia di trasferimento sistemico di geni, per valutare la sicurezza e l’efficacia di SRP-9001 in soggetti deambulanti e non deambulanti con distrofia muscolare di Duchenne (ENVISION), Estudio de fase 3, multinacional, aleatorizado, en doble ciego y controlado con placebo, del tratamiento mediante transferencia génica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos, no ambulatorios y ambulatorios, con distrofia muscular de Duchenne (ENVISION)OPTIONALOPTIONALFalse196942019-003374-91A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) , Étude de phase 3, multinationale, randomisée, en double aveugle, contrôlée par placebo, sur le transfert systémique de gènes, visant à évaluer la sécurité d’emploi et l’efficacité du SRP-9001 chez des sujets atteints de dystrophie musculaire de Duchenne (EMBARK), Estudio de fase III multinacional, aleatorizado, doble ciego, controlado con placebo, de liberación génica sistémica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos con distrofia muscular de Duchenne (EMBARK), Studio di fase 3, internazionale, randomizzato, in doppio cieco, controllato verso placebo, sul rilascio sistemico di geni, per valutare la sicurezza e l’efficacia di SRP-9001 in soggetti con distrofia muscolare di Duchenne (EMBARK)OPTIONALOPTIONALFalseacceptable2025-02-062025-02-065Diseases [C] - Musculoskeletal Diseases [C05]16Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]80162Duchenne muscular dystrophy (DMD)True85532True255266PublicPatient RecruitmentRegistroEspanolDeEstudiosClinicos@druginfo.com+34900834223402436Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active255267ScientificPatient RecruitmentSareptAlly@sarepta.com+18887273782402436Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active367641558772591432Pharmaceutical company10PPD Global LimitedTrueTrueORG-100007533Active601658Granta Park, Great AbingtonGranta ParkGreat AbingtonCambridgeCB21 6GQ826United Kingdom+33182392401EUCTRInquiry.sm@ppd.comTrueORG-1000075335765331576534125765355576536657653775765389+33182392401EUCTRInquiry.sm@ppd.com402436Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active372835402436Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active398226215 1st Street215 1st StreetCambridge02142-1213840United StatesTrueORG-100006645TrueCommercial1This is a Category 1 (Phase 1 trial)8096085Diseases [C] - Musculoskeletal Diseases [C05]80960916Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]245618Delandistrogene moxeparvovec-roklMIA(IMP) 13160/000011Test322454110281347SOLUTION FOR INJECTION/INFUSIONPRD86568511Delandistrogene moxeparvovec-roklSOLUTION FOR INJECTION/INFUSIONSRP-9001DELANDISTROGENE MOXEPARVOVECSUB197789SAREPTA THERAPEUTICS INC10281347235539SAREPTA THERAPEUTICS INCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherDELANDISTROGENE MOXEPARVOVECSUB197789False1TrueEU/3/20/2250Adeno-associated virus serotype RH74 containing the human micro-dystrophin genePRD8656851SRP-9001MIA(IMP) 13160/0000137913SRP-9001-dystrophinAAVFalseTrue311INTRAVENOUS USEFalseDelandistrogene moxeparvovec-rokldelandistrogene moxeparvovecSOLUTION FOR INJECTION/INFUSION245619Idefirix 11 mg powder for concentrate for solution for infusionIMP00065/000011Test322455111507168POWDER FOR CONCENTRATE FOR SOLUTION FOR INFUSIONPRD8297747EU/1/20/1471/0012Idefirix 11 mg powder for concentrate for solution for infusionSOLUTION FOR INFUSIONIMLIFIDASESUB194312EUEMEA/H/C/004849HANSA BIOPHARMA AB11507168352813HANSA BIOPHARMA ABProtein - OtherProtein - OtherIMLIFIDASESUB194312IMMUNOGLOBULIN G DEGRADING ENZYME OF STREPTOCOCCUS PYOGENESHMED-IDESIDESL04AA41-5ImlifidaseFalse1TrueEU/3/16/1826FalseA recombinant cysteine protease and IgG endopeptidase originating from S. pyogenesPRD8297747ImlifidaseIMP00065/0000111INTRAVENOUS USEFalseIdefirix 11 mg powder for concentrate for solution for infusionimlifidaseSOLUTION FOR INFUSIONFalse1248983701637016134302027Ended2024-08-012024-08-01TrueSpain2024-08-29Ended328502023-12-152024-09-02328512023-12-152024-09-02421072024-01-172024-09-02421082024-01-172024-09-02722702024-01-172025-03-312025-03-31722712024-01-172025-03-312025-03-31True437202023-12-152024-09-022024-01-172025-03-31False5009837016Authorised2024-08-29T08:59:05.1124937437016Under evaluation2024-08-23T13:20:10.657967537016Halted2025-03-31T17:42:28.9310647237016Ended2025-10-21T08:38:38.8411Spainacceptable2024-08-29authorized_conditions2024-08-292024-08-297828157553452586089Hospital/Clinic/Other health care facility8Hospital Sant Joan De Deu BarcelonaFalseTrueORG-100023083Active595726Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain+34936009733andres.nascimento@sjd.esTrueORG-100023083923978AndresNascimento Osorio+34936009733andres.nascimento@sjd.es2Servicio de NeurologíaAuthorised21784INITIALAuthorised2024-512624-11-00Ended2024-08-23acceptable_conditions2024-08-291248983701637016Spain2027acceptable2024-08-29authorized_conditions2024-08-29EndedSpainEnded2024-08-29Authorised2024-08-2937016SpainEndedINFalse354502178437016Spain2024-08-29T08:59:05.305authorized_conditionsacceptabledecision12489832109INITIALTrue41643SUBSTANTIAL MODIFICATIONAuthorised2024-512624-11-00Ended2024-11-25acceptable2025-02-062025-02-0637016SpainEndedSM-1PART_IFalse567094164337016Spain2025-02-06T12:51:03.736authorizeddecision65720SUBSTANTIAL MODIFICATIONTrue51098NON SUBSTANTIAL MODIFICATIONAuthorised2024-512624-11-00Ended2025-03-18acceptable2025-02-062025-03-1837016SpainEndedNSM-1PART_IFalse627265109837016Spain2025-03-18T20:34:35.958authorizeddecision78738NON SUBSTANTIAL MODIFICATIONTrueSpain370162024-08-29T08:59:05.3052024-08-29T08:59:05.3058True2022-003407-1537016TH-771652025-03-314Safety related (clinical or pre-clinical results)FalseFalse7Study management relatedFalseFalseTrueIn response to the post marketing Acute liver Failure (ALF) safety event communicated through a Dear Investigator Letter (DIL) enrollment and dosing is temporary halted at the request of EU regulators.Subject safety reasonsTrue2025-03-31T17:42:28The Sponsor will continue patient safety monitoring for already enrolled patients and continue recording data.False2025-03-3137016SpainES37016SpainSTART_OF_TRIAL2023-12-15START_OF_RECRUITMENT2024-01-17END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-31EARLY_TERMINATION2025-10-101Sponsor DecisionTrue53882024-512624-11-00Sarepta_SRP-9001-104_CT Summary Result_04Mar2026_PublicSubmittedSummary of ResultsFinalTrue1275632026-04-07T09:53:56.6489552026-04-03T21:38:09.9332026-04-07T09:53:5613430SUM-12750753892024-512624-11-00Plain Language SummarySubmittedLaypersons Summary of ResultsFinalTrue1275082026-04-07T09:54:05.3632222026-04-03T12:23:10.9892026-04-07T09:54:0513430SUM-127508D1_ Sarepta_SRP_9001-104_2024-512624-11-00_LOAC_Publicda35b560-6656-442d-bc1f-4862bcc65d7f104Protocol (for publication)PDF7873811D1_Sarepta_SRP_9001-104_2024-512624-11-00_Public2b2c9db6-3f62-4322-8eef-41f35b542add104Protocol (for publication)PDF787383.01D1_Sarepta_SRP-9001-104 _Protocol Synopsis_2024-512624-11-00_ENG_Public0321ee38-93ac-49ed-bb51-b031dcfb7a877Synopsis of the protocol (for publication)PDF7873811D1_Sarepta_SRP-9001-104 _Protocol Synopsis_2024-512624-11-00_ESP_Public957cd1fa-4d05-411e-9062-933b297ee7cf7Synopsis of the protocol (for publication)PDF7873811Sarepta_SRP-9001-104_Plain Language Summary_ENG_Public47521d40-d425-4b5e-b701-08d089bbde8046Laypersons summary of results (for publication)PDF5389n/a1Sarepta_SRP-9001-104__Plain Language Summary_ESP_Publicf8f5f397-30d4-4b92-8ebb-a29215e24f1446Laypersons summary of results (for publication)PDF5389N/A1Sarepta_SRP-9001-104_CT Summary Result_Public6d44bc6f-1b62-41d5-b7c0-e91c062c3b34103Summary of results (for publication)PDF5388N/A1EEA1 2023-509901-57-006HaltedA TWO-PART, OPEN-LABEL SYSTEMIC GENE DELIVERY STUDY TO EVALUATE THE SAFETY AND EXPRESSION OF RO7494222 (SRP‑9001) IN SUBJECTS UNDER THE AGE OF FOUR WITH DUCHENNE MUSCULAR DYSTROPHYBN43881Duchenne Muscular DystrophyItaly:6Belgium:6Germany:6France:6Spain:606/08/2024DE: 13/08/2024, BE: 08/08/2024, FR: 09/08/2024, ES: 06/08/2024, IT: 23/09/2024Diseases [C] - Musculoskeletal Diseases [C05]F. 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publication)PDF798161.01Italy196782024-09-23T00:00:002025-03-13T09:08:27.2246Belgium196752024-08-08T12:06:56.3132025-03-12T15:28:52.7066Germany196772024-08-13T14:02:50.5692025-03-17T10:29:12.3336France196762024-08-09T16:53:17.1152025-03-14T16:08:41.8666Spain196792024-08-06T13:11:00.3152025-03-12T08:40:19.917619675TH-771262025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueThe enrollment and one-time dosing in the BN43881 clinical trial has been placed on temporary halt until final assessment of the acute liver failure case has been madeReasons of subject safetyTrue2025-03-31T17:37:43The Sponsor will continue patient safety monitoring for already enrolled patients and will continue recording data.False2025-03-3119675BelgiumBE19677TH-771242025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueThe enrollment and one-time dosing in the BN43881 clinical trial has been placed on temporary halt until final assessment of the acute liver failure case has been madeReasons of subject safetyTrue2025-03-31T17:36:39The Sponsor will continue patient safety monitoring for already enrolled patients and will continue recording data.False2025-03-3119677GermanyDE19676TH-771252025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueThe enrollment and one-time dosing in the BN43881 clinical trial has been placed on temporary halt until final assessment of the acute liver failure case has been madeReasons of subject safetyTrue2025-03-31T17:37:17The Sponsor will continue patient safety monitoring for already enrolled patients and will continue recording data.False2025-03-3119676FranceFR19679TH-771222025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueThe enrollment and one-time dosing in the BN43881 clinical trial has been placed on temporary halt until final assessment of the acute liver failure case has been madeReasons of subject safetyTrue2025-03-31T17:33:18The Sponsor will continue patient safety monitoring for already enrolled patients and will continue recording data.False2025-03-3119679SpainES19678TH-771232025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueThe enrollment and one-time dosing in the BN43881 clinical trial has been placed on temporary halt until final assessment of the acute liver failure case has been madeReasons of subject safetyTrue2025-03-31T17:34:54The Sponsor will continue patient safety monitoring for already enrolled patients and will continue recording data.False2025-03-3119678ItalyIT19675BelgiumSTART_OF_TRIAL2024-05-28START_OF_RECRUITMENT2024-05-31END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3119676FranceSTART_OF_TRIAL2024-06-19START_OF_RECRUITMENT2024-06-27END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3119677GermanySTART_OF_TRIAL2024-02-09START_OF_RECRUITMENT2024-03-25END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3119678ItalySTART_OF_TRIAL2024-04-23START_OF_RECRUITMENT2024-05-14END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-3119679SpainSTART_OF_TRIAL2023-10-26START_OF_RECRUITMENT2023-11-02END_OF_RECRUITMENT2025-03-31TEMPORARY_HALT2025-03-312023-509901-57-006A TWO-PART, OPEN-LABEL SYSTEMIC GENE DELIVERY STUDY TO EVALUATE THE SAFETY AND EXPRESSION OF RO7494222 (SRP‑9001) IN SUBJECTS UNDER THE AGE OF FOUR WITH DUCHENNE MUSCULAR DYSTROPHYBN4388126/10/2023Duchenne Muscular DystrophyItaly:6Belgium:6Germany:6France:6Spain:606/08/2024DE: 13/08/2024, BE: 08/08/2024, FR: 09/08/2024, ES: 06/08/2024, IT: 23/09/2024Diseases [C] - Musculoskeletal Diseases [C05]F. Hoffmann-La Roche AGPharmaceutical companyTherapeutic exploratory (Phase II)Change in quantity of SRP-9001-dystrophin protein expression from baseline to Week 12 as measured by Western blotDelandistrogene moxeparvovec-rokl430-17 yearsMale315Incidence of treatment-emergent adverse events, Incidence of serious adverse events, Incidence of adverse events of special interest, Clinically significant changes in vital signs and physical examination findings, Clinically significant changes in safety laboratory assessments, ECGs, and ECHOsNo26/03/202524/04/20252023-509901-57-00Halted2023-10-262024-08-06T13:11:00.3152025-04-24T20:48:30.9337133376798166100000000556United Kingdom826GBGBRTrue327149110281347SOLUTION FOR INJECTION/INFUSIONPRD86568511Delandistrogene moxeparvovec-roklSOLUTION FOR INJECTION/INFUSIONSRP-9001DELANDISTROGENE MOXEPARVOVECSUB197789SAREPTA THERAPEUTICS INC10281347235539SAREPTA THERAPEUTICS INCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherDELANDISTROGENE MOXEPARVOVECSUB197789True1TrueEU/3/20/2250vector genomes (vg)/mL13300000000000vector genomes (vg)/mL1330000000000011PRD8656851SRP-9001Roche Pharma AG - DE_BW_01_MIA_2020_0096-DE_BW_013846EMA/CAT/113473/20193Delandistrogene moxeparvovec dystrophinAAVFalseTrue38INTRAVENOUS USEFalseDelandistrogene moxeparvovec-rokldelandistrogene moxeparvovecSOLUTION FOR INJECTION/INFUSIONA TWO-PART, OPEN-LABEL SYSTEMIC GENE DELIVERY STUDY TO EVALUATE THE SAFETY AND EXPRESSION OF RO7494222 (SRP‑9001) IN SUBJECTS UNDER THE AGE OF FOUR WITH DUCHENNE MUSCULAR DYSTROPHY59502838eab5c01-4487-4705-8b8b-2e645b62e9a8Estudio abierto, de dos partes, de administración génica sistémica para evaluar la seguridad y la expresión de RO7494222 (SRP-9001) en sujetos menores de cuatro años con Distrofia Muscular de Duchenne7Spanish59502848eab5c01-4487-4705-8b8b-2e645b62e9a8Étude en deux parties, en ouvert, de transfert de gène par voie systémique, évaluant la sécurité et l'expression du RO7494222 (SRP-9001) chez l'enfant âgé de moins de quatre ans présentant une dystrophie musculaire de Duchenne10FrenchStudy to assess the safety and expression of RO7494222 (SRP-9001) in participants under the age of 4 with Duchenne muscular dystrophy5950302d25c671a-8cc9-415a-a619-1f96b6698fafEtude évaluant la sécurité et l'expression de RO7494222 (SRP-9001) chez les particpants de moins de quatre ans présentant une dystrophie musculaire de Duchenne10French5950301d25c671a-8cc9-415a-a619-1f96b6698fafEstudio para evaluar la seguridad y la expresión de RO7494222 (SRP-9001) en sujetos menores de cuatro años con Distrofia Muscular de Duchenne7SpanishBN43881False42This is a phase 2 clinical trial falling under Category 27113881387Duchenne Muscular DystrophyTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False10000001204720.1PTDuchenne muscular dystrophy gene carrier10052655100000004850False723538513Immunogenicity23538352353844235382To evaluate the safety of delandistrogene moxeparvovec59502996c216fc2-536f-4c40-b77b-a8eb53ab8370Évaluer la sécurité du délandistrogène moxéparvovec10French59503006c216fc2-536f-4c40-b77b-a8eb53ab8370Evaluar la seguridad del delandistrogén moxeparvovec7Spanish2664641To evaluate SRP-9001-dystrophin expression from delandistrogene moxeparvovec at 12 weeks as measured by Western blot of biopsied muscle tissue5950304c7e29e7f-a796-48a1-9413-fc174d943025Évaluer l'expression de la SRP-9001- dystrophine liée au délandistrogène moxéparvovec à 12 semaines, d'après les résultats de l'analyse par Western blot d'échantillons de biopsie de tissu musculaire.10French5950303c7e29e7f-a796-48a1-9413-fc174d943025Evaluar la expresión de distrofina SRP-9001 como resultado del delandistrogén moxeparvovec a las 12 semanas, en su medición mediante inmunoelectrotransferencia (Western blot) en una biopsia de tejido muscular7Spanish5167311Signed Informed Consent Form59502853eb3e68c-274d-424b-a508-8b7003d80aa7Firma del documento de consentimiento informado7Spanish59502863eb3e68c-274d-424b-a508-8b7003d80aa7Signature du formulaire de consentement éclairé10French5167322Signed Assent Form when appropriate, as determined by patient's age and individual site and country standards5950287b5e974c7-24e5-4cf4-a6e6-3f2839162bdcSignature du formulaire de consentement éclairé pour mineur (formulaire d'assentiment) le cas échéant, en fonction de l'âge du patient et des normes du pays et de chaque centre d'étude10French5950288b5e974c7-24e5-4cf4-a6e6-3f2839162bdcFirma del documento de asentimiento cuando proceda, según la edad del paciente y las normas de cada centro y país7Spanish5167333Male at birth5950289cb6d55a9-d8dd-4eb7-89fa-68407522a8c2Varón al nacer7Spanish5950290cb6d55a9-d8dd-4eb7-89fa-68407522a8c2Sexe masculin à la naissance10French5167344Meets the following age requirements at the time of study drug infusion: – For Cohort A: 3 years of age – For Cohort B: 2 years of age – For Cohort C: >6 months to <2 years of age – For Cohort D: ≤6 months of age Note: To ensure that cohort-specific age criteria are met at the time of dosing, at the time of signing the Informed Consent Form participants should be approximately 1 month younger than the maximum age to qualify for a cohort that is actively enrolling.59502929ce3a1a8-0b3e-41b5-ba9a-3230d8724bdaRépondant aux critères d'âge suivants au moment de la perfusion du médicament à l'étude : – Pour la cohorte A : 3 ans – Pour la cohorte B : 2 ans – Pour la Cohorte C : > 6 mois à < 2 ans – Pour la Cohorte D : ≤6 mois Remarque : Pour garantir que les critères d'âge spécifiques à la cohorte sont respectés au moment de l'administration du traitement, au moment de la signature du formulaire de consentement éclairé, les participants devront être âgés d'environ un mois de moins que l'âge maximum exigé pour la qualification dans une cohorte en cours de recrutement actif.10French59502919ce3a1a8-0b3e-41b5-ba9a-3230d8724bdaCumplimiento de los siguientes requisitos de edad en el momento de la infusión del fármaco del estudio: - En la Cohorte A: 3 años de edad - En la Cohorte B: 2 años de edad - En la Cohorte C: >6 meses a <2 años de edad - En la Cohorte D: </=6 meses de edad Nota: Para garantizar que se cumplan los criterios de edad específicos de la cohorte en el momento de la administración, cuando se firme el documento de consentimiento informado los participantes deberán tener aproximadamente 1 mes menos de la edad máxima permitida en una cohorte que se encuentre abierta a la inclusión.7Spanish5167355Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. Genetic report must describe a frameshift deletion, frameshift duplication, premature stop ("nonsense"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 79 (inclusive) that is expected to lead to absence of dystrophin protein. – Mutations between or including exons 1-17 are not eligible. – In-frame deletions, in-frame duplications, and variants of uncertain significance ("VUS") are not eligible.59502933cd2265d-0a73-4593-b325-ec3367bcac43Tener un diagnóstico definitivo de distrofia muscular de Duchenne (DMD) antes de la selección, basado en la documentación de los hallazgos clínicos y las pruebas genéticas confirmatorias previas mediante un método de diagnóstico clínico. El informe genético debe describir una deleción con desplazamiento del marco de lectura, una duplicación con desplazamiento del marco de lectura, una parada prematura (“sin sentido”), una mutación del sitio de empalme canónico u otra variante patogénica en el gen de la DMD contenida en su totalidad entre los exones 18 a 79 (inclusive) que se espere que comporte la ausencia de la proteína distrofina. – Las mutaciones entre los exones 1-17 o que los incluyan no son elegibles. – Las deleciones dentro del marco de lectura, las duplicaciones dentro del marco de lectura y las variantes de significado incierto (variants of uncertain significance, VUS) no son elegibles.7Spanish59502943cd2265d-0a73-4593-b325-ec3367bcac43Diagnostic définitif de DMD avant la sélection, fondé sur la documentation des observations cliniques et des analyses génétiques de confirmation effectuées précédemment avec un test génétique diagnostique clinique. Le compte-rendu génétique doit décrire une délétion avec décalage de cadre, une duplication avec décalage de cadre, un arrêt prématuré (« non sens »), une mutation par épissage canonique, ou tout autre variant pathogène du gène DMD totalement contenu entre les exons 18 à 79 (inclus) conduisant à une absence de la protéine dystrophine. – Les mutations entre les exons 1 à 17 ne sont pas éligibles. – Les délétions sans décalage de cadre, duplications sans décalage de cadre ou variant de signification incertaine (VSI) ne sont pas éligibles.10French5167366Able to cooperate with age-appropriate motor assessment testing in the opinion of the investigator.59502962569552b-c38e-4236-b474-5121bf084b5bCapaz de colaborar en las pruebas de evaluación motora adecuadas a su edad, en opinión del investigador7Spanish59502952569552b-c38e-4236-b474-5121bf084b5bCapacité de coopérer pour les tests d'évaluation motrice appropriés par rapport à l'âge, selon l'avis de l'investigateur.10French5167377Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all protocol requirements.5950298d252fe40-3e85-40fc-81a2-77301274cc85Tener uno o más progenitores o tutores legales que sean capaces de entender y cumplir el calendario de visitas del estudio y todos los requisitos del protocolo7Spanish5950297d252fe40-3e85-40fc-81a2-77301274cc85Participant ayant un ou des parent(s) ou aidant(s) légal(aux) capable(s) de comprendre et de respecter le calendrier des visites de l'étude et toutes les exigences du protocole.10French8899291Has elevated anti rAAVrh74 antibody titers as determined by an investigational Elecsys anti-rAAVrh74 antibody assay obtained within 31 days of the infusion day5950245227d6072-c409-4321-b93d-7992a66ce563Tener títulos elevados de anticuerpos anti rAAVrh74 según lo determinado por un ensayo experimental de anticuerpos Elecsys anti-rAAVrh74 obtenido dentro de los 31 días posteriores al día de perfusión7Spanish5950246227d6072-c409-4321-b93d-7992a66ce563Présenter des titres élevés d'anticorps anti-rAAVrh74 déterminés par un dosage expérimental des anticorps anti-rAAVrh74 Elecsys réalisé au cours des 31 jours précédant le jour de la perfusion10French88993010Symptomatic infection (e.g., upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.59502474465449f-faa5-4326-9d6a-45b4581f423dPrésence d'une infection symptomatique (par exemple, infection des voies respiratoires supérieures, pneumonie, pyélonéphrite, méningite) dans les 4 semaines avant le Jour 110French59502484465449f-faa5-4326-9d6a-45b4581f423dInfección sintomática (p. ej., de las vías respiratorias superiores, neumonía, pielonefritis, meningitis) en un plazo de 4 semanas antes del día 1.7Spanish88993111Cohort A and B: Positive serology testing for HIV 1 and/or 2, hepatitis C, or hepatitis B5950249b2948f4e-0e61-4c47-8b83-06d08a2d8cbaCohortes A y B: Pruebas serológicas positivas de los virus de la inmunodeficiencia humana 1 y/o 2, de la hepatitis C o de la hepatitis B7Spanish5950250b2948f4e-0e61-4c47-8b83-06d08a2d8cbaCohortes A et B: Résultat positif à l'analyse sérologique pour le VIH1 et/ou 2, l'hépatite C ou l'hépatite B10French88993212Cohort C and D: – Born prematurely (before completion of gestation at 37 weeks) or relevant pregnancy complications in the opinion of the investigator – Participant's mother: Serological evidence of current, chronic, or active HIV (1 and/or 2), hepatitis B, or hepatitis C infection – Participant's mother if breastfeeding: o Clinical signs of acute CMV infection with confirmation by CMV PCR (urine) o Clinically significant abnormal liver function (GGT, AST, ALT, ALP, total bilirubin, GLDH) indicative of infectious hepatitis o Clinically significant illness or acute infection indicative of hepatotropic virus infection (e.g., CMV, EBV, VZV, etc.) within 6 weeks prior to Day 1 o Known contact with an infected person with acute or active hepatitis within 12 weeks prior to Day 1595025272f5fce7-a1ac-4b6c-b591-a771c34ed66fCohortes C et D: –Naissance prématurée (avant la fin de 37semaines de grossesse à 37semaines) ou complications significatives de la grossesse, selon l'avis de l'investigateur –Mère du participant: Signes sérologiques d'infection en cours, chronique ou active par VIH 1 et/ou 2, le virus de l'hépatite B ou le virus de l'hépatite C –Mère du participant si allaitement: oSignes cliniques d'infection aiguë par le CMV avec confirmation par analyse PCR du CMV (échantillon urinaire) oAnomalies cliniquement significatives de la fonction hépatique (GGT, ASAT, ALAT, PAL, bilirubine totale, GLDH) indiquant une hépatite infectieuse oPathologie cliniquement significative ou infection aiguë indiquant une infection par virus hépatotrope (par exemple, CMV, EBV, VZV, etc) dans les 6 semaines avant le Jour1 oContact connu avec une personne atteinte d'hépatite aiguë ou active dans les 12 semaines avant le Jour110French595025172f5fce7-a1ac-4b6c-b591-a771c34ed66fCohortes C y D: – Nacido prematuramente (antes de completar la gestación a las 37 semanas) o con complicaciones relevantes del embarazo, a juicio del investigador – Madre del participante: Evidencia serológica de infección actual, crónica o activa por los virus del VIH (de tipo 1 y/o 2), de la hepatitis B o de la hepatitis C – Madre del participante en caso de lactancia materna: o Signos clínicos de infección aguda por citomegalovirus con confirmación por PCR (orina) o Alteración funcional hepática clínicamente importante (GGT, AST, ALT, fosfatasa alcalina, bilirrubina total, GLDH) indicativa de hepatitis infecciosa o Enfermedad o proceso agudo clínicamente importante que indique infección por virus hepatotropos (p. ej., citomegalovirus, virus de Epstein-Barr, virus de la varicela-zóster, etc.) en un plazo de 6 semanas antes del día 1 o Contacto conocido con una persona con hepatitis infecciosa aguda o activa en un plazo de 12 semanas antes del día 17Spanish88993313Demonstrates cognitive delay or impairment that could confound motor development in the opinion of the investigator.59502545a7e8601-827e-4c83-9cba-44e098fe5696Demostrar retraso o deterioro cognitivo que pueda suponer un factor de confusión en el desarrollo motor, en opinión del investigador.7Spanish59502535a7e8601-827e-4c83-9cba-44e098fe5696Présence d'un retard cognitif ou d'une insuffisance cognitive susceptible d'interférer sur le développement moteur, selon l'avis de l'investigateur10French88993414Treatment with any of the following therapies during the specified time periods: – Any time: o Gene therapy o Cell-based therapy (e.g., stem cell transplantation) o CRISPR/Cas9, or any other form of gene editing – Within 12 weeks of Day 1 and any time during the study: o Use of human growth factor or vamorolone – Within 6 months of Day 1 and any time during the study: o Any investigational medication o Any treatment designed to increase dystrophin expression (e.g., Translarna™, EXONDYS 51™, VILTEPSO™)5950256291f971a-268d-4fa1-a1f9-b4342488ae58Tratamiento con cualquiera de las siguientes terapias durante los periodos especificados: – En cualquier momento: o Terapia génica o Terapia celular (p. ej., trasplante de células madre) o CRISPR/Cas9 o cualquier otra forma de edición de genes – En un plazo de 12 semanas respecto al día 1 y en cualquier momento durante el estudio: o Administración de factor de crecimiento humano o vamorolona – En un plazo de 6 meses respecto al día 1 y en cualquier momento durante el estudio: o Cualquier medicamento en investigación o Cualquier tratamiento diseñado para aumentar la expresión de la distrofina (p. ej., Translarna™, EXONDYS 51™, VILTEPSO™)7Spanish5950255291f971a-268d-4fa1-a1f9-b4342488ae58Administration d'un des traitemen suivants dans les limites temporelles spécifiées: –À tout moment: oThérapie génique oThérapie cellulaire (par exemple, greffe de cellules souches) oCRISPR/Cas9, ou toute autre forme d'édition des gènes –Dans les 12 semaines avant le Jour1 et à tout moment pendant l'étude: oUtilisation de facteur de croissance humain ou de vamorolone –Dans les 6 mois avant le Jour1 et à tout moment pendant l'étude: oTout médicament expérimental oTout traitement conçu pour augmenter l'expression de la dystrophine (par exemple, Translarna™, EXONDYS 51™, VILTEPSO™)10French88993515Has received a live virus vaccine or mRNA vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit, or expects to receive a vaccination that cannot be reasonably delayed to accommodate concomitant corticosteroid administration during the first 3 months after Day 1.5950258e7b7b56b-9b40-4192-9718-8d01eb8700edHaber recibido una vacuna de virus vivos o una vacuna de ácido ribonucleico mensajero en un plazo de 4 semanas o una vacuna inactiva en un plazo de 2 semanas respecto a la visita del día 1, o esperar recibir una vacuna que no pueda retrasarse razonablemente para adaptarse a la administración concomitante de corticosteroides durante los 3 primeros meses después del día 1.7Spanish5950257e7b7b56b-9b40-4192-9718-8d01eb8700edAdministration d'un vaccin par virus vivant ou d'un vaccin à ARNm dans les 4semaines ou d'un vaccin inactivé dans les 2semaines précédant la visite du Jour1 ou administration prévue d'une vaccination ne pouvant pas être raisonnablement différée pour permettre l'administration concomitante de corticoïdes pendant les 3 premiers mois après le Jour110French88993616Has abnormal laboratory values considered clinically significant including but not limited to: – GGT >2 x upper limit of normal (ULN) – GLDH > ULN – Total bilirubin >ULN. o Elevations in total bilirubin confirmed to be due to Gilbert's syndrome are not exclusionary. – White blood cell count >18,500 per µl – Platelets ≤ 150,000 per µl5950260ac99c281-3bd2-45ff-aa93-8915fc5ae199Presentar valores de laboratorio anómalos que se consideren clínicamente importantes, tales como, entre otros, los siguientes: – GGT >2 veces el límite superior de la normalidad - GLDH > límite superior de la normalidad – Bilirrubina total >límite superior de la normalidad o La elevación de la bilirrubina total confirmada por el síndrome de Gilbert no es motivo de exclusión – Cifra de leucocitos >18.500 por µl – Plaquetas </=150.000 por µl7Spanish5950259ac99c281-3bd2-45ff-aa93-8915fc5ae199Anomalies considérées comme cliniquement significatives des paramètres biologiques, notamment: –GGT> 2 x limite supérieure de la normale (LSN) - GLDH> LSN –Bilirubine totale> LSN oLes élévations de la bilirubine totale confirmées comme étant dues à un syndrome de Gilbert ne constituent pas un critère de non-inclusion –Numération des globules blancs (GB)>18 500 par µl –Plaquettes ≤ 150 000 par µl10French88993717In the opinion of the investigator, the participant is not likely to be compliant with the study protocol.59502628cf3d659-06ec-41aa-b2f6-74652645a7edNo es probable que el participante cumpla el protocolo del estudio, en opinión del investigador.7Spanish59502618cf3d659-06ec-41aa-b2f6-74652645a7edSelon l'avis de l'investigateur, le participant n'est pas susceptible de respecter le protocole10French88993818Family does not want to disclose participant's study participation with general practitioner or primary care physician and other medical providers.5950264dc4295d2-3de9-4205-9595-18477b02ce8aRefus de la famille du participant d'informer le médecin généraliste ou médecin de soins primaires et autres professionnels médicaux de la participation à cette étude10French5950263dc4295d2-3de9-4205-9595-18477b02ce8aFamilia que no desea revelar la participación del participante en el estudio a su médico de cabecera o médico de atención primaria y a otros profesionales sanitarios.7Spanish88993919Positive COVID-19 test (antigen or PCR) on Day 1 prior to infusion5950265255b5e54-0b96-44d3-85c1-c83134bc8b4ePrueba de COVID-19 positiva (antígenos o PCR) el día 1 antes de la infusión7Spanish5950266255b5e54-0b96-44d3-85c1-c83134bc8b4eRésultat positif au test de dépistage de la COVID-19 (test antigénique ou PCR) le Jour 1 avant la perfusion10French8899402Poor peripheral venous access, which, in the opinion of the investigator, will lead to difficulty in venipuncture for the purposes of protocol-mandated procedures.59502685141ecf5-29c0-4f71-8475-36c8bd728d9cAccès veineux périphérique de mauvaise qualité, selon l’investigateur, rendant difficile les ponctions veineuses nécessaires pour les procédures exigées par le protocole.10French59502675141ecf5-29c0-4f71-8475-36c8bd728d9cAcceso venoso periférico deficiente, que, en opinión del investigador, provocará dificultades en las venopunciones precisas para los procedimientos exigidos por el protocolo.7Spanish8899413Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline.595027050c2282f-4898-4b4f-8a55-6784ec13cb36Traitement régulier par corticoïdes oraux comme traitement de la DMD ou prévision de traitement par corticoïdes oraux comme traitement de la DMD dans l'année suivant l'inclusion10French595026950c2282f-4898-4b4f-8a55-6784ec13cb36Recibir con regularidad corticosteroides orales como tratamiento para la DMD o tener previsto recibir corticosteroides orales como tratamiento para la DMD en el plazo de 1 año respecto al momento basal.7Spanish8899424Major surgery within 3 months prior to Day 1 or planned surgery during Part 1 of the study.5950272d081c9a2-e00a-4e39-86a7-1832a37960a7Cirugía mayor en el plazo de los 3 meses anteriores al día 1 o cirugía prevista durante la Parte 1 del estudio.7Spanish5950271d081c9a2-e00a-4e39-86a7-1832a37960a7Intervention chirurgicale majeure dans les 3 mois avant le Jour1 ou prévision d'intervention chirurgicale pendant la Partie110French8899435Any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that creates unnecessary risks to participate in the study in the opinion of the investigator.5950274212364c0-3373-41e7-971c-3abb57e4a803Cualquier otra enfermedad clínicamente importante, entre otras, dolencias cardiacas, pulmonares, hepáticas, renales, hematológicas, inmunológicas o del comportamiento, o infecciones o neoplasias malignas o enfermedades concomitantes o necesidad de tratamiento farmacológico prolongado que generen riesgos innecesarios para la participación en el estudio, a juicio del investigador.7Spanish5950273212364c0-3373-41e7-971c-3abb57e4a803Toute autre pathologie cliniquement significative, y compris cardiaque, pulmonaire, hépatique, rénale, hématologique, immunologique ou comportementale ou infection ou cancer ou pathologie concomitante ou exigence d'un traitement médicament chronique créant des risques inutiles en cas de participation à l'étude,selon l'avis de l'investigateur10French8899446Known hypersensitivity to delandistrogene moxeparvovec or any excipients of the formulation.5950276341de291-0861-4ec3-b4d9-7d4c1d4a4982Hipersensibilidad conocida al delandistrogene moxeparvovec o a cualquier excipiente de la formulación7Spanish5950275341de291-0861-4ec3-b4d9-7d4c1d4a4982Hypersensibilité connue au delandistrogène moxeparvovec ou à l'un des excipients de la formulation10French8899457Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol-required testing or procedures, or compromise the participant's well-being or safety, or clinical interpretability.5950278237da02c-aabe-4c98-ab60-5f349ef61d03Situation médicale ou circonstance exténuante qui, selon l'avis de l'investigateur, est susceptible d'interférer sur la capacité du participant à respecter les évaluations ou procédures exigées par le protocole et/ou avoir des répercussions sur son bien-être ou sa sécurité ou l'interprétation des données cliniques du participant10French5950277237da02c-aabe-4c98-ab60-5f349ef61d03Proceso o circunstancia atenuante que, en opinión del investigador, pueda comprometer la capacidad del participante para cumplir las pruebas o procedimientos exigidos por el protocolo, o comprometer su bienestar o seguridad o la interpretabilidad clínica7Spanish8899468Left ventricular ejection fraction <50% on the screening echocardiogram (ECHO) or clinical signs and/or symptoms of cardiomyopathy5950279e580165e-581b-458e-ada6-53a06e159f9aFracción de eyección del ventrículo izquierdo <50% en el ecocardiograma de selección o signos y/o síntomas clínicos de miocardiopatía7Spanish5950280e580165e-581b-458e-ada6-53a06e159f9aFraction d'éjection ventriculaire gauche (FEVG) < 50 % sur l'échocardiographie de la sélection (ECHO) ou signes cliniques et/ou symptômes de cardiomyopathie10French8899479Known contact with acute or active hepatitis within 12 weeks or known contact with an infected person (e.g., suspected Epstein-Barr virus [EBV], Varicella zoster virus [VZV], parvovirus B19, human herpes virus 6, and cytomegalovirus [CMV]) within 6 weeks prior to Day 15950282ca49eea1-bd4d-4bb2-9eed-b0cf7c1df1c5Contact connu avec une personne atteinte d'hépatite aiguë ou active dans les 12 semaines avant le Jour1 ou contact connu avec une personne ayant une autre infection virale (par exemple, suspicion d'infection par le virus d'Epstein-Barr EBV], virus Varicella zona [VZV], parvovirus B19, herpèsvirus humain 6, et cytomégalovirus [CMV]) dans les 6 semaines avant le Jour110French5950281ca49eea1-bd4d-4bb2-9eed-b0cf7c1df1c5Contacto conocido con hepatitis aguda o activa en un plazo de 12 semanas o contacto conocido con una persona con infección (p. ej., sospecha de virus de Epstein-Barr, virus de la varicela zóster, parvovirus B19, virus del herpes humano 6 o citomegalovirus) en un plazo de 6 semanas antes del día 17Spanish6019721Incidence of treatment-emergent adverse eventsTrue59502342ebb8205-0916-4e3d-8e90-0b72f89bb3aeIncidence des événements indésirables apparaissant sous traitement10French59502332ebb8205-0916-4e3d-8e90-0b72f89bb3aeIncidencia de acontecimientos adversos surgidos durante el tratamiento7Spanish6019732Incidence of serious adverse eventsTrue5950236d1a0c183-91a7-4c3f-832f-5ca54c02d6faIncidence des événements indésirables graves10French5950235d1a0c183-91a7-4c3f-832f-5ca54c02d6faIncidencia de acontecimientos adversos graves7Spanish6019743Incidence of adverse events of special interestTrue5950237af323b76-d4da-489c-a508-ac14ee7e9ffdIncidence des événements indésirables d'intérêt particulier10French5950238af323b76-d4da-489c-a508-ac14ee7e9ffdIncidencia de acontecimientos adversos de especial interés7Spanish6019754Clinically significant changes in vital signs and physical examination findingsTrue595023953c24277-52d4-497d-a31b-d264675c511dModifications cliniquement significatives des signes vitaux et des résultats de l'examen clinique10French595024053c24277-52d4-497d-a31b-d264675c511dCambios clínicamente significativos en las constantes vitales y los hallazgos de la exploración física7Spanish6019765Clinically significant changes in safety laboratory assessments, ECGs, and ECHOsTrue59502412788d968-18c9-48d2-b8dc-885024c2c29aModifications cliniquement significatives des paramètres biologiques de sécurité, des électrocardiogrammes (ECG), des échocardiographies (ECHO)10French59502422788d968-18c9-48d2-b8dc-885024c2c29aCambios clínicamente significativos en las determinaciones de laboratorio con fines de seguridad, ECG y ecocardiogramas7Spanish6019771Change in quantity of SRP-9001-dystrophin protein expression from baseline to Week 12 as measured by Western blotFalse595024355c4505c-d765-4d10-b4b0-5c6a20088a0fModifications de la quantité d'expression protéique de SRP-9001-dystrophine à la Semaine 12, d'après les résultats de l'analyse par Western blot10French595024455c4505c-d765-4d10-b4b0-5c6a20088a0fCambio en la cantidad de expresión de la proteína distrofina SRP-9001 desde el momento basal hasta la semana 12, en su medición mediante Western blot7Spanish2032-11-302032-11-302023-07-0156965F. Hoffmann-La Roche Ltd21674022216742242421674123232PatientsFalseTrueTrue1Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/innovation/process/clinical-trials/data-sharing/).31324450809483102EU Institution/Body/Agency14European Medicines AgencyFalseTrueORG-100013412Active483010Domenico Scarlattilaan 6Domenico Scarlattilaan 6Amsterdam1083 HS2029NetherlandsTrueORG-1000134126699EMEA-002677-PIP01-19199962024-512626-28-00Sarepta Therapeutics Inc.A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)REQUIREDREQUIRED13432True38576031D1_ Agreement from other Sponsor SRP-9001 English BN43881336Agreement from another sponsor 01.001.06Englishec02d80e-665b-4efd-9bdd-595af4961fbfFalse2025-03-26True38576031385760322024-11-29T00:00:00.000Z1.002024-09-27T00:00:00.000Z1.0SM-1, v.1.0, 27Sep2024ec02d80e-665b-4efd-9bdd-595af4961fbfFalseFalse138576032367842151301738576031isSavedtrueFalsem_b11FalseTrueacceptable2025-03-122025-03-125Diseases [C] - Musculoskeletal Diseases [C05]81387Duchenne Muscular DystrophyTrue86803True259019PublicTrial Information System - TISLglobal.eudract@roche.com41616881111275703Pharmaceutical company10F. Hoffmann-La Roche AGTrueTrueORG-100001445Active259020ScientificTrial Information System - TISLglobal.eudract@roche.com41616881111275703Pharmaceutical company10F. Hoffmann-La Roche AGTrueTrueORG-100001445Active374109439545471797Pharmaceutical company10Parexel International (IRL) LimitedTrueTrueORG-100022780Active47057070 Sir John Rogerson's Quay70 Sir John Rogerson's QuayDublin 2D02 R2962017Ireland+3531439500Clinicaltrial.Enquiries@parexel.comTrueORG-10002278058656815865691258657025865715586572658657375865748+3531439500Clinicaltrial.Enquiries@parexel.com275703Pharmaceutical company10F. Hoffmann-La Roche AGTrueTrueORG-100001445Active271768275703Pharmaceutical company10F. Hoffmann-La Roche AGTrueTrueORG-100001445Active289737Grenzacherstrasse 124Grenzacherstrasse 124Basel4058756SwitzerlandTrueORG-100001445TrueCommercial2This is a phase 2 clinical trial falling under Category 28178975Diseases [C] - Musculoskeletal Diseases [C05]248986Delandistrogene moxeparvovec-roklRoche Pharma AG - DE_BW_01_MIA_2020_0096-DE_BW_011Test327149110281347SOLUTION FOR INJECTION/INFUSIONPRD86568511Delandistrogene moxeparvovec-roklSOLUTION FOR INJECTION/INFUSIONSRP-9001DELANDISTROGENE MOXEPARVOVECSUB197789SAREPTA THERAPEUTICS INC10281347235539SAREPTA THERAPEUTICS INCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherDELANDISTROGENE MOXEPARVOVECSUB197789True1TrueEU/3/20/2250vector genomes (vg)/mL13300000000000vector genomes (vg)/mL1330000000000011PRD8656851SRP-9001Roche Pharma AG - DE_BW_01_MIA_2020_0096-DE_BW_013846EMA/CAT/113473/20193Delandistrogene moxeparvovec dystrophinAAVFalseTrue38INTRAVENOUS USEFalseDelandistrogene moxeparvovec-rokldelandistrogene 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IAuthorised1415971967519675105622002Halted2024-07-292024-07-29FalseunwillingBelgium2024-08-08Halted341652024-05-282024-09-11341662024-05-282024-09-11441942024-05-312024-09-11441952024-05-312024-09-11722682024-05-312025-03-312025-03-31722692024-05-312025-03-312025-03-31True456302024-05-282024-09-112024-05-312025-03-31False4340619675Under evaluation2024-07-15T16:58:59.774747919675Authorised2024-08-08T12:06:55.717967419675Halted2025-03-31T17:37:43.5131Belgiumacceptable2025-02-14authorized_conditions2024-08-082024-08-083923109519750552281Hospital/Clinic/Other health care facility8Centre Hospitalier Regional De La CitadelleFalseTrueORG-100028257Active558541Boulevard Du Douzieme De Ligne 1Boulevard Du Douzieme De Ligne 1Liege40002002Belgium+3243218315laurent.servais@citadelle.beTrueORG-1000282571030136LaurentServais+3243218315laurent.servais@citadelle.be2354844, Centre de Référence des Maladies NeuromusculairesAuthorised1415991967619676105622012Halted2024-07-292024-07-29FalseunwillingFrance2024-08-09Halted341672024-06-192024-09-11341682024-06-192024-09-11441962024-06-272024-09-11441972024-06-272024-09-11722662024-06-272025-03-312025-03-31722672024-06-272025-03-312025-03-31True456322024-06-192024-09-112024-06-272025-03-31False4772119676Authorised2024-08-09T16:53:16.5244340719676Under evaluation2024-07-15T16:58:59.8227967319676Halted2025-03-31T17:37:17.6151Franceacceptable2025-02-12authorized_conditions2024-08-092024-08-091923111525422557982Hospital/Clinic/Other health care facility8Hopital Necker Enfants MaladesFalseTrueORG-100023257Active564876149 Rue De Sevres149 Rue De SevresParis750152012France330144495836isabelle.desguerre@aphp.frTrueORG-1000232571030138IsabelleDesguerre330144495836isabelle.desguerre@aphp.fr1service de 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InfantileAuthorised16169INITIALAuthorised2023-509901-57-00Halted2024-07-15acceptable_conditions2024-08-061234821967519675Belgium2002acceptable2024-08-06authorized_conditions2024-08-08HaltedBelgiumHalted2024-08-08Authorised1234831967619676France2012acceptable2024-08-08authorized_conditions2024-08-09HaltedFranceHalted2024-08-09Authorised1234841967719677Germany2013acceptable2024-08-09authorized_conditions2024-08-13HaltedGermanyHalted2024-08-13Authorised1234851967819678Italy2018acceptable2024-09-16authorized_conditions2024-09-23HaltedItalyHalted2024-09-23Authorised1234861967919679Spain2027acceptable2024-08-06authorized_conditions2024-08-06HaltedSpainHalted2024-08-06Authorised2024-08-0619675BelgiumHalted19676FranceHalted19677GermanyHalted19678ItalyHalted19679SpainHaltedINFalse389371616919678Italy2024-09-23T00:00:00authorized_conditionsacceptabledecision12348548838INITIALFalse327871616919675Belgium2024-08-08T12:06:56.313authorized_conditionsacceptabledecision12348248838INITIALFalse333201616919677Germany2024-08-13T14:02:50.569authorized_conditionsacceptabledecision12348448838INITIALFalse329801616919676France2024-08-09T16:53:17.115authorized_conditionsacceptabledecision12348348838INITIALFalse324971616919679Spain2024-08-06T13:11:00.315authorized_conditionsacceptabledecision12348648838INITIALTrue36784SUBSTANTIAL 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MODIFICATIONFalse617473678419675Belgium2025-03-12T15:28:52.706authorizedacceptabledecision14159756343SUBSTANTIAL MODIFICATIONFalse615643678419679Spain2025-03-12T08:40:19.917authorizedacceptabledecision14159856343SUBSTANTIAL MODIFICATIONTrue621913678419676France2025-03-14T16:08:41.866authorizedacceptabledecision14159956343SUBSTANTIAL MODIFICATIONFalse51787NON SUBSTANTIAL MODIFICATIONAuthorised2023-509901-57-00Halted2025-03-26acceptable2025-03-122025-03-2619677GermanyHalted19675BelgiumHalted19676FranceHalted19679SpainHalted19678ItalyHaltedNSM-1PART_IFalse638515178719677Germany2025-03-26T15:07:33.967authorizeddecision79816NON SUBSTANTIAL MODIFICATIONFalse638515178719675Belgium2025-03-26T15:07:33.967authorizeddecision79816NON SUBSTANTIAL MODIFICATIONFalse638515178719676France2025-03-26T15:07:33.967authorizeddecision79816NON SUBSTANTIAL MODIFICATIONFalse638515178719679Spain2025-03-26T15:07:33.967authorizeddecision79816NON SUBSTANTIAL MODIFICATIONTrue638515178719678Italy2025-03-26T15:07:33.967authorizeddecision79816NON SUBSTANTIAL MODIFICATIONFalseItaly196782024-09-23T00:00:002025-03-13T09:08:27.2246Belgium196752024-08-08T12:06:56.3132025-03-12T15:28:52.7066Germany196772024-08-13T14:02:50.5692025-03-17T10:29:12.3336France196762024-08-09T16:53:17.1152025-03-14T16:08:41.8666Spain196792024-08-06T13:11:00.3152025-03-12T08:40:19.9176True2022-000691-1919675TH-771262025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueThe enrollment and one-time dosing in the BN43881 clinical trial has been placed on temporary halt until final assessment of the acute liver failure case has been madeReasons of subject safetyTrue2025-03-31T17:37:43The Sponsor will continue patient safety monitoring for already enrolled patients and will continue recording data.False2025-03-3119675BelgiumBE19677TH-771242025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueThe enrollment and one-time dosing in the BN43881 clinical trial has been placed on temporary halt until final assessment of the acute liver failure case has been madeReasons of subject safetyTrue2025-03-31T17:36:39The Sponsor will continue patient safety monitoring for already enrolled patients and will continue recording data.False2025-03-3119677GermanyDE19676TH-771252025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueThe enrollment and one-time dosing in the BN43881 clinical trial has been placed on temporary halt until final assessment of the acute liver failure case has been madeReasons of subject safetyTrue2025-03-31T17:37:17The Sponsor will continue patient safety monitoring for already enrolled patients and will continue recording data.False2025-03-3119676FranceFR19679TH-771222025-03-314Safety related (clinical or pre-clinical results)FalseFalseTrueThe enrollment and one-time dosing in the BN43881 clinical trial has been placed on temporary halt until final assessment of the acute liver failure 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Intended indication: Duchenne muscular dystrophy and Becker muscular dystrophyGermany:805/08/2024DE: 05/08/2024Diseases [C] - Musculoskeletal Diseases [C05]Santhera Pharmaceuticals (Schweiz) AGPharmaceutical companyHuman Pharmacology (Phase I)- Other18-64 yearsFemale, Male1EEA18No05/08/202427/05/20252024-08-05T14:44:39.9022025-05-27T03:46:00.3395011198Germany299632024-08-05T14:44:39.9022024-08-05T14:44:39.902829963GermanySTART_OF_TRIAL2024-08-13END_OF_TRIAL2024-10-16START_OF_RECRUITMENT2024-08-13END_OF_RECRUITMENT2024-09-102024-513845-36-008Evaluation of vamorolone CYP3A4 induction on midazolam (a sensitive CYP3A4 substrate) pharmacokineticsSNT-I-VAM-02513/08/202416/10/2024Phase I study in healthy male volunteers. Intended indication: Duchenne muscular dystrophy and Becker muscular dystrophyGermany:805/08/2024DE: 05/08/2024Diseases [C] - Musculoskeletal Diseases [C05]Santhera Pharmaceuticals (Schweiz) AGPharmaceutical companyHuman Pharmacology (Phase I)- Other18-64 yearsFemale, Male118No05/08/202427/05/20252024-513845-36-00Ended2024-08-132024-10-162024-08-05T14:44:39.9022025-05-27T03:46:00.3395011198438330Evaluation of vamorolone CYP3A4 induction on midazolam (a sensitive CYP3A4 substrate) pharmacokineticsSNT-I-VAM-025313616041782Phase I study in healthy male volunteers. Intended indication: Duchenne muscular dystrophy and Becker muscular dystrophyTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False2024-10-072024-08-20116305331Healthy volunteersTrueTrueTrueacceptable2024-07-225Diseases [C] - Musculoskeletal Diseases [C05]41782Phase I study in healthy male volunteers. Intended indication: Duchenne muscular dystrophy and Becker muscular dystrophyTrue46067True131053PublicSanthera General Inquiriesoffice@santhera.com+41619068950451668Pharmaceutical company10Santhera Pharmaceuticals (Schweiz) AGTrueTrueORG-100004668Active131054ScientificCatherine Dutreixcatherine.dutreix@santhera.com+33662193215451668Pharmaceutical company10Santhera Pharmaceuticals (Schweiz) AGTrueTrueORG-100004668Active451668Pharmaceutical company10Santhera Pharmaceuticals (Schweiz) AGTrueTrueORG-100004668Active419790451668Pharmaceutical company10Santhera Pharmaceuticals (Schweiz) AGTrueTrueORG-100004668Active449062Hohenrainstrasse 24Hohenrainstrasse 24Pratteln4133756SwitzerlandTrueORG-100004668TrueCommercial14702685Diseases [C] - Musculoskeletal Diseases [C05]1236522996329963146912013Ended2024-07-302024-07-30TrueGermany2024-08-05Ended400152024-08-132024-10-162024-10-25307022024-08-132024-08-14307032024-08-132024-08-14391492024-08-132024-08-14391502024-08-132024-08-14438892024-08-132024-09-102024-09-11438902024-08-132024-09-102024-09-11True539872024-08-132024-10-162024-10-25FalseFalseFalseTrue2024-08-132024-09-10False3755029963Under evaluation2024-06-07T10:49:48.7254693429963Authorised2024-08-05T14:44:39.4755868629963Ended2024-10-25T09:51:57.3591Germanyacceptable2024-08-02authorized2024-08-052024-08-0518812744451542483838Pharmaceutical company10Nuvisan GmbHTrueTrueORG-100011873Active483822Wegenerstrasse 13, LudwigsfeldWegenerstrasse 13LudwigsfeldNeu-Ulm892312013Germany7319840125michael.lissy@nuvisan.comTrueORG-100011873906844MichaelLissy (Physician)7319840125michael.lissy@nuvisan.com3Clinical ServicesAuthorised24482INITIALAuthorised2024-513845-36-00Ended2024-06-07acceptable2024-07-221236522996329963Germany2013acceptable2024-08-02authorized2024-08-05EndedGermanyEnded2024-08-05Authorised2024-08-0529963GermanyEndedINFalse322822448229963Germany2024-08-05T14:44:39.902authorizedacceptabledecision12365243833INITIALTrueGermany299632024-08-05T14:44:39.9022024-08-05T14:44:39.9028False29963GermanySTART_OF_TRIAL2024-08-13END_OF_TRIAL2024-10-16START_OF_RECRUITMENT2024-08-13END_OF_RECRUITMENT2024-09-10EEA1 2024-512265-13-008EndedA multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous injection in nonambulatory participants with Duchenne Muscular Dystrophy1102-DMD-Pre-CT03Duchenne Muscular DystrophyBulgaria:830/07/2024BG: 30/07/2024Diseases [C] - Musculoskeletal Diseases [C05]Antisense Therapeutics LimitedPharmaceutical companyTherapeutic exploratory (Phase II)Blinded Phase (Efficacy): 1.Percentage of treatment responders at Week 25, where a treatment responder is defined as no decline from baseline in Performance of Upper Limb (PUL 2.0)., Blinded Phase (Efficacy) 2.Change in muscle strength as assessed by percent predicted MyoGrip (using the Myoset System) from baseline to Week 25., Blinded Phase (Efficacy) 3.Change in muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from baseline to Week 25., Blinded Phase (Efficacy) 4.The extent of response in a total arm functional score from baseline to Week 25 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, dominant hand and non-dominant hand MyoGrip and MyoPinch) that do not decline. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5., Blinded Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand., Blinded Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from baseline to Week 25., Blinded Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument, PedsQL™ Duchenne Muscular Dystrophy (DMD) Module Patient Report and Parent Report scores (at baseline [Day 1/Week 0], Weeks 12, 24 and pre-dose Week 25)., OLE Phase (Efficacy): 1.Percentage of treatment responders at Week 49, where a treatment responder is defined as no decline from baseline at entry into this extension study in Performance of Upper Limb (PUL 2.0)., OLE Phase (Efficacy) 2.Change in Muscle strength as assessed by and percent predicted MyoGrip (using the Myoset System) from Week 25 to Week 49., OLE Phase (Efficacy) 3.Change in Muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from Week 25 to Week 49., OLE Phase (Efficacy) 4.The extent of response in a total arm functional score from Week 25 to Week 49 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, right hand and left hand MyoGrip and MyoPinch) that do not decline. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5., OLE Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from Week 25 to Week 49 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand., OLE Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from Week 25 to Week 49., OLE Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument PedsQL™ Duchenne Muscular Dystrophy (DMD) Module Patient Reports and Parent Reports scores (at Weeks 25, 37 and 49 and at Week 65)., Combined Blinded and Open Label Phase: 1.Percentage of treatment responders in the OLE phase compared to the blinded phase in each of the 4 treatment groups; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 2.Change in Muscle strength as assessed by percent predicted MyoGrip (using the Myoset System) from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 3.Change in Muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 4.The extent of response in total arm functional score from OLE phase compared to the blinded phase in each of the 4 treatment groups; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 5.Change of Performance of Upper Limb (PUL 2.0) score for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand. from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 6.Respiratory function assessed by percent predicted PEF and, percent predicted FVC from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 7.Quality of Life assessed by percentage of change in the Paediatric Quality of Life instrument PedsQLTM Duchenne Muscular Dystrophy(DMD) Module Patient Reports and Parent Reports scores from baseline (Day1/Week 0) through to Week 25 and Week 25 through to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102.Placebo for ATL1102 solution for injection, CD49d (alpha chain of VLA-4) Antisense|Oligonucleotide560-17 yearsMale3Both1Blinded Phase: Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25., OLE Phase (Efficacy): Change of Performance of Upper Limb (PUL 2.0) score from Week 25 to Week 49., OLE Phase (Safety): 1.Frequency and severity of AEs, SAEs and SUSARs., OLE Phase (Safety): 2.Clinical Laboratory Tests i.Haematology ii.Coagulation (including fibrinogen) iii.Complement iv.Biochemistry (including haptoglobin) v.Urinalysis (including weekly dipsticks), OLE Phase (Safety): 3.Cardiac function (ECG, echocardiogram), OLE Phase (Safety): 4.Respiratory function (as above in efficacy), OLE Phase (Safety): 5.Physical examination, OLE Phase (Safety): 6.Vital Signs (blood pressure, heart rate, respiratory rate, tympanic temperature), OLE Phase (Safety): 7.Events associated with the Safety Monitoring Plan and Stopping Rules i.number, total frequency and percentage of participants overall who experience events defined in the Safety Monitoring Plan and in whom dosing is halted on a temporary basis or on a permanent basis. ii.Number, total frequency and percentage of participants who experience events defined in each category in the Safety Monitoring Planand in whom dosing is halted on a temporary basis or on a permanent basis., Combined Blinded and Open Label Phase: 1.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25 compared to the change from Week 25 to Week 49., Combined Blinded and Open Label Phase: 2.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 49 (for those that remained on the same ATL1102 treatment dose over the blinded and OLE phases of the study).Yes24/01/202513/08/20252024-07-30T08:45:03.2012025-08-13T03:42:42.8262605758K1_Recruitment Procedure Description Bulgarian Publicdd950808-ff66-4ce2-ad32-631e832ad65c14Recruitment arrangements (for publication)PDF1753441.01L1_SIS and Site ICF Assent Minor assent form Bulgarian Public034203e2-bc3d-498d-bfb7-0a8b9fed888a15Subject information and informed consent form (for publication)PDF1753441.11L1_SIS and ICF Main English Public1e13700d-2ff0-4d4d-8430-bde9708aade815Subject information and informed consent form (for publication)PDF1753443.01L1_SIS and ICF Assent Adolescent English Public29e3bd77-4ce3-4f27-9944-9e7537e3535915Subject information and informed consent form (for publication)PDF1753442.01L1_SIS and ICF Caregiver Bulgarian Public10faa1db-2390-42c0-9970-5eb0bb3edecb15Subject information and informed consent form (for publication)PDF1753443.01L1_SIS and Site ICF Assent Adolescent Bulgarian Publica95fa174-e6f2-4c5c-8b61-4cf1a021805015Subject information and informed consent form (for publication)PDF1753442.01L1_SIS and Site ICF caregiver Public935f34a7-9e97-4b3f-a7cb-bcbe4b6c525315Subject information and informed consent form (for publication)PDF1753443.01L1_SIS and Site ICF Main Bulgarian Public46b2dbdf-6ae4-43a3-be83-3554bdff9edf15Subject information and informed consent form (for publication)PDF1753443.01L1_SIS and Site ICF Other Pregnant Partner Bilingual Public2d4b762a-a243-49be-a889-277e17ef357015Subject information and informed consent form (for publication)PDF1753441.01L1_SIS and ICF Assent Adolescent Bulgarian Public50fdfbfc-3197-46be-8949-3b249158f3b515Subject information and informed consent form (for publication)PDF1753442.01L1_SIS and ICF Assent Minor country Bulgarian Public6bb46be3-a806-48b0-9767-4cf8a001ae3915Subject information and informed consent form (for publication)PDF1753441.01L1_SIS and ICF Assent Minor country English Public66cac5c4-29bb-4f71-bc9c-8dacd61fa57915Subject information and informed consent form (for publication)PDF1753441.01L1_SIS and ICF Caregiver English Public85d221a1-1775-4e0a-af80-7d17b486b9e915Subject information and informed consent form (for publication)PDF1753443.01L1_SIS and ICF Other Pregnant Partner English Public694eb35a-2edc-42d5-b065-719d66b5099215Subject information and informed consent form (for publication)PDF1753441.01L1_SIS and ICF Main Bulgarian Public3b8a7444-c67d-47cb-bbdc-31b92a2583f215Subject information and informed consent form (for publication)PDF1753443.01L1_SIS and ICF Other Pregnant Partner Bulgarian Public5f447e6b-f27c-4155-99ff-8ae6033cdcc915Subject information and informed consent form (for publication)PDF1753441.01T1_Summary of Final Results Initial English 1102-DMD-Pre-CT03 Public1fb33373-1c3c-407c-9c6e-dc019afa90c4103Summary of results (for publication)PDF20191.01T1_Lay Summaries of Results Initial Bulgarian 1102-DMD-Pre-CT0301687830-a4d1-4639-9039-bed70ace2f4d46Laypersons summary of results (for publication)PDF20201.01T1_Lay Summaries of Results Initial English 1102-DMD-Pre-CT03f04ca2b9-cee9-4b8c-86f3-6884dabb94e746Laypersons summary of results (for publication)PDF20201.01Bulgaria265622024-07-30T08:45:03.2012025-01-24T15:57:08.628826562BulgariaSTART_OF_TRIAL2023-04-13START_OF_RECRUITMENT2023-11-09EARLY_TERMINATION2025-01-141Sponsor DecisionTrue2025-01-1520192024-512265-13-001102-DMD-Pre-CT03 Summary of Final Results InitialSubmittedSummary of ResultsFinalTrue941782025-08-12T17:15:40.5931732025-08-12T16:51:04.862025-08-12T17:15:5713054SUM-9417820202024-512265-13-001102-DMD-Pre-CT03 Lay Summaries of Results InitialSubmittedLaypersons Summary of ResultsFinalTrue941812025-08-12T17:15:48.3107612025-08-12T16:55:39.5942025-08-12T17:16:0513054SUM-941792024-512265-13-008A multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous injection in nonambulatory participants with Duchenne Muscular Dystrophy1102-DMD-Pre-CT0313/04/202314/01/2025Duchenne Muscular DystrophyBulgaria:830/07/2024BG: 30/07/2024Diseases [C] - Musculoskeletal Diseases [C05]Antisense Therapeutics LimitedPharmaceutical companyTherapeutic exploratory (Phase II)Blinded Phase (Efficacy): 1.Percentage of treatment responders at Week 25, where a treatment responder is defined as no decline from baseline in Performance of Upper Limb (PUL 2.0)., Blinded Phase (Efficacy) 2.Change in muscle strength as assessed by percent predicted MyoGrip (using the Myoset System) from baseline to Week 25., Blinded Phase (Efficacy) 3.Change in muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from baseline to Week 25., Blinded Phase (Efficacy) 4.The extent of response in a total arm functional score from baseline to Week 25 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, dominant hand and non-dominant hand MyoGrip and MyoPinch) that do not decline. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5., Blinded Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand., Blinded Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from baseline to Week 25., Blinded Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument, PedsQL™ Duchenne Muscular Dystrophy (DMD) Module Patient Report and Parent Report scores (at baseline [Day 1/Week 0], Weeks 12, 24 and pre-dose Week 25)., OLE Phase (Efficacy): 1.Percentage of treatment responders at Week 49, where a treatment responder is defined as no decline from baseline at entry into this extension study in Performance of Upper Limb (PUL 2.0)., OLE Phase (Efficacy) 2.Change in Muscle strength as assessed by and percent predicted MyoGrip (using the Myoset System) from Week 25 to Week 49., OLE Phase (Efficacy) 3.Change in Muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from Week 25 to Week 49., OLE Phase (Efficacy) 4.The extent of response in a total arm functional score from Week 25 to Week 49 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, right hand and left hand MyoGrip and MyoPinch) that do not decline. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5., OLE Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from Week 25 to Week 49 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand., OLE Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from Week 25 to Week 49., OLE Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument PedsQL™ Duchenne Muscular Dystrophy (DMD) Module Patient Reports and Parent Reports scores (at Weeks 25, 37 and 49 and at Week 65)., Combined Blinded and Open Label Phase: 1.Percentage of treatment responders in the OLE phase compared to the blinded phase in each of the 4 treatment groups; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 2.Change in Muscle strength as assessed by percent predicted MyoGrip (using the Myoset System) from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 3.Change in Muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 4.The extent of response in total arm functional score from OLE phase compared to the blinded phase in each of the 4 treatment groups; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 5.Change of Performance of Upper Limb (PUL 2.0) score for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand. from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 6.Respiratory function assessed by percent predicted PEF and, percent predicted FVC from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102., Combined Blinded and Open Label Phase: 7.Quality of Life assessed by percentage of change in the Paediatric Quality of Life instrument PedsQLTM Duchenne Muscular Dystrophy(DMD) Module Patient Reports and Parent Reports scores from baseline (Day1/Week 0) through to Week 25 and Week 25 through to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102.Placebo for ATL1102 solution for injection, CD49d (alpha chain of VLA-4) Antisense|Oligonucleotide560-17 yearsMale3115/01/2025Blinded Phase: Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25., OLE Phase (Efficacy): Change of Performance of Upper Limb (PUL 2.0) score from Week 25 to Week 49., OLE Phase (Safety): 1.Frequency and severity of AEs, SAEs and SUSARs., OLE Phase (Safety): 2.Clinical Laboratory Tests i.Haematology ii.Coagulation (including fibrinogen) iii.Complement iv.Biochemistry (including haptoglobin) v.Urinalysis (including weekly dipsticks), OLE Phase (Safety): 3.Cardiac function (ECG, echocardiogram), OLE Phase (Safety): 4.Respiratory function (as above in efficacy), OLE Phase (Safety): 5.Physical examination, OLE Phase (Safety): 6.Vital Signs (blood pressure, heart rate, respiratory rate, tympanic temperature), OLE Phase (Safety): 7.Events associated with the Safety Monitoring Plan and Stopping Rules i.number, total frequency and percentage of participants overall who experience events defined in the Safety Monitoring Plan and in whom dosing is halted on a temporary basis or on a permanent basis. ii.Number, total frequency and percentage of participants who experience events defined in each category in the Safety Monitoring Planand in whom dosing is halted on a temporary basis or on a permanent basis., Combined Blinded and Open Label Phase: 1.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25 compared to the change from Week 25 to Week 49., Combined Blinded and Open Label Phase: 2.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 49 (for those that remained on the same ATL1102 treatment dose over the blinded and OLE phases of the study).Yes24/01/202513/08/20252024-512265-13-00Ended2023-04-132025-01-142024-07-30T08:45:03.2012025-08-13T03:42:42.82626057587159047100000000329Australia36AUAUSTrue100000000549Turkey792TRTURTrue100000000556United Kingdom826GBGBRTrue100000000518Serbia688RSSRBTrue2924461N/AN/AN/APlacebo for ATL1102 solution for injectionN/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AMIA(IMP) 20377FalsePlacebo for ATL1102 solution for injectionn/aN/A292445110395073SOLUTION FOR INJECTIONPRD103950731CD49d (alpha chain of VLA-4) Antisense|OligonucleotideSOLUTION FOR INJECTIONATL11022'-O-(2-METHOXYETHYL) PHOSPHOROTHIOATE ANTISENSE OLIGONUCLEOTIDE TARGETING CD49D RNASUB2167492'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNAANTISENSE THERAPEUTICS LIMITED10395073288699ANTISENSE THERAPEUTICS LIMITEDNucleic AcidNucleic Acid2'-O-(2-METHOXYETHYL) PHOSPHOROTHIOATE ANTISENSE OLIGONUCLEOTIDE TARGETING CD49D RNASUB216749False1TrueEU/3/20/2375mg milligram(s)50mg milligram(s)2500492ChemicalPRD10395073ATL1102MIA(IMP) 2037711SUBCUTANEOUS USEFalseCD49d (alpha chain of VLA-4) Antisense|Oligonucleotide2'-o-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting cd49d rnaSOLUTION FOR INJECTIONA multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous injection in nonambulatory participants with Duchenne Muscular Dystrophy5179049acd98563-e85f-4763-81f1-9f45f843b898Многоцентрово, рандомизирано, двойно-сляпо, плацебо-контролирано и отворено разширено проучване за оценка на ефикасността, безопасността и фармакокинетичния профил на две дозови нива на ATL1102, прилагани чрез подкожна инжекция при неамбулаторни участници с мускулна дистрофия на Дюшен.1BulgarianA multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous injection in nonambulatory participants with Duchenne Muscular Dystrophy51790637922ab3b-e5a5-4222-af68-38662bd0acd6Многоцентрово, рандомизирано, двойно-сляпо, плацебо-контролирано и отворено разширено проучване за оценка на ефикасността, безопасността и фармакокинетичния профил на две дозови нива на ATL1102, прилагани чрез подкожна инжекция при неамбулаторни участници с мускулна дистрофия на Дюшен1Bulgarian1102-DMD-Pre-CT03336892NCT05938023336893NCT05938023ANZCTR6False42This is a Phase II trial6317172417Duchenne Muscular Dystrophy517899577143e3c-ba54-41b3-928c-c0eabb3b75dfМускулна дистрофия на Дюшен1BulgarianTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False620845213Immunogenicity20844832084505208446720844742084499208451Blinded phase: To evaluate the effect of ATL1102 on upper limb muscle function in non-ambulant participants with DMD, as assessed by change in the Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) score compared to placebo. OLE phase: To evaluate the continued safety and tolerability of ATL1102 administered once weekly by subcutaneous injection, in non-ambulant participants with DMD. For Primary Combined Blinded and OLE Phases objectives please see section 4.3 of Protocol51790626b38a91b-52b7-4e98-b741-784119ea0194Заслепена фаза: Да се оцени ефекта на ATL1102 върху мускулната функция на горните крайници при неамбулаторни участници с МДД според оценката на промяната в резултата от Модула за функция на горните крайници за МДД 2.0 (PUL 2.0) в сравнение с плацебо. Открита фаза на продължение (OLE): Да се оцени продължаващата безопасност и поносимост на ATL1102, прилаган веднъж седмично чрез подкожна инжекция, при неамбулантни участници с МДД. За целите на първичните комбинирани заслепени и OLE фази, моля, вижте раздел 4.3 от протокола1Bulgarian2354371Blinded phase: 1.To evaluate the effects of ATL1102 using several additional endpoints for assessment of muscle function, strength, respiratory function and Quality of Life51790646970d372-8309-4b76-8146-90b98b398f96Вторични цели: Заслепена фаза: 1 1. Да се оценят ефектите на ATL1102 чрез няколко допълнителни крайни точки за оценка на мускулната функция и издръжливост, респираторната функция и качеството на живот1Bulgarian2354382Blinded phase: To evaluate the safety and tolerability of ATL1102 administered once weekly, by subcutaneous injection in non-ambulant participants with DMD including events associated with the Safety Monitoring Plan and Stopping Rules.517906572b88c85-60be-4492-9438-59c91ae9e435Да се оцени безопасността и поносимостта на ATL1102, прилаган веднъж седмично, чрез подкожна (SC) инжекция при неамбулаторни участници с МДД, включително събития, свързани с Плана за наблюдение на безопасността и Правилата за спиране1Bulgarian2354393Blinded phase: To evaluate the pharmacokinetic (PK) profile of ATL1102 at the different dose levels, administered once weekly by subcutaneous injection in non-ambulant participants with DMD.5179066f3357490-a8a3-45b8-b467-f21506bb5c0bДа се оцени фармакокинетичният (ФК) профил на ATL1102 при различните дозови нива, прилаган веднъж седмично чрез подкожна (SC) инжекция при неамбулаторни участници с МДД1Bulgarian2354404OLE Phase:1.To evaluate the of effect of ATL1102 on upper limb muscle function in non-ambulant participants with DMD as assessed by change in the Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) score.517906708edecf1-ea66-4ab1-9bf8-c84938cee921Открита фаза на продължение (OLE): 1. Да се оцени ефектът на ATL1102 върху мускулната функция на горните крайници при неамбулаторни участници с МДД според оценката на промяната в скора от Модула за функция на горните крайници за МДД 2.0 (PUL 2.0).1Bulgarian2354415OLE Phase: To evaluate the effects of ATL1102 using several additional endpoints for assessment of muscle function, strength, respiratory function and Quality of Life5179068bab3b18e-06ec-45ed-9b90-2754aa487a0cДа се оценят ефектите на ATL1102 чрез няколко допълнителни крайни точки за оценка на мускулната функция и издръжливост, респираторната функция и качеството на живот1Bulgarian4573721Has a parent/guardian who is capable of understanding the purposes and risks of the study and is able to provide voluntary written informed consent for the participant to participate in the study and assent will be documented.5179050acee0e20-f241-488d-b90e-a9d62c049d07Един участник ще бъде включен в това проучване само ако отговаря на всички от следващите критерии: 1. Има родител/настойник, който е способен да разбере целите и рисковете на проучването и може да предостави доброволно писмено информирано съгласие за участието на участника в проучването, което ще бъде документирано.1Bulgarian45737310Has adequate cardiac function defined as left ventricular ejection fraction (LVEF) ≥45% by echocardiogram and if receiving cardiac medication, must be currently on a stable regimen and doses of cardiac therapy (at least 3 months prior to baseline Day 1) including angiotensin converting enzyme inhibitors (ACEi), angiotensin 2 receptor antagonists (A2RA), aldosterone receptor antagonists (ARA) or beta blockers (BB).517905191175d45-3202-4850-b134-3c0abbb65c50Има адекватна сърдечна функция, дефинирана като левокамерна фракция на изтласкване (left ventricular ejection fraction, LVEF) ≥ 45% от ехокардиограма и ако приема лекарство за сърцето, трябва в момента да е на стабилен режим и дози на сърдечна терапия (най-малко 3 месеца преди Ден 1 от базовото ниво), включително инхибитори на ангиотензин-конвертиращия ензим (ACEi), антагонисти на ангиотензин 2 рецептор (A2RA), антагонисти на алдостерон рецептор (ARA) или бета-блокери (BB).1Bulgarian45737411Participant who is post pubertal and sexually active must agree to use approved methods of contraception (condoms or abstinence) for the duration of the study and until 4 months after administration of the last dose of the study medication. Female sexual partner must also agree to use a medically acceptable form of contraception.517905207e3c5e7-719e-4a7a-9389-7e2d7863bdd3Участникът, който е преминал пубертета и е сексуално активен, трябва да се съгласи да използва одобрени методи за контрацепция (презервативи или въздържание) за периода на проучването и до 4 месеца след прилагането на последната доза от изпитваното лекарство. Сексуалната партньорка от женски пол също трябва да се съгласи да използва медицински приемлива форма на контрацепция. Вижте Изисквания за контрол на раждаемостта.1Bulgarian45737512Participant and their parent/guardian are willing and able to comply with scheduled visits, study medication administration and study procedures.5179053beb35728-108d-4f1d-8c47-067a3028a3efУчастникът и неговият родител/настойник/болногледач желаят и са в състояние да спазват планираните визити, приложението на изпитваното лекарство и процедурите по проучването.1Bulgarian4573762Where required by law or if the Investigator determines the potential participant is of sufficient maturity and has the ability to understand the nature and consequence of the study, participant consent will be obtained. Otherwise, assent will be documented.5179054f295ce4e-24e9-45d3-ac7f-3a53482e33fcКогато това се изисква от закона или ако Изследователят определи, че потенциалният участник е достатъчно зрял и способен да разбере естеството и последствията от проучването, ще бъде получено съгласие от участника. В противен случай съгласието ще бъде документирано.1Bulgarian4573773Has a clinical diagnosis of DMD confirmed by validated genetic testing (i.e., documented deletion, duplication or point mutation in the dystrophin gene).51790550cbe1af7-1bff-46ea-954d-e6b7f9ffeb48Има клинична диагноза за МДД, потвърдена чрез валидирано генетично изследване (т.е. документирана делеция, дублиране или точкова мутация в гена на дистрофина).1Bulgarian4573784Is considered to be non-ambulatory, defined as unable to walk 10 meters without assistance or help at Screening51790566ac5ca89-0179-4af2-b0e4-b7c8b085a84dСчита се за неамбулаторен, определен като неспособен да ходи 10 метра без съдействие или помощ при Скрининг.1Bulgarian4573795Male aged 10 to less than 18 years, at the time of screening and informed consent for participation in the study.51790570fba6efa-33b0-406e-88dd-7f0e4e1901daУчастник от мъжки пол на възраст от 10 до под 18 години към момента на Скрининг и информирано съгласие за участие в проучването.1Bulgarian4573806Body weight of at least 25 kg at Screening.517905877f934a1-83a1-4253-a874-0734e05cc790Телесно тегло най-малко 25 кг при Скрининг.1Bulgarian4573817If receiving corticosteroid therapy, therapy was initiated at least six months prior to the baseline visit and a stable daily dose for at least 3 months prior to baseline and, there is currently no clinical intent to alter the dose during the study period, except for adaption for body weight.5179059915961bd-3a78-4191-bce7-85126db781fdАко лечението с кортикостероиди е започнато най-малко шест месеца преди базовата визита и стабилна дневна доза за поне 3 месеца преди изходното ниво и и към момента няма клинично намерение за промяна на дозата по време на периода на проучването, с изключение на адаптиране според телесното тегло.1Bulgarian4573828On study entry (Screening and Baseline), the participant has a Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A score ≥2.51790608a2aa81e-50c3-4ed1-bc6d-2b1a3221a359При включване в проучването (Скрининг и Базово ниво), участникът има скор ≥ 2 според Модула за функция на горните крайници за МДД 2.0 (PUL 2.0).1Bulgarian4573839Able to perform spirometry and has sufficient Respiratory function defined as reproducible percent predicted FVC ≥50%. Ability to provide reliable and reproducible repeat FVC with the best of three attempts assessed at Baseline being within 20% of the best of three attempts assessed at Screening.51790615945a556-0e39-4568-93cd-3f849d4adebdСпособен да извърши спирометрия и има достатъчна Респираторна функция, дефинирана като възпроизводим процент очакван ФВК ≥ 50%. Способност за осигуряване на надежден и възпроизводим повторен ФВК, като най-добрият от трите опита, оценени на Базово ниво, е в рамките на 20% от най-добрия от трите опита, оценени при Скрининг.1Bulgarian7873461A participant who meets any of the following criteria will be excluded from participating in the study: 1.Participation in another clinical trial (non-interventional) or administration of any investigational product or experimental product within 12 weeks or 5 half-lives (whichever is longer) preceding Day 1.517902820656c7a-87ac-4559-b62c-826e624fe221Критерии за изключване Всеки участник, който отговаря на някой от следните критерии, ще бъде изключен от участие в проучването: 1. Участие в друго клинично изпитване (неинтервенционно) или приложение на изпитвано лекарство или експериментално лекарство в рамките на 12 седмици или 5 полуживота (което е по-дълго) преди Ден 1.1Bulgarian78734710Known history of or a positive test for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibodies, human immunodeficiency virus (HIV) antibodies at Screening.517902939286e08-6085-40b1-b801-08fc001a724eИзвестна анамнеза за или положителни резултати от изследване за повърхностен антиген (HBsAg) на хепатит B, антитела (HCVAb) на вируса на хепатит C или вируса на човешкия имунодефицит (HIV) при Скрининг.1Bulgarian78734811Evidence of renal impairment and/or cystatin C >1.4 mg/L.5179030979d4454-8150-4615-a1b3-8bc630ba1bb8Доказателство за бъбречно увреждане и/или цистатин C > 1,4 mg/l.1Bulgarian78734912Received a live vaccine (including intranasal influenza vaccine) within 4 weeks prior to Day 1 or planned live vaccination during the study period.51790310f127444-8fe9-468f-a307-c498e5c1f5d0Получена жива ваксина (включително интраназална противогрипна ваксина) в рамките на 4 седмици преди Ден 1 или планирана жива ваксина по време на периода на проучването.1Bulgarian78735013Planned or expected surgery during the study period (as judged by the Investigator).51790320dc1eb27-a6e3-46e9-b9dd-3d9e56b10767Планирана или очаквана операция по време на периода на проучването (по преценка на Изследователя).1Bulgarian78735114Asthma (if requiring regular medication), bronchitis/chronic obstructive pulmonary disease (COPD), bronchiectasis, emphysema, pneumonia or the presence of any non-DMD respiratory illness that affects PEF and FVC or other respiratory measures.5179033260a2b00-12fb-47fa-b55a-7ca5de611c5bАстма (ако се налага редовно лечение), бронхит/хронична обструктивна белодробна болест (ХОББ), бронхиектазии, емфизем, пневмония или наличие на каквото и да било респираторно заболяване, различно от МДД, което засяга ВЕД и ФВК или други респираторни измервания.1Bulgarian78735215Requires day-time assisted mechanical or non-invasive ventilation (NIV) (night-time NIV is permitted).5179034321427fd-7274-4e0c-9e6c-d8b1658a0e77Изисква механична или неинвазивна вентилация (non-invasive ventilation, NIV) през деня (NIV през нощта е разрешена).1Bulgarian78735316Unable to form a mouth seal to allow precise respiratory flow measurements and mouth pressure.51790350306bd95-2cc9-4b8c-8a89-f6490fc8670bНе може да затвори плътно с устата, което да позволи прецизно измерване на дихателния поток и налягане в устата.1Bulgarian78735417Chronic use (daily intake >14 days), within one month of Day 1, of beta-2 agonists or any use of other bronchodilating medication (e.g., inhaled steroids, sympathomimetics, anticholinergics).51790363d64dedc-f59d-4b2a-9f11-f68ad2635b33Хронично използване (ежедневен прием > 14 дни) в рамките на един месец от Ден 1, на бета-2 агонисти или използване на друго лекарство с бронходилатиращо действие (напр. инхалаторни стероиди, симпатомиметици, антихолинергици).1Bulgarian78735518Used carnitine, creatine, glutamine, oxatomide, idebenone or other forms of coenzyme Q10 or vitamin E or any other nutritional or antioxidant supplements or herbal medicines or anabolic steroids other than standard corticosteroids or puberty testosterone supplementation within 4 weeks of Day 1. NOTE: daily multivitamin, Vitamin D or calcium supplements are permitted. Used ataluren, eteplirsen, casimersen, golodirsen (or other exon skipping antisense oligonucleotide drugs), and vamorolone within 6 months prior to Day 1. Used systemic immunosuppressants treatment (e.g., mitoxantrone, azathioprine, methotrexate, cyclophosphamide, mycophenolate, TNFɑ inhibitors) within the 3 months prior to Day 1. Used intravenous immunoglobulin (IVIg) within 6 months prior to Day 1. Refer Section 7.6.2 Prohibited Medication.51790377a10b595-d07e-49c1-b708-01dc3aef5ffaИзползване на карнитин, креатин, глутамин, оксатомид, идебенон или други форми на коензим Q10 или витамин E, всякакви други хранителни или антиоксидантни добавки, билкови лекарства, анаболни стероиди, различни от стандартни кортикостероиди, или добавка на тестостерон в пубертета в рамките на 4 седмици от Ден 1. ЗАБЕЛЕЖКА: позволен е ежедневен прием на мултивитамини, витамин D или калциеви добавки. Използвани аталурен, етеплирсен, казимерсен, голодирсен (или друг екзон пропускане на антисенс олигонуклеотидни лекарства) и ваморолон в рамките на 6 месеца преди Ден 1. Използвано системно лечение с имуносупресори (напр. митоксантрон, азатиоприн, метотрексат, циклофосфамид, микофенолат, TNFɑ инхибитори) в рамките на 3 месеца преди Ден 1. Използван интравенозен имуноглобулин (IVIg) в рамките на 6 месеца преди Ден 1. Вижте раздел 7.6.2 Забранени медикаменти.1Bulgarian78735619Other than the condition under study, has a history of or current active medical condition including allergic, skin, cardiovascular, psychiatric disease, drug or alcohol abuse, severe behavioural or cognitive deficits, or laboratory finding, that in the opinion of the Investigator precludes participation in the study, or may interfere with the study objectives/results.517903867bd9d70-d128-40a5-a1cc-9324e8a18dbdОсвен състоянието, което е предмет на проучването, има анамнеза за или текущо активно клинично състояние, включително алергично, кожно, сърдечно-съдово, психиатрично заболяване, злоупотреба с наркотици или алкохол, тежки поведенчески или когнитивни дефицити или лабораторна находка, които според мнението на Изследователя не позволяват участие в проучването или биха могли да повлияят върху целите/резултатите от проучването.1Bulgarian7873572Exposure to more than 3 investigational products within the 12 months prior to Day 1.51790397b68469f-6a62-453b-8889-7007b8cdb3d4Експозиция на повече от 3 изпитвани лекарства в рамките на 12 месеца преди Ден 1.1Bulgarian78735820Has an increased risk for opportunistic infections or systemic medical conditions resulting in significantly compromised immune system function (e.g., HIV, organ transplant, active malignancy).5179040f1f25f7d-8e5d-428d-821f-64919f0a08a0Има повишен риск от опортюнистични инфекции или системни медицински състояния, водещи до значително нарушена функция на имунната система (напр. HIV, органна трансплантация, активно злокачествено заболяване).1Bulgarian78735921An employee of the Sponsor or research site personnel directly affiliated with this study or their immediate family members defined as a spouse, parent, sibling, or child, whether biological or legally adopted.5179041a46bfd1c-5557-46f6-9fbc-8f3aef8ef7edСлужител на Спонсора или персонала на изследователския център, свързан директно с това проучване, или негови/нейни близки членове на семейството, като съпруг/а, родител, брат, сестра или дете, независимо дали биологично или законно осиновено.1Bulgarian7873603Has the following abnormal haematology values during the Screening period: a.Lymphocytes <1.2 x 109/L b.Neutrophils <1.8 x 109/L c.Platelets <150 x 109/L.51790420f039b21-4b55-4aa3-8c5e-3662d65129ccИма следните абнормни хематологични стойности по време на периода на Скрининг: a. Лимфоцити < 1,2 x 109/L b. Неутрофили < 1,8 x 109/L c. Тромбоцити < 150 x 109/L.1Bulgarian7873614Has the following liver function test values during the Screening period: a.Gamma glutamyl transpeptidase (GGT) levels >2.0 x the upper limit of normal (ULN) or b.Total bilirubin concentrations greater than 1.5 x ULN or c. Glutamate dehydrogenase (GLDH) levels greater than 2.0 x ULN.5179043ca888afa-72ef-454c-8fec-d06ae59c19a1Има следните стойности от чернодробен функционален тест по време на периода на Скрининг: a. Нива на гама-глутамил транспептидаза (GGT) > 2,0 x горната граница на нормата (ГГН) или b. Концентрации на общ билирубин над 1,5 x ГГН или c. Нива на глутамат дехидрогеназа (Glutamate dehydrogenase, GLDH), по-високи от 2,0 x ГГН.1Bulgarian7873625History of clinically significant bleeding or coagulation abnormalities.51790445deb7aa9-2b44-4d3e-8844-f6c4c0a9373bАнамнеза за клинично значимо кървене или коагулационни аномалии.1Bulgarian7873636Clinically significant abnormal coagulation parameters.5179045f53ae942-53bb-4f8e-a3fd-5deb9172c256Клинично значими абнормни параметри на коагулация.1Bulgarian7873647Currently receiving antiplatelet or anticoagulant therapy or has taken medication with an antiplatelet or anticoagulant effect within 4 weeks prior Day 1 (e.g., aspirin).5179046ed4abd53-0d38-46c5-9dde-d0d4c202e74fТекущ прием на антитромбоцитна или антикоагулантна терапия или прием на лекарство с антитромбоцитен или антикоагулантен ефект в рамките на 4 седмици преди Ден 1 (напр. аспирин).1Bulgarian7873658History of hypersensitivity to ATL1102 or to any of the excipients in the ATL1102 drug product formulation.5179047ca3ab639-e3ad-41fc-a387-98e0d88120e4Анамнеза за свръхчувствителност към ATL1102 или към някое от помощните вещества в лекарствения продукт ATL1102.1Bulgarian7873669Any evidence of clinically significant structural or functional heart abnormality (cardiomyopathy that is managed by ACEi or beta blockers is acceptable provided the LVEF inclusion criterion is met).517904878df00db-bf97-4b05-96a3-25e8859f5de6Всяко доказателство за клинично значима структурна или функционална сърдечна аномалия (кардиомиопатия, която се контролира чрез АСЕi или бета-блокери, е приемлива, при условие че е изпълнен критерият за включване за LVEF).1Bulgarian5318151Blinded Phase: Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25.True5178996e7f29f65-f7e7-4675-96cf-af0c06994685Заслепена фаза: Промяна в скора за Функция на горните крайници (PUL 2.0) от базовото ниво до Седмица 25.1Bulgarian5318162OLE Phase (Efficacy): Change of Performance of Upper Limb (PUL 2.0) score from Week 25 to Week 49.True51789978bc841d0-23ba-449c-9db4-05153898d30aOLE фаза: Промяна в скора за Функция на горните крайници (PUL 2.0) от Седмица 25 до Седмица 49.1Bulgarian5318173OLE Phase (Safety): 1.Frequency and severity of AEs, SAEs and SUSARs.True517899820c0d12d-7364-4333-9af4-adf69c5a3ab5OLE фаза: (Параметри за безопасност): 1. Честота и тежест на нежелани събития (НС), Сериозни нежелани събития (СНС) и Подозирани неочаквани сериозни нежелани събития (ПНСНР).1Bulgarian5318184OLE Phase (Safety): 2.Clinical Laboratory Tests i.Haematology ii.Coagulation (including fibrinogen) iii.Complement iv.Biochemistry (including haptoglobin) v.Urinalysis (including weekly dipsticks)True51789996c116385-47de-4626-a117-158c9cc054342. Клинични лабораторни изследвания i. Хематология ii. Коагулация (включително фибриноген) iii. Комплемент iv. Биохимия (включително хаптоглобин) v. Анализ на урина (включително ежеседмични тест-ленти).1Bulgarian5318195OLE Phase (Safety): 3.Cardiac function (ECG, echocardiogram)True517900071ea482d-db22-4d9a-91f3-92e1495cfeb23. Сърдечна функция (ЕКГ, ехокардиограма).1Bulgarian5318206OLE Phase (Safety): 4.Respiratory function (as above in efficacy)True5179001e220d80e-e98a-4831-b1af-61791a1836bc4. Респираторна функция (както е посочено по-горе по отношение на ефикасността).1Bulgarian5318217OLE Phase (Safety): 5.Physical examinationTrue51790021ce6b7e3-0e8a-4a40-a813-cb2fafc88e7f5. Физикален преглед.1Bulgarian5318228OLE Phase (Safety): 6.Vital Signs (blood pressure, heart rate, respiratory rate, tympanic temperature)True517900390144129-d2a2-4182-b3d8-30d4bb8e1f546. Жизнени показатели (кръвно налягане, пулс, респираторна честота, тимпанична температура).1Bulgarian5318239OLE Phase (Safety): 7.Events associated with the Safety Monitoring Plan and Stopping Rules i.number, total frequency and percentage of participants overall who experience events defined in the Safety Monitoring Plan and in whom dosing is halted on a temporary basis or on a permanent basis. ii.Number, total frequency and percentage of participants who experience events defined in each category in the Safety Monitoring Planand in whom dosing is halted on a temporary basis or on a permanent basis.True5179004f3190a50-8452-4d22-a126-f640751155f47. Събития, свързани с Плана за наблюдение на безопасността и Правилата за спиране i. Брой, обща честота и процент на участниците като цяло, които изпитват събития, определени в Плана за наблюдение на безопасността (Таблица 3) и на които дозирането се спира временно или за постоянно. ii. рБрой, обща честота и процент на участниците, които изпитват събития, определени във всяка категория в Плана за наблюдение на безопасността и на които дозирането се спира временно или за постоянно.1Bulgarian53182410Combined Blinded and Open Label Phase: 1.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25 compared to the change from Week 25 to Week 49.True51790050a464fbe-66fb-41ed-bc4e-651776786c0bКомбинирани заслепени и OLE фази: 1. Промяна на производителността на Резултат за горен крайник (PUL 2.0) от изходното ниво до седмица 25 в сравнение с промяна от седмица 25 на седмица 49.1Bulgarian53182511Combined Blinded and Open Label Phase: 2.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 49 (for those that remained on the same ATL1102 treatment dose over the blinded and OLE phases of the study).True5179006b6de508d-84c9-45ef-81d7-cd4537e6243c2. Промяна на показателя за ефективност на горния крайник (PUL 2.0) от изходното ниво до Седмица 49 (за тези, които са останали на същата доза за лечение с ATL1102 през заслепените и OLE фазите на изследването).1Bulgarian5318261Blinded Phase (Efficacy): 1.Percentage of treatment responders at Week 25, where a treatment responder is defined as no decline from baseline in Performance of Upper Limb (PUL 2.0).False51790070e7325da-8bb9-4554-81f5-3eda57ffac81Заслепена фаза (ефикасност): 1. Процент на отговорили на лечението в Седмица 25, като отговор на лечението се дефинира като отсъствие на влошаване от базовото ниво в скора за Функция на горните крайници (PUL 2.0).1Bulgarian5318272Blinded Phase (Efficacy) 2.Change in muscle strength as assessed by percent predicted MyoGrip (using the Myoset System) from baseline to Week 25.False5179008b10b7e76-1ea0-4f0d-98af-4fb2399412de2. Промяна в мускулната издръжливост, оценена чрез процент прогнозиран MyoGrip (с помощта на системата Myoset) от базовото ниво до Седмица 25.1Bulgarian5318283Blinded Phase (Efficacy) 3.Change in muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from baseline to Week 25.False517900960b1f173-8247-46bc-acec-c0dcbb88a2ad3. Промяна в мускулната издръжливост, оценена чрез процент прогнозиран MyoPinch (с помощта на системата Myoset) от базовото ниво до седмица 25.1Bulgarian5318294Blinded Phase (Efficacy) 4.The extent of response in a total arm functional score from baseline to Week 25 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, dominant hand and non-dominant hand MyoGrip and MyoPinch) that do not decline. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5.False5179010209b2940-e880-4e32-b85d-42441796cf8f4. Степента на отговор в общия функционален резултат на терапевтичната група от базовото ниво до Седмица 25 ще бъде оценена чрез кумулативното разпределение на броя на компонентите на общия функционален резултат на терапевтичната група (PUL2.0, доминираща ръка и недоминираща ръка MyoGrip и MyoPinch), които не намаляват. Променливата на крайната точка ще бъде броят на компонентите със стабилизиране, което води до бройка в диапазона от 0 до 5.1Bulgarian5318305Blinded Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand.False5179011d362f874-f2c6-4a7e-b870-77215157ac285. Промяна в резултата на ефективността на горния крайник (PUL 2.0) от базовото ниво до Седмица 25 за всяко от измеренията на PUL2.0; високо ниво на рамото, средно ниво на лакътя и дистално ниво на китката и ръката.1Bulgarian5318316Blinded Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from baseline to Week 25.False51790120b84abe0-5b5a-4e28-bdca-5659638060cc6. Респираторна функция, оценена чрез процент прогнозиран PEF и процент прогнозиран ФВК от базово ниво до Седмица 25.1Bulgarian5318327Blinded Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument, PedsQL™ Duchenne Muscular Dystrophy (DMD) Module Patient Report and Parent Report scores (at baseline [Day 1/Week 0], Weeks 12, 24 and pre-dose Week 25).False5179013ee47f70a-d8f7-46fa-9307-fc01b89d03967. Качество на живот, оценено чрез процент на промяната в Инструмента за качество на живот при педиатрични пациенти (PedsQL™) за мускулна дистрофия тип Дюшен (МДД) и резултатите от Доклад на пациента и Доклад на родителя (на базово ниво [Ден 1/Седмица 0], Седмици 12, 24 и 25).1Bulgarian5318338OLE Phase (Efficacy): 1.Percentage of treatment responders at Week 49, where a treatment responder is defined as no decline from baseline at entry into this extension study in Performance of Upper Limb (PUL 2.0).False517901436c37d5a-a7e9-4a29-a3c0-4f2026dfbce0OLE фаза (ефикасност): 1. Процент на отговорили на лечението в Седмица 49, като отговор на лечението се дефинира като отсъствие на влошаване от базовото ниво при влизането в това продължение на проучването в оценката за Функция на горните крайници (PUL 2.0).1Bulgarian5318349OLE Phase (Efficacy) 2.Change in Muscle strength as assessed by and percent predicted MyoGrip (using the Myoset System) from Week 25 to Week 49.False51790157dc2f3d2-f844-4b12-a806-d55997b6e7152. Промяна в мускулната издръжливост, оценена чрез процент прогнозиран MyoGrip (с помощта на системата Myoset) от Седмица 25 до Седмица 49.1Bulgarian53183510OLE Phase (Efficacy) 3.Change in Muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from Week 25 to Week 49.False5179016077c0bcc-192f-489e-a2cc-ef1e572eb2e23. Промяна в мускулната издръжливост, оценена чрез процент прогнозиран MyoPinch (с помощта на системата Myoset) от Седмица 25 до Седмица 49.1Bulgarian53183611OLE Phase (Efficacy) 4.The extent of response in a total arm functional score from Week 25 to Week 49 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, right hand and left hand MyoGrip and MyoPinch) that do not decline. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5.False51790179f0a49b4-86ed-4604-910c-d499873f03694. Степента на отговор в общия функционален резултат на терапевтичната група от Седмица 25 до Седмица 49 ще бъде оценена чрез кумулативното разпределение на броя на компонентите на общия функционален резултат на терапевтичната група (PUL2.0, дясната ръка и лявата ръка MyoGrip и MyoPinch), които не намаляват. Променливата на крайната точка ще бъде броят на компонентите със стабилизиране, което води до бройка в диапазона от 0 до 5.1Bulgarian53183712OLE Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from Week 25 to Week 49 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand.False5179018c253acec-b77b-43de-a1ee-d45d8db46ed85. Промяна в резултата на ефективността на горния крайник (PUL 2.0) от Седмица 25 до Седмица 49 за всяко от измеренията на PUL2.0; високо ниво на рамото, средно ниво на лакътя и дистално ниво на китката и ръката.1Bulgarian53183813OLE Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from Week 25 to Week 49.False517901983315056-27d5-4364-8cf9-708ab537fca96. Респираторна функция, оценена чрез процент прогнозиран PEF и процент прогнозиран ФВК от Седмица 25 до Седмица 49.1Bulgarian53183914OLE Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument PedsQL™ Duchenne Muscular Dystrophy (DMD) Module Patient Reports and Parent Reports scores (at Weeks 25, 37 and 49 and at Week 65).False5179020e36bf297-be84-4869-b3a0-ac774a21d75b7. Качество на живот, оценено чрез процент на промяната в Инструмента за качество на живот при педиатрични пациенти (PedsQL™) за мускулна дистрофия тип Дюшен (МДД) и резултатите от Доклад на пациента и Доклад на родителя (в Седмици 25, 37 и 49 и в Седмица 65).1Bulgarian53184015Combined Blinded and Open Label Phase: 1.Percentage of treatment responders in the OLE phase compared to the blinded phase in each of the 4 treatment groups; and from baseline to Week 49 for those that remained on the same dose of ATL1102.False51790219aca022a-e6ab-4f47-be8f-a358916bd659Комбинирана заслепена и открита фаза: 1. Процент на отговорилите на лечението в OLE фазата в сравнение със заслепената фаза във всяка от 4-те терапевтични групи; и от базовото ниво до Седмица 49 за тези, които са останали на същата доза ATL1102.1Bulgarian53184116Combined Blinded and Open Label Phase: 2.Change in Muscle strength as assessed by percent predicted MyoGrip (using the Myoset System) from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102.False517902237d2bbf9-1347-4817-8f1c-40ba852873fa2. Промяна в мускулната сила, оценена от и процентно предвиден MyoGrip (с помощта на системата Myoset) от базовото ниво до Седмица 25 в сравнение с промяната от Седмица 25 до Седмица 49; и от базовото ниво до Седмица 49 за тези, които са останали на същата доза ATL1102.1Bulgarian53184217Combined Blinded and Open Label Phase: 3.Change in Muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102.False517902395e77342-0407-4a5b-a142-8d42d7f31c713. Промяна в мускулната сила, оценена чрез процентно прогнозиран MyoPinch (с помощта на системата Myoset) от базовото ниво до седмица 25 в сравнение с промяната от седмица 25 до седмица 49; и от базовото ниво до седмица 49 за тези, които са останали на същата доза ATL1102.1Bulgarian53184318Combined Blinded and Open Label Phase: 4.The extent of response in total arm functional score from OLE phase compared to the blinded phase in each of the 4 treatment groups; and from baseline to Week 49 for those that remained on the same dose of ATL1102.False51790242b587c0d-52a3-4409-8fe8-ba97c9ce70f84. Степента на отговор в общ функционален резултат на рамото от OLE фаза в сравнение със заслепената фаза във всяка от 4-те терапевтични групи; и от базовото ниво до Седмица 49 за тези, които са останали на същата доза ATL1102.1Bulgarian53184419Combined Blinded and Open Label Phase: 5.Change of Performance of Upper Limb (PUL 2.0) score for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand. from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102.False5179025218f73c3-7d81-4b5e-a245-58b4ee356ecb5. Промяна на резултата от ефективността на горния крайник (PUL 2.0) за всяко от измеренията на PUL2.0; високо ниво на рамото, средно ниво на лакътя и дистално ниво на китката и ръката от базовото ниво до Седмица 25 в сравнение с промяната от Седмица 25 до Седмица 49 и от базовото ниво до Седмица 49 за тези, които са останали на същата доза ATL1102.1Bulgarian53184520Combined Blinded and Open Label Phase: 6.Respiratory function assessed by percent predicted PEF and, percent predicted FVC from baseline to Week 25 compared to the change from Week 25 to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102.False5179026546e4fa9-0690-400e-86c3-71dc94f2cdba6. Дихателната функция, оценена чрез процент на прогнозирания ВЕД и процент на прогнозирания ФВК от базовото ниво до Седмица 25 в сравнение с промяната от Седмица 25 до Седмица 49; и от базовото ниво до Седмица 49 за тези, които са останали на същата доза ATL1102.1Bulgarian53184621Combined Blinded and Open Label Phase: 7.Quality of Life assessed by percentage of change in the Paediatric Quality of Life instrument PedsQLTM Duchenne Muscular Dystrophy(DMD) Module Patient Reports and Parent Reports scores from baseline (Day1/Week 0) through to Week 25 and Week 25 through to Week 49; and from baseline to Week 49 for those that remained on the same dose of ATL1102.False5179027876e593a-d3e5-4260-83ab-38240716cbd57. 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publication)PDF21663319.02Belgium315472024-07-26T08:59:49.9862025-05-05T16:30:06.8518Ireland315522024-07-29T18:24:54.9942024-07-29T18:24:54.9948Poland315552024-08-04T20:00:14.6252025-06-19T09:53:10.6688Bulgaria315482024-07-29T09:52:28.8252025-05-08T12:08:15.7228Denmark315502024-07-24T23:58:55.2582025-05-02T12:52:19.4298Italy315532024-08-12T00:00:002025-05-06T14:41:04.748Spain315542024-07-22T10:14:04.6642025-05-05T12:46:30.1898Czechia315492024-07-25T14:35:36.4592025-05-06T11:06:35.0878Hungary315512024-07-22T13:14:23.3832025-05-07T16:07:25.472831547BelgiumSTART_OF_TRIAL2017-05-15END_OF_TRIAL2025-09-23START_OF_RECRUITMENT2017-05-31END_OF_RECRUITMENT2022-10-2431548BulgariaSTART_OF_TRIAL2019-04-30END_OF_TRIAL2025-05-08START_OF_RECRUITMENT2019-05-08END_OF_RECRUITMENT2022-03-2231549CzechiaSTART_OF_TRIAL2017-10-23END_OF_TRIAL2025-03-06START_OF_RECRUITMENT2017-10-25END_OF_RECRUITMENT2021-07-2231550DenmarkSTART_OF_TRIAL2020-03-31END_OF_TRIAL2025-04-30START_OF_RECRUITMENT2020-05-04END_OF_RECRUITMENT2022-06-0731551HungarySTART_OF_TRIAL2019-02-27END_OF_TRIAL2025-08-19START_OF_RECRUITMENT2019-05-09END_OF_RECRUITMENT2022-09-0731552IrelandSTART_OF_TRIAL2021-01-13END_OF_TRIAL2025-01-07START_OF_RECRUITMENT2021-02-18END_OF_RECRUITMENT2022-02-1431553ItalySTART_OF_TRIAL2017-07-04END_OF_TRIAL2025-10-09START_OF_RECRUITMENT2017-08-02END_OF_RECRUITMENT2022-11-0231554SpainSTART_OF_TRIAL2017-06-19END_OF_TRIAL2025-10-16START_OF_RECRUITMENT2017-09-14END_OF_RECRUITMENT2022-10-2731555PolandSTART_OF_TRIAL2019-04-30END_OF_TRIAL2025-09-29START_OF_RECRUITMENT2019-07-02END_OF_RECRUITMENT2022-09-202025-10-172024-514698-23-008A Double-blind, Placebo-Controlled, Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy4045-30115/05/201709/10/2025Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping.Belgium:8Ireland:8Poland:8Bulgaria:8Denmark:8Italy:8Spain:8Czechia:8Hungary:822/07/2024IE: 29/07/2024, BE: 26/07/2024, BG: 29/07/2024, CZ: 25/07/2024, DK: 24/07/2024, HU: 22/07/2024, IT: 12/08/2024, ES: 22/07/2024, PL: 04/08/2024Diseases [C] - Musculoskeletal Diseases [C05]Sarepta Therapeutics Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)• Change from Baseline at Week 96 in 6MWT • Change from Baseline at Week 96 in rise from floor velocity (rise/second) • Change from Baseline at Week 144 in 4-step ascend velocity (step/second), • Change from Baseline at Week 96 in 10MWR velocity (meter/second) • Change from Baseline at Weeks 48 or 96 in the quantity of dystrophin protein expression as measured by Western blot of biopsied muscle tissue., • Change from Baseline at Weeks 48 or 96 in the intensity of dystrophin expression in biopsied muscle tissue, as measured by IHC. •Change from Baseline at Week 96 in: NSAA total scoreCASIMERSEN (SRP-4045), GOLODIRSEN (SRP-4053), 0,9% sodium chloride650-17 yearsMale38117/10/2025Double-blind period: Change from Baseline at Week 96 in 4-step ascend velocity (step/second)No19/06/202504/02/20262024-514698-23-00Ended2017-05-152025-10-092024-07-22T10:14:04.6642026-02-04T02:42:39.925028584889261247100000000325Argentina32ARARGTrue100000000465Mexico484MXMEXTrue100000000329Australia36AUAUSTrue100000000556United Kingdom826GBGBRTrue100000000506Russian Federation643RURUSTrue100000000439Korea, Republic of410KRKORTrue100000000518Serbia688RSSRBTrue366716110281534SOLUTION FOR INFUSIONPRD94568141CASIMERSEN (SRP-4045)SOLUTION FOR INFUSIONSRP-4045CASIMERSENSUB188601CASIMERSENSAREPTA THERAPEUTICS INC10281534288376SAREPTA THERAPEUTICS INCNucleic AcidNucleic AcidCASIMERSENSUB188601False1Falsemg/kg milligram(s)/kilogram30mg/kg milligram(s)/kilogram43201442OligomerPRD9456814SRP-404511INTRAVENOUS USEFalseCASIMERSEN (SRP-4045)casimersenSOLUTION FOR INFUSION366717110771830CONCENTRATE FOR SOLUTION FOR INFUSIONPRD19599021GOLODIRSEN (SRP-4053)CONCENTRATE FOR SOLUTION FOR INFUSIONSRP-4053GOLODIRSENSUB188593GOLODIRSENSAREPTA THERAPEUTICS INC10771830337900SAREPTA THERAPEUTICS INCNucleic AcidNucleic AcidGOLODIRSENSUB188593False1Falsemg/kg milligram(s)/kilogram30mg/kg milligram(s)/kilogram43201442OligomerPRD1959902SRP-405311INTRAVENOUS USEFalseGOLODIRSEN (SRP-4053)golodirsenCONCENTRATE FOR SOLUTION FOR INFUSION3667151N/AN/AN/A0,9% sodium chlorideN/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalse0,9% sodium chloriden/aN/AA Double-blind, Placebo-Controlled, Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy686142125150f08-4ab5-4a7d-8605-28fc8b8dc68bKettős vak, placebo-kontrollos, multicentrikus vizsgálat nyílt, kiterjesztett szakasszal az SRP-4045 és az SRP-4053 hatásosságának és biztonságosságának értékelésére Duchenne-izomdystrophiában szenvedő betegeknél.13Hungarian686142025150f08-4ab5-4a7d-8605-28fc8b8dc68bBadanie wieloośrodkowe, prowadzone metodą podwójnie ślepej próby, kontrolowane placebo, z kontynuacją prowadzoną metodą otwartej próby, oceniające skuteczność i bezpieczeństwo stosowania produktów SRP-4045 i SRP-4053 u pacjentów z dystrofią mięśniową Duchenne'a.19Polish686142225150f08-4ab5-4a7d-8605-28fc8b8dc68bДвойно сляпо, плацебо контролирано многоцентрово изпитване с открито продължение за оценка на ефикасността и безопасността на SRP-4045 и SRP-4053 при пациенти с мускулна дистрофия на Дюшен.1Bulgarian686142325150f08-4ab5-4a7d-8605-28fc8b8dc68bEstudio multicéntrico, a doble ciego y controlado con placebo, con una extensión abierta para evaluar la eficacia y la seguridad de SRP-4045 y SRP-4053 en pacientes con distrofia muscular de Duchenne.7SpanishA research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients68614700e728c91-dee6-479e-94d9-6e0e7f38ba3bBadanie dotyczące nowego produktu leczniczego do stosowania w leczeniu pacjentów z dystrofią mięśniową Duchenne'a19Polish68614710e728c91-dee6-479e-94d9-6e0e7f38ba3bEstudio de investigación de un nuevo medicamento en estudio para el tratamiento de pacientes con distrofia muscular de Duchenne7Spanish68614690e728c91-dee6-479e-94d9-6e0e7f38ba3bDuchenne-féle izomdisztrófiában szenvedő betegek kezelésére szolgáló új vizsgálati készítmény kutatási vizsgálata13Hungarian68614680e728c91-dee6-479e-94d9-6e0e7f38ba3bКлинично изпитване на нов експериментален лекарствен продукт за лечение на пациенти с мускулна дистрофия на Дюшен1Bulgarian4045-301394378NCT02500381False52The present trial is a phase III clinical trial and therefore enters into category 2 regarding disclosure rules8028091632Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping.6861364c2c587bf-fc25-498f-a07a-00714a9187e5Distrofia muscular de Duchenne susceptible de tratamiento de omisión de los exones 45 o 53.7Spanish6861367c2c587bf-fc25-498f-a07a-00714a9187e5A 45. vagy 53. exon átugrásával befolyásolható Duchenne-féle izomdisztrófiában szenvedő betegek13Hungarian6861365c2c587bf-fc25-498f-a07a-00714a9187e5Пациенти с мускулна дистрофия на Дюшен, податливи на прескачане на екзон 45 или 53.1Bulgarian6861366c2c587bf-fc25-498f-a07a-00714a9187e5pacjenci z dystrofią mięśniową Duchenne'a podatną na pomijanie egzonu 45 lub 5319PolishTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False32653985265397626539542653967265399Evaluate the effect of SRP-4045 and SRP-4053(combined-active group) compared with placebo on ambulation and muscle function, as measured by the 4-step ascend velocity.6861466dc93ccb0-e278-434f-8b14-818a6cd1edb5ocena wpływu SRP 4045 oraz SRP 4053 (grupa pacjentów z którymkolwiek rodzajem leczenia czynnego) w porównaniu do placebo na zdolność do chodzenia oraz czynność mięśni, zgodnie z wynikami testu oceniającego czas na pokonanie 4 schodów.19Polish6861465dc93ccb0-e278-434f-8b14-818a6cd1edb5Да се оцени ефекта на SRP-4045 и SRP-4053 (комбинирана активна група) спрямо плацебо1Bulgarian6861467dc93ccb0-e278-434f-8b14-818a6cd1edb5по отношение на ходенето, издръжливостта и мускулната функция, измерени с помощта на тест за скорост на изкачване на 4 стъпала13Hungarian6861464dc93ccb0-e278-434f-8b14-818a6cd1edb5Evaluar el efecto de SRP-4045 y SRP-4053 (grupo de tratamiento activo combinado) en comparación con un placebo sobre la deambulación y la función muscular, determinadas mediante la velocidad para subir 4 escalones.7Spanish3028971Double-blind period: evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) on: • Dystrophin protein expression in biopsied muscle tissue as measured by: - Western blot (quantification) - Immunohistochemistry (IHC) fiber intensity6861475e0a02283-23e3-4b6c-a519-317c3b8a9f4eKettős vak szakasz: az SRP-4045 és SRP-4053 (kombinált aktív csoport) hatásának értékelése az alábbiak szempontjából: • A disztrofin fehérje expressziója biopsziás izomszövetben, az alábbiakkal mérve: - Western blot (mennyiségi meghatározás) - Immunhisztokémiával (IHC) meghatározott rostintenzitás13Hungarian6861474e0a02283-23e3-4b6c-a519-317c3b8a9f4eokres leczenia w fazie podwójnie zaślepionej: ocena wpływu SRP 4045 oraz SRP 4053 (grupa pacjentów z którymkolwiek rodzajem leczenia czynnego) na: • ekspresję dystrofiny w tkance mięśniowej pobranej w czasie biopsji, zgodnie z oceną metodami: - western blot (analiza ilościowa); - immunohistochemiczna (ang. immunohistochemistry, IHC) ocena gęstości włókien;19Polish6861472e0a02283-23e3-4b6c-a519-317c3b8a9f4ePeriodo doble ciego: evaluar el efecto de SRP-4045 y SRP-4053 (combinados, grupo de principio activo) sobre • la expresión de la proteína distrofina en el tejido muscular biópsico, según: - La cuantificación mediante inmunoelectrotransferencia (Western blot). - La intensidad de expresión de las fibras mediante pruebas inmunohistoquímicas (IHQ).7Spanish6861473e0a02283-23e3-4b6c-a519-317c3b8a9f4eДвойно сляп период: оценка на ефекта на SRP-4045 и SRP-4053 (комбинирана активна група) по отношение на: • Експресия на белтъка дистрофин в биопсична мускулна тъкан, измерена чрез: - Тест Western blot (количествено определяне) - Интензитет на фибрите, определен чрез имунохистохимия (IHC)1Bulgarian3028982Functional status as measured by: • 6MWT • 10MWR • 4-step ascend velocity • Rise from floor velocity • North Star Ambulatory Assessment (NSAA)686147773c58d57-6d69-44cd-a154-5f84e3d2e0fefunkcjonalny status mierzony testem 6 minutowego marszu (6MWT, ang. 6 minute walk test), testem 10 metrowego marszu/biegu (10MWR, ang. 10-meter walk/run), testem oceniającym czas na pokonanie 4 schodów; ocenę zdolności do samodzielnego wstawania z podłogi, ocenę zdolności do chodzenia w skali NSAA (NSAA, ang. North Star Ambulatory Assessment)19Polish686147673c58d57-6d69-44cd-a154-5f84e3d2e0feEl estado funcional determinado mediante: • la prueba de marcha de seis minutos (PM6M) • la prueba de caminar/correr 10 metros • la velocidad para subir 4 escalones • la velocidad para levantarse del suelo • la evaluación ambulatoria North Star (NSAA)7Spanish686147973c58d57-6d69-44cd-a154-5f84e3d2e0fe-Funkcionális állapot a következők szerint: - 6 perces járásteszt (6MWT) - 10 méteres járásteszt (10MWR) - 4 lépéses emelkedési sebesség - Talajról való felállás sebessége - North Star ambuláns vizsgálat (NSAA)13Hungarian686147873c58d57-6d69-44cd-a154-5f84e3d2e0feФункционален статус, измерен чрез: - 6 -минутен тест за ходете - 10 метра ходене или тичане - Скорост на изкачване на 4 стъпала - Скорост на ставане от пода - Оценка на подвижността на пациента по системата North Star (NSAA)1Bulgarian3028993Double-blind period: evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) on: • Safety and tolerability of SRP-4045 and SRP-4053.6861480d474e63d-cf84-46bf-80d4-41dd9c7636ddДвойно сляп период: оценка на ефекта на SRP-4045 и SRP-4053 (комбинирана активна група) по отношение на: • Безопасност и поносимост на SRP-4045 и SRP-4053.1Bulgarian6861483d474e63d-cf84-46bf-80d4-41dd9c7636ddKettős vak szakasz: az SRP-4045 és SRP-4053 (kombinált aktív csoport) hatásának értékelése az alábbiak szempontjából: • Az SRP-4045 és SRP-4053 biztonságossága és tolerálhatósága.13Hungarian6861482d474e63d-cf84-46bf-80d4-41dd9c7636ddPeriodo doble ciego: evaluar el efecto de SRP-4045 y SRP-4053 (combinados, grupo de principio activo) sobre • La seguridad y la tolerabilidad de SRP-4045 y SRP-4053.7Spanish6861481d474e63d-cf84-46bf-80d4-41dd9c7636ddokres leczenia w fazie podwójnie zaślepionej: ocena wpływu SRP 4045 oraz SRP 4053 (grupa pacjentów z którymkolwiek rodzajem leczenia czynnego) na: • bezpieczeństwo stosowania i tolerancję SRP 4045 i SRP 4053.19Polish3029004Open-label Treatment Period: • Evaluate the long-term effects of SRP-4045 and SRP-4053 treatment on functional status up to 144 weeks.68614853a03e2c0-5eac-4eb2-9a28-ef6795551fa7Periodo abierto de tratamiento del estudio: • Evaluar los efectos a largo plazo del tratamiento con SRP-4045 y SRP-4053 en el estado funcional de los pacientes hasta las 144 semanas7Spanish68614863a03e2c0-5eac-4eb2-9a28-ef6795551fa7Период на открито лечение: • Оценка на дългосрочните ефекти на лечението със SRP-4045 и SRP-4053 върху функционалния статус в продължение на до 144 седмици.1Bulgarian68614873a03e2c0-5eac-4eb2-9a28-ef6795551fa7Okres leczenia w fazie otwartej: • ocena długoterminowego wpływu leczenia SRP 4045 oraz SRP 4053 na zaburzenia czynnościowe w okresie do 144 tygodni;19Polish68614843a03e2c0-5eac-4eb2-9a28-ef6795551fa7Nyílt kezelési szakasz: • Az SRP-4045 és SRP-4053 kezelés funkcionális státuszra gyakorolt hosszú távú hatásainak értékelése a 144. hét végéig.13Hungarian3029015Open-label Treatment Period: • Evaluate the long-term safety and tolerability of SRP-4045 and SRP-4053.686149071d23544-de8b-48f1-9a19-9fc585d8e293Okres leczenia w fazie otwartej: • ocena długoterminowa bezpieczeństwa stosowania i tolerancji na leczenie SRP 4045 oraz SRP 4053;19Polish686149171d23544-de8b-48f1-9a19-9fc585d8e293Nyílt kezelési szakasz: • Az SRP-4045 és SRP-4053 hosszú távú biztonságosságának és tolerálhatóságának értékelése.13Hungarian686148871d23544-de8b-48f1-9a19-9fc585d8e293Период на открито лечение: • Оценка на дългосрочната безопасност и поносимост на SRP-4045 и SRP-4053.1Bulgarian686148971d23544-de8b-48f1-9a19-9fc585d8e293Periodo abierto de tratamiento del estudio: • Evaluar la seguridad y la tolerabilidad a largo plazo de SRP-4045 y SRP-40537Spanish3029026Pharmacokinetic Objective: Evaluate the PK properties of SRP-4045 and SRP-4053 using a population PK model.6861494231622a7-1744-4da6-abb4-1f3d871c7310Objetivo farmacocinético: Evaluar las propiedades FC de SRP-4045 y SRP-4053 mediante un modelo farmacocinético poblacional.7Spanish6861495231622a7-1744-4da6-abb4-1f3d871c7310Cel farmakokinetyczny: ocena własności farmakokinetycznych SRP 4045 oraz SRP 4053 z wykorzystaniem modelu farmakokinetyki populacyjnej.19Polish6861492231622a7-1744-4da6-abb4-1f3d871c7310Фармакокинетична цел: Оценка на ФК свойствата на SRP-4045 и SRP-4053 с помощта на популационен ФК модел.1Bulgarian6861493231622a7-1744-4da6-abb4-1f3d871c7310Farmakokinetikai célkitűzés: Az SRP-4045 és SRP-4053 PK jellemzőinek értékelése populációs PK modell segítségével.13Hungarian5845321Is a male with an established clinical diagnosis of DMD and an out-of-frame deletion amenable to: Exon 45 skipping (including but not limited to deletions of exons such as 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53, or 46-55) OR Exon 53 skipping (including but not limited to deletions of exons such as 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52, or 54-58) As documented prior to screening by a genetic report from an accredited laboratory defining deletion endpoints by multiplex ligation-dependent probe amplification or sequencing. The patient's amenability to exon 45 or exon 53 skipping must be confirmed prior to first dose using the genotyping results obtained during Screening.686142624a16e3e-902b-465a-8480-43be77227784pacjent płci męskiej z potwierdzonym rozpoznaniem dystrofii mięśniowej Duchenne’a oraz delecją zmieniającą ramkę odczytu, odpowiedzialną za: • pominięcie egzonu 45 (włączając w to, ale nie ograniczając do delecji egzonów takich jak 12–44, 18–44, 44, 46–47, 46–48, 46–49, 46–51, 46–53 lub 46–55); LUB • pominięcie egzonu 53 (włączając w to, ale nie ograniczając do delecji egzonów takich jak 42–52, 45–52, 47–52, 48–52, 49–52, 50–52, 52 lub 54–58); zgodnie z raportem zawierającym wyniki badań genetycznych przeprowadzonych w akredytowanym laboratorium przed rozpoczęciem badań przesiewowych, wykazującym granice delecji poprzez multipleksową amplifikację zależną od ligacji lub poprzez sekwencjonowanie. Delecja egzonów 45 lub 53 u pacjenta musi zostać potwierdzona z wykorzystaniem genotypowania przeprowadzonego w czasie badań przesiewowych przed podaniem pierwszej dawki19Polish686142524a16e3e-902b-465a-8480-43be77227784Fiú DMD megállapított klinikai diagnózisával és out-of-frame delécióval, amely befolyásolható az alábbiakkal: • A 45. exon átugrása (beleértve többek között a következő exonok delécióját: 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53 vagy 46-55) VAGY • Az 53. exon átugrása (beleértve többek között a következő exonok delécióját: 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52 vagy 54-58) Akkreditált laboratórium által a szűrés előtt kiadott genetikai lelettel dokumentálva, amely tartalmazza a multiplex ligáció-függő próba amplifikációval vagy szekvenálással meghatározott deléciós végpontokat. A betegnél a 45. exon vagy az 53. exon szkipping általi befolyásolhatóságot az első adag beadása előtt igazolni kell a szűrés alatt kapott genotípus-meghatározás eredményeit felhasználva.13Hungarian686142424a16e3e-902b-465a-8480-43be77227784Ser varón con un diagnóstico clínico confirmado de DMD y una deleción fuera del marco de lectura susceptible de: • Omisión del exón 45 (incluida, entre otras, la deleción de los exones 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53 o 46-55) O BIEN • Omisión del exón 53 (incluida, entre otras, la deleción de los exones 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52 o 54-58) documentada antes de la selección mediante un informe genético de un laboratorio acreditado en el que se defina el valor máximo de deleción mediante secuenciación o amplificación múltiple de sondas ligadas. La susceptibilidad del paciente de recibir una deleción de los exones 45 o 53 se debe confirmar antes de administrar la primera dosis del fármaco. Para ello, han de utilizarse los resultados de genotipado obtenidos durante la selección.7Spanish686142724a16e3e-902b-465a-8480-43be77227784Е от мъжки пол с установена клинична диагноза МДД и делеция извън рамката, податлив на: • Прескачане на екзон 45 (включително, но не само, делеции на екзони като 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53 или 46-55) ИЛИ • Прескачане на екзон 53 (включително, но не само, делеции на екзони като 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52 или 54-58) Документирано преди скрининга чрез генетичен доклад от акредитирана лаборатория, дефиниращ крайните точки на делеция чрез мултиплексна лигазно зависима амплификация или секвениране на проби. Податливостта на пациента към прескачане на екзон 45 или екзон 53 трябва да бъде потвърдена преди първата доза с помощта на получените по време на скрининга резултати за определяне на генотипа1Bulgarian58453310Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.6861430bbfea091-2093-4e8a-860b-3692b1ac4516Estar dispuesto a facilitar su asentimiento informado (si procede) y tener un(os) progenitor(es) o tutor(es) legal(es) dispuesto(s) a facilitar su consentimiento informado por escrito para que el paciente participe en el estudio.7Spanish6861431bbfea091-2093-4e8a-860b-3692b1ac4516Kész a tájékozott hozzájárulásra (ha szükséges), és a szülő(k) vagy a törvényes gondviselő(k) kész(ek) írásos, tájékozott beleegyezésüket adni a beteg vizsgálatban való részvételéhez.13Hungarian6861428bbfea091-2093-4e8a-860b-3692b1ac4516pacjent jest gotowy na wyrażenie pisemnej zgody na udział (formularz świadomej zgody dla pacjenta małoletniego) (jeśli dotyczy) oraz jego rodzic lub opiekun prawny jest gotowy wyrazić pisemną zgodę na jego udział (formularz świadomej zgody).19Polish6861429bbfea091-2093-4e8a-860b-3692b1ac4516Желае да даде информирано одобрение (ако е приложимо) и има родител(и) или законен(ни) настойник(ци), който(които) може(могат) да даде(ат) писмено информирано съгласие за участието на пациента в изпитването.1Bulgarian5845342Is between 6 and 13 years of age, inclusive, at randomization for patients amenable to exon 53 skipping; or is between 7 and 13 years of age, inclusive, at randomization for patients amenable to exon 45 skipping.6861433ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6bPara la aleatorización al grupo de pacientes susceptibles de la omisión del exón 53, tener entre 6 y 13 años de edad (incluidos); para la aleatorización al grupo de pacientes susceptibles de la omisión del exón 45, tener entre 7 y 13 años de edad (incluidos).7Spanish6861432ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6bAz életkor ≥6 és ≤13 év közötti randomizáláskor azoknál a betegeknél, akiknél a betegség az 53. exon átugrásával befolyásolható, vagy az életkor ≥7 és ≤13 év közötti randomizáláskor azoknál a betegeknél, akiknél a betegség a 45. exon átugrásával befolyásolható.13Hungarian6861434ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6bw momencie randomizacji pacjent musi być w wieku od 6 do 13 lat w przypadku pacjentów podatnych na pominięcie egzonu 53 lub w momencie randomizacji pacjent musi być w wieku od 7 do 13 lat w przypadku pacjentów podatnych na pominięcie egzonu 45.19Polish6861435ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6bЕ на възраст между 6 и 13 години включително към момента на рандомизацията, за пациенти, податливи на прескачане на екзон 53; или е на възраст между 7 и 13 години включително към момента на рандомизацията, за пациенти, податливи на прескачане на екзон 45.1Bulgarian5845353Has stable pulmonary function (FVC % of predicted ≥50% and no requirement for nocturnal ventilation) that, in the Investigator's opinion, is unlikely to decompensate over the duration of the study.6861439291b79a5-d06c-4f8e-885a-04acdafb0858czynności oddechowe muszą być stabilne (odsetek wartości należnej FVC ≥50% i nie wymaga wentylacji mechanicznej w nocy), które w opinii badacza nie ulegną dekompensacji w czasie badania.19Polish6861437291b79a5-d06c-4f8e-885a-04acdafb0858Légzésfunkciója stabil (a várt érték százalékában megadott FVC ≥ 50% és éjszakai lélegeztetés nem szükséges), és a vizsgáló véleménye szerint valószínűleg nem fog dekompenzálódni a vizsgálat időtartama alatt.13Hungarian6861438291b79a5-d06c-4f8e-885a-04acdafb0858Има стабилна белодробна функция (предвиден % FVC ≥50% и без необходимост от нощна вентилация), която, по преценка на изследователя, няма да се декомпенсира по време на изпитването.1Bulgarian6861436291b79a5-d06c-4f8e-885a-04acdafb0858Presentar una función pulmonar estable (% CVF ≥50 % de la prevista, y sin necesidad de ventilación mecánica nocturna) que, según la opinión del investigador, probablemente no se descompense durante el estudio.7Spanish5845364Has intact right and left biceps brachii muscles (the preferred biopsy site) or 2 alternative upper arm muscle groups.6861440583cb2b1-0b11-4e81-a6f4-c859399aa25cA jobb és bal musculus biceps brachii (a preferált biopsziás hely) vagy 2 másik felkari izomcsoport intakt.13Hungarian6861442583cb2b1-0b11-4e81-a6f4-c859399aa25cИма непокътнати десен и ляв двуглав мишничен мускул (предпочитано място за биопсия) или 2 алтернативни мускулни групи в горната част на ръката.1Bulgarian6861443583cb2b1-0b11-4e81-a6f4-c859399aa25cPresentar los músculos bíceps braquiales izquierdo y derecho (zona preferida para la biopsia) o 2 grupos de músculos del brazo alternativos intactos.7Spanish6861441583cb2b1-0b11-4e81-a6f4-c859399aa25cmusi mieć nienaruszony prawy i lewy mięsień dwugłowy ramienia (preferowane miejsce biopsji) lub dwie inne grupy mięśniowe w obrębie kończyny górnej.19Polish5845375Has been on a stable dose or dose equivalent of oral corticosteroids for at least 24 weeks prior to Week 1, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight)686144513e66846-08a7-4309-b608-5174d5d186cfVáltozatlan dózisban vagy ekvivalens dózisban orális kortikoszteroidot szedett az 1. hetet megelőző legalább 24 héten át, és a dózis várhatóan változatlan marad a vizsgálat alatt (a testtömeg változása miatt szükséges módosításokat kivéve)13Hungarian686144713e66846-08a7-4309-b608-5174d5d186cfЕ на стабилна доза или дозов еквивалент перорални кортикостероиди в продължение на най-малко 24 седмици преди седмица 1 и се очаква дозата да остане непроменена по време на изпитването (с изключение на модификации, отразяващи промените в теглото)1Bulgarian686144613e66846-08a7-4309-b608-5174d5d186cfHaber recibido una dosis estable o equivalente de corticoesteroides orales durante al menos 24 semanas antes de la semana 1; siempre que se prevea que la dosis se mantenga durante todo el estudio (excepto en el caso de las modificaciones por cambios de peso).7Spanish686144413e66846-08a7-4309-b608-5174d5d186cfotrzymywał stałą dawkę lub ekwiwalent dawki doustnych kortykosteroidów przez przynajmniej 24 tygodnie przed 1. tygodniem i przewiduje się, że dawka pozostanie niezmieniona w czasie badania (poza modyfikacjami uwzględniającymi zmianę masy ciała.19Polish5845386If taking angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), β adrenergic blockers, aldosterone receptor antagonists, potassium, or coenzyme Q, has been on a stable dose for at least 12 weeks prior to Week 1 and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).6861449eb7a5126-13fb-4264-8b8c-686809e6d362jeżeli pacjent przyjmuje inhibitory enzymu konwertującego angiotensynę (ACE), czynniki blokujące receptor dla angiotensyny (ARBs, ang. angiotensin receptor blocking agents), blokery β adrenergiczne, antagonistów receptora aldosteronu, potas lub koenzym Q, przyjmował stałą dawkę przez co najmniej 12 tygodni przed 1. tygodniem i przewiduje się, że dawka pozostanie niezmieniona w czasie badania (poza modyfikacjami uwzględniającymi zmianę masy ciała).19Polish6861451eb7a5126-13fb-4264-8b8c-686809e6d362Ha angiotenzin-konvertáló enzim (ACE) inhibitorokat, angiotenzin receptor blokkoló gyógyszereket (ARB), β-adrenerg blokkolókat, aldoszteron-receptor antagonistákat, káliumot vagy koenzim Q-t szed, ezeket változatlan dózisban kellett kapnia az 1. hetet megelőző legalább 12 héten át, és a dózisnak várhatóan változatlannak kell maradnia a vizsgálat alatt (a testtömeg változása miatt szükséges módosításokat kivéve).13Hungarian6861448eb7a5126-13fb-4264-8b8c-686809e6d362Ако приема инхибитори на ангиотензин конвертиращия ензим (ACE), ангиотензин рецепторни блокери (ARB), β-адренергични блокери, алдостерон рецепторни антагонисти, калий или коензим Q, е на стабилна доза в продължение на най-малко 12 седмици преди седмица 1 и се очаква дозата да остане непроменена по време на изпитването (с изключение на модификации, отразяващи промените в теглото).1Bulgarian6861450eb7a5126-13fb-4264-8b8c-686809e6d362En el caso de que el paciente tome inhibidores de la enzima convertidora de la angiotensina (ECA), antagonistas de los receptores de la angiotensina (ARA), antagonistas adrenérgicos β, antialdosterónicos, potasio o ubiquinona, haber recibido una dosis estable durante al menos 12 semanas antes de la semana 1; siempre que se prevea que la dosis se mantenga durante todo el estudio (excepto en el caso de las modificaciones por cambios de peso).7Spanish5845397Achieved a mean 6MWT distance of ≥300 to ≤ 450 meters (without assistance) at both the Screening and Baseline visits (prior to Week 1). The mean 6MWT distance at the Screening and Baseline visits is the average of 2 separate assessments on 2 consecutive business days at each visit. The Baseline mean (average of Baseline Days 1 and 2) must be within 15% of the Screening mean distance (average of Screening Days 1 and 2).68614525e7b81eb-9aa2-4d41-acbe-8770455b0f4eAlcanzar una distancia media en la PM6M ≥300 y ≤450 metros (sin ayuda) tanto en la visita de selección como en la visita basal (antes de la semana 1). La distancia media de la PM6M en las visitas de selección y basal es el promedio de 2 evaluaciones independientes llevadas a cabo en 2 días laborables consecutivos para cada visita. La distancia media basal (promedio de los días 1 y 2 de la visita basal) debe encontrarse dentro del 15 % de la distancia media de la selección (promedio de los días 1 y 2 de la visita de selección).7Spanish68614535e7b81eb-9aa2-4d41-acbe-8770455b0f4eAz átlagos 6MWT távolság a szűrő és a kiindulási viziteken (az 1. hét előtt) egyaránt ≥300 – ≤450 méter (segítség nélkül). A szűrő és a kiindulási viziteken mért átlagos 6MWT távolság mindegyik vizit esetén 2 egymást követő munkanapon végzett 2 külön értékelés átlaga. A kiindulási átlagnak (az 1. és 2. kiindulási nap átlaga) a szűrési átlagos távolság (az 1. és 2. szűrési nap átlaga) 15%-án belülinek kell lennie.13Hungarian68614545e7b81eb-9aa2-4d41-acbe-8770455b0f4epacjent w teście 6MWT uzyskał średni dystans od ≥300 do ≤450 metrów (bez pomocy) zarówno w czasie badań przesiewowych, jak i w czasie wizyty początkowej (przed 1. tygodniem). Średni dystans w teście 6MWT w czasie badań przesiewowych i w czasie wizyty początkowej jest średnią z 2 niezależnych badań w 2 kolejnych dniach roboczych dla każdej wizyty. Średnia wartość początkowa (średnia dla początkowych dnia 1 i 2) musi mieścić się w 15% średniej wartości dystansu z badań przesiewowych (średnia dni 1 i 2 badań przesiewowych).19Polish68614555e7b81eb-9aa2-4d41-acbe-8770455b0f4eПри 6MWT е достигнал средно разстояние от ≥300 до ≤ 450 метра (без помощ) както на скрининговата визита, така и на визитата за изходно ниво (преди седмица 1). Средното разстояние при 6MWT на скрининговата визита и визитата за изходно ниво е средната стойност от 2 отделни оценки, извършени в 2 последователни работни дни при всяка визита. Средното разстояние от изходното ниво (средно за дни 1 и 2 от изходното ниво) трябва да бъде в рамките на 15% от средното разстояние от скрининга (средно за дни 1 и 2 от скрининга).1Bulgarian5845408If sexually active, agrees to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose. The sexual partner must also use a medically acceptable form of contraceptive (eg, female oral contraceptives) during this time frame. Acceptable methods of contraception include combined (estrogen and progesterone containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal); progesterone-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intrauterine hormone-releasing system; bilateral tubal occlusion, vasectomized partner; sexual abstinence (True abstinence: when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence: such as calendar, ovulation, symptothermal, post-ovulation methods] and withdrawal are not acceptable methods of contraception); or condom in combination with either cap, diaphragm, or sponge with spermicide (double-barrier contraception).6861458b61c3bdd-62d9-4ac0-b4b8-9833ca450d8bEn el caso de que el paciente sea sexualmente activo, aceptar utilizar preservativo masculino durante todo el estudio y durante los 90 días posteriores a la última dosis del fármaco del estudio. La pareja sexual del paciente participante también debe utilizar un método anticonceptivo aceptable desde el punto de vista médico (p. ej., anticonceptivos orales) durante este mismo periodo. Entre los métodos anticonceptivos aceptables, se incluyen los anticonceptivos hormonales combinados (con estrógenos y progesterona) de inhibición de la ovulación (orales, intravaginales o transdérmicos); anticonceptivos hormonales únicamente de progesterona de inhibición de la ovulación (orales, inyectables o implantables); dispositivo intrauterino; sistema intrauterino liberador de un tratamiento anticonceptivo hormonal; oclusión tubárica bilateral; pareja vasectomizada; abstinencia sexual (abstinencia real, siempre que este sea el estilo de vida usual y preferido del sujeto; la abstinencia periódica, como la que se da en los métodos del calendario, de la ovulación, sintotérmico y de la posovulación, así como el coito interrumpido, no son métodos anticonceptivos aceptables) o preservativo con capuchón, diafragma o esponja con espermicida (métodos de doble barrera).7Spanish6861459b61c3bdd-62d9-4ac0-b4b8-9833ca450d8bАко е сексуално активен, е съгласен да използва мъжки презерватив при полови контакти през цялата продължителност на изпитването и в продължение на 90 дни след последната доза. Сексуалната партньорка също трябва да използва приемлива от медицинска гледна точка форма на контрацепция (напр. женски перорални контрацептиви) през този период. Приемливите методи за контрацепция включват комбинирана (съдържаща естроген и прогестерон) хормонална контрацепция, свързана с инхибиране на овулацията (перорална, интравагинална или трансдермална); съдържаща само прогестерон контрацепция, свързана с инхибиране на овулацията (перорална, инжектируема или имплантируема); вътрематочно устройство; хормон-освобождаваща вътрематочна система; двустранна оклузия на тръбите, преминал вазектомия партньор; сексуално въздържание (действително въздържание: когато това съответства на предпочитания и обичаен начин на живот на участника. Периодично въздържание: например, календарни, овулационни, симптотермални и пост-овулационни методи, и преждевременно прекратяване на половия акт не са приемливи методи за контрацепция); или презерватив в комбинация с шапка, диафрагма или гъба със спермицид (двойно-бариерна контрацепция).1Bulgarian6861457b61c3bdd-62d9-4ac0-b4b8-9833ca450d8bHa aktív nemi életet él, bele kell egyeznie, hogy nemi kapcsolat esetén a vizsgálat teljes időtartama alatt és az utolsó dózist követően 90 napig férfi óvszert használ. A szexuális partnernek is orvosilag elfogadott fogamzásgátló módszert kell használnia (pl. női orális fogamzásgátló) ugyanezen időtartam alatt. Az elfogadható fogamzásgátló módszerek közé tartozik a kombinált (ösztrogén és progeszteron tartalmú) hormonális fogamzásgátlás, ami a tüszőrepedés (ovuláció) gátlásán alapszik (orális, intravaginális vagy transzdermális); a csak progeszteront tartalmazó hormonális fogamzásgátlás, ami az ovuláció gátlásán alapszik (orális, injekciós vagy implantátum); méhen belüli eszköz; méhen belüli hormonfelszabadító rendszer; kétoldali petevezeték elkötés, vazektómián átesett partner; tartózkodás a nemi közösüléstől (szexuális absztinencia) (tényleges absztinencia, ha ez a vizsgálati alany által előnyben részesített és megszokott életmóddal összhangban van. A nemi közösüléstől való időszakos tartózkodás, például a naptár-módszer, az ovulációs, szimptotermális, posztovulációs módszerek, továbbá a megszakításos közösülés nem elfogadható fogamzásgátló módszerek); vagy óvszer méhsapkával, pesszáriummal vagy spermicides szivaccsal kombinálva (kettős barrier-elvű fogamzásgátlás).13Hungarian6861456b61c3bdd-62d9-4ac0-b4b8-9833ca450d8bjeżeli pacjent jest aktywny seksualnie, musi wyrazić zgodę na stosowanie prezerwatywy w czasie kontaktów seksualnych w całym okresie leczenia oraz do 90 dni od podania ostatniej dawki. Partnerka seksualna w tym okresie musi również stosować medycznie zatwierdzone środki antykoncepcyjne (np. doustna antykoncepcja dla kobiet). Do dopuszczalnych metod antykoncepcji należą: złożone (zawierające estrogen i progesteron) hormonalne środki antykoncepcyjne hamujące owulację (doustne, dopochwowe lub przezskórne); hormonalne środki antykoncepcyjne hamujące owulację, zawierające wyłącznie progesteron (doustne, wstrzykiwane lub wszczepialne); wkładka domaciczna; wkładka domaciczna uwalniająca hormony; obustronne zamknięcie jajowodów; partner po wazektomii; abstynencja seksualna (pełna abstynencja, jeśli jest to zgodne z preferowanym i zwykłym stylem życia pacjentki. Okresowa abstynencja np. metoda rytmu kalendarzowego, metody objawowo-termiczne [owulacyjne i poowulacyjne] oraz stosunek przerywany nie są dopuszczalnymi metodami antykoncepcji); lub prezerwatywa w połączeniu z kapturkiem, diafragmą lub gąbką ze środkiem plemnikobójczym (podwójna antykoncepcja barierowa).19Polish5845419Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements.68614630065fe7e-e51d-414e-9a49-6e9f8e148ce6ma partnera lub opiekuna prawnego, który jest w stanie zrozumieć wymogi badania i się do nich stosować.19Polish68614600065fe7e-e51d-414e-9a49-6e9f8e148ce6Tener un(os) progenitor(es) o tutor(es) legal(es) que pueda(n) entender todos los requisitos del estudio y cumplir con ellos.7Spanish68614620065fe7e-e51d-414e-9a49-6e9f8e148ce6A szülő(k) vagy a törvényes gondviselő(k) képesek megérteni az összes vizsgálati követelményt, és együttműködni azok szerint.13Hungarian68614610065fe7e-e51d-414e-9a49-6e9f8e148ce6Има родител(и) или законен(ни) настойник(ци), който(които) може(могат) да разбере(ат) и спазва(т) изискванията на изпитването.1Bulgarian10052601Treatment with any of the following investigational therapies according to the time frames specified: ● At any time: - Utrophin upregulating agents (except for Ezutromid) - CRISPR/Cas9, or any other form of gene editing - Gene therapy - Cell-based therapy (e.g., stem cell transplantation) - Any form of nucleic acid antisense therapy, except PRO045 (BMN 045) or PRO053 (BMN 053) (see below) - Exon Skipping Therapies - Drisapersen within 36 weeks prior to Week 1 - PRO045 (BMN 045) Within 24 weeks prior to Week 1 - PRO053 (BMN 053) Within 24 weeks prior to Week 1 - PRO051 (BMN 051) Within 24 weeks prior to Week 1 ●All Anti-Myostatin Therapies within 24 Weeks prior to Week 1 including but not limited to: - Domagrozumab (PF-06252616) - RG-6206 (formally RO-7239361 and BMS-986089) ● Small Molecule Therapies: - Ezutromid (SMT C1100) within 1 week prior to Week 1 ● Within 24 weeks prior to Week 1: - Anti-fibrotic or anti-inflammatory agents including but not limited to: rimeporide, epigallocatechin-gallate, TAS-205, edasalonexent (CAT1004), FG-3019, and halofuginone (HT-100) - Mast cell activation inhibitor (e.g., CRD007 [pemirolast sodium]) - Idebenone (Raxone®) ● Within 12 weeks prior to Week 1: - Nitric oxide -active agents including, but not limited to, metformin and citrulline, isosorbide dinitrate, tadalafil, sildenafil, pentoxifylline if taken as part of a DMD clinical trial and not for a medical indication. If taken for a medical indication, must be on a stable dose for at least 12 weeks prior to Week 1. - Vamorolone (VBP-15) ● For any experimental treatment not otherwise specified in Exclusion Criterion 1, consult the medical monitor.6861387e2ec2eb5-3199-4817-8099-7c158ca0396cleczenie z wykorzystaniem którejkolwiek z wymienionych terapii eksperymentalnych w konkretnym okresie czasu: ● kiedykolwiek: - leki zwiększające ekspresję utrofiny (z wyjątkiem ezutromidu); - terapia CRISPR/Cas9 lub inne terapie wykorzystujące techniki edycji genów; - terapia genowa; - terapia komórkowa (np. przeszczep komórek macierzystych); - jakakolwiek terapia antysensownymi oligonukleotydami, poza PRO045 (BMN 045) lub PRO053 (BMN 053) (patrz poniżej); - terapie pomijania egzonu; - drisapersen w okresie 36 tygodni przed 1. tygodniem; - PRO045 (BMN 045) w okresie 24 tygodni przed 1. tygodniem; - PRO053 (BMN 053) w okresie 24 tygodni przed 1. tygodniem; - PRO051 (BMN 051) w okresie 24 tygodni przed 1. tygodniem; ● wszystkie rodzaje leczenia hamujące miostatynę w okresie 24 tygodni przed 1. tygodniem, w tym między innymi: - domagrozumab (PF-06252616); - RG-6206 (wcześniej RO-7239361 i BMS-986089); ● terapie z zastosowaniem leków drobnocząsteczkowych: - ezutromid (SMT C1100) w okresie 1 tygodnia przed 1. tygodniem; ● w okresie 24 tygodni przed 1. tygodniem: - leki o działaniu przeciwzwłóknieniowym lub przeciwzapalnym, włączając w to, ale nie ograniczając do: rimeporid, galusan epigallokatechiny, TAS-205, edasaloneksent (CAT 1004), FG-3019 i halofuginon (HT-100); - inhibitory aktywacji komórek tucznych (np. CRD007 [sól sodowa pemirolastu]); - idebenon (Raxone®); ● w okresie 12 tygodni przez 1. tygodniem: - leki działające poprzez tlenek azotu, włączając w to (ale nie ograniczając do) metforminę i cytrulinę, diazotan izosorbidu, tadalafil, sildenafil, pentoksyfilinę, jeżeli stosowane są w ramach badania klinicznego dotyczącego leczenia dystrofii mięśniowej Duchenne’a, a nie we wskazaniu klinicznym. Jeżeli stosowane są we wskazaniu klinicznym, muszą być przyjmowane w stałej dawce przez przynajmniej 12 tygodni przed 1. tygodniem; - wamorolon (VBP-15); ● w przypadku wszelkich terapii eksperymentalnych niewymienionych w kryterium wyłączenia 1 należy skonsultować się z monitorem badań klinicznych.19Polish6861384e2ec2eb5-3199-4817-8099-7c158ca0396cHaber recibido tratamiento con uno de los fármacos en estudio que aparecen a continuación y dentro de los periodos especificados: ● En cualquier momento: - Fármacos de aumento de la utrofina (excepto Ezutromid). - CRISPR/Cas9, y cualquier otra forma de modificación del genoma. - Genoterapia - Citoterapia (p. ej., trasplante de hemocitoblastos) - Cualquier forma de tratamiento antisentido con ácido nucleico, excepto PRO045 (BMN 045) o PRO053 (BMN 053) (véase a continuación). - Tratamientos de omisión de exones. - Drisapersen dentro de las 36 semanas anteriores a la semana 1. - PRO045 (BMN 045) dentro de las 24 semanas anteriores a la semana 1. - PRO053 (BMN 053) dentro de las 24 semanas anteriores a la semana 1. - PRO051 (BMN 051) dentro de las 24 semanas anteriores a la semana 1. ● Cualquier tratamiento frente a la miostatina dentro de las 24 semanas anteriores a la semana 1. Entre ellos, se encuentran: - Domagrozumab (PF-06252616). - RG-6206 (formalmente RO-7239361 y BMS-986089). ● Tratamientos tradicionales: - Ezutromid (SMT C1100) en la semanas anterior a la semana 1. ● Dentro de las 24 semanas anteriores a la semana 1: - Fármacos antifibróticos o antiinflamatorios, entre los que se encuentran: rimeporida, galato de epigalocatequina, TAS-205, edasalonexent (CAT1004), FG-3019 y halofuginona (HT-100). - Inhibidor de la activación mastocitaria (p. ej., CRD007 [sodio de pemirolast]). - Idebenona (Raxone®). ● Dentro de las 12 semanas anteriores a la semana 1: - Fármacos que interactúan con el óxido nítrico, entre los que se encuentran la metformina y la citrulina, el dinitrato de isosorbida, el tadalafilo, el sidenafilo o la pentoxifilina si se toma en el marco de un ensayo clínico relacionado con la DMD y no por indicación médica. Si se toma por indicación médica, debe haberse recibido en una dosis estable durante al menos las 12 semanas anteriores a la semana 1. - Vamorolona (VBP-15). ● Para informarse sobre cualquier otro tratamiento en estudio que no se haya especificado en el criterio de exclusión 1, consulte al monitor médico.7Spanish6861385e2ec2eb5-3199-4817-8099-7c158ca0396cKezelés bármely alábbi vizsgálati kezeléssel a megadott időintervallumokon belül: ● Bármikor: - Utrofin-túlszabályozó készítmények (az ezutromid kivételével) - CRISPR/Cas9, vagy a géneditálás bármely más formája - Génterápia - Sejtalapú kezelés (pl. őssejt-transzplantáció) - Nukleinsav antisense kezelés bármely formája, a PRO045 (BMN 045) vagy PRO053 (BMN 053) kivételével (lásd alább) - Exonátugrásos kezelések - Drisapersen az 1. hetet megelőző 36 hétben - PRO045 (BMN 045) az 1. hetet megelőző 24 hétben - PRO053 (BMN 053) az 1. hetet megelőző 24 hétben - PRO051 (BMN 051) az 1. hetet megelőző 24 hétben • Az összes antimyostatin kezelés az 1. hetet megelőző 24 hétben, ideértve többek között az alábbiakat: - Domagrozumab (PF-06252616) - RG-6206 (hivatalosan RO-7239361 és BMS-986089) • Kismolekulájú szerek: - Ezutromid (SMT C1100) az 1. hetet megelőző 1 hétben • Az 1. hetet megelőző 24 hétben: - Antifibrotikus vagy antiinflammatórikus készítmények, beleértve többek között az alábbiakat: rimeporid, epigallokatechin-gallát, TAS-205, edaszalonexent (CAT-1004), FG-3019 és halofuginon (HT-100) - Hízósejt-aktiváció inhibitor (pl. CRD007 [pemirolaszt-nátrium]) - Idebenon (Raxone®) • Az 1. hetet megelőző 12 hétben: - Nitrogén-oxid-aktív készítmények, beleértve többek között a következőket: metformin és citrullin, isoszorbid-dinitrát, tadalafil, szildenafil, pentoxifillin, ha DMD klinikai vizsgálat keretében és nem orvosi javallatra szedte. Ha orvosi javallatra szedte, akkor az 1. hetet megelőző legalább 12 hétben a dózisnak változatlannak kellett lennie. - Vamorolon (VBP-15) • Minden vizsgálati kezelés esetén, amely az 1. kizárási kritériumban nem került felsorolásra, konzultáljon az orvosmonitorral.13Hungarian6861386e2ec2eb5-3199-4817-8099-7c158ca0396cЛечение с която и да е от следните експериментални терапии в съответствие с посочените времеви диапазони: ● В който и да е момент: - Препарати за регулиране на утрофин (с изключение на езутромид) - CRISPR/Cas9 или друга форма на редактиране на генома - Генна терапия - Клетъчна терапия (напр. трансплантация на стволови клетки) - Всяка форма на антисенс терапия с нуклеинова киселина, с изключение на PRO045 (BMN 045) или PRO053 (BMN 053) (вижте по-долу) - Терапии с прескачане на екзон - Дрисаперсен в рамките на 36 седмици преди седмица 1 - PRO045 (BMN 045) в рамките на 24 седмици преди седмица 1 - PRO053 (BMN 053) в рамките на 24 седмици преди седмица 1 - PRO051 (BMN 051) в рамките на 24 седмици преди седмица 1 - Всякакви терапии с анти-миостатин в рамките на 24 седмици преди седмица 1, включително, но не само: - Домагрозумаб (PF-06252616) - RG-6206 (наричано преди RO-7239361 и BMS-986089) ● Терапии с малки молекули: - Езутромид (SMT C1100) в рамките на 1 седмица преди седмица 1 ● В рамките на 24 седмици преди седмица 1: - Антифибротични или противовъзпалителни препарати, включително, но не само: римепорид, епигалокатехин-галат, TAS-205, едазалонексент (CAT1004), FG-3019 и халофугинон (HT-100) - Инхибитор на активиране на мастоцитите (напр. CRD007 [пемироласт натрий]) - Идебенон (Raxone®) ● В рамките на 12 седмици преди седмица 1: - Препарати с активен азотен оксид, включително, но не само, метформин и цитрулин, изосорбид динитрат, тадалафил, силденафил, пентоксифилин, приемани като част от клинично изпитване за МДД, а не за медицинско показание. Ако се приемат за медицинско показание, трябва да са в стабилна доза в продължение на най-малко 12 седмици преди седмица 1. - Ваморолон (VBP-15) ● За всяко експериментално лечение, което не е посочено в Критерий за изключване 1, се консултирайте с медицинския монитор.1Bulgarian10052612Treatment with any of the following non-investigational therapies according to the time frames specified: ● Within 12 weeks prior to Week 1: - Any pharmacologic treatment (other than corticosteroids) that may have an effect on muscle strength or function. Growth hormone for short stature and testosterone for delayed puberty are permitted if a physician has documented the diagnosis and medical necessity of treatment, and the patient started dosing at least 24 weeks prior to Week 1. ● Within 12 weeks prior to Week 1 or anticipated need during the study: - Statins - Aminoglycoside antibiotics6861390128fa312-9f72-46c9-b4fa-299ee1e06cb3Лечение с която и да е от следните неекспериментални терапии в съответствие с посочените времеви диапазони: ● В рамките на 12 седмици преди седмица 1: - Всяко фармакологично лечение (различно от кортикостероиди), което може да има ефект върху мускулната сила или функция. Хормон на растежа за нисък ръст и тестостерон за забавен пубертет са разрешени, ако диагнозата и медицинската необходимост от лечението са документирани от лекар и пациентът е започнал приема на дозата поне 24 седмици преди седмица 1. ● В рамките на 12 седмици преди седмица 1 или ако се очаква, че ще е необходимо по време на изпитването: - Статини - Аминогликозидни антибиотици1Bulgarian6861391128fa312-9f72-46c9-b4fa-299ee1e06cb3Kezelés bármely alábbi nem vizsgálati kezeléssel a megadott időintervallumokon belül: • Az 1. hetet megelőző 12 hétben: - Bármilyen farmakológiai kezelés (a kortikoszteroidok kivételével), amely hatással lehet az izomerőre vagy izomműködésre. Alacsony termetre adott növekedési hormon és megkésett pubertásra adott tesztoszteron megengedett, ha az orvos dokumentálta a diagnózist és a kezelés orvosi szükségességét, és a beteg a kezelést legalább 24 héttel az 1. hét előtt kezdte. • Ha a beteg az 1. hetet megelőző 12 hétben szedte vagy a vizsgálat alatt várhatóan szednie kell a következőket: - Sztatinok - Aminoglikozid antibiotikumok13Hungarian6861388128fa312-9f72-46c9-b4fa-299ee1e06cb3leczenie z wykorzystaniem którejkolwiek z wymienionych terapii nieeksperymentalnych w konkretnym okresie czasu: ● w okresie 12 tygodni przez 1. tygodniem: - jakiekolwiek leczenie farmakologiczne (inne niż z wykorzystaniem kortykosteroidów), które może wpływać na siłę lub czynność mięśni. Hormon wzrostu przy terapii związanej z niskim wzrostem i testosteron stosowany przy opóźnionym dojrzewaniu są dozwolone, jeżeli lekarz posiada udokumentowane rozpoznanie i istnieje medyczna konieczność leczenia, a pacjent rozpoczął przyjmowanie leku przynajmniej 24 tygodnie przed 1. tygodniem ● w okresie 12 tygodni przed 1. tygodniem lub jeżeli przewidywane jest stosowanie podczas badania: - statyn; - antybiotyków aminoglikozydowych.19Polish6861389128fa312-9f72-46c9-b4fa-299ee1e06cb3Haber recibido tratamiento con uno de los fármacos no investigados que aparecen a continuación y dentro de los periodos especificados: ● Dentro de las 12 semanas anteriores a la semana 1: - Cualquier tratamiento farmacológico (diferente de los corticoesteroides) que pueda tener un efecto en la función o la fuerza musculares. Se permiten los tratamientos con hormona del crecimiento para la estatura y testosterona para la pubertad tardía si un médico ha documentado el diagnóstico y la necesidad médica del tratamiento, siempre que el paciente haya empezado el tratamiento al menos 24 semanas antes de la semana 1. ● Dentro de las 12 semanas anteriores a la semana 1 o si se prevé la necesidad de uso durante el estudio: - Estatinas. - Aminoglucósidos.7Spanish10052623Major surgery within 3 months prior to Week 1 or planned surgery for any time during this study, except for protocol-specified surgery, as applicable.68613925e208005-1cc0-4c09-862f-4739d4df011aNagyműtét az 1. hetet megelőző 3 hónapban vagy tervezett műtét a vizsgálat alatt bármikor, a vizsgálati tervben előírt műtét kivételével, ha van.13Hungarian68613935e208005-1cc0-4c09-862f-4739d4df011aduże zabiegi chirurgiczne w okresie 3 miesięcy przed 1. tygodniem lub planowane zabiegi chirurgiczne w dowolnym momencie badania, poza zabiegami przewidzianymi w protokole badania, jeżeli ma to zastosowanie.19Polish68613955e208005-1cc0-4c09-862f-4739d4df011aCirugía mayor dentro de los 3 meses anteriores a la semana 1 o prevista en cualquier momento durante el estudio, excepto en el caso de intervenciones quirúrgicas especificadas en el protocolo, según proceda.7Spanish68613945e208005-1cc0-4c09-862f-4739d4df011aЗначима операция в рамките на 3 месеца преди седмица 1 или планирана операция в който и да е момент по време на изпитването, с изключение на посочена в протокола операция, ако е приложимо1Bulgarian10052634Presence of any other significant genetic disease other than DMD (e.g., dwarfism).6861399235e2d49-9a3e-4022-a3df-d71863a5c6efA DMD-n kívül egyéb jelentős genetikai betegség fennállása (pl. törpeség).13Hungarian6861397235e2d49-9a3e-4022-a3df-d71863a5c6efPresencia de cualquier otra enfermedad genética significativa diferente de la DMD (p. ej., enanismo).7Spanish6861396235e2d49-9a3e-4022-a3df-d71863a5c6efobecność jakichkolwiek innych istotnych chorób genetycznych, innych niż dystrofia mięśniowa Duchenne’a (np. niedobór wzrostu).19Polish6861398235e2d49-9a3e-4022-a3df-d71863a5c6efНаличие на друго значимо генетично заболяване, различно от МДД (напр. нанизъм).1Bulgarian10052645Presence of other clinically significant illness including significant cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or malignancy.6861403a4882533-da28-42ed-9a8b-773ddcd828c3Наличие на друго клинично значимо заболяване, включително значимо сърдечно, белодробно, чернодробно, бъбречно, хематологично, имунологично заболяване или поведенческо разстройство, или злокачествено заболяване.1Bulgarian6861400a4882533-da28-42ed-9a8b-773ddcd828c3Presencia de otras enfermedades clínicamente significativas, entre las que se pueden incluir enfermedades cardíacas, respiratorias, hepáticas, renales, hematológicas o inmunitarias, trastornos de la conducta o neoplasias malignas.7Spanish6861401a4882533-da28-42ed-9a8b-773ddcd828c3Egyéb klinikailag jelentős betegség fennállása, beleértve a jelentős kardiális, pulmonális, hepatikus, renális, hematológiai, immunológiai vagy viselkedési betegséget és a malignitást.13Hungarian6861402a4882533-da28-42ed-9a8b-773ddcd828c3obecność innych istotnych klinicznie chorób, w tym choroby układu krążenia, płuc, nerek, krwi, choroby immunologiczne, zmiany zachowania lub nowotwory.19Polish10052656LVEF <50% on the Screening echocardiogram (ECHO) or QTcF ≥450 msec based on the Screening and Baseline electrocardiogram (ECG).68614071703d685-e1cd-4fa7-9d1a-00e9cc45920dLVEF <50% na podstawie przesiewowego badania echa serca lub odstęp QTcF ≥450 ms w badaniu elektrokardiogramie (EKG) przesiewowym i początkowym.19Polish68614041703d685-e1cd-4fa7-9d1a-00e9cc45920dLVEF <50% a szűrő echokardiográfián (ECHO) vagy QTcF ≥450 ms a szűrő és kiindulási elektrokardiogramon (EKG).13Hungarian68614061703d685-e1cd-4fa7-9d1a-00e9cc45920dFEVI <50 % en el ecocardiograma de la selección o QTcF ≥450 ms según los electrocardiogramas (ECG) de la selección y basal.7Spanish68614051703d685-e1cd-4fa7-9d1a-00e9cc45920dФИЛК <50% от ехокардиограмата (ECHO) от скрининга или QTcF ≥450 msec въз основа на електрокардиограмата (ЕКГ) от скрининга и изходното ниво.1Bulgarian10052667Dorsiflexion range of motion will be measured bilaterally and recorded as degrees from neutral (see figure). The subject will be excluded if the average loss of dorsiflexion of both extremities is > -10 degrees. For example, if the patient has -8 degrees on one side and -12 degrees on the other side, then he would still qualify because the average of the 2 sides is -10 degrees.6861408c476c258-ca25-4c92-9a8d-e17408487236Диапазонът на движение на дорсифлексия ще бъде измерен двустранно и записан като градуси от неутралната позиция (вж. фигурата). Участникът ще бъде изключен, ако средната загуба на дорсифлексия на двата крайника е > -10 градуса. Например, ако участникът има -8 градуса от едната страна и -12 градуса от другата страна, той ще отговаря на критериите за участие, тъй като средната стойност за двете страни е -10 градуса.1Bulgarian6861411c476c258-ca25-4c92-9a8d-e17408487236La amplitud del movimiento de dorsiflexión se determinará de manera bilateral y se registrará como grados desde la posición neutra (véase la figura). El sujeto se excluirá si la pérdida promedio de dorsiflexión de ambas extremidades es >−10 grados. Por ejemplo, si el sujeto presenta −8 grados en un lado y −12 en otro, sería apto para la participación en el estudio, ya que el promedio de ambos lados es −10 grados.7Spanish6861409c476c258-ca25-4c92-9a8d-e17408487236zgięcie grzbietowe stopy będzie mierzone dwustronnie i zapisywane w postaci stopni od pozycji neutralnej (patrz rycina). Pacjent zostanie wyłączony z badania, jeżeli średnia utrata zgięcia grzbietowego w obu kończynach wyniesie >10 stopni. Przykładowo, jeżeli pacjent ma -8 stopni na jednej stronie, a na drugiej -12 stopni, będzie kwalifikował się do badania ponieważ średnia z obu stron wynosi -10 stopni.19Polish6861410c476c258-ca25-4c92-9a8d-e17408487236A dorsalflexio mozgásterjedelmét mindkét oldalon mérjük, és dokumentáljuk a semleges helyzettől mért szöget (lásd az ábrát). A beteg kizárásra kerül, ha a dorsalflexio átlagos beszűkülése mindkét végtagon > -10 fok. Például, ha a betegnél az egyik oldal -8 fok, a másik oldal -12 fok, akkor még mindig alkalmas lehet, mert a két oldal átlaga -10 fok.13Hungarian10052678Prior or ongoing medical condition that could, in the Investigator's opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results. Additionally, patients who seem unable / unwilling to comply with the study procedures, in the Investigator's opinion, are to be excluded.6861415db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5Предходно или настоящо медицинско заболяване, което по преценка на изследователя влияе неблагоприятно на безопасността на пациента, прави малко вероятно изпълнението на курса на лечението или нарушава оценката на резултатите от изпитването. В допълнение, пациентите, които по преценка на изследователя изглеждат неспособни/нежелаещи да спазват процедурите в изпитването, ще бъдат изключени.1Bulgarian6861412db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5Enfermedad previa o actual que, en opinión del investigador, podría afectar negativamente a la seguridad del paciente, hacer improbable que se complete el tratamiento o dificultar la evaluación de los resultados obtenidos durante el estudio. Además, se podrá excluir a los pacientes que, en opinión del investigador, parezcan ser incapaces o no estar dispuestos a cumplir con los procedimientos del estudio.7Spanish6861413db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5Korábbi vagy fennálló egészségügyi állapot, amely a vizsgálóorvos véleménye szerint hátrányosan befolyásolná a beteg biztonságát, valószínűtlenné teszi a kezelés befejezését, vagy akadályozná a vizsgálati eredmények értékelését. Ezen kívül kizárandók azok a betegek, akik a vizsgálóorvos véleménye szerint nem képesek / készek együttműködni a vizsgálati eljárások során.13Hungarian6861414db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5wcześniejsze lub obecne schorzenia, które w opinii badacza mogą negatywnie wpływać na bezpieczeństwo pacjenta, sprawiając, że ukończenie leczenia jest mało prawdopodobne, lub zaburzając ocenę wyników badania. Ponadto wykluczeni zostaną pacjenci, którzy w ocenie badacza mogą być niezdolni lub niechętni do przestrzegania warunków badania.19Polish10052689Known hypersensitivity to the study drug or to any of its components68614163d5dfbd1-8924-43af-a1e9-93594f108b0eHipersensibilidad conocida al fármaco del estudio o a alguno de sus componentes.7Spanish68614183d5dfbd1-8924-43af-a1e9-93594f108b0eA vizsgálati készítmény hatóanyagával vagy bármely összetevőjével szemben fennálló túlérzékenység13Hungarian68614193d5dfbd1-8924-43af-a1e9-93594f108b0eИзвестна свръхчувствителност към лекарството по изпитването или към някой от компонентите му1Bulgarian68614173d5dfbd1-8924-43af-a1e9-93594f108b0enadwrażliwość na badany lek lub którykolwiek z jego składników.19Polish6822881Double-blind period: Change from Baseline at Week 96 in 4-step ascend velocity (step/second)True686137156d221c8-2997-41d9-9b50-7a2aa5066d45Двойно сляп период: Промяна от изходното ниво през седмица 96 в скоростта на изкачване на 4 стъпала (стъпка/секунда)1Bulgarian686136856d221c8-2997-41d9-9b50-7a2aa5066d45okres leczenia w fazie podwójnie zaślepionej: stanowi zmiana w 96. tygodniu w stosunku do punktu początkowego w czasie na pokonanie 4 schodów (stopień/sekundy).19Polish686136956d221c8-2997-41d9-9b50-7a2aa5066d45Periodo doble ciego: Cambio desde el momento basal hasta la semana 96 en la velocidad para subir 4 escalones (escalón/segundo)7Spanish686137056d221c8-2997-41d9-9b50-7a2aa5066d45Kettős vak szakasz: változás a kiindulástól a 96. hétig a 4 lépéses emelkedési sebességben (lépés/másodperc)13Hungarian6822891• Change from Baseline at Week 96 in 6MWT • Change from Baseline at Week 96 in rise from floor velocity (rise/second) • Change from Baseline at Week 144 in 4-step ascend velocity (step/second)False68613726ecbb7b6-5478-44ce-af09-d5f1a0d0e252zmiana w 96. tygodniu w stosunku do punktu początkowego w ocenie 6MWT; zmiana w 96. tygodniu w stosunku do punktu początkowego w ocenie wstawania z podłogi (wstawanie/sekundy); zmiana w 144. tygodniu w stosunku do punktu początkowego w ocenie czasu na pokonanie 4 schodów (stopień/sekundy);19Polish68613736ecbb7b6-5478-44ce-af09-d5f1a0d0e252• Variación desde el momento basal hasta la semana 96 en la prueba de marcha de seis minutos • Variación desde el momento basal hasta la semana 96 en la velocidad para levantarse del suelo (subida/segundo) • Variación desde el momento basal hasta la semana 144 en la velocidad para subir 4 escalones (escalón/segundo)7Spanish68613756ecbb7b6-5478-44ce-af09-d5f1a0d0e252• Промяна от изходното ниво на Седмица 96 в 6-мунитния тест за ходене • Промяна от изходното ниво на Седмица 96 в скоростта на изправяне от пода (ставане/секунда) • Промяна от изходното ниво на Седмица 144 в скоростта на изкачване на 4 стъпала (стъпка/секунда)1Bulgarian68613746ecbb7b6-5478-44ce-af09-d5f1a0d0e252- Változás a kiindulási értékhez képest a 96. héten a 6MWT-ben - Változás a 96. héten a padlóról való felemelkedés sebességében a kiindulási értékhez képest - Változás a kiindulási értékhez képest a 144. héten a 4 lépéses emelkedési sebességben13Hungarian6822902• Change from Baseline at Week 96 in 10MWR velocity (meter/second) • Change from Baseline at Weeks 48 or 96 in the quantity of dystrophin protein expression as measured by Western blot of biopsied muscle tissue.False68613776971a9ea-0519-47c5-a527-034ab98b399d• Variación con respecto al momento basal en la semana 96 en la velocidad de la prueba de caminar/correr 10 metros (metros/segundo) • Variación con respecto al momento basal en las semanas 48 o 96 en la cantidad de expresión de la proteína distrofina medida por inmunoelectrotransferencia de tejido muscular biopsiado.7Spanish68613796971a9ea-0519-47c5-a527-034ab98b399dzmiana w 96. tygodniu w stosunku do punktu początkowego w ocenie 10MWR (metr/sekundy); zmiana w 48. lub 96. tygodniu w stosunku do punktu początkowego w ilości ekspresji dystrofiny w biopsyjnej tkance mięśnia w oparciu o pomiar metodą Western blot19Polish68613786971a9ea-0519-47c5-a527-034ab98b399d• Промяна от изходното ниво на Седмица 96 в скоростта при теста за ходене/бягане на 10 метра (метър/секунда) • Промяна ор изходното ниво на Седмици 48 или 96 в количеството на експресия на белтъка дисторфин, измерено чрез Western blot в биопсична мускулна тъкан.1Bulgarian68613766971a9ea-0519-47c5-a527-034ab98b399d- Változás a kiindulási értékhez képest a 96. héten a 10MWR sebességben - Változás a kiindulási értékhez képest a 48. vagy 96. héten a dystrophin fehérje expressziójának mennyiségében, a biopsziával vizsgált izomszövetből Western blottal mérve.13Hungarian6822913• Change from Baseline at Weeks 48 or 96 in the intensity of dystrophin expression in biopsied muscle tissue, as measured by IHC. •Change from Baseline at Week 96 in: NSAA total scoreFalse68613837af296b7-07f9-439f-b2ea-ba41452548cb• Промяна от изходото ниво на Седмици 48 и 96 ст в интензитета на експресия на белтъка дисторфин в биопсична мускулна тъкан, измерено чрез IHC. • Промяна от изходното ниво през седмица 96 в общата оценка по NSAA1Bulgarian68613827af296b7-07f9-439f-b2ea-ba41452548cb- Változás a kiindulási értékhez képest a 96. héten a 10MWR sebességben - Változás a kiindulási értékhez képest a 48. vagy 96. héten a dystrophin fehérje expressziójának mennyiségében, a biopsziával vizsgált izomszövetből Western blottal mérve.13Hungarian68613807af296b7-07f9-439f-b2ea-ba41452548cb• Variación con respecto al momento basal en las semanas 48 o 96 en la intensidad de la expresión de distrofina en tejido muscular biopsiado, medido mediante IHQ • Variación con respecto al momento basal en la semana 96 en la puntuación total de la NSAA7Spanish68613817af296b7-07f9-439f-b2ea-ba41452548cbzmiana w 48. lub 96. tygodniu w stosunku do punktu początkowego w nasileniu ekspresji dystrofiny w biopsyjnej tkance mięśnia w oparciu o pomiar metodą IHC; zmiana w 96. tygodniu w stosunku do punktu początkowego w łącznym wyniku oceny NSAA19Polish2025-10-312025-09-172017-05-3164277Sarepta Therapeutics, Inc.24336822243369266224337025522PatientsFalseTrueTrue2225412015-002069-52A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy, Estudio doble ciego, controlado con placebo y multicéntrico con una extensión abierta para evaluar la eficacia y la seguridad de SRP-4045 y SRP-4053 en pacientes con distrofia muscular de Duchenne, Dvojitě zaslepená, placebem kontrolovaná, multicentrická studie s nezaslepeným prodloužením k vyhodnocení účinnosti a bezpečnosti látek SRP-4045 a SRP-4053 u pacientů s Duchennovou muskulární dystrofií, Dvojitě zaslepená, placebem kontrolovaná, multicentrická studie s nezaslepeným prodloužením k vyhodnocení účinnosti a bezpečnosti látek SRP-4045 a SRP-4053 u pacientů s Duchennovou muskulární dystrofií, Dvojitě zaslepená, placebem kontrolovaná, multicentrická studie s nezaslepeným prodloužením k vyhodnocení účinnosti a bezpečnosti látek SRP-4045 a SRP-4053 u pacientů s Duchennovou muskulární dystrofií, Dvojitě zaslepená, placebem kontrolovaná, multicentrická studie s nezaslepeným prodloužením k vyhodnocení účinnosti a bezpečnosti látek SRP-4045 a SRP-4053 u pacientů s Duchennovou muskulární dystrofií, Dvojitě zaslepená, placebem kontrolovaná, multicentrická studie s nezaslepeným prodloužením k vyhodnocení účinnosti a bezpečnosti látek SRP-4045 a SRP-4053 u pacientů s Duchennovou muskulární dystrofií, Dvojitě zaslepená, placebem kontrolovaná, multicentrická studie s nezaslepeným prodloužením k vyhodnocení účinnosti a bezpečnosti látek SRP-4045 a SRP-4053 u pacientů s Duchennovou muskulární dystrofií, Dvojitě zaslepená, placebem kontrolovaná, multicentrická studie s nezaslepeným prodloužením k vyhodnocení účinnosti a bezpečnosti látek SRP-4045 a SRP-4053 u pacientů s Duchennovou muskulární dystrofií, Dvojitě zaslepená, placebem kontrolovaná, multicentrická studie s nezaslepeným prodloužením k vyhodnocení účinnosti a bezpečnosti látek SRP-4045 a SRP-4053 u pacientů s Duchennovou muskulární dystrofií, Studio in doppio cieco, controllato con placebo, multicentrico, con un'estensione in aperto per valutare l'efficacia e la sicurezza di SRP-4045 e SRP-4053 in pazienti affetti da distrofia muscolare di DuchenneOPTIONALOPTIONALFalseacceptable2025-05-022025-05-025Diseases [C] - Musculoskeletal Diseases [C05]91632Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping.True97917True292356PublicPatient RecruitmentSareptAlly@sarepta.com+18887273782488781Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active292357ScientificPatient RecruitmentSareptAlly@sarepta.com+18887273782488781Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active427505470125502493Pharmaceutical company10PPD Development LPTrueTrueORG-100011560Active5040503151 South 17th Street3151 South 17th StreetWilmington28412-6461840United States+33182392401EUCTRInquiry.sm@ppd.comTrueORG-10001156066985116698521266985336698545+33182392401EUCTRInquiry.sm@ppd.com427506470123502491Pharmaceutical company10PPD Global Central LabsTrueTrueORG-100046496Active504048Kleine Kloosterstraat 19Kleine Kloosterstraat 19Zaventem19322002Belgium+32489561424Lorraine.mcnamara@ppd.comTrueORG-1000464966698554+32489561424Lorraine.mcnamara@ppd.com488781Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active456477488781Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active489251215 1st Street215 1st StreetCambridge02142-1213840United StatesTrueORG-100006645TrueCommercial2The present trial is a phase III clinical trial and therefore enters into category 2 regarding disclosure rules8863355Diseases [C] - Musculoskeletal Diseases [C05]277618GOLODIRSEN (SRP-4053)1Test366717110771830CONCENTRATE FOR SOLUTION FOR INFUSIONPRD19599021GOLODIRSEN (SRP-4053)CONCENTRATE FOR SOLUTION FOR INFUSIONSRP-4053GOLODIRSENSUB188593GOLODIRSENSAREPTA THERAPEUTICS INC10771830337900SAREPTA THERAPEUTICS INCNucleic AcidNucleic AcidGOLODIRSENSUB188593False1Falsemg/kg milligram(s)/kilogram30mg/kg milligram(s)/kilogram43201442OligomerPRD1959902SRP-405311INTRAVENOUS USEFalseGOLODIRSEN (SRP-4053)golodirsenCONCENTRATE FOR SOLUTION FOR INFUSION277617CASIMERSEN (SRP-4045)1Test366716110281534SOLUTION FOR INFUSIONPRD94568141CASIMERSEN (SRP-4045)SOLUTION FOR INFUSIONSRP-4045CASIMERSENSUB188601CASIMERSENSAREPTA THERAPEUTICS INC10281534288376SAREPTA THERAPEUTICS INCNucleic AcidNucleic AcidCASIMERSENSUB188601False1Falsemg/kg milligram(s)/kilogram30mg/kg milligram(s)/kilogram43201442OligomerPRD9456814SRP-404511INTRAVENOUS USEFalseCASIMERSEN (SRP-4045)casimersenSOLUTION FOR INFUSION2776160,9% sodium chloride3Placebo3667151N/AN/AN/A0,9% sodium chlorideN/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalse0,9% sodium chloriden/aN/AFalse1161083155231552155922017Ended2024-07-042024-07-04FalseunwillingIreland2024-07-29Ended306782021-01-132024-08-13306792021-01-132024-08-13476582021-01-132025-01-072025-01-09391152021-02-182022-02-142024-08-13391162021-02-182022-02-142024-08-13391132021-02-182024-08-13391142021-02-182024-08-13True658762021-01-132025-01-072024-08-13FalseFalseFalseTrue2021-02-182022-02-14False4585831552Authorised2024-07-29T18:24:54.1894055131552Under evaluation2024-06-25T16:30:50.0247344731552Ended2025-01-09T17:04:24.8741Irelandacceptable2024-07-29authorized2024-07-292024-07-293763970491026523458Hospital/Clinic/Other health care facility8Children's Health IrelandFalseTrueORG-100022831Active526865Temple StreetTemple StreetDublin 1D01 YC672017Ireland+35318784722Declan.orourke@cuh.ieTrueORG-100022831852920DeclanO’Rourke+35318784722Declan.orourke@cuh.ie1Department of NeurologyAuthorised1818543154731547155922002Ended2024-07-042024-07-04FalseunwillingBelgium2024-07-26Ended306572017-05-152024-08-13306582017-05-152024-08-13650282017-05-152025-09-232025-09-24390662017-05-312024-08-13390672017-05-312024-08-13390712017-05-312022-10-242024-08-13390722017-05-312022-10-242024-08-13True991132017-05-152025-09-232024-08-13FalseFalseFalseTrue2017-05-312022-10-24False4542331547Authorised2024-07-26T08:59:48.8784054631547Under evaluation2024-06-25T16:30:49.8919413931547Ended2025-09-24T14:31:07.3381Belgiumacceptable2025-04-14authorized_conditions2024-07-262024-07-26101173232484344516756Hospital/Clinic/Other health care facility8UZ LeuvenFalseTrueORG-100006001Active519552Herestraat 49Herestraat 49Leuven30002002Belgium+3216343827liesbeth.dewaele@uzleuven.beTrueORG-1000060011311621LiesbethDe Waele+3216343827liesbeth.dewaele@uzleuven.be2Department Child Neurology1173231694799732968Hospital/Clinic/Other health care facility8Universitair Ziekenhuis GentFalseTrueORG-100021542Active759126Corneel Heymanslaan 10Corneel Heymanslaan 10Gent90002002Belgium+320473966619Nicolas.Deconinck@hunderf.beTrueORG-1000215421311620NicolasDeconinck+320473966619Nicolas.Deconinck@hunderf.be2Paediatric NeurologyAuthorised1943593154831548155922005Ended2024-07-042024-07-04FalseunwillingBulgaria2024-07-29Ended306662019-04-302024-08-13306642019-03-302024-08-13306652019-03-302024-08-13576722019-04-302025-05-082025-05-29573742019-04-302025-05-142025-05-26391072019-05-082022-03-222024-08-13391082019-05-082022-03-222024-08-13391052019-05-082024-08-13391062019-05-082024-08-13True847232019-04-302025-05-082024-08-13FalseFalseFalseTrue2019-05-082022-03-22False4567531548Authorised2024-07-29T09:52:27.6294054731548Under evaluation2024-06-25T16:30:49.9218396731548Authorised2025-05-29T16:02:17.6348362131548Ended2025-05-26T11:09:41.5078396831548Ended2025-05-29T16:02:37.6631Bulgariaacceptable2025-04-22authorized2024-07-292024-07-2951273556490962523394Hospital/Clinic/Other health care facility8Alexandrovska University HospitalFalseTrueORG-100029407Active526800Georgy Sofiiski Str 1Georgy Sofiiski Str 1Sofia14312005Bulgaria+35929230670itournev@emhpf.orgTrueORG-1000294071420415IvayloTarnev+35929230670itournev@emhpf.org2Clinic of Neurological DiseasesAuthorised1818563154931549155922008Ended2024-07-042024-07-04FalseunwillingCzechia2024-07-25Ended306682017-10-232024-08-13306692017-10-232024-08-13520342017-10-232025-03-062025-03-06390932017-10-252024-08-13390942017-10-252024-08-13390972017-10-252021-07-222024-08-13390982017-10-252021-07-222024-08-13True736502017-10-232025-03-062024-08-13FalseFalseFalseTrue2017-10-252021-07-22False4054831549Under evaluation2024-06-25T16:30:49.9484533931549Authorised2024-07-25T14:35:35.6537793631549Ended2025-03-06T20:35:42.9361Czechiaacceptable2025-05-02authorized2024-07-252024-07-2581173234490982523414Hospital/Clinic/Other health care facility8Fakultni Nemocnice V MotoleFalseTrueORG-100012719Active526820V Uvalu 84/1, MotolV Uvalu 84/1MotolPrague150 002008Czechia+420224433301Jana.Haberlova@fnmotol.czTrueORG-1000127191311623JanaHaberlovà+420224433301Jana.Haberlova@fnmotol.cz1Klinika dětské neurologie 2.LF UK a FN MotolAuthorised1818573155031550155922009Ended2024-07-042024-07-04TruewillingDenmark2024-07-24Ended306702020-03-312024-08-13306712020-03-312024-08-13562742020-03-312025-04-302025-05-08390992020-05-042024-08-13391002020-05-042024-08-13391012020-05-042022-06-072024-08-13391022020-05-042022-06-072024-08-13True819032020-03-312025-04-302024-08-13FalseFalseFalseTrue2020-05-042022-06-07True4523131550Authorised2024-07-24T23:58:54.4164054931550Under evaluation2024-06-25T16:30:49.9728251131550Ended2025-05-08T22:06:15.4551Denmarkacceptable2025-04-22authorized2024-07-242024-07-2441173235491001523433Hospital/Clinic/Other health care facility8RigshospitaletFalseTrueORG-100002431Active526840Blegdamsvej 9Blegdamsvej 9Copenhagen Oe21002009Denmark+4535455090christina.hoei-hansen@regionh.dkTrueORG-1000024311311624ChristinaHøi-Hansen+4535455090christina.hoei-hansen@regionh.dk1Børne og Unge KlinikkenAuthorised1818583155131551155922015Ended2024-07-042024-07-04FalseunwillingHungary2024-07-22Ended306762019-02-272024-08-13306772019-02-272024-08-13632072019-02-272025-08-192025-08-26391092019-05-092024-08-13391102019-05-092024-08-13391112019-05-092022-09-072024-08-13391122019-05-092022-09-072024-08-13True952402019-02-272025-08-192024-08-13FalseFalseFalseTrue2019-05-092022-09-07False4055031551Under evaluation2024-06-25T16:30:49.9974462531551Authorised2024-07-22T13:14:22.1489066531551Ended2025-08-26T03:16:55.7271Hungaryacceptable2025-04-02authorized2024-07-222024-07-2211173236491196523628Educational Institution1Semmelweis UniversityTrueTrueORG-100023482Active527052Gyulai Pal Utca 2, KeruletGyulai Pal Utca 2KeruletBudapest VIII10852015Hungary+36206632513molnar.mariajudit@med.semmelweis-univ.huTrueORG-1000234821311625Maria JuditMolnar+36206632513molnar.mariajudit@med.semmelweis-univ.hu1Genomikai Medicina és Ritka Betegségek IntézeteAuthorised1884453155331553155922018Ended2024-07-042024-07-04FalseunwillingItaly2024-08-12Ended306802017-07-042024-08-13306812017-07-042024-08-13706422017-07-042025-10-092025-12-11391172017-08-022024-08-13391182017-08-022024-08-13391192017-08-022022-11-022024-08-13391202017-08-022022-11-022024-08-13True1105792017-07-042025-10-092024-08-13FalseFalseFalseTrue2017-08-022022-11-02False4055231553Under evaluation2024-06-25T16:30:50.0514795631553Authorised2024-08-12T23:59:5917106231553Ended2025-12-11T09:04:36.2311Italyacceptable2025-03-20authorized2024-08-122024-08-12241221683500222532685Hospital/Clinic/Other health care facility8Fondazione Policlinico Universitario Agostino Gemelli IRCCSFalseTrueORG-100014358Active536980Largo Francesco Vito 1Largo Francesco Vito 1Rome001682018Italy+3900630155340mercuri@rm.unicatt.itTrueORG-1000143581364824EugenioMercuri+3900630155340mercuri@rm.unicatt.it2Unità Operativa di Neuropsichiatria Infantile1221684491046523478Hospital/Clinic/Other health care facility8IRCCS Istituto Giannina GasliniFalseTrueORG-100010784Active526889Via Gerolamo Gaslini 5Via Gerolamo Gaslini 5Genoa161472018Italy+39001056362756claudiobruno@gaslini.orgTrueORG-1000107841364825ClaudioBruno+39001056362756claudiobruno@gaslini.org1U.O.S.D. Centro Traslazionale Di Miologia e Patologie NeurodegenerativeAuthorised1818603155431554155922027Ended2024-07-042024-07-04FalseunwillingSpain2024-07-22Ended306842017-06-192024-08-13306852017-06-192024-08-13669782017-06-192025-10-162025-10-22391252017-09-142024-08-13391262017-09-142024-08-13391272017-09-142022-10-272024-08-13391282017-09-142022-10-272024-08-13True1030832017-06-192025-10-162024-08-13FalseFalseFalseTrue2017-09-142022-10-27False4452431554Authorised2024-07-22T10:14:03.7054055331554Under evaluation2024-06-25T16:30:50.07610838731554Ended2025-10-22T08:49:51.7371Spainacceptable2025-04-30authorized2024-07-222024-07-22141173239694232732400Hospital/Clinic/Other health care facility8Hospital Sant Joan De Deu BarcelonaFalseTrueORG-100023083Active758444Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain+34605952542anascimento@hsjdbcn.orgTrueORG-1000230831311628AndresNascimento+34605952542anascimento@hsjdbcn.orgPediatric Neurology Dpt.1173240491021523453Hospital/Clinic/Other health care facility8Hospital Universitario Y Politecnico La FeFalseTrueORG-100029610Active526860Avenida De Fernando Abril Martorell 106Avenida De Fernando Abril Martorell 106Valencia460262027Spain+34961244153nuriamugo@hotmail.comTrueORG-1000296101311629NuriaMuelas Gomez+34961244153nuriamugo@hotmail.com1NeurologiaAuthorised2166333155531555155922031Ended2024-07-042024-07-04FalseunwillingPoland2024-08-04Ended306822019-04-302024-08-13306832019-04-302024-08-13658012019-04-302025-09-292025-10-03391232019-07-022022-09-202024-08-13391242019-07-022022-09-202024-08-13391212019-07-022024-08-13391222019-07-022024-08-13True1005942019-04-302025-09-292024-08-13FalseFalseFalseTrue2019-07-022022-09-20False4677131555Authorised2024-08-04T20:00:13.2374055431555Under evaluation2024-06-25T16:30:50.1029544231555Ended2025-10-03T12:10:39.1321Polandauthorized2024-08-042024-08-04121446062491047523479Hospital/Clinic/Other health care facility8Samodzielny Publiczny Szpital Kliniczny Im. Prof. W. Orlowskiego CMKPFalseTrueORG-100046375Active526890Ul. Czerniakowska 231Ul. Czerniakowska 231Warsaw00-4162031Poland+48225992985akostera@wum.edu.plTrueORG-1000463751610182AnnaKostera-Pruszczyk+48225992985akostera@wum.edu.pl1Klinika Neurologii1446063491209523641Hospital/Clinic/Other health care facility8Uniwersyteckie Centrum KliniczneFalseTrueORG-100042500Active527065Ul. Debinki 7Ul. Debinki 7Gdansk80-9522031Poland+48583492396mmazur@amg.gda.plTrueORG-1000425001610183MariaMazurkiewicz-Beldzinska+48583492396mmazur@amg.gda.pl2Klinika Neurologii RozwojowejAuthorised26701INITIALAuthorised2024-514698-23-00Ended2024-06-25acceptable2024-07-221161033154731547Belgium2002acceptable2024-07-19authorized_conditions2024-07-26EndedBelgiumEnded2024-07-26Authorised1161043154831548Bulgaria2005acceptable2024-07-29authorized2024-07-29EndedBulgariaEnded2024-07-29Authorised1161053154931549Czechia2008acceptable2024-07-19authorized2024-07-25EndedCzechiaEnded2024-07-25Authorised1161063155031550Denmark2009acceptable2024-07-24authorized2024-07-24EndedDenmarkEnded2024-07-24Authorised1161073155131551Hungary2015acceptable2024-07-19authorized2024-07-22EndedHungaryEnded2024-07-22Authorised1161083155231552Ireland2017acceptable2024-07-29authorized2024-07-29EndedIrelandEnded2024-07-29Authorised1161093155331553Italy2018acceptable2024-08-05authorized2024-08-12EndedItalyEnded2024-08-12Authorised1161103155431554Spain2027acceptable2024-07-19authorized2024-07-22EndedSpainEnded2024-07-22Authorised1161113155531555Poland2031acceptable2024-08-01authorized2024-08-04EndedPolandEnded2024-08-04Authorised2024-07-2231547BelgiumEnded31548BulgariaEnded31549CzechiaEnded31550DenmarkEnded31551HungaryEnded31552IrelandEnded31553ItalyEnded31554SpainEnded31555PolandEndedINFalse309532670131547Belgium2024-07-26T08:59:49.986authorized_conditionsacceptabledecision11610345867INITIALFalse313502670131552Ireland2024-07-29T18:24:54.994authorizedacceptabledecision11610845867INITIALFalse321332670131555Poland2024-08-04T20:00:14.625authorizedacceptabledecision11611145867INITIALFalse311372670131548Bulgaria2024-07-29T09:52:28.825authorizedacceptabledecision11610445867INITIALFalse308002670131550Denmark2024-07-24T23:58:55.258authorizedacceptabledecision11610645867INITIALTrue332112670131553Italy2024-08-12T00:00:00authorizedacceptabledecision11610945867INITIALFalse300342670131554Spain2024-07-22T10:14:04.664authorizedacceptabledecision11611045867INITIALFalse309052670131549Czechia2024-07-25T14:35:36.459authorizedacceptabledecision11610545867INITIALFalse301872670131551Hungary2024-07-22T13:14:23.383authorizedacceptabledecision11610745867INITIALFalse45821NON SUBSTANTIAL MODIFICATIONAuthorised2024-514698-23-00Ended2025-01-20acceptable2024-07-222025-01-2031547BelgiumEnded31548BulgariaEnded31549CzechiaEnded31550DenmarkEnded31551HungaryEnded31552IrelandEnded31553ItalyEnded31554SpainEnded31555PolandEndedNSM-1PART_IFalse539774582131547Belgium2025-01-20T19:15:46.965authorizeddecision70801NON SUBSTANTIAL MODIFICATIONFalse539774582131548Bulgaria2025-01-20T19:15:46.965authorizeddecision70801NON SUBSTANTIAL MODIFICATIONFalse539774582131549Czechia2025-01-20T19:15:46.965authorizeddecision70801NON SUBSTANTIAL MODIFICATIONFalse539774582131550Denmark2025-01-20T19:15:46.965authorizeddecision70801NON SUBSTANTIAL MODIFICATIONTrue539774582131551Hungary2025-01-20T19:15:46.965authorizeddecision70801NON SUBSTANTIAL MODIFICATIONFalse539774582131552Ireland2025-01-20T19:15:46.965authorizeddecision70801NON SUBSTANTIAL MODIFICATIONFalse539774582131553Italy2025-01-20T19:15:46.965authorizeddecision70801NON SUBSTANTIAL MODIFICATIONFalse539774582131554Spain2025-01-20T19:15:46.965authorizeddecision70801NON SUBSTANTIAL MODIFICATIONFalse539774582131555Poland2025-01-20T19:15:46.965authorizeddecision70801NON SUBSTANTIAL MODIFICATIONFalse46656SUBSTANTIAL MODIFICATIONAuthorised2024-514698-23-00Ended2025-01-31acceptable2025-05-021818543154731547Belgium2002acceptable2025-04-14authorized_conditions2024-07-26EndedBelgiumEnded2024-07-26Authorised1943593154831548Bulgaria2005acceptable2025-04-22authorized2024-07-29EndedBulgariaEnded2024-07-29Authorised1818563154931549Czechia2008acceptable2025-05-02authorized2024-07-25EndedCzechiaEnded2024-07-25Authorised1818573155031550Denmark2009acceptable2025-04-22authorized2024-07-24EndedDenmarkEnded2024-07-24Authorised1818583155131551Hungary2015acceptable2025-04-02authorized2024-07-22EndedHungaryEnded2024-07-22Authorised1884453155331553Italy2018acceptable2025-03-20authorized2024-08-12EndedItalyEnded2024-08-12Authorised1818603155431554Spain2027acceptable2025-04-30authorized2024-07-22EndedSpainEnded2024-07-22Authorised1994373155531555Poland2031acceptable2025-05-04authorized2024-08-04EndedPolandEnded2024-08-04Authorised2025-05-0231547BelgiumEnded31548BulgariaEnded31549CzechiaEnded31550DenmarkEnded31551HungaryEnded31553ItalyEnded31554SpainEnded31555PolandEndedSM-1PART_I_AND_PART_IIFalse695714665631550Denmark2025-05-02T12:52:19.429authorizedacceptabledecision18185782589SUBSTANTIAL MODIFICATIONTrue698484665631547Belgium2025-05-05T16:30:06.851authorizedacceptabledecision18185482589SUBSTANTIAL MODIFICATIONFalse699574665631549Czechia2025-05-06T11:06:35.087authorized_conditionsacceptabledecision18185682589SUBSTANTIAL MODIFICATIONFalse706414665631555Poland2025-05-09T15:41:02.597authorizedacceptabledecision19943782589SUBSTANTIAL MODIFICATIONFalse703314665631551Hungary2025-05-07T16:07:25.472authorizedacceptabledecision18185882589SUBSTANTIAL MODIFICATIONFalse697854665631554Spain2025-05-05T12:46:30.189authorizedacceptabledecision18186082589SUBSTANTIAL MODIFICATIONFalse704214665631548Bulgaria2025-05-08T12:08:15.722authorizedacceptabledecision19435982589SUBSTANTIAL MODIFICATIONFalse700434665631553Italy2025-05-06T14:41:04.74authorizedacceptabledecision18844582589SUBSTANTIAL MODIFICATIONFalse57856NON SUBSTANTIAL 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10-15_PL_Public3a737a74-6225-4ac2-8ee4-03472a153a3315Subject information and informed consent form (for publication)PDF21663319.02Both3 2023-508510-42-005AuthorisedC3391003 - A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHYC3391003DUCHENNE MUSCULAR DYSTROPHY (DMD)Italy:5Germany:5Belgium:5France:5Spain:510/07/2024FR: 21/08/2024, BE: 12/07/2024, ES: 15/07/2024, IT: 12/08/2024, DE: 10/07/2024Diseases [C] - Musculoskeletal Diseases [C05]Pfizer Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)Change from Baseline in percent normal mini-dystrophin expression level in biceps brachii muscle biopsies at Day 360 using a liquid chromatography mass spectrometry (LC-MS) assay., Change from Baseline in percent of muscle fibers expressing mini-dystrophin in biceps brachii muscle biopsies at Day 360 as assessed by immunofluorescence., Change from Baseline at Week 52 in serum CK concentration., Number of skills gained at Week 52 based on the individual items of the NSAA., Number of skills either improved or maintained at Week 52 based on the individual items of the NSAA., Change from Baseline at Week 52 in the 10 meter run/walk velocity., Change from Baseline at Week 52 in the rise from floor velocity., Change from Baseline at Week 52 in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale (Pediatric Parent)., Change from Baseline at Week 52 in the Modified PODCI: Sports and Physical Functioning Core Scale (Pediatric Parent).Fordadistrogene Movaparvovec, ECULIZUMAB, Placebo for PF-06939926540-17 yearsMale3Both48Change from Baseline at Week 52 in the NSAA total score.No20/02/202621/02/20262024-07-10T08:21:39.0022026-02-21T02:35:27.1591062015C3391003_PH file_SM1_Recruitment completedff28131f-62ac-4e71-85ad-62a560f8e5c714Recruitment arrangements (for publication)PDF273688N/A2L1_ICD 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C3391003 2023-508510-42-00 ES Public7b18f4d3-5459-4dc8-94d8-233d643775fa7Synopsis of the protocol (for publication)PDF119140Am161D2 Protocol Synopsis C3391003 2023-508510-42-00 FR Public75b3568b-9821-4444-bc06-e5fa0dea90c87Synopsis of the protocol (for publication)PDF119140Am161D2 Protocol Synopsis C3391003 2023-508510-42-00 IT Public533a88eb-f36a-4812-81e8-2a53e659aae17Synopsis of the protocol (for publication)PDF119140Am161D2 Protocol Synopsis C3391003 2023-508510-42-00 DE Publicfae470b4-8b0a-4f27-8d16-e5434302c1e87Synopsis of the protocol (for publication)PDF119140Am 161Italy298692024-08-12T00:00:002026-02-20T09:15:15.2795Germany298682024-07-10T08:21:39.0022026-02-20T13:13:22.575Belgium298662024-07-12T15:29:14.8512026-01-20T15:02:12.9445France298672024-08-21T10:43:34.3892026-01-20T15:02:12.9445Spain298702024-07-15T11:46:14.4572026-01-20T15:02:12.944529867TH-440502024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-09-04T09:53:34Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29867FranceFR29868TH-363632024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-07-23T14:41:21Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29868GermanyDE29870TH-363652024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-07-23T14:44:10Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29870SpainES29866TH-363672024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-07-23T14:49:26Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29866BelgiumBE29869TH-416652024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-08-20T14:05:00Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29869ItalyIT29866BelgiumRESTART_OF_TRIAL2025-03-24START_OF_TRIAL2021-12-13START_OF_RECRUITMENT2021-12-15END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-0329867FranceRESTART_OF_TRIAL2025-03-27START_OF_TRIAL2022-09-15START_OF_RECRUITMENT2023-01-24END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-0329868GermanyRESTART_OF_TRIAL2025-03-28START_OF_TRIAL2023-02-24START_OF_RECRUITMENT2023-03-02END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-0329869ItalyRESTART_OF_TRIAL2025-03-27START_OF_TRIAL2020-12-18START_OF_RECRUITMENT2020-12-21END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-0329870SpainRESTART_OF_TRIAL2025-03-26START_OF_TRIAL2020-11-03START_OF_RECRUITMENT2020-11-05END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-032023-508510-42-005C3391003 - A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHYC339100324/03/2025DUCHENNE MUSCULAR DYSTROPHY (DMD)Italy:5Germany:5Belgium:5France:5Spain:510/07/2024FR: 21/08/2024, BE: 12/07/2024, ES: 15/07/2024, IT: 12/08/2024, DE: 10/07/2024Diseases [C] - Musculoskeletal Diseases [C05]Pfizer Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)Change from Baseline in percent normal mini-dystrophin expression level in biceps brachii muscle biopsies at Day 360 using a liquid chromatography mass spectrometry (LC-MS) assay., Change from Baseline in percent of muscle fibers expressing mini-dystrophin in biceps brachii muscle biopsies at Day 360 as assessed by immunofluorescence., Change from Baseline at Week 52 in serum CK concentration., Number of skills gained at Week 52 based on the individual items of the NSAA., Number of skills either improved or maintained at Week 52 based on the individual items of the NSAA., Change from Baseline at Week 52 in the 10 meter run/walk velocity., Change from Baseline at Week 52 in the rise from floor velocity., Change from Baseline at Week 52 in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale (Pediatric Parent)., Change from Baseline at Week 52 in the Modified PODCI: Sports and Physical Functioning Core Scale (Pediatric Parent).Fordadistrogene Movaparvovec, ECULIZUMAB, Placebo for PF-06939926540-17 yearsMale348Change from Baseline at Week 52 in the NSAA total score.No20/02/202621/02/20262023-508510-42-00Authorised2025-03-242024-07-10T08:21:39.0022026-02-21T02:35:27.159106201511914074100000000329Australia36AUAUSTrue100000000506Russian Federation643RURUSTrue100000000439Korea, Republic of410KRKORTrue100000000556United Kingdom826GBGBRTrue100000000536Switzerland756CHCHETrue100000000354Canada124CACANTrue100000000557United States840USUSATrue100000000432Japan392JPJPNTrue100000000538Taiwan158TWTWNTrue100000000429Israel376ILISRTrue493958110928501SOLUTION FOR INFUSIONPRD109285011Fordadistrogene MovaparvovecSOLUTION FOR INFUSIONPF-06939926FORDADISTROGENE MOVAPARVOVECSUB193152PFIZER INC.10928501347699PFIZER INC.Structurally Diverse Substance - OtherStructurally Diverse Substance - OtherFORDADISTROGENE MOVAPARVOVECSUB193152Adeno-associated viral vector serotype 9 containing the human mini-dystrophin genePF-06939926BMD-001False1TrueEU/3/16/1716vector genomes (vg)/mL200000000000000vector genomes (vg)/mL20000000000000011Advanced Therapy IMP (ATIMP)PRD10928501PF-0693992662473DMD generecombinant adeno-associated virusFalseTrue3118INTRAVENOUS USEFalseFordadistrogene Movaparvovecfordadistrogene movaparvovecSOLUTION FOR INFUSION4939593SUB25187-2ECULIZUMABCONCENTRATE FOR SOLUTION FOR INFUSIONECULIZUMABSUB2518725187-ECULIZUMABSUB25187---False4TrueEU/3/09/653mg milligram(s)1200mg milligram(s)1200False11SUB251877SOLUTION FOR INFUSIONFalseECULIZUMABeculizumab4939601N/AN/AN/APlacebo for PF-06939926N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePlacebo for PF-06939926n/aN/AC3391003 - A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY98000054089f63f-28f3-48ce-b5d6-ca9f0a083b29ÉTUDE DE PHASE 3, MULTICENTRIQUE, RANDOMISÉE, EN DOUBLE AVEUGLE ET CONTRÔLÉE PAR PLACEBO ÉVALUANT L'INNOCUITÉ ET L'EFFICACITÉ DU PF-06939926 DANS LE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE DE DUCHENNE34French (France)98000044089f63f-28f3-48ce-b5d6-ca9f0a083b29UN ESTUDIO DE FASE 3, MULTICÉNTRICO, CON ALEATORIZACIÓN, DOBLE CIEGO Y CONTROLADO CON PLACEBO PARA EVALUAR LA SEGURIDAD Y EFICACIA DE PF-06939926 PARA EL TRATAMIENTO DE LA DISTROFIA MUSCULAR DE DUCHENNE7SpanishA PHASE 3, RANDOMIZED, DOUBLE-BLIND,PLACEBO-CONTROLLED STUDY OF PF-06939926 IN DMD98000254b693765-1fee-4fde-a43e-900d0ded2634ÉTUDE DE PHASE 3, RANDOMISEE, EN DOUBLE AVEUGLE ET CONTROLEE PAR PLACEBO SUR LE PF-06939926 DANS LA DMD34French (France)98000244b693765-1fee-4fde-a43e-900d0ded2634UN ESTUDIO DE FASE 3, CON ALEATORIZACIÓN, DOBLE CIEGO Y CONTROLADO CON PLACEBO SOBRE PF-06939926 PARA DMD7SpanishC3391003490153NCT04281485False52This is a Phase 3 Trial109176123960DUCHENNE MUSCULAR DYSTROPHY (DMD)97999480888c61d-509e-4252-b26b-3ecb9156ed1dDISTROFIA MUSCULAR DE DUCHENNE (DMD)7Spanish97999490888c61d-509e-4252-b26b-3ecb9156ed1dDYSTROPHIE MUSCULAIRE DE DUCHENNE34French (France)True10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False536068033606814360682To demonstrate superior efficacy of treatment with fordadistrogene movaparvovec as compared to placebo based on change from Baseline in the North Star Ambulatory Assessment (NSAA).98000223cc12d5c-0e24-40a2-80e8-76da96aff1d3Démontrer la supériorité de l’efficacité du traitement par fordadistrogene movaparvovec comparativement au placebo d’après la variation par rapport à la valeur initiale du score obtenu sur l’Échelle d’évaluation ambulatoire North Star (NSAA)34French (France)98000233cc12d5c-0e24-40a2-80e8-76da96aff1d3Demostrar una eficacia superior del tratamiento con fordadistrogén movaparvovec en comparación con el placebo según el cambio con respecto al valor de referencia en la evaluación ambulatoria North Star (NSAA).7Spanish4192431To quantify the mini-dystrophin expression level in the muscle of participants treated with fordadistrogene movaparvovec9800027b5d9ccee-fcf4-46d2-b6b5-1a74ca5e282bCuantificar el nivel de expresión de minidistrofina en el músculo de los participantes tratados con fordadistrogene movaparvovec7Spanish9800026b5d9ccee-fcf4-46d2-b6b5-1a74ca5e282bQuantifier le niveau d'expression de mini-dystrophine dans le muscle des patients traités par fordadistrogene movaparvovec34French (France)4192442To characterize the distribution of mini-dystrophin expression in the muscle of participants treated with fordadistrogene movaparvovec98000295af41c87-b7fe-453d-957f-cbed9d33b09dCaracterizar la distribución de la expresión de minidistrofina en el músculo de los participantes tratados con fordadistrogene movaparvovec7Spanish98000285af41c87-b7fe-453d-957f-cbed9d33b09dCaractériser la distribution de l’expression de la mini-dystrophine dans le muscle des patients traités par fordadistrogene movaparvovec.34French (France)4192453To characterize the change in serum creatine kinase (CK) concentration in participants treated with fordadistrogene movaparvovec as compared to placebo.9800031e41c3890-e67a-4b6c-9cf5-b04dde381264Caracterizar el cambio en la concentración de creatina quinasa (Creatine Kinase, CK) sérica en los participantes tratados con fordadistrogene movaparvovec en comparación con el placebo.7Spanish9800030e41c3890-e67a-4b6c-9cf5-b04dde381264Caractériser la variation de la concentration sérique de créatine kinase (CK) chez les patients traités par fordadistrogene movaparvovec comparativement au placebo.34French (France)4192464To characterize the skills gained, based on the individual items of the NSAA, in participants treated with PF-06939926 as compared to placebo.9800032645f65a6-ed1c-43dd-ae32-63edb892ad11Caracterizar las habilidades adquiridas, según los elementos individuales de la NSAA, en los participantes tratados con PF-06939926 en comparación con el placebo.7Spanish9800033645f65a6-ed1c-43dd-ae32-63edb892ad11Caractériser les compétences acquises, d’après les items individuels de l’échelle NSAA, chez les patients traités par PF-06939926 comparativement au placebo.34French (France)4192475To characterize the skills either improved or maintained, based on the individual items of the NSAA, in participants treated with fordadistrogene movaparvovec as compared to placebo.9800034579e032a-f340-4fe2-a0dc-eb6f5ad9d9f1Caracterizar las habilidades mejoradas o mantenidas, según los elementos individuales de la NSAA, en los participantes tratados con fordadistrogén movaparvovec en comparación con el placebo7Spanish9800035579e032a-f340-4fe2-a0dc-eb6f5ad9d9f1Caractériser les compétences améliorées ou maintenues, d’après les items individuels de l’échelle NSAA, chez les patients traités par fordadistrogene movaparvovec comparativement au placebo.34French (France)4192486To characterize the 10-meter run/walk velocity in participants treated with PF-06939926 as compared to placebo.98000360aace278-bab7-4387-8e52-e7f9ad761c22Caractériser la vitesse de marche/course sur 10 mètres chez les patients traités par PF-06939926 comparativement au placebo.34French (France)98000370aace278-bab7-4387-8e52-e7f9ad761c22Caracterizar la velocidad de carrera/caminata de 10 metros en los participantes tratados con PF-06939926 en comparación con el placebo.7Spanish4192497To characterize the rise from floor velocity in participants treated with fordadistrogene movaparvovec as compared to placebo.98000383bd5af15-2322-4b29-b826-b7606adbef0dCaractériser la vitesse du lever depuis la position couchée chez les patients traités par fordadistrogene movaparvovec comparativement au placebo.34French (France)98000393bd5af15-2322-4b29-b826-b7606adbef0dCaracterizar la velocidad de elevación desde el piso en los participantes tratados con fordadistrogén movaparvovec en comparación con el placebo.7Spanish4192508To characterize the functional health status in participants treated with fordadistrogene movaparvovec as compared to placebo.9800040a05f0298-03cd-4501-a9f9-060ef93a51b2Caracterizar el estado de salud funcional en los participantes tratados con fordadistrogén movaparvovec en comparación con el placebo.7Spanish9800041a05f0298-03cd-4501-a9f9-060ef93a51b2Caractériser l'état de santé fonctionnel chez les patients traités par fordadistrogene movaparvovec comparativement au placebo.34French (France)8016471Male participants who are ≥4 and <8 years of age at Screening (Visit 1).98000070fb665d2-f149-4345-8f77-6009e75f991aPatients masculins âgés de ≥ 4 ans et < 8 ans au moment de la sélection (visite 1).34French (France)98000060fb665d2-f149-4345-8f77-6009e75f991aParticipantes masculinos que tengan ≥4 y <8 años en la Selección (Visita 1).7Spanish8016482Confirmed diagnosis of DMD by prior genetic testing demonstrating the presence of a mutation in the dystrophin gene consistent with DMD at Screening (Visit 1). If the Investigator determines that the results are inconclusive, a repeat genetic testing will be allowed through the central laboratory at Screening (Visit 1).98000089190e832-baa9-42c0-abdf-3e9b37d6c3cdDiagnóstico confirmado de DMD mediante prueba genética previa que demuestre la presencia de una mutación en el gen distrofina conforme a la DMD en la Selección (Visita 1). Si el investigador determina que los resultados no son concluyentes, se permitirá la repetición de una prueba genética mediante el laboratorio central en la Selección (Visita 1).7Spanish98000099190e832-baa9-42c0-abdf-3e9b37d6c3cdDiagnostic confirmé de DMD par des tests génétiques antérieurs démontrant la présence d'une mutation du gène de la dystrophine compatible avec une DMD lors de la sélection (visite 1). Si l'investigateur décide que les résultats ne sont pas concluants, la réalisation d’un nouveau test génétique par le laboratoire central est autorisée lors de la sélection (visite 1).34French (France)8016493Receipt of a stable daily dose of glucocorticoids (≥0.5 mg/kg/day prednisone, prednisolone, or ≥0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening (Visit 1) and during the period between Screening (Visit 1) and Day 1 (Visit 3). In order to comply with protocol procedures, there should also be a reasonable expectation that this daily dose of glucocorticoids will remain stable for the first 2 years of the study. A stable dose is defined as one in which any change is ≤0.2 mg/kg.980001109a7ce53-d9c0-47f7-b111-fdaa5127f53cRecepción de una dosis diaria estable de glucocorticoides (≥0,5 mg/kg por día de prednisona o prednisolona, o ≥0,75 mg/kg por día de deflazacort) durante al menos 3 meses previos a la Selección (Visita 1) y durante el período entre la Selección (Visita 1) y el Día 1 (Visita 3). Para cumplir con los procedimientos del protocolo, también debe existir una expectativa razonable de que esta dosis diaria de glucocorticoides permanecerá estable durante los primeros 2 años del estudio. Una dosis estable se define como una dosis en la cual cualquier cambio es de ≤0,2 mg/kg7Spanish980001009a7ce53-d9c0-47f7-b111-fdaa5127f53cTraitement par une dose quotidienne stable de glucocorticoïdes (≥ 0,5 mg/kg/jour de prednisone, prednisolone ou ≥ 0,75 mg/kg/jour de déflazacort) pendant au moins 3 mois avant la sélection (visite 1) et pendant la période entre la sélection (visite 1) et le jour 1 (visite 3). Afin de se conformer aux procédures du protocole, il doit aussi pouvoir être raisonnablement supposé que cette dose quotidienne de glucocorticoïdes restera stable pendant les 2 premières années de l’étude. Une dose stable désigne une dose pour laquelle toute modification est ≤ 0,2 mg/kg.34French (France)8016504A NSAA total score >16 and <30 at Screening (Visit 1).9800012ba14d36a-2ed5-45ad-8af8-adde49719d8eUna puntuación total de NSAA es de >16 y <30 en la Selección (Visita 1).7Spanish9800013ba14d36a-2ed5-45ad-8af8-adde49719d8eScore total NSAA > 16 et < 30 à la sélection (visite 1).34French (France)8016515Ambulatory, defined as being able to walk 10 meters unassisted, at Screening (Visit 1).9800015d7b3f99c-a50e-47da-a144-0271627e50abAmbulatorio, definido como capaz de caminar 10 metros sin asistencia, en la Selección (Visita 1).7Spanish9800014d7b3f99c-a50e-47da-a144-0271627e50abÊtre ambulatoire, défini comme le fait d’être capable de marcher sur 10 mètres sans assistance, lors de la sélection (visite 1).34French (France)8016526Participants/legally acceptable representatives who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures including, potentially, open muscle biopsies under general anesthesia and cardiac MRI under general anesthesia.98000164c8f17f0-0a7e-4349-8b76-0f33a38d46e5Patients/représentants légalement acceptables disposés et aptes à se conformer à toutes les visites programmées, au plan de traitement, aux analyses biologiques, aux considérations liées au mode de vie et aux autres procédures de l’étude, y compris, vraisemblablement, à des biopsies musculaires incisionnelles sous anesthésie générale et à une IRM cardiaque sous anesthésie générale.34French (France)98000174c8f17f0-0a7e-4349-8b76-0f33a38d46e5Participantes o representantes autorizados legalmente que están dispuestos y son capaces de cumplir con todas las visitas programadas, el plan del tratamiento, las pruebas de laboratorio, las consideraciones de estilo de vida y otros procedimientos del estudio, incluidas, posiblemente, las biopsias musculares abiertas bajo anestesia general y la RMN cardíaca bajo anestesia general.7Spanish8016537Participants/legally acceptable representatives who are capable of giving assent/signed informed consent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the assent/informed consent document (ICD) and in this protocol.98000197e5d4fd7-b828-424f-a259-96656cf54757Participantes o representantes autorizados legalmente que son capaces de dar el asentimiento o consentimiento informado firmado, como se describe en el apéndice 1, que incluye el cumplimiento de los requisitos y las restricciones que figuran en el asentimiento o documento de consentimiento informado (DCI) y en este protocolo.7Spanish98000187e5d4fd7-b828-424f-a259-96656cf54757Patients/représentants légalement acceptables aptes à donner leur assentiment/consentement éclairé signé comme décrit dans l’annexe 1, ce qui inclut le respect des exigences et des restrictions énumérées dans le document d’assentiment/formulaire de consentement éclairé (DCE) et dans le présent protocole.34French (France)8016548Participants/legally acceptable representatives who are willing to protect the integrity of the study data by not actively seeking sensitive clinical data (eg, CK, ALT, AST, NAb to AAV9) through independent laboratory tests and by not sharing trial experiences with other participants or publicly (eg, through social media).98000204c118d17-8dc1-4762-a722-e62ef741e9e3Patients/représentants légalement acceptables disposés à protéger l’intégrité des données de l’étude en ne recherchant pas activement des données cliniques sensibles (par exemple CK, ALAT, ASAT, AcN anti-AAV9) par le biais d’analyses effectuées dans des laboratoires indépendants et en ne partageant pas leurs expériences concernant l’essai avec d’autres patients ou publiquement (par exemple via les réseaux sociaux).34French (France)98000214c118d17-8dc1-4762-a722-e62ef741e9e3Participantes o representantes autorizados legalmente que están dispuestos a proteger la integridad de los datos del estudio al no buscar de forma activa datos clínicos confidenciales (p. ej., CK, ALT, AST, AcN para VAA9) mediante pruebas de laboratorio independientes y al no compartir las experiencias del ensayo con otros participantes o de forma pública (p. ej., a través de redes sociales).7Spanish13731951Prior treatment with gene therapy, defined as any therapy introducing exogenous DNA or intended to permanently alter the endogenous DNA. Gene therapy (other than IP) will be prohibited for the duration of the study.9799970119c9448-9289-466e-96f4-c4a197e25fcfTratamiento previo con terapia génica, definida como cualquier terapia en la que se introduzca ADN exógeno o destinada a alterar de forma permanente el ADN endógeno. Se prohibirá la terapia génica (que no sea el PEI) durante el estudio.7Spanish9799971119c9448-9289-466e-96f4-c4a197e25fcfTraitement antérieur par thérapie génique, défini comme tout traitement avec introduction d’ADN exogène ou destiné à modifier définitivement l’ADN endogène. Toute thérapie génique (autre que le ME) sera interdite pendant la durée de l’étude.34French (France)137319610Acute infection at Screening (Visit 1) or Baseline (Visit 2) that, in the judgement of the Investigator is not expected to be fully resolved at least 2 weeks before Day 1 (Visit 3). At Day 1 (Visit 3), participants must have been infection-free for at least 2 weeks prior to IP administration. Delay of IP administration for up to 14 days is permitted to enable infections to become fully resolved.9799972354ff010-cdde-40b7-bd77-197a982a99f9Infection aiguë à la sélection (visite 1) ou à l'inclusion (visite 2) qui, de l'avis de l'investigateur, ne devrait pas être totalement résolue au moins 2 semaines avant le jour 1 (visite 3). Au jour 1 (visite 3), les patients doivent avoir été exempts d'infection pendant au moins 2 semaines avant l'administration du ME. Un retard d'administration du ME jusqu'à 14 jours est autorisé pour permettre une résolution complète des infections.34French (France)9799973354ff010-cdde-40b7-bd77-197a982a99f9Infección aguda en la Selección (Visita 1) o el Inicio (Visita 2) cuya resolución completa, a juicio del investigador, no se espere al menos 2 semanas antes del Día 1 (Visita 3). En el Día 1 (Visita 3), los participantes deben estar libres de infecciones durante al menos 2 semanas antes de la administración del PEI. Se permite una demora de la administración del PEI de 14 días como máximo para permitir que las infecciones se resuelvan por completo.7Spanish137319711Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or Pfizer employees, including their family members, directly involved in the conduct of the study.9799975ff8ffa5f-db40-4314-9432-9e9f29d12167Membres du personnel du centre directement impliqués dans la réalisation de l’étude, ainsi que les membres de leur famille, membres du personnel du centre par ailleurs supervisés par l’investigateur, ou employés de Pfizer, ainsi que les membres de leur famille, directement impliqués dans la réalisation de l’étude.34French (France)9799974ff8ffa5f-db40-4314-9432-9e9f29d12167Miembros del personal del centro del investigador implicados directamente en la realización del estudio y sus familiares; miembros del personal del centro que sean supervisados de algún modo por el investigador, o empleados de Pfizer (incluidos sus familiares) implicados directamente en la realización del estudio7Spanish137319812Known hypersensitivity to any of the components of the IP or solution for infusion, such as hypersensitivity to albumin or a diagnosis of HFI. Symptoms suggestive of HFI include nausea, vomiting, bloating, stomach cramps, or diarrhea following the ingestion of sweet foods or drinks, or a pattern of avoiding sweet foods or drinks.97999775fe74b7b-6315-4970-9725-25a29440af74Hipersensibilidad conocida a cualquiera de los componentes del PEI o la solución para infusión, como hipersensibilidad a la albúmina o un diagnóstico de IHF. Los síntomas que sugieren una IHF incluyen náuseas, vómitos, hinchazón, dolor estomacal o diarrea después de la ingesta de alimentos o bebidas dulces, o un patrón de evasión de alimentos o bebidas dulces.7Spanish97999765fe74b7b-6315-4970-9725-25a29440af74Hypersensibilité connue à un des composants du ME ou à une solution de perfusion, comme l’hypersensibilité à l’albumine, ou un diagnostic d’intolérance héréditaire au fructose (IHF). Les symptômes évocateurs de l’IHF sont notamment des nausées, des vomissements, des ballonnements, des crampes abdominales ou de la diarrhée après l’ingestion de boissons ou d’aliments sucrés, ou une habitude d’éviter les boissons ou les aliments sucrés.34French (France)137319913Contraindication to the use of eculizumab, as per the local prescribing information.97999793c2ccb18-5737-4be1-9af0-37e58f6948b7Contre-indication à l’éculizumab, selon les informations locales relatives à la prescription.34French (France)97999783c2ccb18-5737-4be1-9af0-37e58f6948b7Contraindicación del uso de eculizumab, de acuerdo con la información del prospecto local.7Spanish137320014LVEF <50% on echocardiogram performed at the Screening Visit (Visit 1), as evaluated by the central reader.9799981cd041748-d9d7-4413-8b4e-688a84e0a454FEVG < 50 % à l’électrocardiogramme de la visite de sélection (visite 1) telle qu’évaluée par le lecteur central.34French (France)9799980cd041748-d9d7-4413-8b4e-688a84e0a454FEVI <50 % en el ecocardiograma realizado en la visita de selección (Visita 1), según lo determinado por el evaluador central.7Spanish137320115Participants with the following genetic abnormalities in the dystrophin gene as confirmed by the investigator based on the review of DMD genetic testing: a. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and exon 30; OR c. A deletion that affects any exons between 56-71, inclusive.9799982c6f57bc3-0b08-4b88-a49a-c019c676be79Los participantes con las siguientes anomalías genéticas en el gen distrofina confirmado por el investigador con base en la revisión de la prueba genética DMD: a. Cualquier mutación (deleción o duplicación del exón, inserción o mutación puntual) que afecte a cualquier exón entre el exón 9 y el 13 inclusive; O BIEN b. Una deleción que afecte tanto al exón 29 como al exón 30; O BIEN c. Una deleción que afecte a cualquiera de los exones entre el 56 y el 71, incluidos ambos.7Spanish9799983c6f57bc3-0b08-4b88-a49a-c019c676be79Les anomalies génétiques suivantes du gène de la dystrophine, confirmées par l’investigateur sur la base de l’examen des tests génétiques de la DMD : • Toute mutation (délétion d’exon, duplication d’exon, insertion ou mutation ponctuelle) affectant tout exon entre l’exon 9 et l’exon 13, inclusivement ; OU • Une délétion qui affecte à la fois l’exon 29 et l’exon 30 ; OU • Une délétion qui affecte n’importe quel exon entre 56 et 71 inclus34French (France)137320216Cardiac pathologies, as evaluated by a pediatric cardiologist at the Screening Visit (Visit 1): a. Diagnosis of myocarditis (eg, viral): either based on prior medical history or based on findings in cardiac imaging tests; b. Any other cardiac history, and/or condition and/or abnormalities in cardiac imaging, that determine that the participant should not be included in the study, as per the cardiologist.97999855138fa37-cc77-4a6b-8f28-a9ce246e3569Patologías cardíacas, evaluadas por un cardiólogo pediátrico en la visita de selección (Visita 1): a. Diagnóstico de miocarditis (p. ej., viral): basado o bien en antecedentes médicos o en hallazgos en imágenes cardíacas; b. Cualquier otro antecedente cardíaco y/o afección y/o anomalías en las imágenes cardíacas que determinen que no debe incluirse al paciente en el estudio según el cardiólogo.7Spanish97999845138fa37-cc77-4a6b-8f28-a9ce246e3569Les pathologies cardiaques, telles qu’évaluées par le cardiologue pédiatrique lors de la visite de sélection (visite 1) : a. Diagnostic de myocardite (p. ex. virale) : soit à partir des antécédents médicaux soit à partir de découvertes issues des tests d’imagerie ; b. Tout autre antécédent cardiaque et/ou toute autre maladie et/ou anomalie découverte lors d’un test d’imagerie cardiaque qui détermineraient que le patient ne devrait pas être inclus dans l’étude selon le cardiologue.34French (France)137320317Not a candidate for mechanical cardiac or respiratory support, or any other invasive intervention, if indicated for management of an acute event as determined by the cardiologist in consultation with the investigator at the Screening Visit (Visit 1).9799987a30eb0ec-7a72-4c60-a2a0-afe714063303No es candidato para recibir asistencia mecánica cardíaca o respiratoria, o cualquier otra intervención invasiva, si está indicado para la gestión de un episodio agudo según lo determine el cardiólogo en consulta con el investigador en la visita de selección (Visita 1).7Spanish9799986a30eb0ec-7a72-4c60-a2a0-afe714063303Pas candidats à une assistance cardiaque ou respiratoire mécanique, ou à toute autre intervention invasive pour le diagnostic ou la prise en charge d’un événement cardiaque grave, tel que déterminé par le cardiologue en concertation avec l’investigateur à la visite de sélection (visite 1).34French (France)13732042Exposure within 6 months prior to Screening (Visit 1) to any treatment designed to increase dystrophin expression (including, but not limited to exon-skipping and nonsense read-through). These treatments will also be prohibited during the period between Screening (Visit 1) and Day 1 (Visit 3) and for the first 52 weeks of the study. Please note that for participants who are eligible for these treatments: Participants may be enrolled who have previously experienced lack of efficacy, or intolerance, as long as they received their last dose more than 6 months before screening (Visit 1), or who have refused these treatments. Participants receiving these treatments from which there is believed to be benefit should not discontinue them in order to meet this exclusion criterion and enroll in the study.9799988a2dbfcf7-442c-4cac-ae50-d47c1f45310dLa exposición dentro de los 6 meses previos a la Selección (Visita 1) a cualquier tratamiento diseñado para aumentar la expresión de distrofina (incluidas, entre otras, omisiones de exones o translecturas de mutación sin sentido). Estos tratamientos también se prohibirán durante el período entre la Selección (Visita 1) y el Día 1 (Visita 3), y las primeras 52 semanas del estudio. Tenga en cuenta lo siguiente para los participantes que son aptos para estos tratamientos: • Los participantes que hayan experimentado anteriormente falta de eficacia o intolerancia pueden inscribirse, siempre y cuando hayan recibido la última dosis más de 6 meses antes de la selección (Visita 1) o hayan rechazado estos tratamientos. • Los participantes que reciban estos tratamientos, de los cuales se cree que podrían obtener beneficios, no deben interrumpirlos para cumplir este criterio de exclusión e inscribirse en el estudio.7Spanish9799989a2dbfcf7-442c-4cac-ae50-d47c1f45310dExposition dans les 6 mois précédant la sélection (visite 1) à un traitement conçu pour augmenter l’expression de la dystrophine (y compris, mais sans s’y limiter, saut d’exon thérapeutique et translecture non-sens). Ces traitements seront également interdits pendant la période comprise entre la sélection (visite 1) et le jour 1 (visite 3) ainsi que pendant les 52 premières semaines de l’étude. Veuillez noter que dans le cas de patients éligibles pour ces traitements : • Il est possible d’inclure des patients qui ont déjà subi un manque d’efficacité ou une intolérance, à condition qu’ils aient reçu leur dernière dose plus de 6 mois avant la sélection (visite 1), ou qui ont refusé ces traitements. • Les patients recevant ces traitements présumés bénéfiques ne doivent pas les interrompre afin de satisfaire à ce critère et de s’inscrire à l’étude.34French (France)13732053Previous administration with an investigational drug or investigational vaccine within 30 days (or as determined by the local requirement) or 5 half-lives (whichever is longer) at Screening (Visit 1). These treatments will also be prohibited during the period between Screening (Visit 1) and Day 1 (Visit 3) and for the first 2 years of the study.979999007ba5d96-26e4-4652-ad50-8d4f6dbdfc84Administration antérieure d’un médicament ou vaccin expérimental dans les 30 jours (ou selon les exigences locales) ou les 5 demi-vies (selon la durée la plus longue) lors de la sélection (visite 1). Ces traitements seront également interdits pendant la période comprise entre la sélection (visite 1) et le jour 1 (visite 3) ainsi que pendant les 2 premières années de l’étude.34French (France)979999107ba5d96-26e4-4652-ad50-8d4f6dbdfc84Administración previa de un fármaco o vacuna en fase de investigación dentro de 30 días (o según lo determinen los requisitos locales) o 5 semividas (el período que sea más extenso) en la Selección (Visita 1). Estos tratamientos también se prohibirán durante el período entre la Selección (Visita 1) y el Día 1 (Visita 3), y los primeros 2 años del estudio.7Spanish13732064Known cognitive impairment or behavioral issues that would impede the ability to follow instructions, in the judgment of the Investigator, at Screening (Visit 1).97999920a6e42cb-ae93-4df6-88f6-f6cc5c588585Problemas cognitivos o de comportamiento conocidos que dificultarían la capacidad para seguir instrucciones, según el criterio del investigador, en la Selección (Visita 1).7Spanish97999930a6e42cb-ae93-4df6-88f6-f6cc5c588585Déficience cognitive connue ou problèmes comportementaux qui, de l’avis de l’investigateur, sont susceptibles d’entraver la capacité à se conformer aux instructions, lors de la sélection (visite 1).34French (France)13732075Any nonhealed injury at Screening (Visit 1) which, in the opinion of the Investigator, may impact functional testing; additionally, lower limb fractures must have been healed for at least 3 months prior to Screening (Visit 1).97999942dd53d68-3834-4f0b-acd9-cf7f20eed41aCualquier lesión no sanada en la Selección (Visita 1) que, según la opinión del investigador, pueda tener una repercusión sobre las pruebas funcionales; además, las fracturas de miembros inferiores deben haberse consolidado durante al menos 3 meses previos a la Selección (Visita 1).7Spanish97999952dd53d68-3834-4f0b-acd9-cf7f20eed41aToute blessure non cicatrisée lors de la sélection (visite 1) qui, de l'avis de l’investigateur, est susceptible d’avoir une incidence sur les tests fonctionnels ; en outre, les fractures des membres inférieurs doivent être consolidées depuis au moins 3 mois avant la sélection (visite 1).34French (France)13732086Positive test for NAb to AAV9, based on the threshold determined by the Central Laboratory, from a sample taken at Screening (Visit 1)979999776dce107-5c83-4bc5-bf28-e9593c4e4311Résultat positif au test AcN anti-AAV9, d’après le seuil fixé par le laboratoire central, sur un échantillon prélevé lors de la sélection (visite 1).34French (France)979999676dce107-5c83-4bc5-bf28-e9593c4e4311Prueba positiva de AcN para VAA9, según el umbral determinado por el laboratorio central, de una muestra tomada en la Selección (Visita 1).7Spanish13732097Receipt of a live attenuated vaccination within 30 days prior to Screening (Visit 1). Receipt of a live attenuated vaccination will also be prohibited for 90 days before Day 1 (Visit 3), for 90 days prior to Year 2 IP administration, and for the first 2 months after each IP administration.9799998d38eb24d-3f01-461b-8282-b2686ffa9bb1Administración de una vacuna de virus vivos atenuados dentro de los 30 días previos a la Selección (Visita 1). La administración de una vacuna de virus vivos atenuados también estará prohibida durante 90 días antes del Día 1 (Visita 3), durante los 90 días previos a la administración del PEI del Año 2 y los primeros 2 meses tras cada administración del PEI.7Spanish9799999d38eb24d-3f01-461b-8282-b2686ffa9bb1Administration d’un vaccin vivant atténué dans les 30 jours précédant la sélection (visite 1). L’administration d’un vaccin vivant atténué sera également interdite pendant les 90 jours précédant le jour 1 (visite 3), pendant 90 jours avant l’administration du ME de l'année 2 et pendant les 2 premiers mois après chaque administration du ME.34French (France)13732108Abnormality in hematology or chemistry profiles at Screening (Visit 1). A single repeat for value(s) outside allowable limits is permitted to re-assess eligibility: a. Absolute neutrophil count <1000 cells/mm3; b. Platelets <150 x 103/μl; c. Cystatin C >1.2 x ULN; d. Positive hepatitis A virus (anti-HAV) immunoglobulin M, hepatitis B surface antigen (HbsAg), and/or hepatitis C antibody (HCVAb); e. Markers of hepatic inflammation or overt or occult cirrhosis as evidenced by one or more of the following: 1. Prothrombin time (PT) > upper limit of normal (ULN); prolonged international normalized ratio (INR) >ULN; 2. GLDH >2 x ULN; 3. Total bilirubin >1.5 x ULN (unless the participant has a history of Gilbert disease) and direct bilirubin >0.5 mg/dL; 4. Gamma-glutamyl transferase (GGT) >1.5 x ULN.98000013ed641d9-a715-4ff9-95ae-4652309e2daaAnomalie dans des profils hématologiques ou biochimiques à la sélection (visite 1). Une seule répétition d’analyse est permise pour la (les) valeur(s) en dehors des limites autorisées afin de réévaluer l’éligibilité : a. Numération absolue de neutrophiles < 1 000 cellules/mm3 ; b. Plaquettes < 150 x 103/μl ; c. Cystatine C > 1,2 x LSN ; d. Immunoglobulines M anti-virus de l’hépatite A (IgM anti-VHA) positives, antigène de surface de l’hépatite B (AgHBs) et/ou anticorps anti-hépatite C (Ac anti-VHC) ; e. Marqueurs d’inflammation hépatique ou de cirrhose manifeste ou occulte, mise en évidence par un ou plusieurs des paramètres suivants : 1. Temps de prothrombine (PT) > limite supérieure de la normale (LSN) ; rapport normalisé international (INR) prolongé > LSN ; 2. GLDH > 2 x LSN ; 3. Bilirubine totale > 1,5 x LSN (sauf si le patient a des antécédents de maladie de Gilbert) et bilirubine directe > 0,5 mg/dl ; 4. Gamma-glutamyl transférase (GGT) > 1,5 x LSN.34French (France)98000003ed641d9-a715-4ff9-95ae-4652309e2daaAnomalías en los perfiles de hematología o bioquímica en la Selección (Visita 1). Se permite una repetición única para los valores fuera de los límites admisibles a fin de volver a evaluar la elegibilidad: a. recuento absoluto de neutrófilos <1000 células/mm3; b. plaquetas <150 x 103/μl; c. cistatina C >1,2 x LSN; d. inmunoglobulina M contra el virus de la hepatitis A (anti-HAV), antígeno de superficie de la hepatitis B (HbsAg) o anticuerpo de la hepatitis C (HCVAb) positivos; e. marcadores de inflamación hepática o cirrosis evidente u oculta, tal como se demuestra en uno o más de los siguientes: 1. tiempo de protrombina (TP) > límite superior de la normalidad (LSN); índice internacional normalizado (INR) > LSN; 2. GLDH >2 x LSN; 3. bilirrubina total >1,5 x LSN (a menos que el participante tenga antecedentes de síndrome de Gilbert) y bilirrubina directa >0,5 mg/dl; 4. gamma-glutamil transferasa (GGT) >1,5 x LSN.7Spanish13732119Other acute or chronic medical or psychiatric condition at Screening (Visit 1), including recent (within the past year) or active suicidal ideation or behavior (using screening by the Child Behavior Check List (CBCL) and determined by the Investigator, as described in Section 8.2.13) or laboratory abnormality that may increase the risk associated with study participation or IP administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the participant inappropriate for entry into this study.98000024d2d1812-7f20-4886-b751-0f21e09454b2Cualquier otra afección médica o psiquiátrica aguda o crónica en la Selección (Visita 1), incluidas ideas o conductas de suicidio recientes (en el año anterior) (mediante el uso de la selección por parte del inventario del comportamiento de niños [CBCL] y determinadas por el investigador, según lo descrito en el apartado 8.2.11), o anomalías en los análisis clínicos que pudieran aumentar el riesgo asociado a la participación en el estudio o a la administración del PEI, o que pudieran interferir en la interpretación de los resultados del estudio y que, en la opinión del investigador, harían que el participante fuera inapropiado para ingresar en este estudio.7Spanish98000034d2d1812-7f20-4886-b751-0f21e09454b2Autre pathologie médicale ou psychiatrique aiguë ou chronique à la sélection (visite 1), notamment idées ou comportements suicidaires actifs ou récents au cours de l’année passée (avec dépistage à l’aide de l’Échelle du comportement de l’enfant (CBCL) et déterminée par l’investigateur comme décrit à la section 8.2.13), ou anomalies biologiques susceptibles d’accroître les risques associés à la participation à l’étude ou à l’administration du ME, ou susceptibles de nuire à l’interprétation des résultats de l’étude et qui, de l’avis de l’investigateur, rendraient le patient inéligible pour intégrer cette étude.34French (France)9387881Change from Baseline at Week 52 in the NSAA total score.True979995170899929-7419-47f0-8c99-c2fd83533963Variation par rapport à la valeur initiale du score total NSAA à la semaine 52.34French (France)979995070899929-7419-47f0-8c99-c2fd83533963Cambio con respecto al valor de referencia en la Semana 52 en la puntuación total de la NSAA.7Spanish9387891Change from Baseline in percent normal mini-dystrophin expression level in biceps brachii muscle biopsies at Day 360 using a liquid chromatography mass spectrometry (LC-MS) assay.False97999532c26b0ca-e7df-4b14-8740-53bff20bc67fVariation par rapport à la valeur initiale du niveau d'expression normal de la mini-dystrophine en pourcentage dans les biopsies de muscle biceps brachial au jour 360 à l’aide d’un dosage par chromatographie en phase liquide couplée à la spectrométrie de masse (LC-MS).34French (France)97999522c26b0ca-e7df-4b14-8740-53bff20bc67fCambio con respecto al valor de referencia en el porcentaje del nivel de expresión de minidistrofina en biopsias del músculo bíceps braquial en el Día 360 mediante el uso de un ensayo de cromatografía líquida con espectrometría de masa (Liquid Chromatography Mass Spectrometry, LC-MS).7Spanish9387902Change from Baseline in percent of muscle fibers expressing mini-dystrophin in biceps brachii muscle biopsies at Day 360 as assessed by immunofluorescence.False97999545aa556b1-fd9d-496f-9679-2a1d50de6b61Cambio con respecto al valor de referencia en el porcentaje de fibras musculares que expresan minidistrofina en biopsias del músculo bíceps braquial en el Día 360 según la evaluación de inmunofluorescencia.7Spanish97999555aa556b1-fd9d-496f-9679-2a1d50de6b61Variation par rapport à la valeur initiale du pourcentage de fibres musculaires exprimant la mini-dystrophine dans les biopsies de muscle biceps brachial au jour 360, d’après l’évaluation par immunofluorescence.34French (France)9387913Change from Baseline at Week 52 in serum CK concentration.False9799956dab8527e-8d60-482a-a4f8-5728029bf683Variation par rapport à la valeur initiale de la concentration sérique de CK à la semaine 52.34French (France)9799957dab8527e-8d60-482a-a4f8-5728029bf683Cambio con respecto al valor de referencia en la Semana 52 en la concentración de CK sérica.7Spanish9387924Number of skills gained at Week 52 based on the individual items of the NSAA.False979995992f3c413-5120-44fe-8fda-330225d798b5Cantidad de habilidades adquiridas en la Semana 52 según los elementos individuales de la NSAA.7Spanish979995892f3c413-5120-44fe-8fda-330225d798b5Nombre de compétences acquises à la semaine 52 d’après les items individuels de l’échelle NSAA.34French (France)9387935Number of skills either improved or maintained at Week 52 based on the individual items of the NSAA.False9799961cedc92cc-628b-47fe-9337-fdb971553cf6Nombre de compétences améliorées ou maintenues à la semaine 52 d’après les items individuels de l’échelle NSAA.34French (France)9799960cedc92cc-628b-47fe-9337-fdb971553cf6Cantidad de habilidades mejoradas o mantenidas en la Semana 52 según los elementos individuales de la NSAA.7Spanish9387946Change from Baseline at Week 52 in the 10 meter run/walk velocity.False97999635dd32f0c-b37b-4bb9-88d6-51ba6cb9c82bVariation par rapport à la valeur initiale de la vitesse de marche/course sur 10 mètres à la semaine 52.34French (France)97999625dd32f0c-b37b-4bb9-88d6-51ba6cb9c82bCambio con respecto al valor de referencia en la Semana 52 en la velocidad de carrera/caminata de 10 metros.7Spanish9387957Change from Baseline at Week 52 in the rise from floor velocity.False97999642b8c9f40-994a-47a1-ac10-29321fbd942cCambio con respecto al valor de referencia en la Semana 52 en la velocidad de elevación desde el piso.7Spanish97999652b8c9f40-994a-47a1-ac10-29321fbd942cVariation par rapport à la valeur initiale de la vitesse du lever depuis la position couchée à la semaine 52.34French (France)9387968Change from Baseline at Week 52 in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale (Pediatric Parent).False9799966915d8794-2b43-446d-8dd9-03eb129829a2Variation par rapport à la valeur initiale du score de l’instrument de collecte de données sur les résultats pédiatriques (PODCI) modifié, à la semaine 52 : Transfer and Basic Mobility Core Scale (Pediatric Parent) [Échelle générique d’évaluation des transferts et de la mobilité de base (Parent pédiatrique)].34French (France)9799967915d8794-2b43-446d-8dd9-03eb129829a2Cambio con respecto al valor de referencia en la Semana 52 en el instrumento de recopilación de datos de resultados pediátricos (PODCI) modificado: escala principal de transferencia y movilidad básica (padres pediátricos).7Spanish9387979Change from Baseline at Week 52 in the Modified PODCI: Sports and Physical Functioning Core Scale (Pediatric Parent).False9799969031c6ff6-1958-4ac2-87c1-6360708d255bVariation par rapport à la valeur initiale du score PODCI modifié à la semaine 52 : Sports and Physical Functioning Core Scale (Pediatric Parent) [Échelle générique d’évaluation pour les sports et activités physiques (Parent pédiatrique)].34French (France)9799968031c6ff6-1958-4ac2-87c1-6360708d255bCambio con respecto al valor de referencia en la Semana 52 en el PODCI modificado: escala principal de deportes y funcionamiento físico (padres pediátricos).7Spanish2040-01-102039-12-052020-11-0586930Pfizer Inc.32717122327173252532717224242PatientsFalseTrueTrue1Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.acceptable5Diseases [C] - Musculoskeletal Diseases [C05]123960DUCHENNE MUSCULAR DYSTROPHY (DMD)True134892True397613PublicClinical Medical LeadClinicalTrials.gov_Inquiries@pfizer.com+18007181021224122Pharmaceutical company10Pfizer Inc.TrueTrueORG-100004191Active397614ScientificClinical Medical LeadClinicalTrials.gov_Inquiries@pfizer.com+18007181021224122Pharmaceutical company10Pfizer Inc.TrueTrueORG-100004191Active6038739268195409Industry3ICON PLCTrueFalseORL-000000590Active971628282 Halsey Road8282 Halsey RoadWhitesboroNY 13492840United States+13157680838clara.richardson@iconplc.comFalseORL-00000059093919715Anti-drug antibodies (ADA) to mini-dystrophin (anti-transgene protein Ab)During Study Storage and An+13157680838clara.richardson@iconplc.com603879447182479457Non-Pharmaceutical company11ATOM International LimitedTrueTrueORG-100042393Active478985Office 16, Valley House, Seventh Avenue, Team Valley Trading EstateOffice 16Valley HouseSeventh AvenueTeam Valley Trading EstateGatesheadNE11 0JW826United Kingdom+447540051001michelle.eagle@atom-international.orgTrueORG-10004239393920515Training development and delivery - NSAA Rater training+447540051001michelle.eagle@atom-international.org603876432145464357Pharmaceutical company10Cellular Technology Ltd.TrueTrueORG-100046556Active46257220521 Chagrin Boulevard20521 Chagrin BoulevardShaker Heights44122-5350840United States+12163257228ioana.moldovan@immunospot.comTrueORG-10004655693920215BIOANALYTICAL SERVICES - Elispot testing+12163257228ioana.moldovan@immunospot.com603872432136464348Pharmaceutical company10Labcorp Central Laboratory Services LPTrueTrueORG-100032236Active4625638211 Scicor Drive8211 Scicor DriveIndianapolis46214-2942840United States+13172734032David.Root@covance.comTrueORG-10003223693919615CENTRAL LAB SERVICES, BIOANALYTICAL SERVICES+13172734032David.Root@covance.com603878432138464350Pharmaceutical company10Monogram Biosciences Inc.TrueTrueORG-100043273Active462565345 Oyster Point Boulevard345 Oyster Point BoulevardSouth San Francisco94080-1913840United States+16508667482Santoc2@labcorp.comTrueORG-10004327393920415Neutralizing Antibody (NAb) to adenoassociated virus, serotype 9 (AAV9)During Study Storage and Ana+16508667482Santoc2@labcorp.com603877446514478787Pharmaceutical company10Fortrea Inc.TrueTrueORG-100012602Active4782598 Moore Drive8 Moore DriveDurham27709-0009840United States+34936093400Carlos.Vicentegonzalez@fortrea.comTrueORG-10001260293920315Home health+34936093400Carlos.Vicentegonzalez@fortrea.com603874432176464388Pharmaceutical company10Medpace Inc.TrueTrueORG-100026760Active4626045375 Medpace Way5375 Medpace WayCincinnati45227-1543840United States+15135799911v.baker@medpace.comTrueORG-10002676093919815Medical image+15135799911v.baker@medpace.com603875447177479452Pharmaceutical company10Ppd Inc.TrueTrueORG-100018960Active478979929 North Front Street929 North Front StreetWilmington28401-3331840United States+19105582794Margaret.Bockus@ppd.comTrueORG-1000189609391991939200129392015+19105582794Margaret.Bockus@ppd.com224122Pharmaceutical company10Pfizer Inc.TrueTrueORG-100004191Active220421224122Pharmaceutical company10Pfizer Inc.TrueTrueORG-100004191Active23466966 Hudson Boulevard East66 Hudson Boulevard EastNew York10001-2189840United StatesTrueORG-100004191TrueCommercial2This is a Phase 3 Trial10904335Diseases [C] - Musculoskeletal Diseases [C05]369648Fordadistrogene Movaparvovec1Test493958110928501SOLUTION FOR INFUSIONPRD109285011Fordadistrogene MovaparvovecSOLUTION FOR INFUSIONPF-06939926FORDADISTROGENE MOVAPARVOVECSUB193152PFIZER INC.10928501347699PFIZER INC.Structurally Diverse Substance - OtherStructurally Diverse Substance - OtherFORDADISTROGENE MOVAPARVOVECSUB193152Adeno-associated viral vector serotype 9 containing the human mini-dystrophin genePF-06939926BMD-001False1TrueEU/3/16/1716vector genomes (vg)/mL200000000000000vector genomes (vg)/mL20000000000000011Advanced Therapy IMP (ATIMP)PRD10928501PF-0693992662473DMD generecombinant adeno-associated virusFalseTrue3118INTRAVENOUS USEFalseFordadistrogene Movaparvovecfordadistrogene movaparvovecSOLUTION FOR INFUSION369650Placebo for PF-069399263Placebo4939601N/AN/AN/APlacebo for PF-06939926N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePlacebo for PF-06939926n/aN/A369649ECULIZUMAB4Auxiliary4939593SUB25187-2ECULIZUMABCONCENTRATE FOR SOLUTION FOR INFUSIONECULIZUMABSUB2518725187-ECULIZUMABSUB25187---False4TrueEU/3/09/653mg milligram(s)1200mg milligram(s)1200False11SUB251877SOLUTION FOR INFUSIONFalseECULIZUMABeculizumabFalse273688298672986790352012Authorised2024-04-262024-04-26FalseunwillingFrance2024-08-21Authorised330992022-09-152024-09-04331002022-09-152024-09-04596132025-03-272025-06-27596112025-03-272025-06-27596122025-03-272025-06-27424732023-01-242024-05-032024-09-04424742023-01-242024-05-032024-09-04424712023-01-242024-09-04424722023-01-242024-09-04True440482022-09-152024-09-042023-01-242024-05-03False5089129867Halted2024-09-04T09:53:34.9314062829867Under evaluation2024-06-26T10:49:58.8264899429867Authorised2024-08-21T10:43:33.7088629029867Authorised2025-06-27T16:26:39.8261France2025-03-27authorized_conditions2024-08-212024-08-2151876626434896467113Hospital/Clinic/Other health care facility8Centre Hospitalier Universitaire De NantesFalseTrueORG-100007295Active4655161 Place Alexis Ricordeau1 Place Alexis RicordeauNantes440002012France+33240083704yann.pereon@univ-nantes.frTrueORG-1000072952085252YannPereon+33240083704yann.pereon@univ-nantes.frN/A1876627434900467118Hospital/Clinic/Other health care facility8Hopital Necker Enfants MaladesFalseTrueORG-100023257Active465520149 Rue De Sevres149 Rue De SevresParis750152012France+33144494856isabelle.desguerre@aphp.frTrueORG-1000232572085253IsabelleDesguerre+33144494856isabelle.desguerre@aphp.frN/AAuthorised273686298662986690352002Authorised2024-04-262024-04-26FalsewillingBelgium2024-07-12Authorised268442021-12-132024-07-18268452021-12-132024-07-18596142025-03-242025-06-27596152025-03-242025-06-27346882021-12-152024-05-032024-07-23346892021-12-152024-05-032024-07-23337792021-12-152024-07-18337802021-12-152024-07-18True355152021-12-132024-07-182021-12-152024-05-03True4062729866Under evaluation2024-06-26T10:49:58.7854496729866Halted2024-07-23T14:49:26.3344316029866Authorised2024-07-12T15:29:14.3948629129866Authorised2025-06-27T16:27:49.2541Belgium2025-03-24authorized_conditions2024-07-122024-07-1251876622449347481637Hospital/Clinic/Other health care facility8Universitair Ziekenhuis GentFalseTrueORG-100021542Active481396Corneel Heymanslaan 10Corneel Heymanslaan 10Gent90002002Belgium+3293321954nicolas.deconinck@huderf.beTrueORG-1000215422085248NicolasDeconinck+3293321954nicolas.deconinck@huderf.beN/A1876621434890467107Hospital/Clinic/Other health care facility8UZ LeuvenFalseTrueORG-100006001Active465510Herestraat 49Herestraat 49Leuven30002002Belgium+3216343845liesbeth.dewaele@uzleuven.beTrueORG-1000060012085247Liesbeth Monique HugoDe Waele+3216343845liesbeth.dewaele@uzleuven.beN/AAuthorised273687298702987090352027Authorised2024-04-262024-04-26TrueunwillingSpain2024-07-15Authorised268402020-11-032024-07-18268412020-11-032024-07-18597942025-03-262025-07-01597952025-03-262025-07-01337772020-11-052024-07-18337782020-11-052024-07-18346862020-11-052024-05-032024-07-23346872020-11-052024-05-032024-07-23True355112020-11-032024-07-182020-11-052024-05-03False4063129870Under evaluation2024-06-26T10:49:58.9134496529870Halted2024-07-23T14:44:10.4064329829870Authorised2024-07-15T11:46:13.7588656029870Authorised2025-07-01T11:59:10.0651Spain2025-03-26authorized_conditions2024-07-152024-07-15211876624434911467128Hospital/Clinic/Other health care facility8Sant Joan De Deu Barcelona HospitalFalseTrueORG-100023083Active465531Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain+34936009733andres.nascimento@sjd.esTrueORG-1000230832085250AndrésNascimento+34936009733andres.nascimento@sjd.esN/A1876623434912467129Hospital/Clinic/Other health care facility8Hospital Universitari Vall D HebronFalseTrueORG-100030781Active465532Passeig De La Vall D'Hebron 119-129Passeig De La Vall D'Hebron 119-129Barcelona080352027Spain+34934894062francina.munell@vhir.orgTrueORG-1000307812085249FrancinaMunell Casadesús+34934894062francina.munell@vhir.orgN/A1876625434910467127Hospital/Clinic/Other health care facility8Hospital Universitario Y Politecnico La FeFalseTrueORG-100029610Active465530Avenida De Fernando Abril Martorell 106Avenida De Fernando Abril Martorell 106Valencia460262027Spain+34961244153nuriamugo@gmail.comTrueORG-1000296102085251NuriaMuelas Gomez+34961244153nuriamugo@gmail.comN/AAuthorised273717298692986990352018Authorised2024-04-262024-04-26FalseunwillingItaly2024-08-12Authorised313622020-12-182024-08-20313632020-12-182024-08-20599142025-03-272025-07-02599152025-03-272025-07-02400532020-12-212024-05-032024-08-20400542020-12-212024-05-032024-08-20400512020-12-212024-08-20400522020-12-212024-08-20True416632020-12-182024-08-202020-12-212024-05-03False4063029869Under evaluation2024-06-26T10:49:58.8864793629869Authorised2024-08-12T23:59:594885129869Halted2024-08-20T14:05:00.9228668129869Authorised2025-07-02T13:09:53.6151Italy2025-03-27acceptable2026-02-17authorized_conditions2024-08-122024-08-12131876812434908467125Hospital/Clinic/Other health care facility8Ospedale Pediatrico Bambino GesuFalseTrueORG-100009738Active465528Piazza Di Sant'Onofrio 4Piazza Di Sant'Onofrio 4Rome001652018Italy+390668592105Adele2.damico@opbg.netTrueORG-1000097382085461AdeleD' Amico+390668592105Adele2.damico@opbg.net1N/A1876811434909467126Hospital/Clinic/Other health care facility8Fondazione Policlinico Universitario Agostino Gemelli IRCCSFalseTrueORG-100014358Active465529Largo Francesco Vito 1Largo Francesco Vito 1Rome001682018Italy+390630154459eugeniomaria.mercuri@policlinicogemelli.itTrueORG-1000143582085460Eugenio MariaMercuri+390630154459eugeniomaria.mercuri@policlinicogemelli.itN/AAuthorised282774298682986890352013Authorised2024-04-262024-04-26FalseunwillingGermany2024-07-10Authorised268382023-02-242024-07-18268392023-02-242024-07-18597922025-03-282025-07-01597932025-03-282025-07-01337712023-03-022024-07-18337722023-03-022024-07-18346842023-03-022024-05-032024-07-23346852023-03-022024-05-032024-07-23True355042023-02-242024-07-182023-03-022024-05-03False4062929868Under evaluation2024-06-26T10:49:58.8574496429868Halted2024-07-23T14:41:22.1544265629868Authorised2024-07-10T08:21:38.5098655929868Authorised2025-07-01T11:58:16.9561Germany2025-03-28authorized_conditions2024-07-102024-07-1041945954434906467123Hospital/Clinic/Other health care facility8Universitaetsklinikum Essen AöRFalseTrueORG-100009964Active465526Hufelandstrasse 55, HolsterhausenHufelandstrasse 55HolsterhausenEssen451472013Germany00492017232508heike.koelbel@uk-essen.deTrueORG-1000099642161450HeikeKölbel00492017232508heike.koelbel@uk-essen.deN/AAuthorised13343INITIALAuthorised2023-508510-42-00Authorised2024-06-26acceptable_conditions2024-07-08944372986629866Belgium2002acceptable2024-07-12authorized_conditions2024-07-12AuthorisedBelgiumAuthorised2024-07-12Authorised944382986729867France2012acceptable2024-08-18authorized_conditions2024-08-21AuthorisedFranceAuthorised2024-08-21Authorised944392986829868Germany2013acceptable2024-07-08authorized_conditions2024-07-10AuthorisedGermanyAuthorised2024-07-10Authorised944402986929869Italy2018acceptable2024-08-05authorized_conditions2024-08-12AuthorisedItalyAuthorised2024-08-12Authorised944412987029870Spain2027acceptable2024-07-15authorized_conditions2024-07-15AuthorisedSpainAuthorised2024-07-15Authorised2024-07-1029866BelgiumAuthorised29867FranceAuthorised29868GermanyAuthorised29869ItalyAuthorised29870SpainAuthorisedINFalse331861334329869Italy2024-08-12T00:00:00authorized_conditionsacceptabledecision9444019190INITIALFalse284431334329868Germany2024-07-10T08:21:39.002authorized_conditionsacceptabledecision9443919190INITIALFalse288601334329866Belgium2024-07-12T15:29:14.851authorized_conditionsacceptabledecision9443719190INITIALTrue342231334329867France2024-08-21T10:43:34.389authorized_conditionsacceptabledecision9443819190INITIALFalse289801334329870Spain2024-07-15T11:46:14.457authorized_conditionsacceptabledecision9444119190INITIALFalse39179SUBSTANTIAL 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MODIFICATIONFalse1108927771429868Germany2026-02-20T20:05:43.082authorizeddecision119140NON SUBSTANTIAL MODIFICATIONFalseItaly298692024-08-12T00:00:002026-02-20T09:15:15.2795Germany298682024-07-10T08:21:39.0022026-02-20T13:13:22.575Belgium298662024-07-12T15:29:14.8512026-01-20T15:02:12.9445France298672024-08-21T10:43:34.3892026-01-20T15:02:12.9445Spain298702024-07-15T11:46:14.4572026-01-20T15:02:12.9445True2019-002921-3129867TH-440502024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-09-04T09:53:34Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29867FranceFR29868TH-363632024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-07-23T14:41:21Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29868GermanyDE29870TH-363652024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-07-23T14:44:10Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29870SpainES29866TH-363672024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-07-23T14:49:26Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29866BelgiumBE29869TH-416652024-05-034Safety related (clinical or pre-clinical results)FalseFalseTrueOn 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, Pfizer implemented a pause to the dosing of participants in Study C3391003 while the event is investigated. The External Data Monitoring Committee has been notified of this event and agrees with the decision to pause dosing.On 03 May 2024, a fatal SAE of cardiac arrest was reported for a participant in the Phase 2 open-label Study C3391008 (being conducted in the US and Australia). On 03 May 2024, the Sponsor implemented a pause to the dosing of participants in Study C3391003 while the event is investigated.True2024-08-20T14:05:00Subsequent to the temporary halt, the Study C3391003 Primary Analysis was conducted, and the study failed to meet its primary objective of demonstrating that fordadistrogene movaparvovec is superior to placebo based on change from baseline in the North Star Ambulatory Assessment total score at Week 52. Considering the negative efficacy results of Study C3391003, Pfizer has concluded that the potential benefits of fordadistrogene movaparvovec do not outweigh the potential risks of treatment. Therefore, no additional study participants will receive fordadistrogene movaparvovec. All dosed study participants will be followed up for safety. Pfizer is in the process of amending the study protocol to implement this decision. The protocol amendment will be submitted to CTIS for review in due course.False29869ItalyIT29866BelgiumRESTART_OF_TRIAL2025-03-24START_OF_TRIAL2021-12-13START_OF_RECRUITMENT2021-12-15END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-0329867FranceRESTART_OF_TRIAL2025-03-27START_OF_TRIAL2022-09-15START_OF_RECRUITMENT2023-01-24END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-0329868GermanyRESTART_OF_TRIAL2025-03-28START_OF_TRIAL2023-02-24START_OF_RECRUITMENT2023-03-02END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-0329869ItalyRESTART_OF_TRIAL2025-03-27START_OF_TRIAL2020-12-18START_OF_RECRUITMENT2020-12-21END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-0329870SpainRESTART_OF_TRIAL2025-03-26START_OF_TRIAL2020-11-03START_OF_RECRUITMENT2020-11-05END_OF_RECRUITMENT2024-05-03TEMPORARY_HALT2024-05-03C3391003_PH file_SM1_Recruitment completedff28131f-62ac-4e71-85ad-62a560f8e5c714Recruitment arrangements (for publication)PDF273688N/A2L1_ICD 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C3391003 2023-508510-42-00 ES Public7b18f4d3-5459-4dc8-94d8-233d643775fa7Synopsis of the protocol (for publication)PDF119140Am161D2 Protocol Synopsis C3391003 2023-508510-42-00 FR Public75b3568b-9821-4444-bc06-e5fa0dea90c87Synopsis of the protocol (for publication)PDF119140Am161D2 Protocol Synopsis C3391003 2023-508510-42-00 IT Public533a88eb-f36a-4812-81e8-2a53e659aae17Synopsis of the protocol (for publication)PDF119140Am161D2 Protocol Synopsis C3391003 2023-508510-42-00 DE Publicfae470b4-8b0a-4f27-8d16-e5434302c1e87Synopsis of the protocol (for publication)PDF119140Am 161Both3 2023-510351-31-005AuthorisedA Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingDYNE251-DMD-201Duchenne muscular dystrophySpain:5Italy:5Ireland:5Belgium:509/07/2024BE: 16/07/2024, IT: 16/07/2024, ES: 09/07/2024, IE: 09/07/2024Diseases [C] - Musculoskeletal Diseases [C05]Dyne Therapeutics Inc.Pharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansMuscle Tissue Endpoints: Cohorts dosed Q4W or Q8W with a second biopsy at Week 25, Change from Baseline at Week 25: -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue, Change from baseline up to Week 145: -blood CK levels, Cohorts dosed Q8W with a second biopsy at Week 49 Change from Baseline at Week 49 in: -dystrophin protein levels in muscle tissue -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue, Change from Baseline up to Week 145 in: -blood CK level, Functional Endpoints: All cohorts, Change from Baseline up to Week 145 in: -PUL scale V 2.0 score -%pFVC -NSAA total score, TTR, 10MRW, and SV95C in ambulatory participants, Plasma Endpoints: All cohorts -Cmax -tmax -AUCtlast -AUC∞ -λZ -t½ -CL -Vz and Vss, 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publication)PDF1135291.01Spain340912024-07-09T13:22:34.6962025-06-06T09:57:39.0465Italy339842024-07-16T00:00:002025-05-21T10:26:06.8745Ireland340902024-07-09T10:57:59.4742025-06-16T09:54:35.2915Belgium340892024-07-16T13:26:52.5032025-05-23T11:30:19.246533984ItalySTART_OF_TRIAL2023-08-09START_OF_RECRUITMENT2023-08-31END_OF_RECRUITMENT2025-03-0334089BelgiumSTART_OF_TRIAL2023-01-06START_OF_RECRUITMENT2023-01-30END_OF_RECRUITMENT2025-03-0334090IrelandSTART_OF_TRIAL2024-06-28START_OF_RECRUITMENT2024-09-24END_OF_RECRUITMENT2025-03-0334091SpainSTART_OF_TRIAL2023-01-24START_OF_RECRUITMENT2023-02-13END_OF_RECRUITMENT2025-03-032023-510351-31-005A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingDYNE251-DMD-20106/01/2023Duchenne muscular dystrophySpain:5Italy:5Ireland:5Belgium:509/07/2024BE: 16/07/2024, IT: 16/07/2024, ES: 09/07/2024, IE: 09/07/2024Diseases [C] - Musculoskeletal Diseases [C05]Dyne Therapeutics Inc.Pharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansMuscle Tissue Endpoints: Cohorts dosed Q4W or Q8W with a second biopsy at Week 25, Change from Baseline at Week 25: -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue, Change from baseline up to Week 145: -blood CK levels, Cohorts dosed Q8W with a second biopsy at Week 49 Change from Baseline at Week 49 in: -dystrophin protein levels in muscle tissue -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue, Change from Baseline up to Week 145 in: -blood CK level, Functional Endpoints: All cohorts, Change from Baseline up to Week 145 in: -PUL scale V 2.0 score -%pFVC -NSAA total score, TTR, 10MRW, and SV95C in ambulatory participants, Plasma Endpoints: All cohorts -Cmax -tmax -AUCtlast -AUC∞ -λZ -t½ -CL -Vz and Vss, if appropriate, Immunogenicity Endpoint: All cohorts -Incidence of ADAsThe placebo is commercially available 5% dextrose solution intended for IV, DYNE-251, DYNE-2510-17 yearsMale316Number and proportion of participants with treatment-emergent adverse events (TEAEs), treatment emergent serious adverse events (TESAEs), TEAEs considered related to study drug, and TEAEs leading to discontinuation from study drug and discontinuation from the study., Change from Baseline in dystrophin protein levels in muscle tissue as determined by Western blot analysis at Week 25No17/04/202618/04/20262023-510351-31-00Authorised2023-01-062024-07-09T10:57:59.4742026-04-18T03:43:25.028317976511352959100000000557United States840USUSATrue100000000329Australia36AUAUSTrue100000000354Canada124CACANTrue100000000556United Kingdom826GBGBRTrue100000000439Korea, Republic of410KRKORTrue4709711N/AN/AN/AThe placebo is commercially available 5% dextrose solution intended for IVN/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse3N/AFalseThe placebo is commercially available 5% dextrose solution intended for IVn/aN/A470970111189307INFUSIONPRD111893071DYNE-251INFUSIONDYNE-251FRAGMENT ANTIBODY TARGETING HUMAN TFR1 CONJUGATED TO PHOSPHORODIAMIDATE MORPHOLINO OLIGOMERSUB273613DYNE THERAPEUTICS, INC11189307305762DYNE THERAPEUTICS, INCProtein - OtherProtein - OtherFRAGMENT ANTIBODY TARGETING HUMAN TFR1 CONJUGATED TO PHOSPHORODIAMIDATE MORPHOLINO OLIGOMERSUB273613False1Falsebiological / biotechnological origin (other than Advanced Therapy IMP (ATIMP)PRD11189307DYNE-2512037711INTRAVENOUS USEFalseDYNE-251fragment antibody targeting human tfr1 conjugated to phosphorodiamidate morpholino oligomerINFUSION470969111189306INFUSIONPRD101597291DYNE-251INFUSIONDYNE-251FRAGMENT ANTIBODY TARGETING HUMAN TFR1 CONJUGATED TO PHOSPHORODIAMIDATE MORPHOLINO OLIGOMERSUB273613DYNE THERAPEUTICS, INC11189306305762DYNE THERAPEUTICS, INCProtein - OtherProtein - OtherFRAGMENT ANTIBODY TARGETING HUMAN TFR1 CONJUGATED TO PHOSPHORODIAMIDATE MORPHOLINO OLIGOMERSUB273613False1Falsebiological / biotechnological origin (other than Advanced Therapy IMP (ATIMP)PRD10159729DYNE-2512037711INTRAVENOUS USEFalseDYNE-251fragment antibody targeting human tfr1 conjugated to phosphorodiamidate morpholino oligomerINFUSIONA Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping92584366ff91bcb-3102-4688-afc5-049a89cf629eEstudio aleatorizado, doble ciego, controlado con placebo, de dosis escalonadas múltiples para evaluar la seguridad, la tolerabilidad, la farmacodinámica, la eficacia y la farmacocinética de DYNE-251 administrado a pacientes con distrofia muscular de Duchenne susceptibles de omisión del exón 51.7SpanishStudy of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping9258439d92109a3-2660-4d99-a190-d9179e68ad58Estudio de DYNE-251 en pacientes con distrofia muscular de Duchenne susceptibles de omisión del exón 51.7SpanishDYNE251-DMD-201475011NCT05524883False72This is a Phase 1/2103815117880Duchenne muscular dystrophy9258423a03c4897-0188-4a46-aa73-8ca53dde703bDistrofia muscular de Duchenne7SpanishTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False93434027343404534340343434056343401"To evaluate the safety and tolerability of multiple IV doses of DYNE-251 administered to participants with DMD To evaluate dystrophin protein levels in muscle tissue following multiple IV doses of DYNE-251 administered to participants with DMD "92584383f1d5ea5-d208-4d30-809d-48e08c3c11f6Evaluar la seguridad y la tolerabilidad de dosis IV múltiples de DYNE-251 administradas a pacientes con DMD. Evaluar las concentraciones de proteína distrofina en el tejido muscular después de administrar varias dosis IV de DYNE-251 a pacientes con DMD.7Spanish3976231To evaluate the effects on muscle tissue exon skipping, percent dystrophin-positive fibers (PDPFs), and blood creatine kinase (CK) following multiple IV doses of DYNE-251 administered to participants with DMD9258440872f2361-a379-41c9-bcc5-44215bf8c8ddEvaluar los efectos sobre la omisión de exones en el tejido muscular, el porcentaje de fibras positivas para la distrofina (PDPF, percent dystrophin-positive fibers) y la creatina cinasa (CK) en sangre tras la administración de dosis IV múltiples de DYNE-251 a pacientes con DMD.7Spanish3976242To evaluate muscle function following multiple IV doses of DYNE-251 administered to participants with DMD9258441ae8e6d05-c521-4691-96d2-d6c31ec54af6Evaluar la función muscular tras la administración de dosis IV múltiples de DYNE-251 a pacientes con DMD.7Spanish3976253To evaluate plasma and muscle tissue PK following multiple IV doses of DYNE-251 administered to participants with DMD92584421d2ae008-3adf-4f30-96ca-88a22953b209Evaluar la farmacocinética en plasma y tejido muscular tras la administración de dosis IV múltiples de DYNE 251 a pacientes con DMD.7Spanish3976264To evaluate the immunogenicity of multiple IV doses of DYNE-251 administered to participants with DMD925844336088d5a-55de-4d4e-a05b-636b31b3cb04Evaluar la inmunogenicidad de dosis IV múltiples de DYNE-251 administradas a pacientes con DMD.7Spanish7612831• Age 4 to 16 years inclusive, at the time of informed consent/assent. • Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping. • Upper extremity muscle group that is amenable to muscle biopsy. • Brooke Upper Extremity Scale score of 1 or 2. • Ambulatory or non-ambulatory. A non-ambulatory participant must have been non-ambulatory for <2 years before enrolment. • Receiving a stable dosage of glucocorticoids for at least 12 weeks prior to the start of study drug administration. • Left ventricular ejection fraction of ≥50% by echocardiogram or ≥ 55% by cardiac magnetic resonance imaging (MRI).92584370296429d-5d3f-4938-a6db-4bdf57c5351b• Edad de 4 a 16 años, inclusive, en el momento de otorgar el asentimiento/consentimiento informado. • Varón con un diagnóstico confirmado de DMD y con una mutación en el gen de la distrofina caracterizada por deleción del exón susceptible de omisión del exón 51. • Grupo muscular de las extremidades superiores susceptible de biopsia muscular. • Puntuación de 1 o 2 en la escala de Brooke para las extremidades superiores. • Paciente deambulante o no deambulante. Los participantes no deambulantes deben haberlo sido durante <2 años antes de la inclusión en el estudio. • Haber recibido una pauta de tratamiento estable de glucocorticoides durante al menos 12 semanas antes del inicio de la administración del fármaco del estudio. • Fracción de eyección del ventrículo izquierdo ≥50 % determinada mediante ecocardiograma o ≥55 % determinada mediante resonancia magnética nuclear (RMN) cardíaca.7Spanish13043671• Uncontrolled clinical symptoms and signs of congestive heart failure (CHF). • Any change in prophylaxis/treatment for CHF within 3 months prior to the start of study treatment. • History of major surgical procedure within 12 weeks prior to the start of study drug administration or an expectation of a major surgical procedure during the study. • Requirement of daytime ventilator assistance. • Percent predicted FVC <40 % (applies only for participants who are age ≥7 years). • Receipt of eteplirsen, or alternative exon-skipping/dystrophinmodifying therapy, within 12 weeks of randomization. • Receipt of non-exon skipping investigational drug within 4 months before the start of study drug administration. • Receipt of gene therapy at any time.925843551c91ff3-ad8c-4db6-a568-ef57d09318d6• Signos y síntomas clínicos no controlados de insuficiencia cardíaca congestiva (ICC). • Cualquier cambio en la profilaxis/el tratamiento para la ICC en los 3 meses anteriores al inicio del tratamiento del estudio. • Antecedentes de intervención quirúrgica mayor en las 12 semanas anteriores al inicio de la administración del fármaco del estudio o expectativa de que se tenga que practicar una intervención quirúrgica mayor durante el estudio. • Necesidad de asistencia respiratoria diurna. • Porcentaje de FVC previsto <40 % (se aplica solo a los participantes con una edad ≥7 años). • Haber recibido eteplirsén o un tratamiento alternativo de omisión de exón/modificador de la distrofina en las 12 semanas anteriores a la aleatorización. • Haber recibido un fármaco en investigación no dirigido a la omisión del exón en los 4 meses anteriores al inicio de la administración del fármaco del estudio. • Recibir terapia génica en cualquier momento.7Spanish8915801Number and proportion of participants with treatment-emergent adverse events (TEAEs), treatment emergent serious adverse events (TESAEs), TEAEs considered related to study drug, and TEAEs leading to discontinuation from study drug and discontinuation from the study.True9258424ff34bb78-d8f8-40d0-8468-5d39472b1b10Número y proporción de pacientes con acontecimientos adversos aparecidos durante el tratamiento (AAAT), acontecimientos adversos graves aparecidos durante el tratamiento (AAGAT), AAAT que se consideren relacionados con el fármaco del estudio y AAAT que motiven la suspensión del fármaco del estudio y la retirada del estudio.7Spanish8915812Change from Baseline in dystrophin protein levels in muscle tissue as determined by Western blot analysis at Week 25True9258425b43d6776-ea3a-4a17-82ed-d96b55c1d76aVariación de la concentración de proteína distrofina en el tejido muscular, determinada mediante inmunotransferencia, entre el momento basal y la semana 25.7Spanish8915821Muscle Tissue Endpoints: Cohorts dosed Q4W or Q8W with a second biopsy at Week 25False925842602d25803-b8ec-4d46-ab38-d1b0e0837259Criterios de valoración relacionados con el tejido muscular: Cohortes que recibieron dosis C4S o C8S con una segunda biopsia en la semana 257Spanish8915832Change from Baseline at Week 25: -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissueFalse9258427f8050d22-3db6-4dcc-b4c4-abe812971ecbVariación con respecto al momento basal en la semana 25: - Niveles de omisión del exón 51 en el tejido muscular - PFPD en el tejido muscular - Concentración de PMO en el tejido muscular7Spanish8915843Change from baseline up to Week 145: -blood CK levelsFalse9258428f64142c3-cf7f-44a3-8f26-c6281c8d8d2dVariación con respecto al momento basal en la semana 145: - Niveles de creatinina quinasa en sangre7Spanish8915854Cohorts dosed Q8W with a second biopsy at Week 49 Change from Baseline at Week 49 in: -dystrophin protein levels in muscle tissue -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissueFalse9258429a344cae1-f848-4918-8729-1d05c6bca3e6Cohortes que recibieron dosis C8S con una segunda biopsia en la semana 49 Variación con respecto al momento basal en la semana 49: - Niveles de proteína distrofina en el tejido muscular - Niveles de omisión del exón 51 en el tejido muscular - PFPD en el tejido muscular - Concentración de PMO en el tejido muscular7Spanish8915865Change from Baseline up to Week 145 in: -blood CK levelFalse9258430939ca358-a2c1-4e8c-b200-3a852a8fccd7Variación con respecto al momento basal en la semana 145: - Niveles de creatinina quinasa en sangre7Spanish8915876Functional Endpoints: All cohortsFalse9258431900ede9a-0f2a-47ce-ae28-199edd1d3b65Criterios de valoración funcionales: Todas las cohortes7Spanish8915887Change from Baseline up to Week 145 in: -PUL scale V 2.0 score -%pFVC -NSAA total score, TTR, 10MRW, and SV95C in ambulatory participantsFalse9258432af932d13-19ad-4412-bd99-ebc91d8a760eVariación con respecto al momento basal en la semana 145: -Puntuación en la versión 2.0 de la escala PUL - Porcentaje de FVCp -Puntuación total de la NSAA, TTR, 10MRW y SV95C en participantes deambulantes7Spanish8915898Plasma Endpoints: All cohorts -Cmax -tmax -AUCtlast -AUC∞ -λZ -t½ -CL -Vz and Vss, if appropriateFalse9258433aded5373-ce4f-4c6b-9b72-058a2e61b9eaCriterios de valoración plasmáticos: Todas las cohortes – Cmáx. – tmáx. - AUCtúlt. - AUC∞ - λZ -t½ - CL - Vz y Vss, si procede7Spanish8915909Immunogenicity Endpoint: All cohorts -Incidence of ADAsFalse9258434832b9b85-561c-4df2-9f47-4908b1876f3aCriterio de valoración relacionado con la inmunogenicidad: Todas las cohortes - 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ServicesAuthorised20709INITIALAuthorised2024-512101-60-00Ended2024-03-14acceptable2024-04-30899862601726017Germany2013acceptable2024-05-03authorized2024-05-06EndedGermanyEnded2024-05-06Authorised2024-05-0626017GermanyEndedINFalse206122070926017Germany2024-05-06T10:36:46.073authorizedacceptabledecision8998637602INITIALTrueGermany260172024-05-06T10:36:46.0732024-05-06T10:36:46.0738False26017GermanySTART_OF_TRIAL2024-06-05END_OF_TRIAL2024-07-06START_OF_RECRUITMENT2024-06-05END_OF_RECRUITMENT2024-06-26EEA1 2023-509935-23-008EndedA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment5051-201Duchenne Muscular DystrophyNetherlands:8Spain:8Belgium:8Germany:8Italy:806/05/2024DE: 08/05/2024, NL: 06/05/2024, BE: 07/05/2024, IT: 13/05/2024, ES: 06/05/2024Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Diseases [C] - Musculoskeletal Diseases [C05]Sarepta Therapeutics Inc.Pharmaceutical companyTherapeutic exploratory (Phase II)Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen, Pre-dose and at multiple time points (up to 32 hours) after end of infusion, Part A: PK: Urine Concentration of Vesleteplirsen, Pre-dose and at multiple time periods (up to 48 hours) after end of infusion, Part B: Change From Baseline in Exon-Skipping Levels at Week 28, Part B: Incidence of Adverse Events (AEs), Baseline up to Week 304, Part B: PK: Plasma Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: PK: Urine Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28VESLETEPLIRSEN(SRP-5051)0-17 years, 18-64 yearsMale3Both21Part A: 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results (for publication)PDF21001.01D1_Sarepta_SRP-5051-201_Layperson Summary_2023-509935-23-00_DE_de_Publicdfde53f8-057a-4c0b-aefc-bf12aba02eb546Laypersons summary of results (for publication)PDF21001.01Netherlands270012024-05-06T12:07:17.8642024-10-02T12:59:38.4828Spain267962024-05-06T09:31:43.3072024-10-07T08:00:46.9828Belgium269142024-05-07T13:39:17.1412024-10-02T00:00:008Germany267952024-05-08T09:41:22.2382024-10-01T12:35:38.3248Italy269502024-05-13T00:00:002024-10-01T23:06:04.146826795GermanySTART_OF_TRIAL2022-06-20START_OF_RECRUITMENT2022-07-18END_OF_RECRUITMENT2024-03-30EARLY_TERMINATION2024-11-066Safety Related (Clinical or Pre-Clinical Results)False26796SpainSTART_OF_TRIAL2020-09-10START_OF_RECRUITMENT2020-09-17END_OF_RECRUITMENT2024-03-30EARLY_TERMINATION2024-11-066Safety Related (Clinical or Pre-Clinical Results)False26914BelgiumSTART_OF_TRIAL2020-08-21START_OF_RECRUITMENT2020-08-26END_OF_RECRUITMENT2024-03-30EARLY_TERMINATION2024-11-066Safety Related (Clinical or Pre-Clinical Results)False26950ItalySTART_OF_TRIAL2022-07-11START_OF_RECRUITMENT2022-07-13END_OF_RECRUITMENT2024-03-30EARLY_TERMINATION2024-11-066Safety Related (Clinical or Pre-Clinical Results)False27001NetherlandsSTART_OF_TRIAL2022-07-01START_OF_RECRUITMENT2022-07-06END_OF_RECRUITMENT2024-03-30EARLY_TERMINATION2024-11-066Safety Related (Clinical or Pre-Clinical Results)False2025-02-0720952023-509935-23-00B1_Sarepta_SRP 5051-201_CSR SummarySubmittedSummary of ResultsFinalTrue952522025-08-27T06:52:27.7160172025-08-26T06:38:46.1032025-08-27T06:53:0210602SUM-9524621002023-509935-23-00D1_Sarepta_SRP-5051-201_Layperson Summary_2023-509935-23-00SubmittedLaypersons Summary of ResultsFinalTrue952622025-08-27T06:52:45.8308062025-08-26T06:46:49.9272025-08-27T06:53:2010602SUM-952552023-509935-23-008A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment5051-20121/08/202006/11/2024Duchenne Muscular DystrophyNetherlands:8Spain:8Belgium:8Germany:8Italy:806/05/2024DE: 08/05/2024, NL: 06/05/2024, BE: 07/05/2024, IT: 13/05/2024, ES: 06/05/2024Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Diseases [C] - Musculoskeletal Diseases [C05]Sarepta Therapeutics Inc.Pharmaceutical companyTherapeutic exploratory (Phase II)Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen, Pre-dose and at multiple time points (up to 32 hours) after end of infusion, Part A: PK: Urine Concentration of Vesleteplirsen, Pre-dose and at multiple time periods (up to 48 hours) after end of infusion, Part B: Change From Baseline in Exon-Skipping Levels at Week 28, Part B: Incidence of Adverse Events (AEs), Baseline up to Week 304, Part B: PK: Plasma Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: PK: Urine Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28VESLETEPLIRSEN(SRP-5051)0-17 years, 18-64 yearsMale32107/02/2025Part A: Incidence of Adverse Events (AEs), Baseline up to 75 weeks, Part B: Change From Baseline in Dystrophin Protein Level at Week 28Yes07/10/202403/09/20252023-509935-23-00Ended2020-08-212024-11-062024-05-06T09:31:43.3072025-09-03T03:35:32.76006463485363740100000000354Canada124CACANTrue100000000557United States840USUSATrue100000000556United Kingdom826GBGBRTrue215976110281392VIAL FOR INTRAVENOUS USEPRD94569401VESLETEPLIRSEN(SRP-5051)VIAL FOR INTRAVENOUS USESRP-5051VESLETEPLIRSENSUB221175VESLETEPLIRSENSAREPTA THERAPEUTICS INC10281392282173SAREPTA THERAPEUTICS INCNucleic AcidNucleic AcidVESLETEPLIRSENSUB221175False1Falsemg/kg milligram(s)/kilogram0mg/kg milligram(s)/kilogram03002ChemicalPRD9456940SRP-5051DE_BB_01_MIA_2024_0015; IMP11566/0000111INTRAVENOUS USEFalseVESLETEPLIRSEN(SRP-5051)vesleteplirsenVIAL FOR INTRAVENOUS USEA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment36970132552076e-0e7a-48bc-9488-86a6b3fa2ae1Estudio de fase 2, de dos partes, de dosis múltiple ascendente, para la determinación de la dosis de SRP-5051, seguido de ampliación de la dosis, en pacientes con distrofia muscular de Duchenne susceptible al tratamiento de omisión del exón 517Spanish36970142552076e-0e7a-48bc-9488-86a6b3fa2ae1Een fase 2-, tweedelig onderzoek met meerdere oplopende doses SRP-5051 voor dosisbepaling en vervolgens dosisuitbreiding, bij patiënten met Duchenne spierdystrofie die vatbaar zijn voor behandeling met het overslaan van exon 5137Dutch (Netherlands)Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)36970215ed65853-e6ac-495b-9f94-d6ad60017a8cEstudio de dos partes para la determinación de la dosis de vesleteplirsén (SRP 5051) (parte A) y, a continuación, de la eficacia de la dosis (parte B) en participantes con distrofia muscular de Duchenne susceptible al tratamiento de omisión del exón 51 (MOMENTUM)7Spanish36970225ed65853-e6ac-495b-9f94-d6ad60017a8cTweedelig onderzoek voor dosisbepaling van vesleteplirsen (SRP-5051) (deel A), vervolgens werkzaamheid van de dosis (deel B) bij deelnemers met Duchenne spierdystrofie die vatbaar zijn voor behandeling met het overslaan van exon 51 (MOMENTUM)37Dutch (Netherlands)5051-201265532NCT04004065False42According to the "Appendix on transparency rules to the functional specifications of the EU clinical trials portal and database" chapter 4.3.3, Category 2 trials are safety and efficacy trials in patients. The current study meets this requirement, this is a Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment4571752628Duchenne Muscular Dystrophy36969902c65b8c9-678b-42bb-82f3-caf803f9fa21Duchenne spierdystrofie37Dutch (Netherlands)36969892c65b8c9-678b-42bb-82f3-caf803f9fa21Distrofia muscular de Duchenne (DMD)7SpanishTrue10000001204720.1PTDuchenne muscular dystrophy gene carrier10052655100000004850False5152703715270461527013152705915270013Safety; Immunogenicity; Multiple-Ascending-Dose Study for Dose Determination, then Dose Expansion152702Part A: To evaluate the safety and tolerability of multiple ascending doses of SRP-5051, administered intravenously (IV) every 4 weeks, and determine the maximum tolerated dose (MTD) Part B: To evaluate dystrophin protein level in skeletal muscle tissue following SRP-5051 treatment, administered IV every 4 weeks at the doses selected based on data from Part A3697020e2034f99-e998-475d-8b7e-2d3592716b51Deel A: het evalueren van de veiligheid en verdraagbaarheid van meerdere oplopende doses SRP-5051, die elke 4 weken intraveneus (IV) worden toegediend, en het bepalen van de maximaal verdraagbare dosis (MTD) Deel B: het evalueren van het dystrofine-eiwitniveau in skeletspierweefsel na behandeling met SRP-5051, die elke 4 weken intraveneus wordt toegediend in de doses die zijn bepaald op basis van gegevens uit deel A37Dutch (Netherlands)3697019e2034f99-e998-475d-8b7e-2d3592716b51Parte A: Evaluar la seguridad y la tolerabilidad de múltiples dosis ascendentes de SRP-5051 (4 mg/kg, 10 mg/kg, 20 mg/kg y 30 mg/kg), administrado por vía intravenosa (IV) cada 4 semanas (C4S), y determinar la dosis máxima tolerada (MTD). Parte B: Evaluar el nivel de la proteína distrofina en los tejidos de los músculos esqueléticos después del tratamiento con SRP-5051, administrado por vía intravenosa cada 4 semanas a las dosis seleccionadas según los datos de la parte A.7Spanish1702231Part A To determine the pharmacokinetics (PK) of SRP-5051 in plasma and urine at each of the aforementioned multiple ascending doses of SRP-5051, administered IV every 4 weeks Part B • To evaluate exon-skipping level in skeletal muscle tissue following SRP-5051 treatment, administered IV every 4 weeks at the doses selected based on data from Part A • To evaluate the ongoing safety and tolerability of SRP-5051 treatment, administered IV every 4 weeks at the doses selected based on data from Part A • To determine the PK of SRP-5051 in plasma and urine • To evaluate, via immunohistochemistry, percent dystrophin-positive fibers (PDPF) and mean intensity following SRP-5051 treatment, administered IV every 4 weeks at the doses selected based on data from Part A36970234b1aa5f5-4da8-48ec-b65f-49000893338bDeel A Het bepalen van de farmacokinetiek (PK) van SRP-5051 in plasma en urine bij elk van de bovengenoemde meerdere oplopende doses SRP-5051, die elke 4 weken IV worden toegediend Deel B • Het evalueren van het exon-skipping-niveau in skeletspierweefsel na behandeling met SRP-5051, die elke 4 weken IV wordt toegediend in de doses bepaald op basis van gegevens uit deel A • Het evalueren van de voortdurende veiligheid en verdraagbaarheid van de behandeling met SRP-5051, die elke 4 weken IV wordt toegediend in de doses bepaald op basis van gegevens uit deel A • Het bepalen van de PK van SRP-5051 in plasma en urine • Het door middel van immunohistochemie evalueren van het percentage dystrofine-positieve vezels (PDPF) en de gemiddelde intensiteit na behandeling met SRP-5051, die elke 4 weken IV wordt toegediend in de doses bepaald op basis van gegevens uit deel A37Dutch (Netherlands)36970244b1aa5f5-4da8-48ec-b65f-49000893338bParte A Determinar la farmacocinética (PK) de la SRP-5051 en plasma y orina, y del metabolito mayor SRP-5051A en plasma, en cada una de las dosis ascendentes múltiples ya mencionadas de la SRP-5051, administrada por vía intravenosa cada 4 semanas. Parte B • Evaluar el nivel de omisión de exón en los tejidos de los músculos esqueléticos después del tratamiento con SRP-5051, administrado por vía intravenosa cada 4 semanas a las dosis seleccionadas según los datos de la parte A. • Evaluar la seguridad y la tolerabilidad continuas del tratamiento con SRP 5051, administrado por vía intravenosa cada 4 semanas a las dosis seleccionadas según los datos de la parte A. • Determinar la farmacocinética de SRP-5051 en plasma y orina. • Evaluar mediante la inmunohistoquímica el porcentaje de fibras distrofina positivas (PDPF) y la intensidad media tras el tratamiento con SRP-5051, administrado por vía intravenosa cada 4 semanas a partir de las dosis seleccionadas según los datos de la parte A.7Spanish3306691Inclusion Criteria for participants previously treated with Vesleteplirsen: - Has received prior Vesleteplirsen treatment in Part A of this study or in Study 5051-102.36970166e7686c6-7ec7-4d86-837d-c4d2671e1ca2Criterios de inclusión para participantes previamente tratados con vesleteplirsén: - Haber recibido tratamiento previo con vesleteplirsén en la parte A de este estudio o en el estudio 5051-102.7Spanish36970156e7686c6-7ec7-4d86-837d-c4d2671e1ca2Inclusiecriteria voor deelnemers die eerder zijn behandeld met vesleteplirsen: - Heeft eerder een behandeling met vesleteplirsen ondergaan in deel A van dit onderzoek of in onderzoek 5051-102.37Dutch (Netherlands)3306702Inclusion Criteria for treatment-naïve participants enrolling into Part B: - Has a genetic diagnosis of Duchenne muscular dystrophy (DMD) and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment. - Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight), or has not received corticosteroids for at least 12 weeks prior to study drug administration. - Has stable pulmonary function (forced vital capacity [FVC] ≥40% of predicted and no requirement for nocturnal ventilation).3697018e4d79ff0-8e61-4e84-b57e-7146cdb2d35fInclusiecriteria voor behandelingsnaïeve deelnemers die worden geïncludeerd in deel B: - Heeft een genetische diagnose van Duchenne spierdystrofie (DMD) en een out-of-frame-deletiemutatie van het DMD-gen die vatbaar is voor exon 51-skipping-behandeling. - Heeft gedurende ten minste 12 weken voorafgaand aan de toediening van het onderzoeksmiddel een stabiele dosis orale corticosteroïden gebruikt en de dosis zal naar verwachting tijdens het onderzoek constant blijven (behalve aanpassingen om gewichtsveranderingen op te vangen), of heeft gedurende ten minste 12 weken voorafgaand aan de toediening van het onderzoeksmiddel geen corticosteroïden ontvangen. - Heeft een stabiele longfunctie (geforceerde vitale capaciteit [FVC] ≥ 40% van de voorspelde waarde en geen noodzaak voor nachtelijke beademing).37Dutch (Netherlands)3697017e4d79ff0-8e61-4e84-b57e-7146cdb2d35fCriterios de inclusión para participantes sin tratamiento previo que se incluyan en la parte B: - Tener un diagnóstico genético de distrofia muscular de Duchenne (DMD) y una mutación de deleción fuera de marco de lectura del gen DMD susceptible al tratamiento de omisión del exón 51. - Haber recibido una dosis estable de corticoesteroides orales durante al menos 12 semanas antes de la administración del fármaco del estudio y tener previsto que la dosis permanezca constante a lo largo del estudio (excepto por modificaciones para adaptarse a cambios de peso), o no haber recibido corticoesteroides durante al menos 12 semanas antes de la administración del fármaco del estudio. - - Tener una función pulmonar estable (capacidad vital forzada [FVC] ≥40 % del valor previsto y no necesitar respiración artificial nocturna).7Spanish5710751Exclusion Criteria for participants previously treated with Vesleteplirsen and new participants enrolling into Part B: - Presence of other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or any other condition that, in the Investigator's opinion, could interfere with participation in the trial.3697009329fb309-bb41-42e9-a8ac-44667aa6c600Criterios de exclusión para participantes tratados previamente con vesleteplirsén y nuevos participantes que se incluyan en la parte B: - Presencia de otra enfermedad clínicamente significativa, incluidas enfermedades cardíacas, pulmonares, hepáticas, renales, hematológicas, inmunológicas o conductuales, o infección o neoplasias malignas, o cualquier otra enfermedad que, en la opinión del investigador, podría interferir con la participación en el ensayo.7Spanish3697010329fb309-bb41-42e9-a8ac-44667aa6c600Exclusiecriteria voor deelnemers die eerder zijn behandeld met vesleteplirsen en nieuwe deelnemers die worden geïncludeerd in deel B: - Aanwezigheid van een andere klinisch significante ziekte, waaronder hart-, long-, lever-, nier-, hematologische, immunologische of gedragsziekte, of een infectie of maligniteit of een andere aandoening die, naar de mening van de onderzoeker, de deelname aan het onderzoek zou kunnen verstoren.37Dutch (Netherlands)5710762Exclusion Criteria for treatment-naive participants enrolling into Part B: - History of hypomagnesemia within 12 weeks prior to Screening. - Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium. - Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations. - Has a left ventricular ejection fraction (LVEF) <40.0% based on an echocardiogram (ECHO) performed within 12 weeks prior to Screening or at the Screening Visit. - Treatment with any exon 51-skipping therapy within 4 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time. - Other inclusion/exclusion criteria apply.3697011a9731a70-3a2e-4920-97ba-d905d2e879edCriterios de exclusión para participantes sin tratamiento previo que se incluyan en la parte B: - Antecedentes de hipomagnesemia en las 12 semanas previas a la selección. - Inicio o cambio de la pauta posológica (excepto modificaciones para adaptarse a cambios de peso o cambios en el tratamiento de referencia) en las 12 semanas previas a la selección para cualquiera de los siguientes tratamientos: inhibidores de la enzima convertidora de la angiotensina, agentes antagonistas de los receptores de la angiotensina, betabloqueantes o potasio. - En las 12 semanas previas a la selección, inicio o cambio en la posología de preparaciones de venta sin receta, como suplementos a base de hierbas o no, vitaminas, minerales y preparaciones homeopáticas. - Tener una fracción de eyección del ventrículo izquierdo (FEVI) <40,0 % basada en un ecocardiograma realizado en las 12 semanas previas a la selección o en la visita de selección. - Tratamiento con cualquier terapia de omisión del exón 51 en las 4 semanas anteriores a la selección, o con cualquier terapia génica experimental para el tratamiento de la DMD en cualquier momento. - - Se aplican otros criterios de inclusión/exclusión.7Spanish3697012a9731a70-3a2e-4920-97ba-d905d2e879edExclusiecriteria voor behandelingsnaïeve deelnemers die worden geïncludeerd in deel B: - Voorgeschiedenis van hypomagnesiëmie in de 12 weken voorafgaand aan de screening. - Starten of wijzigen van de dosering (behalve voor wijzigingen in verband met gewichtsveranderingen of veranderingen in de standaardzorg) in de 12 weken voorafgaand aan de screening van een van de volgende middelen: angiotensine-converterend-enzymremmers, angiotensinereceptorblokkers, bètablokkers of kalium. - Starten of wijzigen van de dosering in de 12 weken voorafgaand aan de screening van vrij verkrijgbare preparaten, zoals kruiden-/niet-kruidensupplementen, vitamines, mineralen en homeopathische preparaten. - Heeft een linkerventrikelejectiefractie (LVEF) < 40,0% gebaseerd op een echocardiogram (echo) uitgevoerd in de 12 weken voorafgaand aan de screening of tijdens het screeningsbezoek. - Behandeling met een exon 51-skipping-therapie in de 4 weken voorafgaand aan de screening, of met een experimentele gentherapie voor de behandeling van DMD op elk moment. - Er kunnen ook andere inclusie-/exclusiecriteria gelden.37Dutch (Netherlands)3835731Part A: Incidence of Adverse Events (AEs), Baseline up to 75 weeksTrue3696992aa81c546-ad1a-4ca6-b508-406ac6d9229dParte A: Incidencia de efectos adversos (EA), línea de base superior a 75 semanas.7Spanish3696991aa81c546-ad1a-4ca6-b508-406ac6d9229dDeel A: incidentie van bijwerkingen (AE’s), baseline tot 75 weken37Dutch (Netherlands)3835742Part B: Change From Baseline in Dystrophin Protein Level at Week 28True3696993708d93a6-e2c2-487c-a254-4e7babd226abDeel B: verandering ten opzichte van de uitgangswaarde in het dystrofine-eiwitniveau in week 2837Dutch (Netherlands)3696994708d93a6-e2c2-487c-a254-4e7babd226abParte B: Cambio desde la línea de base en el nivel de proteína distrofina (según lo medido por Western blot) a las 28 semanas7Spanish3835751Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen, Pre-dose and at multiple time points (up to 32 hours) after end of infusionFalse3696996c26f5b66-0142-4dbe-8e20-af36325a4d5bParte B: cambio con respecto al momento basal en los niveles de omisión del exón en la semana 28.7Spanish3696995c26f5b66-0142-4dbe-8e20-af36325a4d5bDeel B: verandering ten opzichte van de uitgangswaarde in exon-skipping-niveaus in week 2837Dutch (Netherlands)3835762Part A: PK: Urine Concentration of Vesleteplirsen, Pre-dose and at multiple time periods (up to 48 hours) after end of infusionFalse369699822a85507-d315-4ca9-80a6-447d3d9c320bParte A: FC: concentración de vesleteplirsén en la orina, antes de la administración de la dosis y en varios momentos del estudio (hasta 48 horas) después del final de la infusión.7Spanish369699722a85507-d315-4ca9-80a6-447d3d9c320bDeel A: PK: urineconcentratie van vesleteplirsen, vóór de dosis en op meerdere tijdstippen (tot 48 uur) na het einde van de infusie37Dutch (Netherlands)3835773Part B: Change From Baseline in Exon-Skipping Levels at Week 28False3697000578a3d36-86e2-45b1-8a4d-4f98c4673c5eDeel B: verandering ten opzichte van de uitgangswaarde in exon-skipping-niveaus in week 2837Dutch (Netherlands)3696999578a3d36-86e2-45b1-8a4d-4f98c4673c5eParte B: cambio con respecto al momento basal en los niveles de omisión del exón en la semana 28.7Spanish3835784Part B: Incidence of Adverse Events (AEs), Baseline up to Week 304False3697002d499e02a-1f5b-42cd-af62-855073c91e4fParte B: incidencia de acontecimientos adversos (AA), desde el momento basal hasta la semana 304.7Spanish3697001d499e02a-1f5b-42cd-af62-855073c91e4fDeel B: incidentie van bijwerkingen (AE’s), baseline tot 304 weken37Dutch (Netherlands)3835795Part B: PK: Plasma Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusionFalse3697003da59b419-8410-4614-b707-49073fbee087Deel B: PK: plasmaconcentratie van vesleteplirsen, deel B vóór de dosis en op meerdere tijdstippen (tot 48 uur) na het einde van de infusie37Dutch (Netherlands)3697004da59b419-8410-4614-b707-49073fbee087Parte B: FC: concentración plasmática de vesleteplirsén, antes de la administración de la dosis en la parte B y en varios momentos del estudio (hasta 48 horas) después del final de la infusión.7Spanish3835806Part B: PK: Urine Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusionFalse36970068bc90fe2-664d-4828-91cc-d48dade2b0b6Deel B: PK: urineconcentratie van vesleteplirsen, deel B vóór de dosis en op meerdere tijdstippen (tot 48 uur) na het einde van de infusie37Dutch (Netherlands)36970058bc90fe2-664d-4828-91cc-d48dade2b0b6Parte B: FC: concentración plasmática de vesleteplirsén en la orina, antes de la administración de la dosis en la parte B y en varios momentos del estudio (hasta 48 horas) después del final de la infusión.7Spanish3835817Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28False36970073dcc536f-fa9b-468a-8e02-b85213f21ab1Deel B: verandering ten opzichte van de uitgangswaarde in het percentage dystrofine-positieve vezels (PDPF) en de gemiddelde intensiteit, zoals gemeten met een immunofluorescentietest in week 2837Dutch (Netherlands)36970083dcc536f-fa9b-468a-8e02-b85213f21ab1Parte B: cambio con respecto al momento basal en el porcentaje de fibras positivas para distrofina (PFPD) e intensidad media, medido mediante ensayo de inmunofluorescencia en la semana 28.7Spanish2029-01-312029-01-312019-04-3037498Sarepta Therapeutics, Inc.14410522144106332PatientsFalseTrueTrue2802001Part A Screening ; Treatment and Observation Period; Study Safety Follow-up PeriodA traditional “3 + 3” design has been selected for Part A. In this design scheme, patients will initially be enrolled into the lowest dose level in Part A, and enrollment into the higher dose levels will occur only if lower dose levels are deemed safe and tolerable. Each Part A dose-level cohort will enroll at least 3 patients to evaluate safety and tolerability.3Part A is intended to evaluate the safety and tolerability of SRP-5051 administered IV Q4W at MAD levels, and determine the MTDPart A - Multiple-Ascending-Dose (MAD) for Dose Determination452063802012Part B Screening; Safety Lead-in Period; Treatment and Observation Period; Study Safety FU PeriodThe transition from Part A to Part B of the study will occur once the doses for Part B have been selected and approved by the SRC. The dose levels in Part B are predicted to be in the therapeutic range, based on available dystrophin data from Part A, and thus warrant further evaluation to optimize benefit risk and refine the therapeutic exposure window.3Part B is intended to examine treatment with SRP-5051 administered IV Q4W at the doses selected based on data from Part A, and which includes the following 2 patient cohorts: - Previously Treated (PTx) Cohort: Patients who previously received SRP-5051 treatment in Part A of this study or in Study 5051-102 - Treatment-Naïve (TN) Cohort: Patients newly enrolled in the study at the beginning of Part B who have not previously received SRP-5051 treatmentPart B – Further Evaluation of Selected Doses4520734300EMEA-003305-PIP01-22acceptable_conditions2024-09-272024-09-2716Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]5Diseases [C] - Musculoskeletal Diseases [C05]52628Duchenne Muscular DystrophyTrue56609True165628PublicPatient RecruitmentSareptAlly@sarepta.com+18887273782277577Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active165629ScientificPatient RecruitmentSareptAlly@sarepta.com+18887273782277577Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active236880385681415333Pharmaceutical company10Flagship Biosciences Inc.TrueTrueORG-100043268Active41215811800 Ridge Parkway Suite 45011800 Ridge Parkway Suite 450Broomfield80021-6503840United States+13033255894ddoherty@flagshipbio.comTrueORG-10004326837263315Muscle Biopsy Analysis / Immunochemistry (tissue allocated and sent by UIHC)3726344+13033255894ddoherty@flagshipbio.com236885385645415297Pharmaceutical company10Bioagilytix Labs LLCTrueTrueORG-100013030Active4121192300 Englert Drive Suite G2300 Englert Drive Suite GDurham27713-4450840United States+16174560700sarah.rieveschl@bioagilytix.comTrueORG-10001303037264015Skeletal Muscle Panel Biomarker Analysis3726414+16174560700sarah.rieveschl@bioagilytix.com236876385652415304Pharmaceutical company10Chillibean LimitedTrueTrueORG-100042592Active41212653 Frith Street53 Frith StreetLondonW1D 4SN826United Kingdom+442074797032hannah@chillibean.tvTrueORG-10004259237262643726277+442074797032hannah@chillibean.tv236882385631415283Non-Pharmaceutical company11Suvoda LLCTrueTrueORG-100043523Active412105181 Washington Street Suite 100181 Washington Street Suite 100Conshohocken19428-2082840United States+16102917903mhummel@suvoda.comTrueORG-1000435233726373+16102917903mhummel@suvoda.com236883385642415294Pharmaceutical company10Vitalograph LimitedTrueTrueORG-100039692Active412116Maids Moreton House, Vitalograph Business Park, Maids MoretonMaids Moreton HouseVitalograph Business ParkMaids MoretonBuckinghamMK18 1SW826United Kingdom+441280827110Sian.Blight@vitalograph.co.ukTrueORG-10003969237263815Cardio-respiratory vendor ( i.e., ECGs, FVC, MIP and MEP testing)+441280827110Sian.Blight@vitalograph.co.uk236870385688415340Pharmaceutical company10Frontage Laboratories Inc.TrueTrueORG-100011515Active412166700 Pennsylvania Drive700 Pennsylvania DriveExton19341-1129840United States+16102320100rgrabert@frontagelab.comTrueORG-10001151537261215Biopsy, Plasma and Urine PK sample processing3726134+16102320100rgrabert@frontagelab.com236892385759415411Pharmaceutical company10Syneos Health Inc.TrueTrueORG-100008382Active4122491030 Sync Street1030 Sync StreetMorrisville27560-5468840United States+447976977285iain.low@syneoshealth.comTrueORG-10000838237265213726531137265412372655237265653726576372658737265983726609+447976977285iain.low@syneoshealth.com236877385777415429Pharmaceutical company10Teckro LimitedTrueTrueORG-100041454Active41226963 O'connell Street63 O'connell StreetLimerickV94 XT622017Ireland+35361529003john@teckro.comTrueORG-10004145437262815Study document repository+35361529003john@teckro.com236875385622415274Pharmaceutical company10Labcorp Central Laboratory Services SARLTrueTrueORG-100011524Active412096Rue Moise-Marcinhes 7Rue Moise-Marcinhes 7Meyrin1217756Switzerland+17328822521samantha.covey@labcorp.comTrueORG-1000115243726254+17328822521samantha.covey@labcorp.com236891385611415263Pharmaceutical company10The Andwin Corp.TrueTrueORG-100047847Active412085167 West Cochran Street167 West Cochran StreetSimi Valley93065-6217840United States+18189992828areynolds@andwin.comTrueORG-1000478473726501437265115Equipment Supplier+18189992828areynolds@andwin.com236887385785415437Pharmaceutical company10United Biosource LLCTrueTrueORG-100027856Active412277920 Harvest Drive920 Harvest DriveBlue Bell19422-1968840United States+41792130492Anne.Zufferey@ub.comTrueORG-10002785637264315SAE Reporting/Safety management vendor; safety database, pharmacovigilance3726448+41792130492Anne.Zufferey@ub.com236884385616415268Non-Pharmaceutical company11ATOM International LimitedTrueTrueORG-100042393Active412090Office 16, Valley House, Kingsway South, Team ValleyOffice 16Valley HouseKingsway SouthTeam ValleyGatesheadNE11 0JW826United Kingdom+15137069924kyle.haas@atom-international.orgTrueORG-10004239337263915Cllinical Evaluator Trainer Vendor+15137069924kyle.haas@atom-international.org236881385725415377Pharmaceutical company10Nextcea Inc.TrueTrueORG-100041657Active412211500 West Cummings Park Suite 4550500 West Cummings Park Suite 4550Woburn01801-6503840United States+17814574010caitlyn.collins@nextcea.comTrueORG-10004165737263515Urine Safety Biomarker Analysis3726364+17814574010caitlyn.collins@nextcea.com236888385715415367Hospital/Clinic/Other health care facility8Nationwide Childrens HospitalFalseTrueORG-100032545Active412198700 Childrens Drive Suite W2011700 Childrens Drive Suite W2011Columbus43205-2664840United States+16147226881linda.lowes@nationwidechildrens.orgTrueORG-10003254537264515Functional Assessments (Consulting)+16147226881linda.lowes@nationwidechildrens.org236886385710415362Pharmaceutical company10Matthews Media Group Inc.TrueTrueORG-100045638Active4121937361 Calhoun Place7361 Calhoun PlaceDerwood20855-2765840United States+14102795153TGroller@mmgct.comTrueORG-10004563837264215Patient Recruitment and retention service+14102795153TGroller@mmgct.com236871385601415253Laboratory/Research/Testing facility9Medable Inc.TrueTrueORG-100043083Active412075525 University Avenue Suite A70525 University Avenue Suite A70Palo Alto94301-1924840United States+15513106509nicolas.fuentes@medable.comTrueORG-10004308337261415Patient diaries for Mg supplements3726157+15513106509nicolas.fuentes@medable.com236894385677415329Non-Pharmaceutical company11Medidata Solutions Inc.TrueTrueORG-100016256Active412153350 Hudson Street350 Hudson StreetNew York10014-4504840United States+19518212621rlearybullard@eclinicalsol.comTrueORG-10001625637266363726647+19518212621rlearybullard@eclinicalsol.com236889385740415392Pharmaceutical company10PCI Pharma Services Germany GmbHTrueTrueORG-100031981Active412228Am Wall 5Am Wall 5Großbeeren149792013Germany+441656750561Rebecca.Lewis@pci.comTrueORG-1000319813726461437264715IP Drug Supply / Shipment, Logistics, and Depot+441656750561Rebecca.Lewis@pci.com236890385699415351Pharmaceutical company10Llx Solutions LLCTrueTrueORG-100046614Active4121781400 Main Street Floor 11400 Main Street Floor 1Waltham02451-1602840United States+327814722591Elaine.Dong@llxsolutions.comTrueORG-1000466143726481037264915Biostatistical Programming and Analysis+327814722591Elaine.Dong@llxsolutions.com236874385693415345Pharmaceutical company10Biologics Development Services LLCTrueTrueORG-100044619Active4121714710 Eisenhower Boulevard Building D4710 Eisenhower Boulevard Building DTampa33634-6335840United States+18555237276jambrosius@immunologixlabs.comTrueORG-10004461937262315Immunogenicity sample analysis (anti-SRP5051 and anti-dystrophin testing)3726244+18555237276jambrosius@immunologixlabs.com236873385629415281Pharmaceutical company10Bbk Worldwide LLCTrueTrueORG-100044633Active412103117 Kendrick Street117 Kendrick StreetNeedham02494-2724840United States+16176305539ryan.donnelly@bbkworldwide.comTrueORG-10004463337262215Patient Reimbursement+16176305539ryan.donnelly@bbkworldwide.com236893385670415322Pharmaceutical company10Simulations Plus Inc.TrueTrueORG-100029866Active41214642505 10th Street West42505 10th Street WestLancaster93534-7059840United States+17166333463amparo.delapena@simulations-plus.comTrueORG-10002986637266115PK analysis specifically for 5051-201 and applies to US and Ex-US3726624+17166333463amparo.delapena@simulations-plus.com236878385733415385Pharmaceutical company10Pharpoint Research Inc.TrueTrueORG-100048095Active4122205003 South Miami Boulevard Suite 1005003 South Miami Boulevard Suite 100Durham27703-8589840United States+19103864277Victoria.Elder@pharpoint.comTrueORG-100048095372629103726301437263115Biostatistical Programming and Analysis, Biopsy blinding packet production+19103864277Victoria.Elder@pharpoint.com236879385624415276Laboratory/Research/Testing facility9Medical Research Network LimitedTrueTrueORG-100043138Active412098Talon House, Presley Way, CrownhillTalon HousePresley WayCrownhillMilton KeynesMK8 0ES826United Kingdom+441908261153Helen.Wilkinson@themrn.co.ukTrueORG-10004313837263215Home Health Care Provider+441908261153Helen.Wilkinson@themrn.co.uk236872385597415249Pharmaceutical company10PPD Development L.P.TrueTrueORG-100011560Active4120713151 South 17th Street3151 South 17th StreetWilmington28412-6461840United States+33182392401EUCTRInquiry.sm@ppd.comTrueORG-100011560372616137261712372618133726191437262015SRC management, 3rd party vendor management, Medical Management, and TMF3726215+33182392401EUCTRInquiry.sm@ppd.com277577Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active273631277577Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active291719215 1st Street215 1st StreetCambridge02142-1213840United StatesTrueORG-100006645TrueCommercial2According to the "Appendix on transparency rules to the functional specifications of the EU clinical trials portal and database" chapter 4.3.3, Category 2 trials are safety and efficacy trials in patients. The current study meets this requirement, this is a Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment58579716Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]5857965Diseases [C] - Musculoskeletal Diseases [C05]168102VESLETEPLIRSEN(SRP-5051)DE_BB_01_MIA_2024_0015; IMP11566/000011Test215976110281392VIAL FOR INTRAVENOUS USEPRD94569401VESLETEPLIRSEN(SRP-5051)VIAL FOR INTRAVENOUS USESRP-5051VESLETEPLIRSENSUB221175VESLETEPLIRSENSAREPTA THERAPEUTICS INC10281392282173SAREPTA THERAPEUTICS INCNucleic AcidNucleic AcidVESLETEPLIRSENSUB221175False1Falsemg/kg milligram(s)/kilogram0mg/kg milligram(s)/kilogram03002ChemicalPRD9456940SRP-5051DE_BB_01_MIA_2024_0015; IMP11566/0000111INTRAVENOUS USEFalseVESLETEPLIRSEN(SRP-5051)vesleteplirsenVIAL FOR INTRAVENOUS USEFalse1360682679526795106022013Ended2024-04-102024-04-10FalseunwillingGermany2024-05-08Ended194422022-06-202024-05-14194432022-06-202024-05-14230382022-07-182024-03-302024-05-14230392022-07-182024-03-302024-05-14230222022-07-182024-05-14230232022-07-182024-05-14True250042022-06-202024-05-142022-07-182024-03-30False3338426795Authorised2024-05-08T09:41:21.0642867726795Under evaluation2024-03-28T17:56:56.757051426795Ended2024-11-26T18:31:22.5741Germanyacceptable2024-09-11authorized2024-05-082024-05-083888410387171416831Hospital/Clinic/Other health care facility8Universitaetsklinikum Essen AöRFalseTrueORG-100009964Active413776Hufelandstrasse 55, HolsterhausenHufelandstrasse 55HolsterhausenEssen451472013Germany+492017232508ulrike.schara-schmidt@uk-essen.deTrueORG-100009964991321UlrikeSchara-Schmidt+492017232508ulrike.schara-schmidt@uk-essen.de2Klinik für Kinderheilkunde I, Neuropädiatrie/Sozialpädiatrisches Zentrum888411387179416839Hospital/Clinic/Other health care facility8Klinikum der Universitaet Muenchen AöRFalseTrueORG-100008479Active413784Lindwurmstrasse 4, Ludwigsvorstadt-IsarvorstadtLindwurmstrasse 4Ludwigsvorstadt-IsarvorstadtMunich803372013Germany+4989440055110astrid.blaschek@med.uni-muenchen.deTrueORG-100008479991322AstridBlaschek+4989440055110astrid.blaschek@med.uni-muenchen.de1Kinderklinik und Kinderpoliklinik, Abt. Neuropädiatrie, Entwicklungsneurologie und SozialpädiatrieAuthorised1375112700127001106022029Ended2024-04-102024-04-10FalseunwillingNetherlands2024-05-06Ended194462022-07-012024-05-14194472022-07-012024-05-14230302022-07-062024-03-302024-05-14230312022-07-062024-03-302024-05-14230262022-07-062024-05-14230272022-07-062024-05-14True250062022-07-012024-05-142022-07-062024-03-30False3309027001Authorised2024-05-06T12:07:16.632868027001Under evaluation2024-03-28T17:56:56.8987051327001Ended2024-11-26T18:31:03.061Netherlandsacceptable2024-10-02authorized2024-05-062024-05-061897737387382417046Hospital/Clinic/Other health care facility8Leids Universitair Medisch Centrum (LUMC)FalseTrueORG-100014145Active414004Albinusdreef 2Albinusdreef 2Leiden2333 ZA2029Netherlands+31715262197e.h.niks@lumc.nlTrueORG-1000141451001685ErikNiks+31715262197e.h.niks@lumc.nl1Akademisch Ziekenhuis LeidenAuthorised1379612691426914106022002Ended2024-04-102024-04-10FalseunwillingBelgium2024-05-07Ended194402020-08-212024-05-14194412020-08-212024-05-14230342020-08-262024-03-302024-05-14230352020-08-262024-03-302024-05-14230202020-08-262024-05-14230212020-08-262024-05-14True250032020-08-212024-05-142020-08-262024-03-30False3325326914Authorised2024-05-07T13:39:16.0232867626914Under evaluation2024-03-28T17:56:56.7027051126914Ended2024-11-26T18:30:17.7341Belgiumacceptable2024-09-26authorized2024-05-072024-05-076900723386006415658Hospital/Clinic/Other health care facility8UZ LeuvenFalseTrueORG-100006001Active412516Herestraat 49Herestraat 49Leuven30002002Belgium+3216343827liesbeth.dewaele@uzleuven.beTrueORG-1000060011005032LiesbethDe Waele+3216343827liesbeth.dewaele@uzleuven.be1Neurology900724386008415660Hospital/Clinic/Other health care facility8Universitair Ziekenhuis GentFalseTrueORG-100021542Active412518Corneel Heymanslaan 10Corneel Heymanslaan 10Gent90002002Belgium+32473966619nicolas.deconinck@huderf.beTrueORG-1000215421005033NicolasDeconinck+32473966619nicolas.deconinck@huderf.be1Internal MedicineAuthorised1347132695026950106022018Ended2024-04-102024-04-10FalseunwillingItaly2024-05-13Ended194442022-07-112024-05-14194452022-07-112024-05-14230242022-07-132024-05-14230252022-07-132024-05-14230322022-07-132024-03-302024-05-14230332022-07-132024-03-302024-05-14True250052022-07-112024-05-142022-07-132024-03-30False3401426950Authorised2024-05-13T23:59:592867826950Under evaluation2024-03-28T17:56:56.7997051026950Ended2024-11-26T18:29:51.4571Italyacceptable2024-09-13authorized2024-05-132024-05-137879604384885414535Hospital/Clinic/Other health care facility8Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di TorinoFalseTrueORG-100010457Active411295Piazza Polonia 94Piazza Polonia 94Turin101262018Italy+390113134444federica.ricci@unito.itTrueORG-100010457981552Federica SilviaRicci+390113134444federica.ricci@unito.it1S.C. Neuropsichiatria Infantile879605384881414531Hospital/Clinic/Other health care facility8Fondazione Policlinico Universitario Agostino Gemelli IRCCSFalseTrueORG-100014358Active411288Largo Agostino Gemelli 8Largo Agostino Gemelli 8Rome001682018Italy+390630155340eugeniomaria.mercuri@policlinicogemelli.itTrueORG-100014358981553Eugenio MariaMercuri+390630155340eugeniomaria.mercuri@policlinicogemelli.it1UOC Neuropsichiatria infantileAuthorised1377722679626796106022027Ended2024-04-102024-04-10TrueunwillingSpain2024-05-06Ended194482020-09-102024-05-14194492020-09-102024-05-14230282020-09-172024-05-14230292020-09-172024-05-14230362020-09-172024-03-302024-05-14230372020-09-172024-03-302024-05-14True250072020-09-102024-05-142020-09-172024-03-30False3303226796Authorised2024-05-06T09:31:42.3832867926796Under evaluation2024-03-28T17:56:56.857051226796Ended2024-11-26T18:30:40.3881Spainacceptable2024-10-04authorized2024-05-062024-05-064899471382865412500Hospital/Clinic/Other health care facility8Sant Joan De Deu Barcelona HospitalFalseTrueORG-100023083Active409076Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain+34936009733andres.nascimento@sjd.esTrueORG-1000230831003620AndresNascimiento Osorio+34936009733andres.nascimento@sjd.es1Enfermedades Neuromusculares, 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MODIFICATIONFalse399972972126795Germany2024-10-01T12:35:38.324authorized_conditionsacceptabledecision13606853637SUBSTANTIAL MODIFICATIONFalse401602972127001Netherlands2024-10-02T12:59:38.482authorized_conditionsacceptabledecision13751153637SUBSTANTIAL MODIFICATIONFalse405572972126796Spain2024-10-07T08:00:46.982authorized_conditionsacceptabledecision13777253637SUBSTANTIAL MODIFICATIONTrueNetherlands270012024-05-06T12:07:17.8642024-10-02T12:59:38.4828Spain267962024-05-06T09:31:43.3072024-10-07T08:00:46.9828Belgium269142024-05-07T13:39:17.1412024-10-02T00:00:008Germany267952024-05-08T09:41:22.2382024-10-01T12:35:38.3248Italy269502024-05-13T00:00:002024-10-01T23:06:04.1468True2019-000601-7726795GermanySTART_OF_TRIAL2022-06-20START_OF_RECRUITMENT2022-07-18END_OF_RECRUITMENT2024-03-30EARLY_TERMINATION2024-11-066Safety Related (Clinical or Pre-Clinical Results)False26796SpainSTART_OF_TRIAL2020-09-10START_OF_RECRUITMENT2020-09-17END_OF_RECRUITMENT2024-03-30EARLY_TERMINATION2024-11-066Safety 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results (for publication)PDF21001.01Both3 2023-505043-39-004AuthorisedA Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study (EXPEDITION)SRP-9001-305Duchenne Muscular DystrophySpain:4Italy:4Belgium:4Germany:414/03/2024IT: 21/03/2024, DE: 15/03/2024, ES: 14/03/2024, BE: 19/03/2024Diseases [C] - Musculoskeletal Diseases [C05]Sarepta Therapeutics Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Time of 10-meter Walk/Run (10MWR) from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation., Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in PUL (Version 2.0) Domain Specific Scores from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Forced Vital Capacity Percent (FVC%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Peak Expiratory Flow Percent (PEF%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Cardiac Magnetic Resonance Imaging (MRI) findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Musculoskeletal MRI Findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationDelandistrogene moxeparvovec-rokl4560-17 years, 18-64 yearsMale3Both74Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event 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consent form (for publication)PDF2741024.04BEL Country ICF Procedure English SRP-9001-305 Public8e9bec89-d679-43c6-9f53-6b56b422b63115Subject information and informed consent form (for publication)PDF2741021.01Spain154962024-03-14T10:56:11.0622026-04-20T08:03:01.8854Italy154952024-03-21T11:31:46.2522026-04-20T22:12:00.2524Belgium154932024-03-19T11:55:26.3122026-04-14T14:44:39.6734Germany154942024-03-15T10:58:55.4032026-04-14T12:39:39.713415493BelgiumSTART_OF_TRIAL2024-09-26START_OF_RECRUITMENT2024-10-1615494GermanySTART_OF_TRIAL2024-08-26START_OF_RECRUITMENT2024-09-2315495ItalySTART_OF_TRIAL2024-09-02START_OF_RECRUITMENT2024-09-0415496SpainSTART_OF_TRIAL2024-05-24START_OF_RECRUITMENT2024-05-282023-505043-39-004A Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study (EXPEDITION)SRP-9001-30524/05/2024Duchenne Muscular DystrophySpain:4Italy:4Belgium:4Germany:414/03/2024IT: 21/03/2024, DE: 15/03/2024, ES: 14/03/2024, BE: 19/03/2024Diseases [C] - Musculoskeletal Diseases [C05]Sarepta Therapeutics Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Time of 10-meter Walk/Run (10MWR) from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation., Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in PUL (Version 2.0) Domain Specific Scores from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Forced Vital Capacity Percent (FVC%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Peak Expiratory Flow Percent (PEF%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Cardiac Magnetic Resonance Imaging (MRI) findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation, Change in Musculoskeletal MRI Findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationDelandistrogene moxeparvovec-rokl4560-17 years, 18-64 yearsMale374Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)No20/04/202621/04/20262023-505043-39-00Authorised2024-05-242024-03-14T10:56:11.0622026-04-21T03:42:43.9384998334115127319100000000329Australia36AUAUSTrue100000000557United States840USUSATrue100000000556United Kingdom826GBGBRTrue100000000429Israel376ILISRTrue100000000420Hong Kong344HKHKGTrue100000000538Taiwan158TWTWNTrue100000000432Japan392JPJPNTrue477799110281347SOLUTION FOR INJECTION/INFUSIONPRD86568511Delandistrogene moxeparvovec-roklSOLUTION FOR INJECTION/INFUSIONSRP-9001DELANDISTROGENE MOXEPARVOVECSUB197789SAREPTA THERAPEUTICS INC10281347235539SAREPTA THERAPEUTICS INCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherDELANDISTROGENE MOXEPARVOVECSUB197789True1TrueEU/3/20/2250vector genomes (vg)/mL13300000000000vector genomes (vg)/mL1330000000000011PRD8656851SRP-90016034EMA/CAT/113473/20193Delandistrogene moxeparvovec dystrophinAAVFalseTrue38INTRAVENOUS USEFalseDelandistrogene moxeparvovec-rokldelandistrogene moxeparvovecSOLUTION FOR INJECTION/INFUSIONA Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study (EXPEDITION)9420284a6cbd741-1681-4626-b708-845c97e62686Estudio en fase III, multinacional, de seguimiento a largo plazo para evaluar la seguridad y la eficacia en pacientes que han recibido con anterioridad SRP-9001 en un estudio clínico (EXPEDITION)7SpanishA Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (EXPEDITION)9420288552301aa-1a9b-4b2c-b320-308fe81ef74dEstudio de seguimiento a largo plazo de participantes que recibieron delandistrogén moxeparvovec (SRP-9001) en un estudio clínico anterior (EXPEDITION)7SpanishSRP-9001-305479063U1111-1296-0489479062NCT05967351False52Category 2 (Phase 3) trial105369119572Duchenne Muscular Dystrophy9420272c47fdb8b-1729-40ce-ae40-92b3229d48eaDistrofia muscular de Duchenne7SpanishTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False10000001204720.1PTDuchenne muscular dystrophy gene carrier10052655100000004850False53485264348527To evaluate the long-term safety of delandistrogene moxeparvovec9420287dbecbb55-8943-4255-b12d-20f675e262c5Evaluar la seguridad a largo plazo de delandistrogén moxeparvovec7Spanish4039141To evaluate the long-term efficacy of delandistrogene moxeparvovec9420289f5b675f2-31fb-4597-87dd-732a22a15e81Evaluar la eficacia a largo plazo de delandistrogén moxeparvovec.7Spanish7729611Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study9420285b345b193-7a65-4efc-8451-a0fb9b3c4aedRecibieron delandistrogén moxeparvovec para la distrofia muscular de Duchenne en un estudio clínico anterior7Spanish7729622Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements94202866b79f1ef-b9e5-401f-83ae-49d002a6edafTener progenitor(es) o cuidador(es) legal(es) o tener ≥18 años y ser capaces de comprender y cumplir el calendario de visitas del estudio y todos los demás requisitos del protocolo7Spanish13247721-94202838bef1c16-2a7e-4e69-9501-1d4667527f43-7Spanish9053071Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)True9420273c9b7b0f6-0f3b-4542-941d-301c484c4efdNúmero de participantes con algún acontecimiento adverso surgido durante el tratamiento (AAST), acontecimiento adverso grave (AAG) y acontecimiento adverso de interés especial (AAIE)7Spanish9053081Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationFalse9420274ebdbc802-7c3d-47c6-a2e4-86e204d2468aCambio producido en la puntuación total de la evaluación ambulatoria North Star (North Star Ambulatory Assessment, NSAA) desde el inicio antes de la infusión de delandistrogén moxeparvovec hasta el final de la participación en el estudio.7Spanish9053092Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationFalse94202751e563180-5075-48eb-bac2-e2f932d82fddCambio producido en el tiempo para levantarse del suelo desde el inicio antes de la infusión de delandistrogén moxeparvovec hasta el final de la participación en el estudio.7Spanish9053103Change in Time of 10-meter Walk/Run (10MWR) from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participation.False9420276eaf82668-a016-4aef-9c8b-2041f508da5bCambio producido en el tiempo para caminar/correr 10 metros (10 mM) desde el inicio antes de la infusión de delandistrogén moxeparvovec hasta el final de la participación en el estudio.7Spanish9053114Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationFalse9420277bda9b1fe-f4b4-46ed-9db3-f724e542a81cCambio producido en las puntuaciones totales de desempeño de las extremidades superiores (Performance of Upper Limbs, PUL) (versión 2.0) desde el inicio antes de la infusión de delandistrogén moxeparvovec hasta el final de la participación en el estudio.7Spanish9053125Change in PUL (Version 2.0) Domain Specific Scores from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationFalse94202786f12f904-75cb-4197-a734-fff908654511Cambio producido en las puntuaciones de dominios específicos de PUL (versión 2.0) desde el inicio antes de la infusión de delandistrogén moxeparvovec hasta el final de la participación en el estudio7Spanish9053136Change in Forced Vital Capacity Percent (FVC%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationFalse94202792a8f2746-92e7-45d1-bff1-11bf09e83924Cambio producido en el porcentaje de capacidad vital forzada (%CVF) previsto desde el inicio antes de la infusión de delandistrogén moxeparvovec hasta el final de la participación en el estudio.7Spanish9053147Change in Peak Expiratory Flow Percent (PEF%) Predicted from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationFalse9420280ede8ba78-273b-4566-9d1c-f4acd95ea5edCambio producido en el porcentaje de flujo espiratorio máximo (%FEM) previsto desde el inicio antes de la infusión de delandistrogén moxeparvovec hasta el final de la participación en el estudio.7Spanish9053158Change in Cardiac Magnetic Resonance Imaging (MRI) findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationFalse9420281a8b6206e-ddc7-4ee5-80e1-cbb62a25973aCambio producido en los resultados de la resonancia magnética (RM) cardíaca desde el inicio antes de la infusión de delandistrogén moxeparvovec hasta el final de la participación en el estudio.7Spanish9053169Change in Musculoskeletal MRI Findings from Pre-infusion Baseline of Delandistrogene Moxeparvovec to the end of study participationFalse9420282088e1c00-e0de-4088-8cde-6074caaf4098Cambio producido en los hallazgos de la RM osteomuscular desde el inicio antes de la infusión de delandistrogén moxeparvovec hasta el final de la participación en el estudio.7Spanish2033-10-242033-10-242024-05-2983860Sarepta Therapeutics Inc.31630222316303333163062424316304252531630526262PatientsFalseTrueTrue2296722020-002372-13A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION) , Étude de phase 3, internationale, randomisée, en double aveugle, contrôlée contre placebo, de thérapie génique systémique, destinée à évaluer la sécurité d’emploi et l’efficacité du SRP9001 chez des patients non ambulatoires et ambulatoires atteints de dystrophie musculaire de Duchenne (ENVISION), Studio di fase 3, internazionale, randomizzato, in doppio cieco, controllato verso placebo, sulla terapia di trasferimento sistemico di geni, per valutare la sicurezza e l’efficacia di SRP-9001 in soggetti deambulanti e non deambulanti con distrofia muscolare di Duchenne (ENVISION), Estudio de fase 3, multinacional, aleatorizado, en doble ciego y controlado con placebo, del tratamiento mediante transferencia génica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos, no ambulatorios y ambulatorios, con distrofia muscular de Duchenne (ENVISION)OPTIONALOPTIONALFalse296732022-000691-19A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy, Estudio abierto, de dos partes, de administración génica sistémica para evaluar la seguridad y la expresión de RO7494222 (SRP-9001) en sujetos menores de cuatro años con Distrofia Muscular de Duchenne, Uno studio in due parti, in aperto, sulla terapia genica sistemica per valutare la sicurezza e l’espressione di RO7494222 (SRP-9001) in soggetti di età inferiore a quattro anni affetti da distrofia muscolare di Duchenne, Etude en 2 parties, en ouvert, de transfert de gène par voir systémique, évaluant la sécurité et l'expression de RO7494222 (SRP-9001) chez l'enfant âgé de moins de quatre ans présentant une dystrophie musculaire de Duchenne OPTIONALOPTIONALFalse296712019-003374-91A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) , Étude de phase 3, multinationale, randomisée, en double aveugle, contrôlée par placebo, sur le transfert systémique de gènes, visant à évaluer la sécurité d’emploi et l’efficacité du SRP-9001 chez des sujets atteints de dystrophie musculaire de Duchenne (EMBARK), Estudio de fase III multinacional, aleatorizado, doble ciego, controlado con placebo, de liberación génica sistémica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos con distrofia muscular de Duchenne (EMBARK), Studio di fase 3, internazionale, randomizzato, in doppio cieco, controllato verso placebo, sul rilascio sistemico di geni, per valutare la sicurezza e l’efficacia di SRP-9001 in soggetti con distrofia muscolare di Duchenne (EMBARK)OPTIONALOPTIONALFalseacceptable2026-04-132026-04-135Diseases [C] - Musculoskeletal Diseases [C05]119572Duchenne Muscular DystrophyTrue130117True383780PublicPatient RecruitmentSareptAlly@sarepta.com+18887273782116535Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active383781ScientificSarepta Therapeutics Inc.SareptAlly@sarepta.com+18887273782116535Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active580703247734251561Pharmaceutical company10Parexel International (IRL) LimitedTrueTrueORG-100022780Active26403470 Sir John Rogerson's Quay70 Sir John Rogerson's QuayDublin 2D02 R2962017Ireland+35314739500Clinicaltrial.Enquiries@parexel.comTrueORG-1000227809032711903272129032731390327415Site Management, Site Contract/Budget Negotiation, Import/Export, Equipment Provisioning9032755903276690327779032788+35314739500Clinicaltrial.Enquiries@parexel.com116535Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active113604116535Pharmaceutical company10Sarepta Therapeutics Inc.TrueTrueORG-100006645Active119461215 1st Street215 1st StreetCambridge02142-1213840United StatesTrueLOC-100013341TrueCommercial2Category 2 (Phase 3) trial10646695Diseases [C] - Musculoskeletal Diseases [C05]357677Delandistrogene moxeparvovec-rokl1Test477799110281347SOLUTION FOR INJECTION/INFUSIONPRD86568511Delandistrogene moxeparvovec-roklSOLUTION FOR INJECTION/INFUSIONSRP-9001DELANDISTROGENE MOXEPARVOVECSUB197789SAREPTA THERAPEUTICS INC10281347235539SAREPTA THERAPEUTICS INCStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherDELANDISTROGENE MOXEPARVOVECSUB197789True1TrueEU/3/20/2250vector genomes (vg)/mL13300000000000vector genomes (vg)/mL1330000000000011PRD8656851SRP-90016034EMA/CAT/113473/20193Delandistrogene moxeparvovec dystrophinAAVFalseTrue38INTRAVENOUS USEFalseDelandistrogene moxeparvovec-rokldelandistrogene moxeparvovecSOLUTION FOR INJECTION/INFUSIONFalse274104154951549553702018Authorised2024-02-202024-02-20FalseunwillingItaly2024-03-21Authorised333952024-09-022024-09-05333962024-09-022024-09-05470602024-09-042024-09-27470612024-09-042024-09-27True444722024-09-022024-09-052024-09-04False1656615495Under evaluation2023-11-17T13:24:32.7822755215495Authorised2024-03-21T11:31:45.4071Italyacceptable2026-02-25authorized2024-03-212024-03-21241879675238135241916Hospital/Clinic/Other health care facility8Fondazione Policlinico Universitario Agostino Gemelli IRCCSFalseTrueORG-100014358Active253740Largo Francesco Vito 1Largo Francesco Vito 1Rome001682018Italy+390630157062eugeniomaria.mercuri@policlinicogemelli.itTrueORG-1000143582088627EugenioMercuri+390630157062eugeniomaria.mercuri@policlinicogemelli.it2404: U.O.C. Neuropsichiatria Infantile1879677238136241917Hospital/Clinic/Other health care facility8Giannina Gaslini Institute For Scientific Hospitalization And CareFalseTrueORG-100010784Active253741Via Gerolamo Gaslini 5Via Gerolamo Gaslini 5Genoa161472018Italy+3901056362620claudiobruno@gaslini.orgTrueORG-1000107842088629ClaudioBruno+3901056362620claudiobruno@gaslini.org1416: Centro di Miologia Traslazionale e Sperimentale1879676238141241922Hospital/Clinic/Other health care facility8Fondazione IRCCS Ca Granda Ospedale Maggiore PoliclinicoFalseTrueORG-100006307Active253746Via Francesco Sforza 28Via Francesco Sforza 28Milan201222018Italy+390255033802giacomo.comi@unimi.itTrueORG-1000063072088628Giacomo PietroComi+390255033802giacomo.comi@unimi.it1444: UOC Neurologia Pad. 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Phase 3, Multi-Center, Open Label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)NS-065/NCNP-01-30207/06/202116/01/2025Duchenne Muscular Dystrophy (DMD)Spain:8Italy:8Greece:8Norway:8Czechia:8Netherlands:826/02/2024CZ: 27/02/2024, NO: 27/02/2024, NL: 27/02/2024, IT: 01/03/2024, GR: 09/04/2024, ES: 26/02/2024Diseases [C] - Musculoskeletal Diseases [C05]Ns Pharma Inc.Pharmaceutical companyTherapeutic confirmatory (Phase III)Secondary: - Time to Stand Test (TTSTAND) - Time to Run/Walk 10 Meters Test (TTRW) - Six-minute Walk Test (6MWT) - North Star Ambulatory Assessment (NSAA) - Time to Climb 4 Stairs Test (TTCLIMB) - Quantitative muscle strength measured by hand-held dynamometer (elbow extension, elbow flexion, knee extension, and knee flexion on the dominant side only)Viltolarsen450-17 yearsMale32103/11/2025- Vital signs - Physical examination - Clinical laboratory tests o Hematology and clinical chemistry o Urinalysis o Urine cytology - Antibodies to dystrophin and viltolarsen - 12-lead electrocardiogram (ECG) - Renal ultrasound - Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)No18/06/202510/02/20262023-507146-91-00Ended2021-06-072025-01-162024-02-26T14:55:12.0162026-02-10T02:35:49.57653050988960253100000000354Canada124CACANTrue100000000465Mexico484MXMEXTrue100000000362China156CNCHNTrue100000000439Korea, Republic of410KRKORTrue100000000329Australia36AUAUSTrue100000000506Russian Federation643RURUSTrue100000000482New Zealand554NZNZLTrue100000000361Chile152CLCHLTrue100000000549Turkey792TRTURTrue100000000556United Kingdom826GBGBRTrue368186110857392SOLUTION FOR INFUSIONPRD74983631ViltolarsenSOLUTION FOR INFUSIONVILTOLARSENSUB195543NS PHARMA, INC.10857392347540NS PHARMA, INC.Nucleic AcidNucleic AcidVILTOLARSENSUB195543True1TrueEU/3/20/2282mg/Kg milligram(s)/kilogram80mg/Kg milligram(s)/kilogram7680962Antisense OligonucleotidePRD7498363NS-065/NCNP-0111SOLUTION FOR INFUSIONFalseViltolarsenviltolarsenSOLUTION FOR INFUSIONA Phase 3, Multi-Center, Open Label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)689089734e353c6-d15f-4ca2-9bd5-d1ee8c650ddbStudio di estensione in aperto, multicentrico, di fase 3, volto a valutare la sicurezza e l’efficacia di viltolarsen in ragazzi deambulanti affetti da distrofia muscolare di Duchenne (DMD)14Italian689089834e353c6-d15f-4ca2-9bd5-d1ee8c650ddbEen open-label fase 3-uitbreidingsonderzoek in meerdere centra ter beoordeling van de veiligheid en werkzaamheid van viltolarsen bij ambulante jongens met Duchenne-spierdystrofie (DMD)18Dutch689089634e353c6-d15f-4ca2-9bd5-d1ee8c650ddbΜία φάσης 3, πολυκεντρική, ανοικτής επισήμανσης μελέτη επέκτασης για την αξιολόγηση της ασφάλειας και της αποτελεσματικότητας του viltolarsen σε περιπατητικά αγόρια με μυϊκή δυστροφία Duchenne (DMD)5Greek689089534e353c6-d15f-4ca2-9bd5-d1ee8c650ddbEstudio de extensión de fase 3, multicéntrico y abierto, para evaluar la eficacia y la seguridad de viltolarsén en niños con distrofia muscular de Duchenne (DMD) capaces de caminar7Spanish"A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)"689091587f1a11d-abae-4379-9c44-b7a4b818260aΜία φάσης 3 μελέτη του viltolarsen σε αγόρια με μυϊκή δυστροφία Duchenne (DMD)5Greek689091687f1a11d-abae-4379-9c44-b7a4b818260a"Een fase 3-verlengingsonderzoek naar viltolarsen bij jongens met Duchenne-spierdystrofie (DMD)"18Dutch689091887f1a11d-abae-4379-9c44-b7a4b818260aEstudio de fase 3 de Viltolarsen en niños con distrofia muscular de Duchenne (DMD)7Spanish689091787f1a11d-abae-4379-9c44-b7a4b818260aStudio di fase 3 di Viltolarsen in ragazzi con distrofia muscolare di Duchenne (DMD)14ItalianNS-065/NCNP-01-302396631U1111-1298-6244396630NCT04768062False52VIL302 study is a Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD). In line with EMA guidance on disclosure rules, the study meets the criteria of a Category 2 trial. We consider all of our scientific information to be company confidential and highly proprietary within the maximum limits of the redaction Guidelines, and disclosure of such information could harm the company by enabling competitors to developing products that could compete with ours.8060891987Duchenne Muscular Dystrophy (DMD)6890868c9057370-9f8b-4257-960a-099bc643f2fbΜυϊκή δυστροφία Duchenne (DMD)5Greek6890869c9057370-9f8b-4257-960a-099bc643f2fbDistrofia Muscolare di Duchenne (DMD)14Italian6890867c9057370-9f8b-4257-960a-099bc643f2fbDuchenne-spierdystrofie (DMD)18Dutch6890870c9057370-9f8b-4257-960a-099bc643f2fbDuchenne Muscular Dystrophy (DMD)7SpanishTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False426639913Tolerability26639732663965266398To evaluate the safety and tolerability of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg in boys who have completed the NS-065/NCNP-01-301 study6890911284fa4a9-4a55-4a87-ad65-5bdc417fcfc2Evaluar la seguridad y tolerabilidad de viltolarsén administrado por vía intravenosa (IV) en dosis semanales de 80 mg/kg en niños que hayan completado el estudio NS-065/NCNP-01-3017Spanish6890912284fa4a9-4a55-4a87-ad65-5bdc417fcfc2Het beoordelen van de veiligheid en verdraagbaarheid van viltolarsen, intraveneus (IV) toegediend in wekelijkse doses van 80 mg/kg bij jongens die het NS 065/NCNP 01-301-onderzoek hebben voltooid18Dutch6890913284fa4a9-4a55-4a87-ad65-5bdc417fcfc2Valutare la sicurezza e la tollerabilità di viltolarsen somministrato per via endovenosa (EV) a dosi settimanali di 80 mg/kg in ragazzi che hanno completato lo studio NS‐065/NCNP‐01-30114Italian6890914284fa4a9-4a55-4a87-ad65-5bdc417fcfc2Αξιολόγηση της ασφάλειας και της ανεκτικότητας του viltolarsen, χορηγούμενου ενδοφλεβίως (IV) σε εβδομαδιαίες δόσεις των 80 mg/kg σε αγόρια που έχουν ολοκληρώσει τη μελέτη NS 065/NCNP 01-3015Greek3042631Secondary Objective: To compare the efficacy of viltolarsen administered IV at weekly doses of 80 mg/kg in boys who have completed the NS-065/NCNP-01-301 study over a 96-week treatment period versus natural history controls using strength and endurance outcomes.68909200f92f432-a4d3-4912-aca9-4b4764fa6f10Σύγκριση της αποτελεσματικότητας του viltolarsen, χορηγούμενου IV σε εβδομαδιαίες δόσεις των 80 mg/kg, σε αγόρια που έχουν ολοκληρώσει τη μελέτη NS 065/NCNP 01-301 κατά τη διάρκεια μίας περιόδου θεραπείας 96 εβδομάδων έναντι μαρτύρων με φυσική πορεία της νόσου, με τη χρήση των εκβάσεων δύναμης και αντοχής5Greek68909220f92f432-a4d3-4912-aca9-4b4764fa6f10Het vergelijken van de werkzaamheid van viltolarsen, intraveneus toegediend in wekelijkse doses van 80 mg/kg bij jongens die het NS 065/NCNP 01-301-onderzoek hebben voltooid gedurende een behandelperiode van 96 weken versus controles van natuurlijk verloop, met behulp van uitkomsten op het gebied van kracht en uithoudingsvermogen18Dutch68909190f92f432-a4d3-4912-aca9-4b4764fa6f10Confrontare l’efficacia di viltolarsen somministrato per EV a dosi settimanali di 80 mg/kg in ragazzi che hanno completato lo studio NS‐065/NCNP‐01-301 nell’arco di un periodo di trattamento di 96 settimane rispetto ai controlli dell’anamnesi naturale utilizzando gli esiti di forza e resistenza.14Italian68909210f92f432-a4d3-4912-aca9-4b4764fa6f10Comparar la eficacia de viltolarsén administrado IV en dosis semanales de 80 mg/kg a niños que hayan completado el • Prueba TTSTAND (Time to Stand Test, Tiempo en ponerse de pie) • Tiempo en recorrer corriendo o caminando 10 NS Pharma, Inc. – CONFIDENCIAL Página 2 de 6 estudio NS-065/NCNP-01-301 durante un período de tratamiento de 96 semanas con controles con evolución natural de la enfermedad, utilizando para ello medidas de fuerza y resistencia7Spanish58708711. Patient has completed the NS-065/NCNP-01-301 study;6890900f2917dcb-9d5f-4f13-872d-f1f9f5446ead1.      Ο ασθενής έχει ολοκληρώσει τη μελέτη NS-065/NCNP-01-3015Greek6890899f2917dcb-9d5f-4f13-872d-f1f9f5446ead1. Il paziente ha completato lo studio NS-065/NCNP-01-301.14Italian6890902f2917dcb-9d5f-4f13-872d-f1f9f5446ead1. El paciente ha completado el estudio NS-065/NCNP-01-3017Spanish6890901f2917dcb-9d5f-4f13-872d-f1f9f5446ead1. Patiënt heeft het NS-065/NCNP-01-301-onderzoek afgerond;18Dutch58708822. Patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients will be asked to give written or verbal assent according to local requirements;689090543efb79b-0936-48cd-98d1-fad061bacbc0"2. Ouder(s) of wettelijke voogd(en) van patiënt heeft (hebben) schriftelijk geïnformeerde toestemming en goedkeuring onder de Health Insurance Portability and Accountability Act, indien van toepassing, verstrekt voorafgaand aan enige procedures in het kader van het onderzoek; patiënten wordt gevraagd om schriftelijk of mondeling instemming te geven in overeenstemming met de plaatselijke vereisten;"18Dutch689090443efb79b-0936-48cd-98d1-fad061bacbc02.      Ο(οι) γονέας(-είς) ή ο(οι) νόμιμος(-οι) κηδεμόνας(-ες) έχουν παράσχει γραπτή συναίνεση μετά από ενημέρωση και εξουσιοδότηση σύμφωνα με τον Νόμο περί φορητότητας και λογοδοσίας για την ασφάλιση υγείας, όπου ισχύει, πριν από οποιεσδήποτε σχετιζόμενες με τη μελέτη διαδικασίες. Θα ζητηθεί από τους ασθενείς να παραχωρήσουν γραπτή ή προφορική σύμφωνη γνώμη σύμφωνα με τις τοπικές απαιτήσεις5Greek689090643efb79b-0936-48cd-98d1-fad061bacbc0"2. Il/I genitore/i o tutore/i legale/i del paziente ha/hanno fornito il consenso informato per iscritto e l’autorizzazione secondo la legge sulla portabilità e responsabilità delle polizze di assicurazione sanitaria (Health Insurance Portability and Accountability Act, HIPAA), ove applicabile, prima di iniziare qualsiasi procedura correlata allo studio; ai pazienti sarà chiesto di fornire il proprio assenso per iscritto o verbale secondo i requisiti locali."14Italian689090343efb79b-0936-48cd-98d1-fad061bacbc02. Los padres o el tutor legal del paciente han otorgado su consentimiento informado por escrito y la autorización HIPAA (Health Insurance Portability and Accountability Act, Ley de Responsabilidad y Portabilidad del Seguro de Salud), cuando sea aplicable; se pedirá a los participantes que otorguen su asentimiento verbal o por escrito de conformidad con los requisitos locales7Spanish58708933. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.6890910a904163e-c982-4fc3-b8f1-a2c4fd096b383. El paciente y sus padres o tutor legal están dispuestos a cumplir las visitas programadas, el plan de administración del producto en investigación (PEI) y los procedimientos del estudio, y pueden hacerlo7Spanish6890909a904163e-c982-4fc3-b8f1-a2c4fd096b383.      Ο ασθενής και ο(οι) γονέας(-είς) ή ο(οι) νόμιμος(-οι) κηδεμόνας(-ες) είναι πρόθυμοι και είναι σε θέση να συμμορφωθούν με τις προγραμματισμένες επισκέψεις, το πρόγραμμα χορήγησης του υπό έρευνα προϊόντος (ΥΕΠ) και τις διαδικασίες της μελέτης.5Greek6890907a904163e-c982-4fc3-b8f1-a2c4fd096b383. Patiënt en ouder(s)/voogd(en) zijn bereid en in staat om zich aan de geplande bezoeken, de toedieningsplanning voor het onderzoeksproduct (IP) en de onderzoeksprocedures te houden.18Dutch6890908a904163e-c982-4fc3-b8f1-a2c4fd096b383. Il paziente e il/i genitore/i o tutore/i legale/i sono disposti, oltreché esserne in grado, ad attenersi alle visite programmate, al piano di somministrazione del farmaco dello studio e alle procedure dello studio.14Italian100981711. Patient had an adverse event in Study NS-065/NCNP-01-301 that, in the opinion of the investigator and/or the sponsor, precludes safe use of viltolarsen for the patient in this study;68908797103bbae-cc28-4434-8c42-adefff28407a1. Il paziente ha manifestato un evento avverso nello studio NS‐065/NCNP‐01‐301 che, a giudizio dello sperimentatore e/o dello Sponsor, preclude l’utilizzo sicuro di viltolarsen per il paziente in questo studio.14Italian68908817103bbae-cc28-4434-8c42-adefff28407a1.      Ο ασθενής εμφάνισε ένα ανεπιθύμητο συμβάν στη μελέτη NS-065/NCNP-01-301, το οποίο, κατά τη γνώμη του ερευνητή ή/και του χορηγού, καθιστά αδύνατη την ασφαλή χρήση του viltolarsen για τον ασθενή σε αυτή τη μελέτη.5Greek68908807103bbae-cc28-4434-8c42-adefff28407a1. El paciente ha sufrido un acontecimiento adverso en el estudio NS-065/NCNP-01-301 que, en opinión del investigador y/o del promotor, impide el uso seguro de viltolarsén en el paciente en este estudio7Spanish68908827103bbae-cc28-4434-8c42-adefff28407a1. Patiënt heeft een bijwerking gehad in het onderzoek NS 065/NCNP 01 301 die, naar mening van de onderzoeker en/of sponsor, veilig gebruik van viltolarsen voor de patiënt in dit onderzoek uitsluit;18Dutch100981822. Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NS-065/NCNP-01-301;689088431391515-3901-4641-8418-5be78f6765c82. Il paziente è stato sottoposto a un trattamento che è stato effettuato per finalità di induzione della distrofina o della proteina correlata alla distrofina dopo il completamento dello studio NS-065/NCNP-01-301.14Italian689088331391515-3901-4641-8418-5be78f6765c82.      Ο ασθενής έλαβε μία θεραπεία με σκοπό την επαγωγή της έκφρασης δυστροφίνης ή σχετιζόμενης με τη δυστροφίνη πρωτεΐνης μετά την ολοκλήρωση της μελέτης NS-065/NCNP-01-301.5Greek689088631391515-3901-4641-8418-5be78f6765c82. Patiënt heeft een behandeling ondergaan die als doel had dystrofine- of een aan dystrofine gerelateerde eiwit-inductie na afronding van onderzoek NS 065/NCNP-01-301;18Dutch689088531391515-3901-4641-8418-5be78f6765c8"2. El paciente ha recibido un tratamiento cuyo objetivo era la inducción de la distrofina o una proteína relacionada con la distrofina después de completar el estudio NS-065/NCNP-01- 301"7Spanish100981933. Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;6890889e781194e-3da3-4abc-bd68-96a3e24ac5363. El paciente recibió otro u otros medicamentos en investigación durante el estudio NS-065/NCNP-01-301 o después de completarlo7Spanish6890887e781194e-3da3-4abc-bd68-96a3e24ac5362. Patiënt heeft een behandeling ondergaan die als doel had dystrofine- of een aan dystrofine gerelateerde eiwit-inductie na afronding van onderzoek NS 065/NCNP-01-301;18Dutch6890890e781194e-3da3-4abc-bd68-96a3e24ac5363. Il paziente ha assunto qualsiasi altro farmaco sperimentale durante o dopo il completamento dello studio NS-065/NCNP-01-301.14Italian6890888e781194e-3da3-4abc-bd68-96a3e24ac5363.      Ο ασθενής έλαβε οποιοδήποτε άλλο υπό έρευνα φάρμακο κατά τη διάρκεια ή μετά την ολοκλήρωση της μελέτης NS-065/NCNP-01-301.5Greek100982044. Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.689089113c1d407-76d1-405b-b83c-62cdb15a59694.      Ο ασθενής κρίνεται από τον ερευνητή ή/και τον χορηγό μη κατάλληλος για συμμετοχή στη μελέτη επέκτασης για οποιονδήποτε λόγο.5Greek689089213c1d407-76d1-405b-b83c-62cdb15a5969"4. El investigador y/o el promotor no consideran apropiado que el paciente participe en el estudio de extensión por algún motivo"7Spanish689089413c1d407-76d1-405b-b83c-62cdb15a59693. Il paziente ha assunto qualsiasi altro farmaco sperimentale durante o dopo il completamento dello studio NS-065/NCNP-01-301.14Italian689089313c1d407-76d1-405b-b83c-62cdb15a59693. Patiënt nam een ander(e) onderzoeksgeneesmiddel(en) tijdens of na afronding van onderzoek NS 065/NCNP 01 301;18Dutch6851291- Vital signs - Physical examination - Clinical laboratory tests o Hematology and clinical chemistry o Urinalysis o Urine cytology - Antibodies to dystrophin and viltolarsen - 12-lead electrocardiogram (ECG) - Renal ultrasound - Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)True68908720804d475-85ce-4199-828d-2aea39a8dd8b• Ζωτικά σημεία • Κλινική εξέταση • Κλινικές εργαστηριακές εξετάσεις • Αιματολογικές και κλινικές βιοχημικές εξετάσεις • Ανάλυση ούρων • Κυτταρολογική εξέταση ούρων • Αντισώματα στη δυστροφίνη και το viltolarsen • Ηλεκτροκαρδιογράφημα 12 απαγωγών (ΗΚΓ) • Υπερηχογράφημα νεφρών • Εμφανιζόμενα κατά τη θεραπεία ανεπιθύμητα συμβάντα (TEAE) και σοβαρά ανεπιθύμητα συμβάντα (ΣΑΣ)5Greek68908730804d475-85ce-4199-828d-2aea39a8dd8b• Vitale functies • Lichamelijk onderzoek • Klinische laboratoriumtests o Hematologie en klinische chemie o Urineanalyse o Urine cytologie • Antistoffen tegen dystrofine en viltolarsen • 12-afleidingen-elektrocardiogram (ECG) • Echo van de nieren • Behandelingsgerelateerde bijwerkingen (TEAE´s) en ernstige bijwerkingen (SAE´s)18Dutch68908740804d475-85ce-4199-828d-2aea39a8dd8b• Constantes vitales • Exploración física • Análisis clínicos o Hematología y bioquímica clínica o Análisis de orina o Citología urinaria • Anticuerpos contra la distrofina y viltolarsén • ECG de 12 derivaciones • Ecografía renal • AADT y AAG7Spanish68908710804d475-85ce-4199-828d-2aea39a8dd8b• Segni vitali • Esame obiettivo • Esami clinici di laboratorio o Ematologia e chimica clinica o Esame delle urine o Citologia urinaria • Anticorpi anti-distrofina e anti-viltolarsen • Elettrocardiogramma (ECG) a 12 derivazioni • Ecografia renale • Eventi avversi emergenti dal trattamento (TEAE) ed eventi avversi seri (SAE)14Italian6851301Secondary: - Time to Stand Test (TTSTAND) - Time to Run/Walk 10 Meters Test (TTRW) - Six-minute Walk Test (6MWT) - North Star Ambulatory Assessment (NSAA) - Time to Climb 4 Stairs Test (TTCLIMB) - Quantitative muscle strength measured by hand-held dynamometer (elbow extension, elbow flexion, knee extension, and knee flexion on the dominant side only)False689087514e05552-9793-4598-8173-e3dd8c6bd9ce• Δοκιμασία εκτίμησης του χρόνου μετάβασης σε όρθια θέση (TTSTAND) • Δοκιμασία εκτίμησης του χρόνου τρεξίματος/βάδισης απόστασης 10 μέτρων (TTRW) • Δοκιμασία βάδισης 6 λεπτών (6MWT) • Κλίμακα North Star Ambulatory Assessment (NSAA) • Δοκιμασία εκτίμησης του χρόνου ανάβασης 4 σκαλοπατιών (TTCLIMB) • Ποσοτική μέτρηση της μυϊκής δύναμης με τη χρήση φορητού δυναμόμετρου (έκταση αγκώνα, κάμψη αγκώνα, έκταση γόνατος και κάμψη γόνατος μόνο στην κυρίαρχη πλευρά).5Greek689087714e05552-9793-4598-8173-e3dd8c6bd9ce• Test del tempo impiegato per passare dalla posizione supina a quella eretta (TTSTAND) • Test del tempo impiegato per correre/camminare (TTRW) per 10 metri • Test del cammino dei 6 minuti (6MWT) • Valutazione della mobilità North Star (NSAA) • Test del tempo impiegato per salire 4 gradini (TTCLIMB) • Forza muscolare quantitativa misurata mediante dinamometro palmare (estensione e flessione del gomito, estensione e flessione del ginocchio solo sul lato dominante)14Italian689087814e05552-9793-4598-8173-e3dd8c6bd9ce• Tiempo en ponerse de pie (TTSTAND) • Tiempo en recorrer corriendo o caminando 10 metros (TTRW) • Prueba de marcha durante seis minutos (6MWT) • Evaluación de la capacidad de caminar de North Star (NSAA) • Tiempo en subir 4 escalones (TTCLIMB)• Fuerza muscular cuantitativa medida con un dinamómetro manual (extensión del codo, flexión del codo, extensión de la rodilla y flexión de la rodilla en el lado dominante únicamente)7Spanish689087614e05552-9793-4598-8173-e3dd8c6bd9ce• Time to Stand (TTSTAND)-test • Time to Run/Walk 10 Meters (TTRW)-test • Six-minute Walk (6MWT)-test) • North Star Ambulatory Assessment (NSAA) • Time to Climb 4 Stairs (TTCLIMB)-test • Kwantitatieve spiersterkte, gemeten met behulp van een handdynamometer (elleboogstrekking, elleboogbuiging, kniestrekking en kniebuiging, alleen aan de dominante zijde)18Dutch2025-08-202025-08-202021-04-1364557NS Pharma, Inc.24430522244306242424430725252PatientsFalseTrueTrue37625EMEA-002853-PIP01-20acceptable5Diseases [C] - Musculoskeletal Diseases [C05]91987Duchenne Muscular Dystrophy (DMD)True98324True293601PublicRegulatory SubmissionsRS-Advisor-support@medpace.com0034917900565176563Pharmaceutical company10Ns Pharma Inc.TrueTrueORG-100014459Active293602ScientificRegulatory SubmissionsRS-Advisor-support@medpace.com0034917900565176563Pharmaceutical company10Ns Pharma Inc.TrueTrueORG-100014459Active429164314868344267Health care2RapidTrialsFalseFalseORL-000004982Active3360934 Lee Lane, Wainscott,4 Lee LaneWainscott,NY11975840United States006109890660info@rapidtrials.comFalseORL-0000049826724502006109890660info@rapidtrials.com429160266929270858Pharmaceutical company10Medpace Finland OyTrueTrueORG-100009147Active284544Kaikukatu 4 CKaikukatu 4 CHelsinki005302011Finland0034917900565RS-Advisor-Support@medpace.comTrueORG-100009147672436167243710672438116724391267244015Imaging6724412672442367244346724445672445667244670034917900565RS-Advisor-Support@medpace.com429169307215336583Pharmaceutical company10Bml Inc.TrueTrueORG-100050442Active3277955 Chome 21-35 Chome 21-3Shibuya151-0051392Japan008135305190chiken-eigyou-1@bml.co.jpTrueORG-1000504426724554008135305190chiken-eigyou-1@bml.co.jp429165241757245547Hospital/Clinic/Other health care facility8Scout ClinicalFalseTrueORG-100042228Active25760915770 Dallas Pkwy Ste 107515770 Dallas Pkwy Ste 1075Dallas75248-3331840United States0019725903111hello@scoutclinical.comTrueORG-10004222867245115Patient travel services0019725903111hello@scoutclinical.com429166262065265969Pharmaceutical company10Wuxi Apptec Co. Ltd.TrueTrueORG-100012470Active279323228 Fute Middle Rd, Waigaoqiao228 Fute Middle RdWaigaoqiaoShanghai200131156China004008200985wuxiconcierge@wuxiapptec.comTrueORG-1000124706724524004008200985wuxiconcierge@wuxiapptec.com429172241725245515Laboratory/Research/Testing facility9Arup Laboratories Inc.TrueTrueORG-100041750Active257575500 South Chipeta Way500 South Chipeta WaySalt Lake City84108-1221840United States008002422787clientservices@aruplab.comTrueORG-1000417506724584008002422787clientservices@aruplab.com429162262067265971Laboratory/Research/Testing facility9Revvity Omics Sweden ABTrueTrueORG-100047598Active279326Vetenskapsvagen 10Vetenskapsvagen 10Sollentuna191 382028Sweden0017816636900genomics@perkinelmer.comTrueORG-10004759867244840017816636900genomics@perkinelmer.com429173307222336590Pharmaceutical company10Shin Nippon Biomedical Laboratories Ltd.TrueTrueORG-100020905Active32780216-1 Minamiakasaka16-1 MinamiakasakaKainan6420017392Japan0081734838881info@snbl.co.jpTrueORG-10002090567245940081734838881info@snbl.co.jp429167241727245517Non-Pharmaceutical company11Atom International LimitedTrueTrueORG-100042393Active257577Unit 4, Earls House, Earlsway, Team Valley Trading EstateUnit 4Earls HouseEarlswayTeam Valley Trading EstateGatesheadNE11 0RY826United Kingdom00441912496484michelle.eagle@atom-international.orgTrueORG-10004239367245315Patient travel services00441912496484michelle.eagle@atom-international.org429171241744245534Pharmaceutical company10Iqvia Inc.TrueTrueORG-100010622Active2575965827 South Miami Boulevard5827 South Miami BoulevardMorrisville27560-8394840United States0019196017811linda.park@iqvia.comTrueORG-10001062267245780019196017811linda.park@iqvia.com429163314841344240Laboratory/Research/Testing facility9Quest Diagnostics Inc.TrueTrueORG-100013150Active3360611300 East Newport Center Drive1300 East Newport Center DriveDeerfield Beach33442-7727840United States0018666978378DGXGMWclinicalresearchteam@questdiagnostics.comTrueORG-10001315067244940018666978378DGXGMWclinicalresearchteam@questdiagnostics.com429168241762245552Pharmaceutical company10Pci Pharma ServicesTrueTrueORG-100016314Active257615Biotec House, 14c Western Avenue, Bridgend Industrial EstateBiotec House14c Western AvenueBridgend Industrial EstateBridgendCF31 3RT826United Kingdom0017792081819talkfuture@pci.comTrueORG-100016314672454140017792081819talkfuture@pci.com429170314242343638Industry3TRiNDS, LLCTrueFalseORL-000004966Active3353972349 Railroad Street, Suite 14092349 Railroad StreetSuite 1409Pittsburgh15222840United States004122242030info@trinds.comFalseORL-0000049666724564004122242030info@trinds.com429161262050265954Hospital/Clinic/Other health care facility8Illingworth Research Group LimitedFalseTrueORG-100042356Active279308Hazelwood House, Larkwood Way, Tytherington Business ParkHazelwood HouseLarkwood WayTytherington Business ParkMacclesfieldSK10 2XR826United Kingdom00441625617447info@illingworthresearch.comTrueORG-10004235667244715Home health00441625617447info@illingworthresearch.com176563Pharmaceutical company10Ns Pharma Inc.TrueTrueORG-100014459Active173137176563Pharmaceutical company10Ns Pharma Inc.TrueTrueORG-100014459Active183503140 East Ridgewood Avenue Suite 280s140 East Ridgewood Avenue Suite 280sParamus07652-3914840United StatesTrueLOC-100020706TrueCommercial2VIL302 study is a Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD). In line with EMA guidance on disclosure rules, the study meets the criteria of a Category 2 trial. We consider all of our scientific information to be company confidential and highly proprietary within the maximum limits of the redaction Guidelines, and disclosure of such information could harm the company by enabling competitors to developing products that could compete with ours.8890315Diseases [C] - Musculoskeletal Diseases [C05]278736Viltolarsen1Test368186110857392SOLUTION FOR INFUSIONPRD74983631ViltolarsenSOLUTION FOR INFUSIONVILTOLARSENSUB195543NS PHARMA, INC.10857392347540NS PHARMA, INC.Nucleic AcidNucleic AcidVILTOLARSENSUB195543True1TrueEU/3/20/2282mg/Kg milligram(s)/kilogram80mg/Kg milligram(s)/kilogram7680962Antisense OligonucleotidePRD7498363NS-065/NCNP-0111SOLUTION FOR INFUSIONFalseViltolarsenviltolarsenSOLUTION FOR INFUSIONFalse150261179821798275752008Ended2024-02-012024-02-01FalseunwillingCzechia2024-02-27Ended164902023-02-212024-04-11164912023-02-212024-04-11164892023-02-212024-04-11165022023-06-212024-04-11164862023-02-212024-04-11510652023-06-212025-01-102025-02-21733802023-06-212025-01-162026-01-22188772023-06-212024-04-11188782023-06-212024-04-11188922023-06-212023-06-222024-04-11188932023-06-212023-06-222024-04-11True1159982023-06-212025-01-162024-04-11FalseFalseFalseTrue2023-06-212023-06-22False2104417982Under evaluation2024-01-18T15:45:52.8942498517982Authorised2024-02-27T09:58:56.3537699917982Ended2025-02-21T15:40:55.39426104917982Authorised2026-01-22T13:45:24.33126105717982Ended2026-01-22T14:37:10.1441Czechiaacceptable2024-11-25authorized2024-02-272024-02-271978070308795338168Hospital/Clinic/Other health care facility8Fakultni Nemocnice Hradec KraloveFalseTrueORG-100022983Active329485Sokolska 581Sokolska 581Novy Hradec Kralove500 032008Czechia+420495835310ondrej.dostal@fnhk.czTrueORG-1000229831092041OndřejDostál+420495835310ondrej.dostal@fnhk.cz1Neurologická klinikaAuthorised172037179811798175752030Ended2024-02-012024-02-01TruewillingNorway2024-02-27Ended164872022-12-232024-04-11164882022-12-232024-04-11165052022-12-052024-04-11682322022-12-052025-09-092025-11-06188572022-12-232023-10-192024-04-11188582022-12-232023-10-192024-04-11188262022-12-232024-04-11188272022-12-232024-04-11True1053482022-12-052025-09-092024-04-11FalseFalseFalseTrue2022-12-232023-10-19True2104917981Under evaluation2024-01-18T15:45:53.1252502917981Authorised2024-02-27T15:09:57.72212224017981Ended2025-11-06T15:00:33.4191Norwayacceptable2025-01-30authorized2024-02-272024-02-2721111381314582343980Hospital/Clinic/Other health care facility8Oslo University Hospital HFFalseTrueORG-100021349Active335776Sognsvannsveien 20Sognsvannsveien 20Oslo03722030Norway004723074605seawal@ous-hf.noTrueORG-1000213491242160SeanWallace004723074605seawal@ous-hf.no1Neuromuscular UnitAuthorised170857179801798075752029Ended2024-02-012024-02-01FalseunwillingNetherlands2024-02-27Ended164922021-09-292024-04-11164932021-09-292024-04-11165062021-09-272024-04-11626872021-09-272025-07-312025-08-13188692021-09-292023-09-072024-04-11188702021-09-292023-09-072024-04-11188282021-09-292024-04-11188292021-09-292024-04-11True942172021-09-272025-07-312024-04-11FalseFalseFalseTrue2021-09-292023-09-07False2104817980Under evaluation2024-01-18T15:45:53.0792503117980Authorised2024-02-27T15:29:42.0458990917980Ended2025-08-13T09:02:40.3071Netherlandsacceptable2025-01-15authorized2024-02-272024-02-2791104182314741344140Hospital/Clinic/Other health care facility8Leids Universitair Medisch Centrum (LUMC)FalseTrueORG-100014145Active335945Einthovenweg 20Einthovenweg 20Leiden2333 ZC2029Netherlands0031715262197E.H.Niks@lumc.nlTrueORG-1000141451234271ErikNiks0031715262197E.H.Niks@lumc.nl1Neuromuscular Unit1104181314671344070Hospital/Clinic/Other health care facility8Radboud universitair medisch centrum / RADBOUDUMCFalseTrueORG-100031688Active335871Geert Grooteplein Zuid 10Geert Grooteplein Zuid 10Nijmegen6525 GA2029Netherlands0031650176591Saskia.Houwen@radboudumc.nlTrueORG-1000316881234270SaskiaHouwen-Opstal0031650176591Saskia.Houwen@radboudumc.nl1Neuromuscular UnitAuthorised150273179781797875752018Ended2024-02-012024-02-01FalseunwillingItaly2024-03-01Ended165082021-06-072024-04-11164962021-06-152024-04-11164972021-06-152024-04-11581852021-06-072025-05-162025-06-06581842021-06-072025-05-162025-06-06188322021-06-152024-04-11188332021-06-152024-04-11188732021-06-152023-06-192024-04-11188742021-06-152023-06-192024-04-11True857372021-06-072025-05-162024-04-11FalseFalseFalseTrue2021-06-152023-06-19False2104617978Under evaluation2024-01-18T15:45:52.9882549117978Authorised2024-03-01T09:31:36.0418465317978Ended2025-06-06T13:25:22.1571Italyacceptable2024-12-03authorized2024-03-012024-03-014978159314856344255Hospital/Clinic/Other health care facility8Fondazione Policlinico Universitario Agostino Gemelli IRCCSFalseTrueORG-100014358Active336078Largo Francesco Vito 1Largo Francesco Vito 1Rome001682018Italy0630154860mercuri.trial@policlinicogemelli.itTrueORG-1000143581092143EugenioMercuri0630154860mercuri.trial@policlinicogemelli.it2neuropsychiatryAuthorised154524179771797775752014Ended2024-02-012024-02-01FalseunwillingGreece2024-04-09Ended164982022-05-102024-04-11164992022-05-102024-04-11165092022-05-062024-04-11578302022-05-062025-03-102025-06-02188752022-05-102023-04-122024-04-11188762022-05-102023-04-122024-04-11188342022-05-102024-04-11188352022-05-102024-04-11True850172022-05-062025-03-102024-04-11FalseFalseFalseTrue2022-05-102023-04-12False2968317977Authorised2024-04-09T11:07:08.552104517977Under 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(DMD)Netherlands:4Germany:4France:4Italy:4Belgium:4Czechia:2Spain:2Sweden:2Poland:205/12/2023BE: 11/12/2023, ES: 25/03/2026, SE: 10/03/2026, PL: 27/03/2026, FR: 06/12/2023, CZ: 20/04/2026, DE: 05/12/2023, NL: 05/12/2023, IT: 11/12/2023Diseases [C] - Musculoskeletal Diseases [C05]Italfarmaco S.p.A.Pharmaceutical companyTherapeutic confirmatory (Phase III)•Change from baseline of Peak Expiratory Flow percent predicted (PEF%p) at 18 months of treatment of givinostat compared to placebo group • Change from baseline of Forced Vital Capacity percent predicted (FVC%p) at 18 months of treatment of givinostat compared to placebo group • Cumulative loss of PUL items over 18 months of treatment of givinostat compared to placebo group, •Type incidence and severity of TEAEs •Proportion of patients experiencing TEAEs (baseline to EOS/F-Up) • Changes in vital signs, lab tests, ECG, ECHO •Time to assisted ventilation, rate and severity of respiratory infection and duration and use of antibiotics of 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publication)PDF3000483.00.01Netherlands59242023-12-05T09:10:24.7992026-05-04T13:18:10.5734Germany59202023-12-05T11:30:37.2432026-05-04T13:18:10.5734France59192023-12-06T16:59:58.6322026-03-17T15:02:53.684Italy59212023-12-11T09:29:50.8032026-05-04T13:18:10.5734Belgium59172023-12-11T15:24:37.8822025-02-14T14:19:06.2574Czechia558082026-03-19T15:06:34.5892026-04-20T11:20:06.4012Spain558092026-03-25T10:12:18.2042026-03-25T10:12:18.2042Sweden558102026-03-10T09:52:27.3412026-03-10T09:52:27.3412Poland558112026-03-27T12:19:22.8982026-03-27T12:19:22.89825917BelgiumSTART_OF_TRIAL2024-05-17START_OF_RECRUITMENT2024-10-255919FranceSTART_OF_TRIAL2024-05-16START_OF_RECRUITMENT2024-05-275920GermanySTART_OF_TRIAL2025-04-07START_OF_RECRUITMENT2025-04-285921ItalySTART_OF_TRIAL2024-07-15START_OF_RECRUITMENT2024-08-295924NetherlandsSTART_OF_TRIAL2024-02-09START_OF_RECRUITMENT2024-02-1955808Czechia55809Spain55810Sweden55811PolandSB-165322024-03-012024-02-162024-05-282024-05-28100000000337Belgium2002BEBELTrue100000000403Germany2013DEDEUTrue100000000430Italy2018ITITATrue100000000556United Kingdom826GBGBRTrue100000000478Netherlands2029NLNLDTrue100000000354Canada124CACANTrue100000000395France2012FRFRATrueOtherDescription of serious breach and impacts on trial is provided in the attachment separately.Description of actions taken and planned is provided in the attachment separately.TrueAlthough no subjects were screened at time of the incident, Italfarmaco assessed the potential unblinding issue may have impact on the data integrity and the double-blind fashion of the study could not be ensure without further remediation.BelgiumFranceGermanyItalyNetherlands7716079684PHARMACEUTICAL_COMPANY10Italfarmaco S.p.A.ORG-10000011880910Via Dei Lavoratori 54Via Dei Lavoratori 54Cinisello Balsamo200922018ItalyLOC-100004430Sponsor (commercial)169797173197PHARMACEUTICAL_COMPANY10Alira HealthORG-10003030317984512 Rue Charlot12 Rue CharlotParis750032012FranceLOC-100050379OtherStatistical analysi, data management2ProtocolThis is last update to the Serious Breach. With this information the sponsor considers this case as closed.2023-503521-19-004Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular DystrophyDSC/14/2357/5009/02/2024Duchenne muscular dystrophy (DMD)Netherlands:4Germany:4France:4Italy:4Belgium:4Czechia:2Spain:2Sweden:2Poland:205/12/2023BE: 11/12/2023, ES: 25/03/2026, SE: 10/03/2026, PL: 27/03/2026, FR: 06/12/2023, CZ: 20/04/2026, DE: 05/12/2023, NL: 05/12/2023, IT: 11/12/2023Diseases [C] - Musculoskeletal Diseases [C05]Italfarmaco S.p.A.Pharmaceutical companyTherapeutic confirmatory (Phase III)•Change from baseline of Peak Expiratory Flow percent predicted (PEF%p) at 18 months of treatment of givinostat compared to placebo group • Change from baseline of Forced Vital Capacity percent predicted (FVC%p) at 18 months of treatment of givinostat compared to placebo group • Cumulative loss of PUL items over 18 months of treatment of givinostat compared to placebo group, •Type incidence and severity of TEAEs •Proportion of patients experiencing TEAEs (baseline to EOS/F-Up) • Changes in vital signs, lab tests, ECG, ECHO •Time to assisted ventilation, rate and severity of respiratory infection and duration and use of antibiotics of givinostat to placebo group •Time to onset of diarrhoea •Height and weight Z-scoresPLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspension, ITF23570-17 yearsMale3120Change of PUL total score at 18 months of treatment of givinostat compared to placebo groupNo04/05/202605/05/20262023-503521-19-00Authorised2024-02-092023-12-05T09:10:24.7992026-05-05T03:35:51.992122893412701948100000000354Canada124CACANTrue100000000556United Kingdom826GBGBRTrue5275031N/AN/AN/APLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspensionN/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspensionn/aN/A527502111133213ORAL SUSPENSIONPRD47976781ITF2357ORAL SUSPENSIONITF2357GIVINOSTATSUB189959GIVINOSTATITALFARMACO SPA11133213337329ITALFARMACO SPAChemicalChemicalGIVINOSTATSUB189959False1TrueOrfan EU/3/12/1009mg milligram(s)93.4mg milligram(s)47073183histone deacetylase (HDAC) inhibitorPRD4797678ITF235711ORAL USETrueITF2357givinostatORAL SUSPENSIONRandomised, double blind, placebo controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular Dystrophy1054708721953bec-a517-448f-a694-d5b25028ade1Étude multicentrique, randomisée, en double aveugle, contrôlée par placebo, visant à évaluer l’efficacité, la sécurité d’emploi et la tolérance du givinostat chez des patients non ambulatoires atteints de dystrophie musculaire de Duchenne10French1054708621953bec-a517-448f-a694-d5b25028ade1Estudio multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia, la seguridad y la tolerabilidad de givinostat en pacientes no ambulantes con distrofia muscular de Duchenne7Spanish1054708921953bec-a517-448f-a694-d5b25028ade1Gerandomiseerd, dubbelblind, placebogecontroleerd onderzoek in meerdere centra ter beoordeling van de werkzaamheid, veiligheid en verdraagbaarheid van givinostat bij niet-ambulante patiënten met de ziekte van Duchenne37Dutch (Netherlands)1054708821953bec-a517-448f-a694-d5b25028ade1Wieloośrodkowe, randomizowane badanie prowadzone metodą podwójnie ślepej próby z grupą kontrolną otrzymującą placebo, oceniające skuteczność, bezpieczeństwo stosowania i tolerancję giwinostatu u niechodzących pacjentów z dystrofią mięśniową Duchenne’a19PolishRandomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular Dystrophy105471268c03ae07-a5a8-45f2-a13e-7f55f60cad7bÉtude multicentrique, randomisée, en double aveugle, contrôlée par placebo, visant à évaluer l’efficacité, la sécurité d’emploi et la tolérance du givinostat chez des patients non ambulatoires atteints de dystrophie musculaire de Duchenne34French (France)105471278c03ae07-a5a8-45f2-a13e-7f55f60cad7bGerandomiseerd, dubbelblind, placebogecontroleerd onderzoek in meerdere centra ter beoordeling van de werkzaamheid, veiligheid en verdraagbaarheid van givinostat bij niet-ambulante patiënten met de ziekte van Duchenne37Dutch (Netherlands)105471318c03ae07-a5a8-45f2-a13e-7f55f60cad7bRandomizovaná, dvojitě zaslepená, placebem kontrolovaná, multicentrická studie hodnotící účinnost, bezpečnost a snášenlivost givinostatu u imobilních pacientů s Duchennovou svalovou dystrofií2Czech105471308c03ae07-a5a8-45f2-a13e-7f55f60cad7bRandomiserad, dubbelblind, placebokontrollerad multicenterstudie för att utvärdera effekt, säkerhet och tolerabilitet av givinostat hos patienter med Duchennes muskeldystrofi utan gångförmåga.24Swedish105471298c03ae07-a5a8-45f2-a13e-7f55f60cad7bWieloośrodkowe, randomizowane badanie prowadzone metodą podwójnie ślepej próby z grupą kontrolną otrzymującą placebo, oceniające skuteczność, bezpieczeństwo stosowania i tolerancję giwinostatu u niechodzących pacjentów z dystrofią mięśniową Duchenne’a19Polish105471288c03ae07-a5a8-45f2-a13e-7f55f60cad7bEstudio multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia, la seguridad y la tolerabilidad de givinostat en pacientes no ambulantes con distrofia muscular de Duchenne7SpanishDSC/14/2357/50523603U1111-1295-1799523602NCT05933057523604IND126598FDA6False52This is phase III to evaluate safety efficacy and tolerability116734132405Duchenne muscular dystrophy (DMD)105469762f156e74-e92b-414e-b4c9-94b097130bc7Distrofia muscular de Duchenne (DMD)7Spanish105469742f156e74-e92b-414e-b4c9-94b097130bc7Ziekte van Duchenne37Dutch (Netherlands)105469752f156e74-e92b-414e-b4c9-94b097130bc7Dystrofia mięśniowa Duchenne’a (DMD)19PolishTrue63849985385000338500213tolerability38499973850034385001To demonstrate the efficacy of givinostat in reducing muscle decline in non-ambulant DMD patients.10547125a09981d0-04c1-4f07-a997-dd546e189303Mostrar la eficacia de givinostat en la reducción del deterioro muscular en pacientes no ambulantes con DMD7Spanish10547124a09981d0-04c1-4f07-a997-dd546e189303Démontrer l’efficacité du givinostat dans la réduction du déclin musculaire chez les patients atteints de DMD non ambulatoires10French10547122a09981d0-04c1-4f07-a997-dd546e189303Wykazanie skuteczności giwinostatu w zakresie zmniejszania zaniku mięśni u niechodzących pacjentów z DMD19Polish10547123a09981d0-04c1-4f07-a997-dd546e189303Het aantonen van de werkzaamheid van givinostat wat betreft het verminderen van spierafname bij niet-ambulante DMD-patiënten.37Dutch (Netherlands)4495451To evaluate the safety and tolerability of givinostat in non-ambulant DMD patients105471345052053a-d98f-489c-8f1d-77373e7e8a65Évaluer la sécurité d’emploi et la tolérance du givinostat chez les patients atteints de DMD non ambulatoires.10French105471355052053a-d98f-489c-8f1d-77373e7e8a65Evaluar la seguridad y la tolerabilidad de givinostat en pacientes no ambulantes con DMD7Spanish105471335052053a-d98f-489c-8f1d-77373e7e8a65Ocena bezpieczeństwa stosowania i tolerancji giwinostatu u niechodzących pacjentów z DMD19Polish105471325052053a-d98f-489c-8f1d-77373e7e8a65Het beoordelen van de veiligheid en verdraagbaarheid van givinostat bij niet-ambulante DMD-patiënten.37Dutch (Netherlands)4495462To further investigate the efficacy of givinostat in non-ambulant DMD patients.10547138a4d1c397-50f5-4aa3-8c1c-1c80e59993e5Het verder onderzoeken van de werkzaamheid van givinostat bij niet-ambulante DMD-patiënten.37Dutch (Netherlands)10547136a4d1c397-50f5-4aa3-8c1c-1c80e59993e5Etudier plus en détails l’efficacité du givinostat chez les patients atteints de DMD non ambulatoires10French10547139a4d1c397-50f5-4aa3-8c1c-1c80e59993e5Dalsza ocena skuteczności giwinostatu u niechodzących pacjentów z DMD19Polish10547137a4d1c397-50f5-4aa3-8c1c-1c80e59993e5Investigar más a fondo la eficacia de givinostat en pacientes no ambulantes con DMD7Spanish8581121Children and adolescent males aged ≥ 9 to <18 years at screening (patients ≥ 18 years of age at screening will not be enrolled into the study)10547093f2cce6eb-0d91-4d58-83af-56aff0dbb185Enfants et adolescents de sexe masculin âgés de ≥ 9 à < 18 ans à la sélection (les patients âgés de ≥ 18 ans à la sélection ne seront pas inclus dans l’étude)10French10547092f2cce6eb-0d91-4d58-83af-56aff0dbb185Dzieci i młodzi mężczyźni w wieku od ≥9 do <18 lat podczas badań przesiewowych (pacjenci w wieku ≥18 lat podczas badań przesiewowych nie zostaną włączeni do badania)19Polish10547090f2cce6eb-0d91-4d58-83af-56aff0dbb185Niños y adolescentes de edad ≥9 a <18 años en la selección (no se incluirá en el estudio a los pacientes de edad ≥18 años en la selección)7Spanish10547091f2cce6eb-0d91-4d58-83af-56aff0dbb185Kinderen en jongeren van ≥ 9 tot < 18 jaar bij de screening (patiënten ≥ 18 jaar bij de screening worden niet in het onderzoek ingeschreven37Dutch (Netherlands)8581132Are able to give informed assent and/or consent in writing signed by the patient and/or parent/legal guardian (according to local regulations)10547097fe50ceb9-5f6e-47ea-b719-8be85d356223Être en mesure de donner un assentiment éclairé et/ou un consentement éclairé écrit signé par le patient et/ou parent/tuteur légal (conformément à la réglementation locale)10French10547095fe50ceb9-5f6e-47ea-b719-8be85d356223Zdolność wyrażenia świadomej zgody i/lub zgody na piśmie podpisanej przez pacjenta i/lub jego rodzica/opiekuna prawnego (według miejscowych wymogów)19Polish10547096fe50ceb9-5f6e-47ea-b719-8be85d356223Capacidad de dar el asentimiento o consentimiento informado por escrito firmado por el paciente o el progenitor/tutor legal (de acuerdo con la normativa local)7Spanish10547094fe50ceb9-5f6e-47ea-b719-8be85d356223In staat zijn om schriftelijk geïnformeerde instemming en/of toestemming te geven, ondertekend door de patient en/of ouder/wettelijke voogd (volgens de lokale regelgeving)37Dutch (Netherlands)8581143A genetic diagnosis of DMD10547101298149b7-7e33-4628-9d0b-1812454b975bEen genetische diagnose van DMD hebben37Dutch (Netherlands)10547099298149b7-7e33-4628-9d0b-1812454b975bDiagnóstico genético de DMD7Spanish10547100298149b7-7e33-4628-9d0b-1812454b975bDiagnostic génétique de DMD10French10547098298149b7-7e33-4628-9d0b-1812454b975bRozpoznanie genetyczne DMD19Polish8581154Non-ambulant defined as being wheelchair bound and: a. Unable to perform the 10-meter walk/run test (10MWT), or b. Unable to complete the 10MWT in 30 seconds or less, without any support or devices10547102bcbbfe6b-aa3b-4571-a316-8f0ca8e98b76Niet-ambulant wordt gedefinieerd als rolstoelgebonden en: a. Niet in staat om de 10-meter loop-/rentest (10MWT) uit te voeren, of b. Niet in staat om de 10MWT in 30 seconden of minder te voltooien, zonder ondersteuning of hulpmiddelen37Dutch (Netherlands)10547104bcbbfe6b-aa3b-4571-a316-8f0ca8e98b76Sin ambulación, esto es, en silla de ruedas y: a. sin capacidad de realizar la prueba de marcha/carrera de 10 metros (PM10m), o b. sin capacidad de completar la PM10m en un tiempo igual o inferior a 30 segundos sin ningún tipo de apoyo ni aparato7Spanish10547105bcbbfe6b-aa3b-4571-a316-8f0ca8e98b76„Osoba niechodząca” zdefiniowana jako osoba poruszająca się na wózku inwalidzkim, która: a. Nie jest w stanie wykonać testu 10-metrowego marszu/biegu (10MWT) lub b. Nie jest w stanie ukończyć testu 10MWT w czasie co najwyżej 30 sekund, bez wsparcia lub urządzeń pomocniczych19Polish10547103bcbbfe6b-aa3b-4571-a316-8f0ca8e98b76Être non ambulatoires, c’est-à-dire être en fauteuil roulant et : a. Ne pas être capables d’effectuer le test de marche/course de 10 mètres (10MWT), ou b. Ne pas être capables d’effectuer le 10MWT en 30 secondes ou moins, sans aucune aide ou dispositif10French8581165Performance of the Upper Limb test (PUL version 2.0) entry item scores 3 to 610547107adba8060-cd8d-4ef9-96ba-39b194cc3ec3Prestaties van de test van de bovenste ledematen (PUL versie 2.0) instapitemscores 3 tot 637Dutch (Netherlands)10547106adba8060-cd8d-4ef9-96ba-39b194cc3ec3Puntuación en los ítems de entrada de la escala de rendimiento de las extremidades superiores (PUL, versión 2.0) de 3 a 67Spanish10547109adba8060-cd8d-4ef9-96ba-39b194cc3ec3Wstępne wyniki w pozycjach testu sprawności kończyn górnych (skala PUL wersja 2.0) wynoszą od 3 do 619Polish10547108adba8060-cd8d-4ef9-96ba-39b194cc3ec3Scores d’entrée de 3 à 6 au test de performance des membres supérieurs (version PUL 2.0)10French8581176If on medication for DMD-associated cardiomyopathy, stable for ≥1 month immediately prior to start of study treatment1054711256b76b80-c0c1-4394-8e8c-10e5905433d6Si les patients sont sous traitement pour une cardiomyopathie associée à la DMD (p. ex. inhibiteur de l’ECA, β-bloquant, diurétique), ils doivent être stables depuis ≥ 1 mois immédiatement avant le début du traitement à l’étude, le cas échéant10French1054711356b76b80-c0c1-4394-8e8c-10e5905433d6W przypadku przyjmowania leków na kardiomiopatię związaną z DMD stała dawka przez ≥1 miesiąc bezpośrednio przed rozpoczęciem leczenia w ramach badania19Polish1054711156b76b80-c0c1-4394-8e8c-10e5905433d6En caso de recibir medicamentos para la miocardiopatía asociada a la DMD, en tratamiento estable durante un periodo ≥1 mes inmediatamente antes del inicio del tratamiento del estudio7Spanish1054711056b76b80-c0c1-4394-8e8c-10e5905433d6Bij gebruik van medicatie voor DMD-geassocieerde cardiomyopathie, stabiel gedurende ≥ 1 maand onmiddellijk voorafgaand aan de start van de onderzoeksbehandeling37Dutch (Netherlands)8581187Stable corticosteroids, defined as: a. Receiving systemic corticosteroids for a minimum of 6 months immediately prior to start of study treatment b. No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 6 months immediately prior to start of study treatment1054711422817584-0a45-4069-a277-9937bdffc57aKortykosteroidy w stałej dawce, zdefiniowane jako: a. Przyjmowanie kortykosteroidów o działaniu ogólnoustrojowym przez co najmniej 6 miesięcy bezpośrednio przed rozpoczęciem leczenia w ramach badania b. Brak istotnych zmian dawki lub schematu dawkowania (z wyjątkiem dostosowań wynikających ze zmiany masy ciała) przez co najmniej 6 miesięcy bezpośrednio przed rozpoczęciem leczenia w ramach badania19Polish1054711622817584-0a45-4069-a277-9937bdffc57aTratamiento estable con corticoesteroides, definido de la siguiente manera: a. Haber estado recibiendo corticoesteroides sistémicos durante un mínimo de 6 meses inmediatamente antes del inicio del tratamiento del estudio b. Ausencia de cambios significativos en la dosis o la pauta posológica (excepto ajustes debidos a cambios en el peso corporal) durante un mínimo de 6 meses inmediatamente antes del inicio del tratamiento del estudio7Spanish1054711522817584-0a45-4069-a277-9937bdffc57aCorticoïdes stables, définis comme suit : a. Administration de corticoïdes systémiques pendant au moins 6 mois immédiatement avant le début du traitement à l’étude b. Aucune modification significative de la dose ou du schéma posologique (à l’exception des ajustements dus à une modification du poids corporel) pendant un minimum de 6 mois immédiatement avant le début du traitement à l’étude10French1054711722817584-0a45-4069-a277-9937bdffc57aStabiele corticosteroïden, gedefinieerd als: a. Het krijgen van systemische corticosteroïden gedurende minimaal 6 maanden onmiddellijk voorafgaand aan de start van de onderzoeksbehandeling b. Geen significante verandering in dosis of doseringsschema (behalve aanpassingen als gevolg van verandering in lichaamsgewicht) gedurende minimaal 6 maanden onmiddellijk voorafgaand aan de start van de onderzoeksbehandeling37Dutch (Netherlands)8581198Willing to use adequate contraception. Contraceptive methods must be used from randomisation visit through 3 months after the last dose of study drug.105471194ff8af40-f0f6-4a76-aa4e-0e6c472da682Bereid om adequate anticonceptie te gebruiken. Anticonceptiemethoden moeten worden gebruikt vanaf het randomisatiebezoek tot en met 3 maanden na de laatste dosis onderzoeksmiddel37Dutch (Netherlands)105471204ff8af40-f0f6-4a76-aa4e-0e6c472da682Disposición a utilizar métodos anticonceptivos adecuados. Se deben utilizar métodos anticonceptivos desde la visita de aleatorización hasta 3 meses después de la última dosis del fármaco del estudio7Spanish105471184ff8af40-f0f6-4a76-aa4e-0e6c472da682Chęć stosowania odpowiedniej antykoncepcji. Należy stosować metody antykoncepcji w okresie od wizyty randomizacyjnej do upływu 3 miesięcy po przyjęciu ostatniej dawki badanego leku.19Polish105471214ff8af40-f0f6-4a76-aa4e-0e6c472da682Être disposés à utiliser une méthode de contraception adéquate. Des méthodes contraceptives efficaces doivent être utilisées depuis la visite de randomisation jusqu’à 3 mois après la dernière dose du médicament à l’étude9Finnish14677121Exposure to another investigational drug within 3 months prior to start of study treatment10546993c01ed79c-b669-43e6-9b71-a62d233ad634Exposición a otro medicamento en investigación en los 3 meses anteriores al inicio del tratamiento del estudio7Spanish10546990c01ed79c-b669-43e6-9b71-a62d233ad634Enfants et adolescents de sexe masculin âgés de ≥ 9 à < 18 ans à la sélection (les patients âgés de ≥ 18 ans à la sélection ne seront pas inclus dans l’étude)10French10546992c01ed79c-b669-43e6-9b71-a62d233ad634Blootstelling aan een ander onderzoeksmiddel binnen 3 maanden voorafgaand aan de start van de onderzoeksbehandeling37Dutch (Netherlands)10546991c01ed79c-b669-43e6-9b71-a62d233ad634Narażenie na inny lek eksperymentalny w ciągu 3 miesięcy przed rozpoczęciem leczenia w ramach badania19Polish146771310Episode of respiratory failure within the 8 weeks prior to screening105469949a55bc86-0a61-4e03-89d4-334aeb231245Episode van ademhalingsfalen binnen 8 weken voorafgaand aan de screening37Dutch (Netherlands)105469979a55bc86-0a61-4e03-89d4-334aeb231245Episodio de insuficiencia respiratoria en las 8 semanas anteriores a la selección7Spanish105469969a55bc86-0a61-4e03-89d4-334aeb231245Épisode d’insuffisance respiratoire dans les 8 semaines précédant la sélection10French105469959a55bc86-0a61-4e03-89d4-334aeb231245Epizod niewydolności oddechowej w ciągu 8 tygodni przed badaniami przesiewowymi19Polish146771411Symptomatic cardiomyopathy or heart failure and/or left ventricular ejection fraction <45%10547001b3863967-036b-4c17-88c6-75d823730822Cardiomyopathie symptomatique ou insuffisance cardiaque et/ou fraction d’éjection ventriculaire gauche < 45 %10French10546998b3863967-036b-4c17-88c6-75d823730822Symptomatische cardiomyopathie of hartfalen en/of linkerventrikel ejectiefractie <45%37Dutch (Netherlands)10547000b3863967-036b-4c17-88c6-75d823730822Kardiomiopatia objawowa lub niewydolność serca i/lub wartość frakcji wyrzutowej lewej komory <45%19Polish10546999b3863967-036b-4c17-88c6-75d823730822Miocardiopatía sintomática o insuficiencia cardíaca o fracción de eyección ventricular izquierda <45 %7Spanish146771512Baseline corrected QT interval using Fredericia’s formula (QTcF) >450 msec (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (eg, heart failure, hypokalaemia, or family history of long QT syndrome)1054700532b9f55d-5114-4db6-8cad-e5c5edececc0Intervalle QT corrigé selon la formule de Fredericia (QTcF) > 450 msec (moyenne de 3 mesures consécutives à 5 minutes d’intervalle) ou antécédents de facteurs de risque supplémentaires de torsades de pointes (p. ex., insuffisance cardiaque, hypokaliémie ou antécédents familiaux de syndrome du QT long) à l’entrée dans l’étude10French1054700232b9f55d-5114-4db6-8cad-e5c5edececc0Skorygowany odstęp QT według wzoru Fredericia (QTcF) >450 ms (jako średnia z 3 kolejnych odczytów wykonanych w odstępie 5 minut) w punkcie wyjściowym lub występowanie w wywiadzie dodatkowych czynników ryzyka dla częstoskurczu komorowego typu torsades de pointes (np. niewydolność serca, hipokaliemia lub występowanie w rodzinie zespołu długiego odstępu QT)19Polish1054700432b9f55d-5114-4db6-8cad-e5c5edececc0Gecorrigeerd QT-interval bij de baseline met behulp van Fredericia’s formule (QTcF) > 450 msec (als gemiddelde van 3 opeenvolgende metingen met een tussentijd van 5 minuten) of een voorgeschiedenis van bijkomende risicofactoren voor torsade de pointe (bijv. hartfalen, hypokaliëmie of familiegeschiedenis van lang QT-syndroom)37Dutch (Netherlands)1054700332b9f55d-5114-4db6-8cad-e5c5edececc0Intervalo QT corregido mediante la fórmula de Fridericia (QTcF) al inicio >450 ms (como la media de 3 lecturas consecutivas separadas por 5 minutos) o antecedentes de factores de riesgo adicionales de taquicardia ventricular en entorchado (p. ej., insuficiencia cardíaca, hipopotasemia o antecedentes familiares de síndrome del QT largo)7Spanish146771613Major surgical procedure (including scoliosis surgery) planned within 1 year of the start of study treatment10547007675738fd-2028-40d8-9432-8f53979fd5cfPoważny zabieg chirurgiczny (w tym operacja skoliozy) zaplanowany w ciągu 1 roku od rozpoczęcia leczenia w ramach badania19Polish10547008675738fd-2028-40d8-9432-8f53979fd5cfIntervención de cirugía mayor (incluida la cirugía de escoliosis) prevista en el plazo de 1 año con respecto al inicio del tratamiento del estudio7Spanish10547009675738fd-2028-40d8-9432-8f53979fd5cfIntervention chirurgicale majeure (y compris chirurgie de la scoliose) prévue dans l’année précédant le début du traitement à l’étude10French10547006675738fd-2028-40d8-9432-8f53979fd5cfGrote chirurgische ingreep (waaronder scoliose-operatie) gepland binnen 1 jaar vóór de start van de onderzoeksbehandeling37Dutch (Netherlands)146771714Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the Investigator might impact respiratory function105470128cb5b891-ca68-43d2-8c99-3a03026967d2Niedostatecznie kontrolowana astma lub podstawowa choroba płuc, taka jak zapalenie oskrzeli, rozstrzenie oskrzeli, rozedma płuc, nawracające zapalenie płuc, która w opinii badacza może mieć wpływ na czynność układu oddechowego19Polish105470108cb5b891-ca68-43d2-8c99-3a03026967d2Asthme mal contrôlé ou maladie pulmonaire sous-jacente telle que bronchite, bronchectasie, emphysème, pneumonie récurrente qui, de l’avis de l’investigateur, pourrait avoir un impact sur la fonction respiratoire10French105470138cb5b891-ca68-43d2-8c99-3a03026967d2Asma mal controlada o neumopatía subyacente, como bronquitis, bronquiectasia, enfisema o neumonía recurrente que, en opinión del investigador, podría afectar a la función respiratoria7Spanish105470118cb5b891-ca68-43d2-8c99-3a03026967d2Slecht gecontroleerde astma of onderliggende longziekte zoals bronchitis, bronchiëctasie, emfyseem, herhaalde longontsteking die naar het oordeel van de onderzoeker invloed kan hebben op de ademhalingsfunctie37Dutch (Netherlands)146771815Platelets, white blood cells and/or haemoglobin < lower limit of normal (LLN) at screening105470149ffad6d8-b45d-43f4-9619-b7ebc1592170Plaquetas, leucocitos o hemoglobina 7Spanish105470169ffad6d8-b45d-43f4-9619-b7ebc1592170Płytki krwi, krwinki białe i/lub hemoglobina < dolnej granicy normy (DGN) podczas badań przesiewowych19Polish105470159ffad6d8-b45d-43f4-9619-b7ebc1592170Bloedplaatjes, witte bloedcellen en/of hemoglobine < ondergrens van normaal (LLN) bij de screening37Dutch (Netherlands)105470179ffad6d8-b45d-43f4-9619-b7ebc1592170Plaquettes, globules blancs et/ou hémoglobine < limite inférieure de la normale (LIN) à la sélection (Remarque : en cas de résultats anormaux < LIN aux tests de laboratoire de la sélection, la numération plaquettaire, les globules blancs et l’hémoglobine seront répétés une fois ; si le résultat du test répété est toujours < LIN, le patient devra être exclu)10French146771916Fasting triglycerides >300 mg/dL (3.42 mmol/L) at screening10547019a67934c1-75f4-420d-b9e9-675ff6465d7fNuchtere triglyceriden > 300 mg/dl (3,42 mmol/l) bij de screening37Dutch (Netherlands)10547021a67934c1-75f4-420d-b9e9-675ff6465d7fStężenie trójglicerydów na czczo >300 mg/dl (3,42 mmol/l) podczas badań przesiewowych19Polish10547020a67934c1-75f4-420d-b9e9-675ff6465d7fTriglicéridos en ayunas >300 mg/dl (3,42 mmol/l) en la selección7Spanish10547018a67934c1-75f4-420d-b9e9-675ff6465d7fTriglycérides à jeun > 300 mg/dl (3,42 mmol/l) à la sélection (Remarque : si la valeur est > 300 mg/dl, le dosage des triglycérides sera répété une fois ; si le résultat du test répété est toujours > 300 mg/dl, le patient devra être exclu)10French146772017Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, >1.5 × upper limit of normal [ULN]), unless secondary to Gilbert disease or pattern consistent with Gilbert disease10547023b7f26101-1b7d-41c0-874a-7b9ccda5de95Maladie ou insuffisance hépatique actuelle ou antérieure, y compris, mais sans s’y limiter, un taux de bilirubine totale élevé à l’entrée dans l’étude (c.-à-d. > 1,5 × la limite supérieure de la normale [LSN]), à moins qu’il ne soit secondaire à la maladie de Gilbert ou qu’il ne corresponde à la maladie de Gilbert10French10547024b7f26101-1b7d-41c0-874a-7b9ccda5de95Huidige of voorgeschiedenis van leverziekte of -insufficiëntie, inclusief maar niet beperkt tot een baseline van verhoogd totaal bilirubine (d.w.z. > 1,5 × bovengrens van normaal [ULN]), tenzij secundair aan de ziekte van Gilbert of een patroon dat consistent is met de ziekte van Gilbert37Dutch (Netherlands)10547022b7f26101-1b7d-41c0-874a-7b9ccda5de95Występująca obecnie lub w przeszłości choroba lub upośledzenie czynności wątroby, w tym między innymi podwyższone stężenie bilirubiny całkowitej (tj. >1,5 × górna granica normy [GGN]) w punkcie wyjściowym, chyba że w następstwie choroby Gilberta lub wzorca zgodnego z chorobą Gilberta19Polish10547025b7f26101-1b7d-41c0-874a-7b9ccda5de95Antecedentes o presencia de enfermedad o trastorno hepático, entre otros, concentración de bilirrubina total elevada al inicio (es decir, >1,5 × límite superior de la normalidad [LSN]), a menos que sea secundaria a la enfermedad de Gilbert o un patrón coherente con la enfermedad de Gilbert7Spanish146772118Inadequate renal function, as defined by serum Cystatin C result >2 × ULN105470271357ede9-d146-442e-9468-346439a24a8fFonction rénale inadéquate, telle que définie par un résultat de cystatine C sérique > 2 × LSN (Remarque : si la valeur est > 2 × LSN, le test de cystatine C sérique sera répété une fois ; si le résultat du test répété est toujours > 2 × LSN, le patient devra être exclu)9Finnish105470281357ede9-d146-442e-9468-346439a24a8fNiedostateczna czynność nerek, zdefiniowana jako wynik stężenia cystatyny C w surowicy >2 × GGN19Polish105470261357ede9-d146-442e-9468-346439a24a8fOnvoldoende nierfunctie, zoals gedefinieerd met een cystatine C-serumresultaat > 2 × ULN37Dutch (Netherlands)105470291357ede9-d146-442e-9468-346439a24a8fFunción renal inadecuada, definida por un resultado de cistatina C sérica >2 × LSN7Spanish146772219Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening10547031b6e34cba-869f-4fe6-82a8-3f01749af68fDodatni wynik badania na obecność antygenu powierzchniowego wirusa zapalenia wątroby typu B, przeciwciał przeciwko wirusowi zapalenia wątroby typu C lub ludzkiego wirusa niedoboru odporności podczas badań przesiewowych19Polish10547033b6e34cba-869f-4fe6-82a8-3f01749af68fTest positif pour l’antigène de surface de l’hépatite B, l’anticorps de l’hépatite C ou le virus de l’immunodéficience humaine à la sélection10French10547030b6e34cba-869f-4fe6-82a8-3f01749af68fResultado positivo en el análisis del antígeno de superficie del virus de la hepatitis B, anticuerpos contra el virus de la hepatitis C o del virus de la inmunodeficiencia humana en la selección7Spanish10547032b6e34cba-869f-4fe6-82a8-3f01749af68fPositieve test voor hepatitis B-oppervlakteantigeen, hepatitis C-virus antilichaamserologie of hiv bij de screening37Dutch (Netherlands)14677232Have exposure to any dystrophin restoration product within 6 months prior to the start of study treatment1054703674716e32-0556-4e98-ad01-da0f6ad5d87bBlootstelling aan een dystrofine-herstellend product binnen 6 maanden voorafgaand aan de start van de onderzoeksbehandeling37Dutch (Netherlands)1054703574716e32-0556-4e98-ad01-da0f6ad5d87bExposición a cualquier fármaco de restauración de la distrofina en los 6 meses anteriores al inicio del tratamiento del estudio7Spanish1054703774716e32-0556-4e98-ad01-da0f6ad5d87bExposition à un produit de restauration de la dystrophine (p. ex., ataluren, saut d’exon) dans les 6 mois précédant le début du traitement à l’étude10French1054703474716e32-0556-4e98-ad01-da0f6ad5d87bNarażenie na jakikolwiek produkt przywracający dystrofinę w ciągu 6 miesięcy przed rozpoczęciem leczenia w ramach badania19Polish146772420Hypersensitivity to any component of study medication10547041a6faaa70-dc86-44d4-805a-826318161b82Overgevoeligheid voor een bestanddeel van het onderzoeksmiddel37Dutch (Netherlands)10547039a6faaa70-dc86-44d4-805a-826318161b82Hypersensibilité à l’un des composants du médicament à l’étude10French10547038a6faaa70-dc86-44d4-805a-826318161b82Hipersensibilidad conocida a cualquiera de los componentes del fármaco del estudio7Spanish10547040a6faaa70-dc86-44d4-805a-826318161b82Nadwrażliwość na którykolwiek składnik badanego leku19Polish146772521Sorbitol intolerance or malabsorption, or the hereditary form of fructose intolerance105470458b4e0531-b360-43fc-8532-e7c70d9c59f3Intolérance ou malabsorption du sorbitol, ou forme héréditaire d’intolérance au fructose10French105470438b4e0531-b360-43fc-8532-e7c70d9c59f3Intolerantie of malabsorptie van sorbitol, of de erfelijke vorm van fructose-intolerantie37Dutch (Netherlands)105470428b4e0531-b360-43fc-8532-e7c70d9c59f3Nietolerancja lub złe wchłanianie sorbitolu bądź dziedziczna postać nietolerancji fruktozy19Polish105470448b4e0531-b360-43fc-8532-e7c70d9c59f3Intolerancia al sorbitol o malabsorción, o la forma hereditaria de intolerancia a la fructosa7Spanish146772622Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator judgement10547049354f0e64-5291-46be-9175-8b948f918db3Diagnóstico de otras enfermedades neurológicas no controladas o presencia de trastornos somáticos no controlados de interés que no estén relacionados con la DMD, según el criterio del investigador7Spanish10547048354f0e64-5291-46be-9175-8b948f918db3Diagnose van andere ongecontroleerde neurologische ziekten of aanwezigheid van relevante ongecontroleerde somatische aandoeningen die geen verband houden met DMD, naar inzicht van de onderzoeker37Dutch (Netherlands)10547047354f0e64-5291-46be-9175-8b948f918db3Diagnostic d’autres maladies neurologiques non contrôlées ou présence de troubles somatiques pertinents non contrôlés qui ne sont pas liés à la DMD, selon l’avis de l’investigateur10French10547046354f0e64-5291-46be-9175-8b948f918db3Rozpoznanie innych niekontrolowanych chorób neurologicznych lub występowanie istotnych niekontrolowanych zaburzeń somatycznych, które nie są związane z DMD, na podstawie oceny badacza19Polish146772723Psychiatric illness or social situations rendering the potential patient unable to understand and comply with the muscle function tests and/or with the study protocol procedures, based on Investigator judgement1054705131d44274-65c9-4203-82f4-c7d76c0dea96Enfermedad psiquiátrica o situaciones sociales que impidan al posible paciente comprender y cumplir las pruebas de la función muscular o los procedimientos del protocolo del estudio, según el criterio del investigador7Spanish1054705031d44274-65c9-4203-82f4-c7d76c0dea96Psychiatrische ziekte of sociale omstandigheden waardoor de potentiële patiënt niet in staat is om de spierfunctietests en/of de procedures van het onderzoeksprotocol te begrijpen en na te leven, naar inzicht van de onderzoeker37Dutch (Netherlands)1054705331d44274-65c9-4203-82f4-c7d76c0dea96Choroba psychiczna lub sytuacje społeczne uniemożliwiające potencjalnemu pacjentowi zrozumienie i wykonywanie badań czynności mięśni i/lub procedur protokołu badania, na podstawie oceny badacza19Polish1054705231d44274-65c9-4203-82f4-c7d76c0dea96Maladie psychiatrique ou situation sociale rendant le/la patient(e) potentiel(le) incapable de comprendre et de se conformer aux tests de la fonction musculaire et/ou aux procédures du protocole de l’étude, selon l’avis de l’investigateur10French146772824Have contraindications to Magnetic Resonance Imaging (MRI) scan (eg, claustrophobia, metal implants, or uncontrolled seizure disorder), based on Investigator’s judgement1054705798c5498a-2cf2-4402-a5d3-4ea0b25d2844Contra-indicaties hebben voor magnetische resonantiebeeldvorming (MRI)-scan (bijv. claustrofobie, metalen implantaten of ongecontroleerde epileptische aandoening), naar inzicht van de onderzoeker.37Dutch (Netherlands)1054705598c5498a-2cf2-4402-a5d3-4ea0b25d2844Presencia de contraindicaciones a la resonancia magnética (RM) (p. ej., claustrofobia, implantes metálicos o trastorno convulsivo no controlado), según el criterio del investigador7Spanish1054705698c5498a-2cf2-4402-a5d3-4ea0b25d2844Avoir des contre-indications à l’imagerie par résonance magnétique (IRM) (p. ex., claustrophobie, implants métalliques ou troubles épileptiques non contrôlés), selon l’avis de l’investigateur10French1054705498c5498a-2cf2-4402-a5d3-4ea0b25d2844Występowanie przeciwwskazań do wykonywania badań obrazowych metodą rezonansu magnetycznego (MRI) (np. klaustrofobia, metalowe implanty lub niekontrolowane napady drgawkowe), na podstawie oceny badacza19Polish14677293Having received any gene therapy prior to start of study treatment105470616cf02a20-52a1-43df-a7a9-8fb1d5b81116Avoir reçu une thérapie génique (p. ex. virus adéno-associés, administration de microdystrophine) avant le début du traitement à l’étude10French105470586cf02a20-52a1-43df-a7a9-8fb1d5b81116Cualquier terapia génica antes del inicio del tratamiento del estudio7Spanish105470596cf02a20-52a1-43df-a7a9-8fb1d5b81116Een gentherapie hebben ontvangen voorafgaand aan de start van de onderzoeksbehandeling37Dutch (Netherlands)105470606cf02a20-52a1-43df-a7a9-8fb1d5b81116Otrzymanie jakiejkolwiek terapii genowej przed rozpoczęciem leczenia w ramach badania19Polish14677304Use of any pharmacologic treatment or supplement, (other than corticosteroids), other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to the start of study treatment (eg, growth hormone); vitamin D, calcium and any other supplements will be allowed.10547065a298d319-9276-49cf-baa2-b49af424884cStosowanie jakichkolwiek leków farmakologicznych lub suplementów (innych niż kortykosteroidy), które mogły oddziaływać na siłę lub funkcjonowanie mięśni w ciągu 3 miesięcy przed rozpoczęciem leczenia w ramach badania (np. hormon wzrostu); dopuszczalne jest stosowanie witaminy D, wapnia i innych suplementów.19Polish10547062a298d319-9276-49cf-baa2-b49af424884cCualquier tratamiento o suplemento con propiedades farmacológicas (excepto corticoesteroides) que pudiera haber afectado a la fuerza o la capacidad funcional muscular en los 3 meses anteriores al inicio del tratamiento del estudio (p. ej., hormona del crecimiento); se permitirán la vitamina D, el calcio y cualquier otro suplemento7Spanish10547064a298d319-9276-49cf-baa2-b49af424884cUtilisation de tout traitement ou complément pharmacologique (autre que des corticoïdes) qui pourrait avoir un effet sur la force ou la fonction musculaire dans les 3 mois précédant le début du traitement à l’étude (p. ex., hormone de croissance); la vitamine D, le calcium et tout autre complément seront autorisés10French10547063a298d319-9276-49cf-baa2-b49af424884cGebruik van een farmacologische behandeling of supplement, anders dan corticosteroïden, dat een effect kan hebben gehad op de spierkracht of -functie binnen 3 maanden voorafgaand aan de start van de onderzoeksbehandeling (bijv. groeihormoon); vitamine D, calcium en andere supplementen zijn toegestaan37Dutch (Netherlands)14677315Use of testosterone, unless used as a replacement therapy for the treatment of delayed puberty. The testosterone dose and regimen should be stable within 6 months prior to the start of study treatment.10547067082e31e3-7e15-4864-b467-8ccbb8d05695Stosowanie testosteronu, chyba że jest on stosowany jako terapia zastępcza w leczeniu opóźnionego dojrzewania. Dawka i schemat dawkowania testosteronu powinny być stałe w ciągu 6 miesięcy przed rozpoczęciem leczenia w ramach badania.19Polish10547066082e31e3-7e15-4864-b467-8ccbb8d05695Uso de testosterona, a menos que sea como tratamiento sustitutivo por retraso del desarrollo puberal. La dosis y la pauta de testosterona deben haber sido estables en los 6 meses anteriores al inicio del tratamiento del estudio7Spanish10547069082e31e3-7e15-4864-b467-8ccbb8d05695Utilisation de testostérone, sauf si elle est utilisée comme traitement substitutif pour prendre en charge un retard pubertaire La dose et le schéma thérapeutique de testostérone doivent être stables dans les 6 mois précédant le début du traitement à l’étude, et les taux de testostérone circulante doivent se situer dans les plages normales pour l’âge du/de la patient(e)10French10547068082e31e3-7e15-4864-b467-8ccbb8d05695Gebruik van testosteron, tenzij gebruikt als vervangingstherapie voor de behandeling van vertraagde puberteit. De dosis en het doseringsschema van testosteron moeten stabiel zijn gedurende 6 maanden voorafgaand aan de start van de onderzoeksbehandeling.37Dutch (Netherlands)14677326Elbow-flexion contractures >30° in the dominant arm105470718a6f50fd-6d38-4e15-8019-74e8640e9d7aContractura en flexión de codo >30° en el brazo dominante7Spanish105470708a6f50fd-6d38-4e15-8019-74e8640e9d7aContractures de flexion du coude > 30° dans le groupe dominant10French105470738a6f50fd-6d38-4e15-8019-74e8640e9d7aElleboogflexie contracturen > 30° in de dominante arm37Dutch (Netherlands)105470728a6f50fd-6d38-4e15-8019-74e8640e9d7aPrzykurcze zgięciowe łokcia >30° w ramieniu dominującym19Polish14677337Inability to perform consistent PUL 2.0 measurement within ±2 points without shoulder domain or within ±3 points with shoulder domain during paired testing at screening10547077764310ce-5fe5-494d-a844-020840a5c499Incapacidad para realizar una medición sistemática en la escala PUL 2.0 con un margen de ±2 puntos sin dominio del hombro o con un margen de ±3 puntos con dominio del hombro durante las pruebas pareadas, en la selección7Spanish10547074764310ce-5fe5-494d-a844-020840a5c499Niemożność wykonania spójnego pomiaru w skali PUL 2.0 w zakresie ±2 punktów bez uwzględniania obszaru barkowego lub w zakresie ±3 punktów z uwzględnieniem obszaru barkowego w trakcie sparowanych testów podczas badań przesiewowych19Polish10547076764310ce-5fe5-494d-a844-020840a5c499Niet in staat om consistente PUL 2.0-meting uit te voeren binnen ± 2 punten zonder schouderdomein of binnen ±3 punten met schouderdomein, tijdens gepaarde testen bij de screening37Dutch (Netherlands)10547075764310ce-5fe5-494d-a844-020840a5c499Incapacité à effectuer une mesure cohérente du score PUL 2.0 à ± 2 points sans le domaine de l’épaule ou à ± 3 points avec le domaine de l’épaule lors des tests appariés à la sélection10French14677348Forced Vital Capacity % of predicted <40%105470791a3bd170-613f-4711-86fa-84ce97043925Percentage voorspelde geforceerde vitale capaciteit (FVC) < 40%37Dutch (Netherlands)105470811a3bd170-613f-4711-86fa-84ce97043925Capacité vitale forcée (CVF) en pourcentage de la valeur prédite < 40 %10French105470801a3bd170-613f-4711-86fa-84ce97043925Przewidywana wartość procentowa natężonej pojemności życiowej <40%19Polish105470781a3bd170-613f-4711-86fa-84ce97043925Capacidad vital forzada (CVF) en porcentaje del valor previsto <40 %7Spanish14677359Requirement for daytime ventilator assistance105470827657eaa9-c24a-47eb-ad93-4d2a4d6f0f45Konieczność stosowania wspomagania wentylacji w ciągu dnia19Polish105470857657eaa9-c24a-47eb-ad93-4d2a4d6f0f45Necesidad de asistencia respiratoria durante el día7Spanish105470837657eaa9-c24a-47eb-ad93-4d2a4d6f0f45Beademingsondersteuning overdag vereist37Dutch (Netherlands)105470847657eaa9-c24a-47eb-ad93-4d2a4d6f0f45Nécessité d’une assistance ventilatoire de jour. Remarque : L’assistance respiratoire de nuit et l’utilisation d’un traitement par pression positive à deux niveaux sont autorisées10French10048751Change of PUL total score at 18 months of treatment of givinostat compared to placebo groupTrue1054697878f930d7-0d7e-4a19-9240-c4db0e12c73c• Variation du score total PUL à 18 mois de traitement par givinostat par rapport au groupe placebo.10French1054697778f930d7-0d7e-4a19-9240-c4db0e12c73cCambio en la puntuación total de la escala PUL a los 18 meses de tratamiento con givinostat en comparación con el grupo de placebo7Spanish1054697978f930d7-0d7e-4a19-9240-c4db0e12c73cZmiana całkowitego wyniku w skali PUL po 18 miesiącach leczenia giwinostatem w porównaniu z grupą otrzymującą placebo19Polish1054698078f930d7-0d7e-4a19-9240-c4db0e12c73cVerandering in de PUL-totaalscore na 18 maanden behandeling met givinostat vergeleken met de placebogroep.37Dutch (Netherlands)10048761•Change from baseline of Peak Expiratory Flow percent predicted (PEF%p) at 18 months of treatment of givinostat compared to placebo group • Change from baseline of Forced Vital Capacity percent predicted (FVC%p) at 18 months of treatment of givinostat compared to placebo group • Cumulative loss of PUL items over 18 months of treatment of givinostat compared to placebo groupFalse10546982b62cf1d7-03db-4801-9255-6a6eff7db49aCambio con respecto al inicio en el porcentaje previsto del flujo espiratorio máximo (FEMpp) a los 18 meses de tratamiento con givinostat en comparación con el grupo de placebo • Cambio con respecto al inicio en el porcentaje previsto de la capacidad vital forzada (CVFpp) a los 18 meses de tratamiento con givinostat en comparación con el grupo de placebo • Pérdida acumulada de ítems de la escala PUL durante 18 meses de tratamiento con givinostat en comparación con el grupo de placebo7Spanish10546983b62cf1d7-03db-4801-9255-6a6eff7db49aVerandering ten opzichte van de baseline van het percentage voorspelde piekstroom (PEF%p) na 18 maanden behandeling met givinostat vergeleken met de placebogroep •Verandering ten opzichte van de baseline van percentage voorspelde geforceerde vitale capaciteit (FVC%p) na 18 maanden behandeling met givinostat vergeleken met de placebogroep •Cumulatief verlies van PUL-items gedurende 18 maanden behandeling met givinostat vergeleken met de placebogroep37Dutch (Netherlands)10546984b62cf1d7-03db-4801-9255-6a6eff7db49a• Variation par rapport à la référence du débit expiratoire de pointe en pourcentage de la valeur prédite (% DEPp) à 18 mois de traitement par givinostat par rapport au groupe placebo • Variation par rapport à la référence de la capacité vitale forcée en pourcentage de la valeur prédite (% CVFp) à 18 mois de traitement par givinostat par rapport au groupe placebo • Perte cumulée des items PUL après 18 mois de traitement par givinostat par rapport au groupe placebo.10French10546981b62cf1d7-03db-4801-9255-6a6eff7db49aZmiana w stosunku do punktu wyjściowego przewidywanej procentowej wartości szczytowego przepływu wydechowego (PEF%p) po 18 miesiącach leczenia w porównaniu z grupą otrzymującą placebo • Zmiana w stosunku do punktu wyjściowego przewidywanej procentowej wartości natężonej pojemności życiowej (FVC%p) po 18 miesiącach leczenia w porównaniu z grupą otrzymującą placebo • Łączna liczba utraconych pozycji w skali PUL w okresie 18 miesięcy leczenia w porównaniu z grupą otrzymującą placebo19Polish10048772•Type incidence and severity of TEAEs •Proportion of patients experiencing TEAEs (baseline to EOS/F-Up) • Changes in vital signs, lab tests, ECG, ECHO •Time to assisted ventilation, rate and severity of respiratory infection and duration and use of antibiotics of givinostat to placebo group •Time to onset of diarrhoea •Height and weight Z-scoresFalse105469892dd85a63-c0a5-4d9d-ab5a-7af4dcd51891Rodzaj, częstość występowania i nasilenie TEAE • Odsetek pacjentów, u których wystąpiły TEAE (w okresie od punktu wyjściowego do zakończenia badania/obserwacji kontrolnej) • Zmiany w parametrach życiowych, wynikach badań laboratoryjnych, wynikach badań EKG, ECHO • Czas do rozpoczęcia stosowania wspomaganej wentylacji, częstość występowania i nasilenie infekcji dróg oddechowych oraz czas trwania i stosowanie antybiotyków w grupie otrzymującej giwinostat w porównaniu z grupą otrzymującą placebo •19Polish105469872dd85a63-c0a5-4d9d-ab5a-7af4dcd51891- Variation par rapport à la référence de la capacité vitale forcée en pourcentage de la valeur prédite (% CVFp) après 18 mois de traitement par givinostat par rapport au groupe placebo - Perte cumulée du score total PUL après 18 mois de traitement par givinostat par rapport au groupe placebo - Délai jusqu’à l’apparition de la diarrhée - Z-scores de la taille et du poids10French105469882dd85a63-c0a5-4d9d-ab5a-7af4dcd51891Polish, continue: Czas do wystąpienia biegunki • Wyniki dotyczące wzrostu i masy ciała40Other105469852dd85a63-c0a5-4d9d-ab5a-7af4dcd51891• Tipo, incidencia e intensidad de los AAST • Proporción de pacientes con AAST (desde el inicio hasta el FDE/seguimiento) • Cambio en las constantes vitales, los análisis clínicos, el electrocardiograma (ECG) y el ecocardiograma (ECO) • Tiempo hasta el uso de respiración asistida, tasa e intensidad de infecciones respiratorias, y duración y uso de antibióticos, con givinostat en comparación con el grupo de placebo • Tiempo hasta la aparición de diarrea • Puntuaciones Z de peso y estatura7Spanish105469862dd85a63-c0a5-4d9d-ab5a-7af4dcd51891Type, incidentie en ernst van tijdens de behandeling optredende bijwerkingen (TEAE’s) • Percentage patiënten met TEAE’s (baseline tot EOS/follow-up) • veranderingen in vitale functies, lab onderzoeken, ECG, ECHO • Tijd tot ondersteuning met beademing, frequentie en ernst van luchtweginfectie en duur en gebruik van antibiotica met givinostat vergeleken met de placebogroep • tijd tot begin van diarree • Lengte en gewicht Z scores37Dutch (Netherlands)2027-12-302027-12-302023-12-3092923Italfarmaco348870222PatientsFalseTrueTrue3NAP1828951Main Study• A screening period during which enrolment eligibility will be confirmed within 4 weeks • A baseline visit will occur at Day 1 on Week 1, • A double-blind treatment period, during which patients will be randomised 2:1 to receive either givinostat or placebo for 18 months (approximately 72 weeks) • An EOS visit will occur at the end of treatment period • Long term safety study or a follow-up visit will occur 4 weeks after the EOS visit21828971Subject1828962InvestigatorCentral randomisation using IWRS will be programmed with blind-breaking instructions if the opinion of the Investigator will be to know the teatment group in the patient’s best interest. Sites staff will be requested to sign a blinding plan. The sponsor is informed before blind is broken unless immediate unblindig is required due to emergency situation. The IDSMC will have access to unblinded safety data.2/3 of patients will be randomized to givinostat will be treated for 18 months twice daily (bid) in a fed state.Test1021041/3 of patients will be randomized to placebo and will be treated for 18 months twice daily (bid) in a fed state.placebo102105111291EMEA-000551-PIP04-21523765PMID 27566866PMID 27566866https://pubmed.ncbi.nlm.nih.gov/27566866acceptable5Diseases [C] - Musculoskeletal Diseases [C05]132405Duchenne muscular dystrophy (DMD)True144091True424644PublicSerena Franciosis.franciosi@italfarmacogroup.com+39347633755979684Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active424645ScientificPaolo Tornesep.tornese@italfarmacogroup.com+39026443252179684Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active649383169813173213Pharmaceutical company10Chillibean LimitedTrueTrueORG-100042592Active17986153 Frith Street53 Frith StreetLondonW1D 4SN826United Kingdom+442074797030matt@chillibean.tvTrueLOC-10007039310079467+442074797030matt@chillibean.tv649384169789173189Pharmaceutical company10Fortrea BelgiumTrueTrueORG-100040389Active179835Avenue Du Bourget 50Avenue Du Bourget 50Brussels11302002Belgium00420267317706submissons@fortea.comTrueLOC-10006614710079471100794811100794912100795013100795114100795215Data Analysis10079532100795441007955500420267317706submissons@fortea.com649382169812173212Non-Pharmaceutical company11Atom International LimitedTrueTrueORG-100042393Active179860Unit 4, Earls House, Earlsway, Team Valley Trading EstateUnit 4Earls HouseEarlswayTeam Valley Trading EstateGatesheadNE11 0RY826United Kingdom+447540051001michelle.eagle@atom-international.orgTrueLOC-10007003810079456+447540051001michelle.eagle@atom-international.org649387173720177147Hospital/Clinic/Other health care facility8Syneos Health Clinique Inc.FalseTrueORG-100028348Active1841422500 Einstein St2500 Einstein StQuebecG1P 0A2124Canada+14181005503alexandre.tremblay@syneoshealth.comTrueLOC-10004542410079594+14181005503alexandre.tremblay@syneoshealth.com649386173721177148Educational Institution1University Of FloridaTrueTrueORG-100031776Active184143P. O. Box 114000P. O. Box 114000Gainesville32611-4000840United States+13525945995azmitchell@phhp.ufl.eduTrueLOC-100080301100795815Data Analysis+13525945995azmitchell@phhp.ufl.edu649385169797173197Pharmaceutical company10Alira HealthTrueTrueORG-100030303Active17984512 Rue Charlot12 Rue CharlotParis750032012France+447917973305hannah.baxter@alirahealth.comTrueLOC-10005037910079561010079576+447917973305hannah.baxter@alirahealth.com79684Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active7716079684Pharmaceutical company10Italfarmaco S.p.A.TrueTrueORG-100000118Active80910Via Dei Lavoratori 54Via Dei Lavoratori 54Cinisello Balsamo200922018ItalyTrueLOC-100004430TrueCommercial2This is phase III to evaluate safety efficacy and tolerability11390005Diseases [C] - Musculoskeletal Diseases [C05]393405PLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspension3Placebo5275031N/AN/AN/APLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspensionN/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse32N/AFalsePLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspensionn/aN/A393404ITF23571Test527502111133213ORAL SUSPENSIONPRD47976781ITF2357ORAL SUSPENSIONITF2357GIVINOSTATSUB189959GIVINOSTATITALFARMACO SPA11133213337329ITALFARMACO SPAChemicalChemicalGIVINOSTATSUB189959False1TrueOrfan EU/3/12/1009mg milligram(s)93.4mg milligram(s)47073183histone deacetylase (HDAC) inhibitorPRD4797678ITF235711ORAL USETrueITF2357givinostatORAL SUSPENSIONFalse1727015917591737442002Authorised2023-11-082023-11-08FalseunwillingBelgium2023-12-11Authorised208952024-05-172024-05-29208962024-05-172024-05-29558652024-10-252024-11-15558662024-10-252024-11-15True272162024-05-172024-05-292024-10-25False106605917Under evaluation2023-08-16T19:48:05.368184195917Authorised2023-12-11T15:24:36.7041Belgiumacceptable2025-02-14authorized_conditions2023-12-112023-12-1171115384189656193154Hospital/Clinic/Other health care facility8UZ LeuvenFalseTrueORG-100006001Active201257Herestraat 49Herestraat 49Leuven30002002Belgium3216340610liesbeth.dewaele@uzleuven.beTrueLOC-1000053901246572LiesbethDe Waele3216340610liesbeth.dewaele@uzleuven.be2Paediatric NeurologyAuthorised280639558095580937442027Authorised2026-02-132026-02-13FalseSpain2026-03-25AuthorisedFalseFalse19862655809Under evaluation2025-12-19T11:17:00.91433931255809Authorised2026-03-25T10:12:17.9213Spainacceptable2026-03-17authorized2026-03-252026-03-25121928821884070926096Hospital/Clinic/Other health care facility8Hospital Universitario Y Politecnico La FeFalseTrueORG-100029610Active987122Avenida Fernando Abril Martorell 106Avenida Fernando Abril Martorell 106Valencia460262027Spain0034961244153muelas_nur@gva.esTrueORG-1000296102142799NuriaMuelas Gómez0034961244153muelas_nur@gva.es1Neurology1928822884071926097Hospital/Clinic/Other health care facility8Hospital Sant Joan De Deu BarcelonaFalseTrueORG-100023083Active987123Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain0034936009733carlos.ortez@sjd.esTrueORG-1000230832142800Carlos IgnacioOrtez Gonzalez0034936009733carlos.ortez@sjd.es1NeurologyAuthorised284902558105581037442028Authorised2026-03-022026-03-02FalseSweden2026-03-10AuthorisedFalseFalse19862955810Under evaluation2025-12-19T11:18:26.29432246955810Authorised2026-03-10T09:52:26.853Swedenacceptable2026-03-10authorized2026-03-102026-03-1041961572884069926095Hospital/Clinic/Other health care facility8Karolinska University HospitalFalseTrueORG-100000573Active987121Eugeniavagen 3Eugeniavagen 3Solna171 642028Sweden0046812370000thomas.sejersen@ki.seTrueORG-1000005732178828ThomasSejersen0046812370000thomas.sejersen@ki.se1CKB Centrum för Kliniska Barnstudier, Karolinska vägen 37 A, 171 76 Stockholm, SwedeAuthorised286110558115581137442031Authorised2026-03-052026-03-05FalsePoland2026-03-27AuthorisedFalseFalse19863755811Under evaluation2025-12-19T11:21:09.05833958855811Authorised2026-03-27T12:19:22.3763Polandacceptable2026-03-23authorized2026-03-272026-03-27101969574883874925900Hospital/Clinic/Other health care facility8Uniwersyteckie Centrum KliniczneFalseTrueORG-100042500Active986905Ul. Debinki 7Ul. Debinki 7Gdansk80-9522031Poland0048583492390mmazur@gumed.edu.plTrueORG-1000425002187741MariaMazurkiewicz-Bełdzińska0048583492390mmazur@gumed.edu.pl2Klinika Neurologii Rozwojowej1969575883875925901Hospital/Clinic/Other health care facility8Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu MedycznegoFalseTrueORG-100043164Active986906Ul. Ulica Stefana Banacha 1aUl. 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MODIFICATIONFalse25196NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2024-04-302024-04-305920GermanyAuthorised5924NetherlandsAuthorised5921ItalyAuthorised5917BelgiumAuthorised5919FranceAuthorisedNSM-2PART_IFalse20295251965920Germany2024-04-30T16:37:53.428authorizeddecision37541NON SUBSTANTIAL MODIFICATIONFalse20295251965924Netherlands2024-04-30T16:37:53.428authorizeddecision37541NON SUBSTANTIAL MODIFICATIONFalse20295251965921Italy2024-04-30T16:37:53.428authorizeddecision37541NON SUBSTANTIAL MODIFICATIONTrue20295251965917Belgium2024-04-30T16:37:53.428authorizeddecision37541NON SUBSTANTIAL MODIFICATIONFalse20295251965919France2024-04-30T16:37:53.428authorizeddecision37541NON SUBSTANTIAL MODIFICATIONFalse31276SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2024-07-17acceptable12301359205920Germany2013acceptable2024-08-23authorized2023-12-05AuthorisedGermanyAuthorised2023-12-05Authorised2024-08-265920GermanyAuthorisedSM-3PART_IIFalse34801312765920Germany2024-08-26T13:40:23.221authorizedacceptabledecision12301347410SUBSTANTIAL MODIFICATIONFalse37556NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2024-10-07acceptable14437959215921Italy2018authorized2023-12-11AuthorisedItalyAuthorised2023-12-11Authorised2024-10-075921ItalyAuthorisedNSM-3PART_I_AND_PART_IIFalse40723375565921Italy2024-10-07T16:20:17.782authorizeddecision14437957608NON SUBSTANTIAL MODIFICATIONTrue38737NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2024-10-23acceptable14891859215921Italy2018authorized2023-12-11AuthorisedItalyAuthorised2023-12-11Authorised2024-10-235921ItalyAuthorisedNSM-4PART_I_AND_PART_IIFalse43257387375921Italy2024-10-23T15:38:32.427authorizeddecision14891859622NON SUBSTANTIAL MODIFICATIONTrue39639NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2024-11-01acceptable15237559215921Italy2018authorized2023-12-11AuthorisedItalyAuthorised2023-12-11Authorised2024-11-015921ItalyAuthorisedNSM-5PART_I_AND_PART_IIFalse44644396395921Italy2024-11-01T12:19:48.922authorizeddecision15237561230NON SUBSTANTIAL MODIFICATIONTrue42246NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2024-11-28acceptable16114759215921Italy2018authorized2023-12-11AuthorisedItalyAuthorised2023-12-11Authorised2024-11-285921ItalyAuthorisedNSM-6PART_I_AND_PART_IIFalse48811422465921Italy2024-11-28T15:10:24.606authorizeddecision16114765283NON SUBSTANTIAL MODIFICATIONTrue45411NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2025-01-14acceptable17133259215921Italy2018authorized2023-12-11AuthorisedItalyAuthorised2023-12-11Authorised2025-01-145921ItalyAuthorisedNSM-7PART_I_AND_PART_IIFalse53122454115921Italy2025-01-14T15:31:02.056authorizeddecision17133270077NON SUBSTANTIAL MODIFICATIONTrue45671SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2025-01-17acceptable17270159175917Belgium2002acceptable2025-02-14authorized_conditions2023-12-11AuthorisedBelgiumAuthorised2023-12-11Authorised2025-02-145917BelgiumAuthorisedSM-4PART_IIFalse58044456715917Belgium2025-02-14T14:19:06.257authorizedacceptabledecision17270170519SUBSTANTIAL MODIFICATIONFalse47723NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2025-02-2017956159215921Italy2018authorized2023-12-11AuthorisedItalyAuthorised2023-12-11Authorised2025-02-205921ItalyAuthorisedNSM-8PART_I_AND_PART_IIFalse58881477235921Italy2025-02-20T14:27:20.051authorizeddecision17956173622NON SUBSTANTIAL MODIFICATIONTrue49579SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2025-03-20acceptable2025-06-302025-06-305924NetherlandsAuthorised5919FranceAuthorised5920GermanyAuthorised5917BelgiumAuthorised5921ItalyAuthorisedSM-5PART_IFalse78226495795919France2025-06-30T18:54:44.441authorizeddecision80281SUBSTANTIAL MODIFICATIONFalse78572495795920Germany2025-07-03T09:41:03.864authorizeddecision80281SUBSTANTIAL MODIFICATIONFalse78667495795924Netherlands2025-07-03T15:12:38.384authorizeddecision80281SUBSTANTIAL MODIFICATIONFalse78837495795921Italy2025-07-04T12:41:58.827authorizeddecision80281SUBSTANTIAL MODIFICATIONTrue78905495795917Belgium2025-07-04T15:00:40.865authorizeddecision80281SUBSTANTIAL MODIFICATIONFalse60135NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2025-07-15acceptable2025-06-302025-07-155924NetherlandsAuthorised5919FranceAuthorised5920GermanyAuthorised5917BelgiumAuthorised5921ItalyAuthorisedNSM-9PART_IFalse80480601355924Netherlands2025-07-15T19:07:26.647authorizeddecision92728NON SUBSTANTIAL MODIFICATIONFalse80480601355919France2025-07-15T19:07:26.647authorizeddecision92728NON SUBSTANTIAL MODIFICATIONFalse80480601355920Germany2025-07-15T19:07:26.647authorizeddecision92728NON SUBSTANTIAL MODIFICATIONFalse80480601355917Belgium2025-07-15T19:07:26.647authorizeddecision92728NON SUBSTANTIAL MODIFICATIONFalse80480601355921Italy2025-07-15T19:07:26.647authorizeddecision92728NON SUBSTANTIAL MODIFICATIONTrue60851NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2025-07-21acceptable2025-06-3022688759245924Netherlands2029authorized2023-12-05AuthorisedNetherlandsAuthorised2023-12-05Authorised2025-07-215924NetherlandsAuthorisedNSM-10PART_IIFalse81384608515924Netherlands2025-07-21T19:22:47.008authorizeddecision22688793838NON SUBSTANTIAL MODIFICATIONFalse61421NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2025-07-29acceptable2025-06-3022869959195919France2012authorized2023-12-06AuthorisedFranceAuthorised2023-12-06Authorised2025-07-295919FranceAuthorisedNSM-11PART_IIFalse82488614215919France2025-07-29T13:19:08.238authorizeddecision22869994714NON SUBSTANTIAL MODIFICATIONFalse64514NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2025-09-10acceptable2025-06-3023923659215921Italy2018authorized2023-12-11AuthorisedItalyAuthorised2023-12-11Authorised2025-09-105921ItalyAuthorisedNSM-12PART_IIFalse88773645145921Italy2025-09-10T17:10:50.53authorizeddecision23923699572NON 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MODIFICATIONAuthorised2023-503521-19-00Authorised2025-12-12acceptable26813659205920Germany2013acceptable2026-01-12authorized2023-12-05AuthorisedGermanyAuthorised2023-12-05Authorised2026-01-145920GermanyAuthorisedSM-6PART_IIFalse104829726295920Germany2026-01-14T09:56:28.813authorizedacceptabledecision268136111516SUBSTANTIAL MODIFICATIONFalse75985SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2026-02-03acceptable27719859195919France2012acceptable2026-03-16authorized2023-12-06AuthorisedFranceAuthorised2023-12-06Authorised2026-03-175919FranceAuthorisedSM-7PART_IIFalse114799759855919France2026-03-17T15:02:53.68authorizedacceptabledecision277198116608SUBSTANTIAL MODIFICATIONFalse81167SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2026-04-01acceptable2958335580855808Czechia2008acceptable2026-04-17authorized2026-04-20AuthorisedCzechiaAuthorised2026-04-20Authorised2026-04-2055808CzechiaAuthorisedSM-8PART_IIFalse1199468116755808Czechia2026-04-20T11:20:06.401authorizedacceptabledecision295833124318SUBSTANTIAL MODIFICATIONFalse82984NON SUBSTANTIAL MODIFICATIONAuthorised2023-503521-19-00Authorised2026-05-04acceptable30005059205920Germany2013authorized2023-12-05AuthorisedGermanyAuthorised2023-12-05Authorised30004959245924Netherlands2029authorized2023-12-05AuthorisedNetherlandsAuthorised2023-12-05Authorised30004859215921Italy2018authorized2023-12-11AuthorisedItalyAuthorised2023-12-11Authorised2026-05-045920GermanyAuthorised5924NetherlandsAuthorised5921ItalyAuthorisedNSM-13PART_I_AND_PART_IIFalse122220829845920Germany2026-05-04T13:18:10.573authorizeddecision300050127019NON SUBSTANTIAL MODIFICATIONFalse122222829845921Italy2026-05-04T13:18:10.573authorizeddecision300048127019NON SUBSTANTIAL MODIFICATIONTrue122221829845924Netherlands2026-05-04T13:18:10.573authorizeddecision300049127019NON SUBSTANTIAL MODIFICATIONFalseNetherlands59242023-12-05T09:10:24.7992026-05-04T13:18:10.5734Germany59202023-12-05T11:30:37.2432026-05-04T13:18:10.5734France59192023-12-06T16:59:58.6322026-03-17T15:02:53.684Italy59212023-12-11T09:29:50.8032026-05-04T13:18:10.5734Belgium59172023-12-11T15:24:37.8822025-02-14T14:19:06.2574Czechia558082026-03-19T15:06:34.5892026-04-20T11:20:06.4012Spain558092026-03-25T10:12:18.2042026-03-25T10:12:18.2042Sweden558102026-03-10T09:52:27.3412026-03-10T09:52:27.3412Poland558112026-03-27T12:19:22.8982026-03-27T12:19:22.8982False5917BelgiumSTART_OF_TRIAL2024-05-17START_OF_RECRUITMENT2024-10-255919FranceSTART_OF_TRIAL2024-05-16START_OF_RECRUITMENT2024-05-275920GermanySTART_OF_TRIAL2025-04-07START_OF_RECRUITMENT2025-04-285921ItalySTART_OF_TRIAL2024-07-15START_OF_RECRUITMENT2024-08-295924NetherlandsSTART_OF_TRIAL2024-02-09START_OF_RECRUITMENT2024-02-1955808Czechia55809Spain55810Sweden55811PolandSB-165322024-03-012024-02-162024-05-282024-05-28100000000337Belgium2002BEBELTrue100000000403Germany2013DEDEUTrue100000000430Italy2018ITITATrue100000000556United Kingdom826GBGBRTrue100000000478Netherlands2029NLNLDTrue100000000354Canada124CACANTrue100000000395France2012FRFRATrueOtherDescription of serious breach and impacts on trial is provided in the attachment separately.Description of actions taken and planned is provided in the attachment separately.TrueAlthough no subjects were screened at time of the incident, Italfarmaco assessed the potential unblinding issue may have impact on the data integrity and the double-blind fashion of the study could not be ensure without further remediation.BelgiumFranceGermanyItalyNetherlands7716079684PHARMACEUTICAL_COMPANY10Italfarmaco S.p.A.ORG-10000011880910Via Dei Lavoratori 54Via Dei Lavoratori 54Cinisello Balsamo200922018ItalyLOC-100004430Sponsor (commercial)169797173197PHARMACEUTICAL_COMPANY10Alira HealthORG-10003030317984512 Rue Charlot12 Rue CharlotParis750032012FranceLOC-100050379OtherStatistical analysi, data management2ProtocolThis is last update to the Serious Breach. 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(for publication)PDF1216935.03Italy115062023-12-01T09:04:58.0662025-12-17T12:03:04.6495Spain115072023-11-30T08:20:33.7582026-02-09T17:28:05.0115Netherlands115082024-04-03T14:56:54.9912026-01-15T16:02:15.073511506ItalySTART_OF_TRIAL2024-04-30START_OF_RECRUITMENT2024-09-18END_OF_RECRUITMENT2026-05-1211507SpainSTART_OF_TRIAL2024-03-08START_OF_RECRUITMENT2024-03-18END_OF_RECRUITMENT2026-05-1211508NetherlandsSTART_OF_TRIAL2024-08-29START_OF_RECRUITMENT2024-11-13END_OF_RECRUITMENT2026-05-122023-506737-30-005A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants with Duchenne Muscular Dystrophy351-20108/03/2024Duchenne Muscular DystrophyItaly:5Spain:5Netherlands:530/11/2023ES: 30/11/2023, NL: 03/04/2024, IT: 01/12/2023Diseases [C] - Musculoskeletal Diseases [C05]Biomarin Pharmaceutical Inc.Pharmaceutical companyPhase I and Phase II (Integrated)- First administration to humans1. BMN 351 plasma PK, urine PK and concentration in muscleExon 51 specific phosphorothioate oligonucleotide450-17 yearsMale361. Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiography, 2. Physical examination, 3. Safety laboratory test parameters, 4. ECG parametersNo13/03/202615/05/20262023-506737-30-00Authorised2024-03-082023-11-30T08:20:33.7582026-05-15T03:31:15.566579781512169312100000000549Turkey792TRTURTrue100000000556United Kingdom826GBGBRTrue504718110564554CONCENTRATE FOR SOLUTION FOR INFUSIONPRD105645541Exon 51 specific phosphorothioate oligonucleotideCONCENTRATE FOR SOLUTION FOR INFUSIONBMN 351BMN 351SUB328513BIOMARIN PHARMACEUTICAL INC.10564554328517BIOMARIN PHARMACEUTICAL INC.Nucleic AcidNucleic AcidBMN 351SUB328513NOTASSIGN-False1FalseChemicalPRD10564554BMN 35111INTRAVENIOUS INFUSIONFalseExon 51 specific phosphorothioate oligonucleotidebmn 351CONCENTRATE FOR SOLUTION FOR INFUSIONA Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants with Duchenne Muscular Dystrophy100464258c76ece6-c4a6-43bb-8065-7c77379e9a52Estudio de fase I/II, abierto, de aumento escalonado de la dosis para evaluar la seguridad, la tolerabilidad, la farmacocinética y la farmacodinamia de dosis intravenosas múltiples de BMN 351 en participantes con distrofia muscular de Duchenne7Spanish100464248c76ece6-c4a6-43bb-8065-7c77379e9a52Een fase 1/2, open-label, dosisescalatiestudie om de veiligheid, verdraagbaarheid, farmacokinetiek en farmacodynamiek van meerdere intraveneuze doses BMN 351 te beoordelen bij deelnemers met Duchenne spierdystrofie37Dutch (Netherlands)A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants with Duchenne Muscular Dystrophy100464418c6bbd37-18ff-4a56-ab2f-8f66aea35276Een fase-1/2-onderzoek met dosisescalatie ter beoordeling van de veiligheid, verdraagbaarheid, farmacokinetiek en farmacodynamiek van BMN 351 bij deelnemers met Duchenne spierdystrofie37Dutch (Netherlands)100464408c6bbd37-18ff-4a56-ab2f-8f66aea35276Estudio de fase I/II de aumento escalonado de la dosis para evaluar la seguridad, tolerabilidad, farmacocinética y farmacodinamia de BMN 351 en participantes con distrofia muscular de Duchenne7Spanish351-201504059U1111-1293-4876504058NCT06280209False72Study 351-201 is a first in human integrated phase 1/2 Study. In line with EMA guidance on disclosure rules, the study meets the criteria of a Category 2 trial.111597126641Duchenne Muscular Dystrophy100463990da70665-7a13-4530-88a1-0101a306871eDistrofia muscular de Duchenne7Spanish100463980da70665-7a13-4530-88a1-0101a306871eDuchenne spierdystrofie37Dutch (Netherlands)True10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False736848063684794368478To assess the safety and tolerability of BMN 351 at different dose levels in participants with DMD10046438b7bf7000-b581-4015-8840-edaf11fa975fBeoordeling van de veiligheid en verdraagbaarheid van BMN 351 op verschillende dosisniveaus bij deelnemers met DMD37Dutch (Netherlands)10046439b7bf7000-b581-4015-8840-edaf11fa975fEvaluar la seguridad y la tolerabilidad de BMN 351 a diferentes niveles de dosis en participantes con DMD7Spanish4287331To evaluate the plasma and urine pharmacokinetics and muscle distribution of BMN 35110046443ce943f38-e534-4bfa-a744-3adaa2ddded5Beoordeling van de farmacokinetiek van BMN 351 in plasma en urine en de distributie in de spieren37Dutch (Netherlands)10046442ce943f38-e534-4bfa-a744-3adaa2ddded5Evaluar la farmacocinética en plasma y orina y la distribución muscular de BMN 3517Spanish81933011. Is male and age 4 through 10 years at Screening.1004642656c49031-3499-40d5-8436-57882d2e3ef51. Pacience maculino, de 4 a 10 años de edad durante la selección.7Spanish1004642756c49031-3499-40d5-8436-57882d2e3ef51. Is in de leeftijd van 4 tot 10 jaar bij screening37Dutch (Netherlands)81933122. Clinical diagnosis of Duchenne muscular dystrophy in the opinion of the investigator resulting from a documented dystrophin mutation in the DMD gene amenable to exon 51 skipping as reviewed by a central genetic counselor.1004642871d36f5d-ad85-4ab3-8c8c-5b9b3e5c56db2. Klinische diagnose van duchenne-spierdystrofie naar opvatting van de onderzoeker afkomstig van een vastgestelde dystrofinemutatie in het DMD-gen dat vatbaar is voor exon skipping van het exon 51, zoals beoordeeld door een centraal genetisch adviseur37Dutch (Netherlands)1004642971d36f5d-ad85-4ab3-8c8c-5b9b3e5c56db2. Diagnóstico clínico de distrofia muscular de Duchenne según el criterio del investigador, resultante de una mutación documentada de la distrofina en el gen DMD compatible con omisión del exón 51 según la revisión de un asesor genético centralizado.7Spanish81933233. Ambulatory at Screening, defined as able to walk independently without assistive devices and complete the timed 10 meter walk/run test in 8 seconds or less.1004643139879dcc-f467-4002-9c5f-009b9a3016023. Capacidad de deambulación en la selección, definida como capacidad de caminar de forma independiente sin dispositivos de ayuda y de completar la prueba cronometrada de caminar/correr 10 metros en 8 segundos o menos.7Spanish1004643039879dcc-f467-4002-9c5f-009b9a3016023. Ambulant bij screening, gedefinieerd als onafhankelijk kunnen lopen zonder hulpmiddelen en in staat zijn om de getimede loop/ren test van 10 meter in 8 seconden of minder af te leggen37Dutch (Netherlands)81933344. Not currently daytime ventilator dependent and not expected to need daytime mechanical or noninvasive ventilation within the next year in the opinion of the investigator.10046433b86daa31-8639-4aa8-8427-11b609020aa14. Momenteel overdag niet afhankelijk van een beademingsapparaat en naar verwachting geen mechanische of niet-invasieve beademing overdag nodig binnen het volgende jaar, naar opvatting van de onderzoeker37Dutch (Netherlands)10046432b86daa31-8639-4aa8-8427-11b609020aa14. No dependiente actualmente de ventilación durante el día y no se espera que necesite ventilación mecánica o no invasiva durante el día en el año siguiente, según el criterio del investigador.7Spanish81933455. Currently receiving treatment with oral corticosteroids or vamorolone, on a stable dose for at least 12 weeks prior to Baseline, and must remain on a consistent dose/dose regimen throughout the study except for modifications to accommodate changes in weight.1004643437d39d68-c8fd-477a-9065-c88f18670a2c5. Huidige behandeling met orale corticosteroïden of vamorolon, met een stabiele dosis gedurende minimaal 12 weken voorafgaand aan baseline, en moet gedurende het hele onderzoek op een constante dosis/constant dosisregime worden gehouden, afgezien van aanpassingen ten behoeve van veranderingen in gewicht37Dutch (Netherlands)1004643537d39d68-c8fd-477a-9065-c88f18670a2c5. Tratamiento actual con corticosteroides orales o vamotolona en una dosis estable durante al menos las 12 semanas previas a la visita basal y mantenimiento de una dosis o pauta posológica uniforme durante todo el estudio, excepto modificaciones para adaptarse a las variaciones del peso.7Spanish81933566. Normal urinalysis at Screening (trace protein allowed).1004643798ab52eb-6c20-4718-af05-41c82e9b17226. Uitslag urine-onderzoek normaal bij screening (sporen van eiwitten toegestaan)37Dutch (Netherlands)1004643698ab52eb-6c20-4718-af05-41c82e9b17226. Análisis de orina normal en la selección (se permiten trazas de proteínas).7Spanish140213511. For children 7 years of age or older, forced expiratory volume (FEV1) < 60% of predicted.10046410d2991afa-247a-43dc-a399-caf58a38bdb51. Voor kinderen van 7 jaar of ouder, eensecondewaarde (FEV1) < 60% van voorspeld volume37Dutch (Netherlands)10046411d2991afa-247a-43dc-a399-caf58a38bdb51. En niños de 7 años o más, volumen espiratorio forzado (FEV1) <60 % del valor previsto.7Spanish140213622. Current or history of liver or renal disease.10046413770f1573-bec1-4350-9b29-ba728f0604042. Huidig voorkomende lever- of nierziekten of geschiedenis daarvan37Dutch (Netherlands)10046412770f1573-bec1-4350-9b29-ba728f0604042. Presencia o antecedentes de hepatopatía o nefropatía.7Spanish140213733. Left ventricular ejection fraction (LVEF) < 55% based on an ECHO performed within 3 months prior to the Baseline (Day 1) visit.10046414146b242e-0ad3-40db-9301-4713081a557b3. Linkerventrikel-ejectiefractie (LVEF) < 55% op basis van een ECHO, uitgevoerd binnen 3 maanden voorafgaand van het baselinebezoek (dag 1)37Dutch (Netherlands)10046415146b242e-0ad3-40db-9301-4713081a557b3. Fracción de eyección del ventrículo izquierdo (FEVI) <55 % según un ECG realizado en los 3 meses previos a la visita basal (día 1).7Spanish140213844. Mean QT interval corrected with Fridericia’s method (QTcF) ≥ 450 msec on the Screening electrocardiogram (ECG) conducted in triplicate.100464160d2e5fc5-7eb6-45bd-8ab7-fceaae773f894. Gemiddeld QT-interval, gecorrigeerd met de methode van Fridericia (QTcF) ≥ 450 msec op het elektrocardiogram (ECG) bij screening, in drievoud uitgevoerd37Dutch (Netherlands)100464170d2e5fc5-7eb6-45bd-8ab7-fceaae773f894. Intervalo QT medio corregido por el método de Fridericia (QTcF) ≥450 ms en el electrocardiograma (ECG) de selección realizado por triplicado.7Spanish140213955. Platelet count of < 150 x 10^9/L at Screening.10046418e239e32d-8595-45b2-97a4-3282e0ed61b65. Recuento de plaquetas <150 x 10^9/l en la selección.7Spanish10046419e239e32d-8595-45b2-97a4-3282e0ed61b65. Bloedplaatjestelling van < 150 x 109/L bij screening37Dutch (Netherlands)140214066. Renal function laboratory parameters outside of prespecified values as defined per protocol.1004642152d2204b-9324-41ac-b261-5cfa86764bec6. Laboratoriumparameters voor nierfunctie buiten de voorgeschreven waarden, zoals bepaald in het protocol37Dutch (Netherlands)1004642052d2204b-9324-41ac-b261-5cfa86764bec6. Resultados analíticos renales fuera de los valores preespecificados en el protocolo.7Spanish140214177. Treatment with any exon skipping therapy within 12 weeks prior to Baseline (Day 1) or with any gene therapy for the treatment of DMD at any time.10046423d485ad56-2b0e-4499-8f4e-e8fed6e83ff77. Tratamiento con una terapia aprobada de omisión de exones en las 12 semanas previas a la visita basal (día 1) o cualquier terapia génica para el tratamiento de la DMD en cualquier momento.7Spanish10046422d485ad56-2b0e-4499-8f4e-e8fed6e83ff77. Behandeling met een goedgekeurde exon skipping therapie binnen 12 weken voorafgaand aan de baseline (dag 1) of met enige vorm van gentherapie voor de behandeling van DMD op elk moment37Dutch (Netherlands)96015611. Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiographyTrue1004640137e77793-3ad4-43f4-925a-db7629621b4c1. Incidencia de acontecimientos adversos/AAG/AAIE.7Spanish1004640037e77793-3ad4-43f4-925a-db7629621b4c1. Incidentie van ongewenste voorvallen/SAEs/AESI37Dutch (Netherlands)96015722. Physical examinationTrue100464022e4f0a5e-e53a-4160-9f13-8a8438d0f5d32. Lichamelijk onderzoek37Dutch (Netherlands)100464032e4f0a5e-e53a-4160-9f13-8a8438d0f5d32. Exploración física.7Spanish96015833. Safety laboratory test parametersTrue1004640476cf3deb-80df-46ea-a39e-53a218ebb18d3. Parameters veiligheidsonderzoeken laboratorium37Dutch (Netherlands)1004640576cf3deb-80df-46ea-a39e-53a218ebb18d3. Parámetros analíticos de seguridad.7Spanish96015944. ECG parametersTrue100464071d766f12-9d4f-4619-b15c-7c6d6060d1fc4. Parámetros del ECG.7Spanish100464061d766f12-9d4f-4619-b15c-7c6d6060d1fc4. ECG-parameters37Dutch (Netherlands)96016011. BMN 351 plasma PK, urine PK and concentration in muscleFalse100464080b8e5caa-72ff-473f-95ee-e4fbcfb3cef71. Parámetros farmacocinéticos de MBN 351 en plasma y orina, y concentración en músculo.7Spanish100464090b8e5caa-72ff-473f-95ee-e4fbcfb3cef71. Plasma en urine PK parameters en BMN 351-concentratie in spieren.37Dutch (Netherlands)2026-08-302026-08-302023-11-3088840BioMarin Pharmaceutical Inc.33426222334263242433426425252PatientsFalseTrueTrue351588161914165258Non-EEA National Competent authority17Food And Drug AdministrationFalseTrueORG-100006066Active1711715901 Ammendale Road Ste B5901 Ammendale Road Ste BBeltsville20705-1277840United StatesTrueLOC-10007532051587161912165256EEA National Competent authority16Federal Institute For Drugs And Medical DevicesFalseTrueORG-100003923Active171169Kurt-Georg-Kiesinger-Allee 3, PlittersdorfKurt-Georg-Kiesinger-Allee 3PlittersdorfBonn531752013GermanyTrueLOC-10000002451589161917165261EEA National Competent authority16Medicines Evaluation BoardFalseTrueORG-100003934Active171174Graadt Van Roggenweg 500Graadt Van Roggenweg 500Utrecht3531 AH2029NetherlandsTrueLOC-100000046acceptable2025-12-152025-12-155Diseases [C] - Musculoskeletal Diseases [C05]126641Duchenne Muscular DystrophyTrue137823True406318PublicClinical Trial Information Deskmedinfo@bmrn.com+14155066700164812Pharmaceutical company10Biomarin Pharmaceutical Inc.TrueTrueORG-100006134Active406319ScientificClinical Trial Information Deskmedinfo@bmrn.com+14155066700164812Pharmaceutical company10Biomarin Pharmaceutical Inc.TrueTrueORG-100006134Active619433180547184005Non-Pharmaceutical company11Atom International LimitedTrueTrueORG-100042393Active191469Unit 4, Earls House, Earlsway, Team Valley Trading EstateUnit 4Earls HouseEarlswayTeam Valley Trading EstateGatesheadNE11 0RY826United Kingdom+447540051001michelle.robson@atom-international.orgTrueLOC-10007003896257815Clinicial Evaluator oversight+447540051001michelle.robson@atom-international.org619431180563184021Non-Pharmaceutical company11Suvoda LLCTrueTrueORG-100043523Active191485181 Washington Street Suite 100181 Washington Street Suite 100Conshohocken19428-2082840United States6105722920grey@suvoda.comTrueLOC-10007199696257636105722920grey@suvoda.com619432180574184032Pharmaceutical company10Biomarin Pharmaceutical Inc.TrueTrueORG-100006134Active191498770 Lindaro Street770 Lindaro StreetSan Rafael94901-3991840United States4155066700BAS-Bioanalysis@bmrn.comTrueLOC-10005181596257744155066700BAS-Bioanalysis@bmrn.com619441548857581485Pharmaceutical company10Evidera LimitedTrueTrueORG-100028239Active590668Second Floor, The Ark, 201 Talgarth RoadSecond FloorThe Ark201 Talgarth RoadLondonW6 8BJ826United Kingdom0019102510081Laura.Kelly@evidera.comTrueORG-10002823996258615Embedded interview0019102510081Laura.Kelly@evidera.com619439541041573651Pharmaceutical company10SysnavTrueTrueORG-100026890Active58203572 Rue Emile Loubet72 Rue Emile LoubetVernon272002012France0033278770346contact.syde@sysnav.frTrueORG-10002689096258415Syde device0033278770346contact.syde@sysnav.fr619443180541183999Laboratory/Research/Testing facility9Professional Case Management Clinical Trials LLCTrueTrueORG-100044408Active191461500 East 8th Avenue500 East 8th AvenueDenver80203-3716840United States3032537470legal@procasemanagement.comTrueLOC-10007340396258815home health visits3032537470legal@procasemanagement.com619435180553184011Pharmaceutical company10Cogstate Inc.TrueTrueORG-100045256Active191475195 Church Street Fl 4195 Church Street Fl 4New Haven06510-2009840United States2037735010351-201@cogstate.comTrueLOC-10007464796258015CBCL questionnaire2037735010351-201@cogstate.com619445180566184024Pharmaceutical company10Ardena Bioanalysis B.V.TrueTrueORG-100036987Active191488W.A. Scholtenstraat 7W.A. Scholtenstraat 7Assen9403 AJ2029Netherlands31592344211esther.biemans@ardena.comTrueLOC-100058453962590431592344211esther.biemans@ardena.com619434180565184023Pharmaceutical company10Labcorp Central Laboratory Services S.a.r.l.TrueTrueORG-100011524Active191487Rue Moise-Marcinhes 7Rue Moise-Marcinhes 7Meyrin1217756Switzerland41588227901labcentralcoordinator@covance.comTrueLOC-100022649962579441588227901labcentralcoordinator@covance.com619442180577184035Pharmaceutical company10Precision For Medicine Inc.TrueTrueORG-100041895Active1915018425 Precision Way Suite M8425 Precision Way Suite MFrederick21701-2903840United States2403064100info@precisionformedicine.comTrueLOC-10006907696258715Lab sample storage2403064100info@precisionformedicine.com619444541020573629Pharmaceutical company10Trinds LLCTrueTrueORG-100051849Active5820126425 Living Place6425 Living PlacePittsburgh15206-5122840United States4122242030info@trinds.comTrueORG-10005184996258915Genetic Counseling4122242030info@trinds.com619438180558184016Laboratory/Research/Testing facility9Bioclinica Inc.TrueTrueORG-100033079Active191480211 Carnegie Center211 Carnegie CenterPrinceton08540-6299840United States8007049698customercare@ert.comTrueLOC-10005164296258315ECG/ECHO8007049698customercare@ert.com619430180584184042Pharmaceutical company10Medpace Finland OyTrueTrueORG-100009147Active191508Kaikukatu 4 CKaikukatu 4 CHelsinki005302011Finland+34917900565RS-Advisor-Support@Medpace.comTrueLOC-100073197962573196257429625756+34917900565RS-Advisor-Support@Medpace.com619436180568184026Laboratory/Research/Testing facility9Centogene GmbHTrueTrueORG-100043695Active191491Am Strande 7, StadtmitteAm Strande 7StadtmitteRostock180552013Germany004938180113400limor.oren@centogene.comTrueLOC-1000722489625814004938180113400limor.oren@centogene.com619440541030573640Pharmaceutical company10Agada Biosciences Inc.TrueTrueORG-100051126Active5820241498 Lower Water Street, Mp 11151498 Lower Water StreetMp 1115HalifaxB3J 3R5124Canada9024424011kmcdonald@agadabio.comTrueORG-10005112696258515Dystrophin Western Blot Assay (Muscle Biopsy)9024424011kmcdonald@agadabio.com619446541064573674Non-Pharmaceutical company11Mde Healthcare Services LimitedTrueTrueORG-100052452Active582060Building 329, Doncastle RoadBuilding 329Doncastle RoadBracknellRG12 8PE826United Kingdom00441344300808info@mdgroup.comTrueORG-10005245296259115Patient travel and reimbursement00441344300808info@mdgroup.com619447180916184375Laboratory/Research/Testing facility9LabcorpTrueTrueORG-100011514Active191863P. O. Box 12140P. O. Box 12140Burlington27216-2140840United States3364363522BNCTSUPPORT@Labcorp.comTrueLOC-10008010396259243364363522BNCTSUPPORT@Labcorp.com619437541033573643Pharmaceutical company10Bioagilytix Labs LLCTrueTrueORG-100013030Active5820272300 Englert Drive Suite G2300 Englert Drive Suite GDurham27713-4450840United States8596849473sara.alhakeem@BioAgilytix.comTrueORG-10001303096258215anti-BMN351 abs8596849473sara.alhakeem@BioAgilytix.com164812Pharmaceutical company10Biomarin Pharmaceutical Inc.TrueTrueORG-100006134Active161472164812Pharmaceutical company10Biomarin Pharmaceutical Inc.TrueTrueORG-100006134Active170692105 Digital Drive105 Digital DriveNovato94949-8703840United StatesTrueLOC-100013510TrueCommercial2Study 351-201 is a first in human integrated phase 1/2 Study. In line with EMA guidance on disclosure rules, the study meets the criteria of a Category 2 trial.11046835Diseases [C] - Musculoskeletal Diseases [C05]377176Exon 51 specific phosphorothioate oligonucleotide1Test504718110564554CONCENTRATE FOR SOLUTION FOR INFUSIONPRD105645541Exon 51 specific phosphorothioate oligonucleotideCONCENTRATE FOR SOLUTION FOR INFUSIONBMN 351BMN 351SUB328513BIOMARIN PHARMACEUTICAL INC.10564554328517BIOMARIN PHARMACEUTICAL INC.Nucleic AcidNucleic AcidBMN 351SUB328513NOTASSIGN-False1FalseChemicalPRD10564554BMN 35111INTRAVENIOUS INFUSIONFalseExon 51 specific phosphorothioate oligonucleotidebmn 351CONCENTRATE FOR SOLUTION FOR INFUSIONFalse276037115071150771152027Authorised2023-09-282023-09-28FalseunwillingSpain2023-11-30Authorised134762024-03-082024-03-08134772024-03-082024-03-08157892024-03-182024-03-19891682024-03-182025-10-03891692024-03-182025-10-031108992024-03-182026-05-122026-05-141109002024-03-182026-05-122026-05-14True165312024-03-082024-03-082024-03-182026-05-12False1002311507Under evaluation2023-08-04T11:23:33.6581760111507Authorised2023-11-30T08:20:33.0991Spainacceptable2026-02-02authorized2023-11-302023-11-3021893935186346189828Health care2Hospital Viamed Santa Angela de la CruzFalseFalseORL-000002256Active197737Av. de Jerez, 59Av. de Jerez, 59Sevilla410142027Spain+34639310203mapolgra@yahoo.esFalseORL-0000022562104383MarcosMadruga Garrido+34639310203mapolgra@yahoo.es1Pediatric Neurology1893934185621189097Hospital/Clinic/Other health care facility8Sant Joan De Deu Barcelona HospitalFalseTrueORG-100023083Active196957Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain+34936009733andres.nascimento@sjd.esTrueLOC-1000318322104382Andres EduardoNascimiento Osorio+34936009733andres.nascimento@sjd.esPediatric NeurologyAuthorised271538115081150871152029Authorised2024-03-222024-03-22Trueunwilling_tacitNetherlands2024-04-03Authorised337062024-08-292024-09-09337072024-08-292024-09-09554312024-11-132024-11-13554322024-11-132024-11-131109012024-11-132026-05-122026-05-141109022024-11-132026-05-122026-05-14True449122024-08-292024-09-092024-11-132026-05-12False2046911508Under evaluation2024-01-11T14:35:00.3592909011508Authorised2024-04-03T14:56:53.9311Netherlandsacceptable2026-01-15authorized2024-04-032024-04-0311861093280358284337Hospital/Clinic/Other health care facility8Leids Universitair Medisch Centrum (LUMC)FalseTrueORG-100047879Active298996Albinusdreef 2Albinusdreef 2Leiden2333 ZA2029Netherlands0715262197ehniks@lumc.nlTrueORG-1000478792068275ErikNiks0715262197ehniks@lumc.nlNeurologyAuthorised251128115061150671152018Authorised2023-10-302023-10-30FalseunwillingItaly2023-12-01Authorised184652024-04-302024-05-01184662024-04-302024-05-01463662024-09-182024-09-24463672024-09-182024-09-241108972024-09-182026-05-122026-05-141108982024-09-182026-05-122026-05-14True236982024-04-302024-05-012024-09-182026-05-12False1002211506Under evaluation2023-08-04T11:23:33.6091779711506Authorised2023-12-01T09:04:57.1441Italyacceptable2025-12-15authorized2023-12-012023-12-0131711519183430186900Hospital/Clinic/Other health care facility8Fondazione Policlinico Universitario Agostino Gemelli IRCCSFalseTrueORG-100014358Active194633Largo Francesco Vito 1Largo Francesco Vito 1Rome001682018Italy063015600marika.pane@policlinicogemelli.itTrueLOC-1000202371902645MarikaPane063015600marika.pane@policlinicogemelli.itDepartment of Women's, Child and Public Health Sciences1711521183925187396Hospital/Clinic/Other health care facility8Centro Clinico NemoFalseTrueORG-100011251Active195166Piazza Dell'ospedale Maggiore 3Piazza Dell'ospedale Maggiore 3Milan201622018Italy3495607450valeria.sansone@centrocliniconemo.itTrueLOC-1000157851902647Valeria AdaSansone3495607450valeria.sansone@centrocliniconemo.it1Centro Clinico NeMO of Milano1711520552507585143Hospital/Clinic/Other health care facility8San Raffaele HospitalFalseTrueORG-100029986Active594705Via Olgettina 58Via Olgettina 58Milan201322018Italy0226433036previtali.stefano@hsr.itTrueORG-1000299861902646Stefano 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study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boysGNT-016-MDYFDuchenne Muscular DystrophyFrance:4Belgium:2Spain:221/06/2023FR: 21/06/2023, ES: 08/04/2026, BE: 17/03/2026Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]GenethonLaboratory/Research/Testing facilityPhase I and Phase II (Integrated)- First administration to humans- Safety and tolerability, measured by the incidence of adverse event (AE) or serious adverse event (SAE) evaluated by changes in laboratory parameters, vital signs and in the physical examination, PK/PD endpoints including vector shedding quantification in blood, urine, saliva, feces, Clinical efficacy endpoints including NSAA, Time to 10 Meters Walk/Run Test (10MWRT), Time to Rise From Floor (RFF), 6-Minutes Walk Test (6MWT)Rapamune 1 mg/mL oral solution, rAAV8-hMD1, Placebo AxMP is a solution intended to be administered by oral route for clinical purposes. Placebo AxMP is a pale yellow to yellow solution. Placebo AxMP composition corresponds to the excipients of the authorized product Rapamune® 1 mg/mL oral solution composed of a mixture of Phosal 50PG and Polysorbate 80., RINGER LACTATE FRESENIUS KABI FRANCE, solution pour perfusion, PREDNISOLONE, METHYLPREDNISOLONE, PREDNISOLONE, Soliris 300 mg concentrate for solution for infusion50-17 yearsMale3Both40NSAA: change from baseline at week 52No13/04/202614/04/20262023-06-21T09:09:56.2632026-04-14T03:39:05.8537065824France83522023-06-21T09:09:56.2632026-01-09T17:19:56.8784Belgium565742026-03-17T12:02:29.9732026-04-13T14:54:49.9882Spain565752026-04-08T08:59:35.8032026-04-08T08:59:35.80328352FranceSTART_OF_TRIAL2021-03-01START_OF_RECRUITMENT2021-03-0256574Belgium56575SpainGENETHONUE-986532025-09-052025-09-052025-09-232025-09-23An infusionAn infusion related allergic reaction has been reported. The event was assessed as serious. No sign of severe anaphylaxis.• Add infusion-related reaction (hypersensitivity) in the list of potential risks with GNT0004 Close monitoring of patients, with maintenance of venous access after infu-sion, to manage any potential delayed allergic reaction for at least 3 hours after the end of infusion (duration could be prolonged per investigator’s judgment). • In the event of relevant signs/symptoms, such as tachycardia, tachypnea, lip swelling, difficulty breathing, nasal flaring, urticaria, flushing, lip pruritus, rash, cheilitis, vomiting, nausea, rigors, pyrexia, etc.): the infusion should be stopped immediately and appropriate infusion-related reaction management such as supportive care, antihistamines, and/or other relevant anti-allergic medication (eg additional dose of IV corticosteroid) should be considered • Allergy testing and analysis should be considered as early as possible when the event occurs with the support of an anaphylaxis specialist if deemed necessary. Infusion may be resumed at a reduced rate once symptoms resolve, per PI’s judgment. Infusion should be discontinued in case of severe Anaphylaxis. This urgent safety measure (USM) has been agreed upon by an iDMC convened in emergency. And the iDMC recommended to continue the study with this USM in place, provided the benefice/risk ratio remains positive. The communication with investigators / centers and information to the study participants has been implemented on 19-Sep-2025. The amendment of study core documents (study protocol, IB, ICFs, etc.) will be submitted in due course. Following the recommendation of the iDMC, the study may proceed following implementation of the Urgent Safety Measures (USM).France100000000395France250FRFRATruerAAV8-hMD1GENETHONUS-986542025-09-152025-09-232025-09-23SUSARUE-98653An infusion related allergic reaction has been reported. The event was assessed as serious. No sign of severe anaphylaxis.• Add infusion-related reaction (hypersensitivity) in the list of potential risks with GNT0004 Close monitoring of patients, with maintenance of venous access after infu-sion, to manage any potential delayed allergic reaction for at least 3 hours after the end of infusion (duration could be prolonged per investigator’s judgment). • In the event of relevant signs/symptoms, such as tachycardia, tachypnea, lip swelling, difficulty breathing, nasal flaring, urticaria, flushing, lip pruritus, rash, cheilitis, vomiting, nausea, rigors, pyrexia, etc.): the infusion should be stopped immediately and appropriate infusion-related reaction management such as supportive care, antihistamines, and/or other relevant anti-allergic medication (eg additional dose of IV corticosteroid) should be considered • Allergy testing and analysis should be considered as early as possible when the event occurs with the support of an anaphylaxis specialist if deemed necessary. Infusion may be resumed at a reduced rate once symptoms resolve, per PI’s judgment. Infusion should be discontinued in case of severe Anaphylaxis. This urgent safety measure (USM) has been agreed upon by an iDMC convened in emergency. And the iDMC recommended to continue the study with this USM in place, provided the benefice/risk ratio remains positive. The communication with investigators / centers and information to the study participants has been implemented on 19-Sep-2025. The amendment of study core documents (study protocol, IB, ICFs, etc.) will be submitted in due course. Following the recommendation of the iDMC, the study may proceed following implementation of the Urgent Safety Measures (USM).France2023-505187-11-004Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boysGNT-016-MDYF01/03/2021Duchenne Muscular DystrophyFrance:4Belgium:2Spain:221/06/2023FR: 21/06/2023, ES: 08/04/2026, BE: 17/03/2026Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]GenethonLaboratory/Research/Testing facilityPhase I and Phase II (Integrated)- First administration to humans- Safety and tolerability, measured by the incidence of adverse event (AE) or serious adverse event (SAE) evaluated by changes in laboratory parameters, vital signs and in the physical examination, PK/PD endpoints including vector shedding quantification in blood, urine, saliva, feces, Clinical efficacy endpoints including NSAA, Time to 10 Meters Walk/Run Test (10MWRT), Time to Rise From Floor (RFF), 6-Minutes Walk Test (6MWT)Rapamune 1 mg/mL oral solution, rAAV8-hMD1, Placebo AxMP is a solution intended to be administered by oral route for clinical purposes. Placebo AxMP is a pale yellow to yellow solution. Placebo AxMP composition corresponds to the excipients of the authorized product Rapamune® 1 mg/mL oral solution composed of a mixture of Phosal 50PG and Polysorbate 80., RINGER LACTATE FRESENIUS KABI FRANCE, solution pour perfusion, PREDNISOLONE, METHYLPREDNISOLONE, PREDNISOLONE, Soliris 300 mg concentrate for solution for infusion50-17 yearsMale340NSAA: change from baseline at week 52No13/04/202614/04/20262023-505187-11-00Authorised2021-03-012023-06-21T09:09:56.2632026-04-14T03:39:05.853706582412528350100000000556United Kingdom826GBGBRTrue52016619865104ORAL SOLUTIONPRD3342092EU/1/01/171/0012Rapamune 1 mg/mL oral solutionORAL SOLUTIONSIROLIMUSSUB10537MIGNOEMEA/H/C/000273PFIZER EUROPE MA EEIG9865104233258PFIZER EUROPE MA EEIGChemicalChemicalSIROLIMUSSUB10537MIGL04AA10SIROLIMUS5False4FalseFalsePRD3342092ORAL USETrueRapamune 1 mg/mL oral solutionsirolimusORAL SOLUTION520162110400185SOLUTION FOR INFUSIONPRD103199701rAAV8-hMD1SOLUTION FOR INFUSIONGNT0004ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 8 CONTAINING THE HUMAN MD1 GENESUB206872GENETHON10400185206872GENETHONStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 8 CONTAINING THE HUMAN MD1 GENESUB206872rAAV8-MDYFFalse1TrueEU/3/14/1381ATMP productPRD10319970GNT000466013hMD1This genetically modified recombinant viral vector is composed of an rAAV serotype 8 capsid and an rAAV serotype 2 backbone (rAAV2/8) containing hMD1, a sequence-optimised gene for a human microdystrophin (hMD1) designed to be expressed in appropriate tissues, due to the transcriptional control of a synthetic muscle and cardiac restricted promoter SpC5.12 and to complement disease-associated absence of native DystrophinFalseTrue3118PARENTERAL USEFalserAAV8-hMD1adeno-associated viral vector serotype 8 containing the human md1 geneSOLUTION FOR INFUSION5201631N/AN/AN/APlacebo AxMP is a solution intended to be administered by oral route for clinical purposes. Placebo AxMP is a pale yellow to yellow solution. Placebo AxMP composition corresponds to the excipients of the authorized product Rapamune® 1 mg/mL oral solution composed of a mixture of Phosal 50PG and Polysorbate 80.N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse3N/AFalsePlacebo AxMP is a solution intended to be administered by oral route for clinical purposes. Placebo AxMP is a pale yellow to yellow solution. Placebo AxMP composition corresponds to the excipients of the authorized product Rapamune® 1 mg/mL oral solution composed of a mixture of Phosal 50PG and Polysorbate 80.n/aN/A52016018211916SOLUTION POUR PERFUSIONPRD208541934009 383 178 2 42RINGER LACTATE FRESENIUS KABI FRANCE, solution pour perfusionSOLUTION FOR INJECTION/INFUSIONSODIUM LACTATE SOLUTION, POTASSIUM CHLORIDE, SODIUM CHLORIDE, CALCIUM CHLORIDE DIHYDRATESUB12297MIG, SUB12559MIG, SUB12581MIG, SUB12664MIGFRFRESENIUS KABI FRANCE S.A.S.8211916194446FRESENIUS KABI FRANCE S.A.S.ChemicalChemicalSODIUM LACTATE SOLUTIONSUB12297MIG8211916216616FRESENIUS KABI FRANCE S.A.S.ChemicalChemicalPOTASSIUM CHLORIDESUB12559MIG8211916216617FRESENIUS KABI FRANCE S.A.S.ChemicalChemicalSODIUM CHLORIDESUB12581MIG8211916216618FRESENIUS KABI FRANCE S.A.S.ChemicalChemicalCALCIUM CHLORIDE DIHYDRATESUB12664MIGB05BB01ELECTROLYTES5False3FalseTrueGNT0004 placebo is a sterile solution intended to be administered after dilution by infusion. GNT0004 placebo is composed by the buffer used in the composition of GNT0004 Drug productPRD2085419PARENTERAL USETrueRINGER LACTATE FRESENIUS KABI FRANCE, solution pour perfusionsodium lactate solution, potassium chloride, sodium chloride, calcium chloride dihydrateSOLUTION FOR INJECTION/INFUSION5201643SUB10018MIG-2PREDNISOLONEEFFERVESCENT TABLETPREDNISOLONESUB10018MIG206758ChemicalPREDNISOLONESUB10018MIG---False4FalseFalseCorticosteroidSUB10018MIG11ORAL USEFalsePREDNISOLONEprednisolone5201653SUB08872MIG-2METHYLPREDNISOLONEPOWDER FOR SOLUTION FOR INJECTIONMETHYLPREDNISOLONESUB08872MIG182099ChemicalMETHYLPREDNISOLONESUB08872MIG---False4FalseFalseCorticosteroidSUB08872MIG11INTRAVENIOUS INFUSIONFalseMETHYLPREDNISOLONEmethylprednisolone5201613SUB10018MIG-2PREDNISOLONEEFFERVESCENT TABLETPREDNISOLONESUB10018MIG170418ChemicalPREDNISOLONESUB10018MIG---False4FalseFalseCorticosteroidSUB10018MIG11ORAL USEFalsePREDNISOLONEprednisolone520167110101774CONCENTRATE FOR SOLUTION FOR INFUSIONPRD4318231EU/1/07/393/0012Soliris 300 mg concentrate for solution for infusionSOLUTION FOR INFUSIONECULIZUMABSUB25187ISEMEA/H/C/000791ALEXION EUROPE SAS10101774273378ALEXION EUROPE SASProtein - OtherProtein - OtherECULIZUMABSUB25187ABP-959H5G1.1L04AA25-5False4TrueEU/3/03/166FalsePRD4318231INTRAVENOUS INFUSIONFalseSoliris 300 mg concentrate for solution for infusioneculizumabSOLUTION FOR INFUSIONMicrodystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys103838974f8e8f43-2fa6-4b66-955d-dc5ab5df97b1Essai clinique d’une thérapie génique avec une microdystrophine (GNT0004) dans la myopathie de Duchenne: Étude de phase I/II/III avec une étape de détermination de dose suivie d’une étape d’évaluation de l’efficacité et de la sécurité, en quadruple aveugle comparativement à un placebo, suivie d’une étape de surveillance à long terme chez des garçons ambulatoires34French (France)103838984f8e8f43-2fa6-4b66-955d-dc5ab5df97b1Ensayo clínico de terapia génica con microdistrofina (GNT0004) en distrofia muscular de Duchenne Estudio de fase I/II/III con una parte de determinación de la dosis seguida de una evaluación de la eficacia y la seguridad, parte cuádruple ciega controlada con placebo y luego una parte de seguimiento de la seguridad a largo plazo, en niños ambulantes7SpanishA clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1). After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2). In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3).1038391170796e38-3464-4884-baac-0f0edf079fb0Une étude clinique en 3 parties avec une microdystrophine (appelée GNT0004), une nouvelle thérapie génique chez les garçons atteints de la maladie de Duchenne qui peuvent encore marcher. L'étude commencera par la recherche de la dose de traitement appropriée (partie 1). Ensuite, une étude comparative avec un placebo débutera pour évaluer la sécurité et l'efficacité de la dose appropriée de cette thérapie (partie 2). Enfin, une période de suivi permettra d'étudier la sécurité et l'efficacité du traitement sur une plus longue période (partie 3).34French (France)1038391270796e38-3464-4884-baac-0f0edf079fb0Un estudio clínico en tres partes con microdistrofina (denominada GNT0004), una nueva terapia génica en niños con distrofia muscular de Duchenne que aún pueden caminar. El estudio comenzará con la búsqueda de la dosis adecuada para el tratamiento (parte 1). A continuación, se iniciará un estudio comparativo con placebo para evaluar la seguridad y la eficacia de la dosis adecuada de esta terapia (parte 2). Por último, se llevará a cabo un periodo de seguimiento para seguir investigando la seguridad y la eficacia del tratamiento a largo plazo (parte 3).7SpanishGNT-016-MDYFFalse71Phase I/II trial115086130595Duchenne Muscular Dystrophy103838781a697b45-6814-439c-ba7b-41da8b532765Distrofia muscular de Duchenne7SpanishTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False937973473797314379729637973233797335379730A phase I/II/III study consisting of 3 parts: - Part 1: To determine the dose of IMP: a safe and tolerable dose with acceptable gene expression, to carry over to part 2. - Part 2: To demonstrate clinical efficacy of IMP vs placebo at 1 year after inclusion. To assess the safety and tolerability of IMP vs placebo at 1 year after inclusion. - Part 3. To assess the safety and tolerability of IMP10383909f8f2e4ef-62c1-4617-9e3f-7ac4d571d82dÉtude de phase I/II/III comprenant 3 parties : Partie 1 : - Déterminer la dose de la TG: c’est-à-dire une dose sûre et bien tolérée montrant une expression transgénique acceptable, pour son évaluation en Partie 2 Partie 2 : Avec la dose sélectionnée de la TG: - Démontrer son efficacité clinique par rapport à un placebo un an après le traitement par la TG - Évaluer la sécurité et la tolérance par rapport à un placebo un an après l’inclusion Partie 3 : - Évaluer la sécurité et la tolérance à long terme de la TG34French (France)10383910f8f2e4ef-62c1-4617-9e3f-7ac4d571d82dParte 1: Determinar la dosis del PEI: es decir, una dosis segura y tolerable con una expresión génica aceptable, para pasar a la parte 2 Parte 2: Con la dosis seleccionada del PEI: Demostrar la eficacia clínica del PEI frente a placebo Parte 3: Evaluar la seguridad y la tolerabilidad a largo plazo del PEI.7Spanish4427241Part 2: To assess the biodistribution of IMP10383914a62c07bc-3de6-438e-a144-21ba2b24c22dparte 2: Evaluar la biodistribución del PEI7Spanish10383913a62c07bc-3de6-438e-a144-21ba2b24c22dPartie 2: Évaluer la biodistribution de la TG34French (France)4427252Part 2:To demonstrate the pharmacodynamic activity of IMP103839154de7a73e-5811-402e-b37d-3440b5e1d3fcPartie 2: Démontrer l’activité pharmacodynamique de la TG34French (France)103839164de7a73e-5811-402e-b37d-3440b5e1d3fcParte 2: Demostrar la actividad farmacodinámica del PEI7Spanish4427263Part 2: To assess the immunogenicity of IMP10383917effa0b74-8d2b-4df1-81d3-8402ae5b3fe5Partie 2: Évaluer l’immunogénicité de la TG34French (France)10383918effa0b74-8d2b-4df1-81d3-8402ae5b3fe5Parte 2: Evaluar la inmunogenicidad del PEI7Spanish4427274Part 2:To compare the efficacy on the disease course after 2 years after inclusion, between patients treated with active IMP at first and patients treated after a delay of one year103839207a35ddcf-5384-4a0c-a55a-103d2a3d0632Partie 2: Comparer l’efficacité sur l’évolution de la maladie, 2 ans après l’inclusion, entre les patients traités d'abord par la TG et les patients traités après un délai d’un an34French (France)103839197a35ddcf-5384-4a0c-a55a-103d2a3d0632Parte 2: Comparar la eficacia y los biomarcadores en el curso de la enfermedad entre los participantes tratados con el PEI activo al principio y los participantes tratados tras un retraso de un año.7Spanish8456541Ambulant Male1038390031965eec-e1e9-4e62-bcea-aeeb8b3fd245Varón ambulante7Spanish1038389931965eec-e1e9-4e62-bcea-aeeb8b3fd245De sexe masculin et ambulant34French (France)8456552Being included in the GNT-014-MDYF study1038390262299e2a-9f37-4ecd-913f-920472946a47Estar incluido en el estudio GNT-014-MDYF7Spanish1038390162299e2a-9f37-4ecd-913f-920472946a47Inclus dans l’étude GNT-014-MDYF34French (France)84565636 to 10 years (inclusive)10383904fdfc3313-da31-48f0-887f-dc6d3937dc29Age : 6 à 10 ans (inclus)34French (France)10383903fdfc3313-da31-48f0-887f-dc6d3937dc29De 6 a 10 años de edad (ambos inclusive)7Spanish8456574For participants enrolled in Part 1: BMI scale ≤75th percentile (validated chart in force in country site) For participants enrolled in Part 2: Body weight ≤95th percentile of the BMI or body weight scale (validated chart in force in country site)1038390696047539-722c-40dc-951d-692536c145a3Para los participantes reclutados en la parte 1: escala de IMC percentil ≤75 (tabla validada vigente en el centro del país) Para los participantes reclutados en la parte 2: peso corporal percentil ≤95 de la escala del IMC o del peso corporal (tabla validada vigente en el centro del país)7Spanish1038390596047539-722c-40dc-951d-692536c145a3Poids corporel ≤ 75e percentile de l'échelle de l’IMC (selon l’échelle validée localement)34French (France)8456585Positive gene testing with detailed genotyping confirmation of Duchenne Muscular Dystrophy (DMD), i.e. DMD mutations expected to abolish the production of dystrophin1038390706188b7c-22c4-4ad3-8d50-24bf971effb1Diagnostic génétique positif avec confirmation détaillée du génotype de la myopathie de Duchenne, c'est-à-dire une mutation qui conduit à l’absence de production de dystrophine, sauf pour les patients présentant des mutations affectant les exons 1 à 1734French (France)1038390806188b7c-22c4-4ad3-8d50-24bf971effb1Prueba genética positiva con confirmación detallada de genotipado de distrofia muscular de Duchenne (DMD), es decir, mutaciones de DMD que se prevé que supriman la producción de distrofina7Spanish14465301DMD patients with any mutations affecting: - exons 1 through 17, (and any mutations affecting other exons as per a country’s requirement which will be applicable to this specific country) for Part 1 participants - affecting exons 8 and/or 9 (and any mutations affecting other exons as per a country’s requirement which will be applicable to this specific country) for Part 2 participants103838887e7a4093-6c96-4c5e-b19c-f290aad6a0e6Patients atteints de DMD avec des mutations affectant les exons 1 à 17 et/ou avec des mutations affectant les exons 29 et 3034French (France)103838877e7a4093-6c96-4c5e-b19c-f290aad6a0e6Paciente con DMD con cualquier mutación: - que afecte a los exones 1 a 17 (y cualquier mutación que afecte a otros exones según los requisitos de un país que serán aplicables a este país específico) para los participantes de la parte 1 - que afecte a los exones 8 y/o 9 (y cualquier mutación que afecte a otros exones según los requisitos de un país que serán aplicables a este país específico) para los participantes de la parte 27Spanish14465312Presence of neutralizing antibodies against AAV81038389036f19d5e-d012-412f-ad57-8c7a77c0839dPresencia de anticuerpos neutralizantes contra AAV87Spanish1038388936f19d5e-d012-412f-ad57-8c7a77c0839dPrésence d’anticorps neutralisants contre l’AAV834French (France)14465323Cardiomyopathy based on physical/cardiological examination and echocardiography (or cardiac MRI if available) with Left Ventricular Ejection Fraction (LVEF) below 55% and fractional shortening (SF) below 28%10383892f5c502dd-ee42-408f-9f70-188afa30e100Cardiomiopatía según exploración física/cardiológica y ecocardiografía (o resonancia magnética cardíaca si está disponible) con fracción de eyección del ventrículo izquierdo (FEVI) inferior al 55 % y acortamiento fraccional (AF) inferior al 28 %7Spanish10383891f5c502dd-ee42-408f-9f70-188afa30e100Cardiomyopathie authentifiée sur la base des examens clinique et cardiologique et d’une échocardiographie avec une fraction d'éjection ventriculaire gauche (FEVG) inférieure à 55 % et/ou une diminution de la fraction d’éjection inférieure à 28 %34French (France)14465334Any respiratory assistance needed including non-invasive daytime or nocturnal ventilation1038389385b55b4e-c733-4f3f-80ab-93547edbd561Cualquier asistencia respiratoria necesaria, incluida la ventilación diurna o nocturna no invasiva7Spanish1038389485b55b4e-c733-4f3f-80ab-93547edbd561Assistance respiratoire nécessaire, incluant toute ventilation non invasive diurne ou nocturne34French (France)14465345Inability to perform the planned respiratory functions tests103838950cc77636-87fa-47ee-bdd2-835cc7f5f281Incapacidad para realizar las pruebas de funciones respiratorias previstas7Spanish103838960cc77636-87fa-47ee-bdd2-835cc7f5f281Incapacité à réaliser les tests des fonctions respiratoires34French (France)9904531NSAA: change from baseline at week 52True103838800aa5d95d-be6c-40b0-b646-9054417f67a5North Star Ambulatory Assessment (NSAA): cambio en la puntuación total sin procesar respecto al inicio frente a placebo en la semana 52.7Spanish103838790aa5d95d-be6c-40b0-b646-9054417f67a5NSAA: changement par rapport à la baseline à la semaine 5234French (France)9904541- Safety and tolerability, measured by the incidence of adverse event (AE) or serious adverse event (SAE) evaluated by changes in laboratory parameters, vital signs and in the physical examinationFalse103838825fe92d2f-6c43-4a3f-a097-250741f97d4aCriterios de valoración de seguridad: Incidencia de acontecimientos adversos (AA), acontecimiento(s) adverso(s) grave(s) (AAG), acontecimientos adversos de especial interés (AAEI), reacciones adversas a medicamentos (RAM), constantes vitales, exploración física (sistemas corporales especificados en el protocolo del estudio), valores analíticos y/o procedimientos médicos de anomalías posiblemente de interés clínico (PCSA) (como ECG, ecocardiografía o RM cardíaca).7Spanish103838815fe92d2f-6c43-4a3f-a097-250741f97d4a- Sécurité et tolérabilité, mesurées par l'incidence des événements indésirables (EI) ou des événements indésirables graves (EIG) évalués par des changements dans les paramètres de laboratoire, les signes vitaux et l'examen physique.34French (France)9904552PK/PD endpoints including vector shedding quantification in blood, urine, saliva, fecesFalse10383884703295bb-6f51-430e-b2fc-5e5bc05acb71Criterios de valoración principales PK/PD Cuantificación de la excreción del vector en sangre, orina, saliva y heces (concentración de ADN).7Spanish10383883703295bb-6f51-430e-b2fc-5e5bc05acb71Paramètres pharmacocinétiques et pharmacodynamiques, y compris la quantification de l'excrétion du vecteur dans le sang, l'urine, la salive et les selles34French (France)9904563Clinical efficacy endpoints including NSAA, Time to 10 Meters Walk/Run Test (10MWRT), Time to Rise From Floor (RFF), 6-Minutes Walk Test (6MWT)False10383885564c7434-bfc2-4732-9958-5032e2056edaCriterios de valoración de eficacia: NSAA Tiempo para levantarse del suelo (Rise From Floor, RFF) Tiempo en la prueba de marcha/carrera de 10 metros (10 Meters Walk/Run Test, 10MW/RT) Prueba de marcha de 6 minutos (6-Minute Walk Test, 6MWT).7Spanish10383886564c7434-bfc2-4732-9958-5032e2056edaCritères d'efficacité clinique incluant la NSAA, le temps de marche/course de 10 mètres (10MWRT), le temps pour se relever du sol (RFF), le test de marche de 6 minutes (6MWT).34French (France)2028-10-012028-10-012020-10-0191528Genethon3442552234425625252PatientsFalseTrueTrue353343137541140639EEA National Competent authority16National Agency For The Safety Of Medicine And Health ProductsFalseTrueORG-100003921Active144996143 Boulevard Anatole France143 Boulevard Anatole FranceSt Denis932002012FranceTrueLOC-10005104253344137537140635EU Institution/Body/Agency14European Medicines AgencyFalseTrueORG-100013412Active144992Domenico Scarlattilaan 6Domenico Scarlattilaan 6Amsterdam1083 HS2029NetherlandsTrueLOC-10002026416Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]130595Duchenne Muscular DystrophyTrue142054True418783PublicClinical Development Departmentlthibaut@genethon.fr0169472828120487Laboratory/Research/Testing facility9GenethonTrueTrueORG-100006401Active418784ScientificClinical Development Departmentlthibaut@genethon.fr0169472828120487Laboratory/Research/Testing facility9GenethonTrueTrueORG-100006401Active640058648777686774Laboratory/Research/Testing facility9Mlm Medical Labs GmbHTrueTrueORG-100043721Active704024Dohrweg 63, UeddingDohrweg 63UeddingMönchengladbach410662013Germany4921614642275rmarke@mlm-labs.comTrueORG-10004372199367744921614642275rmarke@mlm-labs.com640063127628130645Pharmaceutical company10SysnavTrueTrueORG-100026890Active13431072 Rue Emile Loubet72 Rue Emile LoubetVernon272002012France33278001098melissa.menard@sysnav.frTrueLOC-10006824099368215Actimyo and Syde devices, for SV 95C measurements.33278001098melissa.menard@sysnav.fr640052127593130610Pharmaceutical company10Eurofins Central Laboratory B.V.TrueTrueORG-100036990Active134274Bergschot 71Bergschot 71Breda4817 PA2029Netherlands0031765737373ingrid.janssens@eurofins.comTrueLOC-10005845799366540031765737373ingrid.janssens@eurofins.com640056127611130628Pharmaceutical company10Eresearchtechnology Inc.TrueTrueORG-100013039Active1342931818 Market Street Ste 26001818 Market Street Ste 2600Philadelphia19103-3600840United States447918055037harry.dorrell@clario.comTrueLOC-10005865799366915ECG central reading447918055037harry.dorrell@clario.com640057123935126934Pharmaceutical company10TFS Trial Form Support ABTrueTrueORG-100008755Active130389P. O. Box 165P. O. Box 165Lund221 002028Sweden0046462801800lakshmi.guduri@tfscro.comTrueLOC-1000540369936701993671119936721299367315Pharmacovigilance9936745993675699367680046462801800lakshmi.guduri@tfscro.com640054649544687542Pharmaceutical company10HistalimTrueTrueORG-100042721Active704927126 Rue Emile Baudot126 Rue Emile BaudotMontpellier340002012France33499549348olucas@cerbaresearch.comTrueORG-100042721993667433499549348olucas@cerbaresearch.com640061127635130652Hospital/Clinic/Other health care facility8Association Institut De MyologieFalseTrueORG-100046467Active134318Batiment Babinski Groupe 47 83, 47 Boulevard De L HopitalBatiment Babinski Groupe 47 8347 Boulevard De L HopitalParis750132012France33142165879jy.hogrel@institut-myologie.orgTrueLOC-10007666599368015Central reading Myotools data33142165879jy.hogrel@institut-myologie.org640055127608130625Pharmaceutical company10Genosafe S.A.S.TrueTrueORG-100013179Active1342891 Rue De L Internationale, P. O. Box 400641 Rue De L InternationaleP. O. Box 40064Evry Cedex910022012France0033648265084cmasurier@genosafe.comTrueLOC-10002114799366840033648265084cmasurier@genosafe.com640059127623130640Non-Pharmaceutical company11Anju Software Inc.TrueTrueORG-100047042Active1343054500 South Lakeshore Drive Suite 6204500 South Lakeshore Drive Suite 620Tempe85282-7298840United States0049228227440Ninel.Dadon@AnjuSoftware.comTrueLOC-10007766199367870049228227440Ninel.Dadon@AnjuSoftware.com640053649558687556Hospital/Clinic/Other health care facility8Hopital Necker Enfants MaladesFalseTrueORG-100023257Active704945149 Rue De Sevres149 Rue De SevresParis750152012France33144494115volodia.dangouloff-ros@aphp.frTrueORG-10002325799366615centralized cardiac MRI reading33144494115volodia.dangouloff-ros@aphp.fr640062649556687554Hospital/Clinic/Other health care facility8Association Institut De MyologieFalseTrueORG-100046467Active704941Batiment Babinski Groupe 47 83, 47 Boulevard De L HopitalBatiment Babinski Groupe 47 8347 Boulevard De L HopitalParis750132012France33142165896h.reyngoudt@institut-myologie.orgTrueORG-10004646799368115Central reading of Muscular MRI33142165896h.reyngoudt@institut-myologie.org640060649604687602Industry3BIOSSECTrueFalseORL-000012060Active7050159 rue Anatole de la Forge9 rue Anatole de la ForgeParis750172012France33635275424guemas@biossec.comFalseORL-0000120609936791033635275424guemas@biossec.com120487Laboratory/Research/Testing facility9GenethonTrueTrueORG-100006401Active117532120487Laboratory/Research/Testing facility9GenethonTrueTrueORG-100006401Active1236061 Rue De L Internationale1 Rue De L InternationaleEvry-Courcouronnes910002012FranceTrueLOC-100011143FalseNon-Commercial1Phase I/II trial112725916Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]388146rAAV8-hMD11Test520162110400185SOLUTION FOR INFUSIONPRD103199701rAAV8-hMD1SOLUTION FOR INFUSIONGNT0004ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 8 CONTAINING THE HUMAN MD1 GENESUB206872GENETHON10400185206872GENETHONStructurally Diverse Substance - OtherStructurally Diverse Substance - OtherADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 8 CONTAINING THE HUMAN MD1 GENESUB206872rAAV8-MDYFFalse1TrueEU/3/14/1381ATMP productPRD10319970GNT000466013hMD1This genetically modified recombinant viral vector is composed of an rAAV serotype 8 capsid and an rAAV serotype 2 backbone (rAAV2/8) containing hMD1, a sequence-optimised gene for a human microdystrophin (hMD1) designed to be expressed in appropriate tissues, due to the transcriptional control of a synthetic muscle and cardiac restricted promoter SpC5.12 and to complement disease-associated absence of native DystrophinFalseTrue3118PARENTERAL USEFalserAAV8-hMD1adeno-associated viral vector serotype 8 containing the human md1 geneSOLUTION FOR INFUSION388144RINGER LACTATE FRESENIUS KABI FRANCE, solution pour perfusion3Placebo52016018211916SOLUTION POUR PERFUSIONPRD208541934009 383 178 2 42RINGER LACTATE FRESENIUS KABI FRANCE, solution pour perfusionSOLUTION FOR INJECTION/INFUSIONSODIUM LACTATE SOLUTION, POTASSIUM CHLORIDE, SODIUM CHLORIDE, CALCIUM CHLORIDE DIHYDRATESUB12297MIG, SUB12559MIG, SUB12581MIG, SUB12664MIGFRFRESENIUS KABI FRANCE S.A.S.8211916194446FRESENIUS KABI FRANCE S.A.S.ChemicalChemicalSODIUM LACTATE SOLUTIONSUB12297MIG8211916216616FRESENIUS KABI FRANCE S.A.S.ChemicalChemicalPOTASSIUM CHLORIDESUB12559MIG8211916216617FRESENIUS KABI FRANCE S.A.S.ChemicalChemicalSODIUM CHLORIDESUB12581MIG8211916216618FRESENIUS KABI FRANCE S.A.S.ChemicalChemicalCALCIUM CHLORIDE DIHYDRATESUB12664MIGB05BB01ELECTROLYTES5False3FalseTrueGNT0004 placebo is a sterile solution intended to be administered after dilution by infusion. GNT0004 placebo is composed by the buffer used in the composition of GNT0004 Drug productPRD2085419PARENTERAL USETrueRINGER LACTATE FRESENIUS KABI FRANCE, solution pour perfusionsodium lactate solution, potassium chloride, sodium chloride, calcium chloride dihydrateSOLUTION FOR INJECTION/INFUSION388147Placebo AxMP is a solution intended to be administered by oral route for clinical purposes. Placebo AxMP is a pale yellow to yellow solution. Placebo AxMP composition corresponds to the excipients of the authorized product Rapamune® 1 mg/mL oral solution composed of a mixture of Phosal 50PG and Polysorbate 80.3Placebo5201631N/AN/AN/APlacebo AxMP is a solution intended to be administered by oral route for clinical purposes. Placebo AxMP is a pale yellow to yellow solution. Placebo AxMP composition corresponds to the excipients of the authorized product Rapamune® 1 mg/mL oral solution composed of a mixture of Phosal 50PG and Polysorbate 80.N/AN/AN/AN/AISN/AN/AN/AN/AN/AN/AFalse3N/AFalsePlacebo AxMP is a solution intended to be administered by oral route for clinical purposes. Placebo AxMP is a pale yellow to yellow solution. Placebo AxMP composition corresponds to the excipients of the authorized product Rapamune® 1 mg/mL oral solution composed of a mixture of Phosal 50PG and Polysorbate 80.n/aN/A388151Soliris 300 mg concentrate for solution for infusion4Auxiliary520167110101774CONCENTRATE FOR SOLUTION FOR INFUSIONPRD4318231EU/1/07/393/0012Soliris 300 mg concentrate for solution for infusionSOLUTION FOR INFUSIONECULIZUMABSUB25187ISEMEA/H/C/000791ALEXION EUROPE SAS10101774273378ALEXION EUROPE SASProtein - OtherProtein - OtherECULIZUMABSUB25187ABP-959H5G1.1L04AA25-5False4TrueEU/3/03/166FalsePRD4318231INTRAVENOUS INFUSIONFalseSoliris 300 mg concentrate for solution for infusioneculizumabSOLUTION FOR INFUSION388149METHYLPREDNISOLONE4Auxiliary5201653SUB08872MIG-2METHYLPREDNISOLONEPOWDER FOR SOLUTION FOR INJECTIONMETHYLPREDNISOLONESUB08872MIG182099ChemicalMETHYLPREDNISOLONESUB08872MIG---False4FalseFalseCorticosteroidSUB08872MIG11INTRAVENIOUS INFUSIONFalseMETHYLPREDNISOLONEmethylprednisolone388150Rapamune 1 mg/mL oral solution4Auxiliary52016619865104ORAL SOLUTIONPRD3342092EU/1/01/171/0012Rapamune 1 mg/mL oral solutionORAL SOLUTIONSIROLIMUSSUB10537MIGNOEMEA/H/C/000273PFIZER EUROPE MA EEIG9865104233258PFIZER EUROPE MA EEIGChemicalChemicalSIROLIMUSSUB10537MIGL04AA10SIROLIMUS5False4FalseFalsePRD3342092ORAL USETrueRapamune 1 mg/mL oral solutionsirolimusORAL SOLUTION388148PREDNISOLONE4Auxiliary5201643SUB10018MIG-2PREDNISOLONEEFFERVESCENT TABLETPREDNISOLONESUB10018MIG206758ChemicalPREDNISOLONESUB10018MIG---False4FalseFalseCorticosteroidSUB10018MIG11ORAL USEFalsePREDNISOLONEprednisolone388145PREDNISOLONE4Auxiliary5201613SUB10018MIG-2PREDNISOLONEEFFERVESCENT TABLETPREDNISOLONESUB10018MIG170418ChemicalPREDNISOLONESUB10018MIG---False4FalseFalseCorticosteroidSUB10018MIG11ORAL USEFalsePREDNISOLONEprednisoloneFalse2704588352835255152012Authorised2023-06-142023-06-14TrueFrance2023-06-21Authorised23522021-03-012023-06-2623532021-03-012023-06-2623542021-03-012023-06-2620492021-03-022023-06-2620502021-03-022023-06-26True26072021-03-012023-06-262021-03-02False59738352Under evaluation2023-05-10T17:48:33.53277018352Authorised2023-06-21T09:09:55.5871Franceauthorized2023-06-212023-06-21201853628127555130572Hospital/Clinic/Other health care facility8Les Hopitaux Universitaires De StrasbourgFalseTrueORG-100006709Active1342341 Avenue Moliere, Bp 491 Avenue MoliereBp 49Strasbourg Cedex 2670982012France0033388127743vincent.laugel@chru-strasbourg.frTrueLOC-1000751132060019VincentLaugel0033388127743vincent.laugel@chru-strasbourg.fr2Service de pédiatrie1853629634078671997Hospital/Clinic/Other health care facility8Hospices Civils De LyonFalseTrueORG-100006597Active68635359 Boulevard Pinel59 Boulevard PinelBron695002012France0033472116675laure.le-goff@chu-lyon.frTrueORG-1000065972060020LaureLe Goff0033472116675laure.le-goff@chu-lyon.fr1Service MPR pédiatrique L’Escale HFME1853631634093672012Educational Institution1University Of BordeauxTrueTrueORG-100030623Active6863681 Place Amelie Raba Leon, Cs 912861 Place Amelie Raba LeonCs 91286Bordeaux Cedex330762012France0033557820234caroline.espil@chu-bordeaux.frTrueORG-1000306232060022CarolineEspil-Taris0033557820234caroline.espil@chu-bordeaux.fr1Neuropédiatric department, Children Hospital1853627127558130575Hospital/Clinic/Other health care facility8Association Institut De MyologieFalseTrueORG-100046467Active134237Porte 20 2eme Etage, 26 Avenue Du Docteur Arnold NetterPorte 20 2eme Etage26 Avenue Du Docteur Arnold NetterParis750122012France0033144736537s.delucia@institut-myologie.orgTrueLOC-1000781452060018SilvanaDe Lucia0033144736537s.delucia@institut-myologie.org1Institut de Myologie1853632638366676305Hospital/Clinic/Other health care facility8Centre Hospitalier Regional Et Universitaire De BrestFalseTrueORG-100007294Active6914885 Avenue Marechal Foch, Bp 8245 Avenue Marechal FochBp 824Brest Cedex 2296092012France0033298223657juliette.ropars@chu-brest.frTrueORG-1000072942060023JulietteRopars0033298223657juliette.ropars@chu-brest.fr1Service de pédiatrie - CRMR Maladies neuromusculaires1853630127565130582Hospital/Clinic/Other health care facility8Centre Hospitalier Universitaire De LilleFalseTrueORG-100006742Active134244Avenue Eugene AvineeAvenue Eugene AvineeLille Cedex590372012France0033320446058jeanbaptiste@chru-lille.frTrueLOC-1000454982060021Jean-BaptisteDavion0033320446058jeanbaptiste@chru-lille.fr1Antenne Pédiatrique du CICAuthorised293850565755657555152027Authorised2026-04-012026-04-01FalseSpain2026-04-08AuthorisedFalseFalse25157356575Under evaluation2026-01-15T12:11:50.86634053356575Authorised2026-04-08T08:59:35.3793Spainacceptable2026-04-01authorized2026-04-082026-04-08102029192896905938978Hospital/Clinic/Other health care facility8Hospital Universitario Y Politecnico La FeFalseTrueORG-100029610Active1002433Avenida Fernando Abril Martorell 106Avenida Fernando Abril Martorell 106Valencia460262027Spain+34961244153muelas_nur@gva.esTrueORG-1000296102253397NuriaMuelas+34961244153muelas_nur@gva.es1Neurology2029191896917938990Hospital/Clinic/Other health care facility8Hospital Sant Joan De Deu BarcelonaFalseTrueORG-100023083Active1002450Passeig De Sant Joan De Deu 2Passeig De Sant Joan De Deu 2Esplugues De Llobregat089502027Spain34936009733andres.nascimento@sjd.esTrueORG-1000230832253396JavierNascimiento34936009733andres.nascimento@sjd.es1NeurologyAuthorised296298565745657455152002Authorised2026-03-022026-03-02FalseBelgium2026-03-17AuthorisedFalseFalse33737556574Authorised2026-03-17T12:02:28.07425157256574Under evaluation2026-01-15T12:09:52.7063Belgiumauthorized2026-03-172026-03-17102050444894705936769Hospital/Clinic/Other health care facility8UZ LeuvenFalseTrueORG-100006001Active999695Herestraat 49Herestraat 49Leuven30002002Belgium+3216343827nmrc.kinderen@uzleuven.beTrueORG-1000060012276355LiesbethDe Waele+3216343827nmrc.kinderen@uzleuven.be2Pediatric Neurology2050445925118967777Hospital/Clinic/Other health care facility8Association Hospitaliere De Bruxelles Hopital Universitaire Des Enfants Reine FabiolaFalseTrueORG-100045965Active1036908Jean Joseph Crocqlaan 15Jean Joseph Crocqlaan 15Brussels10202002Belgium+3224773376nicolas.deconinck@hubruxelles.beTrueORG-1000459652276356NicolasDeconinck+3224773376nicolas.deconinck@hubruxelles.beNeuromuscular Diseases Reference CenterAuthorised7126INITIALAuthorised2023-505187-11-00Authorised2023-05-10acceptable2023-06-092667483528352France2012acceptable2023-06-20authorized2023-06-21AuthorisedFranceAuthorised2023-06-21Authorised2023-06-218352FranceAuthorisedINFalse316171268352France2023-06-21T09:09:56.263authorizedacceptabledecision2667411670INITIALTrue14753NON SUBSTANTIAL MODIFICATIONAuthorised2023-505187-11-00Authorised2023-10-27acceptable2023-06-092023-10-278352FranceAuthorisedNSM-2PART_IFalse7845147538352France2023-10-27T16:06:56.749authorizeddecision21352NON SUBSTANTIAL MODIFICATIONTrue17422NON SUBSTANTIAL MODIFICATIONAuthorised2023-505187-11-00Authorised2023-12-19acceptable2023-06-092023-12-198352FranceAuthorisedNSM-3PART_IFalse10473174228352France2023-12-19T17:43:21.476authorizeddecision25373NON SUBSTANTIAL MODIFICATIONTrue30384NON SUBSTANTIAL MODIFICATIONAuthorised2023-505187-11-00Authorised2024-07-08acceptable2023-06-092024-07-088352FranceAuthorisedNSM-6PART_IFalse28132303848352France2024-07-08T14:30:30.116authorizeddecision46033NON SUBSTANTIAL MODIFICATIONTrue38808SUBSTANTIAL MODIFICATIONAuthorised2023-505187-11-00Authorised2024-11-13acceptable2025-02-1414920483528352France2012acceptable2024-12-03authorized2023-06-21AuthorisedFranceAuthorised2023-06-21Authorised2025-02-178352FranceAuthorisedSM-1PART_I_AND_PART_IIFalse58154388088352France2025-02-17T10:23:40.386authorizedacceptabledecision14920473345SUBSTANTIAL MODIFICATIONTrue51598SUBSTANTIAL MODIFICATIONAuthorised2023-505187-11-00Authorised2025-04-02acceptable2025-07-2419987783528352France2012acceptable2025-05-02authorized2023-06-21AuthorisedFranceAuthorised2023-06-21Authorised2025-07-258352FranceAuthorisedSM-2PART_I_AND_PART_IIFalse82155515988352France2025-07-25T17:27:23.516authorizedacceptabledecision19987789213SUBSTANTIAL MODIFICATIONTrue67709SUBSTANTIAL MODIFICATIONAuthorised2023-505187-11-00Authorised2025-10-16acceptable2026-01-0524983283528352France2012acceptable2025-11-14authorized2023-06-21AuthorisedFranceAuthorised2023-06-21Authorised2026-01-088352FranceAuthorisedSM-3PART_I_AND_PART_IIFalse104286677098352France2026-01-08T18:39:24.23authorizedacceptabledecision249832108676SUBSTANTIAL MODIFICATIONTrue74136NON SUBSTANTIAL MODIFICATIONAuthorised2023-505187-11-00Authorised2026-01-09acceptable2026-01-0527045883528352France2012authorized2023-06-21AuthorisedFranceAuthorised2023-06-21Authorised2026-01-098352FranceAuthorisedNSM-7PART_IIFalse104415741368352France2026-01-09T17:19:56.878authorizeddecision270458113637NON SUBSTANTIAL MODIFICATIONTrue74166NON SUBSTANTIAL MODIFICATIONAuthorised2023-505187-11-00Authorised2026-01-09acceptable2026-01-052026-01-098352FranceAuthorisedNSM-8PART_IFalse104426741668352France2026-01-09T18:27:27.963authorizeddecision113673NON SUBSTANTIAL MODIFICATIONTrue74185SUBSEQUENT ADDITION OF MSCAuthorised2023-505187-11-00Authorised2026-01-152851835657456574Belgium2002acceptable2026-03-16authorized2026-03-17AuthorisedBelgiumAuthorised2026-03-17Authorised2026-03-1756574BelgiumAuthorisedAM-1False1147327418556574Belgium2026-03-17T12:02:29.973authorizedacceptabledecision285183120951SUBSEQUENT ADDITION OF MSCFalse74186SUBSEQUENT ADDITION OF MSCAuthorised2023-505187-11-00Authorised2026-01-152938505657556575Spain2027acceptable2026-04-01authorized2026-04-08AuthorisedSpainAuthorised2026-04-08Authorised2026-04-0856575SpainAuthorisedAM-2False1178727418656575Spain2026-04-08T08:59:35.803authorizedacceptabledecision293850119922SUBSEQUENT ADDITION OF MSCFalse81784NON SUBSTANTIAL MODIFICATIONAuthorised2023-505187-11-00Authorised2026-04-132962985657456574Belgium2002authorized2026-03-17AuthorisedBelgiumAuthorised2026-03-17Authorised2026-04-1356574BelgiumAuthorisedNSM-9PART_IIFalse1186698178456574Belgium2026-04-13T14:54:49.988authorizeddecision296298125283NON SUBSTANTIAL MODIFICATIONFalseFrance83522023-06-21T09:09:56.2632026-01-09T17:19:56.8784Belgium565742026-03-17T12:02:29.9732026-04-13T14:54:49.9882Spain565752026-04-08T08:59:35.8032026-04-08T08:59:35.8032True2020-002093-278352FranceSTART_OF_TRIAL2021-03-01START_OF_RECRUITMENT2021-03-0256574Belgium56575SpainGENETHONUE-986532025-09-052025-09-052025-09-232025-09-23An infusionAn infusion related allergic reaction has been reported. The event was assessed as serious. No sign of severe anaphylaxis.• Add infusion-related reaction (hypersensitivity) in the list of potential risks with GNT0004 Close monitoring of patients, with maintenance of venous access after infu-sion, to manage any potential delayed allergic reaction for at least 3 hours after the end of infusion (duration could be prolonged per investigator’s judgment). • In the event of relevant signs/symptoms, such as tachycardia, tachypnea, lip swelling, difficulty breathing, nasal flaring, urticaria, flushing, lip pruritus, rash, cheilitis, vomiting, nausea, rigors, pyrexia, etc.): the infusion should be stopped immediately and appropriate infusion-related reaction management such as supportive care, antihistamines, and/or other relevant anti-allergic medication (eg additional dose of IV corticosteroid) should be considered • Allergy testing and analysis should be considered as early as possible when the event occurs with the support of an anaphylaxis specialist if deemed necessary. Infusion may be resumed at a reduced rate once symptoms resolve, per PI’s judgment. Infusion should be discontinued in case of severe Anaphylaxis. This urgent safety measure (USM) has been agreed upon by an iDMC convened in emergency. And the iDMC recommended to continue the study with this USM in place, provided the benefice/risk ratio remains positive. The communication with investigators / centers and information to the study participants has been implemented on 19-Sep-2025. The amendment of study core documents (study protocol, IB, ICFs, etc.) will be submitted in due course. Following the recommendation of the iDMC, the study may proceed following implementation of the Urgent Safety Measures (USM).France100000000395France250FRFRATruerAAV8-hMD1GENETHONUS-986542025-09-152025-09-232025-09-23SUSARUE-98653An infusion related allergic reaction has been reported. The event was assessed as serious. No sign of severe anaphylaxis.• Add infusion-related reaction (hypersensitivity) in the list of potential risks with GNT0004 Close monitoring of patients, with maintenance of venous access after infu-sion, to manage any potential delayed allergic reaction for at least 3 hours after the end of infusion (duration could be prolonged per investigator’s judgment). • In the event of relevant signs/symptoms, such as tachycardia, tachypnea, lip swelling, difficulty breathing, nasal flaring, urticaria, flushing, lip pruritus, rash, cheilitis, vomiting, nausea, rigors, pyrexia, etc.): the infusion should be stopped immediately and appropriate infusion-related reaction management such as supportive care, antihistamines, and/or other relevant anti-allergic medication (eg additional dose of IV corticosteroid) should be considered • Allergy testing and analysis should be considered as early as possible when the event occurs with the support of an anaphylaxis specialist if deemed necessary. Infusion may be resumed at a reduced rate once symptoms resolve, per PI’s judgment. Infusion should be discontinued in case of severe Anaphylaxis. This urgent safety measure (USM) has been agreed upon by an iDMC convened in emergency. And the iDMC recommended to continue the study with this USM in place, provided the benefice/risk ratio remains positive. The communication with investigators / centers and information to the study participants has been implemented on 19-Sep-2025. The amendment of study core documents (study protocol, IB, ICFs, etc.) will be submitted in due course. Following the recommendation of the iDMC, the study may proceed following implementation of the Urgent Safety Measures (USM).FranceBoth3 2022-500703-49-014AuthorisedPhase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SQY51 in Paediatric and Adult Patients with a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy, including a i) 13-week Phase 1 Multiple Dose Escalation Phase, a ii) 49-week Phase 2a, a iii) 102-week extension phase, or a iv) 28-months long-term follow-up.AVANCE1-1/2aDuchenne muscular dystrophyFrance:421/11/2022FR: 21/11/2022Diseases [C] - Musculoskeletal Diseases [C05]Sqy TherapeuticsPharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansPhase 2 : Motor function: - Motor Function Measure (MFM) score for ambulant and non-ambulant patients. - Performance Upper Limb (PUL) scale for ambulant and non-ambulant patients. - 6-minute walk test for ambulant patients. - 1-minute walk test (included in the 6-minute walk test) for ambulant patients. - 10-meter run/walk time for ambulant patients. - Rise from the floor for ambulant patients., Phase 2: Respiratory function: -Vital capacity, forced vital capacity, inspiratory capacity, and expiratory residual volume, functional residual capacity, residual volume, total lung capacity, all absolute values as well as percent predicted, as well as forced expiratory flow during 1 s, peak expiratory flow, peak cough flow, Tiffeneau index. -Maximal inspiratory and expiratory pressures, and sniff nasal inspiratory pressure all absolute values as well as % predicted. - Twitch mouth pressure, Phase 2 : Cardiac function : - echocardiography (LVEDD, LVESD, LVSWT, LVPWT, GLS, LVEFMS, LVMI, mitral inflow velocities, DT, E/é, LAVI, TAPSE, RV s', sPAP) - cardiac MRI (LVEDD, RVEDD, LVESD, RVESD, LVEF, RVEF, LGE, GCS, GRS, LVEDV, LVESV, systolic ejection volume, TAPSE, native T1, T2, ECV)., Phase 2 : Muscle strength: Measurements of pinch, maximal isometric grip strength using MyoGrip as well as strength of elbow flexion and extension, and knee flexion and extension using hand-held dynamometry., Phase 2 : Quality of Life Endpoints/Self-Assessment: Pediatric Quality of Life Inventory (PedsQL) – DMD module, Phase 2 : Serum biomarker analysis: Muscle necrosis biomarkers (CPK and Myomesine-3)., Phase 2 : Muscle biomarkers: - Levels of DMD exon-51 transcripts, dystrophin protein, histopathology of muscle biopsy. Lean cross-sectional areas (lCS), fat fraction (FF) and water T2 using quantitative MRI of forearm and lower leg (all patients)., Phase 1: Levels of SQY51, its main metabolites in serum and urine, and AUC calculation., Phase 2: Levels of SQY51 and main metabolites in serum, urine and muscle biopsy sample, PART III - EXTENSION PHASE : Primary and secondary objectives and endpoints of the Extension Phase are identical of the Phase 2a, with the exception that another muscle biopsy will not be performed.59618-64 years, 0-17 yearsMale1EEA12Type, frequency, severity, timing and relationship to SQY51 of: Adverse events (AEs), discontinuations due to AEs, serious AEs (SAEs)., PART III - EXTENSION PHASE : Primary and secondary objectives and endpoints of the Extension Phase are identical of the Phase 2a, with the exception that another muscle biopsy will not be performed.No27/02/202628/02/20262022-11-21T09:35:18.4452026-02-28T02:36:45.7629450834France19022022-11-21T09:35:18.4452026-02-27T15:06:30.84541902TH-173412024-03-156Medicinal Product relatedFalseFalseTrueFollowing an analysis of the batch records (carried out on 12/03/2024) of the finished product SQY51 used in the AVANCE 1 phase 1 clinical study (1st patient included in the study on 03/05/2023) and stability data, SQY Therapeutics discovered a non-compliance regarding the expiry dates of batch DPSQYM001 (packaged in 3 sub-batches). Below is the information recorded in the batch certificates - investigational medicinal product for human use issued by COLCA /EUROMED PHARMA: - Lot DPSQYM001A expiry date 06/2023, batch certificate signed by QP on 20/12/2022 - Lot DPSQYM001B expiry date 05/2024, batch certificate signed by QP on 14/09/2023 - Lot DPSQYM001C expiry date 05/2024, batch certificate signed by QP on 14/09/2023 SQY51 Finished Product Information: - Absence of a technical batch for SQY51 performed prior to the start of the clinical study - 1st batch of SQY 51 DPSQYM001 manufactured at GTP Bioways on 24/11/2021 then packaged and certified for the clinical study by EUROMED PHARMA (ex COLCA) on 20/12/2022 (batch DPSQYM001A), expiry date of certificate 06/2023 - Stabilization of the DPSQYM001 batch at AXOLABS in July 2022 according to the following scheme (Q0 &#34;analytical&#34; &#61; 27/07/2022): o Normal conditions / Storage in stability chamber at -20°C &#43;/- 5°C / T0 &#43; T3M &#43; T6M &#43; T9M &#43; T12M &#43; T18M &#43; T24M o Accelerated conditions / Storage in stability chamber at &#43;5°C &#43;/- 3°C / T0 &#43; T2M &#43; T4M &#43; T6M &#43; T9M &#43; T12M &#43; T18M &#43; T24M o Stress test / Storage in stability chamber at &#43;40°C &#43;/- 2°C / T0 &#43; T1M - Stability results known to date: stress test &#43;40°C &#43;/- 2°C up to T1M, accelerated conditions at &#43;5°C &#43;/- 3°C up to T12M, and normal conditions at -20°C &#43;/- 5°C up to T12M. To date, no OOS has been detected at any of the check-ins. - Extrapolation of stability data is not applicable for SQY51 because the product is stored at -20°C (in accordance with ICH Q1E – Evaluation of stability data) In fact, you will find below the expiry dates that should have been entered on the different dates of issue of the certificates of the batch DPSQYM001 in view of the state of knowledge (stability results and exclusion of extrapolation): - Expiry date 11/2022, on the date of signature of the batch certificate on 20/12/2022 - Expiry Date 07/2023, on the date of signature of the batch certificate on 14/09/2023 Please also note that this non-compliance cannot be considered a public health concern as no serious adverse reactions were reported in the Phase 1 study. Following the observation of this non-compliance, SQY Therapeutics immediately requested that the DPSQYM001 batch be quarantined on 13/03/2024 within the pharmacy of the investigator site. The last administration of this batch took place at the investigator site on 11/03/2024 as part of this AVANCE 1 - phase 1 study. This Phase 1 study has not been yet finalized. Upcoming visits are described in Appendix 1 of the attached letter. Currently, there is another batch of SQY51 finished product, never administered to humans, batch DPSQYM002 manufactured on 27/09/2022 with an expiry date of 05/2024 (batch certificate signed on 23/10/2023) : expiry date in line with the stability data known to date. The 18-month stability data is expected to be confirmed by 22/03/2024. Part of this batch has been sent to the investigator site, but no administration will take place without the authorization of the ANSM. SQY Therapeutics is aware of the seriousness of this non-compliance and inform you that a deviation has been put in place in order to implement emergency actions, a deviation involving the CRO BIOTRIAL, the investigator site and EUROMED PHARMA, all of whom have been informed of this notification.False2024-03-15T17:58:24This same notification will be the subject of an email communication. The actions SQY Therapeutics intends to implement are as follows: - Suspension of the administration of SQY51 in the context of the AVANCE 1 – phase 1 clinical study pending feedback from the ANSM for the continuation of the study with the administration of batch DPSQYM002. 18-month stability data for batch DPSQYM002 is expected to be available by 22/03/2024. - Continuation of analyses, recruitment and follow-up visits of the AVANCE 1 – Phase 1 clinical study, as mentioned in Appendix 1. - Emergency analysis of the remaining vials of the batch DPSQYM001 - Communication with patients after approval by the ANSM - Communication with the DSMB/steering committee after approval by the ANSM - Communication with the hospital of the investigator site after approval by the ANSMFalse2024-03-151902FranceFR1902TH-764482025-03-144Safety related (clinical or pre-clinical results)FalseFalseTrueThe AVANCE1 phase 1/2a trial has temporarily paused injections as a precaution to fully assess recent SUSARs in three patients (one with potential treatment-related proteinuria, one with a Grade 4 Cerebral Venous Thrombosis, and one with Polycythemia). This decision, made with the Steering Committee on March 14, 2025, necessitates a thorough protocol review to refine treatment suspension/discontinuation criteria. While these events haven&#39;t changed the benefit/risk ratio, they were unexpected for the SQY51. Please refer to the attached Cover letter for a detailed overview. Attached are CIOMS forms for patients 01-015 and 01-008 and 01-002. Data collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.False2025-03-26T09:37:33A DSMB meeting was held on March 21st to discuss these events and potential measures, meeting minutes are ongoing. A letter was addressed to the Principal Investigator on the 19th March 2025 for this temporary halt (please see attached). All patients were informed of these injections temporary halt by the Principal Investigator. Encouragingly, early pharmacodynamic data shows reduced DMD-associated serum biomarkers, and the first completed patient&#39;s muscle biopsy revealed dystrophin restoration and stable ambulatory function. Ex-vivo studies support these findings, indicating SQY51&#39;s potential to normalize key muscle-related pathways. The observed polycythemia might even suggest a beneficial normalization of dystrophic musculature. Consequently, adjustments to SQY51 dosage, injection frequency, and treatment criteria are being considered, alongside intensified biomarker monitoring. An urgent meeting with the Agency is requested to discuss this data, protocol adjustments, and the continuation of treatment for favorable responders. Finally, an extension is requested for the substantial protocol amendment submission deadline (currently April 14thfollowing DSUR RFIs). A USM is submitted in parallel of this notificationFalse1902FranceFR1902FranceRESTART_OF_TRIAL2025-11-05START_OF_TRIAL2023-01-05RESTART_OF_RECRUITMENT2024-09-09START_OF_RECRUITMENT2023-04-26TEMPORARY_HALT2025-03-14USM #1US-517832024-10-042024-10-152024-10-15OTHERAs indicated in e-mails sent to ANSM on 07/10/2024 and 14/10/2024, analysis of the areas under the curve (AUC) of patients who completed Part I, suggests the possibility of overexposure to the investigational medicinal product, SQY51, in the heaviest patients, due to the method used to calculate the dose administered: mg/kg. As such, this is a potential risk of overexposure to the investigational medicinal product detected on 30/09/2024. On the same day, members of the Data and Safety Monitoring Board (DSMB) and experts from the Comité de Pilotage, including a methodologist and a pharmacokineticist, met to discuss the reassignment of patients to the cohorts, based on analyses of the pharmacokinetic (PK) data evaluated in Part I of the study. On the basis of these observations, which are also provided for in the protocol, it appears essential to consider blood volume and individual AUC as key criteria to be taken into account before patients are reassigned to the three cohorts in Part II.In the absence of a major adverse event, and in order to eliminate any risk of overexposure in the heaviest patients, even if the risk has not been confirmed, it is necessary to readjust the reassignment of patients in a safe manner. The experts on the steering committee therefore suggest the following allocation: - Cohort 10 mg/kg : 6 participants (including 4 of the heaviest) ; - Cohort 16 mg/kg: 3 participants; - Cohort 25 mg/kg: 3 participants. DSMB members approved this reassignment. This urgent safety measure will be endorsed by a substantial modification for authorization.FranceUSM 2US-764422025-03-182025-03-262025-03-26OTHERThe AVANCE1 phase 1/2a trial has temporarily paused injections as a precaution to fully assess recent SUSARs in three patients (one with potential treatment-related proteinuria, one with a Grade 4 Cerebral Venous Thrombosis, and one with Polycythemia). This decision, made with the Steering Committee on March 14, 2025, necessitates a thorough protocol review to refine treatment suspension/discontinuation criteria. While these events haven&#39;t changed the benefit/risk ratio, they were unexpected for the SQY51. Please refer to the attached Cover letter for a detailed overview. Attached are CIOMS forms for patients 01-015 and 01-008 and a SAE narrative for the patient 01-002 (CIOMS will be available by 26-MAR-2025). Data collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.Data collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed. A DSMB meeting was held on March 21st to discuss these events and potential measures, meeting minutes are ongoing. A letter was addressed to the Principal Investigator on the 19th March 2025 for this temporary halt (please see attached). All patients were informed of these injections temporary halt by the Principal Investigator. Encouragingly, early pharmacodynamic data shows reduced DMD-associated serum biomarkers, and the first completed patient&#39;s muscle biopsy revealed dystrophin restoration and stable ambulatory function. Ex-vivo studies support these findings, indicating SQY51&#39;s potential to normalize key muscle-related pathways. The observed polycythemia might even suggest a beneficial normalization of dystrophic musculature. Consequently, adjustments to SQY51 dosage, injection frequency, and treatment criteria are being considered, alongside intensified biomarker monitoring. An urgent meeting with the Agency is requested to discuss this data, protocol adjustments, and the continuation of treatment for favorable responders. Finally, an extension is requested for the substantial protocol amendment submission deadline (currently April 14thfollowing DSUR RFIs). A temporary halt is submitted in parallel of this notification.FranceCTIS bug2022-500703-49-014Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SQY51 in Paediatric and Adult Patients with a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy, including a i) 13-week Phase 1 Multiple Dose Escalation Phase, a ii) 49-week Phase 2a, a iii) 102-week extension phase, or a iv) 28-months long-term follow-up.AVANCE1-1/2a05/11/2025Duchenne muscular dystrophyFrance:421/11/2022FR: 21/11/2022Diseases [C] - Musculoskeletal Diseases [C05]Sqy TherapeuticsPharmaceutical companyPhase I and Phase II (Integrated)- First administration to humansPhase 2 : Motor function: - Motor Function Measure (MFM) score for ambulant and non-ambulant patients. - Performance Upper Limb (PUL) scale for ambulant and non-ambulant patients. - 6-minute walk test for ambulant patients. - 1-minute walk test (included in the 6-minute walk test) for ambulant patients. - 10-meter run/walk time for ambulant patients. - Rise from the floor for ambulant patients., Phase 2: Respiratory function: -Vital capacity, forced vital capacity, inspiratory capacity, and expiratory residual volume, functional residual capacity, residual volume, total lung capacity, all absolute values as well as percent predicted, as well as forced expiratory flow during 1 s, peak expiratory flow, peak cough flow, Tiffeneau index. -Maximal inspiratory and expiratory pressures, and sniff nasal inspiratory pressure all absolute values as well as % predicted. - Twitch mouth pressure, Phase 2 : Cardiac function : - echocardiography (LVEDD, LVESD, LVSWT, LVPWT, GLS, LVEFMS, LVMI, mitral inflow velocities, DT, E/é, LAVI, TAPSE, RV s', sPAP) - cardiac MRI (LVEDD, RVEDD, LVESD, RVESD, LVEF, RVEF, LGE, GCS, GRS, LVEDV, LVESV, systolic ejection volume, TAPSE, native T1, T2, ECV)., Phase 2 : Muscle strength: Measurements of pinch, maximal isometric grip strength using MyoGrip as well as strength of elbow flexion and extension, and knee flexion and extension using hand-held dynamometry., Phase 2 : Quality of Life Endpoints/Self-Assessment: Pediatric Quality of Life Inventory (PedsQL) – DMD module, Phase 2 : Serum biomarker analysis: Muscle necrosis biomarkers (CPK and Myomesine-3)., Phase 2 : Muscle biomarkers: - Levels of DMD exon-51 transcripts, dystrophin protein, histopathology of muscle biopsy. Lean cross-sectional areas (lCS), fat fraction (FF) and water T2 using quantitative MRI of forearm and lower leg (all patients)., Phase 1: Levels of SQY51, its main metabolites in serum and urine, and AUC calculation., Phase 2: Levels of SQY51 and main metabolites in serum, urine and muscle biopsy sample, PART III - EXTENSION PHASE : Primary and secondary objectives and endpoints of the Extension Phase are identical of the Phase 2a, with the exception that another muscle biopsy will not be performed.59618-64 years, 0-17 yearsMale112Type, frequency, severity, timing and relationship to SQY51 of: Adverse events (AEs), discontinuations due to AEs, serious AEs (SAEs)., PART III - EXTENSION PHASE : Primary and secondary objectives and endpoints of the Extension Phase are identical of the Phase 2a, with the exception that another muscle biopsy will not be performed.No27/02/202628/02/20262022-500703-49-01Authorised2025-11-052022-11-21T09:35:18.4452026-02-28T02:36:45.7629450834115954048101919661263SOLUTION FOR INJECTIONPRD96612631SOLUTION FOR INJECTIONSQY51SQY51SUB235142SQY THERAPEUTICS9661263249917SQY THERAPEUTICSNucleic AcidNucleic AcidSQY51SUB235142False1Falsepalmitoyl-conjugated tricyclo-DNA antisense oligonucleotidePRD9661263SQY5111INTRAVENOUS USEFalsesqy51SOLUTION FOR INJECTIONPhase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SQY51 in Paediatric and Adult Patients with a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy, including a i) 13-week Phase 1 Multiple Dose Escalation Phase, a ii) 49-week Phase 2a, a iii) 102-week extension phase, or a iv) 28-months long-term follow-up.949010212bb4115-18de-487c-81d0-eb66c85c2f4eÉtude de phase 1/2a, monocentrique, ouverte, visant à évaluer la sécurité, la pharmacocinétique et la pharmacodynamique du composé SQY51 chez des patients pédiatriques et adultes ayant reçu un diagnostic génétiquement confirmé de dystrophie musculaire de Duchenne, comprenant : i) une phase 1 de 13 semaines à doses multiples croissantes, ii) une phase 2a de 49 semaines, iii) une phase d'extension de 102 semaines, ou iv) un suivi à long terme de 28 mois.10FrenchPhase 1/2a study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SQY51 in Paediatric and Adult Patients with a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy9490103dc235a7e-faf7-42b5-8495-c46377380ce7Étude de phase 1/2a visant à évaluer la sécurité, la pharmacocinétique et la pharmacodynamique du composé SQY51 chez des patients pédiatriques et adultes ayant reçu un diagnostic génétiquement confirmé de dystrophie musculaire de Duchenne10FrenchAVANCE1-1/2aFalse71First in human study106152120476Duchenne muscular dystrophyTrue10000001254420.0PTDuchenne muscular dystrophy10013801100000004850False635095143509527350950Phase 1: Evaluate the safety and tolerability of SQY51 in DMD (Duchenne Muscular Dystrophy) patients. Phase 2a: Evaluate the safety and tolerance of SQY51 in DMD patients following 49-weeks multiple dosing. PART III - EXTENSION PHASE : Primary and secondary objectives and endpoints of the Extension Phase are identical of the Phase 2a, with the exception that another muscle biopsy will not be performed.4069961For phase 1 study: Characterize the PK profile of SQY51 and its main metabolites in serum and urine4069972For phase 1 study: Determine the effect of SQY51 on specific serum biomarkers4069983For phase 2a study: Determine the biodistribution of SQY51 and its main metabolites in serum and urine as well as in skeletal muscle of muscle biopsy at end of Part II4069994For phase 2a study: Determine the potential benefit of SQY51 on motor, respiratory and cardiac functions compared to baseline status as well as historical cohorts4070005For phase 2a study: Determine the effect of SQY51 on: - dystrophin rescue in muscle biopsy - muscle biomarkers compared to baseline status - quality of life compared to baseline status4070016PART III - EXTENSION PHASE : Primary and secondary objectives and endpoints of the Extension Phase are identical of the Phase 2a, with the exception that another muscle biopsy will not be performed.7785341Boys ≥6 years of age and ≥16 kg body weight.77853510Concomitant regular treatment with corticosteroids (Prednisone or Deflazacort on daily, alternative or intermittent dosing) for at least three months prior enrolment. Corticosteroid treatment is expected to remain stable during the study, however, can be dose/drug adapted according to weight change of patients.77853611If clinically indicated, approved concomitant treatment within standards of care guidelines for DMD, such as antihypertensive, vasodilators, lipid-lowering, thyroid replacement, vitamins, mineral substitution, gastric protectors, nutritional supplements. Patients on the therapies are eligible only if they are on a stable dose for at least one month prior to enrolment until completion of part 2. These treatments can be initiated or adapted as clinically indicated throughout the study.77853712Non-invasive mechanical nocturnal ventilation is permissive if <16 h/day.77853813For the extension phase : Patients must have completed Phase 2a of the study.7785392Being affiliated with a Social Security.7785403Informed consent form signed by the patient or, if minor, by the legal guardian(s).7785414For phase 2a study: Must have completed Phase 1 of the study.7785425Ambulatory or non-ambulatory status, described as Ambulatory stage: Able to rise from the floor and able to walk 10 m without assistance (inclusion of at least 4 patients). Early non-ambulatory stage: Unable to walk 10 m without assistance, including human assistance. Loss of ambulation (LoA) ≤ 5 years preceding enrolment (inclusion of maximal 8 patients). Late non-ambulatory stage: LoA > 5 years preceding enrolment (inclusion of maximal 3 patients).7785436Patients and, if minor, their legal guardians who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.7785447Diagnosed with DMD, genotypically confirmed with DMD mutations amenable to exon-51 skipping.7785458Stable hepatic and renal function: • GGT <1.5×ULN, AST <25×ULN, ALT <15×ULN • Total bilirubin <1.5×ULN. • Alkaline phosphatase ≤1.5×ULN. • Estimated glomerular filtration rate ≥90 mL/min/1.73m2. • Proteinuria and microalbuminuria ≤ULN.7785469Echocardiography left ventricular ejection fraction (LVEF) at screening ≥40%.13344611Patient with any serious medical/surgical or psychiatric condition/illness/history that in the opinion of the investigator would jeopardize patient’s safety or would interfere with the study assessments/results, including insufficient obligatory vaccination against infectious diseases as recommended by national guidelines, medical history of infection with Hepatitis B,C and HIV133446210Abnormal laboratory values in the clinically significant range13344632Patient with a history of significant allergic reactions and with any known allergies to products likely to be used in the study (e.g., antiseptics, anaesthetics), known hypersensitivity to any of the ingredients, or excipients of the study drug.13344643Patient who participated in other investigational study within the last three months, including those with investigational drugs that aim at restoring dystrophin expression such as other antisense oligomers.13344654Patient that received gene therapy.13344665Patient with intellectual disability or behavioral problem such that they cannot comply with the study procedure.13344676Patient with advanced cardiomyopathy and LVEF <40%. Patients with dysrhythmias and being treated for dysrhythmias. Patients with nontreated tachycardia.13344687Patient for whom major orthopedic surgery is planned during the phase 1 of the study (little invasive procedures such as tenotomy are permissible during phase 2, any procedure is permissible during the extension phase).13344698Tracheostomized patients and dependent on invasive mechanical ventilation. Non-invasive mechanical ventilation ≥ 16 h/day. Medical history with more than two respiratory decompensations requiring hospitalization during the previous year. No respiratory decompensation in the four months preceding enrolment.13344709Patients on medications that can restore dystrophin expression, tamoxifen and other drugs without indication for DMD or pediatric population.9120991Type, frequency, severity, timing and relationship to SQY51 of: Adverse events (AEs), discontinuations due to AEs, serious AEs (SAEs).True9121002PART III - EXTENSION PHASE : Primary and secondary objectives and endpoints of the Extension Phase are identical of the Phase 2a, with the exception that another muscle biopsy will not be performed.True9121011Phase 2 : Motor function: - Motor Function Measure (MFM) score for ambulant and non-ambulant patients. - Performance Upper Limb (PUL) scale for ambulant and non-ambulant patients. - 6-minute walk test for ambulant patients. - 1-minute walk test (included in the 6-minute walk test) for ambulant patients. - 10-meter run/walk time for ambulant patients. - Rise from the floor for ambulant patients.False9121022Phase 2: Respiratory function: -Vital capacity, forced vital capacity, inspiratory capacity, and expiratory residual volume, functional residual capacity, residual volume, total lung capacity, all absolute values as well as percent predicted, as well as forced expiratory flow during 1 s, peak expiratory flow, peak cough flow, Tiffeneau index. -Maximal inspiratory and expiratory pressures, and sniff nasal inspiratory pressure all absolute values as well as % predicted. - Twitch mouth pressureFalse9121033Phase 2 : Cardiac function : - echocardiography (LVEDD, LVESD, LVSWT, LVPWT, GLS, LVEFMS, LVMI, mitral inflow velocities, DT, E/é, LAVI, TAPSE, RV s', sPAP) - cardiac MRI (LVEDD, RVEDD, LVESD, RVESD, LVEF, RVEF, LGE, GCS, GRS, LVEDV, LVESV, systolic ejection volume, TAPSE, native T1, T2, ECV).False9121044Phase 2 : Muscle strength: Measurements of pinch, maximal isometric grip strength using MyoGrip as well as strength of elbow flexion and extension, and knee flexion and extension using hand-held dynamometry.False9121055Phase 2 : Quality of Life Endpoints/Self-Assessment: Pediatric Quality of Life Inventory (PedsQL) – DMD moduleFalse9121066Phase 2 : Serum biomarker analysis: Muscle necrosis biomarkers (CPK and Myomesine-3).False9121077Phase 2 : Muscle biomarkers: - Levels of DMD exon-51 transcripts, dystrophin protein, histopathology of muscle biopsy. Lean cross-sectional areas (lCS), fat fraction (FF) and water T2 using quantitative MRI of forearm and lower leg (all patients).False9121088Phase 1: Levels of SQY51, its main metabolites in serum and urine, and AUC calculation.False9121099Phase 2: Levels of SQY51 and main metabolites in serum, urine and muscle biopsy sampleFalse91211010PART III - EXTENSION PHASE : Primary and secondary objectives and endpoints of the Extension Phase are identical of the Phase 2a, with the exception that another muscle biopsy will not be performed.False2028-12-312022-11-0184500SQY Therapeutics31850733318508223185092525318511393931851026262PatientsFalseTrueTrue31683971Phase 1All included patients will receive SQY51 intravenously administered once a week in ascending doses (2, 4, 6, 10, 16 and 25 mg/kg).331683982Phase 2aPhase 2a includes eight blocks of 2-week treatment with the study drug, each block interspaced by a period of minimum 4 weeks without treatment. Patients will be allocated to one of three cohorts: The first cohort, consisting of 4 to maximal 6 patients, will receive a dose of 10 mg/kg/adm or the maximal dose <10 mg/kg that was determined safe during phase 1. In the second cohort, 3-4 patients will receive 16 mg/kg/adm. In the third cohort, 3-4 patients will be administered 25 mg/kg/adm. Cohorts will be constructed as homogenously as possibly with available patients (e.g., or dose ≤ to the dose inducing Cmax and AUC values corresponding to NHP at the NOAEL dose).331683993Part IIIAt the end of the phase 2a, patients will either enter an extension phase and continue to be dosed with SQY51, or being long-term followed at regular intervals33488921799918894EU Institution/Body/Agency14European Medicines AgencyFalseTrueORG-100013412Active18248Domenico Scarlattilaan 6Domenico Scarlattilaan 6Amsterdam1083 HS2029NetherlandsTrueLOC-100020264298892022-500703-49-00Sqy TherapeuticsPhase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SQY51 in Paediatric and Adult Patients with a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy, including a i) 13-week Phase 1 Multiple Dose Escalation Phase, and a ii) 32-week Phase 2aNON_APPLICABLENON_APPLICABLE717Falseacceptable5Diseases [C] - Musculoskeletal Diseases [C05]120476Duchenne muscular dystrophyTrue131090True386592PublicClinical Operations Director - Pharmaceuticalsmicheline.joly@sqy-synthena.com+3376520908518196Pharmaceutical company10Sqy TherapeuticsTrueTrueORG-100034038Active386593ScientificScientific advisorluis.garcia@sqy-synthena.com+3360729143218196Pharmaceutical company10Sqy TherapeuticsTrueTrueORG-100034038Active5849811904220061Pharmaceutical company10BiotrialTrueTrueORG-100006463Active193457 Rue Jean Louis Bertrand7 Rue Jean Louis BertrandRennes350002012France+33299599191iec_submission@biotrial.comTrueLOC-10001141891009219100931091009411910095129100965910097691009879100998+33299599191iec_submission@biotrial.com584982797497836703Laboratory/Research/Testing facility9Unite Paramedicale Ambulatoire De Recherche CliniqueTrueTrueORG-100050242Active88190010 B Rue Richard Lenoir10 B Rue Richard LenoirParis750112012France+33622210134francois.renard@uparc.frTrueORG-10005024291010015Nurses assigned to Raymond-Poincaré Hospital for the Avance1 clinical study Protocol-defined procedures Treatments administration at patients' homes during the extension phase Transport from the hospital pharmacy to patients' homes+33622210134francois.renard@uparc.fr18196Pharmaceutical company10Sqy TherapeuticsTrueTrueORG-100034038Active1732518196Pharmaceutical company10Sqy TherapeuticsTrueTrueORG-100034038Active1753950 Allee De Chaponval50 Allee De ChaponvalNoisy Le Roi785902012FranceTrueLOC-100053795FalseNon-Commercial1First in human study10696505Diseases [C] - Musculoskeletal Diseases [C05]360068null1Test48101919661263SOLUTION FOR INJECTIONPRD96612631SOLUTION FOR INJECTIONSQY51SQY51SUB235142SQY THERAPEUTICS9661263249917SQY THERAPEUTICSNucleic AcidNucleic AcidSQY51SUB235142False1Falsepalmitoyl-conjugated tricyclo-DNA antisense oligonucleotidePRD9661263SQY5111INTRAVENOUS USEFalsesqy51SOLUTION FOR INJECTIONFalse2758361902190216412012Authorised2022-09-212022-09-21TrueFrance2022-11-21Authorised6042023-01-052023-02-066052023-01-052023-02-06251442024-07-042024-07-05251452024-07-042024-07-05681902025-11-052025-11-06681912025-11-052025-11-0611422023-04-262023-04-2711432023-04-262023-04-27155312023-04-262024-03-152024-03-15155322023-04-262024-03-152024-03-15435332024-09-092024-09-09435342024-09-092024-09-09587822024-09-092024-11-29708282024-09-092025-03-142025-03-18708292024-09-092025-03-142025-03-18717562024-09-092025-03-142025-03-26717572024-09-092025-03-26True6652023-01-052023-02-062023-04-26False7461902Under evaluation2022-08-12T15:52:44.61219381902Authorised2022-11-21T09:35:18.08270311902Halted2024-03-15T17:58:24.909419811902Authorised2024-07-05T09:49:43.039787351902Halted2025-03-18T14:41:50.023792271902Authorised2025-03-25T16:31:13.607792661902Halted2025-03-26T09:37:33.4991221981902Authorised2025-11-06T10:49:38.9911France2025-11-052024-09-09acceptable2026-02-27authorized2022-11-212022-11-211218920621733118202Hospital/Clinic/Other health care facility8Assistance Publique Hopitaux De ParisFalseTrueORG-100004082Active17545104 Boulevard Raymond Poincare104 Boulevard Raymond PoincareGarches923802012France0147107890helge.amthor@uvsq.frTrueLOC-1000693322102351HelgeAmthor0147107890helge.amthor@uvsq.fr2Centre de référence des maladies neuromusculairesAuthorised1661INITIALAuthorised2022-500703-49-01Authorised2022-08-12acceptable2022-11-15409019021902France2012acceptable2022-09-22authorized2022-11-21AuthorisedFranceAuthorised2022-11-21Authorised2022-11-211902FranceAuthorisedIN-1False47916611902France2022-11-21T09:35:18.445authorizedacceptabledecision40903609INITIALTrue4696SUBSTANTIAL MODIFICATIONUnder evaluation2022-500703-49-01Authorised2023-02-15acceptable2023-03-311137219021902France2012acceptable2023-04-06authorized2022-11-21AuthorisedFranceAuthorised2022-11-21Under evaluation2023-04-111902FranceAuthorisedSM-1PART_I_AND_PART_IIFalse171846961902France2023-04-11T00:00:00authorizedacceptabledecision113726164SUBSTANTIAL MODIFICATIONTrue16464SUBSTANTIAL MODIFICATIONAuthorised2022-500703-49-01Authorised2023-12-06acceptable2024-03-185830019021902France2012no_conclusion2024-02-12authorized2022-11-21AuthorisedFranceAuthorised2022-11-21Authorised2024-03-251902FranceAuthorisedSM-2PART_I_AND_PART_IIFalse16809164641902France2024-03-25T18:09:21.868authorizedno_conclusiondecision5830028839SUBSTANTIAL MODIFICATIONTrue26679SUBSTANTIAL MODIFICATIONAuthorised2022-500703-49-01Authorised2024-05-22acceptable2024-07-0410124719021902France2012acceptable2024-07-03authorized2022-11-21AuthorisedFranceAuthorised2022-11-21Authorised2024-07-041902FranceAuthorisedSM-3PART_I_AND_PART_IIFalse27834266791902France2024-07-04T16:59:33.259authorizedacceptabledecision10124742263SUBSTANTIAL MODIFICATIONTrue38987SUBSTANTIAL MODIFICATIONAuthorised2022-500703-49-01Authorised2024-10-22acceptable2024-11-152024-11-151902FranceAuthorisedSM-4PART_IFalse46891389871902France2024-11-15T12:47:54.616authorizeddecision60055SUBSTANTIAL MODIFICATIONTrue43453SUBSTANTIAL 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MODIFICATIONAuthorised2022-500703-49-01Authorised2026-01-28acceptable27583619021902France2012acceptable2026-02-27authorized2022-11-21AuthorisedFranceAuthorised2022-11-21Authorised2026-02-271902FranceAuthorisedSM-8PART_IIFalse112000755371902France2026-02-27T15:06:30.845authorizedacceptabledecision275836115954SUBSTANTIAL MODIFICATIONTrueFrance19022022-11-21T09:35:18.4452026-02-27T15:06:30.8454False1902TH-173412024-03-156Medicinal Product relatedFalseFalseTrueFollowing an analysis of the batch records (carried out on 12/03/2024) of the finished product SQY51 used in the AVANCE 1 phase 1 clinical study (1st patient included in the study on 03/05/2023) and stability data, SQY Therapeutics discovered a non-compliance regarding the expiry dates of batch DPSQYM001 (packaged in 3 sub-batches). Below is the information recorded in the batch certificates - investigational medicinal product for human use issued by COLCA /EUROMED PHARMA: - Lot DPSQYM001A expiry date 06/2023, batch certificate signed by QP on 20/12/2022 - Lot DPSQYM001B expiry date 05/2024, batch certificate signed by QP on 14/09/2023 - Lot DPSQYM001C expiry date 05/2024, batch certificate signed by QP on 14/09/2023 SQY51 Finished Product Information: - Absence of a technical batch for SQY51 performed prior to the start of the clinical study - 1st batch of SQY 51 DPSQYM001 manufactured at GTP Bioways on 24/11/2021 then packaged and certified for the clinical study by EUROMED PHARMA (ex COLCA) on 20/12/2022 (batch DPSQYM001A), expiry date of certificate 06/2023 - Stabilization of the DPSQYM001 batch at AXOLABS in July 2022 according to the following scheme (Q0 &#34;analytical&#34; &#61; 27/07/2022): o Normal conditions / Storage in stability chamber at -20°C &#43;/- 5°C / T0 &#43; T3M &#43; T6M &#43; T9M &#43; T12M &#43; T18M &#43; T24M o Accelerated conditions / Storage in stability chamber at &#43;5°C &#43;/- 3°C / T0 &#43; T2M &#43; T4M &#43; T6M &#43; T9M &#43; T12M &#43; T18M &#43; T24M o Stress test / Storage in stability chamber at &#43;40°C &#43;/- 2°C / T0 &#43; T1M - Stability results known to date: stress test &#43;40°C &#43;/- 2°C up to T1M, accelerated conditions at &#43;5°C &#43;/- 3°C up to T12M, and normal conditions at -20°C &#43;/- 5°C up to T12M. To date, no OOS has been detected at any of the check-ins. - Extrapolation of stability data is not applicable for SQY51 because the product is stored at -20°C (in accordance with ICH Q1E – Evaluation of stability data) In fact, you will find below the expiry dates that should have been entered on the different dates of issue of the certificates of the batch DPSQYM001 in view of the state of knowledge (stability results and exclusion of extrapolation): - Expiry date 11/2022, on the date of signature of the batch certificate on 20/12/2022 - Expiry Date 07/2023, on the date of signature of the batch certificate on 14/09/2023 Please also note that this non-compliance cannot be considered a public health concern as no serious adverse reactions were reported in the Phase 1 study. Following the observation of this non-compliance, SQY Therapeutics immediately requested that the DPSQYM001 batch be quarantined on 13/03/2024 within the pharmacy of the investigator site. The last administration of this batch took place at the investigator site on 11/03/2024 as part of this AVANCE 1 - phase 1 study. This Phase 1 study has not been yet finalized. Upcoming visits are described in Appendix 1 of the attached letter. Currently, there is another batch of SQY51 finished product, never administered to humans, batch DPSQYM002 manufactured on 27/09/2022 with an expiry date of 05/2024 (batch certificate signed on 23/10/2023) : expiry date in line with the stability data known to date. The 18-month stability data is expected to be confirmed by 22/03/2024. Part of this batch has been sent to the investigator site, but no administration will take place without the authorization of the ANSM. SQY Therapeutics is aware of the seriousness of this non-compliance and inform you that a deviation has been put in place in order to implement emergency actions, a deviation involving the CRO BIOTRIAL, the investigator site and EUROMED PHARMA, all of whom have been informed of this notification.False2024-03-15T17:58:24This same notification will be the subject of an email communication. The actions SQY Therapeutics intends to implement are as follows: - Suspension of the administration of SQY51 in the context of the AVANCE 1 – phase 1 clinical study pending feedback from the ANSM for the continuation of the study with the administration of batch DPSQYM002. 18-month stability data for batch DPSQYM002 is expected to be available by 22/03/2024. - Continuation of analyses, recruitment and follow-up visits of the AVANCE 1 – Phase 1 clinical study, as mentioned in Appendix 1. - Emergency analysis of the remaining vials of the batch DPSQYM001 - Communication with patients after approval by the ANSM - Communication with the DSMB/steering committee after approval by the ANSM - Communication with the hospital of the investigator site after approval by the ANSMFalse2024-03-151902FranceFR1902TH-764482025-03-144Safety related (clinical or pre-clinical results)FalseFalseTrueThe AVANCE1 phase 1/2a trial has temporarily paused injections as a precaution to fully assess recent SUSARs in three patients (one with potential treatment-related proteinuria, one with a Grade 4 Cerebral Venous Thrombosis, and one with Polycythemia). This decision, made with the Steering Committee on March 14, 2025, necessitates a thorough protocol review to refine treatment suspension/discontinuation criteria. While these events haven&#39;t changed the benefit/risk ratio, they were unexpected for the SQY51. Please refer to the attached Cover letter for a detailed overview. Attached are CIOMS forms for patients 01-015 and 01-008 and 01-002. Data collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.False2025-03-26T09:37:33A DSMB meeting was held on March 21st to discuss these events and potential measures, meeting minutes are ongoing. A letter was addressed to the Principal Investigator on the 19th March 2025 for this temporary halt (please see attached). All patients were informed of these injections temporary halt by the Principal Investigator. Encouragingly, early pharmacodynamic data shows reduced DMD-associated serum biomarkers, and the first completed patient&#39;s muscle biopsy revealed dystrophin restoration and stable ambulatory function. Ex-vivo studies support these findings, indicating SQY51&#39;s potential to normalize key muscle-related pathways. The observed polycythemia might even suggest a beneficial normalization of dystrophic musculature. Consequently, adjustments to SQY51 dosage, injection frequency, and treatment criteria are being considered, alongside intensified biomarker monitoring. An urgent meeting with the Agency is requested to discuss this data, protocol adjustments, and the continuation of treatment for favorable responders. Finally, an extension is requested for the substantial protocol amendment submission deadline (currently April 14thfollowing DSUR RFIs). A USM is submitted in parallel of this notificationFalse1902FranceFR1902FranceRESTART_OF_TRIAL2025-11-05START_OF_TRIAL2023-01-05RESTART_OF_RECRUITMENT2024-09-09START_OF_RECRUITMENT2023-04-26TEMPORARY_HALT2025-03-14USM #1US-517832024-10-042024-10-152024-10-15OTHERAs indicated in e-mails sent to ANSM on 07/10/2024 and 14/10/2024, analysis of the areas under the curve (AUC) of patients who completed Part I, suggests the possibility of overexposure to the investigational medicinal product, SQY51, in the heaviest patients, due to the method used to calculate the dose administered: mg/kg. As such, this is a potential risk of overexposure to the investigational medicinal product detected on 30/09/2024. On the same day, members of the Data and Safety Monitoring Board (DSMB) and experts from the Comité de Pilotage, including a methodologist and a pharmacokineticist, met to discuss the reassignment of patients to the cohorts, based on analyses of the pharmacokinetic (PK) data evaluated in Part I of the study. On the basis of these observations, which are also provided for in the protocol, it appears essential to consider blood volume and individual AUC as key criteria to be taken into account before patients are reassigned to the three cohorts in Part II.In the absence of a major adverse event, and in order to eliminate any risk of overexposure in the heaviest patients, even if the risk has not been confirmed, it is necessary to readjust the reassignment of patients in a safe manner. The experts on the steering committee therefore suggest the following allocation: - Cohort 10 mg/kg : 6 participants (including 4 of the heaviest) ; - Cohort 16 mg/kg: 3 participants; - Cohort 25 mg/kg: 3 participants. DSMB members approved this reassignment. This urgent safety measure will be endorsed by a substantial modification for authorization.FranceUSM 2US-764422025-03-182025-03-262025-03-26OTHERThe AVANCE1 phase 1/2a trial has temporarily paused injections as a precaution to fully assess recent SUSARs in three patients (one with potential treatment-related proteinuria, one with a Grade 4 Cerebral Venous Thrombosis, and one with Polycythemia). This decision, made with the Steering Committee on March 14, 2025, necessitates a thorough protocol review to refine treatment suspension/discontinuation criteria. While these events haven&#39;t changed the benefit/risk ratio, they were unexpected for the SQY51. Please refer to the attached Cover letter for a detailed overview. Attached are CIOMS forms for patients 01-015 and 01-008 and a SAE narrative for the patient 01-002 (CIOMS will be available by 26-MAR-2025). Data collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.Data collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed. A DSMB meeting was held on March 21st to discuss these events and potential measures, meeting minutes are ongoing. A letter was addressed to the Principal Investigator on the 19th March 2025 for this temporary halt (please see attached). All patients were informed of these injections temporary halt by the Principal Investigator. Encouragingly, early pharmacodynamic data shows reduced DMD-associated serum biomarkers, and the first completed patient&#39;s muscle biopsy revealed dystrophin restoration and stable ambulatory function. Ex-vivo studies support these findings, indicating SQY51&#39;s potential to normalize key muscle-related pathways. The observed polycythemia might even suggest a beneficial normalization of dystrophic musculature. Consequently, adjustments to SQY51 dosage, injection frequency, and treatment criteria are being considered, alongside intensified biomarker monitoring. An urgent meeting with the Agency is requested to discuss this data, protocol adjustments, and the continuation of treatment for favorable responders. Finally, an extension is requested for the substantial protocol amendment submission deadline (currently April 14thfollowing DSUR RFIs). A temporary halt is submitted in parallel of this notification.FranceCTIS bugEEA1