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(DMD)","language":7,"languageDescription":"Spanish"},{"id":9737328,"uuid":"20e3247c-35c5-41fe-a920-e865a37be002","attributeTranslation":"Une 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 DMD","language":33,"languageDescription":"French (Belgium)"},{"id":9737325,"uuid":"20e3247c-35c5-41fe-a920-e865a37be002","attributeTranslation":"Badanie fazy 2a, randomizowane, podwójnie zaślepione, kontrolowane placebo, porównujące dawki oraz eksploracyjne badanie skuteczności doustnie podawanego preparatu SAT-3247 u ambulatoryjnych pacjentów z DMD","language":19,"languageDescription":"Polish"},{"id":9737324,"uuid":"20e3247c-35c5-41fe-a920-e865a37be002","attributeTranslation":"Een fase 2a, gerandomiseerd, dubbelblind, placebogecontroleerd dosisvergelijkend en verkennend onderzoek naar de werkzaamheid van oraal toegediend SAT-3247 bij ambulante DMD-patiënten","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737326,"uuid":"20e3247c-35c5-41fe-a920-e865a37be002","attributeTranslation":"Eine randomisierte, doppelblinde, placebokontrollierte Dosisvergleichs- und explorative Wirksamkeitsstudie der Phase 2a mit oral verabreichtem SAT-3247 bei gehfähigen DMD-Patienten","language":28,"languageDescription":"German (Belgium)"}],"publicTitle":"A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients","publicTitleTranslations":[{"id":9737403,"uuid":"7b371a84-6bef-4362-be76-5b56f65b2e37","attributeTranslation":"Eine randomisierte, doppelblinde, placebokontrollierte Dosisvergleichs- und explorative Wirksamkeitsstudie der Phase 2a mit oral verabreichtem SAT-3247 bei gehfähigen DMD-Patienten","language":28,"languageDescription":"German (Belgium)"},{"id":9737402,"uuid":"7b371a84-6bef-4362-be76-5b56f65b2e37","attributeTranslation":"Une 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 DMD","language":33,"languageDescription":"French (Belgium)"},{"id":9737401,"uuid":"7b371a84-6bef-4362-be76-5b56f65b2e37","attributeTranslation":"Badanie fazy 2a, randomizowane, podwójnie zaślepione, kontrolowane placebo, porównujące dawki oraz eksploracyjne badanie skuteczności doustnie podawanego preparatu SAT-3247 u ambulatoryjnych pacjentów z DMD","language":19,"languageDescription":"Polish"},{"id":9737400,"uuid":"7b371a84-6bef-4362-be76-5b56f65b2e37","attributeTranslation":"Een fase 2a, gerandomiseerd, dubbelblind, placebogecontroleerd dosisvergelijkend en verkennend onderzoek naar de werkzaamheid van oraal toegediend SAT-3247 bij ambulante DMD-patiënten","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737399,"uuid":"7b371a84-6bef-4362-be76-5b56f65b2e37","attributeTranslation":"Estudio 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)","language":7,"languageDescription":"Spanish"}],"shortTitle":"SAT-3247-CL-201","secondaryIdentifyingNumbers":{"additionalRegistries":[]}},"trialInformation":{"trialCategory":{"isLowIntervention":false,"trialPhase":"4","trialCategory":"2","justificationForTrialCategory":"This is a phase 2a trial","trialCategoryId":108624},"medicalCondition":{"partIMedicalConditions":[{"id":123323,"medicalCondition":"Duchenne muscular dystrophy","medicalConditionTranslations":[],"isConditionRareDisease":true}]},"trialObjective":{"trialScopes":[{"code":"4","trialScopeId":358769},{"code":"6","trialScopeId":358772},{"code":"9","trialScopeId":358771},{"code":"5","trialScopeId":358770}],"mainObjective":"Safety: The primary safety objective is to evaluate the safety and tolerability of SAT-3247 in ambulatory DMD patients.\nEfficacy: The primary efficacy objective is to determine SAT-3247 effects on muscle force as determined by dynamometry at 12 weeks","mainObjectiveTranslations":[{"id":9737396,"uuid":"4722e808-4398-47d7-bdeb-a16764253ec7","attributeTranslation":"Bezpieczeństwo: Pierwszorzędowym celem w zakresie bezpieczeństwa jest ocena bezpieczeństwa i tolerancji preparatu SAT-3247 u ambulatoryjnych pacjentów z DMD\nSkuteczność: Pierwszorzędowym celem skuteczności jest ocena wpływu preparatu SAT-3247 na siłę mięśni mierzoną za pomocą dynamometrii po 12 tygodniach.","language":19,"languageDescription":"Polish"},{"id":9737395,"uuid":"4722e808-4398-47d7-bdeb-a16764253ec7","attributeTranslation":"Seguridad: El objetivo de seguridad principal es evaluar la seguridad y la tolerabilidad de SAT-3247 en pacientes \nambulatorios con DMD\nEficacia: El objetivo de eficacia principal es determinar los efectos de SAT-3247 sobre la fuerza muscular \ndeterminada por dinamometría al cabo de 12 semanas","language":7,"languageDescription":"Spanish"},{"id":9737394,"uuid":"4722e808-4398-47d7-bdeb-a16764253ec7","attributeTranslation":"Innocuité: L’objectif principal de sécurité est d’évaluer l’innocuité et la tolérabilité du SAT-3247 chez les patients \nambulants atteints de DMD\nEfficacité:  L’objectif principal d’efficacité est de déterminer les effets du SAT-3247 sur la force musculaire déterminée par dynamométrie à 12 semaines.","language":33,"languageDescription":"French (Belgium)"},{"id":9737398,"uuid":"4722e808-4398-47d7-bdeb-a16764253ec7","attributeTranslation":"Veiligheid: Het primaire veiligheidsdoel is het evalueren van de veiligheid en verdraagbaarheid van SAT-3247 bij \nambulante DMD-patiënten\nWerkzaamheid: Het primaire werkzaamheidsdoel is het vaststellen van de effecten van SAT-3247 op de spierkracht, zoals bepaald met dynamometrie na 12 weken","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737397,"uuid":"4722e808-4398-47d7-bdeb-a16764253ec7","attributeTranslation":"Sicherheit: Das primäre Sicherheitsziel ist die Bewertung der Sicherheit und Verträglichkeit von SAT-3247 bei \ngehfähigen DMD-Patienten\nWirksamkeit: Das primäre Wirksamkeitsziel ist die Bestimmung der Auswirkungen von SAT-3247 auf die Muskelkraft, \ndie durch Dynamometrie nach 12 Wochen bestimmt wird","language":28,"languageDescription":"German (Belgium)"}],"secondaryObjectives":[{"id":416715,"number":1,"secondaryObjective":"To determine SAT-3247 effects on intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in vastus lateralis at 12 weeks.","secondaryObjectiveTranslations":[{"id":9737405,"uuid":"b687125d-e5f9-4b68-9b1a-f9d1c10d0e0a","attributeTranslation":"Bestimmung der Auswirkungen von SAT-3247 auf den intramuskulären Fettanteil in der quantitativen Magnetresonanz (qMR) im Vastus lateralis nach 12 Wochen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737404,"uuid":"b687125d-e5f9-4b68-9b1a-f9d1c10d0e0a","attributeTranslation":"Déterminer les effets du SAT-3247 sur la fraction de graisse intramusculaire dans la résonance magnétique quantitative (IRMq) du muscle vaste latéral à 12 semaines.","language":33,"languageDescription":"French (Belgium)"},{"id":9737408,"uuid":"b687125d-e5f9-4b68-9b1a-f9d1c10d0e0a","attributeTranslation":"Determinar 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.","language":7,"languageDescription":"Spanish"},{"id":9737407,"uuid":"b687125d-e5f9-4b68-9b1a-f9d1c10d0e0a","attributeTranslation":"Het 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 weken","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737406,"uuid":"b687125d-e5f9-4b68-9b1a-f9d1c10d0e0a","attributeTranslation":"Określenie wpływu SAT-3247 na frakcję tłuszczu śródmięśniowego w ilościowym rezonansie \nmagnetycznym (qMR) mięśnia obszernego bocznego po 12 tygodniach.","language":19,"languageDescription":"Polish"}]},{"id":416716,"number":2,"secondaryObjective":"To determine SAT-3247 effects on proton muscle transverse relaxation time (T2) in vastus lateralis at 12 weeks","secondaryObjectiveTranslations":[{"id":9737409,"uuid":"3af14c0c-bb41-4a2a-8a06-4b94a35e3a7c","attributeTranslation":"Het bepalen van de effecten van SAT-3247 op de transversale relaxatietijd van protonen in spierweefsel (T2) van de vastus lateralis na 12 weken.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737411,"uuid":"3af14c0c-bb41-4a2a-8a06-4b94a35e3a7c","attributeTranslation":"Déterminer les effets du SAT-3247 sur le temps de relaxation transversale du muscle protonique (T2) dans le muscle vaste latéral à 12 semaines.","language":33,"languageDescription":"French (Belgium)"},{"id":9737413,"uuid":"3af14c0c-bb41-4a2a-8a06-4b94a35e3a7c","attributeTranslation":"Określenie wpływu SAT-3247 na czas relaksacji poprzecznej protonów (T2) w mięśniu obszernym bocznym po 12 tygodniach.","language":19,"languageDescription":"Polish"},{"id":9737412,"uuid":"3af14c0c-bb41-4a2a-8a06-4b94a35e3a7c","attributeTranslation":"Bestimmung der Auswirkungen von SAT-3247 auf die transversale Relaxationszeit (T2) der Muskelprotonen im Vastus lateralis nach 12 Wochen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737410,"uuid":"3af14c0c-bb41-4a2a-8a06-4b94a35e3a7c","attributeTranslation":"Determinar 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.","language":7,"languageDescription":"Spanish"}]},{"id":416717,"number":3,"secondaryObjective":"To determine SAT-3247 effects on the Regeneration Index in open muscle biopsy of the biceps brachii at 12 weeks","secondaryObjectiveTranslations":[{"id":9737415,"uuid":"b5508636-ac46-4f0a-b307-50b3b88b5492","attributeTranslation":"Bestimmung der Auswirkungen von SAT-3247 auf den Regenerationsindex bei offener Muskelbiopsie des Musculus biceps brachii nach 12 Wochen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737416,"uuid":"b5508636-ac46-4f0a-b307-50b3b88b5492","attributeTranslation":"Określenie wpływu SAT-3247 na wskaźnik regeneracji w otwartej biopsji mięśnia dwugłowego ramienia po 12 tygodniach.","language":19,"languageDescription":"Polish"},{"id":9737414,"uuid":"b5508636-ac46-4f0a-b307-50b3b88b5492","attributeTranslation":"Het bepalen van de effecten van SAT-3247 op de regeneratie-index in een open spierbiopt van de biceps brachii na 12 weken.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737417,"uuid":"b5508636-ac46-4f0a-b307-50b3b88b5492","attributeTranslation":"Déterminer les effets du SAT-3247 sur l’indice de régénération dans une biopsie musculaire ouverte du biceps brachii à 12 semaines.","language":33,"languageDescription":"French (Belgium)"},{"id":9737418,"uuid":"b5508636-ac46-4f0a-b307-50b3b88b5492","attributeTranslation":"Determinar los efectos de SAT-3247 sobre el índice de regeneración en biopsia muscular abierta del bíceps braquial al cabo de 12 semanas.","language":7,"languageDescription":"Spanish"}]},{"id":416718,"number":4,"secondaryObjective":"To determine population PK of SAT-3247 in ambulatory patients.","secondaryObjectiveTranslations":[{"id":9737423,"uuid":"88926545-43ed-4cae-b8f0-8532ab772ea8","attributeTranslation":"Déterminer la pharmacocinétique de population du SAT-3247 chez les patients ambulants.","language":33,"languageDescription":"French (Belgium)"},{"id":9737419,"uuid":"88926545-43ed-4cae-b8f0-8532ab772ea8","attributeTranslation":"Określenie farmakokinetyki (PK) SAT-3247 w populacji pacjentów ambulatoryjnych.","language":19,"languageDescription":"Polish"},{"id":9737420,"uuid":"88926545-43ed-4cae-b8f0-8532ab772ea8","attributeTranslation":"Bestimmung der Populations-PK von SAT-3247 bei gehfähigen Patienten.","language":28,"languageDescription":"German (Belgium)"},{"id":9737422,"uuid":"88926545-43ed-4cae-b8f0-8532ab772ea8","attributeTranslation":"Determinar la farmacocinética (PK) poblacional de SAT-3247 en pacientes ambulatorios.","language":7,"languageDescription":"Spanish"},{"id":9737421,"uuid":"88926545-43ed-4cae-b8f0-8532ab772ea8","attributeTranslation":"Het bepalen van de populatie-farmacokinetiek van SAT-3247 bij ambulante patiënten.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":416719,"number":5,"secondaryObjective":"To determine the potential for improvement in muscle function with treatment of SAT-3247 in ambulatory patients","secondaryObjectiveTranslations":[{"id":9737427,"uuid":"2c5989a3-b91b-4a91-a969-b337e9b6b839","attributeTranslation":"Bestimmung des Potenzials für eine Verbesserung der Muskelfunktion durch die Behandlung mit SAT-3247 bei gehfähigen Patienten.","language":28,"languageDescription":"German (Belgium)"},{"id":9737428,"uuid":"2c5989a3-b91b-4a91-a969-b337e9b6b839","attributeTranslation":"Déterminer le potentiel d’amélioration de la fonction musculaire avec le traitement au SAT-3247 chez les patients ambulants.","language":33,"languageDescription":"French (Belgium)"},{"id":9737424,"uuid":"2c5989a3-b91b-4a91-a969-b337e9b6b839","attributeTranslation":"Określenie potencjału poprawy funkcji mięśni w trakcie leczenia SAT-3247 u pacjentów ambulatoryjnych.","language":19,"languageDescription":"Polish"},{"id":9737426,"uuid":"2c5989a3-b91b-4a91-a969-b337e9b6b839","attributeTranslation":"Determinar el potencial de mejora de la función muscular con el tratamiento de SAT-3247 en pacientes ambulatorios.","language":7,"languageDescription":"Spanish"},{"id":9737425,"uuid":"2c5989a3-b91b-4a91-a969-b337e9b6b839","attributeTranslation":"Het bepalen van het potentieel voor verbetering van de spierfunctie door middel van behandeling met SAT-3247 bij ambulante patiënten.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":416720,"number":6,"secondaryObjective":"Exploratory objective: to determine SAT-3247 effects on respiratory function at 12 weeks","secondaryObjectiveTranslations":[{"id":9737431,"uuid":"16826873-e843-4490-98e2-a070b57ec9b5","attributeTranslation":"Cel eksploracyjny: Określenie wpływu SAT-3247 na czynność układu oddechowego po 12 tygodniach.","language":19,"languageDescription":"Polish"},{"id":9737432,"uuid":"16826873-e843-4490-98e2-a070b57ec9b5","attributeTranslation":"Objetivo exploratorio: Determinar los efectos de SAT-3247 sobre la función respiratoria al cabo de 12 semanas.","language":7,"languageDescription":"Spanish"},{"id":9737430,"uuid":"16826873-e843-4490-98e2-a070b57ec9b5","attributeTranslation":"Verkennende doestelling: Het bepalen van de effecten van SAT-3247 op de ademhalingsfunctie na 12 weken.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737433,"uuid":"16826873-e843-4490-98e2-a070b57ec9b5","attributeTranslation":"Exploratives Ziel: Bestimmung der Auswirkungen von SAT-3247 auf die Atmungsfunktion nach 12 Wochen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737429,"uuid":"16826873-e843-4490-98e2-a070b57ec9b5","attributeTranslation":"Objectif exploratoire: Déterminer les effets du SAT-3247 sur la fonction respiratoire à 12 semaines.","language":33,"languageDescription":"French (Belgium)"}]},{"id":416721,"number":7,"secondaryObjective":"Exploratory objective: to determine SAT-3247 effects on muscle histopathology of the biceps brachii at 12 weeks","secondaryObjectiveTranslations":[{"id":9737437,"uuid":"f856ccc4-8573-45d9-b531-c90a0accdc99","attributeTranslation":"Exploratives Ziel: Bestimmung der Auswirkungen von SAT-3247 auf die Muskelhistopathologie des Musculus biceps brachii nach 12 Wochen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737435,"uuid":"f856ccc4-8573-45d9-b531-c90a0accdc99","attributeTranslation":"Verkennende doestelling: Het bepalen van de effecten van SAT-3247 op de spierhistopathologie van de biceps brachii na 12 weken.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737436,"uuid":"f856ccc4-8573-45d9-b531-c90a0accdc99","attributeTranslation":"Cel eksploracyjny: Określenie wpływu SAT-3247 na obraz histopatologiczny mięśnia dwugłowego ramienia po 12 tygodniach.","language":19,"languageDescription":"Polish"},{"id":9737434,"uuid":"f856ccc4-8573-45d9-b531-c90a0accdc99","attributeTranslation":"Objetivo exploratorio: Determinar los efectos de SAT-3247 sobre la histopatología muscular del bíceps braquial al cabo de 12 semanas.","language":7,"languageDescription":"Spanish"},{"id":9737438,"uuid":"f856ccc4-8573-45d9-b531-c90a0accdc99","attributeTranslation":"Objectif exploratoire: Déterminer les effets du SAT-3247 sur l’histopathologie musculaire du biceps brachial à 12 semaines.","language":33,"languageDescription":"French (Belgium)"}]},{"id":416722,"number":8,"secondaryObjective":"Exploratory objective: to compare changes in outcomes to natural history","secondaryObjectiveTranslations":[{"id":9737440,"uuid":"17c05869-6ce9-4a51-a96e-d4f4e5a12943","attributeTranslation":"Objectif exploratoire: Comparer l’évolution des résultats à l’histoire naturelle.","language":33,"languageDescription":"French (Belgium)"},{"id":9737439,"uuid":"17c05869-6ce9-4a51-a96e-d4f4e5a12943","attributeTranslation":"Cel eksploracyjny: Porównanie uzyskanych zmian z naturalnym przebiegiem choroby.","language":19,"languageDescription":"Polish"},{"id":9737443,"uuid":"17c05869-6ce9-4a51-a96e-d4f4e5a12943","attributeTranslation":"Verkennende doestelling: Het vergelijken van veranderingen in resultaten met die bij het natuurlijke beloop van de ziekte.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737442,"uuid":"17c05869-6ce9-4a51-a96e-d4f4e5a12943","attributeTranslation":"Objetivo exploratorio: Comparar los cambios en los resultados con la evolución natural de la enfermedad.","language":7,"languageDescription":"Spanish"},{"id":9737441,"uuid":"17c05869-6ce9-4a51-a96e-d4f4e5a12943","attributeTranslation":"Exploratives Ziel: Vergleich der Veränderungen bei den Ergebnissen mit dem natürlichen Verlauf.","language":28,"languageDescription":"German (Belgium)"}]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":797253,"number":1,"principalInclusionCriteria":"Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene","principalInclusionCriteriaTranslations":[{"id":9737330,"uuid":"b26cc646-e636-4673-b194-6425c4081f63","attributeTranslation":"1. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737333,"uuid":"b26cc646-e636-4673-b194-6425c4081f63","attributeTranslation":"1. Definitieve diagnose van DMD op basis van gedocumenteerde klinische bevindingen en eerdere genetische tests met een bevestigde mutatie in het DMD-gen.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737331,"uuid":"b26cc646-e636-4673-b194-6425c4081f63","attributeTranslation":"1. Definitive Diagnose von DMD aufgrund dokumentierter klinischer Befunde und vorheriger Gentests mit bestätigter Mutation im DMD-Gen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737332,"uuid":"b26cc646-e636-4673-b194-6425c4081f63","attributeTranslation":"1. 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.","language":7,"languageDescription":"Spanish"},{"id":9737329,"uuid":"b26cc646-e636-4673-b194-6425c4081f63","attributeTranslation":"1. Potwierdzona diagnoza dystrofii mięśniowej Duchenne’a (DMD) na podstawie udokumentowanych wyników badań klinicznych oraz wcześniejszych badań genetycznych, z wykrytą mutacją w genie DMD.","language":19,"languageDescription":"Polish"}]},{"id":797254,"number":10,"principalInclusionCriteria":"If 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.","principalInclusionCriteriaTranslations":[{"id":9737335,"uuid":"192a96ba-455c-43cf-8066-0a553295a22d","attributeTranslation":"10. 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.","language":7,"languageDescription":"Spanish"},{"id":9737336,"uuid":"192a96ba-455c-43cf-8066-0a553295a22d","attributeTranslation":"10. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737337,"uuid":"192a96ba-455c-43cf-8066-0a553295a22d","attributeTranslation":"10. Wenn der Teilnehmer pflanzliche Heilmittel und Nahrungsergänzungsmittel einnimmt (oder in den 4 Wochen \nvor 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.","language":28,"languageDescription":"German (Belgium)"},{"id":9737334,"uuid":"192a96ba-455c-43cf-8066-0a553295a22d","attributeTranslation":"10. 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.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737338,"uuid":"192a96ba-455c-43cf-8066-0a553295a22d","attributeTranslation":"10. 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.","language":19,"languageDescription":"Polish"}]},{"id":797255,"number":11,"principalInclusionCriteria":"Participants 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.\tEnrollment of participants previously treated with gene therapy, whether delandistrogene moxeparvovec or another investigational gene therapy product, will be capped at 25% and stratified between cohorts","principalInclusionCriteriaTranslations":[{"id":9737343,"uuid":"478287ff-5cf7-485d-a2f2-f3900ee636aa","attributeTranslation":"11. 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 \ndu dossier, seront éligibles. \na. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737340,"uuid":"478287ff-5cf7-485d-a2f2-f3900ee636aa","attributeTranslation":"11. 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. \na. Liczba uczestników wcześniej leczonych terapią genową – zarówno delandistrogenem moxeparvovec, jak i innym badanym produktem terapii genowej – zostanie ograniczona do 25% i zrównoważona pomiędzy kohortami.","language":19,"languageDescription":"Polish"},{"id":9737342,"uuid":"478287ff-5cf7-485d-a2f2-f3900ee636aa","attributeTranslation":"11. Serán aptos aquellos participantes que hayan recibido previamente moxeparvovec delandistrogénico (nombre \ncomercial 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. \na. Se limitará al 25 % la inscripción de participantes previamente tratados con terapia génica, ya sea \ndelandistrogene moxeparvovec u otro medicamento para terapia génica en investigación, y se estratificará entre cohortes.","language":7,"languageDescription":"Spanish"},{"id":9737339,"uuid":"478287ff-5cf7-485d-a2f2-f3900ee636aa","attributeTranslation":"11. 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. \na. 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.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737341,"uuid":"478287ff-5cf7-485d-a2f2-f3900ee636aa","attributeTranslation":"11. Teilnehmer, die zuvor Delandistrogen Moxeparvovec (Markenname Elevidys) entweder in einer früheren \nklinischen 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. \na. 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.","language":28,"languageDescription":"German (Belgium)"}]},{"id":797256,"number":12,"principalInclusionCriteria":"Participants 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.","principalInclusionCriteriaTranslations":[{"id":9737344,"uuid":"1abd7c23-3870-467c-8234-cce45c63b32e","attributeTranslation":"12. 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.","language":28,"languageDescription":"German (Belgium)"},{"id":9737345,"uuid":"1abd7c23-3870-467c-8234-cce45c63b32e","attributeTranslation":"12. 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.","language":19,"languageDescription":"Polish"},{"id":9737347,"uuid":"1abd7c23-3870-467c-8234-cce45c63b32e","attributeTranslation":"12. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737346,"uuid":"1abd7c23-3870-467c-8234-cce45c63b32e","attributeTranslation":"12. 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.","language":7,"languageDescription":"Spanish"},{"id":9737348,"uuid":"1abd7c23-3870-467c-8234-cce45c63b32e","attributeTranslation":"12. 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.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":797257,"number":13,"principalInclusionCriteria":"If 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.","principalInclusionCriteriaTranslations":[{"id":9737351,"uuid":"e94f456e-aaa7-498e-8843-8082a51b22d2","attributeTranslation":"13. Deelnemers die meedoen aan een fysiotherapie-/ krachttrainingsregime, moeten stabiel zijn gedurende ≥ 2 maanden voorafgaand aan het Screeningbezoek en voor de duur van het onderzoek.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737350,"uuid":"e94f456e-aaa7-498e-8843-8082a51b22d2","attributeTranslation":"13. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737352,"uuid":"e94f456e-aaa7-498e-8843-8082a51b22d2","attributeTranslation":"13. 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.","language":7,"languageDescription":"Spanish"},{"id":9737349,"uuid":"e94f456e-aaa7-498e-8843-8082a51b22d2","attributeTranslation":"13. Wenn Sie an einer Physiotherapie/Krafttraining teilnehmen, muss dieses ≥ 2 Monate vor der Screening-Visite \nund während der gesamten Studiendauer stabil sein.","language":28,"languageDescription":"German (Belgium)"},{"id":9737353,"uuid":"e94f456e-aaa7-498e-8843-8082a51b22d2","attributeTranslation":"13. 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.","language":19,"languageDescription":"Polish"}]},{"id":797258,"number":2,"principalInclusionCriteria":"Male DMD patients who are ambulatory and aged ≥ 7 to < 10 years at the time of screening","principalInclusionCriteriaTranslations":[{"id":9737354,"uuid":"06b12ff3-2292-497d-ba24-e21c6744abd2","attributeTranslation":"2. Pacientes varones con DMD que sean ambulatorios y tengan una edad comprendida entre ≥ 7 y < 10 años en el momento de la selección.","language":7,"languageDescription":"Spanish"},{"id":9737355,"uuid":"06b12ff3-2292-497d-ba24-e21c6744abd2","attributeTranslation":"2. Männliche DMD-Patienten, die gehfähig und zum Zeitpunkt des Screenings ≥ 7 bis < 10 Jahre alt sind.","language":28,"languageDescription":"German (Belgium)"},{"id":9737356,"uuid":"06b12ff3-2292-497d-ba24-e21c6744abd2","attributeTranslation":"2. Patients masculins atteints de DMD, ambulants et âgés de ≥ 7 à < 10 ans au moment de la sélection.","language":33,"languageDescription":"French (Belgium)"},{"id":9737357,"uuid":"06b12ff3-2292-497d-ba24-e21c6744abd2","attributeTranslation":"2. Mężczyźni z DMD, ambulatoryjni, w wieku od ≥ 7 do < 10 lat w momencie badania przesiewowego.","language":19,"languageDescription":"Polish"},{"id":9737358,"uuid":"06b12ff3-2292-497d-ba24-e21c6744abd2","attributeTranslation":"2. DMD-patiënt van het mannelijk geslacht, ambulant en ≥ 7 tot < 10 jaar oud op het moment van screening.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":797259,"number":3,"principalInclusionCriteria":"Completed two four-stair climb assessments at Visit 1 with a mean of 8 seconds or less (≤1 s variance).","principalInclusionCriteriaTranslations":[{"id":9737363,"uuid":"40f3f802-1263-4fc4-9db5-a5076858ecea","attributeTranslation":"3. Bei Visite 1 wurden zwei Vier-Stufen-Steigtests mit einer Durchschnittszeit von 8 Sekunden oder weniger (≤ 1 s Varianz) abgeschlossen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737360,"uuid":"40f3f802-1263-4fc4-9db5-a5076858ecea","attributeTranslation":"3. 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).","language":7,"languageDescription":"Spanish"},{"id":9737361,"uuid":"40f3f802-1263-4fc4-9db5-a5076858ecea","attributeTranslation":"3. Ukończenie dwóch prób wspinania się po czterech stopniach podczas wizyty 1 ze średnim czasem ≤ 8 sekund (przy maksymalnej różnicy ≤ 1 sekundy między próbami).","language":19,"languageDescription":"Polish"},{"id":9737362,"uuid":"40f3f802-1263-4fc4-9db5-a5076858ecea","attributeTranslation":"3. Tijdens Bezoek 1 twee vier-treden-klimtests afgerond binnen gemiddeld 8 seconden of minder (variantie ≤ 1 s).","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737359,"uuid":"40f3f802-1263-4fc4-9db5-a5076858ecea","attributeTranslation":"3. Avoir effectué deux montées chronométrées sur quatre marches lors de la visite 1 avec une moyenne de 8 secondes ou moins (variance ≤1 s).","language":33,"languageDescription":"French (Belgium)"}]},{"id":797260,"number":4,"principalInclusionCriteria":"Have a time to rise of at least 3 seconds but less than 10 seconds.","principalInclusionCriteriaTranslations":[{"id":9737364,"uuid":"9f7a3d57-ab73-4f05-b91e-1cfecbba61ab","attributeTranslation":"4. Czas wstawania z podłogi wynoszący co najmniej 3 sekundy, ale mniej niż 10 sekund.","language":19,"languageDescription":"Polish"},{"id":9737368,"uuid":"9f7a3d57-ab73-4f05-b91e-1cfecbba61ab","attributeTranslation":"4. Avoir un temps de relevé d’au moins 3 secondes mais inférieur à 10 secondes.","language":33,"languageDescription":"French (Belgium)"},{"id":9737366,"uuid":"9f7a3d57-ab73-4f05-b91e-1cfecbba61ab","attributeTranslation":"4. Die Zeit bis zum Aufsteigen muss mindestens 3 Sekunden, aber weniger als 10 Sekunden betragen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737365,"uuid":"9f7a3d57-ab73-4f05-b91e-1cfecbba61ab","attributeTranslation":"4. Overeind kunnen komen binnen 3 tot maximaal 10 seconden.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737367,"uuid":"9f7a3d57-ab73-4f05-b91e-1cfecbba61ab","attributeTranslation":"4. El tiempo de subida del paciente es de al menos 3 segundos pero inferior a 10 segundos.","language":7,"languageDescription":"Spanish"}]},{"id":797261,"number":5,"principalInclusionCriteria":"Healthy, as determined by investigator including medical history, psychiatric history, and no clinically significant findings on physical examination, laboratory tests, and cardiac monitoring.","principalInclusionCriteriaTranslations":[{"id":9737372,"uuid":"cf85ab00-12c5-4be9-82c2-fb82a5475de2","attributeTranslation":"5. 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.","language":19,"languageDescription":"Polish"},{"id":9737371,"uuid":"cf85ab00-12c5-4be9-82c2-fb82a5475de2","attributeTranslation":"5. El paciente está en buen estado de salud, según las valoraciones del investigador, incluyendo los antecedentes \nmédicos y psiquiátricos, y sin hallazgos clínicamente relevantes en la exploración física, las pruebas de laboratorio y el seguimiento de la función cardiaca.","language":7,"languageDescription":"Spanish"},{"id":9737373,"uuid":"cf85ab00-12c5-4be9-82c2-fb82a5475de2","attributeTranslation":"5. Gesund, wie vom Prüfarzt unter Berücksichtigung von Anamnese und psychiatrischer Anamnese festgestellt, und keine klinisch signifikanten Befunde bei körperlicher Untersuchung, Labortests und Herzüberwachung.","language":28,"languageDescription":"German (Belgium)"},{"id":9737369,"uuid":"cf85ab00-12c5-4be9-82c2-fb82a5475de2","attributeTranslation":"5. 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.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737370,"uuid":"cf85ab00-12c5-4be9-82c2-fb82a5475de2","attributeTranslation":"5. 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.","language":33,"languageDescription":"French (Belgium)"}]},{"id":797262,"number":6,"principalInclusionCriteria":"Ability 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 restrictions","principalInclusionCriteriaTranslations":[{"id":9737378,"uuid":"97d351f2-ebb6-4e7f-9463-83a053424d04","attributeTranslation":"6. 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.","language":7,"languageDescription":"Spanish"},{"id":9737375,"uuid":"97d351f2-ebb6-4e7f-9463-83a053424d04","attributeTranslation":"6. 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.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737377,"uuid":"97d351f2-ebb6-4e7f-9463-83a053424d04","attributeTranslation":"6. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737376,"uuid":"97d351f2-ebb6-4e7f-9463-83a053424d04","attributeTranslation":"6. 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.","language":19,"languageDescription":"Polish"},{"id":9737374,"uuid":"97d351f2-ebb6-4e7f-9463-83a053424d04","attributeTranslation":"6. 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.","language":28,"languageDescription":"German (Belgium)"}]},{"id":797263,"number":7,"principalInclusionCriteria":"Participant’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.","principalInclusionCriteriaTranslations":[{"id":9737379,"uuid":"b278e74b-cf3f-45e8-a781-4cbc2f5d235c","attributeTranslation":"7. 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.","language":7,"languageDescription":"Spanish"},{"id":9737383,"uuid":"b278e74b-cf3f-45e8-a781-4cbc2f5d235c","attributeTranslation":"7. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737381,"uuid":"b278e74b-cf3f-45e8-a781-4cbc2f5d235c","attributeTranslation":"7. 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.","language":19,"languageDescription":"Polish"},{"id":9737382,"uuid":"b278e74b-cf3f-45e8-a781-4cbc2f5d235c","attributeTranslation":"7. Die Eltern oder der/die gesetzliche(n) Vormund(e) des Teilnehmers haben ihr schriftliches Einverständnis zur \nTeilnahme 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.","language":28,"languageDescription":"German (Belgium)"},{"id":9737380,"uuid":"b278e74b-cf3f-45e8-a781-4cbc2f5d235c","attributeTranslation":"7. 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.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":797264,"number":8,"principalInclusionCriteria":"Stable 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.\tPatients who are not receiving glucocorticosteroids are also eligible if stopped ≥ 3 months prior to the Screening Visit.","principalInclusionCriteriaTranslations":[{"id":9737387,"uuid":"fb6013c2-5e98-4a72-a3fd-992230871541","attributeTranslation":"8. 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. \na. Patiënten die geen glucocorticosteroïden krijgen, komen ook in aanmerking als ze ≥ 3 maanden voorafgaand aan het Screeningbezoek zijn gestopt.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737386,"uuid":"fb6013c2-5e98-4a72-a3fd-992230871541","attributeTranslation":"8. 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. \na. Les patients qui ne reçoivent pas de glucocorticoïdes sont également éligibles s’ils ont arrêté ≥ 3 mois avant la visite de sélection","language":33,"languageDescription":"French (Belgium)"},{"id":9737385,"uuid":"fb6013c2-5e98-4a72-a3fd-992230871541","attributeTranslation":"8. 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. \na. Pacjenci niestosujący glikokortykosteroidów również mogą zostać włączeni, pod warunkiem że leczenie zostało zakończone co najmniej 3 miesiące przed wizytą przesiewową.","language":19,"languageDescription":"Polish"},{"id":9737384,"uuid":"fb6013c2-5e98-4a72-a3fd-992230871541","attributeTranslation":"8. 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. \na. Los pacientes que no estén recibiendo glucocorticoides también son aptos si se suspenden ≥ 3 meses antes de la visita de selección.","language":7,"languageDescription":"Spanish"},{"id":9737388,"uuid":"fb6013c2-5e98-4a72-a3fd-992230871541","attributeTranslation":"8. 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. \na. Patienten, die keine Glukokortikosteroide erhalten, sind ebenfalls teilnahmeberechtigt, wenn sie diese ≥ 3 Monate vor der Screening-Visite abgesetzt haben.","language":28,"languageDescription":"German (Belgium)"}]},{"id":797265,"number":9,"principalInclusionCriteria":"Stable 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.","principalInclusionCriteriaTranslations":[{"id":9737389,"uuid":"d4389451-9556-4890-be22-b4b9df66f9d2","attributeTranslation":"9. 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.","language":7,"languageDescription":"Spanish"},{"id":9737390,"uuid":"d4389451-9556-4890-be22-b4b9df66f9d2","attributeTranslation":"9. 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.","language":19,"languageDescription":"Polish"},{"id":9737391,"uuid":"d4389451-9556-4890-be22-b4b9df66f9d2","attributeTranslation":"9. 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.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737393,"uuid":"d4389451-9556-4890-be22-b4b9df66f9d2","attributeTranslation":"9. Stabile Dosen von verschreibungspflichtigen Medikamenten, einschließlich ACE-Hemmern, β-Blockern und Diuretika (mit Ausnahme von Glukokortikosteroiden) und rezeptfreien Medikamenten und/oder pflanzlichen \nNahrungsergänzungsmitteln zur unterstützenden Behandlung ≥ 1 Monat vor der Screening-Visite und während der gesamten Dauer der Studie.","language":28,"languageDescription":"German (Belgium)"},{"id":9737392,"uuid":"d4389451-9556-4890-be22-b4b9df66f9d2","attributeTranslation":"9. 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.","language":33,"languageDescription":"French (Belgium)"}]}],"principalExclusionCriteria":[{"id":1366039,"number":1,"principalExclusionCriteria":"Ambulatory patients expected to experience loss of ambulation within ≤ 12 months.","principalExclusionCriteriaTranslations":[{"id":9737246,"uuid":"f41c01c3-39c6-478f-9b98-a5e02cca48b4","attributeTranslation":"1. Patients ambulants dont on s’attend à ce qu’ils perdent leur mobilité dans un délai de ≤ 12 mois.","language":33,"languageDescription":"French (Belgium)"},{"id":9737247,"uuid":"f41c01c3-39c6-478f-9b98-a5e02cca48b4","attributeTranslation":"1. Ambulatoryjni pacjenci, u których przewiduje się utratę zdolności do samodzielnego poruszania się w czasie ≤ 12 miesięcy","language":19,"languageDescription":"Polish"},{"id":9737244,"uuid":"f41c01c3-39c6-478f-9b98-a5e02cca48b4","attributeTranslation":"Pacientes ambulatorios probablemente experimenten pérdida de la deambulación en ≤ 12 meses.","language":7,"languageDescription":"Spanish"},{"id":9737245,"uuid":"f41c01c3-39c6-478f-9b98-a5e02cca48b4","attributeTranslation":"1. Gehfähige Patienten, die voraussichtlich innerhalb von ≤ 12 Monaten ihre Gehfähigkeit verlieren werden.","language":28,"languageDescription":"German (Belgium)"},{"id":9737248,"uuid":"f41c01c3-39c6-478f-9b98-a5e02cca48b4","attributeTranslation":"Een ambulante patiënt die naar verwachting binnen ≤ 12 maanden verlies van ambulantie zal ervaren.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":1366040,"number":10,"principalExclusionCriteria":"Participants for whom MRI or open muscle biopsy are contraindicated.","principalExclusionCriteriaTranslations":[{"id":9737249,"uuid":"e070ae91-51ab-4184-ab15-be40c255b795","attributeTranslation":"4. Teilnehmer, bei denen MRT oder offene Muskelbiopsie kontraindiziert sind.","language":28,"languageDescription":"German (Belgium)"},{"id":9737251,"uuid":"e070ae91-51ab-4184-ab15-be40c255b795","attributeTranslation":"Participantes en los que estén contraindicadas la IRM o la biopsia muscular abierta.","language":7,"languageDescription":"Spanish"},{"id":9737253,"uuid":"e070ae91-51ab-4184-ab15-be40c255b795","attributeTranslation":"4. Participants pour lesquels l’IRM ou la biopsie musculaire ouverte sont contre-indiquées.","language":33,"languageDescription":"French (Belgium)"},{"id":9737252,"uuid":"e070ae91-51ab-4184-ab15-be40c255b795","attributeTranslation":"Een contra-indicatie voor MRI of open spierbiopsie.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737250,"uuid":"e070ae91-51ab-4184-ab15-be40c255b795","attributeTranslation":"4. Przeciwwskazania do wykonania rezonansu magnetycznego (MRI) lub otwartej biopsji mięśnia.","language":19,"languageDescription":"Polish"}]},{"id":1366041,"number":11,"principalExclusionCriteria":"Surgery (e.g., stomach bypass) or medical condition that might affect absorption of medicines.","principalExclusionCriteriaTranslations":[{"id":9737255,"uuid":"e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9","attributeTranslation":"Een operatie (bijv. maagomleiding) of een medische aandoening die de opname van geneesmiddelen kan beïnvloeden.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737256,"uuid":"e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9","attributeTranslation":"5. Przebyte zabiegi chirurgiczne (np. ominięcie żołądkowe) lub schorzenia mogące wpływać na wchłanianie leków.","language":19,"languageDescription":"Polish"},{"id":9737258,"uuid":"e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9","attributeTranslation":"5. Intervention chirurgicale (par exemple, pontage gastrique) ou état de santé susceptible d’affecter l’absorption des médicaments.","language":33,"languageDescription":"French (Belgium)"},{"id":9737254,"uuid":"e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9","attributeTranslation":"5. Operationen (z. B. Magenbypass) oder Erkrankungen, die die Aufnahme von Arzneimitteln beeinträchtigen können.","language":28,"languageDescription":"German (Belgium)"},{"id":9737257,"uuid":"e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9","attributeTranslation":"Cirugía (por ejemplo, bypass gástrico) o enfermedad que pueda afectar a la absorción de los medicamentos.","language":7,"languageDescription":"Spanish"}]},{"id":1366042,"number":12,"principalExclusionCriteria":"Have 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.","principalExclusionCriteriaTranslations":[{"id":9737260,"uuid":"d31c399b-3374-4d7a-97c4-8f409c8effc5","attributeTranslation":"6. Ostre objawy ze strony przewodu pokarmowego (np. nudności, wymioty, biegunka, zgaga), ostra infekcja (np. grypa), poważna infekcja lub rozpoznany proces zapalny podczas wizyty przesiewowej.","language":19,"languageDescription":"Polish"},{"id":9737262,"uuid":"d31c399b-3374-4d7a-97c4-8f409c8effc5","attributeTranslation":"6. 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.","language":28,"languageDescription":"German (Belgium)"},{"id":9737261,"uuid":"d31c399b-3374-4d7a-97c4-8f409c8effc5","attributeTranslation":"6. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737259,"uuid":"d31c399b-3374-4d7a-97c4-8f409c8effc5","attributeTranslation":"Pacientes 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.","language":7,"languageDescription":"Spanish"},{"id":9737263,"uuid":"d31c399b-3374-4d7a-97c4-8f409c8effc5","attributeTranslation":"Acute gastro-intestinale symptomen (zoals misselijkheid, braken, diarree, brandend maagzuur), een acute infectie (zoals influenza) of een ernstige infectie of bekend ontstekingsproces bij screening.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":1366043,"number":13,"principalExclusionCriteria":"Impaired cardiac function defined as a left ventricular ejection fraction of < 50% on screening cardiac assessments (echocardiogram or MRI) or evidence of symptomatic cardiomyopathy.","principalExclusionCriteriaTranslations":[{"id":9737267,"uuid":"75c01d77-900e-4472-92da-22a72c577834","attributeTranslation":"7. Eingeschränkte Herzfunktion, definiert als linksventrikuläre Auswurffraktion < 50 % bei der kardiologischen Screeninguntersuchung (Echokardiogramm oder MRT) oder Anzeichen einer symptomatischen Kardiomyopathie.","language":28,"languageDescription":"German (Belgium)"},{"id":9737265,"uuid":"75c01d77-900e-4472-92da-22a72c577834","attributeTranslation":"Een verminderde hartfunctie, gedefinieerd als een linkerventrikelejectiefractie van < 50% bij hartonderzoek tijdens screening (echocardiogram of MRI) of tekenen van symptomatische cardiomyopathie.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737264,"uuid":"75c01d77-900e-4472-92da-22a72c577834","attributeTranslation":"7. Upośledzona czynność serca, definiowana jako frakcja wyrzutowa lewej komory < 50% w badaniach przesiewowych serca (echokardiografia lub MRI) lub obecność objawowej kardiomiopatii.","language":19,"languageDescription":"Polish"},{"id":9737268,"uuid":"75c01d77-900e-4472-92da-22a72c577834","attributeTranslation":"Función 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.","language":7,"languageDescription":"Spanish"},{"id":9737266,"uuid":"75c01d77-900e-4472-92da-22a72c577834","attributeTranslation":"7. 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.","language":33,"languageDescription":"French (Belgium)"}]},{"id":1366044,"number":14,"principalExclusionCriteria":"A forced vital capacity < 60% predicted at the Screening Visit.","principalExclusionCriteriaTranslations":[{"id":9737270,"uuid":"9d4f2469-e7ca-4afe-ac4b-244ca250b85c","attributeTranslation":"Een tijdens het Screeningbezoek voorspelde geforceerde vitale capaciteit < 60%.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737269,"uuid":"9d4f2469-e7ca-4afe-ac4b-244ca250b85c","attributeTranslation":"Una capacidad vital forzada < 60 % de la prevista en la visita de selección.","language":7,"languageDescription":"Spanish"},{"id":9737273,"uuid":"9d4f2469-e7ca-4afe-ac4b-244ca250b85c","attributeTranslation":"8. Wymuszona pojemność życiowa (FVC) < 60% wartości należnej podczas wizyty przesiewowej.","language":19,"languageDescription":"Polish"},{"id":9737272,"uuid":"9d4f2469-e7ca-4afe-ac4b-244ca250b85c","attributeTranslation":"8. Capacité vitale forcée < 60 % de la valeur prédite lors de la visite de sélection.","language":33,"languageDescription":"French (Belgium)"},{"id":9737271,"uuid":"9d4f2469-e7ca-4afe-ac4b-244ca250b85c","attributeTranslation":"8. Eine forcierte Vitalkapazität < 60 % bei der Screening-Visite.","language":28,"languageDescription":"German (Belgium)"}]},{"id":1366045,"number":15,"principalExclusionCriteria":"Presence 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.","principalExclusionCriteriaTranslations":[{"id":9737277,"uuid":"9a34faa1-c6f8-456a-83c8-6af982b5f84a","attributeTranslation":"9.\tHet 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.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737275,"uuid":"9a34faa1-c6f8-456a-83c8-6af982b5f84a","attributeTranslation":"9. 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.","language":28,"languageDescription":"German (Belgium)"},{"id":9737278,"uuid":"9a34faa1-c6f8-456a-83c8-6af982b5f84a","attributeTranslation":"9.\tWystępowanie 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.","language":19,"languageDescription":"Polish"},{"id":9737274,"uuid":"9a34faa1-c6f8-456a-83c8-6af982b5f84a","attributeTranslation":"9.\tPrésence 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737276,"uuid":"9a34faa1-c6f8-456a-83c8-6af982b5f84a","attributeTranslation":"9. 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 .","language":7,"languageDescription":"Spanish"}]},{"id":1366046,"number":16,"principalExclusionCriteria":"Participants with any known and discernable history of substance abuse and/or dependence.","principalExclusionCriteriaTranslations":[{"id":9737282,"uuid":"ef67e55a-4f3d-4826-9d38-924d70edcd15","attributeTranslation":"16.\tParticipants ayant des antécédents connus et discernables d’abus et/ou de dépendance à des substances","language":33,"languageDescription":"French (Belgium)"},{"id":9737279,"uuid":"ef67e55a-4f3d-4826-9d38-924d70edcd15","attributeTranslation":"16.\tUczestnicy z jakąkolwiek znaną i rozpoznawalną historią nadużywania substancji psychoaktywnych i/lub uzależnienia.","language":19,"languageDescription":"Polish"},{"id":9737283,"uuid":"ef67e55a-4f3d-4826-9d38-924d70edcd15","attributeTranslation":"16.\tDeelnemers met om het even welke bekende en herkenbare voorgeschiedenis van middelenmisbruik en/of -afhankelijkheid.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737281,"uuid":"ef67e55a-4f3d-4826-9d38-924d70edcd15","attributeTranslation":"16.\tParticipantes con cualquier historial conocido y discernible de abuso y/o dependencia de sustancias.","language":7,"languageDescription":"Spanish"},{"id":9737280,"uuid":"ef67e55a-4f3d-4826-9d38-924d70edcd15","attributeTranslation":"16. Teilnehmer mit einer bekannten und erkennbaren Vorgeschichte von Missbrauch und/oder -Abhängigkeit von Substanzen","language":28,"languageDescription":"German (Belgium)"}]},{"id":1366047,"number":2,"principalExclusionCriteria":"Participants maintained on a ten day on/ten day off corticosteroid regimen","principalExclusionCriteriaTranslations":[{"id":9737287,"uuid":"87de9c8e-0315-4fda-ace2-c7301a0b70b4","attributeTranslation":"Een aangehouden corticosteroïdregime van tien dagen op/tien dagen af.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737286,"uuid":"87de9c8e-0315-4fda-ace2-c7301a0b70b4","attributeTranslation":"Participantes que estén siguiendo una pauta de corticosteroides de diez días con medicación seguida de diez días sin medicación.","language":7,"languageDescription":"Spanish"},{"id":9737285,"uuid":"87de9c8e-0315-4fda-ace2-c7301a0b70b4","attributeTranslation":"10. Die Teilnehmer erhielten eine zehntägige Kortikosteroidtherapie mit anschließender zehntägiger Pause.","language":28,"languageDescription":"German (Belgium)"},{"id":9737284,"uuid":"87de9c8e-0315-4fda-ace2-c7301a0b70b4","attributeTranslation":"10. Participants maintenus sous un régime de corticostéroïdes de dix jours avec traitement et dix jours sans traitement.","language":33,"languageDescription":"French (Belgium)"},{"id":9737288,"uuid":"87de9c8e-0315-4fda-ace2-c7301a0b70b4","attributeTranslation":"10. Uczestnicy pozostający na schemacie leczenia glikokortykosteroidami typu dziesięć dni stosowania / dziesięć dni przerwy.","language":19,"languageDescription":"Polish"}]},{"id":1366048,"number":3,"principalExclusionCriteria":"11\tOngoing 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.\tParticipants 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.\tParticipants receiving a stable dose of givinostat (brand name Duvyzat) for at least18 months or longer prior to the Screening Visit will be eligible. a.\tParticipants 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.\tUse 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.","principalExclusionCriteriaTranslations":[{"id":9737291,"uuid":"3e4de5d2-2a85-4bad-a556-4ea75d37edeb","attributeTranslation":"11.\tAktuelle 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.\na.\tTeilnehmer, 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. \nb.\tTeilnehmer, die eine stabile Dosis von Givinostat (Markenname Duvyzat) seit mindestens 18 Monaten oder länger vor der Screening-Visite erhalten, sind teilnahmeberechtigt. a.\tTeilnehmer, 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.\nc.\tDie 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.","language":28,"languageDescription":"German (Belgium)"},{"id":9737290,"uuid":"3e4de5d2-2a85-4bad-a556-4ea75d37edeb","attributeTranslation":"11.\tParticipation 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.\na.\tLes 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.\nb.\tLes 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.\tLes 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\nc.\tL’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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737289,"uuid":"3e4de5d2-2a85-4bad-a556-4ea75d37edeb","attributeTranslation":"11.\tParticipación 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.\na.\tSerán 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.\nb.\tSerán 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.\tLos 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.\nc.\tNo 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.","language":7,"languageDescription":"Spanish"},{"id":9737293,"uuid":"3e4de5d2-2a85-4bad-a556-4ea75d37edeb","attributeTranslation":"11.\tBieżący 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.\na.\tUczestnicy, 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.\nb.\tUczestnicy przyjmujący stabilną dawkę givinostatu (nazwa handlowa: Duvyzat) przez co najmniej 18 miesięcy przed wizytą przesiewową lub dłużej będą kwalifikować się do udziału. a.\tUczestnicy, 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.\nc.\tStosowanie 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.","language":19,"languageDescription":"Polish"},{"id":9737292,"uuid":"3e4de5d2-2a85-4bad-a556-4ea75d37edeb","attributeTranslation":"11.\tLopende 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.\na.\tPersonen 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.\nb.\tPersonen die ten minste 18 maanden of langer voorafgaand aan het Screeningbezoek een stabiele dosis givinostat (merknaam Duvyzat) kregen, komen in aanmerking voor deelname. a.\tPersonen 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\nc.\tHet 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.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":1366049,"number":4,"principalExclusionCriteria":"Received SAT-3247 in another study.","principalExclusionCriteriaTranslations":[{"id":9737297,"uuid":"848aa54d-3f96-4b87-b896-9339f34b63ee","attributeTranslation":"Het hebben gekregen van SAT-3247 in een ander onderzoek.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737295,"uuid":"848aa54d-3f96-4b87-b896-9339f34b63ee","attributeTranslation":"12. Réception de SAT-3247 dans le cadre d’une autre étude.","language":33,"languageDescription":"French (Belgium)"},{"id":9737294,"uuid":"848aa54d-3f96-4b87-b896-9339f34b63ee","attributeTranslation":"12. Uprzednie uczestnictwo w badaniu z zastosowaniem SAT-3247.","language":19,"languageDescription":"Polish"},{"id":9737298,"uuid":"848aa54d-3f96-4b87-b896-9339f34b63ee","attributeTranslation":"Pacientes que hayan recibido SAT-3247 en otro estudio.","language":7,"languageDescription":"Spanish"},{"id":9737296,"uuid":"848aa54d-3f96-4b87-b896-9339f34b63ee","attributeTranslation":"12. SAT-3247 wurde in einer anderen Studie erhalten.","language":28,"languageDescription":"German (Belgium)"}]},{"id":1366050,"number":5,"principalExclusionCriteria":"Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator.","principalExclusionCriteriaTranslations":[{"id":9737299,"uuid":"c24839d9-6102-4c12-a7b2-ed2d8f7f5871","attributeTranslation":"Problemas graves cognitivos o de comportamiento que impidan la participación en el estudio, a juicio del investigador.","language":7,"languageDescription":"Spanish"},{"id":9737303,"uuid":"c24839d9-6102-4c12-a7b2-ed2d8f7f5871","attributeTranslation":"13. Schwere Verhaltensstörungen oder kognitive Probleme, die nach Ansicht des Prüfarztes eine Teilnahme an der Studie ausschließen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737300,"uuid":"c24839d9-6102-4c12-a7b2-ed2d8f7f5871","attributeTranslation":"13. Poważne problemy behawioralne lub poznawcze, które w ocenie badacza uniemożliwiają udział w badaniu.","language":19,"languageDescription":"Polish"},{"id":9737302,"uuid":"c24839d9-6102-4c12-a7b2-ed2d8f7f5871","attributeTranslation":"Ernstige gedrags- of cognitieve problemen die naar de mening van de onderzoeker deelname aan het onderzoek uitsluiten.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737301,"uuid":"c24839d9-6102-4c12-a7b2-ed2d8f7f5871","attributeTranslation":"13. Problèmes comportementaux ou cognitifs graves qui empêchent la participation à l’étude, de l’avis de l’investigateur.","language":33,"languageDescription":"French (Belgium)"}]},{"id":1366051,"number":6,"principalExclusionCriteria":"Positive test for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCV) or HIV at Screening Visit.","principalExclusionCriteriaTranslations":[{"id":9737305,"uuid":"db682db8-30d9-413d-9f9c-ca168c90590a","attributeTranslation":"Positief testresultaat voor hepatitis B surface antigen (HBsAg), antistof voor hepatitis C-virus (HCV) of hiv tijdens Screeningbezoek.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737304,"uuid":"db682db8-30d9-413d-9f9c-ca168c90590a","attributeTranslation":"14. Positiver Test auf Hepatitis-B-Oberflächenantigen (HBsAg), Hepatitis-C-Antikörper (HCV) oder HIV bei der Screening-Visite.","language":28,"languageDescription":"German (Belgium)"},{"id":9737308,"uuid":"db682db8-30d9-413d-9f9c-ca168c90590a","attributeTranslation":"14. 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.","language":19,"languageDescription":"Polish"},{"id":9737307,"uuid":"db682db8-30d9-413d-9f9c-ca168c90590a","attributeTranslation":"14. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737306,"uuid":"db682db8-30d9-413d-9f9c-ca168c90590a","attributeTranslation":"Prueba 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.","language":7,"languageDescription":"Spanish"}]},{"id":1366052,"number":7,"principalExclusionCriteria":"Employee of the Sponsor, the CRO and/or study site or their relatives","principalExclusionCriteriaTranslations":[{"id":9737313,"uuid":"3e13a4dd-04d0-486d-8843-0280a5ae4c2b","attributeTranslation":"15. Mitarbeiter des Sponsors, der CRO und/oder des Studienzentrums oder deren Angehörige.","language":28,"languageDescription":"German (Belgium)"},{"id":9737310,"uuid":"3e13a4dd-04d0-486d-8843-0280a5ae4c2b","attributeTranslation":"Werknemers van de opdrachtgever, de CRO en/of onderzoekscentrum en hun familieleden.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737309,"uuid":"3e13a4dd-04d0-486d-8843-0280a5ae4c2b","attributeTranslation":"Empleado/a del promotor, de la CRO y/o del centro del estudio o sus familiares.","language":7,"languageDescription":"Spanish"},{"id":9737312,"uuid":"3e13a4dd-04d0-486d-8843-0280a5ae4c2b","attributeTranslation":"15. Employés du promoteur, de la CRO et / ou du site de l’étude ou leurs proches.","language":33,"languageDescription":"French (Belgium)"},{"id":9737311,"uuid":"3e13a4dd-04d0-486d-8843-0280a5ae4c2b","attributeTranslation":"15. Pracownik Sponsora, organizacji prowadzącej badanie (CRO) i/lub ośrodka badawczego, lub osoba spokrewniona z którymkolwiek z nich.","language":19,"languageDescription":"Polish"}]},{"id":1366053,"number":8,"principalExclusionCriteria":"Presence 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.","principalExclusionCriteriaTranslations":[{"id":9737315,"uuid":"5ff74ed1-f4a1-405c-9bc3-aed61dd549d4","attributeTranslation":"Presencia 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.","language":7,"languageDescription":"Spanish"},{"id":9737317,"uuid":"5ff74ed1-f4a1-405c-9bc3-aed61dd549d4","attributeTranslation":"2. 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.","language":28,"languageDescription":"German (Belgium)"},{"id":9737318,"uuid":"5ff74ed1-f4a1-405c-9bc3-aed61dd549d4","attributeTranslation":"Een 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.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737314,"uuid":"5ff74ed1-f4a1-405c-9bc3-aed61dd549d4","attributeTranslation":"2. 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.","language":19,"languageDescription":"Polish"},{"id":9737316,"uuid":"5ff74ed1-f4a1-405c-9bc3-aed61dd549d4","attributeTranslation":"2. 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.","language":33,"languageDescription":"French (Belgium)"}]},{"id":1366054,"number":9,"principalExclusionCriteria":"Prior 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.","principalExclusionCriteriaTranslations":[{"id":9737322,"uuid":"b7c64826-10ca-4cd7-8ffa-7e033764a662","attributeTranslation":"3. 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.","language":19,"languageDescription":"Polish"},{"id":9737321,"uuid":"b7c64826-10ca-4cd7-8ffa-7e033764a662","attributeTranslation":"Een 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.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737323,"uuid":"b7c64826-10ca-4cd7-8ffa-7e033764a662","attributeTranslation":"3. 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737320,"uuid":"b7c64826-10ca-4cd7-8ffa-7e033764a662","attributeTranslation":"3. 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.","language":28,"languageDescription":"German (Belgium)"},{"id":9737319,"uuid":"b7c64826-10ca-4cd7-8ffa-7e033764a662","attributeTranslation":"Afección 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.","language":7,"languageDescription":"Spanish"}]}]},"endPoint":{"primaryEndPoints":[{"id":933632,"number":1,"endPoint":"The primary efficacy endpoint is defined as the change from baseline in muscle force measurements as determined by dynamometry at Week 12.","isPrimary":true,"endPointTranslations":[{"id":9737173,"uuid":"19c0199a-3aaa-4ad0-a4e8-b09c4e9f76df","attributeTranslation":"Le 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737170,"uuid":"19c0199a-3aaa-4ad0-a4e8-b09c4e9f76df","attributeTranslation":"Pierwszorzędowy punkt końcowy skuteczności zdefiniowano jako zmianę względem wartości wyjściowej w pomiarach siły mięśniowej, określonych za pomocą dynamometrii, w 12. tygodniu.","language":19,"languageDescription":"Polish"},{"id":9737172,"uuid":"19c0199a-3aaa-4ad0-a4e8-b09c4e9f76df","attributeTranslation":"Der primäre Wirksamkeitsendpunkt ist definiert als die Veränderung der dynamometrisch gemessenen Muskelkraft in Woche 12 im Vergleich zur Baseline.","language":28,"languageDescription":"German (Belgium)"},{"id":9737169,"uuid":"19c0199a-3aaa-4ad0-a4e8-b09c4e9f76df","attributeTranslation":"El 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.","language":7,"languageDescription":"Spanish"},{"id":9737171,"uuid":"19c0199a-3aaa-4ad0-a4e8-b09c4e9f76df","attributeTranslation":"Het primaire eindpunt voor werkzaamheid wordt gedefinieerd als de verandering ten opzichte van de nulmeting voor spierkracht, zoals gemeten met dynamometrie in Week 12.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":933633,"number":2,"endPoint":"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-SSRS","isPrimary":true,"endPointTranslations":[{"id":9737177,"uuid":"70943981-4bb4-4122-af9f-68b6b5f652a6","attributeTranslation":"Zu den Sicherheitsendpunkten gehören das Auftreten, der Schweregrad und die Beziehung zu SAT-3247 von \nunerwünschten Ereignissen sowie das Auftreten klinisch signifikanter Veränderungen bei der körperlichen Untersuchung, bei klinischen Labormessungen, bei den Vitalparametern, beim EKG und beim C-SSRS.","language":28,"languageDescription":"German (Belgium)"},{"id":9737176,"uuid":"70943981-4bb4-4122-af9f-68b6b5f652a6","attributeTranslation":"Los 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).","language":7,"languageDescription":"Spanish"},{"id":9737178,"uuid":"70943981-4bb4-4122-af9f-68b6b5f652a6","attributeTranslation":"Les critères d’évaluation de la sécurité comprennent l’incidence, la gravité et la relation avec le SAT-3247 des \névénements 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737175,"uuid":"70943981-4bb4-4122-af9f-68b6b5f652a6","attributeTranslation":"Punkty 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.","language":19,"languageDescription":"Polish"},{"id":9737174,"uuid":"70943981-4bb4-4122-af9f-68b6b5f652a6","attributeTranslation":"Veiligheidseindpunten zijn onder meer incidentie, ernst en relatie met SAT-3247 van bijwerkingen, evenals het \noptreden van klinisch significante veranderingen bij lichamelijk onderzoek, klinische laboratoriummetingen, vitale functies, ecg en C-SSRS.","language":36,"languageDescription":"Dutch (Belgium)"}]}],"secondaryEndPoints":[{"id":933634,"number":1,"endPoint":"Changes from baseline in intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in vastus lateralis at Week 12","isPrimary":false,"endPointTranslations":[{"id":9737181,"uuid":"f0df4176-1260-4cb0-ae8d-98d7a8f5776b","attributeTranslation":"Veränderungen des intramuskulären Fettanteils in der quantitativen Magnetresonanz (qMR) im Vastus lateralis in Woche 12 gegenüber der Baseline.","language":28,"languageDescription":"German (Belgium)"},{"id":9737179,"uuid":"f0df4176-1260-4cb0-ae8d-98d7a8f5776b","attributeTranslation":"Veranderingen ten opzichte van de nulmeting voor de kwantitatieve magnetische resonantie (qMR) in de intramusculaire vetfractie in spierweefsel van de vastus lateralis na 12 weken.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737182,"uuid":"f0df4176-1260-4cb0-ae8d-98d7a8f5776b","attributeTranslation":"Variaciones 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.","language":7,"languageDescription":"Spanish"},{"id":9737183,"uuid":"f0df4176-1260-4cb0-ae8d-98d7a8f5776b","attributeTranslation":"Les 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737180,"uuid":"f0df4176-1260-4cb0-ae8d-98d7a8f5776b","attributeTranslation":"Zmiany względem wartości wyjściowej w frakcji tłuszczu śródmięśniowego w ilościowym rezonansie magnetycznym (qMR) mięśnia obszernego bocznego w 12. tygodniu.","language":19,"languageDescription":"Polish"}]},{"id":933635,"number":2,"endPoint":"Changes from baseline in proton muscle transverse relaxation time (T2) in vastus lateralis at Week 12.","isPrimary":false,"endPointTranslations":[{"id":9737184,"uuid":"b8d27f0a-5ad5-45c2-9ad2-5943f475c94b","attributeTranslation":"Veranderingen ten opzichte van de nulmeting voor transversale relaxatietijd van protonen in spierweefsel (T2) van de vastus lateralis in Week 12.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737188,"uuid":"b8d27f0a-5ad5-45c2-9ad2-5943f475c94b","attributeTranslation":"Variaciones con respecto al valor basal en el tiempo de relajación transversal de los protones (T2) en el músculo vasto lateral en la semana 12.","language":7,"languageDescription":"Spanish"},{"id":9737187,"uuid":"b8d27f0a-5ad5-45c2-9ad2-5943f475c94b","attributeTranslation":"Les 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737186,"uuid":"b8d27f0a-5ad5-45c2-9ad2-5943f475c94b","attributeTranslation":"Zmiany względem wartości wyjściowej w czasie relaksacji poprzecznej protonów (T2) w mięśniu obszernym bocznym w 12. tygodniu.","language":19,"languageDescription":"Polish"},{"id":9737185,"uuid":"b8d27f0a-5ad5-45c2-9ad2-5943f475c94b","attributeTranslation":"Veränderungen der transversalen Relaxationszeit (T2) der Muskelprotonen im Vastus lateralis in Woche 12 gegenüber der Baseline.","language":28,"languageDescription":"German (Belgium)"}]},{"id":933636,"number":3,"endPoint":"Changes from baseline in Regeneration Index in open muscle biopsy of the biceps brachii at Week 12","isPrimary":false,"endPointTranslations":[{"id":9737191,"uuid":"8c90e126-35ae-4d72-aa40-1ce65a8e357a","attributeTranslation":"Zmiany względem wartości wyjściowej w wskaźniku regeneracji w otwartej biopsji mięśnia dwugłowego ramienia w 12. tygodniu","language":19,"languageDescription":"Polish"},{"id":9737190,"uuid":"8c90e126-35ae-4d72-aa40-1ce65a8e357a","attributeTranslation":"Variaciones con respecto al valor basal en el índice de regeneración en biopsia muscular abierta del bíceps braquial en la semana 12.","language":7,"languageDescription":"Spanish"},{"id":9737193,"uuid":"8c90e126-35ae-4d72-aa40-1ce65a8e357a","attributeTranslation":"Veranderingen ten opzichte van de nulmeting voor regeneratie-index in open spierbiopt van de biceps brachii in Week 12.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737192,"uuid":"8c90e126-35ae-4d72-aa40-1ce65a8e357a","attributeTranslation":"Les 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737189,"uuid":"8c90e126-35ae-4d72-aa40-1ce65a8e357a","attributeTranslation":"Veränderungen des Regenerationsindex bei offener Muskelbiopsie des Musculus biceps brachii in Woche 12 gegenüber der Baseline.","language":28,"languageDescription":"German (Belgium)"}]},{"id":933637,"number":4,"endPoint":"Changes from baseline in function as determined by NSAA assessment at Week 12","isPrimary":false,"endPointTranslations":[{"id":9737195,"uuid":"597000f9-bfbc-4718-8c62-c48b42e23388","attributeTranslation":"Veranderingen ten opzichte van de nulmeting voor functionaliteit zoals bepaald met een NSAA-beoordeling in Week 12.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737196,"uuid":"597000f9-bfbc-4718-8c62-c48b42e23388","attributeTranslation":"Zmiany względem wartości wyjściowej w zakresie funkcji, określone na podstawie oceny NSAA, w 12. tygodniu.","language":19,"languageDescription":"Polish"},{"id":9737194,"uuid":"597000f9-bfbc-4718-8c62-c48b42e23388","attributeTranslation":"Variaciones con respecto al valor basal en la función según la evaluación con la escala North Star Ambulatory Assessment (NSAA) en la semana 12.","language":7,"languageDescription":"Spanish"},{"id":9737197,"uuid":"597000f9-bfbc-4718-8c62-c48b42e23388","attributeTranslation":"Les changements par rapport à la valeur de début d’étude dans la fonction déterminée par le test NSAA à la semaine 12.","language":33,"languageDescription":"French (Belgium)"},{"id":9737198,"uuid":"597000f9-bfbc-4718-8c62-c48b42e23388","attributeTranslation":"Veränderungen der Funktion gegenüber der Baseline, ermittelt durch NSAA-Beurteilung in Woche 12.","language":28,"languageDescription":"German (Belgium)"}]},{"id":933638,"number":5,"endPoint":"Changes from baseline in SV95C at Week 12","isPrimary":false,"endPointTranslations":[{"id":9737201,"uuid":"c17b2d7f-0a79-4011-967d-3f27212d1e35","attributeTranslation":"Veranderingen ten opzichte van de nulmeting voor SV95C in Week 12.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737202,"uuid":"c17b2d7f-0a79-4011-967d-3f27212d1e35","attributeTranslation":"Variaciones respecto al valor basal en SV95C en la semana 12.","language":7,"languageDescription":"Spanish"},{"id":9737203,"uuid":"c17b2d7f-0a79-4011-967d-3f27212d1e35","attributeTranslation":"Veränderungen des SV95C-Werts gegenüber der Baseline in Woche 12.","language":28,"languageDescription":"German (Belgium)"},{"id":9737200,"uuid":"c17b2d7f-0a79-4011-967d-3f27212d1e35","attributeTranslation":"Zmiany względem wartości wyjściowej w SV95C w 12. tygodniu.","language":19,"languageDescription":"Polish"},{"id":9737199,"uuid":"c17b2d7f-0a79-4011-967d-3f27212d1e35","attributeTranslation":"Les changements par rapport à la valeur de début d’étude dans le SV95C à la semaine 12.","language":33,"languageDescription":"French (Belgium)"}]},{"id":933639,"number":6,"endPoint":"Exploratory end point: change from baseline in inflammatory cytokine profile at Week 12","isPrimary":false,"endPointTranslations":[{"id":9737205,"uuid":"de2f2e77-efab-4283-be69-dd712635b558","attributeTranslation":"Verkennend eindpunt: verandering ten opzichte van de nulmeting voor inflammatoir cytokineprofiel in Week 12.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737207,"uuid":"de2f2e77-efab-4283-be69-dd712635b558","attributeTranslation":"Eksploracyjny punkt końcowy: Zmianę względem wartości wyjściowej w profilu cytokin zapalnych w 12. tygodniu.","language":19,"languageDescription":"Polish"},{"id":9737208,"uuid":"de2f2e77-efab-4283-be69-dd712635b558","attributeTranslation":"Critère d’évaluation exploratoire: le changement par rapport à la valeur de début d’étude dans le profil des cytokines \ninflammatoires à la semaine 12.","language":33,"languageDescription":"French (Belgium)"},{"id":9737204,"uuid":"de2f2e77-efab-4283-be69-dd712635b558","attributeTranslation":"Explorativer Endpunkt: veränderung des entzündlichen Zytokinprofils in Woche 12 gegenüber der Baseline.","language":28,"languageDescription":"German (Belgium)"},{"id":9737206,"uuid":"de2f2e77-efab-4283-be69-dd712635b558","attributeTranslation":"Criterio de valoración exploratoria: variación respecto al valor basal en perfil de citoquinas inflamatorias en la semana 12.","language":7,"languageDescription":"Spanish"}]},{"id":933640,"number":7,"endPoint":"Exploratory end point: change from baseline in creatine kinase at Week 12","isPrimary":false,"endPointTranslations":[{"id":9737209,"uuid":"15724a1c-6ff6-4da2-aaad-a3338eba6dba","attributeTranslation":"Criterio de valoración exploratoria: variación respecto al valor basal en la creatina cinasa en la semana 12.","language":7,"languageDescription":"Spanish"},{"id":9737210,"uuid":"15724a1c-6ff6-4da2-aaad-a3338eba6dba","attributeTranslation":"Critère d’évaluation exploratoire: le changement par rapport à la valeur de début d’étude de la créatine kinase à la semaine 12.","language":33,"languageDescription":"French (Belgium)"},{"id":9737213,"uuid":"15724a1c-6ff6-4da2-aaad-a3338eba6dba","attributeTranslation":"Explorativer Endpunkt: veränderung der Kreatinkinase in Woche 12 gegenüber der Baseline.","language":28,"languageDescription":"German (Belgium)"},{"id":9737212,"uuid":"15724a1c-6ff6-4da2-aaad-a3338eba6dba","attributeTranslation":"Verkennend eindpunt: verandering ten opzichte van de nulmeting voor creatinekinase in Week 12.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737211,"uuid":"15724a1c-6ff6-4da2-aaad-a3338eba6dba","attributeTranslation":"Eksploracyjny punkt końcowy: Zmianę względem wartości wyjściowej poziomu kinazy kreatynowej w 12. tygodniu.","language":19,"languageDescription":"Polish"}]},{"id":933641,"number":8,"endPoint":"Exploratory end point: change from baseline in maximum percent predicted forced vial capacity as measured by spirometry at Week 12","isPrimary":false,"endPointTranslations":[{"id":9737217,"uuid":"6345ae9b-4d30-4f9d-92d0-b229ca4f58b9","attributeTranslation":"Explorativer Endpunkt: veränderung gegenüber der Baseline der maximalen prognostizierten forcierten Vitalkapazität in Prozent, gemessen durch Spirometrie in Woche 12.","language":28,"languageDescription":"German (Belgium)"},{"id":9737216,"uuid":"6345ae9b-4d30-4f9d-92d0-b229ca4f58b9","attributeTranslation":"Eksploracyjny punkt końcowy: Zmianę względem wartości wyjściowej maksymalnej procentowej przewidywanej \nwymuszonej pojemności życiowej (FVC), mierzonej za pomocą spirometrii, w 12. tygodniu.","language":19,"languageDescription":"Polish"},{"id":9737214,"uuid":"6345ae9b-4d30-4f9d-92d0-b229ca4f58b9","attributeTranslation":"Verkennend eindpunt: Verandering ten opzichte van de nulmeting voor het maximale percentage voorspelde \ngeforceerde vitale capaciteit zoals gemeten met spirometrie in Week 12.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737215,"uuid":"6345ae9b-4d30-4f9d-92d0-b229ca4f58b9","attributeTranslation":"Critère 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737218,"uuid":"6345ae9b-4d30-4f9d-92d0-b229ca4f58b9","attributeTranslation":"Criterio de valoración exploratoria: variación respecto al valor basal en el porcentaje máximo previsto de capacidad vital forzada medido por espirometría en la semana 12.","language":7,"languageDescription":"Spanish"}]},{"id":933642,"number":9,"endPoint":"Exploratory end point: change in biceps brachii muscle fiber size and fiber size distribution as determined from histopathology at 12 weeks","isPrimary":false,"endPointTranslations":[{"id":9737219,"uuid":"8dafafda-e529-4c90-a446-f8425f61fc2f","attributeTranslation":"Criterio 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.","language":7,"languageDescription":"Spanish"},{"id":9737221,"uuid":"8dafafda-e529-4c90-a446-f8425f61fc2f","attributeTranslation":"Critère 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.","language":33,"languageDescription":"French (Belgium)"},{"id":9737222,"uuid":"8dafafda-e529-4c90-a446-f8425f61fc2f","attributeTranslation":"Verkennend eindpunt: verandering in omvang en verdeling van spiervezels van de biceps brachii zoals bepaald op \ngrond van histopathologie na 12 weken.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737223,"uuid":"8dafafda-e529-4c90-a446-f8425f61fc2f","attributeTranslation":"Explorativer Endpunkt: veränderung der Muskelfasergröße und Fasergrößenverteilung des Musculus biceps brachii, ermittelt durch Histopathologie nach 12 Wochen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737220,"uuid":"8dafafda-e529-4c90-a446-f8425f61fc2f","attributeTranslation":"Eksploracyjny punkt końcowy: Zmianę wielkości włókien mięśniowych mięśnia dwugłowego ramienia oraz rozkładu ich wielkości, określoną na podstawie badania histopatologicznego po 12 tygodniach.","language":19,"languageDescription":"Polish"}]},{"id":933643,"number":10,"endPoint":"Exploratory end point: change in the proportion of embryonic myosin positive fibers as determined from histopathology at 12 weeks.","isPrimary":false,"endPointTranslations":[{"id":9737225,"uuid":"7f3b32a5-0be3-4ead-acc3-a4c3335716ca","attributeTranslation":"Criterio de valoración exploratoria: variación en la proporción de fibras positivas a la miosina embrionaria determinado por histopatología al cabo de 12 semanas.","language":7,"languageDescription":"Spanish"},{"id":9737226,"uuid":"7f3b32a5-0be3-4ead-acc3-a4c3335716ca","attributeTranslation":"Explorativer Endpunkt: veränderung des Anteils der embryonalen Myosin-positiven Fasern, ermittelt durch Histopathologie nach 12 Wochen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737224,"uuid":"7f3b32a5-0be3-4ead-acc3-a4c3335716ca","attributeTranslation":"Eksploracyjny punkt końcowy: Zmianę odsetka włókien dodatnich względem zarodkowej miozyny, określoną na podstawie badania histopatologicznego w 12. tygodniu.","language":19,"languageDescription":"Polish"},{"id":9737228,"uuid":"7f3b32a5-0be3-4ead-acc3-a4c3335716ca","attributeTranslation":"Critère d’évaluation exploratoire: le changement dans la proportion de fibres embryonnaires positives à la myosine, déterminé par l’histopathologie à 12 semaines.","language":33,"languageDescription":"French (Belgium)"},{"id":9737227,"uuid":"7f3b32a5-0be3-4ead-acc3-a4c3335716ca","attributeTranslation":"Verkennend eindpunt: verandering in het aandeel van embryonale myosine-positieve vezels zoals bepaald op grond \nvan histopathologie na 12 weken.","language":36,"languageDescription":"Dutch (Belgium)"}]},{"id":933644,"number":11,"endPoint":"Exploratory end point: change in the number of satellite cells as determined from histopathology at 12 weeks","isPrimary":false,"endPointTranslations":[{"id":9737229,"uuid":"169f8f53-e9e2-41c4-9ff1-cb42ed222dcb","attributeTranslation":"Verkennend eindpunt: verandering in het aantal satellietcellen zoals bepaald op grond van histopathologie na 12 \nweken.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737233,"uuid":"169f8f53-e9e2-41c4-9ff1-cb42ed222dcb","attributeTranslation":"Explorativer Endpunkt;  veränderung der Anzahl der Satellitenzellen, ermittelt durch Histopathologie nach 12 Wochen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737230,"uuid":"169f8f53-e9e2-41c4-9ff1-cb42ed222dcb","attributeTranslation":"Critère d’évaluation exploratoire: le changement dans le nombre de cellules satellites tel que déterminé par l’histopathologie à 12 semaines.","language":33,"languageDescription":"French (Belgium)"},{"id":9737232,"uuid":"169f8f53-e9e2-41c4-9ff1-cb42ed222dcb","attributeTranslation":"Eksploracyjny punkt końcowy: Zmianę liczby komórek satelitarnych, określoną na podstawie badania histopatologicznego po 12 tygodniach.","language":19,"languageDescription":"Polish"},{"id":9737231,"uuid":"169f8f53-e9e2-41c4-9ff1-cb42ed222dcb","attributeTranslation":"Criterio de valoración exploratoria: variación en el número de células satélite determinado por histopatología al cabo de 12 semanas.","language":7,"languageDescription":"Spanish"}]},{"id":933645,"number":12,"endPoint":"Exploratory end point: change in endomysial fibrosis and adipose tissue infiltration as determined from histopathology at 12 weeks.","isPrimary":false,"endPointTranslations":[{"id":9737235,"uuid":"8029651e-b04f-483d-9869-d2b8c257db1d","attributeTranslation":"Critère d’évaluation exploratoire: le changement de la fibrose endomysiale et de l’infiltration du tissu adipeux, déterminées par histopathologie à 12 semaines.","language":33,"languageDescription":"French (Belgium)"},{"id":9737236,"uuid":"8029651e-b04f-483d-9869-d2b8c257db1d","attributeTranslation":"Explorativer Endpunkt: veränderung der endomysialen Fibrose und der Fettgewebsinfiltration, ermittelt durch Histopathologie nach 12 Wochen.","language":28,"languageDescription":"German (Belgium)"},{"id":9737237,"uuid":"8029651e-b04f-483d-9869-d2b8c257db1d","attributeTranslation":"Criterio de valoración exploratoria: variación en la fibrosis endomisial y la infiltración de tejido adiposo determinado por histopatología al cabo de 12 semanas.","language":7,"languageDescription":"Spanish"},{"id":9737234,"uuid":"8029651e-b04f-483d-9869-d2b8c257db1d","attributeTranslation":"Verkennend eindpunt: Verandering in endomysiale fibrose en infiltratie van vetweefsel zoals bepaald op grond van \nhistopathologie na 12 weken.","language":36,"languageDescription":"Dutch (Belgium)"},{"id":9737238,"uuid":"8029651e-b04f-483d-9869-d2b8c257db1d","attributeTranslation":"Eksploracyjny punkt końcowy: Zmianę stopnia zwłóknienia śródmięśniowego oraz nacieku tkanki tłuszczowej, określoną na podstawie badania histopatologicznego po 12 tygodniach.","language":19,"languageDescription":"Polish"}]},{"id":933646,"number":13,"endPoint":"Exploratory end point: change in NSAA score over 12 weeks as compared to natural history","isPrimary":false,"endPointTranslations":[{"id":9737242,"uuid":"6f017bf0-6459-4749-871c-3a1058d18206","attributeTranslation":"Critère d’évaluation exploratoire: le changement du score NSAA sur 12 semaines par rapport à 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Z O.O.","substanceOrigin":"Structurally Diverse Substance - Cell therapy","actSubstOrigin":"Structurally Diverse Substance - Cell therapy","actSubstName":"EX VIVO FUSED NORMAL ALLOGENEIC HUMAN MYOBLAST WITH AUTOLOGOUS HUMAN MYOBLAST DERIVED FROM DUCHENNE MUSCULAR DYSTROPHY AFFECTED DONOR","substanceEvCode":"SUB193214","synonyms":["MB(N)/MB(DMD) DEC","MBN/MBDMD Dystrophin Expressing Chimeric Cell"]}],"activeSubstanceOtherDescriptiveName":""},"isPaediatricFormulation":false,"mpRoleInTrial":"1","orphanDrugEdit":true,"orphanDrugDesigNumber":"EU/3/18/2089","otherMedicinalProduct":"tissue engineered product","evCode":"PRD11689940","therapies":[{"id":6424,"catReferenceNumber":"EMA/H0005097","advancedTherapyType":"2","cellOriginCode":"2","cellTypeCode":"3","cellTypeMoreInformation":"Chimeric cells produced by ex vivo fusion of normal allogeneic human myoblast (MBN) with autologous human myoblast derived from Duchenne muscular dystrophy affected donor (MBDMD)"}],"devices":[],"characteristics":["11"],"routes":["INTRAOSSEOUS USE"],"allSubstancesChemicals":false,"productName":"DT-DEC01","jsonActiveSubstanceNames":"ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from duchenne muscular dystrophy affected donor","pharmaceuticalFormDisplay":"SUSPENSION FOR INJECTION"}],"trialDetails":{"clinicalTrialIdentifiers":{"fullTitle":"A PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY","fullTitleTranslations":[{"id":10140958,"uuid":"519494b6-2a69-4d1c-b35d-7852eb380f34","attributeTranslation":"BADANIE KLINICZNE FAZY 1/2 (OTWARTE, EKSPLORACYJNE) OCENIAJĄCE BEZPIECZEŃSTWO I SKUTECZNOŚĆ TERAPII DT-DEC01 U PACJENTÓW Z DYSTROFIĄ MIĘŚNIOWĄ DUCHENNE’A","language":19,"languageDescription":"Polish"}],"publicTitle":"A PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY","publicTitleTranslations":[{"id":10140967,"uuid":"039937f0-c441-4350-9521-34d6325e1cfb","attributeTranslation":"BADANIE KLINICZNE FAZY 1/2 (OTWARTE, EKSPLORACYJNE) OCENIAJĄCE BEZPIECZEŃSTWO I SKUTECZNOŚĆ TERAPII DT-DEC01 U PACJENTÓW Z DYSTROFIĄ MIĘŚNIOWĄ DUCHENNE’A","language":19,"languageDescription":"Polish"}],"shortTitle":"DT-DEC01-DMD-CT-1/2","secondaryIdentifyingNumbers":{"additionalRegistries":[]}},"trialInformation":{"trialCategory":{"isLowIntervention":false,"trialPhase":"7","trialCategory":"2","justificationForTrialCategory":"This 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.","trialCategoryId":112550},"medicalCondition":{"partIMedicalConditions":[{"id":127684,"medicalCondition":"Duchenne Muscular Dystrophy","medicalConditionTranslations":[],"isConditionRareDisease":true}],"meddraConditionTerms":[{"termId":100000012544,"version":"20.0","level":"PT","termName":"Duchenne muscular dystrophy","classificationCode":"10013801","organClass":100000004850,"active":false}]},"trialObjective":{"trialScopes":[{"code":"4","trialScopeId":371587},{"code":"5","trialScopeId":371588}],"mainObjective":"Phase 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.\n\nPhase 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.","mainObjectiveTranslations":[{"id":10140966,"uuid":"ade61a1a-1ec7-4b42-99e5-b98675fdb2e6","attributeTranslation":"Główne 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.\n\nGłówne 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.","language":19,"languageDescription":"Polish"}],"secondaryObjectives":[]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":826458,"number":1,"principalInclusionCriteria":"Subject 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.","principalInclusionCriteriaTranslations":[{"id":10140959,"uuid":"3b35d593-a777-46c8-b511-2db8d4febe75","attributeTranslation":"Uczestnik 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.","language":19,"languageDescription":"Polish"}]},{"id":826459,"number":2,"principalInclusionCriteria":"Boys of age 5 to 18 years old (at the time of screening), diagnosed with DMD confirmed by genetic testing.","principalInclusionCriteriaTranslations":[{"id":10140960,"uuid":"f9ca6a7f-b2c4-4cc0-8e45-1c4d7aa1b914","attributeTranslation":"Chłopcy w wieku od 5 do 18 lat (w momencie wizyty przesiewowej), u których zdiagnozowano dystrofię mięśniową Duchenne’a potwierdzoną badaniami genetycznymi.","language":19,"languageDescription":"Polish"}]},{"id":826460,"number":3,"principalInclusionCriteria":"Subjects must be receiving glucocorticosteroids for a minimum of 6 months prior to the biopsy of muscle tissue.","principalInclusionCriteriaTranslations":[{"id":10140961,"uuid":"8c004b04-b75a-44b6-918e-84e36a5e1944","attributeTranslation":"Uczestnicy muszą przyjmować glikokortykosteroidy przez co najmniej 6 miesięcy przed biopsją tkanki mięśniowej.","language":19,"languageDescription":"Polish"}]},{"id":826461,"number":4,"principalInclusionCriteria":"Subjects with progressive, symmetrical proximal muscle weakness of arms and legs.","principalInclusionCriteriaTranslations":[{"id":10140962,"uuid":"0e91c130-6a9b-4b1d-aed3-87aa1ce64767","attributeTranslation":"Uczestnicy wykazują postępującą i symetryczną utratę siły mięśniowej w odcinkach proksymalnych kończyn górnych i dolnych.","language":19,"languageDescription":"Polish"}]},{"id":826462,"number":5,"principalInclusionCriteria":"Willingness 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.","principalInclusionCriteriaTranslations":[{"id":10140963,"uuid":"f4a1536d-69a4-4807-9f4e-3234dd68edf0","attributeTranslation":"Uczestnicy 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.","language":19,"languageDescription":"Polish"}]},{"id":826463,"number":6,"principalInclusionCriteria":"Willingness to use acceptable forms of contraception if the subject is sexually active.","principalInclusionCriteriaTranslations":[{"id":10140964,"uuid":"bbc613e4-0832-47c5-bce1-e1a13b121c8f","attributeTranslation":"Uczestnicy wykazują gotowość do stosowania akceptowalnych metod antykoncepcji, jeśli są aktywni seksualnie.","language":19,"languageDescription":"Polish"}]},{"id":826464,"number":7,"principalInclusionCriteria":"Patients 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).","principalInclusionCriteriaTranslations":[{"id":10140965,"uuid":"b5981b9a-4442-498a-a65f-4c73f4d1a4f3","attributeTranslation":"Pacjenci muszą pomyślnie przejść kwalifikację anestezjologiczną do pobrania biopsji tkanki mięśniowej i zabiegu doszpikowego podania DT-DEC01, które zostaną wykonane w znieczuleniu (miejscowym/ogólnym/anagosedacji).","language":19,"languageDescription":"Polish"}]}],"principalExclusionCriteria":[{"id":1414115,"number":1,"principalExclusionCriteria":"Subject 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.","principalExclusionCriteriaTranslations":[{"id":10140944,"uuid":"90299eb3-a665-472c-8b9f-efdaf2fc43b6","attributeTranslation":"Uczestnika poddano uprzednio leczeniu badanym produktem leczniczym, podobną terapią eksperymentalną z zastosowaniem ATMP lub innym produktem opartym na komórkach, terapią genową lub lekiem translarna/ataluren.","language":19,"languageDescription":"Polish"}]},{"id":1414116,"number":10,"principalExclusionCriteria":"Any 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.","principalExclusionCriteriaTranslations":[{"id":10140945,"uuid":"d8a67b6e-8861-4a7a-8b74-c43c296cb17d","attributeTranslation":"U 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.","language":19,"languageDescription":"Polish"}]},{"id":1414117,"number":11,"principalExclusionCriteria":"Presence or history of other musculoskeletal or neurologic disease or somatic disorder not related to DMD including pulmonary and cardiac disease.","principalExclusionCriteriaTranslations":[{"id":10140946,"uuid":"cc3ea729-02df-4ce8-a867-417e64caadc0","attributeTranslation":"U uczestnika stwierdzono obecność lub historię innych chorób układu mięśniowo-szkieletowego, neurologicznych lub zaburzeń somatycznych niezwiązanych z DMD, w tym chorób płuc i serca.","language":19,"languageDescription":"Polish"}]},{"id":1414118,"number":12,"principalExclusionCriteria":"History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of the investigational product.","principalExclusionCriteriaTranslations":[{"id":10140947,"uuid":"6a83bfb3-bd31-405a-beaa-09330565a87f","attributeTranslation":"W przeszłości wystąpiła u uczestnika reakcja alergiczna lub anafilaktyczna na białko terapeutyczne lub diagnostyczne lub na substancje dodatkowe badanego produktu leczniczego.","language":19,"languageDescription":"Polish"}]},{"id":1414119,"number":13,"principalExclusionCriteria":"Ongoing 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.","principalExclusionCriteriaTranslations":[{"id":10140948,"uuid":"07caa099-d6f0-43b2-b0b4-75439636992c","attributeTranslation":"Uczestnik 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.","language":19,"languageDescription":"Polish"}]},{"id":1414120,"number":14,"principalExclusionCriteria":"Other 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.","principalExclusionCriteriaTranslations":[{"id":10140949,"uuid":"081743c5-7f57-410f-8767-993bc4199de3","attributeTranslation":"U 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.","language":19,"languageDescription":"Polish"}]},{"id":1414121,"number":2,"principalExclusionCriteria":"Subject has known history of immune reaction to the administered therapies or history of GvHD.","principalExclusionCriteriaTranslations":[{"id":10140950,"uuid":"26501da5-9f16-4f43-9fbe-a04e80d36e66","attributeTranslation":"W przeszłości wystąpiła u uczestnika reakcja immunologiczna na podany lek lub GvHD.","language":19,"languageDescription":"Polish"}]},{"id":1414122,"number":3,"principalExclusionCriteria":"Subjects with known cognitive impairment or behavioral issues that would impede the ability to follow instructions.","principalExclusionCriteriaTranslations":[{"id":10140951,"uuid":"58fd6d91-5c23-4a77-9b8c-a96f47c6a06d","attributeTranslation":"U uczestnika stwierdzono zaburzenia sfery poznawczej lub problemy z zachowaniem, które upośledzałyby zdolność do wykonywania poleceń.","language":19,"languageDescription":"Polish"}]},{"id":1414123,"number":4,"principalExclusionCriteria":"Subject 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.","principalExclusionCriteriaTranslations":[{"id":10140952,"uuid":"4d6200e9-c1e5-4be5-ab75-30fc3826b7c9","attributeTranslation":"U 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.","language":19,"languageDescription":"Polish"}]},{"id":1414124,"number":5,"principalExclusionCriteria":"Subject has a history of any autoimmune disease.","principalExclusionCriteriaTranslations":[{"id":10140953,"uuid":"8eedb6da-7215-4c09-a910-063dee4ebe4f","attributeTranslation":"W przeszłości wystąpiła u uczestnika choroba o podłożu autoimmunologicznym.","language":19,"languageDescription":"Polish"}]},{"id":1414125,"number":6,"principalExclusionCriteria":"Ongoing participation in any other therapeutic clinical trial.","principalExclusionCriteriaTranslations":[{"id":10140954,"uuid":"b471eb33-bf21-4019-b350-a8576a148c59","attributeTranslation":"Uczestnik aktualnie bierze udział w jakimkolwiek innym badaniu klinicznym o charakterze terapeutycznym.","language":19,"languageDescription":"Polish"}]},{"id":1414126,"number":7,"principalExclusionCriteria":"Presence of pre-existing antibodies in the subject’s serum against the donor lymphocytes.","principalExclusionCriteriaTranslations":[{"id":10140955,"uuid":"713a920f-0358-459d-b750-b59454b94e3d","attributeTranslation":"W surowicy uczestnika wykryto obecność przeciwciał skierowanych przeciwko limfocytom dawcy.","language":19,"languageDescription":"Polish"}]},{"id":1414127,"number":8,"principalExclusionCriteria":"Subject has undergone an organ or bone marrow transplantation.","principalExclusionCriteriaTranslations":[{"id":10140956,"uuid":"3b8e8dcf-1563-4b49-a573-69f842d15efb","attributeTranslation":"Uczestnik przeszedł przeszczep narządu lub szpiku kostnego.","language":19,"languageDescription":"Polish"}]},{"id":1414128,"number":9,"principalExclusionCriteria":"Change 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.","principalExclusionCriteriaTranslations":[{"id":10140957,"uuid":"0692df7f-da04-47c9-a3cd-3e88429a9747","attributeTranslation":"U 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.","language":19,"languageDescription":"Polish"}]}]},"endPoint":{"primaryEndPoints":[{"id":968447,"number":1,"endPoint":"Phase 1: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI.","isPrimary":true,"endPointTranslations":[{"id":10140935,"uuid":"7f451d23-719b-4146-a089-8c5d3b9cb628","attributeTranslation":"Faza 1: Częstość występowania i ciężkość wszystkich zdarzeń niepożądanych: zdarzeń niepożądanych (Adverse Events, AE), ciężkich zdarzeń niepożądanych (Serious Adverse Events, SAE) i zdarzeń 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51","language":19,"languageDescription":"Polish"}],"publicTitle":"A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy","publicTitleTranslations":[{"id":10124244,"uuid":"5d8daa20-b6cb-4f68-a8c4-937d5cb6984a","attributeTranslation":"Estudio de investigación para comparar diferentes dosis de un nuevo medicamento en investigación para el tratamiento de determinados pacientes con distrofia muscular de Duchenne","language":7,"languageDescription":"Spanish"},{"id":10124245,"uuid":"5d8daa20-b6cb-4f68-a8c4-937d5cb6984a","attributeTranslation":"Kutatási vizsgálat egy új vizsgálati készítmény különböző dózisainak összehasonlítására egyes Duchenne-izomdisztrófiás betegek kezelésére.","language":13,"languageDescription":"Hungarian"},{"id":10124242,"uuid":"5d8daa20-b6cb-4f68-a8c4-937d5cb6984a","attributeTranslation":"Badanie naukowe mające na celu porównanie różnych dawek nowego badanego produktu leczniczego w leczeniu niektórych pacjentów z dystrofią mięśniową Duchenne’a","language":19,"languageDescription":"Polish"},{"id":10124241,"uuid":"5d8daa20-b6cb-4f68-a8c4-937d5cb6984a","attributeTranslation":"Μία ερευνητική μελέτη για τη σύγκριση διαφορετικών δόσεων ενός νέου ερευνητικού φαρμακευτικού προϊόντος για τη θεραπεία ορισμένων ασθενών με μυϊκή δυστροφία Duchenne","language":32,"languageDescription":"Greek (Greece)"},{"id":10124243,"uuid":"5d8daa20-b6cb-4f68-a8c4-937d5cb6984a","attributeTranslation":"Een onderzoek voor het vergelijken van verschillende doseringen van een nieuw geneesmiddel voor de behandeling van bepaalde patiënten met Duchenne spierdystrofie","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10124246,"uuid":"5d8daa20-b6cb-4f68-a8c4-937d5cb6984a","attributeTranslation":"Étude 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.","language":34,"languageDescription":"French (France)"}],"shortTitle":"4658-402","secondaryIdentifyingNumbers":{"nctNumber":{"id":505193,"number":"NCT03992430"},"additionalRegistries":[]}},"trialInformation":{"trialCategory":{"isLowIntervention":false,"trialPhase":"5","trialCategory":"2","justificationForTrialCategory":"According 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.","trialCategoryId":112363},"medicalCondition":{"partIMedicalConditions":[{"id":127490,"medicalCondition":"Duchenne Muscular Dystrophy","medicalConditionTranslations":[{"id":10124167,"uuid":"230b573c-4a66-41e3-980a-3321b9056151","attributeTranslation":"Dystrophie Musculaire de Duchenne","language":10,"languageDescription":"French"},{"id":10124163,"uuid":"230b573c-4a66-41e3-980a-3321b9056151","attributeTranslation":"Μυϊκή Δυστροφία Duchenne","language":5,"languageDescription":"Greek"},{"id":10124164,"uuid":"230b573c-4a66-41e3-980a-3321b9056151","attributeTranslation":"Duchenne-féle izomdystrophia","language":13,"languageDescription":"Hungarian"},{"id":10124165,"uuid":"230b573c-4a66-41e3-980a-3321b9056151","attributeTranslation":"Duchenne spierdystrofie","language":18,"languageDescription":"Dutch"},{"id":10124168,"uuid":"230b573c-4a66-41e3-980a-3321b9056151","attributeTranslation":"Distrofia Muscular de Duchenne","language":7,"languageDescription":"Spanish"},{"id":10124166,"uuid":"230b573c-4a66-41e3-980a-3321b9056151","attributeTranslation":"Dystrofią mięśniowa Duchenne’a","language":19,"languageDescription":"Polish"}],"isConditionRareDisease":true}],"meddraConditionTerms":[{"termId":100000012544,"version":"20.0","level":"PT","termName":"Duchenne muscular dystrophy","classificationCode":"10013801","organClass":100000004850,"active":false}]},"trialObjective":{"trialScopes":[{"code":"3","trialScopeId":371058},{"code":"7","trialScopeId":371060},{"code":"13","otherDescription":"The open-label period of the study evaluates tolerability of higher dose eteplirsen in DMD patients","trialScopeId":371056},{"code":"5","trialScopeId":371059},{"code":"9","trialScopeId":371061},{"code":"6","trialScopeId":371057}],"mainObjective":"Open-Label Dose Escalation:\nEvaluate the safety and tolerability of weekly IV doses of 100 and 200 mg/kg of eteplirsen.\n\nDouble-blind Dose Finding and Dose Comparison 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51\n•\tÉvaluer des doses plus élevées d’eteplirsen (100 mg/kg et 200 mg/kg) pour la comparaison de doses","language":34,"languageDescription":"French (France)"},{"id":10124240,"uuid":"d30d817c-a7f5-4a81-a5dc-53203a1949b3","attributeTranslation":"Aumento escalonado de la dosis en régimen abierto:\n•\tEvaluar la seguridad y la tolerabilidad de dosis IV semanales de 100 mg/kg y 200 mg/kg de eteplirsén.\n\nParte en régimen doble ciego de búsqueda y comparación de la dosis:\n•\tInvestigar 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.\n•\tEvaluar dosis más altas de eteplirsén (100 mg/kg y 200 mg/kg) para comparar las dosis.","language":7,"languageDescription":"Spanish"},{"id":10124235,"uuid":"d30d817c-a7f5-4a81-a5dc-53203a1949b3","attributeTranslation":"Nyílt elrendezésű dóziseszkaláció:\nAz eteplirszen heti 100 és 200 mg/kg-os iv. adagja biztonságosságának és tolerálhatóságának kiértékelése\nKettős-vak dóziskereső és dózis-összehasonlító rész:\n• 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\n• Az eteplirszen nagyobb adagjainak (100 mg/kg és 200 mg/kg) dózis-összehasonító kiértékelése","language":13,"languageDescription":"Hungarian"},{"id":10124238,"uuid":"d30d817c-a7f5-4a81-a5dc-53203a1949b3","attributeTranslation":"Eskalacja dawki prowadzona metodą otwartej próby:\nOcena bezpieczeństwa i tolerancji cotygodniowych dawek dożylnych 100 mg/kg mc. i 200 mg/kg mc. eteplirsenu \n\nCzęść badania prowadzona metodą podwójnie ślepej próby mająca na celu ustalenie optymalnej dawki oraz porównanie dawek:\n•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 \n• Ocena wysokich dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) w celu ich porównania","language":19,"languageDescription":"Polish"},{"id":10124237,"uuid":"d30d817c-a7f5-4a81-a5dc-53203a1949b3","attributeTranslation":"Κλιμάκωσης δόσης ανοικτής επισήμανση:\nΑξιολόγηση της ασφάλειας και ανεκτικότητας των εβδομαδιαίων ΕΦ δόσεων 100 και 200 mg/kg του eteplirsen\n\nΔιπλά τυφλό μέρος προσδιορισμού της δόσης και σύγκρισης της δόσης:\n•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\n•Αξιολόγηση υψηλότερων δόσεων του eteplirsen (100 mg/kg και 200 mg/kg) για σύγκριση των δόσεων","language":32,"languageDescription":"Greek (Greece)"},{"id":10124236,"uuid":"d30d817c-a7f5-4a81-a5dc-53203a1949b3","attributeTranslation":"Open-label dosisescalatie:\nHet beoordelen van de veiligheid en verdraagbaarheid van wekelijkse intraveneuze doses van 100 en 200 mg/kg eteplirsen.\n\nDubbelblind deel voor dosisbepaling en dosisvergelijking:\n• 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\n• Het beoordelen van hogere doses eteplirsen (100 mg/kg en 200 mg/kg) voor dosisvergelijking","language":37,"languageDescription":"Dutch (Netherlands)"}],"secondaryObjectives":[{"id":431648,"number":1,"secondaryObjective":"Double-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 IV","secondaryObjectiveTranslations":[{"id":10124247,"uuid":"bb3293b1-c950-424d-b0c5-68fff941e457","attributeTranslation":"Część badania prowadzona metodą podwójnie ślepej próby mająca na celu ustalenie optymalnej dawki oraz porównanie dawek:\n•Ocena wpływu wysokich dawek eteplirsenu w porównaniu z dawką 30 mg/kg mc., podawanej dożylnie raz na tydzień na:  \no sprawność poruszania się \no czynność płuc \no ekspresję dystrofiny \n•Ocena farmakokinetyki (PK) i farmakodynamiki (PD) dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) wyższych niż 30 mg/kg mc.  \n•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 tydzień","language":19,"languageDescription":"Polish"},{"id":10124248,"uuid":"bb3293b1-c950-424d-b0c5-68fff941e457","attributeTranslation":"Parte en régimen doble ciego de búsqueda y comparación de la dosis:\n•\tEvaluar 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: \no\tRendimiento ambulatorio\no\tFunción pulmonar\no\tExpresión de distrofina\n•\tEvaluar la FC y la FD de dosis superiores a 30 mg/kg (100 mg/kg y 200 mg/kg) de eteplirsén\nEvaluar 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 IV","language":7,"languageDescription":"Spanish"},{"id":10124249,"uuid":"bb3293b1-c950-424d-b0c5-68fff941e457","attributeTranslation":"Partie de détermination et comparaison de doses en double aveugle\n• \tÉvaluer l’effet de doses élevées par rapport à une dose IV hebdomadaire de 30 mg/kg d’eteplirsen sur les critères suivants:\no\tPerformances ambulatoires\no\tFonction pulmonaire\no\tExpression de la dystrophine\n• \tÉvaluer la PK et la PD de doses d’eteplirsen (100 mg/kg et 200 mg/kg)supérieures à 30 mg/kg\n•\tÉvaluer 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 semaine","language":34,"languageDescription":"French (France)"},{"id":10124250,"uuid":"bb3293b1-c950-424d-b0c5-68fff941e457","attributeTranslation":"Kettős-vak dóziskereső és dózis-összehasonlító rész\n•\tAz eteplirszen heti egyszer iv. alkalmazott nagy adagja által a következőkre kifejtett hatás kiértékelése, a 30 mg/kg-os adaggal összehasonlítva:\no\tjárás-teljesítőképesség\no\tlégzésfunkció\no\tdisztrofinexpresszió\n•\tAz eteplirszen 30 mg/kg-nál nagyobb adagjai (100 mg/kg és 200 mg/kg) farmakokinetikájának és farmakodinamikájának elemzése.\n•\tAz eteplirszen 30 mg/kg-nál nagyobb (100 mg/kg vagy 200 mg/kg), heti egyszeri iv. adagjai biztonságosságának és tolerálhatóságának kiértékelése","language":13,"languageDescription":"Hungarian"},{"id":10124252,"uuid":"bb3293b1-c950-424d-b0c5-68fff941e457","attributeTranslation":"Dubbelblind deel voor dosisbepaling en dosisvergelijking:\n• Het beoordelen van het effect van hoge doses in vergelijking met 30 mg/kg eteplirsen, wekelijks intraveneus toegediend, op\no Ambulante prestaties o Longfunctie\no Dystrofine-expressie\n• Het beoordelen van de farmacokinetiek en farmacodynamiek van doses (100 mg/kg en 200 mg/kg) hoger dan 30 mg/kg eteplirsen\n• Het beoordelen van de veiligheid en verdraagbaarheid van doses (100 mg/kg en 200 mg/kg) hoger dan 30 mg/kg eteplirsen, wekelijks intraveneus toegediend","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10124251,"uuid":"bb3293b1-c950-424d-b0c5-68fff941e457","attributeTranslation":"Διπλά τυφλό μέρος προσδιορισμού της δόσης και σύγκρισης της δόσης:\n•Αξιολόγηση της επίδρασης των υψηλών δόσεων σε σύγκριση με 30 mg/kg eteplirsen, χορηγούμενου εβδομαδιαίως ΕΦ, ως προς τα εξής:\noΠεριπατητική απόδοση\noΠνευμονική λειτουργία\noΈκφραση δυστροφίνης\n•Αξιολόγηση της ΦΚ και ΦΔ δόσεων (100 mg/kg και 200 mg/kg) του eteplirsen που υπερβαίνουν τα 30 mg/kg\n•Αξιολόγηση της ασφάλειας και της ανεκτικότητας δόσεων (100 mg/kg και 200 mg/kg) του eteplirsen που υπερβαίνουν τα 30 mg/kg, χορηγούμενων ΕΦ εβδομαδιαίως","language":32,"languageDescription":"Greek (Greece)"}]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":825244,"number":1,"principalInclusionCriteria":"1. 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.","principalInclusionCriteriaTranslations":[{"id":10124230,"uuid":"7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0","attributeTranslation":"1.\tFiúknak 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;\n2.\t4 és 13 éves kor között kell lenniük\n3.\tJáróképesnek kell lenniük, képesnek kell lenniük ≤10 másodperc alatt teljesíteni a talajról való felállás tesztet\n4.\tKépes önállóan, segédeszközök nélkül járni.\n5.\tÉp 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)\n6.\tA 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).\n7.\t7 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.\n8.\tHa 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).\n9.\tVan(nak) szülő(k) vagy törvényes gyám(ok), aki(k) képes(ek) megérteni és betartani a vizsgálat összes követelményét.\n10.\tHajlandó 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.","language":13,"languageDescription":"Hungarian"},{"id":10124229,"uuid":"7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0","attributeTranslation":"1. 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).\n2. Wiek od 4 do 13 lat włącznie.\n3. Pacjent chodzący, zdolny do wykonania testu TTRISE (tj. czas do wstania) w czasie maksymalnie 10 sekund w momencie wizyty przesiewowej.\n4. Zdolność samodzielnego chodzenia bez urządzeń wspomagających.\n5. 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).\n6. 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]).\n7. 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.\nPacjenci w wieku od 4 do 6 lat – brak konieczności wspomagania oddychania respiratorem ani stosowania wentylacji nieinwazyjnej w czasie badania przesiewowego.\n8. 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).\n9. Dostępność rodzica/rodziców lub opiekuna prawnego/opiekunów prawnych, którzy są w stanie zrozumieć wszystkie wymogi związane z badaniem i ich przestrzegać.\n10. 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.","language":19,"languageDescription":"Polish"},{"id":10124233,"uuid":"7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0","attributeTranslation":"1.\tDebe 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.\n2.\tTener ente 4 y 13 años, ambos incluidos.\n3.\tSer capaz de levantarse del suelo en ≤10 segundos o menos, en el momento de la visita de sección\n4.\t Ser capaz de caminar de forma independiente sin dispositivos de ayuda.\n5.\tTener 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). \n6.\tHaber recibido una dosis estable o una dosis equivalente de corticosteroides orales durante un mínimo de 12 semanas antes de la aleatorización; \n7.\tSi 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.\n8.\tSi 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).\n9.\tDebe tener uno o más padres o tutores legales que pueden comprender y cumplir con todos los requisitos del estudio.\n10.\t 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.","language":7,"languageDescription":"Spanish"},{"id":10124232,"uuid":"7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0","attributeTranslation":"1. 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).\n2. Να είναι ηλικίας 4 έως 13 ετών.\n3. Να είναι περιπατητικός ασθενής, ικανός να πετύχει TTRISE σε 10 δευτερόλεπτα ή λιγότερο κατά την επίσκεψη διαλογής.\n4. Να έχει δυνατότητα ανεξάρτητης βάδισης χωρίς βοηθητικές συσκευές.\n5. 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).\n6. 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.\n7. 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.\n8. 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).\n9. Να έχει γονείς ή νόμιμους κηδεμόνες που είναι σε θέση να κατανοήσουν και να συμμορφωθούν με όλες τις απαιτήσεις της μελέτης.\n10. 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.","language":32,"languageDescription":"Greek (Greece)"},{"id":10124231,"uuid":"7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0","attributeTranslation":"1. 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).\n2. Van 4 tot en met 13 jaar oud\n3. Ambulante patiënt, in staat om TTRISE in 10 seconden of minder uit te voeren op het moment van het screeningbezoek.\n4. Kan zelfstandig lopen zonder hulpmiddelen.\n5. 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).\n6. 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]).\n7. 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.\nVoor kinderen van 4 tot 6 jaar: heeft geen ondersteuning van beademingsapparatuur of niet-invasieve beademing nodig op het moment van screening.\n8. 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).\n9. Heeft (een) ouder(s) of wettelijke voogd(en) die in staat is (zijn) om alle onderzoeksvereisten te begrijpen en eraan te voldoen.\n10. 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.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10124234,"uuid":"7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0","attributeTranslation":"1. 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).\n2.Être âgé de 4 à 13 ans inclus.\n3. Patient ambulatoire, capable d'effectuer le TTRISE en 10 secondes ou moins au moment de la visite de sélection.\n4. Capable de marcher de façon indépendante, sans dispositifs d’assistance.\n5. 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).\n6. 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.\n7. 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.\n8. 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).\n9. 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.\n10.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.","language":34,"languageDescription":"French (France)"}]}],"principalExclusionCriteria":[{"id":1411961,"number":1,"principalExclusionCriteria":"1. 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.","principalExclusionCriteriaTranslations":[{"id":10124217,"uuid":"5f2989e9-d92b-4a4e-a7cc-73a708a40b67","attributeTranslation":"1. 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.\n2. Huidige of eerdere behandeling met een andere experimentele farmacologische behandeling voor DMD of eerdere blootstelling aan antisense-oligonucleotide, gentherapie of genbewerking, behalve de volgende:\nEzutromid toegediend ten minste 12 weken voorafgaand aan de eerste dosis.\nDrisapersen 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).\nTamoxifen toegediend ten minste 4 weken voorafgaand aan de eerste dosis.\n3. 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.\n4. Aanwezigheid van een andere significante neuromusculaire of genetische ziekte anders dan DMD (bijv. dwerggroei).\n5. Gamma-glutamyltranspeptidase (GGT) > 3 × de bovengrens van normaal (ULN) of serumbilirubine > ULN, niet te verklaren door het syndroom van Gilbert.\n6. 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\n7. Bloedplaatjestelling < de ondergrens van normaal.\n8. Aanwezigheid van andere klinisch significante ziekten, waaronder significante hart-, long-, lever-, nier-, hematologische, immunologische of gedragsziekten of maligniteit.\n9. 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.\n10. 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.\n11. Bekende overgevoeligheid voor eteplirsen of voor een van de hulpstoffen van eteplirsen.\n12. Is naar het oordeel van de onderzoeker niet in staat of niet bereid om zich aan de onderzoeksprocedures te houden.","language":18,"languageDescription":"Dutch"},{"id":10124218,"uuid":"5f2989e9-d92b-4a4e-a7cc-73a708a40b67","attributeTranslation":"1. 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.\n2. Jelenlegi vagy korábbi kezelés bármely más kísérleti farmakológiai kezeléssel a DMD kezelésére, vagy bármilyen korábbi expozíció antisense oligonukleotiddal, génterápiával vagy génszerkesztéssel; kivéve a következőket:\nAz első adagot megelőzően legalább 12 héttel beadott Ezutromid.\nDrisapersen, 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).\n3. Nagyobb műtét a randomizálást megelőző 3 hónapon belül, vagy a vizsgálat során bármikor tervezett műtét, kivéve a protokoll által meghatározott, engedélyezett műtétet, ha alkalmazható.\n4. Bármilyen jelentős neuromuszkuláris vagy genetikai betegség jelenléte a DMD-n kívül (pl. törpeség).\n5. Gamma-glutamil-transzpeptidáz (GGT) > 3 × a normálérték felső határa (ULN) vagy szérum bilirubin > ULN, amelyet nem magyaráz meg Gilbert-szindróma.\n6. 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\n7. Trombocitaszám < a normálérték alsó határa.\n8. Egyéb klinikailag jelentős betegség jelenléte, beleértve a jelentős szív-, tüdő-, máj-, vese-, hematológiai, immunológiai vagy viselkedési betegséget vagy malignitást.\n9. 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.\n10. 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.\n11. Ismert túlérzékenység az eteplirszennel vagy az eteplirszén bármely segédanyagával szemben.\n12. A vizsgáló véleménye szerint nem képes vagy nem hajlandó megfelelni a vizsgálati eljárásoknak.","language":13,"languageDescription":"Hungarian"},{"id":10124222,"uuid":"5f2989e9-d92b-4a4e-a7cc-73a708a40b67","attributeTranslation":"1. 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.\n2. 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:\nEzutromid podawany co najmniej 12 tygodni przed pierwszą dawką.\nDrisapersen 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).\nTamoksyfen podawany co najmniej 4 tygodnie przed pierwszą dawką.\n3. 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.\n4. Występowanie jakiejkolwiek istotnej choroby nerwowo-mięśniowej lub genetycznej innej niż DMD (np. karłowatości).\n5. Aktywność gamma-glutamylotranspeptydazy (GGTP) > 3 × górnej granicy normy (GGN) lub stężenie bilirubiny w surowicy > GGN, którego nie można wyjaśnić występowaniem zespołu Gilberta.\n6. 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.\n7. Liczba płytek krwi < dolnej granicy normy.\n8. Występowanie innej, klinicznie istotnej choroby, w tym istotnej choroby serca, płuc, wątroby, nerek, choroby hematologicznej, immunologicznej lub behawioralnej bądź nowotworu złośliwego.\n9. 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).\n10. 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.\n11. Stwierdzona nadwrażliwość na eteplirsen lub którąkolwiek substancję pomocniczą eteplirsenu.\n12. Brak możliwości lub chęci przestrzegania przez pacjenta procedur badania w opinii badacza.","language":19,"languageDescription":"Polish"},{"id":10124219,"uuid":"5f2989e9-d92b-4a4e-a7cc-73a708a40b67","attributeTranslation":"1.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.\n2.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:\n•Ezutromid χορηγούμενο τουλάχιστον 12 εβδομάδες πριν από την πρώτη δόση.\n•Drisapersen χορηγούμενο τουλάχιστον 36 εβδομάδες πριν από την πρώτη δόση.\n•Suvodirsen χορηγούμενο τουλάχιστον 12 εβδομάδες πριν από την πρώτη δόση.\n•Vamorolone χορηγούμενο τουλάχιστον 12 εβδομάδες πριν από την πρώτη δόση.\n•Eteplirsen (προηγούμενη ή τρέχουσα χρήση).\n•Ταμοξιφαίνη χορηγούμενη τουλάχιστον 4 εβδομάδες πριν από την πρώτη δόση.\n3.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.\n4.Παρουσία οποιασδήποτε σημαντικής νευρομυϊκής ή γενετικής νόσου εκτός της DMD (π.χ. νανισμός).\n5.γ-γλουταμυλ-τρανσπεπτιδάση [GGT] >3 × το ανώτατο φυσιολογικό όριο (ULN) ή χολερυθρίνη ορού >ULN η οποία δεν εξηγείται από το σύνδρομο Gilbert.\n6.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).\n7.Αριθμός αιμοπεταλίων < το κατώτερο φυσιολογικό όριο.\n8.Παρουσία άλλης κλινικά σημαντικής νόσου, συμπεριλαμβανομένης σημαντικής καρδιακής, πνευμονικής, ηπατικής, νεφρικής, αιματολογικής, ανοσολογικής ή συμπεριφορικής νόσου ή κακοήθειας.\n9.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.\n10.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.\n11.Γνωστή υπερευαισθησία στο eteplirsen ή σε κάποιο από τα έκδοχα του eteplirsen.\n12.Είναι, κατά τη γνώμη του Ερευνητή, ανίκανος ή απρόθυμος να συμμορφωθεί με τις διαδικασίες της μελέτης.","language":5,"languageDescription":"Greek"},{"id":10124221,"uuid":"5f2989e9-d92b-4a4e-a7cc-73a708a40b67","attributeTranslation":"1. 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.\n2.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 :\n•\tEzutromid administré au moins 12 semaines avant la première dose. \n•\tDrisapersen administré au moins 36 semaines avant la première dose.\n•\tSuvodirsen administré au moins 12 semaines avant la première dose.\n•\tVamorolone administré au moins 12 semaines avant la première dose.\n•\tEteplirsen (utilisation antérieure ou actuelle).\n•\tTamoxifène administré au moins 4 semaines avant la première dose.\n3. 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.\n4.Présence d'une maladie neuromusculaire ou génétique importante autre que la DMD (par exemple, nanisme).\n5.Gamma-glutamyl transpeptidase [GGT] > 3 fois la limite supérieure de la normale (LSN) ou bilirubine sérique > LSN inexpliquée par le syndrome de Gilbert.\n6.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.\n7.Numération plaquettaire < la limite inférieure de la normale\n8.Présence d'une autre maladie cliniquement significative, y compris une maladie cardiaque, pulmonaire, hépatique, rénale, hématologique, immunologique, comportementale ou une tumeur maligne.\n9.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.\n10.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.\n11.Hypersensibilité connue à l'eteplirsen ou à l'un des excipients de l'eteplirsen.\n12.Est, de l'avis de l'investigateur, incapable ou réticent à se conformer aux procédures de l'étude.\nLes patients qui ne remplissent pas les critères d'éligibilité peuvent faire l'objet d'une nouvelle sélection à la discrétion de l'investigateur.","language":10,"languageDescription":"French"},{"id":10124220,"uuid":"5f2989e9-d92b-4a4e-a7cc-73a708a40b67","attributeTranslation":"1.\tUso 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.\n2.\tTratamiento 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:\nEzutromid administrado al menos 12 semanas antes de la primera dosis.\nDrisapersen 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)\nTamoxifeno administrado al menos 4 semanas antes de la primera dosis.\n3. 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.\n4. Presencia de cualquier enfermedad neuromuscular o genética significativa distinta de la DMD (p. ej., enanismo).\n5. Gamma-glutamil transpeptidasa (GGT) > 3 × el límite superior de lo normal (LSN) o bilirrubina sérica > LSN no explicada por el síndrome de Gilbert.\n6. 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\n7. Recuento de plaquetas < el límite inferior de lo normal.\n8. Presencia de otra enfermedad clínicamente significativa, incluidas enfermedades cardíacas, pulmonares, hepáticas, renales, hematológicas, inmunológicas o conductuales significativas o neoplasias malignas.\n9. 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.\n10. 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.\n11. Hipersensibilidad conocida al eteplirsen o a cualquiera de sus excipientes.\n12. Es, en opinión del investigador, incapaz o no está dispuesto a cumplir con los procedimientos del estudio.","language":7,"languageDescription":"Spanish"}]}]},"endPoint":{"primaryEndPoints":[{"id":966850,"number":1,"endPoint":"Open-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).","isPrimary":true,"endPointTranslations":[{"id":10124173,"uuid":"93f6e994-fe81-42e0-b4c1-4b9f2ac1a18e","attributeTranslation":"Eskalacja dawki prowadzona metodą otwartej próby:\nCzęstość 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)","language":19,"languageDescription":"Polish"},{"id":10124169,"uuid":"93f6e994-fe81-42e0-b4c1-4b9f2ac1a18e","attributeTranslation":"Aumento escalonado de la dosis en régimen abierto:\n\nIncidencia de EA, Incidencia de eventos adversos de interés especial (AESI)\nCambios anormales respecto del valor inicial o empeoramiento de los signos\nvitales o hallazgos del examen físico, Incidencia de EAG, Evaluaciones de\nseguridad de laboratorio, Electrocardiogramas (ECG) y Ecocardiogramas\n(ECO).","language":7,"languageDescription":"Spanish"},{"id":10124171,"uuid":"93f6e994-fe81-42e0-b4c1-4b9f2ac1a18e","attributeTranslation":"Nyílt elrendezésű dóziseszkaláció:\nA 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).","language":13,"languageDescription":"Hungarian"},{"id":10124170,"uuid":"93f6e994-fe81-42e0-b4c1-4b9f2ac1a18e","attributeTranslation":"Open-label dosisverhoging:\nIncidentie 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).","language":18,"languageDescription":"Dutch"},{"id":10124174,"uuid":"93f6e994-fe81-42e0-b4c1-4b9f2ac1a18e","attributeTranslation":"Augmentation de dose en ouvert\nIncidence 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).","language":10,"languageDescription":"French"},{"id":10124172,"uuid":"93f6e994-fe81-42e0-b4c1-4b9f2ac1a18e","attributeTranslation":"Μέρος κλιμάκωσης δόσης ανοικτής επισήμανσης\nΕπίπτωση των ΑΣ, Επίπτωση ανεπιθύμητων συμβάντων ειδικού ενδιαφέροντος (AESI), \nΠαθολογικές μεταβολές από την Έναρξη ή επιδείνωση ζωτικών σημείων ή ευρημάτων κλινικής εξέτασης, Επίπτωση των ΣΑΣ, Εργαστηριακές αξιολογήσεις της ασφάλειας, \nΗλεκτροκαρδιογραφήματα (ΗΚΓ), Ηχοκαρδιογραφήματα (ECHO)","language":5,"languageDescription":"Greek"}]},{"id":966851,"number":2,"endPoint":"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)","isPrimary":true,"endPointTranslations":[{"id":10124179,"uuid":"f9258092-d9e7-4cca-878e-484c98ffba06","attributeTranslation":"Dubbelblind deel voor dosisbepaling en dosisvergelijking:\n-Verandering ten opzichte van baseline in week 72 of week 96 in de North Star Ambulatory Assessment (NSAA)-totaalscore (voor voorwaardelijke tussentijdse werkzaamheidsanalyse)\n-Verandering ten opzichte van baseline in week 144 in de NSAA-totaalscore (voor uiteindelijke analyse)","language":18,"languageDescription":"Dutch"},{"id":10124176,"uuid":"f9258092-d9e7-4cca-878e-484c98ffba06","attributeTranslation":"Διπλά τυφλό μέρος προσδιορισμού της δόσης και σύγκρισης της δόσης:\n•Μεταβολή από την Έναρξη την Εβδομάδα 72 ή την Εβδομάδα 96 στη συνολική βαθμολογία NSAA (για ενδιάμεση ανάλυση αποτελεσματικότητας υπό όρους)\n•Μεταβολή από την Έναρξη την Εβδομάδα 144 στη συνολική βαθμολογία","language":5,"languageDescription":"Greek"},{"id":10124180,"uuid":"f9258092-d9e7-4cca-878e-484c98ffba06","attributeTranslation":"Kettős-vak dóziskereső és dózis-összehasonlító rész:\n-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)\n-Az NSAA összpontszámának változása a kiindulás és a 144. hét között (a végleges elemzéshez)","language":13,"languageDescription":"Hungarian"},{"id":10124177,"uuid":"f9258092-d9e7-4cca-878e-484c98ffba06","attributeTranslation":"Część badania prowadzona metodą podwójnie ślepej próby mająca na celu ustalenie optymalnej dawki oraz porównanie dawek:\n- 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)\n- Zmiana wartości całkowitego wyniku w skali NSAA od punktu wyjściowego w 144. tygodniu (do analizy końcowej)","language":19,"languageDescription":"Polish"},{"id":10124175,"uuid":"f9258092-d9e7-4cca-878e-484c98ffba06","attributeTranslation":"Partie de détermination et comparaison de doses en double aveugle\n•\tÉvolution 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)\n•\tÉvolution par rapport aux valeurs de référence à la semaine 144 du score global NSAA (pour analyse finale)","language":10,"languageDescription":"French"},{"id":10124178,"uuid":"f9258092-d9e7-4cca-878e-484c98ffba06","attributeTranslation":"Parte en régimen doble ciego de búsqueda y comparación de la dosis:\n\n- 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)\n-Cambio con respecto al valor inicial en la semana 144 en la puntuación total de la NSAA (para el análisis final)","language":7,"languageDescription":"Spanish"}]}],"secondaryEndPoints":[{"id":966852,"number":1,"endPoint":"• 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)","isPrimary":false,"endPointTranslations":[{"id":10124183,"uuid":"ca98c0a4-b59d-49c1-9463-d2f8b563a29d","attributeTranslation":"• Cambio con respecto al valor inicial en la semana 144 en \no Tiempo para levantarse (TTRISE) desde el piso \no Tiempo para caminar/correr 10 metros\no Prueba de caminata de 6 minutos (6MWT)\no Prueba de ascenso de escaleras cronometrada de 4 pasos\no Porcentaje de capacidad vital forzada prevista\n(FVC%p)","language":7,"languageDescription":"Spanish"},{"id":10124181,"uuid":"ca98c0a4-b59d-49c1-9463-d2f8b563a29d","attributeTranslation":"• Verandering ten opzichte van baseline in week 144 in o Tijd om op te staan (TTRISE) van de vloer o 10-meter-loop-/rentijd\no 6-minuten-looptest (6MWT)\no Getimede 4-treden-traptest\no Voorspeld percentage geforceerde vitale longcapaciteit (FVC%p)\"","language":18,"languageDescription":"Dutch"},{"id":10124185,"uuid":"ca98c0a4-b59d-49c1-9463-d2f8b563a29d","attributeTranslation":"Évolution observée à la semaine 144 par rapport aux valeurs de référence:\no\tTemps pour se relever du sol\no\tChronométrage de la marche/course sur 10 mètres\no\tTest de marche de 6 minutes(6MWT)\no\tTest chronométré de montée d’escalier sur 4 marches\no \tTaux de déclin annuel de la capacité vitale forcée (CVF) (%) prédite","language":10,"languageDescription":"French"},{"id":10124186,"uuid":"ca98c0a4-b59d-49c1-9463-d2f8b563a29d","attributeTranslation":"Változás a kiindulási értékhez képest a 144. héten:\no\ta talajról történő felálláshoz szükséges idő (TTRISE)\no\t10 méteres járás/futás ideje\no\t6 perces járásvizsgálat (6MWT)\no\t4 lépcsőn fellépdelés időméréses vizsgálata\no\tforszírozott vitálkapacitás, a becsült érték százalékában (FVC%p)","language":13,"languageDescription":"Hungarian"},{"id":10124184,"uuid":"ca98c0a4-b59d-49c1-9463-d2f8b563a29d","attributeTranslation":"•\tZmiana w 144. tygodniu w stosunku do punktu wyjściowego w zakresie poniższych wartości: \no\tCzas potrzebny do wstania (TTRISE) z podłogi \no\tTest 10-metrowego marszu/biegu\no\tTest 6-minutowego marszu (6MWT)\no\tTest wchodzenia po schodach (4 stopnie) z pomiarem czasu\no\tbadania procentowej przewidywanej natężonej pojemności życiowej (FVC%p)","language":19,"languageDescription":"Polish"},{"id":10124182,"uuid":"ca98c0a4-b59d-49c1-9463-d2f8b563a29d","attributeTranslation":"•Μεταβολή από την Έναρξη την Εβδομάδα 144, ως προς τα εξής:\noTTRISE από το δάπεδο\noΧρόνος κάλυψης απόστασης 10 μέτρων με περπάτημα/τρέξιμο\no6MWT\noΧρονομετρημένη δοκιμασία ανάβασης 4 σκαλοπατιών\noΠροβλεπόμενο ποσοστό βίαιης ζωτικής χωρητικότητας (FVC%p)\"","language":5,"languageDescription":"Greek"}]},{"id":966853,"number":2,"endPoint":"• Time to Loss of Ambulation (LOA) through Week 144","isPrimary":false,"endPointTranslations":[{"id":10124189,"uuid":"4d982e92-5019-49d9-83f4-afcc87f20b29","attributeTranslation":"Durée jusqu’à la perte de la marche jusqu’à la semaine 144","language":10,"languageDescription":"French"},{"id":10124190,"uuid":"4d982e92-5019-49d9-83f4-afcc87f20b29","attributeTranslation":"• Tijd tot verlies van loopvermogen (LOA) tot week 144","language":18,"languageDescription":"Dutch"},{"id":10124191,"uuid":"4d982e92-5019-49d9-83f4-afcc87f20b29","attributeTranslation":"• Tiempo transcurrido hasta la pérdida de la deambulación (LOA)\nhasta la semana 144","language":7,"languageDescription":"Spanish"},{"id":10124187,"uuid":"4d982e92-5019-49d9-83f4-afcc87f20b29","attributeTranslation":"•\tCzas do utraty zdolności poruszania się (LOA) do tygodnia 144.","language":19,"languageDescription":"Polish"},{"id":10124188,"uuid":"4d982e92-5019-49d9-83f4-afcc87f20b29","attributeTranslation":"A járóképesség elvesztéséig eltelt idő (LOA) a 144. hétig","language":13,"languageDescription":"Hungarian"},{"id":10124192,"uuid":"4d982e92-5019-49d9-83f4-afcc87f20b29","attributeTranslation":"•Χρόνος έως την LOA έως την Εβδομάδα 144","language":5,"languageDescription":"Greek"}]},{"id":966854,"number":3,"endPoint":"• 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","isPrimary":false,"endPointTranslations":[{"id":10124194,"uuid":"729283fd-ea51-4eff-83b1-d30d067a8bfc","attributeTranslation":"A vázizmok disztrofinexpressziójának változása a kiindulás és a 24., 48. vagy 144. héten, a következő vizsgálatok alapján:\no\tWestern blot (mennyiségi meghatározás)\no\timmunhisztokémia (IHC) – rostintenzitás immunfluoreszcenciával\no\taz exonátugrás mennyiségi meghatározása ún. droplet digitális polimeráz láncreakcióval (ddPCR)\no\tA disztrofin-pozitív rostok százalékos aránya (PDPF) immunfluoreszcenciával","language":13,"languageDescription":"Hungarian"},{"id":10124196,"uuid":"729283fd-ea51-4eff-83b1-d30d067a8bfc","attributeTranslation":"•Μεταβολή από την Έναρξη την Εβδομάδα 24, την Εβδομάδα 48 ή την Εβδομάδα 144 στην έκφραση της δυστροφίνης των σκελετικών μυών μετρημένη με:\noΜέθοδο ανοσοαποτύπωσης Western (ποσοτικοποίηση)\noΈνταση ινών IHC μέσω δοκιμασίας ανοσοφθορισμού\noΠοσοτικοποίηση παράλειψης εξονίων μέσω ddPCR\noPDPF μέσω δοκιμασίας ανοσοφθορισμού","language":5,"languageDescription":"Greek"},{"id":10124197,"uuid":"729283fd-ea51-4eff-83b1-d30d067a8bfc","attributeTranslation":"Évolution 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:\no\tWestern blot (quantification)\no\tImmunohistochimie (IHC) (intensité des fibres par immunofluorescence)\no\tQuantification du saut d’exon par réaction en chaîne par polymérase (PCR)\no\tPourcentage de fibres positives à la dystrophine (PDPF) par immunofluorescence","language":10,"languageDescription":"French"},{"id":10124193,"uuid":"729283fd-ea51-4eff-83b1-d30d067a8bfc","attributeTranslation":"• Verandering ten opzichte van baseline in week 24, week 48 of week 144 in de expressie van skeletspierdystrofine door:\no Western blot (kwantificering)\no Immunohistochemie (IHC)-vezelintensiteit door immunofluorescentie\no Kwantificering van exon-skipping door middel van droplet digital-polymerasekettingreactie (ddPCR)\no Percentage dystrofine-positieve vezels (PDPF) door immunofluorescentie","language":18,"languageDescription":"Dutch"},{"id":10124195,"uuid":"729283fd-ea51-4eff-83b1-d30d067a8bfc","attributeTranslation":"•\tVariación 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:\no\tInmunoelectrotransferencia (cuantificación)\no\tIntensidad de las fibras mediante inmunohistoquímica (IHQ) mediante inmunofluorescencia\no\tCuantificación del salto de exones mediante reacción en cadena de la polimerasa digital en nanogotas (ddPCR)\no\tPorcentaje de fibras positivas para distrofina (PDPF) por inmunofluorescencia","language":7,"languageDescription":"Spanish"},{"id":10124198,"uuid":"729283fd-ea51-4eff-83b1-d30d067a8bfc","attributeTranslation":"•\tZmiana w stosunku do punktu wyjściowego w tygodniu 24., 48. lub 144. w ekspresji dystrofiny w mięśniach szkieletowych na podstawie oznaczenia:\no\tWestern blot (oznaczenie ilościowe)\no\tintensywności włókien w badaniu immunohistochemicznym (IHC) w teście immunofluorescencyjnym\no\tOznaczenie ilościowe z pominięciem egzonu metodą emulsyjnej reakcji łańcuchowej polimerazy (ddPCR)\no\tOdsetek włókien dystrofino-dodatnich (PDPF) w teście immunofluorescencyjnym","language":19,"languageDescription":"Polish"}]},{"id":966855,"number":4,"endPoint":"PK parameters (plasma and muscle biopsy)","isPrimary":false,"endPointTranslations":[{"id":10124200,"uuid":"905c40ec-75d7-4984-89df-cd34aca38540","attributeTranslation":"•\tFarmakokinetikai (PK) paraméterek (plazma- és izombiopsziával)","language":13,"languageDescription":"Hungarian"},{"id":10124204,"uuid":"905c40ec-75d7-4984-89df-cd34aca38540","attributeTranslation":"•Παράμετροι ΦΚ (πλάσματος και με μυϊκή βιοψία)","language":5,"languageDescription":"Greek"},{"id":10124199,"uuid":"905c40ec-75d7-4984-89df-cd34aca38540","attributeTranslation":"Paramètres PK (plasma et biopsie musculaire)","language":10,"languageDescription":"French"},{"id":10124203,"uuid":"905c40ec-75d7-4984-89df-cd34aca38540","attributeTranslation":"•\tParámetros FC (en plasma y biopsia de músculo)","language":7,"languageDescription":"Spanish"},{"id":10124202,"uuid":"905c40ec-75d7-4984-89df-cd34aca38540","attributeTranslation":"PK-parameters (plasma- en spierbiopsie)","language":18,"languageDescription":"Dutch"},{"id":10124201,"uuid":"905c40ec-75d7-4984-89df-cd34aca38540","attributeTranslation":"•\tParametry PK (osocze i biopsja tkanki mięśniowej)","language":19,"languageDescription":"Polish"}]},{"id":966856,"number":5,"endPoint":"• Incidence of AEs • Incidence of AESIs • Incidence of SAEs • Safety laboratory assessments","isPrimary":false,"endPointTranslations":[{"id":10124208,"uuid":"e8d5ce8d-375c-4838-98c4-fa444e213cfa","attributeTranslation":"•Επίπτωση των ΑΣ\n•Επίπτωση των AESI\n•Επίπτωση των ΣΑΣ\n•Εργαστηριακές αξιολογήσεις της ασφάλειας","language":5,"languageDescription":"Greek"},{"id":10124207,"uuid":"e8d5ce8d-375c-4838-98c4-fa444e213cfa","attributeTranslation":"•\tIncidencia de AA\n•\tIncidencia de AAIE\n•\tIncidencia de AAG\n•\tEvaluaciones analíticas de seguridad","language":7,"languageDescription":"Spanish"},{"id":10124209,"uuid":"e8d5ce8d-375c-4838-98c4-fa444e213cfa","attributeTranslation":"•\tAE-k előfordulása\n•\tAz AESI-k előfordulása\n•\tSAE-k előfordulása\n•\tBiztonsági laboratóriumi értékelések","language":13,"languageDescription":"Hungarian"},{"id":10124206,"uuid":"e8d5ce8d-375c-4838-98c4-fa444e213cfa","attributeTranslation":"•Incidence des EI\n• Incidence des EIIS\n• Incidence des EIG\n• Analyses de laboratoire de sécurité","language":10,"languageDescription":"French"},{"id":10124205,"uuid":"e8d5ce8d-375c-4838-98c4-fa444e213cfa","attributeTranslation":"•\tCzęstość występowania zdarzeń niepożądanych\n•\tCzęstość występowania zdarzeń niepożądanych o szczególnym znaczeniu (AESI)\n•\tCzęstość występowania ciężkich zdarzeń niepożądanych (SAE)\n•\tOceny laboratoryjne dotyczące bezpieczeństwa","language":19,"languageDescription":"Polish"},{"id":10124210,"uuid":"e8d5ce8d-375c-4838-98c4-fa444e213cfa","attributeTranslation":"• Incidentie van AE’s\n• Incidentie van AESI’s\n• Incidentie van SAE’s\n• Laboratoriumbeoordelingen voor de veiligheid","language":18,"languageDescription":"Dutch"}]},{"id":966857,"number":6,"endPoint":"• ECGs • ECHO • Abnormal changes from Baseline or worsening of vital signs and physical examination findings","isPrimary":false,"endPointTranslations":[{"id":10124211,"uuid":"7b0ecf78-8c4b-4012-b063-876b928eb522","attributeTranslation":"•\tECG\n•\tEcocardiograma\n•\tVariaciones anormales con respecto al inicio o empeoramiento de los hallazgos de las constantes vitales y la exploración física","language":7,"languageDescription":"Spanish"},{"id":10124215,"uuid":"7b0ecf78-8c4b-4012-b063-876b928eb522","attributeTranslation":"•ΗΚΓ\n•ECHO\n•Παθολογικές μεταβολές από την Έναρξη ή επιδείνωση ζωτικών σημείων και ευρημάτων κλινικής εξέτασης","language":5,"languageDescription":"Greek"},{"id":10124214,"uuid":"7b0ecf78-8c4b-4012-b063-876b928eb522","attributeTranslation":"•      ECG\n•\tECHO\n•\tÉvolutions anormales par rapport aux valeurs de référence ou aggravation des signes vitaux et des résultats de l’examen physique","language":10,"languageDescription":"French"},{"id":10124212,"uuid":"7b0ecf78-8c4b-4012-b063-876b928eb522","attributeTranslation":"• ECG’s\n• ECHO\n• Abnormale veranderingen ten opzichte van baseline of verslechtering van de vitale functies en bevindingen bij lichamelijk onderzoek","language":18,"languageDescription":"Dutch"},{"id":10124216,"uuid":"7b0ecf78-8c4b-4012-b063-876b928eb522","attributeTranslation":"•\tEKG-k\n•\tECHO\n•\tKóros változások a kiindulási értékhez képest vagy a vitális jelek és a fizikális vizsgálati leletek romlása","language":13,"languageDescription":"Hungarian"},{"id":10124213,"uuid":"7b0ecf78-8c4b-4012-b063-876b928eb522","attributeTranslation":"• EKG\n• ECHO\n• Nieprawidłowe zmiany w stosunku do wartości wyjściowej lub pogorszenie parametrów życiowych i wyników badania fizykalnego","language":19,"languageDescription":"Polish"}]}]},"trialDuration":{"estimatedGlobalEndDate":"2026-10-31","estimatedEndDate":"2026-10-31","estimatedRecruitmentStartDate":"2022-05-31"},"sourceOfMonetarySupport":[{"id":89435,"organisationName":"Sarepta Therapeutics, 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(Netherlands)"}],"isConditionRareDisease":true}]},"trialObjective":{"trialScopes":[{"code":"6","trialScopeId":232162},{"code":"13","otherDescription":"Tolerability","trialScopeId":232161},{"code":"7","trialScopeId":232164},{"code":"9","trialScopeId":232165},{"code":"3","trialScopeId":232163},{"code":"4","trialScopeId":232166}],"mainObjective":"CORE PHASE: To assess the pharmacokinetic (PK) profile of givinostat at steady state administered in younger DMD boys in the 2 cohorts.\n\nEXTENSION PHASE: To assess the safety and tolerability of givinostat long-term administered in younger DMD boys in the 2 cohorts (ie, adverse events [AE])","mainObjectiveTranslations":[{"id":5856605,"uuid":"9c9f1692-eca0-4abf-aeb8-00a2a4a75924","attributeTranslation":"CORE PHASE: Het beoordelen van het farmacokinetische (PK) profiel van givinostat in stationaire toestand toegediend bij jongere jongens met DMD in de 2 cohorten.\n\nEXTENSION PHASE: Het beoordelen van de veiligheid en verdraagbaarheid van 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de 2 cohorten","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":262927,"number":3,"secondaryObjective":"CORE PHASE: To evaluate the effect of givinostat on functional health-related quality of life (HRQOL) in Cohort 1 (for subjects ≥4 years only)","secondaryObjectiveTranslations":[{"id":5856609,"uuid":"61b601dc-1608-4f2d-ae20-8502fd6b604b","attributeTranslation":"Het beoordelen van het effect van givinostat op de functionele gezondheidsgerelateerde kwaliteit van leven (HRQOL) in cohort 1 (alleen voor proefpersonen ≥ 4 jaar)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":262928,"number":4,"secondaryObjective":"EXTENSION 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.","secondaryObjectiveTranslations":[{"id":5856610,"uuid":"c2a5ff48-52d8-4ee4-a0ea-423918be7298","attributeTranslation":"Het 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])","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":262929,"number":5,"secondaryObjective":"EXTENSION PHASE: To evaluate the effect of long-term administered givinostat on muscular functional parameters in the 2 cohorts","secondaryObjectiveTranslations":[{"id":5856611,"uuid":"bf64c1b3-5072-4305-992e-9e4f86256549","attributeTranslation":"Het beoordelen van het effect van toediening op lange termijn van givinostat op spierfunctionele parameters in de 2 cohorten","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":262930,"number":6,"secondaryObjective":"EXTENSION PHASE: To evaluate the effect of long-term administered givinostat on functional HRQOL in Cohort 1 (for subjects >4 years only)","secondaryObjectiveTranslations":[{"id":5856612,"uuid":"2e76c2b9-572f-4692-9677-836d6bb6b5b1","attributeTranslation":"Het beoordelen van het effect van toediening op lange termijn van givinostat op de functionele HRQOL in cohort 1 (alleen voor proefpersonen > 4 jaar)","language":37,"languageDescription":"Dutch (Netherlands)"}]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":509722,"number":1,"principalInclusionCriteria":"CORE PHASE: Male children aged ≥2 to <6 years at screening (subjects ≥6 years of age at screening will not be enrolled into the study)","principalInclusionCriteriaTranslations":[{"id":5856598,"uuid":"b6639473-0fe3-4ea1-8ede-768bcaed5e50","attributeTranslation":"Mannelijke kinderen van ≥ 2 tot < 6 jaar bij de screening (proefpersonen ≥ 6 jaar bij de screening worden niet in het onderzoek ingeschreven)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":509723,"number":2,"principalInclusionCriteria":"CORE PHASE: Written consent provided by parent/legal guardian and subject written assent, if applicable (according to local regulation)","principalInclusionCriteriaTranslations":[{"id":5856599,"uuid":"0de7ae24-695a-4296-b238-28200cde8862","attributeTranslation":"Schriftelijke toestemming gegeven door ouder/wettelijke voogd en schriftelijke instemming door de proefpersoon, indien van toepassing (volgens lokale regelgeving)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":509724,"number":3,"principalInclusionCriteria":"CORE PHASE: A genetic diagnosis of DMD","principalInclusionCriteriaTranslations":[{"id":5856600,"uuid":"8c0be8b4-8a77-445e-be27-123e63e84ff4","attributeTranslation":"Een genetische diagnose van DMD hebben","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":509725,"number":4,"principalInclusionCriteria":"CORE PHASE: Corticosteroid treatment considerations: a.\tFor 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.\tFor subjects without current corticosteroids treatment: Must not start corticosteroids during core phase (ie, first 48 weeks)","principalInclusionCriteriaTranslations":[{"id":5856601,"uuid":"c3ade9c8-fa93-412f-a539-8f94ee13ab44","attributeTranslation":"Overwegingen voor behandeling met corticosteroïden:\na. Voor proefpersonen die een stabiele dosis orale systemische corticosteroïden krijgen:\nGeen 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\nof\nb. Voor proefpersonen zonder huidige corticosteroïdenbehandeling:\nMag niet beginnen met corticosteroïden tijdens de kernfase (d.w.z. eerste 48 weken)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":509726,"number":5,"principalInclusionCriteria":"EXTENSION PHASE: Must have participated in the Core Phase study (48 weeks) and have attended the End of Treatment Visit (EOT/V12)","principalInclusionCriteriaTranslations":[{"id":5856602,"uuid":"0b82ffef-3996-4424-ade4-42ddedb42a22","attributeTranslation":"Moet hebben deelgenomen aan het kernfaseonderzoek (48 weken) en hebben deelgenomen aan het bezoek voor einde van behandeling (EvB/B12)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":509727,"number":6,"principalInclusionCriteria":"EXTENSION  PHASE: Give informed consent and /or assent in writing signed by the parent/legal guardian and/or subject (according to local regulation)","principalInclusionCriteriaTranslations":[{"id":5856603,"uuid":"71181c3f-d4d0-4d85-88ea-a0946bfff725","attributeTranslation":"Schriftelijk toestemming en/of instemming geven ondertekend door de ouder/wettelijke vertegenwoordiger en/of proefpersoon (volgens lokale regelgeving)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":509728,"number":7,"principalInclusionCriteria":"EXTENSION  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.","principalInclusionCriteriaTranslations":[{"id":5856604,"uuid":"b0180ae3-4060-4455-928e-62176446910a","attributeTranslation":"Bij 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.","language":37,"languageDescription":"Dutch (Netherlands)"}]}],"principalExclusionCriteria":[{"id":878505,"number":1,"principalExclusionCriteria":"CORE PHASE: Exposure to any other investigational drug within 3 months prior to the start of study drug","principalExclusionCriteriaTranslations":[{"id":5856574,"uuid":"22a1cd75-49c9-46f6-b521-d7648e1e3c05","attributeTranslation":"Blootstelling aan een ander onderzoeksmiddel binnen 3 maanden voorafgaand aan de start van het onderzoeksmiddel","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878506,"number":10,"principalExclusionCriteria":"EXTENSION 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 disease","principalExclusionCriteriaTranslations":[{"id":5856575,"uuid":"13df6b28-bebe-4553-a061-93451c9cb19d","attributeTranslation":"Huidige 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 Gilbert","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878507,"number":11,"principalExclusionCriteria":"EXTENSION PHASE: Inadequate renal function, as defined by serum Cystatin C result >2 × ULN","principalExclusionCriteriaTranslations":[{"id":5856576,"uuid":"ab061dc7-7cfb-431c-b545-4111bae81799","attributeTranslation":"Onvoldoende 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)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878508,"number":12,"principalExclusionCriteria":"CORE PHASE: Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug","principalExclusionCriteriaTranslations":[{"id":5856577,"uuid":"259f4fd2-d6f5-41eb-9b3e-31ec719bbeda","attributeTranslation":"Blootstelling aan een dystrofine-herstellend product (bijv. ataluren, exon-skipping) binnen 6 maanden voorafgaand aan de start van het onderzoeksmiddel","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878509,"number":13,"principalExclusionCriteria":"EXTENSION PHASE: Fasting triglycerides >300 mg/dL (3.42 mmol/L)","principalExclusionCriteriaTranslations":[{"id":5856578,"uuid":"5b49230c-8ee5-4a4d-83f4-fa2292b5f189","attributeTranslation":"Nuchtere 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)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878510,"number":14,"principalExclusionCriteria":"EXTENSION 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 results","principalExclusionCriteriaTranslations":[{"id":5856579,"uuid":"4d17cae1-9fb8-48b0-b0a2-7548ced89066","attributeTranslation":"Aanwezigheid 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 belemmeren","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878511,"number":15,"principalExclusionCriteria":"EXTENSION 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 judgement","principalExclusionCriteriaTranslations":[{"id":5856580,"uuid":"bbd1be31-8ef7-4c31-a708-5109b162481c","attributeTranslation":"Bewijs 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.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878512,"number":16,"principalExclusionCriteria":"CORE PHASE: Received any gene therapy (eg, Adeno-associated viruses Micro dystrophin delivery) within 12 months prior to the start of study drug","principalExclusionCriteriaTranslations":[{"id":5856581,"uuid":"059ac38d-0df5-47dc-9803-833f3111d821","attributeTranslation":"Een gentherapie ontvangen (bijv. adeno-gerelateerde virusgemedieerde toediening van micro-dystrofine) binnen 12 maanden voorafgaand aan de start van het onderzoeksmiddel","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878513,"number":17,"principalExclusionCriteria":"CORE 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)","principalExclusionCriteriaTranslations":[{"id":5856582,"uuid":"814bbfa1-dcdc-4c83-bcc8-33e722389e02","attributeTranslation":"Gebruik 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 toegestaan","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878514,"number":18,"principalExclusionCriteria":"CORE 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 study","principalExclusionCriteriaTranslations":[{"id":5856583,"uuid":"2732df9d-7808-4c71-b159-77123801d01c","attributeTranslation":"Heeft 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 onderzoek","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878515,"number":19,"principalExclusionCriteria":"CORE 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 results","principalExclusionCriteriaTranslations":[{"id":5856584,"uuid":"91a2ea6b-114f-462a-b8db-c0fd46285042","attributeTranslation":"De 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 nageleefd","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878516,"number":2,"principalExclusionCriteria":"CORE 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)","principalExclusionCriteriaTranslations":[{"id":5856585,"uuid":"cdcc20f4-fa4f-42e4-8017-b12e558e1a35","attributeTranslation":"Onvoldoende 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)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878517,"number":20,"principalExclusionCriteria":"CORE 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 judgement","principalExclusionCriteriaTranslations":[{"id":5856586,"uuid":"cf903ed3-eda2-431c-9660-3ecd365d1713","attributeTranslation":"Diagnose van andere ongecontroleerde neurologische ziekten of aanwezigheid van relevante ongecontroleerde somatische aandoeningen die geen 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screening","principalExclusionCriteriaTranslations":[{"id":5856591,"uuid":"d76fabe1-c17b-4fce-b3a0-4af2c4d3e543","attributeTranslation":"Positieve test voor hepatitis B-oppervlakteantigeen, hepatitis C-virus antilichaamserologie of hiv bij de screening","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878523,"number":5,"principalExclusionCriteria":"CORE 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)","principalExclusionCriteriaTranslations":[{"id":5856592,"uuid":"568efb80-ea0a-4ff0-8cf8-8a38b82fc360","attributeTranslation":"Gecorrigeerd 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)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878524,"number":6,"principalExclusionCriteria":"CORE 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 judgement","principalExclusionCriteriaTranslations":[{"id":5856593,"uuid":"70ecfe29-624f-4044-9813-e0620f8e94f4","attributeTranslation":"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","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878525,"number":7,"principalExclusionCriteria":"CORE PHASE: Hypersensitivity to any component of the study drug","principalExclusionCriteriaTranslations":[{"id":5856594,"uuid":"a8201a2b-de37-4965-894c-e7ee14813c0b","attributeTranslation":"Overgevoeligheid voor een bestanddeel van het onderzoeksmiddel","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878526,"number":8,"principalExclusionCriteria":"CORE PHASE: Sorbitol intolerance or malabsorption, or have the hereditary form of fructose intolerance","principalExclusionCriteriaTranslations":[{"id":5856595,"uuid":"e4ad30e7-4c9e-4a16-a6f0-b8c578987ec1","attributeTranslation":"Intolerantie of malabsorptie van sorbitol, of heeft de erfelijke vorm van fructose-intolerantie","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":878527,"number":9,"principalExclusionCriteria":"EXTENSION PHASE: Platelet count, white blood cells, and/or haemoglobin < LLN at EOT/V12","principalExclusionCriteriaTranslations":[{"id":5856596,"uuid":"119c7a62-786d-48fb-a453-4c3854f1f0b0","attributeTranslation":"Aantal 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)","language":37,"languageDescription":"Dutch (Netherlands)"}]}]},"endPoint":{"primaryEndPoints":[{"id":593933,"number":1,"endPoint":"CORE 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)","isPrimary":true,"endPointTranslations":[{"id":5856568,"uuid":"657a90e2-0d80-4bfe-a5cf-324fa03238a7","attributeTranslation":"•\tGebied 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)\n•\tMaximale plasmaconcentratie in stationaire toestand (Cmax,ss) na ten minste 7 dagen dosering (na week 1 en na 6 maanden)\n•\tEliminatiehalfwaardetijd (t1/2) beoordeeld na ten minste 7 dagen dosering (na week 1 en na 6 maanden)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":593934,"number":2,"endPoint":"EXTENSION PHASE: •\tType, incidence, and severity of TEAEs and SAEs from baseline up to Week 144  •\tProportion of subjects experiencing TEAEs from baseline to Week 144","isPrimary":true,"endPointTranslations":[{"id":5856569,"uuid":"f4f82d12-e5f4-4614-b8ea-0751678cd979","attributeTranslation":"•\tType, incidentie en ernst van TEAE’s en SAE’s vanaf de baseline tot week 144\n•\tPercentage proefpersonen dat TEAE’s ervaart vanaf de baseline tot week 144","language":37,"languageDescription":"Dutch (Netherlands)"}]}],"secondaryEndPoints":[{"id":593935,"number":1,"endPoint":"CORE PHASE: •\tType, incidence, and severity of treatment-emergent adverse events (TEAEs) and SAEs from baseline to Week 48 •\tProportion of subjects experiencing TEAEs from baseline to Week 48 •\tChange from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 48 •\tChange from baseline electrocardiogram (ECG) to each postbaseline visit up to Week 48","isPrimary":false,"endPointTranslations":[{"id":5856570,"uuid":"67f1f75f-5791-4049-bd7f-e959a85b712c","attributeTranslation":"•\tType, incidentie en ernst van tijdens de behandeling optredende AE’s (TEAE’s) en SAE’s vanaf de baseline tot week 48\n•\tPercentage proefpersonen dat TEAE’s ervaart vanaf de baseline tot week 48\n•\tVerandering ten opzichte van de baseline in vitale functies en klinisch laboratorium onderzoek tot elk bezoek na de baseline tot week 48\n•\tVerandering ten opzichte van het baseline-ECG tot elk bezoek na de baseline tot week 48","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":593936,"number":2,"endPoint":"CORE PHASE: •\tChange 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 •\tChange 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","isPrimary":false,"endPointTranslations":[{"id":5856571,"uuid":"66896ffe-9c55-4f8a-9714-6b12a508cb7d","attributeTranslation":"•\tVerandering in lichamelijk functioneren volgens de Bayley III Gross Motor-schaal van baseline tot week 48 voor proefpersonen van ≥ 2 tot < 3,5 jaar oud\n•\tVerandering 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 oud","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":593937,"number":3,"endPoint":"CORE PHASE: •\tChange in Paediatric Outcomes Data Collection Instrument (PODCI) from baseline to Week 48 for subjects aged ≥4 years of age in Cohort 1 only","isPrimary":false,"endPointTranslations":[{"id":5856572,"uuid":"434828ee-1a0c-4829-b093-2af4272f99a6","attributeTranslation":"•\tVerandering in Pediatric Outcomes Data Collection Instrument (PODCI) vanaf baseline tot week 48, alleen voor proefpersonen van ≥ 4 jaar in cohort 1","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":593938,"number":4,"endPoint":"EXTENSION PHASE: •\tType, incidence, and severity of TEAEs and SAEs from baseline up to Week 144  •\tProportion of patients experiencing TEAEs from baseline to Week 144 •\tChange from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 144 •\tChange from baseline ECG to each postbaseline up to Week 144","isPrimary":false,"endPointTranslations":[{"id":5856573,"uuid":"9cef4980-b2ab-46ef-8d7b-b2332ff48e3b","attributeTranslation":"•\tVerandering ten opzichte van de baseline in vitale functies en klinisch laboratorium onderzoek tot week 144\n•\tVerandering ten opzichte van het baseline-ECG tot elk bezoek na de baseline tot week 144","language":37,"languageDescription":"Dutch (Netherlands)"}]}]},"trialDuration":{"estimatedGlobalEndDate":"2028-12-31","estimatedEndDate":"2028-12-31","estimatedRecruitmentStartDate":"2024-09-01"},"sourceOfMonetarySupport":[],"populationOfTrialSubjects":{"ageRanges":[{"id":213912,"ageRangeCategoryCode":"2","ageRangeCategory":"2"}],"ageRangeSecondaryIds":[],"clinicalTrialGroups":[{"code":"2","name":"Patients"}],"isFemaleSubjects":false,"isMaleSubjects":true,"isVulnerablePopulationSelected":true},"individualParticipantData":{"planToShareIPD":"3","planDescription":"NAP"}},"protocolInformation":{"studyDesign":{"periodDetails":[{"id":116299,"businessKey":"1","title":"Open label, multicentre, multicountry, 2 cohorts to evaluate the PK profile and safety of givinostat","description":"Subjects 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. 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DMD en lo que respecta a fracturas no vertebrales, cataratas y retraso de la pubertad","language":7,"languageDescription":"Spanish"},{"id":8738060,"uuid":"d8802a8e-6cfd-44fb-85fa-4b8238b39bfc","attributeTranslation":"Het beoordelen van de veiligheid van de behandeling op de lange termijn met vamorolon bij jongens met DMD op niet-wervelfracturen, staargevallen en vertraagde puberteit.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":376101,"number":2,"secondaryObjective":"To evaluate the overall safety of long-term treatment with  vamorolone in boys with DMD","secondaryObjectiveTranslations":[{"id":8738063,"uuid":"a939a4d7-d7e3-49e7-8205-790e8a06a37f","attributeTranslation":"Het beoordelen van de algehele veiligheid van de behandeling op de lange termijn","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":8738065,"uuid":"a939a4d7-d7e3-49e7-8205-790e8a06a37f","attributeTranslation":"Η αξιολόγηση της συνολικής ασφάλειας της μακροχρόνιας θεραπείας με 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gegeven.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":8738042,"uuid":"08dd0957-d18b-4c00-8b13-7afcad5eadcd","attributeTranslation":"Ο ασθενής ή/και ο(οι) γονέας(-είς) ή ο νόμιμος κηδεμόνας του ασθενούς έχει παράσχει γραπτή συναίνεση μετά από ενημέρωση","language":5,"languageDescription":"Greek"}]},{"id":721801,"number":2,"principalInclusionCriteria":"Subject has previously completed either the VBP15-LTE or VBP15-004  study, and transitioned through the CUP, NPP or EAP","principalInclusionCriteriaTranslations":[{"id":8738047,"uuid":"936541b8-f164-416a-90d2-3d5ccfb837ce","attributeTranslation":"Ο ασθενής έχει προηγουμένως ολοκληρώσει είτε τη μελέτη VBP15-LTE είτε τη μελέτη VBP15-004 και έχει μεταβεί μέσω των προγραμμάτων CUP, NPP ή EAP","language":5,"languageDescription":"Greek"},{"id":8738045,"uuid":"936541b8-f164-416a-90d2-3d5ccfb837ce","attributeTranslation":"El sujeto ha completado previamente el estudio VBP15-LTE o el estudio VBP15-004 y ha pasado por un PUC, un PPD o un PAE.","language":7,"languageDescription":"Spanish"},{"id":8738046,"uuid":"936541b8-f164-416a-90d2-3d5ccfb837ce","attributeTranslation":"De 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).","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":721802,"number":3,"principalInclusionCriteria":"Subject is on vamorolone on the day of enrolment","principalInclusionCriteriaTranslations":[{"id":8738049,"uuid":"decf361d-17f2-4d3d-8008-5866d57fd27a","attributeTranslation":"El sujeto está recibiendo vamorolona el día del reclutamiento","language":7,"languageDescription":"Spanish"},{"id":8738048,"uuid":"decf361d-17f2-4d3d-8008-5866d57fd27a","attributeTranslation":"Ο ασθενής λαμβάνει βαμορολόνη την ημέρα της ένταξης","language":5,"languageDescription":"Greek"},{"id":8738050,"uuid":"decf361d-17f2-4d3d-8008-5866d57fd27a","attributeTranslation":"Proefpersoon gebruikt vamorolon op de dag van inschrijving.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":721803,"number":4,"principalInclusionCriteria":"Subject and parent / legal guardian are willing and able to comply with the protocol schedule, assessments and requirements","principalInclusionCriteriaTranslations":[{"id":8738053,"uuid":"15ffa453-3d1e-4440-af9d-04d3cff22fb0","attributeTranslation":"De proefpersoon en zijn ouder/wettelijke vertegenwoordiger zijn bereid en in staat om zich te houden aan het protocolschema, de beoordelingen en de vereisten.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":8738052,"uuid":"15ffa453-3d1e-4440-af9d-04d3cff22fb0","attributeTranslation":"Ο ασθενής και ο γονέας/νόμιμος κηδεμόνας είναι πρόθυμοι και ικανοί να συμμορφωθούν με το χρονοδιάγραμμα, τις αξιολογήσεις και τις απαιτήσεις του πρωτοκόλλου","language":5,"languageDescription":"Greek"},{"id":8738051,"uuid":"15ffa453-3d1e-4440-af9d-04d3cff22fb0","attributeTranslation":"El sujeto y sus padres/tutor legal pueden y están dispuestos a cumplir el calendario, las evaluaciones y los requisitos del protocolo","language":7,"languageDescription":"Spanish"}]}],"principalExclusionCriteria":[{"id":1238562,"number":1,"principalExclusionCriteria":"Any medical condition, which in the opinion of the Investigator, would  affect study participation, performance or interpretation of study  assessments","principalExclusionCriteriaTranslations":[{"id":8738031,"uuid":"46e141c2-33e7-42e8-a2c9-00f021223ada","attributeTranslation":"Οποιαδήποτε ιατρική πάθηση, η οποία, κατά τη γνώμη του ερευνητή, θα επηρέαζε τη συμμετοχή στη μελέτη, την απόδοση ή την ερμηνεία των αξιολογήσεων της μελέτης","language":5,"languageDescription":"Greek"},{"id":8738030,"uuid":"46e141c2-33e7-42e8-a2c9-00f021223ada","attributeTranslation":"Elke 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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)","product":"ITF2357","ageRangeSecondary":["6","5"],"ageGroup":"0-17 years, 18-64 years","gender":"Male","trialRegion":3,"totalNumberEnrolled":"120","primaryEndPoint":"Type, incidence, and severity of treatment related/not related AEs and SAEs","resultsFirstReceived":"No","lastUpdated":"11/07/2025","lastPublicationUpdate":"12/07/2025"},{"ctNumber":"2023-504520-26-00","ctStatus":"Authorised","startDateEU":"2017-10-19","decisionDate":"2024-09-09T08:18:37.276","publishDate":"2025-07-12T03:35:17.885642217","ctPublicStatusCode":4,"authorizedApplication":{"authorizedPartI":{"id":92661,"rowSubjectCount":102,"rowCountriesInfo":[{"eutctId":100000000518,"name":"Serbia","isoNumber":688,"isoAlpha2Code":"RS","isoAlpha3Code":"SRB","current":true},{"eutctId":100000000429,"name":"Israel","isoNumber":376,"isoAlpha2Code":"IL","isoAlpha3Code":"ISR","current":true},{"eutctId":100000000557,"name":"United States","isoNumber":840,"isoAlpha2Code":"US","isoAlpha3Code":"USA","current":true},{"eutctId":100000000354,"name":"Canada","isoNumber":124,"isoAlpha2Code":"CA","isoAlpha3Code":"CAN","current":true},{"eutctId":100000000556,"name":"United Kingdom","isoNumber":826,"isoAlpha2Code":"GB","isoAlpha3Code":"GBR","current":true}],"products":[{"id":381032,"part1MpRoleTypeCode":"1","productDictionaryInfo":{"productPk":"11133213","productPharmForm":"ORAL SUSPENSION","euMpNumber":"PRD4797678","prodAuthStatus":1,"prodName":"ITF2357","pharmForm":"ORAL SUSPENSION","sponsorProductCode":"ITF2357","activeSubstanceName":"GIVINOSTAT","euSubstNumber":"SUB189959","productOtherName":"GIVINOSTAT","nameOrg":"ITALFARMACO SPA","productSubstances":[{"productPk":"11133213","substancePk":"337329","nameOrg":"ITALFARMACO SPA","substanceOrigin":"Chemical","actSubstOrigin":"Chemical","actSubstName":"GIVINOSTAT","substanceEvCode":"SUB189959"}],"activeSubstanceOtherDescriptiveName":""},"isPaediatricFormulation":false,"mpRoleInTrial":"1","orphanDrugEdit":true,"orphanDrugDesigNumber":"EU/3/12/1009","doseUom":"mg milligram(s)","maxDailyDoseAmount":"140","doseUomTotal":"mg milligram(s)","maxTotalDoseAmount":"00","maxTreatmentPeriod":1,"timeUnitCode":"1","otherMedicinalProduct":"Chemical","evCode":"PRD4797678","sponsorProductCodeEdit":"ITF2357","devices":[],"characteristics":["11"],"routes":["ORAL USE"],"allSubstancesChemicals":true,"productName":"ITF2357","jsonActiveSubstanceNames":"givinostat","pharmaceuticalFormDisplay":"ORAL SUSPENSION"}],"trialDetails":{"clinicalTrialIdentifiers":{"fullTitle":"Open 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.","fullTitleTranslations":[{"id":7196004,"uuid":"052a1424-3974-4684-9c3a-1abb0fa8b5d1","attributeTranslation":"Étude 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 GIVINOSTAT","language":10,"languageDescription":"French"},{"id":7196005,"uuid":"052a1424-3974-4684-9c3a-1abb0fa8b5d1","attributeTranslation":"Estudio 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 GIVINOSTAT","language":7,"languageDescription":"Spanish"},{"id":7196006,"uuid":"052a1424-3974-4684-9c3a-1abb0fa8b5d1","attributeTranslation":"Open-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-onderzoeken","language":37,"languageDescription":"Dutch (Netherlands)"}],"publicTitle":"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 past","publicTitleTranslations":[{"id":7196023,"uuid":"4d05e17d-d690-482c-b75d-a27903592f0d","attributeTranslation":"Estudio 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.","language":7,"languageDescription":"Spanish"},{"id":7196022,"uuid":"4d05e17d-d690-482c-b75d-a27903592f0d","attributeTranslation":"Étude 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 passé","language":10,"languageDescription":"French"},{"id":7196024,"uuid":"4d05e17d-d690-482c-b75d-a27903592f0d","attributeTranslation":"Onderzoek 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 behandeld","language":37,"languageDescription":"Dutch (Netherlands)"}],"shortTitle":"DSC/14/2357/51","secondaryIdentifyingNumbers":{"additionalRegistries":[]}},"trialInformation":{"trialCategory":{"isLowIntervention":false,"trialPhase":"5","trialCategory":"2","justificationForTrialCategory":"This 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.","trialCategoryId":83588},"medicalCondition":{"partIMedicalConditions":[{"id":95355,"medicalCondition":"Duchenne Muscular Dystrophy (DMD)","medicalConditionTranslations":[{"id":7195932,"uuid":"a7e0c782-8b90-4bf7-aede-33869d742f6f","attributeTranslation":"Dystrophie musculaire de Duchenne (DMD)","language":10,"languageDescription":"French"},{"id":7195933,"uuid":"a7e0c782-8b90-4bf7-aede-33869d742f6f","attributeTranslation":"Duchenne spierdystrofie (DMD)","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195934,"uuid":"a7e0c782-8b90-4bf7-aede-33869d742f6f","attributeTranslation":"Distrofia muscular de Duchenne (DMD)","language":7,"languageDescription":"Spanish"}],"isConditionRareDisease":true}],"meddraConditionTerms":[{"termId":100000012544,"version":"20.0","level":"PT","termName":"Duchenne muscular dystrophy","classificationCode":"10013801","organClass":100000004850,"active":false}]},"trialObjective":{"trialScopes":[{"code":"5","trialScopeId":276197},{"code":"4","trialScopeId":276199},{"code":"13","otherDescription":"Tollerability","trialScopeId":276198}],"mainObjective":"To 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:\n- all the inclusion criteria and none of the exclusion criteria, \nand\n- never been randomized because the enrollment in the off-target group was completed.","mainObjectiveTranslations":[{"id":7196020,"uuid":"292d7db0-8e45-49bd-939c-31d4b9a53d5b","attributeTranslation":"Évaluer 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 :\n- ont répondu à la totalité des critères d’inclusion et n’ont répondu à aucun des critères d’exclusion, et\n- n’ont jamais été randomisés, car l’inclusion dans la cohorte hors cible était terminée.","language":10,"languageDescription":"French"},{"id":7196021,"uuid":"292d7db0-8e45-49bd-939c-31d4b9a53d5b","attributeTranslation":"Het 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:\n- alle inclusiecriteria en geen van de exclusiecriteria; en\n- nooit zijn gerandomiseerd, omdat de inschrijving in de off-target groep voltooid was.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7196019,"uuid":"292d7db0-8e45-49bd-939c-31d4b9a53d5b","attributeTranslation":"Evaluar 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:\n— cumplieron todos los criterios de inclusión y ninguno de los criterios de exclusión, y\n— nunca habían sido aleatorizados porque se había completado la inclusión en el grupo no objetivo.","language":7,"languageDescription":"Spanish"}],"secondaryObjectives":[{"id":316369,"number":1,"secondaryObjective":"To evaluate the effects of long-term administration of GIVINOSTAT on muscular function and strength;","secondaryObjectiveTranslations":[{"id":7196027,"uuid":"ad8f6886-5827-4370-bb54-45666340d2a8","attributeTranslation":"Evaluar los efectos de la administración a largo plazo de GIVINOSTAT sobre la función y la fuerza muscular","language":7,"languageDescription":"Spanish"},{"id":7196025,"uuid":"ad8f6886-5827-4370-bb54-45666340d2a8","attributeTranslation":"Het evalueren van de effecten van langetermijntoediening van GIVINOSTAT op de spierfunctie en de spierkracht","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7196026,"uuid":"ad8f6886-5827-4370-bb54-45666340d2a8","attributeTranslation":"Évaluer les effets de l’administration à long terme du GIVINOSTAT sur la fonction et la force musculaires","language":10,"languageDescription":"French"}]},{"id":316370,"number":2,"secondaryObjective":"To evaluate the effects of long-term administration of GIVINOSTAT on respiratory function","secondaryObjectiveTranslations":[{"id":7196028,"uuid":"8de26b6c-316c-428d-8d1e-bbb2e69d40d3","attributeTranslation":"Het evalueren van de effecten van langetermijntoediening van GIVINOSTAT op de ademhalingsfunctie.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7196029,"uuid":"8de26b6c-316c-428d-8d1e-bbb2e69d40d3","attributeTranslation":"Evaluar los efectos de la administración a largo plazo de GIVINOSTAT sobre la función respiratoria.","language":7,"languageDescription":"Spanish"},{"id":7196030,"uuid":"8de26b6c-316c-428d-8d1e-bbb2e69d40d3","attributeTranslation":"Évaluer les effets de l’administration à long terme du GIVINOSTAT sur la fonction respiratoire","language":10,"languageDescription":"French"}]},{"id":316371,"number":3,"secondaryObjective":"To evaluate the impact on daily activities and quality of life following long-term administration of GIVINOSTAT.","secondaryObjectiveTranslations":[{"id":7196031,"uuid":"ce4c4e1b-96b1-4cff-8867-7f1762397896","attributeTranslation":"Évaluer l’impact sur les activités quotidiennes et la qualité de vie après l’administration à long terme du GIVINOSTAT","language":10,"languageDescription":"French"},{"id":7196033,"uuid":"ce4c4e1b-96b1-4cff-8867-7f1762397896","attributeTranslation":"Evaluar el impacto en las actividades cotidianas y la calidad de vida después de la administración a largo plazo de GIVINOSTAT.","language":7,"languageDescription":"Spanish"},{"id":7196032,"uuid":"ce4c4e1b-96b1-4cff-8867-7f1762397896","attributeTranslation":"Het evalueren van de impact op de dagelijkse activiteiten en de kwaliteit van leven na langetermijntoediening van GIVINOSTAT","language":37,"languageDescription":"Dutch (Netherlands)"}]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":609245,"number":1,"principalInclusionCriteria":"Subjects 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 completed","principalInclusionCriteriaTranslations":[{"id":7196007,"uuid":"59b9d250-9386-495a-9f9b-f146080469e1","attributeTranslation":"Los 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.","language":7,"languageDescription":"Spanish"},{"id":7196009,"uuid":"59b9d250-9386-495a-9f9b-f146080469e1","attributeTranslation":"Proefpersonen 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.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7196008,"uuid":"59b9d250-9386-495a-9f9b-f146080469e1","attributeTranslation":"Les 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 terminée","language":10,"languageDescription":"French"}]},{"id":609246,"number":2,"principalInclusionCriteria":"Subjects aged ≥6 years","principalInclusionCriteriaTranslations":[{"id":7196010,"uuid":"aaf4cc7b-d817-4286-a7a7-66d84d49bea4","attributeTranslation":"Proefpersonen met een leeftijd ≥ 6 jaar.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7196012,"uuid":"aaf4cc7b-d817-4286-a7a7-66d84d49bea4","attributeTranslation":"Participantes con una edad ≥6 años.","language":7,"languageDescription":"Spanish"},{"id":7196011,"uuid":"aaf4cc7b-d817-4286-a7a7-66d84d49bea4","attributeTranslation":"Patients âgés de ≥ 6 ans","language":10,"languageDescription":"French"}]},{"id":609247,"number":3,"principalInclusionCriteria":"Subjects are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations)","principalInclusionCriteriaTranslations":[{"id":7196015,"uuid":"bb1a4229-58ae-400c-a74d-a7ea35542cd5","attributeTranslation":"Les 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)","language":10,"languageDescription":"French"},{"id":7196013,"uuid":"bb1a4229-58ae-400c-a74d-a7ea35542cd5","attributeTranslation":"Los 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).","language":7,"languageDescription":"Spanish"},{"id":7196014,"uuid":"bb1a4229-58ae-400c-a74d-a7ea35542cd5","attributeTranslation":"Proefpersonen zijn in staat om schriftelijke geïnformeerde instemming en/of toestemming te geven, ondertekend door de proefpersoon en/of ouder/wettelijk voogd (volgens plaatselijke regelgeving).","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":609248,"number":4,"principalInclusionCriteria":"Subjects 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.","principalInclusionCriteriaTranslations":[{"id":7196018,"uuid":"43eafb18-1f7e-49e3-99a6-946398e6c0ef","attributeTranslation":"Proefpersonen moeten bereid zijn om adequate anticonceptie te gebruiken:\nAnticonceptiemethoden moeten zijn toegepast sinds het vorige GIVINOSTAT-onderzoek tot 3 maanden na de laatste dosis onderzoeksmiddel en het volgende omvatten:\n-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.\n- 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.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7196016,"uuid":"43eafb18-1f7e-49e3-99a6-946398e6c0ef","attributeTranslation":"Les patients doivent être disposés à utiliser une contraception adéquate.\nLes 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 :\n- 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.\n- 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.","language":10,"languageDescription":"French"},{"id":7196017,"uuid":"43eafb18-1f7e-49e3-99a6-946398e6c0ef","attributeTranslation":"Los participantes deben estar dispuestos a utilizar métodos anticonceptivos adecuados:\nLos 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:\n— 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.\n— 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.","language":7,"languageDescription":"Spanish"}]}],"principalExclusionCriteria":[{"id":1047253,"number":1,"principalExclusionCriteria":"Use 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;","principalExclusionCriteriaTranslations":[{"id":7195967,"uuid":"045feed2-0630-44cf-ab3b-99f323be8f6d","attributeTranslation":"Utilisation 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 autorisés","language":10,"languageDescription":"French"},{"id":7195965,"uuid":"045feed2-0630-44cf-ab3b-99f323be8f6d","attributeTranslation":"Het 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.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195966,"uuid":"045feed2-0630-44cf-ab3b-99f323be8f6d","attributeTranslation":"Uso 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 suplemento","language":7,"languageDescription":"Spanish"}]},{"id":1047254,"number":10,"principalExclusionCriteria":"Have 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)","principalExclusionCriteriaTranslations":[{"id":7195970,"uuid":"9f415ee4-562c-4c60-837e-1f62297e4fa0","attributeTranslation":"Présenter 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)","language":10,"languageDescription":"French"},{"id":7195969,"uuid":"9f415ee4-562c-4c60-837e-1f62297e4fa0","attributeTranslation":"Tener 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).","language":7,"languageDescription":"Spanish"},{"id":7195968,"uuid":"9f415ee4-562c-4c60-837e-1f62297e4fa0","attributeTranslation":"Het 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).","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1047255,"number":11,"principalExclusionCriteria":"Have 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 procedures","principalExclusionCriteriaTranslations":[{"id":7195972,"uuid":"8e52c6f9-b565-4402-8214-c7ba53f8a7d5","attributeTranslation":"Tener 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.","language":7,"languageDescription":"Spanish"},{"id":7195973,"uuid":"8e52c6f9-b565-4402-8214-c7ba53f8a7d5","attributeTranslation":"Avoir 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 conformer","language":10,"languageDescription":"French"},{"id":7195971,"uuid":"8e52c6f9-b565-4402-8214-c7ba53f8a7d5","attributeTranslation":"Het 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.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1047256,"number":12,"principalExclusionCriteria":"Have any hypersensitivity to the components of study medication;","principalExclusionCriteriaTranslations":[{"id":7195975,"uuid":"77539a2d-03d7-4eb0-9967-9b62539b4962","attributeTranslation":"Tener hipersensibilidad a los componentes del fármaco del estudio.","language":7,"languageDescription":"Spanish"},{"id":7195976,"uuid":"77539a2d-03d7-4eb0-9967-9b62539b4962","attributeTranslation":"Het hebben van een overgevoeligheid voor de bestanddelen van het onderzoeksmiddel","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195974,"uuid":"77539a2d-03d7-4eb0-9967-9b62539b4962","attributeTranslation":"Présenter une hypersensibilité aux composants du médicament à l’étude","language":10,"languageDescription":"French"}]},{"id":1047257,"number":13,"principalExclusionCriteria":"Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance","principalExclusionCriteriaTranslations":[{"id":7195978,"uuid":"336ea243-860d-49af-be84-cc13ce676313","attributeTranslation":"Tener intolerancia al sorbitol o malabsorción de sorbitol o intolerancia hereditaria a la fructosa.","language":7,"languageDescription":"Spanish"},{"id":7195979,"uuid":"336ea243-860d-49af-be84-cc13ce676313","attributeTranslation":"Présenter une intolérance au sorbitol ou une malabsorption du sorbitol, ou une forme héréditaire d’intolérance au fructose","language":10,"languageDescription":"French"},{"id":7195977,"uuid":"336ea243-860d-49af-be84-cc13ce676313","attributeTranslation":"Het hebben van een sorbitolintolerantie of sorbitolmalabsorptie of het hebben van de erfelijke vorm van fructose-intolerantie.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1047258,"number":2,"principalExclusionCriteria":"Use of any current investigational drug other than Givinostat","principalExclusionCriteriaTranslations":[{"id":7195981,"uuid":"a4158122-f312-4762-b770-40cb68259949","attributeTranslation":"Uso de cualquier fármaco en investigación actual distinto de GIVINOSTAT.","language":7,"languageDescription":"Spanish"},{"id":7195982,"uuid":"a4158122-f312-4762-b770-40cb68259949","attributeTranslation":"Utilisation de tout médicament en cours d’investigation autre que le Givinostat","language":10,"languageDescription":"French"},{"id":7195980,"uuid":"a4158122-f312-4762-b770-40cb68259949","attributeTranslation":"Huidig gebruik van een experimenteel geneesmiddel anders dan Givinostat.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1047259,"number":3,"principalExclusionCriteria":"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 results","principalExclusionCriteriaTranslations":[{"id":7195985,"uuid":"f20da03f-d3a6-46f9-b508-e7cf8afcc1c4","attributeTranslation":"Aanwezigheid 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.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195983,"uuid":"f20da03f-d3a6-46f9-b508-e7cf8afcc1c4","attributeTranslation":"Presencia 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.","language":7,"languageDescription":"Spanish"},{"id":7195984,"uuid":"f20da03f-d3a6-46f9-b508-e7cf8afcc1c4","attributeTranslation":"Présenter 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’étude","language":10,"languageDescription":"French"}]},{"id":1047260,"number":4,"principalExclusionCriteria":"Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD","principalExclusionCriteriaTranslations":[{"id":7195986,"uuid":"a7c15cd5-7994-419d-99f0-bc095da5b943","attributeTranslation":"Tener un diagnóstico de otras enfermedades neurológicas no controladas o presencia de trastornos somáticos relevantes no controlados que no estén relacionados con la DMD.","language":7,"languageDescription":"Spanish"},{"id":7195987,"uuid":"a7c15cd5-7994-419d-99f0-bc095da5b943","attributeTranslation":"Avoir reçu le diagnostic d’autres maladies neurologiques non contrôlées ou présenter des troubles somatiques non contrôlés importants qui ne sont pas liés à la DMD","language":10,"languageDescription":"French"},{"id":7195988,"uuid":"a7c15cd5-7994-419d-99f0-bc095da5b943","attributeTranslation":"Het hebben van een diagnose van andere ongecontroleerde neurologische ziekten of aanwezigheid van relevante ongecontroleerde somatische aandoeningen die niet gerelateerd zijn aan DMD.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1047261,"number":5,"principalExclusionCriteria":"Have platelets count, White Blood Cell and Hemoglobin at screening < Lower Limit of Normal (LLN) (for abnormal screening laboratory test results (","principalExclusionCriteriaTranslations":[{"id":7195989,"uuid":"b30aafcf-42fe-433c-82a1-b29622a29520","attributeTranslation":"Présenter 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)","language":10,"languageDescription":"French"},{"id":7195991,"uuid":"b30aafcf-42fe-433c-82a1-b29622a29520","attributeTranslation":"Het 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 (","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195990,"uuid":"b30aafcf-42fe-433c-82a1-b29622a29520","attributeTranslation":"Tener 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 [","language":7,"languageDescription":"Spanish"}]},{"id":1047262,"number":6,"principalExclusionCriteria":"Have 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 exclusionary","principalExclusionCriteriaTranslations":[{"id":7195994,"uuid":"a019d004-78ea-4373-ad43-6782bc00365b","attributeTranslation":"Tener 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.","language":7,"languageDescription":"Spanish"},{"id":7195993,"uuid":"a019d004-78ea-4373-ad43-6782bc00365b","attributeTranslation":"De 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).","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195992,"uuid":"a019d004-78ea-4373-ad43-6782bc00365b","attributeTranslation":"Présenter 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)","language":10,"languageDescription":"French"}]},{"id":1047263,"number":7,"principalExclusionCriteria":"Have 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);","principalExclusionCriteriaTranslations":[{"id":7195997,"uuid":"fdfcb82a-6919-48ca-a55e-87085c04409b","attributeTranslation":"Présenter 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’exclusion","language":10,"languageDescription":"French"},{"id":7195996,"uuid":"fdfcb82a-6919-48ca-a55e-87085c04409b","attributeTranslation":"Het 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).","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195995,"uuid":"fdfcb82a-6919-48ca-a55e-87085c04409b","attributeTranslation":"Presencia 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).","language":7,"languageDescription":"Spanish"}]},{"id":1047264,"number":8,"principalExclusionCriteria":"Have heart failure (New York Heart Association Class III or IV)","principalExclusionCriteriaTranslations":[{"id":7195998,"uuid":"ad19ab18-66a8-48f7-800c-078ff67f4da9","attributeTranslation":"Présenter une insuffisance cardiaque (de classe III ou IV sur l’échelle de la New York Heart Association)","language":10,"languageDescription":"French"},{"id":7196000,"uuid":"ad19ab18-66a8-48f7-800c-078ff67f4da9","attributeTranslation":"Het hebben van hartfalen (New York Heart Association klasse III of IV).","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195999,"uuid":"ad19ab18-66a8-48f7-800c-078ff67f4da9","attributeTranslation":"Tener insuficiencia cardíaca (clase III o IV según la Asociación Cardiológica de Nueva York).","language":7,"languageDescription":"Spanish"}]},{"id":1047265,"number":9,"principalExclusionCriteria":"Have 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's","principalExclusionCriteriaTranslations":[{"id":7196003,"uuid":"ca847bec-46a0-43fb-80d4-96621179e25d","attributeTranslation":"Tener 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.","language":7,"languageDescription":"Spanish"},{"id":7196002,"uuid":"ca847bec-46a0-43fb-80d4-96621179e25d","attributeTranslation":"Présenter 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 Gilbert","language":10,"languageDescription":"French"},{"id":7196001,"uuid":"ca847bec-46a0-43fb-80d4-96621179e25d","attributeTranslation":"Het 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.","language":37,"languageDescription":"Dutch (Netherlands)"}]}]},"endPoint":{"primaryEndPoints":[{"id":711762,"number":1,"endPoint":"Type, incidence, and severity of treatment related/not related AEs and SAEs","isPrimary":true,"endPointTranslations":[{"id":7195936,"uuid":"f6d7dba4-7c04-4b4d-925f-9401c86259f4","attributeTranslation":"Type, incidence et sévérité des EI et des EIG liés ou non liés au traitement","language":10,"languageDescription":"French"},{"id":7195937,"uuid":"f6d7dba4-7c04-4b4d-925f-9401c86259f4","attributeTranslation":"Type, incidentie en ernst van aan de behandeling gerelateerde/niet aan de behandeling gerelateerde ongewenste voorvallen en ernstige ongewenste voorvallen.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195935,"uuid":"f6d7dba4-7c04-4b4d-925f-9401c86259f4","attributeTranslation":"Tipo, incidencia e intensidad de los AA y AAG relacionados o no relacionados con el tratamiento.","language":7,"languageDescription":"Spanish"}]}],"secondaryEndPoints":[{"id":711763,"number":1,"endPoint":"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)","isPrimary":false,"endPointTranslations":[{"id":7195940,"uuid":"cd23f700-c981-46a9-a54c-d2efbef2eff0","attributeTranslation":"Pour les patients capables de se déplacer :\n• 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)","language":10,"languageDescription":"French"},{"id":7195938,"uuid":"cd23f700-c981-46a9-a54c-d2efbef2eff0","attributeTranslation":"Para pacientes deambulantes:\n• 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).","language":7,"languageDescription":"Spanish"},{"id":7195939,"uuid":"cd23f700-c981-46a9-a54c-d2efbef2eff0","attributeTranslation":"Voor poliklinische patiënten:\n• 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).","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":711764,"number":2,"endPoint":"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)","isPrimary":false,"endPointTranslations":[{"id":7195942,"uuid":"0bdcf54b-d110-41ef-a181-0a2c702fbd22","attributeTranslation":"Voor poliklinische patiënten:\nVerandering 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).","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195941,"uuid":"0bdcf54b-d110-41ef-a181-0a2c702fbd22","attributeTranslation":"Pour les patients capables de se déplacer :\nVariation 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)","language":10,"languageDescription":"French"},{"id":7195943,"uuid":"0bdcf54b-d110-41ef-a181-0a2c702fbd22","attributeTranslation":"Para pacientes deambulantes:\nCambio 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).","language":7,"languageDescription":"Spanish"}]},{"id":711765,"number":3,"endPoint":"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)","isPrimary":false,"endPointTranslations":[{"id":7195944,"uuid":"231965e9-bf33-4dc0-86ef-0faec4905918","attributeTranslation":"Pour les patients incapables de se déplacer :\n• 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)","language":10,"languageDescription":"French"},{"id":7195946,"uuid":"231965e9-bf33-4dc0-86ef-0faec4905918","attributeTranslation":"Voor niet-poliklinische patiënten:\n• 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).","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195945,"uuid":"231965e9-bf33-4dc0-86ef-0faec4905918","attributeTranslation":"Para pacientes no deambulantes:\n• 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).","language":7,"languageDescription":"Spanish"}]},{"id":711766,"number":4,"endPoint":"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)","isPrimary":false,"endPointTranslations":[{"id":7195949,"uuid":"0fb80a3e-c3ed-4a7c-8860-30efc5175049","attributeTranslation":"Para pacientes no deambulantes:\n• 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).","language":7,"languageDescription":"Spanish"},{"id":7195947,"uuid":"0fb80a3e-c3ed-4a7c-8860-30efc5175049","attributeTranslation":"Voor niet-poliklinische patiënten:\n• 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).","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195948,"uuid":"0fb80a3e-c3ed-4a7c-8860-30efc5175049","attributeTranslation":"Pour les patients incapables de se déplacer :\n• 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)","language":10,"languageDescription":"French"}]},{"id":711767,"number":5,"endPoint":"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)","isPrimary":false,"endPointTranslations":[{"id":7195952,"uuid":"43887e7c-d9c4-4611-91df-9b2a7c54b2eb","attributeTranslation":"Para pacientes no deambulantes:\n• 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).","language":7,"languageDescription":"Spanish"},{"id":7195951,"uuid":"43887e7c-d9c4-4611-91df-9b2a7c54b2eb","attributeTranslation":"Voor niet-poliklinische patiënten:\n• Verandering in de spierkracht van de bovenste ledematen (week 48 en daarna jaarlijks tot het einde van het onderzoek) geëvalueerd met HHM (handheld myometrie).","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195950,"uuid":"43887e7c-d9c4-4611-91df-9b2a7c54b2eb","attributeTranslation":"Pour les patients incapables de se déplacer :\n• Variation de la force musculaire des membres supérieurs (semaine 48, puis annuellement jusqu’à la fin de l’étude) évaluée par myométrie (appareil portatif)","language":10,"languageDescription":"French"}]},{"id":711768,"number":6,"endPoint":"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)","isPrimary":false,"endPointTranslations":[{"id":7195953,"uuid":"e88e01da-29b6-4a85-a8fd-e13b94bd2901","attributeTranslation":"Voor alle patiënten:\nVerandering 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).","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":7195955,"uuid":"e88e01da-29b6-4a85-a8fd-e13b94bd2901","attributeTranslation":"Pour tous les patients :\nVariation 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)","language":10,"languageDescription":"French"},{"id":7195954,"uuid":"e88e01da-29b6-4a85-a8fd-e13b94bd2901","attributeTranslation":"Para todos los pacientes:\nCambio 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).","language":7,"languageDescription":"Spanish"}]},{"id":711769,"number":7,"endPoint":"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)","isPrimary":false,"endPointTranslations":[{"id":7195958,"uuid":"ce82936c-1add-4215-a943-2e65c8184147","attributeTranslation":"Voor alle patiënten:\nVerandering ten opzichte van de baseline in respiratoire functie (week 48 en daarna jaarlijks tot het einde van het onderzoek) (bijv. 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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.","language":7,"languageDescription":"Spanish"},{"id":5950294,"uuid":"3cd2265d-0a73-4593-b325-ec3367bcac43","attributeTranslation":"Diagnostic 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.\n– Les mutations entre les exons 1 à 17 ne sont pas éligibles.\n– Les délétions sans décalage de cadre, duplications sans décalage de cadre ou variant de signification incertaine (VSI) ne sont pas éligibles.","language":10,"languageDescription":"French"}]},{"id":516736,"number":6,"principalInclusionCriteria":"Able to cooperate with age-appropriate motor assessment testing in the opinion of the investigator.","principalInclusionCriteriaTranslations":[{"id":5950296,"uuid":"2569552b-c38e-4236-b474-5121bf084b5b","attributeTranslation":"Capaz de colaborar en las pruebas de evaluación motora adecuadas a su edad, en opinión del 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capable(s) de comprendre et de respecter le calendrier des visites de l'étude et toutes les exigences du protocole.","language":10,"languageDescription":"French"}]}],"principalExclusionCriteria":[{"id":889929,"number":1,"principalExclusionCriteria":"Has elevated anti rAAVrh74 antibody titers as determined by an investigational Elecsys anti-rAAVrh74 antibody assay obtained within 31 days of the infusion day","principalExclusionCriteriaTranslations":[{"id":5950245,"uuid":"227d6072-c409-4321-b93d-7992a66ce563","attributeTranslation":"Tener 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 perfusión","language":7,"languageDescription":"Spanish"},{"id":5950246,"uuid":"227d6072-c409-4321-b93d-7992a66ce563","attributeTranslation":"Présenter 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 perfusion","language":10,"languageDescription":"French"}]},{"id":889930,"number":10,"principalExclusionCriteria":"Symptomatic infection (e.g., upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.","principalExclusionCriteriaTranslations":[{"id":5950247,"uuid":"4465449f-faa5-4326-9d6a-45b4581f423d","attributeTranslation":"Présence d'une infection symptomatique (par exemple, infection des voies respiratoires supérieures, pneumonie, pyélonéphrite, méningite) dans les 4 semaines avant le Jour 1","language":10,"languageDescription":"French"},{"id":5950248,"uuid":"4465449f-faa5-4326-9d6a-45b4581f423d","attributeTranslation":"Infección sintomática (p. ej., de las vías respiratorias superiores, neumonía, pielonefritis, meningitis) en un plazo de 4 semanas antes del día 1.","language":7,"languageDescription":"Spanish"}]},{"id":889931,"number":11,"principalExclusionCriteria":"Cohort A and B: Positive serology testing for HIV 1 and/or 2, hepatitis C, or hepatitis B","principalExclusionCriteriaTranslations":[{"id":5950249,"uuid":"b2948f4e-0e61-4c47-8b83-06d08a2d8cba","attributeTranslation":"Cohortes 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 B","language":7,"languageDescription":"Spanish"},{"id":5950250,"uuid":"b2948f4e-0e61-4c47-8b83-06d08a2d8cba","attributeTranslation":"Cohortes A et B: Résultat positif à l'analyse sérologique pour le VIH1 et/ou 2, l'hépatite C ou l'hépatite B","language":10,"languageDescription":"French"}]},{"id":889932,"number":12,"principalExclusionCriteria":"Cohort 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 1","principalExclusionCriteriaTranslations":[{"id":5950252,"uuid":"72f5fce7-a1ac-4b6c-b591-a771c34ed66f","attributeTranslation":"Cohortes C et D:\n–Naissance prématurée (avant la fin de 37semaines de grossesse à 37semaines) ou complications significatives de la grossesse, selon l'avis de l'investigateur\n\n–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\n\n–Mère du participant si allaitement:\noSignes cliniques d'infection aiguë par le CMV avec confirmation par analyse PCR du CMV (échantillon urinaire)\noAnomalies cliniquement significatives de la fonction hépatique (GGT, ASAT, ALAT, PAL, bilirubine totale, GLDH) indiquant une hépatite infectieuse\noPathologie cliniquement significative ou infection aiguë indiquant une infection par virus hépatotrope (par exemple, CMV, EBV, VZV, etc) dans les 6 semaines avant le Jour1\noContact connu avec une personne atteinte d'hépatite aiguë ou active dans les 12 semaines avant le Jour1","language":10,"languageDescription":"French"},{"id":5950251,"uuid":"72f5fce7-a1ac-4b6c-b591-a771c34ed66f","attributeTranslation":"Cohortes 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 1","language":7,"languageDescription":"Spanish"}]},{"id":889933,"number":13,"principalExclusionCriteria":"Demonstrates cognitive delay or impairment that could confound motor development in the opinion of the investigator.","principalExclusionCriteriaTranslations":[{"id":5950254,"uuid":"5a7e8601-827e-4c83-9cba-44e098fe5696","attributeTranslation":"Demostrar retraso o deterioro cognitivo que pueda suponer un factor de confusión en el desarrollo motor, en opinión del investigador.","language":7,"languageDescription":"Spanish"},{"id":5950253,"uuid":"5a7e8601-827e-4c83-9cba-44e098fe5696","attributeTranslation":"Présence d'un retard cognitif ou d'une insuffisance cognitive susceptible d'interférer sur le développement moteur, selon l'avis de l'investigateur","language":10,"languageDescription":"French"}]},{"id":889934,"number":14,"principalExclusionCriteria":"Treatment 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™)","principalExclusionCriteriaTranslations":[{"id":5950256,"uuid":"291f971a-268d-4fa1-a1f9-b4342488ae58","attributeTranslation":"Tratamiento 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™)","language":7,"languageDescription":"Spanish"},{"id":5950255,"uuid":"291f971a-268d-4fa1-a1f9-b4342488ae58","attributeTranslation":"Administration d'un des traitemen suivants dans les limites temporelles spécifiées:\n–À tout moment:\noThérapie génique\noThérapie cellulaire (par exemple, greffe de cellules souches)\noCRISPR/Cas9, ou toute autre forme d'édition des gènes\n–Dans les 12 semaines avant le Jour1 et à tout moment pendant l'étude:\noUtilisation de facteur de croissance humain ou de vamorolone \n–Dans les 6 mois avant le Jour1 et à tout moment pendant l'étude:\noTout médicament expérimental\noTout traitement conçu pour augmenter l'expression de la dystrophine (par exemple, Translarna™, EXONDYS 51™, VILTEPSO™)","language":10,"languageDescription":"French"}]},{"id":889935,"number":15,"principalExclusionCriteria":"Has 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.","principalExclusionCriteriaTranslations":[{"id":5950258,"uuid":"e7b7b56b-9b40-4192-9718-8d01eb8700ed","attributeTranslation":"Haber 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.","language":7,"languageDescription":"Spanish"},{"id":5950257,"uuid":"e7b7b56b-9b40-4192-9718-8d01eb8700ed","attributeTranslation":"Administration 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 Jour1","language":10,"languageDescription":"French"}]},{"id":889936,"number":16,"principalExclusionCriteria":"Has 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 µl","principalExclusionCriteriaTranslations":[{"id":5950260,"uuid":"ac99c281-3bd2-45ff-aa93-8915fc5ae199","attributeTranslation":"Presentar 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 µl","language":7,"languageDescription":"Spanish"},{"id":5950259,"uuid":"ac99c281-3bd2-45ff-aa93-8915fc5ae199","attributeTranslation":"Anomalies considérées comme cliniquement significatives des paramètres biologiques, notamment:\n–GGT> 2 x limite supérieure de la normale (LSN)\n- GLDH> LSN\n–Bilirubine totale> LSN\noLes élévations de la bilirubine totale confirmées comme étant dues à un syndrome de Gilbert ne constituent pas un critère de non-inclusion\n–Numération des globules blancs (GB)>18 500 par µl\n–Plaquettes ≤ 150 000 par µl","language":10,"languageDescription":"French"}]},{"id":889937,"number":17,"principalExclusionCriteria":"In the opinion of the investigator, the participant is not likely to be compliant with the study protocol.","principalExclusionCriteriaTranslations":[{"id":5950262,"uuid":"8cf3d659-06ec-41aa-b2f6-74652645a7ed","attributeTranslation":"No es probable que el participante cumpla el protocolo del estudio, en opinión del investigador.","language":7,"languageDescription":"Spanish"},{"id":5950261,"uuid":"8cf3d659-06ec-41aa-b2f6-74652645a7ed","attributeTranslation":"Selon l'avis de l'investigateur, le participant n'est pas susceptible de respecter le protocole","language":10,"languageDescription":"French"}]},{"id":889938,"number":18,"principalExclusionCriteria":"Family does not want to disclose participant's study participation with general practitioner or primary care physician and other medical providers.","principalExclusionCriteriaTranslations":[{"id":5950264,"uuid":"dc4295d2-3de9-4205-9595-18477b02ce8a","attributeTranslation":"Refus de la famille du participant d'informer le médecin généraliste ou médecin de soins primaires et autres professionnels médicaux de la participation à cette étude","language":10,"languageDescription":"French"},{"id":5950263,"uuid":"dc4295d2-3de9-4205-9595-18477b02ce8a","attributeTranslation":"Familia 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.","language":7,"languageDescription":"Spanish"}]},{"id":889939,"number":19,"principalExclusionCriteria":"Positive COVID-19 test (antigen or PCR) on Day 1 prior to infusion","principalExclusionCriteriaTranslations":[{"id":5950265,"uuid":"255b5e54-0b96-44d3-85c1-c83134bc8b4e","attributeTranslation":"Prueba de COVID-19 positiva (antígenos o PCR) el día 1 antes de la infusión","language":7,"languageDescription":"Spanish"},{"id":5950266,"uuid":"255b5e54-0b96-44d3-85c1-c83134bc8b4e","attributeTranslation":"Résultat positif au test de dépistage de la COVID-19 (test antigénique ou PCR) le Jour 1 avant la perfusion","language":10,"languageDescription":"French"}]},{"id":889940,"number":2,"principalExclusionCriteria":"Poor peripheral venous access, which, in the opinion of the investigator, will lead to difficulty in venipuncture for the purposes of protocol-mandated procedures.","principalExclusionCriteriaTranslations":[{"id":5950268,"uuid":"5141ecf5-29c0-4f71-8475-36c8bd728d9c","attributeTranslation":"Accès veineux périphérique de mauvaise qualité, selon l’investigateur, rendant difficile les ponctions veineuses nécessaires pour les procédures exigées par le protocole.","language":10,"languageDescription":"French"},{"id":5950267,"uuid":"5141ecf5-29c0-4f71-8475-36c8bd728d9c","attributeTranslation":"Acceso venoso periférico deficiente, que, en opinión del investigador, provocará dificultades en las venopunciones precisas para los procedimientos exigidos por el protocolo.","language":7,"languageDescription":"Spanish"}]},{"id":889941,"number":3,"principalExclusionCriteria":"Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline.","principalExclusionCriteriaTranslations":[{"id":5950270,"uuid":"50c2282f-4898-4b4f-8a55-6784ec13cb36","attributeTranslation":"Traitement 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'inclusion","language":10,"languageDescription":"French"},{"id":5950269,"uuid":"50c2282f-4898-4b4f-8a55-6784ec13cb36","attributeTranslation":"Recibir 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.","language":7,"languageDescription":"Spanish"}]},{"id":889942,"number":4,"principalExclusionCriteria":"Major surgery within 3 months prior to Day 1 or planned surgery during Part 1 of the study.","principalExclusionCriteriaTranslations":[{"id":5950272,"uuid":"d081c9a2-e00a-4e39-86a7-1832a37960a7","attributeTranslation":"Cirugía mayor en el plazo de los 3 meses anteriores al día 1 o cirugía prevista durante la Parte 1 del estudio.","language":7,"languageDescription":"Spanish"},{"id":5950271,"uuid":"d081c9a2-e00a-4e39-86a7-1832a37960a7","attributeTranslation":"Intervention chirurgicale majeure dans les 3 mois avant le Jour1 ou prévision d'intervention chirurgicale pendant la Partie1","language":10,"languageDescription":"French"}]},{"id":889943,"number":5,"principalExclusionCriteria":"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 creates unnecessary risks to participate in the study in the opinion of the investigator.","principalExclusionCriteriaTranslations":[{"id":5950274,"uuid":"212364c0-3373-41e7-971c-3abb57e4a803","attributeTranslation":"Cualquier 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.","language":7,"languageDescription":"Spanish"},{"id":5950273,"uuid":"212364c0-3373-41e7-971c-3abb57e4a803","attributeTranslation":"Toute 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'investigateur","language":10,"languageDescription":"French"}]},{"id":889944,"number":6,"principalExclusionCriteria":"Known hypersensitivity to delandistrogene moxeparvovec or any excipients of the formulation.","principalExclusionCriteriaTranslations":[{"id":5950276,"uuid":"341de291-0861-4ec3-b4d9-7d4c1d4a4982","attributeTranslation":"Hipersensibilidad conocida al delandistrogene moxeparvovec o a cualquier excipiente de la formulación","language":7,"languageDescription":"Spanish"},{"id":5950275,"uuid":"341de291-0861-4ec3-b4d9-7d4c1d4a4982","attributeTranslation":"Hypersensibilité connue au delandistrogène moxeparvovec ou à l'un des excipients de la formulation","language":10,"languageDescription":"French"}]},{"id":889945,"number":7,"principalExclusionCriteria":"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 well-being or safety, or clinical interpretability.","principalExclusionCriteriaTranslations":[{"id":5950278,"uuid":"237da02c-aabe-4c98-ab60-5f349ef61d03","attributeTranslation":"Situation 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 participant","language":10,"languageDescription":"French"},{"id":5950277,"uuid":"237da02c-aabe-4c98-ab60-5f349ef61d03","attributeTranslation":"Proceso 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 clínica","language":7,"languageDescription":"Spanish"}]},{"id":889946,"number":8,"principalExclusionCriteria":"Left ventricular ejection fraction <50% on the screening echocardiogram (ECHO) or clinical signs and/or symptoms of cardiomyopathy","principalExclusionCriteriaTranslations":[{"id":5950279,"uuid":"e580165e-581b-458e-ada6-53a06e159f9a","attributeTranslation":"Fracción de eyección del ventrículo izquierdo <50% en el ecocardiograma de selección o signos y/o síntomas clínicos de miocardiopatía","language":7,"languageDescription":"Spanish"},{"id":5950280,"uuid":"e580165e-581b-458e-ada6-53a06e159f9a","attributeTranslation":"Fraction d'éjection ventriculaire gauche (FEVG) < 50 % sur l'échocardiographie de la sélection (ECHO) ou signes cliniques et/ou symptômes de cardiomyopathie","language":10,"languageDescription":"French"}]},{"id":889947,"number":9,"principalExclusionCriteria":"Known 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 1","principalExclusionCriteriaTranslations":[{"id":5950282,"uuid":"ca49eea1-bd4d-4bb2-9eed-b0cf7c1df1c5","attributeTranslation":"Contact 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 Jour1","language":10,"languageDescription":"French"},{"id":5950281,"uuid":"ca49eea1-bd4d-4bb2-9eed-b0cf7c1df1c5","attributeTranslation":"Contacto 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 1","language":7,"languageDescription":"Spanish"}]}]},"endPoint":{"primaryEndPoints":[{"id":601972,"number":1,"endPoint":"Incidence of treatment-emergent adverse events","isPrimary":true,"endPointTranslations":[{"id":5950234,"uuid":"2ebb8205-0916-4e3d-8e90-0b72f89bb3ae","attributeTranslation":"Incidence des événements indésirables apparaissant sous traitement","language":10,"languageDescription":"French"},{"id":5950233,"uuid":"2ebb8205-0916-4e3d-8e90-0b72f89bb3ae","attributeTranslation":"Incidencia de acontecimientos adversos surgidos durante el tratamiento","language":7,"languageDescription":"Spanish"}]},{"id":601973,"number":2,"endPoint":"Incidence of serious adverse events","isPrimary":true,"endPointTranslations":[{"id":5950236,"uuid":"d1a0c183-91a7-4c3f-832f-5ca54c02d6fa","attributeTranslation":"Incidence des événements indésirables graves","language":10,"languageDescription":"French"},{"id":5950235,"uuid":"d1a0c183-91a7-4c3f-832f-5ca54c02d6fa","attributeTranslation":"Incidencia de acontecimientos adversos graves","language":7,"languageDescription":"Spanish"}]},{"id":601974,"number":3,"endPoint":"Incidence of adverse events of special interest","isPrimary":true,"endPointTranslations":[{"id":5950237,"uuid":"af323b76-d4da-489c-a508-ac14ee7e9ffd","attributeTranslation":"Incidence des événements indésirables d'intérêt particulier","language":10,"languageDescription":"French"},{"id":5950238,"uuid":"af323b76-d4da-489c-a508-ac14ee7e9ffd","attributeTranslation":"Incidencia de acontecimientos adversos de especial interés","language":7,"languageDescription":"Spanish"}]},{"id":601975,"number":4,"endPoint":"Clinically significant changes in vital signs and physical examination findings","isPrimary":true,"endPointTranslations":[{"id":5950239,"uuid":"53c24277-52d4-497d-a31b-d264675c511d","attributeTranslation":"Modifications cliniquement significatives des signes vitaux et des résultats de l'examen clinique","language":10,"languageDescription":"French"},{"id":5950240,"uuid":"53c24277-52d4-497d-a31b-d264675c511d","attributeTranslation":"Cambios clínicamente significativos en las constantes vitales y los hallazgos de la exploración física","language":7,"languageDescription":"Spanish"}]},{"id":601976,"number":5,"endPoint":"Clinically significant changes in safety laboratory assessments, ECGs, and ECHOs","isPrimary":true,"endPointTranslations":[{"id":5950241,"uuid":"2788d968-18c9-48d2-b8dc-885024c2c29a","attributeTranslation":"Modifications cliniquement significatives des paramètres biologiques de sécurité, des électrocardiogrammes (ECG), des échocardiographies (ECHO)","language":10,"languageDescription":"French"},{"id":5950242,"uuid":"2788d968-18c9-48d2-b8dc-885024c2c29a","attributeTranslation":"Cambios clínicamente significativos en las determinaciones de laboratorio con fines de seguridad, ECG y ecocardiogramas","language":7,"languageDescription":"Spanish"}]}],"secondaryEndPoints":[{"id":601977,"number":1,"endPoint":"Change in quantity of SRP-9001-dystrophin protein expression from baseline to Week 12 as measured by Western blot","isPrimary":false,"endPointTranslations":[{"id":5950243,"uuid":"55c4505c-d765-4d10-b4b0-5c6a20088a0f","attributeTranslation":"Modifications de la quantité d'expression protéique de SRP-9001-dystrophine à la Semaine 12, d'après les résultats de l'analyse par Western blot","language":10,"languageDescription":"French"},{"id":5950244,"uuid":"55c4505c-d765-4d10-b4b0-5c6a20088a0f","attributeTranslation":"Cambio en la cantidad de expresión de la proteína distrofina SRP-9001 desde el momento basal hasta la semana 12, en su medición mediante Western 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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.","product":"Placebo for ATL1102 solution for injection, CD49d (alpha chain of VLA-4) Antisense|Oligonucleotide","ageRangeSecondary":["5","6"],"ageGroup":"0-17 years","gender":"Male","trialRegion":3,"totalNumberEnrolled":"1","endDate":"15/01/2025","primaryEndPoint":"Blinded 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).","resultsFirstReceived":"Yes","lastUpdated":"24/01/2025","lastPublicationUpdate":"13/08/2025"},{"ctNumber":"2024-512265-13-00","ctStatus":"Ended","startDateEU":"2023-04-13","endDateEU":"2025-01-14","decisionDate":"2024-07-30T08:45:03.201","publishDate":"2025-08-13T03:42:42.826260575","ctPublicStatusCode":8,"authorizedApplication":{"authorizedPartI":{"id":71590,"rowSubjectCount":47,"rowCountriesInfo":[{"eutctId":100000000329,"name":"Australia","isoNumber":36,"isoAlpha2Code":"AU","isoAlpha3Code":"AUS","current":true},{"eutctId":100000000549,"name":"Turkey","isoNumber":792,"isoAlpha2Code":"TR","isoAlpha3Code":"TUR","current":true},{"eutctId":100000000556,"name":"United Kingdom","isoNumber":826,"isoAlpha2Code":"GB","isoAlpha3Code":"GBR","current":true},{"eutctId":100000000518,"name":"Serbia","isoNumber":688,"isoAlpha2Code":"RS","isoAlpha3Code":"SRB","current":true}],"products":[{"id":292446,"part1MpRoleTypeCode":"1","productDictionaryInfo":{"productPharmForm":"N/A","euMpNumber":"N/A","marketingAuthNumber":"N/A","prodName":"Placebo for ATL1102 solution for injection","pharmForm":"N/A","activeSubstanceName":"N/A","euSubstNumber":"N/A","productOtherName":"N/A","authorisationCountryCode":"IS","nameOrg":"N/A","sponsorSubstanceCode":"N/A","atcCode":"N/A","atcName":"N/A","atcTermLevel":"N/A","activeSubstanceOtherDescriptiveName":"N/A"},"isPaediatricFormulation":false,"mpRoleInTrial":"3","timeUnitCode":"2","evCode":"N/A","miaNumber":"MIA(IMP) 20377","devices":[],"characteristics":[],"routes":[],"allSubstancesChemicals":false,"productName":"Placebo for ATL1102 solution for injection","jsonActiveSubstanceNames":"n/a","pharmaceuticalFormDisplay":"N/A"},{"id":292445,"part1MpRoleTypeCode":"1","productDictionaryInfo":{"productPk":"10395073","productPharmForm":"SOLUTION FOR INJECTION","euMpNumber":"PRD10395073","prodAuthStatus":1,"prodName":"CD49d (alpha chain of VLA-4) Antisense|Oligonucleotide","pharmForm":"SOLUTION FOR INJECTION","sponsorProductCode":"ATL1102","activeSubstanceName":"2'-O-(2-METHOXYETHYL) PHOSPHOROTHIOATE ANTISENSE OLIGONUCLEOTIDE TARGETING CD49D RNA","euSubstNumber":"SUB216749","productOtherName":"2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNA","nameOrg":"ANTISENSE THERAPEUTICS LIMITED","productSubstances":[{"productPk":"10395073","substancePk":"288699","nameOrg":"ANTISENSE THERAPEUTICS LIMITED","substanceOrigin":"Nucleic Acid","actSubstOrigin":"Nucleic Acid","actSubstName":"2'-O-(2-METHOXYETHYL) PHOSPHOROTHIOATE ANTISENSE OLIGONUCLEOTIDE TARGETING CD49D RNA","substanceEvCode":"SUB216749"}],"activeSubstanceOtherDescriptiveName":""},"isPaediatricFormulation":false,"mpRoleInTrial":"1","orphanDrugEdit":true,"orphanDrugDesigNumber":"EU/3/20/2375","doseUom":"mg milligram(s)","maxDailyDoseAmount":"50","doseUomTotal":"mg milligram(s)","maxTotalDoseAmount":"2500","maxTreatmentPeriod":49,"timeUnitCode":"2","otherMedicinalProduct":"Chemical","evCode":"PRD10395073","sponsorProductCodeEdit":"ATL1102","miaNumber":"MIA(IMP) 20377","devices":[],"characteristics":["11"],"routes":["SUBCUTANEOUS USE"],"allSubstancesChemicals":false,"productName":"CD49d (alpha chain of VLA-4) Antisense|Oligonucleotide","jsonActiveSubstanceNames":"2'-o-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting cd49d rna","pharmaceuticalFormDisplay":"SOLUTION FOR INJECTION"}],"trialDetails":{"clinicalTrialIdentifiers":{"fullTitle":"A 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 Dystrophy","fullTitleTranslations":[{"id":5179049,"uuid":"acd98563-e85f-4763-81f1-9f45f843b898","attributeTranslation":"Многоцентрово, 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.","language":1,"languageDescription":"Bulgarian"}],"publicTitle":"A 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 Dystrophy","publicTitleTranslations":[{"id":5179063,"uuid":"7922ab3b-e5a5-4222-af68-38662bd0acd6","attributeTranslation":"Многоцентрово, 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","language":1,"languageDescription":"Bulgarian"}],"shortTitle":"1102-DMD-Pre-CT03","secondaryIdentifyingNumbers":{"nctNumber":{"id":336892,"number":"NCT05938023"},"additionalRegistries":[{"id":336893,"number":"NCT05938023","otherRegistryName":"ANZCTR","ctRegistryCode":"6"}]}},"trialInformation":{"trialCategory":{"isLowIntervention":false,"trialPhase":"4","trialCategory":"2","justificationForTrialCategory":"This is a Phase II trial","trialCategoryId":63171},"medicalCondition":{"partIMedicalConditions":[{"id":72417,"medicalCondition":"Duchenne Muscular Dystrophy","medicalConditionTranslations":[{"id":5178995,"uuid":"77143e3c-ba54-41b3-928c-c0eabb3b75df","attributeTranslation":"Мускулна дистрофия на Дюшен","language":1,"languageDescription":"Bulgarian"}],"isConditionRareDisease":true}],"meddraConditionTerms":[{"termId":100000012544,"version":"20.0","level":"PT","termName":"Duchenne muscular dystrophy","classificationCode":"10013801","organClass":100000004850,"active":false}]},"trialObjective":{"trialScopes":[{"code":"6","trialScopeId":208452},{"code":"13","otherDescription":"Immunogenicity","trialScopeId":208448},{"code":"3","trialScopeId":208450},{"code":"5","trialScopeId":208446},{"code":"7","trialScopeId":208447},{"code":"4","trialScopeId":208449},{"code":"9","trialScopeId":208451}],"mainObjective":"Blinded 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.\nOLE phase: To evaluate the continued safety and tolerability of ATL1102 administered once weekly by subcutaneous injection, in non-ambulant participants with DMD.\nFor Primary Combined Blinded and OLE Phases objectives please see section 4.3 of Protocol","mainObjectiveTranslations":[{"id":5179062,"uuid":"6b38a91b-52b7-4e98-b741-784119ea0194","attributeTranslation":"Заслепена фаза:\nДа 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.\nОткрита фаза на продължение (OLE): \nДа се оцени продължаващата безопасност и поносимост на ATL1102, прилаган веднъж седмично чрез подкожна инжекция, при неамбулантни участници с МДД.\nЗа целите на първичните комбинирани заслепени и OLE фази, моля, вижте раздел 4.3 от протокола","language":1,"languageDescription":"Bulgarian"}],"secondaryObjectives":[{"id":235437,"number":1,"secondaryObjective":"Blinded phase: 1.To evaluate the effects of ATL1102 using several additional endpoints for assessment of muscle function, strength, respiratory function and Quality of Life","secondaryObjectiveTranslations":[{"id":5179064,"uuid":"6970d372-8309-4b76-8146-90b98b398f96","attributeTranslation":"Вторични цели:      \n Заслепена фаза: 1 1.\tДа се оценят ефектите на ATL1102 чрез няколко допълнителни крайни точки за оценка на мускулната функция и издръжливост, респираторната функция и качеството на живот","language":1,"languageDescription":"Bulgarian"}]},{"id":235438,"number":2,"secondaryObjective":"Blinded 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.","secondaryObjectiveTranslations":[{"id":5179065,"uuid":"72b88c85-60be-4492-9438-59c91ae9e435","attributeTranslation":"Да 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 спиране","language":1,"languageDescription":"Bulgarian"}]},{"id":235439,"number":3,"secondaryObjective":"Blinded 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.","secondaryObjectiveTranslations":[{"id":5179066,"uuid":"f3357490-a8a3-45b8-b467-f21506bb5c0b","attributeTranslation":"Да се оцени фармакокинетичният (ФК) профил на ATL1102 при различните дозови нива, прилаган веднъж седмично чрез подкожна (SC) инжекция при неамбулаторни участници с МДД","language":1,"languageDescription":"Bulgarian"}]},{"id":235440,"number":4,"secondaryObjective":"OLE 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.","secondaryObjectiveTranslations":[{"id":5179067,"uuid":"08edecf1-ea66-4ab1-9bf8-c84938cee921","attributeTranslation":"Открита фаза на продължение (OLE):\n1.\tДа 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).","language":1,"languageDescription":"Bulgarian"}]},{"id":235441,"number":5,"secondaryObjective":"OLE Phase: To evaluate the effects of ATL1102 using several additional endpoints for assessment of muscle function, strength, respiratory function and Quality of Life","secondaryObjectiveTranslations":[{"id":5179068,"uuid":"bab3b18e-06ec-45ed-9b90-2754aa487a0c","attributeTranslation":"Да се оценят ефектите на ATL1102 чрез няколко допълнителни крайни точки за оценка на мускулната функция и издръжливост, респираторната функция и качеството на живот","language":1,"languageDescription":"Bulgarian"}]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":457372,"number":1,"principalInclusionCriteria":"Has 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.","principalInclusionCriteriaTranslations":[{"id":5179050,"uuid":"acee0e20-f241-488d-b90e-a9d62c049d07","attributeTranslation":"Един участник ще бъде включен в това проучване само ако отговаря на всички от следващите критерии:\n1.\tИма 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":457373,"number":10,"principalInclusionCriteria":"Has 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).","principalInclusionCriteriaTranslations":[{"id":5179051,"uuid":"91175d45-3202-4850-b134-3c0abbb65c50","attributeTranslation":"Има 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).","language":1,"languageDescription":"Bulgarian"}]},{"id":457374,"number":11,"principalInclusionCriteria":"Participant 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.","principalInclusionCriteriaTranslations":[{"id":5179052,"uuid":"07e3c5e7-719e-4a7a-9389-7e2d7863bdd3","attributeTranslation":"Участникът, 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":457375,"number":12,"principalInclusionCriteria":"Participant and their parent/guardian are willing and able to comply with scheduled visits, study medication administration and study procedures.","principalInclusionCriteriaTranslations":[{"id":5179053,"uuid":"beb35728-108d-4f1d-8c47-067a3028a3ef","attributeTranslation":"Участникът и неговият родител/настойник/болногледач желаят и са в състояние да спазват планираните визити, приложението на изпитваното лекарство и процедурите по проучването.","language":1,"languageDescription":"Bulgarian"}]},{"id":457376,"number":2,"principalInclusionCriteria":"Where 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.","principalInclusionCriteriaTranslations":[{"id":5179054,"uuid":"f295ce4e-24e9-45d3-ac7f-3a53482e33fc","attributeTranslation":"Когато 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":457377,"number":3,"principalInclusionCriteria":"Has a clinical diagnosis of DMD confirmed by validated genetic testing (i.e., documented deletion, duplication or point mutation in the dystrophin gene).","principalInclusionCriteriaTranslations":[{"id":5179055,"uuid":"0cbe1af7-1bff-46ea-954d-e6b7f9ffeb48","attributeTranslation":"Има клинична диагноза за МДД, потвърдена чрез валидирано генетично изследване (т.е. документирана делеция, дублиране или точкова мутация в гена на дистрофина).","language":1,"languageDescription":"Bulgarian"}]},{"id":457378,"number":4,"principalInclusionCriteria":"Is considered to be non-ambulatory, defined as unable to walk 10 meters without assistance or help at Screening","principalInclusionCriteriaTranslations":[{"id":5179056,"uuid":"6ac5ca89-0179-4af2-b0e4-b7c8b085a84d","attributeTranslation":"Счита се за неамбулаторен, определен като неспособен да ходи 10 метра без съдействие или помощ при Скрининг.","language":1,"languageDescription":"Bulgarian"}]},{"id":457379,"number":5,"principalInclusionCriteria":"Male aged 10 to less than 18 years, at the time of screening and informed consent for participation in the study.","principalInclusionCriteriaTranslations":[{"id":5179057,"uuid":"0fba6efa-33b0-406e-88dd-7f0e4e1901da","attributeTranslation":"Участник от мъжки пол на възраст от 10 до под 18 години към момента на Скрининг и информирано съгласие за участие в проучването.","language":1,"languageDescription":"Bulgarian"}]},{"id":457380,"number":6,"principalInclusionCriteria":"Body weight of at least 25 kg at Screening.","principalInclusionCriteriaTranslations":[{"id":5179058,"uuid":"77f934a1-83a1-4253-a874-0734e05cc790","attributeTranslation":"Телесно тегло най-малко 25 кг при Скрининг.","language":1,"languageDescription":"Bulgarian"}]},{"id":457381,"number":7,"principalInclusionCriteria":"If 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.","principalInclusionCriteriaTranslations":[{"id":5179059,"uuid":"915961bd-3a78-4191-bce7-85126db781fd","attributeTranslation":"Ако 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":457382,"number":8,"principalInclusionCriteria":"On 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.","principalInclusionCriteriaTranslations":[{"id":5179060,"uuid":"8a2aa81e-50c3-4ed1-bc6d-2b1a3221a359","attributeTranslation":"При 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).","language":1,"languageDescription":"Bulgarian"}]},{"id":457383,"number":9,"principalInclusionCriteria":"Able 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.","principalInclusionCriteriaTranslations":[{"id":5179061,"uuid":"5945a556-0e39-4568-93cd-3f849d4adebd","attributeTranslation":"Способен 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.","language":1,"languageDescription":"Bulgarian"}]}],"principalExclusionCriteria":[{"id":787346,"number":1,"principalExclusionCriteria":"A 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.","principalExclusionCriteriaTranslations":[{"id":5179028,"uuid":"20656c7a-87ac-4559-b62c-826e624fe221","attributeTranslation":"Критерии за изключване\nВсеки участник, който отговаря на някой от следните критерии, ще бъде изключен от участие в проучването:\n1.\tУчастие 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":787347,"number":10,"principalExclusionCriteria":"Known history of or a positive test for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibodies, human immunodeficiency virus (HIV) antibodies at Screening.","principalExclusionCriteriaTranslations":[{"id":5179029,"uuid":"39286e08-6085-40b1-b801-08fc001a724e","attributeTranslation":"Известна 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":787348,"number":11,"principalExclusionCriteria":"Evidence of renal impairment and/or cystatin C >1.4 mg/L.","principalExclusionCriteriaTranslations":[{"id":5179030,"uuid":"979d4454-8150-4615-a1b3-8bc630ba1bb8","attributeTranslation":"Доказателство за бъбречно увреждане и/или цистатин C > 1,4 mg/l.","language":1,"languageDescription":"Bulgarian"}]},{"id":787349,"number":12,"principalExclusionCriteria":"Received a live vaccine (including intranasal influenza vaccine) within 4 weeks prior to Day 1 or planned live vaccination during the study period.","principalExclusionCriteriaTranslations":[{"id":5179031,"uuid":"0f127444-8fe9-468f-a307-c498e5c1f5d0","attributeTranslation":"Получена жива ваксина (включително интраназална противогрипна ваксина) в рамките на 4 седмици преди Ден 1 или планирана жива ваксина по време на периода на проучването.","language":1,"languageDescription":"Bulgarian"}]},{"id":787350,"number":13,"principalExclusionCriteria":"Planned or expected surgery during the study period (as judged by the Investigator).","principalExclusionCriteriaTranslations":[{"id":5179032,"uuid":"0dc1eb27-a6e3-46e9-b9dd-3d9e56b10767","attributeTranslation":"Планирана или очаквана операция по време на периода на проучването (по преценка на Изследователя).","language":1,"languageDescription":"Bulgarian"}]},{"id":787351,"number":14,"principalExclusionCriteria":"Asthma (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.","principalExclusionCriteriaTranslations":[{"id":5179033,"uuid":"260a2b00-12fb-47fa-b55a-7ca5de611c5b","attributeTranslation":"Астма (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.","language":1,"languageDescription":"Bulgarian"}]},{"id":787352,"number":15,"principalExclusionCriteria":"Requires day-time assisted mechanical or non-invasive ventilation (NIV) (night-time NIV is permitted).","principalExclusionCriteriaTranslations":[{"id":5179034,"uuid":"321427fd-7274-4e0c-9e6c-d8b1658a0e77","attributeTranslation":"Изисква механична или неинвазивна вентилация (non-invasive ventilation, NIV) през деня (NIV през нощта е разрешена).","language":1,"languageDescription":"Bulgarian"}]},{"id":787353,"number":16,"principalExclusionCriteria":"Unable to form a mouth seal to allow precise respiratory flow measurements and mouth pressure.","principalExclusionCriteriaTranslations":[{"id":5179035,"uuid":"0306bd95-2cc9-4b8c-8a89-f6490fc8670b","attributeTranslation":"Не може да затвори плътно с устата, което да позволи прецизно измерване на дихателния поток и налягане в устата.","language":1,"languageDescription":"Bulgarian"}]},{"id":787354,"number":17,"principalExclusionCriteria":"Chronic 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).","principalExclusionCriteriaTranslations":[{"id":5179036,"uuid":"3d64dedc-f59d-4b2a-9f11-f68ad2635b33","attributeTranslation":"Хронично 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).","language":1,"languageDescription":"Bulgarian"}]},{"id":787355,"number":18,"principalExclusionCriteria":"Used 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.","principalExclusionCriteriaTranslations":[{"id":5179037,"uuid":"7a10b595-d07e-49c1-b708-01dc3aef5ffa","attributeTranslation":"Използване 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.\nИзползвани аталурен, етеплирсен, казимерсен, голодирсен (или друг екзон\nпропускане на антисенс олигонуклеотидни лекарства) и ваморолон в рамките на 6\nмесеца преди Ден 1.\nИзползвано системно лечение с имуносупресори (напр. митоксантрон,\nазатиоприн, метотрексат, циклофосфамид, микофенолат, TNFɑ\nинхибитори) в рамките на 3 месеца преди Ден 1.\nИзползван интравенозен имуноглобулин (IVIg) в рамките на 6 месеца преди Ден 1.\nВижте раздел 7.6.2 Забранени медикаменти.","language":1,"languageDescription":"Bulgarian"}]},{"id":787356,"number":19,"principalExclusionCriteria":"Other 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.","principalExclusionCriteriaTranslations":[{"id":5179038,"uuid":"67bd9d70-d128-40a5-a1cc-9324e8a18dbd","attributeTranslation":"Освен 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":787357,"number":2,"principalExclusionCriteria":"Exposure to more than 3 investigational products within the 12 months prior to Day 1.","principalExclusionCriteriaTranslations":[{"id":5179039,"uuid":"7b68469f-6a62-453b-8889-7007b8cdb3d4","attributeTranslation":"Експозиция на повече от 3 изпитвани лекарства в рамките на 12 месеца преди Ден 1.","language":1,"languageDescription":"Bulgarian"}]},{"id":787358,"number":20,"principalExclusionCriteria":"Has an increased risk for opportunistic infections or systemic medical conditions resulting in significantly compromised immune system function (e.g., HIV, organ transplant, active malignancy).","principalExclusionCriteriaTranslations":[{"id":5179040,"uuid":"f1f25f7d-8e5d-428d-821f-64919f0a08a0","attributeTranslation":"Има повишен риск от опортюнистични инфекции или системни медицински състояния, водещи до значително нарушена функция на имунната система (напр. HIV, органна трансплантация, активно злокачествено заболяване).","language":1,"languageDescription":"Bulgarian"}]},{"id":787359,"number":21,"principalExclusionCriteria":"An 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.","principalExclusionCriteriaTranslations":[{"id":5179041,"uuid":"a46bfd1c-5557-46f6-9fbc-8f3aef8ef7ed","attributeTranslation":"Служител 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":787360,"number":3,"principalExclusionCriteria":"Has 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.","principalExclusionCriteriaTranslations":[{"id":5179042,"uuid":"0f039b21-4b55-4aa3-8c5e-3662d65129cc","attributeTranslation":"Има следните абнормни хематологични стойности по време на периода на Скрининг:\na.\tЛимфоцити < 1,2 x 109/L\nb.\tНеутрофили < 1,8 x 109/L \nc.\tТромбоцити < 150 x 109/L.","language":1,"languageDescription":"Bulgarian"}]},{"id":787361,"number":4,"principalExclusionCriteria":"Has 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.","principalExclusionCriteriaTranslations":[{"id":5179043,"uuid":"ca888afa-72ef-454c-8fec-d06ae59c19a1","attributeTranslation":"Има следните стойности от чернодробен функционален тест по време на периода на Скрининг:\na.\tНива на гама-глутамил транспептидаза (GGT) > 2,0 x горната граница на нормата (ГГН) или\nb.\tКонцентрации на общ билирубин над 1,5 x ГГН или\nc.\tНива на глутамат дехидрогеназа (Glutamate dehydrogenase, GLDH), по-високи от 2,0 x ГГН.","language":1,"languageDescription":"Bulgarian"}]},{"id":787362,"number":5,"principalExclusionCriteria":"History of clinically significant bleeding or coagulation abnormalities.","principalExclusionCriteriaTranslations":[{"id":5179044,"uuid":"5deb7aa9-2b44-4d3e-8844-f6c4c0a9373b","attributeTranslation":"Анамнеза за клинично значимо кървене или коагулационни аномалии.","language":1,"languageDescription":"Bulgarian"}]},{"id":787363,"number":6,"principalExclusionCriteria":"Clinically significant abnormal coagulation parameters.","principalExclusionCriteriaTranslations":[{"id":5179045,"uuid":"f53ae942-53bb-4f8e-a3fd-5deb9172c256","attributeTranslation":"Клинично значими абнормни параметри на коагулация.","language":1,"languageDescription":"Bulgarian"}]},{"id":787364,"number":7,"principalExclusionCriteria":"Currently receiving antiplatelet or anticoagulant therapy or has taken medication with an antiplatelet or anticoagulant effect within 4 weeks prior Day 1 (e.g., aspirin).","principalExclusionCriteriaTranslations":[{"id":5179046,"uuid":"ed4abd53-0d38-46c5-9dde-d0d4c202e74f","attributeTranslation":"Текущ 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).","language":1,"languageDescription":"Bulgarian"}]},{"id":787365,"number":8,"principalExclusionCriteria":"History of hypersensitivity to ATL1102 or to any of the excipients in the ATL1102 drug product formulation.","principalExclusionCriteriaTranslations":[{"id":5179047,"uuid":"ca3ab639-e3ad-41fc-a387-98e0d88120e4","attributeTranslation":"Анамнеза за свръхчувствителност към ATL1102 или към някое от помощните вещества в лекарствения продукт ATL1102.","language":1,"languageDescription":"Bulgarian"}]},{"id":787366,"number":9,"principalExclusionCriteria":"Any 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).","principalExclusionCriteriaTranslations":[{"id":5179048,"uuid":"78df00db-bf97-4b05-96a3-25e8859f5de6","attributeTranslation":"Всяко 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).","language":1,"languageDescription":"Bulgarian"}]}]},"endPoint":{"primaryEndPoints":[{"id":531815,"number":1,"endPoint":"Blinded Phase: Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25.","isPrimary":true,"endPointTranslations":[{"id":5178996,"uuid":"e7f29f65-f7e7-4675-96cf-af0c06994685","attributeTranslation":"Заслепена фаза: Промяна в скора за Функция на горните крайници (PUL 2.0) от базовото ниво до Седмица 25.","language":1,"languageDescription":"Bulgarian"}]},{"id":531816,"number":2,"endPoint":"OLE Phase (Efficacy): Change of Performance of Upper Limb (PUL 2.0) score from Week 25 to Week 49.","isPrimary":true,"endPointTranslations":[{"id":5178997,"uuid":"8bc841d0-23ba-449c-9db4-05153898d30a","attributeTranslation":"OLE фаза: Промяна в скора за Функция на горните крайници (PUL 2.0) от Седмица 25 до Седмица 49.","language":1,"languageDescription":"Bulgarian"}]},{"id":531817,"number":3,"endPoint":"OLE Phase (Safety): 1.Frequency and severity of AEs, SAEs and SUSARs.","isPrimary":true,"endPointTranslations":[{"id":5178998,"uuid":"20c0d12d-7364-4333-9af4-adf69c5a3ab5","attributeTranslation":"OLE фаза: (Параметри за безопасност):\n1.\tЧестота и тежест на нежелани събития (НС), Сериозни нежелани събития (СНС) и Подозирани неочаквани сериозни нежелани събития (ПНСНР).","language":1,"languageDescription":"Bulgarian"}]},{"id":531818,"number":4,"endPoint":"OLE Phase (Safety): 2.Clinical Laboratory Tests i.Haematology ii.Coagulation (including fibrinogen) iii.Complement iv.Biochemistry (including haptoglobin) v.Urinalysis (including weekly dipsticks)","isPrimary":true,"endPointTranslations":[{"id":5178999,"uuid":"6c116385-47de-4626-a117-158c9cc05434","attributeTranslation":"2.\tКлинични лабораторни изследвания\ni.\tХематология \nii.\tКоагулация (включително фибриноген)\niii.\tКомплемент\niv.\tБиохимия (включително хаптоглобин)\nv.\tАнализ на урина (включително ежеседмични тест-ленти).","language":1,"languageDescription":"Bulgarian"}]},{"id":531819,"number":5,"endPoint":"OLE Phase (Safety): 3.Cardiac function (ECG, echocardiogram)","isPrimary":true,"endPointTranslations":[{"id":5179000,"uuid":"71ea482d-db22-4d9a-91f3-92e1495cfeb2","attributeTranslation":"3.\tСърдечна функция (ЕКГ, ехокардиограма).","language":1,"languageDescription":"Bulgarian"}]},{"id":531820,"number":6,"endPoint":"OLE Phase (Safety): 4.Respiratory function (as above in efficacy)","isPrimary":true,"endPointTranslations":[{"id":5179001,"uuid":"e220d80e-e98a-4831-b1af-61791a1836bc","attributeTranslation":"4.\tРеспираторна функция (както е посочено по-горе по отношение на ефикасността).","language":1,"languageDescription":"Bulgarian"}]},{"id":531821,"number":7,"endPoint":"OLE Phase (Safety): 5.Physical examination","isPrimary":true,"endPointTranslations":[{"id":5179002,"uuid":"1ce6b7e3-0e8a-4a40-a813-cb2fafc88e7f","attributeTranslation":"5.\tФизикален преглед.","language":1,"languageDescription":"Bulgarian"}]},{"id":531822,"number":8,"endPoint":"OLE Phase (Safety): 6.Vital Signs (blood pressure, heart rate, respiratory rate, tympanic temperature)","isPrimary":true,"endPointTranslations":[{"id":5179003,"uuid":"90144129-d2a2-4182-b3d8-30d4bb8e1f54","attributeTranslation":"6.\tЖизнени показатели (кръвно налягане, пулс, респираторна честота, тимпанична   температура).","language":1,"languageDescription":"Bulgarian"}]},{"id":531823,"number":9,"endPoint":"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.","isPrimary":true,"endPointTranslations":[{"id":5179004,"uuid":"f3190a50-8452-4d22-a126-f640751155f4","attributeTranslation":"7.\tСъбития, свързани с Плана за наблюдение на безопасността и Правилата за спиране\ni.\tБрой, 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.\nii.\tрБрой, 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":531824,"number":10,"endPoint":"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.","isPrimary":true,"endPointTranslations":[{"id":5179005,"uuid":"0a464fbe-66fb-41ed-bc4e-651776786c0b","attributeTranslation":"Комбинирани заслепени и OLE фази:\n1. Промяна на производителността на\nРезултат за горен крайник (PUL 2.0) от изходното ниво до седмица 25 в сравнение с\nпромяна от седмица 25 на седмица 49.","language":1,"languageDescription":"Bulgarian"}]},{"id":531825,"number":11,"endPoint":"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).","isPrimary":true,"endPointTranslations":[{"id":5179006,"uuid":"b6de508d-84c9-45ef-81d7-cd4537e6243c","attributeTranslation":"2. Промяна на показателя за ефективност на горния крайник (PUL 2.0) от изходното ниво до\nСедмица 49 (за тези, които са останали на същата доза за лечение с ATL1102\nпрез заслепените и OLE фазите на изследването).","language":1,"languageDescription":"Bulgarian"}]}],"secondaryEndPoints":[{"id":531826,"number":1,"endPoint":"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).","isPrimary":false,"endPointTranslations":[{"id":5179007,"uuid":"0e7325da-8bb9-4554-81f5-3eda57ffac81","attributeTranslation":"Заслепена фаза (ефикасност):\n1.\tПроцент 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).","language":1,"languageDescription":"Bulgarian"}]},{"id":531827,"number":2,"endPoint":"Blinded Phase (Efficacy) 2.Change in muscle strength as assessed by percent predicted MyoGrip (using the Myoset System) from baseline to Week 25.","isPrimary":false,"endPointTranslations":[{"id":5179008,"uuid":"b10b7e76-1ea0-4f0d-98af-4fb2399412de","attributeTranslation":"2.\tПромяна в мускулната издръжливост, оценена чрез процент прогнозиран MyoGrip (с помощта на системата Myoset) от базовото ниво до Седмица 25.","language":1,"languageDescription":"Bulgarian"}]},{"id":531828,"number":3,"endPoint":"Blinded Phase (Efficacy) 3.Change in muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from baseline to Week 25.","isPrimary":false,"endPointTranslations":[{"id":5179009,"uuid":"60b1f173-8247-46bc-acec-c0dcbb88a2ad","attributeTranslation":"3.\tПромяна в мускулната издръжливост, оценена чрез процент прогнозиран MyoPinch (с помощта на системата Myoset) от базовото ниво до седмица 25.","language":1,"languageDescription":"Bulgarian"}]},{"id":531829,"number":4,"endPoint":"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. 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(IHQ).","language":7,"languageDescription":"Spanish"},{"id":6861473,"uuid":"e0a02283-23e3-4b6c-a519-317c3b8a9f4e","attributeTranslation":"Двойно сляп период: оценка на ефекта на SRP-4045 и SRP-4053 (комбинирана активна група) по отношение на:\n•\tЕкспресия на белтъка дистрофин в биопсична мускулна тъкан, измерена чрез: \n-\tТест Western blot (количествено определяне)\n-\tИнтензитет на фибрите, определен чрез имунохистохимия (IHC)","language":1,"languageDescription":"Bulgarian"}]},{"id":302898,"number":2,"secondaryObjective":"Functional status as measured by:  • 6MWT • 10MWR • 4-step ascend velocity • Rise from floor velocity  •  North Star Ambulatory Assessment (NSAA)","secondaryObjectiveTranslations":[{"id":6861477,"uuid":"73c58d57-6d69-44cd-a154-5f84e3d2e0fe","attributeTranslation":"funkcjonalny 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)","language":19,"languageDescription":"Polish"},{"id":6861476,"uuid":"73c58d57-6d69-44cd-a154-5f84e3d2e0fe","attributeTranslation":"El estado funcional determinado mediante:\n• la prueba de marcha de seis minutos (PM6M)\n• la prueba de caminar/correr 10 metros\n• la velocidad para subir 4 escalones\n• la velocidad para levantarse del suelo\n• la evaluación ambulatoria North Star (NSAA)","language":7,"languageDescription":"Spanish"},{"id":6861479,"uuid":"73c58d57-6d69-44cd-a154-5f84e3d2e0fe","attributeTranslation":"-Funkcionális állapot a következők szerint: \n- 6 perces járásteszt (6MWT)\n- 10 méteres járásteszt (10MWR)\n- 4 lépéses emelkedési sebesség\n- Talajról való felállás sebessége\n- North Star ambuláns vizsgálat (NSAA)","language":13,"languageDescription":"Hungarian"},{"id":6861478,"uuid":"73c58d57-6d69-44cd-a154-5f84e3d2e0fe","attributeTranslation":"Функционален статус, измерен чрез:\n- 6 -минутен тест за ходете\n-  10 метра ходене или тичане\n- Скорост на изкачване на 4 стъпала\n- Скорост на ставане от пода\n- Оценка на подвижността на пациента по системата North Star (NSAA)","language":1,"languageDescription":"Bulgarian"}]},{"id":302899,"number":3,"secondaryObjective":"Double-blind period: evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) on: •\tSafety and tolerability of SRP-4045 and SRP-4053.","secondaryObjectiveTranslations":[{"id":6861480,"uuid":"d474e63d-cf84-46bf-80d4-41dd9c7636dd","attributeTranslation":"Двойно сляп период: оценка на ефекта на SRP-4045 и SRP-4053 (комбинирана активна група) по отношение на: •\tБезопасност и поносимост на SRP-4045 и SRP-4053.","language":1,"languageDescription":"Bulgarian"},{"id":6861483,"uuid":"d474e63d-cf84-46bf-80d4-41dd9c7636dd","attributeTranslation":"Kettős vak szakasz: az SRP-4045 és SRP-4053 (kombinált aktív csoport) hatásának értékelése az alábbiak szempontjából: •\tAz SRP-4045 és SRP-4053 biztonságossága és tolerálhatósága.","language":13,"languageDescription":"Hungarian"},{"id":6861482,"uuid":"d474e63d-cf84-46bf-80d4-41dd9c7636dd","attributeTranslation":"Periodo doble ciego: evaluar el efecto de SRP-4045 y SRP-4053 (combinados, grupo de principio activo) sobre •\tLa seguridad y la tolerabilidad de SRP-4045 y SRP-4053.","language":7,"languageDescription":"Spanish"},{"id":6861481,"uuid":"d474e63d-cf84-46bf-80d4-41dd9c7636dd","attributeTranslation":"okres leczenia w fazie podwójnie zaślepionej: ocena wpływu SRP 4045 oraz SRP 4053 (grupa pacjentów z którymkolwiek rodzajem leczenia czynnego) na: •\tbezpieczeństwo stosowania i tolerancję SRP 4045 i SRP 4053.","language":19,"languageDescription":"Polish"}]},{"id":302900,"number":4,"secondaryObjective":"Open-label Treatment Period:  •\tEvaluate the long-term effects of SRP-4045 and SRP-4053 treatment on functional status up to 144 weeks.","secondaryObjectiveTranslations":[{"id":6861485,"uuid":"3a03e2c0-5eac-4eb2-9a28-ef6795551fa7","attributeTranslation":"Periodo abierto de tratamiento del estudio: •\tEvaluar los efectos a largo plazo del tratamiento con SRP-4045 y SRP-4053 en el estado funcional de los pacientes hasta las 144 semanas","language":7,"languageDescription":"Spanish"},{"id":6861486,"uuid":"3a03e2c0-5eac-4eb2-9a28-ef6795551fa7","attributeTranslation":"Период на открито лечение: •\tОценка на дългосрочните ефекти на лечението със SRP-4045 и SRP-4053 върху функционалния статус в продължение на до 144 седмици.","language":1,"languageDescription":"Bulgarian"},{"id":6861487,"uuid":"3a03e2c0-5eac-4eb2-9a28-ef6795551fa7","attributeTranslation":"Okres leczenia w fazie otwartej: •\tocena długoterminowego wpływu leczenia SRP 4045 oraz SRP 4053 na zaburzenia czynnościowe w okresie do 144 tygodni;","language":19,"languageDescription":"Polish"},{"id":6861484,"uuid":"3a03e2c0-5eac-4eb2-9a28-ef6795551fa7","attributeTranslation":"Nyílt kezelési szakasz: •\tAz SRP-4045 és SRP-4053 kezelés funkcionális státuszra gyakorolt hosszú távú hatásainak értékelése a 144. hét végéig.","language":13,"languageDescription":"Hungarian"}]},{"id":302901,"number":5,"secondaryObjective":"Open-label Treatment Period:  •\tEvaluate the long-term safety and tolerability of SRP-4045 and SRP-4053.","secondaryObjectiveTranslations":[{"id":6861490,"uuid":"71d23544-de8b-48f1-9a19-9fc585d8e293","attributeTranslation":"Okres leczenia w fazie otwartej: •\tocena długoterminowa bezpieczeństwa stosowania i tolerancji na leczenie SRP 4045 oraz SRP 4053;","language":19,"languageDescription":"Polish"},{"id":6861491,"uuid":"71d23544-de8b-48f1-9a19-9fc585d8e293","attributeTranslation":"Nyílt kezelési szakasz: •\tAz SRP-4045 és SRP-4053 hosszú távú biztonságosságának és tolerálhatóságának értékelése.","language":13,"languageDescription":"Hungarian"},{"id":6861488,"uuid":"71d23544-de8b-48f1-9a19-9fc585d8e293","attributeTranslation":"Период на открито лечение: •\tОценка на дългосрочната безопасност и поносимост на SRP-4045 и SRP-4053.","language":1,"languageDescription":"Bulgarian"},{"id":6861489,"uuid":"71d23544-de8b-48f1-9a19-9fc585d8e293","attributeTranslation":"Periodo abierto de tratamiento del estudio: •\tEvaluar la seguridad y la tolerabilidad a largo plazo de SRP-4045 y SRP-4053","language":7,"languageDescription":"Spanish"}]},{"id":302902,"number":6,"secondaryObjective":"Pharmacokinetic Objective: Evaluate the PK properties of SRP-4045 and SRP-4053 using a population PK model.","secondaryObjectiveTranslations":[{"id":6861494,"uuid":"231622a7-1744-4da6-abb4-1f3d871c7310","attributeTranslation":"Objetivo farmacocinético:\nEvaluar las propiedades FC de SRP-4045 y SRP-4053 mediante un modelo farmacocinético poblacional.","language":7,"languageDescription":"Spanish"},{"id":6861495,"uuid":"231622a7-1744-4da6-abb4-1f3d871c7310","attributeTranslation":"Cel farmakokinetyczny:\nocena własności farmakokinetycznych SRP 4045 oraz SRP 4053 z wykorzystaniem modelu farmakokinetyki populacyjnej.","language":19,"languageDescription":"Polish"},{"id":6861492,"uuid":"231622a7-1744-4da6-abb4-1f3d871c7310","attributeTranslation":"Фармакокинетична цел:\nОценка на ФК свойствата на SRP-4045 и SRP-4053 с помощта на популационен ФК модел.","language":1,"languageDescription":"Bulgarian"},{"id":6861493,"uuid":"231622a7-1744-4da6-abb4-1f3d871c7310","attributeTranslation":"Farmakokinetikai célkitűzés:\nAz SRP-4045 és SRP-4053 PK jellemzőinek értékelése populációs PK modell segítségével.","language":13,"languageDescription":"Hungarian"}]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":584532,"number":1,"principalInclusionCriteria":"Is 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.","principalInclusionCriteriaTranslations":[{"id":6861426,"uuid":"24a16e3e-902b-465a-8480-43be77227784","attributeTranslation":"pacjent płci męskiej z potwierdzonym rozpoznaniem dystrofii mięśniowej Duchenne’a oraz delecją zmieniającą ramkę odczytu, odpowiedzialną za:\n• 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\n• 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);\nzgodnie 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 dawki","language":19,"languageDescription":"Polish"},{"id":6861425,"uuid":"24a16e3e-902b-465a-8480-43be77227784","attributeTranslation":"Fiú DMD megállapított klinikai diagnózisával és out-of-frame delécióval, amely befolyásolható az alábbiakkal:\n• 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\n• 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)\nAkkreditált 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.","language":13,"languageDescription":"Hungarian"},{"id":6861424,"uuid":"24a16e3e-902b-465a-8480-43be77227784","attributeTranslation":"Ser varón con un diagnóstico clínico confirmado de DMD y una deleción fuera del marco de lectura susceptible de:\n• 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\n• 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)\ndocumentada 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.","language":7,"languageDescription":"Spanish"},{"id":6861427,"uuid":"24a16e3e-902b-465a-8480-43be77227784","attributeTranslation":"Е от мъжки пол с установена клинична диагноза МДД и делеция извън рамката, податлив на:\n• 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\n• 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)\nДокументирано 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 генотипа","language":1,"languageDescription":"Bulgarian"}]},{"id":584533,"number":10,"principalInclusionCriteria":"Is 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.","principalInclusionCriteriaTranslations":[{"id":6861430,"uuid":"bbfea091-2093-4e8a-860b-3692b1ac4516","attributeTranslation":"Estar 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.","language":7,"languageDescription":"Spanish"},{"id":6861431,"uuid":"bbfea091-2093-4e8a-860b-3692b1ac4516","attributeTranslation":"Kész 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.","language":13,"languageDescription":"Hungarian"},{"id":6861428,"uuid":"bbfea091-2093-4e8a-860b-3692b1ac4516","attributeTranslation":"pacjent 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).","language":19,"languageDescription":"Polish"},{"id":6861429,"uuid":"bbfea091-2093-4e8a-860b-3692b1ac4516","attributeTranslation":"Желае 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":584534,"number":2,"principalInclusionCriteria":"Is 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.","principalInclusionCriteriaTranslations":[{"id":6861433,"uuid":"ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6b","attributeTranslation":"Para 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).","language":7,"languageDescription":"Spanish"},{"id":6861432,"uuid":"ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6b","attributeTranslation":"Az 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.","language":13,"languageDescription":"Hungarian"},{"id":6861434,"uuid":"ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6b","attributeTranslation":"w 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.","language":19,"languageDescription":"Polish"},{"id":6861435,"uuid":"ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6b","attributeTranslation":"Е 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.","language":1,"languageDescription":"Bulgarian"}]},{"id":584535,"number":3,"principalInclusionCriteria":"Has 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.","principalInclusionCriteriaTranslations":[{"id":6861439,"uuid":"291b79a5-d06c-4f8e-885a-04acdafb0858","attributeTranslation":"czynności 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.","language":19,"languageDescription":"Polish"},{"id":6861437,"uuid":"291b79a5-d06c-4f8e-885a-04acdafb0858","attributeTranslation":"Légzésfunkciója 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.","language":13,"languageDescription":"Hungarian"},{"id":6861438,"uuid":"291b79a5-d06c-4f8e-885a-04acdafb0858","attributeTranslation":"Има 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.","language":1,"languageDescription":"Bulgarian"},{"id":6861436,"uuid":"291b79a5-d06c-4f8e-885a-04acdafb0858","attributeTranslation":"Presentar 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.","language":7,"languageDescription":"Spanish"}]},{"id":584536,"number":4,"principalInclusionCriteria":"Has intact right and left biceps brachii muscles (the preferred biopsy site) or 2 alternative upper arm muscle groups.","principalInclusionCriteriaTranslations":[{"id":6861440,"uuid":"583cb2b1-0b11-4e81-a6f4-c859399aa25c","attributeTranslation":"A jobb és bal musculus biceps brachii (a preferált biopsziás hely) vagy 2 másik felkari izomcsoport intakt.","language":13,"languageDescription":"Hungarian"},{"id":6861442,"uuid":"583cb2b1-0b11-4e81-a6f4-c859399aa25c","attributeTranslation":"Има непокътнати десен и ляв двуглав мишничен мускул (предпочитано място за биопсия) или 2 алтернативни мускулни групи в горната част на ръката.","language":1,"languageDescription":"Bulgarian"},{"id":6861443,"uuid":"583cb2b1-0b11-4e81-a6f4-c859399aa25c","attributeTranslation":"Presentar los músculos bíceps braquiales izquierdo y derecho (zona preferida para la biopsia) o 2 grupos de músculos del brazo alternativos intactos.","language":7,"languageDescription":"Spanish"},{"id":6861441,"uuid":"583cb2b1-0b11-4e81-a6f4-c859399aa25c","attributeTranslation":"musi mieć nienaruszony prawy i lewy mięsień dwugłowy ramienia (preferowane miejsce biopsji) lub dwie inne grupy mięśniowe w obrębie kończyny górnej.","language":19,"languageDescription":"Polish"}]},{"id":584537,"number":5,"principalInclusionCriteria":"Has 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)","principalInclusionCriteriaTranslations":[{"id":6861445,"uuid":"13e66846-08a7-4309-b608-5174d5d186cf","attributeTranslation":"Változatlan 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)","language":13,"languageDescription":"Hungarian"},{"id":6861447,"uuid":"13e66846-08a7-4309-b608-5174d5d186cf","attributeTranslation":"Е 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)","language":1,"languageDescription":"Bulgarian"},{"id":6861446,"uuid":"13e66846-08a7-4309-b608-5174d5d186cf","attributeTranslation":"Haber 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).","language":7,"languageDescription":"Spanish"},{"id":6861444,"uuid":"13e66846-08a7-4309-b608-5174d5d186cf","attributeTranslation":"otrzymywał 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.","language":19,"languageDescription":"Polish"}]},{"id":584538,"number":6,"principalInclusionCriteria":"If 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).","principalInclusionCriteriaTranslations":[{"id":6861449,"uuid":"eb7a5126-13fb-4264-8b8c-686809e6d362","attributeTranslation":"jeżeli 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).","language":19,"languageDescription":"Polish"},{"id":6861451,"uuid":"eb7a5126-13fb-4264-8b8c-686809e6d362","attributeTranslation":"Ha 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).","language":13,"languageDescription":"Hungarian"},{"id":6861448,"uuid":"eb7a5126-13fb-4264-8b8c-686809e6d362","attributeTranslation":"Ако 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).","language":1,"languageDescription":"Bulgarian"},{"id":6861450,"uuid":"eb7a5126-13fb-4264-8b8c-686809e6d362","attributeTranslation":"En 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).","language":7,"languageDescription":"Spanish"}]},{"id":584539,"number":7,"principalInclusionCriteria":"Achieved 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).","principalInclusionCriteriaTranslations":[{"id":6861452,"uuid":"5e7b81eb-9aa2-4d41-acbe-8770455b0f4e","attributeTranslation":"Alcanzar 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).","language":7,"languageDescription":"Spanish"},{"id":6861453,"uuid":"5e7b81eb-9aa2-4d41-acbe-8770455b0f4e","attributeTranslation":"Az 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.","language":13,"languageDescription":"Hungarian"},{"id":6861454,"uuid":"5e7b81eb-9aa2-4d41-acbe-8770455b0f4e","attributeTranslation":"pacjent 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).","language":19,"languageDescription":"Polish"},{"id":6861455,"uuid":"5e7b81eb-9aa2-4d41-acbe-8770455b0f4e","attributeTranslation":"При 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).","language":1,"languageDescription":"Bulgarian"}]},{"id":584540,"number":8,"principalInclusionCriteria":"If 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).","principalInclusionCriteriaTranslations":[{"id":6861458,"uuid":"b61c3bdd-62d9-4ac0-b4b8-9833ca450d8b","attributeTranslation":"En 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. \nLa 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).","language":7,"languageDescription":"Spanish"},{"id":6861459,"uuid":"b61c3bdd-62d9-4ac0-b4b8-9833ca450d8b","attributeTranslation":"Ако 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. \nСексуалната 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).","language":1,"languageDescription":"Bulgarian"},{"id":6861457,"uuid":"b61c3bdd-62d9-4ac0-b4b8-9833ca450d8b","attributeTranslation":"Ha 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. \nA 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).","language":13,"languageDescription":"Hungarian"},{"id":6861456,"uuid":"b61c3bdd-62d9-4ac0-b4b8-9833ca450d8b","attributeTranslation":"jeżeli 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. \nPartnerka 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).","language":19,"languageDescription":"Polish"}]},{"id":584541,"number":9,"principalInclusionCriteria":"Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements.","principalInclusionCriteriaTranslations":[{"id":6861463,"uuid":"0065fe7e-e51d-414e-9a49-6e9f8e148ce6","attributeTranslation":"ma partnera lub opiekuna prawnego, który jest w stanie zrozumieć wymogi badania i się do nich stosować.","language":19,"languageDescription":"Polish"},{"id":6861460,"uuid":"0065fe7e-e51d-414e-9a49-6e9f8e148ce6","attributeTranslation":"Tener un(os) progenitor(es) o tutor(es) legal(es) que pueda(n) entender todos los requisitos del estudio y cumplir con ellos.","language":7,"languageDescription":"Spanish"},{"id":6861462,"uuid":"0065fe7e-e51d-414e-9a49-6e9f8e148ce6","attributeTranslation":"A szülő(k) vagy a törvényes gondviselő(k) képesek megérteni az összes vizsgálati követelményt, és együttműködni azok szerint.","language":13,"languageDescription":"Hungarian"},{"id":6861461,"uuid":"0065fe7e-e51d-414e-9a49-6e9f8e148ce6","attributeTranslation":"Има родител(и) или законен(ни) настойник(ци), който(които) може(могат) да разбере(ат) и спазва(т) изискванията на изпитването.","language":1,"languageDescription":"Bulgarian"}]}],"principalExclusionCriteria":[{"id":1005260,"number":1,"principalExclusionCriteria":"Treatment 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.","principalExclusionCriteriaTranslations":[{"id":6861387,"uuid":"e2ec2eb5-3199-4817-8099-7c158ca0396c","attributeTranslation":"leczenie z wykorzystaniem którejkolwiek z wymienionych terapii eksperymentalnych w konkretnym okresie czasu:\n● kiedykolwiek:\n- leki zwiększające ekspresję utrofiny (z wyjątkiem ezutromidu);\n- terapia CRISPR/Cas9 lub inne terapie wykorzystujące techniki edycji genów;\n- terapia genowa;\n- terapia komórkowa (np. przeszczep komórek macierzystych);\n- jakakolwiek terapia antysensownymi oligonukleotydami, poza PRO045 (BMN 045) lub PRO053 (BMN 053) (patrz poniżej);\n- terapie pomijania egzonu;\n - drisapersen w okresie 36 tygodni przed 1. tygodniem;\n - PRO045 (BMN 045) w okresie 24 tygodni przed 1. tygodniem;\n- PRO053 (BMN 053) w okresie 24 tygodni przed 1. tygodniem;\n - PRO051 (BMN 051) w okresie 24 tygodni przed 1. tygodniem;\n● wszystkie rodzaje leczenia hamujące miostatynę w okresie 24 tygodni przed 1. tygodniem, w tym między innymi:\n- domagrozumab (PF-06252616);\n- RG-6206 (wcześniej RO-7239361 i BMS-986089);\n● terapie z zastosowaniem leków drobnocząsteczkowych:\n- ezutromid (SMT C1100) w okresie 1 tygodnia przed 1. tygodniem;\n● w okresie 24 tygodni przed 1. tygodniem:\n- 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);\n- inhibitory aktywacji komórek tucznych (np. CRD007 [sól sodowa pemirolastu]);\n- idebenon (Raxone®);\n● w okresie 12 tygodni przez 1. tygodniem:\n- 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;\n- wamorolon (VBP-15);\n● w przypadku wszelkich terapii eksperymentalnych niewymienionych w kryterium wyłączenia 1 należy skonsultować się z monitorem badań klinicznych.","language":19,"languageDescription":"Polish"},{"id":6861384,"uuid":"e2ec2eb5-3199-4817-8099-7c158ca0396c","attributeTranslation":"Haber recibido tratamiento con uno de los fármacos en estudio que aparecen a continuación y dentro de los periodos especificados:\n● En cualquier momento:\n- Fármacos de aumento de la utrofina (excepto Ezutromid).\n- CRISPR/Cas9, y cualquier otra forma de modificación del genoma.\n- Genoterapia\n- Citoterapia (p. ej., trasplante de hemocitoblastos)\n- Cualquier forma de tratamiento antisentido con ácido nucleico, excepto PRO045 (BMN 045) o PRO053 (BMN 053) (véase a continuación).\n- Tratamientos de omisión de exones.\n - Drisapersen dentro de las 36 semanas anteriores a la semana 1.\n - PRO045 (BMN 045) dentro de las 24 semanas anteriores a la semana 1.\n- PRO053 (BMN 053) dentro de las 24 semanas anteriores a la semana 1.\n - PRO051 (BMN 051) dentro de las 24 semanas anteriores a la semana 1.\n● Cualquier tratamiento frente a la miostatina dentro de las 24 semanas anteriores a la semana 1. Entre ellos, se encuentran:\n- Domagrozumab (PF-06252616).\n- RG-6206 (formalmente RO-7239361 y BMS-986089).\n● Tratamientos tradicionales:\n- Ezutromid (SMT C1100) en la semanas anterior a la semana 1.\n● Dentro de las 24 semanas anteriores a la semana 1:\n- Fármacos antifibróticos o antiinflamatorios, entre los que se encuentran: rimeporida, galato de epigalocatequina, TAS-205, edasalonexent (CAT1004), FG-3019 y halofuginona \n(HT-100).\n- Inhibidor de la activación mastocitaria (p. ej., CRD007 [sodio de pemirolast]).\n- Idebenona (Raxone®).\n● Dentro de las 12 semanas anteriores a la semana 1:\n- 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.\n- Vamorolona (VBP-15).\n● Para informarse sobre cualquier otro tratamiento en estudio que no se haya especificado en el criterio de exclusión 1, consulte al monitor médico.","language":7,"languageDescription":"Spanish"},{"id":6861385,"uuid":"e2ec2eb5-3199-4817-8099-7c158ca0396c","attributeTranslation":"Kezelés bármely alábbi vizsgálati kezeléssel a megadott időintervallumokon belül:\n● Bármikor:\n- Utrofin-túlszabályozó készítmények (az ezutromid kivételével)\n- CRISPR/Cas9, vagy a géneditálás bármely más formája\n- Génterápia\n- Sejtalapú kezelés (pl. őssejt-transzplantáció)\n- Nukleinsav antisense kezelés bármely formája, a PRO045 (BMN 045) vagy PRO053 (BMN 053) kivételével (lásd alább)\n- Exonátugrásos kezelések\n - Drisapersen az 1. hetet megelőző 36 hétben\n - PRO045 (BMN 045) az 1. hetet megelőző 24 hétben\n- PRO053 (BMN 053) az 1. hetet megelőző 24 hétben\n - PRO051 (BMN 051) az 1. hetet megelőző 24 hétben\n• Az összes antimyostatin kezelés az 1. hetet megelőző 24 hétben, ideértve többek között az alábbiakat:\n- Domagrozumab (PF-06252616)\n- RG-6206 (hivatalosan RO-7239361 és BMS-986089)\n• Kismolekulájú szerek:\n- Ezutromid (SMT C1100) az 1. hetet megelőző 1 hétben\n• Az 1. hetet megelőző 24 hétben:\n- Antifibrotikus vagy antiinflammatórikus készítmények, beleértve többek között az alábbiakat: rimeporid, epigallokatechin-gallát, TAS-205, edaszalonexent (CAT-1004), FG-3019 és halofuginon (HT-100)\n- Hízósejt-aktiváció inhibitor (pl. CRD007 [pemirolaszt-nátrium])\n- Idebenon (Raxone®)\n• Az 1. hetet megelőző 12 hétben:\n- 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.\n- Vamorolon (VBP-15)\n• Minden vizsgálati kezelés esetén, amely az 1. kizárási kritériumban nem került felsorolásra, konzultáljon az orvosmonitorral.","language":13,"languageDescription":"Hungarian"},{"id":6861386,"uuid":"e2ec2eb5-3199-4817-8099-7c158ca0396c","attributeTranslation":"Лечение с която и да е от следните експериментални терапии в съответствие с посочените времеви диапазони:\n● В който и да е момент:\n- Препарати за регулиране на утрофин (с изключение на езутромид)\n- CRISPR/Cas9 или друга форма на редактиране на генома\n- Генна терапия\n- Клетъчна терапия (напр. трансплантация на стволови клетки)\n- Всяка форма на антисенс терапия с нуклеинова киселина, с изключение на PRO045 (BMN 045) или PRO053 (BMN 053) (вижте по-долу)\n- Терапии с прескачане на екзон\n - Дрисаперсен в рамките на 36 седмици преди седмица 1\n - PRO045 (BMN 045) в рамките на 24 седмици преди седмица 1\n- PRO053 (BMN 053) в рамките на 24 седмици преди седмица 1\n - PRO051 (BMN 051) в рамките на 24 седмици преди седмица 1\n- Всякакви терапии с анти-миостатин в рамките на 24 седмици преди седмица 1, включително, но не само:\n- Домагрозумаб (PF-06252616)\n- RG-6206 (наричано преди RO-7239361 и BMS-986089)\n● Терапии с малки молекули:\n- Езутромид (SMT C1100) в рамките на 1 седмица преди седмица 1\n● В рамките на 24 седмици преди седмица 1:\n- Антифибротични или противовъзпалителни препарати, включително, но не само: римепорид, епигалокатехин-галат, TAS-205, едазалонексент (CAT1004), FG-3019 и халофугинон (HT-100)\n- Инхибитор на активиране на мастоцитите (напр. CRD007 [пемироласт натрий])\n- Идебенон (Raxone®)\n● В рамките на 12 седмици преди седмица 1:\n- 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.\n- Ваморолон (VBP-15)\n● За всяко експериментално лечение, което не е посочено в Критерий за изключване 1, се консултирайте с медицинския монитор.","language":1,"languageDescription":"Bulgarian"}]},{"id":1005261,"number":2,"principalExclusionCriteria":"Treatment 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 antibiotics","principalExclusionCriteriaTranslations":[{"id":6861390,"uuid":"128fa312-9f72-46c9-b4fa-299ee1e06cb3","attributeTranslation":"Лечение с която и да е от следните неекспериментални терапии в съответствие с посочените времеви диапазони:\n● В рамките на 12 седмици преди седмица 1:\n- 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.\n● В рамките на 12 седмици преди седмица 1 или ако се очаква, че ще е необходимо по време на изпитването:\n- Статини\n- Аминогликозидни антибиотици","language":1,"languageDescription":"Bulgarian"},{"id":6861391,"uuid":"128fa312-9f72-46c9-b4fa-299ee1e06cb3","attributeTranslation":"Kezelés bármely alábbi nem vizsgálati kezeléssel a megadott időintervallumokon belül:\n• Az 1. hetet megelőző 12 hétben:\n- 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.\n• Ha a beteg az 1. hetet megelőző 12 hétben szedte vagy a vizsgálat alatt várhatóan szednie kell a következőket:\n- Sztatinok\n- Aminoglikozid antibiotikumok","language":13,"languageDescription":"Hungarian"},{"id":6861388,"uuid":"128fa312-9f72-46c9-b4fa-299ee1e06cb3","attributeTranslation":"leczenie z wykorzystaniem którejkolwiek z wymienionych terapii nieeksperymentalnych w konkretnym okresie czasu:\n● w okresie 12 tygodni przez 1. tygodniem:\n- 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\n● w okresie 12 tygodni przed 1. tygodniem lub jeżeli przewidywane jest stosowanie podczas badania:\n- statyn;\n- antybiotyków aminoglikozydowych.","language":19,"languageDescription":"Polish"},{"id":6861389,"uuid":"128fa312-9f72-46c9-b4fa-299ee1e06cb3","attributeTranslation":"Haber recibido tratamiento con uno de los fármacos no investigados que aparecen a continuación y dentro de los periodos especificados:\n● Dentro de las 12 semanas anteriores a la semana 1:\n- 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.\n● Dentro de las 12 semanas anteriores a la semana 1 o si se prevé la necesidad de uso durante el estudio:\n- Estatinas.\n- Aminoglucósidos.","language":7,"languageDescription":"Spanish"}]},{"id":1005262,"number":3,"principalExclusionCriteria":"Major surgery within 3 months prior to Week 1 or planned surgery for any time during this study, except for protocol-specified surgery, as applicable.","principalExclusionCriteriaTranslations":[{"id":6861392,"uuid":"5e208005-1cc0-4c09-862f-4739d4df011a","attributeTranslation":"Nagyműtét az 1. hetet megelőző 3 hónapban vagy tervezett műtét a vizsgálat alatt bármikor, a vizsgálati tervben előírt műtét kivételével, ha van.","language":13,"languageDescription":"Hungarian"},{"id":6861393,"uuid":"5e208005-1cc0-4c09-862f-4739d4df011a","attributeTranslation":"duże 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.","language":19,"languageDescription":"Polish"},{"id":6861395,"uuid":"5e208005-1cc0-4c09-862f-4739d4df011a","attributeTranslation":"Cirugía 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.","language":7,"languageDescription":"Spanish"},{"id":6861394,"uuid":"5e208005-1cc0-4c09-862f-4739d4df011a","attributeTranslation":"Значима 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 приложимо","language":1,"languageDescription":"Bulgarian"}]},{"id":1005263,"number":4,"principalExclusionCriteria":"Presence of any other significant genetic disease other than DMD (e.g., dwarfism).","principalExclusionCriteriaTranslations":[{"id":6861399,"uuid":"235e2d49-9a3e-4022-a3df-d71863a5c6ef","attributeTranslation":"A DMD-n kívül egyéb jelentős genetikai betegség fennállása (pl. törpeség).","language":13,"languageDescription":"Hungarian"},{"id":6861397,"uuid":"235e2d49-9a3e-4022-a3df-d71863a5c6ef","attributeTranslation":"Presencia de cualquier otra enfermedad genética significativa diferente de la DMD (p. ej., enanismo).","language":7,"languageDescription":"Spanish"},{"id":6861396,"uuid":"235e2d49-9a3e-4022-a3df-d71863a5c6ef","attributeTranslation":"obecność jakichkolwiek innych istotnych chorób genetycznych, innych niż dystrofia mięśniowa Duchenne’a (np. niedobór wzrostu).","language":19,"languageDescription":"Polish"},{"id":6861398,"uuid":"235e2d49-9a3e-4022-a3df-d71863a5c6ef","attributeTranslation":"Наличие на друго значимо генетично заболяване, различно от МДД (напр. нанизъм).","language":1,"languageDescription":"Bulgarian"}]},{"id":1005264,"number":5,"principalExclusionCriteria":"Presence of other clinically significant illness including significant cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or malignancy.","principalExclusionCriteriaTranslations":[{"id":6861403,"uuid":"a4882533-da28-42ed-9a8b-773ddcd828c3","attributeTranslation":"Наличие на друго клинично значимо заболяване, включително значимо сърдечно, белодробно, чернодробно, бъбречно, хематологично, имунологично заболяване или поведенческо разстройство, или злокачествено заболяване.","language":1,"languageDescription":"Bulgarian"},{"id":6861400,"uuid":"a4882533-da28-42ed-9a8b-773ddcd828c3","attributeTranslation":"Presencia 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.","language":7,"languageDescription":"Spanish"},{"id":6861401,"uuid":"a4882533-da28-42ed-9a8b-773ddcd828c3","attributeTranslation":"Egyéb klinikailag jelentős betegség fennállása, beleértve a jelentős kardiális, pulmonális, hepatikus, renális, hematológiai, immunológiai vagy viselkedési betegséget és a malignitást.","language":13,"languageDescription":"Hungarian"},{"id":6861402,"uuid":"a4882533-da28-42ed-9a8b-773ddcd828c3","attributeTranslation":"obecność innych istotnych klinicznie chorób, w tym choroby układu krążenia, płuc, nerek, krwi, choroby immunologiczne, zmiany zachowania lub nowotwory.","language":19,"languageDescription":"Polish"}]},{"id":1005265,"number":6,"principalExclusionCriteria":"LVEF <50% on the Screening echocardiogram (ECHO) or QTcF ≥450 msec based on the Screening and Baseline electrocardiogram (ECG).","principalExclusionCriteriaTranslations":[{"id":6861407,"uuid":"1703d685-e1cd-4fa7-9d1a-00e9cc45920d","attributeTranslation":"LVEF <50% na podstawie przesiewowego badania echa serca lub odstęp QTcF ≥450 ms w badaniu elektrokardiogramie (EKG) przesiewowym i początkowym.","language":19,"languageDescription":"Polish"},{"id":6861404,"uuid":"1703d685-e1cd-4fa7-9d1a-00e9cc45920d","attributeTranslation":"LVEF <50% a szűrő echokardiográfián (ECHO) vagy QTcF ≥450 ms a szűrő és kiindulási elektrokardiogramon (EKG).","language":13,"languageDescription":"Hungarian"},{"id":6861406,"uuid":"1703d685-e1cd-4fa7-9d1a-00e9cc45920d","attributeTranslation":"FEVI <50 % en el ecocardiograma de la selección o QTcF ≥450 ms según los electrocardiogramas (ECG) de la selección y basal.","language":7,"languageDescription":"Spanish"},{"id":6861405,"uuid":"1703d685-e1cd-4fa7-9d1a-00e9cc45920d","attributeTranslation":"ФИЛК <50% от ехокардиограмата (ECHO) от скрининга или QTcF ≥450 msec въз основа на електрокардиограмата (ЕКГ) от скрининга и изходното ниво.","language":1,"languageDescription":"Bulgarian"}]},{"id":1005266,"number":7,"principalExclusionCriteria":"Dorsiflexion 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.","principalExclusionCriteriaTranslations":[{"id":6861408,"uuid":"c476c258-ca25-4c92-9a8d-e17408487236","attributeTranslation":"Диапазонът 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.","language":1,"languageDescription":"Bulgarian"},{"id":6861411,"uuid":"c476c258-ca25-4c92-9a8d-e17408487236","attributeTranslation":"La 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.","language":7,"languageDescription":"Spanish"},{"id":6861409,"uuid":"c476c258-ca25-4c92-9a8d-e17408487236","attributeTranslation":"zgięcie 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.","language":19,"languageDescription":"Polish"},{"id":6861410,"uuid":"c476c258-ca25-4c92-9a8d-e17408487236","attributeTranslation":"A 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.","language":13,"languageDescription":"Hungarian"}]},{"id":1005267,"number":8,"principalExclusionCriteria":"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. Additionally, patients who seem unable / unwilling to comply with the study procedures, in the Investigator's opinion, are to be excluded.","principalExclusionCriteriaTranslations":[{"id":6861415,"uuid":"db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5","attributeTranslation":"Предходно 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.","language":1,"languageDescription":"Bulgarian"},{"id":6861412,"uuid":"db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5","attributeTranslation":"Enfermedad 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.","language":7,"languageDescription":"Spanish"},{"id":6861413,"uuid":"db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5","attributeTranslation":"Korábbi 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.","language":13,"languageDescription":"Hungarian"},{"id":6861414,"uuid":"db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5","attributeTranslation":"wcześniejsze 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.","language":19,"languageDescription":"Polish"}]},{"id":1005268,"number":9,"principalExclusionCriteria":"Known hypersensitivity to the study drug or to any of its components","principalExclusionCriteriaTranslations":[{"id":6861416,"uuid":"3d5dfbd1-8924-43af-a1e9-93594f108b0e","attributeTranslation":"Hipersensibilidad conocida al fármaco del estudio o a alguno de sus componentes.","language":7,"languageDescription":"Spanish"},{"id":6861418,"uuid":"3d5dfbd1-8924-43af-a1e9-93594f108b0e","attributeTranslation":"A vizsgálati készítmény hatóanyagával vagy bármely összetevőjével szemben fennálló túlérzékenység","language":13,"languageDescription":"Hungarian"},{"id":6861419,"uuid":"3d5dfbd1-8924-43af-a1e9-93594f108b0e","attributeTranslation":"Известна свръхчувствителност към лекарството по изпитването или към някой от компонентите му","language":1,"languageDescription":"Bulgarian"},{"id":6861417,"uuid":"3d5dfbd1-8924-43af-a1e9-93594f108b0e","attributeTranslation":"nadwrażliwość na badany lek lub którykolwiek z jego składników.","language":19,"languageDescription":"Polish"}]}]},"endPoint":{"primaryEndPoints":[{"id":682288,"number":1,"endPoint":"Double-blind period: Change from Baseline at Week 96 in 4-step ascend velocity (step/second)","isPrimary":true,"endPointTranslations":[{"id":6861371,"uuid":"56d221c8-2997-41d9-9b50-7a2aa5066d45","attributeTranslation":"Двойно сляп период: Промяна от изходното ниво през седмица 96 в скоростта на изкачване на 4 стъпала (стъпка/секунда)","language":1,"languageDescription":"Bulgarian"},{"id":6861368,"uuid":"56d221c8-2997-41d9-9b50-7a2aa5066d45","attributeTranslation":"okres leczenia w fazie podwójnie zaślepionej: stanowi zmiana w 96. tygodniu w stosunku do punktu początkowego w czasie na pokonanie 4 schodów (stopień/sekundy).","language":19,"languageDescription":"Polish"},{"id":6861369,"uuid":"56d221c8-2997-41d9-9b50-7a2aa5066d45","attributeTranslation":"Periodo doble ciego: Cambio desde el momento basal hasta la semana 96 en la velocidad para subir 4 escalones (escalón/segundo)","language":7,"languageDescription":"Spanish"},{"id":6861370,"uuid":"56d221c8-2997-41d9-9b50-7a2aa5066d45","attributeTranslation":"Kettős vak szakasz: változás a kiindulástól a 96. hétig a 4 lépéses emelkedési sebességben (lépés/másodperc)","language":13,"languageDescription":"Hungarian"}]}],"secondaryEndPoints":[{"id":682289,"number":1,"endPoint":"• 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)","isPrimary":false,"endPointTranslations":[{"id":6861372,"uuid":"6ecbb7b6-5478-44ce-af09-d5f1a0d0e252","attributeTranslation":"zmiana 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);","language":19,"languageDescription":"Polish"},{"id":6861373,"uuid":"6ecbb7b6-5478-44ce-af09-d5f1a0d0e252","attributeTranslation":"• Variación desde el momento basal hasta la semana 96 en la prueba de marcha de seis minutos\n• Variación desde el momento basal hasta la semana 96 en la velocidad para levantarse del suelo (subida/segundo)\n• Variación desde el momento basal hasta la semana 144 en la velocidad para subir 4 escalones (escalón/segundo)","language":7,"languageDescription":"Spanish"},{"id":6861375,"uuid":"6ecbb7b6-5478-44ce-af09-d5f1a0d0e252","attributeTranslation":"• Промяна от изходното ниво на Седмица 96 в 6-мунитния тест за ходене\n• Промяна от изходното ниво на Седмица 96 в скоростта на изправяне от пода (ставане/секунда)\n• Промяна от изходното ниво на Седмица 144 в скоростта на изкачване на 4 стъпала (стъпка/секунда)","language":1,"languageDescription":"Bulgarian"},{"id":6861374,"uuid":"6ecbb7b6-5478-44ce-af09-d5f1a0d0e252","attributeTranslation":"- Változás a kiindulási értékhez képest a 96. héten a 6MWT-ben\n- Változás a 96. héten a padlóról való felemelkedés sebességében a kiindulási értékhez képest\n- Változás a kiindulási értékhez képest a 144. héten a 4 lépéses emelkedési sebességben","language":13,"languageDescription":"Hungarian"}]},{"id":682290,"number":2,"endPoint":"• 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.","isPrimary":false,"endPointTranslations":[{"id":6861377,"uuid":"6971a9ea-0519-47c5-a527-034ab98b399d","attributeTranslation":"• Variación con respecto al momento basal en la semana 96 en la velocidad de la prueba de caminar/correr 10 metros (metros/segundo)\n• 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.","language":7,"languageDescription":"Spanish"},{"id":6861379,"uuid":"6971a9ea-0519-47c5-a527-034ab98b399d","attributeTranslation":"zmiana 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 blot","language":19,"languageDescription":"Polish"},{"id":6861378,"uuid":"6971a9ea-0519-47c5-a527-034ab98b399d","attributeTranslation":"• Промяна от изходното ниво на Седмица 96 в скоростта при теста за ходене/бягане на 10 метра (метър/секунда)\n• Промяна ор изходното ниво на Седмици 48 или 96  в количеството на експресия на белтъка дисторфин,\nизмерено чрез Western blot в биопсична мускулна тъкан.","language":1,"languageDescription":"Bulgarian"},{"id":6861376,"uuid":"6971a9ea-0519-47c5-a527-034ab98b399d","attributeTranslation":"- Változás a kiindulási értékhez képest a 96. héten a 10MWR sebességben\n- Változás a kiindulási értékhez képest a 48. vagy 96. héten a dystrophin fehérje expressziójának mennyiségében, a biopsziával vizsgált izomszövetből Western blottal mérve.","language":13,"languageDescription":"Hungarian"}]},{"id":682291,"number":3,"endPoint":"• 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 score","isPrimary":false,"endPointTranslations":[{"id":6861383,"uuid":"7af296b7-07f9-439f-b2ea-ba41452548cb","attributeTranslation":"• Промяна от изходото ниво на Седмици 48 и 96 ст в интензитета на експресия на белтъка дисторфин в биопсична мускулна тъкан,\nизмерено чрез IHC.\n• Промяна от изходното ниво през седмица 96 в общата оценка по NSAA","language":1,"languageDescription":"Bulgarian"},{"id":6861382,"uuid":"7af296b7-07f9-439f-b2ea-ba41452548cb","attributeTranslation":"- Változás a kiindulási értékhez képest a 96. héten a 10MWR sebességben\n- Változás a kiindulási értékhez képest a 48. vagy 96. héten a dystrophin fehérje expressziójának mennyiségében, a biopsziával vizsgált izomszövetből Western blottal mérve.","language":13,"languageDescription":"Hungarian"},{"id":6861380,"uuid":"7af296b7-07f9-439f-b2ea-ba41452548cb","attributeTranslation":"• 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\n• Variación con respecto al momento basal en la semana 96 en la puntuación total de la NSAA","language":7,"languageDescription":"Spanish"},{"id":6861381,"uuid":"7af296b7-07f9-439f-b2ea-ba41452548cb","attributeTranslation":"zmiana 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 NSAA","language":19,"languageDescription":"Polish"}]}]},"trialDuration":{"estimatedGlobalEndDate":"2025-10-31","estimatedEndDate":"2025-09-17","estimatedRecruitmentStartDate":"2017-05-31"},"sourceOfMonetarySupport":[{"id":64277,"organisationName":"Sarepta Therapeutics, Inc."}],"populationOfTrialSubjects":{"ageRanges":[{"id":243368,"ageRangeCategoryCode":"2","ageRangeCategory":"2"}],"ageRangeSecondaryIds":[{"id":243369,"ageRangeCategoryCode":"2","ctAgeRangeCode":"6","ctAgeRange":"6","ageRangeCategory":"2"},{"id":243370,"ageRangeCategoryCode":"2","ctAgeRangeCode":"5","ctAgeRange":"5","ageRangeCategory":"2"}],"clinicalTrialGroups":[{"code":"2","name":"Patients"}],"isFemaleSubjects":false,"isMaleSubjects":true,"isVulnerablePopulationSelected":true},"individualParticipantData":{"planToShareIPD":"2"}},"protocolInformation":{"studyDesign":{"periodDetails":[]}},"scientificAdviceAndPip":{"scientificAdvices":[],"paediatricInvestigationPlan":[]},"associatedClinicalTrials":[{"id":22541,"ctNumber":"2015-002069-52","fullTitle":"A 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 Duchenne","sponsorAgreementOption":"OPTIONAL","sponsorAgreementOptionName":"OPTIONAL","hasDocument":false,"associatedCtDocs":[]}],"references":[],"pubmedCode":[],"pubmedUrl":[]},"assessmentOutcome":"acceptable","assessmentOutcomeDate":"2025-05-02","conclusionDate":"2025-05-02","therapeuticAreas":[{"code":"5","name":"Diseases [C] - Musculoskeletal Diseases [C05]"}],"medicalConditions":[{"id":91632,"medicalCondition":"Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping.","isConditionRareDisease":true}],"sponsors":[{"id":97917,"primary":true,"publicContacts":[{"id":292356,"type":"Public","functionalName":"Patient Recruitment","functionalEmailAddress":"SareptAlly@sarepta.com","telephone":"+18887273782","organisation":{"id":488781,"type":"Pharmaceutical company","typeCode":"10","name":"Sarepta Therapeutics 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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).","product":"Fordadistrogene Movaparvovec, ECULIZUMAB, Placebo for PF-06939926","ageRangeSecondary":["5","4"],"ageGroup":"0-17 years","gender":"Male","trialRegion":3,"totalNumberEnrolled":"48","primaryEndPoint":"Change from Baseline at Week 52 in the NSAA total score.","resultsFirstReceived":"No","lastUpdated":"20/02/2026","lastPublicationUpdate":"21/02/2026"},{"ctNumber":"2023-508510-42-00","ctStatus":"Authorised","startDateEU":"2025-03-24","decisionDate":"2024-07-10T08:21:39.002","publishDate":"2026-02-21T02:35:27.159106201","ctPublicStatusCode":5,"authorizedApplication":{"authorizedPartI":{"id":119140,"rowSubjectCount":74,"rowCountriesInfo":[{"eutctId":100000000329,"name":"Australia","isoNumber":36,"isoAlpha2Code":"AU","isoAlpha3Code":"AUS","current":true},{"eutctId":100000000506,"name":"Russian Federation","isoNumber":643,"isoAlpha2Code":"RU","isoAlpha3Code":"RUS","current":true},{"eutctId":100000000439,"name":"Korea, Republic of","isoNumber":410,"isoAlpha2Code":"KR","isoAlpha3Code":"KOR","current":true},{"eutctId":100000000556,"name":"United 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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).","language":34,"languageDescription":"French (France)"}]},{"id":801649,"number":3,"principalInclusionCriteria":"Receipt 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. 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Une dose stable désigne une dose pour laquelle toute modification est ≤ 0,2 mg/kg.","language":34,"languageDescription":"French (France)"}]},{"id":801650,"number":4,"principalInclusionCriteria":"A NSAA total score >16 and <30 at Screening (Visit 1).","principalInclusionCriteriaTranslations":[{"id":9800012,"uuid":"ba14d36a-2ed5-45ad-8af8-adde49719d8e","attributeTranslation":"Una puntuación total de NSAA es de >16 y <30 en la Selección (Visita 1).","language":7,"languageDescription":"Spanish"},{"id":9800013,"uuid":"ba14d36a-2ed5-45ad-8af8-adde49719d8e","attributeTranslation":"Score total NSAA > 16 et < 30 à la sélection (visite 1).","language":34,"languageDescription":"French (France)"}]},{"id":801651,"number":5,"principalInclusionCriteria":"Ambulatory, defined as being able to walk 10 meters unassisted, at Screening (Visit 1).","principalInclusionCriteriaTranslations":[{"id":9800015,"uuid":"d7b3f99c-a50e-47da-a144-0271627e50ab","attributeTranslation":"Ambulatorio, definido como capaz de caminar 10 metros sin asistencia, en la Selección (Visita 1).","language":7,"languageDescription":"Spanish"},{"id":9800014,"uuid":"d7b3f99c-a50e-47da-a144-0271627e50ab","attributeTranslation":"Être ambulatoire, défini comme le fait d’être capable de marcher sur 10 mètres sans assistance, lors de la sélection (visite 1).","language":34,"languageDescription":"French (France)"}]},{"id":801652,"number":6,"principalInclusionCriteria":"Participants/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.","principalInclusionCriteriaTranslations":[{"id":9800016,"uuid":"4c8f17f0-0a7e-4349-8b76-0f33a38d46e5","attributeTranslation":"Patients/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.","language":34,"languageDescription":"French (France)"},{"id":9800017,"uuid":"4c8f17f0-0a7e-4349-8b76-0f33a38d46e5","attributeTranslation":"Participantes 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.","language":7,"languageDescription":"Spanish"}]},{"id":801653,"number":7,"principalInclusionCriteria":"Participants/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.","principalInclusionCriteriaTranslations":[{"id":9800019,"uuid":"7e5d4fd7-b828-424f-a259-96656cf54757","attributeTranslation":"Participantes 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.","language":7,"languageDescription":"Spanish"},{"id":9800018,"uuid":"7e5d4fd7-b828-424f-a259-96656cf54757","attributeTranslation":"Patients/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.","language":34,"languageDescription":"French (France)"}]},{"id":801654,"number":8,"principalInclusionCriteria":"Participants/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).","principalInclusionCriteriaTranslations":[{"id":9800020,"uuid":"4c118d17-8dc1-4762-a722-e62ef741e9e3","attributeTranslation":"Patients/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).","language":34,"languageDescription":"French (France)"},{"id":9800021,"uuid":"4c118d17-8dc1-4762-a722-e62ef741e9e3","attributeTranslation":"Participantes 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).","language":7,"languageDescription":"Spanish"}]}],"principalExclusionCriteria":[{"id":1373195,"number":1,"principalExclusionCriteria":"Prior 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.","principalExclusionCriteriaTranslations":[{"id":9799970,"uuid":"119c9448-9289-466e-96f4-c4a197e25fcf","attributeTranslation":"Tratamiento 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.","language":7,"languageDescription":"Spanish"},{"id":9799971,"uuid":"119c9448-9289-466e-96f4-c4a197e25fcf","attributeTranslation":"Traitement 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.","language":34,"languageDescription":"French (France)"}]},{"id":1373196,"number":10,"principalExclusionCriteria":"Acute 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.","principalExclusionCriteriaTranslations":[{"id":9799972,"uuid":"354ff010-cdde-40b7-bd77-197a982a99f9","attributeTranslation":"Infection 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.","language":34,"languageDescription":"French (France)"},{"id":9799973,"uuid":"354ff010-cdde-40b7-bd77-197a982a99f9","attributeTranslation":"Infección 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.","language":7,"languageDescription":"Spanish"}]},{"id":1373197,"number":11,"principalExclusionCriteria":"Investigator 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.","principalExclusionCriteriaTranslations":[{"id":9799975,"uuid":"ff8ffa5f-db40-4314-9432-9e9f29d12167","attributeTranslation":"Membres 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.","language":34,"languageDescription":"French (France)"},{"id":9799974,"uuid":"ff8ffa5f-db40-4314-9432-9e9f29d12167","attributeTranslation":"Miembros 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 estudio","language":7,"languageDescription":"Spanish"}]},{"id":1373198,"number":12,"principalExclusionCriteria":"Known 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.","principalExclusionCriteriaTranslations":[{"id":9799977,"uuid":"5fe74b7b-6315-4970-9725-25a29440af74","attributeTranslation":"Hipersensibilidad 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.","language":7,"languageDescription":"Spanish"},{"id":9799976,"uuid":"5fe74b7b-6315-4970-9725-25a29440af74","attributeTranslation":"Hypersensibilité 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.","language":34,"languageDescription":"French (France)"}]},{"id":1373199,"number":13,"principalExclusionCriteria":"Contraindication to the use of eculizumab, as per the local prescribing information.","principalExclusionCriteriaTranslations":[{"id":9799979,"uuid":"3c2ccb18-5737-4be1-9af0-37e58f6948b7","attributeTranslation":"Contre-indication à l’éculizumab, selon les informations locales relatives à la prescription.","language":34,"languageDescription":"French (France)"},{"id":9799978,"uuid":"3c2ccb18-5737-4be1-9af0-37e58f6948b7","attributeTranslation":"Contraindicación del uso de eculizumab, de acuerdo con la información del prospecto local.","language":7,"languageDescription":"Spanish"}]},{"id":1373200,"number":14,"principalExclusionCriteria":"LVEF <50% on echocardiogram performed at the Screening Visit (Visit 1), as evaluated by the central reader.","principalExclusionCriteriaTranslations":[{"id":9799981,"uuid":"cd041748-d9d7-4413-8b4e-688a84e0a454","attributeTranslation":"FEVG < 50 % à l’électrocardiogramme de la visite de sélection (visite 1) telle qu’évaluée par le lecteur central.","language":34,"languageDescription":"French (France)"},{"id":9799980,"uuid":"cd041748-d9d7-4413-8b4e-688a84e0a454","attributeTranslation":"FEVI <50 % en el ecocardiograma realizado en la visita de selección (Visita 1), según lo determinado por el evaluador central.","language":7,"languageDescription":"Spanish"}]},{"id":1373201,"number":15,"principalExclusionCriteria":"Participants 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.","principalExclusionCriteriaTranslations":[{"id":9799982,"uuid":"c6f57bc3-0b08-4b88-a49a-c019c676be79","attributeTranslation":"Los participantes con las siguientes anomalías genéticas en el gen distrofina confirmado por el investigador con base en la revisión de la prueba genética DMD:\na. 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\nb. Una deleción que afecte tanto al exón 29 como al exón 30; O BIEN\nc. Una deleción que afecte a cualquiera de los exones entre el 56 y el 71, incluidos ambos.","language":7,"languageDescription":"Spanish"},{"id":9799983,"uuid":"c6f57bc3-0b08-4b88-a49a-c019c676be79","attributeTranslation":"Les anomalies génétiques suivantes du gène de la dystrophine, confirmées par l’investigateur sur la base de l’examen des tests génétiques de la DMD :\n• 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\n• Une délétion qui affecte à la fois l’exon 29 et l’exon 30 ; OU\n• Une délétion qui affecte n’importe quel exon entre 56 et 71 inclus","language":34,"languageDescription":"French (France)"}]},{"id":1373202,"number":16,"principalExclusionCriteria":"Cardiac 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.","principalExclusionCriteriaTranslations":[{"id":9799985,"uuid":"5138fa37-cc77-4a6b-8f28-a9ce246e3569","attributeTranslation":"Patologías cardíacas, evaluadas por un cardiólogo pediátrico en la visita de selección (Visita 1):\na. Diagnóstico de miocarditis (p. ej., viral): basado o bien en antecedentes médicos o en hallazgos en imágenes cardíacas;\nb. 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.","language":7,"languageDescription":"Spanish"},{"id":9799984,"uuid":"5138fa37-cc77-4a6b-8f28-a9ce246e3569","attributeTranslation":"Les pathologies cardiaques, telles qu’évaluées par le cardiologue pédiatrique lors de la visite de sélection (visite 1) :\na. Diagnostic de myocardite (p. ex. virale) : soit à partir des antécédents médicaux soit à partir de découvertes issues des tests d’imagerie ;\nb. 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.","language":34,"languageDescription":"French (France)"}]},{"id":1373203,"number":17,"principalExclusionCriteria":"Not 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).","principalExclusionCriteriaTranslations":[{"id":9799987,"uuid":"a30eb0ec-7a72-4c60-a2a0-afe714063303","attributeTranslation":"No 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).","language":7,"languageDescription":"Spanish"},{"id":9799986,"uuid":"a30eb0ec-7a72-4c60-a2a0-afe714063303","attributeTranslation":"Pas 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).","language":34,"languageDescription":"French (France)"}]},{"id":1373204,"number":2,"principalExclusionCriteria":"Exposure 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.","principalExclusionCriteriaTranslations":[{"id":9799988,"uuid":"a2dbfcf7-442c-4cac-ae50-d47c1f45310d","attributeTranslation":"La 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:\n• 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.\n• 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.","language":7,"languageDescription":"Spanish"},{"id":9799989,"uuid":"a2dbfcf7-442c-4cac-ae50-d47c1f45310d","attributeTranslation":"Exposition 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 :\n• 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.\n• 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.","language":34,"languageDescription":"French (France)"}]},{"id":1373205,"number":3,"principalExclusionCriteria":"Previous 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.","principalExclusionCriteriaTranslations":[{"id":9799990,"uuid":"07ba5d96-26e4-4652-ad50-8d4f6dbdfc84","attributeTranslation":"Administration 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.","language":34,"languageDescription":"French (France)"},{"id":9799991,"uuid":"07ba5d96-26e4-4652-ad50-8d4f6dbdfc84","attributeTranslation":"Administración 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.","language":7,"languageDescription":"Spanish"}]},{"id":1373206,"number":4,"principalExclusionCriteria":"Known cognitive impairment or behavioral issues that would impede the ability to follow instructions, in the judgment of the Investigator, at Screening (Visit 1).","principalExclusionCriteriaTranslations":[{"id":9799992,"uuid":"0a6e42cb-ae93-4df6-88f6-f6cc5c588585","attributeTranslation":"Problemas cognitivos o de comportamiento conocidos que dificultarían la capacidad para seguir instrucciones, según el criterio del investigador, en la Selección (Visita 1).","language":7,"languageDescription":"Spanish"},{"id":9799993,"uuid":"0a6e42cb-ae93-4df6-88f6-f6cc5c588585","attributeTranslation":"Déficience 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).","language":34,"languageDescription":"French (France)"}]},{"id":1373207,"number":5,"principalExclusionCriteria":"Any 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).","principalExclusionCriteriaTranslations":[{"id":9799994,"uuid":"2dd53d68-3834-4f0b-acd9-cf7f20eed41a","attributeTranslation":"Cualquier 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).","language":7,"languageDescription":"Spanish"},{"id":9799995,"uuid":"2dd53d68-3834-4f0b-acd9-cf7f20eed41a","attributeTranslation":"Toute 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).","language":34,"languageDescription":"French (France)"}]},{"id":1373208,"number":6,"principalExclusionCriteria":"Positive test for NAb to AAV9, based on the threshold determined by the Central Laboratory, from a sample taken at Screening (Visit 1)","principalExclusionCriteriaTranslations":[{"id":9799997,"uuid":"76dce107-5c83-4bc5-bf28-e9593c4e4311","attributeTranslation":"Résultat 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).","language":34,"languageDescription":"French (France)"},{"id":9799996,"uuid":"76dce107-5c83-4bc5-bf28-e9593c4e4311","attributeTranslation":"Prueba positiva de AcN para VAA9, según el umbral determinado por el laboratorio central, de una muestra tomada en la Selección (Visita 1).","language":7,"languageDescription":"Spanish"}]},{"id":1373209,"number":7,"principalExclusionCriteria":"Receipt 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.","principalExclusionCriteriaTranslations":[{"id":9799998,"uuid":"d38eb24d-3f01-461b-8282-b2686ffa9bb1","attributeTranslation":"Administración 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.","language":7,"languageDescription":"Spanish"},{"id":9799999,"uuid":"d38eb24d-3f01-461b-8282-b2686ffa9bb1","attributeTranslation":"Administration 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.","language":34,"languageDescription":"French (France)"}]},{"id":1373210,"number":8,"principalExclusionCriteria":"Abnormality 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.","principalExclusionCriteriaTranslations":[{"id":9800001,"uuid":"3ed641d9-a715-4ff9-95ae-4652309e2daa","attributeTranslation":"Anomalie 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 :\na. Numération absolue de neutrophiles < 1 000 cellules/mm3 ;\nb. Plaquettes < 150 x 103/μl ;\nc. Cystatine C > 1,2 x LSN ;\nd. 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) ;\ne. Marqueurs d’inflammation hépatique ou de cirrhose manifeste ou occulte, mise en évidence par un ou plusieurs des paramètres suivants :\n1. Temps de prothrombine (PT) > limite supérieure de la normale (LSN) ; rapport normalisé international (INR) prolongé > LSN ;\n2. GLDH > 2 x LSN ;\n3. Bilirubine totale > 1,5 x LSN (sauf si le patient a des antécédents de maladie de Gilbert) et bilirubine directe > 0,5 mg/dl ;\n4. Gamma-glutamyl transférase (GGT) > 1,5 x LSN.","language":34,"languageDescription":"French (France)"},{"id":9800000,"uuid":"3ed641d9-a715-4ff9-95ae-4652309e2daa","attributeTranslation":"Anomalías en los perfiles de hematología o bioquímica en la Selección (Visita 1). 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28","product":"VESLETEPLIRSEN(SRP-5051)","ageRangeSecondary":[""],"ageGroup":"0-17 years, 18-64 years","gender":"Male","trialRegion":3,"totalNumberEnrolled":"21","endDate":"07/02/2025","primaryEndPoint":"Part A: Incidence of Adverse Events (AEs), Baseline up to 75 weeks, Part B: Change From Baseline in Dystrophin Protein Level at Week 28","resultsFirstReceived":"Yes","lastUpdated":"07/10/2024","lastPublicationUpdate":"03/09/2025"},{"ctNumber":"2023-509935-23-00","ctStatus":"Ended","startDateEU":"2020-08-21","endDateEU":"2024-11-06","decisionDate":"2024-05-06T09:31:43.307","publishDate":"2025-09-03T03:35:32.760064634","ctPublicStatusCode":8,"authorizedApplication":{"authorizedPartI":{"id":53637,"rowSubjectCount":40,"rowCountriesInfo":[{"eutctId":100000000354,"name":"Canada","isoNumber":124,"isoAlpha2Code":"CA","isoAlpha3Code":"CAN","current":true},{"eutctId":100000000557,"name":"United States","isoNumber":840,"isoAlpha2Code":"US","isoAlpha3Code":"USA","current":true},{"eutctId":100000000556,"name":"United Kingdom","isoNumber":826,"isoAlpha2Code":"GB","isoAlpha3Code":"GBR","current":true}],"products":[{"id":215976,"part1MpRoleTypeCode":"1","productDictionaryInfo":{"productPk":"10281392","productPharmForm":"VIAL FOR INTRAVENOUS USE","euMpNumber":"PRD9456940","prodAuthStatus":1,"prodName":"VESLETEPLIRSEN(SRP-5051)","pharmForm":"VIAL FOR INTRAVENOUS USE","sponsorProductCode":"SRP-5051","activeSubstanceName":"VESLETEPLIRSEN","euSubstNumber":"SUB221175","productOtherName":"VESLETEPLIRSEN","nameOrg":"SAREPTA THERAPEUTICS INC","productSubstances":[{"productPk":"10281392","substancePk":"282173","nameOrg":"SAREPTA THERAPEUTICS INC","substanceOrigin":"Nucleic Acid","actSubstOrigin":"Nucleic Acid","actSubstName":"VESLETEPLIRSEN","substanceEvCode":"SUB221175"}],"activeSubstanceOtherDescriptiveName":""},"isPaediatricFormulation":false,"mpRoleInTrial":"1","orphanDrugEdit":false,"doseUom":"mg/kg milligram(s)/kilogram","maxDailyDoseAmount":"0","doseUomTotal":"mg/kg milligram(s)/kilogram","maxTotalDoseAmount":"0","maxTreatmentPeriod":300,"timeUnitCode":"2","otherMedicinalProduct":"Chemical","evCode":"PRD9456940","sponsorProductCodeEdit":"SRP-5051","miaNumber":"DE_BB_01_MIA_2024_0015; IMP11566/00001","devices":[],"characteristics":["11"],"routes":["INTRAVENOUS USE"],"allSubstancesChemicals":false,"productName":"VESLETEPLIRSEN(SRP-5051)","jsonActiveSubstanceNames":"vesleteplirsen","pharmaceuticalFormDisplay":"VIAL FOR INTRAVENOUS USE"}],"trialDetails":{"clinicalTrialIdentifiers":{"fullTitle":"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 Treatment","fullTitleTranslations":[{"id":3697013,"uuid":"2552076e-0e7a-48bc-9488-86a6b3fa2ae1","attributeTranslation":"Estudio 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 51","language":7,"languageDescription":"Spanish"},{"id":3697014,"uuid":"2552076e-0e7a-48bc-9488-86a6b3fa2ae1","attributeTranslation":"Een 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 51","language":37,"languageDescription":"Dutch (Netherlands)"}],"publicTitle":"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)","publicTitleTranslations":[{"id":3697021,"uuid":"5ed65853-e6ac-495b-9f94-d6ad60017a8c","attributeTranslation":"Estudio 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)","language":7,"languageDescription":"Spanish"},{"id":3697022,"uuid":"5ed65853-e6ac-495b-9f94-d6ad60017a8c","attributeTranslation":"Tweedelig 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)","language":37,"languageDescription":"Dutch (Netherlands)"}],"shortTitle":"5051-201","secondaryIdentifyingNumbers":{"nctNumber":{"id":265532,"number":"NCT04004065"},"additionalRegistries":[]}},"trialInformation":{"trialCategory":{"isLowIntervention":false,"trialPhase":"4","trialCategory":"2","justificationForTrialCategory":"According 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 Treatment","trialCategoryId":45717},"medicalCondition":{"partIMedicalConditions":[{"id":52628,"medicalCondition":"Duchenne Muscular Dystrophy","medicalConditionTranslations":[{"id":3696990,"uuid":"2c65b8c9-678b-42bb-82f3-caf803f9fa21","attributeTranslation":"Duchenne spierdystrofie","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":3696989,"uuid":"2c65b8c9-678b-42bb-82f3-caf803f9fa21","attributeTranslation":"Distrofia muscular de Duchenne (DMD)","language":7,"languageDescription":"Spanish"}],"isConditionRareDisease":true}],"meddraConditionTerms":[{"termId":100000012047,"version":"20.1","level":"PT","termName":"Duchenne muscular dystrophy gene carrier","classificationCode":"10052655","organClass":100000004850,"active":false}]},"trialObjective":{"trialScopes":[{"code":"5","trialScopeId":152703},{"code":"7","trialScopeId":152704},{"code":"6","trialScopeId":152701},{"code":"3","trialScopeId":152705},{"code":"9","trialScopeId":152700},{"code":"13","otherDescription":"Safety; Immunogenicity; Multiple-Ascending-Dose Study for Dose Determination, then Dose Expansion","trialScopeId":152702}],"mainObjective":"Part 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)\nPart 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 A","mainObjectiveTranslations":[{"id":3697020,"uuid":"e2034f99-e998-475d-8b7e-2d3592716b51","attributeTranslation":"Deel 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)\nDeel 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 A","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":3697019,"uuid":"e2034f99-e998-475d-8b7e-2d3592716b51","attributeTranslation":"Parte 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).\nParte 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.","language":7,"languageDescription":"Spanish"}],"secondaryObjectives":[{"id":170223,"number":1,"secondaryObjective":"Part 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 A","secondaryObjectiveTranslations":[{"id":3697023,"uuid":"4b1aa5f5-4da8-48ec-b65f-49000893338b","attributeTranslation":"Deel A\nHet 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\nDeel B\n• 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\n• 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\n• Het bepalen van de PK van SRP-5051 in plasma en urine\n• 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 A","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":3697024,"uuid":"4b1aa5f5-4da8-48ec-b65f-49000893338b","attributeTranslation":"Parte A\nDeterminar 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.\nParte B\n•\tEvaluar 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.\n•\tEvaluar 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. \n•\tDeterminar la farmacocinética de SRP-5051 en plasma y orina.\n•\tEvaluar 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.","language":7,"languageDescription":"Spanish"}]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":330669,"number":1,"principalInclusionCriteria":"Inclusion Criteria for participants previously treated with Vesleteplirsen: - Has received prior Vesleteplirsen treatment in Part A of this study or in Study 5051-102.","principalInclusionCriteriaTranslations":[{"id":3697016,"uuid":"6e7686c6-7ec7-4d86-837d-c4d2671e1ca2","attributeTranslation":"Criterios de inclusión para participantes previamente tratados con vesleteplirsén:\n-\tHaber recibido tratamiento previo con vesleteplirsén en la parte A de este estudio o en el estudio 5051-102.","language":7,"languageDescription":"Spanish"},{"id":3697015,"uuid":"6e7686c6-7ec7-4d86-837d-c4d2671e1ca2","attributeTranslation":"Inclusiecriteria voor deelnemers die eerder zijn behandeld met vesleteplirsen:\n- Heeft eerder een behandeling met vesleteplirsen ondergaan in deel A van dit onderzoek of in onderzoek 5051-102.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":330670,"number":2,"principalInclusionCriteria":"Inclusion 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).","principalInclusionCriteriaTranslations":[{"id":3697018,"uuid":"e4d79ff0-8e61-4e84-b57e-7146cdb2d35f","attributeTranslation":"Inclusiecriteria voor behandelingsnaïeve deelnemers die worden geïncludeerd in deel B:\n- 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.\n- 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.\n- Heeft een stabiele longfunctie (geforceerde vitale capaciteit [FVC] ≥ 40% van de voorspelde waarde en geen noodzaak voor nachtelijke beademing).","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":3697017,"uuid":"e4d79ff0-8e61-4e84-b57e-7146cdb2d35f","attributeTranslation":"Criterios de inclusión para participantes sin tratamiento previo que se incluyan en la parte B:\n-\tTener 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.\n-\tHaber 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.\n-\t- Tener una función pulmonar estable (capacidad vital forzada [FVC] ≥40 % del valor previsto y no necesitar respiración artificial nocturna).","language":7,"languageDescription":"Spanish"}]}],"principalExclusionCriteria":[{"id":571075,"number":1,"principalExclusionCriteria":"Exclusion 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.","principalExclusionCriteriaTranslations":[{"id":3697009,"uuid":"329fb309-bb41-42e9-a8ac-44667aa6c600","attributeTranslation":"Criterios de exclusión para participantes tratados previamente con vesleteplirsén y nuevos participantes que se incluyan en la parte B:\n-\tPresencia 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.","language":7,"languageDescription":"Spanish"},{"id":3697010,"uuid":"329fb309-bb41-42e9-a8ac-44667aa6c600","attributeTranslation":"Exclusiecriteria voor deelnemers die eerder zijn behandeld met vesleteplirsen en nieuwe deelnemers die worden geïncludeerd in deel B:\n- 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.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":571076,"number":2,"principalExclusionCriteria":"Exclusion 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.","principalExclusionCriteriaTranslations":[{"id":3697011,"uuid":"a9731a70-3a2e-4920-97ba-d905d2e879ed","attributeTranslation":"Criterios de exclusión para participantes sin tratamiento previo que se incluyan en la parte B:\n-\tAntecedentes de hipomagnesemia en las 12 semanas previas a la selección.\n-\tInicio 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.\n-\tEn 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.\n-\tTener 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.\n-\tTratamiento 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.\n-\t- Se aplican otros criterios de inclusión/exclusión.","language":7,"languageDescription":"Spanish"},{"id":3697012,"uuid":"a9731a70-3a2e-4920-97ba-d905d2e879ed","attributeTranslation":"Exclusiecriteria voor behandelingsnaïeve deelnemers die worden geïncludeerd in deel B:\n- Voorgeschiedenis van hypomagnesiëmie in de 12 weken voorafgaand aan de screening.\n- 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.\n- 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.\n- Heeft een linkerventrikelejectiefractie (LVEF) < 40,0% gebaseerd op een echocardiogram (echo) uitgevoerd in de 12 weken voorafgaand aan de screening of tijdens het screeningsbezoek.\n- 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.\n- Er kunnen ook andere inclusie-/exclusiecriteria gelden.","language":37,"languageDescription":"Dutch (Netherlands)"}]}]},"endPoint":{"primaryEndPoints":[{"id":383573,"number":1,"endPoint":"Part A: Incidence of Adverse Events (AEs), Baseline up to 75 weeks","isPrimary":true,"endPointTranslations":[{"id":3696992,"uuid":"aa81c546-ad1a-4ca6-b508-406ac6d9229d","attributeTranslation":"Parte A: Incidencia de efectos adversos (EA), línea de base superior a 75 semanas.","language":7,"languageDescription":"Spanish"},{"id":3696991,"uuid":"aa81c546-ad1a-4ca6-b508-406ac6d9229d","attributeTranslation":"Deel A: incidentie van bijwerkingen (AE’s), baseline tot 75 weken","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":383574,"number":2,"endPoint":"Part B: Change From Baseline in Dystrophin Protein Level at Week 28","isPrimary":true,"endPointTranslations":[{"id":3696993,"uuid":"708d93a6-e2c2-487c-a254-4e7babd226ab","attributeTranslation":"Deel B: verandering ten opzichte van de uitgangswaarde in het dystrofine-eiwitniveau in week 28","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":3696994,"uuid":"708d93a6-e2c2-487c-a254-4e7babd226ab","attributeTranslation":"Parte B: Cambio desde la línea de base en el nivel de proteína distrofina (según lo medido por Western blot) a las 28 semanas","language":7,"languageDescription":"Spanish"}]}],"secondaryEndPoints":[{"id":383575,"number":1,"endPoint":"Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen, Pre-dose and at multiple time points (up to 32 hours) after end of infusion","isPrimary":false,"endPointTranslations":[{"id":3696996,"uuid":"c26f5b66-0142-4dbe-8e20-af36325a4d5b","attributeTranslation":"Parte B: cambio con respecto al momento basal en los niveles de omisión del exón en la semana 28.","language":7,"languageDescription":"Spanish"},{"id":3696995,"uuid":"c26f5b66-0142-4dbe-8e20-af36325a4d5b","attributeTranslation":"Deel B: verandering ten opzichte van de uitgangswaarde in exon-skipping-niveaus in week 28","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":383576,"number":2,"endPoint":"Part A: PK: Urine Concentration of Vesleteplirsen, Pre-dose and at multiple time periods (up to 48 hours) after end of infusion","isPrimary":false,"endPointTranslations":[{"id":3696998,"uuid":"22a85507-d315-4ca9-80a6-447d3d9c320b","attributeTranslation":"Parte 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 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(DMD)","language":18,"languageDescription":"Dutch"},{"id":6890896,"uuid":"34e353c6-d15f-4ca2-9bd5-d1ee8c650ddb","attributeTranslation":"Μία 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)","language":5,"languageDescription":"Greek"},{"id":6890895,"uuid":"34e353c6-d15f-4ca2-9bd5-d1ee8c650ddb","attributeTranslation":"Estudio 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 caminar","language":7,"languageDescription":"Spanish"}],"publicTitle":"\"A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy \n(DMD)\"","publicTitleTranslations":[{"id":6890915,"uuid":"87f1a11d-abae-4379-9c44-b7a4b818260a","attributeTranslation":"Μία φάσης 3 μελέτη του viltolarsen σε αγόρια με μυϊκή δυστροφία Duchenne (DMD)","language":5,"languageDescription":"Greek"},{"id":6890916,"uuid":"87f1a11d-abae-4379-9c44-b7a4b818260a","attributeTranslation":"\"Een fase 3-verlengingsonderzoek naar viltolarsen bij jongens met \nDuchenne-spierdystrofie (DMD)\"","language":18,"languageDescription":"Dutch"},{"id":6890918,"uuid":"87f1a11d-abae-4379-9c44-b7a4b818260a","attributeTranslation":"Estudio de fase 3 de Viltolarsen en niños con distrofia muscular de Duchenne (DMD)","language":7,"languageDescription":"Spanish"},{"id":6890917,"uuid":"87f1a11d-abae-4379-9c44-b7a4b818260a","attributeTranslation":"Studio di fase 3 di Viltolarsen in ragazzi con distrofia muscolare di Duchenne (DMD)","language":14,"languageDescription":"Italian"}],"shortTitle":"NS-065/NCNP-01-302","secondaryIdentifyingNumbers":{"whoUniversalTrialNumber":{"id":396631,"number":"U1111-1298-6244"},"nctNumber":{"id":396630,"number":"NCT04768062"},"additionalRegistries":[]}},"trialInformation":{"trialCategory":{"isLowIntervention":false,"trialPhase":"5","trialCategory":"2","justificationForTrialCategory":"VIL302 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.","trialCategoryId":80608},"medicalCondition":{"partIMedicalConditions":[{"id":91987,"medicalCondition":"Duchenne Muscular Dystrophy (DMD)","medicalConditionTranslations":[{"id":6890868,"uuid":"c9057370-9f8b-4257-960a-099bc643f2fb","attributeTranslation":"Μυϊκή δυστροφία Duchenne (DMD)","language":5,"languageDescription":"Greek"},{"id":6890869,"uuid":"c9057370-9f8b-4257-960a-099bc643f2fb","attributeTranslation":"Distrofia Muscolare di Duchenne (DMD)","language":14,"languageDescription":"Italian"},{"id":6890867,"uuid":"c9057370-9f8b-4257-960a-099bc643f2fb","attributeTranslation":"Duchenne-spierdystrofie (DMD)","language":18,"languageDescription":"Dutch"},{"id":6890870,"uuid":"c9057370-9f8b-4257-960a-099bc643f2fb","attributeTranslation":"Duchenne Muscular Dystrophy (DMD)","language":7,"languageDescription":"Spanish"}],"isConditionRareDisease":true}],"meddraConditionTerms":[{"termId":100000012544,"version":"20.0","level":"PT","termName":"Duchenne muscular dystrophy","classificationCode":"10013801","organClass":100000004850,"active":false}]},"trialObjective":{"trialScopes":[{"code":"4","trialScopeId":266399},{"code":"13","otherDescription":"Tolerability","trialScopeId":266397},{"code":"3","trialScopeId":266396},{"code":"5","trialScopeId":266398}],"mainObjective":"To evaluate the safety and tolerability of viltolarsen administered \nintravenously (IV) at weekly doses of 80 mg/kg in boys who have \ncompleted the NS-065/NCNP-01-301 study","mainObjectiveTranslations":[{"id":6890911,"uuid":"284fa4a9-4a55-4a87-ad65-5bdc417fcfc2","attributeTranslation":"Evaluar 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-01-301","language":7,"languageDescription":"Spanish"},{"id":6890912,"uuid":"284fa4a9-4a55-4a87-ad65-5bdc417fcfc2","attributeTranslation":"Het 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 voltooid","language":18,"languageDescription":"Dutch"},{"id":6890913,"uuid":"284fa4a9-4a55-4a87-ad65-5bdc417fcfc2","attributeTranslation":"Valutare 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‐01-301","language":14,"languageDescription":"Italian"},{"id":6890914,"uuid":"284fa4a9-4a55-4a87-ad65-5bdc417fcfc2","attributeTranslation":"Αξιολόγηση 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 01-301","language":5,"languageDescription":"Greek"}],"secondaryObjectives":[{"id":304263,"number":1,"secondaryObjective":"Secondary 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.","secondaryObjectiveTranslations":[{"id":6890920,"uuid":"0f92f432-a4d3-4912-aca9-4b4764fa6f10","attributeTranslation":"Σύγκριση 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 αντοχής","language":5,"languageDescription":"Greek"},{"id":6890922,"uuid":"0f92f432-a4d3-4912-aca9-4b4764fa6f10","attributeTranslation":"Het 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 uithoudingsvermogen","language":18,"languageDescription":"Dutch"},{"id":6890919,"uuid":"0f92f432-a4d3-4912-aca9-4b4764fa6f10","attributeTranslation":"Confrontare 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.","language":14,"languageDescription":"Italian"},{"id":6890921,"uuid":"0f92f432-a4d3-4912-aca9-4b4764fa6f10","attributeTranslation":"Comparar 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 resistencia","language":7,"languageDescription":"Spanish"}]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":587087,"number":1,"principalInclusionCriteria":"1. Patient has completed the NS-065/NCNP-01-301 study;","principalInclusionCriteriaTranslations":[{"id":6890900,"uuid":"f2917dcb-9d5f-4f13-872d-f1f9f5446ead","attributeTranslation":"1.      Ο ασθενής έχει ολοκληρώσει τη μελέτη NS-065/NCNP-01-301","language":5,"languageDescription":"Greek"},{"id":6890899,"uuid":"f2917dcb-9d5f-4f13-872d-f1f9f5446ead","attributeTranslation":"1. Il paziente ha completato lo studio NS-065/NCNP-01-301.","language":14,"languageDescription":"Italian"},{"id":6890902,"uuid":"f2917dcb-9d5f-4f13-872d-f1f9f5446ead","attributeTranslation":"1. El paciente ha completado el estudio NS-065/NCNP-01-301","language":7,"languageDescription":"Spanish"},{"id":6890901,"uuid":"f2917dcb-9d5f-4f13-872d-f1f9f5446ead","attributeTranslation":"1.\tPatiënt heeft het NS-065/NCNP-01-301-onderzoek afgerond;","language":18,"languageDescription":"Dutch"}]},{"id":587088,"number":2,"principalInclusionCriteria":"2. 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;","principalInclusionCriteriaTranslations":[{"id":6890905,"uuid":"43efb79b-0936-48cd-98d1-fad061bacbc0","attributeTranslation":"\"2.\tOuder(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;\"","language":18,"languageDescription":"Dutch"},{"id":6890904,"uuid":"43efb79b-0936-48cd-98d1-fad061bacbc0","attributeTranslation":"2.      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 απαιτήσεις","language":5,"languageDescription":"Greek"},{"id":6890906,"uuid":"43efb79b-0936-48cd-98d1-fad061bacbc0","attributeTranslation":"\"2.\tIl/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.\"","language":14,"languageDescription":"Italian"},{"id":6890903,"uuid":"43efb79b-0936-48cd-98d1-fad061bacbc0","attributeTranslation":"2. 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 locales","language":7,"languageDescription":"Spanish"}]},{"id":587089,"number":3,"principalInclusionCriteria":"3. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.","principalInclusionCriteriaTranslations":[{"id":6890910,"uuid":"a904163e-c982-4fc3-b8f1-a2c4fd096b38","attributeTranslation":"3. 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 hacerlo","language":7,"languageDescription":"Spanish"},{"id":6890909,"uuid":"a904163e-c982-4fc3-b8f1-a2c4fd096b38","attributeTranslation":"3.      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.","language":5,"languageDescription":"Greek"},{"id":6890907,"uuid":"a904163e-c982-4fc3-b8f1-a2c4fd096b38","attributeTranslation":"3.\tPatiënt 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.","language":18,"languageDescription":"Dutch"},{"id":6890908,"uuid":"a904163e-c982-4fc3-b8f1-a2c4fd096b38","attributeTranslation":"3.\tIl 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.","language":14,"languageDescription":"Italian"}]}],"principalExclusionCriteria":[{"id":1009817,"number":1,"principalExclusionCriteria":"1. 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;","principalExclusionCriteriaTranslations":[{"id":6890879,"uuid":"7103bbae-cc28-4434-8c42-adefff28407a","attributeTranslation":"1.\tIl 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.","language":14,"languageDescription":"Italian"},{"id":6890881,"uuid":"7103bbae-cc28-4434-8c42-adefff28407a","attributeTranslation":"1.      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.","language":5,"languageDescription":"Greek"},{"id":6890880,"uuid":"7103bbae-cc28-4434-8c42-adefff28407a","attributeTranslation":"1. 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 estudio","language":7,"languageDescription":"Spanish"},{"id":6890882,"uuid":"7103bbae-cc28-4434-8c42-adefff28407a","attributeTranslation":"1.\tPatiënt 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;","language":18,"languageDescription":"Dutch"}]},{"id":1009818,"number":2,"principalExclusionCriteria":"2. 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;","principalExclusionCriteriaTranslations":[{"id":6890884,"uuid":"31391515-3901-4641-8418-5be78f6765c8","attributeTranslation":"2.\tIl 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.","language":14,"languageDescription":"Italian"},{"id":6890883,"uuid":"31391515-3901-4641-8418-5be78f6765c8","attributeTranslation":"2.      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.","language":5,"languageDescription":"Greek"},{"id":6890886,"uuid":"31391515-3901-4641-8418-5be78f6765c8","attributeTranslation":"2.\tPatiënt 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;","language":18,"languageDescription":"Dutch"},{"id":6890885,"uuid":"31391515-3901-4641-8418-5be78f6765c8","attributeTranslation":"\"2. El paciente ha recibido un tratamiento cuyo objetivo era la \ninducción de la distrofina o una proteína relacionada con la \ndistrofina después de completar el estudio NS-065/NCNP-01-\n301\"","language":7,"languageDescription":"Spanish"}]},{"id":1009819,"number":3,"principalExclusionCriteria":"3. Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;","principalExclusionCriteriaTranslations":[{"id":6890889,"uuid":"e781194e-3da3-4abc-bd68-96a3e24ac536","attributeTranslation":"3. El paciente recibió otro u otros medicamentos en investigación durante el estudio NS-065/NCNP-01-301 o después de completarlo","language":7,"languageDescription":"Spanish"},{"id":6890887,"uuid":"e781194e-3da3-4abc-bd68-96a3e24ac536","attributeTranslation":"2.\tPatiënt 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;","language":18,"languageDescription":"Dutch"},{"id":6890890,"uuid":"e781194e-3da3-4abc-bd68-96a3e24ac536","attributeTranslation":"3.\tIl paziente ha assunto qualsiasi altro farmaco sperimentale durante o dopo il completamento dello studio NS-065/NCNP-01-301.","language":14,"languageDescription":"Italian"},{"id":6890888,"uuid":"e781194e-3da3-4abc-bd68-96a3e24ac536","attributeTranslation":"3.      Ο ασθενής έλαβε οποιοδήποτε άλλο υπό έρευνα φάρμακο κατά τη διάρκεια ή μετά την ολοκλήρωση της μελέτης NS-065/NCNP-01-301.","language":5,"languageDescription":"Greek"}]},{"id":1009820,"number":4,"principalExclusionCriteria":"4. Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.","principalExclusionCriteriaTranslations":[{"id":6890891,"uuid":"13c1d407-76d1-405b-b83c-62cdb15a5969","attributeTranslation":"4.      Ο ασθενής κρίνεται από τον ερευνητή ή/και τον χορηγό μη κατάλληλος για συμμετοχή στη μελέτη επέκτασης για οποιονδήποτε λόγο.","language":5,"languageDescription":"Greek"},{"id":6890892,"uuid":"13c1d407-76d1-405b-b83c-62cdb15a5969","attributeTranslation":"\"4. El investigador y/o el promotor no consideran apropiado que \nel paciente participe en el estudio de extensión por algún \nmotivo\"","language":7,"languageDescription":"Spanish"},{"id":6890894,"uuid":"13c1d407-76d1-405b-b83c-62cdb15a5969","attributeTranslation":"3.\tIl paziente ha assunto qualsiasi altro farmaco sperimentale durante o dopo il completamento dello studio NS-065/NCNP-01-301.","language":14,"languageDescription":"Italian"},{"id":6890893,"uuid":"13c1d407-76d1-405b-b83c-62cdb15a5969","attributeTranslation":"3.\tPatiënt nam een ander(e) onderzoeksgeneesmiddel(en) tijdens of na afronding van onderzoek NS 065/NCNP 01 301;","language":18,"languageDescription":"Dutch"}]}]},"endPoint":{"primaryEndPoints":[{"id":685129,"number":1,"endPoint":"- 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)","isPrimary":true,"endPointTranslations":[{"id":6890872,"uuid":"0804d475-85ce-4199-828d-2aea39a8dd8b","attributeTranslation":"• Ζωτικά σημεία \n• Κλινική εξέταση\n• Κλινικές εργαστηριακές εξετάσεις\n• Αιματολογικές και κλινικές βιοχημικές εξετάσεις\n• Ανάλυση ούρων\n• Κυτταρολογική εξέταση ούρων\n• Αντισώματα στη δυστροφίνη και το viltolarsen\n• Ηλεκτροκαρδιογράφημα 12 απαγωγών (ΗΚΓ)\n• Υπερηχογράφημα νεφρών \n• Εμφανιζόμενα κατά τη θεραπεία ανεπιθύμητα συμβάντα (TEAE) και σοβαρά ανεπιθύμητα συμβάντα (ΣΑΣ)","language":5,"languageDescription":"Greek"},{"id":6890873,"uuid":"0804d475-85ce-4199-828d-2aea39a8dd8b","attributeTranslation":"• Vitale functies\n• Lichamelijk onderzoek\n• Klinische laboratoriumtests\no\tHematologie en klinische chemie\no\tUrineanalyse\no\tUrine cytologie\n• Antistoffen tegen dystrofine en viltolarsen\n• 12-afleidingen-elektrocardiogram (ECG)\n• Echo van de nieren\n• Behandelingsgerelateerde bijwerkingen (TEAE´s) en ernstige bijwerkingen (SAE´s)","language":18,"languageDescription":"Dutch"},{"id":6890874,"uuid":"0804d475-85ce-4199-828d-2aea39a8dd8b","attributeTranslation":"• Constantes vitales \n• Exploración física \n• Análisis clínicos o Hematología y bioquímica clínica o Análisis de orina o Citología urinaria \n• Anticuerpos contra la distrofina y viltolarsén \n• ECG de 12 derivaciones \n• Ecografía renal \n• AADT y AAG","language":7,"languageDescription":"Spanish"},{"id":6890871,"uuid":"0804d475-85ce-4199-828d-2aea39a8dd8b","attributeTranslation":"• Segni vitali \n• Esame obiettivo\n• Esami clinici di laboratorio\no\tEmatologia e chimica clinica\no\tEsame delle urine\no\tCitologia urinaria\n• Anticorpi anti-distrofina e anti-viltolarsen\n• Elettrocardiogramma (ECG) a 12 derivazioni\n• Ecografia renale \n• Eventi avversi emergenti dal trattamento (TEAE) ed eventi avversi seri (SAE)","language":14,"languageDescription":"Italian"}]}],"secondaryEndPoints":[{"id":685130,"number":1,"endPoint":"Secondary: - 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(Netherlands)"}]},{"id":449546,"number":2,"secondaryObjective":"To further investigate the efficacy of givinostat in non-ambulant DMD patients.","secondaryObjectiveTranslations":[{"id":10547138,"uuid":"a4d1c397-50f5-4aa3-8c1c-1c80e59993e5","attributeTranslation":"Het verder onderzoeken van de werkzaamheid van givinostat bij niet-ambulante DMD-patiënten.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547136,"uuid":"a4d1c397-50f5-4aa3-8c1c-1c80e59993e5","attributeTranslation":"Etudier plus en détails l’efficacité du givinostat chez les patients atteints de DMD non ambulatoires","language":10,"languageDescription":"French"},{"id":10547139,"uuid":"a4d1c397-50f5-4aa3-8c1c-1c80e59993e5","attributeTranslation":"Dalsza ocena skuteczności giwinostatu u niechodzących pacjentów z DMD","language":19,"languageDescription":"Polish"},{"id":10547137,"uuid":"a4d1c397-50f5-4aa3-8c1c-1c80e59993e5","attributeTranslation":"Investigar más a fondo la eficacia de givinostat en pacientes no ambulantes con DMD","language":7,"languageDescription":"Spanish"}]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":858112,"number":1,"principalInclusionCriteria":"Children and adolescent males aged ≥ 9 to <18 years at screening (patients ≥ 18 years of age at screening will not be enrolled into the study)","principalInclusionCriteriaTranslations":[{"id":10547093,"uuid":"f2cce6eb-0d91-4d58-83af-56aff0dbb185","attributeTranslation":"Enfants 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)","language":10,"languageDescription":"French"},{"id":10547092,"uuid":"f2cce6eb-0d91-4d58-83af-56aff0dbb185","attributeTranslation":"Dzieci 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)","language":19,"languageDescription":"Polish"},{"id":10547090,"uuid":"f2cce6eb-0d91-4d58-83af-56aff0dbb185","attributeTranslation":"Niños 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)","language":7,"languageDescription":"Spanish"},{"id":10547091,"uuid":"f2cce6eb-0d91-4d58-83af-56aff0dbb185","attributeTranslation":"Kinderen en jongeren van ≥ 9 tot < 18 jaar bij de screening (patiënten ≥ 18 jaar bij de screening worden niet in het onderzoek ingeschreven","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":858113,"number":2,"principalInclusionCriteria":"Are able to give informed assent and/or consent in writing signed by the patient and/or parent/legal guardian (according to local regulations)","principalInclusionCriteriaTranslations":[{"id":10547097,"uuid":"fe50ceb9-5f6e-47ea-b719-8be85d356223","attributeTranslation":"Être 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)","language":10,"languageDescription":"French"},{"id":10547095,"uuid":"fe50ceb9-5f6e-47ea-b719-8be85d356223","attributeTranslation":"Zdolność wyrażenia świadomej zgody i/lub zgody na piśmie podpisanej przez pacjenta i/lub jego rodzica/opiekuna prawnego (według miejscowych wymogów)","language":19,"languageDescription":"Polish"},{"id":10547096,"uuid":"fe50ceb9-5f6e-47ea-b719-8be85d356223","attributeTranslation":"Capacidad de dar el asentimiento o consentimiento informado por escrito firmado por el paciente o el progenitor/tutor legal (de acuerdo con la normativa local)","language":7,"languageDescription":"Spanish"},{"id":10547094,"uuid":"fe50ceb9-5f6e-47ea-b719-8be85d356223","attributeTranslation":"In staat zijn om schriftelijk geïnformeerde instemming en/of toestemming te geven, ondertekend door de patient en/of ouder/wettelijke voogd (volgens de lokale regelgeving)","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":858114,"number":3,"principalInclusionCriteria":"A genetic diagnosis of DMD","principalInclusionCriteriaTranslations":[{"id":10547101,"uuid":"298149b7-7e33-4628-9d0b-1812454b975b","attributeTranslation":"Een genetische diagnose van DMD hebben","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547099,"uuid":"298149b7-7e33-4628-9d0b-1812454b975b","attributeTranslation":"Diagnóstico genético de DMD","language":7,"languageDescription":"Spanish"},{"id":10547100,"uuid":"298149b7-7e33-4628-9d0b-1812454b975b","attributeTranslation":"Diagnostic génétique de DMD","language":10,"languageDescription":"French"},{"id":10547098,"uuid":"298149b7-7e33-4628-9d0b-1812454b975b","attributeTranslation":"Rozpoznanie genetyczne DMD","language":19,"languageDescription":"Polish"}]},{"id":858115,"number":4,"principalInclusionCriteria":"Non-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 devices","principalInclusionCriteriaTranslations":[{"id":10547102,"uuid":"bcbbfe6b-aa3b-4571-a316-8f0ca8e98b76","attributeTranslation":"Niet-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 hulpmiddelen","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547104,"uuid":"bcbbfe6b-aa3b-4571-a316-8f0ca8e98b76","attributeTranslation":"Sin ambulación, esto es, en silla de ruedas y:\na.\tsin capacidad de realizar la prueba de marcha/carrera de 10 metros (PM10m), o\nb.\tsin capacidad de completar la PM10m en un tiempo igual o inferior a 30 segundos sin ningún tipo de apoyo ni aparato","language":7,"languageDescription":"Spanish"},{"id":10547105,"uuid":"bcbbfe6b-aa3b-4571-a316-8f0ca8e98b76","attributeTranslation":"„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 pomocniczych","language":19,"languageDescription":"Polish"},{"id":10547103,"uuid":"bcbbfe6b-aa3b-4571-a316-8f0ca8e98b76","attributeTranslation":"Être non ambulatoires, c’est-à-dire être en fauteuil roulant et :\na. Ne pas être capables d’effectuer le test de marche/course de 10 mètres (10MWT), ou\nb. Ne pas être capables d’effectuer le 10MWT en 30 secondes ou moins, sans aucune aide ou dispositif","language":10,"languageDescription":"French"}]},{"id":858116,"number":5,"principalInclusionCriteria":"Performance of the Upper Limb test (PUL version 2.0) entry item scores 3 to 6","principalInclusionCriteriaTranslations":[{"id":10547107,"uuid":"adba8060-cd8d-4ef9-96ba-39b194cc3ec3","attributeTranslation":"Prestaties van de test van de bovenste ledematen (PUL versie 2.0) instapitemscores 3 tot 6","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547106,"uuid":"adba8060-cd8d-4ef9-96ba-39b194cc3ec3","attributeTranslation":"Puntuación en los ítems de entrada de la escala de rendimiento de las extremidades superiores (PUL, versión 2.0) de 3 a 6","language":7,"languageDescription":"Spanish"},{"id":10547109,"uuid":"adba8060-cd8d-4ef9-96ba-39b194cc3ec3","attributeTranslation":"Wstępne wyniki w pozycjach testu sprawności kończyn górnych (skala PUL wersja 2.0) wynoszą od 3 do 6","language":19,"languageDescription":"Polish"},{"id":10547108,"uuid":"adba8060-cd8d-4ef9-96ba-39b194cc3ec3","attributeTranslation":"Scores d’entrée de 3 à 6 au test de performance des membres supérieurs (version PUL 2.0)","language":10,"languageDescription":"French"}]},{"id":858117,"number":6,"principalInclusionCriteria":"If on medication for DMD-associated cardiomyopathy, stable for ≥1 month immediately prior to start of study treatment","principalInclusionCriteriaTranslations":[{"id":10547112,"uuid":"56b76b80-c0c1-4394-8e8c-10e5905433d6","attributeTranslation":"Si 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 échéant","language":10,"languageDescription":"French"},{"id":10547113,"uuid":"56b76b80-c0c1-4394-8e8c-10e5905433d6","attributeTranslation":"W przypadku przyjmowania leków na kardiomiopatię związaną z DMD stała dawka przez ≥1 miesiąc bezpośrednio przed rozpoczęciem leczenia w ramach badania","language":19,"languageDescription":"Polish"},{"id":10547111,"uuid":"56b76b80-c0c1-4394-8e8c-10e5905433d6","attributeTranslation":"En 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 estudio","language":7,"languageDescription":"Spanish"},{"id":10547110,"uuid":"56b76b80-c0c1-4394-8e8c-10e5905433d6","attributeTranslation":"Bij gebruik van medicatie voor DMD-geassocieerde cardiomyopathie, stabiel gedurende ≥ 1 maand onmiddellijk voorafgaand aan de start van de onderzoeksbehandeling","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":858118,"number":7,"principalInclusionCriteria":"Stable 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 treatment","principalInclusionCriteriaTranslations":[{"id":10547114,"uuid":"22817584-0a45-4069-a277-9937bdffc57a","attributeTranslation":"Kortykosteroidy 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 badania","language":19,"languageDescription":"Polish"},{"id":10547116,"uuid":"22817584-0a45-4069-a277-9937bdffc57a","attributeTranslation":"Tratamiento estable con corticoesteroides, definido de la siguiente manera:\na.\tHaber estado recibiendo corticoesteroides sistémicos durante un mínimo de 6 meses inmediatamente antes del inicio del tratamiento del estudio\nb.\tAusencia 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 estudio","language":7,"languageDescription":"Spanish"},{"id":10547115,"uuid":"22817584-0a45-4069-a277-9937bdffc57a","attributeTranslation":"Corticoïdes stables, définis comme suit :\na. Administration de corticoïdes systémiques pendant au moins 6 mois immédiatement avant le début du traitement à l’étude \nb. 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’étude","language":10,"languageDescription":"French"},{"id":10547117,"uuid":"22817584-0a45-4069-a277-9937bdffc57a","attributeTranslation":"Stabiele corticosteroïden, gedefinieerd als:\na. Het krijgen van systemische corticosteroïden gedurende minimaal 6 maanden onmiddellijk voorafgaand aan de start van de onderzoeksbehandeling\nb. 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 onderzoeksbehandeling","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":858119,"number":8,"principalInclusionCriteria":"Willing to use adequate contraception. Contraceptive methods must be used from randomisation visit through 3 months after the last dose of study drug.","principalInclusionCriteriaTranslations":[{"id":10547119,"uuid":"4ff8af40-f0f6-4a76-aa4e-0e6c472da682","attributeTranslation":"Bereid om adequate anticonceptie te gebruiken. Anticonceptiemethoden moeten worden gebruikt vanaf het randomisatiebezoek tot en met 3 maanden na de laatste dosis onderzoeksmiddel","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547120,"uuid":"4ff8af40-f0f6-4a76-aa4e-0e6c472da682","attributeTranslation":"Disposición 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 estudio","language":7,"languageDescription":"Spanish"},{"id":10547118,"uuid":"4ff8af40-f0f6-4a76-aa4e-0e6c472da682","attributeTranslation":"Chęć stosowania odpowiedniej antykoncepcji. Należy stosować metody antykoncepcji w okresie od wizyty randomizacyjnej do upływu 3 miesięcy po przyjęciu ostatniej dawki badanego leku.","language":19,"languageDescription":"Polish"},{"id":10547121,"uuid":"4ff8af40-f0f6-4a76-aa4e-0e6c472da682","attributeTranslation":"Être 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’étude","language":9,"languageDescription":"Finnish"}]}],"principalExclusionCriteria":[{"id":1467712,"number":1,"principalExclusionCriteria":"Exposure to another investigational drug within 3 months prior to start of study treatment","principalExclusionCriteriaTranslations":[{"id":10546993,"uuid":"c01ed79c-b669-43e6-9b71-a62d233ad634","attributeTranslation":"Exposición a otro medicamento en investigación en los 3 meses anteriores al inicio del tratamiento del estudio","language":7,"languageDescription":"Spanish"},{"id":10546990,"uuid":"c01ed79c-b669-43e6-9b71-a62d233ad634","attributeTranslation":"Enfants 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)","language":10,"languageDescription":"French"},{"id":10546992,"uuid":"c01ed79c-b669-43e6-9b71-a62d233ad634","attributeTranslation":"Blootstelling aan een ander onderzoeksmiddel binnen 3 maanden voorafgaand aan de start van de onderzoeksbehandeling","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10546991,"uuid":"c01ed79c-b669-43e6-9b71-a62d233ad634","attributeTranslation":"Narażenie na inny lek eksperymentalny w ciągu 3 miesięcy przed rozpoczęciem leczenia w ramach badania","language":19,"languageDescription":"Polish"}]},{"id":1467713,"number":10,"principalExclusionCriteria":"Episode of respiratory failure within the 8 weeks prior to screening","principalExclusionCriteriaTranslations":[{"id":10546994,"uuid":"9a55bc86-0a61-4e03-89d4-334aeb231245","attributeTranslation":"Episode van ademhalingsfalen binnen 8 weken voorafgaand aan de screening","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10546997,"uuid":"9a55bc86-0a61-4e03-89d4-334aeb231245","attributeTranslation":"Episodio de insuficiencia respiratoria en las 8 semanas anteriores a la selección","language":7,"languageDescription":"Spanish"},{"id":10546996,"uuid":"9a55bc86-0a61-4e03-89d4-334aeb231245","attributeTranslation":"Épisode d’insuffisance respiratoire dans les 8 semaines précédant la sélection","language":10,"languageDescription":"French"},{"id":10546995,"uuid":"9a55bc86-0a61-4e03-89d4-334aeb231245","attributeTranslation":"Epizod niewydolności oddechowej w ciągu 8 tygodni przed badaniami przesiewowymi","language":19,"languageDescription":"Polish"}]},{"id":1467714,"number":11,"principalExclusionCriteria":"Symptomatic cardiomyopathy or heart failure and/or left ventricular ejection fraction <45%","principalExclusionCriteriaTranslations":[{"id":10547001,"uuid":"b3863967-036b-4c17-88c6-75d823730822","attributeTranslation":"Cardiomyopathie symptomatique ou insuffisance cardiaque et/ou fraction d’éjection ventriculaire gauche < 45 %","language":10,"languageDescription":"French"},{"id":10546998,"uuid":"b3863967-036b-4c17-88c6-75d823730822","attributeTranslation":"Symptomatische cardiomyopathie of hartfalen en/of linkerventrikel ejectiefractie <45%","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547000,"uuid":"b3863967-036b-4c17-88c6-75d823730822","attributeTranslation":"Kardiomiopatia objawowa lub niewydolność serca i/lub wartość frakcji wyrzutowej lewej komory <45%","language":19,"languageDescription":"Polish"},{"id":10546999,"uuid":"b3863967-036b-4c17-88c6-75d823730822","attributeTranslation":"Miocardiopatía sintomática o insuficiencia cardíaca o fracción de eyección ventricular izquierda <45 %","language":7,"languageDescription":"Spanish"}]},{"id":1467715,"number":12,"principalExclusionCriteria":"Baseline 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)","principalExclusionCriteriaTranslations":[{"id":10547005,"uuid":"32b9f55d-5114-4db6-8cad-e5c5edececc0","attributeTranslation":"Intervalle 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’étude","language":10,"languageDescription":"French"},{"id":10547002,"uuid":"32b9f55d-5114-4db6-8cad-e5c5edececc0","attributeTranslation":"Skorygowany 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)","language":19,"languageDescription":"Polish"},{"id":10547004,"uuid":"32b9f55d-5114-4db6-8cad-e5c5edececc0","attributeTranslation":"Gecorrigeerd 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)","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547003,"uuid":"32b9f55d-5114-4db6-8cad-e5c5edececc0","attributeTranslation":"Intervalo 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)","language":7,"languageDescription":"Spanish"}]},{"id":1467716,"number":13,"principalExclusionCriteria":"Major surgical procedure (including scoliosis surgery) planned within 1 year of the start of study treatment","principalExclusionCriteriaTranslations":[{"id":10547007,"uuid":"675738fd-2028-40d8-9432-8f53979fd5cf","attributeTranslation":"Poważny zabieg chirurgiczny (w tym operacja skoliozy) zaplanowany w ciągu 1 roku od rozpoczęcia leczenia w ramach badania","language":19,"languageDescription":"Polish"},{"id":10547008,"uuid":"675738fd-2028-40d8-9432-8f53979fd5cf","attributeTranslation":"Intervención de cirugía mayor (incluida la cirugía de escoliosis) prevista en el plazo de 1 año con respecto al inicio del tratamiento del estudio","language":7,"languageDescription":"Spanish"},{"id":10547009,"uuid":"675738fd-2028-40d8-9432-8f53979fd5cf","attributeTranslation":"Intervention chirurgicale majeure (y compris chirurgie de la scoliose) prévue dans l’année précédant le début du traitement à l’étude","language":10,"languageDescription":"French"},{"id":10547006,"uuid":"675738fd-2028-40d8-9432-8f53979fd5cf","attributeTranslation":"Grote chirurgische ingreep (waaronder scoliose-operatie) gepland binnen 1 jaar vóór de start van de onderzoeksbehandeling","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1467717,"number":14,"principalExclusionCriteria":"Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the Investigator might impact respiratory function","principalExclusionCriteriaTranslations":[{"id":10547012,"uuid":"8cb5b891-ca68-43d2-8c99-3a03026967d2","attributeTranslation":"Niedostatecznie 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 oddechowego","language":19,"languageDescription":"Polish"},{"id":10547010,"uuid":"8cb5b891-ca68-43d2-8c99-3a03026967d2","attributeTranslation":"Asthme 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 respiratoire","language":10,"languageDescription":"French"},{"id":10547013,"uuid":"8cb5b891-ca68-43d2-8c99-3a03026967d2","attributeTranslation":"Asma mal controlada o neumopatía subyacente, como bronquitis, bronquiectasia, enfisema o neumonía recurrente que, en opinión del investigador, podría afectar a la función respiratoria","language":7,"languageDescription":"Spanish"},{"id":10547011,"uuid":"8cb5b891-ca68-43d2-8c99-3a03026967d2","attributeTranslation":"Slecht gecontroleerde astma of onderliggende longziekte zoals bronchitis, bronchiëctasie, emfyseem, herhaalde longontsteking die naar het oordeel van de onderzoeker invloed kan hebben op de ademhalingsfunctie","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1467718,"number":15,"principalExclusionCriteria":"Platelets, white blood cells and/or haemoglobin < lower limit of normal (LLN) at screening","principalExclusionCriteriaTranslations":[{"id":10547014,"uuid":"9ffad6d8-b45d-43f4-9619-b7ebc1592170","attributeTranslation":"Plaquetas, leucocitos o hemoglobina ","language":7,"languageDescription":"Spanish"},{"id":10547016,"uuid":"9ffad6d8-b45d-43f4-9619-b7ebc1592170","attributeTranslation":"Płytki krwi, krwinki białe i/lub hemoglobina < dolnej granicy normy (DGN) podczas badań przesiewowych","language":19,"languageDescription":"Polish"},{"id":10547015,"uuid":"9ffad6d8-b45d-43f4-9619-b7ebc1592170","attributeTranslation":"Bloedplaatjes, witte bloedcellen en/of hemoglobine < ondergrens van normaal (LLN) bij de screening","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547017,"uuid":"9ffad6d8-b45d-43f4-9619-b7ebc1592170","attributeTranslation":"Plaquettes, 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)","language":10,"languageDescription":"French"}]},{"id":1467719,"number":16,"principalExclusionCriteria":"Fasting triglycerides >300 mg/dL (3.42 mmol/L) at screening","principalExclusionCriteriaTranslations":[{"id":10547019,"uuid":"a67934c1-75f4-420d-b9e9-675ff6465d7f","attributeTranslation":"Nuchtere triglyceriden > 300 mg/dl (3,42 mmol/l) bij de screening","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547021,"uuid":"a67934c1-75f4-420d-b9e9-675ff6465d7f","attributeTranslation":"Stężenie trójglicerydów na czczo >300 mg/dl (3,42 mmol/l) podczas badań przesiewowych","language":19,"languageDescription":"Polish"},{"id":10547020,"uuid":"a67934c1-75f4-420d-b9e9-675ff6465d7f","attributeTranslation":"Triglicéridos en ayunas >300 mg/dl (3,42 mmol/l) en la selección","language":7,"languageDescription":"Spanish"},{"id":10547018,"uuid":"a67934c1-75f4-420d-b9e9-675ff6465d7f","attributeTranslation":"Triglycérides 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)","language":10,"languageDescription":"French"}]},{"id":1467720,"number":17,"principalExclusionCriteria":"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 disease","principalExclusionCriteriaTranslations":[{"id":10547023,"uuid":"b7f26101-1b7d-41c0-874a-7b9ccda5de95","attributeTranslation":"Maladie 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 Gilbert","language":10,"languageDescription":"French"},{"id":10547024,"uuid":"b7f26101-1b7d-41c0-874a-7b9ccda5de95","attributeTranslation":"Huidige 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 Gilbert","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547022,"uuid":"b7f26101-1b7d-41c0-874a-7b9ccda5de95","attributeTranslation":"Występująca 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 Gilberta","language":19,"languageDescription":"Polish"},{"id":10547025,"uuid":"b7f26101-1b7d-41c0-874a-7b9ccda5de95","attributeTranslation":"Antecedentes 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 Gilbert","language":7,"languageDescription":"Spanish"}]},{"id":1467721,"number":18,"principalExclusionCriteria":"Inadequate renal function, as defined by serum Cystatin C result >2 × ULN","principalExclusionCriteriaTranslations":[{"id":10547027,"uuid":"1357ede9-d146-442e-9468-346439a24a8f","attributeTranslation":"Fonction 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\nexclu)","language":9,"languageDescription":"Finnish"},{"id":10547028,"uuid":"1357ede9-d146-442e-9468-346439a24a8f","attributeTranslation":"Niedostateczna czynność nerek, zdefiniowana jako wynik stężenia cystatyny C w surowicy >2 × GGN","language":19,"languageDescription":"Polish"},{"id":10547026,"uuid":"1357ede9-d146-442e-9468-346439a24a8f","attributeTranslation":"Onvoldoende nierfunctie, zoals gedefinieerd met een cystatine C-serumresultaat > 2 × ULN","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547029,"uuid":"1357ede9-d146-442e-9468-346439a24a8f","attributeTranslation":"Función renal inadecuada, definida por un resultado de cistatina C sérica >2 × LSN","language":7,"languageDescription":"Spanish"}]},{"id":1467722,"number":19,"principalExclusionCriteria":"Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening","principalExclusionCriteriaTranslations":[{"id":10547031,"uuid":"b6e34cba-869f-4fe6-82a8-3f01749af68f","attributeTranslation":"Dodatni 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 przesiewowych","language":19,"languageDescription":"Polish"},{"id":10547033,"uuid":"b6e34cba-869f-4fe6-82a8-3f01749af68f","attributeTranslation":"Test 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 sélection","language":10,"languageDescription":"French"},{"id":10547030,"uuid":"b6e34cba-869f-4fe6-82a8-3f01749af68f","attributeTranslation":"Resultado 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 selección","language":7,"languageDescription":"Spanish"},{"id":10547032,"uuid":"b6e34cba-869f-4fe6-82a8-3f01749af68f","attributeTranslation":"Positieve test voor hepatitis B-oppervlakteantigeen, hepatitis C-virus antilichaamserologie of hiv bij de screening","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1467723,"number":2,"principalExclusionCriteria":"Have exposure to any dystrophin restoration product within 6 months prior to the start of study treatment","principalExclusionCriteriaTranslations":[{"id":10547036,"uuid":"74716e32-0556-4e98-ad01-da0f6ad5d87b","attributeTranslation":"Blootstelling aan een dystrofine-herstellend product binnen 6 maanden voorafgaand aan de start van de onderzoeksbehandeling","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547035,"uuid":"74716e32-0556-4e98-ad01-da0f6ad5d87b","attributeTranslation":"Exposición a cualquier fármaco de restauración de la distrofina en los 6 meses anteriores al inicio del tratamiento del estudio","language":7,"languageDescription":"Spanish"},{"id":10547037,"uuid":"74716e32-0556-4e98-ad01-da0f6ad5d87b","attributeTranslation":"Exposition 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’étude","language":10,"languageDescription":"French"},{"id":10547034,"uuid":"74716e32-0556-4e98-ad01-da0f6ad5d87b","attributeTranslation":"Narażenie na jakikolwiek produkt przywracający dystrofinę w ciągu 6 miesięcy przed rozpoczęciem leczenia w ramach badania","language":19,"languageDescription":"Polish"}]},{"id":1467724,"number":20,"principalExclusionCriteria":"Hypersensitivity to any component of study medication","principalExclusionCriteriaTranslations":[{"id":10547041,"uuid":"a6faaa70-dc86-44d4-805a-826318161b82","attributeTranslation":"Overgevoeligheid voor een bestanddeel van het onderzoeksmiddel","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547039,"uuid":"a6faaa70-dc86-44d4-805a-826318161b82","attributeTranslation":"Hypersensibilité à l’un des composants du médicament à l’étude","language":10,"languageDescription":"French"},{"id":10547038,"uuid":"a6faaa70-dc86-44d4-805a-826318161b82","attributeTranslation":"Hipersensibilidad conocida a cualquiera de los componentes del fármaco del estudio","language":7,"languageDescription":"Spanish"},{"id":10547040,"uuid":"a6faaa70-dc86-44d4-805a-826318161b82","attributeTranslation":"Nadwrażliwość na którykolwiek składnik badanego leku","language":19,"languageDescription":"Polish"}]},{"id":1467725,"number":21,"principalExclusionCriteria":"Sorbitol intolerance or malabsorption, or the hereditary form of fructose intolerance","principalExclusionCriteriaTranslations":[{"id":10547045,"uuid":"8b4e0531-b360-43fc-8532-e7c70d9c59f3","attributeTranslation":"Intolérance ou malabsorption du sorbitol, ou forme héréditaire d’intolérance au fructose","language":10,"languageDescription":"French"},{"id":10547043,"uuid":"8b4e0531-b360-43fc-8532-e7c70d9c59f3","attributeTranslation":"Intolerantie of malabsorptie van sorbitol, of de erfelijke vorm van fructose-intolerantie","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547042,"uuid":"8b4e0531-b360-43fc-8532-e7c70d9c59f3","attributeTranslation":"Nietolerancja lub złe wchłanianie sorbitolu bądź dziedziczna postać nietolerancji fruktozy","language":19,"languageDescription":"Polish"},{"id":10547044,"uuid":"8b4e0531-b360-43fc-8532-e7c70d9c59f3","attributeTranslation":"Intolerancia al sorbitol o malabsorción, o la forma hereditaria de intolerancia a la fructosa","language":7,"languageDescription":"Spanish"}]},{"id":1467726,"number":22,"principalExclusionCriteria":"Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator judgement","principalExclusionCriteriaTranslations":[{"id":10547049,"uuid":"354f0e64-5291-46be-9175-8b948f918db3","attributeTranslation":"Diagnóstico 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 investigador","language":7,"languageDescription":"Spanish"},{"id":10547048,"uuid":"354f0e64-5291-46be-9175-8b948f918db3","attributeTranslation":"Diagnose van andere ongecontroleerde neurologische ziekten of aanwezigheid van relevante ongecontroleerde somatische aandoeningen die geen verband houden met DMD, naar inzicht van de onderzoeker","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547047,"uuid":"354f0e64-5291-46be-9175-8b948f918db3","attributeTranslation":"Diagnostic 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’investigateur","language":10,"languageDescription":"French"},{"id":10547046,"uuid":"354f0e64-5291-46be-9175-8b948f918db3","attributeTranslation":"Rozpoznanie innych niekontrolowanych chorób neurologicznych lub występowanie istotnych niekontrolowanych zaburzeń somatycznych, które nie są związane z DMD, na podstawie oceny badacza","language":19,"languageDescription":"Polish"}]},{"id":1467727,"number":23,"principalExclusionCriteria":"Psychiatric 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 judgement","principalExclusionCriteriaTranslations":[{"id":10547051,"uuid":"31d44274-65c9-4203-82f4-c7d76c0dea96","attributeTranslation":"Enfermedad 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 investigador","language":7,"languageDescription":"Spanish"},{"id":10547050,"uuid":"31d44274-65c9-4203-82f4-c7d76c0dea96","attributeTranslation":"Psychiatrische 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 onderzoeker","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547053,"uuid":"31d44274-65c9-4203-82f4-c7d76c0dea96","attributeTranslation":"Choroba psychiczna lub sytuacje społeczne uniemożliwiające potencjalnemu pacjentowi zrozumienie i wykonywanie badań czynności mięśni i/lub procedur protokołu badania, na podstawie oceny badacza","language":19,"languageDescription":"Polish"},{"id":10547052,"uuid":"31d44274-65c9-4203-82f4-c7d76c0dea96","attributeTranslation":"Maladie 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’investigateur","language":10,"languageDescription":"French"}]},{"id":1467728,"number":24,"principalExclusionCriteria":"Have contraindications to Magnetic Resonance Imaging (MRI) scan (eg, claustrophobia, metal implants, or uncontrolled seizure disorder), based on Investigator’s judgement","principalExclusionCriteriaTranslations":[{"id":10547057,"uuid":"98c5498a-2cf2-4402-a5d3-4ea0b25d2844","attributeTranslation":"Contra-indicaties hebben voor magnetische resonantiebeeldvorming (MRI)-scan (bijv. claustrofobie, metalen implantaten of ongecontroleerde epileptische aandoening), naar inzicht van de onderzoeker.","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547055,"uuid":"98c5498a-2cf2-4402-a5d3-4ea0b25d2844","attributeTranslation":"Presencia de contraindicaciones a la resonancia magnética (RM) (p. ej., claustrofobia, implantes metálicos o trastorno convulsivo no controlado), según el criterio del investigador","language":7,"languageDescription":"Spanish"},{"id":10547056,"uuid":"98c5498a-2cf2-4402-a5d3-4ea0b25d2844","attributeTranslation":"Avoir 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’investigateur","language":10,"languageDescription":"French"},{"id":10547054,"uuid":"98c5498a-2cf2-4402-a5d3-4ea0b25d2844","attributeTranslation":"Występowanie przeciwwskazań do wykonywania badań obrazowych metodą rezonansu magnetycznego (MRI) (np. klaustrofobia, metalowe implanty lub niekontrolowane napady drgawkowe), na podstawie oceny badacza","language":19,"languageDescription":"Polish"}]},{"id":1467729,"number":3,"principalExclusionCriteria":"Having received any gene therapy prior to start of study treatment","principalExclusionCriteriaTranslations":[{"id":10547061,"uuid":"6cf02a20-52a1-43df-a7a9-8fb1d5b81116","attributeTranslation":"Avoir reçu une thérapie génique (p. ex. virus adéno-associés, administration de microdystrophine) avant le début du traitement à l’étude","language":10,"languageDescription":"French"},{"id":10547058,"uuid":"6cf02a20-52a1-43df-a7a9-8fb1d5b81116","attributeTranslation":"Cualquier terapia génica antes del inicio del tratamiento del estudio","language":7,"languageDescription":"Spanish"},{"id":10547059,"uuid":"6cf02a20-52a1-43df-a7a9-8fb1d5b81116","attributeTranslation":"Een gentherapie hebben ontvangen voorafgaand aan de start van de onderzoeksbehandeling","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547060,"uuid":"6cf02a20-52a1-43df-a7a9-8fb1d5b81116","attributeTranslation":"Otrzymanie jakiejkolwiek terapii genowej przed rozpoczęciem leczenia w ramach badania","language":19,"languageDescription":"Polish"}]},{"id":1467730,"number":4,"principalExclusionCriteria":"Use 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.","principalExclusionCriteriaTranslations":[{"id":10547065,"uuid":"a298d319-9276-49cf-baa2-b49af424884c","attributeTranslation":"Stosowanie 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.","language":19,"languageDescription":"Polish"},{"id":10547062,"uuid":"a298d319-9276-49cf-baa2-b49af424884c","attributeTranslation":"Cualquier 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 suplemento","language":7,"languageDescription":"Spanish"},{"id":10547064,"uuid":"a298d319-9276-49cf-baa2-b49af424884c","attributeTranslation":"Utilisation 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 autorisés","language":10,"languageDescription":"French"},{"id":10547063,"uuid":"a298d319-9276-49cf-baa2-b49af424884c","attributeTranslation":"Gebruik 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 toegestaan","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1467731,"number":5,"principalExclusionCriteria":"Use 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.","principalExclusionCriteriaTranslations":[{"id":10547067,"uuid":"082e31e3-7e15-4864-b467-8ccbb8d05695","attributeTranslation":"Stosowanie 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.","language":19,"languageDescription":"Polish"},{"id":10547066,"uuid":"082e31e3-7e15-4864-b467-8ccbb8d05695","attributeTranslation":"Uso 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 estudio","language":7,"languageDescription":"Spanish"},{"id":10547069,"uuid":"082e31e3-7e15-4864-b467-8ccbb8d05695","attributeTranslation":"Utilisation 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\npour l’âge du/de la patient(e)","language":10,"languageDescription":"French"},{"id":10547068,"uuid":"082e31e3-7e15-4864-b467-8ccbb8d05695","attributeTranslation":"Gebruik 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.","language":37,"languageDescription":"Dutch (Netherlands)"}]},{"id":1467732,"number":6,"principalExclusionCriteria":"Elbow-flexion contractures >30° in the dominant arm","principalExclusionCriteriaTranslations":[{"id":10547071,"uuid":"8a6f50fd-6d38-4e15-8019-74e8640e9d7a","attributeTranslation":"Contractura en flexión de codo >30° en el brazo dominante","language":7,"languageDescription":"Spanish"},{"id":10547070,"uuid":"8a6f50fd-6d38-4e15-8019-74e8640e9d7a","attributeTranslation":"Contractures de flexion du coude > 30° dans le groupe dominant","language":10,"languageDescription":"French"},{"id":10547073,"uuid":"8a6f50fd-6d38-4e15-8019-74e8640e9d7a","attributeTranslation":"Elleboogflexie contracturen > 30° in de dominante arm","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547072,"uuid":"8a6f50fd-6d38-4e15-8019-74e8640e9d7a","attributeTranslation":"Przykurcze zgięciowe łokcia >30° w ramieniu dominującym","language":19,"languageDescription":"Polish"}]},{"id":1467733,"number":7,"principalExclusionCriteria":"Inability to perform consistent PUL 2.0 measurement within ±2 points without shoulder domain or within ±3 points with shoulder domain during paired testing at screening","principalExclusionCriteriaTranslations":[{"id":10547077,"uuid":"764310ce-5fe5-494d-a844-020840a5c499","attributeTranslation":"Incapacidad 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 selección","language":7,"languageDescription":"Spanish"},{"id":10547074,"uuid":"764310ce-5fe5-494d-a844-020840a5c499","attributeTranslation":"Niemożność 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 przesiewowych","language":19,"languageDescription":"Polish"},{"id":10547076,"uuid":"764310ce-5fe5-494d-a844-020840a5c499","attributeTranslation":"Niet 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 screening","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547075,"uuid":"764310ce-5fe5-494d-a844-020840a5c499","attributeTranslation":"Incapacité 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 sélection","language":10,"languageDescription":"French"}]},{"id":1467734,"number":8,"principalExclusionCriteria":"Forced Vital Capacity % of predicted <40%","principalExclusionCriteriaTranslations":[{"id":10547079,"uuid":"1a3bd170-613f-4711-86fa-84ce97043925","attributeTranslation":"Percentage voorspelde geforceerde vitale capaciteit (FVC) < 40%","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547081,"uuid":"1a3bd170-613f-4711-86fa-84ce97043925","attributeTranslation":"Capacité vitale forcée (CVF) en pourcentage de la valeur prédite < 40 %","language":10,"languageDescription":"French"},{"id":10547080,"uuid":"1a3bd170-613f-4711-86fa-84ce97043925","attributeTranslation":"Przewidywana wartość procentowa natężonej pojemności życiowej <40%","language":19,"languageDescription":"Polish"},{"id":10547078,"uuid":"1a3bd170-613f-4711-86fa-84ce97043925","attributeTranslation":"Capacidad vital forzada (CVF) en porcentaje del valor previsto <40 %","language":7,"languageDescription":"Spanish"}]},{"id":1467735,"number":9,"principalExclusionCriteria":"Requirement for daytime ventilator assistance","principalExclusionCriteriaTranslations":[{"id":10547082,"uuid":"7657eaa9-c24a-47eb-ad93-4d2a4d6f0f45","attributeTranslation":"Konieczność stosowania wspomagania wentylacji w ciągu dnia","language":19,"languageDescription":"Polish"},{"id":10547085,"uuid":"7657eaa9-c24a-47eb-ad93-4d2a4d6f0f45","attributeTranslation":"Necesidad de asistencia respiratoria durante el día","language":7,"languageDescription":"Spanish"},{"id":10547083,"uuid":"7657eaa9-c24a-47eb-ad93-4d2a4d6f0f45","attributeTranslation":"Beademingsondersteuning overdag vereist","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10547084,"uuid":"7657eaa9-c24a-47eb-ad93-4d2a4d6f0f45","attributeTranslation":"Nécessité 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 autorisées","language":10,"languageDescription":"French"}]}]},"endPoint":{"primaryEndPoints":[{"id":1004875,"number":1,"endPoint":"Change of PUL total score at 18 months of treatment of givinostat compared to placebo group","isPrimary":true,"endPointTranslations":[{"id":10546978,"uuid":"78f930d7-0d7e-4a19-9240-c4db0e12c73c","attributeTranslation":"•\tVariation du score total PUL à 18 mois de traitement par givinostat par rapport au groupe placebo.","language":10,"languageDescription":"French"},{"id":10546977,"uuid":"78f930d7-0d7e-4a19-9240-c4db0e12c73c","attributeTranslation":"Cambio en la puntuación total de la escala PUL a los 18 meses de tratamiento con givinostat en comparación con el grupo de placebo","language":7,"languageDescription":"Spanish"},{"id":10546979,"uuid":"78f930d7-0d7e-4a19-9240-c4db0e12c73c","attributeTranslation":"Zmiana całkowitego wyniku w skali PUL po 18 miesiącach leczenia giwinostatem w porównaniu z grupą otrzymującą placebo","language":19,"languageDescription":"Polish"},{"id":10546980,"uuid":"78f930d7-0d7e-4a19-9240-c4db0e12c73c","attributeTranslation":"Verandering in de PUL-totaalscore na 18 maanden behandeling met givinostat vergeleken met de placebogroep.","language":37,"languageDescription":"Dutch (Netherlands)"}]}],"secondaryEndPoints":[{"id":1004876,"number":1,"endPoint":"•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","isPrimary":false,"endPointTranslations":[{"id":10546982,"uuid":"b62cf1d7-03db-4801-9255-6a6eff7db49a","attributeTranslation":"Cambio 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\n•\tCambio 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\n•\tPérdida acumulada de ítems de la escala PUL durante 18 meses de tratamiento con givinostat en comparación con el grupo de placebo","language":7,"languageDescription":"Spanish"},{"id":10546983,"uuid":"b62cf1d7-03db-4801-9255-6a6eff7db49a","attributeTranslation":"Verandering 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 placebogroep","language":37,"languageDescription":"Dutch (Netherlands)"},{"id":10546984,"uuid":"b62cf1d7-03db-4801-9255-6a6eff7db49a","attributeTranslation":"•\tVariation 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\n•\tVariation 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\n•\tPerte cumulée des items PUL après 18 mois de traitement par givinostat par rapport au groupe placebo.","language":10,"languageDescription":"French"},{"id":10546981,"uuid":"b62cf1d7-03db-4801-9255-6a6eff7db49a","attributeTranslation":"Zmiana 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 placebo","language":19,"languageDescription":"Polish"}]},{"id":1004877,"number":2,"endPoint":"•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-scores","isPrimary":false,"endPointTranslations":[{"id":10546989,"uuid":"2dd85a63-c0a5-4d9d-ab5a-7af4dcd51891","attributeTranslation":"Rodzaj, częstość występowania i nasilenie TEAE • Odsetek pacjentów, u których wystąpiły TEAE (w okresie od punktu wyjściowego do zakończenia badania/obserwacji kontrolnej) •\nZmiany 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 •","language":19,"languageDescription":"Polish"},{"id":10546987,"uuid":"2dd85a63-c0a5-4d9d-ab5a-7af4dcd51891","attributeTranslation":"- 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 \n- Perte cumulée du score total PUL après 18 mois de traitement par givinostat par rapport au groupe placebo\n- Délai jusqu’à l’apparition de la diarrhée\n- Z-scores de la taille et du poids","language":10,"languageDescription":"French"},{"id":10546988,"uuid":"2dd85a63-c0a5-4d9d-ab5a-7af4dcd51891","attributeTranslation":"Polish, continue: Czas do wystąpienia biegunki • Wyniki dotyczące wzrostu i masy ciała","language":40,"languageDescription":"Other"},{"id":10546985,"uuid":"2dd85a63-c0a5-4d9d-ab5a-7af4dcd51891","attributeTranslation":"•\tTipo, incidencia e intensidad de los AAST\n•\tProporción de pacientes con AAST (desde el inicio hasta el FDE/seguimiento)\n•\tCambio en las constantes vitales, los análisis clínicos, el electrocardiograma (ECG) y el ecocardiograma (ECO)\n•\tTiempo 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\n•\tTiempo hasta la aparición de diarrea\n•\tPuntuaciones Z de peso y estatura","language":7,"languageDescription":"Spanish"},{"id":10546986,"uuid":"2dd85a63-c0a5-4d9d-ab5a-7af4dcd51891","attributeTranslation":"Type, 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\n\nen duur en gebruik van antibiotica met givinostat vergeleken met de placebogroep • tijd tot begin van diarree • Lengte en gewicht Z scores","language":37,"languageDescription":"Dutch (Netherlands)"}]}]},"trialDuration":{"estimatedGlobalEndDate":"2027-12-30","estimatedEndDate":"2027-12-30","estimatedRecruitmentStartDate":"2023-12-30"},"sourceOfMonetarySupport":[{"id":92923,"organisationName":"Italfarmaco"}],"populationOfTrialSubjects":{"ageRanges":[{"id":348870,"ageRangeCategoryCode":"2","ageRangeCategory":"2"}],"ageRangeSecondaryIds":[],"clinicalTrialGroups":[{"code":"2","name":"Patients"}],"isFemaleSubjects":false,"isMaleSubjects":true,"isVulnerablePopulationSelected":true},"individualParticipantData":{"planToShareIPD":"3","planDescription":"NAP"}},"protocolInformation":{"studyDesign":{"periodDetails":[{"id":182895,"businessKey":"1","title":"Main 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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.","armDetails":[{"description":"2/3 of patients will be randomized to givinostat  will be treated for 18 months  twice daily (bid) in a fed state.","title":"Test","id":102104},{"description":"1/3 of patients will be randomized to placebo and  will be treated for 18 months  twice daily (bid) in a fed state.","title":"placebo","id":102105}],"allocationMethod":"1"}]}},"scientificAdviceAndPip":{"scientificAdvices":[],"paediatricInvestigationPlan":[{"id":11291,"paediatricInvestigationNumber":"EMEA-000551-PIP04-21"}]},"associatedClinicalTrials":[],"references":[{"id":523765,"number":"PMID 27566866"}],"pubmedCode":["PMID 27566866"],"pubmedUrl":["https://pubmed.ncbi.nlm.nih.gov/27566866"]},"assessmentOutcome":"acceptable","therapeuticAreas":[{"code":"5","name":"Diseases [C] - Musculoskeletal Diseases [C05]"}],"medicalConditions":[{"id":132405,"medicalCondition":"Duchenne muscular dystrophy 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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).","publicTitleTranslations":[{"id":10383911,"uuid":"70796e38-3464-4884-baac-0f0edf079fb0","attributeTranslation":"Une 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).","language":34,"languageDescription":"French (France)"},{"id":10383912,"uuid":"70796e38-3464-4884-baac-0f0edf079fb0","attributeTranslation":"Un 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).","language":7,"languageDescription":"Spanish"}],"shortTitle":"GNT-016-MDYF","secondaryIdentifyingNumbers":{"additionalRegistries":[]}},"trialInformation":{"trialCategory":{"isLowIntervention":false,"trialPhase":"7","trialCategory":"1","justificationForTrialCategory":"Phase I/II trial","trialCategoryId":115086},"medicalCondition":{"partIMedicalConditions":[{"id":130595,"medicalCondition":"Duchenne Muscular Dystrophy","medicalConditionTranslations":[{"id":10383878,"uuid":"1a697b45-6814-439c-ba7b-41da8b532765","attributeTranslation":"Distrofia muscular de Duchenne","language":7,"languageDescription":"Spanish"}],"isConditionRareDisease":true}],"meddraConditionTerms":[{"termId":100000012544,"version":"20.0","level":"PT","termName":"Duchenne muscular dystrophy","classificationCode":"10013801","organClass":100000004850,"active":false}]},"trialObjective":{"trialScopes":[{"code":"9","trialScopeId":379734},{"code":"7","trialScopeId":379731},{"code":"4","trialScopeId":379729},{"code":"6","trialScopeId":379732},{"code":"3","trialScopeId":379733},{"code":"5","trialScopeId":379730}],"mainObjective":"A phase I/II/III study consisting of 3 parts:\n- Part 1: To determine the dose of IMP: a safe and tolerable dose with acceptable gene expression, to carry over to part 2.\n- 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.\n- Part 3. 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    [28,"2022-500703-49-01",4,"Authorised","Phase 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.","AVANCE1-1/2a","Duchenne muscular dystrophy",["France:4"],"21/11/2022","FR: 21/11/2022",["Diseases [C] - Musculoskeletal Diseases [C05]"],"Sqy Therapeutics","Pharmaceutical company","Phase I and Phase II (Integrated)- First administration to humans","Phase 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. -\tTwitch 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: \t- 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.","",["5","9","6"],"18-64 years, 0-17 years","Male",1,"EEA","12","Type, 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.","No","27/02/2026","28/02/2026","2022-11-21T09:35:18.445","2026-02-28T02:36:45.762945083",4,[],[{"mscName":"France","mscId":1902,"firstDecisionDate":"2022-11-21T09:35:18.445","lastDecisionDate":"2026-02-27T15:06:30.845","mscPublicStatusCode":4}],{"temporaryHaltList":[{"mscId":1902,"businessKey":"TH-17341","haltDate":"2024-03-15","reasonList":[{"code":"6","name":"Medicinal Product related","isSmRequiredForRestart":false,"isCommentRequired":false}],"isTreatmentStopped":true,"sponsorJustificationComment":"Following 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).\n\nBelow is the information recorded in the batch certificates - investigational medicinal product for human use issued by COLCA /EUROMED PHARMA:\n-\tLot DPSQYM001A expiry date 06/2023, batch certificate signed by QP on 20/12/2022\n-\tLot DPSQYM001B expiry date 05/2024, batch certificate signed by QP on 14/09/2023\n-\tLot DPSQYM001C expiry date 05/2024, batch certificate signed by QP on 14/09/2023\n\nSQY51 Finished Product Information:\n-\tAbsence of a technical batch for SQY51 performed prior to the start of the clinical study\n-\t1st 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\n-\tStabilization of the DPSQYM001 batch at AXOLABS in July 2022 according to the following scheme (Q0 &#34;analytical&#34; &#61; 27/07/2022):\n         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 \n         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 \n         o Stress test / Storage in stability chamber at &#43;40°C &#43;/- 2°C / T0 &#43; T1M \n\n-\tStability 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.\n-\tExtrapolation 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)\n\nIn 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):\n-\tExpiry date 11/2022, on the date of signature of the batch certificate on 20/12/2022\n-\tExpiry Date 07/2023, on the date of signature of the batch certificate on 14/09/2023\n\nPlease 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.\n\nFollowing 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.\nThe last administration of this batch took place at the investigator site on 11/03/2024 as part of this AVANCE 1 - phase 1 study.\nThis Phase 1 study has not been yet  finalized. Upcoming visits are described in Appendix 1 of the attached letter.\n\nCurrently, 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.\n\nPart of this batch has been sent to the investigator site, but no administration will take place without the authorization of the ANSM.\n\nSQY 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.","isBenefitRiskBalanceChange":false,"submitDate":"2024-03-15T17:58:24","subjectFuMeasuresComment":"This same notification will be the subject of an email communication.\n\nThe actions SQY Therapeutics intends to implement are as follows:\n-\tSuspension 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.\n-\tContinuation of analyses, recruitment and follow-up visits of the AVANCE 1 – Phase 1 clinical study, as mentioned in Appendix 1.\n-\tEmergency analysis of the remaining vials of the batch DPSQYM001\n-\tCommunication with patients after approval by the ANSM\n-\tCommunication with the DSMB/steering committee after approval by the ANSM\n-\tCommunication with the hospital of the investigator site after approval by the ANSM","isPublished":false,"updatedOn":"2024-03-15","mscList":[{"mscId":1902,"mscCountryName":"France","mscCountryCode":"FR"}]},{"mscId":1902,"businessKey":"TH-76448","haltDate":"2025-03-14","reasonList":[{"code":"4","name":"Safety related (clinical or pre-clinical results)","isSmRequiredForRestart":false,"isCommentRequired":false}],"isTreatmentStopped":true,"sponsorJustificationComment":"The 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).\nThis 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.\nPlease refer to the attached Cover letter for a detailed overview.\nAttached are CIOMS forms for patients 01-015 and 01-008 and 01-002.\nData collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.","isBenefitRiskBalanceChange":false,"submitDate":"2025-03-26T09:37:33","subjectFuMeasuresComment":"A DSMB meeting was held on March 21st to discuss these events and potential measures, meeting minutes are ongoing.\nA 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. \nEncouragingly, 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.\nAn urgent meeting with the Agency is requested to discuss this data, protocol adjustments, and the continuation of treatment for favorable responders.\nFinally, an extension is requested for the substantial protocol amendment submission deadline (currently April 14thfollowing DSUR RFIs).\n A USM is submitted in parallel of this notification","isPublished":false,"mscList":[{"mscId":1902,"mscCountryName":"France","mscCountryCode":"FR"}]}],"trialEvents":[{"mscId":1902,"mscName":"France","events":[{"notificationType":"RESTART_OF_TRIAL","date":"2025-11-05"},{"notificationType":"START_OF_TRIAL","date":"2023-01-05"},{"notificationType":"RESTART_OF_RECRUITMENT","date":"2024-09-09"},{"notificationType":"START_OF_RECRUITMENT","date":"2023-04-26"},{"notificationType":"TEMPORARY_HALT","date":"2025-03-14"}]}],"unexpectedEvents":[],"seriousBreaches":[],"urgentSafetyMeasures":[{"sponsorBusinessKey":"USM #1","businessKey":"US-51783","eventDate":"2024-10-04","submissionDate":"2024-10-15","updatedOn":"2024-10-15","inResponseTo":"OTHER","susarIdList":[],"eventDescription":"As 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.\n\nOn 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.\nOn 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.","measuresTakenDescription":"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: \n\n- Cohort 10 mg/kg : 6 participants (including 4 of the heaviest) ; \n- Cohort 16 mg/kg: 3 participants; \n- Cohort 25 mg/kg: 3 participants. \n\nDSMB members approved this reassignment. This urgent safety measure will be endorsed by a substantial modification for authorization.","mscs":["France"],"unexpectedEvents":[]},{"sponsorBusinessKey":"USM 2","businessKey":"US-76442","eventDate":"2025-03-18","submissionDate":"2025-03-26","updatedOn":"2025-03-26","inResponseTo":"OTHER","susarIdList":[],"eventDescription":"The 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).\nThis 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.\nPlease refer to the attached Cover letter for a detailed overview.\nAttached 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).\nData collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.","measuresTakenDescription":"Data collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.\nA DSMB meeting was held on March 21st to discuss these events and potential measures, meeting minutes are ongoing.\n 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. \n 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.\n An urgent meeting with the Agency is requested to discuss this data, protocol adjustments, and the continuation of treatment for favorable responders.\nFinally, an extension is requested for the substantial protocol amendment submission deadline (currently April 14thfollowing DSUR RFIs).\n A temporary halt is submitted in parallel of this notification.","mscs":["France"],"unexpectedEvents":[],"justification":"CTIS bug"}]},{},{"ctNumber":"2022-500703-49-01","ctStatus":4,"ctTitle":"Phase 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.","shortTitle":"AVANCE1-1/2a","startDateEU":"05/11/2025","conditions":"Duchenne muscular dystrophy","trialCountries":["France:4"],"decisionDateOverall":"21/11/2022","decisionDate":"FR: 21/11/2022","therapeuticAreas":["Diseases [C] - Musculoskeletal Diseases [C05]"],"sponsor":"Sqy Therapeutics","sponsorType":"Pharmaceutical company","trialPhase":"Phase I and Phase II (Integrated)- First administration to humans","endPoint":"Phase 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. -\tTwitch 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: \t- 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.","product":"","ageRangeSecondary":["5","9","6"],"ageGroup":"18-64 years, 0-17 years","gender":"Male","trialRegion":1,"totalNumberEnrolled":"12","primaryEndPoint":"Type, 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.","resultsFirstReceived":"No","lastUpdated":"27/02/2026","lastPublicationUpdate":"28/02/2026"},{"ctNumber":"2022-500703-49-01","ctStatus":"Authorised","startDateEU":"2025-11-05","decisionDate":"2022-11-21T09:35:18.445","publishDate":"2026-02-28T02:36:45.762945083","ctPublicStatusCode":4,"authorizedApplication":{"authorizedPartI":{"id":115954,"rowSubjectCount":0,"products":[{"id":481019,"part1MpRoleTypeCode":"1","productDictionaryInfo":{"productPk":"9661263","productPharmForm":"SOLUTION FOR INJECTION","euMpNumber":"PRD9661263","prodAuthStatus":1,"pharmForm":"SOLUTION FOR INJECTION","sponsorProductCode":"SQY51","activeSubstanceName":"SQY51","euSubstNumber":"SUB235142","nameOrg":"SQY THERAPEUTICS","productSubstances":[{"productPk":"9661263","substancePk":"249917","nameOrg":"SQY THERAPEUTICS","substanceOrigin":"Nucleic Acid","actSubstOrigin":"Nucleic Acid","actSubstName":"SQY51","substanceEvCode":"SUB235142"}],"activeSubstanceOtherDescriptiveName":""},"isPaediatricFormulation":false,"mpRoleInTrial":"1","orphanDrugEdit":false,"otherMedicinalProduct":"palmitoyl-conjugated tricyclo-DNA antisense oligonucleotide","evCode":"PRD9661263","sponsorProductCodeEdit":"SQY51","devices":[],"characteristics":["11"],"routes":["INTRAVENOUS USE"],"allSubstancesChemicals":false,"jsonActiveSubstanceNames":"sqy51","pharmaceuticalFormDisplay":"SOLUTION FOR INJECTION"}],"trialDetails":{"clinicalTrialIdentifiers":{"fullTitle":"Phase 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.","fullTitleTranslations":[{"id":9490102,"uuid":"12bb4115-18de-487c-81d0-eb66c85c2f4e","attributeTranslation":"Étude 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 :\n 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.","language":10,"languageDescription":"French"}],"publicTitle":"Phase 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 Dystrophy","publicTitleTranslations":[{"id":9490103,"uuid":"dc235a7e-faf7-42b5-8495-c46377380ce7","attributeTranslation":"Étude 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 Duchenne","language":10,"languageDescription":"French"}],"shortTitle":"AVANCE1-1/2a","secondaryIdentifyingNumbers":{"additionalRegistries":[]}},"trialInformation":{"trialCategory":{"isLowIntervention":false,"trialPhase":"7","trialCategory":"1","justificationForTrialCategory":"First in human study","trialCategoryId":106152},"medicalCondition":{"partIMedicalConditions":[{"id":120476,"medicalCondition":"Duchenne muscular dystrophy","medicalConditionTranslations":[],"isConditionRareDisease":true}],"meddraConditionTerms":[{"termId":100000012544,"version":"20.0","level":"PT","termName":"Duchenne muscular dystrophy","classificationCode":"10013801","organClass":100000004850,"active":false}]},"trialObjective":{"trialScopes":[{"code":"6","trialScopeId":350951},{"code":"4","trialScopeId":350952},{"code":"7","trialScopeId":350950}],"mainObjective":"Phase 1: Evaluate the safety and tolerability of SQY51 in DMD (Duchenne Muscular Dystrophy) patients.\nPhase 2a: Evaluate the safety and tolerance of SQY51 in DMD patients following 49-weeks multiple dosing.\nPART 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.","mainObjectiveTranslations":[],"secondaryObjectives":[{"id":406996,"number":1,"secondaryObjective":"For phase 1 study: Characterize the PK profile of SQY51 and its main metabolites in serum and urine","secondaryObjectiveTranslations":[]},{"id":406997,"number":2,"secondaryObjective":"For phase 1 study: Determine the effect of SQY51 on specific serum biomarkers","secondaryObjectiveTranslations":[]},{"id":406998,"number":3,"secondaryObjective":"For 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 II","secondaryObjectiveTranslations":[]},{"id":406999,"number":4,"secondaryObjective":"For phase 2a study: Determine the potential benefit of SQY51 on motor, respiratory and cardiac functions compared to baseline status as well as historical cohorts","secondaryObjectiveTranslations":[]},{"id":407000,"number":5,"secondaryObjective":"For 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 status","secondaryObjectiveTranslations":[]},{"id":407001,"number":6,"secondaryObjective":"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.","secondaryObjectiveTranslations":[]}]},"eligibilityCriteria":{"principalInclusionCriteria":[{"id":778534,"number":1,"principalInclusionCriteria":"Boys ≥6 years of age and ≥16 kg body weight.","principalInclusionCriteriaTranslations":[]},{"id":778535,"number":10,"principalInclusionCriteria":"Concomitant 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.","principalInclusionCriteriaTranslations":[]},{"id":778536,"number":11,"principalInclusionCriteria":"If 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.","principalInclusionCriteriaTranslations":[]},{"id":778537,"number":12,"principalInclusionCriteria":"Non-invasive mechanical nocturnal ventilation is permissive if <16 h/day.","principalInclusionCriteriaTranslations":[]},{"id":778538,"number":13,"principalInclusionCriteria":"For the extension phase : Patients must have completed Phase 2a of the study.","principalInclusionCriteriaTranslations":[]},{"id":778539,"number":2,"principalInclusionCriteria":"Being affiliated with a Social Security.","principalInclusionCriteriaTranslations":[]},{"id":778540,"number":3,"principalInclusionCriteria":"Informed consent form signed by the patient or, if minor, by the legal guardian(s).","principalInclusionCriteriaTranslations":[]},{"id":778541,"number":4,"principalInclusionCriteria":"For phase 2a study: Must have completed Phase 1 of the study.","principalInclusionCriteriaTranslations":[]},{"id":778542,"number":5,"principalInclusionCriteria":"Ambulatory 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). 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MODIFICATION","isRMS":true}]},{"id":65534,"type":"SUBSTANTIAL MODIFICATION","status":"Authorised","ctNumber":"2022-500703-49-01","trialStatus":"Authorised","submissionDate":"2025-09-19","partI":{"assessmentOutcome":"acceptable"},"partIIInfo":[{"id":242679,"mscId":1902,"mscInfo":{"id":1902,"mscName":"France","countryOrganisationId":2012,"assessmentOutcome":"acceptable","assessmentOutcomeDate":"2025-10-21","decision":"authorized","decisionDate":"2022-11-21","reportingStatusCode":"Authorised","countryName":"France","trialStatus":"Authorised","firstDecisionDate":"2022-11-21"},"applicationStatusCode":"Authorised"}],"decisionDate":"2025-10-22","ctMSCsByApplication":[{"id":1902,"mscName":"France","reportingStatusCode":"Authorised"}],"businessKey":"SM-7","modScope":"PART_II","allPartTwosOutOfScope":false,"applicationTrialDecisionByMsc":{},"decisions":[{"id":94870,"applicationId":65534,"mscId":1902,"mscName":"France","decisionDate":"2025-10-22T15:52:07.319","decision":"authorized","assessmentOutcome":"acceptable","eventType":"decision","part2Id":242679,"part1Id":101037,"applicationType":"SUBSTANTIAL MODIFICATION","isRMS":true}]},{"id":75537,"type":"SUBSTANTIAL MODIFICATION","status":"Authorised","ctNumber":"2022-500703-49-01","trialStatus":"Authorised","submissionDate":"2026-01-28","partI":{"assessmentOutcome":"acceptable"},"partIIInfo":[{"id":275836,"mscId":1902,"mscInfo":{"id":1902,"mscName":"France","countryOrganisationId":2012,"assessmentOutcome":"acceptable","assessmentOutcomeDate":"2026-02-27","decision":"authorized","decisionDate":"2022-11-21","reportingStatusCode":"Authorised","countryName":"France","trialStatus":"Authorised","firstDecisionDate":"2022-11-21"},"applicationStatusCode":"Authorised"}],"decisionDate":"2026-02-27","ctMSCsByApplication":[{"id":1902,"mscName":"France","reportingStatusCode":"Authorised"}],"businessKey":"SM-8","modScope":"PART_II","allPartTwosOutOfScope":false,"applicationTrialDecisionByMsc":{},"decisions":[{"id":112000,"applicationId":75537,"mscId":1902,"mscName":"France","decisionDate":"2026-02-27T15:06:30.845","decision":"authorized","assessmentOutcome":"acceptable","eventType":"decision","part2Id":275836,"part1Id":115954,"applicationType":"SUBSTANTIAL MODIFICATION","isRMS":true}]}],"trialGlobalEnd":[],"memberStatesConcerned":[{"mscName":"France","mscId":1902,"firstDecisionDate":"2022-11-21T09:35:18.445","lastDecisionDate":"2026-02-27T15:06:30.845","mscPublicStatusCode":4}],"eudraCt":{"isTransitioned":false}},"events":{"temporaryHaltList":[{"mscId":1902,"businessKey":"TH-17341","haltDate":"2024-03-15","reasonList":[{"code":"6","name":"Medicinal Product related","isSmRequiredForRestart":false,"isCommentRequired":false}],"isTreatmentStopped":true,"sponsorJustificationComment":"Following 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).\n\nBelow is the information recorded in the batch certificates - investigational medicinal product for human use issued by COLCA /EUROMED PHARMA:\n-\tLot DPSQYM001A expiry date 06/2023, batch certificate signed by QP on 20/12/2022\n-\tLot DPSQYM001B expiry date 05/2024, batch certificate signed by QP on 14/09/2023\n-\tLot DPSQYM001C expiry date 05/2024, batch certificate signed by QP on 14/09/2023\n\nSQY51 Finished Product Information:\n-\tAbsence of a technical batch for SQY51 performed prior to the start of the clinical study\n-\t1st 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\n-\tStabilization of the DPSQYM001 batch at AXOLABS in July 2022 according to the following scheme (Q0 &#34;analytical&#34; &#61; 27/07/2022):\n         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 \n         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 \n         o Stress test / Storage in stability chamber at &#43;40°C &#43;/- 2°C / T0 &#43; T1M \n\n-\tStability 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.\n-\tExtrapolation 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)\n\nIn 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):\n-\tExpiry date 11/2022, on the date of signature of the batch certificate on 20/12/2022\n-\tExpiry Date 07/2023, on the date of signature of the batch certificate on 14/09/2023\n\nPlease 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.\n\nFollowing 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.\nThe last administration of this batch took place at the investigator site on 11/03/2024 as part of this AVANCE 1 - phase 1 study.\nThis Phase 1 study has not been yet  finalized. Upcoming visits are described in Appendix 1 of the attached letter.\n\nCurrently, 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.\n\nPart of this batch has been sent to the investigator site, but no administration will take place without the authorization of the ANSM.\n\nSQY 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.","isBenefitRiskBalanceChange":false,"submitDate":"2024-03-15T17:58:24","subjectFuMeasuresComment":"This same notification will be the subject of an email communication.\n\nThe actions SQY Therapeutics intends to implement are as follows:\n-\tSuspension 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.\n-\tContinuation of analyses, recruitment and follow-up visits of the AVANCE 1 – Phase 1 clinical study, as mentioned in Appendix 1.\n-\tEmergency analysis of the remaining vials of the batch DPSQYM001\n-\tCommunication with patients after approval by the ANSM\n-\tCommunication with the DSMB/steering committee after approval by the ANSM\n-\tCommunication with the hospital of the investigator site after approval by the ANSM","isPublished":false,"updatedOn":"2024-03-15","mscList":[{"mscId":1902,"mscCountryName":"France","mscCountryCode":"FR"}]},{"mscId":1902,"businessKey":"TH-76448","haltDate":"2025-03-14","reasonList":[{"code":"4","name":"Safety related (clinical or pre-clinical results)","isSmRequiredForRestart":false,"isCommentRequired":false}],"isTreatmentStopped":true,"sponsorJustificationComment":"The 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).\nThis 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.\nPlease refer to the attached Cover letter for a detailed overview.\nAttached are CIOMS forms for patients 01-015 and 01-008 and 01-002.\nData collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.","isBenefitRiskBalanceChange":false,"submitDate":"2025-03-26T09:37:33","subjectFuMeasuresComment":"A DSMB meeting was held on March 21st to discuss these events and potential measures, meeting minutes are ongoing.\nA 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. \nEncouragingly, 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.\nAn urgent meeting with the Agency is requested to discuss this data, protocol adjustments, and the continuation of treatment for favorable responders.\nFinally, an extension is requested for the substantial protocol amendment submission deadline (currently April 14thfollowing DSUR RFIs).\n A USM is submitted in parallel of this notification","isPublished":false,"mscList":[{"mscId":1902,"mscCountryName":"France","mscCountryCode":"FR"}]}],"trialEvents":[{"mscId":1902,"mscName":"France","events":[{"notificationType":"RESTART_OF_TRIAL","date":"2025-11-05"},{"notificationType":"START_OF_TRIAL","date":"2023-01-05"},{"notificationType":"RESTART_OF_RECRUITMENT","date":"2024-09-09"},{"notificationType":"START_OF_RECRUITMENT","date":"2023-04-26"},{"notificationType":"TEMPORARY_HALT","date":"2025-03-14"}]}],"unexpectedEvents":[],"seriousBreaches":[],"urgentSafetyMeasures":[{"sponsorBusinessKey":"USM #1","businessKey":"US-51783","eventDate":"2024-10-04","submissionDate":"2024-10-15","updatedOn":"2024-10-15","inResponseTo":"OTHER","susarIdList":[],"eventDescription":"As 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.\n\nOn 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.\nOn 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.","measuresTakenDescription":"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: \n\n- Cohort 10 mg/kg : 6 participants (including 4 of the heaviest) ; \n- Cohort 16 mg/kg: 3 participants; \n- Cohort 25 mg/kg: 3 participants. \n\nDSMB members approved this reassignment. This urgent safety measure will be endorsed by a substantial modification for authorization.","mscs":["France"],"unexpectedEvents":[]},{"sponsorBusinessKey":"USM 2","businessKey":"US-76442","eventDate":"2025-03-18","submissionDate":"2025-03-26","updatedOn":"2025-03-26","inResponseTo":"OTHER","susarIdList":[],"eventDescription":"The 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).\nThis 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.\nPlease refer to the attached Cover letter for a detailed overview.\nAttached 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).\nData collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.","measuresTakenDescription":"Data collection will continue despite the injection pause, and advancing biopsies for near-completion patients is proposed.\nA DSMB meeting was held on March 21st to discuss these events and potential measures, meeting minutes are ongoing.\n 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. \n 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.\n An urgent meeting with the Agency is requested to discuss this data, protocol adjustments, and the continuation of treatment for favorable responders.\nFinally, an extension is requested for the substantial protocol amendment submission deadline (currently April 14thfollowing DSUR RFIs).\n A temporary halt is submitted in parallel of this notification.","mscs":["France"],"unexpectedEvents":[],"justification":"CTIS bug"}]},"results":{},"documents":[],"trialRegion":"EEA","trialRegionCode":1,"correctiveMeasures":[]}]
]}