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"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-pati\u00ebnten", "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 comparaci\u00f3n de dosis y evaluaci\u00f3n exploratoria de la eficacia de SAT-3247 administrado por v\u00eda 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": "Bezpiecze\u0144stwo: Pierwszorz\u0119dowym celem w zakresie bezpiecze\u0144stwa jest ocena bezpiecze\u0144stwa i tolerancji preparatu SAT-3247 u ambulatoryjnych pacjent\u00f3w z DMD\nSkuteczno\u015b\u0107: Pierwszorz\u0119dowym celem skuteczno\u015bci jest ocena wp\u0142ywu preparatu SAT-3247 na si\u0142\u0119 mi\u0119\u015bni mierzon\u0105 za pomoc\u0105 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 dinamometr\u00eda al cabo de 12 semanas", "language": 7, "languageDescription": "Spanish"}, {"id": 9737394, "uuid": "4722e808-4398-47d7-bdeb-a16764253ec7", "attributeTranslation": "Innocuit\u00e9: L\u2019objectif principal de s\u00e9curit\u00e9 est d\u2019\u00e9valuer l\u2019innocuit\u00e9 et la tol\u00e9rabilit\u00e9 du SAT-3247 chez les patients \nambulants atteints de DMD\nEfficacit\u00e9:  L\u2019objectif principal d\u2019efficacit\u00e9 est de d\u00e9terminer les effets du SAT-3247 sur la force musculaire d\u00e9termin\u00e9e par dynamom\u00e9trie \u00e0 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-pati\u00ebnten\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 prim\u00e4re Sicherheitsziel ist die Bewertung der Sicherheit und Vertr\u00e4glichkeit von SAT-3247 bei \ngehf\u00e4higen DMD-Patienten\nWirksamkeit: Das prim\u00e4re 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 intramuskul\u00e4ren 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": "D\u00e9terminer les effets du SAT-3247 sur la fraction de graisse intramusculaire dans la r\u00e9sonance magn\u00e9tique quantitative (IRMq) du muscle vaste lat\u00e9ral \u00e0 12 semaines.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737408, "uuid": "b687125d-e5f9-4b68-9b1a-f9d1c10d0e0a", "attributeTranslation": "Determinar los efectos de SAT-3247 sobre la fracci\u00f3n de grasa intramuscular mediante resonancia magn\u00e9tica 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": "Okre\u015blenie wp\u0142ywu SAT-3247 na frakcj\u0119 t\u0142uszczu \u015br\u00f3dmi\u0119\u015bniowego w ilo\u015bciowym rezonansie \nmagnetycznym (qMR) mi\u0119\u015bnia 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": "D\u00e9terminer les effets du SAT-3247 sur le temps de relaxation transversale du muscle protonique (T2) dans le muscle vaste lat\u00e9ral \u00e0 12 semaines.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737413, "uuid": "3af14c0c-bb41-4a2a-8a06-4b94a35e3a7c", "attributeTranslation": "Okre\u015blenie wp\u0142ywu SAT-3247 na czas relaksacji poprzecznej proton\u00f3w (T2) w mi\u0119\u015bniu 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 relajaci\u00f3n transversal de los protones (T2) en el m\u00fasculo 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": "Okre\u015blenie wp\u0142ywu SAT-3247 na wska\u017anik regeneracji w otwartej biopsji mi\u0119\u015bnia dwug\u0142owego 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": "D\u00e9terminer les effets du SAT-3247 sur l\u2019indice de r\u00e9g\u00e9n\u00e9ration dans une biopsie musculaire ouverte du biceps brachii \u00e0 12 semaines.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737418, "uuid": "b5508636-ac46-4f0a-b307-50b3b88b5492", "attributeTranslation": "Determinar los efectos de SAT-3247 sobre el \u00edndice de regeneraci\u00f3n en biopsia muscular abierta del b\u00edceps 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": "D\u00e9terminer la pharmacocin\u00e9tique de population du SAT-3247 chez les patients ambulants.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737419, "uuid": "88926545-43ed-4cae-b8f0-8532ab772ea8", "attributeTranslation": "Okre\u015blenie farmakokinetyki (PK) SAT-3247 w populacji pacjent\u00f3w ambulatoryjnych.", "language": 19, "languageDescription": "Polish"}, {"id": 9737421, "uuid": "88926545-43ed-4cae-b8f0-8532ab772ea8", "attributeTranslation": "Het bepalen van de populatie-farmacokinetiek van SAT-3247 bij ambulante pati\u00ebnten.", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9737422, "uuid": "88926545-43ed-4cae-b8f0-8532ab772ea8", "attributeTranslation": "Determinar la farmacocin\u00e9tica (PK) poblacional de SAT-3247 en pacientes ambulatorios.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737420, "uuid": "88926545-43ed-4cae-b8f0-8532ab772ea8", "attributeTranslation": "Bestimmung der Populations-PK von SAT-3247 bei gehf\u00e4higen Patienten.", "language": 28, "languageDescription": "German (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 f\u00fcr eine Verbesserung der Muskelfunktion durch die Behandlung mit SAT-3247 bei gehf\u00e4higen Patienten.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737428, "uuid": "2c5989a3-b91b-4a91-a969-b337e9b6b839", "attributeTranslation": "D\u00e9terminer le potentiel d\u2019am\u00e9lioration 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": "Okre\u015blenie potencja\u0142u poprawy funkcji mi\u0119\u015bni w trakcie leczenia SAT-3247 u pacjent\u00f3w ambulatoryjnych.", "language": 19, "languageDescription": "Polish"}, {"id": 9737426, "uuid": "2c5989a3-b91b-4a91-a969-b337e9b6b839", "attributeTranslation": "Determinar el potencial de mejora de la funci\u00f3n 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 pati\u00ebnten.", "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: Okre\u015blenie wp\u0142ywu SAT-3247 na czynno\u015b\u0107 uk\u0142adu 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 funci\u00f3n respiratoria al cabo de 12 semanas.", "language": 7, "languageDescription": "Spanish"}, {"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: D\u00e9terminer les effets du SAT-3247 sur la fonction respiratoire \u00e0 12 semaines.", "language": 33, "languageDescription": "French (Belgium)"}, {"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": 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": 9737434, "uuid": "f856ccc4-8573-45d9-b531-c90a0accdc99", "attributeTranslation": "Objetivo exploratorio: Determinar los efectos de SAT-3247 sobre la histopatolog\u00eda muscular del b\u00edceps braquial al cabo de 12 semanas.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737438, "uuid": "f856ccc4-8573-45d9-b531-c90a0accdc99", "attributeTranslation": "Objectif exploratoire: D\u00e9terminer les effets du SAT-3247 sur l\u2019histopathologie musculaire du biceps brachial \u00e0 12 semaines.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737436, "uuid": "f856ccc4-8573-45d9-b531-c90a0accdc99", "attributeTranslation": "Cel eksploracyjny: Okre\u015blenie wp\u0142ywu SAT-3247 na obraz histopatologiczny mi\u0119\u015bnia dwug\u0142owego ramienia po 12 tygodniach.", "language": 19, "languageDescription": "Polish"}]}, {"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\u2019\u00e9volution des r\u00e9sultats \u00e0 l\u2019histoire naturelle.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737439, "uuid": "17c05869-6ce9-4a51-a96e-d4f4e5a12943", "attributeTranslation": "Cel eksploracyjny: Por\u00f3wnanie 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": 9737441, "uuid": "17c05869-6ce9-4a51-a96e-d4f4e5a12943", "attributeTranslation": "Exploratives Ziel: Vergleich der Ver\u00e4nderungen bei den Ergebnissen mit dem nat\u00fcrlichen Verlauf.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737442, "uuid": "17c05869-6ce9-4a51-a96e-d4f4e5a12943", "attributeTranslation": "Objetivo exploratorio: Comparar los cambios en los resultados con la evoluci\u00f3n natural de la enfermedad.", "language": 7, "languageDescription": "Spanish"}]}]}, "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 d\u00e9finitif de DMD sur la base de r\u00e9sultats cliniques document\u00e9s et d\u2019un test g\u00e9n\u00e9tique pr\u00e9alable confirmant la pr\u00e9sence d\u2019une mutation dans le g\u00e8ne 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 best\u00e4tigter Mutation im DMD-Gen.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737332, "uuid": "b26cc646-e636-4673-b194-6425c4081f63", "attributeTranslation": "1. El paciente tiene un diagn\u00f3stico definitivo de DMD respaldado por resultados cl\u00ednicos documentados y pruebas gen\u00e9ticas previas con una mutaci\u00f3n confirmada en el gen DMD.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737329, "uuid": "b26cc646-e636-4673-b194-6425c4081f63", "attributeTranslation": "1. Potwierdzona diagnoza dystrofii mi\u0119\u015bniowej Duchenne\u2019a (DMD) na podstawie udokumentowanych wynik\u00f3w bada\u0144 klinicznych oraz wcze\u015bniejszych bada\u0144 genetycznych, z wykryt\u0105 mutacj\u0105 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 est\u00e1 tomando (o ha tomado en las 4 semanas anteriores a la inscripci\u00f3n) remedios y complementos a base de hierbas que puedan afectar a la fuerza y a la funci\u00f3n 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 pr\u00e9c\u00e9dant l\u2019inscription) des rem\u00e8des \u00e0 base de plantes et des suppl\u00e9ments qui peuvent avoir un impact sur la force et la fonction musculaires (par exemple, coenzyme Q10, cr\u00e9atine, etc.), ceux-ci doivent \u00eatre maintenus \u00e0 un r\u00e9gime stable pendant toute la dur\u00e9e de l\u2019\u00e9tude.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737337, "uuid": "192a96ba-455c-43cf-8066-0a553295a22d", "attributeTranslation": "10. Wenn der Teilnehmer pflanzliche Heilmittel und Nahrungserg\u00e4nzungsmittel einnimmt (oder in den 4 Wochen \nvor der Aufnahme eingenommen hat), die sich auf die Muskelkraft und -funktion auswirken k\u00f6nnen (z. B. Co-Enzym Q10, Kreatin usw.), muss f\u00fcr diese f\u00fcr 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 be\u00efnvloeden (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. Je\u015bli uczestnik przyjmuje (lub przyjmowa\u0142 w ci\u0105gu 4 tygodni przed w\u0142\u0105czeniem) preparaty zio\u0142owe i suplementy mog\u0105ce wp\u0142ywa\u0107 na si\u0142\u0119 i funkcjonowanie mi\u0119\u015bni (np. koenzym Q10, kreatyna itp.), ich stosowanie musi by\u0107 kontynuowane w stabilnym schemacie przez ca\u0142y 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 \u2265 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 re\u00e7u auparavant du delandistrogene moxeparvovec (nom de marque Elevidys) dans le cadre d\u2019une \u00e9tude clinique ant\u00e9rieure ou dans un contexte commercial > 18 mois avant la s\u00e9lection et dont les tests de la fonction musculaire se sont stabilis\u00e9s ou ont montr\u00e9 un d\u00e9clin \u2265 3 mois avant la s\u00e9lection, tel que d\u00e9termin\u00e9 par l\u2019investigateur et document\u00e9 dans les notes \ndu dossier, seront \u00e9ligibles. \na. L\u2019inscription de participants d\u00e9j\u00e0 trait\u00e9s par th\u00e9rapie g\u00e9nique, qu\u2019il s\u2019agisse du delandistrogene moxeparvovec ou d\u2019un autre produit de th\u00e9rapie g\u00e9nique exp\u00e9rimental, sera plafonn\u00e9 \u00e0 25 % et stratifi\u00e9 entre les cohortes.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737340, "uuid": "478287ff-5cf7-485d-a2f2-f3900ee636aa", "attributeTranslation": "11. Uczestnicy, kt\u00f3rzy wcze\u015bniej otrzymali delandistrogen moxeparvovec (nazwa handlowa: Elevidys) w ramach wcze\u015bniejszego badania klinicznego lub w warunkach komercyjnych, >18 miesi\u0119cy przed wizyt\u0105 przesiewow\u0105, u kt\u00f3rych testy czynno\u015bci mi\u0119\u015bniowej ustabilizowa\u0142y si\u0119 lub wykaza\u0142y pogorszenie \u2265 3 miesi\u0105ce przed wizyt\u0105 przesiewow\u0105 \u2014 zgodnie z ocen\u0105 badacza i udokumentowane w dokumentacji medycznej \u2014 b\u0119d\u0105 kwalifikowa\u0107 si\u0119 do udzia\u0142u w badaniu. \na. Liczba uczestnik\u00f3w wcze\u015bniej leczonych terapi\u0105 genow\u0105 \u2013 zar\u00f3wno delandistrogenem moxeparvovec, jak i innym badanym produktem terapii genowej \u2013 zostanie ograniczona do 25% i zr\u00f3wnowa\u017cona pomi\u0119dzy kohortami.", "language": 19, "languageDescription": "Polish"}, {"id": 9737342, "uuid": "478287ff-5cf7-485d-a2f2-f3900ee636aa", "attributeTranslation": "11. Ser\u00e1n aptos aquellos participantes que hayan recibido previamente moxeparvovec delandistrog\u00e9nico (nombre \ncomercial Elevidys), ya sea en un estudio previo o en el \u00e1mbito comercial > 18 meses antes de la selecci\u00f3n, cuyas pruebas de funci\u00f3n muscular se hayan estabilizado o hayan presentado un deterioro \u2265 3 meses antes de la selecci\u00f3n, seg\u00fan las valoraciones del investigador y lo documentado en la historia cl\u00ednica. \na. Se limitar\u00e1 al 25 % la inscripci\u00f3n de participantes previamente tratados con terapia g\u00e9nica, ya sea \ndelandistrogene moxeparvovec u otro medicamento para terapia g\u00e9nica en investigaci\u00f3n, y se estratificar\u00e1 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 \u2265 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 fr\u00fcheren \nklinischen Studie oder im kommerziellen Rahmen > 18 Monate vor dem Screening erhalten haben und deren Muskelfunktionstests sich stabilisiert haben oder \u2265 3 Monate vor dem Screening eine Verschlechterung aufweisen, wie vom Pr\u00fcfarzt 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 \u2265 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 \u2265 3 Monate vor dem Screening verschlechtert haben, wie vom Pr\u00fcfarzt festgestellt und in den Aufzeichnungen dokumentiert, sind teilnahmeberechtigt.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737345, "uuid": "1abd7c23-3870-467c-8234-cce45c63b32e", "attributeTranslation": "12. Uczestnicy, kt\u00f3rzy wcze\u015bniej otrzymali lek z grupy exon skipper > 6 miesi\u0119cy przed wizyt\u0105 przesiewow\u0105, u kt\u00f3rych testy funkcji mi\u0119\u015bniowej ustabilizowa\u0142y si\u0119 lub wykaza\u0142y pogorszenie \u2265 3 miesi\u0105ce przed wizyt\u0105 przesiewow\u0105 \u2013 zgodnie z ocen\u0105 badacza i potwierdzone w dokumentacji medycznej \u2013 b\u0119d\u0105 kwalifikowa\u0107 si\u0119 do udzia\u0142u w badaniu.", "language": 19, "languageDescription": "Polish"}, {"id": 9737347, "uuid": "1abd7c23-3870-467c-8234-cce45c63b32e", "attributeTranslation": "12. Les participants trait\u00e9s ant\u00e9rieurement par saut d\u2019exon > 6 mois avant la s\u00e9lection et dont les tests de la fonction musculaire se sont stabilis\u00e9s ou ont montr\u00e9 un d\u00e9clin \u2265 3 mois avant la s\u00e9lection, comme d\u00e9termin\u00e9 par l\u2019investigateur et document\u00e9 dans les notes du dossier, seront \u00e9ligibles.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737346, "uuid": "1abd7c23-3870-467c-8234-cce45c63b32e", "attributeTranslation": "12. Ser\u00e1n aptos aquellos participantes que hayan recibido previamente una terapia de salto de ex\u00f3n exonskipping) > 6 meses antes de la selecci\u00f3n cuyas pruebas de funci\u00f3n muscular se hayan estabilizado o hayan presentado un deterioro \u2265 3 meses antes de la selecci\u00f3n, seg\u00fan las valoraciones del investigador y lo documentado en la historia cl\u00ednica.", "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 \u2265 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 \u2265 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 \u2265 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 \u00e0 un programme de kin\u00e9sith\u00e9rapie ou de musculation, celui-ci doit \u00eatre stable pendant \u2265 2 mois avant la visite de s\u00e9lection et pendant toute la dur\u00e9e de l\u2019\u00e9tude.", "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 \u2265 2 meses antes de la visita de selecci\u00f3n 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 \u2265 2 Monate vor der Screening-Visite \nund w\u00e4hrend 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 si\u0142owego, musi on by\u0107 prowadzony w spos\u00f3b stabilny przez co najmniej 2 miesi\u0105ce przed wizyt\u0105 przesiewow\u0105 i kontynuowany w niezmienionej formie przez ca\u0142y czas trwania badania.", "language": 19, "languageDescription": "Polish"}]}, {"id": 797258, "number": 2, "principalInclusionCriteria": "Male DMD patients who are ambulatory and aged \u2265 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 \u2265 7 y < 10 a\u00f1os en el momento de la selecci\u00f3n.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737355, "uuid": "06b12ff3-2292-497d-ba24-e21c6744abd2", "attributeTranslation": "2. M\u00e4nnliche DMD-Patienten, die gehf\u00e4hig und zum Zeitpunkt des Screenings \u2265 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 \u00e2g\u00e9s de \u2265 7 \u00e0 < 10 ans au moment de la s\u00e9lection.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737357, "uuid": "06b12ff3-2292-497d-ba24-e21c6744abd2", "attributeTranslation": "2. M\u0119\u017cczy\u017ani z DMD, ambulatoryjni, w wieku od \u2265 7 do < 10 lat w momencie badania przesiewowego.", "language": 19, "languageDescription": "Polish"}, {"id": 9737358, "uuid": "06b12ff3-2292-497d-ba24-e21c6744abd2", "attributeTranslation": "2. DMD-pati\u00ebnt van het mannelijk geslacht, ambulant en \u2265 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 (\u22641 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 (\u2264 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 \u22641 segundo).", "language": 7, "languageDescription": "Spanish"}, {"id": 9737361, "uuid": "40f3f802-1263-4fc4-9db5-a5076858ecea", "attributeTranslation": "3. Uko\u0144czenie dw\u00f3ch pr\u00f3b wspinania si\u0119 po czterech stopniach podczas wizyty 1 ze \u015brednim czasem \u2264 8 sekund (przy maksymalnej r\u00f3\u017cnicy \u2264 1 sekundy mi\u0119dzy pr\u00f3bami).", "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 \u2264 1 s).", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9737359, "uuid": "40f3f802-1263-4fc4-9db5-a5076858ecea", "attributeTranslation": "3. Avoir effectu\u00e9 deux mont\u00e9es chronom\u00e9tr\u00e9es sur quatre marches lors de la visite 1 avec une moyenne de 8 secondes ou moins (variance \u22641 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 pod\u0142ogi wynosz\u0105cy co najmniej 3 sekundy, ale mniej ni\u017c 10 sekund.", "language": 19, "languageDescription": "Polish"}, {"id": 9737368, "uuid": "9f7a3d57-ab73-4f05-b91e-1cfecbba61ab", "attributeTranslation": "4. Avoir un temps de relev\u00e9 d\u2019au moins 3 secondes mais inf\u00e9rieur \u00e0 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 odchyle\u0144 w badaniu fizykalnym, wynikach laboratoryjnych i monitorowaniu kardiologicznym.", "language": 19, "languageDescription": "Polish"}, {"id": 9737371, "uuid": "cf85ab00-12c5-4be9-82c2-fb82a5475de2", "attributeTranslation": "5. El paciente est\u00e1 en buen estado de salud, seg\u00fan las valoraciones del investigador, incluyendo los antecedentes \nm\u00e9dicos y psiqui\u00e1tricos, y sin hallazgos cl\u00ednicamente relevantes en la exploraci\u00f3n f\u00edsica, las pruebas de laboratorio y el seguimiento de la funci\u00f3n cardiaca.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737373, "uuid": "cf85ab00-12c5-4be9-82c2-fb82a5475de2", "attributeTranslation": "5. Gesund, wie vom Pr\u00fcfarzt unter Ber\u00fccksichtigung von Anamnese und psychiatrischer Anamnese festgestellt, und keine klinisch signifikanten Befunde bei k\u00f6rperlicher Untersuchung, Labortests und Herz\u00fcberwachung.", "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. \u00catre en bonne sant\u00e9, de l\u2019avis de l\u2019investigateur qui examinera \u00e9galement les ant\u00e9c\u00e9dents m\u00e9dicaux, les ant\u00e9c\u00e9dents psychiatriques et l\u2019absence de r\u00e9sultats cliniquement significatifs \u00e0 l\u2019examen clinique, aux tests de laboratoire et \u00e0 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 capacit\u00e9 du participant et du soignant \u00e0 communiquer de mani\u00e8re satisfaisante avec l\u2019investigateur, \u00e0 participer \u00e0 l\u2019ensemble de l\u2019\u00e9tude et \u00e0 en respecter les exigences, y compris les visites programm\u00e9es, les proc\u00e9dures / \u00e9valuations, les questionnaires, les tests de laboratoire et les restrictions de l\u2019\u00e9tude.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737376, "uuid": "97d351f2-ebb6-4e7f-9463-83a053424d04", "attributeTranslation": "6. Zdolno\u015b\u0107 uczestnika i jego opiekuna do satysfakcjonuj\u0105cej komunikacji z badaczem oraz do uczestniczenia w ca\u0142ym badaniu zgodnie z jego wymaganiami, w tym w zaplanowanych wizytach, procedurach/ocenach, wype\u0142nianiu kwestionariuszy, badaniach laboratoryjnych i przestrzeganiu ogranicze\u0144 badania.", "language": 19, "languageDescription": "Polish"}, {"id": 9737374, "uuid": "97d351f2-ebb6-4e7f-9463-83a053424d04", "attributeTranslation": "6. F\u00e4higkeit des Teilnehmers und der Betreuungsperson, mit dem Pr\u00fcfarzt zufriedenstellend zu kommunizieren und an der gesamten Studie teilzunehmen und deren Anforderungen zu erf\u00fcllen, einschlie\u00dflich geplanter Visiten, Verfahren/Bewertungen, Frageb\u00f6gen, Labortests und Studieneinschr\u00e4nkungen.", "language": 28, "languageDescription": "German (Belgium)"}]}, {"id": 797263, "number": 7, "principalInclusionCriteria": "Participant\u2019s 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 informaci\u00f3n y el formulario de consentimiento, y despu\u00e9s de haber tenido la oportunidad de hablar sobre el estudio con el investigador o su representante; se pedir\u00e1 a los participantes que den su consentimiento por escrito o verbal conforme a los requisitos de cada pa\u00eds.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737383, "uuid": "b278e74b-cf3f-45e8-a781-4cbc2f5d235c", "attributeTranslation": "7. Le consentement \u00e9crit \u00e0 la participation sign\u00e9 par le(s) parent(s) ou le(s) tuteur(s) l\u00e9gal / l\u00e9gaux du participant, apr\u00e8s lecture du document d\u2019information et consentement, et apr\u00e8s avoir eu la possibilit\u00e9 de discuter de l\u2019\u00e9tude avec l\u2019investigateur ou son d\u00e9l\u00e9gu\u00e9 ; il sera demand\u00e9 aux participants de donner leur consentement \u00e9crit ou verbal conform\u00e9ment aux exigences locales.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737381, "uuid": "b278e74b-cf3f-45e8-a781-4cbc2f5d235c", "attributeTranslation": "7. Pisemna zgoda na udzia\u0142 w badaniu udzielona przez rodzic\u00f3w lub opiekun\u00f3w prawnych po zapoznaniu si\u0119 z formularzem informacyjnym i zgod\u0105 oraz po mo\u017cliwo\u015bci przedyskutowania badania z badaczem lub jego przedstawicielem; uczestnicy zostan\u0105 poproszeni o wyra\u017cenie 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 Einverst\u00e4ndnis zur \nTeilnahme gegeben, nachdem sie die Informationen und die Einverst\u00e4ndniserkl\u00e4rung gelesen haben und die M\u00f6glichkeit hatten, die Studie mit dem Pr\u00fcfarzt oder seinem Beauftragten zu besprechen; die Teilnehmer werden entsprechend den \u00f6rtlichen Anforderungen um eine schriftliche oder m\u00fcndliche 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 \u2265 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 \u2265 3 months prior to the Screening Visit.", "principalInclusionCriteriaTranslations": [{"id": 9737387, "uuid": "fb6013c2-5e98-4a72-a3fd-992230871541", "attributeTranslation": "8. Een stabiele dagelijkse dosis systemische glucocortico\u00efden (prednisolon, deflazacort of vamorolon) volgens de zorgstandaard gedurende \u2265 3 maanden voorafgaand aan het Screeningbezoek en voor de duur van het onderzoek. \na. Pati\u00ebnten die geen glucocorticostero\u00efden krijgen, komen ook in aanmerking als ze \u2265 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 glucocortico\u00efdes syst\u00e9miques quotidiens (c\u2019est-\u00e0-dire prednisolone, deflazacort ou vamorolone) conform\u00e9ment \u00e0 la norme de soins pendant \u2265 3 mois avant la visite de s\u00e9lection et pendant toute la dur\u00e9e de l\u2019\u00e9tude. \na. Les patients qui ne re\u00e7oivent pas de glucocortico\u00efdes sont \u00e9galement \u00e9ligibles s\u2019ils ont arr\u00eat\u00e9 \u2265 3 mois avant la visite de s\u00e9lection", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737385, "uuid": "fb6013c2-5e98-4a72-a3fd-992230871541", "attributeTranslation": "8. Stabilna dawka og\u00f3lnoustrojowych glikokortykosteroid\u00f3w (np. prednizolon, deflazakort, wamorolon) zgodnie ze standardem leczenia przez co najmniej 3 miesi\u0105ce przed wizyt\u0105 przesiewow\u0105 i przez ca\u0142y okres trwania badania. \na. Pacjenci niestosuj\u0105cy glikokortykosteroid\u00f3w r\u00f3wnie\u017c mog\u0105 zosta\u0107 w\u0142\u0105czeni, pod warunkiem \u017ce leczenie zosta\u0142o zako\u0144czone co najmniej 3 miesi\u0105ce przed wizyt\u0105 przesiewow\u0105.", "language": 19, "languageDescription": "Polish"}, {"id": 9737384, "uuid": "fb6013c2-5e98-4a72-a3fd-992230871541", "attributeTranslation": "8. Dosis estable de glucocorticoides sist\u00e9micos diarios (es decir, prednisolona, deflazacort o vamorolona) seg\u00fan el tratamiento est\u00e1ndar durante \u2265 3 meses antes de la visita de selecci\u00f3n y durante todo el estudio. \na. Los pacientes que no est\u00e9n recibiendo glucocorticoides tambi\u00e9n son aptos si se suspenden \u2265 3 meses antes de la visita de selecci\u00f3n.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737388, "uuid": "fb6013c2-5e98-4a72-a3fd-992230871541", "attributeTranslation": "8. Stabile Dosis t\u00e4glicher systemischer Glukokortikoide (d. h. Prednisolon, Deflazacort oder Vamorolon) entsprechend dem Behandlungsstandard f\u00fcr \u2265 3 Monate vor der Screening-Visite und f\u00fcr die Dauer der Studie. \na. Patienten, die keine Glukokortikosteroide erhalten, sind ebenfalls teilnahmeberechtigt, wenn sie diese \u2265 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, \u03b2-blockers, and diuretics (excluding glucocorticosteroids) and over-the-counter medicines and/or herbal supplements for supportive care \u2265 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 diur\u00e9ticos (excepto glucocorticoides) y medicamentos de venta libre y/o complementos a base de hierbas para cuidados paliativos \u2265 1 mes antes de la visita de selecci\u00f3n y durante todo el estudio.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737390, "uuid": "d4389451-9556-4890-be22-b4b9df66f9d2", "attributeTranslation": "9. Stabilne dawki lek\u00f3w przepisywanych na recept\u0119, w tym inhibitor\u00f3w ACE, \u03b2-bloker\u00f3w i lek\u00f3w moczop\u0119dnych (z wy\u0142\u0105czeniem glikokortykosteroid\u00f3w), a tak\u017ce lek\u00f3w dost\u0119pnych bez recepty i/lub suplement\u00f3w zio\u0142owych stosowanych wspomagaj\u0105co, przez co najmniej 1 miesi\u0105c przed wizyt\u0105 przesiewow\u0105 i przez ca\u0142y 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, \u03b2-blokkers en diuretica (geen glucocorticostero\u00efden) en vrij verkrijgbare geneesmiddelen en/of kruidensupplementen voor ondersteunende zorg \u2265 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, einschlie\u00dflich ACE-Hemmern, \u03b2-Blockern und Diuretika (mit Ausnahme von Glukokortikosteroiden) und rezeptfreien Medikamenten und/oder pflanzlichen \nNahrungserg\u00e4nzungsmitteln zur unterst\u00fctzenden Behandlung \u2265 1 Monat vor der Screening-Visite und w\u00e4hrend der gesamten Dauer der Studie.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737392, "uuid": "d4389451-9556-4890-be22-b4b9df66f9d2", "attributeTranslation": "9. Des doses stables de m\u00e9dicaments sur ordonnance, y compris les inhibiteurs de l\u2019ECA, les \u03b2bloquants et les diur\u00e9tiques (\u00e0 l\u2019exclusion des glucocorticost\u00e9ro\u00efdes), et de m\u00e9dicaments en vente libre et / ou de suppl\u00e9ments \u00e0 base de plantes pour les soins de soutien \u2265 1 mois avant la visite de s\u00e9lection et pendant toute la dur\u00e9e de l\u2019\u00e9tude.", "language": 33, "languageDescription": "French (Belgium)"}]}], "principalExclusionCriteria": [{"id": 1366039, "number": 1, "principalExclusionCriteria": "Ambulatory patients expected to experience loss of ambulation within \u2264 12 months.", "principalExclusionCriteriaTranslations": [{"id": 9737246, "uuid": "f41c01c3-39c6-478f-9b98-a5e02cca48b4", "attributeTranslation": "1. Patients ambulants dont on s\u2019attend \u00e0 ce qu\u2019ils perdent leur mobilit\u00e9 dans un d\u00e9lai de \u2264 12 mois.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737247, "uuid": "f41c01c3-39c6-478f-9b98-a5e02cca48b4", "attributeTranslation": "1. Ambulatoryjni pacjenci, u kt\u00f3rych przewiduje si\u0119 utrat\u0119 zdolno\u015bci do samodzielnego poruszania si\u0119 w czasie \u2264 12 miesi\u0119cy", "language": 19, "languageDescription": "Polish"}, {"id": 9737244, "uuid": "f41c01c3-39c6-478f-9b98-a5e02cca48b4", "attributeTranslation": "Pacientes ambulatorios probablemente experimenten p\u00e9rdida de la deambulaci\u00f3n en \u2264 12 meses.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737245, "uuid": "f41c01c3-39c6-478f-9b98-a5e02cca48b4", "attributeTranslation": "1. Gehf\u00e4hige Patienten, die voraussichtlich innerhalb von \u2264 12 Monaten ihre Gehf\u00e4higkeit verlieren werden.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737248, "uuid": "f41c01c3-39c6-478f-9b98-a5e02cca48b4", "attributeTranslation": "Een ambulante pati\u00ebnt die naar verwachting binnen \u2264 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 est\u00e9n 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\u2019IRM ou la biopsie musculaire ouverte sont contre-indiqu\u00e9es.", "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 mi\u0119\u015bnia.", "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 be\u00efnvloeden.", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9737256, "uuid": "e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9", "attributeTranslation": "5. Przebyte zabiegi chirurgiczne (np. omini\u0119cie \u017co\u0142\u0105dkowe) lub schorzenia mog\u0105ce wp\u0142ywa\u0107 na wch\u0142anianie lek\u00f3w.", "language": 19, "languageDescription": "Polish"}, {"id": 9737258, "uuid": "e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9", "attributeTranslation": "5. Intervention chirurgicale (par exemple, pontage gastrique) ou \u00e9tat de sant\u00e9 susceptible d\u2019affecter l\u2019absorption des m\u00e9dicaments.", "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 beeintr\u00e4chtigen k\u00f6nnen.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737257, "uuid": "e3d679b2-932f-4d9f-899d-6a1a3bc5f1e9", "attributeTranslation": "Cirug\u00eda (por ejemplo, bypass g\u00e1strico) o enfermedad que pueda afectar a la absorci\u00f3n 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. nudno\u015bci, wymioty, biegunka, zgaga), ostra infekcja (np. grypa), powa\u017cna 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. \u00dcbelkeit, Erbrechen, Durchfall, Sodbrennen) oder eine akute Infektion (z. B. Grippe) oder eine schwerwiegende Infektion oder ein bekannter entz\u00fcndlicher Prozess beim Screening.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737261, "uuid": "d31c399b-3374-4d7a-97c4-8f409c8effc5", "attributeTranslation": "6. Pr\u00e9sence de sympt\u00f4mes gastro-intestinaux aigus (par exemple, naus\u00e9es, vomissements, diarrh\u00e9e, br\u00fblures d\u2019estomac) ou d\u2019une infection aigu\u00eb (telle que la grippe) ou d\u2019une infection importante ou d\u2019un processus inflammatoire connu au moment de la s\u00e9lection.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737259, "uuid": "d31c399b-3374-4d7a-97c4-8f409c8effc5", "attributeTranslation": "Pacientes con s\u00edntomas gastrointestinales agudos (por ejemplo, n\u00e1useas, v\u00f3mitos, diarrea, acidez) o infecci\u00f3n aguda (como gripe) o una infecci\u00f3n relevante o un proceso inflamatorio conocido en el momento de la selecci\u00f3n.", "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. Eingeschr\u00e4nkte Herzfunktion, definiert als linksventrikul\u00e4re 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. Upo\u015bledzona czynno\u015b\u0107 serca, definiowana jako frakcja wyrzutowa lewej komory < 50% w badaniach przesiewowych serca (echokardiografia lub MRI) lub obecno\u015b\u0107 objawowej kardiomiopatii.", "language": 19, "languageDescription": "Polish"}, {"id": 9737268, "uuid": "75c01d77-900e-4472-92da-22a72c577834", "attributeTranslation": "Funci\u00f3n card\u00edaca deteriorada, entendiendo como tal una fracci\u00f3n de eyecci\u00f3n del ventr\u00edculo izquierdo de <50 % en las valoraciones card\u00edacas de selecci\u00f3n (ecocardiograma o resonancia magn\u00e9tica) o evidencias de cardiomiopat\u00eda sintom\u00e1tica.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737266, "uuid": "75c01d77-900e-4472-92da-22a72c577834", "attributeTranslation": "7. Alt\u00e9ration de la fonction cardiaque d\u00e9finie par une fraction d\u2019\u00e9jection du ventricule gauche < 50 % lors des \u00e9valuations cardiaques de s\u00e9lection (\u00e9chocardiographie 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 selecci\u00f3n.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737273, "uuid": "9d4f2469-e7ca-4afe-ac4b-244ca250b85c", "attributeTranslation": "8. Wymuszona pojemno\u015b\u0107 \u017cyciowa (FVC) < 60% warto\u015bci nale\u017cnej podczas wizyty przesiewowej.", "language": 19, "languageDescription": "Polish"}, {"id": 9737272, "uuid": "9d4f2469-e7ca-4afe-ac4b-244ca250b85c", "attributeTranslation": "8. Capacit\u00e9 vitale forc\u00e9e < 60 % de la valeur pr\u00e9dite lors de la visite de s\u00e9lection.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737271, "uuid": "9d4f2469-e7ca-4afe-ac4b-244ca250b85c", "attributeTranslation": "8. Eine forcierte Vitalkapazit\u00e4t < 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, collo\u00efdaal 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 unerw\u00fcnschten Reaktion auf einen der Hilfsstoffe (Laktose-Monohydrat, mikrokristalline Cellulose, kolloidales Siliziumdioxid, Copovidon, Crospovidon, Natriumstearylfumarat) in den Tabletten des Pr\u00fcfpr\u00e4parats oder bekannte Allergie gegen SAT-3247.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737278, "uuid": "9a34faa1-c6f8-456a-83c8-6af982b5f84a", "attributeTranslation": "9.\tWyst\u0119powanie lub przesz\u0142o\u015b\u0107 ci\u0119\u017ckiej reakcji nadwra\u017cliwo\u015bci na kt\u00f3rykolwiek ze sk\u0142adnik\u00f3w 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.\tPr\u00e9sence ou ant\u00e9c\u00e9dents de r\u00e9action ind\u00e9sirable grave \u00e0 l\u2019un des excipients (lactose monohydrat\u00e9, cellulose microcristalline, dioxyde de silicium collo\u00efdal, copovidone, crospovidone, fumarate de st\u00e9aryle de sodium) contenus dans les comprim\u00e9s du m\u00e9dicament \u00e0 l\u2019\u00e9tude 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, di\u00f3xido de silicio coloidal, copovidona, crospovidona, fumarato de estearilo s\u00f3dico) presentes en los comprimidos de la medicaci\u00f3n 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 ant\u00e9c\u00e9dents connus et discernables d\u2019abus et/ou de d\u00e9pendance \u00e0 des substances", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737279, "uuid": "ef67e55a-4f3d-4826-9d38-924d70edcd15", "attributeTranslation": "16.\tUczestnicy z jak\u0105kolwiek znan\u0105 i rozpoznawaln\u0105 histori\u0105 nadu\u017cywania substancji psychoaktywnych i/lub uzale\u017cnienia.", "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 -Abh\u00e4ngigkeit 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 corticostero\u00efdregime van tien dagen op/tien dagen af.", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9737286, "uuid": "87de9c8e-0315-4fda-ace2-c7301a0b70b4", "attributeTranslation": "Participantes que est\u00e9n siguiendo una pauta de corticosteroides de diez d\u00edas con medicaci\u00f3n seguida de diez d\u00edas sin medicaci\u00f3n.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737285, "uuid": "87de9c8e-0315-4fda-ace2-c7301a0b70b4", "attributeTranslation": "10. Die Teilnehmer erhielten eine zehnt\u00e4gige Kortikosteroidtherapie mit anschlie\u00dfender zehnt\u00e4giger Pause.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737284, "uuid": "87de9c8e-0315-4fda-ace2-c7301a0b70b4", "attributeTranslation": "10. Participants maintenus sous un r\u00e9gime de corticost\u00e9ro\u00efdes 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 pozostaj\u0105cy na schemacie leczenia glikokortykosteroidami typu dziesi\u0119\u0107 dni stosowania / dziesi\u0119\u0107 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 \u2265 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 \u2265 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 f\u00fcr eine therapeutische Ma\u00dfnahme, vorherige Behandlung mit einem Gentherapie-Produkt (au\u00dfer Delandistrogen Moxeparvovec) < 24 Monate vor der Screening-Visite, oder Erhalt einer stabilen Dosis einer zugelassenen Exon-Skipping-Therapie oder eines f\u00fcr DMD indizierten Medikaments (au\u00dfer Kortikosteroiden einschlie\u00dflich Vamorolon und Givinostat entsprechend der Ausnahme 11b unten) innerhalb von 6 Monaten vor der Screening-Visite.\na.\tTeilnehmer, die ein kommerziell erh\u00e4ltliches Gentherapieprodukt (z. B. Delandistrogen Moxeparvovec) > 18 Monate vor dem Screening erhalten haben und deren Muskelfunktionstests sich stabilisiert oder verschlechtert haben, wie vom Pr\u00fcfarzt festgestellt und in der Krankenakte \u2265 3 Monate vor dem Screening dokumentiert, sind teilnahmeberechtigt. \nb.\tTeilnehmer, die eine stabile Dosis von Givinostat (Markenname Duvyzat) seit mindestens 18 Monaten oder l\u00e4nger vor der Screening-Visite erhalten, sind teilnahmeberechtigt. a.\tTeilnehmer, die Givinostat nicht vertragen und die Behandlung abgebrochen haben, k\u00f6nnen teilnehmen, wenn das Datum der letzten Dosis \u2265 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 erf\u00fcllen.\nc.\tDie Verwendung von Deflazacort (Markenname Emflaza) oder Vamorolon (Markenname Agamree) in L\u00e4ndern, in denen diese Arzneimittel noch nicht zugelassen sind, ist kein Ausschlusskriterium; die gleichzeitige Teilnahme an einer klinischen Pr\u00fcfung 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 \u00e0 une autre \u00e9tude clinique th\u00e9rapeutique ou \u00e0 une \u00e9tude de suivi pour une intervention th\u00e9rapeutique, traitement ant\u00e9rieur avec un produit de th\u00e9rapie g\u00e9nique exp\u00e9rimental (autre que le delandistrogene moxeparvovec) < 24 mois avant la visite de s\u00e9lection, ou r\u00e9ception d\u2019une dose stable d\u2019une th\u00e9rapie approuv\u00e9e de saut d\u2019exon ou de tout m\u00e9dicament indiqu\u00e9 pour la DMD (autre que les corticost\u00e9ro\u00efdes, y compris la vamorolone et le givinostat, conform\u00e9ment \u00e0 l'exception 11b ci-dessous) dans les 6 mois pr\u00e9c\u00e9dant la visite de s\u00e9lection.\na.\tLes participants ayant re\u00e7u un produit de th\u00e9rapie g\u00e9nique disponible dans le commerce (c.-\u00e0-d. delandistrogene moxeparvovec) > 18 mois avant la s\u00e9lection et dont les tests de la fonction musculaire se sont stabilis\u00e9s ou ont montr\u00e9 un d\u00e9clin tel que d\u00e9termin\u00e9 par l\u2019investigateur et document\u00e9 dans le dossier m\u00e9dical \u2265 3 mois avant la s\u00e9lection seront \u00e9ligibles.\nb.\tLes participants recevant une dose stable de givinostat (nom de marque Duvyzat) pendant au moins 18 mois ou plus avant la visite de s\u00e9lection seront \u00e9ligibles. a.\tLes participants qui ne tol\u00e8rent pas le givinostat et qui ont interrompu leur traitement sont admissibles \u00e0 l\u2019inscription si la date de la derni\u00e8re dose est \u2265 30 jours par rapport \u00e0 la date de s\u00e9lection. Le givinostat ne doit pas \u00eatre arr\u00eat\u00e9, s\u2019il est bien tol\u00e9r\u00e9, afin de satisfaire aux crit\u00e8res d\u2019inclusion dans l\u2019\u00e9tude\nc.\tL\u2019utilisation de deflazacort (nom de marque Emflaza) ou de vamorolone (nom de marque Agamree) dans des juridictions o\u00f9 ces produits sont exp\u00e9rimentaux car ils n\u2019ont pas re\u00e7u d\u2019autorisation de mise sur le march\u00e9 des autorit\u00e9s sanitaires ne sera pas consid\u00e9r\u00e9 comme un crit\u00e8re d\u2019exclusion ; toutefois, la participation simultan\u00e9e \u00e0 une \u00e9tude clinique de deflazacort ou de vamorolone sera exclue.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737289, "uuid": "3e4de5d2-2a85-4bad-a556-4ea75d37edeb", "attributeTranslation": "11.\tParticipaci\u00f3n en curso en cualquier otro estudio terap\u00e9utico o estudio de seguimiento para una intervenci\u00f3n terap\u00e9utica, tratamiento previo con un medicamento para terapia g\u00e9nica en investigaci\u00f3n (que no sea delandistrogene moxeparvovec) < 24 meses antes de la visita de selecci\u00f3n, o administraci\u00f3n de una dosis estable de una terapia aprobada de salto de ex\u00f3n o cualquier medicaci\u00f3n indicada para la DMD (que no sean corticosteroides como la vamorolona y givinostat, de acuerdo con la excepci\u00f3n 11b a continuaci\u00f3n) en los 6 meses previos a la visita de selecci\u00f3n.\na.\tSer\u00e1n aptos aquellos participantes que hayan recibido un medicamento para terapia g\u00e9nica ya comercializado (es decir, moxeparvovec delandistrog\u00e9nico) > 18 meses antes de la selecci\u00f3n cuyas pruebas de funci\u00f3n muscular se hayan estabilizado o hayan presentado un deterioro seg\u00fan las valoraciones del investigador y lo documentado en la historia cl\u00ednica \u2265 3 meses antes de la selecci\u00f3n.\nb.\tSer\u00e1n aptos aquellos participantes que reciban una dosis estable de givinostat (nombre comercial: Duvyzat) durante al menos 18 meses o m\u00e1s antes de la visita de selecci\u00f3n. a.\tLos participantes que no toleren givinostat y que hayan interrumpido el tratamiento podr\u00e1n inscribirse si la fecha de la \u00faltima dosis es \u2265 30 d\u00edas antes de la fecha de la visita de selecci\u00f3n. El tratamiento con givinostat no se debe suspender, si se tolera, para cumplir con los criterios de ingreso al estudio.\nc.\tNo ser\u00e1 un criterio de exclusi\u00f3n el uso de deflazacort (nombre comercial: Emflaza) o vamorolona (nombre comercial: Agamree) en jurisdicciones en las que est\u00e9n en fase de investigaci\u00f3n, ya que no han recibido la autorizaci\u00f3n de comercializaci\u00f3n de las autoridades sanitarias; sin embargo, la participaci\u00f3n simult\u00e1nea en un ensayo cl\u00ednico de deflazacort o vamorolona s\u00ed supondr\u00e1 un criterio de exclusi\u00f3n.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737293, "uuid": "3e4de5d2-2a85-4bad-a556-4ea75d37edeb", "attributeTranslation": "11.\tBie\u017c\u0105cy udzia\u0142 w jakimkolwiek innym terapeutycznym badaniu klinicznym lub badaniu obserwacyjnym po interwencji terapeutycznej; wcze\u015bniejsze leczenie badanym produktem terapii genowej (innym ni\u017c delandistrogene moxeparvovec) < 24 miesi\u0119cy przed wizyt\u0105 przesiewow\u0105; lub przyjmowanie stabilnej dawki zatwierdzonej terapii pomijaj\u0105cej egzon b\u0105d\u017a jakiegokolwiek innego leku wskazanego w leczeniu DMD (z wyj\u0105tkiem kortykosteroid\u00f3w, w tym wamorolonu i giwinostat zgodnie z wyj\u0105tkiem 11b poni\u017cej) w ci\u0105gu 6 miesi\u0119cy przed wizyt\u0105 przesiewow\u0105.\na.\tUczestnicy, kt\u00f3rzy otrzymali komercyjnie dost\u0119pny produkt terapii genowej (tj. delandistrogene moxeparvovec) > 18 miesi\u0119cy przed wizyt\u0105 przesiewow\u0105, u kt\u00f3rych testy czynno\u015bci mi\u0119\u015bniowej ustabilizowa\u0142y si\u0119 lub wykaza\u0142y pogorszenie, zgodnie z ocen\u0105 badacza i potwierdzone w dokumentacji medycznej \u2265 3 miesi\u0105ce przed wizyt\u0105 przesiewow\u0105, b\u0119d\u0105 kwalifikowa\u0107 si\u0119 do udzia\u0142u w badaniu.\nb.\tUczestnicy przyjmuj\u0105cy stabiln\u0105 dawk\u0119 givinostatu (nazwa handlowa: Duvyzat) przez co najmniej 18 miesi\u0119cy przed wizyt\u0105 przesiewow\u0105 lub d\u0142u\u017cej b\u0119d\u0105 kwalifikowa\u0107 si\u0119 do udzia\u0142u. a.\tUczestnicy, kt\u00f3rzy nie tolerowali givinostatu i przerwali leczenie kwalifikuj\u0105 si\u0119 do udzia\u0142u w badaniu, je\u017celi od daty przyj\u0119cia ostatniej dawki up\u0142yn\u0119\u0142o \u2265 30 dni przed wizyt\u0105 przesiewow\u0105. Nie nale\u017cy przerywa\u0107 leczenia givinostatem, je\u015bli jest tolerowane, aby spe\u0142ni\u0107 kryteria w\u0142\u0105czenia do badania.\nc.\tStosowanie deflazacortu (nazwa handlowa: Emflaza) lub vamorolonu (nazwa handlowa: Agamree) w jurysdykcjach, w kt\u00f3rych produkty te pozostaj\u0105 w fazie bada\u0144 i nie uzyska\u0142y jeszcze pozwolenia na dopuszczenie do obrotu, nie stanowi kryterium wykluczenia; jednak jednoczesny udzia\u0142 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 ge\u00efndiceerd geneesmiddel (anders dan corticostero\u00efden, 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 \u2265 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 \u2265 30 dagen v\u00f3\u00f3r 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. R\u00e9ception de SAT-3247 dans le cadre d\u2019une autre \u00e9tude.", "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 participaci\u00f3n en el estudio, a juicio del investigador.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737303, "uuid": "c24839d9-6102-4c12-a7b2-ed2d8f7f5871", "attributeTranslation": "13. Schwere Verhaltensst\u00f6rungen oder kognitive Probleme, die nach Ansicht des Pr\u00fcfarztes eine Teilnahme an der Studie ausschlie\u00dfen.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737300, "uuid": "c24839d9-6102-4c12-a7b2-ed2d8f7f5871", "attributeTranslation": "13. Powa\u017cne problemy behawioralne lub poznawcze, kt\u00f3re w ocenie badacza uniemo\u017cliwiaj\u0105 udzia\u0142 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. Probl\u00e8mes comportementaux ou cognitifs graves qui emp\u00eachent la participation \u00e0 l\u2019\u00e9tude, de l\u2019avis de l\u2019investigateur.", "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-Oberfl\u00e4chenantigen (HBsAg), Hepatitis-C-Antik\u00f6rper (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 obecno\u015b\u0107 antygenu powierzchniowego wirusa zapalenia w\u0105troby typu B (HBsAg), przeciwcia\u0142 przeciwko wirusowi zapalenia w\u0105troby 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\u2019antig\u00e8ne de surface de l\u2019h\u00e9patite B (HBsAg), l\u2019anticorps de l\u2019h\u00e9patite C (HCV) ou le VIH lors de la visite de s\u00e9lection.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737306, "uuid": "db682db8-30d9-413d-9f9c-ca168c90590a", "attributeTranslation": "Prueba positiva del ant\u00edgeno de superficie de la hepatitis B (HBsAg), del anticuerpo de la hepatitis C (VHC) o del VIH en la visita de selecci\u00f3n.", "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 Angeh\u00f6rige.", "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. Employ\u00e9s du promoteur, de la CRO et / ou du site de l\u2019\u00e9tude ou leurs proches.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737311, "uuid": "3e13a4dd-04d0-486d-8843-0280a5ae4c2b", "attributeTranslation": "15. Pracownik Sponsora, organizacji prowadz\u0105cej badanie (CRO) i/lub o\u015brodka badawczego, lub osoba spokrewniona z kt\u00f3rymkolwiek 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 cr\u00f3nica o antecedentes de enfermedad cr\u00f3nica (distinta de DMD) suficiente para invalidar la participaci\u00f3n 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 (au\u00dfer DMD), die nach dem Urteil des Pr\u00fcfarztes die Teilnahme an der Studie unm\u00f6glich macht oder unn\u00f6tig gef\u00e4hrlich 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. Wyst\u0119powanie ostrej lub przewlek\u0142ej choroby b\u0105d\u017a historia przewlek\u0142ej choroby (innej ni\u017c DMD), kt\u00f3ra w ocenie badacza mog\u0142aby uniemo\u017cliwi\u0107 udzia\u0142 w badaniu lub uczyni\u0107 go niepotrzebnie ryzykownym.", "language": 19, "languageDescription": "Polish"}, {"id": 9737316, "uuid": "5ff74ed1-f4a1-405c-9bc3-aed61dd549d4", "attributeTranslation": "2. Pr\u00e9sence d\u2019une maladie aigu\u00eb ou chronique ou d\u2019ant\u00e9c\u00e9dents de maladie chronique (autre que la DMD) de nature \u00e0 invalider la participation \u00e0 l\u2019\u00e9tude ou \u00e0 la rendre inutilement dangereuse, selon le jugement de l\u2019investigateur.", "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. Obecno\u015b\u0107 lub przebieg choroby (np. choroby wsp\u00f3\u0142istniej\u0105ce, zaburzenia psychiatryczne, zaburzenia zachowania), historia medyczna, wyniki badania fizykalnego, EKG lub nieprawid\u0142owo\u015bci laboratoryjne, kt\u00f3re zdaniem badacza mog\u0142yby negatywnie wp\u0142yn\u0105\u0107 na bezpiecze\u0144stwo uczestnika, utrudni\u0107 uko\u0144czenie leczenia lub obserwacji, b\u0105d\u017a zak\u0142\u00f3ci\u0107 ocen\u0119 wynik\u00f3w 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 be\u00efnvloeden, het onwaarschijnlijk maken dat de behandeling of follow-up zal worden afgerond of de beoordeling van de onderzoeksresultaten zouden kunnen be\u00efnvloeden.", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9737323, "uuid": "b7c64826-10ca-4cd7-8ffa-7e033764a662", "attributeTranslation": "3. \u00c9tat de sant\u00e9 ant\u00e9rieur ou actuel (par exemple, maladie concomitante, \u00e9tat psychiatrique, trouble du comportement), ant\u00e9c\u00e9dents m\u00e9dicaux, r\u00e9sultats cliniques, r\u00e9sultats ECG ou anomalies de laboratoire qui, de l\u2019avis de l\u2019investigateur, pourraient nuire \u00e0 la s\u00e9curit\u00e9 du participant, rendre improbable l\u2019ach\u00e8vement du traitement ou du suivi, ou nuire \u00e0 l\u2019\u00e9valuation des r\u00e9sultats de l\u2019\u00e9tude.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737320, "uuid": "b7c64826-10ca-4cd7-8ffa-7e033764a662", "attributeTranslation": "3. Fr\u00fchere oder aktuelle Erkrankungen (z. B. Begleiterkrankungen, psychiatrische Erkrankungen, Verhaltensst\u00f6rungen), Anamnese, k\u00f6rperliche Befunde, EKG-Befunde oder Laboranomalien, die nach Ansicht des Pr\u00fcfarztes die Sicherheit des Teilnehmers beeintr\u00e4chtigen k\u00f6nnten, die es unwahrscheinlich machen, dass die Behandlung oder die Nachuntersuchung abgeschlossen werden kann, oder die die Bewertung der Studienergebnisse beeintr\u00e4chtigen k\u00f6nnten.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737319, "uuid": "b7c64826-10ca-4cd7-8ffa-7e033764a662", "attributeTranslation": "Afecci\u00f3n m\u00e9dica previa o en curso (por ejemplo, enfermedad concomitante, afecci\u00f3n psiqui\u00e1trica o trastorno del comportamiento), antecedentes m\u00e9dicos, hallazgos f\u00edsicos, hallazgos en el ECG o anomal\u00edas en las pruebas de laboratorio que, a juicio del investigador, puedan afectar negativamente a la seguridad del participante, dificulten la finalizaci\u00f3n del tratamiento o el seguimiento, o puedan perjudicar la evaluaci\u00f3n 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 crit\u00e8re primaire d\u2019\u00e9valuation de l\u2019efficacit\u00e9 est d\u00e9fini comme le changement par rapport \u00e0 la valeur de d\u00e9but d\u2019\u00e9tude des mesures de la force musculaire d\u00e9termin\u00e9es par dynamom\u00e9trie \u00e0 la semaine 12.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737170, "uuid": "19c0199a-3aaa-4ad0-a4e8-b09c4e9f76df", "attributeTranslation": "Pierwszorz\u0119dowy punkt ko\u0144cowy skuteczno\u015bci zdefiniowano jako zmian\u0119 wzgl\u0119dem warto\u015bci wyj\u015bciowej w pomiarach si\u0142y mi\u0119\u015bniowej, okre\u015blonych za pomoc\u0105 dynamometrii, w 12. tygodniu.", "language": 19, "languageDescription": "Polish"}, {"id": 9737172, "uuid": "19c0199a-3aaa-4ad0-a4e8-b09c4e9f76df", "attributeTranslation": "Der prim\u00e4re Wirksamkeitsendpunkt ist definiert als die Ver\u00e4nderung 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 valoraci\u00f3n principal de la eficacia se define como el cambio respecto al valor basal en las mediciones de la fuerza muscular mediante dinamometr\u00eda 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 geh\u00f6ren das Auftreten, der Schweregrad und die Beziehung zu SAT-3247 von \nunerw\u00fcnschten Ereignissen sowie das Auftreten klinisch signifikanter Ver\u00e4nderungen bei der k\u00f6rperlichen 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 valoraci\u00f3n de la seguridad incluyen la incidencia, la gravedad y la relaci\u00f3n con SAT-3247 de los acontecimientos adversos, as\u00ed como la aparici\u00f3n de cambios cl\u00ednicamente significativos en la exploraci\u00f3n f\u00edsica, 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 crit\u00e8res d\u2019\u00e9valuation de la s\u00e9curit\u00e9 comprennent l\u2019incidence, la gravit\u00e9 et la relation avec le SAT-3247 des \n\u00e9v\u00e9nements ind\u00e9sirables ainsi que l\u2019apparition de changements cliniquement significatifs dans l\u2019examen clinique, les mesures cliniques de laboratoire, les signes vitaux, l\u2019ECG et le C-SSRS.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737175, "uuid": "70943981-4bb4-4122-af9f-68b6b5f652a6", "attributeTranslation": "Punkty ko\u0144cowe dotycz\u0105ce bezpiecze\u0144stwa obejmuj\u0105 cz\u0119sto\u015b\u0107 wyst\u0119powania, nasilenie i zwi\u0105zek ze stosowaniem SAT-3247 zdarze\u0144 niepo\u017c\u0105danych, a tak\u017ce wyst\u0119powanie klinicznie istotnych zmian w badaniu fizykalnym, laboratoryjnych pomiarach klinicznych, parametrach \u017cyciowych, 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": "Ver\u00e4nderungen des intramuskul\u00e4ren Fettanteils in der quantitativen Magnetresonanz (qMR) im Vastus lateralis in Woche 12 gegen\u00fcber 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 fracci\u00f3n de grasa intramuscular mediante resonancia magn\u00e9tica 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 \u00e0 la valeur de d\u00e9but d\u2019\u00e9tude de la fraction de graisse intramusculaire dans la r\u00e9sonance magn\u00e9tique quantitative (IRMq) du muscle vaste lat\u00e9ral \u00e0 la semaine 12.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737180, "uuid": "f0df4176-1260-4cb0-ae8d-98d7a8f5776b", "attributeTranslation": "Zmiany wzgl\u0119dem warto\u015bci wyj\u015bciowej w frakcji t\u0142uszczu \u015br\u00f3dmi\u0119\u015bniowego w ilo\u015bciowym rezonansie magnetycznym (qMR) mi\u0119\u015bnia 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 relajaci\u00f3n transversal de los protones (T2) en el m\u00fasculo vasto lateral en la semana 12.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737187, "uuid": "b8d27f0a-5ad5-45c2-9ad2-5943f475c94b", "attributeTranslation": "Les changements par rapport \u00e0 la valeur de d\u00e9but d\u2019\u00e9tude dans le temps de relaxation transversale du muscle protonique (T2) dans le muscle vaste lat\u00e9ral \u00e0 la semaine 12.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737186, "uuid": "b8d27f0a-5ad5-45c2-9ad2-5943f475c94b", "attributeTranslation": "Zmiany wzgl\u0119dem warto\u015bci wyj\u015bciowej w czasie relaksacji poprzecznej proton\u00f3w (T2) w mi\u0119\u015bniu obszernym bocznym w 12. tygodniu.", "language": 19, "languageDescription": "Polish"}, {"id": 9737185, "uuid": "b8d27f0a-5ad5-45c2-9ad2-5943f475c94b", "attributeTranslation": "Ver\u00e4nderungen der transversalen Relaxationszeit (T2) der Muskelprotonen im Vastus lateralis in Woche 12 gegen\u00fcber 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 wzgl\u0119dem warto\u015bci wyj\u015bciowej w wska\u017aniku regeneracji w otwartej biopsji mi\u0119\u015bnia dwug\u0142owego 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 \u00edndice de regeneraci\u00f3n en biopsia muscular abierta del b\u00edceps 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 \u00e0 la valeur de d\u00e9but d\u2019\u00e9tude de l\u2019indice de r\u00e9g\u00e9n\u00e9ration dans une biopsie musculaire ouverte du biceps brachial \u00e0 la semaine 12.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737189, "uuid": "8c90e126-35ae-4d72-aa40-1ce65a8e357a", "attributeTranslation": "Ver\u00e4nderungen des Regenerationsindex bei offener Muskelbiopsie des Musculus biceps brachii in Woche 12 gegen\u00fcber 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 wzgl\u0119dem warto\u015bci wyj\u015bciowej w zakresie funkcji, okre\u015blone 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 funci\u00f3n seg\u00fan la evaluaci\u00f3n 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 \u00e0 la valeur de d\u00e9but d\u2019\u00e9tude dans la fonction d\u00e9termin\u00e9e par le test NSAA \u00e0 la semaine 12.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737198, "uuid": "597000f9-bfbc-4718-8c62-c48b42e23388", "attributeTranslation": "Ver\u00e4nderungen der Funktion gegen\u00fcber 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": "Ver\u00e4nderungen des SV95C-Werts gegen\u00fcber der Baseline in Woche 12.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737200, "uuid": "c17b2d7f-0a79-4011-967d-3f27212d1e35", "attributeTranslation": "Zmiany wzgl\u0119dem warto\u015bci wyj\u015bciowej w SV95C w 12. tygodniu.", "language": 19, "languageDescription": "Polish"}, {"id": 9737199, "uuid": "c17b2d7f-0a79-4011-967d-3f27212d1e35", "attributeTranslation": "Les changements par rapport \u00e0 la valeur de d\u00e9but d\u2019\u00e9tude dans le SV95C \u00e0 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 ko\u0144cowy: Zmian\u0119 wzgl\u0119dem warto\u015bci wyj\u015bciowej w profilu cytokin zapalnych w 12. tygodniu.", "language": 19, "languageDescription": "Polish"}, {"id": 9737208, "uuid": "de2f2e77-efab-4283-be69-dd712635b558", "attributeTranslation": "Crit\u00e8re d\u2019\u00e9valuation exploratoire: le changement par rapport \u00e0 la valeur de d\u00e9but d\u2019\u00e9tude dans le profil des cytokines \ninflammatoires \u00e0 la semaine 12.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737204, "uuid": "de2f2e77-efab-4283-be69-dd712635b558", "attributeTranslation": "Explorativer Endpunkt: ver\u00e4nderung des entz\u00fcndlichen Zytokinprofils in Woche 12 gegen\u00fcber der Baseline.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737206, "uuid": "de2f2e77-efab-4283-be69-dd712635b558", "attributeTranslation": "Criterio de valoraci\u00f3n exploratoria: variaci\u00f3n 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 valoraci\u00f3n exploratoria: variaci\u00f3n 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": "Crit\u00e8re d\u2019\u00e9valuation exploratoire: le changement par rapport \u00e0 la valeur de d\u00e9but d\u2019\u00e9tude de la cr\u00e9atine kinase \u00e0 la semaine 12.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737213, "uuid": "15724a1c-6ff6-4da2-aaad-a3338eba6dba", "attributeTranslation": "Explorativer Endpunkt: ver\u00e4nderung der Kreatinkinase in Woche 12 gegen\u00fcber 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 ko\u0144cowy: Zmian\u0119 wzgl\u0119dem warto\u015bci wyj\u015bciowej 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: ver\u00e4nderung gegen\u00fcber der Baseline der maximalen prognostizierten forcierten Vitalkapazit\u00e4t in Prozent, gemessen durch Spirometrie in Woche 12.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737216, "uuid": "6345ae9b-4d30-4f9d-92d0-b229ca4f58b9", "attributeTranslation": "Eksploracyjny punkt ko\u0144cowy: Zmian\u0119 wzgl\u0119dem warto\u015bci wyj\u015bciowej maksymalnej procentowej przewidywanej \nwymuszonej pojemno\u015bci \u017cyciowej (FVC), mierzonej za pomoc\u0105 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": "Crit\u00e8re d\u2019\u00e9valuation exploratoire: le changement par rapport \u00e0 la valeur de d\u00e9but d\u2019\u00e9tude du pourcentage maximal de la capacit\u00e9 respiratoire forc\u00e9e pr\u00e9dite, mesur\u00e9e par spirom\u00e9trie \u00e0 la semaine 12.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737218, "uuid": "6345ae9b-4d30-4f9d-92d0-b229ca4f58b9", "attributeTranslation": "Criterio de valoraci\u00f3n exploratoria: variaci\u00f3n respecto al valor basal en el porcentaje m\u00e1ximo previsto de capacidad vital forzada medido por espirometr\u00eda 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 valoraci\u00f3n exploratoria: variaci\u00f3n en el tama\u00f1o de las fibras musculares del b\u00edceps braquial y en la distribuci\u00f3n del tama\u00f1o de las fibras, determinados a partir de la histopatolog\u00eda al cabo de 12 semanas.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737221, "uuid": "8dafafda-e529-4c90-a446-f8425f61fc2f", "attributeTranslation": "Crit\u00e8re d\u2019\u00e9valuation exploratoire: le changement de la taille des fibres musculaires du biceps brachial et de la distribution de la taille des fibres, d\u00e9termin\u00e9es par histopathologie \u00e0 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: ver\u00e4nderung der Muskelfasergr\u00f6\u00dfe und Fasergr\u00f6\u00dfenverteilung 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 ko\u0144cowy: Zmian\u0119 wielko\u015bci w\u0142\u00f3kien mi\u0119\u015bniowych mi\u0119\u015bnia dwug\u0142owego ramienia oraz rozk\u0142adu ich wielko\u015bci, okre\u015blon\u0105 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 valoraci\u00f3n exploratoria: variaci\u00f3n en la proporci\u00f3n de fibras positivas a la miosina embrionaria determinado por histopatolog\u00eda al cabo de 12 semanas.", "language": 7, "languageDescription": "Spanish"}, {"id": 9737226, "uuid": "7f3b32a5-0be3-4ead-acc3-a4c3335716ca", "attributeTranslation": "Explorativer Endpunkt: ver\u00e4nderung 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 ko\u0144cowy: Zmian\u0119 odsetka w\u0142\u00f3kien dodatnich wzgl\u0119dem zarodkowej miozyny, okre\u015blon\u0105 na podstawie badania histopatologicznego w 12. tygodniu.", "language": 19, "languageDescription": "Polish"}, {"id": 9737228, "uuid": "7f3b32a5-0be3-4ead-acc3-a4c3335716ca", "attributeTranslation": "Crit\u00e8re d\u2019\u00e9valuation exploratoire: le changement dans la proportion de fibres embryonnaires positives \u00e0 la myosine, d\u00e9termin\u00e9 par l\u2019histopathologie \u00e0 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;  ver\u00e4nderung der Anzahl der Satellitenzellen, ermittelt durch Histopathologie nach 12 Wochen.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737230, "uuid": "169f8f53-e9e2-41c4-9ff1-cb42ed222dcb", "attributeTranslation": "Crit\u00e8re d\u2019\u00e9valuation exploratoire: le changement dans le nombre de cellules satellites tel que d\u00e9termin\u00e9 par l\u2019histopathologie \u00e0 12 semaines.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737232, "uuid": "169f8f53-e9e2-41c4-9ff1-cb42ed222dcb", "attributeTranslation": "Eksploracyjny punkt ko\u0144cowy: Zmian\u0119 liczby kom\u00f3rek satelitarnych, okre\u015blon\u0105 na podstawie badania histopatologicznego po 12 tygodniach.", "language": 19, "languageDescription": "Polish"}, {"id": 9737231, "uuid": "169f8f53-e9e2-41c4-9ff1-cb42ed222dcb", "attributeTranslation": "Criterio de valoraci\u00f3n exploratoria: variaci\u00f3n en el n\u00famero de c\u00e9lulas sat\u00e9lite determinado por histopatolog\u00eda 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": "Crit\u00e8re d\u2019\u00e9valuation exploratoire: le changement de la fibrose endomysiale et de l\u2019infiltration du tissu adipeux, d\u00e9termin\u00e9es par histopathologie \u00e0 12 semaines.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737236, "uuid": "8029651e-b04f-483d-9869-d2b8c257db1d", "attributeTranslation": "Explorativer Endpunkt: ver\u00e4nderung 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 valoraci\u00f3n exploratoria: variaci\u00f3n en la fibrosis endomisial y la infiltraci\u00f3n de tejido adiposo determinado por histopatolog\u00eda 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 ko\u0144cowy: Zmian\u0119 stopnia zw\u0142\u00f3knienia \u015br\u00f3dmi\u0119\u015bniowego oraz nacieku tkanki t\u0142uszczowej, okre\u015blon\u0105 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": "Crit\u00e8re d\u2019\u00e9valuation exploratoire: le changement du score NSAA sur 12 semaines par rapport \u00e0 l\u2019\u00e9volution naturelle.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9737241, "uuid": "6f017bf0-6459-4749-871c-3a1058d18206", "attributeTranslation": "Explorativer Endpunkt: ver\u00e4nderung des NSAA-Scores \u00fcber 12 Wochen im Vergleich zum nat\u00fcrlichen Verlauf.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9737239, "uuid": "6f017bf0-6459-4749-871c-3a1058d18206", "attributeTranslation": "Eksploracyjny punkt ko\u0144cowy: Zmian\u0119 wyniku skali NSAA w ci\u0105gu 12 tygodni w por\u00f3wnaniu z histori\u0105 naturalnego przebiegu choroby.", "language": 19, "languageDescription": "Polish"}, {"id": 9737240, "uuid": "6f017bf0-6459-4749-871c-3a1058d18206", "attributeTranslation": "Verkennend eindpunt: Verandering in NSAA-score over 12 weken in vergelijking met die bij het natuurlijke beloop \nvan de ziekte.", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9737243, "uuid": "6f017bf0-6459-4749-871c-3a1058d18206", "attributeTranslation": "Criterio de valoracaci\u00f3n exploratoria: variaci\u00f3n en la puntuaci\u00f3n de NSAA durante 12 semanas en comparaci\u00f3n con la evoluci\u00f3n natural de la enfermedad.", "language": 7, "languageDescription": "Spanish"}]}]}, "trialDuration": {"estimatedGlobalEndDate": "2027-03-31", "estimatedEndDate": "2027-03-31", "estimatedRecruitmentStartDate": "2025-12-15"}, "sourceOfMonetarySupport": [{"id": 86458, "organisationName": "Satellos Bioscience Inc"}], "populationOfTrialSubjects": {"ageRanges": [{"id": 325535, "ageRangeCategoryCode": "2", "ageRangeCategory": "2"}], "ageRangeSecondaryIds": [], "clinicalTrialGroups": [{"code": "2", "name": "Patients"}], "isFemaleSubjects": false, "isMaleSubjects": true, "isVulnerablePopulationSelected": true}, "individualParticipantData": {"planToShareIPD": "2", "planDescription": "Individual participant data will not be shared, as the sponsor does not plan to make the dataset available outside the study team"}}, "protocolInformation": {"studyDesign": {"periodDetails": [{"id": 171943, "businessKey": "1", "title": "Study period 1 - Screening", "description": "Participants will be screened to evaluate their eligibility within 28 days before initiating dosing of investigational product at Baseline. \nDuring the Screening Visit, participants will be also equipped with a wearable device for assessment of their stride velocity. Participants will be trained on proper usage and will be instructed to wear one sensor on each ankle until Baseline/Visit 2.", "blindingMethodCode": "3", "blindedRoles": [], "armDetails": [], "allocationMethod": "3"}, {"id": 171944, "businessKey": "2", "title": "Study Period 2 \u2013 Treatment", "description": "Start event: Randomisation (Visit 2)\nEnd event: Final study visit (Visit 5)\nDuration: Approximately 12 weeks\nDuring this period participants will complete a Baseline visit (Visit 2),  Week 4 (Visit 3), Week 8 (Visit 4), and Week 12 (Visit 5) follow-up visits. \n\nDescription: Randomised treatment and study assessments\nDuring baseline visit (Visit 2), eligible participants will be randomized 1:1:1 to one of three treatment groups:\nSAT-3247 60 mg \nSAT-3247 120 mg\nand/or matched placebo \nadministered once daily, by mouth (PO), for 12 weeks in a blinded manner\nand stratified by:\n\u2022\tPrior DMD treatments (gene therapy, exon skipper, givinostat, or none)\n\u2022\tBaseline corticosteroid regimen (either daily or weekend dose regimen)\nDuring visit 4 Visit, participants will be also equipped again with a wearable device for assessment of their stride velocity. Participants will be trained on proper usage and will be instructed to wear one sensor on each ankle until Visit 5.", "blindingMethodCode": "2", "blindedRoles": [{"id": 171949, "code": 3, "name": "Monitor"}, {"id": 171947, "code": 4, "name": "Analyst"}, {"id": 171945, "code": 2, "name": "Investigator"}, {"id": 171948, "code": 5, "name": "Carer"}, {"id": 171946, "code": 1, "name": "Subject"}], "blindingDetails": "Randomization will be conducted using a computer-generated random sequence schema; randomization assignment will be determined by the programmed Randomization and Trial Supply Management (RTSM) platform.\nIn the event that unblinding is required, (e.g., for a serious adverse event requiring knowledge of treatment assignment), the process is managed through the RTSM platform with notification to and approval from the study medical monitor.  Unblinding would be performed on a per-participant basis, preserving the integrity of the overall study blind.  All unblinding events are logged within the RTSM and are strictly limited to circumstances in which participant safety is at risk.", "armDetails": [{"description": "Participants allocated to 60 mg arm, will take 1 10mg SAT-3247 tablet + 1 50mg SAT-3247 tablet + 1 10mg placebo tablet + 1 50mg placebo tablet for five consecutive days (i.e., weekdays) of each week for 12 weeks and 2 10mg placebo tablets + 2 50mg placebo tablets every weekend day for 12 weeks", "title": "SAT-3247 60 mg", "id": 96403}, {"description": "Participants allocated to 120 mg arm, will take 2 10mg SAT-3247 tablets + 2 50mg SAT-3247 tablets for five consecutive days (i.e., weekdays) of each week for 12 weeks and 2 10mg placebo tablets + 2 50mg placebo tablets every weekend day for 12 weeks", "title": "SAT-3247 120 mg", "id": 96404}, {"description": "Participants allocated to placebo will take 2 10mg placebo tablets + 2 50mg placebo tablets fevery day for 12 weeks", "title": "Placebo", "id": 96405}], 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45-skipping met een initi\u00eble meervoudige oplopende dosis; deel A om de veiligheid, verdraagbaarheid, farmacokinetiek en farmacodynamiek van ENTR-601-45 te beoordelen, gevolgd door deel B om de veiligheid en werkzaamheid van ENTR-601-45 (ELEVATE-45) te beoordelen", "language": 18, "languageDescription": "Dutch"}], "publicTitle": "A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)", "publicTitleTranslations": [{"id": 9743648, "uuid": "8489f332-c19a-4bbf-90c2-0656aa0fb775", "attributeTranslation": "Estudio en dos partes, aleatorizado, doble ciego y controlado con placebo en participantes con distrofia muscular de Duchenne susceptibles de omitir el ex\u00f3n 45 para evaluar la seguridad y la eficacia de ENTR-601-45 (ELEVATE-45)", "language": 7, "languageDescription": "Spanish"}, {"id": 9743647, "uuid": "8489f332-c19a-4bbf-90c2-0656aa0fb775", "attributeTranslation": "Een 2-delig, gerandomiseerd, dubbelblind, placebogecontroleerd onderzoek bij deelnemers met spierdystrofie van Duchenne die ontvankelijk zijn voor exon 45-skipping ter beoordeling van de veiligheid en werkzaamheid van ENTR-601-45 (ELEVATE-45)", "language": 18, "languageDescription": "Dutch"}], "shortTitle": "ENTR-601-45-201", "secondaryIdentifyingNumbers": {"whoUniversalTrialNumber": {"id": 488827, "number": "U1111-1316-6093"}, "additionalRegistries": []}}, "trialInformation": {"trialCategory": {"isLowIntervention": false, "trialPhase": "7", "trialCategory": "2", "justificationForTrialCategory": "Phase I/II integrated trial", "trialCategoryId": 108659}, "medicalCondition": {"partIMedicalConditions": [{"id": 123368, "medicalCondition": "Duchenne Muscular Dystrophy", "medicalConditionTranslations": [{"id": 9743580, "uuid": "0d833443-b494-4c7d-941f-c4ed17a82ac0", "attributeTranslation": "Distrofia muscular de Duchenne", "language": 7, "languageDescription": "Spanish"}, {"id": 9743579, "uuid": "0d833443-b494-4c7d-941f-c4ed17a82ac0", "attributeTranslation": "spierdystrofie van Duchenne", "language": 18, "languageDescription": "Dutch"}], "isConditionRareDisease": true}], "meddraConditionTerms": [{"termId": 100000012544, "version": "27.1", "level": "PT", "termName": "Duchenne muscular dystrophy", "classificationCode": "10013801", "organClass": 100000004850, "active": false}, {"termId": 100000012047, "version": "20.1", "level": "PT", "termName": "Duchenne muscular dystrophy gene carrier", "classificationCode": "10052655", "organClass": 100000004850, "active": false}]}, "trialObjective": {"trialScopes": [{"code": "7", "trialScopeId": 358895}, {"code": "4", "trialScopeId": 358892}, {"code": "5", "trialScopeId": 358893}, {"code": "6", "trialScopeId": 358896}, {"code": "9", "trialScopeId": 358894}], "mainObjective": "Part A and OL Period: To evaluate the safety and tolerability of ENTR-601-45 in participants with Duchenne muscular dystrophy (DMD)", "mainObjectiveTranslations": [{"id": 9743646, "uuid": "5fd6d5a5-cd82-4464-b82f-05ced56ec826", "attributeTranslation": "Deel A en OL-periode: Het beoordelen van de veiligheid en verdraagbaarheid van ENTR-601-45 bij deelnemers met spierdystrofie van Duchenne (DMD)", "language": 18, "languageDescription": "Dutch"}, {"id": 9743645, "uuid": "5fd6d5a5-cd82-4464-b82f-05ced56ec826", "attributeTranslation": "Parte A y per\u00edodo abierto: Evaluar la seguridad y tolerabilidad de ENTR601-45 en participantes con distrofia muscular de Duchenne (DMD).", "language": 7, "languageDescription": "Spanish"}], "secondaryObjectives": [{"id": 417020, "number": 1, "secondaryObjective": "To characterize the pharmacokinetics of ENTR-601-45 in participants with DMD in Part A", "secondaryObjectiveTranslations": [{"id": 9743649, "uuid": "49d32b1b-3c3a-4134-b6c1-079cb2de4666", "attributeTranslation": "Het beschrijven van de farmacokinetiek van ENTR-601-45 bij deelnemers met DMD in Deel A", "language": 18, "languageDescription": "Dutch"}, {"id": 9743650, "uuid": "49d32b1b-3c3a-4134-b6c1-079cb2de4666", "attributeTranslation": "Definir la farmacocin\u00e9tica de ENTR-601-45 en participantes con DMD en la Parte A", "language": 7, "languageDescription": "Spanish"}]}, {"id": 417021, "number": 2, "secondaryObjective": "To characterize the pharmacodynamics of ENTR-601-45 in participants with DMD in Part A", "secondaryObjectiveTranslations": [{"id": 9743652, "uuid": "586210a4-5d09-464c-b8a4-976dd4770909", "attributeTranslation": "Het beschrijven van de farmacodynamiek van ENTR-601-45 bij deelnemers met DMD in Deel A", "language": 18, "languageDescription": "Dutch"}, {"id": 9743651, "uuid": "586210a4-5d09-464c-b8a4-976dd4770909", "attributeTranslation": "Definir la farmacodin\u00e1mica de ENTR-601-45 en participantes con DMD en la Parte A", "language": 7, "languageDescription": "Spanish"}]}, {"id": 417022, "number": 3, "secondaryObjective": "To evaluate the immune response to ENTR-601-45 in participants with DMD (Part A and OL Period)", "secondaryObjectiveTranslations": [{"id": 9743654, "uuid": "19af0a22-fdc6-43c1-9358-b8a7589f0810", "attributeTranslation": "Het beoordelen van de immuunreactie op ENTR-601-45 bij deelnemers met DMD in Deel A", "language": 18, "languageDescription": "Dutch"}, {"id": 9743653, "uuid": "19af0a22-fdc6-43c1-9358-b8a7589f0810", "attributeTranslation": "Evaluar la respuesta inmunitaria a ENTR-601-45 en participantes con DMD (Parte A y per\u00edodo abierto)", "language": 7, "languageDescription": "Spanish"}]}, {"id": 417023, "number": 4, "secondaryObjective": "To evaluate the impact of ENTR-601-45 on measures of function in participants with DMD after extended dosing (Part A and OL Period)", "secondaryObjectiveTranslations": [{"id": 9743656, "uuid": "3522c14e-950e-4383-a307-ad538f448974", "attributeTranslation": "Evaluar el efecto de ENTR-601-45 en las medidas de funci\u00f3n en los participantes con DMD tras la administraci\u00f3n ampliada (Parte A y per\u00edodo abierto)", "language": 7, "languageDescription": "Spanish"}, {"id": 9743655, "uuid": "3522c14e-950e-4383-a307-ad538f448974", "attributeTranslation": "Het beoordelen van de impact van ENTR-601-45 op maten van functie bij deelnemers met DMD na lange dosering (Deel A en OL-periode)", "language": 18, "languageDescription": "Dutch"}]}]}, "eligibilityCriteria": {"principalInclusionCriteria": [{"id": 797561, "number": 1, "principalInclusionCriteria": "Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene that is amenable to exon 45 skipping as reviewed by a central genetic counselor.", "principalInclusionCriteriaTranslations": [{"id": 9743633, "uuid": "6509ce2e-a353-4ae3-b822-dfc9f6787e41", "attributeTranslation": "Genetische diagnose van DMD en bevestigde pathologische variant in het dystrofinegen die geschikt is voor exon 45-skipping zoals beoordeeld door een centrale genetisch consulent.", "language": 18, "languageDescription": "Dutch"}, {"id": 9743634, "uuid": "6509ce2e-a353-4ae3-b822-dfc9f6787e41", "attributeTranslation": "Diagn\u00f3stico gen\u00e9tico de DMD y variante patol\u00f3gica confirmada en el gen de la distrofina susceptible de omitir el ex\u00f3n 45, seg\u00fan la revisi\u00f3n de un asesor gen\u00e9tico central.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 797562, "number": 2, "principalInclusionCriteria": "Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.", "principalInclusionCriteriaTranslations": [{"id": 9743636, "uuid": "3a0f3b50-ce25-44f1-912e-7ad561b14937", "attributeTranslation": "Hombre asignado en el nacimiento con signos cl\u00ednicos compatibles con distrofia muscular de Duchenne, seg\u00fan determine el investigador.", "language": 7, "languageDescription": "Spanish"}, {"id": 9743635, "uuid": "3a0f3b50-ce25-44f1-912e-7ad561b14937", "attributeTranslation": "Mannelijk bij de geboorte met klinische symptomen die passen bij spierdystrofie van Duchenne, zoals bepaald door de onderzoeker.", "language": 18, "languageDescription": "Dutch"}]}, {"id": 797563, "number": 3, "principalInclusionCriteria": "Part A: 4-20 years of age", "principalInclusionCriteriaTranslations": [{"id": 9743638, "uuid": "cea22488-e95b-4ad7-b911-c9d848a906df", "attributeTranslation": "Parte A: 4-20 a\u00f1os de edad", "language": 7, "languageDescription": "Spanish"}, {"id": 9743637, "uuid": "cea22488-e95b-4ad7-b911-c9d848a906df", "attributeTranslation": "Deel A: 4-20 jaar", "language": 18, "languageDescription": "Dutch"}]}, {"id": 797564, "number": 4, "principalInclusionCriteria": "Ambulatory Status Part A: ambulatory with a specific Performance of the Upper Limb v2.0 (PUL 2.0) Entry item at Screening", "principalInclusionCriteriaTranslations": [{"id": 9743640, "uuid": "40313062-f7b9-49bd-ae35-fffc52ea2e62", "attributeTranslation": "Ambulante status\nDeel A: ambulant met een beginwaarde van item A \u2265 3 voor Performance of the Upper Limb v2.0 (PUL 2.0) tijdens de screening", "language": 18, "languageDescription": "Dutch"}, {"id": 9743639, "uuid": "40313062-f7b9-49bd-ae35-fffc52ea2e62", "attributeTranslation": "Estado ambulatorio                                \nParte A: ambulatorio con un \u00edtem de entrada espec\u00edfico en el rendimiento en la funci\u00f3n de las extremidades superiores, versi\u00f3n 2.0 (PUL 2.0) en la selecci\u00f3n.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 797565, "number": 5, "principalInclusionCriteria": "Adequate muscle for obtaining tissue biopsy as assessed by the investigator.", "principalInclusionCriteriaTranslations": [{"id": 9743642, "uuid": "ee684bd3-0c08-4c39-8b9d-f67d02d9793d", "attributeTranslation": "Voldoende spier voor het verkrijgen van een weefselbiopt zoals beoordeeld door de onderzoeker.", "language": 18, "languageDescription": "Dutch"}, {"id": 9743641, "uuid": "ee684bd3-0c08-4c39-8b9d-f67d02d9793d", "attributeTranslation": "M\u00fasculo adecuado para obtener la biopsia de tejido seg\u00fan la evaluaci\u00f3n del investigador.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 797566, "number": 6, "principalInclusionCriteria": "Other protocol-defined criteria apply", "principalInclusionCriteriaTranslations": [{"id": 9743644, "uuid": "9fa9e234-e328-4164-bca7-a5622bf3aa3a", "attributeTranslation": "Andere door het protocol gedefinieerde criteria zijn van toepassing", "language": 18, "languageDescription": "Dutch"}, {"id": 9743643, "uuid": "9fa9e234-e328-4164-bca7-a5622bf3aa3a", "attributeTranslation": "Se aplican otros criterios definidos por el protocolo.", "language": 7, "languageDescription": "Spanish"}]}], "principalExclusionCriteria": [{"id": 1366502, "number": 1, "principalExclusionCriteria": "Any significant concomitant medical condition that interfere with the ability to comply with protocol requirements", "principalExclusionCriteriaTranslations": [{"id": 9743613, "uuid": "1ca07a79-f4f3-4b3b-a9e5-05d1b5e67ec4", "attributeTranslation": "Sufre cualquier afecci\u00f3n m\u00e9dica concomitante significativa que interfiera en la capacidad de cumplir con los requisitos del protocolo", "language": 7, "languageDescription": "Spanish"}, {"id": 9743614, "uuid": "1ca07a79-f4f3-4b3b-a9e5-05d1b5e67ec4", "attributeTranslation": "Elke belangrijke bijkomende medische aandoening die de mogelijkheid om te voldoen aan de protocolvereisten belemmert", "language": 18, "languageDescription": "Dutch"}]}, {"id": 1366503, "number": 2, "principalExclusionCriteria": "Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant\u2019s safety", "principalExclusionCriteriaTranslations": [{"id": 9743615, "uuid": "41736bcd-3cb7-4bb7-8f44-4eaf0e8d94cd", "attributeTranslation": "Heeft een acute ziekte binnen vier weken voorafgaand aan de eerste dosis onderzoeksmiddel die de metingen van het onderzoek kan verstoren of de veiligheid van de deelnemer in gevaar kan brengen", "language": 18, "languageDescription": "Dutch"}, {"id": 9743616, "uuid": "41736bcd-3cb7-4bb7-8f44-4eaf0e8d94cd", "attributeTranslation": "Sufre una enfermedad aguda en las 4 semanas anteriores a la primera dosis del f\u00e1rmaco del estudio que pudiera interferir con las mediciones del estudio o poner en peligro la seguridad del participante", "language": 7, "languageDescription": "Spanish"}]}, {"id": 1366504, "number": 3, "principalExclusionCriteria": "Use of the following medications: a. Prior or current treatment with any exon skipping therapy within the previous 12 months b. Prior or current treatment with any gene therapy  c. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents d. Use of immunosuppressants (other than systemic or oral corticosteroids for chronic non-DMD conditions) e. Treatment with a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat", "principalExclusionCriteriaTranslations": [{"id": 9743617, "uuid": "cd48463a-bdcf-412e-ad7f-338d6e3ddd37", "attributeTranslation": "Uso de los siguientes medicamentos:\na. Tratamiento previo o actual con cualquier tratamiento de omisi\u00f3n del ex\u00f3n en los\n12 meses anteriores.\nb. Tratamiento previo o actual con cualquier terapia g\u00e9nica.\nc. Uso de anticoagulantes, antitromb\u00f3ticos o antiagregantes plaquetarios.\nd. Uso de inmunosupresores (excepto corticoesteroides sist\u00e9micos u orales para\nafecciones cr\u00f3nicas distintas de la DMD). \ne. Tratamiento con un inhibidor de la histona-desacetilasa (HDAC), por ejemplo,\ngivinostat.", "language": 7, "languageDescription": "Spanish"}, {"id": 9743618, "uuid": "cd48463a-bdcf-412e-ad7f-338d6e3ddd37", "attributeTranslation": "Gebruik van de volgende medicatie:\na. Eerdere of huidige behandeling met exon-skippingtherapie binnen de afgelopen \ntwaalf maanden\nb. Eerdere of huidige behandeling met gentherapie\nc. Gebruik van antistollingsmiddelen, antitrombotica of antiplaatjesmiddelen\nd. Gebruik van immuunonderdrukkers (anders dan waaronder systemische of orale corticostero\u00efden voor chronische niet-DMD-aandoeningen)\ne. Behandeling met histondeacetylase (HDAC)-remmer, waaronder o.a. givinostat", "language": 18, "languageDescription": "Dutch"}]}, {"id": 1366505, "number": 4, "principalExclusionCriteria": "Laboratory abnormalities", "principalExclusionCriteriaTranslations": [{"id": 9743620, "uuid": "d16e0796-1fe4-47a9-9315-47a436db4d68", "attributeTranslation": "Anomal\u00edas anal\u00edticas", "language": 7, "languageDescription": "Spanish"}, {"id": 9743619, "uuid": "d16e0796-1fe4-47a9-9315-47a436db4d68", "attributeTranslation": "Afwijkende laboratoriumwaarden", "language": 18, "languageDescription": "Dutch"}]}, {"id": 1366506, "number": 5, "principalExclusionCriteria": "Daytime ventilator dependence, or any use of invasive mechanical ventilation via tracheostomy.", "principalExclusionCriteriaTranslations": [{"id": 9743622, "uuid": "8e5fd4b0-906d-4f71-a902-9e68c76619e4", "attributeTranslation": "Afhankelijkheid van beademing overdag of gebruik van invasieve mechanische beademing via tracheostomie.", "language": 18, "languageDescription": "Dutch"}, {"id": 9743621, "uuid": "8e5fd4b0-906d-4f71-a902-9e68c76619e4", "attributeTranslation": "Dependencia de un respirador durante el d\u00eda o cualquier uso de ventilaci\u00f3n mec\u00e1nica invasiva mediante traqueotom\u00eda.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 1366507, "number": 6, "principalExclusionCriteria": "Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and/or a QT interval with Fridericia correction method (QTcF) >450 msec at Screening or prior to the first dose of study drug on Day 1.", "principalExclusionCriteriaTranslations": [{"id": 9743624, "uuid": "fdd25465-b0be-4a5c-b3a6-ded782fd9881", "attributeTranslation": "Heeft een afwijkend elektrocardiogram (ECG) dat door de onderzoeker als klinisch significant wordt beoordeeld, en/of een QT-interval met Fridericia-correctiemethode \n(QTcF) > 450 msec bij de screening of voor de eerste dosis onderzoeksmiddel op dag 1.", "language": 18, "languageDescription": "Dutch"}, {"id": 9743623, "uuid": "fdd25465-b0be-4a5c-b3a6-ded782fd9881", "attributeTranslation": "Presencia de una lectura an\u00f3mala en el electrocardiograma (ECG) evaluada como\ncl\u00ednicamente significativa por el investigador o un intervalo QT con m\u00e9todo de correcci\u00f3n de Fridericia (QTcF) >450 ms en la selecci\u00f3n o antes de la primera dosis del f\u00e1rmaco del estudio el d\u00eda 1.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 1366508, "number": 7, "principalExclusionCriteria": "Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).", "principalExclusionCriteriaTranslations": [{"id": 9743625, "uuid": "90f5adf6-e445-4e59-84ce-61643479bf66", "attributeTranslation": "Recepci\u00f3n de cualquier f\u00e1rmaco experimental o en investigaci\u00f3n, etc. en los 3 meses anteriores a la primera dosis del f\u00e1rmaco del estudio o en las 5 semividas (lo que sea m\u00e1s largo)", "language": 7, "languageDescription": "Spanish"}, {"id": 9743626, "uuid": "90f5adf6-e445-4e59-84ce-61643479bf66", "attributeTranslation": "Heeft een experimenteel of onderzoeksmiddel, etc. binnen drie maanden voorafgaand aan de eerste dosis onderzoeksmiddel of binnen vijf halfwaardetijden (wat het langst is)", "language": 18, "languageDescription": "Dutch"}]}, {"id": 1366509, "number": 8, "principalExclusionCriteria": "Use of any pharmacologic treatment, other than stable corticosteroids, that might have had an effect on muscle strength or function during the study (eg, growth hormone, testosterone).", "principalExclusionCriteriaTranslations": [{"id": 9743627, "uuid": "48c588e1-e1df-4fe1-bfa9-ccb38926f764", "attributeTranslation": "Gebruik van een farmacologische behandeling, anders dan stabiele corticostero\u00efden, die een effect zou kunnen hebben op de spierkracht of -functie tijdens het onderzoek (bijv. groeihormoon, testosteron)", "language": 18, "languageDescription": "Dutch"}, {"id": 9743628, "uuid": "48c588e1-e1df-4fe1-bfa9-ccb38926f764", "attributeTranslation": "Solo en el caso de la parte B: uso de cualquier tratamiento farmacol\u00f3gico, distinto\nde corticoesteroides estables, que pudiera tener un efecto sobre la fuerza o la funci\u00f3n\nmuscular durante el estudio (p. ej., hormona del crecimiento, testosterona).", "language": 7, "languageDescription": "Spanish"}]}, {"id": 1366510, "number": 9, "principalExclusionCriteria": "Other protocol-defined criteria apply.", "principalExclusionCriteriaTranslations": [{"id": 9743630, "uuid": "a441a961-b52e-42c0-8e6e-7ceaa18f9474", "attributeTranslation": "Andere door het protocol gedefinieerde criteria zijn van toepassing.", "language": 18, "languageDescription": "Dutch"}, {"id": 9743629, "uuid": "a441a961-b52e-42c0-8e6e-7ceaa18f9474", "attributeTranslation": "Se aplican otros criterios definidos por el protocolo.", "language": 7, "languageDescription": "Spanish"}]}]}, "endPoint": {"primaryEndPoints": [{"id": 934077, "number": 1, "endPoint": "Incidence and severity of treatment emergent adverse events (TEAEs) (Part A and OL Period)", "isPrimary": true, "endPointTranslations": [{"id": 9743582, "uuid": "e122b74f-78b1-4ffb-bd2c-1eb794e76c7c", "attributeTranslation": "Incidentie en ernst van tijdens de behandeling ontstane bijwerkingen (TEAE\u2019s) (deel A en OL-periode)", "language": 18, "languageDescription": "Dutch"}, {"id": 9743581, "uuid": "e122b74f-78b1-4ffb-bd2c-1eb794e76c7c", "attributeTranslation": "Incidencia e intensidad de los acontecimientos adversos surgidos durante el tratamiento (AAST) (Parte A y per\u00edodo abierto)", "language": 7, "languageDescription": "Spanish"}]}, {"id": 934078, "number": 2, "endPoint": "Changes in vital sign measurements (Part A and OL Period)", "isPrimary": true, "endPointTranslations": [{"id": 9743584, "uuid": "0870f271-93fd-41b7-be13-ffea8826f964", "attributeTranslation": "Veranderingen in de metingen van de vitale functies (deel A en OL-periode)", "language": 18, "languageDescription": "Dutch"}, {"id": 9743583, "uuid": "0870f271-93fd-41b7-be13-ffea8826f964", "attributeTranslation": "Variaciones de las determinaciones de constantes vitales (Parte A y per\u00edodo abierto)", "language": 7, "languageDescription": "Spanish"}]}, {"id": 934079, "number": 3, "endPoint": "Changes in clinical laboratory results (Part A and OL Period)", "isPrimary": true, "endPointTranslations": [{"id": 9743585, "uuid": "a768c455-1732-4ec3-a9b2-8a511f302918", "attributeTranslation": "Variaciones de los resultados anal\u00edticos (Parte A y per\u00edodo abierto)", "language": 7, "languageDescription": "Spanish"}, {"id": 9743586, "uuid": "a768c455-1732-4ec3-a9b2-8a511f302918", "attributeTranslation": "Veranderingen in de resultaten van de klinische laboratoriumonderzoeken (deel A en OL-periode)", "language": 18, "languageDescription": "Dutch"}]}, {"id": 934080, "number": 4, "endPoint": "Changes in electrocardiogram (ECG) parameters (Part A and OL Period)", "isPrimary": true, "endPointTranslations": [{"id": 9743587, "uuid": "4c629ce0-10f5-477d-9ae1-039bb1df4b37", "attributeTranslation": "Variaciones de los par\u00e1metros electrocardiogr\u00e1ficos (ECG) (Parte A y per\u00edodo abierto)", "language": 7, "languageDescription": "Spanish"}, {"id": 9743588, "uuid": "4c629ce0-10f5-477d-9ae1-039bb1df4b37", "attributeTranslation": "Veranderingen in de parameters van het elektrocardiogram (ecg) (deel A en OL-periode)", "language": 18, "languageDescription": "Dutch"}]}, {"id": 934081, "number": 5, "endPoint": "Changes in physical examination findings (Part A and OL Period)", "isPrimary": true, "endPointTranslations": [{"id": 9743589, "uuid": "1f64d722-4d53-47c9-a33c-c8cb0a5572c5", "attributeTranslation": "Veranderingen in de bevindingen uit het lichamelijk onderzoek (deel A en OL-periode)", "language": 18, "languageDescription": "Dutch"}, {"id": 9743590, "uuid": "1f64d722-4d53-47c9-a33c-c8cb0a5572c5", "attributeTranslation": "Variaciones de los hallazgos de la exploraci\u00f3n f\u00edsica (Parte A y per\u00edodo abierto)", "language": 7, "languageDescription": "Spanish"}]}], "secondaryEndPoints": [{"id": 934082, "number": 1, "endPoint": "Plasma, muscle, and urine concentration of ENTR-601-45 and its final metabolite (Part A and OL Period)", "isPrimary": false, "endPointTranslations": [{"id": 9743591, "uuid": "8500c27e-5b72-412d-9a7d-4459b2c058d3", "attributeTranslation": "De concentratie van ENTR-601-45 en diens uiteindelijke metaboliet in het plasma, de spieren en de urine (Deel A en OL-periode)", "language": 18, "languageDescription": "Dutch"}, {"id": 9743592, "uuid": "8500c27e-5b72-412d-9a7d-4459b2c058d3", "attributeTranslation": "Concentraci\u00f3n plasm\u00e1tica, muscular y en orina de ENTR-601-45 y su metabolito final (Parte A y periodo abierto)", "language": 7, "languageDescription": "Spanish"}]}, {"id": 934083, "number": 2, "endPoint": "Change from baseline to End of Part A  in dystrophin by Western blot from muscle biopsy (Part A)", "isPrimary": false, "endPointTranslations": [{"id": 9743594, "uuid": "453c1d42-d477-452f-b501-d5575f268c96", "attributeTranslation": "Verandering van baseline tot het einde van deel A van dystrofine volgens de Western blot op spierbiopsie (Deel A)", "language": 18, "languageDescription": "Dutch"}, {"id": 9743593, "uuid": "453c1d42-d477-452f-b501-d5575f268c96", "attributeTranslation": "Variaci\u00f3n de la distrofina, determinada mediante inmunotransferencia a partir de una biopsia muscular, entre el momento basal y el final de la Parte A  (Parte A)", "language": 7, "languageDescription": "Spanish"}]}, {"id": 934084, "number": 3, "endPoint": "Change from baseline to End of Part A  in dystrophin expression and localization from muscle biopsy (Part A)", "isPrimary": false, "endPointTranslations": [{"id": 9743595, "uuid": "40ff13b5-d993-4997-bc86-21c9088a4615", 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by the investigator.", "principalInclusionCriteriaTranslations": [{"id": 9324286, "uuid": "878bd61e-caa3-42a4-9fe8-b15c3bcb4cb8", "attributeTranslation": "M\u00fasculo adecuado para obtener una biopsia de tejido, seg\u00fan la evaluaci\u00f3n del investigador.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 765878, "number": 6, "principalInclusionCriteria": "Other protocol-defined criteria apply", "principalInclusionCriteriaTranslations": [{"id": 9324287, "uuid": "38ef7a8e-047e-4943-a4c5-fdb26f2f6108", "attributeTranslation": "Se aplican otros criterios definidos por el protocolo", "language": 7, "languageDescription": "Spanish"}]}], "principalExclusionCriteria": [{"id": 1312439, "number": 1, "principalExclusionCriteria": "Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements", "principalExclusionCriteriaTranslations": [{"id": 9324273, "uuid": "1939600f-2e08-4644-b634-cc7d2a547eaf", "attributeTranslation": 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In accordance with EMA guidance on disclosure rules, the study is classified as a Category 2 trial.", "trialCategoryId": 84058}, "medicalCondition": {"partIMedicalConditions": [{"id": 95915, "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": "5", "trialScopeId": 277765}, {"code": "4", "trialScopeId": 277764}], "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": 7244764, "uuid": "51f13ad7-3c50-4f8d-888f-b7b8b9361f0c", "attributeTranslation": "G\u0142\u00f3wne cele fazy 1 obejmuj\u0105 (i) ocen\u0119 bezpiecze\u0144stwa poprzez kliniczn\u0105 obserwacj\u0119 cz\u0119sto\u015bci wyst\u0119powania i ci\u0119\u017cko\u015bci wszystkich zdarze\u0144 niepo\u017c\u0105danych: zdarze\u0144 niepo\u017c\u0105danych (Adverse Events, AE), ci\u0119\u017ckich zdarze\u0144 niepo\u017c\u0105danych (Serious Adverse Events, SAE) i zdarze\u0144 niepo\u017c\u0105danych o szczeg\u00f3lnym znaczeniu (Adverse Events of Special Interest, AESI) w celu oceny potencjalnych ryzyk; (ii) ocen\u0119 skuteczno\u015bci na podstawie oceny funkcjonalnej si\u0142y i funkcji mi\u0119\u015bni, dostosowanej do stadium choroby: (a) dla pacjent\u00f3w chodz\u0105cych: 6-Minute Walk Test (6MWT); funkcje czasowe NorthStar Ambulatory Assessment (NSAA): wstanie z pozycji le\u017c\u0105cej na plecach oraz 10-metrowy marsz/bieg; (b) dla pacjent\u00f3w niechodz\u0105cych: PUL 2.0 oraz (c) zar\u00f3wno dla pacjent\u00f3w chodz\u0105cych, jak i niechodz\u0105cych: ocena elektromiograficzna (EMG) czasu trwania potencja\u0142u jednostek motorycznych (Motor Unit Potential, MUP) wybranych mi\u0119\u015bni.\n\nG\u0142\u00f3wne cele fazy 2 obejmuj\u0105 ocen\u0119 bezpiecze\u0144stwa poprzez kliniczn\u0105 obserwacj\u0119 cz\u0119sto\u015bci wyst\u0119powania i ci\u0119\u017cko\u015bci wszystkich zdarze\u0144 niepo\u017c\u0105danych: zdarze\u0144 niepo\u017c\u0105danych (Adverse Events, AE), ci\u0119\u017ckich zdarze\u0144 niepo\u017c\u0105danych (Serious Adverse Events, SAE) i zdarze\u0144 niepo\u017c\u0105danych o szczeg\u00f3lnym znaczeniu (Adverse Events of Special Interest, AESI) w celu oceny potencjalnych ryzyk; ocen\u0119 skuteczno\u015bci na podstawie oceny funkcjonalnej si\u0142y i funkcji mi\u0119\u015bni, dostosowanej do stadium choroby: (a) dla pacjent\u00f3w chodz\u0105cych: 6-Minute Walk Test (6MWT); funkcje czasowe NorthStar Ambulatory Assessment (NSAA): wstanie z pozycji le\u017c\u0105cej na plecach oraz 10-metrowy marsz/bieg; (b) dla pacjent\u00f3w niechodz\u0105cych: PUL 2.0 oraz (c) zar\u00f3wno dla pacjent\u00f3w chodz\u0105cych, jak i niechodz\u0105cych: ocena elektromiograficzna (EMG) czasu trwania potencja\u0142u jednostek motorycznych (Motor Unit Potential, MUP) wybranych mi\u0119\u015bni.", "language": 19, "languageDescription": "Polish"}], "secondaryObjectives": []}, "eligibilityCriteria": {"principalInclusionCriteria": [{"id": 612913, "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": 7244757, "uuid": "b26e3bef-ca13-4b30-a775-404b0a92be4d", "attributeTranslation": "Uczestnik i jego przedstawiciel ustawowy / rodzic(e) / opiekun prawny zrozumieli i wyrazili zgod\u0119 na udzia\u0142 w badaniu zgodnie ze wszystkimi procedurami badania, podpisuj\u0105c zatwierdzony formularz \u015bwiadomej zgody.", "language": 19, "languageDescription": "Polish"}]}, {"id": 612914, "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": 7244758, "uuid": "54b29eef-c89f-49cc-b537-3a91bbbdb111", "attributeTranslation": "Ch\u0142opcy w wieku od 5 do 18 lat (w momencie wizyty przesiewowej), u kt\u00f3rych zdiagnozowano dystrofi\u0119 mi\u0119\u015bniow\u0105 Duchenne\u2019a potwierdzon\u0105 badaniami genetycznymi.", "language": 19, "languageDescription": "Polish"}]}, {"id": 612915, "number": 3, "principalInclusionCriteria": "Subjects must be receiving glucocorticosteroids for a minimum of 6 months prior to the biopsy of muscle tissue.", "principalInclusionCriteriaTranslations": [{"id": 7244759, "uuid": "1b8222c8-33d6-4186-bf1f-1f377292a0e2", "attributeTranslation": "Uczestnicy musz\u0105 przyjmowa\u0107 glikokortykosteroidy przez co najmniej 6 miesi\u0119cy przed biopsj\u0105 tkanki mi\u0119\u015bniowej.", "language": 19, "languageDescription": "Polish"}]}, {"id": 612916, "number": 4, "principalInclusionCriteria": "Subjects with progressive, symmetrical proximal muscle weakness of arms and legs.", "principalInclusionCriteriaTranslations": [{"id": 7244760, "uuid": "5edf8e51-39a2-472f-b7b5-d734ac0aa29e", "attributeTranslation": "Uczestnicy wykazuj\u0105 post\u0119puj\u0105c\u0105 i symetryczn\u0105 utrat\u0119 si\u0142y mi\u0119\u015bniowej w odcinkach proksymalnych ko\u0144czyn g\u00f3rnych i dolnych.", "language": 19, "languageDescription": "Polish"}]}, {"id": 612917, "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": 7244761, "uuid": "5356bb35-e1a4-4808-a7e3-f5a51ac95dd3", "attributeTranslation": "Uczestnicy wykazuj\u0105 gotowo\u015b\u0107 i zdolno\u015b\u0107 do przestrzegania termin\u00f3w zaplanowanych wizyt, poddania si\u0119 procedurze biopsji tkanek w znieczuleniu, przestrzegania planu przyjmowania lek\u00f3w, poddawania si\u0119 badaniom laboratoryjnym, zastosowania si\u0119 do ogranicze\u0144 spowodowanych udzia\u0142em w badaniu oraz zastosowania si\u0119 do procedur badania i wykonywania bada\u0144 funkcjonalnych dostosowanych do stopnia zaawansowania choroby.", "language": 19, "languageDescription": "Polish"}]}, {"id": 612918, "number": 6, "principalInclusionCriteria": "Willingness to use acceptable forms of contraception if the subject is sexually active.", "principalInclusionCriteriaTranslations": [{"id": 7244762, "uuid": "40aa92af-80e5-49f1-b8c6-de44da72694b", "attributeTranslation": "Uczestnicy wykazuj\u0105 gotowo\u015b\u0107 do stosowania akceptowalnych metod antykoncepcji, je\u015bli s\u0105 aktywni seksualnie.", "language": 19, "languageDescription": "Polish"}]}, {"id": 612919, "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": 7244763, "uuid": "59deec33-ebeb-4f8d-afdc-d3788e7b30f9", "attributeTranslation": "Pacjenci musz\u0105 pomy\u015blnie przej\u015b\u0107 kwalifikacj\u0119 anestezjologiczn\u0105 do pobrania biopsji tkanki mi\u0119\u015bniowej i zabiegu doszpikowego podania DT-DEC01, kt\u00f3re zostan\u0105 wykonane w znieczuleniu (miejscowym/og\u00f3lnym/anagosedacji).", "language": 19, "languageDescription": "Polish"}]}], "principalExclusionCriteria": [{"id": 1053407, "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": 7244742, "uuid": "5b9a62c4-6cf3-42ce-a4c7-bd867b0e761a", "attributeTranslation": "Uczestnika poddano uprzednio leczeniu badanym produktem leczniczym, podobn\u0105 terapi\u0105 eksperymentaln\u0105 z zastosowaniem ATMP lub innym produktem opartym na kom\u00f3rkach, terapi\u0105 genow\u0105 lub lekiem translarna/ataluren.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053408, "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": 7244743, "uuid": "75ae929e-2298-4dd8-aafc-e4c013969611", "attributeTranslation": "U uczestnika wyst\u0105pi\u0142 jakikolwiek uraz lub zabieg mog\u0105cy utrudni\u0107 ocen\u0119 funkcjonaln\u0105. Przebyte urazy musz\u0105 by\u0107 ca\u0142kowicie wyleczone przed wyra\u017ceniem zgody. Przebyte z\u0142amania ko\u0144czyn dolnych musz\u0105 by\u0107 ca\u0142kowicie wyleczone i od daty urazu musz\u0105 up\u0142yn\u0105\u0107 co najmniej 3 miesi\u0105ce.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053409, "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": 7244744, "uuid": "f4c91c72-9d7c-448d-b261-8134c0785723", "attributeTranslation": "U uczestnika stwierdzono obecno\u015b\u0107 lub histori\u0119 innych chor\u00f3b uk\u0142adu mi\u0119\u015bniowo-szkieletowego, neurologicznych lub zaburze\u0144 somatycznych niezwi\u0105zanych z DMD, w tym chor\u00f3b p\u0142uc i serca.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053410, "number": 12, "principalExclusionCriteria": "History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of the investigational product.", "principalExclusionCriteriaTranslations": [{"id": 7244745, "uuid": "95919680-8e11-4b25-8142-e58973c5cdf4", "attributeTranslation": "W przesz\u0142o\u015bci wyst\u0105pi\u0142a u uczestnika reakcja alergiczna lub anafilaktyczna na bia\u0142ko terapeutyczne lub diagnostyczne lub na substancje dodatkowe badanego produktu leczniczego.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053411, "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": 7244746, "uuid": "22894461-e500-4309-9c2b-1bf27b53a274", "attributeTranslation": "Uczestnik obecnie przewlekle stosuje jakiekolwiek \u015brodki o dzia\u0142aniu immunomoduluj\u0105cym (aktywuj\u0105cym lub hamuj\u0105cym), takie jak (ale nie wy\u0142\u0105cznie) leki immunosupresyjne lub leki zwi\u0105zane z immunoterapi\u0105 (np. oparte na przeciwcia\u0142ach), chemioterapi\u0105 lub podobn\u0105 terapi\u0105 wp\u0142ywaj\u0105c\u0105 na proliferacj\u0119 kom\u00f3rek.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053412, "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": 7244747, "uuid": "e2bed3b4-a25e-415f-9064-3e14f5b51980", "attributeTranslation": "U uczestnika stwierdzono inny powa\u017cny, ostry lub przewlek\u0142y stan chorobowy lub psychiczny albo nieprawid\u0142owo\u015b\u0107 w wynikach bada\u0144 laboratoryjnych, kt\u00f3re mog\u0105 zwi\u0119ksza\u0107 ryzyko zwi\u0105zane z uczestnictwem w badaniu lub podawaniem badanego produktu lub mog\u0105 wp\u0142ywa\u0107 na interpretacj\u0119 wynik\u00f3w badania i, w opinii badacza, sprawia\u0107, \u017ce dana osoba nie nadaje si\u0119 do w\u0142\u0105czenia do tego badania.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053413, "number": 2, "principalExclusionCriteria": "Subject has known history of immune reaction to the administered therapies or history of GvHD.", "principalExclusionCriteriaTranslations": [{"id": 7244748, "uuid": "c8fca15b-2a1c-4a84-b6e6-5b9d93f488aa", "attributeTranslation": "W przesz\u0142o\u015bci wyst\u0105pi\u0142a u uczestnika reakcja immunologiczna na podany lek lub GvHD.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053414, "number": 3, "principalExclusionCriteria": "Subjects with known cognitive impairment or behavioral issues that would impede the ability to follow instructions.", "principalExclusionCriteriaTranslations": [{"id": 7244749, "uuid": "1ee202f8-e343-41cb-8f62-a56df6e4c8d6", "attributeTranslation": "U uczestnika stwierdzono zaburzenia sfery poznawczej lub problemy z zachowaniem, kt\u00f3re upo\u015bledza\u0142yby zdolno\u015b\u0107 do wykonywania polece\u0144.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053415, "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": 7244750, "uuid": "d02ecd1f-818c-4967-9457-cc5f73f4f00d", "attributeTranslation": "U uczestnika stwierdzono podczas wizyty przesiewowej (wizyta V0a) dodatnie wyniki bada\u0144 w kierunku zaka\u017cenia: ludzkim wirusem niedoboru odporno\u015bci (HIV) 1 lub 2, wirusem zapalenia w\u0105troby typu B (HBV) lub C (HCV*), wirusem cytomegalii (CMV*), toksoplazmoz\u0105* lub ki\u0142\u0105. *W przypadku pozytywnego lub w\u0105tpliwego wyniku testu Anty-CMV IgG, Anty- Toxo IgG, Anty-HCV Total decyzja o ostatecznej kwalifikacji tkanki mo\u017ce zosta\u0107 podj\u0119ta po potwierdzeniu ujemnego wyniku testu na obecno\u015b\u0107 kwasu nukleinowego wirusa (NAT) na etapie hodowli kom\u00f3rkowej.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053416, "number": 5, "principalExclusionCriteria": "Subject has a history of any autoimmune disease.", "principalExclusionCriteriaTranslations": [{"id": 7244751, "uuid": "85c93ec3-65dd-4dc7-9e0f-e59be40f252b", "attributeTranslation": "W przesz\u0142o\u015bci wyst\u0105pi\u0142a u uczestnika choroba o pod\u0142o\u017cu autoimmunologicznym.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053417, "number": 6, "principalExclusionCriteria": "Ongoing participation in any other therapeutic clinical trial.", "principalExclusionCriteriaTranslations": [{"id": 7244752, "uuid": "6b01c1de-0ead-4690-85fc-3cfb184364aa", "attributeTranslation": "Uczestnik aktualnie bierze udzia\u0142 w jakimkolwiek innym badaniu klinicznym o charakterze terapeutycznym.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053418, "number": 7, "principalExclusionCriteria": "Presence of pre-existing antibodies in the subject\u2019s serum against the donor lymphocytes.", "principalExclusionCriteriaTranslations": [{"id": 7244753, "uuid": "ca3f1fc3-1862-498d-8391-f6e48f281775", "attributeTranslation": "W surowicy uczestnika wykryto obecno\u015b\u0107 przeciwcia\u0142 skierowanych przeciwko limfocytom dawcy.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053419, "number": 8, "principalExclusionCriteria": "Subject has undergone an organ or bone marrow transplantation.", "principalExclusionCriteriaTranslations": [{"id": 7244754, "uuid": "2a3198ad-b1d9-46b6-b098-2ab5d17f5984", "attributeTranslation": "Uczestnik przeszed\u0142 przeszczep narz\u0105du lub szpiku kostnego.", "language": 19, "languageDescription": "Polish"}]}, {"id": 1053420, "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": 7244755, "uuid": "7649aab1-357b-4ae7-ac39-91d056f5a85c", "attributeTranslation": "U uczestnika wprowadzono zmian\u0119 w og\u00f3lnoustrojowym leczeniu kortykosteroidami (np. rozpocz\u0119cie leczenia, przerwanie leczenia, zmiana dawki, schematu lub rodzaju steryd\u00f3w) w ci\u0105gu 3 miesi\u0119cy przed podaniem produktu badanego.", "language": 19, "languageDescription": "Polish"}]}]}, "endPoint": {"primaryEndPoints": [{"id": 716153, "number": 1, "endPoint": "Phase 1: The frequency of the incidence and severity of all adverse events \u2013 AEs, SAEs and AESI.", "isPrimary": true, "endPointTranslations": [{"id": 7244733, "uuid": "94bcee7d-890d-444b-a72d-ec3e497ffd69", "attributeTranslation": "Faza 1: Cz\u0119sto\u015b\u0107 wyst\u0119powania i ci\u0119\u017cko\u015b\u0107 wszystkich zdarze\u0144 niepo\u017c\u0105danych: zdarze\u0144 niepo\u017c\u0105danych (Adverse Events, AE), ci\u0119\u017ckich zdarze\u0144 niepo\u017c\u0105danych (Serious Adverse Events, SAE) i zdarze\u0144 niepo\u017c\u0105danych o szczeg\u00f3lnym znaczeniu (Adverse Events of Special Interest, AESI).", "language": 19, "languageDescription": "Polish"}]}, {"id": 716154, "number": 2, "endPoint": "Phase 1: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run, for non-ambulatory patients: PUL 2.0, at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).", "isPrimary": true, "endPointTranslations": [{"id": 7244734, "uuid": "18f77c04-08d0-4327-8711-ac1ef13bfabf", "attributeTranslation": "Faza 1: \u015arednie zmiany w stosunku do warto\u015bci wyj\u015bciowych (V0a) odnotowane w ocenie funkcjonalnej dostosowanej do stopnia zaawansowania choroby: dla pacjent\u00f3w chodz\u0105cych: 6MWT, funkcje czasowe NSAA: wstanie z pozycji le\u017c\u0105cej na plecach oraz 10-metrowy marsz/bieg, dla pacjent\u00f3w niechodz\u0105cych: PUL 2.0, w miesi\u0105cu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).", "language": 19, "languageDescription": "Polish"}]}, {"id": 716155, "number": 3, "endPoint": "Phase 1: Mean changes from the baseline (V0a) in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients, at month 3 (V5), 6 (V6), and 12 (V7).", "isPrimary": true, "endPointTranslations": [{"id": 7244735, "uuid": "d6c6def1-dd9c-4235-8405-71fba1a03a4e", "attributeTranslation": "Faza 1: \u015arednie zmiany w stosunku do warto\u015bci wyj\u015bciowych (V0a) w ocenie EMG czasu trwania MUP wybranych mi\u0119\u015bni ko\u0144czyn g\u00f3rnych i dolnych u pacjent\u00f3w chodz\u0105cych i niechodz\u0105cych, w miesi\u0105cu 3 (V5), 6 (V6) i 12 (V7).", "language": 19, "languageDescription": "Polish"}]}, {"id": 716156, "number": 4, "endPoint": "Phase 2: The frequency of the incidence and severity of all adverse events \u2013 AEs, SAEs and AESI.", "isPrimary": true, "endPointTranslations": [{"id": 7244736, "uuid": "c2bf2d47-3ee7-4487-8c37-8d4ce1b0d341", "attributeTranslation": "Faza 2: Cz\u0119sto\u015b\u0107 wyst\u0119powania i ci\u0119\u017cko\u015b\u0107 wszystkich zdarze\u0144 niepo\u017c\u0105danych: zdarze\u0144 niepo\u017c\u0105danych (Adverse Events, AE), ci\u0119\u017ckich zdarze\u0144 niepo\u017c\u0105danych (Serious Adverse Events, SAE) i zdarze\u0144 niepo\u017c\u0105danych o szczeg\u00f3lnym znaczeniu (Adverse Events of Special Interest, AESI).", "language": 19, "languageDescription": "Polish"}]}, {"id": 716157, "number": 5, "endPoint": "Phase 2: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: a. for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run b. for non-ambulatory patients: PUL 2.0 at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).", "isPrimary": true, "endPointTranslations": [{"id": 7244737, "uuid": "afd18f42-1662-463e-9308-01707fe17883", "attributeTranslation": "Faza 2: \u015arednie zmiany w stosunku do warto\u015bci wyj\u015bciowych (V0a) odnotowane w ocenie funkcjonalnej dostosowanej do stopnia zaawansowania choroby: dla pacjent\u00f3w chodz\u0105cych: 6MWT, funkcje czasowe NSAA: wstanie z pozycji le\u017c\u0105cej na plecach oraz 10-metrowy marsz/bieg, dla pacjent\u00f3w niechodz\u0105cych: PUL 2.0, w miesi\u0105cu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).", "language": 19, "languageDescription": "Polish"}]}, {"id": 716158, "number": 6, "endPoint": "Phase 2: Mean changes from the baseline in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients at month 3, 6 and 12 .", "isPrimary": true, "endPointTranslations": [{"id": 7244738, "uuid": "12037456-940e-4a13-8286-86a53e527d9d", "attributeTranslation": "Faza 2: \u015arednie zmiany w stosunku do warto\u015bci wyj\u015bciowych (V0a) w ocenie EMG czasu trwania MUP wybranych mi\u0119\u015bni ko\u0144czyn g\u00f3rnych i dolnych u pacjent\u00f3w chodz\u0105cych i niechodz\u0105cych, w miesi\u0105cu 3 (V5), 6 (V6) i 12 (V7).", "language": 19, "languageDescription": "Polish"}]}], "secondaryEndPoints": [{"id": 716159, "number": 1, "endPoint": "Phase 2: Mean changes from the baseline (V0a) recorded by functional assessments (adjusted to the stage of the disease): for ambulatory patients: PUL 2.0, assessment of grip strength by dynamometer; for non-ambulatory patients: assessment of grip strength by dynamometer. At month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).", "isPrimary": false, "endPointTranslations": [{"id": 7244739, "uuid": "ab75757c-fbad-4f7b-80c5-53fcb8826005", "attributeTranslation": "Faza 2: \u015arednie zmiany w stosunku do warto\u015bci wyj\u015bciowych (V0a) odnotowane w ocenie funkcjonalnej dostosowanej do stopnia zaawansowania choroby: dla pacjent\u00f3w chodz\u0105cych: PUL 2.0, ocena si\u0142y u\u015bcisku r\u0119ki za pomoc\u0105 dynamometru, dla pacjent\u00f3w niechodz\u0105cych: ocena si\u0142y u\u015bcisku r\u0119ki za pomoc\u0105 dynamometru, w miesi\u0105cu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).", "language": 19, "languageDescription": "Polish"}]}, {"id": 716160, "number": 2, "endPoint": "Phase 2: Mean changes from the baseline (V0a) in QoL assessed by PODCI at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).", "isPrimary": false, "endPointTranslations": [{"id": 7244740, "uuid": "9c5a0976-8235-49ff-b518-9746299e156a", "attributeTranslation": "Faza 2: \u015arednie zmiany w stosunku do warto\u015bci wyj\u015bciowych (V0a) w jako\u015bci \u017cycia (Quality of Life, QoL) ocenianej w kwestionariuszu PODCI (Pediatrics Outcomes Data Collection Instrument) w miesi\u0105cu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).", "language": 19, "languageDescription": "Polish"}]}, {"id": 716161, "number": 3, "endPoint": "Phase 2: Evaluation of Overall Treatment Effect at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).", "isPrimary": false, "endPointTranslations": [{"id": 7244741, "uuid": "525ebdf3-248f-436d-bd30-7d55a616af4c", "attributeTranslation": "Faza 2: Ocena og\u00f3lnego wyniku leczenia za pomoc\u0105 kwestionariusza Overall Treatment Effect (OTE) w miesi\u0105cu 1 (V4), 3 (V5), 6 (V6) i 12 (V7).", "language": 19, "languageDescription": "Polish"}]}]}, "trialDuration": {"estimatedEndDate": "2030-12-31", "estimatedRecruitmentStartDate": "2027-12-12"}, "sourceOfMonetarySupport": [], "populationOfTrialSubjects": {"ageRanges": [{"id": 254450, "ageRangeCategoryCode": "2", "ageRangeCategory": "2"}], "ageRangeSecondaryIds": [{"id": 254452, "ageRangeCategoryCode": "2", "ctAgeRangeCode": "5", "ageRangeCategory": "2", "ctAgeRange": "5"}, {"id": 254451, "ageRangeCategoryCode": "2", "ctAgeRangeCode": "4", "ageRangeCategory": "2", "ctAgeRange": "4"}, {"id": 254453, "ageRangeCategoryCode": "2", "ctAgeRangeCode": "6", "ageRangeCategory": "2", "ctAgeRange": "6"}], "clinicalTrialGroups": [{"code": "2", "name": "Patients"}], "isFemaleSubjects": false, "isMaleSubjects": true, "isVulnerablePopulationSelected": true}, "individualParticipantData": {"planToShareIPD": "2"}}, "protocolInformation": {"studyDesign": {"periodDetails": []}}, "scientificAdviceAndPip": {"scientificAdvices": [], "paediatricInvestigationPlan": []}, "associatedClinicalTrials": [], "references": [], "pubmedCode": [], "pubmedUrl": []}, "assessmentOutcome": "acceptable", "assessmentOutcomeDate": "2025-10-06", "conclusionDate": "2025-10-06", "therapeuticAreas": [{"code": "5", "name": "Diseases [C] - Musculoskeletal Diseases [C05]"}], "medicalConditions": [{"id": 95915, "medicalCondition": "Duchenne Muscular Dystrophy", "isConditionRareDisease": true}], "sponsors": [{"id": 102567, "primary": true, "publicContacts": [{"id": 306290, "type": "Public", "functionalName": "Contact Point", "functionalEmailAddress": "siemiom@dystrogen.com", "telephone": "+48530711336", "organisation": {"id": 664419, "type": "Pharmaceutical company", "typeCode": "10", "name": "Dystrogen Therapeutics Technology Polska Sp. z o.o.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100045218", "organisationLocationStatus": "Active"}}], "scientificContacts": [{"id": 306291, "type": "Scientific", "functionalName": "Contact Point", "functionalEmailAddress": "siemiom@dystrogen.com", "telephone": "+48530711336", "organisation": {"id": 664419, "type": "Pharmaceutical company", "typeCode": "10", "name": "Dystrogen Therapeutics Technology Polska Sp. z o.o.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100045218", "organisationLocationStatus": "Active"}}], "thirdParties": [], "organisation": {"id": 664419, "type": "Pharmaceutical company", "typeCode": "10", "name": "Dystrogen Therapeutics Technology Polska Sp. z o.o.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100045218", "organisationLocationStatus": "Active"}, "addresses": [{"id": 626513, "organisation": {"id": 664419, "type": "Pharmaceutical company", "typeCode": "10", "name": "Dystrogen Therapeutics Technology Polska Sp. z o.o.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100045218", "organisationLocationStatus": "Active"}, "address": {"addressId": 677692, "oneLine": "Ul. Wladyslawa Pytlasinskiego 10/12/14", "addressLine1": "Ul. Wladyslawa Pytlasinskiego 10/12/14", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Warsaw", "postcode": "00-777", "country": 2031, "countryName": "Poland"}, "isBusinessKeyValidated": true, "businessKey": "ORG-100045218"}], "isCommercial": true, "commercial": "Commercial"}], "trialCategoryCode": "2", "trialCategoryJustificationComment": "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.", "partOneTherapeuticAreas": [{"id": 937245, "therapeuticArea": {"code": "5", "name": "Diseases [C] - Musculoskeletal Diseases [C05]"}}], "productRoleGroupInfos": [{"id": 289804, "comments": "DT-DEC01", "productRoleCode": "1", "productRoleName": "Test", "products": [{"id": 383128, "part1MpRoleTypeCode": "1", "productDictionaryInfo": {"productPk": "11689940", "productPharmForm": "SUSPENSION FOR INJECTION", "euMpNumber": "PRD11689940", "prodAuthStatus": 1, "prodName": "DT-DEC01", "pharmForm": "SUSPENSION FOR INJECTION", "activeSubstanceName": "EX VIVO FUSED NORMAL ALLOGENEIC HUMAN MYOBLAST WITH AUTOLOGOUS HUMAN MYOBLAST DERIVED FROM DUCHENNE MUSCULAR DYSTROPHY AFFECTED DONOR", "euSubstNumber": "SUB193214", "productOtherName": "MBN/MBDMD DEC", "nameOrg": "DYSTROGEN THERAPEUTICS TECHNOLOGY POLSKA SP. 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Subject's parent(s) or legal guardian(s) has (have) provided written informed consent, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to requirements (>16 years old) 2. Stated willingness to comply with all study procedures and availability for the duration of the study 3. Male, \u2265 8 years and <17 years of age at time of enrolment in the study 4. Ability to take oral medication and be willing to adhere to the study intervention regimen 5. 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Rodzice lub opiekunowie prawni uczestnika badania wyrazili pisemn\u0105 \u015bwiadom\u0105 zgod\u0119, w stosownych przypadkach, przed wykonaniem jakichkolwiek procedur zwi\u0105zanych z badaniem; uczestnicy zostan\u0105 poproszeni o wyra\u017cenie pisemnej lub ustnej zgody zgodnie z wymaganiami (> 16 lat).\n2. Deklarowana gotowo\u015b\u0107 do przestrzegania wszystkich procedur badawczych i dyspozycyjno\u015b\u0107 na czas trwania badania.\n3. M\u0119\u017cczyzna w wieku \u2265 8 lat i <17 lat w momencie w\u0142\u0105czenia do badania.\n4. Zdolno\u015b\u0107 do przyjmowania lek\u00f3w doustnych i ch\u0119\u0107 przestrzegania schematu interwencji w badaniu.\n5. U pacjenta potwierdzono rozpoznanie DMD, zdefiniowanej jako obraz kliniczny zgodny z typow\u0105 DMD oraz:\n\u2022 immunofluorescencja dystrofiny i / lub immunoblot wykazuj\u0105cy ca\u0142kowity niedob\u00f3r dystrofiny lub\n\u2022 mo\u017cliwa do zidentyfikowania mutacja w genie DMD (delecja / duplikacja jednego lub wi\u0119cej ekson\u00f3w), gdzie ramka odczytu mo\u017ce by\u0107 przewidziana jako \u201epoza ramk\u0105\u201d lub\n\u2022 pe\u0142ne sekwencjonowanie genu dystrofiny pokazuj\u0105ce zmian\u0119 (mutacja punktowa, duplikacja, inne), kt\u00f3ra ma uniemo\u017cliwi\u0107 produkcj\u0119 bia\u0142ka dystrofiny (tj. Nonsensowna mutacja, delecja / duplikacja prowadz\u0105ca do dalszego kodonu stop).\n6. Przyjmowanie ACEi w minimalnych wymaganych dawkach, jak przedstawiono w Tabeli, przez co najmniej 30 dni.", "language": 19, "languageDescription": "Polish"}]}], "principalExclusionCriteria": [{"id": 665304, "number": 1, "principalExclusionCriteria": "1. 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Klinicznie istotna bradykardia w spoczynku lub w EKG Holtera, na podstawie warto\u015bci prawid\u0142owych skorygowanych wzgl\u0119dem wieku i p\u0142ci, blok przedsionkowo-komorowy wy\u017cszy ni\u017c pierwszego stopnia w spoczynku lub drugiego stopnia Wenckebacha w nocy, przerwy d\u0142u\u017csze ni\u017c 2,5 sekundy.\n4. Obecno\u015b\u0107 rozrusznika serca lub ICD.\n5. Kliniczne oznaki lub objawy niewydolno\u015bci serca *.\n6. Frakcja wyrzutowa lewej komory * (LVEF) <57% (ocena za pomoc\u0105 echokardiografii Teichholtza).\n7. Brak mo\u017cliwo\u015bci uzyskania odpowiedniej jako\u015bci obraz\u00f3w echokardiograficznych (konieczne do monitorowania pierwszorz\u0119dowego punktu ko\u0144cowego i bezpiecze\u0144stwa).\n8. 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biztons\u00e1goss\u00e1g\u00e1nak \u00e9s hat\u00e1soss\u00e1g\u00e1nak \u00e9rt\u00e9kel\u00e9s\u00e9re, amelyet ny\u00edlt elrendez\u00e9s\u0171 d\u00f3ziseszkal\u00e1ci\u00f3 el\u0151z meg, Duchenne-f\u00e9le izomdystrophi\u00e1ban szenved\u0151, az 51-es exon \u00e1tugr\u00e1s\u00e1val kezelhet\u0151 del\u00e9ci\u00f3s mut\u00e1ci\u00f3val rendelkez\u0151 betegekn\u00e9l", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124225, "uuid": "fcf362f8-7ae6-416a-9044-204c4cbab511", "attributeTranslation": "Estudio aleatorizado, doble ciego, de b\u00fasqueda de dosis y comparaci\u00f3n de la seguridad y la eficacia de dosis altas de eteplirs\u00e9n, precedida de un aumento de la dosis en r\u00e9gimen abierto, en pacientes con distrofia muscular de Duchenne con mutaciones de deleci\u00f3n susceptibles de omisi\u00f3n del ex\u00f3n 51", "language": 7, "languageDescription": "Spanish"}, {"id": 10124224, "uuid": "fcf362f8-7ae6-416a-9044-204c4cbab511", "attributeTranslation": "\"\u039c\u03b9\u03b1 \u03a4\u03c5\u03c7\u03b1\u03b9\u03bf\u03c0\u03bf\u03b9\u03b7\u03bc\u03ad\u03bd\u03b7, \u0394\u03b9\u03c0\u03bb\u03ac \u03a4\u03c5\u03c6\u03bb\u03ae \u039c\u03b5\u03bb\u03ad\u03c4\u03b7 \n\u03a0\u03c1\u03bf\u03c3\u03b4\u03b9\u03bf\u03c1\u03b9\u03c3\u03bc\u03bf\u03cd \u03c4\u03b7\u03c2 \u0394\u03cc\u03c3\u03b7\u03c2 \u03ba\u03b1\u03b9 \u03a3\u03cd\u03b3\u03ba\u03c1\u03b9\u03c3\u03b7\u03c2 \u03c4\u03b7\u03c2 \n\u0391\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1\u03c2 \u03ba\u03b1\u03b9 \u0391\u03c0\u03bf\u03c4\u03b5\u03bb\u03b5\u03c3\u03bc\u03b1\u03c4\u03b9\u03ba\u03cc\u03c4\u03b7\u03c4\u03b1\u03c2 \u03a5\u03c8\u03b7\u03bb\u03ce\u03bd \u0394\u03cc\u03c3\u03b5\u03c9\u03bd\n\u03c4\u03bf\u03c5 Eteplirsen, \u03c4\u03c9\u03bd \u03bf\u03c0\u03bf\u03af\u03c9\u03bd \u03a0\u03c1\u03bf\u03b7\u03b3\u03b5\u03af\u03c4\u03b1\u03b9 \u03bc\u03b9\u03b1 \u039a\u03bb\u03b9\u03bc\u03ac\u03ba\u03c9\u03c3\u03b7 \n\u0394\u03cc\u03c3\u03b7\u03c2 \u0391\u03bd\u03bf\u03b9\u03ba\u03c4\u03ae\u03c2 \u0395\u03c0\u03b9\u03c3\u03ae\u03bc\u03b1\u03bd\u03c3\u03b7\u03c2, \u03c3\u03b5 \u0391\u03c3\u03b8\u03b5\u03bd\u03b5\u03af\u03c2 \u03bc\u03b5 \u039c\u03c5\u03ca\u03ba\u03ae \n\u0394\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03b1 Duchenne \u03bc\u03b5 \u039c\u03b5\u03c4\u03b1\u03bb\u03bb\u03ac\u03be\u03b5\u03b9\u03c2 \u0394\u03b9\u03b1\u03b3\u03c1\u03b1\u03c6\u03ae\u03c2 \u03c0\u03bf\u03c5 \n\u03b5\u03af\u03bd\u03b1\u03b9 \u0391\u03c0\u03bf\u03b4\u03b5\u03ba\u03c4\u03ad\u03c2 \u03b3\u03b9\u03b1 \u03a0\u03b1\u03c1\u03ac\u03bb\u03b5\u03b9\u03c8\u03b7 \u03c4\u03bf\u03c5 \u0395\u03be\u03bf\u03bd\u03af\u03bf\u03c5 51\"", "language": 32, "languageDescription": "Greek (Greece)"}, {"id": 10124228, "uuid": "fcf362f8-7ae6-416a-9044-204c4cbab511", "attributeTranslation": "Randomizowane badanie, prowadzone metod\u0105 podw\u00f3jnie \u015blepej pr\u00f3by, pozwalaj\u0105ce na ustalenie i por\u00f3wnanie dawek, oceniaj\u0105ce skuteczno\u015b\u0107 i bezpiecze\u0144stwo stosowania wysokich dawek Eteplirsenu, poprzedzone zwi\u0119kszaniem dawki w otwartej pr\u00f3bie, u pacjent\u00f3w z dystrofi\u0105 mi\u0119\u015bniow\u0105 Duchenne\u2019a z mutacjami typu delecje podatnymi na pomini\u0119cie egzonu 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 investigaci\u00f3n para comparar diferentes dosis de un nuevo medicamento en investigaci\u00f3n para el tratamiento de determinados pacientes con distrofia muscular de Duchenne", "language": 7, "languageDescription": "Spanish"}, {"id": 10124245, "uuid": "5d8daa20-b6cb-4f68-a8c4-937d5cb6984a", "attributeTranslation": "Kutat\u00e1si vizsg\u00e1lat egy \u00faj vizsg\u00e1lati k\u00e9sz\u00edtm\u00e9ny k\u00fcl\u00f6nb\u00f6z\u0151 d\u00f3zisainak \u00f6sszehasonl\u00edt\u00e1s\u00e1ra egyes Duchenne-izomdisztr\u00f3fi\u00e1s betegek kezel\u00e9s\u00e9re.", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124242, "uuid": "5d8daa20-b6cb-4f68-a8c4-937d5cb6984a", "attributeTranslation": "Badanie naukowe maj\u0105ce na celu por\u00f3wnanie r\u00f3\u017cnych dawek nowego badanego produktu leczniczego w leczeniu niekt\u00f3rych pacjent\u00f3w z dystrofi\u0105 mi\u0119\u015bniow\u0105 Duchenne\u2019a", "language": 19, "languageDescription": "Polish"}, {"id": 10124241, "uuid": "5d8daa20-b6cb-4f68-a8c4-937d5cb6984a", "attributeTranslation": "\u039c\u03af\u03b1 \u03b5\u03c1\u03b5\u03c5\u03bd\u03b7\u03c4\u03b9\u03ba\u03ae \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 \u03b3\u03b9\u03b1 \u03c4\u03b7 \u03c3\u03cd\u03b3\u03ba\u03c1\u03b9\u03c3\u03b7 \u03b4\u03b9\u03b1\u03c6\u03bf\u03c1\u03b5\u03c4\u03b9\u03ba\u03ce\u03bd \u03b4\u03cc\u03c3\u03b5\u03c9\u03bd \u03b5\u03bd\u03cc\u03c2 \u03bd\u03ad\u03bf\u03c5 \u03b5\u03c1\u03b5\u03c5\u03bd\u03b7\u03c4\u03b9\u03ba\u03bf\u03cd \u03c6\u03b1\u03c1\u03bc\u03b1\u03ba\u03b5\u03c5\u03c4\u03b9\u03ba\u03bf\u03cd \u03c0\u03c1\u03bf\u03ca\u03cc\u03bd\u03c4\u03bf\u03c2 \u03b3\u03b9\u03b1 \u03c4\u03b7 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1 \u03bf\u03c1\u03b9\u03c3\u03bc\u03ad\u03bd\u03c9\u03bd \u03b1\u03c3\u03b8\u03b5\u03bd\u03ce\u03bd \u03bc\u03b5 \u03bc\u03c5\u03ca\u03ba\u03ae \u03b4\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03b1 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 pati\u00ebnten met Duchenne spierdystrofie", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 10124246, "uuid": "5d8daa20-b6cb-4f68-a8c4-937d5cb6984a", "attributeTranslation": "\u00c9tude de recherche visant \u00e0 comparer diff\u00e9rentes doses d'un nouveau m\u00e9dicament exp\u00e9rimental 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": "\u039c\u03c5\u03ca\u03ba\u03ae \u0394\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03b1 Duchenne", "language": 5, "languageDescription": "Greek"}, {"id": 10124164, "uuid": "230b573c-4a66-41e3-980a-3321b9056151", "attributeTranslation": "Duchenne-f\u00e9le 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": "Dystrofi\u0105 mi\u0119\u015bniowa Duchenne\u2019a", "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 Part:\n\u2022 To investigate the effect of high doses of eteplirsen (100 mg/kg and 200 mg/kg) as compared with 30 mg/kg, administered weekly IV, on motor function in ambulant DMD patients with confirmed deletion genotypes amenable to Exon 51 skipping\n\u2022 To evaluate higher doses of eteplirsen (100mg/kg and 200 mg/kg) for dose comparison", "mainObjectiveTranslations": [{"id": 10124239, "uuid": "d30d817c-a7f5-4a81-a5dc-53203a1949b3", "attributeTranslation": "Augmentation de dose en ouvert\n\u00c9valuer l\u2019innocuit\u00e9 et la tol\u00e9rabilit\u00e9 de doses IV hebdomadaires de 100 et 200 mg/kg d\u2019eteplirsen\nPartie de d\u00e9termination et comparaison de doses en double aveugle\n\u2022\tExaminer l\u2019effet de doses \u00e9lev\u00e9es d\u2019eteplirsen (100 mg/kg et 200 mg/kg) par rapport \u00e0 une dose de 30 mg/kg, administr\u00e9e une fois par semaine par IV, sur la fonction motrice chez des patients ambulants atteints de dystrophie musculaire de Duchenne (DMD), pr\u00e9sentant des g\u00e9notypes avec d\u00e9l\u00e9tion confirm\u00e9e, susceptibles de r\u00e9pondre au saut de l\u2019exon 51\n\u2022\t\u00c9valuer des doses plus \u00e9lev\u00e9es d\u2019eteplirsen (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 r\u00e9gimen abierto:\n\u2022\tEvaluar la seguridad y la tolerabilidad de dosis IV semanales de 100 mg/kg y 200 mg/kg de eteplirs\u00e9n.\n\nParte en r\u00e9gimen doble ciego de b\u00fasqueda y comparaci\u00f3n de la dosis:\n\u2022\tInvestigar el efecto de dosis altas de eteplirs\u00e9n (100 mg/kg y 200 mg/kg) en comparaci\u00f3n con 30 mg/kg, administrada semanalmente por v\u00eda IV, sobre la funci\u00f3n motora en pacientes con distrofia muscular de Duchenne (DMD) ambulante con genotipos de deleci\u00f3n confirmados susceptibles de omisi\u00f3n del ex\u00f3n 51.\n\u2022\tEvaluar dosis m\u00e1s altas de eteplirs\u00e9n (100 mg/kg y 200 mg/kg) para comparar las dosis.", "language": 7, "languageDescription": "Spanish"}, {"id": 10124235, "uuid": "d30d817c-a7f5-4a81-a5dc-53203a1949b3", "attributeTranslation": "Ny\u00edlt elrendez\u00e9s\u0171 d\u00f3ziseszkal\u00e1ci\u00f3:\nAz eteplirszen heti 100 \u00e9s 200 mg/kg-os iv. adagja biztons\u00e1goss\u00e1g\u00e1nak \u00e9s toler\u00e1lhat\u00f3s\u00e1g\u00e1nak ki\u00e9rt\u00e9kel\u00e9se\nKett\u0151s-vak d\u00f3ziskeres\u0151 \u00e9s d\u00f3zis-\u00f6sszehasonl\u00edt\u00f3 r\u00e9sz:\n\u2022 Az eteplirszen nagy adagjainak (100 mg/kg vagy 200 mg/kg) a motoros funkci\u00f3ra kifejtett hat\u00e1s\u00e1nak tanulm\u00e1nyoz\u00e1sa a 30 mg/kg-os adaggal \u00f6sszehasonl\u00edtva, hetente egyszeri iv. adagol\u00e1s mellett, Duchenne-f\u00e9le izomdystrophi\u00e1ban (DMD) szenved\u0151, az 51-es exon \u00e1tugr\u00e1s\u00e1val potenci\u00e1lisan kezelhet\u0151 del\u00e9ci\u00f3val igazoltan rendelkez\u0151 betegekn\u00e9l\n\u2022 Az eteplirszen nagyobb adagjainak (100 mg/kg \u00e9s 200 mg/kg) d\u00f3zis-\u00f6sszehason\u00edt\u00f3 ki\u00e9rt\u00e9kel\u00e9se", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124238, "uuid": "d30d817c-a7f5-4a81-a5dc-53203a1949b3", "attributeTranslation": "Eskalacja dawki prowadzona metod\u0105 otwartej pr\u00f3by:\nOcena bezpiecze\u0144stwa i tolerancji cotygodniowych dawek do\u017cylnych 100 mg/kg mc. i 200 mg/kg mc. eteplirsenu \n\nCz\u0119\u015b\u0107 badania prowadzona metod\u0105 podw\u00f3jnie \u015blepej pr\u00f3by maj\u0105ca na celu ustalenie optymalnej dawki oraz por\u00f3wnanie dawek:\n\u2022Zbadanie wp\u0142ywu wysokich dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) w por\u00f3wnaniu z 30 mg/kg mc., podawanych co tydzie\u0144 do\u017cylnie, na funkcje motoryczne u chodz\u0105cych pacjent\u00f3w z dystrofi\u0105 mi\u0119\u015bniow\u0105 Duchenne\u2019a (DMD) z potwierdzonymi genotypami delecji podatnymi na pomini\u0119cie egzonu 51 \n\u2022 Ocena wysokich dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) w celu ich por\u00f3wnania", "language": 19, "languageDescription": "Polish"}, {"id": 10124237, "uuid": "d30d817c-a7f5-4a81-a5dc-53203a1949b3", "attributeTranslation": "\u039a\u03bb\u03b9\u03bc\u03ac\u03ba\u03c9\u03c3\u03b7\u03c2 \u03b4\u03cc\u03c3\u03b7\u03c2 \u03b1\u03bd\u03bf\u03b9\u03ba\u03c4\u03ae\u03c2 \u03b5\u03c0\u03b9\u03c3\u03ae\u03bc\u03b1\u03bd\u03c3\u03b7:\n\u0391\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1\u03c2 \u03ba\u03b1\u03b9 \u03b1\u03bd\u03b5\u03ba\u03c4\u03b9\u03ba\u03cc\u03c4\u03b7\u03c4\u03b1\u03c2 \u03c4\u03c9\u03bd \u03b5\u03b2\u03b4\u03bf\u03bc\u03b1\u03b4\u03b9\u03b1\u03af\u03c9\u03bd \u0395\u03a6 \u03b4\u03cc\u03c3\u03b5\u03c9\u03bd 100 \u03ba\u03b1\u03b9 200 mg/kg \u03c4\u03bf\u03c5 eteplirsen\n\n\u0394\u03b9\u03c0\u03bb\u03ac \u03c4\u03c5\u03c6\u03bb\u03cc \u03bc\u03ad\u03c1\u03bf\u03c2 \u03c0\u03c1\u03bf\u03c3\u03b4\u03b9\u03bf\u03c1\u03b9\u03c3\u03bc\u03bf\u03cd \u03c4\u03b7\u03c2 \u03b4\u03cc\u03c3\u03b7\u03c2 \u03ba\u03b1\u03b9 \u03c3\u03cd\u03b3\u03ba\u03c1\u03b9\u03c3\u03b7\u03c2 \u03c4\u03b7\u03c2 \u03b4\u03cc\u03c3\u03b7\u03c2:\n\u2022\u0394\u03b9\u03b5\u03c1\u03b5\u03cd\u03bd\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03b5\u03c0\u03af\u03b4\u03c1\u03b1\u03c3\u03b7\u03c2 \u03c5\u03c8\u03b7\u03bb\u03ce\u03bd \u03b4\u03cc\u03c3\u03b5\u03c9\u03bd \u03c4\u03bf\u03c5 eteplirsen (100 mg/kg \u03ba\u03b1\u03b9 200 mg/kg) \u03c3\u03b5 \u03c3\u03cd\u03b3\u03ba\u03c1\u03b9\u03c3\u03b7 \u03bc\u03b5 30 mg/kg, \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03b7\u03c2 \u03b5\u03b2\u03b4\u03bf\u03bc\u03b1\u03b4\u03b9\u03b1\u03af\u03c9\u03c2 \u0395\u03a6, \u03c3\u03c4\u03b7\u03bd \u03ba\u03b9\u03bd\u03b7\u03c4\u03b9\u03ba\u03ae \u03bb\u03b5\u03b9\u03c4\u03bf\u03c5\u03c1\u03b3\u03af\u03b1 \u03c3\u03b5 \u03c0\u03b5\u03c1\u03b9\u03c0\u03b1\u03c4\u03b7\u03c4\u03b9\u03ba\u03bf\u03cd\u03c2 \u03b1\u03c3\u03b8\u03b5\u03bd\u03b5\u03af\u03c2 \u03bc\u03b5 DMD \u03bc\u03b5 \u03b5\u03c0\u03b9\u03b2\u03b5\u03b2\u03b1\u03b9\u03c9\u03bc\u03ad\u03bd\u03bf\u03c5\u03c2 \u03b3\u03bf\u03bd\u03cc\u03c4\u03c5\u03c0\u03bf\u03c5\u03c2 \u03b4\u03b9\u03b1\u03b3\u03c1\u03b1\u03c6\u03ae\u03c2 \u03c0\u03bf\u03c5 \u03b5\u03af\u03bd\u03b1\u03b9 \u03b1\u03c0\u03bf\u03b4\u03b5\u03ba\u03c4\u03bf\u03af \u03b3\u03b9\u03b1 \u03c0\u03b1\u03c1\u03ac\u03bb\u03b5\u03b9\u03c8\u03b7 \u03c4\u03bf\u03c5 \u0395\u03be\u03bf\u03bd\u03af\u03bf\u03c5 51\n\u2022\u0391\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c5\u03c8\u03b7\u03bb\u03cc\u03c4\u03b5\u03c1\u03c9\u03bd \u03b4\u03cc\u03c3\u03b5\u03c9\u03bd \u03c4\u03bf\u03c5 eteplirsen (100 mg/kg \u03ba\u03b1\u03b9 200 mg/kg) \u03b3\u03b9\u03b1 \u03c3\u03cd\u03b3\u03ba\u03c1\u03b9\u03c3\u03b7 \u03c4\u03c9\u03bd \u03b4\u03cc\u03c3\u03b5\u03c9\u03bd", "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\u2022 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 pati\u00ebnten met Duchenne spierdystrofie (DMD) met bevestigde deletiegenotypes die vatbaar zijn voor exon 51-skipping\n\u2022 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 : \u2022 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 \u2022 To evaluate the PK and PD of doses (100mg/kg and 200mg/kg) higher than 30 mg/kg of eteplirsen \u2022 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": "Cz\u0119\u015b\u0107 badania prowadzona metod\u0105 podw\u00f3jnie \u015blepej pr\u00f3by maj\u0105ca na celu ustalenie optymalnej dawki oraz por\u00f3wnanie dawek:\n\u2022Ocena wp\u0142ywu wysokich dawek eteplirsenu w por\u00f3wnaniu z dawk\u0105 30 mg/kg mc., podawanej do\u017cylnie raz na tydzie\u0144 na:  \no sprawno\u015b\u0107 poruszania si\u0119 \no czynno\u015b\u0107 p\u0142uc \no ekspresj\u0119 dystrofiny \n\u2022Ocena farmakokinetyki (PK) i farmakodynamiki (PD) dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) wy\u017cszych ni\u017c 30 mg/kg mc.  \n\u2022Ocena bezpiecze\u0144stwa i tolerancji dawek eteplirsenu (100 mg/kg mc. oraz 200 mg/kg mc.) wy\u017cszych ni\u017c 30 mg/kg mc. podawanych do\u017cylnie raz na tydzie\u0144", "language": 19, "languageDescription": "Polish"}, {"id": 10124248, "uuid": "bb3293b1-c950-424d-b0c5-68fff941e457", "attributeTranslation": "Parte en r\u00e9gimen doble ciego de b\u00fasqueda y comparaci\u00f3n de la dosis:\n\u2022\tEvaluar el efecto de dosis altas en comparaci\u00f3n con 30 mg/kg de eteplirs\u00e9n, administrada por v\u00eda IV una vez a la semana, sobre los siguientes par\u00e1metros: \no\tRendimiento ambulatorio\no\tFunci\u00f3n pulmonar\no\tExpresi\u00f3n de distrofina\n\u2022\tEvaluar la FC y la FD de dosis superiores a 30 mg/kg (100 mg/kg y 200 mg/kg) de eteplirs\u00e9n\nEvaluar la seguridad y la tolerabilidad de dosis superiores a 30 mg/kg (100 mg/kg y 200 mg/kg) de eteplirs\u00e9n administradas semanalmente por v\u00eda IV", "language": 7, "languageDescription": "Spanish"}, {"id": 10124249, "uuid": "bb3293b1-c950-424d-b0c5-68fff941e457", "attributeTranslation": "Partie de d\u00e9termination et comparaison de doses en double aveugle\n\u2022 \t\u00c9valuer l\u2019effet de doses \u00e9lev\u00e9es par rapport \u00e0 une dose IV hebdomadaire de 30 mg/kg d\u2019eteplirsen sur les crit\u00e8res suivants:\no\tPerformances ambulatoires\no\tFonction pulmonaire\no\tExpression de la dystrophine\n\u2022 \t\u00c9valuer la PK et la PD de doses d\u2019eteplirsen (100 mg/kg et 200 mg/kg)sup\u00e9rieures \u00e0 30 mg/kg\n\u2022\t\u00c9valuer l\u2019innocuit\u00e9 et la tol\u00e9rabilit\u00e9 de doses d\u2019eteplirsen (100 mg/kg et 200 mg/kg) sup\u00e9rieures \u00e0 30 mg/kg administr\u00e9es par IV une fois par semaine", "language": 34, "languageDescription": "French (France)"}, {"id": 10124250, "uuid": "bb3293b1-c950-424d-b0c5-68fff941e457", "attributeTranslation": "Kett\u0151s-vak d\u00f3ziskeres\u0151 \u00e9s d\u00f3zis-\u00f6sszehasonl\u00edt\u00f3 r\u00e9sz\n\u2022\tAz eteplirszen heti egyszer iv. alkalmazott nagy adagja \u00e1ltal a k\u00f6vetkez\u0151kre kifejtett hat\u00e1s ki\u00e9rt\u00e9kel\u00e9se, a 30 mg/kg-os adaggal \u00f6sszehasonl\u00edtva:\no\tj\u00e1r\u00e1s-teljes\u00edt\u0151k\u00e9pess\u00e9g\no\tl\u00e9gz\u00e9sfunkci\u00f3\no\tdisztrofinexpresszi\u00f3\n\u2022\tAz eteplirszen 30 mg/kg-n\u00e1l nagyobb adagjai (100 mg/kg \u00e9s 200 mg/kg) farmakokinetik\u00e1j\u00e1nak \u00e9s farmakodinamik\u00e1j\u00e1nak elemz\u00e9se.\n\u2022\tAz eteplirszen 30 mg/kg-n\u00e1l nagyobb (100 mg/kg vagy 200 mg/kg), heti egyszeri iv. adagjai biztons\u00e1goss\u00e1g\u00e1nak \u00e9s toler\u00e1lhat\u00f3s\u00e1g\u00e1nak ki\u00e9rt\u00e9kel\u00e9se", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124252, "uuid": "bb3293b1-c950-424d-b0c5-68fff941e457", "attributeTranslation": "Dubbelblind deel voor dosisbepaling en dosisvergelijking:\n\u2022 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\u2022 Het beoordelen van de farmacokinetiek en farmacodynamiek van doses (100 mg/kg en 200 mg/kg) hoger dan 30 mg/kg eteplirsen\n\u2022 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": "\u0394\u03b9\u03c0\u03bb\u03ac \u03c4\u03c5\u03c6\u03bb\u03cc \u03bc\u03ad\u03c1\u03bf\u03c2 \u03c0\u03c1\u03bf\u03c3\u03b4\u03b9\u03bf\u03c1\u03b9\u03c3\u03bc\u03bf\u03cd \u03c4\u03b7\u03c2 \u03b4\u03cc\u03c3\u03b7\u03c2 \u03ba\u03b1\u03b9 \u03c3\u03cd\u03b3\u03ba\u03c1\u03b9\u03c3\u03b7\u03c2 \u03c4\u03b7\u03c2 \u03b4\u03cc\u03c3\u03b7\u03c2:\n\u2022\u0391\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03b5\u03c0\u03af\u03b4\u03c1\u03b1\u03c3\u03b7\u03c2 \u03c4\u03c9\u03bd \u03c5\u03c8\u03b7\u03bb\u03ce\u03bd \u03b4\u03cc\u03c3\u03b5\u03c9\u03bd \u03c3\u03b5 \u03c3\u03cd\u03b3\u03ba\u03c1\u03b9\u03c3\u03b7 \u03bc\u03b5 30 mg/kg eteplirsen, \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03bf\u03c5 \u03b5\u03b2\u03b4\u03bf\u03bc\u03b1\u03b4\u03b9\u03b1\u03af\u03c9\u03c2 \u0395\u03a6, \u03c9\u03c2 \u03c0\u03c1\u03bf\u03c2 \u03c4\u03b1 \u03b5\u03be\u03ae\u03c2:\no\u03a0\u03b5\u03c1\u03b9\u03c0\u03b1\u03c4\u03b7\u03c4\u03b9\u03ba\u03ae \u03b1\u03c0\u03cc\u03b4\u03bf\u03c3\u03b7\no\u03a0\u03bd\u03b5\u03c5\u03bc\u03bf\u03bd\u03b9\u03ba\u03ae \u03bb\u03b5\u03b9\u03c4\u03bf\u03c5\u03c1\u03b3\u03af\u03b1\no\u0388\u03ba\u03c6\u03c1\u03b1\u03c3\u03b7 \u03b4\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03bd\u03b7\u03c2\n\u2022\u0391\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03a6\u039a \u03ba\u03b1\u03b9 \u03a6\u0394 \u03b4\u03cc\u03c3\u03b5\u03c9\u03bd (100 mg/kg \u03ba\u03b1\u03b9 200 mg/kg) \u03c4\u03bf\u03c5 eteplirsen \u03c0\u03bf\u03c5 \u03c5\u03c0\u03b5\u03c1\u03b2\u03b1\u03af\u03bd\u03bf\u03c5\u03bd \u03c4\u03b1 30 mg/kg\n\u2022\u0391\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1\u03c2 \u03ba\u03b1\u03b9 \u03c4\u03b7\u03c2 \u03b1\u03bd\u03b5\u03ba\u03c4\u03b9\u03ba\u03cc\u03c4\u03b7\u03c4\u03b1\u03c2 \u03b4\u03cc\u03c3\u03b5\u03c9\u03bd (100 mg/kg \u03ba\u03b1\u03b9 200 mg/kg) \u03c4\u03bf\u03c5 eteplirsen \u03c0\u03bf\u03c5 \u03c5\u03c0\u03b5\u03c1\u03b2\u03b1\u03af\u03bd\u03bf\u03c5\u03bd \u03c4\u03b1 30 mg/kg, \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03c9\u03bd \u0395\u03a6 \u03b5\u03b2\u03b4\u03bf\u03bc\u03b1\u03b4\u03b9\u03b1\u03af\u03c9\u03c2", "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 \u226550% 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.\tFi\u00faknak kell lenni\u00fck, rendelkezni\u00fck kell a DMD klinikai diagn\u00f3zis\u00e1val \u00e9s a DMD g\u00e9n 51-es exon \u00e1tugr\u00e1s\u00e1val kezelhet\u0151 (p\u00e9ld\u00e1ul a 45-50, 47-50, 48-50, 49-50, 50, 52 \u00e9s 52-63 exonok del\u00e9ci\u00f3ja), olvas\u00e1si kereten k\u00edv\u00fcli (out of frame) del\u00e9ci\u00f3s mut\u00e1ci\u00f3j\u00e1val;\n2.\t4 \u00e9s 13 \u00e9ves kor k\u00f6z\u00f6tt kell lenni\u00fck\n3.\tJ\u00e1r\u00f3k\u00e9pesnek kell lenni\u00fck, k\u00e9pesnek kell lenni\u00fck \u226410 m\u00e1sodperc alatt teljes\u00edteni a talajr\u00f3l val\u00f3 fel\u00e1ll\u00e1s tesztet\n4.\tK\u00e9pes \u00f6n\u00e1ll\u00f3an, seg\u00e9deszk\u00f6z\u00f6k n\u00e9lk\u00fcl j\u00e1rni.\n5.\t\u00c9p bicepsz vagy alternat\u00edv felkar izomcsoporttal kell rendelkezni\u00fck, amely lehet\u0151v\u00e9 teszi 1 cm 3-es izombiopszia v\u00e9tel\u00e9t a kezel\u00e9s el\u0151tt \u00e9s alatt (a vizsg\u00e1lat kett\u0151s vak r\u00e9sz\u00e9ben szerepl\u0151 betegek eset\u00e9ben)\n6.\tA randomiz\u00e1l\u00e1st megel\u0151z\u0151en legal\u00e1bb 12 h\u00e9tig stabil d\u00f3zisban vagy egyen\u00e9rt\u00e9k\u0171 d\u00f3zisban r\u00e9szes\u00fcltek or\u00e1lis kortikoszteroid-kezel\u00e9sben \u00e9s az adag v\u00e1rhat\u00f3an \u00e1lland\u00f3 marad (kiv\u00e9ve a nemr\u00e9giben k\u00f6zz\u00e9tett ir\u00e1nyelvek [Birnkrant 2018, Kinnett 2017] szerinti, a tests\u00falyv\u00e1ltoz\u00e1shoz \u00e9s a stresszel kapcsolatos sz\u00fcks\u00e9gletekhez val\u00f3 alkalmazkod\u00e1shoz sz\u00fcks\u00e9ges m\u00f3dos\u00edt\u00e1sokat) a vizsg\u00e1lat sor\u00e1n).\n7.\t7 \u00e9ves \u00e9s id\u0151sebb kor\u00faak eset\u00e9n stabil t\u00fcd\u0151funkci\u00f3val kell rendelkezni\u00fck (FVC \u2265 a becs\u00fclt \u00e9rt\u00e9k 50%-\u00e1n\u00e1l \u00e9s nem ig\u00e9nyelnek \u00e9jszakai l\u00e9legeztet\u00e9st), ami a vizsg\u00e1l\u00f3 v\u00e9lem\u00e9nye szerint nem val\u00f3sz\u00edn\u0171, hogy jelent\u0151sen dekompenz\u00e1l\u00f3dik a vizsg\u00e1lat id\u0151tartama alatt. A 4-6 \u00e9ves koroszt\u00e1ly eset\u00e9ben a sz\u0171r\u00e9s id\u0151pontj\u00e1ban nem ig\u00e9nyelnek l\u00e9gz\u00e9st\u00e1mogat\u00e1st l\u00e9legeztet\u0151g\u00e9p vagy non-invaz\u00edv l\u00e9legeztet\u00e9s form\u00e1j\u00e1ban.\n8.\tHa szexu\u00e1lisan akt\u00edv, v\u00e1llalja, hogy a vizsg\u00e1lat teljes id\u0151tartama alatt \u00e9s az utols\u00f3 adagot k\u00f6vet\u0151 90 napig f\u00e9rfi \u00f3vszert haszn\u00e1l. A szexu\u00e1lis partnernek is orvosilag elfogadhat\u00f3 fogamz\u00e1sg\u00e1tl\u00f3t (pl. n\u0151i or\u00e1lis fogamz\u00e1sg\u00e1tl\u00f3t) kell haszn\u00e1lnia ezen id\u0151szak alatt. Az elfogadhat\u00f3 fogamz\u00e1sg\u00e1tl\u00f3 m\u00f3dszerek k\u00f6z\u00e9 tartozik a kombin\u00e1lt (\u00f6sztrog\u00e9n- \u00e9s progeszterontartalm\u00fa) hormon\u00e1lis fogamz\u00e1sg\u00e1tl\u00e1s, amely az ovul\u00e1ci\u00f3 g\u00e1tl\u00e1s\u00e1val j\u00e1r (or\u00e1lis, intravagin\u00e1lis vagy transzderm\u00e1lis); csak progeszteront tartalmaz\u00f3 hormon\u00e1lis fogamz\u00e1sg\u00e1tl\u00e1s, amely az ovul\u00e1ci\u00f3 g\u00e1tl\u00e1s\u00e1val j\u00e1r (or\u00e1lis, injekci\u00f3s vagy be\u00fcltethet\u0151); m\u00e9hen bel\u00fcli eszk\u00f6z; m\u00e9hen bel\u00fcli hormont felszabad\u00edt\u00f3 rendszer; k\u00e9toldali petevezet\u00e9k-elz\u00e1r\u00e1s; vasektomiz\u00e1lt partner; szexu\u00e1lis absztinencia (True abstinence: Ha ez \u00f6sszhangban van a beteg prefer\u00e1lt \u00e9s szok\u00e1sos \u00e9letm\u00f3dj\u00e1val. Az id\u0151szakos absztinencia [mint p\u00e9ld\u00e1ul a napt\u00e1ri, ovul\u00e1ci\u00f3s, t\u00fcneti, ovul\u00e1ci\u00f3 ut\u00e1ni m\u00f3dszerek] \u00e9s a megvon\u00e1s nem elfogadhat\u00f3 fogamz\u00e1sg\u00e1tl\u00f3 m\u00f3dszerek); vagy \u00f3vszer kombin\u00e1lva sapk\u00e1val, pessz\u00e1riummal vagy spermiciddel ell\u00e1tott szivaccsal (kett\u0151s g\u00e1tas fogamz\u00e1sg\u00e1tl\u00e1s).\n9.\tVan(nak) sz\u00fcl\u0151(k) vagy t\u00f6rv\u00e9nyes gy\u00e1m(ok), aki(k) k\u00e9pes(ek) meg\u00e9rteni \u00e9s betartani a vizsg\u00e1lat \u00f6sszes k\u00f6vetelm\u00e9ny\u00e9t.\n10.\tHajland\u00f3 t\u00e1j\u00e9kozott beleegyez\u00e9st adni (ha alkalmazhat\u00f3), \u00e9s rendelkezik (egy) sz\u00fcl\u0151vel (sz\u00fcl\u0151kkel) vagy t\u00f6rv\u00e9nyes gy\u00e1mmal (gy\u00e1mokkal), aki (akik) hajland\u00f3(ak) t\u00e1j\u00e9kozott beleegyez\u00e9st adni a betegnek a vizsg\u00e1latban val\u00f3 r\u00e9szv\u00e9tel\u00e9hez.", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124229, "uuid": "7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0", "attributeTranslation": "1. M\u0119\u017cczyzna z ustalonym rozpoznaniem klinicznym dystrofii mi\u0119\u015bniowej Duchenne\u2019a (DMD) i mutacj\u0105 delecyjn\u0105 w genie DMD z zaburzeniem ramki odczytu (ang. out-of-frame), w przypadku kt\u00f3rej mo\u017cliwe jest pomini\u0119cie egzonu 51 (na przyk\u0142ad delecje egzon\u00f3w 45\u201350, 47\u201350, 48\u201350, 49\u201350, 50, 52 i 52\u201363).\n2. Wiek od 4 do 13 lat w\u0142\u0105cznie.\n3. Pacjent chodz\u0105cy, zdolny do wykonania testu TTRISE (tj. czas do wstania) w czasie maksymalnie 10 sekund w momencie wizyty przesiewowej.\n4. Zdolno\u015b\u0107 samodzielnego chodzenia bez urz\u0105dze\u0144 wspomagaj\u0105cych.\n5. Bez zmian dotycz\u0105cych prawego i lewego mi\u0119\u015bnia dwug\u0142owego (preferowane miejsce biopsji) lub alternatywnej grupy mi\u0119\u015bni ramienia, co umo\u017cliwi pobranie biopsji mi\u0119\u015bni o wystarczaj\u0105cej obj\u0119to\u015bci (1 cm3) przed leczeniem i w jego trakcie (dotyczy pacjent\u00f3w uczestnicz\u0105cych w cz\u0119\u015bci badania prowadzonej metod\u0105 podw\u00f3jnie \u015blepej pr\u00f3by).\n6. Przyjmowanie sta\u0142ej dawki lub dawki r\u00f3wnowa\u017cnej doustnych kortykosteroid\u00f3w przez co najmniej 12 tygodni przed randomizacj\u0105, przy czym oczekuje si\u0119, \u017ce dawka pozostanie sta\u0142a w trakcie ca\u0142ego badania (z wyj\u0105tkiem modyfikacji zwi\u0105zanych ze zmian\u0105 masy cia\u0142a i potrzebami zwi\u0105zanymi ze stresem, zgodnie z niedawno opublikowanymi wytycznymi [Birnkrant 2018, Kinnett 2017]).\n7. Pacjenci w wieku 7 lat i starsi \u2013 stabilna czynno\u015b\u0107 p\u0142uc (wymuszona pojemno\u015b\u0107 \u017cyciowa \u226550% przewidywanej warto\u015bci nale\u017cnej i brak konieczno\u015bci wspomagania oddychania w nocy), z niewielkim prawdopodobie\u0144stwem istotnej dekompensacji w czasie trwania badania w opinii badacza.\nPacjenci w wieku od 4 do 6 lat \u2013 brak konieczno\u015bci wspomagania oddychania respiratorem ani stosowania wentylacji nieinwazyjnej w czasie badania przesiewowego.\n8. Pacjenci aktywni seksualnie \u2013 zgoda na stosowanie prezerwatywy m\u0119skiej podczas takiej aktywno\u015bci przez ca\u0142y czas trwania badania oraz przez 90 dni od przyj\u0119cia ostatniej dawki. W tym okresie partnerka seksualna musi r\u00f3wnie\u017c stosowa\u0107 medycznie akceptowaln\u0105 metod\u0119 antykoncepcji (tj. doustne \u015brodki antykoncepcyjne dla kobiet). Dopuszczalne metody antykoncepcji obejmuj\u0105 z\u0142o\u017con\u0105 (zawieraj\u0105c\u0105 estrogen i progesteron) antykoncepcj\u0119 hormonaln\u0105 zwi\u0105zan\u0105 z hamowaniem owulacji (w postaci doustnej, dopochwowej lub przezsk\u00f3rnej), antykoncepcj\u0119 hormonaln\u0105 zawieraj\u0105c\u0105 sam progesteron zwi\u0105zan\u0105 z hamowaniem owulacji (w postaci doustnej, we wstrzykni\u0119ciach lub w implantach), wk\u0142adk\u0119 wewn\u0105trzmaciczn\u0105, system wewn\u0105trzmaciczny uwalniaj\u0105cy hormony, obustronne zamkni\u0119cie jajowod\u00f3w, partnera po wazektomii, abstynencj\u0119 seksualn\u0105 (prawdziwa abstynencja: je\u017celi jest zgodna z preferowanym i typowym stylem \u017cycia pacjenta. Okresowa abstynencja [taka jak metody kalendarzykowe, owulacyjne, objawowo-termiczne, poowulacyjne] i stosunek przerywany nie s\u0105 akceptowalnymi metodami antykoncepcji) lub prezerwatyw\u0119 w po\u0142\u0105czeniu z kapturkiem antykoncepcyjnym, diafragm\u0105 lub g\u0105bk\u0105 ze \u015brodkiem plemnikob\u00f3jczym (podw\u00f3jna metoda barierowa).\n9. Dost\u0119pno\u015b\u0107 rodzica/rodzic\u00f3w lub opiekuna prawnego/opiekun\u00f3w prawnych, kt\u00f3rzy s\u0105 w stanie zrozumie\u0107 wszystkie wymogi zwi\u0105zane z badaniem i ich przestrzega\u0107.\n10. Ch\u0119\u0107 wyra\u017cenia \u015bwiadomej zgody osoby ma\u0142oletniej (je\u015bli ma to zastosowanie) i dost\u0119pno\u015b\u0107 rodzica/rodzic\u00f3w lub opiekuna prawnego/opiekun\u00f3w prawnych, kt\u00f3rzy s\u0105 sk\u0142onni wyrazi\u0107 \u015bwiadom\u0105 zgod\u0119 na udzia\u0142 pacjenta w badaniu.", "language": 19, "languageDescription": "Polish"}, {"id": 10124233, "uuid": "7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0", "attributeTranslation": "1.\tDebe ser un var\u00f3n, con un diagn\u00f3stico cl\u00ednico establecido de DMD y una mutaci\u00f3n por deleci\u00f3n fuera del marco del gen DMD susceptible de omisi\u00f3n del ex\u00f3n 51.\n2.\tTener ente 4 y 13 a\u00f1os, ambos incluidos.\n3.\tSer capaz de levantarse del suelo en \u226410 segundos o menos, en el momento de la visita de secci\u00f3n\n4.\t Ser capaz de caminar de forma independiente sin dispositivos de ayuda.\n5.\tTener intactos los b\u00edceps 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 m\u00ednimo de 12 semanas antes de la aleatorizaci\u00f3n; \n7.\tSi tiene \u22657 a\u00f1os de edad, presentar una funci\u00f3n pulmonar estable (CVF \u226550 % del valor te\u00f3rico y sin necesidad de ventilaci\u00f3n nocturna) que, en opini\u00f3n del investigador, es improbable que se descompense significativamente durante el estudio y, en el caso de pacientes de 4 a 6 a\u00f1os de edad, no requerir apoyo con respirador ni ventilaci\u00f3n no invasiva en el momento de la selecci\u00f3n.\n8.\tSi es sexualmente activo, aceptar el uso del preservativo masculino durante dicha actividad, durante toda la duraci\u00f3n del estudio y durante 90 d\u00edas despu\u00e9s de la \u00faltima dosis. La pareja sexual tambi\u00e9n debe usar una forma de anticoncepci\u00f3n m\u00e9dicamente aceptable (es decir, anticonceptivos orales femeninos) durante este per\u00edodo de tiempo. Los m\u00e9todos anticonceptivos aceptables incluyen la anticoncepci\u00f3n hormonal combinada (que contiene estr\u00f3geno y progesterona) asociada con la inhibici\u00f3n de la ovulaci\u00f3n (oral, intravaginal o transd\u00e9rmica); anticoncepci\u00f3n hormonal solo con progesterona asociada con la inhibici\u00f3n de la ovulaci\u00f3n (oral, inyectable o implantable); dispositivo intrauterino; sistema de liberaci\u00f3n de hormonas intrauterinas; oclusi\u00f3n tub\u00e1rica bilateral; pareja vasectomizada; abstinencia sexual (abstinencia verdadera: cuando esto est\u00e1 en l\u00ednea con el estilo de vida preferido y habitual del paciente. La abstinencia peri\u00f3dica [como el calendario, la ovulaci\u00f3n, los m\u00e9todos sintot\u00e9rmicos, los m\u00e9todos postovulatorios] y el coito interrumpido no son m\u00e9todos anticonceptivos aceptables). o cond\u00f3n en combinaci\u00f3n con capuch\u00f3n, diafragma o esponja con espermicida (anticoncepci\u00f3n de doble barrera).\n9.\tDebe tener uno o m\u00e1s 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 m\u00e1s padres o tutores legales que est\u00e9n 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. \u039d\u03b1 \u03b5\u03af\u03bd\u03b1\u03b9 \u03ac\u03bd\u03b4\u03c1\u03b1\u03c2 \u03bc\u03b5 \u03c4\u03b5\u03ba\u03bc\u03b7\u03c1\u03b9\u03c9\u03bc\u03ad\u03bd\u03b7 \u03ba\u03bb\u03b9\u03bd\u03b9\u03ba\u03ae \u03b4\u03b9\u03ac\u03b3\u03bd\u03c9\u03c3\u03b7 DMD \u03ba\u03b1\u03b9 \u03bc\u03b5\u03c4\u03ac\u03bb\u03bb\u03b1\u03be\u03b7 \u03b4\u03b9\u03b1\u03b3\u03c1\u03b1\u03c6\u03ae\u03c2 \u03b5\u03ba\u03c4\u03cc\u03c2 \u03c0\u03bb\u03b1\u03b9\u03c3\u03af\u03bf\u03c5 \u03c4\u03bf\u03c5 \u03b3\u03bf\u03bd\u03b9\u03b4\u03af\u03bf\u03c5 \u03c4\u03b7\u03c2 DMD \u03c0\u03bf\u03c5 \u03b5\u03af\u03bd\u03b1\u03b9 \u03b1\u03c0\u03bf\u03b4\u03b5\u03ba\u03c4\u03cc \u03b3\u03b9\u03b1 \u03c0\u03b1\u03c1\u03ac\u03bb\u03b5\u03b9\u03c8\u03b7 \u03c4\u03bf\u03c5 \u0395\u03be\u03bf\u03bd\u03af\u03bf\u03c5 51 (\u03b3\u03b9\u03b1 \u03c0\u03b1\u03c1\u03ac\u03b4\u03b5\u03b9\u03b3\u03bc\u03b1, \u03b4\u03b9\u03b1\u03b3\u03c1\u03b1\u03c6\u03ad\u03c2 \u03c4\u03c9\u03bd \u0395\u03be\u03bf\u03bd\u03af\u03c9\u03bd 45-50, 47-50, 48-50, 49-50, 50, 52 \u03ba\u03b1\u03b9 52-63).\n2. \u039d\u03b1 \u03b5\u03af\u03bd\u03b1\u03b9 \u03b7\u03bb\u03b9\u03ba\u03af\u03b1\u03c2 4 \u03ad\u03c9\u03c2 13 \u03b5\u03c4\u03ce\u03bd.\n3. \u039d\u03b1 \u03b5\u03af\u03bd\u03b1\u03b9 \u03c0\u03b5\u03c1\u03b9\u03c0\u03b1\u03c4\u03b7\u03c4\u03b9\u03ba\u03cc\u03c2 \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2, \u03b9\u03ba\u03b1\u03bd\u03cc\u03c2 \u03bd\u03b1 \u03c0\u03b5\u03c4\u03cd\u03c7\u03b5\u03b9 TTRISE \u03c3\u03b5 10 \u03b4\u03b5\u03c5\u03c4\u03b5\u03c1\u03cc\u03bb\u03b5\u03c0\u03c4\u03b1 \u03ae \u03bb\u03b9\u03b3\u03cc\u03c4\u03b5\u03c1\u03bf \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7\u03bd \u03b5\u03c0\u03af\u03c3\u03ba\u03b5\u03c8\u03b7 \u03b4\u03b9\u03b1\u03bb\u03bf\u03b3\u03ae\u03c2.\n4. \u039d\u03b1 \u03ad\u03c7\u03b5\u03b9 \u03b4\u03c5\u03bd\u03b1\u03c4\u03cc\u03c4\u03b7\u03c4\u03b1 \u03b1\u03bd\u03b5\u03be\u03ac\u03c1\u03c4\u03b7\u03c4\u03b7\u03c2 \u03b2\u03ac\u03b4\u03b9\u03c3\u03b7\u03c2 \u03c7\u03c9\u03c1\u03af\u03c2 \u03b2\u03bf\u03b7\u03b8\u03b7\u03c4\u03b9\u03ba\u03ad\u03c2 \u03c3\u03c5\u03c3\u03ba\u03b5\u03c5\u03ad\u03c2.\n5. \u039d\u03b1 \u03ad\u03c7\u03b5\u03b9 \u03ac\u03b8\u03b9\u03ba\u03c4\u03bf \u03b4\u03b5\u03be\u03b9\u03cc \u03ba\u03b1\u03b9 \u03b1\u03c1\u03b9\u03c3\u03c4\u03b5\u03c1\u03cc \u03b4\u03b9\u03ba\u03ad\u03c6\u03b1\u03bb\u03bf \u03bc\u03c5 (\u03c4\u03bf \u03c0\u03c1\u03bf\u03c4\u03b9\u03bc\u03ce\u03bc\u03b5\u03bd\u03bf \u03c3\u03b7\u03bc\u03b5\u03af\u03bf \u03b2\u03b9\u03bf\u03c8\u03af\u03b1\u03c2) \u03ae \u03bc\u03b9\u03b1 \u03b5\u03bd\u03b1\u03bb\u03bb\u03b1\u03ba\u03c4\u03b9\u03ba\u03ae \u03bf\u03bc\u03ac\u03b4\u03b1 \u03bc\u03c5\u03ce\u03bd \u03c4\u03bf\u03c5 \u03ac\u03bd\u03c9 \u03bc\u03ad\u03c1\u03bf\u03c5\u03c2 \u03c4\u03bf\u03c5 \u03b2\u03c1\u03b1\u03c7\u03af\u03bf\u03bd\u03b1 \u03c0\u03bf\u03c5 \u03b8\u03b1 \u03b5\u03c0\u03b9\u03c4\u03c1\u03ad\u03c8\u03b5\u03b9 \u03c4\u03b7 \u03bb\u03ae\u03c8\u03b7 \u03bc\u03c5\u03ca\u03ba\u03ce\u03bd \u03b2\u03b9\u03bf\u03c8\u03b9\u03ce\u03bd \u03b5\u03c0\u03b1\u03c1\u03ba\u03bf\u03cd\u03c2 \u03bc\u03b5\u03b3\u03ad\u03b8\u03bf\u03c5\u03c2 (1 cm3) \u03c0\u03c1\u03b9\u03bd \u03ba\u03b1\u03b9 \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b4\u03b9\u03ac\u03c1\u03ba\u03b5\u03b9\u03b1 \u03c4\u03b7\u03c2 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1\u03c2 (\u03b3\u03b9\u03b1 \u03c4\u03bf\u03c5\u03c2 \u03b1\u03c3\u03b8\u03b5\u03bd\u03b5\u03af\u03c2 \u03c3\u03c4\u03bf \u03b4\u03b9\u03c0\u03bb\u03ac \u03c4\u03c5\u03c6\u03bb\u03cc \u03bc\u03ad\u03c1\u03bf\u03c2 \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2).\n6. \u039d\u03b1 \u03bb\u03b1\u03bc\u03b2\u03ac\u03bd\u03b5\u03b9 \u03c3\u03c4\u03b1\u03b8\u03b5\u03c1\u03ae \u03b4\u03cc\u03c3\u03b7 \u03ae \u03b9\u03c3\u03bf\u03b4\u03cd\u03bd\u03b1\u03bc\u03b7 \u03b4\u03cc\u03c3\u03b7 \u03b1\u03c0\u03cc \u03c4\u03bf\u03c5 \u03c3\u03c4\u03cc\u03bc\u03b1\u03c4\u03bf\u03c2 \u03ba\u03bf\u03c1\u03c4\u03b9\u03ba\u03bf\u03c3\u03c4\u03b5\u03c1\u03bf\u03b5\u03b9\u03b4\u03ce\u03bd \u03b3\u03b9\u03b1 \u03c4\u03bf\u03c5\u03bb\u03ac\u03c7\u03b9\u03c3\u03c4\u03bf\u03bd 12 \u03b5\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b5\u03c2 \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c4\u03c5\u03c7\u03b1\u03b9\u03bf\u03c0\u03bf\u03af\u03b7\u03c3\u03b7 \u03ba\u03b1\u03b9 \u03b7 \u03b4\u03cc\u03c3\u03b7 \u03b1\u03bd\u03b1\u03bc\u03ad\u03bd\u03b5\u03c4\u03b1\u03b9 \u03bd\u03b1 \u03c0\u03b1\u03c1\u03b1\u03bc\u03b5\u03af\u03bd\u03b5\u03b9 \u03c3\u03c4\u03b1\u03b8\u03b5\u03c1\u03ae (\u03b5\u03ba\u03c4\u03cc\u03c2 \u03b1\u03c0\u03cc \u03c4\u03b9\u03c2 \u03c4\u03c1\u03bf\u03c0\u03bf\u03c0\u03bf\u03b9\u03ae\u03c3\u03b5\u03b9\u03c2 \u03b3\u03b9\u03b1 \u03c4\u03b7\u03bd \u03c0\u03c1\u03bf\u03c3\u03b1\u03c1\u03bc\u03bf\u03b3\u03ae \u03c3\u03c4\u03b9\u03c2 \u03b1\u03bb\u03bb\u03b1\u03b3\u03ad\u03c2 \u03c4\u03bf\u03c5 \u03b2\u03ac\u03c1\u03bf\u03c5\u03c2 \u03ba\u03b1\u03b9 \u03c3\u03c4\u03b9\u03c2 \u03b1\u03bd\u03ac\u03b3\u03ba\u03b5\u03c2 \u03c0\u03bf\u03c5 \u03c3\u03c7\u03b5\u03c4\u03af\u03b6\u03bf\u03bd\u03c4\u03b1\u03b9 \u03bc\u03b5 \u03c4\u03bf \u03c3\u03c4\u03c1\u03b5\u03c2, \u03c3\u03cd\u03bc\u03c6\u03c9\u03bd\u03b1 \u03bc\u03b5 \u03c4\u03b9\u03c2 \u03c0\u03c1\u03cc\u03c3\u03c6\u03b1\u03c4\u03b1 \u03b4\u03b7\u03bc\u03bf\u03c3\u03b9\u03b5\u03c5\u03bc\u03ad\u03bd\u03b5\u03c2 \u03ba\u03b1\u03c4\u03b5\u03c5\u03b8\u03c5\u03bd\u03c4\u03ae\u03c1\u03b9\u03b5\u03c2 \u03bf\u03b4\u03b7\u03b3\u03af\u03b5\u03c2 [Birnkrant 2018, Kinnett 2017]) \u03ba\u03b1\u03b8\u2019 \u03cc\u03bb\u03b7 \u03c4\u03b7 \u03b4\u03b9\u03ac\u03c1\u03ba\u03b5\u03b9\u03b1 \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2.\n7. \u0393\u03b9\u03b1 \u03b7\u03bb\u03b9\u03ba\u03af\u03b5\u03c2 7 \u03b5\u03c4\u03ce\u03bd \u03ba\u03b1\u03b9 \u03ac\u03bd\u03c9, \u03bd\u03b1 \u03ad\u03c7\u03b5\u03b9 \u03c3\u03c4\u03b1\u03b8\u03b5\u03c1\u03ae \u03c0\u03bd\u03b5\u03c5\u03bc\u03bf\u03bd\u03b9\u03ba\u03ae \u03bb\u03b5\u03b9\u03c4\u03bf\u03c5\u03c1\u03b3\u03af\u03b1 (FVC \u226550% \u03c4\u03b7\u03c2 \u03c0\u03c1\u03bf\u03b2\u03bb\u03b5\u03c0\u03cc\u03bc\u03b5\u03bd\u03b7\u03c2 \u03ba\u03b1\u03b9 \u03ba\u03b1\u03bc\u03af\u03b1 \u03b1\u03c0\u03b1\u03af\u03c4\u03b7\u03c3\u03b7 \u03b3\u03b9\u03b1 \u03bd\u03c5\u03ba\u03c4\u03b5\u03c1\u03b9\u03bd\u03cc \u03b1\u03b5\u03c1\u03b9\u03c3\u03bc\u03cc) \u03c0\u03bf\u03c5, \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b3\u03bd\u03ce\u03bc\u03b7 \u03c4\u03bf\u03c5 \u0395\u03c1\u03b5\u03c5\u03bd\u03b7\u03c4\u03ae, \u03b4\u03b5\u03bd \u03b5\u03af\u03bd\u03b1\u03b9 \u03c0\u03b9\u03b8\u03b1\u03bd\u03cc \u03bd\u03b1 \u03c0\u03b1\u03c1\u03bf\u03c5\u03c3\u03b9\u03ac\u03c3\u03b5\u03b9 \u03c3\u03b7\u03bc\u03b1\u03bd\u03c4\u03b9\u03ba\u03ae \u03b1\u03bd\u03c4\u03b9\u03c1\u03c1\u03cc\u03c0\u03b7\u03c3\u03b7 \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b4\u03b9\u03ac\u03c1\u03ba\u03b5\u03b9\u03b1 \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2. \u0393\u03b9\u03b1 \u03b7\u03bb\u03b9\u03ba\u03af\u03b5\u03c2 4 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\u03c4\u03c1\u03b1\u03c7\u03b7\u03bb\u03b9\u03ba\u03ae \u03ba\u03b1\u03bb\u03cd\u03c0\u03c4\u03c1\u03b1, \u03b4\u03b9\u03ac\u03c6\u03c1\u03b1\u03b3\u03bc\u03b1 \u03ae \u03c3\u03c0\u03cc\u03b3\u03b3\u03bf \u03bc\u03b5 \u03c3\u03c0\u03b5\u03c1\u03bc\u03b1\u03c4\u03bf\u03ba\u03c4\u03cc\u03bd\u03bf (\u03b1\u03bd\u03c4\u03b9\u03c3\u03cd\u03bb\u03bb\u03b7\u03c8\u03b7 \u03b4\u03b9\u03c0\u03bb\u03bf\u03cd \u03c6\u03c1\u03b1\u03b3\u03bc\u03bf\u03cd).\n9. \u039d\u03b1 \u03ad\u03c7\u03b5\u03b9 \u03b3\u03bf\u03bd\u03b5\u03af\u03c2 \u03ae \u03bd\u03cc\u03bc\u03b9\u03bc\u03bf\u03c5\u03c2 \u03ba\u03b7\u03b4\u03b5\u03bc\u03cc\u03bd\u03b5\u03c2 \u03c0\u03bf\u03c5 \u03b5\u03af\u03bd\u03b1\u03b9 \u03c3\u03b5 \u03b8\u03ad\u03c3\u03b7 \u03bd\u03b1 \u03ba\u03b1\u03c4\u03b1\u03bd\u03bf\u03ae\u03c3\u03bf\u03c5\u03bd \u03ba\u03b1\u03b9 \u03bd\u03b1 \u03c3\u03c5\u03bc\u03bc\u03bf\u03c1\u03c6\u03c9\u03b8\u03bf\u03cd\u03bd \u03bc\u03b5 \u03cc\u03bb\u03b5\u03c2 \u03c4\u03b9\u03c2 \u03b1\u03c0\u03b1\u03b9\u03c4\u03ae\u03c3\u03b5\u03b9\u03c2 \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2.\n10. \u039d\u03b1 \u03b5\u03af\u03bd\u03b1\u03b9 \u03c0\u03c1\u03cc\u03b8\u03c5\u03bc\u03bf\u03c2 \u03bd\u03b1 \u03c0\u03b1\u03c1\u03ac\u03c3\u03c7\u03b5\u03b9 \u03c3\u03c5\u03bd\u03b1\u03af\u03bd\u03b5\u03c3\u03b7 \u03b1\u03bd\u03b7\u03bb\u03af\u03ba\u03bf\u03c5 \u03bc\u03b5\u03c4\u03ac \u03b1\u03c0\u03cc \u03b5\u03bd\u03b7\u03bc\u03ad\u03c1\u03c9\u03c3\u03b7 (\u03b5\u03ac\u03bd \u03b9\u03c3\u03c7\u03cd\u03b5\u03b9) \u03ba\u03b1\u03b9 \u03bd\u03b1 \u03ad\u03c7\u03b5\u03b9 \u03b3\u03bf\u03bd\u03b5\u03af\u03c2 \u03ae \u03bd\u03cc\u03bc\u03b9\u03bc\u03bf\u03c5\u03c2 \u03ba\u03b7\u03b4\u03b5\u03bc\u03cc\u03bd\u03b5\u03c2 \u03c0\u03bf\u03c5 \u03b5\u03af\u03bd\u03b1\u03b9 \u03c0\u03c1\u03cc\u03b8\u03c5\u03bc\u03bf\u03b9 \u03bd\u03b1 \u03c0\u03b1\u03c1\u03ac\u03c3\u03c7\u03bf\u03c5\u03bd \u03c3\u03c5\u03bd\u03b1\u03af\u03bd\u03b5\u03c3\u03b7 \u03bc\u03b5\u03c4\u03ac \u03b1\u03c0\u03cc \u03b5\u03bd\u03b7\u03bc\u03ad\u03c1\u03c9\u03c3\u03b7 \u03b3\u03b9\u03b1 \u03c4\u03b7 \u03c3\u03c5\u03bc\u03bc\u03b5\u03c4\u03bf\u03c7\u03ae \u03c4\u03bf\u03c5 \u03b1\u03c3\u03b8\u03b5\u03bd\u03bf\u03cd\u03c2 \u03c3\u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7.", "language": 32, "languageDescription": "Greek (Greece)"}, {"id": 10124231, "uuid": "7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0", "attributeTranslation": "1. Mannelijke pati\u00ebnt 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 pati\u00ebnt, 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) spierbiopsie\u00ebn te verkrijgen v\u00f3\u00f3r en tijdens de behandeling (voor pati\u00ebnten 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 corticostero\u00efden 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 \u2265 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 pati\u00ebnt. 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-barri\u00e8re-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 ge\u00efnformeerde instemming te geven (indien van toepassing) en heeft (een) ouder(s) of wettelijke voogd(en) die bereid is (zijn) schriftelijke ge\u00efnformeerde toestemming te geven voor de pati\u00ebnt om aan het onderzoek mee te doen.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 10124234, "uuid": "7a3047cd-bf3c-49ad-8e8a-4b442b5df1f0", "attributeTranslation": "1. \u00catre un gar\u00e7on avec un diagnostic clinique \u00e9tabli de DMD et une mutation par d\u00e9l\u00e9tion hors cadre du g\u00e8ne DMD, susceptible de r\u00e9pondre au saut de l'exon 51 (par exemple, d\u00e9l\u00e9tions des exons 45 50, 47 50, 48 50, 49 50, 50, 52 et 52 63).\n2.\u00catre \u00e2g\u00e9 de 4 \u00e0 13 ans inclus.\n3. Patient ambulatoire, capable d'effectuer le TTRISE en 10 secondes ou moins au moment de la visite de s\u00e9lection.\n4. Capable de marcher de fa\u00e7on ind\u00e9pendante, sans dispositifs d\u2019assistance.\n5. Pr\u00e9sente un biceps ou autre groupe musculaire alternatif intact au niveau du bras sup\u00e9rieur, qui permette l\u2019obtention de biopsies musculaires de 1 cm3 avant et pendant le traitement (pour les patients enr\u00f4l\u00e9s dans la partie en double aveugle de l\u2019\u00e9tude).\n6. Avoir suivi un traitement \u00e0 dose stable ou dose \u00e9quivalente de cortico\u00efdes oraux pendant au moins 12 semaines avant la randomisation et la dose devrait rester constante (\u00e0 l'exception des modifications pour tenir compte des changements de poids et des besoins li\u00e9s au stress, conform\u00e9ment aux lignes directrices r\u00e9cemment publi\u00e9es [Birnkrant 2018, Kinnett 2017]) tout au long de l'\u00e9tude.\n7. Pour les plus de 7 ans, avoir une fonction pulmonaire stable (CVF \u2265 50 % de la valeur pr\u00e9dite et aucun recours \u00e0 la ventilation nocturne) qui, selon l\u2019investigateur, est peu susceptible de d\u00e9compenser significativement pendant la dur\u00e9e de l\u2019\u00e9tude ; et pour les \u00e2ges de 4 \u00e0 6 ans, ne pas avoir besoin de l\u2019assistance d\u2019un respirateur ou d\u2019une ventilation non invasive au moment de la s\u00e9lection.\n8. S'il est sexuellement actif, il accepte d'utiliser un pr\u00e9servatif masculin pendant toute la dur\u00e9e de l'\u00e9tude et pendant 90 jours apr\u00e8s la derni\u00e8re dose. La partenaire sexuelle doit \u00e9galement utiliser une forme de contraception m\u00e9dicalement acceptable (c'est-\u00e0-dire des contraceptifs oraux f\u00e9minins) pendant cette p\u00e9riode. Les m\u00e9thodes de contraception acceptables sont les suivantes : contraception hormonale combin\u00e9e (contenant des \u0153strog\u00e8nes et de la progest\u00e9rone) associ\u00e9e \u00e0 une inhibition de l'ovulation (par voie orale, intravaginale ou transdermique) ; contraception hormonale \u00e0 base de progest\u00e9rone uniquement associ\u00e9e \u00e0 une inhibition de l'ovulation (par voie orale, injectable ou implantable) ; dispositif intra-ut\u00e9rin ; syst\u00e8me intra-ut\u00e9rin de lib\u00e9ration d'hormones ; occlusion tubaire bilat\u00e9rale ; partenaire vasectomis\u00e9 ; abstinence sexuelle. (V\u00e9ritable abstinence : Lorsqu'elle correspond au mode de vie pr\u00e9f\u00e9r\u00e9 et habituel de la patiente. L'abstinence p\u00e9riodique [telle que les m\u00e9thodes calendaires, d'ovulation, symptothermiques, post-ovulatoires] et le retrait ne sont pas des m\u00e9thodes de contraception acceptables) ; ou le pr\u00e9servatif en combinaison avec une cape, un diaphragme ou une \u00e9ponge avec spermicide (contraception \u00e0 double barri\u00e8re).\n9. A un (des) parent(s) ou un (des) tuteur(s) l\u00e9gal(aux) qui est (sont) capable(s) de comprendre et de se conformer \u00e0 toutes les exigences de l'\u00e9tude.\n10.Est dispos\u00e9 \u00e0 donner son consentement \u00e9clair\u00e9 (le cas \u00e9ch\u00e9ant) et a un (des) parent(s) ou un (des) tuteur(s) l\u00e9gal(aux) qui est (sont) dispos\u00e9(s) \u00e0 donner son (leur) consentement \u00e9clair\u00e9 pour que le patient participe \u00e0 l'\u00e9tude.", "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 \u00d7 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] \u2264 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) \u2265450 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 corticostero\u00efden) 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 pati\u00ebnt 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 \u00d7 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] \u2264 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) \u2265 450 milliseconden, gebaseerd op de elektrocardiogrammen (ECG\u2019s) bij screening.\n10. Eerdere of aanhoudende medische aandoeningen die, naar de mening van de onderzoeker, de veiligheid van de pati\u00ebnt negatief kunnen be\u00efnvloeden, 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. B\u00e1rmilyen farmakol\u00f3giai kezel\u00e9s (a kortikoszteroidok kiv\u00e9tel\u00e9vel) alkalmaz\u00e1sa a randomiz\u00e1l\u00e1st megel\u0151z\u0151 12 h\u00e9ten bel\u00fcl, amely hat\u00e1ssal lehet az izomer\u0151re vagy az izomfunkci\u00f3ra. N\u00f6veked\u00e9si hormon alacsony termetre \u00e9s tesztoszteron k\u00e9sleltetett pubert\u00e1s eset\u00e9n enged\u00e9lyezett, ha az orvos dokument\u00e1lta a diagn\u00f3zist \u00e9s a kezel\u00e9s orvosi sz\u00fcks\u00e9gess\u00e9g\u00e9t, \u00e9s ha a beteg a randomiz\u00e1l\u00e1s el\u0151tt legal\u00e1bb 24 h\u00e9tig stabil d\u00f3zist kapott.\n2. Jelenlegi vagy kor\u00e1bbi kezel\u00e9s b\u00e1rmely m\u00e1s k\u00eds\u00e9rleti farmakol\u00f3giai kezel\u00e9ssel a DMD kezel\u00e9s\u00e9re, vagy b\u00e1rmilyen kor\u00e1bbi expoz\u00edci\u00f3 antisense oligonukleotiddal, g\u00e9nter\u00e1pi\u00e1val vagy g\u00e9nszerkeszt\u00e9ssel; kiv\u00e9ve a k\u00f6vetkez\u0151ket:\nAz els\u0151 adagot megel\u0151z\u0151en legal\u00e1bb 12 h\u00e9ttel beadott Ezutromid.\nDrisapersen, amelyet legal\u00e1bb 36 h\u00e9ttel az els\u0151 adag el\u0151tt adtak be Suvodirsen, amelyet legal\u00e1bb 12 h\u00e9ttel az els\u0151 adag el\u0151tt adtak be. Vamorolon, amelyet legal\u00e1bb 12 h\u00e9ttel az els\u0151 adag el\u0151tt adtak be. Eteplirsen (kor\u00e1bbi vagy jelenlegi alkalmaz\u00e1s).\n3. Nagyobb m\u0171t\u00e9t a randomiz\u00e1l\u00e1st megel\u0151z\u0151 3 h\u00f3napon bel\u00fcl, vagy a vizsg\u00e1lat sor\u00e1n b\u00e1rmikor tervezett m\u0171t\u00e9t, kiv\u00e9ve a protokoll \u00e1ltal meghat\u00e1rozott, enged\u00e9lyezett m\u0171t\u00e9tet, ha alkalmazhat\u00f3.\n4. B\u00e1rmilyen jelent\u0151s neuromuszkul\u00e1ris vagy genetikai betegs\u00e9g jelenl\u00e9te a DMD-n k\u00edv\u00fcl (pl. t\u00f6rpes\u00e9g).\n5. Gamma-glutamil-transzpeptid\u00e1z (GGT) > 3 \u00d7 a norm\u00e1l\u00e9rt\u00e9k fels\u0151 hat\u00e1ra (ULN) vagy sz\u00e9rum bilirubin > ULN, amelyet nem magyar\u00e1z meg Gilbert-szindr\u00f3ma.\n6. A vesefunkci\u00f3 b\u00e1rmilyen ismert k\u00e1rosod\u00e1sa (pl. a Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] cystatin C alap\u00fa egyenlete [Inker, 2012, Filler 2012] alapj\u00e1n becs\u00fclt glomerul\u00e1ris filtr\u00e1ci\u00f3s r\u00e1ta [eGFR] \u2264 60 ml/perc), vagy m\u00e9r\u0151szelv\u00e9ny feh\u00e9rjeeredm\u00e9nye +2, vagy tart\u00f3s \u00e9s megmagyar\u00e1zhatatlan m\u00e9r\u0151szelv\u00e9ny feh\u00e9rjeeredm\u00e9nye +1\n7. Trombocitasz\u00e1m < a norm\u00e1l\u00e9rt\u00e9k als\u00f3 hat\u00e1ra.\n8. Egy\u00e9b klinikailag jelent\u0151s betegs\u00e9g jelenl\u00e9te, bele\u00e9rtve a jelent\u0151s sz\u00edv-, t\u00fcd\u0151-, m\u00e1j-, vese-, hematol\u00f3giai, immunol\u00f3giai vagy viselked\u00e9si betegs\u00e9get vagy malignit\u00e1st.\n9. Kardiomiop\u00e1ti\u00e1ra utal\u00f3 jelek, amelyet a sz\u0171r\u0151 EKG alapj\u00e1n a bal kamrai ejekci\u00f3s frakci\u00f3 <50% vagy a Fridericia korrekci\u00f3s k\u00e9plet (QTcF) \u2265450 milliszekundum alapj\u00e1n a sz\u0171r\u0151 EKG-ken meghat\u00e1rozott.\n10. Kor\u00e1bbi vagy folyamatban l\u00e9v\u0151 eg\u00e9szs\u00e9g\u00fcgyi \u00e1llapot, amely a vizsg\u00e1l\u00f3 v\u00e9lem\u00e9nye szerint h\u00e1tr\u00e1nyosan befoly\u00e1solhatja a beteg biztons\u00e1g\u00e1t, val\u00f3sz\u00edn\u0171tlenn\u00e9 teheti a kezel\u00e9s befejez\u00e9s\u00e9t, vagy ronthatja a vizsg\u00e1lati eredm\u00e9nyek \u00e9rt\u00e9kel\u00e9s\u00e9t.\n11. Ismert t\u00fal\u00e9rz\u00e9kenys\u00e9g az eteplirszennel vagy az eteplirsz\u00e9n b\u00e1rmely seg\u00e9danyag\u00e1val szemben.\n12. A vizsg\u00e1l\u00f3 v\u00e9lem\u00e9nye szerint nem k\u00e9pes vagy nem hajland\u00f3 megfelelni a vizsg\u00e1lati elj\u00e1r\u00e1soknak.", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124222, "uuid": "5f2989e9-d92b-4a4e-a7cc-73a708a40b67", "attributeTranslation": "1. Stosowanie w ci\u0105gu 12 tygodni przed randomizacj\u0105 jakiegokolwiek leczenia farmakologicznego (innego ni\u017c kortykosteroidy), kt\u00f3re mo\u017ce mie\u0107 wp\u0142yw na si\u0142\u0119 lub czynno\u015b\u0107 mi\u0119\u015bni. Dopuszczalne jest stosowanie hormonu wzrostu w przypadku niskiego wzrostu oraz testosteronu w przypadku op\u00f3\u017anionego dojrzewania, je\u015bli lekarz udokumentowa\u0142 rozpoznanie oraz medyczn\u0105 konieczno\u015b\u0107 leczenia i je\u015bli pacjent przyjmowa\u0142 sta\u0142\u0105 dawk\u0119 przez co najmniej 24 tygodnie przed randomizacj\u0105.\n2. Aktualne lub wcze\u015bniejsze leczenie DMD z zastosowaniem jakiejkolwiek innej eksperymentalnej terapii farmakologicznej lub jakakolwiek wcze\u015bniejsza ekspozycja na oligonukleotyd antysensowny, terapi\u0119 genow\u0105 lub edycj\u0119 gen\u00f3w; wyj\u0105tek stanowi\u0105 nast\u0119puj\u0105ce leki:\nEzutromid podawany co najmniej 12 tygodni przed pierwsz\u0105 dawk\u0105.\nDrisapersen podawany co najmniej 36 tygodni przed pierwsz\u0105 dawk\u0105. Suvodirsen podawany co najmniej 12 tygodni przed pierwsz\u0105 dawk\u0105. Vamorolone podawany co najmniej 12 tygodni przed pierwsz\u0105 dawk\u0105. Eteplirsen (stosowanie w przesz\u0142o\u015bci lub obecnie).\nTamoksyfen podawany co najmniej 4 tygodnie przed pierwsz\u0105 dawk\u0105.\n3. Powa\u017cny zabieg chirurgiczny w ci\u0105gu 3 miesi\u0119cy przed randomizacj\u0105 lub planowany zabieg chirurgiczny w dowolnym momencie w trakcie tego badania, z wyj\u0105tkiem dozwolonych w protokole zabieg\u00f3w chirurgicznych, je\u015bli dotyczy.\n4. Wyst\u0119powanie jakiejkolwiek istotnej choroby nerwowo-mi\u0119\u015bniowej lub genetycznej innej ni\u017c DMD (np. kar\u0142owato\u015bci).\n5. Aktywno\u015b\u0107 gamma-glutamylotranspeptydazy (GGTP) > 3 \u00d7 g\u00f3rnej granicy normy (GGN) lub st\u0119\u017cenie bilirubiny w surowicy > GGN, kt\u00f3rego nie mo\u017cna wyja\u015bni\u0107 wyst\u0119powaniem zespo\u0142u Gilberta.\n6. Wszelkie znane zaburzenia czynno\u015bci nerek (np. szacunkowy wska\u017anik filtracji k\u0142\u0119buszkowej [eGFR] \u2264 60 ml/min, obliczony na podstawie wzoru Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI], opartego na st\u0119\u017ceniu cystatyny C [Inker, 2012, Filler 2012]) lub wynik testu paskowego na obecno\u015b\u0107 bia\u0142ka wynosz\u0105cy +2 b\u0105d\u017a utrzymuj\u0105cy si\u0119 i niewyja\u015bniony wynik testu paskowego na obecno\u015b\u0107 bia\u0142ka wynosz\u0105cy +1.\n7. Liczba p\u0142ytek krwi < dolnej granicy normy.\n8. Wyst\u0119powanie innej, klinicznie istotnej choroby, w tym istotnej choroby serca, p\u0142uc, w\u0105troby, nerek, choroby hematologicznej, immunologicznej lub behawioralnej b\u0105d\u017a nowotworu z\u0142o\u015bliwego.\n9. Cechy kardiomiopatii, zdefiniowanej jako frakcja wyrzutowa lewej komory wynosz\u0105ca <50% w przesiewowym badaniu echokardiograficznym (ECHO) lub okre\u015blonej na podstawie skorygowanego wzoru Fridericii (QTcF) \u2265450 milisekund w przesiewowym badaniu elektrokardiograficznym (EKG).\n10. Przebyte lub utrzymuj\u0105ce si\u0119 aktualnie schorzenie, kt\u00f3re w opinii badacza mog\u0142oby niekorzystnie wp\u0142yn\u0105\u0107 na bezpiecze\u0144stwo pacjenta, sprawia\u0107, \u017ce prawdopodobie\u0144stwo uko\u0144czenia leczenia by\u0142oby niewielkie lub zak\u0142\u00f3ca\u0107 ocen\u0119 wynik\u00f3w badania.\n11. Stwierdzona nadwra\u017cliwo\u015b\u0107 na eteplirsen lub kt\u00f3r\u0105kolwiek substancj\u0119 pomocnicz\u0105 eteplirsenu.\n12. Brak mo\u017cliwo\u015bci lub ch\u0119ci przestrzegania przez pacjenta procedur badania w opinii badacza.", "language": 19, "languageDescription": "Polish"}, {"id": 10124219, "uuid": "5f2989e9-d92b-4a4e-a7cc-73a708a40b67", "attributeTranslation": "1.\u03a7\u03c1\u03ae\u03c3\u03b7 \u03bf\u03c0\u03bf\u03b9\u03b1\u03c3\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03c6\u03b1\u03c1\u03bc\u03b1\u03ba\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ae\u03c2 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1\u03c2 (\u03b5\u03ba\u03c4\u03cc\u03c2 \u03b1\u03c0\u03cc \u03c4\u03b1 \u03ba\u03bf\u03c1\u03c4\u03b9\u03ba\u03bf\u03c3\u03c4\u03b5\u03c1\u03bf\u03b5\u03b9\u03b4\u03ae) \u03b5\u03bd\u03c4\u03cc\u03c2 12 \u03b5\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03c9\u03bd \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c4\u03c5\u03c7\u03b1\u03b9\u03bf\u03c0\u03bf\u03af\u03b7\u03c3\u03b7 \u03c0\u03bf\u03c5 \u03bc\u03c0\u03bf\u03c1\u03b5\u03af \u03bd\u03b1 \u03ad\u03c7\u03b5\u03b9 \u03b5\u03c0\u03af\u03b4\u03c1\u03b1\u03c3\u03b7 \u03c3\u03c4\u03b7 \u03bc\u03c5\u03ca\u03ba\u03ae \u03b4\u03cd\u03bd\u03b1\u03bc\u03b7 \u03ae \u03bb\u03b5\u03b9\u03c4\u03bf\u03c5\u03c1\u03b3\u03af\u03b1. \u0397 \u03b1\u03c5\u03be\u03b7\u03c4\u03b9\u03ba\u03ae \u03bf\u03c1\u03bc\u03cc\u03bd\u03b7 \u03b3\u03b9\u03b1 \u03c4\u03bf \u03b2\u03c1\u03b1\u03c7\u03cd \u03b1\u03bd\u03ac\u03c3\u03c4\u03b7\u03bc\u03b1 \u03ba\u03b1\u03b9 \u03b7 \u03c4\u03b5\u03c3\u03c4\u03bf\u03c3\u03c4\u03b5\u03c1\u03cc\u03bd\u03b7 \u03b3\u03b9\u03b1 \u03c4\u03b7\u03bd \u03ba\u03b1\u03b8\u03c5\u03c3\u03c4\u03b5\u03c1\u03b7\u03bc\u03ad\u03bd\u03b7 \u03b5\u03c6\u03b7\u03b2\u03b5\u03af\u03b1 \u03b5\u03c0\u03b9\u03c4\u03c1\u03ad\u03c0\u03bf\u03bd\u03c4\u03b1\u03b9 \u03b5\u03ac\u03bd \u03ad\u03bd\u03b1\u03c2 \u03b9\u03b1\u03c4\u03c1\u03cc\u03c2 \u03ad\u03c7\u03b5\u03b9 \u03c4\u03b5\u03ba\u03bc\u03b7\u03c1\u03b9\u03ce\u03c3\u03b5\u03b9 \u03c4\u03b7 \u03b4\u03b9\u03ac\u03b3\u03bd\u03c9\u03c3\u03b7 \u03ba\u03b1\u03b9 \u03c4\u03b7\u03bd \u03b9\u03b1\u03c4\u03c1\u03b9\u03ba\u03ae \u03b1\u03bd\u03b1\u03b3\u03ba\u03b1\u03b9\u03cc\u03c4\u03b7\u03c4\u03b1 \u03c4\u03b7\u03c2 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1\u03c2 \u03ba\u03b1\u03b9 \u03b5\u03ac\u03bd \u03bf \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03bb\u03ac\u03bc\u03b2\u03b1\u03bd\u03b5 \u03c3\u03c4\u03b1\u03b8\u03b5\u03c1\u03ae \u03b4\u03cc\u03c3\u03b7 \u03b3\u03b9\u03b1 \u03c4\u03bf\u03c5\u03bb\u03ac\u03c7\u03b9\u03c3\u03c4\u03bf\u03bd 24 \u03b5\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b5\u03c2 \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c4\u03c5\u03c7\u03b1\u03b9\u03bf\u03c0\u03bf\u03af\u03b7\u03c3\u03b7.\n2.\u03a4\u03c1\u03ad\u03c7\u03bf\u03c5\u03c3\u03b1 \u03ae \u03c0\u03c1\u03bf\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03b7 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1 \u03bc\u03b5 \u03bf\u03c0\u03bf\u03b9\u03b1\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03ac\u03bb\u03bb\u03b7 \u03c0\u03b5\u03b9\u03c1\u03b1\u03bc\u03b1\u03c4\u03b9\u03ba\u03ae \u03c6\u03b1\u03c1\u03bc\u03b1\u03ba\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ae \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1 \u03b3\u03b9\u03b1 DMD \u03ae \u03bf\u03c0\u03bf\u03b9\u03b1\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03c0\u03c1\u03bf\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03b7 \u03ad\u03ba\u03b8\u03b5\u03c3\u03b7 \u03c3\u03b5 \u03b1\u03bd\u03c4\u03b9\u03bd\u03bf\u03b7\u03bc\u03b1\u03c4\u03b9\u03ba\u03cc \u03bf\u03bb\u03b9\u03b3\u03bf\u03bd\u03bf\u03c5\u03ba\u03bb\u03b5\u03bf\u03c4\u03af\u03b4\u03b9\u03bf, \u03b3\u03bf\u03bd\u03b9\u03b4\u03b9\u03b1\u03ba\u03ae \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1 \u03ae \u03b5\u03c0\u03b5\u03be\u03b5\u03c1\u03b3\u03b1\u03c3\u03af\u03b1 \u03b3\u03bf\u03bd\u03b9\u03b4\u03af\u03c9\u03bd, \u03b5\u03ba\u03c4\u03cc\u03c2 \u03b1\u03c0\u03cc \u03c4\u03b1 \u03b1\u03ba\u03cc\u03bb\u03bf\u03c5\u03b8\u03b1:\n\u2022Ezutromid \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03bf \u03c4\u03bf\u03c5\u03bb\u03ac\u03c7\u03b9\u03c3\u03c4\u03bf\u03bd 12 \u03b5\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b5\u03c2 \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c0\u03c1\u03ce\u03c4\u03b7 \u03b4\u03cc\u03c3\u03b7.\n\u2022Drisapersen \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03bf \u03c4\u03bf\u03c5\u03bb\u03ac\u03c7\u03b9\u03c3\u03c4\u03bf\u03bd 36 \u03b5\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b5\u03c2 \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c0\u03c1\u03ce\u03c4\u03b7 \u03b4\u03cc\u03c3\u03b7.\n\u2022Suvodirsen \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03bf \u03c4\u03bf\u03c5\u03bb\u03ac\u03c7\u03b9\u03c3\u03c4\u03bf\u03bd 12 \u03b5\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b5\u03c2 \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c0\u03c1\u03ce\u03c4\u03b7 \u03b4\u03cc\u03c3\u03b7.\n\u2022Vamorolone \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03bf \u03c4\u03bf\u03c5\u03bb\u03ac\u03c7\u03b9\u03c3\u03c4\u03bf\u03bd 12 \u03b5\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b5\u03c2 \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c0\u03c1\u03ce\u03c4\u03b7 \u03b4\u03cc\u03c3\u03b7.\n\u2022Eteplirsen (\u03c0\u03c1\u03bf\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03b7 \u03ae \u03c4\u03c1\u03ad\u03c7\u03bf\u03c5\u03c3\u03b1 \u03c7\u03c1\u03ae\u03c3\u03b7).\n\u2022\u03a4\u03b1\u03bc\u03bf\u03be\u03b9\u03c6\u03b1\u03af\u03bd\u03b7 \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03b7 \u03c4\u03bf\u03c5\u03bb\u03ac\u03c7\u03b9\u03c3\u03c4\u03bf\u03bd 4 \u03b5\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b5\u03c2 \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c0\u03c1\u03ce\u03c4\u03b7 \u03b4\u03cc\u03c3\u03b7.\n3.\u039c\u03b5\u03af\u03b6\u03bf\u03bd\u03b1 \u03c7\u03b5\u03b9\u03c1\u03bf\u03c5\u03c1\u03b3\u03b9\u03ba\u03ae \u03b5\u03c0\u03ad\u03bc\u03b2\u03b1\u03c3\u03b7 \u03b5\u03bd\u03c4\u03cc\u03c2 3 \u03bc\u03b7\u03bd\u03ce\u03bd \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03c4\u03c5\u03c7\u03b1\u03b9\u03bf\u03c0\u03bf\u03af\u03b7\u03c3\u03b7 \u03ae \u03c0\u03c1\u03bf\u03b3\u03c1\u03b1\u03bc\u03bc\u03b1\u03c4\u03b9\u03c3\u03bc\u03ad\u03bd\u03b7 \u03c7\u03b5\u03b9\u03c1\u03bf\u03c5\u03c1\u03b3\u03b9\u03ba\u03ae \u03b5\u03c0\u03ad\u03bc\u03b2\u03b1\u03c3\u03b7 \u03bf\u03c0\u03bf\u03b9\u03b1\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03c3\u03c4\u03b9\u03b3\u03bc\u03ae \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b4\u03b9\u03ac\u03c1\u03ba\u03b5\u03b9\u03b1 \u03b1\u03c5\u03c4\u03ae\u03c2 \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2, \u03b5\u03ba\u03c4\u03cc\u03c2 \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03b5\u03c0\u03b9\u03c4\u03c1\u03b5\u03c0\u03cc\u03bc\u03b5\u03bd\u03b7 \u03c7\u03b5\u03b9\u03c1\u03bf\u03c5\u03c1\u03b3\u03b9\u03ba\u03ae \u03b5\u03c0\u03ad\u03bc\u03b2\u03b1\u03c3\u03b7 \u03c0\u03bf\u03c5 \u03bf\u03c1\u03af\u03b6\u03b5\u03c4\u03b1\u03b9 \u03b1\u03c0\u03cc \u03c4\u03bf \u03c0\u03c1\u03c9\u03c4\u03cc\u03ba\u03bf\u03bb\u03bb\u03bf, \u03ba\u03b1\u03c4\u03ac \u03c0\u03b5\u03c1\u03af\u03c0\u03c4\u03c9\u03c3\u03b7.\n4.\u03a0\u03b1\u03c1\u03bf\u03c5\u03c3\u03af\u03b1 \u03bf\u03c0\u03bf\u03b9\u03b1\u03c3\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03c3\u03b7\u03bc\u03b1\u03bd\u03c4\u03b9\u03ba\u03ae\u03c2 \u03bd\u03b5\u03c5\u03c1\u03bf\u03bc\u03c5\u03ca\u03ba\u03ae\u03c2 \u03ae \u03b3\u03b5\u03bd\u03b5\u03c4\u03b9\u03ba\u03ae\u03c2 \u03bd\u03cc\u03c3\u03bf\u03c5 \u03b5\u03ba\u03c4\u03cc\u03c2 \u03c4\u03b7\u03c2 DMD (\u03c0.\u03c7. \u03bd\u03b1\u03bd\u03b9\u03c3\u03bc\u03cc\u03c2).\n5.\u03b3-\u03b3\u03bb\u03bf\u03c5\u03c4\u03b1\u03bc\u03c5\u03bb-\u03c4\u03c1\u03b1\u03bd\u03c3\u03c0\u03b5\u03c0\u03c4\u03b9\u03b4\u03ac\u03c3\u03b7 [GGT] >3 \u00d7 \u03c4\u03bf \u03b1\u03bd\u03ce\u03c4\u03b1\u03c4\u03bf \u03c6\u03c5\u03c3\u03b9\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03cc \u03cc\u03c1\u03b9\u03bf (ULN) \u03ae \u03c7\u03bf\u03bb\u03b5\u03c1\u03c5\u03b8\u03c1\u03af\u03bd\u03b7 \u03bf\u03c1\u03bf\u03cd >ULN \u03b7 \u03bf\u03c0\u03bf\u03af\u03b1 \u03b4\u03b5\u03bd \u03b5\u03be\u03b7\u03b3\u03b5\u03af\u03c4\u03b1\u03b9 \u03b1\u03c0\u03cc \u03c4\u03bf \u03c3\u03cd\u03bd\u03b4\u03c1\u03bf\u03bc\u03bf Gilbert.\n6.\u039f\u03c0\u03bf\u03b9\u03b1\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03b3\u03bd\u03c9\u03c3\u03c4\u03ae \u03ad\u03ba\u03c0\u03c4\u03c9\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03bd\u03b5\u03c6\u03c1\u03b9\u03ba\u03ae\u03c2 \u03bb\u03b5\u03b9\u03c4\u03bf\u03c5\u03c1\u03b3\u03af\u03b1\u03c2 (\u03c0.\u03c7. \u03b5\u03ba\u03c4\u03b9\u03bc\u03ce\u03bc\u03b5\u03bd\u03bf\u03c2 \u03c1\u03c5\u03b8\u03bc\u03cc\u03c2 \u03c3\u03c0\u03b5\u03b9\u03c1\u03b1\u03bc\u03b1\u03c4\u03b9\u03ba\u03ae\u03c2 \u03b4\u03b9\u03ae\u03b8\u03b7\u03c3\u03b7\u03c2 [eGFR] \u226460 mL/min, \u03cc\u03c0\u03c9\u03c2 \u03b1\u03be\u03b9\u03bf\u03bb\u03bf\u03b3\u03b5\u03af\u03c4\u03b1\u03b9 \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03b5\u03be\u03af\u03c3\u03c9\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03a3\u03c5\u03bd\u03b5\u03c1\u03b3\u03b1\u03c3\u03af\u03b1\u03c2 \u0395\u03c0\u03b9\u03b4\u03b7\u03bc\u03b9\u03bf\u03bb\u03bf\u03b3\u03af\u03b1\u03c2 \u03a7\u03c1\u03cc\u03bd\u03b9\u03b1\u03c2 \u039d\u03b5\u03c6\u03c1\u03b9\u03ba\u03ae\u03c2 \u039d\u03cc\u03c3\u03bf\u03c5 [CKD-EPI] \u03b2\u03ac\u03c3\u03b5\u03b9 \u03ba\u03c5\u03c3\u03c4\u03b1\u03c4\u03af\u03bd\u03b7\u03c2 C [Inker 2012, Filler 2012]) \u03ae \u03b1\u03c0\u03bf\u03c4\u03ad\u03bb\u03b5\u03c3\u03bc\u03b1 \u03c0\u03c1\u03c9\u03c4\u03b5\u0390\u03bd\u03b7\u03c2 +2 \u03bc\u03b5 \u03c4\u03b1\u03b9\u03bd\u03af\u03b1 \u03bf\u03cd\u03c1\u03c9\u03bd (dipstick) \u03ae \u03b5\u03c0\u03af\u03bc\u03bf\u03bd\u03bf \u03ba\u03b1\u03b9 \u03b1\u03bd\u03b5\u03be\u03ae\u03b3\u03b7\u03c4\u03bf \u03b1\u03c0\u03bf\u03c4\u03ad\u03bb\u03b5\u03c3\u03bc\u03b1 \u03c0\u03c1\u03c9\u03c4\u03b5\u0390\u03bd\u03b7\u03c2 +1 \u03bc\u03b5 \u03c4\u03b1\u03b9\u03bd\u03af\u03b1 \u03bf\u03cd\u03c1\u03c9\u03bd (dipstick).\n7.\u0391\u03c1\u03b9\u03b8\u03bc\u03cc\u03c2 \u03b1\u03b9\u03bc\u03bf\u03c0\u03b5\u03c4\u03b1\u03bb\u03af\u03c9\u03bd < \u03c4\u03bf \u03ba\u03b1\u03c4\u03ce\u03c4\u03b5\u03c1\u03bf \u03c6\u03c5\u03c3\u03b9\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03cc \u03cc\u03c1\u03b9\u03bf.\n8.\u03a0\u03b1\u03c1\u03bf\u03c5\u03c3\u03af\u03b1 \u03ac\u03bb\u03bb\u03b7\u03c2 \u03ba\u03bb\u03b9\u03bd\u03b9\u03ba\u03ac \u03c3\u03b7\u03bc\u03b1\u03bd\u03c4\u03b9\u03ba\u03ae\u03c2 \u03bd\u03cc\u03c3\u03bf\u03c5, \u03c3\u03c5\u03bc\u03c0\u03b5\u03c1\u03b9\u03bb\u03b1\u03bc\u03b2\u03b1\u03bd\u03bf\u03bc\u03ad\u03bd\u03b7\u03c2 \u03c3\u03b7\u03bc\u03b1\u03bd\u03c4\u03b9\u03ba\u03ae\u03c2 \u03ba\u03b1\u03c1\u03b4\u03b9\u03b1\u03ba\u03ae\u03c2, \u03c0\u03bd\u03b5\u03c5\u03bc\u03bf\u03bd\u03b9\u03ba\u03ae\u03c2, \u03b7\u03c0\u03b1\u03c4\u03b9\u03ba\u03ae\u03c2, \u03bd\u03b5\u03c6\u03c1\u03b9\u03ba\u03ae\u03c2, \u03b1\u03b9\u03bc\u03b1\u03c4\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ae\u03c2, \u03b1\u03bd\u03bf\u03c3\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ae\u03c2 \u03ae \u03c3\u03c5\u03bc\u03c0\u03b5\u03c1\u03b9\u03c6\u03bf\u03c1\u03b9\u03ba\u03ae\u03c2 \u03bd\u03cc\u03c3\u03bf\u03c5 \u03ae \u03ba\u03b1\u03ba\u03bf\u03ae\u03b8\u03b5\u03b9\u03b1\u03c2.\n9.\u0388\u03c7\u03b5\u03b9 \u03b5\u03bd\u03b4\u03b5\u03af\u03be\u03b5\u03b9\u03c2 \u03ba\u03b1\u03c1\u03b4\u03b9\u03bf\u03bc\u03c5\u03bf\u03c0\u03ac\u03b8\u03b5\u03b9\u03b1\u03c2, \u03cc\u03c0\u03c9\u03c2 \u03bf\u03c1\u03af\u03b6\u03b5\u03c4\u03b1\u03b9 \u03b1\u03c0\u03cc \u03ba\u03bb\u03ac\u03c3\u03bc\u03b1 \u03b5\u03be\u03ce\u03b8\u03b7\u03c3\u03b7\u03c2 \u03b1\u03c1\u03b9\u03c3\u03c4\u03b5\u03c1\u03ae\u03c2 \u03ba\u03bf\u03b9\u03bb\u03af\u03b1\u03c2 <50% \u03c3\u03c4\u03bf ECHO \u03b4\u03b9\u03b1\u03bb\u03bf\u03b3\u03ae\u03c2 \u03ae \u03c3\u03c4\u03b7 \u03b4\u03b9\u03cc\u03c1\u03b8\u03c9\u03c3\u03b7 \u03ba\u03b1\u03c4\u03ac Fridericia (QTcF) \u2265450 \u03c7\u03b9\u03bb\u03b9\u03bf\u03c3\u03c4\u03ac \u03c4\u03bf\u03c5 \u03b4\u03b5\u03c5\u03c4\u03b5\u03c1\u03bf\u03bb\u03ad\u03c0\u03c4\u03bf\u03c5 \u03bc\u03b5 \u03b2\u03ac\u03c3\u03b7 \u03c4\u03b1 \u0397\u039a\u0393 \u03b4\u03b9\u03b1\u03bb\u03bf\u03b3\u03ae\u03c2.\n10.\u03a0\u03c1\u03bf\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03b7 \u03ae \u03c3\u03c5\u03bd\u03b5\u03c7\u03b9\u03b6\u03cc\u03bc\u03b5\u03bd\u03b7 \u03b9\u03b1\u03c4\u03c1\u03b9\u03ba\u03ae \u03c0\u03ac\u03b8\u03b7\u03c3\u03b7 \u03c0\u03bf\u03c5 \u03b8\u03b1 \u03bc\u03c0\u03bf\u03c1\u03bf\u03cd\u03c3\u03b5, \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b3\u03bd\u03ce\u03bc\u03b7 \u03c4\u03bf\u03c5 \u0395\u03c1\u03b5\u03c5\u03bd\u03b7\u03c4\u03ae, \u03bd\u03b1 \u03b5\u03c0\u03b7\u03c1\u03b5\u03ac\u03c3\u03b5\u03b9 \u03b4\u03c5\u03c3\u03bc\u03b5\u03bd\u03ce\u03c2 \u03c4\u03b7\u03bd \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1 \u03c4\u03bf\u03c5 \u03b1\u03c3\u03b8\u03b5\u03bd\u03bf\u03cd\u03c2, \u03bd\u03b1 \u03ba\u03b1\u03c4\u03b1\u03c3\u03c4\u03ae\u03c3\u03b5\u03b9 \u03b1\u03c0\u03af\u03b8\u03b1\u03bd\u03bf \u03bd\u03b1 \u03bf\u03bb\u03bf\u03ba\u03bb\u03b7\u03c1\u03c9\u03b8\u03b5\u03af \u03b7 \u03c0\u03bf\u03c1\u03b5\u03af\u03b1 \u03c4\u03b7\u03c2 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1\u03c2 \u03ae \u03bd\u03b1 \u03b5\u03c0\u03b7\u03c1\u03b5\u03ac\u03c3\u03b5\u03b9 \u03c4\u03b7\u03bd \u03b1\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03c9\u03bd \u03b1\u03c0\u03bf\u03c4\u03b5\u03bb\u03b5\u03c3\u03bc\u03ac\u03c4\u03c9\u03bd \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2.\n11.\u0393\u03bd\u03c9\u03c3\u03c4\u03ae \u03c5\u03c0\u03b5\u03c1\u03b5\u03c5\u03b1\u03b9\u03c3\u03b8\u03b7\u03c3\u03af\u03b1 \u03c3\u03c4\u03bf eteplirsen \u03ae \u03c3\u03b5 \u03ba\u03ac\u03c0\u03bf\u03b9\u03bf \u03b1\u03c0\u03cc \u03c4\u03b1 \u03ad\u03ba\u03b4\u03bf\u03c7\u03b1 \u03c4\u03bf\u03c5 eteplirsen.\n12.\u0395\u03af\u03bd\u03b1\u03b9, \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b3\u03bd\u03ce\u03bc\u03b7 \u03c4\u03bf\u03c5 \u0395\u03c1\u03b5\u03c5\u03bd\u03b7\u03c4\u03ae, \u03b1\u03bd\u03af\u03ba\u03b1\u03bd\u03bf\u03c2 \u03ae \u03b1\u03c0\u03c1\u03cc\u03b8\u03c5\u03bc\u03bf\u03c2 \u03bd\u03b1 \u03c3\u03c5\u03bc\u03bc\u03bf\u03c1\u03c6\u03c9\u03b8\u03b5\u03af \u03bc\u03b5 \u03c4\u03b9\u03c2 \u03b4\u03b9\u03b1\u03b4\u03b9\u03ba\u03b1\u03c3\u03af\u03b5\u03c2 \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2.", "language": 5, "languageDescription": "Greek"}, {"id": 10124221, "uuid": "5f2989e9-d92b-4a4e-a7cc-73a708a40b67", "attributeTranslation": "1. Utilisation de tout traitement pharmacologique (autre que les corticost\u00e9ro\u00efdes) dans les 12 semaines pr\u00e9c\u00e9dant la randomisation, susceptible d'avoir un effet sur la force ou la fonction musculaire. L'hormone de croissance pour la petite taille et la testost\u00e9rone pour le retard de pubert\u00e9 sont autoris\u00e9es si un m\u00e9decin a document\u00e9 le diagnostic et la n\u00e9cessit\u00e9 m\u00e9dicale du traitement et si le patient a pris une dose stable pendant au moins 24 semaines avant la randomisation.\n2.Traitement actuel ou ant\u00e9rieur par tout autre traitement pharmacologique exp\u00e9rimental de la DMD ou toute exposition ant\u00e9rieure \u00e0 un oligonucl\u00e9otide antisens, \u00e0 la th\u00e9rapie g\u00e9nique ou \u00e0 l'\u00e9dition de g\u00e8nes, \u00e0 l'exception des cas suivants :\n\u2022\tEzutromid administr\u00e9 au moins 12 semaines avant la premi\u00e8re dose. \n\u2022\tDrisapersen administr\u00e9 au moins 36 semaines avant la premi\u00e8re dose.\n\u2022\tSuvodirsen administr\u00e9 au moins 12 semaines avant la premi\u00e8re dose.\n\u2022\tVamorolone administr\u00e9 au moins 12 semaines avant la premi\u00e8re dose.\n\u2022\tEteplirsen (utilisation ant\u00e9rieure ou actuelle).\n\u2022\tTamoxif\u00e8ne administr\u00e9 au moins 4 semaines avant la premi\u00e8re dose.\n3. Chirurgie majeure dans les 3 mois pr\u00e9c\u00e9dant la randomisation ou chirurgie planifi\u00e9e \u00e0 tout moment au cours de cette \u00e9tude, \u00e0 l'exception de la chirurgie autoris\u00e9e sp\u00e9cifi\u00e9e par le protocole, le cas \u00e9ch\u00e9ant.\n4.Pr\u00e9sence d'une maladie neuromusculaire ou g\u00e9n\u00e9tique importante autre que la DMD (par exemple, nanisme).\n5.Gamma-glutamyl transpeptidase [GGT] > 3 fois la limite sup\u00e9rieure de la normale (LSN) ou bilirubine s\u00e9rique > LSN inexpliqu\u00e9e par le syndrome de Gilbert.\n6.Toute alt\u00e9ration connue de la fonction r\u00e9nale (par exemple, d\u00e9bit de filtration glom\u00e9rulaire estim\u00e9 [DFGe] \u2264 60 ml/min, tel qu'\u00e9valu\u00e9 par l'\u00e9quation bas\u00e9e sur la cystatine C de la Chronic Kidney Disease Epidemiology Collaboration [CKD EPI] [Inker 2012, Filler 2012]), ou r\u00e9sultat prot\u00e9ique \u00e0 la bandelette +2, ou r\u00e9sultat prot\u00e9ique \u00e0 la bandelette +1 persistant et inexpliqu\u00e9.\n7.Num\u00e9ration plaquettaire < la limite inf\u00e9rieure de la normale\n8.Pr\u00e9sence d'une autre maladie cliniquement significative, y compris une maladie cardiaque, pulmonaire, h\u00e9patique, r\u00e9nale, h\u00e9matologique, immunologique, comportementale ou une tumeur maligne.\n9.Pr\u00e9sence d'une cardiomyopathie, d\u00e9finie par une fraction d'\u00e9jection du ventricule gauche < 50 % sur l'ECHO de s\u00e9lection ou une formule de correction de Fridericia (QTcF) \u2265 450 millisecondes sur la base des ECG de s\u00e9lection.\n10.\u00c9tat de sant\u00e9 ant\u00e9rieur ou actuel qui, de l'avis de l'investigateur, pourrait nuire \u00e0 la s\u00e9curit\u00e9 du patient, rendre improbable l'ach\u00e8vement du traitement ou compromettre l'\u00e9valuation des r\u00e9sultats de l'\u00e9tude.\n11.Hypersensibilit\u00e9 connue \u00e0 l'eteplirsen ou \u00e0 l'un des excipients de l'eteplirsen.\n12.Est, de l'avis de l'investigateur, incapable ou r\u00e9ticent \u00e0 se conformer aux proc\u00e9dures de l'\u00e9tude.\nLes patients qui ne remplissent pas les crit\u00e8res d'\u00e9ligibilit\u00e9 peuvent faire l'objet d'une nouvelle s\u00e9lection \u00e0 la discr\u00e9tion de l'investigateur.", "language": 10, "languageDescription": "French"}, {"id": 10124220, "uuid": "5f2989e9-d92b-4a4e-a7cc-73a708a40b67", "attributeTranslation": "1.\tUso de cualquier tratamiento farmacol\u00f3gico (excepto corticosteroides) dentro de las 12 semanas previas a la aleatorizaci\u00f3n que pueda tener un efecto sobre la fuerza o funci\u00f3n muscular. Se permite la hormona del crecimiento para la baja estatura y la testosterona para la pubertad tard\u00eda si el m\u00e9dico ha documentado el diagn\u00f3stico y la necesidad m\u00e9dica del tratamiento y si el paciente ha recibido una dosis estable durante al menos 24 semanas antes de la aleatorizaci\u00f3n.\n2.\tTratamiento actual o previo con cualquier otro tratamiento farmacol\u00f3gico experimental para la DMD o cualquier exposici\u00f3n previa a oligonucle\u00f3tidos antisentido, terapia g\u00e9nica o edici\u00f3n g\u00e9nica; 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. Cirug\u00eda mayor dentro de los 3 meses previos a la aleatorizaci\u00f3n o cirug\u00eda planificada para cualquier momento durante este estudio, excepto la cirug\u00eda permitida especificada en el protocolo, seg\u00fan corresponda.\n4. Presencia de cualquier enfermedad neuromuscular o gen\u00e9tica significativa distinta de la DMD (p. ej., enanismo).\n5. Gamma-glutamil transpeptidasa (GGT) > 3 \u00d7 el l\u00edmite superior de lo normal (LSN) o bilirrubina s\u00e9rica > LSN no explicada por el s\u00edndrome de Gilbert.\n6. Cualquier deterioro conocido de la funci\u00f3n renal (p. ej., tasa de filtraci\u00f3n glomerular estimada [TFGe] \u2264 60 ml/min seg\u00fan la ecuaci\u00f3n basada en cistatina C de la Colaboraci\u00f3n de Epidemiolog\u00eda de la Enfermedad Renal Cr\u00f3nica [CKD-EPI] [Inker, 2012, Filler 2012]), o resultado de prote\u00edna en tira reactiva +2, o resultado de prote\u00edna en tira reactiva persistente e inexplicable +1\n7. Recuento de plaquetas < el l\u00edmite inferior de lo normal.\n8. Presencia de otra enfermedad cl\u00ednicamente significativa, incluidas enfermedades card\u00edacas, pulmonares, hep\u00e1ticas, renales, hematol\u00f3gicas, inmunol\u00f3gicas o conductuales significativas o neoplasias malignas.\n9. Tiene evidencia de miocardiopat\u00eda, definida por una fracci\u00f3n de eyecci\u00f3n del ventr\u00edculo izquierdo <50 % en el ecocardiograma de detecci\u00f3n o la f\u00f3rmula de correcci\u00f3n de Fridericia (QTcF) \u2265450 milisegundos seg\u00fan los ECG de detecci\u00f3n.\n10. Condici\u00f3n m\u00e9dica previa o actual que podr\u00eda, en opini\u00f3n del investigador, afectar negativamente la seguridad del paciente, hacer improbable que se complete el tratamiento o perjudicar la evaluaci\u00f3n de los resultados del estudio.\n11. Hipersensibilidad conocida al eteplirsen o a cualquiera de sus excipientes.\n12. Es, en opini\u00f3n del investigador, incapaz o no est\u00e1 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 metod\u0105 otwartej pr\u00f3by:\nCz\u0119sto\u015b\u0107 wyst\u0119powania zdarze\u0144 niepo\u017c\u0105danych (AE), Cz\u0119sto\u015b\u0107 wyst\u0119powania zdarze\u0144 niepo\u017c\u0105danych o szczeg\u00f3lnym znaczeniu (AESI), Nieprawid\u0142owe zmiany w stosunku do punktu wyj\u015bciowego lub pogorszenie parametr\u00f3w \u017cyciowych lub wynik\u00f3w badania fizykalnego, Cz\u0119sto\u015b\u0107 wyst\u0119powania ci\u0119\u017ckich zdarze\u0144 niepo\u017c\u0105danych (SAE), Oceny laboratoryjne dotycz\u0105ce bezpiecze\u0144stwa, 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 r\u00e9gimen abierto:\n\nIncidencia de EA, Incidencia de eventos adversos de inter\u00e9s especial (AESI)\nCambios anormales respecto del valor inicial o empeoramiento de los signos\nvitales o hallazgos del examen f\u00edsico, 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": "Ny\u00edlt elrendez\u00e9s\u0171 d\u00f3ziseszkal\u00e1ci\u00f3:\nA nemk\u00edv\u00e1natos esem\u00e9nyek (AE-k) gyakoris\u00e1ga, a k\u00fcl\u00f6n\u00f6s figyelmet \u00e9rdeml\u0151 nemk\u00edv\u00e1natos esem\u00e9nyek (AESI-k) incidenci\u00e1ja, a k\u00f3ros elt\u00e9r\u00e9sek a kiindul\u00e1shoz k\u00e9pest, illetve az \u00e9lettani param\u00e9terek vagy a fizik\u00e1lis vizsg\u00e1lati leletek rosszabbod\u00e1sa, a s\u00falyos nemk\u00edv\u00e1natos esem\u00e9nyek (serious adverse events, SAE-k) incidenci\u00e1ja, biztons\u00e1goss\u00e1gi laborat\u00f3riumi vizsg\u00e1latok, elektrokardiogr\u00e1fi\u00e1s vizsg\u00e1latok (EKG-k), sz\u00edv-ultrahangvizsg\u00e1latok (echokardiogr\u00e1fia).", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124170, "uuid": "93f6e994-fe81-42e0-b4c1-4b9f2ac1a18e", "attributeTranslation": "Open-label dosisverhoging:\nIncidentie van bijwerkingen (AE\u2019s), incidentie van bijwerkingen van bijzonder belang (AESI\u2019s), abnormale veranderingen ten opzichte van baseline of verslechtering van de vitale functies of bevindingen bij lichamelijk onderzoek, incidentie van ernstige bijwerkingen (SAE\u2019s), laboratoriumbeoordelingen voor de veiligheid, elektrocardiogrammen (ECG\u2019s) en echocardiogrammen (ECHO).", "language": 18, "languageDescription": "Dutch"}, {"id": 10124174, "uuid": "93f6e994-fe81-42e0-b4c1-4b9f2ac1a18e", "attributeTranslation": "Augmentation de dose en ouvert\nIncidence des \u00e9v\u00e9nements ind\u00e9sirables (EI), Incidence des \u00e9v\u00e9nements ind\u00e9sirables d\u2019int\u00e9r\u00eat sp\u00e9cifique (EIIS), \u00c9volutions anormales par rapport aux valeurs de r\u00e9f\u00e9rence ou aggravation des signes vitaux et des r\u00e9sultats de l\u2019examen physique, Incidence des \u00e9v\u00e9nements ind\u00e9sirables graves (EIG), Analyses de laboratoire de s\u00e9curit\u00e9, \u00c9lectrocardiogrammes (ECG), \u00c9chocardiogrammes (ECHO).", "language": 10, "languageDescription": "French"}, {"id": 10124172, "uuid": "93f6e994-fe81-42e0-b4c1-4b9f2ac1a18e", "attributeTranslation": "\u039c\u03ad\u03c1\u03bf\u03c2 \u03ba\u03bb\u03b9\u03bc\u03ac\u03ba\u03c9\u03c3\u03b7\u03c2 \u03b4\u03cc\u03c3\u03b7\u03c2 \u03b1\u03bd\u03bf\u03b9\u03ba\u03c4\u03ae\u03c2 \u03b5\u03c0\u03b9\u03c3\u03ae\u03bc\u03b1\u03bd\u03c3\u03b7\u03c2\n\u0395\u03c0\u03af\u03c0\u03c4\u03c9\u03c3\u03b7 \u03c4\u03c9\u03bd \u0391\u03a3, \u0395\u03c0\u03af\u03c0\u03c4\u03c9\u03c3\u03b7 \u03b1\u03bd\u03b5\u03c0\u03b9\u03b8\u03cd\u03bc\u03b7\u03c4\u03c9\u03bd \u03c3\u03c5\u03bc\u03b2\u03ac\u03bd\u03c4\u03c9\u03bd \u03b5\u03b9\u03b4\u03b9\u03ba\u03bf\u03cd \u03b5\u03bd\u03b4\u03b9\u03b1\u03c6\u03ad\u03c1\u03bf\u03bd\u03c4\u03bf\u03c2 (AESI), \n\u03a0\u03b1\u03b8\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ad\u03c2 \u03bc\u03b5\u03c4\u03b1\u03b2\u03bf\u03bb\u03ad\u03c2 \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u0388\u03bd\u03b1\u03c1\u03be\u03b7 \u03ae \u03b5\u03c0\u03b9\u03b4\u03b5\u03af\u03bd\u03c9\u03c3\u03b7 \u03b6\u03c9\u03c4\u03b9\u03ba\u03ce\u03bd \u03c3\u03b7\u03bc\u03b5\u03af\u03c9\u03bd \u03ae \u03b5\u03c5\u03c1\u03b7\u03bc\u03ac\u03c4\u03c9\u03bd \u03ba\u03bb\u03b9\u03bd\u03b9\u03ba\u03ae\u03c2 \u03b5\u03be\u03ad\u03c4\u03b1\u03c3\u03b7\u03c2, \u0395\u03c0\u03af\u03c0\u03c4\u03c9\u03c3\u03b7 \u03c4\u03c9\u03bd \u03a3\u0391\u03a3, \u0395\u03c1\u03b3\u03b1\u03c3\u03c4\u03b7\u03c1\u03b9\u03b1\u03ba\u03ad\u03c2 \u03b1\u03be\u03b9\u03bf\u03bb\u03bf\u03b3\u03ae\u03c3\u03b5\u03b9\u03c2 \u03c4\u03b7\u03c2 \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1\u03c2, \n\u0397\u03bb\u03b5\u03ba\u03c4\u03c1\u03bf\u03ba\u03b1\u03c1\u03b4\u03b9\u03bf\u03b3\u03c1\u03b1\u03c6\u03ae\u03bc\u03b1\u03c4\u03b1 (\u0397\u039a\u0393), \u0397\u03c7\u03bf\u03ba\u03b1\u03c1\u03b4\u03b9\u03bf\u03b3\u03c1\u03b1\u03c6\u03ae\u03bc\u03b1\u03c4\u03b1 (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": "\u0394\u03b9\u03c0\u03bb\u03ac \u03c4\u03c5\u03c6\u03bb\u03cc \u03bc\u03ad\u03c1\u03bf\u03c2 \u03c0\u03c1\u03bf\u03c3\u03b4\u03b9\u03bf\u03c1\u03b9\u03c3\u03bc\u03bf\u03cd \u03c4\u03b7\u03c2 \u03b4\u03cc\u03c3\u03b7\u03c2 \u03ba\u03b1\u03b9 \u03c3\u03cd\u03b3\u03ba\u03c1\u03b9\u03c3\u03b7\u03c2 \u03c4\u03b7\u03c2 \u03b4\u03cc\u03c3\u03b7\u03c2:\n\u2022\u039c\u03b5\u03c4\u03b1\u03b2\u03bf\u03bb\u03ae \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u0388\u03bd\u03b1\u03c1\u03be\u03b7 \u03c4\u03b7\u03bd \u0395\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b1 72 \u03ae \u03c4\u03b7\u03bd \u0395\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b1 96 \u03c3\u03c4\u03b7 \u03c3\u03c5\u03bd\u03bf\u03bb\u03b9\u03ba\u03ae \u03b2\u03b1\u03b8\u03bc\u03bf\u03bb\u03bf\u03b3\u03af\u03b1 NSAA (\u03b3\u03b9\u03b1 \u03b5\u03bd\u03b4\u03b9\u03ac\u03bc\u03b5\u03c3\u03b7 \u03b1\u03bd\u03ac\u03bb\u03c5\u03c3\u03b7 \u03b1\u03c0\u03bf\u03c4\u03b5\u03bb\u03b5\u03c3\u03bc\u03b1\u03c4\u03b9\u03ba\u03cc\u03c4\u03b7\u03c4\u03b1\u03c2 \u03c5\u03c0\u03cc \u03cc\u03c1\u03bf\u03c5\u03c2)\n\u2022\u039c\u03b5\u03c4\u03b1\u03b2\u03bf\u03bb\u03ae \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u0388\u03bd\u03b1\u03c1\u03be\u03b7 \u03c4\u03b7\u03bd \u0395\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b1 144 \u03c3\u03c4\u03b7 \u03c3\u03c5\u03bd\u03bf\u03bb\u03b9\u03ba\u03ae \u03b2\u03b1\u03b8\u03bc\u03bf\u03bb\u03bf\u03b3\u03af\u03b1", "language": 5, "languageDescription": "Greek"}, {"id": 10124180, "uuid": "f9258092-d9e7-4cca-878e-484c98ffba06", "attributeTranslation": "Kett\u0151s-vak d\u00f3ziskeres\u0151 \u00e9s d\u00f3zis-\u00f6sszehasonl\u00edt\u00f3 r\u00e9sz:\n-A North Star j\u00e1r\u00e1svizsg\u00e1lat (North Star Ambulatory Assessment, NSAA) \u00f6sszpontsz\u00e1m\u00e1nak v\u00e1ltoz\u00e1sa a kiindul\u00e1shoz k\u00e9pest a 72. h\u00e9ten \u00e9s a 96. h\u00e9ten (felt\u00e9teles id\u0151k\u00f6zi hat\u00e1soss\u00e1gi elemz\u00e9shez)\n-Az NSAA \u00f6sszpontsz\u00e1m\u00e1nak v\u00e1ltoz\u00e1sa a kiindul\u00e1s \u00e9s a 144. h\u00e9t k\u00f6z\u00f6tt (a v\u00e9gleges elemz\u00e9shez)", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124177, "uuid": "f9258092-d9e7-4cca-878e-484c98ffba06", "attributeTranslation": "Cz\u0119\u015b\u0107 badania prowadzona metod\u0105 podw\u00f3jnie \u015blepej pr\u00f3by maj\u0105ca na celu ustalenie optymalnej dawki oraz por\u00f3wnanie dawek:\n- Zmiana warto\u015bci ca\u0142kowitego wyniku w skali North Star Ambulatory Assessment (NSAA) od punktu wyj\u015bciowego w 72. tygodniu lub 96. tygodniu (do warunkowej, \u015br\u00f3dokresowej analizy skuteczno\u015bci)\n- Zmiana warto\u015bci ca\u0142kowitego wyniku w skali NSAA od punktu wyj\u015bciowego w 144. tygodniu (do analizy ko\u0144cowej)", "language": 19, "languageDescription": "Polish"}, {"id": 10124175, "uuid": "f9258092-d9e7-4cca-878e-484c98ffba06", "attributeTranslation": "Partie de d\u00e9termination et comparaison de doses en double aveugle\n\u2022\t\u00c9volution par rapport aux valeurs de r\u00e9f\u00e9rence \u00e0 la semaine 72 ou \u00e0 la semaine 96 du score global sur l\u2019\u00e9chelle d\u2019\u00e9valuation North Star Ambulatory Assessment (NSAA) (pour l\u2019analyse interm\u00e9diaire conditionnelle de l\u2019efficacit\u00e9)\n\u2022\t\u00c9volution par rapport aux valeurs de r\u00e9f\u00e9rence \u00e0 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 r\u00e9gimen doble ciego de b\u00fasqueda y comparaci\u00f3n de la dosis:\n\n- Cambio con respecto al valor inicial en la semana 72 o la semana 96 en la puntuaci\u00f3n total de la NSAA (para el an\u00e1lisis provisional de eficacia condicional)\n-Cambio con respecto al valor inicial en la semana 144 en la puntuaci\u00f3n total de la NSAA (para el an\u00e1lisis final)", "language": 7, "languageDescription": "Spanish"}]}], "secondaryEndPoints": [{"id": 966852, "number": 1, "endPoint": "\u2022 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": "\u2022 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": "\u2022 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": "\u00c9volution observ\u00e9e \u00e0 la semaine 144 par rapport aux valeurs de r\u00e9f\u00e9rence:\no\tTemps pour se relever du sol\no\tChronom\u00e9trage de la marche/course sur 10 m\u00e8tres\no\tTest de marche de 6 minutes(6MWT)\no\tTest chronom\u00e9tr\u00e9 de mont\u00e9e d\u2019escalier sur 4 marches\no \tTaux de d\u00e9clin annuel de la capacit\u00e9 vitale forc\u00e9e (CVF) (%) pr\u00e9dite", "language": 10, "languageDescription": "French"}, {"id": 10124186, "uuid": "ca98c0a4-b59d-49c1-9463-d2f8b563a29d", "attributeTranslation": "V\u00e1ltoz\u00e1s a kiindul\u00e1si \u00e9rt\u00e9khez k\u00e9pest a 144. h\u00e9ten:\no\ta talajr\u00f3l t\u00f6rt\u00e9n\u0151 fel\u00e1ll\u00e1shoz sz\u00fcks\u00e9ges id\u0151 (TTRISE)\no\t10 m\u00e9teres j\u00e1r\u00e1s/fut\u00e1s ideje\no\t6 perces j\u00e1r\u00e1svizsg\u00e1lat (6MWT)\no\t4 l\u00e9pcs\u0151n fell\u00e9pdel\u00e9s id\u0151m\u00e9r\u00e9ses vizsg\u00e1lata\no\tforsz\u00edrozott vit\u00e1lkapacit\u00e1s, a becs\u00fclt \u00e9rt\u00e9k sz\u00e1zal\u00e9k\u00e1ban (FVC%p)", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124184, "uuid": "ca98c0a4-b59d-49c1-9463-d2f8b563a29d", "attributeTranslation": "\u2022\tZmiana w 144. tygodniu w stosunku do punktu wyj\u015bciowego w zakresie poni\u017cszych warto\u015bci: \no\tCzas potrzebny do wstania (TTRISE) z pod\u0142ogi \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 nat\u0119\u017conej pojemno\u015bci \u017cyciowej (FVC%p)", "language": 19, "languageDescription": "Polish"}, {"id": 10124182, "uuid": "ca98c0a4-b59d-49c1-9463-d2f8b563a29d", "attributeTranslation": "\u2022\u039c\u03b5\u03c4\u03b1\u03b2\u03bf\u03bb\u03ae \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u0388\u03bd\u03b1\u03c1\u03be\u03b7 \u03c4\u03b7\u03bd \u0395\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b1 144, \u03c9\u03c2 \u03c0\u03c1\u03bf\u03c2 \u03c4\u03b1 \u03b5\u03be\u03ae\u03c2:\noTTRISE \u03b1\u03c0\u03cc \u03c4\u03bf \u03b4\u03ac\u03c0\u03b5\u03b4\u03bf\no\u03a7\u03c1\u03cc\u03bd\u03bf\u03c2 \u03ba\u03ac\u03bb\u03c5\u03c8\u03b7\u03c2 \u03b1\u03c0\u03cc\u03c3\u03c4\u03b1\u03c3\u03b7\u03c2 10 \u03bc\u03ad\u03c4\u03c1\u03c9\u03bd \u03bc\u03b5 \u03c0\u03b5\u03c1\u03c0\u03ac\u03c4\u03b7\u03bc\u03b1/\u03c4\u03c1\u03ad\u03be\u03b9\u03bc\u03bf\no6MWT\no\u03a7\u03c1\u03bf\u03bd\u03bf\u03bc\u03b5\u03c4\u03c1\u03b7\u03bc\u03ad\u03bd\u03b7 \u03b4\u03bf\u03ba\u03b9\u03bc\u03b1\u03c3\u03af\u03b1 \u03b1\u03bd\u03ac\u03b2\u03b1\u03c3\u03b7\u03c2 4 \u03c3\u03ba\u03b1\u03bb\u03bf\u03c0\u03b1\u03c4\u03b9\u03ce\u03bd\no\u03a0\u03c1\u03bf\u03b2\u03bb\u03b5\u03c0\u03cc\u03bc\u03b5\u03bd\u03bf \u03c0\u03bf\u03c3\u03bf\u03c3\u03c4\u03cc \u03b2\u03af\u03b1\u03b9\u03b7\u03c2 \u03b6\u03c9\u03c4\u03b9\u03ba\u03ae\u03c2 \u03c7\u03c9\u03c1\u03b7\u03c4\u03b9\u03ba\u03cc\u03c4\u03b7\u03c4\u03b1\u03c2 (FVC%p)\"", "language": 5, "languageDescription": "Greek"}]}, {"id": 966853, "number": 2, "endPoint": "\u2022 Time to Loss of Ambulation (LOA) through Week 144", "isPrimary": false, "endPointTranslations": [{"id": 10124189, "uuid": "4d982e92-5019-49d9-83f4-afcc87f20b29", "attributeTranslation": "Dur\u00e9e jusqu\u2019\u00e0 la perte de la marche jusqu\u2019\u00e0 la semaine 144", "language": 10, "languageDescription": "French"}, {"id": 10124190, "uuid": "4d982e92-5019-49d9-83f4-afcc87f20b29", "attributeTranslation": "\u2022 Tijd tot verlies van loopvermogen (LOA) tot week 144", "language": 18, "languageDescription": "Dutch"}, {"id": 10124191, "uuid": "4d982e92-5019-49d9-83f4-afcc87f20b29", "attributeTranslation": "\u2022 Tiempo transcurrido hasta la p\u00e9rdida de la deambulaci\u00f3n (LOA)\nhasta la semana 144", "language": 7, "languageDescription": "Spanish"}, {"id": 10124187, "uuid": "4d982e92-5019-49d9-83f4-afcc87f20b29", "attributeTranslation": "\u2022\tCzas do utraty zdolno\u015bci poruszania si\u0119 (LOA) do tygodnia 144.", "language": 19, "languageDescription": "Polish"}, {"id": 10124188, "uuid": "4d982e92-5019-49d9-83f4-afcc87f20b29", "attributeTranslation": "A j\u00e1r\u00f3k\u00e9pess\u00e9g elveszt\u00e9s\u00e9ig eltelt id\u0151 (LOA) a 144. h\u00e9tig", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124192, "uuid": "4d982e92-5019-49d9-83f4-afcc87f20b29", "attributeTranslation": "\u2022\u03a7\u03c1\u03cc\u03bd\u03bf\u03c2 \u03ad\u03c9\u03c2 \u03c4\u03b7\u03bd LOA \u03ad\u03c9\u03c2 \u03c4\u03b7\u03bd \u0395\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b1 144", "language": 5, "languageDescription": "Greek"}]}, {"id": 966854, "number": 3, "endPoint": "\u2022 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 v\u00e1zizmok disztrofinexpresszi\u00f3j\u00e1nak v\u00e1ltoz\u00e1sa a kiindul\u00e1s \u00e9s a 24., 48. vagy 144. h\u00e9ten, a k\u00f6vetkez\u0151 vizsg\u00e1latok alapj\u00e1n:\no\tWestern blot (mennyis\u00e9gi meghat\u00e1roz\u00e1s)\no\timmunhisztok\u00e9mia (IHC) \u2013 rostintenzit\u00e1s immunfluoreszcenci\u00e1val\no\taz exon\u00e1tugr\u00e1s mennyis\u00e9gi meghat\u00e1roz\u00e1sa \u00fan. droplet digit\u00e1lis polimer\u00e1z l\u00e1ncreakci\u00f3val (ddPCR)\no\tA disztrofin-pozit\u00edv rostok sz\u00e1zal\u00e9kos ar\u00e1nya (PDPF) immunfluoreszcenci\u00e1val", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124196, "uuid": "729283fd-ea51-4eff-83b1-d30d067a8bfc", "attributeTranslation": "\u2022\u039c\u03b5\u03c4\u03b1\u03b2\u03bf\u03bb\u03ae \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u0388\u03bd\u03b1\u03c1\u03be\u03b7 \u03c4\u03b7\u03bd \u0395\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b1 24, \u03c4\u03b7\u03bd \u0395\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b1 48 \u03ae \u03c4\u03b7\u03bd \u0395\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03b1 144 \u03c3\u03c4\u03b7\u03bd \u03ad\u03ba\u03c6\u03c1\u03b1\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03b4\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03bd\u03b7\u03c2 \u03c4\u03c9\u03bd \u03c3\u03ba\u03b5\u03bb\u03b5\u03c4\u03b9\u03ba\u03ce\u03bd \u03bc\u03c5\u03ce\u03bd \u03bc\u03b5\u03c4\u03c1\u03b7\u03bc\u03ad\u03bd\u03b7 \u03bc\u03b5:\no\u039c\u03ad\u03b8\u03bf\u03b4\u03bf \u03b1\u03bd\u03bf\u03c3\u03bf\u03b1\u03c0\u03bf\u03c4\u03cd\u03c0\u03c9\u03c3\u03b7\u03c2 Western (\u03c0\u03bf\u03c3\u03bf\u03c4\u03b9\u03ba\u03bf\u03c0\u03bf\u03af\u03b7\u03c3\u03b7)\no\u0388\u03bd\u03c4\u03b1\u03c3\u03b7 \u03b9\u03bd\u03ce\u03bd IHC \u03bc\u03ad\u03c3\u03c9 \u03b4\u03bf\u03ba\u03b9\u03bc\u03b1\u03c3\u03af\u03b1\u03c2 \u03b1\u03bd\u03bf\u03c3\u03bf\u03c6\u03b8\u03bf\u03c1\u03b9\u03c3\u03bc\u03bf\u03cd\no\u03a0\u03bf\u03c3\u03bf\u03c4\u03b9\u03ba\u03bf\u03c0\u03bf\u03af\u03b7\u03c3\u03b7 \u03c0\u03b1\u03c1\u03ac\u03bb\u03b5\u03b9\u03c8\u03b7\u03c2 \u03b5\u03be\u03bf\u03bd\u03af\u03c9\u03bd \u03bc\u03ad\u03c3\u03c9 ddPCR\noPDPF \u03bc\u03ad\u03c3\u03c9 \u03b4\u03bf\u03ba\u03b9\u03bc\u03b1\u03c3\u03af\u03b1\u03c2 \u03b1\u03bd\u03bf\u03c3\u03bf\u03c6\u03b8\u03bf\u03c1\u03b9\u03c3\u03bc\u03bf\u03cd", "language": 5, "languageDescription": "Greek"}, {"id": 10124197, "uuid": "729283fd-ea51-4eff-83b1-d30d067a8bfc", "attributeTranslation": "\u00c9volution par rapport aux valeurs de r\u00e9f\u00e9rence \u00e0 la semaine 24, la semaine 48 ou la semaine 144 dans l\u2019expression musculo-squelettique de la dystrophine, selon les m\u00e9thodes suivantes:\no\tWestern blot (quantification)\no\tImmunohistochimie (IHC) (intensit\u00e9 des fibres par immunofluorescence)\no\tQuantification du saut d\u2019exon par r\u00e9action en cha\u00eene par polym\u00e9rase (PCR)\no\tPourcentage de fibres positives \u00e0 la dystrophine (PDPF) par immunofluorescence", "language": 10, "languageDescription": "French"}, {"id": 10124193, "uuid": "729283fd-ea51-4eff-83b1-d30d067a8bfc", "attributeTranslation": "\u2022 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": "\u2022\tVariaci\u00f3n entre el momento basal y la semana 24, la semana 48 o la semana 144 de la expresi\u00f3n de distrofina en el m\u00fasculo esquel\u00e9tico mediante:\no\tInmunoelectrotransferencia (cuantificaci\u00f3n)\no\tIntensidad de las fibras mediante inmunohistoqu\u00edmica (IHQ) mediante inmunofluorescencia\no\tCuantificaci\u00f3n del salto de exones mediante reacci\u00f3n 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": "\u2022\tZmiana w stosunku do punktu wyj\u015bciowego w tygodniu 24., 48. lub 144. w ekspresji dystrofiny w mi\u0119\u015bniach szkieletowych na podstawie oznaczenia:\no\tWestern blot (oznaczenie ilo\u015bciowe)\no\tintensywno\u015bci w\u0142\u00f3kien w badaniu immunohistochemicznym (IHC) w te\u015bcie immunofluorescencyjnym\no\tOznaczenie ilo\u015bciowe z pomini\u0119ciem egzonu metod\u0105 emulsyjnej reakcji \u0142a\u0144cuchowej polimerazy (ddPCR)\no\tOdsetek w\u0142\u00f3kien dystrofino-dodatnich (PDPF) w te\u015bcie 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": "\u2022\tFarmakokinetikai (PK) param\u00e9terek (plazma- \u00e9s izombiopszi\u00e1val)", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124204, "uuid": "905c40ec-75d7-4984-89df-cd34aca38540", "attributeTranslation": "\u2022\u03a0\u03b1\u03c1\u03ac\u03bc\u03b5\u03c4\u03c1\u03bf\u03b9 \u03a6\u039a (\u03c0\u03bb\u03ac\u03c3\u03bc\u03b1\u03c4\u03bf\u03c2 \u03ba\u03b1\u03b9 \u03bc\u03b5 \u03bc\u03c5\u03ca\u03ba\u03ae \u03b2\u03b9\u03bf\u03c8\u03af\u03b1)", "language": 5, "languageDescription": "Greek"}, {"id": 10124199, "uuid": "905c40ec-75d7-4984-89df-cd34aca38540", "attributeTranslation": "Param\u00e8tres PK (plasma et biopsie musculaire)", "language": 10, "languageDescription": "French"}, {"id": 10124203, "uuid": "905c40ec-75d7-4984-89df-cd34aca38540", "attributeTranslation": "\u2022\tPar\u00e1metros FC (en plasma y biopsia de m\u00fasculo)", "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": "\u2022\tParametry PK (osocze i biopsja tkanki mi\u0119\u015bniowej)", "language": 19, "languageDescription": "Polish"}]}, {"id": 966856, "number": 5, "endPoint": "\u2022 Incidence of AEs \u2022 Incidence of AESIs \u2022 Incidence of SAEs \u2022 Safety laboratory assessments", "isPrimary": false, "endPointTranslations": [{"id": 10124208, "uuid": "e8d5ce8d-375c-4838-98c4-fa444e213cfa", "attributeTranslation": "\u2022\u0395\u03c0\u03af\u03c0\u03c4\u03c9\u03c3\u03b7 \u03c4\u03c9\u03bd \u0391\u03a3\n\u2022\u0395\u03c0\u03af\u03c0\u03c4\u03c9\u03c3\u03b7 \u03c4\u03c9\u03bd AESI\n\u2022\u0395\u03c0\u03af\u03c0\u03c4\u03c9\u03c3\u03b7 \u03c4\u03c9\u03bd \u03a3\u0391\u03a3\n\u2022\u0395\u03c1\u03b3\u03b1\u03c3\u03c4\u03b7\u03c1\u03b9\u03b1\u03ba\u03ad\u03c2 \u03b1\u03be\u03b9\u03bf\u03bb\u03bf\u03b3\u03ae\u03c3\u03b5\u03b9\u03c2 \u03c4\u03b7\u03c2 \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1\u03c2", "language": 5, "languageDescription": "Greek"}, {"id": 10124207, "uuid": "e8d5ce8d-375c-4838-98c4-fa444e213cfa", "attributeTranslation": "\u2022\tIncidencia de AA\n\u2022\tIncidencia de AAIE\n\u2022\tIncidencia de AAG\n\u2022\tEvaluaciones anal\u00edticas de seguridad", "language": 7, "languageDescription": "Spanish"}, {"id": 10124209, "uuid": "e8d5ce8d-375c-4838-98c4-fa444e213cfa", "attributeTranslation": "\u2022\tAE-k el\u0151fordul\u00e1sa\n\u2022\tAz AESI-k el\u0151fordul\u00e1sa\n\u2022\tSAE-k el\u0151fordul\u00e1sa\n\u2022\tBiztons\u00e1gi laborat\u00f3riumi \u00e9rt\u00e9kel\u00e9sek", "language": 13, "languageDescription": "Hungarian"}, {"id": 10124206, "uuid": "e8d5ce8d-375c-4838-98c4-fa444e213cfa", "attributeTranslation": "\u2022Incidence des EI\n\u2022 Incidence des EIIS\n\u2022 Incidence des EIG\n\u2022 Analyses de laboratoire de s\u00e9curit\u00e9", "language": 10, "languageDescription": "French"}, {"id": 10124205, "uuid": "e8d5ce8d-375c-4838-98c4-fa444e213cfa", "attributeTranslation": "\u2022\tCz\u0119sto\u015b\u0107 wyst\u0119powania zdarze\u0144 niepo\u017c\u0105danych\n\u2022\tCz\u0119sto\u015b\u0107 wyst\u0119powania zdarze\u0144 niepo\u017c\u0105danych o szczeg\u00f3lnym znaczeniu (AESI)\n\u2022\tCz\u0119sto\u015b\u0107 wyst\u0119powania 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Diseases [C05]"], "sponsor": "Italfarmaco S.p.A.", "sponsorType": "Pharmaceutical company", "trialPhase": "Therapeutic exploratory (Phase II)", "endPoint": "CORE PHASE: \u2022\tType, incidence, and severity of treatment-emergent adverse events (TEAEs) and SAEs from baseline to Week 48 \u2022\tProportion of subjects experiencing TEAEs from baseline to Week 48 \u2022\tChange from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 48 \u2022\tChange from baseline electrocardiogram (ECG) to each postbaseline visit up to Week 48, CORE PHASE: \u2022\tChange in physical function as per the Bayley III Gross Motor scale from baseline to Week 48 for subjects aged \u22652 to <3.5 years of age \u2022\tChange in physical function as per the North Star Ambulatory Assessment (NSAA) total score from baseline to Week 48 for subjects aged \u22653.5 years of age, CORE PHASE: \u2022\tChange in Paediatric Outcomes Data Collection Instrument (PODCI) from baseline to Week 48 for subjects aged \u22654 years of age in Cohort 1 only, EXTENSION PHASE: \u2022\tType, incidence, and severity of TEAEs and SAEs from baseline up to Week 144  \u2022\tProportion of patients experiencing TEAEs from baseline to Week 144 \u2022\tChange from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 144 \u2022\tChange from baseline ECG to each postbaseline up to Week 144", "product": "ITF2357", "ageRangeSecondary": [""], "ageGroup": "0-17 years", "gender": "Male", "trialRegion": 3, "totalNumberEnrolled": "10", "primaryEndPoint": "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), EXTENSION PHASE: \u2022\tType, incidence, and severity of TEAEs and SAEs from baseline up to Week 144  \u2022\tProportion of subjects experiencing TEAEs from baseline to Week 144", "resultsFirstReceived": "No", "lastUpdated": "21/03/2025", "lastPublicationUpdate": "24/04/2025"},"rawRetrieveRecord":{"ctNumber": "2024-511823-32-00", "ctStatus": "Authorised", "startDateEU": "2024-11-06", "decisionDate": "2024-10-07T15:44:11.177", "publishDate": "2025-04-24T20:31:47.557506658", "ctPublicStatusCode": 4, "authorizedApplication": {"authorizedPartI": {"id": 78845, "rowSubjectCount": 8, "rowCountriesInfo": [{"eutctId": 100000000556, "name": "United Kingdom", "isoNumber": 826, "isoAlpha2Code": "GB", "isoAlpha3Code": "GBR", "current": true}], "products": [{"id": 322860, "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": "66.6", "doseUomTotal": "g gram(s)", "maxTotalDoseAmount": "67.1", "maxTreatmentPeriod": 36, "timeUnitCode": "3", "otherMedicinalProduct": "histone deacetylase (HDAC) inhibitor", "evCode": "PRD4797678", "sponsorProductCodeEdit": "ITF2357", "devices": [], "characteristics": ["11"], "routes": ["ORAL USE"], "allSubstancesChemicals": true, "productName": "ITF2357", "jsonActiveSubstanceNames": "givinostat", "pharmaceuticalFormDisplay": "ORAL SUSPENSION"}], "trialDetails": {"clinicalTrialIdentifiers": {"fullTitle": "A Phase 2 Open label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2\u202fYears to Less Than 6 Years Old", "fullTitleTranslations": [{"id": 5856597, "uuid": "84ed9e8c-b97c-4f47-a094-34a95f3f8481", "attributeTranslation": "Een open-label fase 2-onderzoek (kernfase plus uitbreidingsfase) met 2 cohorten ter beoordeling van de farmacokinetiek en veiligheid van givinostat bij DMD-pati\u00ebnten in de leeftijd van ten minste 2 jaar tot minder dan 6 jaar oud", "language": 37, "languageDescription": "Dutch (Netherlands)"}], "publicTitle": "A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old", "publicTitleTranslations": [{"id": 5856606, "uuid": "3ab585e1-e572-4f84-9e53-ebe47aa7f692", "attributeTranslation": "Een open-label fase 2-onderzoek (kernfase plus uitbreidingsfase) met 2 cohorten ter beoordeling van de farmacokinetiek en veiligheid van givinostat bij DMD-pati\u00ebnten in de leeftijd van ten minste 2 jaar tot minder dan 6 jaar oud", "language": 37, "languageDescription": "Dutch (Netherlands)"}], "shortTitle": "DSC/14/2357/52", "secondaryIdentifyingNumbers": {"whoUniversalTrialNumber": {"id": 361377, "number": "U1111-1308-1552"}, "additionalRegistries": []}}, "trialInformation": {"trialCategory": {"isLowIntervention": false, "trialPhase": "4", "trialCategory": "2", "justificationForTrialCategory": "Phase II Duchenne Muscular Dystrophy", "trialCategoryId": 70181}, "medicalCondition": {"partIMedicalConditions": [{"id": 80278, "medicalCondition": "Duchenne muscular dystrophy (DMD)", "medicalConditionTranslations": [{"id": 5856567, "uuid": "74c64b67-9d89-463d-ba0f-bb4e42a94b2e", "attributeTranslation": "Ziekte van Duchenne (DMD)", "language": 37, "languageDescription": "Dutch (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 givinostat op lange termijn toegediend bij jongere jongens met DMD in de 2 cohorten (d.w.z. bijwerkingen [AE])", "language": 37, "languageDescription": "Dutch (Netherlands)"}], "secondaryObjectives": [{"id": 262925, "number": 1, "secondaryObjective": "CORE PHASE: To assess the safety and tolerability of givinostat administered in younger DMD boys in the 2 cohorts", "secondaryObjectiveTranslations": [{"id": 5856607, "uuid": "052db8bf-8adc-4fe4-95c5-b48d2a7f1b5e", "attributeTranslation": "Het beoordelen van de veiligheid en verdraagbaarheid van givinostat toegediend bij jongere jongens met DMD in de 2 cohorten", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 262926, "number": 2, "secondaryObjective": "CORE PHASE: To evaluate the effect of givinostat on muscular functional parameters in the 2 cohorts", "secondaryObjectiveTranslations": [{"id": 5856608, "uuid": "8b8211aa-b74e-4a26-957a-e71680003775", "attributeTranslation": "Het beoordelen van het effect van givinostat op spierfunctieparameters in 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 \u22654 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 \u2265 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 \u22652 to <6 years at screening (subjects \u22656 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 \u2265 2 tot < 6 jaar bij de screening (proefpersonen \u2265 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 corticostero\u00efden:\na. Voor proefpersonen die een stabiele dosis orale systemische corticostero\u00efden 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 corticostero\u00efdenbehandeling:\nMag niet beginnen met corticostero\u00efden 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\u2019s clinical medical judgement.", "principalInclusionCriteriaTranslations": [{"id": 5856604, "uuid": "b0180ae3-4060-4455-928e-62176446910a", "attributeTranslation": "Bij stabiele behandeling met orale systemische corticostero\u00efden zonder belangrijke verandering in dosis of doseringsschema (behalve aanpassingen als gevolg van verandering in lichaamsgewicht). Voor proefpersonen zonder corticostero\u00efden 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 \u00d7 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 -insuffici\u00ebntie, inclusief maar niet beperkt tot een verhoogd totaal bilirubine (d.w.z. > 1,5 \u00d7 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 \u00d7 ULN", "principalExclusionCriteriaTranslations": [{"id": 5856576, "uuid": "ab061dc7-7cfb-431c-b545-4111bae81799", "attributeTranslation": "Onvoldoende nierfunctie, zoals gedefinieerd met een cystatine C-serumresultaat > 2 \u00d7 ULN (opmerking: als de waarde > 2 \u00d7 ULN is, wordt de cystatine C-serumtest eenmaal herhaald; als het herhaalde testresultaat nog steeds > 2 \u00d7 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\u2019s 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\u2019s clinical medical judgement", "principalExclusionCriteriaTranslations": [{"id": 5856580, "uuid": "bbd1be31-8ef7-4c31-a708-5109b162481c", "attributeTranslation": "Bewijs van psychiatrische ziekte of sociale omstandigheden waardoor de potenti\u00eble 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 corticostero\u00efden, 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\u2019s opinion, could adversely affect subject\u2019s 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 be\u00efnvloeden, 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 \u00d7 ULN (Note: if the value is >2 \u00d7 ULN, the serum Cystatin C will be repeated once; if the repeated test result is still >2 \u00d7 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 \u00d7 ULN (opmerking: als de waarde > 2 \u00d7 ULN is, wordt de cystatine C-serumtest eenmaal herhaald; als het herhaalde testresultaat nog steeds > 2 \u00d7 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 verband houden met DMD, op basis van het klinisch-medische oordeel van de onderzoeker", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 878518, "number": 21, "principalExclusionCriteria": "CORE PHASE: Platelet count, white blood cells, and/or haemoglobin < lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results [", "principalExclusionCriteriaTranslations": [{"id": 5856587, "uuid": "75a2f924-2b30-4309-ae73-454053c7e987", "attributeTranslation": "Aantal bloedplaatjes, witte bloedcellen en/of hemoglobine < ondergrens van normaal (LLN) bij de screening (NB: voor afwijkende laboratoriumtestresultaten bij de screening [< LLN], worden het aantal bloedplaatjes, witte bloedcellen en hemoglobine eenmaal herhaald; als het herhaalde testresultaat nog steeds < LLN is, moet de proefpersoon worden uitgesloten)", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 878519, "number": 22, "principalExclusionCriteria": "CORE PHASE: Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, >1.5 \u00d7 upper limit of normal [ULN]), unless secondary to Gilbert disease or pattern consistent with Gilbert disease", "principalExclusionCriteriaTranslations": [{"id": 5856588, "uuid": "e370a7f6-5613-43b7-9e3c-e3ce8264e285", "attributeTranslation": "Huidige of voorgeschiedenis van leverziekte of -insuffici\u00ebntie, inclusief maar niet beperkt tot een baseline van verhoogd totaal bilirubine (d.w.z. > 1,5 \u00d7 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": 878520, "number": 23, "principalExclusionCriteria": "CORE PHASE: Body weight <10 kg at screening", "principalExclusionCriteriaTranslations": [{"id": 5856589, "uuid": "a535291b-4146-4c69-a8b7-dc58b217cf3b", "attributeTranslation": "Lichaamsgewicht < 10 kg bij screening", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 878521, "number": 3, "principalExclusionCriteria": "CORE PHASE: Fasting triglycerides >300 mg/dL (3.42 mmol/L) at screening (Note: if the value is >300 mg/dL, the triglycerides will be repeated once; if the repeated test result is still >300 mg/dL in fasting condition, the subject should be excluded)", "principalExclusionCriteriaTranslations": [{"id": 5856590, "uuid": "08cc2974-95e3-440f-a164-0050fd03d626", "attributeTranslation": "Nuchtere triglyceriden > 300 mg/dl (3,42 mmol/l) bij de screening (opmerking: als de waarde > 300 mg/dl is, worden de triglyceriden eenmaal herhaald; als het herhaalde testresultaat nog steeds > 300 mg/dl is in nuchtere toestand, moet de pati\u00ebnt worden uitgesloten)", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 878522, "number": 4, "principalExclusionCriteria": "CORE PHASE: Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at 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\u2019s 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\u2019s 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, hypokali\u00ebmie 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\u2019s clinical medical judgement", "principalExclusionCriteriaTranslations": [{"id": 5856593, "uuid": "70ecfe29-624f-4044-9813-e0620f8e94f4", "attributeTranslation": "Psychiatrische ziekte of sociale omstandigheden waardoor de potenti\u00eble 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": "\u2022\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\u2022\tMaximale plasmaconcentratie in stationaire toestand (Cmax,ss) na ten minste 7 dagen dosering (na week 1 en na 6 maanden)\n\u2022\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: \u2022\tType, incidence, and severity of TEAEs and SAEs from baseline up to Week 144  \u2022\tProportion of subjects experiencing TEAEs from baseline to Week 144", "isPrimary": true, "endPointTranslations": [{"id": 5856569, "uuid": "f4f82d12-e5f4-4614-b8ea-0751678cd979", "attributeTranslation": "\u2022\tType, incidentie en ernst van TEAE\u2019s en SAE\u2019s vanaf de baseline tot week 144\n\u2022\tPercentage proefpersonen dat TEAE\u2019s ervaart vanaf de baseline tot week 144", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}], "secondaryEndPoints": [{"id": 593935, "number": 1, "endPoint": "CORE PHASE: \u2022\tType, incidence, and severity of treatment-emergent adverse events (TEAEs) and SAEs from baseline to Week 48 \u2022\tProportion of subjects experiencing TEAEs from baseline to Week 48 \u2022\tChange from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 48 \u2022\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": "\u2022\tType, incidentie en ernst van tijdens de behandeling optredende AE\u2019s (TEAE\u2019s) en SAE\u2019s vanaf de baseline tot week 48\n\u2022\tPercentage proefpersonen dat TEAE\u2019s ervaart vanaf de baseline tot week 48\n\u2022\tVerandering ten opzichte van de baseline in vitale functies en klinisch laboratorium onderzoek tot elk bezoek na de baseline tot week 48\n\u2022\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: \u2022\tChange in physical function as per the Bayley III Gross Motor scale from baseline to Week 48 for subjects aged \u22652 to <3.5 years of age \u2022\tChange in physical function as per the North Star Ambulatory Assessment (NSAA) total score from baseline to Week 48 for subjects aged \u22653.5 years of age", "isPrimary": false, "endPointTranslations": [{"id": 5856571, "uuid": "66896ffe-9c55-4f8a-9714-6b12a508cb7d", "attributeTranslation": "\u2022\tVerandering in lichamelijk functioneren volgens de Bayley III Gross Motor-schaal van baseline tot week 48 voor proefpersonen van \u2265 2 tot < 3,5 jaar oud\n\u2022\tVerandering in lichamelijk functioneren volgens de totale score van de North Star Ambulatory Assessment (NSAA) vanaf de baseline tot week 48 voor proefpersonen \u2265 3,5 jaar oud", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 593937, "number": 3, "endPoint": "CORE PHASE: \u2022\tChange in Paediatric Outcomes Data Collection Instrument (PODCI) from baseline to Week 48 for subjects aged \u22654 years of age in Cohort 1 only", "isPrimary": false, "endPointTranslations": [{"id": 5856572, "uuid": "434828ee-1a0c-4829-b093-2af4272f99a6", "attributeTranslation": "\u2022\tVerandering in Pediatric Outcomes Data Collection Instrument (PODCI) vanaf baseline tot week 48, alleen voor proefpersonen van \u2265 4 jaar in cohort 1", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 593938, "number": 4, "endPoint": "EXTENSION PHASE: \u2022\tType, incidence, and severity of TEAEs and SAEs from baseline up to Week 144  \u2022\tProportion of patients experiencing TEAEs from baseline to Week 144 \u2022\tChange from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 144 \u2022\tChange from baseline ECG to each postbaseline up to Week 144", "isPrimary": false, "endPointTranslations": [{"id": 5856573, "uuid": "9cef4980-b2ab-46ef-8d7b-b2332ff48e3b", "attributeTranslation": "\u2022\tVerandering ten opzichte van de baseline in vitale functies en klinisch laboratorium onderzoek tot week 144\n\u2022\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 \u22654 to <6 years for Cohort 1 and aged \u22652 to <4 years for Cohort 2. The starting dose for Cohort 2 will be confirmed/adjusted with results of the interim analysis of Cohort 1. The study will consist of 2 phases, a core phase and an extension phase. Two final analysis will be conducted, the first at the end of the Core Phase and the second at the end of the Extension Phase (core and extension data will be combined)", "blindingMethodCode": "3", "blindedRoles": [], "armDetails": [{"description": "An open label core phase for both cohorts during which subjects will receive givinostat for 48 weeks", "title": "Test", "id": 65473}], "allocationMethod": "3"}]}}, "scientificAdviceAndPip": {"scientificAdvices": [], "paediatricInvestigationPlan": [{"id": 6569, "paediatricInvestigationNumber": "EMEA-000551-PIP04-21"}]}, "associatedClinicalTrials": [{"id": 19736, "ctNumber": "2023-503521-19-00", "sponsorName": "Italfarmaco S.p.A.", "fullTitle": "Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular Dystrophy", "sponsorAgreementOption": "NON_APPLICABLE", "sponsorAgreementOptionName": "NON_APPLICABLE", "parentClinicalTrialId": 3744, "hasDocument": false, "associatedCtDocs": []}], "references": [{"id": 361378, "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": 80278, "medicalCondition": "Duchenne muscular dystrophy (DMD)", "isConditionRareDisease": true}], "sponsors": [{"id": 85657, "primary": true, "publicContacts": [{"id": 255660, "type": "Public", "functionalName": "Patient Advocacy Representative of Italfarmaco S.p.A", "functionalEmailAddress": "patientadvocacy@italfarmacogroup.com", "telephone": "+00000", "organisation": {"id": 371837, "type": "Pharmaceutical company", "typeCode": "10", "name": "Italfarmaco S.p.A.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100000118", "organisationLocationStatus": "Active"}}], "scientificContacts": [{"id": 255661, "type": "Scientific", "functionalName": "Nicoletta Coceani \u2013 Clinical Scientist", "functionalEmailAddress": "n.coceani@italfarmacogroup.com", "telephone": "390264432512", "organisation": {"id": 371837, "type": "Pharmaceutical company", "typeCode": "10", "name": "Italfarmaco S.p.A.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100000118", "organisationLocationStatus": "Active"}}], "thirdParties": [{"id": 368324, "organisationAddress": {"id": 456446, "organisation": {"id": 488750, "type": "Non-Pharmaceutical company", "typeCode": "11", "name": "ATOM International Limited", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100042393", "organisationLocationStatus": "Active"}, "address": {"addressId": 489212, "oneLine": "Office 16, Valley House, Seventh Avenue, Team Valley Trading Estate", "addressLine1": "Office 16", "addressLine2": "Valley House", "addressLine3": "Seventh Avenue", "addressLine4": "Team Valley Trading Estate", "city": "Gateshead", "postcode": "NE11 0JW", "country": 826, "countryName": "United Kingdom"}, "phone": "000000000", "email": "michelle.eagle@atom-international.org", "isBusinessKeyValidated": true, "businessKey": "ORG-100042393"}, "sponsorDuties": [{"id": 577588, "code": "6"}], "phoneNumber": "000000000", "email": "michelle.eagle@atom-international.org"}, {"id": 368323, "organisationAddress": {"id": 454923, "organisation": {"id": 487226, "type": "Pharmaceutical company", "typeCode": "10", "name": "Alira Health", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100030303", "organisationLocationStatus": "Active"}, "address": {"addressId": 487570, "oneLine": "12 Rue Charlot", "addressLine1": "12 Rue Charlot", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Paris", "postcode": "75003", "country": 2012, "countryName": "France"}, "phone": "000000000", "email": "lisa.comarella@alirahealth.com", "isBusinessKeyValidated": true, "businessKey": "ORG-100030303"}, "sponsorDuties": [{"id": 577586, "code": "10"}, {"id": 577587, "code": "6"}], "phoneNumber": "000000000", "email": "lisa.comarella@alirahealth.com"}, {"id": 368325, "organisationAddress": {"id": 454926, "organisation": {"id": 487229, "type": "Hospital/Clinic/Other health care facility", "typeCode": "8", "name": "Syneos Health Clinique Inc.", "commercial": false, "isBusinessKeyValidated": true, "businessKey": "ORG-100028348", "organisationLocationStatus": "Active"}, "address": {"addressId": 487574, "oneLine": "2500 Einstein St", "addressLine1": "2500 Einstein St", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Quebec", "postcode": "G1P 0A2", "country": 124, "countryName": "Canada"}, "phone": "000000000", "email": "na@na.com", "isBusinessKeyValidated": true, "businessKey": "ORG-100028348"}, "sponsorDuties": [{"id": 577589, "code": "4"}], "phoneNumber": "000000000", "email": "na@na.com"}, {"id": 368321, "organisationAddress": {"id": 454907, "organisation": {"id": 487210, "type": "Pharmaceutical company", "typeCode": "10", "name": "Fortrea Inc.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100012602", "organisationLocationStatus": "Active"}, "address": {"addressId": 487551, "oneLine": "8 Moore Drive", "addressLine1": "8 Moore Drive", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Durham", "postcode": "27709-0009", "country": 840, "countryName": "United States"}, "phone": "33147168200", "email": "submissons@fortea.com", "isBusinessKeyValidated": true, "businessKey": "ORG-100012602"}, "sponsorDuties": [{"id": 577577, "code": "1"}, {"id": 577578, "code": "11"}, {"id": 577579, "code": "12"}, {"id": 577580, "code": "13"}, {"id": 577581, "code": "14"}, {"id": 577582, "code": "2"}, {"id": 577583, "code": "4"}, {"id": 577584, "code": "5"}], "phoneNumber": "33147168200", "email": "submissons@fortea.com"}, {"id": 368322, "organisationAddress": {"id": 465360, "organisation": {"id": 497682, "type": "Hospital/Clinic/Other health care facility", "typeCode": "8", "name": "Blueprint Genetics Oy", "commercial": false, "isBusinessKeyValidated": true, "businessKey": "ORG-100050758", "organisationLocationStatus": "Active"}, "address": {"addressId": 498876, "oneLine": "Keilaranta 16 A", "addressLine1": "Keilaranta 16 A", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Espoo", "postcode": "02150", "country": 2011, "countryName": "Finland"}, "phone": "000000000", "email": "adriano.saitta@blueprintgenetics.com", "isBusinessKeyValidated": true, "businessKey": "ORG-100050758"}, "sponsorDuties": [{"id": 577585, "code": "4"}], "phoneNumber": "000000000", "email": "adriano.saitta@blueprintgenetics.com"}], "organisation": {"id": 371837, "type": "Pharmaceutical company", "typeCode": "10", "name": "Italfarmaco S.p.A.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100000118", 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\u03bc\u03c5\u03ca\u03ba\u03ae \u03b4\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03b1 Duchenne, \u03c0\u03bf\u03c5 \u03ad\u03c7\u03bf\u03c5\u03bd \u03bf\u03bb\u03bf\u03ba\u03bb\u03b7\u03c1\u03ce\u03c3\u03b5\u03b9 \u03c0\u03c1\u03bf\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03b5\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b5\u03c2 \u03bc\u03b5 \u03b2\u03b1\u03bc\u03bf\u03c1\u03bf\u03bb\u03cc\u03bd\u03b7 (\u0397 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 GUARDIAN)", "language": 5, "languageDescription": "Greek"}, {"id": 8738058, "uuid": "a8847e9c-15db-4bdf-80c9-52ad28187533", "attributeTranslation": "Een openlabel onderzoek om informatie te verzamelen over de veiligheid en werkzaamheid van de behandeling op de lange termijn met vamorolon bij jongens met Duchenne spierdystrofie (DMD) die eerdere onderzoeken met vamorolon hebben voltooid (het GUARDIAN-onderzoek)", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738059, "uuid": "a8847e9c-15db-4bdf-80c9-52ad28187533", "attributeTranslation": "Estudio abierto para recopilar informaci\u00f3n relativa a seguridad y eficacia del tratamiento prolongado con vamorolona en chicos con distrofia muscular de Duchenne que han completado estudios previos con vamorolona Estudio GUARDIAN", "language": 7, "languageDescription": "Spanish"}], "shortTitle": "SNT-IV-VAM-011", "secondaryIdentifyingNumbers": {"additionalRegistries": []}}, "trialInformation": {"trialCategory": {"isLowIntervention": false, "trialPhase": "6", "trialCategory": "3", "justificationForTrialCategory": "This is a phase IV study, thus under trial category 3. This is an open-label, multi-center trial designed to collect long-term safety and effectiveness data from boys with Duchenne Muscular Dystrophy (DMD) who have participated in studies VBP15-002/003/LTE and VBP15-004 of vamorolone clinical development program.", "trialCategoryId": 98635}, "medicalCondition": {"partIMedicalConditions": [{"id": 112113, "medicalCondition": "Duchenne Muscular Dystrophy", "medicalConditionTranslations": [{"id": 8737986, "uuid": "8f38bb37-753c-4552-95f9-f2e20deadff0", "attributeTranslation": "\u039c\u03c5\u03ca\u03ba\u03ae \u03b4\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03b1 Duchenne", "language": 5, "languageDescription": "Greek"}, {"id": 8737985, "uuid": "8f38bb37-753c-4552-95f9-f2e20deadff0", "attributeTranslation": "Distrofia muscular de Duchenne (DMD)", "language": 7, "languageDescription": "Spanish"}, {"id": 8737987, "uuid": "8f38bb37-753c-4552-95f9-f2e20deadff0", "attributeTranslation": "Duchenne spierdystrofie", "language": 37, "languageDescription": "Dutch (Netherlands)"}], "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": 326379}, {"code": "5", "trialScopeId": 326378}], "mainObjective": "To evaluate the safety of long-term treatment with vamorolone in boys with DMD regarding vertebral fractures", "mainObjectiveTranslations": [{"id": 8738054, "uuid": "971dd6fd-fcb5-448f-83ea-d66e8baab4d7", "attributeTranslation": "Het beoordelen van de veiligheid van de behandeling op de lange termijn met vamorolon bij jongens met DMD met betrekking tot wervelfracturen.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738055, "uuid": "971dd6fd-fcb5-448f-83ea-d66e8baab4d7", "attributeTranslation": "Evaluar la seguridad del tratamiento prolongado con vamorolona en chicos con DMD en lo que respecta a fracturas vertebrales", "language": 7, "languageDescription": "Spanish"}, {"id": 8738056, "uuid": "971dd6fd-fcb5-448f-83ea-d66e8baab4d7", "attributeTranslation": "\u0397 \u03b1\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1\u03c2 \u03c4\u03b7\u03c2 \u03bc\u03b1\u03ba\u03c1\u03bf\u03c7\u03c1\u03cc\u03bd\u03b9\u03b1\u03c2 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1\u03c2 \u03bc\u03b5 \u03b2\u03b1\u03bc\u03bf\u03c1\u03bf\u03bb\u03cc\u03bd\u03b7 \u03c3\u03b5 \u03b1\u03b3\u03cc\u03c1\u03b9\u03b1 \u03bc\u03b5 DMD \u03c3\u03b5 \u03c3\u03c7\u03ad\u03c3\u03b7 \u03bc\u03b5 \u03c3\u03c0\u03bf\u03bd\u03b4\u03c5\u03bb\u03b9\u03ba\u03ac \u03ba\u03b1\u03c4\u03ac\u03b3\u03bc\u03b1\u03c4\u03b1", "language": 5, "languageDescription": "Greek"}], "secondaryObjectives": [{"id": 376100, "number": 1, "secondaryObjective": "To evaluate the safety of long-term treatment with vamorolone in boys with DMD on non-vertebral fractures, cataracts, and delayed puberty.", "secondaryObjectiveTranslations": [{"id": 8738061, "uuid": "d8802a8e-6cfd-44fb-85fa-4b8238b39bfc", "attributeTranslation": "\u0397 \u03b1\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1\u03c2 \u03c4\u03b7\u03c2 \u03bc\u03b1\u03ba\u03c1\u03bf\u03c7\u03c1\u03cc\u03bd\u03b9\u03b1\u03c2 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1\u03c2 \u03bc\u03b5 \u03b2\u03b1\u03bc\u03bf\u03c1\u03bf\u03bb\u03cc\u03bd\u03b7 \u03c3\u03b5 \u03b1\u03b3\u03cc\u03c1\u03b9\u03b1 \u03bc\u03b5 DMD \u03c3\u03b5 \u03c3\u03c7\u03ad\u03c3\u03b7 \u03bc\u03b5 \u03bc\u03b7 \u03c3\u03c0\u03bf\u03bd\u03b4\u03c5\u03bb\u03b9\u03ba\u03ac \u03ba\u03b1\u03c4\u03ac\u03b3\u03bc\u03b1\u03c4\u03b1, \u03ba\u03b1\u03c4\u03b1\u03c1\u03c1\u03ac\u03ba\u03c4\u03b7 \u03ba\u03b1\u03b9 \u03ba\u03b1\u03b8\u03c5\u03c3\u03c4\u03b5\u03c1\u03b7\u03bc\u03ad\u03bd\u03b7 \u03b5\u03c6\u03b7\u03b2\u03b5\u03af\u03b1.", "language": 5, "languageDescription": "Greek"}, {"id": 8738062, "uuid": "d8802a8e-6cfd-44fb-85fa-4b8238b39bfc", "attributeTranslation": "Evaluar la seguridad del tratamiento prolongado con vamorolona en chicos con 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": "\u0397 \u03b1\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03c3\u03c5\u03bd\u03bf\u03bb\u03b9\u03ba\u03ae\u03c2 \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1\u03c2 \u03c4\u03b7\u03c2 \u03bc\u03b1\u03ba\u03c1\u03bf\u03c7\u03c1\u03cc\u03bd\u03b9\u03b1\u03c2 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1\u03c2 \u03bc\u03b5 \u03b2\u03b1\u03bc\u03bf\u03c1\u03bf\u03bb\u03cc\u03bd\u03b7 \u03c3\u03b5 \u03b1\u03b3\u03cc\u03c1\u03b9\u03b1 \u03bc\u03b5 DMD", "language": 5, "languageDescription": "Greek"}, {"id": 8738064, "uuid": "a939a4d7-d7e3-49e7-8205-790e8a06a37f", "attributeTranslation": "Evaluar la seguridad global del tratamiento prolongado con vamorolona en chicos con DMD", "language": 7, "languageDescription": "Spanish"}]}, {"id": 376102, "number": 3, "secondaryObjective": "To evaluate the long-term treatment effect of vamorolone on  ambulatory and non-ambulatory function", "secondaryObjectiveTranslations": [{"id": 8738067, "uuid": "a7590339-8aef-4a56-adf6-ab64d738caf5", "attributeTranslation": "\u0397 \u03b1\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03bc\u03b1\u03ba\u03c1\u03bf\u03c7\u03c1\u03cc\u03bd\u03b9\u03b1\u03c2 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03c5\u03c4\u03b9\u03ba\u03ae\u03c2 \u03b5\u03c0\u03af\u03b4\u03c1\u03b1\u03c3\u03b7\u03c2 \u03c4\u03b7\u03c2 \u03b2\u03b1\u03bc\u03bf\u03c1\u03bf\u03bb\u03cc\u03bd\u03b7\u03c2 \u03c3\u03c4\u03b7\u03bd \u03c0\u03b5\u03c1\u03b9\u03c0\u03b1\u03c4\u03b7\u03c4\u03b9\u03ba\u03ae \u03ba\u03b1\u03b9 \u03bc\u03b7 \u03c0\u03b5\u03c1\u03b9\u03c0\u03b1\u03c4\u03b7\u03c4\u03b9\u03ba\u03ae \u03bb\u03b5\u03b9\u03c4\u03bf\u03c5\u03c1\u03b3\u03af\u03b1", "language": 5, "languageDescription": "Greek"}, {"id": 8738066, "uuid": "a7590339-8aef-4a56-adf6-ab64d738caf5", "attributeTranslation": "Het beoordelen van het behandelingseffect op de lange termijn van vamorolon op ambulante en niet-ambulante functies.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738068, "uuid": "a7590339-8aef-4a56-adf6-ab64d738caf5", "attributeTranslation": "Evaluar el efecto del tratamiento prolongado con vamorolona sobre la funci\u00f3n deambulatoria y no deambulatoria", "language": 7, "languageDescription": "Spanish"}]}]}, "eligibilityCriteria": {"principalInclusionCriteria": [{"id": 721800, "number": 1, "principalInclusionCriteria": "Subject and/or subject\u2019s parent(s) or legal guardian has provided written informed consent", "principalInclusionCriteriaTranslations": [{"id": 8738043, "uuid": "08dd0957-d18b-4c00-8b13-7afcad5eadcd", "attributeTranslation": "El sujeto y/o sus padres o su tutor legal han otorgado su consentimiento informado por escrito", "language": 7, "languageDescription": "Spanish"}, {"id": 8738044, "uuid": "08dd0957-d18b-4c00-8b13-7afcad5eadcd", "attributeTranslation": "De proefpersoon en/of de ouder(s) of wettelijke vertegenwoordiger van de proefpersoon heeft/hebben schriftelijke ge\u00efnformeerde toestemming gegeven.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738042, "uuid": "08dd0957-d18b-4c00-8b13-7afcad5eadcd", "attributeTranslation": "\u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03ae/\u03ba\u03b1\u03b9 \u03bf(\u03bf\u03b9) \u03b3\u03bf\u03bd\u03ad\u03b1\u03c2(-\u03b5\u03af\u03c2) \u03ae \u03bf \u03bd\u03cc\u03bc\u03b9\u03bc\u03bf\u03c2 \u03ba\u03b7\u03b4\u03b5\u03bc\u03cc\u03bd\u03b1\u03c2 \u03c4\u03bf\u03c5 \u03b1\u03c3\u03b8\u03b5\u03bd\u03bf\u03cd\u03c2 \u03ad\u03c7\u03b5\u03b9 \u03c0\u03b1\u03c1\u03ac\u03c3\u03c7\u03b5\u03b9 \u03b3\u03c1\u03b1\u03c0\u03c4\u03ae \u03c3\u03c5\u03bd\u03b1\u03af\u03bd\u03b5\u03c3\u03b7 \u03bc\u03b5\u03c4\u03ac \u03b1\u03c0\u03cc \u03b5\u03bd\u03b7\u03bc\u03ad\u03c1\u03c9\u03c3\u03b7", "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": "\u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03ad\u03c7\u03b5\u03b9 \u03c0\u03c1\u03bf\u03b7\u03b3\u03bf\u03c5\u03bc\u03ad\u03bd\u03c9\u03c2 \u03bf\u03bb\u03bf\u03ba\u03bb\u03b7\u03c1\u03ce\u03c3\u03b5\u03b9 \u03b5\u03af\u03c4\u03b5 \u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 VBP15-LTE \u03b5\u03af\u03c4\u03b5 \u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 VBP15-004 \u03ba\u03b1\u03b9 \u03ad\u03c7\u03b5\u03b9 \u03bc\u03b5\u03c4\u03b1\u03b2\u03b5\u03af \u03bc\u03ad\u03c3\u03c9 \u03c4\u03c9\u03bd \u03c0\u03c1\u03bf\u03b3\u03c1\u03b1\u03bc\u03bc\u03ac\u03c4\u03c9\u03bd CUP, NPP \u03ae 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 est\u00e1 recibiendo vamorolona el d\u00eda del reclutamiento", "language": 7, "languageDescription": "Spanish"}, {"id": 8738048, "uuid": "decf361d-17f2-4d3d-8008-5866d57fd27a", "attributeTranslation": "\u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03bb\u03b1\u03bc\u03b2\u03ac\u03bd\u03b5\u03b9 \u03b2\u03b1\u03bc\u03bf\u03c1\u03bf\u03bb\u03cc\u03bd\u03b7 \u03c4\u03b7\u03bd \u03b7\u03bc\u03ad\u03c1\u03b1 \u03c4\u03b7\u03c2 \u03ad\u03bd\u03c4\u03b1\u03be\u03b7\u03c2", "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": "\u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03ba\u03b1\u03b9 \u03bf \u03b3\u03bf\u03bd\u03ad\u03b1\u03c2/\u03bd\u03cc\u03bc\u03b9\u03bc\u03bf\u03c2 \u03ba\u03b7\u03b4\u03b5\u03bc\u03cc\u03bd\u03b1\u03c2 \u03b5\u03af\u03bd\u03b1\u03b9 \u03c0\u03c1\u03cc\u03b8\u03c5\u03bc\u03bf\u03b9 \u03ba\u03b1\u03b9 \u03b9\u03ba\u03b1\u03bd\u03bf\u03af \u03bd\u03b1 \u03c3\u03c5\u03bc\u03bc\u03bf\u03c1\u03c6\u03c9\u03b8\u03bf\u03cd\u03bd \u03bc\u03b5 \u03c4\u03bf \u03c7\u03c1\u03bf\u03bd\u03bf\u03b4\u03b9\u03ac\u03b3\u03c1\u03b1\u03bc\u03bc\u03b1, \u03c4\u03b9\u03c2 \u03b1\u03be\u03b9\u03bf\u03bb\u03bf\u03b3\u03ae\u03c3\u03b5\u03b9\u03c2 \u03ba\u03b1\u03b9 \u03c4\u03b9\u03c2 \u03b1\u03c0\u03b1\u03b9\u03c4\u03ae\u03c3\u03b5\u03b9\u03c2 \u03c4\u03bf\u03c5 \u03c0\u03c1\u03c9\u03c4\u03bf\u03ba\u03cc\u03bb\u03bb\u03bf\u03c5", "language": 5, "languageDescription": "Greek"}, {"id": 8738051, "uuid": "15ffa453-3d1e-4440-af9d-04d3cff22fb0", "attributeTranslation": "El sujeto y sus padres/tutor legal pueden y est\u00e1n 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": "\u039f\u03c0\u03bf\u03b9\u03b1\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03b9\u03b1\u03c4\u03c1\u03b9\u03ba\u03ae \u03c0\u03ac\u03b8\u03b7\u03c3\u03b7, \u03b7 \u03bf\u03c0\u03bf\u03af\u03b1, \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b3\u03bd\u03ce\u03bc\u03b7 \u03c4\u03bf\u03c5 \u03b5\u03c1\u03b5\u03c5\u03bd\u03b7\u03c4\u03ae, \u03b8\u03b1 \u03b5\u03c0\u03b7\u03c1\u03ad\u03b1\u03b6\u03b5 \u03c4\u03b7 \u03c3\u03c5\u03bc\u03bc\u03b5\u03c4\u03bf\u03c7\u03ae \u03c3\u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7, \u03c4\u03b7\u03bd \u03b1\u03c0\u03cc\u03b4\u03bf\u03c3\u03b7 \u03ae \u03c4\u03b7\u03bd \u03b5\u03c1\u03bc\u03b7\u03bd\u03b5\u03af\u03b1 \u03c4\u03c9\u03bd \u03b1\u03be\u03b9\u03bf\u03bb\u03bf\u03b3\u03ae\u03c3\u03b5\u03c9\u03bd \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2", "language": 5, "languageDescription": "Greek"}, {"id": 8738030, "uuid": "46e141c2-33e7-42e8-a2c9-00f021223ada", "attributeTranslation": "Elke medische aandoening die, naar het oordeel van de onderzoeker, deelname aan of uitvoering van het onderzoek of de interpretatie van de onderzoeksbeoordelingen zou be\u00efnvloeden.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738032, "uuid": "46e141c2-33e7-42e8-a2c9-00f021223ada", "attributeTranslation": "Cualquier trastorno m\u00e9dico que, en opini\u00f3n del investigador, pueda afectar a la participaci\u00f3n en el estudio o a la realizaci\u00f3n o interpretaci\u00f3n de las evaluaciones del estudio.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 1238563, "number": 2, "principalExclusionCriteria": "Vamorolone treatment discontinued for \u22656 months within the year  prior to enrolment for a non-safety reason, or vamorolone treatment  previously discontinued at any time for a safety reason", "principalExclusionCriteriaTranslations": [{"id": 8738035, "uuid": "bd111b46-d30a-49c8-895a-23843251279c", "attributeTranslation": "Behandeling met vamorolon die gedurende \u2265 6 maanden binnen het jaar voorafgaand aan inschrijving werd stopgezet om een andere reden dan vanwege de veiligheid, of behandeling met vamorolon die eerder op enig moment vanwege veiligheidsredenen werd stopgezet.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738033, "uuid": "bd111b46-d30a-49c8-895a-23843251279c", "attributeTranslation": "\u0397 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1 \u03bc\u03b5 \u03b2\u03b1\u03bc\u03bf\u03c1\u03bf\u03bb\u03cc\u03bd\u03b7 \u03b4\u03b9\u03b1\u03ba\u03cc\u03c0\u03b7\u03ba\u03b5 \u03b3\u03b9\u03b1 \u22656 \u03bc\u03ae\u03bd\u03b5\u03c2 \u03b5\u03bd\u03c4\u03cc\u03c2 \u03c4\u03bf\u03c5 \u03ad\u03c4\u03bf\u03c5\u03c2 \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03c4\u03b7\u03bd \u03ad\u03bd\u03c4\u03b1\u03be\u03b7 \u03b3\u03b9\u03b1 \u03bb\u03cc\u03b3\u03bf \u03bc\u03b7 \u03c3\u03c7\u03b5\u03c4\u03b9\u03b6\u03cc\u03bc\u03b5\u03bd\u03bf \u03bc\u03b5 \u03c4\u03b7\u03bd \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1 \u03ae \u03b7 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1 \u03bc\u03b5 \u03b2\u03b1\u03bc\u03bf\u03c1\u03bf\u03bb\u03cc\u03bd\u03b7 \u03b4\u03b9\u03b1\u03ba\u03cc\u03c0\u03b7\u03ba\u03b5 \u03c0\u03c1\u03bf\u03b7\u03b3\u03bf\u03c5\u03bc\u03ad\u03bd\u03c9\u03c2 \u03bf\u03c0\u03bf\u03b9\u03b1\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03c3\u03c4\u03b9\u03b3\u03bc\u03ae \u03b3\u03b9\u03b1 \u03bb\u03cc\u03b3\u03bf \u03c3\u03c7\u03b5\u03c4\u03b9\u03b6\u03cc\u03bc\u03b5\u03bd\u03bf \u03bc\u03b5 \u03c4\u03b7\u03bd \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1", "language": 5, "languageDescription": "Greek"}, {"id": 8738034, "uuid": "bd111b46-d30a-49c8-895a-23843251279c", "attributeTranslation": "Suspensi\u00f3n del tratamiento con vamorolona durante \u2265 6 meses en el a\u00f1o previo al reclutamiento por un motivo no relacionado con la seguridad o suspensi\u00f3n previa del tratamiento con vamorolona en cualquier momento por un motivo de seguridad", "language": 7, "languageDescription": "Spanish"}]}, {"id": 1238564, "number": 3, "principalExclusionCriteria": "Severe hepatic impairment", "principalExclusionCriteriaTranslations": [{"id": 8738036, "uuid": "819d1773-e0d5-480d-8b8f-8d0f402bc9f0", "attributeTranslation": "Ernstige leverinsuffici\u00ebntie.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738037, "uuid": "819d1773-e0d5-480d-8b8f-8d0f402bc9f0", "attributeTranslation": "Insuficiencia hep\u00e1tica grave", "language": 7, "languageDescription": "Spanish"}, {"id": 8738038, "uuid": "819d1773-e0d5-480d-8b8f-8d0f402bc9f0", "attributeTranslation": "\u03a3\u03bf\u03b2\u03b1\u03c1\u03ae \u03b7\u03c0\u03b1\u03c4\u03b9\u03ba\u03ae \u03b4\u03c5\u03c3\u03bb\u03b5\u03b9\u03c4\u03bf\u03c5\u03c1\u03b3\u03af\u03b1", "language": 5, "languageDescription": "Greek"}]}]}, "endPoint": {"primaryEndPoints": [{"id": 845139, "number": 1, "endPoint": "Number of vertebral fractures per 1000 person-years based on X- ray central reading", "isPrimary": true, "endPointTranslations": [{"id": 8737988, "uuid": "ada8ca76-4542-4552-b418-058af6ef89cf", "attributeTranslation": "N\u00famero de fracturas vertebrales por 1000 a\u00f1os-persona seg\u00fan la interpretaci\u00f3n radiol\u00f3gica centralizada", "language": 7, "languageDescription": "Spanish"}, {"id": 8737990, "uuid": "ada8ca76-4542-4552-b418-058af6ef89cf", "attributeTranslation": "\u0391\u03c1\u03b9\u03b8\u03bc\u03cc\u03c2 \u03c3\u03c0\u03bf\u03bd\u03b4\u03c5\u03bb\u03b9\u03ba\u03ce\u03bd \u03ba\u03b1\u03c4\u03b1\u03b3\u03bc\u03ac\u03c4\u03c9\u03bd \u03b1\u03bd\u03ac 1000 \u03b1\u03bd\u03b8\u03c1\u03c9\u03c0\u03bf\u03ad\u03c4\u03b7 \u03bc\u03b5 \u03b2\u03ac\u03c3\u03b7 \u03c4\u03b7\u03bd \u03ba\u03b5\u03bd\u03c4\u03c1\u03b9\u03ba\u03ae \u03b1\u03bd\u03ac\u03b3\u03bd\u03c9\u03c3\u03b7 \u03c4\u03c9\u03bd \u03b1\u03ba\u03c4\u03b9\u03bd\u03bf\u03b3\u03c1\u03b1\u03c6\u03b9\u03ce\u03bd", "language": 5, "languageDescription": "Greek"}, {"id": 8737989, "uuid": "ada8ca76-4542-4552-b418-058af6ef89cf", "attributeTranslation": "Aantal wervelfracturen per 1000 manjaren op basis van centrale r\u00f6ntgenuitlezing.", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}], "secondaryEndPoints": [{"id": 845140, "number": 1, "endPoint": "Time to first vertebral fractures (cumulative incidence)", "isPrimary": false, "endPointTranslations": [{"id": 8737992, "uuid": "66a8c324-1e05-41f4-9b6a-6f72e40b3436", "attributeTranslation": "Tiempo hasta la primera fractura vertebral (incidencia acumulada)", "language": 7, "languageDescription": "Spanish"}, {"id": 8737993, "uuid": "66a8c324-1e05-41f4-9b6a-6f72e40b3436", "attributeTranslation": "Tijd tot eerste wervelfracturen (cumulatieve incidentie).", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8737991, "uuid": "66a8c324-1e05-41f4-9b6a-6f72e40b3436", "attributeTranslation": "\u03a7\u03c1\u03cc\u03bd\u03bf\u03c2 \u03ad\u03c9\u03c2 \u03c4\u03bf \u03c0\u03c1\u03ce\u03c4\u03bf \u03c3\u03c0\u03bf\u03bd\u03b4\u03c5\u03bb\u03b9\u03ba\u03cc \u03ba\u03ac\u03c4\u03b1\u03b3\u03bc\u03b1 (\u03c3\u03c9\u03c1\u03b5\u03c5\u03c4\u03b9\u03ba\u03ae \u03b5\u03c0\u03af\u03c0\u03c4\u03c9\u03c3\u03b7)", "language": 5, "languageDescription": "Greek"}]}, {"id": 845141, "number": 2, "endPoint": "Number of non-vertebral fractures per 1000 person-years based  on investigator reporting", "isPrimary": false, "endPointTranslations": [{"id": 8737995, "uuid": "ee941788-93a1-4a9c-9306-deef1a9dbdc1", "attributeTranslation": "\u0391\u03c1\u03b9\u03b8\u03bc\u03cc\u03c2 \u03bc\u03b7 \u03c3\u03c0\u03bf\u03bd\u03b4\u03c5\u03bb\u03b9\u03ba\u03ce\u03bd \u03ba\u03b1\u03c4\u03b1\u03b3\u03bc\u03ac\u03c4\u03c9\u03bd \u03b1\u03bd\u03ac 1000 \u03b1\u03bd\u03b8\u03c1\u03c9\u03c0\u03bf\u03ad\u03c4\u03b7 \u03bc\u03b5 \u03b2\u03ac\u03c3\u03b7 \u03c4\u03b7\u03bd \u03b1\u03bd\u03b1\u03c6\u03bf\u03c1\u03ac \u03c4\u03bf\u03c5 \u03b5\u03c1\u03b5\u03c5\u03bd\u03b7\u03c4\u03ae", "language": 5, "languageDescription": "Greek"}, {"id": 8737994, "uuid": "ee941788-93a1-4a9c-9306-deef1a9dbdc1", "attributeTranslation": "Aantal niet-wervelfracturen per 1000 manjaren op basis van rapportage door de onderzoeker.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8737996, "uuid": "ee941788-93a1-4a9c-9306-deef1a9dbdc1", "attributeTranslation": "N\u00famero de fracturas no vertebrales por 1000 a\u00f1os-persona seg\u00fan la evaluaci\u00f3n del investigador", "language": 7, "languageDescription": "Spanish"}]}, {"id": 845142, "number": 3, "endPoint": "Time to first non-vertebral fractures (cumulative incidence)", "isPrimary": false, "endPointTranslations": [{"id": 8737999, "uuid": "03ae3fbc-2e93-4e15-b95e-0389736ca096", "attributeTranslation": "Tijd tot eerste niet-wervelfracturen (cumulatieve incidentie).", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8737997, "uuid": "03ae3fbc-2e93-4e15-b95e-0389736ca096", "attributeTranslation": "Tiempo hasta la primera fractura no vertebral (incidencia acumulada)", "language": 7, "languageDescription": "Spanish"}, {"id": 8737998, "uuid": "03ae3fbc-2e93-4e15-b95e-0389736ca096", "attributeTranslation": "\u03a7\u03c1\u03cc\u03bd\u03bf\u03c2 \u03ad\u03c9\u03c2 \u03c4\u03bf \u03c0\u03c1\u03ce\u03c4\u03bf \u03bc\u03b7 \u03c3\u03c0\u03bf\u03bd\u03b4\u03c5\u03bb\u03b9\u03ba\u03cc \u03ba\u03ac\u03c4\u03b1\u03b3\u03bc\u03b1 (\u03c3\u03c9\u03c1\u03b5\u03c5\u03c4\u03b9\u03ba\u03ae \u03b5\u03c0\u03af\u03c0\u03c4\u03c9\u03c3\u03b7)", "language": 5, "languageDescription": "Greek"}]}, {"id": 845143, "number": 4, "endPoint": "Number of cataracts per 1000 person-years based on ophthalmologist assessment", "isPrimary": false, "endPointTranslations": [{"id": 8738001, "uuid": "ff788f34-2af4-4e49-878c-d21abebd4c36", "attributeTranslation": "Aantal staargevallen per 1000 manjaren op basis van beoordeling door oogarts.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738000, "uuid": "ff788f34-2af4-4e49-878c-d21abebd4c36", "attributeTranslation": "N\u00famero de cataratas por 1000 a\u00f1os-persona seg\u00fan la evaluaci\u00f3n del oftalm\u00f3logo", "language": 7, "languageDescription": "Spanish"}, {"id": 8738002, "uuid": "ff788f34-2af4-4e49-878c-d21abebd4c36", "attributeTranslation": "\u0391\u03c1\u03b9\u03b8\u03bc\u03cc\u03c2 \u03c0\u03b5\u03c1\u03b9\u03c3\u03c4\u03b1\u03c4\u03b9\u03ba\u03ce\u03bd \u03ba\u03b1\u03c4\u03b1\u03c1\u03c1\u03ac\u03ba\u03c4\u03b7 \u03b1\u03bd\u03ac 1000 \u03b1\u03bd\u03b8\u03c1\u03c9\u03c0\u03bf\u03ad\u03c4\u03b7 \u03bc\u03b5 \u03b2\u03ac\u03c3\u03b7 \u03c4\u03b7\u03bd \u03bf\u03c6\u03b8\u03b1\u03bb\u03bc\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ae \u03b1\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7", "language": 5, "languageDescription": "Greek"}]}, {"id": 845144, "number": 5, "endPoint": "Number of subjects not reaching Tanner stage 2 by 15 years of age", "isPrimary": false, "endPointTranslations": [{"id": 8738004, "uuid": "31e2f304-137f-4094-b80d-78266c2459c1", "attributeTranslation": "Aantal proefpersonen dat op 15-jarige leeftijd Tanner-stadium 2 niet bereikt.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738003, "uuid": "31e2f304-137f-4094-b80d-78266c2459c1", "attributeTranslation": "N\u00famero de sujetos que no hayan alcanzado el estadio 2 de Tanner a los 15 a\u00f1os de edad", "language": 7, "languageDescription": "Spanish"}, {"id": 8738005, "uuid": "31e2f304-137f-4094-b80d-78266c2459c1", "attributeTranslation": "\u0391\u03c1\u03b9\u03b8\u03bc\u03cc\u03c2 \u03b1\u03c3\u03b8\u03b5\u03bd\u03ce\u03bd \u03c0\u03bf\u03c5 \u03b4\u03b5\u03bd \u03ad\u03c6\u03c4\u03b1\u03c3\u03b1\u03bd \u03c3\u03b5 \u03c3\u03c4\u03ac\u03b4\u03b9\u03bf Tanner 2 \u03ad\u03c9\u03c2 \u03c4\u03b7\u03bd \u03b7\u03bb\u03b9\u03ba\u03af\u03b1 \u03c4\u03c9\u03bd 15 \u03b5\u03c4\u03ce\u03bd", "language": 5, "languageDescription": "Greek"}]}, {"id": 845145, "number": 6, "endPoint": "Frequency of adverse events (AEs) and serious adverse events (SAEs)", "isPrimary": false, "endPointTranslations": [{"id": 8738006, "uuid": "cb00984f-c064-425f-9c2c-2809e0ea0a40", "attributeTranslation": "Frecuencia de acontecimientos adversos (AA) y acontecimientos adversos graves (AAG)", "language": 7, "languageDescription": "Spanish"}, {"id": 8738007, "uuid": "cb00984f-c064-425f-9c2c-2809e0ea0a40", "attributeTranslation": "Frequentie van bijwerkingen (AE\u2019s) en ernstige bijwerkingen (SAE\u2019s).", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738008, "uuid": "cb00984f-c064-425f-9c2c-2809e0ea0a40", "attributeTranslation": "\u03a3\u03c5\u03c7\u03bd\u03cc\u03c4\u03b7\u03c4\u03b1 \u03b1\u03bd\u03b5\u03c0\u03b9\u03b8\u03cd\u03bc\u03b7\u03c4\u03c9\u03bd \u03c3\u03c5\u03bc\u03b2\u03ac\u03bd\u03c4\u03c9\u03bd (\u0391\u03a3) \u03ba\u03b1\u03b9 \u03c3\u03bf\u03b2\u03b1\u03c1\u03ce\u03bd \u03b1\u03bd\u03b5\u03c0\u03b9\u03b8\u03cd\u03bc\u03b7\u03c4\u03c9\u03bd \u03c3\u03c5\u03bc\u03b2\u03ac\u03bd\u03c4\u03c9\u03bd (\u03a3\u0391\u03a3)", "language": 5, "languageDescription": "Greek"}]}, {"id": 845146, "number": 7, "endPoint": "Change from baseline in body weight, height and body mass index (BMI)", "isPrimary": false, "endPointTranslations": [{"id": 8738009, "uuid": "c99dc9bd-e4a5-4d4d-9aea-6d163814857c", "attributeTranslation": "Variaci\u00f3n con respecto al valor basal del peso corporal, la estatura y el \u00edndice de masa corporal (IMC)", "language": 7, "languageDescription": "Spanish"}, {"id": 8738011, "uuid": "c99dc9bd-e4a5-4d4d-9aea-6d163814857c", "attributeTranslation": "Verandering ten opzichte van de baseline in lichaamsgewicht, lengte en body mass index (BMI).", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 8738010, "uuid": "c99dc9bd-e4a5-4d4d-9aea-6d163814857c", "attributeTranslation": "\u039c\u03b5\u03c4\u03b1\u03b2\u03bf\u03bb\u03ae \u03c3\u03b5 \u03c3\u03c7\u03ad\u03c3\u03b7 \u03bc\u03b5 \u03c4\u03b7\u03bd \u03b1\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03b1\u03bd\u03b1\u03c6\u03bf\u03c1\u03ac\u03c2 \u03c4\u03bf\u03c5 \u03c3\u03c9\u03bc\u03b1\u03c4\u03b9\u03ba\u03bf\u03cd \u03b2\u03ac\u03c1\u03bf\u03c5\u03c2, \u03c4\u03bf\u03c5 \u03cd\u03c8\u03bf\u03c5\u03c2 \u03ba\u03b1\u03b9 \u03c4\u03bf\u03c5 \u03b4\u03b5\u03af\u03ba\u03c4\u03b7 \u03bc\u03ac\u03b6\u03b1\u03c2 \u03c3\u03ce\u03bc\u03b1\u03c4\u03bf\u03c2 (\u0394\u039c\u03a3)", "language": 5, "languageDescription": "Greek"}]}, {"id": 845147, "number": 8, "endPoint": "Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisol", "isPrimary": false, "endPointTranslations": [{"id": 8738014, "uuid": "ebe6b197-1cc5-4f11-90e5-02f6dd4ce241", "attributeTranslation": "\u0391\u03c1\u03b9\u03b8\u03bc\u03cc\u03c2 \u03b1\u03c3\u03b8\u03b5\u03bd\u03ce\u03bd \u03bc\u03b5 \u03ba\u03bb\u03b9\u03bd\u03b9\u03ba\u03ac \u03c3\u03b7\u03bc\u03b1\u03bd\u03c4\u03b9\u03ba\u03ad\u03c2 \u03b5\u03c1\u03b3\u03b1\u03c3\u03c4\u03b7\u03c1\u03b9\u03b1\u03ba\u03ad\u03c2 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"After receipt of the Dear Investigator Letter: \u201cDelandistrogene moxeparvovec: fatal case of acute liver failure and intracranial hemorrhage\u201d dated 18 Mar 2025, we ask the Sponsor to immediately put on hold enrollment in France, until analysis of the fatal case has been finalised by the RMSs.\n\nThe temporay halt was already notified in the other european countries concerned, except France as the clinical trial had not started. The sponsor should confirm that the enrollment, is put on hold in France.\n\nFurthermore, the Sponsor has to notify this fatal case through the EU portal without undue delay.\n\n Indeed and according to the article 53 of the REGULATION (EU) No 536/2014 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC, the sponsor shall notify the Member States concerned through the EU portal of all unexpected events which affect the benefit-risk balance of the clinical trial, but are not suspected unexpected serious adverse reactions as referred to in Article 42. That notification shall be made without undue delay but no later than 15 days from the date the sponsor became aware of this event.", "isImmediateActionRequired": true, "memberStateConcerned": {"id": 28446, "mscName": "France", "countryName": "France"}, "sponsorSubmitDate": "2025-04-02"}]}},{"_id":13,"ctNumber":"2024-512383-65-00","ctStatusCode":5,"ctStatus":"Authorised","ctTitle":"A Phase II Multicenter, Open-Label Study to evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Satralizumab in Pediatric Patients with Duchenne Muscular Dystrophy (SHIELD DMD)","shortTitle":"BN45398","conditions":"Duchenne Muscular Dystrophy (DMD)","trialCountries":["Poland:5", "Spain:5", "Italy:5", "Denmark:5"],"decisionDateOverall":"09/09/2024","decisionDateByCountry":"PL: 16/09/2024, ES: 09/09/2024, IT: 10/09/2024, DK: 22/09/2025","therapeuticAreas":["Diseases [C] - Musculoskeletal Diseases [C05]", "Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]", "Diseases [C] - Nervous System Diseases [C10]"],"sponsor":"F. Hoffmann-La Roche AG","sponsorType":"Pharmaceutical company","trialPhase":"Therapeutic exploratory (Phase II)","endPoint":"1. Incidence of treatment-emergent adverse events, 2. Incidence of serious adverse events, 3. Incidence of adverse events of special interest, 4. Clinically significant changes in vital signs and physical examination findings, 5. Clinically significant changes in safety laboratory assessments, ECGs, 6. Change from baseline to Weeks 24, 52 and 104 in LS BMD Z-score measured by DEXA, 7. Change from baseline to Weeks 24, and 52 and 104 in TBLH BMD Z-score measured by DEXA, 8. Change from baseline to Week 24, week 52 and week 104 in total hip BMD Z-score measured by DEXA, 9 . Change from baseline to Weeks 12, 24 and 52 in circulating bone metabolism biomarkers, 10 . Number of new low-trauma long-bone or vertebral fractures by Week 52 and week 104, 11 . Proportion of Participants with new low-trauma long-bone or vertebral fracture by Week 52 and week 104, 12. Summary of observed serum concentration of satralizumab at specified trough timepoints up to Week 104, 13. Population and individual estimates of PK parameters (e.g., apparent clearance and apparent volume of distribution) and secondary PK parameters (e.g., area under the concentration-time curve), 14. Prevalence of ADAs at baseline and incidence of ADAs during the study","product":"Satralizumab, Satralizumab","ageRangeSecondary":["5", "6"],"ageGroup":"0-17 years","gender":"Male","trialRegionCode":3,"trialRegion":"Both","totalNumberEnrolled":"28","primaryEndPoint":"1. 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Summary of observed serum concentration of satralizumab at specified trough timepoints up to Week 104, 13. Population and individual estimates of PK parameters (e.g., apparent clearance and apparent volume of distribution) and secondary PK parameters (e.g., area under the concentration-time curve), 14. Prevalence of ADAs at baseline and incidence of ADAs during the study", "product": "Satralizumab, Satralizumab", "ageRangeSecondary": ["5", "6"], "ageGroup": "0-17 years", "gender": "Male", "trialRegion": 3, "totalNumberEnrolled": "28", "primaryEndPoint": "1. 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Muscular Dystrophy (SHIELD DMD)", "fullTitleTranslations": [{"id": 8534791, "uuid": "8c01dead-0e7a-4ae6-8c29-bb66c770d415", "attributeTranslation": "Wieloo\u015brodkowe, prowadzone metod\u0105 otwartej pr\u00f3by badanie fazy II maj\u0105ce na celu ocen\u0119 skuteczno\u015bci, bezpiecze\u0144stwa stosowania, farmakokinetyki i farmakodynamiki satralizumabu u dzieci i m\u0142odzie\u017cy z dystrofi\u0105 mi\u0119\u015bniow\u0105 Duchenne\u2019a (SHIELD DMD)", "language": 19, "languageDescription": "Polish"}, {"id": 8534792, "uuid": "8c01dead-0e7a-4ae6-8c29-bb66c770d415", "attributeTranslation": "Estudio Abierto, Multic\u00e9ntrico, de Fase 2 para evaluar la Eficacia, Seguridad, Farmacocin\u00e9tica y Farmacodin\u00e1mica de Satralizumab en Pacientes Pedi\u00e1tricos con Distrofia Muscular de Duchenne (SHIELD DMD)", "language": 7, "languageDescription": "Spanish"}], "publicTitle": "A study to check the effect of satralizumab in children and adolescents living with Duchenne muscular dystrophy, to check if it is safe and also how it affects the different parts of the body and how it is eliminated from the body (SHIELD DMD)", "publicTitleTranslations": [{"id": 8534807, "uuid": "03ea9571-5843-4213-ad6e-659190c3efa6", "attributeTranslation": "Badanie maj\u0105ce na celu sprawdzenie, jak dzia\u0142a satralizumab u dzieci i m\u0142odzie\u017cy z dystrofi\u0105 mi\u0119\u015bniow\u0105 Duchenne'a, czy jest bezpieczny, a tak\u017ce jaki ma wp\u0142yw na poszczeg\u00f3lne cz\u0119\u015bci cia\u0142a oraz w jaki spos\u00f3b jest usuwany z organizmu (SHIELD DMD)", "language": 19, "languageDescription": "Polish"}, {"id": 8534808, "uuid": "03ea9571-5843-4213-ad6e-659190c3efa6", "attributeTranslation": "Estudio para comprobar el efecto de satralizumab en ni\u00f1os y adolescentes con distrofia muscular de Duchenne, para comprobar si es seguro y tambi\u00e9n c\u00f3mo afecta a las diferentes partes del cuerpo y c\u00f3mo se elimina del cuerpo (SHIELD DMD)", "language": 7, "languageDescription": "Spanish"}], "shortTitle": "BN45398", "secondaryIdentifyingNumbers": {"additionalRegistries": []}}, "trialInformation": {"trialCategory": {"isLowIntervention": false, "trialPhase": "4", "trialCategory": "2", "justificationForTrialCategory": "Deferral is selected because this is a confirmatory/registrational trial and the development program for the molecule is ongoing", "trialCategoryId": 96700}, "medicalCondition": {"partIMedicalConditions": [{"id": 109983, "medicalCondition": "Duchenne Muscular Dystrophy (DMD)", "medicalConditionTranslations": [{"id": 8534748, "uuid": "25c8640b-b762-48a1-8c0d-75dd3dcfc1f8", "attributeTranslation": "Distrofia muscular de Duchenne (DMD)", "language": 7, "languageDescription": "Spanish"}, {"id": 8534747, "uuid": "25c8640b-b762-48a1-8c0d-75dd3dcfc1f8", "attributeTranslation": "Dystrofia mi\u0119\u015bniowa Duchenne'a (DMD)", "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": "13", "otherDescription": "immunogenicity", "trialScopeId": 319821}, {"code": "7", "trialScopeId": 319823}, {"code": "5", "trialScopeId": 319822}, {"code": "6", "trialScopeId": 319820}, {"code": "4", "trialScopeId": 319819}], "mainObjective": "To evaluate the efficacy of satralizumab in bone mineral density (BMD) as assessed by dual-energy X-ray absorptiometry (DEXA) in fracture-na\u00efve participants", "mainObjectiveTranslations": [{"id": 8534805, "uuid": "1afb6720-ce27-4848-bdb7-51bcaf41ade4", "attributeTranslation": "Ocena skuteczno\u015bci satralizumabu w odniesieniu do g\u0119sto\u015bci mineralnej ko\u015bci (BMD) na podstawie oceny z u\u017cyciem dwuwi\u0105zkowej absorpcjometrii rentgenowskiej (DEXA) u pacjent\u00f3w bez z\u0142ama\u0144 w wywiadzie", "language": 19, "languageDescription": "Polish"}, {"id": 8534806, "uuid": "1afb6720-ce27-4848-bdb7-51bcaf41ade4", "attributeTranslation": "Evaluar la eficacia de satralizumab en cuanto a la densidad mineral \u00f3sea (DMO), determinada mediante absorciometr\u00eda con rayos X de doble energ\u00eda (DEXA) en participantes sin fractura previa", "language": 7, "languageDescription": "Spanish"}], "secondaryObjectives": [{"id": 368467, "number": 1, "secondaryObjective": "To evaluate the safety of satralizumab in DMD", "secondaryObjectiveTranslations": [{"id": 8534809, "uuid": "1927209e-410b-4db8-9605-e3c36e7442e4", "attributeTranslation": "Evaluar la seguridad de satralizumab en la DMD", "language": 7, "languageDescription": "Spanish"}, {"id": 8534810, "uuid": "1927209e-410b-4db8-9605-e3c36e7442e4", "attributeTranslation": "Ocena bezpiecze\u0144stwa stosowania satralizumabu w leczeniu DMD", "language": 19, "languageDescription": "Polish"}]}, {"id": 368468, "number": 2, "secondaryObjective": "To evaluate the efficacy of satralizumab in BMD as assessed by DEXA in all participants", "secondaryObjectiveTranslations": [{"id": 8534811, "uuid": "5e3db21a-23e1-4158-8cb7-b95b2943365d", "attributeTranslation": "Evaluar la eficacia de satralizumab en cuanto a la DMD, determinada mediante DEXA en todos los participantes", "language": 7, "languageDescription": "Spanish"}, {"id": 8534812, "uuid": "5e3db21a-23e1-4158-8cb7-b95b2943365d", "attributeTranslation": "Ocena skuteczno\u015bci satralizumabu w odniesieniu do g\u0119sto\u015bci mineralnej ko\u015bci (BMD) na podstawie badania DEXA u wszystkich uczestnik\u00f3w", "language": 19, "languageDescription": "Polish"}]}, {"id": 368469, "number": 3, "secondaryObjective": "To evaluate the efficacy of satralizumab in bone metabolism biomarkers in all participants", "secondaryObjectiveTranslations": [{"id": 8534813, "uuid": "a2f84b58-f764-4d94-8671-5fc63116d1e8", "attributeTranslation": "Ocena skuteczno\u015bci satralizumabu w odniesieniu do biomarker\u00f3w metabolizmu kostnego u wszystkich uczestnik\u00f3w", "language": 19, "languageDescription": "Polish"}, {"id": 8534814, "uuid": "a2f84b58-f764-4d94-8671-5fc63116d1e8", "attributeTranslation": "Evaluar la eficacia de satralizumab en cuanto a biomarcadores del metabolismo \u00f3seo en todos los participantes", "language": 7, "languageDescription": "Spanish"}]}, {"id": 368470, "number": 4, "secondaryObjective": "To evaluate the efficacy of satralizumab in the incidence of fractures", "secondaryObjectiveTranslations": [{"id": 8534816, "uuid": "fc151b42-d256-4478-9e74-fe689ea449e5", "attributeTranslation": "Evaluar la eficacia de satralizumab en cuanto a la incidencia de fracturas", "language": 7, "languageDescription": "Spanish"}, {"id": 8534815, "uuid": "fc151b42-d256-4478-9e74-fe689ea449e5", "attributeTranslation": "Ocena skuteczno\u015bci satralizumabu w odniesieniu do cz\u0119sto\u015bci wyst\u0119powania z\u0142ama\u0144", "language": 19, "languageDescription": "Polish"}]}, {"id": 368471, "number": 5, "secondaryObjective": "To characterize the pharmacokinetics of satralizumab", "secondaryObjectiveTranslations": [{"id": 8534818, "uuid": "ebbbb8d5-5fec-455b-bf65-2116b27ebbe9", "attributeTranslation": "Caracterizar la farmacocin\u00e9tica de satralizumab", "language": 7, "languageDescription": "Spanish"}, {"id": 8534817, "uuid": "ebbbb8d5-5fec-455b-bf65-2116b27ebbe9", "attributeTranslation": "Charakterystyka farmakokinetyki satralizumabu", "language": 19, "languageDescription": "Polish"}]}, {"id": 368472, "number": 6, "secondaryObjective": "To evaluate the immunogenicity of satralizumab", "secondaryObjectiveTranslations": [{"id": 8534820, "uuid": "97825809-7acd-415e-a4ae-c9aef4027a69", "attributeTranslation": "Evaluar la inmunogenicidad de satralizumab", "language": 7, "languageDescription": "Spanish"}, {"id": 8534819, "uuid": "97825809-7acd-415e-a4ae-c9aef4027a69", "attributeTranslation": "Ocena immunogenno\u015bci satralizumabu", "language": 19, "languageDescription": "Polish"}]}, {"id": 368473, "number": 7, "secondaryObjective": "To evaluate the efficacy of satralizumab in bone metabolism biomarkers in fracture naive participants", "secondaryObjectiveTranslations": [{"id": 8534822, "uuid": "227696b8-1a1d-44b0-a117-261534bc574a", "attributeTranslation": "Evaluar la eficacia de satralizumab en cuanto a biomarcadores del metabolismo \u00f3seo en participantes sin fracturas previas", "language": 7, "languageDescription": "Spanish"}, {"id": 8534821, "uuid": "227696b8-1a1d-44b0-a117-261534bc574a", "attributeTranslation": "Ocena skuteczno\u015bci satralizumabu w odniesieniu do biomarker\u00f3w metabolizmu kostnego u uczestnik\u00f3w bez wcze\u015bniejszych z\u0142ama\u0144", "language": 19, "languageDescription": "Polish"}]}]}, "eligibilityCriteria": {"principalInclusionCriteria": [{"id": 707186, "number": 1, "principalInclusionCriteria": "Age \u2265 8 and < 18 years at the time of signing Informed Consent Form", "principalInclusionCriteriaTranslations": [{"id": 8534794, "uuid": "fdbea1d5-43d2-4cc0-803c-a6e973d2f286", "attributeTranslation": "Wiek \u22658 i <18 lat w momencie podpisania formularza \u015bwiadomej zgody", "language": 19, "languageDescription": "Polish"}, {"id": 8534793, "uuid": "fdbea1d5-43d2-4cc0-803c-a6e973d2f286", "attributeTranslation": "Edad \u22658 y < 18 a\u00f1os en el momento de firmar el consentimiento informado", "language": 7, "languageDescription": "Spanish"}]}, {"id": 707187, "number": 2, "principalInclusionCriteria": "Male at birth", "principalInclusionCriteriaTranslations": [{"id": 8534796, "uuid": "a7d144a1-1082-4e32-af3e-7fbf050bec1b", "attributeTranslation": "Var\u00f3n al nacer", "language": 7, "languageDescription": "Spanish"}, {"id": 8534795, "uuid": "a7d144a1-1082-4e32-af3e-7fbf050bec1b", "attributeTranslation": "P\u0142e\u0107 m\u0119ska w czasie narodzin", "language": 19, "languageDescription": "Polish"}]}, {"id": 707188, "number": 3, "principalInclusionCriteria": "Group 2 participants are required to meet the following criteria: Be fracture na\u00efve, defined as: No history of prior low-trauma fractures before the baseline visit nor any radiological findings indicative of prevalent vertebral fracture (VF) at the screening visit \u2013 Be ambulatory, defined as able to walk independently without assistive devices. \u2013 Age \u2265 8 to < 12 years old at the time of screening", "principalInclusionCriteriaTranslations": [{"id": 8534798, "uuid": "289ab5ee-96ac-4525-8168-ff35f5582127", "attributeTranslation": "Uczestnicy z Grupy 2 musz\u0105 spe\u0142ni\u0107 nast\u0119puj\u0105ce kryteria:\n- Bez wcze\u015bniejszych z\u0142ama\u0144, co definiuje si\u0119 jako brak wyst\u0119powania z\u0142ama\u0144 niskoenergetycznych w wywiadzie przed wizyt\u0105 wyj\u015bciow\u0105 oraz brak wynik\u00f3w bada\u0144 radiologicznych wskazuj\u0105cych na przebyte z\u0142amania kr\u0119g\u00f3w (VF) podczas wizyty przesiewowej\n\u2013 Uczestnik chodz\u0105cy, co jest zdefiniowane jako zdolno\u015b\u0107 do samodzielnego poruszania si\u0119 bez korzystania z przyrz\u0105d\u00f3w wspomagaj\u0105cych\n\u2013 Wiek \u22658 do <12 lat w czasie oceny przesiewowej", "language": 19, "languageDescription": "Polish"}, {"id": 8534797, "uuid": "289ab5ee-96ac-4525-8168-ff35f5582127", "attributeTranslation": "Los participantes del grupo 2 deber\u00e1n cumplir los criterios siguientes:\n- No tener fracturas previas, definido como: ausencia de antecedentes de fracturas previas por traumatismos de baja energ\u00eda antes de la visita basal y de signos radiol\u00f3gicos indicativos de fractura vertebral (FV) prevalente en la visita de selecci\u00f3n\n- Con capacidad deambulatoria, definida como la capacidad de caminar de forma independiente sin dispositivos de ayuda.\n- Edad entre \u2265 8 y < 12 a\u00f1os en el momento de la selecci\u00f3n", "language": 7, "languageDescription": "Spanish"}]}, {"id": 707189, "number": 4, "principalInclusionCriteria": "Group 1 participants are required to meet the following criteria: \u2013 SDI \u2264 3 - Age \u2265 8 to < 18 years old at the time of screening \u2013 If ambulatory (defined as able to walk independently without assistive devices) the participant must have a prior history of fractures Prior history of low-trauma fracture defined as: evidence of at least one prevalent vertebral compression fracture of Genant Grade 1 or 2 (or radiographic signs of VF) or history of at least one low-trauma long bone fracture (upper or lower extremity) but no more than two events incurring in low-trauma fractures (at any anatomical site) OR \u2013 If non-ambulatory, characterized as being non-ambulatory for a minimum of 6 months with onset of non-ambulatory status defined as participant- or caregiver-reported age of continuous wheelchair use, approximated to the nearest month, and an NSAA walk score of \"0\" and inability to perform the 10MWR at the baseline visit, the participant can be with or without prevalent fractures at baseline", "principalInclusionCriteriaTranslations": [{"id": 8534800, "uuid": "b63b8625-26b9-4d1b-8249-5959ce23deb5", "attributeTranslation": "Uczestnicy z Grupy 1 musz\u0105 spe\u0142nia\u0107 nast\u0119puj\u0105ce kryteria:\n- Wynik SDI \u22643\n- Wiek od \u22658 do <18 lat podczas bada\u0144 przesiewowych\n- W przypadku uczestnik\u00f3w chodz\u0105cych (definiowanych jako posiadaj\u0105cych zdolno\u015b\u0107 do samodzielnego poruszania si\u0119 bez korzystania z przyrz\u0105d\u00f3w wspomagaj\u0105cych) musz\u0105 wyst\u0119powa\u0107 wcze\u015bniejsze z\u0142amania w wywiadzie\nWcze\u015bniejsze z\u0142amania niskoenergetyczne w wywiadzie definiuje si\u0119 w nast\u0119puj\u0105cy spos\u00f3b: cechy co najmniej jednego przebytego z\u0142amania kompresyjnego kr\u0119g\u00f3w (VF) stopnia 1 lub 2 w klasyfikacji Genanta (albo cechy VF w badaniu radiograficznym) lub co najmniej jedno niskoenergetyczne z\u0142amanie ko\u015bci d\u0142ugich (ko\u0144czyny g\u00f3rnej lub dolnej) w wywiadzie, ale nie wi\u0119cej ni\u017c dwa zdarzenia zwi\u0105zane ze z\u0142amaniami niskoenergetycznymi (w dowolnej lokalizacji anatomicznej)\nLUB\nW przypadku uczestnik\u00f3w niechodz\u0105cych, charakteryzuj\u0105cych si\u0119 brakiem zdolno\u015bci chodzenia przez co najmniej 6 miesi\u0119cy, przy czym pocz\u0105tek stanu niezdolno\u015bci do chodzenia zdefiniowano jako zg\u0142aszany przez uczestnika lub opiekuna wiek, w kt\u00f3rym pacjent rozpocz\u0105\u0142 sta\u0142e korzystanie z w\u00f3zka inwalidzkiego, w przybli\u017ceniu do najbli\u017cszego miesi\u0105ca, oraz wynik \u201e0\u201d w skali oceny zdolno\u015bci chodzenia North Star Ambulatory Assessment (NSAA), a tak\u017ce niezdolno\u015b\u0107 do wykonania testu chodzenia na dystansie 10 m (10MWR) podczas wizyty wyj\u015bciowej, u uczestnika mog\u0105, lecz nie musz\u0105 wyj\u015bciowo wyst\u0119powa\u0107 przebyte z\u0142amania", "language": 19, "languageDescription": "Polish"}, {"id": 8534799, "uuid": "b63b8625-26b9-4d1b-8249-5959ce23deb5", "attributeTranslation": "Los participantes del grupo 1 deber\u00e1n cumplir los criterios siguientes:\n- Puntuaci\u00f3n SDI < 3\n- Tener entre \u2265 8 y < 18 a\u00f1os en el momento de la selecci\u00f3n\n- Si tiene capacidad deambulatoria (definida como la capacidad de caminar de forma independiente sin dispositivos de ayuda) el participante debe tener antecedentes de fracturas.\nAntecedentes de fracturas por traumatismo de baja energ\u00eda, definidas como signos de al menos una fractura por compresi\u00f3n vertebral prevalente de Genant de grado 1 o superior (o signos radiol\u00f3gicos de fracturas vertebrales) o antecedentes de al menos una fractura de huesos largos (extremidad superior o inferior) por traumatismos de baja energ\u00eda, pero no m\u00e1s de dos episodios con fracturas por traumatismos de baja energ\u00eda (en cualquier localizaci\u00f3n anat\u00f3mica)\nO\n- Si no tiene capacidad deambulatoria, caracterizada como la ausencia de deambulaci\u00f3n durante un m\u00ednimo de 6 meses, defini\u00e9ndose el comienzo de la ausencia de capacidad deambulatoria como la edad indicada por el participante o cuidador de uso continuo de una silla de ruedas, redondeada al mes m\u00e1s pr\u00f3ximo, y una puntuaci\u00f3n de marcha de la NSAA de \"0\" e incapacidad para realizar la prueba 10MWR en la visita basal, el participante puede tener o no fracturas prevalentes en el momento basal.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 707190, "number": 5, "principalInclusionCriteria": "For ambulatory participants: Group 1 and 2: NSAA total score \u2265 16 as assessed at the screening visit", "principalInclusionCriteriaTranslations": [{"id": 8534801, "uuid": "38a9754b-6f4a-426b-9338-9a8b904238ac", "attributeTranslation": "Dotyczy uczestnik\u00f3w chodz\u0105cych: Grupa 1 i 2: wynik ca\u0142kowity w skali NSAA \u226516, zgodnie z ocen\u0105 podczas wizyty przesiewowej", "language": 19, "languageDescription": "Polish"}, {"id": 8534802, "uuid": "38a9754b-6f4a-426b-9338-9a8b904238ac", "attributeTranslation": "Para los participantes con capacidad deambulatoria: Grupo 1 y 2: puntuaci\u00f3n total de NSSA \u2265 16, evaluada en la visita de selecci\u00f3n", "language": 7, "languageDescription": "Spanish"}]}, {"id": 707191, "number": 6, "principalInclusionCriteria": "Daily oral corticosteroids for at least 12 months with a stable dose for at least 12 weeks prior to screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the study", "principalInclusionCriteriaTranslations": [{"id": 8534803, "uuid": "16262121-5632-4a84-9de3-c96dd7c1a706", "attributeTranslation": "Administraci\u00f3n de corticosteroides orales a diario durante al menos 12 meses, con una dosis estable durante al menos 12 semanas antes de la selecci\u00f3n, y previsi\u00f3n de que la dosis se mantenga constante (excepto en caso de modificaciones para adaptarse a las variaciones de peso) durante todo el estudio", "language": 7, "languageDescription": "Spanish"}, {"id": 8534804, "uuid": "16262121-5632-4a84-9de3-c96dd7c1a706", "attributeTranslation": "Codzienne stosowanie doustnych kortykosteroid\u00f3w przez co najmniej 12 miesi\u0119cy w stabilnej dawce przez co najmniej 12 tygodni przed ocen\u0105 przesiewow\u0105 oraz spodziewane utrzymanie sta\u0142ej dawki (z wyj\u0105tkiem modyfikacji zwi\u0105zanych ze zmianami masy cia\u0142a) przez ca\u0142y czas trwania badania", "language": 19, "languageDescription": "Polish"}]}], "principalExclusionCriteria": [{"id": 1213836, "number": 1, "principalExclusionCriteria": "Major surgery (e.g. spinal surgery) within 3 months prior to Baseline or planned surgery or procedure that would interfere with the conduct of the study for any time during this study", "principalExclusionCriteriaTranslations": [{"id": 8534779, "uuid": "be546aa5-8a8d-4204-9a70-fa4d4ad45a7c", "attributeTranslation": "Intervenci\u00f3n de cirug\u00eda mayor (p. ej., cirug\u00eda de columna vertebral) en los 3 meses previos a la visita basal o\nintervenci\u00f3n quir\u00fargica o procedimiento programado en cualquier momento del estudio que pudiera interferir en su realizaci\u00f3n", "language": 7, "languageDescription": "Spanish"}, {"id": 8534780, "uuid": "be546aa5-8a8d-4204-9a70-fa4d4ad45a7c", "attributeTranslation": "Powa\u017cny zabieg chirurgiczny (np. operacja kr\u0119gos\u0142upa) w okresie 3 miesi\u0119cy przed ocen\u0105 wyj\u015bciow\u0105 b\u0105d\u017a planowana\noperacja lub zabieg, kt\u00f3re mog\u0142yby zak\u0142\u00f3ci\u0107 przebieg badania w dowolnym momencie tego badania", "language": 19, "languageDescription": "Polish"}]}, {"id": 1213837, "number": 2, "principalExclusionCriteria": "Presence of any clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for the participant or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the subject\u2019s ability to comply with the protocol required testing or procedures or compromise the subject\u2019s wellbeing, safety, or clinical interpretability", "principalExclusionCriteriaTranslations": [{"id": 8534782, "uuid": "0f8eae60-ea06-4c45-a2d9-a689f2514133", "attributeTranslation": "Presencia de cualquier enfermedad cl\u00ednicamente significativa, como enfermedad card\u00edaca, pulmonar, hep\u00e1tica, renal, hematol\u00f3gica, inmunol\u00f3gica o conductual, infecci\u00f3n o neoplasia maligna, de una enfermedad concomitante o necesidad de tratamiento farmacol\u00f3gico cr\u00f3nico que, en opini\u00f3n del investigador, suponga riesgos innecesarios para el participante o de una afecci\u00f3n m\u00e9dica o circunstancia atenuante que, en opini\u00f3n del investigador, pueda afectar a la capacidad del sujeto para cumplir con las pruebas o procedimientos exigidos en el protocolo o a su bienestar, seguridad o interpretabilidad cl\u00ednica", "language": 7, "languageDescription": "Spanish"}, {"id": 8534781, "uuid": "0f8eae60-ea06-4c45-a2d9-a689f2514133", "attributeTranslation": "Wyst\u0119powanie jakiejkolwiek istotnej klinicznie choroby, w tym choroby serca, p\u0142uc, w\u0105troby, nerek, choroby hematologicznej, immunologicznej lub behawioralnej, b\u0105d\u017a zaka\u017cenia lub nowotworu z\u0142o\u015bliwego albo choroby wsp\u00f3\u0142istniej\u0105cej lub te\u017c konieczno\u015b\u0107 stosowania przewlek\u0142ego leczenia farmakologicznego, kt\u00f3re w opinii badacza stwarzaj\u0105 niepotrzebne ryzyko dla uczestnika, b\u0105d\u017a wyst\u0119powanie stanu medycznego lub szczeg\u00f3lnych okoliczno\u015bci, kt\u00f3re w opinii badacza mog\u0105 negatywnie wp\u0142yn\u0105\u0107 na zdolno\u015b\u0107 uczestnika do przestrzegania wymog\u00f3w dotycz\u0105cych bada\u0144 lub procedur wymienionych w protokole, b\u0105d\u017a pogorszy\u0107 samopoczucie uczestnika, zagra\u017ca\u0107 jego bezpiecze\u0144stwu lub zak\u0142\u00f3ca\u0107 mo\u017cliwo\u015b\u0107 interpretacji obrazu klinicznego", "language": 19, "languageDescription": "Polish"}]}, {"id": 1213838, "number": 3, "principalExclusionCriteria": "Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection", "principalExclusionCriteriaTranslations": [{"id": 8534784, "uuid": "73244a0f-6d81-4751-a650-2ae5264fdbe7", "attributeTranslation": "Wyniki bada\u0144 serologicznych wskazuj\u0105ce na wyst\u0119powanie aktualnego, przewlek\u0142ego lub czynnego zaka\u017cenia ludzkim wirusem niedoboru odporno\u015bci, wirusem zapalenia w\u0105troby typu C lub wirusem zapalenia w\u0105troby typu B", "language": 19, "languageDescription": "Polish"}, {"id": 8534783, "uuid": "73244a0f-6d81-4751-a650-2ae5264fdbe7", "attributeTranslation": "Presenta signos serol\u00f3gicos de infecci\u00f3n presente, cr\u00f3nica o activa por el virus de la inmunodeficiencia humana, o los virus de la hepatitis C o B", "language": 7, "languageDescription": "Spanish"}]}, {"id": 1213839, "number": 4, "principalExclusionCriteria": "Has a symptomatic infection (e.g. upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to baseline", "principalExclusionCriteriaTranslations": [{"id": 8534786, "uuid": "47a4bbc6-54a7-44e1-aebd-1971b8f6e243", "attributeTranslation": "Objawowe zaka\u017cenie (np. zaka\u017cenie g\u00f3rnych dr\u00f3g oddechowych, zapalenie p\u0142uc, odmiedniczkowe zapalenie nerek, zapalenie opon m\u00f3zgowo-rdzeniowych) w ci\u0105gu 4 tygodni przed ocen\u0105 wyj\u015bciow\u0105", "language": 19, "languageDescription": "Polish"}, {"id": 8534785, "uuid": "47a4bbc6-54a7-44e1-aebd-1971b8f6e243", "attributeTranslation": "Presencia de una infecci\u00f3n sintom\u00e1tica (p. ej., infecci\u00f3n respiratoria de las v\u00edas altas, neumon\u00eda, pielonefritis, meningitis) en las 4 semanas previas a la visita basal", "language": 7, "languageDescription": "Spanish"}]}, {"id": 1213840, "number": 5, "principalExclusionCriteria": "History or laboratory evidence of coagulation disorders", "principalExclusionCriteriaTranslations": [{"id": 8534788, "uuid": "2906007a-0f84-4e74-919e-e35a158dea8d", "attributeTranslation": "Antecedentes o datos anal\u00edticos de trastornos de la coagulaci\u00f3n", "language": 7, "languageDescription": "Spanish"}, {"id": 8534787, "uuid": "2906007a-0f84-4e74-919e-e35a158dea8d", "attributeTranslation": "Historia lub wyniki bada\u0144 laboratoryjnych wskazuj\u0105ce na zaburzenia krzepni\u0119cia", "language": 19, "languageDescription": "Polish"}]}, {"id": 1213841, "number": 6, "principalExclusionCriteria": "Body weight at screening < 20 or > 100 kg", "principalExclusionCriteriaTranslations": [{"id": 8534789, "uuid": "84ee9086-3766-4d6f-b479-fcd3cba91351", "attributeTranslation": "Masa cia\u0142a <20 lub >100 kg podczas bada\u0144 przesiewowych", "language": 19, "languageDescription": "Polish"}, {"id": 8534790, "uuid": "84ee9086-3766-4d6f-b479-fcd3cba91351", "attributeTranslation": "Peso corporal en la selecci\u00f3n < 20 o >100 kg", "language": 7, "languageDescription": "Spanish"}]}]}, "endPoint": {"primaryEndPoints": [{"id": 828209, "number": 1, "endPoint": "1. Change from baseline to Week 52 in LS BMD Z-score measured by DEXA", "isPrimary": true, "endPointTranslations": [{"id": 8534749, "uuid": "3b3e238e-49e7-4d4e-a7db-0b792e364082", "attributeTranslation": "1. Zmiana wska\u017anika Z (Z-score) dla g\u0119sto\u015bci mineralnej ko\u015bci odcinka l\u0119d\u017awiowego kr\u0119gos\u0142upa (LS BMD) od warto\u015bci wyj\u015bciowych do Tygodnia 52, mierzona na podstawie badania DEXA", "language": 19, "languageDescription": "Polish"}, {"id": 8534750, "uuid": "3b3e238e-49e7-4d4e-a7db-0b792e364082", "attributeTranslation": "1. Variaci\u00f3n entre el momento basal y la semana 52 de la puntuaci\u00f3n Z de la DMO en la columna lumbar (DMO-CL) medida mediante DEXA", "language": 7, "languageDescription": "Spanish"}]}], "secondaryEndPoints": [{"id": 828210, "number": 1, "endPoint": "1. Incidence of treatment-emergent adverse events", "isPrimary": false, "endPointTranslations": [{"id": 8534751, "uuid": "e15f0003-2327-493e-8555-f0cfd28b41c2", "attributeTranslation": "1. Incidencia de acontecimientos adversos surgidos durante el tratamiento", "language": 7, "languageDescription": "Spanish"}, {"id": 8534752, "uuid": "e15f0003-2327-493e-8555-f0cfd28b41c2", "attributeTranslation": "1. Cz\u0119sto\u015b\u0107 wyst\u0119powania zdarze\u0144 niepo\u017c\u0105danych zwi\u0105zanych z leczeniem", "language": 19, "languageDescription": "Polish"}]}, {"id": 828211, "number": 2, "endPoint": "2. Incidence of serious adverse events", "isPrimary": false, "endPointTranslations": [{"id": 8534753, "uuid": "7dbddd53-d98d-4888-ae97-2dacf54a0ec4", "attributeTranslation": "2. Cz\u0119sto\u015b\u0107 wyst\u0119powania ci\u0119\u017ckich zdarze\u0144 niepo\u017c\u0105danych", "language": 19, "languageDescription": "Polish"}, {"id": 8534754, "uuid": "7dbddd53-d98d-4888-ae97-2dacf54a0ec4", "attributeTranslation": "2. Incidencia de acontecimientos adversos graves", "language": 7, "languageDescription": "Spanish"}]}, {"id": 828212, "number": 3, "endPoint": "3. Incidence of adverse events of special interest", "isPrimary": false, "endPointTranslations": [{"id": 8534755, "uuid": "540e5c8b-3bc6-4da0-9539-ccca3efc6a3a", "attributeTranslation": "3. Incidencia de acontecimientos adversos de inter\u00e9s especial", "language": 7, "languageDescription": "Spanish"}, {"id": 8534756, "uuid": "540e5c8b-3bc6-4da0-9539-ccca3efc6a3a", "attributeTranslation": "3. Cz\u0119sto\u015b\u0107 wyst\u0119powania zdarze\u0144 niepo\u017c\u0105danych o szczeg\u00f3lnym znaczeniu", "language": 19, "languageDescription": "Polish"}]}, {"id": 828213, "number": 4, "endPoint": "4. Clinically significant changes in vital signs and physical examination findings", "isPrimary": false, "endPointTranslations": [{"id": 8534757, "uuid": "4ea1f210-397d-42d4-a006-8b10d27c9f03", "attributeTranslation": "4. Variaciones cl\u00ednicamente significativas de las constantes vitales y los hallazgos de la exploraci\u00f3n f\u00edsica", "language": 7, "languageDescription": "Spanish"}, {"id": 8534758, "uuid": "4ea1f210-397d-42d4-a006-8b10d27c9f03", "attributeTranslation": "4. Klinicznie istotne zmiany parametr\u00f3w \u017cyciowych i wynik\u00f3w badania fizykalnego", "language": 19, "languageDescription": "Polish"}]}, {"id": 828214, "number": 5, "endPoint": "5. Clinically significant changes in safety laboratory assessments, ECGs", "isPrimary": false, "endPointTranslations": [{"id": 8534759, "uuid": "f1f09e65-5448-43dc-ac8b-a3cc73849381", "attributeTranslation": "5. Variaciones cl\u00ednicamente significativas de las determinaciones anal\u00edticas de seguridad, y los ECG", "language": 7, "languageDescription": "Spanish"}, {"id": 8534760, "uuid": "f1f09e65-5448-43dc-ac8b-a3cc73849381", "attributeTranslation": "5. Klinicznie istotne zmiany w badaniach laboratoryjnych dotycz\u0105cych bezpiecze\u0144stwa i badaniach EKG", "language": 19, "languageDescription": "Polish"}]}, {"id": 828215, "number": 6, "endPoint": "6. Change from baseline to Weeks 24, 52 and 104 in LS BMD Z-score measured by DEXA", "isPrimary": false, "endPointTranslations": [{"id": 8534761, "uuid": "82b9bddc-5462-44cd-a5f8-d871f439d1af", "attributeTranslation": "6. Variaci\u00f3n entre el momento basal y las semanas 24, 52 y 104 de la puntuaci\u00f3n Z de la DMO-CL medida mediante DEXA", "language": 7, "languageDescription": "Spanish"}, {"id": 8534762, "uuid": "82b9bddc-5462-44cd-a5f8-d871f439d1af", "attributeTranslation": "6. Zmiana wska\u017anika Z (Z-score) dla g\u0119sto\u015bci mineralnej ko\u015bci odcinka l\u0119d\u017awiowego kr\u0119gos\u0142upa (LS BMD) od warto\u015bci wyj\u015bciowych do Tygodni 24, 52 i 104, mierzona na podstawie badania DEXA", "language": 19, "languageDescription": "Polish"}]}, {"id": 828216, "number": 7, "endPoint": "7. Change from baseline to Weeks 24, and 52 and 104 in TBLH BMD Z-score measured by DEXA", "isPrimary": false, "endPointTranslations": [{"id": 8534763, "uuid": "0d3c5e03-940e-40c7-ba94-d792d4575ad8", "attributeTranslation": "7. Zmiana wska\u017anika Z (Z-score) w zakresie g\u0119sto\u015bci mineralnej ko\u015bci ca\u0142ego cia\u0142a z wy\u0142\u0105czeniem g\u0142owy (TBLH BMD) od warto\u015bci wyj\u015bciowych do Tygodni 24, 52 i 104, mierzona na podstawie badania DEXA", "language": 19, "languageDescription": "Polish"}, {"id": 8534764, "uuid": "0d3c5e03-940e-40c7-ba94-d792d4575ad8", "attributeTranslation": "7. Variaci\u00f3n entre el momento basal y las semanas 24, 52 y 104 de la puntuaci\u00f3n Z de la DMO total del cuerpo menos la cabeza (TBLH) medida mediante DEXA", "language": 7, "languageDescription": "Spanish"}]}, {"id": 828217, "number": 8, "endPoint": "8. Change from baseline to Week 24, week 52 and week 104 in total hip BMD Z-score measured by DEXA", "isPrimary": false, "endPointTranslations": [{"id": 8534765, "uuid": "f6bb5698-5f44-40a3-954b-7ba0a820b147", "attributeTranslation": "8. Variaci\u00f3n entre el momento basal y las semanas 24, 52 y 104 de la puntuaci\u00f3n Z total de la DMO en la cadera medida mediante DEXA", "language": 7, "languageDescription": "Spanish"}, {"id": 8534766, "uuid": "f6bb5698-5f44-40a3-954b-7ba0a820b147", "attributeTranslation": "8. Zmiana wska\u017anika Z (Z-score) w zakresie g\u0119sto\u015bci mineralnej ko\u015bci dla ca\u0142ego biodra wzgledem warto\u015bci wyj\u015bciowych do Tygodni 24, 52 i 104, mierzona na podstawie badania DEXA", "language": 19, "languageDescription": "Polish"}]}, {"id": 828218, "number": 9, "endPoint": "9 . Change from baseline to Weeks 12, 24 and 52 in circulating bone metabolism biomarkers", "isPrimary": false, "endPointTranslations": [{"id": 8534768, "uuid": "56404221-9778-4c57-b9e8-87afbc596812", "attributeTranslation": "9. Zmiana kr\u0105\u017c\u0105cych biomarker\u00f3w metabolizmu kostnego od warto\u015bci wyj\u015bciowych do Tygodni 12, 24 i 52", "language": 19, "languageDescription": "Polish"}, {"id": 8534767, "uuid": "56404221-9778-4c57-b9e8-87afbc596812", "attributeTranslation": "9. Variaci\u00f3n entre el momento basal y las semanas 12, 24 y 52 de biomarcadores circulantes del metabolismo \u00f3seo", "language": 7, "languageDescription": "Spanish"}]}, {"id": 828219, "number": 10, "endPoint": "10 . Number of new low-trauma long-bone or vertebral fractures by Week 52 and week 104", "isPrimary": false, "endPointTranslations": [{"id": 8534770, "uuid": "a71c8ae2-ae46-40ae-9a81-31b402c86438", "attributeTranslation": "10. Liczba nowych niskoenergetycznych z\u0142ama\u0144 ko\u015bci d\u0142ugich lub z\u0142ama\u0144 kr\u0119g\u00f3w do Tygodnia 52 i Tygodnia 104", "language": 7, "languageDescription": "Spanish"}, {"id": 8534769, "uuid": "a71c8ae2-ae46-40ae-9a81-31b402c86438", "attributeTranslation": "10. N\u00famero de nuevas fracturas de fracturas vertebrales o de huesos largos por traumatismos de baja energ\u00eda en las semanas 52 y 104", "language": 19, "languageDescription": "Polish"}]}, {"id": 828220, "number": 11, "endPoint": "11 . 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Prevalencia de anticuerpos antiterap\u00e9uticos (AcAT) en el momento basal e incidencia de AcAT durante el estudio", "language": 7, "languageDescription": "Spanish"}]}]}, "trialDuration": {"estimatedGlobalEndDate": "2027-07-31", "estimatedEndDate": "2027-07-31", "estimatedRecruitmentStartDate": "2024-11-26"}, "sourceOfMonetarySupport": [{"id": 77222, "organisationName": "F. 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"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": "\u00c9tude en ouvert sur la s\u00e9curit\u00e9 d\u2019emploi, la tol\u00e9rance et l\u2019efficacit\u00e9 \u00e0 long terme du GIVINOSTAT chez tous les patients atteints de DMD qui ont d\u00e9j\u00e0 \u00e9t\u00e9 trait\u00e9s dans l\u2019une des \u00e9tudes 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-pati\u00ebnten 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 f\u00e1rmaco en investigaci\u00f3n 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": "\u00c9tude dans laquelle tous les patients prennent le m\u00eame m\u00e9dicament exp\u00e9rimental dans le but d\u2019\u00e9valuer la s\u00e9curit\u00e9 d\u2019emploi, la tol\u00e9rance et l\u2019efficacit\u00e9 \u00e0 long terme du GIVINOSTAT chez tous les patients atteints de dystrophie musculaire de Duchenne qui ont d\u00e9j\u00e0 \u00e9t\u00e9 trait\u00e9s dans l\u2019une des \u00e9tudes sur le GIVINOSTAT par le pass\u00e9", "language": 10, "languageDescription": "French"}, {"id": 7196024, "uuid": "4d05e17d-d690-482c-b75d-a27903592f0d", "attributeTranslation": "Onderzoek waarin alle pati\u00ebnten hetzelfde onderzoeksmiddel ontvangen met als doel het evalueren van de langetermijnveiligheid, -verdraagbaarheid en -werkzaamheid van GIVINOSTAT bij alle pati\u00ebnten 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 na\u00efve 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": "\u00c9valuer la s\u00e9curit\u00e9 d\u2019emploi et la tol\u00e9rance \u00e0 long terme du GIVINOSTAT chez des patients atteints de DMD apr\u00e8s un programme de protocoles de base et chez des patients atteints de DMD na\u00effs de GIVINOSTAT, c\u2019est-\u00e0-dire des patients s\u00e9lectionn\u00e9s dans l\u2019\u00e9tude DSC/14/2357/48 qui :\n- ont r\u00e9pondu \u00e0 la totalit\u00e9 des crit\u00e8res d\u2019inclusion et n\u2019ont r\u00e9pondu \u00e0 aucun des crit\u00e8res d\u2019exclusion, et\n- n\u2019ont jamais \u00e9t\u00e9 randomis\u00e9s, car l\u2019inclusion dans la cohorte hors cible \u00e9tait termin\u00e9e.", "language": 10, "languageDescription": "French"}, {"id": 7196021, "uuid": "292d7db0-8e45-49bd-939c-31d4b9a53d5b", "attributeTranslation": "Het beoordelen van de langetermijnveiligheid en -verdraagbaarheid van GIVINOSTAT bij pati\u00ebnten 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\u2014 cumplieron todos los criterios de inclusi\u00f3n y ninguno de los criterios de exclusi\u00f3n, y\n\u2014 nunca hab\u00edan sido aleatorizados porque se hab\u00eda completado la inclusi\u00f3n 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 administraci\u00f3n a largo plazo de GIVINOSTAT sobre la funci\u00f3n 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": "\u00c9valuer les effets de l\u2019administration \u00e0 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 administraci\u00f3n a largo plazo de GIVINOSTAT sobre la funci\u00f3n respiratoria.", "language": 7, "languageDescription": "Spanish"}, {"id": 7196030, "uuid": "8de26b6c-316c-428d-8d1e-bbb2e69d40d3", "attributeTranslation": "\u00c9valuer les effets de l\u2019administration \u00e0 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": "\u00c9valuer l\u2019impact sur les activit\u00e9s quotidiennes et la qualit\u00e9 de vie apr\u00e8s l\u2019administration \u00e0 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 despu\u00e9s de la administraci\u00f3n 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 \u22645% 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 deb\u00edan haber sido seleccionados en el estudio DSC/14/2357/48 y cumplir lo siguiente: todos los criterios de inclusi\u00f3n y ninguno de los criterios de exclusi\u00f3n, presentaban una fracci\u00f3n de grasa del m\u00fasculo vasto externo (vastus lateralis muscle fat fraction, VL MFF) inicial evaluada mediante MRS en el intervalo \u22645 % o >30 %, es decir, haberse incluido en el grupo \u00abno objetivo\u00bb, nunca haber sido aleatorizado porque la inscripci\u00f3n en el grupo no objetivo se hab\u00eda 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 \u2264 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 particip\u00e9 \u00e0 l\u2019une des \u00e9tudes pr\u00e9c\u00e9dentes portant sur le GIVINOSTAT dans la DMD et avoir effectu\u00e9 la visite de fin de l\u2019\u00e9tude, ou doivent avoir \u00e9t\u00e9 s\u00e9lectionn\u00e9s dans l\u2019\u00e9tude DSC/14/2357/48 et : avoir r\u00e9pondu \u00e0 la totalit\u00e9 des crit\u00e8res d\u2019inclusion et n\u2019avoir r\u00e9pondu \u00e0 aucun des crit\u00e8res d\u2019exclusion, avoir pr\u00e9sent\u00e9 une fraction graisseuse du muscle vaste lat\u00e9ral (FG MVL) de r\u00e9f\u00e9rence \u00e9valu\u00e9e par spectrom\u00e9trie par r\u00e9sonance magn\u00e9tique (SRM) dans la plage \u2264 5 % ou > 30 %, c\u2019est-\u00e0-dire avoir \u00e9t\u00e9 inclus dans la cohorte \u00ab hors cible \u00bb, ne jamais avoir \u00e9t\u00e9 randomis\u00e9s, car l\u2019inclusion dans la cohorte hors cible \u00e9tait termin\u00e9e", "language": 10, "languageDescription": "French"}]}, {"id": 609246, "number": 2, "principalInclusionCriteria": "Subjects aged \u22656 years", "principalInclusionCriteriaTranslations": [{"id": 7196010, "uuid": "aaf4cc7b-d817-4286-a7a7-66d84d49bea4", "attributeTranslation": "Proefpersonen met een leeftijd \u2265 6 jaar.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 7196012, "uuid": "aaf4cc7b-d817-4286-a7a7-66d84d49bea4", "attributeTranslation": "Participantes con una edad \u22656 a\u00f1os.", "language": 7, "languageDescription": "Spanish"}, {"id": 7196011, "uuid": "aaf4cc7b-d817-4286-a7a7-66d84d49bea4", "attributeTranslation": "Patients \u00e2g\u00e9s de \u2265 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 \u00e9clair\u00e9 par \u00e9crit, sign\u00e9 par le patient et/ou son parent/tuteur l\u00e9gal (conform\u00e9ment \u00e0 la r\u00e9glementation 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 ge\u00efnformeerde 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 ge\u00efmplanteerde stero\u00efde-bevattende anticonceptiemethode, of een pessarium of een barri\u00e8remethode 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 \u00eatre dispos\u00e9s \u00e0 utiliser une contraception ad\u00e9quate.\nLes m\u00e9thodes de contraception doivent \u00eatre utilis\u00e9es \u00e0 partir de la pr\u00e9c\u00e9dente \u00e9tude sur le GIVINOSTAT et jusqu\u2019\u00e0 3 mois apr\u00e8s la derni\u00e8re dose du m\u00e9dicament \u00e0 l\u2019\u00e9tude, et comprennent les suivantes :\n- Abstinence totale (absence de tout rapport sexuel), lorsqu\u2019elle correspond au mode de vie pr\u00e9f\u00e9r\u00e9 et habituel du patient. L\u2019abstinence p\u00e9riodique (p. ex., m\u00e9thode du calendrier, m\u00e9thode de la glaire cervicale, m\u00e9thode sympto-thermique, m\u00e9thode des temp\u00e9ratures) et le retrait ne sont pas des m\u00e9thodes de contraception acceptables.\n- Pr\u00e9servatif avec spermicide et utilisation par la partenaire f\u00e9minine d\u2019une m\u00e9thode de contraception acceptable, comme un contraceptif oral, transdermique, injectable ou implant\u00e9 \u00e0 base de st\u00e9ro\u00efdes, ou un diaphragme ou une m\u00e9thode barri\u00e8re associ\u00e9e \u00e0 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 m\u00e9todos anticonceptivos adecuados:\nLos m\u00e9todos anticonceptivos deben utilizarse desde el estudio de GIVINOSTAT anterior hasta 3 meses despu\u00e9s de la \u00faltima dosis del f\u00e1rmaco del estudio, y son los siguientes:\n\u2014 Abstinencia verdadera (ausencia de relaciones sexuales), si se ajusta al estilo de vida preferido y habitual del participante. No se consideran m\u00e9todos anticonceptivos aceptables la abstinencia sexual peri\u00f3dica (p. ej., m\u00e9todos de calendario, ovulaci\u00f3n, sintot\u00e9rmico o posovulaci\u00f3n) ni el coito interrumpido.\n\u2014 Preservativo con espermicida, y la pareja de sexo femenino debe usar un m\u00e9todo anticonceptivo aceptable, como un anticonceptivo oral, transd\u00e9rmico, inyectable o implantado con esteroides, o un diafragma o un m\u00e9todo anticonceptivo de barrera junto con gel espermicida, como un capuch\u00f3n 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 corticost\u00e9ro\u00efdes, qui pourrait avoir un effet sur la force ou la fonction musculaire dans les 3 mois pr\u00e9c\u00e9dant l\u2019inclusion dans cette \u00e9tude (p. ex., hormone de croissance) ; la vitamine D, le calcium et tout autre suppl\u00e9ment seront autoris\u00e9s", "language": 10, "languageDescription": "French"}, {"id": 7195965, "uuid": "045feed2-0630-44cf-ab3b-99f323be8f6d", "attributeTranslation": "Het gebruik van een farmacologische behandeling, anders dan corticostero\u00efden, 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 farmacol\u00f3gico, distinto de los corticoesteroides, que pueda haber tenido un efecto sobre la fuerza o la funci\u00f3n muscular en los 3 meses anteriores a la inscripci\u00f3n en este estudio (p. ej., hormona de crecimiento); se permitir\u00e1 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": "Pr\u00e9senter un QTcF de r\u00e9f\u00e9rence > 450 ms (moyenne de 3 lectures cons\u00e9cutives espac\u00e9es de 5 minutes) ou des ant\u00e9c\u00e9dents de facteurs de risque suppl\u00e9mentaires de torsades de pointes (p. ex., insuffisance cardiaque, hypokali\u00e9mie ou ant\u00e9c\u00e9dents 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 card\u00edaca, hipopotasiemia o antecedentes familiares del s\u00edndrome 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, hypokali\u00ebmie 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 psiqui\u00e1trica o situaciones sociales que hagan que el posible participante no pueda comprender ni cumplir con las pruebas de funci\u00f3n 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 proc\u00e9dures du protocole de l\u2019\u00e9tude et de s\u2019y conformer", "language": 10, "languageDescription": "French"}, {"id": 7195971, "uuid": "8e52c6f9-b565-4402-8214-c7ba53f8a7d5", "attributeTranslation": "Het hebben van een psychiatrische ziekte/sociale situatie die de potenti\u00eble 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 f\u00e1rmaco 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": "Pr\u00e9senter une hypersensibilit\u00e9 aux composants du m\u00e9dicament \u00e0 l\u2019\u00e9tude", "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 malabsorci\u00f3n de sorbitol o intolerancia hereditaria a la fructosa.", "language": 7, "languageDescription": "Spanish"}, {"id": 7195979, "uuid": "336ea243-860d-49af-be84-cc13ce676313", "attributeTranslation": "Pr\u00e9senter une intol\u00e9rance au sorbitol ou une malabsorption du sorbitol, ou une forme h\u00e9r\u00e9ditaire d\u2019intol\u00e9rance 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 f\u00e1rmaco en investigaci\u00f3n actual distinto de GIVINOSTAT.", "language": 7, "languageDescription": "Spanish"}, {"id": 7195982, "uuid": "a4158122-f312-4762-b770-40cb68259949", "attributeTranslation": "Utilisation de tout m\u00e9dicament en cours d\u2019investigation 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 be\u00efnvloeden.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 7195983, "uuid": "f20da03f-d3a6-46f9-b508-e7cf8afcc1c4", "attributeTranslation": "Presencia de otra enfermedad cl\u00ednicamente significativa, que, en opini\u00f3n del investigador, podr\u00eda 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 evaluaci\u00f3n de los resultados del estudio.", "language": 7, "languageDescription": "Spanish"}, {"id": 7195984, "uuid": "f20da03f-d3a6-46f9-b508-e7cf8afcc1c4", "attributeTranslation": "Pr\u00e9senter une autre maladie cliniquement significative qui, de l\u2019avis de l\u2019investigateur, pourrait nuire \u00e0 la s\u00e9curit\u00e9 du patient, rendant peu probable l\u2019ach\u00e8vement du traitement ou du suivi, ou pourrait nuire \u00e0 l\u2019\u00e9valuation des r\u00e9sultats de l\u2019\u00e9tude", "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 diagn\u00f3stico de otras enfermedades neurol\u00f3gicas no controladas o presencia de trastornos som\u00e1ticos relevantes no controlados que no est\u00e9n relacionados con la DMD.", "language": 7, "languageDescription": "Spanish"}, {"id": 7195987, "uuid": "a7c15cd5-7994-419d-99f0-bc095da5b943", "attributeTranslation": "Avoir re\u00e7u le diagnostic d\u2019autres maladies neurologiques non contr\u00f4l\u00e9es ou pr\u00e9senter des troubles somatiques non contr\u00f4l\u00e9s importants qui ne sont pas li\u00e9s \u00e0 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": "Pr\u00e9senter une num\u00e9ration plaquettaire, une num\u00e9ration des globules blancs et un taux d\u2019h\u00e9moglobine < limite inf\u00e9rieure de la normale (LIN) \u00e0 la s\u00e9lection (en cas de r\u00e9sultats anormaux des analyses biologiques de s\u00e9lection [< LIN], le dosage des plaquettes, des globules blancs et de l\u2019h\u00e9moglobine sera r\u00e9p\u00e9t\u00e9 une fois ; si le r\u00e9sultat des nouveaux dosages est toujours < LIN, cela constituera un crit\u00e8re d\u2019exclusion)", "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 selecci\u00f3n < l\u00edmite inferior de la normalidad (LIN) (para los resultados an\u00f3malos en los an\u00e1lisis cl\u00ednicos de la selecci\u00f3n [", "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 triglic\u00e9ridos >300 mg/dl (3,42 mmol/l) en ayunas en la visita de selecci\u00f3n* (para los resultados an\u00f3malos en los an\u00e1lisis cl\u00ednicos de selecci\u00f3n (>300 mg/dl); el an\u00e1lisis de los triglic\u00e9ridos se repetir\u00e1 una vez; si el resultado de la repetici\u00f3n sigue siendo >300 mg/dl, ser\u00e1 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 \u00e9\u00e9n 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": "Pr\u00e9senter un taux de triglyc\u00e9rides > 300 mg/dl (3,42 mmol/l) \u00e0 jeun lors de la visite de s\u00e9lection* (en cas de r\u00e9sultats anormaux des analyses biologiques de s\u00e9lection [> 300 mg/dl], le dosage des triglyc\u00e9rides sera r\u00e9p\u00e9t\u00e9 une fois ; si le r\u00e9sultat du nouveau dosage est toujours > 300 mg/dl, cela constituera un crit\u00e8re d\u2019exclusion)", "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": "Pr\u00e9senter une fonction r\u00e9nale inad\u00e9quate, d\u00e9finie par un taux s\u00e9rique de cystatine C > 2 x la limite sup\u00e9rieure de la normale (LSN) lors de la visite de s\u00e9lection. Si la valeur est > 2 x LSN, le dosage s\u00e9rique de la cystatine C sera r\u00e9p\u00e9t\u00e9 une fois ; si le r\u00e9sultat du nouveau dosage est toujours > 2 x LSN, cela constituera un crit\u00e8re d\u2019exclusion", "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 funci\u00f3n renal insuficiente, definida mediante cistatina C en suero >2 veces el l\u00edmite superior de la normalidad (LSN) en la visita de selecci\u00f3n. Si el valor es >2 veces el LSN, se repetir\u00e1 el an\u00e1lisis de la cistatina C en suero una vez; si el resultado de la repetici\u00f3n sigue siendo >2 veces el LSN, se deber\u00e1 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": "Pr\u00e9senter une insuffisance cardiaque (de classe III ou IV sur l\u2019\u00e9chelle 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 card\u00edaca (clase III o IV seg\u00fan la Asociaci\u00f3n Cardiol\u00f3gica 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 hep\u00e1tico 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 patr\u00f3n coincidente con la enfermedad de Gilbert.", "language": 7, "languageDescription": "Spanish"}, {"id": 7196002, "uuid": "ca847bec-46a0-43fb-80d4-96621179e25d", "attributeTranslation": "Pr\u00e9senter actuellement une maladie ou une insuffisance h\u00e9patique, y compris, mais sans s\u2019y limiter, une bilirubine totale \u00e9lev\u00e9e (c\u2019est-\u00e0-dire > 1,5 x LSN), \u00e0 moins qu\u2019elle ne soit secondaire \u00e0 la maladie de Gilbert ou \u00e0 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 s\u00e9v\u00e9rit\u00e9 des EI et des EIG li\u00e9s ou non li\u00e9s 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 d\u00e9placer :\n\u2022 Variation par rapport \u00e0 la r\u00e9f\u00e9rence de la fonction physique, mesur\u00e9e par l\u2019\u00e9preuve 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\u2019\u00e0 la fin de l\u2019\u00e9tude)", "language": 10, "languageDescription": "French"}, {"id": 7195938, "uuid": "cd23f700-c981-46a9-a54c-d2efbef2eff0", "attributeTranslation": "Para pacientes deambulantes:\n\u2022 Cambio con respecto al inicio en la funci\u00f3n f\u00edsica medida mediante 6MWT, NSAA, pruebas de funci\u00f3n temporal (p. ej. tiempo para levantarse del suelo, tiempo para subir 4 escalones, tiempo para caminar 10 metros) (semana 48 y luego cada a\u00f1o hasta el final del estudio).", "language": 7, "languageDescription": "Spanish"}, {"id": 7195939, "uuid": "cd23f700-c981-46a9-a54c-d2efbef2eff0", "attributeTranslation": "Voor poliklinische pati\u00ebnten:\n\u2022 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 pati\u00ebnten:\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 d\u00e9placer :\nVariation par rapport \u00e0 la r\u00e9f\u00e9rence de la force musculaire (p. ex., extension du genou et flexion du coude), mesur\u00e9e par myom\u00e9trie (appareil portatif ; semaine 48, puis annuellement jusqu\u2019\u00e0 la fin de l\u2019\u00e9tude)", "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., extensi\u00f3n de la rodilla y flexi\u00f3n del codo) medida mediante miometr\u00eda manual (handheld myometry, HHM) (semana 48 y luego cada a\u00f1o 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 d\u00e9placer :\n\u2022 Variation par rapport \u00e0 la r\u00e9f\u00e9rence de la fonction physique selon le score Egen Klassifikation (EK) (semaine 48, puis annuellement jusqu\u2019\u00e0 la fin de l\u2019\u00e9tude)", "language": 10, "languageDescription": "French"}, {"id": 7195946, "uuid": "231965e9-bf33-4dc0-86ef-0faec4905918", "attributeTranslation": "Voor niet-poliklinische pati\u00ebnten:\n\u2022 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\u2022 Cambio con respecto al inicio en la funci\u00f3n f\u00edsica en la puntuaci\u00f3n de Egen Klassifikation (EK) (semana 48 y luego cada a\u00f1o 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\u2022 Cambios en los informes de los pacientes o progenitores o cuidadores en las actividades cotidianas medidas por el \u00cdndice de Barthel (semana 48 y luego cada a\u00f1o hasta el final del estudio).", "language": 7, "languageDescription": "Spanish"}, {"id": 7195947, "uuid": "0fb80a3e-c3ed-4a7c-8860-30efc5175049", "attributeTranslation": "Voor niet-poliklinische pati\u00ebnten:\n\u2022 Verandering in door de pati\u00ebnt 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 d\u00e9placer :\n\u2022 Variation dans les rapports des patients et/ou des parents/aidants sur les activit\u00e9s de la vie quotidienne, mesur\u00e9e par l\u2019indice de Barthel (semaine 48, puis annuellement jusqu\u2019\u00e0 la fin de l\u2019\u00e9tude)", "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\u2022 Cambios en la fuerza muscular de las extremidades superiores (semana 48 y luego cada a\u00f1o hasta el final del estudio) evaluada mediante miometr\u00eda manual (HHM).", "language": 7, "languageDescription": "Spanish"}, {"id": 7195951, "uuid": "43887e7c-d9c4-4611-91df-9b2a7c54b2eb", "attributeTranslation": "Voor niet-poliklinische pati\u00ebnten:\n\u2022 Verandering in de spierkracht van de bovenste ledematen (week 48 en daarna jaarlijks tot het einde van het onderzoek) ge\u00ebvalueerd met HHM (handheld myometrie).", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 7195950, "uuid": "43887e7c-d9c4-4611-91df-9b2a7c54b2eb", "attributeTranslation": "Pour les patients incapables de se d\u00e9placer :\n\u2022 Variation de la force musculaire des membres sup\u00e9rieurs (semaine 48, puis annuellement jusqu\u2019\u00e0 la fin de l\u2019\u00e9tude) \u00e9valu\u00e9e par myom\u00e9trie (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 pati\u00ebnten:\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 \u00e0 la r\u00e9f\u00e9rence de la fonction physique, mesur\u00e9e par la performance des membres sup\u00e9rieurs (Performance of Upper Limb, PUL) et la MFM (semaine 48, puis annuellement jusqu\u2019\u00e0 la fin de l\u2019\u00e9tude)", "language": 10, "languageDescription": "French"}, {"id": 7195954, "uuid": "e88e01da-29b6-4a85-a8fd-e13b94bd2901", "attributeTranslation": "Para todos los pacientes:\nCambio con respecto al inicio en la funci\u00f3n f\u00edsica medida por el rendimiento de la extremidad superior (RES) y MFM (semana 48 y luego cada a\u00f1o 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 pati\u00ebnten:\nVerandering ten opzichte van de baseline in respiratoire functie (week 48 en daarna jaarlijks tot het einde van het onderzoek) (bijv. FVC, FEV1, PEF).", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 7195956, "uuid": "ce82936c-1add-4215-a943-2e65c8184147", "attributeTranslation": "Para todos los pacientes:\nCambio con respecto al inicio en la funci\u00f3n respiratoria (semana 48 y luego cada a\u00f1o hasta el final del estudio) (p. ej., CVF, VEM1, FEM).", "language": 7, "languageDescription": "Spanish"}, {"id": 7195957, "uuid": "ce82936c-1add-4215-a943-2e65c8184147", "attributeTranslation": "Pour tous les patients :\nVariation par rapport \u00e0 la r\u00e9f\u00e9rence de la fonction respiratoire (semaine 48, puis annuellement jusqu\u2019\u00e0 la fin de l\u2019\u00e9tude) (p. ex. CVF, VEMS, DEP)", "language": 10, "languageDescription": "French"}]}, {"id": 711770, "number": 8, "endPoint": "For all patients: Change in patient and/or parent/caregiver reports of quality of life as measured by PedsQL for paediatric patients and by SF-36 for adults patients (week 48 and then yearly till the end of the study)", "isPrimary": false, "endPointTranslations": [{"id": 7195961, "uuid": "bfd2db52-3029-473e-b943-2126bfd4cb60", "attributeTranslation": "Pour tous les patients :\nVariation dans les rapports des patients et/ou des parents/aidants sur la qualit\u00e9 de vie mesur\u00e9e par le PedsQL pour les patients p\u00e9diatriques et par le SF-36 pour les patients adultes (semaine 48, puis annuellement jusqu\u2019\u00e0 la fin de l\u2019\u00e9tude)", "language": 10, "languageDescription": "French"}, {"id": 7195959, "uuid": "bfd2db52-3029-473e-b943-2126bfd4cb60", "attributeTranslation": "Voor alle pati\u00ebnten:\nVerandering in door de pati\u00ebnt en/of ouder/verzorger gedane meldingen betreffende de kwaliteit van leven zoals gemeten aan de hand van PedsQL voor pediatrische pati\u00ebnten en aan de hand van SF-36 voor volwassen pati\u00ebnten (week 48 en daarna jaarlijks tot het einde van het onderzoek).", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 7195960, "uuid": "bfd2db52-3029-473e-b943-2126bfd4cb60", "attributeTranslation": "Para todos los pacientes:\nCambio en los informes de calidad de vida del paciente o del progenitor o cuidador, medido mediante PedsQL para pacientes pedi\u00e1tricos y mediante SF-36 para pacientes adultos (semana 48 y luego cada a\u00f1o hasta el final del estudio).", "language": 7, "languageDescription": "Spanish"}]}, {"id": 711771, "number": 9, "endPoint": "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)", "isPrimary": false, "endPointTranslations": [{"id": 7195963, "uuid": "c9985344-4bbf-4aa2-8869-4c7aa58808db", "attributeTranslation": "Para todos los pacientes:\nEdad en los hitos m\u00e1s importantes de la enfermedad (p. ej., edad en el momento de la p\u00e9rdida de la deambulaci\u00f3n, edad en la que se necesita asistencia respiratoria durante el d\u00eda, edad en la cirug\u00eda de escoliosis, edad en el momento de la muerte).", "language": 7, "languageDescription": "Spanish"}, {"id": 7195962, "uuid": "c9985344-4bbf-4aa2-8869-4c7aa58808db", "attributeTranslation": "Pour tous les patients :\n\u00c2ge aux principales \u00e9tapes de la maladie (p. ex., \u00e2ge au moment de la perte de la capacit\u00e9 ambulatoire, \u00e2ge auquel une assistance respiratoire est devenue n\u00e9cessaire pendant la journ\u00e9e, \u00e2ge au moment de la chirurgie correctrice de scoliose, \u00e2ge au d\u00e9c\u00e8s)", "language": 10, "languageDescription": "French"}, {"id": 7195964, "uuid": "c9985344-4bbf-4aa2-8869-4c7aa58808db", "attributeTranslation": "Voor alle pati\u00ebnten:\nLeeftijd op moment van belangrijke mijlpalen van de ziekte (bijv. leeftijd bij verlies van mobiliteit, leeftijd wanneer ademhalingsondersteuning overdag nodig is, leeftijd bij scoliose-operatie, leeftijd bij overlijden).", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}]}, "trialDuration": {"estimatedGlobalEndDate": "2027-12-03", "estimatedEndDate": "2027-12-03", "estimatedRecruitmentStartDate": "2017-07-03"}, "sourceOfMonetarySupport": [{"id": 66918, "organisationName": "ITALFARMACO S.p.A."}], "populationOfTrialSubjects": {"ageRanges": [{"id": 253029, "ageRangeCategoryCode": "2", "ageRangeCategory": "2"}, {"id": 253028, "ageRangeCategoryCode": "3", "ageRangeCategory": "3"}], "ageRangeSecondaryIds": [{"id": 253030, "ageRangeCategoryCode": "2", "ctAgeRangeCode": "6", "ageRangeCategory": "2", "ctAgeRange": "6"}, {"id": 253031, "ageRangeCategoryCode": "2", "ctAgeRangeCode": "5", "ageRangeCategory": "2", "ctAgeRange": "5"}], "clinicalTrialGroups": [{"code": "2", "name": "Patients"}], "isFemaleSubjects": false, "isMaleSubjects": true, "isVulnerablePopulationSelected": true}, "individualParticipantData": {"planToShareIPD": "3"}}, "protocolInformation": {"studyDesign": {"periodDetails": [{"id": 135410, "businessKey": "1", "title": "Treatment", "description": "GIVINOSTAT oral suspension (10 mg/mL) has to be administered orally as 2 oral doses daily while the subject is in a fed state. As weight affects GIVINOSTAT exposures, the dosage will be modified based on subject weight according to the rules detailed in the study protocol.", "blindingMethodCode": "3", "blindedRoles": [], "armDetails": [], "allocationMethod": "3"}]}}, "scientificAdviceAndPip": {"scientificAdvices": [], "paediatricInvestigationPlan": [{"id": 7895, "paediatricInvestigationNumber": "EMEA-000551-PIP04-21"}]}, "associatedClinicalTrials": [], "references": [], "pubmedCode": [], "pubmedUrl": []}, "assessmentOutcome": "acceptable", "therapeuticAreas": [{"code": "5", "name": "Diseases [C] - Musculoskeletal Diseases [C05]"}], "medicalConditions": [{"id": 95355, "medicalCondition": "Duchenne Muscular Dystrophy (DMD)", "isConditionRareDisease": true}], "sponsors": [{"id": 101950, "primary": true, "publicContacts": [{"id": 304577, "type": "Public", "functionalName": "Patient Advocacy Representative og Italfarmaco S.p.A", "functionalEmailAddress": "patientadvocacy@italfarmacogroup.com", "telephone": "+390264432529", "organisation": {"id": 102894, "type": "Pharmaceutical company", "typeCode": "10", "name": "Italfarmaco S.p.A.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100000118", "organisationLocationStatus": "Active"}}], "scientificContacts": [{"id": 304578, "type": "Scientific", "functionalName": "Nicoletta Coceani", "functionalEmailAddress": "n.coceani@italfarmacogroup.com", "telephone": "+390264432529", "organisation": {"id": 102894, "type": "Pharmaceutical company", "typeCode": "10", "name": "Italfarmaco S.p.A.", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100000118", "organisationLocationStatus": "Active"}}], "thirdParties": [{"id": 447289, "organisationAddress": {"id": 660950, "organisation": {"id": 698983, "type": "Hospital/Clinic/Other health care facility", "typeCode": "8", "name": "Illingworth Research Group Limited", "commercial": false, "isBusinessKeyValidated": true, "businessKey": "ORG-100042356", "organisationLocationStatus": "Active"}, "address": {"addressId": 718797, "oneLine": "Hazelwood House, Larkwood Way, Tytherington Business Park", "addressLine1": "Hazelwood House", "addressLine2": "Larkwood Way", "addressLine3": "Tytherington Business Park", "addressLine4": "", "city": "Macclesfield", "postcode": "SK10 2XR", "country": 826, "countryName": "United Kingdom"}, "phone": "+441625310020", "email": "robert.miller@illingworthresearch.com", "isBusinessKeyValidated": true, "businessKey": "ORG-100042356"}, "sponsorDuties": [{"id": 700094, "code": "15", "value": "Home Nursing"}], "phoneNumber": "+441625310020", "email": "robert.miller@illingworthresearch.com"}, {"id": 447283, "organisationAddress": {"id": 462561, "organisation": {"id": 494878, "type": "Pharmaceutical company", "typeCode": "10", "name": "Catalent Germany Schorndorf GmbH", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100011845", "organisationLocationStatus": "Active"}, "address": {"addressId": 495875, "oneLine": "Steinbeisstrasse 1-2", "addressLine1": "Steinbeisstrasse 1-2", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Schorndorf", "postcode": "73614", "country": 2013, "countryName": "Germany"}, "phone": "+49718170000", "email": "Lauren.Carter@catalent.com", "isBusinessKeyValidated": true, "businessKey": "ORG-100011845"}, "sponsorDuties": [{"id": 700087, "code": "14"}], "phoneNumber": "+49718170000", "email": "Lauren.Carter@catalent.com"}, {"id": 447291, "organisationAddress": {"id": 660944, "organisation": {"id": 698977, "type": "Non-Pharmaceutical company", "typeCode": "11", "name": "Fountayn LLC", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100051582", "organisationLocationStatus": "Active"}, "address": {"addressId": 718791, "oneLine": "5900 Landerbrook Drive Suite 170", "addressLine1": "5900 Landerbrook Drive Suite 170", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Mayfield Heights", "postcode": "44124-4085", "country": 840, "countryName": "United States"}, "phone": "+390458202666", "email": "valentina.biasi@alirahealth.com", "isBusinessKeyValidated": true, "businessKey": "ORG-100051582"}, "sponsorDuties": [{"id": 700096, "code": "7"}], "phoneNumber": "+390458202666", "email": "valentina.biasi@alirahealth.com"}, {"id": 447292, "organisationAddress": {"id": 660947, "organisation": {"id": 698980, "type": "Non-Pharmaceutical company", "typeCode": "11", "name": "ATOM International Limited", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100042393", "organisationLocationStatus": "Active"}, "address": {"addressId": 718794, "oneLine": "Office 16, Valley House, Seventh Avenue, Team Valley Trading Estate", "addressLine1": "Office 16", "addressLine2": "Valley House", "addressLine3": "Seventh Avenue", "addressLine4": "Team Valley Trading Estate", "city": "Gateshead", "postcode": "NE11 0JW", "country": 826, "countryName": "United Kingdom"}, "phone": "000000000", "email": "michelle.eagle@atom-international.org", "isBusinessKeyValidated": true, "businessKey": "ORG-100042393"}, "sponsorDuties": [{"id": 700097, "code": "15", "value": "physioterapist training and evaluation"}], "phoneNumber": "000000000", "email": "michelle.eagle@atom-international.org"}, {"id": 447281, "organisationAddress": {"id": 471441, "organisation": {"id": 503816, "type": "Pharmaceutical company", "typeCode": "10", "name": "IQVIA Limited", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100008655", "organisationLocationStatus": "Active"}, "address": {"addressId": 505494, "oneLine": "3 Forbury Place, 23 Forbury Road", "addressLine1": "3 Forbury Place", "addressLine2": "23 Forbury Road", "addressLine3": "", "addressLine4": "", "city": "Reading", "postcode": "RG1 3JH", "country": 826, "countryName": "United Kingdom"}, "phone": "+441184506016", "email": "eu_clinical_trials_information@iqvia.com", "isBusinessKeyValidated": true, "businessKey": "ORG-100008655"}, "sponsorDuties": [{"id": 700080, "code": "1"}, {"id": 700081, "code": "11"}, {"id": 700082, "code": "12"}, {"id": 700083, "code": "13"}, {"id": 700084, "code": "5"}, {"id": 700085, "code": "8"}], "phoneNumber": "+441184506016", "email": "eu_clinical_trials_information@iqvia.com"}, {"id": 447294, "organisationAddress": {"id": 462572, "organisation": {"id": 494889, "type": "Laboratory/Research/Testing facility", "typeCode": "9", "name": "Acm Global Central Laboratory Limited", "commercial": true, "isBusinessKeyValidated": true, "businessKey": "ORG-100042459", "organisationLocationStatus": "Active"}, "address": {"addressId": 495887, "oneLine": "23 Hospital Fields Road, Fulford Industrial Estate", "addressLine1": "23 Hospital Fields Road", "addressLine2": "Fulford Industrial Estate", "addressLine3": "", "addressLine4": "", "city": "York", "postcode": "YO10 4DZ", "country": 826, "countryName": "United Kingdom"}, "phone": "+441904699400", "email": "ACMYorkEUCTAGroup@acmgloballab.com", "isBusinessKeyValidated": true, "businessKey": "ORG-100042459"}, "sponsorDuties": [{"id": 700099, "code": "15", "value": "Primary/ surrogate endpoint test"}, {"id": 700100, "code": "4"}], "phoneNumber": "+441904699400", "email": "ACMYorkEUCTAGroup@acmgloballab.com"}, {"id": 447288, "organisationAddress": {"id": 660951, "organisation": {"id": 698984, "type": "Hospital/Clinic/Other health care facility", "typeCode": "8", "name": "Patient Primary Limited", "commercial": false, "isBusinessKeyValidated": true, "businessKey": "ORG-100052459", "organisationLocationStatus": "Active"}, "address": {"addressId": 718799, "oneLine": "Building 329, Doncastle Road", "addressLine1": "Building 329", "addressLine2": "Doncastle Road", "addressLine3": "", "addressLine4": "", "city": "Bracknell", "postcode": "RG12 8PE", "country": 826, "countryName": "United Kingdom"}, "phone": "+441344577929", "email": "matthew.newell@patientprimary.com", "isBusinessKeyValidated": 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fibras con positividad para la distrofina en un ensayo de inmunofluorescencia (PDPF) [plazo: momento basal, semana 12].\n\u2022\tConcentraci\u00f3n media de copias de vectores gen\u00f3micos, determinada mediante una reacci\u00f3n en cadena de la polimerasa con muestras de biopsia de tejido muscular despu\u00e9s de la administraci\u00f3n de delandistrog\u00e9n moxeparvovec [plazo: semana 12].", "language": 7, "languageDescription": "Spanish"}], "secondaryObjectives": [{"id": 262518, "number": 1, "secondaryObjective": "Maximum Observed Plasma Concentration (Cmax) of Imlifidase [Time Frame: Up to Day 7]", "secondaryObjectiveTranslations": [{"id": 5845449, "uuid": "b79a0c84-a770-447e-8438-9b22c4214b97", "attributeTranslation": "Concentraci\u00f3n plasm\u00e1tica m\u00e1xima (Cm\u00e1x) observada de imlifidasa [plazo: hasta el d\u00eda 7].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 262519, "number": 2, "secondaryObjective": "Total IgG in Serum After Imlifidase Administration [Time Frame: Up to Week 12]", "secondaryObjectiveTranslations": [{"id": 5845450, "uuid": "c78e573a-2d46-4f54-89b0-dd1ccaea81f1", "attributeTranslation": "IgG total en suero despu\u00e9s de la administraci\u00f3n de imlifidasa [plazo: hasta la semana 12].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 262520, "number": 3, "secondaryObjective": "rAAVrh74 Antibody Titers After Imlifidase Administration [Time Frame: Up to Hour 120]", "secondaryObjectiveTranslations": [{"id": 5845451, "uuid": "a0a2643e-9af0-4b6a-82d4-e8c81b3323c3", "attributeTranslation": "Valores de anticuerpos contra rAAVrh74 despu\u00e9s de la administraci\u00f3n de imlifidasa [plazo: hasta la hora 120].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 262521, "number": 4, "secondaryObjective": "Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration [Time Frame: Up to Day 7]", "secondaryObjectiveTranslations": [{"id": 5845452, "uuid": "04539dbd-3bdf-4ce7-b8e3-6552c0dc772d", "attributeTranslation": "Concentraci\u00f3n de copias de vectores gen\u00f3micos, determinada mediante una reacci\u00f3n en cadena de la polimerasa en suero despu\u00e9s de la administraci\u00f3n de delandistrog\u00e9n moxeparvovec [plazo: hasta el d\u00eda 7].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 262522, "number": 5, "secondaryObjective": "Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [Time Frame: Up to Week 104]", "secondaryObjectiveTranslations": [{"id": 5845453, "uuid": "62c36ca8-b152-4de6-a015-1e7ef7479a19", "attributeTranslation": "N\u00famero de participantes con un acontecimiento adverso surgido durante el tratamiento (AAST), un acontecimiento adverso de especial inter\u00e9s (AAEI) y un acontecimiento adverso grave (AAG) [plazo: hasta la semana 104].", "language": 7, "languageDescription": "Spanish"}]}]}, "eligibilityCriteria": {"principalInclusionCriteria": [{"id": 508996, "number": 1, "principalInclusionCriteria": "Ambulatory per protocol specified criteria.", "principalInclusionCriteriaTranslations": [{"id": 5845441, "uuid": "5dcde588-edad-4336-b9c0-0131decf2474", "attributeTranslation": "Ser deambulante, seg\u00fan los criterios especificados en el protocolo.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 508997, "number": 2, "principalInclusionCriteria": "Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.", "principalInclusionCriteriaTranslations": [{"id": 5845442, "uuid": "2104f2e4-2aa0-45ac-982a-4a8ba222a440", "attributeTranslation": "Tener un diagn\u00f3stico definitivo de DMD antes de la selecci\u00f3n, en base a hallazgos cl\u00ednicos documentados y pruebas gen\u00e9ticas confirmatorias.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 508998, "number": 3, "principalInclusionCriteria": "Ability to cooperate with motor assessment testing.", "principalInclusionCriteriaTranslations": [{"id": 5845443, "uuid": "fadd293b-53d6-46ca-80d3-f6048d7155d8", "attributeTranslation": "Ser capaz de cooperar durante las pruebas de evaluaci\u00f3n motora.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 508999, "number": 4, "principalInclusionCriteria": "Has elevated rAAVrh74 antibody titers per protocol-specified requirements.", "principalInclusionCriteriaTranslations": [{"id": 5845444, "uuid": "ca9c2d84-cd85-4324-ac03-7c3e0eda8974", "attributeTranslation": "Tener valores elevados de anticuerpos contra rAAVrh74 seg\u00fan los requisitos especificados en el protocolo.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 509000, "number": 5, "principalInclusionCriteria": "A pathogenic frameshift mutation, nonsense mutation or premature stop codon or pathogenic variant in the DMD gene that is expected to lead to absence of dystrophin protein.", "principalInclusionCriteriaTranslations": [{"id": 5845445, "uuid": "98de0cf3-c7ba-42bd-a2db-ac7eeb18e8b3", "attributeTranslation": "Tener una mutaci\u00f3n pat\u00f3gena de desplazamiento del marco de lectura, una mutaci\u00f3n interruptora o un cod\u00f3n finalizador prematuro, u otra variante pat\u00f3gena en el gen DMD que se espera que conlleve la ausencia de prote\u00edna distrofina.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 509001, "number": 6, "principalInclusionCriteria": "Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).", "principalInclusionCriteriaTranslations": [{"id": 5845446, "uuid": "ce32f2dd-7af9-42e0-b5db-88156347e64b", "attributeTranslation": "Haber recibido una dosis diaria estable de corticoesteroides orales durante un m\u00ednimo de 12 semanas antes de la selecci\u00f3n y tener previsto que la dosis se mantenga constante durante todo el estudio (excepto posibles modificaciones para adaptarla a los cambios de peso).", "language": 7, "languageDescription": "Spanish"}]}], "principalExclusionCriteria": [{"id": 877302, "number": 1, "principalExclusionCriteria": "Previous treatment with imlifidase.", "principalExclusionCriteriaTranslations": [{"id": 5845436, "uuid": "74cdc2c7-2502-4271-8ddb-404c829497ea", "attributeTranslation": "Haber recibido tratamiento previo con imlifidasa.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 877303, "number": 2, "principalExclusionCriteria": "Presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for receiving the study drugs or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.", "principalExclusionCriteriaTranslations": [{"id": 5845437, "uuid": "2c76d8d1-4e7d-4df5-87b7-b2882a9bced6", "attributeTranslation": "Presentar cualquier otra enfermedad cl\u00ednicamente significativa, como enfermedades card\u00edacas, pulmonares, hep\u00e1ticas, renales, hematol\u00f3gicas, inmunitarias o del comportamiento; una infecci\u00f3n, neoplasia maligna, enfermedad concomitante o necesidad de tratamiento farmacol\u00f3gico cr\u00f3nico que, en opini\u00f3n del investigador, suponga riesgos innecesarios a la hora de recibir los f\u00e1rmacos del estudio; o una afecci\u00f3n m\u00e9dica o circunstancia atenuante que, en opini\u00f3n del investigador, podr\u00eda afectar a la capacidad del participante de completar las pruebas o los procedimientos requeridos por el protocolo, o bien comprometer el bienestar, la seguridad o la interpretabilidad cl\u00ednica del participante y sus resultados.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 877304, "number": 3, "principalExclusionCriteria": "Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.", "principalExclusionCriteriaTranslations": [{"id": 5845438, "uuid": "10ea8d9f-0e35-46c7-b191-af7558e6655c", "attributeTranslation": "Haber recibido tratamiento g\u00e9nico, alguna medicaci\u00f3n en investigaci\u00f3n u otro tratamiento especificado en el protocolo dentro de los l\u00edmites de tiempo especificados en el protocolo.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 877305, "number": 4, "principalExclusionCriteria": "Abnormality in protocol-specified diagnostic evaluations or laboratory tests. Note: Other inclusion or exclusion criteria could apply.", "principalExclusionCriteriaTranslations": [{"id": 5845439, "uuid": "4290b32e-f4f6-46bc-92f1-01e39efa700e", "attributeTranslation": "Presentar alguna anomal\u00eda en las evaluaciones diagn\u00f3sticas o pruebas anal\u00edticas especificadas en el protocolo.\nNota: Pueden aplicarse otros criterios de inclusi\u00f3n o exclusi\u00f3n.", "language": 7, "languageDescription": "Spanish"}]}]}, "endPoint": {"primaryEndPoints": [{"id": 592975, "number": 1, "endPoint": "Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content [Time Frame: Baseline, Week 12]", "isPrimary": true, "endPointTranslations": [{"id": 5845427, "uuid": "97471ffb-a0a9-4746-a39a-b7bc94ff46c9", "attributeTranslation": "\u2022\tCambio desde el momento basal en el nivel de expresi\u00f3n de la prote\u00edna distrofina producida a partir del delandistrog\u00e9n moxeparvovec, determinado mediante un ensayo de inmunoelectrotransferencia Western blot ajustado por el contenido muscular [plazo: momento basal, semana 12].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 592976, "number": 2, "endPoint": "Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity [Time Frame: Baseline, Week 12]", "isPrimary": true, "endPointTranslations": [{"id": 5845428, "uuid": "3f76e64a-bc4a-4797-a0bb-cb098709a6b5", "attributeTranslation": "\u2022\tCambio desde el momento basal en el nivel de expresi\u00f3n de la prote\u00edna distrofina producida a partir del delandistrog\u00e9n moxeparvovec en muestras de biopsia de tejido muscular, determinado seg\u00fan la intensidad de las fibras en un ensayo de inmunofluorescencia [plazo: momento basal, semana 12].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 592977, "number": 3, "endPoint": "Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF) [Time Frame: Baseline, Week 12]", "isPrimary": true, "endPointTranslations": [{"id": 5845429, "uuid": "447bb0cf-bd36-496f-bfab-e99d14e668b0", "attributeTranslation": "\u2022\tCambio desde el momento basal en el nivel de expresi\u00f3n de la prote\u00edna distrofina producida a partir del delandistrog\u00e9n moxeparvovec en muestras de biopsia de tejido muscular, determinado seg\u00fan el porcentaje de fibras con positividad para la distrofina en un ensayo de inmunofluorescencia (PDPF) [plazo: momento basal, semana 12].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 592978, "number": 4, "endPoint": "Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration [Time Frame: Week 12]", "isPrimary": true, "endPointTranslations": [{"id": 5845430, "uuid": "3fcf5256-912d-4e81-ab25-1705bf8050f9", "attributeTranslation": "\u2022\tConcentraci\u00f3n media de copias de vectores gen\u00f3micos, determinada mediante una reacci\u00f3n en cadena de la polimerasa con muestras de biopsia de tejido muscular despu\u00e9s de la administraci\u00f3n de delandistrog\u00e9n moxeparvovec [plazo: semana 12].", "language": 7, "languageDescription": "Spanish"}]}], "secondaryEndPoints": [{"id": 592979, "number": 1, "endPoint": "Maximum Observed Plasma Concentration (Cmax) of Imlifidase [Time Frame: Up to Day 7]", "isPrimary": false, "endPointTranslations": [{"id": 5845431, "uuid": "be42918d-265c-414f-b003-077e7be1b6ba", "attributeTranslation": "Concentraci\u00f3n plasm\u00e1tica m\u00e1xima (Cm\u00e1x) observada de imlifidasa [plazo: hasta el d\u00eda 7].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 592980, "number": 2, "endPoint": "Total IgG in Serum After Imlifidase Administration [Time Frame: Up to Week 12]", "isPrimary": false, "endPointTranslations": [{"id": 5845432, "uuid": "45f61fe4-029b-43f3-a36b-7f873b85211e", "attributeTranslation": "IgG total en suero despu\u00e9s de la administraci\u00f3n de imlifidasa [plazo: hasta la semana 12].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 592981, "number": 3, "endPoint": "rAAVrh74 Antibody Titers After Imlifidase Administration [Time Frame: Up to Hour 120]", "isPrimary": false, "endPointTranslations": [{"id": 5845433, "uuid": "532d58a5-2584-4e4d-ba16-bc86831656f4", "attributeTranslation": "Valores de anticuerpos contra rAAVrh74 despu\u00e9s de la administraci\u00f3n de imlifidasa [plazo: hasta la hora 120].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 592982, "number": 4, "endPoint": "Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration [Time Frame: Up to Day 7]", "isPrimary": false, "endPointTranslations": [{"id": 5845434, "uuid": "0b387c7a-2f61-4de3-9647-9e545d49f40e", "attributeTranslation": "Concentraci\u00f3n de copias de vectores gen\u00f3micos, determinada mediante una reacci\u00f3n en cadena de la polimerasa en suero despu\u00e9s de la administraci\u00f3n de delandistrog\u00e9n moxeparvovec [plazo: hasta el d\u00eda 7].", "language": 7, "languageDescription": "Spanish"}]}, {"id": 592983, "number": 5, "endPoint": "Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [Time Frame: Up to Week 104]", "isPrimary": false, "endPointTranslations": [{"id": 5845435, "uuid": "34266710-58ea-4460-9bf3-e189febd110b", "attributeTranslation": "N\u00famero de participantes con un acontecimiento adverso surgido durante el tratamiento (AAST), un acontecimiento adverso de especial inter\u00e9s (AAEI) y un acontecimiento adverso grave (AAG) [plazo: hasta la semana 104].", "language": 7, "languageDescription": "Spanish"}]}]}, "trialDuration": {"estimatedEndDate": "2027-06-30", "estimatedRecruitmentStartDate": "2024-01-17"}, "sourceOfMonetarySupport": [{"id": 56140, "organisationName": "Sarepta Therapeutics, Inc"}], "populationOfTrialSubjects": {"ageRanges": [{"id": 213563, "ageRangeCategoryCode": "2", "ageRangeCategory": "2"}], "ageRangeSecondaryIds": [{"id": 213565, "ageRangeCategoryCode": "2", "ctAgeRangeCode": "4", "ageRangeCategory": "2", "ctAgeRange": "4"}, {"id": 213564, "ageRangeCategoryCode": "2", "ctAgeRangeCode": "5", "ageRangeCategory": "2", "ctAgeRange": "5"}], "clinicalTrialGroups": [{"code": "2", "name": "Patients"}], "isFemaleSubjects": false, "isMaleSubjects": true, "isVulnerablePopulationSelected": true}, "individualParticipantData": {"planToShareIPD": "2"}}, "protocolInformation": {"studyDesign": {"periodDetails": []}}, "scientificAdviceAndPip": {"scientificAdvices": [{"id": 30786, "competentAuthority": {"id": 559911, "organisation": {"id": 592573, "type": "EU Institution/Body/Agency", "typeCode": "14", "name": "European Medicines Agency", "commercial": false, "isBusinessKeyValidated": true, "businessKey": "ORG-100013412", "organisationLocationStatus": "Active"}, "address": {"addressId": 602937, "oneLine": "Domenico Scarlattilaan 6", "addressLine1": "Domenico Scarlattilaan 6", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Amsterdam", "postcode": "1083 HS", "country": 2029, "countryName": "Netherlands"}, "isBusinessKeyValidated": true, "businessKey": "ORG-100013412"}}, {"id": 30787, "competentAuthority": {"id": 559913, "organisation": {"id": 592575, "type": "EEA National Competent authority", "typeCode": "16", "name": "Federal Agency For Medicines And Health Products", "commercial": false, "isBusinessKeyValidated": true, "businessKey": "ORG-100003913", "organisationLocationStatus": "Active"}, "address": {"addressId": 602939, "oneLine": "Galileelaan 5/03", "addressLine1": "Galileelaan 5/03", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Sint-Joost-Ten-Node", "postcode": "1210", "country": 2002, "countryName": "Belgium"}, "isBusinessKeyValidated": true, "businessKey": "ORG-100003913"}}], "paediatricInvestigationPlan": []}, "associatedClinicalTrials": [{"id": 19695, "ctNumber": "2022-000691-19", "fullTitle": "A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy, Estudio abierto, de dos partes, de administraci\u00f3n g\u00e9nica sist\u00e9mica para evaluar la seguridad y la expresi\u00f3n de RO7494222 (SRP-9001) en sujetos menores de cuatro a\u00f1os con Distrofia Muscular de Duchenne, Uno studio in due parti, in aperto, sulla terapia genica sistemica per valutare la sicurezza e l\u2019espressione di RO7494222 (SRP-9001) in soggetti di et\u00e0 inferiore a quattro anni affetti da distrofia muscolare di Duchenne, Etude en 2 parties, en ouvert, de transfert de g\u00e8ne par voir syst\u00e9mique, \u00e9valuant la s\u00e9curit\u00e9 et l'expression de RO7494222 (SRP-9001) chez l'enfant \u00e2g\u00e9 de moins de quatre ans pr\u00e9sentant une dystrophie musculaire de Duchenne\n", "sponsorAgreementOption": "OPTIONAL", "sponsorAgreementOptionName": "OPTIONAL", "hasDocument": false, "associatedCtDocs": []}, {"id": 19696, "ctNumber": "2020-002372-13", "fullTitle": "A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)\n\n, \u00c9tude de phase 3, internationale, randomis\u00e9e, en double aveugle, contr\u00f4l\u00e9e contre placebo, de th\u00e9rapie g\u00e9nique syst\u00e9mique, destin\u00e9e \u00e0 \u00e9valuer la s\u00e9curit\u00e9 d\u2019emploi et l\u2019efficacit\u00e9 du SRP9001 chez des patients non ambulatoires et ambulatoires atteints de dystrophie musculaire de Duchenne (ENVISION), Studio di fase 3, internazionale, randomizzato, in doppio cieco, controllato verso placebo, sulla terapia di trasferimento sistemico di geni, per valutare la sicurezza e l\u2019efficacia di SRP-9001 in soggetti deambulanti e non deambulanti con distrofia muscolare di Duchenne (ENVISION), Estudio de fase 3, multinacional, aleatorizado, en doble ciego y controlado con placebo, del tratamiento mediante transferencia g\u00e9nica para evaluar la seguridad y la eficacia de SRP-9001 en sujetos, no ambulatorios y ambulatorios, con distrofia muscular de Duchenne (ENVISION)", "sponsorAgreementOption": "OPTIONAL", "sponsorAgreementOptionName": "OPTIONAL", "hasDocument": false, "associatedCtDocs": []}, {"id": 19694, "ctNumber": "2019-003374-91", "fullTitle": "A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)\n, \u00c9tude de phase 3, 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"language": 7, "languageDescription": "Spanish"}, {"id": 5950286, "uuid": "3eb3e68c-274d-424b-a508-8b7003d80aa7", "attributeTranslation": "Signature du formulaire de consentement \u00e9clair\u00e9", "language": 10, "languageDescription": "French"}]}, {"id": 516732, "number": 2, "principalInclusionCriteria": "Signed Assent Form when appropriate, as determined by patient's age and individual site and country standards", "principalInclusionCriteriaTranslations": [{"id": 5950287, "uuid": "b5e974c7-24e5-4cf4-a6e6-3f2839162bdc", "attributeTranslation": "Signature du formulaire de consentement \u00e9clair\u00e9 pour mineur (formulaire d'assentiment) le cas \u00e9ch\u00e9ant, en fonction de l'\u00e2ge du patient et des normes du pays et de chaque centre d'\u00e9tude", "language": 10, "languageDescription": "French"}, {"id": 5950288, "uuid": "b5e974c7-24e5-4cf4-a6e6-3f2839162bdc", "attributeTranslation": "Firma del documento de asentimiento cuando proceda, seg\u00fan la edad del paciente y las normas de cada centro y pa\u00eds", "language": 7, "languageDescription": "Spanish"}]}, {"id": 516733, "number": 3, "principalInclusionCriteria": "Male at birth", "principalInclusionCriteriaTranslations": [{"id": 5950289, "uuid": "cb6d55a9-d8dd-4eb7-89fa-68407522a8c2", "attributeTranslation": "Var\u00f3n al nacer", "language": 7, "languageDescription": "Spanish"}, {"id": 5950290, "uuid": "cb6d55a9-d8dd-4eb7-89fa-68407522a8c2", "attributeTranslation": "Sexe masculin \u00e0 la naissance", "language": 10, "languageDescription": "French"}]}, {"id": 516734, "number": 4, "principalInclusionCriteria": "Meets the following age requirements at the time of study drug infusion: \u2013 For Cohort A: 3 years of age \u2013 For Cohort B: 2 years of age \u2013 For Cohort C: >6 months to <2 years of age \u2013 For Cohort D: \u22646 months of age Note: To ensure that cohort-specific age criteria are met at the time of dosing, at the time of signing the Informed Consent Form participants should be approximately 1 month younger than the maximum age to qualify for a cohort that is actively enrolling.", "principalInclusionCriteriaTranslations": [{"id": 5950292, "uuid": "9ce3a1a8-0b3e-41b5-ba9a-3230d8724bda", "attributeTranslation": "R\u00e9pondant aux crit\u00e8res d'\u00e2ge suivants au moment de la perfusion du m\u00e9dicament \u00e0 l'\u00e9tude :\n\u2013 Pour la cohorte A : 3 ans\n\u2013 Pour la cohorte B : 2 ans\n\u2013 Pour la Cohorte C : > 6 mois \u00e0 < 2 ans\n\u2013 Pour la Cohorte D : \u22646 mois\nRemarque : Pour garantir que les crit\u00e8res d'\u00e2ge sp\u00e9cifiques \u00e0 la cohorte sont respect\u00e9s au moment de l'administration du traitement, au moment de la signature du formulaire de consentement \u00e9clair\u00e9, les participants devront \u00eatre \u00e2g\u00e9s d'environ un mois de moins que l'\u00e2ge maximum exig\u00e9 pour la qualification dans une cohorte en cours de recrutement actif.", "language": 10, "languageDescription": "French"}, {"id": 5950291, "uuid": "9ce3a1a8-0b3e-41b5-ba9a-3230d8724bda", "attributeTranslation": "Cumplimiento de los siguientes requisitos de edad en el momento de la infusi\u00f3n del f\u00e1rmaco del estudio: - En la Cohorte A: 3 a\u00f1os de edad - En la Cohorte B: 2 a\u00f1os de edad - En la Cohorte C: >6 meses a <2 a\u00f1os de edad - En la Cohorte D: </=6 meses de edad Nota: Para garantizar que se cumplan los criterios de edad espec\u00edficos de la cohorte en el momento de la administraci\u00f3n, cuando se firme el documento de consentimiento informado los participantes deber\u00e1n tener aproximadamente 1 mes menos de la edad m\u00e1xima permitida en una cohorte que se encuentre abierta a la inclusi\u00f3n.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 516735, "number": 5, "principalInclusionCriteria": "Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. Genetic report must describe a frameshift deletion, frameshift duplication, premature stop (\"nonsense\"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 79 (inclusive) that is expected to lead to absence of dystrophin protein. \u2013 Mutations between or including exons 1-17 are not eligible. \u2013 In-frame deletions, in-frame duplications, and variants of uncertain significance (\"VUS\") are not eligible.", "principalInclusionCriteriaTranslations": [{"id": 5950293, "uuid": "3cd2265d-0a73-4593-b325-ec3367bcac43", "attributeTranslation": "Tener un diagn\u00f3stico definitivo de distrofia muscular de Duchenne (DMD) antes de la selecci\u00f3n, basado en la documentaci\u00f3n de los hallazgos cl\u00ednicos y las pruebas gen\u00e9ticas confirmatorias previas mediante un m\u00e9todo de diagn\u00f3stico cl\u00ednico. El informe gen\u00e9tico debe describir una deleci\u00f3n con desplazamiento del marco de lectura, una duplicaci\u00f3n con desplazamiento del marco de lectura, una parada prematura (\u201csin sentido\u201d), una mutaci\u00f3n del sitio de empalme can\u00f3nico u otra variante patog\u00e9nica 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 prote\u00edna distrofina. \u2013 Las mutaciones entre los exones 1-17 o que los incluyan no son elegibles. \u2013 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 d\u00e9finitif de DMD avant la s\u00e9lection, fond\u00e9 sur la documentation des observations cliniques et des analyses g\u00e9n\u00e9tiques de confirmation effectu\u00e9es pr\u00e9c\u00e9demment avec un test g\u00e9n\u00e9tique diagnostique clinique. Le compte-rendu g\u00e9n\u00e9tique doit d\u00e9crire une d\u00e9l\u00e9tion avec d\u00e9calage de cadre, une duplication avec d\u00e9calage de cadre, un arr\u00eat pr\u00e9matur\u00e9 (\u00ab non sens \u00bb), une mutation par \u00e9pissage canonique, ou tout autre variant pathog\u00e8ne du g\u00e8ne DMD totalement contenu entre les exons 18 \u00e0 79 (inclus) conduisant \u00e0 une absence de la prot\u00e9ine dystrophine.\n\u2013 Les mutations entre les exons 1 \u00e0 17 ne sont pas \u00e9ligibles.\n\u2013 Les d\u00e9l\u00e9tions sans d\u00e9calage de cadre, duplications sans d\u00e9calage de cadre ou variant de signification incertaine (VSI) ne sont pas \u00e9ligibles.", "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 evaluaci\u00f3n motora adecuadas a su edad, en opini\u00f3n del investigador", "language": 7, "languageDescription": "Spanish"}, {"id": 5950295, "uuid": "2569552b-c38e-4236-b474-5121bf084b5b", "attributeTranslation": "Capacit\u00e9 de coop\u00e9rer pour les tests d'\u00e9valuation motrice appropri\u00e9s par rapport \u00e0 l'\u00e2ge, selon l'avis de l'investigateur.", "language": 10, "languageDescription": "French"}]}, {"id": 516737, "number": 7, "principalInclusionCriteria": "Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all protocol requirements.", "principalInclusionCriteriaTranslations": [{"id": 5950298, "uuid": "d252fe40-3e85-40fc-81a2-77301274cc85", "attributeTranslation": "Tener uno o m\u00e1s progenitores o tutores legales que sean capaces de entender y cumplir el calendario de visitas del estudio y todos los requisitos del protocolo", "language": 7, "languageDescription": "Spanish"}, {"id": 5950297, "uuid": "d252fe40-3e85-40fc-81a2-77301274cc85", "attributeTranslation": "Participant ayant un ou des parent(s) ou aidant(s) l\u00e9gal(aux) capable(s) de comprendre et de respecter le calendrier des visites de l'\u00e9tude 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 t\u00edtulos elevados de anticuerpos anti rAAVrh74 seg\u00fan lo determinado por un ensayo experimental de anticuerpos Elecsys anti-rAAVrh74 obtenido dentro de los 31 d\u00edas posteriores al d\u00eda de perfusi\u00f3n", "language": 7, "languageDescription": "Spanish"}, {"id": 5950246, "uuid": "227d6072-c409-4321-b93d-7992a66ce563", "attributeTranslation": "Pr\u00e9senter des titres \u00e9lev\u00e9s d'anticorps anti-rAAVrh74 d\u00e9termin\u00e9s par un dosage exp\u00e9rimental des anticorps anti-rAAVrh74 Elecsys r\u00e9alis\u00e9 au cours des 31 jours pr\u00e9c\u00e9dant 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": "Pr\u00e9sence d'une infection symptomatique (par exemple, infection des voies respiratoires sup\u00e9rieures, pneumonie, py\u00e9lon\u00e9phrite, m\u00e9ningite) dans les 4 semaines avant le Jour 1", "language": 10, "languageDescription": "French"}, {"id": 5950248, "uuid": "4465449f-faa5-4326-9d6a-45b4581f423d", "attributeTranslation": "Infecci\u00f3n sintom\u00e1tica (p. ej., de las v\u00edas respiratorias superiores, neumon\u00eda, pielonefritis, meningitis) en un plazo de 4 semanas antes del d\u00eda 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 serol\u00f3gicas 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: R\u00e9sultat positif \u00e0 l'analyse s\u00e9rologique pour le VIH1 et/ou 2, l'h\u00e9patite C ou l'h\u00e9patite B", "language": 10, "languageDescription": "French"}]}, {"id": 889932, "number": 12, "principalExclusionCriteria": "Cohort C and D: \u2013 Born prematurely (before completion of gestation at 37 weeks) or relevant pregnancy complications in the opinion of the investigator \u2013 Participant's mother: Serological evidence of current, chronic, or active HIV (1 and/or 2), hepatitis B, or hepatitis C infection \u2013 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\u2013Naissance pr\u00e9matur\u00e9e (avant la fin de 37semaines de grossesse \u00e0 37semaines) ou complications significatives de la grossesse, selon l'avis de l'investigateur\n\n\u2013M\u00e8re du participant: Signes s\u00e9rologiques d'infection en cours, chronique ou active par VIH 1 et/ou 2, le virus de l'h\u00e9patite B ou le virus de l'h\u00e9patite C\n\n\u2013M\u00e8re du participant si allaitement:\noSignes cliniques d'infection aigu\u00eb par le CMV avec confirmation par analyse PCR du CMV (\u00e9chantillon urinaire)\noAnomalies cliniquement significatives de la fonction h\u00e9patique (GGT, ASAT, ALAT, PAL, bilirubine totale, GLDH) indiquant une h\u00e9patite infectieuse\noPathologie cliniquement significative ou infection aigu\u00eb indiquant une infection par virus h\u00e9patotrope (par exemple, CMV, EBV, VZV, etc) dans les 6 semaines avant le Jour1\noContact connu avec une personne atteinte d'h\u00e9patite aigu\u00eb 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: \u2013 Nacido prematuramente (antes de completar la gestaci\u00f3n a las 37 semanas) o con complicaciones relevantes del embarazo, a juicio del investigador \u2013 Madre del participante: Evidencia serol\u00f3gica de infecci\u00f3n actual, cr\u00f3nica o activa por los virus del VIH (de tipo 1 y/o 2), de la hepatitis B o de la hepatitis C \u2013 Madre del participante en caso de lactancia materna: o Signos cl\u00ednicos de infecci\u00f3n aguda por citomegalovirus con confirmaci\u00f3n por PCR (orina) o Alteraci\u00f3n funcional hep\u00e1tica cl\u00ednicamente importante (GGT, AST, ALT, fosfatasa alcalina, bilirrubina total, GLDH) indicativa de hepatitis infecciosa o Enfermedad o proceso agudo cl\u00ednicamente importante que indique infecci\u00f3n por virus hepatotropos (p. ej., citomegalovirus, virus de Epstein-Barr, virus de la varicela-z\u00f3ster, etc.) en un plazo de 6 semanas antes del d\u00eda 1 o Contacto conocido con una persona con hepatitis infecciosa aguda o activa en un plazo de 12 semanas antes del d\u00eda 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 confusi\u00f3n en el desarrollo motor, en opini\u00f3n del investigador.", "language": 7, "languageDescription": "Spanish"}, {"id": 5950253, "uuid": "5a7e8601-827e-4c83-9cba-44e098fe5696", "attributeTranslation": "Pr\u00e9sence d'un retard cognitif ou d'une insuffisance cognitive susceptible d'interf\u00e9rer sur le d\u00e9veloppement 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: \u2013 Any time: o Gene therapy o Cell-based therapy (e.g., stem cell transplantation) o CRISPR/Cas9, or any other form of gene editing \u2013 Within 12 weeks of Day 1 and any time during the study: o Use of human growth factor or vamorolone \u2013 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\u2122, EXONDYS 51\u2122, VILTEPSO\u2122)", "principalExclusionCriteriaTranslations": [{"id": 5950256, "uuid": "291f971a-268d-4fa1-a1f9-b4342488ae58", "attributeTranslation": "Tratamiento con cualquiera de las siguientes terapias durante los periodos especificados: \u2013 En cualquier momento: o Terapia g\u00e9nica o Terapia celular (p. ej., trasplante de c\u00e9lulas madre) o CRISPR/Cas9 o cualquier otra forma de edici\u00f3n de genes \u2013 En un plazo de 12 semanas respecto al d\u00eda 1 y en cualquier momento durante el estudio: o Administraci\u00f3n de factor de crecimiento humano o vamorolona \u2013 En un plazo de 6 meses respecto al d\u00eda 1 y en cualquier momento durante el estudio: o Cualquier medicamento en investigaci\u00f3n o Cualquier tratamiento dise\u00f1ado para aumentar la expresi\u00f3n de la distrofina (p. ej., Translarna\u2122, EXONDYS 51\u2122, VILTEPSO\u2122)", "language": 7, "languageDescription": "Spanish"}, {"id": 5950255, "uuid": "291f971a-268d-4fa1-a1f9-b4342488ae58", "attributeTranslation": "Administration d'un des traitemen suivants dans les limites temporelles sp\u00e9cifi\u00e9es:\n\u2013\u00c0 tout moment:\noTh\u00e9rapie g\u00e9nique\noTh\u00e9rapie cellulaire (par exemple, greffe de cellules souches)\noCRISPR/Cas9, ou toute autre forme d'\u00e9dition des g\u00e8nes\n\u2013Dans les 12 semaines avant le Jour1 et \u00e0 tout moment pendant l'\u00e9tude:\noUtilisation de facteur de croissance humain ou de vamorolone \n\u2013Dans les 6 mois avant le Jour1 et \u00e0 tout moment pendant l'\u00e9tude:\noTout m\u00e9dicament exp\u00e9rimental\noTout traitement con\u00e7u pour augmenter l'expression de la dystrophine (par exemple, Translarna\u2122, EXONDYS 51\u2122, VILTEPSO\u2122)", "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 \u00e1cido ribonucleico mensajero en un plazo de 4 semanas o una vacuna inactiva en un plazo de 2 semanas respecto a la visita del d\u00eda 1, o esperar recibir una vacuna que no pueda retrasarse razonablemente para adaptarse a la administraci\u00f3n concomitante de corticosteroides durante los 3 primeros meses despu\u00e9s del d\u00eda 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 \u00e0 ARNm dans les 4semaines ou d'un vaccin inactiv\u00e9 dans les 2semaines pr\u00e9c\u00e9dant la visite du Jour1 ou administration pr\u00e9vue d'une vaccination ne pouvant pas \u00eatre raisonnablement diff\u00e9r\u00e9e pour permettre l'administration concomitante de cortico\u00efdes pendant les 3 premiers mois apr\u00e8s le Jour1", "language": 10, "languageDescription": "French"}]}, {"id": 889936, "number": 16, "principalExclusionCriteria": "Has abnormal laboratory values considered clinically significant including but not limited to: \u2013 GGT >2 x upper limit of normal (ULN) \u2013 GLDH > ULN \u2013 Total bilirubin >ULN. o Elevations in total bilirubin confirmed to be due to Gilbert's syndrome are not exclusionary. \u2013 White blood cell count >18,500 per \u00b5l \u2013 Platelets \u2264 150,000 per \u00b5l", "principalExclusionCriteriaTranslations": [{"id": 5950260, "uuid": "ac99c281-3bd2-45ff-aa93-8915fc5ae199", "attributeTranslation": "Presentar valores de laboratorio an\u00f3malos que se consideren cl\u00ednicamente importantes, tales como, entre otros, los siguientes: \u2013 GGT >2 veces el l\u00edmite superior de la normalidad - GLDH > l\u00edmite superior de la normalidad \u2013 Bilirrubina total >l\u00edmite superior de la normalidad o La elevaci\u00f3n de la bilirrubina total confirmada por el s\u00edndrome de Gilbert no es motivo de exclusi\u00f3n \u2013 Cifra de leucocitos >18.500 por \u00b5l \u2013 Plaquetas </=150.000 por \u00b5l", "language": 7, "languageDescription": "Spanish"}, {"id": 5950259, "uuid": "ac99c281-3bd2-45ff-aa93-8915fc5ae199", "attributeTranslation": "Anomalies consid\u00e9r\u00e9es comme cliniquement significatives des param\u00e8tres biologiques, notamment:\n\u2013GGT> 2 x limite sup\u00e9rieure de la normale (LSN)\n- GLDH> LSN\n\u2013Bilirubine totale> LSN\noLes \u00e9l\u00e9vations de la bilirubine totale confirm\u00e9es comme \u00e9tant dues \u00e0 un syndrome de Gilbert ne constituent pas un crit\u00e8re de non-inclusion\n\u2013Num\u00e9ration des globules blancs (GB)>18 500 par \u00b5l\n\u2013Plaquettes \u2264 150 000 par \u00b5l", "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 opini\u00f3n 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 m\u00e9decin g\u00e9n\u00e9raliste ou m\u00e9decin de soins primaires et autres professionnels m\u00e9dicaux de la participation \u00e0 cette \u00e9tude", "language": 10, "languageDescription": "French"}, {"id": 5950263, "uuid": "dc4295d2-3de9-4205-9595-18477b02ce8a", "attributeTranslation": "Familia que no desea revelar la participaci\u00f3n del participante en el estudio a su m\u00e9dico de cabecera o m\u00e9dico de atenci\u00f3n 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 (ant\u00edgenos o PCR) el d\u00eda 1 antes de la infusi\u00f3n", "language": 7, "languageDescription": "Spanish"}, {"id": 5950266, "uuid": "255b5e54-0b96-44d3-85c1-c83134bc8b4e", "attributeTranslation": "R\u00e9sultat positif au test de d\u00e9pistage de la COVID-19 (test antig\u00e9nique 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": "Acc\u00e8s veineux p\u00e9riph\u00e9rique de mauvaise qualit\u00e9, selon l\u2019investigateur, rendant difficile les ponctions veineuses n\u00e9cessaires pour les proc\u00e9dures exig\u00e9es par le protocole.", "language": 10, "languageDescription": "French"}, {"id": 5950267, "uuid": "5141ecf5-29c0-4f71-8475-36c8bd728d9c", "attributeTranslation": "Acceso venoso perif\u00e9rico deficiente, que, en opini\u00f3n del investigador, provocar\u00e1 dificultades en las venopunciones precisas para los 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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., Blinded Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand., Blinded Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from baseline to Week 25., Blinded Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument, PedsQL™ Duchenne Muscular Dystrophy (DMD) Module Patient Report and Parent Report scores (at baseline [Day 1/Week 0], Weeks 12, 24 and pre-dose Week 25)., OLE Phase (Efficacy): 1.Percentage of treatment responders at Week 49, where a treatment responder is defined as no decline from baseline at entry into this extension study in Performance of Upper Limb (PUL 2.0)., OLE Phase (Efficacy) 2.Change in Muscle strength as assessed by and percent predicted MyoGrip (using the Myoset System) from Week 25 to Week 49., OLE Phase (Efficacy) 3.Change in Muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from Week 25 to Week 49., OLE Phase (Efficacy) 4.The extent of response in a total arm functional score from Week 25 to Week 49 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, right hand and left hand MyoGrip and MyoPinch) that do not decline. 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"14/01/2025", "conditions": "Duchenne Muscular Dystrophy", "trialCountries": ["Bulgaria:8"], "decisionDateOverall": "30/07/2024", "decisionDate": "BG: 30/07/2024", "therapeuticAreas": ["Diseases [C] - Musculoskeletal Diseases [C05]"], "sponsor": "Antisense Therapeutics Limited", "sponsorType": "Pharmaceutical company", "trialPhase": "Therapeutic exploratory (Phase II)", "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)., Blinded Phase (Efficacy) 2.Change in muscle strength as assessed by percent predicted MyoGrip (using the Myoset System) from baseline to Week 25., Blinded Phase (Efficacy) 3.Change in muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from baseline to Week 25., Blinded Phase (Efficacy) 4.The extent of response in a total arm functional score from baseline to Week 25 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, dominant hand and non-dominant hand MyoGrip and MyoPinch) that do not decline. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5., Blinded Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand., Blinded Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from baseline to Week 25., Blinded Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument, PedsQL\u2122 Duchenne Muscular Dystrophy (DMD) Module Patient Report and Parent Report scores (at baseline [Day 1/Week 0], Weeks 12, 24 and pre-dose Week 25)., OLE Phase (Efficacy): 1.Percentage of treatment responders at Week 49, where a treatment responder is defined as no decline from baseline at entry into this extension study in Performance of Upper Limb (PUL 2.0)., OLE Phase (Efficacy) 2.Change in Muscle strength as assessed by and percent predicted MyoGrip (using the Myoset System) from Week 25 to Week 49., OLE Phase (Efficacy) 3.Change in Muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from Week 25 to Week 49., OLE Phase (Efficacy) 4.The extent of response in a total arm functional score from Week 25 to Week 49 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, right hand and left hand MyoGrip and MyoPinch) that do not decline. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5., OLE Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from Week 25 to Week 49 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand., OLE Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from Week 25 to Week 49., OLE Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument PedsQL\u2122 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"},"rawRetrieveRecord":{"ctNumber": "2024-512265-13-00", "ctStatus": "Ended", "startDateEU": "2023-04-13", "endDateEU": "2025-01-14", 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[{"id": 5178995, "uuid": "77143e3c-ba54-41b3-928c-c0eabb3b75df", "attributeTranslation": "\u041c\u0443\u0441\u043a\u0443\u043b\u043d\u0430 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u044f \u043d\u0430 \u0414\u044e\u0448\u0435\u043d", "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": "\u0417\u0430\u0441\u043b\u0435\u043f\u0435\u043d\u0430 \u0444\u0430\u0437\u0430:\n\u0414\u0430 \u0441\u0435 \u043e\u0446\u0435\u043d\u0438 \u0435\u0444\u0435\u043a\u0442\u0430 \u043d\u0430 ATL1102 \u0432\u044a\u0440\u0445\u0443 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f \u043d\u0430 \u0433\u043e\u0440\u043d\u0438\u0442\u0435 \u043a\u0440\u0430\u0439\u043d\u0438\u0446\u0438 \u043f\u0440\u0438 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\u043f\u0440\u043e\u0434\u044a\u043b\u0436\u0430\u0432\u0430\u0449\u0430\u0442\u0430 \u0431\u0435\u0437\u043e\u043f\u0430\u0441\u043d\u043e\u0441\u0442 \u0438 \u043f\u043e\u043d\u043e\u0441\u0438\u043c\u043e\u0441\u0442 \u043d\u0430 ATL1102, \u043f\u0440\u0438\u043b\u0430\u0433\u0430\u043d \u0432\u0435\u0434\u043d\u044a\u0436 \u0441\u0435\u0434\u043c\u0438\u0447\u043d\u043e \u0447\u0440\u0435\u0437 \u043f\u043e\u0434\u043a\u043e\u0436\u043d\u0430 \u0438\u043d\u0436\u0435\u043a\u0446\u0438\u044f, \u043f\u0440\u0438 \u043d\u0435\u0430\u043c\u0431\u0443\u043b\u0430\u043d\u0442\u043d\u0438 \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u0446\u0438 \u0441 \u041c\u0414\u0414.\n\u0417\u0430 \u0446\u0435\u043b\u0438\u0442\u0435 \u043d\u0430 \u043f\u044a\u0440\u0432\u0438\u0447\u043d\u0438\u0442\u0435 \u043a\u043e\u043c\u0431\u0438\u043d\u0438\u0440\u0430\u043d\u0438 \u0437\u0430\u0441\u043b\u0435\u043f\u0435\u043d\u0438 \u0438 OLE \u0444\u0430\u0437\u0438, \u043c\u043e\u043b\u044f, \u0432\u0438\u0436\u0442\u0435 \u0440\u0430\u0437\u0434\u0435\u043b 4.3 \u043e\u0442 \u043f\u0440\u043e\u0442\u043e\u043a\u043e\u043b\u0430", "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": "\u0412\u0442\u043e\u0440\u0438\u0447\u043d\u0438 \u0446\u0435\u043b\u0438:      \n \u0417\u0430\u0441\u043b\u0435\u043f\u0435\u043d\u0430 \u0444\u0430\u0437\u0430: 1 1.\t\u0414\u0430 \u0441\u0435 \u043e\u0446\u0435\u043d\u044f\u0442 \u0435\u0444\u0435\u043a\u0442\u0438\u0442\u0435 \u043d\u0430 ATL1102 \u0447\u0440\u0435\u0437 \u043d\u044f\u043a\u043e\u043b\u043a\u043e \u0434\u043e\u043f\u044a\u043b\u043d\u0438\u0442\u0435\u043b\u043d\u0438 \u043a\u0440\u0430\u0439\u043d\u0438 \u0442\u043e\u0447\u043a\u0438 \u0437\u0430 \u043e\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f \u0438 \u0438\u0437\u0434\u0440\u044a\u0436\u043b\u0438\u0432\u043e\u0441\u0442, \u0440\u0435\u0441\u043f\u0438\u0440\u0430\u0442\u043e\u0440\u043d\u0430\u0442\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f \u0438 \u043a\u0430\u0447\u0435\u0441\u0442\u0432\u043e\u0442\u043e \u043d\u0430 \u0436\u0438\u0432\u043e\u0442", "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": "\u0414\u0430 \u0441\u0435 \u043e\u0446\u0435\u043d\u0438 \u0431\u0435\u0437\u043e\u043f\u0430\u0441\u043d\u043e\u0441\u0442\u0442\u0430 \u0438 \u043f\u043e\u043d\u043e\u0441\u0438\u043c\u043e\u0441\u0442\u0442\u0430 \u043d\u0430 ATL1102, \u043f\u0440\u0438\u043b\u0430\u0433\u0430\u043d \u0432\u0435\u0434\u043d\u044a\u0436 \u0441\u0435\u0434\u043c\u0438\u0447\u043d\u043e, \u0447\u0440\u0435\u0437 \u043f\u043e\u0434\u043a\u043e\u0436\u043d\u0430 (SC) \u0438\u043d\u0436\u0435\u043a\u0446\u0438\u044f \u043f\u0440\u0438 \u043d\u0435\u0430\u043c\u0431\u0443\u043b\u0430\u0442\u043e\u0440\u043d\u0438 \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u0446\u0438 \u0441 \u041c\u0414\u0414, \u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u0441\u044a\u0431\u0438\u0442\u0438\u044f, \u0441\u0432\u044a\u0440\u0437\u0430\u043d\u0438 \u0441 \u041f\u043b\u0430\u043d\u0430 \u0437\u0430 \u043d\u0430\u0431\u043b\u044e\u0434\u0435\u043d\u0438\u0435 \u043d\u0430 \u0431\u0435\u0437\u043e\u043f\u0430\u0441\u043d\u043e\u0441\u0442\u0442\u0430 \u0438 \u041f\u0440\u0430\u0432\u0438\u043b\u0430\u0442\u0430 \u0437\u0430 \u0441\u043f\u0438\u0440\u0430\u043d\u0435", "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": "\u0414\u0430 \u0441\u0435 \u043e\u0446\u0435\u043d\u0438 \u0444\u0430\u0440\u043c\u0430\u043a\u043e\u043a\u0438\u043d\u0435\u0442\u0438\u0447\u043d\u0438\u044f\u0442 (\u0424\u041a) \u043f\u0440\u043e\u0444\u0438\u043b \u043d\u0430 ATL1102 \u043f\u0440\u0438 \u0440\u0430\u0437\u043b\u0438\u0447\u043d\u0438\u0442\u0435 \u0434\u043e\u0437\u043e\u0432\u0438 \u043d\u0438\u0432\u0430, \u043f\u0440\u0438\u043b\u0430\u0433\u0430\u043d \u0432\u0435\u0434\u043d\u044a\u0436 \u0441\u0435\u0434\u043c\u0438\u0447\u043d\u043e \u0447\u0440\u0435\u0437 \u043f\u043e\u0434\u043a\u043e\u0436\u043d\u0430 (SC) \u0438\u043d\u0436\u0435\u043a\u0446\u0438\u044f \u043f\u0440\u0438 \u043d\u0435\u0430\u043c\u0431\u0443\u043b\u0430\u0442\u043e\u0440\u043d\u0438 \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u0446\u0438 \u0441 \u041c\u0414\u0414", "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": "\u041e\u0442\u043a\u0440\u0438\u0442\u0430 \u0444\u0430\u0437\u0430 \u043d\u0430 \u043f\u0440\u043e\u0434\u044a\u043b\u0436\u0435\u043d\u0438\u0435 (OLE):\n1.\t\u0414\u0430 \u0441\u0435 \u043e\u0446\u0435\u043d\u0438 \u0435\u0444\u0435\u043a\u0442\u044a\u0442 \u043d\u0430 ATL1102 \u0432\u044a\u0440\u0445\u0443 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f \u043d\u0430 \u0433\u043e\u0440\u043d\u0438\u0442\u0435 \u043a\u0440\u0430\u0439\u043d\u0438\u0446\u0438 \u043f\u0440\u0438 \u043d\u0435\u0430\u043c\u0431\u0443\u043b\u0430\u0442\u043e\u0440\u043d\u0438 \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u0446\u0438 \u0441 \u041c\u0414\u0414 \u0441\u043f\u043e\u0440\u0435\u0434 \u043e\u0446\u0435\u043d\u043a\u0430\u0442\u0430 \u043d\u0430 \u043f\u0440\u043e\u043c\u044f\u043d\u0430\u0442\u0430 \u0432 \u0441\u043a\u043e\u0440\u0430 \u043e\u0442 \u041c\u043e\u0434\u0443\u043b\u0430 \u0437\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f \u043d\u0430 \u0433\u043e\u0440\u043d\u0438\u0442\u0435 \u043a\u0440\u0430\u0439\u043d\u0438\u0446\u0438 \u0437\u0430 \u041c\u0414\u0414 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": "\u0414\u0430 \u0441\u0435 \u043e\u0446\u0435\u043d\u044f\u0442 \u0435\u0444\u0435\u043a\u0442\u0438\u0442\u0435 \u043d\u0430 ATL1102 \u0447\u0440\u0435\u0437 \u043d\u044f\u043a\u043e\u043b\u043a\u043e \u0434\u043e\u043f\u044a\u043b\u043d\u0438\u0442\u0435\u043b\u043d\u0438 \u043a\u0440\u0430\u0439\u043d\u0438 \u0442\u043e\u0447\u043a\u0438 \u0437\u0430 \u043e\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f \u0438 \u0438\u0437\u0434\u0440\u044a\u0436\u043b\u0438\u0432\u043e\u0441\u0442, \u0440\u0435\u0441\u043f\u0438\u0440\u0430\u0442\u043e\u0440\u043d\u0430\u0442\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f \u0438 \u043a\u0430\u0447\u0435\u0441\u0442\u0432\u043e\u0442\u043e \u043d\u0430 \u0436\u0438\u0432\u043e\u0442", "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": "\u0415\u0434\u0438\u043d \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u043a \u0449\u0435 \u0431\u044a\u0434\u0435 \u0432\u043a\u043b\u044e\u0447\u0435\u043d \u0432 \u0442\u043e\u0432\u0430 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435 \u0441\u0430\u043c\u043e \u0430\u043a\u043e \u043e\u0442\u0433\u043e\u0432\u0430\u0440\u044f \u043d\u0430 \u0432\u0441\u0438\u0447\u043a\u0438 \u043e\u0442 \u0441\u043b\u0435\u0434\u0432\u0430\u0449\u0438\u0442\u0435 \u043a\u0440\u0438\u0442\u0435\u0440\u0438\u0438:\n1.\t\u0418\u043c\u0430 \u0440\u043e\u0434\u0438\u0442\u0435\u043b/\u043d\u0430\u0441\u0442\u043e\u0439\u043d\u0438\u043a, \u043a\u043e\u0439\u0442\u043e \u0435 \u0441\u043f\u043e\u0441\u043e\u0431\u0435\u043d \u0434\u0430 \u0440\u0430\u0437\u0431\u0435\u0440\u0435 \u0446\u0435\u043b\u0438\u0442\u0435 \u0438 \u0440\u0438\u0441\u043a\u043e\u0432\u0435\u0442\u0435 \u043d\u0430 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e \u0438 \u043c\u043e\u0436\u0435 \u0434\u0430 \u043f\u0440\u0435\u0434\u043e\u0441\u0442\u0430\u0432\u0438 \u0434\u043e\u0431\u0440\u043e\u0432\u043e\u043b\u043d\u043e \u043f\u0438\u0441\u043c\u0435\u043d\u043e \u0438\u043d\u0444\u043e\u0440\u043c\u0438\u0440\u0430\u043d\u043e \u0441\u044a\u0433\u043b\u0430\u0441\u0438\u0435 \u0437\u0430 \u0443\u0447\u0430\u0441\u0442\u0438\u0435\u0442\u043e \u043d\u0430 \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u043a\u0430 \u0432 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e, \u043a\u043e\u0435\u0442\u043e \u0449\u0435 \u0431\u044a\u0434\u0435 \u0434\u043e\u043a\u0443\u043c\u0435\u043d\u0442\u0438\u0440\u0430\u043d\u043e.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 457373, "number": 10, "principalInclusionCriteria": "Has adequate cardiac function defined as left ventricular ejection fraction (LVEF) \u226545% 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": "\u0418\u043c\u0430 \u0430\u0434\u0435\u043a\u0432\u0430\u0442\u043d\u0430 \u0441\u044a\u0440\u0434\u0435\u0447\u043d\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f, \u0434\u0435\u0444\u0438\u043d\u0438\u0440\u0430\u043d\u0430 \u043a\u0430\u0442\u043e \u043b\u0435\u0432\u043e\u043a\u0430\u043c\u0435\u0440\u043d\u0430 \u0444\u0440\u0430\u043a\u0446\u0438\u044f \u043d\u0430 \u0438\u0437\u0442\u043b\u0430\u0441\u043a\u0432\u0430\u043d\u0435 (left ventricular ejection fraction, LVEF) \u2265 45% \u043e\u0442 \u0435\u0445\u043e\u043a\u0430\u0440\u0434\u0438\u043e\u0433\u0440\u0430\u043c\u0430 \u0438 \u0430\u043a\u043e \u043f\u0440\u0438\u0435\u043c\u0430 \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u043e \u0437\u0430 \u0441\u044a\u0440\u0446\u0435\u0442\u043e, \u0442\u0440\u044f\u0431\u0432\u0430 \u0432 \u043c\u043e\u043c\u0435\u043d\u0442\u0430 \u0434\u0430 \u0435 \u043d\u0430 \u0441\u0442\u0430\u0431\u0438\u043b\u0435\u043d \u0440\u0435\u0436\u0438\u043c \u0438 \u0434\u043e\u0437\u0438 \u043d\u0430 \u0441\u044a\u0440\u0434\u0435\u0447\u043d\u0430 \u0442\u0435\u0440\u0430\u043f\u0438\u044f (\u043d\u0430\u0439-\u043c\u0430\u043b\u043a\u043e 3 \u043c\u0435\u0441\u0435\u0446\u0430 \u043f\u0440\u0435\u0434\u0438 \u0414\u0435\u043d 1 \u043e\u0442 \u0431\u0430\u0437\u043e\u0432\u043e\u0442\u043e \u043d\u0438\u0432\u043e), \u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u0438\u043d\u0445\u0438\u0431\u0438\u0442\u043e\u0440\u0438 \u043d\u0430 \u0430\u043d\u0433\u0438\u043e\u0442\u0435\u043d\u0437\u0438\u043d-\u043a\u043e\u043d\u0432\u0435\u0440\u0442\u0438\u0440\u0430\u0449\u0438\u044f \u0435\u043d\u0437\u0438\u043c (ACEi), \u0430\u043d\u0442\u0430\u0433\u043e\u043d\u0438\u0441\u0442\u0438 \u043d\u0430 \u0430\u043d\u0433\u0438\u043e\u0442\u0435\u043d\u0437\u0438\u043d 2 \u0440\u0435\u0446\u0435\u043f\u0442\u043e\u0440 (A2RA), \u0430\u043d\u0442\u0430\u0433\u043e\u043d\u0438\u0441\u0442\u0438 \u043d\u0430 \u0430\u043b\u0434\u043e\u0441\u0442\u0435\u0440\u043e\u043d \u0440\u0435\u0446\u0435\u043f\u0442\u043e\u0440 (ARA) \u0438\u043b\u0438 \u0431\u0435\u0442\u0430-\u0431\u043b\u043e\u043a\u0435\u0440\u0438 (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": "\u0423\u0447\u0430\u0441\u0442\u043d\u0438\u043a\u044a\u0442, \u043a\u043e\u0439\u0442\u043e \u0435 \u043f\u0440\u0435\u043c\u0438\u043d\u0430\u043b \u043f\u0443\u0431\u0435\u0440\u0442\u0435\u0442\u0430 \u0438 \u0435 \u0441\u0435\u043a\u0441\u0443\u0430\u043b\u043d\u043e \u0430\u043a\u0442\u0438\u0432\u0435\u043d, \u0442\u0440\u044f\u0431\u0432\u0430 \u0434\u0430 \u0441\u0435 \u0441\u044a\u0433\u043b\u0430\u0441\u0438 \u0434\u0430 \u0438\u0437\u043f\u043e\u043b\u0437\u0432\u0430 \u043e\u0434\u043e\u0431\u0440\u0435\u043d\u0438 \u043c\u0435\u0442\u043e\u0434\u0438 \u0437\u0430 \u043a\u043e\u043d\u0442\u0440\u0430\u0446\u0435\u043f\u0446\u0438\u044f (\u043f\u0440\u0435\u0437\u0435\u0440\u0432\u0430\u0442\u0438\u0432\u0438 \u0438\u043b\u0438 \u0432\u044a\u0437\u0434\u044a\u0440\u0436\u0430\u043d\u0438\u0435) \u0437\u0430 \u043f\u0435\u0440\u0438\u043e\u0434\u0430 \u043d\u0430 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e \u0438 \u0434\u043e 4 \u043c\u0435\u0441\u0435\u0446\u0430 \u0441\u043b\u0435\u0434 \u043f\u0440\u0438\u043b\u0430\u0433\u0430\u043d\u0435\u0442\u043e \u043d\u0430 \u043f\u043e\u0441\u043b\u0435\u0434\u043d\u0430\u0442\u0430 \u0434\u043e\u0437\u0430 \u043e\u0442 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u043e\u0442\u043e \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u043e. \u0421\u0435\u043a\u0441\u0443\u0430\u043b\u043d\u0430\u0442\u0430 \u043f\u0430\u0440\u0442\u043d\u044c\u043e\u0440\u043a\u0430 \u043e\u0442 \u0436\u0435\u043d\u0441\u043a\u0438 \u043f\u043e\u043b \u0441\u044a\u0449\u043e \u0442\u0440\u044f\u0431\u0432\u0430 \u0434\u0430 \u0441\u0435 \u0441\u044a\u0433\u043b\u0430\u0441\u0438 \u0434\u0430 \u0438\u0437\u043f\u043e\u043b\u0437\u0432\u0430 \u043c\u0435\u0434\u0438\u0446\u0438\u043d\u0441\u043a\u0438 \u043f\u0440\u0438\u0435\u043c\u043b\u0438\u0432\u0430 \u0444\u043e\u0440\u043c\u0430 \u043d\u0430 \u043a\u043e\u043d\u0442\u0440\u0430\u0446\u0435\u043f\u0446\u0438\u044f. \u0412\u0438\u0436\u0442\u0435 \u0418\u0437\u0438\u0441\u043a\u0432\u0430\u043d\u0438\u044f \u0437\u0430 \u043a\u043e\u043d\u0442\u0440\u043e\u043b \u043d\u0430 \u0440\u0430\u0436\u0434\u0430\u0435\u043c\u043e\u0441\u0442\u0442\u0430.", "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": "\u0423\u0447\u0430\u0441\u0442\u043d\u0438\u043a\u044a\u0442 \u0438 \u043d\u0435\u0433\u043e\u0432\u0438\u044f\u0442 \u0440\u043e\u0434\u0438\u0442\u0435\u043b/\u043d\u0430\u0441\u0442\u043e\u0439\u043d\u0438\u043a/\u0431\u043e\u043b\u043d\u043e\u0433\u043b\u0435\u0434\u0430\u0447 \u0436\u0435\u043b\u0430\u044f\u0442 \u0438 \u0441\u0430 \u0432 \u0441\u044a\u0441\u0442\u043e\u044f\u043d\u0438\u0435 \u0434\u0430 \u0441\u043f\u0430\u0437\u0432\u0430\u0442 \u043f\u043b\u0430\u043d\u0438\u0440\u0430\u043d\u0438\u0442\u0435 \u0432\u0438\u0437\u0438\u0442\u0438, \u043f\u0440\u0438\u043b\u043e\u0436\u0435\u043d\u0438\u0435\u0442\u043e \u043d\u0430 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u043e\u0442\u043e \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u043e \u0438 \u043f\u0440\u043e\u0446\u0435\u0434\u0443\u0440\u0438\u0442\u0435 \u043f\u043e \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e.", "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": "\u041a\u043e\u0433\u0430\u0442\u043e \u0442\u043e\u0432\u0430 \u0441\u0435 \u0438\u0437\u0438\u0441\u043a\u0432\u0430 \u043e\u0442 \u0437\u0430\u043a\u043e\u043d\u0430 \u0438\u043b\u0438 \u0430\u043a\u043e \u0418\u0437\u0441\u043b\u0435\u0434\u043e\u0432\u0430\u0442\u0435\u043b\u044f\u0442 \u043e\u043f\u0440\u0435\u0434\u0435\u043b\u0438, \u0447\u0435 \u043f\u043e\u0442\u0435\u043d\u0446\u0438\u0430\u043b\u043d\u0438\u044f\u0442 \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u043a \u0435 \u0434\u043e\u0441\u0442\u0430\u0442\u044a\u0447\u043d\u043e \u0437\u0440\u044f\u043b \u0438 \u0441\u043f\u043e\u0441\u043e\u0431\u0435\u043d \u0434\u0430 \u0440\u0430\u0437\u0431\u0435\u0440\u0435 \u0435\u0441\u0442\u0435\u0441\u0442\u0432\u043e\u0442\u043e \u0438 \u043f\u043e\u0441\u043b\u0435\u0434\u0441\u0442\u0432\u0438\u044f\u0442\u0430 \u043e\u0442 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e, \u0449\u0435 \u0431\u044a\u0434\u0435 \u043f\u043e\u043b\u0443\u0447\u0435\u043d\u043e \u0441\u044a\u0433\u043b\u0430\u0441\u0438\u0435 \u043e\u0442 \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u043a\u0430. \u0412 \u043f\u0440\u043e\u0442\u0438\u0432\u0435\u043d \u0441\u043b\u0443\u0447\u0430\u0439 \u0441\u044a\u0433\u043b\u0430\u0441\u0438\u0435\u0442\u043e \u0449\u0435 \u0431\u044a\u0434\u0435 \u0434\u043e\u043a\u0443\u043c\u0435\u043d\u0442\u0438\u0440\u0430\u043d\u043e.", "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": "\u0418\u043c\u0430 \u043a\u043b\u0438\u043d\u0438\u0447\u043d\u0430 \u0434\u0438\u0430\u0433\u043d\u043e\u0437\u0430 \u0437\u0430 \u041c\u0414\u0414, \u043f\u043e\u0442\u0432\u044a\u0440\u0434\u0435\u043d\u0430 \u0447\u0440\u0435\u0437 \u0432\u0430\u043b\u0438\u0434\u0438\u0440\u0430\u043d\u043e \u0433\u0435\u043d\u0435\u0442\u0438\u0447\u043d\u043e \u0438\u0437\u0441\u043b\u0435\u0434\u0432\u0430\u043d\u0435 (\u0442.\u0435. \u0434\u043e\u043a\u0443\u043c\u0435\u043d\u0442\u0438\u0440\u0430\u043d\u0430 \u0434\u0435\u043b\u0435\u0446\u0438\u044f, \u0434\u0443\u0431\u043b\u0438\u0440\u0430\u043d\u0435 \u0438\u043b\u0438 \u0442\u043e\u0447\u043a\u043e\u0432\u0430 \u043c\u0443\u0442\u0430\u0446\u0438\u044f \u0432 \u0433\u0435\u043d\u0430 \u043d\u0430 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u043d\u0430).", "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": "\u0421\u0447\u0438\u0442\u0430 \u0441\u0435 \u0437\u0430 \u043d\u0435\u0430\u043c\u0431\u0443\u043b\u0430\u0442\u043e\u0440\u0435\u043d, \u043e\u043f\u0440\u0435\u0434\u0435\u043b\u0435\u043d \u043a\u0430\u0442\u043e \u043d\u0435\u0441\u043f\u043e\u0441\u043e\u0431\u0435\u043d \u0434\u0430 \u0445\u043e\u0434\u0438 10 \u043c\u0435\u0442\u0440\u0430 \u0431\u0435\u0437 \u0441\u044a\u0434\u0435\u0439\u0441\u0442\u0432\u0438\u0435 \u0438\u043b\u0438 \u043f\u043e\u043c\u043e\u0449 \u043f\u0440\u0438 \u0421\u043a\u0440\u0438\u043d\u0438\u043d\u0433.", "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": "\u0423\u0447\u0430\u0441\u0442\u043d\u0438\u043a \u043e\u0442 \u043c\u044a\u0436\u043a\u0438 \u043f\u043e\u043b \u043d\u0430 \u0432\u044a\u0437\u0440\u0430\u0441\u0442 \u043e\u0442 10 \u0434\u043e \u043f\u043e\u0434 18 \u0433\u043e\u0434\u0438\u043d\u0438 \u043a\u044a\u043c \u043c\u043e\u043c\u0435\u043d\u0442\u0430 \u043d\u0430 \u0421\u043a\u0440\u0438\u043d\u0438\u043d\u0433 \u0438 \u0438\u043d\u0444\u043e\u0440\u043c\u0438\u0440\u0430\u043d\u043e \u0441\u044a\u0433\u043b\u0430\u0441\u0438\u0435 \u0437\u0430 \u0443\u0447\u0430\u0441\u0442\u0438\u0435 \u0432 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e.", "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": "\u0422\u0435\u043b\u0435\u0441\u043d\u043e \u0442\u0435\u0433\u043b\u043e \u043d\u0430\u0439-\u043c\u0430\u043b\u043a\u043e 25 \u043a\u0433 \u043f\u0440\u0438 \u0421\u043a\u0440\u0438\u043d\u0438\u043d\u0433.", "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": "\u0410\u043a\u043e \u043b\u0435\u0447\u0435\u043d\u0438\u0435\u0442\u043e \u0441 \u043a\u043e\u0440\u0442\u0438\u043a\u043e\u0441\u0442\u0435\u0440\u043e\u0438\u0434\u0438 \u0435 \u0437\u0430\u043f\u043e\u0447\u043d\u0430\u0442\u043e \u043d\u0430\u0439-\u043c\u0430\u043b\u043a\u043e \u0448\u0435\u0441\u0442 \u043c\u0435\u0441\u0435\u0446\u0430 \u043f\u0440\u0435\u0434\u0438 \u0431\u0430\u0437\u043e\u0432\u0430\u0442\u0430 \u0432\u0438\u0437\u0438\u0442\u0430 \u0438 \u0441\u0442\u0430\u0431\u0438\u043b\u043d\u0430 \u0434\u043d\u0435\u0432\u043d\u0430 \u0434\u043e\u0437\u0430 \u0437\u0430 \u043f\u043e\u043d\u0435 3 \u043c\u0435\u0441\u0435\u0446\u0430 \u043f\u0440\u0435\u0434\u0438 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u0438 \u0438 \u043a\u044a\u043c \u043c\u043e\u043c\u0435\u043d\u0442\u0430 \u043d\u044f\u043c\u0430 \u043a\u043b\u0438\u043d\u0438\u0447\u043d\u043e \u043d\u0430\u043c\u0435\u0440\u0435\u043d\u0438\u0435 \u0437\u0430 \u043f\u0440\u043e\u043c\u044f\u043d\u0430 \u043d\u0430 \u0434\u043e\u0437\u0430\u0442\u0430 \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u043f\u0435\u0440\u0438\u043e\u0434\u0430 \u043d\u0430 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e, \u0441 \u0438\u0437\u043a\u043b\u044e\u0447\u0435\u043d\u0438\u0435 \u043d\u0430 \u0430\u0434\u0430\u043f\u0442\u0438\u0440\u0430\u043d\u0435 \u0441\u043f\u043e\u0440\u0435\u0434 \u0442\u0435\u043b\u0435\u0441\u043d\u043e\u0442\u043e \u0442\u0435\u0433\u043b\u043e.", "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 \u22652.", "principalInclusionCriteriaTranslations": [{"id": 5179060, "uuid": "8a2aa81e-50c3-4ed1-bc6d-2b1a3221a359", "attributeTranslation": "\u041f\u0440\u0438 \u0432\u043a\u043b\u044e\u0447\u0432\u0430\u043d\u0435 \u0432 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e (\u0421\u043a\u0440\u0438\u043d\u0438\u043d\u0433 \u0438 \u0411\u0430\u0437\u043e\u0432\u043e \u043d\u0438\u0432\u043e), \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u043a\u044a\u0442 \u0438\u043c\u0430 \u0441\u043a\u043e\u0440 \u2265 2 \u0441\u043f\u043e\u0440\u0435\u0434 \u041c\u043e\u0434\u0443\u043b\u0430 \u0437\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f \u043d\u0430 \u0433\u043e\u0440\u043d\u0438\u0442\u0435 \u043a\u0440\u0430\u0439\u043d\u0438\u0446\u0438 \u0437\u0430 \u041c\u0414\u0414 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 \u226550%. 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": "\u0421\u043f\u043e\u0441\u043e\u0431\u0435\u043d \u0434\u0430 \u0438\u0437\u0432\u044a\u0440\u0448\u0438 \u0441\u043f\u0438\u0440\u043e\u043c\u0435\u0442\u0440\u0438\u044f \u0438 \u0438\u043c\u0430 \u0434\u043e\u0441\u0442\u0430\u0442\u044a\u0447\u043d\u0430 \u0420\u0435\u0441\u043f\u0438\u0440\u0430\u0442\u043e\u0440\u043d\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f, \u0434\u0435\u0444\u0438\u043d\u0438\u0440\u0430\u043d\u0430 \u043a\u0430\u0442\u043e \u0432\u044a\u0437\u043f\u0440\u043e\u0438\u0437\u0432\u043e\u0434\u0438\u043c \u043f\u0440\u043e\u0446\u0435\u043d\u0442 \u043e\u0447\u0430\u043a\u0432\u0430\u043d \u0424\u0412\u041a \u2265 50%. \u0421\u043f\u043e\u0441\u043e\u0431\u043d\u043e\u0441\u0442 \u0437\u0430 \u043e\u0441\u0438\u0433\u0443\u0440\u044f\u0432\u0430\u043d\u0435 \u043d\u0430 \u043d\u0430\u0434\u0435\u0436\u0434\u0435\u043d \u0438 \u0432\u044a\u0437\u043f\u0440\u043e\u0438\u0437\u0432\u043e\u0434\u0438\u043c \u043f\u043e\u0432\u0442\u043e\u0440\u0435\u043d \u0424\u0412\u041a, \u043a\u0430\u0442\u043e \u043d\u0430\u0439-\u0434\u043e\u0431\u0440\u0438\u044f\u0442 \u043e\u0442 \u0442\u0440\u0438\u0442\u0435 \u043e\u043f\u0438\u0442\u0430, \u043e\u0446\u0435\u043d\u0435\u043d\u0438 \u043d\u0430 \u0411\u0430\u0437\u043e\u0432\u043e \u043d\u0438\u0432\u043e, \u0435 \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 20% \u043e\u0442 \u043d\u0430\u0439-\u0434\u043e\u0431\u0440\u0438\u044f \u043e\u0442 \u0442\u0440\u0438\u0442\u0435 \u043e\u043f\u0438\u0442\u0430, \u043e\u0446\u0435\u043d\u0435\u043d\u0438 \u043f\u0440\u0438 \u0421\u043a\u0440\u0438\u043d\u0438\u043d\u0433.", "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": "\u041a\u0440\u0438\u0442\u0435\u0440\u0438\u0438 \u0437\u0430 \u0438\u0437\u043a\u043b\u044e\u0447\u0432\u0430\u043d\u0435\n\u0412\u0441\u0435\u043a\u0438 \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u043a, \u043a\u043e\u0439\u0442\u043e \u043e\u0442\u0433\u043e\u0432\u0430\u0440\u044f \u043d\u0430 \u043d\u044f\u043a\u043e\u0439 \u043e\u0442 \u0441\u043b\u0435\u0434\u043d\u0438\u0442\u0435 \u043a\u0440\u0438\u0442\u0435\u0440\u0438\u0438, \u0449\u0435 \u0431\u044a\u0434\u0435 \u0438\u0437\u043a\u043b\u044e\u0447\u0435\u043d \u043e\u0442 \u0443\u0447\u0430\u0441\u0442\u0438\u0435 \u0432 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e:\n1.\t\u0423\u0447\u0430\u0441\u0442\u0438\u0435 \u0432 \u0434\u0440\u0443\u0433\u043e \u043a\u043b\u0438\u043d\u0438\u0447\u043d\u043e \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435 (\u043d\u0435\u0438\u043d\u0442\u0435\u0440\u0432\u0435\u043d\u0446\u0438\u043e\u043d\u043d\u043e) \u0438\u043b\u0438 \u043f\u0440\u0438\u043b\u043e\u0436\u0435\u043d\u0438\u0435 \u043d\u0430 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u043e \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u043e \u0438\u043b\u0438 \u0435\u043a\u0441\u043f\u0435\u0440\u0438\u043c\u0435\u043d\u0442\u0430\u043b\u043d\u043e \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u043e \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 12 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u0438\u043b\u0438 5 \u043f\u043e\u043b\u0443\u0436\u0438\u0432\u043e\u0442\u0430 (\u043a\u043e\u0435\u0442\u043e \u0435 \u043f\u043e-\u0434\u044a\u043b\u0433\u043e) \u043f\u0440\u0435\u0434\u0438 \u0414\u0435\u043d 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": "\u0418\u0437\u0432\u0435\u0441\u0442\u043d\u0430 \u0430\u043d\u0430\u043c\u043d\u0435\u0437\u0430 \u0437\u0430 \u0438\u043b\u0438 \u043f\u043e\u043b\u043e\u0436\u0438\u0442\u0435\u043b\u043d\u0438 \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442\u0438 \u043e\u0442 \u0438\u0437\u0441\u043b\u0435\u0434\u0432\u0430\u043d\u0435 \u0437\u0430 \u043f\u043e\u0432\u044a\u0440\u0445\u043d\u043e\u0441\u0442\u0435\u043d \u0430\u043d\u0442\u0438\u0433\u0435\u043d (HBsAg) \u043d\u0430 \u0445\u0435\u043f\u0430\u0442\u0438\u0442 B, \u0430\u043d\u0442\u0438\u0442\u0435\u043b\u0430 (HCVAb) \u043d\u0430 \u0432\u0438\u0440\u0443\u0441\u0430 \u043d\u0430 \u0445\u0435\u043f\u0430\u0442\u0438\u0442 C \u0438\u043b\u0438 \u0432\u0438\u0440\u0443\u0441\u0430 \u043d\u0430 \u0447\u043e\u0432\u0435\u0448\u043a\u0438\u044f \u0438\u043c\u0443\u043d\u043e\u0434\u0435\u0444\u0438\u0446\u0438\u0442 (HIV) \u043f\u0440\u0438 \u0421\u043a\u0440\u0438\u043d\u0438\u043d\u0433.", "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": "\u0414\u043e\u043a\u0430\u0437\u0430\u0442\u0435\u043b\u0441\u0442\u0432\u043e \u0437\u0430 \u0431\u044a\u0431\u0440\u0435\u0447\u043d\u043e \u0443\u0432\u0440\u0435\u0436\u0434\u0430\u043d\u0435 \u0438/\u0438\u043b\u0438 \u0446\u0438\u0441\u0442\u0430\u0442\u0438\u043d 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": "\u041f\u043e\u043b\u0443\u0447\u0435\u043d\u0430 \u0436\u0438\u0432\u0430 \u0432\u0430\u043a\u0441\u0438\u043d\u0430 (\u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u0438\u043d\u0442\u0440\u0430\u043d\u0430\u0437\u0430\u043b\u043d\u0430 \u043f\u0440\u043e\u0442\u0438\u0432\u043e\u0433\u0440\u0438\u043f\u043d\u0430 \u0432\u0430\u043a\u0441\u0438\u043d\u0430) \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 4 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0414\u0435\u043d 1 \u0438\u043b\u0438 \u043f\u043b\u0430\u043d\u0438\u0440\u0430\u043d\u0430 \u0436\u0438\u0432\u0430 \u0432\u0430\u043a\u0441\u0438\u043d\u0430 \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u043f\u0435\u0440\u0438\u043e\u0434\u0430 \u043d\u0430 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e.", "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": "\u041f\u043b\u0430\u043d\u0438\u0440\u0430\u043d\u0430 \u0438\u043b\u0438 \u043e\u0447\u0430\u043a\u0432\u0430\u043d\u0430 \u043e\u043f\u0435\u0440\u0430\u0446\u0438\u044f \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u043f\u0435\u0440\u0438\u043e\u0434\u0430 \u043d\u0430 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e (\u043f\u043e \u043f\u0440\u0435\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u0418\u0437\u0441\u043b\u0435\u0434\u043e\u0432\u0430\u0442\u0435\u043b\u044f).", "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": "\u0410\u0441\u0442\u043c\u0430 (\u0430\u043a\u043e \u0441\u0435 \u043d\u0430\u043b\u0430\u0433\u0430 \u0440\u0435\u0434\u043e\u0432\u043d\u043e \u043b\u0435\u0447\u0435\u043d\u0438\u0435), \u0431\u0440\u043e\u043d\u0445\u0438\u0442/\u0445\u0440\u043e\u043d\u0438\u0447\u043d\u0430 \u043e\u0431\u0441\u0442\u0440\u0443\u043a\u0442\u0438\u0432\u043d\u0430 \u0431\u0435\u043b\u043e\u0434\u0440\u043e\u0431\u043d\u0430 \u0431\u043e\u043b\u0435\u0441\u0442 (\u0425\u041e\u0411\u0411), \u0431\u0440\u043e\u043d\u0445\u0438\u0435\u043a\u0442\u0430\u0437\u0438\u0438, \u0435\u043c\u0444\u0438\u0437\u0435\u043c, \u043f\u043d\u0435\u0432\u043c\u043e\u043d\u0438\u044f \u0438\u043b\u0438 \u043d\u0430\u043b\u0438\u0447\u0438\u0435 \u043d\u0430 \u043a\u0430\u043a\u0432\u043e\u0442\u043e \u0438 \u0434\u0430 \u0431\u0438\u043b\u043e \u0440\u0435\u0441\u043f\u0438\u0440\u0430\u0442\u043e\u0440\u043d\u043e \u0437\u0430\u0431\u043e\u043b\u044f\u0432\u0430\u043d\u0435, \u0440\u0430\u0437\u043b\u0438\u0447\u043d\u043e \u043e\u0442 \u041c\u0414\u0414, \u043a\u043e\u0435\u0442\u043e \u0437\u0430\u0441\u044f\u0433\u0430 \u0412\u0415\u0414 \u0438 \u0424\u0412\u041a \u0438\u043b\u0438 \u0434\u0440\u0443\u0433\u0438 \u0440\u0435\u0441\u043f\u0438\u0440\u0430\u0442\u043e\u0440\u043d\u0438 \u0438\u0437\u043c\u0435\u0440\u0432\u0430\u043d\u0438\u044f.", "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": "\u0418\u0437\u0438\u0441\u043a\u0432\u0430 \u043c\u0435\u0445\u0430\u043d\u0438\u0447\u043d\u0430 \u0438\u043b\u0438 \u043d\u0435\u0438\u043d\u0432\u0430\u0437\u0438\u0432\u043d\u0430 \u0432\u0435\u043d\u0442\u0438\u043b\u0430\u0446\u0438\u044f (non-invasive ventilation, NIV) \u043f\u0440\u0435\u0437 \u0434\u0435\u043d\u044f (NIV \u043f\u0440\u0435\u0437 \u043d\u043e\u0449\u0442\u0430 \u0435 \u0440\u0430\u0437\u0440\u0435\u0448\u0435\u043d\u0430).", "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": "\u041d\u0435 \u043c\u043e\u0436\u0435 \u0434\u0430 \u0437\u0430\u0442\u0432\u043e\u0440\u0438 \u043f\u043b\u044a\u0442\u043d\u043e \u0441 \u0443\u0441\u0442\u0430\u0442\u0430, \u043a\u043e\u0435\u0442\u043e \u0434\u0430 \u043f\u043e\u0437\u0432\u043e\u043b\u0438 \u043f\u0440\u0435\u0446\u0438\u0437\u043d\u043e \u0438\u0437\u043c\u0435\u0440\u0432\u0430\u043d\u0435 \u043d\u0430 \u0434\u0438\u0445\u0430\u0442\u0435\u043b\u043d\u0438\u044f \u043f\u043e\u0442\u043e\u043a \u0438 \u043d\u0430\u043b\u044f\u0433\u0430\u043d\u0435 \u0432 \u0443\u0441\u0442\u0430\u0442\u0430.", "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": "\u0425\u0440\u043e\u043d\u0438\u0447\u043d\u043e \u0438\u0437\u043f\u043e\u043b\u0437\u0432\u0430\u043d\u0435 (\u0435\u0436\u0435\u0434\u043d\u0435\u0432\u0435\u043d \u043f\u0440\u0438\u0435\u043c > 14 \u0434\u043d\u0438) \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 \u0435\u0434\u0438\u043d \u043c\u0435\u0441\u0435\u0446 \u043e\u0442 \u0414\u0435\u043d 1, \u043d\u0430 \u0431\u0435\u0442\u0430-2 \u0430\u0433\u043e\u043d\u0438\u0441\u0442\u0438 \u0438\u043b\u0438 \u0438\u0437\u043f\u043e\u043b\u0437\u0432\u0430\u043d\u0435 \u043d\u0430 \u0434\u0440\u0443\u0433\u043e \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u043e \u0441 \u0431\u0440\u043e\u043d\u0445\u043e\u0434\u0438\u043b\u0430\u0442\u0438\u0440\u0430\u0449\u043e \u0434\u0435\u0439\u0441\u0442\u0432\u0438\u0435 (\u043d\u0430\u043f\u0440. \u0438\u043d\u0445\u0430\u043b\u0430\u0442\u043e\u0440\u043d\u0438 \u0441\u0442\u0435\u0440\u043e\u0438\u0434\u0438, \u0441\u0438\u043c\u043f\u0430\u0442\u043e\u043c\u0438\u043c\u0435\u0442\u0438\u0446\u0438, \u0430\u043d\u0442\u0438\u0445\u043e\u043b\u0438\u043d\u0435\u0440\u0433\u0438\u0446\u0438).", "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, TNF\u0251 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": "\u0418\u0437\u043f\u043e\u043b\u0437\u0432\u0430\u043d\u0435 \u043d\u0430 \u043a\u0430\u0440\u043d\u0438\u0442\u0438\u043d, \u043a\u0440\u0435\u0430\u0442\u0438\u043d, \u0433\u043b\u0443\u0442\u0430\u043c\u0438\u043d, \u043e\u043a\u0441\u0430\u0442\u043e\u043c\u0438\u0434, \u0438\u0434\u0435\u0431\u0435\u043d\u043e\u043d \u0438\u043b\u0438 \u0434\u0440\u0443\u0433\u0438 \u0444\u043e\u0440\u043c\u0438 \u043d\u0430 \u043a\u043e\u0435\u043d\u0437\u0438\u043c Q10 \u0438\u043b\u0438 \u0432\u0438\u0442\u0430\u043c\u0438\u043d E, \u0432\u0441\u044f\u043a\u0430\u043a\u0432\u0438 \u0434\u0440\u0443\u0433\u0438 \u0445\u0440\u0430\u043d\u0438\u0442\u0435\u043b\u043d\u0438 \u0438\u043b\u0438 \u0430\u043d\u0442\u0438\u043e\u043a\u0441\u0438\u0434\u0430\u043d\u0442\u043d\u0438 \u0434\u043e\u0431\u0430\u0432\u043a\u0438, \u0431\u0438\u043b\u043a\u043e\u0432\u0438 \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u0430, \u0430\u043d\u0430\u0431\u043e\u043b\u043d\u0438 \u0441\u0442\u0435\u0440\u043e\u0438\u0434\u0438, \u0440\u0430\u0437\u043b\u0438\u0447\u043d\u0438 \u043e\u0442 \u0441\u0442\u0430\u043d\u0434\u0430\u0440\u0442\u043d\u0438 \u043a\u043e\u0440\u0442\u0438\u043a\u043e\u0441\u0442\u0435\u0440\u043e\u0438\u0434\u0438, \u0438\u043b\u0438 \u0434\u043e\u0431\u0430\u0432\u043a\u0430 \u043d\u0430 \u0442\u0435\u0441\u0442\u043e\u0441\u0442\u0435\u0440\u043e\u043d \u0432 \u043f\u0443\u0431\u0435\u0440\u0442\u0435\u0442\u0430 \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 4 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043e\u0442 \u0414\u0435\u043d 1. \u0417\u0410\u0411\u0415\u041b\u0415\u0416\u041a\u0410: \u043f\u043e\u0437\u0432\u043e\u043b\u0435\u043d \u0435 \u0435\u0436\u0435\u0434\u043d\u0435\u0432\u0435\u043d \u043f\u0440\u0438\u0435\u043c \u043d\u0430 \u043c\u0443\u043b\u0442\u0438\u0432\u0438\u0442\u0430\u043c\u0438\u043d\u0438, \u0432\u0438\u0442\u0430\u043c\u0438\u043d D \u0438\u043b\u0438 \u043a\u0430\u043b\u0446\u0438\u0435\u0432\u0438 \u0434\u043e\u0431\u0430\u0432\u043a\u0438.\n\u0418\u0437\u043f\u043e\u043b\u0437\u0432\u0430\u043d\u0438 \u0430\u0442\u0430\u043b\u0443\u0440\u0435\u043d, \u0435\u0442\u0435\u043f\u043b\u0438\u0440\u0441\u0435\u043d, \u043a\u0430\u0437\u0438\u043c\u0435\u0440\u0441\u0435\u043d, \u0433\u043e\u043b\u043e\u0434\u0438\u0440\u0441\u0435\u043d (\u0438\u043b\u0438 \u0434\u0440\u0443\u0433 \u0435\u043a\u0437\u043e\u043d\n\u043f\u0440\u043e\u043f\u0443\u0441\u043a\u0430\u043d\u0435 \u043d\u0430 \u0430\u043d\u0442\u0438\u0441\u0435\u043d\u0441 \u043e\u043b\u0438\u0433\u043e\u043d\u0443\u043a\u043b\u0435\u043e\u0442\u0438\u0434\u043d\u0438 \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u0430) \u0438 \u0432\u0430\u043c\u043e\u0440\u043e\u043b\u043e\u043d \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 6\n\u043c\u0435\u0441\u0435\u0446\u0430 \u043f\u0440\u0435\u0434\u0438 \u0414\u0435\u043d 1.\n\u0418\u0437\u043f\u043e\u043b\u0437\u0432\u0430\u043d\u043e \u0441\u0438\u0441\u0442\u0435\u043c\u043d\u043e \u043b\u0435\u0447\u0435\u043d\u0438\u0435 \u0441 \u0438\u043c\u0443\u043d\u043e\u0441\u0443\u043f\u0440\u0435\u0441\u043e\u0440\u0438 (\u043d\u0430\u043f\u0440. \u043c\u0438\u0442\u043e\u043a\u0441\u0430\u043d\u0442\u0440\u043e\u043d,\n\u0430\u0437\u0430\u0442\u0438\u043e\u043f\u0440\u0438\u043d, \u043c\u0435\u0442\u043e\u0442\u0440\u0435\u043a\u0441\u0430\u0442, \u0446\u0438\u043a\u043b\u043e\u0444\u043e\u0441\u0444\u0430\u043c\u0438\u0434, \u043c\u0438\u043a\u043e\u0444\u0435\u043d\u043e\u043b\u0430\u0442, TNF\u0251\n\u0438\u043d\u0445\u0438\u0431\u0438\u0442\u043e\u0440\u0438) \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 3 \u043c\u0435\u0441\u0435\u0446\u0430 \u043f\u0440\u0435\u0434\u0438 \u0414\u0435\u043d 1.\n\u0418\u0437\u043f\u043e\u043b\u0437\u0432\u0430\u043d \u0438\u043d\u0442\u0440\u0430\u0432\u0435\u043d\u043e\u0437\u0435\u043d \u0438\u043c\u0443\u043d\u043e\u0433\u043b\u043e\u0431\u0443\u043b\u0438\u043d (IVIg) \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 6 \u043c\u0435\u0441\u0435\u0446\u0430 \u043f\u0440\u0435\u0434\u0438 \u0414\u0435\u043d 1.\n\u0412\u0438\u0436\u0442\u0435 \u0440\u0430\u0437\u0434\u0435\u043b 7.6.2 \u0417\u0430\u0431\u0440\u0430\u043d\u0435\u043d\u0438 \u043c\u0435\u0434\u0438\u043a\u0430\u043c\u0435\u043d\u0442\u0438.", "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": "\u041e\u0441\u0432\u0435\u043d \u0441\u044a\u0441\u0442\u043e\u044f\u043d\u0438\u0435\u0442\u043e, \u043a\u043e\u0435\u0442\u043e \u0435 \u043f\u0440\u0435\u0434\u043c\u0435\u0442 \u043d\u0430 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e, \u0438\u043c\u0430 \u0430\u043d\u0430\u043c\u043d\u0435\u0437\u0430 \u0437\u0430 \u0438\u043b\u0438 \u0442\u0435\u043a\u0443\u0449\u043e \u0430\u043a\u0442\u0438\u0432\u043d\u043e \u043a\u043b\u0438\u043d\u0438\u0447\u043d\u043e \u0441\u044a\u0441\u0442\u043e\u044f\u043d\u0438\u0435, \u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u0430\u043b\u0435\u0440\u0433\u0438\u0447\u043d\u043e, \u043a\u043e\u0436\u043d\u043e, \u0441\u044a\u0440\u0434\u0435\u0447\u043d\u043e-\u0441\u044a\u0434\u043e\u0432\u043e, \u043f\u0441\u0438\u0445\u0438\u0430\u0442\u0440\u0438\u0447\u043d\u043e \u0437\u0430\u0431\u043e\u043b\u044f\u0432\u0430\u043d\u0435, \u0437\u043b\u043e\u0443\u043f\u043e\u0442\u0440\u0435\u0431\u0430 \u0441 \u043d\u0430\u0440\u043a\u043e\u0442\u0438\u0446\u0438 \u0438\u043b\u0438 \u0430\u043b\u043a\u043e\u0445\u043e\u043b, \u0442\u0435\u0436\u043a\u0438 \u043f\u043e\u0432\u0435\u0434\u0435\u043d\u0447\u0435\u0441\u043a\u0438 \u0438\u043b\u0438 \u043a\u043e\u0433\u043d\u0438\u0442\u0438\u0432\u043d\u0438 \u0434\u0435\u0444\u0438\u0446\u0438\u0442\u0438 \u0438\u043b\u0438 \u043b\u0430\u0431\u043e\u0440\u0430\u0442\u043e\u0440\u043d\u0430 \u043d\u0430\u0445\u043e\u0434\u043a\u0430, \u043a\u043e\u0438\u0442\u043e \u0441\u043f\u043e\u0440\u0435\u0434 \u043c\u043d\u0435\u043d\u0438\u0435\u0442\u043e \u043d\u0430 \u0418\u0437\u0441\u043b\u0435\u0434\u043e\u0432\u0430\u0442\u0435\u043b\u044f \u043d\u0435 \u043f\u043e\u0437\u0432\u043e\u043b\u044f\u0432\u0430\u0442 \u0443\u0447\u0430\u0441\u0442\u0438\u0435 \u0432 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e \u0438\u043b\u0438 \u0431\u0438\u0445\u0430 \u043c\u043e\u0433\u043b\u0438 \u0434\u0430 \u043f\u043e\u0432\u043b\u0438\u044f\u044f\u0442 \u0432\u044a\u0440\u0445\u0443 \u0446\u0435\u043b\u0438\u0442\u0435/\u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442\u0438\u0442\u0435 \u043e\u0442 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e.", "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": "\u0415\u043a\u0441\u043f\u043e\u0437\u0438\u0446\u0438\u044f \u043d\u0430 \u043f\u043e\u0432\u0435\u0447\u0435 \u043e\u0442 3 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0438 \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u0430 \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 12 \u043c\u0435\u0441\u0435\u0446\u0430 \u043f\u0440\u0435\u0434\u0438 \u0414\u0435\u043d 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": "\u0418\u043c\u0430 \u043f\u043e\u0432\u0438\u0448\u0435\u043d \u0440\u0438\u0441\u043a \u043e\u0442 \u043e\u043f\u043e\u0440\u0442\u044e\u043d\u0438\u0441\u0442\u0438\u0447\u043d\u0438 \u0438\u043d\u0444\u0435\u043a\u0446\u0438\u0438 \u0438\u043b\u0438 \u0441\u0438\u0441\u0442\u0435\u043c\u043d\u0438 \u043c\u0435\u0434\u0438\u0446\u0438\u043d\u0441\u043a\u0438 \u0441\u044a\u0441\u0442\u043e\u044f\u043d\u0438\u044f, \u0432\u043e\u0434\u0435\u0449\u0438 \u0434\u043e \u0437\u043d\u0430\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u043d\u0430\u0440\u0443\u0448\u0435\u043d\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f \u043d\u0430 \u0438\u043c\u0443\u043d\u043d\u0430\u0442\u0430 \u0441\u0438\u0441\u0442\u0435\u043c\u0430 (\u043d\u0430\u043f\u0440. HIV, \u043e\u0440\u0433\u0430\u043d\u043d\u0430 \u0442\u0440\u0430\u043d\u0441\u043f\u043b\u0430\u043d\u0442\u0430\u0446\u0438\u044f, \u0430\u043a\u0442\u0438\u0432\u043d\u043e \u0437\u043b\u043e\u043a\u0430\u0447\u0435\u0441\u0442\u0432\u0435\u043d\u043e \u0437\u0430\u0431\u043e\u043b\u044f\u0432\u0430\u043d\u0435).", "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": "\u0421\u043b\u0443\u0436\u0438\u0442\u0435\u043b \u043d\u0430 \u0421\u043f\u043e\u043d\u0441\u043e\u0440\u0430 \u0438\u043b\u0438 \u043f\u0435\u0440\u0441\u043e\u043d\u0430\u043b\u0430 \u043d\u0430 \u0438\u0437\u0441\u043b\u0435\u0434\u043e\u0432\u0430\u0442\u0435\u043b\u0441\u043a\u0438\u044f \u0446\u0435\u043d\u0442\u044a\u0440, \u0441\u0432\u044a\u0440\u0437\u0430\u043d \u0434\u0438\u0440\u0435\u043a\u0442\u043d\u043e \u0441 \u0442\u043e\u0432\u0430 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435, \u0438\u043b\u0438 \u043d\u0435\u0433\u043e\u0432\u0438/\u043d\u0435\u0439\u043d\u0438 \u0431\u043b\u0438\u0437\u043a\u0438 \u0447\u043b\u0435\u043d\u043e\u0432\u0435 \u043d\u0430 \u0441\u0435\u043c\u0435\u0439\u0441\u0442\u0432\u043e\u0442\u043e, \u043a\u0430\u0442\u043e \u0441\u044a\u043f\u0440\u0443\u0433/\u0430, \u0440\u043e\u0434\u0438\u0442\u0435\u043b, \u0431\u0440\u0430\u0442, \u0441\u0435\u0441\u0442\u0440\u0430 \u0438\u043b\u0438 \u0434\u0435\u0442\u0435, \u043d\u0435\u0437\u0430\u0432\u0438\u0441\u0438\u043c\u043e \u0434\u0430\u043b\u0438 \u0431\u0438\u043e\u043b\u043e\u0433\u0438\u0447\u043d\u043e \u0438\u043b\u0438 \u0437\u0430\u043a\u043e\u043d\u043d\u043e \u043e\u0441\u0438\u043d\u043e\u0432\u0435\u043d\u043e.", "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": "\u0418\u043c\u0430 \u0441\u043b\u0435\u0434\u043d\u0438\u0442\u0435 \u0430\u0431\u043d\u043e\u0440\u043c\u043d\u0438 \u0445\u0435\u043c\u0430\u0442\u043e\u043b\u043e\u0433\u0438\u0447\u043d\u0438 \u0441\u0442\u043e\u0439\u043d\u043e\u0441\u0442\u0438 \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u043f\u0435\u0440\u0438\u043e\u0434\u0430 \u043d\u0430 \u0421\u043a\u0440\u0438\u043d\u0438\u043d\u0433:\na.\t\u041b\u0438\u043c\u0444\u043e\u0446\u0438\u0442\u0438 < 1,2 x 109/L\nb.\t\u041d\u0435\u0443\u0442\u0440\u043e\u0444\u0438\u043b\u0438 < 1,8 x 109/L \nc.\t\u0422\u0440\u043e\u043c\u0431\u043e\u0446\u0438\u0442\u0438 < 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": "\u0418\u043c\u0430 \u0441\u043b\u0435\u0434\u043d\u0438\u0442\u0435 \u0441\u0442\u043e\u0439\u043d\u043e\u0441\u0442\u0438 \u043e\u0442 \u0447\u0435\u0440\u043d\u043e\u0434\u0440\u043e\u0431\u0435\u043d \u0444\u0443\u043d\u043a\u0446\u0438\u043e\u043d\u0430\u043b\u0435\u043d \u0442\u0435\u0441\u0442 \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u043f\u0435\u0440\u0438\u043e\u0434\u0430 \u043d\u0430 \u0421\u043a\u0440\u0438\u043d\u0438\u043d\u0433:\na.\t\u041d\u0438\u0432\u0430 \u043d\u0430 \u0433\u0430\u043c\u0430-\u0433\u043b\u0443\u0442\u0430\u043c\u0438\u043b \u0442\u0440\u0430\u043d\u0441\u043f\u0435\u043f\u0442\u0438\u0434\u0430\u0437\u0430 (GGT) > 2,0 x \u0433\u043e\u0440\u043d\u0430\u0442\u0430 \u0433\u0440\u0430\u043d\u0438\u0446\u0430 \u043d\u0430 \u043d\u043e\u0440\u043c\u0430\u0442\u0430 (\u0413\u0413\u041d) \u0438\u043b\u0438\nb.\t\u041a\u043e\u043d\u0446\u0435\u043d\u0442\u0440\u0430\u0446\u0438\u0438 \u043d\u0430 \u043e\u0431\u0449 \u0431\u0438\u043b\u0438\u0440\u0443\u0431\u0438\u043d \u043d\u0430\u0434 1,5 x \u0413\u0413\u041d \u0438\u043b\u0438\nc.\t\u041d\u0438\u0432\u0430 \u043d\u0430 \u0433\u043b\u0443\u0442\u0430\u043c\u0430\u0442 \u0434\u0435\u0445\u0438\u0434\u0440\u043e\u0433\u0435\u043d\u0430\u0437\u0430 (Glutamate dehydrogenase, GLDH), \u043f\u043e-\u0432\u0438\u0441\u043e\u043a\u0438 \u043e\u0442 2,0 x \u0413\u0413\u041d.", "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": "\u0410\u043d\u0430\u043c\u043d\u0435\u0437\u0430 \u0437\u0430 \u043a\u043b\u0438\u043d\u0438\u0447\u043d\u043e \u0437\u043d\u0430\u0447\u0438\u043c\u043e \u043a\u044a\u0440\u0432\u0435\u043d\u0435 \u0438\u043b\u0438 \u043a\u043e\u0430\u0433\u0443\u043b\u0430\u0446\u0438\u043e\u043d\u043d\u0438 \u0430\u043d\u043e\u043c\u0430\u043b\u0438\u0438.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 787363, "number": 6, "principalExclusionCriteria": "Clinically significant abnormal coagulation parameters.", "principalExclusionCriteriaTranslations": [{"id": 5179045, "uuid": "f53ae942-53bb-4f8e-a3fd-5deb9172c256", "attributeTranslation": "\u041a\u043b\u0438\u043d\u0438\u0447\u043d\u043e \u0437\u043d\u0430\u0447\u0438\u043c\u0438 \u0430\u0431\u043d\u043e\u0440\u043c\u043d\u0438 \u043f\u0430\u0440\u0430\u043c\u0435\u0442\u0440\u0438 \u043d\u0430 \u043a\u043e\u0430\u0433\u0443\u043b\u0430\u0446\u0438\u044f.", "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": "\u0422\u0435\u043a\u0443\u0449 \u043f\u0440\u0438\u0435\u043c \u043d\u0430 \u0430\u043d\u0442\u0438\u0442\u0440\u043e\u043c\u0431\u043e\u0446\u0438\u0442\u043d\u0430 \u0438\u043b\u0438 \u0430\u043d\u0442\u0438\u043a\u043e\u0430\u0433\u0443\u043b\u0430\u043d\u0442\u043d\u0430 \u0442\u0435\u0440\u0430\u043f\u0438\u044f \u0438\u043b\u0438 \u043f\u0440\u0438\u0435\u043c \u043d\u0430 \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u043e \u0441 \u0430\u043d\u0442\u0438\u0442\u0440\u043e\u043c\u0431\u043e\u0446\u0438\u0442\u0435\u043d \u0438\u043b\u0438 \u0430\u043d\u0442\u0438\u043a\u043e\u0430\u0433\u0443\u043b\u0430\u043d\u0442\u0435\u043d \u0435\u0444\u0435\u043a\u0442 \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 4 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0414\u0435\u043d 1 (\u043d\u0430\u043f\u0440. \u0430\u0441\u043f\u0438\u0440\u0438\u043d).", "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": "\u0410\u043d\u0430\u043c\u043d\u0435\u0437\u0430 \u0437\u0430 \u0441\u0432\u0440\u044a\u0445\u0447\u0443\u0432\u0441\u0442\u0432\u0438\u0442\u0435\u043b\u043d\u043e\u0441\u0442 \u043a\u044a\u043c ATL1102 \u0438\u043b\u0438 \u043a\u044a\u043c \u043d\u044f\u043a\u043e\u0435 \u043e\u0442 \u043f\u043e\u043c\u043e\u0449\u043d\u0438\u0442\u0435 \u0432\u0435\u0449\u0435\u0441\u0442\u0432\u0430 \u0432 \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u0435\u043d\u0438\u044f \u043f\u0440\u043e\u0434\u0443\u043a\u0442 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": "\u0412\u0441\u044f\u043a\u043e \u0434\u043e\u043a\u0430\u0437\u0430\u0442\u0435\u043b\u0441\u0442\u0432\u043e \u0437\u0430 \u043a\u043b\u0438\u043d\u0438\u0447\u043d\u043e \u0437\u043d\u0430\u0447\u0438\u043c\u0430 \u0441\u0442\u0440\u0443\u043a\u0442\u0443\u0440\u043d\u0430 \u0438\u043b\u0438 \u0444\u0443\u043d\u043a\u0446\u0438\u043e\u043d\u0430\u043b\u043d\u0430 \u0441\u044a\u0440\u0434\u0435\u0447\u043d\u0430 \u0430\u043d\u043e\u043c\u0430\u043b\u0438\u044f (\u043a\u0430\u0440\u0434\u0438\u043e\u043c\u0438\u043e\u043f\u0430\u0442\u0438\u044f, \u043a\u043e\u044f\u0442\u043e \u0441\u0435 \u043a\u043e\u043d\u0442\u0440\u043e\u043b\u0438\u0440\u0430 \u0447\u0440\u0435\u0437 \u0410\u0421\u0415i \u0438\u043b\u0438 \u0431\u0435\u0442\u0430-\u0431\u043b\u043e\u043a\u0435\u0440\u0438, \u0435 \u043f\u0440\u0438\u0435\u043c\u043b\u0438\u0432\u0430, \u043f\u0440\u0438 \u0443\u0441\u043b\u043e\u0432\u0438\u0435 \u0447\u0435 \u0435 \u0438\u0437\u043f\u044a\u043b\u043d\u0435\u043d \u043a\u0440\u0438\u0442\u0435\u0440\u0438\u044f\u0442 \u0437\u0430 \u0432\u043a\u043b\u044e\u0447\u0432\u0430\u043d\u0435 \u0437\u0430 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": "\u0417\u0430\u0441\u043b\u0435\u043f\u0435\u043d\u0430 \u0444\u0430\u0437\u0430: \u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u0432 \u0441\u043a\u043e\u0440\u0430 \u0437\u0430 \u0424\u0443\u043d\u043a\u0446\u0438\u044f \u043d\u0430 \u0433\u043e\u0440\u043d\u0438\u0442\u0435 \u043a\u0440\u0430\u0439\u043d\u0438\u0446\u0438 (PUL 2.0) \u043e\u0442 \u0431\u0430\u0437\u043e\u0432\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u0434\u043e \u0421\u0435\u0434\u043c\u0438\u0446\u0430 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 \u0444\u0430\u0437\u0430: \u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u0432 \u0441\u043a\u043e\u0440\u0430 \u0437\u0430 \u0424\u0443\u043d\u043a\u0446\u0438\u044f \u043d\u0430 \u0433\u043e\u0440\u043d\u0438\u0442\u0435 \u043a\u0440\u0430\u0439\u043d\u0438\u0446\u0438 (PUL 2.0) \u043e\u0442 \u0421\u0435\u0434\u043c\u0438\u0446\u0430 25 \u0434\u043e \u0421\u0435\u0434\u043c\u0438\u0446\u0430 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 \u0444\u0430\u0437\u0430: (\u041f\u0430\u0440\u0430\u043c\u0435\u0442\u0440\u0438 \u0437\u0430 \u0431\u0435\u0437\u043e\u043f\u0430\u0441\u043d\u043e\u0441\u0442):\n1.\t\u0427\u0435\u0441\u0442\u043e\u0442\u0430 \u0438 \u0442\u0435\u0436\u0435\u0441\u0442 \u043d\u0430 \u043d\u0435\u0436\u0435\u043b\u0430\u043d\u0438 \u0441\u044a\u0431\u0438\u0442\u0438\u044f (\u041d\u0421), \u0421\u0435\u0440\u0438\u043e\u0437\u043d\u0438 \u043d\u0435\u0436\u0435\u043b\u0430\u043d\u0438 \u0441\u044a\u0431\u0438\u0442\u0438\u044f (\u0421\u041d\u0421) \u0438 \u041f\u043e\u0434\u043e\u0437\u0438\u0440\u0430\u043d\u0438 \u043d\u0435\u043e\u0447\u0430\u043a\u0432\u0430\u043d\u0438 \u0441\u0435\u0440\u0438\u043e\u0437\u043d\u0438 \u043d\u0435\u0436\u0435\u043b\u0430\u043d\u0438 \u0441\u044a\u0431\u0438\u0442\u0438\u044f (\u041f\u041d\u0421\u041d\u0420).", "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.\t\u041a\u043b\u0438\u043d\u0438\u0447\u043d\u0438 \u043b\u0430\u0431\u043e\u0440\u0430\u0442\u043e\u0440\u043d\u0438 \u0438\u0437\u0441\u043b\u0435\u0434\u0432\u0430\u043d\u0438\u044f\ni.\t\u0425\u0435\u043c\u0430\u0442\u043e\u043b\u043e\u0433\u0438\u044f \nii.\t\u041a\u043e\u0430\u0433\u0443\u043b\u0430\u0446\u0438\u044f (\u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u0444\u0438\u0431\u0440\u0438\u043d\u043e\u0433\u0435\u043d)\niii.\t\u041a\u043e\u043c\u043f\u043b\u0435\u043c\u0435\u043d\u0442\niv.\t\u0411\u0438\u043e\u0445\u0438\u043c\u0438\u044f (\u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u0445\u0430\u043f\u0442\u043e\u0433\u043b\u043e\u0431\u0438\u043d)\nv.\t\u0410\u043d\u0430\u043b\u0438\u0437 \u043d\u0430 \u0443\u0440\u0438\u043d\u0430 (\u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u0435\u0436\u0435\u0441\u0435\u0434\u043c\u0438\u0447\u043d\u0438 \u0442\u0435\u0441\u0442-\u043b\u0435\u043d\u0442\u0438).", "language": 1, "languageDescription": "Bulgarian"}]}, 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(\u043a\u0440\u044a\u0432\u043d\u043e \u043d\u0430\u043b\u044f\u0433\u0430\u043d\u0435, \u043f\u0443\u043b\u0441, \u0440\u0435\u0441\u043f\u0438\u0440\u0430\u0442\u043e\u0440\u043d\u0430 \u0447\u0435\u0441\u0442\u043e\u0442\u0430, \u0442\u0438\u043c\u043f\u0430\u043d\u0438\u0447\u043d\u0430   \u0442\u0435\u043c\u043f\u0435\u0440\u0430\u0442\u0443\u0440\u0430).", "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, 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"\u041a\u043e\u043c\u0431\u0438\u043d\u0438\u0440\u0430\u043d\u0438 \u0437\u0430\u0441\u043b\u0435\u043f\u0435\u043d\u0438 \u0438 OLE \u0444\u0430\u0437\u0438:\n1. \u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u043d\u0430 \u043f\u0440\u043e\u0438\u0437\u0432\u043e\u0434\u0438\u0442\u0435\u043b\u043d\u043e\u0441\u0442\u0442\u0430 \u043d\u0430\n\u0420\u0435\u0437\u0443\u043b\u0442\u0430\u0442 \u0437\u0430 \u0433\u043e\u0440\u0435\u043d \u043a\u0440\u0430\u0439\u043d\u0438\u043a (PUL 2.0) \u043e\u0442 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u0434\u043e \u0441\u0435\u0434\u043c\u0438\u0446\u0430 25 \u0432 \u0441\u0440\u0430\u0432\u043d\u0435\u043d\u0438\u0435 \u0441\n\u043f\u0440\u043e\u043c\u044f\u043d\u0430 \u043e\u0442 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 25 \u043d\u0430 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 49.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 531825, "number": 11, "endPoint": "Combined Blinded and Open 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predicted MyoGrip (using the Myoset System) from baseline to Week 25.", "isPrimary": false, "endPointTranslations": [{"id": 5179008, "uuid": "b10b7e76-1ea0-4f0d-98af-4fb2399412de", "attributeTranslation": "2.\t\u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u0432 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0438\u0437\u0434\u0440\u044a\u0436\u043b\u0438\u0432\u043e\u0441\u0442, \u043e\u0446\u0435\u043d\u0435\u043d\u0430 \u0447\u0440\u0435\u0437 \u043f\u0440\u043e\u0446\u0435\u043d\u0442 \u043f\u0440\u043e\u0433\u043d\u043e\u0437\u0438\u0440\u0430\u043d MyoGrip (\u0441 \u043f\u043e\u043c\u043e\u0449\u0442\u0430 \u043d\u0430 \u0441\u0438\u0441\u0442\u0435\u043c\u0430\u0442\u0430 Myoset) \u043e\u0442 \u0431\u0430\u0437\u043e\u0432\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u0434\u043e \u0421\u0435\u0434\u043c\u0438\u0446\u0430 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.\t\u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u0432 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0438\u0437\u0434\u0440\u044a\u0436\u043b\u0438\u0432\u043e\u0441\u0442, \u043e\u0446\u0435\u043d\u0435\u043d\u0430 \u0447\u0440\u0435\u0437 \u043f\u0440\u043e\u0446\u0435\u043d\u0442 \u043f\u0440\u043e\u0433\u043d\u043e\u0437\u0438\u0440\u0430\u043d MyoPinch (\u0441 \u043f\u043e\u043c\u043e\u0449\u0442\u0430 \u043d\u0430 \u0441\u0438\u0441\u0442\u0435\u043c\u0430\u0442\u0430 Myoset) \u043e\u0442 \u0431\u0430\u0437\u043e\u0432\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u0434\u043e \u0441\u0435\u0434\u043c\u0438\u0446\u0430 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. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5.", "isPrimary": false, "endPointTranslations": [{"id": 5179010, "uuid": "209b2940-e880-4e32-b85d-42441796cf8f", "attributeTranslation": "4.\t\u0421\u0442\u0435\u043f\u0435\u043d\u0442\u0430 \u043d\u0430 \u043e\u0442\u0433\u043e\u0432\u043e\u0440 \u0432 \u043e\u0431\u0449\u0438\u044f \u0444\u0443\u043d\u043a\u0446\u0438\u043e\u043d\u0430\u043b\u0435\u043d \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442 \u043d\u0430 \u0442\u0435\u0440\u0430\u043f\u0435\u0432\u0442\u0438\u0447\u043d\u0430\u0442\u0430 \u0433\u0440\u0443\u043f\u0430 \u043e\u0442 \u0431\u0430\u0437\u043e\u0432\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u0434\u043e \u0421\u0435\u0434\u043c\u0438\u0446\u0430 25 \u0449\u0435 \u0431\u044a\u0434\u0435 \u043e\u0446\u0435\u043d\u0435\u043d\u0430 \u0447\u0440\u0435\u0437 \u043a\u0443\u043c\u0443\u043b\u0430\u0442\u0438\u0432\u043d\u043e\u0442\u043e \u0440\u0430\u0437\u043f\u0440\u0435\u0434\u0435\u043b\u0435\u043d\u0438\u0435 \u043d\u0430 \u0431\u0440\u043e\u044f \u043d\u0430 \u043a\u043e\u043c\u043f\u043e\u043d\u0435\u043d\u0442\u0438\u0442\u0435 \u043d\u0430 \u043e\u0431\u0449\u0438\u044f \u0444\u0443\u043d\u043a\u0446\u0438\u043e\u043d\u0430\u043b\u0435\u043d \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442 \u043d\u0430 \u0442\u0435\u0440\u0430\u043f\u0435\u0432\u0442\u0438\u0447\u043d\u0430\u0442\u0430 \u0433\u0440\u0443\u043f\u0430 (PUL2.0, \u0434\u043e\u043c\u0438\u043d\u0438\u0440\u0430\u0449\u0430 \u0440\u044a\u043a\u0430 \u0438 \u043d\u0435\u0434\u043e\u043c\u0438\u043d\u0438\u0440\u0430\u0449\u0430 \u0440\u044a\u043a\u0430 MyoGrip \u0438 MyoPinch), \u043a\u043e\u0438\u0442\u043e \u043d\u0435 \u043d\u0430\u043c\u0430\u043b\u044f\u0432\u0430\u0442. \u041f\u0440\u043e\u043c\u0435\u043d\u043b\u0438\u0432\u0430\u0442\u0430 \u043d\u0430 \u043a\u0440\u0430\u0439\u043d\u0430\u0442\u0430 \u0442\u043e\u0447\u043a\u0430 \u0449\u0435 \u0431\u044a\u0434\u0435 \u0431\u0440\u043e\u044f\u0442 \u043d\u0430 \u043a\u043e\u043c\u043f\u043e\u043d\u0435\u043d\u0442\u0438\u0442\u0435 \u0441\u044a\u0441 \u0441\u0442\u0430\u0431\u0438\u043b\u0438\u0437\u0438\u0440\u0430\u043d\u0435, \u043a\u043e\u0435\u0442\u043e \u0432\u043e\u0434\u0438 \u0434\u043e \u0431\u0440\u043e\u0439\u043a\u0430 \u0432 \u0434\u0438\u0430\u043f\u0430\u0437\u043e\u043d\u0430 \u043e\u0442 0 \u0434\u043e 5.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 531830, "number": 5, "endPoint": "Blinded Phase (Efficacy) 5.Change of Performance of Upper Limb (PUL 2.0) score from baseline to Week 25 for each of the dimensions of the PUL2.0; high level shoulder, mid-level elbow and distal level wrist and hand.", "isPrimary": false, "endPointTranslations": [{"id": 5179011, "uuid": "d362f874-f2c6-4a7e-b870-77215157ac28", "attributeTranslation": "5.\t\u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u0432 \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442\u0430 \u043d\u0430 \u0435\u0444\u0435\u043a\u0442\u0438\u0432\u043d\u043e\u0441\u0442\u0442\u0430 \u043d\u0430 \u0433\u043e\u0440\u043d\u0438\u044f \u043a\u0440\u0430\u0439\u043d\u0438\u043a (PUL 2.0) \u043e\u0442 \u0431\u0430\u0437\u043e\u0432\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u0434\u043e \u0421\u0435\u0434\u043c\u0438\u0446\u0430 25 \u0437\u0430 \u0432\u0441\u044f\u043a\u043e \u043e\u0442 \u0438\u0437\u043c\u0435\u0440\u0435\u043d\u0438\u044f\u0442\u0430 \u043d\u0430 PUL2.0; \u0432\u0438\u0441\u043e\u043a\u043e \u043d\u0438\u0432\u043e \u043d\u0430 \u0440\u0430\u043c\u043e\u0442\u043e, \u0441\u0440\u0435\u0434\u043d\u043e \u043d\u0438\u0432\u043e \u043d\u0430 \u043b\u0430\u043a\u044a\u0442\u044f \u0438 \u0434\u0438\u0441\u0442\u0430\u043b\u043d\u043e \u043d\u0438\u0432\u043e \u043d\u0430 \u043a\u0438\u0442\u043a\u0430\u0442\u0430 \u0438 \u0440\u044a\u043a\u0430\u0442\u0430.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 531831, "number": 6, "endPoint": "Blinded Phase (Efficacy) 6.Respiratory function assessed by percent predicted PEF and percent predicted FVC from baseline to Week 25.", "isPrimary": false, "endPointTranslations": [{"id": 5179012, "uuid": "0b84abe0-5b5a-4e28-bdca-5659638060cc", "attributeTranslation": "6.\t\u0420\u0435\u0441\u043f\u0438\u0440\u0430\u0442\u043e\u0440\u043d\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f, \u043e\u0446\u0435\u043d\u0435\u043d\u0430 \u0447\u0440\u0435\u0437 \u043f\u0440\u043e\u0446\u0435\u043d\u0442 \u043f\u0440\u043e\u0433\u043d\u043e\u0437\u0438\u0440\u0430\u043d PEF \u0438 \u043f\u0440\u043e\u0446\u0435\u043d\u0442 \u043f\u0440\u043e\u0433\u043d\u043e\u0437\u0438\u0440\u0430\u043d \u0424\u0412\u041a \u043e\u0442 \u0431\u0430\u0437\u043e\u0432\u043e \u043d\u0438\u0432\u043e \u0434\u043e \u0421\u0435\u0434\u043c\u0438\u0446\u0430 25.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 531832, "number": 7, "endPoint": "Blinded Phase (Efficacy) 7.Quality of Life assessed by percentage of change in the Paediatric quality of life instrument, PedsQL\u2122 Duchenne Muscular Dystrophy (DMD) Module Patient Report and Parent Report scores (at baseline [Day 1/Week 0], Weeks 12, 24 and pre-dose Week 25).", "isPrimary": false, "endPointTranslations": [{"id": 5179013, "uuid": "ee47f70a-d8f7-46fa-9307-fc01b89d0396", "attributeTranslation": "7.\t\u041a\u0430\u0447\u0435\u0441\u0442\u0432\u043e \u043d\u0430 \u0436\u0438\u0432\u043e\u0442, \u043e\u0446\u0435\u043d\u0435\u043d\u043e \u0447\u0440\u0435\u0437 \u043f\u0440\u043e\u0446\u0435\u043d\u0442 \u043d\u0430 \u043f\u0440\u043e\u043c\u044f\u043d\u0430\u0442\u0430 \u0432 \u0418\u043d\u0441\u0442\u0440\u0443\u043c\u0435\u043d\u0442\u0430 \u0437\u0430 \u043a\u0430\u0447\u0435\u0441\u0442\u0432\u043e \u043d\u0430 \u0436\u0438\u0432\u043e\u0442 \u043f\u0440\u0438 \u043f\u0435\u0434\u0438\u0430\u0442\u0440\u0438\u0447\u043d\u0438 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u0438 (PedsQL\u2122) \u0437\u0430 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u044f \u0442\u0438\u043f \u0414\u044e\u0448\u0435\u043d (\u041c\u0414\u0414) \u0438 \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442\u0438\u0442\u0435 \u043e\u0442 \u0414\u043e\u043a\u043b\u0430\u0434 \u043d\u0430 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u0430 \u0438 \u0414\u043e\u043a\u043b\u0430\u0434 \u043d\u0430 \u0440\u043e\u0434\u0438\u0442\u0435\u043b\u044f (\u043d\u0430 \u0431\u0430\u0437\u043e\u0432\u043e \u043d\u0438\u0432\u043e [\u0414\u0435\u043d 1/\u0421\u0435\u0434\u043c\u0438\u0446\u0430 0], \u0421\u0435\u0434\u043c\u0438\u0446\u0438 12, 24 \u0438 25).", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 531833, "number": 8, "endPoint": "OLE Phase (Efficacy): 1.Percentage of treatment responders at Week 49, where a treatment responder is defined as no decline from baseline at entry into this extension study in Performance of Upper Limb (PUL 2.0).", "isPrimary": false, "endPointTranslations": [{"id": 5179014, "uuid": "36c37d5a-a7e9-4a29-a3c0-4f2026dfbce0", "attributeTranslation": "OLE \u0444\u0430\u0437\u0430 (\u0435\u0444\u0438\u043a\u0430\u0441\u043d\u043e\u0441\u0442):\n1.\t\u041f\u0440\u043e\u0446\u0435\u043d\u0442 \u043d\u0430 \u043e\u0442\u0433\u043e\u0432\u043e\u0440\u0438\u043b\u0438 \u043d\u0430 \u043b\u0435\u0447\u0435\u043d\u0438\u0435\u0442\u043e \u0432 \u0421\u0435\u0434\u043c\u0438\u0446\u0430 49, \u043a\u0430\u0442\u043e \u043e\u0442\u0433\u043e\u0432\u043e\u0440 \u043d\u0430 \u043b\u0435\u0447\u0435\u043d\u0438\u0435\u0442\u043e \u0441\u0435 \u0434\u0435\u0444\u0438\u043d\u0438\u0440\u0430 \u043a\u0430\u0442\u043e \u043e\u0442\u0441\u044a\u0441\u0442\u0432\u0438\u0435 \u043d\u0430 \u0432\u043b\u043e\u0448\u0430\u0432\u0430\u043d\u0435 \u043e\u0442 \u0431\u0430\u0437\u043e\u0432\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u043f\u0440\u0438 \u0432\u043b\u0438\u0437\u0430\u043d\u0435\u0442\u043e \u0432 \u0442\u043e\u0432\u0430 \u043f\u0440\u043e\u0434\u044a\u043b\u0436\u0435\u043d\u0438\u0435 \u043d\u0430 \u043f\u0440\u043e\u0443\u0447\u0432\u0430\u043d\u0435\u0442\u043e \u0432 \u043e\u0446\u0435\u043d\u043a\u0430\u0442\u0430 \u0437\u0430 \u0424\u0443\u043d\u043a\u0446\u0438\u044f \u043d\u0430 \u0433\u043e\u0440\u043d\u0438\u0442\u0435 \u043a\u0440\u0430\u0439\u043d\u0438\u0446\u0438 (PUL 2.0).", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 531834, "number": 9, "endPoint": "OLE Phase (Efficacy) 2.Change in Muscle strength as assessed by and percent predicted MyoGrip (using the Myoset System) from Week 25 to Week 49.", "isPrimary": false, "endPointTranslations": [{"id": 5179015, "uuid": "7dc2f3d2-f844-4b12-a806-d55997b6e715", "attributeTranslation": "2.\t\u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u0432 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0438\u0437\u0434\u0440\u044a\u0436\u043b\u0438\u0432\u043e\u0441\u0442, \u043e\u0446\u0435\u043d\u0435\u043d\u0430 \u0447\u0440\u0435\u0437 \u043f\u0440\u043e\u0446\u0435\u043d\u0442 \u043f\u0440\u043e\u0433\u043d\u043e\u0437\u0438\u0440\u0430\u043d MyoGrip (\u0441 \u043f\u043e\u043c\u043e\u0449\u0442\u0430 \u043d\u0430 \u0441\u0438\u0441\u0442\u0435\u043c\u0430\u0442\u0430 Myoset) \u043e\u0442 \u0421\u0435\u0434\u043c\u0438\u0446\u0430 25 \u0434\u043e \u0421\u0435\u0434\u043c\u0438\u0446\u0430 49.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 531835, "number": 10, "endPoint": "OLE Phase (Efficacy) 3.Change in Muscle strength as assessed by percent predicted MyoPinch (using the Myoset System) from Week 25 to Week 49.", "isPrimary": false, "endPointTranslations": [{"id": 5179016, "uuid": "077c0bcc-192f-489e-a2cc-ef1e572eb2e2", "attributeTranslation": "3.\t\u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u0432 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0438\u0437\u0434\u0440\u044a\u0436\u043b\u0438\u0432\u043e\u0441\u0442, \u043e\u0446\u0435\u043d\u0435\u043d\u0430 \u0447\u0440\u0435\u0437 \u043f\u0440\u043e\u0446\u0435\u043d\u0442 \u043f\u0440\u043e\u0433\u043d\u043e\u0437\u0438\u0440\u0430\u043d MyoPinch (\u0441 \u043f\u043e\u043c\u043e\u0449\u0442\u0430 \u043d\u0430 \u0441\u0438\u0441\u0442\u0435\u043c\u0430\u0442\u0430 Myoset) \u043e\u0442 \u0421\u0435\u0434\u043c\u0438\u0446\u0430 25 \u0434\u043e \u0421\u0435\u0434\u043c\u0438\u0446\u0430 49.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 531836, "number": 11, "endPoint": "OLE Phase (Efficacy) 4.The extent of response in a total arm functional score from Week 25 to Week 49 will be assessed by the cumulative distribution of the number of the components of total arm functional score (PUL2.0, right hand and left hand MyoGrip and MyoPinch) that do not decline. The endpoint variable will be the count of the number of components with stabilisation yielding a count ranging from 0 to 5.", "isPrimary": false, "endPointTranslations": [{"id": 5179017, "uuid": "9f0a49b4-86ed-4604-910c-d499873f0369", "attributeTranslation": "4.\t\u0421\u0442\u0435\u043f\u0435\u043d\u0442\u0430 \u043d\u0430 \u043e\u0442\u0433\u043e\u0432\u043e\u0440 \u0432 \u043e\u0431\u0449\u0438\u044f \u0444\u0443\u043d\u043a\u0446\u0438\u043e\u043d\u0430\u043b\u0435\u043d \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442 \u043d\u0430 \u0442\u0435\u0440\u0430\u043f\u0435\u0432\u0442\u0438\u0447\u043d\u0430\u0442\u0430 \u0433\u0440\u0443\u043f\u0430 \u043e\u0442 \u0421\u0435\u0434\u043c\u0438\u0446\u0430 25 \u0434\u043e \u0421\u0435\u0434\u043c\u0438\u0446\u0430 49 \u0449\u0435 \u0431\u044a\u0434\u0435 \u043e\u0446\u0435\u043d\u0435\u043d\u0430 \u0447\u0440\u0435\u0437 \u043a\u0443\u043c\u0443\u043b\u0430\u0442\u0438\u0432\u043d\u043e\u0442\u043e \u0440\u0430\u0437\u043f\u0440\u0435\u0434\u0435\u043b\u0435\u043d\u0438\u0435 \u043d\u0430 \u0431\u0440\u043e\u044f \u043d\u0430 \u043a\u043e\u043c\u043f\u043e\u043d\u0435\u043d\u0442\u0438\u0442\u0435 \u043d\u0430 \u043e\u0431\u0449\u0438\u044f \u0444\u0443\u043d\u043a\u0446\u0438\u043e\u043d\u0430\u043b\u0435\u043d \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442 \u043d\u0430 \u0442\u0435\u0440\u0430\u043f\u0435\u0432\u0442\u0438\u0447\u043d\u0430\u0442\u0430 \u0433\u0440\u0443\u043f\u0430 (PUL2.0, \u0434\u044f\u0441\u043d\u0430\u0442\u0430 \u0440\u044a\u043a\u0430 \u0438 \u043b\u044f\u0432\u0430\u0442\u0430 \u0440\u044a\u043a\u0430 MyoGrip \u0438 MyoPinch), \u043a\u043e\u0438\u0442\u043e \u043d\u0435 \u043d\u0430\u043c\u0430\u043b\u044f\u0432\u0430\u0442. \u041f\u0440\u043e\u043c\u0435\u043d\u043b\u0438\u0432\u0430\u0442\u0430 \u043d\u0430 \u043a\u0440\u0430\u0439\u043d\u0430\u0442\u0430 \u0442\u043e\u0447\u043a\u0430 \u0449\u0435 \u0431\u044a\u0434\u0435 \u0431\u0440\u043e\u044f\u0442 \u043d\u0430 \u043a\u043e\u043c\u043f\u043e\u043d\u0435\u043d\u0442\u0438\u0442\u0435 \u0441\u044a\u0441 \u0441\u0442\u0430\u0431\u0438\u043b\u0438\u0437\u0438\u0440\u0430\u043d\u0435, \u043a\u043e\u0435\u0442\u043e \u0432\u043e\u0434\u0438 \u0434\u043e \u0431\u0440\u043e\u0439\u043a\u0430 \u0432 \u0434\u0438\u0430\u043f\u0430\u0437\u043e\u043d\u0430 \u043e\u0442 0 \u0434\u043e 5.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 531837, "number": 12, "endPoint": "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.", "isPrimary": false, "endPointTranslations": [{"id": 5179018, "uuid": "c253acec-b77b-43de-a1ee-d45d8db46ed8", "attributeTranslation": "5.\t\u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u0432 \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442\u0430 \u043d\u0430 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"Bulgarian"}]}, {"id": 531842, "number": 17, "endPoint": "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.", "isPrimary": false, "endPointTranslations": [{"id": 5179023, "uuid": "95e77342-0407-4a5b-a142-8d42d7f31c71", "attributeTranslation": "3.\t\u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u0432 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0441\u0438\u043b\u0430, \u043e\u0446\u0435\u043d\u0435\u043d\u0430 \u0447\u0440\u0435\u0437 \u043f\u0440\u043e\u0446\u0435\u043d\u0442\u043d\u043e \u043f\u0440\u043e\u0433\u043d\u043e\u0437\u0438\u0440\u0430\u043d MyoPinch (\u0441 \u043f\u043e\u043c\u043e\u0449\u0442\u0430 \u043d\u0430 \u0441\u0438\u0441\u0442\u0435\u043c\u0430\u0442\u0430 Myoset) \u043e\u0442 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45 o 53.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861367, "uuid": "c2c587bf-fc25-498f-a07a-00714a9187e5", "attributeTranslation": "A 45. vagy 53. exon \u00e1tugr\u00e1s\u00e1val befoly\u00e1solhat\u00f3 Duchenne-f\u00e9le izomdisztr\u00f3fi\u00e1ban szenved\u0151 betegek", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861365, "uuid": "c2c587bf-fc25-498f-a07a-00714a9187e5", "attributeTranslation": "\u041f\u0430\u0446\u0438\u0435\u043d\u0442\u0438 \u0441 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u044f \u043d\u0430 \u0414\u044e\u0448\u0435\u043d, \u043f\u043e\u0434\u0430\u0442\u043b\u0438\u0432\u0438 \u043d\u0430 \u043f\u0440\u0435\u0441\u043a\u0430\u0447\u0430\u043d\u0435 \u043d\u0430 \u0435\u043a\u0437\u043e\u043d 45 \u0438\u043b\u0438 53.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861366, "uuid": "c2c587bf-fc25-498f-a07a-00714a9187e5", "attributeTranslation": "pacjenci z dystrofi\u0105 mi\u0119\u015bniow\u0105 Duchenne'a podatn\u0105 na pomijanie egzonu 45 lub 53", "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": 265398}, {"code": "5", "trialScopeId": 265397}, {"code": "6", "trialScopeId": 265395}, {"code": "4", "trialScopeId": 265396}, {"code": "7", "trialScopeId": 265399}], "mainObjective": "Evaluate the effect of SRP-4045 and SRP-4053(combined-active group) compared with placebo on ambulation and muscle function, as\nmeasured by the 4-step ascend velocity.", "mainObjectiveTranslations": [{"id": 6861466, "uuid": "dc93ccb0-e278-434f-8b14-818a6cd1edb5", "attributeTranslation": "ocena wp\u0142ywu SRP 4045 oraz SRP 4053 (grupa pacjent\u00f3w z kt\u00f3rymkolwiek\nrodzajem leczenia czynnego) w por\u00f3wnaniu do placebo na zdolno\u015b\u0107 do chodzenia oraz czynno\u015b\u0107 mi\u0119\u015bni, zgodnie z wynikami testu oceniaj\u0105cego czas na pokonanie 4 schod\u00f3w.", "language": 19, "languageDescription": "Polish"}, {"id": 6861465, "uuid": "dc93ccb0-e278-434f-8b14-818a6cd1edb5", "attributeTranslation": "\u0414\u0430 \u0441\u0435 \u043e\u0446\u0435\u043d\u0438 \u0435\u0444\u0435\u043a\u0442\u0430 \u043d\u0430 SRP-4045 \u0438 SRP-4053 (\u043a\u043e\u043c\u0431\u0438\u043d\u0438\u0440\u0430\u043d\u0430 \u0430\u043a\u0442\u0438\u0432\u043d\u0430 \u0433\u0440\u0443\u043f\u0430) \u0441\u043f\u0440\u044f\u043c\u043e \u043f\u043b\u0430\u0446\u0435\u0431\u043e", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861467, "uuid": "dc93ccb0-e278-434f-8b14-818a6cd1edb5", "attributeTranslation": "\u043f\u043e \u043e\u0442\u043d\u043e\u0448\u0435\u043d\u0438\u0435 \u043d\u0430 \u0445\u043e\u0434\u0435\u043d\u0435\u0442\u043e, \u0438\u0437\u0434\u0440\u044a\u0436\u043b\u0438\u0432\u043e\u0441\u0442\u0442\u0430 \u0438 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f, \u0438\u0437\u043c\u0435\u0440\u0435\u043d\u0438 \u0441 \u043f\u043e\u043c\u043e\u0449\u0442\u0430 \u043d\u0430 \u0442\u0435\u0441\u0442 \u0437\u0430 \u0441\u043a\u043e\u0440\u043e\u0441\u0442 \u043d\u0430 \u0438\u0437\u043a\u0430\u0447\u0432\u0430\u043d\u0435 \u043d\u0430 4 \u0441\u0442\u044a\u043f\u0430\u043b\u0430", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861464, "uuid": "dc93ccb0-e278-434f-8b14-818a6cd1edb5", "attributeTranslation": "Evaluar el efecto de SRP-4045 y SRP-4053 (grupo de tratamiento activo combinado) en comparaci\u00f3n con un placebo sobre la deambulaci\u00f3n y la funci\u00f3n muscular, determinadas mediante la velocidad para subir 4 escalones.", "language": 7, "languageDescription": "Spanish"}], "secondaryObjectives": [{"id": 302897, "number": 1, "secondaryObjective": "Double-blind period: evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) on: \u2022\tDystrophin protein expression in biopsied muscle tissue as measured by:  -\tWestern blot (quantification) -\tImmunohistochemistry (IHC) fiber intensity", "secondaryObjectiveTranslations": [{"id": 6861475, "uuid": "e0a02283-23e3-4b6c-a519-317c3b8a9f4e", "attributeTranslation": "Kett\u0151s vak szakasz: az SRP-4045 \u00e9s SRP-4053 (kombin\u00e1lt akt\u00edv csoport) hat\u00e1s\u00e1nak \u00e9rt\u00e9kel\u00e9se az al\u00e1bbiak szempontj\u00e1b\u00f3l: \u2022\tA disztrofin feh\u00e9rje expresszi\u00f3ja biopszi\u00e1s izomsz\u00f6vetben, az al\u00e1bbiakkal m\u00e9rve: \n-\tWestern blot (mennyis\u00e9gi meghat\u00e1roz\u00e1s)\n-\tImmunhisztok\u00e9mi\u00e1val (IHC) meghat\u00e1rozott rostintenzit\u00e1s", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861474, "uuid": "e0a02283-23e3-4b6c-a519-317c3b8a9f4e", "attributeTranslation": "okres leczenia w fazie podw\u00f3jnie za\u015blepionej: ocena wp\u0142ywu SRP 4045 oraz SRP 4053 (grupa pacjent\u00f3w z kt\u00f3rymkolwiek rodzajem leczenia czynnego) na: \u2022\tekspresj\u0119 dystrofiny w tkance mi\u0119\u015bniowej pobranej w czasie biopsji, zgodnie z ocen\u0105 metodami: \n-\twestern blot (analiza ilo\u015bciowa);\n-\timmunohistochemiczna (ang. immunohistochemistry, IHC) ocena g\u0119sto\u015bci w\u0142\u00f3kien;", "language": 19, "languageDescription": "Polish"}, {"id": 6861472, "uuid": "e0a02283-23e3-4b6c-a519-317c3b8a9f4e", "attributeTranslation": "Periodo doble ciego: evaluar el efecto de SRP-4045 y SRP-4053 (combinados, grupo de principio activo) sobre \u2022\tla expresi\u00f3n de la prote\u00edna distrofina en el tejido muscular bi\u00f3psico, seg\u00fan: \n-\tLa cuantificaci\u00f3n mediante inmunoelectrotransferencia (Western blot).\n-\tLa intensidad de expresi\u00f3n de las fibras mediante pruebas inmunohistoqu\u00edmicas (IHQ).", "language": 7, "languageDescription": "Spanish"}, {"id": 6861473, "uuid": "e0a02283-23e3-4b6c-a519-317c3b8a9f4e", "attributeTranslation": "\u0414\u0432\u043e\u0439\u043d\u043e \u0441\u043b\u044f\u043f \u043f\u0435\u0440\u0438\u043e\u0434: \u043e\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u0435\u0444\u0435\u043a\u0442\u0430 \u043d\u0430 SRP-4045 \u0438 SRP-4053 (\u043a\u043e\u043c\u0431\u0438\u043d\u0438\u0440\u0430\u043d\u0430 \u0430\u043a\u0442\u0438\u0432\u043d\u0430 \u0433\u0440\u0443\u043f\u0430) \u043f\u043e \u043e\u0442\u043d\u043e\u0448\u0435\u043d\u0438\u0435 \u043d\u0430:\n\u2022\t\u0415\u043a\u0441\u043f\u0440\u0435\u0441\u0438\u044f \u043d\u0430 \u0431\u0435\u043b\u0442\u044a\u043a\u0430 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u043d \u0432 \u0431\u0438\u043e\u043f\u0441\u0438\u0447\u043d\u0430 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430 \u0442\u044a\u043a\u0430\u043d, \u0438\u0437\u043c\u0435\u0440\u0435\u043d\u0430 \u0447\u0440\u0435\u0437: \n-\t\u0422\u0435\u0441\u0442 Western blot (\u043a\u043e\u043b\u0438\u0447\u0435\u0441\u0442\u0432\u0435\u043d\u043e \u043e\u043f\u0440\u0435\u0434\u0435\u043b\u044f\u043d\u0435)\n-\t\u0418\u043d\u0442\u0435\u043d\u0437\u0438\u0442\u0435\u0442 \u043d\u0430 \u0444\u0438\u0431\u0440\u0438\u0442\u0435, \u043e\u043f\u0440\u0435\u0434\u0435\u043b\u0435\u043d \u0447\u0440\u0435\u0437 \u0438\u043c\u0443\u043d\u043e\u0445\u0438\u0441\u0442\u043e\u0445\u0438\u043c\u0438\u044f (IHC)", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 302898, "number": 2, "secondaryObjective": "Functional status as measured by:  \u2022 6MWT \u2022 10MWR \u2022 4-step ascend velocity \u2022 Rise from floor velocity  \u2022  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 oceniaj\u0105cym czas na pokonanie 4 schod\u00f3w; ocen\u0119 zdolno\u015bci do samodzielnego wstawania z pod\u0142ogi, ocen\u0119 zdolno\u015bci 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\u2022 la prueba de marcha de seis minutos (PM6M)\n\u2022 la prueba de caminar/correr 10 metros\n\u2022 la velocidad para subir 4 escalones\n\u2022 la velocidad para levantarse del suelo\n\u2022 la evaluaci\u00f3n ambulatoria North Star (NSAA)", "language": 7, "languageDescription": "Spanish"}, {"id": 6861479, "uuid": "73c58d57-6d69-44cd-a154-5f84e3d2e0fe", "attributeTranslation": "-Funkcion\u00e1lis \u00e1llapot a k\u00f6vetkez\u0151k szerint: \n- 6 perces j\u00e1r\u00e1steszt (6MWT)\n- 10 m\u00e9teres j\u00e1r\u00e1steszt (10MWR)\n- 4 l\u00e9p\u00e9ses emelked\u00e9si sebess\u00e9g\n- Talajr\u00f3l val\u00f3 fel\u00e1ll\u00e1s sebess\u00e9ge\n- North Star ambul\u00e1ns vizsg\u00e1lat (NSAA)", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861478, "uuid": "73c58d57-6d69-44cd-a154-5f84e3d2e0fe", "attributeTranslation": "\u0424\u0443\u043d\u043a\u0446\u0438\u043e\u043d\u0430\u043b\u0435\u043d \u0441\u0442\u0430\u0442\u0443\u0441, \u0438\u0437\u043c\u0435\u0440\u0435\u043d \u0447\u0440\u0435\u0437:\n- 6 -\u043c\u0438\u043d\u0443\u0442\u0435\u043d \u0442\u0435\u0441\u0442 \u0437\u0430 \u0445\u043e\u0434\u0435\u0442\u0435\n-  10 \u043c\u0435\u0442\u0440\u0430 \u0445\u043e\u0434\u0435\u043d\u0435 \u0438\u043b\u0438 \u0442\u0438\u0447\u0430\u043d\u0435\n- \u0421\u043a\u043e\u0440\u043e\u0441\u0442 \u043d\u0430 \u0438\u0437\u043a\u0430\u0447\u0432\u0430\u043d\u0435 \u043d\u0430 4 \u0441\u0442\u044a\u043f\u0430\u043b\u0430\n- \u0421\u043a\u043e\u0440\u043e\u0441\u0442 \u043d\u0430 \u0441\u0442\u0430\u0432\u0430\u043d\u0435 \u043e\u0442 \u043f\u043e\u0434\u0430\n- \u041e\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u043f\u043e\u0434\u0432\u0438\u0436\u043d\u043e\u0441\u0442\u0442\u0430 \u043d\u0430 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u0430 \u043f\u043e \u0441\u0438\u0441\u0442\u0435\u043c\u0430\u0442\u0430 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: \u2022\tSafety and tolerability of SRP-4045 and SRP-4053.", "secondaryObjectiveTranslations": [{"id": 6861480, "uuid": "d474e63d-cf84-46bf-80d4-41dd9c7636dd", "attributeTranslation": "\u0414\u0432\u043e\u0439\u043d\u043e \u0441\u043b\u044f\u043f \u043f\u0435\u0440\u0438\u043e\u0434: \u043e\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u0435\u0444\u0435\u043a\u0442\u0430 \u043d\u0430 SRP-4045 \u0438 SRP-4053 (\u043a\u043e\u043c\u0431\u0438\u043d\u0438\u0440\u0430\u043d\u0430 \u0430\u043a\u0442\u0438\u0432\u043d\u0430 \u0433\u0440\u0443\u043f\u0430) \u043f\u043e \u043e\u0442\u043d\u043e\u0448\u0435\u043d\u0438\u0435 \u043d\u0430: \u2022\t\u0411\u0435\u0437\u043e\u043f\u0430\u0441\u043d\u043e\u0441\u0442 \u0438 \u043f\u043e\u043d\u043e\u0441\u0438\u043c\u043e\u0441\u0442 \u043d\u0430 SRP-4045 \u0438 SRP-4053.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861483, "uuid": "d474e63d-cf84-46bf-80d4-41dd9c7636dd", "attributeTranslation": "Kett\u0151s vak szakasz: az SRP-4045 \u00e9s SRP-4053 (kombin\u00e1lt akt\u00edv csoport) hat\u00e1s\u00e1nak \u00e9rt\u00e9kel\u00e9se az al\u00e1bbiak szempontj\u00e1b\u00f3l: \u2022\tAz SRP-4045 \u00e9s SRP-4053 biztons\u00e1goss\u00e1ga \u00e9s toler\u00e1lhat\u00f3s\u00e1ga.", "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 \u2022\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 podw\u00f3jnie za\u015blepionej: ocena wp\u0142ywu SRP 4045 oraz SRP 4053 (grupa pacjent\u00f3w z kt\u00f3rymkolwiek rodzajem leczenia czynnego) na: \u2022\tbezpiecze\u0144stwo stosowania i tolerancj\u0119 SRP 4045 i SRP 4053.", "language": 19, "languageDescription": "Polish"}]}, {"id": 302900, "number": 4, "secondaryObjective": "Open-label Treatment Period:  \u2022\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: \u2022\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": "\u041f\u0435\u0440\u0438\u043e\u0434 \u043d\u0430 \u043e\u0442\u043a\u0440\u0438\u0442\u043e \u043b\u0435\u0447\u0435\u043d\u0438\u0435: \u2022\t\u041e\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u0434\u044a\u043b\u0433\u043e\u0441\u0440\u043e\u0447\u043d\u0438\u0442\u0435 \u0435\u0444\u0435\u043a\u0442\u0438 \u043d\u0430 \u043b\u0435\u0447\u0435\u043d\u0438\u0435\u0442\u043e \u0441\u044a\u0441 SRP-4045 \u0438 SRP-4053 \u0432\u044a\u0440\u0445\u0443 \u0444\u0443\u043d\u043a\u0446\u0438\u043e\u043d\u0430\u043b\u043d\u0438\u044f \u0441\u0442\u0430\u0442\u0443\u0441 \u0432 \u043f\u0440\u043e\u0434\u044a\u043b\u0436\u0435\u043d\u0438\u0435 \u043d\u0430 \u0434\u043e 144 \u0441\u0435\u0434\u043c\u0438\u0446\u0438.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861487, "uuid": "3a03e2c0-5eac-4eb2-9a28-ef6795551fa7", "attributeTranslation": "Okres leczenia w fazie otwartej: \u2022\tocena d\u0142ugoterminowego wp\u0142ywu leczenia SRP 4045 oraz SRP 4053 na zaburzenia czynno\u015bciowe w okresie do 144 tygodni;", "language": 19, "languageDescription": "Polish"}, {"id": 6861484, "uuid": "3a03e2c0-5eac-4eb2-9a28-ef6795551fa7", "attributeTranslation": "Ny\u00edlt kezel\u00e9si szakasz: \u2022\tAz SRP-4045 \u00e9s SRP-4053 kezel\u00e9s funkcion\u00e1lis st\u00e1tuszra gyakorolt hossz\u00fa t\u00e1v\u00fa hat\u00e1sainak \u00e9rt\u00e9kel\u00e9se a 144. h\u00e9t v\u00e9g\u00e9ig.", "language": 13, "languageDescription": "Hungarian"}]}, {"id": 302901, "number": 5, "secondaryObjective": "Open-label Treatment Period:  \u2022\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: \u2022\tocena d\u0142ugoterminowa bezpiecze\u0144stwa stosowania i tolerancji na leczenie SRP 4045 oraz SRP 4053;", "language": 19, "languageDescription": "Polish"}, {"id": 6861491, "uuid": "71d23544-de8b-48f1-9a19-9fc585d8e293", "attributeTranslation": "Ny\u00edlt kezel\u00e9si szakasz: \u2022\tAz SRP-4045 \u00e9s SRP-4053 hossz\u00fa t\u00e1v\u00fa biztons\u00e1goss\u00e1g\u00e1nak \u00e9s toler\u00e1lhat\u00f3s\u00e1g\u00e1nak \u00e9rt\u00e9kel\u00e9se.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861488, "uuid": "71d23544-de8b-48f1-9a19-9fc585d8e293", "attributeTranslation": "\u041f\u0435\u0440\u0438\u043e\u0434 \u043d\u0430 \u043e\u0442\u043a\u0440\u0438\u0442\u043e \u043b\u0435\u0447\u0435\u043d\u0438\u0435: \u2022\t\u041e\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u0434\u044a\u043b\u0433\u043e\u0441\u0440\u043e\u0447\u043d\u0430\u0442\u0430 \u0431\u0435\u0437\u043e\u043f\u0430\u0441\u043d\u043e\u0441\u0442 \u0438 \u043f\u043e\u043d\u043e\u0441\u0438\u043c\u043e\u0441\u0442 \u043d\u0430 SRP-4045 \u0438 SRP-4053.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861489, "uuid": "71d23544-de8b-48f1-9a19-9fc585d8e293", "attributeTranslation": "Periodo abierto de tratamiento del estudio: \u2022\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 farmacocin\u00e9tico:\nEvaluar las propiedades FC de SRP-4045 y SRP-4053 mediante un modelo farmacocin\u00e9tico poblacional.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861495, "uuid": "231622a7-1744-4da6-abb4-1f3d871c7310", "attributeTranslation": "Cel farmakokinetyczny:\nocena w\u0142asno\u015bci farmakokinetycznych SRP 4045 oraz SRP 4053 z wykorzystaniem modelu farmakokinetyki populacyjnej.", "language": 19, "languageDescription": "Polish"}, {"id": 6861492, "uuid": "231622a7-1744-4da6-abb4-1f3d871c7310", "attributeTranslation": "\u0424\u0430\u0440\u043c\u0430\u043a\u043e\u043a\u0438\u043d\u0435\u0442\u0438\u0447\u043d\u0430 \u0446\u0435\u043b:\n\u041e\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u0424\u041a \u0441\u0432\u043e\u0439\u0441\u0442\u0432\u0430\u0442\u0430 \u043d\u0430 SRP-4045 \u0438 SRP-4053 \u0441 \u043f\u043e\u043c\u043e\u0449\u0442\u0430 \u043d\u0430 \u043f\u043e\u043f\u0443\u043b\u0430\u0446\u0438\u043e\u043d\u0435\u043d \u0424\u041a \u043c\u043e\u0434\u0435\u043b.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861493, "uuid": "231622a7-1744-4da6-abb4-1f3d871c7310", "attributeTranslation": "Farmakokinetikai c\u00e9lkit\u0171z\u00e9s:\nAz SRP-4045 \u00e9s SRP-4053 PK jellemz\u0151inek \u00e9rt\u00e9kel\u00e9se popul\u00e1ci\u00f3s PK modell seg\u00edts\u00e9g\u00e9vel.", "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 p\u0142ci m\u0119skiej z potwierdzonym rozpoznaniem dystrofii mi\u0119\u015bniowej Duchenne\u2019a oraz delecj\u0105 zmieniaj\u0105c\u0105 ramk\u0119 odczytu, odpowiedzialn\u0105 za:\n\u2022 pomini\u0119cie egzonu 45 (w\u0142\u0105czaj\u0105c w to, ale nie ograniczaj\u0105c do delecji egzon\u00f3w takich jak 12\u201344, 18\u201344, 44, 46\u201347, 46\u201348, 46\u201349, 46\u201351, 46\u201353 lub 46\u201355); LUB\n\u2022 pomini\u0119cie egzonu 53 (w\u0142\u0105czaj\u0105c w to, ale nie ograniczaj\u0105c do delecji egzon\u00f3w takich jak 42\u201352, 45\u201352, 47\u201352, 48\u201352, 49\u201352, 50\u201352, 52 lub 54\u201358);\nzgodnie z raportem zawieraj\u0105cym wyniki bada\u0144 genetycznych przeprowadzonych w akredytowanym laboratorium przed rozpocz\u0119ciem bada\u0144 przesiewowych, wykazuj\u0105cym granice delecji poprzez multipleksow\u0105 amplifikacj\u0119 zale\u017cn\u0105 od ligacji lub poprzez sekwencjonowanie. Delecja egzon\u00f3w 45 lub 53 u pacjenta musi zosta\u0107 potwierdzona z wykorzystaniem genotypowania przeprowadzonego w czasie bada\u0144 przesiewowych przed podaniem pierwszej dawki", "language": 19, "languageDescription": "Polish"}, {"id": 6861425, "uuid": "24a16e3e-902b-465a-8480-43be77227784", "attributeTranslation": "Fi\u00fa DMD meg\u00e1llap\u00edtott klinikai diagn\u00f3zis\u00e1val \u00e9s out-of-frame del\u00e9ci\u00f3val, amely befoly\u00e1solhat\u00f3 az al\u00e1bbiakkal:\n\u2022 A 45. exon \u00e1tugr\u00e1sa (bele\u00e9rtve t\u00f6bbek k\u00f6z\u00f6tt a k\u00f6vetkez\u0151 exonok del\u00e9ci\u00f3j\u00e1t: 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53 vagy 46-55) VAGY\n\u2022 Az 53. exon \u00e1tugr\u00e1sa (bele\u00e9rtve t\u00f6bbek k\u00f6z\u00f6tt a k\u00f6vetkez\u0151 exonok del\u00e9ci\u00f3j\u00e1t: 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52 vagy 54-58)\nAkkredit\u00e1lt laborat\u00f3rium \u00e1ltal a sz\u0171r\u00e9s el\u0151tt kiadott genetikai lelettel dokument\u00e1lva, amely tartalmazza a multiplex lig\u00e1ci\u00f3-f\u00fcgg\u0151 pr\u00f3ba amplifik\u00e1ci\u00f3val vagy szekven\u00e1l\u00e1ssal meghat\u00e1rozott del\u00e9ci\u00f3s v\u00e9gpontokat. A betegn\u00e9l a 45. exon vagy az 53. exon szkipping \u00e1ltali befoly\u00e1solhat\u00f3s\u00e1got az els\u0151 adag bead\u00e1sa el\u0151tt igazolni kell a sz\u0171r\u00e9s alatt kapott genot\u00edpus-meghat\u00e1roz\u00e1s eredm\u00e9nyeit felhaszn\u00e1lva.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861424, "uuid": "24a16e3e-902b-465a-8480-43be77227784", "attributeTranslation": "Ser var\u00f3n con un diagn\u00f3stico cl\u00ednico confirmado de DMD y una deleci\u00f3n fuera del marco de lectura susceptible de:\n\u2022 Omisi\u00f3n del ex\u00f3n 45 (incluida, entre otras, la deleci\u00f3n de los exones 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53 o 46-55) O BIEN\n\u2022 Omisi\u00f3n del ex\u00f3n 53 (incluida, entre otras, la deleci\u00f3n de los exones 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52 o 54-58)\ndocumentada antes de la selecci\u00f3n mediante un informe gen\u00e9tico de un laboratorio acreditado en el que se defina el valor m\u00e1ximo de deleci\u00f3n mediante secuenciaci\u00f3n o amplificaci\u00f3n m\u00faltiple de sondas ligadas. La susceptibilidad del paciente de recibir una deleci\u00f3n de los exones 45 o 53 se debe confirmar antes de administrar la primera dosis del f\u00e1rmaco. Para ello, han de utilizarse los resultados de genotipado obtenidos durante la selecci\u00f3n.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861427, "uuid": "24a16e3e-902b-465a-8480-43be77227784", "attributeTranslation": "\u0415 \u043e\u0442 \u043c\u044a\u0436\u043a\u0438 \u043f\u043e\u043b \u0441 \u0443\u0441\u0442\u0430\u043d\u043e\u0432\u0435\u043d\u0430 \u043a\u043b\u0438\u043d\u0438\u0447\u043d\u0430 \u0434\u0438\u0430\u0433\u043d\u043e\u0437\u0430 \u041c\u0414\u0414 \u0438 \u0434\u0435\u043b\u0435\u0446\u0438\u044f \u0438\u0437\u0432\u044a\u043d \u0440\u0430\u043c\u043a\u0430\u0442\u0430, \u043f\u043e\u0434\u0430\u0442\u043b\u0438\u0432 \u043d\u0430:\n\u2022 \u041f\u0440\u0435\u0441\u043a\u0430\u0447\u0430\u043d\u0435 \u043d\u0430 \u0435\u043a\u0437\u043e\u043d 45 (\u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e, \u043d\u043e \u043d\u0435 \u0441\u0430\u043c\u043e, \u0434\u0435\u043b\u0435\u0446\u0438\u0438 \u043d\u0430 \u0435\u043a\u0437\u043e\u043d\u0438 \u043a\u0430\u0442\u043e 12-44, 18-44, 44, 46-47, 46-48, 46-49, 46-51, 46-53 \u0438\u043b\u0438 46-55) \u0418\u041b\u0418\n\u2022 \u041f\u0440\u0435\u0441\u043a\u0430\u0447\u0430\u043d\u0435 \u043d\u0430 \u0435\u043a\u0437\u043e\u043d 53 (\u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e, \u043d\u043e \u043d\u0435 \u0441\u0430\u043c\u043e, \u0434\u0435\u043b\u0435\u0446\u0438\u0438 \u043d\u0430 \u0435\u043a\u0437\u043e\u043d\u0438 \u043a\u0430\u0442\u043e 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52 \u0438\u043b\u0438 54-58)\n\u0414\u043e\u043a\u0443\u043c\u0435\u043d\u0442\u0438\u0440\u0430\u043d\u043e \u043f\u0440\u0435\u0434\u0438 \u0441\u043a\u0440\u0438\u043d\u0438\u043d\u0433\u0430 \u0447\u0440\u0435\u0437 \u0433\u0435\u043d\u0435\u0442\u0438\u0447\u0435\u043d \u0434\u043e\u043a\u043b\u0430\u0434 \u043e\u0442 \u0430\u043a\u0440\u0435\u0434\u0438\u0442\u0438\u0440\u0430\u043d\u0430 \u043b\u0430\u0431\u043e\u0440\u0430\u0442\u043e\u0440\u0438\u044f, \u0434\u0435\u0444\u0438\u043d\u0438\u0440\u0430\u0449 \u043a\u0440\u0430\u0439\u043d\u0438\u0442\u0435 \u0442\u043e\u0447\u043a\u0438 \u043d\u0430 \u0434\u0435\u043b\u0435\u0446\u0438\u044f \u0447\u0440\u0435\u0437 \u043c\u0443\u043b\u0442\u0438\u043f\u043b\u0435\u043a\u0441\u043d\u0430 \u043b\u0438\u0433\u0430\u0437\u043d\u043e \u0437\u0430\u0432\u0438\u0441\u0438\u043c\u0430 \u0430\u043c\u043f\u043b\u0438\u0444\u0438\u043a\u0430\u0446\u0438\u044f \u0438\u043b\u0438 \u0441\u0435\u043a\u0432\u0435\u043d\u0438\u0440\u0430\u043d\u0435 \u043d\u0430 \u043f\u0440\u043e\u0431\u0438. \u041f\u043e\u0434\u0430\u0442\u043b\u0438\u0432\u043e\u0441\u0442\u0442\u0430 \u043d\u0430 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u0430 \u043a\u044a\u043c \u043f\u0440\u0435\u0441\u043a\u0430\u0447\u0430\u043d\u0435 \u043d\u0430 \u0435\u043a\u0437\u043e\u043d 45 \u0438\u043b\u0438 \u0435\u043a\u0437\u043e\u043d 53 \u0442\u0440\u044f\u0431\u0432\u0430 \u0434\u0430 \u0431\u044a\u0434\u0435 \u043f\u043e\u0442\u0432\u044a\u0440\u0434\u0435\u043d\u0430 \u043f\u0440\u0435\u0434\u0438 \u043f\u044a\u0440\u0432\u0430\u0442\u0430 \u0434\u043e\u0437\u0430 \u0441 \u043f\u043e\u043c\u043e\u0449\u0442\u0430 \u043d\u0430 \u043f\u043e\u043b\u0443\u0447\u0435\u043d\u0438\u0442\u0435 \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u0441\u043a\u0440\u0438\u043d\u0438\u043d\u0433\u0430 \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442\u0438 \u0437\u0430 \u043e\u043f\u0440\u0435\u0434\u0435\u043b\u044f\u043d\u0435 \u043d\u0430 \u0433\u0435\u043d\u043e\u0442\u0438\u043f\u0430", "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": "K\u00e9sz a t\u00e1j\u00e9kozott hozz\u00e1j\u00e1rul\u00e1sra (ha sz\u00fcks\u00e9ges), \u00e9s a sz\u00fcl\u0151(k) vagy a t\u00f6rv\u00e9nyes gondvisel\u0151(k) k\u00e9sz(ek) \u00edr\u00e1sos, t\u00e1j\u00e9kozott beleegyez\u00e9s\u00fcket adni a beteg vizsg\u00e1latban val\u00f3 r\u00e9szv\u00e9tel\u00e9hez.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861428, "uuid": "bbfea091-2093-4e8a-860b-3692b1ac4516", "attributeTranslation": "pacjent jest gotowy na wyra\u017cenie pisemnej zgody na udzia\u0142 (formularz \u015bwiadomej zgody dla pacjenta ma\u0142oletniego) (je\u015bli dotyczy) oraz jego rodzic lub opiekun prawny jest gotowy wyrazi\u0107 pisemn\u0105 zgod\u0119 na jego udzia\u0142 (formularz \u015bwiadomej zgody).", "language": 19, "languageDescription": "Polish"}, {"id": 6861429, "uuid": "bbfea091-2093-4e8a-860b-3692b1ac4516", "attributeTranslation": "\u0416\u0435\u043b\u0430\u0435 \u0434\u0430 \u0434\u0430\u0434\u0435 \u0438\u043d\u0444\u043e\u0440\u043c\u0438\u0440\u0430\u043d\u043e \u043e\u0434\u043e\u0431\u0440\u0435\u043d\u0438\u0435 (\u0430\u043a\u043e \u0435 \u043f\u0440\u0438\u043b\u043e\u0436\u0438\u043c\u043e) \u0438 \u0438\u043c\u0430 \u0440\u043e\u0434\u0438\u0442\u0435\u043b(\u0438) \u0438\u043b\u0438 \u0437\u0430\u043a\u043e\u043d\u0435\u043d(\u043d\u0438) \u043d\u0430\u0441\u0442\u043e\u0439\u043d\u0438\u043a(\u0446\u0438), \u043a\u043e\u0439\u0442\u043e(\u043a\u043e\u0438\u0442\u043e) \u043c\u043e\u0436\u0435(\u043c\u043e\u0433\u0430\u0442) \u0434\u0430 \u0434\u0430\u0434\u0435(\u0430\u0442) \u043f\u0438\u0441\u043c\u0435\u043d\u043e \u0438\u043d\u0444\u043e\u0440\u043c\u0438\u0440\u0430\u043d\u043e \u0441\u044a\u0433\u043b\u0430\u0441\u0438\u0435 \u0437\u0430 \u0443\u0447\u0430\u0441\u0442\u0438\u0435\u0442\u043e \u043d\u0430 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u0430 \u0432 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e.", "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 aleatorizaci\u00f3n al grupo de pacientes susceptibles de la omisi\u00f3n del ex\u00f3n 53, tener entre 6 y 13 a\u00f1os de edad (incluidos); para la aleatorizaci\u00f3n al grupo de pacientes susceptibles de la omisi\u00f3n del ex\u00f3n 45, tener entre 7 y 13 a\u00f1os de edad (incluidos).", "language": 7, "languageDescription": "Spanish"}, {"id": 6861432, "uuid": "ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6b", "attributeTranslation": "Az \u00e9letkor \u22656 \u00e9s \u226413 \u00e9v k\u00f6z\u00f6tti randomiz\u00e1l\u00e1skor azokn\u00e1l a betegekn\u00e9l, akikn\u00e9l a betegs\u00e9g az 53. exon \u00e1tugr\u00e1s\u00e1val befoly\u00e1solhat\u00f3, vagy az \u00e9letkor \u22657 \u00e9s \u226413 \u00e9v k\u00f6z\u00f6tti randomiz\u00e1l\u00e1skor azokn\u00e1l a betegekn\u00e9l, akikn\u00e9l a betegs\u00e9g a 45. exon \u00e1tugr\u00e1s\u00e1val befoly\u00e1solhat\u00f3.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861434, "uuid": "ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6b", "attributeTranslation": "w momencie randomizacji pacjent musi by\u0107 w wieku od 6 do 13 lat w przypadku pacjent\u00f3w podatnych na pomini\u0119cie egzonu 53 lub w momencie randomizacji pacjent musi by\u0107 w wieku od 7 do 13 lat w przypadku pacjent\u00f3w podatnych na pomini\u0119cie egzonu 45.", "language": 19, "languageDescription": "Polish"}, {"id": 6861435, "uuid": "ca0c8beb-629e-4c56-aa68-6c3c7e5d1b6b", "attributeTranslation": "\u0415 \u043d\u0430 \u0432\u044a\u0437\u0440\u0430\u0441\u0442 \u043c\u0435\u0436\u0434\u0443 6 \u0438 13 \u0433\u043e\u0434\u0438\u043d\u0438 \u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u043a\u044a\u043c \u043c\u043e\u043c\u0435\u043d\u0442\u0430 \u043d\u0430 \u0440\u0430\u043d\u0434\u043e\u043c\u0438\u0437\u0430\u0446\u0438\u044f\u0442\u0430, \u0437\u0430 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u0438, \u043f\u043e\u0434\u0430\u0442\u043b\u0438\u0432\u0438 \u043d\u0430 \u043f\u0440\u0435\u0441\u043a\u0430\u0447\u0430\u043d\u0435 \u043d\u0430 \u0435\u043a\u0437\u043e\u043d 53; \u0438\u043b\u0438 \u0435 \u043d\u0430 \u0432\u044a\u0437\u0440\u0430\u0441\u0442 \u043c\u0435\u0436\u0434\u0443 7 \u0438 13 \u0433\u043e\u0434\u0438\u043d\u0438 \u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u043a\u044a\u043c \u043c\u043e\u043c\u0435\u043d\u0442\u0430 \u043d\u0430 \u0440\u0430\u043d\u0434\u043e\u043c\u0438\u0437\u0430\u0446\u0438\u044f\u0442\u0430, \u0437\u0430 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u0438, \u043f\u043e\u0434\u0430\u0442\u043b\u0438\u0432\u0438 \u043d\u0430 \u043f\u0440\u0435\u0441\u043a\u0430\u0447\u0430\u043d\u0435 \u043d\u0430 \u0435\u043a\u0437\u043e\u043d 45.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 584535, "number": 3, "principalInclusionCriteria": "Has stable pulmonary function (FVC % of predicted \u226550% 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": "czynno\u015bci oddechowe musz\u0105 by\u0107 stabilne (odsetek warto\u015bci nale\u017cnej FVC \u226550% i nie wymaga wentylacji mechanicznej w nocy), kt\u00f3re w opinii badacza nie ulegn\u0105 dekompensacji w czasie badania.", "language": 19, "languageDescription": "Polish"}, {"id": 6861437, "uuid": "291b79a5-d06c-4f8e-885a-04acdafb0858", "attributeTranslation": "L\u00e9gz\u00e9sfunkci\u00f3ja stabil (a v\u00e1rt \u00e9rt\u00e9k sz\u00e1zal\u00e9k\u00e1ban megadott FVC \u2265 50% \u00e9s \u00e9jszakai l\u00e9legeztet\u00e9s nem sz\u00fcks\u00e9ges), \u00e9s a vizsg\u00e1l\u00f3 v\u00e9lem\u00e9nye szerint val\u00f3sz\u00edn\u0171leg nem fog dekompenz\u00e1l\u00f3dni a vizsg\u00e1lat id\u0151tartama alatt.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861438, "uuid": "291b79a5-d06c-4f8e-885a-04acdafb0858", "attributeTranslation": "\u0418\u043c\u0430 \u0441\u0442\u0430\u0431\u0438\u043b\u043d\u0430 \u0431\u0435\u043b\u043e\u0434\u0440\u043e\u0431\u043d\u0430 \u0444\u0443\u043d\u043a\u0446\u0438\u044f (\u043f\u0440\u0435\u0434\u0432\u0438\u0434\u0435\u043d % FVC \u226550% \u0438 \u0431\u0435\u0437 \u043d\u0435\u043e\u0431\u0445\u043e\u0434\u0438\u043c\u043e\u0441\u0442 \u043e\u0442 \u043d\u043e\u0449\u043d\u0430 \u0432\u0435\u043d\u0442\u0438\u043b\u0430\u0446\u0438\u044f), \u043a\u043e\u044f\u0442\u043e, \u043f\u043e \u043f\u0440\u0435\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u0438\u0437\u0441\u043b\u0435\u0434\u043e\u0432\u0430\u0442\u0435\u043b\u044f, \u043d\u044f\u043c\u0430 \u0434\u0430 \u0441\u0435 \u0434\u0435\u043a\u043e\u043c\u043f\u0435\u043d\u0441\u0438\u0440\u0430 \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861436, "uuid": "291b79a5-d06c-4f8e-885a-04acdafb0858", "attributeTranslation": "Presentar una funci\u00f3n pulmonar estable (% CVF \u226550 % de la prevista, y sin necesidad de ventilaci\u00f3n mec\u00e1nica nocturna) que, seg\u00fan la opini\u00f3n 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 \u00e9s bal musculus biceps brachii (a prefer\u00e1lt biopszi\u00e1s hely) vagy 2 m\u00e1sik felkari izomcsoport intakt.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861442, "uuid": "583cb2b1-0b11-4e81-a6f4-c859399aa25c", "attributeTranslation": "\u0418\u043c\u0430 \u043d\u0435\u043f\u043e\u043a\u044a\u0442\u043d\u0430\u0442\u0438 \u0434\u0435\u0441\u0435\u043d \u0438 \u043b\u044f\u0432 \u0434\u0432\u0443\u0433\u043b\u0430\u0432 \u043c\u0438\u0448\u043d\u0438\u0447\u0435\u043d \u043c\u0443\u0441\u043a\u0443\u043b (\u043f\u0440\u0435\u0434\u043f\u043e\u0447\u0438\u0442\u0430\u043d\u043e \u043c\u044f\u0441\u0442\u043e \u0437\u0430 \u0431\u0438\u043e\u043f\u0441\u0438\u044f) \u0438\u043b\u0438 2 \u0430\u043b\u0442\u0435\u0440\u043d\u0430\u0442\u0438\u0432\u043d\u0438 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0438 \u0433\u0440\u0443\u043f\u0438 \u0432 \u0433\u043e\u0440\u043d\u0430\u0442\u0430 \u0447\u0430\u0441\u0442 \u043d\u0430 \u0440\u044a\u043a\u0430\u0442\u0430.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861443, "uuid": "583cb2b1-0b11-4e81-a6f4-c859399aa25c", "attributeTranslation": "Presentar los m\u00fasculos b\u00edceps braquiales izquierdo y derecho (zona preferida para la biopsia) o 2 grupos de m\u00fasculos del brazo alternativos intactos.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861441, "uuid": "583cb2b1-0b11-4e81-a6f4-c859399aa25c", "attributeTranslation": "musi mie\u0107 nienaruszony prawy i lewy mi\u0119sie\u0144 dwug\u0142owy ramienia (preferowane miejsce biopsji) lub dwie inne grupy mi\u0119\u015bniowe w obr\u0119bie ko\u0144czyny g\u00f3rnej.", "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": "V\u00e1ltozatlan d\u00f3zisban vagy ekvivalens d\u00f3zisban or\u00e1lis kortikoszteroidot szedett az 1. hetet megel\u0151z\u0151 legal\u00e1bb 24 h\u00e9ten \u00e1t, \u00e9s a d\u00f3zis v\u00e1rhat\u00f3an v\u00e1ltozatlan marad a vizsg\u00e1lat alatt (a testt\u00f6meg v\u00e1ltoz\u00e1sa miatt sz\u00fcks\u00e9ges m\u00f3dos\u00edt\u00e1sokat kiv\u00e9ve)", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861447, "uuid": "13e66846-08a7-4309-b608-5174d5d186cf", "attributeTranslation": "\u0415 \u043d\u0430 \u0441\u0442\u0430\u0431\u0438\u043b\u043d\u0430 \u0434\u043e\u0437\u0430 \u0438\u043b\u0438 \u0434\u043e\u0437\u043e\u0432 \u0435\u043a\u0432\u0438\u0432\u0430\u043b\u0435\u043d\u0442 \u043f\u0435\u0440\u043e\u0440\u0430\u043b\u043d\u0438 \u043a\u043e\u0440\u0442\u0438\u043a\u043e\u0441\u0442\u0435\u0440\u043e\u0438\u0434\u0438 \u0432 \u043f\u0440\u043e\u0434\u044a\u043b\u0436\u0435\u043d\u0438\u0435 \u043d\u0430 \u043d\u0430\u0439-\u043c\u0430\u043b\u043a\u043e 24 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1 \u0438 \u0441\u0435 \u043e\u0447\u0430\u043a\u0432\u0430 \u0434\u043e\u0437\u0430\u0442\u0430 \u0434\u0430 \u043e\u0441\u0442\u0430\u043d\u0435 \u043d\u0435\u043f\u0440\u043e\u043c\u0435\u043d\u0435\u043d\u0430 \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e (\u0441 \u0438\u0437\u043a\u043b\u044e\u0447\u0435\u043d\u0438\u0435 \u043d\u0430 \u043c\u043e\u0434\u0438\u0444\u0438\u043a\u0430\u0446\u0438\u0438, \u043e\u0442\u0440\u0430\u0437\u044f\u0432\u0430\u0449\u0438 \u043f\u0440\u043e\u043c\u0435\u043d\u0438\u0442\u0435 \u0432 \u0442\u0435\u0433\u043b\u043e\u0442\u043e)", "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": "otrzymywa\u0142 sta\u0142\u0105 dawk\u0119 lub ekwiwalent dawki doustnych kortykosteroid\u00f3w przez przynajmniej 24 tygodnie przed 1. tygodniem i przewiduje si\u0119, \u017ce dawka pozostanie niezmieniona w czasie badania (poza modyfikacjami uwzgl\u0119dniaj\u0105cymi zmian\u0119 masy cia\u0142a.", "language": 19, "languageDescription": "Polish"}]}, {"id": 584538, "number": 6, "principalInclusionCriteria": "If taking angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), \u03b2 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": "je\u017celi pacjent przyjmuje inhibitory enzymu konwertuj\u0105cego angiotensyn\u0119 (ACE), czynniki blokuj\u0105ce receptor dla angiotensyny (ARBs, ang. angiotensin receptor blocking agents), blokery \u03b2 adrenergiczne, antagonist\u00f3w receptora aldosteronu, potas lub koenzym Q, przyjmowa\u0142 sta\u0142\u0105 dawk\u0119 przez co najmniej 12 tygodni przed 1. tygodniem i przewiduje si\u0119, \u017ce dawka pozostanie niezmieniona w czasie badania (poza modyfikacjami uwzgl\u0119dniaj\u0105cymi zmian\u0119 masy cia\u0142a).", "language": 19, "languageDescription": "Polish"}, {"id": 6861451, "uuid": "eb7a5126-13fb-4264-8b8c-686809e6d362", "attributeTranslation": "Ha angiotenzin-konvert\u00e1l\u00f3 enzim (ACE) inhibitorokat, angiotenzin receptor blokkol\u00f3 gy\u00f3gyszereket (ARB), \u03b2-adrenerg blokkol\u00f3kat, aldoszteron-receptor antagonist\u00e1kat, k\u00e1liumot vagy koenzim Q-t szed, ezeket v\u00e1ltozatlan d\u00f3zisban kellett kapnia az 1. hetet megel\u0151z\u0151 legal\u00e1bb 12 h\u00e9ten \u00e1t, \u00e9s a d\u00f3zisnak v\u00e1rhat\u00f3an v\u00e1ltozatlannak kell maradnia a vizsg\u00e1lat alatt (a testt\u00f6meg v\u00e1ltoz\u00e1sa miatt sz\u00fcks\u00e9ges m\u00f3dos\u00edt\u00e1sokat kiv\u00e9ve).", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861448, "uuid": "eb7a5126-13fb-4264-8b8c-686809e6d362", "attributeTranslation": "\u0410\u043a\u043e \u043f\u0440\u0438\u0435\u043c\u0430 \u0438\u043d\u0445\u0438\u0431\u0438\u0442\u043e\u0440\u0438 \u043d\u0430 \u0430\u043d\u0433\u0438\u043e\u0442\u0435\u043d\u0437\u0438\u043d \u043a\u043e\u043d\u0432\u0435\u0440\u0442\u0438\u0440\u0430\u0449\u0438\u044f \u0435\u043d\u0437\u0438\u043c (ACE), \u0430\u043d\u0433\u0438\u043e\u0442\u0435\u043d\u0437\u0438\u043d \u0440\u0435\u0446\u0435\u043f\u0442\u043e\u0440\u043d\u0438 \u0431\u043b\u043e\u043a\u0435\u0440\u0438 (ARB), \u03b2-\u0430\u0434\u0440\u0435\u043d\u0435\u0440\u0433\u0438\u0447\u043d\u0438 \u0431\u043b\u043e\u043a\u0435\u0440\u0438, \u0430\u043b\u0434\u043e\u0441\u0442\u0435\u0440\u043e\u043d \u0440\u0435\u0446\u0435\u043f\u0442\u043e\u0440\u043d\u0438 \u0430\u043d\u0442\u0430\u0433\u043e\u043d\u0438\u0441\u0442\u0438, \u043a\u0430\u043b\u0438\u0439 \u0438\u043b\u0438 \u043a\u043e\u0435\u043d\u0437\u0438\u043c Q, \u0435 \u043d\u0430 \u0441\u0442\u0430\u0431\u0438\u043b\u043d\u0430 \u0434\u043e\u0437\u0430 \u0432 \u043f\u0440\u043e\u0434\u044a\u043b\u0436\u0435\u043d\u0438\u0435 \u043d\u0430 \u043d\u0430\u0439-\u043c\u0430\u043b\u043a\u043e 12 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1 \u0438 \u0441\u0435 \u043e\u0447\u0430\u043a\u0432\u0430 \u0434\u043e\u0437\u0430\u0442\u0430 \u0434\u0430 \u043e\u0441\u0442\u0430\u043d\u0435 \u043d\u0435\u043f\u0440\u043e\u043c\u0435\u043d\u0435\u043d\u0430 \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e (\u0441 \u0438\u0437\u043a\u043b\u044e\u0447\u0435\u043d\u0438\u0435 \u043d\u0430 \u043c\u043e\u0434\u0438\u0444\u0438\u043a\u0430\u0446\u0438\u0438, \u043e\u0442\u0440\u0430\u0437\u044f\u0432\u0430\u0449\u0438 \u043f\u0440\u043e\u043c\u0435\u043d\u0438\u0442\u0435 \u0432 \u0442\u0435\u0433\u043b\u043e\u0442\u043e).", "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 adren\u00e9rgicos \u03b2, antialdoster\u00f3nicos, 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  \u2265300 to \u2264 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 \u2265300 y \u2264450 metros (sin ayuda) tanto en la visita de selecci\u00f3n como en la visita basal (antes de la semana 1). La distancia media de la PM6M en las visitas de selecci\u00f3n y basal es el promedio de 2 evaluaciones independientes llevadas a cabo en 2 d\u00edas laborables consecutivos para cada visita. La distancia media basal (promedio de los d\u00edas 1 y 2 de la visita basal) debe encontrarse dentro del 15 % de la distancia media de la selecci\u00f3n (promedio de los d\u00edas 1 y 2 de la visita de selecci\u00f3n).", "language": 7, "languageDescription": "Spanish"}, {"id": 6861453, "uuid": "5e7b81eb-9aa2-4d41-acbe-8770455b0f4e", "attributeTranslation": "Az \u00e1tlagos 6MWT t\u00e1vols\u00e1g a sz\u0171r\u0151 \u00e9s a kiindul\u00e1si viziteken (az 1. h\u00e9t el\u0151tt) egyar\u00e1nt \u2265300 \u2013 \u2264450 m\u00e9ter (seg\u00edts\u00e9g n\u00e9lk\u00fcl). A sz\u0171r\u0151 \u00e9s a kiindul\u00e1si viziteken m\u00e9rt \u00e1tlagos 6MWT t\u00e1vols\u00e1g mindegyik vizit eset\u00e9n 2 egym\u00e1st k\u00f6vet\u0151 munkanapon v\u00e9gzett 2 k\u00fcl\u00f6n \u00e9rt\u00e9kel\u00e9s \u00e1tlaga. A kiindul\u00e1si \u00e1tlagnak (az 1. \u00e9s 2. kiindul\u00e1si nap \u00e1tlaga) a sz\u0171r\u00e9si \u00e1tlagos t\u00e1vols\u00e1g (az 1. \u00e9s 2. sz\u0171r\u00e9si nap \u00e1tlaga) 15%-\u00e1n bel\u00fclinek kell lennie.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861454, "uuid": "5e7b81eb-9aa2-4d41-acbe-8770455b0f4e", "attributeTranslation": "pacjent w te\u015bcie 6MWT uzyska\u0142 \u015bredni dystans od \u2265300 do \u2264450 metr\u00f3w (bez pomocy) zar\u00f3wno w czasie bada\u0144 przesiewowych, jak i w czasie wizyty pocz\u0105tkowej (przed 1. tygodniem). \u015aredni dystans w te\u015bcie 6MWT w czasie bada\u0144 przesiewowych i w czasie wizyty pocz\u0105tkowej jest \u015bredni\u0105 z 2 niezale\u017cnych bada\u0144 w 2 kolejnych dniach roboczych dla ka\u017cdej wizyty. \u015arednia warto\u015b\u0107 pocz\u0105tkowa (\u015brednia dla pocz\u0105tkowych dnia 1 i 2) musi mie\u015bci\u0107 si\u0119 w 15% \u015bredniej warto\u015bci dystansu z bada\u0144 przesiewowych (\u015brednia dni 1 i 2 bada\u0144 przesiewowych).", "language": 19, "languageDescription": "Polish"}, {"id": 6861455, "uuid": "5e7b81eb-9aa2-4d41-acbe-8770455b0f4e", "attributeTranslation": "\u041f\u0440\u0438 6MWT \u0435 \u0434\u043e\u0441\u0442\u0438\u0433\u043d\u0430\u043b \u0441\u0440\u0435\u0434\u043d\u043e \u0440\u0430\u0437\u0441\u0442\u043e\u044f\u043d\u0438\u0435 \u043e\u0442 \u2265300 \u0434\u043e \u2264 450 \u043c\u0435\u0442\u0440\u0430 (\u0431\u0435\u0437 \u043f\u043e\u043c\u043e\u0449) \u043a\u0430\u043a\u0442\u043e \u043d\u0430 \u0441\u043a\u0440\u0438\u043d\u0438\u043d\u0433\u043e\u0432\u0430\u0442\u0430 \u0432\u0438\u0437\u0438\u0442\u0430, \u0442\u0430\u043a\u0430 \u0438 \u043d\u0430 \u0432\u0438\u0437\u0438\u0442\u0430\u0442\u0430 \u0437\u0430 \u0438\u0437\u0445\u043e\u0434\u043d\u043e \u043d\u0438\u0432\u043e (\u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1). \u0421\u0440\u0435\u0434\u043d\u043e\u0442\u043e \u0440\u0430\u0437\u0441\u0442\u043e\u044f\u043d\u0438\u0435 \u043f\u0440\u0438 6MWT \u043d\u0430 \u0441\u043a\u0440\u0438\u043d\u0438\u043d\u0433\u043e\u0432\u0430\u0442\u0430 \u0432\u0438\u0437\u0438\u0442\u0430 \u0438 \u0432\u0438\u0437\u0438\u0442\u0430\u0442\u0430 \u0437\u0430 \u0438\u0437\u0445\u043e\u0434\u043d\u043e \u043d\u0438\u0432\u043e \u0435 \u0441\u0440\u0435\u0434\u043d\u0430\u0442\u0430 \u0441\u0442\u043e\u0439\u043d\u043e\u0441\u0442 \u043e\u0442 2 \u043e\u0442\u0434\u0435\u043b\u043d\u0438 \u043e\u0446\u0435\u043d\u043a\u0438, \u0438\u0437\u0432\u044a\u0440\u0448\u0435\u043d\u0438 \u0432 2 \u043f\u043e\u0441\u043b\u0435\u0434\u043e\u0432\u0430\u0442\u0435\u043b\u043d\u0438 \u0440\u0430\u0431\u043e\u0442\u043d\u0438 \u0434\u043d\u0438 \u043f\u0440\u0438 \u0432\u0441\u044f\u043a\u0430 \u0432\u0438\u0437\u0438\u0442\u0430. \u0421\u0440\u0435\u0434\u043d\u043e\u0442\u043e \u0440\u0430\u0437\u0441\u0442\u043e\u044f\u043d\u0438\u0435 \u043e\u0442 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e (\u0441\u0440\u0435\u0434\u043d\u043e \u0437\u0430 \u0434\u043d\u0438 1 \u0438 2 \u043e\u0442 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e) \u0442\u0440\u044f\u0431\u0432\u0430 \u0434\u0430 \u0431\u044a\u0434\u0435 \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 15% \u043e\u0442 \u0441\u0440\u0435\u0434\u043d\u043e\u0442\u043e \u0440\u0430\u0437\u0441\u0442\u043e\u044f\u043d\u0438\u0435 \u043e\u0442 \u0441\u043a\u0440\u0438\u043d\u0438\u043d\u0433\u0430 (\u0441\u0440\u0435\u0434\u043d\u043e \u0437\u0430 \u0434\u043d\u0438 1 \u0438 2 \u043e\u0442 \u0441\u043a\u0440\u0438\u043d\u0438\u043d\u0433\u0430).", "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 d\u00edas posteriores a la \u00faltima dosis del f\u00e1rmaco del estudio. \nLa pareja sexual del paciente participante tambi\u00e9n debe utilizar un m\u00e9todo anticonceptivo aceptable desde el punto de vista m\u00e9dico (p. ej., anticonceptivos orales) durante este mismo periodo. Entre los m\u00e9todos anticonceptivos aceptables, se incluyen los anticonceptivos hormonales combinados (con estr\u00f3genos y progesterona) de inhibici\u00f3n de la ovulaci\u00f3n (orales, intravaginales o transd\u00e9rmicos); anticonceptivos hormonales \u00fanicamente de progesterona de inhibici\u00f3n de la ovulaci\u00f3n (orales, inyectables o implantables); dispositivo intrauterino; sistema intrauterino liberador de un tratamiento anticonceptivo hormonal; oclusi\u00f3n tub\u00e1rica bilateral; pareja vasectomizada; abstinencia sexual (abstinencia real, siempre que este sea el estilo de vida usual y preferido del sujeto; la abstinencia peri\u00f3dica, como la que se da en los m\u00e9todos del calendario, de la ovulaci\u00f3n, sintot\u00e9rmico y de la posovulaci\u00f3n, as\u00ed como el coito interrumpido, no son m\u00e9todos anticonceptivos aceptables) o preservativo con capuch\u00f3n, diafragma o esponja con espermicida (m\u00e9todos de doble barrera).", "language": 7, "languageDescription": "Spanish"}, {"id": 6861459, "uuid": "b61c3bdd-62d9-4ac0-b4b8-9833ca450d8b", "attributeTranslation": "\u0410\u043a\u043e \u0435 \u0441\u0435\u043a\u0441\u0443\u0430\u043b\u043d\u043e \u0430\u043a\u0442\u0438\u0432\u0435\u043d, \u0435 \u0441\u044a\u0433\u043b\u0430\u0441\u0435\u043d \u0434\u0430 \u0438\u0437\u043f\u043e\u043b\u0437\u0432\u0430 \u043c\u044a\u0436\u043a\u0438 \u043f\u0440\u0435\u0437\u0435\u0440\u0432\u0430\u0442\u0438\u0432 \u043f\u0440\u0438 \u043f\u043e\u043b\u043e\u0432\u0438 \u043a\u043e\u043d\u0442\u0430\u043a\u0442\u0438 \u043f\u0440\u0435\u0437 \u0446\u044f\u043b\u0430\u0442\u0430 \u043f\u0440\u043e\u0434\u044a\u043b\u0436\u0438\u0442\u0435\u043b\u043d\u043e\u0441\u0442 \u043d\u0430 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e \u0438 \u0432 \u043f\u0440\u043e\u0434\u044a\u043b\u0436\u0435\u043d\u0438\u0435 \u043d\u0430 90 \u0434\u043d\u0438 \u0441\u043b\u0435\u0434 \u043f\u043e\u0441\u043b\u0435\u0434\u043d\u0430\u0442\u0430 \u0434\u043e\u0437\u0430. \n\u0421\u0435\u043a\u0441\u0443\u0430\u043b\u043d\u0430\u0442\u0430 \u043f\u0430\u0440\u0442\u043d\u044c\u043e\u0440\u043a\u0430 \u0441\u044a\u0449\u043e \u0442\u0440\u044f\u0431\u0432\u0430 \u0434\u0430 \u0438\u0437\u043f\u043e\u043b\u0437\u0432\u0430 \u043f\u0440\u0438\u0435\u043c\u043b\u0438\u0432\u0430 \u043e\u0442 \u043c\u0435\u0434\u0438\u0446\u0438\u043d\u0441\u043a\u0430 \u0433\u043b\u0435\u0434\u043d\u0430 \u0442\u043e\u0447\u043a\u0430 \u0444\u043e\u0440\u043c\u0430 \u043d\u0430 \u043a\u043e\u043d\u0442\u0440\u0430\u0446\u0435\u043f\u0446\u0438\u044f (\u043d\u0430\u043f\u0440. \u0436\u0435\u043d\u0441\u043a\u0438 \u043f\u0435\u0440\u043e\u0440\u0430\u043b\u043d\u0438 \u043a\u043e\u043d\u0442\u0440\u0430\u0446\u0435\u043f\u0442\u0438\u0432\u0438) \u043f\u0440\u0435\u0437 \u0442\u043e\u0437\u0438 \u043f\u0435\u0440\u0438\u043e\u0434. \u041f\u0440\u0438\u0435\u043c\u043b\u0438\u0432\u0438\u0442\u0435 \u043c\u0435\u0442\u043e\u0434\u0438 \u0437\u0430 \u043a\u043e\u043d\u0442\u0440\u0430\u0446\u0435\u043f\u0446\u0438\u044f \u0432\u043a\u043b\u044e\u0447\u0432\u0430\u0442 \u043a\u043e\u043c\u0431\u0438\u043d\u0438\u0440\u0430\u043d\u0430 (\u0441\u044a\u0434\u044a\u0440\u0436\u0430\u0449\u0430 \u0435\u0441\u0442\u0440\u043e\u0433\u0435\u043d \u0438 \u043f\u0440\u043e\u0433\u0435\u0441\u0442\u0435\u0440\u043e\u043d) \u0445\u043e\u0440\u043c\u043e\u043d\u0430\u043b\u043d\u0430 \u043a\u043e\u043d\u0442\u0440\u0430\u0446\u0435\u043f\u0446\u0438\u044f, \u0441\u0432\u044a\u0440\u0437\u0430\u043d\u0430 \u0441 \u0438\u043d\u0445\u0438\u0431\u0438\u0440\u0430\u043d\u0435 \u043d\u0430 \u043e\u0432\u0443\u043b\u0430\u0446\u0438\u044f\u0442\u0430 (\u043f\u0435\u0440\u043e\u0440\u0430\u043b\u043d\u0430, \u0438\u043d\u0442\u0440\u0430\u0432\u0430\u0433\u0438\u043d\u0430\u043b\u043d\u0430 \u0438\u043b\u0438 \u0442\u0440\u0430\u043d\u0441\u0434\u0435\u0440\u043c\u0430\u043b\u043d\u0430); \u0441\u044a\u0434\u044a\u0440\u0436\u0430\u0449\u0430 \u0441\u0430\u043c\u043e \u043f\u0440\u043e\u0433\u0435\u0441\u0442\u0435\u0440\u043e\u043d \u043a\u043e\u043d\u0442\u0440\u0430\u0446\u0435\u043f\u0446\u0438\u044f, \u0441\u0432\u044a\u0440\u0437\u0430\u043d\u0430 \u0441 \u0438\u043d\u0445\u0438\u0431\u0438\u0440\u0430\u043d\u0435 \u043d\u0430 \u043e\u0432\u0443\u043b\u0430\u0446\u0438\u044f\u0442\u0430 (\u043f\u0435\u0440\u043e\u0440\u0430\u043b\u043d\u0430, \u0438\u043d\u0436\u0435\u043a\u0442\u0438\u0440\u0443\u0435\u043c\u0430 \u0438\u043b\u0438 \u0438\u043c\u043f\u043b\u0430\u043d\u0442\u0438\u0440\u0443\u0435\u043c\u0430); \u0432\u044a\u0442\u0440\u0435\u043c\u0430\u0442\u043e\u0447\u043d\u043e \u0443\u0441\u0442\u0440\u043e\u0439\u0441\u0442\u0432\u043e; \u0445\u043e\u0440\u043c\u043e\u043d-\u043e\u0441\u0432\u043e\u0431\u043e\u0436\u0434\u0430\u0432\u0430\u0449\u0430 \u0432\u044a\u0442\u0440\u0435\u043c\u0430\u0442\u043e\u0447\u043d\u0430 \u0441\u0438\u0441\u0442\u0435\u043c\u0430; \u0434\u0432\u0443\u0441\u0442\u0440\u0430\u043d\u043d\u0430 \u043e\u043a\u043b\u0443\u0437\u0438\u044f \u043d\u0430 \u0442\u0440\u044a\u0431\u0438\u0442\u0435, \u043f\u0440\u0435\u043c\u0438\u043d\u0430\u043b \u0432\u0430\u0437\u0435\u043a\u0442\u043e\u043c\u0438\u044f \u043f\u0430\u0440\u0442\u043d\u044c\u043e\u0440; \u0441\u0435\u043a\u0441\u0443\u0430\u043b\u043d\u043e \u0432\u044a\u0437\u0434\u044a\u0440\u0436\u0430\u043d\u0438\u0435 (\u0434\u0435\u0439\u0441\u0442\u0432\u0438\u0442\u0435\u043b\u043d\u043e \u0432\u044a\u0437\u0434\u044a\u0440\u0436\u0430\u043d\u0438\u0435: \u043a\u043e\u0433\u0430\u0442\u043e \u0442\u043e\u0432\u0430 \u0441\u044a\u043e\u0442\u0432\u0435\u0442\u0441\u0442\u0432\u0430 \u043d\u0430 \u043f\u0440\u0435\u0434\u043f\u043e\u0447\u0438\u0442\u0430\u043d\u0438\u044f \u0438 \u043e\u0431\u0438\u0447\u0430\u0435\u043d \u043d\u0430\u0447\u0438\u043d \u043d\u0430 \u0436\u0438\u0432\u043e\u0442 \u043d\u0430 \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u043a\u0430. \u041f\u0435\u0440\u0438\u043e\u0434\u0438\u0447\u043d\u043e \u0432\u044a\u0437\u0434\u044a\u0440\u0436\u0430\u043d\u0438\u0435: \u043d\u0430\u043f\u0440\u0438\u043c\u0435\u0440, \u043a\u0430\u043b\u0435\u043d\u0434\u0430\u0440\u043d\u0438, \u043e\u0432\u0443\u043b\u0430\u0446\u0438\u043e\u043d\u043d\u0438, \u0441\u0438\u043c\u043f\u0442\u043e\u0442\u0435\u0440\u043c\u0430\u043b\u043d\u0438 \u0438 \u043f\u043e\u0441\u0442-\u043e\u0432\u0443\u043b\u0430\u0446\u0438\u043e\u043d\u043d\u0438 \u043c\u0435\u0442\u043e\u0434\u0438, \u0438 \u043f\u0440\u0435\u0436\u0434\u0435\u0432\u0440\u0435\u043c\u0435\u043d\u043d\u043e \u043f\u0440\u0435\u043a\u0440\u0430\u0442\u044f\u0432\u0430\u043d\u0435 \u043d\u0430 \u043f\u043e\u043b\u043e\u0432\u0438\u044f \u0430\u043a\u0442 \u043d\u0435 \u0441\u0430 \u043f\u0440\u0438\u0435\u043c\u043b\u0438\u0432\u0438 \u043c\u0435\u0442\u043e\u0434\u0438 \u0437\u0430 \u043a\u043e\u043d\u0442\u0440\u0430\u0446\u0435\u043f\u0446\u0438\u044f); \u0438\u043b\u0438 \u043f\u0440\u0435\u0437\u0435\u0440\u0432\u0430\u0442\u0438\u0432 \u0432 \u043a\u043e\u043c\u0431\u0438\u043d\u0430\u0446\u0438\u044f \u0441 \u0448\u0430\u043f\u043a\u0430, \u0434\u0438\u0430\u0444\u0440\u0430\u0433\u043c\u0430 \u0438\u043b\u0438 \u0433\u044a\u0431\u0430 \u0441\u044a\u0441 \u0441\u043f\u0435\u0440\u043c\u0438\u0446\u0438\u0434 (\u0434\u0432\u043e\u0439\u043d\u043e-\u0431\u0430\u0440\u0438\u0435\u0440\u043d\u0430 \u043a\u043e\u043d\u0442\u0440\u0430\u0446\u0435\u043f\u0446\u0438\u044f).", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861457, "uuid": "b61c3bdd-62d9-4ac0-b4b8-9833ca450d8b", "attributeTranslation": "Ha akt\u00edv nemi \u00e9letet \u00e9l, bele kell egyeznie, hogy nemi kapcsolat eset\u00e9n a vizsg\u00e1lat teljes id\u0151tartama alatt \u00e9s az utols\u00f3 d\u00f3zist k\u00f6vet\u0151en 90 napig f\u00e9rfi \u00f3vszert haszn\u00e1l. \nA szexu\u00e1lis partnernek is orvosilag elfogadott fogamz\u00e1sg\u00e1tl\u00f3 m\u00f3dszert kell haszn\u00e1lnia (pl. n\u0151i or\u00e1lis fogamz\u00e1sg\u00e1tl\u00f3) ugyanezen id\u0151tartam alatt. Az elfogadhat\u00f3 fogamz\u00e1sg\u00e1tl\u00f3 m\u00f3dszerek k\u00f6z\u00e9 tartozik a kombin\u00e1lt (\u00f6sztrog\u00e9n \u00e9s progeszteron tartalm\u00fa) hormon\u00e1lis fogamz\u00e1sg\u00e1tl\u00e1s, ami a t\u00fcsz\u0151reped\u00e9s (ovul\u00e1ci\u00f3) g\u00e1tl\u00e1s\u00e1n alapszik (or\u00e1lis, intravagin\u00e1lis vagy transzderm\u00e1lis); a csak progeszteront tartalmaz\u00f3 hormon\u00e1lis fogamz\u00e1sg\u00e1tl\u00e1s, ami az ovul\u00e1ci\u00f3 g\u00e1tl\u00e1s\u00e1n alapszik (or\u00e1lis, injekci\u00f3s vagy implant\u00e1tum); m\u00e9hen bel\u00fcli eszk\u00f6z; m\u00e9hen bel\u00fcli hormonfelszabad\u00edt\u00f3 rendszer; k\u00e9toldali petevezet\u00e9k elk\u00f6t\u00e9s, vazekt\u00f3mi\u00e1n \u00e1tesett partner; tart\u00f3zkod\u00e1s a nemi k\u00f6z\u00f6s\u00fcl\u00e9st\u0151l (szexu\u00e1lis absztinencia) (t\u00e9nyleges absztinencia, ha ez a vizsg\u00e1lati alany \u00e1ltal el\u0151nyben r\u00e9szes\u00edtett \u00e9s megszokott \u00e9letm\u00f3ddal \u00f6sszhangban van. A nemi k\u00f6z\u00f6s\u00fcl\u00e9st\u0151l val\u00f3 id\u0151szakos tart\u00f3zkod\u00e1s, p\u00e9ld\u00e1ul a napt\u00e1r-m\u00f3dszer, az ovul\u00e1ci\u00f3s, szimptoterm\u00e1lis, posztovul\u00e1ci\u00f3s m\u00f3dszerek, tov\u00e1bb\u00e1 a megszak\u00edt\u00e1sos k\u00f6z\u00f6s\u00fcl\u00e9s nem elfogadhat\u00f3 fogamz\u00e1sg\u00e1tl\u00f3 m\u00f3dszerek); vagy \u00f3vszer m\u00e9hsapk\u00e1val, pessz\u00e1riummal vagy spermicides szivaccsal kombin\u00e1lva (kett\u0151s barrier-elv\u0171 fogamz\u00e1sg\u00e1tl\u00e1s).", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861456, "uuid": "b61c3bdd-62d9-4ac0-b4b8-9833ca450d8b", "attributeTranslation": "je\u017celi pacjent jest aktywny seksualnie, musi wyrazi\u0107 zgod\u0119 na stosowanie prezerwatywy w czasie kontakt\u00f3w seksualnych w ca\u0142ym okresie leczenia oraz do 90 dni od podania ostatniej dawki. \nPartnerka seksualna w tym okresie musi r\u00f3wnie\u017c stosowa\u0107 medycznie zatwierdzone \u015brodki antykoncepcyjne (np. doustna antykoncepcja dla kobiet). Do dopuszczalnych metod antykoncepcji nale\u017c\u0105: z\u0142o\u017cone (zawieraj\u0105ce estrogen i progesteron) hormonalne \u015brodki antykoncepcyjne hamuj\u0105ce owulacj\u0119 (doustne, dopochwowe lub przezsk\u00f3rne); hormonalne \u015brodki antykoncepcyjne hamuj\u0105ce owulacj\u0119, zawieraj\u0105ce wy\u0142\u0105cznie progesteron (doustne, wstrzykiwane lub wszczepialne); wk\u0142adka domaciczna; wk\u0142adka domaciczna uwalniaj\u0105ca hormony; obustronne zamkni\u0119cie jajowod\u00f3w; partner po wazektomii; abstynencja seksualna (pe\u0142na abstynencja, je\u015bli jest to zgodne z preferowanym i zwyk\u0142ym stylem \u017cycia pacjentki. Okresowa abstynencja np. metoda rytmu kalendarzowego, metody objawowo-termiczne [owulacyjne i poowulacyjne] oraz stosunek przerywany nie s\u0105 dopuszczalnymi metodami antykoncepcji); lub prezerwatywa w po\u0142\u0105czeniu z kapturkiem, diafragm\u0105 lub g\u0105bk\u0105 ze \u015brodkiem plemnikob\u00f3jczym (podw\u00f3jna 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, kt\u00f3ry jest w stanie zrozumie\u0107 wymogi badania i si\u0119 do nich stosowa\u0107.", "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 sz\u00fcl\u0151(k) vagy a t\u00f6rv\u00e9nyes gondvisel\u0151(k) k\u00e9pesek meg\u00e9rteni az \u00f6sszes vizsg\u00e1lati k\u00f6vetelm\u00e9nyt, \u00e9s egy\u00fcttm\u0171k\u00f6dni azok szerint.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861461, "uuid": "0065fe7e-e51d-414e-9a49-6e9f8e148ce6", "attributeTranslation": "\u0418\u043c\u0430 \u0440\u043e\u0434\u0438\u0442\u0435\u043b(\u0438) \u0438\u043b\u0438 \u0437\u0430\u043a\u043e\u043d\u0435\u043d(\u043d\u0438) \u043d\u0430\u0441\u0442\u043e\u0439\u043d\u0438\u043a(\u0446\u0438), \u043a\u043e\u0439\u0442\u043e(\u043a\u043e\u0438\u0442\u043e) \u043c\u043e\u0436\u0435(\u043c\u043e\u0433\u0430\u0442) \u0434\u0430 \u0440\u0430\u0437\u0431\u0435\u0440\u0435(\u0430\u0442) \u0438 \u0441\u043f\u0430\u0437\u0432\u0430(\u0442) \u0438\u0437\u0438\u0441\u043a\u0432\u0430\u043d\u0438\u044f\u0442\u0430 \u043d\u0430 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e.", "language": 1, "languageDescription": "Bulgarian"}]}], "principalExclusionCriteria": [{"id": 1005260, "number": 1, "principalExclusionCriteria": "Treatment with any of the following investigational therapies according to the time frames specified: \u25cf 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 \u25cfAll 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) \u25cf Small Molecule Therapies: - Ezutromid (SMT C1100) within 1 week prior to Week 1 \u25cf 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\u00ae) \u25cf 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) \u25cf 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 kt\u00f3rejkolwiek z wymienionych terapii eksperymentalnych w konkretnym okresie czasu:\n\u25cf kiedykolwiek:\n- leki zwi\u0119kszaj\u0105ce ekspresj\u0119 utrofiny (z wyj\u0105tkiem ezutromidu);\n- terapia CRISPR/Cas9 lub inne terapie wykorzystuj\u0105ce techniki edycji gen\u00f3w;\n- terapia genowa;\n- terapia kom\u00f3rkowa (np. przeszczep kom\u00f3rek macierzystych);\n- jakakolwiek terapia antysensownymi oligonukleotydami, poza PRO045 (BMN 045) lub PRO053 (BMN 053) (patrz poni\u017cej);\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\u25cf wszystkie rodzaje leczenia hamuj\u0105ce miostatyn\u0119 w okresie 24 tygodni przed 1. tygodniem, w tym mi\u0119dzy innymi:\n- domagrozumab (PF-06252616);\n- RG-6206 (wcze\u015bniej RO-7239361 i BMS-986089);\n\u25cf terapie z zastosowaniem lek\u00f3w drobnocz\u0105steczkowych:\n- ezutromid (SMT C1100) w okresie 1 tygodnia przed 1. tygodniem;\n\u25cf w okresie 24 tygodni przed 1. tygodniem:\n- leki o dzia\u0142aniu przeciwzw\u0142\u00f3knieniowym lub przeciwzapalnym, w\u0142\u0105czaj\u0105c w to, ale nie ograniczaj\u0105c do: rimeporid, galusan epigallokatechiny, TAS-205, edasaloneksent (CAT 1004), FG-3019 i halofuginon (HT-100);\n- inhibitory aktywacji kom\u00f3rek tucznych (np. CRD007 [s\u00f3l sodowa pemirolastu]);\n- idebenon (Raxone\u00ae);\n\u25cf w okresie 12 tygodni przez 1. tygodniem:\n- leki dzia\u0142aj\u0105ce poprzez tlenek azotu, w\u0142\u0105czaj\u0105c w to (ale nie ograniczaj\u0105c do) metformin\u0119 i cytrulin\u0119, diazotan izosorbidu, tadalafil, sildenafil, pentoksyfilin\u0119, je\u017celi stosowane s\u0105 w ramach badania klinicznego dotycz\u0105cego leczenia dystrofii mi\u0119\u015bniowej Duchenne\u2019a, a nie we wskazaniu klinicznym. Je\u017celi stosowane s\u0105 we wskazaniu klinicznym, musz\u0105 by\u0107 przyjmowane w sta\u0142ej dawce przez przynajmniej 12 tygodni przed 1. tygodniem;\n- wamorolon (VBP-15);\n\u25cf w przypadku wszelkich terapii eksperymentalnych niewymienionych w kryterium wy\u0142\u0105czenia 1 nale\u017cy skonsultowa\u0107 si\u0119 z monitorem bada\u0144 klinicznych.", "language": 19, "languageDescription": "Polish"}, {"id": 6861384, "uuid": "e2ec2eb5-3199-4817-8099-7c158ca0396c", "attributeTranslation": "Haber recibido tratamiento con uno de los f\u00e1rmacos en estudio que aparecen a continuaci\u00f3n y dentro de los periodos especificados:\n\u25cf En cualquier momento:\n- F\u00e1rmacos de aumento de la utrofina (excepto Ezutromid).\n- CRISPR/Cas9, y cualquier otra forma de modificaci\u00f3n del genoma.\n- Genoterapia\n- Citoterapia (p. ej., trasplante de hemocitoblastos)\n- Cualquier forma de tratamiento antisentido con \u00e1cido nucleico, excepto PRO045 (BMN 045) o PRO053 (BMN 053) (v\u00e9ase a continuaci\u00f3n).\n- Tratamientos de omisi\u00f3n 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\u25cf 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\u25cf Tratamientos tradicionales:\n- Ezutromid (SMT C1100) en la semanas anterior a la semana 1.\n\u25cf Dentro de las 24 semanas anteriores a la semana 1:\n- F\u00e1rmacos antifibr\u00f3ticos 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 activaci\u00f3n mastocitaria (p. ej., CRD007 [sodio de pemirolast]).\n- Idebenona (Raxone\u00ae).\n\u25cf Dentro de las 12 semanas anteriores a la semana 1:\n- F\u00e1rmacos que interact\u00faan con el \u00f3xido n\u00edtrico, 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 cl\u00ednico relacionado con la DMD y no por indicaci\u00f3n m\u00e9dica. Si se toma por indicaci\u00f3n m\u00e9dica, debe haberse recibido en una dosis estable durante al menos las 12 semanas anteriores a la semana 1.\n- Vamorolona (VBP-15).\n\u25cf Para informarse sobre cualquier otro tratamiento en estudio que no se haya especificado en el criterio de exclusi\u00f3n 1, consulte al monitor m\u00e9dico.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861385, "uuid": "e2ec2eb5-3199-4817-8099-7c158ca0396c", "attributeTranslation": "Kezel\u00e9s b\u00e1rmely al\u00e1bbi vizsg\u00e1lati kezel\u00e9ssel a megadott id\u0151intervallumokon bel\u00fcl:\n\u25cf B\u00e1rmikor:\n- Utrofin-t\u00falszab\u00e1lyoz\u00f3 k\u00e9sz\u00edtm\u00e9nyek (az ezutromid kiv\u00e9tel\u00e9vel)\n- CRISPR/Cas9, vagy a g\u00e9nedit\u00e1l\u00e1s b\u00e1rmely m\u00e1s form\u00e1ja\n- G\u00e9nter\u00e1pia\n- Sejtalap\u00fa kezel\u00e9s (pl. \u0151ssejt-transzplant\u00e1ci\u00f3)\n- Nukleinsav antisense kezel\u00e9s b\u00e1rmely form\u00e1ja, a PRO045 (BMN 045) vagy PRO053 (BMN 053) kiv\u00e9tel\u00e9vel (l\u00e1sd al\u00e1bb)\n- Exon\u00e1tugr\u00e1sos kezel\u00e9sek\n - Drisapersen az 1. hetet megel\u0151z\u0151 36 h\u00e9tben\n - PRO045 (BMN 045) az 1. hetet megel\u0151z\u0151 24 h\u00e9tben\n- PRO053 (BMN 053) az 1. hetet megel\u0151z\u0151 24 h\u00e9tben\n - PRO051 (BMN 051) az 1. hetet megel\u0151z\u0151 24 h\u00e9tben\n\u2022 Az \u00f6sszes antimyostatin kezel\u00e9s az 1. hetet megel\u0151z\u0151 24 h\u00e9tben, ide\u00e9rtve t\u00f6bbek k\u00f6z\u00f6tt az al\u00e1bbiakat:\n- Domagrozumab (PF-06252616)\n- RG-6206 (hivatalosan RO-7239361 \u00e9s BMS-986089)\n\u2022 Kismolekul\u00e1j\u00fa szerek:\n- Ezutromid (SMT C1100) az 1. hetet megel\u0151z\u0151 1 h\u00e9tben\n\u2022 Az 1. hetet megel\u0151z\u0151 24 h\u00e9tben:\n- Antifibrotikus vagy antiinflammat\u00f3rikus k\u00e9sz\u00edtm\u00e9nyek, bele\u00e9rtve t\u00f6bbek k\u00f6z\u00f6tt az al\u00e1bbiakat: rimeporid, epigallokatechin-gall\u00e1t, TAS-205, edaszalonexent (CAT-1004), FG-3019 \u00e9s halofuginon (HT-100)\n- H\u00edz\u00f3sejt-aktiv\u00e1ci\u00f3 inhibitor (pl. CRD007 [pemirolaszt-n\u00e1trium])\n- Idebenon (Raxone\u00ae)\n\u2022 Az 1. hetet megel\u0151z\u0151 12 h\u00e9tben:\n- Nitrog\u00e9n-oxid-akt\u00edv k\u00e9sz\u00edtm\u00e9nyek, bele\u00e9rtve t\u00f6bbek k\u00f6z\u00f6tt a k\u00f6vetkez\u0151ket: metformin \u00e9s citrullin, isoszorbid-dinitr\u00e1t, tadalafil, szildenafil, pentoxifillin, ha DMD klinikai vizsg\u00e1lat keret\u00e9ben \u00e9s nem orvosi javallatra szedte. Ha orvosi javallatra szedte, akkor az 1. hetet megel\u0151z\u0151 legal\u00e1bb 12 h\u00e9tben a d\u00f3zisnak v\u00e1ltozatlannak kellett lennie.\n- Vamorolon (VBP-15)\n\u2022 Minden vizsg\u00e1lati kezel\u00e9s eset\u00e9n, amely az 1. kiz\u00e1r\u00e1si krit\u00e9riumban nem ker\u00fclt felsorol\u00e1sra, konzult\u00e1ljon az orvosmonitorral.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861386, "uuid": "e2ec2eb5-3199-4817-8099-7c158ca0396c", "attributeTranslation": "\u041b\u0435\u0447\u0435\u043d\u0438\u0435 \u0441 \u043a\u043e\u044f\u0442\u043e \u0438 \u0434\u0430 \u0435 \u043e\u0442 \u0441\u043b\u0435\u0434\u043d\u0438\u0442\u0435 \u0435\u043a\u0441\u043f\u0435\u0440\u0438\u043c\u0435\u043d\u0442\u0430\u043b\u043d\u0438 \u0442\u0435\u0440\u0430\u043f\u0438\u0438 \u0432 \u0441\u044a\u043e\u0442\u0432\u0435\u0442\u0441\u0442\u0432\u0438\u0435 \u0441 \u043f\u043e\u0441\u043e\u0447\u0435\u043d\u0438\u0442\u0435 \u0432\u0440\u0435\u043c\u0435\u0432\u0438 \u0434\u0438\u0430\u043f\u0430\u0437\u043e\u043d\u0438:\n\u25cf \u0412 \u043a\u043e\u0439\u0442\u043e \u0438 \u0434\u0430 \u0435 \u043c\u043e\u043c\u0435\u043d\u0442:\n- \u041f\u0440\u0435\u043f\u0430\u0440\u0430\u0442\u0438 \u0437\u0430 \u0440\u0435\u0433\u0443\u043b\u0438\u0440\u0430\u043d\u0435 \u043d\u0430 \u0443\u0442\u0440\u043e\u0444\u0438\u043d (\u0441 \u0438\u0437\u043a\u043b\u044e\u0447\u0435\u043d\u0438\u0435 \u043d\u0430 \u0435\u0437\u0443\u0442\u0440\u043e\u043c\u0438\u0434)\n- CRISPR/Cas9 \u0438\u043b\u0438 \u0434\u0440\u0443\u0433\u0430 \u0444\u043e\u0440\u043c\u0430 \u043d\u0430 \u0440\u0435\u0434\u0430\u043a\u0442\u0438\u0440\u0430\u043d\u0435 \u043d\u0430 \u0433\u0435\u043d\u043e\u043c\u0430\n- \u0413\u0435\u043d\u043d\u0430 \u0442\u0435\u0440\u0430\u043f\u0438\u044f\n- \u041a\u043b\u0435\u0442\u044a\u0447\u043d\u0430 \u0442\u0435\u0440\u0430\u043f\u0438\u044f (\u043d\u0430\u043f\u0440. \u0442\u0440\u0430\u043d\u0441\u043f\u043b\u0430\u043d\u0442\u0430\u0446\u0438\u044f \u043d\u0430 \u0441\u0442\u0432\u043e\u043b\u043e\u0432\u0438 \u043a\u043b\u0435\u0442\u043a\u0438)\n- \u0412\u0441\u044f\u043a\u0430 \u0444\u043e\u0440\u043c\u0430 \u043d\u0430 \u0430\u043d\u0442\u0438\u0441\u0435\u043d\u0441 \u0442\u0435\u0440\u0430\u043f\u0438\u044f \u0441 \u043d\u0443\u043a\u043b\u0435\u0438\u043d\u043e\u0432\u0430 \u043a\u0438\u0441\u0435\u043b\u0438\u043d\u0430, \u0441 \u0438\u0437\u043a\u043b\u044e\u0447\u0435\u043d\u0438\u0435 \u043d\u0430 PRO045 (BMN 045) \u0438\u043b\u0438 PRO053 (BMN 053) (\u0432\u0438\u0436\u0442\u0435 \u043f\u043e-\u0434\u043e\u043b\u0443)\n- \u0422\u0435\u0440\u0430\u043f\u0438\u0438 \u0441 \u043f\u0440\u0435\u0441\u043a\u0430\u0447\u0430\u043d\u0435 \u043d\u0430 \u0435\u043a\u0437\u043e\u043d\n - \u0414\u0440\u0438\u0441\u0430\u043f\u0435\u0440\u0441\u0435\u043d \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 36 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1\n - PRO045 (BMN 045) \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 24 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1\n- PRO053 (BMN 053) \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 24 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1\n - PRO051 (BMN 051) \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 24 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1\n- \u0412\u0441\u044f\u043a\u0430\u043a\u0432\u0438 \u0442\u0435\u0440\u0430\u043f\u0438\u0438 \u0441 \u0430\u043d\u0442\u0438-\u043c\u0438\u043e\u0441\u0442\u0430\u0442\u0438\u043d \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 24 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1, \u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e, \u043d\u043e \u043d\u0435 \u0441\u0430\u043c\u043e:\n- \u0414\u043e\u043c\u0430\u0433\u0440\u043e\u0437\u0443\u043c\u0430\u0431 (PF-06252616)\n- RG-6206 (\u043d\u0430\u0440\u0438\u0447\u0430\u043d\u043e \u043f\u0440\u0435\u0434\u0438 RO-7239361 \u0438 BMS-986089)\n\u25cf \u0422\u0435\u0440\u0430\u043f\u0438\u0438 \u0441 \u043c\u0430\u043b\u043a\u0438 \u043c\u043e\u043b\u0435\u043a\u0443\u043b\u0438:\n- \u0415\u0437\u0443\u0442\u0440\u043e\u043c\u0438\u0434 (SMT C1100) \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 1 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1\n\u25cf \u0412 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 24 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1:\n- \u0410\u043d\u0442\u0438\u0444\u0438\u0431\u0440\u043e\u0442\u0438\u0447\u043d\u0438 \u0438\u043b\u0438 \u043f\u0440\u043e\u0442\u0438\u0432\u043e\u0432\u044a\u0437\u043f\u0430\u043b\u0438\u0442\u0435\u043b\u043d\u0438 \u043f\u0440\u0435\u043f\u0430\u0440\u0430\u0442\u0438, \u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e, \u043d\u043e \u043d\u0435 \u0441\u0430\u043c\u043e: \u0440\u0438\u043c\u0435\u043f\u043e\u0440\u0438\u0434, \u0435\u043f\u0438\u0433\u0430\u043b\u043e\u043a\u0430\u0442\u0435\u0445\u0438\u043d-\u0433\u0430\u043b\u0430\u0442, TAS-205, \u0435\u0434\u0430\u0437\u0430\u043b\u043e\u043d\u0435\u043a\u0441\u0435\u043d\u0442 (CAT1004), FG-3019 \u0438 \u0445\u0430\u043b\u043e\u0444\u0443\u0433\u0438\u043d\u043e\u043d (HT-100)\n- \u0418\u043d\u0445\u0438\u0431\u0438\u0442\u043e\u0440 \u043d\u0430 \u0430\u043a\u0442\u0438\u0432\u0438\u0440\u0430\u043d\u0435 \u043d\u0430 \u043c\u0430\u0441\u0442\u043e\u0446\u0438\u0442\u0438\u0442\u0435 (\u043d\u0430\u043f\u0440. CRD007 [\u043f\u0435\u043c\u0438\u0440\u043e\u043b\u0430\u0441\u0442 \u043d\u0430\u0442\u0440\u0438\u0439])\n- \u0418\u0434\u0435\u0431\u0435\u043d\u043e\u043d (Raxone\u00ae)\n\u25cf \u0412 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 12 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1:\n- \u041f\u0440\u0435\u043f\u0430\u0440\u0430\u0442\u0438 \u0441 \u0430\u043a\u0442\u0438\u0432\u0435\u043d \u0430\u0437\u043e\u0442\u0435\u043d \u043e\u043a\u0441\u0438\u0434, \u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e, \u043d\u043e \u043d\u0435 \u0441\u0430\u043c\u043e, \u043c\u0435\u0442\u0444\u043e\u0440\u043c\u0438\u043d \u0438 \u0446\u0438\u0442\u0440\u0443\u043b\u0438\u043d, \u0438\u0437\u043e\u0441\u043e\u0440\u0431\u0438\u0434 \u0434\u0438\u043d\u0438\u0442\u0440\u0430\u0442, \u0442\u0430\u0434\u0430\u043b\u0430\u0444\u0438\u043b, \u0441\u0438\u043b\u0434\u0435\u043d\u0430\u0444\u0438\u043b, \u043f\u0435\u043d\u0442\u043e\u043a\u0441\u0438\u0444\u0438\u043b\u0438\u043d, \u043f\u0440\u0438\u0435\u043c\u0430\u043d\u0438 \u043a\u0430\u0442\u043e \u0447\u0430\u0441\u0442 \u043e\u0442 \u043a\u043b\u0438\u043d\u0438\u0447\u043d\u043e \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435 \u0437\u0430 \u041c\u0414\u0414, \u0430 \u043d\u0435 \u0437\u0430 \u043c\u0435\u0434\u0438\u0446\u0438\u043d\u0441\u043a\u043e \u043f\u043e\u043a\u0430\u0437\u0430\u043d\u0438\u0435. \u0410\u043a\u043e \u0441\u0435 \u043f\u0440\u0438\u0435\u043c\u0430\u0442 \u0437\u0430 \u043c\u0435\u0434\u0438\u0446\u0438\u043d\u0441\u043a\u043e \u043f\u043e\u043a\u0430\u0437\u0430\u043d\u0438\u0435, \u0442\u0440\u044f\u0431\u0432\u0430 \u0434\u0430 \u0441\u0430 \u0432 \u0441\u0442\u0430\u0431\u0438\u043b\u043d\u0430 \u0434\u043e\u0437\u0430 \u0432 \u043f\u0440\u043e\u0434\u044a\u043b\u0436\u0435\u043d\u0438\u0435 \u043d\u0430 \u043d\u0430\u0439-\u043c\u0430\u043b\u043a\u043e 12 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1.\n- \u0412\u0430\u043c\u043e\u0440\u043e\u043b\u043e\u043d (VBP-15)\n\u25cf \u0417\u0430 \u0432\u0441\u044f\u043a\u043e \u0435\u043a\u0441\u043f\u0435\u0440\u0438\u043c\u0435\u043d\u0442\u0430\u043b\u043d\u043e \u043b\u0435\u0447\u0435\u043d\u0438\u0435, \u043a\u043e\u0435\u0442\u043e \u043d\u0435 \u0435 \u043f\u043e\u0441\u043e\u0447\u0435\u043d\u043e \u0432 \u041a\u0440\u0438\u0442\u0435\u0440\u0438\u0439 \u0437\u0430 \u0438\u0437\u043a\u043b\u044e\u0447\u0432\u0430\u043d\u0435 1, \u0441\u0435 \u043a\u043e\u043d\u0441\u0443\u043b\u0442\u0438\u0440\u0430\u0439\u0442\u0435 \u0441 \u043c\u0435\u0434\u0438\u0446\u0438\u043d\u0441\u043a\u0438\u044f \u043c\u043e\u043d\u0438\u0442\u043e\u0440.", "language": 1, "languageDescription": "Bulgarian"}]}, {"id": 1005261, "number": 2, "principalExclusionCriteria": "Treatment with any of the following non-investigational therapies according to the time frames specified: \u25cf 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. \u25cf 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": "\u041b\u0435\u0447\u0435\u043d\u0438\u0435 \u0441 \u043a\u043e\u044f\u0442\u043e \u0438 \u0434\u0430 \u0435 \u043e\u0442 \u0441\u043b\u0435\u0434\u043d\u0438\u0442\u0435 \u043d\u0435\u0435\u043a\u0441\u043f\u0435\u0440\u0438\u043c\u0435\u043d\u0442\u0430\u043b\u043d\u0438 \u0442\u0435\u0440\u0430\u043f\u0438\u0438 \u0432 \u0441\u044a\u043e\u0442\u0432\u0435\u0442\u0441\u0442\u0432\u0438\u0435 \u0441 \u043f\u043e\u0441\u043e\u0447\u0435\u043d\u0438\u0442\u0435 \u0432\u0440\u0435\u043c\u0435\u0432\u0438 \u0434\u0438\u0430\u043f\u0430\u0437\u043e\u043d\u0438:\n\u25cf \u0412 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 12 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1:\n- \u0412\u0441\u044f\u043a\u043e \u0444\u0430\u0440\u043c\u0430\u043a\u043e\u043b\u043e\u0433\u0438\u0447\u043d\u043e \u043b\u0435\u0447\u0435\u043d\u0438\u0435 (\u0440\u0430\u0437\u043b\u0438\u0447\u043d\u043e \u043e\u0442 \u043a\u043e\u0440\u0442\u0438\u043a\u043e\u0441\u0442\u0435\u0440\u043e\u0438\u0434\u0438), \u043a\u043e\u0435\u0442\u043e \u043c\u043e\u0436\u0435 \u0434\u0430 \u0438\u043c\u0430 \u0435\u0444\u0435\u043a\u0442 \u0432\u044a\u0440\u0445\u0443 \u043c\u0443\u0441\u043a\u0443\u043b\u043d\u0430\u0442\u0430 \u0441\u0438\u043b\u0430 \u0438\u043b\u0438 \u0444\u0443\u043d\u043a\u0446\u0438\u044f. \u0425\u043e\u0440\u043c\u043e\u043d \u043d\u0430 \u0440\u0430\u0441\u0442\u0435\u0436\u0430 \u0437\u0430 \u043d\u0438\u0441\u044a\u043a \u0440\u044a\u0441\u0442 \u0438 \u0442\u0435\u0441\u0442\u043e\u0441\u0442\u0435\u0440\u043e\u043d \u0437\u0430 \u0437\u0430\u0431\u0430\u0432\u0435\u043d \u043f\u0443\u0431\u0435\u0440\u0442\u0435\u0442 \u0441\u0430 \u0440\u0430\u0437\u0440\u0435\u0448\u0435\u043d\u0438, \u0430\u043a\u043e \u0434\u0438\u0430\u0433\u043d\u043e\u0437\u0430\u0442\u0430 \u0438 \u043c\u0435\u0434\u0438\u0446\u0438\u043d\u0441\u043a\u0430\u0442\u0430 \u043d\u0435\u043e\u0431\u0445\u043e\u0434\u0438\u043c\u043e\u0441\u0442 \u043e\u0442 \u043b\u0435\u0447\u0435\u043d\u0438\u0435\u0442\u043e \u0441\u0430 \u0434\u043e\u043a\u0443\u043c\u0435\u043d\u0442\u0438\u0440\u0430\u043d\u0438 \u043e\u0442 \u043b\u0435\u043a\u0430\u0440 \u0438 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u044a\u0442 \u0435 \u0437\u0430\u043f\u043e\u0447\u043d\u0430\u043b \u043f\u0440\u0438\u0435\u043c\u0430 \u043d\u0430 \u0434\u043e\u0437\u0430\u0442\u0430 \u043f\u043e\u043d\u0435 24 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1.\n\u25cf \u0412 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 12 \u0441\u0435\u0434\u043c\u0438\u0446\u0438 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1 \u0438\u043b\u0438 \u0430\u043a\u043e \u0441\u0435 \u043e\u0447\u0430\u043a\u0432\u0430, \u0447\u0435 \u0449\u0435 \u0435 \u043d\u0435\u043e\u0431\u0445\u043e\u0434\u0438\u043c\u043e \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e:\n- \u0421\u0442\u0430\u0442\u0438\u043d\u0438\n- \u0410\u043c\u0438\u043d\u043e\u0433\u043b\u0438\u043a\u043e\u0437\u0438\u0434\u043d\u0438 \u0430\u043d\u0442\u0438\u0431\u0438\u043e\u0442\u0438\u0446\u0438", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861391, "uuid": "128fa312-9f72-46c9-b4fa-299ee1e06cb3", "attributeTranslation": "Kezel\u00e9s b\u00e1rmely al\u00e1bbi nem vizsg\u00e1lati kezel\u00e9ssel a megadott id\u0151intervallumokon bel\u00fcl:\n\u2022 Az 1. hetet megel\u0151z\u0151 12 h\u00e9tben:\n- B\u00e1rmilyen farmakol\u00f3giai kezel\u00e9s (a kortikoszteroidok kiv\u00e9tel\u00e9vel), amely hat\u00e1ssal lehet az izomer\u0151re vagy izomm\u0171k\u00f6d\u00e9sre. Alacsony termetre adott n\u00f6veked\u00e9si hormon \u00e9s megk\u00e9sett pubert\u00e1sra adott tesztoszteron megengedett, ha az orvos dokument\u00e1lta a diagn\u00f3zist \u00e9s a kezel\u00e9s orvosi sz\u00fcks\u00e9gess\u00e9g\u00e9t, \u00e9s a beteg a kezel\u00e9st legal\u00e1bb 24 h\u00e9ttel az 1. h\u00e9t el\u0151tt kezdte.\n\u2022 Ha a beteg az 1. hetet megel\u0151z\u0151 12 h\u00e9tben szedte vagy a vizsg\u00e1lat alatt v\u00e1rhat\u00f3an szednie kell a k\u00f6vetkez\u0151ket:\n- Sztatinok\n- Aminoglikozid antibiotikumok", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861388, "uuid": "128fa312-9f72-46c9-b4fa-299ee1e06cb3", "attributeTranslation": "leczenie z wykorzystaniem kt\u00f3rejkolwiek z wymienionych terapii nieeksperymentalnych w konkretnym okresie czasu:\n\u25cf w okresie 12 tygodni przez 1. tygodniem:\n- jakiekolwiek leczenie farmakologiczne (inne ni\u017c z wykorzystaniem kortykosteroid\u00f3w), kt\u00f3re mo\u017ce wp\u0142ywa\u0107 na si\u0142\u0119 lub czynno\u015b\u0107 mi\u0119\u015bni. Hormon wzrostu przy terapii zwi\u0105zanej z niskim wzrostem i testosteron stosowany przy op\u00f3\u017anionym dojrzewaniu s\u0105 dozwolone, je\u017celi lekarz posiada udokumentowane rozpoznanie i istnieje medyczna konieczno\u015b\u0107 leczenia, a pacjent rozpocz\u0105\u0142 przyjmowanie leku przynajmniej 24 tygodnie przed 1. tygodniem\n\u25cf w okresie 12 tygodni przed 1. tygodniem lub je\u017celi przewidywane jest stosowanie podczas badania:\n- statyn;\n- antybiotyk\u00f3w aminoglikozydowych.", "language": 19, "languageDescription": "Polish"}, {"id": 6861389, "uuid": "128fa312-9f72-46c9-b4fa-299ee1e06cb3", "attributeTranslation": "Haber recibido tratamiento con uno de los f\u00e1rmacos no investigados que aparecen a continuaci\u00f3n y dentro de los periodos especificados:\n\u25cf Dentro de las 12 semanas anteriores a la semana 1:\n- Cualquier tratamiento farmacol\u00f3gico (diferente de los corticoesteroides) que pueda tener un efecto en la funci\u00f3n o la fuerza musculares. Se permiten los tratamientos con hormona del crecimiento para la estatura y testosterona para la pubertad tard\u00eda si un m\u00e9dico ha documentado el diagn\u00f3stico y la necesidad m\u00e9dica del tratamiento, siempre que el paciente haya empezado el tratamiento al menos 24 semanas antes de la semana 1.\n\u25cf Dentro de las 12 semanas anteriores a la semana 1 o si se prev\u00e9 la necesidad de uso durante el estudio:\n- Estatinas.\n- Aminogluc\u00f3sidos.", "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": "Nagym\u0171t\u00e9t az 1. hetet megel\u0151z\u0151 3 h\u00f3napban vagy tervezett m\u0171t\u00e9t a vizsg\u00e1lat alatt b\u00e1rmikor, a vizsg\u00e1lati tervben el\u0151\u00edrt m\u0171t\u00e9t kiv\u00e9tel\u00e9vel, ha van.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861393, "uuid": "5e208005-1cc0-4c09-862f-4739d4df011a", "attributeTranslation": "du\u017ce zabiegi chirurgiczne w okresie 3 miesi\u0119cy przed 1. tygodniem lub planowane zabiegi chirurgiczne w dowolnym momencie badania, poza zabiegami przewidzianymi w protokole badania, je\u017celi ma to zastosowanie.", "language": 19, "languageDescription": "Polish"}, {"id": 6861395, "uuid": "5e208005-1cc0-4c09-862f-4739d4df011a", "attributeTranslation": "Cirug\u00eda 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 quir\u00fargicas especificadas en el protocolo, seg\u00fan proceda.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861394, "uuid": "5e208005-1cc0-4c09-862f-4739d4df011a", "attributeTranslation": "\u0417\u043d\u0430\u0447\u0438\u043c\u0430 \u043e\u043f\u0435\u0440\u0430\u0446\u0438\u044f \u0432 \u0440\u0430\u043c\u043a\u0438\u0442\u0435 \u043d\u0430 3 \u043c\u0435\u0441\u0435\u0446\u0430 \u043f\u0440\u0435\u0434\u0438 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 1 \u0438\u043b\u0438 \u043f\u043b\u0430\u043d\u0438\u0440\u0430\u043d\u0430 \u043e\u043f\u0435\u0440\u0430\u0446\u0438\u044f \u0432 \u043a\u043e\u0439\u0442\u043e \u0438 \u0434\u0430 \u0435 \u043c\u043e\u043c\u0435\u043d\u0442 \u043f\u043e \u0432\u0440\u0435\u043c\u0435 \u043d\u0430 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e, \u0441 \u0438\u0437\u043a\u043b\u044e\u0447\u0435\u043d\u0438\u0435 \u043d\u0430 \u043f\u043e\u0441\u043e\u0447\u0435\u043d\u0430 \u0432 \u043f\u0440\u043e\u0442\u043e\u043a\u043e\u043b\u0430 \u043e\u043f\u0435\u0440\u0430\u0446\u0438\u044f, \u0430\u043a\u043e \u0435 \u043f\u0440\u0438\u043b\u043e\u0436\u0438\u043c\u043e", "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 k\u00edv\u00fcl egy\u00e9b jelent\u0151s genetikai betegs\u00e9g fenn\u00e1ll\u00e1sa (pl. t\u00f6rpes\u00e9g).", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861397, "uuid": "235e2d49-9a3e-4022-a3df-d71863a5c6ef", "attributeTranslation": "Presencia de cualquier otra enfermedad gen\u00e9tica significativa diferente de la DMD (p. ej., enanismo).", "language": 7, "languageDescription": "Spanish"}, {"id": 6861396, "uuid": "235e2d49-9a3e-4022-a3df-d71863a5c6ef", "attributeTranslation": "obecno\u015b\u0107 jakichkolwiek innych istotnych chor\u00f3b genetycznych, innych ni\u017c dystrofia mi\u0119\u015bniowa Duchenne\u2019a (np. niedob\u00f3r wzrostu).", "language": 19, "languageDescription": "Polish"}, {"id": 6861398, "uuid": "235e2d49-9a3e-4022-a3df-d71863a5c6ef", "attributeTranslation": "\u041d\u0430\u043b\u0438\u0447\u0438\u0435 \u043d\u0430 \u0434\u0440\u0443\u0433\u043e \u0437\u043d\u0430\u0447\u0438\u043c\u043e \u0433\u0435\u043d\u0435\u0442\u0438\u0447\u043d\u043e \u0437\u0430\u0431\u043e\u043b\u044f\u0432\u0430\u043d\u0435, \u0440\u0430\u0437\u043b\u0438\u0447\u043d\u043e \u043e\u0442 \u041c\u0414\u0414 (\u043d\u0430\u043f\u0440. \u043d\u0430\u043d\u0438\u0437\u044a\u043c).", "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": "\u041d\u0430\u043b\u0438\u0447\u0438\u0435 \u043d\u0430 \u0434\u0440\u0443\u0433\u043e \u043a\u043b\u0438\u043d\u0438\u0447\u043d\u043e \u0437\u043d\u0430\u0447\u0438\u043c\u043e \u0437\u0430\u0431\u043e\u043b\u044f\u0432\u0430\u043d\u0435, \u0432\u043a\u043b\u044e\u0447\u0438\u0442\u0435\u043b\u043d\u043e \u0437\u043d\u0430\u0447\u0438\u043c\u043e \u0441\u044a\u0440\u0434\u0435\u0447\u043d\u043e, \u0431\u0435\u043b\u043e\u0434\u0440\u043e\u0431\u043d\u043e, \u0447\u0435\u0440\u043d\u043e\u0434\u0440\u043e\u0431\u043d\u043e, \u0431\u044a\u0431\u0440\u0435\u0447\u043d\u043e, \u0445\u0435\u043c\u0430\u0442\u043e\u043b\u043e\u0433\u0438\u0447\u043d\u043e, \u0438\u043c\u0443\u043d\u043e\u043b\u043e\u0433\u0438\u0447\u043d\u043e \u0437\u0430\u0431\u043e\u043b\u044f\u0432\u0430\u043d\u0435 \u0438\u043b\u0438 \u043f\u043e\u0432\u0435\u0434\u0435\u043d\u0447\u0435\u0441\u043a\u043e \u0440\u0430\u0437\u0441\u0442\u0440\u043e\u0439\u0441\u0442\u0432\u043e, \u0438\u043b\u0438 \u0437\u043b\u043e\u043a\u0430\u0447\u0435\u0441\u0442\u0432\u0435\u043d\u043e \u0437\u0430\u0431\u043e\u043b\u044f\u0432\u0430\u043d\u0435.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861400, "uuid": "a4882533-da28-42ed-9a8b-773ddcd828c3", "attributeTranslation": "Presencia de otras enfermedades cl\u00ednicamente significativas, entre las que se pueden incluir enfermedades card\u00edacas, respiratorias, hep\u00e1ticas, renales, hematol\u00f3gicas o inmunitarias, trastornos de la conducta o neoplasias malignas.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861401, "uuid": "a4882533-da28-42ed-9a8b-773ddcd828c3", "attributeTranslation": "Egy\u00e9b klinikailag jelent\u0151s betegs\u00e9g fenn\u00e1ll\u00e1sa, bele\u00e9rtve a jelent\u0151s kardi\u00e1lis, pulmon\u00e1lis, hepatikus, ren\u00e1lis, hematol\u00f3giai, immunol\u00f3giai vagy viselked\u00e9si betegs\u00e9get \u00e9s a malignit\u00e1st.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861402, "uuid": "a4882533-da28-42ed-9a8b-773ddcd828c3", "attributeTranslation": "obecno\u015b\u0107 innych istotnych klinicznie chor\u00f3b, w tym choroby uk\u0142adu kr\u0105\u017cenia, p\u0142uc, 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 \u2265450 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 odst\u0119p QTcF \u2265450 ms w badaniu elektrokardiogramie (EKG) przesiewowym i pocz\u0105tkowym.", "language": 19, "languageDescription": "Polish"}, {"id": 6861404, "uuid": "1703d685-e1cd-4fa7-9d1a-00e9cc45920d", "attributeTranslation": "LVEF <50% a sz\u0171r\u0151 echokardiogr\u00e1fi\u00e1n (ECHO) vagy QTcF \u2265450 ms a sz\u0171r\u0151 \u00e9s kiindul\u00e1si elektrokardiogramon (EKG).", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861406, "uuid": "1703d685-e1cd-4fa7-9d1a-00e9cc45920d", "attributeTranslation": "FEVI <50 % en el ecocardiograma de la selecci\u00f3n o QTcF \u2265450 ms seg\u00fan los electrocardiogramas (ECG) de la selecci\u00f3n y basal.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861405, "uuid": "1703d685-e1cd-4fa7-9d1a-00e9cc45920d", "attributeTranslation": "\u0424\u0418\u041b\u041a <50% \u043e\u0442 \u0435\u0445\u043e\u043a\u0430\u0440\u0434\u0438\u043e\u0433\u0440\u0430\u043c\u0430\u0442\u0430 (ECHO) \u043e\u0442 \u0441\u043a\u0440\u0438\u043d\u0438\u043d\u0433\u0430 \u0438\u043b\u0438 QTcF \u2265450 msec \u0432\u044a\u0437 \u043e\u0441\u043d\u043e\u0432\u0430 \u043d\u0430 \u0435\u043b\u0435\u043a\u0442\u0440\u043e\u043a\u0430\u0440\u0434\u0438\u043e\u0433\u0440\u0430\u043c\u0430\u0442\u0430 (\u0415\u041a\u0413) \u043e\u0442 \u0441\u043a\u0440\u0438\u043d\u0438\u043d\u0433\u0430 \u0438 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e.", "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": "\u0414\u0438\u0430\u043f\u0430\u0437\u043e\u043d\u044a\u0442 \u043d\u0430 \u0434\u0432\u0438\u0436\u0435\u043d\u0438\u0435 \u043d\u0430 \u0434\u043e\u0440\u0441\u0438\u0444\u043b\u0435\u043a\u0441\u0438\u044f \u0449\u0435 \u0431\u044a\u0434\u0435 \u0438\u0437\u043c\u0435\u0440\u0435\u043d \u0434\u0432\u0443\u0441\u0442\u0440\u0430\u043d\u043d\u043e \u0438 \u0437\u0430\u043f\u0438\u0441\u0430\u043d \u043a\u0430\u0442\u043e \u0433\u0440\u0430\u0434\u0443\u0441\u0438 \u043e\u0442 \u043d\u0435\u0443\u0442\u0440\u0430\u043b\u043d\u0430\u0442\u0430 \u043f\u043e\u0437\u0438\u0446\u0438\u044f (\u0432\u0436. \u0444\u0438\u0433\u0443\u0440\u0430\u0442\u0430). \u0423\u0447\u0430\u0441\u0442\u043d\u0438\u043a\u044a\u0442 \u0449\u0435 \u0431\u044a\u0434\u0435 \u0438\u0437\u043a\u043b\u044e\u0447\u0435\u043d, \u0430\u043a\u043e \u0441\u0440\u0435\u0434\u043d\u0430\u0442\u0430 \u0437\u0430\u0433\u0443\u0431\u0430 \u043d\u0430 \u0434\u043e\u0440\u0441\u0438\u0444\u043b\u0435\u043a\u0441\u0438\u044f \u043d\u0430 \u0434\u0432\u0430\u0442\u0430 \u043a\u0440\u0430\u0439\u043d\u0438\u043a\u0430 \u0435 > -10 \u0433\u0440\u0430\u0434\u0443\u0441\u0430. \u041d\u0430\u043f\u0440\u0438\u043c\u0435\u0440, \u0430\u043a\u043e \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u043a\u044a\u0442 \u0438\u043c\u0430 -8 \u0433\u0440\u0430\u0434\u0443\u0441\u0430 \u043e\u0442 \u0435\u0434\u043d\u0430\u0442\u0430 \u0441\u0442\u0440\u0430\u043d\u0430 \u0438 -12 \u0433\u0440\u0430\u0434\u0443\u0441\u0430 \u043e\u0442 \u0434\u0440\u0443\u0433\u0430\u0442\u0430 \u0441\u0442\u0440\u0430\u043d\u0430, \u0442\u043e\u0439 \u0449\u0435 \u043e\u0442\u0433\u043e\u0432\u0430\u0440\u044f \u043d\u0430 \u043a\u0440\u0438\u0442\u0435\u0440\u0438\u0438\u0442\u0435 \u0437\u0430 \u0443\u0447\u0430\u0441\u0442\u0438\u0435, \u0442\u044a\u0439 \u043a\u0430\u0442\u043e \u0441\u0440\u0435\u0434\u043d\u0430\u0442\u0430 \u0441\u0442\u043e\u0439\u043d\u043e\u0441\u0442 \u0437\u0430 \u0434\u0432\u0435\u0442\u0435 \u0441\u0442\u0440\u0430\u043d\u0438 \u0435 -10 \u0433\u0440\u0430\u0434\u0443\u0441\u0430.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861411, "uuid": "c476c258-ca25-4c92-9a8d-e17408487236", "attributeTranslation": "La amplitud del movimiento de dorsiflexi\u00f3n se determinar\u00e1 de manera bilateral y se registrar\u00e1 como grados desde la posici\u00f3n neutra (v\u00e9ase la figura). El sujeto se excluir\u00e1 si la p\u00e9rdida promedio de dorsiflexi\u00f3n de ambas extremidades es >\u221210 grados. Por ejemplo, si el sujeto presenta \u22128 grados en un lado y \u221212 en otro, ser\u00eda apto para la participaci\u00f3n en el estudio, ya que el promedio de ambos lados es \u221210 grados.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861409, "uuid": "c476c258-ca25-4c92-9a8d-e17408487236", "attributeTranslation": "zgi\u0119cie grzbietowe stopy b\u0119dzie mierzone dwustronnie i zapisywane w postaci stopni od pozycji neutralnej (patrz rycina). Pacjent zostanie wy\u0142\u0105czony z badania, je\u017celi \u015brednia utrata zgi\u0119cia grzbietowego w obu ko\u0144czynach wyniesie >10 stopni. Przyk\u0142adowo, je\u017celi pacjent ma -8 stopni na jednej stronie, a na drugiej -12 stopni, b\u0119dzie kwalifikowa\u0142 si\u0119 do badania poniewa\u017c \u015brednia z obu stron wynosi -10 stopni.", "language": 19, "languageDescription": "Polish"}, {"id": 6861410, "uuid": "c476c258-ca25-4c92-9a8d-e17408487236", "attributeTranslation": "A dorsalflexio mozg\u00e1sterjedelm\u00e9t mindk\u00e9t oldalon m\u00e9rj\u00fck, \u00e9s dokument\u00e1ljuk a semleges helyzett\u0151l m\u00e9rt sz\u00f6get (l\u00e1sd az \u00e1br\u00e1t). A beteg kiz\u00e1r\u00e1sra ker\u00fcl, ha a dorsalflexio \u00e1tlagos besz\u0171k\u00fcl\u00e9se mindk\u00e9t v\u00e9gtagon > -10 fok. P\u00e9ld\u00e1ul, ha a betegn\u00e9l az egyik oldal -8 fok, a m\u00e1sik oldal -12 fok, akkor m\u00e9g mindig alkalmas lehet, mert a k\u00e9t oldal \u00e1tlaga -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": "\u041f\u0440\u0435\u0434\u0445\u043e\u0434\u043d\u043e \u0438\u043b\u0438 \u043d\u0430\u0441\u0442\u043e\u044f\u0449\u043e \u043c\u0435\u0434\u0438\u0446\u0438\u043d\u0441\u043a\u043e \u0437\u0430\u0431\u043e\u043b\u044f\u0432\u0430\u043d\u0435, \u043a\u043e\u0435\u0442\u043e \u043f\u043e \u043f\u0440\u0435\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u0438\u0437\u0441\u043b\u0435\u0434\u043e\u0432\u0430\u0442\u0435\u043b\u044f \u0432\u043b\u0438\u044f\u0435 \u043d\u0435\u0431\u043b\u0430\u0433\u043e\u043f\u0440\u0438\u044f\u0442\u043d\u043e \u043d\u0430 \u0431\u0435\u0437\u043e\u043f\u0430\u0441\u043d\u043e\u0441\u0442\u0442\u0430 \u043d\u0430 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u0430, \u043f\u0440\u0430\u0432\u0438 \u043c\u0430\u043b\u043a\u043e \u0432\u0435\u0440\u043e\u044f\u0442\u043d\u043e \u0438\u0437\u043f\u044a\u043b\u043d\u0435\u043d\u0438\u0435\u0442\u043e \u043d\u0430 \u043a\u0443\u0440\u0441\u0430 \u043d\u0430 \u043b\u0435\u0447\u0435\u043d\u0438\u0435\u0442\u043e \u0438\u043b\u0438 \u043d\u0430\u0440\u0443\u0448\u0430\u0432\u0430 \u043e\u0446\u0435\u043d\u043a\u0430\u0442\u0430 \u043d\u0430 \u0440\u0435\u0437\u0443\u043b\u0442\u0430\u0442\u0438\u0442\u0435 \u043e\u0442 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e. \u0412 \u0434\u043e\u043f\u044a\u043b\u043d\u0435\u043d\u0438\u0435, \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u0438\u0442\u0435, \u043a\u043e\u0438\u0442\u043e \u043f\u043e \u043f\u0440\u0435\u0446\u0435\u043d\u043a\u0430 \u043d\u0430 \u0438\u0437\u0441\u043b\u0435\u0434\u043e\u0432\u0430\u0442\u0435\u043b\u044f \u0438\u0437\u0433\u043b\u0435\u0436\u0434\u0430\u0442 \u043d\u0435\u0441\u043f\u043e\u0441\u043e\u0431\u043d\u0438/\u043d\u0435\u0436\u0435\u043b\u0430\u0435\u0449\u0438 \u0434\u0430 \u0441\u043f\u0430\u0437\u0432\u0430\u0442 \u043f\u0440\u043e\u0446\u0435\u0434\u0443\u0440\u0438\u0442\u0435 \u0432 \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e, \u0449\u0435 \u0431\u044a\u0434\u0430\u0442 \u0438\u0437\u043a\u043b\u044e\u0447\u0435\u043d\u0438.", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861412, "uuid": "db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5", "attributeTranslation": "Enfermedad previa o actual que, en opini\u00f3n del investigador, podr\u00eda afectar negativamente a la seguridad del paciente, hacer improbable que se complete el tratamiento o dificultar la evaluaci\u00f3n de los resultados obtenidos durante el estudio. Adem\u00e1s, se podr\u00e1 excluir a los pacientes que, en opini\u00f3n 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": "Kor\u00e1bbi vagy fenn\u00e1ll\u00f3 eg\u00e9szs\u00e9g\u00fcgyi \u00e1llapot, amely a vizsg\u00e1l\u00f3orvos v\u00e9lem\u00e9nye szerint h\u00e1tr\u00e1nyosan befoly\u00e1soln\u00e1 a beteg biztons\u00e1g\u00e1t, val\u00f3sz\u00edn\u0171tlenn\u00e9 teszi a kezel\u00e9s befejez\u00e9s\u00e9t, vagy akad\u00e1lyozn\u00e1 a vizsg\u00e1lati eredm\u00e9nyek \u00e9rt\u00e9kel\u00e9s\u00e9t. Ezen k\u00edv\u00fcl kiz\u00e1rand\u00f3k azok a betegek, akik a vizsg\u00e1l\u00f3orvos v\u00e9lem\u00e9nye szerint nem k\u00e9pesek / k\u00e9szek egy\u00fcttm\u0171k\u00f6dni a vizsg\u00e1lati elj\u00e1r\u00e1sok sor\u00e1n.", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861414, "uuid": "db21cae1-8cf6-4d5f-b4c7-ab8a183c8af5", "attributeTranslation": "wcze\u015bniejsze lub obecne schorzenia, kt\u00f3re w opinii badacza mog\u0105 negatywnie wp\u0142ywa\u0107 na bezpiecze\u0144stwo pacjenta, sprawiaj\u0105c, \u017ce uko\u0144czenie leczenia jest ma\u0142o prawdopodobne, lub zaburzaj\u0105c ocen\u0119 wynik\u00f3w badania. Ponadto wykluczeni zostan\u0105 pacjenci, kt\u00f3rzy w ocenie badacza mog\u0105 by\u0107 niezdolni lub niech\u0119tni do przestrzegania warunk\u00f3w 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 f\u00e1rmaco del estudio o a alguno de sus componentes.", "language": 7, "languageDescription": "Spanish"}, {"id": 6861418, "uuid": "3d5dfbd1-8924-43af-a1e9-93594f108b0e", "attributeTranslation": "A vizsg\u00e1lati k\u00e9sz\u00edtm\u00e9ny hat\u00f3anyag\u00e1val vagy b\u00e1rmely \u00f6sszetev\u0151j\u00e9vel szemben fenn\u00e1ll\u00f3 t\u00fal\u00e9rz\u00e9kenys\u00e9g", "language": 13, "languageDescription": "Hungarian"}, {"id": 6861419, "uuid": "3d5dfbd1-8924-43af-a1e9-93594f108b0e", "attributeTranslation": "\u0418\u0437\u0432\u0435\u0441\u0442\u043d\u0430 \u0441\u0432\u0440\u044a\u0445\u0447\u0443\u0432\u0441\u0442\u0432\u0438\u0442\u0435\u043b\u043d\u043e\u0441\u0442 \u043a\u044a\u043c \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u043e\u0442\u043e \u043f\u043e \u0438\u0437\u043f\u0438\u0442\u0432\u0430\u043d\u0435\u0442\u043e \u0438\u043b\u0438 \u043a\u044a\u043c \u043d\u044f\u043a\u043e\u0439 \u043e\u0442 \u043a\u043e\u043c\u043f\u043e\u043d\u0435\u043d\u0442\u0438\u0442\u0435 \u043c\u0443", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861417, "uuid": "3d5dfbd1-8924-43af-a1e9-93594f108b0e", "attributeTranslation": "nadwra\u017cliwo\u015b\u0107 na badany lek lub kt\u00f3rykolwiek z jego sk\u0142adnik\u00f3w.", "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": "\u0414\u0432\u043e\u0439\u043d\u043e \u0441\u043b\u044f\u043f \u043f\u0435\u0440\u0438\u043e\u0434: \u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u043e\u0442 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u043f\u0440\u0435\u0437 \u0441\u0435\u0434\u043c\u0438\u0446\u0430 96 \u0432 \u0441\u043a\u043e\u0440\u043e\u0441\u0442\u0442\u0430 \u043d\u0430 \u0438\u0437\u043a\u0430\u0447\u0432\u0430\u043d\u0435 \u043d\u0430 4 \u0441\u0442\u044a\u043f\u0430\u043b\u0430 (\u0441\u0442\u044a\u043f\u043a\u0430/\u0441\u0435\u043a\u0443\u043d\u0434\u0430)", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861368, "uuid": "56d221c8-2997-41d9-9b50-7a2aa5066d45", "attributeTranslation": "okres leczenia w fazie podw\u00f3jnie za\u015blepionej: stanowi zmiana w 96. tygodniu w stosunku do punktu pocz\u0105tkowego w czasie na pokonanie 4 schod\u00f3w (stopie\u0144/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 (escal\u00f3n/segundo)", "language": 7, "languageDescription": "Spanish"}, {"id": 6861370, "uuid": "56d221c8-2997-41d9-9b50-7a2aa5066d45", "attributeTranslation": "Kett\u0151s vak szakasz: v\u00e1ltoz\u00e1s a kiindul\u00e1st\u00f3l a 96. h\u00e9tig a 4 l\u00e9p\u00e9ses emelked\u00e9si sebess\u00e9gben (l\u00e9p\u00e9s/m\u00e1sodperc)", "language": 13, "languageDescription": "Hungarian"}]}], "secondaryEndPoints": [{"id": 682289, "number": 1, "endPoint": "\u2022 Change from Baseline at Week 96 in 6MWT \u2022 Change from Baseline at Week 96 in rise from floor velocity (rise/second) \u2022 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 pocz\u0105tkowego w ocenie 6MWT; zmiana w 96. tygodniu w stosunku do punktu pocz\u0105tkowego w ocenie wstawania z pod\u0142ogi (wstawanie/sekundy); zmiana w 144. tygodniu w stosunku do punktu pocz\u0105tkowego w ocenie czasu na pokonanie 4 schod\u00f3w (stopie\u0144/sekundy);", "language": 19, "languageDescription": "Polish"}, {"id": 6861373, "uuid": "6ecbb7b6-5478-44ce-af09-d5f1a0d0e252", "attributeTranslation": "\u2022 Variaci\u00f3n desde el momento basal hasta la semana 96 en la prueba de marcha de seis minutos\n\u2022 Variaci\u00f3n desde el momento basal hasta la semana 96 en la velocidad para levantarse del suelo (subida/segundo)\n\u2022 Variaci\u00f3n desde el momento basal hasta la semana 144 en la velocidad para subir 4 escalones (escal\u00f3n/segundo)", "language": 7, "languageDescription": "Spanish"}, {"id": 6861375, "uuid": "6ecbb7b6-5478-44ce-af09-d5f1a0d0e252", "attributeTranslation": "\u2022 \u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u043e\u0442 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u043d\u0430 \u0421\u0435\u0434\u043c\u0438\u0446\u0430 96 \u0432 6-\u043c\u0443\u043d\u0438\u0442\u043d\u0438\u044f \u0442\u0435\u0441\u0442 \u0437\u0430 \u0445\u043e\u0434\u0435\u043d\u0435\n\u2022 \u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u043e\u0442 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u043d\u0430 \u0421\u0435\u0434\u043c\u0438\u0446\u0430 96 \u0432 \u0441\u043a\u043e\u0440\u043e\u0441\u0442\u0442\u0430 \u043d\u0430 \u0438\u0437\u043f\u0440\u0430\u0432\u044f\u043d\u0435 \u043e\u0442 \u043f\u043e\u0434\u0430 (\u0441\u0442\u0430\u0432\u0430\u043d\u0435/\u0441\u0435\u043a\u0443\u043d\u0434\u0430)\n\u2022 \u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u043e\u0442 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u043d\u0430 \u0421\u0435\u0434\u043c\u0438\u0446\u0430 144 \u0432 \u0441\u043a\u043e\u0440\u043e\u0441\u0442\u0442\u0430 \u043d\u0430 \u0438\u0437\u043a\u0430\u0447\u0432\u0430\u043d\u0435 \u043d\u0430 4 \u0441\u0442\u044a\u043f\u0430\u043b\u0430 (\u0441\u0442\u044a\u043f\u043a\u0430/\u0441\u0435\u043a\u0443\u043d\u0434\u0430)", "language": 1, "languageDescription": "Bulgarian"}, {"id": 6861374, "uuid": "6ecbb7b6-5478-44ce-af09-d5f1a0d0e252", "attributeTranslation": "- V\u00e1ltoz\u00e1s a kiindul\u00e1si \u00e9rt\u00e9khez k\u00e9pest a 96. h\u00e9ten a 6MWT-ben\n- V\u00e1ltoz\u00e1s a 96. h\u00e9ten a padl\u00f3r\u00f3l val\u00f3 felemelked\u00e9s sebess\u00e9g\u00e9ben a kiindul\u00e1si \u00e9rt\u00e9khez k\u00e9pest\n- V\u00e1ltoz\u00e1s a kiindul\u00e1si \u00e9rt\u00e9khez k\u00e9pest a 144. h\u00e9ten a 4 l\u00e9p\u00e9ses emelked\u00e9si sebess\u00e9gben", "language": 13, "languageDescription": "Hungarian"}]}, {"id": 682290, "number": 2, "endPoint": "\u2022 Change from Baseline at Week 96 in 10MWR velocity (meter/second) \u2022 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": "\u2022 Variaci\u00f3n con respecto al momento basal en la semana 96 en la velocidad de la prueba de caminar/correr 10 metros (metros/segundo)\n\u2022 Variaci\u00f3n con respecto al momento basal en las semanas 48 o 96 en la cantidad de expresi\u00f3n de la prote\u00edna 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 pocz\u0105tkowego w ocenie 10MWR (metr/sekundy); zmiana w 48. lub 96. tygodniu w stosunku do punktu pocz\u0105tkowego w ilo\u015bci ekspresji dystrofiny w biopsyjnej tkance mi\u0119\u015bnia w oparciu o pomiar metod\u0105 Western blot", "language": 19, "languageDescription": "Polish"}, {"id": 6861378, "uuid": "6971a9ea-0519-47c5-a527-034ab98b399d", "attributeTranslation": "\u2022 \u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u043e\u0442 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u043d\u0430 \u0421\u0435\u0434\u043c\u0438\u0446\u0430 96 \u0432 \u0441\u043a\u043e\u0440\u043e\u0441\u0442\u0442\u0430 \u043f\u0440\u0438 \u0442\u0435\u0441\u0442\u0430 \u0437\u0430 \u0445\u043e\u0434\u0435\u043d\u0435/\u0431\u044f\u0433\u0430\u043d\u0435 \u043d\u0430 10 \u043c\u0435\u0442\u0440\u0430 (\u043c\u0435\u0442\u044a\u0440/\u0441\u0435\u043a\u0443\u043d\u0434\u0430)\n\u2022 \u041f\u0440\u043e\u043c\u044f\u043d\u0430 \u043e\u0440 \u0438\u0437\u0445\u043e\u0434\u043d\u043e\u0442\u043e \u043d\u0438\u0432\u043e \u043d\u0430 \u0421\u0435\u0434\u043c\u0438\u0446\u0438 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{"productPharmForm": "N/A", "euMpNumber": "N/A", "marketingAuthNumber": "N/A", "prodName": "Placebo for PF-06939926", "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", "devices": [], "characteristics": [], "routes": [], "allSubstancesChemicals": false, "productName": "Placebo for PF-06939926", "jsonActiveSubstanceNames": "n/a", "pharmaceuticalFormDisplay": "N/A"}], "trialDetails": {"clinicalTrialIdentifiers": {"fullTitle": "C3391003 - A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY", "fullTitleTranslations": [{"id": 9800005, "uuid": "4089f63f-28f3-48ce-b5d6-ca9f0a083b29", "attributeTranslation": "\u00c9TUDE DE PHASE 3, MULTICENTRIQUE, RANDOMIS\u00c9E, EN DOUBLE AVEUGLE ET CONTR\u00d4L\u00c9E PAR PLACEBO \u00c9VALUANT L'INNOCUIT\u00c9 ET L'EFFICACIT\u00c9 DU PF-06939926 DANS LE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE DE DUCHENNE", "language": 34, "languageDescription": "French (France)"}, {"id": 9800004, "uuid": "4089f63f-28f3-48ce-b5d6-ca9f0a083b29", "attributeTranslation": "UN ESTUDIO DE FASE 3, MULTIC\u00c9NTRICO, CON ALEATORIZACI\u00d3N, DOBLE CIEGO Y CONTROLADO CON PLACEBO PARA EVALUAR LA SEGURIDAD Y EFICACIA DE PF-06939926 PARA EL TRATAMIENTO DE LA DISTROFIA MUSCULAR DE DUCHENNE", "language": 7, "languageDescription": "Spanish"}], "publicTitle": "A PHASE 3, RANDOMIZED, DOUBLE-BLIND,PLACEBO-CONTROLLED STUDY OF PF-06939926 IN DMD", "publicTitleTranslations": [{"id": 9800025, "uuid": "4b693765-1fee-4fde-a43e-900d0ded2634", "attributeTranslation": "\u00c9TUDE DE PHASE 3, RANDOMISEE, EN DOUBLE AVEUGLE ET CONTROLEE PAR PLACEBO SUR LE PF-06939926 DANS LA DMD", "language": 34, "languageDescription": "French (France)"}, {"id": 9800024, "uuid": "4b693765-1fee-4fde-a43e-900d0ded2634", "attributeTranslation": "UN ESTUDIO DE FASE 3, CON ALEATORIZACI\u00d3N, DOBLE CIEGO Y CONTROLADO CON PLACEBO SOBRE PF-06939926 PARA DMD", "language": 7, "languageDescription": "Spanish"}], "shortTitle": "C3391003", "secondaryIdentifyingNumbers": {"nctNumber": {"id": 490153, "number": "NCT04281485"}, "additionalRegistries": []}}, "trialInformation": {"trialCategory": {"isLowIntervention": false, "trialPhase": "5", "trialCategory": "2", "justificationForTrialCategory": "This is a Phase 3 Trial", "trialCategoryId": 109176}, "medicalCondition": {"partIMedicalConditions": [{"id": 123960, "medicalCondition": "DUCHENNE MUSCULAR DYSTROPHY (DMD)", "medicalConditionTranslations": [{"id": 9799948, "uuid": "0888c61d-509e-4252-b26b-3ecb9156ed1d", "attributeTranslation": "DISTROFIA MUSCULAR DE DUCHENNE (DMD)", "language": 7, "languageDescription": "Spanish"}, {"id": 9799949, "uuid": "0888c61d-509e-4252-b26b-3ecb9156ed1d", "attributeTranslation": "DYSTROPHIE MUSCULAIRE DE DUCHENNE", "language": 34, "languageDescription": "French (France)"}], "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": 360680}, {"code": "3", "trialScopeId": 360681}, {"code": "4", "trialScopeId": 360682}], "mainObjective": "To demonstrate superior efficacy of treatment with fordadistrogene movaparvovec as compared to placebo based on change from Baseline in the North Star Ambulatory Assessment (NSAA).", "mainObjectiveTranslations": [{"id": 9800022, "uuid": "3cc12d5c-0e24-40a2-80e8-76da96aff1d3", "attributeTranslation": "D\u00e9montrer la sup\u00e9riorit\u00e9 de l\u2019efficacit\u00e9 du traitement par fordadistrogene movaparvovec comparativement au placebo d\u2019apr\u00e8s la variation par rapport \u00e0 la valeur initiale du score obtenu sur l\u2019\u00c9chelle d\u2019\u00e9valuation ambulatoire North Star (NSAA)", "language": 34, "languageDescription": "French (France)"}, {"id": 9800023, "uuid": "3cc12d5c-0e24-40a2-80e8-76da96aff1d3", "attributeTranslation": "Demostrar una eficacia superior del tratamiento con fordadistrog\u00e9n movaparvovec en comparaci\u00f3n con el placebo seg\u00fan el cambio con respecto al valor de referencia en la evaluaci\u00f3n ambulatoria North Star (NSAA).", "language": 7, "languageDescription": "Spanish"}], "secondaryObjectives": [{"id": 419243, "number": 1, "secondaryObjective": "To quantify the mini-dystrophin expression level in the muscle of participants treated with fordadistrogene movaparvovec", "secondaryObjectiveTranslations": [{"id": 9800027, "uuid": "b5d9ccee-fcf4-46d2-b6b5-1a74ca5e282b", "attributeTranslation": "Cuantificar el nivel de expresi\u00f3n de minidistrofina en el m\u00fasculo de los participantes tratados con fordadistrogene movaparvovec", "language": 7, "languageDescription": "Spanish"}, {"id": 9800026, "uuid": "b5d9ccee-fcf4-46d2-b6b5-1a74ca5e282b", "attributeTranslation": "Quantifier le niveau d'expression de mini-dystrophine dans le muscle des patients trait\u00e9s par fordadistrogene movaparvovec", "language": 34, "languageDescription": "French (France)"}]}, {"id": 419244, "number": 2, "secondaryObjective": "To characterize the distribution of mini-dystrophin expression in the muscle of participants treated with fordadistrogene movaparvovec", "secondaryObjectiveTranslations": [{"id": 9800029, "uuid": "5af41c87-b7fe-453d-957f-cbed9d33b09d", "attributeTranslation": "Caracterizar la distribuci\u00f3n de la expresi\u00f3n de minidistrofina en el m\u00fasculo de los participantes tratados con fordadistrogene movaparvovec", "language": 7, "languageDescription": "Spanish"}, {"id": 9800028, "uuid": "5af41c87-b7fe-453d-957f-cbed9d33b09d", "attributeTranslation": "Caract\u00e9riser la distribution de l\u2019expression de la mini-dystrophine dans le muscle des patients trait\u00e9s par fordadistrogene movaparvovec.", "language": 34, "languageDescription": "French (France)"}]}, {"id": 419245, "number": 3, "secondaryObjective": "To characterize the change in serum creatine kinase (CK) concentration in participants treated with fordadistrogene movaparvovec as compared to placebo.", "secondaryObjectiveTranslations": [{"id": 9800031, "uuid": "e41c3890-e67a-4b6c-9cf5-b04dde381264", "attributeTranslation": "Caracterizar el cambio en la concentraci\u00f3n de creatina quinasa (Creatine Kinase, CK) s\u00e9rica en los participantes tratados con fordadistrogene movaparvovec en comparaci\u00f3n con el placebo.", "language": 7, "languageDescription": "Spanish"}, {"id": 9800030, "uuid": "e41c3890-e67a-4b6c-9cf5-b04dde381264", "attributeTranslation": "Caract\u00e9riser la variation de la concentration s\u00e9rique de cr\u00e9atine kinase (CK) chez les patients trait\u00e9s par fordadistrogene movaparvovec comparativement au placebo.", "language": 34, "languageDescription": "French (France)"}]}, {"id": 419246, "number": 4, "secondaryObjective": "To characterize the skills gained, based on the individual items of the NSAA, in participants treated with PF-06939926 as compared to placebo.", "secondaryObjectiveTranslations": [{"id": 9800032, "uuid": "645f65a6-ed1c-43dd-ae32-63edb892ad11", "attributeTranslation": "Caracterizar las habilidades adquiridas, seg\u00fan los elementos individuales de la NSAA, en los participantes tratados con PF-06939926 en comparaci\u00f3n con el placebo.", "language": 7, "languageDescription": "Spanish"}, {"id": 9800033, "uuid": "645f65a6-ed1c-43dd-ae32-63edb892ad11", "attributeTranslation": "Caract\u00e9riser les comp\u00e9tences acquises, d\u2019apr\u00e8s les items individuels de l\u2019\u00e9chelle NSAA, chez les patients trait\u00e9s par PF-06939926 comparativement au placebo.", "language": 34, "languageDescription": "French (France)"}]}, {"id": 419247, "number": 5, "secondaryObjective": "To characterize the skills either improved or maintained, based on the individual items of the NSAA, in participants treated with fordadistrogene movaparvovec as compared to placebo.", "secondaryObjectiveTranslations": [{"id": 9800034, "uuid": "579e032a-f340-4fe2-a0dc-eb6f5ad9d9f1", "attributeTranslation": "Caracterizar las habilidades mejoradas o mantenidas, seg\u00fan los elementos individuales de la NSAA, en los participantes tratados con fordadistrog\u00e9n movaparvovec en comparaci\u00f3n con el placebo", "language": 7, "languageDescription": "Spanish"}, {"id": 9800035, "uuid": "579e032a-f340-4fe2-a0dc-eb6f5ad9d9f1", "attributeTranslation": "Caract\u00e9riser les comp\u00e9tences am\u00e9lior\u00e9es ou maintenues, d\u2019apr\u00e8s les items individuels de l\u2019\u00e9chelle NSAA, chez les patients trait\u00e9s par fordadistrogene movaparvovec comparativement au placebo.", "language": 34, "languageDescription": "French (France)"}]}, {"id": 419248, "number": 6, "secondaryObjective": "To characterize the 10-meter run/walk velocity in participants treated with PF-06939926 as compared to placebo.", "secondaryObjectiveTranslations": [{"id": 9800036, "uuid": "0aace278-bab7-4387-8e52-e7f9ad761c22", "attributeTranslation": "Caract\u00e9riser la vitesse de marche/course sur 10 m\u00e8tres chez les patients trait\u00e9s par PF-06939926 comparativement au placebo.", "language": 34, "languageDescription": "French (France)"}, {"id": 9800037, "uuid": "0aace278-bab7-4387-8e52-e7f9ad761c22", "attributeTranslation": "Caracterizar la velocidad de carrera/caminata de 10 metros en los participantes tratados con PF-06939926 en comparaci\u00f3n con el placebo.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 419249, "number": 7, "secondaryObjective": "To characterize the rise from floor velocity in participants treated with fordadistrogene movaparvovec as compared to placebo.", "secondaryObjectiveTranslations": [{"id": 9800038, "uuid": "3bd5af15-2322-4b29-b826-b7606adbef0d", "attributeTranslation": "Caract\u00e9riser la vitesse du lever depuis la position couch\u00e9e chez les patients trait\u00e9s par fordadistrogene movaparvovec comparativement au placebo.", "language": 34, "languageDescription": "French (France)"}, {"id": 9800039, "uuid": "3bd5af15-2322-4b29-b826-b7606adbef0d", "attributeTranslation": "Caracterizar la velocidad de elevaci\u00f3n desde el piso en los participantes tratados con fordadistrog\u00e9n movaparvovec en comparaci\u00f3n con el placebo.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 419250, "number": 8, "secondaryObjective": "To characterize the functional health status in participants treated with fordadistrogene movaparvovec as compared to placebo.", "secondaryObjectiveTranslations": [{"id": 9800040, "uuid": "a05f0298-03cd-4501-a9f9-060ef93a51b2", "attributeTranslation": "Caracterizar el estado de salud funcional en los participantes tratados con fordadistrog\u00e9n movaparvovec en comparaci\u00f3n con el placebo.", "language": 7, "languageDescription": "Spanish"}, {"id": 9800041, "uuid": "a05f0298-03cd-4501-a9f9-060ef93a51b2", "attributeTranslation": "Caract\u00e9riser l'\u00e9tat de sant\u00e9 fonctionnel chez les patients trait\u00e9s par fordadistrogene movaparvovec comparativement au placebo.", "language": 34, "languageDescription": "French (France)"}]}]}, "eligibilityCriteria": {"principalInclusionCriteria": [{"id": 801647, "number": 1, "principalInclusionCriteria": "Male participants who are \u22654 and <8 years of age at Screening (Visit 1).", "principalInclusionCriteriaTranslations": [{"id": 9800007, "uuid": "0fb665d2-f149-4345-8f77-6009e75f991a", "attributeTranslation": "Patients masculins \u00e2g\u00e9s de \u2265 4 ans et < 8 ans au moment de la s\u00e9lection (visite 1).", "language": 34, "languageDescription": "French (France)"}, {"id": 9800006, "uuid": "0fb665d2-f149-4345-8f77-6009e75f991a", "attributeTranslation": "Participantes masculinos que tengan \u22654 y <8 a\u00f1os en la Selecci\u00f3n (Visita 1).", "language": 7, "languageDescription": "Spanish"}]}, {"id": 801648, "number": 2, "principalInclusionCriteria": "Confirmed diagnosis of DMD by prior genetic testing demonstrating the presence of a mutation in the dystrophin gene consistent with DMD at Screening (Visit 1). If the Investigator determines that the results are inconclusive, a repeat genetic testing will be allowed through the central laboratory at Screening (Visit 1).", "principalInclusionCriteriaTranslations": [{"id": 9800008, "uuid": "9190e832-baa9-42c0-abdf-3e9b37d6c3cd", "attributeTranslation": "Diagn\u00f3stico confirmado de DMD mediante prueba gen\u00e9tica previa que demuestre la presencia de una mutaci\u00f3n en el gen distrofina conforme a la DMD en la Selecci\u00f3n (Visita 1). Si el investigador determina que los resultados no son concluyentes, se permitir\u00e1 la repetici\u00f3n de una prueba gen\u00e9tica mediante el laboratorio central en la Selecci\u00f3n (Visita 1).", "language": 7, "languageDescription": "Spanish"}, {"id": 9800009, "uuid": "9190e832-baa9-42c0-abdf-3e9b37d6c3cd", "attributeTranslation": "Diagnostic confirm\u00e9 de DMD par des tests g\u00e9n\u00e9tiques ant\u00e9rieurs d\u00e9montrant la pr\u00e9sence d'une mutation du g\u00e8ne de la dystrophine compatible avec une DMD lors de la s\u00e9lection (visite 1). Si l'investigateur d\u00e9cide que les r\u00e9sultats ne sont pas concluants, la r\u00e9alisation d\u2019un nouveau test g\u00e9n\u00e9tique par le laboratoire central est autoris\u00e9e lors de la s\u00e9lection (visite 1).", "language": 34, "languageDescription": "French (France)"}]}, {"id": 801649, "number": 3, "principalInclusionCriteria": "Receipt of a stable daily dose of glucocorticoids (\u22650.5 mg/kg/day prednisone, prednisolone, or \u22650.75 mg/kg/day deflazacort) for at least 3 months prior to Screening (Visit 1) and during the period between Screening (Visit 1) and Day 1 (Visit 3). In order to comply with protocol procedures, there should also be a reasonable expectation that this daily dose of glucocorticoids will remain stable for the first 2 years of the study. A stable dose is defined as one in which any change is \u22640.2 mg/kg.", "principalInclusionCriteriaTranslations": [{"id": 9800011, "uuid": "09a7ce53-d9c0-47f7-b111-fdaa5127f53c", "attributeTranslation": "Recepci\u00f3n de una dosis diaria estable de glucocorticoides (\u22650,5 mg/kg por d\u00eda de prednisona o prednisolona, o \u22650,75 mg/kg por d\u00eda de deflazacort) durante al menos 3 meses previos a la Selecci\u00f3n (Visita 1) y durante el per\u00edodo entre la Selecci\u00f3n (Visita 1) y el D\u00eda 1 (Visita 3). Para cumplir con los procedimientos del protocolo, tambi\u00e9n debe existir una expectativa razonable de que esta dosis diaria de glucocorticoides permanecer\u00e1 estable durante los primeros 2 a\u00f1os del estudio. Una dosis estable se define como una dosis en la cual cualquier cambio es de \u22640,2 mg/kg", "language": 7, "languageDescription": "Spanish"}, {"id": 9800010, "uuid": "09a7ce53-d9c0-47f7-b111-fdaa5127f53c", "attributeTranslation": "Traitement par une dose quotidienne stable de glucocortico\u00efdes (\u2265 0,5 mg/kg/jour de prednisone, prednisolone ou \u2265 0,75 mg/kg/jour de d\u00e9flazacort) pendant au moins 3 mois avant la s\u00e9lection (visite 1) et pendant la p\u00e9riode entre la s\u00e9lection (visite 1) et le jour 1 (visite 3). Afin de se conformer aux proc\u00e9dures du protocole, il doit aussi pouvoir \u00eatre raisonnablement suppos\u00e9 que cette dose quotidienne de glucocortico\u00efdes restera stable pendant les 2 premi\u00e8res ann\u00e9es de l\u2019\u00e9tude. Une dose stable d\u00e9signe une dose pour laquelle toute modification est \u2264 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 puntuaci\u00f3n total de NSAA es de >16 y <30 en la Selecci\u00f3n (Visita 1).", "language": 7, "languageDescription": "Spanish"}, {"id": 9800013, "uuid": "ba14d36a-2ed5-45ad-8af8-adde49719d8e", "attributeTranslation": "Score total NSAA > 16 et < 30 \u00e0 la s\u00e9lection (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 Selecci\u00f3n (Visita 1).", "language": 7, "languageDescription": "Spanish"}, {"id": 9800014, "uuid": "d7b3f99c-a50e-47da-a144-0271627e50ab", "attributeTranslation": "\u00catre ambulatoire, d\u00e9fini comme le fait d\u2019\u00eatre capable de marcher sur 10 m\u00e8tres sans assistance, lors de la s\u00e9lection (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/repr\u00e9sentants l\u00e9galement acceptables dispos\u00e9s et aptes \u00e0 se conformer \u00e0 toutes les visites programm\u00e9es, au plan de traitement, aux analyses biologiques, aux consid\u00e9rations li\u00e9es au mode de vie et aux autres proc\u00e9dures de l\u2019\u00e9tude, y compris, vraisemblablement, \u00e0 des biopsies musculaires incisionnelles sous anesth\u00e9sie g\u00e9n\u00e9rale et \u00e0 une IRM cardiaque sous anesth\u00e9sie g\u00e9n\u00e9rale.", "language": 34, "languageDescription": "French (France)"}, {"id": 9800017, "uuid": "4c8f17f0-0a7e-4349-8b76-0f33a38d46e5", "attributeTranslation": "Participantes o representantes autorizados legalmente que est\u00e1n 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 card\u00edaca 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 ap\u00e9ndice 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/repr\u00e9sentants l\u00e9galement acceptables aptes \u00e0 donner leur assentiment/consentement \u00e9clair\u00e9 sign\u00e9 comme d\u00e9crit dans l\u2019annexe 1, ce qui inclut le respect des exigences et des restrictions \u00e9num\u00e9r\u00e9es dans le document d\u2019assentiment/formulaire de consentement \u00e9clair\u00e9 (DCE) et dans le pr\u00e9sent 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/repr\u00e9sentants l\u00e9galement acceptables dispos\u00e9s \u00e0 prot\u00e9ger l\u2019int\u00e9grit\u00e9 des donn\u00e9es de l\u2019\u00e9tude en ne recherchant pas activement des donn\u00e9es cliniques sensibles (par exemple CK, ALAT, ASAT, AcN anti-AAV9) par le biais d\u2019analyses effectu\u00e9es dans des laboratoires ind\u00e9pendants et en ne partageant pas leurs exp\u00e9riences concernant l\u2019essai avec d\u2019autres patients ou publiquement (par exemple via les r\u00e9seaux sociaux).", "language": 34, "languageDescription": "French (France)"}, {"id": 9800021, "uuid": "4c118d17-8dc1-4762-a722-e62ef741e9e3", "attributeTranslation": "Participantes o representantes autorizados legalmente que est\u00e1n dispuestos a proteger la integridad de los datos del estudio al no buscar de forma activa datos cl\u00ednicos 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 p\u00fablica (p. ej., a trav\u00e9s 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 g\u00e9nica, definida como cualquier terapia en la que se introduzca ADN ex\u00f3geno o destinada a alterar de forma permanente el ADN end\u00f3geno. Se prohibir\u00e1 la terapia g\u00e9nica (que no sea el PEI) durante el estudio.", "language": 7, "languageDescription": "Spanish"}, {"id": 9799971, "uuid": "119c9448-9289-466e-96f4-c4a197e25fcf", "attributeTranslation": "Traitement ant\u00e9rieur par th\u00e9rapie g\u00e9nique, d\u00e9fini comme tout traitement avec introduction d\u2019ADN exog\u00e8ne ou destin\u00e9 \u00e0 modifier d\u00e9finitivement l\u2019ADN endog\u00e8ne. Toute th\u00e9rapie g\u00e9nique (autre que le ME) sera interdite pendant la dur\u00e9e de l\u2019\u00e9tude.", "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 aigu\u00eb \u00e0 la s\u00e9lection (visite 1) ou \u00e0 l'inclusion (visite 2) qui, de l'avis de l'investigateur, ne devrait pas \u00eatre totalement r\u00e9solue au moins 2 semaines avant le jour 1 (visite 3). Au jour 1 (visite 3), les patients doivent avoir \u00e9t\u00e9 exempts d'infection pendant au moins 2 semaines avant l'administration du ME. Un retard d'administration du ME jusqu'\u00e0 14 jours est autoris\u00e9 pour permettre une r\u00e9solution compl\u00e8te des infections.", "language": 34, "languageDescription": "French (France)"}, {"id": 9799973, "uuid": "354ff010-cdde-40b7-bd77-197a982a99f9", "attributeTranslation": "Infecci\u00f3n aguda en la Selecci\u00f3n (Visita 1) o el Inicio (Visita 2) cuya resoluci\u00f3n completa, a juicio del investigador, no se espere al menos 2 semanas antes del D\u00eda 1 (Visita 3). En el D\u00eda 1 (Visita 3), los participantes deben estar libres de infecciones durante al menos 2 semanas antes de la administraci\u00f3n del PEI. Se permite una demora de la administraci\u00f3n del PEI de 14 d\u00edas como m\u00e1ximo 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 impliqu\u00e9s dans la r\u00e9alisation de l\u2019\u00e9tude, ainsi que les membres de leur famille, membres du personnel du centre par ailleurs supervis\u00e9s par l\u2019investigateur, ou employ\u00e9s de Pfizer, ainsi que les membres de leur famille, directement impliqu\u00e9s dans la r\u00e9alisation de l\u2019\u00e9tude.", "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 realizaci\u00f3n del estudio y sus familiares; miembros del personal del centro que sean supervisados de alg\u00fan modo por el investigador, o empleados de Pfizer (incluidos sus familiares) implicados directamente en la realizaci\u00f3n 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 soluci\u00f3n para infusi\u00f3n, como hipersensibilidad a la alb\u00famina o un diagn\u00f3stico de IHF. Los s\u00edntomas que sugieren una IHF incluyen n\u00e1useas, v\u00f3mitos, hinchaz\u00f3n, dolor estomacal o diarrea despu\u00e9s de la ingesta de alimentos o bebidas dulces, o un patr\u00f3n de evasi\u00f3n de alimentos o bebidas dulces.", "language": 7, "languageDescription": "Spanish"}, {"id": 9799976, "uuid": "5fe74b7b-6315-4970-9725-25a29440af74", "attributeTranslation": "Hypersensibilit\u00e9 connue \u00e0 un des composants du ME ou \u00e0 une solution de perfusion, comme l\u2019hypersensibilit\u00e9 \u00e0 l\u2019albumine, ou un diagnostic d\u2019intol\u00e9rance h\u00e9r\u00e9ditaire au fructose (IHF). Les sympt\u00f4mes \u00e9vocateurs de l\u2019IHF sont notamment des naus\u00e9es, des vomissements, des ballonnements, des crampes abdominales ou de la diarrh\u00e9e apr\u00e8s l\u2019ingestion de boissons ou d\u2019aliments sucr\u00e9s, ou une habitude d\u2019\u00e9viter les boissons ou les aliments sucr\u00e9s.", "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 \u00e0 l\u2019\u00e9culizumab, selon les informations locales relatives \u00e0 la prescription.", "language": 34, "languageDescription": "French (France)"}, {"id": 9799978, "uuid": "3c2ccb18-5737-4be1-9af0-37e58f6948b7", "attributeTranslation": "Contraindicaci\u00f3n del uso de eculizumab, de acuerdo con la informaci\u00f3n 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 % \u00e0 l\u2019\u00e9lectrocardiogramme de la visite de s\u00e9lection (visite 1) telle qu\u2019\u00e9valu\u00e9e 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 selecci\u00f3n (Visita 1), seg\u00fan 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 anomal\u00edas gen\u00e9ticas en el gen distrofina confirmado por el investigador con base en la revisi\u00f3n de la prueba gen\u00e9tica DMD:\na. Cualquier mutaci\u00f3n (deleci\u00f3n o duplicaci\u00f3n del ex\u00f3n, inserci\u00f3n o mutaci\u00f3n puntual) que afecte a cualquier ex\u00f3n entre el ex\u00f3n 9 y el 13 inclusive; O BIEN\nb. Una deleci\u00f3n que afecte tanto al ex\u00f3n 29 como al ex\u00f3n 30; O BIEN\nc. Una deleci\u00f3n 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 g\u00e9n\u00e9tiques suivantes du g\u00e8ne de la dystrophine, confirm\u00e9es par l\u2019investigateur sur la base de l\u2019examen des tests g\u00e9n\u00e9tiques de la DMD :\n\u2022 Toute mutation (d\u00e9l\u00e9tion d\u2019exon, duplication d\u2019exon, insertion ou mutation ponctuelle) affectant tout exon entre l\u2019exon 9 et l\u2019exon 13, inclusivement ; OU\n\u2022 Une d\u00e9l\u00e9tion qui affecte \u00e0 la fois l\u2019exon 29 et l\u2019exon 30 ; OU\n\u2022 Une d\u00e9l\u00e9tion qui affecte n\u2019importe 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": "Patolog\u00edas card\u00edacas, evaluadas por un cardi\u00f3logo pedi\u00e1trico en la visita de selecci\u00f3n (Visita 1):\na. Diagn\u00f3stico de miocarditis (p. ej., viral): basado o bien en antecedentes m\u00e9dicos o en hallazgos en im\u00e1genes card\u00edacas;\nb. Cualquier otro antecedente card\u00edaco y/o afecci\u00f3n y/o anomal\u00edas en las im\u00e1genes card\u00edacas que determinen que no debe incluirse al paciente en el estudio seg\u00fan el cardi\u00f3logo.", "language": 7, "languageDescription": "Spanish"}, {"id": 9799984, "uuid": "5138fa37-cc77-4a6b-8f28-a9ce246e3569", "attributeTranslation": "Les pathologies cardiaques, telles qu\u2019\u00e9valu\u00e9es par le cardiologue p\u00e9diatrique lors de la visite de s\u00e9lection (visite 1) :\na. Diagnostic de myocardite (p. ex. virale) : soit \u00e0 partir des ant\u00e9c\u00e9dents m\u00e9dicaux soit \u00e0 partir de d\u00e9couvertes issues des tests d\u2019imagerie ;\nb. Tout autre ant\u00e9c\u00e9dent cardiaque et/ou toute autre maladie et/ou anomalie d\u00e9couverte lors d\u2019un test d\u2019imagerie cardiaque qui d\u00e9termineraient que le patient ne devrait pas \u00eatre inclus dans l\u2019\u00e9tude 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 mec\u00e1nica card\u00edaca o respiratoria, o cualquier otra intervenci\u00f3n invasiva, si est\u00e1 indicado para la gesti\u00f3n de un episodio agudo seg\u00fan lo determine el cardi\u00f3logo en consulta con el investigador en la visita de selecci\u00f3n (Visita 1).", "language": 7, "languageDescription": "Spanish"}, {"id": 9799986, "uuid": "a30eb0ec-7a72-4c60-a2a0-afe714063303", "attributeTranslation": "Pas candidats \u00e0 une assistance cardiaque ou respiratoire m\u00e9canique, ou \u00e0 toute autre intervention invasive pour le diagnostic ou la prise en charge d\u2019un \u00e9v\u00e9nement cardiaque grave, tel que d\u00e9termin\u00e9 par le cardiologue en concertation avec l\u2019investigateur \u00e0 la visite de s\u00e9lection (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 exposici\u00f3n dentro de los 6 meses previos a la Selecci\u00f3n (Visita 1) a cualquier tratamiento dise\u00f1ado para aumentar la expresi\u00f3n de distrofina (incluidas, entre otras, omisiones de exones o translecturas de mutaci\u00f3n sin sentido). Estos tratamientos tambi\u00e9n se prohibir\u00e1n durante el per\u00edodo entre la Selecci\u00f3n (Visita 1) y el D\u00eda 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\u2022 Los participantes que hayan experimentado anteriormente falta de eficacia o intolerancia pueden inscribirse, siempre y cuando hayan recibido la \u00faltima dosis m\u00e1s de 6 meses antes de la selecci\u00f3n (Visita 1) o hayan rechazado estos tratamientos.\n\u2022 Los participantes que reciban estos tratamientos, de los cuales se cree que podr\u00edan obtener beneficios, no deben interrumpirlos para cumplir este criterio de exclusi\u00f3n e inscribirse en el estudio.", "language": 7, "languageDescription": "Spanish"}, {"id": 9799989, "uuid": "a2dbfcf7-442c-4cac-ae50-d47c1f45310d", "attributeTranslation": "Exposition dans les 6 mois pr\u00e9c\u00e9dant la s\u00e9lection (visite 1) \u00e0 un traitement con\u00e7u pour augmenter l\u2019expression de la dystrophine (y compris, mais sans s\u2019y limiter, saut d\u2019exon th\u00e9rapeutique et translecture non-sens). Ces traitements seront \u00e9galement interdits pendant la p\u00e9riode comprise entre la s\u00e9lection (visite 1) et le jour 1 (visite 3) ainsi que pendant les 52 premi\u00e8res semaines de l\u2019\u00e9tude. Veuillez noter que dans le cas de patients \u00e9ligibles pour ces traitements :\n\u2022 Il est possible d\u2019inclure des patients qui ont d\u00e9j\u00e0 subi un manque d\u2019efficacit\u00e9 ou une intol\u00e9rance, \u00e0 condition qu\u2019ils aient re\u00e7u leur derni\u00e8re dose plus de 6 mois avant la s\u00e9lection (visite 1), ou qui ont refus\u00e9 ces traitements.\n\u2022 Les patients recevant ces traitements pr\u00e9sum\u00e9s b\u00e9n\u00e9fiques ne doivent pas les interrompre afin de satisfaire \u00e0 ce crit\u00e8re et de s\u2019inscrire \u00e0 l\u2019\u00e9tude.", "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 ant\u00e9rieure d\u2019un m\u00e9dicament ou vaccin exp\u00e9rimental dans les 30 jours (ou selon les exigences locales) ou les 5 demi-vies (selon la dur\u00e9e la plus longue) lors de la s\u00e9lection (visite 1). Ces traitements seront \u00e9galement interdits pendant la p\u00e9riode comprise entre la s\u00e9lection (visite 1) et le jour 1 (visite 3) ainsi que pendant les 2 premi\u00e8res ann\u00e9es de l\u2019\u00e9tude.", "language": 34, "languageDescription": "French (France)"}, {"id": 9799991, "uuid": "07ba5d96-26e4-4652-ad50-8d4f6dbdfc84", "attributeTranslation": "Administraci\u00f3n previa de un f\u00e1rmaco o vacuna en fase de investigaci\u00f3n dentro de 30 d\u00edas (o seg\u00fan lo determinen los requisitos locales) o 5 semividas (el per\u00edodo que sea m\u00e1s extenso) en la Selecci\u00f3n (Visita 1). Estos tratamientos tambi\u00e9n se prohibir\u00e1n durante el per\u00edodo entre la Selecci\u00f3n (Visita 1) y el D\u00eda 1 (Visita 3), y los primeros 2 a\u00f1os 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 dificultar\u00edan la capacidad para seguir instrucciones, seg\u00fan el criterio del investigador, en la Selecci\u00f3n (Visita 1).", "language": 7, "languageDescription": "Spanish"}, {"id": 9799993, "uuid": "0a6e42cb-ae93-4df6-88f6-f6cc5c588585", "attributeTranslation": "D\u00e9ficience cognitive connue ou probl\u00e8mes comportementaux qui, de l\u2019avis de l\u2019investigateur, sont susceptibles d\u2019entraver la capacit\u00e9 \u00e0 se conformer aux instructions, lors de la s\u00e9lection (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 lesi\u00f3n no sanada en la Selecci\u00f3n (Visita 1) que, seg\u00fan la opini\u00f3n del investigador, pueda tener una repercusi\u00f3n sobre las pruebas funcionales; adem\u00e1s, las fracturas de miembros inferiores deben haberse consolidado durante al menos 3 meses previos a la Selecci\u00f3n (Visita 1).", "language": 7, "languageDescription": "Spanish"}, {"id": 9799995, "uuid": "2dd53d68-3834-4f0b-acd9-cf7f20eed41a", "attributeTranslation": "Toute blessure non cicatris\u00e9e lors de la s\u00e9lection (visite 1) qui, de l'avis de l\u2019investigateur, est susceptible d\u2019avoir une incidence sur les tests fonctionnels ; en outre, les fractures des membres inf\u00e9rieurs doivent \u00eatre consolid\u00e9es depuis au moins 3 mois avant la s\u00e9lection (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": "R\u00e9sultat positif au test AcN anti-AAV9, d\u2019apr\u00e8s le seuil fix\u00e9 par le laboratoire central, sur un \u00e9chantillon pr\u00e9lev\u00e9 lors de la s\u00e9lection (visite 1).", "language": 34, "languageDescription": "French (France)"}, {"id": 9799996, "uuid": "76dce107-5c83-4bc5-bf28-e9593c4e4311", "attributeTranslation": "Prueba positiva de AcN para VAA9, seg\u00fan el umbral determinado por el laboratorio central, de una muestra tomada en la Selecci\u00f3n (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": "Administraci\u00f3n de una vacuna de virus vivos atenuados dentro de los 30 d\u00edas previos a la Selecci\u00f3n (Visita 1). La administraci\u00f3n de una vacuna de virus vivos atenuados tambi\u00e9n estar\u00e1 prohibida durante 90 d\u00edas antes del D\u00eda 1 (Visita 3), durante los 90 d\u00edas previos a la administraci\u00f3n del PEI del A\u00f1o 2 y los primeros 2 meses tras cada administraci\u00f3n del PEI.", "language": 7, "languageDescription": "Spanish"}, {"id": 9799999, "uuid": "d38eb24d-3f01-461b-8282-b2686ffa9bb1", "attributeTranslation": "Administration d\u2019un vaccin vivant att\u00e9nu\u00e9 dans les 30 jours pr\u00e9c\u00e9dant la s\u00e9lection (visite 1). L\u2019administration d\u2019un vaccin vivant att\u00e9nu\u00e9 sera \u00e9galement interdite pendant les 90 jours pr\u00e9c\u00e9dant le jour 1 (visite 3), pendant 90 jours avant l\u2019administration du ME de l'ann\u00e9e 2 et pendant les 2 premiers mois apr\u00e8s 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/\u03bcl; 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 h\u00e9matologiques ou biochimiques \u00e0 la s\u00e9lection (visite 1). Une seule r\u00e9p\u00e9tition d\u2019analyse est permise pour la (les) valeur(s) en dehors des limites autoris\u00e9es afin de r\u00e9\u00e9valuer l\u2019\u00e9ligibilit\u00e9 :\na. Num\u00e9ration absolue de neutrophiles < 1 000 cellules/mm3 ;\nb. Plaquettes < 150 x 103/\u03bcl ;\nc. Cystatine C > 1,2 x LSN ;\nd. Immunoglobulines M anti-virus de l\u2019h\u00e9patite A (IgM anti-VHA) positives, antig\u00e8ne de surface de l\u2019h\u00e9patite B (AgHBs) et/ou anticorps anti-h\u00e9patite C (Ac anti-VHC) ;\ne. Marqueurs d\u2019inflammation h\u00e9patique ou de cirrhose manifeste ou occulte, mise en \u00e9vidence par un ou plusieurs des param\u00e8tres suivants :\n1. Temps de prothrombine (PT) > limite sup\u00e9rieure de la normale (LSN) ; rapport normalis\u00e9 international (INR) prolong\u00e9 > LSN ;\n2. GLDH > 2 x LSN ;\n3. Bilirubine totale > 1,5 x LSN (sauf si le patient a des ant\u00e9c\u00e9dents de maladie de Gilbert) et bilirubine directe > 0,5 mg/dl ;\n4. Gamma-glutamyl transf\u00e9rase (GGT) > 1,5 x LSN.", "language": 34, "languageDescription": "French (France)"}, {"id": 9800000, "uuid": "3ed641d9-a715-4ff9-95ae-4652309e2daa", "attributeTranslation": "Anomal\u00edas en los perfiles de hematolog\u00eda o bioqu\u00edmica en la Selecci\u00f3n (Visita 1). Se permite una repetici\u00f3n \u00fanica para los valores fuera de los l\u00edmites admisibles a fin de volver a evaluar la elegibilidad:\na. recuento absoluto de neutr\u00f3filos <1000 c\u00e9lulas/mm3;\nb. plaquetas <150 x 103/\u03bcl;\nc. cistatina C >1,2 x LSN;\nd. inmunoglobulina M contra el virus de la hepatitis A (anti-HAV), ant\u00edgeno de superficie de la hepatitis B (HbsAg) o anticuerpo de la hepatitis C (HCVAb) positivos;\ne. marcadores de inflamaci\u00f3n hep\u00e1tica o cirrosis evidente u oculta, tal como se demuestra en uno o m\u00e1s de los siguientes:\n1. tiempo de protrombina (TP) > l\u00edmite superior de la normalidad (LSN); \u00edndice internacional normalizado (INR) > LSN;\n2. GLDH >2 x LSN;\n3. bilirrubina total >1,5 x LSN (a menos que el participante tenga antecedentes de s\u00edndrome de Gilbert) y bilirrubina directa >0,5 mg/dl;\n4. gamma-glutamil transferasa (GGT) >1,5 x LSN.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 1373211, "number": 9, "principalExclusionCriteria": "Other acute or chronic medical or psychiatric condition at Screening (Visit 1), including recent (within the past year) or active suicidal ideation or behavior (using screening by the Child Behavior Check List (CBCL) and determined by the Investigator, as described in Section 8.2.13) or laboratory abnormality that may increase the risk associated with study participation or IP administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the participant inappropriate for entry into this study.", "principalExclusionCriteriaTranslations": [{"id": 9800002, "uuid": "4d2d1812-7f20-4886-b751-0f21e09454b2", "attributeTranslation": "Cualquier otra afecci\u00f3n m\u00e9dica o psiqui\u00e1trica aguda o cr\u00f3nica en la Selecci\u00f3n (Visita 1), incluidas ideas o conductas de suicidio recientes (en el a\u00f1o anterior) (mediante el uso de la selecci\u00f3n por parte del inventario del comportamiento de ni\u00f1os [CBCL] y determinadas por el investigador, seg\u00fan lo descrito en el apartado 8.2.11), o anomal\u00edas en los an\u00e1lisis cl\u00ednicos que pudieran aumentar el riesgo asociado a la participaci\u00f3n en el estudio o a la administraci\u00f3n del PEI, o que pudieran interferir en la interpretaci\u00f3n de los resultados del estudio y que, en la opini\u00f3n del investigador, har\u00edan que el participante fuera inapropiado para ingresar en este estudio.", "language": 7, "languageDescription": "Spanish"}, {"id": 9800003, "uuid": "4d2d1812-7f20-4886-b751-0f21e09454b2", "attributeTranslation": "Autre pathologie m\u00e9dicale ou psychiatrique aigu\u00eb ou chronique \u00e0 la s\u00e9lection (visite 1), notamment id\u00e9es ou comportements suicidaires actifs ou r\u00e9cents au cours de l\u2019ann\u00e9e pass\u00e9e (avec d\u00e9pistage \u00e0 l\u2019aide de l\u2019\u00c9chelle du comportement de l\u2019enfant (CBCL) et d\u00e9termin\u00e9e par l\u2019investigateur comme d\u00e9crit \u00e0 la section 8.2.13), ou anomalies biologiques susceptibles d\u2019accro\u00eetre les risques associ\u00e9s \u00e0 la participation \u00e0 l\u2019\u00e9tude ou \u00e0 l\u2019administration du ME, ou susceptibles de nuire \u00e0 l\u2019interpr\u00e9tation des r\u00e9sultats de l\u2019\u00e9tude et qui, de l\u2019avis de l\u2019investigateur, rendraient le patient in\u00e9ligible pour int\u00e9grer cette \u00e9tude.", "language": 34, "languageDescription": "French (France)"}]}]}, "endPoint": {"primaryEndPoints": [{"id": 938788, "number": 1, "endPoint": "Change from Baseline at Week 52 in the NSAA total score.", "isPrimary": true, "endPointTranslations": [{"id": 9799951, "uuid": "70899929-7419-47f0-8c99-c2fd83533963", "attributeTranslation": "Variation par rapport \u00e0 la valeur initiale du score total NSAA \u00e0 la semaine 52.", "language": 34, "languageDescription": "French (France)"}, {"id": 9799950, "uuid": "70899929-7419-47f0-8c99-c2fd83533963", "attributeTranslation": "Cambio con respecto al valor de referencia en la Semana 52 en la puntuaci\u00f3n total de la NSAA.", "language": 7, "languageDescription": "Spanish"}]}], "secondaryEndPoints": [{"id": 938789, "number": 1, "endPoint": "Change from Baseline in percent normal mini-dystrophin expression level in biceps brachii muscle biopsies at Day 360 using a liquid chromatography mass spectrometry (LC-MS) assay.", "isPrimary": false, "endPointTranslations": [{"id": 9799953, "uuid": "2c26b0ca-e7df-4b14-8740-53bff20bc67f", "attributeTranslation": "Variation par rapport \u00e0 la valeur initiale du niveau d'expression normal de la mini-dystrophine en pourcentage dans les biopsies de muscle biceps brachial au jour 360 \u00e0 l\u2019aide d\u2019un dosage par chromatographie en phase liquide coupl\u00e9e \u00e0 la spectrom\u00e9trie de masse (LC-MS).", "language": 34, "languageDescription": "French (France)"}, {"id": 9799952, "uuid": "2c26b0ca-e7df-4b14-8740-53bff20bc67f", "attributeTranslation": "Cambio con respecto al valor de referencia en el porcentaje del nivel de expresi\u00f3n de minidistrofina en biopsias del m\u00fasculo b\u00edceps braquial en el D\u00eda 360 mediante el uso de un ensayo de cromatograf\u00eda l\u00edquida con espectrometr\u00eda de masa (Liquid Chromatography Mass Spectrometry, LC-MS).", "language": 7, "languageDescription": "Spanish"}]}, {"id": 938790, "number": 2, "endPoint": "Change from Baseline in percent of muscle fibers expressing mini-dystrophin in biceps brachii muscle biopsies at Day 360 as assessed by immunofluorescence.", "isPrimary": false, "endPointTranslations": [{"id": 9799954, "uuid": "5aa556b1-fd9d-496f-9679-2a1d50de6b61", "attributeTranslation": "Cambio con respecto al valor de referencia en el porcentaje de fibras musculares que expresan minidistrofina en biopsias del m\u00fasculo b\u00edceps braquial en el D\u00eda 360 seg\u00fan la evaluaci\u00f3n de inmunofluorescencia.", "language": 7, "languageDescription": "Spanish"}, {"id": 9799955, "uuid": "5aa556b1-fd9d-496f-9679-2a1d50de6b61", "attributeTranslation": "Variation par rapport \u00e0 la valeur initiale du pourcentage de fibres musculaires exprimant la mini-dystrophine dans les biopsies de muscle biceps brachial au jour 360, d\u2019apr\u00e8s l\u2019\u00e9valuation par immunofluorescence.", "language": 34, "languageDescription": "French (France)"}]}, {"id": 938791, "number": 3, "endPoint": "Change from Baseline at Week 52 in serum CK concentration.", "isPrimary": false, "endPointTranslations": [{"id": 9799956, "uuid": "dab8527e-8d60-482a-a4f8-5728029bf683", "attributeTranslation": "Variation par rapport \u00e0 la valeur initiale de la concentration s\u00e9rique de CK \u00e0 la semaine 52.", "language": 34, "languageDescription": "French (France)"}, {"id": 9799957, "uuid": "dab8527e-8d60-482a-a4f8-5728029bf683", "attributeTranslation": "Cambio con respecto al valor de referencia en la Semana 52 en la concentraci\u00f3n de CK s\u00e9rica.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 938792, "number": 4, "endPoint": "Number of skills gained at Week 52 based on the individual items of the NSAA.", "isPrimary": false, "endPointTranslations": [{"id": 9799959, "uuid": "92f3c413-5120-44fe-8fda-330225d798b5", "attributeTranslation": "Cantidad de habilidades adquiridas en la Semana 52 seg\u00fan los elementos individuales de la NSAA.", "language": 7, "languageDescription": "Spanish"}, {"id": 9799958, "uuid": "92f3c413-5120-44fe-8fda-330225d798b5", "attributeTranslation": "Nombre de comp\u00e9tences acquises \u00e0 la semaine 52 d\u2019apr\u00e8s les items individuels de l\u2019\u00e9chelle NSAA.", "language": 34, "languageDescription": "French (France)"}]}, {"id": 938793, "number": 5, "endPoint": "Number of skills either improved or maintained at Week 52 based on the individual items of the NSAA.", "isPrimary": false, "endPointTranslations": [{"id": 9799961, "uuid": "cedc92cc-628b-47fe-9337-fdb971553cf6", "attributeTranslation": "Nombre de comp\u00e9tences am\u00e9lior\u00e9es ou maintenues \u00e0 la semaine 52 d\u2019apr\u00e8s les items individuels de l\u2019\u00e9chelle NSAA.", "language": 34, "languageDescription": "French (France)"}, {"id": 9799960, "uuid": "cedc92cc-628b-47fe-9337-fdb971553cf6", "attributeTranslation": "Cantidad de habilidades mejoradas o mantenidas en la Semana 52 seg\u00fan los elementos individuales de la NSAA.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 938794, "number": 6, "endPoint": "Change from Baseline at Week 52 in the 10 meter run/walk velocity.", "isPrimary": false, "endPointTranslations": [{"id": 9799963, "uuid": "5dd32f0c-b37b-4bb9-88d6-51ba6cb9c82b", "attributeTranslation": "Variation par rapport \u00e0 la valeur initiale de la vitesse de marche/course sur 10 m\u00e8tres \u00e0 la semaine 52.", "language": 34, "languageDescription": "French (France)"}, {"id": 9799962, "uuid": "5dd32f0c-b37b-4bb9-88d6-51ba6cb9c82b", "attributeTranslation": "Cambio con respecto al valor de referencia en la Semana 52 en la velocidad de carrera/caminata de 10 metros.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 938795, "number": 7, "endPoint": "Change from Baseline at Week 52 in the rise from floor velocity.", "isPrimary": false, "endPointTranslations": [{"id": 9799964, "uuid": "2b8c9f40-994a-47a1-ac10-29321fbd942c", "attributeTranslation": "Cambio con respecto al valor de referencia en la Semana 52 en la velocidad de elevaci\u00f3n desde el piso.", "language": 7, "languageDescription": "Spanish"}, {"id": 9799965, "uuid": "2b8c9f40-994a-47a1-ac10-29321fbd942c", "attributeTranslation": "Variation par rapport \u00e0 la valeur initiale de la vitesse du lever depuis la position couch\u00e9e \u00e0 la semaine 52.", "language": 34, "languageDescription": "French (France)"}]}, {"id": 938796, "number": 8, "endPoint": "Change from Baseline at Week 52 in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale (Pediatric Parent).", "isPrimary": false, "endPointTranslations": [{"id": 9799966, "uuid": "915d8794-2b43-446d-8dd9-03eb129829a2", "attributeTranslation": "Variation par rapport \u00e0 la valeur initiale du score de l\u2019instrument de collecte de donn\u00e9es sur les r\u00e9sultats p\u00e9diatriques (PODCI) modifi\u00e9, \u00e0 la semaine 52 : Transfer and Basic Mobility Core Scale (Pediatric Parent) [\u00c9chelle g\u00e9n\u00e9rique d\u2019\u00e9valuation des transferts et de la mobilit\u00e9 de base (Parent p\u00e9diatrique)].", "language": 34, "languageDescription": "French (France)"}, {"id": 9799967, "uuid": "915d8794-2b43-446d-8dd9-03eb129829a2", "attributeTranslation": "Cambio con respecto al valor de referencia en la Semana 52 en el instrumento de recopilaci\u00f3n de datos de resultados pedi\u00e1tricos (PODCI) modificado: escala principal de transferencia y movilidad b\u00e1sica (padres pedi\u00e1tricos).", "language": 7, "languageDescription": "Spanish"}]}, {"id": 938797, "number": 9, "endPoint": "Change from Baseline at Week 52 in the Modified PODCI: Sports and Physical Functioning Core Scale (Pediatric Parent).", "isPrimary": false, "endPointTranslations": [{"id": 9799969, "uuid": "031c6ff6-1958-4ac2-87c1-6360708d255b", "attributeTranslation": "Variation par rapport \u00e0 la valeur initiale du score PODCI modifi\u00e9 \u00e0 la semaine 52 : Sports and Physical Functioning Core Scale (Pediatric Parent) [\u00c9chelle g\u00e9n\u00e9rique d\u2019\u00e9valuation pour les sports et activit\u00e9s physiques (Parent p\u00e9diatrique)].", "language": 34, "languageDescription": "French (France)"}, {"id": 9799968, "uuid": "031c6ff6-1958-4ac2-87c1-6360708d255b", "attributeTranslation": "Cambio con respecto al valor de referencia en la Semana 52 en el PODCI modificado: escala principal de deportes y funcionamiento f\u00edsico (padres pedi\u00e1tricos).", "language": 7, "languageDescription": "Spanish"}]}]}, "trialDuration": {"estimatedGlobalEndDate": "2040-01-10", "estimatedEndDate": "2039-12-05", "estimatedRecruitmentStartDate": "2020-11-05"}, "sourceOfMonetarySupport": [{"id": 86930, "organisationName": "Pfizer Inc."}], "populationOfTrialSubjects": {"ageRanges": [{"id": 327171, "ageRangeCategoryCode": "2", "ageRangeCategory": "2"}], "ageRangeSecondaryIds": [{"id": 327173, "ageRangeCategoryCode": "2", "ctAgeRangeCode": "5", "ageRangeCategory": "2", "ctAgeRange": "5"}, {"id": 327172, "ageRangeCategoryCode": "2", "ctAgeRangeCode": "4", "ageRangeCategory": "2", "ctAgeRange": "4"}], "clinicalTrialGroups": [{"code": "2", "name": "Patients"}], "isFemaleSubjects": false, "isMaleSubjects": true, "isVulnerablePopulationSelected": true}, "individualParticipantData": {"planToShareIPD": "1", "planDescription": "Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. 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"Spanish"}], "shortTitle": "DYNE251-DMD-201", "secondaryIdentifyingNumbers": {"nctNumber": {"id": 381977, "number": "NCT05524883"}, "additionalRegistries": []}}, "trialInformation": {"trialCategory": {"isLowIntervention": false, "trialPhase": "7", "trialCategory": "2", "justificationForTrialCategory": "This is a Phase 1/2", "trialCategoryId": 76447}, "medicalCondition": {"partIMedicalConditions": [{"id": 87343, "medicalCondition": "Duchenne muscular dystrophy", "medicalConditionTranslations": [{"id": 6475755, "uuid": "8fc66e4f-0300-4a34-93a8-bd022cdad18b", "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": "6", "trialScopeId": 252623}, {"code": "9", "trialScopeId": 252622}, {"code": "5", "trialScopeId": 252619}, {"code": "7", "trialScopeId": 252621}, {"code": "4", "trialScopeId": 252620}], "mainObjective": "\"To evaluate the safety and tolerability of multiple IV doses of DYNE-251 administered to participants with DMD\n\nTo evaluate dystrophin protein levels in muscle tissue following multiple IV doses of DYNE-251 administered to participants with DMD \"", "mainObjectiveTranslations": [{"id": 6475770, "uuid": "f98d1111-3ac8-46f4-95eb-d84ae2675cc4", "attributeTranslation": "Evaluar la seguridad y la tolerabilidad de dosis IV m\u00faltiples de DYNE-251 administradas a pacientes con DMD.\n\nEvaluar las concentraciones de prote\u00edna distrofina en el tejido muscular despu\u00e9s de administrar varias dosis IV de DYNE-251 a pacientes con DMD.", "language": 7, "languageDescription": "Spanish"}], "secondaryObjectives": [{"id": 287626, "number": 1, "secondaryObjective": "To evaluate the effects on muscle tissue exon skipping, percent dystrophin-positive fibers (PDPFs), and blood creatine kinase (CK) following multiple IV doses of DYNE-251 administered to participants with DMD", "secondaryObjectiveTranslations": [{"id": 6475772, "uuid": "d563765d-c0e2-4828-b16b-a861b6fb5ab7", "attributeTranslation": "Evaluar los efectos sobre la omisi\u00f3n de exones en el tejido muscular, el porcentaje de fibras positivas para la distrofina (PDPF, percent dystrophin-positive fibers) y la creatina cinasa (CK) en sangre tras la administraci\u00f3n de dosis IV m\u00faltiples de DYNE-251 a pacientes con DMD.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 287627, "number": 2, "secondaryObjective": "To evaluate muscle function following multiple IV doses of DYNE-251 administered to participants with DMD", "secondaryObjectiveTranslations": [{"id": 6475773, "uuid": "75234db2-1b7b-438c-a6a7-5ac9bdbfa51d", "attributeTranslation": "Evaluar la funci\u00f3n muscular tras la administraci\u00f3n de dosis IV m\u00faltiples de DYNE-251 a pacientes con DMD.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 287628, "number": 3, "secondaryObjective": "To evaluate plasma and muscle tissue PK following multiple IV doses of DYNE-251 administered to participants with DMD", "secondaryObjectiveTranslations": [{"id": 6475774, "uuid": "bc46ea72-e900-410d-8896-9d583a02a225", "attributeTranslation": "Evaluar la farmacocin\u00e9tica en plasma y tejido muscular tras la administraci\u00f3n de dosis IV m\u00faltiples de DYNE 251 a pacientes con DMD.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 287629, "number": 4, "secondaryObjective": "To evaluate the immunogenicity of multiple IV doses of DYNE-251 administered to participants with DMD", "secondaryObjectiveTranslations": [{"id": 6475775, "uuid": "a43136ad-7f2f-4a20-8a73-922e0ac5d932", "attributeTranslation": "Evaluar la inmunogenicidad de dosis IV m\u00faltiples de DYNE-251 administradas a pacientes con DMD.", "language": 7, "languageDescription": "Spanish"}]}]}, "eligibilityCriteria": {"principalInclusionCriteria": [{"id": 555356, "number": 1, "principalInclusionCriteria": "\u2022  Age 4 to 16 years inclusive, at the time of informed consent/assent. \u2022  Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping. \u2022  Upper extremity muscle group that is amenable to muscle biopsy. \u2022  Brooke Upper Extremity Scale score of 1 or 2.  \u2022  Ambulatory or non-ambulatory. A non-ambulatory participant must have been non-ambulatory for <2 years before enrolment. \u2022  Receiving a stable dosage of glucocorticoids for at least 12 weeks prior to the start of study drug administration. \u2022  Left ventricular ejection fraction of \u226550% by echocardiogram or \u2265 55% by cardiac magnetic resonance imaging (MRI).", "principalInclusionCriteriaTranslations": [{"id": 6475769, "uuid": "25a5bc45-7c29-4f1c-bf5d-9b4a75bd421c", "attributeTranslation": "\u2022 Edad de 4 a 16 a\u00f1os, inclusive, en el momento de otorgar el asentimiento/consentimiento informado.\n\u2022 Var\u00f3n con un diagn\u00f3stico confirmado de DMD y con una mutaci\u00f3n en el gen de la distrofina caracterizada por deleci\u00f3n del ex\u00f3n susceptible de omisi\u00f3n del ex\u00f3n 51.\n\u2022 Grupo muscular de las extremidades superiores susceptible de biopsia muscular.\n\u2022 Puntuaci\u00f3n de 1 o 2 en la escala de Brooke para las extremidades superiores. \n\u2022 Paciente deambulante o no deambulante. Los participantes no deambulantes deben haberlo sido durante <2 a\u00f1os antes de la inclusi\u00f3n en el estudio.\n\u2022 Haber recibido una pauta de tratamiento estable de glucocorticoides durante al menos 12 semanas antes del inicio de la administraci\u00f3n del f\u00e1rmaco del estudio.\n\u2022 Fracci\u00f3n de eyecci\u00f3n del ventr\u00edculo izquierdo \u226550 % determinada mediante ecocardiograma o \u226555 % determinada mediante resonancia magn\u00e9tica nuclear (RMN) card\u00edaca.", "language": 7, "languageDescription": "Spanish"}]}], "principalExclusionCriteria": [{"id": 955551, "number": 1, "principalExclusionCriteria": "\u2022  Uncontrolled clinical symptoms and signs of congestive heart failure (CHF). \u2022  Any change in prophylaxis/treatment for CHF within 3 months prior to the start of study treatment. \u2022  History of major surgical procedure within 12 weeks prior to the start of study drug administration or an expectation of a major surgical procedure during the study. \u2022  Requirement of daytime ventilator assistance. \u2022  Percent predicted FVC <40 % (applies only for participants who are age \u22657 years). \u2022  Receipt of eteplirsen, or alternative exon-skipping/dystrophinmodifying therapy, within 12 weeks of randomization. \u2022  Receipt of non-exon skipping investigational drug within 4 months before the start of study drug administration. \u2022  Receipt of gene therapy at any time.", "principalExclusionCriteriaTranslations": [{"id": 6475767, "uuid": "2bec8b91-bdb9-40df-8bd8-4bbe781f446d", "attributeTranslation": "\u2022 Signos y s\u00edntomas cl\u00ednicos no controlados de insuficiencia card\u00edaca congestiva (ICC).\n\u2022 Cualquier cambio en la profilaxis/el tratamiento para la ICC en los 3 meses anteriores al inicio del tratamiento del estudio.\n\u2022 Antecedentes de intervenci\u00f3n quir\u00fargica mayor en las 12 semanas anteriores al inicio de la administraci\u00f3n del f\u00e1rmaco del estudio o expectativa de que se tenga que practicar una intervenci\u00f3n quir\u00fargica mayor durante el estudio.\n\u2022 Necesidad de asistencia respiratoria diurna.\n\u2022 Porcentaje de FVC previsto <40 % (se aplica solo a los participantes con una edad \u22657 a\u00f1os).\n\u2022 Haber recibido eteplirs\u00e9n o un tratamiento alternativo de omisi\u00f3n de ex\u00f3n/modificador de la distrofina en las 12 semanas anteriores a la aleatorizaci\u00f3n.\n\u2022 Haber recibido un f\u00e1rmaco en investigaci\u00f3n no dirigido a la omisi\u00f3n del ex\u00f3n en los 4 meses anteriores al inicio de la administraci\u00f3n del f\u00e1rmaco del estudio.\n\u2022 Recibir terapia g\u00e9nica en cualquier momento.", "language": 7, "languageDescription": "Spanish"}]}]}, "endPoint": {"primaryEndPoints": [{"id": 648346, "number": 1, "endPoint": "Number and proportion of participants with treatment-emergent adverse events (TEAEs), treatment  emergent serious adverse events (TESAEs), TEAEs considered related to study drug, and TEAEs leading to discontinuation from study drug and discontinuation from the study.", "isPrimary": true, "endPointTranslations": [{"id": 6475756, "uuid": "0ec2821e-8d01-4095-a5fa-22e0cffe2839", "attributeTranslation": "N\u00famero y proporci\u00f3n de pacientes con acontecimientos adversos aparecidos durante el tratamiento (AAAT), acontecimientos adversos graves aparecidos durante el tratamiento (AAGAT), AAAT que se consideren relacionados con el f\u00e1rmaco del estudio y AAAT que motiven la suspensi\u00f3n del f\u00e1rmaco del estudio y la retirada del estudio.", "language": 7, "languageDescription": "Spanish"}]}, {"id": 648347, "number": 2, "endPoint": "Change from Baseline in dystrophin protein levels in muscle tissue as determined by Western blot analysis at Week 25", "isPrimary": true, "endPointTranslations": [{"id": 6475757, "uuid": "d0ed0588-a579-4476-a227-b87c308456bb", "attributeTranslation": "Variaci\u00f3n de la concentraci\u00f3n de prote\u00edna distrofina en el tejido muscular, determinada mediante inmunotransferencia, entre el momento basal y la semana 25.", "language": 7, "languageDescription": "Spanish"}]}], "secondaryEndPoints": [{"id": 648348, "number": 1, "endPoint": "Muscle Tissue Endpoints: Cohorts dosed Q4W or Q8W with a second biopsy at Week 25", "isPrimary": false, "endPointTranslations": [{"id": 6475758, "uuid": "5dca5c53-30d3-47b7-bdcb-e029dc4e387e", "attributeTranslation": "Criterios de valoraci\u00f3n relacionados con el tejido muscular:\nCohortes que recibieron dosis C4S o C8S con una segunda biopsia en la semana 25", "language": 7, "languageDescription": "Spanish"}]}, {"id": 648349, "number": 2, "endPoint": "Change from Baseline at Week 25: -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue", "isPrimary": false, "endPointTranslations": [{"id": 6475759, "uuid": "c6fd2b68-ae3b-4b24-a07b-c627d79e8e62", "attributeTranslation": "Variaci\u00f3n con respecto al momento basal en la semana 25:\n- Niveles de omisi\u00f3n del ex\u00f3n 51 en el tejido muscular\n- PFPD en el tejido muscular\n- Concentraci\u00f3n de PMO en el tejido muscular", "language": 7, "languageDescription": "Spanish"}]}, {"id": 648350, "number": 3, "endPoint": "Change from baseline up to Week 145: -blood CK levels", "isPrimary": false, "endPointTranslations": [{"id": 6475760, "uuid": "f0759e2d-808a-467b-9ef2-12c643d635a3", "attributeTranslation": "Variaci\u00f3n con respecto al momento basal en la semana 145:\n- Niveles de creatinina quinasa en sangre", "language": 7, "languageDescription": "Spanish"}]}, {"id": 648351, "number": 4, "endPoint": "Cohorts dosed Q8W with a second biopsy at Week 49 Change from Baseline at Week 49 in: -dystrophin protein levels in muscle tissue -exon 51 skipping levels in muscle tissue -PDPF in muscle tissue -PMO concentration in muscle tissue", "isPrimary": false, "endPointTranslations": [{"id": 6475761, "uuid": "56a8ab2a-75c9-4c4d-b541-9444c1b3f58c", "attributeTranslation": "Cohortes que recibieron dosis C8S con una segunda biopsia en la semana 49\nVariaci\u00f3n con respecto al momento basal en la semana 49:\n- Niveles de prote\u00edna distrofina en el tejido muscular\n- Niveles de omisi\u00f3n del ex\u00f3n 51 en el tejido muscular\n- PFPD en el tejido muscular\n- Concentraci\u00f3n de PMO en el tejido muscular", "language": 7, "languageDescription": "Spanish"}]}, {"id": 648352, "number": 5, "endPoint": "Change from Baseline up to Week 145 in: -blood CK level", "isPrimary": false, "endPointTranslations": [{"id": 6475762, "uuid": "47f24d3d-934d-4bda-88b8-ca864775ce17", "attributeTranslation": "Variaci\u00f3n con respecto al momento basal en la semana 145:\n- Niveles de creatinina quinasa en sangre", "language": 7, "languageDescription": "Spanish"}]}, {"id": 648353, "number": 6, "endPoint": "Functional Endpoints: All cohorts", "isPrimary": false, "endPointTranslations": [{"id": 6475763, "uuid": "bf071692-87f2-4aa4-845a-3072abcb7619", "attributeTranslation": "Criterios de valoraci\u00f3n funcionales:\nTodas las cohortes", "language": 7, "languageDescription": "Spanish"}]}, {"id": 648354, "number": 7, "endPoint": "Change from Baseline up to Week 145 in:  -PUL scale V 2.0 score  -%pFVC -NSAA total score, TTR, 10MRW, and SV95C in ambulatory participants", "isPrimary": false, "endPointTranslations": [{"id": 6475764, "uuid": "5b825069-4c1f-4907-a097-323121355bb5", "attributeTranslation": "Variaci\u00f3n con respecto al momento basal en la semana 145: \n-Puntuaci\u00f3n en la versi\u00f3n 2.0 de la escala PUL \n- Porcentaje de FVCp\n-Puntuaci\u00f3n total de la NSAA, TTR, 10MRW y SV95C en participantes deambulantes", "language": 7, "languageDescription": "Spanish"}]}, {"id": 648355, "number": 8, "endPoint": "Plasma Endpoints: All cohorts -Cmax -tmax -AUCtlast -AUC\u221e -\u03bbZ -t\u00bd -CL -Vz and Vss, if appropriate", "isPrimary": false, "endPointTranslations": [{"id": 6475765, "uuid": "f643e594-f2f3-45b1-8e1d-9957e041ec09", "attributeTranslation": "Criterios de valoraci\u00f3n plasm\u00e1ticos:\nTodas las cohortes\n\u2013 Cm\u00e1x.\n\u2013 tm\u00e1x.\n- AUCt\u00falt.\n- AUC\u221e\n- \u03bbZ\n-t\u00bd\n- CL\n- Vz y Vss, si procede", "language": 7, "languageDescription": "Spanish"}]}, {"id": 648356, "number": 9, "endPoint": "Immunogenicity Endpoint: All cohorts -Incidence of ADAs", "isPrimary": false, "endPointTranslations": [{"id": 6475766, "uuid": "b8192009-9123-4b64-9fbb-f128220892b7", "attributeTranslation": "Criterio de valoraci\u00f3n relacionado con la inmunogenicidad:\nTodas las cohortes\n- Incidencia de ACF", "language": 7, "languageDescription": "Spanish"}]}]}, "trialDuration": {"estimatedGlobalEndDate": "2027-12-04", "estimatedEndDate": "2027-12-04", "estimatedRecruitmentStartDate": "2023-01-30"}, "sourceOfMonetarySupport": [{"id": 61197, "organisationName": "Dyne Therapeutics, Inc."}], "populationOfTrialSubjects": {"ageRanges": [{"id": 232116, "ageRangeCategoryCode": "2", "ageRangeCategory": "2"}], "ageRangeSecondaryIds": [], "clinicalTrialGroups": [{"code": "2", "name": "Patients"}], "isFemaleSubjects": false, "isMaleSubjects": true, "isVulnerablePopulationSelected": true}, "individualParticipantData": {"planToShareIPD": "2"}}, "protocolInformation": {"studyDesign": {"periodDetails": []}}, "scientificAdviceAndPip": {"scientificAdvices": [], "paediatricInvestigationPlan": []}, "associatedClinicalTrials": [], "references": [], "pubmedCode": [], "pubmedUrl": []}, "assessmentOutcome": "acceptable", "assessmentOutcomeDate": "2025-05-16", "conclusionDate": "2025-05-16", "therapeuticAreas": [{"code": "5", "name": "Diseases [C] - Musculoskeletal Diseases [C05]"}], "medicalConditions": [{"id": 87343, "medicalCondition": "Duchenne muscular dystrophy", "isConditionRareDisease": true}], "sponsors": [{"id": 93237, "primary": true, "publicContacts": [{"id": 278423, "type": "Public", "functionalName": "Dyne Clinical Trials", 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{"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 m\u00faltiple ascendente, para la determinaci\u00f3n de la dosis de SRP-5051, seguido de ampliaci\u00f3n de la dosis, en pacientes con distrofia muscular de Duchenne susceptible al tratamiento de omisi\u00f3n del ex\u00f3n 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 pati\u00ebnten 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 determinaci\u00f3n de la dosis de vesleteplirs\u00e9n (SRP 5051) (parte A) y, a continuaci\u00f3n, de la eficacia de la dosis (parte B) en participantes con distrofia muscular de Duchenne susceptible al tratamiento de omisi\u00f3n del ex\u00f3n 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 m\u00faltiples dosis ascendentes de SRP-5051 (4 mg/kg, 10 mg/kg, 20 mg/kg y 30 mg/kg), administrado por v\u00eda intravenosa (IV) cada 4 semanas (C4S), y determinar la dosis m\u00e1xima tolerada (MTD).\nParte B: Evaluar el nivel de la prote\u00edna distrofina en los tejidos de los m\u00fasculos esquel\u00e9ticos despu\u00e9s del tratamiento con SRP-5051, administrado por v\u00eda intravenosa cada 4 semanas a las dosis seleccionadas seg\u00fan 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 \u2022 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 \u2022 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 \u2022 To determine the PK of SRP-5051 in plasma and urine \u2022 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\u2022 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\u2022 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\u2022 Het bepalen van de PK van SRP-5051 in plasma en urine\n\u2022 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 farmacocin\u00e9tica (PK) de la SRP-5051 en plasma y orina, y del metabolito mayor SRP-5051A en plasma, en cada una de las dosis ascendentes m\u00faltiples ya mencionadas de la SRP-5051, administrada por v\u00eda intravenosa cada 4 semanas.\nParte B\n\u2022\tEvaluar el nivel de omisi\u00f3n de ex\u00f3n en los tejidos de los m\u00fasculos esquel\u00e9ticos despu\u00e9s del tratamiento con SRP-5051, administrado por v\u00eda intravenosa cada 4 semanas a las dosis seleccionadas seg\u00fan los datos de la parte A.\n\u2022\tEvaluar la seguridad y la tolerabilidad continuas del tratamiento con SRP 5051, administrado por v\u00eda intravenosa cada 4 semanas a las dosis seleccionadas seg\u00fan los datos de la parte A. \n\u2022\tDeterminar la farmacocin\u00e9tica de SRP-5051 en plasma y orina.\n\u2022\tEvaluar mediante la inmunohistoqu\u00edmica el porcentaje de fibras distrofina positivas (PDPF) y la intensidad media tras el tratamiento con SRP-5051, administrado por v\u00eda intravenosa cada 4 semanas a partir de las dosis seleccionadas seg\u00fan 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 inclusi\u00f3n para participantes previamente tratados con vesleteplirs\u00e9n:\n-\tHaber recibido tratamiento previo con vesleteplirs\u00e9n 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-na\u00efve 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] \u226540% of predicted and no requirement for nocturnal ventilation).", "principalInclusionCriteriaTranslations": [{"id": 3697018, "uuid": "e4d79ff0-8e61-4e84-b57e-7146cdb2d35f", "attributeTranslation": "Inclusiecriteria voor behandelingsna\u00efeve deelnemers die worden ge\u00efncludeerd 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 corticostero\u00efden 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 corticostero\u00efden ontvangen.\n- Heeft een stabiele longfunctie (geforceerde vitale capaciteit [FVC] \u2265 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 inclusi\u00f3n para participantes sin tratamiento previo que se incluyan en la parte B:\n-\tTener un diagn\u00f3stico gen\u00e9tico de distrofia muscular de Duchenne (DMD) y una mutaci\u00f3n de deleci\u00f3n fuera de marco de lectura del gen DMD susceptible al tratamiento de omisi\u00f3n del ex\u00f3n 51.\n-\tHaber recibido una dosis estable de corticoesteroides orales durante al menos 12 semanas antes de la administraci\u00f3n del f\u00e1rmaco 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 administraci\u00f3n del f\u00e1rmaco del estudio.\n-\t- Tener una funci\u00f3n pulmonar estable (capacidad vital forzada [FVC] \u226540 % del valor previsto y no necesitar respiraci\u00f3n 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 exclusi\u00f3n para participantes tratados previamente con vesleteplirs\u00e9n y nuevos participantes que se incluyan en la parte B:\n-\tPresencia de otra enfermedad cl\u00ednicamente significativa, incluidas enfermedades card\u00edacas, pulmonares, hep\u00e1ticas, renales, hematol\u00f3gicas, inmunol\u00f3gicas o conductuales, o infecci\u00f3n o neoplasias malignas, o cualquier otra enfermedad que, en la opini\u00f3n del investigador, podr\u00eda interferir con la participaci\u00f3n 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 ge\u00efncludeerd 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, \u03b2-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 exclusi\u00f3n para participantes sin tratamiento previo que se incluyan en la parte B:\n-\tAntecedentes de hipomagnesemia en las 12 semanas previas a la selecci\u00f3n.\n-\tInicio o cambio de la pauta posol\u00f3gica (excepto modificaciones para adaptarse a cambios de peso o cambios en el tratamiento de referencia) en las 12 semanas previas a la selecci\u00f3n 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 selecci\u00f3n, inicio o cambio en la posolog\u00eda de preparaciones de venta sin receta, como suplementos a base de hierbas o no, vitaminas, minerales y preparaciones homeop\u00e1ticas.\n-\tTener una fracci\u00f3n de eyecci\u00f3n del ventr\u00edculo izquierdo (FEVI) <40,0 % basada en un ecocardiograma realizado en las 12 semanas previas a la selecci\u00f3n o en la visita de selecci\u00f3n.\n-\tTratamiento con cualquier terapia de omisi\u00f3n del ex\u00f3n 51 en las 4 semanas anteriores a la selecci\u00f3n, o con cualquier terapia g\u00e9nica experimental para el tratamiento de la DMD en cualquier momento.\n-\t- Se aplican otros criterios de inclusi\u00f3n/exclusi\u00f3n.", "language": 7, "languageDescription": "Spanish"}, {"id": 3697012, "uuid": "a9731a70-3a2e-4920-97ba-d905d2e879ed", "attributeTranslation": "Exclusiecriteria voor behandelingsna\u00efeve deelnemers die worden ge\u00efncludeerd in deel B:\n- Voorgeschiedenis van hypomagnesi\u00ebmie 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, b\u00e8tablokkers 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), l\u00ednea 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\u2019s), 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 l\u00ednea de base en el nivel de prote\u00edna distrofina (seg\u00fan 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 omisi\u00f3n del ex\u00f3n 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 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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": "\u039c\u03c5\u03ca\u03ba\u03ae \u03b4\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03b1 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 viltolars\u00e9n administrado por v\u00eda intravenosa (IV) en dosis semanales de 80 mg/kg en ni\u00f1os 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 tollerabilit\u00e0 di viltolarsen somministrato per via endovenosa (EV) a dosi settimanali di 80\u00a0mg/kg in ragazzi che hanno completato lo studio NS\u2010065/NCNP\u201001-301", "language": 14, "languageDescription": "Italian"}, {"id": 6890914, "uuid": "284fa4a9-4a55-4a87-ad65-5bdc417fcfc2", "attributeTranslation": "\u0391\u03be\u03b9\u03bf\u03bb\u03cc\u03b3\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03b1\u03c3\u03c6\u03ac\u03bb\u03b5\u03b9\u03b1\u03c2 \u03ba\u03b1\u03b9 \u03c4\u03b7\u03c2 \u03b1\u03bd\u03b5\u03ba\u03c4\u03b9\u03ba\u03cc\u03c4\u03b7\u03c4\u03b1\u03c2 \u03c4\u03bf\u03c5 viltolarsen, \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03bf\u03c5 \u03b5\u03bd\u03b4\u03bf\u03c6\u03bb\u03b5\u03b2\u03af\u03c9\u03c2 (IV) \u03c3\u03b5 \u03b5\u03b2\u03b4\u03bf\u03bc\u03b1\u03b4\u03b9\u03b1\u03af\u03b5\u03c2 \u03b4\u03cc\u03c3\u03b5\u03b9\u03c2 \u03c4\u03c9\u03bd 80\u00a0mg/kg \u03c3\u03b5 \u03b1\u03b3\u03cc\u03c1\u03b9\u03b1 \u03c0\u03bf\u03c5 \u03ad\u03c7\u03bf\u03c5\u03bd \u03bf\u03bb\u03bf\u03ba\u03bb\u03b7\u03c1\u03ce\u03c3\u03b5\u03b9 \u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 NS\u00a0065/NCNP\u00a001-301", "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": "\u03a3\u03cd\u03b3\u03ba\u03c1\u03b9\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03b1\u03c0\u03bf\u03c4\u03b5\u03bb\u03b5\u03c3\u03bc\u03b1\u03c4\u03b9\u03ba\u03cc\u03c4\u03b7\u03c4\u03b1\u03c2 \u03c4\u03bf\u03c5 viltolarsen, \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd\u03bc\u03b5\u03bd\u03bf\u03c5 IV \u03c3\u03b5 \u03b5\u03b2\u03b4\u03bf\u03bc\u03b1\u03b4\u03b9\u03b1\u03af\u03b5\u03c2 \u03b4\u03cc\u03c3\u03b5\u03b9\u03c2 \u03c4\u03c9\u03bd 80 mg/kg, \u03c3\u03b5 \u03b1\u03b3\u03cc\u03c1\u03b9\u03b1 \u03c0\u03bf\u03c5 \u03ad\u03c7\u03bf\u03c5\u03bd \u03bf\u03bb\u03bf\u03ba\u03bb\u03b7\u03c1\u03ce\u03c3\u03b5\u03b9 \u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 NS 065/NCNP 01-301 \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b4\u03b9\u03ac\u03c1\u03ba\u03b5\u03b9\u03b1 \u03bc\u03af\u03b1\u03c2 \u03c0\u03b5\u03c1\u03b9\u03cc\u03b4\u03bf\u03c5 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1\u03c2 96 \u03b5\u03b2\u03b4\u03bf\u03bc\u03ac\u03b4\u03c9\u03bd \u03ad\u03bd\u03b1\u03bd\u03c4\u03b9 \u03bc\u03b1\u03c1\u03c4\u03cd\u03c1\u03c9\u03bd \u03bc\u03b5 \u03c6\u03c5\u03c3\u03b9\u03ba\u03ae \u03c0\u03bf\u03c1\u03b5\u03af\u03b1 \u03c4\u03b7\u03c2 \u03bd\u03cc\u03c3\u03bf\u03c5, \u03bc\u03b5 \u03c4\u03b7 \u03c7\u03c1\u03ae\u03c3\u03b7 \u03c4\u03c9\u03bd \u03b5\u03ba\u03b2\u03ac\u03c3\u03b5\u03c9\u03bd \u03b4\u03cd\u03bd\u03b1\u03bc\u03b7\u03c2 \u03ba\u03b1\u03b9 \u03b1\u03bd\u03c4\u03bf\u03c7\u03ae\u03c2", "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\u2019efficacia di viltolarsen somministrato per EV a dosi settimanali di 80 mg/kg in ragazzi che hanno completato lo studio NS\u2010065/NCNP\u201001-301 nell\u2019arco di un periodo di trattamento di 96 settimane rispetto ai controlli dell\u2019anamnesi 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 viltolars\u00e9n administrado IV en dosis semanales de 80 mg/kg a ni\u00f1os que hayan completado el \u2022 Prueba TTSTAND (Time to Stand Test, Tiempo en ponerse de pie) \u2022 Tiempo en recorrer corriendo o caminando 10 NS Pharma, Inc. \u2013 CONFIDENCIAL P\u00e1gina 2 de 6 estudio NS-065/NCNP-01-301 durante un per\u00edodo de tratamiento de 96 semanas con controles con evoluci\u00f3n 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.\u00a0\u00a0\u00a0\u00a0\u00a0 \u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03ad\u03c7\u03b5\u03b9 \u03bf\u03bb\u03bf\u03ba\u03bb\u03b7\u03c1\u03ce\u03c3\u03b5\u03b9 \u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 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.\tPati\u00ebnt 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 pati\u00ebnt heeft (hebben) schriftelijk ge\u00efnformeerde 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; pati\u00ebnten 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.\u00a0\u00a0\u00a0\u00a0\u00a0 \u039f(\u03bf\u03b9) \u03b3\u03bf\u03bd\u03ad\u03b1\u03c2(-\u03b5\u03af\u03c2) \u03ae \u03bf(\u03bf\u03b9) \u03bd\u03cc\u03bc\u03b9\u03bc\u03bf\u03c2(-\u03bf\u03b9) \u03ba\u03b7\u03b4\u03b5\u03bc\u03cc\u03bd\u03b1\u03c2(-\u03b5\u03c2) \u03ad\u03c7\u03bf\u03c5\u03bd \u03c0\u03b1\u03c1\u03ac\u03c3\u03c7\u03b5\u03b9 \u03b3\u03c1\u03b1\u03c0\u03c4\u03ae \u03c3\u03c5\u03bd\u03b1\u03af\u03bd\u03b5\u03c3\u03b7 \u03bc\u03b5\u03c4\u03ac \u03b1\u03c0\u03cc \u03b5\u03bd\u03b7\u03bc\u03ad\u03c1\u03c9\u03c3\u03b7 \u03ba\u03b1\u03b9 \u03b5\u03be\u03bf\u03c5\u03c3\u03b9\u03bf\u03b4\u03cc\u03c4\u03b7\u03c3\u03b7 \u03c3\u03cd\u03bc\u03c6\u03c9\u03bd\u03b1 \u03bc\u03b5 \u03c4\u03bf\u03bd \u039d\u03cc\u03bc\u03bf \u03c0\u03b5\u03c1\u03af \u03c6\u03bf\u03c1\u03b7\u03c4\u03cc\u03c4\u03b7\u03c4\u03b1\u03c2 \u03ba\u03b1\u03b9 \u03bb\u03bf\u03b3\u03bf\u03b4\u03bf\u03c3\u03af\u03b1\u03c2 \u03b3\u03b9\u03b1 \u03c4\u03b7\u03bd \u03b1\u03c3\u03c6\u03ac\u03bb\u03b9\u03c3\u03b7 \u03c5\u03b3\u03b5\u03af\u03b1\u03c2, \u03cc\u03c0\u03bf\u03c5 \u03b9\u03c3\u03c7\u03cd\u03b5\u03b9, \u03c0\u03c1\u03b9\u03bd \u03b1\u03c0\u03cc \u03bf\u03c0\u03bf\u03b9\u03b5\u03c3\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03c3\u03c7\u03b5\u03c4\u03b9\u03b6\u03cc\u03bc\u03b5\u03bd\u03b5\u03c2 \u03bc\u03b5 \u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 \u03b4\u03b9\u03b1\u03b4\u03b9\u03ba\u03b1\u03c3\u03af\u03b5\u03c2. \u0398\u03b1 \u03b6\u03b7\u03c4\u03b7\u03b8\u03b5\u03af \u03b1\u03c0\u03cc \u03c4\u03bf\u03c5\u03c2 \u03b1\u03c3\u03b8\u03b5\u03bd\u03b5\u03af\u03c2 \u03bd\u03b1 \u03c0\u03b1\u03c1\u03b1\u03c7\u03c9\u03c1\u03ae\u03c3\u03bf\u03c5\u03bd \u03b3\u03c1\u03b1\u03c0\u03c4\u03ae \u03ae \u03c0\u03c1\u03bf\u03c6\u03bf\u03c1\u03b9\u03ba\u03ae \u03c3\u03cd\u03bc\u03c6\u03c9\u03bd\u03b7 \u03b3\u03bd\u03ce\u03bc\u03b7 \u03c3\u03cd\u03bc\u03c6\u03c9\u03bd\u03b1 \u03bc\u03b5 \u03c4\u03b9\u03c2 \u03c4\u03bf\u03c0\u03b9\u03ba\u03ad\u03c2 \u03b1\u03c0\u03b1\u03b9\u03c4\u03ae\u03c3\u03b5\u03b9\u03c2", "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\u2019autorizzazione secondo la legge sulla portabilit\u00e0 e responsabilit\u00e0 delle polizze di assicurazione sanitaria (Health Insurance Portability and Accountability Act, HIPAA), ove applicabile, prima di iniziare qualsiasi procedura correlata allo studio; ai pazienti sar\u00e0 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 autorizaci\u00f3n HIPAA (Health Insurance Portability and Accountability Act, Ley de Responsabilidad y Portabilidad del Seguro de Salud), cuando sea aplicable; se pedir\u00e1 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 est\u00e1n dispuestos a cumplir las visitas programadas, el plan de administraci\u00f3n del producto en investigaci\u00f3n (PEI) y los procedimientos del estudio, y pueden hacerlo", "language": 7, "languageDescription": "Spanish"}, {"id": 6890909, "uuid": "a904163e-c982-4fc3-b8f1-a2c4fd096b38", "attributeTranslation": "3.\u00a0\u00a0\u00a0\u00a0\u00a0 \u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03ba\u03b1\u03b9 \u03bf(\u03bf\u03b9) \u03b3\u03bf\u03bd\u03ad\u03b1\u03c2(-\u03b5\u03af\u03c2) \u03ae \u03bf(\u03bf\u03b9) \u03bd\u03cc\u03bc\u03b9\u03bc\u03bf\u03c2(-\u03bf\u03b9) \u03ba\u03b7\u03b4\u03b5\u03bc\u03cc\u03bd\u03b1\u03c2(-\u03b5\u03c2) \u03b5\u03af\u03bd\u03b1\u03b9 \u03c0\u03c1\u03cc\u03b8\u03c5\u03bc\u03bf\u03b9 \u03ba\u03b1\u03b9 \u03b5\u03af\u03bd\u03b1\u03b9 \u03c3\u03b5 \u03b8\u03ad\u03c3\u03b7 \u03bd\u03b1 \u03c3\u03c5\u03bc\u03bc\u03bf\u03c1\u03c6\u03c9\u03b8\u03bf\u03cd\u03bd \u03bc\u03b5 \u03c4\u03b9\u03c2 \u03c0\u03c1\u03bf\u03b3\u03c1\u03b1\u03bc\u03bc\u03b1\u03c4\u03b9\u03c3\u03bc\u03ad\u03bd\u03b5\u03c2 \u03b5\u03c0\u03b9\u03c3\u03ba\u03ad\u03c8\u03b5\u03b9\u03c2, \u03c4\u03bf \u03c0\u03c1\u03cc\u03b3\u03c1\u03b1\u03bc\u03bc\u03b1 \u03c7\u03bf\u03c1\u03ae\u03b3\u03b7\u03c3\u03b7\u03c2 \u03c4\u03bf\u03c5 \u03c5\u03c0\u03cc \u03ad\u03c1\u03b5\u03c5\u03bd\u03b1 \u03c0\u03c1\u03bf\u03ca\u03cc\u03bd\u03c4\u03bf\u03c2 (\u03a5\u0395\u03a0) \u03ba\u03b1\u03b9 \u03c4\u03b9\u03c2 \u03b4\u03b9\u03b1\u03b4\u03b9\u03ba\u03b1\u03c3\u03af\u03b5\u03c2 \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2.", "language": 5, "languageDescription": "Greek"}, {"id": 6890907, "uuid": "a904163e-c982-4fc3-b8f1-a2c4fd096b38", "attributeTranslation": "3.\tPati\u00ebnt 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, oltrech\u00e9 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\u2010065/NCNP\u201001\u2010301 che, a giudizio dello sperimentatore e/o dello Sponsor, preclude l\u2019utilizzo sicuro di viltolarsen per il paziente in questo studio.", "language": 14, "languageDescription": "Italian"}, {"id": 6890881, "uuid": "7103bbae-cc28-4434-8c42-adefff28407a", "attributeTranslation": "1.\u00a0\u00a0\u00a0\u00a0\u00a0 \u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03b5\u03bc\u03c6\u03ac\u03bd\u03b9\u03c3\u03b5 \u03ad\u03bd\u03b1 \u03b1\u03bd\u03b5\u03c0\u03b9\u03b8\u03cd\u03bc\u03b7\u03c4\u03bf \u03c3\u03c5\u03bc\u03b2\u03ac\u03bd \u03c3\u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 NS-065/NCNP-01-301, \u03c4\u03bf \u03bf\u03c0\u03bf\u03af\u03bf, \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b3\u03bd\u03ce\u03bc\u03b7 \u03c4\u03bf\u03c5 \u03b5\u03c1\u03b5\u03c5\u03bd\u03b7\u03c4\u03ae \u03ae/\u03ba\u03b1\u03b9 \u03c4\u03bf\u03c5 \u03c7\u03bf\u03c1\u03b7\u03b3\u03bf\u03cd, \u03ba\u03b1\u03b8\u03b9\u03c3\u03c4\u03ac \u03b1\u03b4\u03cd\u03bd\u03b1\u03c4\u03b7 \u03c4\u03b7\u03bd \u03b1\u03c3\u03c6\u03b1\u03bb\u03ae \u03c7\u03c1\u03ae\u03c3\u03b7 \u03c4\u03bf\u03c5 viltolarsen \u03b3\u03b9\u03b1 \u03c4\u03bf\u03bd \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae \u03c3\u03b5 \u03b1\u03c5\u03c4\u03ae \u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7.", "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 opini\u00f3n del investigador y/o del promotor, impide el uso seguro de viltolars\u00e9n en el paciente en este estudio", "language": 7, "languageDescription": "Spanish"}, {"id": 6890882, "uuid": "7103bbae-cc28-4434-8c42-adefff28407a", "attributeTranslation": "1.\tPati\u00ebnt 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 pati\u00ebnt 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 \u00e8 stato sottoposto a un trattamento che \u00e8 stato effettuato per finalit\u00e0 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.\u00a0\u00a0\u00a0\u00a0\u00a0 \u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03ad\u03bb\u03b1\u03b2\u03b5 \u03bc\u03af\u03b1 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1 \u03bc\u03b5 \u03c3\u03ba\u03bf\u03c0\u03cc \u03c4\u03b7\u03bd \u03b5\u03c0\u03b1\u03b3\u03c9\u03b3\u03ae \u03c4\u03b7\u03c2 \u03ad\u03ba\u03c6\u03c1\u03b1\u03c3\u03b7\u03c2 \u03b4\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03bd\u03b7\u03c2 \u03ae \u03c3\u03c7\u03b5\u03c4\u03b9\u03b6\u03cc\u03bc\u03b5\u03bd\u03b7\u03c2 \u03bc\u03b5 \u03c4\u03b7 \u03b4\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03bd\u03b7 \u03c0\u03c1\u03c9\u03c4\u03b5\u0390\u03bd\u03b7\u03c2 \u03bc\u03b5\u03c4\u03ac \u03c4\u03b7\u03bd \u03bf\u03bb\u03bf\u03ba\u03bb\u03ae\u03c1\u03c9\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2 NS-065/NCNP-01-301.", "language": 5, "languageDescription": "Greek"}, {"id": 6890886, "uuid": "31391515-3901-4641-8418-5be78f6765c8", "attributeTranslation": "2.\tPati\u00ebnt 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 \ninducci\u00f3n de la distrofina o una prote\u00edna relacionada con la \ndistrofina despu\u00e9s 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 recibi\u00f3 otro u otros medicamentos en investigaci\u00f3n durante el estudio NS-065/NCNP-01-301 o despu\u00e9s de completarlo", "language": 7, "languageDescription": "Spanish"}, {"id": 6890887, "uuid": "e781194e-3da3-4abc-bd68-96a3e24ac536", "attributeTranslation": "2.\tPati\u00ebnt 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.\u00a0\u00a0\u00a0\u00a0\u00a0 \u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03ad\u03bb\u03b1\u03b2\u03b5 \u03bf\u03c0\u03bf\u03b9\u03bf\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03ac\u03bb\u03bb\u03bf \u03c5\u03c0\u03cc \u03ad\u03c1\u03b5\u03c5\u03bd\u03b1 \u03c6\u03ac\u03c1\u03bc\u03b1\u03ba\u03bf \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b4\u03b9\u03ac\u03c1\u03ba\u03b5\u03b9\u03b1 \u03ae \u03bc\u03b5\u03c4\u03ac \u03c4\u03b7\u03bd \u03bf\u03bb\u03bf\u03ba\u03bb\u03ae\u03c1\u03c9\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7\u03c2 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.\u00a0\u00a0\u00a0\u00a0\u00a0 \u039f \u03b1\u03c3\u03b8\u03b5\u03bd\u03ae\u03c2 \u03ba\u03c1\u03af\u03bd\u03b5\u03c4\u03b1\u03b9 \u03b1\u03c0\u03cc \u03c4\u03bf\u03bd \u03b5\u03c1\u03b5\u03c5\u03bd\u03b7\u03c4\u03ae \u03ae/\u03ba\u03b1\u03b9 \u03c4\u03bf\u03bd \u03c7\u03bf\u03c1\u03b7\u03b3\u03cc \u03bc\u03b7 \u03ba\u03b1\u03c4\u03ac\u03bb\u03bb\u03b7\u03bb\u03bf\u03c2 \u03b3\u03b9\u03b1 \u03c3\u03c5\u03bc\u03bc\u03b5\u03c4\u03bf\u03c7\u03ae \u03c3\u03c4\u03b7 \u03bc\u03b5\u03bb\u03ad\u03c4\u03b7 \u03b5\u03c0\u03ad\u03ba\u03c4\u03b1\u03c3\u03b7\u03c2 \u03b3\u03b9\u03b1 \u03bf\u03c0\u03bf\u03b9\u03bf\u03bd\u03b4\u03ae\u03c0\u03bf\u03c4\u03b5 \u03bb\u03cc\u03b3\u03bf.", "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 extensi\u00f3n por alg\u00fan \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.\tPati\u00ebnt 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": "\u2022 \u0396\u03c9\u03c4\u03b9\u03ba\u03ac \u03c3\u03b7\u03bc\u03b5\u03af\u03b1 \n\u2022 \u039a\u03bb\u03b9\u03bd\u03b9\u03ba\u03ae \u03b5\u03be\u03ad\u03c4\u03b1\u03c3\u03b7\n\u2022 \u039a\u03bb\u03b9\u03bd\u03b9\u03ba\u03ad\u03c2 \u03b5\u03c1\u03b3\u03b1\u03c3\u03c4\u03b7\u03c1\u03b9\u03b1\u03ba\u03ad\u03c2 \u03b5\u03be\u03b5\u03c4\u03ac\u03c3\u03b5\u03b9\u03c2\n\u2022 \u0391\u03b9\u03bc\u03b1\u03c4\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ad\u03c2 \u03ba\u03b1\u03b9 \u03ba\u03bb\u03b9\u03bd\u03b9\u03ba\u03ad\u03c2 \u03b2\u03b9\u03bf\u03c7\u03b7\u03bc\u03b9\u03ba\u03ad\u03c2 \u03b5\u03be\u03b5\u03c4\u03ac\u03c3\u03b5\u03b9\u03c2\n\u2022 \u0391\u03bd\u03ac\u03bb\u03c5\u03c3\u03b7 \u03bf\u03cd\u03c1\u03c9\u03bd\n\u2022 \u039a\u03c5\u03c4\u03c4\u03b1\u03c1\u03bf\u03bb\u03bf\u03b3\u03b9\u03ba\u03ae \u03b5\u03be\u03ad\u03c4\u03b1\u03c3\u03b7 \u03bf\u03cd\u03c1\u03c9\u03bd\n\u2022 \u0391\u03bd\u03c4\u03b9\u03c3\u03ce\u03bc\u03b1\u03c4\u03b1 \u03c3\u03c4\u03b7 \u03b4\u03c5\u03c3\u03c4\u03c1\u03bf\u03c6\u03af\u03bd\u03b7 \u03ba\u03b1\u03b9 \u03c4\u03bf viltolarsen\n\u2022 \u0397\u03bb\u03b5\u03ba\u03c4\u03c1\u03bf\u03ba\u03b1\u03c1\u03b4\u03b9\u03bf\u03b3\u03c1\u03ac\u03c6\u03b7\u03bc\u03b1 12 \u03b1\u03c0\u03b1\u03b3\u03c9\u03b3\u03ce\u03bd (\u0397\u039a\u0393)\n\u2022 \u03a5\u03c0\u03b5\u03c1\u03b7\u03c7\u03bf\u03b3\u03c1\u03ac\u03c6\u03b7\u03bc\u03b1 \u03bd\u03b5\u03c6\u03c1\u03ce\u03bd \n\u2022 \u0395\u03bc\u03c6\u03b1\u03bd\u03b9\u03b6\u03cc\u03bc\u03b5\u03bd\u03b1 \u03ba\u03b1\u03c4\u03ac \u03c4\u03b7 \u03b8\u03b5\u03c1\u03b1\u03c0\u03b5\u03af\u03b1 \u03b1\u03bd\u03b5\u03c0\u03b9\u03b8\u03cd\u03bc\u03b7\u03c4\u03b1 \u03c3\u03c5\u03bc\u03b2\u03ac\u03bd\u03c4\u03b1 (TEAE) \u03ba\u03b1\u03b9 \u03c3\u03bf\u03b2\u03b1\u03c1\u03ac \u03b1\u03bd\u03b5\u03c0\u03b9\u03b8\u03cd\u03bc\u03b7\u03c4\u03b1 \u03c3\u03c5\u03bc\u03b2\u03ac\u03bd\u03c4\u03b1 (\u03a3\u0391\u03a3)", "language": 5, "languageDescription": "Greek"}, {"id": 6890873, "uuid": "0804d475-85ce-4199-828d-2aea39a8dd8b", "attributeTranslation": "\u2022 Vitale functies\n\u2022 Lichamelijk onderzoek\n\u2022 Klinische laboratoriumtests\no\tHematologie en klinische chemie\no\tUrineanalyse\no\tUrine cytologie\n\u2022 Antistoffen tegen dystrofine en viltolarsen\n\u2022 12-afleidingen-elektrocardiogram (ECG)\n\u2022 Echo van de nieren\n\u2022 Behandelingsgerelateerde bijwerkingen (TEAE\u00b4s) en ernstige bijwerkingen (SAE\u00b4s)", "language": 18, "languageDescription": "Dutch"}, {"id": 6890874, "uuid": "0804d475-85ce-4199-828d-2aea39a8dd8b", "attributeTranslation": "\u2022 Constantes vitales \n\u2022 Exploraci\u00f3n f\u00edsica \n\u2022 An\u00e1lisis cl\u00ednicos o Hematolog\u00eda y bioqu\u00edmica cl\u00ednica o An\u00e1lisis de orina o Citolog\u00eda urinaria \n\u2022 Anticuerpos contra la distrofina y viltolars\u00e9n \n\u2022 ECG de 12 derivaciones \n\u2022 Ecograf\u00eda renal \n\u2022 AADT y AAG", "language": 7, "languageDescription": "Spanish"}, {"id": 6890871, "uuid": "0804d475-85ce-4199-828d-2aea39a8dd8b", "attributeTranslation": "\u2022 Segni vitali \n\u2022 Esame obiettivo\n\u2022 Esami clinici di laboratorio\no\tEmatologia e chimica clinica\no\tEsame delle urine\no\tCitologia urinaria\n\u2022 Anticorpi anti-distrofina e anti-viltolarsen\n\u2022 Elettrocardiogramma (ECG) a 12 derivazioni\n\u2022 Ecografia renale \n\u2022 Eventi avversi emergenti dal trattamento (TEAE) ed eventi avversi seri (SAE)", "language": 14, "languageDescription": "Italian"}]}], "secondaryEndPoints": [{"id": 685130, "number": 1, "endPoint": "Secondary: - Time to Stand Test (TTSTAND) - Time to Run/Walk 10 Meters Test (TTRW) - Six-minute Walk Test (6MWT) - North Star Ambulatory Assessment (NSAA) - Time to Climb 4 Stairs Test (TTCLIMB) - Quantitative muscle strength measured by hand-held dynamometer  (elbow extension, elbow flexion, knee extension, and knee flexion on the dominant side only)", "isPrimary": false, "endPointTranslations": [{"id": 6890875, "uuid": "14e05552-9793-4598-8173-e3dd8c6bd9ce", "attributeTranslation": "\u2022 \u0394\u03bf\u03ba\u03b9\u03bc\u03b1\u03c3\u03af\u03b1 \u03b5\u03ba\u03c4\u03af\u03bc\u03b7\u03c3\u03b7\u03c2 \u03c4\u03bf\u03c5 \u03c7\u03c1\u03cc\u03bd\u03bf\u03c5 \u03bc\u03b5\u03c4\u03ac\u03b2\u03b1\u03c3\u03b7\u03c2 \u03c3\u03b5 \u03cc\u03c1\u03b8\u03b9\u03b1 \u03b8\u03ad\u03c3\u03b7 (TTSTAND)\n\u2022 \u0394\u03bf\u03ba\u03b9\u03bc\u03b1\u03c3\u03af\u03b1 \u03b5\u03ba\u03c4\u03af\u03bc\u03b7\u03c3\u03b7\u03c2 \u03c4\u03bf\u03c5 \u03c7\u03c1\u03cc\u03bd\u03bf\u03c5 \u03c4\u03c1\u03b5\u03be\u03af\u03bc\u03b1\u03c4\u03bf\u03c2/\u03b2\u03ac\u03b4\u03b9\u03c3\u03b7\u03c2 \u03b1\u03c0\u03cc\u03c3\u03c4\u03b1\u03c3\u03b7\u03c2 10 \u03bc\u03ad\u03c4\u03c1\u03c9\u03bd (TTRW)\n\u2022 \u0394\u03bf\u03ba\u03b9\u03bc\u03b1\u03c3\u03af\u03b1 \u03b2\u03ac\u03b4\u03b9\u03c3\u03b7\u03c2 6 \u03bb\u03b5\u03c0\u03c4\u03ce\u03bd (6MWT)\n\u2022 \u039a\u03bb\u03af\u03bc\u03b1\u03ba\u03b1 North Star Ambulatory Assessment (NSAA)\n\u2022 \u0394\u03bf\u03ba\u03b9\u03bc\u03b1\u03c3\u03af\u03b1 \u03b5\u03ba\u03c4\u03af\u03bc\u03b7\u03c3\u03b7\u03c2 \u03c4\u03bf\u03c5 \u03c7\u03c1\u03cc\u03bd\u03bf\u03c5 \u03b1\u03bd\u03ac\u03b2\u03b1\u03c3\u03b7\u03c2 4 \u03c3\u03ba\u03b1\u03bb\u03bf\u03c0\u03b1\u03c4\u03b9\u03ce\u03bd (TTCLIMB)\n\u2022 \u03a0\u03bf\u03c3\u03bf\u03c4\u03b9\u03ba\u03ae \u03bc\u03ad\u03c4\u03c1\u03b7\u03c3\u03b7 \u03c4\u03b7\u03c2 \u03bc\u03c5\u03ca\u03ba\u03ae\u03c2 \u03b4\u03cd\u03bd\u03b1\u03bc\u03b7\u03c2 \u03bc\u03b5 \u03c4\u03b7 \u03c7\u03c1\u03ae\u03c3\u03b7 \u03c6\u03bf\u03c1\u03b7\u03c4\u03bf\u03cd \u03b4\u03c5\u03bd\u03b1\u03bc\u03cc\u03bc\u03b5\u03c4\u03c1\u03bf\u03c5 (\u03ad\u03ba\u03c4\u03b1\u03c3\u03b7 \u03b1\u03b3\u03ba\u03ce\u03bd\u03b1, 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confirmatory  (Phase III)", "endPoint": "\u2022Change from baseline of Peak Expiratory Flow percent predicted (PEF%p) at 18 months of treatment of givinostat compared to placebo group \u2022 Change from baseline of Forced Vital Capacity percent predicted (FVC%p) at 18 months of treatment of givinostat compared to placebo group \u2022 Cumulative loss of PUL items over 18 months of treatment of givinostat compared to placebo group, \u2022Type incidence and severity of TEAEs \u2022Proportion of patients experiencing TEAEs  (baseline to EOS/F-Up) \u2022 Changes in vital signs, lab tests, ECG, ECHO \u2022Time to assisted ventilation, rate and severity of respiratory infection and duration and use of antibiotics of givinostat to placebo group \u2022Time to onset of diarrhoea \u2022Height and weight Z-scores", "product": "ITF2357, PLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspension", "ageRangeSecondary": [""], "ageGroup": "0-17 years", "gender": "Male", "trialRegion": 3, "totalNumberEnrolled": "120", "primaryEndPoint": "Change of PUL total score at 18 months of treatment of givinostat compared to placebo group", "resultsFirstReceived": "No", "lastUpdated": "27/03/2026", "lastPublicationUpdate": "28/03/2026"},"rawRetrieveRecord":{"ctNumber": "2023-503521-19-00", "ctStatus": "Authorised", "startDateEU": "2024-02-09", "decisionDate": "2023-12-05T09:10:24.799", "publishDate": "2026-03-28T02:35:00.843225085", "ctPublicStatusCode": 4, "authorizedApplication": {"authorizedPartI": {"id": 116608, "rowSubjectCount": 48, "rowCountriesInfo": [{"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": 484059, "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": "Orfan EU/3/12/1009", "doseUom": "mg milligram(s)", "maxDailyDoseAmount": "93.4", "doseUomTotal": "mg milligram(s)", "maxTotalDoseAmount": "47073", "maxTreatmentPeriod": 18, "timeUnitCode": "3", "otherMedicinalProduct": "histone deacetylase (HDAC) inhibitor", "evCode": "PRD4797678", "sponsorProductCodeEdit": "ITF2357", "devices": [], "characteristics": ["11"], "routes": ["ORAL USE"], "allSubstancesChemicals": true, "productName": "ITF2357", "jsonActiveSubstanceNames": "givinostat", "pharmaceuticalFormDisplay": "ORAL SUSPENSION"}, {"id": 484058, "part1MpRoleTypeCode": "1", "productDictionaryInfo": {"productPharmForm": "N/A", "euMpNumber": "N/A", "marketingAuthNumber": "N/A", "prodName": "PLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspension", "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", "devices": [], "characteristics": [], "routes": [], "allSubstancesChemicals": false, "productName": "PLACEBO for GIVINOSTAT hydrochloride monohydrate (ITF2357) 10 mg/mL oral suspension", "jsonActiveSubstanceNames": "n/a", "pharmaceuticalFormDisplay": "N/A"}], "trialDetails": {"clinicalTrialIdentifiers": {"fullTitle": "Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular Dystrophy", "fullTitleTranslations": [{"id": 9552432, "uuid": "618122d7-f300-47cd-950c-97de609732c4", "attributeTranslation": "\u00c9tude multicentrique, randomis\u00e9e, en double aveugle, contr\u00f4l\u00e9e par placebo, visant \u00e0 \u00e9valuer l\u2019efficacit\u00e9, la s\u00e9curit\u00e9 d\u2019emploi et la tol\u00e9rance du givinostat chez des patients non ambulatoires atteints de dystrophie musculaire de Duchenne", "language": 10, "languageDescription": "French"}, {"id": 9552431, "uuid": "618122d7-f300-47cd-950c-97de609732c4", "attributeTranslation": "Gerandomiseerd, dubbelblind, placebogecontroleerd onderzoek in meerdere centra ter beoordeling van de werkzaamheid, veiligheid en verdraagbaarheid van givinostat bij niet-ambulante pati\u00ebnten met de ziekte van Duchenne", "language": 37, "languageDescription": "Dutch (Netherlands)"}], "publicTitle": "Randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular Dystrophy", "publicTitleTranslations": [{"id": 9552451, "uuid": "41875623-87c3-481c-af8c-de8ec01aa580", "attributeTranslation": "\u00c9tude multicentrique, randomis\u00e9e, en double aveugle, contr\u00f4l\u00e9e par placebo, visant \u00e0 \u00e9valuer l\u2019efficacit\u00e9, la s\u00e9curit\u00e9 d\u2019emploi et la tol\u00e9rance du givinostat chez des patients non ambulatoires atteints de dystrophie musculaire de Duchenne", "language": 34, "languageDescription": "French (France)"}, {"id": 9552452, "uuid": "41875623-87c3-481c-af8c-de8ec01aa580", "attributeTranslation": "Gerandomiseerd, dubbelblind, placebogecontroleerd onderzoek in meerdere centra ter beoordeling van de werkzaamheid, veiligheid en verdraagbaarheid van givinostat bij niet-ambulante pati\u00ebnten met de ziekte van Duchenne", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 10109277, "uuid": "41875623-87c3-481c-af8c-de8ec01aa580", "attributeTranslation": "Randomiserad, dubbelblind, placebokontrollerad multicenterstudie f\u00f6r att utv\u00e4rdera effekt, s\u00e4kerhet och tolerabilitet av givinostat hos patienter med Duchennes muskeldystrofi utan g\u00e5ngf\u00f6rm\u00e5ga.", "language": 24, "languageDescription": "Swedish"}], "shortTitle": "DSC/14/2357/50", "secondaryIdentifyingNumbers": {"whoUniversalTrialNumber": {"id": 483716, "number": "U1111-1295-1799"}, "nctNumber": {"id": 483715, "number": "NCT05933057"}, "additionalRegistries": [{"id": 483717, "number": "IND126598", "otherRegistryName": "FDA", "ctRegistryCode": "6"}]}}, "trialInformation": {"trialCategory": {"isLowIntervention": false, "trialPhase": "5", "trialCategory": "2", "justificationForTrialCategory": "This is phase III to evaluate safety efficacy and tolerability", "trialCategoryId": 106773}, "medicalCondition": {"partIMedicalConditions": [{"id": 121190, "medicalCondition": "Duchenne muscular dystrophy (DMD)", "medicalConditionTranslations": [{"id": 9552376, "uuid": "9c5d270a-0c86-4488-8889-90f188ad3e87", "attributeTranslation": "Ziekte van Duchenne", "language": 37, "languageDescription": "Dutch (Netherlands)"}], "isConditionRareDisease": true}]}, "trialObjective": {"trialScopes": [{"code": "13", "otherDescription": "tolerability", "trialScopeId": 352957}, {"code": "7", "trialScopeId": 352954}, {"code": "4", "trialScopeId": 352956}, {"code": "3", "trialScopeId": 352953}, {"code": "6", "trialScopeId": 352952}, {"code": "5", "trialScopeId": 352955}], "mainObjective": "To demonstrate the efficacy of givinostat in reducing muscle decline in non-ambulant DMD patients.", "mainObjectiveTranslations": [{"id": 9552449, "uuid": "46e41498-affd-4e79-817c-bb44f0b6c1c0", "attributeTranslation": "Het aantonen van de werkzaamheid van givinostat wat betreft het verminderen van spierafname bij niet-ambulante DMD-pati\u00ebnten.", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552450, "uuid": "46e41498-affd-4e79-817c-bb44f0b6c1c0", "attributeTranslation": "D\u00e9montrer l\u2019efficacit\u00e9 du givinostat dans la r\u00e9duction du d\u00e9clin musculaire chez les patients atteints de DMD non ambulatoires", "language": 10, "languageDescription": "French"}], "secondaryObjectives": [{"id": 409527, "number": 1, "secondaryObjective": "To evaluate the safety and tolerability of givinostat in non-ambulant DMD patients", "secondaryObjectiveTranslations": [{"id": 9552454, "uuid": "d13df40d-2e46-44dd-a93c-fdd28c91fea8", "attributeTranslation": "\u00c9valuer la s\u00e9curit\u00e9 d\u2019emploi et la tol\u00e9rance du givinostat chez les patients atteints de DMD non ambulatoires.", "language": 10, "languageDescription": "French"}, {"id": 9552453, "uuid": "d13df40d-2e46-44dd-a93c-fdd28c91fea8", "attributeTranslation": "Het beoordelen van de veiligheid en verdraagbaarheid van givinostat bij niet-ambulante DMD-pati\u00ebnten.", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 409528, "number": 2, "secondaryObjective": "To further investigate the efficacy of givinostat in non-ambulant DMD patients.", "secondaryObjectiveTranslations": [{"id": 9552455, "uuid": "9560c8f7-6227-4f3c-a4a5-6d132f1367ad", "attributeTranslation": "Etudier plus en d\u00e9tails l\u2019efficacit\u00e9 du givinostat chez les patients atteints de DMD non ambulatoires", "language": 10, "languageDescription": "French"}, {"id": 9552456, "uuid": "9560c8f7-6227-4f3c-a4a5-6d132f1367ad", "attributeTranslation": "Het verder onderzoeken van de werkzaamheid van givinostat bij niet-ambulante DMD-pati\u00ebnten.", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}]}, "eligibilityCriteria": {"principalInclusionCriteria": [{"id": 783423, "number": 1, "principalInclusionCriteria": "Children and adolescent males aged \u2265 9 to <18 years at screening (patients \u2265 18 years of age at screening will not be enrolled into the study)", "principalInclusionCriteriaTranslations": [{"id": 9552434, "uuid": "0eff514c-5441-425a-a8b1-29d58033376f", "attributeTranslation": "Enfants et adolescents de sexe masculin \u00e2g\u00e9s de \u2265 9 \u00e0 < 18 ans \u00e0 la s\u00e9lection (les patients \u00e2g\u00e9s de \u2265 18 ans \u00e0 la s\u00e9lection ne seront pas inclus dans l\u2019\u00e9tude)", "language": 10, "languageDescription": "French"}, {"id": 9552433, "uuid": "0eff514c-5441-425a-a8b1-29d58033376f", "attributeTranslation": "Kinderen en jongeren van \u2265 9 tot < 18 jaar bij de screening (pati\u00ebnten \u2265 18 jaar bij de screening worden niet in het onderzoek ingeschreven", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 783424, "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": 9552436, "uuid": "1e8b296e-b9ac-4bb0-9a83-e2e2af72103e", "attributeTranslation": "In staat zijn om schriftelijk ge\u00efnformeerde 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": 9552435, "uuid": "1e8b296e-b9ac-4bb0-9a83-e2e2af72103e", "attributeTranslation": "\u00catre en mesure de donner un assentiment \u00e9clair\u00e9 et/ou un consentement \u00e9clair\u00e9 \u00e9crit sign\u00e9 par le patient et/ou parent/tuteur l\u00e9gal (conform\u00e9ment \u00e0 la r\u00e9glementation locale)", "language": 10, "languageDescription": "French"}]}, {"id": 783425, "number": 3, "principalInclusionCriteria": "A genetic diagnosis of DMD", "principalInclusionCriteriaTranslations": [{"id": 9552437, "uuid": "7f23398d-da5b-4adb-9e5f-0ba70a456859", "attributeTranslation": "Een genetische diagnose van DMD hebben", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552438, "uuid": "7f23398d-da5b-4adb-9e5f-0ba70a456859", "attributeTranslation": "Diagnostic g\u00e9n\u00e9tique de DMD", "language": 10, "languageDescription": "French"}]}, {"id": 783426, "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": 9552440, "uuid": "6d940ff2-c802-4981-9c83-ba3aa3757c4e", "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": 9552439, "uuid": "6d940ff2-c802-4981-9c83-ba3aa3757c4e", "attributeTranslation": "\u00catre non ambulatoires, c\u2019est-\u00e0-dire \u00eatre en fauteuil roulant et :\na. Ne pas \u00eatre capables d\u2019effectuer le test de marche/course de 10 m\u00e8tres (10MWT), ou\nb. Ne pas \u00eatre capables d\u2019effectuer le 10MWT en 30 secondes ou moins, sans aucune aide ou dispositif", "language": 10, "languageDescription": "French"}]}, {"id": 783427, "number": 5, "principalInclusionCriteria": "Performance of the Upper Limb test (PUL version 2.0) entry item scores 3 to 6", "principalInclusionCriteriaTranslations": [{"id": 9552441, "uuid": "981d00e1-0716-41e0-9219-60d946266c2b", "attributeTranslation": "Scores d\u2019entr\u00e9e de 3 \u00e0 6 au test de performance des membres sup\u00e9rieurs (version PUL 2.0)", "language": 10, "languageDescription": "French"}, {"id": 9552442, "uuid": "981d00e1-0716-41e0-9219-60d946266c2b", "attributeTranslation": "Prestaties van de test van de bovenste ledematen (PUL versie 2.0) instapitemscores 3 tot 6", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 783428, "number": 6, "principalInclusionCriteria": "If on medication for DMD-associated cardiomyopathy, stable for \u22651 month immediately prior to start of study treatment", "principalInclusionCriteriaTranslations": [{"id": 9552443, "uuid": "f71f7d3a-4940-40d9-a763-33cb299d66a2", "attributeTranslation": "Si les patients sont sous traitement pour une cardiomyopathie associ\u00e9e \u00e0 la DMD (p. ex. inhibiteur de l\u2019ECA, \u03b2-bloquant, diur\u00e9tique), ils doivent \u00eatre stables depuis \u2265 1 mois imm\u00e9diatement avant le d\u00e9but du traitement \u00e0 l\u2019\u00e9tude, le cas \u00e9ch\u00e9ant", "language": 10, "languageDescription": "French"}, {"id": 9552444, "uuid": "f71f7d3a-4940-40d9-a763-33cb299d66a2", "attributeTranslation": "Bij gebruik van medicatie voor DMD-geassocieerde cardiomyopathie, stabiel gedurende \u2265 1 maand onmiddellijk voorafgaand aan de start van de onderzoeksbehandeling", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 783429, "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": 9552446, "uuid": "81d64ea8-fca3-414a-ac20-9784588f803e", "attributeTranslation": "Stabiele corticostero\u00efden, gedefinieerd als:\na. Het krijgen van systemische corticostero\u00efden 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": 9552445, "uuid": "81d64ea8-fca3-414a-ac20-9784588f803e", "attributeTranslation": "Cortico\u00efdes stables, d\u00e9finis comme suit :\na. Administration de cortico\u00efdes syst\u00e9miques pendant au moins 6 mois imm\u00e9diatement avant le d\u00e9but du traitement \u00e0 l\u2019\u00e9tude \nb. Aucune modification significative de la dose ou du sch\u00e9ma posologique (\u00e0 l\u2019exception des ajustements dus \u00e0 une modification du poids corporel) pendant un minimum de 6 mois imm\u00e9diatement avant le d\u00e9but du traitement \u00e0 l\u2019\u00e9tude", "language": 10, "languageDescription": "French"}]}, {"id": 783430, "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": 9552448, "uuid": "60dbcdb9-22f4-40f9-b078-8f35c6578eeb", "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": 9552447, "uuid": "60dbcdb9-22f4-40f9-b078-8f35c6578eeb", "attributeTranslation": "\u00catre dispos\u00e9s \u00e0 utiliser une m\u00e9thode de contraception ad\u00e9quate. Des m\u00e9thodes contraceptives efficaces doivent \u00eatre utilis\u00e9es depuis la visite de randomisation jusqu\u2019\u00e0 3 mois apr\u00e8s la derni\u00e8re dose du m\u00e9dicament \u00e0 l\u2019\u00e9tude", "language": 9, "languageDescription": "Finnish"}]}], "principalExclusionCriteria": [{"id": 1342674, "number": 1, "principalExclusionCriteria": "Exposure to another investigational drug within 3 months prior to start of study treatment", "principalExclusionCriteriaTranslations": [{"id": 9552383, "uuid": "aa0ac8b3-0f76-4c21-bcc3-8c383f0a47e2", "attributeTranslation": "Enfants et adolescents de sexe masculin \u00e2g\u00e9s de \u2265 9 \u00e0 < 18 ans \u00e0 la s\u00e9lection (les patients \u00e2g\u00e9s de \u2265 18 ans \u00e0 la s\u00e9lection ne seront pas inclus dans l\u2019\u00e9tude)", "language": 10, "languageDescription": "French"}, {"id": 9552384, "uuid": "aa0ac8b3-0f76-4c21-bcc3-8c383f0a47e2", "attributeTranslation": "Blootstelling aan een ander onderzoeksmiddel binnen 3 maanden voorafgaand aan de start van de onderzoeksbehandeling", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 1342675, "number": 10, "principalExclusionCriteria": "Episode of respiratory failure within the 8 weeks prior to screening", "principalExclusionCriteriaTranslations": [{"id": 9552385, "uuid": "de728419-7ced-4040-b895-4ce807845e12", "attributeTranslation": "Episode van ademhalingsfalen binnen 8 weken voorafgaand aan de screening", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552386, "uuid": "de728419-7ced-4040-b895-4ce807845e12", "attributeTranslation": "\u00c9pisode d\u2019insuffisance respiratoire dans les 8 semaines pr\u00e9c\u00e9dant la s\u00e9lection", "language": 10, "languageDescription": "French"}]}, {"id": 1342676, "number": 11, "principalExclusionCriteria": "Symptomatic cardiomyopathy or heart failure and/or left ventricular ejection fraction <45%", "principalExclusionCriteriaTranslations": [{"id": 9552388, "uuid": "b198b1fa-8d97-4002-a758-45d4406207e6", "attributeTranslation": "Symptomatische cardiomyopathie of hartfalen en/of linkerventrikel ejectiefractie <45%", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552387, "uuid": "b198b1fa-8d97-4002-a758-45d4406207e6", "attributeTranslation": "Cardiomyopathie symptomatique ou insuffisance cardiaque et/ou fraction d\u2019\u00e9jection ventriculaire gauche < 45 %", "language": 10, "languageDescription": "French"}]}, {"id": 1342677, "number": 12, "principalExclusionCriteria": "Baseline corrected QT interval using Fredericia\u2019s 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": 9552389, "uuid": "f1a62866-c9e9-40ce-9e94-70c210dff049", "attributeTranslation": "Gecorrigeerd QT-interval bij de baseline met behulp van Fredericia\u2019s 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, hypokali\u00ebmie of familiegeschiedenis van lang QT-syndroom)", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552390, "uuid": "f1a62866-c9e9-40ce-9e94-70c210dff049", "attributeTranslation": "Intervalle QT corrig\u00e9 selon la formule de Fredericia (QTcF) > 450 msec (moyenne de 3 mesures cons\u00e9cutives \u00e0 5 minutes d\u2019intervalle) ou ant\u00e9c\u00e9dents de facteurs de risque suppl\u00e9mentaires de torsades de pointes (p. ex., insuffisance cardiaque, hypokali\u00e9mie ou ant\u00e9c\u00e9dents familiaux de syndrome du QT long) \u00e0 l\u2019entr\u00e9e dans l\u2019\u00e9tude", "language": 10, "languageDescription": "French"}]}, {"id": 1342678, "number": 13, "principalExclusionCriteria": "Major surgical procedure (including scoliosis surgery) planned within 1 year of the start of study treatment", "principalExclusionCriteriaTranslations": [{"id": 9552392, "uuid": "4563f798-31da-490a-b2c8-5be1efcc79e6", "attributeTranslation": "Grote chirurgische ingreep (waaronder scoliose-operatie) gepland binnen 1 jaar v\u00f3\u00f3r de start van de onderzoeksbehandeling", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552391, "uuid": "4563f798-31da-490a-b2c8-5be1efcc79e6", "attributeTranslation": "Intervention chirurgicale majeure (y compris chirurgie de la scoliose) pr\u00e9vue dans l\u2019ann\u00e9e pr\u00e9c\u00e9dant le d\u00e9but du traitement \u00e0 l\u2019\u00e9tude", "language": 10, "languageDescription": "French"}]}, {"id": 1342679, "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": 9552393, "uuid": "51645c1d-8647-4654-ae0a-dcef0cea9330", "attributeTranslation": "Asthme mal contr\u00f4l\u00e9 ou maladie pulmonaire sous-jacente telle que bronchite, bronchectasie, emphys\u00e8me, pneumonie r\u00e9currente qui, de l\u2019avis de l\u2019investigateur, pourrait avoir un impact sur la fonction respiratoire", "language": 10, "languageDescription": "French"}, {"id": 9552394, "uuid": "51645c1d-8647-4654-ae0a-dcef0cea9330", "attributeTranslation": "Slecht gecontroleerde astma of onderliggende longziekte zoals bronchitis, bronchi\u00ebctasie, emfyseem, herhaalde longontsteking die naar het oordeel van de onderzoeker invloed kan hebben op de ademhalingsfunctie", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 1342680, "number": 15, "principalExclusionCriteria": "Platelets, white blood cells and/or haemoglobin < lower limit of normal (LLN) at screening", "principalExclusionCriteriaTranslations": [{"id": 9552395, "uuid": "45a8ba8b-d3e8-49cd-addf-646d65c9ec9c", "attributeTranslation": "Plaquettes, globules blancs et/ou h\u00e9moglobine < limite inf\u00e9rieure de la normale (LIN) \u00e0 la s\u00e9lection (Remarque : en cas de r\u00e9sultats anormaux < LIN aux tests de laboratoire de la s\u00e9lection, la num\u00e9ration plaquettaire, les globules blancs et l\u2019h\u00e9moglobine seront r\u00e9p\u00e9t\u00e9s une fois ; si le r\u00e9sultat du test r\u00e9p\u00e9t\u00e9 est toujours < LIN, le patient devra \u00eatre exclu)", "language": 10, "languageDescription": "French"}, {"id": 9552396, "uuid": "45a8ba8b-d3e8-49cd-addf-646d65c9ec9c", "attributeTranslation": "Bloedplaatjes, witte bloedcellen en/of hemoglobine < ondergrens van normaal (LLN) bij de screening", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 1342681, "number": 16, "principalExclusionCriteria": "Fasting triglycerides >300 mg/dL (3.42 mmol/L) at screening", "principalExclusionCriteriaTranslations": [{"id": 9552398, "uuid": "97b2ce25-f67d-40b9-b19e-0ace8deb3916", "attributeTranslation": "Nuchtere triglyceriden > 300 mg/dl (3,42 mmol/l) bij de screening", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552397, "uuid": "97b2ce25-f67d-40b9-b19e-0ace8deb3916", "attributeTranslation": "Triglyc\u00e9rides \u00e0 jeun > 300 mg/dl (3,42 mmol/l) \u00e0 la s\u00e9lection (Remarque : si la valeur est > 300 mg/dl, le dosage des triglyc\u00e9rides sera r\u00e9p\u00e9t\u00e9 une fois ; si le r\u00e9sultat du test r\u00e9p\u00e9t\u00e9 est toujours > 300 mg/dl, le patient devra \u00eatre exclu)", "language": 10, "languageDescription": "French"}]}, {"id": 1342682, "number": 17, "principalExclusionCriteria": "Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, >1.5 \u00d7 upper limit of normal [ULN]), unless secondary to Gilbert disease or pattern consistent with Gilbert disease", "principalExclusionCriteriaTranslations": [{"id": 9552400, "uuid": "723fe68a-a38a-4620-84ab-b003b7670559", "attributeTranslation": "Maladie ou insuffisance h\u00e9patique actuelle ou ant\u00e9rieure, y compris, mais sans s\u2019y limiter, un taux de bilirubine totale \u00e9lev\u00e9 \u00e0 l\u2019entr\u00e9e dans l\u2019\u00e9tude (c.-\u00e0-d. > 1,5 \u00d7 la limite sup\u00e9rieure de la normale [LSN]), \u00e0 moins qu\u2019il ne soit secondaire \u00e0 la maladie de Gilbert ou qu\u2019il ne corresponde \u00e0 la maladie de Gilbert", "language": 10, "languageDescription": "French"}, {"id": 9552399, "uuid": "723fe68a-a38a-4620-84ab-b003b7670559", "attributeTranslation": "Huidige of voorgeschiedenis van leverziekte of -insuffici\u00ebntie, inclusief maar niet beperkt tot een baseline van verhoogd totaal bilirubine (d.w.z. > 1,5 \u00d7 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": 1342683, "number": 18, "principalExclusionCriteria": "Inadequate renal function, as defined by serum Cystatin C result >2 \u00d7 ULN", "principalExclusionCriteriaTranslations": [{"id": 9552401, "uuid": "9c28eeb6-8b28-429d-9583-012681774b13", "attributeTranslation": "Onvoldoende nierfunctie, zoals gedefinieerd met een cystatine C-serumresultaat > 2 \u00d7 ULN", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552402, "uuid": "9c28eeb6-8b28-429d-9583-012681774b13", "attributeTranslation": "Fonction r\u00e9nale inad\u00e9quate, telle que d\u00e9finie par un r\u00e9sultat de cystatine C s\u00e9rique > 2 \u00d7 LSN (Remarque : si la valeur est > 2 \u00d7 LSN, le test de cystatine C s\u00e9rique sera r\u00e9p\u00e9t\u00e9 une fois ; si le r\u00e9sultat du test r\u00e9p\u00e9t\u00e9 est toujours > 2 \u00d7 LSN, le patient devra \u00eatre\nexclu)", "language": 9, "languageDescription": "Finnish"}]}, {"id": 1342684, "number": 19, "principalExclusionCriteria": "Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening", "principalExclusionCriteriaTranslations": [{"id": 9552403, "uuid": "ee0708a0-264e-4a1f-bf31-3e9b364436e4", "attributeTranslation": "Test positif pour l\u2019antig\u00e8ne de surface de l\u2019h\u00e9patite B, l\u2019anticorps de l\u2019h\u00e9patite C ou le virus de l\u2019immunod\u00e9ficience humaine \u00e0 la s\u00e9lection", "language": 10, "languageDescription": "French"}, {"id": 9552404, "uuid": "ee0708a0-264e-4a1f-bf31-3e9b364436e4", "attributeTranslation": "Positieve test voor hepatitis B-oppervlakteantigeen, hepatitis C-virus antilichaamserologie of hiv bij de screening", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 1342685, "number": 2, "principalExclusionCriteria": "Have exposure to any dystrophin restoration product within 6 months prior to the start of study treatment", "principalExclusionCriteriaTranslations": [{"id": 9552406, "uuid": "d32059f8-97ec-4dda-9347-c9978c24bfe0", "attributeTranslation": "Exposition \u00e0 un produit de restauration de la dystrophine (p. ex., ataluren, saut d\u2019exon) dans les 6 mois pr\u00e9c\u00e9dant le d\u00e9but du traitement \u00e0 l\u2019\u00e9tude", "language": 10, "languageDescription": "French"}, {"id": 9552405, "uuid": "d32059f8-97ec-4dda-9347-c9978c24bfe0", "attributeTranslation": "Blootstelling aan een dystrofine-herstellend product binnen 6 maanden voorafgaand aan de start van de onderzoeksbehandeling", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 1342686, "number": 20, "principalExclusionCriteria": "Hypersensitivity to any component of study medication", "principalExclusionCriteriaTranslations": [{"id": 9552407, "uuid": "c8817860-ab3b-4ebb-8eba-b2de224a0e93", "attributeTranslation": "Overgevoeligheid voor een bestanddeel van het onderzoeksmiddel", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552408, "uuid": "c8817860-ab3b-4ebb-8eba-b2de224a0e93", "attributeTranslation": "Hypersensibilit\u00e9 \u00e0 l\u2019un des composants du m\u00e9dicament \u00e0 l\u2019\u00e9tude", "language": 10, "languageDescription": "French"}]}, {"id": 1342687, "number": 21, "principalExclusionCriteria": "Sorbitol intolerance or malabsorption, or the hereditary form of fructose intolerance", "principalExclusionCriteriaTranslations": [{"id": 9552410, "uuid": "c06e7603-ce54-45aa-88e1-c7fe8726ae1a", "attributeTranslation": "Intolerantie of malabsorptie van sorbitol, of de erfelijke vorm van fructose-intolerantie", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552409, "uuid": "c06e7603-ce54-45aa-88e1-c7fe8726ae1a", "attributeTranslation": "Intol\u00e9rance ou malabsorption du sorbitol, ou forme h\u00e9r\u00e9ditaire d\u2019intol\u00e9rance au fructose", "language": 10, "languageDescription": "French"}]}, {"id": 1342688, "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": 9552411, "uuid": "270f721d-fc5c-42db-a9ba-fdbf1f429d9f", "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": 9552412, "uuid": "270f721d-fc5c-42db-a9ba-fdbf1f429d9f", "attributeTranslation": "Diagnostic d\u2019autres maladies neurologiques non contr\u00f4l\u00e9es ou pr\u00e9sence de troubles somatiques pertinents non contr\u00f4l\u00e9s qui ne sont pas li\u00e9s \u00e0 la DMD, selon l\u2019avis de l\u2019investigateur", "language": 10, "languageDescription": "French"}]}, {"id": 1342689, "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": 9552413, "uuid": "3d5076b3-ca02-40c4-8193-795572a2a16e", "attributeTranslation": "Psychiatrische ziekte of sociale omstandigheden waardoor de potenti\u00eble pati\u00ebnt 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": 9552414, "uuid": "3d5076b3-ca02-40c4-8193-795572a2a16e", "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 proc\u00e9dures du protocole de l\u2019\u00e9tude, selon l\u2019avis de l\u2019investigateur", "language": 10, "languageDescription": "French"}]}, {"id": 1342690, "number": 24, "principalExclusionCriteria": "Have contraindications to Magnetic Resonance Imaging (MRI) scan (eg, claustrophobia, metal implants, or uncontrolled seizure disorder), based on Investigator\u2019s judgement", "principalExclusionCriteriaTranslations": [{"id": 9552415, "uuid": "76b5961e-47e2-43a6-878a-45831b1b29e6", "attributeTranslation": "Avoir des contre-indications \u00e0 l\u2019imagerie par r\u00e9sonance magn\u00e9tique (IRM) (p. ex., claustrophobie, implants m\u00e9talliques ou troubles \u00e9pileptiques non contr\u00f4l\u00e9s), selon l\u2019avis de l\u2019investigateur", "language": 10, "languageDescription": "French"}, {"id": 9552416, "uuid": "76b5961e-47e2-43a6-878a-45831b1b29e6", "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": 1342691, "number": 3, "principalExclusionCriteria": "Having received any gene therapy prior to start of study treatment", "principalExclusionCriteriaTranslations": [{"id": 9552417, "uuid": "2e5a1d6b-88a2-46e7-baeb-36d147b794ae", "attributeTranslation": "Een gentherapie hebben ontvangen voorafgaand aan de start van de onderzoeksbehandeling", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552418, "uuid": "2e5a1d6b-88a2-46e7-baeb-36d147b794ae", "attributeTranslation": "Avoir re\u00e7u une th\u00e9rapie g\u00e9nique (p. ex. virus ad\u00e9no-associ\u00e9s, administration de microdystrophine) avant le d\u00e9but du traitement \u00e0 l\u2019\u00e9tude", "language": 10, "languageDescription": "French"}]}, {"id": 1342692, "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": 9552419, "uuid": "8fe5bb83-6a7f-440d-81e4-0a024d40961b", "attributeTranslation": "Gebruik van een farmacologische behandeling of supplement, anders dan corticostero\u00efden, 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": 9552420, "uuid": "8fe5bb83-6a7f-440d-81e4-0a024d40961b", "attributeTranslation": "Utilisation de tout traitement ou compl\u00e9ment pharmacologique (autre que des cortico\u00efdes) qui pourrait avoir un effet sur la force ou la fonction musculaire dans les 3 mois pr\u00e9c\u00e9dant le d\u00e9but du traitement \u00e0 l\u2019\u00e9tude (p. ex., hormone de croissance); la vitamine D, le calcium et tout autre compl\u00e9ment seront autoris\u00e9s", "language": 10, "languageDescription": "French"}]}, {"id": 1342693, "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": 9552421, "uuid": "dc31e454-1347-42ae-82f9-ff4455223515", "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": 9552422, "uuid": "dc31e454-1347-42ae-82f9-ff4455223515", "attributeTranslation": "Utilisation de testost\u00e9rone, sauf si elle est utilis\u00e9e comme traitement substitutif pour prendre en charge un retard pubertaire La dose et le sch\u00e9ma th\u00e9rapeutique de testost\u00e9rone doivent \u00eatre stables dans les 6 mois pr\u00e9c\u00e9dant le d\u00e9but du traitement \u00e0 l\u2019\u00e9tude, et les taux de testost\u00e9rone circulante doivent se situer dans les plages normales\npour l\u2019\u00e2ge du/de la patient(e)", "language": 10, "languageDescription": "French"}]}, {"id": 1342694, "number": 6, "principalExclusionCriteria": "Elbow-flexion contractures >30\u00b0 in the dominant arm", "principalExclusionCriteriaTranslations": [{"id": 9552424, "uuid": "2ea7947e-9ef3-44ed-b386-3346be87ee6b", "attributeTranslation": "Elleboogflexie contracturen > 30\u00b0 in de dominante arm", "language": 37, "languageDescription": "Dutch (Netherlands)"}, {"id": 9552423, "uuid": "2ea7947e-9ef3-44ed-b386-3346be87ee6b", "attributeTranslation": "Contractures de flexion du coude > 30\u00b0 dans le groupe dominant", "language": 10, "languageDescription": "French"}]}, {"id": 1342695, "number": 7, "principalExclusionCriteria": "Inability to perform consistent PUL 2.0 measurement within \u00b12 points without shoulder domain or within \u00b13 points with shoulder domain during paired testing at screening", "principalExclusionCriteriaTranslations": [{"id": 9552425, "uuid": "54e917ec-ecbb-4f67-8616-a4c16316005f", "attributeTranslation": "Incapacit\u00e9 \u00e0 effectuer une mesure coh\u00e9rente du score PUL 2.0 \u00e0 \u00b1 2 points sans le domaine de l\u2019\u00e9paule ou \u00e0 \u00b1 3 points avec le domaine de l\u2019\u00e9paule lors des tests appari\u00e9s \u00e0 la s\u00e9lection", "language": 10, "languageDescription": "French"}, {"id": 9552426, "uuid": "54e917ec-ecbb-4f67-8616-a4c16316005f", "attributeTranslation": "Niet in staat om consistente PUL 2.0-meting uit te voeren binnen \u00b1 2 punten zonder schouderdomein of binnen \u00b13 punten met schouderdomein, tijdens gepaarde testen bij de screening", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 1342696, "number": 8, "principalExclusionCriteria": "Forced Vital Capacity % of predicted <40%", "principalExclusionCriteriaTranslations": [{"id": 9552427, "uuid": "78f72779-d623-4951-95d8-2a9cfcfca69e", "attributeTranslation": "Capacit\u00e9 vitale forc\u00e9e (CVF) en pourcentage de la valeur pr\u00e9dite < 40 %", "language": 10, "languageDescription": "French"}, {"id": 9552428, "uuid": "78f72779-d623-4951-95d8-2a9cfcfca69e", "attributeTranslation": "Percentage voorspelde geforceerde vitale capaciteit (FVC) < 40%", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 1342697, "number": 9, "principalExclusionCriteria": "Requirement for daytime ventilator assistance", "principalExclusionCriteriaTranslations": [{"id": 9552429, "uuid": "ca384f56-1236-435a-9daa-f0fa5dd2454a", "attributeTranslation": "N\u00e9cessit\u00e9 d\u2019une assistance ventilatoire de jour. Remarque : L\u2019assistance respiratoire de nuit et l\u2019utilisation d\u2019un traitement par pression positive \u00e0 deux niveaux sont autoris\u00e9es", "language": 10, "languageDescription": "French"}, {"id": 9552430, "uuid": "ca384f56-1236-435a-9daa-f0fa5dd2454a", "attributeTranslation": "Beademingsondersteuning overdag vereist", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}]}, "endPoint": {"primaryEndPoints": [{"id": 917801, "number": 1, "endPoint": "Change of PUL total score at 18 months of treatment of givinostat compared to placebo group", "isPrimary": true, "endPointTranslations": [{"id": 9552378, "uuid": "c128d46e-4959-4956-861e-b9b5a0a175b8", "attributeTranslation": "\u2022\tVariation du score total PUL \u00e0 18 mois de traitement par givinostat par rapport au groupe placebo.", "language": 10, "languageDescription": "French"}, {"id": 9552377, "uuid": "c128d46e-4959-4956-861e-b9b5a0a175b8", "attributeTranslation": "Verandering in de PUL-totaalscore na 18 maanden behandeling met givinostat vergeleken met de placebogroep.", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}], "secondaryEndPoints": [{"id": 917802, "number": 1, "endPoint": "\u2022Change from baseline of Peak Expiratory Flow percent predicted (PEF%p) at 18 months of treatment of givinostat compared to placebo group \u2022 Change from baseline of Forced Vital Capacity percent predicted (FVC%p) at 18 months of treatment of givinostat compared to placebo group \u2022 Cumulative loss of PUL items over 18 months of treatment of givinostat compared to placebo group", "isPrimary": false, "endPointTranslations": [{"id": 9552379, "uuid": "ffff8064-bf92-41bc-bca0-dec09e743ccb", "attributeTranslation": "\u2022\tVariation par rapport \u00e0 la r\u00e9f\u00e9rence du d\u00e9bit expiratoire de pointe en pourcentage de la valeur pr\u00e9dite (% DEPp) \u00e0 18 mois de traitement par givinostat par rapport au groupe placebo\n\u2022\tVariation par rapport \u00e0 la r\u00e9f\u00e9rence de la capacit\u00e9 vitale forc\u00e9e en pourcentage de la valeur pr\u00e9dite (% CVFp) \u00e0 18 mois de traitement par givinostat par rapport au groupe placebo\n\u2022\tPerte cumul\u00e9e des items PUL apr\u00e8s 18 mois de traitement par givinostat par rapport au groupe placebo.", "language": 10, "languageDescription": "French"}, {"id": 9552380, "uuid": "ffff8064-bf92-41bc-bca0-dec09e743ccb", "attributeTranslation": "Verandering ten opzichte van de baseline van het percentage voorspelde piekstroom (PEF%p) na 18 maanden behandeling met givinostat vergeleken met de placebogroep \u2022Verandering ten opzichte van de baseline van percentage voorspelde geforceerde vitale capaciteit (FVC%p) na 18 maanden behandeling met givinostat vergeleken met de placebogroep \u2022Cumulatief verlies van PUL-items gedurende 18 maanden behandeling met givinostat vergeleken met de placebogroep", "language": 37, "languageDescription": "Dutch (Netherlands)"}]}, {"id": 917803, "number": 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"languageDescription": "French"}, {"id": 9552382, "uuid": "81c953c9-9c41-4b24-b283-e6207e3ffaff", "attributeTranslation": "Type, incidentie en ernst van tijdens de behandeling optredende bijwerkingen (TEAE\u2019s) \u2022 Percentage pati\u00ebnten met TEAE\u2019s (baseline tot EOS/follow-up) \u2022 veranderingen in vitale functies, lab onderzoeken, ECG, ECHO \u2022 Tijd tot ondersteuning met beademing, frequentie en ernst van luchtweginfectie\n\nen duur en gebruik van antibiotica met givinostat vergeleken met de placebogroep \u2022 tijd tot begin van diarree \u2022 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": 85009, "organisationName": "Italfarmaco"}], "populationOfTrialSubjects": {"ageRanges": [{"id": 320313, "ageRangeCategoryCode": "2", "ageRangeCategory": "2"}], 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serious breach and impacts on trial is provided in the attachment separately.", "actionsTaken": "Description of actions taken and planned is provided in the attachment separately.", "isBenefitRiskBalanceChanged": true, "benefitRiskBalanceChangeDescription": "Although  no subjects were screened at time of the incident, Italfarmaco assessed the potential unblinding issue may have  impact on the data integrity and  the double-blind fashion of the  study could not be ensure without further remediation.", "mscs": ["Belgium", "France", "Germany", "Italy", "Netherlands"], "seriousBreachSites": [{"organisationAddressInfo": {"id": 77160, "organisation": {"id": 79684, "type": "PHARMACEUTICAL_COMPANY", "typeCode": "10", "name": "Italfarmaco S.p.A.", "businessKey": "ORG-100000118"}, "address": {"addressId": 80910, "oneLine": "Via Dei Lavoratori 54", "addressLine1": "Via Dei Lavoratori 54", "city": "Cinisello Balsamo", "postcode": "20092", "country": 2018, "countryName": "Italy"}, "businessKey": 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"mpRoleInTrial": "3", "orphanDrugEdit": false, "productChangedRelationMA": true, "productChangeDescription": "GNT0004 placebo is a sterile solution intended to be administered after dilution by infusion. GNT0004 placebo is composed by the buffer used in the composition of GNT0004 Drug product", "evCode": "PRD2085419", "devices": [], "characteristics": [], "routes": ["PARENTERAL USE"], "allSubstancesChemicals": true, "productName": "RINGER LACTATE FRESENIUS KABI FRANCE, solution pour perfusion", "jsonActiveSubstanceNames": "sodium lactate solution, potassium chloride, sodium chloride, calcium chloride dihydrate", "pharmaceuticalFormDisplay": "SOLUTION FOR INJECTION/INFUSION"}], "trialDetails": {"clinicalTrialIdentifiers": {"fullTitle": "Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys", "fullTitleTranslations": [{"id": 9971114, "uuid": "e9acbd29-f812-4bc3-b6d6-de6f856335f9", "attributeTranslation": "Klinische Pr\u00fcfung zur Gentherapie mit Mikro-Dystrophin (GNT0004) bei Patienten mit Duchenne-Muskeldystrophie\nEine Studie der Phasen I, II und III mit einem Dosisfindungsabschnitt, einer anschlie\u00dfenden Beurteilung der Wirksamkeit und Sicherheit und einem vierfach verblindeten placebokontrollierten Abschnitt sowie einer abschlie\u00dfenden Nachbeobachtung zur Beurteilung der Langzeit-Sicherheit bei gehf\u00e4higen Jungen", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971113, "uuid": "e9acbd29-f812-4bc3-b6d6-de6f856335f9", "attributeTranslation": "Essai clinique d\u2019une th\u00e9rapie g\u00e9nique avec une microdystrophine (GNT0004) dans la myopathie de Duchenne: \u00c9tude de phase I/II/III avec une \u00e9tape de d\u00e9termination de dose suivie d\u2019une \u00e9tape d\u2019\u00e9valuation de l\u2019efficacit\u00e9 et de la s\u00e9curit\u00e9, en quadruple aveugle comparativement \u00e0 un placebo, suivie d\u2019une \u00e9tape de surveillance \u00e0 long terme chez des gar\u00e7ons ambulatoires", "language": 34, "languageDescription": "French (France)"}, {"id": 9971112, "uuid": "e9acbd29-f812-4bc3-b6d6-de6f856335f9", "attributeTranslation": "Klinische studie naar microdystrofine (GNT0004) gentherapie bij Duchenne spierdystrofie\nEen fase I/II/III-studie met een dosisbepalingsdeel, gevolgd door een viervoudig blind placebo-gecontroleerd deel ter evaluatie van de werkzaamheid en veiligheid, en daarna door een langetermijn-veiligheidsopvolgingsdeel bij ambulante jongens", "language": 36, "languageDescription": "Dutch (Belgium)"}], "publicTitle": "A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1). After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2). In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3).", "publicTitleTranslations": [{"id": 9971140, "uuid": "8c424b2f-aef4-465c-a09a-fa0b20973779", "attributeTranslation": "Une \u00e9tude clinique en 3 parties avec une microdystrophine (appel\u00e9e GNT0004), une nouvelle th\u00e9rapie g\u00e9nique chez les gar\u00e7ons atteints de la maladie de Duchenne qui peuvent encore marcher. L'\u00e9tude commencera par la recherche de la dose de traitement appropri\u00e9e (partie 1). Ensuite, une \u00e9tude comparative avec un placebo d\u00e9butera pour \u00e9valuer la s\u00e9curit\u00e9 et l'efficacit\u00e9 de la dose appropri\u00e9e de cette th\u00e9rapie (partie 2). Enfin, une p\u00e9riode de suivi permettra d'\u00e9tudier la s\u00e9curit\u00e9 et l'efficacit\u00e9 du traitement sur une plus longue p\u00e9riode (partie 3).", "language": 34, "languageDescription": "French (France)"}, {"id": 9971141, "uuid": "8c424b2f-aef4-465c-a09a-fa0b20973779", "attributeTranslation": "Eine klinische Studie in 3 Teilen mit einem Mikro-Dystrophin (mit der Bezeichnung GNT0004), einer neuen Gentherapie bei Jungen mit Duchenne-Muskeldystrophie, die noch laufen k\u00f6nnen. Die Studie beginnt mit der Ermittlung der richtigen Behandlungsdosis (Teil 1). Danach wird in einer vergleichenden Studie mit Placebo die Sicherheit und Wirksamkeit der richtigen Dosis dieser Therapie bewertet (Teil 2). Am Ende folgt eine Nachbeobachtungsphase zur weiteren Untersuchung der Sicherheit und Wirksamkeit \u00fcber einen l\u00e4ngeren Zeitraum (Teil 3).", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971142, "uuid": "8c424b2f-aef4-465c-a09a-fa0b20973779", "attributeTranslation": "Een klinisch onderzoek in 3 delen met een microdystrofine (GNT0004 genaamd), een nieuwe gentherapie bij jongens met de ziekte van Duchenne die nog kunnen lopen. Het onderzoek begint met het zoeken naar de juiste behandeldosis (deel 1). Daarna gaat een vergelijkend onderzoek met een placebo van start ter bepaling van de veiligheid en effectiviteit van de juiste dosis van deze behandeling (deel 2). Tot slot wordt er in een opvolgingsperiode nader onderzoek gedaan naar de veiligheid en werkzaamheid van de behandeling gedurende een langere periode (deel 3).", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971139, "uuid": "8c424b2f-aef4-465c-a09a-fa0b20973779", "attributeTranslation": "Une \u00e9tude clinique en 3 parties avec une microdystrophine (appel\u00e9e GNT0004), une nouvelle th\u00e9rapie g\u00e9nique chez les gar\u00e7ons atteints de la maladie de Duchenne qui peuvent encore marcher. L'\u00e9tude commencera par la recherche de la dose de traitement appropri\u00e9e (partie 1). Ensuite, une \u00e9tude comparative avec un placebo d\u00e9butera pour \u00e9valuer la s\u00e9curit\u00e9 et l'efficacit\u00e9 de la dose appropri\u00e9e de cette th\u00e9rapie (partie 2). Enfin, une p\u00e9riode de suivi permettra d'\u00e9tudier la s\u00e9curit\u00e9 et l'efficacit\u00e9 du traitement sur une plus longue p\u00e9riode (partie 3).", "language": 33, "languageDescription": "French (Belgium)"}], "shortTitle": "GNT-016-MDYF", "secondaryIdentifyingNumbers": {"additionalRegistries": []}}, "trialInformation": {"trialCategory": {"isLowIntervention": false, "trialPhase": "7", "trialCategory": "1", "justificationForTrialCategory": "Phase I/II trial", "trialCategoryId": 110893}, "medicalCondition": {"partIMedicalConditions": [{"id": 125855, "medicalCondition": "Duchenne Muscular Dystrophy", "medicalConditionTranslations": [{"id": 9971086, "uuid": "e05e990c-93f9-4e56-981a-0ccb53e0b165", "attributeTranslation": "Duchenne spierdystrofie", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971087, "uuid": "e05e990c-93f9-4e56-981a-0ccb53e0b165", "attributeTranslation": "Duchenne-Muskeldystrophie", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971085, "uuid": "e05e990c-93f9-4e56-981a-0ccb53e0b165", "attributeTranslation": "myopathie de Duchenne", "language": 33, "languageDescription": "French (Belgium)"}], "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": 366186}, {"code": "6", "trialScopeId": 366188}, {"code": "5", "trialScopeId": 366187}, {"code": "9", "trialScopeId": 366183}, {"code": "3", "trialScopeId": 366185}, {"code": "7", "trialScopeId": 366184}], "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. To assess the safety and tolerability of IMP", "mainObjectiveTranslations": [{"id": 9971135, "uuid": "9ca48550-d266-4c8d-b1c8-ea43f87b0ac0", "attributeTranslation": "Teil 1: Bestimmung der Dosis des Pr\u00fcfpr\u00e4parats, d. h. einer sicheren und vertr\u00e4glichen Dosis mit akzeptabler Genexpression, die in Teil 2 \u00fcbernommen werden soll\nTeil 2: Mit der gew\u00e4hlten Dosis des Pr\u00fcfpr\u00e4parats: Nachweis der klinischen Wirksamkeit des Pr\u00fcfpr\u00e4parats gegen\u00fcber Placebo\nTeil 3: Bewertung der langfristigen Sicherheit und Vertr\u00e4glichkeit des Pr\u00fcfpr\u00e4parats", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971136, "uuid": "9ca48550-d266-4c8d-b1c8-ea43f87b0ac0", "attributeTranslation": "\u00c9tude de phase I/II/III comprenant 3 parties : \nPartie 1 : - D\u00e9terminer la dose de la TG: c\u2019est-\u00e0-dire une dose s\u00fbre et bien tol\u00e9r\u00e9e montrant une expression transg\u00e9nique acceptable, pour son \u00e9valuation en Partie 2 \nPartie 2 : Avec la dose s\u00e9lectionn\u00e9e de la TG: - D\u00e9montrer son efficacit\u00e9 clinique par rapport \u00e0 un placebo un an apr\u00e8s le traitement par la TG - \u00c9valuer la s\u00e9curit\u00e9 et la tol\u00e9rance par rapport \u00e0 un placebo un an apr\u00e8s l\u2019inclusion \nPartie 3 : - \u00c9valuer la s\u00e9curit\u00e9 et la tol\u00e9rance \u00e0 long terme de la TG", "language": 34, "languageDescription": "French (France)"}, {"id": 9971137, "uuid": "9ca48550-d266-4c8d-b1c8-ea43f87b0ac0", "attributeTranslation": "Partie 1 : D\u00e9terminer la dose de m\u00e9dicament exp\u00e9rimental s\u00fbre et tol\u00e9rable avec une expression g\u00e9nique acceptable, pour passer \u00e0 la partie 2\nPartie 2 : Avec la dose s\u00e9lectionn\u00e9e du m\u00e9dicament exp\u00e9rimental : D\u00e9montrer l\u2019efficacit\u00e9 clinique du m\u00e9dicament exp\u00e9rimental par rapport au placebo\nPartie 3 : \u00c9valuer la s\u00e9curit\u00e9 et la tol\u00e9rabilit\u00e9 du m\u00e9dicament exp\u00e9rimental sur le long terme", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971138, "uuid": "9ca48550-d266-4c8d-b1c8-ea43f87b0ac0", "attributeTranslation": "Deel 1: Om de dosis van het studiegeneesmiddel te bepalen: d.w.z. een veilige en verdraagbare dosis met een aanvaardbare genexpressie, om over te dragen naar Deel 2\nDeel 2: Met de geselecteerde dosis van het studiegeneesmiddel: Om de klinische werkzaamheid ten opzichte van de placebo aan te tonen \nDeel 3: Om de veiligheid en verdraagbaarheid van het studiegeneesmiddel op lange termijn te beoordelen", "language": 36, "languageDescription": "Dutch (Belgium)"}], "secondaryObjectives": [{"id": 425871, "number": 1, "secondaryObjective": "Part 2: To assess the biodistribution of IMP", "secondaryObjectiveTranslations": [{"id": 9971145, "uuid": "89c333d1-8bc0-4ba0-823b-8d237ef66dd1", "attributeTranslation": "-\tBewertung der Biodistribution des Pr\u00fcfpr\u00e4parats", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971146, "uuid": "89c333d1-8bc0-4ba0-823b-8d237ef66dd1", "attributeTranslation": "Om de biodistributie van het studiegeneesmiddel te beoordelen", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971143, "uuid": "89c333d1-8bc0-4ba0-823b-8d237ef66dd1", "attributeTranslation": "Partie 2: \u00c9valuer la biodistribution de la TG", "language": 34, "languageDescription": "French (France)"}, {"id": 9971144, "uuid": "89c333d1-8bc0-4ba0-823b-8d237ef66dd1", "attributeTranslation": "\u00c9valuer la biodistribution du m\u00e9dicament exp\u00e9rimental", "language": 33, "languageDescription": "French (Belgium)"}]}, {"id": 425872, "number": 2, "secondaryObjective": "Part 2:To demonstrate the pharmacodynamic activity of IMP", "secondaryObjectiveTranslations": [{"id": 9971149, "uuid": "0574d14a-bf1a-4be7-9235-f1dec4ecdfb2", "attributeTranslation": "Partie 2: D\u00e9montrer l\u2019activit\u00e9 pharmacodynamique de la TG", "language": 34, "languageDescription": "French (France)"}, {"id": 9971150, "uuid": "0574d14a-bf1a-4be7-9235-f1dec4ecdfb2", "attributeTranslation": "D\u00e9montrer l\u2019activit\u00e9 pharmacodynamique du m\u00e9dicament exp\u00e9rimental", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971147, "uuid": "0574d14a-bf1a-4be7-9235-f1dec4ecdfb2", "attributeTranslation": "Om de farmacodynamische activiteit van het studiegeneesmiddel aan te tonen", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971148, "uuid": "0574d14a-bf1a-4be7-9235-f1dec4ecdfb2", "attributeTranslation": "Nachweis der pharmakodynamischen Aktivit\u00e4t des Pr\u00fcfpr\u00e4parats", "language": 28, "languageDescription": "German (Belgium)"}]}, {"id": 425873, "number": 3, "secondaryObjective": "Part 2: To assess the immunogenicity of IMP", "secondaryObjectiveTranslations": [{"id": 9971154, "uuid": "6592fb8d-ecd2-4e07-bc77-e4fd81279c6b", "attributeTranslation": "\u00c9valuer l\u2019immunog\u00e9nicit\u00e9 du m\u00e9dicament exp\u00e9rimental", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971151, "uuid": "6592fb8d-ecd2-4e07-bc77-e4fd81279c6b", "attributeTranslation": "Bewertung der Immunogenit\u00e4t des Pr\u00fcfpr\u00e4parats", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971152, "uuid": "6592fb8d-ecd2-4e07-bc77-e4fd81279c6b", "attributeTranslation": "Om de immunogeniciteit van het studiegeneesmiddel te beoordelen", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971153, "uuid": "6592fb8d-ecd2-4e07-bc77-e4fd81279c6b", "attributeTranslation": "Partie 2: \u00c9valuer l\u2019immunog\u00e9nicit\u00e9 de la TG", "language": 34, "languageDescription": "French (France)"}]}, {"id": 425874, "number": 4, "secondaryObjective": "Part 2:To compare the efficacy on the disease course after 2 years after inclusion, between patients treated with active IMP at first and patients treated after a delay of one year", "secondaryObjectiveTranslations": [{"id": 9971156, "uuid": "5e092971-8927-4c2b-9143-579e7ec239b5", "attributeTranslation": "Partie 2: Comparer l\u2019efficacit\u00e9 sur l\u2019\u00e9volution de la maladie, 2 ans apr\u00e8s l\u2019inclusion, entre les patients trait\u00e9s d'abord par la TG et les patients trait\u00e9s apr\u00e8s un d\u00e9lai d\u2019un an", "language": 34, "languageDescription": "French (France)"}, {"id": 9971157, "uuid": "5e092971-8927-4c2b-9143-579e7ec239b5", "attributeTranslation": "Teil 2: Vergleich der Wirksamkeit und der Biomarker im Krankheitsverlauf zwischen Studienteilnehmern, die zuerst mit dem aktiven Pr\u00fcfpr\u00e4parat behandelt wurden, und Studienteilnehmern, die mit einem Jahr Verz\u00f6gerung behandelt wurden.", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971155, "uuid": "5e092971-8927-4c2b-9143-579e7ec239b5", "attributeTranslation": "Partie 2: Comparer l\u2019efficacit\u00e9 et les taux de biomarqueurs sur l\u2019\u00e9volution de la maladie entre les participants trait\u00e9s par le m\u00e9dicament exp\u00e9rimental actif d\u00e8s le d\u00e9but et les participants trait\u00e9s apr\u00e8s un d\u00e9lai d\u2019un an.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971158, "uuid": "5e092971-8927-4c2b-9143-579e7ec239b5", "attributeTranslation": "Deel 2: Om de werkzaamheid en biomarkers voor deze ziekte te vergelijken bij deelnemers die eerst behandeld werden met het actieve studiegeneesmiddel ten opzichte van deelnemers die na een jaar pas behandeld werden.", "language": 36, "languageDescription": "Dutch (Belgium)"}]}]}, "eligibilityCriteria": {"principalInclusionCriteria": [{"id": 813970, "number": 1, "principalInclusionCriteria": "Ambulant Male", "principalInclusionCriteriaTranslations": [{"id": 9971117, "uuid": "a3e45bb2-b98a-469b-a1f6-867f2cca0e5a", "attributeTranslation": "Gar\u00e7ons ambulatoires", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971115, "uuid": "a3e45bb2-b98a-469b-a1f6-867f2cca0e5a", "attributeTranslation": "De sexe masculin et ambulant", "language": 34, "languageDescription": "French (France)"}, {"id": 9971118, "uuid": "a3e45bb2-b98a-469b-a1f6-867f2cca0e5a", "attributeTranslation": "Ambulante jongen", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971116, "uuid": "a3e45bb2-b98a-469b-a1f6-867f2cca0e5a", "attributeTranslation": "M\u00e4nnlich und gehf\u00e4hig", "language": 28, "languageDescription": "German (Belgium)"}]}, {"id": 813971, "number": 2, "principalInclusionCriteria": "Being included in the GNT-014-MDYF study", "principalInclusionCriteriaTranslations": [{"id": 9971121, "uuid": "c54acb50-ddfa-4188-8daa-3ba618f40a58", "attributeTranslation": "Inclus dans l\u2019\u00e9tude GNT-014-MDYF", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971122, "uuid": "c54acb50-ddfa-4188-8daa-3ba618f40a58", "attributeTranslation": "Teilnahme an der GNT-014-MDYF-Studie", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971119, "uuid": "c54acb50-ddfa-4188-8daa-3ba618f40a58", "attributeTranslation": "Inclus dans l\u2019\u00e9tude GNT-014-MDYF", "language": 34, "languageDescription": "French (France)"}, {"id": 9971120, "uuid": "c54acb50-ddfa-4188-8daa-3ba618f40a58", "attributeTranslation": "Opgenomen zijn in de GNT-014-MDYF-studie", "language": 36, "languageDescription": "Dutch (Belgium)"}]}, {"id": 813972, "number": 3, "principalInclusionCriteria": "6 to 10 years (inclusive)", "principalInclusionCriteriaTranslations": [{"id": 9971125, "uuid": "660eea4a-bdbf-4964-8ac0-c3376dfa1ac0", "attributeTranslation": "\u00c2g\u00e9 de 6 \u00e0 10 ans (inclus)", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971126, "uuid": "660eea4a-bdbf-4964-8ac0-c3376dfa1ac0", "attributeTranslation": "6 bis 10 Jahre (einschlie\u00dflich)", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971124, "uuid": "660eea4a-bdbf-4964-8ac0-c3376dfa1ac0", "attributeTranslation": "6 tot en met 10 jaar", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971123, "uuid": "660eea4a-bdbf-4964-8ac0-c3376dfa1ac0", "attributeTranslation": "Age : 6 \u00e0 10 ans (inclus)", "language": 34, "languageDescription": "French (France)"}]}, {"id": 813973, "number": 4, "principalInclusionCriteria": "For participants enrolled in Part 1: BMI scale \u226475th percentile (validated chart in force in country site)  For participants enrolled in Part 2: Body weight \u226495th percentile of the BMI or body weight scale (validated chart in force in country site)", "principalInclusionCriteriaTranslations": [{"id": 9971128, "uuid": "13cd4c97-3487-45f0-9bdf-6c322bd0684e", "attributeTranslation": "Teilnehmer, die f\u00fcr Teil 1 rekrutiert wurden: BMI-Skala \u2264 75. Perzentil (validierte, g\u00fcltige Tabelle im jeweiligen Land) \nTeilnehmer, die f\u00fcr Teil 2 rekrutiert wurden: K\u00f6rpergewicht \u2264 95. Perzentil der BMI- oder K\u00f6rpergewichtsskala (validierte, g\u00fcltige Tabelle im jeweiligen Land)", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971129, "uuid": "13cd4c97-3487-45f0-9bdf-6c322bd0684e", "attributeTranslation": "Voor deelnemers die ingeschreven zijn in Deel 1: BMI-schaal \u226475ste percentiel (gevalideerde chart van kracht op de lokale site) \nVoor deelnemers die ingeschreven zijn in Deel 2: Lichaamsgewicht \u2264 95ste percentiel van de BMI of lichaamsgewichtschaal (gevalideerde chart van kracht op de lokale site)", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971127, "uuid": "13cd4c97-3487-45f0-9bdf-6c322bd0684e", "attributeTranslation": "Poids corporel \u2264 75e percentile de l'\u00e9chelle de l\u2019IMC (selon l\u2019\u00e9chelle valid\u00e9e localement)", "language": 34, "languageDescription": "French (France)"}, {"id": 9971130, "uuid": "13cd4c97-3487-45f0-9bdf-6c322bd0684e", "attributeTranslation": "Pour les participants recrut\u00e9s dans la partie 1 : IMC \u2264 75e percentile (graphique valid\u00e9 utilis\u00e9 dans le centre du pays) \nPour les participants recrut\u00e9s dans la partie 2 : Poids \u2264 95e percentile de l\u2019IMC ou du poids corporel (graphique valid\u00e9 utilis\u00e9 dans le centre du pays)", "language": 33, "languageDescription": "French (Belgium)"}]}, {"id": 813974, "number": 5, "principalInclusionCriteria": "Positive gene testing with detailed genotyping confirmation of Duchenne Muscular Dystrophy (DMD), i.e. DMD mutations expected to abolish the production of dystrophin", "principalInclusionCriteriaTranslations": [{"id": 9971133, "uuid": "141b3fa9-f07f-49dd-bbae-c7c1a3b8977d", "attributeTranslation": "Testen op positieve genen met bevestiging van een gedetailleerde genotypering van Duchenne spierdystrofie (DMD), d.w.z. er wordt verwacht dat DMD-mutaties de productie van dystrofine zullen stoppen", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971131, "uuid": "141b3fa9-f07f-49dd-bbae-c7c1a3b8977d", "attributeTranslation": "Positiver Gentest mit detaillierter Genotypisierung zur Best\u00e4tigung der Duchenne-Muskeldystrophie (DMD), d. h. DMD-Mutationen, die die Produktion von Dystrophin aufheben", "language": 29, "languageDescription": "German (Germany)"}, {"id": 9971134, "uuid": "141b3fa9-f07f-49dd-bbae-c7c1a3b8977d", "attributeTranslation": "Diagnostic g\u00e9n\u00e9tique positif avec confirmation d\u00e9taill\u00e9e du g\u00e9notype de la myopathie de Duchenne, c'est-\u00e0-dire une mutation qui conduit \u00e0 l\u2019absence de production de dystrophine, sauf pour les patients pr\u00e9sentant des mutations affectant les exons 1 \u00e0 17", "language": 34, "languageDescription": "French (France)"}, {"id": 9971132, "uuid": "141b3fa9-f07f-49dd-bbae-c7c1a3b8977d", "attributeTranslation": "Analyse g\u00e9n\u00e9tique positive avec confirmation par g\u00e9notypage d\u00e9taill\u00e9 de myopathie de Duchenne (DMD), c\u2019est-\u00e0-dire pr\u00e9sence de mutations susceptibles d\u2019abolir la production de la dystrophine", "language": 33, "languageDescription": "French (Belgium)"}]}], "principalExclusionCriteria": [{"id": 1393282, "number": 1, "principalExclusionCriteria": "DMD patients with any mutations affecting: - exons 1 through 17, (and any mutations affecting other exons as per a country\u2019s requirement which will be applicable to this specific country) for Part 1 participants  - affecting exons 8 and/or 9 (and any mutations affecting other exons as per a country\u2019s requirement which will be applicable to this specific country) for Part 2 participants", "principalExclusionCriteriaTranslations": [{"id": 9971092, "uuid": "b3fe58d5-3298-493a-8ec7-2300c95e10cc", "attributeTranslation": "Patient atteint de DMD pr\u00e9sentant des mutations \n- touchant les exons 1 \u00e0 17 (et toutes les mutations touchant d\u2019autres exons conform\u00e9ment aux exigences sp\u00e9cifiques en vigueur dans le pays) pour les participants inclus dans la partie 1\n- touchant les exons 8 et/ou 9 (et toutes les mutations touchant d\u2019autres exons conform\u00e9ment aux exigences sp\u00e9cifiques en vigueur dans le pays) pour les participants inclus dans la partie 2", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971095, "uuid": "b3fe58d5-3298-493a-8ec7-2300c95e10cc", "attributeTranslation": "DMD-pati\u00ebnt met mutaties die van invloed zijn op: \n- exons 1 tot 17 (en eventuele mutaties die van invloed zijn op andere exons in overeenstemming met de specifieke vereiste van het land) voor deelnemers aan Deel 1\n- exons 8 en/of 9 (en eventuele mutaties die van invloed zijn op andere exons in overeenstemming met de specifieke vereiste van het land) voor deelnemers aan Deel 2", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971094, "uuid": "b3fe58d5-3298-493a-8ec7-2300c95e10cc", "attributeTranslation": "Patients atteints de DMD avec des mutations affectant les exons 1 \u00e0 17 et/ou avec des mutations affectant les exons 29 et 30", "language": 34, "languageDescription": "French (France)"}, {"id": 9971093, "uuid": "b3fe58d5-3298-493a-8ec7-2300c95e10cc", "attributeTranslation": "DMD-Patienten mit Mutationen, die \n- die Exons 1 bis 17 betreffen (und alle Mutationen, die andere Exons betreffen, je nach den f\u00fcr dieses spezifische Land geltenden Anforderungen) f\u00fcr Teilnehmer an Teil 1\n- die Exons 8 und/oder 9 betreffen (und alle Mutationen, die andere Exons betreffen, je nach den f\u00fcr dieses spezifische Land geltenden Anforderungen) f\u00fcr Teilnehmer an Teil 2", "language": 28, "languageDescription": "German (Belgium)"}]}, {"id": 1393283, "number": 2, "principalExclusionCriteria": "Presence of neutralizing antibodies against AAV8", "principalExclusionCriteriaTranslations": [{"id": 9971098, "uuid": "79a3b41f-a787-4399-b119-96c49e34137b", "attributeTranslation": "Pr\u00e9sence d\u2019anticorps neutralisants contre l\u2019AAV8", "language": 34, "languageDescription": "French (France)"}, {"id": 9971096, "uuid": "79a3b41f-a787-4399-b119-96c49e34137b", "attributeTranslation": "Aanwezigheid van neutraliserende antilichamen tegen AAV8", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971099, "uuid": "79a3b41f-a787-4399-b119-96c49e34137b", "attributeTranslation": "Pr\u00e9sence d\u2019anticorps neutralisants dirig\u00e9s contre l\u2019AAV8", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971097, "uuid": "79a3b41f-a787-4399-b119-96c49e34137b", "attributeTranslation": "Vorhandensein von neutralisierenden Antik\u00f6rpern gegen AAV8", "language": 28, "languageDescription": "German (Belgium)"}]}, {"id": 1393284, "number": 3, "principalExclusionCriteria": "Cardiomyopathy based on physical/cardiological examination and echocardiography (or cardiac MRI if available) with Left Ventricular Ejection Fraction (LVEF) below 55% and fractional shortening (SF) below 28%", "principalExclusionCriteriaTranslations": [{"id": 9971100, "uuid": "2fc559c2-9a64-47f9-94d1-148e196389ac", "attributeTranslation": "Cardiomyopathie constat\u00e9e \u00e0 l\u2019examen physique/cardiologique et \u00e0 l\u2019\u00e9chocardiographie (ou \u00e0 l\u2019IRM cardiaque si disponible) avec une fraction d\u2019\u00e9jection ventriculaire gauche (FEVG) inf\u00e9rieure \u00e0 55 % et une fraction de raccourcissement (FR) inf\u00e9rieure \u00e0 28 %.", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971102, "uuid": "2fc559c2-9a64-47f9-94d1-148e196389ac", "attributeTranslation": "Cardiomyopathie gebaseerd op lichamelijk/cardiologisch onderzoek en echocardiografie (of een MRI van het hart indien beschikbaar) met een linkerventrikelejectiefractie (LVEF) van minder dan 55% en een fractionele verkorting (SF) van minder dan 28%", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971103, "uuid": "2fc559c2-9a64-47f9-94d1-148e196389ac", "attributeTranslation": "Cardiomyopathie authentifi\u00e9e sur la base des examens clinique et cardiologique et d\u2019une \u00e9chocardiographie avec une fraction d'\u00e9jection ventriculaire gauche (FEVG) inf\u00e9rieure \u00e0 55 % et/ou une diminution de la fraction d\u2019\u00e9jection inf\u00e9rieure \u00e0 28 %", "language": 34, "languageDescription": "French (France)"}, {"id": 9971101, "uuid": "2fc559c2-9a64-47f9-94d1-148e196389ac", "attributeTranslation": "Kardiomyopathie auf der Grundlage einer k\u00f6rperlichen/kardiologischen Untersuchung und einer Echokardiographie (oder eines kardialen MRT, falls verf\u00fcgbar) mit einer linksventrikul\u00e4ren Ejektionsfraktion (LVEF) unter 55 % und einer fraktionellen Verk\u00fcrzung (SF) unter 28 %", "language": 28, "languageDescription": "German (Belgium)"}]}, {"id": 1393285, "number": 4, "principalExclusionCriteria": "Any respiratory assistance needed including non-invasive daytime or nocturnal ventilation", "principalExclusionCriteriaTranslations": [{"id": 9971106, "uuid": "7d229413-99a0-4b7f-a5bf-d00f5646fc8a", "attributeTranslation": "Noodzakelijke mechanische beademing, met inbegrip van niet-invasieve beademing overdag of 's nachts", "language": 36, "languageDescription": "Dutch (Belgium)"}, {"id": 9971104, "uuid": "7d229413-99a0-4b7f-a5bf-d00f5646fc8a", "attributeTranslation": "Toute assistance respiratoire n\u00e9cessaire y compris la ventilation non invasive diurne ou nocturne", "language": 33, "languageDescription": "French (Belgium)"}, {"id": 9971107, "uuid": "7d229413-99a0-4b7f-a5bf-d00f5646fc8a", "attributeTranslation": "Erforderliche Atemhilfe, einschlie\u00dflich nicht-invasiver Beatmung bei Tag oder in der Nacht", "language": 28, "languageDescription": "German (Belgium)"}, {"id": 9971105, "uuid": "7d229413-99a0-4b7f-a5bf-d00f5646fc8a", "attributeTranslation": "Assistance respiratoire n\u00e9cessaire, incluant toute ventilation non invasive diurne ou nocturne", "language": 34, "languageDescription": "French (France)"}]}, {"id": 1393286, "number": 5, "principalExclusionCriteria": "Inability to perform the planned respiratory functions tests", "principalExclusionCriteriaTranslations": [{"id": 9971111, "uuid": 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"applicationStatusCode": "Authorised"}], "decisionDate": "2025-07-25", "ctMSCsByApplication": [{"id": 8352, "mscName": "France", "reportingStatusCode": "Authorised"}], "businessKey": "SM-2", "modScope": "PART_I_AND_PART_II", "allPartTwosOutOfScope": false, "applicationTrialDecisionByMsc": {}, "decisions": [{"id": 82155, "applicationId": 51598, "mscId": 8352, "mscName": "France", "decisionDate": "2025-07-25T17:27:23.516", "decision": "authorized", "assessmentOutcome": "acceptable", "eventType": "decision", "part2Id": 199877, "part1Id": 89213, "applicationType": "SUBSTANTIAL MODIFICATION", "isRMS": true}]}, {"id": 67709, "type": "SUBSTANTIAL MODIFICATION", "status": "Authorised", "ctNumber": "2023-505187-11-00", "trialStatus": "Authorised", "submissionDate": "2025-10-16", "partI": {"assessmentOutcome": "acceptable", "assessmentOutcomeDate": "2026-01-05"}, "partIIInfo": [{"id": 249832, "mscId": 8352, "mscInfo": {"id": 8352, "mscName": "France", "countryOrganisationId": 2012, "assessmentOutcome": "acceptable", "assessmentOutcomeDate": "2025-11-14", "decision": "authorized", "decisionDate": "2023-06-21", "reportingStatusCode": "Authorised", "countryName": "France", "trialStatus": "Authorised", "firstDecisionDate": "2023-06-21"}, "applicationStatusCode": "Authorised"}], "decisionDate": "2026-01-08", "ctMSCsByApplication": [{"id": 8352, "mscName": "France", "reportingStatusCode": "Authorised"}], "businessKey": "SM-3", "modScope": "PART_I_AND_PART_II", "allPartTwosOutOfScope": false, "applicationTrialDecisionByMsc": {}, "decisions": [{"id": 104286, "applicationId": 67709, "mscId": 8352, "mscName": "France", "decisionDate": "2026-01-08T18:39:24.23", "decision": "authorized", "assessmentOutcome": "acceptable", "eventType": "decision", "part2Id": 249832, "part1Id": 108676, "applicationType": "SUBSTANTIAL MODIFICATION", "isRMS": true}]}, {"id": 74136, "type": "NON SUBSTANTIAL MODIFICATION", "status": "Authorised", "ctNumber": "2023-505187-11-00", "trialStatus": "Authorised", "submissionDate": "2026-01-09", "partI": {"assessmentOutcome": "acceptable", "assessmentOutcomeDate": "2026-01-05"}, "partIIInfo": [{"id": 270458, "mscId": 8352, "mscInfo": {"id": 8352, "mscName": "France", "countryOrganisationId": 2012, "decision": "authorized", "decisionDate": "2023-06-21", "reportingStatusCode": "Authorised", "countryName": "France", "trialStatus": "Authorised", "firstDecisionDate": "2023-06-21"}, "applicationStatusCode": "Authorised"}], "decisionDate": "2026-01-09", "ctMSCsByApplication": [{"id": 8352, "mscName": "France", "reportingStatusCode": "Authorised"}], "businessKey": "NSM-7", "modScope": "PART_II", "allPartTwosOutOfScope": false, "applicationTrialDecisionByMsc": {}, "decisions": [{"id": 104415, "applicationId": 74136, "mscId": 8352, "mscName": "France", "decisionDate": "2026-01-09T17:19:56.878", "decision": "authorized", "eventType": "decision", "part2Id": 270458, "part1Id": 113637, "applicationType": "NON SUBSTANTIAL MODIFICATION", "isRMS": true}]}, {"id": 74166, "type": "NON SUBSTANTIAL MODIFICATION", "status": "Authorised", "ctNumber": "2023-505187-11-00", "trialStatus": "Authorised", "submissionDate": "2026-01-09", "partI": {"assessmentOutcome": "acceptable", "assessmentOutcomeDate": "2026-01-05"}, "partIIInfo": [], "decisionDate": "2026-01-09", "ctMSCsByApplication": [{"id": 8352, "mscName": "France", "reportingStatusCode": "Authorised"}], "businessKey": "NSM-8", "modScope": "PART_I", "allPartTwosOutOfScope": false, "applicationTrialDecisionByMsc": {}, "decisions": [{"id": 104426, "applicationId": 74166, "mscId": 8352, "mscName": "France", "decisionDate": "2026-01-09T18:27:27.963", "decision": "authorized", "eventType": "decision", "part1Id": 113673, "applicationType": "NON SUBSTANTIAL MODIFICATION", "isRMS": true}]}, {"id": 74185, "type": "SUBSEQUENT ADDITION OF MSC", "status": "Authorised", "ctNumber": "2023-505187-11-00", "trialStatus": "Authorised", "submissionDate": "2026-01-15", "partI": {}, "partIIInfo": [{"id": 285183, "mscId": 56574, "mscInfo": {"id": 56574, "mscName": "Belgium", "countryOrganisationId": 2002, "assessmentOutcome": "acceptable", "assessmentOutcomeDate": "2026-03-16", "decision": "authorized", "decisionDate": "2026-03-17", "reportingStatusCode": "Authorised", "countryName": "Belgium", "trialStatus": "Authorised", "firstDecisionDate": "2026-03-17"}, "applicationStatusCode": "Authorised"}], "decisionDate": "2026-03-17", "ctMSCsByApplication": [{"id": 56574, "mscName": "Belgium", "reportingStatusCode": "Authorised"}], "businessKey": "AM-1", "allPartTwosOutOfScope": false, "applicationTrialDecisionByMsc": {}, "decisions": [{"id": 114732, "applicationId": 74185, "mscId": 56574, "mscName": "Belgium", "decisionDate": "2026-03-17T12:02:29.973", "decision": "authorized", "assessmentOutcome": "acceptable", "eventType": "decision", "part2Id": 285183, "part1Id": 120951, "applicationType": "SUBSEQUENT ADDITION OF MSC", "isRMS": false}]}], "trialGlobalEnd": [], "memberStatesConcerned": [{"mscName": "France", "mscId": 8352, "firstDecisionDate": "2023-06-21T09:09:56.263", "lastDecisionDate": "2026-01-09T17:19:56.878", "mscPublicStatusCode": 4}, {"mscName": "Belgium", "mscId": 56574, "firstDecisionDate": "2026-03-17T12:02:29.973", "lastDecisionDate": "2026-03-17T12:02:29.973", "mscPublicStatusCode": 2}], "eudraCt": {"isTransitioned": true, "eudraCtCode": "2020-002093-27"}}, "events": {"temporaryHaltList": [], "trialEvents": [{"mscId": 8352, "mscName": "France", "events": [{"notificationType": "START_OF_TRIAL", "date": "2021-03-01"}, {"notificationType": "START_OF_RECRUITMENT", "date": "2021-03-02"}]}, {"mscId": 56574, "mscName": "Belgium", "events": []}, {"mscId": 56575, "mscName": "Spain", "events": []}], "unexpectedEvents": [{"sponsorBusinessKey": "GENETHON", "businessKey": "UE-98653", "awareDate": "2025-09-05", "eventDate": "2025-09-05", "submissionDate": "2025-09-23", "updatedOn": "2025-09-23", "clinicalProcedures": "An infusion", "eventDescription": "An infusion related allergic reaction has been reported. The event was assessed as serious. No sign of severe anaphylaxis.", "measuresTakenDescription": "\u2022\tAdd infusion-related reaction (hypersensitivity) in the list of potential risks with GNT0004 Close monitoring of patients, with maintenance of venous access after infu-sion, to manage any potential delayed allergic reaction for at least 3 hours after the end of infusion (duration could be prolonged per investigator\u2019s judgment). \n\u2022\tIn the event of relevant signs/symptoms, such as tachycardia, tachypnea, lip swelling, difficulty breathing, nasal flaring, urticaria, flushing, lip pruritus, rash, cheilitis, vomiting, nausea, rigors, pyrexia, etc.): \nthe infusion should be stopped immediately and appropriate infusion-related reaction management such as supportive care, antihistamines, and/or other relevant anti-allergic medication (eg additional dose of IV corticosteroid) should be considered \n\u2022\tAllergy testing and analysis should be considered as early as possible when the event occurs with the support of an anaphylaxis specialist if deemed necessary. Infusion may be resumed at a reduced rate once symptoms resolve, per PI\u2019s judgment. Infusion should be discontinued in case of severe Anaphylaxis.\nThis urgent safety measure (USM) has been agreed upon by an iDMC convened in emergency. And the iDMC recommended to continue the study with this USM in place, provided the benefice/risk ratio remains positive. The communication with investigators / centers and information to the study participants has been implemented on 19-Sep-2025. The amendment of study core documents (study protocol, IB, ICFs, etc.) will be submitted in due course. Following the recommendation of the iDMC, the study may proceed following implementation of the Urgent Safety Measures (USM).", "mscs": ["France"], "countries": [{"eutctId": 100000000395, "name": "France", "isoNumber": 250, "isoAlpha2Code": "FR", "isoAlpha3Code": "FRA", "current": true}], "products": ["rAAV8-hMD1"], "substances": [], "devices": []}], "seriousBreaches": [], "urgentSafetyMeasures": [{"sponsorBusinessKey": "GENETHON", "businessKey": "US-98654", "eventDate": "2025-09-15", "submissionDate": "2025-09-23", "updatedOn": "2025-09-23", "inResponseTo": "SUSAR", "susarIdList": ["UE-98653"], "eventDescription": "An infusion related allergic reaction has been reported. The event was assessed as serious. No sign of severe anaphylaxis.", "measuresTakenDescription": "\u2022\tAdd infusion-related reaction (hypersensitivity) in the list of potential risks with GNT0004 Close monitoring of patients, with maintenance of venous access after infu-sion, to manage any potential delayed allergic reaction for at least 3 hours after the end of infusion (duration could be prolonged per investigator\u2019s judgment). \n\u2022\tIn the event of relevant signs/symptoms, such as tachycardia, tachypnea, lip swelling, difficulty breathing, nasal flaring, urticaria, flushing, lip pruritus, rash, cheilitis, vomiting, nausea, rigors, pyrexia, etc.): \nthe infusion should be stopped immediately and appropriate infusion-related reaction management such as supportive care, antihistamines, and/or other relevant anti-allergic medication (eg additional dose of IV corticosteroid) should be considered \n\u2022\tAllergy testing and analysis should be considered as early as possible when the event occurs with the support of an anaphylaxis specialist if deemed necessary. Infusion may be resumed at a reduced rate once symptoms resolve, per PI\u2019s judgment. Infusion should be discontinued in case of severe Anaphylaxis.\nThis urgent safety measure (USM) has been agreed upon by an iDMC convened in emergency. And the iDMC recommended to continue the study with this USM in place, provided the benefice/risk ratio remains positive. The communication with investigators / centers and information to the study participants has been implemented on 19-Sep-2025. The amendment of study core documents (study protocol, IB, ICFs, etc.) will be submitted in due course. Following the recommendation of the iDMC, the study may proceed following implementation of the Urgent Safety Measures (USM).", "mscs": ["France"], "unexpectedEvents": []}]}, "results": {}, "documents": [], "trialRegion": "Both", "trialRegionCode": 3, "correctiveMeasures": []}},{"_id":29,"ctNumber":"2022-500703-49-01","ctStatusCode":4,"ctStatus":"Authorised","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","conditions":"Duchenne muscular dystrophy","trialCountries":["France:4"],"decisionDateOverall":"21/11/2022","decisionDateByCountry":"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","trialRegionCode":1,"trialRegion":"EEA","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","decisionDate":"2022-11-21T09:35:18.445","publishDate":"2026-02-28T02:36:45.762945083","ctPublicStatusCode":4,"documents":[],"memberStatesConcerned":[{"mscName": "France", "mscId": 1902, "firstDecisionDate": "2022-11-21T09:35:18.445", "lastDecisionDate": "2026-02-27T15:06:30.845", "mscPublicStatusCode": 4}],"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\u00b0C &#43;/- 5\u00b0C / T0 &#43; T3M &#43; T6M &#43; T9M &#43; T12M &#43; T18M &#43; T24M \n         o Accelerated conditions / Storage in stability chamber at &#43;5\u00b0C &#43;/- 3\u00b0C / 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\u00b0C &#43;/- 2\u00b0C / T0 &#43; T1M \n\n-\tStability results known to date: stress test &#43;40\u00b0C &#43;/- 2\u00b0C up to T1M, accelerated conditions at &#43;5\u00b0C &#43;/- 3\u00b0C up to T12M, and normal conditions at -20\u00b0C &#43;/- 5\u00b0C 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\u00b0C (in accordance with ICH Q1E \u2013 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 \u2013 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 \u2013 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 Comit\u00e9 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":{},"rawSearchRecord":{"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/\u00e9, 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) \u2013 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"},"rawRetrieveRecord":{"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": "\u00c9tude de phase 1/2a, monocentrique, ouverte, visant \u00e0 \u00e9valuer la s\u00e9curit\u00e9, la pharmacocin\u00e9tique et la pharmacodynamique du compos\u00e9 SQY51 chez des patients p\u00e9diatriques et adultes ayant re\u00e7u un diagnostic g\u00e9n\u00e9tiquement confirm\u00e9 de dystrophie musculaire de Duchenne, comprenant :\n i) une phase 1 de 13 semaines \u00e0 doses multiples croissantes, ii) une phase 2a de 49 semaines, iii) une phase d'extension de 102 semaines, ou iv) un suivi \u00e0 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": "\u00c9tude de phase 1/2a visant \u00e0 \u00e9valuer la s\u00e9curit\u00e9, la pharmacocin\u00e9tique et la pharmacodynamique du compos\u00e9 SQY51 chez des patients p\u00e9diatriques et adultes ayant re\u00e7u un diagnostic g\u00e9n\u00e9tiquement confirm\u00e9 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 \u22656 years of age and \u226516 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). Early non-ambulatory stage: Unable to walk 10 m without assistance, including human assistance. Loss of ambulation (LoA) \u2264 5 years preceding enrolment (inclusion of maximal 8 patients). Late non-ambulatory stage: LoA > 5 years preceding enrolment (inclusion of maximal 3 patients).", "principalInclusionCriteriaTranslations": []}, {"id": 778543, "number": 6, "principalInclusionCriteria": "Patients and, if minor, their legal guardians who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.", "principalInclusionCriteriaTranslations": []}, {"id": 778544, "number": 7, "principalInclusionCriteria": "Diagnosed with DMD, genotypically confirmed with DMD mutations amenable to exon-51 skipping.", "principalInclusionCriteriaTranslations": []}, {"id": 778545, "number": 8, "principalInclusionCriteria": "Stable hepatic and renal function: \u2022 GGT <1.5\u00d7ULN, AST <25\u00d7ULN, ALT <15\u00d7ULN \u2022 Total bilirubin <1.5\u00d7ULN. \u2022 Alkaline phosphatase \u22641.5\u00d7ULN. \u2022 Estimated glomerular filtration rate \u226590 mL/min/1.73m2. \u2022 Proteinuria and microalbuminuria \u2264ULN.", "principalInclusionCriteriaTranslations": []}, {"id": 778546, "number": 9, "principalInclusionCriteria": "Echocardiography left ventricular ejection fraction (LVEF) at screening \u226540%.", "principalInclusionCriteriaTranslations": []}], "principalExclusionCriteria": [{"id": 1334461, "number": 1, "principalExclusionCriteria": "Patient with any serious medical/surgical or psychiatric condition/illness/history that in the opinion of the investigator would jeopardize patient\u2019s safety or would interfere with the study assessments/results, including insufficient obligatory vaccination against infectious diseases as recommended by national guidelines, medical history of infection with Hepatitis B,C and HIV", "principalExclusionCriteriaTranslations": []}, {"id": 1334462, "number": 10, "principalExclusionCriteria": "Abnormal laboratory values in the clinically significant range", "principalExclusionCriteriaTranslations": []}, {"id": 1334463, "number": 2, "principalExclusionCriteria": "Patient with a history of significant allergic reactions and with any known allergies to products likely to be used in the study (e.g., antiseptics, anaesthetics), known hypersensitivity to any of the ingredients, or excipients of the study drug.", "principalExclusionCriteriaTranslations": []}, {"id": 1334464, "number": 3, "principalExclusionCriteria": "Patient who participated in other investigational study within the last three months, including those with investigational drugs that aim at restoring dystrophin expression such as other antisense oligomers.", "principalExclusionCriteriaTranslations": []}, {"id": 1334465, "number": 4, "principalExclusionCriteria": "Patient that received gene therapy.", "principalExclusionCriteriaTranslations": []}, {"id": 1334466, "number": 5, "principalExclusionCriteria": "Patient with intellectual disability or behavioral problem such that they cannot comply with the study procedure.", "principalExclusionCriteriaTranslations": []}, {"id": 1334467, "number": 6, "principalExclusionCriteria": "Patient with advanced cardiomyopathy and LVEF <40%. Patients with dysrhythmias and being treated for dysrhythmias. Patients with nontreated tachycardia.", "principalExclusionCriteriaTranslations": []}, {"id": 1334468, "number": 7, "principalExclusionCriteria": "Patient for whom major orthopedic surgery is planned during the phase 1 of the study (little invasive procedures such as tenotomy are permissible during phase 2, any procedure is permissible during the extension phase).", "principalExclusionCriteriaTranslations": []}, {"id": 1334469, "number": 8, "principalExclusionCriteria": "Tracheostomized patients and dependent on invasive mechanical ventilation. Non-invasive mechanical ventilation \u2265 16 h/day. Medical history with more than two respiratory decompensations requiring hospitalization during the previous year. No respiratory decompensation in the four months preceding enrolment.", "principalExclusionCriteriaTranslations": []}, {"id": 1334470, "number": 9, "principalExclusionCriteria": "Patients on medications that can restore dystrophin expression, tamoxifen and other drugs without indication for DMD or pediatric population.", "principalExclusionCriteriaTranslations": []}]}, "endPoint": {"primaryEndPoints": [{"id": 912099, "number": 1, "endPoint": "Type, frequency, severity, timing and relationship to SQY51 of: Adverse events (AEs), discontinuations due to AEs, serious AEs (SAEs).", "isPrimary": true, "endPointTranslations": []}, {"id": 912100, "number": 2, "endPoint": "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.", "isPrimary": true, "endPointTranslations": []}], "secondaryEndPoints": [{"id": 912101, "number": 1, "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.", "isPrimary": false, "endPointTranslations": []}, {"id": 912102, "number": 2, "endPoint": "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", "isPrimary": false, "endPointTranslations": []}, {"id": 912103, "number": 3, "endPoint": "Phase 2 : Cardiac function : - echocardiography (LVEDD, LVESD, LVSWT, LVPWT, GLS, LVEFMS, LVMI, mitral inflow velocities, DT, E/\u00e9, 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).", "isPrimary": false, "endPointTranslations": []}, {"id": 912104, "number": 4, "endPoint": "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.", "isPrimary": false, "endPointTranslations": []}, {"id": 912105, "number": 5, "endPoint": "Phase 2 : Quality of Life Endpoints/Self-Assessment: Pediatric Quality of Life Inventory (PedsQL) \u2013 DMD module", "isPrimary": false, "endPointTranslations": []}, {"id": 912106, "number": 6, "endPoint": "Phase 2 : Serum biomarker analysis: Muscle necrosis biomarkers (CPK and Myomesine-3).", "isPrimary": false, "endPointTranslations": []}, {"id": 912107, "number": 7, "endPoint": "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).", "isPrimary": false, "endPointTranslations": []}, {"id": 912108, "number": 8, "endPoint": "Phase 1: Levels of SQY51, its main metabolites in serum and urine, and AUC calculation.", "isPrimary": false, "endPointTranslations": []}, {"id": 912109, "number": 9, "endPoint": "Phase 2: Levels of SQY51 and main metabolites in serum, urine and muscle biopsy sample", "isPrimary": false, "endPointTranslations": []}, {"id": 912110, "number": 10, "endPoint": "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.", "isPrimary": false, "endPointTranslations": []}]}, "trialDuration": {"estimatedEndDate": "2028-12-31", "estimatedRecruitmentStartDate": "2022-11-01"}, "sourceOfMonetarySupport": [{"id": 84500, "organisationName": "SQY 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"businessKey": "3", "title": "Part III", "description": "At the end of the phase 2a, patients will either enter an extension phase and continue to be dosed with SQY51, or being long-term followed at regular intervals", "blindingMethodCode": "3", "blindedRoles": [], "armDetails": [], "allocationMethod": "3"}]}}, "scientificAdviceAndPip": {"scientificAdvices": [{"id": 48892, "competentAuthority": {"id": 17999, "organisation": {"id": 18894, "type": "EU Institution/Body/Agency", "typeCode": "14", "name": "European Medicines Agency", "commercial": false, "isBusinessKeyValidated": true, "businessKey": "ORG-100013412", "organisationLocationStatus": "Active"}, "address": {"addressId": 18248, "oneLine": "Domenico Scarlattilaan 6", "addressLine1": "Domenico Scarlattilaan 6", "addressLine2": "", "addressLine3": "", "addressLine4": "", "city": "Amsterdam", "postcode": "1083 HS", "country": 2029, "countryName": "Netherlands"}, "isBusinessKeyValidated": true, "businessKey": "LOC-100020264"}}], "paediatricInvestigationPlan": []}, "associatedClinicalTrials": [{"id": 29889, "ctNumber": "2022-500703-49-00", "sponsorName": "Sqy Therapeutics", "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, and a ii) 32-week Phase 2a", "sponsorAgreementOption": "NON_APPLICABLE", "sponsorAgreementOptionName": "NON_APPLICABLE", "parentClinicalTrialId": 717, "hasDocument": false, "associatedCtDocs": []}], "references": [], "pubmedCode": [], "pubmedUrl": []}, "assessmentOutcome": "acceptable", "therapeuticAreas": [{"code": "5", "name": "Diseases [C] - Musculoskeletal Diseases [C05]"}], "medicalConditions": [{"id": 120476, "medicalCondition": "Duchenne muscular dystrophy", "isConditionRareDisease": true}], "sponsors": [{"id": 131090, "primary": true, 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"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\u00b0C &#43;/- 5\u00b0C / T0 &#43; T3M &#43; T6M &#43; T9M &#43; T12M &#43; T18M &#43; T24M \n         o Accelerated conditions / Storage in stability chamber at &#43;5\u00b0C &#43;/- 3\u00b0C / 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\u00b0C &#43;/- 2\u00b0C / T0 &#43; T1M \n\n-\tStability results known to date: stress test &#43;40\u00b0C &#43;/- 2\u00b0C up to T1M, accelerated conditions at &#43;5\u00b0C &#43;/- 3\u00b0C up to T12M, and normal conditions at -20\u00b0C &#43;/- 5\u00b0C 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\u00b0C (in accordance with ICH Q1E \u2013 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 \u2013 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 \u2013 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 Comit\u00e9 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": []}}], "fields": [{"id": "_id", "type": "int"}, {"id": "ctNumber", "type": "text", "info": {"label": "CT Number"}}, {"id": "ctStatusCode", "type": "int4"}, {"id": "ctStatus", "type": "text"}, {"id": "ctTitle", "type": "text"}, {"id": "shortTitle", "type": "text"}, {"id": "conditions", "type": "text"}, {"id": "trialCountries", "type": "json"}, {"id": "decisionDateOverall", "type": "text"}, {"id": "decisionDateByCountry", "type": "text"}, {"id": "therapeuticAreas", "type": "json"}, {"id": "sponsor", "type": "text"}, {"id": "sponsorType", "type": "text"}, {"id": "trialPhase", "type": "text"}, {"id": "endPoint", "type": "text"}, {"id": "product", "type": "text"}, {"id": "ageRangeSecondary", "type": "json"}, {"id": "ageGroup", "type": "text"}, {"id": "gender", "type": "text"}, {"id": "trialRegionCode", "type": "int4"}, {"id": "trialRegion", "type": "text"}, {"id": "totalNumberEnrolled", "type": "text"}, {"id": "primaryEndPoint", "type": "text"}, {"id": "resultsFirstReceived", "type": "text"}, {"id": "lastUpdated", "type": "text"}, {"id": "lastPublicationUpdate", "type": "text"}, {"id": "decisionDate", "type": "text"}, {"id": "publishDate", "type": "text"}, {"id": "ctPublicStatusCode", "type": "int4"}, {"id": "documents", "type": "json"}, {"id": "memberStatesConcerned", "type": "json"}, {"id": "events", "type": "json"}, {"id": "results", "type": "json"}, {"id": "rawSearchRecord", "type": "json"}, {"id": "rawRetrieveRecord", "type": "json"}], "_links": {"start": "/api/3/action/datastore_search?resource_id=468133e6-3935-4b69-bea1-34943422242e", "next": "/api/3/action/datastore_search?resource_id=468133e6-3935-4b69-bea1-34943422242e&offset=100"}, "total": 29, "total_was_estimated": false}}