113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details


臨床試験数 : 646 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170

  
4 trials found
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PhaseCountries
1EUCTR2019-003374-91-BE
(EUCTR)
30/06/202215/10/2021Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular DystrophyA 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) Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: SRP-9001
INN or Proposed INN: Not available
Other descriptive name: ADENO-ASSOCIATED VIRUS SEROTYPE RH74 CONTAINING THE HUMAN MICRO-DYSTROPHIN GENE
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 3France;United States;Taiwan;Hong Kong;Spain;Belgium;Germany;United Kingdom;Italy;Japan
2EUCTR2019-003374-91-ES
(EUCTR)
21/02/202220/10/2021Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular DystrophyA 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) Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: SRP-9001
INN or Proposed INN: Not available
Other descriptive name: ADENO-ASSOCIATED VIRUS SEROTYPE RH74 CONTAINING THE HUMAN MICRO-DYSTROPHIN GENE
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 3France;United States;Taiwan;Hong Kong;Belgium;Spain;Germany;United Kingdom;Italy;Japan
3NCT05096221
(ClinicalTrials.gov)
October 27, 202114/10/2021A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD)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)Duchenne Muscular DystrophyGenetic: SRP-9001;Genetic: PlaceboSarepta Therapeutics, Inc.Hoffmann-La RocheActive, not recruiting4 Years7 YearsMale126Phase 3United States;Belgium;Germany;Hong Kong;Italy;Japan;Spain;Taiwan;United Kingdom
4NCT04626674
(ClinicalTrials.gov)
November 23, 20206/11/2020A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD)An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)Muscular Dystrophy, DuchenneGenetic: SRP-9001Sarepta Therapeutics, Inc.Hoffmann-La RocheEnrolling by invitation3 YearsN/AMale46Phase 1United States