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)

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Study title: 
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)
Long title: 
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)
EU record number: 
B/BE/21/BVW5
EudraCT number: 
2019-003374-91
Company / Sponsor: 
Sarepta Therapeutics
Phase: 
III
Treated organism: 
Humans
Indication category: 
Neuromuscular disorders
Disease: 
Treatment of Duchenne Muscular Dystrophy
Therapeutic approach: 
Gene therapy
Genetic modification: 
Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the human micro-dystrophin (hMicro-Dys) gene
Method of transfer of nucleic acid of interest: 
Non-replicating recombinant adeno-associated virus serotype rh74
Administered biological material: 
Genetically modified virus
Route of administration: 
Intravenous
Locations in Belgium: 
UZ Leuven, UZ Gent
Nr of subjects: 
Up to 120 patients overall and 6 patients in Belgium
Foreseen duration: 
In Belgium 28 JAN 2022 – 30 NOV 2024
Type of procedure: 
Contained use and Deliberate release
Current status: 
Authorized

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