B/BE/21/BVW5 - Summary Notification Information Format (SNIF)Public information presented according to the provisions of Directive 2001/18/EC
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