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)

Study title: 
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)
Long title: 
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)
Date receipt dossier: 
24 mrt 2023
EU record number: 
B/BE/22/BVW6
EudraCT number: 
CTA 2020-002372-13
Company / Sponsor: 
Sarepta Therapeutics
Phase: 
III
Treated organism: 
Humans
Indication category: 
Neuromuscular disorders
Disease: 
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 vector derived from adeno-associated virus serotype rh74
Administered biological material: 
Genetically modified virus
Route of administration: 
Intravenous
Locations in Belgium: 
UZ Gent Neuromuscular reference center (NMRC)
Nr of subjects: 
Up to 116 patients overall and 10 patients in Belgium
Foreseen duration: 
from Q2 2023 till Q2 2026
Type of procedure: 
Contained use and Deliberate release
Current status: 
Under evaluation

Information for the public

Information related to the decision procedure