B/BE/22/BVW6 - Information sheet for the public (prepared by the notifier)Public information according to the provisions of Directive 2001/18/EC
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
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
Authorized