A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchennne Muscular Dystrophy (ENVOL)

Study title: 
A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchennne Muscular Dystrophy (ENVOL)
Long title: 
A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchennne Muscular Dystrophy
Date receipt dossier: 
21 Oct 2022
EU record number: 
B/BE/22/BVW5
EudraCT number: 
2022-000691-19
Company / Sponsor: 
F. Hoffmann-La Roche
Phase: 
II
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 adeno-associated virus serotype rh74
Administered biological material: 
Genetically modified virus
Route of administration: 
Intravenous
Locations in Belgium: 
CRMN-Liege CHR Citadelle
Nr of subjects: 
Up to 21 patients overall and 4 patients in Belgium
Foreseen duration: 
In Belgium Q4 2022 - Q4 2027 with safety follow-up (no IMP administered) to Q4 2032
Type of procedure: 
Contained use and Deliberate release
Current status: 
Assessed

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