B/BE/22/BVW5 - Information sheet for the public (prepared by the notifier)Public information according to the provisions of Directive 2001/18/EC
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
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
Authorized