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 okt 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
Information for the public
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Public information according to the provisions of Directive 2001/18/EC
Information related to the decision procedure
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17 July 2023 - The Biosafety Advisory Council issues a positive advice (with conditions) for this deliberate release
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23 August 2023 - The Federal Ministers give a final decision (positive with conditions) for this trial