Study title:
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Date receipt dossier:
18 sep 2020
EU record number:
B/BE/20/BVW4
EudraCT number:
2019-002921-31
Pharmaceutical study code:
C3391003
Company / Sponsor:
Pfizer
Phase:
III
Treated organism:
Humans
Indication category:
Neuromuscular disorders
Disease:
Treatment of Duchenne Muscular Dystrophy
Therapeutic approach:
Gene therapy
Genetic modification:
Non-replicating recombinant vector derived from adeno-associated virus AAV9, lacking all AAV viral genes and containing a miniaturized version of the human dystrophin gene
Method of transfer of nucleic acid of interest:
Non-replicating recombinant adeno-associated virus serotype 9 (AAV9)
Administered biological material:
Genetically modified virus
Route of administration:
Intravenous
Locations in Belgium:
UZ Gent, UZ Leuven, CHR Citadelle Liège
Nr of subjects:
Approximately 99 patients overall and 3 patients in Belgium are foreseen.
Foreseen duration:
Q3 2020 to end of treatment period ~Q4 2021
Type of procedure:
Contained use and Deliberate release
Current status:
Authorized
Information for the public
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Public information presented according to the provisions of Directive 2001/18/EC
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Information related to the decision procedure
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5 February 2021 - The Biosafety Advisory Council issues a positive advice (with conditions) for this deliberate release
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5 March 2021: The Federal Ministers give a final decision (positive) for this trial