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

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

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