A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)
Status:
Not yet recruiting
Trial end date:
2021-12-31
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer
in DMD patients by measuring biological and clinical endpoints in two parts: a 48-week
randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded
extension period (Part 2).