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active, not recruiting

A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)

key information

study id #: NCT03769116

condition: Muscular Dystrophy, Duchenne

status: active, not recruiting


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 three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Patients who are randomized to placebo in Part 1 will have the opportunity for treatment with SRP-9001 in Part 2.

intervention: SRP-9001, Placebo

mechanism of action: Gene therapy to introduce a version of dystrophin

results: https://clinicaltrials.gov/ct2/show/results/NCT03769116

last updated: October 06, 2020