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: 24
• Participant gender: Male

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).

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sarepta Therapeutics

Criteria

Inclusion Criteria:

• Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype.

• Indication of symptomatic muscular dystrophy by protocol-specified criteria.

• Ability to cooperate with motor assessment testing.

• Stable dose equivalent of oral corticosteroids for at least 12 weeks.

Exclusion Criteria:

• Impaired cardiovascular function on ECHO.

• Prior or ongoing medical condition on physical examination, ECG, or laboratory findings that could adversely affect subject safety, compromise completion of follow-up, or impair assessment of study results.

• Exposure to another investigational drug or exon skipping medication within months.

• Exposure to an investigational or commercial gene therapy product.

• Abnormal liver or renal function by protocol-specified criteria

Other inclusion/exclusion criteria apply.