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

Sarepta Therapeutics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Muscular Dystrophy, Duchenne

Treatments: Genetic: delandistrogene moxeparvovec; Genetic: placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03769116

SRP-9001-102

2021-000078-27 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants 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). Participants who are randomized to placebo in Part 1 will have the opportunity for treatment with delandistrogene moxeparvovec in Part 2.

In order to provide a uniform approach to monitoring long-term safety and efficacy in participants who received SRP-9001 in a clinical trial, the Sponsor has amended Study Completion for this study to occur at Week 130. Therefore, participants have transitioned and will complete the remainder of the Part 3 follow up visits in a long-term extension study, SRP-9001-305 (NCT05967351).

Enrollment: 41 patients

Sex: Male

Ages: 4 to 7 years old

Volunteers: No Healthy Volunteers

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.
• A frameshift mutation contained between exons 18 and 58 (inclusive).

Exclusion criteria

• Impaired cardiovascular function on echocardiogram.
• Prior or ongoing medical condition on physical examination, electrocardiogram, or laboratory findings that could adversely affect participant safety, compromise completion of follow-up, or impair assessment of study results.
• Exposure to another investigational drug or exon skipping medication within 6 months of screening.
• Exposure to an investigational or commercial gene therapy product.
• Abnormal liver or renal function by protocol-specified criteria.

Other inclusion/exclusion criteria apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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41 participants in 2 patient groups

Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2

Experimental group

Description:

Participant will receive delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3.

Treatment:

Genetic: placebo

Genetic: delandistrogene moxeparvovec

Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2

Experimental group

Description:

Participant will receive matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3.

Treatment:

Genetic: placebo

Genetic: delandistrogene moxeparvovec