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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-12-05

Study Completion Date

2023-08-16

Brief Summary

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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).

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Detailed Description

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Conditions

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Muscular Dystrophy, Duchenne

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Parallel up to the measurement of the primary outcome at Week 48. At the beginning of Part 2, participants who were originally assigned to placebo will have the opportunity to receive delandistrogene moxeparvovec. All participants will be followed for 5 years following treatment with delandistrogene moxeparvovec.

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2

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

Group Type EXPERIMENTAL

delandistrogene moxeparvovec

Intervention Type GENETIC

Single IV infusion of delandistrogene moxeparvovec

placebo

Intervention Type GENETIC

Single IV infusion of matching placebo

Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2

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

Group Type EXPERIMENTAL

delandistrogene moxeparvovec

Intervention Type GENETIC

Single IV infusion of delandistrogene moxeparvovec

placebo

Intervention Type GENETIC

Single IV infusion of matching placebo

Interventions

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delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec

Intervention Type GENETIC

placebo

Single IV infusion of matching placebo

Intervention Type GENETIC

Other Intervention Names

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SRP-9001 delandistrogene moxeparvovec-rokl ELEVIDYS

Eligibility Criteria

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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.

Minimum Eligible Age

4 Years

Maximum Eligible Age

7 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No