A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)

Sarepta Therapeutics

Status and phase: Completed; Phase 3

Conditions: Duchenne Muscular Dystrophy

Treatments: Genetic: placebo; Genetic: delandistrogene moxeparvovec

Study type: Interventional

Funder types: Industry

Identifiers

NCT05096221

2019-003374-91 (EudraCT Number)

SRP-9001-301

Details and patient eligibility

About

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

Enrollment: 126 patients

Sex: Male

Ages: 4 to 7 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Is ambulatory and from 4 to under 8 years of age at time of randomization.
• Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
• Ability to cooperate with motor assessment testing.
• Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
• rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
• A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.

Exclusion criteria

• Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
• Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
• Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.

Other inclusion or exclusion criteria could apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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126 participants in 2 patient groups, including a placebo group

Delandistrogene Moxeparvovec followed by Placebo

Experimental group

Description:

Participants will receive single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at Year 2.

Treatment:

Genetic: placebo

Genetic: delandistrogene moxeparvovec

Placebo followed by Delandistrogene Moxeparvovec

Placebo Comparator group

Description:

Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at Year 2.

Treatment:

Genetic: placebo

Genetic: delandistrogene moxeparvovec