Gene Therapy for Duchenne Muscular Dystrophy
(ENDEAVOR Trial)

Recruiting in Palo Alto (17 mi)

+4 other locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Sarepta Therapeutics, Inc.

No Placebo Group

Trial Summary

What is the purpose of this trial?

This trial is testing a new gene therapy called delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy (DMD). The therapy aims to insert a healthy gene into the body to help improve muscle function. The study will last several years to evaluate safety and effectiveness.

Eligibility Criteria

This trial is for boys with Duchenne Muscular Dystrophy (DMD). Different age groups can join: 4-8, ≥8 to <18 years old, and some who don't need steroids yet. They must be able to do motor tests and not have taken gene therapy or certain drugs recently. Those on stable steroid doses can also participate.

Inclusion Criteria

I have been officially diagnosed with Duchenne Muscular Dystrophy.

My genetic test results match the study's specific requirements.

I have been on a stable dose of oral corticosteroids for at least 12 weeks.

See 10 more

Exclusion Criteria

Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer

You have abnormal results in specific medical tests.

I have not had gene therapy or experimental treatments to boost dystrophin recently.

Treatment Details

Interventions

SRP-9001 (Gene Therapy)

Trial OverviewThe study is testing delandistrogene moxeparvovec, a gene transfer therapy for DMD. It's an open-label study where everyone gets the treatment to see if it's safe and how well it works over about three years.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Delandistrogene MoxeparvovecExperimental Treatment1 Intervention

Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.