Gene Transfer Therapy for Duchenne Muscular Dystrophy
(EMBARK Trial)
Recruiting at 43 trial locations
MI
ST
Overseen BySarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
Age: < 18
Sex: Male
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Waitlist Available
Sponsor: Sarepta Therapeutics, Inc.
Pivotal Trial (Near Approval)
Prior Safety Data
Trial Summary
What is the purpose of this trial?
This trial tests a gene therapy that adds healthy genes to help boys with Duchenne Muscular Dystrophy (DMD) improve their muscle function. Gene therapy has been explored as a potential treatment for Duchenne Muscular Dystrophy (DMD) through various studies and trials.
Research Team
MD
Medical Director
Principal Investigator
Sarepta Therapeutics, Inc.
Eligibility Criteria
This trial is for boys aged 4 to under 8 with Duchenne Muscular Dystrophy (DMD), confirmed by genetic testing. They must be able to do motor tests, on a stable dose of corticosteroids, and not have high rAAVrh74 antibody levels or previous gene therapy.Inclusion Criteria
I have been officially diagnosed with Duchenne Muscular Dystrophy.
I can participate in tests that measure my muscle movements.
I am between 4 and 7 years old and can walk on my own.
See 9 more
Exclusion Criteria
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.
Your medical tests show something abnormal according to the study's rules.
I have not used any experimental drugs or treatments to increase dystrophin recently.
Treatment Details
Interventions
- Placebo (Genetic)
- SRP-9001 (Delandistrogene Moxeparvovec) (Gene Therapy)
Trial OverviewThe study tests the safety and effectiveness of delandistrogene moxeparvovec (SRP-9001), a gene transfer therapy against a placebo in boys with DMD. It's randomized and double-blind; those initially receiving placebo may get the therapy after one year.
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Delandistrogene Moxeparvovec followed by PlaceboExperimental Treatment2 Interventions
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.
Group II: Placebo followed by Delandistrogene MoxeparvovecPlacebo Group2 Interventions
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.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sarepta Therapeutics, Inc.
Lead Sponsor
Trials
54
Recruited
34,000+
Hoffmann-La Roche
Industry Sponsor
Trials
2,482
Recruited
1,107,000+
Headquarters
Basel, Switzerland
Known For
Precision medicine
Top Products
Avastin, Herceptin, Rituxan, Accu-Chek
Dr. Levi Garraway
Hoffmann-La Roche
Chief Medical Officer since 2019
MD from the University of Basel
Dr. Thomas Schinecker
Hoffmann-La Roche
Chief Executive Officer since 2023
PhD in Molecular Biology from New York University