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

NCTID	NCT05881408 (View at clinicaltrials.gov)
Description	The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2. (Show More)
Development Status	Approved
Indication	Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term	DOID:11723
Compound Name	ELEVIDYS
Compound Alias	delandistrogene moxeparvovec, SRP-9001
Compound Description	rAAVrh74.MCK.Micro-Dystrophin
Sponsor	Sarepta Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	148 (ESTIMATED)
Results Posted	Not Available

Therapy Information

Target Gene/Variant	Micro-dystrophin
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Cardiac and skeletal muscle
Delivery System	Viral transduction
Vector Type	AAVrh74
Editor Type	none
Dose 1	For patients < 70kg: 1.33E14 vg/kg
Dose 2	For patients >70kg: 9.3E15 vg
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage	Phase3
Submit Date	2023-05-19
Completion Date	2028-06-30
Last Update	2025-06-22

Participation Criteria

Eligible Age
Standard Ages	Child, Adult, Older adult
Sexes Eligible for Study	MALE

Locations

No.of Trial Sites	46
Locations	Hong Kong,United States,Japan,United Kingdom,Spain,Canada,South Korea,Sweden,Belgium,Taiwan,Italy,Israel,Australia,Germany

Regulatory Information

Has US IND	True
FDA Designations	Accelerated Approval, Fast Track, Orphan Drug Designation, Priority Review, Rare Pediatric Disease Designation
Recent Updates	First approval was June 2023, expanded indication to all DMD patients regardless of ambulatory status or age on 6/20/24; new black box warning on risk of liver injury and liver failure added November 2025

Resources/Links

Clinical Publications

Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: An Exploratory Analysis From the EMBARK Randomized Clinical Trial
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR)
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial
Acute Liver Injury Following Delandistrogene Moxeparvovec Gene Therapy Requiring Intravenous Immunoglobulin
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy
Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial
Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy
(Abstract #P89) Long-Term Safety and Tolerability of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Phase 1 to Phase 3 Clinical Trials - MDA 2025
(Abstract #P70) Adverse Events post gene therapy in patients with Duchenne Muscular Dystrophy: A single center experience. - MDA 2025
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial