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	Recruiting
Enrollment Count	148
Results Posted	Not Available

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

No.of Trial Sites	43
Locations	Hong Kong,United States,Japan,United Kingdom,Spain,Sweden,Belgium,Taiwan,Korea, Republic of,Italy,Israel,Australia,Germany

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