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)
Indication	Duchenne Muscular Dystrophy
Compound Name	ELEVIDYS/delandistrogene moxeparvovec
Sponsor	Sarepta Therapeutics, Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	148

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	42
Locations	Hong Kong,United States,Japan,United Kingdom,Spain,Sweden,Belgium,Taiwan,Korea, Republic of,Italy,Israel,Australia,Germany

Has US IND	True
Recent Updates	First approval was June 2023, expanded indication to all DMD patients regardless of ambulatory status or age on 6/20/24