A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)

NCTID	NCT01976091 (View at clinicaltrials.gov)
Description	This is an open-label, dose escalation gene transfer therapy study evaluating the safety of SRP-9004 (patidistrogene bexoparvovec) via isolated limb infusion (ILI) administration in approximately 6 participants with LGMD2D. (Show More)
Development Status	Active
Indication	Limb-Girdle Muscular Dystrophy, Type 2D/R3
Disease Ontology Term	DOID:0110278
Compound Name	SRP-9004
Compound Alias	Patidistrogene bexoparvovec
Compound Description	rAAVrh74-tMCK-SGCA
Sponsor	Sarepta Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	6
Results Posted	View Results

Target Gene/Variant	SGCA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Isolated limb infusion
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh74
Editor Type	none
Dose 1	1E12 vg/kg/limb
Dose 2	3E12 vg/kg/limb
Dose 3
Dose 4
Dose 5

No.of Trial Sites
Locations

Has US IND	True
FDA Designations
Recent Updates	Phase 1b study initiating in December 2024 (NCT06747273)