Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

NCTID	NCT03333590 (View at clinicaltrials.gov)
Description	The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients. There will be a modified intravascular limb infusion (ILI) procedure that will be used to sequentially deliver vector to each whole lower limb of DMD subjects via a major lower limb artery. (Show More)
Development Status	Inactive
Indication	Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term	DOID:11723
Compound Name	RAAVrh74.MCK.GALGT2
Sponsor	Kevin Flanigan
Funder Type	Other
Recruitment Status	Completed
Enrollment Count	2
Results Posted	View Results

Target Gene/Variant	GALGT2
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Isolated limb infusion (femoral artery)
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh74
Editor Type	none
Dose 1	2.5E13 vg/kg/leg
Dose 2	5E13 vg/kg/leg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Licensed to Sarepta in December 2015, only 2 patients were dosed out of 6 planned, efficacy data was not compelling