Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCTID	NCT00999609 (View at clinicaltrials.gov)
Description	The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at either The Children's Hospital of Philadelphia or University of Iowa to determine if it improves visual and retinal function in individuals with RPE65 gene mutations. (Show More)
Indication	Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Compound Name	LUXTURNA/voretigene neparvovec (AAV2-hRPE65v2)
Sponsor	Spark Therapeutics, Inc.
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	31

Target Gene/Variant	RPE65
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type	none
Dose 1	1E11 vg/eye (0.3mL)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
Recent Updates	FDA approved 12/19/17, Price/treatment $850K/eye