At a glance
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A Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]
In Brief
A Phase 3 clinical trial evaluating AAV2-hRPE65v2,voretigene neparvovec-rzyl for Inherited Retinal Dystrophy Due to RPE65 Mutations and Leber Congenital Amaurosis. Active but no longer recruiting, targeting 31 participants across 2 sites.
Detailed Summary
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.
Study Details
Timeline
Interventions
Subretinal administration of gene therapy vector AAV2-hRPE65v2 (1.5E11 vector genomes per eye) to both eyes via surgical procedures on separate days.