NCT04516369View on ClinicalTrials.gov

Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

November 24, 2020

Primary Completion Date

April 5, 2022

Study Completion Date

June 22, 2026

Conditions
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Interventions
GENETIC

voretigene neparvovec

Voretigene neparvovec is an adeno-associated viral type 2 (AAV2) gene therapy vector driving expression of normal human retinal pigment epithelium 65 kDa protein (hRPE65) gene.

Trial Locations (1)

152-8902

Novartis Investigative Site, Meguro-ku

Sponsors
All Listed Sponsors
lead

Novartis Pharmaceuticals

INDUSTRY