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

Biallelic RPE65 Mutation-associated Retinal Dystrophy Clinical Trial

Official title: An Open-label, Single-arm Study to Provide Efficacy and Safety Data of Voretigene Neparvovec Administered as Subretinal Injection in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Status Active, not recruiting

Clinical Trial Summary

The purpose of this study is to provide safety and efficacy data for voretigene neparvovec, administered as subretinal injection, in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy.

Clinical Trial Description

This is an open-label, single-arm study to evaluate the safety and efficacy of bilateral subretinal administration of voretigene neparvovec in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include full-field light sensitivity threshold testing, visual fields, visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment. ;

Related Conditions & MeSH terms

• Biallelic RPE65 Mutation-associated Retinal Dystrophy
• Retinal Dystrophies

• NCT number: NCT04516369
• Study type: Interventional
• Source: Novartis
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: November 24, 2020
• Completion date: May 31, 2026