HomeTrialNCT04516369
🔬NCT04516369Phase 3ACTIVE NOT RECRUITING

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

Study 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.

Interventions / Drugs
View on ClinicalTrials.gov ↗
Study Details
Enrollment4 participants
Primary Completion
Lead SponsorNovartis Pharmaceuticals
Data Retrieved2026-04-11 02:55:49.151860+00:00
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