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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.

Conditions
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Interventions / Drugs
voretigene neparvovec
View on ClinicalTrials.gov ↗
Sponsor
NVS
Novartis AG
Diversified Pharma
D36/100
Study Details
Enrollment4 participants
Primary Completion—
Lead SponsorNovartis Pharmaceuticals
Data Retrieved2026-04-11 02:55:49.151860+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 4 participants
Sponsor Catalyst Profile

Novartis AG (NVS) is the sponsoring company for this trial. BiotechSign currently grades this company D (36/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

NVS Full Pipeline →All Companies →
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