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

> **NCT04516369** · PHASE3 · ACTIVE_NOT_RECRUITING · sponsor: **Novartis Pharmaceuticals** · enrollment: 4 (actual)

## Conditions studied

- Biallelic RPE65 Mutation-associated Retinal Dystrophy

## Interventions

- **GENETIC:** voretigene neparvovec

## Key facts

- **NCT ID:** NCT04516369
- **Lead sponsor:** Novartis Pharmaceuticals
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2020-11-24
- **Primary completion:** 2022-04-05
- **Final completion:** 2026-06-22
- **Target enrollment:** 4 (ACTUAL)
- **Last updated:** 2025-04-25


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04516369

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04516369, "Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy". Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/clinical/NCT04516369. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*