# A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

> **NCT03597399** · — · COMPLETED · sponsor: **Spark Therapeutics, Inc.** · enrollment: 87 (actual)

## Conditions studied

- Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy

## Interventions

- **BIOLOGICAL:** AAV2-hRPE65v2,voretigene neparvovec-rzyl

## Key facts

- **NCT ID:** NCT03597399
- **Lead sponsor:** Spark Therapeutics, Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** —
- **Study type:** OBSERVATIONAL
- **Status:** COMPLETED
- **Start date:** 2019-01-10
- **Primary completion:** 2025-06-30
- **Final completion:** 2025-06-30
- **Target enrollment:** 87 (ACTUAL)
- **Last updated:** 2025-09-22


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03597399

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03597399, "A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US". Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/clinical/NCT03597399. Licensed CC0.

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