# Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

> **NCT00999609** · PHASE3 · ACTIVE_NOT_RECRUITING · sponsor: **Spark Therapeutics, Inc.** · enrollment: 31 (actual)

## Conditions studied

- Inherited Retinal Dystrophy Due to RPE65 Mutations
- Leber Congenital Amaurosis

## Interventions

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

## Key facts

- **NCT ID:** NCT00999609
- **Lead sponsor:** Spark Therapeutics, Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2012-10
- **Primary completion:** 2015-07
- **Final completion:** 2030-01
- **Target enrollment:** 31 (ACTUAL)
- **Last updated:** 2025-04-23

## Collaborators

- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00999609

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00999609, "Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis". Retrieved via AI Analytics 2026-07-18 from https://api.ai-analytics.org/clinical/NCT00999609. Licensed CC0.

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