# Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations

> **NCT00481546** · PHASE1 · ACTIVE_NOT_RECRUITING · sponsor: **University of Pennsylvania** · enrollment: 15 (estimated)

## Conditions studied

- Amaurosis of Leber
- Retinal Diseases

## Interventions

- **GENETIC:** rAAV2-CBSB-hRPE65

## Key facts

- **NCT ID:** NCT00481546
- **Lead sponsor:** University of Pennsylvania
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2007-07
- **Primary completion:** 2026-06
- **Final completion:** 2026-06
- **Target enrollment:** 15 (ESTIMATED)
- **Last updated:** 2025-08-05

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00481546

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00481546, "Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations". Retrieved via AI Analytics 2026-07-11 from https://api.ai-analytics.org/clinical/NCT00481546. Licensed CC0.

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