Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations
Public ClinicalTrials.gov record NCT00481546. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-CBSB-hRPE65) Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations (Clinical Trials of Gene Therapy for Leber Congenital Amaurosis)
Study identification
- NCT ID
- NCT00481546
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- University of Pennsylvania
- Other
- Enrollment
- 15 participants
Conditions and interventions
Conditions
Interventions
- rAAV2-CBSB-hRPE65 Genetic
Genetic
Eligibility (public fields only)
- Age range
- 8 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 30, 2007
- Primary completion
- Nov 30, 2016
- Completion
- Nov 30, 2016
- Last update posted
- Jul 16, 2026
2007 – 2016
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Shands Children's Hospital, University of Florida | Gainesville | Florida | 32610 | — |
| Scheie Eye Institute, University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00481546, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 16, 2026 · Synced Jul 21, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00481546 live on ClinicalTrials.gov.