Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Public ClinicalTrials.gov record NCT00999609. 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
A Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]
Study identification
- NCT ID
- NCT00999609
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Enrollment
- 31 participants
Conditions and interventions
Interventions
- AAV2-hRPE65v2,voretigene neparvovec-rzyl Biological
Biological
Eligibility (public fields only)
- Age range
- 3 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
- Sep 30, 2012
- Primary completion
- Jun 30, 2015
- Completion
- Dec 31, 2029
- Last update posted
- Apr 22, 2025
2012 – 2030
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Iowa | Iowa City | Iowa | 52242 | — |
| Children's Hospital of Philadelphia | 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 NCT00999609, 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 Apr 22, 2025 · Synced Jul 21, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00999609 live on ClinicalTrials.gov.