Clinical trial
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
NCT00999609 · Spark Therapeutics, Inc., Industry · Active, not recruiting
Key facts
| NCT number | NCT00999609 |
|---|---|
| Title | Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis |
| 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] |
| Lead sponsor | Spark Therapeutics, Inc., Industry |
| Collaborators | Children's Hospital of Philadelphia University of Iowa |
| Status | Active, not recruiting |
| Phase | Phase 3 |
| Study type | Interventional |
| Conditions | Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis |
| Intervention type | Biological |
| Interventions | AAV2-hRPE65v2, voretigene neparvovec-rzyl |
| Enrollment | 31 |
| Start date | Oct 1, 2012 |
| Primary completion | Jul 1, 2015 |
| Completion | Jan 1, 2030 |
| First posted | Oct 22, 2009 |
| Last update posted | Apr 23, 2025 |
| Countries | United States |
At a glance
- 1–9 Study sites
- Yes Results posted
- None Linked FDA submissions
Counts are shown as ranges. Exact figures, trends and every underlying record are available in Orca1.
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Source: U.S. National Library of Medicine (NLM), ClinicalTrials.gov · Source date: