Trials / NCT01208389
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
- Sponsor
- Spark Therapeutics, Inc.
- Treatment
- voretigene neparvovec-rzyl
- Gene
- RPE65
- Conditions
- Leber Congenital Amaurosis
- Ages
- 8 Years to no upper limit
- Enrollment
- 12 participants
- Started
- 2010-11
- Main results expected
- 2027-10
- Results posted
- Not yet
- Record last updated
- 2025-04-29
What the study is testing
The study is a follow-on to a Phase 1 dose-escalation and safety study.
Where it runs
United States (1)
- The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania