Trials / NCT00999609
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Underway, not enrolling Phase 3 Gene therapy
- Sponsor
- Spark Therapeutics, Inc.
- Treatment
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Gene
- RPE65
- Conditions
- Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
- Ages
- 3 Years to no upper limit
- Enrollment
- 31 participants
- Started
- 2012-10
- Main results expected
- 2015-07
- Results posted
- Yes
- Record last updated
- 2025-04-23
What the study is testing
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at either The Children's Hospital of Philadelphia or University of Iowa to determine if it improves visual and retinal function in individuals with RPE65 gene mutations.
Where it runs
United States (2)
- University of Iowa, Iowa City, Iowa
- Children's Hospital of Philadelphia, Philadelphia, Pennsylvania