Trials / NCT00516477
Safety Study in Subjects With Leber Congenital Amaurosis
Completed Phase 1 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
- 2007-09
- Main results expected
- 2018-03-20
- Results posted
- Not yet
- Record last updated
- 2020-11-02
What the study is testing
The purpose of this study is to determine whether gene transfer will be safe and effective in the treatment of Leber Congenital Amaurosis (LCA).
Where it runs
United States (1)
- The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania