Trials / NCT00643747
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Completed Phase 1 / Phase 2 Gene therapy
- Sponsor
- University College, London
- Treatment
- tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)
- Gene
- RPE65
- Conditions
- Retinal Degeneration
- Ages
- 5 Years to 30 Years
- Enrollment
- 12 participants
- Started
- 2007-01
- Main results expected
- 2014-12
- Results posted
- Not yet
- Record last updated
- 2015-12-07
What the study is testing
The purpose of the study is to determine whether gene therapy is safe and effective for the treatment of severe childhood blindness caused by mutations in RPE65.
Where it runs
United Kingdom (1)
- Moorfields Eye Hospital NHS Foundation Trust, London