Trials / NCT01496040
Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Completed Phase 1 / Phase 2 Gene therapy
- Sponsor
- Nantes University Hospital
- Treatment
- rAAV2/4.hRPE65
- Gene
- RPE65
- Conditions
- Leber Congenital Amaurosis
- Ages
- 6 Years to 50 Years
- Enrollment
- 9 participants
- Started
- 2011-09
- Main results expected
- 2014-08
- Results posted
- Not yet
- Record last updated
- 2026-09-14
What the study is testing
The purpose of the study is to assess the safety and efficacy of the active substance rAAV-2/4.hRPE65 in patients with Leber Congenital Amaurosis or Congenital severe early-onset retinal degeneration associated with RPE65 mutation.
Where it runs
France (1)
- CHU Nantes, Nantes