Trials / NCT02946879
Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
Completed Observational Natural history / observational
- Sponsor
- MeiraGTx UK II Ltd
- Treatment
- AAV OPTIRPE65
- Gene
- RPE65
- Conditions
- Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
- Ages
- 3 Years to 100 Years
- Enrollment
- 15 participants
- Started
- 2016-11
- Main results expected
- 2023-07
- Results posted
- Not yet
- Record last updated
- 2024-04-11
What the study is testing
This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.
Where it runs
United Kingdom (1)
- Moorfields Eye Hospital NHS Foundation Trust, London
United States (1)
- Kellogg Eye Center, University of Michigan Health, Ann Arbor, Michigan