Trials / NCT01461213
Gene Therapy for Blindness Caused by Choroideremia
Completed Phase 1 / Phase 2 Gene therapy
- Sponsor
- University of Oxford
- Treatment
- rAAV2.REP1
- Gene
- Not stated
- Conditions
- Choroideremia
- Ages
- 18 Years to no upper limit
- Enrollment
- 14 participants
- Started
- 2011-10
- Main results expected
- 2017-10
- Results posted
- Not yet
- Record last updated
- 2017-11-17
What the study is testing
- Primary objective: To assess the safety and tolerability of the AAV.REP1 vector, administered at two different doses to the retina in 12 patients with a diagnosis of choroideremia.
- Secondary Objective: To identify any therapeutic benefit as evidenced by a slowing down of the retinal degeneration assessed by functional and anatomical methods in the treated eye compared to the control eye 24 months after gene delivery.
Where it runs
United Kingdom (4)
- Moorfields Eye Hospital NHS Foundation Trust, London
- St Mary's Hospital, Central Manchester University Hospitals NHS Foundation Trust, Manchester
- Oxford Radcliffe Hospitals NHS Trust, Oxford
- Eye Unit, Southampton University Hospitals NHS Trust, Southampton