Gene Therapy for Blindness Caused by Choroideremia
Status:
Completed
Trial end date:
2017-10-01
Target enrollment:
Participant gender:
Summary
- 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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
University of Oxford
Collaborators:
Central Manchester University Hospitals NHS Foundation Trust Manchester University NHS Foundation Trust Moorfields Eye Hospital NHS Foundation Trust Oxford University Hospitals NHS Trust University College, London University Hospital Southampton NHS Foundation Trust University of Manchester University of Southampton