Study 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.
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rAAV2.REP1DRUG
Single subretinal injection of rAAV2.REP1 vector suspension containing 10e12 genome particles per ml. Dose 1 = dose containing approximately 10e10 rAAV2.REP1 genome particles. Dose 2 = dose containing approximately 10e11 rAAV2.REP1 genome particles.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Moorfields Eye Hospital NHS Foundation Trust | London | United Kingdom | |
| St Mary's Hospital, Central Manchester University Hospitals NHS Foundation Trust | Manchester | United Kingdom | |
| Oxford Radcliffe Hospitals NHS Trust | Oxford | United Kingdom | |
| Eye Unit, Southampton University Hospitals NHS Trust | Southampton | United Kingdom |