Choroideremia Clinical Trial
Official title:
An Open Label Dose Escalation Phase 1 Clinical Trial of Retinal Gene Therapy for Choroideraemia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)
- 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.
Detailed description may be found in the following scientific publication:
Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2
clinical trial, The Lancet, Volume 383, Issue 9923, Pages 1129 - 1137 (29 March 2014).
Links: www.thelancet.com/journals/lancet/article/PIIS0140-6736(13)62117-0/abstract ;
http://dx.doi.org/doi:10.1016/S0140-6736(13)62117-0
;
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