Clinical Trial: Gene Therapy for Blindness Caused by Choroideremia

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

Brief 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.


Detailed Summary:

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


Sponsor: University of Oxford

Current Primary Outcome: Visual acuity [ Time Frame: 6 months ]

Best corrected visual acuity, following cataract surgery if indicated


Original Primary Outcome: Same as current

Current Secondary Outcome: Microperimetry, OCT and fundus autofluorescence [ Time Frame: 24 months ]

Structure function correlations at the margins of the retinal degeneration


Original Secondary Outcome: Same as current

Information By: University of Oxford

Dates:
Date Received: October 21, 2011
Date Started: October 2011
Date Completion: June 2017
Last Updated: March 16, 2016
Last Verified: March 2016