Clinical Trial: REP1 Gene Replacement Therapy for Choroideremia
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)
Brief Summary: The assessment of the efficacy (with respect to preservation of visual function and retinal structure) and safety of a single subretinal injection of AAV2.REP1 in participants with a confirmed diagnosis of choroideremia, as evaluated by various functional and anatomical outcomes measured over a number of time points up to 24 months post-treatment.
Detailed Summary:
Sponsor: University of Oxford
Current Primary Outcome: Change from baseline in best corrected visual acuity in the treated eye [ Time Frame: 2 years ]
Original Primary Outcome: Change from baseline in best corrected visual acuity in treated eye, compared to untreated control eye [ Time Frame: 2 years ]
Current Secondary Outcome:
- Change from baseline in the central visual field in the treated eye as determined by microperimetry [ Time Frame: 2 years ]
- Change from baseline in the area of surviving retinal pigment epithelium in the treated eye as measured by fundus autofluorescence, compared to the untreated fellow eye (control eye) after randomisation of treatment to one eye or the other [ Time Frame: 2 years ]
Original Secondary Outcome:
- Change from baseline in autofluorescence evaluation in treated eye, compared to untreated control eye [ Time Frame: 2 years ]
- Change from baseline in central visual field using microperimetry readings in treated eye, compared to untreated control eye [ Time Frame: 2 years ]
Information By: University of Oxford
Dates:
Date Received: March 10, 2015
Date Started: August 2016
Date Completion:
Last Updated: May 2, 2017
Last Verified: May 2017
