Clinical Trial: An Open Label Clinical Trial of Retinal Gene Therapy for Choroideremia
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: An Open Label Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein-1 (REP1)
Brief Summary: A project has been developed in Edmonton, Alberta, Canada to enable male patients with choroideremia to access a clinical trial that replaces the defective gene with a normal copy. This experiment is designed to show that the transfer of a normal copy of the gene to the eye is not only safe but may improve the sight of patients. Only Canadian subjects who meet criteria will be recruited.
Detailed Summary: This is an open label study involving a total of 6 male patients. Screening and patient medical records will determine patient eligibility. Patients will receive a subretinal injection of the rAAV2.REP1 vector by a trained vitreoretinal surgeon in one eye. Each patient will be followed up for 24 months after treatment to assess the primary and secondary endpoints of this study using a number of outcome measures. However, further follow-up will continue after the study on an annual basis for a minimum of ten years. Data will continue to be analyzed by members of the study group after this study is complete.
Sponsor: Ian M. MacDonald
Current Primary Outcome: Number of patients with ocular and systemic adverse events [ Time Frame: 2 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Changes in visual field [ Time Frame: Baseline and up to 2 years following vector delivery ]This is assessed by Goldmann perimetry and microperimetry; measurements before and after vector delivery are compared.
- Changes in visual function [ Time Frame: Baseline and 2 years following vector delivery ]This is assessed by multifocal electrophysiology, full field scotopic threshold, spectral domain optical coherent tomography, fundus photography and fundus autofluorescence; measurements before and after vector delivery are compared.
Original Secondary Outcome: Same as current
Information By: University of Alberta
Dates:
Date Received: February 26, 2014
Date Started: April 2015
Date Completion: December 2018
Last Updated: May 1, 2017
Last Verified: May 2017