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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
No additional details needed.
University of Alberta
Edmonton, Alberta, Canada
Number of patients with ocular and systemic adverse events
This is assessed by standard ocular examinations and vector dissemination and inflammation assays.
Time frame: 2 years
Changes in visual field
This is assessed by Goldmann perimetry and microperimetry; measurements before and after vector delivery are compared.
Time frame: Baseline and up to 2 years following vector delivery
Changes in visual function
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.
Time frame: Baseline and 2 years following vector delivery
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