An Open Label Clinical Trial of Retinal Gene Therapy for Choroideremia
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.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|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)|
- Number of patients with ocular and systemic adverse events [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]This is assessed by standard ocular examinations and vector dissemination and inflammation assays.
- Changes in visual field [ Time Frame: Baseline and up to 2 years following vector delivery ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: Yes ]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.
|Study Start Date:||April 2015|
|Estimated Study Completion Date:||December 2018|
|Estimated Primary Completion Date:||March 2017 (Final data collection date for primary outcome measure)|
Experimental: Open Label
Patients will receive a subretinal injection of 0.10 ml of the rAAV2.REP1 vector drug substance (10e11 AAV2 genome particles). It is a colourless opalescent frozen liquid with no visible particles. Each patient will be given a one-time dose in one eye.
It is the same vector used in the United Kingdom Phase I trial logged at: http://clinicaltrials.gov/ct2/show/NCT01461213.
Genetic: rAAV2.REP1 vector
No additional details needed.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02077361
|University of Alberta|
|Edmonton, Alberta, Canada, T6G 2E1|
|Principal Investigator:||Ian M MacDonald, MD, CM||University of Alberta|