An Open Label Clinical Trial of Retinal Gene Therapy for Choroideremia
|ClinicalTrials.gov Identifier: NCT02077361|
Recruitment Status : Completed
First Posted : March 4, 2014
Last Update Posted : November 7, 2017
|Condition or disease||Intervention/treatment||Phase|
|Choroideremia||Genetic: rAAV2.REP1 vector||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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)|
|Study Start Date :||April 2015|
|Actual Primary Completion Date :||August 30, 2017|
|Actual Study Completion Date :||August 30, 2017|
Experimental: Open Label
Patients will receive a subretinal injection of 0.10 ml of the rAAV2.REP1 vector drug substance. 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/II trial logged at: http://clinicaltrials.gov/ct2/show/NCT01461213.
Genetic: rAAV2.REP1 vector
No additional details needed.
- Number of patients with ocular and systemic adverse events [ Time Frame: 2 years ]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 ]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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02077361
|University of Alberta|
|Edmonton, Alberta, Canada, T6G 2E1|
|Principal Investigator:||Ian M MacDonald, MD, CM||University of Alberta|