An Open Label Clinical Trial of Retinal Gene Therapy for Choroideremia
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|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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|