An Open Label Clinical Trial of Retinal Gene Therapy for Choroideremia

Status Completed

Clinical Trial 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.

Clinical Trial Description

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 Design

Related Conditions & MeSH terms

Choroideremia

Clinical Trial Details

NCT number	NCT02077361
Study type	Interventional
Source	University of Alberta
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	April 2015
Completion date	May 16, 2022

View Details

See also
Status	Clinical Trial	Phase
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Completed	`NCT01461213` - Gene Therapy for Blindness Caused by Choroideremia	Phase 1/Phase 2
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Withdrawn	`NCT05045703` - The Dark-Adapted Retinal Function Response in Choroideremia (DARC) Study	N/A
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Completed	`NCT01603576` - Pilot Study of a Suprachoroidal Retinal Prosthesis	N/A
Enrolling by invitation	`NCT03584165` - Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa	Phase 3
Completed	`NCT03359551` - Natural History of the Progression of Choroideremia Study
Recruiting	`NCT02435940` - Inherited Retinal Degenerative Disease Registry
Active, not recruiting	`NCT04483440` - Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia	Phase 1
Completed	`NCT02671539` - THOR - Tübingen Choroideremia Gene Therapy Trial	Phase 2
Completed	`NCT02670980` - Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy	N/A
Completed	`NCT02341807` - Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations	Phase 1/Phase 2
Completed	`NCT03406416` - Study of a Suprachoroidal Retinal Prosthesis	N/A
Enrolling by invitation	`NCT05158049` - Longitudinal Study of a Bionic Eye
Terminated	`NCT02994368` - "Natural History" Study of Choroideremia
Terminated	`NCT01654562` - The Short-term Effects of Simvastatin on the Vision of Males Affected by Choroideremia	Phase 1/Phase 2
Active, not recruiting	`NCT00427180` - IRIS PILOT - Extended Pilot Study With a Retinal Implant System	N/A