Status:
COMPLETED
Gene Therapy for Blindness Caused by Choroideremia
Lead Sponsor:
University of Oxford
Collaborating Sponsors:
Oxford University Hospitals NHS Trust
Moorfields Eye Hospital NHS Foundation Trust
Conditions:
Choroideremia
Eligibility:
MALE
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
\- Primary objective: To assess the safety and tolerability of the AAV.REP1 vector, administered at two different doses to the retina in 12 patients with a diagnosis of choroideremia. \- Secondary Ob...
Detailed Description
Detailed description may be found in the following scientific publication: Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial, The Lancet, Volume 38...
Eligibility Criteria
Inclusion
- Participant is willing and able to give informed consent for participation in the study,
- Male aged 18 years or above,
- Diagnosed with choroideraemia and in good health,
- Active disease with SLO changes visible within the macula region,
- Willing to allow his or her General Practitioner and consultant, if appropriate, to be notified of participation in the study,
- Vision at least 6/60 or better in the study eye.
Exclusion
- Female and child participants (under the age of 18),
- Men unwilling to use barrier contraception methods, if relevant,
- Previous history of retinal surgery or ocular inflammatory disease (uveitis),
- Grossly asymmetrical disease or other ocular morbidity which might confound use of the fellow eye as a long-term control,
- Any other significant disease or disorder which, in the opinion of the Investigator, may either put the participants at risk because of participation in the study, or may influence the result of the study, or the participant's ability to participate in the study,
- Participants who have participated in another research study involving an investigational product in the previous 12 weeks.
Key Trial Info
Start Date :
October 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2017
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT01461213
Start Date
October 1 2011
End Date
October 1 2017
Last Update
November 17 2017
Active Locations (4)
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1
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom, EC1V 2PD
2
St Mary's Hospital, Central Manchester University Hospitals NHS Foundation Trust
Manchester, United Kingdom, M13 9WL
3
Oxford Radcliffe Hospitals NHS Trust
Oxford, United Kingdom, OX3 9DU
4
Eye Unit, Southampton University Hospitals NHS Trust
Southampton, United Kingdom, SO16 6YD