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Health Condition(s) or Problem
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Choroideremia
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Lay Summary
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- 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 Objective: To identify any therapeutic benefit as evidenced by a slowing down of
the retinal degeneration assessed by functional and anatomical methods in the treated eye
compared to the control eye 24 months after gene delivery.
(from ClinicalTrials.gov)
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Who can enter the trial
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Inclusion Criteria:
- 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 Criteria:
- 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.
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Who cannot enter the trial
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Inclusion Criteria:
- 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 Criteria:
- 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.
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What will happen
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Drug; rAAV2.REP1; Single subretinal injection of vector suspension containing 10e12 genome particles per ml; Dose 1; Dose 2; Adeno-associated viral vector
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Primary aim
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Visual acuity
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Secondary Aim
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Microperimetry, OCT and fundus autofluorescence; 24 months; No; Structure function correlations at the margins of the retinal degeneration
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Participant Information Sheet
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Sorry, not currently available
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Website
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Sorry, not currently available
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Recruitment Status
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Recruiting
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Nation
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England
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Location
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London, Manchester, Oxford, Southampton
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