This study examined a gene therapy called AAV2-REP1, delivered by injection into the eye, for the inherited eye disease choroideremia. It enrolled men aged 18 and older with a genetically confirmed diagnosis of choroideremia. It was conducted in Phase 2 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * 18 Years and older * Male * Able to give informed consent * Genetically confirmed diagnosis of choroideremia * Active disease visible clinically within the macula region * Best-corrected visual acuity equal to or worse than 20/32 but better than or equal to 20/200 in the study eye. Exclusion Criteria: * Female * Under the age of 18 * History of amblyopia in the study eye * Men unwilling to use barrier contraception methods * Relevant grossly asymmetrical disease or other ocular morbidity which might confound use of the fellow eye as a long-term control * Any other significant ocular and non-ocular disease/disorder or retinal surgery * Contraindication to use of medications or contrast agents * Participated in research study involving an investigational product in the past 12 weeks * Having had gene or cellular therapy at any time prior to this study.