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This study examined AAV-mediated REP1 gene replacement therapy, a gene therapy, in choroideremia, an inherited eye disease caused by REP1 protein deficiency. It enrolled men aged 18 and older with a genetically confirmed diagnosis of choroideremia. It was a Phase 2 study 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: 1. Candidate is willing and able to give informed consent for participation in the study. 2. Male aged 18 years or above. 3. Genetic or molecular confirmed diagnosis of choroideremia (REP1 protein deficiency). 4. Active disease visible clinically within the macula region. 5. Best corrected visual acuity better than or equal to 6/60 (20/200; Decimal 0.1; LogMAR 1.0) in the study eye. Exclusion Criteria: 1. Any female, or a male aged below 18 years. 2. An additional cause for sight loss (e.g. amblyopia) in the eye to be treated. 3. Any other significant ocular and non-ocular disease or disorder which, in the opinion of the investigator, may put the participants at risk because of participation in the study. 4. Inability to take systemic prednisolone for a period of 45 days. 5. Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery. 6. Participation in another research study involving an investigational product in the preceding 12 weeks.