This study evaluated the safety and different doses of the gene therapy AAV2-hCHM in participants with choroideremia, an inherited eye disease caused by CHM gene mutations. It enrolled men aged 18 and older with a confirmed diagnosis. It was a Phase 1/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: * Male at least 18 years of age diagnosed with CHM gene mutation * Central visual field (VF) \<30° in any of the 24 meridians (using Goldmann perimetry III4e isopter) in the eye to be injected * Any evidence of functioning outer retinal cells within the central 10° Exclusion Criteria: * Previous history of ocular inflammatory disease (uveitis) * Prior intraocular surgery within six months * Participation in a previous gene therapy research trial within one year of enrollment or participation in any other ocular gene therapy trial * Participation in a clinical study with an investigational drug in the past six months * Grossly asymmetrical disease, or other eye morbidity, which may render the contralateral eye ineffective as a control * Visual acuity \<20/200 on standard Early Treatment of Diabetic Retinopathy Study (ETDRS) testing in the eye to be injected * Presence of disease which may preclude the participant from participation in this trial * Use of medications known to be neuroprotective or retino-toxic that could potentially interfere with the disease process and/or cause ocular adverse events; individuals who discontinue use of these compounds for 6 months may become eligible * Identification by the investigator as being unable or unwilling to perform/be compliant with study procedures.