This study examines a gene therapy (an adeno-associated virus vector called AAV-CNGA3) in patients with achromatopsia, an inherited eye condition caused by CNGA3 gene changes that leads to color blindness. Patients whose achromatopsia had been confirmed by a retinal specialist were able to take part. The study was carried out in Phase 1/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: * Are aged years or over * Have achromatopsia confirmed by a retinal specialist investigator Exclusion Criteria: * Are females who are pregnant or breastfeeding * Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months * Have any other condition that the investigator considers makes them inappropriate for entry into the trial