This study examines a gene therapy (AAV-CNGB3) delivered using an AAV vector in people with achromatopsia (an inherited eye disease causing color blindness) linked to the CNGB3 gene. It enrolled people aged 3 and over whose achromatopsia was confirmed by a retinal specialist. 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 3 years or older * Have achromatopsia confirmed by a retinal specialist (CI or PI) 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 CI/PI considers makes them inappropriate for entry into the trial