This study evaluated the safety and effect of a gene therapy called AAV RPE65 in patients with Leber congenital amaurosis (LCA) caused by RPE65 deficiency. It included people aged 3 and older with early-onset severe retinal dystrophy consistent with RPE65 deficiency. It was conducted 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.
Key Inclusion Criteria: * Aged 3 years or older * Early-onset severe retinal dystrophy consistent with RPE65 deficiency Key Exclusion Criteria: * Females who are pregnant or breastfeeding * Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.