This study looks at the safety of the RPE65 gene therapy called tgAAG76 in Leber congenital amaurosis, an inherited cause of vision loss due to mutations in the RPE65 gene. People with a confirmed missense mutation in RPE65 and a clinical diagnosis of severe early-onset retinal dystrophy could 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: * Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65 Exclusion Criteria: * Visual acuity in the study eye better than 6/36 Snellen * Hypertension * Diabetes mellitus * Tuberculosis * Renal impairment * Immunocompromise * Osteoporosis * Gastric ulceration * Severe affective disorder) * Pregnancy or lactation