This study examined a gene therapy called rAAV2/4.hRPE65 in patients with retinal dystrophy caused by defects in the RPE65 gene. It enrolled patients with mutations leading to an abnormal RPE65 protein and low visual acuity. It 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: * Mutations that code for abnormal RPE65 protein * Presence of characteristic abnormalities in fundus * Dramatic reduction of both rods ans cones ERG responses * Low visual acuity \<0.32 * inform consent signed Exclusion Criteria: * Patients with chronic conditions such a haematological, cardiac, renal diseases * Patients with, within the past 6 months, a clinically significant cardiac disease or known congestive heart failure, cardiac rhytm and conduction abnormalities * Patients with pulmonaty dysfunction * Patients with suspected rheumatoid arthritis * Patients with current systemic infection........