Safety and Effectiveness of Subretinal AAVB-039 Treatment in Stargardt Disease Patients Aged 8 to 55

What this Trial Is About

Researchers are evaluating the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease caused by mutations in the ABCA4 gene. This study also aims to assess the initial effects of AAVB-039 treatment for this inherited eye condition. The trial is an open-label, multicenter study conducted in two parts, using ascending doses followed by a controlled phase. Participants receive one subretinal injection of AAVB-039 at one of three dose levels low, mid, or high. The study involves sequential cohorts receiving these doses to assess safety and efficacy. There is no placebo group, and all participants are treated with AAVB-039. The treatment involves a single administration directly under the retina. During the study, participants will be monitored for treatment-related adverse events for up to 60 months. Researchers will also measure changes in the area of retinal atrophy, ellipsoid zone loss, macular sensitivity via microperimetry, and visual acuity. Follow-up visits and assessments will help track the long-term safety and potential effects of the gene therapy on vision over several years.

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)