Phase 12 Study of Subretinal SB-007 Gene Therapy for Stargardt Disease Caused by ABCA4 Gene Mutations

What this Trial Is About

This research aims to evaluate the safety, tolerability, and preliminary effectiveness of SB-007, a gene therapy delivered by subretinal injection, in people with Stargardt Disease type 1 STGD1. STGD1 is an inherited retinal disorder caused by mutations in the ABCA4 gene leading to vision loss, typically starting in childhood or early adulthood. The study will enroll about 57 participants and follow them for 96 weeks after treatment to determine the best dose of SB-007. Participants will receive a single subretinal injection of SB-007. The study includes several groups receiving different doses low, medium, high, and doses based on the maximum tolerated dose MTD identified in earlier phases. There is also a control group that will not receive the active treatment. SB-007 uses a novel dual AAV8 gene delivery method aiming to restore the full-length ABCA4 protein in the retina to potentially slow vision loss. During the study, participants will undergo regular safety checks and eye examinations over 96 weeks. Assessments include measuring changes in lesion size, retinal sensitivity, visual acuity under normal and low light, and quality of life through questionnaires. Researchers will monitor safety and treatment effects closely to understand how SB-007 works and its impact on disease progression. The total study duration includes treatment and follow-up.

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)