A Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa Due to Mutations in Exon 13 of the USH2A Gene

What this Trial Is About

Researchers are evaluating the safety and tolerability of ultevursen, given by intravitreal injection, in people with Retinitis Pigmentosa (RP) caused by mutations in exon 13 of the USH2A gene. This Phase 2b study is randomized, double-masked, and sham-controlled, involving 81 subjects. It aims to improve understanding of this treatment's effects in this specific genetic form of RP over two years. Participants will be randomly assigned in a 2:1 ratio to receive either ultevursen or a sham procedure. Those in the ultevursen group will receive four doses via intravitreal injection in the treatment eye: an initial dose on Day 1, followed by maintenance doses at Months 6, 12, and 18. Participants assigned to the sham group will undergo similar procedures without receiving the active drug. Throughout the study, participants will attend visits to assess vision and retinal function using tests such as static perimetry, microperimetry, and visual acuity charts. Researchers will also evaluate retinal structure with imaging methods and collect questionnaire data related to vision and anxiety. Safety will be monitored by tracking adverse events, and systemic drug exposure will be measured. The study duration is 24 months, during which efficacy and safety outcomes will be carefully assessed.

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene