A Double-Masked Study to Test Sepofarsen Treatment in People with Leber Congenital Amaurosis Type 10 Caused by a CEP290 Gene Mutation

What this Trial Is About

This research aims to evaluate the efficacy, safety, and tolerability of Sepofarsen in people with Leber Congenital Amaurosis LCA caused by a specific mutation in the CEP290 gene. The study is a double-masked, randomized, placebo-controlled trial where each participants two eyes are treated differently to compare effects. It focuses on patients with LCA type 10 and uses a paired-eye design to better understand treatment outcomes. Participants will receive Sepofarsen or a placebo injected into one eye every six months. In the first year, one eye gets Sepofarsen and the other eye receives placebo. During the second year, the eye initially treated with Sepofarsen continues with it, while the eye initially treated with placebo either continues with placebo or starts Sepofarsen, depending on random assignment. The doses start with 160 g then 40 g for Sepofarsen in the initial treatment and 40 g in later treatments. Throughout the study, participants will undergo regular eye exams including tests for visual acuity, retinal sensitivity, contrast sensitivity, and patient-reported vision changes. Researchers will measure changes in best-corrected visual acuity after 12 months as the main outcome. The study lasts up to two years, with ongoing safety and efficacy monitoring. Participants visual function and quality of life will also be assessed using questionnaires and clinical tests related to their eye condition.

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)