Phase 12 Open-label Study of SKY-GJB2 Gene Therapy Delivered by SKY-CAT Device for Children Aged 9 Months to 7 Years With GJB2-related Hearing Loss

What this Trial Is About

Researchers are studying an investigational gene therapy called SKY-GJB2 to treat children with hearing loss caused by changes in the GJB2 gene, also known as Connexin 26. The purpose is to learn about the safety and tolerability of SKY-GJB2 as well as how well it works in improving hearing in pediatric subjects with this genetic condition. Participants will receive a one-time intracochlear infusion of SKY-GJB2 delivered using a sterile, single-use investigational device called SKY-CAT. Two groups of children will be treated one group aged 2 to 7 years and another aged 9 months to less than 2 years. The therapy is administered in one ear only during the study. During the study, participants will be monitored for safety and adverse events up to approximately one year after treatment. Hearing will be assessed using Auditory Brainstem Response ABR thresholds over this period. The performance of the delivery device SKY-CAT will also be evaluated one month after the procedure. Parents or guardians will provide informed consent and children able to assent will do so. The study lasts about one year per participant after treatment.

Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss