A Phase 12 Study to Evaluate the Safety and Effectiveness of DB-OTO Gene Therapy in Children, Infants, Adolescents, and Adults With Severe Hearing Loss Due to Otoferlin Gene Changes

What this Trial Is About

Researchers are evaluating DB-OTO, an investigational gene therapy, for children, infants, adolescents, and adults who have severe to profound hearing loss caused by mutations in the otoferlin gene. This study aims to learn about the safety of DB-OTO, how well it is tolerated without ongoing discomfort, and how effective it is in improving hearing abilities. Participants will receive DB-OTO through one of several dosing approaches including unilateral intracochlear injection, bilateral intracochlear injection based on safety and efficacy data from an initial phase, or either unilateral or bilateral dosing. The trial is open-label and includes sequential cohorts to evaluate safety, tolerability, and efficacy. The study consists of different parts where dosing and monitoring are tailored accordingly. During the study, participants will undergo various assessments to measure hearing sensitivity using Pure Tone Audiometry and Auditory Brainstem Response tests, as well as speech perception and auditory skill evaluations. Safety will be monitored by tracking treatment-emergent adverse events up to week 104. Participants and their guardians will be closely followed for over two years to evaluate hearing improvements and any changes in speech abilities or safety outcomes.

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations