Gene Therapy Study Using AAVAnc80-hOTOF for People With Hearing Loss Caused by Otoferlin Gene Mutations

What this Trial Is About

Researchers are evaluating a gene therapy called AAVAnc80-hOTOF for individuals with sensorineural hearing loss caused by mutations in the otoferlin gene. This trial will assess the safety and tolerability of a single surgical administration procedure using the Akouos Delivery Device and Precision Delivery Mechanism. The study aims to safely deliver the gene therapy to one or both ears and monitor the effects over approximately one year. The trial includes several groups receiving different doses of AAVAnc80-hOTOF administered directly into the cochlea using a sterile, one-time use investigational device. Dose levels tested are up to 4.1E11 and 8.1E11 total viral genomes per cochlea. Participants may receive treatment in one ear or both ears simultaneously using the delivery devices. The study involves cohorts based on age and dosing, including a bilateral expansion group. Participants will undergo evaluations throughout the trial, including monitoring for adverse events and hearing function via auditory brainstem response tests. The performance of the delivery devices will also be assessed. Participants must be able and willing to comply with all study visits and procedures, including a separate long-term follow-up study after the trial. The total participation time includes approximately one year of safety and efficacy monitoring.

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss