A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
Akouos, Inc.
Summary
Part A of this trial will evaluate the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos Delivery Device, together with the Precision Delivery Mechanism, to safely achieve the intended product performance.
Description
The following two dose levels will be tested: * Up to 4.1E11 total vg/cochlea * Up to 8.1E11 total vg/cochlea
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
Criteria for Inclusion: 1. Participants may be of any age, based on Cohort Criteria 2. At least two mutations in the otoferlin gene 3. Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR 4. Preserved distortion product otoacoustic emissions (DPOAEs) 5. Able and willing to comply with all study requirements, including willingness to participate in a separate long term follow-up study after completion of this trial Criteria for Exclusion: 1. Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contrai…
Interventions
- Combination ProductAAVAnc80-hOTOF via Akouos Delivery Device
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
- Combination ProductAAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time bilateral intracochlear administration using the Akouos Delivery Device together with the Precision Delivery Mechanism.
Locations (9)
- University of California, San Francisco Benioff Children's HospitalSan Francisco, California
- University of IowaIowa City, Iowa
- Boston Children's HospitalBoston, Massachusetts
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania
- Vanderbilt Bill Wilkerson CenterNashville, Tennessee