RecruitingPhase 1Phase 2NCT05821959

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations

Sponsor

Akouos, Inc.

Enrollment

22 participants

Start Date

Sep 15, 2023

Study Type

INTERVENTIONAL

Conditions

Sensorineural Hearing Loss, Bilateral

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.

Eligibility

Inclusion Criteria5

Participants may be of any age, based on Cohort Criteria
At least two mutations in the otoferlin gene
Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR
Preserved distortion product otoacoustic emissions (DPOAEs)
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

Exclusion Criteria3

Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy
Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF
Prior participation in a clinical trial with an investigational drug, within six months prior to administration, or any prior participation in a gene therapy clinical trial

Print for Your Doctor

Interested in this trial?

Get notified about updates and connect with the research team.

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.