Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

NCT05821959 · tracked via the Priya Life Science UK tracker

Sponsor

Akouos, Inc.

Phase

Phase 1/2

Started

2023-09-15

Last updated

2026-03-04

Condition(s) studied

Sensorineural Hearing Loss, Bilateral

Investigational drug(s) / intervention(s)

AAVAnc80-hOTOF via Akouos Delivery DeviceAAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism

AAVAnc80-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.

AAVAnc80-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.

Study 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

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

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 contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy 2. Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF 3. 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

Primary outcome measure(s)

Frequency of Adverse Events (AEs) — through trial completion, approximately 1 year
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery system)

Trial sites (9)

Facility	City	Region	Status
University of California, San Francisco Benioff Children's Hospital	San Francisco	California	Recruiting
University of Iowa	Iowa City	Iowa	Recruiting
Boston Children's Hospital	Boston	Massachusetts	Recruiting
Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	Recruiting
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	Recruiting
Vanderbilt Bill Wilkerson Center	Nashville	Tennessee	Recruiting
The Hospital for Sick Children Jaina.negandhi@sickkids.ca +1 (416) 813 6683	Toronto	Ontario	Recruiting
National Taiwan University Hospital	Taipei	Taiwan	Recruiting
University College London	London	United Kingdom	Recruiting

More Akouos, Inc. trials in the UK

Otoferlin Gene-mediated Hearing Loss Natural History Study Recruiting

Other trials for the same condition

Otoferlin Gene-mediated Hearing Loss Natural History Study Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05821959 on ClinicalTrials.gov ↗ ← All trials in the UK