RECRUITING Phase 3 ClinicalTrials.gov

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Sponsor: Eli Lilly and Company • ClinicalTrials.gov ID: NCT05821959

Registry updated: 2026-09-29

Recruiting for Similar Conditions?

Sponsors are looking for patients like you. See if you qualify for compensated research studies.

Description

Part A of this trial evaluated 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 (LY888891) and evaluated the Akouos Delivery Device, together with the Precision Delivery Mechanism (as applicable), to safely achieve the intended product performance. Part B of the trial is an open-label, global expansion phase to confirm the efficacy and safety of one dose level of AAVAnc80-hOTOF administered in one or both ear(s). Part B will also evaluate the Intracochlear Delivery System (IDS), which consists of the Akouos Delivery Device, the Precision Delivery Mechanism, and an off-the-shelf syringe. Part C is a region-specific expansion phase. Part C participants will receive the same dose level of AAVAnc80-hOTOF as participants in Part B, in one or both ear(s) utilizing the IDS. Part D is a sequential administration phase that allows participants who received AAVAnc80-hOTOF previously in a single ear to receive AAVAnc80-hOTOF administration in the other ear utilizing the IDS.

Interventions

AAVAnc80-hOTOF via Akouos Delivery Device (Combination product)
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 (Combination product)
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.

Eligibility criteria

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 (Part A) or Severe to Profound (Parts B, C, and D) 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 (with the exception of AK-OTOF-101 for Part D participants)

Conditions

Sensorineural Hearing Loss, Bilateral

Locations

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

Partner with Nurenyx

Showcase your study to thousands of motivated patients matching your criteria.

Learn More

Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

View Official Record

Accelerate Your Clinical Trial Recruitment