NCT05821959From ClinicalTrials.govRecruiting

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

Sensorineural Hearing Loss, Bilateral

At a glance

Phase: Phase 3
Study type: Interventional
Sponsor: Eli Lilly and Company
Enrolment target: 48
Started: 15 September 2023
Main results due: October 2028
Study sites: 9
Registry updated: 29 September 2026

Can you take part?

No age limit is stated.
Open to any sex.
You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full 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)

What this study is about

In the sponsor’s own words, from the registry.

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.

Study sites(9)

University of California, San Francisco Benioff Children's Hospital
San Francisco, California, United States
Recruiting
University of Iowa
Iowa City, Iowa, United States
Recruiting
Boston Children's Hospital
Boston, Massachusetts, United States
Active, not recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Active, not recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Active, not recruiting
Vanderbilt Bill Wilkerson Center
Nashville, Tennessee, United States
Recruiting
The Hospital for Sick Children Jaina.negandhi@sickkids.ca +1 (416) 813 6683
Toronto, Ontario, Canada
Active, not recruiting
National Taiwan University Hospital
Taipei, Taiwan
Active, not recruiting
University College London
London, United Kingdom, United Kingdom
Active, not recruiting

Showing 9 of 9 sites. 3 of the 9 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT05821959. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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