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RecruitingPhase 3

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Eli Lilly and Company · NCT05821959

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Any age listed · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 3
Target enrollment
48
US sites recruiting
3
Study start
September 15, 2023
Record last updated
September 29, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: AAVAnc80-hOTOF via Akouos Delivery Device; AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism

Conditions: Sensorineural Hearing Loss, Bilateral

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

Criteria for Inclusion

  • Participants may be of any age, based on Cohort Criteria
  • At least two mutations in the otoferlin gene
  • Clinical presentation of Profound (Part A) or Severe to Profound (Parts B, C, and D) 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

Criteria for Exclusion

  • 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 (with the exception of AK-OTOF-101 for Part D participants)

Recruiting US locations

  • University of California, San Francisco Benioff Children's Hospital

    San Francisco, CA 94158

    Site contact: Jenny Stephans · 415-353-2012 · Jihyun.Stephans@ucsf.edu

  • University of Iowa

    Iowa City, IA 52242

    Site contact: Kesten Anderson · +1 319-356-8320 · Kesten-Anderson@uiowa.edu

  • Vanderbilt Bill Wilkerson Center

    Nashville, TN 37232

    Site contact: Taha Jan, MD · +1 615-421-1228 · taha.a.jan@vumc.org

Study contacts (from the official record)

  • Lilly Clinical Trials · +1 857-410-1816 · AkouosClinicalTrials@Lilly.com
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Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss (NCT05821959) | Bored Studies