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Taking peoplePhase 3

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Key facts

Where
Toronto, CanadaSee the site
Phase
Phase 3

Where in Canada

  • Toronto, OntarioHospital not named in the registryContact: Lilly Clinical TrialsNo longer taking part

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

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Trial details

Who runs it
Eli Lilly and Company
Trial code
NCT05821959EU 2026-525943-33-00
Record updated
29 September 2026
Original 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.Read full original descriptionShow less

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

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.

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).

ClinicalTrials.gov NCT05821959 · EU CTIS 2026-525943-33-00