Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Key facts
- Where
- Boston, Cincinnati, Iowa City, Nashville, Philadelphia, and San Francisco, United StatesSee the 6 sites
- Phase
- Phase 3
Where in United States
- Boston Children's HospitalBoston, United StatesContact: Lilly Clinical TrialsNo longer taking part
- Cincinnati Children's Hospital Medical CenterCincinnati, United StatesContact: Lilly Clinical TrialsNo longer taking part
- University of IowaIowa City, United StatesContact: Kesten AndersonTaking people
- Vanderbilt Bill Wilkerson CenterNashville, United StatesContact: Taha Jan, MDTaking people
- Children's Hospital of PhiladelphiaPhiladelphia, United StatesContact: Lilly Clinical TrialsNo longer taking part
- University of California San Francisco-Benioff Children's HospitalSan Francisco, United StatesContact: Jenny StephansTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial 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).