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Clinical Trials in Taiwan / NCT05821959
Recruiting Phase 3

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

NCT05821959 · tracked via the Priya Life Science Taiwan tracker
Sponsor
Eli Lilly and Company
Phase
Phase 3
Started
2023-09-15
Last updated
2026-09-29

Condition(s) studied

Sensorineural Hearing Loss, Bilateral

Investigational drug(s) / intervention(s)

AAVAnc80-hOTOF via Akouos Delivery DeviceAAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism

AAVAnc80-hOTOF via Akouos Delivery Device: 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: 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.

Study summary

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.

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
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)

Primary outcome measure(s)

  • Part A: Frequency of Adverse Events (AEs) — through trial completion, approximately 1 year
    AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery system)
  • Co-Primary Parts B, C, and D: ABR Threshold — through trial completion, approximately 1 year
    Changes from baseline in ABR intensity threshold (decibels normal hearing level \[dB nHL\]
  • Co-Primary Parts B, C, and D: Pure-tone Average — through trial completion, approximately 1 year
    Changes from baseline in the average of behavioral pure-tone audiometry thresholds

Trial sites (9)

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

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Other trials for the same condition

Otoferlin Gene-mediated Hearing Loss Natural History Study Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05821959 on ClinicalTrials.gov ↗ ← All trials in Taiwan