Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Public ClinicalTrials.gov record NCT05821959. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 identification
- NCT ID
- NCT05821959
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 48 participants
Conditions and interventions
Conditions
Interventions
- AAVAnc80-hOTOF via Akouos Delivery Device Combination Product
- AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism Combination Product
Combination Product
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 15, 2023 (Actual)
- Primary completion
- Oct 2028 (Estimated)
- Completion
- Dec 2029 (Estimated)
- Last update posted
- Sep 29, 2026
2023 – 2029
United States locations
- U.S. sites
- 6
- U.S. states
- 6
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, San Francisco Benioff Children's Hospital | San Francisco | California | 94158 | Recruiting |
| University of Iowa | Iowa City | Iowa | 52242 | Recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Active, not recruiting |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | Active, not recruiting |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19146 | Active, not recruiting |
| Vanderbilt Bill Wilkerson Center | Nashville | Tennessee | 37232 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05821959, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 29, 2026 · Synced Oct 20, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT05821959 live on ClinicalTrials.gov.