Actively Recruiting
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Led by Akouos, Inc. · Updated on 2026-03-04
22
Participants Needed
9
Research Sites
263 weeks
Total Duration
On this page
Sponsors
A
Akouos, Inc.
Lead Sponsor
E
Eli Lilly and Company
Collaborating Sponsor
AI-Summary
What this Trial Is About
Part A of this trial will evaluate 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 and will evaluate the Akouos Delivery Device, together with the Precision Delivery Mechanism, to safely achieve the intended product performance.
CONDITIONS
Official Title
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants may be of any age, based on Cohort Criteria
- At least two mutations in the otoferlin gene
- Clinical presentation of profound 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
You will not qualify if you...
- 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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 9 locations
1
University of California, San Francisco Benioff Children's Hospital
San Francisco, California, United States, 94158
Actively Recruiting
2
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
3
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
5
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19146
Actively Recruiting
6
Vanderbilt Bill Wilkerson Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
7
The Hospital for Sick Children Jaina.negandhi@sickkids.ca +1 (416) 813 6683
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
8
National Taiwan University Hospital
Taipei, Taiwan, 100
Actively Recruiting
9
University College London
London, United Kingdom, United Kingdom, NW1 2PG
Actively Recruiting
Research Team
A
Akouos Clinical Trials
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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