Actively Recruiting
A Trial of AAVAnc80-hOTOF Gene Therapy for Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
Led by Akouos, Inc. · Updated on 2026-03-04
22
Participants Needed
9
Research Sites
N/A
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
Researchers are evaluating a gene therapy called AAVAnc80-hOTOF for individuals with sensorineural hearing loss caused by mutations in the otoferlin gene. This trial will assess the safety and tolerability of a single surgical administration procedure using the Akouos Delivery Device and Precision Delivery Mechanism. The study aims to safely deliver the gene therapy to one or both ears and monitor the effects over approximately one year. The trial includes several groups receiving different doses of AAVAnc80-hOTOF administered directly into the cochlea using a sterile, one-time use investigational device. Dose levels tested are up to 4.1E11 and 8.1E11 total viral genomes per cochlea. Participants may receive treatment in one ear or both ears simultaneously using the delivery devices. The study involves cohorts based on age and dosing, including a bilateral expansion group. Participants will undergo evaluations throughout the trial, including monitoring for adverse events and hearing function via auditory brainstem response tests. The performance of the delivery devices will also be assessed. Participants must be able and willing to comply with all study visits and procedures, including a separate long-term follow-up study after the trial. The total participation time includes approximately one year of safety and efficacy monitoring.
CONDITIONS
Brief 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 confirmed by auditory brainstem response testing
- Preserved distortion product otoacoustic emissions (DPOAEs)
- Able and willing to comply with all study requirements, including participation in a long term follow-up study after trial completion
You will not qualify if you...
- Persistent ear infections, ear abnormalities, or medical conditions that contraindicate surgery, anesthesia, or gene therapy administration
- Presence of cochlear implant(s) in the ear(s) to receive the gene therapy
- Prior participation in a clinical trial with an investigational drug within six months or any prior gene therapy trial
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day
Participants receive a one-time intracochlear administration of AAVAnc80-hOTOF gene therapy using investigational delivery devices.
1 administration visit (in-person)
Duration - Approximately 1 year
Participants are monitored for safety, auditory function, and device performance for approximately 1 year after gene therapy administration.
Regular visits during follow-up period
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
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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