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Gene therapy aims to restore hearing in children with rare genetic deafness

NCT ID NCT05821959

Disease control Sponsor: Akouos, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This trial tests a gene therapy called AAVAnc80-hOTOF for people with severe hearing loss caused by mutations in the otoferlin gene. The therapy is given as a single injection into the inner ear during surgery. The study will check if the treatment is safe and if it can improve hearing. About 22 participants of any age with confirmed otoferlin mutations will take part.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 22 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2023

Expected to finish

Oct 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 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

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Conditions

The condition(s) this trial relates to.

auditory neuropathy autosomal recessive nonsyndromic hearing loss 9 Deafness Deafness, Autosomal Recessive 9 hearing loss disorder Hearing Loss, Bilateral Hearing Loss, Sensorineural sensorineural hearing loss disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Sensorineural hearing loss, bilateral

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    9 sites in 4 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

    Contact Email: •••••@•••••

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19146, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • National Taiwan University Hospital

    RECRUITING

    Taipei, 100, Taiwan

  • The Hospital for Sick Children [email protected] +1 (416) 813 6683

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

  • University College London

    RECRUITING

    London, United Kingdom, NW1 2PG, United Kingdom

  • University of California, San Francisco Benioff Children's Hospital

    RECRUITING

    San Francisco, California, 94158, United States

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • Vanderbilt Bill Wilkerson Center

    RECRUITING

    Nashville, Tennessee, 37232, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.