Cure My Disease

Don't miss your cure!

 Log in / Sign up

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

NCT ID NCT05821959

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

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for SENSORINEURAL HEARING LOSS DUE TO OTOFERLIN GENE MUTATIONS are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

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.