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

NCT ID NCT05821959

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.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19146, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • National Taiwan University Hospital

    RECRUITING

    Taipei, 100, Taiwan

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University College London

    RECRUITING

    London, United Kingdom, NW1 2PG, United Kingdom

    Contact Email: •••••@•••••

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

    RECRUITING

    San Francisco, California, 94158, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Vanderbilt Bill Wilkerson Center

    RECRUITING

    Nashville, Tennessee, 37232, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••