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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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19146, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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National Taiwan University Hospital
RECRUITINGTaipei, 100, Taiwan
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Hospital for Sick Children [email protected] +1 (416) 813 6683
RECRUITINGToronto, Ontario, M5G 1X8, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University College London
RECRUITINGLondon, United Kingdom, NW1 2PG, United Kingdom
Contact Email: •••••@•••••
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University of California, San Francisco Benioff Children's Hospital
RECRUITINGSan Francisco, California, 94158, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Vanderbilt Bill Wilkerson Center
RECRUITINGNashville, Tennessee, 37232, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••