Could gene editing one day restore hearing? scientists begin by studying families with inherited hearing loss.
NCT ID NCT04501081
First seen Jun 27, 2026 · Last updated Jul 24, 2026 · Updated 2 times
Summary
This study looks at people with a type of inherited hearing loss (called DFNA) and their family members to learn which genes cause it. Researchers will collect hearing tests, balance tests, blood samples, and skin biopsies over up to 20 years. The goal is to gather information that may help develop gene-editing treatments in the future.
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Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 1,100 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Feb 2021
- Expected to finish
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Aug 2029
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
We will be recruiting individuals with DFNA and their family members. Based on the investigator and clinic's capacity to evaluate an average of 20 subjects and 4 (80) additional family members annually, we project our study may accrue up to 200 probands and 800 family members in the first 10 years and for the duration of the study. Target enrollment is 1000, accrual ceiling is 1100 to allow for screen failures, withdrawals and dropouts)
- Ages
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3 to 99 years
- Sex
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Anyone
- Healthy volunteers
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Accepted
You do not need to have the condition being studied to take part.
Show the full entry requirements Hide 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.
* INCLUSION CRITERIA: * Affected persons with autosomal dominant hereditary sensorineural hearing loss, preferably confirmed by prior genetic testing * Affected family members of enrolled participants with known autosomal dominant hereditary hearing loss * Unaffected Family Members (Healthy Volunteers) of enrolled participant * Adults must be able to provide informed consent * Minors must have a parent or guardian able to provide informed consent * Subjects must be 3-99 years of age EXCLUSION CRITERIA: * Persons with sensorineural hearing loss (SNHL) and/or peripheral vestibular dysfunction associated with a non-genetic etiology such as infection, metabolic or immunologic disorders, or exposure to ototoxic agents such as cisplatin, or aminoglycoside antibiotics will not be included in this protocol. * Persons with sensorineural hearing loss known to be associated with surgical intervention (e.g. acoustic neuroma removal, failed stapedectomy). Prospective study subjects who are cognitively impaired and lack consent capacity, will not be enrolled. The pre-screening eligibility checklist, which will be used and documented for registration under this protocol, is provided in a separate document.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
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Other studies related to the condition(s) this trial covers.
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