Understanding the genetics of hereditary hearing loss

Natural History of Autosomal Dominant Hearing Loss

Observational National Institutes of Health Clinical Center (CC) · NCT04501081

This study is trying to find out more about the genetics of a type of hearing loss that runs in families by looking at people with the condition and their relatives to help guide future treatments.

Quick facts

Study typeObservational
Enrollment1100 (estimated)
Ages3 Years to 99 Years
SexAll
SponsorNational Institutes of Health Clinical Center (CC) NIH
Locations1 site (Bethesda, Maryland)
Trial IDNCT04501081 on ClinicalTrials.gov

What this trial studies

This observational study aims to investigate the natural history of non-syndromic autosomal dominant hearing loss (DFNA) by examining affected individuals and their family members. Participants aged 3 to 99 will undergo medical and hearing history screenings, hearing tests, and balance assessments to gather comprehensive data. The study seeks to identify the genetic mutations responsible for DFNA and to establish baseline data that will inform future gene therapy trials. By following participants longitudinally, researchers hope to better characterize the progression of this condition.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 3 to 99 with confirmed autosomal dominant hereditary sensorineural hearing loss and their family members.

Not a fit: Patients with non-genetic causes of sensorineural hearing loss or those who have had surgical interventions related to their hearing loss may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could pave the way for gene therapy approaches that may restore natural hearing in individuals with hereditary hearing loss.

How similar studies have performed: While gene therapy for hearing loss is a novel approach, preliminary studies in animal models have shown promise, indicating potential for success in human applications.

Eligibility criteria

Show full inclusion / exclusion criteria
* 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.

Where this trial is running

Bethesda, Maryland

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Hearing LossDFNAGenome EditingNatural History
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.