Actively Recruiting

Age: 0 - 44Years
All Genders
NCT05572073

Otoferlin Gene-mediated Hearing Loss Natural History Study

Led by Akouos, Inc. · Updated on 2026-03-04

150

Participants Needed

10

Research Sites

342 weeks

Total Duration

On this page

Sponsors

A

Akouos, Inc.

Lead Sponsor

E

Eli Lilly and Company

Collaborating Sponsor

AI-Summary

What this Trial Is About

This is a retrospective and prospective longitudinal study in participants with Otoferlin Gene-Mediated Hearing Loss.

CONDITIONS

Official Title

Otoferlin Gene-mediated Hearing Loss Natural History Study

Who Can Participate

Age: 0 - 44Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Clinical presentation of bilateral sensorineural hearing loss, including auditory neuropathy spectrum disorder phenotype or history
  • Mutation(s) in the otoferlin gene
  • Able and willing to comply with all study requirements and complete informed consent
  • For prospective phase: presence of otoacoustic emissions or cochlear microphonics and absent or abnormal auditory brainstem responses in at least one ear without cochlear implant within 12 months prior to or at Month 0 visit
Not Eligible

You will not qualify if you...

  • Unwillingness or inability to comply with all protocol requirements
  • Presence of cochlear nerve deficiency or cochlear nerve dysplasia
  • For prospective phase: bilateral cochlear implants at record review or planned within 6 months
  • For prospective phase: presence of middle ear or auditory brainstem implants at record review or planned within 6 months
  • Any condition preventing completion of follow-up assessments or making participant unsuitable in investigator's opinion

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 10 locations

1

University of Iowa

Iowa City, Iowa, United States, 52242

Actively Recruiting

2

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Actively Recruiting

3

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

4

Vanderbilt University Medical Center

Nashville, Tennessee, United States, 37232-8605

Actively Recruiting

5

Murdoch Children's Research Institute

Parkville, Victoria, Australia, VIC 3052

Withdrawn

6

University Hospital in Tübingen

Tübingen, Germany, 72076

Actively Recruiting

7

Sant Joan de Déu Barcelona Hospital

Esplugues de Llobregat, Barcelona, Spain, 08950

Actively Recruiting

8

National Taiwan University Hospital

Taipei, Taiwan, 100

Actively Recruiting

9

Ankara University

Ankara, Turkey, Turkey (Türkiye), 06230

Actively Recruiting

10

University College London

London, United Kingdom, United Kingdom, NW1 2PG

Actively Recruiting

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Research Team

A

Akouos Clinical Trials

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

2

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Otoferlin Gene-mediated Hearing Loss Natural History Study | DecenTrialz