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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05572073
Registration number
NCT05572073
Ethics application status
Date submitted
5/10/2022
Date registered
7/10/2022
Date last updated
13/02/2024
Titles & IDs
Public title
Otoferlin Gene-mediated Hearing Loss Natural History Study
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Scientific title
A Natural History Study in Individuals With Otoferlin Gene-mediated Hearing Loss
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Secondary ID [1]
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AK-OTOF-NHS-002
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Sensorineural Hearing Loss, Bilateral
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Condition category
Condition code
Ear
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Deafness
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Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Other interventions - Natural History Study
Retrospective -
Prospective -
Other interventions: Natural History Study
Natural History Study
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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ABR
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Assessment method [1]
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Auditory Brainstem Response
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Timepoint [1]
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First audiologic data in participant medical record through five-year prospective follow up
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Secondary outcome [1]
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OAE
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Assessment method [1]
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Otoacoustic Emissions
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Timepoint [1]
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First audiologic data in participant medical record through the five-year prospective follow up
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Eligibility
Key inclusion criteria
1. Clinical presentation of bilateral sensorineural hearing loss (SNHL), including
auditory neuropathy (AN) / auditory neuropathy spectrum disorder (ANSD) phenotype or
medical history of AN / ANSD phenotype earlier in life
2. Mutation(s) in the otoferlin gene
3. Able and willing to comply with all study requirements, as evidenced by successful
completion of the informed consent (and assent, if applicable) process
Additional Criteria for Inclusion in the Prospective Phase:
4. Presence of OAE / CM and absent / abnormal ABRs in at least one ear (that does not
have a cochlear implant) within 12 months prior to or at the Month 0 visit
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Minimum age
No limit
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Maximum age
44
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Unwillingness or inability of the potential participant and/or legally authorized
representative to comply with all protocol requirements
2. Presence of cochlear nerve deficiency and/or cochlear nerve dysplasia
Additional Criteria for Exclusion from the Prospective Phase:
3. Presence of bilateral cochlear implants at the time of record review or planned within
the next 6 months
4. Presence of middle ear or auditory brainstem implant(s) at the time of record review
or planned within the next 6 months
5. Any condition that would not allow the potential participant to complete follow-up
assessments during the course of the study and/or, in the opinion of the Investigator,
makes the potential participant unsuitable for the study
Note: Potential participants will not be excluded based on their sex, gender, race, or
ethnicity
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Study design
Purpose
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Duration
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Selection
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Timing
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
14/07/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/02/2029
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Actual
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Sample size
Target
150
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Murdoch Children's Research Institute - Parkville
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Recruitment postcode(s) [1]
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VIC 3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Iowa
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Country [2]
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United States of America
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State/province [2]
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Ohio
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Country [3]
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United States of America
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State/province [3]
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Pennsylvania
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Country [4]
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United States of America
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State/province [4]
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Tennessee
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Country [5]
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Germany
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State/province [5]
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Tübingen
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Country [6]
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Spain
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State/province [6]
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Barcelona
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Country [7]
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Taiwan
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State/province [7]
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Taipei
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Country [8]
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United Kingdom
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State/province [8]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Akouos, Inc.
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Address
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Country
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Other collaborator category [1]
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Commercial sector/Industry
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Name [1]
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Eli Lilly and Company
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Address [1]
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Country [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a retrospective and prospective longitudinal study in participants with Otoferlin
Gene-Mediated Hearing Loss.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05572073
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Kathy Reape, MD
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Address
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Akouos, Inc.
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Akouos Clinical Trials
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Address
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Country
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Phone
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(1) 8574101816
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05572073
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