At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Natural History Study (other)
- How long the study runs
- Study runs about 79 months (dates as stated)
- About the drug or intervention
- Natural History Study — other: Natural History Study
- Patient visit burden
- Not specified by the sponsor
In plain English
This is a natural history study looking at hearing loss caused by changes (mutations) in a gene called otoferlin. It focuses on people with hearing loss in both ears, including a type called auditory neuropathy. The sponsor is Akouos, Inc.
Who can take part
- Hearing loss in both ears, including a pattern called auditory neuropathy (AN) or auditory neuropathy spectrum disorder (ANSD), now or earlier in life
- A change (mutation) in the otoferlin gene
- Able and willing to follow all study requirements and give consent (or assent, where relevant)
- For the prospective phase only: certain hearing test results in at least one ear without a cochlear implant within 12 months before or at the Month 0 visit
Who may not be able to
- Unable or unwilling to follow all study requirements
- Problems with the hearing (cochlear) nerve, such as the nerve being missing or not formed properly
- For the prospective phase only: cochlear implants in both ears, or plans to get them in the next 6 months
- For the prospective phase only: a middle ear or auditory brainstem implant, or plans to get one in the next 6 months
- Any condition that would stop someone completing follow-up assessments, or that the investigator thinks makes them unsuitable
What taking part involves
- • Not stated — ask the trial team (this is a natural history study, which normally involves observations and tests rather than treatment)
Time commitment: Not stated — ask the trial team about the number of visits, how long the study lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- Up to 44 Years
- Who
- All
- Number of participants
- 150
- Started
- 2022-07-14
- Last checked
- 2025-12
Plain English Summary
What is this study?
- • Testing a new treatment for sensorineural hearing loss, bilateral
- • Clinical study - 150 participants
- • This is a retrospective and prospective longitudinal study in participants with Otoferlin Gene-Mediated Hearing Loss
Who can take part?
- • Ages Up to 44 Years
- • Diagnosed with sensorineural hearing loss, bilateral
Where?
- • London - University College London
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a retrospective and prospective longitudinal study in participants with Otoferlin Gene-Mediated Hearing Loss.
Sensorineural Hearing Loss, Bilateral
How this trial compares with your answers
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What we know so far
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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Up to 44 Years
- Who can join: All genders
What the study is looking for
- ✓Mutation(s) in the otoferlin gene
- ✓Able and willing to comply with all study requirements, as evidenced by successful completion of the informed...
- ✓Additional Criteria for Inclusion in the Prospective Phase:
- ✓Presence of OAE / CM and absent / abnormal ABRs in at least one ear (that does not have a cochlear implant) within...
Who cannot take part
- ✗Unwillingness or inability of the potential participant and/or legally authorized representative to comply with all...
- ✗Presence of cochlear nerve deficiency and/or cochlear nerve dysplasia
- ✗Additional Criteria for Exclusion from the Prospective Phase:
- ✗Presence of bilateral cochlear implants at the time of record review or planned within the next 6 months
- ✗Presence of middle ear or auditory brainstem implant(s) at the time of record review or planned within the next 6 months
See the full criteria
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
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
Where Is This Study? (1 UK site)
University College London
London NW1 2PG, United Kingdom
Hospital R&D contact (matched)
Rajinder Sidhu - Associate Director, Research Governance and Operations
uclh.jro-communications@nhs.net020 3447 9825How to Get in Touch
Akouos Clinical Trials
Sponsor contactCONTACT
+1 857 410 1816 AkouosClinicalTrials@Lilly.com
Data sourced from ClinicalTrials.gov · Last verified: 2025-12