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Gene therapy aims to restore hearing in children born deaf

NCT ID NCT05788536

Cure Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 06, 2026 · Updated 4 times

Summary

This study tests a new gene therapy called DB-OTO for children with hearing loss caused by changes in the otoferlin gene. The treatment is injected into the inner ear to help restore hearing. Up to 30 children and infants will take part to check safety and how well it works.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 36 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2023

Expected to finish

Feb 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show 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.

Key Inclusion Criteria: 1. Willingness to provide written informed consent (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) and willingness to comply with trial protocol 2. Willingness to consent to genetic testing for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) in order to evaluate a panel of hearing loss-related genes 3. Willingness to consent to vaccinations for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) in accordance with the country-specific, age-appropriate immunization schedule, as described in the protocol 4. Participant able to perform all necessary assessments to qualify for enrollment and dosing in the corresponding cohort at the time the participant or parent/legal guardian signing the informed consent form (and participant providing assent, when applicable) 5. Presence of biallelic, likely pathogenic or pathogenic OTOF variants 6. No clinically significant laboratory findings on clinical laboratory tests at time of Screening as described in the protocol 7. Audiological Criteria: 1. Investigator diagnoses the participant with profound sensorineural hearing loss (SNHL; \>90 dB HL) based on behavioral and physiologic measurements (ABR) of inner ear function 2. Outer hair cell presence is confirmed via presence of otoacoustic emissions (≥6 dBSNR) at ≥3 frequencies from 1 to 8 kHz in the ear(s) to be infused with DB-OTO. Alternatively, for participants \>24 months of age, outer hair cell presence can be confirmed via presence of the cochlear microphonic in the ear(s) to be infused with DB-OTO. 8. No evidence from measures of hearing loss that show a dependence on body temperature 9. From study start and for the duration of the short-term follow-up period (48 weeks): Female participants of childbearing potential and fertile males, must agree to use highly effective contraception. Female participants must agree not to become pregnant. Fertile male participants must agree not to father a child or donate sperm, for 48 weeks and in cases of early withdrawal, for at least 12 months after DB-OTO administration. Key Exclusion Criteria: 1. History of prior treatment with gene therapy 2. Surgical anatomy that would preclude or meaningfully impact the planned surgical approach as indicated by medical imaging (eg, Computed Tomography \[CT\] or Magnetic Resonance Imaging \[MRI\]) in the ear(s) to be infused with DB-OTO 3. History or presence of other permanent or untreatable hearing loss conditions 4. Prior or current history of malignancies 5. Prior or current history of meningitis 6. History or presence of cochlear implants in the ear(s) to be infused with DB-OTO 7. History of risk factor(s) for auditory neuropathy not caused by OTOF pathogenic variants including but not limited to: prematurity, low birth weight, hyperbilirubinemia, Neonatal Intensive Care Unit (NICU) admission, and/or low Apgar scores as described in the protocol Note: Other protocol-defined inclusion/exclusion criteria apply

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Conditions

The condition(s) this trial relates to.

auditory neuropathy autosomal recessive nonsyndromic hearing loss 9 Deafness, Autosomal Recessive 9 hearing disorder hearing loss disorder Hearing Loss, Sensorineural

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Congenital hearing loss secondary to biallelic mutations of the otoferlin gene (OTOF)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    16 sites in 5 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • Addenbrooke's Hospital, Cambridge University Hospitals NHS FT

    RECRUITING

    Cambridge, CB2 0QQ, United Kingdom

  • Boston Children's Hospital - Main

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Clinica Universidad de Navarra- Pamplona

    RECRUITING

    Pamplona, Navarre, 31008, Spain

  • Columbia University Irving Medical Center

    RECRUITING

    New York, New York, 10032, United States

  • Great Ormond Street Hospital For Children NHS Foundation Trust

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hospital Sant Joan de Deu

    RECRUITING

    Barcelona, 08950, Spain

  • Hospital Universitario Materno Infantil en las Palmas de Gran Canaria

    WITHDRAWN

    Las Palmas de Gran Canaria, 35016, Spain

  • Medical College of Wisconsin

    RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • Nemours Children s Clinic

    RECRUITING

    Jacksonville, Florida, 32207, United States

  • Nemours Childrens Hospital

    RECRUITING

    Orlando, Florida, 32827, United States

  • Rady Children's Hospital

    RECRUITING

    San Diego, California, 92123, United States

  • Ramon y Cajal University Hospital

    RECRUITING

    Madrid, 28034, Spain

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • Shinshu University Hospital

    RECRUITING

    Matsumoto-shi, Nagano, 390-8621, Japan

  • University Hospital Tubingen

    RECRUITING

    Tübingen, 72076, Germany

  • University of California Los Angeles Medical Center

    RECRUITING

    Los Angeles, California, 90024, United States