Under construction – not a live service
Sign in
Taking people in JapanPhase 1/2

DB-OTO gene therapy for hearing loss caused by otoferlin gene changes

Tests DB-OTO gene therapy in people with hearing loss from otoferlin mutations.

Key facts

Who runs it
Regeneron Pharmaceuticals
Where
Matsumoto-shi, JapanSee the siteAlso in 4 other countries, 15 sites
Registry title
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations
Status
Recruiting
Phase
Phase 1/2
People taking part
36 people, planned
Started
27 Jun 2023
Expected to end
25 Feb 2032
Type of study
Interventional, 3 groups
Treatments tested
  • DB-OTO
Record updated
5 August 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for children, infants, teens and adults with profound hearing loss caused by changes in both copies of the otoferlin (OTOF) gene. They get the gene therapy DB-OTO injected into the inner ear.

Age
The record does not give an age range. It names children, infants, adolescents and adults.
Sex
All sexes can join.
Disease stage
Profound sensorineural (inner ear) hearing loss, >90 dB HL, shown on hearing tests including ABR. Outer hair cells in the ear to be treated must still work, shown by otoacoustic emissions (≥6 dBSNR) at ≥3 frequencies from 1 to 8 kHz. For people >24 months of age, a cochlear microphonic test can show this instead. Hearing loss must not change with body temperature.
Earlier treatment
People who had gene therapy before cannot join. People with a cochlear implant now or in the past in the ear to be treated cannot join.
Biomarker or test result
Changes in both copies of the OTOF gene that are pathogenic or likely pathogenic (disease-causing). A genetic test of hearing loss genes is needed.

Main reasons for not taking part

  • Prior gene therapy
  • Ear anatomy on CT or MRI that would block or strongly affect the planned surgery
  • Other permanent or untreatable hearing loss conditions
  • Cancer now or in the past, or meningitis now or in the past
  • Risk factors for other auditory nerve problems, such as being born early, low birth weight, high bilirubin (jaundice), newborn intensive care stay or low Apgar scores

The registry criteria, word for word

Show all 16 criteriaShow less

Who can take part

  • 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
  • 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
  • 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
  • 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)
  • Presence of biallelic, likely pathogenic or pathogenic OTOF variants
  • No clinically significant laboratory findings on clinical laboratory tests at time of Screening as described in the protocol
  • Audiological Criteria:; Investigator diagnoses the participant with profound sensorineural hearing loss (SNHL; >90 dB HL) based on behavioral and physiologic measurements (ABR) of inner ear function; 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
  • No evidence from measures of hearing loss that show a dependence on body temperature
  • 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

Who cannot take part

  • History of prior treatment with gene therapy
  • 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
  • Prior or current history of meningitis
  • History or presence of cochlear implants in the ear(s) to be infused with DB-OTO
  • History or presence of other permanent or untreatable hearing loss conditions
  • Prior or current history of malignancies
  • 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

Where in Japan

Also in 4 other countries, 15 sites

  • Shinshu University HospitalMatsumoto-shi, JapanContact: Yutaka TakumiTaking people

About the trial

What the trial wants to find out

Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult participants who have severe-to profound and profound hearing loss due to changes in the otoferlin gene.

Read full original descriptionShow less

The purpose of this study is to:

* Learn about the safety of DB-OTO * Determine how well DB-OTO is tolerated (does not cause ongoing discomfort) * Evaluate the efficacy of DB-OTO (how well DB-OTO works)

The 3 groups in the trial

  • DB-OTO - Part ATreatment being tested

    Unilateral intracochlear dosing

    Treatment: DB-OTO

  • DB-OTO - Part BTreatment being tested

    Bilateral intracochlear dosing using the dose selected based on safety and efficacy data from Part A

    Treatment: DB-OTO

  • DB-OTO - Part CTreatment being tested

    Unilateral or bilateral intracochlear dosing

    Treatment: DB-OTO

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
A Phase 1/2, Open-Label, Multicenter Trial With a Single Ascending Dose Cohort With Unilateral Intracochlear Injection Followed by a Bilateral Injection Expansion Cohort to Evaluate the Safety, Tolerability, and Efficacy of DB-OTO in Children and Infants With Biallelic hOTOF Mutations
Plain-language title
DB-OTO gene therapy for hearing loss caused by otoferlin gene changesGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 14 November 2026.
Record updated
5 August 2026