A Phase 1/2 interventional study of DB-OTO in Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF), sponsored by Regeneron Pharmaceuticals. Recruiting at 17 sites in 5 countries. Per ClinicalTrials.gov, last updated 2026-08-05.
Sponsored by Regeneron Pharmaceuticals · Phase 1/2, Interventional, and Treatment
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.
The purpose of this study is to:
Former Sponsor Decibel Therapeutics
346 studies on the registry are indexed under Hearing Loss, Sensorineural; 100 are open to participants now.
This study's planned enrollment of 36 is above the median of 32 across 247 interventional studies indexed under Hearing Loss, Sensorineural.
Browse Hearing Loss, Sensorineural studies →Regeneron Pharmaceuticals is the lead sponsor of 411 studies on the registry; 90 are open to participants now.
Of its 125 completed or terminated interventional studies of FDA-regulated products, 79 (63%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Audiological Criteria:
Key Exclusion Criteria:
Note: Other protocol-defined inclusion/exclusion criteria apply
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Unilateral intracochlear dosing
Genetic: DB-OTO
Bilateral intracochlear dosing using the dose selected based on safety and efficacy data from Part A
Genetic: DB-OTO
Unilateral or bilateral intracochlear dosing
Genetic: DB-OTO
Administered per the protocol
Also known as: Otarmeni™, Otoferlin
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to week 104
Achievement of a hearing sensitivity threshold of ≤70 dB assessed by average Pure Tone Audiometry (PTA)
Time frame: Up to week 104
Auditory Brainstem Response (ABR) to click stimulus at ≤90 dB normalized Hearing Level (nHL)
Time frame: Up to week 48
Achievement of hearing sensitivity threshold of ≤45 dB assessed by average PTA
Time frame: Up to week 104
Achievement of hearing sensitivity threshold of ≤25 dB assessed by average PTA
Time frame: Up to week 104
Achievement of a score ≥3 on the Early Speech Perception (ESP) test
Time frame: At week 104
Achievement of a score of 4 on the ESP test
Time frame: At week 104
Speech perception scores by age-appropriate tests
Time frame: Up to week 104
Speech Awareness Threshold (SAT): achievement of a threshold of ≤70 dB
Time frame: Up to week 48
SAT: achievement of a threshold of ≤45 dB
Time frame: Up to week 48
SAT: achievement of threshold of ≤25 dB
Time frame: Up to week 48
Severity in speech perception ability assessed by Global Impression scales (clinician and parent/legal guardian)
Time frame: At week 104
Change in speech perception ability assessed by Global Impression scales (clinician and parent/legal guardian)
Time frame: At week 104
Average PTA threshold in the subset of participants who achieved an average PTA threshold ≤70 dB
Time frame: Up to week 104
Average PTA threshold in the subset of participants who achieved an average PTA threshold >70 dB but ≤85 dB
Time frame: Up to week 48
Achievement of a hearing sensitivity threshold improvement of ≤85 dB HL, as assessed by PTA
Time frame: Up to week 48
Achievement of a hearing sensitivity threshold improvement of ≥10 dB from baseline
Time frame: Up to week 48
Time to an average PTA threshold ≤70 dB
Time frame: Up to week 104
Persistence of an average PTA threshold ≤70 dB
Time frame: Up to week 104
Incidence of participants who regress to >70 dB after having achieved average PTA threshold
Time frame: Up to week 104
Presence of ABR to click at ≤90 dB nHL
Time frame: At week 104
Change from baseline on LittlEARS®
Time frame: At week 104
Change in scores on LittlEARS®
Time frame: At week 104
Change from baseline on Auditory Skills Checklist
Time frame: At week 104
Change in scores on Auditory Skills Checklist
Time frame: At week 104
Change from baseline on MacArthur-Bates Communicative Development Inventories (MB-CDI)
Time frame: At week 104
Change in scores on MB-CDI
Time frame: At week 104
Clinical assessments of speech production: articulation
Time frame: At week 104
Clinical assessments of speech production: speech intelligibility
Time frame: At week 104
Not enrolling at this site.
Plan to share: Yes — All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report, Analytic code
Time frame: When Regeneron has: received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry) the legal authority to share the data, and ensured the ability to protect participant privacy.
Access criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
Data sharing details ↗ · vivli.org
Started Jun 2023 (actual). Primary completion is estimated for Feb 2029, 67 months after the start.
How these figures are worked out, and which studies are left out of them.
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