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:
* 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)
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)
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
History or presence of other permanent or untreatable hearing loss conditions
Prior or current history of malignancies
Sequential
DB-OTO
GeneticAdministered per the protocol
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
Achievement of a hearing sensitivity threshold of ≤70 dB assessed by average Pure Tone Audiometry (PTA)
Auditory Brainstem Response (ABR) to click stimulus at ≤90 dB normalized Hearing Level (nHL)
Achievement of hearing sensitivity threshold of ≤45 dB assessed by average PTA
Achievement of hearing sensitivity threshold of ≤25 dB assessed by average PTA
Achievement of a score ≥3 on the Early Speech Perception (ESP) test
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