A trial to find the optimal dose of DB-OTO in infants and children with hearing loss (CHORD)
- Conditions
- Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)MedDRA version: 20.0Level: LLTClassification code: 10011874Term: Deaf Class: 10013993Therapeutic area: Phenomena and Processes [G] - Genetic Phenomena [G05]
- Registration Number
- CTIS2024-511342-40-00
- Lead Sponsor
- Regeneron Pharmaceuticals Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 22
Presence of pathogenic or likely pathogenic mutations in both alleles of the OTOF gene., Patient is <18 years of age and of the appropriate age to qualify for enrollment in the corresponding age cohort at the time the parent/legal guardian signs the informed consent form. Participant to provide assent, when applicable., Audiological Criteria: Infants =24 months of age: a. Absence of an ABR neural signal in response to a click stimulus =85 dB nHL in the ear(s) to be injected with DB-OTO. b. Presence of otoacoustic emissions (OAE) in the ear(s) to receive DB-OTO., Audiological Criteria: Children >24 months to <18 years of age: a. Absence of an ABR neural signal in response to a click stimulus =85 dB nHL in the ear(s) to be injected with DB-OTO. b. Presence of otoacoustic emissions (OAE) in the ear(s) to receive DB-OTO OR c. Presence of a cochlear microphonic in ears to receive DB-OTO., Willingness of at least 1 parent/legal guardian to consent to vaccinations for the patient in accordance with the country-specific pediatric immunization schedule, No clinically significant laboratory findings on clinical laboratory tests at time of Screening, No evidence that hearing loss is dependent on body temperature, From study start and for the duration of the short-term follow-up period (18 months): Female patients of childbearing potential and fertile males, must agree to use highly effective contraception. Female patients must agree not to become pregnant. Fertile male patients must agree not to father a child or donate sperm., Additional Protocol defined inclusion criteria may apply.
History or presence of other permanent or untreatable hearing loss conditions., Prior or current cochlear implants in the ear(s) to be injected with DB-OTO, History of risk factor(s) for auditory neuropathy not caused by OTOF mutations including: prematurity, low birth weight, hyperbilirubinemia, neonatal intensive care unit (NICU) admission, and/or low Apgar scores., Prior or current history of malignancies., Prior or current history of meningitis., History of prior treatment with gene therapy., Surgical anatomy that would preclude the planned surgical approach as indicated by medical imaging (eg, computed tomography [CT] or magnetic resonance imaging [MRI]) in the ear(s) to be injected with DB-OTO., Additional Protocol defined exclusion criteria may apply.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the safety and tolerability of DB-OTO in children and infants diagnosed with biallelic otoferlin gene (hOTOF) mutations;Secondary Objective: To evaluate preliminary efficacy of DB-OTO in children and infants diagnosed with biallelic hOTOF mutations;Primary end point(s): Incidence and severity of treatment-emergent systemic and local adverse events
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Auditory brainstem response (ABR) – change in intensity threshold (decibels Hearing Level [dB nHL]) across frequency domains.;Secondary end point(s):Behavioral audiometry with pure-tone audiometry – change in intensity thresholds (dB HL) in treated ear across frequency domains, and speech awareness threshold (SAT) and speech reception threshold (SRT)– change in threshold in treated ear.