Health Trials

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

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Review clinical trials related to Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF). Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

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)

Participants needed: 36
Trial details
Phase: Phase 1, Phase 2Biological sex: AllType: InterventionalSponsor: Regeneron PharmaceuticalsUpdated: Jul 1, 2026Locations: 16
Eligibility criteria

Willingness to provide written informed consent (by at least one parent/legal gu... [+9]

History of prior treatment with gene therapy [+6]

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