A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

Study Summary

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 children who have 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)

Conditions

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

Interventions / Drugs

DB-OTO DB-OTO

View on ClinicalTrials.gov ↗

Sponsor

REGN

Regeneron Pharmaceuticals

Antibody Therapeutics

F34/100

Study Details

Enrollment30 participants

Primary Completion—

Lead SponsorRegeneron Pharmaceuticals

Data Retrieved2026-04-11 02:55:54.866020+00:00

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