DB-OTO

DRUG2 trials

Sponsors

Regeneron Pharmaceuticals Inc., Regeneron Pharmaceuticals

Conditions

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

Phase 1

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

RecruitingNCT05788536

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

Start: 2023-06-27End: 2032-02-25Target: 36Updated: 2026-08-05

A Phase 1/2, Open-Label, Multicenter Trial with a Single Ascending Dose Cohort with Unilateral Intracochlear Injection Followed by a Bilateral Injection Expansion Cohort to Evaluate the Safety, Tolerability, and Efficacy of DB-OTO in Children and Infants with Biallelic hOTOF Mutations

RecruitingCTIS2024-511342-40-00

Regeneron Pharmaceuticals Inc.Congenital Hearing Loss Secondary to Biallelic Pathogenic Variants of the Otoferlin Gene (OTOF)

Start: 2024-03-04Target: 15Updated: 2025-10-16

Related Papers

Inner-Ear Gene Therapy for Hereditary Hearing Loss: A PubMed-Based Scoping Review of Published Human Studies and the Clinical Trial Registry Landscape

Inquisiva Open2026-08-07

DB-OTO Gene Therapy for Inherited Deafness.

2025-10-122 citations