MedTrace Logo

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

NCT05788536 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2026-05-05

No results posted yet for this 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

GENETIC

DB-OTO

DB-OTO will be administered as a single intracochlear injection into one ear (Part A). * LD Cohort (lower dose) * HD Cohort (high dose) - not implemented

GENETIC

DB-OTO

DB-OTO will be administered as a single intracochlear injection into both ears (Part B). For bilateral injections (Part B), patients will receive DB-OTO in 1 surgical session.

Sponsors & Collaborators

Principal Investigators

  • Clinical Trial Management · Regeneron Pharmaceuticals

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-27
Primary Completion
2032-02-28
Completion
2032-02-28
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Japan
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05788536 on ClinicalTrials.gov