MedTrace Logo

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

NCT05788536 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2026-08-05

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 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)

Conditions

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

Interventions

GENETIC

DB-OTO

Administered per the protocol

Sponsors & Collaborators

Principal Investigators

  • Clinical Trial Management · Regeneron Pharmaceuticals

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-27
Primary Completion
2029-02-26
Completion
2032-02-25
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