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
Recruiting · Phase 1, Phase 2 · NCT05788536
Conditions: Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
At a glance
- Research payments reported, 2025
- $657,795
- U.S. investigators named
- 5
- Sites paid
- 4
- Planned or actual enrollment
- 36
About the trial
- Sponsor
- Regeneron Pharmaceuticals
- Tested
- DB-OTO (genetic)
- Dates
- Started 2023-06-27; completion 2032-02-25
- Results posted
- Not yet
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)
From ClinicalTrials.gov, last updated 2026-08-05. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Regeneron Pharmaceuticals, Inc. reported $657,795 in research payments for this study in 2025 (152 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Evie C. Landry, MD, FRCSC ENT (Otolaryngology) · Washington, DC
- Cedric V. Pritchett, MD ENT (Otolaryngology) · Orlando, FL
- Lawrence R. Lustig, MD ENT (Otolaryngology) · New York, NY
- John H. Greinwald JR., M.D. ENT (Otolaryngology) · Cincinnati, OH
- Jay T. Rubinstein ENT (Otolaryngology) · Seattle, WA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Cincinnati Children's Hospital Medical Center, OH $411,908
- Trustees of Columbia University in The City of New York $174,238
- Nemours Childrens Hospital, Florida, FL $32,544
- Seattle Children's Hospital, WA $23,306
- $15,799
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.