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 now · Phase 1/Phase 2
Conditions studied: Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
In brief
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)
Key facts
- Study ID
- NCT05788536
- Run by
- Regeneron Pharmaceuticals
- People needed
- 36
- Starts
- 2023-06-27
- Expected to finish
- 2032-02-25
- Last updated by the study team
- 2026-08-05
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Great Ormond Street Hospital For Children NHS Foundation Trust — London, United Kingdom (enrolling)
- Rady Children's Hospital — San Diego, California, United States (enrolling)
- Nemours Children s Clinic — Jacksonville, Florida, United States (enrolling)
- Nemours Childrens Hospital — Orlando, Florida, United States (enrolling)
- Boston Children's Hospital - Main — Boston, Massachusetts, United States (enrolling)
- Columbia University Irving Medical Center — New York, New York, United States (enrolling)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
- Seattle Children's Hospital — Seattle, Washington, United States (enrolling)
- Medical College of Wisconsin — Milwaukee, Wisconsin, United States (enrolling)
- University Hospital Tubingen — Tübingen, Germany (enrolling)
- Shinshu University Hospital — Matsumoto-shi, Nagano, Japan (enrolling)
- Clinica Universidad de Navarra- Pamplona — Pamplona, Navarre, Spain (enrolling)
- Hospital Sant Joan de Deu — Barcelona, Spain (enrolling)
- University of California Los Angeles Medical Center — Los Angeles, California, United States (enrolling)
- Ramon y Cajal University Hospital — Madrid, Spain (enrolling)
- Addenbrooke's Hospital, Cambridge University Hospitals NHS FT — Cambridge, United Kingdom (enrolling)
- Hospital Universitario Materno Infantil en las Palmas de Gran Canaria — Las Palmas de Gran Canaria, Spain
Full record on ClinicalTrials.gov
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