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
Regeneron Pharmaceuticals
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)
Description
Former Sponsor Decibel Therapeutics
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Willingness to provide written informed consent (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) and willingness to comply with trial protocol 2. Willingness to consent to genetic testing for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) in order to evaluate a panel of hearing loss-related genes 3. Willingness to consent to vaccinations for th…
Interventions
- GeneticDB-OTO
Administered per the protocol
Locations (16)
- University of California Los Angeles Medical CenterLos Angeles, California
- Rady Children's HospitalSan Diego, California
- Nemours Children s ClinicJacksonville, Florida
- Nemours Childrens HospitalOrlando, Florida
- Boston Children's Hospital - MainBoston, Massachusetts
- Columbia University Irving Medical CenterNew York, New York