ClinicaliQ Trial Snapshot
- 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 I / Phase II • Rare Diseases • NCT05788536.
- Gene therapy trial testing safety and effectiveness of DB-OTO for hearing loss caused by otoferlin gene mutations in children and adults.
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What This Trial Is Studying
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…