Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) Clinical Trial
Official title:
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 is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat children who have 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)
Former Sponsor Decibel Therapeutics ;