A Phase ½, Open-Label, Multicenter Trial with a Single Ascending Dose Cohort with Unilateral Intracochlear Injection Followed by Bilateral Injection Expansion Cohort to Evaluate the Safety, Tolerability, and Efficacy of DB-OTO in Children and Infants with Biallelic hOTOF Mutations
What is the goal of the study?
This is a multicenter international phase 1/2, open-label, 2-part trial with a single-ascending dose patient cohort (Part A) and a bilateral expansion patient cohort (Part B) to evaluate the safety, tolerability, and preliminary efficacy of DB-OTO in in young children and infants diagnosed with biallelic otoferlin gene (hOTOF) mutations.
Who can participate in the study?
Please contact the study team listed below to learn more.