Clinical Trial: A Study of DB-OTO, an AAV Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
Study Status: Completed
Recruit Status: RECRUITING
Condition: Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Study Type: INTERVENTIONAL
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, T
Brief Summary:
This is a first-in-human, multicenter, 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, an AAV based gene therapy in pediatric patients with biallelic OTOF mutations
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