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

Congenital Hearing Loss Secondary to Biallelic Pathogenic Variants of the Otoferlin Gene (OTOF)

Incidence and severity of treatment-emergent adverse events, Achievement of a hearing sensitivity threshold of ≤70 dB assessed by average pure tone audiometry (PTA)

Auditory Brainstem Response (ABR) to click, Achievement of a hearing sensitivity threshold of ≤45 dB assessed by average PTA, Achievement of hearing sensitivity threshold of ≤25 dB assessed by average PTA, Speech perception scores by age-appropriate tests, Speech Awareness Threshold (SAT): achievement of a threshold of ≤70 dB, SAT: achievement of a threshold of ≤45 dB, SAT: achievement of threshold of ≤25 dB, Speech Reception Threshold (SRT): achievement of a threshold of ≤70 dB, SRT: achievement of a threshold of ≤45 dB, SRT: achievement of a threshold of ≤25 dB, Severity in speech perception ability assessed by Global Impression scales, determined by clinician, Severity in speech perception ability assessed by Global Impression scales, determined by parent/legal guardian, Change in speech perception ability assessed by Global Impression scales, determined by clinician, Change in speech perception ability assessed by Global Impression scales, determined by parent/legal guardian, Average PTA threshold in the subset of patients who achieved an average PTA threshold ≤70 dB, Average PTA threshold in the subset of patients who achieved an average PTA threshold >70 dB but ≤85 dB, Achievement of a hearing sensitivity threshold improvement of ≥10 dB from baseline, Time to an average PTA threshold ≤70 dB, Incidence of patients who regress to >70 dB after having achieved average PTA threshold ≤70 dB, Persistence of an average PTA threshold ≤70 dB

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Germany, Spain