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First received: February 08, 2023

Clinical Trial: Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Study Status: Completed

Recruit Status: RECRUITING

Condition: Sensorineural Hearing Loss, Bilateral

Study Type: INTERVENTIONAL


Official Title: A Phase 1/2 Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Biallelic Otoferlin Gene Mutations

Brief Summary: Part A of this trial will evaluate the safety and tolerability of a single unilateral administration of one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos delivery device to safely achieve the intended product performance.

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