Trial Profile

A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 11 Jun 2025

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At a glance

Drugs AK OTOF (Primary)
Indications Sensorineural hearing loss
Focus Adverse reactions; First in man
Sponsors Akouos

Most Recent Events

30 May 2024 The protocol has been amended to change in study time frame
23 Jan 2024 According to an Eli Lilly and Company media release, first participant to receive AK-OTOF in the study, an 11-year-old at the time of AK-OTOF administration with profound hearing loss from birth, experienced restored hearing within 30 days of AK-OTOF administration.
23 Jan 2024 According to an Eli Lilly and Company media release, John Germiller, M.D., Ph.D., Director of Clinical Research in the Division of Otolaryngology at Children's Hospital of Philadelphia, is the principal investigator of this clinical trial.

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