NCT05821959

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 22 (estimated)
• Sponsor: Akouos, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

Part A of this trial will evaluate the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos Delivery Device, together with the Precision Delivery Mechanism, to safely achieve the intended product performance.

Detailed description

The following two dose levels will be tested: * Up to 4.1E11 total vg/cochlea * Up to 8.1E11 total vg/cochlea

Conditions

• Sensorineural Hearing Loss, Bilateral

Interventions

Type	Name	Description
COMBINATION_PRODUCT	AAVAnc80-hOTOF via Akouos Delivery Device	AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
COMBINATION_PRODUCT	AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism	AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time bilateral intracochlear administration using the Akouos Delivery Device together with the Precision Delivery Mechanism.

Timeline

• Start date: 2023-09-15
• Primary completion: 2028-10-01
• Completion: 2028-10-01
• First posted: 2023-04-20
• Last updated: 2026-03-04

Locations

9 sites across 4 countries: United States, Canada, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study
• FDA-regulated device study