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NCT Number: NCT05821959

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

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.

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Key information

Conditions

Sensorineural Hearing Loss, Bilateral Auditory neuropathy Deafness Deafness, Autosomal Recessive 9 Ear Diseases Hearing Disorders Hearing Loss Hearing Loss, Bilateral Hearing Loss, Sensorineural Nervous System Diseases Neurologic Manifestations Otorhinolaryngologic Diseases Pathological Conditions, Signs and Symptoms Sensation Disorders Signs and Symptoms

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

The Hospital for Sick Children [email protected] +1 (416) 813 6683, Toronto, Ontario, Canada

Loading trial locations.

About this study

The following two dose levels will be tested:

  • Up to 4.1E11 total vg/cochlea
  • Up to 8.1E11 total vg/cochlea

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Criteria for Inclusion:

  • Participants may be of any age, based on Cohort Criteria
  • At least two mutations in the otoferlin gene
  • Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR
  • Preserved distortion product otoacoustic emissions (DPOAEs)
  • Able and willing to comply with all study requirements, including willingness to participate in a separate long term follow-up study after completion of this trial

Criteria for Exclusion:

  • Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy
  • Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF
  • Prior participation in a clinical trial with an investigational drug, within six months prior to administration, or any prior participation in a gene therapy clinical trial

Treatment and study plan

AAVAnc80-hOTOF via Akouos Delivery Device

Combination Product

AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.

AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism

Combination Product

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.

Primary outcomes

  1. Frequency of Adverse Events (AEs)

    Time frame: through trial completion, approximately 1 year

    AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery system)

Secondary outcomes

  1. Auditory Brainstem Response (ABR) Threshold

    Time frame: through trial completion, approximately 1 year

    Changes from baseline in ABR intensity threshold (decibels normal hearing level [dB nHL])

  2. Performance of the Akouos Delivery Device and Precision Delivery Mechanism

    Time frame: through trial completion, approximately 1 year

    Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-hOTOF to the intracochlear space

Study contacts

Contact information is provided by the study sponsor or research team.

Akouos Clinical Trials

CONTACT

[email protected]

+1 857-410-1816

Sponsors and collaborators

Lead sponsor

Akouos, Inc.

Industry

Collaborators

  • Eli Lilly and Company

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2028
Study completion
2028
First posted
Apr 20, 2023
Registry last updated
Mar 4, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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