Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

February 6, 2024 updated by: Akouos, Inc.

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The following two dose levels will be tested sequentially:

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

Study Type

Interventional

Enrollment (Estimated)

14

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Taiwan
      • Taipei City, Taiwan, 100
        • Recruiting
        • National Taiwan University Hospital
        • Contact:
  • United States
    • Iowa
      • Iowa City, Iowa, United States, 52242
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19146
        • Recruiting
        • Children's Hospital of Philadelphia
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age 2 to 17 years old (inclusive) at the time of AAVAnc80-hOTOF administration
  2. Biallelic mutations in the otoferlin gene
  3. Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR
  4. Preserved distortion product otoacoustic emissions (DPOAEs)
  5. 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

Exclusion Criteria:

  1. 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
  2. Bilateral cochlear implants
  3. 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1a and Cohort 1b

Cohort 1a: Participants aged 7 to 17 years old (inclusive) to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device

Cohort 1b: Participants aged 2 to 17 years old (inclusive) at the time of AAVAnc80-hOTOF administration to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device
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.
Experimental: Cohort 2
Cohort 2: Participants aged 2 to 17 years old (inclusive) at the time of AAVAnc80-hOTOF administration to receive intracochlear administration of AAVAnc80-hOTOF (dose level 2) in the study ear using a sterile, one-time use investigational medical device
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency of Adverse Events (AEs)
Time Frame: through trial completion, approximately 2 years
AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device)
through trial completion, approximately 2 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Auditory Brainstem Response (ABR) Threshold
Time Frame: through trial completion, approximately 2 years
Changes from baseline in ABR intensity threshold (decibels normal hearing level [dB nHL])
through trial completion, approximately 2 years
Performance of the Akouos Delivery Device
Time Frame: through trial completion, approximately 2 years
Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-hOTOF to the intracochlear space
through trial completion, approximately 2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Akouos, Inc.

Collaborators

Eli Lilly and Company

Investigators

  • Study Director: Kathy Reape, MD, Akouos, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 15, 2023

Primary Completion (Estimated)

October 1, 2028

Study Completion (Estimated)

October 1, 2028

Study Registration Dates

First Submitted

February 8, 2023

First Submitted That Met QC Criteria

April 18, 2023

First Posted (Actual)

April 20, 2023

Study Record Updates

Last Update Posted (Actual)

February 8, 2024

Last Update Submitted That Met QC Criteria

February 6, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AK-OTOF-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Sensorineural Hearing Loss, Bilateral

Clinical Trials on AAVAnc80-hOTOF via Akouos Delivery Device

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Liberia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe