Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy

April 1, 2026 updated by: Akouos, Inc.

An Evaluation of the Long Term Safety and Efficacy of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations

This multi-center, observational, post-intervention long term follow-up (LTFU) study will monitor for safety and efficacy of AAVAnc80-hOTOF in individuals with sensorineural hearing loss due to otoferlin gene mutations who have previously received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial.

Study Overview

Status

Enrolling by invitation

Conditions

Detailed Description

After completing one year of initial safety and efficacy assessments following an intracochlear administration of AAVAnc80-hOTOF in AK-OTOF-101, participants will continue to be observed for an additional approximately four years of safety and efficacy follow-up in this LTFU study, for a total of approximately five years of safety and efficacy observations after vector administration. For each participant, evaluations in the LTFU study will occur at approximately one and a half years and two years after vector administration, followed by annual visits through Year 5; the duration in this LTFU study is approximately four years, with the aim of understanding and mitigating any risks related to delayed adverse events, as well as characterizing the durability of effect of the AAVAnc80-hOTOF.

Study Type

Observational

Enrollment (Estimated)

30

Contacts and Locations

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

Study Locations

  • Spain
    • Barcelona
      • Esplugues de Llobregat, Barcelona, Spain, 08950
        • Hospital Sant Joan De Deu
  • Taiwan
    • Taiwan
      • Taipei, Taiwan, Taiwan, (R.O.C), 100
        • National Taiwan University Hospital
  • United States
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19146
        • Children's Hospital of Philadelphia

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
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial.

Description

Inclusion Criteria:

  • Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial
  • Legally authorized representative and/or participant willingness to comply with all study requirements, as evidenced by successful completion of the informed consent process or permission and participant assent process (where appropriate)

Exclusion Criteria:

  • Any condition that would not allow the potential participant to complete follow-up examinations during the course of the study and/or, in the opinion of the Investigator, makes the potential participant unsuitable for the study

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

Cohorts and Interventions

Group / Cohort
Study Participants
Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Long term safety of AAVAnc80-hOTOF, including the incidence and frequency of late-occurring adverse events (AEs)
Time Frame: Through study completion, approximately four years.

The following adverse events will be collected:

  1. The development of new or exacerbation of malignancies (oncologic), neurologic, rheumatologic or other autoimmune, hematologic disorders, or new infections (if the infection is potentially related to AAVAnc80-hOTOF
  2. AEs that are related to AAVAnc80-hOTOF and/or the administration procedure
  3. AEs of special interest
  4. All serious adverse events regardless of severity or of relationship to participation in the AK-OTOF-101 Clinical Trial
Through study completion, approximately four years.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Auditory Brainstem Response (ABR) Threshold
Time Frame: Through study completion, approximately four years.
Changes in ABR intensity threshold (decibels normal hearing level [dB nHL])
Through study completion, approximately four 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: Peter Marks, M.D., Eli Lilly and Company

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

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)

November 11, 2024

Primary Completion (Estimated)

April 1, 2033

Study Completion (Estimated)

April 1, 2033

Study Registration Dates

First Submitted

November 18, 2024

First Submitted That Met QC Criteria

November 19, 2024

First Posted (Actual)

November 20, 2024

Study Record Updates

Last Update Posted (Actual)

April 6, 2026

Last Update Submitted That Met QC Criteria

April 1, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AK-OTOF-LTFU

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

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.

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