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

NCT06696456 | Enrolling By Invitation DMC

• 1 other identifier: AK-OTOF-LTFU
• Study Type: observational
• Target: 30
• Locations: 3 countries
• Sites: 4

Brief Summary

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.

Conditions

Otoferlin Gene-mediated Hearing Loss

Keywords

Otoferlin; Deafness, autosomal recessive 9 (DFNB9); Hearing loss; Hearing loss, bilateral; Hearing loss, sensorineural; Auditory neuropathy (AN)

Outcome Measures

Primary Outcomes (1)

• Long term safety of AAVAnc80-hOTOF, including the incidence and frequency of late-occurring adverse events (AEs)

  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 Outcomes (1)

• Auditory Brainstem Response (ABR) Threshold

  Through study completion, approximately four years.

Study Arms (1)

Study Participants

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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Akouos, Inc.: lead
• Eli Lilly and Company: collaborator

Study Sites (4)

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19146, United States

Location

Hospital Sant Joan de Déu

Esplugues de Llobregat, Barcelona, 08950, Spain

Location

National Taiwan University Hospital

Taipei, Taiwan, (R.O.C), 100, Taiwan

Location

Related Links

• AK-OTOF-101: Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

MeSH Terms

Conditions

Deafness, Autosomal Recessive 9; Deafness; Hearing Loss; Hearing Loss, Bilateral; Hearing Loss, Sensorineural; Auditory neuropathy

Condition Hierarchy (Ancestors)

Hearing Disorders; Ear Diseases; Otorhinolaryngologic Diseases; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Peter Marks, M.D.

  Eli Lilly and Company

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2024
• First Posted: November 20, 2024
• Study Start: November 11, 2024
• Primary Completion (Estimated): April 1, 2033
• Study Completion (Estimated): April 1, 2033
• Last Updated: March 4, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (2); ES (1); TW (1)