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

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

Status

Enrolling by invitation

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06696456

Enrollment

Registered

2024-11-20

Start date

2024-11-11

Completion date

2033-04-01

Last updated

2026-04-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Otoferlin Gene-mediated Hearing Loss

Keywords

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

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.

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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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

Design outcomes

Primary

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

Secondary

Measure	Time frame	Description
Auditory Brainstem Response (ABR) Threshold	Through study completion, approximately four years.	Changes in ABR intensity threshold (decibels normal hearing level \[dB nHL\])

Countries

Spain, Taiwan, United States

Contacts

STUDY_DIRECTORPeter Marks, M.D.

Eli Lilly and Company

Outcome results