Clinical trial NCT06696456: Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
Official title: 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
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Otoferlin Gene-mediated Hearing Loss
Keywords: Otoferlin
Deafness, autosomal recessive 9 (DFNB9)
Hearing loss
Hearing loss, bilateral
Hearing loss, sensorineural
Auditory neuropathy (AN)
Lead sponsor: Akouos, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Eli Lilly and Company
First posted: 2024-11-20
Enrollment: 30
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Long term safety of AAVAnc80-hOTOF, including the incidence and frequency of late-occurring adverse events (AEs)
timeFrame: Through study completion, approximately four years.
Secondary outcomes:
measure: Auditory Brainstem Response (ABR) Threshold
timeFrame: Through study completion, approximately four years.
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Esplugues de Llobregat
country: Spain
facility: Hospital Sant Joan de Déu
state: Barcelona
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
Study officials:
affiliation: Akouos, Inc.
name: Kathy Reape, M.D.
role: STUDY_DIRECTOR
References: