Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Otoferlin Gene-mediated Hearing Loss. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Spain, Taiwan
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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.
Primary outcome measures
- 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.]
Secondary outcome measures (1)
- Auditory Brainstem Response (ABR) Threshold [Time frame: Through study completion, approximately four years.]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
United States · 3 centers
- University of Iowa — Iowa City
- Boston Children's Hospital — Boston
- Children's Hospital of Philadelphia — Philadelphia
Spain · 1 center
- Hospital Sant Joan de Déu — Esplugues de Llobregat
Taiwan · 1 center
- National Taiwan University Hospital — Taipei
Identifiers
NCT: NCT06696456 · AK-OTOF-LTFU