NCT06696456

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
• Phase: —
• Study type: Observational
• Enrollment: 30 (estimated)
• Sponsor: Akouos, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Otoferlin Gene-mediated Hearing Loss

Timeline

• Start date: 2024-11-11
• Primary completion: 2033-04-01
• Completion: 2033-04-01
• First posted: 2024-11-20
• Last updated: 2026-04-06

Locations

5 sites across 3 countries: United States, Spain, Taiwan