Ignite Creation Date:
2025-12-24 @ 11:30 PM
Ignite Modification Date:
2026-01-01 @ 5:20 PM
Study NCT ID:
NCT06696456
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-05-31
First Post:
2024-11-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
Sponsor:
Akouos, Inc.
Organization:
Study Overview
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
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2025-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: