Ignite Creation Date:
2025-12-24 @ 2:43 PM
Ignite Modification Date:
2025-12-26 @ 12:29 AM
Study NCT ID:
NCT05821959
Status:
RECRUITING
Last Update Posted:
2025-12-18
First Post:
2023-02-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Sponsor:
Akouos, Inc.
Organization:
Study Overview
Official Title:
A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
Status:
RECRUITING
Status Verified Date:
2025-12
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:
Part A of this trial will evaluate the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos Delivery Device to safely achieve the intended product performance.
Detailed Description:
The following two dose levels will be tested:
* Up to 4.1E11 total vg/cochlea
* Up to 8.1E11 total vg/cochlea
* Up to 4.1E11 total vg/cochlea
* Up to 8.1E11 total vg/cochlea
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
True
Is an Unapproved Device?:
True
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: