A Phase 1/2 Trial of AAVAnc80-hOTOF Gene Therapy in Individuals with Sensorineural Hearing Loss due to Biallelic Otoferlin Gene (OTOF) Mutations

Status

Not yet recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-505128-76-00

Acronym

AK-OTOF-101

Enrollment

Registered

2023-10-05

Start date

Unknown

Completion date

Unknown

Last updated

2023-10-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Otoferlin gene-mediated hearing loss

Brief summary

Frequency of AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device, used as part of a delivery system), if able to be determined.

Detailed description

Change from baseline in diagnostic auditory brainstem response (ABR) testing threshold.

Interventions

DRUGAAVAnc80-hOTOF

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Frequency of AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device, used as part of a delivery system), if able to be determined.	—

Secondary

Measure	Time frame
Change from baseline in diagnostic auditory brainstem response (ABR) testing threshold.	—

Countries

Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026