drugset / Trial / NCT05821959
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Non-randomizedSequentialOpen-labelTreatment
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, together with the Precision Delivery Mechanism, to safely achieve the intended product performance.
Timeline
- Start
- 2023-09-15
- Primary completion
- 2028-10
- Completion
- 2028-10
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAVAnc80-hOTOF | Gene therapy (AAV / viral vector) | 4.1e+11 vg | Other |
| Subject | AAVAnc80-hOTOF | Gene therapy (AAV / viral vector) | 8.1e+11 vg | Other |