drugset / Trial / NCT06270316
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
Non-randomizedSequentialOpen-labelTreatment
Summary
The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.
Timeline
- Start
- 2024-06-05
- Primary completion
- 2027-12-01
- Completion
- 2031-04-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AMT-191 | Gene therapy (AAV / viral vector) | — | Intravenous |