Clinical Trial

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Recruiting Phase 1/2
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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.
Protocol Amendment History 9 amendments
This ClinicalTrials.gov record has been amended 9 times since 2024-02-20; most recent amendment 2025-10-21.
Status change: Not Yet Recruiting → Recruiting 2024-05-16
Trial Details
NCT Number NCT06270316
Lead Sponsor UniQure Biopharma B.V.
Conditions Fabry Disease
Enrollment 12 participants
Start Date 2024-06-05
Primary Completion 2027-12-01 (estimated)
Study Completion 2031-04-30 (estimated)
Updated on ClinicalTrials.gov 2025-10-23