Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
Recruiting now · Phase 1/Phase 2
Conditions studied: Fabry Disease
In brief
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.
Key facts
- Study ID
- NCT06270316
- Run by
- UniQure Biopharma B.V.
- People needed
- 12
- Starts
- 2024-06-05
- Expected to finish
- 2031-04-30
- Last updated by the study team
- 2025-10-23
Who can join
Age: 18 and older, up to 50. Sex: male. Healthy volunteers: not accepted.
Where it is running
- The Kirklin Clinic Of university of Alabama Birmingham Hospital — Birmingham, Alabama, United States (enrolling)
- Emory University School of Medicine — Atlanta, Georgia, United States (enrolling)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
- MHealth Fairview University of Minnesota Medical Center East Bank — Minneapolis, Minnesota, United States (enrolling)
- NYC Health + Hospitals/Metropolitan — New York, New York, United States (enrolling)
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)
- University of Utah, Clinical and Translational Sciences Institute — Salt Lake City, Utah, United States (enrolling)
- Lysosomal & Rare Disorders Research and Treatment Center, Inc — Fairfax, Virginia, United States (enrolling)
Full record on ClinicalTrials.gov
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