New hope for fabry patients with failing kidneys: drug safety study underway
NCT ID NCT04020055
Summary
This study aims to check the safety and measure how the drug migalastat behaves in the body of people with Fabry disease who also have severe kidney damage or need dialysis. It will enroll about 14 adults who have a specific genetic type of Fabry disease that responds to this drug. The main goal is to understand how the drug is processed and removed by the body in patients whose kidneys don't work well.
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Contacts and locations
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Locations
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Centro Hospitalar e Universitário de Coimbra (CHUC)
Coimbra, 3041-801, Portugal
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Emory University
Atlanta, Georgia, 30322, United States
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Hospital General Universitario Gregorio Marañon
Madrid, 28007, Spain
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Hospital General Universitario de Elda
Elda, 03600, Spain
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Hospital Universitari(o) de Bellvitge (HUB) Feixa Llarga
Barcelona, 08907, Spain
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Lysosomal and Rare Disorders Research and Treatment Center, Inc
Fairfax, Virginia, 22030, United States
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Osaka University Hospital
Suita, Osaka, 565-0871, Japan
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Royal Melbourne Hospital
Parkville, Victoria, 3050, Australia
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Royal Perth Hospital
Perth, Washington, 6000, Australia
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Salford Royal Hospital
Salford, England, M6 8HD, United Kingdom
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Shizuoka General Hospital
Shizuoka, Shizuoka, 420-8527, Japan
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The Cleveland Clinic
Cleveland, Ohio, 44195, United States
Conditions
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