New hope for fabry patients with kidney failure: drug safety study underway
NCT ID NCT04020055
First seen Oct 31, 2025 · Last updated May 13, 2026 · Updated 23 times
Summary
This study looks at the safety and how the body processes the drug migalastat in people with Fabry disease who also have severe kidney disease or are on dialysis. About 14 adults with a specific genetic change that makes them eligible for migalastat will take part. The goal is to ensure the drug is safe and to understand its levels in the blood for these patients.
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
Explore the condition pages connected to this study.