New hope for fabry patients with kidney failure: drug dosing study underway
NCT ID NCT04020055
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests the safety and how the body processes migalastat in 14 adults with Fabry disease who have severe kidney impairment or are on dialysis. Participants take migalastat capsules, and researchers measure drug levels in blood and dialysate. The goal is to ensure proper dosing for this vulnerable group.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
migalastat (a drug that helps break down fatty substances in cells)
What this could lead to
If successful, this could confirm that migalastat is safe and properly dosed for Fabry patients with severe kidney problems, expanding treatment options for this group.
What could go wrong
This is a small, early-stage study (14 people) focused on safety and drug levels, not on whether the drug improves symptoms. It may not lead to a new treatment if safety issues arise.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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