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New hope for fabry patients with kidney failure?

NCT ID NCT04020055

First seen Oct 31, 2025 · Last updated Jun 18, 2026 · Updated 26 times

Summary

This study looks at how safe the drug migalastat is and how it works in the body for people with Fabry disease who also have severe kidney issues or are on dialysis. It involves 14 adults with a specific genetic type of Fabry disease. The goal is to understand how the drug behaves in these patients to help guide future treatment.

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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.

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Contacts and locations

Locations

  • Centro Hospitalar e Universitário de Coimbra (CHUC)

    Coimbra, 3041-801, Portugal

  • Emory University

    Atlanta, Georgia, 30322, United States

  • Hospital General Universitario Gregorio Marañon

    Madrid, 28007, Spain

  • Hospital General Universitario de Elda

    Elda, 03600, Spain

  • Hospital Universitari(o) de Bellvitge (HUB) Feixa Llarga

    Barcelona, 08907, Spain

  • Lysosomal and Rare Disorders Research and Treatment Center, Inc

    Fairfax, Virginia, 22030, United States

  • Osaka University Hospital

    Suita, Osaka, 565-0871, Japan

  • Royal Melbourne Hospital

    Parkville, Victoria, 3050, Australia

  • Royal Perth Hospital

    Perth, Washington, 6000, Australia

  • Salford Royal Hospital

    Salford, England, M6 8HD, United Kingdom

  • Shizuoka General Hospital

    Shizuoka, Shizuoka, 420-8527, Japan

  • The Cleveland Clinic

    Cleveland, Ohio, 44195, United States

Conditions

The condition(s) this trial relates to.

chronic kidney disease Fabry disease kidney disorder kidney failure

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.