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

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

Explore the condition pages connected to this study.