Extended treatment trial offers hope for managing rare genetic disorder
NCT ID NCT03614234
Summary
This study continues testing an enzyme replacement therapy called pegunigalsidase alfa in adults with Fabry disease who finished a previous trial. Researchers want to understand the long-term safety and effectiveness of receiving this medication through an IV every four weeks. They will monitor kidney and heart function, pain levels, and overall quality of life over an extended period.
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Contacts and locations
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Locations
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Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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Azienda Ospedaliera Universitaria "Federico II"
Naples, Via Pansini, 80131, Italy
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Emory University School of Medicine
Atlanta, Georgia, 30307, United States
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Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
Prague, 128 08, Czechia
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Helse Bergen HF Haukeland Universitetssykehus
Bergen, 5021, Norway
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Infusion Associates
Grand Rapids, Michigan, 49525, United States
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Medical Endocrinology PE 2132, Rigshospitalet
Copenhagen, 2100, Denmark
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O & O Alpan
Fairfax, Virginia, 22030, United States
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Renal Disease Research Institute, LLC
Dallas, Texas, 75235, United States
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The Royal Free Hospital
London, United Kingdom
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UAB Medicine
Birmingham, Alabama, 35233, United States
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UZ Antwerpen
Edegem, 2650, Belgium
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University of Iowa Hospitals and Clinica
Iowa City, Iowa, 52242, United States
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University of Utah Hospitals & Clinics
Salt Lake City, Utah, 84112, United States
Conditions
Explore the condition pages connected to this study.