New pill aims to free kids from frequent blood transfusions
NCT ID NCT05144256
Summary
This study is testing whether an oral medication called mitapivat can help children with pyruvate kinase deficiency (PK deficiency), a rare genetic disorder that destroys red blood cells. Children in the trial, who currently need regular blood transfusions to survive, will take either the drug or a placebo pill for about 8 months to see if the drug safely reduces their need for transfusions. Those who complete this part can continue taking the drug in a 5-year follow-up study.
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Contacts and locations
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Locations
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Aarhus University Hospital
Aarhus, Central Jutland, 8200, Denmark
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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CHUV University Hospital of Lausanne
Lausanne, Canton of Bern, Switzerland
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Charite - UB - CVK - Medizinische Klinik
Berlin, 13353, Germany
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Children's Healthcare of Atlanta - Emory
Atlanta, Georgia, 30322, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Comprehensive Hemophilia Care Clinic at CHEO (Children's Hospital Eastern Ontario)
Ottawa, Ontario, K1H 5B2, Canada
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Cure 4 the Kids Foundation, A Division of Roseman University of Health Sciences
Las Vegas, Nevada, 89135, United States
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Ege University Faculty of Medicine
Izmir, Adana, Turkey (Türkiye)
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Fakultní Nemocnice Olomouc
Olomouc, 779 00, Czechia
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Hacettepe University
Ankara, Turkey (Türkiye)
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Hospital Clinico Universitario Virgen de la Arrixaca
Madrid, 30120, Spain
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Hospital Universitario Vall d'Hebron
Barcelona, 08035, Spain
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King's College Hospital NHS
London, SE5 9RS, United Kingdom
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Rigshospitalet
Copenhagen, 2100, Denmark
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Stanford Medicine
Palo Alto, California, 94304, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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UT Southwestern Medical Center
Dallas, Texas, 75390, United States
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
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Weill Cornell Medical College
New York, New York, 10065, United States
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İstanbul Üniversitesi Tıp Fakültesi [Istanbul University Faculty of Medicine] Çocuk Sağlığı Enstitüsü [Institute of Child Health]
Istanbul, 34093, Turkey (Türkiye)
Conditions
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