New pill offers hope for kids with rare, debilitating blood disease
NCT ID NCT05175105
Summary
This study is testing whether an oral drug called mitapivat is safe and effective for children with pyruvate kinase deficiency (PKD), a rare genetic disorder that causes chronic anemia. About 30 children, aged 1 to 18, who do not regularly need blood transfusions will take either the drug or a placebo for 20 weeks, followed by an option to take the drug for up to 5 more years. The main goal is to see if the drug can safely and significantly increase their red blood cell count (hemoglobin).
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Contacts and locations
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Locations
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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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Centre hospitalier Universitaire de Sainte-Justine
Montreal, Quebec, QC H3T 1C5, Canada
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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 Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Michigan
Detroit, Michigan, 48304, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Hospital Universitario Infantil Nino Jesus
Madrid, 28009, Spain
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Hospital Universitario Vall d'Hebron
Barcelona, 08035, Spain
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Hôpital Pellegrin
Bordeaux, Aquitaine, 33000, France
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St Jude's Children's Research Hospital
Memphis, Tennessee, 38105, United States
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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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UChicago Medicine
Chicago, Illinois, 60637, 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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Universitatsklinikum Wurzburg
Würzburg, Bavaria, 97080, Germany
Conditions
Explore the condition pages connected to this study.