New hope for kids with rare blood disorder: pill may reduce need for transfusions
NCT ID NCT05175105
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 24 times
Summary
This study tests an oral drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic anemia that causes red blood cells to break down too quickly. The goal is to see if the drug can raise hemoglobin levels and reduce the need for blood transfusions. Participants are randomly assigned to receive either mitapivat or a placebo for about 5 months, followed by up to 5 years of open-label treatment where everyone gets the active drug.
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This is a summary of
the original study
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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.
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
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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.