New pill aims to free kids from frequent blood transfusions
NCT ID NCT05144256
First seen Nov 01, 2025 · Last updated Apr 11, 2026 · Updated 23 times
Summary
This study is testing an oral drug called mitapivat in children with pyruvate kinase deficiency, a rare genetic condition that destroys red blood cells and forces them to need regular blood transfusions. The main goal is to see if taking mitapivat daily can significantly reduce or even eliminate the need for these transfusions over 32 weeks. Children who complete the initial study can continue taking the drug in a 5-year follow-up period to monitor long-term safety and effects.
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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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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
Explore the condition pages connected to this study.