Could a daily pill help kids with rare blood disorder?
NCT ID NCT05175105
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The trial compares mitapivat to a placebo to see if it can raise hemoglobin levels without the need for regular blood transfusions. About 30 children will take part, and those who finish the initial 20-week study can continue on mitapivat for up to 5 more years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
mitapivat
What this could lead to
If it works, this could provide a daily pill to control anemia in children with pyruvate kinase deficiency, reducing symptoms and improving quality of life.
What could go wrong
This is a small, early-phase trial with only 30 children. The drug may not improve hemoglobin levels significantly, and long-term safety is still being studied.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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