Could a daily pill help kids with rare blood disorder?
NCT ID NCT05175105
First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 2 times
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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
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