Could a pill replace blood transfusions for kids with rare anemia?
NCT ID NCT05144256
First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 2 times
Summary
This phase 3 trial tests the drug mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic disorder that causes red blood cells to break down too quickly. These children need regular blood transfusions. The study compares mitapivat to a placebo to see if the drug can safely reduce how many transfusions they need. About 49 children will take part, and those who finish the main study can continue on mitapivat in a longer follow-up.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Mitapivat (a drug taken as tablets or granules)
- What this could lead to
- If it works, mitapivat could reduce or eliminate the need for regular blood transfusions in children with pyruvate kinase deficiency, improving their quality of life.
- What could go wrong
- This is a relatively small trial (49 children) and results may not apply to all patients. The drug may cause side effects or fail to show a meaningful reduction in transfusions.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PEDIATRIC HEMOLYTIC ANEMIA are added.
By submitting, you agree to our Terms of use
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
-
Aarhus University Hospital
Aarhus, Central Jutland, 8200, Denmark
-
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
-
CHUV University Hospital of Lausanne
Lausanne, Canton of Bern, Switzerland
-
Charite - UB - CVK - Medizinische Klinik
Berlin, 13353, Germany
-
Children's Healthcare of Atlanta - Emory
Atlanta, Georgia, 30322, United States
-
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
Comprehensive Hemophilia Care Clinic at CHEO (Children's Hospital Eastern Ontario)
Ottawa, Ontario, K1H 5B2, Canada
-
Cure 4 the Kids Foundation, A Division of Roseman University of Health Sciences
Las Vegas, Nevada, 89135, United States
-
Ege University Faculty of Medicine
Izmir, Adana, Turkey (Türkiye)
-
Fakultní Nemocnice Olomouc
Olomouc, 779 00, Czechia
-
Hacettepe University
Ankara, Turkey (Türkiye)
-
Hospital Clinico Universitario Virgen de la Arrixaca
Madrid, 30120, Spain
-
Hospital Universitario Vall d'Hebron
Barcelona, 08035, Spain
-
King's College Hospital NHS
London, SE5 9RS, United Kingdom
-
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
-
Rigshospitalet
Copenhagen, 2100, Denmark
-
Stanford Medicine
Palo Alto, California, 94304, United States
-
Texas Children's Hospital
Houston, Texas, 77030, United States
-
UT Southwestern Medical Center
Dallas, Texas, 75390, United States
-
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
-
Weill Cornell Medical College
New York, New York, 10065, United States
-
İstanbul Üniversitesi Tıp Fakültesi [Istanbul University Faculty of Medicine] Çocuk Sağlığı Enstitüsü [Institute of Child Health]
Istanbul, 34093, Turkey (Türkiye)
More trials for these conditions
Other studies related to the condition(s) this trial covers.