Could a pill replace blood transfusions for kids with rare anemia?

NCT ID NCT05144256

Ongoing Disease control Sponsor: Agios Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

Disclaimer Read more

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.

anemia hematologic disorder hemolytic anemia metabolic disease pyruvate kinase deficiency of red cells

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PEDIATRIC HEMOLYTIC ANEMIA PEDIATRIC PYRUVATE KINASE DEFICIENCY

Contacts and locations

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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)