Mitapivat for Children With Non-Transfusion-Dependent Thalassemia
Treatments studied
Part of Blood & lymphatic clinical trials.
This study tests a medicine called mitapivat in children ages 1–17 with thalassemia who don't need frequent blood transfusions. The medicine may help their bodies make more healthy red blood cells and reduce anemia symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child is between 1 and 18 years old and weighs at least 7 kilograms (about 15 pounds)
- Confirmed diagnosis of thalassemia (alpha or beta type) from blood tests or genetic testing
- Low hemoglobin level (≤10 g/dL) measured at least twice, 7 days apart
- Received 5 or fewer blood transfusions in the past 6 months, and none in the past 8 weeks or during screening
- No history of gene therapy, bone marrow/stem cell transplant, or certain blood-boosting medications in past 18 weeks
- No serious heart, lung, liver, or kidney disease; no active infections or hepatitis
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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