Mitapivat for children with transfusion-dependent thalassemia
Treatments studied
Part of Blood & lymphatic clinical trials.
This study tests a study pill called mitapivat for children aged 1 to 17 with transfusion-dependent thalassemia. The goal is to see if mitapivat can reduce the need for blood transfusions and improve blood counts.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be between 1 and 17 years old and weigh at least 15 pounds (7 kg).
- Your child must have transfusion-dependent thalassemia and have needed 6 to 20 blood transfusions in the past 24 weeks, with no more than 6 weeks between transfusions.
- If your child takes hydroxyurea, the dose must have been steady for at least 16 weeks.
- Female participants who have started their period must agree to two methods of birth control, including one highly effective method.
- Your child cannot have had a bone marrow transplant, stem cell transplant, or gene therapy in the past.
- Your child must not have certain other medical conditions (like severe liver, kidney, or heart problems) or be on certain medications (like strong CYP3A inhibitors or anabolic steroids).
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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