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A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Studying Mitapivat's effects in children with transfusion-dependent thalassemia.

Recruiting
1-17 years
All
Phase 3

This study is testing a new drug called mitapivat for kids with a condition called thalassemia. Thalassemia is a blood disorder where the body makes fewer healthy red blood cells, leading to frequent blood transfusions. The study aims to see if mitapivat can help reduce these transfusions. A placebo is a "fake" treatment used to compare results.

Eligibility: Kids aged 1 to 17 years who are dependent on transfusions and weigh at least 7 kilograms can join. They must have a confirmed diagnosis of thalassemia. Kids taking hydroxyurea, a medication to help blood conditions, can participate if their dose hasn't changed for 16 weeks. Girls who have started menstruating need to avoid pregnancy during the study.

  • The study requires you to follow all procedures for the duration of the study.
  • Pregnant or breastfeeding participants cannot join the study.
  • Participants with certain health conditions or past treatments might not be eligible.
Study details
    Transfusion-dependent Alpha-Thalassemia
    Transfusion-dependent Beta-Thalassemia

NCT07506863

Agios Pharmaceuticals, Inc.

17 October 2026

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