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A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

Exploring mitapivat's effects in children with specific thalassemia types.

Recruiting
1-17 years
All
Phase 3

This study looks at whether a drug called mitapivat helps kids with a condition called thalassemia, a blood disorder where the body doesn't make enough healthy red blood cells. "Non-transfusion-dependent" means these kids don't need regular blood transfusions. *Mitapivat* is compared to a placebo, which is a fake treatment often used in studies to see if the real drug works better. Participants must be between 1 and 18 years old and weigh at least 7 kilograms. They should not have received more than 5 blood transfusions in the past 24 weeks or any transfusions in the last 8 weeks. Also, some medications and conditions like certain liver, kidney, or heart diseases can disqualify participation.

  • Study Duration: Participants will be involved until the study is completed.
  • Eligibility: Kids aged 1 to 18 with thalassemia not needing regular transfusions.
  • Risks and Benefits: Possible side effects from the drug; potential improvement in anemia.
Study details
    Non-Transfusion-dependent Alpha-Thalassemia
    Non-Transfusion-dependent Beta-Thalassemia

NCT07517133

Agios Pharmaceuticals, Inc.

17 October 2026

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