The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.
Written informed consent/assent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained before any study-related procedures are conducted and participants must be willing to comply with all study procedures for the duration of the study.
Aged 1 to <18 years and weighing at least 7 kilograms (kg) at the time of providing informed consent/assent.
Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)/β-thalassemia, or α-thalassemia/hemoglobin H (HbH) disease) based on Hemoglobin (Hb) electrophoresis, Hb high-performance liquid chromatography, and/or DNA analysis from the participant's medical record. If this information is not available from the participant's medical record, the test(s) can be performed by a local laboratory during the Screening Period. If a local laboratory is unable to perform the test(s), results from the comprehensive α- and β-globin genotyping performed by the study central laboratory can be used.
Hb concentration ≤10.0 grams per deciliter (g/dL) [100.0 grams per liter (g/L)], based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period.
Pregnant or breastfeeding.
Documented history of homozygous or heterozygous hemoglobin S (HbS) or hemoglobin C (HbC).
Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any prior exposure to myeloablative chemotherapy.
Any conditions other than thalassemia expected to affect sexual maturation.
Parallel
Mitapivat
DrugTablets or Granules
Placebo Matching Mitapivat
DrugTablets or Granules
Percentage of Participants Who Achieved a Hemoglobin (Hb) Response
Hb response is defined as a ≥1.0 grams per deciliter (g/dL) increase in average Hb concentration from Week 12 through Week 24 compared with baseline.
Change From Baseline in Average Hb Concentration From Week 12 Through Week 24
Percentage of Participants Who Achieved a Hb 1.5+ Response
Hb 1.5+ response is defined as a ≥1.5 g/dL increase in average Hb concentration from Week 12 through Week 24 compared with Baseline.
Change From Baseline in Indirect Bilirubin at Week 24
Change From Baseline in Lactate Dehydrogenase (LDH) at Week 24
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