A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β-Transfusion-Dependent Thalassemia (ENERGIZEKids-T)

Transfusion-Dependent Alpha- or Beta-Thalassemia

Transfusion reduction response (TRR), defined as a ≥50% reduction in transfused red blood cell (RBC) volume (normalized by weight) in any consecutive 12- week period through Week 48, compared with historical RBC transfusion volume (normalized by weight) and standardized to 12 weeks

1. TRR2, defined as ≥50% reduction in transfused RBC volume (normalized by weight) from Week 13 to Week 24 compared with historical RBC transfusion volume (normalized by weight) and standardized to 12 weeks, 2. TRR3, defined as ≥50% reduction in transfused RBC volume (normalized by weight) from Week 25 to Week 36 compared with historical RBC transfusion volume (normalized by weight) and standardized to 12 weeks, 3. TRR4, defined as ≥50% reduction in transfused RBC volume (normalized by weight) from Week 37 to Week 48 compared with historical RBC transfusion volume (normalized by weight) and standardized to 12 weeks, 4. TRR5, defined as ≥50% reduction in transfused RBC volume (normalized by weight) from Week 25 to Week 48 compared with historical RBC transfusion volume (normalized by weight) and standardized to 24 weeks, 5. Percent change in transfused RBC volume (normalized by weight) from Week 13 through Week 48 compared with historical transfusion volume (normalized by weight) and standardized to 36 weeks, 6. Transfusion independence, defined as transfusion-free for greater than or equal to 8 consecutive weeks through Week 48, 7. Change in the number of transfusion episodes (ie, transfusion events) from Week 13 through Week 48 compared with historical number of transfusion episodes standardized to 36 weeks, 8. Change from baseline in markers of iron metabolism (serum iron, total iron-binding capacity, transferrin, transferrin saturation) and indicators of iron overload (serum ferritin) at Week 48, 9. Change from baseline in sex hormones, sexual maturity rating with Tanner stage, and ovarian cysts (female subjects only) through Week 48 for patients in Cohort 1, 10. Changes over time in height-for-age z-score, weight-for-age z-score, and body mass index–for-age z-score, 11. Change from baseline in bone mineral density through Week 48, 12. Type, severity, and relationship of adverse events and serious adverse events, 13. Plasma concentrations and pharmacokinetic parameters of mitapivat at Week 4

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