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-Non–Transfusion-Dependent Thalassemia (ENERGIZEKids)

Non-Transfusion-Dependent Alpha- or Beta-Thalassemia

Hb response, defined as a ≥1.0 g/dL increase in average Hb concentration from Week 12 through Week 24 compared with baseline

1.Change from baseline in average Hb concentration from Week 12 through Week 24, 2. Hb 1.5+ response, defined as a ≥1.5 g/dL increase in average Hb concentration from Week 12 through Week 24 compared with baseline, 3. Change from baseline in indirect bilirubin, lactate dehydrogenase, and haptoglobin at Week 24, 4. Change from baseline in reticulocytes and erythropoietin at Week 24, 5. Changes from baseline in sex hormones, sexual maturity rating with Tanner stage, and ovarian cysts (female subjects only) through Week 24 for patients in Cohort 1, 6. Changes over time in height-for-age z-score, weight-for-age z-score, and body mass index-for-age z-score, 7. Changes from baseline in bone mineral density through Week 24, 8. Type, severity, and relationship of AEs and SAEs, 9. Change from baseline through Week 24 on patient reported fatigue and other symptoms of thalassemia to assess the impact of these symptoms on HRQOL including physical and school function as measured by the PedsQL Multidimensional Fatigue Scale and PedsQL 4.0 Generic Core Scales, 10. 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 24, 11. Plasma concentrations and pharmacokinetic parameters of mitapivat at Week 4.

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