Non-Transfusion-dependent Alpha-Thalassemia Clinical Trial
Official title:
A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)
The primary purpose of this study is to compare the effect of mitapivat versus placebo on anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).
The mitapivat group will include approximately 114 participants. The placebo group will include approximately 57 participants. ;