The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.
Age ≥12 years.
Documented diagnosis of SCD (hemoglobin SS (HbSS), combined heterozygosity for hemoglobins S and C (HbSC), sickle cell hemoglobin (HbS)/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants).
No more than 10 SCPCs in the 12 months before providing informed assent/consent.
At least 1 transfusion of packed RBCs in the 12 months before informed assent/consent.
Pregnant, breastfeeding, or parturient.
Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion); episodic transfusion in response to worsened anemia or vaso-occlusive crisis (VOC) is permitted. Additionally, a subject who requires episodic transfusion(s) may not have received a transfusion(s) within 60 days before providing informed assent/consent or during the Screening Period.
Hospitalized for an SCPC and/or other vaso-occlusive event within 14 days prior to providing informed assent/consent or during the Screening Period. A hospitalization is defined as an in-patient admission to a hospital that may or may not be preceded by an emergency room or outpatient clinic visit. A visit to an emergency room that does not result in an in-patient admission does not meet the definition of hospitalization.
Currently receiving treatment with a disease-modifying therapy for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of hydroxyurea. The last dose of voxelotor, crizanlizumab, and L-glutamine must have been administered at least 90 days before randomization.
Parallel
Mitapivat Matched Placebo
DrugTablets
Mitapivat
DrugTablets
Percentage of Subjects who are Transfusion Free From Week 4 Through Week 52
Number of Red Blood Cells (RBC) Units Transfused From Week 4 Through Week 52
Percentage of Subjects 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 24 through Week 52 compared with baseline.
Average Change From Baseline in Hb Concentration From Week 24 Through Week 52
Average Change From Baseline in Indirect Bilirubin From Week 24 Through Week 52
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