3 results
Approved WMORecruiting
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the reduction in transfusion burden in pediatric subjects with pyruvate kinase deficiency (PK deficiency) who are regularly transfused.
Approved WMORecruiting
The primary objective to demonstrate that in CNOpatients with pain 3-monthly treatment with pamidronate will result in significant decrease in maximum pain score (as measured with BPI) as compared to placebo.Secondary objectives:•…
Approved WMORecruiting
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in pediatric subjects with PK deficiency who are not regularly transfused