3 results
Approved WMOCompleted
The study aims to limit the use of anthracyclines and to reduce the dose of ATRA. Another aim is to stratify treatment by risk group: standard risk - WBC <10 x 109/l : high risk - WBC >=10 x 109/l. Furthermore this study aims to…
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
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