3 results
Approved WMORecruiting
Primary objective- Survival without BPD at 36 weeks gestational age (GA)Secondary objectives- Neurodevelopment at a corrected age of 18-22 months- Adverse treatment effects- Mortality at 36 weeks gestational age- BPD incidence at 36 weeks…
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