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 WMOCompleted
The primary objective of the study is to estimate the clinical benefit of cemiplimab + ISA101b after progression on first line chemotherapy, as assessed by objective response rate (ORR).The secondary objectives of the study are:• To characterize the…
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