3 results
Approved WMOWill not start
The primary objective of this study is to determine if ambrisentan is effective in delaying disease progression and death in subjects with IPF.Secondary objectives include evaluation of the safety and effect of ambrisentan on development of…
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