3 results
Approved WMOPending
To assess efficacy and safety of temoporfin (Foscan*) photodynamic therapy in the treatment of locally advanced perihilar bile duct carcinoma without distant metastases.
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
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.