3 results
Approved WMORecruiting
Primary objectives:Phase 1: To determine the MTD and/or RP2D of oral ponatinib administered QD in pediatric participants with selected advanced hematologic malignancies or solid tumors.Phase 2- Group A (CP-CML): To determine the efficacy of oral…
Approved WMORecruiting
The aim of this study is to investigate the efficacy and tolerability of pyridostigmine monotherapy and amifampridine as add-on compared to placebo in patients with MG.
Approved WMORecruiting
Primary: To assess the proportion of patients with intervention failure at 12 months after dose reduction, defined as patients who have restarted their initial dose due to (expected) loss of major molecular response.