2 results
Approved WMOCompleted
Main objectivePart A: To evaluate the safety and efficacy of treatment with VX-371, administered with and without 4.2% hypertonic saline (HS) in subjects with primary ciliary dyskinesia (PCD) who are *12 years of age.Part B: To evaluate the safety…
Approved WMOCompleted
Primary objective: Estimate the MTD and/or RDE of LDK378 as a single agent when administered orally to pediatric patients with ALK-activated tumors in the fasted and in fed stateSecondary objectives: Objective 1: Characterize the safety and…