2 results
Approved WMOCompleted
1: Compare the differences in the PRAGMA-CF score: the volume proportion of the lung with structural airways disease (%Dis), measured from chest CT images at 48 weeks between treatment arms 2: Compare the differences in PRAGMA-CF subscores: the…
Approved WMOCompleted
Primary:To assess the safety and tolerability of Firdapse after single and multiple doses Secondary:Part 1 only: To determine the dose-related PK profile of single, escalating doses of Firdapse;Part 2 only: To assess the steady-state plasma PK and…