3 results
Approved WMOPending
Primary:Is the degree of pupildilation, achieved after administrating a 4.5 microliter microdrop tropicamide 0.5% equivalent to the pupildilation after a regular 30 microliter eydrop tropicamide 0.5% in healthy volunteers?Secondary: Is there a…
Approved WMOCompleted
Primary objectiveTo evaluate the long-term safety of 10-, 10-, 20-mg/kg ataluren in patients with nonsense mutation cystic fibrosis (nmCF), who previously participated in pivotal study PTC124-GD-021-CF, as determined by adverse events and laboratory…
Approved WMOPending
Primary:To assess the safety and tolerability of DS-1211b compared with placebo in subjects with PXE.To assess the dose response by assessing the treatment changes in PD endpoints.