2 results
Approved WMOWill not start
Part 1 - To determine the efficacy of oral BCX9930 monotherapy administered for 24 weeks, compared to continued complement component 5 (C5) inhibitor therapy, in subjects with paroxysmal nocturnal hemoglobinuria (PNH) with an inadequate response to…
Approved WMOCompleted
The primary objective of the study is to determine the proportion of patients with elevated TGs, without Familial ChylomicronemiaSyndrome (FCS) due to loss of function (LoF) mutations in lipoprotein lipase (LPL), and a history of…