3 results
Approved WMOWill not start
Evaluate whether FVIII/VWF concentrates successfully induce immune tolerance in patients who have already experienced and failed immune tolerance induction with VWF-free FVIII concentrates.
A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase deficiency
The primary objective of this study is to evaluate the safety of sebelipase alfa in a more broad population of subjects with LAL Deficiency than have been previously studied.The secondary objectives of this study are (1) to evaluate effects of…
Approved WMOCompleted
The primary objective of this study is to evaluate the safety and tolerability using bleeding as the primary endpoint. Secondarily, this study will serve as a proof-of concept by evaluating the effect that DS-1040b administration has on total…