2 results
Approved WMOCompleted
During the course of the study 2 randomisation questions will be adressed. Both questions concern patients in the high risk group.Randomisation question 1: Will the addition of Doxorubicin to the first 4 standard IVA chemotherapy courses lead to a…
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…