3 results
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
To demonstrate non-inferiority of a T2T strategy in which conventional synthetic disease modifying drugs (csDMARDs) refractory RApatients are initially treated with tsDMARD baricitinib versus the comparable T2T strategy in which patients are…
Approved WMOCompleted
To investigate the pharmacokinetics of hospital pharmacy compounded CDCA capsules, compared to the commercial alternative.