3 results
Approved WMORecruiting
the primary objective of our study is to define a set of patient characteristics that can predict the effectiveness of an MRA in treating symptomatic OSA. The aim of our study is to develop a prediction model to calculate the success rate in an…
Approved WMORecruiting
The primary objective of this study is to evaluate the efficacy of olipudase alfa in adult patients with acid sphingomyelinase deficiency.
Approved WMORecruiting
Primary:• To evaluate the safety of ORGN001 (formerly ALXN1101) over the first 6 months of treatmentSecondary:• To characterize the pharmacokinetics (PK) of increasing doses of ORGN001 (formerly ALXN1101) • To evaluate the effect of ORGN001 (…