5 results
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
The primary objective of the study is:* To demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab 15 mg/kg intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial…
The primary objective of this clinical trial is to obtain pilot safety and efficacy data on treatment of PAH patients by 6-MP. The secondary objective of this clfnical trial is to determine whether LEPs transcriptome analysis will identify a subset…
PrimaryThe primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).The primary objective for Part B of the study is to demonstrate a…
The aim of this study is to investigate the efficacy of subcutaneous IFX in the treatment of moderate to severe Crohn*s disease with and without concomitant immunosuppression, as measured by the proportion of patients in corticosteroid-free clinical…