2 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
Primary Objective - To determine if afamelanotide can reduce the severity of PLE related pruritis.Secondary Objectives- To determine if afamelanotide can reduce the frequency of PLE episodes;- To determine if afamelanotide can reduce the duration of…