3 results
Primary objective:The primary objective of this study is to assess the time to painful VOC resolution, measured from the first dose of sevuparine given to achievement of crises resolution, as compared to placebo. Secondary objectives: The secondary…
Part 1:• To evaluate the effect of IV sevuparin on inflammatory responses following an intradermal (ID) LPS challenge.Part 2:• To evaluate the effect of IV sevuparin on safety and tolerability and inflammatory responses following an intravenous (IV…
The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease) with an experimental oral study drug named eliglustat in pediatric patients from 2 to 18 years with Gaucher disease.