2 results
Approved WMOCompleted
Primary Objective: Studying the phenomenon of tolerance by evaluating pharmacokinetic- and dynamic parameters, in patients receiving long-term ITB therapy.Secondary Objective(s): 1.To specify the intrathecal pharmacokinetics and -dynamics of…
Approved WMORecruiting
Phase 2:Primary ObjectivesTo determine the recommended Phase3 dose of mitapivat by evaluating the effect of 2 dose levels of mitapivat versus placebo on:• Anemia in subjects with sickle cell disease (SCD)• SafetySecondary ObjectivesTo evaluate the…