3 results
Primary Objective: Measure QoL (VEINES QoL/Sym) change in patients with DVO at one year after PTA and stenting compared to conventional therapy (short class II elastic compressions stockings, exercise, lymph drainage therapy and the use of (pain)…
The objectives of the study are to:* Identify a dose and dosing regimen of KRN23, based on safety and PD effect, in pediatric XLH patients* Establish the safety profile of KRN23 for the treatment of children with XLH including ectopic mineralization…
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…