3 results
Primary objective:• Assess the pharmacokinetics (PK) of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary VWDSecundary objectives:• Compare the…
The primary objective of this study is to prospectively evaluate the annualized bleeding rate (ABR) for spontaneous bleeding episodes while on prophylactic treatment with rVWF (vonicog alfa) and to compare it to the subject's historical ABR for…
Primary objectives: 1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and2. To evaluate the safety and…