2 results
Approved WMOCompleted
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…
Approved WMORecruiting
Primary:• To evaluate the safety of ORGN001 (formerly ALXN1101) over the first 6 months of treatmentSecondary:• To characterize the pharmacokinetics (PK) of increasing doses of ORGN001 (formerly ALXN1101) • To evaluate the effect of ORGN001 (…