A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
Study ID: STU 032018-026
Summary
This study is to evaluate the long-term efficacy, safety, tolerability, PD, and population pharmacokinetics of vamorolone (the investigational medicine) compared to prednisone (active control) and placebo over a Treatment Period of 24 weeks in boys ages 4 to less than 7 years with DMD, and determine the persistence of effect over a total Treatment Period of 48 weeks.
Primary objectives: To compare the efficacy and to evaluate the safety and tolerability of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to less than 7 years with DMD. Secondary objectives: To compare the safety and efficacy of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg over a 24-week treatment period vs. daily prednisone 0.75 mg/kg in ambulant boys ages 4 to less than 7 years with DMD; To compare the efficacy of vamorolone administered orally at daily doses of 2.0 mg/kg vs. vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period in ambulant boys ages 4 to less than 7 years with DMD; To compare the safety and efficacy of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg over a 48-week treatment period in ambulant boys ages 4 to less than 7 years with DMD vs. untreated and prednisone treated DMD historical controls; To evaluate the population pharmacokinetics (PK) of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg in ambulant boys ages 4 to less than 7 years with DMD.