At a glance
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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)
In Brief
A Phase 2 clinical trial evaluating Vamorolone, Prednisone, and 1 other intervention for Duchenne Muscular Dystrophy. Completed, enrolled 121 participants across 33 sites in 11 countries.
Detailed Summary
Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to \<7 years with DMD.
Study Details
Timeline
Interventions
Oral administration of 2.0 mg/kg/day for the duration of the study.
Oral administration of 0.75 mg/kg/day for 24 weeks.
Oral administration of placebo daily for 24 weeks.
Oral administration of 6.0 mg/kg/day for the duration of the study.
Oral administration of 0.75 mg/kg/day for 24 weeks.
Oral administration of placebo daily for 24 weeks.
Oral administration of 2.0 mg/kg/day for 20 weeks.
Oral administration of 6.0 mg/kg/day for 20 weeks.