A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Status:
Completed
Trial end date:
2021-08-19
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
ReveraGen BioPharma, Inc.
Collaborators:
Cooperative International Neuromuscular Research Group European Union Newcastle University University of Pittsburgh