A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)
Study ID: STU 052016-072
This Phase iia extension study is an open-label, multicenter study to evaluate the long-term safety, tolerability, clinical efficacy, and PD of vamorolone at dose levels up to 8.0 mg/kg administered daily by liquid oral suspension over a Treatment Period of 24 weeks to boys ages 4-7 years with DMD. Subjects who have completed the Phase iia VBP15-002 Study Week 4 Follow-up assessments within 8 weeks prior to enrollment are eligible. The study is comprised of a Pretreatment Baseline Period of up to 24 hours, which begins with the signing of the extension study-specific informed consent form (iCF), a 24-week Treatment Period, and a 2- to 5-week Dose-tapering Period for subjects who elect to transition off vamorolone treatment at the end of the study. Subjects are enrolled at the time of iCF signing. Parents or guardians of eligible subjects may provide written informed consent for VBP15-003 extension study participation, and subjects may undergo Baseline assessments, at the final VBP15-002 Week 4 Visit, following completion of all VBP15-002 Week 4 assessments.
1. Subject[Single Quote]s parent or legal guardian has provided written informed consent/HIPAA authorization prior to any extension study-specific procedures 2. Subject has previously completed study VBP15-002 up to and including the Week 4 Follow-up assessments within 8 weeks prior to enrollment 3. Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.