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)

Fecha de inicio: 28 de julio de 2016

Inclusion Criteria: 1. Participant's parent or legal guardian has provided written informed consent/HIPAA authorization prior to any extension study-specific procedures; 2. Participant has previously completed study VBP15-002 up to and including the Week 4 Follow-up assessments within 8 weeks prior to enrollment; and 3. Participant and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures. Exclusion Criteria: 1. Participant had a serious or severe adverse event in study VBP15-002 that, in the opinion of the Investigator, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this study; 2. Participant has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression; 3. Participant has current or history of chronic systemic fungal or viral infections; 4. Participant has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication; 5. Participant has evidence of symptomatic cardiomyopathy. \[Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary\]; 6. Participant is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents. \[Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical corticosteroids prescribed for an indication other than DMD are permitted but must be administered at stable dose for at least 3 months prior to study drug administration\]; 7. Subject has used idebenone within 4 weeks prior to the first dose of study medication; 8. Participant has an allergy or hypersensitivity to the study medication or to any of its constituents; 9. Participant has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator; 10. Participant has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator; or 11. Participant is currently taking any investigational drug, or has taken any investigational drug other than vamorolone within 3 months prior to the start of study treatment. Note: Participants may be re-evaluated if ineligible due to a transient condition which would prevent the subject from participating