An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: Vamorolone 0.25 mg/day/day; Vamorolone 0.75 mg/day/day; Vamorolone 2.0 mg/day/day; Vamorolone 6.0 mg/day/day
Intervention description: Oral administration of 0.25 mg/kg/day daily for 24 weeks.; Oral administration of 0.75 mg/kg/day daily for 24 weeks.; Oral administration of 2.0 mg/kg/day daily for 24 weeks.; Oral administration of 6.0 mg/kg/day daily for 24 weeks.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Sequential Assignment
Masking: None (Open Label)
Enrollment: 48.0
Sex: Male
Minimum age: 4 Years
Maximum age: 7 Years
Healthy volunteers: No
Keywords: Duchenne muscular dystrophy; vamorolone

Summary:

      The main purposes of this study are to see if it is safe to use a new medication called
      vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see
      if vamorolone works for the treatment for DMD, and to see how any potential side effects
      compare to those seen in boys using steroids.
    

Eligibility criteria:

        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