Long-term 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 months.; Oral administration of 0.75 mg/kg/day daily for 24 months.; Oral administration of 2.0 mg/kg/day daily for 24 months.; Oral administration of 6.0 mg/kg/day daily for 24 months.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 46.0
Sex: Male
Minimum age: 4 Years
Maximum age: 7 Years
Healthy volunteers: No
Keywords: Duchenne muscular dystrophy; vamorolone

Summary:

      This long-term extension study is an open-label, multiple-dose study to evaluate the
      long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by
      liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who
      participated in the VBP15-002 Phase IIa and VBP15-003 Phase IIa extension core studies.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Subject's parent or legal guardian has provided written informed consent and HIPAA
             authorization (if applicable) prior to any VBP15-LTE long-term extension
             study-specific procedures;

          2. Subject has previously completed study VBP15-003 up to and including the Week 24 Final
             assessments, prior to enrolling in the VBP15-LTE study at the conclusion of the
             VBP15-003 Week 24 Visit [Note: if entering the dose-tapering period, subject is
             enrolling within 8 weeks after the VBP15-003 final visit following dose-tapering]; and

          3. Subject and parent/guardian are willing and able to comply with scheduled visits,
             study drug administration plan, and study procedures.

        Exclusion Criteria:

          1. Subject had a serious or severe adverse event in study VBP15-003 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 long-term extension study;

          2. Subject has current or history of major renal or hepatic impairment, diabetes mellitus
             or immunosuppression;

          3. Subject has current or history of chronic systemic fungal or viral infections;

          4. Subject 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. Subject has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac
             abnormality on investigation would not be exclusionary];

          6. Subject 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
             glucocorticoids 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. Subject has an allergy or hypersensitivity to the study medication or to any of its
             constituents;

          9. Subject has severe behavioral or cognitive problems that preclude participation in the
             study, in the opinion of the Investigator;

         10. Subject 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

         11. Subject 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: Subjects may be re-evaluated if ineligible due to a transient condition which would
        prevent the subject from participating.