Patient case (clinical description): An 8-year-old boy is brought to the clinic by his parents because of weakness and difficulty of standing up from a sitting position. The mother is healthy but had a brother who died in his 20th after being disabled and using wheelchairs in the last few years of his life. Physical examination shows 3/5 lower extremity muscle strength and enlarged calf muscles. The other physical examination and vital signs are unremarkable. Muscle biopsy showed absence of dystrophin protein. The patient is diagnosed with DMD.

Patient's own account: I brought my  8-year-old son to the doctor's clinic. He was struggling to stand up from a chair for the past few days. My wife is healthy but she was pretty worried since his brother died in his 20s after being disabled and using a wheelchair for the last years of his life. The doctor did an exam which highlighted weakness in the leg muscles and unusually large calf muscles. The rest of the exam was fine, and his vitals were normal. My son did a muscle biopsy, and it showed that the protein important for muscle strength and function is missing. The doctor diagnosed him with Duchenne muscular dystrophy.

Clinical trial: A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Eligibility criteria:
Inclusion Criteria:

          1. Subject's parent(s) or legal guardian(s) has (have) provided written informed consent
             and Health Insurance Portability and Accountability Act (HIPAA) authorization, where
             applicable, prior to any study-related procedures; participants will be asked to give
             written or verbal assent according to local requirements

          2. Subject has a centrally confirmed (by TRiNDS central genetic counselor[s]) diagnosis
             of DMD as defined as:

               -  Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin
                  deficiency, and clinical picture consistent with typical DMD, OR

               -  Identifiable mutation within the DMD gene (deletion/duplication of one or more
                  exons), where reading frame can be predicted as 'out-of-frame,' and clinical
                  picture consistent with typical DMD, OR

               -  Complete dystrophin gene sequencing showing an alteration (point mutation,
                  duplication, other) that is expected to preclude production of the dystrophin
                  protein (i.e., nonsense mutation, deletion/duplication leading to a downstream
                  stop codon), with a clinical picture consistent with typical DMD;

          3. Subject is ≥ 4 years and <7 years of age at time of enrollment in the study;

          4. Subject weighs >13.0 kg and ≤ 39.9 kg at the Screening Visit;

          5. Subject is able to walk independently without assistive devices;

          6. Subject is able to complete the Time to Stand Test (TTSTAND) without assistance in <10
             seconds, as assessed at the Screening Visit;

          7. Clinical laboratory test results are within the normal range at the Screening Visit,
             or if abnormal, are not clinically significant, in the opinion of the Investigator.
             [Notes: Serum gamma glutamyl transferase (GGT), creatinine, and total bilirubin all
             must be ≤ upper limit of the normal range at the Screening Visit. An abnormal vitamin
             D level that is considered clinically significant will not exclude a subject from
             randomization];

          8. Subject has evidence of chicken pox immunity as determined by:

               -  Presence of IgG antibodies to varicella, as documented by a positive test result
                  from the local laboratory from blood collected during the Screening Period, OR

               -  Documentation, provided at the Screening Visit, that the subject has had 2 doses
                  of varicella vaccine, with or without serologic evidence of immunity; the second
                  of the 2 immunizations must have been given at least 14 days prior to
                  randomization.

          9. Subject is able to swallow tablets, as confirmed by successful test swallowing of
             placebo tablets during the Screening Period; and

         10. Subject and parent(s)/guardian(s) are willing and able to comply with scheduled
             visits, study drug administration plan, and study procedures.

        Exclusion Criteria:

          1. Subject has current or history of major renal or hepatic impairment, diabetes mellitus
             or immunosuppression;

          2. Subject has current or history of chronic systemic fungal or viral infections;

          3. Subject has had an acute illness within 4 weeks prior to the first dose of study
             medication;

          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 a history of primary hyperaldosteronism;

          6. Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac
             abnormality on investigation would not be exclusionary];

          7. 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 1 month
             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, unless discontinued for
             intolerance. Inhaled and/or topical glucocorticoids are permitted if last use is at
             least 4 weeks prior to first dose of study medication or if administered at stable
             dose beginning at least 4 weeks prior to first dose of study medication and
             anticipated to be used at the stable dose regimen for the duration of the study];

          8. Subject has an allergy or hypersensitivity to the study medication or to any of its
             constituents;

          9. Subject has used idebenone within 4 weeks prior to the first dose of study medication;

         10. Subject has severe behavioral or cognitive problems that preclude participation in the
             study, in the opinion of the Investigator;

         11. 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;

         12. Subject is taking (or has taken within 4 weeks prior to the first dose of study
             medication) herbal remedies and supplements which can impact muscle strength and
             function (e.g., Co-enzyme Q10, creatine, etc);

         13. Subject is taking (or has taken within 3 months prior to the first dose of study
             medication) any medication indicated for DMD, including Exondys51 and Translarna;

         14. Subject has been administered a live attenuated vaccine within 14 days prior to the
             first dose of study medication;

         15. Subject is currently taking any other investigational drug or has taken any other
             investigational drug within 3 months prior to the first dose of study medication;

         16. Subject has a sibling who is currently enrolled in any vamorolone study or Expanded
             Access Program, or who intends to enroll in any vamorolone study or Expanded Access
             Program during the subject's participation in the VBP15-004 study; or

         17. Subject has previously been enrolled in the study. Note: Any parameter/test may be
             repeated at the Investigator's discretion during Screening to determine
             reproducibility. In addition, subjects may be rescreened if ineligible due to a
             transient condition which would prevent the subject from participating, such as an
             upper respiratory tract infection or injury, or if ineligible due to negative
             anti-varicella IgG antibody test result.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 4 Years old.
Subject must be at most 7 Years

Eligibility (NLI label): Contradiction