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Phase 2a

The Phase 2a trials will be carried out in established CINRG sites in the US, Canada, Sweden, Israel, UK and Australia. The trials are unblinded, and all DMD boys will be treated with vamorolone (there is no placebo arm).

The Phase 2a trials are in two parts with different purposes as outlined below:

  • Phase 2a (VBP15-002)
    • Two weeks daily treatment with vamorolone at one of 4 dose levels (0.25, 0.75, 2.0, 6.0 mg/kg/day
    • Pharmacokinetics (drug metabolism), and comparison of DMD children to completed Phase 1 adult studies.
    • Short-term tolerability and safety, and comparison of DMD children to Phase 1 adults
  • Phase 2a extension (VBP15-003) 
    • Six months daily treatment with vamorolone at one of 4 dose levels
    • Long-term tolerability and safety
    • Dose-finding
      • Which of the four doses show any safety signals?
        • Tested by pharmacodynamic biomarkers for adrenal suppression, bone turnover, insulin resistance, and immune suppression
        • Tested for changes in body mass index (weight gain)
        • Are the findings in DMD children same or different to Phase 1 adults?
      • Which of the four doses show hints of efficacy (benefit)?
        • Tested by improvements in timed function tests (time to stand from floor, and others)
        • Tested by exploratory pharmacodynamic efficacy biomarkers

With completion of the Phase 2a studies, we hope to have a very good understanding of the degree to which improvements in safety are seen, and a comparison of DMD children and the completed Phase 1 adult studies. We also hope to have hints of doses that show clinical improvement of DMD boys, and these doses will be carried forward into the pivotal Phase 2b studies.