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Phase 2b

The Phase 2b trial is considered the pivotal trial, and will be blinded, placebo-controlled, and include a prednisone arm as well. The EMA requested a prednisone arm to demonstrate superior safety compared to standard of care (prednisone and deflazacort). We anticipate testing two dose groups of vamorolone. There is a six month blinded study, and then a six month extension study where all patients will be treated with vamorolone.

  • Phase 2b (VBP15-004)
    • 6 months daily treatment
    • Randomization to one of four groups: Vamorolone dose 1, vamorolone dose 2, prednisolone, placebo
    • Efficacy of two doses of vamorolone vs. placebo
    • Safety of two doses of vamorolone vs. prednisolone
  • Phase 2b extension (VBP15-005)
    • Six months daily treatment with vamorolone at one of 2 dose levels
    • Long-term tolerability, safety, and efficacy