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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.

  • Phase 2b (VBP15-004)
    • Period 1: 24 weeks. 50% DMD patients vamorolone (2 doses), 25% placebo, 25% prednisone
    • Period 2: 24 weeks. 100% patients vamorolone (2 doses)
    • 120 DMD boys, 4 to <7 years, not previously treated with steroids
    • Visits: Designed with DMD parent involvement, burden kept to minimum. ~1 visit per month
    • Dosing: Daily by mouth in morning at home