P.336Vision DMD: a phase IIb randomized, double-blind, parallel group, placebo- and active-controlled study to assess the efficacy and safety of vamorolone in boys with Duchenne muscular dystrophy

Vamorolone is a first-in-class steroid that shows the anti-inflammatory efficacy of glucocorticoids while reducing adverse effects of glucocorticoids. Vamorolone, unlike glucocorticoids (prednisone and deflazacort), also shows mineralocorticoid receptor antagonist activity and membrane stabilizing properties. VBP15-004 is a Phase IIb, randomised, double blind, parallel group, placebo and active (prednisone 0.75mg/kg/day) controlled study of two doses of vamorolone in 120 steroid naïve, ambulant DMD boys aged 4 to <7 years. The study primary objectives are to evaluate safety and tolerability of daily doses of vamorolone of 2.0 and 6.0 mg/kg/day and to compare its efficacy (Time to Stand from supine) over a 24-week treatment period vs. placebo. Secondary objectives include additional measures of strength and function (efficacy), and measures of safety. The study includes innovative outcome measures assessing feasibility and clinical relevance for use in future clinical trials including: exploratory safety and efficacy pharmacodynamic biomarkers; novel evaluation of the impact of vamorolone and prednisone on bone health and turnover; mobile health band study investigating the feasibility of wearable healthcare technology for recording activity data; innovative application program for consenting/assenting patients designed to provide information to young children about research and the clinical trial. The study, which opened in August 2018, has been designed in collaboration with patient organisations and is being conducted in 34 sites across 11 countries (USA, Canada, UK, Israel, Australia, Sweden, Czech Republic, Belgium, Spain, Greece, Netherlands). Vamorolone has been developed by ReveraGen Biopharma under a venture philanthropy model, in partnership with international non-profit foundations, and the US and EU governments.