Abstract-Text (inkl. Referenzen)

Vamorolone is a dissociative steroidal anti-inflammatory drug that seeks to retain efficacy and reduce safety concerns in patients with DMD compared to corticosteroids. VISION-DMD is a 48-week RCT consisting of two 24-week periods to evaluate efficacy and safety of continuous 48-week vamorolone treatment. During p1, 121 patients were randomized 1:1:1:1 to vamorolone 2 or 6 mg/kg/day, prednisone 0.75 mg/kg/day or placebo for 24 weeks. During p2, patients continued their initial vamorolone dose or crossed over to vamorolone 2 or 6 mg/kg/day. For the continuous vamorolone groups, efficacy of vamorolone 6 mg/kg/day established at 24 weeks was maintained over 48 weeks across all outcome measures, while only across some measures for 2 mg/kg/day. There was no loss of efficacy after switching from prednisone to vamorolone 6 mg/kg/day. Three serious adverse events were reported during the 48 weeks, all considered unrelated to vamorolone. Stunting of growth was not seen and body mass index stabilized after an initial increase during the first 24 weeks. Subjects switching from prednisone to vamorolone resumed normal growth, and experienced reductions in select other side effects, as well as reduced impact on bone biomarkers. Vamorolone treatment was generally well tolerated on both dose levels throughout 48 weeks. On behalf of the VISION-DMD investigators.