Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study
Fig 1
Participant-level longitudinal data and aggregated cross-sectional data comparing vamorolone-associated efficacy to CINRG DNHS external comparators.
Left panels show participant-level change from baseline after an 18-month treatment period. Vamorolone group A was treated with 2.0 or 6.0 mg/kg/day for the last 3–9 months of the 18-month period, group B was treated with 2.0 or 6.0 mg/kg/day for the last 9–11 months, and groups C and D with 2.0 or 6.0 mg/kg/day for all 18 months. The specific dose of each participant at the end of the 18-month period is indicated (red = 2.0 mg/kg/day; blue = 6.0 mg/kg/day). Dose groups B, C, and D show mean improvements over baseline compared to matched corticosteroid-naïve participants from CINRG DNHS (n = 19). Right panels show mean group cross-sectional analysis at age 5.5–8.5 years. The baseline mean is shown for each vamorolone-treated group (black line). The corticosteroid-treated natural history group (n = 68) has no baseline shown, as the age at initiation of corticosteroids was variable. This panel shows improvement over baseline in vamorolone-treated groups B, C, and D, with the cross-sectional data suggesting an effect size similar to that of age-group-matched corticosteroid-treated participants in CINRG DNHS. CINRG, Cooperative International Neuromuscular Research Group; DNHS, Duchenne Natural History Study; SEM, standard error of the mean; TTCLIMB, time to climb 4 stairs; TTRW, time to run/walk 10 meters; TTSTAND, time to stand from supine.