Test-Retest Reliability of Motor Function and Myometry Outcomes From the Vamorolone Trials in Duchenne Muscular Dystrophy

Reliable outcome measures, appropriate for the age of patients, are key to improving power in clinical trials, leading to fewer participants needed and reducing patient and site burden. We found NSAA, RWV, and CLIMBV to be the most reliable. This should be considered in the context of sensitivity to drug effect, and clinical meaningfulness of changes observed: all 5 motor outcomes were sensitive to drug effect of vamorolone and prednisone, whereas myometry was not.