Outcomes and assessments
NSAA
ADVERSE EVENTS, SECONDARY
Efficacy assessments were performed every 12 weeks. Efficacy was measured by timed function tests, including TTSTAND (primary endpoint), secondary endpoints of TTRW, time to climb 4 stairs (TTCLIMB), North Star Ambulatory Assessment (NSAA), and six-minute walk test (6MWT). The timed results for TTSTAND, TTCLIMB, and TTRW were converted to velocities, and both time and velocity results are presented. The rationale for converting to velocity is to provide a way to include participants who were no longer able to perform the tests mentioned in the analysis for that visit. Efficacy assessments were compared to historical controls (CINRG DNHS), who were group matched for geographic location, age, glucocorticoid use, and ambulatory ability at baseline, defined as all participants being able to execute TTSTAND, TTRW, and TTCLIMB, at baseline. The last time point in which efficacy assessments were available for both the viltolarsen and CINRG DNHS groups was week 205 due to the schedule of CINRG DNHS assessments; therefore, week 205 is the last assessment date in the analysis set. Safety was assessed at each visit or participant contact throughout the duration of the study. Treatment-emergent adverse events (TEAEs) and SAEs were assessed during the safety analysis. Before performing any study-related activities, parents or legal guardians of the participants provided written informed consent and Health Insurance Portability and Accountability Act authorization.