Minimal detectable changes in functional measures in Duchenne muscular dystrophy: a study of multiple centers, networks and trial arms
Minimal detectable changes in functional measures in Duchenne muscular dystrophy: a study of multiple centers, networks and trial arms
The design of clinical trials and evaluations of treatment efficacy in DMD require an understanding of the meaningfulness of changes in functional measures. An important concept is the minimal detectable change (MDC), i.e., the minimal magnitude of measured change indicative of true changes due to acquisition or loss of motor function related to the disease, rather than transient variation or measurement error. We used data from six real-world/natural history data sources (RWD/NHD) and eight clinical trial arms to obtain MDC estimates for North Star Ambulatory Assessment (NSAA) total score, 4-stair climb (4SC) completion time and velocity, and 6-minute walk distance (6MWD).
This poster was presented at WMS 2021.
Authors: F. Muntoni, J. Signorovitch, G. Sajeev, N. Done, Z. Yao, N. Goemans, C. McDonald, E. Mercuri, E. Niks, B. Wong, L. Servais, V. Straub, I. de Groot, C. Tian, A. Manzur, K. Vandenborne, I. Dieye, H. Lane, and S. Ward.