Clinical trials of genotype-targeted treatments in Duchenne muscular dystrophy (DMD) traditionally compare treated patients to untreated patients with the same DMD genotype class. This avoids confounding of drug efficacy by genotype effects but also shrinks the pool of eligible controls, increasing challenges for trial enrollment in this already rare disease. To evaluate the suitability of genotypically unmatched controls in DMD, we quantified effects of genotype class on 1-year changes in motor function endpoints used in...
Using external controls based on real-world or natural history data (RWD/NHD) for drug evaluations in Duchenne muscular dystrophy (DMD) is appealing given the challenges of enrolling placebo-controlled trials, especially for multi-year trials. Comparisons to external controls, however, face risks of bias due to differences in outcomes between trial and RWD/NHD settings. Read Abstract or Full Publication
Suitability of external controls for drug evaluation in Duchenne muscular dystrophy Nathalie Goemans, James Signorovitch, Gautam Sajeev, Zhiwen Yao, Heather Gordish-Dressman, Craig M. McDonald, Krista Vandenborne, Debra Miller, Susan J. Ward, Eugenio Mercuri, for the investigators from PRO-DMD-01 Study, CINRG DNHS, ImagingDMD, and the DMD Italian Group View the Full Article
Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collaboration Goemans N, Wong B, Van den Hauwe M, Signorovitch J, Sajeev G, Cox D, et al. (2020). PLoS ONE 11(10): e0164684. doi:10.1371/journal.pone.0164684. View the Full PDF
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta‐analysis of disease progression rates in recent multicenter clinical trials Craig M. McDonald, MD, Gautam Sajeev, ScD, Zhiwen Yao, BA, Erin McDonnell, MS, Gary Elfring, MS, Marcio Souza, PharmD, MBA, Stuart W. Peltz, PhD, Basil T. Darras, MD, Perry B. Shieh, MD, PhD, David A. Cox, PhD, John Landry, MMath, James Signorovitch, PhD, and for the ACT DMD Study Group and the Tadalafil DMD Study Group....
Marden JR, Freimark J, Yao Z, Signorovitch J, Tian C, Wong BL. Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center. J Comp Eff Res. 2020;9(3):177-189. View PDF
Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy Francesco Muntoni, Joana Domingos, Adnan Y. Manzur, Anna Mayhew, Michela Guglieri, The UK NorthStar Network, Gautam Sajeev, James Signorovitch, Susan J. Ward. PLoS One. 2019 Sep 3;14(9):e0221097. doi: 10.1371/journal.pone.0221097. eCollection 2019 View the Full PDF
Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy. Goemans N*, Vanden Hauwe M, Signorovitch J, Swallow E, Song J; Collaborative Trajectory Analysis Project (cTAP). PLoS One. 2016 Oct 13;11(10):e0164684. doi: 10.1371/journal.pone.0164684. eCollection 2016 1 University Hospitals Leuven, Child Neurology, Leuven, Belgium, 2 Analysis Group, Inc., 111 Huntington Ave, 14th floor, Boston, Massachusetts, United States of America, 3 The Trajectory Analysis Project (TAP) Collaboration, One Broadway, 14th floor, Cambridge, Massachusetts,…
Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy. Mercuri E, Signorovitch JE, Swallow E, Song J, Ward SJ; DMD Italian Group; Trajectory Analysis Project (cTAP). Neuromuscul Disord. 2016 Sep;26(9):576-83. doi: 10.1016/j.nmd.2016.05.016. Epub 2016 May 27 Eugenio Mercuri a,*, James Edward Signorovitch b,c, Elyse Swallow b, Jinlin Song b, Susan J. Ward c for the DMD Italian Group # and the collaborative Trajectory Analysis Project…
