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
cTAP founders Susan J. Ward and James Signorovitch have written an article featured in Genetic Engineering & Biotechnology News: The Problem with Clinical Trials in Duchenne—and What We Can Do About It. View the Full Article
Susan J. Ward presents at the RE(ACT) CONGRESS and IRDIRC Conference. The talk is entitled “Learning from natural history patient data to drive smaller, faster, trials – a case study in Duchenne Muscular Dystrophy (DMD).” View the PDF Abstract
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
Results published in peer-reviewed journal Neurology indicate that real-world data (RWD) and natural history data (NHD) could augment or replace placebo controls in DMD clinical trials. Use of RHD and NHD have potential to streamline and accelerate clinical programs targeting new treatments for DMD. CAMBRIDGE, Mass., September 8, 2020 /PRNewswire/ – The Collaborative Trajectory Analysis Project (cTAP), a multi-stakeholder, global research coalition in Duchenne muscular dystrophy (DMD), today announced results from the largest ever multi-national,…
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
