Accelerating clinical development of new therapeutics with patient data: evidence from the collaborative Trajectory Analysis Program (cTAP) in DMD A retrospective analysis of studies conducted by cTAP and collaborators over a 6-year period from 2015 –2021. Versions of this poster were presented at WMS 2022, the International Myology Congress 2022, and the International Conference on Duchenne and Becker Muscular Dystrophy, 2023. Authors: C. McDonald, S. Ward, J. Signorovitch, F. Muntoni, N. Goemans, B. Wong, K....

Validation of a composite prognostic score for time to loss of ambulation in Duchenne Muscular Dystrophy An independent validation of a previously developed composite prognostic score for LoA using data the CINRG Duchenne natural history study, which was not used for score development. This poster was presented at WMS 2022 and MDA 2023. Authors: C. McDonald, H. Gordish-Dressman, J. Signorovitch, G. Sajeev, M. Fillbrunn, M. Frean, S. Ward, N. Goemans, K. Vandenborne, E. Mercuri, F....

Genotype-unmatched controls are feasible for drug development in Duchenne Muscular Dystrophy (DMD) This study evaluated the prospect of using genotype-unmatched controls when 1) DMD genotype class effects are small and precisely known and 2) stronger baseline prognostic factors are accounted for in a hypothetical clinical trial for effects on 1-year change in the North Star Ambulatory Assessment total score (ΔNSAA). This poster was presented at WMS 2022 and MDA 2023. Authors: F. Muntoni, J. Signorovitch,...

Development and Evaluation of a Time to Event Endpoint for Clinical Trials in Duchenne Muscular Dystrophy (DMD) Event-driven clinical trials, commonly used in oncology, offer several benefits over continuous outcome measures: i) each patient is assessed individually, ii) after the event has occurred, patients can receive therapy without breaking the blind. This study aimed to develop a time-to-event outcome measure for the North Star Ambulatory Assessment (NSAA) corresponding to clinically meaningful disease progression in DMD....

Consistency of changes in %-predicted forced vital capacity between real-world data and trial placebo arms in ambulatory Duchenne muscular dystrophy To assess risk of bias in the use of real-world and natural history data (RWD/NHD) as external controls, this study compared change in pulmonary function, as measured by percent-predicted forced vital capacity (FVC%p) between RWD/NHD sources and trial placebo arms in ambulatory boys with DMD. This poster was presented at WMS 2022 and MDA 2023....

A Concisely Recorded Ambulatory Assessment for Enhancing Real-World Outcomes Research in Duchenne Muscular Dystrophy: Development and Validation At many centers and practices, the recording of standardized ambulatory assessments in DMD medical records is limited or inconsistent, which hinders retrospective learning from real-word data in this rare, progressive and life-limiting disease. In this study, we evaluated whether consistently recording a concise subset of ambulatory assessments, conducted as part of a complete assessment per care guidelines, could...

A Concisely Recorded Ambulatory Assessment for Enhancing Real-World Outcomes Research in Duchenne Muscular Dystrophy: Development and Validation View the PDF

Predicting Trajectories of Ambulatory Function in Duchenne Muscular Dystrophy View the PDF