ABSTRACT
Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin. In ongoing delandistrogene moxeparvovec Studies 101 and 102, remote functional assessment was initiated during the COVID-19 pandemic, in accordance with US Food and drug administration guidance. To evaluate the reliability of remote assessment of functional measures versus in-person testing, we assessed the reproducibility of remote North Star Ambulatory Assessment (NSAA), 10-metre Walk/Run (10MWR), and Time to Rise scores against in-person scores using pre-specified statistical analyses—including intraclass correlation coefficient (ICC), Pearson, Spearman, and Bland-Altman analyses. Preliminary results from eight patients with Duchenne muscular dystrophy (DMD), who completed an in-clinic assessment within 2 weeks of a remote assessment in Part 1 of Study 102, found strong correlations between remote and in-person NSAA scores (ICC=0.97 [95% confidence interval 0.87–0.99];Pearson=0.98 [95% CI 0.88–1.00];Spearman=0.93 [95% CI 0.66–0.99]). Analysis of all eight patients from Part 1 of Study 102 showed no statistical or clinical differences in NSAA scores attained remotely versus in person. Results from additional patients in Studies 101 and 102 (Parts 1 and 2), including correlations between remote and in-person scores on the 10MWR and time to rise, will also be presented. These findings suggest that remote functional assessment of patients with DMD is not statistically different from in-person assessment and has comparable clinical meaningfulness, validating its use in clinical trials of delandistrogene moxeparvovec. Given the burden that treatment and monitoring place on patients with DMD and their caregivers, remote assessment may be beneficial in future research, clinical trials, and clinical settings. [ FROM AUTHOR] Copyright of Neuromuscular Disorders is the property of Elsevier B.V. and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full . (Copyright applies to all s.)