Speech-Based Digital Endpoints Track ALS Progression and Align with Standard Clinical Outcomes: Evidence from the VRG50635 Trial

Speech-Based Digital Endpoints Track ALS Progression and Align with Standard Clinical Outcomes: Evidence from the VRG50635 Trial | Research Square window.SnipcartSettings = { analytics: { enabled: false } }; (function() { var accessVector = localStorage.getItem('access_vector') || ''; window.dataLayer = window.dataLayer || []; if (accessVector) { window.dataLayer.push({ user: { profile: { profileInfo: { snid: accessVector } } } }); } })(); (function(w,d,s,l,i){w[l]=w[l]||[];w[l].push({'gtm.start':new Date().getTime(),event:'gtm.js'});var f=d.getElementsByTagName(s)[0],j=d.createElement(s),dl=l!='dataLayer'?'&l='+l:'';j.async=true;j.src='https://www.googletagmanager.com/gtm.js?id='+i+dl;f.parentNode.insertBefore(j,f);})(window,document,'script','dataLayer','GTM-K279D39R'); Browse Preprints In Review Journals COVID-19 Preprints AJE Video Bytes Research Tools Research Promotion AJE Professional Editing AJE Rubriq About Preprint Platform In Review Editorial Policies Our Team Advisory Board Help Center Sign In Submit a Preprint Cite Share Download PDF Article Speech-Based Digital Endpoints Track ALS Progression and Align with Standard Clinical Outcomes: Evidence from the VRG50635 Trial Michael Neumann, Hardik Kothare, Meredith Bartlett, Oliver Roesler, and 20 more This is a preprint; it has not been peer reviewed by a journal. https://doi.org/ 10.21203/rs.3.rs-8229857/v1 This work is licensed under a CC BY 4.0 License Status: Posted Version 1 posted You are reading this latest preprint version Abstract We report on the clinical validity of speech-based digital endpoints measured during a Phase 1b study of VRG50635 in Amyotrophic Lateral Sclerosis (ALS). Fifty-four participants with ALS were enrolled and participated in an 8-week pretreatment run-in, followed by three 8-week dosing periods and an 8-week follow-up. They completed a speech assessment every two weeks in the clinic or at home. We observed moderate to high correlations between digital measures of speech timing and articulatory motor function and traditional clinical outcome measures -- such as the ALS Functional Rating Scale-Revised, slow vital capacity and plasma neurofilament light chain. Furthermore, speech measures can show functional decline before the ALSFRS-R does, while also capturing differences between participants with bulbar symptoms and those without. The results support the feasibility, utility, and validity of digital speech endpoints to study disease impact in ALS clinical trials. Health sciences/Medical research Health sciences/Neurology Biological sciences/Neuroscience Full Text Additional Declarations Competing interest reported. MN, HK, LA, DP, VR, MB, OR, CSO, DSO, and AH are, or were at the time of the study, employees of Modality.AI. DC, RHS, IH, AT, and TH are, or were at the time of the study, employees of Verge Genomics. SR, PVD, PT, MJ, AG, CO, RVE, and LVDB declare no competing interests. Cite Share Download PDF Status: Posted Version 1 posted You are reading this latest preprint version Research Square lets you share your work early, gain feedback from the community, and start making changes to your manuscript prior to peer review in a journal. As a division of Research Square Company, we’re committed to making research communication faster, fairer, and more useful. We do this by developing innovative software and high quality services for the global research community. Our growing team is made up of researchers and industry professionals working together to solve the most critical problems facing scientific publishing. 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