The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials
Genge, Angela; Cedarbaum, Jesse M.; Shefner, Jeremy; Chio, Adriano; Al-Chalabi, Ammar; Van Damme, Philip; McDermott, Chris; Glass, Jonathan; Berry, James; van Eijk, Ruben P.A.; Fournier, Christina; Grosskreutz, Julian; Andrews, Jinsy; Bertone, Vanessa; Bunte, Tommy M.; Couillard, Mathias; Cummings, Cathy; Kittle, Gale; Polzer, John; Salmon, Kristiana; Straub, Corey; van den Berg, Leonard H.
(2024) Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, volume 25, issue 3-4, pp. 382 - 387
(Article)
Abstract
The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) was developed more than 25 years ago as an instrument to monitor functional change over time in patients with ALS. It has since been revised and extended to meet the needs of high data quality in ALS trials (ALSFRS-R), however a full
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re-validation of the scale was not completed. Despite this, the scale has remained a primary outcome measure in clinical trials. We convened a group of clinical trialists to discuss and explore opportunities to improve the scale and propose alternative measures. In this meeting report, we present a call to action on the use of the ALSFRS-Revised scale in clinical trials, focusing on the need for (1) harmonization of the ALSFRS-R administration globally, (2) alignment on a set of recommendations for clinical trial design and statistical analysis plans (SAPs), and (3) use of additional outcome measures.
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Keywords: Clinical trial design, clinical trial outcome measures, functional rating scale, harmonization, statistical analysis, Neurology, Clinical Neurology
ISSN: 2167-8421
Publisher: Informa Healthcare
Note: Publisher Copyright: © 2024 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
(Peer reviewed)
