The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials

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 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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Item Type:	Article
Authors/Creators:	Genge, A. Cedarbaum, J.M. Shefner, J. Chio, A. Al-Chalabi, A. Van Damme, P. McDermott, C. https://orcid.org/0000-0002-1269-9053 Glass, J. Berry, J. van Eijk, R.P.A. https://orcid.org/0000-0002-7132-5967 Fournier, C. Grosskreutz, J. Andrews, J. Bertone, V. Bunte, T.M. Couillard, M. Cummings, C. Kittle, G. Polzer, J. Salmon, K. Straub, C. van den Berg, L.H.
Copyright, Publisher and Additional Information:	© 2024 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group. This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License (http://creativecommons.org/licenses/by-nc-nd/4.0/), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited, and is not altered, transformed, or built upon in any way. The terms on which this article has been published allow the posting of the Accepted Manuscript in a repository by the author(s) or with their consent.
Keywords:	Clinical trial design; clinical trial outcome measures; functional rating scale; harmonization; statistical analysis; Humans; Amyotrophic Lateral Sclerosis; Severity of Illness Index; Disease Progression
Dates:	Published: 23 February 2024 Published (online): 23 February 2024 Accepted: 12 February 2024 Submitted: 14 December 2023
Institution:	The University of Sheffield
Academic Units:	The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Medicine and Population Health
Depositing User:	Symplectic Sheffield
Date Deposited:	14 Jun 2024 11:43
Last Modified:	14 Jun 2024 11:43
Status:	Published
Publisher:	Informa UK Limited
Refereed:	Yes
Identification Number:	10.1080/21678421.2024.2320880
Related URLs:	PubMed URL
Open Archives Initiative ID (OAI ID):	oai:eprints.whiterose.ac.uk:213414

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The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials

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