The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials
Publication date
2024-05
Authors
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.
Editors
Advisors
Supervisors
Document Type
Article
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cc_by_nc_nd
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.
Keywords
Clinical trial design, clinical trial outcome measures, functional rating scale, harmonization, statistical analysis, Neurology, Clinical Neurology
Citation
Genge, A, Cedarbaum, J M, Shefner, J, Chio, A, Al-Chalabi, A, Van Damme, P, McDermott, C, Glass, J, Berry, J, van Eijk, R P A, 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 2024, 'The ALSFRS-R Summit : a global call to action on the use of the ALSFRS-R in ALS clinical trials', Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol. 25, no. 3-4, pp. 382-387. https://doi.org/10.1080/21678421.2024.2320880