Application of the ENCALS predictive survival model in assessing the effect of the 24/44 inclusion criteria in FORTITUDE-ALS

Tyrell Simkins Lead; Jeremy M Shefner; Stuart Kupfer; Fady I Malik; Lisa Meng; Stacy A Rudnicki; Jenny Wei; Ruben Pa van Eijk

doi:10.1177/22143602251336058

Application of the ENCALS predictive survival model in assessing the effect of the 24/44 inclusion criteria in FORTITUDE-ALS

Tyrell Simkins Lead^*
, Jeremy M Shefner
, Stuart Kupfer
, Fady I Malik
, Lisa Meng
, Stacy A Rudnicki
, Jenny Wei
, Ruben Pa van Eijk

^*Corresponding author for this work

Research output: Contribution to journal › Letter › Academic › peer-review

46 Downloads (Pure)

Abstract

FORTITUDE-ALS was a study evaluating reldesemtiv in people living with ALS. Post-hoc analysis identified larger treatment effects in those with symptom onset ≤24 months and baseline ALSFRS-R ≤ 44 (24/44 criteria). Using the ENCALS risk score (RS), we analyzed how the 24/44 criteria changed the eligible population. Of the 272 participants meeting the 24/44 criteria, 73% had very short to intermediate RS compared to 18% not meeting the criteria. Though the 24/44 criteria enriched the FORTITUDE-ALS population with rapidly progressing patients, they did not completely exclude all patients with a very long predicted survival.

Original language	English
Pages (from-to)	679-682
Number of pages	4
Journal	Journal of Neuromuscular Diseases
Volume	12
Issue number	5
Early online date	5 May 2025
DOIs	https://doi.org/10.1177/22143602251336058
Publication status	Published - Sept 2025

Keywords

ALS
ENCALS survival prediction model
FORTITUDE-ALS
clinical trials design

Access to Document

10.1177/22143602251336058Licence: CC BY-NC

simkins-(lead)-et-al-2025-application-of-the-encals-predictive-survival-model-in-assessing-the-effect-of-the-24-44Final published version, 210 KBLicence: CC BY-NC

Cite this

@article{c888e49152e145a3a9fbd55c6fb00e72,

title = "Application of the ENCALS predictive survival model in assessing the effect of the 24/44 inclusion criteria in FORTITUDE-ALS",

abstract = "FORTITUDE-ALS was a study evaluating reldesemtiv in people living with ALS. Post-hoc analysis identified larger treatment effects in those with symptom onset ≤24 months and baseline ALSFRS-R ≤ 44 (24/44 criteria). Using the ENCALS risk score (RS), we analyzed how the 24/44 criteria changed the eligible population. Of the 272 participants meeting the 24/44 criteria, 73\% had very short to intermediate RS compared to 18\% not meeting the criteria. Though the 24/44 criteria enriched the FORTITUDE-ALS population with rapidly progressing patients, they did not completely exclude all patients with a very long predicted survival.",

keywords = "ALS, ENCALS survival prediction model, FORTITUDE-ALS, clinical trials design",

author = "\{Simkins Lead\}, Tyrell and Shefner, \{Jeremy M\} and Stuart Kupfer and Malik, \{Fady I\} and Lisa Meng and Rudnicki, \{Stacy A\} and Jenny Wei and \{van Eijk\}, \{Ruben Pa\}",

note = "Publisher Copyright: {\textcopyright} The Author(s) 2025. This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (https://creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access page (https://us.sagepub.com/en-us/nam/open-access-at-sage).",

year = "2025",

month = sep,

doi = "10.1177/22143602251336058",

language = "English",

volume = "12",

pages = "679--682",

journal = "Journal of Neuromuscular Diseases",

issn = "2214-3599",

publisher = "SAGE Publications Ltd",

number = "5",

}

TY - JOUR

T1 - Application of the ENCALS predictive survival model in assessing the effect of the 24/44 inclusion criteria in FORTITUDE-ALS

AU - Simkins Lead, Tyrell

AU - Shefner, Jeremy M

AU - Kupfer, Stuart

AU - Malik, Fady I

AU - Meng, Lisa

AU - Rudnicki, Stacy A

AU - Wei, Jenny

AU - van Eijk, Ruben Pa

N1 - Publisher Copyright: © The Author(s) 2025. This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (https://creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access page (https://us.sagepub.com/en-us/nam/open-access-at-sage).

PY - 2025/9

Y1 - 2025/9

N2 - FORTITUDE-ALS was a study evaluating reldesemtiv in people living with ALS. Post-hoc analysis identified larger treatment effects in those with symptom onset ≤24 months and baseline ALSFRS-R ≤ 44 (24/44 criteria). Using the ENCALS risk score (RS), we analyzed how the 24/44 criteria changed the eligible population. Of the 272 participants meeting the 24/44 criteria, 73% had very short to intermediate RS compared to 18% not meeting the criteria. Though the 24/44 criteria enriched the FORTITUDE-ALS population with rapidly progressing patients, they did not completely exclude all patients with a very long predicted survival.

AB - FORTITUDE-ALS was a study evaluating reldesemtiv in people living with ALS. Post-hoc analysis identified larger treatment effects in those with symptom onset ≤24 months and baseline ALSFRS-R ≤ 44 (24/44 criteria). Using the ENCALS risk score (RS), we analyzed how the 24/44 criteria changed the eligible population. Of the 272 participants meeting the 24/44 criteria, 73% had very short to intermediate RS compared to 18% not meeting the criteria. Though the 24/44 criteria enriched the FORTITUDE-ALS population with rapidly progressing patients, they did not completely exclude all patients with a very long predicted survival.

KW - ALS

KW - ENCALS survival prediction model

KW - FORTITUDE-ALS

KW - clinical trials design

U2 - 10.1177/22143602251336058

DO - 10.1177/22143602251336058

M3 - Letter

C2 - 40324960

SN - 2214-3599

VL - 12

SP - 679

EP - 682

JO - Journal of Neuromuscular Diseases

JF - Journal of Neuromuscular Diseases

IS - 5

ER -

Application of the ENCALS predictive survival model in assessing the effect of the 24/44 inclusion criteria in FORTITUDE-ALS

Abstract

Keywords

Access to Document

Fingerprint

Cite this