Improved ALS clinical trials through frequent at-home self-assessment: a proof of concept study.

Annals of Clinical and Translational Neurology
Seward B RutkoveJeremy M Shefner

Abstract

To determine the potential for improving amyotrophic lateral sclerosis (ALS) clinical trials by having patients or caregivers perform frequent self-assessments at home. We enrolled ALS patients into a nonblinded, longitudinal 9-month study in which patients and caregivers obtained daily data using several different instruments, including a slow-vital capacity device, a hand grip dynamometer, an electrical impedance myography-based fitness device, an activity tracker, a speech app, and the ALS functional rating scale-revised. Questions as to acceptability were asked at two time points. A total of 113 individuals enrolled, with 61 (43 men, 18 women, mean age 60.1 ± 9.9 years) collecting a minimum of 7 days data and being included in the analysis. Daily measurements resulted in more accurate assessments of the slope of progression of the disease, resulting in smaller sample size estimates for a hypothetical clinical trial. For example, by performing daily slow-vital capacity measurements, calculated sample size was reduced to 182 subjects/study arm from 882/arm for monthly measurements. Similarly, performing the ALS functional rating scale weekly rather than monthly led to a calculated sample size of 73/arm as compared to 274/arm....Continue Reading

References

Mar 3, 1994·The New England Journal of Medicine·G BensimonV Meininger
May 20, 2017·Lancet Neurology·UNKNOWN Writing Group, UNKNOWN Edaravone (MCI-186) ALS 19 Study Group
Sep 29, 2018·Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration·Jeremy M ShefnerJose L Bohorquez
Nov 30, 2018·Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration·Seward B RutkoveJeremy M Shefner
Jan 22, 2019·Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration·R S BedlackGhazaleh Sadri-Vakili
May 14, 2019·Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration·Jeremy M ShefnerUNKNOWN VITALITY-ALS Study Group
Jan 4, 2020·Proceedings of the ... IEEE International Conference on Acoustics, Speech, and Signal Processing·Jacob PeplinskiKerisa Shelton

❮ Previous
Next ❯

Citations

Sep 19, 2020·Annals of Clinical and Translational Neurology·Seward B RutkoveJeremy M Shefner
Sep 19, 2020·Annals of Clinical and Translational Neurology·Ruben P A van Eijk
Apr 27, 2021·Clinical Neurophysiology : Official Journal of the International Federation of Clinical Neurophysiology·Seward B RutkoveJeremy M Shefner
Jul 25, 2021·The Cerebellum·Nergis C KhanAnoopum S Gupta
Aug 6, 2021·Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration·Gabriela M StegmannVisar Berisha
Aug 7, 2021·Annals of Clinical and Translational Neurology·Arne MuellerC J Malanga
Aug 10, 2021·Annals of Clinical and Translational Neurology
Jan 12, 2022·Seminars in Neurology·Anoopum S Gupta

❮ Previous
Next ❯

Software Mentioned

REDCap
Google Fit
Aural Analytics
ALS AT HOME
Air
Google Ads

Related Concepts

Related Feeds

Amyloid Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS) is a progressive nervous system disease associated with the death of neurons that control voluntary muscles. Discover the latest research on ALS here.