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Improved ALS clinical trials through frequent at‐home self‐assessment: a proof of concept study
OBJECTIVE: To determine the potential for improving amyotrophic lateral sclerosis (ALS) clinical trials by having patients or caregivers perform frequent self‐assessments at home. METHODS AND PARTICIPANTS: We enrolled ALS patients into a nonblinded, longitudinal 9‐month study in which patients and c...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7359124/ https://www.ncbi.nlm.nih.gov/pubmed/32515889 http://dx.doi.org/10.1002/acn3.51096 |
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author | Rutkove, Seward B. Narayanaswami, Pushpa Berisha, Visar Liss, Julie Hahn, Shira Shelton, Kerisa Qi, Kristin Pandeya, Sarbesh Shefner, Jeremy M. |
author_facet | Rutkove, Seward B. Narayanaswami, Pushpa Berisha, Visar Liss, Julie Hahn, Shira Shelton, Kerisa Qi, Kristin Pandeya, Sarbesh Shefner, Jeremy M. |
author_sort | Rutkove, Seward B. |
collection | PubMed |
description | OBJECTIVE: To determine the potential for improving amyotrophic lateral sclerosis (ALS) clinical trials by having patients or caregivers perform frequent self‐assessments at home. METHODS AND PARTICIPANTS: 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. RESULTS: 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. Participants generally found the procedures acceptable and, for many, improved their sense of control of their disease. INTERPRETATION: Frequent at‐home measurements using standard tools holds the prospect of tracking progression and reducing sample size requirements for clinical trials in ALS while also being acceptable to the patients. Future studies in this and other neurological disorders should consider adopting this approach to data collection. |
format | Online Article Text |
id | pubmed-7359124 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-73591242020-07-17 Improved ALS clinical trials through frequent at‐home self‐assessment: a proof of concept study Rutkove, Seward B. Narayanaswami, Pushpa Berisha, Visar Liss, Julie Hahn, Shira Shelton, Kerisa Qi, Kristin Pandeya, Sarbesh Shefner, Jeremy M. Ann Clin Transl Neurol Research Articles OBJECTIVE: To determine the potential for improving amyotrophic lateral sclerosis (ALS) clinical trials by having patients or caregivers perform frequent self‐assessments at home. METHODS AND PARTICIPANTS: 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. RESULTS: 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. Participants generally found the procedures acceptable and, for many, improved their sense of control of their disease. INTERPRETATION: Frequent at‐home measurements using standard tools holds the prospect of tracking progression and reducing sample size requirements for clinical trials in ALS while also being acceptable to the patients. Future studies in this and other neurological disorders should consider adopting this approach to data collection. John Wiley and Sons Inc. 2020-06-09 /pmc/articles/PMC7359124/ /pubmed/32515889 http://dx.doi.org/10.1002/acn3.51096 Text en © 2020 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
spellingShingle | Research Articles Rutkove, Seward B. Narayanaswami, Pushpa Berisha, Visar Liss, Julie Hahn, Shira Shelton, Kerisa Qi, Kristin Pandeya, Sarbesh Shefner, Jeremy M. Improved ALS clinical trials through frequent at‐home self‐assessment: a proof of concept study |
title | Improved ALS clinical trials through frequent at‐home self‐assessment: a proof of concept study |
title_full | Improved ALS clinical trials through frequent at‐home self‐assessment: a proof of concept study |
title_fullStr | Improved ALS clinical trials through frequent at‐home self‐assessment: a proof of concept study |
title_full_unstemmed | Improved ALS clinical trials through frequent at‐home self‐assessment: a proof of concept study |
title_short | Improved ALS clinical trials through frequent at‐home self‐assessment: a proof of concept study |
title_sort | improved als clinical trials through frequent at‐home self‐assessment: a proof of concept study |
topic | Research Articles |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7359124/ https://www.ncbi.nlm.nih.gov/pubmed/32515889 http://dx.doi.org/10.1002/acn3.51096 |
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