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Clinical trials in amyotrophic lateral sclerosis : a systematic review and perspective
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(2021) Clinical trials in amyotrophic lateral sclerosis : a systematic review and perspective. Brain Communications . doi:10.1093/braincomms/fcab242 (In Press)
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WRAP-Clinical-trials-amyotrophic-lateral-sclerosis-systematic-review-perspective-2021.pdf - Accepted Version - Requires a PDF viewer. Available under License Creative Commons Attribution 4.0. Download (1328Kb) | Preview |
Official URL: http://dx.doi.org/10.1093/braincomms/fcab242
Abstract
Amyotrophic lateral sclerosis is a progressive and devastating neurodegenerative disease. Despite decades of clinical trials, effective disease modifying drugs remain scarce. To understand the challenges of trial design and delivery, we performed a systematic review of phase II, phase II/III and phase III amyotrophic lateral sclerosis clinical drug trials on trial registries and PubMed between 2008 and 2019. We identified 125 trials, investigating 76 drugs and recruiting more than 15000 people with amyotrophic lateral sclerosis. 90% of trials used traditional fixed designs. The limitations in understanding of disease biology, outcome measures, resources and barriers to trial participation in a rapidly progressive, disabling and heterogenous disease hindered timely and definitive evaluation of drugs in two-arm trials. Innovative trial designs, especially adaptive platform trials may offer significant efficiency gains to this end. We propose a flexible and scalable multi-arm, multi-stage trial platform where opportunities to participate in a clinical trial can become the default for people with amyotrophic lateral sclerosis.
| Item Type: | Journal Article | |||||||||||||||
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| Subjects: | R Medicine > RC Internal medicine | |||||||||||||||
| Divisions: | Faculty of Medicine > Warwick Medical School > Health Sciences Faculty of Medicine > Warwick Medical School > Health Sciences > Statistics and Epidemiology Faculty of Medicine > Warwick Medical School |
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| Library of Congress Subject Headings (LCSH): | Amyotrophic lateral sclerosis, Amyotrphic lateral sclerosis -- Research, Clinical trials | |||||||||||||||
| Journal or Publication Title: | Brain Communications | |||||||||||||||
| Publisher: | Oxford University Press | |||||||||||||||
| ISSN: | 2632-1297 | |||||||||||||||
| Official Date: | 2021 | |||||||||||||||
| Dates: |
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| DOI: | 10.1093/braincomms/fcab242 | |||||||||||||||
| Status: | Peer Reviewed | |||||||||||||||
| Publication Status: | In Press | |||||||||||||||
| Access rights to Published version: | Open Access | |||||||||||||||
| RIOXX Funder/Project Grant: |
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