Clinical trials in amyotrophic lateral sclerosis: a systematic review and perspective | Zendy

Charis Wong | Zendy; Maria Stavrou | Zendy; Elizabeth Elliott | Zendy; Jenna M. Gregory | Zendy; Nigel Leigh | Zendy; Ashwin Pinto | Zendy; Timothy L. Williams | Zendy; Jeremy Chataway | Zendy; Robert Swingler | Zendy; Mahesh Parmar | Zendy; Nigel Stallard | Zendy; Christopher J. Weir | Zendy; Richard A. Parker | Zendy; Amina Chaouch | Zendy; Hisham Hamdalla | Zendy; John Ealing | Zendy; George Gorrie | Zendy; Ian Morrison | Zendy; Callum Duncan | Zendy; Peter Connelly | Zendy; Francisco Javier Carod-Artal | Zendy; Richard Davenport | Zendy; Pablo Garcia Reitboeck | Zendy; Aleksandar Radunović | Zendy; Venkataramanan Srinivasan | Zendy; Jenny Preston | Zendy; Arpan R. Mehta | Zendy; Danielle Leighton | Zendy; Stella A. Glasmacher | Zendy; Emily Beswick | Zendy; J Williamson | Zendy; Amy Stenson | Zendy; Christine Weaver | Zendy; Judith Newton | Zendy; Dawn Lyle | Zendy; Rachel Dakin | Zendy; Malcolm Macleod | Zendy; Suvankar Pal | Zendy; Siddharthan Chandran | Zendy

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Clinical trials in amyotrophic lateral sclerosis: a systematic review and perspective

Author(s) -

Charis Wong,

Maria Stavrou,

Elizabeth Elliott,

Jenna M. Gregory,

Nigel Leigh,

Ashwin Pinto,

Timothy L. Williams,

Jeremy Chataway,

Robert Swingler,

Mahesh Parmar,

Nigel Stallard,

Christopher J. Weir,

Richard A. Parker,

Amina Chaouch,

Hisham Hamdalla,

John Ealing,

George Gorrie,

Ian Morrison,

Callum Duncan,

Peter Connelly,

Francisco Javier Carod-Artal,

Richard Davenport,

Pablo Garcia Reitboeck,

Aleksandar Radunović,

Venkataramanan Srinivasan,

Jenny Preston,

Arpan R. Mehta,

Danielle Leighton,

Stella A. Glasmacher,

Emily Beswick,

J Williamson,

Amy Stenson,

Christine Weaver,

Judith Newton,

Dawn Lyle,

Rachel Dakin,

Malcolm Macleod,

Suvankar Pal,

Siddharthan Chandran

Publication year - 2021

Publication title -

brain communications

Language(s) - English

Resource type - Journals

ISSN - 2632-1297

DOI - 10.1093/braincomms/fcab242

Subject(s) - amyotrophic lateral sclerosis , perspective (graphical) , clinical trial , medicine , physical medicine and rehabilitation , disease , pathology , computer science , artificial intelligence

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 15 000 people with amyotrophic lateral sclerosis. About 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.

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