Clinical trials in amyotrophic lateral sclerosis: a systematic review and perspective.
Wong, Charis Stavrou, Maria Elliott, Elizabeth Gregory, Jenna M Leigh, Nigel Pinto, Ashwin A Williams, Timothy L Chataway, Jeremy Swingler, Robert Parmar, Mahesh K B
Wong, Charis
Stavrou, Maria
Elliott, Elizabeth
Gregory, Jenna M
Leigh, Nigel
Pinto, Ashwin A
Williams, Timothy L
Chataway, Jeremy
Swingler, Robert
Parmar, Mahesh K B
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2021-10-23
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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 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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Wong C, Stavrou M, Elliott E, Gregory JM, Leigh N, Pinto AA, Williams TL, Chataway J, Swingler R, Parmar MKB, Stallard N, Weir CJ, Parker RA, Chaouch A, Hamdalla H, Ealing J, Gorrie G, Morrison I, Duncan C, Connelly P, Carod-Artal FJ, Davenport R, Reitboeck PG, Radunovic A, Srinivasan V, Preston J, Mehta AR, Leighton D, Glasmacher S, Beswick E, Williamson J, Stenson A, Weaver C, Newton J, Lyle D, Dakin R, Macleod M, Pal S, Chandran S. Clinical trials in amyotrophic lateral sclerosis: a systematic review and perspective. Brain Commun. 2021 Oct 23;3(4):fcab242. doi: 10.1093/braincomms/fcab242
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