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Review
. 2025 Jan;56(1):198-208.
doi: 10.1161/STROKEAHA.124.045754. Epub 2024 Dec 20.

Adaptive Platform Trials in Stroke

Elizabeth Lorenzi  1 , Amy M Crawford  1 , Craig S Anderson  2   3 , Bijoy Menon  4 , Xiaoying Chen  2 , Eva Mistry  5 , Pooja Khatri  5 , Jordan J Elm  6 , Jonathan Beall  6 , Benjamin R Saville  7   8 , Scott M Berry  1 , Roger J Lewis  1   9
Affiliations
Review

Adaptive Platform Trials in Stroke

Elizabeth Lorenzi et al. Stroke. 2025 Jan.

Abstract

Clinical trials of treatments for stroke have generally utilized 2-arm, randomized designs to evaluate a single intervention against a control. Running separate clinical trials, with each addressing a single therapeutic question, is resource intensive and slows evidence generation, especially in a field with rapidly expanding treatment options and evolving practices. Platform trials-randomized clinical trials designed to evaluate multiple interventions that may enter and exit the ongoing platform based on a master protocol-accelerate the investigation of multiple therapeutic options within a single infrastructure. This in turn has the potential to accelerate access to new interventions for patients with stroke that can save lives and improve outcomes. In the context of acute ischemic stroke, 2 new platform trials have been established, the STEP trial (StrokeNet Thrombectomy Endovascular Platform) and ACT-GLOBAL (A Multi-Factorial, Multi-Arm, Multi-Stage, Randomised, Global Adaptive Platform Trial for Stroke), to address multiple therapeutic questions simultaneously using a multifactorial design including Bayesian modeling and other adaptive features. These trials are designed to maximize the information obtained from each participant, to align clinical research more closely with the complexities of clinical care, and to accelerate the identification of effective therapies. This article explores conceptual, practical, and statistical considerations in the design and implementation of adaptive platform trials and highlights their potential to accelerate the identification of new therapies, management, and rehabilitation in stroke.

Keywords: Bayesian analysis; adaptive clinical trials; randomized control trials; research design; stroke.

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Conflict of interest statement

Drs Lorenzi and Crawford report employment by Berry Consultants LLC. Dr Anderson reports grants from the National Health and Medical Research Council, compensation from AstraZeneca Australia for consultant services, and grants from Medical Research Foundation of the United Kingdom to other. Dr Menon reports compensation from Boehringer Ingelheim for consultant services, employment by the University of Calgary, and stock options in Neurostasis. E. Mistry reports grants from the National Institute of Neurological Disorders and Stroke; compensation from the American Heart Association for consultant services; compensation from AbbVie for consultant services; grants from the Patient-Centered Outcomes Research Institute; compensation from Silver Creek Pharmaceuticals, Inc, for other services; compensation from Translational Sciences for other services; grants from the Society of Vascular and Interventional Neurology; compensation from RAPID AI for consultant services; grants from the National Institutes of Health; and employment by the University of Cincinnati. Drs Khatri, Elm, and Beall report grants from the National Institutes of Health. Dr Saville reports an ownership stake in Adaptix Trials. Dr Berry reports part ownership of Consultants LLC. Dr Lewis reports employment by the Journal of the American Medical Association and employment by Berry Consultants LLC. Berry Consultants LLC is a consulting company specializing in the design, conduct, oversight, and analysis of adaptive and platform clinical trials. Employees of Berry Consultants are involved in the design of STEP (StrokeNet Thrombectomy Endovascular Platform) and ACT-GLOBAL (A Multi-Factorial, Multi-Arm, Multi-Stage, Randomised, Global Adaptive Platform Trial for Stroke), which are referenced in this review. The other author reports no conflicts.

References

    1. Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research at the U.S. Food and Drug Administration. Adaptive Designs for Clinical Trials of Drugs and Biologics: Guidance for Industry [Internet]. 2019. [cited 2024 November 14]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents...
    1. Crawford AM, Lorenzi EC, Saville BR, Lewis RJ, Anderson CS. Adaptive Clinical Trials in Stroke. Stroke. 2024;55:2731–2741 - PubMed
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