Adaptive Platform Trials in Stroke

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.