Invited Speaker Australian and New Zealand Stroke Organisation Conference 2025

A systematic scoping review of early-phase stroke trials testing multidimensional pharmacological, non-pharmaceutical behavioural, and device-based interventions (122767)

Odkhishig Ganbold 1 , Hannah Johns 2 , Kate Hayward 3 , Bruce Campbell 2 , Emily Dalton 3 , Leonid Churilov 2
  1. Department of Medicine, Royal Melbourne Hospital, The University of Melbourne, Melbourne, Victoria, Australia
  2. Departments of Medicine and Neurology, Melbourne Brain Centre, Royal Melbourne Hospital, The University of Melbourne, Melbourne, Victoria, Australia
  3. Department of Physiotherapy, The University of Melbourne, Melbourne, Victoria, Australia

Background/Aims: Stroke interventions that combine multiple therapies, modes of action or utilise complex dosing regimens (multidimensional interventions) may better address the heterogeneity of stroke. Early-phase stroke trials (Phase I-IIa) establish safety parameters, feasible dose ranges, and promise of efficacy of interventions. Systematic investigation of early-phase stroke trials of multidimensional interventions, and how multidimensionality is addressed at the stage of trial design or analysis, is rare. We aim to (a) identify pharmacological, non-pharmaceutical (behavioural), and device-based early-phase stroke trials of multidimensional interventions, and (b) describe how the multidimensionality of an intervention was dealt with at the trial design and analysis stages.

Methods: This review followed Joanna Briggs Institute and PRISMA-ScR guidelines. We conducted a comprehensive systematic scoping review of early phase acute and recovery stroke trials testing multidimensional pharmacological, non-pharmaceutical (behavioural), and device-based interventions (combination therapies, multidimensional dosing regimens, or multimodal approaches) for all types of stroke. Data on study characteristics, populations, interventions, methodological approaches, and outcomes were systematically synthesised.

Results: From 3660 titles identified, over 300 studies were eligible for analysis. The majority of studies tested combination therapy approaches, while multidimensional dosing interventions appeared to be scarce in the preliminary analysis.

Conclusion: To our knowledge, this scoping review provides the first systematic investigation of early-phase stroke trials testing multidimensional interventions and how multidimensionality is addressed at trial design and analysis stages. Findings will inform the design of complex stroke intervention trials and highlight methodological approaches to guide the evaluation of multidimensional interventions.