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Adaptive trials and new designs

Year: 2026

Type: article

Abstract: Neurological therapeutics are advancing rapidly, yet many neurodegenerative diseases still lack effective disease-modifying treatments, in part because traditional trial designs are ill-suited to slowly progressive, heterogeneous disorders. Adaptive methods and multi-arm approaches have improved early-phase efficiency but remain limited when used within isolated trials. Multi-arm multi-stage (MAMS) platform designs offer a more powerful solution by evaluating multiple interventions concurrently against a shared control, incorporating interim analyses to stop futile arms early, and enabling addition of new treatments and biomarkers over time within a single, continuous phase-3-capable infrastructure. Experience from oncology and infectious disease demonstrates the feasibility and impact of these platforms. Emerging neurological MAMS trials - such as MND-SMART, ACT-PD, AD-SMART and OCTOPUS in progressive multiple sclerosis - show that these designs can accelerate evaluation, reduce cost and participant burden, and remain responsive to scientific advances.

Source: Revue Neurologique

DOI: 10.1016/j.neurol.2026.02.155

Authors: C. Wade, S. Apap Mangion, J. Chataway

Institutions: National Hospital for Neurology and Neurosurgery, UCL Queen Square Institute of Neurology, University College London, National Institute for Health and Care Research, UCL Biomedical Research Centre

Language: English

Topic: Statistical Methods in Clinical Trials

Subfield: Statistics and Probability

Field: Mathematics

Domain: Physical Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $3,620