Adaptive trials and new designs - 12/05/26

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.
Le texte complet de cet article est disponible en PDF.Keywords : Trial design, Multiple sclerosis
Plan
Vol 182 - N° 5
P. 428-432 - mai 2026 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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