Rationale and design of a multicentre, double-blind, prospective, randomized, European and Canadian study: evaluating patient outcomes and costs of managing adults with post-stroke focal spasticity.

Abstract

OBJECTIVE: This report describes the design of a study aiming to provide evidence for the extended use of botulinum toxin A in focal post-stroke upper and lower limb spasticity and to evaluate the impact of incorporating botulinum toxin treatment into the rehabilitation of patients with spasticity. DESIGN: International, prospective, randomized, double-blind, placebo-controlled study with an open-label extension. METHODS: Approximately 300 adults with a stroke occurring ≥ 3 months before screening, presenting with symptoms and signs of an upper motor neuron syndrome and focal spasticity-related functional impairment, were randomized to botulinum toxin (BOTOX®, Allergan Inc.) + standard care or placebo + standard care. Study medication was administered at baseline and again at Week 12 if required, with follow-up to 52 weeks. The primary endpoint was the number of patients who achieved their investigator-rated principal active functional goal (as measured by Goal Attainment Scaling), at 10 weeks after the second injection (Weeks 22-34) or at the 24-week visit if no second injection was administered. Secondary endpoints included changes from baseline in level of goal achievement, health-related quality of life and resource utilization. CONCLUSION: Botulinum toxin A Economic Spasticity Trial (BEST) will provide information regarding clinical and cost-effectiveness of botulinum toxin + standard care vs standard care alone in patients with upper and/or lower limb post-stroke spasticity typically seen in clinical practice. TRIAL REGISTRATION: ClinicalTrials.gov number NCT-00549783.