Safety and efficacy of multipotent adult progenitor cells in acute ischaemic stroke (MASTERS): a randomised, double-blind, placebo-controlled, phase 2 trial.

Background: Multipotent adult progenitor cells are a bone marrow-derived, allogeneic, cell therapy product that modulates the immune system, and represents a promising therapy for acute stroke. We aimed to identify the highest, well-tolerated, and safest single dose of multipotent adult progenitor cells, and if they were efficacious as a treatment for stroke recovery.

Methods: We did a phase 2, randomised, double-blind, placebo-controlled, dose-escalation trial of intravenous multipotent adult progenitor cells in 33 centres in the UK and the USA.

The challenges of conducting clinical endpoint studies.

In order to evaluate the effects of a particular treatment strategy on mortality and major morbidity within a disease entity, large, multinational, relatively long-term clinical endpoint studies are often conducted. The primary challenge of conducting these studies is to maintain consistency in the interpretation of the clinical endpoints across different geographic areas and over the long time course of the study. The success of a clinical endpoint study depends on understanding the challenges and incorporating the special requirements of these studies into the protocol design and operational procedures throughout the study.