Design and Implementation of a Multicenter Trial of Vitamin E in Aging Individuals with Down Syndrome

Older individuals with Down syndrome have an extremely high risk of Alzheimer's disease (AD). Advances in understanding the pathophysiology of AD, and the development of effective therapies for individuals with sporadic AD, have raised the possibility that aging Down syndrome individuals may benefit from therapy directed against AD. As no prior large clinical trials had been conducted with this population, the authors launched “A Multicenter Trial of Vitamin E in Aging Individuals with Down Syndrome.” The authors describe how they assembled an international group of investigators to develop the infrastructure and methodology for studying the efficacy of therapeutic interventions aimed at slowing the process of AD in Down syndrome. The process of designing and implementing the trial drew together clinical scientists and clinicians from two distinct but overlapping areas: researchers in the areas of Down syndrome and AD therapeutics. The authors review the issues considered and decisions made regarding various aspects of the trial design.