A Review of Clinical Trials in Down Syndrome.

Abstract

Down syndrome (DS) is a prevalent neurogenetic condition that impacts thousands of individuals, their families, and their communities each year. Due to the relatively high prevalence of DS and co-occurring conditions associated with this diagnosis, it is increasingly becoming the focus of clinical trials. In an effort to review the scope of interventions for this population across time, we reviewed ClinicalTrials.gov for clinical trials being conducted in DS. The goal was to evaluate the targets of clinical trials with individuals with DS, describe changes with the onset of the INCLUDE project, and identify gaps in clinical trial targets with individuals with DS. Across 138 clinical trials related to DS registered on ClinicalTrials.gov, the following target condition emerged (with some trials targeting more than one condition): motor functioning (n = 46), cognition (n = 24), Alzheimer's disease (n = 17), sleep (n = 12), adaptive daily living skills (n = 11), leukemia (n = 10), communication (n = 7), prenatal (n = 4), dental (n = 3), Attention Deficit Hyperactivity Disorder (n = 3), and other (n = 13; any category with <3 registered trials). Collectively, across these conditions, the number and variety in clinical trials in DS have increased substantially post-INCLUDE. Implications and future directions across each area of research are discussed.