Trials and Tribulations - The Challenges of Clinical Trials in Children

In this issue of NEJM Evidence, we see the results of a randomized clinical trial of dapagliflozin or saxagliptin in pediatric type 2 diabetes (T2D). In children and adolescents with T2D, dapagliflozin achieved significant improvements in glycemia in the trial.¹ In June 2023, following another pivotal trial, the U.S. Food and Drug Administration (FDA) approved empagliflozin and the combination of empagliflozin and metformin as additions to diet and exercise to improve blood sugar control in children 10 years and older with T2D. Metformin, the only other oral therapy available for the treatment of children with T2D, was first approved for pediatric use in 2000.