Redefining success in pediatric clinical trials

Abstract

Background

Historically, there have been limited clinical trials in pediatric patients with cardiomyopathy or heart failure. Although the number of these studies has increased in recent years, many have failed to meet their primary endpoints. However, these “negative” trials have been invaluable to the field of pediatric cardiology.

Aim of review

This review aims to highlight the importance of negative clinical trials in pediatric cardiology. It focuses on how these trials, despite not meeting their primary objectives, have provided critical insights into safety, drug metabolism, biomarkers, and have contributed to refining trial design for future studies.

Key scientific concepts of review

Negative trials have played a key role in advancing pediatric heart failure treatment by revealing essential data on drug metabolism, particularly across different age groups, and by identifying novel biomarkers for monitoring treatment efficacy. These trials have also led to improvements in trial design, ensuring better patient selection and more accurate evaluation of therapeutic interventions. Despite not meeting their primary endpoints, these studies have provided a foundation for future innovations and have helped shape treatment strategies in pediatric cardiology.