Characteristics of Scientific Success in Phase 1 Pediatric Oncology Clinical Trials: Establishing a Need for Integrated Patient-Reported Outcomes Even in Early Phase Trials

Abstract

Background: Pediatric clinical trials are particularly vulnerable to challenges in participant accrual, making them more likely to encounter early termination. Objective: To describe clinical trial characteristics of phase 1 pediatric oncology clinical trials registered in ClinicalTrials.gov through 2017. Method: We identified a retrospective cohort and abstracted key clinical trial characteristics from this data source. We calculated a bivariate logistic regression to explore factors related to early trial termination. Results: Over 1400 phase 1 trials were included in this analysis. Only 3.2% of trials included any type of patient-reported outcome (PRO) as a secondary outcome and 21.6% of phase 1 trials did not meet scientific success due to early termination. National Institutes of Health (NIH)-funded phase 1 trials had the lowest percentage of early termination. Conclusions: Phase 1 clinical trials for pediatric patients with cancer are at risk of early termination due to lack of enrollment. A deeper understanding of clinical trial design elements, characteristics, and eligibility determination may contribute to a higher proportion of trials meeting successful enrollment. Implications for Practice: Oncology nurses and nurse scientists uniquely contribute to an understanding of family-centered approaches and should take an active role in trial design for pediatric cancers that could help capture meaningful PROs during a vulnerable clinical trajectory. What Is Foundational: Very little attention has been given to understanding characteristics of phase 1 clinical trials for pediatric cancers that may impact the state of the science and care delivery for patients with advanced pediatric cancer diagnoses.