Examination of reporting status of pediatric oncology trials within the national library of medicine’s trial database

Abstract

Finding safer and more effective treatment options are critical in progressing the field of pediatric oncology. These treatment options are discovered through completion and publication of clinical trials. The primary objective of this study was to assess the overall study characteristics of pediatric oncology clinical trials initiated between 2008 and 2021. The secondary objective of our study was to assess rates of discontinuation and reporting of results as required by the Food and Drug Administration (FDA). After acquiring pediatric oncology clinical trials from ClinicalTrials.gov, a cross-sectional study was performed. Included trials have an intervention exclusive to pediatrics and were conducted between 2008 and 2021. The results measured were characteristics of the clinical trials and their rate of discontinuation. Of the 7,930 trials originally returned from the search, 349 trials met inclusion criteria. The majority of the trials were phase 1 and 2 pharmaceutical interventions studying brain and blood cancer. Our study found that 14.9% (52) of the pediatric oncology trials were discontinued. Given the breadth of study within pediatric oncology, our overarching assessment shows that drug trials geared toward treating cancers of the brain and blood were dominant in the field. It is crucial for the advancement of science that results of trials are known. This avoids duplication of studies and waste of funds. Of the trials that were completed, 40.3% (58) did not report results to ClinicalTrials.gov. The nonreporting of this data limits the information available delaying the advancement of treatment options.

Keywords:

• Clinical trials
• pediatric oncology
• trial discontinuation

Authors’ contribution

Carly Stewart carried out initial data analysis, drafted initial manuscript, and reviewed and revised the manuscript. Dr. Micah Hartwell conceptualized and designed the study, performed statistical analysis, drafted the initial manuscript, reviewed and revised the manuscript, and supervised all aspects of the project. Liza-Ann Whitaker carried out the initial data analyses and drafted the initial manuscript. Kristen McPherson drafted the initial manuscript and reviewed and revised the manuscript. Nicholas B. Sajjadi critically reviewed and revised the manuscript. Randi Kerr drafted initial manuscript. Dr. Michael Baxter provided clinical insight. All authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work.

Disclosure of interest

No financial or other sources of support were provided during the development of this Protocol. Micah Hartwell receives research support through the National Institute of Justice and the Human Resources Services Agency, unrelated to the current work. The authors declare no conflicts of interest.

What is known on this subject

Clinical trials are necessary for the progression of safer and more effective treatment options within pediatric oncology. Discontinuation of trials and nonreporting of results leads to financial waste and limits the treatment options available.

What this study adds

While there have been studies investigating the rates of trial discontinuation in pediatrics, to our knowledge there are no studies investigating pediatric oncology specifically. This study allows for a comprehensive view of study characteristics and the rate of discontinuation.

Figure 1. PRISMA diagram showing excluded trials and reasons for primary and secondary objectives.

Additional information

Funding

No funding was provided for this study.