Recent Trends in Clinical Trials for Pediatric Sarcoma in the United States: An Analysis of ClinicalTrials.gov

Background/Objectives: Pediatric sarcomas are rare and heterogeneous malignancies for which clinical trials are essential to advance treatment and improve outcomes. However, the characteristics and trends of sarcoma clinical trials enrolling children in the United States have not been comprehensively described. This study aimed to characterize U.S.-based sarcoma clinical trials enrolling pediatric patients and to evaluate trends over time. Methods: ClinicalTrials.gov was searched for interventional sarcoma trials conducted in the United States that enrolled patients ≤ 17 years of age and were posted between 27 September 2007 and 11 January 2023. Trials were categorized as pediatric (maximum eligible age ≤ 21 years) or pediatric/adult (>21 years). Trial characteristics, including phase, intervention type, funding source, geographic scope, and reasons for early termination, were analyzed. Results: A total of 273 eligible trials were identified, of which 79% enrolled both pediatric and adult patients. Most studies were early phase (Phase 1, 2, or 1/2; 59%) and primarily evaluated drug or biologic therapies (73%). Trials involving mixed cancer types were most common (26%). The majority were multi-institutional (66%), non-industry funded (57%) and conducted exclusively in the United States (75%). Trial activations increased over time (p-value = 0.01), with a higher proportion of industry-funded studies initiated between 2016 and 2022 (p-value = 0.009). Twenty-three trials (8.4%) were terminated early, most commonly due to slow accrual (39%). Conclusions: Most sarcoma clinical trials enrolling pediatric patients continue to include both adult and pediatric populations, which may limit the development of therapies tailored to the unique biology of pediatric sarcomas. Improving outcomes will require greater emphasis on pediatric-focused research, enhanced collaboration across institutions, and increased awareness of clinical and regulatory frameworks to support the initiation of industry-funded trials.