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

Highlights: What are the main findings? Most sarcoma clinical trials enrolling children in the United States include both pediatric and adult patients rather than being pediatric-specific. Pediatric sarcoma trials are predominantly early-phase studies focused on drug or biologic therapies and are la...

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Publicado en:Children Vol. 13; no. 4; pp. 455 - 470
Autores principales: Alkhawaldeh, Khaled, Thorpe, Signe, Cho, Sukjoo, Miller, Alexandra, Alleyne, Maua, Jones, Jennifer, Beaupin, Lynda, Gupta, Ajay, Metts, Jonathan
Formato: research tables/charts Journal Article
Publicado: MDPI Apr2026
Acceso en línea:Ver este registro en EBSCOhost
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        atl: Recent Trends in Clinical Trials for Pediatric Sarcoma in the United States: An Analysis of ClinicalTrials.gov.
      aug:
        au:
          Alkhawaldeh, Khaled
          Thorpe, Signe
          Cho, Sukjoo
          Miller, Alexandra
          Alleyne, Maua
          Jones, Jennifer
          Beaupin, Lynda
          Gupta, Ajay
          Metts, Jonathan
        affil: Sarcoma Department, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL 33612, USA
      sug:
        subj:
          Clinical Trials Trends
          Sarcoma In Infancy and Childhood
          Research Subjects
          Patient Participation
          Human
          United States
          Female
          Male
          Child
          Adolescence
          Eligibility Determination
          Research Support
          Statistical Significance
          T-Tests
          Chi Square Test
          Fisher's Exact Test
          Wilcoxon Rank Sum Test
          Mann-Whitney U Test
          Kendall's tau
          Data Analysis Software
          Descriptive Statistics
          Funding Source
          Child: 6-12 years
          Adolescent: 13-18 years
          Female
          Male
      ab: Highlights: What are the main findings? Most sarcoma clinical trials enrolling children in the United States include both pediatric and adult patients rather than being pediatric-specific. Pediatric sarcoma trials are predominantly early-phase studies focused on drug or biologic therapies and are largely conducted across multiple institutions. What are the implications of the main findings? Limited pediatric-only trials may restrict age-specific insights into treatment efficacy and toxicity for children with sarcoma. Expanded pediatric-focused trial designs and improved accrual strategies are needed to advance outcomes in this underserved population. 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.
      pubtype: Academic Journal
      doctype:
        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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