Clinical Trials on Cellular Therapy for Children and Adolescents With Cancer: A 15-Year Trend in the United States

Sukjoo Cho; Alexandra Miller; Maua Mosha; Kevin O McNerney; Jonathan Metts

doi:10.7759/cureus.47885

Clinical Trials on Cellular Therapy for Children and Adolescents With Cancer: A 15-Year Trend in the United States

Cureus. 2023 Oct 28;15(10):e47885. doi: 10.7759/cureus.47885. eCollection 2023 Oct.

Authors

Sukjoo Cho^{1

2}, Alexandra Miller³, Maua Mosha³, Kevin O McNerney⁴, Jonathan Metts^{5

6}

Affiliations

¹ Department of Pediatrics, University of South Florida Morsani College of Medicine, Tampa, USA.
² Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta and Emory University, Atlanta, USA.
³ Data Coordinating Center for Pediatric Multicenter Studies, Institute for Clinical and Translational Research, Johns Hopkins All Children's Hospital, St. Petersburg, USA.
⁴ Department of Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, USA.
⁵ Cancer and Blood Disorders Institute, Johns Hopkins All Children's Hospital, St. Petersburg, USA.
⁶ Sarcoma Department, H. Lee Moffitt Cancer Center and Research Institute, Tampa, USA.

Abstract

Introduction: Cellular therapies are frequently studied in clinical trials for pediatric patients with malignant disease. Characteristics of ongoing and completed cellular therapy clinical trials in the U.S. involving children and adolescents have not previously been reported.

Methods: We searched ClinicalTrials.gov for clinical trials involving cellular therapies enrolling patients under 18 years of age in the U.S. Trials were initially stratified into child-only (maximum age of eligibility <18 years), child/adolescent and young adult (AYA) (maximum age of eligibility ≤21 years), and child/adult (maximum age of eligibility >21 years). Descriptive characteristics and trends over time were analyzed.

Results: We included 202 trials posted 2007-2022. Of the 202 trials, only three trials were child-only; thus, our subsequent analysis focused on comparing child/AYA (≤21 years) and child/adult trials (>21 years). One hundred sixty-nine (84%) enrolled both child and adult populations. The vast majority of trials were early phase (phase 1, 1/2, and 2, 198/202, 98%). Chimeric antigen receptor T cell therapies were most commonly studied (88/202, 44%), while natural-killer cell therapies were most common in child/AYA trials (42% vs. 16%). Most trials were single institution-only (130/202, 64%) and did not receive industry funding (163/202, 81%). Studies with industry funding were more likely to be multicenter (64% vs. 29%) and international (31% vs. 0.6%). Notably, no central nervous system tumor-specific trials had industry funding. There was no difference in therapy type based on funding source. Yearly new trial activations increased over the time period studied (p=0.01).

Conclusion: The frequency of cellular therapy trial activations enrolling child/AYA patients with cancer in the U.S. has increased over time. Most studies were phase 1 or 2, single institution-only, and not industry-supported. Future opportunities for cell therapy for pediatric cancer should include multi-institutional approaches.

Keywords: cancer immunotherapy; cellular immunotherapy; clinical trials; developmental therapeutics; pediatric hematology-oncology.