Pediatric blood & cancer
Alternate Journal
Pediatr Blood Cancer

OBJECTIVES: Understanding trends in characteristics of early phase trials that allow minors with cancer to participate may inform additional efforts to improve cancer drug development for young people.

METHODS: We accessed data for oncology phase 1 or phase 1/2 trials in the United States from with lower age bound for eligibility

RESULTS: Six hundred twelve trials met inclusion criteria. Sixty-five percent of trials were for older adults that also allowed minors, while 9% were exclusively for patients ≤18 years of age. Eighty-three percent of trials included at least one novel agent, while 17% studied only conventional therapies. Fifty-eight percent of trials studied treatments not yet Food and Drug Administration (FDA) approved (48% if exclusively for patients ≤18 years). Fifteen percent of trials for which dose-escalation method could be determined, utilized a model-based design. Eighteen percent of all trials were industry sponsored (48% if exclusively for patients ≤18 years). Forty-nine percent of all trials were multicenter (69% if exclusively for patients ≤18 years). There was an increase in trials exclusively focused on patients with central nervous system (CNS) tumors over the study period (P ≤ .02). No other temporal trends were seen. The median times from first-in-adult to first-in-pediatric for monotherapy and combination trials were 5.7 and 3.3 years, respectively.

CONCLUSION: The paucity of clear temporal trends highlights the need for innovation in early drug development for young people. Our analysis serves as a benchmark against which to evaluate initiatives to improve pediatric cancer drug development.

Nader JH, Neel D, Shulman DS, Ma C, Bourgeois F, DuBois SG. Landscape of phase 1 clinical trials for minors with cancer in the United States. Pediatric blood & cancer. 2020:e28694. doi:10.1002/pbc.28694.