Experimental therapeutic trial design for pediatric brain tumors

Miriam Bornhorst, Eugene I. Hwang*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


Pediatric brain tumors are the leading cause of cancer-related death during childhood. Since the first pediatric brain tumor clinical trials, the field has seen improved outcomes in some, but not all tumor types. In the past few decades, a number of promising new therapeutic agents have emerged, yet only a few of these agents have been incorporated into clinical trials for pediatric brain tumors. In this review, the authors discuss the process of and challenges in pediatric clinical trial design; this will allow for highly efficient and effective clinical trials with appropriate endpoints to ensure rapid and safe investigation of novel therapeutics for children with brain tumors.

Original languageEnglish (US)
Pages (from-to)1421-1432
Number of pages12
JournalJournal of child neurology
Issue number12
StatePublished - Oct 1 2016
Externally publishedYes


  • clinical trial
  • design
  • pediatric
  • targeted therapy
  • treatment

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Clinical Neurology


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