Experimental Therapeutic Trial Design for Pediatric Brain Tumors.
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.
Department of Pediatric Hematology-Oncology, Children's National Medical Center, Washington, DC, USA Brain Tumor Institute, Washington, DC, USA.
Miriam
M
Bornhorst
Department of Pediatric Hematology-Oncology, Children's National Medical Center, Washington, DC, USA Gilbert Family Neurofibromatosis Institute, Centers for Cancer and Immunology Research & Neuroscience Research, Children's National Medical Center, Washington, DC, USA EHwang@childrensnational.org.
Eugene I
EI
Hwang
D000818:Animals; D001932:Brain Neoplasms / Q000628:therapy*; D002648:Child; D002986:Clinical Trials as Topic / Q000379:methods; D006801:Humans; D012107:Research Design
clinical trial; design; pediatric; targeted therapy; treatment