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Brief Title: Molecular Profiling for Individualized Treatment Plan for DIPG
Official Title: A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children With Newly Diagnosed DIPG
Study ID: NCT02274987
Brief Summary: This is a single arm multi-center pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The current study will use a new treatment approach based on each patient's tumor genomic profiling consisting of whole exome sequencing and RNA sequencing as well as predictive modeling.
Detailed Description: The current study will test whether patients gain a clinical benefit from such a treatment approach by comparing overall survival at 12 months (OS12) to historical controls. Newly diagnosed patients will receive an individualized treatment recommendation including up to four FDA approved drugs based on the molecular profile of the patient's tumor as determined by gene expression analysis, Whole-exome sequencing (WES) and predictive modeling, age of the patient and other existing medical conditions. Initial therapy will consist of standard radiation therapy per institutional guidelines followed by molecular based therapy with FDA approved drugs.
Minimum Age:
Eligible Ages: CHILD, ADULT
Sex: ALL
Healthy Volunteers: No
Rady Children's Hospital, San Diego, California, United States
University of California, San Francisco, San Francisco, California, United States
Children's National Medical Center, Washington, District of Columbia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
University of Utah, Salt Lake City, Utah, United States
Name: Sabine Mueller, MD
Affiliation: University of California, San Francisco
Role: PRINCIPAL_INVESTIGATOR