A pilot trial testing the clinical benefit of using molecular profiling to determine an individualized treatment plan in children and young adults with newly diagnosed DIPG


Trial Requirements and Treatment

Please note, this study has closed to accrual and is no longer accepting patients. Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) who undergo a biopsy as part of their standard of care will be eligible for this study. Part of the tissue will be sent to TGEN for detailed profiling. After the biopsy, patients will be treated with focal irradiation per institutional guidelines. After completion of radiation therapy, patients will follow the specialized tumor board recommendation. The treatment will be based on each child’s individual tumor profile and will include up to 4 medications. Depending on the individual treatment plan, the tumor board will also recommend required study observations to assure safety for the patient.

Rationale for Study

This study will use a new treatment approach based on each patient’s tumor gene expression and whole exome sequencing. The study will test the feasibility of a specialized tumor board assigning a treatment plan based on gene expression analysis, whole exome sequencing, and predictive modeling to establish a molecular-based-therapy approach at initial diagnosis. The treatment plan will take into consideration each patient’s tumor expression profile, gene mutations, and predictive modeling, as well as prior history and treatment(s), other medical conditions, and the age of the patient.

Primary Objectives

To determine the overall survival at 12 months of children with newly diagnosed DIPG that have been treated based on a personalized treatment recommendation based on WES and RNA seq analysis of the tumor.

Secondary Objectives 

  1. To determine the safety and describe the toxicity of using a molecularly based treatment approach and specialized tumor board recommendation in children and young adults with newly diagnosed DIPG.
  2. To evaluate the safety of performing biopsy and obtaining tissue for molecular and genomic profiling in children and young adults with newly diagnosed DIPG.

Exploratory Objectives 

  1. To assess if information derived from WGS, or use of WGS findings to detect circulating tumor DNA, would have changed the treatment recommendations from the molecular tumor board. 
  2. To determine the frequency of success and genomic fidelity (to the patient’s original tumor) in patient derived xenograft models from these patients at initial diagnosis as well as at time of progression or recurrence when sufficient tissue is available.
  3. To archive tumor and normal DNA from each patient at time of initial diagnosis along with serial blood draw following therapies as biospecimens for later studies to determine whether ctDNA sequences in the patient’s blood serve as biomarkers of tumor burden, response to therapy, or development of drug resistance.


Funding is provided by The V Foundation