NCT02274987

Molecular Profiling for Individualized Treatment Plan for DIPG

A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children With Newly Diagnosed DIPG

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 38 (actual)
• Sponsor: University of California, San Francisco · Academic / Other
• Sex: All
• Age: 25 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Diffuse Intrinsic Pontine Glioma (DIPG)

Interventions

Type	Name	Description
OTHER	Specialized tumor board recommendation	A combination of up to four FDA approved drugs based on the molecular profile of the patient's tumor as determined by gene expression analysis, WES and predictive modeling.
RADIATION	Standard radiation therapy	Initial therapy will consist of standard radiation therapy per institutional guidelines followed by molecular based therapy with FDA approved drugs.

Timeline

• Start date: 2014-09-16
• Primary completion: 2018-12-19
• Completion: 2019-02-24
• First posted: 2014-10-27
• Last updated: 2022-11-04

Locations

5 sites across 1 country: United States