Molecular Profiling for Individualized Treatment Plan for DIPG

Not Applicable

Completed

• Conditions: Diffuse Intrinsic Pontine Glioma (DIPG)
• Interventions: Other: Specialized tumor board recommendation; Radiation: Standard radiation therapy

• Registration Number: NCT02274987
• Lead Sponsor: University of California, San Francisco

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.

Study Timeline

• Study Start: 2014-09-16
• Study First Submitted: 2014-10-21
• Study First Submitted QC: 2014-10-22
• Study First Posted: 2014-10-27
• Primary Completion: 2018-12-19
• Study Completion: 2019-02-24
• Status Verified: 2022-11
• Last Update Submitted: 2022-11-02
• Last Update Posted: 2022-11-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 38

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment	Specialized tumor board recommendation	The treatment plan for each patient is individualized and different depending on what the Specialized Tumor Board recommends depending on the molecular profile of the patient's tumor.
Treatment	Standard radiation therapy	The treatment plan for each patient is individualized and different depending on what the Specialized Tumor Board recommends depending on the molecular profile of the patient's tumor.

Primary Outcome Measures

Name	Time	Method
To determine OS12 of children with newly diagnosed DIPG that are being treated based on a specialized tumor board recommendation which is based on RNA based expression analysis, WES and predictive modeling.	12 months

Trial Locations

Locations (5)

University of Utah; 🇺🇸 Salt Lake City, Utah, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

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