Molecular Analysis of Samples From Patients With Diffuse Intrinsic Pontine Glioma and Brainstem Glioma

February 8, 2023 updated by: Javad Nazarian, Children's National Research Institute
The purpose of this study is to prospectively collect specimens from pediatric patients with diffuse intrinsic pontine glioma or brainstem glioma, either during therapy or at autopsy, in order to characterize the molecular abnormalities of this tumor.

Study Overview

Status

Recruiting

Conditions

Detailed Description

High grade diffuse intrinsic pontine glioma (DIPG) accounts for approximately 80% of pediatric brainstem tumors and 10% of pediatric brain tumors, and is the most lethal form of brainstem gliomas in children. There is currently no effective therapy to treat these tumors. We hypothesize that this tumor exhibits unique molecular abnormalities leading to altered RNA and protein expression. The aim of this trial is to collect specimens from pediatric patients with diffuse intrinsic pontine glioma including serum, cerebrospinal fluid, urine, brain tumor and other constitutional tissue, during therapy and/or at autopsy. Our goal is to study this tissue to characterize the genetic abnormalities that lead to tumor formation in order to identify key molecules as biomarkers which we can target to design and test new and more effective treatments.

Study Type

Observational

Enrollment (Anticipated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Recruiting
        • Children's National Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 21 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Community sample

Description

Inclusion Criteria:

  • Patients of any age with clinical and radiologic diagnosis of diffuse intrinsic pontine glioma
  • Patients with other high-grade gliomas originating in the brainstem
  • Patients with focal gliomas (WHO grade I/II) of the brainstem

Exclusion Criteria:

  • Patients with any type of infiltrative low grade (WHO grade I and II) or high grade glioma (WHO grade III and IV) originating outside the brainstem
  • Patients harboring primary brainstem tumors with other histologic diagnoses (e.g., PNET)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Patient samples
Fresh-frozen and fixed tumor samples, correspondent normal brain tissue samples, cerebrospinal fluid, urine, and serum samples from patients affected with diffuse intrinsic pontine glioma or brainstem glioma

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Genome-wide expression patterns of RNA in tumor samples, normal brainstem tissue and cerebrospinal fluid using Affymetrix gene expression profiling
Time Frame: 5 years
Collected tumor and normal samples will potentially be used for RNA genome-wide expression pattern profiling.
5 years
Validation of results of the genome-wide analysis
Time Frame: 5 years
The molecular analysis done on collected samples will be validated through whole genome sequencing.
5 years
Proteomic profiling of tumor, normal brainstem tissue and cerebrospinal fluid
Time Frame: 5 years
To obtain full characterization of collected samples, proteomic profiling will be done on tumor and normal samples collected.
5 years
Protein expression patterns as assessed by immunohistochemistry and western blot compared to normal brainstem tissue
Time Frame: 5 years
Collected tumor and normal samples will have the immunochemistry and western blot compared to assess protein expression variation.
5 years
Genome-wide analysis of tumor samples and normal brainstem tissue
Time Frame: 5 years
To obtain full characterization of collected samples, whole genome sequencing will be done on tumor and normal samples collected.
5 years
In vitro and in vivo molecular analysis of collected samples
Time Frame: 5 years
Collected samples will potentially be used for in vitro analysis and generation of animal models of this tumor.
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assess aspects associated with specimen acquisition, including potential benefits and drawbacks
Time Frame: 5 years
In an effort to continue to draw knowledge from samples collected, potential benefits and drawbacks from specimen acquisition will be continuously accessed.
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's National Research Institute

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

April 1, 2010

Primary Completion (ANTICIPATED)

April 1, 2025

Study Completion (ANTICIPATED)

April 1, 2030

Study Registration Dates

First Submitted

April 19, 2010

First Submitted That Met QC Criteria

April 19, 2010

First Posted (ESTIMATE)

April 20, 2010

Study Record Updates

Last Update Posted (ACTUAL)

February 10, 2023

Last Update Submitted That Met QC Criteria

February 8, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DIPG-1

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Diffuse Intrinsic Pontine Glioma

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in South Korea

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe