Skip to main content
OpenTrials
Recruiting

NCT Number: NCT01106794

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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Diffuse Intrinsic Pontine Glioma Brain Diseases Brain Neoplasms Brain Stem Neoplasms Brainstem Glioma Central Nervous System Diseases Central Nervous System Neoplasms Glioma Infratentorial Neoplasms Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Nervous System Diseases Nervous System Neoplasms Neuroectodermal Tumors

Age range

Up to 21 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location status: Recruiting

Location contact

Amanda L Muhs, MD

SUB_INVESTIGATOR

Javad Nazarian, PhD

PRINCIPAL_INVESTIGATOR

Roger Packer, MD

SUB_INVESTIGATOR

Suresh Magge, MD

SUB_INVESTIGATOR

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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. 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.

  6. 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.

Secondary outcomes

  1. 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Caroline Kopsidas, BS

CONTACT

[email protected]

240-393-3359

Javad Nazarian, PhD

CONTACT

[email protected]

202-476-6022

Sponsors and collaborators

Lead sponsor

Children's National Research Institute

Other

Registry information

Important dates

Study start
2010
Primary completion
2025
Study completion
2030
First posted
Apr 20, 2010
Registry last updated
Aug 20, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.