Clinical trial NCT01106794: Molecular Analysis of Samples From Patients With Diffuse Intrinsic Pontine Glioma and Brainstem Glioma
Official title: Molecular Analysis of Samples From Patients With Diffuse Intrinsic Pontine Glioma and Brainstem Glioma
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Diffuse Intrinsic Pontine Glioma
Brainstem Glioma
Keywords: diffuse intrinsic pontine glioma
DIPG
childhood brainstem glioma
pediatric brainstem glioma
brainstem glioma
BSG
Lead sponsor: Children's National Research Institute
Sponsor class: OTHER
First posted: 2010-04-20
Enrollment: 100
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Community sample

Brief summary: 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.

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.

Eligibility criteria: 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)

Primary outcomes:
measure: Genome-wide expression patterns of RNA in tumor samples, normal brainstem tissue and cerebrospinal fluid using Affymetrix gene expression profiling
timeFrame: 5 years
measure: Validation of results of the genome-wide analysis
timeFrame: 5 years
measure: Proteomic profiling of tumor, normal brainstem tissue and cerebrospinal fluid
timeFrame: 5 years
measure: Protein expression patterns as assessed by immunohistochemistry and western blot compared to normal brainstem tissue
timeFrame: 5 years
measure: Genome-wide analysis of tumor samples and normal brainstem tissue
timeFrame: 5 years
measure: In vitro and in vivo molecular analysis of collected samples
timeFrame: 5 years
Secondary outcomes:
measure: Assess aspects associated with specimen acquisition, including potential benefits and drawbacks
timeFrame: 5 years
Other outcomes:
Locations:
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
Study officials:
affiliation: Children's National Research Institute
name: Javad Nazarian, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Becher OJ, Hambardzumyan D, Walker TR, Helmy K, Nazarian J, Albrecht S, Hiner RL, Gall S, Huse JT, Jabado N, MacDonald TJ, Holland EC. Preclinical evaluation of radiation and perifosine in a genetically and histologically accurate model of brainstem glioma. Cancer Res. 2010 Mar 15;70(6):2548-57. doi: 10.1158/0008-5472.CAN-09-2503. Epub 2010 Mar 2.
citation: Panditharatna E, Yaeger K, Kilburn LB, Packer RJ, Nazarian J. Clinicopathology of diffuse intrinsic pontine glioma and its redefined genomic and epigenomic landscape. Cancer Genet. 2015 Jul-Aug;208(7-8):367-73. doi: 10.1016/j.cancergen.2015.04.008. Epub 2015 May 1.
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citation: Kambhampati M, Perez JP, Yadavilli S, Saratsis AM, Hill AD, Ho CY, Panditharatna E, Markel M, Packer RJ, Nazarian J. A standardized autopsy procurement allows for the comprehensive study of DIPG biology. Oncotarget. 2015 May 20;6(14):12740-7. doi: 10.18632/oncotarget.3374.
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