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

Children's National

Status: Enrolling

Conditions: Brainstem Glioma; Diffuse Intrinsic Pontine Glioma

Study type: Observational

Funder types: Other

Identifiers

NCT01106794

DIPG-1

Details and patient eligibility

About

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.

Full 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.

Enrollment: 100 estimated patients

Sex: All

Ages: Under 21 years old

Volunteers: No Healthy Volunteers

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)

Trial design

100 participants in 1 patient group

Patient samples

Description:

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