Study of Stored Tumor Samples in Young Patients With Brain Tumors

Molecular and Histopathologic Characterization of Atypical Teratoid Rhabdoid Tumors, Choroid Plexus Carcinomas, Ependymomas, Medulloblastoma HGNET, CNS Embryonal Tumors and Gliomas of the Pediatric CNS

This laboratory study is looking at stored tumor samples in young patients with brain tumors. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.

Study Overview

• Status: Recruiting
• Conditions: Brain and Central Nervous System Tumors

Detailed Description

The overall objective of this non-therapeutic protocol is to develop and molecularly characterize patient-derived orthotopic xenografts (PDOXs), organoids and in vitro models derived from medulloblastomas, High Grade Neuroepithelial Tumors (HGNET), CNS embryonal tumors, Atypical Teratoid Rhabdoid Tumors (ATRTs), Choroid Plexus Carcinomas (CPCs), ependymomas, and gliomas. The investigators will characterize the genome-wide mutation, expression and epigenetic signatures of these models and compare them with the primary tumors from which they were derived, thus creating a well-characterized and invaluable resource for research on these rare and deadly pediatric brain tumors. This will also provide important insights into intratumoral heterogeneity, and molecular abnormalities that may influence the selective pressures driving evolution, and tumor growth as in PDOXs, organoids or in vitro cultures and define the relationship between these abnormalities and tumor histologic and clinical characteristics. This objective will be achieved by applying state-of-the-art DNA, RNA and epigenome analysis tools to the study of fresh frozen and/or cryopreserved, fixed and cultured tumor cells, PDOXs and organoids. The establishment of patient-derived orthotopic xenografts, organoids and cell cultures from each tumor sample will also allow for in vitro and in vivo analysis of tumor cell growth, signaling and therapeutic response.

• Study Type: Observational
• Enrollment (Estimated): 500

Contacts and Locations

• Study Contact: Name: Tabatha E. Doyle, RN; Phone Number: 901-595-2544; Email: tabatha.doyle@stjude.org

Study Locations

• United States
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • Recruiting
      • St. Jude Children'S Research Hospital
      • Contact: Tabatha E. Doyle, RN; Phone Number: 901-595-2544; Email: tabatha.doyle@stjude.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 39 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants will all brain tumor types will be included with a focus on medulloblastoma, HGNET, CNS embryonal tumors, gliomas, ependymoma, CPC and ATRT tumors. All studies will be conducted using tumor material collected prospectively from a clinically well characterized patient cohort.

Description

Inclusion Criteria

• Tumor may be primary, progressive or recurrent CNS tumor including brain and/or spine. All tumor types will be included with a focus on medulloblastoma, HGNET, CNS embryonal tumors, gliomas, ependymoma, CPC and ATRT tumors. Low grade gliomas are currently very challenging to culture and implant but if techniques mature these will also be included. Although rare, patients with ATRT may present with a primary renal and CNS tumor. In these instances samples will be collected from both the kidney and CNS tumor for analysis if available.
• Tumor may be collected at surgery prior to histologic confirmation
• Age less than 40 years at the time of initial diagnosis.
• Enrollment in the current version of the institution's banking protocol

Exclusion Criteria

• Diagnosis of tumor outside the central nervous system.
• Age greater than or equal to 40 years at the time of diagnosis

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Tumor/Tissue Sample; Tumor material collected prospectively from a clinically well characterized patient cohort

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Relationship between molecular abnormalities and tumor histologic and clinical characteristics	20 Years

Collaborators and Investigators

• Sponsor: St. Jude Children's Research Hospital
• Investigators: Principal Investigator: Amar Gajjar, MD, St. Jude Children'S Research Hospital

Publications and helpful links

Helpful Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

Study record dates

Study Major Dates

• Study Start (Actual): November 30, 2004
• Primary Completion (Estimated): February 1, 2026
• Study Completion (Estimated): February 1, 2026

Study Registration Dates

• First Submitted: May 9, 2009
• First Submitted That Met QC Criteria: May 9, 2009
• First Posted (Estimated): May 12, 2009

Study Record Updates

• Last Update Posted (Actual): August 2, 2023
• Last Update Submitted That Met QC Criteria: August 1, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Keywords: childhood infratentorial ependymoma; childhood atypical teratoid/rhabdoid tumor; childhood supratentorial ependymoma; recurrent childhood supratentorial primitive neuroectodermal tumor; recurrent childhood ependymoma; recurrent childhood medulloblastoma; childhood choroid plexus tumor; gliomas; untreated childhood supratentorial primitive neuroectodermal tumor; childhood medulloblastoma
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neoplasms, Complex and Mixed; Neuroectodermal Tumors, Primitive; Nervous System Neoplasms; Central Nervous System Neoplasms; Ependymoma; Medulloblastoma; Rhabdoid Tumor
• Other Study ID Numbers: NBTP01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No