Study of Stored Tumor Samples in Young Patients With Brain Tumors


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.

Full Title of Study: “Molecular and Histopathologic Characterization of Atypical Teratoid Rhabdoid Tumors, Choroid Plexus Carcinomas, Ependymomas, Medulloblastoma HGNET, CNS Embryonal Tumors and Gliomas of the Pediatric CNS”

Study Type

  • Study Type: Observational
  • Study Design
    • Time Perspective: Prospective
  • Study Primary Completion Date: February 2026

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.

Arms, Groups and Cohorts

  • Tumor/Tissue Sample
    • Tumor material collected prospectively from a clinically well characterized patient cohort

Clinical Trial Outcome Measures

Primary Measures

  • Relationship between molecular abnormalities and tumor histologic and clinical characteristics
    • Time Frame: 20 Years

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: 39 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • St. Jude Children’s Research Hospital
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Amar Gajjar, MD, Principal Investigator, St. Jude Children’s Research Hospital
  • Overall Contact(s)
    • Tabatha E. Doyle, RN, 901-595-2544,

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.