Clinical trial NCT00897286: Study of Stored Tumor Samples in Young Patients With Brain Tumors
Official title: 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: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Brain and Central Nervous System Tumors
Keywords: childhood atypical teratoid/rhabdoid tumor
childhood choroid plexus tumor
childhood medulloblastoma
recurrent childhood medulloblastoma
childhood infratentorial ependymoma
childhood supratentorial ependymoma
recurrent childhood ependymoma
recurrent childhood supratentorial primitive neuroectodermal tumor
untreated childhood supratentorial primitive neuroectodermal tumor
gliomas
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
First posted: 2009-05-12
Enrollment: 500
Sex: ALL
Maximum age (years): 39
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Relationship between molecular abnormalities and tumor histologic and clinical characteristics
timeFrame: 20 Years
Secondary outcomes:
Other outcomes:
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Amar Gajjar, MD
role: PRINCIPAL_INVESTIGATOR
References: