Biomarkers in Young Patients With Neuroblastoma
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|ClinicalTrials.gov Identifier: NCT01169376|
Recruitment Status : Completed
First Posted : July 26, 2010
Last Update Posted : May 18, 2016
RATIONALE: Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer.
PURPOSE: This research study is studying biomarkers in young patients with neuroblastoma.
|Condition or disease||Intervention/treatment|
|Neuroblastoma||Genetic: DNA analysis Genetic: DNA methylation analysis Genetic: RNA analysis Genetic: comparative genomic hybridization Genetic: mutation analysis Genetic: polymorphism analysis|
- To discover the therapeutically relevant driver mutations in high-risk pediatric neuroblastoma.
- To identify a set of highly annotated neuroblastoma specimens (primary tumors and cell lines) for comprehensive genomic analyses, validation studies, resequencing efforts, and future functional assays.
- To define genome-wide DNA copy number and allelic status in at least 300 high-risk and 50 low-risk neuroblastoma primary untreated tumors, and 30 human neuroblastoma-derived cell lines.
- To define the genome-wide methylation profile of neuroblastoma in a minimum of 200 high-risk cases.
- To define the genome-wide microRNA expression profile of neuroblastoma in a minimum of 200 high-risk cases.
- To define genome-wide RNA expression signatures, including splice variations, in the same tumors and cell lines studied above.
- To identify mutations in candidate therapeutic targets using a staged resequencing strategy with ultimate genome-scale next generation resequencing of 3 genomes for 200 high-risk cases: the neuroblastoma genome and transcriptome as well as the paired constitutional genome.
- To characterize the relapsed high-risk neuroblastoma genome and epigenome.
OUTLINE: This is a multicenter study.
Previously collected samples are analyzed to define the genome-wide DNA copy number and allelic status; to define the genome-wide methylation profile of high-risk neuroblastoma cases; to define the genome-wide microRNA expression profile of high-risk neuroblastoma cases; to define the genome-wide RNA expression and relating gene expression to DNA copy number and gene polymorphisms, DNA methylation, and microRNA expression; to resequence three genomes: the neuroblastoma genome, the transcriptome, and the paired constitutional genome; and to characterize the relapsed high-risk neuroblastoma genome and epigenome.
PROJECTED ACCRUAL: A total of 300 tumor samples from patients with high-risk disease, 50 tumor samples from patients with low-risk primary neuroblastoma, and 30 human neuroblastoma-derived cell lines will be accrued for this study.
|Study Type :||Observational|
|Estimated Enrollment :||380 participants|
|Official Title:||Therapeutically Applicable Research to Generate Effective Treatments (TARGET) for Neuroblastoma|
|Study Start Date :||July 2010|
|Actual Primary Completion Date :||May 2016|
|Actual Study Completion Date :||May 2016|
- Discovery of therapeutically relevant driver mutations
- Identification of a set of neuroblastoma specimens for analyses
- Genome-wide DNA copy number and allelic status
- Genome-wide methylation profile
- Genome-wide microRNA expression profile
- Genome-wide RNA expression signatures
- Identification of mutations in candidate therapeutic targets
- Characterization of the relapsed high-risk neuroblastoma genome and epigenome
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01169376
|Principal Investigator:||John M. Maris, MD||Children's Hospital of Philadelphia|