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Biomarkers in Young Patients With Neuroblastoma

This study has been completed.
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group
ClinicalTrials.gov Identifier:
NCT01169376
First received: July 23, 2010
Last updated: May 17, 2016
Last verified: May 2016
  Purpose

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 Intervention
Neuroblastoma
Genetic: DNA analysis
Genetic: DNA methylation analysis
Genetic: RNA analysis
Genetic: comparative genomic hybridization
Genetic: mutation analysis
Genetic: polymorphism analysis

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Retrospective
Official Title: Therapeutically Applicable Research to Generate Effective Treatments (TARGET) for Neuroblastoma

Resource links provided by NLM:


Further study details as provided by Children's Oncology Group:

Primary Outcome Measures:
  • Discovery of therapeutically relevant driver mutations [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Identification of a set of neuroblastoma specimens for analyses [ Designated as safety issue: No ]
  • Genome-wide DNA copy number and allelic status [ Designated as safety issue: No ]
  • Genome-wide methylation profile [ Designated as safety issue: No ]
  • Genome-wide microRNA expression profile [ Designated as safety issue: No ]
  • Genome-wide RNA expression signatures [ Designated as safety issue: No ]
  • Identification of mutations in candidate therapeutic targets [ Designated as safety issue: No ]
  • Characterization of the relapsed high-risk neuroblastoma genome and epigenome [ Designated as safety issue: No ]

Estimated Enrollment: 380
Study Start Date: July 2010
Study Completion Date: May 2016
Primary Completion Date: May 2016 (Final data collection date for primary outcome measure)
Detailed Description:

OBJECTIVES:

Primary

  • To discover the therapeutically relevant driver mutations in high-risk pediatric neuroblastoma.

Secondary

  • 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.

  Eligibility

Ages Eligible for Study:   up to 30 Years   (Child, Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients registered on the COG-ANBL00B1 Neuroblastoma Biology Study
Criteria

DISEASE CHARACTERISTICS:

  • Registered on the COG-ANBL00B1 Neuroblastoma Biology Study or its CCG or POG precursor
  • Sufficient high-quality tumor material available for the proposed studies meeting the following criteria:

    • Tissue histopathologic review with > 70% tumor cells in sections adjacent to areas used for nucleic acid preparation
    • Matched normal cells (blood or uninvolved bone marrow) available
    • ≥ 5 μg DNA available
    • ≥ 5 μg RNA available
    • ≥ 200 mg tissue available
  • Tumor samples must meet 1 of the following criteria:

    • High-risk tumor

      • With or without MYCN amplification
      • With or without tumor progression or relapse (during ≥ 2.5 years of follow up)
      • Patients aged 18 months to 5 years
    • Low-risk tumor

      • Primary neuroblastoma
      • Stage I disease (completely resected)
      • No event in ≥ 3 years of follow up
    • Cell lines representing diverse high-risk genetics including with or without MYCN amplification and clinical course (at diagnosis or after relapse)

PATIENT CHARACTERISTICS:

  • Not specified

PRIOR CONCURRENT THERAPY:

  • Not specified
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01169376

Sponsors and Collaborators
Children's Oncology Group
National Cancer Institute (NCI)
Investigators
Principal Investigator: John M. Maris, MD Children's Hospital of Philadelphia
  More Information

Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT01169376     History of Changes
Other Study ID Numbers: ANBL10B1  COG-ANBL10B1  CDR0000681912  NCI-2011-02246 
Study First Received: July 23, 2010
Last Updated: May 17, 2016
Health Authority: United States: Federal Government

Keywords provided by Children's Oncology Group:
disseminated neuroblastoma
localized resectable neuroblastoma
localized unresectable neuroblastoma
regional neuroblastoma
stage 4S neuroblastoma

Additional relevant MeSH terms:
Neuroblastoma
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue

ClinicalTrials.gov processed this record on December 07, 2016