Biomarkers in Young Patients With Neuroblastoma

Neuroblastoma Clinical Trial

Official title:

Therapeutically Applicable Research to Generate Effective Treatments (TARGET) for Neuroblastoma

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01169376
• Other study ID #: ANBL10B1
• Secondary ID: COG-ANBL10B1CDR0
• Status: Completed
• Phase: N/A
• First received: July 23, 2010
• Last updated: May 17, 2016
• Start date: July 2010
• Est. completion date: May 2016

Study information

• Verified date: May 2016
• Source: Children's Oncology Group
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Observational

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 380
• Est. completion date: May 2016
• Est. primary completion date: May 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 30 Years

Eligibility

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

Study Design

Observational Model: Case-Only, Time Perspective: Retrospective

Related Conditions & MeSH terms

• Neuroblastoma

Intervention

Genetic:
• DNA analysis

• DNA methylation analysis

• RNA analysis

• comparative genomic hybridization

• mutation analysis

• polymorphism analysis

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Children's Oncology Group	National Cancer Institute (NCI)

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Discovery of therapeutically relevant driver mutations			No
Secondary	Identification of a set of neuroblastoma specimens for analyses			No
Secondary	Genome-wide DNA copy number and allelic status			No
Secondary	Genome-wide methylation profile			No
Secondary	Genome-wide microRNA expression profile			No
Secondary	Genome-wide RNA expression signatures			No
Secondary	Identification of mutations in candidate therapeutic targets			No
Secondary	Characterization of the relapsed high-risk neuroblastoma genome and epigenome			No

External Links

•   Clinical trial summary from the National Cancer Institute's PDQ® database