NeuroblastomaNCT01169376
Biomarkers in Young Patients With Neuroblastoma
An observational study of Neuroblastoma, run by Children's Oncology Group.
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. Read more about this study
- Who is it for?
- Ages Up to 30 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- DNA analysis, DNA methylation analysis and RNA analysis
- How many people
- 380 participants
- Where
- No sites listed yet
- Study dates
- Jul 2010to May 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients registered on the COG-ANBL00B1 Neuroblastoma Biology Study
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
About this study
Official title: Therapeutically Applicable Research to Generate Effective Treatments (TARGET) for Neuroblastoma
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.
Read the 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.
- Conditions
- Neuroblastoma
- Being tested
- DNA analysis, DNA methylation analysis, RNA analysis, comparative genomic hybridization, mutation analysis
- Keywords
- disseminated neuroblastoma, localized resectable neuroblastoma, localized unresectable neuroblastoma, regional neuroblastoma, stage 4S neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DNA analysisGenetic
DNA methylation analysisGenetic
RNA analysisGenetic
comparative genomic hybridizationGenetic
mutation analysisGenetic
polymorphism analysisGenetic
What is being measured
The primary outcome is the main question the study is designed to answer.
Discovery of therapeutically relevant driver mutations
Secondary outcomes (7)
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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2010 (estimated)
- Study startJul 2010
- Primary completionMay 2016
- Study completionMay 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 380 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- John M. Maris, MD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01169376
- Sponsor's protocol ID
- ANBL10B1
- Other IDs
- COG-ANBL10B1, CDR0000681912, NCI-2011-02246
- Status last verified
- May 2016
- First submitted
- 23 Jul 2010
- Last updated
- 18 May 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Therapeutically Applicable Research to Generate Effective Treatments (TARGET) for Neuroblastoma
- ClinicalTrials.gov ID
- NCT01169376
- Acronym / sponsor ID
- ANBL10B1
- Conditions
- Neuroblastoma
- Keywords
- disseminated neuroblastoma, localized resectable neuroblastoma, localized unresectable neuroblastoma, regional neuroblastoma, stage 4S neuroblastoma
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 380 participants (estimated)
- Lead sponsor
- Children's Oncology GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jul 2010
- Primary completion
- May 2016 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 26 Jul 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients registered on the COG-ANBL00B1 Neuroblastoma Biology Study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DNA analysis | Genetic | – |
| DNA methylation analysis | Genetic | – |
| RNA analysis | Genetic | – |
| comparative genomic hybridization | Genetic | – |
| mutation analysis | Genetic | – |
| polymorphism analysis | Genetic | – |
Primary outcome
Discovery of therapeutically relevant driver mutations
7 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2016.
Related trials for Neuroblastoma
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John M. Maris, MD principal investigator
Children's Hospital of Philadelphia
Questions about this trial
What is this clinical trial studying?
An observational study of Neuroblastoma, run by Children's Oncology Group.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 May 2016. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Discovery of therapeutically relevant driver mutations. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2016.