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NCT00904241 · CITED IN SOURCE DOCUMENTS

Biomarkers in Tumor Tissue Samples From Patients With Newly Diagnosed Neuroblastoma or Ganglioneuroblastoma

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase
Not reported
Status at capture
COMPLETED
Registry last update
2026-07-09

This research trial studies biomarkers in tumor tissue samples from patients with newly diagnosed neuroblastoma or ganglioneuroblastoma. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

No posted result sections were captured. Registered plans do not establish that a treatment works.

Who could take part
eligibility Criteria
Inclusion Criteria: * All newly diagnosed patients with suspected neuroblastoma, suspected ganglioneuroblastoma, or suspected ganglioneuroma/maturing subtype seen at Children's Oncology Group (COG) institutions are eligible for this study * There will be no penalty under any circumstances for enrollment of a patient whose definitive institutional diagnosis, or central review diagnosis, is found to be a tumor other than neuroblastoma, ganglioneuroblastoma, or ganglioneuroma/ maturing subtype * Patients may not have received chemotherapy prior to enrollment on ANBL00B1 and procurement of study-related tissues with the following exception: * Patients that in the opinion of the treating physician are too ill to undergo pre-treatment tissue biopsy and require EMERGENT chemotherapy may be enrolled on ANBL00B1; documentation of the emergent nature of therapy initiation is required * It is required that a good faith effort (documented by specimen tracking) be made to submit a neuroblastoma sample (tumor, metastasis, and/or tumor-involved bone marrow) of sufficient quality for MYCN analysis in the Neuroblastoma Reference Laboratory in order for any newly diagnosed patient to be enrolled on ANBL00B1; this should be obtained prior to initiation of therapy * Exceptions * In rare cases, patients may be deemed too ill to undergo pre-treatment tissue biopsy and require EMERGENT therapy; the following eligibility guidelines apply to these cases: * For presumed INSS stage 4S patients: Efforts to submit tumor tissue (e.g., primary tumor, skin nodule, or metastatic site) within 96 hours of EMERGENT therapy initiation should be made; however, if the child is deemed too unstable for such a procedure they may still be enrolled as long as pre-treatment peripheral blood and serum have been submitted * For all other INSS stages: tumor tissue should be obtained as soon as possible within 96 hours of EMERGENT therapy initiation; patients without tumor tissues submitted within this time-frame are not eligible for enrollment * Note: it may not be possible to obtain all necessary tumor biomarkers for therapy stratification in such cases; if a patient enrolled on ANBL00B1 undergoes an additional diagnostic procedure within 96 hours of initiating therapy, additional tumor specimens may be submitted to obtain biomarkers used for risk classification; the decision to perform such procedures, and/or submit these specimens, is to be made by the managing clinicians and should reflect the clinical need to know the status of such biomarkers * Patients enrolled on ANBL1232 in Group A (either A1 or A2) will not have a tumor biopsy or resection upfront; tumor tissue submission is therefore not required for these patients to enroll on ANBL00B1; a peripheral blood and serum sample is the only specimen required to be submitted for this group of patients; should they undergo a biopsy or resection at a later date tumor can be submitted for biomarker testing at this time * All patients and/or their parents or legal guardians must sign a written informed consent * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met Exclusion Criteria: * Patients with relapsed neuroblastoma who were not enrolled on ANBL00B1 at original diagnosis are NOT eligible; samples should be submitted as part of the ABTR04B1 protocol
healthy Volunteers
false
maximum Age
30 Years
sampling Method
NON_PROBABILITY_SAMPLE
sex
ALL
std Ages
  1. CHILD
  2. ADULT
Treatment arms and interventions
arm Groups
  1. description
    Patients undergo collection of blood, tissue, and bone marrow samples for analysis via RT-PCR, quantitative PCR, flow cytometry, and FISH.
    intervention Names
    1. Other: Cytology Specimen Collection Procedure
    2. Other: Laboratory Biomarker Analysis
    label
    Ancillary-Correlative (cytology specimen collection)
interventions
  1. arm Group Labels
    1. Ancillary-Correlative (cytology specimen collection)
    description
    Correlative studies
    name
    Cytology Specimen Collection Procedure
    other Names
    1. Cytologic Sampling
    type
    OTHER
  2. arm Group Labels
    1. Ancillary-Correlative (cytology specimen collection)
    description
    Correlative studies
    name
    Laboratory Biomarker Analysis
    type
    OTHER
Study designSource null
Enrollment
count
10000
type
ESTIMATED
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    Life tables, Kaplan-Meier survival curves, log-rank tests, and Cox regression will be used to explore the relationship of laboratory variables to outcome.
    measure
    Factors currently used for risk-group assignment (DNA content, MYCN copy number, and tumor histology)
    time Frame
    Up to 3 years
  2. description
    These biological variables will be analyzed for independent clinical significance compared to MYCN amplification, INSS stage, age, ploidy, and histologic variables in predicting either response to treatment or outcome.
    measure
    Prevalence of 1p, 11q, 14q, and 17q allelic status
    time Frame
    Up to 3 years
  3. description
    These biological variables will be analyzed for independent clinical significance compared to MYCN amplification, INSS stage, age, ploidy, and histologic variables in predicting either response to treatment or outcome.
    measure
    MYCN copy number by quantitative PCR
    time Frame
    Up to 3 years
  4. description
    These biological variables will be analyzed for independent clinical significance compared to MYCN amplification, INSS stage, age, ploidy, and histologic variables in predicting either response to treatment or outcome.
    measure
    Expression pattern of neurotrophin-related genes in diagnostic neuroblastoma tumors
    time Frame
    Up to 3 years
  5. description
    These biological variables will be analyzed for independent clinical significance compared to MYCN amplification, INSS stage, age, ploidy, and histologic variables in predicting either response to treatment or outcome.
    measure
    Presence of rare tumor cells in biological specimens by RT-PCR
    time Frame
    Up to 3 years
  6. description
    During the testing for treatment effect in Phase III trials, the biologic prognostic factors may be needed for adjustment in the Cox regression model-building process.
    measure
    Database of the known biologic prognostic factors for patients on therapeutic studies
    time Frame
    Up to 3 years
secondary Outcomes
  1. description
    Cross tabulations of MYCN status per tumor versus MYCN status per blood will be generated, the percentage concordant and the percentage discordant will be calculated, and receiver operating characteristic (ROC) analyses will be performed. Kaplan-Meier curves of MYCN status per blood will be generated, and a logrank test comparison performed. The prognostic ability of MYCN status per tumor versus MYCN status per blood will be tested in a multivariable Cox model.
    measure
    MYCN status per tumor
    time Frame
    Up to 3 years
  2. description
    Cross tabulations of MYCN status per tumor versus MYCN status per blood will be generated, the percentage concordant and the percentage discordant will be calculated, and ROC analyses will be performed. Kaplan-Meier curves of MYCN status per blood will be generated, and a logrank test comparison performed. The prognostic ability of MYCN status per tumor versus MYCN status per blood will be tested in a multivariable Cox model.
    measure
    MYCN status per blood
    time Frame
    Up to 3 years
  3. description
    Descriptive analysis will be performed.
    measure
    Incidence of OMA
    time Frame
    Up to 3 years
  4. description
    Descriptive analysis will be performed.
    measure
    Incidence of spinal cord compression
    time Frame
    Up to 3 years
  5. description
    Descriptive analysis will be performed.
    measure
    Presentation with multifocal primary tumors
    time Frame
    Up to 3 years
Full study description
brief Summary
This research trial studies biomarkers in tumor tissue samples from patients with newly diagnosed neuroblastoma or ganglioneuroblastoma. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer.
detailed Description
PRIMARY OBJECTIVES: I. To prospectively analyze the factors that are currently used for risk-group assignment (v-myc avian myelocytomatosis viral oncogene neuroblastoma derived homolog \[MYCN\] copy number by fluorescent in situ hybridization \[FISH\], deoxyribonucleic acid \[DNA\] content by flow cytometry, and tumor histology using the International Neuroblastoma Pathologic Classification System) in neuroblastoma tumors at the time of diagnosis. II. To maintain a reference bank containing clinically and genetically characterized frozen tumor tissue, tumor DNA and ribonucleic acid (RNA), histology slides and paraffin blocks, neuroblastoma-derived cell lines, patient serum and paired normal DNA obtained at the time of diagnosis, at the time of second-look surgery and at the time of relapse for future research studies. III. To prospectively analyze 1p, 11q, 14q and 17q allelic status, MYCN copy number by quantitative polymerase chain reaction (PCR); and the expression pattern of neurotrophin-related genes in diagnostic neuroblastoma tumors, and assay for the presence of rare tumor cells in biological specimens by reverse transcription (RT)-PCR; these biological variables will be analyzed for independent clinical significance compared to MYCN amplification, International Neuroblastoma Staging System (INSS) stage, age, ploidy, and histologic variables in predicting either response to treatment or outcome. IV. To build a database of the known biologic prognostic factors for patients on therapeutic studies. V. To serve as a Registry for neuroblastoma patients whose tumors demonstrate clinical and genetic features defined as ?Low Risk? for treatment failure in the absence of adjuvant therapy. SECONDARY OBJECTIVES: I. To prospectively analyze the concordance between detection of MYCN amplification in tumor samples and quantitative detection of MYCN DNA in serum, and to analyze the prognostic significance of MYCN amplification as detected in serum samples. II. To build a database that includes information regarding the presentation and natural history of neuroblastoma-associated health problems including but not limited to opsoclonus myoclonus ataxia (OMA) and/or spinal cord compression. OUTLINE: Patients undergo collection of blood, tissue, and bone marrow samples for analysis via RT-PCR, quantitative PCR, flow cytometry, and FISH. After completion of study, patients are followed up periodically.
Source references
references
  1. citation
    Davini M, Naranjo A, Chen L, Bagatell R, DuBois SG, Irwin MS, Goldsmith KC, Hogarty MD, Matthay KK. Factors Impacting Overall Survival Post-Relapse in High-Risk Neuroblastoma: Children's Oncology Group Outcomes From 2000 to 2019. Pediatr Blood Cancer. 2026 Jul 23:e70520. doi: 10.1002/1545-5017.70520. Online ahead of print.
    pmid
    42487567
    type
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  2. citation
    Furner B, Cheng A, Desai AV, Benedetti DJ, Friedman DL, Wyatt KD, Watkins M, Volchenboum SL, Cohn SL. Extracting Electronic Health Record Neuroblastoma Treatment Data With High Fidelity Using the REDCap Clinical Data Interoperability Services Module. JCO Clin Cancer Inform. 2024 May;8:e2400009. doi: 10.1200/CCI.24.00009.
    pmid
    38815188
    type
    DERIVED
Source notices and limitations
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        ssion analyses of prognostic factors.[<a href="#cit/section_9.1">1</a>,<a href="#cit/section_9.2">2</a>] In the <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> study of 4,832 newly diagnosed patients enrolled between 2007 to 2017, the 5-year EFS and OS rates were 77% and 87%, respectively, for patients whose tumo
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        fants and children</h5> <p id="_312" tabindex="-1">The effect of age at diagnosis on 5-year survival is profound. In the COG <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> study of 4,832 patients with newly diagnosed neuroblastoma, those younger than 18 months had a 5-year EFS rate of 82% and an OS rat
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        <p id="_312" tabindex="-1">The effect of age at diagnosis on 5-year survival is profound. In the COG <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> study of 4,832 patients with newly diagnosed neuroblastoma, those younger than 18 months had a 5-year EFS rate of 82% and an OS rate of 91%. In comparison
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        al lymph node involvement.</p> <p id="_2430" tabindex="-1">For the patients with newly diagnosed neuroblastoma enrolled in the <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> study, the 5-year EFS and OS rates, according to INRGSS stage, were the following:[<a href="#cit/section_1.78">78</a>]</p>
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        nt.</p> <p id="_2430" tabindex="-1">For the patients with newly diagnosed neuroblastoma enrolled in the <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> study, the 5-year EFS and OS rates, according to INRGSS stage, were the following:[<a href="#cit/section_1.78">78</a>]</p> <div class="pdq-content
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        ion and <a href="/types/neuroblastoma/hp/neuroblastoma-treatment-pdq#_780">Table 2</a>.</p> <p id="_2429" tabindex="-1">In the <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> study of 4,832 patients with newly diagnosed neuroblastoma, 52% of tumors were classified as favorable and 48% as unfavorable, acco
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        <h3 id="_17_toc">Children&#8217;s Oncology Group (COG) Neuroblastoma Risk Grouping</h3> <p id="_600" tabindex="-1">The COG <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> biology study served as the infrastructure for rapid and reliable acquisition of the clinical and biological prognostic markers use
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        Children&#8217;s Oncology Group (COG) Neuroblastoma Risk Grouping</h3> <p id="_600" tabindex="-1">The COG <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> biology study served as the infrastructure for rapid and reliable acquisition of the clinical and biological prognostic markers used for risk classificati
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        all multivariate regression analyses of prognostic factors.[<a href="#cit/section_4.1">1</a>,<a href="#cit/section_4.2">2</a>] In the <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> study of 4,832 newly diagnosed patients enrolled between 2007 to 2017, the 5-year EFS and OS rates were 77% and 87%, respectively,
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        ssion analyses of prognostic factors.[<a href="#cit/section_4.1">1</a>,<a href="#cit/section_4.2">2</a>] In the <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> study of 4,832 newly diagnosed patients enrolled between 2007 to 2017, the 5-year EFS and OS rates were 77% and 87%, respectively, for patients whose tumo
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        ll survival (OS) rate was 98% for the low-risk patients among more than 5,000 patients enrolled in the Children's Oncology Group (COG) <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> biology study.[<a href="#cit/section_6.1">1</a>]</li><li><strong>Intermediate risk.</strong> For patients with intermediate-risk tu
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        as 98% for the low-risk patients among more than 5,000 patients enrolled in the Children's Oncology Group (COG) <a href="/clinicaltrials/NCT00904241">ANBL00B1 (NCT00904241)</a> biology study.[<a href="#cit/section_6.1">1</a>]</li><li><strong>Intermediate risk.</strong> For patients with intermediate-risk tumors, chemotherapy is o
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    Preserved source evidence · Independent clinical review pending · Not medical advice