CLINICAL TRIAL / NCT00904241
Biomarkers in Tumor Tissue Samples From Patients With Newly Diagnosed Neuroblastoma or Ganglioneuroblastoma
- Observational
- Recruiting
- NCT00904241
Contact Information
Neuroblastoma Biology Studies
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.
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.
Gender
All
Age Group
30 Years and under
Accepting Healthy Volunteers
No
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
- Neuroblastoma