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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00904241




Registration number
NCT00904241
Ethics application status
Date submitted
16/05/2009
Date registered
16/05/2009
Date last updated
12/04/2019

Titles & IDs
Public title
Biomarkers in Tumor Tissue Samples From Patients With Newly Diagnosed Neuroblastoma or Ganglioneuroblastoma
Scientific title
Neuroblastoma Biology Studies
Secondary ID [1] 0 0
NCI-2009-00397
Secondary ID [2] 0 0
ANBL00B1
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ganglioneuroblastoma 0 0
Localized Resectable Neuroblastoma 0 0
Localized Unresectable Neuroblastoma 0 0
Regional Neuroblastoma 0 0
Stage 4 Neuroblastoma 0 0
Stage 4S Neuroblastoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Neuroendocrine tumour (NET)
Cancer 0 0 0 0
Children's - Other
Cancer 0 0 0 0
Children's - Brain
Cancer 0 0 0 0
Brain

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Cytology Specimen Collection Procedure
Other interventions - Laboratory Biomarker Analysis

Ancillary-Correlative (cytology specimen collection) - Patients undergo collection of blood, tissue, and bone marrow samples for analysis via RT-PCR, quantitative PCR, flow cytometry, and FISH.


Other interventions: Cytology Specimen Collection Procedure
Correlative studies

Other interventions: Laboratory Biomarker Analysis
Correlative studies

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Factors currently used for risk-group assignment (DNA content, MYCN copy number, and tumor histology) - Life tables, Kaplan-Meier survival curves, log-rank tests, and Cox regression will be used to explore the relationship of laboratory variables to outcome.
Timepoint [1] 0 0
Up to 3 years
Primary outcome [2] 0 0
Prevalence of 1p, 11q, 14q, and 17q allelic status - 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.
Timepoint [2] 0 0
Up to 3 years
Primary outcome [3] 0 0
MYCN copy number by quantitative PCR - 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.
Timepoint [3] 0 0
Up to 3 years
Primary outcome [4] 0 0
Expression pattern of neurotrophin-related genes in diagnostic neuroblastoma tumors - 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.
Timepoint [4] 0 0
Up to 3 years
Primary outcome [5] 0 0
Presence of rare tumor cells in biological specimens by RT-PCR - 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.
Timepoint [5] 0 0
Up to 3 years
Primary outcome [6] 0 0
Database of the known biologic prognostic factors for patients on therapeutic studies - 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.
Timepoint [6] 0 0
Up to 3 years
Secondary outcome [1] 0 0
MYCN status per tumor - 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.
Timepoint [1] 0 0
Up to 3 years
Secondary outcome [2] 0 0
MYCN status per blood - 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.
Timepoint [2] 0 0
Up to 3 years
Secondary outcome [3] 0 0
Incidence of OMA - Descriptive analysis will be performed.
Timepoint [3] 0 0
Up to 3 years
Secondary outcome [4] 0 0
Incidence of spinal cord compression - Descriptive analysis will be performed.
Timepoint [4] 0 0
Up to 3 years
Secondary outcome [5] 0 0
Presentation with multifocal primary tumors - Descriptive analysis will be performed.
Timepoint [5] 0 0
Up to 3 years

Eligibility
Key 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
Minimum age
No limit
Maximum age
30 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
John Hunter Children's Hospital - Hunter Regional Mail Centre
Recruitment hospital [2] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [3] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [4] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [5] 0 0
Royal Children's Hospital-Brisbane - Herston
Recruitment hospital [6] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [7] 0 0
Women's and Children's Hospital-Adelaide - North Adelaide
Recruitment hospital [8] 0 0
Royal Children's Hospital - Parkville
Recruitment hospital [9] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment hospital [10] 0 0
Perth Children's Hospital - Perth
Recruitment postcode(s) [1] 0 0
2310 - Hunter Regional Mail Centre
Recruitment postcode(s) [2] 0 0
2031 - Randwick
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment postcode(s) [4] 0 0
4029 - Herston
Recruitment postcode(s) [5] 0 0
4101 - South Brisbane
Recruitment postcode(s) [6] 0 0
5006 - North Adelaide
Recruitment postcode(s) [7] 0 0
3052 - Parkville
Recruitment postcode(s) [8] 0 0
6008 - Perth
Recruitment postcode(s) [9] 0 0
6009 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Alaska
Country [3] 0 0
United States of America
State/province [3] 0 0
Arizona
Country [4] 0 0
United States of America
State/province [4] 0 0
Arkansas
Country [5] 0 0
United States of America
State/province [5] 0 0
California
Country [6] 0 0
United States of America
State/province [6] 0 0
Colorado
Country [7] 0 0
United States of America
State/province [7] 0 0
Connecticut
Country [8] 0 0
United States of America
State/province [8] 0 0
Delaware
Country [9] 0 0
United States of America
State/province [9] 0 0
District of Columbia
Country [10] 0 0
United States of America
State/province [10] 0 0
Florida
Country [11] 0 0
United States of America
State/province [11] 0 0
Georgia
Country [12] 0 0
United States of America
State/province [12] 0 0
Hawaii
Country [13] 0 0
United States of America
State/province [13] 0 0
Idaho
Country [14] 0 0
United States of America
State/province [14] 0 0
Illinois
Country [15] 0 0
United States of America
State/province [15] 0 0
Indiana
Country [16] 0 0
United States of America
State/province [16] 0 0
Iowa
Country [17] 0 0
United States of America
State/province [17] 0 0
Kansas
Country [18] 0 0
United States of America
State/province [18] 0 0
Kentucky
Country [19] 0 0
United States of America
State/province [19] 0 0
Louisiana
Country [20] 0 0
United States of America
State/province [20] 0 0
Maine
Country [21] 0 0
United States of America
State/province [21] 0 0
Maryland
Country [22] 0 0
United States of America
State/province [22] 0 0
Massachusetts
Country [23] 0 0
United States of America
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Michigan
Country [24] 0 0
United States of America
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Minnesota
Country [25] 0 0
United States of America
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Mississippi
Country [26] 0 0
United States of America
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Missouri
Country [27] 0 0
United States of America
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Nebraska
Country [28] 0 0
United States of America
State/province [28] 0 0
Nevada
Country [29] 0 0
United States of America
State/province [29] 0 0
New Hampshire
Country [30] 0 0
United States of America
State/province [30] 0 0
New Jersey
Country [31] 0 0
United States of America
State/province [31] 0 0
New Mexico
Country [32] 0 0
United States of America
State/province [32] 0 0
New York
Country [33] 0 0
United States of America
State/province [33] 0 0
North Carolina
Country [34] 0 0
United States of America
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North Dakota
Country [35] 0 0
United States of America
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Ohio
Country [36] 0 0
United States of America
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Oklahoma
Country [37] 0 0
United States of America
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Oregon
Country [38] 0 0
United States of America
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Pennsylvania
Country [39] 0 0
United States of America
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Rhode Island
Country [40] 0 0
United States of America
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South Carolina
Country [41] 0 0
United States of America
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South Dakota
Country [42] 0 0
United States of America
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Tennessee
Country [43] 0 0
United States of America
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Texas
Country [44] 0 0
United States of America
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Utah
Country [45] 0 0
United States of America
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Vermont
Country [46] 0 0
United States of America
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Virginia
Country [47] 0 0
United States of America
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Washington
Country [48] 0 0
United States of America
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West Virginia
Country [49] 0 0
United States of America
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Wisconsin
Country [50] 0 0
Canada
State/province [50] 0 0
Alberta
Country [51] 0 0
Canada
State/province [51] 0 0
British Columbia
Country [52] 0 0
Canada
State/province [52] 0 0
Manitoba
Country [53] 0 0
Canada
State/province [53] 0 0
Newfoundland and Labrador
Country [54] 0 0
Canada
State/province [54] 0 0
Nova Scotia
Country [55] 0 0
Canada
State/province [55] 0 0
Ontario
Country [56] 0 0
Canada
State/province [56] 0 0
Quebec
Country [57] 0 0
Canada
State/province [57] 0 0
Saskatchewan
Country [58] 0 0
Netherlands
State/province [58] 0 0
Groningen
Country [59] 0 0
New Zealand
State/province [59] 0 0
Auckland
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New Zealand
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Christchurch
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Puerto Rico
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Caguas
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Puerto Rico
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San Juan
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Saudi Arabia
State/province [63] 0 0
Riyadh
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Switzerland
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Bern
Country [65] 0 0
Switzerland
State/province [65] 0 0
Geneva
Country [66] 0 0
Switzerland
State/province [66] 0 0
Lausanne

Funding & Sponsors
Primary sponsor type
Other
Name
Children's Oncology Group
Address
Country
Other collaborator category [1] 0 0
Government body
Name [1] 0 0
National Cancer Institute (NCI)
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT00904241
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Michael Hogarty
Address 0 0
Children's Oncology Group
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT00904241