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Clinical Trials in Australia / NCT02176967
Active, not recruiting Phase 3

Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma

NCT02176967 · tracked via the Priya Life Science Australia tracker
Phase
Phase 3
Started
2014-08-08
Last updated
2026-08-25

Condition(s) studied

GanglioneuroblastomaLocalized Resectable NeuroblastomaLocalized Unresectable NeuroblastomaNeuroblastoma

Investigational drug(s) / intervention(s)

BiopsyBone Marrow AspirationBone Marrow BiopsyCarboplatin →Clinical ObservationComputed TomographyCyclophosphamide →Doxorubicin Hydrochloride →Etoposide →Magnetic Resonance ImagingUltrasound

Biopsy: Undergo biopsy

Bone Marrow Aspiration: Undergo bone marrow aspiration

Bone Marrow Biopsy: Undergo bone marrow biopsy

Carboplatin: Given IV

Clinical Observation: Undergo clinical observation

Computed Tomography: Undergo CT

Cyclophosphamide: Given IV

Doxorubicin Hydrochloride: Given IV

Etoposide: Given IV

Magnetic Resonance Imaging: Undergo MRI

Ultrasound: Undergo ultrasound

Study summary

This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and what the best treatment is. Response and biology-based risk factor-guided therapy may be effective in treating patients with non-high risk neuroblastoma and may help to avoid some of the risks and side effects related to standard treatment.

Eligibility

Sex
ALL
Min age
—
Max age
18 Months
Healthy volunteers
No
Inclusion Criteria: * Patients must be: * \< 12 months (\< 365 days) of age at diagnosis with INRG stage L1; or * \< 18 months (\< 547 days) of age at diagnosis with INRG stage L2 or stage Ms neuroblastoma/ganglioneuroblastoma * Enrollment on ANBL00B1 or APEC14B1 is required for all newly diagnosed patients * Patients must have newly diagnosed v-myc avian myelocytomatosis viral oncogene neuroblastoma derived homolog (MYCN) non-amplified neuroblastoma (International Classification of Diseases for Oncology \[ICD-O\] morphology 9500/3) or MYCN non-amplified ganglioneuroblastoma verified by histology * Patients must meet the specified criteria for one of the treatment groups defined below; genomic features include MYCN gene amplification, segmental chromosome aberrations (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q) and deoxyribonucleic acid (DNA) index * "Favorable" genomic features are defined by one or more whole-chromosome gains or hyperdiploid tumor (DNA index \> 1) in the absence of segmental chromosome aberrations as defined above * "Unfavorable" genomic features are defined by the presence of any segmental chromosome aberration (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q) or diploid tumor (DNA index = 1); this includes copy neutral loss of heterozygosity (LOH) * Only patients with MYCN non-amplified tumors are eligible for this study * Group A: patients \< 12 months (\< 365 days) of age with newly diagnosed INRG stage L1 neuroblastoma/ganglioneuroblastoma who meet the following criteria: * Greatest tumor diameter \< 5 cm of adrenal or non-adrenal origin * Patients with non-adrenal primaries are eligible, but must have positive uptake on metaiodobenzylguanidine (MIBG) scan or elevated catecholamine metabolites (urine or serum) to support the diagnosis of neuroblastoma * No prior tumor resection or biopsy * Group A will be further split into two subsets, which are mutually exclusive, for statistical purposes * Group A1: * \> 6 months and \< 12 months of age with an adrenal primary tumor \< 5 cm in greatest diameter OR * Patients less than 6 months of age with an adrenal primary tumor \> 3.1 and \< 5 cm in greatest diameter OR * \< 12 months of age with a non-adrenal primary site \< 5 cm in greatest diameter * Group A2: =\< 6 months of age with an adrenal primary site and tumor =\< 3.1 cm in greatest diameter * Group B: patients \< 18 months (\< 547 days) of age with newly diagnosed INRG stage L2 neuroblastoma/ganglioneuroblastoma who meet the following criteria: * No life threatening symptoms or no impending neurologic or other organ function compromise (e.g. epidural or intraspinal tumors with existing or impending neurologic impairment, periorbital or calvarial-based lesions with existing or impending cranial nerve impairment, anatomic or mechanical compromise of critical organ function by tumor \[abdominal compartment syndrome, urinary obstruction, etc.\]); horner syndrome is not considered neurologic compromise * No prior tumor resection, tumor biopsy ONLY * Only patients with both favorable histology and favorable genomic features will remain on study as part of Group B; the institution will be notified of histologic and genomic results within 3 weeks of specimen submission on ANBL00B1 or APEC14B1 * Group C: patients \< 18 months (\< 547 days) of age with newly diagnosed INRG stage Ms neuroblastoma/ganglioneuroblastoma * No prior radiotherapy or chemotherapy, with the exception of dexamethasone, which is allowed * 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 MYCN amplified tumors are not eligible * Group B and C patients who do not enroll on ANBL1232 within 4 weeks of definitive diagnostic procedure * Group A and C patients, not required to undergo tumor biopsy, who do not enroll on ANBL1232 within 4 weeks of confirmatory imaging study

Primary outcome measure(s)

Trial sites (222)

FacilityCityRegionStatus
Children's Hospital of Alabama Birmingham Alabama
USA Health Strada Patient Care Center Mobile Alabama
Providence Alaska Medical Center Anchorage Alaska
Banner Children's at Desert Mesa Arizona
Phoenix Childrens Hospital Phoenix Arizona
Banner University Medical Center - Tucson Tucson Arizona
Arkansas Children's Hospital Little Rock Arkansas
Kaiser Permanente Downey Medical Center Downey California
City of Hope Comprehensive Cancer Center Duarte California
Loma Linda University Medical Center Loma Linda California
Miller Children's and Women's Hospital Long Beach Long Beach California
Children's Hospital Los Angeles Los Angeles California
Cedars-Sinai Medical Center Los Angeles California
Valley Children's Hospital Madera California
UCSF Benioff Children's Hospital Oakland Oakland California
Kaiser Permanente-Oakland Oakland California
Children's Hospital of Orange County Orange California
Lucile Packard Children's Hospital Stanford University Palo Alto California
Sutter Medical Center Sacramento Sacramento California
University of California Davis Comprehensive Cancer Center Sacramento California
Rady Children's Hospital - San Diego San Diego California
Naval Medical Center -San Diego San Diego California
UCSF Medical Center-Mission Bay San Francisco California
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance California
Children's Hospital Colorado Aurora Colorado
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center Denver Colorado
Connecticut Children's Medical Center Hartford Connecticut
Yale University New Haven Connecticut
Alfred I duPont Hospital for Children Wilmington Delaware
MedStar Georgetown University Hospital Washington D.C. District of Columbia
Children's National Medical Center Washington D.C. District of Columbia
Broward Health Medical Center Fort Lauderdale Florida
Golisano Children's Hospital of Southwest Florida Fort Myers Florida
UF Health Cancer Institute - Gainesville Gainesville Florida
Memorial Regional Hospital/Joe DiMaggio Children's Hospital Hollywood Florida
Nemours Children's Clinic-Jacksonville Jacksonville Florida
University of Miami Miller School of Medicine-Sylvester Cancer Center Miami Florida
Nicklaus Children's Hospital Miami Florida
Miami Cancer Institute Miami Florida
AdventHealth Orlando Orlando Florida

+ 182 more sites — see the full list on the official registry below.

More Children's Oncology Group trials in Australia

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT02176967 on ClinicalTrials.gov ↗ ← All trials in Australia