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

Status: Recruiting
Phase:
DFCI Protocol ID: 14-449

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.

Conducting Institutions:

Children's Hospital Boston, Dana-Farber Cancer Institute, Massachusetts General Hospital

Overall PI:

Suzanne Shusterman, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:

Mary Huang, MD, Massachusetts General Hospital

Contacts:

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org
Massachusetts General Hospital: Cancer Trials Call Center, 877-789-6100

Eligibility Criteria

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 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.])

       -  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

  -  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

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