Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma


The purpose of this study is to see 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.


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

For additional eligibility criteria please contact the study team.


  • Observation
  • Surgery 
  • Chemotherapy

For additional study details and procedures will be explained by the study team.

Contact the Study Team

Additional eligibility criteria and study procedures details can be explained by the study team.  Please call or email:

  • Phone:  (352) 273-8675
  • Email:  trials@cancer.ufl.edu


18 and under



Monetary compensation


Can be done from home



Neuroblastoma, Ganglioneuroblastoma, Tumors, Neoplasm, Neuroectodermal

Principal Investigator



Contact Information


(352) 273-8675

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