Neuroblastoma Precision Trial

Status: Recruiting
DFCI Protocol ID: 16-624

This proposal sets forth the platform for a Precision Medicine clinical trial through the New Approaches to Neuroblastoma Therapy (NANT) consortium. The plan is to utilize NANT's established multi-institutional infrastructure and Translational Genomics Research Institute GEM sequencing platform for acquisition and gene panel sequencing of relapsed biological specimens in relapsed/refractory neuroblastoma (rNB) including those obtained from the bone, bone marrow or soft tissue. Our primary aim is to identify subgroups of rNB patients who have potentially targetable genetic (ALK, MAPK pathway, Metabolic-related genes) and/or immunologic (tumor-associated macrophage infiltration and/or programmed death ligand [PD-L1] expression) biomarkers in rNB. Additional potential novel biomarkers will also be evaluated and reported in this cohort of patients.

Conducting Institutions:

Dana-Farber Cancer Institute, Brigham and Women's Hospital

Overall PI:

Suzanne Shusterman, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:


Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,

Eligibility Criteria

Inclusion Criteria:

  -  Patients must be ≥ 1 year and ≤ 30 years of age at study registration

  -  Patients must have had a diagnosis of neuroblastoma either by histological
     verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow
     with increased urinary catecholamines.

  -  Patients must have a history of high-risk neuroblastoma according to

  -  COG risk classification at the time of study registration. Patients must have at
     least one of the following: Recurrent/progressive disease, Refractory disease,
     Persistent disease

  -  Patient must be willing to undergo a clinically indicated biopsy and meet at least
     one of the following requirements: Bone biopsy, Soft tissue biopsy, Bone marrow
     biopsy and aspirate

  -  Patients must not be receiving any other anti-cancer agents or radiotherapy during
     the interval

Exclusion Criteria:

  -  Patients with disease of any major organ system that would compromise their ability
     to withstand biopsy procedures of soft tissue, bone and/or bone marrow.

  -  Patients who enroll and successfully receive a NANT Precision Report may not
     re-enroll at a future time.

  -  Patient declines participation in NANT 2004-05, the NANT Biology Study.

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