Neuroblastoma Precision Trial
N2015-01: Neuroblastoma Precision Trial
1 other identifier
observational
93
1 country
11
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Aug 2016
Longer than P75 for all trials
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 3, 2016
CompletedFirst Posted
Study publicly available on registry
August 16, 2016
CompletedStudy Start
First participant enrolled
August 22, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2026
March 23, 2026
March 1, 2026
10.3 years
June 3, 2016
March 19, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Identify genomic alterations, whether targetable alterations are present and within 4 defined NBL subgroups or outside of these 4 defined NBL subgroups. Identify presence/absence of immunologic biomarkers common to NBL.
Archival or biopsied tumor specimens undergo gene panel sequencing and/or immunohistochemistry and a list of all genetic/immunologic alterations are identified. Was an actionable genetic alteration identified and were any of these alterations within 4 NBL specific subgroups (ALK, MAPK, Metabolic, Immune reactive) or outside of the 4 NBL specific subgroups.
6 weeks
Secondary Outcomes (2)
How many subjects are biopsied or provide available tumor that is found to be adequate for gene panel sequencing and produces a clinical report
6 weeks
How many patients with bone marrow aspirates performed that contain < 30% tumor cells are able to be enriched and genetic alterations identified
1 year
Study Arms (1)
Relapsed/Refractory Neuroblastoma Pts
History of high risk neuroblastoma (NBL) according to Childrens Oncology Group (COG) risk classification with relapsed/progressive, refractory or persistent neuroblastoma. Archival or biopsied tumor specimens are provided for gene panel sequencing to subjects with potentially targetable genetic and/or immunologic biomarkers. A clinical report will be provided to subjects/subject physician detailing observed mutations and identified NBL subgroups and information on clinical trials that best match them. Subjects will be followed and data collected on treatments administered for one year after receiving this clinical report.
Interventions
Archival or biopsied tumor specimens will undergo gene panel sequencing to identify subgroups with targetable genetic (ALK, MAPK pathway, Metabolic-related genes) and/or immunologic (tumor associated macrophage infiltration, PD-L1 expression) biomarkers in neuroblastoma. A clinical report will be provided to subjects/subject physician detailing observed mutations and identified NBL subgroups and information on clinical trials that best match them. Subjects will be followed and data collected on treatments administered for one year after receipt of clinical report.
Eligibility Criteria
High risk neuroblastoma
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- New Approaches to Neuroblastoma Therapy Consortiumlead
- Children's Hospital Los Angelescollaborator
- The Evan Foundationcollaborator
- St. Baldrick's Foundationcollaborator
- Press On Fundcollaborator
- Rising Tide Foundationcollaborator
Study Sites (11)
Children's Hospital Los Angeles
Los Angeles, California, 90027-0700, United States
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94143, United States
Children Hospital of Colorado
Aurora, Colorado, 80045, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Children's Memorial Hospital - Chicago
Chicago, Illinois, 60614, United States
Childrens Hospital Boston, Dana-Farber Cancer Institute.
Boston, Massachusetts, 02115, United States
C.S Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
University of North Carolina
Chapel Hill, North Carolina, 27599, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3039, United States
Cook Children's Healthcare System
Fort Worth, Texas, 76104, United States
Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, 98105, United States
Biospecimen
Blood obtained (any time prior to and up to the day of biopsy) Biopsy of soft tissue, bone, bone marrow Specimens tumor content for genetic +/- IHC analysis.
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Shahab Asgharzadeh, MD
Children's Hospital Los Angeles
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 3, 2016
First Posted
August 16, 2016
Study Start
August 22, 2016
Primary Completion (Estimated)
December 1, 2026
Study Completion (Estimated)
December 1, 2026
Last Updated
March 23, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will share
Individual participant data will be shared with the treating investigator and the study participant.