NeuroblastomaNCT02868268
Neuroblastoma Precision Trial
An observational study of Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 11 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 1 year to 30 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Gene panel sequencing of tumor specimens
- How many people
- 93 participants
- Where
- 11 sitesUnited States
- Study dates
- 22 Aug 2016to Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: High risk neuroblastoma
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
C.S Mott Children's Hospital
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Children Hospital of Colorado
Aurora, Colorado, 80045
Childrens Hospital Boston, Dana-Farber Cancer Institute.
Boston, Massachusetts, 02115
University of North Carolina
Chapel Hill, North Carolina, 27599
Children's Memorial Hospital - Chicago
Chicago, Illinois, 60614
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3039
Cook Children's Healthcare System
Fort Worth, Texas, 76104
Children's Hospital Los Angeles
Los Angeles, California, 90027-0700
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94143
Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, 98105
About this study
Official title: N2015-01: Neuroblastoma Precision Trial
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.
Read the detailed description
Neuroblastoma (NB) is the most common extracranial solid tumor of childhood. High-risk NB is highly lethal and responsible for over 15% of childhood cancer related deaths. The majority of patients with metastatic NB respond to upfront cytotoxic chemotherapy, yet patients who die of recurrent disease do so from tumor acquired resistance to treatment. Thus, understanding the repertoire of tumor specific genomic alterations leading to tumor progression and therapy resistance is critical to devising novel targeted therapy options for patients with recurrent or refractory (r)NB. Limited data exists regarding the genetic and immunologic predictive biomarkers in rNB, which can be used to direct targeted therapies. Another barrier to clinical implementation of genetic testing of tumor samples from children with rNB is obtaining sufficient number of tumor cells from bone marrow (BM) specimens, the most easily accessible and common site of relapse. 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 (TGen) GEMTM sequencing platform for acquisition and gene panel sequencing of relapsed biological specimens in 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 PD-L1 expression) biomarkers in rNB. Additional potential novel biomarkers will also be evaluated and reported in this cohort of patients. This aim has immediate impact on the lives of children with rNB as it provides a clinical report to patients and their physicians detailing observed mutations and rNB subgroups and information on clinical trials that best match them. Our second aim will assess a novel method for enriching tumor cells from bone marrow aspirates containing less than 30% tumor involvement so that next generation sequencing can be performed. Our bone marrow (BM) enrichment protocol has both methodological and patient significance; 1) BM enrichment will allow a much larger group of rNB patients access to future personalized medicine trials and 2) Successful confirmation that BM enrichment can produce quality DNA for genetic analysis serves as proof of principal that this method can be used for genetic testing of BM with evidence of metastasis in other adult or pediatric solid tumors. In summary, our proposal will define the genetic and immunologic landscape of rNB and contribute to our understanding and ability to therapeutically target the dynamic alterations in tumor biology of children with rNB.
- Conditions
- Neuroblastoma
- Being tested
- Gene panel sequencing of tumor specimens
- Keywords
- Neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene panel sequencing of tumor specimensOther
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: Gene panel sequencing of tumor specimens |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Identify number of subjects who successfully provide tumor from either a procedure or archival sources and whether this tumor is adequate for analysis (>= 30% tumor present/specimen) as determined locally and centrally. Of these specimens that are found to be adequate and sent to the gene sequencing center, how many result in a clinical report being issued.
Measured: 6 weeks
How many patients with bone marrow aspirates performed that contain < 30% tumor cells are able to be enriched and genetic alterations identified
Identify number of subjects who successfully provide tumor from either a bone marrow aspiration or archival sources and < 30% tumor present. Was tumor enrichment attempted and # of patients where enrichment was attempted and successful to allow gene sequencing and identification of genetic/immunologic alterations. 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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2016 (estimated)
- Study start22 Aug 2016
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 93 participants (actual)
Sponsor and investigators
- Lead sponsor
- New Approaches to Neuroblastoma Therapy Consortium
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital Los Angeles, The Evan Foundation, St. Baldrick's Foundation, Press On Fund, Rising Tide Foundation
- Responsible party
- Sponsor
- Investigators
- Shahab Asgharzadeh, MD, Study chair, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02868268
- Sponsor's protocol ID
- N2015-01
- Other IDs
- N2015-01
- Status last verified
- Mar 2026
- First submitted
- 3 Jun 2016
- Last updated
- 23 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Individual participant data will be shared with the treating investigator and the study participant.
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- N2015-01: Neuroblastoma Precision Trial
- ClinicalTrials.gov ID
- NCT02868268
- Acronym / sponsor ID
- N2015-01
- Conditions
- Neuroblastoma
- Keywords
- Neuroblastoma
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 93 participants (actual)
- Lead sponsor
- New Approaches to Neuroblastoma Therapy ConsortiumUniversity, hospital or other
- Collaborators
- Children's Hospital Los Angeles, The Evan Foundation, St. Baldrick's Foundation, Press On Fund, Rising Tide Foundation
- Study start
- 22 Aug 2016 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 16 Aug 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Neuroblastoma (NB) is the most common extracranial solid tumor of childhood. High-risk NB is highly lethal and responsible for over 15% of childhood cancer related deaths. The majority of patients with metastatic NB respond to upfront cytotoxic chemotherapy, yet patients who die of recurrent disease do so from tumor acquired resistance to treatment. Thus, understanding the repertoire of tumor specific genomic alterations leading to tumor progression and therapy resistance is critical to devising novel targeted therapy options for patients with recurrent or refractory (r)NB. Limited data exists regarding the genetic and immunologic predictive biomarkers in rNB, which can be used to direct targeted therapies. Another barrier to clinical implementation of genetic testing of tumor samples from children with rNB is obtaining sufficient number of tumor cells from bone marrow (BM) specimens, the most easily accessible and common site of relapse. 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 (TGen) GEMTM sequencing platform for acquisition and gene panel sequencing of relapsed biological specimens in 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 PD-L1 expression) biomarkers in rNB. Additional potential novel biomarkers will also be evaluated and reported in this cohort of patients. This aim has immediate impact on the lives of children with rNB as it provides a clinical report to patients and their physicians detailing observed mutations and rNB subgroups and information on clinical trials that best match them. Our second aim will assess a novel method for enriching tumor cells from bone marrow aspirates containing less than 30% tumor involvement so that next generation sequencing can be performed. Our bone marrow (BM) enrichment protocol has both methodological and patient significance; 1) BM enrichment will allow a much larger group of rNB patients access to future personalized medicine trials and 2) Successful confirmation that BM enrichment can produce quality DNA for genetic analysis serves as proof of principal that this method can be used for genetic testing of BM with evidence of metastasis in other adult or pediatric solid tumors. In summary, our proposal will define the genetic and immunologic landscape of rNB and contribute to our understanding and ability to therapeutically target the dynamic alterations in tumor biology of children with rNB.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Relapsed/Refractory Neuroblastoma Pts | – | Other: Gene panel sequencing of tumor specimensHistory 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. |
Who may qualify
- Ages
- 1 year to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- High risk neuroblastoma
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Gene panel sequencing of tumor specimensArchival 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. | Other | Relapsed/Refractory Neuroblastoma Pts |
Primary outcome
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.
Time frame: 6 weeks
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.
2 secondary outcomes
How many subjects are biopsied or provide available tumor that is found to be adequate for gene panel sequencing and produces a clinical report
Time frame: 6 weeks
How many patients with bone marrow aspirates performed that contain < 30% tumor cells are able to be enriched and genetic alterations identified
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2026.
- Individual participant dataSharing planned: Yes. Individual participant data will be shared with the treating investigator and the study participant.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shahab Asgharzadeh, MD study chair
Children's Hospital Los Angeles
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 11 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 23 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2026.