Pediatric Precision Laboratory Advanced Neuroblastoma Therapy
PEDS-PLAN
A Study Using Molecular Guided Therapy With Induction Chemotherapy Followed by a Randomized Controlled Trial of Standard Immunotherapy With or Without DFMO Followed by DFMO Maintenance for Subjects With Newly Diagnosed High-Risk Neuroblastoma
1 other identifier
interventional
500
2 countries
28
Brief Summary
A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with standard therapy followed by a Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Sep 2015
Longer than P75 for phase_2
28 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
Study Start
First participant enrolled
September 1, 2015
CompletedFirst Submitted
Initial submission to the registry
September 22, 2015
CompletedFirst Posted
Study publicly available on registry
September 24, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 1, 2035
April 28, 2026
April 1, 2026
15 years
September 22, 2015
April 24, 2026
Conditions
Outcome Measures
Primary Outcomes (3)
Number of days from start of therapy to date of first relapse
To measure the response of treatments chosen based on: • Event free survival (EFS)
Up to 8 years
Number of subjects that have a targeted agent chosen for treatment.
At completion of the induction therapy, the investigators will determine feasibility of adding molecularly guided targeted therapy to standard of care chemotherapy. Feasibility will be defined as: 1. Subject has a targeted agent identified 2. Receives 75% of dosing of medications while on study protocol during cycles 3-6 3. Subject is not removed from study due to targeted agent drug related toxicity.
2 years
Number of subjects that receive 75% of dosing of medications while on study protocol during cycles 3-6.
At completion of the induction therapy, the investigators will determine feasibility of adding molecularly guided targeted therapy to standard of care chemotherapy. Feasibility will be defined as: 1. Subject has a targeted agent identified 2. Receives 75% of dosing of medications while on study protocol during cycles 3-6 3. Subject is not removed from study due to targeted agent drug related toxicity.
2 years
Secondary Outcomes (5)
Number of days that subjects remain alive
3 years plus 5 years follow up
Overall Response Rate (ORR) of Participants by the presence of radiologically assessable disease by cross-sectional CT or MRI imaging and/or by MIBG or PET scans.
Up to 8 years
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
3 years
Amount of pain medicine required by Arm A versus Arm B
3 years
Number of subjects required to go off therapy due to treatment-related adverse events as assessed by CTCAE v4.0.
1 year
Study Arms (2)
Standard Immunotherapy without DFMO
ACTIVE COMPARATOROne of the following drugs will be chosen for each subject based on molecular guided results: ceritinib, dasatinib, sorafenib or vorinostat. This will be followed standard immunotherapy with Dinutuximab/GM-CSF/IL-2 and isotretinoin. At the end of immunotherapy, DFMO will be given to all subjects BID for 730 days.
Standard Immunotherapy with DFMO
ACTIVE COMPARATOROne of the following drugs will be chosen for each subject based on molecular guided results: ceritinib, dasatinib, sorafenib or vorinostat. This will be followed standard immunotherapy with Dinutuximab/GM-CSF/IL-2 and isotretinoin PLUS 1000mg/m2 BID of DFMO. At the end of immunotherapy, all subjects will go on to receive DFMO BID for 730 days.
Interventions
One of the following drugs will be chosen for each subject based on molecular guided results: Ceritinib, dasatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy +/- DFMO, and then all subjects will receive DFMO for 2 years as maintenance.
One of the following drugs will be chosen for each subject based on molecular guided results: Ceritinib, dasatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy +/- DFMO, and then all subjects will receive DFMO for 2 years as maintenance.
One of the following drugs will be chosen for each subject based on molecular guided results: Ceritinib, dasatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy +/- DFMO, and then all subjects will receive DFMO for 2 years as maintenance.
One of the following drugs will be chosen for each subject based on molecular guided results: Ceritinib, dasatinib, sorafenib or vorinostat. This will be followed by consolidation, immunotherapy +/- DFMO, and then all subjects will receive DFMO for 2 years as maintenance.
DFMO will be given to Arm B during immunotherapy and then for 2 years as maintenance to all subjects completing immunotherapy.
Eligibility Criteria
You may not qualify if:
- Subjects who are 12-18 months of age with INSS Stage 4 and all stage 3 subjects with favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index \> 1) are not eligible.
- Lactating females are not eligible unless they have agreed not to breastfeed their infants.
- Subjects receiving any investigational drug concurrently.
- Subjects with any other medical condition, including but not limited to malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Beat NB Cancer Foundationcollaborator
- Team Parker for Lifecollaborator
- Giselle Shollerlead
- Dell, Inc.collaborator
- K C Pharmaceuticals Inc.collaborator
Study Sites (28)
University of Alabama/Children's of Alabama
Birmingham, Alabama, 35233, United States
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
Rady Children's Hospital
San Diego, California, 92123, United States
Connecticut Children's Hospital
Hartford, Connecticut, 06106, United States
Nicklaus Children's Miami
Miami, Florida, 33155, United States
Arnold Palmer Hospital for Children
Orlando, Florida, 32806, United States
St. Joseph's Children's Hospital
Tampa, Florida, 33614, United States
Augusta University Health
Augusta, Georgia, 30912, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96813, United States
St. Lukes
Boise, Idaho, 83712, United States
Advocate Aurora Research Institute
Chicago, Illinois, 60453, United States
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine
Louisville, Kentucky, 40202, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
Children's Hospital and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Levine Children's Hospital
Charlotte, North Carolina, 28204, United States
Randall Children's Hospital
Portland, Oregon, 97227, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Dell Children's Blood and Cancer Center
Austin, Texas, 78723, United States
Children's Medical Center
Dallas, Texas, 75235, United States
Alberta Children's Hospital
Calgary, Alberta, AB T3B 6A8, Canada
CHU Sainte-Justine
Montreal, Quebec, QC H3S 2G4, Canada
CHUQ
Québec, Quebec, QC G1V 4W6, Canada
CIUSSS de l'Estrie-CHUS
Sherbrooke, Quebec, QC J1H 5H3, Canada
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Giselle Sholler, MD
Beat Childhood Cancer
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Beat Childhood Cancer Chair
Study Record Dates
First Submitted
September 22, 2015
First Posted
September 24, 2015
Study Start
September 1, 2015
Primary Completion (Estimated)
September 1, 2030
Study Completion (Estimated)
September 1, 2035
Last Updated
April 28, 2026
Record last verified: 2026-04