NCT02559778 · Giselle Sholler
Pediatric Precision Laboratory Advanced Neuroblastoma Therapy
(PEDS-PLAN)
What this study is about
A forward-looking open label, conducted at multiple hospitals study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with standard therapy followed by a randomly assigned Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma.
View original scientific description
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.
Interventions
DRUG
Ceritinib
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.
DRUG
dasatinib
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.
DRUG
sorafenib
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.
DRUG
vorinostat
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.
DRUG
DFMO
DFMO will be given to Arm B during immunotherapy and then for 2 years as maintenance to all subjects completing immunotherapy.
Primary outcome measures
Number of days from start of therapy to date of first relapse
Time frame: Up to 8 years
To measure the response of treatments chosen based on: • Event free survival (EFS)
Number of subjects that have a targeted agent chosen for treatment.
Time frame: 2 years
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.
Number of subjects that receive 75% of dosing of medications while on study protocol during cycles 3-6.
Time frame: 2 years
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Part A- CLOSED: 1. Diagnosis: Subjects must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. Subjects with the following disease stages at diagnosis are eligible, if they meet the other specified criteria: a) Subjects with newly diagnosed neuroblastoma with INSS Stage 4 are eligible with the following: i. Age \> 18 months (\> 547 days) regardless of biologic features or ii. Age 12-18 months (365-547 days) with any of the following 3 unfavorable biologic features (MYCN amplification, unfavorable pathology and/or DNA index = 1) or iii. MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features. b) Subjects with newly diagnosed neuroblastoma with INSS Stage 3 are eligible with the following: i. MYCN amplification (\> 4-fold increase in MYCN s
Where
- Birmingham, Alabama
- Little Rock, Arkansas
- Oakland, California
- San Diego, California
- Hartford, Connecticut
- Miami, Florida
- Orlando, Florida
- Tampa, Florida
- Augusta, Georgia
- Honolulu, Hawaii
- Boise, Idaho
- Chicago, Illinois
And 12 more locations — see the full list below.
Collaborators
Dell, Inc., Beat NB Cancer Foundation, K C Pharmaceuticals Inc., Team Parker for Life
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 28, 2026 · Source of record for eligibility and locations