NeuroblastomaPhase 2PEDS-PLANNCT02559778
Pediatric Precision Laboratory Advanced Neuroblastoma Therapy
A Phase 2 study testing Ceritinib, dasatinib and sorafenib in people with Neuroblastoma, run by Giselle Sholler at 28 sites in 2 countries.
Trial 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 Newly Diagnosed High-Risk… Read more about this study
- Who is it for?
- Ages Up to 22 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Ceritinib, dasatinib and sorafenib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 500 participants
- Where
- 28 sitesin 2 countries
- Study dates
- Sep 2015to Sep 2035
- Phase
- Phase 2
- 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 Up to 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
None listed.
Who cannot join
- Participants who are 12-18 months of age with INSS Stage 4 and all stage 3 participants 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.
- Participants receiving any investigational drug concurrently.
- Participants 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 participant's ability to sign or the legal guardian's ability to sign the informed consent, and participant's ability to cooperate and participate in the study
Trial locations and contacts
28 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
28 sites in 2 countries
United States24 sites, 20 recruiting
- Recruiting
Augusta University Health
Augusta, Georgia, 30912
Kimberly Gray, kigray@augusta.edu
Coleen McDonough
- Recruiting
Dell Children's Blood and Cancer Center
Austin, Texas, 78723
Rhea Robinson, rmrobinson@ascension.org
Virginia Harrod
- Recruiting
University of Alabama/Children's of Alabama
Birmingham, Alabama, 35233
Bridget Tate, btate@peds.uab.edu
Elizabeth Alva
- Completed
St. Lukes
Boise, Idaho, 83712
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
Shanta Salzar, MD, salzers@musc.edu
Jaqueline Kraveka
- Recruiting
Levine Children's Hospital
Charlotte, North Carolina, 28204
Jontyce Green, Jontyce.Green@advocatehealth.org
Thomas Russell
- Completed
Advocate Aurora Research Institute
Chicago, Illinois, 60453
- Recruiting
Children's Medical Center
Dallas, Texas, 75235
Rachel Nam, rachel.nam@childrens.com
Tanya Watt
- Recruiting
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Mary Beth Readwin, mary.readwin2@corewellhealth.org
David Hoogstra
- Completed
Hackensack University Medical Center
Hackensack, New Jersey, 07601
- Recruiting
Connecticut Children's Hospital
Hartford, Connecticut, 06106
Adam Barselau, Abarselau@connecticutchildrens.org
Michael Isakoff
- Recruiting
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033
Penn State Clinical Trials, ExtractClinicalTrials@pennstatehealth.psu.edu
Valerie Brown
- Recruiting
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96813
Andrea Siu, andrea.siu@kapiolani.org
Randal Wada
- Completed
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Susan Hall, HallSF@archildrens.org
Kevin Bielamowicz
- Recruiting
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine
Louisville, Kentucky, 40202
Jennifer Miller, Jennifer.Miller4@nortonhealthcare.org
Michael Ferguson
- Recruiting
Nicklaus Children's Miami
Miami, Florida, 33155
Aixa Guadarrama, Aixa.Guadarrama@Nicklaushealth.org
Guillermo De Angulo
- Recruiting
Children's Hospital and Clinics of Minnesota
Minneapolis, Minnesota, 55404
Pauline Mitby, pauline.mitby@childrensmn.org
Jawhar Rawwas
- Recruiting
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Group Contact, PedOncRschOAK@ucsf.edu
Jennifer Michlitsch
- Recruiting
Arnold Palmer Hospital for Children
Orlando, Florida, 32806
Marie Frankos, marie.frankos@orlandohealth.com
Jamie Libes-Bander
- Recruiting
Randall Children's Hospital
Portland, Oregon, 97227
Aaron White, AJWHITE@lhs.org
Jason Glover
- Recruiting
Rady Children's Hospital
San Diego, California, 92123
Megan Saenz, msaenz@rchsd.org
William Roberts
- Recruiting
Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104
Gina Martin, RN, gina.martin@health.slu.edu
William Ferguson
- Recruiting
St. Joseph's Children's Hospital
Tampa, Florida, 33614
Jennifer Manns, RN, jennifer.manns@baycare.org
Don Eslin
Canada4 sites, 4 recruiting
- Recruiting
Alberta Children's Hospital
Calgary, Alberta, AB T3B 6A8
Sherry Qian, Sherry.Qian@albertahealthservices.ca
Melanie Finkbeiner
- Recruiting
CHU Sainte-Justine
Montreal, Quebec, QC H3S 2G4
Guillaume Leblanc, guillaume.leblanc.hsj@ssss.gouv.qc.ca
Pierre Tiera
- Recruiting
CHUQ
Québec, Quebec, QC G1V 4W6
Valerie-Eve Julien, Valerie-Eve.Julien@crchudequebec.ulaval.ca
Bruno Michon
- Recruiting
CIUSSS de l'Estrie-CHUS
Sherbrooke, Quebec, QC J1H 5H3
Cassandra Leblanc-Desrochers, cassandra.leblanc-desrochers.ciussse-chus@ssss.gouv.qc.ca
Josee Brossard
About this study
Official title: 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 Newly Diagnosed High-Risk Neuroblastoma
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 Newly Diagnosed High-Risk Neuroblastoma.
- Conditions
- Neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CeritinibDrug
Also called Zykadia
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.
dasatinibDrug
Also called Sprycel
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.
sorafenibDrug
Also called Nexavar
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.
vorinostatDrug
Also called ZOLINZA
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.
DFMODrug
Also called Eflornithine, α-difluoromethylornithine
DFMO will be given to Arm B during immunotherapy and then for 2 years as maintenance to all subjects completing immunotherapy.
| Study group | Role | What they receive |
|---|---|---|
| Standard Immunotherapy without DFMO | Active comparator | 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 standard anti-GD2 immunotherapy and isotretinoin. At the end of immunotherapy, DFMO will be given to all subjects BID for 730 days. Receives: Drug: Ceritinib; Drug: dasatinib; Drug: sorafenib; Drug: vorinostat |
| Standard Immunotherapy with DFMO | Active comparator | 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 standard anti-GD2 immunotherapy and isotretinoin PLUS twice a day DFMO. At the end of immunotherapy, all subjects will go on to receive DFMO twice a day for 730 days. Receives: Drug: Ceritinib; Drug: dasatinib; Drug: sorafenib; Drug: vorinostat; Drug: DFMO |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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:
- Subject has a targeted agent identified
- Receives 75% of dosing of medications while on study protocol during cycles 3-6
- Subject is not removed from study due to targeted agent drug related toxicity.
Measured: 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:
- Subject has a targeted agent identified
- Receives 75% of dosing of medications while on study protocol during cycles 3-6
- Subject is not removed from study due to targeted agent drug related toxicity.
Measured: 2 years
Secondary outcomes (5)
Number of days that subjects remain alive
To measure the response of treatments chosen based on:
- Overall response rate (ORR) after induction therapy
- Overall survival (OS)
Measured: 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.
To measure the response of treatments chosen based on:
- Overall response rate (ORR) after induction therapy
Measured: Up to 8 years
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
To compare toxicity effects of difluoromethylornithine (DFMO) in combination with anti-GD2 immunotherapy and isotretinoin versus anti-GD2 immunotherapy and isotretinoin alone.
Measured: 3 years
Amount of pain medicine required by Arm A versus Arm B
To compare level of pain medicine needed during immunotherapy in patients receiving difluoromethylornithine (DFMO) in combination with anti-GD2 immunotherapy and Isotretinoin versus those receiving anti-GD2 immunotherapy and isotretinoin alone.
Measured: 3 years
Number of subjects required to go off therapy due to treatment-related adverse events as assessed by CTCAE v4.0.
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:
- Subject has a targeted agent identified
- Receives 75% of dosing of medications while on study protocol during cycles 3-6
- Subject is not removed from study due to targeted agent drug related toxicity.
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 posted24 Sep 2015 (estimated)
- Study startSep 2015
- Primary completionSep 2030 (estimated)
- Study completionSep 2035 (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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Giselle Sholler
- Sponsor type
- University, hospital or other
- Collaborators
- Dell, Inc., Beat NB Cancer Foundation, K C Pharmaceuticals Inc., Team Parker for Life
- Responsible party
- Sponsor-investigator, Giselle Sholler, Milton S. Hershey Medical Center
- Investigators
- Giselle Sholler, MD, Study chair, Beat Childhood Cancer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02559778
- Sponsor's protocol ID
- NMTRC012
- Acronym
- PEDS-PLAN
- Status last verified
- Sep 2026
- First submitted
- 22 Sep 2015
- Last updated
- 4 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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 Newly Diagnosed High-Risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT02559778
- Acronym / sponsor ID
- PEDS-PLAN / NMTRC012
- Conditions
- Neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- Giselle ShollerUniversity, hospital or other
- Collaborators
- Dell, Inc., Beat NB Cancer Foundation, K C Pharmaceuticals Inc., Team Parker for Life
- Study start
- Sep 2015 (actual)
- Primary completion
- Sep 2030 (estimated)
- Study completion
- Sep 2035 (estimated)
- First posted
- 24 Sep 2015 (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.
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 Newly Diagnosed High-Risk Neuroblastoma.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard Immunotherapy without DFMO | Active comparator | Drug: Ceritinib, Drug: dasatinib, Drug: sorafenib, Drug: vorinostatOne 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 standard anti-GD2 immunotherapy and isotretinoin. At the end of immunotherapy, DFMO will be given to all subjects BID for 730 days. |
| Standard Immunotherapy with DFMO | Active comparator | Drug: Ceritinib, Drug: dasatinib, Drug: sorafenib, Drug: vorinostat, Drug: DFMOOne 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 standard anti-GD2 immunotherapy and isotretinoin PLUS twice a day DFMO. At the end of immunotherapy, all subjects will go on to receive DFMO twice a day for 730 days. |
Who may qualify
- Ages
- Up to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (0 listed)
Key exclusion criteria (4 listed)
- Participants who are 12-18 months of age with INSS Stage 4 and all stage 3 participants 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.
- Participants receiving any investigational drug concurrently.
- Participants 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 participant's ability to sign or the legal guardian's ability to sign the informed consent, and participant's ability to cooperate and participate in the 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 |
|---|---|---|
| CeritinibAlso: ZykadiaOne 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 | Standard Immunotherapy with DFMO, Standard Immunotherapy without DFMO |
| dasatinibAlso: SprycelOne 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 | Standard Immunotherapy with DFMO, Standard Immunotherapy without DFMO |
| sorafenibAlso: NexavarOne 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 | Standard Immunotherapy with DFMO, Standard Immunotherapy without DFMO |
| vorinostatAlso: ZOLINZAOne 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 | Standard Immunotherapy with DFMO, Standard Immunotherapy without DFMO |
| DFMOAlso: Eflornithine, α-difluoromethylornithineDFMO will be given to Arm B during immunotherapy and then for 2 years as maintenance to all subjects completing immunotherapy. | Drug | Standard Immunotherapy with DFMO |
Primary outcome
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.
5 secondary outcomes
Number of days that subjects remain alive
Time frame: 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.
Time frame: Up to 8 years
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 3 years
Amount of pain medicine required by Arm A versus Arm B
Time frame: 3 years
Number of subjects required to go off therapy due to treatment-related adverse events as assessed by CTCAE v4.0.
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 4 Sep 2026.
- Beat Childhood Cancer Main WebsiteLink provided by the sponsor.
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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.
Giselle Sholler, MD study chair
Beat Childhood Cancer
28 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ceritinib, dasatinib and sorafenib in people with Neuroblastoma, run by Giselle Sholler at 28 sites in 2 countries.
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 0 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 4 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, Canada. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact BCC Enroll (7175310003, BCCEnroll@pennstatehealth.psu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of days from start of therapy to date of first relapse (time frame: Up to 8 years). The study lists 3 primary outcomes in all. 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 4 Sep 2026.