Pediatric Precision Laboratory Advanced Neuroblastoma Therapy
NCT ID: NCT02559778
Last Updated: 2025-08-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
500 participants
INTERVENTIONAL
2015-09-30
2035-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Standard Immunotherapy without DFMO
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 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.
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.
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.
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.
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.
Standard Immunotherapy with DFMO
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 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.
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.
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.
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.
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.
DFMO
DFMO will be given to Arm B during immunotherapy and then for 2 years as maintenance to all subjects completing immunotherapy.
Interventions
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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.
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.
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.
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.
DFMO
DFMO will be given to Arm B during immunotherapy and then for 2 years as maintenance to all subjects completing immunotherapy.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
b) Subjects with newly diagnosed neuroblastoma with INSS Stage 3 are eligible with the following: i. MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features or ii. Age \> 18 months (\> 547 days) with unfavorable pathology, regardless of MYCN status.
c) Subjects with newly diagnosed neuroblastoma with INSS Stage 2A/2B with MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features.
2. Subjects must be age ≤ 21 years at initial diagnosis
3. Subjects must not have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (as per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification status and histology.
4. Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing.
5. Ability to tolerate PBSC collection: No known contraindication to PBSC collection. Examples of contraindications would include a weight or size less than that determined to be feasible at the collecting institution, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure.
Part A and B both- Part A CLOSED, Part B- OPEN:
6. Adequate Cardiac Function Defined As:
1. Shortening fraction of ≥ 27% by echocardiogram, or
2. Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram.
7. Adequate liver function must be demonstrated, defined as:
c. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND d. ALT (SGPT) \< 10 x upper limit of normal (ULN) for age
8. Subjects must have adequate renal function defined as a serum creatinine based on age/gender as follows:
Age Maximum Serum Creatinine (mg/dL) Male Female 1 month to \< 6 months 0.4 0.4 6 months to \< 1 year 0.5 0.5 1 to \< 2 years 0.6 0.6 2 to \< 6 year 0.8 0.8 6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4
≥ 16 years 1.7 1.4
9. A negative serum pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses)
10. Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.
11. Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines.
Part B- OPEN:
12. All patients must have a pathologically confirmed diagnosis of neuroblastoma, be age ≤ 21 years at initial diagnosis, and classified as high risk by the criteria used by COG or SIOPEN at the time of diagnosis. Exception: patients who are initially diagnosed as non-high-risk neuroblastoma, but later converted (and/or relapsed) to high risk neuroblastoma are also eligible.
13. Previous Therapy- subjects must fit into one of the strata categories listed in section 10.5 to be eligible to enroll on Part B of this study.
14. Pre-enrollment tumor survey:
Prior to enrollment on Part B, a determination of mandatory disease staging must be performed. Tumor imaging studies including CT or MRI, MIBG or PET, and VMA/HVA (PET scan should be done for patients with prior disease that was MIBG non-avid). Bone marrow aspirates and biopsies are required.
This disease assessment is required for eligibility and should be done preferably within 2 weeks, but must be done within a maximum of 4 weeks before first dose of study drug.
15. Timing- Enrollment to occur prior to Day + 120 post-transplant, preferably when the subject is within 28 days after completing local radiation therapy (if given).
Exclusion Criteria
2. Lactating females are not eligible unless they have agreed not to breastfeed their infants.
3. Subjects receiving any investigational drug concurrently.
4. 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
22 Years
ALL
No
Sponsors
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Dell, Inc.
INDUSTRY
Beat NB Cancer Foundation
OTHER
K C Pharmaceuticals Inc.
INDUSTRY
Team Parker for Life
UNKNOWN
Giselle Sholler
OTHER
Responsible Party
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Giselle Sholler
Beat Childhood Cancer Chair
Principal Investigators
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Giselle Sholler, MD
Role: STUDY_CHAIR
Beat Childhood Cancer
Locations
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University of Alabama, Children's of Alabama
Birmingham, Alabama, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
UCSF Benioff Children's Hospital Oakland-
Oakland, California, United States
Rady Children's Hospital
San Diego, California, United States
Connecticut Children's Hospital
Hartford, Connecticut, United States
Nicklaus Children's Miami
Miami, Florida, United States
Arnold Palmer Hospital for Children
Orlando, Florida, United States
St. Joseph's Children's Hospital
Tampa, Florida, United States
Augusta University Health
Augusta, Georgia, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States
St. Lukes
Boise, Idaho, United States
Advocate Children's Medical Group
Chicago, Illinois, United States
University of Louisville
Louisville, Kentucky, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Children's Hospital and Clinics of Minnesota
Minneapolis, Minnesota, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States
Cardinal Glennon Children's Medical Center
St Louis, Missouri, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Levine Children's Hospital
Charlotte, North Carolina, United States
Randall Children's Hospital
Portland, Oregon, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Dell Children's Blood and Cancer Center
Austin, Texas, United States
Children's Medical Center
Dallas, Texas, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States
UHC Sainte-Justine
Montreal, Quebec, Canada
Countries
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Central Contacts
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Facility Contacts
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Related Links
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Beat Childhood Cancer Main Website
Other Identifiers
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NMTRC012
Identifier Type: -
Identifier Source: org_study_id
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