NCT03786783CompletedPhase 2
Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma
A Pilot Induction Regimen Incorporating Chimeric 14.18 Antibody (ch14.18, Dinutuximab) (NSC# 764038) and Sargramostim (GM-CSF) for the Treatment of Newly Diagnosed High-Risk Neuroblastoma
Source: ClinicalTrials.gov · Last updated 2 Jun 2026
Brief summary (registry, in English)
This phase II pilot trial studies the side effects and how well dinutuximab and sargramostim work when combined with chemotherapy in patients with high-risk neuroblastoma. Immunotherapy with monoclonal antibodies, such as dinutuximab, may induce changes in the body's immune system and may interfere with the ability of tumor cells to grow and spread. Sargramostim helps the body produce normal infection-fighting white blood cells. These cells also help the dinutuximab work better. Giving chemotherapy before a stem cell transplant, with drugs such as cisplatin, etoposide, vincristine, doxorubicin, cyclophosphamide, thiotepa, melphalan, etoposide, carboplatin, topotecan, and isotretinoin, helps kill cancer cells that are in the body and helps make room in a patient's bone marrow for new blood-forming cells (stem cells). Giving dinutuximab and sargramostim with combination chemotherapy may work better than combination chemotherapy alone in treating patients with high-risk neuroblastoma.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 42 (actual)
- First submitted
- 24 Dec 2018
- Start date
- 4 Mar 2019
- Primary completion date
- 31 Dec 2021
- Lead sponsor
- National Cancer Institute (NCI)
- Diseases
- Neuroblastoma, Childhood cancers
- NCT number
- NCT03786783
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 42
- From 4 Mar 2019 to the primary completion date, 31 Dec 2021
- 10 sites in 3 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Female patients who are pregnant are ineligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential.
- Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method during study therapy and for two months after the last dose of ch14.18 (dinutuximab) are not eligible.
Other illness
- Patients with bone marrow failure syndromes.
- Patients on immunosuppressive medications (e.g. tacrolimus, cyclosporine, corticosteroids for reasons other than prevention/treatment of allergic reactions, adrenal replacement therapy, etc.) are not eligible.
Age
- Patients \>18 months of age with INRG stage L2, MYCN non-amplified, regardless of additional biologic features.
- Patients that are \>= 12 and =\< 18 months of age with INRG stage M and all 3 favorable biologic features (i.e., non-amplified MYCN, favorable pathology, and deoxyribonucleic acid \[DNA\] index \> 1) are not eligible.
Other
- Lactating females who plan to breastfeed their infants.
Who the study is looking for
- Patients must be enrolled on ANBL00B1 or APEC14B1 prior to enrollment on ANBL17P1.
- Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. The following disease groups are eligible:
- Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features:
- MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features; OR
- Age \> 547 days regardless of biologic features;
- Patients with INRG stage MS disease with MYCN amplification
- Patients with INRG stage L2 disease with MYCN amplification
- Patients \> 547 days of age initially diagnosed with INRG stage L1, L2 or MS disease who progress to stage M without prior chemotherapy may enroll within 4 weeks of progression to stage M.
- Patients \>= 365 days of age initially diagnosed with MYCN amplified INRG stage L1 disease who progress to stage M without systemic therapy may enroll within 4 weeks of progression to stage M.
- Patients initially recognized to have high-risk disease must have had no prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing as described).
- Patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high risk disease but subsequently found to meet the criteria will also be eligible.
- Patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible.
- Creatinine clearance (CrCl) or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/sex as follows:
- Age 1 month to \< 6 months (male 0.4 mg/dL, female 0.4 mg/dL)
- Age 6 months to \< 1 year (male 0.5 mg/dL, female 0.5 mg/dL)
- Age 1 to \< 2 years (male 0.6 mg/dL, female 0.6 mg/dL)
- Age 2 to \< 6 years (male 0.8 mg/dL, female 0.8 mg/dL)
- Age 6 to \< 10 years (male 1 mg/dL, female 1 mg/dL)
- Age 10 to \< 13 years (male 1.2 mg/dL, female 1.2 mg/dL)
- Age 13 to \< 16 years (male 1.5 mg/dL, female 1.4 mg/dL)
- Age \>= 16 years (male 1.7 mg/dL, female 1.4 mg/dL) (within 7 days prior to enrollment).
- Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment).
- Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 10 x ULN. For the purposes of this study, ULN for ALT is 45 IU/L (within 7 days prior to enrollment).
- Shortening fraction of \>= 27% by echocardiogram (within 7 days prior to enrollment).
- Ejection fraction of \>= 50% by echocardiogram or radionuclide angiogram (within 7 days prior to enrollment).
- No known contraindication to peripheral blood stem cell (PBSC) collection. Examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure.
- All patients and/or their parents or legal guardians must sign a written informed consent.
- All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.
Age in the registry: - to 30 years · Sex: All · Does not accept healthy volunteers
Sites (10)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Royal Children's Hospital Coordinator of this site? Confirm its status | Parkville | Australia | Unknown status |
| The Children's Hospital at Westmead Coordinator of this site? Confirm its status | Westmead | Australia | Unknown status |
| Starship Children's Hospital Coordinator of this site? Confirm its status | Grafton | New Zealand | Unknown status |
| Dana-Farber Cancer Institute Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Children's Hospital Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Saint Jude Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
| NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Children's Hospital of Pittsburgh of UPMC Coordinator of this site? Confirm its status | Pittsburgh | United States | Unknown status |
| Primary Children's Hospital Coordinator of this site? Confirm its status | Salt Lake City | United States | Unknown status |
| Children's National Medical Center Coordinator of this site? Confirm its status | Washington D.C. | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.