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CompletedNCT03786783Updated Jun 2, 2026Results posted

Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma

A Phase 2 interventional study of Autologous Hematopoietic Stem Cell Transplantation and Carboplatin in Ganglioneuroblastoma and High Risk Neuroblastoma, sponsored by National Cancer Institute (NCI). Completed at 10 sites in 3 countries. Open to participants aged Up to 30 Years. Per ClinicalTrials.gov, last updated 2026-06-02.

Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
42
Allocation
Not applicable
Ages
Up to 30 Years
Sex
All
01

Study summary

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.

Read the detailed description

PRIMARY OBJECTIVE:

I. To assess the feasibility and tolerability of administering ch14.18 (dinutuximab) and sargramostim (GM-CSF) in combination with a multi-agent chemotherapy regimen during cycles 3-5 of the Induction phase for patients with newly-diagnosed high-risk neuroblastoma.

SECONDARY OBJECTIVE:

I. To describe the response rates, event-free survival (EFS) and overall survival (OS) for patients receiving the combination of standard Induction chemotherapy and ch14.18 (dinutuximab) followed by tandem transplant, radiation therapy, and post-consolidation immunotherapy.

EXPLORATORY OBJECTIVES:

I. To describe the clinical relevance of naturally occurring anti-glycan antibodies in patients receiving ch14.18 (dinutuximab).

II. To describe the clinical relevance of natural killer (NK) receptor NKp30 isoforms in patients receiving ch14.18 (dinutuximab).

III. To describe the association between host factors, including human anti-chimeric antibodies (HACA), and response to protocol therapy.

IV. To describe the immune environment (gene expression; immune effector cells, activities and signaling molecules; immune target expression) during and following treatment.

V. To describe the association between levels of circulating GD2, and tumor cell GD2 expression with response to therapy.

OUTLINE:

INDUCTION CYCLES 1-2 (21 days): Patients receive cyclophosphamide intravenously (IV) over 15-30 minutes and topotecan IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 2 cycles in the absence of disease progression or unacceptable toxicity.

INDUCTION CYCLE 3: Patients receive cisplatin IV over 1 hour on days 1-3, etoposide IV over 2 hours on days 1-3, dinutuximab IV over 10-20 hours on days 2-5, and sargramostim subcutaneously (SC) on day 6 or 7 of a 21-day cycle.

INDUCTION CYCLE 4: Patients receive vincristine IV over 1 minute on day 1, doxorubicin IV over 1-15 minutes on days 1-2, cyclophosphamide IV over 1 hour on days 1-2, dinutuximab IV over 10-20 hours on days 2-5, and sargramostim SC on day 6 or 7 of a 21-day cycle.

INDUCTION CYCLE 5: Patients receive cisplatin IV over 1 hour on days 1-3, etoposide IV over 2 hours on days 1-3, dinutuximab IV over 10-20 hours on days 2-5, and sargramostim SC on day 6 or 7 of a 21-day cycle.

Patients may undergo surgery after the fourth or fifth cycle of Induction at the discretion of treating doctor. Patients with stable disease or better tumor response at the end of Induction proceed to Consolidation. Consolidation treatment begins between 4 and 6 weeks from the start date of Induction chemotherapy cycle 5. For patients who have surgical resection delayed until after Induction chemotherapy cycle 5, Consolidation starts within 4 weeks from the date of surgery.

CONSOLIDATION #1: Patients receive thiotepa IV over 2 hours on days -7 to -5 and cyclophosphamide IV over 1 hour on days -5 to -2. Patients then undergo autologous stem cell transplant (ASCT) on day 0.

CONSOLIDATION #2: Patients receive melphalan IV over 30 minutes on days -7 to -5, etoposide IV over 24 hours on days -7 to -4, and carboplatin IV over 24 hours on days -7 to -4. Patients then undergo ASCT on day 0.

RADIATION THERAPY: Beginning 42-80 days following Consolidation #2, patients receive external beam radiation therapy (EBRT) daily for up to 20 days.

Patients then receive post-Consolidation therapy starting at least 1 week following radiation therapy.

POST-CONSOLIDATION CYCLES 1-5: Patients receive sargramostim SC on days 1-14, dinutuximab IV over 10-20 hours on days 4-7, and isotretinoin orally (PO) twice daily (BID) on days 11-24. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity.

POST-CONSOLIDATION CYCLE 6: Patients receive isotretinoin PO BID on days 15-28 of a 28-day cycle.

After completion of study treatment, patients are followed up at months 3, 6, 9, 12, 15, 18, 24, 30, 36, 42, 48, 54, and 60.

02

Conditions studied

  • Ganglioneuroblastoma
  • High Risk Neuroblastoma

Browse trials for

03

In context

Ganglioneuroblastoma

23 studies on the registry are indexed under Ganglioneuroblastoma; 10 are open to participants now.

This study's enrollment of 42 is below the median of 71 across 18 interventional studies indexed under Ganglioneuroblastoma.

Browse Ganglioneuroblastoma studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,506 studies on the registry; 334 are open to participants now.

Of its 402 completed or terminated interventional studies of FDA-regulated products, 365 (91%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 30 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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.

Exclusion criteria

Exclusion Criteria:

  • Patients >18 months of age with INRG stage L2, MYCN non-amplified, regardless of additional biologic features.
  • Patients with bone marrow failure syndromes.
  • 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.
  • 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.
  • 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.
  • Lactating females who plan to breastfeed their infants.
  • 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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
42 participants (actual)

Study arms

  • Experimental
    Treatment(chemotherapy, dinutuximab, sargramostim, ASCT, EBRT)

    See Detailed Description

    Procedure: Autologous Hematopoietic Stem Cell Transplantation · Drug: Carboplatin · Drug: Cisplatin · Drug: Cyclophosphamide · Drug: Dexrazoxane · Biological: Dinutuximab · Drug: Doxorubicin · Drug: Etoposide · Radiation: External Beam Radiation Therapy · Drug: Isotretinoin · Drug: Melphalan · Biological: Sargramostim · Drug: Thiotepa · Drug: Topotecan · Drug: Vincristine

Interventions

  • ProcedureAutologous Hematopoietic Stem Cell Transplantation

    Undergo ASCT

    Also known as: AHSCT, Autologous, Autologous Hematopoietic Cell Transplantation, Autologous Stem Cell Transplant, Autologous Stem Cell Transplantation, Stem Cell Transplantation, Autologous

  • DrugCarboplatin

    Given IV

    Also known as: Blastocarb, Carboplat, Carboplatin Hexal, Carboplatino, Carboplatinum, Carbosin, Carbosol, Carbotec, CBDCA, Displata, Ercar, JM-8, JM8, Nealorin, Novoplatinum, Paraplatin, Paraplatin AQ, Paraplatine, Platinwas, Ribocarbo

  • DrugCisplatin

    Given IV

    Also known as: Abiplatin, Blastolem, Briplatin, CDDP, Cis-diammine-dichloroplatinum, Cis-diamminedichloridoplatinum, Cis-diamminedichloro Platinum (II), Cis-diamminedichloroplatinum, Cis-dichloroammine Platinum (II), Cis-platinous Diamine Dichloride, Cis-platinum, Cis-platinum II, Cis-platinum II Diamine Dichloride, Cismaplat, Cisplatina, Cisplatinum, Cisplatyl, Citoplatino, Citosin, Cysplatyna, DDP, Lederplatin, Metaplatin, Neoplatin, Peyrone's Chloride, Peyrone's Salt, Placis, Plastistil, Platamine, Platiblastin, Platiblastin-S, Platinex, Platinol, Platinol- AQ, Platinol-AQ, Platinol-AQ VHA Plus, Platinoxan, Platinum, Platinum Diamminodichloride, Platiran, Platistin, Platosin

  • DrugCyclophosphamide

    Given IV

    Also known as: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518, B-518, B518, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamide Monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR 138719, WR- 138719, WR-138719, WR138719

  • DrugDexrazoxane

    Given IV

    Also known as: 2, 6-Piperazinedione, 4,4'-propylenedi-, (P)- (8CI), 2,6-Piperazinedione, 4, 4'-(1-methyl-1,2-ethanediyl)bis-, (S)- (9CI), ADR 529, ADR-529, ADR529, ICRF 187, ICRF-187, ICRF187, Razoxane (+)-form, Soluble ICRF (L-isomer)

  • BiologicalDinutuximab

    Given IV

    Also known as: Ch 14.18UTC, Ch14.18, Dinutuximab Beta, MOAB Ch14.18, monoclonal antibody Ch14.18, Qarziba, Unituxin

  • DrugDoxorubicin

    Given IV

    Also known as: Adriablastin, Hydroxydaunomycin, Hydroxyl Daunorubicin, Hydroxyldaunorubicin

  • DrugEtoposide

    Given IV

    Also known as: Demethyl Epipodophyllotoxin Ethylidine Glucoside, EPEG, Lastet, Toposar, Vepesid, VP 16, VP 16-213, VP 16213, VP-16, VP-16-213, VP-16213, VP16, VP16213

  • RadiationExternal Beam Radiation Therapy

    Undergo EBRT

    Also known as: Definitive Radiation Therapy, EBRT, External Beam Radiation, External Beam Radiotherapy, External Beam Radiotherapy (conventional), External Beam RT, external radiation, External Radiation Therapy, external-beam radiation, Radiation, External Beam, Teleradiotherapy, Teletherapy, Teletherapy Radiation

  • DrugIsotretinoin

    Given PO

    Also known as: 13-cis retinoic acid, 13-cis-Retinoate, 13-cis-Retinoic Acid, 13-cis-Vitamin A Acid, 13-cRA, Absorica, Accure, Accutane, Amnesteem, cis-Retinoic Acid, Cistane, Claravis, Isotretinoinum, Isotrex, Isotrexin, Myorisan, Neovitamin A, Neovitamin A Acid, Oratane, Retinoicacid-13-cis, Ro 4-3780, Ro-4-3780, Roaccutan, Roaccutane, Roacutan, Sotret, ZENATANE

  • DrugMelphalan

    Given IV

    Also known as: Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine Mustard, L-Sarcolysin, L-Sarcolysin Phenylalanine mustard, L-Sarcolysine, Melphalan for Injection-Hepatic Delivery System, Melphalanum, Phenylalanine Mustard, Phenylalanine Nitrogen Mustard, Sarcoclorin, Sarkolysin, WR-19813

  • BiologicalSargramostim

    Given SC

    Also known as: 23-L-Leucinecolony-Stimulating Factor 2, DRG-0012, Leukine, Prokine, rhu GM-CFS, Sagramostim, Sargramostatin

  • DrugThiotepa

    Given IV

    Also known as: 1,1',1''-Phosphinothioylidynetrisaziridine, Girostan, N,N', N''-Triethylenethiophosphoramide, Oncotiotepa, SH 105, SH-105, SH105, STEPA, Tepadina, Tepylute, TESPA, Tespamin, Tespamine, Thio-Tepa, Thiofosfamide, Thiofozil, Thiophosphamide, Thiophosphoamide, Thiophosphoramide, Thiotef, Tifosyl, TIO TEF, Tio-tef, Triethylene Thiophosphoramide, Triethylenethiophosphoramide, Tris(1-aziridinyl)phosphine sulfide, TSPA, WR 45312

  • DrugTopotecan

    Given IV

    Also known as: Hycamptamine, Topotecan Lactone

  • DrugVincristine

    Given IV

    Also known as: LCR, Leurocristine, VCR, Vincrystine

06

What researchers measure

Primary outcomes

  1. Percentage of Participants With Unacceptable Toxicity

    Assessed with National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Assessed by estimation of the combined toxic death and unacceptable toxicity rate during Induction cycles 3-5 together with a 95% confidence interval.

    Time frame: Up to the first 5 cycles of treatment

  2. Percentage of Participants Who Are Feasibility "Failure"

    Feasibility "failures" were defined as patients that did not receive \>= 75% of the planned dinutuximab doses during Induction cycles 3-5. Assessed by estimation of the feasibility "failure" rate together with a 95% confidence interval.

    Time frame: Up to the first 5 cycles of treatment

Secondary outcomes

  1. Response Rate

    Per the revised INRC, response is comprised by responses in 3 components: primary tumor, soft tissue and bone metastases, and bone marrow. Primary and metastatic soft tissue sites were assessed using Response Evaluation Criteria in Solid Tumors and MIBG scans or FDG-PET scans if the tumor was MIBG non-avid. Bone marrow was assessed by histology or immunohistochemistry and cytology or immunocytology. Complete response (CR) - All components meet criteria for CR. Partial response (PR) - PR in at least one component and all other components are either CR, minimal disease (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with progressive disease (PD). Minor response (MR) - PR or CR in at least one component but at least one other component with stable disease; no component with PD. Stable disease (SD) - Stable disease in one component with no better than SD or NI in any other component; no component with PD. Progressive disease (PD) - Any component with PD.

    Time frame: Up to the first 5 cycles of treatment

  2. Event-free Survival

    Per the revised INRC, progressive disease is: 1) \> 20% increase in the longest diameter of the primary tumor, taking as reference the smallest sum and ¬\> increase of 5 mm in longest dimension, 2) Any new soft tissue lesion detected by CT/MRI that is MIBG avid or FDG-PET avid, 3) Any new soft tissue lesion seen on CT/MRI that is biopsied and found to be neuroblastoma or ganglioneuroblastoma, 4) Any new bone site that is MIBG avid, 5) Any new bone site that is FDG-PET avid and has CT/MRI findings of tumor or is histologically neuroblastoma or ganglioneuroblastoma 6) A metastatic soft tissue site with \> 20% increase in longest diameter, taking as reference the smallest sum on study, and with \> 5mm in sum of diameters of target soft tissue lesions, 7) A relative MIBG score ¬\> 1.2, 8) Bone marrow without tumor infiltration that becomes \>5% tumor infiltration, 9) Bone marrow with tumor infiltration that increases by \> 2-fold and has \> 20% tumor infiltration on reassessment.

    Time frame: Up to 1 year

  3. Overall Survival

    Kaplan-Meier method was used to estimate overall survival (OS). OS was defined as the time from study enrollment to death. 1-year OS is provided.

    Time frame: Up to 1 year

Other outcomes

  1. Incidence of Naturally Occurring Anti-glycan Antibodies

    The Incidence of Naturally Occurring Anti-glycan Antibodies was calculated, including placement of a 95% CI on the incidence. In addition, anti-glycan levels prior to the start of Induction therapy and prior to the start of post-Consolidation therapy was compared with Wilcoxon's signed-rank test for paired data.

    Time frame: Up to 5 years

  2. Incidence of Natural Killer (NK) Receptor NKp30 Isoforms

    Assessed by calculating the incidence of NK receptor NKp30 isoforms, including placement of a 95% CI on the incidence.

    Time frame: Up to 5 years

  3. Response of Host Factors, Including Naturally Occurring Anti-glycan Antibodies, KIR/KIR-L Genotyping, Fc Receptor Genotyping, Human Anti-chimeric Antibodies (HACA)

    Explored with Fisher's exact test for categorical and Wilcoxon rank-sum test for continuous host factors. Both the presence/absence and level of naturally occurring anti-glycan antibodies was considered. For the KIR/KIR-L analysis, patients were categorized as either matched or mismatched. Patients were grouped into one of the three genotype subgroups of Fc receptor genotyping for that analysis. The presence/absence of HACA, anti-idiotype, and pretreatment anti-therapeutic antibodies (PATA)/anti-allotype antibody was considered for the HACA analysis.

    Time frame: Up to 5 years

  4. Immune Environment (Gene Expression; Immune Effector Cells, Activities and Signaling Molecules; Immune Target Expression)

    The incidence of NK receptor NKp30 isoforms was calculated, including placement of a 95% CI on each incidence rate. Summary statistics were generated for serum cytokine (IL6, CXCL9) levels and gene expression of circulating immune function cells.

    Time frame: Up to 5 years

  5. Levels of Circulating GD2 and Tumor Cell GD2 Expression

    Assessed by exploring the relationship between response to treatment with \> PR (response vs. non- response) with circulating GD2 levels, and GD2 tumor cell expression following therapy with a Wilcoxon rank-sum test. Changes from baseline were also analyzed.

    Time frame: Up to 5 years

07

Results

Posted Mar 14, 2023

Participant flow

Patients with newly diagnosed high-risk Neuroblastoma enrolled between 1/14/2019 and 9/4/2020 across 10 institutions.

Participant flow — Overall Study
MilestoneTreatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Started42
Completed22
Not completed20
Withdrew: Death1
Withdrew: Physician decision6
Withdrew: Withdrawal by subject2
Withdrew: Disease progression7
Withdrew: Patient/parent refusal4

Outcome measures

PrimaryPercentage of Participants With Unacceptable Toxicity

Assessed with National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Assessed by estimation of the combined toxic death and unacceptable toxicity rate during Induction cycles 3-5 together with a 95% confidence interval.

Time frame:
Up to the first 5 cycles of treatment
Reported as:
Number · percentage of patients
Percentage of Participants With Unacceptable Toxicity
percentage of patientsTreatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Percentage of Participants With Unacceptable Toxicity0.0 (0.0 to 0.0)
PrimaryPercentage of Participants Who Are Feasibility "Failure"

Feasibility "failures" were defined as patients that did not receive \>= 75% of the planned dinutuximab doses during Induction cycles 3-5. Assessed by estimation of the feasibility "failure" rate together with a 95% confidence interval.

Time frame:
Up to the first 5 cycles of treatment
Reported as:
Number · Percentage of patients
Percentage of Participants Who Are Feasibility "Failure"
Percentage of patientsTreatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Percentage of Participants Who Are Feasibility "Failure"0.0 (0.0 to 0.0)
SecondaryResponse Rate

Per the revised INRC, response is comprised by responses in 3 components: primary tumor, soft tissue and bone metastases, and bone marrow. Primary and metastatic soft tissue sites were assessed using Response Evaluation Criteria in Solid Tumors and MIBG scans or FDG-PET scans if the tumor was MIBG non-avid. Bone marrow was assessed by histology or immunohistochemistry and cytology or immunocytology. Complete response (CR) - All components meet criteria for CR. Partial response (PR) - PR in at least one component and all other components are either CR, minimal disease (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with progressive disease (PD). Minor response (MR) - PR or CR in at least one component but at least one other component with stable disease; no component with PD. Stable disease (SD) - Stable disease in one component with no better than SD or NI in any other component; no component with PD. Progressive disease (PD) - Any component with PD.

Time frame:
Up to the first 5 cycles of treatment
Reported as:
Number · Percentage of patients
Response Rate
Percentage of patientsTreatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Response Rate78.6 (64.1 to 88.3)
SecondaryEvent-free Survival

Per the revised INRC, progressive disease is: 1) \> 20% increase in the longest diameter of the primary tumor, taking as reference the smallest sum and ¬\> increase of 5 mm in longest dimension, 2) Any new soft tissue lesion detected by CT/MRI that is MIBG avid or FDG-PET avid, 3) Any new soft tissue lesion seen on CT/MRI that is biopsied and found to be neuroblastoma or ganglioneuroblastoma, 4) Any new bone site that is MIBG avid, 5) Any new bone site that is FDG-PET avid and has CT/MRI findings of tumor or is histologically neuroblastoma or ganglioneuroblastoma 6) A metastatic soft tissue site with \> 20% increase in longest diameter, taking as reference the smallest sum on study, and with \> 5mm in sum of diameters of target soft tissue lesions, 7) A relative MIBG score ¬\> 1.2, 8) Bone marrow without tumor infiltration that becomes \>5% tumor infiltration, 9) Bone marrow with tumor infiltration that increases by \> 2-fold and has \> 20% tumor infiltration on reassessment.

Time frame:
Up to 1 year
Reported as:
Number · Percent Probability
Event-free Survival
Percent ProbabilityTreatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Event-free Survival82.6 (70.8 to 94.4)
SecondaryOverall Survival

Kaplan-Meier method was used to estimate overall survival (OS). OS was defined as the time from study enrollment to death. 1-year OS is provided.

Time frame:
Up to 1 year
Reported as:
Number · Percent Probability
Overall Survival
Percent ProbabilityTreatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Overall Survival95.0 (88.1 to 100.0)
Other pre-specifiedIncidence of Naturally Occurring Anti-glycan Antibodies

The Incidence of Naturally Occurring Anti-glycan Antibodies was calculated, including placement of a 95% CI on the incidence. In addition, anti-glycan levels prior to the start of Induction therapy and prior to the start of post-Consolidation therapy was compared with Wilcoxon's signed-rank test for paired data.

Time frame:
Up to 5 years

Results for this outcome have not been posted.

Other pre-specifiedIncidence of Natural Killer (NK) Receptor NKp30 Isoforms

Assessed by calculating the incidence of NK receptor NKp30 isoforms, including placement of a 95% CI on the incidence.

Time frame:
Up to 5 years

Results for this outcome have not been posted.

Other pre-specifiedResponse of Host Factors, Including Naturally Occurring Anti-glycan Antibodies, KIR/KIR-L Genotyping, Fc Receptor Genotyping, Human Anti-chimeric Antibodies (HACA)

Explored with Fisher's exact test for categorical and Wilcoxon rank-sum test for continuous host factors. Both the presence/absence and level of naturally occurring anti-glycan antibodies was considered. For the KIR/KIR-L analysis, patients were categorized as either matched or mismatched. Patients were grouped into one of the three genotype subgroups of Fc receptor genotyping for that analysis. The presence/absence of HACA, anti-idiotype, and pretreatment anti-therapeutic antibodies (PATA)/anti-allotype antibody was considered for the HACA analysis.

Time frame:
Up to 5 years

Results for this outcome have not been posted.

Other pre-specifiedImmune Environment (Gene Expression; Immune Effector Cells, Activities and Signaling Molecules; Immune Target Expression)

The incidence of NK receptor NKp30 isoforms was calculated, including placement of a 95% CI on each incidence rate. Summary statistics were generated for serum cytokine (IL6, CXCL9) levels and gene expression of circulating immune function cells.

Time frame:
Up to 5 years

Results for this outcome have not been posted.

Other pre-specifiedLevels of Circulating GD2 and Tumor Cell GD2 Expression

Assessed by exploring the relationship between response to treatment with \> PR (response vs. non- response) with circulating GD2 levels, and GD2 tumor cell expression following therapy with a Wilcoxon rank-sum test. Changes from baseline were also analyzed.

Time frame:
Up to 5 years

Results for this outcome have not been posted.

Adverse events

Collected over While patients were on Protocol Therapy, an average of 12 months. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)1/42 (2.4%)14/42 (33.3%)42/42 (100%)
Most frequent serious events
Showing 10 of 28
Most frequent serious events
EventTreatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
HypophosphatemiaMetabolism and nutrition disorders4/42
Chylous ascitesGastrointestinal disorders2/42
Hemolytic uremic syndromeBlood and lymphatic system disorders2/42
HypokalemiaMetabolism and nutrition disorders2/42
NauseaGastrointestinal disorders2/42
VomitingGastrointestinal disorders2/42
Abdominal painGastrointestinal disorders1/42
Acute kidney injuryRenal and urinary disorders1/42
Adult respiratory distress syndromeRespiratory, thoracic and mediastinal disorders1/42
Alanine aminotransferase increasedInvestigations1/42
Most frequent other events
Showing 10 of 87
Most frequent other events
EventTreatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Febrile neutropeniaBlood and lymphatic system disorders32/42
Mucositis oralGastrointestinal disorders31/42
AnorexiaMetabolism and nutrition disorders21/42
FeverGeneral disorders21/42
HypokalemiaMetabolism and nutrition disorders16/42
SepsisInfections and infestations16/42
Hearing impairedEar and labyrinth disorders15/42
HypertensionVascular disorders12/42
Alanine aminotransferase increasedInvestigations11/42
VomitingGastrointestinal disorders11/42

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
<=18 years42
Between 18 and 65 years0
>=65 years0
Age, Continuous
Age, Continuous(years)Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Median3.4 (0.3 to 17.4)
Sex: Female, Male
Sex: Female, Male(Participants)Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Female20
Male22
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
Hispanic or Latino4
Not Hispanic or Latino32
Unknown or Not Reported6
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
American Indian or Alaska Native0
Asian4
Native Hawaiian or Other Pacific Islander0
Black or African American4
White22
More than one race1
Unknown or Not Reported11
Region of Enrollment
Region of Enrollment(participants)Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)
United States30
Australia7
New Zealand5
08

Study locations

10 sites
  • Children's Hospital Los Angeles
    Los Angeles, California 90027, United States
  • Children's National Medical Center
    Washington D.C., District of Columbia 20010, United States
  • Dana-Farber Cancer Institute
    Boston, Massachusetts 02215, United States
  • NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
    New York, New York 10032, United States
  • Children's Hospital of Pittsburgh of UPMC
    Pittsburgh, Pennsylvania 15224, United States
  • Saint Jude Children's Research Hospital
    Memphis, Tennessee 38105, United States
  • Primary Children's Hospital
    Salt Lake City, Utah 84113, United States
  • The Children's Hospital at Westmead
    Westmead, New South Wales 2145, Australia
  • Royal Children's Hospital
    Parkville, Victoria 3052, Australia
  • Starship Children's Hospital
    Grafton, Auckland 1145, New Zealand
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Oct 24, 2019

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 2, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03786783
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Dec 26, 2018
Start date
Mar 4, 2019
Primary completion
Dec 31, 2021
Completion
Mar 31, 2026
Results posted
Mar 14, 2023
Last update
Jun 2, 2026

Study contacts

Sara M Federico
principal investigator · Children's Oncology Group

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in May 2026. You cannot join it, but the record below documents what was studied.

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