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Taking people in Puerto RicoPhase 3

Adds dinutuximab to standard treatment for children with high-risk neuroblastoma

Tests adding dinutuximab to chemotherapy in children with newly diagnosed high-risk neuroblastoma.

Key facts

Who runs it
National Cancer Institute (NCI)
Where
San Juan, Puerto RicoSee the siteAlso in 4 other countries, 179 sites
Trial code
Registry title
Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
Status
Recruiting
Phase
Phase 3
People taking part
478 people, planned
Started
19 Apr 2024
Expected to end
31 Dec 2029
Type of study
Interventional, 2 groupsOne group gets an existing treatment, for comparison.
Treatments tested
  • Biospecimen Collection
  • Bone Marrow Aspiration
  • Bone Marrow Biopsy
  • Carboplatin
  • Cisplatin
  • Computed Tomography
  • Cyclophosphamide
  • Dinutuximab
  • Doxorubicin
  • Echocardiography Test
  • Etoposide
  • FDG-Positron Emission Tomography and Computed Tomography Scan
  • Hematopoietic Cell Transplantation
  • Irinotecan
  • Isotretinoin
  • Leukapheresis
  • Magnetic Resonance Imaging
  • Melphalan
  • Multigated Acquisition Scan
  • Radiation Therapy
  • Radionuclide Imaging
  • Survey Administration
  • Temozolomide
  • Thiotepa
  • Topotecan
  • Tumor Resection
  • Vincristine
Record updated
30 October 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for children and young people up to 30 years old with newly diagnosed high-risk neuroblastoma. It tests adding dinutuximab to chemotherapy and surgery.

Age
Up to 30 years at the time of first diagnosis with high-risk disease.
Sex
All sexes can join.
Disease stage
Newly diagnosed high-risk neuroblastoma or nodular ganglioneuroblastoma, confirmed by tumor pathology or tumor cells in bone marrow with raised urinary catecholamines. It must be INRG stage L2, MS or M with MYCN amplification, or age 547 days or more with stage M. Some patients first diagnosed with stage L1, L2 or MS who progressed to stage M without systemic chemotherapy can also join.
Earlier treatment
No earlier anti-cancer treatment, with a few exceptions: urgent topotecan/cyclophosphamide, one cycle of low- or intermediate-risk chemotherapy, or emergency radiation to dangerous disease sites.
Biomarker or test result
MYCN amplification is needed for some groups. Clinical MYCN testing is not required for those aged 547 days or more with stage M. Patients must also be enrolled on APEC14B1 and have consented to testing through the Molecular Characterization Initiative.

Main reasons for not taking part

  • Age 365-546 days with INRG stage M and MYCN non-amplified disease
  • Age 547 days or more with INRG stage L2 and MYCN non-amplified disease
  • Known bone marrow failure syndromes
  • Long-term immunosuppressive medicines, apart from the uses the record allows
  • Pregnant women, or breastfeeding women who plan to continue

The registry criteria, word for word

Show all 21 criteriaShow less

Who can take part

  • Patients must be enrolled on APEC14B1 and have consented to testing through the Molecular Characterization Initiative (MCI), prior to enrollment on ANBL2131
  • ≤ 30 years at the time of initial diagnosis with high-risk disease
  • * Must have a diagnosis of neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines; Newly diagnosed, high risk neuroblastoma (HRNBL) defined as one of the following:; Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M and MYCN amplification; Age ≥ 547 days and INRG stage M regardless of biologic features (clinical MYCN testing not required prior to enrollment); Any age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to stage M without systemic chemotherapy; Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to stage M without systemic chemotherapy (clinical MYCN testing not required prior to enrollment)
  • Patients must have a body surface area (BSA) ≥ 0.25 m\^2
  • No prior anti-cancer therapy except as outlined below:; Patients initially recognized to have high-risk disease treated with topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing, and with consent; 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; Patients who received localized emergency radiation to sites of life threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis
  • Human immunodeficiency virus (HIV) -infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
  • A serum creatinine based on age/sex as follows:; 1 month to < 6 months: Male 0.4 mg/dL and female 0.4mg/dL; 6 months to < 1 year: Male 0.5 mg/dL and female 0.5 mg/dL; 1 to < 2 years: Male 0.6 mg/dL and female 0.6 mg/dL; 2 to < 6 years: Male 0.8 mg/dL and female 0.8 mg/dL; 6 to < 10 years: Male 1 mg/dL and female 1 mg/dL; 10 to < 13 years: Male 1.2 mg/dL and female 1.2 mg/dL; 13 to < 16 years: Male 1.5 mg/dL and female 1.4 mg/dL; ≥ 16 years: Male 1.7 mg/dL and female 1.4 mg/dL; The threshold creatinine values were derived from the Schwartz formula for estimating glomerular filtration rate (GFR) utilizing child length and stature data published by the Centers for Disease Control (CDC); or a 24-hour urine creatinine clearance ≥ 70 mL/min/1.73 m\^2 or; or a GFR ≥ 70 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method or direct small molecule clearance method (iothalamate or other molecule per institutional standard); Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility
  • Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age
  • Serum glutamic pyruvic transaminase (SGPT) (Alanine aminotransferase [ALT]) ≤ 10 x ULN*; Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L
  • * Shortening fraction of ≥ 27% by echocardiogram, or; Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram
  • Ability to tolerate Peripheral Blood Stem Cell (PBSC) collection:; No known contraindication to 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

Who cannot take part

  • Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required prior to enrollment for female patients of childbearing potential
  • Patients with known bone marrow failure syndromes
  • Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention/treatment of allergic reactions and adrenal replacement therapy are not eligible. Topical and inhaled corticosteroids are acceptable
  • Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy
  • All institutional, food and drug administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
  • Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features
  • Patients ≥ 547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features
  • Lactating females who plan to breastfeed their infants
  • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
  • All patients and/or their parents or legal guardians must sign a written informed consent

Where in Puerto Rico

Also in 4 other countries, 179 sites

  • University Pediatric HospitalSan Juan, Puerto RicoContact: Maria E. Echevarria (principal investigator)Taking people

About the trial

What the trial wants to find out

This phase III trial tests how well the addition of dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy works for treating children with newly diagnosed high-risk neuroblastoma. Dinutuximab is a monoclonal antibody that binds to a molecule called GD2, which is found on the surface of neuroblastoma cells, but is not present on many healthy or normal cells in the body. When dinutuximab binds to the neuroblastoma cells, it helps signal the immune system to kill the tumor cells. This helps the cells of the immune system kill the cancer cells, this is a type of immunotherapy. When chemotherapy and immunotherapy are given together, during the same treatment cycle, it is called chemoimmunotherapy. This clinical trial randomly assigns patients to receive either standard chemotherapy and surgery or chemoimmunotherapy (chemotherapy plus dinutuximab) and surgery during Induction therapy. Chemotherapy drugs administered during Induction include, cyclophosphamide, topotecan, cisplatin, etoposide, vincristine, and doxorubicin. These drugs work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing or by stopping them from spreading. Upon completion of 5 cycles of Induction therapy, a disease evaluation is completed to determine how well the treatment worked. If the tumor responds to therapy, patients receive a tandem transplantation with stem cell rescue. If the tumor has little improvement or worsens, patients receive chemoimmunotherapy on Extended Induction. During Extended Induction, dinutuximab is given with irinotecan, temozolomide. Patients with a good response to therapy move on to Consolidation therapy, when very high doses of chemotherapy are given at two separate points to kill any remaining cancer cells. Following, transplant, radiation therapy is given to the site where the cancer originated (primary site) and to any other areas that are still active at the end of Induction. The final stage of therapy is Post-Consolidation. During Post-Consolidation, dinutuximab is given with isotretinoin, with the goal of maintaining the response achieved with the previous therapy. Adding dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy may be better at treating children with newly diagnosed high-risk neuroblastoma.

The 2 groups in the trial

  • Arm A (SOC treatment)Existing treatment, for comparison

    See detailed description

    Treatment: Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Carboplatin, Cisplatin, Computed Tomography, Cyclophosphamide, Dinutuximab, Doxorubicin, Echocardiography Test, Etoposide, FDG-Positron Emission Tomography and Computed Tomography Scan, Hematopoietic Cell Transplantation, Irinotecan, Isotretinoin, Leukapheresis, Magnetic Resonance Imaging, Melphalan, Multigated Acquisition Scan, and Radiation Therapy

  • Arm B (Dinutuximab in induction)Treatment being tested

    See detailed description

    Treatment: Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Carboplatin, Cisplatin, Computed Tomography, Cyclophosphamide, Dinutuximab, Doxorubicin, Echocardiography Test, Etoposide, FDG-Positron Emission Tomography and Computed Tomography Scan, Hematopoietic Cell Transplantation, Irinotecan, Isotretinoin, Leukapheresis, Magnetic Resonance Imaging, Melphalan, Multigated Acquisition Scan, and Radiation Therapy

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma
Plain-language title
Adds dinutuximab to standard treatment for children with high-risk neuroblastomaGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Record updated
30 October 2026