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Combining dinutuximab with chemotherapy and surgery for treating high-risk neuroblastoma in children

A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma

Phase 3 Interventional National Cancer Institute (NCI) · NCT06172296

This study is testing if adding a new medicine called dinutuximab to regular chemotherapy and surgery can help children with high-risk neuroblastoma live longer without their cancer coming back.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment478 (estimated)
AgesN/A to 30 Years
SexAll
SponsorNational Cancer Institute (NCI) NIH
Drugs / interventionschemotherapy, radiation, cyclophosphamide, dinutuximab, immunotherapy, doxorubicin
Locations177 sites (Birmingham, Alabama and 176 other locations)
Trial IDNCT06172296 on ClinicalTrials.gov

What this trial studies

This phase III trial evaluates the effectiveness of adding dinutuximab, a monoclonal antibody, to standard chemotherapy and surgical procedures for children diagnosed with high-risk neuroblastoma. The study aims to determine if this combination improves event-free survival compared to standard treatment alone. Participants will be randomly assigned to receive either standard chemotherapy or the combination of chemotherapy and dinutuximab during the induction phase of treatment. The trial also assesses the safety and response rates associated with this new treatment approach.

Who should consider this trial

Good fit: Ideal candidates are children aged 30 years or younger with newly diagnosed high-risk neuroblastoma or ganglioneuroblastoma.

Not a fit: Patients with low-risk neuroblastoma or those who do not meet the specific diagnostic criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve survival rates and outcomes for children with high-risk neuroblastoma.

How similar studies have performed: Previous studies have shown promising results with similar immunotherapy approaches in treating neuroblastoma, indicating potential for success in this trial.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* 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

Exclusion Criteria:

* 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
* 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
* 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
* 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
* All institutional, food and drug administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Where this trial is running

Birmingham, Alabama and 176 other locations

+127 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Ganglioneuroblastoma, NodularNeuroblastoma
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.