Clinical trial Recruiting at 177 sites Phase 3 Interventional Ages Any age to 30

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

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

Run by National Cancer Institute (NCI) · NCT06172296 · Data updated 2026-11-12

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.

More Neuroblastoma trials

Quick facts

PhasePhase 3
Study typeInterventional
AgesAny age to 30 Years
SexAll
Enrollment478 (estimated)
SponsorNational Cancer Institute (NCI)
NIH
Drugs / interventionschemotherapy, radiation, cyclophosphamide, dinutuximab, immunotherapy, doxorubicin
Locations180 sites (Birmingham, Alabama and 179 other locations)
StatusRecruiting
StartedApril 19, 2024
Main results expectedDecember 31, 2029
Study endsDecember 31, 2029
Registry record updatedOctober 30, 2026
Trial IDNCT06172296 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

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

Probably not a fit

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

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

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

Who can join: the full rules

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 it's running

  • Children's Hospital of Alabama
    Birmingham, Alabama, United States
    Recruiting
  • USA Health Strada Patient Care Center
    Mobile, Alabama, United States
    Recruiting
  • Banner Children's at Desert
    Mesa, Arizona, United States
    Recruiting
  • Phoenix Childrens Hospital
    Phoenix, Arizona, United States
    Recruiting
  • Banner University Medical Center - Tucson
    Tucson, Arizona, United States
    Recruiting
  • Arkansas Children's Hospital
    Little Rock, Arkansas, United States
    Recruiting
Show 44 more locations
  • Kaiser Permanente Downey Medical Center
    Downey, California, United States
    Recruiting
  • City of Hope Comprehensive Cancer Center
    Duarte, California, United States
    Recruiting
  • Loma Linda University Medical Center
    Loma Linda, California, United States
    Recruiting
  • Miller Children's and Women's Hospital Long Beach
    Long Beach, California, United States
    Recruiting
  • Children's Hospital Los Angeles
    Los Angeles, California, United States
    Recruiting
  • Cedars-Sinai Medical Center
    Los Angeles, California, United States
    Recruiting
  • Mattel Children's Hospital UCLA
    Los Angeles, California, United States
    Recruiting
  • Valley Children's Hospital
    Madera, California, United States
    Recruiting
  • UCSF Benioff Children's Hospital Oakland
    Oakland, California, United States
    Recruiting
  • Kaiser Permanente-Oakland
    Oakland, California, United States
    Recruiting
  • Children's Hospital of Orange County
    Orange, California, United States
    Recruiting
  • Lucile Packard Children's Hospital Stanford University
    Palo Alto, California, United States
    Recruiting
  • Sutter Medical Center Sacramento
    Sacramento, California, United States
    Recruiting
  • University of California Davis Comprehensive Cancer Center
    Sacramento, California, United States
    Recruiting
  • Rady Children's Hospital - San Diego
    San Diego, California, United States
    Recruiting
  • UCSF Medical Center-Mission Bay
    San Francisco, California, United States
    Recruiting
  • Children's Hospital Colorado
    Aurora, Colorado, United States
    Recruiting
  • Connecticut Children's Medical Center
    Hartford, Connecticut, United States
    Recruiting
  • Yale University
    New Haven, Connecticut, United States
    Recruiting
  • Alfred I duPont Hospital for Children
    Wilmington, Delaware, United States
    Recruiting
  • Children's National Medical Center
    Washington D.C., District of Columbia, United States
    Recruiting
  • Broward Health Medical Center
    Fort Lauderdale, Florida, United States
    Recruiting
  • Golisano Children's Hospital of Southwest Florida
    Fort Myers, Florida, United States
    Recruiting
  • UF Health Cancer Institute - Gainesville
    Gainesville, Florida, United States
    Recruiting
  • Memorial Regional Hospital/Joe DiMaggio Children's Hospital
    Hollywood, Florida, United States
    Recruiting
  • Nemours Children's Clinic-Jacksonville
    Jacksonville, Florida, United States
    Recruiting
  • University of Miami Miller School of Medicine-Sylvester Cancer Center
    Miami, Florida, United States
    Recruiting
  • Nicklaus Children's Hospital
    Miami, Florida, United States
    Recruiting
  • AdventHealth Orlando
    Orlando, Florida, United States
    Recruiting
  • Arnold Palmer Hospital for Children
    Orlando, Florida, United States
    Recruiting
  • Nemours Children's Hospital
    Orlando, Florida, United States
    Recruiting
  • Johns Hopkins All Children's Hospital
    St. Petersburg, Florida, United States
    Recruiting
  • Saint Mary's Medical Center
    West Palm Beach, Florida, United States
    Recruiting
  • Children's Healthcare of Atlanta - Arthur M Blank Hospital
    Atlanta, Georgia, United States
    Recruiting
  • Memorial Health University Medical Center
    Savannah, Georgia, United States
    Recruiting
  • Kapiolani Medical Center for Women and Children
    Honolulu, Hawaii, United States
    Recruiting
  • Lurie Children's Hospital-Chicago
    Chicago, Illinois, United States
    Recruiting
  • University of Illinois
    Chicago, Illinois, United States
    Recruiting
  • University of Chicago Comprehensive Cancer Center
    Chicago, Illinois, United States
    Recruiting
  • Advocate Children's Hospital-Oak Lawn
    Oak Lawn, Illinois, United States
    Recruiting
  • Advocate Children's Hospital-Park Ridge
    Park Ridge, Illinois, United States
    Recruiting
  • OSF Children's Hospital of Illinois
    Peoria, Illinois, United States
    Recruiting
  • Southern Illinois University School of Medicine
    Springfield, Illinois, United States
    Recruiting
  • Riley Hospital for Children
    Indianapolis, Indiana, United States
    Recruiting

+130 more sites. See ClinicalTrials.gov for the full list.

Study contacts

  • Principal investigator: Sara M Federico — Children's Oncology Group

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 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.