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.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Ages | Any age to 30 Years |
| Sex | All |
| Enrollment | 478 (estimated) |
| Sponsor | National Cancer Institute (NCI) NIH |
| Drugs / interventions | chemotherapy, radiation, cyclophosphamide, dinutuximab, immunotherapy, doxorubicin |
| Locations | 180 sites (Birmingham, Alabama and 179 other locations) |
| Status | Recruiting |
| Started | April 19, 2024 |
| Main results expected | December 31, 2029 |
| Study ends | December 31, 2029 |
| Registry record updated | October 30, 2026 |
| Trial ID | NCT06172296 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
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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
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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.
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rhIL-15 with dinutuximab plus irinotecan and temozolomide for relapsed or refractory neuroblastoma in children and young adults
This trial will test whether adding rhIL-15 to dinutuximab with irinotecan and temozolomide helps children and young adults (ages 3-35) whose neuroblastoma has returned or not responded to prior treatments.
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Dinutuximab beta combined with two induction chemotherapy regimens for newly diagnosed high-risk neuroblastoma
This will test different doses of dinutuximab beta given with two standard induction chemotherapy regimens in children (18 months to under 18 years) with newly diagnosed high‑risk neuroblastoma to find a safe recommended dose.
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STING: sequential temozolomide, irinotecan, donor NK cells, and dinutuximab for neuroblastoma
This will test whether adding donor TGF‑beta‑imprinted NK cells to dinutuximab plus temozolomide, irinotecan, and GM‑CSF helps children and young adults with relapsed or refractory high‑risk neuroblastoma.
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Using expanded gamma delta T cells with chemotherapy for treating relapsed neuroblastoma and osteosarcoma
This study is testing if a new treatment using special immune cells along with chemotherapy can help children with relapsed neuroblastoma and osteosarcoma feel better and improve their chances of recovery.
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Adding iobenguane I-131 or lorlatinib to intensive treatment for children and young adults with high-risk neuroblastoma
This trial tests whether adding the radioactive drug iobenguane I-131 or the targeted drug lorlatinib to standard intensive therapy helps children and young adults with newly diagnosed high-risk neuroblastoma or nodular ganglioneuroblastoma stay free of disease longer.
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Neuroblastoma patients' genomic and biological profile study
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.