New combo therapy aims to keep high-risk neuroblastoma from coming back

NCT ID NCT04385277

Disease control Sponsor: Children's Oncology Group Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether adding two chemotherapy drugs (irinotecan and temozolomide) to the standard immunotherapy regimen (dinutuximab, GM-CSF, isotretinoin) can safely prevent high-risk neuroblastoma from returning after intensive initial therapy. About 41 children and young adults under 31 who have finished their first round of treatment without their cancer getting worse will receive up to 5 cycles of this combined chemo-immunotherapy. The main goal is to see if patients can complete all cycles without the disease progressing.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

41 people

The number who actually took part.

Started

Dec 2020

Expected to finish

Sep 2027

An estimate. End dates often move.

Lead sponsor

A research network

The lead sponsor is a research network or cooperative group.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 30 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients must be \< 31 years of age at the time of enrollment. * 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 at the time of diagnosis) and have been designated as having high-risk disease based on Children's Oncology Group (COG) risk classification. The following disease groups are eligible: * Patients with International Neuroblastoma Risk Group (INRG) Stage M disease with any of the following features: * MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR * Age \> 547 days at the time of diagnosis regardless of biologic features; OR * Age 365-547 days at the time of diagnosis with tumors with unfavorable histology and/or deoxyribonucleic acid (DNA) index = 1 * Patients with INRG Stage MS disease with MYCN amplification * Patients with INRG Stage L2 disease with either of the following features: * MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR * Age \> 547 days at the time of diagnosis with MYCN non-amplified tumors with unfavorable histology * Note: Patients observed or patients 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 criteria will also be eligible * Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years * Prior therapy * All patients must have completed high-risk Induction therapy with 4-6 cycles of chemotherapy * After completion of Induction therapy, patients may have received no more than 4 cycles of bridging chemotherapy or chemo-immunotherapy prior to ASCT * Patients cannot have previously progressed on immunotherapy with dinutuximab or other anti-GD2 monoclonal antibody * All patients must have had undergone surgical resection of their primary tumor as part of frontline therapy. Exceptions to this requirement include patients who had a complete response to Induction chemotherapy, patients with no identifiable primary tumor, and patients for whom the institutional surgical team determined that potential risks outweighed potential benefits of resection * All patients must have undergone tandem high-dose chemotherapy with ASCT as part of Consolidation * Patients must enroll between day +56 and day +200 from the peripheral blood stem cell (PBSC) infusion following the last dose of high-dose chemotherapy during Consolidation * All patients must have undergone external beam radiation therapy. Exceptions to this requirement include patients who had no identifiable primary tumor and no persistent metastatic disease at the end of Induction. For patients who received radiotherapy, at least 7 days must have elapsed between completion of radiotherapy and enrollment on this study * Patients must not have received long-acting myeloid growth factors (e.g., Neulasta) within 14 days of entry on this study. Seven days must have elapsed since administration of a short-acting myeloid growth factor * Peripheral absolute neutrophil count (ANC) \>= 750/uL * Platelet count \>= 50,000/uL (transfusion independent for \>= 7 days) * Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2, or a serum creatinine based on age/gender as follows: * Age: Maximum Serum Creatinine (mg/dL) * 6 months to \< 1 year: 0.5 (male and female) * 1 to \< 2 years: 0.6 (male and female) * 2 to \< 6 years: 0.8 (male and female) * 6 to \< 10 years: 1 (male and female) * 10 to \< 13 years: 1.2 (male and female) * 13 to \< 16 years: 1.5 (male), 1.4 (female) * \>= 16 years: 1.7 (male), 1.4 (female) * Note: Patients with history of transplant associated-thrombotic microangiopathy (TA-TMA) must have a creatinine clearance or radioisotope GFR at baseline to assess renal function and must meet the above criteria * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age * Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 5.0 x ULN for age (=\< 225 U/L) * 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 * Absence of dyspnea at rest * If pulmonary function tests (PFTs) are performed, forced expiratory volume in 1 second (FEV1)/forced vital capacity (FVC) must be \> 60% * No clinical evidence of active central nervous system (CNS) disease at the time of study enrollment * Patients with seizure disorder may be enrolled if on non-enzyme-inducing anticonvulsants and well controlled * CNS toxicity from prior therapy =\< grade 2 Exclusion Criteria: * Patients must not have had progressive disease (PD) per the revised International Neuroblastoma Risk Criteria (INRC) since the initial diagnosis of high-risk neuroblastoma * Exception: Progressive disease within the first 2 cycles of Induction chemotherapy consisting of cyclophosphamide and topotecan is allowed. Patients with progression subsequent to initial cyclophosphamide and topotecan cycles are excluded * Patients may not have received additional systemic cancer-directed therapy following completion of the last planned high-dose chemotherapy with ASCT prior to enrollment on this trial * Patients may not have received iodine-131 (131I)-metaiodobenzylguanidine (MIBG) therapy at any time prior to enrollment on this trial * Patients who received single (rather than tandem) high-dose chemotherapy with ASCT are excluded * Patients cannot be receiving other ongoing anticancer therapy * Patients who were enrolled onto ANBL1531 AND underwent arm assignment are not eligible. Patients who enrolled onto ANBL1531 who declined second consent may be eligible for ANBL19P1 if all other criteria are met * Patients enrolled onto ANBL17P1 are not eligible * Patients must have been off pharmacologic doses of systemic steroids for at least 7 days prior to enrollment * Patients who require or are likely to require pharmacologic doses of systemic corticosteroids while receiving treatment on this study are ineligible. The only exception is for patients known to require 2 mg/kg or less of hydrocortisone (or an equivalent dose of an alternative corticosteroid) as premedication for blood product administration in order to avoid allergic transfusion reactions * Note: The use of conventional doses of inhaled steroids for the treatment of asthma is permitted, as is the use of physiologic doses of steroids for patients with known adrenal insufficiency * Patients on any other immunosuppressive medications (e.g., cyclosporine, tacrolimus) are not eligible. However, prior or planned concomitant treatment with eculizumab is permitted (e.g., treatment of TA-TMA) * Patients must not have received enzyme-inducing anticonvulsants including phenytoin, phenobarbital, valproic acid, or carbamazepine for at least 7 days prior to study enrollment * Note: Patients receiving non-enzyme inducing anticonvulsants such as gabapentin or levetiracetam are eligible * Patients must not have received drugs that are strong inducers or inhibitors of CYP3A4 within 7 days prior to study enrollment * Patients must not have been diagnosed with myelodysplastic syndrome or with any malignancy other than neuroblastoma * Patients with symptoms of congestive heart failure are not eligible * Patients with moderate or large pericardial effusions are not eligible * Patients must not have \>= grade 2 diarrhea * Patients must not have uncontrolled infection * Patients with a history of grade 4 allergic reactions to anti-GD2 antibodies or reactions that required discontinuation of the anti-GD2 therapy are not eligible * Patients with a significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of study agents or to significantly increase the severity of the toxicities experienced from study treatment are not eligible * 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 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

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Conditions

The condition(s) this trial relates to.

ganglioneuroblastoma neuroblastoma nodular ganglioneuroblastoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Ganglioneuroblastoma, nodular Neuroblastoma

Contacts and locations

Locations

Albany Medical Center

Albany, New York, 12208, United States
Alfred I duPont Hospital for Children

Wilmington, Delaware, 19803, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202-3591, United States
Banner Children's at Desert

Mesa, Arizona, 85202, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, 77030, United States
C S Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States
Carolinas Medical Center/Levine Cancer Institute

Charlotte, North Carolina, 28203, United States
Children's Healthcare of Atlanta - Egleston

Atlanta, Georgia, 30322, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital Medical Center of Akron

Akron, Ohio, 44308, United States
Children's Hospital and Medical Center of Omaha

Omaha, Nebraska, 68114, United States
Children's Hospital of Orange County

Orange, California, 92868, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States
Children's Hospital of San Antonio

San Antonio, Texas, 78207, United States
Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States
Children's Hospitals and Clinics of Minnesota - Minneapolis

Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Christchurch Hospital

Christchurch, 8011, New Zealand
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States
Cook Children's Medical Center

Fort Worth, Texas, 76104, United States
Covenant Children's Hospital

Lubbock, Texas, 79410, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Dell Children's Medical Center of Central Texas

Austin, Texas, 78723, United States
El Paso Children's Hospital

El Paso, Texas, 79905, United States
Golisano Children's Hospital of Southwest Florida

Fort Myers, Florida, 33908, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
IWK Health Centre

Halifax, Nova Scotia, B3K 6R8, Canada
Kaiser Permanente Downey Medical Center

Downey, California, 90242, United States
Kaiser Permanente-Oakland

Oakland, California, 94611, United States
Lurie Children's Hospital-Chicago

Chicago, Illinois, 60611, United States
Medical City Dallas Hospital

Dallas, Texas, 75230, United States
Memorial Regional Hospital/Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States
Mercy Hospital Saint Louis

St Louis, Missouri, 63141, United States
Methodist Children's Hospital of South Texas

San Antonio, Texas, 78229, United States
Montefiore Medical Center - Moses Campus

The Bronx, New York, 10467, United States
Morristown Medical Center

Morristown, New Jersey, 07960, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
NYU Winthrop Hospital

Mineola, New York, 11501, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Nemours Children's Clinic-Jacksonville

Jacksonville, Florida, 32207, United States
Nemours Children's Hospital

Orlando, Florida, 32827, United States
Norton Children's Hospital

Louisville, Kentucky, 40202, United States
Ochsner Medical Center Jefferson

New Orleans, Louisiana, 70121, United States
Perth Children's Hospital

Perth, Western Australia, 6009, Australia
Primary Children's Hospital

Salt Lake City, Utah, 84113, United States
Queensland Children's Hospital

South Brisbane, Queensland, 4101, Australia
Rainbow Babies and Childrens Hospital

Cleveland, Ohio, 44106, United States
Riley Hospital for Children

Indianapolis, Indiana, 46202, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center

Denver, Colorado, 80218, United States
Royal Children's Hospital

Parkville, Victoria, 3052, Australia
Saint Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States
Saint Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Saint Jude Midwest Affiliate

Peoria, Illinois, 61637, United States
Saint Luke's Cancer Institute - Boise

Boise, Idaho, 83712, United States
Sanford Broadway Medical Center

Fargo, North Dakota, 58122, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Starship Children's Hospital

Grafton, Auckland, 1145, New Zealand
State University of New York Upstate Medical University

Syracuse, New York, 13210, United States
The Children's Hospital at TriStar Centennial

Nashville, Tennessee, 37203, United States
The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia
UCSF Medical Center-Mission Bay

San Francisco, California, 94158, United States
UMC Cancer Center / UMC Health System

Lubbock, Texas, 79415, United States
UT Southwestern/Simmons Cancer Center-Dallas

Dallas, Texas, 75390, United States
University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637, United States
University of Florida Health Science Center - Gainesville

Gainesville, Florida, 32610, United States
University of Illinois

Chicago, Illinois, 60612, United States
University of Iowa/Holden Comprehensive Cancer Center

Iowa City, Iowa, 52242, United States
University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States
University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States
University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States
Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States
Walter Reed National Military Medical Center

Bethesda, Maryland, 20889-5600, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States

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