Immunotherapy combo shows promise in tough childhood cancer

NCT ID NCT00026312

Disease control Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

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

Summary

This study tested whether adding an immunotherapy drug (dinutuximab) and immune-boosting medicines to standard treatment helps children with high-risk neuroblastoma stay cancer-free longer. About 1,400 children who had already received intensive chemotherapy and a stem cell transplant took part. The goal was to see if the combination therapy improves event-free survival compared to standard treatment alone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

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

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

1,449 people

The number who actually took part.

Started

Oct 2001

Finished

Dec 2025

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

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: * All patients must be diagnosed with neuroblastoma, and categorized as high risk at the time of diagnosis; exception: patients who are initially diagnosed as non-high-risk neuroblastoma, but later converted (and/or relapsed) to high risk neuroblastoma are also eligible * All patients must have completed therapy including intensive induction followed by ASCT and radiotherapy to be eligible for ANBL0032; radiotherapy may be waived for patients who either have small adrenal masses which are completely resected up front, or who never have an identifiable primary tumor; examples of such therapies include: * Following treatment per A3973 protocol * Following treatment per Pediatric Oncology Group (POG)-9341/9342 protocol * Following treatment per CCG3891 * Following treatment on New Approaches to Neuroblastoma Therapy (NANT) 2001-02 * Enrollment on or following treatment per ANBL02P1 * Enrollment on or following treatment per ANBL07P1 * Tandem transplant patients are eligible: * Following treatment on or per ANBL0532 * Following treatment per POG 9640 * Following treatment per COG ANBL00P1 * Following treatment per CHP 594/Dana-Farber Cancer Institute (DFCI) 34-DAT * No more than 12 months from the date of starting the first induction chemotherapy after diagnosis to the date of ASCT except for the rare occasions as noted below; for tandem ASCT patients, this will be the date of the FIRST stem cell infusion; exception: for those who are initially diagnosed as non-high risk neuroblastoma, but later converted (and/or relapsed) to high risk neuroblastoma, the 12 months restriction should start from the date of induction therapy for high risk neuroblastoma (not from the initial induction therapy for non-high risk disease), to the date of ASCT * At pre-ASCT evaluation patients must meet the International Neuroblastoma Response Criteria (INRC) for CR, VGPR, or PR for primary site, soft tissue metastases and bone metastases; patients who meet those criteria must also meet the protocol specified criteria for bone marrow response as outlined below: * =\< 10% tumor (of total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy * Patient who have no tumor seen on the prior bone marrow, and then have =\< 10% tumor on any of the bilateral marrow aspirate/biopsy specimens done at pre-ASCT and/or pre-enrollment evaluation will also be eligible (note that per INRC this would have been defined as "overall" response of progressive disease \[PD\]) * Prior to enrollment on ANBL0032, a determination of mandatory disease staging must be performed (tumor imaging studies including computed tomography \[CT\] or magnetic resonance imaging \[MRI\], MIBG scan, and vanillylmandelic acid \[VMA\]/homovanillic acid \[HVA\]; bone marrow aspirates are required but biopsy may be omitted if negative prior to ASCT); this disease assessment is required for eligibility and should be done preferably within 2 weeks, but must be done within a maximum of 4 weeks before enrollment * For those with residual disease before radiotherapy, re-evaluation of irradiated residual tumors is preferably performed at the earliest 5 days after completing radiotherapy; patients with residual disease are eligible; biopsy is not required; patients who have biopsy proven residual disease after ASCT will be enrolled on Stratum 07 * Patients must not have progressive disease at the time of study enrollment except for protocol specified bone marrow response and except for elevations of catecholamines as the only sign of disease in a patient who had normal catecholamines at pre-ASCT evaluation * Patients must be enrolled before treatment begins; the date protocol therapy is projected to start must be no later than ten (10) calendar days after the date of study enrollment; patients should be enrolled preferably between day 56 and day 85 after peripheral blood stem cell (PBSC) infusion (day from 2nd stem cell infusion for tandem transplant); patients must be enrolled no later than day 200 after PBSC infusion; enrollment must occur after completion of radiotherapy, and after completion of tumor assessment post-ASCT and radiotherapy; informed consent should be obtained within 3 weeks pre-ASCT up to the time of registration * Patients must not have received prior anti-disialoganglioside (GD2) antibody therapy * Patients must have a Lansky or Karnofsky performance scale score of \>= 50% and patients must have a life expectancy of \>= 2 months * Total absolute phagocyte count (APC = %neutrophils + %monocytes) X white blood cell (WBC) is at least 1000/uL * 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: * No greater than 0.4 mg/dL (1 month to \< 6 months) * No greater than 0.5 mg/dL (6 months to \< 1 year) * No greater than 0.6 mg/dL (1 to \< 2 years) * No greater than 0.8 mg/dL (2 to \< 6 years) * No greater than 1.0 mg/dL (6 to \< 10 years) * No greater than 1.2 mg/dL (10 to \< 13 years) * No greater than 1.4 mg/dL (\>= 13 years \[female\]) * No greater than 1.5 mg/dL (13 to \< 16 years \[male\]) * No greater than 1.7 mg/dL (\>= 16 years \[male\]) * Total bilirubin =\< 1.5 x normal * Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 5 x normal * Veno-occlusive disease, if present, should be stable or improving * Shortening fraction of \>= 27% by echocardiogram, or if shortening fraction abnormal, ejection fraction of \>= 55% by gated radionuclide study or echocardiogram; note: the echocardiogram or gated radionuclide study must be performed within 4 weeks prior to enrollment * Forced expiratory volume in one second (FEV1) and forced vital capacity (FVC) \> 60% predicted by pulmonary function test; for children who are unable to do pulmonary function tests (PFTs), no evidence of dyspnea at rest and no exercise intolerance should be documented; note: the pulmonary function test must be performed within 4 weeks prior to enrollment * Patients with seizure disorder may be enrolled if on anticonvulsants and well-controlled; central nervous system (CNS) toxicity \< grade 2 * Written informed consent in accordance with institutional and Food and Drug Administration (FDA) guidelines must be obtained from parent or legal guardian * Females of childbearing potential must have a negative pregnancy test; patients of childbearing potential must agree to use an effective birth control method; female patients who are lactating must agree to stop breast-feeding

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Conditions

The condition(s) this trial relates to.

neuroblastoma

As listed by the trial registrant

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

Localized resectable neuroblastoma Localized unresectable neuroblastoma Recurrent neuroblastoma Regional neuroblastoma Stage 4 neuroblastoma Stage 4S neuroblastoma

Contacts and locations

Locations

AdventHealth Orlando

Orlando, Florida, 32803, United States
Advocate Children's Hospital-Oak Lawn

Oak Lawn, Illinois, 60453, United States
Albany Medical Center

Albany, New York, 12208, United States
Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada
Alfred I duPont Hospital for Children

Wilmington, Delaware, 19803, United States
Alliance for Childhood Diseases/Cure 4 the Kids Foundation

Las Vegas, Nevada, 89135, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202-3591, United States
Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States
Ascension Saint Vincent Indianapolis Hospital

Indianapolis, Indiana, 46260, United States
Augusta University Medical Center

Augusta, Georgia, 30912, United States
BI-LO Charities Children's Cancer Center

Greenville, South Carolina, 29605, United States
Banner University Medical Center - Tucson

Tucson, Arizona, 85719, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, 77030, United States
Baystate Medical Center

Springfield, Massachusetts, 01199, United States
Blank Children's Hospital

Des Moines, Iowa, 50309, United States
British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada
Bronson Methodist Hospital

Kalamazoo, Michigan, 49007, United States
Broward Health Medical Center

Fort Lauderdale, Florida, 33316, United States
C S Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States
CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)

Québec, G1V 4G2, Canada
CancerCare Manitoba

Winnipeg, Manitoba, R3E 0V9, Canada
Cardinal Glennon Children's Medical Center

St Louis, Missouri, 63104, United States
Carilion Children's

Roanoke, Virginia, 24014, United States
Carolinas Medical Center/Levine Cancer Institute

Charlotte, North Carolina, 28203, United States
Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada
Children's Healthcare of Atlanta - Arthur M Blank Hospital

Atlanta, Georgia, 30329, United States
Children's Hospital

London, Ontario, N6A 5W9, Canada
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 New Orleans

New Orleans, Louisiana, 70118, United States
Children's Hospital and Medical Center of Omaha

Omaha, Nebraska, 68114, United States
Children's Hospital of Alabama

Birmingham, Alabama, 35233, United States
Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada
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 The King's Daughters

Norfolk, Virginia, 23507, United States
Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, 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
City of Hope Comprehensive Cancer Center

Duarte, California, 91010, United States
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Connecticut Children's Medical Center

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

Fort Worth, Texas, 76104, United States
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center

Lebanon, New Hampshire, 03756, United States
Dayton Children's Hospital

Dayton, Ohio, 45404, United States
Dell Children's Medical Center of Central Texas

Austin, Texas, 78723, United States
Driscoll Children's Hospital

Corpus Christi, Texas, 78411, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
East Tennessee Childrens Hospital

Knoxville, Tennessee, 37916, United States
Geisinger Medical Center

Danville, Pennsylvania, 17822, United States
Golisano Children's Hospital of Southwest Florida

Fort Myers, Florida, 33908, United States
Greenville Cancer Treatment Center

Greenville, South Carolina, 29605, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Henry Ford Health Saint John Hospital

Detroit, Michigan, 48236, United States
Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada
Hurley Medical Center

Flint, Michigan, 48503, United States
IWK Health Centre

Halifax, Nova Scotia, B3K 6R8, Canada
Inova Fairfax Hospital

Falls Church, Virginia, 22042, United States
Janeway Child Health Centre

St. John's, Newfoundland and Labrador, A1B 3V6, Canada
John Hunter Children's Hospital

Hunter Regional Mail Centre, New South Wales, 2310, Australia
Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States
Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, 21287, United States
Kaiser Permanente Downey Medical Center

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

Oakland, California, 94611, United States
Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96826, United States
Laura and Isaac Perlmutter Cancer Center at NYU Langone

New York, New York, 10016, United States
Lee Memorial Health System

Fort Myers, Florida, 33901, United States
Legacy Emanuel Children's Hospital

Portland, Oregon, 97227, United States
Legacy Emanuel Hospital and Health Center

Portland, Oregon, 97227, United States
Lehigh Valley Hospital - Muhlenberg

Bethlehem, Pennsylvania, 18017, United States
Loma Linda University Medical Center

Loma Linda, California, 92354, United States
Lucile Packard Children's Hospital Stanford University

Palo Alto, California, 94304, United States
Lurie Children's Hospital-Chicago

Chicago, Illinois, 60611, United States
Maine Children's Cancer Program

Scarborough, Maine, 04074, United States
Marshfield Medical Center-Marshfield

Marshfield, Wisconsin, 54449, United States
Massachusetts General Hospital Cancer Center

Boston, Massachusetts, 02114, United States
Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States
McMaster Children's Hospital at Hamilton Health Sciences

Hamilton, Ontario, L8N 3Z5, Canada
MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States
Medical City Dallas Hospital

Dallas, Texas, 75230, United States
Medical University of South Carolina

Charleston, South Carolina, 29425, United States
Memorial Health University Medical Center

Savannah, Georgia, 31404, United States
Memorial Regional Hospital/Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States
Mercy Children's Hospital

Toledo, Ohio, 43608, 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
Miami Cancer Institute

Miami, Florida, 33176, United States
Michigan State University

East Lansing, Michigan, 48823, United States
Miller Children's and Women's Hospital Long Beach

Long Beach, California, 90806, United States
Mission Hospital

Asheville, North Carolina, 28801, United States
Montefiore Medical Center - Moses Campus

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

Morristown, New Jersey, 07960, United States
Mount Sinai Hospital

New York, New York, 10029, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
NYU Langone Hospital - Long Island

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

Columbus, Ohio, 43205, United States
Naval Medical Center - Portsmouth

Portsmouth, Virginia, 23708-2197, United States
Nemours Children's Clinic - Orlando

Orlando, Florida, 32806, United States
Nemours Children's Clinic - Pensacola

Pensacola, Florida, 32504, United States
Nemours Children's Clinic-Jacksonville

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

Orlando, Florida, 32827, United States
Nevada Cancer Research Foundation NCORP

Las Vegas, Nevada, 89120, United States
New York Medical College

Valhalla, New York, 10595, United States
Newark Beth Israel Medical Center

Newark, New Jersey, 07112, United States
Nicklaus Children's Hospital

Miami, Florida, 33155, United States
Norton Children's Hospital

Louisville, Kentucky, 40202, United States
Novant Health Presbyterian Medical Center

Charlotte, North Carolina, 28204, United States
Oregon Health and Science University

Portland, Oregon, 97239, United States
Orlando Health Cancer Institute

Orlando, Florida, 32806, United States
Overlook Hospital

Summit, New Jersey, 07902, United States
Penn State Children's Hospital

Hershey, Pennsylvania, 17033, United States
Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States
Primary Children's Hospital

Salt Lake City, Utah, 84113, United States
Princess Margaret Hospital for Children

Perth, Western Australia, 6008, Australia
Prisma Health Richland Hospital

Columbia, South Carolina, 29203, United States
ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital

Toledo, Ohio, 43606, United States
Providence Alaska Medical Center

Anchorage, Alaska, 99508, United States
Providence Sacred Heart Medical Center and Children's Hospital

Spokane, Washington, 99204, United States
Queensland Children's Hospital

South Brisbane, Queensland, 4101, Australia
Rady Children's Hospital - San Diego

San Diego, California, 92123, United States
Rainbow Babies and Childrens Hospital

Cleveland, Ohio, 44106, United States
Rhode Island Hospital

Providence, Rhode Island, 02903, 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
Roswell Park Cancer Institute

Buffalo, New York, 14263, United States
Royal Brisbane and Women's Hospital

Herston, Queensland, 4029, Australia
Royal Children's Hospital

Parkville, Victoria, 3052, Australia
Royal Children's Hospital-Brisbane

Herston, Queensland, 4029, Australia
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital

New Brunswick, New Jersey, 08903, United States
Saint Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States
Saint Joseph's Hospital/Children's Hospital-Tampa

Tampa, Florida, 33607, United States
Saint Jude Children's Research Hospital

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

Peoria, Illinois, 61637, United States
Saint Mary's Medical Center

West Palm Beach, Florida, 33407, United States
Saint Peter's University Hospital

New Brunswick, New Jersey, 08901, United States
Saint Vincent Hospital Cancer Center Green Bay

Green Bay, Wisconsin, 54301, United States
San Jorge Children's Hospital

San Juan, 00912, Puerto Rico
Sanford Broadway Medical Center

Fargo, North Dakota, 58122, United States
Sanford USD Medical Center - Sioux Falls

Sioux Falls, South Dakota, 57117-5134, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Sinai Hospital of Baltimore

Baltimore, Maryland, 21215, United States
Southern Illinois University School of Medicine

Springfield, Illinois, 62702, 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
Sutter Medical Center Sacramento

Sacramento, California, 95816, United States
Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia
T C Thompson Children's Hospital

Chattanooga, Tennessee, 37403, United States
The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia
The Montreal Children's Hospital of the MUHC

Montreal, Quebec, H3H 1P3, Canada
The Steven and Alexandra Cohen Children's Medical Center of New York

New Hyde Park, New York, 11040, United States
Tripler Army Medical Center

Honolulu, Hawaii, 96859, United States
Tufts Children's Hospital

Boston, Massachusetts, 02111, United States
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States
UCSF Medical Center-Mission Bay

San Francisco, California, 94158, United States
UCSF Medical Center-Parnassus

San Francisco, California, 94143, United States
UF Health Cancer Institute - Gainesville

Gainesville, Florida, 32610, United States
UNC Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, 27599, United States
UT Southwestern/Simmons Cancer Center-Dallas

Dallas, Texas, 75390, United States
University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States
University of Alberta Hospital

Edmonton, Alberta, T6G 2B7, Canada
University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States
University of California Davis Comprehensive Cancer Center

Sacramento, California, 95817, United States
University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637, United States
University of Hawaii Cancer Center

Honolulu, Hawaii, 96813, 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 Kentucky/Markey Cancer Center

Lexington, Kentucky, 40536, United States
University of Maryland/Greenebaum Cancer Center

Baltimore, Maryland, 21201, United States
University of Miami Miller School of Medicine-Sylvester Cancer Center

Miami, Florida, 33136, United States
University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States
University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States
University of Missouri Children's Hospital

Columbia, Missouri, 65212, United States
University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States
University of New Mexico Cancer Center

Albuquerque, New Mexico, 87106, United States
University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States
University of Rochester

Rochester, New York, 14642, United States
University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States
University of Vermont and State Agricultural College

Burlington, Vermont, 05405, United States
University of Virginia Cancer Center

Charlottesville, Virginia, 22908, United States
University of Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, 53792, United States
VCU Massey Comprehensive Cancer Center

Richmond, Virginia, 23298, United States
Valley Children's Hospital

Madera, California, 93636, United States
Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States
Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, 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
Wayne State University/Karmanos Cancer Institute

Detroit, Michigan, 48201, United States
West Virginia University Healthcare

Morgantown, West Virginia, 26506, United States
Women's and Children's Hospital-Adelaide

North Adelaide, South Australia, 5006, Australia
Yale University

New Haven, Connecticut, 06520, United States

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