New antibody combo shows promise in tough childhood cancer

NCT ID NCT01041638

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

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial tested the safety of giving a monoclonal antibody (dinutuximab) along with immune-boosting drugs and a vitamin-like drug to children with high-risk neuroblastoma after a stem cell transplant. The goal was to see if this combination could be given safely and help prevent the cancer from coming back. 105 children participated, and the study tracked serious side effects like pain, allergic reactions, and low blood pressure.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: dinutuximab (Ch14.18 antibody) plus sargramostim (GM-CSF), aldesleukin (IL-2), and isotretinoin
What this could lead to: If successful, this combination could become a standard post-transplant therapy to improve survival in children with high-risk neuroblastoma.
What could go wrong: This trial primarily measures safety, not effectiveness. Side effects like pain, low blood pressure, and allergic reactions are common. The long-term benefit is still uncertain.

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

105 people

The number who actually took part.

Started

Dec 2009

Finished

Jun 2021

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

Children (under 18), adults (18 to 64) and older adults (65 and over)

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 * At pre-ASCT evaluation, patients must meet the International Neuroblastoma Response Criteria (INRC) for complete response (CR), very good partial response (VGPR), or partial response (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; patients 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 an "overall" response of progressive disease \[PD\]) * Prior to enrollment on ANBL0931, a determination of residual disease must be performed (tumor imaging studies including metaiodobenzylguanidine \[MIBG\] scan, computed tomography \[CT\] or magnetic resonance imaging \[MRI\], bone marrow aspiration and biopsy); 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 * Patients with residual disease are eligible; biopsy is not required * Patients must not have progressive disease except for protocol specified bone marrow response * All patients must have completed therapy including intensive induction chemotherapy followed by ASCT and radiotherapy to be eligible; radiotherapy may be waived for patients who either had a small adrenal mass which was completely resected upfront, or who never had an identifiable primary tumor * No more than 9 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 9 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 * Use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age; required patients must have a Lansky or Karnofsky performance scale score of \>= 50% * Patients must have a life expectancy of \>= 2 months (8 weeks) * Total absolute phagocyte count (APC = neutrophils + monocytes) 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: * 1 month to \< 6 months: 0.4 mg/dL * 6 months to \< 1 year: 0.5 mg/dL * 1 to \< 2 years: 0.6 mg/dL * 2 to \< 6 years: 0.8 mg/dL * 6 to \< 10 years: 1 mg/dL * 10 to \< 13 years: 1.2 mg/dL * 13 to \< 16 years: 1.5 mg/dL (male), 1.4 mg/dL (female) * \>= 16 years: 1.7 mg/dL (male), 1.4 mg/dL (female) * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age * Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 5 x upper limit of normal (ULN) for age * SOS (sinusoidal obstruction syndrome, formerly known as veno-occlusive disease \[VOD\]), if present, should be stable or improving * Shortening fraction of \>= 27% by echocardiogram, or ejection fraction of \>= 55% by radionuclide angiography * No evidence of dyspnea at rest * If pulmonary function tests (PFTs) are performed, forced expiratory volume in one second (FEV1)/forced vital capacity (FVC) \> 60% by pulmonary function test * Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled * Central nervous system (CNS) toxicity \< grade 2 Exclusion Criteria: * 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 * Patients must not have received prior anti-GD2 antibody therapy * Patients must not have received prior vaccine therapy administered as treatment of neuroblastoma not routine infectious disease vaccinations

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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.

High-risk neuroblastoma

Contacts and locations

Locations

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
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 New Orleans

New Orleans, Louisiana, 70118, 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 Hospitals and Clinics of Minnesota - Minneapolis

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

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

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

Fort Worth, Texas, 76104, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
Emory University Hospital/Winship Cancer Institute

Atlanta, Georgia, 30322, United States
Indiana University/Melvin and Bren Simon Cancer Center

Indianapolis, Indiana, 46202, 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
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
New York Medical College

Valhalla, New York, 10595, United States
Rady Children's Hospital - San Diego

San Diego, California, 92123, United States
Riley Hospital for Children

Indianapolis, Indiana, 46202, United States
Saint Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
UCSF Medical Center-Mount Zion

San Francisco, California, 94115, United States
UCSF Medical Center-Parnassus

San Francisco, California, 94143, 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 Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States
University of Wisconsin Hospital and Clinics

Madison, Wisconsin, 53792, United States
Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States

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