New combo therapy shows promise for tough childhood cancer
NCT ID NCT01175356
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a very strong treatment plan for children newly diagnosed with high-risk neuroblastoma, a serious nerve cell cancer. The approach combined intensive chemotherapy, a radioactive drug called 131I-MIBG, and a stem cell transplant to try to kill more cancer cells. The goal was to see if this intense regimen was feasible and safe for patients.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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99 people
The number who actually took part.
- Started
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Dec 2011
- Finished
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Dec 2025
- Lead sponsor
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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
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1 year to 30 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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 have a diagnosis of neuroblastoma (International Classification of Diseases for Oncology \[ICD-O\] morphology 9500/3) or ganglioneuroblastoma verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; patients with the following disease stages at diagnosis are eligible, if they meet the other specified criteria: * Patients with newly diagnosed neuroblastoma with International Neuroblastoma Staging System (INSS) stage 4 are eligible with the following: * v-MYC myelocytomatosis viral related oncogene, neuroblastoma derived (avian) (MYCN) amplification (\> 4-fold increase in MYCN signals as compared to reference signals) and age \>= 365 days regardless of additional biologic features * Age \> 18 months (\> 547 days) regardless of biologic features * Age 12-18 months (365-547 days) with any of the following 3 unfavorable biologic features (MYCN amplification, unfavorable pathology and/or deoxyribonucleic acid \[DNA\] index = 1) or any biologic feature that is indeterminant/unsatisfactory/unknown * Patients with newly diagnosed neuroblastoma with INSS stage 3 are eligible with the following: * MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), and age \>= 365 days, regardless of additional biologic features * Age \> 18 months (\> 547 days) with unfavorable pathology, regardless of MYCN status * Patients with newly diagnosed INSS stage 2a/2b with MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals) and age \>= 365 days, regardless of additional biologic features * Patients \>= 365 days initially diagnosed with: INSS stage 1, 2, 4S who progressed to a stage 4 without interval chemotherapy; these patients must have been enrolled on ANBL00B1; it is to be noted that study enrollment must occur within 4 weeks of progression to stage 4 for INSS stage 1, 2, 4S * Patients must have had no prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy per low- or intermediate-risk neuroblastoma therapy (P9641, A3961, ANBL0531) prior to determination of MYCN amplification and histology * Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 OR serum creatinine based on age and/or gender as follows: * =\< 0.6 mg/dL (1 to \< 2 years of age) * =\< 0.8 mg/dL (2 to \< 6 years of age) * =\< 1.0 mg/dL (6 to \< 10 years of age) * =\< 1.2 mg/dL (10 to \< 13 years of age) * =\< 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to \< 16 years of age) * =\< 1.7 mg/dL (male) or 1.4 mg/dL (female) ( \>= 16 years of age) * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age * Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase \[AST\]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 10 x ULN for age * Shortening fraction \>= 27% by echocardiogram or * Ejection fraction \>= 50% by radionuclide evaluation * No known contraindication to peripheral blood stem cell (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 * 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 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 that are 12-18 months of age with INSS stage 4 and all 3 favorable biologic features (i.e., non-amplified MYCN, favorable pathology, and DNA index \> 1) are not eligible * Patients are not eligible if they have received local radiation which includes any of the following: 1200 centigray (cGy) to more than 33% of both kidneys (patient must have at least 1 kidney that has not exceeded the dose/volume of radiation listed) or 1800 cGy to more than 30% of liver and/or 900 cGy to more than 50% of liver; emergency local irradiation is allowed prior to study entry, provided the patient still meets eligibility criteria
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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C S Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
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Children's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, 30329, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Alabama
Birmingham, Alabama, 35233, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Connecticut Children's Medical Center
Hartford, Connecticut, 06106, United States
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Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
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Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Phoenix Childrens Hospital
Phoenix, Arizona, 85016, United States
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, 99204, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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UCSF Medical Center-Mission Bay
San Francisco, California, 94158, United States
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UCSF Medical Center-Parnassus
San Francisco, California, 94143, United States
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UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27599, United States
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UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, 75390, United States
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University of Alabama at Birmingham Cancer Center
Birmingham, Alabama, 35233, United States
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University of Chicago Comprehensive Cancer Center
Chicago, Illinois, 60637, United States
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University of Wisconsin Carbone Cancer Center - University Hospital
Madison, Wisconsin, 53792, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Engineered immune cells take aim at childhood cancer
- Engineered immune cells take aim at childhood cancer
- New hope for tough tumors: Half-Matched bone marrow transplant trial
- New hope for kids with aggressive cancer: tailored therapy trial launches
- New hope for kids with aggressive nerve cancer: immune-boosting combo trial opens
- Smart treatment strategy aims to spare kids with neuroblastoma from unnecessary side effects