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NCT01175356CompletedPhase 1

Induction Therapy Including 131 I-MIBG and Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma Undergoing Stem Cell Transplant, Radiation Therapy, and Maintenance Therapy With Isotretinoin

A COG Pilot Study of Intensive Induction Chemotherapy and 131I-MIBG Followed by Myeloablative Busulfan/Melphalan (Bu/Mel) for Newly Diagnosed High-Risk Neuroblastoma

Source: ClinicalTrials.gov · Last updated 23 Jan 2026

Brief summary (registry, in English)

This pilot clinical trial studies induction therapy followed by iobenguane I 131 and chemotherapy in treating patients with newly diagnosed high-risk neuroblastoma undergoing stem cell transplant, radiation therapy, and maintenance therapy with isotretinoin. Radioisotope therapy, such as iobenguane I 131, releases radiation that kills tumor cells. Drugs used in chemotherapy, such as carboplatin, etoposide phosphate, busulfan, and melphalan, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell transplant may be able to replace blood-forming cells that are destroyed by iobenguane I 131 and chemotherapy. Giving radioisotope therapy, chemotherapy, and peripheral stem cell transplant may kill more tumor cells.

Status (registry)
Completed
Phase (registry)
Phase 1
Study type
Interventional
Enrollment
99 (actual)
First submitted
3 Aug 2010
Start date
21 Dec 2011
Primary completion date
28 Sept 2016
Lead sponsor
Children's Oncology Group
NCT number
NCT01175356

What you commit to

  • Study type: Interventional · Phase 1
  • Participants enrolled: 99
  • From 21 Dec 2011 to the primary completion date, 28 Sept 2016
  • 25 sites in 1 country
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • 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

Age

  • 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

Other

  • 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

Who the study is looking for

  • 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

Age in the registry: 1 year to 30 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (25)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
C S Mott Children's Hospital
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Ann ArborUnited StatesUnknown status
Children's Healthcare of Atlanta - Arthur M Blank Hospital
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AtlantaUnited StatesUnknown status
Children's Hospital Colorado
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AuroraUnited StatesUnknown status
University of Alabama at Birmingham Cancer Center
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BirminghamUnited StatesUnknown status
Children's Hospital of Alabama
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BirminghamUnited StatesUnknown status
Dana-Farber Cancer Institute
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BostonUnited StatesUnknown status
UNC Lineberger Comprehensive Cancer Center
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Chapel HillUnited StatesUnknown status
Medical University of South Carolina
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CharlestonUnited StatesUnknown status
University of Chicago Comprehensive Cancer Center
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ChicagoUnited StatesUnknown status
Cincinnati Children's Hospital Medical Center
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CincinnatiUnited StatesUnknown status
UT Southwestern/Simmons Cancer Center-Dallas
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DallasUnited StatesUnknown status
Duke University Medical Center
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DurhamUnited StatesUnknown status
Cook Children's Medical Center
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Fort WorthUnited StatesUnknown status
Connecticut Children's Medical Center
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HartfordUnited StatesUnknown status
Children's Hospital Los Angeles
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Los AngelesUnited StatesUnknown status
University of Wisconsin Carbone Cancer Center - University Hospital
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MadisonUnited StatesUnknown status
Children's Hospital of Wisconsin
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MilwaukeeUnited StatesUnknown status
Children's Hospital of Philadelphia
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PhiladelphiaUnited StatesUnknown status
Phoenix Childrens Hospital
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PhoenixUnited StatesUnknown status
Primary Children's Hospital
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Salt Lake CityUnited StatesUnknown status
UCSF Medical Center-Parnassus
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San FranciscoUnited StatesUnknown status
UCSF Medical Center-Mission Bay
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San FranciscoUnited StatesUnknown status
Seattle Children's Hospital
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SeattleUnited StatesUnknown status
Providence Sacred Heart Medical Center and Children's Hospital
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SpokaneUnited StatesUnknown status
Children's National Medical Center
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Washington D.C.United StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.