Can a radioated molecule shrink untreatable neuroblastoma?
NCT ID NCT01370330
First seen Jul 23, 2026 · Last updated Jul 24, 2026 · Updated 1 time
Summary
This trial offers a radioactive drug called 131I-MIBG to children and adults with advanced neuroblastoma that has not responded to standard treatments. The drug is designed to seek out and deliver radiation directly to tumor cells. The goal is to provide palliative control of the disease in a compassionate-use setting.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- a radioactive drug called 131I-MIBG (metaiodobenzylguanidine) that delivers radiation directly to tumor cells
- What this could lead to
- If it works, this could offer a palliative treatment option for people with advanced neuroblastoma who have no standard therapies left.
- What could go wrong
- This is a small, compassionate-use protocol, not a controlled trial, so results may be limited. The treatment involves significant radiation exposure and requires stem cell backup for higher doses.
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Study facts
What this study's own registry entry says, in plain language.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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 and older
- 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: * Diagnosis: Refractory or relapsed neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical tumor cells in the bone marrow. Refractory, progressive or metastatic pheochromocytoma/paraganglioma or related tumor. * MIBG uptake: Tumors must be shown to be MIBG avid within 6 weeks prior to enrollment * Age \> 1 year and able to cooperate with radiation safety restrictions during therapy period. Patients with pheochromocytoma/paraganglioma and related tumors must be between 1 and 12 years of age. * Life Expectancy: greater than 6 weeks. * Lansky and Karnofsky Performance Status: 60% or higher. * Disease status: Failure to respond to standard therapy (usually combination chemotherapy with or without radiation and surgery) or development of progressive disease at any time (any new lesion or an increase in size of \>25% of a pre-existing lesion). Disease evaluable by MIBG scan must be present within 6 weeks of study entry and subsequent to any intervening therapy. * Stem cells: Patients must have an autologous hematopoietic stem cell product available for re-infusion after MIBG treatment at doses of \>12 mCi/kg if needed. The minimum quantity for purged or unpurged peripheral blood stem cells is 1.0 x 10\^6 cluster of differentiation 34 (CD34)+ cells/kg (optimum \> 2 x 10\^6 CD34+ cells/kg). The minimum dose for bone marrow is 1.0 x 10\^8 mononuclear cells/kg (optimum \> 2.0 x 10\^8 mononuclear cells/kg). If no stem cells are available, then the dose of 131I-MIBG should be \<12 mCi/kg . * Prior Therapy: Patients may enter this study with or without re-induction therapy for recurrent tumor. Patients must have fully recovered from the toxic effects of any prior therapy. At least 2 weeks should have elapsed since any anti-tumor therapy and the patient must meet hematologic criteria below. Three months should have elapsed in the case of completing radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation). Cytokine therapy \[eg granulocyte-colony stimulating factor (G-CSF), granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-6 (IL-6), erythropoietin\] must be discontinued a minimum or 24 hours prior to MIBG therapy. Prior 131I-MIBG therapy is allowed if \> 6 months previous and if the patient has adequate hematopoietic stem cells available and if cumulative 131I-MIBG dose will not exceed 60 mCi/kg. * Organ Function * Liver function: bilirubin \<2x normal and aspartate aminotransferase (AST)/Alanine aminotransferase (ALT) \< 10x normal. * Kidney function: Creatinine less than or equal to 2 * Hematopoietic Criteria Patients must have adequate hematopoietic function (without transfusion): absolute neutrophil count (ANC) \>.750 x 10E9/L; Platelets \>50 x 10E9/L if stem cells are not available; if stem cells are available, the patient should be independent of platelet transfusions with a platelet count of at least 20 x 10E9/L. Hemoglobin \>10g/dl at time of treatment (transfusion allowed). Patients with granulocytopenia and/or thrombocytopenia due to tumor metastatic to the bone marrow may be eligible after discussion with study chair or designee. * Normal lung function as manifested by no dyspnea at rest or exercise intolerance, no oxygen requirement. * No clinically significant cardiac dysfunction * Signed informed consent: The patient and/or the patient's legally authorized guardian must acknowledge in writing that consent to become a study subject has been obtained, in accordance with institutional policies approved by the U.S. Department of Health and Human Services. Exclusion Criteria: * Patients with disease of any major organ system that would compromise their ability to withstand therapy. Any significant organ impairment should be discussed with the Study Chair or Vice Chair prior to patient entry. * Because of the teratogenic potential of the study medications, no patients who are pregnant or lactating will be allowed. Patients of childbearing potential must practice an effective method of birth control while participating on this study, to avoid possible pregnancy. * Patients who are on hemodialysis. * Patients with active infections that meet grade 3-4 toxicity criteria. * Patients with pheochromocytoma or paraganglioma who have any proteinuria on urinalysis must have a 24-hr urine collection for protein. If there is proteinuria above the reference range on a 24-hour urine collection, they are excluded due to increased risk of respiratory complications.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
1 site. The list below names each one and where it is.
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The official record
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Contacts and locations
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Locations
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University of California, San Francisco
AVAILABLESan Francisco, California, 94143, United States
Contact Email: •••••@•••••
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