Can a radioated molecule shrink untreatable neuroblastoma?

NCT ID NCT01370330

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access This study
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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.

Lead sponsor

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

1 year and older

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: * 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

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  1. The study's own enquiry address

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  2. The places running it

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Contacts and locations

Locations

  • University of California, San Francisco

    AVAILABLE

    San Francisco, California, 94143, United States

    Contact Email: •••••@•••••

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