Can a radioactive 'Smart Bomb' take down resistant childhood cancers?
NCT ID NCT03015844
First seen Aug 06, 2026 · Last updated Aug 07, 2026 · Updated 1 time
Summary
This study offers access to a radioactive therapy called 131I-MIBG for people with neuroblastoma or pheochromocytoma that has returned or stopped responding to standard treatments. The therapy is designed to seek out and deliver radiation directly to tumor cells. Researchers are evaluating how well it controls symptoms, its side effects, and how long it may slow disease progression.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- 131I-MIBG (iobenguane), a radioactive molecule that targets and delivers radiation to tumor cells
- What this could lead to
- If effective, this therapy could offer a way to shrink tumors and ease symptoms for people with these rare cancers who have run out of standard options.
- What could go wrong
- This is an early-access, compassionate-use program, not a large controlled trial, so benefits are uncertain. The treatment carries risks of toxicity, including damage to bone marrow and other organs.
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
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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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365 days to 29 years
- Sex
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Anyone
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: Relapsed/Refractory neuroblastoma, with an original diagnosis made histologically or from elevated urine catecholamines with abnormal tumor cells in bone marrow OR relapsed/refractory pheochromocytoma. * Disease status: Progressive disease at any time (defined as any new lesion or an increase in size by \>25% of pre-existing disease), or a failure to respond to standard therapy. Patients must have evidence of MIBG uptake into tumor at ≥ one site within 6 weeks prior to entry on study and subsequent to any intervening therapy * Prior therapy: A minimum of two weeks since should have elapsed since any chemotherapy causing myelosuppression. It must be a minimum of three months since receiving radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation. For any other sites of radiation, at least 2 weeks should have elapsed. For patients who received radiation to the only site of MIBG-avid disease within two months of study entry, biopsy confirmation of residual active disease is required, with positive bone marrow being sufficient. At least 7 days should have elapsed since completion of therapy with a biologic agent and at least 3 half-lives should have elapsed since therapy with a monoclonal antibody. No cytokine therapy may be given within 24 hours of receiving 131I-MIBG. Patients may have received prior MIBG therapy, provided they demonstrated a response or stable disease initially, with progressive disease occurring at least 35 days following treatment. * Organ function: ANC \>500/uL, platelets \>20,000/uL with transfusion allowed. Bilirubin ≤2x ULN, AST/ALT ≤10x ULN.Serum Creatinine ≤2x ULN OR 24-hour creatinine clearance OR GFR ≥60ml/min/1.73m2. Normal lung function demonstrated by no dyspnea, exercise intolerance or oxygen requirement. Oxygen saturation ≥94% on room air. No clinically significant cardiac dysfunction and ejection fraction ≥45% on echocardiogram. * Stem cells: Patients must have a minimum of 2.0 x106/kg viable CD34+ peripheral blood stem cells for re-infusion following 131I-MIBG. An additional back-up of 2.0x106/kg CD34+ cells is recommended but not required. * Life expectancy longer than 8 weeks,Karnofsky or Lansky performance status of ≥ 50% Exclusion Criteria: * Pregnant or lactating patients * Disease of any organ system that would compromise the patient's ability to participate in the study, including hemodialysis. Significant organ impairment should be discussed with the Principal Investigator prior to study entry * Patients with active grade 3-4 infection, as defined by the NCI CTCAE V4.0. * Patients with known MBIG-avid brain parenchymal disease (leptomeningeal or skull based metastases are eligible). * In patients with metastatic pheochromocytoma, a urinalysis must be preformed prior to study enrollment. If proteinuria is present, a 24 hour urine must be collected and total protein determined. If the 24-hour urine protein is above the institutional upper limit of normal, the patient is excluded.
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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 places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Cohen Children's Medical Center
AVAILABLENew Hyde Park, New York, 11040, United States
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