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Radioactive 'Smart Bomb' targets tough childhood cancer

NCT ID NCT01163383

What the study statuses mean

This study's is highlighted.

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This expanded access program provides 131 I-MIBG therapy for children and young adults with refractory or relapsed neuroblastoma, a cancer that hasn't responded to standard treatments. The therapy uses a radioactive compound that is taken up by cancer cells, delivering radiation directly to kill them. Patients receive intravenous doses every 4-8 weeks, and the program also collects data on how well it works and any side effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
131 I-Metaiodobenzylguanidine (131 I-MIBG)
What this could lead to
If successful, this therapy could offer a treatment option for children with neuroblastoma that hasn't responded to standard treatments, potentially shrinking tumors and improving outcomes.
What could go wrong
This is an expanded access protocol, not a formal trial, so data is limited. Response rates vary, and there are risks from radiation exposure, including potential damage to healthy tissues.

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: * Refractory or relapsed neuroblastoma * Age greater than 1 year. * Performance Level: Patients must have a Karnofsky or Lansky performance status of equal to or greater than 50 percent * 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. Disease evaluable by MIBG scan must be present within 8 weeks of study entry and subsequent to any intervening therapy. The principal or co-investigator can waive the requirement for intervening therapy if in their judgment this would pose undue risk and would not affect ability to judge treatment effectiveness. * Stem cells: Patients must have a hematopoietic stem cell product available for re-infusion after MIBG treatment at doses of \>/= 12 mCi/kg. The recommended minimum quantity for peripheral blood stem cells based on documentation from the time of collection is 1.0 x 10\^6 CD34+ cells/kg. The minimum dose for bone marrow is 1.0 x 10\^8 mononuclear cells/kg. If no stem cells are available, 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. Subjects cannot be receiving chemotherapy, cytokine therapy or other investigational agents, and must have fully recovered from the toxic effects of any prior therapy. No investigational agents are allowed in this time frame, but FDA-approved drugs for other indications that are not cytotoxic are allowed to be used off label if this is considered in the best interest of the patient by the investigator. No concomitant cytotoxic therapy is permitted with the exception of vorinostat. * Liver function: Bilirubin ≤2x upper limit of normal; AST/ALT ≤10x upper limit of normal * Kidney function: Creatinine ≤3x upper limit of normal * Signed informed consent: The patient and/or the patient's legally authorized guardian must provide written informed consent to participate in this expanded access protocol. Exclusion criteria * Patients with disease of any major organ system that would compromise their ability to withstand therapy, as deemed by the principal investigator or treating sub-investigator. * 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 damage to the fetus. * Patients who are on hemodialysis * Patients with uncontrolled infections * Exceptions to the above eligibility criteria may be allowed if approved by the principal investigator as long as exception does not compromise the safety of the subject and the exception is clearly documented. Each protocol exception must be reviewed by the Institutional Review Board before therapy is initiated.

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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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names. We don't republish those.

    Open the record ↗

  3. 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

  • The Children's Hospital of Philadelphia

    AVAILABLE

    Philadelphia, Pennsylvania, 19104, United States

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