New hope for kids with resistant neuroblastoma: targeted radiation plus chemo tested

NCT ID NCT00509353

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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 This study
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 Results may not be published yet.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
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

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Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase study tested a combination of two chemotherapy drugs (irinotecan and vincristine) plus a radioactive drug (131I-MIBG) in 26 children with high-risk neuroblastoma that had come back or not responded to standard treatment. The goal was to find the safest dose of the radioactive drug when given with the chemo. The approach aims to deliver radiation directly to tumor cells while using chemo to stop cancer growth.

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.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

26 people

The number who actually took part.

Start date

Jan 2007

Finished

May 2012

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 to 30 years

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.

DISEASE CHARACTERISTICS: Inclusion criteria: * Must have a diagnosis of neuroblastoma by histologic verification and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines * Must have high-risk neuroblastoma AND meets at least one of the following criteria: * Recurrent or progressive disease at any time * Biopsy not required, even if there is partial response to intervening therapy * Refractory disease (i.e., less than a partial response to frontline therapy, including a minimum of 4 courses of chemotherapy) * Biopsy not required * If the patient has not had previous myeloablative therapy, preference will be given to NANT-2001-02 (iodine I 131 metaiodobenzylguanidine \[\^131I-MIBG\] + CEM) * Persistent disease after at least a partial response to frontline therapy (i.e., patient still has residual disease by MIBG scan, CT/MRI scan, or bone marrow) * Biopsy required (bone marrow biopsy included) of at least one residual site demonstrating viable neuroblastoma * If the patient has not had previous myeloablative therapy, preference will be given to NANT-2001-02 (\^131I-MIBG + CEM) * Must have evidence of MIBG uptake into tumor at ≥ 1 site within 4 weeks prior to study entry and subsequent to any intervening therapy * Must have autologous hematopoietic stem cell product available and it must be free of tumor cell contamination (0 tumor cells /1,000,000 nucleated cells), cryopreserved, and available for re-infusion after \^131I-MIBG treatment, if immunocytology has been performed on the stem cell product * If immunocytology has not been performed on the stem cell product, then bilateral bone marrow aspirates and biopsies must have been negative by morphology within 4 weeks before or after the stem cell collection * If the patient had no bone marrow disease documented at diagnosis or at any time prior to peripheral blood stem cell (PBSC) harvest then the criteria for bilateral bone marrow aspirates/biopsies is waived * The minimum dose is as follows: * Purged PBSC 2.0 x 10\^6 viable CD34+ cells/kg * Immuno-magnetically purged cells are permitted * Unpurged PBSC 2 x 10\^6 CD34+ cells/kg (minimum is same for PBSC from identical twin) * Cells from identical twins are permitted * Other allogeneic cells are not allowed * CD34+ selected cells are not permitted PATIENT CHARACTERISTICS: Inclusion criteria: * Lansky or Karnofsky performance status ≥ 50% * Life expectancy ≥ 6 weeks * Hemoglobin ≥ 8 g/dL (transfusion allowed) * ANC ≥ 750/μL (no hematopoietic growth factors within 7 days of starting irinotecan hydrochloride) * Platelet count ≥ 50,000/μL (transfusion independent, defined as no platelet transfusion for 2 weeks) * Glomerular filtration rate (GFR) or creatinine clearance ≥ 60 mL/min OR age-adjusted serum creatinine ≤ 1.5 x normal, according to the following: * 0.8 mg/dL (≤ 5 years of age) * 1.0 mg/dL (6 to 10 years of age) * 1.2 mg/dL (11 to 15 years of age) * 1.5 mg/dL (≥ 16 years of age) * Total bilirubin ≤ 1.5 x normal for age * ALT and AST \< 3 x normal for age * All post-menarchal females must have a negative beta-HCG * Males and females of reproductive age and childbearing potential must use effective contraception for the duration of study participation * Ejection fraction ≥ 55% by echocardiogram or radionuclide MUGA OR fractional shortening ≥ 27% by echocardiogram * Normal lung function * Patients with other ongoing serious medical issues must be approved by the study chair prior to study registration Exclusion criteria: * Pregnancy or breast feeding * Dyspnea at rest, exercise intolerance, pleural effusion, or oxygen requirement * Disease of any major organ system that would compromise the patient's ability to withstand therapy * Documented allergy to third generation cephalosporins * Active diarrhea (defined as ≥ grade 2 per CTCAE v3) * Active or uncontrolled infection, including C. difficile * Patients on prolonged antifungal therapy are eligible if suspected radiographic lesions are culture and biopsy negative and patient meets other organ function criteria * Patients and/or families who are physically and psychologically unable to cooperate with the radiation safety isolation * Patient weight that would require exceeding a maximum total allowable dose of \^131I-MIBG (per institutional guidelines) * Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study PRIOR CONCURRENT THERAPY: Inclusion criteria: * Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy before study entry * At least 3 weeks since prior myelosuppressive or biologic therapy * At least 2 weeks since prior radiation therapy * Radiation therapy should not be given to the only site of measurable or evaluable disease * At least 3 months since prior large field radiation therapy (i.e., craniospinal radiation therapy, total lung radiation therapy, or radiation therapy to \> 50% of marrow space) * At least 3 months since prior autologous stem cell transplantation * Must meet adequate bone marrow function postmyeloablative therapy * At least 7 days since prior cytokines or hematopoietic growth factors * Prior irinotecan hydrochloride and vincristine therapy allowed provided the patient recovered to adequate bone marrow function as specified in the protocol Exclusion criteria: * Prior \^131I-MIBG * Prior external beam radiation therapy to the liver or kidneys * Prior allogeneic stem cell transplantation * Prior whole abdominal radiation therapy, total-body irradiation, or local radiation therapy that includes any of the following: * 1,200 cGy to more than 33% of both kidneys (patient must have at least one kidney that has not exceeded the dose/volume of radiation listed) * 1,800 cGy to more than 30% of liver and/or 900 cGy to more than 50% of liver * Other concurrent cancer chemotherapy or immunomodulating agents (including steroids) * Steroids may be used in the prevention and treatment of transfusion/infusion reactions and for the treatment of edema associated with CNS lesions * Concurrent palliative radiotherapy to localized painful lesions * Concurrent aprepitant (Emend) * Concurrent ketoconazole or St. John's wort * Medications that interfere with MIBG uptake during the week prior to or after MIBG therapy * Concurrent enzyme-inducing anticonvulsants (e.g., phenobarbital, phenytoin, or carbamazepine) * Nonenzyme-inducing anticonvulsants (e.g., Keppra) may be allowed * Concurrent hemodialysis * Any other concurrent anticancer agents or radiation therapy

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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.

Contacts and locations

Locations

  • AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus

    Atlanta, Georgia, 30322, United States

  • C.S. Mott Children's Hospital at University of Michigan Medical Center

    Ann Arbor, Michigan, 48109-0286, United States

  • Children's Hospital Boston

    Boston, Massachusetts, 02115, United States

  • Children's Hospital and Regional Medical Center - Seattle

    Seattle, Washington, 98105, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104-4318, United States

  • Childrens Hospital Los Angeles

    Los Angeles, California, 90027-0700, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • Cook Children's Medical Center - Fort Worth

    Fort Worth, Texas, 76104, United States

  • Lucile Packard Children's Hospital at Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • UCSF Helen Diller Family Comprehensive Cancer Center

    San Francisco, California, 94115, United States

  • University of Chicago Comer Children's Hospital

    Chicago, Illinois, 60637, United States

  • University of Wisconsin Paul P. Carbone Comprehensive Cancer Center

    Madison, Wisconsin, 53792-6164, United States

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