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NeuroblastomaPhase 1NCT00509353

N2004-06: Irinotecan and Vincristine With 131I-MIBG Therapy for Resistant/Relapsed High-Risk Neuroblastoma

A Phase 1 study testing irinotecan hydrochloride, vincristine sulfate and iobenguane I 131 in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 12 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

RATIONALE: Radioactive drugs, such as iodine I 131 metaiodobenzylguanidine (MIGB), may carry radiation directly to tumor cells and not harm normal cells. Drugs used in chemotherapy, such as irinotecan and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping… Read more about this study

Who is it for?
Ages 1 year to 30 years
Conditions the study is for
Neuroblastoma
What is being tested?
irinotecan hydrochloride, vincristine sulfate and iobenguane I 131
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
26 participants
Where
12 sitesUnited States
Study dates
Jan 2007to May 2012
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 30 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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
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  • 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:
  • 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
  • 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

Who cannot join

  • 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
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  • 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:
  • 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

Trial locations and contacts

12 sites in 1 country.

12 sites in 1 country

United States12 sites

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

    Ann Arbor, Michigan, 48109-0286

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

    Atlanta, Georgia, 30322

  • Children's Hospital Boston

    Boston, Massachusetts, 02115

  • University of Chicago Comer Children's Hospital

    Chicago, Illinois, 60637

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039

  • Cook Children's Medical Center - Fort Worth

    Fort Worth, Texas, 76104

  • Childrens Hospital Los Angeles

    Los Angeles, California, 90027-0700

  • University of Wisconsin Paul P. Carbone Comprehensive Cancer Center

    Madison, Wisconsin, 53792-6164

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

    Palo Alto, California, 94304

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104-4318

  • UCSF Helen Diller Family Comprehensive Cancer Center

    San Francisco, California, 94115

  • Children's Hospital and Regional Medical Center - Seattle

    Seattle, Washington, 98105

Other trials in United States

About this study

Official title: Irinotecan and Vincristine With 131I-MIBG Therapy for Resistant/Relapsed High-Risk Neuroblastoma, A Phase I Study

RATIONALE: Radioactive drugs, such as iodine I 131 metaiodobenzylguanidine (MIGB), may carry radiation directly to tumor cells and not harm normal cells. Drugs used in chemotherapy, such as irinotecan and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving iodine I 131 MIGB together with irinotecan and vincristine may kill more tumor cells.

PURPOSE: This phase I trial is studying the side effects and best dose of iodine I 131 MIGB when given together with irinotecan and vincristine in treating young patients with resistant or relapsed high-risk neuroblastoma.

Read the detailed description

OBJECTIVES:

Primary

  • To determine the maximum tolerated dose (MTD) of iodine I 131 metaiodobenzylguanidine when given in combination with fixed-dose irinotecan hydrochloride and vincristine in young patients with refractory or relapsed high-risk neuroblastoma.
  • To determine the dose-limiting toxicities of iodine I 131 metaiodobenzylguanidine when combined with fixed-dose irinotecan hydrochloride and vincristine.

Secondary

  • To determine if there is a therapeutic response to this regimen.

OUTLINE: This is a multicenter, dose-escalation study of iodine I 131 metaiodobenzylguanidine (^131I-MIBG).

Patients receive ^131I-MIBG IV over 1½-2 hours on day 1, vincristine IV on days 0 and 7, and irinotecan hydrochloride IV over 1 hour on days 0-4 and 7-11. Treatment repeats every 56 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed every 3 months for 1 year and then every 6 months thereafter.

Conditions
Neuroblastoma
Being tested
irinotecan hydrochloride, vincristine sulfate, iobenguane I 131
Keywords
recurrent neuroblastoma
Medical subject headings
Neuroblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT00509353
Study groupRoleWhat they receive
Single GroupExperimentalNot described
Receives: Drug: irinotecan hydrochloride; Drug: vincristine sulfate; Radiation: iobenguane I 131

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To determine the maximum tolerated dose (MTD) of 131I-MIBG given in combination with fixed-dose irinotecan/vincristine to children with high-risk refractory/relapsed neuroblastoma.

    Measured: Tolerability will be assessed throughout the study.

  2. To determine the dose limiting toxicities of 131I-MIBG combined with irinotecan/vincristine.

    Measured: Adverse events, clinically significant changes in laboratory results, and vital signs, to be measured throughout the study.

Secondary outcomes (1)

  1. Within the confines of a Phase I study, to determine if there is a therapeutic response to this regimen.

    Disease response will be evaluated by any of the following CT, MRI, MIBG, Bone Marrow, Urine Catecholamines at baseline, prior to each cycle and at the end of treatment.

    Measured: Disease response will be evaluated at baseline, prior to each cycle and at the end of treatment.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Jul 2007 (estimated)
  2. Study startJan 2007
  3. Primary completionOct 2010
  4. Study completionMay 2012

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
26 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00509353
Sponsor's protocol ID
CDR0000558042
Other IDs
P01CA081403, N2004-06, NANT-Draximage-2007-01
Status last verified
Nov 2023
First submitted
30 Jul 2007
Last updated
14 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing irinotecan hydrochloride, vincristine sulfate and iobenguane I 131 in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 12 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 48 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 14 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To determine the maximum tolerated dose (MTD) of 131I-MIBG given in combination with fixed-dose irinotecan/vincristine to children with high-risk refractory/relapsed neuroblastoma. (time frame: Tolerability will be assessed throughout the study.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00509353.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2026.

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