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.
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
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.
iobenguane I 131Radiation
| Study group | Role | What they receive |
|---|---|---|
| Single Group | Experimental | Not 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.
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.
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)
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.
- First posted31 Jul 2007 (estimated)
- Study startJan 2007
- Primary completionOct 2010
- Study completionMay 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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)
Sponsor and investigators
- Lead sponsor
- New Approaches to Neuroblastoma Therapy Consortium
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Steven DuBois, MD, Principal investigator, UCSF Medical Center at Parnassus
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
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Irinotecan and Vincristine With 131I-MIBG Therapy for Resistant/Relapsed High-Risk Neuroblastoma, A Phase I Study
- ClinicalTrials.gov ID
- NCT00509353
- Acronym / sponsor ID
- CDR0000558042
- Conditions
- Neuroblastoma
- Keywords
- recurrent neuroblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- New Approaches to Neuroblastoma Therapy ConsortiumUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jan 2007
- Primary completion
- Oct 2010 (actual)
- Study completion
- May 2012 (actual)
- First posted
- 31 Jul 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Group | Experimental | Drug: irinotecan hydrochloride, Drug: vincristine sulfate, Radiation: iobenguane I 131 |
Who may qualify
- Ages
- 1 year to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (48 listed)
- 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:
- 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
- Lansky or Karnofsky performance status ≥ 50%
Key exclusion criteria (27 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| irinotecan hydrochloride | Drug | Single Group |
| vincristine sulfate | Drug | Single Group |
| iobenguane I 131 | Radiation | Single Group |
Primary outcome
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.
To determine the dose limiting toxicities of 131I-MIBG combined with irinotecan/vincristine.
Time frame: Adverse events, clinically significant changes in laboratory results, and vital signs, to be measured throughout the study.
1 secondary outcome
Within the confines of a Phase I study, to determine if there is a therapeutic response to this regimen.
Time frame: Disease response will be evaluated at baseline, prior to each cycle and at the end of treatment.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven DuBois, MD principal investigator
UCSF Medical Center at Parnassus
12 sites in 1 country: see every site and its status.
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?
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; AzTrials last refreshed its copy on 8 Nov 2026.