NeuroblastomaPhase 2NCT00253435
N2001-02: I-MIBG With Intensive Chemotherapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma
A Phase 2 study testing Filgrastim, Carboplatin and Etoposide in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 15 sites in 2 countries.
Trial summary
RATIONALE: Radioactive drugs, such as iodine I 131 metaiodobenzylguanidine, may carry radiation directly to tumor cells and not harm normal cells. Drugs used in chemotherapy, such as carboplatin, etoposide, and melphalan, work in different ways to stop the growth of tumor cells, either by killing the cells or by… Read more about this study
- Who is it for?
- Ages 1 year to 29 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Filgrastim, Carboplatin and Etoposide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 15 sitesin 2 countries
- Study dates
- Sep 2005to Dec 2013
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 29 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Diagnosis of relapsed or refractory neuroblastoma
- Histologically confirmed and/or demonstration of tumor cells in bone marrow with elevated urinary catecholamine metabolites
- High-risk neuroblastoma must meet one of the following:
- Progressive disease prior to or after completion of induction therapy
- Mixed response or no response after completion of 4 courses of induction therapy
- Partial response after 4 courses of induction therapy allowed provided no prior participation in COG-A3973 or other phase III COG trials
- Measurable disease, defined as at least one metaiodobenzylguanidine (MIBG)-avid target lesion determined by diagnostic MIBG scan within 6 weeks of study entry (tumor sites that have received local irradiation within 3 months of study entry are not considered target lesions)
PATIENT CHARACTERISTICS:
Performance status
- Lansky 60-100% OR
- Karnofsky 60-100%
Life expectancy
- At least 2 months
Hematopoietic
- Hemoglobin ≥ 10 g/dL
- Absolute neutrophil count ≥ 750/mm^3
- Platelet count ≥ 50,000/mm^3 (if no marrow involvement by morphologic exam/no transfusion allowed) (> 20,000/mm^3 if metastatic tumor involvement of marrow by morphologic exam/transfusion allowed)
Hepatic
- Bilirubin < 1.3 mg/dL
- SGOT and SGPT < 5 times normal
- Hepatitis B surface antigen negative
- Hepatitis C negative
Renal
- Glomerular filtration rate or creatinine clearance ≥ 60 ml/min
- Creatinine ≤ 1.5 times normal for age as follows:
- 0.8 mg/dL (for patients ≤ 5 years of age)
- 1.0 mg/dL (for patients 6 to 10 years of age)
- 1.2 mg/dL (for patients 11 to 15 years of age)
- 1.5 mg/dL (for patients > 15 years of age)
Cardiovascular
- Ejection fraction ≥ 55% by echocardiogram or radionuclide MUGA OR
- Fractional shortening ≥ 27% by echocardiogram
Pulmonary
- Normal lung function defined as no dyspnea at rest and no oxygen requirement OR measured oxygen saturation > 93% on room air
Other
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- No disease of any major organ system that would preclude study compliance
- No concurrent hemodialysis
- No active infection requiring IV antivirals, antibiotics, or antifungals (patients on antifungal therapy are eligible provided they are culture- and biopsy-negative in suspected residual radiographic lesions)
- Patient weight within limits to receive ≤ maximum total allowable dose of ^131I-MIBG
PRIOR CONCURRENT THERAPY:
Biologic therapy
- No prior myeloablative transplantation
- Prior submyeloablative transplantation allowed at discretion of principal investigator
- More than 3 weeks since prior biologic therapy
Chemotherapy
- More than 3 weeks since prior myelosuppressive chemotherapy (6 weeks for mitomycin C or nitrosoureas)
- No prior melphalan therapy with a total dose of > 100 mg/m^2
Radiotherapy
- See Disease Characteristics
- At least 6 weeks since prior radiotherapy (6 months for craniospinal or whole lung radiotherapy)
- No prior total body irradiation
- No prior iodine I 131 MIBG (^131I-MIBG)
- No prior total abdominal or whole liver radiotherapy
- No prior local radiotherapy, including any of the following:
- 1200 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)
- 1800 cGy to more than 30% of liver and/or 900 cGy to more than 50% of liver
Other
- Recovered from all prior therapy
- No medications with a potential interference of ^131I-MIBG uptake 1 week before and 2 weeks after completion of ^131I-MIBG
Trial locations and contacts
15 sites in 2 countries.
15 sites in 2 countries
United States14 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
Dana-Farber/Harvard Cancer Center at Dana-Farber Cancer Institute
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
Texas Children's Cancer Center and Hematology Service at Texas Children's Hospital
Houston, Texas, 77030-2399
Childrens Hospital Los Angeles
Los Angeles, California, 90027-0700
University of Wisconsin Paul P. Carbone Comprehensive Cancer Center
Madison, Wisconsin, 53792-6164
Morgan Stanley Children's Hospital of New York-Presbyterian
New York, New York, 10032
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, 94143
Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, 98105
About this study
Official title: I-Metaiodobenzylguanidine (MIBG) With Intensive Chemotherapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma
RATIONALE: Radioactive drugs, such as iodine I 131 metaiodobenzylguanidine, may carry radiation directly to tumor cells and not harm normal cells. Drugs used in chemotherapy, such as carboplatin, etoposide, and melphalan, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Radiation therapy uses high-energy x-rays to kill tumor cells. An autologous peripheral stem cell or bone marrow transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy. Giving iodine I 131 metaiodobenzylguanidine and combination chemotherapy with an autologous peripheral stem cell or bone marrow transplant may allow more chemotherapy to be given so that more tumor cells are killed. Giving radiation therapy after an autologous peripheral stem cell or bone marrow transplant may kill any remaining tumor cells.
PURPOSE: This phase II trial is studying how well giving iodine I 131 metaiodobenzylguanidine together with combination chemotherapy and radiation therapy works in treating patients who are undergoing an autologous peripheral stem cell or bone marrow transplant for relapsed or refractory neuroblastoma.
Read the detailed description
OBJECTIVES:
Primary
- Determine the response rate in patients with relapsed or refractory neuroblastoma treated with iodine I 131 metaiodobenzylguanidine (^131I-MIBG) and combination chemotherapy comprising carboplatin, etoposide, and melphalan followed by autologous bone marrow or peripheral blood stem cell transplantation and radiotherapy.
Secondary
- Determine the hematopoietic and nonhematopoietic toxicity of this regimen in these patients.
- Determine the tumor self-absorbed radiation dose (TSARD) in patients with measurable soft tissue lesions treated with this regimen.
- Correlate the TSARD with tumor response in patients with measurable residual soft tissue disease treated with this regimen.
OUTLINE: This is a multicenter study. Patients are stratified according to risk (poor-risk group [mixed or no response to induction therapy or progression during or after induction therapy] vs good-risk group [partial response after 4 courses of induction therapy]) and kidney function at study entry (glomerular filtration rate [GFR] ≥ 100 mL/min vs GFR 60-99 mL/min)
- Stem cell harvest: Patients undergo a peripheral blood stem cell harvest or bone marrow harvest provided they have an adequate number of cells available. At least 2 weeks later, patients proceed to iodine I 131 metaiodobenzylguanidine (^131I-MIBG) and combination chemotherapy.
- 131I-MIBG and combination chemotherapy: Patients receive ^131I-MIBG IV over 2 hours on day -21, carboplatin IV continuously on days -7 to -4, etoposide IV continuously on days -7 to -4, and melphalan IV over 1 hour on days -7 to -5.
- Stem cell infusion and filgrastim (G-CSF): Three days after completion of chemotherapy, patients undergo transplantation of either stem cells or bone marrow on day 0. Patients also receive G-CSF subcutaneously or IV over 1 hour once daily beginning on day 0 and continuing until blood counts return to normal.
- Radiotherapy: Once blood counts return to normal, patients undergo radiotherapy to primary and metastatic sites that have not received previous irradiation over 12 days beginning after day 42.
After completion of study treatment, patients are followed for 2 years and then periodically thereafter.
PROJECTED ACCRUAL: Approximately 50 patients (40 low-risk patients and 8-10 high-risk patients) will be accrued for this study.
- Conditions
- Neuroblastoma
- Being tested
- Filgrastim, Carboplatin, Etoposide, Melphalan, Peripheral blood stem cell infusion
- 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.
FilgrastimBiological
Also called G-CSF
Filgrastim 5 micrograms/kg/day S.C. or IV will be given daily beginning on Day 0. The first dose should begin four hours after the stem cell infusion is completed. Filgrastim will continue daily until the ANC >=1500/uL for three consecutive days.
CarboplatinDrug
Also called cis-diammine; Paraplatin; Paraplatin-AQ
The carboplatin will be administered as a continuous IV infusion Day - 7 through Day - 4, with dosing based upon pretreatment GFR levels. The carboplatin should be diluted to a concentration of 0.3 mg/ml in D5W 0.45NS and infused concomitantly with etoposide through the same central venous catheter using a "Y" connector; a controlled rate infusion pump is used for each arm of the "Y".
EtoposideDrug
Also called VP-16; Etopophos
The etoposide shall be administered day -7 through day -4 via continuous intravenous infusion over 96 hours. For patients with a corrected GFR >= 100 ml/min/1.72 m^2, a dose of 300 mg/m^2/day (10 mg/kg/day if child is < 12 kg) shall be given. For patients with a corrected GFR 60-99 ml/min/1.72 m^2, the etoposide will be administered at a dose of 160 mg/m^2/day (5.3 mg/kg/day). The etoposide will be diluted in D5W 0.45%NS at a concentration of < 0.4 mg/ml. Etoposide should not be mixed with carboplatin, but administered using a Y-connector.
MelphalanDrug
Also called L-phenylalanine mustard; L-PAM; Alkeran; Sarcolysin
For patients in either the normal GFR strata (>=100 ml/min/1.73 m^2), or reduced GFR strata (60-99 ml/min/1.73m^2), melphalan shall be administered at a dose of 60 mg/m^2/day (2 mg/kg/day if child is < 12 kg) on day -7, -6, and -5 of study. The melphalan should be infused at a rate of less than 10 mg/minute, and should complete within 1 hour of reconstitution each day. The melphalan should be diluted in 0.9% NaCl at a concentration < 2 mg/ml. The total dosage of melphalan to be administered will be 180 mg/m^2.
Peripheral blood stem cell infusionProcedure or surgery
Stem cells or marrow will be infused on day 0 of study therapy. Where the DMSO concentration in the stem cell product would exceed accepted level for infusion within a 24 hour period, stem cell products may be infused over two days to meet this standard. For purged PBSC: A minimum of 2.0 x 10^6 viable CD34+ cells/kg must be available. For unpurged PBSC, a minimum of 2.0 x 10^6 viable CD34+ cells/kg must be available. Having a back-up of 2.0 x 10^6 viable CD34+ cells/kg purged or unpurged PBSC is recommended but not required. For purged bone marrow, a minimum of 1.5 x10^8 mononuclear cells/kg must be available.
131I-MIBGRadiation
Also called Iobenguane I 131
Therapeutic 131I MIBG will be synthesized by Draximage Canada.with specific activity between 15 and 25mCi/ml. Radiopurity will be initially determined by Draximage, prior to shipment to participating centers. Free radioiodide content must then be rechecked at the treating center prior to infusion using HPLC or Sep-Pac methodology.
Radiation therapyRadiation
Local irradiation is to be given to previously non-irradiated primary and metastatic sites of disease. Local irradiation should not start till the patient is medically stable, has an ANC > 1000/uL, platelets > 30,000 / uL, and is > 42 days post transplant. Recommended radiation guidelines consist of 2160 cGy total, given over 12 days using a single 180 cGy fraction/day. Any delay in local radiation that would extend treatment beyond day +84 should be discussed with the study chair. Local radiation will be administered at a participating NANT member site.
| Study group | Role | What they receive |
|---|---|---|
| All the patients enrolled in the study | Experimental | This is a single arm study. The following description applies to all the patients who are enrolled in the study:
On Day -21, patients receive 131I-MIBG infusion.
On Day -7, Day -6, Day -5, patients receive Carboplatin, Etoposide, Melphalan.
On Day -4, patients receive Carboplatin, Etoposide.
On Day -3, Day -2, Day -1, patients rest.
On Day 0, patients receive peripheral blood stem cell infusion.
Dosing of Carboplatin, Etoposide and Melphalan is based upon whole body dosimetry (cGy) estimates.
Filgrastim 5 micrograms/kg/day S.C. or IV will be given daily beginning on Day 0.
Local radiation therapy is to be given to previously non-irradiated primary and metastatic sites of disease. Receives: Biological: Filgrastim; Drug: Carboplatin; Drug: Etoposide; Drug: Melphalan; Procedure: Peripheral blood stem cell infusion; Radiation: 131I-MIBG; Radiation: Radiation therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion
Tumor response based on evaluation performed on day 60 or at the time of disease progression/recurrence or start of another treatment - whichever comes first. Such evaluations will include 123I-MIBG scan, CT/MRI, urine catecholamine measurement, and bone marrow analysis (for those with marrow disease at study entry).
Measured: Response assessed 60 days post stem cell infusion
Secondary outcomes (3)
Event-free Survival (EFS) at 3 Years
EFS will be measured from start of treatment until progression, death or start of another treatment - whichever comes first. We report the estimated probability of EFS at 3 years.
Measured: 3 years since start of treatment
Engraftment DLT
- Engraftment toxicity: delayed engraftment and/or failure to engraft defined as:
- neutrophils (ANC) < 500/μL by day 28 post transplant, or
- platelets < 20,000 /μL by day 56 post transplant, or
- if additional stem cells are required to be infused for any medical reason prior to initial engraftment of neutrophils or platelets.
Measured: From treatment start until 60 days post stem cell infusion
Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS
Dose limiting veno-occlusive disease (VOD) defined as:
- the presence of hepatomegaly with right upper quadrant tenderness and an elevation of total bilirubin > grade 1, PLUS
- the presence of grade 3 abnormalities of any ONE of the following: total bilirubin, hypoalbuminemia, weight gain, or hypoxia without other attribution
Measured: Between start of MIBG treatment and 60 days post stem cell infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2005 (estimated)
- Study startSep 2005
- Primary completionDec 2012
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 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
- Gregory Yanik, MD, Study chair, University of Michigan Rogel Cancer CenterKatherine K. Matthay, MD, Principal investigator, University of California, San FranciscoJohn M. Maris, MD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00253435
- Sponsor's protocol ID
- CDR0000450148
- Other IDs
- P01CA081403, N2001-02
- Status last verified
- Apr 2023
- First submitted
- 11 Nov 2005
- Last updated
- 28 Apr 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- I-Metaiodobenzylguanidine (MIBG) With Intensive Chemotherapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT00253435
- Acronym / sponsor ID
- CDR0000450148
- Conditions
- Neuroblastoma
- Keywords
- recurrent neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- New Approaches to Neuroblastoma Therapy ConsortiumUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Sep 2005
- Primary completion
- Dec 2012 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 15 Nov 2005 (estimated)
- Results
- Posted 24 Aug 2016
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, may carry radiation directly to tumor cells and not harm normal cells. Drugs used in chemotherapy, such as carboplatin, etoposide, and melphalan, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Radiation therapy uses high-energy x-rays to kill tumor cells. An autologous peripheral stem cell or bone marrow transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy. Giving iodine I 131 metaiodobenzylguanidine and combination chemotherapy with an autologous peripheral stem cell or bone marrow transplant may allow more chemotherapy to be given so that more tumor cells are killed. Giving radiation therapy after an autologous peripheral stem cell or bone marrow transplant may kill any remaining tumor cells.
PURPOSE: This phase II trial is studying how well giving iodine I 131 metaiodobenzylguanidine together with combination chemotherapy and radiation therapy works in treating patients who are undergoing an autologous peripheral stem cell or bone marrow transplant for relapsed or refractory neuroblastoma.
Detailed description
OBJECTIVES:
Primary
- Determine the response rate in patients with relapsed or refractory neuroblastoma treated with iodine I 131 metaiodobenzylguanidine (^131I-MIBG) and combination chemotherapy comprising carboplatin, etoposide, and melphalan followed by autologous bone marrow or peripheral blood stem cell transplantation and radiotherapy.
Secondary
- Determine the hematopoietic and nonhematopoietic toxicity of this regimen in these patients.
- Determine the tumor self-absorbed radiation dose (TSARD) in patients with measurable soft tissue lesions treated with this regimen.
- Correlate the TSARD with tumor response in patients with measurable residual soft tissue disease treated with this regimen.
OUTLINE: This is a multicenter study. Patients are stratified according to risk (poor-risk group [mixed or no response to induction therapy or progression during or after induction therapy] vs good-risk group [partial response after 4 courses of induction therapy]) and kidney function at study entry (glomerular filtration rate [GFR] ≥ 100 mL/min vs GFR 60-99 mL/min)
- Stem cell harvest: Patients undergo a peripheral blood stem cell harvest or bone marrow harvest provided they have an adequate number of cells available. At least 2 weeks later, patients proceed to iodine I 131 metaiodobenzylguanidine (^131I-MIBG) and combination chemotherapy.
- 131I-MIBG and combination chemotherapy: Patients receive ^131I-MIBG IV over 2 hours on day -21, carboplatin IV continuously on days -7 to -4, etoposide IV continuously on days -7 to -4, and melphalan IV over 1 hour on days -7 to -5.
- Stem cell infusion and filgrastim (G-CSF): Three days after completion of chemotherapy, patients undergo transplantation of either stem cells or bone marrow on day 0. Patients also receive G-CSF subcutaneously or IV over 1 hour once daily beginning on day 0 and continuing until blood counts return to normal.
- Radiotherapy: Once blood counts return to normal, patients undergo radiotherapy to primary and metastatic sites that have not received previous irradiation over 12 days beginning after day 42.
After completion of study treatment, patients are followed for 2 years and then periodically thereafter.
PROJECTED ACCRUAL: Approximately 50 patients (40 low-risk patients and 8-10 high-risk patients) will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All the patients enrolled in the study | Experimental | Biological: Filgrastim, Drug: Carboplatin, Drug: Etoposide, Drug: Melphalan, Procedure: Peripheral blood stem cell infusion, Radiation: 131I-MIBG, Radiation: Radiation therapyThis is a single arm study. The following description applies to all the patients who are enrolled in the study: On Day -21, patients receive 131I-MIBG infusion. On Day -7, Day -6, Day -5, patients receive Carboplatin, Etoposide, Melphalan. On Day -4, patients receive Carboplatin, Etoposide. On Day -3, Day -2, Day -1, patients rest. On Day 0, patients receive peripheral blood stem cell infusion. Dosing of Carboplatin, Etoposide and Melphalan is based upon whole body dosimetry (cGy) estimates. Filgrastim 5 micrograms/kg/day S.C. or IV will be given daily beginning on Day 0. Local radiation therapy is to be given to previously non-irradiated primary and metastatic sites of disease. |
Who may qualify
- Ages
- 1 year to 29 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
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 |
|---|---|---|
| FilgrastimAlso: G-CSFFilgrastim 5 micrograms/kg/day S.C. or IV will be given daily beginning on Day 0. The first dose should begin four hours after the stem cell infusion is completed. Filgrastim will continue daily until the ANC >=1500/uL for three consecutive days. | Biological | All the patients enrolled in the study |
| CarboplatinAlso: cis-diammine; Paraplatin; Paraplatin-AQThe carboplatin will be administered as a continuous IV infusion Day - 7 through Day - 4, with dosing based upon pretreatment GFR levels. The carboplatin should be diluted to a concentration of 0.3 mg/ml in D5W 0.45NS and infused concomitantly with etoposide through the same central venous catheter using a "Y" connector; a controlled rate infusion pump is used for each arm of the "Y". | Drug | All the patients enrolled in the study |
| EtoposideAlso: VP-16; EtopophosThe etoposide shall be administered day -7 through day -4 via continuous intravenous infusion over 96 hours. For patients with a corrected GFR >= 100 ml/min/1.72 m^2, a dose of 300 mg/m^2/day (10 mg/kg/day if child is < 12 kg) shall be given. For patients with a corrected GFR 60-99 ml/min/1.72 m^2, the etoposide will be administered at a dose of 160 mg/m^2/day (5.3 mg/kg/day). The etoposide will be diluted in D5W 0.45%NS at a concentration of < 0.4 mg/ml. Etoposide should not be mixed with carboplatin, but administered using a Y-connector. | Drug | All the patients enrolled in the study |
| MelphalanAlso: L-phenylalanine mustard; L-PAM; Alkeran; SarcolysinFor patients in either the normal GFR strata (>=100 ml/min/1.73 m^2), or reduced GFR strata (60-99 ml/min/1.73m^2), melphalan shall be administered at a dose of 60 mg/m^2/day (2 mg/kg/day if child is < 12 kg) on day -7, -6, and -5 of study. The melphalan should be infused at a rate of less than 10 mg/minute, and should complete within 1 hour of reconstitution each day. The melphalan should be diluted in 0.9% NaCl at a concentration < 2 mg/ml. The total dosage of melphalan to be administered will be 180 mg/m^2. | Drug | All the patients enrolled in the study |
| Peripheral blood stem cell infusionStem cells or marrow will be infused on day 0 of study therapy. Where the DMSO concentration in the stem cell product would exceed accepted level for infusion within a 24 hour period, stem cell products may be infused over two days to meet this standard. For purged PBSC: A minimum of 2.0 x 10^6 viable CD34+ cells/kg must be available. For unpurged PBSC, a minimum of 2.0 x 10^6 viable CD34+ cells/kg must be available. Having a back-up of 2.0 x 10^6 viable CD34+ cells/kg purged or unpurged PBSC is recommended but not required. For purged bone marrow, a minimum of 1.5 x10^8 mononuclear cells/kg must be available. | Procedure or surgery | All the patients enrolled in the study |
| 131I-MIBGAlso: Iobenguane I 131Therapeutic 131I MIBG will be synthesized by Draximage Canada.with specific activity between 15 and 25mCi/ml. Radiopurity will be initially determined by Draximage, prior to shipment to participating centers. Free radioiodide content must then be rechecked at the treating center prior to infusion using HPLC or Sep-Pac methodology. | Radiation | All the patients enrolled in the study |
| Radiation therapyLocal irradiation is to be given to previously non-irradiated primary and metastatic sites of disease. Local irradiation should not start till the patient is medically stable, has an ANC > 1000/uL, platelets > 30,000 / uL, and is > 42 days post transplant. Recommended radiation guidelines consist of 2160 cGy total, given over 12 days using a single 180 cGy fraction/day. Any delay in local radiation that would extend treatment beyond day +84 should be discussed with the study chair. Local radiation will be administered at a participating NANT member site. | Radiation | All the patients enrolled in the study |
Primary outcome
Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion
Time frame: Response assessed 60 days post stem cell infusion
Tumor response based on evaluation performed on day 60 or at the time of disease progression/recurrence or start of another treatment - whichever comes first. Such evaluations will include 123I-MIBG scan, CT/MRI, urine catecholamine measurement, and bone marrow analysis (for those with marrow disease at study entry).
3 secondary outcomes
Event-free Survival (EFS) at 3 Years
Time frame: 3 years since start of treatment
Engraftment DLT
Time frame: From treatment start until 60 days post stem cell infusion
Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS
Time frame: Between start of MIBG treatment and 60 days post stem cell infusion
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Gregory Yanik, MD study chair
University of Michigan Rogel Cancer Center
Katherine K. Matthay, MD principal investigator
University of California, San Francisco
John M. Maris, MD principal investigator
Children's Hospital of Philadelphia
15 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Filgrastim, Carboplatin and Etoposide in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 15 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 28 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 15 sites in United States, Canada. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Aug 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion (time frame: Response assessed 60 days post stem cell infusion). 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 28 Apr 2026.