Trial · NCT00253435

N2001-02: I-MIBG With Intensive Chemotherapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma

NCT00253435, Phase 2, Completed, New Approaches to Neuroblastoma Therapy Consortium; results first posted 2016-08-24

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. N2001-02: I-MIBG With Intensive Chemotherapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma. Trials, record NCT00253435. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00253435

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: N2001-02: I-MIBG With Intensive Chemotherapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma
NCT number: NCT00253435
Status: Completed
Phases: Phase 2
Enrollment: 50 participants, actual
Lead sponsor: New Approaches to Neuroblastoma Therapy Consortium
Conditions: Neuroblastoma
Interventions: Filgrastim, Carboplatin, Etoposide, Melphalan, Peripheral blood stem cell infusion, 131I-MIBG, Radiation therapy
First posted: 2005-11-15
Last updated: 2026-04-28
Results first posted: 2016-08-24

Source ClinicalTrials.gov

Registered outcomes4

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion	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).
Secondary	Event-free Survival (EFS) at 3 Years	3 years since start of treatment	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.
Secondary	Engraftment DLT	From treatment start until 60 days post stem cell infusion	• 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.
Secondary	Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS	Between start of MIBG treatment and 60 days post stem cell infusion	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

Source ClinicalTrials.gov

Outcomes4

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion	Response assessed 60 days post stem cell infusion	One patient in the Poor Risk Cohort underwent surgery for resection (of his only measureable lesion) prior to post-treatment assessment and is not evaluable for response.	participants	NUMBER
SECONDARY	Event-free Survival (EFS) at 3 Years	3 years since start of treatment	Estimated probability of 3-year progression free survival for all patients enrolled in each risk group.	Estimated probability	NUMBER
SECONDARY	Engraftment DLT	From treatment start until 60 days post stem cell infusion	All patients who began treatment	participants	NUMBER
SECONDARY	Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS	Between start of MIBG treatment and 60 days post stem cell infusion	All patients who began treatment	participants	NUMBER

Source ClinicalTrials.gov

Results by group8

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Units
Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion	Poor Risk Patients	4	participants
Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion	Good Risk Patients	3	participants
Event-free Survival (EFS) at 3 Years	Poor Risk Patients	0.20	Estimated probability
Event-free Survival (EFS) at 3 Years	Good Risk Patients	0.38	Estimated probability
Engraftment DLT	Poor Risk Patients	2	participants
Engraftment DLT	Good Risk Patients	1	participants
Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS	Poor Risk Patients	6	participants
Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS	Good Risk Patients	0	participants

Source ClinicalTrials.gov

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Poor Risk Patients	42
Overall Study	STARTED	Good Risk Patients	8
Overall Study	1st Interim Analysis	Poor Risk Patients	15
Overall Study	1st Interim Analysis	Good Risk Patients	8
Overall Study	2nd Interim Analysis	Poor Risk Patients	10
Overall Study	2nd Interim Analysis	Good Risk Patients	0
Overall Study	3rd Interim Analysis	Poor Risk Patients	10
Overall Study	3rd Interim Analysis	Good Risk Patients	0
Overall Study	COMPLETED	Poor Risk Patients	42
Overall Study	COMPLETED	Good Risk Patients	8

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events528

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Blood/Bone Marrow - Other (DELAYED ENGRAFTMENT ANC)	Blood and lymphatic system disorders	Poor Risk Patients	0	42	0
other	Blood/Bone Marrow - Other (DELAYED ENGRAFTMENT ANC)	Blood and lymphatic system disorders	Good Risk Patients	1	8	1
other	Blood/Bone Marrow - Other (INFUSION OF BACKUP SC FOR DELAYED ENGRAFTMENT)	Blood and lymphatic system disorders	Poor Risk Patients	0	42	0
other	Blood/Bone Marrow - Other (INFUSION OF BACKUP SC FOR DELAYED ENGRAFTMENT)	Blood and lymphatic system disorders	Good Risk Patients	1	8	1
other	Febrile neutropenia (fever of unknown origin)	Blood and lymphatic system disorders	Poor Risk Patients	31	42	31
other	Febrile neutropenia (fever of unknown origin)	Blood and lymphatic system disorders	Good Risk Patients	6	8	6
other	Hemoglobin	Blood and lymphatic system disorders	Poor Risk Patients	40	42	40
other	Hemoglobin	Blood and lymphatic system disorders	Good Risk Patients	7	8	7
other	Hemolysis (e.g., immune hemolytic anemia, drug-related hemolysis)	Blood and lymphatic system disorders	Poor Risk Patients	1	42	1
other	Hemolysis (e.g., immune hemolytic anemia, drug-related hemolysis)	Blood and lymphatic system disorders	Good Risk Patients	0	8	0

1 to 10 of 528Source ClinicalTrials.gov

See all 528 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Poor Risk Patients
Total, all-cause mortality	Good Risk Patients
Total, other adverse events	Poor Risk Patients	42	42
Total, other adverse events	Good Risk Patients	8	8
Total, serious adverse events	Poor Risk Patients	12	42
Total, serious adverse events	Good Risk Patients	3	8

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants1

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
P01CA081403	Biology and Therapy of High Risk Neuroblastoma	SEEGER, ROBERT CHARLES	CHILDREN'S HOSPITAL OF LOS ANGELES	NATIONAL CANCER INSTITUTE	2000 to 2014

Source NIH RePORTER

Trial comparisons4

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Poor Risk Patients vs Good Risk Patients: Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS	Trial comparisons
Poor Risk Patients vs Good Risk Patients: Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion	Trial comparisons
Poor Risk Patients vs Good Risk Patients: Engraftment DLT	Trial comparisons
Poor Risk Patients vs Good Risk Patients: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT00253435

GET https://conductscience.org/api/trials/v1/NCT00253435/events?page=2

API reference For AI assistants