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High-Dose Iodine I 131 Metaiodobenzylguanidine, Topotecan, and Peripheral Stem Cell Transplant in Treating Young Patients With Relapsed Stage 4 Neuroblastoma or Primary Resistant High-Risk Neuroblastoma

This study has been withdrawn prior to enrollment.
(Withdrawn because protocol has been discontinued. It was never opened.)
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: October 18, 2006
Last updated: July 9, 2013
Last verified: June 2007

RATIONALE: Radioisotope therapy, such as iodine I 131 metaiodobenzylguanidine (MIBG), releases radiation that kills tumor cells. Drugs used in chemotherapy, such as topotecan, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Topotecan may also make tumor cells more sensitive to iodine I 131 MIBG. A peripheral stem cell transplant may be able to replace blood-forming cells that were destroyed by iodine I 131 MIBG and topotecan. This may allow more iodine I 131 MIBG and topotecan to be given so that more tumor cells are killed.

PURPOSE: This phase II trial is studying how well giving high-dose iodine I 131 MIBG together with topotecan and peripheral stem cell transplant works in treating young patients with relapsed stage 4 neuroblastoma or primary resistant high-risk neuroblastoma.

Condition Intervention Phase
Neuroblastoma Drug: iodine I 131 metaiodobenzylguanidine Drug: topotecan hydrochloride Procedure: chemotherapy Procedure: peripheral blood stem cell transplantation Procedure: radioisotope therapy Procedure: radionuclide imaging Procedure: radiosensitization Procedure: total-body irradiation Phase 2

Study Type: Interventional
Study Design: Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: International Phase II Studies of I-mIBG in Combination With Topotecan and Peripheral Blood Stem Cell Rescue for (A) Primary Resistant High Risk Neuroblastoma and (B) Relapsed Stage 4 Neuroblastoma

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Proportion of patients who respond to treatment (partial response and complete response at metastatic sites) as measured by metaiodobenzylguanidine scintigraphy and positron emission tomography and CT imaging
  • Proportion of patients who are able to progress to potentially curative treatment with surgery and further systemic treatment
  • Correlation of tumor dosimetry with response
  • Time to tumor progression

Enrollment: 0
Study Start Date: July 2008
Estimated Primary Completion Date: July 2008 (Final data collection date for primary outcome measure)
Detailed Description:


  • Determine response (partial and complete response at metastatic sites) in children with relapsed stage 4 neuroblastoma or primary resistant high-risk neuroblastoma treated with high-dose iodine I 131 metaiodobenzylguanidine, topotecan hydrochloride, and peripheral blood stem cell transplantation.
  • Determine the proportion of patients who, as a result of this treatment, are able to progress to potentially curative surgery and further systemic treatment.
  • Correlate tumor dosimetry (to determine whether the tumor absorbed the radiation dose) with response in patients treated with this regimen.
  • Determine the time to tumor progression.

OUTLINE: This is an open-label, multicenter study. Patients are stratified according to disease type (relapsed stage 4 vs primary resistant high-risk neuroblastoma).

Patients receive topotecan hydrochloride IV over 30 minutes on days 1-5 and 15-19 and high-dose iodine I 131 metaiodobenzylguanidine (^131I-MIBG) IV over 30 minutes on days 1 and 15. Patients receive autologous CD 34+ peripheral blood stem cells when ^131I-MIBG dosimetry levels reach an acceptable low on days 25-29.

Total whole-body absorbed dose is measured periodically after the first ^131I-MIBG dose is administered and periodically thereafter.

After completion of study treatment, patients are followed periodically for up to 10 years.

PROJECTED ACCRUAL: A total of 67 patients will be accrued for this study.


Ages Eligible for Study:   1 Year to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Confirmed diagnosis of neuroblastoma meeting the 1 of the following criteria:

    • Primary resistant high-risk disease meeting the following criteria:

      • International neuroblastoma staging system (INSS) stage 4, or stage 2 or 3 with myelocytomatosis viral-related oncogene (MycN) amplification
      • Failed to achieve satisfactory remission with induction chemotherapy, defined as one of the following:

        • Less than 50% reduction or > 3 positive sites on iodine I 131 metaiodobenzylguanidine (^131I-MIBG) scintigraphy
        • Persistent cytomorphological positive disease in bone marrow aspirates or trephine biopsies
        • Progressive disease necessitating a change of treatment
    • Relapsed stage 4 disease meeting the following criteria:

      • High-risk neuroblastoma (INSS stage 4, or stage 2 or 3 with MycN amplification)
      • Relapsed after intensive treatment including high-dose chemotherapy and hematopoietic progenitor cell support

        • Patients may be entered at the time of relapse, or at any point subsequently after other treatments
  • ^131I-MIBG-positive disease on diagnostic scintigraphy
  • Peripheral blood stem cell harvest ≥ 300,000/mm³ CD 34+ cells
  • Enrolled in or has been treated on protocol SIOP-NB-2009 or a similar protocol


  • Glomerular filtration rate ≥ 50 mL/min
  • Considered fit enough to undergo proposed study treatment


  • See Disease Characteristics
  Contacts and Locations
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Please refer to this study by its identifier: NCT00389766

Sponsors and Collaborators
Children's Cancer and Leukaemia Group
Study Chair: Mark N. Gaze, MD University College London Hospitals
  More Information Identifier: NCT00389766     History of Changes
Other Study ID Numbers: CCLG-NB-2006-08
CDR0000508611 ( Registry Identifier: PDQ (Physician Data Query) )
Study First Received: October 18, 2006
Last Updated: July 9, 2013

Keywords provided by National Cancer Institute (NCI):
localized unresectable neuroblastoma
recurrent neuroblastoma
regional neuroblastoma
localized resectable neuroblastoma
disseminated neuroblastoma

Additional relevant MeSH terms:
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Anti-Infective Agents, Local
Anti-Infective Agents
Trace Elements
Growth Substances
Physiological Effects of Drugs
Topoisomerase I Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Radiopharmaceuticals processed this record on September 21, 2017