Tandem High Dose Chemotherapy With 131I-MIBG Treatment in High Risk Neuroblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03061656
Recruitment Status : Active, not recruiting
First Posted : February 23, 2017
Last Update Posted : September 18, 2018
Ministry of Health, Republic of Korea
Information provided by (Responsible Party):
Samsung Medical Center

Brief Summary:
The purpose of this study is to evaluate the efficacy and toxicity of tandem HDCT/ASCT including high-dose 131I-metaiodobenzylguanidine (MIBG) treatment. In the present study, a single arm trial of tandem HDCT/ASCT will be carried out.

Condition or disease Intervention/treatment Phase
High Risk Neuroblastoma Drug: Cyclophosphamide Drug: Carboplatin Drug: Etoposide Radiation: 131I-MIBG Drug: Thiotepa Drug: Melphalan Phase 2

Detailed Description:
Although the outcome of high-risk neuroblastoma has improved after the introduction of HDCT/ASCT, the outcome was still unsatisfactory with 30-40% of survival. We previously reported the results of a single arm prospective trial (SMC NB-2004 study) using tandem HDCT/auto-SCT for high-risk neuroblastoma. In the NB-2004 trial, total body irradiation (TBI) was incorporated in second transplantation. Survival rates were very encouraging; however, short- and long-term toxicities associated with tandem HDCT/auto-SCT, particularly TBI, were also very significant. For this reason, we designed a new prospective trial (SMC NB-2009 study), in which only TBI in the second HDCT/auto-SCT of NB-2004 study was substituted with high-dose 131I-MIBG treatment in order to reduce short- and long-term toxicities without jeopardizing survival rate.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: High-dose 131I-MIBG Treatment Incorporated Into Tandem High-dose Chemotherapy and Autologous Stem Cell Transplantation in Patients With High-risk Neuroblastoma
Actual Study Start Date : January 1, 2009
Actual Primary Completion Date : December 31, 2013
Estimated Study Completion Date : December 31, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma

Arm Intervention/treatment
Experimental: High risk neuroblastoma
  1. Conventional chemotherapy (9 cycles)
  2. Surgery conventional chemotherapy (after 6 cycles of chemotherapy)
  3. Tandem HDCT/autoSCT

    • First HDCT (cyclophosphamide, etoposide, carboplatin)
    • Second HDCT (high-dose 131I-MIBG, thiotepa, melphalan)
  4. Local radiotherapy
  5. Retinoic acid, interleukin-2
Drug: Cyclophosphamide
1st HDCT

Drug: Carboplatin
1st HDCT

Drug: Etoposide
1st HDCT

Radiation: 131I-MIBG
2nd HDCT

Drug: Thiotepa
2nd HDCT

Drug: Melphalan
2nd HDCT

Primary Outcome Measures :
  1. Rate of event free survival [ Time Frame: Up to 5 years ]
    Event is defined as relapse, disease progression or treatment-related mortality.

Secondary Outcome Measures :
  1. Rate of treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: Up to 5 years ]

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with high-risk neuroblastoma

Exclusion Criteria:

  • Patients with progressive disease before high-dose chemotherapy
  • Patients whose parents want to stop or change the planned treatment
  • Patients with organ toxicities of NCI grade >2 before high-dose chemotherapy

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03061656

Sponsors and Collaborators
Samsung Medical Center
Ministry of Health, Republic of Korea
Principal Investigator: Ki Woong Sung Samsung Medical Center

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Samsung Medical Center Identifier: NCT03061656     History of Changes
Other Study ID Numbers: 2011-10-020
First Posted: February 23, 2017    Key Record Dates
Last Update Posted: September 18, 2018
Last Verified: September 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

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
Etoposide phosphate
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antineoplastic Agents, Phytogenic
Topoisomerase II Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors