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Tandem High-dose Chemotherapy and Autologous Stem Cell Rescue in Patients With High-risk Neuroblastoma

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00793845
First Posted: November 19, 2008
Last Update Posted: May 3, 2017
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.
Information provided by (Responsible Party):
Samsung Medical Center
  Purpose
The purpose of this study is to evaluate the efficacy and toxicity of tandem HDCT/ASCR in children with high-risk neuroblastoma. In the present study, a single arm trial of tandem HDCT/ASCR will be carried out. In the present study, the investigators will investigate whether tandem HDCT/ASCR might improve the survival of patients with high-risk neuroblastoma with acceptable toxicity.

Condition Intervention Phase
Neuroblastoma Drug: Cyclophosphamide Drug: Etoposide Drug: Carboplatin Drug: Thiotepa Drug: Melphalan Radiation: Total body irradiation Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Tandem High-dose Chemotherapy and Autologous Stem Cell Rescue in Patients With High-risk Neuroblastoma

Resource links provided by NLM:


Further study details as provided by Samsung Medical Center:

Primary Outcome Measures:
  • Overall survival and event-free survival, short-term and long-term toxicity of tandem high-dose chemotherapy and autologous stem cell transplantation [ Time Frame: from 1 year after second high-dose chemotherapy ]

Estimated Enrollment: 40
Study Start Date: August 2008
Estimated Study Completion Date: January 2018
Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 (total body irradiation, thiotepa, melphalan)
  4. Local radiotherapy
  5. Retinoic acid, interleukin-2
Drug: Cyclophosphamide
First HDCT
Drug: Etoposide
First HDCT
Drug: Carboplatin
First HDCT
Drug: Thiotepa
Second HDCT
Drug: Melphalan
Second HDCT
Radiation: Total body irradiation
Second HDCT

Detailed Description:

The prognosis of high-risk neuroblastoma after conventional chemoradiotherapy is generally poor. Therefore, a strategy using high-dose chemotherapy and autologous stem cell rescue (HDCT/ASCR) has been explored to improve the prognosis of patients with high-risk neuroblastoma. This strategy is based on the hypothesis that dose escalation might improve the survival of children with high-risk neuroblastoma. The results of randomized trials comparing HDCT/ASCR with chemotherapy alone showed a better event-free survival (EFS) in the HDCT/ASCR arm than in the continuous chemotherapy arm. However, the overall EFS was unsatisfactory.

In this context, investigators have examined the efficacy of double or triple tandem HDCT/ASCR to further improve the outcome of high-risk neuroblastoma patients. George et al. carried out a single arm trial of tandem transplantation as consolidation therapy, and reported improved long-term survival (5-year progression-free survival 47%) with acceptable toxicity. Kletzel et al. also conducted a single arm trial of triple tandem transplantation and reported improved survival (3-year EFS 57%). They demonstrated that further dose escalation using sequential HDCT/ASCR might result in further improvements in the survival of patients with high-risk neuroblastoma.

Investigators in the present study also carried out tandem transplantation as consolidation therapy, and reported improved long-term survival (5-year progression-free survival 62%) with acceptable toxicity. However, throughout our previous study, multiple modifications were made in the treatment plan, which resulted in significant variability over time between patients. This variability may create doubt as to whether tandem HDCT/ASCR itself resulted in the improved outcome. In addition, toxic death rate was relatively high (15.4%), although final survival rate was very high (best survival rate ever reported). Therefore, prospective study is needed to evaluate the efficacy and toxicity of tandem HDCT/ASCR.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with high-risk neuroblastoma
  • Patients with intermediate-risk neuroblastoma if gross tumor remained after surgery

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
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT00793845


Locations
Korea, Republic of
Samsung Medical Center
Seoul, Korea, Republic of
Sponsors and Collaborators
Samsung Medical Center
Investigators
Principal Investigator: Ki Woong Sung, MD Samsung Medical Center
  More Information

Responsible Party: Samsung Medical Center
ClinicalTrials.gov Identifier: NCT00793845     History of Changes
Other Study ID Numbers: 2008-07-002
First Submitted: November 17, 2008
First Posted: November 19, 2008
Last Update Posted: May 3, 2017
Last Verified: October 2016

Keywords provided by Samsung Medical Center:
tandem high-dose chemotherapy for high-risk neuroblastoma

Additional relevant MeSH terms:
Neuroblastoma
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Cyclophosphamide
Melphalan
Thiotepa
Etoposide phosphate
Carboplatin
Etoposide
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