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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified October 2009 by Samsung Medical Center.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Samsung Medical Center
ClinicalTrials.gov Identifier:
NCT00793845
First received: November 17, 2008
Last updated: October 11, 2009
Last verified: October 2009
  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
Procedure: tandem high-dose chemotherapy
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 40
Study Start Date: August 2008
Estimated Study Completion Date: July 2012
Estimated Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
Intervention Details:
    Procedure: tandem high-dose chemotherapy
    • Conventional chemotherapy (6 cycles) -> surgery -> conventional chemotherapy (3 cycles) -> first HDCT/ASCR -> second HDCT/ASCR -> Local RT + Retinoic acid + IL-2
    • First HDCT/ASCR: CEC regimen
    • Second HDCT/ASCR:

    TM-TBI (in case of stage 4 neuroblastoma) or TM regimen (in case of stage 3 neuroblastoma)

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

Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00793845

Contacts
Contact: Ki Woong Sung 82-2-3410-3529 kwsped@skku.edu

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

No publications provided

Responsible Party: Ki Woong Sung, Associate Professor, Samsung Medical Center
ClinicalTrials.gov Identifier: NCT00793845     History of Changes
Other Study ID Numbers: 2008-07-002
Study First Received: November 17, 2008
Last Updated: October 11, 2009
Health Authority: Korea: Institutional Review Board

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

ClinicalTrials.gov processed this record on September 29, 2014