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

Neuroblastoma Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00793845
• Other study ID #: 2008-07-002
• Status: Completed
• Phase: Phase 2
• Start date: August 2008
• Est. completion date: January 2018

Study information

• Verified date: September 2018
• Source: Samsung Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: January 2018
• Est. primary completion date: January 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Neuroblastoma

Intervention

Drug:
• Cyclophosphamide
First HDCT
• Etoposide
First HDCT
• Carboplatin
First HDCT
• Thiotepa
Second HDCT
• Melphalan
Second HDCT

Radiation:
• Total body irradiation
Second HDCT

Locations

Country	Name	City	State
Korea, Republic of	Samsung Medical Center	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
Samsung Medical Center

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall survival and event-free survival, short-term and long-term toxicity of tandem high-dose chemotherapy and autologous stem cell transplantation		from 1 year after second high-dose chemotherapy