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

Samsung Medical Center

Status and phase: Unknown; Phase 2

Conditions: High Risk Neuroblastoma

Treatments: Drug: Thiotepa; Drug: Carboplatin; Drug: Etoposide; Drug: Cyclophosphamide; Drug: Melphalan; Radiation: 131I-MIBG

Study type: Interventional

Funder types: Other

Identifiers

NCT03061656

2011-10-020

Details and patient eligibility

About

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.

Full 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.

Enrollment: 40 estimated patients

Sex: All

Ages: Under 21 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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40 participants in 1 patient group

High risk neuroblastoma

Experimental group

Description:

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

Treatment:

Drug: Etoposide

Drug: Cyclophosphamide

Drug: Carboplatin

Radiation: 131I-MIBG

Drug: Melphalan

Drug: Thiotepa