Myeloablative Consolidation Therapy and Tandem Autologous Stem Cell Rescue in Patients With High-Risk Neuroblastoma

Phase 2

Recruiting

• Conditions: Neuroblastoma
• Interventions: Drug: Thiotepa; Drug: Cyclophosphamide; Drug: Melphalan; Drug: Etoposide; Drug: Carboplatin; Biological: Autologous Stem Cell Infusion; Biological: Granulocyte colony stimulating factor

• Registration Number: NCT02605421
• Lead Sponsor: Masonic Cancer Center, University of Minnesota

Brief Summary

This is a phase II single center study to administer two courses of myeloablative consolidation chemotherapy each followed by an autologous peripheral blood stem cell (PBSC) rescue in patients with high-risk neuroblastoma who have completed induction chemotherapy (independent of this study). Ideally, patients should begin consolidation chemotherapy no later than 8 weeks after the start of Induction Cycle #5; however it is strongly recommended to begin consolidation within 4-6 weeks after the start of Induction Cycle #5.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-11-12
• Study First Submitted QC: 2015-11-12
• Study First Posted: 2015-11-16
• Study Start: 2016-06
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-17
• Last Update Posted: 2024-07-19
• Primary Completion: 2025-10-01
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Patients Treated for Neuroblastoma	Melphalan	Consolidation course #1 consists of thiotepa and cyclophosphamide followed by a PBSC rescue. Consolidation course #2 consists of melphalan, etoposide and carboplatin followed by a second PBSC rescue. Post infusion, patients will receive Granulocyte-Colony Stimulating Factor beginning on Day 0 of each consolidation course.
Patients Treated for Neuroblastoma	Cyclophosphamide	Consolidation course #1 consists of thiotepa and cyclophosphamide followed by a PBSC rescue. Consolidation course #2 consists of melphalan, etoposide and carboplatin followed by a second PBSC rescue. Post infusion, patients will receive Granulocyte-Colony Stimulating Factor beginning on Day 0 of each consolidation course.
Patients Treated for Neuroblastoma	Granulocyte colony stimulating factor	Consolidation course #1 consists of thiotepa and cyclophosphamide followed by a PBSC rescue. Consolidation course #2 consists of melphalan, etoposide and carboplatin followed by a second PBSC rescue. Post infusion, patients will receive Granulocyte-Colony Stimulating Factor beginning on Day 0 of each consolidation course.
Patients Treated for Neuroblastoma	Autologous Stem Cell Infusion	Consolidation course #1 consists of thiotepa and cyclophosphamide followed by a PBSC rescue. Consolidation course #2 consists of melphalan, etoposide and carboplatin followed by a second PBSC rescue. Post infusion, patients will receive Granulocyte-Colony Stimulating Factor beginning on Day 0 of each consolidation course.
Patients Treated for Neuroblastoma	Thiotepa	Consolidation course #1 consists of thiotepa and cyclophosphamide followed by a PBSC rescue. Consolidation course #2 consists of melphalan, etoposide and carboplatin followed by a second PBSC rescue. Post infusion, patients will receive Granulocyte-Colony Stimulating Factor beginning on Day 0 of each consolidation course.
Patients Treated for Neuroblastoma	Etoposide	Consolidation course #1 consists of thiotepa and cyclophosphamide followed by a PBSC rescue. Consolidation course #2 consists of melphalan, etoposide and carboplatin followed by a second PBSC rescue. Post infusion, patients will receive Granulocyte-Colony Stimulating Factor beginning on Day 0 of each consolidation course.
Patients Treated for Neuroblastoma	Carboplatin	Consolidation course #1 consists of thiotepa and cyclophosphamide followed by a PBSC rescue. Consolidation course #2 consists of melphalan, etoposide and carboplatin followed by a second PBSC rescue. Post infusion, patients will receive Granulocyte-Colony Stimulating Factor beginning on Day 0 of each consolidation course.

Primary Outcome Measures

Name	Time	Method
Progression Free Survival	3 years from first PBSC infusion	Percentage of patients with progression free survial. Kaplan-Meier curves and 95% confidence intervals will be used to estimate.

Secondary Outcome Measures

Name	Time	Method
Time to Engraftment	Day 42	Defined as an absolute neutrophil count (ANC) greater than or equal to 0.5 x 109/L for three consecutive days by day 42 after first transplant.
Overall Survival	3 years from first PBSC infusion	Kaplan-Meier curves and 95% confidence intervals will be used to estimate overall survival.
Relapse	3 years from first PBSC infusion	Percentage of patients with relapse. Relapse will be defined as any new lesion; increase of any measurable lesion by \>25%; previous negative bone is positive.

Trial Locations

Locations (1)

Masonic Cancer Center, University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States