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

University of Minnesota (UMN)

Status and phase

Enrolling

Phase 2

Conditions

Neuroblastoma

Treatments

Drug: Thiotepa

Biological: Autologous Stem Cell Infusion

Drug: Carboplatin

Biological: Granulocyte colony stimulating factor

Drug: Etoposide

Drug: Cyclophosphamide

Drug: Melphalan

Study type

Interventional

Funder types

Other

Identifiers

NCT02605421

2015LS108

Details and patient eligibility

About

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.

Enrollment

12 estimated patients

Sex

All

Ages

Under 30 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Less than 30 years of age at diagnosis of neuroblastoma
End of Induction disease evaluation demonstrating CR, PR, MR or SD
Hematopoietic Recovery from last induction course of chemotherapy
No uncontrolled infection
Minimum frozen PBSCs of 2 x 10^6 CD34 cells/kg for each transplant are mandatory and a PBSC of 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of no less than 6 x 10^6 CD34 cells/kg is encouraged). These must all be collected prior to the initiation of consolidation.
Adequate organ function defined as:
- Hepatic: AST and ALT < 3 x upper limit of institutional normal; ALT ≤ 3 x ULN for age; total bilirubin ≤ 1.5 x ULN for age, if baseline was normal, > 1.0 1.5 x baseline if baseline was abnormal
- Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 45%, no clinical congestive heart failure
- Pulmonary: no evidence of dyspnea at rest and norequirement for supplemental oxygen
- Renal: Creatinine clearance or GFR > 60 mL/min/1.73m^2. If a creatinine clearance is performed at end induction and the result is < 100 ml/min/1.73m^2, a GFR must then be performed using a nuclear blood sampling method or iothalamate clearance method. Camera method is NOT allowed as measure of GFR prior to or during Consolidation therapy for patients with GFR or creatinine clearance of < 100 ml/min/1.73m^2
Recovery from acute toxicities of last cycle of induction chemotherapy
Appropriate written consent - adult or parent/guardian if patient is < 18 years of age and minor information sheet if patient is > 8 years of age

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

12 participants in 1 patient group

Patients Treated for Neuroblastoma

Experimental group

Description:

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.