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

Tandem Myeloablative Consolidation Therapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma

Status
Recruiting
Phases
Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT02605421
Enrollment
12
Registered
2015-11-16
Start date
2016-06-30
Completion date
2026-12-31
Last updated
2025-10-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neuroblastoma

Keywords

High-risk neuroblastoma, Neuroblastoma, Myeloablative chemotherapy, Myeloablative consolidation chemotherapy, Autologous peripheral blood stem cell rescue, PBSC, Autologous stem cell rescue

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.

Interventions

DRUGThiotepa

Thiotepa by IV once daily for 3 doses on Days -7, -6 and -5. Given as part of Consolidation Course #1 along with Cyclophosphamide.

DRUGCyclophosphamide

Cyclophosphamide by IV once daily for 4 doses on Days -5, -4, -3 and -2. Given as part of Consolidation Course #1 along with Thiotepa.

DRUGMelphalan

Melphalan by IV once daily for 3 doses on Days -8, -7, and -6. Given as part of Consolidation Course #2 along with Etoposide and Carboplatin.

DRUGEtoposide

Etoposide by IV once daily for 4 doses on Days -8, -7, -6 and -5. Given as part of Consolidation Course #2 along with Melphalan and Carboplatin.

DRUGCarboplatin

Carboplatin by IV once daily for 4 doses on Days -8, -7, -6 and -5. Given as part of Consolidation Course #2 along with Etoposide and Melphalan.

On Day 0 the stem cells will be infused immediately after thawing over 15-60 minutes per institutional guidelines.

Beginning on day 0 after infusion of the PBSC, patients will receive G-CSF SQ or IV (SQ preferred) 5 micrograms/kg once daily and continuing once daily until post-nadir ANC \> 2000/μL for 3 consecutive days.

Sponsors

Masonic Cancer Center, University of Minnesota
Lead SponsorOTHER

Study design

Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
No minimum to 30 Years
Healthy volunteers
No

Inclusion 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

Design outcomes

Primary

MeasureTime frameDescription
Progression Free Survival3 years from first PBSC infusionPercentage of patients with progression free survial. Kaplan-Meier curves and 95% confidence intervals will be used to estimate.

Secondary

MeasureTime frameDescription
Time to EngraftmentDay 42Defined 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.
Relapse3 years from first PBSC infusionPercentage of patients with relapse. Relapse will be defined as any new lesion; increase of any measurable lesion by \>25%; previous negative bone is positive.
Overall Survival3 years from first PBSC infusionKaplan-Meier curves and 95% confidence intervals will be used to estimate overall survival.

Countries

United States

Contacts

Primary ContactLisa Burke
lburke3@Fairview.org612-273-8482
Backup ContactAshish Gupta, MBBS, MPH
stef0030@umn.edu612-626-2961

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026