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

NCT02605421 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 2015LS108
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Neuroblastoma

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Progression Free Survival

  Percentage of patients with progression free survial. Kaplan-Meier curves and 95% confidence intervals will be used to estimate.

  3 years from first PBSC infusion

Secondary Outcomes (3)

• Time to Engraftment

  Day 42

• Relapse

  3 years from first PBSC infusion

• Overall Survival

  3 years from first PBSC infusion

Study Arms (1)

Patients Treated for Neuroblastoma

EXPERIMENTAL

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.

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

Interventions

Thiotepa DRUG

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

Patients Treated for Neuroblastoma

Cyclophosphamide DRUG

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.

Patients Treated for Neuroblastoma

Melphalan DRUG

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.

Patients Treated for Neuroblastoma

Etoposide DRUG

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.

Patients Treated for Neuroblastoma

Carboplatin DRUG

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.

Patients Treated for Neuroblastoma

Autologous Stem Cell Infusion BIOLOGICAL

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

Patients Treated for Neuroblastoma

Granulocyte colony stimulating factor BIOLOGICAL

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.

Also known as: G-CSF

Patients Treated for Neuroblastoma

Eligibility Criteria

• Age: Up to 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

* 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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

MeSH Terms

Conditions

Neuroblastoma

Interventions

Thiotepa; Cyclophosphamide; Melphalan; Etoposide; Carboplatin; Granulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Phosphoramides; Organophosphorus Compounds; Organic Chemicals; Triethylenephosphoramide; Aziridines; Azirines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Podophyllotoxin; Tetrahydronaphthalenes; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Polycyclic Compounds; Glucosides; Glycosides; Carbohydrates; Coordination Complexes; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Proteins; Biological Factors

Study Officials

• Ashish Gupta, MBBS, MPH

  Masonic Cancer Center, University of Minnesota

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lisa Burke

CONTACT

612-273-8482; lburke3@Fairview.org

Ashish Gupta, MBBS, MPH

CONTACT

612-626-2961; stef0030@umn.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 12, 2015
• First Posted: November 16, 2015
• Study Start: June 1, 2016
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: October 21, 2025

Record last verified: 2025-10

Locations

US (1)