Recruiting
Phase 2

Myeloablative Consolidation Therapy & Tandem Autologous Stem Cell Rescue

Sponsor:

Masonic Cancer Center, University of Minnesota

Code:

NCT02605421

Conditions

Neuroblastoma

Eligibility Criteria

Sex: All

Age: 0 - 30

Healthy Volunteers: Not accepted

Interventions

Thiotepa

Cyclophosphamide

Melphalan

Etoposide

Carboplatin

Study Details

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

Study ID

NCT02605421

Start date

Jun, 2016

Status verified date

Oct, 2025

Completion date

Dec, 2026

Anticipated

Primary completion date

Oct 1, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 30

Healthy Volunteers: Not accepted

  • 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

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Patients Treated for Neuroblastoma

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.

Interventions

Thiotepa

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

Cyclophosphamide

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.

Melphalan

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.

Etoposide

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.

Carboplatin

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.

Autologous Stem Cell Infusion

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

Granulocyte colony stimulating factor

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.

Primary outcome measure

  • Progression Free Survival [ Time Frame: 3 years from first PBSC infusion ]

Central Contacts and Locations

Central contacts

Lisa Burke

612-273-8482lburke3@Fairview.org

Ashish Gupta, MBBS, MPH

612-626-2961stef0030@umn.edu

Locations

Masonic Cancer Center, University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55455

Contacts

Lisa Burke, RN

612-273-8482lburke3@Fairview.org

Principal Investigator:

Ashish Gupta, MBBS, MPH

More Information

Sponsor

Masonic Cancer Center, University of Minnesota

Last update posted

Oct 21, 2025

Last verified

Oct, 2025

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Masonic Cancer Center, University of Minnesota on 2025-10-21.