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

NCT ID: NCT02605421

Last Updated: 2025-10-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-06-30

Study Completion Date

2026-12-31

Brief Summary

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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

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Conditions

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Neuroblastoma

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Thiotepa

Intervention Type 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.

Cyclophosphamide

Intervention Type 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.

Melphalan

Intervention Type 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.

Etoposide

Intervention Type 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.

Carboplatin

Intervention Type 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.

Autologous Stem Cell Infusion

Intervention Type BIOLOGICAL

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

Granulocyte colony stimulating factor

Intervention Type 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.

Interventions

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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.

Intervention Type DRUG

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.

Intervention Type DRUG

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.

Intervention Type DRUG

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.

Intervention Type DRUG

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.

Intervention Type DRUG

Autologous Stem Cell Infusion

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

Intervention Type BIOLOGICAL

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.

Intervention Type BIOLOGICAL

Other Intervention Names

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G-CSF

Eligibility Criteria

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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
Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ashish Gupta, MBBS, MPH

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

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Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Lisa Burke

Role: CONTACT

612-273-8482

Ashish Gupta, MBBS, MPH

Role: CONTACT

612-626-2961

Facility Contacts

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Lisa Burke, RN

Role: primary

612-273-8482

Other Identifiers

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2015LS108

Identifier Type: -

Identifier Source: org_study_id

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