Back to results

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

Primary Purpose

Neuroblastoma

Status

Recruiting

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Thiotepa

Cyclophosphamide

Melphalan

Etoposide

Carboplatin

Autologous Stem Cell Infusion

Granulocyte colony stimulating factor

About this trial

This is an interventional treatment trial for Neuroblastoma focused on measuring High-risk neuroblastoma, Neuroblastoma, Myeloablative chemotherapy, Myeloablative consolidation chemotherapy, Autologous peripheral blood stem cell rescue, PBSC, Autologous stem cell rescue

Eligibility Criteria

undefined - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Masonic Cancer Center, University of MinnesotaRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Patients Treated for Neuroblastoma

Arm 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.

Outcomes

Primary Outcome Measures

Progression Free Survival

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

Secondary Outcome Measures

Time to Engraftment

Defined 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.

Relapse

Percentage 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 Survival

Kaplan-Meier curves and 95% confidence intervals will be used to estimate overall survival.

Full Information

NCT ID

NCT02605421

First Posted

November 12, 2015

Last Updated

October 2, 2023

Sponsor

Masonic Cancer Center, University of Minnesota

1. Study Identification

Unique Protocol Identification Number

NCT02605421

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Recruiting

Study Start Date

June 2016 (Actual)

Primary Completion Date

July 2024 (Anticipated)

Study Completion Date

July 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Masonic Cancer Center, University of Minnesota

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Neuroblastoma

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Patients Treated for Neuroblastoma

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Thiotepa

Intervention Description

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

Intervention Name(s)

Cyclophosphamide

Intervention Description

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

Intervention Name(s)

Melphalan

Intervention Description

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

Intervention Name(s)

Etoposide

Intervention Description

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

Intervention Name(s)

Carboplatin

Intervention Description

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

Biological

Intervention Name(s)

Autologous Stem Cell Infusion

Intervention Description

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

Intervention Type

Biological

Intervention Name(s)

Granulocyte colony stimulating factor

Other Intervention Name(s)

G-CSF

Intervention Description

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 Information:

Title

Progression Free Survival

Description

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

Time Frame

3 years from first PBSC infusion

Secondary Outcome Measure Information:

Title

Time to Engraftment

Description

Defined 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.

Time Frame

Day 42

Title

Relapse

Description

Percentage of patients with relapse. Relapse will be defined as any new lesion; increase of any measurable lesion by >25%; previous negative bone is positive.

Time Frame

3 years from first PBSC infusion

Title

Overall Survival

Description

Kaplan-Meier curves and 95% confidence intervals will be used to estimate overall survival.

Time Frame

3 years from first PBSC infusion

10. Eligibility

Sex

All

Maximum Age & Unit of Time

30 Years

Accepts Healthy Volunteers

Eligibility 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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Lisa Burke

Phone

612-273-8482

lburke3@Fairview.org

First Name & Middle Initial & Last Name or Official Title & Degree

Ashish Gupta, MBBS, MPH

Phone

612-626-2961

stef0030@umn.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Ashish Gupta, MBBS, MPH

Organizational Affiliation

Masonic Cancer Center, University of Minnesota

Official's Role

Principal Investigator

Facility Information:

Facility Name

Masonic Cancer Center, University of Minnesota

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55455

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Lisa Burke, RN

Phone

612-273-8482

lburke3@Fairview.org

First Name & Middle Initial & Last Name & Degree

Ashish Gupta, MBBS, MPH

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs