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High Dose Chemotherapy and Autologous Transplant for Neuroblastoma

Primary Purpose

Neuroblastoma

Status

Recruiting

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

Carboplatin

Autologous stem cell infusion

Granulocyte colony stimulating factor

Radiation therapy

Isotretinoin (13-cis-retinoic acid)

Melphalan

Etoposide

About this trial

This is an interventional treatment trial for Neuroblastoma focused on measuring peripheral blood stem cell transplantation, autologous stem cell transplant

Eligibility Criteria

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

Inclusion Criteria:

Less than 30 years of age at diagnosis of neuroblastoma
No evidence of disease progression: defined as increase in tumor size of >25% or new lesions
Recovery from last induction course of chemotherapy (absolute neutrophil count > 500 and platelet > 20,000)
No uncontrolled infection
Minimum frozen peripheral blood stem cells (PBSCs) of 2 x 10^6 CD34 cells/kg for transplant are mandatory and 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of 4 x 106 CD34 cells/kg is encouraged)
Adequate organ function defined as:
- Hepatic: aspartate aminotransferase (AST) < 3 x upper limit of institutional normal 8 Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 50%, no clinical congestive heart failure 8 Renal: Creatinine clearance or glomerular filtration rate (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

Exclusion Criteria

Patients with progressive disease should consider participating in phase I studies since consolidation therapy using the regimen outlined in this document have not been determined to be useful.
Patients who are delayed in consolidation chemotherapy beyond 8 weeks, and don't meet organ function criteria.

Sites / Locations

Masonic Cancer Center, University of MinnesotaRecruiting

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Patients Treated for Neuroblastoma

Arm Description

According to patient weight and renal function, consolidation chemotherapy using various doses of Melphalan, Etoposide, and Carboplatin followed by autologous stem cell infusion and serial post-transplant Granulocyte Colony Stimulating Factor, radiation therapy and Isotretinoin maintenance therapy.

Outcomes

Primary Outcome Measures

Number of Patients with Successful Engraftment

The time to neutrophil engraftment will be assessed by standard statistical approaches.

Secondary Outcome Measures

Number of Patients with Disease Free Survival

The number of patients alive and disease free will be assessed using standard statistical approaches.

Overall Survival

The number of patients alive will be assessed by standard statistical approaches.

Number of Patients with Treatment Related Death

The rate of treatment related mortality will be assessed by cumulative incidence approach.

Number of Patients with Disease Free Survival

The number of patients alive and disease free will be assessed using standard statistical approaches.

Overall Survival

The number of patients alive will be assessed by standard statistical approaches.

Full Information

NCT ID

NCT01526603

First Posted

January 31, 2012

Last Updated

March 15, 2023

Sponsor

Masonic Cancer Center, University of Minnesota

1. Study Identification

Unique Protocol Identification Number

NCT01526603

Brief Title

High Dose Chemotherapy and Autologous Transplant for Neuroblastoma

Official Title

High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Rescue for Neuroblastoma: Standard of Care Considerations

Study Type

Interventional

2. Study Status

Record Verification Date

March 2023

Overall Recruitment Status

Recruiting

Study Start Date

March 28, 2012 (Actual)

Primary Completion Date

February 2024 (Anticipated)

Study Completion Date

February 2024 (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

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

This is a standard of care document, outlining the therapy for children with high risk neuroblastoma who are not eligible for Children's Oncology Group (COG) studies.

Detailed Description

This therapy involves the use of melphalan, etoposide, and carboplatin (consolidation chemotherapy); autologous stem cell rescue, post-transplant radiation therapy and a maintenance phase with Isotretinoin (Accutane, 13-cis-retinoic acid) therapy. If available, patients should also consider post-transplant therapy with cytokines and monoclonal antibody (ch14.18) on a COG or New Approaches to Neuroblastoma Therapy (NANT) trial.

6. Conditions and Keywords

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

Neuroblastoma

Keywords

peripheral blood stem cell transplantation, autologous stem cell transplant

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Patients Treated for Neuroblastoma

Arm Type

Other

Arm Description

Intervention Type

Drug

Intervention Name(s)

Carboplatin

Other Intervention Name(s)

Paraplatin

Intervention Description

Carboplatin intravenously (IV), 425 mg/m2/dose (or if ≤ 12kg, 14.2 mg/kg/dose) once daily x 4 doses on days 7 through 4 pretransplant.

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 subcutaneously (SQ) or IV (SQ preferred) 5 micrograms/kg once daily and continuing once daily until post-nadir absolute neutrophil count (ANC) > 2000/μL for 3 consecutive days.

Intervention Type

Radiation

Intervention Name(s)

Radiation therapy

Intervention Description

It is suggested that patients who have a complete surgical resection of the primary tumor receive 21.6 Gy external beam radiation therapy (EBRT) to the post-induction chemotherapy, pre-operative primary tumor volume. It is suggested that patients who have an incomplete surgical resection of the primary tumor (residual soft tissue mass measuring >1 cm3) will receive 21.6 Gy EBRT to the postinduction chemotherapy, pre-operative primary tumor volume and an additional boost of 14.4 Gy EBRT to the gross residual tumor (total dose 36 Gy to gross residual tumor volume). Radiation should be given after stem cell transplantation and should start no sooner than 28 days post transplant.

Intervention Type

Drug

Intervention Name(s)

Isotretinoin (13-cis-retinoic acid)

Other Intervention Name(s)

Accutane

Intervention Description

Post-transplant maintenance therapy with cis-RA daily for 14 days every 28 days repeated for 6 months. This phase of the therapy can be initiated by the BMT team and continued by the referring physician. It is recommended to begin Isotretinoin at day 66 post-transplant and no later than day 100. For patients ≤12 kg, isotretinoin (accutane) should be administered at 5.33 mg/kg/dose divided twice daily. For patients >12 kg isotretinoin (accutane) should be administered at 160 mg/m^2/day divided twice a day. Patients should be considered for monoclonal antibody therapy against GD2, such as ch14.18 if such trials are available.

Intervention Type

Drug

Intervention Name(s)

Melphalan

Other Intervention Name(s)

Alkeran

Intervention Description

Melphalan Intravenously (IV), 70 mg/m2/dose (or if ≤ 12 kg, 2.3 mg/kg/dose) once daily x 3 doses on days 7 through 5 pretransplant

Intervention Type

Drug

Intervention Name(s)

Etoposide

Other Intervention Name(s)

Eposin, VP-16

Intervention Description

Etoposide intravenously (IV), 338 mg/m2/dose (or if ≤ 12kg, 11.3 mg/kg/dose) once daily x 4 doses on days 7 through 4 pretransplant

Primary Outcome Measure Information:

Title

Number of Patients with Successful Engraftment

Description

The time to neutrophil engraftment will be assessed by standard statistical approaches.

Time Frame

Day 42

Secondary Outcome Measure Information:

Title

Number of Patients with Disease Free Survival

Description

The number of patients alive and disease free will be assessed using standard statistical approaches.

Time Frame

2 Years

Title

Overall Survival

Description

The number of patients alive will be assessed by standard statistical approaches.

Time Frame

2 Years

Title

Number of Patients with Treatment Related Death

Description

The rate of treatment related mortality will be assessed by cumulative incidence approach.

Time Frame

1 Year

Title

Number of Patients with Disease Free Survival

Description

The number of patients alive and disease free will be assessed using standard statistical approaches.

Time Frame

5 Years

Title

Overall Survival

Description

The number of patients alive will be assessed by standard statistical approaches.

Time Frame

5 Years

10. Eligibility

Sex

All

Maximum Age & Unit of Time

30 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Less than 30 years of age at diagnosis of neuroblastoma No evidence of disease progression: defined as increase in tumor size of >25% or new lesions Recovery from last induction course of chemotherapy (absolute neutrophil count > 500 and platelet > 20,000) No uncontrolled infection Minimum frozen peripheral blood stem cells (PBSCs) of 2 x 10^6 CD34 cells/kg for transplant are mandatory and 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of 4 x 106 CD34 cells/kg is encouraged) Adequate organ function defined as: Hepatic: aspartate aminotransferase (AST) < 3 x upper limit of institutional normal 8 Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 50%, no clinical congestive heart failure 8 Renal: Creatinine clearance or glomerular filtration rate (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 Exclusion Criteria Patients with progressive disease should consider participating in phase I studies since consolidation therapy using the regimen outlined in this document have not been determined to be useful. Patients who are delayed in consolidation chemotherapy beyond 8 weeks, and don't meet organ function criteria.

Central Contact Person:

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

Lisa Burke

Phone

612-273-8482

lburke3@Fairview.org

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

Phone

612-273-8482

lburke3@Fairview.org

12. IPD Sharing Statement

Learn more about this trial

High Dose Chemotherapy and Autologous Transplant for Neuroblastoma

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