ClinicalTrials.gov
ClinicalTrials.gov Menu

High Dose Chemotherapy and Autologous Transplant for Neuroblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01526603
Recruitment Status : Recruiting
First Posted : February 6, 2012
Last Update Posted : March 23, 2018
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

January 31, 2012
February 6, 2012
March 23, 2018
March 28, 2012
February 2019   (Final data collection date for primary outcome measure)
Number of Patients with Successful Engraftment [ Time Frame: Day 42 ]
The time to neutrophil engraftment will be assessed by standard statistical approaches.
Same as current
Complete list of historical versions of study NCT01526603 on ClinicalTrials.gov Archive Site
  • Number of Patients with Disease Free Survival [ Time Frame: 2 Years ]
    The number of patients alive and disease free will be assessed using standard statistical approaches.
  • Overall Survival [ Time Frame: 2 Years ]
    The number of patients alive will be assessed by standard statistical approaches.
  • Number of Patients with Treatment Related Death [ Time Frame: 1 Year ]
    The rate of treatment related mortality will be assessed by cumulative incidence approach.
  • Number of Patients with Disease Free Survival [ Time Frame: 5 Years ]
    The number of patients alive and disease free will be assessed using standard statistical approaches.
  • Overall Survival [ Time Frame: 5 Years ]
    The number of patients alive will be assessed by standard statistical approaches.
Same as current
Not Provided
Not Provided
 
High Dose Chemotherapy and Autologous Transplant for Neuroblastoma
High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Rescue for Neuroblastoma: Standard of Care Considerations
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.
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.
Interventional
Not Applicable
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Neuroblastoma
  • Drug: Carboplatin
    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.
    Other Name: Paraplatin
  • Biological: Autologous stem cell infusion
    On day 0 the stem cells will be infused immediately after thawing over 15-60 minutes per institutional guidelines.
  • Biological: Granulocyte colony stimulating factor
    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.
    Other Name: G-CSF
  • Radiation: Radiation therapy
    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.
  • Drug: Isotretinoin (13-cis-retinoic acid)
    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.
    Other Name: Accutane
  • Drug: Melphalan
    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
    Other Name: Alkeran
  • Drug: Etoposide
    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
    Other Names:
    • Eposin
    • VP-16
Patients Treated for Neuroblastoma
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.
Interventions:
  • Drug: Carboplatin
  • Biological: Autologous stem cell infusion
  • Biological: Granulocyte colony stimulating factor
  • Radiation: Radiation therapy
  • Drug: Isotretinoin (13-cis-retinoic acid)
  • Drug: Melphalan
  • Drug: Etoposide
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
20
Same as current
February 2020
February 2019   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
up to 30 Years   (Child, Adult)
No
Contact: Kim Nelson 612-273-2925 knelso62@fairview.org
United States
 
 
NCT01526603
2011OC072
MT2011-11C ( Other Identifier: Blood and Marrow Transplantation Program )
No
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Masonic Cancer Center, University of Minnesota
Masonic Cancer Center, University of Minnesota
Not Provided
Principal Investigator: Heather Stefanski, MD Masonic Cancer Center, University of Minnesota
Masonic Cancer Center, University of Minnesota
December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP