High Dose Chemotherapy and Autologous Transplant for Neuroblastoma
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.
Biological: Autologous stem cell infusion
Biological: Granulocyte colony stimulating factor
Radiation: Radiation therapy
Drug: Isotretinoin (13-cis-retinoic acid)
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Rescue for Neuroblastoma: Standard of Care Considerations|
- Number of Patients with Successful Engraftment [ Time Frame: Day 42 ] [ Designated as safety issue: No ]The time to neutrophil engraftment will be assessed by standard statistical approaches.
- Number of Patients with Disease Free Survival [ Time Frame: 2 Years ] [ Designated as safety issue: No ]The number of patients alive and disease free will be assessed using standard statistical approaches.
- Overall Survival [ Time Frame: 2 Years ] [ Designated as safety issue: No ]The number of patients alive will be assessed by standard statistical approaches.
- Number of Patients with Treatment Related Death [ Time Frame: 1 Year ] [ Designated as safety issue: Yes ]The rate of treatment related mortality will be assessed by cumulative incidence approach.
- Number of Patients with Disease Free Survival [ Time Frame: 5 Years ] [ Designated as safety issue: No ]The number of patients alive and disease free will be assessed using standard statistical approaches.
- Overall Survival [ Time Frame: 5 Years ] [ Designated as safety issue: No ]The number of patients alive will be assessed by standard statistical approaches.
|Study Start Date:||March 2012|
|Estimated Study Completion Date:||February 2018|
|Estimated Primary Completion Date:||February 2017 (Final data collection date for primary outcome measure)|
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.
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: ParaplatinBiological: 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-CSFRadiation: 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: AccutaneDrug: 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: AlkeranDrug: 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
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01526603
|Contact: Patricia Kleinkefirstname.lastname@example.org|
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Michael R. Verneris, M.D. 612-626-2961 email@example.com|
|Principal Investigator: Michael R. Verneris, M.D.|
|Principal Investigator:||Michael R. Verneris, M.D.||Masonic Cancer Center, University of Minnesota|