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Neuroblastoma | High Dose Chemotherapy and Autologous Transplant for Neuroblastoma

Neuroblastoma research study

What is the primary objective of this study?

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.

Who is eligible to participate?

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.

Which medical condition, disease, disorder, syndrome, illness, or injury is researched?

Neuroblastoma

Study Interventions

Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.

Drug:CarboplatinCarboplatin 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.

Biological:Autologous stem cell infusionOn day 0 the stem cells will be infused immediately after thawing over 15-60 minutes per institutional guidelines.

Biological:Granulocyte colony stimulating factorBeginning 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.

Radiation:Radiation therapyIt 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.

Drug:MelphalanMelphalan 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

Drug:EtoposideEtoposide 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

Study Arms

Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.

Patients Treated for NeuroblastomaAccording 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.

Study Status

Recruiting

Start Date: March 28, 2012

Completed Date: February 2020

Phase: N/A

Type: Interventional

Design:

Primary Outcome: Number of Patients with Successful Engraftment

Secondary Outcome: Number of Patients with Disease Free Survival

Study sponsors, principal investigator, and references

Principal Investigator: Heather Stefanski, MD

Lead Sponsor: Masonic Cancer Center, University of Minnesota

Collaborator:

More information:https://clinicaltrials.gov/show/NCT01526603

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