Status:
COMPLETED
Busulfan, Melphalan, and Stem Cell Transplant After Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma
Lead Sponsor:
Children's Oncology Group
Collaborating Sponsors:
National Cancer Institute (NCI)
Conditions:
Ganglioneuroblastoma
Stage 1 Neuroblastoma
Eligibility:
All Genders
Up to 30 years
Phase:
PHASE1
Brief Summary
This pilot clinical trial studies busulfan, melphalan, and stem cell transplant after chemotherapy in treating patients with newly diagnosed neuroblastoma that is likely to come back or spread. Giving...
Detailed Description
PRIMARY OBJECTIVES: I. To determine if the acute toxicity of an autologous stem cell transplant with a busulfan-melphalan (BuMel) based regimen is tolerable when given as consolidation therapy for hi...
Eligibility Criteria
Inclusion
- Patients must have a diagnosis of neuroblastoma (International Classification of Diseases for Oncology \[ICD-O\] morphology 9500/3) or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; patients with the following disease stages at diagnosis are eligible, if they meet the other specified criteria
- Patients with newly diagnosed neuroblastoma with International Neuroblastoma Staging System (INSS) stage 4 are eligible with the following:
- V-myc avian myelocytomatosis viral oncogene neuroblastoma derived homolog (MYCN) amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features or
- Age \> 18 months (\> 547 days) regardless of biologic features or
- Age 12-18 months (365-547 days) with any of the following 3 unfavorable biologic features (MYCN amplification, unfavorable pathology and/or deoxyribonucleic acid \[DNA\] index = 1) or any biologic feature that is indeterminate/unsatisfactory/unknown
- Patients with newly diagnosed neuroblastoma with INSS stage 3 are eligible with the following:
- MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features or
- Age \> 18 months (\> 547 days) with unfavorable pathology, regardless of MYCN status
- Patients with newly diagnosed neuroblastoma with INSS stage 2A/2B with MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
- Patients with newly diagnosed neuroblastoma with INSS stage 4S with MYCN amplification (\> 4-fold increase in MYCN expression signals as compared to reference signals), regardless of additional biologic features
- Patients \>= 365 days initially diagnosed with neuroblastoma INSS stage 1, 2, 4S who progressed to a stage 4 without interval chemotherapy; these patients must have been enrolled on ANBL00B1; study enrollment on ANBL12P1 must occur within 4 weeks of progression to stage 4 for INSS stage 1, 2, 4S
- Patients must not have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (as per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification status and histology
- Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/gender as follows:
- Age 1 month to \< 6 months: 0.4 mg/dL
- Age 6 months to \< 1 year: 0.5 mg/dL
- Age 1 to \< 2 years: 0.6 mg/dL
- Age 2 to \< 6 years: 0.8 mg/dL
- Age 6 to \< 10 years: 1 mg/dL
- Age 10 to \< 13 years: 1.2 mg/dL
- Age 13 to \< 16 years: 1.5 mg/dL (males), 1.4 mg/dL (females)
- Age \>= 16 years: 1.7 mg/dL (males), 1.4 mg/dL (females)
- Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age, and
- Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase \[AST\]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 10 x ULN for age
- Shortening fraction of \>= 27% by echocardiogram, or
- Ejection fraction of \>= 50% by radionuclide evaluation
- No known contraindication to peripheral blood stem cell (PBSC) collection; examples of contraindications might be a weight or size less than that determined to be feasible at the collecting institution, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure
- All patients and/or their parents or legal guardians must sign a written informed consent
- All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion
- Patients that are 12-18 months of age with INSS stage 4 and all 3 favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index \> 1) are not eligible
- Female patients who are pregnant are ineligible
- Lactating females are not eligible unless they have agreed not to breastfeed their infants
- Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained
- Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation
Key Trial Info
Start Date :
April 9 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 30 2024
Estimated Enrollment :
150 Patients enrolled
Trial Details
Trial ID
NCT01798004
Start Date
April 9 2013
End Date
June 30 2024
Last Update
July 15 2024
Active Locations (123)
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1
Children's Hospital of Alabama
Birmingham, Alabama, United States, 35233
2
Kaiser Permanente Downey Medical Center
Downey, California, United States, 90242
3
City of Hope Comprehensive Cancer Center
Duarte, California, United States, 91010
4
Loma Linda University Medical Center
Loma Linda, California, United States, 92354