Busulfan, Melphalan, and Stem Cell Transplant After Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma

Completed · Phase 1

Conditions studied: Ganglioneuroblastoma, Stage 1 Neuroblastoma, Stage 2 Neuroblastoma, Stage 2A Neuroblastoma, Stage 2B Neuroblastoma, Stage 3 Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma

In brief

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 chemotherapy to the entire body before a stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood and stored. More chemotherapy or radiation therapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

Key facts

Study ID: NCT01798004
Run by: Children's Oncology Group
People needed: 150
Starts: 2013-04-09
Expected to finish: 2024-06-30
Last updated by the study team: 2024-07-15

Who can join

Age: any, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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

Where it is running

Kaiser Permanente Downey Medical Center — Downey, California, United States
City of Hope Comprehensive Cancer Center — Duarte, California, United States
Loma Linda University Medical Center — Loma Linda, California, United States
Children's Hospital Los Angeles — Los Angeles, California, United States
UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
Kaiser Permanente-Oakland — Oakland, California, United States
Children's Hospital of Orange County — Orange, California, United States
Lucile Packard Children's Hospital Stanford University — Palo Alto, California, United States
University of California Davis Comprehensive Cancer Center — Sacramento, California, United States
Rady Children's Hospital - San Diego — San Diego, California, United States
UCSF Medical Center-Parnassus — San Francisco, California, United States
UCSF Medical Center-Mission Bay — San Francisco, California, United States
Children's Hospital Colorado — Aurora, Colorado, United States
Connecticut Children's Medical Center — Hartford, Connecticut, United States
Alfred I duPont Hospital for Children — Wilmington, Delaware, United States
MedStar Georgetown University Hospital — Washington D.C., District of Columbia, United States
Children's National Medical Center — Washington D.C., District of Columbia, United States
Lee Memorial Health System — Fort Myers, Florida, United States
Golisano Children's Hospital of Southwest Florida — Fort Myers, Florida, United States
University of Florida Health Science Center - Gainesville — Gainesville, Florida, United States
Memorial Regional Hospital/Joe DiMaggio Children's Hospital — Hollywood, Florida, United States
Nemours Children's Clinic-Jacksonville — Jacksonville, Florida, United States
University of Miami Miller School of Medicine-Sylvester Cancer Center — Miami, Florida, United States
Miami Cancer Institute — Miami, Florida, United States
Children's Hospital of Alabama — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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