Comparing Two Different Myeloablation Therapies in Treating Young Patients Who Are Undergoing a Stem Cell Transplant for High-Risk Neuroblastoma

About this trial

Last updated 4 years ago

Study ID

ANBL0532

Status

Completed

Type

Interventional

Phase

Phase 3

Placebo

Accepting

Up to 30 Years

All Sexes

Trial Timing

Ended 4 years ago

What is this trial about?

This randomized phase III trial compares two different high-dose chemotherapy regimens followed by a stem cell transplant in treating younger patients with high-risk neuroblastoma. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving combination chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving these treatments before a peripheral blood stem cell transplant helps kill any tumor cells that are in the body and helps make room in the patient?s bone marrow for new blood-forming cells (stem cells) to grow. After treatment, stem cells are collected from the patient's blood and stored. High-dose chemotherapy and radiation therapy is then 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 high- chemotherapy. It is not yet known which regimen of high-dose chemotherapy is more effective for patients with high-risk neuroblastoma undergoing a peripheral blood stem cell transplant.

What are the participation requirements?

Inclusion Criteria

* Diagnosis of neuroblastoma or ganglioneuroblastoma by histology or as evidenced by the presence of clumps of tumor cells in bone marrow and elevated catecholamine metabolites in urine meeting any of the following criteria:

* Patients with newly diagnosed neuroblastoma with International Neuroblastoma Staging System (INSS) stage 4 disease are eligible with the following:

* MYCN amplification (i.e., greater than four-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features

* Age > 18 months (i.e., > 547 days) regardless of biologic features

* Age 12-18 months (i.e., 365-547 days) with none of the following three favorable biologic features (i.e., non-amplified MYCN, favorable pathology, and deoxyribonucleic acid [DNA] index > 1)

* Patients with newly diagnosed neuroblastoma with INSS stage 3 are eligible with the following:

* MYCN amplification (i.e., greater than four-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features

* Age > 18 months (i.e., > 547 days) with unfavorable pathology, regardless of MYCN status

* Patients with newly diagnosed INSS stage 2a or 2b with MYCN amplification (i.e., greater than four-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features

* Patients with newly diagnosed INSS stage 4s with MYCN amplification (i.e., greater than four-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features

* Patients >= 365 days initially diagnosed with INSS stage 1, 2, or 4S and who progressed to a stage 4 without interval chemotherapy

* Must have been enrolled on COG-ANBL00B1

* Creatinine clearance or radioisotope glomerular filtration rate ? 70mL/min OR serum creatinine based on age/gender as follows:

* 1 month to < 6 months: 0.4 mg/dL

* 6 months to < 1 year: 0.5 mg/dL

* 1 to < 2 years: 0.6 mg/dL

* 2 to < 6 years: 0.8 mg/dL

* 6 to < 10 years: 1 mg/dL

* 10 to < 13 years: 1.2 mg/dL

* 10 to < 16 years: 1.5 mg/dL (male), 1.4 mg/dL (female)

* >= 16 years: 1.7 mg/dL (male), 1.4 mg/dL (female)

* Total bilirubin ? 1.5 times upper limit of normal (ULN) for age

* Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) < 10 times ULN for age

* Not pregnant or nursing

* Negative pregnancy test

* Shortening fraction >= 27% by echocardiogram (ECHO) OR left ventricular ejection fraction (LVEF) >= 50% by radionuclide angiogram

* No known contraindication (e.g., size, weight or physical condition) to peripheral blood stem cell collection

* No prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease

* No more than one course of chemotherapy per low- or intermediate-risk neuroblastoma therapy prior to determination of MYCN amplification and histology