RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the safety of delivering intrathecal busulfan in children and adolescents who have refractory CNS cancer and to estimate the maximum tolerated dose of this treatment regimen.
OBJECTIVES: * Determine the qualitative and quantitative toxicities of intrathecally administered busulfan in children and adolescents with refractory CNS malignancies. * Determine the maximum tolerated dose of this treatment regimen in these patients. * Determine the cerebrospinal fluid and serum pharmacokinetics of this treatment regimen in these patients. * Determine the efficacy of this treatment regimen in these patients. OUTLINE: This is a dose-escalation study. Patients receive intrathecal busulfan twice a week, at least 3 days apart, for 2 weeks. Patients with complete or partial response or stable disease may continue therapy once a week for 2 weeks, once a week every other week for 2 treatments, and then once a month thereafter in the absence of disease progression or unacceptable toxicity. Cohorts of 3-6 patients receive escalating doses of busulfan until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicities. Patients are followed every 3 months for the first year, every 6 months for 4 years, and then annually for 5 years. PROJECTED ACCRUAL: Approximately 18-24 patients will be accrued for this study over 18-38 months.
Study Type
INTERVENTIONAL
Purpose
TREATMENT
Enrollment
28
UCSF Cancer Center and Cancer Research Institute
San Francisco, California, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
Duke Comprehensive Cancer Center
Durham, North Carolina, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Baylor College of Medicine
Houston, Texas, United States
Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, United States
Toxicities of IT administered busulfan in children and adolescents with refractory CNS malignancies
Maximum tolerated dose of IT administered busulfan
Serum and CSF pharmacokinetics of IT administered busulfan
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.