Phase I Study of Intrathecal Spartaject-Busulfan in Children With Neoplastic Meningitis
- 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
Endpoint Classification: Safety Study, Primary Purpose: Treatment
Toxicities of IT administered busulfan in children and adolescents with refractory CNS malignancies
Sri Gururangan, MD
United States: Food and Drug Administration
|Baylor College of Medicine||Houston, Texas 77030|
|Children's Hospital of Philadelphia||Philadelphia, Pennsylvania 19104|
|Duke Comprehensive Cancer Center||Durham, North Carolina 27710|
|UCSF Cancer Center and Cancer Research Institute||San Francisco, California 94115-0128|
|Dana-Farber Cancer Institute||Boston, Massachusetts 02115|
|Children's National Medical Center||Washington, District of Columbia 20010-2970|
|Children's Hospital of Pittsburgh||Pittsburgh, Pennsylvania 15213|
|Children's Hospital and Regional Medical Center - Seattle||Seattle, Washington 98105|