Busulfan in Treating Children and Adolescents With Refractory CNS Cancer

Phase 1

Completed

• Conditions: Brain and Central Nervous System Tumors; Childhood Germ Cell Tumor; Leukemia; Lymphoma; Metastatic Cancer; Retinoblastoma; Sarcoma

• Registration Number: NCT00006246
• Lead Sponsor: Pediatric Brain Tumor Consortium

Brief Summary

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.

Detailed Description

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 Timeline

• Study First Submitted: 2000-09-11
• Study Start: 2000-11
• Study First Submitted QC: 2003-01-26
• Study First Posted: 2003-01-27
• Primary Completion: 2003-05
• Status Verified: 2009-10
• Last Update Submitted: 2009-10-06
• Last Update Posted: 2009-10-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 28

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (8)

Children's Hospital and Regional Medical Center - Seattle; 🇺🇸 Seattle, Washington, United States

Children's National Medical Center; 🇺🇸 Washington, District of Columbia, United States

Children's Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

UCSF Cancer Center and Cancer Research Institute; 🇺🇸 San Francisco, California, 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

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States