Phase I Study of Doxorubicin HCl Liposome in Pediatric Patients With Refractory Solid Tumors
OBJECTIVES: I. Determine the tolerance to and toxicity profile of doxorubicin HCl liposome
(Lipodox) at standard doxorubicin doses and doses of Lipodox that were tolerable in adults
administered every 3 weeks in pediatric patients with refractory solid tumors.
II. Determine the maximum tolerated dose of this drug in these patients if dose-limiting
toxicity is observed at doses of 105 mg/m2 or less.
III. Determine the pharmacokinetics of this drug in these patients. IV. Assess the
cardiotoxicity of this drug in children who have previously been treated with free
doxorubicin and in children who have not previously received doxorubicin.
V. Evaluate the feasibility of using cardiac MRI functional imaging as a screening tool for
the quantitative assessment of doxorubicin-induced cardiotoxicity.
VI. Determine if serum troponin t levels are a useful biomarker for doxorubicin-induced
PROTOCOL OUTLINE: This is a dose-escalation, multicenter study. Patients receive doxorubicin
HCl liposome IV over 60 minutes. Treatment repeats every 4 weeks for a maximum of 10
courses in the absence of disease progression or unacceptable toxicity.
Cohorts of 4-6 patients receive escalating doses of doxorubicin HCl liposome until the
maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that
at which at least 2 of 4 or 2 of 6 patients experience dose-limiting toxicity.
Patients are followed every 3 months for 1 year, every 6 months for 2 years, and then
A total of 21-36 patients will be accrued for this study within 1-2 years.
Primary Purpose: Treatment
National Cancer Institute (NCI)
United States: Federal Government
|Children's Hospital of Philadelphia||Philadelphia, Pennsylvania 19104|
|Pediatric Oncology Branch||Bethesda, Maryland 20892|