A Phase I Study of Intravenous (Emulsion) Fenretinide (4-HPR, NSC 374551) in Children With Recurrent or Resistant Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), and Non-Hodgkin's Lymphoma (NHL) IND #70,058"
Fenretinide is a cytotoxic retinoid that has activity against a variety of cell lines in
vitro in a dose-related manner. The exact mechanism of fenretinide cytotoxicity in leukemia
and lymphoma cell lines is not known, but may include the de novo ceramide synthesis of
ceramides and the generation of reactive oxygen species. The malignancy-specific nature of
fenretinide-induced ceramides suggests that combinations of the drug with other ceramide
modulating agents may have a favorable therapeutic index.
In this study, the primary aims are to define the maximum tolerated dose, toxicity profile,
and pharmacokinetics of IV fenretinide when given continuously in pediatric patients with
ALL, AML, and NHL. The drug will be administered via a central venous or percutaneous
indwelling central catheter in an inpatient hospital setting.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Determine maximum tolerated dose
end of study
Anna R Franklin, MD
M.D. Anderson Cancer Center
United States: Food and Drug Administration
|MD Anderson Cancer Center||Houston, Texas 77030-4096|
|University of Oklahoma Health Sciences Center||Oklahoma City, Oklahoma 73104|