Phase I and Pharmacokinetic Trial of PTC299 in Pediatric Patients With Refractory or Recurrent CNS Tumors
- To estimate the maximum-tolerated dose and the recommended phase II dose of VEGF
inhibitor PTC299 (PTC299) in pediatric patients with recurrent or progressive primary
central nervous system (CNS) tumors.
- To evaluate and characterize the adverse events associated with this regimen in these
- To evaluate and characterize the pharmacokinetics and pharmacodynamics of this regimen
in these patients.
- To investigate the relationships between PTC299 plasma exposure and other outcomes
- To evaluate the antitumor activity of this regimen in these patients.
- To evaluate changes in angiogenic and inflammatory markers in the blood and the
relationship between these changes and other outcome measures.
- To obtain preliminary evidence of biologic activity of PTC299 by using magnetic
resonance diffusion to assess tumor cellularity.
OUTLINE: This is a multicenter, dose-escalation study.
Patients receive oral VEGF inhibitor PTC299 twice or thrice daily. Treatment repeats every
28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Blood samples are collected at baseline and periodically during study for pharmacokinetic
and pharmacodynamic studies by ELISA.
After completion of study therapy, patients are followed up for 30 days.
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
First four weeks of treatment
Roger J. Packer, MD
Children's Research Institute
United States: Food and Drug Administration
|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|
|Children's National Medical Center||Washington, District of Columbia 20010-2970|
|Children's Hospital of Pittsburgh||Pittsburgh, Pennsylvania 15213|
|Children's Memorial Hospital - Chicago||Chicago, Illinois 60614|
|St. Jude Children's Research Hospital||Memphis, Tennessee 38105-2794|
|Texas Children's Cancer Center and Hematology Service at Texas Children's Hospital||Houston, Texas 77030-2399|