PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors

2014-08-27 03:12:23 | BioPortfolio


RATIONALE: PTC299 may stop the growth of tumor cells by blocking blood flow to the tumor.

PURPOSE: This phase I trial is studying the side effects and the best dose of PTC299 in treating young patients with recurrent or refractory primary central nervous system 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 patients.

- 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 measures.

- 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.

Study Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Brain and Central Nervous System Tumors


VEGF inhibitor PTC299


UCSF Cancer Center and Cancer Research Institute
San Francisco
United States




Pediatric Brain Tumor Consortium

Results (where available)

View Results


Published on BioPortfolio: 2014-08-27T03:12:23-0400

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Diseases of the parasympathetic or sympathetic divisions of the AUTONOMIC NERVOUS SYSTEM; which has components located in the CENTRAL NERVOUS SYSTEM and PERIPHERAL NERVOUS SYSTEM. Autonomic dysfunction may be associated with HYPOTHALAMIC DISEASES; BRAIN STEM disorders; SPINAL CORD DISEASES; and PERIPHERAL NERVOUS SYSTEM DISEASES. Manifestations include impairments of vegetative functions including the maintenance of BLOOD PRESSURE; HEART RATE; pupil function; SWEATING; REPRODUCTIVE AND URINARY PHYSIOLOGY; and DIGESTION.

The main information-processing organs of the nervous system, consisting of the brain, spinal cord, and meninges.

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