PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
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.
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
Pediatric Brain Tumor Consortium
Results (where available)
- Source: http://clinicaltrials.gov/show/NCT01158300
- Information obtained from ClinicalTrials.gov on May 16, 2012
Medical and Biotech [MESH] Definitions
An inhibitor of DOPA DECARBOXYLASE that does not enter the central nervous system. It is often given with LEVODOPA in the treatment of parkinsonism to prevent the conversion of levodopa to dopamine in the periphery, thereby increasing the amount that reaches the central nervous system and reducing the required dose. It has no antiparkinson actions when given alone.
Neoplasms of the intracranial components of the central nervous system, including the cerebral hemispheres, basal ganglia, hypothalamus, thalamus, brain stem, and cerebellum. Brain neoplasms are subdivided into primary (originating from brain tissue) and secondary (i.e., metastatic) forms. Primary neoplasms are subdivided into benign and malignant forms. In general, brain tumors may also be classified by age of onset, histologic type, or presenting location in the brain.
Neuroectodermal Tumors, Primitive
A group of malignant tumors of the nervous system that feature primitive cells with elements of neuronal and/or glial differentiation. Use of this term is limited by some authors to central nervous system tumors and others include neoplasms of similar origin which arise extracranially (i.e., NEUROECTODERMAL TUMORS, PRIMITIVE, PERIPHERAL). This term is also occasionally used as a synonym for MEDULLOBLASTOMA. In general, these tumors arise in the first decade of life and tend to be highly malignant. (From DeVita et al., Cancer: Principles and Practice of Oncology, 5th ed, p2059)
Autonomic Nervous System Diseases
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.
Central Nervous System
The main information-processing organs of the nervous system, consisting of the brain, spinal cord, and meninges.
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