PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01158300|
Recruitment Status : Completed
First Posted : July 8, 2010
Last Update Posted : May 4, 2015
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.
|Condition or disease||Intervention/treatment||Phase|
|Brain and Central Nervous System Tumors||Drug: VEGF inhibitor PTC299||Phase 1|
- 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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||28 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I and Pharmacokinetic Trial of PTC299 in Pediatric Patients With Refractory or Recurrent CNS Tumors|
|Study Start Date :||November 2010|
|Actual Primary Completion Date :||January 2015|
|Actual Study Completion Date :||January 2015|
- Drug: VEGF inhibitor PTC299
This is a dose escalation study. Study participants will receive .6 or 1.2 mg/kg orally twice daily or 1.2, 1.5, or 2.0 mg/kg orally three times daily for four consecutive weeks (a course). In the absence of unacceptable toxicity or disease progression, treatment may continue for up to 12 courses (approximately one year)
- Maximum-tolerated dose [ Time Frame: First four weeks of treatment ]
- Adverse events [ Time Frame: From the first day of treatment until 30 days after the last dose ]
- Percentage of study participants with complete response or partial response to the study treatment [ Time Frame: Every 8 weeks ]Brain images to assess partial or complete response are performed every 8 weeks after the first dose of the study drug.
- Pharmacokinetics [ Time Frame: Day 1 and day 28 of course 1 ]Blood samples from study participants will be collected on day 1 and day 28 of course 1 for standard full pharmacokinetic studies.
- Change from baseline in blood angiogenic markers and cytokines at discontinuation or completion of treatment [ Time Frame: Before the first dose of drug on day 1 of course 1 and at the discontinuation or completion of treatment ]Blood samples will be collected and analyzed on Day 1 of pre-AM dosing at baseline and at the discontinuation or completion of treatment. Changes from baseline in blood angiogenic markers and cytokines (VEGF-A, VEGF-C, VEGF-D, PlGF, VEGFR-1, VEGFR-2, IL-6, and IL-8) will be assessed.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01158300
|United States, California|
|UCSF Cancer Center and Cancer Research Institute|
|San Francisco, California, United States, 94143-0128|
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010-2970|
|United States, Illinois|
|Children's Memorial Hospital - Chicago|
|Chicago, Illinois, United States, 60614|
|United States, North Carolina|
|Duke Comprehensive Cancer Center|
|Durham, North Carolina, United States, 27710|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104-4318|
|Children's Hospital of Pittsburgh|
|Pittsburgh, Pennsylvania, United States, 15213|
|United States, Tennessee|
|St. Jude Children's Research Hospital|
|Memphis, Tennessee, United States, 38105|
|United States, Texas|
|Texas Children's Cancer Center and Hematology Service at Texas Children's Hospital|
|Houston, Texas, United States, 77030-2399|
|Principal Investigator:||Roger J. Packer, MD||Children's Research Institute|