Clinical Trial: Selecting Patient-Specific Biologically Targeted Therapy for Pediatric Patients With Refractory Or Recurrent Brain Tumors

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: The Feasibility of Selecting Patient-Specific Biologically Targeted Therapy With Sorafenib, Everolimus, Erlotinib or Dasatinib for Pediatric Patients With Refractory Or Recurrent Brain Tumors

Brief Summary: This research study is a Feasibility clinical trial. In this trial, researchers are trying to figure out whether a medication can be chosen based on rapid testing done on tumor tissue. Information from a feasibility or pilot trial will hopefully help researchers plan larger trials in the future to determine the effect of this therapy.

Detailed Summary:

This research study will assign a specific drug treatment based on lab tests performed on the participant's tumor from tumor tissue taken from a biopsy done when he/she was first diagnosed or if taken when he/she relapsed or progressed. All participants will get Temozolomide and Etoposide to start. Then depending on review of the participant's tumor tissue he/she will also receive one of the following: Sorafenib, Everolimus, Erlotinib, or Dasatinib.

The purpose of this research study is to learn about the feasibility of obtaining and using information from studies done on tumor tissue in order to help make treatment decisions for patients with relapsed or refractory pediatric brain tumors. The investigators also want to find out the effects this therapy has on the participant and the participant's brain tumor.


Sponsor: Seattle Children's Hospital

Current Primary Outcome: Feasibility [ Time Frame: 3 years ]

It will be considered feasible to obtain individual biologic testing if at least 80% of patients receive results of biology studies within two weeks of study enrollment.

It will be considered feasible to treat patients based on biologic testing if at least and 50% of patients who consent for biology testing start therapy with one of four regimens within four weeks of study enrollment.



Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Efficacy [ Time Frame: 3 years ]
    Efficacy endpoint for patients with evaluable or measurable disease will be best objective response (CR, PR, SD or PD) measured by MRI imaging.
  • Survival [ Time Frame: 3 years ]
    Survival endpoints will be estimated including time to progression as well as progression-free and overall survival rates at the 6 month, 1 year and 2 year time point from start of study treatment.
  • Toxicity [ Time Frame: 3 years ]
    Toxicity endpoints will be descriptive and include grading of patient toxicity according to the National Cancer Institute common terminology criteria for adverse events (CTCAE version 4.0). This study is not intended or powered to compare toxicity between treatment arms. The tolerability of selected therapy with the addition of a kinase inhibitor will be described for all patients as a group.


Original Secondary Outcome: Same as current

Information By: Seattle Children's Hospital

Dates:
Date Received: December 13, 2013
Date Started: December 2013
Date Completion: December 2017
Last Updated: December 14, 2016
Last Verified: December 2016