NCT02015728

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

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

• Status: Unknown
• Phase: N/A
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Seattle Children's Hospital · Academic / Other
• Sex: All
• Age: 1 Month – 30 Years
• Healthy volunteers: Not accepted

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 description

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.

Conditions

• Recurrent Childhood Brain Tumor

Interventions

Type	Name	Description
DEVICE	Tumor biology testing	Tumor biology studies will be performed in a CLIA-approved clinical pathology laboratory using standard procedures. Immunohistochemical (IHC) testing will be performed on formalin fixed tumor obtained at the time of diagnosis and/or relapse. Results will be interpreted by a qualified pediatric pathologist and will be scored on a scale of 0 to 4+ commenting on both percentage of positive cells and intensity of staining. Results will further be reported as a binary result (positive/negative). If more than one tumor specimen is available from different surgical procedures (e.g. initial diagnosis and relapse), the results from the relapse specimen will be prioritized. Results will determine kinase inhibitor treatment arm assignment which will be administered in addition to the "best available" combination of low-dose oral cytotoxic agents, including temozolomide and etoposide.
DRUG	Temozolomide	Temozolomide combined with Etoposide is considered the "best available" combination of low-dose oral cytotoxic agents for patients with refractory or recurrent CNS tumors.
DRUG	Etoposide	Etoposide combined with Temozolomide is considered the "best available" combination of low-dose oral cytotoxic agents for patients with refractory or recurrent CNS tumors.
DRUG	Sorafenib	Sorafenib is a broad-spectrum kinase inhibitor.
DRUG	Everolimus	Everolimus is an mTOR pathway inhibitor.
DRUG	Erlotinib	Erlotinib is a tyrosine kinase inhibitor of the ERBB family of proteins.
DRUG	Dasatinib	Dasatinib is a broad spectrum SRC inhibitor.

Timeline

• Start date: 2013-12-01
• Primary completion: 2016-11-01
• Completion: 2017-12-01
• First posted: 2013-12-19
• Last updated: 2016-12-15

Locations

1 site across 1 country: United States