This randomized clinical trial studies how well next generation sequence target-directed therapy works in treating patients with cancer. Next generation sequencing is a test that screens for mutations to cancer related genes. Target-directed therapy is a type of treatment that uses drugs or other substances to identify and attack specific types of cancer cells that may have less harm to normal cells. Next generation sequencing may help identify these specific types of cancer cells.
PRIMARY OBJECTIVES: I. Overall (composite) response rate (ORR). SECONDARY OBJECTIVES: I. 4-month progression free survival (PFS). II. Mutation rate. III. Adverse event rate/severity. IV. Overall survival. TERTIARY OBJECTIVES: I. Targeted agent rate. II. Available protocol rate. III. Protocol enrollment rate. IV. Disease site influence. OUTLINE: Patients are randomized to 1 of 2 treatment arms. ARM A: Patients undergo collection of tissue and blood samples for analysis via next generation sequencing. Patients receive standard of care therapy based on the discretion of the treating physician. ARM B: Patients undergo collection of tissue and blood samples for analysis via next generation sequencing. Based on the results of the next generation sequencing, patients receive target-directed therapy. After completion of study treatment, patients are followed up every 3 months for 2 years, every 6 months for 2 years, and then annually thereafter.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Undergo collection of tissue and blood samples
Receive target-directed therapy
Receive standard of care therapy
Fox Chase Cancer Center
Philadelphia, Pennsylvania, United States
Overall (composite) response rate
The overall (composite) response rate will be defined by tumor response rate according to RECIST 1.1 or by tumor marker response for patients without measurable disease defined by RECIST 1.1. Tumor marker response will be quantified as a 50% reduction in the marker of interest, when compared to baseline, without any radiographic evidence of progressive disease.
Time frame: Up to 2 years
Progression free survival
The percentage of patients progression free and alive will be estimated using the method of Kaplan and Meier. PFS in control will be compared to those in the experimental arm using log-rank tests.
Time frame: Time from randomization to time of progression or death, whichever occurs first, assessed at 4 months
Mutation rate
Mutation rate, defined as the percentage of patients with \>= 1 mutation identified will be estimated using the method of Pearson and Clopper as binomial proportions. 95% confidence intervals will be provided for these proportions.
Time frame: Up to 2 years
Actionable mutation rate
The percentage of patients with "actionable" mutation rate will be estimated using the method of Pearson and Clopper as binomial proportions. 95% confidence intervals will be provided for these proportions.
Time frame: Up to 2 years
Incidence of adverse events graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0
The rate of adverse events will be estimated using the method of Pearson and Clopper as binomial proportions. 95% confidence intervals will be provided for these proportions. Adverse events will be tabulated according to severity.
Time frame: Up to 2 years
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Correlative studies
Median overall survival
OS will be estimated using the method of Kaplan and Meier. OS in control will be compared to those in the experimental arm using log-rank tests.
Time frame: Up to 2 years