This phase II trial studies how well drugs targeted at genetic mutations work in treating cancer patients with gene mutations. Genetic testing of cancer tumors is a relatively new process. Although a lot of information is learned from this testing, doctors do not always know what the best approach for treatment of cancer is. This is because tumors can have different kinds of genetic mutations and there is not always a known treatment for these mutations. Selecting a medication for cancer treatment based on genetic information about the cancer may bypass ineffective treatments and target specific genetic mutations present in cancer cells.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT04111107.
PRIMARY OBJECTIVE:
I. To estimate the progression-free ratio, as defined by the progression-free survival time on study treatment divided by the progression-free survival time on the last treatment received by patient, for an identified actionable mutation, who will be treated with an off-label treatment off label therapy based on a simplified selection methodology using the next generation sequencing (NGS) results.
SECONDARY OBJECTIVES:
I. To estimate patient response rate on off-label treatments for actionable mutations based on NGS results.
II. To estimate overall survival (OS) for patients treated with off-label treatments for actionable mutations based on NGS results.
III. To describe the safety of using off-label or other experimental treatments for patients with actionable mutations based on NGS results.
EXPLORATORY OBJECTIVES:
I. To describe health related quality of life in patients undergoing off-label treatment targeting genetic mutations, as measured by the Patient-Reported Outcomes Measurement Information System (PROMIS)-29 Overall Health-Related Quality of Life, including 4-Item Anxiety Subscale.
II. Using the Satisfaction with Medical Decision Scale, to describe patient satisfaction with decision to pursue off-label treatment.
III. To identify types of actionable mutations with available targeted treatment occurring in cancer patients.
IV. To characterize the historical treatment regimens for these patients relative to the targetable mutation.
V. To describe patient clinical and demographic characteristics of those with actionable mutations based on NGS results.
VI. To identify barriers to treatment based on NGS results.
OUTLINE:
Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 3, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for 30 days and then every 2 months thereafter.
Lead OrganizationWake Forest University Health Sciences
Principal InvestigatorStefan Charles Grant
