The purpose of this study is to evaluate the effects of the study drug, BKM120. The study drug, BKM120, is an inhibitor of a protein called phosphatidyl inositol-3-kinase (PI3K). This protein is found in normal cells and in cancer cells, but often in many cancer cells this protein is overactive. Inhibiting the protein may slow the growth of prostate cancer but this has not been tested yet in men with prostate cancer.
This is an open label, single arm phase II study of BKM120 in men with metastatic castration resistant prostate cancer (CRPC) who have progressed on or following completion of docetaxel-based chemotherapy. Prior progression on cabazitaxel, provenge, abiraterone, or enzalutamide (MDV3100) is also permitted. Patients must have baseline evaluations performed prior to the first dose of study drug and must meet all inclusion and exclusion criteria. Results of the baseline evaluations, which assure that all inclusion and exclusion criteria have been satisfied, must be reviewed by the Principal Investigator or his/her designee prior to enrollment of that patient. In addition, the patient must be thoroughly informed about all aspects of the study, including the study visit schedule and required evaluations and all regulatory requirements for informed consent. The written informed consent must be obtained from the patient prior to protocol-specific screening tests. The following criteria apply to all patients enrolled onto the study unless otherwise specified.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
BKM120 at the maximum tolerated dose (MTD) of 100mg orally daily
Duke Cancer Institute
Durham, North Carolina, United States
OHSU Knight Cancer Institute
Portland, Oregon, United States
University of Washington
Seattle, Washington, United States
Progression Free Survival (PFS) Prostate Cancer Working Group 2 (PCWG2) Criteria or Based on the Onset of a Skeletal Related Event.
Time in months from the start of study treatment to the date of first progression or death due to any cause. Progression was determined either radiographically using a composite of the Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 and Prostate Cancer Working Group 2 (PCWG2) criteria or clinically as judged from a skeletal-related event, need for change in therapy, or clinical deterioration. Patients alive who had not progressed as of the last follow-up had PFS censored at the last follow-up date. Median PFS was estimated using a Kaplan-Meier curve. Response and progression are evaluated using a combination of Response Evaluation Criteria in Solid Tumors (RECIST v1.1) and guidelines for prostate cancer endpoints developed by the Prostate Cancer Clinical Trials Working Group (PCWG2). Per RECIST, progression is defined as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
Time frame: 5 years
Radiologic Response
The number of patients achieving a complete response (CR) or partial response (PR) based on RECIST 1.1 criteria. Response and progression is evaluated using a combination of the Response Evaluation Criteria in Solid Tumors Criteria (RECIST v1.1) and the guidelines for prostate cancer endpoints developed by the Prostate Cancer Clinical Trials Working Group (PCWG2). Per RECIST, progression is defined as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
Time frame: 2 years
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Number of patients experiencing grade 3-5 adverse events as defined by the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Time frame: Up to 28 days post last study drug dose. This is the follow-up safety visit.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Prostate Specific Antigen (PSA) Response
The number of patients with a 30% and 50% decrease in PSA from baseline.
Time frame: 2 years
Overall Survival of Participants.
Time in months from the start of study treatment to date of death due to any cause. Patients alive as of the last follow-up had OS censored at the last follow-up date. Median OS was estimated using a Kaplan-Meier curve.
Time frame: 5 years
Change in Circulating Tumor Cell (CTC) Levels From Baseline
The number of patients with a baseline CTC level of at least 5 who achieved a CTC level of less than 5 during the study.
Time frame: 2 years
Time to New Metastatic Disease From the Baseline Visit
Time in days from the start of study treatment to time of new metastatic disease. Time to new metastatic disease is, defined from the date of first study agent administration to the onset of a new evaluable site of disease as per PCWG2 and RECIST 1.1 guidelines, excluding the primary site and all sites documented at baseline will be assessed. Only those patients that experienced a new lesion per this definition are included.
Time frame: 5 years