Trial Outcomes & Findings for Neoadjuvant BKM120 in High-risk Prostate Cancer (NCT NCT01695473)
NCT ID: NCT01695473
Last Updated: 2021-01-13
Results Overview
Percentage of men with downstream target inhibition of PI3K in prostate tumor tissue as measured by phosphorylated S6 immunohistochemistry (ICH) when treated with 100 mg/day of BKM120 using paired tumor biopsies from before and after drug administration and defined as ≥ 60% decrease in phosphorylated S6 (pS6) from baseline by Immunohistochemistry (IHC).
TERMINATED
PHASE2
11 participants
Up to 3 months
2021-01-13
Participant Flow
Participant milestones
| Measure |
Neoadjuvant BKM120
Two weeks after confirmatory biopsy, patients will begin taking 100 mg/day of BKM120. BKM120 will be given at this dose level orally once daily for 14 days prior to radical prostatectomy. Radical prostatectomy will be performed on the day of the last dose of BKM120 at day 14. No further drug will be administered after radical prostatectomy. For unforeseen delays in OR scheduling, up to 7 additional days of BKM120 may be administered prior to surgery.
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|---|---|
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Overall Study
STARTED
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11
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Overall Study
COMPLETED
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11
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Neoadjuvant BKM120 in High-risk Prostate Cancer
Baseline characteristics by cohort
| Measure |
Neoadjuvant BKM120
n=11 Participants
Two weeks after confirmatory biopsy, patients will begin taking 100 mg/day of BKM120. BKM120 will be given at this dose level orally once daily for 14 days prior to radical prostatectomy. Radical prostatectomy will be performed on the day of the last dose of BKM120 at day 14. No further drug will be administered after radical prostatectomy. For unforeseen delays in OR scheduling, up to 7 additional days of BKM120 may be administered prior to surgery.
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|---|---|
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Age, Continuous
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61 Years
n=99 Participants
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Sex: Female, Male
Female
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0 Participants
n=99 Participants
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Sex: Female, Male
Male
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11 Participants
n=99 Participants
|
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Ethnicity (NIH/OMB)
Hispanic or Latino
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2 Participants
n=99 Participants
|
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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6 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
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3 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
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Race (NIH/OMB)
Black or African American
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0 Participants
n=99 Participants
|
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Race (NIH/OMB)
White
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6 Participants
n=99 Participants
|
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Race (NIH/OMB)
More than one race
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0 Participants
n=99 Participants
|
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Race (NIH/OMB)
Unknown or Not Reported
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5 Participants
n=99 Participants
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Region of Enrollment
United States
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11 participants
n=99 Participants
|
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Pre-treatment Prostate-specific antigen (PSA)
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21.7 nanograms per mililiter (ng/mL)
n=99 Participants
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PRIMARY outcome
Timeframe: Up to 3 monthsPopulation: One patient did not have pretreatment tissue evaluable for IHC
Percentage of men with downstream target inhibition of PI3K in prostate tumor tissue as measured by phosphorylated S6 immunohistochemistry (ICH) when treated with 100 mg/day of BKM120 using paired tumor biopsies from before and after drug administration and defined as ≥ 60% decrease in phosphorylated S6 (pS6) from baseline by Immunohistochemistry (IHC).
Outcome measures
| Measure |
Neoadjuvant BKM120
n=10 Participants
Two weeks after confirmatory biopsy, patients will begin taking 100 mg/day of BKM120. BKM120 will be given at this dose level orally once daily for 14 days prior to radical prostatectomy. Radical prostatectomy will be performed on the day of the last dose of BKM120 at day 14. No further drug will be administered after radical prostatectomy. For unforeseen delays in OR scheduling, up to 7 additional days of BKM120 may be administered prior to surgery.
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|---|---|
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Percentage of Participants With Decrease in Phosphorylated S6 Immunohistochemistry (ICH) From Baseline
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70 percentage of participants
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SECONDARY outcome
Timeframe: Up to 3 monthsPopulation: One patient did not have pretreatment tissue evaluable for IHC
Percentage of men with downstream target inhibition of phosphatidylinositol 3-kinase (PI3K) in prostate tumor tissue as measured by 4E-binding protein1 (4EBP1) protein phosphorylation immunohistochemistry (IHC) using paired tumor biopsies from before and after drug administration and defined as \>= 60% decline in p4EBP1 IHC
Outcome measures
| Measure |
Neoadjuvant BKM120
n=10 Participants
Two weeks after confirmatory biopsy, patients will begin taking 100 mg/day of BKM120. BKM120 will be given at this dose level orally once daily for 14 days prior to radical prostatectomy. Radical prostatectomy will be performed on the day of the last dose of BKM120 at day 14. No further drug will be administered after radical prostatectomy. For unforeseen delays in OR scheduling, up to 7 additional days of BKM120 may be administered prior to surgery.
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|---|---|
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Percentage of Participants With Decrease in 4E-binding protein1 (p4EBP1) Protein Phosphorylation From Baseline
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100 percentage of participants
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SECONDARY outcome
Timeframe: Up to 3 monthsPopulation: One patient did not have pretreatment tissue evaluable for IHC
Proportion of men with downstream target inhibition of PI3K in prostate tumor tissue as measured by Protein kinase B (pAKT) IHC determined by a \>= 60% decline in pAKT IHC
Outcome measures
| Measure |
Neoadjuvant BKM120
n=10 Participants
Two weeks after confirmatory biopsy, patients will begin taking 100 mg/day of BKM120. BKM120 will be given at this dose level orally once daily for 14 days prior to radical prostatectomy. Radical prostatectomy will be performed on the day of the last dose of BKM120 at day 14. No further drug will be administered after radical prostatectomy. For unforeseen delays in OR scheduling, up to 7 additional days of BKM120 may be administered prior to surgery.
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|---|---|
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Percentage of Participants With Decrease in AKT Protein From Baseline
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70 percentage of participants
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SECONDARY outcome
Timeframe: 1 Day, immediately prior to surgeryActivity of short term BKM120 administration in prostate cancer was determined by measured PSA response immediately prior to radical prostatectomy
Outcome measures
| Measure |
Neoadjuvant BKM120
n=11 Participants
Two weeks after confirmatory biopsy, patients will begin taking 100 mg/day of BKM120. BKM120 will be given at this dose level orally once daily for 14 days prior to radical prostatectomy. Radical prostatectomy will be performed on the day of the last dose of BKM120 at day 14. No further drug will be administered after radical prostatectomy. For unforeseen delays in OR scheduling, up to 7 additional days of BKM120 may be administered prior to surgery.
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|---|---|
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Number of Participants Displaying Activity of Short Term BKM120 Administration
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0 Participants
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Adverse Events
Neoadjuvant BKM120
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Neoadjuvant BKM120
n=11 participants at risk
Two weeks after confirmatory biopsy, patients will begin taking 100 mg/day of BKM120. BKM120 will be given at this dose level orally once daily for 14 days prior to radical prostatectomy. Radical prostatectomy will be performed on the day of the last dose of BKM120 at day 14. No further drug will be administered after radical prostatectomy. For unforeseen delays in OR scheduling, up to 7 additional days of BKM120 may be administered prior to surgery.
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|---|---|
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General disorders
Irritability
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9.1%
1/11 • Number of events 1 • Up to 3 months
All adverse events, with severity/grading as determined by the NCI Common Terminology Criteria for Adverse Events (CTCAE).
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Metabolism and nutrition disorders
Hyperglycemia
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9.1%
1/11 • Number of events 1 • Up to 3 months
All adverse events, with severity/grading as determined by the NCI Common Terminology Criteria for Adverse Events (CTCAE).
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Nervous system disorders
Dizziness
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9.1%
1/11 • Number of events 1 • Up to 3 months
All adverse events, with severity/grading as determined by the NCI Common Terminology Criteria for Adverse Events (CTCAE).
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Renal and urinary disorders
Urinary incontinence
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9.1%
1/11 • Number of events 1 • Up to 3 months
All adverse events, with severity/grading as determined by the NCI Common Terminology Criteria for Adverse Events (CTCAE).
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Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
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9.1%
1/11 • Number of events 1 • Up to 3 months
All adverse events, with severity/grading as determined by the NCI Common Terminology Criteria for Adverse Events (CTCAE).
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Additional Information
Dr. Won Kim, MD
University of California, San Francisco
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place