Trial Outcomes & Findings for PI3K Inhibitor BKM120 and Cetuximab in Treating Patients With Recurrent or Metastatic Head and Neck Cancer (NCT NCT01816984)
NCT ID: NCT01816984
Last Updated: 2021-10-05
Results Overview
Performed using snap frozen tissue samples using the well-established PamGene Kinase array platform available in the Salgia/Seiwert laboratories.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
12 participants
Primary outcome timeframe
1 week
Results posted on
2021-10-05
Participant Flow
Participant milestones
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
COMPLETED
|
11
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Overall Study
declining health status
|
1
|
Baseline Characteristics
PI3K Inhibitor BKM120 and Cetuximab in Treating Patients With Recurrent or Metastatic Head and Neck Cancer
Baseline characteristics by cohort
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
n=12 Participants
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Age, Continuous
|
61 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
11 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
African American
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 weekPopulation: Data not collected
Performed using snap frozen tissue samples using the well-established PamGene Kinase array platform available in the Salgia/Seiwert laboratories.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 28 daysPopulation: 0 patients out of 3 from the BKM 80mg/day cohort had DLT (dose limiting toxicities). 0 patients out of 9 from the BKM 100mg/day cohort had DLT.
Maximum tolerated dose (MTD) is defined as the dose level preceding the dose in which greater than or equal to 2 out of 3-6 patients experience a dose limiting toxicity (DLT) assessed using CTCAE v4
Outcome measures
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
n=12 Participants
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Maximum Tolerated Dose (MTD)
|
100 mg / day
|
SECONDARY outcome
Timeframe: Up to 28 daysPopulation: Data not collected.
Measured by Terminal deoxynucleotidyl transferase dUTP nick end labeling (TUNEL) assay on Formalin-Fixed, Paraffin-Embedded (FFPE) sections in a descriptive manner.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 28 daysPercentage of patients whose cancer shrinks or disappears after treatment.
Outcome measures
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
n=12 Participants
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Response Rate Assessed Using RECIST
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to 28 daysPercentage of patients with prior EGFR failure assessed using RECIST whose cancer shrinks or disappears after treatment.
Outcome measures
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
n=11 Participants
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Response Rate in Patients With Prior EGFR Failure Assessed Using RECIST
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to 28 daysPopulation: Data not collected.
Tumor shrinkage will be visualized as a waterfall plot for graphical (qualitative) comparison.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 4 years and 3 monthsOverall survival is defined as the time from study initiation to death due to any reason.
Outcome measures
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
n=12 Participants
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Overall Survival
|
9.3 months
Interval 5.5 to 14.0
|
SECONDARY outcome
Timeframe: 4 years and 3 monthsProgression free survival is defined as the time from study initiation to first evidence of progression of disease.
Outcome measures
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
n=12 Participants
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Progression Free Survival
|
2 months
Interval 1.7 to 5.0
|
Adverse Events
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
Serious events: 4 serious events
Other events: 12 other events
Deaths: 12 deaths
Serious adverse events
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
n=12 participants at risk
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Metabolism and nutrition disorders
Electrolyte abnormality and anemia
|
8.3%
1/12 • 4 years and 3 months
|
|
Vascular disorders
Orthostatis
|
8.3%
1/12 • 4 years and 3 months
|
|
Renal and urinary disorders
Acute Kidney Injury
|
16.7%
2/12 • 4 years and 3 months
|
Other adverse events
| Measure |
Arm I (BKM120 PO and Cetuximab 500 mg IV 14 Days)
n=12 participants at risk
Patients receive PI3K inhibitor BKM120 PO QD 100 mg/day on days -7 to 0. Patients complete 1 week washout. After dose escalation phase, 3 patients receive BKM120 PO 80mg / day and cetuximab 500 mg IV /14 days and 9 patients receive BKM120 PO 100mg / day and cetuximab 500 mg IV /14 days thereafter.
All patients receive PI3K inhibitor BKM120 PO QD day on days 1-28 and cetuximab IV over 60-120 minutes on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
PI3K inhibitor BKM120: Given PO
cetuximab: Given IV
|
|---|---|
|
Metabolism and nutrition disorders
Hyperglycemia
|
91.7%
11/12 • 4 years and 3 months
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
83.3%
10/12 • 4 years and 3 months
|
|
Gastrointestinal disorders
Anorexia
|
66.7%
8/12 • 4 years and 3 months
|
|
General disorders
Fatigue
|
66.7%
8/12 • 4 years and 3 months
|
|
General disorders
Pain
|
66.7%
8/12 • 4 years and 3 months
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
58.3%
7/12 • 4 years and 3 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
58.3%
7/12 • 4 years and 3 months
|
|
Metabolism and nutrition disorders
ALT Increase
|
58.3%
7/12 • 4 years and 3 months
|
|
Metabolism and nutrition disorders
AST increase
|
50.0%
6/12 • 4 years and 3 months
|
|
Gastrointestinal disorders
Diarrhea
|
50.0%
6/12 • 4 years and 3 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
41.7%
5/12 • 4 years and 3 months
|
|
Nervous system disorders
Anxiety
|
33.3%
4/12 • 4 years and 3 months
|
|
Gastrointestinal disorders
Constipation
|
33.3%
4/12 • 4 years and 3 months
|
|
General disorders
Mood changes
|
25.0%
3/12 • 4 years and 3 months
|
|
Gastrointestinal disorders
Mucositis
|
25.0%
3/12 • 4 years and 3 months
|
|
Gastrointestinal disorders
Nausea
|
25.0%
3/12 • 4 years and 3 months
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
25.0%
3/12 • 4 years and 3 months
|
|
General disorders
Transaminitis
|
25.0%
3/12 • 4 years and 3 months
|
|
General disorders
Anemia
|
16.7%
2/12 • 4 years and 3 months
|
|
Gastrointestinal disorders
Dysphagia
|
16.7%
2/12 • 4 years and 3 months
|
|
Metabolism and nutrition disorders
Increased BUN
|
16.7%
2/12 • 4 years and 3 months
|
|
General disorders
Leg tremor
|
16.7%
2/12 • 4 years and 3 months
|
|
Blood and lymphatic system disorders
Total bilirubin increase
|
16.7%
2/12 • 4 years and 3 months
|
|
Gastrointestinal disorders
Vomiting
|
16.7%
2/12 • 4 years and 3 months
|
Additional Information
Dr Everett Vokes, MD
University of Chicago Medicine and Biological Sciences
Phone: 855-702-8222
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place