Trial Outcomes & Findings for A Study of Palbociclib in Combination With Fulvestrant or Tamoxifen as Treatment for Metastatic Breast Cancer (NCT NCT02384239)
NCT ID: NCT02384239
Last Updated: 2022-03-16
Results Overview
Grade 3/4 neutropenia as defined by NCI Common Terminology Criteria for Adverse Events (CTCAE) v4.03 in patients with prior exposure to 1-3 lines of chemotherapy for metastatic breast cancer
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
70 participants
Primary outcome timeframe
Up to 24 months
Results posted on
2022-03-16
Participant Flow
Participant milestones
| Measure |
Palbociclib 100mg
Palbociclib dose 100mg, and either fulvestrant (500 mg intramuscular injection (IM) on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Overall Study
STARTED
|
36
|
34
|
|
Overall Study
COMPLETED
|
36
|
34
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of Palbociclib in Combination With Fulvestrant or Tamoxifen as Treatment for Metastatic Breast Cancer
Baseline characteristics by cohort
| Measure |
Palbociclib 100mg
n=36 Participants
Palbociclib dose 100mg, and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=34 Participants
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Total
n=70 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
30-39 years old
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Customized
40-49 years old
|
5 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Age, Customized
50-59 years old
|
11 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Age, Customized
60-69 years old
|
12 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Age, Customized
70-79 years old
|
5 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Age, Customized
80-89 years old
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
36 Participants
n=5 Participants
|
33 Participants
n=7 Participants
|
69 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 Participants
n=5 Participants
|
33 Participants
n=7 Participants
|
66 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
28 Participants
n=5 Participants
|
26 Participants
n=7 Participants
|
54 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
36 participants
n=5 Participants
|
34 participants
n=7 Participants
|
70 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 24 monthsGrade 3/4 neutropenia as defined by NCI Common Terminology Criteria for Adverse Events (CTCAE) v4.03 in patients with prior exposure to 1-3 lines of chemotherapy for metastatic breast cancer
Outcome measures
| Measure |
Palbociclib 100mg
n=36 Participants
Palbociclib dose 100mg, and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=34 Participants
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Percentage of Participants With Grade 3 or 4 Neutropenia
|
19.4 percentage of participants
|
20.5 percentage of participants
|
SECONDARY outcome
Timeframe: Up to 24 monthsPFS defined as the interval from study entry to the first documented evidence of disease progression by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 as at least a 20% increase in the sum of the longest diameter (SLD) of target lesions, taking as reference the smallest sum SLD recorded since the treatment started and minimum 5 mm increase over the nadir, or the appearance of one or more new lesions. Patients who remain progression-free at the time of analysis will be censored at their last date of follow-up.
Outcome measures
| Measure |
Palbociclib 100mg
n=36 Participants
Palbociclib dose 100mg, and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=34 Participants
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Progression-free Survival (PFS)
|
6.53 months
Interval 2.33 to 12.2
|
9.4 months
Interval 3.56 to 19.7
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: Two participants in the 125mg group did not have an evaluable disease assessment 24 weeks
Defined as the proportion of patients whose best overall response, according to RECIST, is either complete response (CR), a partial response (PR) or stable disease (SD) at 24 weeks. Only those patients who have measurable disease present at baseline, have received at least one cycle of therapy, and have had their disease re-evaluated will be considered evaluable for response.
Outcome measures
| Measure |
Palbociclib 100mg
n=36 Participants
Palbociclib dose 100mg, and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=32 Participants
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Proportion of Participants With Demonstrated Clinical Benefit
|
0.67 proportion of participants
|
0.75 proportion of participants
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: Two participants in the 125mg group did not have an evaluable disease assessment 24 weeks
Defined as the proportion of patients whose best overall response, according to RECIST, is either complete response (CR), a partial response (PR) at 24 weeks. Only those patients who have measurable disease present at baseline, have received at least one cycle of therapy, and have had their disease re-evaluated will be considered evaluable for response.
Outcome measures
| Measure |
Palbociclib 100mg
n=36 Participants
Palbociclib dose 100mg, and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=32 Participants
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Proportion of Participants With an Objective Response
|
0.1111 proportion of participants
|
0.0625 proportion of participants
|
SECONDARY outcome
Timeframe: Up to 24 monthsMedian change in percent positive cells of Ki-67 from baseline will be reported with IQR
Outcome measures
| Measure |
Palbociclib 100mg
n=36 Participants
Palbociclib dose 100mg, and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=34 Participants
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Median Change in Percent Positive Cells From Baseline of Ki-67
|
-8 change in percent positive cells
Interval -22.0 to 0.0
|
-6 change in percent positive cells
Interval -19.0 to -1.0
|
SECONDARY outcome
Timeframe: Up to 24 monthsMedian change in percent positive cells of Total-Rb from baseline will be reported with interquartile range (IQR)
Outcome measures
| Measure |
Palbociclib 100mg
n=36 Participants
Palbociclib dose 100mg, and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=34 Participants
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Median Change in Percent Positive Cells From Baseline of Total-Rb
|
4 change in percent positive cells
Interval -25.0 to 30.0
|
5 change in percent positive cells
Interval -15.0 to 29.0
|
SECONDARY outcome
Timeframe: Up to 24 monthsMedian change in percent positive cells of pS780-Rb from baseline will be reported with IQR
Outcome measures
| Measure |
Palbociclib 100mg
n=36 Participants
Palbociclib dose 100mg, and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=34 Participants
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Median Change in Percent Positive Cells From Baseline of pS780-Rb
|
-3 change in percent positive cells
Interval -32.0 to 4.0
|
-6 change in percent positive cells
Interval -14.0 to 12.0
|
Adverse Events
Palbociclib 100mg
Serious events: 5 serious events
Other events: 7 other events
Deaths: 3 deaths
Palbociclib 125mg
Serious events: 2 serious events
Other events: 9 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Palbociclib 100mg
n=36 participants at risk
Palbociclib dose 100mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=34 participants at risk
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other
|
2.8%
1/36 • Number of events 1 • Up to 24 months
|
2.9%
1/34 • Number of events 1 • Up to 24 months
|
|
Infections and infestations
Infections and infestations - Other
|
2.8%
1/36 • Number of events 1 • Up to 24 months
|
0.00%
0/34 • Up to 24 months
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/36 • Up to 24 months
|
2.9%
1/34 • Number of events 1 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
5.6%
2/36 • Number of events 2 • Up to 24 months
|
0.00%
0/34 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other
|
2.8%
1/36 • Number of events 1 • Up to 24 months
|
0.00%
0/34 • Up to 24 months
|
Other adverse events
| Measure |
Palbociclib 100mg
n=36 participants at risk
Palbociclib dose 100mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
Palbociclib 125mg
n=34 participants at risk
Palbociclib dose 125mg and either fulvestrant (500 mg IM on days 1 and 15 in the first 28 days, then every 28 days thereafter) or tamoxifen (20 mg PO daily by physician choice)
|
|---|---|---|
|
Investigations
Neutrophil count decreased
|
13.9%
5/36 • Number of events 12 • Up to 24 months
|
26.5%
9/34 • Number of events 19 • Up to 24 months
|
|
Vascular disorders
Hypertension
|
5.6%
2/36 • Number of events 2 • Up to 24 months
|
0.00%
0/34 • Up to 24 months
|
Additional Information
Dr. Hope Rugo, MD
University of California, San Francisco
Phone: (415) 353-7618
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place