Trial Outcomes & Findings for Hormone Therapy Or Chemotherapy Before Surgery Based on Gene Expression Analysis in Treating Patients With Breast Cancer (NCT NCT01293032)
NCT ID: NCT01293032
Last Updated: 2016-07-12
Results Overview
The primary purpose of this trial is to determine the feasibility of carrying out a large multi-center trial with a similar design. Feasibility, in terms of less than 1/3 of patients with intermediate (11-25) Recurrence Score (RS) who refused the assigned treatment (Group 2) or refused randomization between hormonal (Arm 1) or chemotherapy (Arm 2). The confidence interval will be 95%. The proportion (and 95% confidence interval) of patients with RS 11-25 who refuse the assigned treatment will be calculated.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
59 participants
Primary outcome timeframe
Up to 2 years
Results posted on
2016-07-12
Participant Flow
Once a patient consented to this study and was deemed eligible the core biopsy blocks or slides were sent to obtain the Oncotype DX Breast Cancer Assay. After the Recurrence Score(RS) results were available the subject was assigned to Group 1, 2, or 3. If assigned to Group 2 they were randomized to Arm 1 or Arm 2.
5/64 subjects were not assigned or randomized to an arm: delayed oncotype result, core block lost, discrepancy in a bio-marker test, and two subjects deemed not eligible. There were 5 subjects who refused assigned randomization Group 2 Arm 2. 2/5 subjects who refused Group 2 Arm 2 were treated and evaluable for response on Arm 1.
Participant milestones
| Measure |
Group 1 (RS < 11)
Patients with a RS of less than 11 were assigned to Group 1. They received neoadjuvant hormonal therapy comprised of tamoxifen (pre-menopausal women) or an aromatase inhibitor (post-menopausal women) for 4-6 months in the absence of disease progression or unacceptable toxicity.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/ Oncotype DX Gene Expression Profiling System
* Hormonal therapy:
* Tamoxifen Citrate (pre-menopausal women) OR
* Aromatase Inhibition Therapy (post-menopausal women)
|
Group 2 Arm 1 (RS 11-25)
Patients with an intermediate RS (11-25) were assigned to Group 2. If randomized to Arm 1 they received neoadjuvant hormonal therapy as in Group 1.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/ Oncotype DX Gene Expression Profiling System
* Hormonal therapy:
* Tamoxifen Citrate (pre-menopausal women) OR
* Aromatase Inhibition Therapy (post-menopausal women)
|
Group 2 Arm 2 (RS 11-25)
Patients with an intermediate RS (11-25) were assigned to Group 2. If randomized to Arm 2 they received 6-8 courses of neoadjuvant chemotherapy comprised of anthracycline/taxane based regimen over 4-6 months in the absence of disease progression or unacceptable toxicity.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/Oncotype DX Gene Expression Profiling System
* Systemic chemotherapy
|
Group 3 (RS > 25)
Patients with a high RS (\> 25) were assigned to Group 3. They received chemotherapy, neoadjuvant chemotherapy as in Group 2 Arm 2.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/Oncotype DX Gene Expression Profiling System
* Systemic chemotherapy
|
|---|---|---|---|---|
|
Recurrence Score(RS)Assigned/Randomized
STARTED
|
12
|
17
|
16
|
14
|
|
Recurrence Score(RS)Assigned/Randomized
Assigned/Randomized to Arm
|
12
|
17
|
16
|
14
|
|
Recurrence Score(RS)Assigned/Randomized
COMPLETED
|
12
|
17
|
11
|
14
|
|
Recurrence Score(RS)Assigned/Randomized
NOT COMPLETED
|
0
|
0
|
5
|
0
|
|
Treatment
STARTED
|
12
|
19
|
11
|
14
|
|
Treatment
COMPLETED
|
10
|
14
|
10
|
13
|
|
Treatment
NOT COMPLETED
|
2
|
5
|
1
|
1
|
Reasons for withdrawal
| Measure |
Group 1 (RS < 11)
Patients with a RS of less than 11 were assigned to Group 1. They received neoadjuvant hormonal therapy comprised of tamoxifen (pre-menopausal women) or an aromatase inhibitor (post-menopausal women) for 4-6 months in the absence of disease progression or unacceptable toxicity.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/ Oncotype DX Gene Expression Profiling System
* Hormonal therapy:
* Tamoxifen Citrate (pre-menopausal women) OR
* Aromatase Inhibition Therapy (post-menopausal women)
|
Group 2 Arm 1 (RS 11-25)
Patients with an intermediate RS (11-25) were assigned to Group 2. If randomized to Arm 1 they received neoadjuvant hormonal therapy as in Group 1.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/ Oncotype DX Gene Expression Profiling System
* Hormonal therapy:
* Tamoxifen Citrate (pre-menopausal women) OR
* Aromatase Inhibition Therapy (post-menopausal women)
|
Group 2 Arm 2 (RS 11-25)
Patients with an intermediate RS (11-25) were assigned to Group 2. If randomized to Arm 2 they received 6-8 courses of neoadjuvant chemotherapy comprised of anthracycline/taxane based regimen over 4-6 months in the absence of disease progression or unacceptable toxicity.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/Oncotype DX Gene Expression Profiling System
* Systemic chemotherapy
|
Group 3 (RS > 25)
Patients with a high RS (\> 25) were assigned to Group 3. They received chemotherapy, neoadjuvant chemotherapy as in Group 2 Arm 2.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/Oncotype DX Gene Expression Profiling System
* Systemic chemotherapy
|
|---|---|---|---|---|
|
Recurrence Score(RS)Assigned/Randomized
Refused Assigned Arm
|
0
|
0
|
5
|
0
|
|
Treatment
Adverse Event
|
0
|
0
|
1
|
1
|
|
Treatment
Withdrawal by Subject
|
2
|
4
|
0
|
0
|
|
Treatment
Disease Progression, relapse
|
0
|
1
|
0
|
0
|
Baseline Characteristics
Hormone Therapy Or Chemotherapy Before Surgery Based on Gene Expression Analysis in Treating Patients With Breast Cancer
Baseline characteristics by cohort
| Measure |
Group 1 (RS < 11)
n=12 Participants
Patients with a Recurrence Score (RS) of less than 11 were assigned to Group 1. They received neoadjuvant hormonal therapy comprised of tamoxifen (pre-menopausal women) or an aromatase inhibitor (post-menopausal women) for 4-6 months in the absence of disease progression or unacceptable toxicity.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/ Oncotype DX Gene Expression Profiling System
* Hormonal therapy:
* Tamoxifen Citrate (pre-menopausal women) OR
* Aromatase Inhibition Therapy (post-menopausal women)
|
Group 2 Arm 1 (RS 11-25)
n=17 Participants
Patients with an intermediate RS (11-25) were assigned to Group 2. If randomized to Arm 1 they received neoadjuvant hormonal therapy as in Group 1.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/ Oncotype DX Gene Expression Profiling System
* Hormonal therapy:
* Tamoxifen Citrate (pre-menopausal women) OR
* Aromatase Inhibition Therapy (post-menopausal women)
|
Group 2 Arm 2 (RS 11-25)
n=16 Participants
Patients with an intermediate RS (11-25) were assigned to Group 2. If randomized to Arm 2 they received 6-8 courses of neoadjuvant chemotherapy comprised of anthracycline/taxane based regimen over 4-6 months in the absence of disease progression or unacceptable toxicity.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/Oncotype DX Gene Expression Profiling System
* Systemic chemotherapy
|
Group 3 (RS > 25)
n=14 Participants
Patients with a high RS (\> 25) were assigned to Group 3. They received chemotherapy, neoadjuvant chemotherapy as in Group 2 Arm 2.
Treatment:
* Neoadjuvant therapy
* Therapeutic conventional surgery
* Laboratory biomarker analysis/Correlative studies
* Gene Expression Analysis/Oncotype DX Gene Expression Profiling System
* Systemic chemotherapy
|
Total
n=59 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
36 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
7 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
23 Participants
n=21 Participants
|
|
Age, Continuous
|
55.3125 years
STANDARD_DEVIATION 5.931426 • n=5 Participants
|
63.64706 years
STANDARD_DEVIATION 11.5539 • n=7 Participants
|
55.3125 years
STANDARD_DEVIATION 12.2556 • n=5 Participants
|
60.64286 years
STANDARD_DEVIATION 7.312867 • n=4 Participants
|
61.25423729 years
STANDARD_DEVIATION 10.52188724 • n=21 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
14 Participants
n=4 Participants
|
59 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
14 Participants
n=4 Participants
|
56 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
11 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
48 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
Canada
|
1 participants
n=5 Participants
|
4 participants
n=7 Participants
|
2 participants
n=5 Participants
|
4 participants
n=4 Participants
|
11 participants
n=21 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=5 Participants
|
13 participants
n=7 Participants
|
14 participants
n=5 Participants
|
10 participants
n=4 Participants
|
48 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Up to 2 yearsPopulation: Patients with an intermediate RS(11-25) assigned to Group 2, Arm 1, and Arm 2 were combined in the analysis.
The primary purpose of this trial is to determine the feasibility of carrying out a large multi-center trial with a similar design. Feasibility, in terms of less than 1/3 of patients with intermediate (11-25) Recurrence Score (RS) who refused the assigned treatment (Group 2) or refused randomization between hormonal (Arm 1) or chemotherapy (Arm 2). The confidence interval will be 95%. The proportion (and 95% confidence interval) of patients with RS 11-25 who refuse the assigned treatment will be calculated.
Outcome measures
| Measure |
Group 2 (RS 11-25)
n=33 Participants
Patients with an intermediate RS (11-25) were assigned to Group 2. The subject was then randomized to treatment Arm 1, neoadjuvant hormonal therapy, or treatment Arm 2, neoadjuvant chemotherapy.
|
|---|---|
|
The Proportion of Patients With RS 11-25 Who Refused the Assigned Treatment
|
0.15 proportion of participants
Interval 0.051 to 0.319
|
Adverse Events
Group 1 (RS < 11)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Group 2 Arm 1 (RS 11-25)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Group 2 Arm 2 (RS 11-25)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Group 3 (RS > 25)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Harry D Bear, MD, PhD
Virginia Commonwealth University/Massey Cancer Center
Phone: 804-828-9325
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place