Trial Outcomes & Findings for Neoadjuvant Endocrine Therapy in Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor 2 (HER2)-Negative Node-Negative Breast Cancer (NCT NCT03219476)

NCT ID: NCT03219476

Last Updated: 2025-04-29

Results Overview

ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of HER2 receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+ (up to 25% of cells stained), it's called "HER2 negative." If the score is 2+ (approximately 50% of cells stained), it's called "borderline." A score of 3+ (approximately 75% or more of cells stained) is called "HER2 positive." Upregulation means increased staining from a prior observation; Downregulation means decreased staining from a prior observation; and No Change means similar staining from a prior observation.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

37 participants

Primary outcome timeframe

At four weeks.

Results posted on

2025-04-29

Participant Flow

Participant milestones

Participant milestones
Measure
Neoadjuvant Endocrine Therapy Treatment (Physician's Choice)
Once enrolled, patients would be treated with the current standard-of-care endocrine therapy. Choice of endocrine therapy (aromatase inhibitors or tamoxifen) would be decided by medical oncologist, following a review of the patient's medical history and menstrual status. The patient would be treated with endocrine therapy in a neoadjuvant setting for four weeks, with dosing continuing until surgery.
Overall Study
STARTED
37
Overall Study
COMPLETED
37
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Neoadjuvant Endocrine Therapy in Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor 2 (HER2)-Negative Node-Negative Breast Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Neoadjuvant Endocrine Therapy Treatment (Physician's Choice)
n=37 Participants
Once enrolled, patients would be treated with the current standard-of-care endocrine therapy. Choice of endocrine therapy (aromatase inhibitors or tamoxifen) would be decided by medical oncologist, following a review of the patient's medical history and menstrual status. The patient would be treated with endocrine therapy in a neoadjuvant setting for four weeks, with dosing continuing until surgery. Anastrozole: 1 mg once daily. Letrozole: 2.5mg once daily. Exemestane: 25 mg once daily. Tamoxifen: 20 mg once daily.
Age, Categorical
<=18 years
0 Participants
n=39 Participants
Age, Categorical
Between 18 and 65 years
25 Participants
n=39 Participants
Age, Categorical
>=65 years
12 Participants
n=39 Participants
Sex: Female, Male
Female
37 Participants
n=39 Participants
Sex: Female, Male
Male
0 Participants
n=39 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants
n=39 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
36 Participants
n=39 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=39 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=39 Participants
Race (NIH/OMB)
Asian
0 Participants
n=39 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=39 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=39 Participants
Race (NIH/OMB)
White
36 Participants
n=39 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=39 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=39 Participants
Region of Enrollment
United States
37 participants
n=39 Participants

PRIMARY outcome

Timeframe: At four weeks.

Population: All 37 subjects were analyzed for each of the protein markers.

ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of HER2 receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+ (up to 25% of cells stained), it's called "HER2 negative." If the score is 2+ (approximately 50% of cells stained), it's called "borderline." A score of 3+ (approximately 75% or more of cells stained) is called "HER2 positive." Upregulation means increased staining from a prior observation; Downregulation means decreased staining from a prior observation; and No Change means similar staining from a prior observation.

Outcome measures

Outcome measures
Measure
Change From Baseline in Human Epidermal Growth Factor Receptor 1 (HER1) Cell Protein Levels.
n=37 Participants
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 1 (HER1) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER1 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER1 positive."
Change From Baseline in Human Epidermal Growth Factor Receptor 2 (HER2) Cell Protein Levels.
n=37 Participants
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 2 (HER2) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER2 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER2 positive."
Change From Baseline in Human Epidermal Growth Factor Receptor 3 (HER3) Cell Protein Levels.
n=37 Participants
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 3 (HER3) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER3 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER3 positive."
Change From Baseline in Human Epidermal Growth Factor Receptor 4 (HER4) Cell Protein Levels.
n=37 Participants
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 4 (HER4) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER4 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER4 positive."
Change in Baseline of Cancer Cell Protein Levels of Human Epidermal Growth Factor Receptor (HER) Family Members (HER1-4) Following Neoadjuvant Endocrine Therapy.
Upregulation
1 Participants
17 Participants
1 Participants
1 Participants
Change in Baseline of Cancer Cell Protein Levels of Human Epidermal Growth Factor Receptor (HER) Family Members (HER1-4) Following Neoadjuvant Endocrine Therapy.
Downregulation
0 Participants
2 Participants
18 Participants
18 Participants
Change in Baseline of Cancer Cell Protein Levels of Human Epidermal Growth Factor Receptor (HER) Family Members (HER1-4) Following Neoadjuvant Endocrine Therapy.
No Change
36 Participants
18 Participants
18 Participants
18 Participants

SECONDARY outcome

Timeframe: At four weeks.

This will be assessed by World Health Organization (WHO) criteria: * Complete Response (CR): The disappearance of all known disease, based on a comparison between the measurements at baseline and after four weeks of treatment with neoadjuvant therapy. * Partial Response (PR): A 50% or greater decrease in the product of the bi-dimensional measurements of the lesion (total tumor size), based on a comparison between the measurements at baseline and after four weeks of treatment with neoadjuvant therapy. * No Change (NC): A 50% decrease in total tumor size cannot be established nor has a 25% increase in the size of the lesion been demonstrated. * Progressive Disease (PD): A 25% or greater increase in the total tumor size of the measurable lesions (calculated on the smallest diameter recorded over time).

Outcome measures

Outcome measures
Measure
Change From Baseline in Human Epidermal Growth Factor Receptor 1 (HER1) Cell Protein Levels.
n=37 Participants
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 1 (HER1) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER1 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER1 positive."
Change From Baseline in Human Epidermal Growth Factor Receptor 2 (HER2) Cell Protein Levels.
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 2 (HER2) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER2 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER2 positive."
Change From Baseline in Human Epidermal Growth Factor Receptor 3 (HER3) Cell Protein Levels.
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 3 (HER3) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER3 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER3 positive."
Change From Baseline in Human Epidermal Growth Factor Receptor 4 (HER4) Cell Protein Levels.
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 4 (HER4) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER4 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER4 positive."
Number of Subjects Who Achieve Complete Radiographic Response.
0 Participants

SECONDARY outcome

Timeframe: At four weeks.

This will be assessed by World Health Organization (WHO) criteria: * Complete Response (CR): The disappearance of all known disease, based on a comparison between the measurements at baseline and after four weeks of treatment with neoadjuvant therapy. * Partial Response (PR): A 50% or greater decrease in the product of the bi-dimensional measurements of the lesion (total tumor size), based on a comparison between the measurements at baseline and after four weeks of treatment with neoadjuvant therapy. * No Change (NC): A 50% decrease in total tumor size cannot be established nor has a 25% increase in the size of the lesion been demonstrated. * Progressive Disease (PD): A 25% or greater increase in the total tumor size of the measurable lesions (calculated on the smallest diameter recorded over time).

Outcome measures

Outcome measures
Measure
Change From Baseline in Human Epidermal Growth Factor Receptor 1 (HER1) Cell Protein Levels.
n=37 Participants
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 1 (HER1) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER1 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER1 positive."
Change From Baseline in Human Epidermal Growth Factor Receptor 2 (HER2) Cell Protein Levels.
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 2 (HER2) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER2 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER2 positive."
Change From Baseline in Human Epidermal Growth Factor Receptor 3 (HER3) Cell Protein Levels.
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 3 (HER3) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER3 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER3 positive."
Change From Baseline in Human Epidermal Growth Factor Receptor 4 (HER4) Cell Protein Levels.
ImmunoHistoChemistry (IHC) will be used. The IHC test gives a score of 0 to 3+ that measures the amount of Human Epidermal Growth Factor Receptor 4 (HER4) receptor protein on the surface of cells in a breast cancer tissue sample. If the score is 0 to 1+, it's called "HER4 negative." If the score is 2+, it's called "borderline." A score of 3+ is called "HER4 positive."
Number of Subjects Who Achieve a Partial Radiographic Response.
1 Participants

Adverse Events

Neoadjuvant Endocrine Therapy Treatment (Physician's Choice)

Serious events: 1 serious events
Other events: 2 other events
Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure
Neoadjuvant Endocrine Therapy Treatment (Physician's Choice)
n=37 participants at risk
Once enrolled, patients would be treated with the current standard-of-care endocrine therapy. Choice of endocrine therapy (aromatase inhibitors or tamoxifen) would be decided by medical oncologist, following a review of the patient's medical history and menstrual status. The patient would be treated with endocrine therapy in a neoadjuvant setting for four weeks, with dosing continuing until surgery.
Infections and infestations
Infection
2.7%
1/37 • Number of events 1 • Four weeks.

Other adverse events

Other adverse events
Measure
Neoadjuvant Endocrine Therapy Treatment (Physician's Choice)
n=37 participants at risk
Once enrolled, patients would be treated with the current standard-of-care endocrine therapy. Choice of endocrine therapy (aromatase inhibitors or tamoxifen) would be decided by medical oncologist, following a review of the patient's medical history and menstrual status. The patient would be treated with endocrine therapy in a neoadjuvant setting for four weeks, with dosing continuing until surgery.
Musculoskeletal and connective tissue disorders
Arthralgia
5.4%
2/37 • Number of events 2 • Four weeks.
Vascular disorders
Hypertension
5.4%
2/37 • Number of events 2 • Four weeks.

Additional Information

Lubna Chaudhary

Medical College of Wisconsin

Phone: 414-805-4600

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place