NCT03219476

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

Completed Phase 2 Results posted Last updated 29 April 2025

What this trial tests

Phase 2 trial testing Anastrozole in Breast Cancer in 37 participants. Completed in 20 March 2025.

Timeline

5 February 2017

Primary endpoint
4 February 2020

20 March 2025

Quick facts

Lead sponsor	Medical College of Wisconsin
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	37
Start date	5 February 2017
Primary completion	4 February 2020
Estimated completion	20 March 2025
Sites	1 location across United States

Drugs / interventions tested

• Anastrozole (anastrozole) — full drug profile →
• Letrozole — full drug profile →
• Exemestane (exemestane) — full drug profile →
• Tamoxifen

Conditions studied

• Breast Cancer — all drugs for Breast Cancer →
• Invasive Breast Cancer — all drugs for Invasive Breast Cancer →

Sponsor

Medical College of Wisconsin

Who can join

18 and older, female only, with Breast Cancer or Invasive Breast Cancer. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change in Baseline of Cancer Cell Protein Levels of Human Epidermal Growth Factor Receptor (HER) Family Members (HER1-4) Following Neoadjuvant Endocrine Therapy. Primary · At four weeks.

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 sta

Upregulation

Group	Value	95% CI
Change From Baseline in Human Epidermal Growth Factor Receptor 1 (HER1) Cell Protein Levels.	1
Change From Baseline in Human Epidermal Growth Factor Receptor 2 (HER2) Cell Protein Levels.	17
Change From Baseline in Human Epidermal Growth Factor Receptor 3 (HER3) Cell Protein Levels.	1
Change From Baseline in Human Epidermal Growth Factor Receptor 4 (HER4) Cell Protein Levels.	1

Downregulation

Group	Value	95% CI
Change From Baseline in Human Epidermal Growth Factor Receptor 1 (HER1) Cell Protein Levels.	0
Change From Baseline in Human Epidermal Growth Factor Receptor 2 (HER2) Cell Protein Levels.	2
Change From Baseline in Human Epidermal Growth Factor Receptor 3 (HER3) Cell Protein Levels.	18
Change From Baseline in Human Epidermal Growth Factor Receptor 4 (HER4) Cell Protein Levels.	18

No Change

Group	Value	95% CI
Change From Baseline in Human Epidermal Growth Factor Receptor 1 (HER1) Cell Protein Levels.	36
Change From Baseline in Human Epidermal Growth Factor Receptor 2 (HER2) Cell Protein Levels.	18
Change From Baseline in Human Epidermal Growth Factor Receptor 3 (HER3) Cell Protein Levels.	18
Change From Baseline in Human Epidermal Growth Factor Receptor 4 (HER4) Cell Protein Levels.	18

Number of Subjects Who Achieve Complete Radiographic Response. Secondary · 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% incr

Group	Value	95% CI
Neoadjuvant Endocrine Therapy Treatment (Physician's Choice)	0

Number of Subjects Who Achieve a Partial Radiographic Response. Secondary · 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% incr

Group	Value	95% CI
Neoadjuvant Endocrine Therapy Treatment (Physician's Choice)	1

Adverse events — posted to ClinicalTrials.gov

Time frame: Four weeks.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Neoadjuvant Endocrine Therapy Treatment (Physician's Choice)

Serious: 1/37 (3%)

Deaths: 1/37

Serious adverse events (1 terms)

Reaction	System	Neoadjuvant Endocrine Ther…
Infection	Infections and infestations	—

Other adverse events (2 terms — click to expand)

Reaction	System	Neoadjuvant Endocrine Ther…
Arthralgia	Musculoskeletal and connective tissue disorders	—
Hypertension	Vascular disorders	—

Most-reported serious reactions: Infection.

Data from ClinicalTrials.gov NCT03219476 adverse events section.

Sponsor's own description

This is an exploratory interventional study that initiates standard-of-care anti-estrogen treatment preoperatively for four weeks.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Frequent upregulation of HER2 protein in hormone-receptor-positive HER2-negative breast cancer after short-term neoadjuvant endocrine therapy.
   Chaudhary LN, Jorns JM, Sun Y, Cheng YC, et al · · 2023 · cited 7× · PMID 37460683 · DOI 10.1007/s10549-023-07038-3
2. Frequent Upregulation Of HER2 Protein In Hormone Receptor-Positive HER2-Negative Breast Cancer After Short-Term Neoadjuvant Endocrine Therapy
   Chaudhary LN, Jorns J, Sun Y, Cheng YC, et al · · 2023 · DOI 10.21203/rs.3.rs-2777910/v1