Neoadjuvant Darolutamide Alone or in Combination With Standard Therapy for Stage II-IIIA, AR+, TNBC

NCT ID: NCT07016399

Last Updated: 2025-10-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

51 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-09-09

Study Completion Date

2033-10-31

Brief Summary

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This phase II trial compares the effect of adding darolutamide to standard therapy versus standard therapy alone before surgery for the treatment of patients with stage II-IIIA androgen receptor positive triple-negative breast carcinoma. Standard therapy before surgery for triple-negative breast cancer typically consists of a combination of chemotherapy and immunotherapy drugs. Chemotherapy drugs, such as carboplatin, paclitaxel, doxorubicin and cyclophosphamide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Darolutamide is in a class of medications called androgen receptor inhibitors. It works by blocking the effects of androgen (a male reproductive hormone) to stop the growth and spread of tumor cells. Giving darolutamide in combination with standard therapy before surgery may make the tumor smaller and may reduce the amount of normal tissue that needs to be removed.

Detailed Description

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PRIMARY OBJECTIVE:

I. To compare the mean ΔKi-67 level between the darolutamide therapy arm and the control arm.

SECONDARY OBJECTIVES:

I. To determine the pathologic complete response rate (pCR) and overall response rate (ORR) in patients on the darolutamide and control arms.

II. To determine event-free survival (EFS) in patients with androgen receptor positive (AR+) breast cancer in both arms.

III. To correlate the change in Ki-67 at 2 weeks and 6 months with pCR rates and EFS in patients with AR+ breast cancer in both arms.

IV. To monitor circulating tumor deoxyribonucleic acid (ctDNA) throughout treatment and correlate with response in both arms.

V. To correlate percent of nuclear AR positivity with pCR and EFS in both arms.

EXPLORATORY OBJECTIVES:

I. To determine if patients with triple-negative breast cancer (TNBC) experience changes in ctDNA levels while undergoing neoadjuvant therapy.

II. To assess if changes in ctDNA levels correlate with response to neoadjuvant therapy observed in breast cancer tissue biopsies/surgical specimens.

III. To identify AR amplification and ligand binding mutations by whole exome sequencing and evaluate AR transcription targets and AR splice variants by ribonucleic acid sequencing (RNAseq) in baseline, week 2 and residual disease of patients on the darolutamide arm.

IV. To evaluate cell populations and AR transcriptional activity by single-cell RNAseq (scRNAseq) using baseline, week-2 biopsies and residual disease from patients on the darolutamide arm.

V. To assess tumor microenvironment changes in hormone receptors and immune cell populations by multiple immunofluorescences using the CO-Detection by indEXing (CODEX) platform.

VI. To evaluate AR-independent mechanisms of resistance to AR inhibitors using in vitro models for the discovery phase and human tissue for validation studies.

OUTLINE: Patients are randomized to 1 of 2 arms.

ARM A: Patients receive pembrolizumab intravenously (IV) over 30 minutes on day 1 of each cycle, paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin IV on days 1, 8, and 15 of each cycle. Cycles repeat every 21 days for up to 4 cycles (cycles 1-4) in the absence of disease progression or unacceptable toxicity. Then, patients receive pembrolizumab IV over 30 minutes, cyclophosphamide IV, and doxorubicin IV or epirubicin IV on day 1 of subsequent cycles. Cycles repeat every 21 days for up to an additional 4 cycles (cycles 5-8) in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo surgery on study, as well as ultrasound (US) or magnetic resonance imaging (MRI), blood sample collection, and breast biopsies throughout the study.

ARM B: Patients receive darolutamide orally (PO) twice daily (BID) for 14 days in the absence of disease progression or unacceptable toxicity. Patients then receive darolutamide PO BID, pembrolizumab IV over 30 minutes on day 1 of each cycle, and paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin IV on days 1, 8, and 15 of each cycle. Cycles repeat every 21 days for up to 4 cycles (cycles 1-4) in the absence of disease progression or unacceptable toxicity. Then, patients receive pembrolizumab IV over 30 minutes, cyclophosphamide IV, and doxorubicin IV or epirubicin IV on day 1 of subsequent cycles. Cycles repeat every 21 days for up to an additional 4 cycles (cycles 5-8) in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo surgery on study, as well as US or MRI, blood sample collection, and breast biopsies throughout the study.

After completion of study treatment, patients are followed up after 30-37 days, at 6 months, 12 months, then yearly for up to 5 years.

Conditions

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Anatomic Stage II Breast Cancer AJCC v8 Anatomic Stage IIIA Breast Cancer AJCC v8 Triple-Negative Breast Carcinoma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm A (Standard chemotherapy + immunotherapy)

Patients receive pembrolizumab IV over 30 minutes on day 1 of each cycle, paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin IV on days 1, 8, and 15 of each cycle. Cycles repeat every 21 days for up to 4 cycles (cycles 1-4) in the absence of disease progression or unacceptable toxicity. Then, patients receive pembrolizumab IV over 30 minutes, cyclophosphamide IV, and doxorubicin IV or epirubicin IV on day 1 of subsequent cycles. Cycles repeat every 21 days for up to an additional 4 cycles (cycles 5-8) in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo surgery on study, as well as US or MRI, blood sample collection, and breast biopsies throughout the study.

Group Type ACTIVE_COMPARATOR

Biospecimen Collection

Intervention Type PROCEDURE

Undergo blood sample collection

Breast Biopsy Procedure

Intervention Type PROCEDURE

Undergo breast biopsies

Carboplatin

Intervention Type DRUG

Given IV

Cyclophosphamide

Intervention Type DRUG

Given IV

Doxorubicin

Intervention Type DRUG

Given IV

Epirubicin

Intervention Type DRUG

Given IV

Magnetic Resonance Imaging

Intervention Type PROCEDURE

Undergo MRI

Paclitaxel

Intervention Type DRUG

Given IV

Pembrolizumab

Intervention Type BIOLOGICAL

Given IV

Surgical Procedure

Intervention Type PROCEDURE

Undergo breast surgery

Ultrasound Imaging

Intervention Type PROCEDURE

Undergo US

Arm B (Standard chemotherapy + immunotherapy + darolutamide)

Patients receive darolutamide PO BID for 14 days in the absence of disease progression or unacceptable toxicity. Patients then receive darolutamide PO BID, pembrolizumab IV over 30 minutes on day 1 of each cycle, and paclitaxel IV on days 1, 8, and 15 of each cycle, and carboplatin IV on days 1, 8, and 15 of each cycle. Cycles repeat every 21 days for up to 4 cycles (cycles 1-4) in the absence of disease progression or unacceptable toxicity. Then, patients receive pembrolizumab IV over 30 minutes, cyclophosphamide IV, and doxorubicin IV or epirubicin IV on day 1 of subsequent cycles. Cycles repeat every 21 days for up to an additional 4 cycles (cycles 5-8) in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo surgery on study, as well as US or MRI, blood sample collection, and breast biopsies throughout the study.

Group Type EXPERIMENTAL

Biospecimen Collection

Intervention Type PROCEDURE

Undergo blood sample collection

Breast Biopsy Procedure

Intervention Type PROCEDURE

Undergo breast biopsies

Carboplatin

Intervention Type DRUG

Given IV

Cyclophosphamide

Intervention Type DRUG

Given IV

Darolutamide

Intervention Type DRUG

Given PO

Doxorubicin

Intervention Type DRUG

Given IV

Epirubicin

Intervention Type DRUG

Given IV

Magnetic Resonance Imaging

Intervention Type PROCEDURE

Undergo MRI

Paclitaxel

Intervention Type DRUG

Given IV

Pembrolizumab

Intervention Type BIOLOGICAL

Given IV

Surgical Procedure

Intervention Type PROCEDURE

Undergo breast surgery

Ultrasound Imaging

Intervention Type PROCEDURE

Undergo US

Interventions

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Biospecimen Collection

Undergo blood sample collection

Intervention Type PROCEDURE

Breast Biopsy Procedure

Undergo breast biopsies

Intervention Type PROCEDURE

Carboplatin

Given IV

Intervention Type DRUG

Cyclophosphamide

Given IV

Intervention Type DRUG

Darolutamide

Given PO

Intervention Type DRUG

Doxorubicin

Given IV

Intervention Type DRUG

Epirubicin

Given IV

Intervention Type DRUG

Magnetic Resonance Imaging

Undergo MRI

Intervention Type PROCEDURE

Paclitaxel

Given IV

Intervention Type DRUG

Pembrolizumab

Given IV

Intervention Type BIOLOGICAL

Surgical Procedure

Undergo breast surgery

Intervention Type PROCEDURE

Ultrasound Imaging

Undergo US

Intervention Type PROCEDURE

Other Intervention Names

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Biological Sample Collection Biospecimen Collected Specimen Collection Breast Biopsy Blastocarb Carboplat Carboplatin Hexal Carboplatino Carboplatinum Carbosin Carbosol Carbotec CBDCA Displata Ercar JM-8 JM8 Nealorin Novoplatinum Paraplatin Paraplatin AQ Paraplatine Platinwas Ribocarbo (-)-Cyclophosphamide 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate Asta B 518 B 518 B-518 B518 Carloxan Ciclofosfamida Ciclofosfamide Cicloxal Clafen Claphene CP monohydrate CTX CYCLO-cell Cycloblastin Cycloblastine Cyclophospham Cyclophosphamid monohydrate Cyclophosphamide Monohydrate Cyclophosphamidum Cyclophosphan Cyclophosphane Cyclophosphanum Cyclostin Cyclostine Cytophosphan Cytophosphane Cytoxan Fosfaseron Genoxal Genuxal Ledoxina Mitoxan Neosar Revimmune Syklofosfamid WR 138719 WR- 138719 WR-138719 WR138719 Antiandrogen ODM-201 BAY 1841788 BAY-1841788 BAY1841788 Nubeqa ODM 201 ODM-201 ODM201 Adriablastin Hydroxydaunomycin Hydroxyl Daunorubicin Hydroxyldaunorubicin 4'-epi DX 4'-Epiadriamycin 4'-Epidoxorubicin Epi DX Epidoxorubicin Pidorubicin Magnetic Resonance Magnetic Resonance Imaging (MRI) Magnetic resonance imaging (procedure) Magnetic Resonance Imaging Scan Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance MR MR Imaging MRI MRI Scan MRIs NMR Imaging NMRI Nuclear Magnetic Resonance Imaging sMRI Structural MRI Anzatax Asotax Bristaxol Praxel Taxol Taxol Konzentrat BCD-201 GME 751 GME751 Keytruda Lambrolizumab MK 3475 MK-3475 MK3475 Pembrolizumab Biosimilar BCD-201 Pembrolizumab Biosimilar GME751 Pembrolizumab Biosimilar QL2107 Pembrolizumab Biosimilar RPH-075 Pembrolizumab Biosimilar SB27 QL2107 RPH 075 RPH-075 RPH075 SB 27 SB-27 SB27 SCH 900475 SCH-900475 SCH900475 Operation Surgery Surgery Type Surgery, NOS Surgical Surgical Intervention Surgical Interventions Surgical Procedures Type of Surgery 2-Dimensional Grayscale Ultrasound Imaging 2-Dimensional Ultrasound Imaging 2D-US Ultrasonography Ultrasound Ultrasound Test Ultrasound, Medical US

Eligibility Criteria

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Inclusion Criteria

* Signed and dated written informed consent as well as the ability to understand and the willingness to sign written consent prior to study registration
* Male or female ≥ 18 years of age on the day of signing informed consent
* Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
* Histologically confirmed newly diagnosed breast cancer with the following requirements:

* \<10% staining for estrogen receptor (ER) and progesterone receptor (PR) by immunohistochemistry (IHC)
* HER2 negative by fluorescence in situ hybridization (FISH)
* AR positive: defined as ≥ 80% staining for AR by IHC
* Primary tumor clinically or radiographically ≥ 1cm in size or stage II-IIIA and eligible for neoadjuvant treatment
* Absolute neutrophil count (ANC) ≥ 1500/µL (≤ 28 days prior to first dose of protocol-indicated treatment)
* Platelets ≥ 100,000/µL (≤ 28 days prior to first dose of protocol-indicated treatment)
* Hemoglobin ≥ 9.0 g/dL or ≥ 5.6 mmol/L (≤ 28 days prior to first dose of protocol-indicated treatment)
* Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min (as calculated by the Cockcroft-Gault Formula or calculated/measured by an alternative established institutional standard consistently applied across participants at the site) (≤ 28 days prior to first dose of protocol-indicated treatment)
* Total bilirubin ≤ 1.5 times institutional upper limit of normal (ULN), or direct bilirubin ≤ ULN for participants with total bilirubin \> 1.5 x ULN (≤ 28 days prior to first dose of protocol-indicated treatment)
* Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\]) and alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) ≤ 2.5 times institutional upper limit of normal (ULN) (≤ 28 days prior to first dose of protocol-indicated treatment)
* Calcium ≤ 11.5 mg/dL or ≤ 2.9 mmol/L; in patients with albumin outside the normal range, calcium (corrected for albumin) must be ≤ 11.5 mg/dL or ≤ 2.9 mmol/L (≤ 28 days prior to first dose of protocol-indicated treatment)
* Women must not be breastfeeding and further agree to not breastfeed during study treatment and for at least 120 days after completion of treatment
* A woman of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test during screening within 21 days prior to receiving first dose of protocol-indicated treatment, and must agree to follow instructions for using acceptable contraception from the time of signing consent, and until at least 120 days after completion of treatment
* Men must refrain from donating sperm for at least 120 days after completion of treatment
* A man able to father children who is sexually active with a WOCBP must agree to follow instructions for using acceptable contraception, from the time of signing consent, and until at least 120 days after completion of treatment

Exclusion Criteria

* Non-resectable breast cancer as assessed by the primary treating surgeon or evidence of metastatic disease
* Malignancies other than TNBC within 5 years prior to randomization, with the exception of those with a negligible risk of metastases or death and treated with expected curative outcome (such as adequately treated carcinoma in situ of the cervix or basal or squamous cell skin cancer)
* Patient is pregnant or breastfeeding
* Patients with moderate hepatic impairment (Child-Pugh Class B cirrhosis or higher)
* Is currently participating in or within four weeks prior to receiving first dose of study treatment in a study of an investigational agent or investigational device

* Participants who have entered the follow-up phase of an investigational study may participate as long as it has been four weeks after the last dose or last exposure to the previous investigational agent or investigational device
* Recipient of previous allogeneic tissue/solid organ transplant
* Known severe hypersensitivity (≥ Grade 3) to study drug, pembrolizumab, carboplatin, doxorubicin/epirubicin, paclitaxel, or cyclophosphamide and/or any of the excipients of these drugs
* History of myocarditis or pericarditis or other known underlying heart disease that is clinically significant by investigator judgment (for example, cardiomyopathy, congestive heart failure with New York Heart Association \[NYHA\] functional classification III or IV, symptomatic arrhythmia not controlled by medication, unstable angina, history of acute myocardial infarction). History of cerebrovascular accident (including transient ischemic attack \[TIA\]) within the past six months (24 weeks) prior to starting study treatment
* Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection/sepsis, or psychiatric illness/social situations that would limit compliance with study requirements
* Known conditions that would preclude the use of checkpoint inhibitors
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Vandana Abramson

OTHER

Sponsor Role lead

Responsible Party

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Vandana Abramson

Principal Investigator

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Vandana G Abramson

Role: PRINCIPAL_INVESTIGATOR

Vanderbilt University/Ingram Cancer Center

Locations

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Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, United States

Site Status RECRUITING

Countries

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United States

Facility Contacts

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Vandana G. Abramson

Role: primary

615-936-8422

Other Identifiers

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NCI-2025-03600

Identifier Type: REGISTRY

Identifier Source: secondary_id

VICC-VCBRE23490

Identifier Type: OTHER

Identifier Source: secondary_id

P30CA068485

Identifier Type: NIH

Identifier Source: secondary_id

View Link

VICC-VCBRE23490

Identifier Type: -

Identifier Source: org_study_id

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