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Atorvastatin in Treating Patients With Stage IIb-III Triple Negative Breast Cancer Who Did Not Achieve a Pathologic Complete Response After Receiving Neoadjuvant Chemotherapy

Atorvastatin in Triple-Negative Breast Cancer (TNBC) Patients Who Did Not Achieve a Pathologic Complete Response (pCR) After Receiving Neoadjuvant Chemotherapy, a Multicenter Pilot Study

Status
Terminated
Phases
Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT03872388
Enrollment
6
Registered
2019-03-13
Start date
2019-09-23
Completion date
2023-10-19
Last updated
2024-10-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Anatomic Stage IIB Breast Cancer AJCC v8, Anatomic Stage IIIA Breast Cancer AJCC v8, Anatomic Stage IIIB Breast Cancer AJCC v8, Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IIIC Breast Cancer AJCC v8, Estrogen Receptor Negative, HER2/Neu Negative, Inflammatory Breast Carcinoma, Progesterone Receptor Negative, Prognostic Stage IIB Breast Cancer AJCC v8, Prognostic Stage IIIA Breast Cancer AJCC v8, Prognostic Stage IIIB Breast Cancer AJCC v8, Prognostic Stage III Breast Cancer AJCC v8, Prognostic Stage IIIC Breast Cancer AJCC v8, Stage IIB Breast Cancer AJCC v6 and v7, Stage IIIA Breast Cancer AJCC v7, Stage IIIB Breast Cancer AJCC v7, Stage III Breast Cancer AJCC v7, Stage IIIC Breast Cancer AJCC v7, Triple-Negative Breast Carcinoma

Brief summary

This phase II trial studies how well atorvastatin works in treating patients with stages IIb-III triple negative breast cancer who did not achieve a pathologic complete response to neoadjuvant chemotherapy. Pathologic complete response is the lack of all signs of cancer in tissue samples removed during surgery after upfront chemotherapy. Atorvastatin is used for the treatment of high cholesterol and may reduce the risk of triple negative breast cancer from coming back. Triple-negative breast cancer is a type of breast malignancy that is comprised of cancer cells that do not have estrogen receptors, progesterone receptors, or large amounts of HER2/neu protein. Patients with TNBC do not have established systemic therapies such as anti-estrogens or HER2-targeting agents to reduce recurrence after surgery, and residual cancer found at surgery is associated with higher relapse rate.

Detailed description

PRIMARY OBJECTIVES: I. To determine the proportion of patients with undetectable circulating tumor cells (CTCs) at 6 months in patients with stage IIB/III triple negative breast cancer (TNBC) who did not achieve a pathologic complete response a (pCR) or Residual Cancer Burden-I (RCB-I) after receiving neoadjuvant chemotherapy (NAC) with and without atorvastatin therapy. SECONDARY OBJECTIVES: I. To determine if baseline fasting lipid profile level (low density lipoprotein cholesterol \[LDL-C\]) and/or change in serum lipid levels are a predictive biomarker of change in the proportion of patients with CTCs. II. To assess effect of biomarkers on atorvastatin treatment response, defined as CTCs, circulating tumor deoxyribonucleic acid (DNA) (ctDNA), erythrocyte sedimentation rate (ESR), C-reactive protein (CRP), serum Interleukin-6 (IL-6) and other inflammatory cytokines, for the purpose of identifying the optimal patient population for future larger scale adjuvant studies. III. To determine if baseline fasting lipid profile level (LDL-C) and/or change in serum lipid levels are associated with 2-year relapse free survival (RFS) rate. IV. To determine if baseline CRP and/or change in serum lipid levels are a predictive biomarker of change in the proportion of patients with CTCs. V. To determine if baseline C-reactive protein (CRP) and/or change in CRP are associated with 2-year RFS rate. VI. To determine if baseline absolute number of CTCs and/or CTC change are associated with 2-year RFS rate. VII. To estimate the 2-year RFS rate of patients with TNBC who did not achieve pCR with and without atorvastatin therapy. VIII. To describe the toxicity and adverse events profile of atorvastatin treatment when given concurrently with standard doses of radiotherapy to the chest wall and regional nodes. EXPLORATORY OBJECTIVES: I. To evaluate the correlation between multiplexed imaging biomarkers in the normal or tumor tissue taken at the time of surgery, and response to atorvastatin-induced CTC changes or with measured outcomes. OUTLINE: Patients are assigned to 1 of 2 groups. GROUP I: Patients receive standard of care atorvastatin orally (PO) once daily (QD) for up to 24 months. A physical exam is performed, and blood drawn at 3, 6, 12, 18 and 24 months after starting standard of care treatment or at any time the disease appears to get worse. GROUP II: Patients not eligible to receive atorvastatin, will be enrolled into non-statin observation group with/without capecitabine treatment.

Interventions

DRUGAtorvastatin

Patients will receive Atorvastatin per ASCVD guidelines dosed as either a moderate intensity (20mg/day) or high intensity statin (80mg/day). Tablets are available in either 20mg or 80mg and will be dispensed as per standard of care for up to 24 months as part of the study. Patients may also receive capecitabine concurrently with Atorvastatin per physician discretion as standard of care. Standard of care dosing of Capecitabine is a 21 day cycle consisting 14 days on and 7 days off. The starting dosing will be per physician discretion, but may be up to 2500mg/m2 per day.

DRUGCapecitabine

Patients not eligible to receive atorvastatin in group II will be enrolled into non-statin observation group with/without capecitabine treatment. Capecitabine administration will be dosed as per standard of care. Standard of care dosing of Capecitabine is a 21 day cycle consisting 14 days on and 7 days off. The starting dosing will be per physician discretion, but may be up to 2500mg/m2 per day.

Sponsors

National Cancer Institute (NCI)
CollaboratorNIH
M.D. Anderson Cancer Center
Lead SponsorOTHER

Study design

Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No

Inclusion criteria

* Is willing and able to provide written informed consent for the trial * Diagnosis of TNBC (including patients with a clinical diagnosis of triple negative inflammatory breast cancer) * Has histological confirmation of breast carcinoma * Have stage IIB or III disease as defined by the American Joint Committee on Cancer version 7 or 8 * Has confirmed TNBC, defined as having estrogen and progesterone receptor \< 10% positivity by immunohistochemistry (IHC) and HER2 normal, which is 0 or 1+ by IHC and negative by fluorescence in situ hybridization (FISH) if performed or HER2 2+ by IHC and negative by FISH or HER2 negative by FISH if IHC is not performed * Received neoadjuvant chemotherapy and did not achieve pCR nor had an RCB-I (we will enroll patients with an RCB-II or RCB-III) following neoadjuvant chemotherapy. Since the RCB index has not been validated in IBC, any amount of residual disease will be allowed. pCR is defined as: a) the absence of residual invasive cancer on hematoxylin and eosin evaluation of the complete resected breast specimen and all sampled regional lymph nodes following completion of neoadjuvant systemic therapy (i.e., ypT0/Tis ypN0 in the current American Joint Committee on Cancer \[AJCC\] staging system). Or b) the absence of residual invasive and in situ cancer on hematoxylin and eosin evaluation of the complete resected breast specimen and all sampled regional lymph nodes following completion of neoadjuvant systemic therapy (i.e., ypT0 ypN0 in the current AJCC staging system) * Has a performance status of 0-1 on the Eastern Cooperative Oncology Group (ECOG) performance scale * Absolute neutrophil count (ANC) \>= 1,500/mcL * Platelets \>=100,000 /mcL * Hemoglobin (Hgb) \>= 8 g/dL * Creatinine levels \< 2.0 x upper limit of normal (ULN) * Total bilirubin =\< 1.5 x ULN * Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =\< 3.0 x ULN * Subjects of childbearing potential should be willing to use effective methods of birth control or be surgically sterile, or abstain from heterosexual activity for the course of the study through at least 4 months after the last dose of study drug; subjects of childbearing potential are those who have not been surgically sterilized or have not been free from menses for \> 1 year; effective methods of birth control include 1). Use of hormonal birth control methods: pills, shots/injections, implants (placed under the skin by a health care provider), or patches (placed on the skin); 2). Intrauterine devices (IUDs); 3). Using 2 barrier methods (each partner must use 1 barrier method) with a spermicide. Males must use the male condom (latex or other synthetic material) with spermicide. Females must choose either a diaphragm with spermicide, or cervical cap with spermicide, or a sponge (spermicide is already in the contraceptive sponge) * Within 3 months from completion of definitive surgery after neoadjuvant chemotherapy * Willing to take statin for minimum of two years

Exclusion criteria

* Has not recovered from adverse events due to prior therapies, i.e. monoclonal antibody, chemotherapy, targeted small molecule therapy, radiation therapy, or surgery * Note: Subjects with =\< grade 2 neuropathy, alopecia and general disorders and administration site conditions are an exception to this criterion and may qualify for the study * Has a known malignancy (other than breast cancer) except basal cell carcinoma or squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy * Has known psychiatric or substance abuse disorders and assessed by attending physician that would interfere with cooperation with the requirements of the trial * Has received prior therapy with a statin within past 6 months or is currently receiving statin therapy; patients who previously received a statin more than 6 months prior to beginning study therapy and who discontinued treatment for reasons other than severe toxicity or allergic reaction are eligible * Is currently receiving another anti-lipidemic agent other than statin: fibric acid derivatives (i.e. fenofibrate, gemfibrozil), bile acid sequestrants (i.e. cholestyramine, colestipol), ezetimibe, niacin, lovaza (omega-3-acid ethyl esters), red yeast rice, orlistat, phytosterol, and lomitapide * Known hypersensitivity to statin or any component of the formulation * Active liver disease or unexplained persistent elevations of serum transaminases, defined as elevated transaminases \> 3 x ULN on at least 2 separate occasions 1 week apart * Pregnancy or women who may become pregnant and not on acceptable form of contraception; lactating women * Has evidence of distant metastasis * Record of myocardial infarction within 6 months before starting therapy, symptomatic congestive heart failure (New York Heart Association \> class II), unstable angina, or unstable cardiac arrhythmia requiring medication * Chronic steroid use as this may prevent any immunomodulatory roles of statin treatment, defined as anticipating need of supraphysiologic dose of steroids for at least 12 weeks while on study

Design outcomes

Primary

MeasureTime frameDescription
Proportions of Patients With Undetectable Circulating Tumor Cells (CTC)At 6 monthsEstimate the proportion of patients with undetectable circulating tumor cells (CTCs) at 6 months in patients with stage IIB/III TNBC who did not achieve a pCR or Residual Cancer Burden-I (RCB-I) after receiving neoadjuvant chemotherapy (NAC) with and without atorvastatin therapy.

Secondary

MeasureTime frameDescription
Biomarkers on Atorvastatin Treatment ResponseUp to 2 yearsAssess effect of biomarkers on atorvastatin treatment response, defined as CTCs, circulating tumor DNA (ctDNA), erythrocyte sedimentation rate (ESR), C-reactive protein (CRP), serum Interleukin-6 (IL-6) and other inflammatory cytokines, for the purpose of identifying the optimal patient population for future larger scale adjuvant studies.
Fasting Lipid Profile Level (LDL-C) and 2-year RFS RateBaseline up to 2 yearsFasting lipid profile level (LDL-C) and 2-year RFS rate
Baseline C-reactive Protein (CRP) and/or Change in Serum Lipid LevelsBaseline up to 2 yearsDetermine if baseline CRP and/or change in serum lipid levels are a predictive biomarker of change in the proportion of patients with CTCs.
Fasting Lipid Profile Level (Low Density Lipoprotein Cholesterol [LDL-C]) and/or Change in Serum Lipid LevelsBaseline up to 2 yearsDetermine if baseline fasting lipid profile level (LDL-C) and/or change in serum lipid levels are a predictive biomarker of change in the proportion of patients with CTCs.
Recurrence-free Survival (RFS)At 2 yearsEstimate the 2-year RFS rate of patients with TNBC who did not achieve pCR with and without atorvastatin therapy.
Incidence of Adverse EventsUp to 24 monthsAdverse events, grade and relationship will be tabulated by treatment arms.
C-reactive Protein (CRP) and 2-year RFS RateBaseline up to 2 yearsDetermine if baseline C-reactive protein (CRP) and/or change in CRP are associated with 2-year RFS rate.
CTCs and 2-year RFS RateBaseline up to 2 yearsDetermine if baseline absolute number of CTCs and/or CTC change are associated with 2-year RFS rate.

Countries

United States

Participant flow

Recruitment details

September 2019- May 2022. All recruitment was done at The University of Texas MD Anderson Cancer Center.

Pre-assignment details

6 participants were accrued and assessed for eligibility and 4 patients were assigned to the cohort.

Participants by arm

ArmCount
Cohort 1a
Atorvastatin treatment starting at the beginning of radiation. No xeloda.
0
Cohort 1b
No Atorvastatin treatment, assessment before starting radiation, no xeloda.
0
Cohort 2a
Atorvastatin treatment after xeloda but starting concurrently with radiation.
0
Cohort 2b
No atorvastatin treatment, assessment after xeloda but prior to radiation.
0
Cohort 3a
Atorvastatin treatment after radiation and xeloda are complete, with optional pre-xeloda blood draw.
0
Cohort 3b
No atorvastatin treatment, assessment after radiation and xeloda are complete, with optional pre-xeloda timepoint
0
Cohort 4a
Atorvastatin treatment, after xeloda +/- radiation, enrolled after all other therapies are complete.
2
Cohort 4b
No atorvastatin treatment, assessment after xeloda +/- radiation, enrolled after all other therapies are complete.
2
Total4

Withdrawals & dropouts

PeriodReasonFG000FG001FG002FG003FG004FG005FG006FG007
Overall StudyDisease Progression00000011

Baseline characteristics

CharacteristicCohort 4aCohort 4bTotal
Age, Categorical
<=18 years
0 Participants0 Participants0 Participants
Age, Categorical
>=65 years
0 Participants0 Participants0 Participants
Age, Categorical
Between 18 and 65 years
2 Participants2 Participants4 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants0 Participants0 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
2 Participants2 Participants4 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Asian
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Black or African American
0 Participants0 Participants0 Participants
Race (NIH/OMB)
More than one race
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants0 Participants0 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants
Race (NIH/OMB)
White
2 Participants2 Participants4 Participants
Region of Enrollment
United States
2 participants2 participants4 participants
Sex: Female, Male
Female
2 Participants2 Participants4 Participants
Sex: Female, Male
Male
0 Participants0 Participants0 Participants

Adverse events

Event typeEG000
affected / at risk
EG001
affected / at risk
EG002
affected / at risk
EG003
affected / at risk
EG004
affected / at risk
EG005
affected / at risk
EG006
affected / at risk
EG007
affected / at risk
deaths
Total, all-cause mortality
0 / 00 / 00 / 00 / 00 / 00 / 01 / 20 / 2
other
Total, other adverse events
0 / 00 / 00 / 00 / 00 / 00 / 02 / 22 / 2
serious
Total, serious adverse events
0 / 00 / 00 / 00 / 00 / 00 / 00 / 20 / 2

Outcome results

Primary

Proportions of Patients With Undetectable Circulating Tumor Cells (CTC)

Estimate the proportion of patients with undetectable circulating tumor cells (CTCs) at 6 months in patients with stage IIB/III TNBC who did not achieve a pCR or Residual Cancer Burden-I (RCB-I) after receiving neoadjuvant chemotherapy (NAC) with and without atorvastatin therapy.

Time frame: At 6 months

Population: Data were not collected.

Secondary

Baseline C-reactive Protein (CRP) and/or Change in Serum Lipid Levels

Determine if baseline CRP and/or change in serum lipid levels are a predictive biomarker of change in the proportion of patients with CTCs.

Time frame: Baseline up to 2 years

Population: Data were not collected.

Secondary

Biomarkers on Atorvastatin Treatment Response

Assess effect of biomarkers on atorvastatin treatment response, defined as CTCs, circulating tumor DNA (ctDNA), erythrocyte sedimentation rate (ESR), C-reactive protein (CRP), serum Interleukin-6 (IL-6) and other inflammatory cytokines, for the purpose of identifying the optimal patient population for future larger scale adjuvant studies.

Time frame: Up to 2 years

Population: Data were not collected.

Secondary

C-reactive Protein (CRP) and 2-year RFS Rate

Determine if baseline C-reactive protein (CRP) and/or change in CRP are associated with 2-year RFS rate.

Time frame: Baseline up to 2 years

Population: Data were not collected.

Secondary

CTCs and 2-year RFS Rate

Determine if baseline absolute number of CTCs and/or CTC change are associated with 2-year RFS rate.

Time frame: Baseline up to 2 years

Population: Data were not collected.

Secondary

Fasting Lipid Profile Level (LDL-C) and 2-year RFS Rate

Fasting lipid profile level (LDL-C) and 2-year RFS rate

Time frame: Baseline up to 2 years

Population: Data were not collected.

Secondary

Fasting Lipid Profile Level (Low Density Lipoprotein Cholesterol [LDL-C]) and/or Change in Serum Lipid Levels

Determine if baseline fasting lipid profile level (LDL-C) and/or change in serum lipid levels are a predictive biomarker of change in the proportion of patients with CTCs.

Time frame: Baseline up to 2 years

Population: Data were not collected.

Secondary

Incidence of Adverse Events

Adverse events, grade and relationship will be tabulated by treatment arms.

Time frame: Up to 24 months

Population: Only 1 patient who was treated with atorvastatin came for any follow up appointments, however she missed the 6 month timepoint which is the primary end-point of the study. Therefore she was deemed inevaluable. One other treated patient started treatment and died before the 3 month follow up visit, and hence no data was obtained on any patients for cohort 1-3.

ArmMeasureGroupValue (NUMBER)
Cohort 4aIncidence of Adverse EventsGrade 14 events
Cohort 4aIncidence of Adverse EventsGrade 20 events
Cohort 4bIncidence of Adverse EventsGrade 112 events
Cohort 4bIncidence of Adverse EventsGrade 23 events
Secondary

Recurrence-free Survival (RFS)

Estimate the 2-year RFS rate of patients with TNBC who did not achieve pCR with and without atorvastatin therapy.

Time frame: At 2 years

Population: Data were not collected.

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026