NCT02648477

Brief Summary

This phase II trial studies how well pembrolizumab and doxorubicin hydrochloride works compared to pembrolizumab with anti-estrogen therapy (anastrozole, letrozole, or exemestane) in treating patients with triple-negative or hormone-receptor positive breast cancer that has spread from the primary site (place where it started) to other places in the body. Pembrolizumab is an antibody drug that blocks a molecule called programmed death (PD)-1. PD-1 is a molecule that shuts down the body's immune responses and prevents the immune system from attacking the cancer. Doxorubicin hydrochloride is a drug used in chemotherapy that works to stop the growth of tumor cells by stopping them from dividing and by causing them to die. Anti-estrogen therapy, including anastrozole, letrozole, and exemestane, lowers estrogen levels in the body, which may help treat cancer that is hormone receptor-positive. Giving pembrolizumab together with standard treatment of either doxorubicin hydrochloride (triple-negative cancer) or anti-estrogen therapy (hormone receptor-positive cancer) may be an effective treatment for these types of breast cancer.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Mar 2016

Longer than P75 for phase_2

Geographic Reach
1 country

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 5, 2016

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 7, 2016

Completed
3 months until next milestone

Study Start

First participant enrolled

March 28, 2016

Completed
6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2022

Completed
1 year until next milestone

Results Posted

Study results publicly available

April 19, 2023

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2023

Completed
Last Updated

March 19, 2024

Status Verified

February 1, 2024

Enrollment Period

6 years

First QC Date

January 5, 2016

Results QC Date

February 28, 2023

Last Update Submit

February 20, 2024

Conditions

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Overall Response

    Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR

    Up to 3 years

Secondary Outcomes (3)

  • Clinical Benefit Rate

    Up to 6 months

  • Overall Survival (OS)

    Up to 3 years

  • Progression-free Survival (PFS)

    Up to 3 years

Study Arms (2)

Cohort 1 (pembrolizumab, doxorubicin hydrochloride)

EXPERIMENTAL

Patients receive pembrolizumab IV over 30 minutes on day 1 and doxorubicin hydrochloride IV on day 1. Treatment repeats every 3 weeks for 6 courses, and then continues for up to 24 months with pembrolizumab alone in the absence of disease progression or unacceptable toxicity. Patients who stop pembrolizumab with stable disease or better may receive additional pembrolizumab therapy for up to 1 year if they progress after stopping study treatment.

Drug: Doxorubicin HydrochlorideOther: Laboratory Biomarker AnalysisBiological: Pembrolizumab

Cohort 2 (pembrolizumab, anti-estrogen therapy)

EXPERIMENTAL

Patients receive pembrolizumab IV over 30 minutes on day 1 and an aromatase inhibitor (exemestane, anastrozole, or letrozole) PO QD on days 1-21. Treatment repeats every 3 weeks for 24 months in the absence of disease progression or unacceptable toxicity. In both arms, patients who stop pembrolizumab with stable disease or better may receive additional pembrolizumab therapy for up to 1 year if they progress after stopping study treatment.

Drug: AnastrozoleDrug: ExemestaneOther: Laboratory Biomarker AnalysisDrug: LetrozoleBiological: Pembrolizumab

Interventions

Given PO

Also known as: Arimidex, ICI D1033, ICI-D1033, ZD-1033
Cohort 2 (pembrolizumab, anti-estrogen therapy)

Given IV

Also known as: 5,12-Naphthacenedione, 10-[(3-amino-2,3,6-trideoxy-alpha-L-lyxo-hexopyranosyl)oxy]-7,8, 9,10-tetrahydro-6,8,11-trihydroxy-8-(hydroxyacetyl)-1-methoxy-, hydrochloride, (8S-cis)- (9CI), ADM, Adriacin, Adriamycin, Adriamycin Hydrochloride, Adriamycin PFS, Adriamycin RDF, ADRIAMYCIN, HYDROCHLORIDE, Adriamycine, Adriblastina, Adriblastine, Adrimedac, Chloridrato de Doxorrubicina, DOX, DOXO-CELL, Doxolem, Doxorubicin.HCl, Doxorubin, Farmiblastina, FI 106, FI-106, hydroxydaunorubicin, Rubex
Cohort 1 (pembrolizumab, doxorubicin hydrochloride)

Given PO

Also known as: Aromasin, FCE-24304
Cohort 2 (pembrolizumab, anti-estrogen therapy)

Correlative studies

Cohort 1 (pembrolizumab, doxorubicin hydrochloride)Cohort 2 (pembrolizumab, anti-estrogen therapy)

Given PO

Also known as: CGS 20267, Femara
Cohort 2 (pembrolizumab, anti-estrogen therapy)
PembrolizumabBIOLOGICAL

Given IV

Also known as: Keytruda, Lambrolizumab, MK-3475, SCH 900475
Cohort 1 (pembrolizumab, doxorubicin hydrochloride)Cohort 2 (pembrolizumab, anti-estrogen therapy)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with 1) stage IV metastatic triple negative breast cancer (triple negative is defined as estrogen receptor \[ER\] and progesterone receptor \[PgR\] status is \< 1% of tumor cell nuclei are immunoreactive for ER or PgR, and HER2 status is fluorescence in situ hybridization \[FISH\] negative or immunohistochemistry \[IHC\] 0 or 1+), or 2) stage IV HR+ HER2- (HR+) breast cancer (defined as ER or PgR \> 1% of tumor cell nuclei are immunoreactive for ER or PgR and HER2 statis is FISH negative or IHC - or 1+)
  • Be willing and able to provide written informed consent/assent for the trial
  • Have measurable disease based on RECIST 1.1
  • Be willing to provide tissue from a newly obtained core or excisional biopsy of a tumor lesion; newly-obtained in defined as a specimen obtained up to 6 weeks (42 days) prior to initiation of treatment on day 1; subjects for whom newly-obtained samples cannot be provided (e.g. inaccessible or subject safety concern) may submit an archived specimen only upon agreement from the sponsor
  • Have a performance status of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) performance scale
  • Absolute neutrophil count (ANC) \>= 1,500/mcL
  • Platelets \>= 100,000/mcL
  • Hemoglobin \>= 9 g/dL or \>= 5.6 mmol/L without transfusion or erythropoietin (EPO) dependency (within 7 days of assessment)
  • Serum creatinine =\< 1.5 x upper limit of normal (ULN) OR measured or calculated creatinine clearance \>= 60 mL/min for subject with creatinine levels \> 1.5 x institutional ULN (glomerular filtration rate \[GFR\] can also be used in place of creatinine or creatinine clearance \[CrCl\]); creatinine clearance should be calculated per institutional standard
  • Left ventricular ejection fraction by multigated acquisition scan (MUGA) or echocardiogram \>= 55% for patients with triple negative breast cancer; \>= upper limit of institutional normal for patient with HR+ breast cancer
  • Serum total bilirubin =\< 1.5 x ULN OR direct bilirubin =\< ULN for subjects with total bilirubin levels \> 1.5 ULN
  • Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2.5 x ULN OR =\< 5 x ULN for subjects with liver metastases
  • Albumin \>= 2.5 mg/dL
  • International normalized ratio (INR) or prothrombin time (PT) =\< 1.5 x ULN unless subject is receiving anticoagulant therapy as long as PT or partial thromboplastin time (PTT) is within therapeutic range of intended use of anticoagulants
  • Activated partial thromboplastin time (aPTT) =\< 1.5 x ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants
  • +7 more criteria

You may not qualify if:

  • Cohort 1: Has triple negative breast cancer, is considered for cohort 1 participation, and received prior anthracycline therapy
  • Cohort 2: Has received prior aromatase inhibitor therapy and is deemed to be resistant to all three (anastrozole, letrozole, exemestane) approved AIs; resistance is defined as progression within 12 months or while on an AI
  • Patient is premenopausal (medical ovarian suppression is allowed); is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment
  • Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment
  • Has a known history of active TB (Bacillus tuberculosis)
  • Hypersensitivity to pembrolizumab or any of its excipients
  • Suboptimal cardiac function as defined by decreased left ventricular ejection fraction \< 55% for cohort 1, and \< 50% for cohort 2
  • Prior pembrolizumab
  • Has had a prior anti-cancer monoclonal antibody (mAb) within 4 weeks prior to study day 1 or who has not recovered (i.e., =\< grade 1 or at baseline) from adverse events due to agents administered more than 4 weeks earlier
  • Has had prior chemotherapy, targeted small molecule therapy, or radiation therapy within 2 weeks prior to study day 1 or who has not recovered (i.e., =\< grade 1 or at baseline) from adverse events due to a previously administered agent; Note: subjects with =\< grade 2 neuropathy are an exception to this criterion and may qualify for the study; Note: if subject received major surgery, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy
  • Has a known additional malignancy that has progressed or required active treatment in the past 5 years; exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer
  • Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis; subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of trial treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 7 days prior to trial treatment; this exception does not include carcinomatous meningitis which is excluded regardless of clinical stability
  • Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs); replacement therapy (e.g. thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment
  • Has a history of, active pneumonitis requiring treatment with steroids or history of/active interstitial lung disease
  • Has an active infection requiring systemic therapy
  • +8 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

City of Hope Corona

Corona, California, 92879, United States

Location

City of Hope Medical Center

Duarte, California, 91010, United States

Location

City of Hope Antelope Valley

Lancaster, California, 93534, United States

Location

City of Hope Rancho Cucamonga

Rancho Cucamonga, California, 91730, United States

Location

City of Hope South Pasadena

South Pasadena, California, 91030, United States

Location

City of Hope West Covina

West Covina, California, 91790, United States

Location

Related Publications (1)

  • Egelston CA, Guo W, Yost SE, Ge X, Lee JS, Frankel PH, Cui Y, Ruel C, Schmolze D, Murga M, Tang A, Martinez N, Karimi M, Somlo G, Lee PP, Waisman JR, Yuan Y. Immunogenicity and efficacy of pembrolizumab and doxorubicin in a phase I trial for patients with metastatic triple-negative breast cancer. Cancer Immunol Immunother. 2023 Sep;72(9):3013-3027. doi: 10.1007/s00262-023-03470-y. Epub 2023 Jun 9.

MeSH Terms

Conditions

Breast NeoplasmsTriple Negative Breast Neoplasms

Interventions

AnastrozoleDoxorubicinexemestaneLetrozolepembrolizumab

Condition Hierarchy (Ancestors)

Neoplasms by SiteNeoplasmsBreast DiseasesSkin DiseasesSkin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

NitrilesOrganic ChemicalsTriazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsDaunorubicinAnthracyclinesNaphthacenesPolycyclic Aromatic HydrocarbonsHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsPolycyclic CompoundsAminoglycosidesGlycosidesCarbohydrates

Results Point of Contact

Title
Paul Frankel, Ph.D.
Organization
City of Hope

Study Officials

  • James Waisman, MD

    City of Hope Medical Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 5, 2016

First Posted

January 7, 2016

Study Start

March 28, 2016

Primary Completion

April 1, 2022

Study Completion

December 30, 2023

Last Updated

March 19, 2024

Results First Posted

April 19, 2023

Record last verified: 2024-02

Locations