Neratinib +/- Fulvestrant in Metastatic HER2 Non-amplified But HER2 Mutant Breast Cancer

NCT ID: NCT01670877

Last Updated: 2022-04-15

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

56 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-12-11

Study Completion Date

2021-03-11

Brief Summary

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This phase II study will test cancer to see if it has a HER2 mutation and, if so, see how HER2 mutated cancer responds to treatment with neratinib.

Detailed Description

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Overexpression of HER2 due to gene amplification is an established therapeutic target in breast cancer for which multiple HER2 targeted drugs are now available. However, the majority of breast cancers are without HER2 overexpression/non-amplified and not currently eligible to receive HER2 targeted drugs. Advances in tumor genome sequencing technology led to the identification of recurrent HER2 mutations (HER2mut) in approximately 2% of HER2 non-amplified primary breast cancers, and 3-5% of metastatic tumors. Importantly, tumor cells harboring HER2mut are sensitive to the anti-tumor effects of HER2-targeted agents in preclinical models, especially neratinib, a potent irreversible pan-HER inhibitor. However, neratinib monotherapy has demonstrated only modest single agent activity in HER2mut,, non-amplified metastatic breast cancer (MBC). Based on the hypothesis that the combination of neratinib and fulvestrant will be more effective than neratinib alone in ER+/HER2mut, non-amplified MBC the investigators conducted a single arm phase II study of neratinib plus fulvestrant with 2 cohorts, fulvestrant (FUL)-treated and FUL-naïve, for patients with ER+/HER2mut, non-amplified MBC to assess the anti-tumor effects of this combination. An exploratory ER-negative (ER-) HER2mut cohort was also included for the efficacy of neratinib monotherapy.

Conditions

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Breast Neoplasms

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Part I: Neratinib Only

-Patients will receive neratinib PO daily on Days 1-28. Each cycle is 4 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.

Group Type EXPERIMENTAL

Neratinib

Intervention Type DRUG

Tumor biopsy

Intervention Type PROCEDURE

-Optional at baseline and disease progression

Research blood sample

Intervention Type PROCEDURE

-Baseline, cycle 1 day 15, cycle 2 day 1, cycle 3 day 1, day 1 of each odd cycle, end of treatment (progression)

Part II: Neratinib Only (ER-)

-Patients will receive neratinib PO daily on Days 1-28. Each cycle is 4 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.

Group Type EXPERIMENTAL

Neratinib

Intervention Type DRUG

Tumor biopsy

Intervention Type PROCEDURE

-Optional at baseline and disease progression

Research blood sample

Intervention Type PROCEDURE

-Baseline, cycle 1 day 15, cycle 2 day 1, cycle 3 day 1, day 1 of each odd cycle, end of treatment (progression)

Part II: Neratinib + Fulvestrant (ER+, fulvestrant-naive)

-Patients will receive neratinib PO daily on Days 1-28 and fulvestrant on Day 1 of each cycle (and C1D15). Each cycle is 4 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.

Group Type EXPERIMENTAL

Neratinib

Intervention Type DRUG

Fulvestrant

Intervention Type DRUG

Tumor biopsy

Intervention Type PROCEDURE

-Optional at baseline and disease progression

Research blood sample

Intervention Type PROCEDURE

-Baseline, cycle 1 day 15, cycle 2 day 1, cycle 3 day 1, day 1 of each odd cycle, end of treatment (progression)

Part II: Neratinib + Fulvestrant (ER+. prior fulvestrant-tx)

-Patients will receive neratinib PO daily on Days 1-28 and fulvestrant on Day 1 of each cycle (and C1D15). Each cycle is 4 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.

Group Type EXPERIMENTAL

Neratinib

Intervention Type DRUG

Fulvestrant

Intervention Type DRUG

Tumor biopsy

Intervention Type PROCEDURE

-Optional at baseline and disease progression

Research blood sample

Intervention Type PROCEDURE

-Baseline, cycle 1 day 15, cycle 2 day 1, cycle 3 day 1, day 1 of each odd cycle, end of treatment (progression)

Crossover: Neratinib + Trastuzumab

-If a participant experiences disease progression during Part I or Part II following neratinib, trastuzumab (or FDA approved biosimilar) may be added to the treatment regimen. Trastuzumab may be administered intravenously with a loading dose of 6 mg/kg then 4 mg/kg every 2 weeks. A cycle will be defined as 28 days. Patients will continue to receive neratinib PO daily on Days 1-28. Each cycle is 4 weeks.

Group Type EXPERIMENTAL

Neratinib

Intervention Type DRUG

Trastuzumab

Intervention Type DRUG

Tumor biopsy

Intervention Type PROCEDURE

-Optional at baseline and disease progression

Research blood sample

Intervention Type PROCEDURE

-Baseline, cycle 1 day 15, cycle 2 day 1, cycle 3 day 1, day 1 of each odd cycle, end of treatment (progression)

Crossover: Neratinib + Fulvestrant + Trastuzumab

-If a participant experiences disease progression during Part I or Part II following neratinib, trastuzumab (or FDA approved biosimilar) may be added to the treatment regimen. Trastuzumab may be administered intravenously with a loading dose of 6 mg/kg then 4 mg/kg every 2 weeks. A cycle will be defined as 28 days. Patients will receive neratinib PO daily on Days 1-28 and fulvestrant on Day 1 of each cycle (and C1D15). Each cycle is 4 weeks.

Group Type EXPERIMENTAL

Neratinib

Intervention Type DRUG

Fulvestrant

Intervention Type DRUG

Trastuzumab

Intervention Type DRUG

Tumor biopsy

Intervention Type PROCEDURE

-Optional at baseline and disease progression

Research blood sample

Intervention Type PROCEDURE

-Baseline, cycle 1 day 15, cycle 2 day 1, cycle 3 day 1, day 1 of each odd cycle, end of treatment (progression)

Interventions

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Neratinib

Intervention Type DRUG

Fulvestrant

Intervention Type DRUG

Trastuzumab

Intervention Type DRUG

Tumor biopsy

-Optional at baseline and disease progression

Intervention Type PROCEDURE

Research blood sample

-Baseline, cycle 1 day 15, cycle 2 day 1, cycle 3 day 1, day 1 of each odd cycle, end of treatment (progression)

Intervention Type PROCEDURE

Other Intervention Names

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PF-05208767 Faslodex Herceptin

Eligibility Criteria

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Inclusion Criteria

* Histologically or cytologically confirmed HER2-negative (0 or 1+ by IHC or non-amplified by FISH) breast cancer that is stage IV.
* Agree to provide archival tumor material for research
* There is no limitation on the number of prior lines of systemic therapy.
* Presence of measurable or non-measurable disease by RECIST 1.1 is acceptable, except to be eligible for the Part II fulvestrant-naive ER+ cohort, at least one measurable disease by RECIST 1.1 is required.
* At least 18 years of age.
* ECOG performance status ≤ 2
* Adequate organ function as defined below within 8 weeks of pre-registration:

* Serum creatinine ≤1.5 x ULN
* Chil-Pugh class A if with liver disease
* Able to understand and willing to sign an IRB approved written informed consent document.

Note: HER2 mutation testing may be performed while the patient is receiving active systemic therapy for metastatic breast cancer so that the result can be used to determine eligibility for study drug therapy in the future.


* Tumor tissue tested positive for HER2 mutation. HER2 mutations detected by Guardant360 are also eligible.
* Agree to provide archival tumor material for research
* ECOG performance status ≤2
* Adequate organ function as defined below within 2 weeks of registration:

* ANC ≥1.5 x 10\^9/L
* Platelet count ≥100 x 10\^9/L
* Serum creatinine ≤1.5 x ULN
* Child-Pugh class A if with liver disease
* The patient must have completed radiation therapy and be at least 1 week from the last systemic chemotherapy administration, with adequate recovery of bone marrow and organ functions, before starting neratinib.
* Presence of disease progression on the most recent disease evaluation.
* Patients with known brain metastasis are eligible, but must have received radiation and be off steroids and stable (without evidence of disease progression by imaging or exam) for 3 months.
* QTc interval ≤450 msec for men or \< or ≤ 470 msec for women within 2 weeks of registration.
* LVEF \> or = institutional ILLN within 4 weeks of registration.
* Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately. Men must agree and commit to use a barrier method of contraception while on treatment and for 3 months after the last dose of the investigational product.
* Able to understand and willing to sign an IRB approved written informed consent document.
* There is no limitation on the number of prior lines of systemic therapy.
* To be eligible for the Part II fulvestrant-naive ER+ cohort, prior treatment with fulvestrant is not allowed. In addition, ER and/or PR positivity by institutional standard is required on pathology from the most recent tumor specimen if biopsy was done unless the tissue source (for example, pleural effusion or ascites or bone biopsy) may yield false negative ER and/or PR result, in which case the pathology from an earlier time point could be used and a discussion with the study chair is required.
* To be eligible for the Part II fulvestrant-treated ER+ cohort, prior disease progression on fulvestrant is required. In addition, ER and/or PR positivity by institutional standard is required on pathology from the most recent tumor specimen unless the tissue source (for example, pleural effusion or ascites or bone biopsy) may yield false negative ER and/or PR result, in which case the pathology form an earlier time point could be used and a discussion with the study chair is required.


* Histologically or cytologically confirmed HER2-negative (0 or 1+ by IHC or non-amplified by FISH) breast cancer that is stage IV.
* Tumor tissue or circulating tumor DNA tested positive for HER2 mutation. Mutations outside the list will be assessed on a case-by-case basis by the study team to determine eligibility.
* Presence of measurable or non-measurable disease by RECIST 1.1 is acceptable, except to be eligible for the Part II fulvestrant-naïve ER+ cohort, at least one measurable disease by RECIST 1.1 is required.
* At least 18 years of age.
* ECOG performance status \< 2 (see Appendix A).
* Adequate organ function as defined below within 2 weeks of registration:

* Absolute neutrophil count: ≥1.5 × 109/L (1500/mm3)
* Platelet count: ≥100 × 109/L (100,000/mm3)
* Serum creatinine: ≤1.5 x ULN
* Child-Pugh class A if with liver disease
* The patient must have completed radiation therapy and be at least 1 week from the last systemic therapy administration, with adequate recovery of bone marrow and organ functions, before starting neratinib.
* Presence of disease progression on the most recent disease evaluation.
* Patients with known treated brain metastasis are eligible, but must have received radiation and be off steroids and stable (without evidence of disease progression by imaging or exam) for 3 months.
* QTc interval ≤ 450 msec for men or ≤ 470 msec for women within 2 weeks of registration.
* LVEF \> institutional LLN within 4 weeks of registration.
* Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately. Men must agree and commit to use a barrier method of contraception while on treatment and for 3 months after the last dose of the investigational product
* Able to understand and willing to sign an IRB approved written informed consent document.
* There is no limitation on the number of prior lines of systemic therapy.
* To be eligible for the Part II fulvestrant-naïve ER+ cohort, prior treatment with fulvestrant is not allowed. In addition, ER and/or PR positivity by institutional standard is required on pathology from the most recent tumor specimen if biopsy was done unless the tissue source (for example, pleural effusion or ascites or bone biopsy) may yield false negative ER and/or PR result, in which case the pathology from an earlier time point could be used and a discussion with the study chair is required.
* To be eligible for the Part II fulvestrant-treated ER+ cohort, prior disease progression on fulvestrant is required. In addition, ER and/or PR positivity by institutional standard is required on pathology from the most recent tumor specimen unless the tissue source (for example, pleural effusion or ascites or bone biopsy) may yield false negative ER and/or PR result, in which case the pathology from an earlier time point could be used and a discussion with the study chair is required.

Exclusion Criteria

* Testing for LVEF is not required for pre-registration, but patient must not have a recent LVEF \< LLN or have symptoms of congestive heart failure.
* Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
* Acute or currently active hepatic or biliary disease requiring antiviral therapy (with the exception of Gilbert's syndrome, asymptomatic gallstones, liver metastases, or stable chronic liver disease per investigator assessment).
* History of significant cardiac disease, cardiac risk factors, or uncontrolled arrhythmias.
* Symptomatic intrinsic lung disease or extensive tumor involvement of the lungs resulting in dyspnea at rest.


* Currently receiving any other investigational agents or systemic cancer therapy.
* Currently taking medications and herbal or dietary supplements that are strong cytochrome P450 (CYP) 3A4 inducers or inhibitors. The washout period must have been completed prior to the start of neratinib if the patient was taking any of these agents. If unavoidable, patients taking CYP3A4 inhibitors should be monitored closely.
* Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
* Acute or currently active/requiring antiviral therapy hepatic or biliary disease (with the exception of patients with Gilbert's syndrome, asymptomatic gallstones, liver metastases, or stable chronic liver disease per investigator assessment).
* Pregnant and/or breastfeeding.
* History of significant cardiac disease, cardiac risk factors, or uncontrolled arrhythmias.
* Symptomatic intrinsic lung disease or extensive tumor involvement of the lungs resulting in dyspnea at rest.
* Experiencing grade 2 or greater diarrhea.
* Prior treatment with neratinib
* Child-Pugh class B or C liver dysfunction
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Puma Biotechnology, Inc.

INDUSTRY

Sponsor Role collaborator

United States Department of Defense

FED

Sponsor Role collaborator

Washington University School of Medicine

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Cynthia Ma, M.D., Ph.D

Role: PRINCIPAL_INVESTIGATOR

Washington University School of Medicine

Locations

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University of Alabama Cancer Center

Birmingham, Alabama, United States

Site Status

Mayo Clinic

Phoenix, Arizona, United States

Site Status

University of Southern California Keck School of Medicine

Los Angeles, California, United States

Site Status

Stanford Medicine Cancer Institute

Stanford, California, United States

Site Status

University of Miami Hospital and Clinics

Miami, Florida, United States

Site Status

Northwestern University - Feinberg School of Medicine

Chicago, Illinois, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

Dana-Farber Cancer Institute, Harvard University

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

St. Luke's Cancer Institute

Kansas City, Missouri, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

University of North Carolina at Chapel Hill (Lineberger Comprehensive Cancer Center)

Chapel Hill, North Carolina, United States

Site Status

Duke Cancer Institute at Duke University Medical Center

Durham, North Carolina, United States

Site Status

Avera Cancer Institute

Sioux Falls, South Dakota, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

BC Cancer Agency

Vancouver, British Columbia, Canada

Site Status

Princess Margaret Cancer Centre

Toronto, Ontario, Canada

Site Status

Countries

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United States Canada

References

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Jhaveri K, Eli LD, Wildiers H, Hurvitz SA, Guerrero-Zotano A, Unni N, Brufsky A, Park H, Waisman J, Yang ES, Spanggaard I, Reid S, Burkard ME, Vinayak S, Prat A, Arnedos M, Bidard FC, Loi S, Crown J, Bhave M, Piha-Paul SA, Suga JM, Chia S, Saura C, Garcia-Saenz JA, Gambardella V, de Miguel MJ, Gal-Yam EN, Rapael A, Stemmer SM, Ma C, Hanker AB, Ye D, Goldman JW, Bose R, Peterson L, Bell JSK, Frazier A, DiPrimeo D, Wong A, Arteaga CL, Solit DB. Neratinib + fulvestrant + trastuzumab for HR-positive, HER2-negative, HER2-mutant metastatic breast cancer: outcomes and biomarker analysis from the SUMMIT trial. Ann Oncol. 2023 Oct;34(10):885-898. doi: 10.1016/j.annonc.2023.08.003. Epub 2023 Aug 18.

Reference Type DERIVED
PMID: 37597578 (View on PubMed)

Shishido SN, Masson R, Xu L, Welter L, Prabakar RK, D' Souza A, Spicer D, Kang I, Jayachandran P, Hicks J, Lu J, Kuhn P. Disease characterization in liquid biopsy from HER2-mutated, non-amplified metastatic breast cancer patients treated with neratinib. NPJ Breast Cancer. 2022 Feb 18;8(1):22. doi: 10.1038/s41523-022-00390-5.

Reference Type DERIVED
PMID: 35181666 (View on PubMed)

Exman P, Garrido-Castro AC, Hughes ME, Freedman RA, Li T, Trippa L, Bychkovsky BL, Barroso-Sousa R, Di Lascio S, Mackichan C, Lloyd MR, Krevalin M, Cerami E, Merrill MS, Santiago R, Crowley L, Kuhnly N, Files J, Lindeman NI, MacConaill LE, Kumari P, Tolaney SM, Krop IE, Bose R, Johnson BE, Ma CX, Dillon DA, Winer EP, Wagle N, Lin NU. Identifying ERBB2 Activating Mutations in HER2-Negative Breast Cancer: Clinical Impact of Institute-Wide Genomic Testing and Enrollment in Matched Therapy Trials. JCO Precis Oncol. 2019 Nov 15;3:PO.19.00087. doi: 10.1200/PO.19.00087. eCollection 2019.

Reference Type DERIVED
PMID: 32923853 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Related Links

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http://www.siteman.wustl.edu

Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

Other Identifiers

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201209135

Identifier Type: -

Identifier Source: org_study_id

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