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Breast CancerPhase 2NCT02260531

Cabozantinib +/- Trastuzumab In Breast Cancer Patients w/ Brain Metastases

A Phase 2 study testing Cabozantinib and Trastuzumab in people with Breast Cancer and Brain Tumor - Metastatic, run by Dana-Farber Cancer Institute at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This research study is evaluating the effectiveness of the drug called cabozantinib (alone or in combination with trastuzumab) as a possible treatment for advanced breast cancer in which the cancer has spread to the brain. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Breast Cancer, Brain Tumor - Metastatic
What is being tested?
Cabozantinib and Trastuzumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
36 participants
Where
2 sitesUnited States
Study dates
Nov 2014to 17 Mar 2020
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients must have histologically or cytologically confirmed invasive breast cancer, with stage IV disease.
  • New or progressive CNS lesions, as assessed by the patient's treating physician.
  • For patients who have received prior cranial radiation, no increase in corticosteroid dose in the week prior to the baseline brain MRI
  • Discontinued prior therapy (with the exception of trastuzumab for patients with HER2+ breast cancer)
  • Recovery to baseline or ≤ Grade 1 CTCAE v.4.0 from toxicities related to any prior treatments, unless AE(s) are clinically nonsignificant and/or stable on supportive therapy;
  • The subject has an ECOG performance status of 0 or 1
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  • Patients must have normal organ and marrow function and laboratory values as follows within 14 days before the first dose of cabozantinib
  • Sexually active subjects (men and women) must agree to use medically accepted barrier methods of contraception (e.g., male or female condom) during the course of the study and for 4 months after the last dose of study drug(s)
  • Subjects of childbearing potential must not be pregnant at screening.
  • Patients on bisphosphonates may continue receiving bisphosphonate therapy during study. Patients wanting to initiate bisphosphonate therapy may do so.
  • The subject has had an assessment of all known disease sites eg, by computerized tomography (CT) scan, magnetic resonance imaging (MRI), bone scan as appropriate, within 28 days before the first dose of cabozantinib

Who cannot join

  • The subject has received cabozantinib or another c-Met inhibitor (please note ARQ 197 is not considered a MET inhibitor for purposes of this study given data to suggest it inhibits tubulin)
  • The subject has uncontrolled, significant intercurrent or recent illness
  • Leptomeningeal disease as the only site of CNS involvement
  • Known contraindication to MRI with gadolinium contrast, such as cardiac pacemaker, shrapnel, or ocular foreign body
  • More than 2 seizures over the last 4 weeks prior to study entry
  • Grade 1 or higher CNS hemorrhage on baseline brain MRI, or history of grade 2 or higher CNS hemorrhage within 12 months
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  • Has experienced clinically-significant GI bleeding within 6 months before first dose of cabozantinib; hemoptysis of ≥ 0.5 teaspoon (2.5ml) of red blood within 3 months before the first dose of cabozantinib; any other signs indicative of pulmonary hemorrhage within 3 months before the first dose of cabozantinib
  • The subject has tumor in contact with, invading or encasing any major blood vessels
  • The subject has evidence of tumor invading the GI tract (esophagus, stomach, small or large bowel, rectum or anus), or any evidence of endotracheal or endobronchial tumor within 28 days before the first dose of cabozantinib
  • The subject requires concomitant treatment, in therapeutic doses, with anticoagulants. Low dose aspirin (≤ 81 mg/day), low-dose warfarin ( ≤1 mg/day), and prophylactic LMWH are permitted.
  • The subject has prothrombin time (PT)/INR or partial thromboplastin time (PTT) test ≥1.3 × the laboratory ULN within 7 days before the first dose of cabozantinib.
  • Inability to swallow intact tablets
  • Pregnant or lactating females
  • Diagnosis of another malignancy within 2 years before the first dose of cabozantinib, except for superficial skin cancers, or localized, low grade tumors deemed cured and not treated with systemic therapy
  • Subjects with clinically relevant ongoing complications from prior radiation therapy are not eligible
  • The subject is known to be positive for the human immunodeficiency virus (HIV)
  • Subjects with clinically relevant ongoing complications from prior surgery are not eligible
  • QTcF > 500 msec on average of screening EKGs performed within 28 days of first dose of cabozantinib. Three EKGs must be performed at screening. If the average of these three consecutive results for QTcF is > 500 msec, the subject is ineligible.
  • Active infection requiring IV antibiotics at Day 1 of cycle 1
  • No prior lapatinib within 7 days prior to initiation of protocol treatment
  • Receive concurrent investigational agents while on study
  • Receive any concurrent chemotherapy, radiotherapy, or hormonal therapy while on study
  • Previously identified allergy or hypersensitivity to components of the cabozantinib formulations
  • The subject requires chronic concomitant treatment with strong CYP3A4 inducers

Trial locations and contacts

2 sites in 1 country.

United States2 sites
  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

Other trials in United States

About this study

Official title: A Phase II Study of Cabozantinib Alone or in Combination With Trastuzumab in Breast Cancer Patients With Brain Metastases

This research study is evaluating the effectiveness of the drug called cabozantinib (alone or in combination with trastuzumab) as a possible treatment for advanced breast cancer in which the cancer has spread to the brain.

Read the detailed description

This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational intervention to learn whether the intervention works in treating a specific disease. "Investigational" means that the intervention is being studied.

The FDA (the U.S. Food and Drug Administration) has not approved cabozantinib for your specific disease but it has been approved for other uses.

Few treatments exist for brain metastases from breast cancer. Radiation and surgery are generally included as a possible standard of care treatments for this diagnosis.

In this research study, the investigator are looking at how well cabozantinib works in treating breast cancer that has spread to the brain. Cabozantinib has been used in some phase I studies and information from those other research studies suggests that cabozantinib may help to shrink or stabilize the participant's breast cancer. In addition, information from these studies has shown that cabozantinib may pass through the blood brain barrier (a protective layer that prevents most large molecules and cells found in the blood from entering the brain tissue) and may be an effective treatment for brain metastases.

If the participant has HER2-positive breast cancer, they will receive trastuzumab in addition to cabozantinib. Trastuzumab is an FDA approved drug for the treatment of HER2-positive metastatic breast cancer. However, the combination of cabozantinib and trastuzumab has not yet been tested. Trastuzumab may help to shrink or stabilize breast cancer in combination with cabozantinib. If the participant's breast cancer is HER2-negative, they will not receive trastuzumab as part of this clinical trial.

The names of the study interventions involved in this study are:

  • Cabozantinib (XL184)
  • Trastuzumab (herceptin) (participants with HER2-positive disease only)
Keywords
Metastatic breast cancer and brain metastases, HER-2 Positive Breast Cancer, ER-Positive PR-Positive HER-2 Negative Breast Cancer, ER-Negative PR-Negative HER2-Negative Breast Cancer
Medical subject headings
Breast Neoplasms, Brain Neoplasms, Triple Negative Breast Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT02260531
Study groupRoleWhat they receive
Cohort 1 - Cabozantinib, Trastuzumab for HER2+ExperimentalHER2-positive * Cabozantinib- orally administered daily per treatment cycle, 60 mg per day * Trastuzumab- IV administered once per cycle, 8 mg/kg IV loading dose followed by 6 mg/kg IV Cycle duration equals 3 weeks. Patients are treated indefinitely based on unacceptable toxicity, disease progression, or withdrawal for other reasons.
Receives: Drug: Cabozantinib; Drug: Trastuzumab
Cohort 2 - Cabozantinib for ER+ and/or PR+ExperimentalHormone receptor-positive (ER+ and/or PR+) - Cabozantinib- orally administered daily per treatment cycle, 60 mg per day Cycle duration equals 3 weeks. Patients are treated indefinitely based on unacceptable toxicity, disease progression, or withdrawal for other reasons.
Receives: Drug: Cabozantinib
Cohort 3 - Cabozantinib for ER-, PR-, HER2-ExperimentalTriple negative (ER-, PR-, HER2-) - Cabozantinib- orally administered daily per treatment cycle, 60 mg per day Cycle duration equals 3 weeks. Patients are treated indefinitely based on unacceptable toxicity, disease progression, or withdrawal for other reasons.
Receives: Drug: Cabozantinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. CNS Objective Response Rate (ORR)

    The central nervous system (CNS) ORR was defined as the percentage of participants achieving complete response (CR) or partial response (PR) based on RECIST 1.1 criteria in the evaluation CNS lesions on treatment: CR is complete disappearance of all target lesions and PR is at least a 30% decrease in the sum of longest diameter (LD) of target lesions, taking as reference baseline sum LD. PR or better overall response assumes at a minimum incomplete response/stable disease (SD) for the evaluation of non-target lesions and absence of new lesions.

    Measured: Disease assessments occurred every 6 cycles. Patients with stable or responsive disease after completion of 6 cycles could reduce frequency of assessments to every 3 cycles. Response was evaluated up to 25 months.

Secondary outcomes (7)
  1. CNS Volumetric Objective Response Rate (ORR)

    CNS volumetric ORR was defined as the percentage of participants achieving complete response (CR) or partial response (PR) defined as:

    CR

    • Complete resolution of all measurable (≥ 1 cm diameter) and non-measurable brain metastases
    • No new CNS lesions (new lesion defined as ≥ 6 mm diameter) PR
    • ≥ 50% reduction in the volumetric sum of all measurable (≥ 1 cm diameter) brain metastases compared to baseline
    • No progression of non-measurable lesions
    • No new lesions (new lesion defined as ≥ 6 mm)

    ORR also requires:

    • Stable or decreasing steroid dose
    • No new/progressive tumor-related neurologic signs or symptoms

    Measured: Disease assessments occurred every 6 cycles. Patients with stable or responsive disease after completion of 6 cycles could reduce frequency of assessments to every 3 cycles. Response was evaluated up to 25 months .

  2. Non-CNS Objective Response Rate (ORR)

    The non-central nervous system (CNS) ORR was defined as the percentage of participants achieving complete response (CR) or partial response (PR) based on RECIST 1.1 criteria in the evaluation non-CNS lesions on treatment: CR is complete disappearance of all target lesions and PR is at least a 30% decrease in the sum of longest diameter (LD) of target lesions, taking as reference baseline sum LD. PR or better overall response assumes at a minimum incomplete response/stable disease (SD) for the evaluation of non-target lesions and absence of new lesions.

    Measured: Disease assessments occurred every 6 cycles. Patients with stable or responsive disease after completion of 6 cycles could reduce frequency of assessments to every 3 cycles. Response was evaluated up to 25 months.

  3. Median Progression-Free Survival (PFS)

    Progression-free survival based on the Kaplan-Meier method is defined as the duration of time from study entry to documented disease progression (PD) or death. Per RECIST 1.1 criteria: progressive disease (PD) is at least a 20% increase in the sum of longest diameter (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. PD for the evaluation of non-target lesions is the appearance of one or more new lesions and/or unequivocal progression of non-target lesions.

    Measured: Disease assessments occurred every 6 cycles. Patients with stable or responsive disease after completion could reduce frequency to every 3 cycles. In long-term follow-up, assessments occurred every 6 months until death. Follow-up time is up to 25 months.

  4. 12-Week Clinical Benefit Rate

    Clinical benefit rate (CBR) was defined as the percentage of patients achieving complete response (CR), partial response (PR), or stable disease (SD) based on RECIST 1.1 criteria by 12 weeks. Per RECIST 1.1 for target lesions: CR is complete disappearance of all target lesions and PR is at least a 30% decrease in the sum of longest diameter (LD) of target lesions, taking as reference baseline sum LD. PD is at least a 20% increase in sum LD of target lesions (smallest sum LD reference), new lesions, and/or unequivocal progression of existing non-target lesions. Stable disease (SD) is defined as any condition not meeting the above criteria.

    Measured: Evaluate at 12 weeks.

  5. First Progression Site

    Site that a patient has his/her first tumor progression (CNS vs Non-CNS)

    Measured: Disease assessments occurred every 6 cycles. Patients with stable or responsive disease after completion could reduce frequency to every 3 cycles. In long-term follow-up, assessments occurred every 6 months until death. Follow-up time is up to 25 months.

  6. Overall Survival (OS)

    OS based on the Kaplan-Meier method is defined as the time from study entry to death or censored at date last known alive.

    Measured: Off-treatment patients followed every 6 months until death. Follow-up time is up to 25 months.

  7. Incidence of Grade 4 Treatment-Related Toxicity

    All grade 4 adverse events (AE) with treatment attribution of possibly, probably or definite based on CTCAEv4 as reported on case report forms were counted. Incidence is the number of patients experiencing at least one treatment-related grade 4 AE of any type during the time of observation.

    Measured: Participants should be re-evaluated for response every 6 weeks. Patients with stable or responsive disease after completion of 6 cycles may have the frequency of scans reduced to once every 3 cycles, and in long-term follow-up every 6 months until death.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Oct 2014 (estimated)
  2. Study startNov 2014
  3. Primary completion4 Jan 2019
  4. Study completion17 Mar 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
36 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02260531
Sponsor's protocol ID
14-359
Status last verified
May 2021
First submitted
17 Sep 2014
Last updated
20 May 2021
Data monitoring committee
Yes

Publications

  • Leone JP, Duda DG, Hu J, Barry WT, Trippa L, Gerstner ER, Jain RK, Tan S, Lawler E, Winer EP, Lin NU, Tolaney SM. A phase II study of cabozantinib alone or in combination with trastuzumab in breast cancer patients with brain metastases. Breast Cancer Res Treat. 2020 Jan;179(1):113-123. doi: 10.1007/s10549-019-05445-z. Epub 2019 Sep 20.

    PubMed 31541381

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Cabozantinib and Trastuzumab in people with Breast Cancer and Brain Tumor - Metastatic, run by Dana-Farber Cancer Institute at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

CNS Objective Response Rate (ORR) (time frame: Disease assessments occurred every 6 cycles. Patients with stable or responsive disease after completion of 6 cycles could reduce frequency of assessments to every 3 cycles. Response was evaluated up to 25 months.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02260531, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2021.

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