Breast NeoplasmsPhase 2NCT01779050
Effect of Trastuzumab on Disease Free Survival in Early Stage HER2-Negative Breast Cancer Patients With ERBB2 Expressing Disseminated Tumor Cells
A Phase 2 study testing Doxorubicin, Trastuzumab and Cyclophosphamide in people with Breast Neoplasms, run by Washington University School of Medicine at 1 site in United States.
Trial summary
This phase II trial studies the efficacy of trastuzumab treatment in breast cancer patients with stage II-III human epidermal growth factor receptor 2 (HER2)-negative tumors and HER2-expressing bone marrow disseminated tumor cells (DTCs). Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Breast Neoplasms
- What is being tested?
- Doxorubicin, Trastuzumab and Cyclophosphamide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Dec 2013to 1 Sep 2021
- 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
- Women only
- No healthy volunteers
- Adult, Older adult
Pre-Registration Inclusion Criteria:
- Histologically confirmed HER2-negative primary invasive ductal or invasive lobular breast carcinoma. For patients enrolling for neoadjuvant treatment, diagnosis must be clinical stage II or III; for patients enrolling for adjuvant treatment, diagnosis must be pathologic stage IIA to IIIC. Standard HER2 testing will be performed in the surgical specimen at Washington University according to the standard of care in the Department of Pathology. A HER2-negative primary breast cancer sample from a patient eligible for randomization should have a HER2 IHC score of 0 or 1+ Those patients with IHC score of 2+ should be HER2 FISH-negative in standard testing. Patient will have undergone staging studies including a CT of the chest/abdomen/pelvis and bone scan and/or PET scan either prior to the initiation of treatment or prior to entry into the trial. In addition, patients with non-metastatic, HER2-negative, recurrent tumors who need chemotherapy are eligible.
- Planning to receive best practice adjuvant or neoadjuvant chemotherapy according to institutional guidelines. Adjuvant tamoxifen or aromatase inhibitors treatment will be allowed for hormone receptor-positive patients. Patients who have failed neoadjuvant endocrine therapy will also be eligible.
- At least 18 years old.
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1.
- Patient (or legally authorized representative) must be able to understand and willing to sign a written informed consent document.
Pre-Registration Exclusion Criteria:
- Prior chemotherapy for this cancer (excluding initiation of best practice chemotherapy to be given as standard of care as described in this protocol, which may be initiated after the pre-registration bone marrow collection but before final confirmation of eligibility and randomization).
- Previous treatment with trastuzumab or any other Her2 targeted therapy.
- Presence of an 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.
Registration Inclusion Criteria
- Presence of bone marrow ERBB2 overexpressing DTCs at the time of diagnosis; bone marrow aspiration will be performed in consented patients to evaluate DTCs following pre-registration provided patients meet all eligibility criteria as described in this section.
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1.
- Adequate cardiac function as demonstrated by LVEF of >55% performed no more than 4 weeks prior to randomization.
- Normal organ and marrow function as defined below:
- leukocytes ≥3,000/mcL
- absolute neutrophil count ≥1,500/mcL
- platelets ≥100,000/mcL
- hemoglobin ≥ 10 g/dL
- total bilirubin within institutional upper limits of normal unless related to primary disease
- AST(SGOT)/ALT(SGPT) ≤2.0 X institutional upper limit of normal
- Creatinine ≤ 1.5 institutional upper limits of normal OR creatinine clearance ≥60 mL/min/1.73 m2 for patients with creatinine levels above institutional normal
- If a woman of childbearing potential, patient must use two forms of effective contraception for a minimum of 6 months following trastuzumab. Effective methods of birth control include use of established oral, injected, or implanted hormonal methods of birth control, IUD, IUS, and condoms.
Registration Exclusion Criteria
- Evidence of distant metastasis present by CT scan, bone scan, or physical exam.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to trastuzumab.
- Prior chemotherapy for this cancer (excluding initiation of best practice chemotherapy to be given as standard of care described in this protocol, which may be initiated after the pre-registration bone marrow collection but before final confirmation of eligibility and randomization).
- History of other malignancy ≤ 5 years previous with the exception of basal cell or squamous cell carcinoma of the skin which were treated with local resection only or carcinoma in situ of the cervix.
- Pregnant or breastfeeding. Patient must have a negative serum pregnancy test ≤ 7 days from date of registration (if a woman of childbearing potential).
- Clinically important history of active liver disease, including viral or other hepatitis or cirrhosis.
- Uncontrolled hypocalcemia, hypomagnesemia, hyponatremia, or hypokalemia defined as less than the lower limit of normal for the institution despite adequate electrolyte supplementation.
- Symptomatic intrinsic lung disease or extensive tumor involvement of the lungs resulting in dyspnea at rest.
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: A Phase II Randomized Trial Evaluating the Effect of Trastuzumab on Disease Free Survival in Early Stage HER2-Negative Breast Cancer Patients With ERBB2 Expressing Bone Marrow Disseminated Tumor Cells
This phase II trial studies the efficacy of trastuzumab treatment in breast cancer patients with stage II-III human epidermal growth factor receptor 2 (HER2)-negative tumors and HER2-expressing bone marrow disseminated tumor cells (DTCs). Administering targeted trastuzumab therapy to these patients may result in the elimination of HER2 expressing disseminated tumor cells and improved disease free survival.
- Conditions
- Breast Neoplasms
- Being tested
- Doxorubicin, Trastuzumab, Cyclophosphamide, Paclitaxel, Epirubicin
- Medical subject headings
- Breast Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DoxorubicinDrug
Also called Adriamycin®, Rubex®, Hydroxydaunomycin Hydrochloride, Hydroxydoxorubicin Hydrochloride
TrastuzumabBiological
Also called •Herceptin®
CyclophosphamideDrug
Also called Cytoxan®, CPM, CTX, CYT
PaclitaxelDrug
Also called Abraxane®, Onxol®
EpirubicinDrug
Also called Ellence, Pharmorubicin, Epirubicin ebewe
DocetaxelDrug
Also called Taxotere, Docefrez
CarboplatinDrug
Also called cis-Diammine(1,1-cyclobutanedicarboxylato)platinum(II), Paraplatin, Paraplatin-AQ
FluorouracilDrug
Also called 5-FU, Adrucil
| Study group | Role | What they receive |
|---|---|---|
| Arm I (definitive therapy) | Active comparator | Patients receive definitive surgery and best practice standard chemotherapy according to NCCN guidelines. The 5 chemo backbone options are:
* doxorubicin or epirubicin plus cyclophosphamide followed by paclitaxel or docetaxel
* docetaxel plus cyclophosphamide
* single-agent paclitaxel
* docetaxel plus carboplatin
* fluorouracil plus epirubicin plus cyclophosphamide followed by paclitaxel or docetaxel Receives: Drug: Doxorubicin; Drug: Cyclophosphamide; Drug: Paclitaxel; Drug: Epirubicin; Drug: Docetaxel; Drug: Fluorouracil |
| Arm II (definitive therapy, trastuzumab) | Experimental | Patients receive definitive surgery and best practice standard chemotherapy according to NCCN guidelines. The 5 chemo backbone options are:
* doxorubicin or epirubicin plus cyclophosphamide followed by paclitaxel or docetaxel
* docetaxel plus cyclophosphamide
* single-agent paclitaxel
* docetaxel plus carboplatin
* fluorouracil plus epirubicin plus cyclophosphamide followed by paclitaxel or docetaxel
Patients will also receive trastuzumab IV over 30-90 minutes for 52 weeks starting such that there is a minimum of 8 weeks of overlap with the standard of care chemotherapy. Trastuzumab may be given weekly, every 2 weeks, or every 3 weeks while overlapping with standard of care chemotherapy. Trastuzumab will be given every 3 weeks after all standard of care chemotherapy has concluded. Treatment continues in the absence of disease progression or unacceptable toxicity. Receives: Drug: Doxorubicin; Biological: Trastuzumab; Drug: Cyclophosphamide; Drug: Paclitaxel; Drug: Epirubicin; Drug: Docetaxel; Drug: Carboplatin; Drug: Fluorouracil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Disease Recurrence
-Disease recurrence is defined as the documented appearance of local (breast, chest wall, axillary, supraclavicular nodes) or distant disease.
Measured: Up to 3 years after completion of treatment (estimated to be 4 years)
Death Rate
Measured: Up to 3 years after completion of treatment (estimated to be 4 years)
Secondary outcomes (1)
Elimination of ERBB2 Overexpressing Bone Marrow DTCs
Bone marrow disseminated tumor cells (DTCs) are evaluated by RT-PCR performed on specimens collected 6-18 months apart (one before and one after therapy). The proportion of samples turned negative after therapy will be calculated. Samples will be considered negative for ERBB2-expression if expression from bone marrow collected from each iliac crest is less than 2 standard deviations above the ERBB2-level in pooled normal bone marrow specimens.
Measured: Before treatment and after treatment (estimated to be 6-18 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2013 (estimated)
- Study start19 Dec 2013
- Primary completion1 Sep 2021
- Study completion1 Sep 2021
Why it stopped: Loss of study funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Breast NeoplasmsStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Rebecca Aft, M.D., Ph.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01779050
- Sponsor's protocol ID
- 201309084
- Status last verified
- Oct 2022
- First submitted
- 25 Jan 2013
- Last updated
- 1 Nov 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Siddappa CM, Watson MA, Pillai SG, Trinkaus K, Fleming T, Aft R. Detection of disseminated tumor cells in the bone marrow of breast cancer patients using multiplex gene expression measurements identifies new therapeutic targets in patients at high risk for the development of metastatic disease. Breast Cancer Res Treat. 2013 Jan;137(1):45-56. doi: 10.1007/s10549-012-2279-y. Epub 2012 Nov 6.
PubMed 23129172Rack B, Juckstock J, Gunthner-Biller M, Andergassen U, Neugebauer J, Hepp P, Schoberth A, Mayr D, Zwingers T, Schindlbeck C, Friese K, Janni W. Trastuzumab clears HER2/neu-positive isolated tumor cells from bone marrow in primary breast cancer patients. Arch Gynecol Obstet. 2012 Feb;285(2):485-92. doi: 10.1007/s00404-011-1954-2. Epub 2011 Jun 30.
PubMed 21717141Vincent-Salomon A, Pierga JY, Couturier J, d'Enghien CD, Nos C, Sigal-Zafrani B, Lae M, Freneaux P, Dieras V, Thiery JP, Sastre-Garau X. HER2 status of bone marrow micrometastasis and their corresponding primary tumours in a pilot study of 27 cases: a possible tool for anti-HER2 therapy management? Br J Cancer. 2007 Feb 26;96(4):654-9. doi: 10.1038/sj.bjc.6603584. Epub 2007 Jan 30.
PubMed 17262082
Study at a glance
From the registry record, last updated by the sponsor on 1 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Randomized Trial Evaluating the Effect of Trastuzumab on Disease Free Survival in Early Stage HER2-Negative Breast Cancer Patients With ERBB2 Expressing Bone Marrow Disseminated Tumor Cells
- ClinicalTrials.gov ID
- NCT01779050
- Acronym / sponsor ID
- 201309084
- Conditions
- Breast Neoplasms
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Loss of study funding
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- 19 Dec 2013 (actual)
- Primary completion
- 1 Sep 2021 (actual)
- Study completion
- 1 Sep 2021 (actual)
- First posted
- 29 Jan 2013 (estimated)
- Results
- Posted 1 Nov 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies the efficacy of trastuzumab treatment in breast cancer patients with stage II-III human epidermal growth factor receptor 2 (HER2)-negative tumors and HER2-expressing bone marrow disseminated tumor cells (DTCs). Administering targeted trastuzumab therapy to these patients may result in the elimination of HER2 expressing disseminated tumor cells and improved disease free survival.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (definitive therapy) | Active comparator | Drug: Doxorubicin, Drug: Cyclophosphamide, Drug: Paclitaxel, Drug: Epirubicin, Drug: Docetaxel, Drug: FluorouracilPatients receive definitive surgery and best practice standard chemotherapy according to NCCN guidelines. The 5 chemo backbone options are: * doxorubicin or epirubicin plus cyclophosphamide followed by paclitaxel or docetaxel * docetaxel plus cyclophosphamide * single-agent paclitaxel * docetaxel plus carboplatin * fluorouracil plus epirubicin plus cyclophosphamide followed by paclitaxel or docetaxel |
| Arm II (definitive therapy, trastuzumab) | Experimental | Drug: Doxorubicin, Biological: Trastuzumab, Drug: Cyclophosphamide, Drug: Paclitaxel, Drug: Epirubicin, Drug: Docetaxel, Drug: Carboplatin, Drug: FluorouracilPatients receive definitive surgery and best practice standard chemotherapy according to NCCN guidelines. The 5 chemo backbone options are: * doxorubicin or epirubicin plus cyclophosphamide followed by paclitaxel or docetaxel * docetaxel plus cyclophosphamide * single-agent paclitaxel * docetaxel plus carboplatin * fluorouracil plus epirubicin plus cyclophosphamide followed by paclitaxel or docetaxel Patients will also receive trastuzumab IV over 30-90 minutes for 52 weeks starting such that there is a minimum of 8 weeks of overlap with the standard of care chemotherapy. Trastuzumab may be given weekly, every 2 weeks, or every 3 weeks while overlapping with standard of care chemotherapy. Trastuzumab will be given every 3 weeks after all standard of care chemotherapy has concluded. Treatment continues in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DoxorubicinAlso: Adriamycin®, Rubex®, Hydroxydaunomycin Hydrochloride, Hydroxydoxorubicin Hydrochloride | Drug | Arm I (definitive therapy), Arm II (definitive therapy, trastuzumab) |
| TrastuzumabAlso: •Herceptin® | Biological | Arm II (definitive therapy, trastuzumab) |
| CyclophosphamideAlso: Cytoxan®, CPM, CTX, CYT | Drug | Arm I (definitive therapy), Arm II (definitive therapy, trastuzumab) |
| PaclitaxelAlso: Abraxane®, Onxol® | Drug | Arm I (definitive therapy), Arm II (definitive therapy, trastuzumab) |
| EpirubicinAlso: Ellence, Pharmorubicin, Epirubicin ebewe | Drug | Arm I (definitive therapy), Arm II (definitive therapy, trastuzumab) |
| DocetaxelAlso: Taxotere, Docefrez | Drug | Arm I (definitive therapy), Arm II (definitive therapy, trastuzumab) |
| CarboplatinAlso: cis-Diammine(1,1-cyclobutanedicarboxylato)platinum(II), Paraplatin, Paraplatin-AQ | Drug | Arm II (definitive therapy, trastuzumab) |
| FluorouracilAlso: 5-FU, Adrucil | Drug | Arm I (definitive therapy), Arm II (definitive therapy, trastuzumab) |
Primary outcome
Number of Participants With Disease Recurrence
Time frame: Up to 3 years after completion of treatment (estimated to be 4 years)
-Disease recurrence is defined as the documented appearance of local (breast, chest wall, axillary, supraclavicular nodes) or distant disease.
Death Rate
Time frame: Up to 3 years after completion of treatment (estimated to be 4 years)
1 secondary outcome
Elimination of ERBB2 Overexpressing Bone Marrow DTCs
Time frame: Before treatment and after treatment (estimated to be 6-18 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 23129172Siddappa CM, Watson MA, Pillai SG, Trinkaus K, Fleming T, Aft R. Detection of disseminated tumor cells in the bone marrow of breast cancer patients using multiplex gene expression measurements identifies new therapeutic targets in patients at high risk for the development of metastatic disease. Breast Cancer Res Treat. 2013 Jan;137(1):45-56. doi: 10.1007/s10549-012-2279-y. Epub 2012 Nov 6.
- Background: PubMed 21717141Rack B, Juckstock J, Gunthner-Biller M, Andergassen U, Neugebauer J, Hepp P, Schoberth A, Mayr D, Zwingers T, Schindlbeck C, Friese K, Janni W. Trastuzumab clears HER2/neu-positive isolated tumor cells from bone marrow in primary breast cancer patients. Arch Gynecol Obstet. 2012 Feb;285(2):485-92. doi: 10.1007/s00404-011-1954-2. Epub 2011 Jun 30.
- Background: PubMed 17262082Vincent-Salomon A, Pierga JY, Couturier J, d'Enghien CD, Nos C, Sigal-Zafrani B, Lae M, Freneaux P, Dieras V, Thiery JP, Sastre-Garau X. HER2 status of bone marrow micrometastasis and their corresponding primary tumours in a pilot study of 27 cases: a possible tool for anti-HER2 therapy management? Br J Cancer. 2007 Feb 26;96(4):654-9. doi: 10.1038/sj.bjc.6603584. Epub 2007 Jan 30.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rebecca Aft, M.D., Ph.D. principal investigator
Washington University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Doxorubicin, Trastuzumab and Cyclophosphamide in people with Breast Neoplasms, run by Washington University School of Medicine at 1 site 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?
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: stopped early, as of the sponsor's last update on 1 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 1 Nov 2022). 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?
Number of Participants With Disease Recurrence (time frame: Up to 3 years after completion of treatment (estimated to be 4 years)). The study lists 2 primary outcomes in all. 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 NCT01779050, which also lists 3 linked publications.
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 1 Nov 2022.