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Randomized Clinical Trial to Evaluate the Predictive Accuracy of a Gene Expression Profile-Based Test to Select Patients for Preoperative Taxane/Anthracycline Chemotherapy for Stage I-III Breast Cancer

Phase 3
Not Enrolling
Breast Cancer

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Trial Information

Randomized Clinical Trial to Evaluate the Predictive Accuracy of a Gene Expression Profile-Based Test to Select Patients for Preoperative Taxane/Anthracycline Chemotherapy for Stage I-III Breast Cancer

The new test being studied measures the presence or absence of each of 12,000-15,000 human
genes in the cancer using "DNA chip" technology. All of the therapeutic treatment that you
will receive is part of the standard of care. Only the biopsy and Deoxyribonucleic acid
(DNA) testing is investigational.

Before and during chemotherapy, you will need to have routine clinical and lab tests that
are part of your standard of care. These tests may include x-rays such as computed
tomography (CT) scan of the liver or lung, bone scan and mammograms, or ultra sonograms of
the breast to find out the extent of the cancer at the time of diagnosis. Blood tests (1-2
tablespoon) will be performed before each FAC (5-fluorouracil, doxorubicin,
cyclophosphamide) or FEC (5-fluorouracil, epirubicin, cyclophosphamide) chemotherapy end at
least once every 2 weeks during weekly paclitaxel therapy to make sure that it is safe to
receive further treatment. All of these tests are part of good clinical care and are not
considered investigational.

During treatment with FAC or FEC chemotherapy, you will be seen by your physician every 3
weeks. During the weekly paclitaxel treatment you will be seen by your physician every 4-6
weeks. Additional visits may be necessary depending on your clinical course.

During this study, you will be asked to have a fine needle aspiration (FNA) of the cancer in
your breast or lymph nodes. The FNA procedure involves insertion of a small needle into the
tumor to suction out (aspirate) cells from the cancer 3-4 times in one session. A physician
who is trained in this method will perform the procedure. It could be done at the same time
when the diagnosis of your cancer is made or it may be done at later time point. The needle
aspiration will have to be done before you start any chemotherapy for your cancer. A DNA
chip test called transcriptional profiling will be performed on the FNA specimens at M. D.
Anderson in Houston (TX, USA). The test will be done to try to predict if an individual is
likely to have response to paclitaxel/FAC chemotherapy or not. This study is done to see how
accurate this test is. The chemotherapy response prediction test was developed by
investigators at Millennium Pharmaceuticals Inc. (Cambridge, MA, USA) and clinical
scientists at M. D. Anderson. This research may lead to the development of a commercial
diagnostic test.

Only patients who need chemotherapy to improve their chance of cure may participate in this
study. Whether you would benefit from chemotherapy or not will be decided by your physician.
During this study 18-24 weeks of chemotherapy will be given to you before surgery. It is
clear from several large clinical studies that chemotherapy works equally well regardless of
whether you receive it before or after breast surgery. By giving chemotherapy before
surgery, researchers can study why some cancers respond so well to treatment. You may expect
that 80-90% of the time the cancer will shrink and about 15-30% of the time the cancer will
completely disappear from the breast or lymph nodes by the time you finish chemotherapy.
Many of the patients who experience the complete disappearance of cancer will be cured. The
purpose of this research is to find out who these individuals are and develop a test that
could identify them at the time of the diagnosis. About 5-10% of patients do not experience
any shrinkage of the cancer and may experience growth of the tumor. If the tumor grows
during treatment, you will be switched over to another chemotherapy, you may receive
radiation treatment, or you may have surgery. What is the best option for you will need to
be decided by your treating physician.

You will be randomly assigned (as in the toss of a coin) to one of two treatment groups.

- Participants in the first group will receive weekly treatments with the chemotherapy
drug paclitaxel by vein. After completion of 12 paclitaxel treatments, you will receive
4 additional treatments with FAC or FEC combination chemotherapy. The FAC or FEC
treatments are given once every 3 weeks. You and your physician will decide together if
FAC or FEC is the more appropriate treatment for you. Both of these treatments are
believed to be equally effective but FAC is a longer treatment that requires 72-hour
infusion whereas FEC can be given in one day. The total length of treatment is 24 weeks
after which you will have breast and lymph node surgery.

- Participants in the second group will receive 6 treatments (6 times 3 = 18 weeks) with
FAC or FEC before surgery. Those patients who still have cancer in their breast or
lymph nodes at the time of surgery may receive additional chemotherapy after the
surgery with paclitaxel or a similar drug called docetaxel. One large clinical study
showed that addition of 4 courses of paclitaxel after 4 treatments with AC
(doxorubicin, cyclophosphamide) chemotherapy improved survival. This is the reason why
you may be recommended to receive paclitaxel or docetaxel treatment after surgery.
Whether you need additional postoperative chemotherapy or not will need to be discussed
between you and your physician.

After you completed all your treatments, you will be contacted (on the phone or by letter)
every 6 months for 10 years to find out how well you do and if the cancer has come back.

This research will generate a large database of molecular abnormalities found in breast
cancer. Researchers will study the presence or absence of 12,000-15,000 human genes in the
cancer of each patient who participates in this study. This information along with the
clinical features of the cancer will be stored in the database. The age and race of patients
as well as the long-term outcome of treatment will also be included in the database. Other
information about your health and family history of cancer may be added to the database in
the future. However, your name, address or date of birth will not be part of this research
database. The molecular data and the clinical information will be used to learn about the
causes of breast cancer and to develop the best individual therapy for future patents.

This is an investigational study. The chemotherapy that you will receive is not
experimental. Paclitaxel, FAC or FEC and their combination are commercially available drugs
and are all approved by the FDA to treat newly diagnosed breast cancer. A total of up to 273
patients will take part in this multicenter study. Up to 150 may be enrolled at M. D.

Inclusion Criteria:

1. Histologically confirmed stage I-III invasive carcinoma of the breast for whom
adjuvant chemotherapy is indicated. Patients must have intact or measurable residual
cancer (by mammogram, ultra sonogram or physical exam) in the breast. Women of
childbearing potential must have a negative pregnancy test (serum or urine beta Human
chorionic gonadotropin (HCG)) prior to initiation of chemotherapy.

2. Patients should have adequate organ function to tolerate chemotherapy.

3. Patient must be willing to undergo a one-time pretreatment research FNA biopsy

Exclusion Criteria:

1. Patients who have completed lumpectomy, segmental mastectomy or modified radical
mastectomy and, therefore no longer have any measurable cancer left in their breast
are not eligible.

2. Patients with stage IV, metastatic breast cancers are not eligible.

3. Patients for whom anthracycline or paclitaxel chemotherapies are contraindicated, for
example Patients who are pregnant or lactating are not eligible.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Pathologic Complete Response Rate in breast and axillary lymph nodes

Outcome Description:

Pathologic Complete Response Rate after completion of preoperative chemotherapy, based on routine clinical pathology report where Pathologic complete response defined as complete absence of any viable invasive cancer cells in resected breast and lymph nodes. Specimens in breast may contain in situ cancer (ductal or lobular carcinoma in situ) and still be considered complete response.

Outcome Time Frame:

After completion of preoperative chemotherapy then every 6 months for 10 years.

Safety Issue:


Principal Investigator

Lajos Pusztai, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

September 2003

Completion Date:

September 2010

Related Keywords:

  • Breast Cancer
  • Breast Cancer
  • Gene Expression
  • Fluorouracil
  • Cyclophosphamide
  • Doxorubicin
  • Paclitaxel
  • Breast Neoplasms



UT MD Anderson Cancer Center Houston, Texas  77030