Herceptin and GM-CSF for Metastatic Breast Cancer

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00429104
Recruitment Status : Completed
First Posted : January 31, 2007
Results First Posted : April 15, 2011
Last Update Posted : December 6, 2012
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:

Primary Objectives:

  1. To determine the patient's tumor response rate that this protocol will produce.
  2. To determine the 1 year progression-free survival that this protocol will produce.

Secondary Objective:

1. To determine whether antibody-dependent cell-mediated cytotoxicity (ADCC) is the mechanism of overcoming Herceptin-resistance by use of Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF).

Condition or disease Intervention/treatment Phase
Breast Cancer Drug: Herceptin Drug: GM-CSF Phase 2

Detailed Description:

GM-CSF stimulates the immune system and may increase the effectiveness of Herceptin.

Before you can start treatment on this study, you will have what are called "screening tests." These tests will help the doctor decide if you are eligible to take part in this study. You will have a complete medical history and physical exam. This includes blood tests (about 2 tea spoons), and x-rays. Women who are able to have children must have a negative blood or urine pregnancy test.

If you are found to be eligible to take part in this study, you will receive trastuzumab through a vein (IV) every week until the disease gets worse. GM-CSF will be injected under the skin at least once a day until the white blood cell count is stable. GM-CSF will also continue during the course of study until the disease progresses.

You will have further evaluation of your disease by computed tomography-CT scan, bone scan, chest X-ray, etc. at 2, 4, 6, 9, 12, 18, and 24 months after the start of treatment. You will have blood tests (about 2 tea spoons) at least twice a week until the appropriate dose of GM-CSF is found. The dose may increase or decrease depending on the blood test.

You will have blood (about 2 tablespoons) drawn before treatment at the 2nd and 4th month, and if the disease gets worse.

You will be removed from the study if the disease is progressing or severe side effects occur.

This is an investigational study. The FDA has approved trastuzumab and GM-CSF, but their use in this study is experimental. A total of 36 patients will take part in this study. All will be enrolled at UT MD Anderson Cancer Center.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Study of GM-CSF to Overcome Herceptin-Resistant HER-2/Neu-Overexpressing Metastatic Breast Cancer
Study Start Date : August 2002
Actual Primary Completion Date : November 2009
Actual Study Completion Date : November 2009

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Breast Cancer

Arm Intervention/treatment
Experimental: HER2+ Metastatic Breast Cancer
Herceptin 4 mg/kg IV Over 90 Minutes + GM-CSF 250 mcg/m^2 subcutaneously
Drug: Herceptin
4 mg/kg IV Over 90 Minutes
Other Name: Trastuzumab

Drug: GM-CSF
250 mcg/m^2 Subcutaneously
Other Names:
  • Sargramostim
  • Leukine

Primary Outcome Measures :
  1. Number of Participants With Tumor Response (Stable Disease) [ Time Frame: 2 months ]
    Number of participants with response defined as stable disease or better using Response Evaluation Criteria In Solid Tumors (RECIST) at the month 2 evaluation.

Secondary Outcome Measures :
  1. Duration of Stable Disease [ Time Frame: 6 Years ]
    Stable disease is measured from the start of the treatment until the RECIST criteria for disease progression is met.

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Histological confirmation of invasive carcinoma of the breast.
  2. HER-2/neu overexpression: 3+ by immunohistochemical staining or Fluorescence in situ hybridization (FISH) (+).
  3. Stage IV breast cancer with measurable disease.
  4. Patient receiving progressive disease after Herceptin plus chemotherapy or Herceptin alone. No more than two Herceptin containing regimens.
  5. Zubrod performance status 0 or 1.
  6. Adequate hematological parameters (White Blood cells-WBC > 3,000/mm3, platelet count > 100,000/mm3), adequate renal function (serum creatinine < 2.0 mg/dl), adequate liver function (total bilirubin, aspartate aminotransferase (AST or SGOT) or alanine aminotransferase (ALT or SGPT) < 3 x normal).

Exclusion Criteria:

  1. Active Brain metastasis.
  2. No measurable disease at the time of registration (e.g. bone only, leptomeningeal disease alone or pleural effusion alone).
  3. More than 2 Herceptin containing regimens in metastatic breast cancer.
  4. Known history of HIV positive.
  5. Chronic active hepatitis or cirrhosis.
  6. Symptomatic pulmonary disease.
  7. Use of steroid of non-steroidal anti-inflammatory analgesic or Cox-2 inhibitor 1 week prior to registration.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00429104

United States, Texas
U.T.M.D. Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Principal Investigator: Naoto Ueno, MD, PhD M.D. Anderson Cancer Center

Additional Information:
Responsible Party: M.D. Anderson Cancer Center Identifier: NCT00429104     History of Changes
Other Study ID Numbers: DM01-0100
First Posted: January 31, 2007    Key Record Dates
Results First Posted: April 15, 2011
Last Update Posted: December 6, 2012
Last Verified: December 2012

Keywords provided by M.D. Anderson Cancer Center:
Breast Cancer
Carcinoma of the Breast
HER-2/neu Overexpression

Additional relevant MeSH terms:
Breast Neoplasms
Neoplasms by Site
Breast Diseases
Skin Diseases
Antineoplastic Agents