GM-CSF and Combination Chemotherapy in Treating Patients Who Are Undergoing Surgery for Stage II or Stage III Colon 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: NCT00262808
Recruitment Status : Completed
First Posted : December 7, 2005
Last Update Posted : October 16, 2013
Information provided by (Responsible Party):
University of Rochester

Brief Summary:

RATIONALE: Colony-stimulating factors, such as GM-CSF, may increase the number of immune cells found in bone marrow or peripheral blood and may help the immune system recover from the side effects of chemotherapy or kill tumor cells. Drugs used in chemotherapy, such as fluorouracil, leucovorin, and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Giving combination chemotherapy after surgery may kill any remaining tumor cells. Sometimes, after surgery, the tumor may not need additional treatment until it progresses. In this case, observation may be sufficient.

PURPOSE: This phase II trial is studying how well GM-CSF and combination chemotherapy work in treating patients who are undergoing surgery for stage II or stage III colon cancer.

Condition or disease Intervention/treatment Phase
Colorectal Cancer Biological: sargramostim Drug: fluorouracil Drug: leucovorin calcium Drug: oxaliplatin Procedure: adjuvant therapy Procedure: conventional surgery Procedure: neoadjuvant therapy Phase 2

Detailed Description:



  • Determine the safety of neoadjuvant and adjuvant sargramostim (GM-CSF) and adjuvant chemotherapy in patients with resectable stage II or III colon cancer.
  • Determine the efficacy, in terms of enhanced tumor-associated macrophage response, of this regimen in these patients.


  • Determine overall survival and time to progression in patients treated with this regimen.


  • Neoadjuvant therapy and surgery: Patients receive sargramostim (GM-CSF) subcutaneously (SC) once daily beginning between days -16 and -12 and continuing until day -1. Patients undergo surgical resection on day 0. Patients with stage I or IV disease are removed from the study. All other patients proceed to adjuvant chemotherapy or observation.
  • Adjuvant chemotherapy or observation: Patients with high-risk stage II or any stage III disease are assigned to group 1 or 2. Patients with low-risk stage II disease are assigned to group 3.

    • Group 1 (adjuvant therapy for high-risk stage II disease): Patients receive leucovorin calcium IV over 2 hours and fluorouracil IV on days 1, 8, 15, 22, 29 and 36. Patients also receive GM-CSF SC once daily on days 50-54. Treatment repeats every 56 days for up to 4 courses in the absence of disease progression or unacceptable toxicity.
    • Group 2 (adjuvant therapy for stage III disease): Patients receive adjuvant chemotherapy and GM-CSF as in group 1. Alternatively, patients may receive oxaliplatin IV over 2 hours and leucovorin calcium IV over 2 hours on day 1 and fluorouracil IV over 46 hours beginning on day 1. These patients also receive GM-CSF SC once daily on days 10-14 of every fourth course. Treatment repeats every 14 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
    • Group 3 (low-risk stage II disease): Patients undergo observation only every 3 months.

After completion of study treatment, patients are followed periodically for survival.

PROJECTED ACCRUAL: A total of 50 patients will be accrued for this study.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A PHASE II Study of GM-CSF As Pre- And Post-operative Adjuvant Therapy For Stage II And III Colon Cancer
Study Start Date : March 2004
Actual Primary Completion Date : September 2006
Actual Study Completion Date : September 2006

Primary Outcome Measures :
  1. Proportion of patients with a change in tumor-associated macrophage VEGF expression

Secondary Outcome Measures :
  1. Disease-free and overall survival

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed adenocarcinoma of the colon

    • Stage II or III disease
    • No carcinoma in situ
    • No perforated or obstructed tumors
    • No dual primary lesions by colonoscopy or barium enema
  • Resectable disease

    • Distal and proximal bowel end must be > 5 cm from tumor
    • Tumor must not extend below peritoneal reflection
  • No distant intra-abdominal metastases (even if resected)
  • No rectal cancer

    • No tumors that require opening of the pelvic peritoneum to define the extent of disease


Performance status

  • ECOG 0-2

Life expectancy

  • Not specified


  • WBC ≥ 3,000/mm^3
  • Platelet count ≥ 100,000/mm^3


  • Bilirubin ≤ 2.0 mg/dL


  • Creatinine ≤ 2.0 mg/dL


  • No symptomatic congestive heart failure
  • No unstable angina pectoris
  • No uncontrolled cardiac arrhythmia


  • No ongoing or active infection
  • No allergy to yeast or yeast-based products
  • No allergy to sargramostim (GM-CSF)
  • No allergy to fluorouracil
  • No allergy to leucovorin calcium


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No history of Crohn's disease
  • No history of ulcerative colitis
  • No other malignancy within the past 3 years except superficial squamous cell or basal cell skin cancer or carcinoma in situ of the cervix
  • No psychiatric illness or social situation that would preclude study compliance
  • No other uncontrolled illness



  • No prior chemotherapy, including fluorouracil, for colon cancer
  • No other concurrent chemotherapy


  • No prior radiotherapy for colon cancer
  • No concurrent radiotherapy


  • No other prior therapy for colon cancer
  • No concurrent immunosuppressant therapy
  • No other concurrent investigational agents
  • No other concurrent anticancer therapy

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): NCT00262808

United States, New York
James P. Wilmot Cancer Center at University of Rochester Medical Center
Rochester, New York, United States, 14642
United States, North Carolina
Duke Comprehensive Cancer Center
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
University of Rochester
Principal Investigator: Alok A. Khorana, MD James P. Wilmot Cancer Center

Responsible Party: University of Rochester Identifier: NCT00262808     History of Changes
Other Study ID Numbers: CDR0000448633
First Posted: December 7, 2005    Key Record Dates
Last Update Posted: October 16, 2013
Last Verified: October 2013

Keywords provided by University of Rochester:
stage II colon cancer
stage III colon cancer
adenocarcinoma of the colon

Additional relevant MeSH terms:
Colorectal Neoplasms
Colonic Neoplasms
Intestinal Neoplasms
Gastrointestinal Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Gastrointestinal Diseases
Colonic Diseases
Intestinal Diseases
Rectal Diseases
Antineoplastic Agents
Molecular Mechanisms of Pharmacological Action
Antimetabolites, Antineoplastic
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Protective Agents