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Vaccine Therapy and GM-CSF in Treating Patients With Acute Myeloid Leukemia in Remission

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: NCT00454168
Recruitment Status : Unknown
Verified February 2009 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
First Posted : March 30, 2007
Last Update Posted : January 6, 2014
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Vaccines made from a peptide may help the body build an effective immune response to kill cancer cells. Colony-stimulating factors, such as GM-CSF, increase the number of white blood cells and platelets found in bone marrow or peripheral blood. Giving vaccine therapy together with GM-CSF may be an effective treatment for acute myeloid leukemia. It is not yet known whether giving vaccine therapy together with GM-CSF is more effective than giving placebo together with GM-CSF in treating acute myeloid leukemia.

PURPOSE: This randomized phase III trial is studying vaccine therapy and GM-CSF to see how well they work compared with a placebo and GM-CSF in treating patients with acute myeloid leukemia in remission.

Condition or disease Intervention/treatment Phase
Leukemia Biological: PR1 leukemia peptide vaccine Biological: sargramostim Other: placebo Phase 3

Detailed Description:



  • Compare improvement of overall survival of patients with acute myeloid leukemia treated with PR1 leukemia peptide vaccine and sargramostim (GM-CSF) vs placebo vaccine and GM-CSF.


  • Compare improvement of relapse-free survival of patients treated with these regimens.
  • Compare remission duration in patients treated with these regimens.
  • Compare immune response, as measured by PR1-HLA-A2 tetramer assay, in patients treated with these regimens.

OUTLINE: This is a randomized, placebo-controlled, multicenter study. Patients are stratified according to age and complete remission (CR) (≥ 18 years of age and in second CR vs ≥ 55 years of age and in first CR), type of acute myeloid leukemia (de novo vs secondary), and cytogenetics (unfavorable vs favorable and intermediate). Patients are randomized to 1 of 2 treatment arms.

  • Arm I: Patients receive PR1 leukemia peptide vaccine and sargramostim (GM-CSF) subcutaneously (SC).
  • Arm II: Patients receive placebo vaccine and GM-CSF SC.

PROJECTED ACCRUAL: A total of 244 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 244 participants
Allocation: Randomized
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Multicenter Study of Proteinase 3 PR1 Peptide Mixed With Montanide ISA-51 VG Adjuvant and Administered With GM-CSF in Elderly Patients With AML in First Complete Remission or Adults in Second Complete Remission: A Pivotal Study
Study Start Date : May 2005
Estimated Primary Completion Date : April 2009

Arm Intervention/treatment
Experimental: Arm I
Patients receive PR1 leukemia peptide vaccine and sargramostim (GM-CSF) subcutaneously.
Biological: PR1 leukemia peptide vaccine
Given subcutaneously

Biological: sargramostim
Given subcutaneously

Active Comparator: Arm II
Patients receive placebo vaccine and GM-CSF subcutaneously.
Biological: sargramostim
Given subcutaneously

Other: placebo
Given subcutaneously

Primary Outcome Measures :
  1. Overall survival

Secondary Outcome Measures :
  1. Relapse-free survival
  2. Remission duration
  3. Immune response as measured by PR1-HLA-A2 tetramer assay

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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


  • Diagnosis of acute myeloid leukemia (AML), defined by the presence of > 20% blasts in marrow or blood, including the following subtypes:

    • De novo AML, defined as AML with no clinical history of prior myelodysplastic syndromes (MDS) or myeloproliferative disorder (MPD) or exposure to potentially leukemogenic therapies or agents
    • Secondary AML, defined as the following:

      • AML secondary to prior existing MDS or MPD or development of AML secondary to proven leukemogenic exposure
      • History of fatigue, bleeding, or recurrent infections preceding diagnosis of AML by ≥ 1 month with confirmation of existing peripheral blood film that demonstrates morphologic dysplasia
  • In first complete remission (CR) (patients ≥ 55 years of age) OR second CR (patients ≥ 18 years of age) within the past month

    • FAB stages M0-M2 and M4-M7 allowed if in first CR

      • No acute promyelocytic leukemia in first CR
    • FAB stages M0-M7 allowed if in second CR
    • Marrow blast count < 5% (≤ 200 nucleated cell count)

      • No blasts in blood
  • HLA-A2 positive at 1 allele
  • No extramedullary disease
  • No Auer rods
  • No active meningeal or CNS leukemia


  • ECOG performance status 0-1
  • Life expectancy must not be severely limited by other diseases
  • Absolute neutrophil count > 1,000/mm^3
  • Platelet count > 100,000/mm^3
  • Bilirubin < 2 mg/mL
  • ALT < 2 times upper limit of normal
  • Creatinine ≤ 1.6 mg/mL
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Antineutrophil cytoplasmic antibody negative
  • No serious medical condition, laboratory abnormality, or psychiatric illness that would preclude study compliance or increase risk to patient
  • No other malignancy within the past 5 years except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast
  • No known allergy to incomplete Freund's adjuvant
  • No hypercalcemia
  • No progressive viral or bacterial infection

    • Must be afebrile for 7 days without antibiotics
  • No symptomatic cardiac disease
  • LVEF ≥ 40%
  • No symptomatic pulmonary disease
  • FEV_1, FVC, and DLCO ≥ 50% of predicated (without bronchodilator)
  • No history of HIV positivity or AIDS
  • No known hypersensitivity to sargramostim (GM-CSF), yeast-derived products, or any component of this product
  • No history of Wegener's granulomatosis or vasculitis


  • Recovered from prior surgery and/or radiotherapy
  • No prior allogeneic or syngeneic stem cell transplantation
  • No prior solid organ transplantation
  • No prior vaccine therapy for AML
  • More than 28 days since prior chronic use (> 2 weeks) of corticosteroids > 10 mg/day (prednisone [or equivalent])

    • Concurrent topical or inhaled corticosteroids allowed
  • More than 3 months since prior experimental therapy, cyclosporine, or tacrolimus
  • No concurrent radiotherapy

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): NCT00454168

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United States, Arizona
Mayo Clinic Scottsdale
Scottsdale, Arizona, United States, 85259-5499
United States, California
Jonsson Comprehensive Cancer Center at UCLA
Los Angeles, California, United States, 90095-1781
Vaccine Company
South San Francisco, California, United States, 94080
United States, Illinois
Rush Cancer Institute at Rush University Medical Center
Chicago, Illinois, United States, 60612
University of Chicago Cancer Research Center
Chicago, Illinois, United States, 60637-1470
United States, Indiana
Indiana University Melvin and Bren Simon Cancer Center
Indianapolis, Indiana, United States, 46202-5289
St. Francis Hospital Cancer Care Services
Indianapolis, Indiana, United States, 46237
United States, Kansas
Kansas Masonic Cancer Research Institute at the University of Kansas Medical Center
Kansas City, Kansas, United States, 66160-7357
United States, Maryland
Greenebaum Cancer Center at University of Maryland Medical Center
Baltimore, Maryland, United States, 21201
United States, North Carolina
Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill
Chapel Hill, North Carolina, United States, 27599-7295
United States, Ohio
Case Comprehensive Cancer Center
Cleveland, Ohio, United States, 44106-5065
United States, Pennsylvania
UPMC Cancer Centers
Pittsburgh, Pennsylvania, United States, 15232
United States, South Carolina
Hollings Cancer Center at Medical University of South Carolina
Charleston, South Carolina, United States, 29425
United States, Texas
Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas
Dallas, Texas, United States, 75390
Cancer Care Centers of South Texas - Southeast
San Antonio, Texas, United States, 78222
Sponsors and Collaborators
The Vaccine Company
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Study Chair: Craig S. Rosenfeld, MD The Vaccine Company

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Responsible Party: Regulatory Protocol Associate, Vaccine Company Identifier: NCT00454168    
Other Study ID Numbers: CDR0000510853
First Posted: March 30, 2007    Key Record Dates
Last Update Posted: January 6, 2014
Last Verified: February 2009
Keywords provided by National Cancer Institute (NCI):
adult acute minimally differentiated myeloid leukemia (M0)
adult acute myeloblastic leukemia with maturation (M2)
adult acute myeloblastic leukemia without maturation (M1)
adult acute myeloid leukemia in remission
adult acute myeloid leukemia with 11q23 (MLL) abnormalities
adult acute myeloid leukemia with inv(16)(p13;q22)
adult acute myeloid leukemia with t(15;17)(q22;q12)
adult acute myeloid leukemia with t(16;16)(p13;q22)
adult acute myeloid leukemia with t(8;21)(q22;q22)
adult acute myelomonocytic leukemia (M4)
adult acute promyelocytic leukemia (M3)
secondary acute myeloid leukemia
adult acute monoblastic leukemia (M5a)
adult acute monocytic leukemia (M5b)
adult erythroleukemia (M6a)
adult pure erythroid leukemia (M6b)
adult acute megakaryoblastic leukemia (M7)
Additional relevant MeSH terms:
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Neoplasms by Histologic Type
Immunologic Factors
Physiological Effects of Drugs