Vaccine Therapy and GM-CSF in Treating Patients With Low-Risk or Intermediate-Risk Myelodysplastic Syndrome

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: NCT00513578
Recruitment Status : Unknown
Verified February 2009 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
First Posted : August 8, 2007
Last Update Posted : January 6, 2014
Information provided by:
National Cancer Institute (NCI)

August 6, 2007
August 8, 2007
January 6, 2014
January 2007
January 2009   (Final data collection date for primary outcome measure)
Immunologic response after four injections of vaccine formulation as determined by an increase in the absolute PR1-HLA-A2 tetramer count by at least 0.5/μl
Same as current
Complete list of historical versions of study NCT00513578 on Archive Site
  • Conversion of non-immunologic responders to immunologic responders by administering 4 additional doses of vaccine
  • Clinical response as determined by modified IWG criteria
Same as current
Not Provided
Not Provided
Vaccine Therapy and GM-CSF in Treating Patients With Low-Risk or Intermediate-Risk Myelodysplastic Syndrome
Phase 2 Study of Proteinase 3 PR1 Peptide Mixed With Montanide ISA 51 VG Adjuvant and Administered With GM-CSF in Low Risk and Intermediate-1 MDS

RATIONALE: Vaccines made from peptides 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 kill more cancer cells.

PURPOSE: This phase II trial is studying how well giving vaccine therapy together with GM-CSF works in treating patients with low-risk or intermediate-risk myelodysplastic syndrome.



  • To determine the immunologic response, using a PR1-HLA-A2 tetramer assay, to 4 subcutaneous injections of PR1 leukemia peptide vaccine formulated in incomplete Freund's adjuvant (IFA) followed by sargramostim (GM-CSF) in patients with low- and intermediate-1-risk myelodysplastic syndromes.


  • To determine if non-immunologic responders to 4 subcutaneous injections of PR1 leukemia peptide vaccine formulated in IFA followed by GM-CSF can be converted to immunologic responders by administering 4 additional doses of this treatment.
  • To determine the clinical response to 4 or 8 subcutaneous injections of this vaccine.

OUTLINE: This is a multicenter study.

Patients will receive proteinase PR1 leukemia peptide vaccine (TVC-PR1) conjugated with incomplete Freund's adjuvant administered subcutaneously with sargramostim (GM-CSF). Patients will receive a series of four vaccinations at 3-week intervals. Non-immunologic responders after 4 doses of vaccine are eligible to receive 4 additional doses of TVC-PR1 vaccine with the same dose and same dosing intervals. Patients who mount an immunologic response after 4 doses will not receive additional doses of TVC-PR1 vaccine.

After completion of study therapy, patients are followed monthly for up to 6 months.

Phase 2
Masking: None (Open Label)
Primary Purpose: Treatment
Myelodysplastic Syndromes
  • Biological: PR1 leukemia peptide vaccine
  • Biological: incomplete Freund's adjuvant
  • Biological: sargramostim
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Unknown status
Same as current
Not Provided
January 2009   (Final data collection date for primary outcome measure)


Inclusion criteria:

  • Diagnosis of myelodysplastic syndromes (MDS) and must meet all of the following criteria:

    • FAB class refractory anemia (RA), RA with excess blasts (RAEB), or RA with ringed sideroblasts (RARS)
    • WHO Classification RA, RARS, refractory cytopenia with multilineage dysplasia (RCMD), RCMD with ringed sideroblasts, or RAEB-1
    • Less than 20% blasts on marrow aspirate
    • IPSS risks groups intermediate-1- OR transfusion dependent low-risk
    • Patients with de novo or therapy-related MDS eligible
  • HLA-A2 positive at one allele

Exclusion criteria:

  • RAEB in transformation or RAEB-2
  • Chloroma
  • Marrow blasts on aspirate ≥ 20%
  • Blood blasts > 1%
  • Inaspirable bone marrow
  • History or current myelosclerosis occupying > 30% of marrow space
  • History of acute myeloid leukemia
  • Other causes of cytopenia not related to MDS (i.e., gastrointestinal blood loss)


Inclusion criteria:

  • ECOG performance status 0 or 1
  • Women of childbearing potential must have a negative serum pregnancy test within 30 days of starting study drug
  • Male or female of child-bearing potential must agree to use adequate contraceptive methods
  • Serum bilirubin < 2 mg/mL
  • Creatinine ≤ 1.5 mg/mL
  • ALT < 2 times upper normal limit
  • Antineutrophil cytoplasmic antibody (cANCA) negative

Exclusion criteria:

  • Pregnant or lactating
  • Iron absence on marrow examination or transferrin saturation < 20% and serum ferritin < 50ng/mL
  • B12 deficiency
  • Folate deficiency
  • History of immune-related hematological disorder (i.e., idiopathic thrombocytopenic purpura, autoimmune hemolytic anemia)
  • Life expectancy severely limited by diseases other than MDS
  • Prior history of malignancy other than MDS (except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast) unless the subject has been free of disease for ≥ 5 years
  • Known allergy to incomplete Freund's adjuvant
  • Hypercalcemia
  • Progressive viral or bacterial infection

    • All infections must be resolved and the patient has remained afebrile for seven days without antibiotics
  • Cardiac disease of symptomatic nature or cardiac ejection fraction < 40%
  • History of Wegener granulomatosis or vasculitis
  • Symptomatic pulmonary disease or FEV_1, FVC, and DLCO ≤ 50% predicted
  • History of HIV positivity or AIDS
  • Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form or that will place the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret the data


Exclusion criteria:

  • Has received specific therapy for MDS within the past 4 weeks
  • Prior allogeneic or syngeneic transplant
  • Prior solid organ transplant
  • Chronic use (> 2 weeks) of greater than physiologic doses of a corticosteroid agent (dose equivalent to > 10 mg/day of prednisone) within 30 days of the first day of study drug treatment

    • Topical and inhaled corticosteroids are permitted
  • Experimental therapy, cyclosporine, antithymocyte globulin, or tacrolimus within 3 months of study entry
  • Treatment with androgenic hormones, danazol, colony-stimulating factors, erythropoietin, thalidomide, arsenic trioxide or other agents used to treat MDS within four weeks of the first day of study treatment
  • Prior vaccine therapy for MDS
  • Prohibited medications during study, including any of the following:

    • Systemic steroids except as required for transfusion reactions
    • Chemotherapy or other investigational drugs
    • Sargramostim (GM-CSF) (except as part of study regimen)
    • Filgrastim (G-CSF)
    • Interleukin-11
Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
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Not Provided
The Vaccine Company
Not Provided
Study Chair: Craig S. Rosenfeld, MD The Vaccine Company
National Cancer Institute (NCI)
February 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP