Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes

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: NCT00361296
Recruitment Status : Unknown
Verified October 2015 by Sidney Kimmel Comprehensive Cancer Center.
Recruitment status was:  Active, not recruiting
First Posted : August 8, 2006
Last Update Posted : November 2, 2015
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center

Brief Summary:

RATIONALE: Vaccines made from cancer cells may help the body build an effective immune response to kill abnormal cells.

PURPOSE: This clinical trial is studying how well vaccine therapy works in treating patients with myelodysplastic syndromes (MDS).

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Biological: GM-K562 cell vaccine Early Phase 1

Detailed Description:



  • Determine the safety of GM-K562 cell vaccine in patients with myelodysplastic syndromes.
  • Determine the hematologic and cytogenetic response in patients treated with this vaccine.


  • Determine if vaccination with GM-K562 cell vaccine can induce an immune response to common myeloid antigens (e.g., Wilms' tumor-1 [WT-1], survivin, or proteinase-3), as defined by a 30% increase from baseline in specific cytotoxic T-cells measured by Elispot assay, in patients with myelodysplastic syndromes.
  • Determine if immune response correlates with any clinical responses (e.g., hematologic response, resolution of cytogenetic abnormalities, or decrease in other parameters, such as WT-1 mRNA levels).

OUTLINE: This is an open-label study.

Patients receive GM-K562 cell vaccine subcutaneously once in weeks 0, 3, 6, 9, and 17 in the absence of disease progression or unacceptable toxicity.

Blood and tissue samples are collected periodically for correlative and biomarker studies. Samples are analyzed by cytogenetic studies, fluorescent in situ hybridization (FISH), and flow cytometry. Elispot is used to quantify cellular cytotoxic T-cell response to Wilms' tumor-1 (WT-1), survivin, and proteinase 3.

After completion of study treatment, patients are followed every 3 months for 1 year.

PROJECTED ACCRUAL: A total of 15 patients will be accrued for this study.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: K562/GM-CSF Vaccination in Patients With Myelodysplastic Syndrome
Study Start Date : August 2006
Estimated Primary Completion Date : December 2016
Estimated Study Completion Date : December 2017

Arm Intervention/treatment
Experimental: GM-K562 cell vaccine Biological: GM-K562 cell vaccine

Primary Outcome Measures :
  1. Safety
  2. Hematologic response, defined as achieving a major response in ≥ 1 lineage as described by an erythroid increase > 2 g/dL, platelet increase of 30,000/mm³, or neutrophil increase by 100%
  3. Cytogenetic response, defined as normalization of pretreatment cytogenetic abnormalities

Secondary Outcome Measures :
  1. Immune response to common myeloid antigens (e.g., Wilms' tumor-1 [WT-1], survivin, or proteinase-3) as measured by Elispot assay
  2. Correlation of immune response with clinical response (hematologic response, resolution of cytogenetic abnormalities, or decrease in other parameters, such as WT-1 mRNA levels)

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.

Ages Eligible for Study:   18 Years to 120 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Pathologically confirmed myelodysplastic syndromes (MDS), including any of the following:

    • Refractory anemia (RA)
    • RA with ringed sideroblasts
    • Refractory cytopenias with multilineage dysplasia (RCMD)
    • RCMD with ringed sideroblasts
    • RA with excess blasts 1 (5-9% blasts)
    • RA with excess blasts 2 (10-19% blasts)
  • Must have poor-risk MDS, defined by the following:

    • At least 2 lineages involved
    • Unfavorable cytogenetics (i.e., abnormalities of chromosome 5 or 7, 11q23, t[6;9], trisomy 8, inv3, or multiple/complex karyotype)
    • Transfusion requirement of > 2 units of packed red blood cells monthly
  • No chronic myelomonocytic leukemia
  • No transformation to acute myeloid leukemia


  • ECOG performance status 0-2
  • Creatinine < 2.5 mg/dL
  • Bilirubin < 2.5 mg/dL (unless due to Gilbert's syndrome)
  • Room air oxygen saturation ≥ 94% at rest
  • Fertile patients must use effective contraception
  • Negative pregnancy test
  • No other malignancy within the past 5 years except in situ cervical cancer or adequately treated nonmelanoma skin cancer
  • No active autoimmune disease or history of autoimmune disease requiring systemic immunosuppressants including, but not limited to, any of the following:

    • Autoimmune hemolytic anemia
    • Idiopathic thrombocytopenia purpura
    • Inflammatory bowel disease
    • Vasculitis
    • Thyroiditis
    • Rheumatic illnesses
  • No known HIV serum antibody positivity
  • No other disease requiring long-term corticosteroids or other immunosuppressants, such as severe chronic obstructive pulmonary disease or asthma


  • At least 2 weeks since prior systemic corticosteroids or other immunosuppressants (e.g., cyclosporine, azathioprine, tacrolimus, or mycophenolate mofetil)
  • At least 3 weeks since prior growth factors
  • At least 2 months since prior azacitidine for MDS
  • No prior bone marrow or other organ transplantation
  • No concurrent cytotoxic-based therapy for MDS
  • No other concurrent growth factors, including epoetin alfa, filgrastim (G-CSF), or sargramostim (GM-CSF)

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): NCT00361296

United States, Maryland
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center
National Cancer Institute (NCI)
Principal Investigator: B. Douglas Smith, MD Sidney Kimmel Comprehensive Cancer Center

Responsible Party: Sidney Kimmel Comprehensive Cancer Center Identifier: NCT00361296     History of Changes
Other Study ID Numbers: J05115, CDR0000491987
P30CA006973 ( U.S. NIH Grant/Contract )
NA_00001530 ( Other Identifier: JHMIRB )
First Posted: August 8, 2006    Key Record Dates
Last Update Posted: November 2, 2015
Last Verified: October 2015

Keywords provided by Sidney Kimmel Comprehensive Cancer Center:
refractory anemia with excess blasts
refractory anemia with ringed sideroblasts
refractory anemia
refractory cytopenia with multilineage dysplasia
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Immunologic Factors
Physiological Effects of Drugs