Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov Identifier:
NCT00361296
First received: August 4, 2006
Last updated: March 20, 2014
Last verified: March 2014
  Purpose

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 Intervention Phase
Myelodysplastic Syndromes
Biological: GM-K562 cell vaccine
Phase 0

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: K562/GM-CSF Vaccination in Patients With Myelodysplastic Syndrome

Resource links provided by NLM:


Further study details as provided by Sidney Kimmel Comprehensive Cancer Center:

Primary Outcome Measures:
  • Safety [ Designated as safety issue: Yes ]
  • 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% [ Designated as safety issue: No ]
  • Cytogenetic response, defined as normalization of pretreatment cytogenetic abnormalities [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Immune response to common myeloid antigens (e.g., Wilms' tumor-1 [WT-1], survivin, or proteinase-3) as measured by Elispot assay [ Designated as safety issue: No ]
  • Correlation of immune response with clinical response (hematologic response, resolution of cytogenetic abnormalities, or decrease in other parameters, such as WT-1 mRNA levels) [ Designated as safety issue: No ]

Enrollment: 7
Study Start Date: August 2006
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GM-K562 cell vaccine Biological: GM-K562 cell vaccine

Detailed Description:

OBJECTIVES:

Primary

  • 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.

Secondary

  • 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • 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

PATIENT CHARACTERISTICS:

  • 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

PRIOR CONCURRENT THERAPY:

  • 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)
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00361296

Locations
United States, Maryland
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center
Investigators
Principal Investigator: B. Douglas Smith, MD Sidney Kimmel Comprehensive Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov Identifier: NCT00361296     History of Changes
Other Study ID Numbers: J05115, CDR0000491987, P30CA006973, JHOC-J05115
Study First Received: August 4, 2006
Last Updated: March 20, 2014
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration

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
Preleukemia
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms

ClinicalTrials.gov processed this record on August 26, 2014