K562/GM-CSF Vaccination in Patients With Myelodysplastic Syndrome
18 Years
N/A
Both
Myelodysplastic Syndromes
Trial Information
K562/GM-CSF Vaccination in Patients With Myelodysplastic Syndrome
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.
Inclusion 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)
Type of Study:
Interventional
Study Design:
Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Safety
Safety Issue:
Yes
Principal Investigator
B. Douglas Smith, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Sidney Kimmel Comprehensive Cancer Center
Authority:
United States: Institutional Review Board
Study ID:
J05115, CDR0000491987
NCT ID:
NCT00361296
Start Date:
August 2006
Completion Date:
Related Keywords:
- Myelodysplastic Syndromes
- 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
- Myelodysplastic Syndromes
- Preleukemia
Name | Location |
|---|---|
| Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Baltimore, Maryland 21231-2410 |
