This trial is active, not recruiting.

Condition myelodysplastic syndromes
Treatment gm-k562 cell vaccine
Phase phase 0
Sponsor Sidney Kimmel Comprehensive Cancer Center
Collaborator National Cancer Institute (NCI)
Start date August 2006
End date December 2016
Trial size 7 participants
Trial identifier NCT00361296, J05115, CDR0000491987, JHOC-J05115, NA_00001530, P30CA006973


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).

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Intervention model single group assignment
Masking open label
Primary purpose treatment
gm-k562 cell vaccine

Primary Outcomes

time frame:
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%
time frame:
Cytogenetic response, defined as normalization of pretreatment cytogenetic abnormalities
time frame:

Secondary Outcomes

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

Eligibility Criteria

Male or female participants from 18 years up to 120 years old.

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)

Additional Information

Official title K562/GM-CSF Vaccination in Patients With Myelodysplastic Syndrome
Principal investigator B. Douglas Smith, MD
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.
Trial information was received from ClinicalTrials.gov and was last updated in October 2015.
Information provided to ClinicalTrials.gov by Sidney Kimmel Comprehensive Cancer Center.