Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes
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).
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||K562/GM-CSF Vaccination in Patients With Myelodysplastic Syndrome|
- 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 ]
- 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 ]
|Study Start Date:||August 2006|
|Estimated Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00361296
|United States, Maryland|
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins|
|Baltimore, Maryland, United States, 21231|
|Principal Investigator:||B. Douglas Smith, MD||Sidney Kimmel Comprehensive Cancer Center|