Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes
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|ClinicalTrials.gov Identifier: NCT00361296|
Recruitment Status : Terminated (Loss of funding)
First Posted : August 8, 2006
Results First Posted : November 16, 2018
Last Update Posted : November 16, 2018
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: K562/GM-CSF cell vaccine||Early Phase 1|
- 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 :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||K562/GM-CSF Vaccination in Patients With Myelodysplastic Syndrome|
|Actual Study Start Date :||September 2007|
|Actual Primary Completion Date :||October 2009|
|Actual Study Completion Date :||January 2010|
Experimental: K562/GM-CSF cell vaccine
Vaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.
Biological: K562/GM-CSF cell vaccine
- Hematologic Response Rate as Assessed by Number of Participants Achieving a Major Hematologic Response [ Time Frame: Baseline, week 21 post-intervention ]A major hematologic response is defined as any of the following: hemoglobin increase >= 2 g/dL from baseline; platelet increase >= 30k/mcL from baseline; or neutrophil increase >= 100% or >= 500/mcL from baseline.
- Cytogenetic Response Rate as Assessed by Number of Participants Achieving a Cytogenetic Response [ Time Frame: Week 21 ]Cytogenetic response is defined as normalization of pretreatment cytogenetic abnormalities.
- Immune Response Rate as Assessed by Number of Participants Who Exhibit Induced Immune Response to WT-1, Survivin, or Proteinase-3 [ Time Frame: Baseline, week 21 post-intervention ]Immune response to WT-1, survivin, or proteinase-3 as defined by a 30% increase from baseline in cytotoxic T cells measured by Elispot analysis.
- Combined Immune and Clinical Response Rate [ Time Frame: Week 21 post-intervention ]Number of participants who exhibited both an immune response as defined by Outcome 3 and a hematologic or cytogenetic response as defined by Outcomes 1 and 2, respectively.
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 ClinicalTrials.gov identifier (NCT number): 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 at Johns Hopkins|