An Early Phase 1 interventional study of K562/GM-CSF cell vaccine in Myelodysplastic Syndromes, sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. Terminated at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-03-23.
Sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Early Phase 1, Interventional, and Treatment
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).
OBJECTIVES:
Primary
Secondary
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.
1,317 studies on the registry are indexed under Preleukemia; 57 are open to participants now.
This study's enrollment of 9 is below the median of 36 across 1,060 interventional studies indexed under Preleukemia.
Browse Preleukemia studies →Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins is the lead sponsor of 572 studies on the registry; 73 are open to participants now.
Of its 111 completed or terminated interventional studies of FDA-regulated products, 67 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
DISEASE CHARACTERISTICS:
Pathologically confirmed myelodysplastic syndromes (MDS), including any of the following:
Must have poor-risk MDS, defined by the following:
PATIENT CHARACTERISTICS:
No active autoimmune disease or history of autoimmune disease requiring systemic immunosuppressants including, but not limited to, any of the following:
PRIOR CONCURRENT THERAPY:
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
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.
Time frame: Baseline, week 21 post-intervention
Cytogenetic Response Rate as Assessed by Number of Participants Achieving a Cytogenetic Response
Cytogenetic response is defined as normalization of pretreatment cytogenetic abnormalities.
Time frame: Week 21
Immune Response Rate as Assessed by Number of Participants Who Exhibit Induced Immune Response to WT-1, Survivin, or Proteinase-3
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.
Time frame: Baseline, week 21 post-intervention
Combined Immune and Clinical Response Rate
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.
Time frame: Week 21 post-intervention
| Milestone | K562/GM-CSF Cell Vaccine |
|---|---|
| Started | 5 |
| Completed | 2 |
| Not completed | 3 |
| Withdrew: Physician decision | 1 |
| Withdrew: Lack of efficacy | 2 |
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.
| Participants | K562/GM-CSF Cell Vaccine |
|---|---|
| Hematologic Response Rate as Assessed by Number of Participants Achieving a Major Hematologic Response | 1 |
Cytogenetic response is defined as normalization of pretreatment cytogenetic abnormalities.
| Participants | K562/GM-CSF Cell Vaccine |
|---|---|
| Cytogenetic Response Rate as Assessed by Number of Participants Achieving a Cytogenetic Response | 0 |
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.
| Participants | K562/GM-CSF Cell Vaccine |
|---|---|
| Immune Response Rate as Assessed by Number of Participants Who Exhibit Induced Immune Response to WT-1, Survivin, or Proteinase-3 | 0 |
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.
| Participants | K562/GM-CSF Cell Vaccine |
|---|---|
| Combined Immune and Clinical Response Rate | 0 |
Collected over Up to 18 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| K562/GM-CSF Cell Vaccine | 1/5 (20%) | 0/5 (0%) | 5/5 (100%) |
| Event | K562/GM-CSF Cell Vaccine |
|---|---|
| Injection site reactionsImmune system disorders | 5/5 |
| CoughRespiratory, thoracic and mediastinal disorders | 4/5 |
| HeadacheGeneral disorders | 4/5 |
| EdemaCardiac disorders | 3/5 |
| NauseaGastrointestinal disorders | 3/5 |
| AdenopathyImmune system disorders | 2/5 |
| ALT elevatedInvestigations | 2/5 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 2/5 |
| AST elevatedInvestigations | 2/5 |
| ConstipationGastrointestinal disorders | 2/5 |
| Age, Categorical(Participants) | K562/GM-CSF Cell Vaccine |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 3 |
| >=65 years | 2 |
| Age, Continuous(years) | K562/GM-CSF Cell Vaccine |
|---|---|
| Median | 64 (55 to 75) |
| Sex: Female, Male(Participants) | K562/GM-CSF Cell Vaccine |
|---|---|
| Female | 1 |
| Male | 4 |
| Ethnicity (NIH/OMB)(Participants) | K562/GM-CSF Cell Vaccine |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 5 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | K562/GM-CSF Cell Vaccine |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 2 |
| White | 3 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
Plan to share: No
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Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins