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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
Sponsor:
Collaborators:
National Cancer Institute (NCI)
Alliance for Cancer Gene Therapy
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Myelodysplastic Syndromes
Intervention Biological: K562/GM-CSF cell vaccine
Enrollment 9
Recruitment Details  
Pre-assignment Details 2 participants were screen failures. 2 additional participants were removed from study by physician decision prior to receiving protocol intervention, so they never started the study.
Arm/Group Title K562/GM-CSF Cell Vaccine
Hide Arm/Group Description

Vaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.

K562/GM-CSF cell vaccine

Period Title: Overall Study
Started 5
Completed 2
Not Completed 3
Reason Not Completed
Physician Decision             1
Lack of Efficacy             2
Arm/Group Title K562/GM-CSF Cell Vaccine
Hide Arm/Group Description

Vaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.

K562/GM-CSF cell vaccine

Overall Number of Baseline Participants 5
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
<=18 years
0
   0.0%
Between 18 and 65 years
3
  60.0%
>=65 years
2
  40.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 5 participants
64
(55 to 75)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Female
1
  20.0%
Male
4
  80.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
5
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
2
  40.0%
White
3
  60.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
1.Primary Outcome
Title Hematologic Response Rate as Assessed by Number of Participants Achieving a Major Hematologic Response
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title K562/GM-CSF Cell Vaccine
Hide Arm/Group Description:

Vaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.

K562/GM-CSF cell vaccine

Overall Number of Participants Analyzed 5
Measure Type: Count of Participants
Unit of Measure: Participants
1
  20.0%
2.Primary Outcome
Title Cytogenetic Response Rate as Assessed by Number of Participants Achieving a Cytogenetic Response
Hide Description Cytogenetic response is defined as normalization of pretreatment cytogenetic abnormalities.
Time Frame Week 21
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title K562/GM-CSF Cell Vaccine
Hide Arm/Group Description:

Vaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.

K562/GM-CSF cell vaccine

Overall Number of Participants Analyzed 5
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
3.Secondary Outcome
Title Immune Response Rate as Assessed by Number of Participants Who Exhibit Induced Immune Response to WT-1, Survivin, or Proteinase-3
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title K562/GM-CSF Cell Vaccine
Hide Arm/Group Description:

Vaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.

K562/GM-CSF cell vaccine

Overall Number of Participants Analyzed 5
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
4.Secondary Outcome
Title Combined Immune and Clinical Response Rate
Hide Description 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title K562/GM-CSF Cell Vaccine
Hide Arm/Group Description:

Vaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.

K562/GM-CSF cell vaccine

Overall Number of Participants Analyzed 5
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
Time Frame Up to 18 months
Adverse Event Reporting Description Adverse events were collected at weeks 3, 6, 9, 13, 17, and every three months after week 21 for a total of one additional year.
 
Arm/Group Title K562/GM-CSF Cell Vaccine
Hide Arm/Group Description

Vaccinations of 1x10^8 cells are given to participants at weeks 0, 3, 6, 9, and 17.

K562/GM-CSF cell vaccine

All-Cause Mortality
K562/GM-CSF Cell Vaccine
Affected / at Risk (%)
Total   1/5 (20.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
K562/GM-CSF Cell Vaccine
Affected / at Risk (%) # Events
Total   0/5 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
K562/GM-CSF Cell Vaccine
Affected / at Risk (%) # Events
Total   5/5 (100.00%)    
Cardiac disorders   
Heart failure  1  1/5 (20.00%)  1
Edema  1  3/5 (60.00%)  5
Tachycardia  1  1/5 (20.00%)  1
Eye disorders   
Bleeding - eye  1  1/5 (20.00%)  1
Xerophthalmia  1  1/5 (20.00%)  1
Gastrointestinal disorders   
Abdominal distension  1  1/5 (20.00%)  1
Constipation  1  2/5 (40.00%)  2
Gastroenteritis  1  1/5 (20.00%)  1
Hemorrhoids  1  1/5 (20.00%)  1
Nausea  1  3/5 (60.00%)  4
Vomiting  1  1/5 (20.00%)  1
General disorders   
Anorexia  1  1/5 (20.00%)  1
Bruising  1  1/5 (20.00%)  1
Fatigue  1  1/5 (20.00%)  2
Fever  1  1/5 (20.00%)  1
Flu-like symptoms  1  1/5 (20.00%)  3
Flushing  1  1/5 (20.00%)  1
Headache  1  4/5 (80.00%)  6
Insomnia  1  2/5 (40.00%)  2
Epistaxis  1  1/5 (20.00%)  1
Night sweats  1  1/5 (20.00%)  1
Sore throat  1  1/5 (20.00%)  1
Swollen gums  1  1/5 (20.00%)  1
Weight gain  1  1/5 (20.00%)  1
Immune system disorders   
Adenopathy  1  2/5 (40.00%)  2
Injection site reactions  1  5/5 (100.00%)  25
Transfusion reaction  1  1/5 (20.00%)  2
Infections and infestations   
Bacteremia  1  1/5 (20.00%)  1
Lyme disease  1  1/5 (20.00%)  1
Respiratory infection  1  2/5 (40.00%)  3
Zoster infection  1  1/5 (20.00%)  2
Investigations   
ALT elevated  1  2/5 (40.00%)  2
Anemia  1  1/5 (20.00%)  2
AST elevated  1  2/5 (40.00%)  2
Creatinine elevated  1  1/5 (20.00%)  1
Hyperbilirubinemia  1  1/5 (20.00%)  1
Hyperglycemia  1  1/5 (20.00%)  1
Hyperphosphatemia  1  1/5 (20.00%)  1
Hyperuricemia  1  1/5 (20.00%)  1
Leukopenia  1  1/5 (20.00%)  1
Neutropenia  1  1/5 (20.00%)  1
Thrombocytopenia  1  2/5 (40.00%)  4
Musculoskeletal and connective tissue disorders   
Arthralgia  1  2/5 (40.00%)  2
Pain - flank  1  1/5 (20.00%)  1
Nervous system disorders   
Altered mental status  1  1/5 (20.00%)  1
Dizziness  1  2/5 (40.00%)  2
Neuropathy  1  2/5 (40.00%)  2
Taste changes  1  1/5 (20.00%)  2
Tinnitus  1  1/5 (20.00%)  1
Vision changes  1  1/5 (20.00%)  1
Respiratory, thoracic and mediastinal disorders   
Cough  1  4/5 (80.00%)  4
Dyspnea  1  2/5 (40.00%)  2
Nasal congestion  1  2/5 (40.00%)  2
Skin and subcutaneous tissue disorders   
Erythema - mouth  1  1/5 (20.00%)  1
Rash  1  2/5 (40.00%)  2
Xerosis  1  1/5 (20.00%)  1
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Doug Smith, MD
Organization: Johns Hopkins University
Phone: 4102872935
Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier: NCT00361296     History of Changes
Other Study ID Numbers: J05115
P30CA006973 ( U.S. NIH Grant/Contract )
NA_00001530 ( Other Identifier: JHMIRB )
First Submitted: August 4, 2006
First Posted: August 8, 2006
Results First Submitted: October 18, 2018
Results First Posted: November 16, 2018
Last Update Posted: November 16, 2018