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Peptide Vaccinations to Treat Patients With Low-Risk Myeloid Cancers

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ClinicalTrials.gov Identifier: NCT00488592
Recruitment Status : Completed
First Posted : June 20, 2007
Results First Posted : July 9, 2014
Last Update Posted : July 9, 2014
Sponsor:
Information provided by (Responsible Party):
Minoo Battiwalla, M.D., National Heart, Lung, and Blood Institute (NHLBI)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Myelodysplastic Syndrome (MDS)
Acute Myeloid Leukemia (AML)
Chronic Myeloid Leukemia (CML)
Interventions Biological: WT1:126-134
Biological: PR1:169-177 Peptide
Drug: GM-CSF (Sargramostim)
Biological: Montanide adjuvant
Enrollment 10

Recruitment Details  
Pre-assignment Details  
Arm/Group Title WT1/PR1
Hide Arm/Group Description Subjects with myeloid malignacies will receive Wilms' tumor (WT1)/ pathogenesis- related 1 (PR1) vaccine to evaluate an immune response to the intervention
Period Title: Overall Study
Started 10 [1]
Completed 7 [2]
Not Completed 3
Reason Not Completed
Withdrawal by Subject             1
Disease progression             1
Enrolled but not treated             1
[1]
10 subjects were accrued
[2]
7 completed the initial series of 6 vaccinations
Arm/Group Title WT1/PR1 Vaccine
Hide Arm/Group Description Subjects with myeloid malignacies will receive Wilms' tumor (WT1)/ pathogenesis- related 1 (PR1) vaccine to evaluate an immune response to the intervention
Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
<=18 years
0
   0.0%
Between 18 and 65 years
6
  60.0%
>=65 years
4
  40.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 10 participants
58.9  (13.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
7
  70.0%
Male
3
  30.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants
10
1.Primary Outcome
Title Efficacy in Inducing or Boosting a Cellular Immune Response
Hide Description A T-cell response was considered positive if the frequencies of interferon (IFN-γ+) cluster of differentiation (CD8+) T cells in peptide-stimulated peripheral bloody mono-nucleated cells (PBMCs) were 2-fold or more higher than the frequencies of interferon (IFN-γ+) CD8+ T cells in unstimulated PBMCs and if there was a minimum of 0.05% Interferon (IFNγ+) CD8+ T cells (after subtracting the frequencies of interferon (IFNγ+) CD8+ T cells in unstimulated PBMCs). A significant vaccine-induced CD8+ T-cell response was defined as the emergence of detectable PR1 or WT1-specific CD8+ T cells when the pre-study analysis found no response, or a 2-fold increase in frequencies when responses were present before vaccination.
Time Frame 16 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title WT1/PR1 Vaccine
Hide Arm/Group Description:
Subjects with myeloid malignancies will receive WT1/PR1 vaccine to evaluate immune response to intervention.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: participants
Cellular immune response 5
No cellular immune response 2
2.Secondary Outcome
Title Clinical Response
Hide Description Hematological response status
Time Frame 16 weeks
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title WT1/ PR1 Vaccine
Hide Arm/Group Description Subjects with myeloid malignacies will receive Wilms' tumor (WT1)/ pathogenesis- related 1 (PR1) vaccine to evaluate an immune response to the intervention
All-Cause Mortality
WT1/ PR1 Vaccine
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
WT1/ PR1 Vaccine
Affected / at Risk (%) # Events
Total   2/10 (20.00%)    
Gastrointestinal disorders   
Abdominal pain * [1]  1/10 (10.00%)  1
Infections and infestations   
Upper Respiratory Infection * [2]  1/10 (10.00%)  1
*
Indicates events were collected by non-systematic assessment
[1]
found to be related to relapsed disease
[2]
Bronchoscopy positive for staph and strep
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
WT1/ PR1 Vaccine
Affected / at Risk (%) # Events
Total   4/10 (40.00%)    
Blood and lymphatic system disorders   
decreased hemoglobin *  1/10 (10.00%)  1
Gastrointestinal disorders   
indigestion *  1/10 (10.00%)  1
loose stools *  2/10 (20.00%)  2
General disorders   
fatigue *  1/10 (10.00%)  1
lightheadness *  1/10 (10.00%)  1
malaise *  1/10 (10.00%)  1
rigors *  1/10 (10.00%)  1
Immune system disorders   
back pressure *  2/10 (20.00%)  2
Musculoskeletal and connective tissue disorders   
arthralgia *  4/10 (40.00%)  4
myalgia *  4/10 (40.00%)  4
Nervous system disorders   
headache *  2/10 (20.00%)  2
Skin and subcutaneous tissue disorders   
skin rash *  4/10 (40.00%)  4
*
Indicates events were collected by non-systematic assessment

Although not tested or described formally, no clinically meaningful hematological responses were seen.

Despite boosting, cellular immune responses were only transient.

Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Minoo Battiwalla, MD
Organization: Hematology Branch, NHLBI
Phone: 301 827 0939
Responsible Party: Minoo Battiwalla, M.D., National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT00488592     History of Changes
Obsolete Identifiers: NCT00499772
Other Study ID Numbers: 070159
07-H-0159 ( Other Identifier: National Heart Lung and Blood Institute )
First Submitted: June 19, 2007
First Posted: June 20, 2007
Results First Submitted: April 30, 2013
Results First Posted: July 9, 2014
Last Update Posted: July 9, 2014