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Pilot Trial of a WT-1 Analog Peptide Vaccine in Patients With Myeloid Neoplasms

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ClinicalTrials.gov Identifier: NCT00665002
Recruitment Status : Completed
First Posted : April 23, 2008
Results First Posted : July 24, 2014
Last Update Posted : February 27, 2015
Sponsor:
Collaborator:
Innovive Pharmaceuticals
Information provided by (Responsible Party):
H. Lee Moffitt Cancer Center and Research Institute

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Leukemia
Interventions Biological: WT-1
Drug: Montanide
Drug: Sargramostim (GM-CSF)
Enrollment 16
Recruitment Details Participants were recruited at Moffitt Cancer Center from 06/30/2008 to 12/13/2012.
Pre-assignment Details  
Arm/Group Title Experimental: WT-1 Analog Peptide Vaccine
Hide Arm/Group Description Participants received 6 bi-weekly vaccinations over 10 weeks. WT-1 vaccine was given with Montanide. Participants also received an injection of Sargramostim (GM-CSF) two days before each vaccination and again on the day of the WT-1 injection at the same spot.
Period Title: Overall Study
Started 16
Completed 4
Not Completed 12
Reason Not Completed
Progression/relapse during treatment             11
Cytogenetic resistance             1
Arm/Group Title Experimental: WT-1 Analog Peptide Vaccine
Hide Arm/Group Description Participants received 6 bi-weekly vaccinations over 10 weeks. WT-1 vaccine was given with Montanide. Participants also received an injection of Sargramostim (GM-CSF) two days before each vaccination and again on the day of the WT-1 injection at the same spot.
Overall Number of Baseline Participants 16
Hide Baseline Analysis Population Description
All participants
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 16 participants
73
(65 to 82)
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
<=18 years
0
   0.0%
Between 18 and 65 years
0
   0.0%
>=65 years
16
 100.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
Female
6
  37.5%
Male
10
  62.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 16 participants
16
1.Primary Outcome
Title Number of Participants With Adverse Events (AEs)
Hide Description Toxicities were tabulated according to the NCI Common Toxicity (version 3.0) by grade and category. If more than one patient developed ≥ grade 3 non-hematologic toxicity or grade 4 hematologic toxicity, the study accrual was to be suspended immediately for a careful toxicity data evaluation. Depending upon the findings of such safety/toxicity data assessment and consultation with the supporting pharmaceutical company, the principal investigator of this trial would have the option of terminating this trial permanently, amending the study protocol, or resuming the patient accrual.
Time Frame 12 weeks to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
All participants
Arm/Group Title Experimental: WT-1 Analog Peptide Vaccine
Hide Arm/Group Description:
Participants received 6 bi-weekly vaccinations over 10 weeks. WT-1 vaccine was given with Montanide. Participants also received an injection of Sargramostim (GM-CSF) two days before each vaccination and again on the day of the WT-1 injection at the same spot.
Overall Number of Participants Analyzed 16
Measure Type: Number
Unit of Measure: participants
15
2.Secondary Outcome
Title Participants Whose Samples Demonstrated Immunological Response After Vaccination
Hide Description

Immune Response: Immune reactivity was measured for all participants. Immune response was measured by T cell proliferative response and DTH against WT-1 peptides. In patients with adequate samples, T cell gamma interferon release as measured by ELISPOT and/or multiparameter intracellular staining by flow cytometry were performed as well.

ELISPOT Assay: CD4+ immune response, CD4+ and CD8+ response. The samples of participants' blood obtained at baseline and week 12 were tested for CD4 T cell proliferation, CD4 and CD8 T cell interferon release.

Tetramer Analysis of WT1-specific Immune responses: subtle WT1 T cell expansion, positive by ELISPOT and T cell expansion.

Delayed-type Hypersensitivity (DTH): measurable DTH response without overlap with ELISPOT or tetramer responders).

Overall: any form of immune response.

Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All participants
Arm/Group Title Experimental: WT-1 Analog Peptide Vaccine
Hide Arm/Group Description:
Participants received 6 bi-weekly vaccinations over 10 weeks. WT-1 vaccine was given with Montanide. Participants also received an injection of Sargramostim (GM-CSF) two days before each vaccination and again on the day of the WT-1 injection at the same spot.
Overall Number of Participants Analyzed 16
Measure Type: Number
Unit of Measure: participants
CD4+ Immune Response 4
CD4+ and CD8+ Response 3
WT1 T Cell Expansion 2
Positive by ELISPOT and T Cell Expansion 2
Measurable DTH Response 2
Any form of Immune Response 6
Time Frame 4 years, 4 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Experimental: WT-1 Analog Peptide Vaccine
Hide Arm/Group Description Participants received 6 bi-weekly vaccinations over 10 weeks.
All-Cause Mortality
Experimental: WT-1 Analog Peptide Vaccine
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Experimental: WT-1 Analog Peptide Vaccine
Affected / at Risk (%) # Events
Total   2/16 (12.50%)    
Gastrointestinal disorders   
Gastrointestinal - Other - Appendicitis - unrelated  1  1/16 (6.25%)  1
General disorders   
Death Within 100 days - unrelated  1  1/16 (6.25%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Experimental: WT-1 Analog Peptide Vaccine
Affected / at Risk (%) # Events
Total   15/16 (93.75%)    
Blood and lymphatic system disorders   
Platelets - low  1  10/16 (62.50%)  16
Leukocytes (total WBC) - low  1  4/16 (25.00%)  5
Neutrophils/granulocytes (ANC/AGC) - low  1  4/16 (25.00%)  12
Hemoglobin - low  1  3/16 (18.75%)  7
Bone marrow cellularity  1  1/16 (6.25%)  1
Petechiae/purpura (hemorrhage/bleeding into skin or mucosa)  1  2/16 (12.50%)  2
Coagulation - Other  1  1/16 (6.25%)  1
Thrombotic microangiopathy  1  1/16 (6.25%)  1
Edema: limb  1  1/16 (6.25%)  1
Endocrine disorders   
Hot flashes/flushes  1  1/16 (6.25%)  1
Eye disorders   
Dry eye syndrome  1  1/16 (6.25%)  1
Ocular/Visual - Other  1  1/16 (6.25%)  1
Gastrointestinal disorders   
Constipation  1  4/16 (25.00%)  5
Diarrhea  1  3/16 (18.75%)  3
Nausea  1  3/16 (18.75%)  5
Anorexia  1  2/16 (12.50%)  2
Distension/bloating, abdominal  1  2/16 (12.50%)  2
Gastrointestinal - Other  1  2/16 (12.50%)  2
Dry mouth/salivary gland (xerostomia)  1  1/16 (6.25%)  1
Mucositis/stomatitis (clinical exam) - Anus  1  1/16 (6.25%)  1
Mucositis/stomatitis (clinical exam) - Oral cavity  1  1/16 (6.25%)  1
Vomiting  1  1/16 (6.25%)  1
General disorders   
Fatigue (asthenia, lethargy, malaise)  1  8/16 (50.00%)  10
Insomnia  1  1/16 (6.25%)  1
Rigors/chills  1  1/16 (6.25%)  1
Pain - Abdomen NOS  1  3/16 (18.75%)  4
Pain - Other  1  3/16 (18.75%)  5
Pain - Bone  1  1/16 (6.25%)  1
Pain - Head/headache  1  1/16 (6.25%)  1
Pain - Joint  1  1/16 (6.25%)  3
Pain - Muscle  1  1/16 (6.25%)  1
Pain - Neck  1  1/16 (6.25%)  2
Pain - Sinus  1  1/16 (6.25%)  1
Flu-like syndrome  1  1/16 (6.25%)  1
Immune system disorders   
Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip  1  1/16 (6.25%)  1
Metabolism and nutrition disorders   
Glucose, serum-high (hyperglycemia)  1  2/16 (12.50%)  2
Metabolic/Laboratory - Other  1  2/16 (12.50%)  4
Alkaline phosphatase  1  1/16 (6.25%)  2
ALT, SGPT (serum glutamic pyruvic transaminase)  1  1/16 (6.25%)  1
Bilirubin (hyperbilirubinemia)  1  1/16 (6.25%)  1
Potassium, serum-low (hypokalemia)  1  1/16 (6.25%)  1
Musculoskeletal and connective tissue disorders   
Arthritis (non-septic)  1  1/16 (6.25%)  1
Fracture  1  1/16 (6.25%)  1
Muscle weakness, generalized or specific area (not due to neuropathy) Whole body/generalized  1  1/16 (6.25%)  3
Nervous system disorders   
Dizziness  1  2/16 (12.50%)  2
Neuropathy: sensory  1  2/16 (12.50%)  4
Memory impairment  1  1/16 (6.25%)  1
Psychiatric disorders   
Mood alteration - Anxiety  1  1/16 (6.25%)  1
Renal and urinary disorders   
Renal/Genitourinary - Other  1  1/16 (6.25%)  1
Urinary frequency/urgency  1  1/16 (6.25%)  1
Respiratory, thoracic and mediastinal disorders   
Cough  1  4/16 (25.00%)  5
Dyspnea (shortness of breath)  1  1/16 (6.25%)  1
Skin and subcutaneous tissue disorders   
Injection site reaction/extravasation changes  1  7/16 (43.75%)  19
Dermatology/Skin - Other  1  3/16 (18.75%)  3
Pruritus/itching  1  2/16 (12.50%)  2
Rash: erythema multiforme  1  2/16 (12.50%)  3
Bruising (in absence of Grade 3 or 4 thrombocytopenia)  1  1/16 (6.25%)  2
Dry skin  1  1/16 (6.25%)  1
Flushing  1  1/16 (6.25%)  1
Hair loss/alopecia (scalp or body)  1  1/16 (6.25%)  1
Vascular disorders   
Hematoma  1  1/16 (6.25%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jeffrey E. Lancet, M.D.
Organization: H. Lee Moffitt Cancer Center and Research Institute
Phone: 813-745-6841
EMail: jeffrey.lancet@moffitt.org
Layout table for additonal information
Responsible Party: H. Lee Moffitt Cancer Center and Research Institute
ClinicalTrials.gov Identifier: NCT00665002     History of Changes
Other Study ID Numbers: MCC-15025
INNO-305 WT-1 ( Other Identifier: Innovive )
105946 ( Other Identifier: USF IRB )
First Submitted: April 18, 2008
First Posted: April 23, 2008
Results First Submitted: June 25, 2014
Results First Posted: July 24, 2014
Last Update Posted: February 27, 2015