Trial Outcomes & Findings for Pilot Trial of a WT-1 Analog Peptide Vaccine in Patients With Myeloid Neoplasms (NCT NCT00665002)
NCT ID: NCT00665002
Last Updated: 2015-02-27
Results Overview
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.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
16 participants
Primary outcome timeframe
12 weeks to 6 months
Results posted on
2015-02-27
Participant Flow
Participants were recruited at Moffitt Cancer Center from 06/30/2008 to 12/13/2012.
Participant milestones
| Measure |
Experimental: WT-1 Analog Peptide Vaccine
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 Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
12
|
Reasons for withdrawal
| Measure |
Experimental: WT-1 Analog Peptide Vaccine
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.
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|---|---|
|
Overall Study
Progression/relapse during treatment
|
11
|
|
Overall Study
Cytogenetic resistance
|
1
|
Baseline Characteristics
Pilot Trial of a WT-1 Analog Peptide Vaccine in Patients With Myeloid Neoplasms
Baseline characteristics by cohort
| Measure |
Experimental: WT-1 Analog Peptide Vaccine
n=16 Participants
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.
|
|---|---|
|
Age, Continuous
|
73 years
n=5 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
16 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
16 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 weeks to 6 monthsPopulation: All participants
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.
Outcome measures
| Measure |
Experimental: WT-1 Analog Peptide Vaccine
n=16 Participants
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.
|
|---|---|
|
Number of Participants With Adverse Events (AEs)
|
15 participants
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: All participants
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.
Outcome measures
| Measure |
Experimental: WT-1 Analog Peptide Vaccine
n=16 Participants
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.
|
|---|---|
|
Participants Whose Samples Demonstrated Immunological Response After Vaccination
CD4+ Immune Response
|
4 participants
|
|
Participants Whose Samples Demonstrated Immunological Response After Vaccination
CD4+ and CD8+ Response
|
3 participants
|
|
Participants Whose Samples Demonstrated Immunological Response After Vaccination
WT1 T Cell Expansion
|
2 participants
|
|
Participants Whose Samples Demonstrated Immunological Response After Vaccination
Positive by ELISPOT and T Cell Expansion
|
2 participants
|
|
Participants Whose Samples Demonstrated Immunological Response After Vaccination
Any form of Immune Response
|
6 participants
|
|
Participants Whose Samples Demonstrated Immunological Response After Vaccination
Measurable DTH Response
|
2 participants
|
Adverse Events
Experimental: WT-1 Analog Peptide Vaccine
Serious events: 2 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Experimental: WT-1 Analog Peptide Vaccine
n=16 participants at risk
Participants received 6 bi-weekly vaccinations over 10 weeks.
|
|---|---|
|
Gastrointestinal disorders
Gastrointestinal - Other - Appendicitis - unrelated
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
General disorders
Death Within 100 days - unrelated
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
Other adverse events
| Measure |
Experimental: WT-1 Analog Peptide Vaccine
n=16 participants at risk
Participants received 6 bi-weekly vaccinations over 10 weeks.
|
|---|---|
|
Blood and lymphatic system disorders
Platelets - low
|
62.5%
10/16 • Number of events 16 • 4 years, 4 months
|
|
Blood and lymphatic system disorders
Leukocytes (total WBC) - low
|
25.0%
4/16 • Number of events 5 • 4 years, 4 months
|
|
Blood and lymphatic system disorders
Neutrophils/granulocytes (ANC/AGC) - low
|
25.0%
4/16 • Number of events 12 • 4 years, 4 months
|
|
Blood and lymphatic system disorders
Hemoglobin - low
|
18.8%
3/16 • Number of events 7 • 4 years, 4 months
|
|
Blood and lymphatic system disorders
Bone marrow cellularity
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Injection site reaction/extravasation changes
|
43.8%
7/16 • Number of events 19 • 4 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin - Other
|
18.8%
3/16 • Number of events 3 • 4 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Pruritus/itching
|
12.5%
2/16 • Number of events 2 • 4 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Rash: erythema multiforme
|
12.5%
2/16 • Number of events 3 • 4 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Bruising (in absence of Grade 3 or 4 thrombocytopenia)
|
6.2%
1/16 • Number of events 2 • 4 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Flushing
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Skin and subcutaneous tissue disorders
Hair loss/alopecia (scalp or body)
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
General disorders
Fatigue (asthenia, lethargy, malaise)
|
50.0%
8/16 • Number of events 10 • 4 years, 4 months
|
|
General disorders
Insomnia
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
General disorders
Rigors/chills
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Gastrointestinal disorders
Constipation
|
25.0%
4/16 • Number of events 5 • 4 years, 4 months
|
|
Gastrointestinal disorders
Diarrhea
|
18.8%
3/16 • Number of events 3 • 4 years, 4 months
|
|
Gastrointestinal disorders
Nausea
|
18.8%
3/16 • Number of events 5 • 4 years, 4 months
|
|
Gastrointestinal disorders
Anorexia
|
12.5%
2/16 • Number of events 2 • 4 years, 4 months
|
|
Gastrointestinal disorders
Distension/bloating, abdominal
|
12.5%
2/16 • Number of events 2 • 4 years, 4 months
|
|
Gastrointestinal disorders
Gastrointestinal - Other
|
12.5%
2/16 • Number of events 2 • 4 years, 4 months
|
|
Gastrointestinal disorders
Dry mouth/salivary gland (xerostomia)
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Gastrointestinal disorders
Mucositis/stomatitis (clinical exam) - Anus
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Gastrointestinal disorders
Mucositis/stomatitis (clinical exam) - Oral cavity
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Gastrointestinal disorders
Vomiting
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
General disorders
Pain - Abdomen NOS
|
18.8%
3/16 • Number of events 4 • 4 years, 4 months
|
|
General disorders
Pain - Other
|
18.8%
3/16 • Number of events 5 • 4 years, 4 months
|
|
General disorders
Pain - Bone
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
General disorders
Pain - Head/headache
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
General disorders
Pain - Joint
|
6.2%
1/16 • Number of events 3 • 4 years, 4 months
|
|
General disorders
Pain - Muscle
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
General disorders
Pain - Neck
|
6.2%
1/16 • Number of events 2 • 4 years, 4 months
|
|
General disorders
Pain - Sinus
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Metabolism and nutrition disorders
Glucose, serum-high (hyperglycemia)
|
12.5%
2/16 • Number of events 2 • 4 years, 4 months
|
|
Metabolism and nutrition disorders
Metabolic/Laboratory - Other
|
12.5%
2/16 • Number of events 4 • 4 years, 4 months
|
|
Metabolism and nutrition disorders
Alkaline phosphatase
|
6.2%
1/16 • Number of events 2 • 4 years, 4 months
|
|
Metabolism and nutrition disorders
ALT, SGPT (serum glutamic pyruvic transaminase)
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Metabolism and nutrition disorders
Bilirubin (hyperbilirubinemia)
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Metabolism and nutrition disorders
Potassium, serum-low (hypokalemia)
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Nervous system disorders
Dizziness
|
12.5%
2/16 • Number of events 2 • 4 years, 4 months
|
|
Nervous system disorders
Neuropathy: sensory
|
12.5%
2/16 • Number of events 4 • 4 years, 4 months
|
|
Nervous system disorders
Memory impairment
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Psychiatric disorders
Mood alteration - Anxiety
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.0%
4/16 • Number of events 5 • 4 years, 4 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Blood and lymphatic system disorders
Petechiae/purpura (hemorrhage/bleeding into skin or mucosa)
|
12.5%
2/16 • Number of events 2 • 4 years, 4 months
|
|
Vascular disorders
Hematoma
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Arthritis (non-septic)
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Fracture
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness, generalized or specific area (not due to neuropathy) Whole body/generalized
|
6.2%
1/16 • Number of events 3 • 4 years, 4 months
|
|
Eye disorders
Dry eye syndrome
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Eye disorders
Ocular/Visual - Other
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Renal and urinary disorders
Renal/Genitourinary - Other
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Renal and urinary disorders
Urinary frequency/urgency
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Immune system disorders
Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Blood and lymphatic system disorders
Coagulation - Other
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Blood and lymphatic system disorders
Thrombotic microangiopathy
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Endocrine disorders
Hot flashes/flushes
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
Blood and lymphatic system disorders
Edema: limb
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
|
General disorders
Flu-like syndrome
|
6.2%
1/16 • Number of events 1 • 4 years, 4 months
|
Additional Information
Jeffrey E. Lancet, M.D.
H. Lee Moffitt Cancer Center and Research Institute
Phone: 813-745-6841
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place