Trial Outcomes & Findings for Peptide Vaccinations to Treat Patients With Low-Risk Myeloid Cancers (NCT NCT00488592)
NCT ID: NCT00488592
Last Updated: 2021-07-12
Results Overview
Number of participants diagnosed with myelodysplastic syndrome (MDS), acute myeloid leukemia (AML) or chronic myeloid leukemia (CML) who experienced an inducing or boosting of a cellular immune response following Wilm's Tumor 1 (WT1) and PR1 vaccine. A T-cell immune 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.
COMPLETED
PHASE2
10 participants
16 weeks
2021-07-12
Participant Flow
Participant milestones
| Measure |
PR1/WT1 Vaccine Response in Participants With Low-Risk Myeloid Cancers
Participants were given subcutaneous 6 injections of PR1:169-177 in "Montanide" adjuvant and 6 doses of WT1:126-134 in "Montanide" adjuvant at 2 weekly intervals. GM-CSF (Sargramostim) was co administered with each vaccine dose. Subjects with immunological response to one or both peptide vaccines had the option of receiving a maximum of 6 additional boosters of the WT-1:126-134 and PR1:169-177 peptide vaccines at 3 monthly intervals.
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|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
7
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
PR1/WT1 Vaccine Response in Participants With Low-Risk Myeloid Cancers
Participants were given subcutaneous 6 injections of PR1:169-177 in "Montanide" adjuvant and 6 doses of WT1:126-134 in "Montanide" adjuvant at 2 weekly intervals. GM-CSF (Sargramostim) was co administered with each vaccine dose. Subjects with immunological response to one or both peptide vaccines had the option of receiving a maximum of 6 additional boosters of the WT-1:126-134 and PR1:169-177 peptide vaccines at 3 monthly intervals.
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|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Disease progression
|
1
|
|
Overall Study
Enrolled but not treated
|
1
|
Baseline Characteristics
Peptide Vaccinations to Treat Patients With Low-Risk Myeloid Cancers
Baseline characteristics by cohort
| Measure |
PR1/WT1 Vaccine Response in Participants With Low-Risk Myeloid Cancers
n=10 Participants
Participants were given subcutaneous 6 injections of PR1:169-177 in "Montanide" adjuvant and 6 doses of WT1:126-134 in "Montanide" adjuvant at 2 weekly intervals. GM-CSF (Sargramostim) was co administered with each vaccine dose. Subjects with immunological response to one or both peptide vaccines had the option of receiving a maximum of 6 additional boosters of the WT-1:126-134 and PR1:169-177 peptide vaccines at 3 monthly intervals.
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|---|---|
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Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=5 Participants
|
|
Age, Continuous
|
58.9 years
STANDARD_DEVIATION 13.7 • n=5 Participants
|
|
Sex: Female, Male
Female
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7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
United States
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10 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 16 weeksNumber of participants diagnosed with myelodysplastic syndrome (MDS), acute myeloid leukemia (AML) or chronic myeloid leukemia (CML) who experienced an inducing or boosting of a cellular immune response following Wilm's Tumor 1 (WT1) and PR1 vaccine. A T-cell immune 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.
Outcome measures
| Measure |
PR1/WT1 Vaccine Response in Participants With Low-Risk Myeloid Cancers
n=7 Participants
Participants were given subcutaneous 6 injections of PR1:169-177 in "Montanide" adjuvant and 6 doses of WT1:126-134 in "Montanide" adjuvant at 2 weekly intervals. GM-CSF (Sargramostim) was co administered with each vaccine dose. Subjects with immunological response to one or both peptide vaccines had the option of receiving a maximum of 6 additional boosters of the WT-1:126-134 and PR1:169-177 peptide vaccines at 3 monthly intervals.
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|---|---|
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Number of Participants Who Experienced Inducing or Boosting of a Cellular Immune Response
Cellular immune response
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5 participants
|
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Number of Participants Who Experienced Inducing or Boosting of a Cellular Immune Response
No cellular immune response
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2 participants
|
SECONDARY outcome
Timeframe: 16 weeksNumber of participants diagnosed with myelodysplastic syndrome (MDS), acute myeloid leukemia (AML) or chronic myeloid leukemia (CML) who experienced a hematological response following Wilm's Tumor (WT1) and PR1 peptide vaccine. Hematological response is defined by reduction in marrow blast cells, changes in blood counts.
Outcome measures
| Measure |
PR1/WT1 Vaccine Response in Participants With Low-Risk Myeloid Cancers
n=7 Participants
Participants were given subcutaneous 6 injections of PR1:169-177 in "Montanide" adjuvant and 6 doses of WT1:126-134 in "Montanide" adjuvant at 2 weekly intervals. GM-CSF (Sargramostim) was co administered with each vaccine dose. Subjects with immunological response to one or both peptide vaccines had the option of receiving a maximum of 6 additional boosters of the WT-1:126-134 and PR1:169-177 peptide vaccines at 3 monthly intervals.
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|---|---|
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Number of Participants Who Experienced a Hematological Response
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0 participants
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Adverse Events
PR1/WT1 Vaccine Response in Participants With Leukemias
Serious adverse events
| Measure |
PR1/WT1 Vaccine Response in Participants With Leukemias
n=10 participants at risk
Participants were given subcutaneous 6 injections of PR1:169-177 in "Montanide" adjuvant and 6 doses of WT1:126-134 in "Montanide" adjuvant at 2 weekly intervals. GM-CSF (Sargramostim) was co administered with each vaccine dose. Subjects with immunological response to one or both peptide vaccines had the option of receiving a maximum of 6 additional boosters of the WT-1:126-134 and PR1:169-177 peptide vaccines at 3 monthly intervals.
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|---|---|
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Gastrointestinal disorders
Abdominal pain
|
10.0%
1/10 • Number of events 1 • 16 weeks
|
|
Infections and infestations
Upper Respiratory Infection
|
10.0%
1/10 • Number of events 1 • 16 weeks
|
Other adverse events
| Measure |
PR1/WT1 Vaccine Response in Participants With Leukemias
n=10 participants at risk
Participants were given subcutaneous 6 injections of PR1:169-177 in "Montanide" adjuvant and 6 doses of WT1:126-134 in "Montanide" adjuvant at 2 weekly intervals. GM-CSF (Sargramostim) was co administered with each vaccine dose. Subjects with immunological response to one or both peptide vaccines had the option of receiving a maximum of 6 additional boosters of the WT-1:126-134 and PR1:169-177 peptide vaccines at 3 monthly intervals.
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|---|---|
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Blood and lymphatic system disorders
decreased hemoglobin
|
10.0%
1/10 • Number of events 1 • 16 weeks
|
|
Immune system disorders
back pressure
|
20.0%
2/10 • Number of events 2 • 16 weeks
|
|
General disorders
fatigue
|
10.0%
1/10 • Number of events 1 • 16 weeks
|
|
General disorders
lightheadness
|
10.0%
1/10 • Number of events 1 • 16 weeks
|
|
General disorders
malaise
|
10.0%
1/10 • Number of events 1 • 16 weeks
|
|
General disorders
rigors
|
10.0%
1/10 • Number of events 1 • 16 weeks
|
|
Skin and subcutaneous tissue disorders
skin rash
|
40.0%
4/10 • Number of events 4 • 16 weeks
|
|
Gastrointestinal disorders
indigestion
|
10.0%
1/10 • Number of events 1 • 16 weeks
|
|
Gastrointestinal disorders
loose stools
|
20.0%
2/10 • Number of events 2 • 16 weeks
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
40.0%
4/10 • Number of events 4 • 16 weeks
|
|
Musculoskeletal and connective tissue disorders
myalgia
|
40.0%
4/10 • Number of events 4 • 16 weeks
|
|
Nervous system disorders
headache
|
20.0%
2/10 • Number of events 2 • 16 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place