Trial Outcomes & Findings for Augmentation of the Graft vs. Leukemia Effect Via Checkpoint Blockade With Pembrolizumab (NCT NCT03286114)
NCT ID: NCT03286114
Last Updated: 2025-05-11
Results Overview
This study will assess if the study drug is promising for further study. The study drug will be considered promising if at least 4 patients receive a clinical benefit or if any complete response is seen. Clinical benefit is defined as either stable disease, partial remission or complete remission to treatment. Complete remission (CR) will be defined as achieving a morphologic leukemia free state by achieving all of the following criteria: bone marrow myeloblasts \< 5% by morphologic assessment; AND absence of circulating blasts with phenotypic or morphologic features of leukemia (e.g. Auer rods) AND no evidence of extramedulary disease. Partial remission (PR) will be defined as a ≥ 50% reduction in bone marrow blast percentage to 5-25% or marrow blasts \< 5% with persistent Auer rods, flow cytometric or cytogenetic disease. SD will be defined as ≤ 5% increase in blasts or decreased blast percentage in the bone marrow that does not meet the criteria for PR.
TERMINATED
PHASE1
16 participants
Day 77
2025-05-11
Participant Flow
Participant milestones
| Measure |
Pembrolizumab
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
7
|
|
Overall Study
NOT COMPLETED
|
9
|
Reasons for withdrawal
| Measure |
Pembrolizumab
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
Overall Study
Adverse Event
|
6
|
|
Overall Study
Death
|
3
|
Baseline Characteristics
Augmentation of the Graft vs. Leukemia Effect Via Checkpoint Blockade With Pembrolizumab
Baseline characteristics by cohort
| Measure |
Pembrolizumab
n=16 Participants
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
12 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
16 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 77This study will assess if the study drug is promising for further study. The study drug will be considered promising if at least 4 patients receive a clinical benefit or if any complete response is seen. Clinical benefit is defined as either stable disease, partial remission or complete remission to treatment. Complete remission (CR) will be defined as achieving a morphologic leukemia free state by achieving all of the following criteria: bone marrow myeloblasts \< 5% by morphologic assessment; AND absence of circulating blasts with phenotypic or morphologic features of leukemia (e.g. Auer rods) AND no evidence of extramedulary disease. Partial remission (PR) will be defined as a ≥ 50% reduction in bone marrow blast percentage to 5-25% or marrow blasts \< 5% with persistent Auer rods, flow cytometric or cytogenetic disease. SD will be defined as ≤ 5% increase in blasts or decreased blast percentage in the bone marrow that does not meet the criteria for PR.
Outcome measures
| Measure |
Pembrolizumab
n=16 Participants
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
The Number of Patients That Demonstrate Clinical Benefit From Treatment
|
6 Participants
|
PRIMARY outcome
Timeframe: Day 77This study will assess the number of patients that respond to treatment by overall response rate (ORR). ORR is defined as the number of patients will complete remission and partial remission. Complete remission (CR) will be defined as achieving a morphologic leukemia free state by achieving all of the following criteria: bone marrow myeloblasts \< 5% by morphologic assessment; AND absence of circulating blasts with phenotypic or morphologic features of leukemia (e.g. Auer rods) AND no evidence of extramedulary disease. Partial remission (PR) will be defined as a ≥ 50% reduction in bone marrow blast percentage to 5-25% or marrow blasts \< 5% with persistent Auer rods, flow cytometric or cytogenetic disease.
Outcome measures
| Measure |
Pembrolizumab
n=16 Participants
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
The Number of Patients That Respond to Treatment
|
3 Participants
|
PRIMARY outcome
Timeframe: 30 Days Post TreatmentThe number of patients that experience Graft Versus Host Disease (GvHD) or other significant immune mediated toxicities
Outcome measures
| Measure |
Pembrolizumab
n=16 Participants
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
Graft Versus Host Disease (GvHD) or Other Significant Immune Mediated Toxicities
GVHD
|
9 Participants
|
|
Graft Versus Host Disease (GvHD) or Other Significant Immune Mediated Toxicities
mmune-related SAEs
|
2 Participants
|
SECONDARY outcome
Timeframe: 1 YearThe number of patients alive at 1 year
Outcome measures
| Measure |
Pembrolizumab
n=16 Participants
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
Overall Survival
|
37.5 percentage of participants
Interval 15.4 to 59.8
|
SECONDARY outcome
Timeframe: 1 YearThe number of patients alive at 1 year without disease
Outcome measures
| Measure |
Pembrolizumab
n=16 Participants
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
Event- Free Survival
|
31.3 percentage of participants
Interval 11.1 to 53.7
|
Adverse Events
Pembrolizumab
Serious adverse events
| Measure |
Pembrolizumab
n=16 participants at risk
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
Renal and urinary disorders
Acute kidney injury
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Investigations
Alanine aminotransferase increased
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Investigations
Aspartate aminotransferase increased
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Investigations
Blood bilirubin increased
|
12.5%
2/16 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Metabolism and nutrition disorders
Dehydration
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
General disorders
Fatigue
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
43.8%
7/16 • Number of events 7 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
General disorders
Fever
|
12.5%
2/16 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Injury, poisoning and procedural complications
Fracture
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Investigations
Hyponatremia
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Infections and infestations
Lung infection
|
12.5%
2/16 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
General disorders
Mucositis oral
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
General disorders
Pain
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Cardiac disorders
Pericarditis
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Investigations
Platelet count decreased
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
18.8%
3/16 • Number of events 3 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Infections and infestations
Sepsis
|
12.5%
2/16 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Cardiac disorders
Syncope
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Gastrointestinal disorders
Upper gastrointestinal hemorrhage
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Immune system disorders
Immune system disorders - Other, specify
|
18.8%
3/16 • Number of events 3 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Infections and infestations
Infections and infestations - Other, specify
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
Other adverse events
| Measure |
Pembrolizumab
n=16 participants at risk
Pembrolizumab: 200mg IV every 21 days
|
|---|---|
|
Investigations
Alanine aminotransferase increased
|
12.5%
2/16 • Number of events 3 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Investigations
Aspartate aminotransferase increased
|
12.5%
2/16 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Investigations
Blood bilirubin increased
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Metabolism and nutrition disorders
Dehydration
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Investigations
Hyponatremia
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
6.2%
1/16 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
|
Immune system disorders
Immune system disorders - Other, specify
|
12.5%
2/16 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment. An average of 3 months.
|
Additional Information
University of Michigan Rogel Cancer Center ClinicalTrials.gov Admin
University of Michigan Rogel Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place