Trial Outcomes & Findings for Azacitidine and Pembrolizumab in Treating Patients With Myelodysplastic Syndrome (NCT NCT03094637)
NCT ID: NCT03094637
Last Updated: 2023-03-22
Results Overview
Will estimate the ORR for the experimental treatments, along with the 95% credible intervals. The association between ORR and patient's clinical characteristics will be examined by Wilcoxon's rank sum test or Fisher's exact test, as appropriate.
Recruitment status
UNKNOWN
Study phase
PHASE2
Target enrollment
40 participants
Primary outcome timeframe
Up to 2 years 4 months
Results posted on
2023-03-22
Participant Flow
Recruitment Period: November 2017 to March 2020
Participant milestones
| Measure |
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) failureHMA Failure
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
|---|---|---|
|
Overall Study
STARTED
|
17
|
20
|
|
Overall Study
COMPLETED
|
17
|
20
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Azacitidine and Pembrolizumab in Treating Patients With Myelodysplastic Syndrome
Baseline characteristics by cohort
| Measure |
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
n=17 Participants
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) Failure
n=20 Participants
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
Total
n=37 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
13 Participants
n=99 Participants
|
20 Participants
n=107 Participants
|
33 Participants
n=206 Participants
|
|
Age, Continuous
|
72 years
n=99 Participants
|
75 years
n=107 Participants
|
74 years
n=206 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=99 Participants
|
15 Participants
n=107 Participants
|
25 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
16 Participants
n=99 Participants
|
19 Participants
n=107 Participants
|
35 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
17 participants
n=99 Participants
|
20 participants
n=107 Participants
|
37 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Up to 2 years 4 monthsWill estimate the ORR for the experimental treatments, along with the 95% credible intervals. The association between ORR and patient's clinical characteristics will be examined by Wilcoxon's rank sum test or Fisher's exact test, as appropriate.
Outcome measures
| Measure |
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
n=17 Participants
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) Failure
n=20 Participants
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
|---|---|---|
|
Overall Response Rate (ORR) Defined as Complete Response + Partial Response + Hematological Improvement
|
13 Participants
|
5 Participants
|
PRIMARY outcome
Timeframe: Up to 4 yearsWill be estimated using the method of Kaplan and Meier. Comparisons of time-to-event endpoints by important subgroups will be made using the log-rank tests.
Outcome measures
| Measure |
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
n=17 Participants
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) Failure
n=20 Participants
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
|---|---|---|
|
Event Free Survival
|
9.24 Months
Interval 3.98 to 31.23
|
5.39 Months
Interval 1.48 to 9.07
|
PRIMARY outcome
Timeframe: Up to 4 yearsWill be estimated using the method of Kaplan and Meier. Comparisons of time-to-event endpoints by important subgroups will be made using the log-rank tests. Overall Survival will be presented by median survival, which is the time point at which the cumulative survival drops below 50%. If there is no median survival (not reached), it means the cumulative survival was more than 50%.
Outcome measures
| Measure |
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
n=17 Participants
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) Failure
n=20 Participants
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
|---|---|---|
|
Overall Survival
|
NA Months
Interval 1.0 to 32.0
Median survival for frontline patients cohort was not reached as the cumulative survival was greater than 50%.
|
5.79 Months
Interval 4.47 to 18.61
|
Adverse Events
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
Serious events: 9 serious events
Other events: 15 other events
Deaths: 1 deaths
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) Failure
Serious events: 13 serious events
Other events: 14 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
n=17 participants at risk
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) Failure
n=20 participants at risk
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
|---|---|---|
|
Investigations
Hyperbilirubinemia
|
5.9%
1/17 • Number of events 2 • Up to 4 years
|
0.00%
0/20 • Up to 4 years
|
|
Gastrointestinal disorders
Colitis
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
0.00%
0/20 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
General disorders
Fatigue
|
0.00%
0/17 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Blood and lymphatic system disorders
Neutropenic Fever
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
10.0%
2/20 • Number of events 4 • Up to 4 years
|
|
General disorders
fever
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
0.00%
0/20 • Up to 4 years
|
|
Blood and lymphatic system disorders
anemia
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Hepatobiliary disorders
Hepatobiliary/Pancreas
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
0.00%
0/20 • Up to 4 years
|
|
Infections and infestations
Infection
|
23.5%
4/17 • Number of events 6 • Up to 4 years
|
55.0%
11/20 • Number of events 12 • Up to 4 years
|
|
Infections and infestations
Infection G 3 or 4 neutrophils
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Injury, poisoning and procedural complications
Intra-operative Injury
|
0.00%
0/17 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Blood and lymphatic system disorders
Leukocytes
|
0.00%
0/17 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Blood and lymphatic system disorders
Neutrophils
|
0.00%
0/17 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
General disorders
Pain
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Pruritis/itching
|
0.00%
0/17 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Renal and urinary disorders
Renal/Genitourinary
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
0.00%
0/20 • Up to 4 years
|
|
Nervous system disorders
Syncope
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
0.00%
0/20 • Up to 4 years
|
Other adverse events
| Measure |
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
n=17 participants at risk
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) Failure
n=20 participants at risk
Patients receive azacitidine IV over 10-40 minutes or SC on days 1-7, and pembrolizumab IV over 30 minutes every 3 weeks. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Azacitidine: Given IV
Pembrolizumab: Given IV
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
17.6%
3/17 • Number of events 4 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Investigations
Alanine Aminotransferase
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
20.0%
4/20 • Number of events 4 • Up to 4 years
|
|
Gastrointestinal disorders
Anorexia
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Investigations
Asparatate Aminotransferase
|
0.00%
0/17 • Up to 4 years
|
20.0%
4/20 • Number of events 4 • Up to 4 years
|
|
Gastrointestinal disorders
Constipation
|
11.8%
2/17 • Number of events 2 • Up to 4 years
|
30.0%
6/20 • Number of events 6 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Dermatology Skin/Other
|
11.8%
2/17 • Number of events 2 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Gastrointestinal disorders
Diarrhea
|
11.8%
2/17 • Number of events 2 • Up to 4 years
|
0.00%
0/20 • Up to 4 years
|
|
General disorders
Fever
|
0.00%
0/17 • Up to 4 years
|
10.0%
2/20 • Number of events 2 • Up to 4 years
|
|
Blood and lymphatic system disorders
anemia
|
11.8%
2/17 • Number of events 2 • Up to 4 years
|
0.00%
0/20 • Up to 4 years
|
|
Infections and infestations
Infection
|
17.6%
3/17 • Number of events 3 • Up to 4 years
|
5.0%
1/20 • Number of events 2 • Up to 4 years
|
|
General disorders
Injection Site Reaction
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
10.0%
2/20 • Number of events 2 • Up to 4 years
|
|
Investigations
Neutropenia
|
17.6%
3/17 • Number of events 3 • Up to 4 years
|
35.0%
7/20 • Number of events 7 • Up to 4 years
|
|
General disorders
Pain
|
35.3%
6/17 • Number of events 6 • Up to 4 years
|
15.0%
3/20 • Number of events 3 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Pruritis/itching
|
5.9%
1/17 • Number of events 1 • Up to 4 years
|
5.0%
1/20 • Number of events 1 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
17.6%
3/17 • Number of events 4 • Up to 4 years
|
10.0%
2/20 • Number of events 2 • Up to 4 years
|
Additional Information
Guillermo Garcia-Manero MD/Professor
The University of Texas MD Anderson Cancer Center
Phone: 713-745-3428
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place