Trial Outcomes & Findings for Azacitidine and Pembrolizumab in Pancreatic Cancer (NCT NCT03264404)
NCT ID: NCT03264404
Last Updated: 2025-06-17
Results Overview
PFS is defined as the time from the first day of trial treatment to the first documented disease progression per RECIST 1.1 (At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. (Note: the appearance of one or more new lesions is also considered progression)) or death due to any cause, whichever occurs first.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
36 participants
Primary outcome timeframe
24 months
Results posted on
2025-06-17
Participant Flow
Participant milestones
| Measure |
Pembrolizumab
Patients with advanced pancreatic cancer will receive pembrolizumab with the hypomethylating agent azacitidine.
Pembrolizumab: Pembrolizumab 200 mg IV every 3 weeks until progression
Azacitidine: 50 mg/m2 subcutaneous daily for 5 days every 28 days
|
|---|---|
|
Overall Study
STARTED
|
36
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
34
|
Reasons for withdrawal
| Measure |
Pembrolizumab
Patients with advanced pancreatic cancer will receive pembrolizumab with the hypomethylating agent azacitidine.
Pembrolizumab: Pembrolizumab 200 mg IV every 3 weeks until progression
Azacitidine: 50 mg/m2 subcutaneous daily for 5 days every 28 days
|
|---|---|
|
Overall Study
Death
|
2
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Disease Progression
|
28
|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Off study prior to treatment
|
2
|
Baseline Characteristics
Azacitidine and Pembrolizumab in Pancreatic Cancer
Baseline characteristics by cohort
| Measure |
Pembrolizumab
n=36 Participants
Patients with advanced pancreatic cancer will receive pembrolizumab with the hypomethylating agent azacitidine.
Pembrolizumab: Pembrolizumab 200 mg IV every 3 weeks until progression
Azacitidine: 50 mg/m2 subcutaneous daily for 5 days every 28 days
|
|---|---|
|
Age, Continuous
|
64.3 years
STANDARD_DEVIATION 9.79 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 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
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
28 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
36 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 monthsPFS is defined as the time from the first day of trial treatment to the first documented disease progression per RECIST 1.1 (At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. (Note: the appearance of one or more new lesions is also considered progression)) or death due to any cause, whichever occurs first.
Outcome measures
| Measure |
Pembrolizumab
n=36 Participants
Patients with advanced pancreatic cancer will receive pembrolizumab with the hypomethylating agent azacitidine.
Pembrolizumab: Pembrolizumab 200 mg IV every 3 weeks until progression
Azacitidine: 50 mg/m2 subcutaneous daily for 5 days every 28 days
|
|---|---|
|
Progression-Free Survival (PFS)
|
1.58 months
Standard Deviation 1.02
|
SECONDARY outcome
Timeframe: 24 monthsORR is defined as the proportion of the subjects in the analysis population who have a complete response (CR) or partial response (PR). Responses are based on assessments per RECIST 1.1.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsFor subjects who demonstrate CR or PR, based on assessments per RECIST 1.1, DOR is defined as the time from first documented evidence of CR or PR until disease progression or death due to any cause, whichever occurs first.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 24 monthsDCR is defined as the percentage of subjects who have achieved CR, PR, or stable disease (SD) based on assessments per RECIST 1.1.
Outcome measures
Outcome data not reported
Adverse Events
Pembrolizumab
Serious events: 9 serious events
Other events: 36 other events
Deaths: 26 deaths
Serious adverse events
| Measure |
Pembrolizumab
n=36 participants at risk
Patients with advanced pancreatic cancer will receive pembrolizumab with the hypomethylating agent azacitidine.
Pembrolizumab: Pembrolizumab 200 mg IV every 3 weeks until progression
Azacitidine: 50 mg/m2 subcutaneous daily for 5 days every 28 days
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
5.6%
2/36 • Number of events 2 • 24 months
|
|
General disorders
Gastric hemorrhage
|
5.6%
2/36 • Number of events 2 • 24 months
|
|
Endocrine disorders
Endocrine disorders
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
Nervous system disorders
Nervous system disorders
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Nervous system disorders
Stroke
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Infections and infestations
Sepsis
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Cardiac disorders
Myocarditis
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Hepatobiliary disorders
Hepatobiliary disorders
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Vascular disorders
Vascular disorders
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Gastrointestinal disorders
Gastroparesis
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Hepatobiliary disorders
Hepatic hemorrhage
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Gastrointestinal disorders
Abdominal pain
|
5.6%
2/36 • Number of events 4 • 24 months
|
|
Gastrointestinal disorders
Ascites
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
Blood and lymphatic system disorders
Anemia
|
2.8%
1/36 • Number of events 4 • 24 months
|
|
Vascular disorders
Thromboembolic event
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Hepatobiliary disorders
Portal vein thrombosis
|
2.8%
1/36 • Number of events 1 • 24 months
|
Other adverse events
| Measure |
Pembrolizumab
n=36 participants at risk
Patients with advanced pancreatic cancer will receive pembrolizumab with the hypomethylating agent azacitidine.
Pembrolizumab: Pembrolizumab 200 mg IV every 3 weeks until progression
Azacitidine: 50 mg/m2 subcutaneous daily for 5 days every 28 days
|
|---|---|
|
Gastrointestinal disorders
Vomiting
|
13.9%
5/36 • Number of events 7 • 24 months
|
|
Gastrointestinal disorders
Abdominal Pain
|
25.0%
9/36 • Number of events 13 • 24 months
|
|
Gastrointestinal disorders
Diarrhea
|
36.1%
13/36 • Number of events 21 • 24 months
|
|
Investigations
Alanine aminotransferase increase
|
13.9%
5/36 • Number of events 9 • 24 months
|
|
Investigations
International Normalized Ratio (INR) Increased
|
2.8%
1/36 • Number of events 3 • 24 months
|
|
Investigations
Hyponatremia
|
13.9%
5/36 • Number of events 6 • 24 months
|
|
Investigations
Hypercalcemia
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Investigations
Creatinine Increased
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
Investigations
Hyperkalemia
|
8.3%
3/36 • Number of events 4 • 24 months
|
|
Investigations
Hypoalbuminemia
|
5.6%
2/36 • Number of events 3 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
Investigations
Alkaline phosphatase increased
|
19.4%
7/36 • Number of events 17 • 24 months
|
|
General disorders
Pain
|
8.3%
3/36 • Number of events 3 • 24 months
|
|
General disorders
Chills
|
11.1%
4/36 • Number of events 5 • 24 months
|
|
Investigations
Aspartate aminotransferase increased
|
13.9%
5/36 • Number of events 10 • 24 months
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Investigations
Blood bilirubin increased
|
19.4%
7/36 • Number of events 12 • 24 months
|
|
Gastrointestinal disorders
Bloating
|
19.4%
7/36 • Number of events 11 • 24 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
8.3%
3/36 • Number of events 4 • 24 months
|
|
Hepatobiliary disorders
Hepatobiliary disorder
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
General disorders
Cough
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
General disorders
Headache
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
General disorders
Flu like symptoms
|
5.6%
2/36 • Number of events 2 • 24 months
|
|
General disorders
Fatigue
|
22.2%
8/36 • Number of events 11 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
16.7%
6/36 • Number of events 8 • 24 months
|
|
Gastrointestinal disorders
Constipation
|
33.3%
12/36 • Number of events 13 • 24 months
|
|
Renal and urinary disorders
Urinary tract pain
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.6%
2/36 • Number of events 2 • 24 months
|
|
General disorders
Injection site reaction
|
19.4%
7/36 • Number of events 7 • 24 months
|
|
Gastrointestinal disorders
Nausea
|
16.7%
6/36 • Number of events 7 • 24 months
|
|
Metabolism and nutrition disorders
Anorexia
|
25.0%
9/36 • Number of events 13 • 24 months
|
|
Investigations
Hypokalemia
|
8.3%
3/36 • Number of events 9 • 24 months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorder
|
5.6%
2/36 • Number of events 2 • 24 months
|
|
Blood and lymphatic system disorders
Anemia
|
11.1%
4/36 • Number of events 10 • 24 months
|
|
General disorders
Fever
|
5.6%
2/36 • Number of events 2 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Chest wall pain
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Gastrointestinal disorders
Anal pain
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Reproductive system and breast disorders
Vaginal dryness
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
8.3%
3/36 • Number of events 3 • 24 months
|
|
Investigations
Platelet count decreased
|
5.6%
2/36 • Number of events 4 • 24 months
|
|
Infections and infestations
Infections and infestations
|
16.7%
6/36 • Number of events 8 • 24 months
|
|
Renal and urinary disorders
Acute kidney injury
|
5.6%
2/36 • Number of events 3 • 24 months
|
|
Investigations
Weight loss
|
11.1%
4/36 • Number of events 6 • 24 months
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders
|
8.3%
3/36 • Number of events 3 • 24 months
|
|
Psychiatric disorders
Anxiety
|
5.6%
2/36 • Number of events 3 • 24 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
8.3%
3/36 • Number of events 3 • 24 months
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
General disorders
Sore throat
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.6%
2/36 • Number of events 3 • 24 months
|
|
Endocrine disorders
Hypothyroidism
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Gastrointestinal disorders
Gastrointestinal disorder
|
8.3%
3/36 • Number of events 3 • 24 months
|
|
General disorders
Confusion
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Gastrointestinal disorders
Colitis
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Gastrointestinal disorders
Ascites
|
8.3%
3/36 • Number of events 4 • 24 months
|
|
Ear and labyrinth disorders
Ear and labyrinth disorders
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Eye disorders
Watering eyes
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Vascular disorders
Thromboembolic event
|
13.9%
5/36 • Number of events 8 • 24 months
|
|
Gastrointestinal disorders
Mucositis oral
|
5.6%
2/36 • Number of events 3 • 24 months
|
|
Hepatobiliary disorders
Portal hypertension
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Infections and infestations
Lung infection
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Investigations
Hypernatremia
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Endocrine disorders
Endocrine disorders
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Vascular disorders
Hypotension
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
Infections and infestations
Sepsis
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
Investigations
Neutrophil count decreased
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Investigations
White blood cell count decreased
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
General disorders
Edema, Limbs
|
5.6%
2/36 • Number of events 2 • 24 months
|
|
General disorders
Hot flashes
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.8%
1/36 • Number of events 4 • 24 months
|
|
Nervous system disorders
Stroke
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Infections and infestations
Urinary tract infection
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Psychiatric disorders
Insomnia
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Investigations
Hypocalcemia
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Investigations
Hypomagnesemia
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications
|
2.8%
1/36 • Number of events 2 • 24 months
|
|
Injury, poisoning and procedural complications
Bruising
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
General disorders
Non-cardiac chest pain
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Psychiatric disorders
Hallucinations
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder
|
5.6%
2/36 • Number of events 2 • 24 months
|
|
Injury, poisoning and procedural complications
Fall
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Investigations
Cardiac troponin T increased
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
2.8%
1/36 • Number of events 1 • 24 months
|
|
Investigations
Weight gain
|
2.8%
1/36 • Number of events 2 • 24 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place