Trial Outcomes & Findings for Study of Pembrolizumab With or Without Defactinib Following Chemotherapy as a Neoadjuvant and Adjuvant Treatment for Resectable Pancreatic Ductal Adenocarcinoma (NCT NCT03727880)

Last Updated: 2026-02-04

Results Overview

Percent of subjects with a pathologic complete response (pCR) per the tumor regression grade scores established by the College of American Pathologist: Grade 0= complete response (no viable cancer cells), Grade 1= near complete response (single cells or rare small groups of cancer cells), Grade 2= partial response (residual tumor with evidence of regression), or Grade 3= no response (extensive residual tumor with no evidence of regression).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

28 participants

Primary outcome timeframe

4 years

Results posted on

2026-02-04

Participant Flow

Participant milestones

Participant milestones
Measure	Arm A - Pembrolizumab and Defactinib Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart. Defactinib: Following 2 cycles of standard of care neoadjuvant chemotherapy, subjects will receive 400 mg defactinib twice a day up until 2 days preceding their surgery (approximately 6 weeks) during the immunotherapy cycles with pembrolizumab. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 400mg defactinib twice a day for 24 weeks.	Arm B - Pembrolizumab Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart.
Overall Study STARTED	14	14
Overall Study COMPLETED	13	10
Overall Study NOT COMPLETED	1	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Pembrolizumab With or Without Defactinib Following Chemotherapy as a Neoadjuvant and Adjuvant Treatment for Resectable Pancreatic Ductal Adenocarcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm A - Pembrolizumab and Defactinib n=13 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart. Defactinib: Following 2 cycles of standard of care neoadjuvant chemotherapy, subjects will receive 400 mg defactinib twice a day up until 2 days preceding their surgery (approximately 6 weeks) during the immunotherapy cycles with pembrolizumab. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 400mg defactinib twice a day for 24 weeks.	Arm B - Pembrolizumab n=10 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart.	Total n=23 Participants Total of all reporting groups
Region of Enrollment United States	13 participants n=41 Participants	10 participants n=1581 Participants	23 participants n=4626 Participants
Age, Continuous	71.54 Years STANDARD_DEVIATION 11.39 • n=41 Participants	73.5 Years STANDARD_DEVIATION 6.62 • n=1581 Participants	72.4 Years STANDARD_DEVIATION 9.01 • n=4626 Participants
Sex: Female, Male Female	6 Participants n=41 Participants	3 Participants n=1581 Participants	9 Participants n=4626 Participants
Sex: Female, Male Male	7 Participants n=41 Participants	7 Participants n=1581 Participants	14 Participants n=4626 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants
Race (NIH/OMB) Asian	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants
Race (NIH/OMB) Black or African American	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants
Race (NIH/OMB) White	10 Participants n=41 Participants	10 Participants n=1581 Participants	20 Participants n=4626 Participants
Race (NIH/OMB) More than one race	0 Participants n=41 Participants	0 Participants n=1581 Participants	0 Participants n=4626 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=41 Participants	0 Participants n=1581 Participants	3 Participants n=4626 Participants

PRIMARY outcome

Timeframe: 4 years

Population: Participants that reached pCR

Outcome measures

Outcome measures
Measure	Arm A - Pembrolizumab and Defactinib n=10 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart. Defactinib: Following 2 cycles of standard of care neoadjuvant chemotherapy, subjects will receive 400 mg defactinib twice a day up until 2 days preceding their surgery (approximately 6 weeks) during the immunotherapy cycles with pembrolizumab. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 400mg defactinib twice a day for 24 weeks.	Arm B - Pembrolizumab n=8 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart.
Pathologic Complete Response (pCR) Rate	0 Percentage of participants	0 Percentage of participants

SECONDARY outcome

Timeframe: 4 years

Population: Participants evaluable were those that were randomized to intervention and completed the study

Number of months until death

Outcome measures

Outcome measures
Measure	Arm A - Pembrolizumab and Defactinib n=13 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart. Defactinib: Following 2 cycles of standard of care neoadjuvant chemotherapy, subjects will receive 400 mg defactinib twice a day up until 2 days preceding their surgery (approximately 6 weeks) during the immunotherapy cycles with pembrolizumab. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 400mg defactinib twice a day for 24 weeks.	Arm B - Pembrolizumab n=10 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart.
Overall Survival (OS)	25.01 months Standard Deviation 14.48	27.39 months Standard Deviation 19.54

SECONDARY outcome

Timeframe: 4 years

Population: For Arm A overall study was completed by 13 pt, DFS evalauted for 11. The remaining 2 participants were not evaluable for DFS because surgery was not performed and therefore they never achieved a disease-free baseline For Arm B overall study was completed by 10 pt, DFS evalauted for 8. The 2 participants whose surgeries were attempted but aborted were not evaluable for DFS because they never achieved a disease-free baseline."

Number of months until disease recurrence

Outcome measures

Outcome measures
Measure	Arm A - Pembrolizumab and Defactinib n=11 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart. Defactinib: Following 2 cycles of standard of care neoadjuvant chemotherapy, subjects will receive 400 mg defactinib twice a day up until 2 days preceding their surgery (approximately 6 weeks) during the immunotherapy cycles with pembrolizumab. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 400mg defactinib twice a day for 24 weeks.	Arm B - Pembrolizumab n=8 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart.
Disease Free Survival (DFS)	16.59 months Standard Deviation 14.33	15.99 months Standard Deviation 15.20

SECONDARY outcome

Timeframe: 4 years

Population: Includes all participants that both completed and did not complete the study

Number of participants experiencing drug-related adverse events as defined by CTCAE v5.0

Outcome measures

Outcome measures
Measure	Arm A - Pembrolizumab and Defactinib n=14 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart. Defactinib: Following 2 cycles of standard of care neoadjuvant chemotherapy, subjects will receive 400 mg defactinib twice a day up until 2 days preceding their surgery (approximately 6 weeks) during the immunotherapy cycles with pembrolizumab. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 400mg defactinib twice a day for 24 weeks.	Arm B - Pembrolizumab n=14 Participants Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart.
Number of Participants Experiencing Study Drug-related Toxicities	6 participants	3 participants

Adverse Events

Arm A - Pembrolizumab and Defactinib

Serious events: 2 serious events

Other events: 4 other events

Deaths: 10 deaths

Arm B - Pembrolizumab

Serious events: 3 serious events

Other events: 3 other events

Deaths: 6 deaths

Serious adverse events

Serious adverse events
Measure	Arm A - Pembrolizumab and Defactinib n=14 participants at risk Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart. Defactinib: Following 2 cycles of standard of care neoadjuvant chemotherapy, subjects will receive 400 mg defactinib twice a day up until 2 days preceding their surgery (approximately 6 weeks) during the immunotherapy cycles with pembrolizumab. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 400mg defactinib twice a day for 24 weeks.	Arm B - Pembrolizumab n=14 participants at risk Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart.
General disorders Non-pancreatic cancer	14.3% 2/14 • Number of events 2 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0	21.4% 3/14 • Number of events 3 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0

Other adverse events

Other adverse events
Measure	Arm A - Pembrolizumab and Defactinib n=14 participants at risk Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart. Defactinib: Following 2 cycles of standard of care neoadjuvant chemotherapy, subjects will receive 400 mg defactinib twice a day up until 2 days preceding their surgery (approximately 6 weeks) during the immunotherapy cycles with pembrolizumab. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 400mg defactinib twice a day for 24 weeks.	Arm B - Pembrolizumab n=14 participants at risk Pembrolizumab: Following standard of care neoadjuvant chemotherapy, subjects will receive two doses of pembrolizumab (200mg) IV 3 weeks apart prior to surgery. After surgery, subjects will receive adjuvant standard of care chemotherapy. Following adjuvant chemotherapy, subjects will receive 8 doses of pembrolizumab (200mg) IV 3 weeks apart.
Blood and lymphatic system disorders Increased bilirubin	21.4% 3/14 • Number of events 9 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0	0.00% 0/14 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0
Hepatobiliary disorders Elevated aspartate aminotransferase (AST)	7.1% 1/14 • Number of events 7 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0	0.00% 0/14 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0
Hepatobiliary disorders Elevated Alanine transaminase (ALT)	7.1% 1/14 • Number of events 8 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0	0.00% 0/14 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0
Skin and subcutaneous tissue disorders Acnieform rash	7.1% 1/14 • Number of events 1 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0	0.00% 0/14 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0
Musculoskeletal and connective tissue disorders Myasthenia gravis	0.00% 0/14 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0	7.1% 1/14 • Number of events 3 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/14 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0	7.1% 1/14 • Number of events 1 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0
Gastrointestinal disorders Colitis	0.00% 0/14 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0	7.1% 1/14 • Number of events 8 • Adverse events were collected from baseline to 4 years Adverse events were assessed according to CTCAEv5.0

Additional Information

Arsen Osipov, MD

Cedars Sinai

Phone: 310-423-6313

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place