Trial Outcomes & Findings for Pembrolizumab in Treating Participants With Metastatic, Recurrent or Locally Advanced Cancer and Genomic Instability (NCT NCT03428802)

Last Updated: 2026-04-03

Results Overview

Overall Response Rate (ORR) defined as the proportion of participants with Complete Response (CR) or Partial Response (PR) as their best overall response. Tumor response was assessed per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 using CT or MRI imaging. Complete Response (CR): Disappearance of all target lesions. Partial Response (PR): At least a 30% decrease in the sum of diameters of target lesions. ORR = (CR + PR) / number of participants analyzed.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

Up to 2.5 years

Results posted on

2026-04-03

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Pembrolizumab) Participants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Participants with disease progression may continue pembrolizumab for up to 1 year. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV
Overall Study STARTED	21
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pembrolizumab in Treating Participants With Metastatic, Recurrent or Locally Advanced Cancer and Genomic Instability

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm 1 Treatment (Pembrolizumab) - POLE Mutations n=1 Participants Participants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Participants with disease progression may continue pembrolizumab for up to 1 year. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV	Arm 2 Treatment (Pembrolizumab) - BRCA Mutations n=20 Participants Participants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Participants with disease progression may continue pembrolizumab for up to 1 year. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV	Total n=21 Participants Total of all reporting groups
Age, Customized 40 -49	1 Participants n=5 Participants	5 Participants n=5 Participants	6 Participants n=10 Participants
Age, Customized 50 -59	0 Participants n=5 Participants	6 Participants n=5 Participants	6 Participants n=10 Participants
Age, Customized 60 -69	0 Participants n=5 Participants	1 Participants n=5 Participants	1 Participants n=10 Participants
Age, Customized 70 -79	0 Participants n=5 Participants	5 Participants n=5 Participants	5 Participants n=10 Participants
Age, Customized 80 -89	0 Participants n=5 Participants	3 Participants n=5 Participants	3 Participants n=10 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	16 Participants n=5 Participants	17 Participants n=10 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	4 Participants n=5 Participants	4 Participants n=10 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	3 Participants n=5 Participants	4 Participants n=10 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	1 Participants n=5 Participants	1 Participants n=10 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=5 Participants	1 Participants n=10 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	13 Participants n=5 Participants	13 Participants n=10 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	2 Participants n=5 Participants	2 Participants n=10 Participants
Region of Enrollment United States	1 participants n=5 Participants	20 participants n=5 Participants	21 participants n=10 Participants

PRIMARY outcome

Timeframe: Up to 2.5 years

Outcome measures

Outcome measures
Measure	Laboratory Biomarker Analysis n=20 Participants Correlative studies Biological/Vaccine: Pembrolizumab Given IV Other Names: Keytruda Lambrolizumab MK-3475 PEMBROLIZUMAB SCH 900475
Overall Response Rate (ORR) Per RECIST v1.1	20 Participants

Adverse Events

Arm 1 Treatment (Pembrolizumab) - POLE Mutations

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Arm 2 Treatment (Pembrolizumab) - BRCA Mutations

Serious events: 12 serious events

Other events: 20 other events

Deaths: 7 deaths

Serious adverse events

Serious adverse events
Measure	Arm 1 Treatment (Pembrolizumab) - POLE Mutations n=1 participants at risk Participants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Participants with disease progression may continue pembrolizumab for up to 1 year. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV	Arm 2 Treatment (Pembrolizumab) - BRCA Mutations n=20 participants at risk Participants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Participants with disease progression may continue pembrolizumab for up to 1 year. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV
Musculoskeletal and connective tissue disorders Muscle weakness	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Metabolism and nutrition disorders Dehydration	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Blood and lymphatic system disorders Anemia	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Blood and lymphatic system disorders Platelet count decreased	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
General disorders Abdominal pain	0.00% 0/1 • From baseline up to two and a half years.	25.0% 5/20 • Number of events 5 • From baseline up to two and a half years.
Gastrointestinal disorders Diarrhea	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Gastrointestinal disorders Hepatobiliary disorders	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Infections and infestations Sepsis	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Gastrointestinal disorders Small intestinal obstruction	0.00% 0/1 • From baseline up to two and a half years.	15.0% 3/20 • Number of events 3 • From baseline up to two and a half years.
Renal and urinary disorders Urinary tract infection	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Renal and urinary disorders Acute kidney injury	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Metabolism and nutrition disorders Hypokalemia	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Metabolism and nutrition disorders Ascites	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.

Other adverse events

Additional Information

Eugenia Girda, MD

Cancer Institute of New Jersey Rutgers

Phone: 732-235-2465

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Arm 1 Treatment (Pembrolizumab) - POLE Mutations n=1 participants at risk Participants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Participants with disease progression may continue pembrolizumab for up to 1 year. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV	Arm 2 Treatment (Pembrolizumab) - BRCA Mutations n=20 participants at risk Participants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Participants with disease progression may continue pembrolizumab for up to 1 year. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV
Cardiac disorders Sinus tachycardia	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Endocrine disorders Alanine aminotransferase increased	100.0% 1/1 • Number of events 1 • From baseline up to two and a half years.	0.00% 0/20 • From baseline up to two and a half years.
Metabolism and nutrition disorders Alkaline phosphatase increased	100.0% 1/1 • Number of events 1 • From baseline up to two and a half years.	0.00% 0/20 • From baseline up to two and a half years.
Metabolism and nutrition disorders Aspartate aminotransferase increased	100.0% 1/1 • Number of events 1 • From baseline up to two and a half years.	0.00% 0/20 • From baseline up to two and a half years.
Immune system disorders Lymphocyte count decreased	100.0% 1/1 • Number of events 1 • From baseline up to two and a half years.	0.00% 0/20 • From baseline up to two and a half years.
Endocrine disorders Hyperglycemia	100.0% 1/1 • Number of events 1 • From baseline up to two and a half years.	0.00% 0/20 • From baseline up to two and a half years.
Skin and subcutaneous tissue disorders Rash maculo-papular	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 4 • From baseline up to two and a half years.
Blood and lymphatic system disorders Anemia	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Cardiac disorders Palpitations	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Endocrine disorders Endocrine disorders	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Endocrine disorders Hyperthyroidism	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Gastrointestinal disorders Abdominal pain	0.00% 0/1 • From baseline up to two and a half years.	25.0% 5/20 • Number of events 5 • From baseline up to two and a half years.
Gastrointestinal disorders Constipation	0.00% 0/1 • From baseline up to two and a half years.	25.0% 5/20 • Number of events 5 • From baseline up to two and a half years.
Gastrointestinal disorders Mucositis oral	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Gastrointestinal disorders Nausea	0.00% 0/1 • From baseline up to two and a half years.	15.0% 3/20 • Number of events 3 • From baseline up to two and a half years.
Gastrointestinal disorders Vomiting	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
General disorders Edema limbs	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 2 • From baseline up to two and a half years.
General disorders Edema trunk	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
General disorders Fatigue	0.00% 0/1 • From baseline up to two and a half years.	55.0% 11/20 • Number of events 11 • From baseline up to two and a half years.
General disorders General disorders	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Cardiac disorders Non-cardiac chest pain	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
General disorders Pain	0.00% 0/1 • From baseline up to two and a half years.	25.0% 5/20 • Number of events 5 • From baseline up to two and a half years.
Hepatobiliary disorders Hepatobiliary disorders	0.00% 0/1 • From baseline up to two and a half years.	15.0% 3/20 • Number of events 3 • From baseline up to two and a half years.
Investigations Alanine aminotransferase increased	0.00% 0/1 • From baseline up to two and a half years.	15.0% 3/20 • Number of events 3 • From baseline up to two and a half years.
Investigations Alkaline phosphatase increased	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 7 • From baseline up to two and a half years.
Investigations Aspartate aminotransferase increased	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 5 • From baseline up to two and a half years.
Investigations Weight loss	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 2 • From baseline up to two and a half years.
Metabolism and nutrition disorders Anorexia	0.00% 0/1 • From baseline up to two and a half years.	15.0% 3/20 • Number of events 3 • From baseline up to two and a half years.
Metabolism and nutrition disorders Hyperkalemia	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 1 • From baseline up to two and a half years.
Metabolism and nutrition disorders Hyperuricemia	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 2 • From baseline up to two and a half years.
Metabolism and nutrition disorders Hyponatremia	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 2 • From baseline up to two and a half years.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 5 • From baseline up to two and a half years.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 3 • From baseline up to two and a half years.
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 8 • From baseline up to two and a half years.
Nervous system disorders Headache	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 3 • From baseline up to two and a half years.
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 4 • From baseline up to two and a half years.
Respiratory, thoracic and mediastinal disorders Dyspnea	0.00% 0/1 • From baseline up to two and a half years.	10.0% 2/20 • Number of events 4 • From baseline up to two and a half years.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders	0.00% 0/1 • From baseline up to two and a half years.	5.0% 1/20 • Number of events 2 • From baseline up to two and a half years.