Trial Outcomes & Findings for Pembrolizumab in Ultramutated and Hypermutated Endometrial Cancer (NCT NCT02899793)
NCT ID: NCT02899793
Last Updated: 2025-06-11
Results Overview
RECIST (Response Evaluation Criteria In Solid Tumors) is a set of published rules that define when cancer patients improve ("respond"), stay the same ("stable") or worsen ("progression") during treatments. Complete Response (CR) = complete disappearance of all clinical evidence of disease; Partial Response (PR) = regression of measurable disease; Stable Disease (SD) = failure to attain CR, PR, or PD; Progressive Disease (PD) = PD in any compartment; Relapse = recurrence of disease in prior CR in any compartment.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
4 years
Results posted on
2025-06-11
Participant Flow
Participant milestones
| Measure |
Pembrolizumab
Pembrolizumab 200 mg, Q3W, IV Infusion, Day 1 of each 3 week cycle
Pembrolizumab: Pembrolizumab 200 mg (fixed dose) IV every 3 weeks (+/- 3 days) until progression or adverse effects prohibit therapy
|
|---|---|
|
Overall Study
STARTED
|
25
|
|
Overall Study
Excluded From Analysis Due to Tumor Status
|
1
|
|
Overall Study
COMPLETED
|
24
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pembrolizumab in Ultramutated and Hypermutated Endometrial Cancer
Baseline characteristics by cohort
| Measure |
Pembrolizumab
n=24 Participants
Pembrolizumab 200 mg, Q3W, IV Infusion, Day 1 of each 3 week cycle
Pembrolizumab: Pembrolizumab 200 mg (fixed dose) IV every 3 weeks (+/- 3 days) until progression or adverse effects prohibit therapy
|
|---|---|
|
Age, Continuous
|
69 years
STANDARD_DEVIATION 10.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
22 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
23 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
24 participants
n=5 Participants
|
|
International Federation of Gynecology and Obstetrics (FIGO) Stage at diagnosis, No. (%)
Stage I
|
12 Participants
n=5 Participants
|
|
International Federation of Gynecology and Obstetrics (FIGO) Stage at diagnosis, No. (%)
Stage II
|
1 Participants
n=5 Participants
|
|
International Federation of Gynecology and Obstetrics (FIGO) Stage at diagnosis, No. (%)
Stage III
|
8 Participants
n=5 Participants
|
|
International Federation of Gynecology and Obstetrics (FIGO) Stage at diagnosis, No. (%)
Stage IV
|
3 Participants
n=5 Participants
|
|
Histology/grade of Endometrial Adenocarcinoma (EAC)
EAC G1
|
2 Participants
n=5 Participants
|
|
Histology/grade of Endometrial Adenocarcinoma (EAC)
EAC G2
|
9 Participants
n=5 Participants
|
|
Histology/grade of Endometrial Adenocarcinoma (EAC)
EAC G3
|
13 Participants
n=5 Participants
|
|
Microsatellite instability-high (MSI-H)/mismatch repair deficiency (dMMR) Subgroups Characteristics
Lynch-like
|
6 Participants
n=5 Participants
|
|
Microsatellite instability-high (MSI-H)/mismatch repair deficiency (dMMR) Subgroups Characteristics
Methylated
|
18 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 4 yearsRECIST (Response Evaluation Criteria In Solid Tumors) is a set of published rules that define when cancer patients improve ("respond"), stay the same ("stable") or worsen ("progression") during treatments. Complete Response (CR) = complete disappearance of all clinical evidence of disease; Partial Response (PR) = regression of measurable disease; Stable Disease (SD) = failure to attain CR, PR, or PD; Progressive Disease (PD) = PD in any compartment; Relapse = recurrence of disease in prior CR in any compartment.
Outcome measures
| Measure |
Pembrolizumab
n=24 Participants
Pembrolizumab 200 mg, Q3W, IV Infusion, Day 1 of each 3 week cycle
Pembrolizumab: Pembrolizumab 200 mg (fixed dose) IV every 3 weeks (+/- 3 days) until progression or adverse effects prohibit therapy
|
|---|---|
|
Frequency of Objective Tumor Response as Assessed by RECIST 1.1- Overall Response Rate (ORR)
CR
|
5 Participants
|
|
Frequency of Objective Tumor Response as Assessed by RECIST 1.1- Overall Response Rate (ORR)
PR
|
9 Participants
|
|
Frequency of Objective Tumor Response as Assessed by RECIST 1.1- Overall Response Rate (ORR)
SD
|
7 Participants
|
|
Frequency of Objective Tumor Response as Assessed by RECIST 1.1- Overall Response Rate (ORR)
PD
|
3 Participants
|
PRIMARY outcome
Timeframe: 4 yearsCommon Terminology Criteria for Adverse Events (CTCAE) are a set of criteria for the standardized classification of adverse effects of drugs used in cancer therapy. Number of patients affected by each adverse event grade for Other (non-serious) adverse events with in the treatment period.
Outcome measures
| Measure |
Pembrolizumab
n=25 Participants
Pembrolizumab 200 mg, Q3W, IV Infusion, Day 1 of each 3 week cycle
Pembrolizumab: Pembrolizumab 200 mg (fixed dose) IV every 3 weeks (+/- 3 days) until progression or adverse effects prohibit therapy
|
|---|---|
|
Toxicity Grade of Adverse Events as Assessed by CTCAE v4
Grade 1
|
19 Participants
|
|
Toxicity Grade of Adverse Events as Assessed by CTCAE v4
Grade 2
|
6 Participants
|
|
Toxicity Grade of Adverse Events as Assessed by CTCAE v4
Grade 3
|
0 Participants
|
|
Toxicity Grade of Adverse Events as Assessed by CTCAE v4
Grade 4
|
0 Participants
|
SECONDARY outcome
Timeframe: 6 yearsProgression-free survival is defined as the duration of time from study entry to time of progression, death, or the date of last contact, whichever occurs first.
Outcome measures
| Measure |
Pembrolizumab
n=24 Participants
Pembrolizumab 200 mg, Q3W, IV Infusion, Day 1 of each 3 week cycle
Pembrolizumab: Pembrolizumab 200 mg (fixed dose) IV every 3 weeks (+/- 3 days) until progression or adverse effects prohibit therapy
|
|---|---|
|
Duration of Progression-free Survival (PFS)
|
27.4 months
Interval 10.7 to
Upper limit confidence interval was not reached due to insufficient number of events
|
SECONDARY outcome
Timeframe: 6 yearsOverall survival is defined as the duration of time from study entry to time of death or the date of last contact.
Outcome measures
| Measure |
Pembrolizumab
n=24 Participants
Pembrolizumab 200 mg, Q3W, IV Infusion, Day 1 of each 3 week cycle
Pembrolizumab: Pembrolizumab 200 mg (fixed dose) IV every 3 weeks (+/- 3 days) until progression or adverse effects prohibit therapy
|
|---|---|
|
Overall Survival (OS)
|
71.5 months
Interval 28.6 to
Upper limit confidence interval was not reached due to insufficient number of events
|
Adverse Events
Pembrolizumab
Serious events: 23 serious events
Other events: 25 other events
Deaths: 12 deaths
Serious adverse events
| Measure |
Pembrolizumab
n=25 participants at risk
Pembrolizumab 200 mg, Q3W, IV Infusion, Day 1 of each 3 week cycle
Pembrolizumab: Pembrolizumab 200 mg (fixed dose) IV every 3 weeks (+/- 3 days) until progression or adverse effects prohibit therapy
|
|---|---|
|
Endocrine disorders
Hyperthyroidism
|
4.0%
1/25 • 6 years
|
|
Gastrointestinal disorders
Abdominal pain
|
16.0%
4/25 • 6 years
|
|
Gastrointestinal disorders
Diarrhea
|
4.0%
1/25 • 6 years
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
8.0%
2/25 • 6 years
|
|
General disorders
Fever
|
8.0%
2/25 • 6 years
|
|
General disorders
Pain
|
4.0%
1/25 • 6 years
|
|
Infections and infestations
Urinary tract infection
|
16.0%
4/25 • 6 years
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
4.0%
1/25 • 6 years
|
|
Injury, poisoning and procedural complications
Hip fracture
|
4.0%
1/25 • 6 years
|
|
Nervous system disorders
Headache
|
4.0%
1/25 • 6 years
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, specify
|
4.0%
1/25 • 6 years
|
|
Renal and urinary disorders
Urinary retention
|
4.0%
1/25 • 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
4.0%
1/25 • 6 years
|
|
Vascular disorders
Hypotension
|
8.0%
2/25 • 6 years
|
Other adverse events
| Measure |
Pembrolizumab
n=25 participants at risk
Pembrolizumab 200 mg, Q3W, IV Infusion, Day 1 of each 3 week cycle
Pembrolizumab: Pembrolizumab 200 mg (fixed dose) IV every 3 weeks (+/- 3 days) until progression or adverse effects prohibit therapy
|
|---|---|
|
General disorders
Fatigue
|
64.0%
16/25 • 6 years
|
|
General disorders
Pain
|
40.0%
10/25 • 6 years
|
|
General disorders
Edema limbs
|
32.0%
8/25 • 6 years
|
|
General disorders
Fever
|
12.0%
3/25 • 6 years
|
|
General disorders
Chills
|
8.0%
2/25 • 6 years
|
|
General disorders
Infusion related reaction
|
8.0%
2/25 • 6 years
|
|
Gastrointestinal disorders
Diarrhea
|
28.0%
7/25 • 6 years
|
|
Gastrointestinal disorders
Nausea
|
28.0%
7/25 • 6 years
|
|
Gastrointestinal disorders
Constipation
|
20.0%
5/25 • 6 years
|
|
Gastrointestinal disorders
Abdominal pain
|
16.0%
4/25 • 6 years
|
|
Gastrointestinal disorders
Vomiting
|
16.0%
4/25 • 6 years
|
|
Gastrointestinal disorders
Dry mouth
|
12.0%
3/25 • 6 years
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder -Other, specify
|
44.0%
11/25 • 6 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
32.0%
8/25 • 6 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
20.0%
5/25 • 6 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
16.0%
4/25 • 6 years
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
12.0%
3/25 • 6 years
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
8.0%
2/25 • 6 years
|
|
Infections and infestations
Urinary tract infection
|
32.0%
8/25 • 6 years
|
|
Infections and infestations
Infections and infestations - Other, specify
|
20.0%
5/25 • 6 years
|
|
Infections and infestations
Upper respiratory infection
|
20.0%
5/25 • 6 years
|
|
Infections and infestations
Skin infection
|
12.0%
3/25 • 6 years
|
|
Infections and infestations
Tooth infection
|
8.0%
2/25 • 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
20.0%
5/25 • 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
16.0%
4/25 • 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.0%
3/25 • 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
8.0%
2/25 • 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
8.0%
2/25 • 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
8.0%
2/25 • 6 years
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
20.0%
5/25 • 6 years
|
|
Metabolism and nutrition disorders
Anorexia
|
12.0%
3/25 • 6 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
12.0%
3/25 • 6 years
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
12.0%
3/25 • 6 years
|
|
Metabolism and nutrition disorders
Hypokalemia
|
8.0%
2/25 • 6 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
8.0%
2/25 • 6 years
|
|
Renal and urinary disorders
Hematuria
|
16.0%
4/25 • 6 years
|
|
Renal and urinary disorders
Urinary frequency
|
16.0%
4/25 • 6 years
|
|
Renal and urinary disorders
Urinary incontinence
|
12.0%
3/25 • 6 years
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, specify
|
8.0%
2/25 • 6 years
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
28.0%
7/25 • 6 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
20.0%
5/25 • 6 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
8.0%
2/25 • 6 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
8.0%
2/25 • 6 years
|
|
Endocrine disorders
Hypothyroidism
|
24.0%
6/25 • 6 years
|
|
Endocrine disorders
Endocrine disorders - Other, specify
|
8.0%
2/25 • 6 years
|
|
Investigations
Creatinine increased
|
20.0%
5/25 • 6 years
|
|
Investigations
Alanine aminotransferase increased
|
8.0%
2/25 • 6 years
|
|
Investigations
Aspartate aminotransferase increased
|
8.0%
2/25 • 6 years
|
|
Investigations
Weight loss
|
8.0%
2/25 • 6 years
|
|
Nervous system disorders
Headache
|
16.0%
4/25 • 6 years
|
|
Vascular disorders
Hypertension
|
12.0%
3/25 • 6 years
|
|
Vascular disorders
Hypotension
|
8.0%
2/25 • 6 years
|
|
Blood and lymphatic system disorders
Anemia
|
16.0%
4/25 • 6 years
|
|
Cardiac disorders
Atrial fibrillation
|
8.0%
2/25 • 6 years
|
|
Ear and labyrinth disorders
Vertigo
|
8.0%
2/25 • 6 years
|
|
Eye disorders
Dry eye
|
12.0%
3/25 • 6 years
|
|
Eye disorders
Eye disorders - Other, specify
|
8.0%
2/25 • 6 years
|
|
Psychiatric disorders
Anxiety
|
8.0%
2/25 • 6 years
|
Additional Information
Dr. Alessandro Santin
Yale University School of Medicine
Phone: 203-737-4450
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place