Trial Outcomes & Findings for Pembrolizumab Before Surgery for the Treatment of Mismatch Repair Deficient Locally Advanced Solid Cancers (NCT NCT04082572)
NCT ID: NCT04082572
Last Updated: 2025-05-13
Results Overview
Number of patients with Pathologic complete response (pCR) as defined by absence of any residual viable tumor of the macroscopically identifiable tumor bed or lymph node after 3 doses.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
35 participants
Primary outcome timeframe
1 year
Results posted on
2025-05-13
Participant Flow
October 31, 2019, and March 25, 2021
Participant milestones
| Measure |
Neoadjuvant Pembrolizumab in Localized MSI Instability High/Deficient Mismatch Repair Solid Tumors
Pembrolizumab given pre-operatively for 6 months in patients with locally advanced (unresectable primary cancer or a resectable primary cancer with a high chance of recurrence) dMMR solid organ tumors
|
|---|---|
|
Overall Study
STARTED
|
35
|
|
Overall Study
COMPLETED
|
35
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pembrolizumab Before Surgery for the Treatment of Mismatch Repair Deficient Locally Advanced Solid Cancers
Baseline characteristics by cohort
| Measure |
Neoadjuvant Pembrolizumab in Localized MSI Instability High/Deficient Mismatch Repair Solid Tumors
n=35 Participants
Pembrolizumab given pre-operatively for 6 months in patients with locally advanced (unresectable primary cancer or a resectable primary cancer with a high chance of recurrence) dMMR solid organ tumors
|
|---|---|
|
Age, Continuous
|
62 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
28 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
33 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
35 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 1 yearPopulation: 35 patients were enrolled in the study. The primary efficacy end point was evaluable in only 15 patients. 17 patients (49%) had surgical resection. However, 2 patients had a resection after 1 and 2 cycles of treatment. Thus only 15 patients were found to be eligible (resection after 3 cycles of treatment with pembrolizumab).
Number of patients with Pathologic complete response (pCR) as defined by absence of any residual viable tumor of the macroscopically identifiable tumor bed or lymph node after 3 doses.
Outcome measures
| Measure |
Neoadjuvant Pembrolizumab in Localized MSI Instability High/Deficient Mismatch Repair Solid Tumors
n=15 Participants
Pembrolizumab given pre-operatively for 6 months in patients with locally advanced (unresectable primary cancer or a resectable primary cancer with a high chance of recurrence) dMMR solid organ tumors
|
|---|---|
|
Pathological Complete Response (pCR).
|
67 Percentage
|
PRIMARY outcome
Timeframe: At 1 yearNumber of patients that developed toxicity as assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 4.
Outcome measures
| Measure |
Neoadjuvant Pembrolizumab in Localized MSI Instability High/Deficient Mismatch Repair Solid Tumors
n=35 Participants
Pembrolizumab given pre-operatively for 6 months in patients with locally advanced (unresectable primary cancer or a resectable primary cancer with a high chance of recurrence) dMMR solid organ tumors
|
|---|---|
|
Safety of Neo-adjuvant Pembrolizumab in Patients With Locally Advanced (Unresectable Primary Cancer or Resectable Primary Cancer With a High Chance of Recurrence) Mismatch Repair Protein Deficiency (dMMR) Solid Organ Tumors.
|
35 participants
|
PRIMARY outcome
Timeframe: Up to 2 yearsPopulation: 35 patients were enrolled in the study. Only 17 patients underwent resection. out of the 17 patients that underwent resection, 3 patients developed post surgical complications.
Number of patients with post surgical complication based on the Clavien-Dindo Classification
Outcome measures
| Measure |
Neoadjuvant Pembrolizumab in Localized MSI Instability High/Deficient Mismatch Repair Solid Tumors
n=17 Participants
Pembrolizumab given pre-operatively for 6 months in patients with locally advanced (unresectable primary cancer or a resectable primary cancer with a high chance of recurrence) dMMR solid organ tumors
|
|---|---|
|
Post-surgical Complication Assessment by the Clavien- Dindo Classification.
|
3 participants
|
SECONDARY outcome
Timeframe: From treatment start till death or last follow-up, assessed up to 2 yearsPopulation: 35 patients were enrolled but only 33 patients were evaluable for radiographic response. 2 patients were not evaluable due to clinical progression and death due to an unrelated hip fracture in a 90 year old patient before restaging.
The ORR was estimated using Recist 1.1. ORR (Complete response + Partial response)
Outcome measures
| Measure |
Neoadjuvant Pembrolizumab in Localized MSI Instability High/Deficient Mismatch Repair Solid Tumors
n=33 Participants
Pembrolizumab given pre-operatively for 6 months in patients with locally advanced (unresectable primary cancer or a resectable primary cancer with a high chance of recurrence) dMMR solid organ tumors
|
|---|---|
|
Overall Response Rate
|
27 participants
|
SECONDARY outcome
Timeframe: 1 yearPopulation: 35 patients were enrolled but only 33 patients were evaluable for radiographic response. 2 patients were not evaluable due to clinical progression and death due to an unrelated hip fracture in a 90 year old patient before restaging
Best overall RECIST response as determined by the number of patients with Partial Response and or Complete Response measured using Recist v1.1
Outcome measures
| Measure |
Neoadjuvant Pembrolizumab in Localized MSI Instability High/Deficient Mismatch Repair Solid Tumors
n=33 Participants
Pembrolizumab given pre-operatively for 6 months in patients with locally advanced (unresectable primary cancer or a resectable primary cancer with a high chance of recurrence) dMMR solid organ tumors
|
|---|---|
|
Radiographic Tumor Response to Neoadjuvant Pembrolizumab.
|
27 participants
|
Adverse Events
Neoadjuvant Pembrolizumab in Localized MSI Instability High/Deficient Mismatch Repair Solid Tumors
Serious events: 0 serious events
Other events: 35 other events
Deaths: 2 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Neoadjuvant Pembrolizumab in Localized MSI Instability High/Deficient Mismatch Repair Solid Tumors
n=35 participants at risk
Pembrolizumab
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
88.6%
31/35 • 2 Year
|
|
Cardiac disorders
Sinus Tachycardia
|
11.4%
4/35 • 2 Year
|
|
Cardiac disorders
Sinus bradycardia
|
5.7%
2/35 • 2 Year
|
|
Cardiac disorders
Cardiac disorders - Other, specify
|
2.9%
1/35 • 2 Year
|
|
Nervous system disorders
Hearing impaired
|
2.9%
1/35 • 2 Year
|
|
Endocrine disorders
Hypothyroidism
|
11.4%
4/35 • 2 Year
|
|
Endocrine disorders
Hypoparathyroidism
|
2.9%
1/35 • 2 Year
|
|
Endocrine disorders
Hyperthryroidism
|
5.7%
2/35 • 2 Year
|
|
Eye disorders
Blurred Vision
|
8.6%
3/35 • 2 Year
|
|
Eye disorders
Watering Eyes
|
2.9%
1/35 • 2 Year
|
|
Eye disorders
Photophobia
|
2.9%
1/35 • 2 Year
|
|
Eye disorders
Eye disorders - Other, specify
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Abdominal distension
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Abdominal pain
|
25.7%
9/35 • 2 Year
|
|
Gastrointestinal disorders
Anal hemorrhage
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Constipation
|
8.6%
3/35 • 2 Year
|
|
Gastrointestinal disorders
Diarrhea
|
5.7%
2/35 • 2 Year
|
|
Gastrointestinal disorders
Dry mouth
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Dyspepsia
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Gastritis
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Nausea
|
11.4%
4/35 • 2 Year
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Rectal pain
|
2.9%
1/35 • 2 Year
|
|
Gastrointestinal disorders
Vomiting
|
2.9%
1/35 • 2 Year
|
|
General disorders
Fatigue
|
20.0%
7/35 • 2 Year
|
|
General disorders
Fever
|
2.9%
1/35 • 2 Year
|
|
General disorders
Flu like symptoms
|
2.9%
1/35 • 2 Year
|
|
General disorders
Pain
|
2.9%
1/35 • 2 Year
|
|
Infections and infestations
Sepsis
|
5.7%
2/35 • 2 Year
|
|
Renal and urinary disorders
Urinary Tract Infection
|
5.7%
2/35 • 2 Year
|
|
Infections and infestations
Vulval Infection
|
2.9%
1/35 • 2 Year
|
|
Injury, poisoning and procedural complications
Fall
|
5.7%
2/35 • 2 Year
|
|
Investigations
Alanine aminotransferase increased
|
40.0%
14/35 • 2 Year
|
|
Investigations
Alkaline phosphatase increased
|
25.7%
9/35 • 2 Year
|
|
Investigations
Aspartate aminotransferase increased
|
31.4%
11/35 • 2 Year
|
|
Investigations
Blood bilirubin increased
|
8.6%
3/35 • 2 Year
|
|
Investigations
Creatinine increased
|
14.3%
5/35 • 2 Year
|
|
Investigations
Investigations - Other, specify
|
77.1%
27/35 • 2 Year
|
|
Investigations
Neutrophil count decreased
|
2.9%
1/35 • 2 Year
|
|
Investigations
Weight loss
|
5.7%
2/35 • 2 Year
|
|
Investigations
White blood cell decreased
|
2.9%
1/35 • 2 Year
|
|
Investigations
Hypercalcemia
|
8.6%
3/35 • 2 Year
|
|
Investigations
Hyperglycemia
|
57.1%
20/35 • 2 Year
|
|
Investigations
Hyperkalemia
|
8.6%
3/35 • 2 Year
|
|
Investigations
Hypermagnesemia
|
2.9%
1/35 • 2 Year
|
|
Investigations
Hyperuricemia
|
31.4%
11/35 • 2 Year
|
|
Investigations
Hypoalbuminemia
|
2.9%
1/35 • 2 Year
|
|
Investigations
Hypokalemia
|
20.0%
7/35 • 2 Year
|
|
Investigations
Hypomagnesemia
|
2.9%
1/35 • 2 Year
|
|
Investigations
Hyponatremia
|
22.9%
8/35 • 2 Year
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders - Other, specify
|
2.9%
1/35 • 2 Year
|
|
Metabolism and nutrition disorders
Anorexia
|
8.6%
3/35 • 2 Year
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.7%
2/35 • 2 Year
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.7%
2/35 • 2 Year
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
2.9%
1/35 • 2 Year
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
2.9%
1/35 • 2 Year
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal deformity
|
2.9%
1/35 • 2 Year
|
|
Musculoskeletal and connective tissue disorders
Osteonecrosis of jaw
|
2.9%
1/35 • 2 Year
|
|
Nervous system disorders
Dysgeusia
|
2.9%
1/35 • 2 Year
|
|
Nervous system disorders
Headache
|
8.6%
3/35 • 2 Year
|
|
Nervous system disorders
Memory impairment
|
2.9%
1/35 • 2 Year
|
|
Nervous system disorders
Concentration impairment
|
2.9%
1/35 • 2 Year
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
5.7%
2/35 • 2 Year
|
|
Nervous system disorders
Presyncope
|
2.9%
1/35 • 2 Year
|
|
Nervous system disorders
Syncope
|
2.9%
1/35 • 2 Year
|
|
Nervous system disorders
Dizziness
|
2.9%
1/35 • 2 Year
|
|
Nervous system disorders
Insomnia
|
2.9%
1/35 • 2 Year
|
|
Renal and urinary disorders
Urinary Urgency
|
2.9%
1/35 • 2 Year
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, specify
|
2.9%
1/35 • 2 Year
|
|
Reproductive system and breast disorders
Vaginal Pain
|
2.9%
1/35 • 2 Year
|
|
Reproductive system and breast disorders
Uterine Hemorrahage
|
2.9%
1/35 • 2 Year
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
5.7%
2/35 • 2 Year
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
2.9%
1/35 • 2 Year
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.9%
1/35 • 2 Year
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
2.9%
1/35 • 2 Year
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
2.9%
1/35 • 2 Year
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
8.6%
3/35 • 2 Year
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
2.9%
1/35 • 2 Year
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
5.7%
2/35 • 2 Year
|
|
Vascular disorders
Hypertension
|
20.0%
7/35 • 2 Year
|
|
Vascular disorders
Hypotension
|
5.7%
2/35 • 2 Year
|
|
Vascular disorders
Thromboembolic event
|
2.9%
1/35 • 2 Year
|
|
Vascular disorders
Vascular disorders - Other, specif
|
2.9%
1/35 • 2 Year
|
|
General disorders
Hot flashes
|
2.9%
1/35 • 2 Year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place