Trial Outcomes & Findings for Pembrolizumab (MK-3475) in Hepatocellular Carcinoma (NCT NCT02940496)
NCT ID: NCT02940496
Last Updated: 2025-07-15
Results Overview
HCC stiffness was measured using magnetic resonance elastography (MRE) in kilopascals (kPa) and the change was compared with overall survival (OS). Analysis was performed using descriptive statistics and Spearman correlation (R); p-value \< 0.05 was considered statistically significant.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
up to 2 years
Results posted on
2025-07-15
Participant Flow
Eligible patients were over 18 years of age with radiographic disease progression on sorafenib or intolerance to sorafenib treatment, and ECOG 0 or 1. All patients underwent liver MRI with MR Elastography (MRE) and liver biopsy at baseline and at 6 weeks of therapy. Only the Pembrolizumab arm enrolled patients.
Participant milestones
| Measure |
"ARM A Pembrolizumab (Q3W)"
Biopsy proven advanced HCC (not amenable to curative therapy), Child-Pugh Score A, who were treated with anti-PD-1, Pembrolizumab monotherapy.
|
"ARM B Pembrolizumab (Q3W)+Grazoprevir (qd x 12 Weeks)+Elbasvir (QD x 12 Weeks"
This arm was closed after activation and no patients was enrolled into this arm.
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
0
|
|
Overall Study
COMPLETED
|
9
|
0
|
|
Overall Study
NOT COMPLETED
|
6
|
0
|
Reasons for withdrawal
| Measure |
"ARM A Pembrolizumab (Q3W)"
Biopsy proven advanced HCC (not amenable to curative therapy), Child-Pugh Score A, who were treated with anti-PD-1, Pembrolizumab monotherapy.
|
"ARM B Pembrolizumab (Q3W)+Grazoprevir (qd x 12 Weeks)+Elbasvir (QD x 12 Weeks"
This arm was closed after activation and no patients was enrolled into this arm.
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Death
|
2
|
0
|
|
Overall Study
Hospice Care
|
1
|
0
|
|
Overall Study
No MRE Done
|
1
|
0
|
|
Overall Study
MRE Failure
|
1
|
0
|
Baseline Characteristics
Pembrolizumab (MK-3475) in Hepatocellular Carcinoma
Baseline characteristics by cohort
| Measure |
"ARM A Pembrolizumab (Q3W)"
n=15 Participants
Biopsy proven advanced HCC (not amenable to curative therapy), Child-Pugh Score A, who were treated with anti-PD-1, Pembrolizumab monotherapy.
|
"ARM B Pembrolizumab (Q3W)+Grazoprevir (qd x 12 Weeks)+Elbasvir (QD x 12 Weeks"
This arm was closed after activation and no patients was enrolled into this arm.
|
Total
n=15 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
71 years
n=5 Participants
|
—
|
71 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
—
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
—
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=5 Participants
|
—
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
—
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
—
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
—
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=5 Participants
|
—
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
—
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
—
|
15 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: up to 2 yearsPopulation: Arm B was closed after activation and no patients was enrolled into this arm.
HCC stiffness was measured using magnetic resonance elastography (MRE) in kilopascals (kPa) and the change was compared with overall survival (OS). Analysis was performed using descriptive statistics and Spearman correlation (R); p-value \< 0.05 was considered statistically significant.
Outcome measures
| Measure |
"ARM A Pembrolizumab (Q3W)"
n=9 Participants
Biopsy proven advanced HCC (not amenable to curative therapy), Child-Pugh Score A, who were treated with anti-PD-1, Pembrolizumab monotherapy.
|
"ARM B Pembrolizumab (Q3W)+Grazoprevir (qd x 12 Weeks)+Elbasvir (QD x 12 Weeks"
This arm was closed after activation and no patients was enrolled into this arm.
|
|---|---|---|
|
Correlation of HCC Stiffness to Overall Survival
|
0.12 kilopascals
Interval -2.1 to 2.8
|
—
|
PRIMARY outcome
Timeframe: up to 2 yearsPopulation: Arm B was closed after activation and no patients was enrolled into this arm.
HCC stiffness was measured using magnetic resonance elastography (MRE) in kilopascals (kPa) and the change was compared with time to disease progression (TTP). Analysis was performed using descriptive statistics and Spearman correlation (R); p-value \< 0.05 was considered statistically significant.
Outcome measures
| Measure |
"ARM A Pembrolizumab (Q3W)"
n=9 Participants
Biopsy proven advanced HCC (not amenable to curative therapy), Child-Pugh Score A, who were treated with anti-PD-1, Pembrolizumab monotherapy.
|
"ARM B Pembrolizumab (Q3W)+Grazoprevir (qd x 12 Weeks)+Elbasvir (QD x 12 Weeks"
This arm was closed after activation and no patients was enrolled into this arm.
|
|---|---|---|
|
Correlation of HCC Stiffness to Time To Disease Progression (TTP)
|
0.12 kilopascals
Interval -2.1 to 2.8
|
—
|
PRIMARY outcome
Timeframe: assessed prior to treatment at baselinePopulation: Arm B was closed after activation and no patients was enrolled into this arm.
HCC stiffness was measured using magnetic resonance elastography (MRE) in kilopascals (kPa) prior to treatment. Intratumoral CD3+ T lymphocytes count was also obtained through a biopsy prior to treatment and correlation was made between HCC stiffness and CD3+ T Lymphocyte count and the analysis was performed using descriptive statistics and Spearman correlation (R); p-value \< 0.05 was considered statistically significant.
Outcome measures
| Measure |
"ARM A Pembrolizumab (Q3W)"
n=9 Participants
Biopsy proven advanced HCC (not amenable to curative therapy), Child-Pugh Score A, who were treated with anti-PD-1, Pembrolizumab monotherapy.
|
"ARM B Pembrolizumab (Q3W)+Grazoprevir (qd x 12 Weeks)+Elbasvir (QD x 12 Weeks"
This arm was closed after activation and no patients was enrolled into this arm.
|
|---|---|---|
|
Correlation of HCC Stiffness to the Number of Intratumoral CD3+ T Lymphocytes.
|
5.01 kilopascals
Standard Deviation 1.91
|
—
|
Adverse Events
"ARM A Pembrolizumab (Q3W)"
Serious events: 0 serious events
Other events: 3 other events
Deaths: 2 deaths
"ARM B Pembrolizumab (Q3W)+Grazoprevir (qd x 12 Weeks)+Elbasvir (QD x 12 Weeks"
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
"ARM A Pembrolizumab (Q3W)"
n=15 participants at risk
Biopsy proven advanced HCC (not amenable to curative therapy), Child-Pugh Score A, who were treated with anti-PD-1, Pembrolizumab monotherapy.
|
"ARM B Pembrolizumab (Q3W)+Grazoprevir (qd x 12 Weeks)+Elbasvir (QD x 12 Weeks"
This arm was closed after activation and no patients was enrolled into this arm.
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
13.3%
2/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Hyperglycemia
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
13.3%
2/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Gastrointestinal disorders
Diarrhea
|
13.3%
2/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Vascular disorders
Hypertension
|
13.3%
2/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Nervous system disorders
Insomnia
|
13.3%
2/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
General disorders
Pain
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Anemia
|
13.3%
2/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Aspartate aminotransferase increased
|
20.0%
3/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Alanine aminotransferase increased
|
20.0%
3/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Alkaline phosphatase increased
|
13.3%
2/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Creatinine increased
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Nervous system disorders
Anxiety
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Gastrointestinal disorders
Bloating
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
INR increased
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Hypercalcemia
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Investigations - (Other), specify PTT increased
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Gastrointestinal disorders
Constipation
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Musculoskeletal and connective tissue disorders
Rash maculo-papular
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Blood and lymphatic system disorders - (Other), specify Iron deficiency
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
General disorders
Fatigue
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Gastrointestinal disorders
Abdominal pain
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Blood bilirubin increased
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
General disorders
Chills
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
General disorders
Fever
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
General disorders
Anorexia
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Infections and infestations
Sepsis
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Investigations
Platelet count decreased
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
|
Gastrointestinal disorders
Gastrointestinal disorders - (Other), specify Esophageal Varices
|
6.7%
1/15 • up to 2 years
Adverse events that occurred during the conduct of the study
|
—
0/0 • up to 2 years
Adverse events that occurred during the conduct of the study
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place