Trial Outcomes & Findings for Pembrolizumab in Advanced Hepatocellular Carcinoma as Second-line Treatment After Failure of Sorafenib (NCT NCT03163992)

Last Updated: 2022-08-08

Results Overview

Overall response rate (ORR) is defined as the proportion of patients who have a partial or complete response to therapy; it does not include stable disease and is a direct measure of drug tumoricidal activity. (biomarker negative vs biomarker Advanced Hepatocellular Carcinoma) Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by CT or MRI Complete Response (CR); Disappearance of all target lesions Partial Response (PR); \>=30% decrease in the sum of the longest diameter of target lesions Overall Response (OR) = CR + PR

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

60 participants

Primary outcome timeframe

through study completion, an average of 24 months

Results posted on

2022-08-08

Participant Flow

The first patient enrollment date was 26 Dec 2017 and the last patient enrollment date was 11 Jun 2019.

Participant milestones

Participant milestones
Measure	Pembrolizumab Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W) Pembrolizumab: Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W)
Overall Study STARTED	60
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	59

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pembrolizumab in Advanced Hepatocellular Carcinoma as Second-line Treatment After Failure of Sorafenib

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pembrolizumab n=60 Participants Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W) Pembrolizumab: Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W)
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	28 Participants n=5 Participants
Age, Categorical >=65 years	32 Participants n=5 Participants
Age, Continuous	60 years n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants
Sex: Female, Male Male	48 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	60 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	60 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	0 Participants n=5 Participants
Race (NIH/OMB) White	0 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 South Korea	60 Participants n=5 Participants

PRIMARY outcome

Timeframe: through study completion, an average of 24 months

Outcome measures

Outcome measures
Measure	Pembrolizumab n=60 Participants Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W) Pembrolizumab: Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W)
Overall Response Rate NA	7 Participants
Overall Response Rate PR	6 Participants
Overall Response Rate SD	30 Participants
Overall Response Rate PD	17 Participants
Overall Response Rate CR	0 Participants

SECONDARY outcome

Timeframe: through study completion, an average of 24 months

Progression-free survival (PFS) was defined as the time from the started of treatment until the date of disease progression or death resulting from any cause. Overall survival was measured from the started of treatment to the date of death from any cause. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Progression-free survival (PFS) is the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse.

Outcome measures

Outcome measures
Measure	Pembrolizumab n=60 Participants Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W) Pembrolizumab: Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W)
Progression-free Survival (PFS) and Overall Survival (OS) PFS	2.8 months Interval 2.6 to 4.7
Progression-free Survival (PFS) and Overall Survival (OS) OS	8.3 months Interval 5.4 to 10.9

Adverse Events

Pembrolizumab

Serious events: 8 serious events

Other events: 24 other events

Deaths: 35 deaths

Serious adverse events

Serious adverse events
Measure	Pembrolizumab n=60 participants at risk Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W) Pembrolizumab: Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W)
General disorders fatigue	13.3% 8/60 • Number of events 8 • 2 year

Other adverse events

Other adverse events
Measure	Pembrolizumab n=60 participants at risk Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W) Pembrolizumab: Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W)
Skin and subcutaneous tissue disorders pruritis	8.3% 5/60 • Number of events 5 • 2 year
Gastrointestinal disorders diarrhea	6.7% 4/60 • Number of events 4 • 2 year
Gastrointestinal disorders constipation	3.3% 2/60 • Number of events 2 • 2 year
Hepatobiliary disorders elevated liver enzyme	10.0% 6/60 • Number of events 6 • 2 year
Hepatobiliary disorders hyperbilirubinemina	8.3% 5/60 • Number of events 5 • 2 year
Endocrine disorders hyperthyroidism	3.3% 2/60 • Number of events 2 • 2 year

Additional Information

Jeeyun Lee

Samsung Medical Center

Phone: 8223410-1779

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place