Trial Outcomes & Findings for Sorafenib Tosylate and Pembrolizumab in Treating Patients With Advanced or Metastatic Liver Cancer (NCT NCT03211416)

NCT ID: NCT03211416

Last Updated: 2026-02-05

Results Overview

The response rate will be estimated as the binomial proportion of responders among evaluable patients, and supported by Jeffreys? 95% confidence interval.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 37 participants
• Primary outcome timeframe: Up to 6 months
• Results posted on: 2026-02-05

Participant Flow

Participant milestones

Measure	Treatment (Sorafenib Tosylate, Pembrolizumab) Patients receive sorafenib tosylate PO BID on days -28 to -1 and 1-21. Patients also receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV Sorafenib Tosylate: Given PO
Overall Study STARTED	37
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Measure	Treatment (Sorafenib Tosylate, Pembrolizumab) Patients receive sorafenib tosylate PO BID on days -28 to -1 and 1-21. Patients also receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV Sorafenib Tosylate: Given PO
Overall Study Adverse Event	7
Overall Study Lack of Efficacy	2
Overall Study Physician Decision	1

Baseline Characteristics

Sorafenib Tosylate and Pembrolizumab in Treating Patients With Advanced or Metastatic Liver Cancer

Baseline characteristics by cohort

Measure	Treatment (Sorafenib Tosylate, Pembrolizumab) n=37 Participants Patients receive sorafenib tosylate PO BID on days -28 to -1 and 1-21. Patients also receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV Sorafenib Tosylate: Given PO
Age, Continuous	67.4 years STANDARD_DEVIATION 9.7 • n=25 Participants
Sex: Female, Male Female	11 Participants n=25 Participants
Sex: Female, Male Male	26 Participants n=25 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=25 Participants
Race (NIH/OMB) Asian	0 Participants n=25 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=25 Participants
Race (NIH/OMB) Black or African American	5 Participants n=25 Participants
Race (NIH/OMB) White	26 Participants n=25 Participants
Race (NIH/OMB) More than one race	0 Participants n=25 Participants
Race (NIH/OMB) Unknown or Not Reported	6 Participants n=25 Participants
Region of Enrollment United States	37 participants n=25 Participants
Body Mass index	28.5 kg/m^2 STANDARD_DEVIATION 5.3 • n=25 Participants

PRIMARY outcome

Timeframe: Up to 6 months

Population: 10 subjects were not evaluable for response.

The response rate will be estimated as the binomial proportion of responders among evaluable patients, and supported by Jeffreys? 95% confidence interval.

Outcome measures

Measure	Treatment (Sorafenib Tosylate, Pembrolizumab) n=27 Participants Patients receive sorafenib tosylate PO BID on days -28 to -1 and 1-21. Patients also receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV Sorafenib Tosylate: Given PO
Overall Response Rate Defined as Partial or Complete Response Per Immune-related Response Evaluation Criteria in Solid Tumors Responder (CR+PR)	8 Participants
Overall Response Rate Defined as Partial or Complete Response Per Immune-related Response Evaluation Criteria in Solid Tumors non-Responder (SD+PD)	19 Participants

SECONDARY outcome

Timeframe: From the date of study enrollment to the time of death from any cause, assessed up to 3 year

Will be estimated using the Kaplan-Meier method.

Outcome measures

Measure	Treatment (Sorafenib Tosylate, Pembrolizumab) n=37 Participants Patients receive sorafenib tosylate PO BID on days -28 to -1 and 1-21. Patients also receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV Sorafenib Tosylate: Given PO
Overall Survival	17.1 months Interval 6.2 to 37.2

SECONDARY outcome

Timeframe: From the date of study enrollment to the first observation of progressive disease, assessed up to 3 years

Will be estimated using the Kaplan-Meier method, where the median will be estimated with a 95% confidence interval.

Outcome measures

Measure	Treatment (Sorafenib Tosylate, Pembrolizumab) n=37 Participants Patients receive sorafenib tosylate PO BID on days -28 to -1 and 1-21. Patients also receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV Sorafenib Tosylate: Given PO
Time to Tumor Progression	3.7 months Interval 2.5 to 8.7

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 3 years

Population: Only 20 subjects had paired pre-treatment and post-treatment flow data available.

The effect of treatment will be quantified as the post/pre-treatment mean ratio of the expression measurements, assessed using a two-sided one sample t-test.

Outcome measures

Measure	Treatment (Sorafenib Tosylate, Pembrolizumab) n=20 Participants Patients receive sorafenib tosylate PO BID on days -28 to -1 and 1-21. Patients also receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV Sorafenib Tosylate: Given PO
Change in Functional Activity of Effector T Cells	1.17 ratio Standard Deviation 0.84

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 3 years

Population: Data was not captured.

The effect of treatment will be quantified as the post/pre-treatment mean ratio of the expression measurements, assessed using a two-sided one sample t-test.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Sorafenib Tosylate, Pembrolizumab)

Serious events: 20 serious events

Other events: 36 other events

Deaths: 24 deaths

Serious adverse events

Measure	Treatment (Sorafenib Tosylate, Pembrolizumab) n=37 participants at risk Patients receive sorafenib tosylate PO BID on days -28 to -1 and 1-21. Patients also receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV Sorafenib Tosylate: Given PO
Cardiac disorders Aortic valve disease	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Cardiac disorders Heart failure	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Abdominal pain	5.4% 2/37 • Number of events 2 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Duodenal hemorrhage	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Lower gastrointestinal hemorrhage	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Oral pain	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
General disorders Death NOS	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
General disorders Fever	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
General disorders Multi-organ failure	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Hepatobiliary disorders Hepatic failure	5.4% 2/37 • Number of events 2 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Immune system disorders Immune system disorders - Other	10.8% 4/37 • Number of events 4 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Infections and infestations Appendicitis perforated	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Infections and infestations Enterocolitis infectious	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Infections and infestations Infections and infestations - Other	5.4% 2/37 • Number of events 2 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Infections and infestations Sepsis	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Metabolism and nutrition disorders Anorexia	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Metabolism and nutrition disorders Dehydration	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Metabolism and nutrition disorders Hyperkalemia	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Metabolism and nutrition disorders Hyponatremia	5.4% 2/37 • Number of events 2 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Respiratory, thoracic and mediastinal disorders Pulmonary edema	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Respiratory, thoracic and mediastinal disorders Respiratory failure	2.7% 1/37 • Number of events 1 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.

Other adverse events

Measure	Treatment (Sorafenib Tosylate, Pembrolizumab) n=37 participants at risk Patients receive sorafenib tosylate PO BID on days -28 to -1 and 1-21. Patients also receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pembrolizumab: Given IV Sorafenib Tosylate: Given PO
Blood and lymphatic system disorders Anemia	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Endocrine disorders Hypothyroidism	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Abdominal pain	21.6% 8/37 • Number of events 8 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Constipation	13.5% 5/37 • Number of events 5 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Diarrhea	62.2% 23/37 • Number of events 23 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Mucositis oral	10.8% 4/37 • Number of events 4 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Nausea	21.6% 8/37 • Number of events 8 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Gastrointestinal disorders Vomiting	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
General disorders Chills	16.2% 6/37 • Number of events 6 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
General disorders Edema limbs	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
General disorders Fatigue	56.8% 21/37 • Number of events 21 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
General disorders Fever	10.8% 4/37 • Number of events 4 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Investigations Alanine aminotransferase increased	10.8% 4/37 • Number of events 4 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Investigations Aspartate aminotransferase increased	18.9% 7/37 • Number of events 7 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Investigations Blood bilirubin increased	24.3% 9/37 • Number of events 9 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Investigations Lymphocyte count decreased	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Investigations Weight loss	35.1% 13/37 • Number of events 13 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Metabolism and nutrition disorders Anorexia	54.1% 20/37 • Number of events 20 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Musculoskeletal and connective tissue disorders Arthralgia	13.5% 5/37 • Number of events 5 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	24.3% 9/37 • Number of events 9 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Musculoskeletal and connective tissue disorders Myalgia	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Nervous system disorders Dizziness	10.8% 4/37 • Number of events 4 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Nervous system disorders Dysgeusia	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Respiratory, thoracic and mediastinal disorders Dyspnea	8.1% 3/37 • Number of events 3 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Respiratory, thoracic and mediastinal disorders Hoarseness	10.8% 4/37 • Number of events 4 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Skin and subcutaneous tissue disorders Alopecia	10.8% 4/37 • Number of events 4 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Skin and subcutaneous tissue disorders Dry skin	16.2% 6/37 • Number of events 6 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrome	43.2% 16/37 • Number of events 16 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Skin and subcutaneous tissue disorders Pruritus	10.8% 4/37 • Number of events 4 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Skin and subcutaneous tissue disorders Rash maculo-papular	29.7% 11/37 • Number of events 11 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.
Vascular disorders Hypertension	21.6% 8/37 • Number of events 8 • Adverse events were followed for up to 30 days post-treatment initiation, which was a median of 140 days (range = 41 to 1,162 days). All cause mortality was monitored for up to 3 years following treatment initiation.

Additional Information

Kris Attwood

Roswell Park Comprehensive Cancer Center

Phone: 8772757724

Email: Kris.Attwood@roswellpark.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place