Trial Outcomes & Findings for Regorafenib and Durvalumab for the Treatment of High-Risk Liver Cancer (NCT NCT05194293)
NCT ID: NCT05194293
Last Updated: 2026-07-20
Results Overview
An objective response is defined as a complete response (CR) or partial response (PR). Disease status will be assessed using Response Evaluation Criteria in Solid Tumors (RECIST) version (v).1.1 criteria. ORR is defined as the proportion of evaluable patient who experience a CR or PR divided by the total number of evaluable patients. The final ORR point estimate and corresponding 95% confidence interval will be reported.
ACTIVE_NOT_RECRUITING
PHASE2
4 participants
16 weeks
2026-07-20
Participant Flow
Participant milestones
| Measure |
Treatment (Regorafenib, Durvalumab)
Patients receive regorafenib PO QD on days 1-21 and durvalumab IV on day 1. Treatment repeats every 28 days for a maximum of 2 years from registration or until decision to proceed to surgery, disease progression, excessive toxicity, or patient withdrawal.\> \> Durvalumab: Given IV\>
\> Regorafenib: Given PO
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Regorafenib and Durvalumab for the Treatment of High-Risk Liver Cancer
Baseline characteristics by cohort
| Measure |
Treatment (Regorafenib, Durvalumab)
n=4 Participants
Patients receive regorafenib PO QD on days 1-21 and durvalumab IV on day 1. Treatment repeats every 28 days for a maximum of 2 years from registration or until decision to proceed to surgery, disease progression, excessive toxicity, or patient withdrawal.\> \> Durvalumab: Given IV\>
\> Regorafenib: Given PO
|
|---|---|
|
Age, Continuous
|
67.5 years
STANDARD_DEVIATION 12.12 • n=20 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=20 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=20 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=20 Participants
|
|
ECOG Performance Status
0
|
3 Participants
n=20 Participants
|
|
ECOG Performance Status
1
|
1 Participants
n=20 Participants
|
PRIMARY outcome
Timeframe: 16 weeksAn objective response is defined as a complete response (CR) or partial response (PR). Disease status will be assessed using Response Evaluation Criteria in Solid Tumors (RECIST) version (v).1.1 criteria. ORR is defined as the proportion of evaluable patient who experience a CR or PR divided by the total number of evaluable patients. The final ORR point estimate and corresponding 95% confidence interval will be reported.
Outcome measures
| Measure |
Treatment (Regorafenib, Durvalumab)
n=4 Participants
Patients receive regorafenib PO QD on days 1-21 and durvalumab IV on day 1. Treatment repeats every 28 days for a maximum of 2 years from registration or until decision to proceed to surgery, disease progression, excessive toxicity, or patient withdrawal.\> \> Durvalumab: Given IV\>
\> Regorafenib: Given PO
|
|---|---|
|
Objective Response Rate (ORR) (Unconfirmed)
|
0 percentage of participants
Interval 0.0 to 0.6
|
SECONDARY outcome
Timeframe: 10 monthsThe proportion of patients who underwent surgery within 4 months of the last treatment dose during the course of the study. The 95% confidence interval calculated by Clopper and Pearson will be reported.
Outcome measures
| Measure |
Treatment (Regorafenib, Durvalumab)
n=4 Participants
Patients receive regorafenib PO QD on days 1-21 and durvalumab IV on day 1. Treatment repeats every 28 days for a maximum of 2 years from registration or until decision to proceed to surgery, disease progression, excessive toxicity, or patient withdrawal.\> \> Durvalumab: Given IV\>
\> Regorafenib: Given PO
|
|---|---|
|
Rate of Surgery During the Course of the Study
|
1.0 proportion of participants
Interval 0.4 to 1.0
|
SECONDARY outcome
Timeframe: 2 yearsThe rate of patients experiencing a grade 3+ adverse event will be reported. Further analyses of adverse event rates will be considered exploratory.
Outcome measures
| Measure |
Treatment (Regorafenib, Durvalumab)
n=4 Participants
Patients receive regorafenib PO QD on days 1-21 and durvalumab IV on day 1. Treatment repeats every 28 days for a maximum of 2 years from registration or until decision to proceed to surgery, disease progression, excessive toxicity, or patient withdrawal.\> \> Durvalumab: Given IV\>
\> Regorafenib: Given PO
|
|---|---|
|
Number of Patients Experiencing Adverse Events
|
1 Participants
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: All enrolled patients underwent resection, no patients were evaluated for PFS as per protocol
PFS is defined as the time from registration to the first of either disease progression or death from any cause, where disease progression is determined based on RECIST 1.1 criteria. Patients who do not experience disease progression or death while on protocol will be censored at the last disease assessment date. PFS will be estimated using the Kaplan-Meier method. Patients in the primary evaluable population who are not in the resectable evaluable population will be eligible for the analysis of this endpoint. The median PFS and corresponding 95% confidence interval (by Brookmeyer and Crowley) will be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 yearsOS is defined as the time from registration to death from any cause. Patients who are alive will be censored at the last follow-up date. OS will be estimated using the Kaplan-Meier method. Patients in the primary evaluable population will be eligible for the analysis of this endpoint.. The median OS and corresponding 95% confidence interval (by Brookmeyer and Crowley) will be reported.
Outcome measures
| Measure |
Treatment (Regorafenib, Durvalumab)
n=4 Participants
Patients receive regorafenib PO QD on days 1-21 and durvalumab IV on day 1. Treatment repeats every 28 days for a maximum of 2 years from registration or until decision to proceed to surgery, disease progression, excessive toxicity, or patient withdrawal.\> \> Durvalumab: Given IV\>
\> Regorafenib: Given PO
|
|---|---|
|
Overall Survival (OS)
|
NA months
The median and CI were not able to be estimated due to no death events occurring
|
SECONDARY outcome
Timeframe: 2 yearsRFS is defined as the time from surgical resection to first of either disease recurrence or death from any cause. Patients who do not experience disease recurrence or death while on protocol will be censored at the last disease assessment date. RFS will be estimated using the Kaplan-Meier method. The resectable evaluable population will be used for the analysis of this endpoint. The median RFS and corresponding 95% confidence interval (by Brookmeyer and Crowley) will be reported.
Outcome measures
| Measure |
Treatment (Regorafenib, Durvalumab)
n=4 Participants
Patients receive regorafenib PO QD on days 1-21 and durvalumab IV on day 1. Treatment repeats every 28 days for a maximum of 2 years from registration or until decision to proceed to surgery, disease progression, excessive toxicity, or patient withdrawal.\> \> Durvalumab: Given IV\>
\> Regorafenib: Given PO
|
|---|---|
|
Recurrence-free Survival (RFS)
|
12.2 months
Only one event occurred, and the remaining three patients were censored prior to that event. The limited number of observed events, in addition to the early censoring, precluded reliable estimation around the median using the Kaplan-Meier method. Therefore, the confidence interval for the median is reported as not estimable (NE) for both the upper and lower bound.
|
SECONDARY outcome
Timeframe: 10 monthsA pathologic complete response must satisfy all of the following: \*No metastatic disease (i.e. pM0 or pMx, pNo or pNx) \*Zero percent residual tumor cells in the specimen including the presumed prior tumor bed and elsewhere \*Negative resection margin on all margin examined. The proportion of patients experiencing pathologic complete response and corresponding 95% confidence interval will be reported.
Outcome measures
| Measure |
Treatment (Regorafenib, Durvalumab)
n=4 Participants
Patients receive regorafenib PO QD on days 1-21 and durvalumab IV on day 1. Treatment repeats every 28 days for a maximum of 2 years from registration or until decision to proceed to surgery, disease progression, excessive toxicity, or patient withdrawal.\> \> Durvalumab: Given IV\>
\> Regorafenib: Given PO
|
|---|---|
|
Pathologic Complete Response Rate
|
0.0 proportion of participants
Interval 0.0 to 0.6
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Analyze the effect of regorafenib and durvalumab on immune biomarkers in the tumor microenvironment and systemic circulation At baseline, week 8, 12, 20, and at off protocol treatmentOutcome measures
Outcome data not reported
Adverse Events
Treatment (Regorafenib, Durvalumab)
Serious adverse events
| Measure |
Treatment (Regorafenib, Durvalumab)
n=4 participants at risk
Regorafenib: Given PO
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
25.0%
1/4 • Number of events 1 • 2 years
|
Other adverse events
| Measure |
Treatment (Regorafenib, Durvalumab)
n=4 participants at risk
Regorafenib: Given PO
|
|---|---|
|
Skin and subcutaneous tissue disorders
Skin and subcut tissue disord - Oth spec
|
50.0%
2/4 • Number of events 2 • 2 years
|
|
Vascular disorders
Hypertension
|
75.0%
3/4 • Number of events 3 • 2 years
|
|
Gastrointestinal disorders
Constipation
|
50.0%
2/4 • Number of events 3 • 2 years
|
|
Gastrointestinal disorders
Dyspepsia
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
Nausea
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
Oral dysesthesia
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Gastrointestinal disorders
Oral pain
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
General disorders and administration site conditions
Fatigue
|
50.0%
2/4 • Number of events 2 • 2 years
|
|
General disorders and administration site conditions
Fever
|
25.0%
1/4 • Number of events 2 • 2 years
|
|
General disorders and administration site conditions
Pain
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Infections and infestations
Thrush
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Investigations
Alanine aminotransferase increased
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Investigations
Aspartate aminotransferase increased
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Investigations
Blood bilirubin increased
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Metabolism and nutrition disorders
Anorexia
|
50.0%
2/4 • Number of events 2 • 2 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Muscle cramp
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Nervous system disorders
Headache
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Psychiatric disorders
Anxiety
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Renal and urinary disorders
Urinary incontinence
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
25.0%
1/4 • Number of events 1 • 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place