Regorafenib Plus Pembrolizumab in Patients With Advanced or Spreading Liver Cancer Who Have Been Previously Treated With PD-1/PD-L1 Immune Checkpoint Inhibitors
CompletedPhase 2Results postedLast updated 13 May 2025
What this trial tests
Phase 2 trial testing Pembrolizumab in Hepatocellular Carcinoma in 95 participants. Completed in 23 April 2024.
18 and older, any sex, with Hepatocellular Carcinoma. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Overall Response Rate (ORR) Per RECIST 1.1 by Central AssessmentPrimary· Up to 15 months. Data up to 38 months are now available and are also reported for full transparency.
Overall response rate (ORR) is defined as the percentage of participants with best overall response of confirmed complete response (CR) or partial response (PR). ORR per RECIST 1.1 by independent central assessment is reported). RECIST 1.1: response evaluation criteria in solid tumors version 1.1
Up to 15 months (primary outcome as previously reported)
Cohort 2 (Regorafenib + Pembrolizumab [1L: Any Other IO Containing Treatment])
14.8
4.2 – 33.7
Overall Response Rate (ORR) Per RECIST 1.1 by Investigator AssessmentSecondary· Up to 38 months
Overall response rate (ORR) is defined as the percentage of participants with best overall response of confirmed complete response (CR) or partial response (PR). ORR by RECIST 1.1 investigator review is reported. RECIST 1.1: response evaluation criteria in solid tumors version 1.1
Cohort 2 (Regorafenib + Pembrolizumab [1L: Any Other IO Containing Treatment])
18.5
6.3 – 38.1
Duration of Response (DOR) Per RECIST 1.1 by Central Assessment and Investigator AssessmentSecondary· Up to 38 months
Duration of response (DOR) for partial response (PR) and complete response (CR) was defined as the time from the first documented objective response of PR or CR, whichever noted earlier, to disease progression or death (if death occurs before progression was documented). DOR was defined for confirmed responders only, i.e., participants with a CR or PR. RECIST 1.1: response evaluation criteria in solid tumors version 1.1
Cohort 2 (Regorafenib + Pembrolizumab [1L: Any Other IO Containing Treatment])
364
117 – 839
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Secondary· Up to 38 months
An AE was considered as treatment-emergent (TEAE) if arising or worsening after start of first study intervention administration until 30 days after administration of any study intervention. In addition, any AEs qualifying as a serious adverse event (SAE) were collected for 90 days after the last dose of pembrolizumab, unless a new anti-cancer therapy had been initiated.
Cohort 2 (Regorafenib + Pembrolizumab [1L: Any Other IO Containing Treatment])
11.1
Adverse events — posted to ClinicalTrials.gov
Time frame: These data extend to the final release date of the clinical database (38 months)..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Researchers are looking for a better way to treat people diagnosed with liver cancer which may have spread to nearby tissue and is unlikely to be cured or controlled with treatment (advanced metastatic hepatocellular carcinoma, HCC). Before a treatment can be approved for people to take, researchers do clinical trials to better understand its safety and how it works.
In this trial, the researchers will learn more about the trial treatment, regorafenib, in a small number of participants. They will study the results when the trial treatment is taken with another cancer treatment called pembrolizumab.
There will be 2 parts to this trial. The part 1 (pilot phase) will include about 52 men and women. The part 2 (expansion phase) will include about 67 men and women. All of the participants will have HCC and will be aged 18 years or older. All of the participants will have tried other treatments that did not help their HCC. These other treatments (PD-1/PD-L1 Immune Checkpoint Inhibitors) are designed to work by stopping the activity of certain proteins in the immune system thought to play a role in HCC.
During both parts of the trial, the participants will take regorafenib and receive pembrolizumab. In the pilot phase, there will be 2 groups of participants. The group that each participant joins will be based on the treatment they already received for their HCC. The researchers will review the results in each group to learn if regorafenib and pembrolizumab are helping one group of participants more than others. Outcome of this review will determine the population to be treated in the expansion phase.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07572123 — Evaluating the Addition of Maintenance Immunotherapy Compared to the Usual Treatment of Chemotherapy and Autologous Stem
· Phase 2, PHASE3
· not yet recruiting
NCT07275216 — Pembrolizumab in Combination With Chemotherapy for the Treatment of Frail Hodgkin Lymphoma Patients Ineligible for Stand
· Phase 2
· not yet recruiting
NCT07302347 — A Study of Pembrolizumab in Japanese Pediatric Participants With Solid Tumors or Lymphomas and Japanese Adult Participan
· Phase 1, PHASE2
· recruiting
NCT06724042 — Study of ISM5939 in Patients With Advanced and/or Metastatic Solid Tumors
· Phase 1
· not yet recruiting
NCT07383441 — Adding Biotherapy or Placebo to Standard Treatment for Advanced Kidney Cancer
· Phase 3
· not yet recruiting
Other recruiting trials for Hepatocellular Carcinoma
Currently open trials in the same condition.
NCT06902246 — Regorafenib and Yttrium-90 Radioembolization for Unresectable Hepatocellular Carcinoma
· Phase 2
· recruiting
NCT07417397 — Adjuvant TACE in HCC With High-risk Recurrence Factors
· Phase 3
· recruiting
NCT07317414 — β-alanine in the Treatment of Advanced Hepatocellular Carcinoma
· Phase 2
· recruiting
NCT07148050 — Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimer
· Phase 1
· recruiting
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Trials by the same sponsor.
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Bayer
Last refreshed: 13 May 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04696055.