Observational Study to Evaluate, Under Real-world Practice Conditions, the Safety and Effectiveness of Regorafenib in Patients Diagnosed With Unresectable Hepatocellular Carcinoma (uHCC)

Study Overview
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Brief Summary
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Observational study to evaluate, under real-world practice conditions, the safety and effectiveness of regorafenib in patients diagnosed with unresectable hepatocellular carcinoma (uHCC)

Official Title
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REFINE: Regorafenib Observational Study in Hepatocellular Carcinoma

Conditions
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Liver Neoplasms

Intervention / Treatment
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* Drug: Regorafenib (Stivarga, BAY73-4506)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with confirmed diagnosis of unresectable HCC Physician-initiated decision to treat with regorafenib (prior to study enrollment) Exclusion Criteria: Participation in an investigational program with interventions outside of routine clinical practice Past treatment with regorafenib
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| uHCC patients treated with regorafenib<br>Patients with a confirmed diagnosis of uHCC and for whom a decision to treat with regorafenib has been made (by the treating physician) | Drug: Regorafenib (Stivarga, BAY73-4506)<br>* As per the treating physicians discretion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of patients with treatment emergent adverse events (TEAEs) leading to dose modifications (including reductions, interruptions and permanent discontinuation) | The primary endpoint is the safety of regorafenib in patients with uHCC, defined as the frequency of documented TEAEs, including serious adverse events (SAEs). Safety will be assessed in all patients who receive at least one dose of regorafenib regardless of prior treatment. | Up to 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival (OS) | Overall survival (OS) is defined as the time (days) from the start of regorafenib treatment to the date of death, due to any reason. Patients alive or lost to follow-up at the time of analysis will be censored at their last date of follow-up | Up to 24 months | 
| Progression-free survival (PFS) | Progression-free survival (PFS) is defined as the time (days) from the start of regorafenib treatment to the date of first observed disease progression (radiological or clinical, whichever is earlier) or death due to any cause, if death occurs before progression is documented | Up to 24 months | 
| Time to progression (TTP) | Time to progression (TTP) is defined as the time (days) from the start of regorafenib treatment to the first documented disease progression | Up to 24 months | 
| Best overall tumor response (ORR) | Best overall tumor response (ORR) will be defined according to investigator-assessed data according to local standard | Up to 24 months | 
| Duration of regorafenib treatment | Duration of regorafenib treatment, defined by the time interval from the start of regorafenib treatment to the day of permanent discontinuation of regorafenib (including death) | Up to 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Liver cancer, unresectable hepatocellular carcinoma, real-world practice conditions, safety and effectiveness, management and treatment of liver cancer