REFINE: Regorafenib observational study in hepatocellular carcinoma

Study Objective

The purpose of this observational study is to evaluate, in real-world practice conditions, the safety and effectiveness of regorafenib in patients with unresectable hepatocellular carcinoma (uHCC) for whom a decision to treat with regorafenib has been made before study enrollment. The study will also evaluate regorafenib treatment in a variety of HCC patient subsets, such as grouping by Child–Pugh score regionally and globally, that were not addressed in the

RESORCE study, as well as provide information on treatment patterns and outcomes for patients with uHCC in the real-world setting.

The primary objective of this study:

is to evaluate the safety of regorafenib in patients with uHCC, including incidence of all treatment-emergent adverse events (TEAEs) and dose modifications due to TEAEs in real-world practice conditions.

Secondary objectives are:

• To describe the effectiveness of regorafenib, including overall survival (OS), progression-free survival (PFS), time to progression (TTP), and overall tumor response (ORR)

• To describe the patterns of regorafenib treatment, including actual doses, duration of treatment (DOT), and other dosing parameters

• To describe patient characteristics, comorbidities, and their influence on treatment and outcomes under real-world conditions

• To describe physicians’ practice patterns, including regional difference in management of and treatments for uHCC in real-world conditions

Tertiary objectives are:

• To describe sorafenib treatment prior to regorafenib treatment including doses, duration of treatment, best response, and reasons for discontinuation (in relevant patients)

• To describe other therapies for HCC prior to regorafenib, including doses, duration of treatment, best response, and reasons for discontinuation (in relevant patients)

Participation conditions

Inclusion criteria

• Patients with confirmed diagnosis of unresectable HCC

• Physician-initiated decision to treat with regorafenib (prior to study enrollment)

• Signed informed consent form

Exclusion criteria

• Participation in an investigational program with interventions outside of routine clinical practice.

• Past treatment with regorafenib

CHL, Dr. Catherine Herremans