REFINE: Regorafenib observational study in hepatocellular carcinoma

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  International, prospective, open-label, multicenter, observational study

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (L01XE21) regorafenib

  regorafenib
• Medical condition to be studied:
  Hepatocellular carcinoma

Population studied

• Short description of the study population:
  The study population included male and female patients diagnosed with unresectable hepatocellular carcinoma (uHCC) initiated treatment with regorafenib under routine clinical practice.; 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
• Age groups:
  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with unresectable hepatocellular carcinoma
• Estimated number of subjects:
  1000

Study design details

• Main study objective:
  To evaluate the safety of regorafenib in patients with uHCC, including incidences of all treatment-emergent adverse events (TEAEs) and dose modifications due to TEAEs in real-world practice conditions.
• Outcomes:
  Number of patients with treatment emergent adverse events (TEAEs) leading to dose modifications (including reductions, interruptions and permanent discontinuation), Overall survival (OS) Progression-free survival (PFS) Time to progression (TTP) Best overall tumor response (ORR) Duration of regorafenib treatment
• Data analysis plan:
  Statistical analyses will be of explorative and descriptive nature. The study is not aimed to confirm or reject pre-defined hypotheses. All variables will be analyzed descriptively with appropriate statistical methods: categorical variables by frequency tables (absolute and relative frequencies) and continuous variables by sample statistics (i.e. mean, standard deviation, minimum, median, quartiles, 5th and 95th percentiles, and maximum). Continuous variables will be described by absolute value and as change from baseline per analysis time point, if applicable.Patients who took at least one dose of regorafenib will be included in the safety analysis set (SAF).Patients who took at least one dose of regorafenib, did not violate a major inclusion/exclusion criterion, and had at least one follow-up assessment after receiving regorafenib will be included in the full analysis set (FAS). Safety data will be analyzed on the SAF, effectiveness data on the FAS.