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

REFINE: Regorafenib Observational Study in Hepatocellular Carcinoma

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

• Status: Completed
• Conditions: Liver Neoplasms
• Intervention / Treatment: Drug: Regorafenib (Stivarga, BAY73-4506)

• Study Type: Observational
• Enrollment (Actual): 1010

Contacts and Locations

Study Locations

• Argentina
  • Multiple Locations, Argentina
    • Many Locations
• Austria
  • Multiple Locations, Austria
    • Many Locations
• Belgium
  • Multiple Locations, Belgium
    • Many Locations
• Canada
  • Multiple Locations, Canada
    • Many Locations
• China
  • Multiple Locations, China
    • Many Locations
• Denmark
  • Multiple Locations, Denmark
    • Many Locations
• Egypt
  • Multiple Locations, Egypt
    • Many Locations
• France
  • Multiple Locations, France
    • Many Locations
• Greece
  • Multiple Locations, Greece
    • Many Locations
• Italy
  • Multiple Locations, Italy
    • Many Locations
• Japan
  • Multiple Locations, Japan
    • Many Locations
• Korea, Republic of
  • Multiple Locations, Korea, Republic of
    • Many Locations
• Netherlands
  • Multiple Locations, Netherlands
    • Many Locations
• Russian Federation
  • Multiple Locations, Russian Federation
    • Many Locations
• Saudi Arabia
  • Multiple Locations, Saudi Arabia
    • Many Locations
• Spain
  • Multiple Locations, Spain
    • Many Locations
• Sweden
  • Multiple Locations, Sweden
    • Many Locations
• Taiwan
  • Multiple Locations, Taiwan
    • Many Locations
• Thailand
  • Multiple Locations, Thailand
    • Many Locations
• Turkey
  • Multiple Locations, Turkey
    • Many Locations
• United States
  • Arizona
    • Gilbert, Arizona, United States, 85234
      • Banner MD Anderson Cancer Center
    • Phoenix, Arizona, United States, 85013
      • St. Joseph´s Hospital and Medical Center
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida Health
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • University of Louisville - Clinical Trials Unit
  • Louisiana
    • New Orleans, Louisiana, United States, 70112
      • University Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21202
      • Mercy Medical Center
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan
    • Detroit, Michigan, United States, 48202
      • Henry Ford Health System
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • New Jersey
    • Newark, New Jersey, United States, 07101-1709
      • Rutgers University
  • New York
    • Buffalo, New York, United States, 14263
      • Roswell Park Cancer Institute
    • Glens Falls, New York, United States, 12801
      • C.R.Wood Cancer Center, Glen Falls Hospital
  • Ohio
    • Cincinnati, Ohio, United States, 45267
      • University of Cincinatti
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19141
      • Einstein Medical Center
    • Philadelphia, Pennsylvania, United States, 19104
      • Corporal Michael J. Crescenz VA Medical Center
  • Texas
    • Dallas, Texas, United States, 75390
      • UT Southwestern Medical Center
    • Houston, Texas, United States, 77030
      • MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Source population of the study are patients with unresectable hepatocellular carcinoma. Patients with this condition are mostly managed at specialist centers and cancer hospitals. The participation of these specialized centers in the study is going to ensure the representativeness of the study population. Physicians will be asked to sample consecutive patients whenever possible to avoid selection bias and thus increase the likelihood of representativeness.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
uHCC patients treated with regorafenib; 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); As per the treating physicians discretion

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Bayer

Publications and helpful links

Helpful Links

• Click here to find information for studies related to Bayer products. To find this study enter the ClinicalTrials.gov identifier (NCT) number or Bayer Study Identifier (ID) in the search field.

Study record dates

Study Major Dates

• Study Start (Actual): September 13, 2017
• Primary Completion (Actual): January 31, 2022
• Study Completion (Actual): June 21, 2022

Study Registration Dates

• First Submitted: September 18, 2017
• First Submitted That Met QC Criteria: September 19, 2017
• First Posted (Actual): September 20, 2017

Study Record Updates

• Last Update Posted (Estimated): June 13, 2023
• Last Update Submitted That Met QC Criteria: June 12, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: Liver cancer; unresectable hepatocellular carcinoma; safety and effectiveness; real-world practice conditions; management and treatment of liver cancer
• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Adenocarcinoma; Neoplasms, Glandular and Epithelial; Digestive System Neoplasms; Liver Diseases; Carcinoma; Carcinoma, Hepatocellular; Liver Neoplasms
• Other Study ID Numbers: 19244; EUPAS20981 (Registry Identifier: EMA)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No