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

• ClinicalTrials.gov Identifier: NCT03289273
• Recruitment Status: Recruiting
• First Posted: September 20, 2017
• Last Update Posted: August 27, 2019
• Sponsor: Bayer
• Information provided by (Responsible Party): Bayer

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment
Liver Neoplasms	Drug: Regorafenib (Stivarga, BAY73-4506)

Study Design

• Study Type: Observational
• Estimated Enrollment: 1000 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: REFINE: Regorafenib Observational Study in Hepatocellular Carcinoma
• Actual Study Start Date: September 13, 2017
• Estimated Primary Completion Date: July 30, 2022
• Estimated Study Completion Date: October 1, 2022

Groups and Cohorts

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

Outcome Measures

Primary Outcome Measures :

1. Number of patients with treatment emergent adverse events (TEAEs) leading to dose modifications (including reductions, interruptions and permanent discontinuation) [ Time Frame: Up to 24 months ]

   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.

Secondary Outcome Measures :

1. Overall survival (OS) [ Time Frame: Up to 24 months ]
   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
2. Progression-free survival (PFS) [ Time Frame: Up to 24 months ]
   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
3. Time to progression (TTP) [ Time Frame: Up to 24 months ]
   Time to progression (TTP) is defined as the time (days) from the start of regorafenib treatment to the first documented disease progression
4. Best overall tumor response (ORR) [ Time Frame: Up to 24 months ]
   Best overall tumor response (ORR) will be defined according to investigator-assessed data according to local standard
5. Duration of regorafenib treatment [ Time Frame: Up to 24 months ]
   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)

Eligibility Criteria

• Ages Eligible for Study: Child, Adult, Older Adult
• Sexes Eligible for Study: All
• 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.

Criteria

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

Contacts and Locations

Contacts

Contact: Bayer Clinical Trials Contact; (+) 1-888-8422937; clinical-trials-contact@bayer.com

  Show 56 Study Locations

Sponsors and Collaborators

Bayer

More Information

• Responsible Party: Bayer
• ClinicalTrials.gov Identifier: NCT03289273 History of Changes
• Other Study ID Numbers: 19244; EUPAS20981 ( Registry Identifier: EMA )
• First Posted: September 20, 2017
• Last Update Posted: August 27, 2019
• Last Verified: August 2019

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Bayer:

Liver cancer; unresectable hepatocellular carcinoma; real-world practice conditions; safety and effectiveness; management and treatment of liver cancer

Additional relevant MeSH terms:

Carcinoma; Carcinoma, Hepatocellular; Liver Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Adenocarcinoma; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases