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

Sponsor

Bayer (Industry)

Overall Status

Completed

CT.gov ID

NCT03289273

Collaborator

(none)

1,010

Enrollment

Locations

57.2

Actual Duration (Months)

27.3

Patients Per Site

0.5

Patients Per Site Per Month

Study Details

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	Phase
Liver Neoplasms	Drug: Regorafenib (Stivarga, BAY73-4506)

Study Design

Study Type:

Observational

Actual Enrollment :

1010 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

REFINE: Regorafenib Observational Study in Hepatocellular Carcinoma

Actual Study Start Date :

Sep 13, 2017

Actual Primary Completion Date :

Jan 31, 2022

Actual Study Completion Date :

Jun 21, 2022

Arms and Interventions

Arm	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

Arm

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

Number of patients with treatment emergent adverse events (TEAEs) leading to dose modifications (including reductions, interruptions and permanent discontinuation) [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

Overall survival (OS) [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
Progression-free survival (PFS) [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
Time to progression (TTP) [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
Best overall tumor response (ORR) [Up to 24 months]
Best overall tumor response (ORR) will be defined according to investigator-assessed data according to local standard
Duration of regorafenib treatment [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

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	Banner MD Anderson Cancer Center	Gilbert	Arizona	United States	85234
2	St. Joseph´s Hospital and Medical Center	Phoenix	Arizona	United States	85013
3	University of Florida Health	Gainesville	Florida	United States	32610
4	University of Louisville - Clinical Trials Unit	Louisville	Kentucky	United States	40202
5	University Medical Center	New Orleans	Louisiana	United States	70112
6	Mercy Medical Center	Baltimore	Maryland	United States	21202
7	University of Michigan	Ann Arbor	Michigan	United States	48109
8	Henry Ford Health System	Detroit	Michigan	United States	48202
9	Mayo Clinic	Rochester	Minnesota	United States	55905
10	Rutgers University	Newark	New Jersey	United States	07101-1709
11	Roswell Park Cancer Institute	Buffalo	New York	United States	14263
12	C.R.Wood Cancer Center, Glen Falls Hospital	Glens Falls	New York	United States	12801
13	University of Cincinatti	Cincinnati	Ohio	United States	45267
14	Corporal Michael J. Crescenz VA Medical Center	Philadelphia	Pennsylvania	United States	19104
15	Einstein Medical Center	Philadelphia	Pennsylvania	United States	19141
16	UT Southwestern Medical Center	Dallas	Texas	United States	75390
17	MD Anderson Cancer Center	Houston	Texas	United States	77030
18	Many locations	Multiple Locations		Argentina
19	Many locations	Multiple Locations		Austria
20	Many locations	Multiple Locations		Belgium
21	Many locations	Multiple Locations		Canada
22	Many locations	Multiple Locations		China
23	Many locations	Multiple Locations		Denmark
24	Many locations	Multiple Locations		Egypt
25	Many locations	Multiple Locations		France
26	Many locations	Multiple Locations		Greece
27	Many locations	Multiple Locations		Italy
28	Many locations	Multiple Locations		Japan
29	Many locations	Multiple Locations		Korea, Republic of
30	Many locations	Multiple Locations		Netherlands
31	Many locations	Multiple Locations		Russian Federation
32	Many locations	Multiple Locations		Saudi Arabia
33	Many locations	Multiple Locations		Spain
34	Many locations	Multiple Locations		Sweden
35	Many locations	Multiple Locations		Taiwan
36	Many locations	Multiple Locations		Thailand
37	Many locations	Multiple Locations		Turkey