Liver NeoplasmsNCT03289273
Observational Study to Evaluate, Under Real-world Practice Conditions, the Safety and Effectiveness of Regorafenib in Patients Diagnosed With Unresectable Hepatocellular Carcinoma (uHCC)
An observational study of Liver Neoplasms, run by Bayer at 37 sites in 21 countries.
Trial summary
Observational study to evaluate, under real-world practice conditions, the safety and effectiveness of regorafenib in patients diagnosed with unresectable hepatocellular carcinoma (uHCC) Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Liver Neoplasms
- What is being tested?
- Regorafenib (Stivarga, BAY73-4506)
- How many people
- 1,010 participants
- Where
- 37 sitesin 21 countries
- Study dates
- 13 Sep 2017to 21 Jun 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- Patients with confirmed diagnosis of unresectable HCC
- Physician-initiated decision to treat with regorafenib (prior to study enrollment)
Who cannot join
- Participation in an investigational program with interventions outside of routine clinical practice
- Past treatment with regorafenib
Trial locations and contacts
37 sites in 21 countries.
37 sites in 21 countries
United States17 sites
University of Michigan
Ann Arbor, Michigan, 48109
Mercy Medical Center
Baltimore, Maryland, 21202
Roswell Park Cancer Institute
Buffalo, New York, 14263
University of Cincinatti
Cincinnati, Ohio, 45267
UT Southwestern Medical Center
Dallas, Texas, 75390
Henry Ford Health System
Detroit, Michigan, 48202
University of Florida Health
Gainesville, Florida, 32610
Banner MD Anderson Cancer Center
Gilbert, Arizona, 85234
C.R.Wood Cancer Center, Glen Falls Hospital
Glens Falls, New York, 12801
MD Anderson Cancer Center
Houston, Texas, 77030
University of Louisville - Clinical Trials Unit
Louisville, Kentucky, 40202
University Medical Center
New Orleans, Louisiana, 70112
Rutgers University
Newark, New Jersey, 07101-1709
Corporal Michael J. Crescenz VA Medical Center
Philadelphia, Pennsylvania, 19104
Einstein Medical Center
Philadelphia, Pennsylvania, 19141
St. Joseph´s Hospital and Medical Center
Phoenix, Arizona, 85013
Mayo Clinic
Rochester, Minnesota, 55905
About this study
Official title: 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)
- Conditions
- Liver Neoplasms
- Being tested
- Regorafenib (Stivarga, BAY73-4506)
- Keywords
- Liver cancer, unresectable hepatocellular carcinoma, real-world practice conditions, safety and effectiveness, management and treatment of liver cancer
- Medical subject headings
- Liver Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Regorafenib (Stivarga, BAY73-4506)Drug
As per the treating physicians discretion
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Drug: Regorafenib (Stivarga, BAY73-4506) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 24 months
Secondary outcomes (5)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2017
- Study start13 Sep 2017
- Primary completion31 Jan 2022
- Study completion21 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Liver NeoplasmsStudies of the same condition, open and under way.
- More trials run by BayerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,010 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03289273
- Sponsor's protocol ID
- 19244
- Other IDs
- EUPAS20981
- Status last verified
- Jun 2023
- First submitted
- 18 Sep 2017
- Last updated
- 13 Jun 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: 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.
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- REFINE: Regorafenib Observational Study in Hepatocellular Carcinoma
- ClinicalTrials.gov ID
- NCT03289273
- Acronym / sponsor ID
- 19244
- Conditions
- Liver Neoplasms
- Keywords
- Liver cancer, unresectable hepatocellular carcinoma, real-world practice conditions, safety and effectiveness, management and treatment of liver cancer
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,010 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 13 Sep 2017 (actual)
- Primary completion
- 31 Jan 2022 (actual)
- Study completion
- 21 Jun 2022 (actual)
- First posted
- 20 Sep 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Observational study to evaluate, under real-world practice conditions, the safety and effectiveness of regorafenib in patients diagnosed with unresectable hepatocellular carcinoma (uHCC)
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| uHCC patients treated with regorafenib | – | Drug: Regorafenib (Stivarga, BAY73-4506)Patients with a confirmed diagnosis of uHCC and for whom a decision to treat with regorafenib has been made (by the treating physician) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- Patients with confirmed diagnosis of unresectable HCC
- Physician-initiated decision to treat with regorafenib (prior to study enrollment)
Key exclusion criteria (2 listed)
- Participation in an investigational program with interventions outside of routine clinical practice
- Past treatment with regorafenib
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Regorafenib (Stivarga, BAY73-4506)As per the treating physicians discretion | Drug | uHCC patients treated with regorafenib |
Primary outcome
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.
5 secondary outcomes
Overall survival (OS)
Time frame: Up to 24 months
Progression-free survival (PFS)
Time frame: Up to 24 months
Time to progression (TTP)
Time frame: Up to 24 months
Best overall tumor response (ORR)
Time frame: Up to 24 months
Duration of regorafenib treatment
Time frame: Up to 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2023.
- Individual participant dataSharing planned: Undecided. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
37 sites in 21 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Liver Neoplasms, run by Bayer at 37 sites in 21 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, Argentina, Austria, Belgium, Canada and 16 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jun 2023.