NCT01774344

Brief Summary

The objective of this study was to evaluate efficacy and safety of regorafenib in patients with advanced liver cancer who had progressed after sorafenib treatment. Patients were treated with regorafenib or placebo using a 2:1 randomization scheme.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
573

participants targeted

Target at P75+ for phase_3

Timeline
Completed

Started May 2013

Longer than P75 for phase_3

Geographic Reach
20 countries

144 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 21, 2013

Completed
3 days until next milestone

First Posted

Study publicly available on registry

January 24, 2013

Completed
4 months until next milestone

Study Start

First participant enrolled

May 14, 2013

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 29, 2016

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

June 5, 2017

Completed
2.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 5, 2019

Completed
Last Updated

August 20, 2020

Status Verified

August 1, 2020

Enrollment Period

2.8 years

First QC Date

January 21, 2013

Results QC Date

February 9, 2017

Last Update Submit

August 11, 2020

Conditions

Keywords

RegorafenibBAY73-4506

Outcome Measures

Primary Outcomes (1)

  • Overall Survival (OS)

    Overall Survival (OS) was defined as the time from date of randomization (Day 1) to death due to any cause. Subjects still alive at the time of analysis were censored at their last date of last contact.

    From randomization (Day 1) of the first subject until 419 days later

Secondary Outcomes (4)

  • Time to Progression (TTP)

    From date of randomization until 30 days after last study treatment (assessed every 6 weeks until PD; and after 8 cycle assessed every 12 weeks) (approximately 33 months)

  • Progression Free Survival (PFS)

    From date of randomization until 30 days after last study treatment (assessed every 6 weeks until PD; and after 8 cycle assessed every 12 weeks)

  • Objective Tumor Response Rate (ORR)

    From date of randomization until 30 days after last study treatment (assessed every 6 weeks until PD; and after 8 cycle assessed every 12 weeks) (approximately 33 months)

  • Disease Control Rate (DCR)

    From date of randomization until 30 days after last study treatment (assessed every 6 weeks until PD; and after 8 cycle assessed every 12 weeks) (approximately 33 months)

Other Outcomes (1)

  • Overall Survival (OS)

    From randomization (Day 1) of the first subject to end of follow upto 1710 days

Study Arms (2)

Regorafenib

EXPERIMENTAL

160 mg orally (p.o.) every day (qd) for 3 weeks of every 4 week cycle (i.e. 3 weeks on, 1 week off) plus BSC (Best Supportive Care)

Drug: Regorafenib (Stivarga, BAY73-4506)

Placebo

PLACEBO COMPARATOR

4 matching placebo tablets for 3 weeks of every 4 week cycle (i.e. 3 weeks on, 1 week off) plus BSC

Drug: Placebo

Interventions

Regorafenib, 40 mg tablets

Regorafenib

Placebo tablets matching in appearance

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Histological or cytological confirmation of HCC (hepatocellular carcinoma) or non-invasive diagnosis of HCC as per American Association for the Study of Liver Diseases criteria in patients with a confirmed diagnosis of cirrhosis
  • Barcelona Clinic Liver Cancer stage Category B or C that cannot benefit from treatments of established efficacy with higher priority such as resection, local ablation, chemoembolization or systemic sorafenib.
  • Failure to prior treatment with sorafenib (defined as documented radiological progression according to the radiology charter). Randomization needs to be performed within 10 weeks after the last treatment with sorafenib.
  • Tolerability of prior treatment with sorafenib defined as not less than 20 days at a minimum daily dose of 400 mg QD within the last 28 days prior to withdrawal.
  • Liver function status Child-Pugh Class A. Child Pugh status should be calculated based on clinical findings and laboratory results during the screening period.
  • Local or loco-regional therapy of intrahepatic tumor lesions (e.g. surgery, radiation therapy, hepatic arterial embolization, chemoembolization, radiofrequency ablation, percutaneous ethanol injection, or cryoablation) must have been completed \>/=4 weeks before first dose of study medication. Note: patients who received sole intrahepatic intraarterial chemotherapy, without lipiodol or embolizing agents are not eligible.
  • Eastern Cooperative Oncology Group Performance Status of 0 or 1.
  • Adequate bone marrow, liver and renal function as assessed by the following laboratory tests conducted within 7 days before randomization.
  • Glomerular filtration rate \>/= 30 ml/min/1.73 m\^2 according to the Modification of diet in renal disease study equation.
  • At least one uni-dimensional measurable lesion by computed tomography (CT) scan or magnetic resonance imaging (MRI) according to RECIST (RECIST version 1.1), and modified RECIST for HCC. Tumor lesions situated in a previously irradiated area, or in an area subjected to other loco-regional therapy, may be considered measurable if there has been demonstrated progression in the lesion.
  • Life expectancy of at least 3 months.
  • Women of childbearing potential and men must agree to use adequate contraception .

You may not qualify if:

  • Sorafenib treatment within 2 weeks of randomization.
  • Prior systemic treatment for HCC, except sorafenib.
  • Permanent discontinuation of prior sorafenib therapy due to sorafenib related toxicity.
  • Known history or symptomatic metastatic brain or meningeal tumors (head CT or MRI at screening to confirm the absence of central nervous system \[CNS\] disease if patient has symptoms suggestive or consistent with CNS disease).
  • Uncontrolled hypertension (systolic blood pressure \[BP\] \> 150 mmHg or diastolic pressure \> 90 mmHg despite optimal medical management).
  • Uncontrolled ascites (defined as not easily controlled with diuretic or paracentesis treatment).
  • Ongoing infection \> Grade 2 according to NCI-CTCAE (National Cancer Institute - Common Terminology Criteria for Adverse Events) v. 4.0. Hepatitis B is allowed if no active replication is present. Hepatitis C is allowed if no antiviral treatment is required.
  • Clinically significant bleeding NCI-CTCAE version 4.0 Grade 3 or higher within 30 days before randomization.
  • Arterial or venous thrombotic or embolic events such as cerebrovascular accident (including transient ischemic attacks), deep vein thrombosis or pulmonary embolism within 6 months before the start of study medication.
  • Patients unable to swallow oral medications.
  • Interstitial lung disease with ongoing signs and symptoms at the time of screening.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (147)

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Los Angeles, California, 90048, United States

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Los Angeles, California, 90095, United States

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Orange, California, 92868-3201, United States

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Aurora, Colorado, 80045, United States

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Washington D.C., District of Columbia, 20007-2197, United States

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Gainesville, Florida, 32610-0286, United States

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Tampa, Florida, 33606, United States

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Chicago, Illinois, 60637, United States

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Louisville, Kentucky, 40202, United States

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Worcester, Massachusetts, 01655, United States

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Minneapolis, Minnesota, 55455, United States

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St Louis, Missouri, 63110, United States

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New York, New York, 10029, United States

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Rochester, New York, 14642, United States

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Pittsburgh, Pennsylvania, 15232, United States

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Richmond, Virginia, 23249, United States

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Seattle, Washington, 98101, United States

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Pilar, Buenos Aires, B1629ODT, Argentina

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Camperdown, New South Wales, 2050, Australia

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Liverpool, New South Wales, 2170, Australia

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Herston, Queensland, 4029, Australia

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Clayton, Victoria, 3168, Australia

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Box Hill, 3128, Australia

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Graz, Styria, 8036, Austria

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Linz, Upper Austria, 4020, Austria

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Vienna, 1090, Austria

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Bruxelles - Brussel, 1090, Belgium

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La Louvière, 7100, Belgium

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Salvador, Estado de Bahia, 41950-610, Brazil

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São Paulo, 05403-000, Brazil

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Hefei, Anhui, 230022, China

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Fuzhou, Fujian, 350025, China

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Guangzhou, Guangdong, 510060, China

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Guangzhou, Guangdong, 510080, China

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Guangzhou, Guangdong, 510515, China

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Nanning, Guangxi, 530021, China

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Harbin, Heilongjiang, 150081, China

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Wuhan, Hubei, 430030, China

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Changsha, Hunan, 410013, China

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Nanjing, Jiangsu, 210002, China

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Suzhou, Jiangsu, 215006, China

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Dalian, Liaoning, 116011, China

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Xi'an, Shaanxi, 710032, China

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Xi'an, Shaanxi, 710038, China

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Xi'an, Shaanxi, 710061, China

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Chengdu, Sichuan, 610041, China

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Beijing, 100039, China

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Beijing, 100069, China

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Beijing, 100071, China

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Beijing, 100142, China

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Chongqing, 400038, China

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Shanghai, 200001, China

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Shanghai, 200032, China

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Shanghai, 201805, China

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Tianjin, 300060, China

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Hradec Králové, 500 05, Czechia

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Olomouc, 775 20, Czechia

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Prague, 128 08, Czechia

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Angers, 49100, France

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Caen, F-14033, France

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Clichy, 92110, France

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Créteil, 94010, France

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Dijon, 21000, France

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La Tronche, 38700, France

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Lille, 59037, France

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Lyon, 69004, France

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Marseille, 13005, France

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Montpellier, 34295, France

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Nice, 06202, France

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Paris, 75020, France

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Paris, 75651, France

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Perpignan, 66000, France

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Reims, 51092, France

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Rennes, 35062, France

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Toulouse, 31059, France

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Vandœuvre-lès-Nancy, 54500, France

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Villejuif, 94805, France

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Heidelberg, Baden-Wurttemberg, 69120, Germany

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München, Bavaria, 81377, Germany

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Frankfurt am Main, Hesse, 60590, Germany

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Hanover, Lower Saxony, 30625, Germany

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Aachen, North Rhine-Westphalia, 52074, Germany

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Cologne, North Rhine-Westphalia, 50937, Germany

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Essen, North Rhine-Westphalia, 45136, Germany

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Mainz, Rhineland-Palatinate, 55131, Germany

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Magdeburg, Saxony-Anhalt, 39120, Germany

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Berlin, 13353, Germany

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Hamburg, 20246, Germany

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Budapest, 1097, Hungary

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Debrecen, 4032, Hungary

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Kaposvár, 7400, Hungary

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Foggia, Apulia, 71013, Italy

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Napoli, Campania, 80131, Italy

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Bologna, Emilia-Romagna, 40138, Italy

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Modena, Emilia-Romagna, 41124, Italy

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Rome, Lazio, 00168, Italy

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Genoa, Liguria, 16132, Italy

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Bergamo, Lombardy, 24127, Italy

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Milan, Lombardy, 20089, Italy

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Milan, Lombardy, 20122, Italy

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Novara, Piedmont, 28100, Italy

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Turin, Piedmont, 10126, Italy

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Cagliari, Sardinia, 09134, Italy

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Pisa, Tuscany, 56126, Italy

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Padua, Veneto, 35128, Italy

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Chiba, Chiba, 260-8677, Japan

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Kashiwa-shi, Chiba, 277-8577, Japan

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Fukuoka, Fukuoka, 810-8563, Japan

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Iizuka-shi, Fukuoka, 820-8505, Japan

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Yokohama, Kanagawa, 232-0024, Japan

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Kumamoto, Kumamoto, 860-8556, Japan

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Osaka, Osaka, 541-8567, Japan

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Osakasayama-shi, Osaka, 589-8511, Japan

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Utsunomiya, Tochigi, 321-0974, Japan

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Chiyoda-ku, Tokyo, 101-0062, Japan

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Chuo-ku, Tokyo, 104-0045, Japan

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Musashino-shi, Tokyo, 180-8610, Japan

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Amsterdam, 1105 AZ, Netherlands

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Leiden, 2333 ZA, Netherlands

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Barnaul, 656045, Russia

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Moscow, 119991, Russia

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Moscow, Russia

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Singapore, 119228, Singapore

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Busan, Busan Gwang''yeogsi, 49241, South Korea

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Daegu, 700-721, South Korea

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Seoul, 06351, South Korea

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Seoul, 110-744, South Korea

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Oviedo, Principality of Asturias, 33011, Spain

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Alicante, 03010, Spain

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Barcelona, 08035, Spain

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Barcelona, 08036, Spain

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Córdoba, 14004, Spain

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Madrid, 28007, Spain

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Madrid, 28041, Spain

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Madrid, 28050, Spain

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Zaragoza, 50009, Spain

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Bellinzona, Canton Ticino, 6500, Switzerland

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Bern, 3010, Switzerland

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Kaohsiung City, Kaohsiung, 83301, Taiwan

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Taipei, 10016, Taiwan

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Taipei, 11217, Taiwan

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Taoyuan District, 333, Taiwan

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Birmingham, West Midlands, B15 2WB, United Kingdom

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Leeds, West Yorkshire, LS9 7TF, United Kingdom

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Bristol, BS2 8ED, United Kingdom

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London, SE5 9RS, United Kingdom

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London, W12 0HS, United Kingdom

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Related Publications (4)

  • Finn RS, Merle P, Granito A, Huang YH, Bodoky G, Pracht M, Yokosuka O, Rosmorduc O, Gerolami R, Caparello C, Cabrera R, Chang C, Sun W, LeBerre MA, Baumhauer A, Meinhardt G, Bruix J. Outcomes of sequential treatment with sorafenib followed by regorafenib for HCC: Additional analyses from the phase III RESORCE trial. J Hepatol. 2018 Aug;69(2):353-358. doi: 10.1016/j.jhep.2018.04.010. Epub 2018 Apr 26.

  • Bruix J, Qin S, Merle P, Granito A, Huang YH, Bodoky G, Pracht M, Yokosuka O, Rosmorduc O, Breder V, Gerolami R, Masi G, Ross PJ, Song T, Bronowicki JP, Ollivier-Hourmand I, Kudo M, Cheng AL, Llovet JM, Finn RS, LeBerre MA, Baumhauer A, Meinhardt G, Han G; RESORCE Investigators. Regorafenib for patients with hepatocellular carcinoma who progressed on sorafenib treatment (RESORCE): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2017 Jan 7;389(10064):56-66. doi: 10.1016/S0140-6736(16)32453-9. Epub 2016 Dec 6.

  • Wu W, Mao H, Song J, Yang F. Bibliometric analysis of hepatocellular carcinoma and tyrosine kinase inhibitors. Medicine (Baltimore). 2025 May 16;104(20):e42015. doi: 10.1097/MD.0000000000042015.

  • Teufel M, Seidel H, Kochert K, Meinhardt G, Finn RS, Llovet JM, Bruix J. Biomarkers Associated With Response to Regorafenib in Patients With Hepatocellular Carcinoma. Gastroenterology. 2019 May;156(6):1731-1741. doi: 10.1053/j.gastro.2019.01.261. Epub 2019 Feb 6.

Related Links

MeSH Terms

Conditions

Carcinoma, Hepatocellular

Interventions

regorafenib

Condition Hierarchy (Ancestors)

AdenocarcinomaCarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeNeoplasmsLiver NeoplasmsDigestive System NeoplasmsNeoplasms by SiteDigestive System DiseasesLiver Diseases

Limitations and Caveats

Decimal places were automatically truncated if last decimal equals zero.

Results Point of Contact

Title
Therapeutic Area Head
Organization
Bayer AG

Study Officials

  • Bayer Study Director

    Bayer

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 21, 2013

First Posted

January 24, 2013

Study Start

May 14, 2013

Primary Completion

February 29, 2016

Study Completion

July 5, 2019

Last Updated

August 20, 2020

Results First Posted

June 5, 2017

Record last verified: 2020-08

Locations