Global Study Looking at the Combination of RAD001 Plus Best Supportive Care (BSC) and Placebo Plus BSC to Treat Patients With Advanced Hepatocellular Carcinoma.

NCT ID: NCT01035229

Last Updated: 2016-09-22

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

546 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-04-30

Study Completion Date

2013-10-31

Brief Summary

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The purpose of this study is to compare treatment with RAD001 plus best supportive care (BSC) to placebo plus BSC in patients with advanced HCC whose disease progressed while on or after sorafenib treatment or who are intolerant to sorafenib.

Detailed Description

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Conditions

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Carcinoma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Everolimus + Best Supportice Care (BSC)

Patients were assigned to the Everolimus + BSC arm in a ratio of 2:1 over the Placebo arm. Everolimus was taken as a daily oral dose of 7.5 mg and was defined as the investigational drug. In addition to taking Everolimus, all patients also received BSC as per normal local practice.

Group Type EXPERIMENTAL

Everolimus

Intervention Type DRUG

Everolimus (labeled as RAD001) was formulated as tablets of 2.5 mg strength and blisterpacked in units of 10 tablets.

Best Supportive Care (BSC)

Intervention Type OTHER

BSC was defined as drug or non-drug therapies, nutritional support, physical therapy or anything that the Investigator believed to be in the patient's best interest, but excluding other antineoplastic treatments. BSC administered to the patient throughout the study was to be reported on the Concomitant Medication/Significant Non-Drug Therapy electronic case report from (eCRF). Permitted BSC treatments during the study included, but were not limited to, the following: Pain medication to allow the patient to be as comfortable as possible, Bisphosphonates for bone metastases, Localized radiotherapy, for the treatment of pre-existing, painful bone metastases, Nutritional support or appetite stimulants (i.e. megestrol) as recommended by the Investigator, Oxygen therapy and blood products or transfusions

Placebo + Best Supportive Care

Placebo Everolimus was taken as a daily oral dose of 7.5 mg and was defined as the control drug. In addition to taking Placeb Everolimus, all patients also received BSC as per normal local practice.

Group Type PLACEBO_COMPARATOR

Everolimus Placebo

Intervention Type DRUG

Everolimus Placebo matched to the everolimus 2.5 mg tablet strength was blister-packed in units of 10 tablets. Matching placebo tablets were formulated to be indistinguishable from the everolimus tablets. Everolimus placebo was taken as a daily oral dose of 7.5 mg and was defined as the control drug.

Best Supportive Care (BSC)

Intervention Type OTHER

BSC was defined as drug or non-drug therapies, nutritional support, physical therapy or anything that the Investigator believed to be in the patient's best interest, but excluding other antineoplastic treatments. BSC administered to the patient throughout the study was to be reported on the Concomitant Medication/Significant Non-Drug Therapy electronic case report from (eCRF). Permitted BSC treatments during the study included, but were not limited to, the following: Pain medication to allow the patient to be as comfortable as possible, Bisphosphonates for bone metastases, Localized radiotherapy, for the treatment of pre-existing, painful bone metastases, Nutritional support or appetite stimulants (i.e. megestrol) as recommended by the Investigator, Oxygen therapy and blood products or transfusions

Interventions

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Everolimus

Everolimus (labeled as RAD001) was formulated as tablets of 2.5 mg strength and blisterpacked in units of 10 tablets.

Intervention Type DRUG

Everolimus Placebo

Everolimus Placebo matched to the everolimus 2.5 mg tablet strength was blister-packed in units of 10 tablets. Matching placebo tablets were formulated to be indistinguishable from the everolimus tablets. Everolimus placebo was taken as a daily oral dose of 7.5 mg and was defined as the control drug.

Intervention Type DRUG

Best Supportive Care (BSC)

BSC was defined as drug or non-drug therapies, nutritional support, physical therapy or anything that the Investigator believed to be in the patient's best interest, but excluding other antineoplastic treatments. BSC administered to the patient throughout the study was to be reported on the Concomitant Medication/Significant Non-Drug Therapy electronic case report from (eCRF). Permitted BSC treatments during the study included, but were not limited to, the following: Pain medication to allow the patient to be as comfortable as possible, Bisphosphonates for bone metastases, Localized radiotherapy, for the treatment of pre-existing, painful bone metastases, Nutritional support or appetite stimulants (i.e. megestrol) as recommended by the Investigator, Oxygen therapy and blood products or transfusions

Intervention Type OTHER

Other Intervention Names

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RAD001

Eligibility Criteria

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Inclusion Criteria

* Advanced liver cancer
* Prior systemic treatment with sorafenib for advanced HCC and for whom their disease progressed during or after sorafenib treatment, or were intolerant to sorafenib treatment. Specifically, this can be defined as:

* Documented radiological confirmation (radiology scans or report) of disease progression during or after sorafenib treatment
* Intolerance to sorafenib (at any dose and/or duration) is defined as documented sorafenib-related grade 3 or 4 adverse events that led to sorafenib discontinuation.

NOTE:

* Sorafenib must be the last antineoplastic treatment before randomization
* Prior local and/or hormonal therapy (e.g., tamoxifen) before sorafenib is allowed
* One systemic chemotherapy regimen for advanced HCC is allowed before sorafenib treatment

* ECOG performance status of ≤ 2
* Child-Pugh A

Exclusion Criteria

* Active bleeding during the last 28 days
* Prior therapy with mTOR inhibitors
* Prior liver or other organ transplantation which mandates systemic immunosuppression
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Highlands Oncology Group Dept of Highlands Oncology Grp

Fayetteville, Arkansas, United States

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Compassionate Cancer Care Medical Group CCCMG

Fountain Valley, California, United States

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University of California San Diego - Moores Cancer Center SC - 3

La Jolla, California, United States

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California Pacific Medical Center California Pacific Med

San Francisco, California, United States

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Rocky Mountain Cancer Centers RMCC - Denver-Midtown (4)

Greenwood Village, Colorado, United States

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Queen's Medical Center Queens Cancer Center

Honolulu, Hawaii, United States

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The Sidney Kimmel Cancer Center at Johns Hopkins Hospital Dept. of SKCC @ JHU

Baltimore, Maryland, United States

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Massachusetts General Hospital Dept. of Mass General Hospital

Boston, Massachusetts, United States

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Midwest Cancer Care Physicians Research Medical Center

Kansas City, Missouri, United States

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VA Sierra Nevada Health Care System Dept. of VA Sierra Nevada HCS

Reno, Nevada, United States

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University of Rochester Medical Center Rochester

Rochester, New York, United States

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Northwest Cancer Specialists Rose Quarter Cancer Center

Portland, Oregon, United States

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St. Luke's Hospital and Health Network St. Luke's Cancer Network (2)

Bethlehem, Pennsylvania, United States

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Texas Cancer Center - Abilene

Abilene, Texas, United States

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Methodist Charlton Cancer Center Methodist

Dallas, Texas, United States

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University of Texas Southwestern Medical Center DeptofSimmons Cancer Center(3)

Dallas, Texas, United States

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Cancer Care Centers of South Texas / HOAST CCC of So. TX- San Antonio

San Antonio, Texas, United States

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Blue Ridge Research Center at Roanoke Neurological Center Blue Ridge Cancer Care

Roanoke, Virginia, United States

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University of Washington Cancer Care SC

Seattle, Washington, United States

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Novartis Investigative Site

Camperdown, New South Wales, Australia

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Kogarah, New South Wales, Australia

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Westmead, New South Wales, Australia

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Heidelberg, Victoria, Australia

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Parkville, Victoria, Australia

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Graz, , Austria

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Innsbruck, , Austria

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Vienna, , Austria

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Brussels, , Belgium

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Brussels, , Belgium

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Edegem, , Belgium

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Leuven, , Belgium

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London, Ontario, Canada

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Toronto, Ontario, Canada

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Montreal, Quebec, Canada

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Montreal, Quebec, Canada

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Nanjing, Jiangsu, China

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Xi’an, Shanxi, China

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Chengdu, Sichuan, China

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Hangzhou, Zhejiang, China

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Beijing, , China

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Guangzhou, , China

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Amiens, , France

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Avignon, , France

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Bordeaux, , France

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Caen Cedex9, , France

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Chambray-lès-Tours, , France

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Clermont-Ferrand, , France

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Clichy, , France

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Dijon, , France

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Lille, , France

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Lyon, , France

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Marseille Cédex 5, , France

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Montpellier, , France

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Nantes, , France

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Nice, , France

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Reims, , France

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Rouen, , France

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Saint-Priest-en-Jarez, , France

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Strasbourg, , France

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Mannheim, Baden-Wurttemberg, Germany

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Berlin, , Germany

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Essen, , Germany

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Esslingen am Neckar, , Germany

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Frankfurt, , Germany

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Göttingen, , Germany

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Hamburg, , Germany

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Hanover, , Germany

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Leipzig, , Germany

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München, , Germany

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Würzburg, , Germany

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Thessaloniki, Greece, Greece

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Athens, GR, Greece

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Larissa, GR, Greece

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Hong Kong, , Hong Kong

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Budapest, , Hungary

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Debrecen, , Hungary

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Szeged, , Hungary

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Szombathely, , Hungary

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Petah Tikva, , Israel

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Ramat Gan, , Israel

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Benevento, BN, Italy

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Bologna, BO, Italy

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Foggia, FG, Italy

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Milan, MI, Italy

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Rozzano, MI, Italy

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Modena, MO, Italy

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Palermo, PA, Italy

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Padua, PD, Italy

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Aviano, PN, Italy

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Pavia, PV, Italy

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Roma, RM, Italy

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Roma, RM, Italy

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Frattamaggiore, , Italy

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Napoli, , Italy

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Nagoya, Aichi-ken, Japan

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Chiba, Chiba, Japan

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Kashiwa, Chiba, Japan

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Matsuyama, Ehime, Japan

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Fukuoka, Fukuoka, Japan

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Fukuoka, Fukuoka, Japan

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Iizuka, Fukuoka, Japan

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Gifu, Gifu, Japan

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Ōgaki, Gifu, Japan

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Kanazawa, Ishikawa-ken, Japan

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Yokohama, Kanagawa, Japan

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Kumamoto, Kumamoto, Japan

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Sendai, Miyagi, Japan

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Osaka, Osaka, Japan

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Sayama, Osaka, Japan

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Chuo-ku, Tokyo, Japan

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Mitaka, Tokyo, Japan

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Shinagawa-ku, Tokyo, Japan

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Seoul, Korea, South Korea

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Seoul, Korea, South Korea

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Seoul, Korea, South Korea

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Seoul, Korea, South Korea

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Córdoba, Andalusia, Spain

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Sabadell, Barcelona, Spain

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Barcelona, Catalonia, Spain

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Barcelona, Catalonia, Spain

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Madrid, Madrid, Spain

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Pamplona, Navarre, Spain

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Niaosong Township, Taiwan, Taiwan

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Taichung, Taiwan, Taiwan

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Tainan City, Taiwan, Taiwan

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Taipei, Taiwan, Taiwan

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Taipei, Taiwan, ROC, Taiwan

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Linkou District, , Taiwan

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Liouying Township, , Taiwan

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Bangkok, , Thailand

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Bangkok, , Thailand

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Countries

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United States Australia Austria Belgium Canada China France Germany Greece Hong Kong Hungary Israel Italy Japan South Korea Spain Taiwan Thailand

References

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Zhu AX, Chen D, He W, Kanai M, Voi M, Chen LT, Daniele B, Furuse J, Kang YK, Poon RT, Vogel A, Chiang DY. Integrative biomarker analyses indicate etiological variations in hepatocellular carcinoma. J Hepatol. 2016 Aug;65(2):296-304. doi: 10.1016/j.jhep.2016.04.015. Epub 2016 Apr 27.

Reference Type DERIVED
PMID: 27130844 (View on PubMed)

Zhu AX, Kudo M, Assenat E, Cattan S, Kang YK, Lim HY, Poon RT, Blanc JF, Vogel A, Chen CL, Dorval E, Peck-Radosavljevic M, Santoro A, Daniele B, Furuse J, Jappe A, Perraud K, Anak O, Sellami DB, Chen LT. Effect of everolimus on survival in advanced hepatocellular carcinoma after failure of sorafenib: the EVOLVE-1 randomized clinical trial. JAMA. 2014 Jul 2;312(1):57-67. doi: 10.1001/jama.2014.7189.

Reference Type DERIVED
PMID: 25058218 (View on PubMed)

Related Links

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Other Identifiers

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2009-010196-25

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CRAD001O2301

Identifier Type: -

Identifier Source: org_study_id

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