An Immuno-therapy Study to Evaluate the Effectiveness, Safety and Tolerability of Nivolumab or Nivolumab in Combination With Other Agents in Patients With Advanced Liver Cancer

NCT ID: NCT01658878

Last Updated: 2025-12-24

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

657 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-10-30

Study Completion Date

2024-11-12

Brief Summary

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The first part of the study is the Dose Escalation Phase designed to establish the safety of nivolumab at different dose levels for each of the three cohorts (uninfected hepatocellular carcinoma (HCC) subjects, hepatitis C virus (HCV)-infected HCC subjects, and hepatitis B virus (HBV)-infected subjects).

The second part of the study is the Expansion Phase designed to generate additional clinical data at specified doses for each of the 3 cohorts. A third cohort has been added in this study to compare the efficacy of nivolumab and sorafenib in the treatment of Advanced HCC. A fourth cohort will generate data on the safety and efficacy of the combination nivolumab plus ipilimumab in the treatment of Advanced HCC. In the fifth cohort, additional clinical data will be generated for Child-Pugh B subjects. A Cabozantinib Combination Cohort has been added to evaluate the safety and tolerability of nivolumab in combination with cabozantinib and nivolumab with ipilimumab in combination with cabozantinib.

Detailed Description

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Study Classification: Pharmacokinetics/Pharmacodynamics

Conditions

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Hepatocellular Carcinoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Non-infected: Nivolumab

Nivolumab intravenous solution on specific days

Group Type EXPERIMENTAL

Nivolumab

Intervention Type BIOLOGICAL

HCV-infected: Nivolumab

Nivolumab intravenous solution on specific days

Group Type EXPERIMENTAL

Nivolumab

Intervention Type BIOLOGICAL

HBV-infected: Nivolumab

Nivolumab intravenous solution on specific days

Group Type EXPERIMENTAL

Nivolumab

Intervention Type BIOLOGICAL

Nivolumab

Nivolumab intravenous solution on specific days

Group Type EXPERIMENTAL

Nivolumab

Intervention Type BIOLOGICAL

Sorafenib

Sorafenib tablets on specific days

Group Type ACTIVE_COMPARATOR

Sorafenib

Intervention Type DRUG

Nivolumab plus Ipilimumab Combination

Nivolumab intravenous solution + Ipilimumab intravenous solution on specific days

Group Type EXPERIMENTAL

Nivolumab

Intervention Type BIOLOGICAL

Ipilimumab

Intervention Type DRUG

Child-Pugh B

Nivolumab intravenous solution on specific days

Group Type EXPERIMENTAL

Nivolumab

Intervention Type BIOLOGICAL

Nivolumab plus Cabozantinib Combination

Nivolumab intravenous solution + cabozantinib oral tablets on specific days

Group Type EXPERIMENTAL

Cabozantinib

Intervention Type DRUG

Nivolumab plus Ipilimumab plus Cabozantinib

Nivolumab intravenous solution + Ipilimumab intravenous solution + cabozantinib oral tablets on specific days

Group Type EXPERIMENTAL

Cabozantinib

Intervention Type DRUG

Interventions

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Nivolumab

Intervention Type BIOLOGICAL

Sorafenib

Intervention Type DRUG

Ipilimumab

Intervention Type DRUG

Cabozantinib

Intervention Type DRUG

Other Intervention Names

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BMS-936558

Eligibility Criteria

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Inclusion Criteria

* Subjects of 18 years or older (men and women) with histologically confirmed advanced hepatocellular carcinoma, not eligible for surgical and/or locoregional therapies; or progressive disease after surgical and /or locoregional therapies
* Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 1
* Dose Escalation Phase: Child-Pugh score of 7 points or less. Cohort 5: Child-Pugh Class B (B7-B8). For all other cohorts Child-Pugh score of 6 points or less

Exclusion Criteria

* History of autoimmune disease
* Any prior or current clinically significant ascites
* Any history of hepatic encephalopathy
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ono Pharmaceutical Co. Ltd

INDUSTRY

Sponsor Role collaborator

Bristol-Myers Squibb

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Bristol-Myers Squibb

Role: STUDY_DIRECTOR

Bristol-Myers Squibb

Locations

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Local Institution - 0008

Los Angeles, California, United States

Site Status

Local Institution - 0048

Washington D.C., District of Columbia, United States

Site Status

Local Institution - 0053

Pensacola, Florida, United States

Site Status

Local Institution - 0047

Atlanta, Georgia, United States

Site Status

Local Institution - 0025

Boston, Massachusetts, United States

Site Status

Local Institution - 0002

Ann Arbor, Michigan, United States

Site Status

Local Institution - 0054

Hackensack, New Jersey, United States

Site Status

Local Institution - 0067

Paterson, New Jersey, United States

Site Status

Local Institution - 0001

Portland, Oregon, United States

Site Status

The University Of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Local Institution - 0065

Halifax, Nova Scotia, Canada

Site Status

Local Institution - 0039

Toronto, Ontario, Canada

Site Status

Local Institution - 0022

Montreal, Quebec, Canada

Site Status

Local Institution - 0061

Angers, , France

Site Status

Local Institution - 0064

Créteil, , France

Site Status

Local Institution - 0062

Marseille, , France

Site Status

Local Institution - 0059

Marseille, , France

Site Status

Local Institution - 0042

Paris, , France

Site Status

Local Institution - 0060

Reims, , France

Site Status

Local Institution - 0058

Vandœuvre-lès-Nancy, , France

Site Status

Local Institution - 0030

Essen, , Germany

Site Status

Local Institution - 0028

Frankfurt, , Germany

Site Status

Local Institution - 0029

Hanover, , Germany

Site Status

Local Institution - 0031

Heidelberg, , Germany

Site Status

Local Institution - 0005

Hong Kong, , Hong Kong

Site Status

Local Institution - 0006

Hong Kong, , Hong Kong

Site Status

Local Institution - 0032

Bologna, , Italy

Site Status

Local Institution - 0056

Florence, , Italy

Site Status

Local Institution - 0063

Meldola (FC), , Italy

Site Status

Local Institution - 0055

Milan, , Italy

Site Status

Local Institution - 0034

Napoli, , Italy

Site Status

Local Institution - 0035

Padua, , Italy

Site Status

Local Institution - 0040

Rozzano, , Italy

Site Status

Local Institution - 0036

Kashiwa-shi, Chiba, Japan

Site Status

Local Institution - 0038

Kurume-shi, Fukuoka, Japan

Site Status

Local Institution - 0049

Yokohama, Kanagawa, Japan

Site Status

Local Institution - 0050

Kyoto, Kyoto, Japan

Site Status

Local Institution - 0037

Ōsaka-sayama, Osaka, Japan

Site Status

Local Institution - 0051

Saga, , Japan

Site Status

Local Institution - 0070

San Juan, , Puerto Rico

Site Status

Local Institution - 0017

Singapore, , Singapore

Site Status

Local Institution - 0009

Singapore, , Singapore

Site Status

Local Institution - 0007

Singapore, , Singapore

Site Status

Local Institution - 0066

Seoul, , South Korea

Site Status

Local Institution - 0021

Seoul, , South Korea

Site Status

Local Institution - 0026

Seoul, , South Korea

Site Status

Local Institution - 0016

Seoul, , South Korea

Site Status

Local Institution - 0019

Barcelona, , Spain

Site Status

Local Institution - 0020

Madrid, , Spain

Site Status

Local Institution - 0018

Madrid, , Spain

Site Status

Local Institution - 0003

Pamplona, , Spain

Site Status

Local Institution - 0027

Taipei, , Taiwan

Site Status

Local Institution - 0024

Taipei, , Taiwan

Site Status

Local Institution - 0023

Taoyuan District, , Taiwan

Site Status

Local Institution - 0011

London, Greater London, United Kingdom

Site Status

Local Institution - 0014

Manchester, Greater Manchester, United Kingdom

Site Status

Local Institution - 0012

Glasgow, Lanarkshire, United Kingdom

Site Status

Local Institution - 0015

Metropolitan Borough of Wirral, Merseyside, United Kingdom

Site Status

Local Institution - 0013

Birmingham, West Midlands, United Kingdom

Site Status

Local Institution - 0010

London, , United Kingdom

Site Status

Countries

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United States Canada France Germany Hong Kong Italy Japan Puerto Rico Singapore South Korea Spain Taiwan United Kingdom

References

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Zhou X, Cao J, Topatana W, Xie T, Chen T, Hu J, Li S, Juengpanic S, Lu Z, Zhang B, Wang K, Feng X, Shen J, Chen M. Evaluation of PD-L1 as a biomarker for immunotherapy for hepatocellular carcinoma: systematic review and meta-analysis. Immunotherapy. 2023 Apr;15(5):353-365. doi: 10.2217/imt-2022-0168. Epub 2023 Feb 27.

Reference Type DERIVED
PMID: 36852452 (View on PubMed)

Yau T, Zagonel V, Santoro A, Acosta-Rivera M, Choo SP, Matilla A, He AR, Cubillo Gracian A, El-Khoueiry AB, Sangro B, Eldawy TE, Bruix J, Frassineti GL, Vaccaro GM, Tschaika M, Scheffold C, Koopmans P, Neely J, Piscaglia F. Nivolumab Plus Cabozantinib With or Without Ipilimumab for Advanced Hepatocellular Carcinoma: Results From Cohort 6 of the CheckMate 040 Trial. J Clin Oncol. 2023 Mar 20;41(9):1747-1757. doi: 10.1200/JCO.22.00972. Epub 2022 Dec 13.

Reference Type DERIVED
PMID: 36512738 (View on PubMed)

Sove RJ, Verma BK, Wang H, Ho WJ, Yarchoan M, Popel AS. Virtual clinical trials of anti-PD-1 and anti-CTLA-4 immunotherapy in advanced hepatocellular carcinoma using a quantitative systems pharmacology model. J Immunother Cancer. 2022 Nov;10(11):e005414. doi: 10.1136/jitc-2022-005414.

Reference Type DERIVED
PMID: 36323435 (View on PubMed)

Kudo M, Matilla A, Santoro A, Melero I, Gracian AC, Acosta-Rivera M, Choo SP, El-Khoueiry AB, Kuromatsu R, El-Rayes B, Numata K, Itoh Y, Di Costanzo F, Crysler O, Reig M, Shen Y, Neely J, Tschaika M, Wisniewski T, Sangro B. CheckMate 040 cohort 5: A phase I/II study of nivolumab in patients with advanced hepatocellular carcinoma and Child-Pugh B cirrhosis. J Hepatol. 2021 Sep;75(3):600-609. doi: 10.1016/j.jhep.2021.04.047. Epub 2021 May 26.

Reference Type DERIVED
PMID: 34051329 (View on PubMed)

Yau T, Kang YK, Kim TY, El-Khoueiry AB, Santoro A, Sangro B, Melero I, Kudo M, Hou MM, Matilla A, Tovoli F, Knox JJ, Ruth He A, El-Rayes BF, Acosta-Rivera M, Lim HY, Neely J, Shen Y, Wisniewski T, Anderson J, Hsu C. Efficacy and Safety of Nivolumab Plus Ipilimumab in Patients With Advanced Hepatocellular Carcinoma Previously Treated With Sorafenib: The CheckMate 040 Randomized Clinical Trial. JAMA Oncol. 2020 Nov 1;6(11):e204564. doi: 10.1001/jamaoncol.2020.4564. Epub 2020 Nov 12.

Reference Type DERIVED
PMID: 33001135 (View on PubMed)

Sangro B, Melero I, Wadhawan S, Finn RS, Abou-Alfa GK, Cheng AL, Yau T, Furuse J, Park JW, Boyd Z, Tang HT, Shen Y, Tschaika M, Neely J, El-Khoueiry A. Association of inflammatory biomarkers with clinical outcomes in nivolumab-treated patients with advanced hepatocellular carcinoma. J Hepatol. 2020 Dec;73(6):1460-1469. doi: 10.1016/j.jhep.2020.07.026. Epub 2020 Jul 22.

Reference Type DERIVED
PMID: 32710922 (View on PubMed)

Yau T, Hsu C, Kim TY, Choo SP, Kang YK, Hou MM, Numata K, Yeo W, Chopra A, Ikeda M, Kuromatsu R, Moriguchi M, Chao Y, Zhao H, Anderson J, Cruz CD, Kudo M. Nivolumab in advanced hepatocellular carcinoma: Sorafenib-experienced Asian cohort analysis. J Hepatol. 2019 Sep;71(3):543-552. doi: 10.1016/j.jhep.2019.05.014. Epub 2019 Jun 7.

Reference Type DERIVED
PMID: 31176752 (View on PubMed)

El-Khoueiry AB, Sangro B, Yau T, Crocenzi TS, Kudo M, Hsu C, Kim TY, Choo SP, Trojan J, Welling TH Rd, Meyer T, Kang YK, Yeo W, Chopra A, Anderson J, Dela Cruz C, Lang L, Neely J, Tang H, Dastani HB, Melero I. Nivolumab in patients with advanced hepatocellular carcinoma (CheckMate 040): an open-label, non-comparative, phase 1/2 dose escalation and expansion trial. Lancet. 2017 Jun 24;389(10088):2492-2502. doi: 10.1016/S0140-6736(17)31046-2. Epub 2017 Apr 20.

Reference Type DERIVED
PMID: 28434648 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

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Other Identifiers

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2012-001514-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CA209-040

Identifier Type: -

Identifier Source: org_study_id