Study of Pembrolizumab (MK-3475) or Placebo Given With Best Supportive Care in Asian Participants With Previously Treated Advanced Hepatocellular Carcinoma (MK-3475-394/KEYNOTE-394)

NCT ID: NCT03062358

Last Updated: 2025-09-30

Study Results

Results available

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

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

453 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-04-27

Study Completion Date

2024-10-15

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The purpose of this study is to determine the efficacy and safety of pembrolizumab or placebo given with best supportive care (BSC) in Asian participants with previously systemically treated advanced hepatocellular carcinoma (HCC). The primary hypothesis of this study is that overall survival is prolonged in participants who receive pembrolizumab compared to those who receive placebo.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Carcinoma, Hepatocellular

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

pembrolizumab + BSC

Participants receive pembrolizumab by intravenous (IV) infusion on Day 1 of each 3-week cycle for up to 35 cycles of treatment plus BSC.

Group Type EXPERIMENTAL

pembrolizumab

Intervention Type BIOLOGICAL

Administered as an intravenous (IV) infusion every 3 weeks (Q3W)

best supportive care (BSC)

Intervention Type OTHER

BSC will include pain management and management of other potential complications including ascites per local standards of care.

placebo + BSC

Participants receive placebo by intravenous (IV) infusion on Day 1 of each 3-week cycle for up to 35 cycles of treatment plus BSC.

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type DRUG

Normal saline solution administered as an IV infusion Q3W

best supportive care (BSC)

Intervention Type OTHER

BSC will include pain management and management of other potential complications including ascites per local standards of care.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

pembrolizumab

Administered as an intravenous (IV) infusion every 3 weeks (Q3W)

Intervention Type BIOLOGICAL

placebo

Normal saline solution administered as an IV infusion Q3W

Intervention Type DRUG

best supportive care (BSC)

BSC will include pain management and management of other potential complications including ascites per local standards of care.

Intervention Type OTHER

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

KEYTRUDA®

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Has a HCC diagnosis confirmed by radiology, histology, or cytology (fibrolamellar, and mixed hepatocellular/cholangiocarcinoma subtypes are not eligible)
* Has Barcelona Clinic Liver Cancer (BCLC) Stage C disease or BCLC Stage B disease not amenable to locoregional therapy or refractory to locoregional therapy and not amenable to a curative treatment approach.
* Has a Child-Pugh A liver score within 7 days prior to first dose of study medication
* Has a life expectancy of \>3 months
* Has at least one measurable lesion based on RECIST version 1.1 as determined by investigator.
* Has Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 performed within 7 days prior to receiving the first dose of study medication.
* Has documented objective radiographic progression during or after treatment with sorafenib or oxaliplatin-based chemotherapy, or else intolerance to sorafenib or oxaliplatin-based chemotherapy
* Female participants of childbearing potential must have a negative urine or serum pregnancy test within 72 hours prior to receiving the first dose of study therapy
* Female and male participants of reproductive potential must agree to use adequate contraception starting from the first dose of study medication, throughout the study period, and for up to 120 days after the last dose of study medication

Exclusion Criteria

* Is currently participating or has participated in a study with an investigational agent or using an investigational device within 4 weeks of the first dose of study medication
* Has received sorafenib or oxaliplatin-based chemotherapy within 14 days of first dose of study medication
* Has had esophageal or gastric variceal bleeding within the last 6 months
* Has clinically apparent ascites on physical examination
* Has portal vein invasion at the main portal branch (Vp4), inferior vena cava, or cardiac involvement of HCC based on imaging
* Has had clinically diagnosed hepatic encephalopathy in the last 6 months
* Has had a solid organ or hematologic transplant
* Has had prior systemic therapy for HCC in the advanced (incurable) setting other than sorafenib or oxaliplatin-based chemotherapy, prior to start of study medication
* Has an active autoimmune disease that has required systemic treatment in the past 2 years. Replacement therapy is not considered a form of systemic treatment.
* Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of study medication
* Has received locoregional therapy to liver (transcatheter chemoembolization \[TACE\], transcatheter embolization \[TAE\], hepatic arterial infusion \[HAI\], radiation, radioembolization, or ablation) within 4 weeks prior to the first dose of study medication
* Has had major surgery to liver or other site within 4 weeks prior to the first dose of study medication
* Has had a minor surgery ≤7 days prior to the first dose of study medication
* Has not recovered adequately (i.e., Grade ≤1 or baseline) from the toxicity and/or complications from any intervention prior to study start
* Has a diagnosed additional malignancy within 3 years prior to first dose of study medication with the exception of curatively treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin and/or curatively resected in situ cancers
* Has a known history of, or any evidence of, central nervous system (CNS) metastases and/or carcinomatous meningitis
* Has a history of (non-infectious) pneumonitis that required steroids or current pneumonitis
* Has an active infection requiring systemic therapy
* Is pregnant or breast feeding or expecting to conceive or father starting from the first dose of study medication, throughout the study period, and for up to 120 days after the last dose of study medication
* Has received prior immunotherapy with an anti-Programmed Cell Death Receptor 1 (PD-1), Programmed Cell Death Receptor Ligand 1 (anti-PD-L1), or anti-Programmed Cell Death Receptor Ligand 2 (PD-L2) or has previously participated in clinical studies with pembrolizumab
* Has a known history of human immunodeficiency virus (HIV)
* Has untreated active Hepatitis B
* Has Hepatitis C in which participants received therapy for HCV \<4 weeks prior to receiving pembrolizumab
* Has received a live vaccine within 30 days prior to the first dose of study therapy
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Merck Sharp & Dohme LLC

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Medical Director

Role: STUDY_DIRECTOR

Merck Sharp & Dohme LLC

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Anhui Provincial Hospital ( Site 0032)

Hefei, Anhui, China

Site Status

The First Affiliated Hospital of Anhui Medical University ( Site 0005)

Hefei, Anhui, China

Site Status

Fuzhou General Hospital of Nanjing Military Command ( Site 0019)

Fuzhou, Fujian, China

Site Status

The First People s Hospital of Foshan ( Site 0033)

Foshan, Guangdong, China

Site Status

Guangdong General Hospital ( Site 0015)

Guangzhou, Guangdong, China

Site Status

Harbin Medical University Cancer Hospital ( Site 0007)

Harbin, Heilongjiang, China

Site Status

Wuhan Tongji Hospital ( Site 0021)

Wuhan, Hubei, China

Site Status

Hubei Cancer Hospital ( Site 0035)

Wuhan, Hubei, China

Site Status

Hunan Cancer Hospital ( Site 0027)

Changsha, Hunan, China

Site Status

The Third Xiangya Hospital of Central South University ( Site 0026)

Changsha, Hunan, China

Site Status

Jiangsu Cancer Hospital ( Site 0003)

Nanjing, Jiangsu, China

Site Status

The 81st Hospital of PLA ( Site 0016)

Nanjing, Jiangsu, China

Site Status

Nantong Tumor Hospital ( Site 0028)

Nantong, Jiangsu, China

Site Status

The First Affiliated Hospital of Soochow University ( Site 0025)

Suzhou, Jiangsu, China

Site Status

Yangzhou No.1 People's Hospital ( Site 0023)

Yangzhou, Jiangsu, China

Site Status

Jilin Province Cancer Hospital, Department of Chemotherapy ( Site 0002)

Changchun, Jilin, China

Site Status

The First Hospital Of Jilin University ( Site 0001)

Changchun, Jilin, China

Site Status

The First Affiliated Hospital of Dalian Medical University ( Site 0022)

Dalian, Liaoning, China

Site Status

Fudan University Shanghai Cancer Center ( Site 0024)

Shanghai, Shanghai Municipality, China

Site Status

The first affiliated Hospital of Xi an Jiaotong University ( Site 0014)

Xi’an, Shanxi, China

Site Status

West China Hospital of Sichuan University ( Site 0030)

Chengdu, Sichuan, China

Site Status

The First affiliated Hospital Zhejing University ( Site 0034)

Hangzhou, Zhejiang, China

Site Status

Zhejiang Cancer Hospital ( Site 0011)

Hangzhou, Zhejiang, China

Site Status

Beijing Cancer Hospital ( Site 0010)

Beijing, , China

Site Status

Bengbu Medical College First Affiliated Hospital ( Site 0020)

Bengbu, , China

Site Status

The Second Affiliated Hospital of Anhui Medical University ( Site 0008)

Hefei, , China

Site Status

Zhongshan Hospital Fudan University ( Site 0012)

Shanghai, , China

Site Status

Renji Hosp,Shanghai Jiao Tong University School of Medicine ( Site 0017)

Shanghai, , China

Site Status

Hong Kong Sanatorium Hospital ( Site 0053)

Hong Kong, , Hong Kong

Site Status

Pamela Youde Nethersole Eastern Hospital ( Site 0052)

Hong Kong, , Hong Kong

Site Status

Princess Margaret Hospital. ( Site 0051)

Hong Kong, , Hong Kong

Site Status

University Malaya Medical Centre ( Site 0091)

Kuala Lumpur, Kuala Lumpur, Malaysia

Site Status

Beacon Hospital Sdn Bhd ( Site 0092)

Petaling Jaya, Selangor, Malaysia

Site Status

Hospital Universiti Kebangsaan Malaysia ( Site 0093)

Cheras, , Malaysia

Site Status

Seoul National University Hospital ( Site 0074)

Seoul, , South Korea

Site Status

Severance Hospital Yonsei University Health System ( Site 0073)

Seoul, , South Korea

Site Status

Asan Medical Center ( Site 0072)

Seoul, , South Korea

Site Status

Samsung Medical Center ( Site 0071)

Seoul, , South Korea

Site Status

Chia-Yi Chang Gung Memorial Hospital ( Site 0133)

Chiayi City, , Taiwan

Site Status

China Medical University Hospital ( Site 0131)

Taichung, , Taiwan

Site Status

National Cheng Kung University Hospital ( Site 0132)

Tainan City, , Taiwan

Site Status

Countries

Review the countries where the study has at least one active or historical site.

China Hong Kong Malaysia South Korea Taiwan

References

Explore related publications, articles, or registry entries linked to this study.

Qin S, Chen Z, Fang W, Ren Z, Xu R, Ryoo BY, Meng Z, Bai Y, Chen X, Liu X, Xiao J, Ho GF, Mao Y, Wang X, Ying J, Li J, Zhong W, Zhou Y, Siegel AB, Hao C. Pembrolizumab Versus Placebo as Second-Line Therapy in Patients From Asia With Advanced Hepatocellular Carcinoma: A Randomized, Double-Blind, Phase III Trial. J Clin Oncol. 2023 Mar 1;41(7):1434-1443. doi: 10.1200/JCO.22.00620. Epub 2022 Dec 1.

Reference Type BACKGROUND
PMID: 36455168 (View on PubMed)

Finn RS, Gu K, Chen X, Merle P, Lee KH, Bouattour M, Cao P, Wang W, Cheng AL, Zhu L, Lim HY, Kudo M, Pan Y, Chang TT, Edeline J, Li W, Yang P, Li C, Li J, Siegel AB, Qin S. Second-line pembrolizumab for advanced HCC: Meta-analysis of the phase III KEYNOTE-240 and KEYNOTE-394 studies. JHEP Rep. 2025 Feb 4;7(6):101350. doi: 10.1016/j.jhepr.2025.101350. eCollection 2025 Jun.

Reference Type DERIVED
PMID: 40486134 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

Access external resources that provide additional context or updates about the study.

https://www.merckclinicaltrials.com/

Merck Clinical Trials Information

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

MK-3475-394

Identifier Type: OTHER

Identifier Source: secondary_id

3475-394

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Neoadjuvant Anti-PD1 in HCC
NCT05471674 COMPLETED PHASE2
Longitudinal Immune-phenotyping of HCC Following MK-3475
NCT04224480 ACTIVE_NOT_RECRUITING PHASE1