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Biliary Tract CarcinomaPhase 3NCT04924062

Pembrolizumab (MK-3475) Plus Gemcitabine/Cisplatin Versus Placebo Plus Gemcitabine/Cisplatin for First-Line Advanced and/or Unresectable Biliary Tract Carcinoma (BTC) (MK-3475-966/KEYNOTE-966)-China Extension Study

A Phase 3 study testing Pembrolizumab, Gemcitabine and Cisplatin in people with Biliary Tract Carcinoma, run by Merck Sharp & Dohme LLC at 22 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

In this China Extension study, pembrolizumab plus gemcitabine/cisplatin will be compared with placebo plus gemcitabine/cisplatin as first-line therapy in Chinese adults with advanced and/or unresectable biliary tract carcinoma. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Biliary Tract Carcinoma
What is being tested?
Pembrolizumab, Gemcitabine and Cisplatin
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
158 participants
Where
22 sitesChina
Study dates
10 Jul 2020to 25 Mar 2025
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Has histologically confirmed diagnosis of advanced (metastatic) and/or unresectable (locally advanced) biliary tract cancer (intra-or extrahepatic cholangiocarcinoma or gallbladder cancer)
  • Has measurable disease based on Response Evaluation Criteria in Solid Tumors (RECIST 1.1), as determined by the site investigator
  • Participants with a history of hepatitis B or hepatitis C can be enrolled if they meet study criteria
  • Is able to provide archival tumor tissue sample or newly obtained core or excisional biopsy of a tumor lesion
  • Has a life expectancy of greater than 3 months
  • Has adequate organ function

Who cannot join

  • Has had previous systemic therapy for advanced (metastatic) or unresectable (locally advanced) biliary tract cancer (intra-or extra hepatic cholangiocarcinoma or gallbladder cancer)
  • Has ampullary cancer
  • Has small cell cancer, neuroendocrine tumors, lymphoma, sarcoma, mixed tumor histology and/or mucinous cystic neoplasms
  • Has received prior therapy with an anti-programmed cell death 1 (anti-PD-1), anti- programmed cell death ligand 1 or 2 (anti-PD-L1, anti-PD-L2) agent or with an agent directed to another stimulatory or coinhibitory T-cell receptor (e.g., cytotoxic T-lymphocyte-associated protein 4 [CTLA-4], OX-40, CD137)
  • Has a known history of, or any evidence of, central nervous system (CNS) metastases and/or carcinomatous meningitis, as assessed by local site investigator
  • Has had an allogenic tissue/solid organ transplant

Trial locations and contacts

22 sites in 1 country.

22 sites in 1 country

China22 sites

  • Beijing Cancer Hospital ( Site 0138)

    Beijing, Beijing Municipality, 100036

  • Peking Union Medical College Hospital ( Site 0150)

    Beijing, Beijing Municipality, 100730

  • The First Hospital of Jilin University ( Site 0131)

    Changchun, Jilin, 130021

  • Hunan Provincial People Hospital ( Site 0142)

    Changsha, Hunan, 410005

  • Hunan Cancer Hospital ( Site 0132)

    Changsha, Hunan, 410013

  • The Third Xiangya Hospital of Central South University ( Site 0157)

    Changsha, Hunan, 410013

  • West China Hospital of Sichuan University ( Site 0147)

    Chengdu, Sichuan, 610041

  • First Affiliated Hospital of The Third Military Medical University ( Site 0130)

    Chongqing, Chongqing Municipality, 400038

  • Fujian Provincial Cancer Hospital ( Site 0154)

    Fuzhou, Fujian, 350014

  • 900 Hospital of the Joint ( Site 0137)

    Fuzhou, Fujian, 350025

  • Guangdong Provincial People s Hospital ( Site 0161)

    Guangzhou, Guangdong, 510080

  • The First Affiliated Hospital Zhejiang University ( Site 0136)

    Hangzhou, Zhejiang, 310003

  • Zhejiang Cancer Hospital ( Site 0134)

    Hangzhou, Zhejiang, 310022

  • Harbin Medical University Cancer Hospital ( Site 0133)

    Harbin, Heilongjiang, 610000

  • Anhui Provincial Hospital ( Site 0140)

    Hefei, Anhui, 230001

  • The 81st Hospital of PLA ( Site 0128)

    Nanjing, Jiangsu, 210031

  • Zhongshan Hospital Fudan University ( Site 0129)

    Shanghai, Shanghai Municipality, 200032

  • Renji Hospital Shanghai Jiaotong University School of Medicine ( Site 0158)

    Shanghai, Shanghai Municipality, 200127

  • Fudan University Shanghai Cancer Center ( Site 0160)

    Shanghai, Shanghai Municipality, 201315

  • Tianjin Medical University Cancer Institute & Hospital ( Site 0155)

    Tianjin, Tianjin Municipality, 300060

  • Tangdu Hospital ( Site 0146)

    Xi’an, Shanxi, 710038

  • The First Affiliated Hospital of Xi an Jiaotong University ( Site 0145)

    Xi’an, Shanxi, 710048

Other trials in China

About this study

Official title: A Phase 3 Randomized, Double Blind Study of Pembrolizumab Plus Gemcitabine/Cisplatin Versus Placebo Plus Gemcitabine/Cisplatin as First-Line Therapy in Participants With Advanced and/or Unresectable Biliary Tract Carcinoma

In this China Extension study, pembrolizumab plus gemcitabine/cisplatin will be compared with placebo plus gemcitabine/cisplatin as first-line therapy in Chinese adults with advanced and/or unresectable biliary tract carcinoma. The primary hypothesis is pembrolizumab plus gemcitabine/cisplatin is superior to placebo plus gemcitabine/cisplatin with respect to overall survival (OS).

Read the detailed description

The China extension study will include participants previously enrolled in China in the global study for MK-3475-966 (NCT04003636) plus those enrolled during the China extension enrollment period. A total of approximately 158 Chinese participants will be enrolled.

Being tested
Pembrolizumab, Gemcitabine, Cisplatin, Placebo
Keywords
Programmed cell death 1 (PD-1), Pembrolizumab, Cholangiocarcinoma, Gallbladder cancer, Checkpoint inhibitor, Immunotherapy, Biliary, Keytruda, Bile Duct Cancer
Medical subject headings
Parkinson Disease 4, Autosomal Dominant Lewy Body, Cholangiocarcinoma, Gallbladder Neoplasms, Bile Duct Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PembrolizumabBiological

    Also called MK-3475

    Pembrolizumab by intravenous (IV) infusion

  • GemcitabineDrug

    Also called Gemzar

    Gemcitabine by IV infusion

  • CisplatinDrug

    Also called Platinol®, Platinol®-AQ

    Cisplatin by IV infusion

  • PlaceboDrug

    Placebo to pembrolizumab by IV infusion

Study groups in NCT04924062
Study groupRoleWhat they receive
Arm A: Pembrolizumab + ChemotherapyExperimentalParticipants receive pembrolizumab, 200 mg intravenous (IV) infusion, every 3 weeks (Q3W), Day 1 of each 3-week cycle for up to 35 cycles PLUS gemcitabine, 1000 mg/m^2, IV infusion Q3W, Day 1 and Day 8 of each 3-week cycle until progressive disease or unacceptable toxicity PLUS cisplatin, 25 mg/m^2 IV infusion, Q3W, Day 1 and Day 8 of each 3-week cycle for up to 8 cycles. Eligible participants who stop the initial course of pembrolizumab with stable disease (SD) or better but progress after discontinuation will initiate a second course of pembrolizumab 200 mg IV Q3W, Day 1 of each 3-week cycle for up to 17 cycles. Treatment with gemcitabine can be stopped at any time in first or second course due to toxicity or disease progression.
Receives: Biological: Pembrolizumab; Drug: Gemcitabine; Drug: Cisplatin
Arm B: Placebo + ChemotherapyPlacebo comparatorParticipants receive placebo to pembrolizumab, 200 mg IV infusion, every 3 weeks (Q3W), Day 1 of each 3-week cycle for up to 35 cycles PLUS gemcitabine, 1000 mg/m^2 IV infusion, Q3W, Day 1 and Day 8 of each 3-week cycle until progressive disease or unacceptable toxicity PLUS cisplatin, 25 mg/m^2 IV infusion, Q3W, Day 1 and Day 8 of each 3-week cycle for up to 8 cycles.
Receives: Drug: Gemcitabine; Drug: Cisplatin; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Survival (OS)

    Overall survival was defined as the time from randomization to death due to any cause. Per protocol the final reported outcome for OS did not include any sensitivity or supportive analysis.

    Measured: Up to approximately 29 months

Secondary outcomes (5)

  1. Progression-free Survival (PFS) Per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) as Assessed by BICR

    PFS was defined as the time from randomization to the first documented disease progression (PD) or death due to any cause, whichever occurred first. Per RECIST 1.1, PD was defined as ≥20% increase in the sum of diameters of target lesions. In addition to the relative increase of 20%, the sum must also have demonstrated an absolute increase of ≥5 mm. The appearance of one or more new lesions was also considered PD. PFS as assessed by BICR per RECIST 1.1 was presented.

    Measured: Up to approximately 29 months

  2. Objective Response Rate (ORR) Per RECIST 1.1 as Assessed by BICR

    ORR was defined as the percentage of participants who had a confirmed Complete Response (CR: disappearance of all target lesions) or Partial Response (PR: a ≥30% decrease in the sum of diameters [SOD] of target lesions) as assessed by BICR per RECIST 1.1, which was adjusted for this study to allow a maximum of 10 target lesions in total and 5 per organ.

    Measured: Up to approximately 29 months

  3. Duration of Response (DOR) Per RECIST 1.1 as Assessed by BICR

    For participants who demonstrated a confirmed CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions) per RECIST 1.1, DOR was defined as the time from first documented evidence of a CR or PR until PD or death. DOR for participants who had not progressed or died at the time of analysis was to be censored at the date of their last tumor assessment. Per RECIST 1.1, PD was defined as at least a 20% increase in the sum of diameters of target lesions as well as an absolute increase of at least a 5 mm in the sum of diameters. The appearance of one or more new lesions was also considered PD. DOR assessments were based on BICR with confirmation. The DOR as assessed using RECIST 1.1 for all participants who experienced a confirmed CR or PR was presented.

    Measured: Up to approximately 29 months

  4. Number of Participants Who Experienced One or More Adverse Events (AEs)

    An adverse event (AE) was defined as any unfavorable and unintended sign including an abnormal laboratory finding, symptom or disease associated with the use of a medical treatment or procedure, regardless of whether it was considered related to the medical treatment or procedure, that occurred during the course of the study. Number of participants who experienced one or more AEs was reported.

    Measured: Up to approximately 29 months

  5. Number of Participants Who Discontinued Study Intervention Due to an AE

    An AE was defined as any unfavorable and unintended sign including an abnormal laboratory finding, symptom or disease associated with the use of a medical treatment or procedure, regardless of whether it was considered related to the medical treatment or procedure, that occurred during the course of the study. Number of participants who discontinued study intervention (includes any study medication given during the study) due to an AE were reported.

    Measured: Up to approximately 29 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Jun 2021
  2. Study start10 Jul 2020
  3. Primary completion15 Dec 2022
  4. Study completion25 Mar 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Investigator, Outcomes assessor
Enrolment
158 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04924062
Sponsor's protocol ID
3475-966 China Extension
Other IDs
MK-3475-966, KEYNOTE-966, 195007
Status last verified
Apr 2026
First submitted
8 Jun 2021
Last updated
15 Apr 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: https://trialstransparency.msdclinicaltrials.com/pdf/ProcedureAccessClinicalTrialData.pdf

Publications

  • Ren Z, Liang T, Gu S, Gou H, Peng C, Pan Y, Song T, Su H, Cao K, Liang H, Ying J, Geng Z, Yu W, Zhao H, Bai Y, Hao C, Mao Y, Li W, Wang L, Li D, Wang W, Li N, Malhotra U, Qin S. First-Line Pembrolizumab Plus Chemotherapy for Advanced Biliary Tract Cancer: China Subgroup Analysis of the Randomized Phase 3 KEYNOTE-966 Study. Adv Ther. 2026 Sep;43(9):3871-3885. doi: 10.1007/s12325-026-03578-4. Epub 2026 May 23.

    PubMed 42176183

  • Yoo C, Ueno M, Klumpen HJ, Kelley RK, Vogel A, Furuse J, Ren Z, Yau T, Chan SL, Ozaka M, Oh SC, Gu S, Park JO, Valle JW, Edeline J, Kim JG, Kamble S, Norquist JM, Yu L, Malhotra U, Finn RS. Health-related quality of life in participants with advanced biliary tract cancer from the randomized phase III KEYNOTE-966 study. J Hepatol. 2025 Sep;83(3):692-700. doi: 10.1016/j.jhep.2025.03.019. Epub 2025 Mar 27.

    PubMed 40154623

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Pembrolizumab, Gemcitabine and Cisplatin in people with Biliary Tract Carcinoma, run by Merck Sharp & Dohme LLC at 22 sites in China.

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 6 inclusion and 6 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 15 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in China. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Dec 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Overall Survival (OS) (time frame: Up to approximately 29 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04924062, which also lists 2 linked publications.

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 15 Apr 2026.

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