NCT04066491

Brief Summary

Study consisted of an open-label, safety run-in part and a randomized, double-blind, placebo-controlled Phase 2/3 part. In the Phase 2/3 part, the study was evaluated whether bintrafusp alfa in combination with the current standard of care (SoC) (gemcitabine plus cisplatin) improves overall survival (OS) in chemotherapy and immunotherapy-naïve participants with locally advanced or metastatic Biliary Tract Cancer (BTC) compared to placebo, gemcitabine and cisplatin.

Trial Health

68
Monitor

Trial Health Score

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

Enrollment
309

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Sep 2019

Typical duration for phase_2

Geographic Reach
15 countries

99 active sites

Status
terminated

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

August 22, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

August 26, 2019

Completed
25 days until next milestone

Study Start

First participant enrolled

September 20, 2019

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 20, 2021

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

June 13, 2022

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 10, 2022

Completed
Last Updated

November 14, 2023

Status Verified

October 1, 2023

Enrollment Period

1.7 years

First QC Date

August 22, 2019

Results QC Date

May 16, 2022

Last Update Submit

October 26, 2023

Conditions

Keywords

Metastatic Biliary Tract CancerCholangiocarcinomaGallbladder CancerAmpullary cancerM7824Bintrafusp alfaTransforming growth factor-betaProgrammed death-ligand 1

Outcome Measures

Primary Outcomes (2)

  • Safety Run-in Part: Number of Participants Who Experienced Dose Limiting Toxicities (DLTs)

    A DLT is a toxicity related to the study intervention that meets the following criteria as evaluated in the open-label, safety run-in: Grade 3 or 4 Immune-related adverse event (irAE) that needs permanent discontinuation of M7824 treatment; a malignant skin lesion induced by M7824 that is local and can be resected with a negative resection margin is not a DLT; Grade 3 or 4 nonhematologic toxicity other than irAE, A life threatening hematological toxicity (unless clearly attributable to chemotherapy alone), which is hardly medically manageable, including a bleeding event resulting in urgent intervention and admission to an intensive care unit and Grade 5 toxicity.

    Day 1 up to Day 21 of Cycle 1 (each Cycle is of 21 days)

  • Double-blind Part: Overall Survival

    Overall Survival was defined as the time from study day 1 to the date of death due to any cause. The overall survival was analyzed by using the Kaplan-Meier method.

    Time from study day 1 up to data cutoff (assessed up to 609 days)

Secondary Outcomes (7)

  • Safety Run-in Part: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs (SAEs) and Treatment Related TEAEs According to National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 5.0

    Time from first treatment up to data cutoff (assessed up to 609 days)

  • Safety Run-in Part: Number of Participants With Grade Greater Than or Equal (>=) 3 Laboratory Abnormalities

    Time from first treatment up to data cutoff (assessed up to 609 days)

  • Double-blind Part: Progression-Free Survival (PFS) According to Response Evaluation Criteria in Solid Tumors (RECIST Version 1.1) Assessed by Independent Review Committee (IRC)

    Time from randomization of study drug until the first documentation of PD or death, assessed up to 609 days

  • Double-blind Part: Percentage of Participants With Confirmed Objective Response According to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) as Assessed by Independent Review Committee (IRC)

    Time from randomization of study drug up to data cut off (assessed up to 609 days)

  • Double-blind Part: Duration of Response (DOR) According to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) as Assessed by Independent Review Committee (IRC)

    From first documented objective response to PD or death due to any cause, assessed up to 609 days

  • +2 more secondary outcomes

Study Arms (3)

Safety Run-In Part: M7824 + Gemcitabine + Cisplatin

EXPERIMENTAL
Drug: M7824Drug: GemcitabineDrug: Cisplatin

Double-blinded Part: M7824 + Gemcitabine + Cisplatin

EXPERIMENTAL
Drug: M7824Drug: GemcitabineDrug: Cisplatin

Double-blinded Part: Placebo + Gemcitabine + Cisplatin

PLACEBO COMPARATOR
Drug: PlaceboDrug: GemcitabineDrug: Cisplatin

Interventions

M7824DRUG

Participants received intravenous infusion of M7824 at a dose of 2400 milligrams (mg), once every 3 weeks (Q3W) 2 years (in case of Complete Response), otherwise until criterion pre-sepcified in protocol for discontinuation is met, in combination with intravenous infusion of Gemcitabine and Cisplatin at a dose of 1000 milligram per meter square (mg/m\^2) and 25 mg/m\^2 respectively on Day 1 and Day 8 of 21- day cycle, for 8 cycles every 3 weeks.

Double-blinded Part: M7824 + Gemcitabine + CisplatinSafety Run-In Part: M7824 + Gemcitabine + Cisplatin

Participants received intravenous infusion of M7824 matched placebo, once every 3 weeks (Q3W) until 2 years (in case of CR), otherwise until crtiterion pre-sepcified in protocol for discontinuation is met.

Double-blinded Part: Placebo + Gemcitabine + Cisplatin

Gemcitabine was received intravenously at a dose of 1000 milligram per meter square (mg/m\^2) on Day 1 and Day 8 of 21- day cycle, for 8 cycles every 3 weeks (Q3W).

Double-blinded Part: M7824 + Gemcitabine + CisplatinDouble-blinded Part: Placebo + Gemcitabine + CisplatinSafety Run-In Part: M7824 + Gemcitabine + Cisplatin

Cisplatin was received intravenously at a dose of 25 mg/m\^2 on Day 1 and Day 8 of 21-day cycle, for 8 cycles every 3 weeks (Q3W).

Double-blinded Part: M7824 + Gemcitabine + CisplatinDouble-blinded Part: Placebo + Gemcitabine + CisplatinSafety Run-In Part: M7824 + Gemcitabine + Cisplatin

Eligibility Criteria

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

You may qualify if:

  • Are participants with histologically or cytologically confirmed locally advanced or metastatic BTC
  • Participants must have available tumor tissue (primary or metastatic) (archival or fresh biopsies) before the first administration of study treatment
  • At least 1 measurable lesion according to RECIST 1.1
  • Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 or 1 at study entry and at Week 1, Day 1 prior to dosing
  • Life expectancy of \>= 12 weeks, as judged by the Investigator
  • Adequate hematological function, hepatic function, renal function, coagulation function as defined in the protocol
  • Hepatitis B virus (HBV) deoxyribonucleic acid (DNA) positive participants must be treated and on a stable dose of antivirals

You may not qualify if:

  • Previous and/or intercurrent cancers
  • Receipt of any organ transplantation, including allogeneic stem-cell transplantation, but with the exception of transplants that do not require immunosuppression
  • Participants with symptomatic central nervous system (CNS) metastases
  • Significant acute or chronic infection including known history of positive test for human immunodeficiency virus (HIV), active tuberculosis, uncontrolled biliary infection and active bacterial or fungal infection requiring systemic therapy (with the exception of hepatitis B and hepatitis C) requiring systemic therapy at study entry and at Week 1 Day 1 prior to dosing.
  • Active autoimmune disease that might deteriorate when receiving an immunostimulatory agent
  • History of or concurrent interstitial lung disease
  • History of hypersensitivity reactions to bintrafusp alfa, anaphylaxis, or recent (within 5 months) uncontrolled asthma, cardiovascular/cerebrovascular disease
  • Chronic obstructive pulmonary disease exacerbation or other respiratory illness requiring hospitalization or precluding study therapy within 30 days before randomization
  • Prior therapy with any antibody/drug targeting T-cell coregulatory proteins (immune checkpoints)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (99)

Ironwood Cancer & Research Centers - Chandler II

Chandler, Arizona, 85224, United States

Location

Banner MD Anderson Cancer Center

Gilbert, Arizona, 85234, United States

Location

Mayo Clinic Arizona

Phoenix, Arizona, 85054, United States

Location

Beverly Hills Cancer Center

Beverly Hills, California, 90211, United States

Location

Mayo Clinic in Florida - Department of Neurology

Jacksonville, Florida, 32224, United States

Location

Mount Sinai Medical Center

Miami Beach, Florida, 33140, United States

Location

University of Kansas Medical Center Research Institute, Inc. - University of Kansas Cancer Center/Bloch Pavilion

Westwood, Kansas, 66216, United States

Location

Sidney Kimmel Comprehensive Cancer Center at John Hopkins

Baltimore, Maryland, 21287, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

Methodist Transplant Physicians

Dallas, Texas, 75203, United States

Location

MD Anderson Cancer Center - Unit 429

Houston, Texas, 77030, United States

Location

Renovatio Clinical - CENTRAL SITE

The Woodlands, Texas, 77380, United States

Location

Hospital de Gastroenterologia Dr. Carlos Bonorino Udaondo

Ciudad Autonoma Buenos Aires, Argentina

Location

Instituto de Investigaciones Metabolicas (IDIM)

Ciudad Autonoma Buenos Aires, Argentina

Location

Instituto Medico Especializado Alexander Fleming

Ciudad Autonoma Buenos Aires, Argentina

Location

Centro Oncologico Riojano Integral (CORI)

La Rioja, Argentina

Location

CEDIT

Salta, Argentina

Location

Centro Medico San Roque S.R.L.

San Miguel de Tucumán, Argentina

Location

Fundacion ARS Medica

San Salvador de Jujuy, Argentina

Location

Blacktown Hospital - PARENT

Blacktown, Australia

Location

Monash Health

Clayton, Australia

Location

Epworth Freemasons

Melbourne, Australia

Location

Icon Cancer Care South Brisbane

South Brisbane, Australia

Location

Hospital de Câncer de Barretos - Fundação Pio XII

Barretos, Brazil

Location

INCA - Instituto Nacional de Câncer

Rio de Janeiro, Brazil

Location

CEPHO - Centro de Estudos e Pesquisas de Hematologia e Oncologia

Santo André, Brazil

Location

Fundação Faculdade Regional de Medicina de São José do Rio Preto

Sao Jose Rio Preto, Brazil

Location

A. C. Camargo Cancer Center

São Paulo, Brazil

Location

ICESP - Instituto do Câncer do Estado de São Paulo Octavio Frias de Oliveira

São Paulo, Brazil

Location

IC la serena Research

La Serena, Chile

Location

Centro de Investigación Clínica Bradford Hill

Santiago, Chile

Location

Hospital Clínico Universidad de Chile

Santiago, Chile

Location

Prosalud

Santiago, Chile

Location

Instituto Clinico Oncologico del Sur (ICOS)

Temuco, Chile

Location

Beijing Cancer Hospital

Beijing, China

Location

Beijing Chao Yang Hospital

Beijing, China

Location

Beijing Friendship Hospital, Capital Medical University

Beijing, China

Location

West China Hospital, Sichuan University

Chengdu, China

Location

Sir Run Run Shaw Hospital, Zhejiang University, School of Medicine

Hangzhou, China

Location

The Affiliated Hospital of Qingdao University

Qingdao, China

Location

Fudan University Shanghai Cancer Hospital

Shanghai, China

Location

The Second Affiliated Hospital of Soochow University

Suzhou, China

Location

Tianjin Medical University Cancer Institute & Hospital

Tianjin, China

Location

Union Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, China

Location

ICO - Site Paul Papin - service d'oncologie medicale

Angers, France

Location

Centre Georges François Leclerc - Oncologie Médicale

Dijon, France

Location

CHU Lille - Hôpital Claude Huriez

Lille, France

Location

ICO - Site René Gauducheau

Saint-Herblain, France

Location

CHU de Toulouse - Hôpital Ranguei

Toulouse, France

Location

Vivantes Klinikum Neukoelln - Haematologie und Onkologie

Berlin, Germany

Location

Universitaetsklinikum Bonn AoeR - Medizinische Klinik I Gastroenterologie

Bonn, Germany

Location

Universitaetsklinikum Carl Gustav Carus TU Dresden - Medizinische Klinik I

Dresden, Germany

Location

Klinikum der Johann Wolfgang Goethe-Universitaet

Frankfurt, Germany

Location

Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz - Medizinische Klinik I - HCC Ambulanz

Mainz, Germany

Location

Fondazione IRCCS Istituto Nazionale dei Tumori Milano

Milan, Italy

Location

IOV - Istituto Oncologico Veneto IRCCS - S.Semplice Dip.Oncologia dei Melanomi

Padua, Italy

Location

Università Campus Bio-Medico di Roma

Roma, Italy

Location

Azienda Ospedaliera Universitaria Integrata Verona (Ospedale Borgo Roma) - UOC Oncologia

Verona, Italy

Location

Chiba Cancer Center

Chiba, Japan

Location

National Cancer Center Hospital - Dept of Hepatobiliary and Pancreatic Oncology

Chūōku, Japan

Location

NHO Kyushu Cancer Center - Dept of Gastroenterology/Hepatology/Pancreatology

Fukuoka, Japan

Location

National Cancer Center Hospital East

Kashiwa-shi, Japan

Location

Cancer Institute Hospital of JFCR - Dept of Hepato,Biliary and Pancreatic Medicine

Kōtoku, Japan

Location

Kyorin University Hospital

Mitaka-shi, Japan

Location

Aichi Cancer Center Hospital

Nagoya, Japan

Location

Osaka City University Hospital

Osaka, Japan

Location

Kindai University Hospital

Osakasayama-shi, Japan

Location

Kanagawa Cancer Center

Yokohama, Japan

Location

Centrum Onkologii-Instytut im.M.Sklodowskiej Curie

Gliwice, Poland

Location

Pratia

Krakow, Poland

Location

Narodowy Instytut Onkologii im. Marii Skłodowskiej-Curie - Państwowy Instytut Badawczy

Warsaw, Poland

Location

ETG Zamosc

Zamość, Poland

Location

Chungnam National University Hospital - Department of Internal Medicine (Rheumatology)

Daejeon, South Korea

Location

Seoul National University Bundang Hospital

Seongnam-si, South Korea

Location

Asan Medical Center

Seoul, South Korea

Location

Korea University Anam Hospital

Seoul, South Korea

Location

Korea University Guro Hospital

Seoul, South Korea

Location

Samsung Medical Center

Seoul, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, South Korea

Location

The Catholic University of Korea, Seoul St. Mary's Hospital

Seoul, South Korea

Location

Hospital Clinic i Provincial de Barcelona - Servicio de Oncologia

Barcelona, Spain

Location

Hospital Universitari Vall d'Hebron - Dept of Oncology

Barcelona, Spain

Location

Hospital San Pedro de Alcantara - Servicio de Oncologia

Cáceres, Spain

Location

Hospital Universitario Reina Sofia - Dept of Oncology

Córdoba, Spain

Location

ICO l´Hospitalet - Hospital Duran i Reynals

L'Hospitalet de Llobregat, Spain

Location

Hospital General Universitario Gregorio Marañon - Servicio de Oncologia Medica

Madrid, Spain

Location

Hospital Universitario Clinico San Carlos - Servicio de Oncologia

Madrid, Spain

Location

Hospital Universitario HM Madrid Sanchinarro - Servicio de Oncologia

Madrid, Spain

Location

Hospital Clinico Universitario de Valencia - Servicio de Hematologia y Oncologia Medica

Valencia, Spain

Location

Hospital Universitari i Politecnic La Fe - Servicio de Oncologia Medica

Valencia, Spain

Location

Kaohsiung Chang Gung Memorial Hospital

Kaohsiung City, Taiwan

Location

China Medical University Hospital

Taichung, Taiwan

Location

Taichung Veterans General Hospital

Taichung, Taiwan

Location

National Cheng Kung University Hospital

Tainan, Taiwan

Location

National Taiwan University Hospital

Taipei, Taiwan

Location

Taipei Veterans General Hospital

Taipei, Taiwan

Location

Chang Gung Memorial Hospital, Linkou

Taoyuan District, Taiwan

Location

The Christie - Dept of Oncology

Manchester, United Kingdom

Location

Related Publications (1)

  • Yoo C, Oh DY, Choi HJ, Kudo M, Ueno M, Kondo S, Chen LT, Osada M, Helwig C, Dussault I, Ikeda M. Phase I study of bintrafusp alfa, a bifunctional fusion protein targeting TGF-beta and PD-L1, in patients with pretreated biliary tract cancer. J Immunother Cancer. 2020 May;8(1):e000564. doi: 10.1136/jitc-2020-000564.

Related Links

MeSH Terms

Conditions

Biliary Tract NeoplasmsCholangiocarcinomaGallbladder NeoplasmsCamurati-Engelmann Syndrome

Interventions

GemcitabineCisplatin

Condition Hierarchy (Ancestors)

Digestive System NeoplasmsNeoplasms by SiteNeoplasmsBiliary Tract DiseasesDigestive System DiseasesAdenocarcinomaCarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeGallbladder DiseasesOsteochondrodysplasiasBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Heterocyclic CompoundsDeoxycytidineCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingChlorine CompoundsInorganic ChemicalsNitrogen CompoundsPlatinum Compounds

Limitations and Caveats

Data collection and analysis of Pharmacokinetics and Immunogenicity were omitted and not conducted due to business reason.

Results Point of Contact

Title
Communication Center
Organization
Merck Healthcare KGaA, Darmstadt Germany, an affiliate of Merck KGaA, Darmstadt, Germany

Study Officials

  • Medical Responsible

    Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
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

August 22, 2019

First Posted

August 26, 2019

Study Start

September 20, 2019

Primary Completion

May 20, 2021

Study Completion

November 10, 2022

Last Updated

November 14, 2023

Results First Posted

June 13, 2022

Record last verified: 2023-10

Data Sharing

IPD Sharing
Will share

We are committed to enhancing public health through responsible sharing of clinical trial data. Following approval of a new product or a new indication for an approved product in both the US and European Union, the study sponsor and/or its affiliated companies will share study protocols, anonymized patient data and study level data, and redacted clinical study reports with qualified scientific and medical researchers, upon request, as necessary for conducting legitimate research. Further information on how to request data can be found on our website bit.ly/IPD21

Shared Documents
STUDY PROTOCOL, SAP, CSR
Time Frame
Within six months after the approval of a new product or a new indication for an approved product in both the United States and the European Union.
Access Criteria
Qualified scientific and medical researchers can request the data. Such requests must be submitted in writing to the company's portal and will be internally reviewed regarding criteria for researchers' qualification and legitimacy of the research proposal.
More information

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