Can a targeted pill outperform chemo for bile duct cancer?

NCT ID NCT03656536

Disease control Sponsor: Incyte Corporation Source: ClinicalTrials.gov ↗

First seen Aug 11, 2026 · Last updated Aug 12, 2026 · Updated 1 time

Summary

This trial is testing whether the targeted drug pemigatinib can slow or shrink a specific type of bile duct cancer (cholangiocarcinoma) that has a genetic change called FGFR2 rearrangement and cannot be removed by surgery. Participants will receive either pemigatinib as a daily pill or standard chemotherapy (gemcitabine plus cisplatin) given by infusion. The study aims to see if pemigatinib is more effective at delaying cancer progression and shrinking tumors, and whether it has a better safety profile.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Pemigatinib, an oral drug that targets FGFR2, compared to standard chemotherapy (gemcitabine plus cisplatin).
What this could lead to: If pemigatinib works better than chemotherapy, it could become a new first-line treatment for people with this specific type of bile duct cancer, potentially slowing tumor growth and extending life.
What could go wrong: This is a phase 3 trial, but results are not guaranteed. Pemigatinib may not be more effective than chemotherapy, and it can cause side effects like high phosphate levels, fatigue, and liver issues.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

167 people

The number who actually took part.

Started

Jun 2019

Finished

Jul 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Male and female participants at least 18 years of age at the time of signing the informed consent form (ICF). * Histologically or cytologically confirmed cholangiocarcinoma that is previously untreated and considered unresectable and/or metastatic (Stage IV per the American Joint Committee on Cancer (AJCC) Cancer Staging Manual). * Radiographically measurable or evaluable disease by CT or MRI per RECIST v1.1 criteria. * Eastern Cooperative Oncology Group performance status 0 to 1. * Documented FGFR2 rearrangement. * Willingness to avoid pregnancy or fathering children. Exclusion Criteria: * Received prior anticancer systemic therapy for unresectable and/or metastatic disease (not including adjuvant/neo-adjuvant treatment completed at least 6 months prior to enrollment, and participants that have received treatment for locally advanced disease with trans-arterial chemoembolization or selective internal radiation therapy, if clear evidence of radiological progression is observed before enrollment, or enrolled as of Amendment 6 (or Amendment 5-JP2) and the participant received 1 cycle of gemcitabine plus cisplatin \[the start of study drug {Cycle 1 Day 1} must be at least 14 days and ≤ 4 weeks {28 days} from the last dose of gemcitabine plus cisplatin\]). * Child-Pugh B and C. * Toxicities related to prior therapy(ies) must be Common Terminology Criteria for Adverse Events (CTCAE) v5.0 ≤ Grade 1 at the time of screening. * Concurrent anticancer therapy, other than the therapies being tested in this study. * Participant is a candidate for potentially curative surgery. * Current evidence of clinically significant corneal (including but not limited to bullous/band keratopathy, corneal abrasion, inflammation/ulceration, and keratoconjunctivitis) or retinal disorder (including but not limited to central serous retinopathy, macular/retinal degeneration, diabetic retinopathy, retinal detachment) as confirmed by ophthalmologic examination. * Radiation therapy administered within 4 weeks of enrollment/randomization/first dose of study treatment. * Known central nervous system (CNS) metastases or history of uncontrolled seizures. * Known additional malignancy that is progressing or requires active treatment (exceptions: basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy). * Laboratory values at screening outside the protocol-defined range. * History of calcium and phosphate hemostasis disorder or systemic mineral imbalance with ectopic calcification of soft tissues (exception: commonly observed calcifications in soft tissues, such as the skin, kidney, tendons or vessels due to injury, disease, and aging, in the absence of systemic mineral imbalance). * Significant gastrointestinal disorders that could interfere with absorption, metabolism, or excretion of pemigatinib. * Clinically significant or uncontrolled cardiac disease. * History or presence of an abnormal ECG, which, in the investigator's opinion, is clinically meaningful. * Chronic or current active infectious disease requiring systemic antibiotics or antifungal or antiviral treatment within 2 weeks prior to enrollment (participants with asymptomatic chronic infections on prophylactic treatment are allowed). Note: HIV-positive participants are allowed if all of the following criteria are met: CD4+ count ≥ 300/µL, undetectable viral load, receiving antiretroviral therapy that does not interact with study drug, and no HIV/AIDS-associated opportunistic infection in the last 12 months. * Use of any potent CYP3A4 inhibitors or inducers or moderate CYP3A4 inducers within 14 days or 5 half-lives (whichever is longer) before the first dose of study treatment. Note: Moderate CYP3A4 inhibitors are not prohibited * Known hypersensitivity or severe reaction to pemigatinib, gemcitabine, cisplatin, or their excipients. * Inadequate recovery from toxicity and/or complications from a major surgery before starting therapy.

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Conditions

The condition(s) this trial relates to.

cholangiocarcinoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Metastatic cholangiocarcinoma Unresectable cholangiocarcinoma

Contacts and locations

Locations

A.O.U. Di Modena - Policlinico

Modena, 41124, Italy
Aberdeen Royal Infirmary

Aberdeen, AB25 2ZN, United Kingdom
Addenbrookes Hospital

Cambridge, CB2 0QQ, United Kingdom
Aichi Cancer Center Hospital

Nagoya, 464-8681, Japan
Allgemeines Krankenhaus Der Stadt Wien

Vienna, 01090, Austria
Amsterdam University Medical Centre

Amsterdam, 1100 DD, Netherlands
Asklepios Klinik Altona

Hamburg, 22763, Germany
Aurora Research Institute

Wauwatosa, Wisconsin, 53226, United States
Az Groeninge Campus Kennedylaan

Kortrijk, 08500, Belgium
Azienda Ospedaliera San Camillo Forlanini

Rome, 00152, Italy
Azienda Ospedaliera Universitaria Pisana

Pisa, 56126, Italy
Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi

Bologna, 40138, Italy
Azienda Ospedaliera Universitaria San Luigi Gonzaga Orbassano

Orbassano, 10043, Italy
Azienda Ospedaliera Universitaria Senese Policlinico Santa Maria Alle Scotte

Siena, 53100, Italy
Azienda Ospedaliero Universitaria Delle Marche

Ancona, 60126, Italy
Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda

Milan, 20162, Italy
Barbara Ann Karmanos Cancer Hospital

Detroit, Michigan, 48201, United States
Baylor Scott and White Research Institute

Dallas, Texas, 75246, United States
Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States
Boston Medical Center

Boston, Massachusetts, 02118, United States
Cancer Institute Hospital of Jfcr

Kōtoku, 135-8550, Japan
Cancercare Manitoba

Winnipeg, Manitoba, R3E 0V9, Canada
Centre Antoine Laccassagne

Nice, 06189, France
Centre Hospitalier Universitaire de Bordeaux - Hospital Haut-Leveque

Pessac, 336600, France
Centre Hospitalier Universitaire de Nantes

Nantes, 44000, France
Centre de Lutte Contre Le Cancer - Institut de Cancerologie de L'Ouest - Rene Gauducheau

Saint-Herblain, 44800, France
Centro Ricerche Cliniche Di Verona (Crc)

Verona, 37134, Italy
Charite - Campus Virchow-Klinikum

Berlin, 13353, Germany
Charite Universitaetsmedizin Berlin - Campus Charite Mitte

Berlin, 13353, Germany
Chiba Cancer Center

Chiba, 260-8717, Japan
Chiba University Hospital

Chiba, 260-8677, Japan
Chu Besancon Hospital Jean Minjoz

Besançon, 25030, France
Chu Hopital de La Timone

Marseille, 13385, France
Chu Toulouse Hopital Rangueil

Toulouse, 31059, France
Chu Ucl Namur University Hospital Mont-Godinne

Yvoir, 05530, Belgium
Chu Vandoeuvre-Les-Nancy Hopital Brabois

Vandœuvre-lès-Nancy, 54511, France
Chu de Limoges - Hospital Dupuytren

Limoges, 87042, France
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Clinica Universidad de Navarra (Cun)

Pamplona, 31008, Spain
Comitato Etico Fondazione Irccs Istituto Nazionale Dei Tumori Milano

Milan, 20133, Italy
Comprehensive Cancer Centers of Nevada-Twain

Las Vegas, Nevada, 89169, United States
Docrates Cancer Center

Helsinki, 00180, Finland
Erasmus Medical Center

Rotterdam, 3015 CE, Netherlands
European Institute of Oncology

Milan, 20141, Italy
Fondazione Del Piemonte Per L'Oncologia Ircc Candiolo

Candiolo, 10060, Italy
Fondazione Policlinico Universitario Agostino Gemelli Irccs

Roma, 00137, Italy
Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States
Fujian Cancer Hospital

Fuzhou, 350005, China
Georgetown University-Lombardi Comprehensive Cancer Center

Washington D.C., District of Columbia, 20007, United States
Greenville Hospital System University Medical Center Institute For Translational Oncology Research

Greenville, South Carolina, 29605, United States
HOSPITAL UNiVERSITARIO DONOSTIA

Donostia / San Sebastian, 20014, Spain
Hadassah University Hospital

Jerusalem, 9112001, Israel
Hannover Medical School

Hanover, 30625, Germany
Heilongjiang Province Cancer Hospital

Harbin, 150081, China
Helsinki University Central Hospital

Helsinki, 00290, Finland
Henry Ford Hospital

Detroit, Michigan, 48202, United States
Herlev Og Gentofte Hospital

Herlev, 02730, Denmark
Hiroshima University Hospital

Hiroshima, 734-8551, Japan
Hokkaido University Hospital

Sapporo, 060-8648, Japan
Hopital Beaujon

Clichy, 92110, France
Hopital Charles Nicolle Chu Rouen Hospital de Bois-Guillaume

Rouen, 76031, France
Hopital Europeen Georges Pompidou (Hegp)

Paris, 75015, France
Hopital Prive Jean Mermoz

Lyon, 69373, France
Hospital Clinic Barcelona Main

Barcelona, 08036, Spain
Hospital General Universitario Gregorio Maranon

Madrid, 28007, Spain
Hospital General Universitario Vall D Hebron

Barcelona, 08035, Spain
Hospital General Universitario de Valencia

Valencia, 46014, Spain
Hospital Materno Teresa Herrera

A Coruña, 15006, Spain
Hospital Regional Universitario de Malaga

Málaga, 29010, Spain
Hospital Universitaire Pitie-Salpetriere

Paris, 75013, France
Hospital Universitari Parc Tauli

Sabadell, 08208, Spain
Hospital Universitario Hm Sanchinarro

Madrid, 28050, Spain
Hospital Universitario Marques de Valdecilla

Santander, 39008, Spain
Hospital Universitario Ramon Y Cajal

Madrid, 28034, Spain
Hospital Universitario Reina Sofia

Córdoba, 14004, Spain
Hospital de Jolimont

Haine-st-paul, 07100, Belgium
Hospital de La Miletrie

Poitiers, 86021, France
Hospital de La Santa Creu I Sant Pau

Barcelona, 08026, Spain
Houston Methodist Research Institute

Houston, Texas, 77030, United States
Hubei Cancer Hospital

Wuhan, 430079, China
Hyogo College of Medicine Hospital

Hyōgo, 663-8501, Japan
Icahn School of Medicine At Mount Sinai

New York, New York, 10029, United States
Imperial College Healthcare Nhs Trust - Hammersmith Hospital

London, W12 0HS, United Kingdom
Innsbruck University Hospital

Innsbruck, A-6020, Austria
Inselspital - Universitaetsspital Bern

Bern, 03010, Switzerland
Institut Bergonie

Bordeaux, 33076, France
Institut Gustave Roussy

Villejuif, 94805, France
Institut Sainte Catherine

Avignon, 84918, France
Iov - Istituto Oncologico Veneto Irccs

Padova, 35128, Italy
Irccs Azienda Ospedaliera Universitaria San Martino

Genova, 16132, Italy
Irccs Istituto Clinico Humanitas

Rozzano, 20089, Italy
Istituto Di Ricovero E Cura A Carattere Scientifico (Irccs) Ospedale San Raffaele

Milan, 20132, Italy
Istituto Nazionale Tumori Irccs Fondazione Pascale

Naples, 80131, Italy
Istituto Nazionale Tumori Regina Elena Irccs

Roma, 00144, Italy
Istituto Tumori Giovanni Paolo Ii Irccs Ospedale Oncologico Bari

Bari, 70124, Italy
Jiangsu Province Hospital

Nanjing, 210029, China
Jichi Medical University Hospital

Shimotsuke-shi, 329-0498, Japan
Johns Hopkins Oncology Center

Baltimore, Maryland, 21287, United States
Kanagawa Cancer Center

Yokohama, 241-8515, Japan
Kanazawa University Hospital

Ishikawa, 920-8641, Japan
Karmanos Cancer Institute

Farmington Hills, Michigan, 48334, United States
Karolinska Institute Universitetssjukhuset Solna

Solna, 171 64, Sweden
Keio University Hospital

Shinjuku-ku, 160-8582, Japan
Kent Oncology Centre - Maidstone Hospital

Maidstone, ME16 9QQ, United Kingdom
Kindai University Hospital

Sayama, 589-8511, Japan
Klinikum Bremen-Nord

Bremen, 28755, Germany
Klinikum Der Johann Wolfgang Goethe University

Frankfurt am Main, 60590, Germany
Klinikum Ludwigsburg

Ludwigsburg, 71640, Germany
Klinikum Nuernberg

Nuremberg, 90419, Germany
Kobe University Hospital

Kobe, 650-0017, Japan
Kunming 1St People'S Hospital

Kunming, 650032, China
Kyorin University Hospital

Mitaka-shi, 181-8611, Japan
Kyoto University Hospital

Kyoto, 606-8507, Japan
Kyushu University Hospital

Fukuoka, 812-8582, Japan
Landeskrankenhaus Steyr

Steyr, 04400, Austria
Landeskrankenhaus Universitatsklinikum Graz

Graz, 08036, Austria
Maastricht Umc+

Maastricht, 6202 AZ, Netherlands
Marin Cancer Care

Greenbrae, California, 94904, United States
Mayo Clinic

Rochester, Minnesota, 55905, United States
Mayo Clinic Arizona

Phoenix, Arizona, 85054, United States
Mayo Clinic-Florida

Jacksonville, Florida, 32224, United States
McGill University Health Centre Research Institute

Montreal, Quebec, H4A 3J1, Canada
Mount Sinai Medical Center Comprehensive Cancer Center

Miami Beach, Florida, 33140, United States
National Hospital Organization Kyushu Cancer Center

Fukuoka, 811-1395, Japan
Nho Shikoku Cancer Center

Matsuyama, 791-0280, Japan
Niigata Cancer Center Hospital

Niigata, 951-8566, Japan
Northern Jiangsu Peoples Hospital

Yangzhou, 225001, China
Nova Scotia Health Authority/Qeii Health Sciences Centre

Halifax, Nova Scotia, B3H 4K4, Canada
Ochsner Clinic Foundation Ocf Ochsner Cancer Institute Oci

New Orleans, Louisiana, 70121, United States
Oita University Hospital

Yufu-shi, 879-5593, Japan
Ordensklinikum Krankenhaus Der Barmherzigen Schwestern Linz

Linz, 04010, Austria
Oregon Health & Science University

Portland, Oregon, 97239, United States
Osaka International Cancer Institute

Osaka, 541-8567, Japan
Osaka University Hospital

Suita-shi, 565-0871, Japan
Oslo University Hospital

Oslo, 00450, Norway
Ospedale Degli Infermi - Faenza

Faenza, 48018, Italy
Ospedale Papa Giovanni Xxiii

Bergamo, 24127, Italy
Otto-Von-Guericke-Universitat Magdeburg

Magdeburg, 39120, Germany
Parkview Research Center

Fort Wayne, Indiana, 46845, United States
Peking Union Medical College Hospital

Beijing, 100032, China
Presidio Ospedaliero Garibaldi Nesima

Catania, 95100, Italy
Presidio Ospedaliero Pescara

Pescara, 65124, Italy
Princess Margaret Cancer Center

Toronto, Ontario, MG5 2M9, Canada
Providence Portland Med. Ctr

Portland, Oregon, 97213, United States
Rabin Medical Center - Beilinson Hospital

Petah Tikva, 4941492, Israel
Rambam Health Care Campus

Haifa, 3109601, Israel
Riverside Regional Medical Center

Newport News, Virginia, 23601, United States
Saitama Cancer Center

Saitama, 362-0806, Japan
Salzburger Universitatsklinikum

Salzburg, 05020, Austria
San Bartolo Hospital

Vicenza, 36100, Italy
Shizuoka Cancer Center

Shizuoka, 411-8777, Japan
Shulan Hangzhou Hospital Co Ltd

Hangzhou, 310000, China
Sichuan Cancer Hospital

Sichuan, 610041, China
Soroka University Medical Center

Beersheba, 84001, Israel
St. Vincent'S University Hospital

Dublin, D04 Y8V0, Ireland
Summit Medical Group

Florham Park, New Jersey, 07932, United States
Sun Yat-Sen Memorial Hospital Sun Yat-Sen University

Guangdong, 510000, China
Sun Yat-Sen Memorial Hospital Sun Yat-Sen University

Guangzhou, 510000, China
Tampere University Hospital

Tampere, 33521, Finland
Teikyo University Hospital

Itabashi-ku, 173-8606, Japan
Tel Aviv Sourasky Medical Center

Tel Aviv, 64239, Israel
The Affiliated Hospital of Qingdao University

Shandong, 266071, China
The Christie Nhs Foundation Trust

Manchester, M20 4BV, United Kingdom
The Royal Marsden Nhs Foundation Trust - Chelsea

London, SW3 6JJ, United Kingdom
The Royal Marsden Nhs Foundation Trust - Sutton

Sutton, SM2 5PT, United Kingdom
The Second Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, 310009, China
The University of Kansas Cancer Center

Westwood, Kansas, 66205, United States
Tianjin Medical University Cancer Institute Hospital

Tianjin, 300060, China
Tohoku University Hospital

Sendai, 980-8574, Japan
Tom Baker Cancer Centre

Calgary, Alberta, T2N 4N2, Canada
Tongji Hospital Huazhong University of Science and Technology

Wuhan, 430030, China
Toyama University Hospital

Toyama, 930-0194, Japan
UC Irvine Medical Center

Orange, California, 92868-3201, United States
Ulb Hospital Erasme

Brussels, 01070, Belgium
Umc Utrecht

Utrecht, 3584 CX, Netherlands
Universita Campus Bio Medico Di Roma

Rome, 00128, Italy
Universita Degli Studi Della Campania Luigi Vanvitelli U.O.C. Oncologia Medica

Naples, 80138, Italy
Universitaetsklinikum Des Saarlandes

Homburg / SAAR, 66421, Germany
Universitaetsklinikum in Tubingen

Tübingen, 72076, Germany
Universitair Ziekenhuis Brussel

Brussels, 01090, Belgium
Universitair Ziekenhuis Gent

Ghent, 09000, Belgium
Universitaire Ziekenhuis Leuven - Gasthuisberg

Leuven, 03000, Belgium
Universitatkinikums Ulm

Ulm, 89081, Germany
Universitatsklinikum Bonn Aoer

Bonn, 53127, Germany
Universitatsklinikum Hamburg Eppendorf

Hamburg, 20246, Germany
Universitatsklinikum Koln

Cologne, 50937, Germany
Universitatsklinikum Leipzig Aor

Leipzig, 04103, Germany
Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Iii

Mainz, 55131, Germany
Universitatsspital Zurich

Zurich, 08091, Switzerland
University Clinic Carl Gustav Carus Technical University Dresden

Dresden, 01307, Germany
University College London Hospitals (Uclh)

London, WC1E 6BT, United Kingdom
University Hospital Coventry and Warwickshire

Coventry, CV2 2DX, United Kingdom
University Hospital Grosshadern Munich

Munich, 81377, Germany
University Hospital Tuebingen

Tübingen, 72076, Germany
University Hospital of Saint Etienne

Saint-Etienne, 42055, France
University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States
University Medical Center Freiburg

Freiburg im Breisgau, 79106, Germany
University Medical Center Rwth Aachen

Aachen, 52074, Germany
University of Chicago Medical Center

Chicago, Illinois, 60637-1447, United States
University of Iowa Hospital and Clinics

Iowa City, Iowa, 52242, United States
University of Science and Technology of China-First Affiliated Hospital (Anhui Provincial Hospital)

Hefei, 230001, China
University of Tokyo Hospital

Bunkyō City, 113-8655, Japan
Velindre Cancer Centre

Cardiff, CF14 2TL, United Kingdom
Virginia Commonwealth University

Richmond, Virginia, 23229, United States
Virginia Mason Medical Center

Seattle, Washington, 98101, United States
Wakayama Medical University Hospital

Wakayama, 641-8509, Japan
West China Hospital Sichuan University

Chengdu, 610041, China
West Virginia University Cancer Institute

Morgantown, West Virginia, 26506, United States
White Plains Hospital

White Plains, New York, 10601, United States
Winship Cancer Institute of Emory University

Atlanta, Georgia, 30322, United States
Xinhua Hospital

Shanghai, 200092, China
Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, 200092, China
Yamaguchi University Hospital

Ube, 755-8505, Japan
Yokohama City University Medical Center

Yokohama, 232-0024, Japan
Zhongshan Hospital Fudan University

Shanghai, 200032, China

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