NCT02924376

Brief Summary

The purpose of this study is evaluate the efficacy of pemigatinib in subjects with advanced/metastatic or surgically unresectable cholangiocarcinoma with FGFR2 translocation who have failed at least 1 previous treatment.

Trial Health

98
On Track

Trial Health Score

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

Strong global presence with extensive site network
Enrollment
147

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Jan 2017

Longer than P75 for phase_2

Geographic Reach
12 countries

120 active sites

Status
completed

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

October 4, 2016

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 5, 2016

Completed
3 months until next milestone

Study Start

First participant enrolled

January 16, 2017

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2022

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

February 23, 2023

Completed
Last Updated

August 14, 2025

Status Verified

August 1, 2025

Enrollment Period

5 years

First QC Date

October 4, 2016

Results QC Date

January 26, 2023

Last Update Submit

August 12, 2025

Conditions

Keywords

Cholangiocarcinomafibroblast growth factor (FGF)fibroblast growth factor receptor (FGFR)FGF/FGFR alterations

Outcome Measures

Primary Outcomes (1)

  • Objective Response Rate (ORR) in Participants With FGFR2 Rearrangements or Fusions

    ORR was defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR) at any post-Baseline visit prior to first progressive disease (PD), per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1). CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.

    up to 1527 days

Secondary Outcomes (12)

  • ORR in Participants FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions

    up to 424 days

  • ORR in All Participants With FGF/FGFR Alterations

    up to 1527 days

  • ORR in Participants Negative for FGF/FGFR Alterations

    up to 143 days

  • Progression-free Survival (PFS)

    up to 50.17 months

  • Duration of Response (DOR)

    up to 47.11 months

  • +7 more secondary outcomes

Study Arms (3)

Cohort A Pemigatinib

EXPERIMENTAL

Pemigatinib in subjects with FGFR2 translocation with a documented fusion partner in central laboratory report

Drug: Pemigatinib

Cohort B Pemigatinib

EXPERIMENTAL

Pemigatinibin subjects with other FGF/FGFR alterations

Drug: Pemigatinib

Cohort C Pemigatinib

EXPERIMENTAL

Pemigatinib in subjects negative for FGF/FGFR alteration

Drug: Pemigatinib

Interventions

Pemigatinibonce a day by mouth for 2 consecutive weeks and 1 week off therapy

Also known as: INCB054828
Cohort A PemigatinibCohort B PemigatinibCohort C Pemigatinib

Eligibility Criteria

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

You may qualify if:

  • Histologically or cytologically confirmed cholangiocarcinoma.
  • Radiographically measurable or evaluable disease per RECIST v1.1.
  • Tumor assessment for FGF/FGFR gene alteration status.
  • Documented disease progression after at least 1 line of prior systemic therapy.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
  • Life expectancy ≥ 12 weeks.

You may not qualify if:

  • Prior receipt of a selective FGFR inhibitor.
  • History of and/or current evidence of ectopic mineralization/calcification, including but not limited to soft tissue, kidneys, intestine, myocardia, or lung, excepting calcified lymph nodes and asymptomatic arterial or cartilage/tendon calcifications.
  • Current evidence of clinically significant corneal or retinal disorder confirmed by ophthalmologic examination.
  • Use of any potent CYP3A4 inhibitors or inducers within 14 days or 5 half-lives, whichever is shorter, before the first dose of study drug. Topical ketoconazole will be allowed.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (120)

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Anchorage, Alaska, United States

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Phoenix, Arizona, United States

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Tempe, Arizona, United States

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Tucson, Arizona, United States

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Orange, California, United States

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San Francisco, California, United States

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Aurora, Colorado, United States

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Denver, Colorado, United States

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New Haven, Connecticut, United States

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Newark, Delaware, United States

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

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Gainesville, Florida, United States

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Miami Beach, Florida, United States

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Chicago, Illinois, United States

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Evanston, Illinois, United States

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Fort Wayne, Indiana, United States

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Sioux City, Iowa, United States

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Westwood, Kansas, United States

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Louisville, Kentucky, United States

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Baltimore, Maryland, United States

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Ann Arbor, Michigan, United States

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Detroit, Michigan, United States

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Rochester, Minnesota, United States

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Woodbury, Minnesota, United States

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St Louis, Missouri, United States

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Billings, Montana, United States

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Omaha, Nebraska, United States

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Morristown, New Jersey, United States

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New York, New York, United States

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Rochester, New York, United States

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Charlotte, North Carolina, United States

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Goldsboro, North Carolina, United States

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Cincinnati, Ohio, United States

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Cleveland, Ohio, United States

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Portland, Oregon, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Sioux Falls, South Dakota, United States

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Arlington, Texas, United States

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Dallas, Texas, United States

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Grapevine, Texas, United States

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San Antonio, Texas, United States

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The Woodlands, Texas, United States

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Tyler, Texas, United States

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Waco, Texas, United States

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Salt Lake City, Utah, United States

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Fairfax, Virginia, United States

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Seattle, Washington, United States

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Madison, Wisconsin, United States

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Brussels, Belgium

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Ghent, Belgium

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Kortrijk, Belgium

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Leuven, Belgium

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Montpellier, Herault, France

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Villejuif, Herault, France

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Avignon, France

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Clichy, France

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Paris, France

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Toulouse, France

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Berlin, Germany

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Bonn, Germany

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Dresden, Germany

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Hanover, Germany

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Homburg, Germany

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Leipzig, Germany

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Mainz, Germany

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Tübingen, Germany

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Jerusalem, Israel

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Petah Tikva, Israel

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Ramat Gan, Israel

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Tel Aviv, Israel

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Bari, Castellana Grotte, Italy

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San Giovanni Rotondo, Foggia, Italy

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Bergamo, Italy

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Bologna, Italy

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Candiolo, Italy

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Catania, Italy

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Faenza, Italy

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Lecce, Italy

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Meldola, Italy

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Milan, Italy

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Napoli, Italy

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Rimini, Italy

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Siena, Italy

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Verona, Italy

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Nagoya, Aichi-ken, Japan

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Chiba, Chiba, Japan

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Fukuoka, Fukuoka, Japan

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Yokohama, Kanagawa, Japan

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Kyoto, Kyoto, Japan

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Osaka, Osaka, Japan

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Osakasayama-shi, Osaka, Japan

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Kitaadachi, Saitama, Japan

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Sunto, Shizuoka, Japan

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Kōtoku, Tokyo-To, Japan

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Shinjuku-ku, Tokyo-To, Japan

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Goyang-si, South Korea

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Seongnam-si, South Korea

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Seoul, South Korea

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Pamplona, Navarre, Spain

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Barcelona, Spain

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Girona, Spain

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Madrid, Spain

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Taichung, Taiwan

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Tainan, Taiwan

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Taipei, Taiwan

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Chiang Mai, Muang, Thailand

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Khon Kaen, Muang, Thailand

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Udon Thani, Muang, Thailand

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Bangkok, Patumwan, Thailand

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Bangkoknoi, Thailand

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Cambridge, Cambridgeshire, United Kingdom

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Bournemouth, Dorset, United Kingdom

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Aberdeen, Grampian Region, United Kingdom

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London, Greater London, United Kingdom

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Cardiff, United Kingdom

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Glasgow, United Kingdom

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Manchester, United Kingdom

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Metropolitan Borough of Wirral, United Kingdom

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Sheffield, United Kingdom

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Related Publications (3)

  • Patel TH, Marcus L, Horiba MN, Donoghue M, Chatterjee S, Mishra-Kalyani PS, Schuck RN, Li Y, Zhang X, Fourie Zirkelbach J, Charlab R, Liu J, Yang Y, Lemery SJ, Pazdur R, Theoret MR, Fashoyin-Aje LA. FDA Approval Summary: Pemigatinib for Previously Treated, Unresectable Locally Advanced or Metastatic Cholangiocarcinoma with FGFR2 Fusion or Other Rearrangement. Clin Cancer Res. 2023 Mar 1;29(5):838-842. doi: 10.1158/1078-0432.CCR-22-2036.

  • Bibeau K, Feliz L, Lihou CF, Ren H, Abou-Alfa GK. Progression-Free Survival in Patients With Cholangiocarcinoma With or Without FGF/FGFR Alterations: A FIGHT-202 Post Hoc Analysis of Prior Systemic Therapy Response. JCO Precis Oncol. 2022 Apr;6:e2100414. doi: 10.1200/PO.21.00414.

  • Abou-Alfa GK, Sahai V, Hollebecque A, Vaccaro G, Melisi D, Al-Rajabi R, Paulson AS, Borad MJ, Gallinson D, Murphy AG, Oh DY, Dotan E, Catenacci DV, Van Cutsem E, Ji T, Lihou CF, Zhen H, Feliz L, Vogel A. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study. Lancet Oncol. 2020 May;21(5):671-684. doi: 10.1016/S1470-2045(20)30109-1. Epub 2020 Mar 20.

MeSH Terms

Conditions

CholangiocarcinomaAcrocephalosyndactylia

Interventions

pemigatinib

Condition Hierarchy (Ancestors)

AdenocarcinomaCarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeNeoplasmsCraniosynostosesSynostosisDysostosesBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesSyndactylyCraniofacial AbnormalitiesMusculoskeletal AbnormalitiesLimb Deformities, CongenitalCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title
Study Director
Organization
Incyte Corporation

Study Officials

  • Luis Féliz Vinas, MD

    Incyte Corporation

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

October 4, 2016

First Posted

October 5, 2016

Study Start

January 16, 2017

Primary Completion

February 1, 2022

Study Completion

February 1, 2022

Last Updated

August 14, 2025

Results First Posted

February 23, 2023

Record last verified: 2025-08

Data Sharing

IPD Sharing
Will share

Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
Access Criteria
Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.
More information

Locations