Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1-3 Alterations

Last updated: October 3, 2025
Sponsor: Incyte Corporation
Overall Status: Terminated

Phase

2

Condition

Brain Cancer

Cancer/tumors

Gliomas

Treatment

Pemigatinib

Clinical Study ID

NCT05267106
INCB 54828-209
2021-004740-24
  • Ages 18-99
  • All Genders

Study Summary

This is an open-label, monotherapy study of pemigatinib in participants with recurrent glioblastoma (GBM) or other recurrent gliomas, circumscribed astrocytic gliomas, and glioneuronal and neuronal tumors with an activating FGFR1-3 mutation or fusion/rearrangement. This study consists of 2 cohorts, Cohorts A, and B, and will enroll approximately 82 participants into each cohort. Participants will receive pemigatinib 13.5 mg QD on a 2-week on-therapy and 1-week off-therapy schedule as long as they are receiving benefit and have not met any criteria for study withdrawal.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Histological, cytological, or molecular confirmation of recurrent GBM or otherglioma, circumscribed astrocytic glioma, or glioneuronalor neuronal tumors that hasrecurred.

  • Radiographically measurable disease.

. -Karnofsky performance status ≥ 60.

  • Life expectancy ≥ 12 weeks.

  • Documentation of an actionable FGFR1-3 gene mutation or fusion/rearrangement fromtissue : FGFR1-3 fusions or other rearrangements (FGFR1-3 in-frame fusions, anyFGFR2 rearrangement, or FGFR1/3 rearrangement with known partner) or a definedFGFR1-3 activating mutation or in-frame deletion. Only participants with FGFRfusions or rearrangements with an intact kinase domain are eligible.

  • MRI-documented objective progression after prior therapy and must have no therapyavailable that is likely to provide clinical benefit.

  • Most recent archival tumor specimen must be a tumor block or a minimum of 15unstained slides from biopsy or resection of primary tumor or metastasis.

  • Willingness to avoid pregnancy or fathering children.

Exclusion

Exclusion Criteria:

  • Prior receipt of an FGFR inhibitor.

  • Receipt of anticancer medications or investigational drugs for any indication orreason within 28 days before first dose of study drug.

  • Participants may have had treatment for an unlimited number of prior relapses butmust not have had prior bevacizumab or other VEGF/VEGFR inhibitors (exception: priorbevacizumab is allowed if it was administered for the treatment of radiationnecrosis rather than progressive tumor and was stopped at least 12 weeks prior toMRI showing tumor progression).

  • Concurrent anticancer therapy

  • Candidate for potentially curative surgery.

  • Dexamethasone (or equivalent) > 4 mg daily at the time of study registration

  • Current evidence of clinically significant corneal or retinal disorder as confirmedby ophthalmologic examination.

  • Diffuse leptomeningeal disease.

  • Radiation therapy administered within 12 weeks before enrollment/first dose of studydrug.

  • Known additional malignancy that is progressing or requires active systemictreatment.

Study Design

Total Participants: 83
Treatment Group(s): 1
Primary Treatment: Pemigatinib
Phase: 2
Study Start date:
May 20, 2022
Estimated Completion Date:
December 17, 2024

Connect with a study center

  • Aalborg Universitets Hospital

    Aalborg, 09000
    Denmark

    Site Not Available

  • Aalborg Universitets Hospital

    Aalborg 2624886, 09000
    Denmark

    Site Not Available

  • Aarhus University Hospital

    Aarhus, 08200
    Denmark

    Site Not Available

  • Rigshospitalet Uni of Hospital of Copenhagen

    Copenhagen, 02100
    Denmark

    Site Not Available

  • Rigshospitalet Uni of Hospital of Copenhagen

    Copenhagen 2618425, 02100
    Denmark

    Site Not Available

  • Odense University Hospital

    Odense C, 05000
    Denmark

    Site Not Available

  • Odense University Hospital

    Odense C 11746507, 05000
    Denmark

    Site Not Available

  • Chru de Lille Hopital Claude Huriez

    Lille, 59037
    France

    Site Not Available

  • Chru de Lille Hopital Claude Huriez

    Lille 2998324, 59037
    France

    Site Not Available

  • Chru de Lille Hopital Claude Huriez

    Lille Cedex, 59037
    France

    Site Not Available

  • Chu Hopital de La Timone

    Marseille, 13005
    France

    Site Not Available

  • Chu Hopital de La Timone

    Marseille 2995469, 13005
    France

    Site Not Available

  • Chu Hopital de La Timone

    Marseille Cedex 05, 13385
    France

    Site Not Available

  • Hospital Universitaire Pitie-Salpetriere

    Paris, 75013
    France

    Site Not Available

  • Hospital Universitaire Pitie-Salpetriere

    Paris 2988507, 75013
    France

    Site Not Available

  • Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole

    Toulouse, 31059
    France

    Site Not Available

  • Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole

    Toulouse 2972315, 31059
    France

    Site Not Available

  • Universitatsklinikum Bonn Aoer

    Bonn, 53127
    Germany

    Site Not Available

  • Universitatsklinikum Bonn Aoer

    Bonn 2946447, 53127
    Germany

    Site Not Available

  • Klinikum Der Johann Wolfgang Goethe University

    Frankfurt, 60528
    Germany

    Site Not Available

  • Klinikum Der Johann Wolfgang Goethe University

    Frankfurt 2925536, 60528
    Germany

    Site Not Available

  • Universitaetsklinikum Heidelberg

    Heidelberg, 69120
    Germany

    Site Not Available

  • Universitaetsklinikum Heidelberg

    Heidelberg 2907911, 69120
    Germany

    Site Not Available

  • University Hospital Grosshadern Munich

    Muenchen, 81377
    Germany

    Site Not Available

  • University Hospital Tuebingen

    Tuebingen, 72076
    Germany

    Site Not Available

  • University Hospital Tuebingen

    Tübingen 2820860, 72076
    Germany

    Site Not Available

  • Ospedale Bellaria

    Bologna, 40139
    Italy

    Site Not Available

  • Ospedale Bellaria

    Bologna 3181928, 40139
    Italy

    Site Not Available

  • Istituto Di Ricovero E Cura A Carattere Scientifico (Irccs) Ospedale San Raffaele

    Milan 6951411, 20132
    Italy

    Site Not Available

  • Istituto Di Ricovero E Cura A Carattere Scientifico (Irccs) Ospedale San Raffaele

    Milano, 20132
    Italy

    Site Not Available

  • Iov - Istituto Oncologico Veneto Irccs

    Padova, 35128
    Italy

    Site Not Available

  • Iov - Istituto Oncologico Veneto Irccs

    Padua 3171728, 35128
    Italy

    Site Not Available

  • A.S.L. Napoli 1 Centro Ospedale Del Mare

    Ponticelli, 80147
    Italy

    Site Not Available

  • A.S.L. Napoli 1 Centro Ospedale Del Mare

    Ponticelli 3170206, 80147
    Italy

    Site Not Available

  • Irccs Istituto Clinico Humanitas

    Rozzano, 20089
    Italy

    Site Not Available

  • Irccs Istituto Clinico Humanitas

    Rozzano 3168837, 20089
    Italy

    Site Not Available

  • Azienda Ospedaliero Universitaria Citta Della Salute E Della Scienza

    Torino, 10124
    Italy

    Site Not Available

  • Azienda Ospedaliero Universitaria Citta Della Salute E Della Scienza

    Torino 8980539, 10124
    Italy

    Site Not Available

  • University of Tokyo Hospital

    Bunkyo-ku, 113-8655
    Japan

    Site Not Available

  • University of Tokyo Hospital

    Bunkyō City 11790632, 113-8655
    Japan

    Site Not Available

  • National Cancer Center Hospital

    Chuo-ku, 104-0045
    Japan

    Site Not Available

  • National Cancer Center Hospital

    Chūōku 10262791, 104-0045
    Japan

    Site Not Available

  • Kyushu University Hospital

    Fukuoka 1863967, 812-8582
    Japan

    Site Not Available

  • Kyushu University Hospital

    Fukuoka-shi, 812-8582
    Japan

    Site Not Available

  • Kyoto University Hospital

    Kyoto 1857910, 606-8507
    Japan

    Site Not Available

  • Kyoto University Hospital

    Kyoto-shi, 606-8507
    Japan

    Site Not Available

  • Nagoya University Hospital

    Nagoya 1856057, 466-8560
    Japan

    Site Not Available

  • Nagoya University Hospital

    Nagoya-shi, 466-8560
    Japan

    Site Not Available

  • Tohoku University Hospital

    Sendai 2111149, 980-8574
    Japan

    Site Not Available

  • Tohoku University Hospital

    Sendai-shi, 980-8574
    Japan

    Site Not Available

  • Netherlands Cancer Institute Antoni Van Leeuwenhoek Ziekenhuis

    Amsterdam, 1066 CX
    Netherlands

    Site Not Available

  • Netherlands Cancer Institute Antoni Van Leeuwenhoek Ziekenhuis

    Amsterdam 2759794, 1066 CX
    Netherlands

    Site Not Available

  • Erasmus Mc Cancer Institute

    Rotterdam, 3015 GD
    Netherlands

    Site Not Available

  • Erasmus Mc Cancer Institute

    Rotterdam 2747891, 3015 GD
    Netherlands

    Site Not Available

  • University Medical Center Utrecht

    Utrecht, 3584 CX
    Netherlands

    Site Not Available

  • Hospital Clinic Barcelona Main

    Barcelona, 08036
    Spain

    Site Not Available

  • Hospital Clinic de Barcelona

    Barcelona, 08036
    Spain

    Active - Recruiting

  • Hospital Del Mar

    Barcelona, 08003
    Spain

    Site Not Available

  • Hospital General Universitario Vall D Hebron

    Barcelona, 08035
    Spain

    Site Not Available

  • Hospital de La Santa Creu I Sant Pau

    Barcelona, 08041
    Spain

    Site Not Available

  • Ico Institut Catala D Oncologia

    Barcelona, 08908
    Spain

    Site Not Available

  • Hospital Clinic Barcelona Main

    Barcelona 3128760, 08036
    Spain

    Site Not Available

  • Hospital Del Mar

    Barcelona 3128760, 08003
    Spain

    Site Not Available

  • Hospital General Universitario Vall D Hebron

    Barcelona 3128760, 08035
    Spain

    Site Not Available

  • Hospital de La Santa Creu I Sant Pau

    Barcelona 3128760, 08041
    Spain

    Site Not Available

  • Ico Girona Hospital Universitari de Girona Dr Josep Trueta

    Girona, 17007
    Spain

    Site Not Available

  • Ico Girona Hospital Universitari de Girona Dr Josep Trueta

    Girona 3121456, 17007
    Spain

    Site Not Available

  • Ico Institut Catala D Oncologia

    L'Hospitalet de Llobregat 3120619, 08908
    Spain

    Site Not Available

  • Ico Institut Catala D Oncologia

    L'hospitalet de Llobregat, 08908
    Spain

    Site Not Available

  • Hospital General Universitario Gregorio Maranon

    Madrid, 28007
    Spain

    Site Not Available

  • Hospital Universitario 12 de Octubre

    Madrid, 28041
    Spain

    Site Not Available

  • Hospital Universitario Hm Sanchinarro

    Madrid, 28050
    Spain

    Site Not Available

  • Hospital Universitario Ramon Y Cajal

    Madrid, 28034
    Spain

    Site Not Available

  • Hospital General Universitario Gregorio Maranon

    Madrid 3117735, 28007
    Spain

    Site Not Available

  • Hospital Universitario 12 de Octubre

    Madrid 3117735, 28041
    Spain

    Site Not Available

  • Hospital Universitario Hm Sanchinarro

    Madrid 3117735, 28050
    Spain

    Site Not Available

  • Hospital Universitario Ramon Y Cajal

    Madrid 3117735, 28034
    Spain

    Site Not Available

  • Clinica Universidad de Navarra (Cun)

    Pamplona, 31008
    Spain

    Site Not Available

  • Clinica Universidad de Navarra (Cun)

    Pamplona 3114472, 31008
    Spain

    Site Not Available

  • Hospital General de Catalunya

    Sant Cugat Del Valles, 08190
    Spain

    Site Not Available

  • Hospital General de Catalunya

    Sant Cugat del Vallès 3110718, 08190
    Spain

    Site Not Available

  • Hospital Universitario Virgen Del Rocio

    Sevilla, 41013
    Spain

    Site Not Available

  • Hospital Universitario Virgen Del Rocio

    Seville 2510911, 41013
    Spain

    Site Not Available

  • Hospital General Universitario de Valencia

    Valencia, 46014
    Spain

    Site Not Available

  • Hospital General Universitario de Valencia

    Valencia 2509954, 46014
    Spain

    Site Not Available

  • Addenbrooke'S Hospital

    Cambridge, CB2 0QQ
    United Kingdom

    Site Not Available

  • Addenbrooke'S Hospital

    Cambridge 2653941, CB2 0QQ
    United Kingdom

    Site Not Available

  • Velindre Cancer Centre

    Cardiff, CF14 2TL
    United Kingdom

    Site Not Available

  • Velindre Cancer Centre

    Cardiff 2653822, CF14 2TL
    United Kingdom

    Site Not Available

  • St James'S University Hospital

    Leeds, LS9 7TF
    United Kingdom

    Site Not Available

  • St James'S University Hospital

    Leeds 2644688, LS9 7TF
    United Kingdom

    Site Not Available

  • Guys Hospital

    London, SE1 9RT
    United Kingdom

    Site Not Available

  • The Royal Marsden Nhs Foundation Trust - Chelsea

    London, SW36JJ
    United Kingdom

    Site Not Available

  • The Royal Marsden Nhs Foundation Trust - Sutton

    London, SW3 6JJ
    United Kingdom

    Site Not Available

  • Guys Hospital

    London 2643743, SE1 9RT
    United Kingdom

    Site Not Available

  • The Royal Marsden Nhs Foundation Trust - Chelsea

    London 2643743, SW36JJ
    United Kingdom

    Site Not Available

  • The Royal Marsden Nhs Foundation Trust - Sutton

    London 2643743, SW3 6JJ
    United Kingdom

    Site Not Available

  • The Christie Nhs Foundation Trust Uk

    Manchester, M20 4BX
    United Kingdom

    Site Not Available

  • The Christie Nhs Foundation Trust Uk

    Manchester 2643123, M20 4BX
    United Kingdom

    Site Not Available

  • The Clatterbridge Cancer Centre

    Metropolitan Borough of Wirral 7733088, CH63 4JY
    United Kingdom

    Site Not Available

  • The Clatterbridge Cancer Centre

    Wirral, CH63 4JY
    United Kingdom

    Site Not Available

  • Valkyrie Clinical Trials

    Beverly Hills, California 90211
    United States

    Site Not Available

  • City of Hope National Medical Center

    Duarte, California 91010
    United States

    Site Not Available

  • Providence Medical Foundation

    Fullerton, California 92835
    United States

    Site Not Available

  • Usc Norris Comprehensive Cancer Center

    Los Angeles, California 90033
    United States

    Site Not Available

  • Stanford Neuroscience Health Center

    Sacramento, California 94304
    United States

    Site Not Available

  • Sharp Memorial Hospital

    San Diego, California 92123
    United States

    Site Not Available

  • University of California San Francisco

    San Francisco, California 94143
    United States

    Site Not Available

  • Providence St Joseph Hospital Orange Center For Cancer Prevention and Treatment

    Santa Monica, California 90404
    United States

    Site Not Available

  • Valkyrie Clinical Trials

    Beverly Hills 5328041, California 5332921 90211
    United States

    Site Not Available

  • City of Hope National Medical Center

    Duarte 5344147, California 5332921 91010
    United States

    Site Not Available

  • Providence Medical Foundation

    Fullerton 5351247, California 5332921 92835
    United States

    Site Not Available

  • Usc Norris Comprehensive Cancer Center

    Los Angeles 5368361, California 5332921 90033
    United States

    Site Not Available

  • Stanford Neuroscience Health Center

    Sacramento 5389489, California 5332921 94304
    United States

    Site Not Available

  • Sharp Memorial Hospital

    San Diego 5391811, California 5332921 92123
    United States

    Site Not Available

  • University of California San Francisco

    San Francisco 5391959, California 5332921 94143
    United States

    Site Not Available

  • Providence St Joseph Hospital Orange Center For Cancer Prevention and Treatment

    Santa Monica 5393212, California 5332921 90404
    United States

    Site Not Available

  • Yale Cancer Center

    New Haven, Connecticut 06510-3220
    United States

    Site Not Available

  • Yale Cancer Center

    New Haven 4839366, Connecticut 4831725 06510-3220
    United States

    Site Not Available

  • Lynn Cancer Institute Marcus Neuroscience Institute

    Boca Raton, Florida 33486-2304
    United States

    Site Not Available

  • Baptist Md Anderson Cancer Center

    Jacksonville, Florida 32207
    United States

    Site Not Available

  • Miami Cancer Institute

    Miami, Florida 33176
    United States

    Site Not Available

  • Orlando Health Cancer Institute Downtown Orlando

    Orlando, Florida 32806
    United States

    Site Not Available

  • H. Lee Moffitt Cancer Center and Research Institute Hospital

    Tampa, Florida 33612
    United States

    Site Not Available

  • Tampa General Hospital/University of South Florida

    Tampa, Florida 33606
    United States

    Site Not Available

  • Baptist Md Anderson Cancer Center

    Jacksonville 4160021, Florida 4155751 32207
    United States

    Site Not Available

  • Miami Cancer Institute

    Miami 4164138, Florida 4155751 33176
    United States

    Site Not Available

  • Orlando Health Cancer Institute Downtown Orlando

    Orlando 4167147, Florida 4155751 32806
    United States

    Site Not Available

  • H. Lee Moffitt Cancer Center and Research Institute Hospital

    Tampa 4174757, Florida 4155751 33612
    United States

    Site Not Available

  • Rush University Medical Center-Consultants in Hematology

    Chicago, Illinois 60612
    United States

    Site Not Available

  • University of Chicago

    Chicago, Illinois 60612
    United States

    Site Not Available

  • University of Illinois Hospital & Health Sciences System

    Chicago, Illinois 60612
    United States

    Site Not Available

  • University of Illinois Hospital & Health Sciences System

    Chicago 4887398, Illinois 4896861 60612
    United States

    Site Not Available

  • University of Iowa Hospital and Clinics

    Iowa City, Iowa 52242
    United States

    Site Not Available

  • University of Iowa Hospital and Clinics

    Iowa City 4862034, Iowa 4862182 52242
    United States

    Site Not Available

  • University Medical Center New Orleans

    New Orleans, Louisiana 70112
    United States

    Site Not Available

  • University Medical Center New Orleans

    New Orleans 4335045, Louisiana 4331987 70112
    United States

    Site Not Available

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • University of Minnesota Health Clinics and Surgery Center

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • University of Minnesota Health Clinics and Surgery Center

    Minneapolis 5037649, Minnesota 5037779 55455
    United States

    Site Not Available

  • Washington University

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Northwell Health

    Lake Success, New York 11042
    United States

    Site Not Available

  • Columbia University Irving Medical Center

    New York, New York 10032
    United States

    Site Not Available

  • Memorial Sloan Kettering Cancer Center

    New York, New York 10065
    United States

    Site Not Available

  • Northwell Health

    Lake Success 5123853, New York 5128638 11042
    United States

    Site Not Available

  • Columbia University Irving Medical Center

    New York 5128581, New York 5128638 10032
    United States

    Site Not Available

  • Memorial Sloan Kettering Cancer Center

    New York 5128581, New York 5128638 10065
    United States

    Site Not Available

  • East Carolina University

    Greenville, North Carolina 27834
    United States

    Site Not Available

  • Vidant Medical Center

    Greenville, North Carolina 27834
    United States

    Site Not Available

  • Comprehensive Cancer Center of Wake Forest University

    Winston-Salem, North Carolina 27157
    United States

    Active - Recruiting

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina 27157
    United States

    Site Not Available

  • East Carolina University

    Greenville 4469160, North Carolina 4482348 27834
    United States

    Site Not Available

  • Wake Forest Baptist Health

    Winston-Salem 4499612, North Carolina 4482348 27157
    United States

    Site Not Available

  • UC Health At Cincinnati Va Medical Center

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • Cleveland Clinic Foundation

    Cleveland, Ohio 44195
    United States

    Site Not Available

  • The Ohio State University

    Columbus, Ohio 43210
    United States

    Site Not Available

  • UC Health At Cincinnati Va Medical Center

    Cincinnati 4508722, Ohio 5165418 45229
    United States

    Site Not Available

  • Cleveland Clinic Foundation

    Cleveland 5150529, Ohio 5165418 44195
    United States

    Site Not Available

  • Spoknwrd Clinical Trials Inc.

    Easton, Pennsylvania 18045
    United States

    Site Not Available

  • University of Pennsylvania Hospital

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • University of Pittsburgh Medical Center Health System

    Pittsburgh, Pennsylvania 15232
    United States

    Site Not Available

  • University of Pennsylvania Hospital

    Philadelphia 4560349, Pennsylvania 6254927 19104
    United States

    Site Not Available

  • University of Pittsburgh Medical Center Health System

    Pittsburgh 5206379, Pennsylvania 6254927 15232
    United States

    Site Not Available

  • Rhode Island Hospital

    Providence, Rhode Island 02903
    United States

    Site Not Available

  • Tennessee Oncology

    Chattanooga, Tennessee 37403
    United States

    Site Not Available

  • Tennessee Oncology

    Nashville, Tennessee 37203
    United States

    Site Not Available

  • Tennessee Oncology

    Chattanooga 4612862, Tennessee 4662168 37403
    United States

    Site Not Available

  • Tennessee Oncology

    Nashville 4644585, Tennessee 4662168 37203
    United States

    Site Not Available

  • Baylor University Medical Center

    Dallas, Texas 75246
    United States

    Site Not Available

  • Jps Health Network

    Fort Worth, Texas 76104
    United States

    Site Not Available

  • Houston Methodist Hospital

    Houston, Texas 77030
    United States

    Site Not Available

  • Baylor University Medical Center

    Dallas 4684888, Texas 4736286 75246
    United States

    Site Not Available

  • Houston Methodist Hospital

    Houston 4699066, Texas 4736286 77030
    United States

    Site Not Available

  • Huntsman Cancer Institute

    Salt Lake City, Utah 84112
    United States

    Site Not Available

  • Huntsman Cancer Institute

    Salt Lake City 5780993, Utah 5549030 84112
    United States

    Site Not Available

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington 98109
    United States

    Site Not Available

  • Fred Hutchinson Cancer Research Center

    Seattle 5809844, Washington 5815135 98109
    United States

    Site Not Available

  • West Virginia University Cancer Institute

    Morgantown, West Virginia 26506
    United States

    Site Not Available

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