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
NCT ID: NCT05267106
Last Updated: 2025-12-30
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2
83 participants
INTERVENTIONAL
2022-05-20
2024-12-17
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort A: IDH-wild-type GBM
Participants with histopathologically proven, WHO Grade 4, IDH-wild-type GBM OR molecular diagnosis of IDH-wild-type, diffuse astrocytic glioma with molecular features of Grade 4 GBM that are recurrent, harboring FGFR1-3 fusions/or other rearrangements, or with a defined FGFR1-3 mutation or in-frame deletion.
Pemigatinib
13.5mg tablet taken every morning (unless otherwise directed) for 2 weeks and then 1 week off.
Cohort B: Other gliomas other than GBM
Participants with other histopathologically proven gliomas other than GBM, circumscribed astrocytic gliomas, and glioneuronal and neuronal tumors that are recurrent, harboring FGFR1-3 fusions/or other rearrangements or with a defined FGFR1-3 activating mutation or in-frame deletion
Pemigatinib
13.5mg tablet taken every morning (unless otherwise directed) for 2 weeks and then 1 week off.
Interventions
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Pemigatinib
13.5mg tablet taken every morning (unless otherwise directed) for 2 weeks and then 1 week off.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Radiographically measurable disease.
. -Karnofsky performance status ≥ 60.
* Life expectancy ≥ 12 weeks.
* Documentation of an actionable FGFR1-3 gene mutation or fusion/rearrangement from tissue : FGFR1-3 fusions or other rearrangements (FGFR1-3 in-frame fusions, any FGFR2 rearrangement, or FGFR1/3 rearrangement with known partner) or a defined FGFR1-3 activating mutation or in-frame deletion. Only participants with FGFR fusions or rearrangements with an intact kinase domain are eligible.
* MRI-documented objective progression after prior therapy and must have no therapy available that is likely to provide clinical benefit.
* Most recent archival tumor specimen must be a tumor block or a minimum of 15 unstained slides from biopsy or resection of primary tumor or metastasis.
* Willingness to avoid pregnancy or fathering children.
Exclusion Criteria
* Receipt of anticancer medications or investigational drugs for any indication or reason within 28 days before first dose of study drug.
* Participants may have had treatment for an unlimited number of prior relapses but must not have had prior bevacizumab or other VEGF/VEGFR inhibitors (exception: prior bevacizumab is allowed if it was administered for the treatment of radiation necrosis rather than progressive tumor and was stopped at least 12 weeks prior to MRI 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 confirmed by ophthalmologic examination.
* Diffuse leptomeningeal disease.
* Radiation therapy administered within 12 weeks before enrollment/first dose of study drug.
* Known additional malignancy that is progressing or requires active systemic treatment.
18 Years
99 Years
ALL
No
Sponsors
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Incyte Corporation
INDUSTRY
Responsible Party
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Principal Investigators
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Victoria Ebiana, MD
Role: STUDY_DIRECTOR
Incyte Corporation
Locations
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Valkyrie Clinical Trials
Beverly Hills, California, United States
City of Hope National Medical Center
Duarte, California, United States
Providence Medical Foundation
Fullerton, California, United States
Usc Norris Comprehensive Cancer Center
Los Angeles, California, United States
Stanford Neuroscience Health Center
Sacramento, California, United States
Sharp Memorial Hospital
San Diego, California, United States
University of California San Francisco
San Francisco, California, United States
Providence St Joseph Hospital Orange Center For Cancer Prevention and Treatment
Santa Monica, California, United States
Yale Cancer Center
New Haven, Connecticut, United States
Baptist Md Anderson Cancer Center
Jacksonville, Florida, United States
Miami Cancer Institute
Miami, Florida, United States
Orlando Health Cancer Institute Downtown Orlando
Orlando, Florida, United States
H. Lee Moffitt Cancer Center and Research Institute Hospital
Tampa, Florida, United States
University of Illinois Hospital & Health Sciences System
Chicago, Illinois, United States
University of Iowa Hospital and Clinics
Iowa City, Iowa, United States
University Medical Center New Orleans
New Orleans, Louisiana, United States
University of Minnesota Health Clinics and Surgery Center
Minneapolis, Minnesota, United States
Northwell Health
Lake Success, New York, United States
Columbia University Irving Medical Center
New York, New York, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
East Carolina University
Greenville, North Carolina, United States
Wake Forest Baptist Health
Winston-Salem, North Carolina, United States
UC Health At Cincinnati Va Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
University of Pennsylvania Hospital
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center Health System
Pittsburgh, Pennsylvania, United States
Tennessee Oncology
Chattanooga, Tennessee, United States
Tennessee Oncology
Nashville, Tennessee, United States
Baylor University Medical Center
Dallas, Texas, United States
Houston Methodist Hospital
Houston, Texas, United States
Huntsman Cancer Institute
Salt Lake City, Utah, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States
Aalborg Universitets Hospital
Aalborg, , Denmark
Rigshospitalet Uni of Hospital of Copenhagen
Copenhagen, , Denmark
Odense University Hospital
Odense C, , Denmark
Chru de Lille Hopital Claude Huriez
Lille, , France
Chu Hopital de La Timone
Marseille, , France
Hospital Universitaire Pitie-Salpetriere
Paris, , France
Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole
Toulouse, , France
Universitatsklinikum Bonn Aoer
Bonn, , Germany
Klinikum Der Johann Wolfgang Goethe University
Frankfurt, , Germany
Universitaetsklinikum Heidelberg
Heidelberg, , Germany
University Hospital Tuebingen
Tübingen, , Germany
Ospedale Bellaria
Bologna, , Italy
Istituto Di Ricovero E Cura A Carattere Scientifico (Irccs) Ospedale San Raffaele
Milan, , Italy
Iov - Istituto Oncologico Veneto Irccs
Padua, , Italy
A.S.L. Napoli 1 Centro Ospedale Del Mare
Ponticelli, , Italy
Irccs Istituto Clinico Humanitas
Rozzano, , Italy
Azienda Ospedaliero Universitaria Citta Della Salute E Della Scienza
Torino, , Italy
University of Tokyo Hospital
Bunkyō City, , Japan
National Cancer Center Hospital
Chūōku, , Japan
Kyushu University Hospital
Fukuoka, , Japan
Kyoto University Hospital
Kyoto, , Japan
Nagoya University Hospital
Nagoya, , Japan
Tohoku University Hospital
Sendai, , Japan
Netherlands Cancer Institute Antoni Van Leeuwenhoek Ziekenhuis
Amsterdam, , Netherlands
Erasmus Mc Cancer Institute
Rotterdam, , Netherlands
Hospital Del Mar
Barcelona, , Spain
Hospital General Universitario Vall D Hebron
Barcelona, , Spain
Hospital Clinic Barcelona Main
Barcelona, , Spain
Hospital de La Santa Creu I Sant Pau
Barcelona, , Spain
Ico Girona Hospital Universitari de Girona Dr Josep Trueta
Girona, , Spain
Ico Institut Catala D Oncologia
L'Hospitalet de Llobregat, , Spain
Hospital General Universitario Gregorio Maranon
Madrid, , Spain
Hospital Universitario Ramon Y Cajal
Madrid, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Hospital Universitario Hm Sanchinarro
Madrid, , Spain
Clinica Universidad de Navarra (Cun)
Pamplona, , Spain
Hospital General de Catalunya
Sant Cugat del Vallès, , Spain
Hospital Universitario Virgen Del Rocio
Seville, , Spain
Hospital General Universitario de Valencia
Valencia, , Spain
Addenbrooke'S Hospital
Cambridge, , United Kingdom
Velindre Cancer Centre
Cardiff, , United Kingdom
St James'S University Hospital
Leeds, , United Kingdom
Guys Hospital
London, , United Kingdom
The Royal Marsden Nhs Foundation Trust - Sutton
London, , United Kingdom
The Royal Marsden Nhs Foundation Trust - Chelsea
London, , United Kingdom
The Christie Nhs Foundation Trust Uk
Manchester, , United Kingdom
The Clatterbridge Cancer Centre
Metropolitan Borough of Wirral, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2021-004740-24
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
INCB 54828-209
Identifier Type: -
Identifier Source: org_study_id