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Targeted drug pemigatinib tested in brain tumors with specific genetic markers

NCT ID NCT05267106

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 2 trial tested the drug pemigatinib in people with recurrent glioblastoma or other brain tumors that have specific changes in FGFR1-3 genes. Participants took a daily tablet for two weeks followed by one week off. The study aimed to see if the drug could shrink tumors, but it was terminated early and results are limited.

What this could mean

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

Active substance
Pemigatinib (a targeted cancer drug taken as a tablet)
What this could lead to
If successful, pemigatinib could offer a new treatment option for people with certain brain tumors that have specific genetic changes (FGFR1-3 alterations).
What could go wrong
This was a small, early-phase (phase 2) study that was terminated early, so results are limited. The drug may not shrink tumors or improve survival, and side effects are possible.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

83 people

The number who actually took part.

Started

May 2022

Finished

Dec 2024

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 to 99 years

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: * Histological, cytological, or molecular confirmation of recurrent GBM or other glioma, circumscribed astrocytic glioma, or glioneuronalor neuronal tumors that has recurred. * 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: * Prior receipt of an FGFR inhibitor. * 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.

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Conditions

The condition(s) this trial relates to.

astrocytic tumor central nervous system cancer glioblastoma glioma mixed neuronal-glial tumor nervous system cancer oligodendroglioma pilocytic astrocytoma

As listed by the trial registrant

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

Adult-type diffuse gliomas Glioblastoma

Contacts and locations

Locations

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

    Ponticelli, 80147, Italy

  • Aalborg Universitets Hospital

    Aalborg, 09000, Denmark

  • Addenbrooke'S Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Azienda Ospedaliero Universitaria Citta Della Salute E Della Scienza

    Torino, 10124, Italy

  • Baptist Md Anderson Cancer Center

    Jacksonville, Florida, 32207, United States

  • Baylor University Medical Center

    Dallas, Texas, 75246, United States

  • Chru de Lille Hopital Claude Huriez

    Lille, 59037, France

  • Chu Hopital de La Timone

    Marseille, 13005, France

  • City of Hope National Medical Center

    Duarte, California, 91010, United States

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • Clinica Universidad de Navarra (Cun)

    Pamplona, 31008, Spain

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • East Carolina University

    Greenville, North Carolina, 27834, United States

  • Erasmus Mc Cancer Institute

    Rotterdam, 3015 GD, Netherlands

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98109, United States

  • Guys Hospital

    London, SE1 9RT, United Kingdom

  • H. Lee Moffitt Cancer Center and Research Institute Hospital

    Tampa, Florida, 33612, United States

  • Hospital Clinic Barcelona Main

    Barcelona, 08036, Spain

  • Hospital Del Mar

    Barcelona, 08003, 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 General de Catalunya

    Sant Cugat del Vallès, 08190, Spain

  • Hospital Universitaire Pitie-Salpetriere

    Paris, 75013, France

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Hospital Universitario Hm Sanchinarro

    Madrid, 28050, Spain

  • Hospital Universitario Ramon Y Cajal

    Madrid, 28034, Spain

  • Hospital Universitario Virgen Del Rocio

    Seville, 41013, Spain

  • Hospital de La Santa Creu I Sant Pau

    Barcelona, 08041, Spain

  • Houston Methodist Hospital

    Houston, Texas, 77030, United States

  • Huntsman Cancer Institute

    Salt Lake City, Utah, 84112, United States

  • Ico Girona Hospital Universitari de Girona Dr Josep Trueta

    Girona, 17007, Spain

  • Ico Institut Catala D Oncologia

    L'Hospitalet de Llobregat, 08908, Spain

  • Iov - Istituto Oncologico Veneto Irccs

    Padova, 35128, Italy

  • Irccs Istituto Clinico Humanitas

    Rozzano, 20089, Italy

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

    Milan, 20132, Italy

  • Klinikum Der Johann Wolfgang Goethe University

    Frankfurt, 60528, Germany

  • Kyoto University Hospital

    Kyoto, 606-8507, Japan

  • Kyushu University Hospital

    Fukuoka, 812-8582, Japan

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • Miami Cancer Institute

    Miami, Florida, 33176, United States

  • Nagoya University Hospital

    Nagoya, 466-8560, Japan

  • National Cancer Center Hospital

    Chūōku, 104-0045, Japan

  • Netherlands Cancer Institute Antoni Van Leeuwenhoek Ziekenhuis

    Amsterdam, 1066 CX, Netherlands

  • Northwell Health

    Lake Success, New York, 11042, United States

  • Odense University Hospital

    Odense C, 05000, Denmark

  • Orlando Health Cancer Institute Downtown Orlando

    Orlando, Florida, 32806, United States

  • Ospedale Bellaria

    Bologna, 40139, Italy

  • Providence Medical Foundation

    Fullerton, California, 92835, United States

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

    Santa Monica, California, 90404, United States

  • Rigshospitalet Uni of Hospital of Copenhagen

    Copenhagen, 02100, Denmark

  • Sharp Memorial Hospital

    San Diego, California, 92123, United States

  • St James'S University Hospital

    Leeds, LS9 7TF, United Kingdom

  • Stanford Neuroscience Health Center

    Sacramento, California, 94304, United States

  • Tennessee Oncology

    Chattanooga, Tennessee, 37403, United States

  • Tennessee Oncology

    Nashville, Tennessee, 37203, United States

  • The Christie Nhs Foundation Trust Uk

    Manchester, M20 4BX, United Kingdom

  • The Clatterbridge Cancer Centre

    Metropolitan Borough of Wirral, CH63 4JY, United Kingdom

  • The Royal Marsden Nhs Foundation Trust - Chelsea

    London, SW36JJ, United Kingdom

  • The Royal Marsden Nhs Foundation Trust - Sutton

    London, SW3 6JJ, United Kingdom

  • Tohoku University Hospital

    Sendai, 980-8574, Japan

  • UC Health At Cincinnati Va Medical Center

    Cincinnati, Ohio, 45229, United States

  • Universitaetsklinikum Heidelberg

    Heidelberg, 69120, Germany

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

    Toulouse, 31059, France

  • Universitatsklinikum Bonn Aoer

    Bonn, 53127, Germany

  • University Hospital Tuebingen

    Tübingen, 72076, Germany

  • University Medical Center New Orleans

    New Orleans, Louisiana, 70112, United States

  • University of California San Francisco

    San Francisco, California, 94143, United States

  • University of Illinois Hospital & Health Sciences System

    Chicago, Illinois, 60612, United States

  • University of Iowa Hospital and Clinics

    Iowa City, Iowa, 52242, United States

  • University of Minnesota Health Clinics and Surgery Center

    Minneapolis, Minnesota, 55455, United States

  • University of Pennsylvania Hospital

    Philadelphia, Pennsylvania, 19104, United States

  • University of Pittsburgh Medical Center Health System

    Pittsburgh, Pennsylvania, 15232, United States

  • University of Tokyo Hospital

    Bunkyō City, 113-8655, Japan

  • Usc Norris Comprehensive Cancer Center

    Los Angeles, California, 90033, United States

  • Valkyrie Clinical Trials

    Beverly Hills, California, 90211, United States

  • Velindre Cancer Centre

    Cardiff, CF14 2TL, United Kingdom

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina, 27157, United States

  • Yale Cancer Center

    New Haven, Connecticut, 06510-3220, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.