Evidence map›Paper›PMID 38813112›Full record

ArticleNeuro-oncology advances

TARGET: A phase I/II open-label multicenter study to assess safety and efficacy of fexagratinib in patients with relapsed/refractory FGFR fusion-positive glioma.

Alberto Picca, Anna Luisa Di Stefano, Julien Savatovsky, François Ducray, Olivier Chinot, Elisabeth Cohen-Jonathan Moyal, Paule Augereau, Emilie Le Rhun, Yohann Schmitt, Nabila Rousseaux and 11 more

Registry-linked trialAbstract read
In one paragraph

Article in Neuro-oncology advances. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02824133 (A Phase I/II, Open-Label, Multicentre Study to Assess The Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of AZD4547 in Patients With Relapsed/Refractory Glioma Positive for an FGFR Fusion), which is not on this map. Cited by 16 papers.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02824133 phase1 / phase2completednot on this map

A Phase I/II, Open-Label, Multicentre Study to Assess The Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of AZD4547 in Patients With Relapsed/Refractory Glioma Positive for an FGFR Fusion

TypeinterventionalSponsorAssistance Publique - Hôpitaux de ParisRan2015 to 2018Enrolled14ConditionsRecurrent IDHwt Gliomas With FGFR3-TACC3 Fusion, Recurrent IDHwt Gliomas With FGFR1-TACC1 FusionArmsAZD4547
3 · Its place in the literature

Who cites it

16 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
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  5. Recent Advances in Targeted Therapies for Adult Gliomas.Current neurology and neuroscience reports · 2026
    Review
  6. Review
  7. Article
  8. Review
  9. Article
  10. Review
  11. Diffuse glioma withRadiology case reports · 2025
    Article
  12. Review
  13. Review
  14. Review
  15. Review
  16. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

21 authors.

Alberto PiccaService de Neuro-Oncologie, Institut de Neurologie, DMU Neurosciences, AP-HP, Hôpital de la Pitié-Salpêtrière, Paris, France.ORCID https://orcid.org/0000-0001-7807-5393
Anna Luisa Di StefanoDepartment of Neurology, Foch Hospital, Suresnes, France.
Julien SavatovskyDepartment of Radiology, Hôpital Fondation A. de Rothschild, Paris, France.
François DucrayDepartment of Neuro-Oncology, East Group Hospital, Hospices Civils de Lyon, Lyon, France.
Olivier ChinotDepartment of Neuro-Oncology, AP-HM, University Hospital Timone, Marseille, France.
Elisabeth Cohen-Jonathan MoyalDepartment of Radiotherapy, Claudius Regaud Institute, Cancer University Institute of Toulouse, Oncopole 1, Paul Sabatier University, Toulouse III, Toulouse, France.
Paule AugereauDepartment of Medical Oncology, Institut de Cancérologie de L'ouest- Paul Papin, Angers, France.
Emilie Le RhunDepartment of Neurosurgery, Lille University Hospital, Lille, France.ORCID https://orcid.org/0000-0002-9408-3278
Yohann SchmittSorbonne Université, Inserm, CNRS, UMR S 1127, Paris Brain Institute (ICM), Paris, France.
Nabila RousseauxService de Neuro-Oncologie, Institut de Neurologie, DMU Neurosciences, AP-HP, Hôpital de la Pitié-Salpêtrière, Paris, France.
Ariane Murielle Mbekwe YepnangAP-HP, Hôpital Pitié Salpêtrière, Unité de Recherche Clinique PSL-CFX, Paris, France.
Candice EstellatSorbonne Université, INSERM, Institut Pierre Louis d'Epidémiologie et de Santé Publique-IPLESP, AP-HP, Hôpital Pitié Salpêtrière, Département de Santé Publique, Unité de Recherche Clinique PSL-CFX, Paris, France.
Frédérique CharbonneauDepartment of Radiology, Hôpital Fondation A. de Rothschild, Paris, France.
Quentin LetourneurSorbonne Université, INSERM, UMR S 938 and SIRIC CURAMUS, Centre de Recherche Saint-Antoine (CRSA), Paris, France.
Dominique Figarella BrangerDepartment of Pathology and Neuropathology, La Timone Hospital, Aix Marseille University, Marseille, France.
David MeyronetDepartment of Neuropathology, Hospices Civils de Lyon, Lyon, France.ORCID https://orcid.org/0000-0001-9943-4578
Christine FardeauDepartment of Ophthalmology, Hôpital de la Pitié-Salpêtrière, Paris, France.
Karima MokhtariSorbonne Université, Inserm, CNRS, UMR S 1127, Paris Brain Institute (ICM), Paris, France.
Franck BielleSorbonne Université, Inserm, CNRS, UMR S 1127, Paris Brain Institute (ICM), Paris, France.ORCID https://orcid.org/0000-0001-6564-6388
Antonio IavaroneInstitute for Cancer Genetics, Columbia University Medical Center, New York, New York, USA.ORCID https://orcid.org/0000-0002-0683-4634
Marc SansonService de Neuro-Oncologie, Institut de Neurologie, DMU Neurosciences, AP-HP, Hôpital de la Pitié-Salpêtrière, Paris, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Oncogenic FGFR-TACC fusions are present in 3-5% of high-grade gliomas (HGGs). Fexagratinib (AZD4547) is an oral FGFR1-3 inhibitor with preclinical activity in FGFR-TACC+ gliomas. We tested its safety and efficacy in patients with recurrent FGFR-TACC + HGGs. Patients and Methods: TARGET (NCT02824133) is a phase I/II open-label multicenter study that included adult patients with FGFR-TACC + HGGs relapsing after ≥1 line of standard chemoradiation. Patients received fexagratinib 80 mg bd on a continuous schedule until disease progression or unacceptable toxicity. The primary endpoint was the 6-month progression-free survival rate (PFS6). Results: Twelve patients with recurrent IDH wildtype FGFR-TACC + HGGs (all FGFR3-TACC3+) were included in the efficacy cohort (male/female ratio = 1.4, median age = 61.5 years). Most patients (67%) were included at the first relapse. The PFS6 was 25% (95% confidence interval 5-57%), with a median PFS of 1.4 months. All patients without progression at 6 months ( Conclusions: Fexagratinib exhibited acceptable toxicity but limited efficacy in recurrent FGFR3-TACC3 + HGGs. Patients treated at first recurrence appeared more likely to benefit, yet additional evidence is required.

Indexed as

clinical trialfexagratinibFGFR3–TACC3FGFR inhibitorglioblastomaglioma

Identifiers

PMID38813112
PMCPMC11135358

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.