Vimseltinib for patients with tenosynovial giant cell tumor: A multicenter, open‐label, phase 2 trial

BACKGROUND: Tenosynovial giant cell tumor (TGCT) is a locally aggressive neoplasm caused by dysregulation of the colony-stimulating factor 1 (CSF1) gene. Patients report substantial pain, stiffness, and declining physical function; those whose disease is not amenable to surgery require systemic therapy. Vimseltinib is an oral, switch-control kinase inhibitor of the CSF1 receptor (CSF1R). Here, the authors report safety and efficacy of vimseltinib in patients with TGCT based on prior treatment. Cohort A included patients who did not receive prior specific anti-CSF1/CSF1R agents (n = 46; prior imatinib/nilotinib allowed), and cohort B included patients who received prior specific agents (n = 20). METHODS: The phase 2 (expansion) portion of this ongoing, multicenter, open-label, phase 1/2 study (NCT03069469) enrolled adults (≥18 years) with histologically-confirmed TGCT not amenable to surgery. Patients received vimseltinib 30 mg twice weekly (recommended phase 2 dose). The primary objectives were to assess safety and antitumor activity; secondary objectives included assessment of active range of motion (ROM) and patient-reported outcomes. RESULTS: Most treatment-emergent adverse events were grade 1/2, and there was no evidence of cholestatic hepatotoxicity or drug-induced liver injury. Best overall response rates were 64% (29 of 45) and 37% (7 of 19) for cohorts A and B after mean follow-up of 23 and 19 months, respectively. Most patients experienced meaningful improvements in active ROM and patient-reported physical function, stiffness, health status, and pain. CONCLUSIONS: Vimseltinib had a manageable safety profile, demonstrated durable antitumor activity, and provided functional and symptomatic improvements in patients with TGCT, offering an effective treatment option regardless of previous treatment with anti-CSF1/CSF1R agents.

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Journal
Cancer
Published
2026-08-25
DOI
https://doi.org/10.1002/cncr.70564
Primary Topic
Musculoskeletal synovial abnormalities and treatments
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article
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article

Vimseltinib for patients with tenosynovial giant cell tumor: A multicenter, open‐label, phase 2 trial

Andrew J. Wagner, Bartłomiej Szostakowski, Alejandro Falcón, Kristen N. Ganjoo et al.
Cancer
Musculoskeletal synovial abnormalities and treatments
article

Vimseltinib for patients with tenosynovial giant cell tumor: A multicenter, open‐label, phase 2 trial

Andrew J. Wagner, Bartłomiej Szostakowski, Alejandro Falcón, Kristen N. Ganjoo, Hans A. J. Gelderblom, Michiel A. J. van de Sande, Virginia Ferraresi, Mahbubl Ahmed, Gina Z. D’Amato, Jean‐Yves Blay, Christopher W. Ryan, César Serrano, Vinod Ravi, Silvia Stacchiotti, Maitreyi G. Sharma, Jayesh Desai, Emanuela Palmerini, Antonio Casado Herráez, Supraja Narasimhan, Brooke Harrow, Amanda Saunders, Nicholas M. Bernthal, Ramy Saleh, Breelyn A. Wilky, Matthew L. Sherman, William D. Tap, Jasmine Sen, Mehdi Brahmi, Piotr Rutkowski, Nicholas A. Zeringo, Albiruni Abdul Razak, Axel Le Cesne
article en

Abstract

BACKGROUND: Tenosynovial giant cell tumor (TGCT) is a locally aggressive neoplasm caused by dysregulation of the colony-stimulating factor 1 (CSF1) gene. Patients report substantial pain, stiffness, and declining physical function; those whose disease is not amenable to surgery require systemic therapy. Vimseltinib is an oral, switch-control kinase inhibitor of the CSF1 receptor (CSF1R). Here, the authors report safety and efficacy of vimseltinib in patients with TGCT based on prior treatment. Cohort A included patients who did not receive prior specific anti-CSF1/CSF1R agents (n = 46; prior imatinib/nilotinib allowed), and cohort B included patients who received prior specific agents (n = 20). METHODS: The phase 2 (expansion) portion of this ongoing, multicenter, open-label, phase 1/2 study (NCT03069469) enrolled adults (≥18 years) with histologically-confirmed TGCT not amenable to surgery. Patients received vimseltinib 30 mg twice weekly (recommended phase 2 dose). The primary objectives were to assess safety and antitumor activity; secondary objectives included assessment of active range of motion (ROM) and patient-reported outcomes. RESULTS: Most treatment-emergent adverse events were grade 1/2, and there was no evidence of cholestatic hepatotoxicity or drug-induced liver injury. Best overall response rates were 64% (29 of 45) and 37% (7 of 19) for cohorts A and B after mean follow-up of 23 and 19 months, respectively. Most patients experienced meaningful improvements in active ROM and patient-reported physical function, stiffness, health status, and pain. CONCLUSIONS: Vimseltinib had a manageable safety profile, demonstrated durable antitumor activity, and provided functional and symptomatic improvements in patients with TGCT, offering an effective treatment option regardless of previous treatment with anti-CSF1/CSF1R agents.

CancerVol. 132(17)
Memorial Sloan Kettering Cancer Center (US), University College London Hospitals NHS Foundation Trust (GB), The University of Texas MD Anderson Cancer Center (US), University of Miami (US), University of California, Los Angeles (US), Oregon Health & Science University (US), Leiden University Medical Center (NL), Peter MacCallum Cancer Centre (AU), McGill University Health Centre (CA), Institut Gustave Roussy (FR), Princess Margaret Cancer Centre (CA), Hospital Clínico San Carlos (ES), Stanford Health Care (US), Centre Léon Bérard (FR), Dana-Farber Cancer Institute (US), National Institute of Oncology (HU), Istituto Ortopedico Rizzoli (IT), Deciphera Pharmaceuticals (United States) (US), University of Colorado Cancer Center (US), Stanford Medicine (US), Tumori Foundation (US), The Maria Sklodowska-Curie National Research Institute of Oncology (PL), Hospital Universitario Virgen del Rocío (ES), Fondazione IRCCS Istituto Nazionale dei Tumori (IT), Sylvester Comprehensive Cancer Center (US), Vall d'Hebron Institute of Oncology (ES), University College London (GB), University of Colorado Denver (US)
Openalex Percentile: Top 9%
Musculoskeletal synovial abnormalities and treatments
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