Vimseltinib for patients with tenosynovial giant cell tumor: A multicenter, open‐label, phase 2 trial
Year: 2026
Type: article
Study design: Clinical Trial
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.
Source: Cancer
DOI: 10.1002/cncr.70564
Authors: Silvia Stacchiotti, Jean‐Yves Blay, Piotr Rutkowski, Hans Gelderblom, Alejandro Falcón, Axel Le Cesne, Jayesh Desai, Emanuela Palmerini, César Serrano, Kristen N. Ganjoo, Gina Z. D’Amato, Christopher W. Ryan, Breelyn A. Wilky, Virginia Ferraresi, Ramy Saleh, Vinod Ravi, Albiruni Abdul Razak, Antonio Casado Herráez, Mahbubl Ahmed, Nicholas M. Bernthal, Michiel van de Sande, Mehdi Brahmi, Bartlomiej Szostakowski, Jasmine Sen, Nicholas A. Zeringo, Brooke Harrow, Supraja Narasimhan, Amanda Saunders, Maitreyi G. Sharma, Matthew L. Sherman, Andrew J. Wagner, William D. Tap
Institutions: Fondazione IRCCS Istituto Nazionale dei Tumori, Centre Léon Bérard, The Maria Sklodowska-Curie National Research Institute of Oncology, Leiden University Medical Center, Hospital Universitario Virgen del Rocío, Institut Gustave Roussy, Peter MacCallum Cancer Centre, University of Miami, Istituto Ortopedico Rizzoli, Vall d'Hebron Institute of Oncology, Stanford University, Sylvester Comprehensive Cancer Center, Oregon Health & Science University, OHSU Knight Cancer Institute, University of Colorado Cancer Center, Instituto Nazionale Tumori Regina Elena, McGill University Health Centre, Research Institute of the McGill University Health Centre, The University of Texas MD Anderson Cancer Center, Princess Margaret Cancer Centre, Hospital Clínico San Carlos, University College London Hospitals NHS Foundation Trust, University of California, Los Angeles, Deciphera Pharmaceuticals (United States), Dana-Farber Cancer Institute, Memorial Sloan Kettering Cancer Center
Language: English
Subfield: Rheumatology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,790
Funder: Deciphera Pharmaceuticals
OpenAlex ID: W7204257727