ArticleOncology and therapy2025
A Phase 3 Study of the Efficacy and Safety of Pexidartinib in East Asian Patients with Tenosynovial Giant Cell Tumor.
Article in Oncology and therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04488822 (Multicenter, Single Arm Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With Tenosynovial Giant Cell Tumor), which is not on this map. Cited by 2 papers.
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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.
Multicenter, Single Arm Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With Tenosynovial Giant Cell Tumor
Who cites it
2 citing papers in PubMed.
- Pexidartinib: Current advances in the symptomatic treatment of tenosynovial giant cell tumors.Cancer metastasis reviews · 2026Review
- Tumor-associated macrophages in cancer: from mechanisms to application.Molecular biomedicine · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionThe aim of this study was to assess the efficacy and safety of pexidartinib, an inhibitor of colony-stimulating factor 1 receptor, in East Asian patients with tenosynovial giant cell tumor (TGCT).
methodsThis multicenter, single-arm, phase 3 study (NCT04488822) enrolled patients with symptomatic TGCT to receive pexidartinib 400 mg twice daily. The primary efficacy endpoint was overall response rate (ORR) by Response Evaluation Criteria in Solid Tumors (RECIST v1.1) per central review at week 25. Secondary endpoints included ORR by tumor volume score (TVS), change in range of motion (ROM) of the affected joint, best overall response (BOR), and treatment-emergent adverse events (TEAEs).
resultsA total of 40 East Asian patients with TGCT received pexidartinib. RECIST v1.1 ORR was 22.5% (95% confidence interval [CI] 10.8-38.5), with no complete responses (CRs), 9 partial responses (PRs), 21 patients with stable disease (Sd), and 1 patient with progressive disease (PD). TVS ORR was 47.5% (95% CI 31.5-63.9), with no CRs, 19 PRs, 12 patients with Sd, and none with PD. At week 25, mean (standard error [SE]) change in ROM was 23.05 (3.207), with improvement noted at week 13 (15.64 [3.312]). RECIST v1.1 BOR was 30.0% (95% CI 16.6-46.5), and TVS BOR was 47.5% (95% CI 31.5-63.9). All patients had TEAEs, and grade ≥ 3 TEAEs were reported in 16 (40.0%) patients. The most common TEAEs were hair color changes (77.5%); increases in alanine aminotransferase (60.0%), aspartate aminotransferase (57.5%), and blood lactate dehydrogenase (47.5%); and pruritus (47.5%).
conclusionsIn East Asian patients with symptomatic TGCT, pexidartinib demonstrated clinical benefits, with a safety profile comparable to previous reports.
trial registrationClinicalTrials.gov identifier NCT04488822.
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