Journal article
Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial
WD Tap, H Gelderblom, E Palmerini, J Desai, S Bauer, JY Blay, T Alcindor, K Ganjoo, J Martín-Broto, CW Ryan, DM Thomas, C Peterfy, JH Healey, M van de Sande, HL Gelhorn, DE Shuster, Q Wang, A Yver, HH Hsu, PS Lin Show all
Lancet | ELSEVIER SCIENCE INC | Published : 2019
Abstract
Background: Tenosynovial giant cell tumour (TGCT), a rare, locally aggressive neoplasm, overexpresses colony-stimulating factor 1 (CSF1). Surgery is standard with no approved systemic therapy. We aimed to evaluate pexidartinib, a CSF1 receptor inhibitor, in patients with TGCT to provide them with a viable systemic treatment option, especially in cases that are not amenable to surgical resection. Methods: This phase 3 randomised trial had two parts. Part one was a double-blind study in which patients with symptomatic, advanced TGCT for whom surgery was not recommended were randomly assigned via an integrated web response system (1:1) to the pexidartinib or placebo group. Individuals in the pe..
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Awarded by National Institutes of Health
Funding Acknowledgements
We thank the patients who volunteered to participate in this study; their family members and caregivers; the study centre staff members who cared for the patients; the sponsor staff involved in data collection and analyses; Stefano Ferrari (IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy) for his contributions to the study; and Heather Nyce (SciStrategy Communications) for medical writing assistance in the development of the manuscript. Research and manuscript support was provided by Daiichi Sankyo (Tokyo, Japan). All research at Memorial Sloan Kettering is supported in part by a grant from the National Institutes of Health National Cancer Institute (#P30 CA008748).