Niu Xiaohui, Ravi Vinod, Shan Boyao, Guo Qiuxiang, Shi Haosong, Zou Qingping, Gelderblom Hans
Department of Orthopaedic Oncology Surgery, Beijing Ji Shui Tan Hospital, Beijing, 100035, P.R.China.
The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Future Oncol. 2024 Sep 17:1-8. doi: 10.1080/14796694.2024.2396227.
Tenosynovial giant cell tumor (TGCT) is a rare, locally invasive soft tissue tumor arising from the synovium of joints, bursa and tendon sheaths and is associated with the overexpression of the (CSF-1) gene. Pimicotinib is an orally available, highly selective and potent small molecule CSF-1 receptor (CSF-1R) inhibitor with robust efficacy and safety profile in patients with TGCT and is under development in multiple diseases. In an open-label Phase I study in patients with TGCT not amenable to surgery, pimicotinib showed superior efficacy and safety. In this article, we elucidate the rationale and study design of the multi-region Phase III MANEUVER trial (NCT05804045), which is designed to assess the efficacy and safety of pimicotinib in patients with TGCT not amenable to surgical resection in Asia, North America and Europe.
腱鞘巨细胞瘤（TGCT）是一种罕见的、局部侵袭性软组织肿瘤，起源于关节、滑囊和腱鞘的滑膜，与集落刺激因子-1（CSF-1）基因的过表达有关。匹莫替尼是一种口服可用的、高度选择性且强效的小分子CSF-1受体（CSF-1R）抑制剂，在TGCT患者中具有强大的疗效和安全性，并且正在针对多种疾病进行研发。在一项针对无法进行手术的TGCT患者的开放标签I期研究中，匹莫替尼显示出卓越的疗效和安全性。在本文中，我们阐明了多区域III期MANEUVER试验（NCT05804045）的原理和研究设计，该试验旨在评估匹莫替尼在亚洲、北美和欧洲无法进行手术切除的TGCT患者中的疗效和安全性。
