Department of Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Sidney Kimmel Cancer Center of Jefferson University Health, Philadelphia, PA 19107, USA.
Future Oncol. 2022 Sep;18(28):3143-3150. doi: 10.2217/fon-2022-0657. Epub 2022 Aug 15.
Selpercatinib is a first-in-class, highly selective and potent, central nervous system-active kinase inhibitor. In the phase I/II trial, selpercatinib demonstrated clinically meaningful antitumor activity with manageable toxicity in heavily pre-treated and treatment-naive patients with -mutant medullary thyroid cancer (MTC). LIBRETTO-531 (NCT04211337) is a multicenter, open-label, randomized, controlled, phase III trial comparing selpercatinib to cabozantinib or vandetanib in patients with advanced/metastatic -mutant MTC. The primary objective is to compare progression-free survival (per RECIST 1.1) by blinded independent central review of patients with progressive, advanced, multikinase inhibitor-naive, -mutant MTC treated with selpercatinib versus cabozantinib or vandetanib. Key secondary objectives are to compare other efficacy outcomes (per RECIST 1.1) and tolerability of selpercatinib versus cabozantinib or vandetanib.
塞尔帕替尼是一种首创的、高度选择性和有效的中枢神经系统活性激酶抑制剂。在 I/II 期试验中，塞尔帕替尼在先前大量治疗且未经治疗的 - 突变型甲状腺髓样癌 (MTC) 患者中显示出具有临床意义的抗肿瘤活性，且毒性可管理。LIBRETTO-531（NCT04211337）是一项多中心、开放标签、随机、对照、III 期试验，比较了在晚期/转移性 - 突变型 MTC 患者中，塞尔帕替尼与卡博替尼或凡德他尼的疗效。主要目的是通过对进展期、晚期、多激酶抑制剂初治、- 突变型 MTC 患者进行盲法独立中心评估，比较接受塞尔帕替尼与卡博替尼或凡德他尼治疗的患者的无进展生存期（根据 RECIST 1.1 评估）。次要关键目标是比较塞尔帕替尼与卡博替尼或凡德他尼的其他疗效结果（根据 RECIST 1.1 评估）和耐受性。
