西那卡塞治疗中国维持性血液透析继发性甲状旁腺功能亢进的多中心临床研究(ACTIVE)方案

Rationale and design of cinacalcet in Chinese maintenance hemodialysis patients with secondary hyperparathyroidism(ACTIVE)study:A phase Ⅳ,multicenter,open-label study

继发性甲状旁腺功能亢进(SHPT)可增加慢性肾脏病(CKD)患者的心血管事件风险和心血管死亡率。西那卡塞可安全有效地降低伴SHPT的CKD患者的甲状旁腺激素(PTH)水平,但目前尚缺乏该药在中国终末期肾病患者有效性和安全性数据。本研究拟通过Ⅳ期多中心、开放标签研究(ACTIVE)探究西那卡塞在中国维持性血液透析患者的有效性和安全性。研究分两个阶段进行:第一阶段是为期32周的药物干预队列研究,旨在观察西那卡塞治疗的有效性和安全性;第二阶段是为期20周的真实世界观察,完成药物干预队列的患者自行选择是否继续服用西那卡塞治疗。患者在入组前基线全段甲状旁腺激素(iPTH)≥300 pg/ml,且至少已接受了12周的维持性血液透析治疗。主要疗效指标是西那卡塞治疗20周和32周患者的iPTH达标率(150 pg/ml≤iPTH<300 pg/ml)。

Patients with chronic kidney disease(CKD)and uncontrolled secondary hyperparathyroidism(SHPT)are at high risk of cardiovascular events and associated mortality.Cinacalcet,a calcimimetic agent,was reported to effectively and safely reduce parathyroid hormone(PTH)levels in CKD patients with SHPT.However,data on efficacy and safety of cinacalcet in Chinese end stage renal disease patients are lacking.A phase IV,multicenter,open-label study(ACTIVE)had been conducted to examine the effectiveness and safety of cinacalcet in Chinese maintenance hemodialysis(MHD)patients.This study had been performed in two phases.In phase 1,a cohort study with 32 weeks'follow-up was designed to explore the efficacy and safety of cinacalcetIn phase 2,a real-world study of 20 additional weeks,patients who completed the cohort study decided independently whether to continue taking cinacalcet at their own costMHD patients with a baseline intact PTH(iPTH)≥300 pg/ml were enrolled.The primary efficacy outcome is the proportion of patients achieving iPTH targets(150 pg/ml≤iPTH<300 pg/ml)after 20 weeks and 32 weeks of cinacalcet treatment.