来氟米特与安慰剂联合基础泼尼松治疗活动期大动脉炎患者的疗效和安全性比较：一项随机、双盲对照试验的研究方案（中国大动脉炎临床试验：TACTIC）
Comparison of the efficacy and safety of leflunomide versus placebo combined with basic prednisone therapy in patients with active disease phase of Takayasu arteritis: study protocol for a randomized, double-blinded controlled trial (Takayasu arteritis clinical trial in China: TACTIC).
作者信息
Sun Ying, Wu Bingjie, Zhang Wei, Ma Lili, Kong Xiufang, Chen Huiyong, Jiang Lindi
机构信息
Department of Rheumatology, Zhongshan Hospital, Fudan University, Shanghai, P.R. China.
Biomedical Information & Statistics Center, School of Public Health, Fudan University, Shanghai, P.R. China.
出版信息
Ther Adv Chronic Dis. 2023 Mar 4;14:20406223231158567. doi: 10.1177/20406223231158567. eCollection 2023.
BACKGROUND
Takayasu arteritis (TAK) is an immune-induced granulomatous vasculitis that occurs primarily in young Asian women. Our previous cohort studies have indicated that leflunomide (LEF), which can lead to rapid induction and might be a promising alternative treatment for TAK.
OBJECTIVES
To compare the efficacy and safety of LEF placebo combined with prednisone for active TAK in a Chinese population.
DESIGN
This will be a multicenter, randomized, double-blinded controlled trial aiming to recruit 116 TAK patients with active disease. This study will last 52 weeks.
METHODS AND ANALYSIS
Participants will be assigned randomly to the LEF intervention arm or placebo control arm at a 1:1 ratio. Initially, LEF combined with prednisone will be given to the intervention arm and a placebo tablet combined with prednisone will be given to the placebo arm. At the end of week 24, subjects who achieved clinical remission or partial clinical remission will proceed to maintenance therapy with LEF to the end of week 52; those who did not achieve clinical remission or partial clinical remission in the LEF intervention arm will drop out from the study, and those in the placebo control arm will switch to LEF treatment to week 52. The primary endpoint will be the clinical remission rate of LEF placebo at the end of week 24. The secondary endpoints will be the time to clinical remission, mean dose of prednisone, disease recurrence, time to recurrence, adverse events, as well as clinical remission in subjects who switched from the placebo control arm to LEF therapy after week 24. Intention to treat will be the primary analysis.
DISCUSSION
This is the first randomized double-blinded placebo-controlled trial to clarify the efficacy and safety of LEF in treating active TAK. The results will provide more evidence for TAK management.
REGISTRATION
ClinicalTrials.gov identifier: NCT02981979.
背景
大动脉炎（TAK）是一种免疫介导的肉芽肿性血管炎，主要发生在年轻亚洲女性中。我们之前的队列研究表明，来氟米特（LEF）可迅速诱导病情缓解，可能是TAK一种有前景的替代治疗方法。
目的
比较来氟米特与安慰剂联合泼尼松治疗中国人群活动性TAK的疗效和安全性。
设计
这将是一项多中心、随机、双盲对照试验，旨在招募116例活动性TAK患者。本研究将持续52周。
方法与分析
参与者将按1:1的比例随机分配到来氟米特干预组或安慰剂对照组。最初，干预组给予来氟米特联合泼尼松，安慰剂组给予安慰剂片联合泼尼松。在第24周结束时，达到临床缓解或部分临床缓解的受试者将继续接受来氟米特维持治疗至第52周结束；来氟米特干预组中未达到临床缓解或部分临床缓解的受试者将退出研究，安慰剂对照组的受试者将改用LEF治疗至第52周。主要终点将是第24周结束时来氟米特与安慰剂的临床缓解率。次要终点将是临床缓解时间、泼尼松平均剂量、疾病复发、复发时间、不良事件，以及第24周后从安慰剂对照组改用LEF治疗的受试者的临床缓解情况。意向性分析将作为主要分析方法。
讨论
这是第一项阐明来氟米特治疗活动性TAK疗效和安全性的随机双盲安慰剂对照试验。研究结果将为TAK的治疗提供更多证据。
注册信息
ClinicalTrials.gov标识符：NCT02981979。
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