Randomized Controlled Trial of Pirfenidone in Patients With Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis
试验速览
- 阶段
- 4 期
- 入组人数
- 57
- 试验地点
- 1
- 主要终点
- changes of 12-month survival from the onset of ILD
研究概览
简要总结
Interstitial lung disease (ILD) is a common complication of dermatomyositis (DM) with prevalence up to 65%, and is considered to be one of the determining factors of prognosis. Clinical amyopathic dermatomyositis (CADM), which is a special phenotype of DM, with characteristic cutaneous manifestations but no or only subclinical myopathy. Many studies, mainly from Asia, including ours, have demonstrated that these patients with CADM tend to develop a rapidly progressive ILD (RPILD) and have a poor response to conventional therapy, such as high-dose corticosteroids and immunosuppressants, leading to lethal outcome with a 6-month survival rate of less than 50%.
Pirfenidone, a new oral antifibrotic agent, has been approved for the treatment of idiopathic pulmonary fibrosis (IPF). Randomized controlled trials of pirfenidone in patients with IPF suggested that it could ameliorate pulmonary function decline and improve the progression-free survival. Its utility in connective tissue disease (CTD) related ILD has been implicated, but no evidence has yet demonstrated its efficacy. Therefore, the investigators conduct this study to evaluate the possible therapeutic effects of pirfenidone on RPILD associated with CADM.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willingness of the subject to participate in the study, proven by signing the informed consent;
- •All participants fulfilled the provisional diagnosis of CADM according to the modified Sontheimer's criteria.
- •The course of ILD is longer than 3 months, but shorter than 6 months, presenting as increase in level of dyspnea, and worsening of fibrosis on pulmonary HRCT with >10% increase of HRCT score, and/or decrease in %FVC by >10% absolute value.
排除标准
- •Participants who are unwilling to sign the inform consent;
- •The course of participants ever treated with biologics including basiliximab, or malignancy-associated CADM or overlapped with other CTD, or with alanine transaminase more than 2 times the upper normal limits;
- •Pregnancy or lactation.
研究组 & 干预措施
pirfenidone
Eligible participants for clinical trial were randomized in a 2:1 ratio to pirfenidone/blank add-on. Pirfenidone was administered in three divided doses (200mg tid), and increased to the manufacturer's instructed target dose (600mg tid) over a 2-week period. Investigators were allowed to adjust the dose according to the participants' tolerance.
干预措施: Pirfenidone (Drug)
结局指标
主要结局
changes of 12-month survival from the onset of ILD
时间窗: 12 months
the effect of pirfenidone on improving the survival rate
次要结局
- changes of high-resolution computed tomography (HRCT) scores from baseline at each visit(one year)
- changes of pulmonary function test from baseline at each visit(one year)
研究者
Shuang Ye
Executive Director, Dept. Rheumatology, Renji Hospital South Campus
RenJi Hospital