A Randomized, Double-blinded, Placebo Controlled Study to Evaluate Clinical Efficacy and Safety of Pirfenidone for Skin Fibrosis in Systemic Sclerosis
试验速览
- 阶段
- 2 期
- 入组人数
- 72
- 试验地点
- 1
- 主要终点
- Modified Rodnan Skin Score (mRSS）
研究概览
简要总结
This study is to evaluate the efficacy and safety of an antifibrotic agent, pirfenidone as treatment of systemic sclerosis. The primary outcome of this study is improvement of skin fibrosis.
详细描述
All patients enrolled should suffer from moderate to severe skin fibrosis at the screening stage.
The study period is 52 week. The study contains two stages, a 24-week blind stage following with open stage for another 24-week period of time. Subjects will receive pirfenidone or placebo randomized in the first 24-week and will all receive pirfenidone treatment in the second 24-week, with of combination of low dose steroids.
The secondary outcomes of this study include but not limit to safety, improvement of lung fibrosis and digital ulcer burden.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Fulfill EULAR/ACR classification criteria (2013) for systemic sclerosis (SSc).
- •Subjects must have mRSS≥10 at screening stage
- •Subjects must have signs of fibrosis in their chest CT at screening stage
- •If a subject has received treatments before, it should be stable at least 3 months until screening and with no significant efficacy (ΔmRSS≤-2)
排除标准
- •Subjects with other connective tissue diseases overlapping with SSc
- •Subjects with skin atrophy as the main cutaneous manifestation
- •Subjects with active digital ulcers or gangrene
- •Active severe SSc-driven renal disease or heart dysfunction at screening
- •Subjects with significant hematologic abnormalities
- •Abnormal liver function test at screening (ALT, AST or total bilirubin over 2 fold of upper normal level
- •Clinically significant active infection including ongoing and chronic infections
- •History of human immunodeficiency virus (HIV)
- •Confirmed Positive tests for hepatitis B or positive test for hepatitis C
- •Active tuberculosis
- •Live or attenuated vaccine within 4 weeks prior to screening
- •Subjects with significant hematologic abnormalities
研究组 & 干预措施
Pirfenidone group
The subjects will receive pirfenidone from 200mg three times a day, oral administrated, with low dose steroids.
干预措施: Pirfenidone (Drug)
Pirfenidone group
The subjects will receive pirfenidone from 200mg three times a day, oral administrated, with low dose steroids.
干预措施: Steroids (Drug)
Control group
The subjects will receive placebo within first 24-week, and pirfenidone 200mg three times a day for the second 24-week, with low dose steroids.
干预措施: Pirfenidone (Drug)
Control group
The subjects will receive placebo within first 24-week, and pirfenidone 200mg three times a day for the second 24-week, with low dose steroids.
干预措施: Placebo oral capsule (Drug)
Control group
The subjects will receive placebo within first 24-week, and pirfenidone 200mg three times a day for the second 24-week, with low dose steroids.
干预措施: Steroids (Drug)
结局指标
主要结局
Modified Rodnan Skin Score (mRSS）
时间窗: Week 52
A semi-quantitative score for skin fibrosis of every subjects
次要结局
- Assessment of chest CT(Week 24)
- Hand function assessment(Week 24)
- Proportion of subjects with increased mRSS(Week 24)
- Modified Rodnan Skin Score(Week 24)
- Forced vital capacity(Week 24)
- Diffusing capacity(Week 24)
- 6 minute walk test(Week 24)
- Quality of life(Week 24)
- Safety and Tolerability(Week 24)