A Multicenter, Randomized, Double-blind, Placebo-controlled Trial for the Safety and Efficacy of Pirfenidone in the Treatment of Idiopathic Pulmonary Fibrosis (IPF)
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 80
- 试验地点
- 1
- 主要终点
- Changes in forced vital capacity (FVC)
研究概览
简要总结
Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive form of lung disease characterized by fibrosis of the supporting framework (interstitium) of the lungs. By definition, the term is used only when the cause of the pulmonary fibrosis is unknown ("idiopathic"). Microscopically, lung tissue from patients shows a characteristic set of histologic/pathologic features known as usual interstitial pneumonia (UIP). UIP is therefore the pathologic counterpart of IPF.Idiopathic pulmonary fibrosis is characterized by radiographically evident interstitial infiltrates predominantly affecting the lung bases and by progressive dyspnea and worsening of pulmonary function. No therapy has been clearly shown to prolong survival. The current strict definition of idiopathic pulmonary fibrosis provides a new focus for basic and clinical research that will improve insight into the pathogenesis of this disorder and stimulate the development of novel therapies.
Pirfenidone has proven antifibrotic and anti-inflammatory properties in various in vitro systems and animal models of pulmonary fibrosis, although its precise mechanism of action remains unclear. It attenuates fibroblast proliferation, production of fibrosis-associated proteins and cytokines, and the increased biosynthesis and accumulation of extracellular matrix in response to cytokines such as transforming growth factor-β. It is also shown to slow tumor cell proliferation by inhibiting fibroblast growth factor, epidermal growth factor and platelet-derived growth factor.
Pirfenidone has not been widely approved for clinical use in China, in this study, safety and efficacy were evaluated to see if pirfenidone has a significant advantage over placebo in terms of improving lung function and life quality etc. (see primary and secondary criteria) or slows down the deterioration of lung function in Chinese subjects diagnosed with IPF.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent signed;
- •18-75 years of age;
- •Clinically or multidisciplinarily diagnosed idiopathic pulmonary fibrosis(see 2011 guidance );
- •Resting state PaO2≥50mg, FVC%≥45% normal predicted value and DLCO≥30% normal predicted value.
排除标准
- •Allergic to pirfenidone;
- •Dyspnea symptoms relieved in the past 6 months;
- •Patients in acute exacerbation phase;
- •Diabetic patients whose fasting venous glucose >11.1 mmol/L;
- •Patients with malignant tumor and hemorrhagic diseases;
- •Patients with serious underlying pulmonary disease;
- •Patients with serious heart disease(NYHA class Ⅲ-Ⅳ), liver disease(ALT or AST 2 times above the upper level of normal value range), kidney disease(Cr above the upper level of normal value range);
- •Patients who has taken Acetylcysteine in the past 3 months;
- •Patients who has taken Prednisone>15mg/day(or other equivalent amount of glucocorticoid) and/or Immunosuppresants in the past 3 months;
- •Patients who has taken interferon, penicillamine, colchine or other agents for the treatment of IPF;
- •Pregnant or lactating women;
- •Participated in other clinical trials in the past 1 month;
- •The investigator assessed as inappropriate to participate in this clinical trial.
研究组 & 干预措施
Pirfenidone(200mg)
Pirfenidone（200mg）tablets will be taken 3 times a day during the whole study process. For the first week, 1 tablet will be taken each time. For the second week, 2 tablets will be taken each time. From the third week to the 48th week, 3 tablets will be taken each time. Base drug Acetyl Cysteine Tablets（600mg）will be taken once a day, 1 tablet each time from the first to the 48th week.
干预措施: Pirfenidone (Drug)
Placebo (without active ingredient)
干预措施: Placebo (Drug)
结局指标
主要结局
Changes in forced vital capacity (FVC)
时间窗: 48 weeks
Changes in FVC from 48 weeks to baseline
次要结局
- Changes in lung function (including arterial blood gas analysis)(48 weeks)
- Acute Exacerbation during the whole treatment procedure（frequency and severity）(48 weeks)
- Progression-free time(48 weeks)
- 6 Minute Walk Test (6MWT )： Changes in 6 minute walk distance (6MWD) and SpO2 from 48 weeks to baseline(48 weeks)
- Borg RPE scale rating improvement rate during the whole study period(48 weeks)
- Lung interstitial change observed by HRCT(48 weeks)
- Life quality: assessed by St. George respiratory questionnaire (SGRQ).(48 weeks)
研究者
Zuojun Xu
Chief Physician
Beijing Kawin Technology Share-Holding Co., Ltd.