A Randomized Controlled Trial to Compare the Efficacy of Oral Mycophenolate Mofetil With Placebo in Patients With Systemic Sclerosis Related Early Interstitial Lung Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 41
- 试验地点
- 1
- 主要终点
- Change from baseline in Forced vital capacity (FVC) at 6 months, after treatment with oral mycophenolate mofetil or placebo
研究概览
简要总结
Systemic sclerosis is a multisystem disease and can involve the lungs in the form of ILD. Lung involvement is the most common cause of death in these patients. The present study is performed to study the efficacy of oral mycophenolate mofetil in treating early and mild ILD in patients of SSc. The efficacy and side effects of mycophenolate mofetil will be compared with that of oral placebo.
详细描述
Lung involvement is the leading cause of death among patients with systemic sclerosis (SSc). Treatment with immunosuppression drugs helps in retarding the progression of interstitial lung disease (ILD) and improves the morbidity and mortality among these patients. Presently, cyclophosphamide has been shown to be useful in stabilizing the lung functions among patients of systemic sclerosis with ILD. But use of cyclophosphamide is also associated with many adverse effects including infections, cytopenias, gonadal dysfunction and malignancies. Use of oral mycophenolate mofetil (MMF) in SSc-ILD in recent studies has been shown to be effective in retarding progression of ILD among these patients with a better side effect profile compared to cyclophosphamide. Contemporary expert opinion dictates that the treatment for SSc-ILD needs to be individualized. Generally, intense immunosuppression is required in patients with FVC <70% of the predicted. In patients with FVC >70% of the predicted, the need for high dose immunosuppression is not clear and varies from center-to-center. The present study is designed to determine the efficacy of oral MMF in patients with SSc related early ILD. The subjects in this study will be given either oral MMF or placebo and will be monitored for their response and adverse events. Informed consent will be taken from the subjects before including in the study.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of systemic sclerosis with presence of interstitial lung disease on High Resolution Computer Tomography (HRCT) chest
- •FVC ≥ 70% of predicted on pulmonary function tests
- •Age ≥18 years
- •Consenting for participating in study
排除标准
- •Received immunosuppression (except low dose steroids, prednisolone equivalent ≤10 mg/day) for ILD in the last 3 years
- •Persistent leucopenia or thrombocytopenia
- •Pregnant or breastfeeding females
- •Severe pulmonary arterial hypertension (mean pulmonary arterial pressure >55mmHg) requiring drug therapy
- •Uncontrolled congestive heart failure
- •Any other abnormalities noted on chest X-ray or HRCT other than ILD
- •Active infection
- •Inflammatory myositis
- •Overlap syndrome
- •Mixed connective tissue disease
- •Other serious co-morbidities which could compromise patient's ability to complete the study
研究组 & 干预措施
Placebo
Subjects in this arm will be given matching placebo, made of lactulose, starting at two tablets per day and increased by one tablet every 2 weeks to a target of 4 tablets per day.
干预措施: Placebo (Drug)
Mycophenolate mofetil
Subjects will be started on Mycophenolate Mofetil 500mg twice a day and increased by 500mg every 2 weeks, if tolerated, to a target dose of 2gram per day.
干预措施: Mycophenolate mofetil (Drug)
结局指标
主要结局
Change from baseline in Forced vital capacity (FVC) at 6 months, after treatment with oral mycophenolate mofetil or placebo
时间窗: 6 months
次要结局
- Number of participants with serious and non seroius adverse events with mycophenolate mofetil (MMF) and placebo(6 months)
- Change in Forced Vital Capacity (FVC) from baseline to 6 months according to antibody (anti-centromere and anti-topoisomerase1) profile(6 months)
- Change from baseline in Mahler Dyspnoea Index (MDI) at 6 months(6 months)
- Change from baseline in Quality of Life (QoL) score by Medical Outcome Short Form 36 (SF-36v2) at 6 months(6 months)
研究者
GSRSNK NAIDU
SENIOR RESIDENT
Post Graduate Institute of Medical Education and Research, Chandigarh