The LOTUSS Trial: An Open-Label, Randomized, Phase 2 Study of the Safety and Tolerability of Pirfenidone When Administered to Patients With Systemic Sclerosis-Related Interstitial Lung Disease (SSc-ILD) (LOTUSS)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 63
- 试验地点
- 22
- 主要终点
- Percentage of Participants With Treatment-Emergent Serious Adverse Events (SAEs)
研究概览
简要总结
PSSc-001 (LOTUSS)
This study is a Phase 2, multinational, open-label, randomized, parallel-group, safety and tolerability study of pirfenidone in participants with systemic sclerosis-related interstitial lung disease (SSc-ILD).
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of systemic sclerosis-related (SSc) confirmed by the American College of Rheumatology classification criteria of systemic sclerosis (Masi 1980); duration of diagnosis less than (<) 7 years
- •Diagnosis of SSc-ILD based on an high-resolution computed tomography (HRCT) scan
- •Screening forced vital capacity (FVC) greater than equal to (>=) 50 percent (%) of the predicted value, and screening carbon monoxide diffusing capacity (DLCO) >=40% of the predicted value
- •At study entry, the participant either was not taking SSc-ILD medication or was taking cyclophosphamide or mycophenolate
排除标准
- •Clinically significant pulmonary hypertension
- •Known underlying liver disease
- •Clinical evidence of significant aspiration or uncontrolled gastroesophageal reflux
- •History of clinically significant asthma or chronic obstructive pulmonary disease
- •Active infection
- •Diagnosis of another connective tissue disorder
- •Evidence of a malignancy that is likely to result in significant disability or require significant medical or surgical intervention
- •History of unstable or deteriorating cardiac or pulmonary disease (other than SSc-ILD)
- •Pregnancy or lactation
- •Creatinine clearance <40 milliliters per minute (mL/min)
- •Prior use of pirfenidone
- •Unsuitable for enrollment or unlikely to comply with study requirements
研究组 & 干预措施
Pirfenidone: 4-Week Titration Group
Participants will receive one 267 milligrams (mg) oral pirfenidone capsule three times daily (TID) (801 mg per day [mg/day]) for 2 weeks followed by two 267 mg oral pirfenidone capsules TID (1602 mg/day) for 2 weeks (titration period) and then three 267 mg oral pirfenidone capsules TID (2403 mg/day) for 12 weeks maintenance period).
干预措施: Pirfenidone (Drug)
Pirfenidone: 2-Week Titration Group
Participants will receive one 267 mg oral pirfenidone capsule TID (801 mg/day) for 1 week followed by two 267 mg oral pirfenidone capsules TID (1602 mg/day) for 1 week (titration period) and then three 267 mg oral pirfenidone capsules TID (2403 mg/day) for 14 weeks (maintenance period).
干预措施: Pirfenidone (Drug)
结局指标
主要结局
Percentage of Participants With Treatment-Emergent Serious Adverse Events (SAEs)
时间窗: From baseline up to 28 days after the last dose of study drug (last dose = Week 16)
An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pretreatment state.
Percentage of Participants With Treatment-Emergent Adverse Events (AEs)
时间窗: From baseline up to 28 days after the last dose of study drug (last dose = Week 16)
Percentage of participants who had treatment-emergent AEs, defined as newly occurring or worsening after first dose. Relatedness to (study drug) was assessed by the investigator (Yes/No). Participants with multiple occurrences of an AE within a category were counted once within the category.
次要结局
- University of California at Los Angeles (UCLA) Scleroderma Clinical Trial Consortium (SCTC) Gastrointestinal Trial (GIT) Questionnaire Scale Scores(Baseline, Weeks 4, 8, 12, and 16)