Phase 2 Study of Safety, Tolerability and Efficacy of Pirfenidone in Patients With Rheumatoid Arthritis Interstitial Lung Disease (TRAIL1)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 123
- 试验地点
- 33
- 主要终点
- Number of Participants Who Developed Any Element of the Composite Endpoint
研究概览
简要总结
The purpose of this study is to to assess the safety and tolerability of pirfenidone 2403 mg/day for the treatment of RA-associated interstitial lung disease.
详细描述
This is a phase 2, randomized, double blind, placebo controlled trial of pirfenidone for the treatment of RA associated interstitial lung disease. Approximately 270 subjects will be randomized to receive Pirfenidone 2403 mg per day or placebo in a 1:1 ratio. The primary outcome of this study is to assess the efficacy of pirfenidone 2403 mg/day versus placebo in patients with RA associated interstitial lung disease, as defined by progression free survival over the 52 weeks of treatment. Patients will receive blinded study treatment from the time of randomization until the Week 52 Visit.
Eligible patients aged 18 to 85 years must meet 2010 ACR/EULAR criteria for RA (Aletaha, Neogi et al. 2010) as well as RA-associated ILD, as determined by imaging and, when available, lung biopsy. Patients will be required to have a % predicted FVC ≥40 and % predicted DLCO or TLCO ≥30 at screening.
The dose of study treatment will be titrated over 14 days. Patients will receive a telephone assessment at Weeks 1 and 2, and visit the clinic at Weeks 4, 8, 13, 19, 26, 39, and 52. Subjects will have a follow up phone call 28 days after completion of the study drug. Patients should complete a compliance diary between visits. If patients discontinue study treatment for any reason before the end of the study, they should continue with all scheduled study procedures through Week 52. If subjects are unable to complete the study visits as scheduled, all efforts should be made to complete an early termination visit.
The primary outcome variable of this study will be progression free survival, defined as progression free from decline in FVC of 10% or greater during the 52 week study period.
More information can be found at www.ralung.org.
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 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Pirfenidone
Pirfenidone 2403 mg/d for 52 weeks
干预措施: Pirfenidone (Drug)
Placebo
Placebo for 52 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants Who Developed Any Element of the Composite Endpoint
时间窗: 52 weeks
Number of participants who developed any element of the composite endpoint of decline in percent predicted FVC of 10% or greater or death.
次要结局
- All Cause Hospitalization(52 weeks)
- Acute Exacerbations Requiring Hospitalization(52 weeks)
- Treatment-emergent Adverse Events (AEs)(52 weeks)
- Hospitalization for Respiratory Cause(52 weeks)
- Number of Participants With FVC Decline From Baseline of 10% or Greater(52 weeks)
- All-cause Mortality(52 weeks)
- Change in Absolute Value FVC Over the 52 Week Study Period(52 weeks)
- Change in PRO of Dyspnea(52 weeks)
- Treatment-emergent/Treatment-related AEs(52 weeks)
- Number of Participants With Progressive Disease(52 weeks)
- Time to Composite of Decline in FVC or Death(52 weeks)
- Change in % Predicted FVC From Baseline to End of Study Over the 52 Week Study Period(52 weeks)
- Treatment-emergent Serious Adverse Events (SAEs)(52 weeks)
- Treatment-emergent/Treatment-related SAEs(52 weeks)
- AEs Leading to Early Discontinuation of Study Treatment(52 weeks)
- Treatment-emergent Death or Transplant(52 weeks)
- Treatment-emergent RA-ILD-related Mortality(52 weeks)
研究者
Hilary J. Goldberg, M.D.
Attending Physician
Brigham and Women's Hospital