Multicenter, Randomized, Open Label Trial to Evaluate the Safety and Efficacy of Oral Treprostinil in Subjects With Pulmonary Hypertension Associated With Pulmonary Fibrosis
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 试验地点
- 7
- 主要终点
- Change in pulmonary vascular resistance (PVR)
研究概览
简要总结
This multicenter, randomized, open-label study will assess the safety and efficacy of oral treprostinil in subjects diagnosed with pulmonary hypertension associated with pulmonary fibrosis.
详细描述
This study will evaluate how oral treprostinil can affect pulmonary vascular resistance, as assessed by right heart catheterization, and exercise capacity, as assessed by the Six-Minute Walk Test. This is a 16-week study that will involve at least 10 clinical trial centers. The expected enrollment period is around 24 months and 52 subjects will be entered into 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
- None
入排标准
- 年龄范围
- 18 Years 至 79 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Voluntarily gives informed consent
- •Must meet criteria for lung transplant referral but is not required to be on an active lung transplant list
- •Stable and significant diffuse parenchymal lung disease with a diagnosis of interstitial lung disease
- •6MWD greater than or equal to 75 meters
- •Right heart catheterization with a mean pulmonary arterial pressure >= 30 mgHg, pulmonary capillary wedge pressure <= 15 mmHg and pulmonary vascular resistance > 240 dynes
- •Echo-Doppler examination showing evidence of right ventricular dysfunction and normal left diastolic ventricular function
- •Either not receiving any PAH-approved oral therapy, or is receiving monotherapy (ERA, PDE-5I, or riociguat) for > 60 days and receiving a stable dose for > 30 days prior to enrollment
- •Able to communicate effectively with study personnel and will to be cooperative with protocol requirements
排除标准
- •History of repaired or unrepaired congenital heart disease
- •Received prostanoid therapy in the past 30 days, or has shown an intolerance or lack of efficacy to prostanoid therapy resulting in discontinuation or inability to titrate prostacyclins
- •Diagnosis of sarcoidosis
- •History of thromboembolic disease
- •Chronic renal insufficiency
- •Pregnancy or lactating
- •Currently receiving an investigational drug, has an investigational device in place, or has participated in an investigational drug or device study within 30 days prior to Screening
研究组 & 干预措施
Individual Maximum Tolerated Dose (iMTD)
Subjects are allowed to titrate oral treprostinil up to a maximum dose of 12 mg TID.
干预措施: Oral treprostinil (Drug)
Fixed Dose (FD)
Subjects are allowed to titrate oral treprostinil up to a maximum dose of 1 mg TID.
干预措施: Oral treprostinil (Drug)
结局指标
主要结局
Change in pulmonary vascular resistance (PVR)
时间窗: Baseline to Week 16
Change in PVR between iMTD and fixed dose groups
次要结局
- Change in Six-minute walk distance (6MWD)(Baseline to Week 16)
- Change in N-terminal pro-brain natriuretic peptide (NT-ProBNP)(Baseline to Week 16)