An Open-Label Study of Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension and Interstitial Lung Disease (SAPPHIRE)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- PVR by Right heart catheterization (RHC)
研究概览
简要总结
This study aims to evaluate the efficacy and safety of inhaled treprostinil in subjects with sarcoidosis-associated interstitial lung disease and pulmonary hypertension.
详细描述
Pulmonary sarcoidosis-associated pulmonary hypertension is classified as WHO Group 5 pulmonary hypertension and may occur in anywhere from 5-20% of sarcoidosis patients. Inhaled treprostinil has shown clinical improvements in exercise capacity after 12 weeks of therapy in patients with WHO Group 1 pulmonary hypertension. More recently, there has been interest in using inhaled PAH-specific therapies for the treatment of pulmonary hypertension associated with interstitial lung disease.
The investigators believe that those patients with pulmonary hypertension in the setting of sarcoidosis-associated interstitial lung disease are a unique population which may potentially benefit from inhaled, targeted pulmonary arterial hypertension therapy (inhaled treprostinil) while minimizing the adverse effects associated with systemic pulmonary vasodilators. This study aims to evaluate the efficacy and safety of inhaled treprostinil in subjects with sarcoidosis-associated interstitial lung disease and pulmonary hypertension.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Study participant willing and able to provide informed consent
- •Negative urine pregnancy test at baseline for females of childbearing potential
- •Established diagnosis of sarcoidosis by ATS/ERS/WASOG 1999 Statement on of Sarcoidosis
- •Presence of interstitial lung disease by Scadding Stage IV chest radiograph or extensive fibrosis on chest computed tomography
- •Right heart catheterization within six months of baseline visit showing precapillary pulmonary hypertension (mPAP ≥ 25 mmHg, PCWP ≤ 15 mmHg, and PVR > 3 WU)
- •Patient on stable sarcoidosis therapy for at least three months prior to screening
- •If patients are on oral PAH therapy (PDE5i/SCGS and/or ERA) then dose should be stable for at least three months prior to screening
- •A 6MWT within three months of screening visit of > 100 meters
排除标准
- •Pregnant patients or those who are actively lactating
- •Patient not willing to use form of birth control (if applicable) during the study
- •Inability to undergo 6MWT, RHC, PFTs or CMRI
- •Predicted survival < 6 months
- •Patient on any prostanoid or prostanoid analog therapy
- •Patients with left sided heart disease as defined by either a PCWP > 15 mmHg and/or left ventricular ejection fraction < 40%
- •Use of any investigational drug/device, or participation in any investigational study with therapeutic intent within 30 days prior to randomization.
研究组 & 干预措施
Study Group
Sarcoidosis patients with interstitial lung disease and precapillary pulmonary hypertension based on right heart catheterization (RHC).
All subjects will initiate inhaled treprostinil at a dose of 3 breaths (18 mcg) four times daily. Study drug doses escalations (additional one breath four times daily) can occur every three days with a maximum dosing regimen of up to 12 breaths (72 mcg) four times daily, as clinically tolerated.
干预措施: Inhaled Treprostinil (Drug)
结局指标
主要结局
PVR by Right heart catheterization (RHC)
时间窗: Baseline, Week 16
Change in RHC parameter PVR (pulmonary vascular resistance )
mPAP by Right heart catheterization (RHC)
时间窗: Baseline, Week 16
Change in RHC parameter mPAP (mean pulmonary arterial pressure)
次要结局
- Change in 6-Minute Walk Test (6MWT)(Baseline, Week 8, Week 16)
- Change in Cardiac MRI parameters(Baseline, Week 16)
- Change in Pulmonary Function Testing(Baseline, Week 16)
- Change in Brain Natriuretic Peptide (BNP)(Baseline, Week 16)
- Change in WHO Functional Class (WHO FC)(Baseline, Week 8, Week 16)