An Open-Label Study to Assess the Safety and Efficacy of Treprostinil to Facilitate Liver Transplantation in Patients With Portopulmonary Hypertension
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 13
- 试验地点
- 4
- 主要终点
- Number of Subjects Who Achieved Hemodynamic Parameters Appropriate for Orthotopic Liver Transplantation Candidacy at Week 24.
研究概览
简要总结
This was a multicenter, prospective, observational, open-label study. Patients meeting inclusion/exclusion criteria received treatment with treprostinil as recommended by their treating physicians and were followed according to standard of care. This observational study collected clinical data and biologic specimens from patients who were treated for portopulmonary hypertension (PoPH), with a goal of achieving hemodynamic parameters appropriate for orthotopic liver transplantation candidacy, including mean pulmonary arterial pressure (mPAP) less than 35 mmHg and pulmonary vascular resistance (PVR) less than 3 Wood-units (WU) at Week 24 in patients with severe PoPH.
详细描述
Treprostinil is approved as a continuous subcutaneous (SC) or intravenous (IV) infusion by the FDA for the treatment of WHO group I PAH with New York Heart Association (NYHA) Functional Class II, III or IV symptomatology. To date, treprostinil has not been studied in the setting of PoPH; however, it is commonly prescribed in this setting. This was an observational, open-label, multicenter study which documented the safety and efficacy profile of this agent in PoPH to facilitate orthotopic liver transplantation (OLT).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must:
- •Had portal hypertension.
- •Be otherwise suitable candidates for OLT.
- •Had severe pulmonary arterial hypertension (PAH) defined as a resting mean pulmonary arterial pressure (mPAP) >35 mmHg and pulmonary vascular resistance (PVR) ≥3 Wood Units (WU) by right heart catheterization (RHC) performed as part of standard of care evaluation within 90 days of enrollment.
- •Treprostinil therapy must be recommended by the treating physician per standard of care.
- •Be NYHA Functional Class II, III, or IV.
- •Had pulmonary capillary wedge (PCW) pressure ≤18 mmHg and transpulmonary gradient (TPG) ≥15 mmHg.
排除标准
- •Patients must not:
- •Had received any any investigational therapy as part of a clinical trial for any indication within 30 days prior to enrollment.
- •Had a change in dose of treatment for PAH (bosentan [Tracleer], ambrisentan [Letairis], tadalafil [Adcirca], or sildenafil [Revatio]), within 30 days prior to enrollment. That is, subjects may have been treated with any of these agents provided the dose was stable for at least 30 days prior to enrollment.
- •Had renal failure requiring hemodialysis.
研究组 & 干预措施
Portopulmonary hypertension
干预措施: Treprostinil (Drug)
结局指标
主要结局
Number of Subjects Who Achieved Hemodynamic Parameters Appropriate for Orthotopic Liver Transplantation Candidacy at Week 24.
时间窗: 24 Weeks
The primary efficacy endpoint was the number of subjects who achieved a mean pulmonary arterial pressure (mPAP) less than 35 mmHg and a pulmonary vascular resistance (PVR) less than 3 Wood units (WU) at Week 24 in patients with severe portopulmonary hypertension (PoPH).
次要结局
- Change in Hemodynamic Parameters (Via Right Heart Catheterization [RHC]) at Rest From Baseline to Week 24(24 weeks)
- Change in Heart Rate at Rest From Baseline to Week 24(24 weeks)
- Change in Cardiac Output at Rest From Baseline to Week 24(24 weeks)
- Change in Arterial and Venous Oxygen Saturation at Rest From Baseline to Week 24(24 weeks)
- Change in Pulmonary Vascular Resistance (PVR) at Rest From Baseline to Week 24(24 weeks)
- Change in 6-minute Walk Distance (6MWD) From Baseline to Weeks 12 and 24.(Baseline and Weeks 12 and 24)
- Change in Echocardiogram Parameters (Right Atrium and Right Ventricle Area) From Baseline to Weeks 12 and 24(Baseline and Weeks 12 and 24)
- Change in Echocardiogram Parameters (Right Ventricle Diameter) From Baseline to Weeks 12 and 24(Baseline and Weeks 12 and 24)
- Change in Echocardiogram Parameters (Right Ventricular Systolic Pressure) From Baseline to Weeks 12 and 24(Baseline and Weeks 12 and 24)
- Change in Echocardiogram Parameters (Tricuspid Annular Plane Systolic Excursion) From Baseline to Weeks 12 and 24(Baseline and Weeks 12 and 24)
- Change in Quality of Life From Baseline to Weeks 12 and 24(Baseline and Weeks 12 and 24)
- Change in Plasma Brain N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Weeks 12 and 24(Baseline to Weeks 12 and 24)