A Randomized, Placebo-Controlled Pilot Study to Determine the Acute Effects of Inhaled Treprostinil on Exercise, Vascular Function, and Exercise Induced Liver Stiffness in Fontan Patients
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Reduction in Liver Stiffness Through the Measurement of Shear Wave Ultrasound Elastography (m/s)
研究概览
简要总结
In this pilot study investigators propose to explore the effects of a locally delivered inhaled pulmonary vasodilator (inhaled treprostinil) on exercise performance, pulmonary blood flow, venous pressure response and vascular function in stable Fontan patients. Investigators will also assess the effects of resting and acute rises in exercise induced systemic venous pressure on liver stiffness and will also assess whether treprostinil will attenuate the acute stiffness increase that investigators expect to see. This will be a prospective, randomized, double-blinded placebo controlled, crossover trial. Following recruitment and informed consent, each participant will undergo three study visits, including baseline testing (visit 1), followed by two sets of exercise and vascular function tests (visit 2 and visit 3) at CCHMC. Twenty-six patients will be enrolled in this study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Supportive Care
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients age 18 years and older
- •Single ventricle patients status post Fontan procedure
排除标准
- •Clinically unstable: these are patients who are experiencing new cardiovascular symptoms such as worsening shortness of breath, new onset arrhythmia, uncontrolled heart failure, or evidence of clinically significant cirrhosis or renal failure.
- •Evidence of Fontan pathway or intra cardiac obstruction as identified on prior clinically indicated imaging studies (echocardiography and MRI);
- •Evidence of left or right systemic ventricular systolic dysfunction with an Ejection fraction of <40% on either echocardiogram or MRI from previously documented clinical data;
- •Presence of uncontrolled arrhythmias;
- •Unable to perform exercise testing for any reason or if deemed by the PI or designee that exercise testing would not be in the best interest of the participant
- •Currently pregnant and/or breastfeeding
- •Patient unable to provide informed consent
- •BMI > 30 mg/m2
研究组 & 干预措施
Treprostinil
A dose of 18mcg (3 breaths) will be administered using the Tyvaso® (treprostinil) inhalation system. Tyvaso® (treprostinil) inhalation solution is supplied in 2.9 mL clear ampules packaged as four ampules in a foil pouch. Frequency and duration- 3 times over 1 study visit.
干预措施: Treprostinil (Drug)
Placebo
A dose of 3 breaths of placebo will be administered using the Tyvaso® (treprostinil) inhalation system. Placebo will be supplies in matching ampules to treprostinil. Volume will match that of treprostinil. Frequency and duration- 3 times over 1 study visit.
干预措施: Placebo (Drug)
结局指标
主要结局
Reduction in Liver Stiffness Through the Measurement of Shear Wave Ultrasound Elastography (m/s)
时间窗: At rest and following dose 2
At rest and then following maximal exercise, shear wave ultrasound elastography will be performed to assess if the study medication shows an improvement in liver stiffness (reduction in m/s number) following maximal exercise versus at rest.
次要结局
- Minute Volume and Carbon Dioxide Volume Slope (VE/VCO2) During Incremental Exercise(Maximal exercise test (5-10 minutes))
- Venous Pressure (mmHg) at Rest and Peak Exercise(At rest and at peak incremental exercise)
- Exercise Endurance Time (in Minutes)(Immediately following dose 3)
- VO2 (ml/kg/Min) at Anaerobic Threshold(Maximal exercise test (5-10 minutes))
- Peak Volume of Oxygen Consumption (VO2) (ml/kg/Min)(Maximal exercise test (5-10 minutes))