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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
SUPPORTIVE_CARE
Masking
TRIPLE
Enrollment
15
A dose of 18mcg (3 breaths) will be administered 3 times: at baseline, 1.5 hrs. post baseline and then again 2 hrs later following maximal exercise testing.
3 breaths of placebo (via inhalation devices) will be administered 3 times: at baseline, 1.5 hrs. post baseline and then again 2 hrs later following maximal exercise testing.
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Reduction in Liver Stiffness Through the Measurement of Shear Wave Ultrasound Elastography (m/s)
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.
Time frame: At rest and following dose 2
Venous Pressure (mmHg) at Rest and Peak Exercise
Venous pressure was measured both at rest and during an incremental ramp exercise test. Comparison was made to evaluate if the treprostinil study medication had a different effect in venous pressure measurements during peak exercise versus placebo.
Time frame: At rest and at peak incremental exercise
Exercise Endurance Time (in Minutes)
A constant work rate exercise test will be performed to determine exercise endurance
Time frame: Immediately following dose 3
Peak Volume of Oxygen Consumption (VO2) (ml/kg/Min)
Measuring the difference in peak/maximal VO2 during incremental exercise testing to assess if the response during peak exercise is different when utilizing treprostinil versus a placebo.
Time frame: Maximal exercise test (5-10 minutes)
Minute Volume and Carbon Dioxide Volume Slope (VE/VCO2) During Incremental Exercise
Measuring the difference in peak/maximal VE/VCO2 during incremental exercise testing to assess if the response during peak exercise is different when utilizing treprostinil versus a placebo. The VE/VCO2 slope is used to show the relationship between minute ventilation and carbon dioxide production.
Time frame: Maximal exercise test (5-10 minutes)
VO2 (ml/kg/Min) at Anaerobic Threshold
Measuring the difference in VO2 at anaerobic threshold of an incremental exercise test to assess the variance in response with treprostinil versus placebo
Time frame: Maximal exercise test (5-10 minutes)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.