Active clinical trials for "Familial Primary Pulmonary Hypertension"

Pulmonary arterial hypertension (PAH) is a progressive disease that results in severe activity limitation and death. There are few treatments for PAH and the available medications are expensive, difficult to administer and have significant toxicities. (-)-Epicatechin is a non-toxic compound that naturally occurs in foods such as tea, wine and chocolate. Clinical intervention studies using dark chocolate in normal volunteers and subjects at risk for or with established cardiovascular disease have demonstrated improvements in peripheral and coronary vascular endothelial function, blood pressure, lipids, glucose tolerance and inflammatory markers. Our study intends to examine the hemodynamics effects of purified (-)-epicatechin in subjects with pulmonary arterial hypertension. We hypothesize purified (-)-epicatechin will reduce pulmonary vascular resistance in patients with pulmonary arterial hypertension.

This is a multicenter, open-label trial to evaluate the safety of continued therapy with subcutaneous Remodulin® in subjects with pulmonary arterial hypertension (PAH) who complete the CVT-CV-003 study. The study will include about 50 subjects at up to 10 clinical trial centers in China who completed all required assessments in the CVT-CV-003 study. Study visits for data collection will occur at month 6 and 12 with yearly visits beyond 12 months until the study is discontinued by the sponsor.

The investigators have recently evidenced the presence of antibodies to endothelial cells and fibroblasts in patients with idiopathic or SSc-associated PAH. The investigators also have identified several target antigens of anti-fibroblasts antibodies. The objective of this study is to further investigate for the presence of antibodies to endothelial cells and fibroblasts in patients and characterize the antigen specificity of autoantibodies in patients with different types of non idiopathic and non SSc-associated PAH, such as PAH associated with HIV infection, porto-pulmonary hypertension, congenital heart diseases, systemic lupus erythematosus, mixed connective tissue disease and Sjögren's syndrome

The early detection of pulmonary arterial hypertension may help to improve prognosis of the disease. It is assumed that in the early stages of pulmonary arterial hypertension, pulmonary arterial pressure values may be normal at rest, but the remodelling of small arteries leads to stiffening resulting in increased pulmonary arterial pressure during exercise. In the present study we investigate patients with risk factors for pulmonary arterial hypertension (e.g. connective tissue disease) by combining exercise tricuspid echo doppler and cardiopulmonary exercise test to screen patients for exercise-induced pulmonary hypertension and control the results by the gold standard right heart catheterisation at rest and during exercise. We expect that using this screening method, patients with pulmonary arterial hypertension would be recognized earlier.

This study will examine and test healthy volunteers and patients with pulmonary hypertension to try to learn more about the disease and find better ways to detect, treat, and, if possible, slow progression. Pulmonary hypertension is a rare blood vessel disorder of the lung in which the pressure in the pulmonary artery (the blood vessel that leads from the heart to the lungs) rises above normal levels and may become life-threatening. Normal volunteers and patients with pulmonary hypertension 18 years of age and older may be eligible for this study. All candidates are screened with a review of their medical records. Normal volunteers also have a medical history, electrocardiogram, echocardiogram (heart ultrasound), and pulmonary function test, in which the subject breathes in and out of a tube that measures lung volume, mechanics and function. All participants undergo the following tests and procedures: Echocardiogram to measure heart function and blood pressure in the lungs. A small probe held against the chest uses sound waves to obtain pictures of the heart. Magnetic resonance imaging (MRI) to evaluate the heart's pumping action. Subjects lie on a stretcher that slides into a long, tube-shaped scanner. The machine uses a magnetic field and radio waves to obtain images of the heart. 6-minute walk to measure how far the subject can walk in 6 minutes. Subjects walk around the hospital for 6 minutes at a comfortable pace. Exercise testing to measure the ability to exercise and the subject's oxygen levels during exercise. Subjects exercise on a bike or treadmill while the oxygen and carbon dioxide they breathe are measured using a small device placed in the mouth. Right heart catheterization to measure pressure in the heart and lungs. A small catheter (plastic tube) is placed in an arm vein. A longer catheter called a central line is placed in a deeper vein in the neck or just below the neck, or in the leg or arm. A long, thin catheter that measures blood pressure directly is then inserted into the vein and advanced through the chambers of the heart into the lung artery to measure all the pressures in the heart and obtain blood samples. Genetic and protein studies. DNA, RNA, and proteins from blood samples are studied for genes and proteins that might predict the development or progression of pulmonary hypertension. In addition to the above, patients whose pulmonary hypertension was caused by a blood vessel injury undergo the tests described below. The right heart catheter inserted for the catheterization procedure remains in place to obtain measurements of the effects of nitric oxide and nitrite in the following procedures: Inhalation of nitric oxide (a gas naturally produced by cells lining arteries) at 30-minute intervals to examine its effect on lung and heart pressures. Inhalation of aerosolized nitrite at 5-minute intervals to measure its effects on lung and heart pressures. Inhalation of nitric oxide for up to 24 hours to obtain multiple measurements of its effect on lung and heart pressures. Blood draws for laboratory tests. In patients whose pulmonary hypertension was caused by a blood vessel injury, we also plan to follow response to standard therapy. After the initiation of standard therapy, we will restudy the same parameters (excluding NO and sodium nitrite studies) in these patients at approximately 4 months, and yearly for 5 years

A multinational, multicenter, double-blind, randomized, placebo-controlled, Phase III study to assess the efficacy and safety of BPS 314d-MR in subjects with pulmonary arterial hypertension currently receiving treatment with an Endothelin Receptor Antagonist (ERA) and/or a Phosphodiesterase-5 Inhibitor (PDE-5 inhibitor). Approximately 100 centers will be participating in the study. Approximately 630 eligible subjects will be randomized 1:1 into two groups, BPS-314d-MR (active) or placebo study drug.

The main OBJECTIVE of this proposal is to extend the investigator's preclinical findings on the role of epigenetics and DNA damage and Bromodomain-Containing Protein 4 (BRD4) inhibition as a therapy for a devastating disease, pulmonary arterial hypertension (PAH). There is strong evidence that BRD4 plays a key role in the pathological phenotype in PAH accounting for disease progression and that BRD4 inhibition can reverse PAH in several animal models. Intriguingly, coronary artery disease (CAD) and metabolic syndrome are more prevalent in PAH compared with the global population, suggesting a link between these diseases. Interestingly, BRD4 is also a trigger for calcification and remodeling processes and regulates transcription of lipoprotein and inflammatory factors, all of which are important in PAH and CAD. Apabetalone, an orally available BRD4 inhibitor, is now in a clinical development stage with a good safety profile. At this stage, the investigators propose a pilot study to assess the feasibility of a Phase 2 clinical trial assessing apabetalone in the PAH population. The overall HYPOTHESIS is that BRD4 inhibition with apabetalone is a safe and effective therapy for PAH.

Evaluate for accuracy and reproducibility of data collected via the participant-operated Walk.Talk.Track. (WTT) app combined with Apple Watch during in-clinic, technician proctored 6MWT's. Determine whether the WTT app on the Apple Watch can accurately collect information on distance traveled and heart rate (HR) during in-clinic 6MWT run by American Thoracic Society (ATS) guidelines Determine whether participants can operate the WTT app and Apple Watch effectively to gather accurate data in a monitored and home-based setting Prospectively monitor for changes in WTT app recorded 6MWT results following initiation of therapy in a treatment naïve cohort of PAH participants Evaluate whether changes from baseline in 6 minute walk distance (6MWD) and heart rate recovery at one minute (HRR1) as well as other variables that have been associated with disease severity in PAH and left-sided heart disease (resting HR, heart rate variability [HRV], chronotropic index [CI]) can be identified before the 12-week follow up when comparing the treatment arm and the control arm Evaluate whether changes from baseline in the HRR1, resting HR, HRV and/or CI are more evident in treatment responders when compared to treatment non-responders.

This study will determine the safety and feasibility of using a β-blocker (in this case carvedilol) in the treatment of pediatric patients with Left Heart Failure (LHF) in children with Pulmonary Arterial Hypertension (PAH). Carvedilol affects the nervous system, the same system that is highly activated in response to stress in patients with PAH. Each patient is administered a dosage of carvedilol, according to their weight. This dosage is increased incrementally over the span of the study, if the patient responds well to the drug. The study will determine whether the potential adverse side effects of carvedilol outweigh the possible positive results in reducing LHF. The hypothesis of this study predicts that carvedilol will have positive effects in treating LHF, similar to their use in treatment of Right Heart Failure (RHF). This is a single-centered pilot study. Each patient will be studied for approximately 31 weeks.

Open-label extension of RIN-PH-302.