Active clinical trials for "Familial Primary Pulmonary Hypertension"

The primary objectives of the study are to evaluate the safety and tolerability, and pharmacokinetics (PK) of sotatercept over 24 weeks of treatment in children ≥1 to <18 years of age with PAH World Health Organization (WHO) Group 1 on standard of care (SoC). There is no formal hypothesis.

Pulmonary Arterial Hypertension is characterized by a progressive increase in pulmonary vascular resistance inducing shortness of breath and exercise intolerance. We aim to correlate cardiac function (evaluated at rest by right heart catheterism and RMN) to exercise capacity (evaluated by endurance time at 75% of maximal workout), in prevalent patients with pulmonary arterial hypertension, and their evolution at three and twelve months.

Pulmonary arterial hypertension (PAH) is characterized by a progressive increase in pulmonary vascular resistance leading to right ventricular failure and eventually to death. The therapeutic strategy has become complex and needs to perform recurring follow up evaluations including right heart catheterizations (RHC). Cardiac magnetic resonance imaging (cMRI) has the advantage to accurately assess right ventricular volumes and important prognostic predictors such as cardiac index, stroke volume and right ventricular ejection fraction. The main objective of EVITA is to assess the hemodynamic diagnosis performances at baseline and at follow up visits of cMRI in comparison with the results of the RHC (current guidelines) to detect an unfavorable hemodynamic status. The primary endpoint is sensitivity and specificity of cMRI for the diagnosis of an unfavorable status defined by the current RHC criteria (with 95% confidence interval). The secondary objectives are 1) to identify clinical and hemodynamic variables independently contributing to prognosis, 2) to describe complications due to cMRI and to RHC, 3) to compare acceptability and tolerability of cMRI over RHC for the patient and 4) to constitute biological collection of blood samples to determine diagnostic and prognostic PAH biomarkers. PAH patients will be recruited in centers of the French network of severe pulmonary hypertension in a prospective cohort study. 180 subjects will be enrolled in the study: that size will give the study 90% power to find significant at the 5%-level. If the primary endpoint were achieved, since first, strategies and procedures planed in this project are consistent with those currently used in routine and second, inclusion criteria are not limited to a sub-population of PAH patients, positive results could allow to broadly extend our findings. Therefore, it will be possible to decrease the number of RHC, an invasive and cumbersome procedure without altering the prognosis. Moreover, all clinical procedures would be performed in outpatient clinics and thereby would reduce the cost to assess the severity of the disease. Current recommendations for evaluation of severity and follow-up being mainly derived from consensus of opinion of the experts, positive results will also improve the level evidence of severity assessment of PAH patients. According to secondary objectives we expect to better predict morbimortality events with cMRI compared to RHC.

Pulmonary arterial hypertension (PAH) is mortal disease affecting the blood vessels of the lung. Despite its morbid prognosis, PAH is often misdiagnosed or ignored, with an average time of 44 months between onset of symptoms to diagnosis and substantial progression of disease severity. Therefore, a pressing need exists to develop non-invasive diagnostic imaging tools, particularly that can detect early disease stages. Efforts have been made to develop such imaging capabilities through platform development of echocardiography, cardiac MRI, chest computed tomography (CT), and positron emission tomography (PET), among others. While some have demonstrated promise, few have shown a precise ability to offer disease quantifications of the diseased lung and vasculature itself, to detect early stages of disease, and to reflect alterations of the lung, vasculature, and right ventricle that reflect the molecular origins of this disease. [F-18]FGln has been previously utilized in oncology studies as a non-invasive in vivo imaging biomarker of tumor glutamine flux and metabolism. Our preliminary in vivo pre-clinical rodent studies demonstrated that [F-18]FGln demonstrated increased uptake in diseased pulmonary vessels and the right ventricle in a rodent model of PAH. The proposed research study will provide preliminary evidence of the potential to utilize [F-18]FGln as a non-invasive imaging biomarker of glutamine flux and metabolism across a range of PAH subjects.

Study ROR-PH-303, ADVANCE EXTENSION, is an open-label extension (OLE) study for participants with WHO Group 1 PAH who have participated in another Phase 2 or Phase 3 study of ralinepag.

The prevalence of critical ab extrinsic compression of left main coronary artery (LMCA) is very high in patients with pulmonary arterial hypertension (PAH) symptomatic for angina (up to 40% according to a recent study of 121 patients with PAH). The element that most of all correlates with the degree of coronary stenosis is the diameter of the pulmonary artery (PA). In particular, a diameter ≥ 40 mm has a sensitivity of 83% and a specificity of 70% in patients with angina. Critical stenosis of LMCA is a risk factor for sudden death and in these condition percutaneous coronary angioplasty with stent implantation has proven to be a safe and effective long-term procedure. Preliminary data from a retrospective analysis of the registry of patients with PAH in Bologna (ARCA registry, 109/2016/U/Oss) highlights that even in PAH patients asymptomatic for angina, compression of LMCA can occur in up to 13% of patients and the main predictive parameter of compression was found to be a diameter ≥ 42 mm (with a sensitivity of 87% and a specificity of 77%). Performing a screening test by coronary-CT scan in all subjects suffering of PAH with a PA diameter ≥ 40 mm even if asymptomatic for angina could therefore help to identify patients with PAH at increased risk for sudden death at an early stage.

The purpose of the study is to assess the effects of selexipag on right ventricular (RV) function in participants with Pulmonary arterial hypertension (PAH).

The purpose of this study is to investigate the extent to which diet and exercise may improve PAH through the modulation of insulin sensitivity. The central hypothesis is that dysregulated glucose metabolism elicits a response in PAH patients that can be modified by exercise and diet, thereby leading to improvements in pulmonary vascular disease.

The purpose of this study to confirm the selexipag starting dose(s), selected based on pharmacokinetic (PK) extrapolation from adults, that leads to similar exposure as adults doses in children from greater than or equal to (>=) 2 to less than (˂) 18 years of age with Pulmonary Arterial Hypertension (PAH), by investigating the PK of selexipag and its active metabolite ACT-333679 in this population.

This local Phase 3 study is planned to confirm the efficacy and safety in Japanese PAH participants. The primary population of this study is Japanese PAH participants with World Health Organization Functional Class (WHO FC) II or III while the study includes PAH participants with WHO FC I or IV as other populations. There are no hypotheses for this study.