Pulmonary Arterial Hypertension (PAH)

Comparison of the efficacy and safety of three vasoactive agents-dopamine, norepinephrine, and epinephrine-in the treatment of patients with pulmonary hypertension crisis: prospective, randomised controlled trial monitored by haemodynamic monitoring

Pulmonary rehabilitation is a key treatment for lung diseases, but many patients struggle to attend sessions due to travel distances, physical limitations, or logistical barriers. The purpose of this study is to evaluate whether a tele-pulmonary rehabilitation (TPR) program can improve the rates of treatment initiation and adherence among patients with chronic lung diseases compared to traditional, center-based pulmonary rehabilitation. Additionally, the study will assess improvements in quality of life, physical symptoms, and safety in both groups. This study will enroll 90 patients from two medical centers in Israel (Tel Aviv and Barzilai). Participants will be randomly assigned to one of two groups: 1. The control group will receive usual care with a referral by a pulmonologist to standard pulmonary rehabilitation at a medical center. 2. The intervention group will participate in a supervised remote tele-rehabilitation program using a dedicated application and remote monitoring.

This study is researching an experimental drug called REGN13335. The study is focused on participants with Pulmonary Arterial Hypertension (PAH). The aim of the study is to see how safe and effective REGN13335 is in participants with PAH who are taking other PAH medicines. The study is looking at several other research questions, including: * What side effects may happen from taking REGN13335 * How much REGN13335 is in the blood at different times * Whether the body makes antibodies against REGN13335 (which could make REGN13335 less effective or could lead to side effects)

Pulmonary arterial hypertension (PAH) is a rare, progressive disease characterized by structural changes in the pulmonary arteries, leading to increased pulmonary vascular resistance and elevated pulmonary arterial pressure and, if untreated, right heart failure. Diagnosis requires a comprehensive evaluation, including right heart catheterization performed in specialized centers. Despite advances in the understanding and management of the disease, PAH remains a severe condition. Current approved therapies primarily target three key pathways involved in endothelial dysfunction: the endothelin, nitric oxide, and prostacyclin pathways. Pulmonary arterial remodeling is characterized by alterations in endothelial cells, smooth muscle cells, and fibroblasts, with fibroblast activation and macrophage involvement contributing to disease progression. Two positron emission tomography/computed tomography (PET/CT) imaging approaches are currently under investigation in PAH. \[⁶⁸Ga\]Ga-FAPI PET/CT targets activated fibroblasts and enables noninvasive assessment of fibroblast activity and tissue remodeling. \[⁶⁸Ga\]Ga-MAA lung perfusion PET/CT is an emerging imaging technique that provides higher spatial resolution and sensitivity than conventional lung perfusion imaging and allows evaluation of regional pulmonary perfusion. Sotatercept is a novel fusion protein that modulates signaling within the transforming growth factor-beta (TGF-β) superfamily by binding select ligands involved in vascular remodeling. Its mechanism of action is distinct from that of currently approved PAH therapies. Sotatercept has been evaluated in clinical development programs, including the PULSAR and STELLAR studies. Reported adverse events include epistaxis, dizziness, increased hemoglobin levels, and changes in blood pressure. This study is designed with the following objectives: Primary objective: To assess pulmonary vascular remodeling in patients with PAH using \[⁶⁸Ga\]Ga-FAPI PET/CT imaging. Secondary objectives: To evaluate \[⁶⁸Ga\]Ga-FAPI uptake and regional lung perfusion using \[⁶⁸Ga\]Ga-MAA lung perfusion PET/CT imaging at predefined study time points.

Background: A number of diseases can cause a type of lung injury called pulmonary arterial hypertension (PAH). Most people who develop PAH do not survive more than a few years. A new study drug (ZMA001) may help. ZMA001 is a monoclonal antibody. This type of drug consists of proteins, made in a facility, that are very similar to proteins in a human body. But before giving ZMA001 to people sick with PAH, researchers want to find out how the drug affects healthy people. Objective: To test a drug (ZMA001) in healthy volunteers. Eligibility: Healthy adults aged 18 to 60 years. Design: Participants will be screened. They will have a physical exam with blood tests. They will have a urine test for drug use. They will have a test of their heart function. Participants will come to the clinic for 1 inpatient visit of up to 48 hours. ZMA001 is a liquid administered through a tube attached to a needle inserted into a vein in the arm. Participants will receive this drug only once, during their inpatient stay. Some participants will receive the drug; others will receive a placebo in Cohort 1 only. A placebo is a treatment that looks just like the real drug but contains no medicine. Participants will not know which treatment they are getting in Cohort 1. Cohorts 2-4 will receive a single dose of the study drug, administered through a tube attached to a needle inserted into a vein in the arm. After a screening visit, participants will have 1 inpatient visit and up to 8 outpatient visits over 16 weeks after receiving the treatment. Blood draws and other tests will be repeated. Each outpatient visit is approximately 2 hours long. This study is the first time ZMA001 will be administered to people.

This is a multicenter, open-label extension (OLE), single-arm study designed to assess the long-term safety and tolerability of oral IKT-001 administered once daily.

This study evaluates the effect of ROC-101 in adults with either Pulmonary Arterial Hypertension (PAH) or Pulmonary Hypertension Associated with Interstitial Lung Disease (ILD-PH). Each eligible participant will receive standard of care (SOC) plus ROC-101 for a 24-week treatment period, followed by a long-term extension period of the study through the end of the program or marketing approval/authorization.

This study evaluates the effects of VI-0106 (an extended-release formulation of tacrolimus) in participants with pulmonary arterial hypertension (PAH) who continue to have functional limitations despite being on optimized background PAH therapy. Participants will be randomly assigned with equal chance to receive either VI-0106 or placebo in a double-blind fashion to assess whether VI-0106 improves outcomes in this population.

The primary objective of this study is to compare the efficacy, safety, and tolerability of tiprelestat plus Standard of Care (SOC) compared with placebo plus SOC in patients with World Health Organization (WHO) functional class II-IV pulmonary arterial hypertension (PAH).

This is a Phase Ib/IIa clinical study of IMC-003 treatment in pulmonary arterial hypertension (PAH) patients receiving background therapy

Pulmonary arterial hypertension (PAH) is a rare, progressive disease associated with poor prognosis, especially in patients with cardiovascular comorbidities. Current guidelines recommend initial combination therapy, but evidence is lacking for patients with significant comorbidities who are often excluded from clinical trials. The COMMODITIES trial is a multicenter, randomized, controlled study designed to compare the efficacy and safety of initial dual oral combination therapy (tadalafil and ambrisentan) versus oral monotherapy in newly diagnosed PAH patients with at least two cardiovascular comorbidities. The study aims to provide robust evidence to guide treatment strategies in this high-risk population.

This study aims to develop and validate a culturally appropriate, reliable, and clinically applicable patient-reported outcome (PRO) instrument to assess quality of life in patients with pulmonary arterial hypertension (PAH) in China. The study includes two phases: (1) scale development using literature review, Delphi expert consultation, and patient interviews; and (2) multicenter prospective observational validation to evaluate reliability, validity, and responsiveness. The final scale is expected to support clinical assessment, treatment monitoring, and health policy decision-making.

This is a 20-week, Phase 1, single-center, open-label, dose-escalation study evaluating the safety and tolerability of daily oral artesunate in patients with PAH.

The goal of this study is to learn more about how sotatercept works and if it helps the lung arteries become healthier. Sotatercept will be associated with the following: 1. Improvement in capillary blush, reduce the tapering and tortuosity of affected vessels on pulmonary wedge angiography and decreased wall thickness on intravascular ultrasound in previously affected areas. 2. Improvement in previously poorly or non-perfused areas rather than increased perfusion to previously perfused areas. 3. No changes in baseline ventilation and improvement and ventilation/perfusion matching.

This methodological study aims to develop and validate a questionnaire named for the Assessment of Patient Knowledge in Pulmonary Arterial Hypertension. The study will be conducted in four phases: (1) questionnaire development based on guidelines and literature; (2) content validation by expert judges; (3) semantic validation with patients; and (4) psychometric testing in a sample of up to 200 patients with confirmed pulmonary arterial hypertension (PAH). In addition to validation, the study will collect clinical and functional data from medical records, including risk stratification using the COMPERA 2.0 method The final instrument is expected to support patient education strategies and contribute to improved clinical management of PAH.

Pulmonary Arterial Hypertension is a rare and progressive condition that compromises pulmonary circulation and can lead to right ventricular failure. Despite recent advances in diagnosis and treatment, the median survival of patients is only 2.8 years. The treatment for this disease is based on drugs that act on three main pathways: prostacyclin, endothelin, and nitric oxide. Iloprost, a prostacyclin analogue available in an inhaled form, is an important and well-established treatment. However, its mandatory frequent administration, the need for a specific inhalation technique, and its adverse event profile make its use complex. Although pharmacotherapeutic and inhalation technique follow-up by a qualified professional is widely studied in diseases like asthma and COPD, its application in Pulmonary Arterial Hypertension still lacks evidence. Thus, this study aims to evaluate how a pharmacist's intervention can improve treatment adherence, mitigate side effects and difficulties associated with inhalation, in addition to optimizing clinical and hemodynamic outcomes in patients with Pulmonary Arterial Hypertension using iloprost.

Schistosomiasis-associated pulmonary arterial hypertension is a serious condition that can lead to shortness of breath, heart failure, frequent hospitalizations, and early death. Although treatments for pulmonary arterial hypertension have improved over time, patients with this specific cause of the disease are often not included in long-term studies. Selexipag is an oral medication used to treat pulmonary arterial hypertension and is part of routine clinical care in Brazil. Its long-term effects in patients with schistosomiasis-associated pulmonary arterial hypertension are not well understood. The PROPULSE-Sch study aims to evaluate long-term clinical outcomes in patients with schistosomiasis-associated pulmonary arterial hypertension who received selexipag, compared with similar patients who did not receive this medication before it became available at the study center. This is an observational study using data from routine medical care. All treatments are prescribed by the treating physicians, and participation in the study does not change patient care. The results may help improve understanding of long-term outcomes and support treatment decisions in this population.

Pulmonary arterial hypertension (PAH) is a rare and incurable disease affecting people of all ages. It is characterized by obstructive remodeling of the small pulmonary arteries, responsible for an increase in pulmonary arterial pressure, leading to right heart failure and death in the absence of treatment. PAH can be associated with a variety of diseases, but around half of all PAH cases are idiopathic or hereditary, and may develop on predisposed terrain following a "second hit", as suggested by the identification of PAH cases associated with the use of anorectic drugs, methamphetamine and occupational exposure to organic solvents. No study has systematically analyzed the exposome of patients with PAH, combining environmental and occupational exposures as well as drugs and medications. The exposome of patients with PAH without associated causes will be compared with that of patients with another form of pulmonary hypertension (PH), linked to thromboembolic risk factors: chronic thromboembolic PH (CTEPH), which will constitute the control group.

Pulmonary arterial hypertension (PAH) is a rare and serious condition that affects the blood vessels of the lungs and can significantly limit daily activities and quality of life. Some patients with PAH use inhaled iloprost, a medication that requires several inhalations per day, which can be difficult to maintain over time. Oral selexipag is an alternative treatment that may reduce treatment burden and improve adherence. The PROMISE study aims to evaluate how switching from inhaled iloprost to oral selexipag affects patients' quality of life, satisfaction with treatment, and adherence in real-world clinical practice. Patient-reported outcome questionnaires will be used to understand patients' perceptions of symptoms, daily functioning, and overall improvement after the transition. Adult patients with PAH who are receiving inhaled iloprost and whose physicians decide to switch treatment to oral selexipag will be followed over time. A comparison group of patients who continue using inhaled iloprost will also be observed. The study does not involve any experimental treatment or changes to routine clinical care. All medications are prescribed as part of standard medical practice. The results of this study may help improve understanding of the patient experience during treatment transitions in PAH and support more patient-centered treatment decisions.

The goal of this clinical trial is to evaluate the safety and potential efficacy of PNEUMOSTEM® for improving respiratory outcomes in very premature infants diagnosed with Early Pulmonary Arterial Hypertension. The main questions it aims to answer are: * In very premature infants diagnosed with early pulmonary arterial hypertension, will a single intratracheal administration of PNEUMOSTEM®(Allogeneic umbilical cord blood-derived mesenchymal stem cells) result in improvement of pulmonary arterial hypertension based on echocardiographic assessment? * In very premature infants diagnosed with early pulmonary arterial hypertension who show improvement of pulmonary arterial hypertension based on echocardiographic assessment following a single intratracheal administration of PNEUMOSTEM®(Allogeneic umbilical cord blood-derived mesenchymal stem cells), at what time point does this improvement occur? Participants will: * Single intratracheal dose of PNEUMOSTEM® at 2.0 x 10,000,000 cells/kg * Acute adverse event monitoring: 24 hours post-administration for safety assessment * Follow- up time points: Day 1(Baseline, PNEUMOSTEM® administration), Day 2, Week 1, Week 2, Postnatal Day 28, PMA 36\~40 weeks

Congenital portosystemic shunt (CPSS) are rare vascular malformations causing blood from the intestines to bypass the liver and directly flow into body's general circulation. Such liver bypass can cause several health problems, one of the most severe being portopulmonary hypertension (PoPH). The goal of this study is to identify pathogenic and potentially pathogenic genetic variants in patients who have both CPSS and PoPH. Future research will assess the contribution of these genetic variants to the development of PoPH. The long-term goal is to use genetic information to identify patients with congenital portosystemic shunts (CPSS) or chronic liver disease who are at risk of developing PoPH to offer anticipatory management. Children and adult patients with both CPSS and PoPH, as well as their close relatives (patient's parents and siblings) can take part in the study. Genetic variations within each family will be studied.

The study aims to see how 24 weeks of triple therapy-an endothelin receptor antagonist (ERA), a phosphodiesterase-5 inhibitor (PDE5i), and sotatercept-affects pulmonary vascular resistance (PVR) in patients with newly diagnosed pulmonary arterial hypertension (PAH). SIRIUS is a 24-week, single-arm, open-label study with up to 42 days of screening and a 28-day safety follow-up. It will enroll 25 patients and will be conducted only in countries where all treatments are available and covered. After 24 weeks, PAH treatment is decided by the doctor.

Pulmonary hypertension is a serious disease that affects patients' health, daily life, and emotional well-being. Many patients and their caregivers actively look for information to better understand the condition and its treatment. However, the quality of information found in different sources, such as the internet, social media, health professionals, and patient groups, can vary. This study will use a short questionnaire to learn where patients and caregivers search for information, what topics they look for, how satisfied they are with what they find, and which sources they trust most. The results will help improve communication strategies, educational materials, and support programs for people living with pulmonary hypertension and their families.

Pulmonary arterial hypertension (PAH) is a rare, progressive, and potentially fatal disease characterized by increased pulmonary vascular resistance and right ventricular dysfunction. Among the four major molecular pathways involved in PAH pathophysiology-nitric oxide, prostacyclin, activin, and endothelin-1 (ET-1)-the endothelin pathway plays a central role. Endothelin-1 acts on ETA and ETB receptors, inducing vasoconstriction and vascular remodeling. Endothelin receptor antagonists (ERAs) are cornerstone therapies in PAH. Ambrisentan is selective for ETA and associated with a lower risk of hepatotoxicity. Bosentan, a dual ERA (ETA/ETB), has well-established efficacy but a higher incidence of liver enzyme elevation, with approximately 9% of patients experiencing hepatic side effects and about 2% discontinuing therapy due to hepatotoxicity. While transitions between ERAs occur in routine clinical practice, data on their clinical impact are scarce. This prospective, observational, single-center cohort study aims to evaluate the effect of switching from ambrisentan to bosentan on risk stratification using the COMPERA 2.0 and REVEAL Lite 2.0 scores at 3-6 months post-switch. Secondary outcomes include variations in functional class (WHO/NYHA), 6-minute walk distance (6MWD), NT-proBNP levels, incidence of adverse events (with a focus on hepatotoxicity), and hematologic parameters such as anemia. The study will enroll adult patients (≥18 years) with confirmed PAH by right heart catheterization who have undergone a documented switch from ambrisentan 10 mg to bosentan 125 mg within the last 6 months. The primary endpoint is the proportion of patients whose risk category changes post-transition according to COMPERA 2.0 and REVEAL Lite 2.0. The results are expected to provide clinically relevant insights into therapeutic decisions involving ERA transitions in PAH management.

Understanding Delays in the Diagnosis of Pulmonary Arterial Hypertension and Rare Diseases in Brazil: A Multicenter Observational Study \--- Pulmonary arterial hypertension (PAH) is a rare, progressive, and life-threatening disease that affects the arteries of the lungs and the right side of the heart. Early diagnosis is essential to initiate appropriate treatment and improve patient outcomes. However, worldwide studies show that there is often a significant delay between the onset of symptoms and the final diagnosis. This delay may lead to disease progression and worse survival. This multicenter observational study aims to understand the time from the first symptoms to the diagnosis of PAH and other rare diseases across several Brazilian reference centers. By analyzing medical records and patient journeys, the investigators intends to identify factors contributing to delayed diagnosis and potential opportunities for earlier detection. The study includes adult patients diagnosed with PAH or other selected rare diseases within the last five years. The investigators will analyze time to diagnosis, number and type of physicians consulted, tests performed, and possible misdiagnoses. Our goal is to support the development of strategies that reduce diagnostic delay and improve access to specialized care for people living with rare diseases. This study does not involve any intervention and poses no additional risk to participants, as it is based solely on retrospective data from medical records.