Clinical Research Directory

A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY)

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.

A Study of Inhaled Treprostinil (LIQ861) in Adults With Pulmonary Arterial Hypertension Transitioning From Oral Selexipag

This study will look at safety and tolerability of LIQ861 (treprostinil) inhalation powder in adults with pulmonary arterial hypertension (PAH, WHO Group 1) who switch from selexipag, a medicine taken by mouth, to treprostinil, a medicine breathed into the lungs. Everyone who takes part will receive LIQ861. On the first study day, participants will stop taking selexipag and begin taking LIQ861 four times a day using a small handheld dry powder inhaler. The dose may be increased over time based on how well it is tolerated. Participants will take LIQ861 for 16 weeks. The study will also look at whether switching to LIQ861 affects walking distance, a blood marker of heart strain, and symptoms reported by participants.

PAH-SDB: Sleep-Disordered Breathing and Outcomes in Pulmonary Arterial Hypertension

Pulmonary arterial hypertension (PAH) is a progressive pulmonary vascular disease characterized by increased pulmonary vascular resistance, right ventricular dysfunction, and clinically important morbidity and mortality. Sleep-disordered breathing (SDB) and sleep-related hypoxemia are common in patients with PAH, but their prevalence, physiological determinants, longitudinal trajectories, prognostic significance, and potential role in individualized management remain incompletely defined. PAH-SDB is a prospective, multicenter, observational patient registry enrolling adults with right-heart-catheterization-confirmed World Health Organization Group 1 PAH at pulmonary vascular centers across China. Participants will undergo standardized nocturnal sleep and cardiorespiratory monitoring together with comprehensive PAH assessment, including clinical characteristics, functional status, biomarkers, echocardiography, hemodynamics, treatment, and outcomes. Selected participants may undergo additional polysomnography, cardiac magnetic resonance imaging, wearable monitoring, repeated sleep assessments, or biospecimen collection when available and ethically approved. The registry will characterize the prevalence and phenotypes of sleep-disordered breathing and nocturnal physiological abnormalities; examine their relationships with PAH severity and right ventricular-pulmonary vascular function; determine their value for predicting clinical worsening, hospitalization, transplantation, and mortality; evaluate longitudinal changes in sleep physiology in relation to PAH treatment and disease trajectory; and develop and validate multimodal statistical and artificial-intelligence models for phenotyping, risk prediction, and treatment-response stratification. Clinical management will remain at the discretion of treating physicians, and the registry will not assign PAH or sleep-related therapies.

Study of REGN13335 in Adult Participants With Pulmonary Arterial Hypertension (PAH)

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)

Tele-Pulmonary Rehabilitation For Patients With Chronic Lung Diseases

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.

Exercise Performance on Ambient Air vs. Low-Flow Liquid Oxygen Therapy in Chronic Lung Diseases

The investigators aim to study the effect of liquid oxygen therapy (4l/min, nasal) )in Swiss residents with pulmonary vascular diseases (PVD) defined as pulmonary arterial hypertension (PAH) or chronic thromboembolic pulmonary hypertension (CTEPH).

Exercise Performance on Ambient Air vs. Low-Flow Liquid Oxygen Therapy in Pulmonary Vascular Disease (PVD)

The investigators aim to study the effect of liquid oxygen therapy (4l/min, nasal) )in Swiss residents with pulmonary vascular diseases (PVD) defined as pulmonary arterial hypertension (PAH) or chronic thromboembolic pulmonary hypertension (CTEPH).

Assessment of Six-minute Walking Distance 6MWD Between High Altitude HA vs. Sea Level LA in Patients With Pulmonary Arterial Hypertension PAH

To study the effect of relocation from high altitude (Quito: 2850m) to sea level on six-minute walking distance and scores in patients with pulmonary arterial hypertension PAH, who live permanently \>2600m.

Assessment of Lung Function (Lufu) at Sea Level LA vs. High Altitude HA in Patients With Pulmonary Arterial Hypertension PAH

To study the effect of a re-ascent to high altitude (Quito Ecuador: 2850m) after a one-week stay at sea level (after prior descent from 2850m) on lung function and pulmonary resistance/reactance in patients with pulmonary arterial disease PAH, who live permanently \> 2600m.

Assessment of Total Pulmonary Resistance sPAP/CO Between High Altitude HA vs. Sea Level LA in Patients With Pulmonary Arterial Hypertension PAH

To study the effect of relocation from high altitude (Quito Ecuador: 2850m) to sea level on pulmonary resistance (sPAP/CO) and other hemodynamic outcomes in patients with pulmonary arterial hypertension PAH, who live permanently \>2600m.

Assessment of Total Pulmonary Resistance sPAP/CO Between Sea Level LA vs. High Altitude HA After Re-ascent in Patients With Pulmonary Arterial Hypertension PAH

To study the effect of a re-ascent after a one-week stay at sea level (after prior descent from 2850m) to high altitude (Quito Ecuador: 2850m) on pulmonary resistance (sPAP/CO) and other hemodynamic outcomes in patients with pulmonary arterial hypertension PAH, who live permanently \>2600m.

Assessment of Heart Rate Variability HRV During Descent DE From 2850m to Sea Level and During Re-ascent A From Sea Level to 2850m in Patients With Pulmonary Arterial Hypertension PAH

To study the effect of relocation from high altitude (Quito Ecuador: 2850m) to sea level and re-ascent from sea level to 2850m on heart rate variability and other hemodynamic parameters in patients with pulmonary arterial hypertension PAH, who live permanently \>2600m.

Sleep-Assessment in Patients Between High Altitude HA vs. Sea Level LA in Patients With Pulmonary Arterial Hypertension PAH

To study the effect of relocation from high altitude (Quito Ecuador: 2850m) to sea level on sleeping parameters by polygraphy in patients with pulmonary arterial hypertension PAH, who live permanently \>2600m.

Telerehabilitation in Pulmonary Arterial Hypertension

Context: Pulmonary arterial hypertension (PAH) causes severe exercise intolerance and reduced quality of life, while access to supervised rehabilitation remains limited. Objective: Evaluate the safety, feasibility, and clinical effects of an 8-week structured home-based telerehabilitation program in stable PAH patients. Methods: A multicenter, prospective, single-arm study including 34 adults with Group 1 PAH (WHO functional class II-III) performing three weekly exercise sessions via a digital platform. Outcomes: The primary endpoint is peak oxygen consumption (VO₂peak) via cardiopulmonary exercise testing, alongside 6-minute walk distance, quality of life questionnaires, inflammatory biomarkers, and safety tracking.

TREPROSTINIL in Pulmonary Hypertension: Evidence and Clinical Trends

TRESPECT is an international, multicenter, observational registry designed to describe the real-world use, effectiveness, and tolerability of parenteral treprostinil in adults with newly diagnosed pulmonary arterial hypertension (PAH). The registry will include patients who initiated subcutaneous or intravenous treprostinil within 12 months of PAH diagnosis. Both retrospective and prospective data will be collected from routine clinical care. Data will include patient characteristics, PAH treatment patterns, treprostinil dosing and route of administration, functional status, exercise capacity, biomarkers, echocardiographic and hemodynamic parameters, clinical events, and adverse events. Participants will be followed for at least 36 months or until a registry termination criterion is met. The registry is expected to enroll approximately 100 to 300 patients across specialized PAH centers in several European countries.

Randomized Study of Triple Therapy vs Sildenafil Dose Optimization in Pulmonary Arterial Hypertension

Pulmonary arterial hypertension (PAH) is a rare and progressive disease characterized by increased pressure in the pulmonary arteries, leading to right heart failure and premature death. Although combination therapy has improved outcomes, many patients remain at intermediate or high clinical risk despite treatment. When patients do not reach low-risk status, treatment escalation is recommended. However, different escalation strategies are used in clinical practice, including increasing the dose of existing medications or adding a third drug that targets a different biological pathway. There is limited prospective randomized evidence directly comparing these approaches. The ASCEND-PAH study is a prospective, randomized, open-label clinical trial designed to compare two therapeutic escalation strategies in adults with PAH who remain at intermediate or high risk despite dual therapy with an endothelin receptor antagonist and sildenafil. Participants will be randomized to either: (1) escalation to triple therapy with the addition of a prostacyclin pathway agent, or (2) optimization of dual therapy by increasing the dose of sildenafil. The primary objective is to compare the proportion of patients who improve their risk category according to the COMPERA 2.0 four-stratum risk model within 3 to 6 months after randomization. Secondary outcomes include changes in functional status, exercise capacity, biomarkers, clinical worsening, safety, and treatment persistence

What is the Role of the Exposome in Pulmonary Hypertension

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.

The Impact of ERA Switching on Risk Stratification in Pulmonary Arterial Hypertension

Pulmonary arterial hypertension (PAH) is a rare, progressive, and potentially life-threatening disease characterized by pulmonary vascular remodeling, increased pulmonary vascular resistance, and right ventricular dysfunction. The endothelin pathway plays a central role in its pathophysiology and is targeted by endothelin receptor antagonists (ERAs), including ambrisentan and bosentan. Ambrisentan is a selective ETA receptor antagonist, whereas bosentan blocks both ETA and ETB receptors. Although transitions between ERAs occur in clinical practice, evidence regarding the clinical impact of switching from ambrisentan to bosentan remains limited. ACTION is a retrospective, observational, single-center cohort study evaluating adult patients with pulmonary arterial hypertension (World Health Organization Group 1) and/or chronic thromboembolic pulmonary hypertension (World Health Organization Group 4) confirmed by right heart catheterization. Patients who switched from ambrisentan to bosentan because of a national ambrisentan shortage will be compared with clinically similar patients who remained on ambrisentan. Clinical, functional, and laboratory data recorded at baseline and at 3 to 6 months of follow-up will be assessed. The primary outcome is the proportion of patients with worsening risk stratification after switching from ambrisentan to bosentan compared with patients who continued ambrisentan. Risk will be evaluated using the COMPERA 2.0 and REVEAL Lite 2 assessment tools. Secondary outcomes include changes in World Health Organization/New York Heart Association functional class, 6-minute walk distance, BNP levels, individual risk-assessment components, hepatic enzymes, hemoglobin levels, and clinically relevant events such as hospitalization, emergency department visits, initiation of supplemental oxygen, and right heart failure decompensation.

Vasopressor Agents in Pulmonary Hypertension Crisis

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

Impact of Sotatercept on Pulmonary Artery and Right Ventricle Remodeling Imaging Assessed With 68Ga-FAPI PET/CT in Patients With PAH

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.

Open-Label Study of IKT-001 in Pulmonary Arterial Hypertension (PAH)

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.

COMMODITIES Trial: Initial Dual Oral Therapy vs Monotherapy in PAH With Cardiovascular Comorbidities

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.

Development and Validation of a Chinese Quality of Life Scale for Pulmonary Arterial Hypertension

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.

A Study Evaluating the Safety and Tolerability of Artesunate in Patients With Pulmonary Arterial Hypertension

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.