Clinical Research Directory

A Study Evaluating the Long-Term Efficacy and Safety of Ralinepag in Subjects With PAH Via an Open-Label Extension

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.

TSPO Endothelial - EMPATHY

The aim of the study is to investigate whether the cells which line the blood vessels (called "endothelial cells") are involved in the processes which contribute to Pulmonary Arterial Hypertension (PAH). PAH is a rare condition in which a narrowing of blood vessels carrying blood through the lungs puts an increased workload on the heart; it has to work harder to pump blood through the lungs. Previous studies have shown that these "endothelial cells" may be involved in causing this narrowing. We have shown that a particular protein (called TSPO) which is present on these cells, might be involved in this process. The purpose of the study is to use a drug (called XBD173) which targets TSPO, in order to learn how it affects endothelial cells and the blood vessels. Patients will be invited to participate by their local pulmonary hypertension hospital. Participants will be treated with XBD173 (180mg daily, in three divided doses of 60mg). The total period of treatment for each patient is 6 weeks, followed by 4 weeks follow up off XBD173. If a patient does not tolerate the XBD173 the local PI will determine whether to stop XBD173, reduce the dose to 60mg OD or BD, or pause dosing and restart at 60mg OD or BD. Participants will undergo an echocardiogram and an 18-FDG PET scan of the heart and lung before and after the intervention period. Doses of 180mg per day (in two doses of 90mg) have been well tolerated in patients with cerebral small vessel disease in another ongoing study (IRAS 339664). The proposed study will recruit patients with PAH attending specialist clinics that have an approved implanted cardiopulmonary monitor (CardioMEMSTM Heart Failure system) that permits remote sensing. The monitor will have been implanted on clinical grounds. Patients with stable readings for at least a month will be considered for recruitment. These stable readings provide a good baseline from which to measure the effect of the study drug and relate to changes in blood biomarkers. Remote sensing offers greater oversight of the patient and closer safety monitoring. Remotely acquired patient data are reviewed twice a week via a central clinical team based in Sheffield. Relevant data will be shared as de-identifiable information, and sent to Imperial College London (upon request) as pseudonymised (coded) data if there are any concerns regarding patients' safety. Follow-up would be scheduled by telephone or video conferencing with hospital visits being optional. The most common side effects reported with XBD173 include nervous system and gastrointestinal symptoms, but the frequencies of these side effects are no higher than placebo. The end of the study will be the last visit of the last subject.

Impact of an Optimized Omega-3 Formulation on Inflammation and Endothelial Dysfunction in Pulmonary Arterial Hypertension

The objective of this research project is to evaluate the biological effects of long-chain omega-3 supplementation, administered in an optimized, high-purity formulation (EPA:DHA 6:1, \>95% v/v), as an adjunct to standard-of-care treatment in patients with pulmonary arterial hypertension (PAH). The expected results are confirmation in humans of our preliminary data, namely a beneficial effect on systemic inflammation and pulmonary endothelial dysfunction in PAH. If our hypothesis is confirmed, omega-3s could constitute a complementary nutritional approach to current PAH therapies, subsequently requiring validation through a larger-scale, randomized, controlled study.

A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients

Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health Organization (WHO) Group 1 PAH.

Sota-ES - Sotatercept in Patients With Congenital Heart Disease and Eisenmenger´s Syndrome

The present study seeks to provide pilot data on the safety and efficacy of medical therapy with sotatercept in patients with an established diagnosis of congenital heart disease and Eisenmenger syndrome. CHASE is an interventional, single-arm, open-label study, that will enroll 40 patients with an established diagnosis of CHD and Eisenmenger syndrome. PAH background therapy may be present at the discretion of the investigators at the time of enrolment. CHASE will be performed only in countries where standard PAH therapies are available and reimbursed. At the end of the 24-week patient period, PAH treatment is left to the investigator's discretion.

Effects of Pulmonary Rehabilitation on Quality of Life and Health in Pulmonary Arterial Hypertension Patients

Pulmonary hypertension (PH) is a major global health concern, affecting approximately 1% of the world's population. With global aging and increased life expectancy, its incidence continues to rise. PH is a progressive and disabling disease, with studies showing its progression correlates with worsening symptoms and increased mortality. Even with targeted medications, the prognosis remains poor across PH subtypes, with PAH patients showing only a 49% 7-year survival rate. The 2022 ESC/ERS guidelines emphasize that PH management requires a comprehensive, multidisciplinary approach. Beyond pharmacological and surgical treatments, rehabilitation has demonstrated benefits in improving exercise capacity, quality of life, functional class, and peak oxygen consumption. However, research on specific and effective comprehensive pulmonary rehabilitation programs remains lacking.