Pulmonary Arterial Hypertension (PAH) Clinical Trials

Pulmonary arterial hypertension (PAH) is a rare progressive disease of the pulmonary vasculature characterized by increased pulmonary vascular resistance and elevated right ventricular afterload, leading to right heart failure and death if untreated. PAH involves complex pathophysiology including endothelial dysfunction, vasoconstriction (abnormal nitric oxide and prostacyclin signaling), thrombosis, and vascular remodeling with progressive reduction of cross-sectional area available for blood flow. Idiopathic PAH (IPAH) has no identifiable cause but involves genetic predisposition (BMPR2, ALK1, ENG mutations).

The most common symptoms of Pulmonary Arterial Hypertension include Shortness of breath, especially with exertion, Chest pain or pressure, Syncope (fainting) or near-syncope, Fatigue and weakness, Swelling in legs and abdomen, among others. About 26 to 52 per million people in a Scottish national study, with about 7 new cases per million people each year (Peacock et al., European Respiratory Journal, 2007). Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Pulmonary Arterial Hypertension from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. The treatment approach for pulmonary arterial hypertension has shifted significantly toward early, aggressive combination therapy targeting multiple pathways at once, and current trials are building on that momentum. Sotatercept (Winrevair), an activin signaling inhibitor given as an injection under the skin every 3 weeks, was FDA approved in March 2024 for adults with PAH and works on a different pathway from the established endothelin, nitric oxide, and prostacyclin drugs.

Yes. As of October 21, 2026, 13 FDA-approved medicines have a current label that names Pulmonary Arterial Hypertension: Adcirca (tadalafil), first approved in 2009; Adempas (riociguat), first approved in 2013; Flolan (epoprostenol), first approved in 1995; Letairis (ambrisentan), first approved in 2007; Opsumit (macitentan), first approved in 2013; Revatio (sildenafil), first approved in 2005; Tracleer (bosentan), first approved in 2001; Tyvaso (treprostinil), first approved in 2002; Uptravi (selexipag), first approved in 2015; Ventavis (iloprost), first approved in 2004; Winrevair (sotatercept), first approved in 2024; Yutrepia (treprostinil inhalation powder), first approved in 2025; and Opsynvi (macitentan and tadalafil). Corticosteroids and immune globulins count once each. The FDA is due to decide on Ralinepag by June 24, 2027; the FDA calendar page carries the outcome the day it lands.

Pulmonary Arterial Hypertension is caused by variants in the BMPR2, ACVRL1, ENG, EIF2AK4, CAV1 and KCNK3 genes. Most PAH is sporadic and idiopathic; BMPR2 is by far the most common cause of heritable PAH, and about 20% of idiopathic cases carry a variant in a known gene.

It follows autosomal dominant with reduced penetrance inheritance. About 26 to 52 per million people in a Scottish national study, with about 7 new cases per million people each year (Peacock et al., European Respiratory Journal, 2007). Genetic testing confirms the diagnosis.

Yes. Genetic testing for Pulmonary Arterial Hypertension looks at the BMPR2, ACVRL1, ENG, EIF2AK4, CAV1 and KCNK3 genes. Most PAH is sporadic and idiopathic; BMPR2 is by far the most common cause of heritable PAH, and about 20% of idiopathic cases carry a variant in a known gene.

It follows autosomal dominant with reduced penetrance inheritance.

A confirmed variant establishes the diagnosis and is often required to enroll in gene-specific trials. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Pulmonary Arterial Hypertension clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Pulmonary Arterial Hypertension clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.