Pulmonary arterial hypertension (PAH) is a rare and serious condition where the small arteries in the lungs become narrowed and stiff, dramatically raising the pressure the right heart must work against. Without treatment, right heart failure typically develops within years of diagnosis.
What's actually going on in research
The approval of sotatercept — which targets aberrant cell proliferation in pulmonary arteries — represents the most significant advance in PAH in years, and combination trials are exploring it alongside all three established drug classes. Early intervention with multiple drugs from the time of diagnosis is being tested against sequential addition to see if aggressive upfront treatment halts vascular remodeling. Gene therapy and RNA interference drugs targeting specific genetic mutations in heritable PAH are in early-phase trials.
Sotatercept combinations
Sotatercept restores the balance between growth-promoting and growth-inhibiting signals in pulmonary artery walls. Trials are testing it as initial combination therapy alongside existing PAH drug classes.
Upfront triple therapy
Starting all three drug classes — endothelin antagonists, PDE-5 inhibitors, and prostacyclins — simultaneously at diagnosis is being compared with sequential addition in newly diagnosed PAH.
RNA interference therapy
Drugs targeting BMPR2 mutations and other genetic causes of heritable PAH using RNA interference are in early trials with the goal of correcting the underlying molecular defect.
What to know before you search
Eligibility requires confirmed PAH with right heart catheterization, WHO functional class, prior treatment status, and sometimes genetic testing for heritable forms.
What types of trials are currently open
- Drug combination trials — Testing new combinations of PAH medications including sotatercept with existing drug classes.
- Newly diagnosed PAH trials — Comparing upfront combination strategies versus sequential escalation in treatment-naive patients.
- Gene and RNA therapy trials — Evaluating genetic correction approaches for heritable PAH with BMPR2 or other mutations.
- Exercise and rehabilitation trials — Testing supervised exercise training and its effects on function and right heart remodeling in PAH.
- Biomarker trials — Validating BNP, troponin, and imaging markers to guide treatment escalation decisions.
Recently added Pulmonary Arterial Hypertension trials
Prevalence, Pathogenesis, and Prognosis of Pulmonary Hypertension in Dialysis Patients
This prospective multicenter longitudinal study aims to investigate the prevalence, pathogenesis, and prognosis of pulmonary hypertension in patients with end-stage renal disease undergoing chronic hemodialysis. The research seeks to identify specific clinical factors and biomarkers like angiopoietin-2 that contribute to the development of this condition, while evaluating the prognostic value of right ventricular function measured via TAPSE. Participants undergo a standardized screening echocardiogram the day after their intermediate dialysis session to determine the probability of pulmonary hypertension. Those identified as high-risk receive further diagnostic confirmation through right heart catheterization, respiratory function tests, and lung scans to clarify the underlying etiology. The protocol also evaluates the hemodynamic impact of high-flow arteriovenous fistulas and volume overload on pulmonary pressures. Clinical follow-up is conducted at baseline and subsequently at 6, 12, and 24 months to monitor patient outcomes and standardize therapeutic management according to established European guidelines.
Mechanistic Study of Nicotinamide Riboside on NAD+ Biology in Individuals With Combined Pulmonary Hypertension
Pulmonary hypertension (PH) is a serious condition that puts strain on the heart and lungs and often leads to frequent hospital stays and shortened life expectancy. The most common cause is heart disease affecting the left side of the heart. A particularly high-risk form, called combined pre- and post-capillary pulmonary hypertension (CPH), occurs in about one in four people with heart failure. There are currently no approved treatments for CPH, and many patients develop right-sided heart failure and die earlier than expected. This study is based on a new approach that uses advanced computer methods to analyze a patient's unique biology and identify potential drug targets. Using this method, we identified nicotinamide riboside (NR) as a promising option for people with CPH. NR is a form of vitamin B3 that helps the body make NAD⁺, a substance essential for how cells produce energy and stay healthy. NAD⁺ plays an important role in how heart and blood vessel cells function. Previous research in animals suggests NR may help improve blood vessel changes in the lungs and support heart function. NR has also shown potential benefits in human studies related to cell energy, mitochondrial health, and reducing oxidative stress. In this study, NR is used only as a dietary supplement that supports normal body processes, not as a proven treatment. The investigators will conduct a small, carefully controlled study in which participants receive NR and a placebo at different times. The goal is to understand how NR affects biological and biochemical markers in the body, not to test whether it improves symptoms or outcomes. Any clinical measurements are included only to help interpret the biological effects.
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