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Condition Guide

New Treatments & Clinical Trials for Pulmonary Arterial Hypertension

Last updated May 2026Data from ClinicalTrials.gov394 active trials
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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 trialsTesting new combinations of PAH medications including sotatercept with existing drug classes.
  • Newly diagnosed PAH trialsComparing upfront combination strategies versus sequential escalation in treatment-naive patients.
  • Gene and RNA therapy trialsEvaluating genetic correction approaches for heritable PAH with BMPR2 or other mutations.
  • Exercise and rehabilitation trialsTesting supervised exercise training and its effects on function and right heart remodeling in PAH.
  • Biomarker trialsValidating BNP, troponin, and imaging markers to guide treatment escalation decisions.

Recently added Pulmonary Arterial Hypertension trials

RecruitingObservational study

Share medical records from your cancer treatment

This multicenter real-world study assesses the efficacy and safety of adjuvant therapies in postoperative intrahepatic cholangiocarcinoma (ICC) patients with high-risk recurrence factors. 90 eligible patients will be assigned to: Cohort 1: GP (gemcitabine/cisplatin) + adebrelimab Cohort 2: Apatinib + adebrelimab Cohort 3: S-1 (tegafur/gimeracil/oteracil) + adebrelimab Outcomes will be compared against historical real-world controls receiving standard chemotherapy.

Beijing, China
RecruitingPost-approval monitoring

Pre-discharge Influenza Vaccination in Patients Hospitalized for Acute Cardiac Conditions

Patients hospitalized for acute cardiac conditions-including acute myocardial infarction, acute heart failure, pulmonary embolism, arrhythmias, and hypertensive emergencies-represent a heterogeneous population at very high risk of recurrent cardiovascular events. Influenza infection may act as a trigger for adverse cardiovascular events. Given the persistently low influenza vaccination uptake despite evidence-based benefits observed in vulnerable populations, including patients with cardiac conditions, new strategies to improve vaccination coverage are being explored. Recently, increasing attention has been directed toward an approach already used in fields such as neonatology, where vaccinations are administered prior to hospital discharge. In this investigator-initiated, single-center, randomized, open-label interventional study, we will evaluate whether influenza vaccination administered within 24 hours before hospital discharge in patients hospitalized for acute cardiac conditions is safe and effective in reducing subsequent infections, cardiovascular events, and mortality during the 6 months following hospitalization.

Wroclaw, Poland
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