Pulmonary arterial hypertension is a rare disease where blood pressure in the lung arteries rises dangerously high, straining the heart's right side. About 500 to 1,000 people in the US are diagnosed each year. Current treatments include prostacyclin pathway drugs, endothelin receptor antagonists, and phosphodiesterase-5 inhibitors, often used in combination to keep symptoms manageable.
What's actually going on in research
Trials are testing sotatercept, a drug that targets abnormal cell growth in artery walls and was FDA-approved in 2024. Researchers are studying ways to reverse artery damage rather than just dilate vessels, testing stem cell therapies, and looking at earlier intervention before the heart weakens. Gene therapy and drugs targeting inflammation pathways are also in development.
Sotatercept and related drugs
Sotatercept works differently from older PAH drugs by slowing the thickening of artery walls. Trials are testing it alongside standard treatments to see if it can improve exercise capacity and delay disease worsening.
Reversing artery damage
Several drugs aim to undo the structural changes in lung arteries, not just open them wider. If successful, they could change how far the disease progresses rather than just managing symptoms.
Earlier treatment
Studies are testing whether starting treatment sooner, when symptoms are milder, prevents permanent heart damage. Researchers are also refining biomarkers to catch the disease earlier.
What to know before you search
Eligibility typically depends on PAH type, severity measured by walk distance and heart function, current medications, and whether other lung or heart conditions are present.
What types of trials are currently open
- Add-on treatment trials — Testing new drugs added to standard PAH medications to see if they further improve exercise tolerance and slow disease progression.
- Monotherapy trials — Testing whether a single new drug works well enough on its own, particularly in people newly diagnosed with milder disease.
- Combination strategy trials — Comparing different combinations of PAH drugs to find which work best together and when to add them.
- Stem cell trials — Studies testing whether stem cells can repair damaged lung arteries or help the heart adapt to high pressure.
- Device and surgical trials — Testing devices that reduce pressure between heart chambers or comparing lung transplant approaches.
Recently added Pulmonary Arterial Hypertension trials
Take an extra dose of blood pressure medication before exercise
The goal of this clinical study is to investigate whether the additional intake of sildenafil 20mg on top of standard medical treatment results in a benefit on maximum exercise workload (Wmax) in patients with pulmonary arterial hypertension (PAH).
Share your quality of life experiences through a brief questionnaire
Pulmonary hypertension (PH) is a severe and heterogeneous condition associated with poor prognosis and significant impairment in quality of life. Patient-reported outcome questionnaires are useful, low-cost tools for clinical assessment and risk stratification. The EmPHasis-10 questionnaire is a brief and easy-to-administer instrument that has demonstrated good correlation with clinical outcomes across different PH populations; however, a validated Brazilian Portuguese version is not yet available. This study aims to translate, culturally adapt, and validate the EmPHasis-10 questionnaire for Brazilian patients with pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH). The investigators designed a cross-sectional observational study including 50 adult patients with a confirmed diagnosis of PAH (group 1) or CTEPH (group 4), followed at the Pulmonary Circulation Outpatient Clinic of Hospital de Clínicas de Porto Alegre. The EmPHasis-10-BR will be administered alongside the SF-36 questionnaire for concurrent validity analysis, as well as frailty assessment instruments. Reliability will be evaluated through test-retest analysis in both in-person and remote settings. Clinical, functional, and hemodynamic variables will also be collected to investigate associations between EmPHasis-10-BR scores and risk stratification parameters recommended by international guidelines. Statistical analyses will include Cronbach's alpha to assess internal consistency, Spearman's correlation coefficients for test-retest reliability, and comparisons with previously validated instruments. The investigators expect that the validation of the EmPHasis-10-BR will provide a practical, reliable, and culturally appropriate tool for quality-of-life assessment in Brazilian patients with PH, supporting standardized clinical follow-up, risk stratification, and potentially contributing to therapeutic decision-making.
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