Ongoing Clinical Trials for Pulmonary Arterial Hypertension

This article provides information about 29 ongoing clinical trials for pulmonary arterial hypertension, a rare condition where blood pressure in the lung arteries becomes abnormally high, making it harder for the heart to pump blood through the lungs. The trials are testing various medications including new treatments and existing drugs being studied for this condition, taking place across multiple European countries.

Clinical trial locations

• Austria
  • Study on the Safety and Tolerability of Treprostinil Sodium for Children with Pulmonary Arterial Hypertension
  • Study on the Effectiveness and Safety of Treprostinil and Drug Combination for Patients with Pulmonary Arterial Hypertension (Group I)
  • Study on the Safety and Effects of Riociguat for Children Aged 6 to 17 with Pulmonary Arterial Hypertension
  • Study on Treprostinil Palmitil Inhalation Powder for Patients with Pulmonary Arterial Hypertension
  • Long-Term Safety Study of Inhaled Imatinib for Patients with Pulmonary Arterial Hypertension
  • Study Comparing the Effectiveness and Safety of Macitentan 75 mg and 10 mg for Patients with Pulmonary Arterial Hypertension
  • Study of AZD3427 for Patients with Heart Failure and Pulmonary Hypertension from Left Heart Disease
  • Long-term Study of Sotatercept for Adults with Pulmonary Arterial Hypertension (PAH)
  • Study on Long-Term Safety and Effectiveness of Treprostinil Palmitil for Patients with Pulmonary Arterial Hypertension
  • Study on the Safety and Effectiveness of Inhaled Imatinib (AV-101) for Patients with Pulmonary Arterial Hypertension (PAH)
• Belgium
  • A study of LTP001 in combination with standard treatment for adults with pulmonary arterial hypertension
  • Study on the Safety and Effects of PF-07868489 in Patients with Pulmonary Arterial Hypertension
  • Long-Term Safety Study of Inhaled Imatinib for Patients with Pulmonary Arterial Hypertension
  • Study Comparing the Effectiveness and Safety of Macitentan 75 mg and 10 mg for Patients with Pulmonary Arterial Hypertension
  • Long-term Study of Sotatercept for Adults with Pulmonary Arterial Hypertension (PAH)
  • Study on Long-Term Safety and Effectiveness of Treprostinil Palmitil for Patients with Pulmonary Arterial Hypertension
  • Study on the Safety and Effectiveness of Inhaled Imatinib (AV-101) for Patients with Pulmonary Arterial Hypertension (PAH)
  • Study on Selexipag and Bosentan for Children with Pulmonary Arterial Hypertension
  • Study on Seralutinib for Treating Pulmonary Arterial Hypertension in Patients
  • Study on the Effects of MK-5475 for Adults with Pulmonary Arterial Hypertension
• Bulgaria
  • Study Comparing the Effectiveness and Safety of Macitentan 75 mg and 10 mg for Patients with Pulmonary Arterial Hypertension
  • Study on Selexipag and Bosentan for Children with Pulmonary Arterial Hypertension
• Croatia
  • Long-term Study of Sotatercept for Adults with Pulmonary Arterial Hypertension (PAH)
  • Study of Sotatercept for Newly Diagnosed Intermediate- and High-risk Pulmonary Arterial Hypertension Patients
• Czechia
  • A study of LTP001 in combination with standard treatment for adults with pulmonary arterial hypertension
  • Study on Inhaled Seralutinib (GB002) for Patients with Pulmonary Arterial Hypertension (PAH)
  • Study on Long-term Safety of Seralutinib for Patients with Pulmonary Arterial Hypertension
  • Study on the Safety and Effects of PF-07868489 in Patients with Pulmonary Arterial Hypertension
  • Study on the Safety and Tolerability of Sotatercept for Patients with Pulmonary Arterial Hypertension
  • Study on the Effectiveness and Safety of Treprostinil and Drug Combination for Patients with Pulmonary Arterial Hypertension (Group I)
  • Long-Term Safety Study of Inhaled Imatinib for Patients with Pulmonary Arterial Hypertension
  • Study Comparing the Effectiveness and Safety of Macitentan 75 mg and 10 mg for Patients with Pulmonary Arterial Hypertension

A study of LTP001 in combination with standard treatment for adults with pulmonary arterial hypertension

This trial investigates LTP001, a new oral medication taken in capsule form, for adults with pulmonary arterial hypertension. The study aims to determine if LTP001 can improve patient outcomes when added to standard treatments.

Who can participate: Adults aged 18 or older with confirmed PAH who can walk between 150 and 450 meters in a 6-minute walking test. Participants must be on stable doses of current PAH medications and have a pulmonary vascular resistance of at least 400 dyn.sec.cm.

Who cannot participate: Pregnant or breastfeeding women, people with severe liver or kidney problems, those who have had a heart attack or stroke in the last 6 months, individuals with uncontrolled high blood pressure or active bleeding problems, those taking certain interacting medications, people with serious infections or severe psychiatric conditions, and those with a history of substance abuse within the past year.

Treatment approach: Participants receive either LTP001 capsules or placebo capsules to take orally, alongside their regular PAH medications. The study monitors changes in blood vessel resistance in the lungs, walking ability, and heart function over an initial 24-week period, followed by an extended period of up to 18 months.

Investigational drug: LTP001 is being studied for safety and effectiveness when combined with standard PAH treatments in patients with moderate to severe symptoms.

Study on Dapagliflozin for Patients with Pulmonary Arterial Hypertension to Improve Exercise Capacity and Heart Function

This Danish trial evaluates whether dapagliflozin, a medication originally developed for diabetes, can improve exercise capacity and heart function in PAH patients when added to standard treatments.

Who can participate: Adults aged 18 or older with confirmed idiopathic, heritable, or connective tissue disease-associated PAH in WHO functional class II-III. Participants must be on stable pulmonary vasodilator treatment for at least four weeks and able to perform a cardiopulmonary exercise test. Women of childbearing potential must use reliable birth control and have negative pregnancy tests.

Who cannot participate: Pregnant or breastfeeding women, people with recent heart attacks or strokes (within 6 months), those with severe liver or kidney disease, individuals currently in other trials, people with drug or alcohol abuse history, those allergic to the study medication, individuals with uncontrolled blood pressure, and those with recent cancer diagnoses (within 5 years, except certain skin cancers).

Treatment approach: Participants randomly receive either dapagliflozin 10 mg tablets or placebo tablets daily for 90 days, continuing their regular PAH medications. The study measures maximum oxygen consumption during exercise, walking distance, heart and lung function, and various health indicators throughout the trial.

Investigational drug: Dapagliflozin is being studied for its potential to improve heart and lung function in PAH patients by helping the body remove excess sugar through urine, which may reduce heart workload and improve blood flow.

Study on Inhaled Seralutinib (GB002) for Patients with Pulmonary Arterial Hypertension (PAH)

This trial studies the long-term safety and effectiveness of seralutinib, delivered through a dry powder inhaler, for PAH patients who completed a previous seralutinib study.

Who can participate: Adults aged 18-80 (women) or 50-80 (men) who completed a previous seralutinib PAH study without early discontinuation, following all study requirements. Participants must be on stable doses of standard PAH treatments. Women of childbearing potential need negative pregnancy tests and must use highly effective birth control. Men must use condoms and not donate sperm during the study and for 90 days after.

Who cannot participate: Patients with pulmonary hypertension types other than PAH, those outside the specified age ranges, individuals unable to use inhaled medication, those with other serious interfering health conditions, pregnant or breastfeeding women, participants in other trials, those who recently had or plan surgery, people with drug or alcohol abuse history, and those allergic to the study medication or its ingredients.

Treatment approach: Participants receive seralutinib as an inhalation powder through a dry powder inhaler, with dosage determined by weight. The study continues until April 2027, with regular monitoring for adverse events and assessments of exercise capacity and overall health status.

Investigational drug: Seralutinib (GB002) is being studied for long-term safety and effectiveness in treating PAH, administered through oral inhalation to reach the lungs directly.

Study on Long-term Safety of Seralutinib for Patients with Pulmonary Arterial Hypertension

This extension study evaluates the long-term safety and tolerability of seralutinib, taken as an inhalation powder in hard capsules, for PAH patients who completed a prior seralutinib trial.

Who can participate: Participants must have completed a previous seralutinib PAH study. Women of childbearing potential must have negative pregnancy tests before starting, agree to ongoing testing, use highly effective birth control during the study and for 30 days after, and not donate eggs. Women of non-childbearing potential include those post-menopausal or surgically sterile. Male participants must use condoms when sexually active with women of childbearing potential and not donate sperm during the study and for 90 days after. All participants must provide informed consent and be able to attend visits and follow study procedures.

Who cannot participate: The exclusion criteria were not specifically detailed in the source data beyond general requirements for study compliance and safety.

Treatment approach: Participants receive seralutinib as an inhalation powder in hard capsule form through oral inhalation. The study runs until October 31, 2028, monitoring for adverse events and measuring changes in 6-minute walk distance, NT-proBNP levels, and other health parameters throughout the treatment period.

Investigational drug: Seralutinib is being studied for long-term safety and tolerability in PAH patients, administered through oral inhalation as a way to deliver medication directly to the lungs.

Study on Sotatercept for Children with Pulmonary Arterial Hypertension Receiving Standard Care

This trial investigates sotatercept (MK-7962), given as a solution injected under the skin, for children aged 1 to less than 18 years with PAH who are already receiving standard care.

Who can participate: Children aged 1 to less than 18 years with documented PAH diagnosis confirmed by right heart catheterization at any time before screening. Participants must be on stable doses of background PAH therapy including medications like PDE5 inhibitors, endothelin receptor antagonists, soluble guanylate cyclase stimulators, or prostanoids. Males must agree to abstain from heterosexual intercourse or use contraception during the study and for 16 weeks after the last dose. Females must either be of non-childbearing potential or use highly effective contraception or abstain during the study and for 16 weeks after. Males must also agree not to donate blood or sperm, and females not to donate blood, eggs, or ovum during this period.

Who cannot participate: Patients with other serious interfering health conditions, pregnant or breastfeeding individuals, recent trial participants, those with allergic reactions to similar medications, people unable to follow study procedures, those with certain heart conditions, individuals with severe liver or kidney problems, those taking interacting medications, people with drug or alcohol abuse history, individuals with uncontrolled high blood pressure, those with certain infections, recent surgery patients or those planning surgery, people with recent cancer (unless in remission for a certain period), those with certain blood disorders, individuals with certain autoimmune diseases.

Treatment approach: Participants receive sotatercept through subcutaneous injection over 24 weeks, with regular monitoring including blood tests, blood pressure checks, and heart function assessments. The study evaluates safety, tolerability, and how the body processes the medication, along with changes in physical performance, heart function, and quality of life measures.

Investigational drug: Sotatercept (MK-7962) is being studied to assess its safety and tolerability in children with PAH when used alongside existing treatments over a 24-week period.

Study on Sotatercept for Improving Heart Function in Patients with Pulmonary Arterial Hypertension

This Netherlands-based trial studies how sotatercept, given as a subcutaneous injection, affects right heart function in PAH patients over 24 weeks.

Who can participate: Adults aged 18-70 with idiopathic or hereditary PAH in WHO functional class II-IV. Participants must have hemodynamic PAH diagnosis confirmed by right heart catheterization showing mean pulmonary artery pressure greater than 20 mmHg, pulmonary capillary wedge pressure or left ventricular end diastolic pressure of 15 mmHg or less, and pulmonary vascular resistance of at least 4 Wood units. Stable background therapy for at least 3 months before screening is required, along with NTproBNP levels greater than 300 ng/L. Written informed consent is mandatory.

Who cannot participate: Patients with other serious interfering conditions, those who had heart attacks or strokes in the past 6 months, individuals with uncontrolled high blood pressure, those with severe liver or kidney disease, pregnant or breastfeeding women, current participants in other trials, organ transplant recipients, people with drug or alcohol abuse in the past year, and those with known allergies to the study medication.

Treatment approach: Participants receive sotatercept as a subcutaneous injection (60 mg of Winrevair powder mixed into solution) throughout a 24-week treatment period. The study monitors right ventricular function and the connection between the heart and pulmonary arteries, using regular heart imaging, blood flow measurements, and exercise capacity assessments.

Investigational drug: Sotatercept is being studied for its potential to improve right heart function by targeting specific proteins involved in blood vessel growth and repair, aiming to enhance the heart’s efficiency and its connection with lung blood vessels.

Study on the Effectiveness and Safety of Treprostinil and Drug Combination for Patients with Pulmonary Arterial Hypertension (Group I)

This trial compares initial triple therapy (including parenteral treprostinil with two oral medications) versus initial double oral therapy in treatment-naive PAH patients.

Who can participate: Adults aged 18-70 with confirmed idiopathic, heritable, drug/toxin-induced PAH, or PAH associated with connective tissue disease or corrected congenital heart disease. Participants must not have received prior PAH treatment. Required measurements include pre-randomization pulmonary vascular resistance of at least 400 dyn.sec.cm, 6-minute walk distance between 150-450 meters, and intermediate-high risk status or intermediate-low risk with severe heart/lung function issues (mean right atrial pressure of 20 mmHg or more, cardiac index less than 2.0 liters per minute, stroke volume index less than 31 milliliters, pulmonary vascular resistance of 12 Wood Units or more). Right heart catheterization must show mean pulmonary arterial pressure greater than 20 mmHg, pulmonary capillary wedge pressure of 15 mmHg or less, and pulmonary vascular resistance greater than 2 Wood Units. Women of childbearing potential must have negative pregnancy tests and use reliable birth control.

Who cannot participate: Patients without PAH diagnosis, those outside the specified age range, vulnerable populations needing special protection, individuals unable to take study medications, those with other interfering medical conditions, people unable to follow study procedures or attend visits, pregnant or breastfeeding women, recent trial participants, those with drug or alcohol abuse history.

Treatment approach: Participants are randomly assigned to receive either triple therapy (oral ERA and PDE-5i plus parenteral treprostinil infusion) or double therapy (oral ERA and PDE-5i only) for up to 48 weeks. The study monitors achievement of low-risk status, changes in walking ability, blood vessel resistance, heart function, and safety throughout the trial period.

Investigational drug: Treprostinil is administered through continuous infusion as part of triple therapy, working by widening blood vessels in the lungs to lower blood pressure and improve symptoms. The study also includes standard oral PAH medications (ERA and PDE-5i).

Study on the Safety and Effects of PF-07868489 in Patients with Pulmonary Arterial Hypertension

This trial evaluates PF-07868489, administered as subcutaneous injections, for safety and effects on blood markers related to heart stress in PAH patients.

Who can participate: Adults at least 18 years old with confirmed idiopathic, heritable, drug-induced, or connective tissue disease-associated PAH in WHO functional class II or III. Participants must have PAH confirmed by right heart catheterization with pulmonary vascular resistance of at least 400 dyn·sec/cm5 (5 Wood units). Pulmonary function tests are required unless the participant is a smoker with significant smoking history. Documentation must confirm that chronic thromboembolic pulmonary hypertension has been excluded. Participants must be able to walk between 150-500 meters in the 6-minute walk test with consistent results. Stable treatment with at least two standard PAH medications for at least 60 days before the study is required. Body mass index must be between 16-35 kg/m2. Signed informed consent and ability to follow study requirements are mandatory.

Who cannot participate: Patients with other serious interfering conditions, pregnant or breastfeeding women, those with recent heart attacks or strokes, individuals with severe allergic reaction history to medications, current participants in other trials, people with drug or alcohol abuse history, those with uncontrolled high blood pressure, individuals with severe liver or kidney disease, people with cancer diagnoses in the past five years, those with lung diseases other than PAH.

Treatment approach: Participants receive repeat doses of PF-07868489 or placebo through subcutaneous injections while continuing their standard PAH medications. The study monitors safety, tolerability, and changes in NTproBNP blood markers and 6-minute walk distance over the treatment period, with regular health assessments and blood tests.

Investigational drug: PF-07868489 is being studied for its potential to help manage PAH by influencing biological pathways that reduce heart strain and improve lung blood flow, aiming to improve blood marker levels indicating heart stress.

Study on the Safety and Tolerability of Sotatercept for Patients with Pulmonary Arterial Hypertension

This extension study evaluates the long-term safety and tolerability of sotatercept in PAH patients who completed the previous MK-7962-024 (LIGHTRAY) study, using a weight-banded dosing approach.

Who can participate: Patients who completed the treatment period of the MK-7962-024 (LIGHTRAY) study, including Visit 11, without discontinuing study treatment. Participants must not have started commercially available sotatercept treatment. All genders are eligible, including adults and older adults within the study’s specified age range.

Who cannot participate: Patients with other serious interfering conditions, pregnant or breastfeeding women, those with recent heart attacks or strokes, individuals with uncontrolled high blood pressure, people who had major surgery within the last 3 months, those with severe allergic reaction history to similar medications, current participants in other trials, individuals with severe liver or kidney disease, people with drug or alcohol abuse within the last year, those unable to comply with study procedures.

Treatment approach: Participants receive sotatercept through subcutaneous injections using a weight-banded dosing approach throughout the extension period. The study, expected to run until September 30, 2027, monitors adverse events and assesses the treatment’s long-term safety profile with regular health evaluations.

Investigational drug: Sotatercept (MK-7962) is being studied for long-term safety and tolerability when used alongside standard PAH treatments, with dosing adjusted based on patient weight.

Study on the Safety and Tolerability of Treprostinil Sodium for Children with Pulmonary Arterial Hypertension

This trial studies the safety and tolerability of preservative-free treprostinil sodium formulations in children under 18 years with severe PAH requiring prostacyclin infusion treatment.

Who can participate: Children from birth to under 18 years at consent signing with confirmed severe PAH diagnosis requiring prostacyclin infusion treatment. Current PAH diagnosis must be confirmed by right heart catheterization or echocardiography (if catheterization not possible) showing mean pulmonary arterial pressure greater than 20 mmHg and pulmonary vascular resistance index greater than 3 Wood Units m². Participants may be treatment-naive or previously treated with treprostinil (subcutaneous or intravenous) before the study. Non-childbearing potential or, if of childbearing potential, negative pregnancy test, not breastfeeding, and agreement to use two reliable birth control methods if sexually active until study end and 30 days after last dose. Signed informed consent by parents/legal representatives and child assent if developmentally capable.

Who cannot participate: Patients without PAH diagnosis, those 18 years or older, individuals not new to or previously treated with treprostinil, those not part of the vulnerable population requiring special care.

Treatment approach: Participants receive preservative-free treprostinil through subcutaneous or intravenous injection over a 5-month (20 weeks ± 1 week) treatment period. The study monitors adverse events, drug reactions, and changes in quality of life, physical capacity, and heart function through regular assessments including questionnaires, physical tests, and blood tests.

Investigational drug: Treprostinil sodium (various preservative-free formulations: TREPaed_0.5, TREPaed_1, TREPaed_2.5, TREPaed_5, TREPaed_10) is being studied for safety and tolerability in children with PAH, helping to relax and widen lung blood vessels.

Study comparing tadalafil with ambrisentan versus tadalafil alone in newly diagnosed patients with pulmonary arterial hypertension who have cardiovascular conditions

This French trial compares combination therapy (ambrisentan plus tadalafil) versus single therapy (tadalafil plus placebo) in newly diagnosed PAH patients who also have cardiovascular conditions.

Who can participate: Adults 18 years or older with PAH diagnosed within the last 6 months, confirmed by right heart catheterization showing mean pulmonary artery pressure of 25 mmHg or higher, pulmonary artery wedge pressure of 15 mmHg or less, and pulmonary vascular resistance of 3 Wood Units or higher. Participants must be treatment-naive for PAH. PAH must be inherited, without known cause, drug-related, connected to tissue disease, associated with HIV infection, or related to corrected heart defect (fixed more than 1 year ago). Participants must have at least two cardiovascular conditions: high blood pressure, diabetes (any type), obesity (BMI of 30 or higher), or heart disease (such as previous heart attack, heart procedures, blocked arteries, or stable chest pain).

Who cannot participate: Patients below 18 or above 75 years, those with previous PAH treatment, pregnant or breastfeeding women, individuals with severe liver or kidney disease (requiring dialysis for kidney disease), those with heart attack or stroke in the past 6 months, people with uncontrolled high blood pressure, individuals with active bleeding disorders, those using nitrate medications for chest pain, people with known allergic reactions to tadalafil or ambrisentan, participants in other trials within the past 30 days, those with life-threatening conditions requiring immediate treatment, people unable to provide informed consent, individuals with substance abuse or addiction interfering with participation, those with mental health conditions affecting compliance.

Treatment approach: Participants receive either combination therapy (ambrisentan 5 mg plus tadalafil daily) or monotherapy (tadalafil plus placebo daily) for approximately 175 days. The study monitors walking ability through 6-minute walk tests, blood markers for heart function, heart and lung function tests, quality of life measures, blood pressure, heart rate, and weight throughout the treatment period until the study’s expected completion in October 2028.

Investigational drugs: Tadalafil helps relax blood vessels and improve lung blood flow as a PDE5 inhibitor. Ambrisentan works by blocking substances that cause blood vessel narrowing, helping widen lung blood vessels to improve exercise ability and slow symptom worsening.

Summary

These 29 clinical trials represent a diverse research effort across Europe to advance treatment options for pulmonary arterial hypertension. The trials span multiple countries, with particularly strong concentrations in Germany, Italy, Spain, France, Belgium, and Austria, reflecting these nations’ commitment to PAH research.

A notable feature is the variety of investigational approaches being studied. Several trials focus on sotatercept, a newer agent being tested in different patient populations including children and adults at various disease stages. Inhaled therapies represent another significant area of investigation, with multiple trials studying seralutinib and treprostinil palmitil inhalation powder, offering potentially more targeted drug delivery to the lungs.

The trials also demonstrate attention to special populations, with several studies specifically designed for children with PAH, addressing a critical need for pediatric treatment options. Some trials examine repurposing existing medications like dapagliflozin and empagliflozin (originally developed for diabetes) for PAH treatment, representing innovative thinking in drug development.

Many studies are evaluating combination therapies or add-on treatments to standard care, recognizing that PAH often requires multiple medications to manage effectively. Long-term extension studies are also prominent, demonstrating commitment to understanding the sustained safety and effectiveness of these treatments over time.