In short
There are currently 2 clinical trials investigating new treatments for heritable pulmonary arterial hypertension, a rare inherited condition causing high blood pressure in the lung arteries. These studies are taking place in Denmark and the Netherlands, testing medications that may help improve heart function and exercise capacity in patients already receiving standard treatments.
Clinical trial locations
- Denmark
- Netherlands
Study on Dapagliflozin for Patients with Pulmonary Arterial Hypertension to Improve Exercise Capacity and Heart Function
This trial is investigating whether dapagliflozin, also known as Forxiga, can help improve exercise capacity and heart and lung function in patients with pulmonary arterial hypertension who are already receiving other treatments. The study will last approximately three months, during which participants will take either the medication or a placebo tablet by mouth once daily.
Who can participate:
- Adults 18 years or older with a diagnosis of pulmonary arterial hypertension, including idiopathic type (no known cause), heritable type (runs in families), or connective tissue disease-associated type
- Patients experiencing slight to moderate limitations in physical activity (WHO functional class II-III)
- Those who have been stable on pulmonary vasodilator treatment for at least four weeks without recent worsening of symptoms or unplanned hospital visits in the last three months
- Women of childbearing age must use reliable birth control and have a negative pregnancy test
- Must be able to understand written information in Danish and provide informed consent
- Must be able to perform a cardiopulmonary exercise test
Who cannot participate:
- Patients with any other serious medical condition that might interfere with the study
- Those who are pregnant or breastfeeding
- Patients who have had a heart attack or stroke in the last 6 months
- Those with severe liver or kidney disease
- Patients currently participating in another clinical trial
- Those with a history of drug or alcohol abuse
- Patients allergic to the study medication or similar drugs
- Those with uncontrolled high blood pressure
- Patients diagnosed with cancer in the last 5 years, except for non-melanoma skin cancer
Study focus: The trial aims to determine whether dapagliflozin can improve the health and quality of life for people with pulmonary arterial hypertension. Researchers will measure changes in exercise ability through cardiopulmonary exercise tests, assess six-minute walking distance, and evaluate heart and lung function throughout the 90-day treatment period.
Investigational drug: Dapagliflozin works by helping the body remove excess sugar through urine, which may also reduce the workload on the heart and improve blood flow. In this study, researchers are exploring how this medication affects exercise capacity and heart function when added to existing treatments.
Study on Sotatercept for Improving Heart Function in Patients with Pulmonary Arterial Hypertension
This open-label clinical trial is testing sotatercept, also known as ACE-011, to see how it affects the function of the right side of the heart in patients with pulmonary arterial hypertension. The medication is given as an injection under the skin over a 24-week period.
Who can participate:
- Adults between 18-70 years of age with a diagnosis of idiopathic (unknown cause) or hereditary (inherited) pulmonary arterial hypertension
- Patients classified as WHO functional class II to IV, which describes the severity of symptoms
- Those with specific blood pressure measurements in the lungs confirmed by right heart catheterization: mean pulmonary arterial pressure greater than 20 mmHg, pulmonary capillary wedge pressure or left ventricular end diastolic pressure less than or equal to 15 mmHg, and pulmonary vascular resistance greater than or equal to 4 Wood units
- Patients on stable background therapy for at least 3 months before screening
- NTproBNP level greater than 300 ng/L (a blood test measuring heart strain)
- Able to provide signed informed consent
Who cannot participate:
- Patients with any other serious medical condition that might interfere with the study
- Those who have had a heart attack or stroke in the past 6 months
- Patients with uncontrolled high blood pressure
- Those with severe liver or kidney disease
- Patients who are pregnant or breastfeeding
- Those currently participating in another clinical trial
- Patients who have had an organ transplant
- Those with a history of drug or alcohol abuse in the past year
- Patients with a known allergy to the study medication
Study focus: The trial will observe how sotatercept affects the right side of the heart and how it interacts with the blood vessels in the lungs. Researchers will measure changes in heart power per beat and the right ventricular pulmonary artery coupling index, along with various heart volumes and ejection fractions using imaging techniques.
Investigational drug: Sotatercept works by targeting specific proteins in the body that are involved in blood vessel growth and repair. It blocks certain proteins that can cause blood vessels to narrow, potentially helping to reduce blood pressure in the lungs and improve how the right side of the heart works.
Summary
Currently, there are two clinical trials available for patients with heritable pulmonary arterial hypertension. Each trial is conducted in a different European country, with one in Denmark and one in the Netherlands. The studies are testing two different approaches to treatment: dapagliflozin, a medication taken orally that was originally developed for diabetes management, and sotatercept, an injectable medication that targets proteins involved in blood vessel function.
Both trials focus on improving heart function, though they use different measures and timelines. The dapagliflozin study emphasizes exercise capacity improvements over a 3-month period, while the sotatercept trial specifically examines right heart function over 6 months. Patients interested in participating should note that both studies require stable current treatment before enrollment and have specific requirements regarding heart function measurements and symptom severity.
