Clinical Research Directory

Sota-ES - Sotatercept in Patients With Congenital Heart Disease and Eisenmenger´s Syndrome

The present study seeks to provide pilot data on the safety and efficacy of medical therapy with sotatercept in patients with an established diagnosis of congenital heart disease and Eisenmenger syndrome. CHASE is an interventional, single-arm, open-label study, that will enroll 40 patients with an established diagnosis of CHD and Eisenmenger syndrome. PAH background therapy may be present at the discretion of the investigators at the time of enrolment. CHASE will be performed only in countries where standard PAH therapies are available and reimbursed. At the end of the 24-week patient period, PAH treatment is left to the investigator's discretion.

A Study of Sotatercept for Patients With Eisenmenger Syndrome or Unrepaired Shunt-Associated Pulmonary Arterial Hypertension Resistant to Vasodilator Therapy

What is this study about? This study will test whether adding sotatercept to usual medicines for pulmonary arterial hypertension (PAH) can help adults who have PAH due to unrepaired congenital heart defects (atrial or ventricular septal defect, or patent ductus arteriosus), including Eisenmenger syndrome. These conditions often cause long-standing changes in the lung blood vessels and low oxygen levels. Who can join? About 36 adults (age ≥18 years) in Japan whose PAH has not improved enough with pulmonary vasodilators may join. People with very severe symptoms (WHO class IV) or other serious illnesses will not be enrolled. What will happen if I join? Participants will be randomly assigned (like a coin flip, in a 2:1 ratio) to: Sotatercept + vasodilator-based PAH care, or vasodilator-based PAH care alone. The study lasts 24 weeks. Those who receive sotatercept will have injections every 3 weeks. All participants will have clinic visits and tests at the start, week 12, and week 24, including a 6-minute walk test (how far you can walk in 6 minutes), blood tests, questionnaires, and other heart-lung assessments used in routine PAH care. What are the possible benefits? Sotatercept improved exercise capacity and heart-lung measures in other PAH studies, but people with unrepaired heart defects were not included. This study may or may not help you directly, but it may help doctors learn how to use sotatercept safely in this group. What are the possible risks? Side effects seen with sotatercept include increase in haemoglobin, low platelets, nosebleeds, telangiectasia (small dilated blood vessels), bleeding, and blood clots. People with Eisenmenger syndrome can have both bleeding (for example, haemoptysis) and clotting risks. The study will check complete blood counts (CBC) regularly and adjust or pause dosing using label-based rules. Other risks are those of standard PAH care and blood tests. Time and location The study is conducted at multiple hospitals in Japan. Study participation lasts about 6 months. Costs and payments The study drug and study-specific tests will be provided at no cost. Usual medical care not required by the study will follow each hospital's standard billing. There is no required payment to join. Any travel reimbursement or stipends will follow site policy. Privacy Your information will be kept confidential. Results will be shared in journals and at meetings without using your name. Who to contact If you are interested or have questions, please contact the study team at the participating hospital.

Pentoxifylline as an Adjunct Therapy for Patients With Eisenmenger Syndrome

The Eisenmenger syndrome corresponds to the most advanced form of pulmonary arterial hypertension associated with congenital heart disease. The syndrome causes chronic hypoxemia, with an increase in erythrocyte mass, which predisposes to thrombotic complications. Pentoxifylline is a xanthine derivative and it is considered as a hemorrheological agent with described effects of reduction in erythrocyte and platelet aggregation, adhesion and activation of leukocytes, and endothelial damage. The main objective of this study is to verify if the chronic oral administration of pentoxifylline to Eisenmenger patients induces an increase in the circulating levels of thrombomodulin, a naturally occurring proteoglycan with anticoagulant, anti thrombotic and anti-inflammatory properties.