Sotatercept added to usual treatment for pulmonary hypertension from unrepaired heart defects
Tests sotatercept added to usual medicines in adults with Eisenmenger syndrome.
Plain-language title generated from the registry record
Registry title: A Study of Sotatercept for Patients With Eisenmenger Syndrome or Unrepaired Shunt-Associated Pulmonary Arterial Hypertension Resistant to Vasodilator Therapy
Key facts
- Where
- Fukuoka, Kobe, Kyoto, Nagoya, Okayama, Sendai, and Tokyo, JapanSee the 10 sites
- Phase
- Phase 4
Where in Japan
- Kyushu University HospitalFukuoka, JapanTaking people
- The Second Department of Internal Medicine, University of Occupational and Environmental HealthFukuoka, JapanTaking people
- Division of Cardiovascular Medicine, Kobe University HospitalKobe, JapanTaking people
- Department of Cardiovascular Medicine, Graduate School of Medical Science, Kyoto Prefectural University of MedicineKyoto, JapanTaking people
- Department of Cardiology, Nagoya University HospitalNagoya, JapanTaking people
- Department of Cardiovascular Medicine, Faculty of Medicine, Dentistry and Pharmaceutical Sciences, Okayama UniversityOkayama, JapanTaking people
- Department of Cardiovascular Medicine, Tohoku University Graduate School of MedicineSendai, JapanTaking people
- Department of Cardiology, Keio University School of MedicineTokyo, JapanTaking people
- Department of Cardiovascular Medicine, Kyorin University School of MedicineTokyo, JapanTaking people
- Division of Cardiovascular Medicine, Department of Internal Medicine, Showa University Graduate School of MedicineTokyo, JapanTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Kazuya Hosokawa
- Trial code
- NCT07356778
- Record updated
- 21 January 2026
- Original description
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.Read full original descriptionShow less
An Open-label, Randomized, Controlled Trial to Evaluate the Efficacy of Sotatercept Add-on Therapy Compared to Standard PAH Therapy With Pulmonary Vasodilators for Pulmonary Arterial Hypertension Associated With Pulmonary Vasodilator-resistant, Unrepaired Congenital Shunts (ASD, VSD, PDA) Including Eisenmenger Syndrome：SuMILE Trial
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.
Inclusion: adults (≥18 years); unrepaired ASD, VSD or PDA; ≥90 days of pulmonary vasodilator therapy; and either (i) pulmonary vascular resistance (PVR) ≥5 Wood units and mean pulmonary arterial pressure (mPAP) > 20 mm Hg on right heart catheterization within 180 days, or (ii) echocardiographic tricuspid regurgitation velocity >3.4 m/s with right-to-left/bidirectional shunt plus resting SpO₂ ≤92% consistent with cyanosis; baseline 6MWD ≥100 m; ability to complete questionnaires.
Exclusion: prior sotatercept use; severe renal/hepatic/parenchymal lung disease or LVEF <40%; WHO functional class IV; other unrepaired intracardiac shunts; contraindication to sotatercept per label; investigator-judged unsuitability.