An Open-label, Randomized, Controlled Trial to Evaluate Sotatercept Add-on Therapy Compared to Standard Pulmonary Vasodilator Therapy for Pulmonary Arterial Hypertension Due to Unrepaired Congenital Heart Defects Including Eisenmenger Syndrome
Led by Kazuya Hosokawa · Updated on 2026-01-21
36
Participants Needed
10
Research Sites
34 weeks
Total Duration
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What this Trial Is About
Researchers are evaluating whether adding sotatercept to standard medicines can help adults with pulmonary arterial hypertension (PAH) caused by unrepaired congenital heart defects such as atrial or ventricular septal defects and patent ductus arteriosus, including those with Eisenmenger syndrome. This condition affects lung blood vessels and oxygen levels, and the study focuses on adults whose PAH has not improved enough with current vasodilator treatments. The trial is a randomized Phase 4 study conducted in Japan, aiming to understand sotatercept's effects in this specific group.
Participants will be randomly assigned in a 2:1 ratio to receive either sotatercept plus usual vasodilator therapy or vasodilator therapy alone for 24 weeks. Sotatercept is given as subcutaneous injections every 3 weeks, starting with a lower dose and increasing if safety criteria are met, with dose adjustments based on blood counts. All participants continue their stable PAH therapy, and changes are generally discouraged unless needed for safety.
During the study, participants will attend visits at the start, week 12, and week 24 to undergo tests including a 6-minute walk test, blood tests, questionnaires, and heart-lung assessments used in routine PAH care. Researchers will monitor changes in walking distance, clinical events, and heart-lung function. Safety monitoring includes regular blood counts to manage potential side effects. The total participation lasts about six months, with study treatments and tests provided at no cost.
CONDITIONS
Brief Title
A Study of Sotatercept for Patients With Eisenmenger Syndrome or Unrepaired Shunt-Associated Pulmonary Arterial Hypertension Resistant to Vasodilator Therapy
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
Adults aged 18 years or older
Diagnosis of unrepaired atrial septal defect, ventricular septal defect, or patent ductus arteriosus
At least 90 days of pulmonary vasodilator therapy
Pulmonary vascular resistance of 5 or more Wood units and mean pulmonary arterial pressure over 20 mm Hg on right heart catheterization within 180 days, or echocardiographic tricuspid regurgitation velocity over 3.4 m/s with right-to-left or bidirectional shunt plus resting oxygen saturation 92% or less indicating cyanosis
Baseline 6-minute walk distance of at least 100 meters
Ability to complete study questionnaires
You will not qualify if you...
WHO functional class IV symptoms or other unrepaired intracardiac shunts
Severe kidney, liver, or lung disease or left ventricular ejection fraction below 40%
Prior use of sotatercept
Contraindications to sotatercept according to its label
Judged unsuitable for the study by the investigator
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Treatment
Duration - 24 weeks
Participants receive sotatercept injections every 3 weeks for 24 weeks as add-on to stable pulmonary vasodilator therapy or continue stable pulmonary vasodilator therapy alone.
8 visits every 3 weeks for sotatercept injections; similar visit schedule for comparator group
Trial Site Locations
Total: 10 locations
1
Kyushu University Hospital
Fukuoka, Not Required For This Country, Japan, 8150014
Actively Recruiting
2
The Second Department of Internal Medicine, University of Occupational and Environmental Health
Fukuoka, Japan
Actively Recruiting
3
Division of Cardiovascular Medicine, Kobe University Hospital
Kobe, Japan
Actively Recruiting
4
Department of Cardiovascular Medicine, Graduate School of Medical Science, Kyoto Prefectural University of Medicine
Kyoto, Japan
Actively Recruiting
5
Department of Cardiology, Nagoya University Hospital
Nagoya, Japan
Actively Recruiting
6
Department of Cardiovascular Medicine, Faculty of Medicine, Dentistry and Pharmaceutical Sciences, Okayama University
Okayama, Japan
Actively Recruiting
7
Department of Cardiovascular Medicine, Tohoku University Graduate School of Medicine
Sendai, Japan
Actively Recruiting
8
Department of Cardiology, Keio University School of Medicine
Tokyo, Japan
Actively Recruiting
9
Department of Cardiovascular Medicine, Kyorin University School of Medicine
Tokyo, Japan
Actively Recruiting
10
Division of Cardiovascular Medicine, Department of Internal Medicine, Showa University Graduate School of Medicine
Protocol for an open-label, randomised, controlled trial to evaluate the efficacy and safety of sotatercept add-on therapy compared with pulmonary vasodilator-based standard of care for pulmonary vasodilator-resistant pulmonary arterial hypertension associated with unrepaired congenital shunts (atrial septal defect, ventricular septal defect or patent ductus arteriosus), including Eisenmenger syndrome: the SuMILE trial.
