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

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 - SuMILE trial

Status

Recruiting

Phases

Phase 4

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT1071250069

Enrollment

Registered

2025-09-12

Start date

2025-11-12

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Uncorrected CHD-PAH, Eisenmenger syndrome

Interventions

Sotatercept Add-on Therapy, Pulmonary Vasodilator-based Standard of Care

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Male or female patients aged 18 or more years at the time of informed consent. 2. Patients diagnosed with unrepaired atrial septal defect, ventricular septal defect, or patent ductus arteriosus. 3. Patients who have been receiving at least one pulmonary vasodilator for 90 days or more and who, within 180 days prior to enrollment, meet one of the following: MPAP >20 mmHg and PVR >=5 Wood units confirmed by right heart catheterization, or Evidence of pulmonary hypertension (tricuspid regurgitation velocity >3.4 m/s) with right to left shunting confirmed by echocardiography within 180 days prior to enrollment, together with resting hypoxemia (SpO2 92% or less). 4. Six minute walk distance (6MWD) 100 meters or more at screening. 5. Ability to complete questionnaires.

Exclusion criteria

Exclusion criteria: 1. Patients in World Health Organization functional class (WHO-FC) IV. 2. Patients with unrepaired intracardiac shunts other than ASD, VSD, PDA, or partial anomalous pulmonary venous return. 3. Patients with severe renal impairment, hepatic impairment, parenchymal lung disease (e.g., interstitial lung disease, chronic obstructive pulmonary disease), or left heart failure with reduced ejection fraction (LVEF < 40%). 4. Patients with a history of sotatercept administration. 5. Patients in whom sotatercept is contraindicated or otherwise not permitted in accordance with the package insert. 6. Patients deemed inappropriate for study enrollment by the principal investigator.

Design outcomes

Primary

Measure	Time frame
6MWD change from baseline at Week 24	—

Secondary

Measure	Time frame
1. Proportion of patients with death or lung transplantation at Week 24 after enrollment. 2. Proportion of patients with hospitalization for worsening pulmonary hypertension or initiation of continuous subcutaneous or intravenous prostacyclin therapy at Week 24 after enrollment. 3. Change from baseline in World Health Organization functional class (WHO FC) at Weeks 12 and 24 after enrollment. 4. Change from baseline in N terminal pro brain natriuretic peptide (NT proBNP) at Weeks 12 and 24 after enrollment. 5. Change from baseline in emPHasis 10 score at Weeks 12 and 24 after enrollment.	—

Measure

Time frame

1. Proportion of patients with death or lung transplantation at Week 24 after enrollment. 2. Proportion of patients with hospitalization for worsening pulmonary hypertension or initiation of continuous subcutaneous or intravenous prostacyclin therapy at Week 24 after enrollment. 3. Change from baseline in World Health Organization functional class (WHO FC) at Weeks 12 and 24 after enrollment. 4. Change from baseline in N terminal pro brain natriuretic peptide (NT proBNP) at Weeks 12 and 24 after enrollment. 5. Change from baseline in emPHasis 10 score at Weeks 12 and 24 after enrollment.

—

Contacts

Public ContactKeimei Yoshida

Kyushu University

yoshida.keimei.713@m.kyushu-u.ac.jp+81-926425360

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026