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

NCT07498803 | Not Yet Recruiting Phase 2 DMC

• 2 other identifiers: KKS-3442026-525914-68-00
• Study Type: interventional
• Target: 40
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Congenital Heart Disease (CHD); Eisenmenger Syndrome; PAH

Keywords

Congenital heart disease with Eisenmenger syndrome; WHO-FC II or III; PAH therapy

Outcome Measures

Primary Outcomes (1)

• Assessment of the effect on pulmonary vascular resistance (PVR)

  Change in PVR from screening at week 24 after 24 weeks of treatment with Sotatercept

  24weeks

Study Arms (1)

Single arm

EXPERIMENTAL

Sotatercept will be administered subcutaneously in 3-weekly intervals at study sites beginning at a dose of 0.3 mg/kg body weight followed by up titration to the target dose of 0.7 mg/kg body weight

Drug: Sotatercept

Interventions

Sotatercept DRUG

Sotatercept will be administered subcutaneously in 3-weekly intervals at study sites beginning at a dose of 0.3 mg/kg body weight followed by up titration to the target dose of 0.7 mg/kg body weight.

Single arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥18 years
• Congenital heart disease with Eisenmenger syndrome (known unrepaired atrial septal defect, and/or ventricular septal defect, and/or patent ductus arteriosus; patients with anomalous pulmonary venous drainage will not be considered)
• Eisenmenger syndrome defined as right-to-left or bi-directional shunt with a mPAP \>25 mmHg, PAWP \< 15 mmHg, and PVR \>5 WU
• In patients with pre-tricuspid shunt, the consideration of Eisenmenger syndrome requires one of the following: Systemic arterial O2 saturation (SaO2) at rest \<88% and more than 70%, and/or SaO2 \<80% during 6MWT, and secondary erythrocytosis (Hb \> 15.0 g/dl for females and 16.0 g/dl for males)
• On stable doses of background PAH therapy\* and diuretics (i.e., patient-individual dose goal for each therapy achieved) for ≥30 days
• minute walking distance \>100 m
• WHO-FC II or III
• Written informed consent

You may not qualify if:

• Age \<18 years
• Diagnosis of pulmonary hypertension groups 2, 3, 4, or 5
• Hospitalization or change in PAH background therapies within 30 days prior to screening (changes in dose of diuretics or parenteral prostanoids \[\<10% change in infusion rate over the preceding 3 months\] are allowed)
• Uncontrolled systemic hypertension as evidenced by sitting systolic blood pressure \>160 mmHg or sitting diastolic blood pressure \>100 mmHg during screening visit after a period of rest
• Baseline systolic blood pressure \<90 mmHg at screening
• Left ventricular systolic dysfunction (LVEF 40%)
• Restrictive lung disease with a TLC \< 60% AND demonstration of more than mild fibrosis on chest CT prior to enrolment (note that patients with congenital heart disease may have thoracic cage deformities \[e.g. pectus\] that may lead to thoracic cage restriction in the absence of parenchymal lung disease).
• Obstructive lung disease (FEV1 \< 60% pred. and FEV1/FVC \<60%)
• Significant liver disease (Child II or III)
• Any of the following clinical laboratory values at the screening visit:
• Estimated glomerular filtration rate (eGFR) \<30 mL/min/m2 (as defined by the Modification of Diet in Renal Disease \[MDRD\] equation)
• Serum alanine aminotransferase, aspartate aminotransferase, or total bilirubin levels \>3 × ULN (bilirubin criterion waived if there is a documented history of Gilbert's syndrome)
• Baseline platelet count \<50,000/µl (\<50.0 x 109/L) at screening
• Documented episodes of previous repetitive hyperviscosity syndrome
• History of haemoptysis within 12 months prior to screening, and/or repeated severe epistaxis (≥ 1 episode per month)

Sponsors & Collaborators

• Philipps University Marburg: lead

MeSH Terms

Conditions

Heart Defects, Congenital; Eisenmenger Complex

Interventions

ACE-011

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Stephan Rosenkranz, Prof. Dr.

  University Hospital Cologne Department of Cardiology, Pulmonology, and Intensive Care Medicine Heart Center Cologne Cardiovascular Research Center (CCRC) Center for Molecular Medicine Cologne (CMMC)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

CHASE Study Team

CONTACT

+4915114980915; chase@kks.uni-marburg.de

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is an interventional, prospective, non-randomized, single-arm, open-label, multi-center, phase II study.

Study Record Dates

• First Submitted: March 19, 2026
• First Posted: March 27, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): March 31, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: March 27, 2026

Record last verified: 2026-03