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 vaso
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- #Clinical Trial
- #Pulmonary Arterial Hypertension
- #Congenital Heart Disease
- The SuMILE trial evaluates sotatercept add-on therapy versus standard vasodilator-based care for pulmonary arterial hypertension (PAH) in patients with unrepaired congenital shunts.
- 36 adults with vasodilator-resistant PAH due to unrepaired ASD, VSD, or PDA, including Eisenmenger syndrome, will be randomized 2:1 to receive sotatercept plus standard therapy or standard therapy alone.
- Primary endpoint: Change in 6-minute walk distance from baseline to week 24. Secondary endpoints include mortality, lung transplantation, hospitalizations, and changes in functional class and biomarkers.
- Sotatercept is administered subcutaneously every 3 weeks, with dose adjustments based on hemoglobin and platelet levels.
- The study is a prospective, exploratory, multicenter, open-label, randomized controlled trial conducted at 11 Japanese tertiary centers.
- Findings will be disseminated via peer-reviewed journals, conferences, and trial registries.