[PLACEHOLDER—wording to be provided by study team] This research study is studying Tiprelestat (elafin) as a possible treatment for pulmonary arterial hypertension.
This study will compare Tiprelestat to placebo (an inactive substance that looks like Tiprelestat and is given the same way but has no effect on the body) to determine the efficacy, safety and tolerability of Tiprelestat. Stanford University is sponsoring this research study.
[PLACEHOLDER—wording to be provided by study team] PAH is a disease of progressive obliteration of the lung vasculature that results from elastase mediated degradation of elastin, endothelial dysfunction, smooth muscle cell proliferation and chronic peri-and intravascular inflammation. These features can be a consequence of reduced bone morphogenetic protein receptor 2 (BMPR2), the most common mutation associated with PAH. While current PAH treatments largely aim to dilate unobstructed pulmonary arteries, there is an unmet need to find a therapy that is disease modifying by way of addressing these underlying cellular and molecular features of PAH.
The purpose of this study is to compare the effects of Tiprelestat to placebo on PAH patients who are already taking other PAH medications to determine the efficacy, safety and tolerability of Tiprelestat.

