STDY OF SIROLIMUS/RAPAMYCIN IN PTS W/ADVNCD PAH REFRACTORY TO CRNTLY AVAIL THRPY

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这项先导性研究是为了评估西罗莫司在严重的晚期肺动脉高压患者中的安全性，尽管目前已有最大限度的治疗，但这些患者的病情没有改善。这项研究将在少数晚期疾病患者身上测试一种对PAH患者来说是新的生物医学干预(西罗莫司)，以评估安全性和有意义的疗效趋势，这可能会支持未来进行更大规模的单中心临床试验。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This pilot study is being done to assess the safety of sirolimus in patients with severe, late-stage pulmonary arterial hypertension who are not improving despite maximal currently available therapy. This study will be testing a biomedical intervention (Sirolimus) that is new to patients with PAH in a small number of patients with advanced disease to assess safety and meaningful trends toward efficacy that would potentially support the future conduct of a larger single-center clinical trial.