A PHASE II STUDY OF THE MTOR INHIBITOR SIROLIMUS IN NEUROFIBROMATOSIS TYPE 1

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。 这项研究的主要目的是：1)确定mTOR抑制剂西罗莫司是否在药动学指导下按连续给药计划每日两次口服给药：a)基于1型神经纤维瘤病(NF1)和无法手术的进展性丛状神经纤维瘤(PN)的儿童和青年患者的容量MRI测量，增加疾病进展的时间；b)在试验开始时没有明确的放射学进展记录的情况下，根据儿童和成人NF1和无法手术的PN的体积MRI测量结果产生客观的放射学反应；2)评估西罗莫司在这类患者中长期应用的可行性和毒性；以及3)描述西罗莫斯对这些患者的药代动力学特征。

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. The primary aims of this study are to: 1) determine whether Mtor Inhibitor Sirolimus, administered orally twice daily on a continuous dosing schedule using pharmacokinetically-guided dosing: a) increases time to diseas progresssion based on volumetric MRI measurements in chldren and young adults with neurofibromatosis Type 1 (NF1) and inoperable progresssive plexiform neurofibromas (PN), b) results in objective radiographic responses based on volumetric MRI measurements in children and adults with NF1 and inoperable PN in the absence of documented radiographic progression at trial entry; 2) evaluate the feasibility and toxicity of chronic Sirolimus adminstration in this patient population; and, 3) characterize the pharmacokinetic profile of Sirolimus when administered to this patient population.