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

该子项目是利用 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得主要资金， 因此可以在其他CRISP条目中表示。列出的机构是 中心，不一定是研究者的机构。 本研究的主要目的是：1）确定Mtor抑制剂西罗莫司，使用药代动力学指导的给药，以连续给药方案每日两次口服给药：a）在患有神经纤维瘤病1型（NF 1）和不可手术的进行性丛状神经纤维瘤（PN）的儿童和年轻成人中，基于体积MRI测量，增加疾病进展的时间，B）在试验入组时没有记录的放射学进展的情况下，在患有NF 1和不可手术PN的儿童和成人中，基于体积MRI测量结果，导致客观放射学缓解; 2）评价西罗莫司长期给药在该患者人群中的可行性和毒性;和3）表征西罗莫司给药于该患者群体时的药代动力学特征。

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.