CLINICAL TRIAL: A PHASE II STUDY OF THE MTOR INHIBITOR SIROLIMUS IN NEUROFIBROMA

临床试验：MTOR 抑制剂西罗莫司治疗神经纤维瘤的 II 期研究

• 批准号: 8167357
• 负责人: Roger Packer
• 金额: $ 0.49万
• 依托单位: CHILDREN'S RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-01-20 至 2010-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8167357
• 关键词: Adult; Child; Chronic; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Dose; Drug Kinetics; Funding; Grant; Institution; Magnetic Resonance Imaging; Measurement; Neurofibromatosis 1; Phase; Plexiform Neurofibroma; Research; Research Personnel; Resources; Schedule; Sirolimus; Source; Time; Toxic effect; United States National Institutes of Health; base; inhibitor/antagonist; neurofibroma; patient population; response; young adult

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.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 这项研究的主要目的是：1）确定使用药代动力学引导的给药的连续给药时间表，每天两次口服MTOR抑制剂Sirolimus是否会增加疾病进度的时间基于Chldren和Youngs in Chld Neurofibromatsis type 1型（NFEX）（NFEX）（NF）（nF1 nf）的疾病进度的时间（ b）基于NF1儿童和成年人的体积MRI测量和无法使用的PN的体积射线照相响应，在试验进入时没有记录的X光摄影进展； 2）评估该患者人群中慢性西罗莫司施用的可行性和毒性； 3）特征是对该患者人群给药时Sirolimus的药代动力学特征。