CLINICAL TRIAL: VX-770 CFTR ACTIVITY IN CYSTIC FIBROSIS (CF) SUBJECTS WITH GENOT

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。 本研究旨在评价多剂量口服VX-770对G551D基因型囊性纤维化(CF)患者的安全性和耐受性。 这是一项IIa期随机、双盲、安慰剂对照研究。 囊性纤维化(CF)是一种隐性遗传病，在美国约有30,000名儿童和成人，在欧洲约有20,000名儿童和成人。1尽管囊性纤维化的治疗取得了进展，但尚无治愈方法。预测的CF患者的存活年龄中值约为35岁左右。 本研究的研究对象为G551D突变(至少1个等位基因，研究第一部分)或G551D，R117H，2789+5G--&GT；A(至少一个等位基因，研究第二部分)确诊的18岁及以上的男性和女性。 调查人员希望了解或实现以下研究目标： 首要目标 -评价G551D基因携带者多次口服VX-770的安全性和耐受性。 次要目标 -检测G551D基因携带者多次口服VX-770后CFTR活性的生物标志物。 -研究G551D基因携带者多次口服VX-770后VX-770的PK。 -在R117H、2789+5G-&gt；A等位基因携带者多次口服VX-770后，检测CFTR活性的生物标志物，并调查VX-770的PK。 -选择VX-770的剂量进行进一步的临床研究。

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 study is designed to evaluate the safety and tolerability of multiple dose administrations of orally administered VX-770 given to cystic fibrosis (CF) subjects with genotype G551D. This is a Phase IIa, Randomized, Double-Blind, Placebo-Controlled Study. Cystic fibrosis (CF) is a recessive genetic disease that affects approximately 30,000 children and adults in the United States and approximately 20,000 children and adults in Europe.1 Despite progress in the treatment of CF, there is no cure. The predicted median approximate age of survival for a person with CF is the mid-30s. The study subject population of this study is Male and female with G551D mutation (at least 1 allele, Part 1 of study) or G551D, R117H, 2789 + 5G-->A (at least one allele, Part 2 of study) confirmed CF aged 18 years and above. The investigators hope to learn or achieve the following study objectives: Primary Objective - To evaluate the safety and tolerability following multiple dose administrations of orally administered VX-770 given to CF subjects with genotype G551D. Secondary Objectives - To examine biomarkers of CFTR activity following multiple dose administrations of orally administered VX-770 given to CF subjects with genotype G551D. - To investigate the PK of VX-770, following multiple dose administrations of orally administered VX-770 given to CF subjects with genotype G551D. - To examine the biomarkers of CFTR activity and investigate the PK of VX-770, following multiple dose administrations of orally administered VX-770 given to CF subjects with genotype R117H, 2789 + 5G-->A. - To select a dose of VX-770 for further clinical study.