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的一个 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来源获得了主要资金， 因此可在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 本研究旨在评价基因型为G551 D的囊性纤维化（CF）受试者多次口服给予VX-770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）是一种隐性遗传疾病，在美国影响约30，000名儿童和成人，在欧洲影响约20，000名儿童和成人。CF患者的预测中位近似生存年龄为35岁左右。 本研究的研究受试者群体是具有G551 D突变（至少一个等位基因，研究的第1部分）或G551 D、R117 H、2789 + 5G-->A（至少一个等位基因，研究的第2部分）的男性和女性，其证实了18岁及以上的CF。 研究者希望了解或达到以下研究目的： 主要目的 - 评价基因型G551 D CF受试者多次口服VX-770后的安全性和耐受性。 次要目的 - 检查对具有基因型G551 D的CF受试者多次口服给予VX-770后CFTR活性的生物标志物。 - 研究基因型G551 D CF受试者多次口服VX-770后VX-770的PK。 - 检查CFTR活性的生物标志物，并研究在对具有基因型R117 H，2789 + 5G-->A的CF受试者多次口服给予VX-770后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.