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

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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.