Cystic fibrosis is caused by changes in the genes of your DNA (Deoxyribonucleic Acid) encoding for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. These changes cause the CFTR to stop working as it should. GLPG2222 is being studied for treatment of cystic fibrosis. The study drug is designed to improve the expression and function of CFTR, which may improve some of the symptoms of cystic fibrosis.
The purpose of this study is to learn more about the safety and efficacy of the combination of tezacaftor/ivacaftor (TEZ/IVA) in subjects with CF who have previously discontinued Orkambi. Orkambi® is a combination of ivacaftor with another drug called lumacaftor. This study is being conducted to test whether combining ivacaftor with tezacaftor instead will improve symptoms of cystic fibrosis and cause fewer respiratory side effects.
This study is being done to evaluate an experimental treatment for people with Cystic Fibrosis who have a bacterial infection in their lungs. Subjects will be randomized (selected by chance) to either be treated with an investigational inhaled gaseous drug (in 99.5% nitrogen) that is mixed with room air or breathe 100% nitrogen that is mixed with room air. If you are randomized to the investigational treatment, we are asking you to breathe in a gas called ?nitric oxide?. Participation in the study will take about 43 days
This is a Phase 2, 2-part study. Adults over the age of 18 with cystic fibrosis are eligible.
VX-152 given alone or in combination with VX-661 and ivacaftor is an investigational drug and is still being tested for safety and effectiveness. VX-661 given alone or with ivacaftor is investigational and is still being tested for safety and effectiveness. Ivacaftor, the third drug used in this study is approved in a number of countries globally for use in patients with CF as young as 2 years, depending on the country. Ivacaftor (Kalydeco) is approved in the United States for use in patients with CF.
This study will evaluate the pharmacokinetic of VX-661 and ivacaftor after administration of multiple doses of VX-661 in combination with ivacaftor over 8 weeks with subjects 6 to 11 years old who are homozygous or heterozygous for the F508del-CFTR Mutation. The 8 weeks will consist of a screening period of 4 weeks, a treatment period that will last 2 weeks, and if needed, a follow up visit taking place 2 weeks after the first dose of the Study Drug.
This is an open-label study with two treatment groups based on weight. The purpose of this study is to learn more about the safety and tolerability of the combination of VX-661 and ivacaftor in subjects with CF. Part A of the study will be receiving one 50mg tablet of VX-661 in the morning, one 75mg capsule of ivacaftor in the morning and one 75mg capsule of ivacaftor in the evening. Part B receiving one 50mg tablet of VX-661 in the morning, one 150mg tablet of ivacaftor in the morning and one 150mg tablet of ivacaftor in the evening.
This study aims to test the safety and tolerability of the study drug, PTI-428, when used to treat cystic fibrosis. Approximately 36 individuals will take part study-wide and adult males and females of the ages 18-55 years old with a diagnosis of Cystic Fibrosis are eligible to participate.
There are 2 different groups within the study and depending on the group you paticipate in the study will last between 5 to 7 weeks.
The study is being used to evaluate the efficacy and safety of differing durations of IV antibiotic treatment for CF pulmonary exacerbations. Subject eligibility includes males and females 18 years of age or older. Participation in the study will take 3 visits over a period of 24 to 35 days.
You are invited to volunteer for this non-interventional study if you have been prescribed tobramycin inhalation powder (TOBI® Podhaler™) or another inhaled medication used to treat Cystic Fibrosis (CF) related Pseudomonas aeruginosa (bacteria).
The purpose of this non-interventional study is to assess how well TOBI Podhaler or other approved inhaled antibiotic medications work on treating this bacterium in the lungs, and their safety as used in medical practice. A non-interventional study is a study in which individuals are only observed and certain outcomes are measured. The study will entail around 20 visits over the course of 5 years.
This study will evaluate the safety of QR-010 in subjects with Cystic Fibroisis 18 to 60 years old who are homozygous for the F508del mutation.
This study has 3 phases: Screening, In-Hospital (treatment), and Follow- Up. Subjects will be assigned to one of 8 dosing groups. Four of the groups will receive 1 dose of drug (Single Dose Groups), and the other 4 groups will receive 12 doses of drug (Multiple Dose Groups).