The main purpose of this study is to see if important factors can be identified in each of the three categories (patient, catheter, and catheter management) that are linked to blood clotting complications. This is a multicenter, prospective observational study to evaluate risk factors associated with complications of peripherally inserted central catheter (PICCs) and midline catheters. Both adult and pediatric patients with CF who receive care at participating centers will be eligible for participation. This study can last for 2 weeks.
The purpose of this study is to evaluate the efficacy and safety of open-label exposure of gNO in patients with NTM lung disease. Subjects will receive the study drug by inhaling through a nasal mask. Subjects will be treated for 3 weeks (5 days per week) and followed monthly for 3 months.
The purpose of this stuThe purpose of this study is to learn more about the safety and effectiveness of VX-445 used in combination with TEZ and VX-561 in patients with cystic fibrosis. Participation could last for approximately 100 weeks. This will include a treatment period that will last approximately 96 weeks, and a safety follow-up period that will last approximately 4 weeks after the last dose of Study Drug. Approximately 400 people will participate in this study.
In this study, PTI-808 will be given alone and in combination with two other experimental drugs, PTI-801 and PTI-428. PTI-428, PTI-801, and PTI-808 are considered cystic fibrosis transmembrane conductance regulator (CFTR) modulators being developed to treat the underlying mechanism of cystic fibrosis. PTI-428 is a CFTR amplifier that increases the amount of CFTR protein in the cell, PTI-801 is a CFTR corrector that improves the processing of CFTR protein in the cell, and PTI-808 is a CFTR potentiator to enhance the activity of CFTR at the cell surface. Individual modulators have been shown to have limited activity, and therefore it is predicted that a combination of modulators will be required to fully address the CFTR protein defects. The study will also obtain information on how the study drug moves into, through, and out of the body. The expected duration of this study is up to 74 days
This is a research study to find out if a drug called lenabasum can help people with Cystic Fibrosis (CF) and if it is safe to take without causing too many side effects. There are 9 planned study visits in the institution during this study.
This is a research study to test a new investigational drug, PTI-801. An investigational drug is one that is not approved by the US FDA. In this study, PTI-801 will be compared to placebo (a capsule that looks like the investigational drug, but does not contain an active drug substance). Although PTI-801 has been given to normal healthy adults, this is the FIRST time that PTI-801 has been given to people with CF.
This study has two parts and will assess your hearing and the potential effects of antibiotic-induced ototoxicity. Ototoxicity is a potential effect of drugs used to treat your lung infections, and its effect on your ears (oto=ears), particularly structures in your inner ear that enable hearing and balance. Part One of the study will assess your inner ear function that may be associated with hearing loss and / or loss of balance in cystic fibrosis patients taking tobramycin. Part Two is looking to determine if SPI-1005, an investigational drug, influences hearing loss, due to mediations (tobramycin).
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
Lamprene®/Clofazimine, is a product of the pharmaceutical company named Novartis Pharmaceuticals Corporation. Lamprene®/Clofazimine is approved by FDA (the U.S Food and Drug Administration) for the treatment of leprosy. It is being tested in non-Novartis clinical studies for drug resistant tuberculosis and non-tuberculous mycobacteria (NTM).
If you have been diagnosed with NTM, then your doctor may decide that this infection can be treated with Lamprene®/Clofazimine. This medicine is provided to you in an expanded access program. This means that this medicine is not registered for the treatment of NTM, but it can be used in special situations where there are no other possible treatments. For example, this may be because you have a type of Mycobacterial infection that is resistant or failed to respond optimally to other drugs, or because you have had side effects that prevent the use of other drugs.