The purpose of this program is to make available the TRIPLE COMBO to patients with Cystic Fibrosis to improve their health. The program drug, elexacaftor-tezacaftor-ivacaftor, is not available to patients in the US except through a special access program filed with the FDA. Elexacaftor-tezacaftor-ivacaftor (no trade name as of yet) is a drug combination that is currently under FDA review, but the FDA has approved expanded access to the TRIPLE COMBO while it is reviewing the application for final drug approval. Those eligible for the TRIPLE COMBO must have at least one copy of the CF gene mutation F508del.
In order to participate in this program, participants must be at least 12 years of age or older (there is no upper age limit). Participation in this program may last until a decision has been made on the approval of the TRIPLE COMBO. If the drug is approved and available by prescription, a prescription will be submitted. Once that has been filled, the participant will be discontinued from this expanded access program. There may be up to 20 subjects enrolled at this site.
The purpose of this study is to learn more about the effectiveness and safety of VX-561 used in patients with cystic fibrosis. Patients who have cystic fibrosis and are currently taking a stable dose of ivacaftor (IVA) are invited to take part in this research study. VX-561 is a form of IVA, an approved medication for some types of CF, that lasts longer in the body, allowing the medicine to be taken once a day instead of twice a day. Participation could last for approximately 20 weeks. There will be approximately 88 people participating in this study.
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.
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 randomized, double-blind, multi-center study in pediatric
subjects age 3 months to less than 18 years with CF and newly
detected PA respiratory tract colonization/infection. The study
schedule will consist of a minimum of 13 visits: Screening, Day 1
(Baseline and Randomization), Day 29, Weeks 6, 8, 16, and at
12-week intervals thereafter through Week 112. Subjects may be
screened up to 14 days prior to the Baseline visit to determine
eligibility for participation in the study. Screening and Baseline may
occur on the same day for subjects.
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 an observational study which will collect sweat chloride values from approximately 1000 cystic fibrosis subjects who are currently on cystic fibrosis transmembrane conductance regulator (CFTR) modulators. This study will require one visit to an MUSC clinic which will last approximately 2 hours. Compensation is provided.
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).