The purpose of the trial is to evaluate the safety and efficacy of the new combination therapy. Over a period of about four weeks, subjects will take the study medication once daily, while other participants receive a matching placebo. Safety will be monitored through reports of any side effects, regular blood tests, standard 12‑lead ECG recordings to check heart rhythm, and measurements of Sweat chloride levels, which reflect how the disease is affecting the body.

Effectiveness will be assessed by measuring lung function using the percent predicted forced expiratory volume in one second (ppFEV1) and by having participants complete a disease‑specific questionnaire that evaluates breathing symptoms (CFQ R). The study involves several clinic visits for these tests and for overall health checks.

The purpose of this study is to measure the levels of these medications in the blood of patients who are already taking this drug combination. The medications are available as Kaftrio (which contains all three drugs) and Kalydeco (which contains ivacaftor alone). The study will look at how these medications circulate in the body after patients have been taking them regularly.

During the study, participants will continue their regular treatment with these medications, and blood samples will be collected to measure drug levels. The study will also track how well patients respond to the treatment and monitor any side effects that may occur. This research will help better understand how these medications work in people with Cystic Fibrosis.

The study will be conducted in two parts. Initially, healthy participants will receive single doses of RCT2100 to assess its safety. Following this, participants with Cystic Fibrosis will receive multiple doses to further evaluate safety and to see how the medication is distributed in the body. The trial will also explore whether the treatment can improve lung function and quality of life for those with Cystic Fibrosis. Participants will be monitored for any side effects and changes in their condition throughout the study.

The trial aims to provide valuable information about the potential of RCT2100 as a treatment for Cystic Fibrosis. By understanding how the medication works in the body and its effects on the disease, researchers hope to develop a new option for managing this challenging condition. The study is expected to continue until early 2025, with recruitment starting in mid-2024.

The purpose of the study is to evaluate how safe and well-tolerated tamoxifen citrate is for these patients. Participants in the study will take tamoxifen citrate in the form of a film-coated tablet, which is taken orally. The study will last for 24 weeks, during which time the participants will be monitored for any side effects or adverse events. The study will also look at changes in lung function, the number of lung infections, hospitalizations, and other health measures related to cystic fibrosis.

Throughout the study, researchers will collect data on various health indicators, such as lung function tests and the frequency of lung infections. They will also monitor the use of antibiotics and any changes in body weight and sweat chloride levels, which are important in understanding the impact of cystic fibrosis. The goal is to gather information that could help improve treatment options for patients with cystic fibrosis who are not currently eligible for CFTR modulator therapies.

Participants in the study will receive either Levofloxacin or Piperacillin/Tazobactam as part of their treatment. The study will involve taking samples from the lungs to measure the concentration of the antibiotics. This will be done using three methods: collecting fluid from the lining of the lungs, taking a small tissue sample, and using a technique called in-vivo microdialysis, which involves measuring the drug concentration in the body over a period of time. These methods will help researchers understand how the drugs are distributed in the lungs.

The study will be conducted over a short period, with the main focus on comparing the three methods of measurement. The results will provide valuable information on how these antibiotics work in the lungs of patients with Chronic Obstructive Pulmonary Disease and Cystic Fibrosis, potentially leading to better treatment strategies in the future.

Participants in the study will use a device called the Aerogen InnoSpire Go nebulizer to inhale the medication. This device helps deliver the treatment deep into the lungs. The study will compare the effects of SPL84 with a placebo solution, which looks like the treatment but does not contain the active substance. The trial will monitor participants for any side effects and changes in their health, including lung function and overall well-being.

The study will take place over several months, during which participants will receive multiple doses of SPL84. Researchers will collect information on how the body processes the medication and its impact on symptoms of cystic fibrosis. The goal is to determine the most suitable dose that is both safe and effective for patients with this genetic mutation. Participants will be closely monitored throughout the study to ensure their safety and to gather valuable data on the treatment’s potential benefits.

The purpose of the study is to evaluate how safe and tolerable this combination therapy is for individuals with Cystic Fibrosis who are 2 years of age and older. Participants in the study will receive the treatment over a period of time, and their health will be monitored to observe any side effects or changes in their condition. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment.

Throughout the study, participants will undergo regular health checks, including assessments of their lung function and other health indicators. The study aims to provide valuable information on the long-term use of this combination therapy, helping to improve treatment options for those living with Cystic Fibrosis.

The purpose of the study is to assess how the body processes these medications and to ensure they are safe and well-tolerated in children aged 1 to 11 years. Participants will be divided into different groups, and the study will be conducted in two parts. In the first part, the focus will be on understanding how the medications are absorbed and processed by the body. In the second part, the safety and tolerability of the treatment will be monitored over a period of 24 weeks.

Throughout the study, participants will receive either the combination of VX-121, Tezacaftor, and Deutivacaftor or a placebo. The study aims to gather information on any side effects and how the treatment affects the symptoms of Cystic Fibrosis. This research is important for developing new therapies that could improve the quality of life for those living with this condition.

The purpose of the study is to determine if the higher dose of 6000 IU of cholecalciferol is better at normalizing bone metabolism and enhancing bone density and muscle strength in children with cystic fibrosis. Participants will receive either the higher dose or the lower dose of cholecalciferol, and some may receive a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving which treatment, to ensure unbiased results.

Throughout the study, participants will have regular check-ups to monitor their health and measure the effects of the treatment. These check-ups will include assessments of vitamin D levels, bone density, and muscle strength. The study is expected to last for two years, providing valuable insights into the potential benefits of higher doses of cholecalciferol for improving bone health in children with cystic fibrosis.

Participants in the study will receive Teicoplanin through inhalation at a dose of 200 mg twice a day. The treatment will be given in two cycles, each lasting 28 days, with a 28-day break in between the cycles. The study will monitor the participants’ health and any side effects they may experience during and after the treatment cycles. The goal is to ensure that the medication is safe and to observe how it affects the bacterial infection in the lungs.

Throughout the study, various health checks will be conducted to assess lung function and the presence of the bacteria in the lungs. These checks will help determine if the treatment is effective in reducing the bacterial load and improving lung health. The study will also look at any changes in symptoms such as coughing, chest tightness, and breathing difficulties. The information gathered will contribute to understanding how well Teicoplanin works for treating these infections in cystic fibrosis patients.

The purpose of this study is to evaluate how Thymosin alpha 1 affects certain inflammatory substances in the body, which are often elevated in people with Cystic Fibrosis. These substances, known as cytokines, include IL-1ß, IL-8, IL-17A, IL-6, and TNF-alpha. The study will monitor changes in these cytokines to understand the medication’s activity. Participants will receive the medication over a period of time, and their health will be closely monitored through various tests and assessments to ensure safety and tolerability.

Throughout the study, researchers will also evaluate the medication’s impact on lung function and quality of life. This will involve checking changes in lung capacity and using a questionnaire designed for people with Cystic Fibrosis. The study aims to provide valuable insights into the potential benefits of Thymosin alpha 1 for managing Cystic Fibrosis symptoms and improving patients’ overall well-being.

The purpose of the study is to assess how well these treatments are tolerated by young children with Cystic Fibrosis, specifically those aged 12 to less than 24 months. Participants will receive the treatment orally in the form of granules. The study will last for a period of 24 weeks, during which the safety of the treatment will be closely monitored. This includes checking for any side effects, changes in vital signs, and other health indicators.

Throughout the study, researchers will also measure the levels of the treatment in the body and observe any changes in sweat chloride levels, which is a marker used to assess the effectiveness of the treatment in Cystic Fibrosis. The trial aims to provide valuable information on the safety and potential benefits of these treatments for young children with this condition.

The study uses a crossover design, which means participants receive both anakinra and placebo at different times during the study. The treatment involves daily injections of 100 mg anakinra or placebo for 28 days. The main purpose is to assess whether anakinra can improve breathing function in adults with Cystic Fibrosis, with the possibility of later including adolescents if the treatment proves effective in adults.

During the study, researchers will monitor lung function through various breathing tests. They will also check how well participants tolerate the medication by performing physical examinations and laboratory tests. Additional assessments include examining changes in lung structure using MRI scans, testing samples of mucus from the lungs, and evaluating quality of life through questionnaires.

The purpose of the study is to assess how well these medications work over an 8-week period in adults aged 18 and older who have cystic fibrosis with specific rare genetic mutations. Participants will be randomly assigned to receive either the investigational medications or a placebo. The study is designed as a crossover trial, meaning that participants will receive both the investigational medications and the placebo at different times during the study. This helps researchers understand the effects of the medications more clearly.

Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. These check-ups will include tests to measure lung function, sweat chloride levels, and body weight, as well as assessments of overall health and any side effects. The study aims to provide valuable information on the potential benefits and safety of Dirocaftor, Posenacaftor, and Nesolicaftor for people living with cystic fibrosis.

The purpose of the study is to evaluate the long-term safety and tolerability of this combination therapy in individuals with cystic fibrosis. Participants will take the medication over a period of time and will have regular check-ups to monitor their health. These check-ups will include assessments of vital signs, laboratory tests, and other evaluations to ensure the treatment is safe and well-tolerated.

Throughout the study, researchers will also look at how the treatment affects lung function, measured by a test called percent predicted forced expiratory volume in 1 second (ppFEV1), and the level of sweat chloride, which is a marker of cystic fibrosis. Additionally, the study will track the number of lung infections, known as pulmonary exacerbations, that participants experience. This information will help determine the effectiveness of the treatment in managing cystic fibrosis symptoms over the long term.

Participants in the study will receive either the VX-522 treatment alone or in combination with Ivacaftor. The study will involve different groups receiving varying doses to determine the most effective and safe dosage. The study will monitor participants’ health and any side effects they may experience during the treatment period. The eFlow Nebuliser System is specifically designed to deliver the VX-522 treatment effectively to the lungs.

The study will take place over several weeks, with participants attending regular check-ups to assess their health and the effects of the treatment. The goal is to gather information on how well the treatment works and how safe it is for people with Cystic Fibrosis. This research could lead to new treatment options for those who have not benefited from current therapies.

The purpose of the study is to see how well BI 1291583 is tolerated by people with cystic fibrosis bronchiectasis. Participants will take the medication or placebo for 12 weeks. During this time, researchers will monitor the participants to see if they experience any side effects and how their bodies process the medication. The study will also involve the use of salbutamol sulfate, a common medication used to help open the airways in the lungs, which will be administered through inhalation.

Throughout the study, participants will have regular check-ups to ensure their safety and to gather information on how the treatment is working. The trial aims to provide valuable insights into the safety and effectiveness of BI 1291583 for treating cystic fibrosis bronchiectasis, potentially leading to better treatment options for those affected by this condition.

The purpose of the study is to evaluate how well ETD001 works and how safe it is for people with Cystic Fibrosis. Participants will use the ETD001 treatment or a placebo over a period of time, and researchers will monitor their lung function and overall health. The study is divided into two parts: the first part focuses on the safety and tolerability of the treatment, while the second part assesses its impact on lung function, specifically measuring the forced expiratory volume in one second (FEV1), which is a common test to check how well the lungs are working.

Throughout the study, participants will use the eFlow® Nebulizer System, a quiet and lightweight device that helps deliver the medication directly to the lungs. The trial aims to provide valuable information on the potential benefits of ETD001 for individuals living with Cystic Fibrosis, contributing to the development of new treatment options for this condition.

The purpose of the study is to assess how well these treatments work over a period of 8 weeks. Participants will be randomly assigned to receive either the investigational treatments or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure that the results are not biased. The study will follow a crossover design, which means that participants will have the opportunity to receive both the investigational treatments and the placebo at different times during the study.

Throughout the study, participants will have regular check-ups to monitor their health and assess the effects of the treatments. These check-ups will include measurements of lung function, sweat chloride levels, and weight, as well as questionnaires about respiratory symptoms. The study aims to provide valuable information on the potential benefits of these treatments for people with cystic fibrosis, particularly those with rare genetic mutations. The total duration of the study is expected to be 16 weeks, with participants receiving treatment for 8 weeks and then switching to the other treatment option for another 8 weeks.

The purpose of this study is to evaluate the long-term safety and effectiveness of this triple combination therapy in individuals with cystic fibrosis who are 1 year of age and older. Participants in the study will take the medication orally, meaning they will swallow the tablets, over a period of time. The study will monitor how well the treatment is tolerated by the participants and will look for any side effects or changes in health indicators such as weight, height, and lung function.

Throughout the study, researchers will collect information on various health measures, including changes in lung function and the number of hospital visits related to cystic fibrosis. The study aims to provide valuable insights into how this combination therapy can help manage cystic fibrosis over the long term, ensuring that it is safe and beneficial for those who take it.

The purpose of this research is to evaluate how safe and effective this medication combination is when used over an extended period. Participants in the study will take the medication and attend scheduled follow-up visits where various assessments will be performed. These assessments will monitor lung function, weight changes, quality of life, and the amount of salt in sweat (a marker used to evaluate cystic fibrosis severity).

The study is designed in two parts (Part A and Part B), allowing for continuous evaluation of the medication’s effects. Throughout the study, participants will maintain their regular cystic fibrosis treatments (except for other CFTR modulators, which are medications that target the underlying cause of cystic fibrosis by improving the function of the defective protein responsible for the disease).

The purpose of the study is to evaluate how well this combination of medications works over a long period and to monitor any potential side effects. Participants in the study will take the medications in the form of granules, which are taken orally. The study will last for up to 96 weeks, during which participants will regularly visit the clinic for check-ups and assessments. These assessments will include monitoring vital signs, conducting laboratory tests, and checking heart function using an ECG, which is a test that records the electrical activity of the heart.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how the treatment affects their health. The study will also measure changes in sweat chloride levels, which is an indicator of how well the treatment is working. This trial is open-label, meaning that both the participants and the researchers know which treatment is being administered. The study is intended for individuals aged 12 months and older who have Cystic Fibrosis and have previously participated in a related study.