View clinical trials related to Cystic Fibrosis.
Filter by:This study will investigate if parental physical activity levels, assessed by providing a physical activity questionnaire to parents of children aged 6-16 with Cystic Fibrosis (CF), is associated with their child with CF's physical activity levels. Children's activity levels will be taken from electronic records where a questionnaire is routinely given at annual reviews to analyse this. Parental activity levels will also be compared against adherence to nebulisers as a proxy for adherence to treatment, this data is again in the electronic records of patients and is collected at annual reviews.
Cystic fibrosis (CF) is the UK s most common inherited genetic condition and affects more than 10,500 people. The disease causes problems with the movement of salt and water in the body, resulting in sticky mucus building up, mostly in the lungs and gut. Thick mucus in the airways leads to repeated infections which, over time, damage the lungs. Chest physiotherapy is prescribed to loosen and clear sticky thick mucus from the airways and so to help to reduce lung infection. Chest physiotherapy is a routine treatment to keep people with CF healthy. However, many say it is time-consuming and a burden. People with CF have asked if doing exercise could have the same effect as chest physiotherapy sessions for helping clear mucus. Exercise could be more enjoyable and less burdensome. Through a recognised priority setting partnership, the CF community recently ranked research to reduce the burden of their care and answer whether exercise can replace chest physiotherapy , as their number 1 and 7 priorities. Surveys show that many people with CF have occasionally chosen to replace chest physiotherapy with exercise for airway clearance, and we recently confirmed this through a UK-wide survey. It is not known if they would be willing to take part in research that asks some to stop chest physiotherapy and to exercise (with coughs and huffs) instead. New medicine (modulators) have recently become available for many people with CF, bringing dramatic improvements in their health. Some people who have started modulators are considering whether they can reduce or stop treatments - including chest physiotherapy. So, the effects of stopping chest physiotherapy need to be investigated and also if exercise can be used instead - this research study aims to understand this. A recent survey in people with CF, their families, physiotherapists and doctors, conducted by this research team, showed us that many consider hard exercise with coughs and huffs to be able to clear mucus from the airways. This study will recruit 50 people with CF (>12 years old) for 28-days. This study will ask half of them to continue their usual care, and half to stop chest physiotherapy and do exercise that gets them breathing deeply (with coughs and huffs) instead. This study will see if people are willing to start and continue with such a study and what they think of the study processes. It will also see how stopping chest physiotherapy and replacing it with exercise affects measurements of their lung function. The study will also involve talking with people with CF and members of their CF team to understand their experiences. This information will reveal whether a larger study can answer the question of whether certain forms of exercise can safely be used as an alternative to chest physiotherapy.
The aim of this study was to examine the effect of the tele-exercise program applied to children with cystic fibrosis in the Covid-19 pandemic on the quality of life and the symptoms experienced during exercise
A study to examine if the gamification of routine airway clearance can improve the quality of live for young people living with chronic health conditions.
The purpose of this study is to determine if use of a nonmydriatic fundus camera using autonomous artificial intelligence software at the point of care increases the proportion of underserved youth with diabetes screened for diabetic retinopathy.
The purpose of this study is to determine the ability of electrical impedance tomography (EIT) to identify structural and functional physiological changes that occur with disease progression in cystic fibrosis patients. The investigators also aim to determine whether EIT can serve as an alternative for CT to identify regions of air trapping and consolidation, whether EIT can provide clinically useful information about response to treatment for an acute PE, and whether EIT can provide longitudinal information about structural changes in the lung.
This is a phase 1b/2 study of a single dose of intravenous (IV) bacteriophage in males and non-pregnant females, at least 18 years old, diagnosed with Cystic Fibrosis (CF). This clinical trial is designed to assess the safety and microbiological activity of bacteriophage product WRAIR-PAM-CF1, directed at Pseudomonas aeruginosa in clinically stable CF individuals chronically colonized with P. aeruginosa. WRAIR-PAM-CF1 is a 4 component anti-pseudomonal bacteriophage mixture containing between 4 x 10^7 and 4 x 10^9 Plaque Forming Units (PFU) of bacteriophage. Enrollment will occur at up to 20 clinical sites in the United States. In stage 1, two eligible subjects will be assigned to each of the three dosing arms receiving a single dosage of the IV bacteriophage therapy (4 x 10^7 PFU, 4 x 10^8 PFU, and 4 x 10^9 PFU; total of 6 sentinel subjects), followed by 30 ± 7 days observation period. If no SAEs (related to the study product) are identified during the 96 hours after bacteriophage administration for all Sentinel Subjects in Stage 1, the study will proceed to Stage 2. In Stage 2a, 32 subjects will be enrolled into one of 4 arms (placebo IV, 4 x 10^7 PFU, 4 x 10^8 PFU, and 4 x 10^9 PFU) in a 1:1:1:1 allocation. An interim analysis will be performed after all subjects have completed follow up visit 7 on Day 30 to select the IV bacteriophage dose with the most favorable safety and microbiological activity profile. During Stage 2b, subjects will be randomized into the bacteriophage (dose selected based on Interim Analysis following Stage 2a) or placebo arm. The final sample size is expected to be up to 72 subjects total with up to 25 subjects in the placebo arm and up to 25 subjects in the Stage 2b bacteriophage dose.
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.
The purpose of this study is to evaluate the pharmacokinetics (PK) and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with moderate hepatic impairment and in matched healthy participants.
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.