View clinical trials related to Cystic Fibrosis.
Filter by:Whole genome sequencing of Korean patients with idiopathic bronchiectasis and their family will perform to identify disease-causing variants.
This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF).
Parents of children with cystic fibrosis (CF) experience high levels of stress and problems. Family empowerment interventions provide important support for many patients with CF and their families. However,there is a lack of research exploring the effects of such empowerment programs on coping, and Problem Solving among parents, and Quality of Life in Children with CF. The present study aimed to assess the effect of a family empowerment program-based nursing intervention on parents coping with stress and problem solving, and quality of life children with CF.
The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000. This pilot study evaluated subject satisfaction with the therapy and adherence to the therapy during the 90-day treatment period, and also collected clinical outcome data. Outcomes were assessed before, during and after the MN4000 treatment period.
The purpose of this observational research study is to determine the effects of clinically prescribed Orkambi treatment on 2 to 5 year old children homozygous for the F508del Mutations in the Cystic fibrosis transmembrane conductance regulator (CFTR) gene on sleeping energy expenditure, growth status and gut health and function.
The purpose of this research study is to determine the effects of clinically prescribed ivacaftor treatment on 4-24 month old children with CF and gating mutations on sleeping energy expenditure, growth status and gut health and function.
Cystic Fibrosis (CF) is a lifelong condition which causes the lungs and digestive system to become clogged with thick, sticky mucus. This leads to recurrent chest infections and reduced nutrient absorption from food. The average age at death is 31 years, usually from respiratory failure. The nutritional status of people with CF (PWCF) is important to help them live healthier and longer. It is recommended that adults with CF achieve a BMI of 23 for males and 22 for females. However, fewer than 50% of adults with CF achieved that target BMI despite effective nutritional support to help weight gain. There is a clear need for a behavioural intervention that can help PWCF use the available nutritional support. This is a feasibility study to try out a multi-component behavioural intervention supported by a nutrition app. The intervention is designed to help PWCF use their nutritional support to gain weight. It will focus on testing the methods and procedures to be used on a larger scale, improving the behavioural intervention and estimating how many people are needed for the larger trial. Eligible patients will be invited to participate, and the investigators anticipate recruiting 6 participants. The participants will have 6 weeks of intensive intervention, which will include a mix of clinic visits, home visits and telephone calls followed by 6 weeks of maintenance phase. Data will be collected during clinic visits at baseline, week 6 and week 12 along with weight monitoring at home. The investigators will interview each participant at the end of the study period to improve the intervention and study processes based on participant feedback. The investigators hypothesised that using a nutrition app, along with regular input from a dietitian, will help PWCF to use the available nutritional support.
This is an open-label, single center, prospective study of patients with a diagnosis of cystic fibrosis (CF) and a history of methicillin-resistant Staphylococcus aureus (MRSA), also known as oxacillin-resistant Staphylococcus aureus (ORSA), being treated with intravenous ceftaroline. Patients will have intravenous ceftaroline dosed according to current recommendations based upon its approved usage in pediatric patients for community acquired pneumonia. The investigators will analyze pharmacokinetic (PK) and pharmacodynamic (PD) data using serum concentrations of ceftaroline measured at various time-points following infusion of ceftaroline to develop PK/PD guided dosing strategies of ceftaroline in pediatric CF patients that would be tailored to account for bacterial susceptibility and disease progression.
The purpose of this study is to evaluate safety and tolerability of VX-121 in healthy subjects and in subjects with cystic fibrosis (CF).
Although some studies have brought some evidences about the efficacy of positive expiratory pressure (PEP)-mask therapy as an airway clearance technique, yet it is not clearly understood what is the contribution of this technique in modifying peripheral ventilation inhomogeneity, a typical feature of patients with Cystic Fibrosis (CF). The aim of this study is to investigate how PEP-MASK affects ventilation inhomogeneity in children and adolescents, with moderate to normal CF lung disease by the change in acinar airways (Sacin), lung clearance index (LCI) and conductive airways (Scond) indexes derived from nitrogen multiple-breath washout test (N2MBW).