View clinical trials related to Cystic Fibrosis.
Filter by:Portal hypertension (PHT) and its sequelae are the most clinically important manifestation in cystic fibrosis related liver disease (CFLD), with end-stage liver failure as a late and rare manifestation. The aim is to evaluate the safety and efficacy of a pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) for the prophylaxis of variceal bleeding in pediatric CFLD patients with subclinical non-cirrhotic portal hypertension (NCPH)
The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).
The main objective of this study is to assess the ovarian reserve of CF patients via measurement of AMH and AFC. Secondly, we aim to correlate between the ovarian reserve and demographic and clinical characteristics of these patients. Outcomes measured will be levels of AMH and AFC, as compared to standard means in the general population. There is no need for a control group in this study since the standardized means of AMH and AFC in the general population are a more accurate comparable measure, which is based on data collected from large-scale populations, and thus accounts for confounding age factor in a more complete manner than can be obtained via a control group (Almog et al., 2011, La Marca et al., 2012, Penzias et al., 2020).
Cystic fibrosis (CF) is a genetic disease that affects many organs and systems, especially respiratory system problems due to lung damage. Patients often have difficulty in removing the sticky and viscous secretion that accumulates in the respiratory tract, and the risk of mortality increases with the development of respiratory failure. In patients with CF, exercise capacity, peripheral muscle strength, core endurance, flexibility, postural stability, physical activity level, and quality of life also decrease secondarily. Recently published guidelines recommend respiratory physiotherapy for coping with CF-related symptoms and recommend referral of patients to physical activity and exercise. Hippotherapy simulator is a mechanical exercise tool that imitates the walking movement of a real horse and is used to increase physical fitness parameters. This study aims to show the effects of exercises performed with a hippotherapy simulator in addition to respiratory physiotherapy on physical fitness, sputum production, physical activity and quality of life of children with CF.
Cystic fibrosis is a genetic disease (autosomal recessive) which involves malfunction of the exocrine glands, leading to abnormal secretions in the body. It is a progressive disease that causes persistent lung infections and limits the ability to breathe over time. Clinical symptoms include persistent coughing, at times with phlegm, wheezing or shortness of breath, fatigue, difficulty with bowel movements sinus infections, poor growth, clubbing of the fingers and toes, and infertility in most males. The disease must be managed throughout life with diet, medication and preventive chest physical therapy as soon as any symptoms are noted in the young child. The purpose of the study was to evaluate the difference between the effects of Manual Chest Physiotherapy (CPT) and Active Cycle of Breathing Techniques (ACBT) in patients of Cystic Fibrosis. The tools of our study were Modified Borg Dyspnea Scale and Quality of well-being Scale. The total sample of our study was 14 out of which 7 were included in GROUP A and 7 Group B. SPSS 23 was used for statistical analysis and parametric tests were used for analysis
Coronavirus disease 2019 (COVID-19) which is caused by the virus Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2) has resulted in an ongoing global pandemic. It is unclear whether the relatively low number of reported cases of COVID-19 in people with CF (pwCF) is due to enhanced infection prevention practices or whether pwCF have protective genetic/immune factors. This study aims to prospectively assess the proportion of pwCF, including both adults and children with CF who have evidence of SARS-CoV-2 antibodies over a two-year period. This study will also examine whether pwCF who have antibodies for SARS-CoV-2 have a different clinical presentation and what impact this has on their CF disease. The proposed study will recruit pwCF from paediatric and adult CF centres throughout the United Kingdom. Serological testing to detect antibodies will be performed on blood samples taken at month 0, 6, 12, 18 and 24 with additional time-points if bloodwork is available via normal clinical care. Clinical data on, lung function, CF-related medical history, pulmonary exacerbations, antibiotic use, and microbiology and vaccination receipt, will be collected during routine clinical assessments. Associations will be examined between socio-demographic and clinical variables and serologic testing. The investigators will also examine the effects of SARS-CoV-2 infection on clinical outcomes and analyse end-points to explore any age-related or gender-based differences, as well as subgroup analysis of outcomes in lung-transplant recipients and pwCF receiving cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies. As pwCF receive COVID-19 vaccination the investigators will perform a comparison of the development and progression of anti-SARS-CoV-2 antibodies in pwCF following natural infection and vaccination SARS-CoV-2 over time.
This is a Phase 1b/2a study with the primary objective to determine if BX004-A is safe and tolerable. Exploratory objectives include whether BX004-A reduces sputum Pseudomonas aeruginosa (PsA) bacterial load in CF subjects with chronic PsA pulmonary infection.
With this retrospective observational cohort study, we performed a longitudinal assessment of the renal function of the adult CF patients who underwent LUTX, aiming 1) to describe possible risk factors associated with perioperative AKI and 2) to describe AKI short and long term effects on clinical outcomes.
This is a Patient-Centered Outcomes Research Institute engagement effort aimed at training researchers/providers and patients to work in research teams together online throughout the research process (including: development, design, and dissemination) to address critical gaps in their care. This is a change from the typical research done with people with CF as they are frequently isolated from other members of the CF community because of infection control guidelines that restrict in-person contact to avoid the spread of bacteria between patients. This project has four aims: 1. build capacity for PCOR knowledge and skills applicable for longitudinal online engagement, 2. create and disseminate a best practices PCOR user guide for populations that solely engage online, 3. to create an interactive web-based version of our User Guide through a survey and three modified Delphi rounds, and 4. to create a comprehensive training manual for conducting PCOR online (step-by-step instructions), which will incorporate the aforementioned user guide.
In this observational study, the investigators evaluate the sensitivity of T1-MRI to identify lung perfusion changes in pediatric patients with CF (age = 6-11) before and after initiating FDA-approved Trikafta therapy. The investigators compare these Lung T1 MRI assessments (% Normal lung perfusion) to currently best-available assessments of lung function in CF patients (i.e., MBW (LCI( and Spirometry (FEV1 % Predicted).