Active clinical trials for "Cystic Fibrosis"

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.

Patients with chronic lung diseases travelling by plane often suffer with symptoms related to lower oxygen levels they are exposed to while flying. Therefore, patients with respiratory conditions are routinely assessed to establish if they need supplemental oxygen in flight. A hypoxic altitude simulation test (HAST) is often part of this assessment and consists in having patients breathe a oxygen/nitrogen blend with a lower oxygen concentration compared to normal room air, simulating in-flight conditions. Oxygen levels are measured before and after the test through a blood sample (from the earlobe or an artery in the wrist) and with a finger probe. In-flight oxygen is required if the oxygen level in the blood is lower than 6.6 kPa. HASTs are time consuming, costly, and require a dedicated hospital appointment. Using historical data, the Investigators developed scores based on capillary blood gas (blood sample from the earlobe), diagnosis and sex to predict the outcome of the HASTs. The Investigators validated the proposed scores in a separate historic cohort of patients and showed it had good concordance with the HASTs results. In this study, the Investigators want to confirm prospectively if the score, based on blood results (venous and/or earlobe), can predict the outcome of the HASTs and therefore reduce the number of tests performed, travel time for patients, and costs for the NHS. All patients, aged 18 or older, who are having a HAST for clinical purposes at the cardio-respiratory lab at Leeds Teaching Hospital NHS Trust will be invited to take part in the study. The Investigators will record diagnosis, results of HAST and previous spirometry from the medical notes, perform a spirometry if not done in the previous 12 months and collect a blood sample (one tube, 4 mls). With these data, the Investigators will calculate the score and assess its agreement with the outcome of the HAST. Each participant's involvement in the study will last for approximately 90-120 minutes, which is the normal duration of a HAST. The Investigators aim to include up to 280 subjects in the study.

In this study new hand-held devices for measuring exhaled breath will be tested in children with asthma, CF, and healthy controls. Main objectives will be feasibility and discriminative value of these techniques.

An open label non-randomized study enrolling up to 20 healthy participants and up to 30 participants with cystic fibrosis to establish a healthy versus disease comparison. Each participant will receive a mixture of inert gas (perfluoropropane (PFP)) in a ratio of 79% PFP to 21% oxygen as a contrast agent to enhance visualization of the airway and alveolar spaces using magnetic resonance imaging of inert gas/oxygen mixtures. The study consists of a screening visit followed by up to 2 study visits.

The long-term goal is to study the safety and effectiveness of hormonal contraception for women with cystic fibrosis (CF) and contribute to national guidelines that the Cystic Fibrosis Foundation and the Centers for Disease Control and Prevention (CDC) provide to clinicians. The study objectives are to determine whether hormonal contraceptive methods improve overall pulmonary health, worsen CF-related disease or CF liver disease, or are effective against unwanted pregnancy with concomitant CF transmembrane conductance regulator (CFTR) modulator use. The hypothesis is that hormonal contraceptive methods are safe and do not worsen CF-related complications over time,improve FEV-1 when compared to non-hormonal users, and oral birth control methods with CFTR modulator use.

An observational study of patients with cystic fibrosis (CF) starting treatment with Kaftrio (Elexacaftor / Tezacaftor / Ivacaftor) as part of routine clinical care, following EMA licensing (approved end of Aug 2020). Patients with CF who are p.Phe508del homozygotes will already be receiving the less effective CFTR modulator drug Symkevi (Tezacaftor / Ivacaftor) and will switch to KaftrioTM. Patients who are who are compound heterozygotes with at least 1 copy of p.Phe508del currently have access to no effective CFTR modulator and will be starting a CFTR modulator (Kaftrio) for the first time. Participants attend a study visit before Kaftrio treatment commences, followed by visits at 12 and 24 weeks after starting treatment. At each visit they will be scanned before and after standardised meals in the morning and mid-day (11 scans in total over 6 hours). No intravenous contrast or bowel preparation will be used. Participants will complete questionnaires on gastrointestinal symptoms as well as providing stool and sputum samples for assessment of microbiome and stool for inflammatory mediators and pancreatic function (elastase). **Following an extension, participants had a further visit at 76 weeks post starting Kaftrio, updated in detailed description**

The study aims to determine if a popular dextrose candy alternative yields a similar glycemic curve compared to the standard oral Dextrose solution used in the Oral Glucose Tolerance Test.

Cystic fibrosis (CF) pulmonary disease is a major cause of morbidity and mortality in CF patients and is punctuated by episodes of acute exacerbation that require antibiotic treatment. Pseudomonas aeruginosa is the predominant bacterial pathogen isolated in patients with acute exacerbations, and practice guidelines recommend combination antibiotics directed against this pathogen as initial therapy. Such therapy traditionally consists of an antipseudomonal beta-lactam with either an antipseudomonal fluoroquinolone or an aminoglycoside. With growing P. aeruginosa multi-drug resistance, more adult patients present with isolates resistant to these traditional options. The polymyxins are a class of cyclic peptide antibiotics that exert bactericidal activity through binding to the lipopolysaccharide component of gram-negative bacterial membranes and include colistin and polymyxin B (PMB). In recent years, there is growing evidence of increased rates of acute kidney injury associated with colistin in critically ill patients. Additionally, population pharmacokinetic (PK) studies suggest that fixed drug dosing may yield an improved therapeutic index over the traditional weight-based dosing of this agent. Thus there is growing interest in use of PMB as an alternative in CF acute exacerbations but the optimal dosage regimen is not known. This is a single-center, open-label, non-interventional study to characterize the pharmacokinetics and safety of fixed-dose PMB in adult patients with CF by measuring serum concentrations in patients receiving IV therapy as a part of routine care. This study will help to validate existing population PK models and allow for adjustment of patient specific covariates (i.e. weight, renal function) unique to adult patients with CF. The study will also monitor for nephrotoxicity and neurotoxicity to determine if PMB has an acceptable margin of safety in this patient population. This investigation is the first to prospectively validate the pharmacokinetics and toxicities of fixed-dose PMB in CF and will guide optimal use of this compound in the management of acute pulmonary exacerbations.

The objective of study is to prospectively determine if CPET with blood gas analysis should have a prognostic value in CF. The study plans to include 300 cystic fibrosis patients. Inclusion criteria will be: age >15 years, cystic fibrosis confirmed by chloride sweat test or genetic analysis, clinical and functional stability in the 2 month before CPET. Patients will perform a maximal exercise test on a cycloergometer during the inclusion visit, with pulmonary function testing and a six-minute walk test. The study will also include a visit every 6 months with: body mass index calculation, pulmonary function testing with DLCO (diffusing lung capacity for carbon monoxide), a six minute walk test, and antibacteriological study of sputum. The results of this study could help identify earlier the patients for referral to a lung transplantation centre, by using the usual criteria and the CPET abnormalities.

When developing new medications for lung diseases like Cystic Fibrosis (CF), scientists perform lab experiments using cells from the airways, physiology studies of how the lungs change when a drug is given, and clinical studies to determine how drugs affect overall health. The investigators of this study are seeking to develop computer models that will predict how patients will respond to drugs by just doing lab studies on cell samples from their noses. Such models would allow for medications to be developed more rapidly for all patients and allow treatments to be personalized as well. In order to develop these computer models a series of tests will be performed on patients who have CF. Tests will include sampling cells from the nose and measuring lung physiology using a combination of different imaging, breathing, and other studies performed both before and after participants take a therapy. Similar tests will be performed on people who do not have CF, and on the parents of the CF participants who carry a single CF gene because this will provide information on how specific genes might affect CF lung disease.