View clinical trials related to Cystic Fibrosis.
Filter by:The study aims to evaluate MuCopilot, a smartphone application to measure objective data on lung function (cough and dyspnea tests), global exercise capacity (walking test) and patient-reported outcomes (PROs) of patients with Cystic Fibrosis (CF). These data are collected during unsupervised digital tests performed in the patient's home environment between consultations. The primary objective is to demonstrate the correlation of the MuCopilot digital tests at home (D1) with the results of their standard counterparts (D0) carried out in-clinic. A secondary objective is to determine the accuracy, reliability and reproducibility of tests results, as well as to study the test-retest of the PRO. The study also aims to assess the safety, usability, and satisfaction of the solution. Exploratory objectives include evaluating the relationship between MuCopilot's scores and other standards such as FVC, FEV1/FVC ratio as well as to explore the correlation with the cough monitoring with patient's perception. Patients will be able to download MuCopilot app. They will participate in 1 inclusion visit and 7 follow-up visits, scheduled at Day 1, Day 3, Day 5, Day 7, 1 month, 2 months, 3 months - 1 day. The study will include 70 CF patients and will be conducted in France.
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnosis for single-gene disorders (SGD-NIPD). This can be performed from 9 weeks of amenorrhea and offers an early, safe and accurate definitive diagnosis without the miscarriage risk associated with invasive procedures. One of the major difficulties is distinguishing fetal genotype in the high background of maternal cfDNA, which leads to several technical and analytical challenges. Besides, unlike noninvasive prenatal testing for aneuploidy, NIPD for monogenic diseases represent a smaller market opportunity, and many cases must be provided on a bespoke, patient- or disease-specific basis. As a result, implementation of SGD-NIPD remained sparse, with most testing being delivered in a research setting. The present project aims to take advantage of the unique French collaborative network to make SGD-NIPD possible for theoretically any monogenic disorder and any family.
The study aims to assess the usability and safety of use of MuCopilot, a smartphone application that measures objective data on lung function, global exercise capacity and patient reported outcomes of patients with Cystic Fibrosis (CF). These data are collected during unsupervised digital tests performed in the patient's home environment between consultations. The primary objective is to validate the usability and safety of use, in order to assure that the patients use the medical device as intended without any unacceptable error of use and without unacceptable risk. The study will include 17 CF patients and will be conducted in France. They will participate in 1 inclusion visit and 1 visit in-clinic (1h30). Patients will be able to download the free MuCopilot mobile application. During the visit, patients will complete 3 digital tests in order to monitor CF functions (cough, dyspnea & walking) and 1 symptom questionnaire.
Cystic Fibrosis (CF) affects more than one body system, mainly respiratory and digestive.The quality of life of individuals with CF is adversely affected by the increasing treatment burden in addition to multi-system involvement. The International Classification of Functioning, Disability and Health (ICF) describes human functioning and states of disability and provides a framework for organizing this information. ICF Core Sets are created by selecting the appropriate categories for the current disease from the ICF classification. ICF Core Sets are smaller than ICF, allowing practical evaluation based on ICF in clinical use. It evaluates the current situation of the patient quickly and practically, and improves interdisciplinary cooperation. Our study, it was aimed to develop a core set that quickly and practically evaluates the current state of the patient with CF in Turkey, based on ICF, and increases coordination within the interdisciplinary team. In our study, the established, very comprehensive, scientific and evidence-based guide that should be followed while creating the ICF core set will be followed. The guide consists of 4 stages: The first stage is the literature review which allows us to see the disease from the perspective of the researcher by scanning the studies on CF in the last 10 years. The second stage is the patient interview, which includes interviewing these individuals, and allows us to see the disease from the perspective of individuals diagnosed with CF. The third phase is the expert questionnaire, which we will look at from the perspective of health professionals who are knowledgeable about treating individuals with CF. The fourth stage is the consensus stage, where the final core set is decided, which includes team discussion. With the final core set developed as a result of these stages, a core set that will quickly and practically evaluate the current situation of the patient with CF in Turkey based on ICF and increase coordination within the interdisciplinary team will be ensured. Hypothesis(s) and purpose(s) on which this thesis proposal is based: H1: The ICF Core Set will be instrumental in understanding CF-specific health, disability, and function. H2: It will be a step in the development of a standard tool for the assessment of adults with CF. In our study, we propose to examine CF from the perspective of the researcher as a result of the literature review, from the perspective of the patient as a result of the qualitative research, from the perspective of the health professional as a result of the expert survey, to create a core set specific to these patients, and thus to see the different characteristics of individuals with CF in terms of function, activity, participation, and environmental factors.
Dynamic chest X-ray (DCR) is a novel radiographic technique that enables us to visualise the movement of the chest and lungs in real-time by recording a series of x-ray images over 10-20 second interval. DCR provides an assessment of structures involved in respiration over a time period and could therefore provide us with insights into respiratory function. It may be possible that relating DCR to conventional breathing tests allows it to be used as a surrogate tool to measure lung function. Alternatively, it may provide different parameters of lung function which don't directly correlate with conventional testing, but are of themselves clinically relevant. Cystic fibrosis (CF) is a genetic condition that results in the body producing thick , dehydrated secretions. It is usually characterised by a cycle of recurring lung infections and inflammation resulting in progressive lung damage. A key clinical investigation for people with cystic fibrosis (pwCF), and other chest conditions, is a spirometry breathing test. Despite spirometry's fundamental role in chest medicine it can be poorly tolerated. In order to evaluate the utility of DCR as a tool for assessing lung function, it is important to be able to compare it to currently available pulmonary function tests such as spirometry. Previous studies have compared sequential lung function testing to DCR images; however, there may be significant variability in 2 separate manoeuvres as they are effort and technique dependent (i.e. when performing a DCR followed by spirometry or vice versa). Currently, there have been no studies that compare DCR with a simultaneous lung function test such as spirometry. This observational, prospective, non-randomised, single centre , pilot study aims to address this issue by performing simultaneous spirometry and DCR to establish how DCR compares with spirometry in a healthy population of individuals. The investigators would seek to also perform the DCR with simultaneous spirometry in a cohort of pwCF, allowing for comparison between the two groups. Furthermore DCR imaging has been part of the annual review process for pwCF at Liverpool Heart and Chest Hospital since 2019. The study investigators therefore intend to review how changes in DCR metrics over time relate to changes in other key clinical markers of pwCF, such as spirometry. A questionnaire on patient experiences of DCR will be provided to study participants to be filled in after imaging acquisition.
The goal of this clinical trial is to investigate the effects of yoga participation for adults with cystic fibrosis. The main question it aims to answer is: does participation in yoga affect health-related quality of life for adults with cystic fibrosis? Researchers will compare a group completing a 12-week yoga programme alongside usual cystic fibrosis care, to a group completing usual care alone to see if the addition of yoga effects health-related quality of life.
This study capitalizes on the emerging technology of 19F MRI, using conventional 'thermally' polarized perfluorinated gas (perfluoropropane, or PFP) mixed with oxygen and studied with magnetic resonance imaging (MRI) to visualize ventilation. This technique has not been studied in children. Children and adolescents (6-17 years old) with cystic fibrosis (CF) who have normal spirometry will undergo 19F MRI with the inhalation of an inert contrast gas to study ventilation. Comparisons will be made to a cohort of healthy children (6-17 years old) who will perform the same measures. The primary outcome measure is the feasibility of conducting these studies in the pediatric population. Parallel performance of multiple breath nitrogen washout (MBW) and spirometry will be used to compare the sensitivity of these outcomes to the presence of mild lung disease in these children. Finally, the investigators will compare data obtained during standard breath holds with a novel "free-breathing" technique that will eliminate the need for breath holds during MRI acquisition.
The aim of this observational study is to better understand the role and involvement of the regulatory elements of the CFTR gene, with the aim of better describing the 3D organisation of chromatin at the CFTR locus in epididymal cells in patients with male infertility of any kind, or with cystic fibrosis or bilateral agenesis of the vas deferens, requiring scheduled surgery. The main questions it aims to answer are: - to better characterise this 3D organisation of the CFTR locus, the study of regulatory elements in primary epididymal cells is the most relevant and realistic model. - to gain a better understanding of the regulation of the CFTR gene in epididymal cells in order to gain a better understanding of the pathology of male infertility caused by bilateral agenesis of the vas deferens, a symptom and also a borderline form of cystic fibrosis. Participants will Epididymal samples will be taken by a urologist for the AMP department during the planned surgery. The rest of the samples taken will be recovered for research purposes, with the aim of recovering the epididymal cells contained in the sample. This is in no way an additional procedure and will have no impact on the patient's health..
The goal of this clinical trial is to assess the feasibility of ceftolozane/tazobactam (C/T) administered on an outpatient parenteral antibiotic therapy programme to patients with a current infective exacerbation of bronchiectasis or cystic fibrosis related to pseudomonas aeruginosa or burkholderia cepacia spp. organisms. The main question[s] it aims to answer are: - Is C/T effective, safe, well-tolerated and able to induce clinical and microbiologic response? - What are mechanisms of antimicrobial resistance are induced by administration of C/T?
The goal of the clinical trial is to test whether a mental health program that is delivered through the Internet works well for children and adolescents with cystic fibrosis (CF) and their healthy siblings. The main questions it aims to answer are: - Does the program improve the mental health such as depression and anxiety symptoms? - Does the program improve overall quality of life? - Does the program improve self-efficacy - an individual's belief in their ability to complete tasks to achieve their goals? Participants will: - Fill out an online survey asking questions about their personal and health information, as well as their mental health before the program - Complete the online mental health program - Fill out an online survey asking questions about their mental health after completing the program, and 1-month and 3-months following completing the program Participants be compared against another group of children with CF and their healthy siblings who are on a waitlist and receiving usual CF treatment. Researchers will compare participants scores before starting the program with their scores immediately following completing the program, 1-month, and 3-month after completing the program. Researchers hope to develop a program that improves mental health, quality of life, self-efficacy, and knowledge about CF.