View clinical trials related to Cystic Fibrosis.
Filter by:In this project the investigators aim to identify new biological markers by characterizing the response/inflammation associated with the development and progression of M. abscessus lung disease in patients suffering from cystic fibrosis with the aim of increasing current knowledge available on the development and progression of lung disease.
Chronic constipation is a feature of children with cystic fibrosis (CF). This is postulated to be a result of inhibition of secretory activity of the gastrointestinal luminal cells due to ineffective chloride channel function. Typical laxatives that work as osmotic agents fail to produce adequate relief in this population. Maralixibat is a non-systemic bile acid transport inhibitor (IBATi) that acts by interrupting bile acid reabsorption in the ileum thus interrupting the normal enterohepatic circulation. This interruption results in a larger volume of bile acids reaching the colon and being excreted in stool. Bile acids are known to decrease bowel transit time, increase mucosal permeability and secretions, as well as alter gut microbiota resulting in diarrhea. The overarching hypothesis of the study is that Maralixibat will improve stool consistency in children (Age <18 years) with cystic fibrosis and constipation (Bristol Stool Scale <4). Specifically, we aim to test the hypothesis that IBATi improves the consistency of stool to Bristol scale >4 in children with CF and constipation. We will recruit a total of 20 patients with CF and constipation (defined as Bristol Stool Scale <4 for 1 week prior to enrollment while on a stable laxative regimen for at least 4 weeks.) Design is a 'Within-Subjects' study by which each enrolled patient will take Maralixibat for 2 weeks total in addition to their stable laxative regimen during the study. Stool consistency & ease of defecation will be recorded before and during the study period by families of enrolled patients via materials provided by the investigators. Stool consistency and ease of defecation will be compared before and after initiation of Maralixibat. The primary endpoint: Improvement in stool consistency to Bristol scale >4 in children with CF and constipation. The secondary endpoint: Improvement in ease of defecation in children with CF and constipation. This will be measured via survey using a standardized scale (Bristol Stool Scale) and questionnaires developed by the research team. Analysis will involve comparison of pre-intervention to post-intervention stool consistency & survey
The main objective of the study is to assess the potential of time-frequency representation and analysis of pulmonary sounds collected with an electronic stethoscope, as part of the routine monitoring of patients with cystic fibrosis, COPD or pulmonary fibrosis.
The pathogenicity of Achromobacter bacteria is not yet well established, but studies show a decline in respiratory function and an increase in mortality associated with chronic colonisation, making it possible to classify the Achromobacter genus as an emerging pathogen in cystic fibrosis. It is possible that certain species or clones are more virulent or resistant, requiring the adaptation of measures to prevent cross-transmission in the centres concerned. However, until now, the identification of Achromobacter species has involved the use of molecular biology techniques that are not routinely applicable in diagnostic laboratories, limiting studies and the collection of epidemiological data. Recently, a database using MALDI-TOF mass spectrometry has been built for rapid and accurate species identification. In view of the local epidemiology and the current lack of data, it would be necessary and interesting to use this tool to study a cohort of cystic fibrosis patients in Réunion island (North and South sites) to see whether one species has a greater clinical impact than another (pathogenicity), and/or is more responsible for chronic colonisation.
Cystic fibrosis (CF) is a rare disease affecting one out of 4,500 newborns in France (INSERM 2021). Despite major advances in patient care over the past two decades, with significant improvements in life expectancy, cystic fibrosis remains a pathology that considerably impairs quality of life. Several studies have reported the possibility of respiratory and non-respiratory sleep disorders (SD) in patients with CF. Respiratory disorders are reported to affect 30% of children with CF (Barbosa 2020). Among non-respiratory SD, sleep onset and maintenance insomnia are well known in these patients, while chronotype abnormalities (circadian rhythm disorders) are understudied. Chronotype refers to a person's tendency to be more efficient in the morning or evening. The existence of chronotype abnormalities has been suggested in CF patients, but no precise data are available (Louis 2022). The involvement of CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) protein dysfunction in the central nervous system (CNS) has been hypothesized as a contributory factor. In vivo, in a mouse model of CF, dysregulation of clock genes such as Clock, Cry2 and Per2 was found in the CNS (Barbato 2019). Among them, certain genes such as Rev-erbĪ± could regulate endobronchial inflammation and contribute to the severity of respiratory pathology. All in all, chronotype abnormalities could be at the origin of sleep debt, impaired cognitive functions or metabolic disturbances. In the era of highly effective modulator therapy (HEMT) for the treatment of CF, the impact of these new therapies on chronotype has been understudied. Assuming that chronotype abnormalities are a direct consequence of CFTR protein dysfunction in the retina and anterior hypothalamus, HEMT should improve sleep quality. However, between 20% and 30% of adult and pediatric patients express an increase in chronotype abnormalities following initiation of treatment. Paradoxically, the perceived gain in respiratory quality of life is counterbalanced by the occurrence of these disorders. Some patients would effectively reverse their treatment in order to limit the phenomenon. A single polysomnographic study evaluated the effect of HEMT Kaftrio-Kalydeco on sleep in adults with CF (Welsner 2022). After 3 months of treatment, patients had a significant reduction in respiratory events, with no change in total sleep time, sleep efficiency or sleep architecture. Chronotype was not mentioned. Currently, no studies on chronotype in children or adults with CF have been carried out. Our hypothesis is that CF patients treated with HEMT would develop an abnormal chronotype of late sleep onset. The aim of this study is to evaluate the chronotype of children with CF treated with HEMT. Chronotype abnormalities could have major consequences for quality of life, the immune system, cognitive functions and metabolism. Systematic detection of these disorders via anamnesis, followed by diagnosis by questionnaire, actimetrics and/or urinary melatonin dosage, would enable their early management, starting with the reversal of Kaftrio-Kalydeco intake between morning and evening.
The goal of this clinical trial is to test use of losartan in those with cystic fibrosis (CF) on modulator therapy. The main question it aims to answer is if treatment with losartan improves response of the CF transmembrane conductance regulator (CFTR) channel to modulator therapy. Participants will be asked take losartan or placebo for twelve weeks and will have changes in sweat chloride levels measured as a marker of CFTR function.
Cystic Fibrosis (CF) is a rare hereditary disease with autosomal recessive transmission, affecting 1 in 4700 births in France. Numerous studies have explored the links between oral health and CF, predominantly focusing on a children population. These studies reveal hyposalivation, a risk of dental erosion, an increased prevalence of enamel structural defects, but a reduced prevalence of dental caries in CF children, potentially explained by better oral hygiene. Periodontal disease does not appear to be increased in this population, while the oral quality of life of CF patients has been insufficiently studied. Today, emerging challenges arise due to the increased life expectancy of CF patients, attributed to the rise of modulators such as Kaftrio®, resulting in an adult-majority population in France. The study of periodontal diseases, associated with oral dysbiosis, becomes relevant as they represent bacterial reservoirs that could impact respiratory complications in CF patients. To deepen understanding of the links between oral health and CF, as well as to improve oral health of these patients, it is crucial to update the specific oral profile of this population. A cross-sectional survey using a questionnaire is proposed to include a large number of CF patients in France, aiming for real-life data. This questionnaire is constructed around internationally recognized tools for comparative analysis with normative data. Collaboration with the Patients Association "Vaincre la Mucoviscidose" (VLM) facilitates questionnaire creation, dissemination, and interpretation of results.
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating cystic fibrosis (CF), it is still largely unknown whether or not other chronic therapies can be safely stopped. This SIMPLIFY sub-study is being done to test whether or not it is safe to stop taking dornase alfa (Dnase) in those people that are also taking elexacaftor/tezacaftor/ivacaftor (ETI). ETI is a combination CFTR modulator therapy that was approved by the Food and Drug Administration for people with CF who have at least one F508del mutation. The three drugs that make up ETI work together to allow many more chloride ions to move into and out of the cells, improving the balance of salt and water in the lungs. These changes result in better clearance of mucus from the lungs and improvements in lung function. Dornase alfa (Dnase) also improves clearance of mucus from the lungs to support lung function and has been available to people with CF for many years. Dnase is considered to be relatively burdensome and it is not known whether Dnase can improve or maintain lung function above what is already gained through ETI use. The goal of this SIMPLIFY sub-study is to get information about whether or not it is safe to stop Dnase by testing if there is a change in lung function in participants with cystic fibrosis (CF) who are assigned to stop taking Dnase as compared to those who are assigned to keep taking Dnase while continuing to take ETI. This is a sub study of master protocol SIMPLIFY-IP-19, NCT04378153. The sub study investigating the impact of discontinuing and continuing hypertonic saline is registered under NCTXXXXXXX (will add once available).
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating cystic fibrosis (CF), it is still largely unknown whether or not other chronic therapies can be safely stopped. This SIMPLIFY sub-study is being done to test whether or not it is safe to stop taking inhaled hypertonic saline in those people that are also taking elexacaftor/tezacaftor/ivacaftor (ETI). ETI is a combination CFTR modulator therapy that was approved by the Food and Drug Administration for people with CF who have at least one F508del mutation. The three drugs that make up ETI work together to allow many more chloride ions to move into and out of the cells, improving the balance of salt and water in the lungs. These changes result in better clearance of mucus from the lungs and improvements in lung function. Inhaled hypertonic saline (HS) also improves clearance of mucus from the lungs to support lung function and has been available to people with CF for many years. HS is considered to be relatively burdensome and it is not known whether HS can improve or maintain lung function above what is already gained through ETI use. The goal of this SIMPLIFY sub-study is to get information about whether or not it is safe to stop hypertonic saline by testing if there is a change in lung function in participants with cystic fibrosis (CF) who are assigned to stop taking HS as compared to those who are assigned to keep taking HS while continuing to take ETI. This is a sub study of master protocol SIMPLIFY-IP-19, NCT04378153. The sub study investigating the impact of discontinuing and continuing dornase alfa is registered under NCTXXXXXXX (will add once available).
The goal of this study is to determine the extent to which excess dietary simple sugars serve as a secondary mediating factor in Cystic fibrosis-related diabetes (CFRD) development. The main questions it aims to answer are: - Whether conducting a randomized 2x2 factorial design that evaluates acute postprandial changes in glucose over 2 hours following ingestion of a mixed meal challenge that varies by glycemic index and consumption of a sugar-sweetened beverage is acceptable and feasible. - What are the preliminary changes in postprandial hyperglycemia, islet cell function, and incretin response to a high or low Glycemic Index mixed meal tolerance test (MMTT) with and without Sugar-Sweetened Beverages (SSB) in adolescents and young adults with CF Participants will be randomized to a mixed diet and blood will be drawn before and after the mixed meal challenge.