View clinical trials related to Cystic Fibrosis.
Filter by:Specific aims: 1. To elucidate the biological mechanism that leads to pulmonary and nutritional improvement in CF patients following treatment with ivacaftor using advanced techniques to assess changes of the pulmonary and nutritional status 2. To examine the relation between the individual response to ivacaftor and the presence of modifier genes associated with CF disease severity, 3. To assess altered CFTR function using new available in vivo tests, 4. To validate newly developed in vivo sweat tests with well established functional tests, 5. To establish correlation between the CFTR response to Vx-770 measured in a new ex vivo method (organoids) and the actual clinical and/or functional response in individual patients, 6. To examine response in other CF-specific features such as aqua wrinkling. 7. To examine if sleep/activity level changes. 8. To establish a biorepository to enable further investigations.
Very low birth weight infants who are at risk for chronic lung diseases may also be at risk for brain anomalies such as increased echogenicity, leukomalacia and intracranial hemorrhage. Infants with bronchopulmonary dysplasia have been reported to have worse neurodevelopmental outcomes than healthy infants. It has also been pointed out that babies with prolonged and recurrent apneas during sleep may have weak General Movements (GMs) repertoire. It has been mentioned that motor development retardation may also occur in neurodevelopmental diseases, genetic diseases and chronic lung diseases, as well as in cystic fibrosis. In infants with cystic fibrosis, motor development may be affected by increased incidence of hospitalization, previous infections, malnutrition, respiratory and digestive system disorders. There is no research done with GMs assessment to determine motor dysfunction in infants with cystic fibrosis and this topic is open to research. Having more information about the motor development of babies by determining the motor characteristics and motor performance of infants with cystic fibrosis, it may be possible to start the disease-specific physiotherapy and rehabilitation programs as early as possible. For this reasons, in the study the investigators aimed to investigate the characteristics of GMs in the "Fidgety" period of 3-5 month term infants diagnosed with cystic fibrosis, to determine the motor performances and to investigate the relation between the GMs characteristics and the features of the disease. The hypotheses the investigators have set for this study are listed below; Ho: Spontaneous movements of the "Fidgety" period of infants diagnosed with 3-5 months of cystic fibrosis are not different from normal infants. H1: Spontaneous movements of "Fidgety" period of infants diagnosed with cystic fibrosis between 3-5 months are different from normal infants.
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.
It is extensively reported in the literature that patients with chronic obstructive lung disease may have impairments in balance and postural control which further increase the disease burden. Mechanisms related to these impairments include, but are not limited to increased work of breathing, diaphragm weakness, peripheral muscle weakness and systemic inflammation. Since the similar symptoms are reported for the children with cystic fibrosis, it is hypothesized that balance and postural control may also be compromised in these patients. Inspiratory muscle training (IMT) is shown to improve diaphragm strength and pulmonary function. Considering the relation between diaphragm which is one of the core muscles, and balance, IMT may also have an impact on postural control and balance alongside the standard clinical parameters such as respiratory muscle strength, pulmonary function and functional capacity in these patients. Thus, the aim of this study was to investigate the effects of inspiratory muscle training and conventional chest physiotherapy on postural stability, balance, pulmonary function and functional capacity in children with cystic fibrosis.
This Phase 1/2, first-in-human study will evaluate the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract of adult subjects with CF.
Cystic Fibrosis (CF) is a hereditary multi-system disease affecting approximately 30,000n children and adults in the USA. The diagnosis of CF requires biochemical confirmation (either abnormal sweat chloride measurement and/or identification of two CF disease causing mutations) plus clinical symptomatology. Measurements of sweat chloride remain cumbersome and although most common methodology to confirm CF diagnosis with limitations especially in young children less than 6 months of age and in areas that lack ability for the complex testing. The study objectives of this current research proposal include: A) To expand upon previously obtained pilot study data "Evaluation of a fluorescent-based chloride sensor as an optical sweat test to diagnose cystic fibrosis" B) To add the exploratory measurement of sweat Bromide as a first in human assessment observation, C) To Evaluate the development of smartphone based point-of-care technology for chloride and bromide sensor measurements, D) To further expand the class of citrate-based sensors with improved fluorescence and sensing properties for the design of new fluorescence-based analytical and diagnostic solutions based on the automated multi-halide detection system, and E) To develop point-of-care systems that can successfully integrate into clinical settings to improve current practices and facilitate early detection of disease.
Pulmonary transplantation is the reference treatment for chronic terminal respiratory failure in patients with cystic fibrosis. These are mainly bi-pulmonary transplants (cardiopulmonary transplants are exceptional). The annual number of pulmonary transplants in France for cystic fibrosis is about 90. In 2013, the transplant involves a total of more than 600 patients with cystic fibrosis. The average age at the time of the transplant is 28.5 years (2013 data, French cystic fibrosis register), compared to 58 years for patients transplanted to all pathologies. Cystic fibrosis accounts for 25% of adult bi-pulmonary grafts. Pediatric transplants are currently very rare. The median survival after pulmonary transplantation in cystic fibrosis is currently 8.5 years (and 10 years when considering patients surviving 3 months, ie excluding early mortality). Cystic fibrosis is the pathology associated with better survival after pulmonary transplantation given the young age of patients (28.5 years on average). The non-respiratory comorbidities associated with transplantation, all underlying pathologies combined, and referenced in the Registry of the International Society for Heart and Lung Transplantation (ISHLT) are: hypertension, diabetes, renal insufficiency, Dyslipidemia, cancers. Their frequency increases with the survival time of transplanted patients. Cystic fibrosis is associated with non-respiratory comorbidities, the frequency of which increases with age - diabetes, osteoporosis, renal insufficiency, hepatopathy, neoplastic pathologies - and may become worse after transplantation. The main objective is to estimate the incidence of non-respiratory co-morbidities after lung transplantation in the cohort of patients with cystic fibrosis grafted in the Rhône-Alpes region.
The aim of the study is to assess the diagnostic sensitivity of MRI to detect changes in Helbich-Bhalla scoring over time in patients with cystic fibrosis
This is a multicenter, cross-sectional, cohort study which will collect contemporary sweat chloride (SC) values from approximately 5000 Cystic Fibrosis (CF) patients prescribed and currently receiving commercially approved Cystic Fibrosis transmembrane conductance regulator (CFTR) modulator therapies.
To assess whether the inhalative combination of Tobramycin/Colistin is more effective in reducing Pseudomonas colony forming units (CFUs) and improvement of lung function than Colistin in mono-therapy.