Active clinical trials for "Cystic Fibrosis"

The purpose of this study is to assess the agreement of clinical performance between the proposed NeoPlex 4 assay and NeoPlex System and the comparator devices in clinical use in newborn screening programs for detection of T4, TSH, 17-OHP and IRT.

Shortness of breath (dyspnea) during exercise is a major source of distress and is a commonly reported symptom in patients with cystic fibrosis (CF). A recent treatment option known as Orkambi, which combines the drugs lumacaftor and ivacaftor, may be used in patients with CF to help improve lung health. However, the effects of this combination therapy on dyspnea and exercise performance, a known predictor of survival in CF, are not clear. The investigators aim to understand the effects of Orkambi on these symptoms and to gain new insight into the potential health improvements in CF from using this treatment option.

In patients with cystic fibrosis (CF) the commonest cause of death is respiratory failure. Respiratory failure can have many causes. However, in patients with CF a major contributor is the impairment of the muscles required for breathing (respiratory muscles). Respiratory muscle impairment can result from poor nutrition. Lung function declines particularly during adolescence whilst body composition also changes at the same time. Thus, the investigators plan to study the relationship of nutrition and body composition to respiratory muscle strength and lung function in children and young people with CF aged between 12-18 years. The body mass index (BMI) is currently used in the clinical setting to measure nutritional status in CF. At King's College Hospital (KCH) there are portable devices to assess both respiratory muscle function and lung function. The research team will use a Bioelectrical Impedance Analysis (BIA) device to assess body composition, including BMI and lean body mass (LBM). The aim of the study is primarily to assess whether measurements of LBM impairment may better relate to poor lung function compared to BMI and secondly to examine whether lung and respiratory muscle function correlates with exercise tolerance.

A hot topic in lung transplantation is the treatment of persisting sinus disease/colonization in CF patients to prevent descending graft colonization and chronic allograft dysfunction. From 2012, the Hannover transplantation group has been using a conservative approach with topical nasal inhalation. It is now necessary to analyse the impact of the new approach on graft colonization, incidence of BOS, symptoms, QoL etc in comparison to a historical cohort. It is also important to establish which is the best among the different inhaled antibiotic regimens currently available.

The objective in this study is collecting post-marketing information on the safety and efficacy of Ciproxan injection under the routine clinical practice.

Cystic fibrosis (CF) is a disease that affects multiple systems, however, the accumulation of secretion in the airways contributes to the fact that pulmonary complications are the main responsible for the high rates of morbidity and mortality. Physical exercise can be an important component in the treatment of these patients, and modalities such as Nintendo Wii and Xbox One stand out as feasible and innovative methods that can contribute to increase adherence to rehabilitation. Thus, the objective of this study is to compare the cardiorespiratory responses during a maximal exercise test with the use of Nintendo Wii and Xbox One in patients with CF and healthy individuals. Patients with a clinical diagnosis of CF, aged from 6 to 30 years old and in stable clinical conditions will be included. For healthy individuals, those who are considered healthy from the application of a health questionnaire and also aged from 6 to 30 years old will be included. Patients in both groups with cardiopathies, musculoskeletal diseases, neurological diseases or signs of pulmonary exacerbation will be excluded. Participants will complete a questionnaire to assess their level of physical activity. Afterwards, cardiopulmonary exercise test (CPET) will be performed (visit 1), followed by the use of video games (visit 2). The video game will be practiced using a Nintendo Wii and a Xbox One (10 minutes each). A rest of 10 minutes will be provided between the use of each video game. During the interactive games, patients will use an accelerometer to assess the level of physical activity. In parallel to this, healthy subjects will be invited to perform the same tests in two visits. To evaluate the perception of fatigue, the OMNI scale will be used. In addition, individuals will be asked to evaluate their level of satisfaction after each game.

identify and validate biomarkers that might reflect partial restoration of CFTR function and can be used to monitor disease progression, and ii) evaluate the mechanistic effects of CFTR modulators and other relevant therapies in individuals with CF

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.

Respiratory Viral Infections (RVI) are particularly frequent in young children. Old data mention the deleterious role of some viruses such as the Respiratory Syncytial Virus in young children with cystic fibrosis (CF). However, recent epidemiological data on RVI in CF children are rare and the impact of most frequent viruses such as human rhinoviruses is usually not correctly evaluated. The aim of this study is to assess the frequency of lower and upper RVI during a 1 year follow-up in CF infants and to evaluate the impact of RVI at a clinical, microbiological and therapeutic level. Our hypothesis is that frequent and/or clinically severe RVIs have the worst impact in the short term and without any particular link with a specific virus as previously described.

The purpose of the study is to examine the real-life safety and effectiveness of the novel combination ivacaftor+lumacaftor in eligible patients with cystic fibrosis (CF). All patients with CF were eligible if they were 12 years and older, started ivacaftor+lumacaftor outside of a clinical trial between December 15th 2017 and December 15th 2018 in an accredited CF center in France. Patient followed-up is based on standardized recommendation of the French Cystic Fibrosis Society. Each patient is followed 1 year.