Active clinical trials for "Cystic Fibrosis"

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.

Clinical studies of lumacaftor + ivacaftor (combo therapy) produced better FEV1 (forced expiratory volume in 1 second) improvements than ivacaftor alone, without further improvement in sweat chloride results. To help understand why sweat chloride was unresponsive, the investigators will use a newly developed sweat secretion test that provides accurate, in vivo readout of CFTR (cystic fibrosis transmembrane conductance regulator) function in the sweat gland secretory coil. The investigators devised a protocol to determine if short courses of ivacaftor (3.5 days) will produce significant increases in WT (Wild-Type, i.e. normal) CFTR open probability by measuring CFTR-dependent sweating (C-sweat) in subjects with WT CFTR.

Adult patients with cystic fibrosis (CF) are treated with high dose antibiotics to reduce the long term damage to their lungs from infection. This would typically be with a two week course of intravenous antibiotics each time they have a chest infection (typically three to four times a year). The most effective and commonly used antibiotic in most cases is tobramycin. If this cannot be used because of previous side effects, allergy or a resistant infection then colomycin or amikacin are usually used. Each of these antibiotics are known to be toxic to both the kidneys and ear. As patients are living longer (into their forties), the total amount of these antibiotics they are receiving over their lifetime is increasing. This is now leading to increased complications such as kidney damage and hearing loss. Because of this, the investigators need to look at methods to accurately quantify damage and reduce potential kidney and hearing damage. The investigators intend to quantify kidney damage by measuring new protein markers within the urine and blood that signify kidney damage before more conventional and currently available methods are able to.In those patients treated with intravenous tobramycin the investigators will also look at an alternative method used to calculate the most appropriate dose of antibiotic for each participant. This dosing method is called 'area under the curve or AUC' dose monitoring. This method currently in clinical use in other countries is thought to more accurately reflect the most appropriate dose for each participant and thus reduce the chance of kidney and hearing problems. This 'AUC' method requires two rather than one dose level to be checked each time a dose calculation is made. Participants receiving tobramycin will be randomised to receive dosing by this method or the investigators' currently used method of 'trough' monitoring.

The purpose of this study is to develop rapid MRI techniques for imaging the lung with hyperpolarized helium-3 gas as an inhaled contrast agent. These techniques will be piloted in adults and older children before testing them in younger children and infants. The purpose is to enable imaging of non-sedated infants by imaging so fast as to freeze motion.

Ivacaftor is a novel, FDA approved new therapy that addresses Cystic fibrosis transmembrane conductance regulator (CFTR) dysfunctions in subjects with Cystic fibrosis (CF) and "gating mutations". The primary aim is to determine the mechanism(s) for weight gain in participants whom Ivacaftor treatment was initiated based on clinical indications by CF Care Team. This longitudinal study will assess in detail energy expenditure, weight gain, body composition, and lung function in 24 subjects ≥6 years old with CF with a gating mutation before treatment and after three months treatment with Ivacaftor. All subjects will be seen at the Children's Hospital of Philadelphia's Clinical Translational Research Center.

The major causes of morbidity and mortality in Cystic Fibrosis (CF) are linked to the process of chronic inflammatory of the airway, leading to the progressive damage of the small bronchioles and subsequently to the proximal bronchi. A connection between weaknesses of respiratory muscles in CF and deficits of CFTR in the muscle has been established. Insufficient cough in CF patients may advance re-current respiratory infections. A voluntary cough flow volume (C-FVC) profile incorporates the characteristics of the forced expiratory flow volume curve (FE-VC). The study aims to explore the correspondence of voluntary cough-flow-volume and maximum expiratory flow-volume maneuvers in relation to disease complications in CF patients.

The purpose of this study is to determine whether the treatment with Ivacaftor remains effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR mutation) in the real life setting, after the drug has been approved by the Health authorities.

Lung Health research study (Development of a predictive model) - To determine whether the I-neb breathing parameters (flow data) can act as a surrogate marker for lung function (Forced Expiratory Volume in 1 second FEV1) hence allow early detection of decline in lung function in cystic fibrosis patients.

Main objective : This project aims to assess the educational needs of patients who received a lung transplantation or a simultaneous heart-lung transplantation for mucoviscidosis. Specific objectives : Evaluate the specific needs dependent on seniority transplantation : 3 to 6 months, 6 months to 2 years, more than 2 years after transplantation Secondary objectives : Assess the state of health Assess the knowledge Assess gestural know-how Assess the cognitive know-how Assess the behaviour adopted in reality by the patient Assess motivation psychosocial factors of : self-efficacy and inefficiency representation of obstacles, disadvantages of treatment sense of control over their health feeling of being supported by health professionals, by his close representation of his responsibility to take care of his health, to follow his treatment, to be active about his treatment

Cystic fibrosis (CF) is a life-shortening disease that causes breathing and digestive problems, but can now be diagnosed at the time of birth. Lung function is very hard to measure in infants, but growth is not. In this study the investigators aim to define growth in infants with CF in the first year of life with research quality precision and to understand factors that interfere with good growth. Pancreatic enzyme replacement therapy (PERT) will be also be studied in a subgroup of infants. Two different doses of PERT will be evaluated for improving fat and nitrogen absorption in infants with CF.