Active clinical trials for "Cystic Fibrosis"

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.

Pharmacokinetics of colimycin will be assessed during 12 hours after administration of 2M UI colimycin either as an aerosol or after IV injection, the administration being separated by 5 to 10 days intervals

The purpose of the study is to investigate the relationship between hypoxemia achieved during Hypoxia Altitude Simulation test and sea level values of pulmonary function, arterial blood gases, pulse oximetry and cardiopulmonary exercise test variables. In addition, to study the effect of slow walk in a hypoxic environment, comparable to slow walking along the aisle.

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

The aim is to evaluate the impact of a 3-week lasting residential rehabilitation program for patients with cystic fibrosis on their nutritional status measured as BMI and body composition and pulmonary function.

The purpose of this research is to determine if an investigational device called the 13C-Spirulina Gastric Emptying Breath Test (GEBT), can accurately diagnose gastroparesis (delayed emptying of the stomach) in patients with Cystic Fibrosis (CF).

The present application proposes to study the role the composition of the pediatric CF airway microbiota plays in frequent pulmonary exacerbations in pediatric CF patients. In order to accomplish this goal the dynamics of the composition of the CF airway microbiota in two distinct subsets of pediatric patients with CF will be characterized, those with frequent pulmonary exacerbations and clinically stable children. Clinical measures of pulmonary function, patient reported symptoms, sleep quality, and antibiotic usage will be recorded, and these findings will be correlated with the lung microbiota data. This strategy promises to identify the key characteristics of the pediatric CF microbiota, which can in turn be used as noninvasive markers to identify those patients at a higher risk for experiencing repeated pulmonary exacerbations.

The objective of the study is to implement a new method of performing chest CT imaging in young children with cystic fibrosis at Packard Children's Hospital. This technique will be used to evaluate early lung disease comparing quantitative chest CT air trapping and airway measurements with lung function measurements in infants, toddlers, and young children with chronic lung disease.

The aim of the study is identify biological parameters reflecting proteolytic activity in the exhaled condenstaes which might be useful to follow up pulmonary inflammation in various conditions including cystic fibrosis, COPD, asthma, tobacco exposure. Three proteases will be analyzed, i.e. elastase, proteinase 3 and cathepsin G. Results in the condensates will be compared to those obtained in sputum.