Active clinical trials for "Cystic Fibrosis"

Approximately half of the variation in the presentation and disease status of patients with Cystic Fibrosis (CF) is secondary to non-genetic variables.[1] These include household income, compliance with therapy, second hand smoke exposure and possibly exercise (although exercise has less evidence to support its influence on disease status). Recently increased attention has been focused on the role of exercise and quality of life for patients with CF. This study will evaluate the role of exercise in relation to the patient's perception of their quality of life. The primary outcome will be the overall change in the patient's Cystic Fibrosis Questionnaire - Revised (CFQ-R) over a one year period during which the participants are encouraged to exercise and use a personal fitness device to track their exercise. This is a prospective single group non-blinded study. Participants will be asked to wear a personal fitness device for 11-13 months. The change in CFQ-R score is the primary outcome and other secondary outcomes will include; (1) exacerbations of cystic fibrosis (through use of antibiotics), (2) change in subject's FEV1, (3) inpatient admission rates, (4) change in BMI and (5) change in 6 minute walk test. The study will take place at the Adult CF Clinic on the University of Oklahoma Health Sciences Center campus.

The main risk factor for the development of cervical lesions is human papillomavirus (HPV) infection. Vaccination against human papillomavirus, which is offered to all girls aged 11 to 14, and catching up to girls aged 15 to 19, is an effective method of preventing cervical pathology. Despite this, human papillomavirus vaccination coverage remains low in France. Some women with cystic fibrosis might need a transplantation, which is a factor associated with increased risk of human papillomavirus carriage and cervical pathology. An over-risk of cervical pathology would also be present in non-transplanted women with cystic fibrosis. Particular attention should therefore be paid to vaccination in this population. The objective is to estimate the frequency of human papillomavirus vaccination in young patients with cystic fibrosis, and to evaluate the reasons for non-vaccination.

characterization of CFTR function and expression in nasal primary cells collected from patients with cystic fibrosis in comparison to their parents, healthy heterozygotes and healthy controls

Cystic fibrosis can affect organs other than the lungs. Liver disease affects about 30% of patients: its main manifestation is the development of portal hypertension (PHT). The pathophysiology of this comorbidity is still poorly understood. It was previously considered secondary to the formation of biliary cirrhosis but another hypothesis would be that of a primitive pathology of venous vessels may cause the gradual emergence of portal hypertension without cirrhosis. Evidence indiscutly suggest that cystic fibrosis is associated with a specific endothelial dysfunction, especially as the CFTR (Cystic Fibrosis Transmembrane conductance Regulator) protein is expressed on the surface of endothelial cells. The investigators hypothesize that liver disease related to PHT-associated cystic fibrosis is associated with systemic endothelial dysfunction. The aim is: To demonstrate a systemic endothelial dysfunction in patients with cystic fibrosis when associated with PHT. To study the correlations between measures of systemic endothelial function and serum markers of endothelial dysfunction and between measures of liver stiffness and systemic endothelial function.

Background: Lumacaftor/Ivacaftor (LUM-IVA), a CFTR corrector-potentiator combination, was found to improve lung function and reduce pulmonary exacerbations (PEx). However, cystic fibrosis (CF) is a multi-organ disease and therefore there is a need for more information on the systemic effects of CFTR modulators. Aim: To evaluate pancreatic function, bone metabolism and respiratory changes through a year of LUM-IVA treatment. Methods: A prospective real world, one-year study on F508del homozygous adult CF patients who commenced treatment with LUM-IVA. Visits were scheduled on the first day of treatment and every 3 months evaluating: symptoms, Body Mass Index (BMI), spirometry, laboratory tests and Quality of life. At baseline and at 12 months, the patients underwent sweat test, oral glucose tolerance test (OGTT), chest CT and dual-energy X-ray absorptiometry (DEXA).

This study aims to assess the feasibility of a new, brief intervention, the 'coopeRATE Prompt', for informing conversations between patients and physicians in routine tele-health and in-person adult Cystic Fibrosis (CF) care. The coopeRATE Prompt is two questions designed to elicit patients' concerns and goals to facilitate collaborative goal setting within the health care visit. This is a prospective single arm study that will be conducted at four CF care centers in the United States.

The purpose of this study is to explore and collect the perceptions, expectations and needs of CF patients about parenting. This will be done in the context of several small groups of patients led by a psychologist who will ensure that all the participants express themselves; he will encourage them to develop their points of view, their divergences and their common points about what constitutes to be a parent. The collected information should make it possible to develop and propose adapted medico-psycho-social interventions, if necessary, in connection with patient associations

The purpose of this study is to investigate the expression of a certain class of molecules, called costimulatory molecules, in humans with Cystic Fibrosis. Cystic Fibrosis is a genetic disorder which renders the lung susceptible to persistent inflammation which, at times, can worsen, resulting in accelerated decline in lung function and eventually death or transplant. Our goal is to determine if the levels of costimulatory markers can be used to predict exacerbation and subsequent lung function decline in subjects with Cystic Fibrosis.

Cystic fibrosis (CF) affects an estimated 30,000 people in the United States and is caused by a mutation in the gene encoding a protein called CF transmembrane regulator (CFTR). The hallmarks of CF are recurrent pulmonary exacerbations and declining pulmonary function. However, there are other problems in CF that affect both health and quality of life. These include CF related diabetes, liver disease, and bone disease. The median age of survival for patients with CF has been increasing steadily and is currently more than 37 years. With this improvement in life expectancy, it has become increasingly important to address the long-term complications of CF. Currently, patients with CF are evaluated annually for bone disease with dual X-ray absorptiometry (DXA), and screening usually starts at age 12. However, this may not be sufficient to detect early bone changes that may impact fracture risk. Furthermore, bone disease in children may manifest earlier than adolescence, which would suggest that screening should start at an earlier age in these vulnerable patients. The following study is therefore proposed to examine the potential role of peripheral quantitative computed tomography (pQCT) as a screening approach for bone disease in children with CF. The investigators expect to find bone problems by pQCT but not DXA.

This study aims at collecting the information related to the safety and effectiveness in the pancreatic exocrine insufficiency patients due to cystic fibrosis receiving the treatment with LipaCreon in order to evaluate the effective and safe use of LipaCreon.