Active clinical trials for "Cystic Fibrosis"

The purpose of this study is to characterize the rheological properties of cystic fibrosis (CF) and healthy sputum and to examine the effects of mucoactive agents on the rheology of CF and healthy sputum. By collaborating with Genentech, the investigators (scientists at UCSF) plan to incorporate the latest scientific findings into our work to discover and develop new treatments for CF.

It is well known that an association exists between the acquisition of some respiratory pathogens and prognosis of CF people. About 57 % of patients has a lung infection due to P.aeruginosa (PA). Transmission of PA between CF patients is possible and several prevention measures are recommended. Among these measures, all international guidelines recommend that CF people maintain a minimum distance of 1 meter between them. However, this recommendation is not supported by specific studies and scientific evidence. In other words, the investigators don't know if this measure is sufficient or excessive, as it is based only on a theoretical rationale. This study aims at measuring experimentally the distance that can be reached by PA emitted from airways of colonized CF patients during cough and during conversation. To this purpose, the investigators will evacuate the presence of PA on surfaces placed at 4 different distances from patients, through the collection of 8 swabs, 4 of which following cough and 4 following conversation. Swabs will be collected on sterile surfaces comprised between tra 0 - 0,5 mt; 0,5 -1,00 mt.; 1,00 - 1,5 mt and 1.5-2 mt. All the PA-positive CF patients of the CF Centre of Tuscany aged 14 or more will be recruited.

Cystic fibrosis (CF) is the most common lethal, inherited disorder among Caucasians. Choline is an essential vitamin and as a methyl donor is critically needed to support the normal metabolism. Our previous studies have demonstrated that children with CF have depleted levels of choline. The purpose of this study is to gather data on the status of choline and related metabolites in children with Cystic Fibrosis by age and gender. The hypothesis for this study is that in children with CF, deficiency of choline and related metabolites will increase with increasing age.

This experiment is designed to test the effectiveness of a new electronic nose device, which allows a non-invasive breath test for markers of lower respiratory tract infection, which may predict the probability of bacterial organisms in the lower respiratory tract. It consists of: A breath collection apparatus for collection of volatile organic compounds in breath onto a sorbent trap and Tedlar bag, as well as for the collection of a separate sample of room air. Analysis of the volatile organic compounds in breath and room air by short acoustic wave/gas chromatography. Interpretation of the volatile organic compounds with a proprietary algorithm in order to predict the probability of lower respiratory tract colonization and infection. This study will test the hypothesis that the investigators can identify the presence of Pseudomonas aeruginosa by sampling the "head space" above culture media of sputum provided by patients with cystic fibrosis. This study will test the additional hypothesis that the investigators can identify the presence of Pseudomonas aeruginosa by sampling exhaled breath from the patient providing the sputum.

Mucociliary clearance, in which mucus secretions are cleared from the breathing airways, is the primary defense mechanism for the lungs. Inhaled particles, including microbes that can cause infections, are normally entrapped in mucus on the airway surfaces and then cleared out by the coordinated action of tiny hair-like structures called cilia. Individuals with primary ciliary dyskinesia, variant cystic fibrosis, and pseudohypoaldosteronism have defective mucociliary clearance. The purpose of this study is to collect clinical and genetic information about these three airway diseases to improve current diagnostic procedures.

In 2006, the International Patient Decision Aids Collaboration (IPDAS) reached agreement on criteria for evaluation. Accordingly, the primary outcome is 'decision quality' 3 weeks after using the decision aid. Decision quality is defined as the extent to which a patient decision aid improves the match between the chosen option and the features that matter most to the informed patient. Measures include: a) patients' knowledge of essential facts [options, benefits, harms, and outcome probabilities]; and b. the congruence between the option chosen and patients' informed values regarding benefits and risks. In the past twenty years lung transplantation has become the most widely accepted option of treating cystic fibrosis patients with severe lung disease. Lung transplantation can be a good experience for many patients, improving their quality of life and their survival. However there are potential risks of lung transplantation including infection, organ rejection and early death. Cystic fibrosis patients are often faced with making a choice of whether to be referred for lung transplantation when they are very sick and there is the immediate need to survive. Our group has developed a tool called a decision aid which we hope will assist the patient and family in making this choice. The decision aid guides the patient through a series of steps where they weigh the benefits and risks of being referred for lung transplantation and the benefits and risks of receiving 'usual care' without the option of referral.

The purpose of this study is to find out whether measurement of body composition has a prognostic value on clinical and overall outcome in pediatric patients with cystic fibrosis.

This study is a single center study of clinical and laboratory outcomes in patients ≥ 12 who transition from use of Orkambi to tez/iva. Clinical and laboratory measurements will be measured at baseline, 1 month, 3 months, and 6 months after initiation of tez/iva. Change from baseline at 6 months pre-specified will be reported. The length of study participation will be approximately 6 months.

For the Project VEMSE-CF, a comprehensive patient-centered outpatient health service model was developed offering focused interventions in different areas. These included patient education, as well as nutritional and exercise counselling. Special emphasis was given to the psycho-social services. The implementation was supported by a case manager. The model was implemented in three German CF-Centers. For evaluation, Patient data from 13 additional German CF-Centers offering standard care was used as comparison. In total, 153 patients in the Intervention Group and 163 control patients aged 5-52 years contributed data. The primary endpoint was the number of BMI- and FEV1-measurements over 24 months. Secondary endpoints included mortality, lung transplantation, FEV1, BMI, adherence to therapy, Quality of life, and mental stress.

Background The main risk factor for cervical cancer is the infection by human papillomavirus (HPV). Vaccination against HPV, offered to all girls aged 11 to 14 is an effective method of prevention against cervical pathology. Despite this, vaccination coverage against HPV remains low in France. A proportion of women with cystic fibrosis may be involved in transplantation, a factor associated with a higher risk of HPV carriage and cervical pathology. An over-risk of cervical pathology would also be present in women with non-transplanted cystic fibrosis. Particular attention to vaccination should therefore be included in this population. Objectives of the study The main objective of the study is to estimate the frequency of HPV vaccination in young girls with CF over 9 years and followed in a pediatric CF center. The secondary objectives are to know: The type of vaccine used (bivalent / quadrivalent / nonavalent) The proportion of vaccinated girls with respect of the vaccination schedule (number of injections / spacing between doses) Reasons for non-use of vaccination Study design The study will last 12 months. It is a cross-sectional, non-interventional, multicenter conducted by self-administered questionnaire. Population young girls aged 9 years or older with Cystic fibrosis Followed in a pediatric or mixed CF center in the France (Rhone-Alpes Auvergne Region and Ile de France Region) With parents who did not object to participation in the study Number of subject: 62 patients Expected results Knowledge of HPV vaccination coverage in young girls with CF. Sensitization of patients, their parents and health professionals to HPV vaccination. Understand the barriers and reasons for refusing vaccination to promote actions to improve immunization coverage.