View clinical trials related to Cystic Fibrosis.
Filter by:Background/Rationale Acute rejection (AR) is common in the first year after lung transplantation. AR has usually been reversible with treatment, but it can trigger chronic rejection that is the leading causes of late morbidity and mortality. Extracorporeal photopheresis (ECP) has emerged as a promising treatment for chronic rejection. The investigators postulate that the immunoregulatory property of ECP could promote graft tolerance immediately after lung transplantation. Objectives The aim of this trial is to evaluate the safety and efficacy of ECP as induction therapy for prevention of AR in recipients affected with cystic fibrosis in the first year after lung transplantation. The extracellular vesicles in the cell-to-cell communication and immunomodulation will be also investigated. Preliminary results (personal) A preliminary study, conducted in Vienna, demonstrated that 9 patients treated with ECP as induction therapy had 0% of chronic rejection versus 50% in the control group. The Institution hosting the current project is among largest lung transplantation centers in Italy with high rate of cystic fibrosis recipients. The Institution has experience in ECP and a dedicated instrument was specifically bought for the project. Internal collaborators have strong expertise in biological aspects including the extracellular vesicle compartment.
An increase in the prevalence of infections due to non-tuberculous mycobacteria (NTM) is observed in many countries and recent data suggest the circulation of dominant clones with a possibility of human-to-human contamination. The hypothesis is made that these infections are also increasing in France and that dominant NTM clones are circulating. The last French study carried out in 2004 already showed prevalences of up to 10% in certain French regions. It is essential to know the prevalence 8 years later, taking advantage of the new recommendations for the management of patients and samples, which will homogenize practices on French territory. No data are currently available in France on the prevalence of positive serological responses in cystic fibrosis patients. Serological analyzes of the sera collected during this study will enable us to evaluate the performance of serology in mycobacterial culture and to identify patients with no positive respiratory specimen in culture but with positive serology indicating potential contact with a mycobacterium. The establishment of a serological follow-up of these patients will allow to correlate this result with a clinical evolution and / or the detection of NTM in subsequent samples. Serology is an innovative aspect of the CIMENT study.
Interventional trial to study the influence of the use of MyCyFAPP (mobile application) on the gastro-intestinal related quality of life. This mobile APP has been developed during previous workpackages of the Horizon2020 Project and contains several modules: - mathematical prediction model to calculate the needed dose for pancreatic enzyme replacement therapy - educational games and other educational material - communication with doctor/dietician through professional webtool - diary to register symptoms and data on nutrition. The app will be introduced and used during 6 months. Primary outcome parameter will be change in modified PedsQL GI after 3 months. PedsQL GI is an existing questionnaire that evaluates gastro-intestinal related quality of life in children. We validated it for use in cystic fibrosis in a previous observational study.
Diagnosis of hepatic fibrosis is challenging as specific tests for detection of fibrosis in pediatric Cystic Fibrosis associated liver disease (CFALD) have not been developed and existing investigations do not correlate well with presence or severity of disease. Using a Liver biopsy it is difficult to diagnose this condition because of the patchy nature of the disease. Investigators intend to identify hepatic and pancreatic fibrosis in Cystic Fibrosis patients using Elastography and correlate this with their biochemical markers as well as histological findings of patients who have undergone liver biopsy for diagnosis of CFALD.
This study is a multicenter, prospective cohort study of patients diagnosed with cystic fibrosis, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 10 years.
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.
Cystic fibrosis related diabetes (CFRD) is a common co-morbidity in patients with CF. The underlying pathophysiology of cystic fibrosis related diabetes (CFRD) is still a matter of investigation. In addition to localized tissue damage developing similar to that of the exocrine pancreas, other mechanisms may be involved. We have shown that a potential contributing factor to the patho-physiology of CFRD may be an abnormal gut derived hormonal profile, specifically of lower incretin hormone responses, prior to development of CFRD. We propose that an altered incretin response, probably due to impaired interaction of nutrients with the gut mucosa due to thickened secretions, may play a role in the development of the disease. Specifically, low GIP and GLP-1, may explain the poor β-cell function observed in these patients prior to CFRD appearance. These incretins have known trophic effects on β-cells, and thus their lower levels may contribute to the development of quantitative as well as qualitative defects in β-cell function and thus may lead to the development of CFRD. Thus, increasing levels of these incretins using a DPP-IV inhibitor may improve glucose metabolism and delay/prevent the development of CFRD. We hypothesize that Saxagliptin will increase the oDI compared to placebo and will thus provide relative protection from diabetes development and in addition we expect that Saxagliptin will lead to overall increased insulin concentrations and thus shift the metabolic milieu to a more anabolic state. This will manifest as weight gain and reduction in inflammation.
Adolescents with cystic fibrosis are particularly vulnerable to poor adherence, which negatively impacts their health status, quality of life and long term survival. CFFONE: A Cell Phone Support Program for Adolescents with Cystic Fibrosis, will make use of cutting-edge technology- a broadband capable, cellular telephone keyed into a highly-interactive informational web site. This web site will provide engaging online learning activities and resources specific to adolescents with cystic fibrosis. We believe the information and activities contained in CFFONE will improve adolescents knowledge, attitudes, and practices around cystic fibrosis and that adolescents exposed to the CFFONE program will demonstrate an increase in adherence to their treatment regimens and related improvements in their health status and quality of life.
The purpose of the study is to evaluate whether insulin treatment during pulmonary exacerbation (PE) in patients with Cystic Fibrosis (CF)and normoglycemia improves their short term outcome by normalizing the glycemic profile and enhancing recovery. the investigators would like to evaluate whether insulin treatment during exacerbation improves both the general clinical condition of these patients and also has a protecting effect on ß-cells by preventing the deleterious effect of "chronic" hyperglycemia.
Cystic Fibrosis patients attending with infective exacerbations will be enrolled into the study. The trial is a double blinded, randomised trial with patients randomised to 10,14 or 21 days of antibiotic therapy, comprising of tobramycin and either ceftazidime or meropenem.