Active clinical trials for "Cystic Fibrosis"

Inflammation is present in the Cystic fibrosis (CF) airway from the time of infancy, and worsens with the onset of chronic infection. Therapies with proven benefit are associated with decreased airway inflammation. Thus, sensitive and reproducible biomarkers of airway inflammation have long been sought as a necessary component to improved clinical care and to facilitate therapeutic trials for CF. FEV1, the standard outcome measure in CF, is recognized as an insensitive outcome measure. the investigators have identified a panel of 10 genes which sensitively predict resolution of pulmonary inflammation, in response to therapy of an acute pulmonary exacerbation. With the goal of yielding a technically simple but unique CF biomarker assay, the investigators have tested whether proteins signified by these genes show large changes in expression following treatment of acute pulmonary exacerbations. Protein quantifications are among the most common measurements performed in clinical laboratories around the world. Based on preliminary findings that changes in white blood cell proteins mirror changes seen in the genes, the investigators propose to identify top candidate proteins, from the investigators gene panel, which change in response to exacerbation therapy. Once identified, these proteins will be quantified directly with a new blood test which is inexpensive and simple to perform. The investigators propose to validate this blood test in a single site trial. If successful, this proposal will yield a biomarker assay that will be available to validate in a multi-site trial and provide unique insights into mechanisms that regulate white blood cell activation and recruitment in CF lung disease.

Currently the management of cystic fibrosis (CF) patients is mainly based on symptomatic treatments. The arrival of children in day hospital, every 2 to 3 months, is for many of them and those around them, stressful, rarely expressed, but noticeable by the care team. In line with the recommendations of the High Authority of Health, the multidisciplinary team of Resources and Skills Centre for Cystic Fibrosis (CRCM) Pediatric of Nantes University Hospital offers a therapeutic education program tailored to the needs of children with cystic fibrosis and their families . The aim is to support children and parents in the acquisition of self-care skills (knowledge and skills) and coping skills, such as managing stress and emotions . Our goal is to demonstrate that art therapy, mobilizing coping skills of the child, facilitates its expressive capabilities and reduces anxiety.

Infants included in the study will be divided into two groups as healthy infants and cystic fibrosis infants . Demographic, natal and postnatal information of the infants will be available from patient files. 12 months infants with cystic fibrosis and healthy infants will be assessed by the Bayley-III Baby and Child Development Assessment Scale (Bayley III) and the Gross Motor Function Measure. The assessment outcomes of healthy and cystic fibrosis infants will be compared.

Airway disease, featuring intense inflammation, is the main cause of morbidity and mortality in cystic fibrosis (CF). Mechanisms of CF airway inflammation remain unclear, hampering development of better treatments.This time-sensitive ancillary study leverages a unique longitudinal cohort of CF infants, assessing the early phase of airway disease. Through the use of innovative cell and fluid based tools for in vivo profiling and in vitro testing of BALF samples, this translational effort will yield unprecedented insights into mechanisms of PMN dysfunction in CF, and assess new paths for early intervention.

Breathing movements, called chest wall motion, are very complex. The investigators are studying how movement of the abdomen, ribs and diaphragm contribute to breathing and how this differs with different diseases in the chest. Breathing movements may help with diagnosis, assessment of severity or assessing the impact of treatments for chest conditions. The investigators are following people who have a chest disease, measuring their chest wall motion and comparing it to their diagnosis and and how their treatment works. Chest wall motion can be measured in different ways at rest and whilst exercising. Small stickers on the chest can be used to reflect infra red light or visible squares of light can be shone onto the chest without using stickers.

Little is known about gynaecological follow-up and cervical screening in Cystic Fibrosis (CF). Only few studies have described contraceptive practices in cohorts of CF women. The investigators did a cross-sectional study in a cohort of 155 CF women attending the Lyon adult centre. Women attending the CF adult centre in 2014 completed a written questionnaire about their contraceptive choices, frequency of gynaecological follow-up and cervical screening. Other clinical data were collected from the CF adult centre registry. One hundred and twenty women (100%) answered the questionnaire, among whom two were post-menopausal (46 and 59 years of age), and five were pregnant. Seventy-four per cent of the women declared they had undergone gynaecological followup (89% of the women with transplantation), and only 55% reported having at least one previous Pap smear test. Among the transplanted patients, only 58% had had a Papsmear test, despite immunosuppressive treatment. The overall rate of contraception was only 64% and in diabetic women, it was 61%. Among contraception users; 65% used oral contraception, predominantly combined estrogen-progestagen (47%); among diabetic patients, 26% used progestin-only contraception. Intrauterine device accounted for 10% of patients using contraception, and tubal ligation only 4%. This study is limited by its cross-sectional design. Despite an internal validation of the questionnaire showing an almost perfect agreement, the risk of recall bias has to be taken into account. This study of practices highlights the importance of improved information regarding sexuality, fertility and reproductive health in young women with CF. A regular gynaecological follow-up and cervical screening is mandatory in this population. Better gynaecological care and contraceptive advice would help to avoid unplanned pregnancies, and optimize contraceptive selection in relationship to specific clinical conditions.

The main objective of this project is to evaluate the agreement between nasopharyngeal and sputum specimens in terms of detection of viral infection in Cystic Fibrosis (CF) participants.This is a 12-month national, multicenter prospective study (7 centers). Inclusions concern CF participants (children or adults) with signs of acute respiratory infection seen in consultation or hospitalized in their CF Research Center. A molecular viral multiplex search is performed on both nasopharyngeal and expectoration collected samples for each included participant. Determination of viral detection agreement between the two CF respiratory samples is then performed.

A survey of adults and parents of children with cystic fibrosis on food insecurity

prospective multicentric study protocol in french CF center, exploring adolescent avec parents needs during transition from pediatric CF center to adult CF center

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.