View clinical trials related to Cystic Fibrosis.
Filter by:This is an observational study for children with Cystic Fibrosis (CF) who are eligible based on their CF gene type. One group will be called the treatment group because they have the gene type (homozygous F508del) that makes them clinically eligible through their CF care provider to begin treatment with the new FDA approved CF drug called orkambi. For the control group, children will be enrolled who have a similar CF gene type (heterozygous F508del) but are not eligible to be prescribed orkambi. The two groups will be followed for four visits over about 3 to 4 years to observe changes in the lungs. Methods to measure the changes in lung disease will include: MRI with non-FDA approved inhaled xenon gas to take detailed images of the lungs, Pulmonary Function Tests (PFT), Lung Clearance Index (LCI), Baseline CT image of the lungs if not ordered as part of usual clinical care. The first two visits will be done before starting clinical treatment with orkambi and will be a minimum of 28 days apart and up to 18 months. The third visit will be scheduled about 3 months after starting orkambi and the fourth visit about 18 months later. For the control group, the timing of visits will be similar to treatment group and visits may be scheduled around annual CF care visits.
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.
Respiratory Viral Infections (RVI) are particularly frequent in young children. Old data mention the deleterious role of some viruses such as the Respiratory Syncytial Virus in young children with cystic fibrosis (CF). However, recent epidemiological data on RVI in CF children are rare and the impact of most frequent viruses such as human rhinoviruses is usually not correctly evaluated. The aim of this study is to assess the frequency of lower and upper RVI during a 1 year follow-up in CF infants and to evaluate the impact of RVI at a clinical, microbiological and therapeutic level. Our hypothesis is that frequent and/or clinically severe RVIs have the worst impact in the short term and without any particular link with a specific virus as previously described.
In this trial, various factors that may influence the antibiotic concentrations measured in the sputum of cystic fibrosis patients are studied. A first factor is aerosol use. As cystic fibrosis patients often use aerosols, such as hypertonic saline, dilution of the antibiotics in sputum can be expected. The extent of this dilution is unknown and will be determined by comparing sputum samples collected before and after the use of an aerosol. A second factor is the homogeneity of the antibiotics within one sputum sample. Multiple aliquots of the same sputum sample will be compared. A third factor is the variability between several sputum samples collected during a drainage session. The antibiotic concentrations in 3 separate sputum samples will be compared. The final goal is to standardise the sputum sample collection and processing of the samples to ensure a accurate concentration measurements in sputum.
Evolution of medical care for patients with cystic fibrosis appeared in recent years: prolongation of life expectancy with consequently increased comorbidities and the use of lung transplantation, systematic neonatal screening of new born since 2002, prescription of expensive new molecules or new presentation. This evolution justifies to realize an update of statistics of costs of care and to assess adherence to treatment
This is a pilot cross-sectional study of measured transcutaneous CO-oximetry in children with inflammatory and non-inflammatory conditions.
Liprotamase consists of 3 soluble, non-porcine digestive enzymes, lipase, protease, and amylase, combined in a fixed ratio. Liprotamase is stable in the stomach and can be formulated without enteric coating for administration either as a capsule or as a dosing solution dissolved in water or juice.
The study will evaluate the impact of smart adherence technology for monitoring on lumacaftor/ivacaftor (LUM/IVA) adherence rates among subjects 16 years of age and older with Cystic Fibrosis (CF) who are homozygous for the F508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mutation.
Shortness of breath (dyspnea) during exercise is a major source of distress and is a commonly reported symptom in patients with cystic fibrosis (CF). A recent treatment option known as Orkambi, which combines the drugs lumacaftor and ivacaftor, may be used in patients with CF to help improve lung health. However, the effects of this combination therapy on dyspnea and exercise performance, a known predictor of survival in CF, are not clear. The investigators aim to understand the effects of Orkambi on these symptoms and to gain new insight into the potential health improvements in CF from using this treatment option.
With improved survival, the clinical spectrum of cystic fibrosis (CF), a complex multi systemic disease, continue to evolve. A major emerging complication is CF related diabetes (CFRD) which is occurring in 40-50% of adults. Patients who develop CFRD are at increase risk of morbidity and mortality and they are also facing an increased medical burden with insulin therapy, the only recommended treatment. Accelerated decline in weight and/or pulmonary function start 2 to 4 years before CFRD onset and this period is characterized by frequent Post-Prandial Glycemic (PPG) excursions. Higher PPG excursions are associated with lower pulmonary function and it predicts future CFRD risk. To the improved nutritional status, lung function and survival of patients, the nutritional approach for patients with CF focuses on high-energy high-fat diet and a pancreatic enzyme supplementation. However, such diet also contributes to increased PPG excursions. Based on the beneficial effects of nutrition therapy to improve PPG in other forms of pre-diabetes and diabetes, extending such benefits to patients with CF is important. The investigators aim to test the feasibility and the effectiveness of a viscous fiber supplement to reduce PPG in adult patients with CF. Using a randomized crossover design, the investigators will study the impact of two doses of a viscous fiber supplement as compared to a placebo.