Active clinical trials for "Cystic Fibrosis"

Cystic fibrosis (CF)-related diabetes (CFRD) is the most common complication after pulmonary complications. This specific form of diabetes is associated with an increased morbidity and mortality. CFRD prevalence at the age of 10 is 10% and reaches 40 to 50% in adulthood, while another 35% of adult patients presents impaired glucose tolerance. In order to identify patients at risk and to implement early therapeutic measures, an annual CFRD screening test should therefore be undertaken for CF patients after 10 years of age. The 2-hour oral glucose tolerance test (OGTT) with a sweet beverage is the recommended screening test. However, participation rates for screening tests are far from optimal. For examples, in 2015, the investigators observed that only 47% of non-diabetic patients attended to planned screening despite large availability and advertisement (unpublished data). Comparable low levels of screening for CFRD, usually below 33%, have been reported by various teams. Several reasons could explain these low adherence rates. Some factors are related to patients perceptions and experience: OGTT is perceived as an additional medical burden requiring a scheduled appointment (several weeks after the last exacerbation); overnight fasting followed by the intake of a large glucose load within 5 minutes can lead to nausea, headache, bloating and fatigue; some patients fear multiple blood sampling, etc. In addition, in case of CFRD diagnosis, recommended capillary blood glucose monitoring, nutritional advice and treatment (insulin) are perceived as extremely invasive and complex, thus some patients prefer avoiding screening test. To date, no alternative screening method has demonstrated its effectiveness to screen for CFRD. The investigators of this study believe that a simplified version of the OGTT would be more attractive, would make it more acceptable for patients and has the potential to improve their adherence to screening tests, simplify CF-team works and reduce costs. By allowing appropriate education and introduction of treatment in a timely manner, improved adherence to annual screening for dysglycemia has the potential to minimize or prevent clinical deterioration observed in years preceding CFRD onset.

This study will evaluate the efficacy and safety of an oral ARV-1801(ACG-701) plus optimized background therapy (OBT) compared to oral placebo plus OBT, each administered for 14 days, in the treatment of participants with Cystic Fibrosis-related pulmonary exacerbations (PEx).

To identify whether home monitoring of multiple physiological parameters and biomarkers in sputum could provide advanced warning of an infective exacerbation or treatment failure before changes in patient-reported symptoms.

Cystic fibrosis (CF) is a complex systemic disease that mainly involves the respiratory and gastrointestinal (GI) tracts. The polymicrobial community composition of respiratory and GI tracts is influenced by both genetic and environmental factors. Children with CF may harbor an abnormal intestinal microflora, because of altered Cystic fibrosis transmembrane conductance regulator (CFTR) function and heavy drug load (antibiotics, pancreatic enzymes and acid suppressors). The investigators previously demonstrated that intestinal inflammation is highly frequent in CF children, being a major feature of intestinal involvement. In addition, specific probiotics significantly improved airway and GI inflammation in a preliminary trial. The aim of the study is to characterize intestinal and respiratory microflora in CF patients and to investigate the effects of daily Lactobacillus GG (LGG) supplementation on both GI and airway microflora and the eventual relationship between probiotic assumption and clinical and inflammation markers. The aim is to study the effect of microflora modification on intestinal and extraintestinal inflammation to eventually improve the quality of life of CF patients, who often suffer from intestinal and respiratory progressive disease, through a non invasive intervention consisting in the supplementation of probiotic bacteria.

This pilot study will be conducted in adolescents with cystic fibrosis (CF) without diabetes but with abnormal glucose tolerance, and will assess the effects of sitagliptin on glucose regulation. An oral glucose tolerance test (OGTT) and a mixed meal tolerance test (MMTT), will be performed at baseline and again ~4 weeks after treatment with study drug. We will also look at blood sugars throughout the day using a continuous glucose monitor (CGM) before each time the MMTT/OGTT are performed. Several hormones that may affect the way the body regulates blood sugars will be measured in blood when the OGTT and MMTT are done. We will assess the effect this medicine has on blood sugars (using CGM) and the effect the medicine has on the hormones measured during the OGTT and MMTT.

The hypothesis of this study is that feeding infants diagnosed with CF via newborn screening a formula enhanced with a specific fish-oil fatty acid known as DHA will improve growth and decrease pancreatic dysfunction (as measured by human fecal elastase-1 in stool) over the first year of life. Briefly, infants diagnosed with CF in the first month of life whose parents chose not to breast feed their babies will be invited to enroll in a study comparing a standard commercial infant formula (Enfamil) with a formula enriched with arachidonic acid (AA) and docosahexaenoic acid (DHA). The study formula has 3 times the amount of DHA available in commercially available formulas. Infants will have monthly tests of stool elastase and blood work at entry, 3, 6, 9 and 12 months of age.

Vitamin D deficiency is common in cystic fibrosis. Vitamin D deficiency frequently persists despite aggressive treatment with ergocalciferol, a vitamin D preparation also known as vitamin D2. Cholecalciferol, a vitamin D preparation also known as vitamin D3,may work better to increase vitamin D levels. Vitamin D is important for absorption of calcium from the diet and bone health. Vitamin D more recently has been found to play a role in regulating the normal inflammatory process. Since cystic fibrosis is a state of excessive inflammation, vitamin D may be playing a role in cystic fibrosis. We hypothesize: cholecalciferol will work better to increase vitamin D levels in patients iwth cystic fibrosis and that it will have an effect on markers of inflammation.

A major factor in the respiratory health of Cystic Fibrosis (CF) subjects is the prevalence of chronic Pseudomonas aeruginosa infections. The Pseudomonas aeruginosa infection rate in CF patients increases with age and by age 18 years approximately 85% of CF patients in the US are infected. Liposomal amikacin for inhalation (Arikace™) was developed as a possible treatment for chronic infection due to Pseudomonas aeruginosa in CF patients. The purpose of this double-blind, placebo controlled study is to determine whether Arikace™ is effective in treating chronic lung infections caused by Pseudomonas aeruginosa in Cystic Fibrosis subjects. The study will enroll approximately 300 subjects in clinics in the US, Canada, Europe, Australia and New Zealand. Subjects will be randomized to 590 mg Arikace™ or placebo and will receive treatment for 28 days followed by a 56 day safety follow-up period. The subjects will be required to visit the clinic 8 times (including the Screening visit) over a period of approximately 3 months. No overnight stays at the clinic will be required. At the completion of the TR02-109 protocol, subjects who have consented and meet study safety criteria may enroll in the long-term, open-label, multi-cycle extension study of 590 mg of Arikace™ (under a separate protocol TR02-110).

Portal hypertension (PHT) and its sequelae are the most clinically important manifestation in cystic fibrosis related liver disease (CFLD), with end-stage liver failure as a late and rare manifestation. The aim is to evaluate the safety and efficacy of a pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) for the prophylaxis of variceal bleeding in pediatric CFLD patients with subclinical non-cirrhotic portal hypertension (NCPH)

A Totally Implantable Venous Access Device (TIVAD) that is no longer in use for intravenous therapy, should be flushed at established intervals to promote and maintain patency. No consensus has been established regarding the optimal duration of the interval between 2 maintenance sessions. This exploratory study will focus on catheter status under the current 3-monthly flush regimen.