Active clinical trials for "Cystic Fibrosis"

This was a multicenter, randomized, double-blind, placebo-controlled study of patients with severe, though stable, cystic fibrosis (CF) whose routine treatment included Pulmozyme. Patients were randomized to either continue Pulmozyme or have therapy withdrawn for 2 weeks (placebo group). Patients must have had stable CF symptoms without any change in therapy for 2 weeks prior to enrollment in order to participate.

This is a prospective, single-center, nonrandomized observational study to investigate the utility of urine lipoarabinomannan (LAM) as a test to identify individuals at very low risk for having a sputum culture positive for Nontuberculous Mycobacteria (NTM). The study is designed to evaluate if a urine test can eliminate the need for obtaining a sputum specimen to screen for NTM in individuals with Cystic Fibrosis (CF). The participants will be asked to provide 3 urine samples either in person or by mail over approximately 3 years. The 3 urine sample requests will be timed to coincide with their usual clinical care and routine sputum collection.

This research is being done to find the genes and other factors that are responsible for differences among persons with cystic fibrosis. We are particularly interested in the factors that relate to the development of Cystic Fibrosis Related Diabetes (CFRD).

To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation

The purpose of the study is to assess the efficacy of Alginate oligosaccharide (OligoG) dry powder for inhalation in cystic fibrosis (CF) patients with a Burkholderia spp. infection.

Insulin replacement therapy may be effective in breaking the cycle of protein catabolism, undernutrition and overall clinical deterioration in pre-diabetic, insulin insufficient CF youth because of its potent anabolic effect. A significant number of CF patients might benefit from this therapeutic approach with a substantial impact on morbidity and mortality.

Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB use, in patients with cystic fibrosis and exocrine pancreatic insufficiency.

Evaluate the safety and tolerability of VX-659 in healthy subjects

Physical activity (PA) in individuals with cystic fibrosis (CF) improves exercise capacity, slows decline in lung function, increases mucus clearance and improves health-related quality of life (HRQoL). Establishing and maintaining an exercise routine remains challenging and programs promoting PA in people with CF have poor participation. Moreover, while the positive effects of physical conditioning on lung function have been well reported, conventional measurements of lung function may lack the sensitivity to reveal improvement in mild lung disease. This randomized control trial (RCT; N = 60) evaluates the Do More, B'More, Live Fit, a 6-month fitness program designed to optimize exercise habits of 12-21 year-olds with CF through structured exercises with personalized coaching, exercise equipment including the FitBit Flex, online support and motivational messages delivered electronically. The intervention incorporates fitness preferences and encompasses endurance, strength and flexibility exercises while adjusting to physical fitness needs. The hypothesis is that intervention participants will have increased and sustained engagement and better health outcomes compared to control group participants. The investigators' specific aims are to: Increase daily PA and measures of fitness Improve lung clearance index (LCI) and participant HRQoL Demonstrate feasibility, accessibility and satisfaction of intervention using semi-structured interviews The results of this pilot evaluation of the Do More, B'More, Live Fit program will offer novel insight into factors that sustain engagement in exercise programs and identify if LCI is an appropriate clinical outcome to assess PA interventions. Results will inform future RCT of interventions to optimize exercise habits of adolescents with CF.

This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate 4 different doses of GLPG2222 administered for 4 weeks to adult subjects with a confirmed diagnosis of CF and homozygous for the F508del Cystic Fibrosis Transmembrane conductance Regulator (CFTR) mutation.