A Study to Assess the Effect of AZD5634 on Mucociliary Clearance, Safety, Tolerability and Pharmacokinetic...
Pulmonary/Respiratory DiseasesThis study will assess the effect of inhaled AZD5634 on Mucociliary clearance (MCC) in patients with Cystic fibrosis (CF) after single-dose administration.
Video Game Exercise Effectiveness of a Domiciliary Pulmonary Rehabilitation Program in Cystic Fibrosis...
Cystic FibrosisThe aim of this study is to evaluate the efficacy of a long-term domiciliary exercise program using the Wii video game platform as a training modality in Cystic Fibrosis (CF) patients. Investigators hypothesize that an exercise program performed by active videogames (AVG) will produce an improvement in exercise tolerance and muscular strength to be maintained over time in young CF patients.
A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12...
Cystic FibrosisThis is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study of tezacaftor in combination with ivacaftor in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.
EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult...
Cystic FibrosisThis study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF.
Omega 3 Supplementation in Cystic Fibrosis Patients
Cystic FibrosisEssential fatty acids (EFA) deficiency has been often reported in patients with cystic fibrosis (CF), particularly in those homozygous for the DF508 mutation. Clinical symptoms of CF may be influenced by correcting EFA deficiency. Nevertheless, the value of EFA supplementation in CF remains controversial. Within this multicentric and international randomized placebo-controlled trial it will be evaluated, according to recommendations of Cochrane analysis, beneficial effects of an oral supplementation with polyunsaturated fatty acids on selected biochemical and functional outcome parameters such as inflammatory biomarkers, incorporation into cell membrane phospholipids, lung function, exercise tolerance, clinical and nutritional status and properties of transepithelial ion transport. The study will be undertaken in a cohort of CF patients aged over 6 years old (60 patients), homozygous for the DF508 mutation and treated by Azithromycine. Supplementation will be performed with a triglyceride source at a daily dose of 60 mg/kg of omega-3 polyunsaturated fatty acids (Omega 3 Premiumâ, Laboratoires Ponroy, France). Before enrolled into the trial and during the study, patients will undergo nutritional assessment by evaluation of total and fat dietary intake and overall calorie intake using a 3-days diet records and a food frequency questionnaire. Plasma and erythrocyte membrane EFA profiles and inflammatory markers will be monitored in baseline conditions, at 3, at 6 and 12 months after starting the treatment. Lung function will be performed at each patient visit and an exercise test will be done before and at the end of the treatment. Properties of ion transport will be searched by sweat testing before and at the end of the treatment.
Improving Adherence and Clinical Outcome in Cystic Fibrosis (CF) Patients
Cystic FibrosisThe purpose of this study is to assess the impact of a collaborative active intervention program of a multi-disciplinary team on improving adherence to chronic medications and improve clinical outcomes in CF patients.
Treatment of Anxiety/Depression and Pain Through Relaxation Yoga for Patient With Cystic Fibrosis...
Cystic FibrosisThe investigators research question is whether or not yoga effectively alleviates symptoms of pain, sleep disturbance, anxiety and depression in children with cystic fibrosis. If yoga does prove to be effective, the investigators will educate our CF population about the benefits they may experience if they choose to incorporate yoga into their CF therapy. The investigators will also go on to do further research of other complementary alternative medicine therapies. This is a pre/post-test study with 20 subjects. The subjects will serve as their own controls. Each subject will participate in six yoga sessions over a ten week period. Subjects' symptoms will be evaluated with the use of questionnaires. The investigators will also test cortisol levels on weeks -2, -1, 1, 6, 7, 8. Cortisol is a hormone that is affected by stress. Testing of cortisol throughout the study will help us determine the subjects' stress level throughout the study.
Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the...
Cystic FibrosisThe purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D-CFTR mutation on at least 1 allele.
Drug-Drug Interaction Study of VX-770 and VX-809 in Healthy Subjects
Cystic FibrosisThe purpose of this study is to assess the pharmacokinetics and safety of VX-809 and VX-770 alone and in combination in healthy subjects.
Control of Steatorrhea in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency...
Exocrine Pancreatic InsufficiencyCystic FibrosisThis study by Aptalis (formerly Axcan) assesses the efficacy and safety of Panzytrat® 25,000 compared to Kreon® 25,000 in the control of steatorrhea in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).