Active clinical trials for "Cystic Fibrosis"

This is a Phase 2 open-label, dose-escalation study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele. In total, up to 16 patients will be enrolled in the trial; up to 4 patients will be homozygotes for G542X, and the remaining patients will be compound heterozygotes with one G542X or phenotypically similar nonsense allele and any Class 1 or Class 2 mutation. Each patient will receive up to 5 escalating doses as follows: ELX-02 0.3 mg/kg per day SC ELX-02 0.75 mg/kg per day SC ELX-02 1.5 mg/kg per day SC An individualized dose of ELX-02, as high as 3.0 mg/kg per day SC, based on the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests. ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid

This is a phase 1b multiple ascending dose escalation study to evaluate the safety and tolerability of arginase inhibitor CB-280 in subjects with cystic fibrosis.

Chest physiotherapy plays a crucial role in treatment of lung disease in cystic fibrosis (CF). New airway clearance techniques (ACTs) adapted to individual needs are still being sought to achieve the best effect of airway clearance. The primary aim of this study is to assess the efficacy of a new ACT (Simeox) on pulmonary function in children with CF. 40 CF patients with stable respiratory function will be randomized 1:1 to Simeox or conventional chest physiotherapy (CCPT) therapy (control group) and treated at home during 1 month. After a short washout period, patients will be treated at home onto the alternative treatment for 1 month (crossover design). Lung function, quality of life, pulmonary exacerbation and safety will be evaluated at 1 month for each therapy period.

The investigators will recruit 15 patients with cystic fibrosis 18 years of age and older who present with constipation. The investigators will assess baseline motility symptoms with a survey. Patients will then ingest a SmartPill (trademark) to obtain baseline motility within the GI lumen. All patients will undergo intervention with taking polyethylene glycol (PEG) or Miralax (brand name) 17 grams once daily. After two weeks of therapy, the patient will repeat the motility survey and again ingest a smart pill to assess the change in motility symptoms while on therapy.

Cystic fibrosis and bronchiectasis are the most seen problems in children with chronic pulmonary diseases. İt is a genetic, chronic system disease that reduces life expectancy, and life quality as well. Chronic lung disease , malnutrition, and reduced activity, caused by disease lead to postural disorders. Muscle force, endurance, activity of Daily living are adversely affected. İn the treatment pulmonary rehabilitation are using. Airway clearance technique, pulmonary exercises, upper extremity ergometer, dumbbells, elastic bands, proprioceptive neuromuscular facilitation technique are applying. Resistance training using elastic bands has become an increasingly common intervention aiming to improve function by increasing muscular strength. İn one study indicated that muscle strength can be improved through three dimensional spiral large scale resistive exercises using proprioceptive neuromuscular facilitation. İn the literature there isn't any research , uses elastic bands with proprioceptive neuromuscular facilitation for the upper extremity and evaluating pulmonary functions, posture, quality of life, muscle force. The aim of this study is giving exercises programme with proprioceptive neuromuscular facilitation and elastic bands and to evaluate pulmonary muscle force, pulmonary functions, posture, activity of daily living, quality of life, functional capacity. And to evaluate the effect of treatment programme on these parameters. The subjects were divided in two groups. An experimental group three times a week for 12 weeks will perform proprioceptive neuromuscular facilitation exercises with elastic bands, and pulmonary exercises. The control group will apply only pulmonary exercises at home programme.

Two parts, two periods, crossover study with part 2 is optional. In both parts, subjects will be randomized to sequentially receive both sublingual and oral formulations of FDL169.

This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

The trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.

This is a multi-center, randomized phase III study to evaluate the clinical effectiveness of AeroVanc in persistent methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic fibrosis (CF).

The aim of this study is to assess the effectiveness of an Airway Clearance System.