Active clinical trials for "Cystic Fibrosis"

The objectives of the study are to demonstrate that Tobrineb®/Actitob®/Bramitob® is non-inferior to TOBI® in the primary efficacy variable, forced expiratory volume in one second (FEV1) percent of predicted normal, and to compare the safety in participants with cystic fibrosis and chronic infection of the lungs with Pseudomonas aeruginosa.

The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.

This Phase IIB proof-of-concept study would examine the effects of an investigational product called N-acetylcysteine (NAC) on the basic processes that cause inflammation in CF lung disease. We hope to learn more about the causes of lung disease in cystic fibrosis by studying the characteristics of the inflammation in the lungs of patients who have CF.

The majority of cystic fibrosis (CF) patients die from a progressive pulmonary disease.Airway inflammation plays a major role for the pathogenesis of CF lung disease, and ultimately leads to lung destruction. The release of oxidants during the inflammation process leads to a chronic imbalance of oxidants and antioxidants and may be a central component leading to irreversible lung damage in CF patients. The antioxidant glutathione, which is a naturally occurring tripeptide, is depleted in the extracellular epithelial lining fluid of the CF lung. The elevation of reduced level to normal and also the augmentation of glutathione concentrations above the normal level, as observed in smokers and during defence of Pseudomonas infection, may be desirable to avoid lung damage. Data from pilot studies in humans and animals have indicated that the glutathione concentrations in epithelial lining fluid can be elevated by aerosol application. The main objective of this trial is to evaluate the effect of a 24-week treatment with inhaled glutathione compared with control inhalations (normal saline) on pulmonary function in adult and pediatric CF patients. Secondary objectives are to determine the effects of inhaled glutathione on inflammatory variables, glutathione levels and free elastase in induced sputum and to evaluate the safety and tolerability of the 24-week treatment with inhaled GSH. There is considerable hope within the CF community that the addition of anti-oxidative therapy to an already comprehensive program for treating the lungs will decrease morbidity and improve the quality of life for patients with CF.

Our data indicate that the CFTR-molecule functions as a transporter for sphingosine-1-phosphate and sphingosine or regulates the uptake of these sphingolipids by epithelial cells. The disturbed uptake of sphingosine and sphingosine-1-phosphate over the cell membrane results in an accumulation of ceramide in the cell membrane, which finally triggers a pro-inflammatory and pro-apoptotic status in the respiratory tract of cystic fibrosis patients. Amitriptyline reduces the cera-mide levels in the lung tissue, normalises the activity of cytokines and prevents constitutive cell death of epithelial cells observed in CFTR-deficient mice. Most important, amitriptyline prevents pulmonary infections of CFTR-deficient mice with P. aeruginosa. These effects of amitriptyline may result in an improved lung function of cystic fibrosis patients.

This study will assess the effect of pancrelipase delayed release 24,000 unit capsules on fat and nitrogen absorption in subjects with PEI due to Cystic Fibrosis.

Subject with Cystic Fibrosis have increased clearance of many drugs. Based on pre-clinical data SB656933 was found to have low clearance and high bio-availability. This study will characterize the PK profile of a single dose of SB656933 in patients with Cystic Fibrosis. There will be two groups of subjects. The first group of subjects will receive a single dose of 50mg SB-656933. The second group of subjects will receive a single dose of up to 300 mg SB-656933. Subjects will first be screened for eligibility related to cystic fibrosis history. Safety evaluations will be undertaken and plasma samples for pharmacokinetic analysis will be collected. Additional blood samples will be taken for the pharmacodynamic endpoints CD11b and GAFS. Subjects are not required to stay overnight after their 12 hour PK sample collection on Day 1, although they may do so if they wish. On Day 2 and 3, they will return for collection of additional safety measurements, and further plasma and blood samples will be taken for 24 and 48 h pharmacokinetics and 24h CD11b/GAFS measurements, respectively. A follow up visit (Visit 3) will be made 4-7 days after the treatment period. Subjects will be enrolled in the study for approximately 3to 7 weeks (from screening to follow-up).

Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to eradicate and been implicated as the major cause of morbidity and mortality in CF patients. Aerosol delivery of antibiotics directly to the lung increases the local concentrations of antibiotic at the site of infection resulting in improved antimicrobial effects compared to systemic administration. Bacterial resistance to current aerosol antibiotic treatments indicate a need for improved therapies to treat CF patients with pulmonary infections caused by multi-drug resistant Pseudomonas aeruginosa and other bacteria. High concentrations of MP-376 delivered directly to the lung are projected to have antimicrobial effects on even the most resistant organisms. MP-376 is a novel formulation of the fluoroquinolone levofloxacin that has been optimized for aerosol delivery using the PARI electronic eFlow® nebulizer. Preclinical and early clinical studies in adults show that aerosol doses of MP-376 appear to be safe and well tolerated, and exert an antimicrobial effect when administered once or twice daily. High concentrations of levofloxacin in the lung delivered using MP-376 are expected to be active against CF pathogens such as P. aeruginosa and S. aureus, including those resistant to aminoglycosides (such as TOBI®) and other inhaled antimicrobial agents. Inhaled MP-376 can be delivered rapidly and efficiently using the PARI eFlow® nebulizer system. This Phase 1 study is being performed to obtain safety, tolerability and PK data in children ages 6-16 in order to determine if MP-376 is safe, prior to enrolling children of these ages in the planned pivotal Phase 3 studies.

To evaluate the change in forced expiratory volume (FEV1) from baseline to Day 28-30 between Cipro Inhale-treated and placebo-treated subjects after a 4-week treatment period.

Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to eradicate and been implicated as the major cause of morbidity and mortality in CF patients. Aerosol delivery of antibiotics directly to the lung increases the local concentrations of antibiotic at the site of infection resulting in improved antimicrobial effects compared to systemic administration. Bacterial resistance to current aerosol antibiotic treatments indicate a need for improved therapies to treat CF patients with pulmonary infections caused by multi-drug resistant Pseudomonas aeruginosa and other bacteria. High concentrations of MP-376 delivered directly to the lung are projected to have antimicrobial effects on even the most resistant organisms.