
Study of Individualized Physiotherapy for Airway Clearance in Cystic Fibrosis.
Cystic FibrosisThe purpose of this study is to investigate individual efficacy in Physiotherapy for Airway Clearance, and to investigate user experience, i.e.utility value and preference.

Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous...
Cystic FibrosisThe purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the F508del-CFTR mutation.

A Safety and Tolerability Study of Denufosol in 2-4 Year Olds
Cystic FibrosisThe purpose of this study is to evaluate the safety and tolerability of denufosol 60 mg TID in pediatric CF patients 2 to 4 years of age

Study in Subjects With PAH and PH Secondary to IPF Using Inhaled GeNOsyl.
Pulmonary Arterial HypertensionIdiopathic Pulmonary FibrosisA Phase 2 open label, dose escalation study to find the minimally and maximum effective dose (dose beyond which no further effect on PVR is seen) of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo.

A Study Evaluating the Efficacy and Safety of Pregabalin Against Frequent Muscle Cramp in Patients...
Muscle CrampLiver CirrhosisMuscle cramp is defined as a paroxysmal, involuntary, and painful contraction of skeletal muscle. Cirrhotic patients can encounter with muscle cramp frequently, which might be associated with poor quality of life. Gabapentin can be prescribed for muscle cramp. However, patients with liver cirrhosis have limited access to gabapentin which is metabolized primarily in liver. Pregabalin with a similar mechanism of action to gabapentin undergoes negligible metabolism owing to its improved pharmacokinetic properties. Thus, pregabalin might be a promising therapeutic option for patients with liver cirrhosis who are suffering from muscle cramp and susceptible to drug-induced hepatotoxicity. Therefore, the investigators hypothesize that pregabalin could effectively reduce painful symptoms derived from muscle cramp. In the current study, the investigators are going to evaluate the efficacy and safety of pregabalin by comparing outcomes between two groups (treatment group vs. placebo group).

Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis
Liver CirrhosisBiliaryOBJECTIVES: I. Compare the efficacy of low-dose oral pulse methotrexate (MTX) and ursodiol versus colchicine and ursodiol in patients with primary biliary cirrhosis (PBC). II. Determine the optimum dose and duration of MTX treatment. III. Investigate the role of fibrogenic cytokines (FC) in PBC pathogenesis and the effect of treatment on FC production.

Phase I Pilot Study of Ad5-CB-CFTR, an Adenovirus Vector Containing the Cystic Fibrosis Transmembrane...
Cystic FibrosisOBJECTIVES: I. Assess the safety and efficacy of gene transfer into the nasal epithelium using Ad5-CB-CFTR, an E1-deleted adenovirus vector containing the cystic fibrosis transmembrane conductance regulator gene, in patients with cystic fibrosis (CF). II. Determine whether ion transport abnormalities in CF airway cells can be corrected.

Phase II Study of Lactulose and Circadian Rhythms in Patients With Cirrhosis
Portal HypertensionHepatic Encephalopathy1 moreOBJECTIVES: I. Assess the effect of lactulose on the circadian rhythm of plasma melatonin in patients with subclinical hepatic encephalopathy. II. Assess the intrasubject variability of circadian melatonin levels and neuropsychological tests. III. Assess which elements of the neuropsychological test battery show the response to lactulose.

Study of Pancreatic Enzyme Product in Pediatric Participants With Cystic Fibrosis and Exocrine Pancreatic...
Cystic FibrosisExocrine Pancreatic InsufficiencyThis is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand) pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).

A Study of Tobramycin Inhalation Powder From a Modified Manufacturing Process Versus Placebo
Cystic FibrosisThis study is designed to show how well tobramycin inhalation powder works and how safe it is when produced by a modified manufacturing process