Families-At-risk for Interstitial Lung Disease Study
Interstitial Lung DiseaseIdiopathic Pulmonary FibrosisThe interstitial lung diseases (ILDs) are a family of closely related lung conditions characterized by alveolar inflammation, injury, and fibrosis not due to infection or neoplasia. While previously considered to be rare, a recent nationwide study found that idiopathic pulmonary fibrosis (IPF), a fibrotic ILD with a median survival of only 3.8 years, affects nearly 0.5% of older adults in the U.S. While pirfenidone and nintedanib slow the progression of IPF, neither reverses fibrosis nor prevents progression of the disease,and no studies to date have tested interventions that prevent the development of fibrotic ILDs.
Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
Cystic FibrosisThis study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects with F/F or F/MF genotypes. The study was discontinued after completion of Part A due to Sponsor's discretion.
Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR...
Cystic FibrosisThis study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
Preoperative Portal and Splanchnic Flow Measurement Using MRI
Liver CirrhosesThis study aims to assess the alteration of portal and splanchnic flow using MRI after liver transplantation in patients with splenomegaly or splenic varix.
Effect of Albumin Infusion on Oxidative Albumin Modification, Albumin Binding Capacity and Plasma...
Decompensated CirrhosisThe aim of this study is to investigate the effect of albumin infusion on oxidative albumin modification, on plasma thiol status and on albumin binding capacity for DS in patients who routinely receive albumin infusion for various indications and to relate these findings with neurohumoral parameters, bacterial products such as endotoxin, and neutrophil function
Tobramycin Delivered by Nebulized Sonic Aerosol for Chronic Rhinosinusitis Treatment of Cystic Fibrosis...
Cystic FibrosisRhinosinusitis1 morePatients with cystic fibrosis frequently develop chronic rhinosinusitis. Bacterial colonization is facilitated by a reduced mucociliary function and some previous studies suggest that the microbiology of the upper airways might influence the microbiology of the lower airway. The aim of this randomized control study is to demonstrate efficacy of antibiotic delivered by nebulized sonic aerosol therapy to decrease the bacterial load in sinuses and medium ostia and to improve the sino-nasal symptoms and endoscopic scores, quality of life and lung function
A 4-week Study to Test Different Doses of BI 1265162 in Adolescents and Adults With Cystic Fibrosis...
Cystic FibrosisThe primary objective of this trial is to assess the efficacy, safety and pharmacokinetics of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler versus placebo in adolescents and adult patients with cystic fibrosis.
Functional Respiratory Imaging and Orkambi in CF
Cystic FibrosisOpen-label study to investigate the effects of Orkambi in CF patients homozygous for the F508del mutation by functional respiratory imaging. Primary endpoints in this study are the changes in Specific airway volumes (siVaw) and Specific Airway resistance (siRaw). A total of 20 ORKAMBI-naive patients with Cystic Fibrosis, homozygous for the F508del mutation will be included in this open label study and will be followed through 3 months of treatment. The treatment will be started after all assessments are performed at visit 1. After the start of the treatment some baseline measurements will be repeated throughout the 3-month treatment period. The patient will be asked to visit the hospital monthly. All study visits should be scheduled around the same time.
CFTR Modulators and Gastrointestinal Complications
Cystic FibrosisGastrointestinal System Disease5 moreTo elucidate the similarities and distinctions in non-pulmonary manifestations of cystic fibrosis (CF) including distal intestinal obstruction syndrome (DIOS) incidence and pancreatic enzyme replacement therapy (PERT) use between US and UK CF populations in a parallel study using data from the UK and US CF registries. To assess how CFTR modulators impacted upon recorded PERT use and incidence of DIOS.
Liver Fibrosis in Zambian HIV-HBV Co-infected Patients
Human Immunodeficiency VirusHBV7 moreIn this study the investigators will determine risk factors for liver fibrosis among HIV-HBV co-infected patients in Lusaka, Zambia, and assess the long-term effectiveness of antiretroviral drugs in the prevention and/or reduction of liver disease.