
Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant...
EmphysemaChronic Obstructive Pulmonary Disease (COPD)6 moreThe purpose of this research study is to learn about the safety of transplanting lungs obtained from non-heart-beating donors (NHBDs) that have been ventilated (attached to a breathing machine or ventilator to deliver oxygen) and perfused with a lung perfusion solution (Steen solution™, made by Vitrolife). This ventilation and perfusion will be done outside the body (ex-vivo) in a modified cardiopulmonary bypass circuit (the kind of device used routinely during most heart surgeries). The purpose of performing ex-vivo perfusion and ventilation is to learn how well the lungs work, and whether they are likely safe to transplant.

Sofosbuvir/Velpatasvir Fixed-Dose Combination in Adults With Chronic HCV Infection and Child-Pugh...
Hepatitis C Virus InfectionThe primary objectives of this study are to evaluate the efficacy, safety, and tolerability of sofosbuvir (SOF)/velpatasvir (VEL) fixed dose combination (FDC) with and without ribavirin (RBV) for 12 weeks and SOF/VEL FDC for 24 weeks in adults with chronic hepatitis C virus (HCV) infection and Child-Pugh-Turcotte (CPT) class B cirrhosis.

Safety Study of Fenretinide in Adult Patients With Cystic Fibrosis
Cystic FibrosisThe purpose of this study is to assess the safety and tolerability of ascending doses of a novel oral formulation of Fenretinide to adult cystic fibrosis (CF) patients, once-daily for 21 days (treatment cycle). This study will include up to three (3) dose levels with minimum 7 day breaks in between treatment cycles. For each dose level, blood samples will be collected for exploratory pharmacokinetic (PK) and pharmacodynamic (PD) evaluation.

Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation
Cystic FibrosisThis Phase 1b study in F508del-CFTR homozygous CF patients is being conducted to assess the safety of N91115 as the sole cystic fibrosis transmembrane conductance regulator (CFTR) modulator at doses near the expected therapeutic exposure level in preparation for Phase 2 studies of N91115 added to the CFTR modulator combination lumacaftor/ivacaftor when launched.

Resistive Inspiratory Manoeuvre as Airway Clearance Technique in Cystic Fibrosis
Cystic FibrosisThis trial aims to analyze whether the resistive inspiratory manoeuvre (RIM) performed previously to autogenic drainage (AD) technique improves the effectiveness in terms of mucus clearance during a typical bronchial drainage session in adults, stable CF patients.

INSPIRE-CF: an Alternative Physiotherapy Model for Children With Cystic Fibrosis
Cystic FibrosisThe primary aim of the research is to evaluate whether an alternative model of cystic fibrosis (CF) physiotherapy care can produce statistically significant improvements in clinical and patient reported outcomes, and whether this alternative model is economically advantageous and/or sustainable. Children randomised to the control group will receive 24-months of current model of CF care at Great Ormond Street Hospital (GOSH). Children randomised to the intervention group will receive 24-months of current model of CF care at GOSH PLUS a weekly structured, individually prescribed and personally supervised exercise intervention at a local fitness facility or at school. The exercise prescription will include aerobic, anaerobic, strength, core conditioning and stretching components. The main objectives of the study are: Determine differences, if any, in lung function between the two groups; Determine differences, if any, in exercise capacity between the two groups; Evaluate cost of care of alternate model of care versus current model of care.

A Multipart, Open-label Study to Evaluate the Safety and Efficacy of ABT-450/r/ABT-267 With and...
Hepatitis C Virus InfectionHuman Immunodeficiency Virus Infection2 moreThe primary objectives of this study are to assess the safety of ABT-450/r/ABT-267 with and without ABT-333 coadministered with and without ribavirin (RBV) for 12 and 24 weeks in HCV GT1- or 4-infected participants with HIV-1 coinfection and to evaluate the percentage of subjects achieving HCV ribonucleic acid (RNA) < lower limit of quantification (LLOQ) 12 weeks following treatment.

Efficacy and Tolerance of Treatment With DHA, Choline and Vitamin E in Children With Non-alcoholic...
Fatty LiverLiver Fibrosis3 moreThe purpose of this interventional study is to evaluate the efficacy and tolerability of docosahexaenoic acid (DHA), Vitamin E and Choline in children or adolescents with well-characterized and liver biopsy confirmed nonalcoholic fatty liver disease (NAFLD).

A Study of IDN-6556 in Subjects With Liver Cirrhosis
Liver CirrhosisHepatic CirrhosisThis is a multicenter study to see if treatment with IDN-6556 can help improve the liver function of patients with liver cirrhosis with Model for End-Stage Liver Disease scores between 11-18.

A Study of IDN-6556 in Cirrhotic Subjects With Portal Hypertension
Liver CirrhosisHepatic Cirrhosis1 moreThis is an open-label pilot study to evaluate the safety, tolerability, and efficacy of IDN-6556 in treating portal hypertension in subjects with liver cirrhosis.