PEP and OPEP Devices in Cystic Fibrosis
Cystic FibrosisCystic fibrosis (CF) is the most common severe autosomal recessive genetic disease in Caucasians. The CFTR (cystic fibrosis transmembrane regulator) gene, which encodes the chloride channel of the epithelial cell membrane, is responsible for the development of the disease. Respiratory physiotherapy, especially bronchial drainage is one of the basic elements of comprehensive management in patients with CF. Among the many procedures used in CF drainage physiotherapy, the most commonly include these using positive expiratory pressure (PEP) and oscillating positive expiratory pressure (OPEP). The aim of the study is to assess the efficacy of the usage of selected PEP and OPEP devices in bronchial drainage in cystic fibrosis patients during exacerbation of the disease.
Single and Multiple Dose Escalation of PHIN-214 in Child-Pugh A and B Liver Cirrhotics
CirrhosisLiver2 moreThis single and multiple ascending dose (SAD and MAD) study evaluates PHIN-214, being studied to determine the safety, tolerability, and pharmacokinetics, and establish the maximum tolerated dose of this compound in patients with Child Pugh A and B Cirrhosis.
Intestinal Microbiota Transplant in Alcohol-Associated Liver Disease
Liver Disease; Alcohol-RelatedCirrhosis1 moreThe purpose of this research study is to test the safety, tolerability, and effectiveness of the capsules that contain bacteria from healthy individuals when used to treat alcohol craving and drinking.
Symptom Based Performance of Airway Clearance After Starting Highly Effective Modulators for Cystic...
Cystic FibrosisMucociliary Clearance DefectCystic Fibrosis (CF) is an autosomal recessive disease cause by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) manifesting in multiple organs, the most common cause of morbidity and mortality continues to be the pulmonary manifestation. CFTR dysfunction leads to reduced mucociliary clearance, impaired innate immune system function in the lungs (within the airway surface liquid [ASL] lining the epithelial barrier of the lungs) and reduced ASL hydration (stickier mucus). To try and help correct this underlying defect patients have been performing airway clearance for decades using different techniques (Percussion and postural drainage [P&PD], Positive expiratory pressure [PEP], Oscillatory positive expiratory pressure [OPEP], High-frequency chest compression [HFCC], exercise), inhaled mucolytics (Hypertonic Saline, Pulmozyme) and inhaled antibiotics. However, performing daily airway clearance can be a large burden on patients and their families with a median number of daily therapies around 7 and average time spent on therapies at almost 2 hours daily. This high treatment burden leads many patients to have reduced adherence to their regimens and multiple studies have shown around 20% of patients performing no daily airway clearance. Since the release of highly effective CFTR modulator therapy patients have experienced improvements in lung function measurements and imaging-based ventilation measurements, reduction in pulmonary exacerbations, and improvement in daily symptom scores. Over 80% of patients and their families and over 95% of clinicians in the United States support the idea of trials looking into the simplification of airway clearance regimens. Combining the inability of most patients to complete their daily regimens, patient and clinician interest in treatment simplification research, and the overwhelming cost of most inhaled medications in cystic fibrosis with the improvement in mucociliary transport and symptoms with highly effective modulator therapy suggests a research program aimed at reducing the treatment burden of daily airway clearance should be considered. The investigators propose the following: determine if there is additional benefit in continuous airway clearance regimens after starting Elexacaftor-Tezacaftor-Ivacaftor (ETI) and if so, is this benefit noticeable on pulmonary function testing and imaging.
Clinical Trial to Evaluate the Safety and Efficacy of DWN12088 in Patients With IPF
Idiopathic Pulmonary FibrosisThis is a randomized, double-blinded, placebo-controlled multicenter study to evaluate the safety and efficacy of DWN12088 in patients with Idiopathic Pulmonary Fibrosis.
Effect of CPAP on Abnormal Gastroesophageal Reflux and Lung Inflammation in IPF
Idiopathic Pulmonary FibrosisObstructive Sleep Apnea1 moreThis study will evaluate the effect of CPAP therapy on esophageal pH and lung inflammation in patients with idiopathic pulmonary fibrosis (IPF) and sleep apnea.
Prednisone in Cystic Fibrosis Pulmonary Exacerbations
Cystic Fibrosis Pulmonary ExacerbationThis will be a 5 year randomized, double blind, placebo controlled trial of 7 days of oral prednisone in cystic fibrosis (CF) patients receiving intravenous (IV) antibiotic treatment for a pulmonary exacerbation at the Hospital for Sick Children and other study sub-sites across Canada. The intervention will be oral prednisone 2 mg/kg/day (max 60 mg) divided twice daily for 7 days as an adjunctive therapy for pulmonary exacerbations in CF patients who have not recovered their baseline forced expiratory volume in 1 second (FEV1) after 7 days of IV antibiotic treatment. The primary outcome will be the proportion of subjects who achieve >90% of their baseline FEV1 % predicted at day 14 of IV antibiotic treatment for a pulmonary exacerbation in each treatment arm.
Study of Orphenadrine' in the Treatment of Muscle Cramps in Patients With Cirrhosis
Liver CirrhosisMuscle CrampsMuscle cramps are common in patients with liver disease and associated with significantly diminished quality of life. Patients with cirrhosis often experience muscle cramps with varied frequency and severity. The exact mechanisms by which they occur remain unclear, although a number of pathophysiological events unique to liver disease may contribute. Clinical studies have identified alterations in 3 areas: nerve function, energy metabolism, and plasma volume/electrolytes (1) Orphenadrine is an anticholinergic drug with prominent central nervous system (CNS) and peripheral actions used to treat painful muscle spasms and other similar conditions. The combination of anticholinergic effects and CNS penetration make orphenadrine useful for pain of all etiologies, including from: radiculopathy, muscles, and headaches. [3,4]
Body Composition Changes After TIPS and Associated Clinical Outcomes
CirrhosisLiver1 moreThe purpose of this study is to prospectively assess the impact of TIPS creation on muscle mass and physical function in patients with cirrhosis, and to determine whether these changes correlate with improved outcomes in patients awaiting liver transplantation. Retrospective observational studies have shown improvement in muscle mass and body composition in cirrhotic patients undergoing TIPS. The investigators aim to now prospectively study this through a pilot randomized controlled trial tracking patients managed with TIPS creation compared to those managed without TIPS to determine whether these observational findings can be seen in a randomized cohort. The investigators hypothesize that TIPS creation will lead to improved muscle mass, body composition and muscle function within the first 12 months after the procedure compared to a control group without TIPS, and that these changes will improve liver disease outcomes in patients awaiting liver transplantation.
The Effect of Laparoscopic Splenectomy and Azygoportal Disconnection on Liver Reserve Function for...
CirrhosisLiver2 moreIn this study, the investigators compared the improvement of liver reserve function related indicators in patients with liver cirrhosis after laparoscopic splenectomy and azygoportal disconnection. To determine whether surgical treatment can help enhance postoperative liver reserve function and improve patient prognosis.