Active clinical trials for "Fibrosis"

Duchenne muscular dystrophy (DMD) is the most common and devastating form of muscular dystrophy, caused by an X-chromosome gene mutation resulting in the absence of the protein dystrophin. Gene therapy by exon skipping or stop codon read-through and cell therapy are at the stage of clinical assays with very promising results. Nevertheless, they will not allow a complete cure of DMD patients and they will concern only specific types of mutations. It is therefore crucial to develop other therapeutic strategies related to the natural history of the disease and targeted not on the dystrophin itself, but on the consequences of its absence. Another crucial pathophysiological pathway in DMD is muscle cell calcium homeostasis, particularly via the ryanodine recepteur (RyR1). Our study focus on the relationship between endomysial fibrosis, abnormal inflammation response and calcium homeostasis dysfunction which are not entirely established in DMD. The identification of the biological mechanisms that play a role in the severity of the phenotype, particularly endomysial fibrosis, should allow the development of targeted pharmacotherapy as a complementary strategy for the future treatment of DMD.

The purpose of this study is to test the accuracy of urinary neutrophil-gelatinase associated lipocalin (NGAL) and other biomarkers (plasma renin, norepinephrine) to predict acute kidney injury (AKI) development in patients with cirrhosis and bacterial infection and to predict response to AKI treatment with albumin and albumin with terlipressin in patients with suspected hepatorenal syndrome.

Patients with cirrhosis can have abnormalities in laboratory tests reflecting changes in primary and secondary haemostasis. Such changes have been considered particularly relevant in the bleeding complications that occur in cirrhosis. However, several studies have shown that routine diagnostic tests are not clinically useful to stratify bleeding risk in patients with cirrhosis. Moreover, treatments used to increase platelet count or to modulate platelet function could potentially do harm. Consequently the optimal management of bleeding complications is still a matter of discussion. Moreover, in the last two decades there has been an increased recognition that not only bleeding but also thrombosis complicates the clinical course of cirrhosis. Over the last years, emerge that in vivo platelet function and coagulation cascade might be modulated by an alteration of pro-oxidant and antioxidant balance. Thus It has previously been demonstrated that chronic liver diseases are characterized by increased oxidative stress state. Aim of the study is to analyse the relationship between oxidative stress, haemostatic balance and clinical complications in cirrhosis.

In patients with liver cirrhosis elevated levels of von Willebrand factor antigen (vWF-Ag) are found frequently but the clinical significance is unclear. vWF-Ag plays an important role in primary haemostasis and development of thrombotic vascular obliteration is discussed as a possible mechanism leading to portal hypertension. Invasive measurement of hepatic venous pressure gradient (HVPG) is the current gold standard for the diagnosis of portal hypertension. The investigators hypothesize that vWF-Ag levels in plasma may correlate with portal pressure and predict clinically significant portal hypertension (CSPH, HVPG >=10mmHg) and its complications.

The purpose of this study is to identify genetic determinants of susceptibility to liver cirrhosis and hepatocellular carcinoma. It will assist in predicting individual risks of disease progression and would help to clarify pathophysiologic mechanisms of liver cirrhosis and hepatocellular carcinoma.

It has been shown that neutrophils (a specific type of cell) are involved in inflammation in the lungs of CF patients. Neutrophil levels in CF patients have been measured by bronchoalveolar lavage (BAL), which samples cells in the fluid lining of the lungs. Other studies have measured neutrophil levels and inflammation in other parts of the body using PET scanning. This study aims to show that PET scanning can be used as a non-invasive marker of inflammation in the lungs of patients with CF, which would be a useful tool in treatment. The primary goal of this study is to draw a connection between the level of inflammation shown in the PET scan and the number of neutrophils obtained from the BAL. This study will also look at how the PET images relate to inflammatory molecules in the lungs and to the FEV-1 obtained through spirometry.

AIM: To evaluate the role of Strain Elastography in the assessment of liver fibrosis in chronic hepatopathy

Cystic Fibrosis (CF) is a chronic disease characterized by recurrent pulmonary infections and exocrine pancreatic insufficiency. The vast majority of patients with CF will develop pancreatic endocrine insufficiency over time manifested as altered glucose metabolism. The presence of overt diabetes in patients with CF is associated with adverse clinical outcomes. The underlying pathophysiology of cystic fibrosis related diabetes (CFRD) is still a matter of investigation. In addition to localized tissue damage developing similar to that of the exocrine pancreas, additional mechanisms may be involved. The investigators have recently shown that insulin secretion in patients with CF is significantly altered prior to the development of diabetes. This phenomenon is associated with reduced secretion of gut derived incretins (specifically GIP). The blunting of incretin induced insulin secretion (whether due to a deranged interaction of gastrointestinal contents with enterocytes resulting in reduced secretion or due to rapid clearance of such peptides) may be a major underlying driver of altered glucose metabolism in such patients.

The purpose of this study is to determine the effect of transjugular intrahepatic portosystemic shunt on gut microbiota and associated inflammatory factors in cirrhotic patients.

Cystic fibrosis (CF) is characterized by airway inflammation and infection leading to progressive destruction of lungs. One of the most important abnormalities in CF is an abnormal processing of the mutated CFTR protein through the endoplasmic reticulum that causes abnormal location or even absence of the protein at the apical plasma membrane of airway epithelial cells. This abnormality results in a marked dehydration of the airway surface fluid, decreased mucus transport and airway obstruction. Nevertheless, the events that occur very early during the progression of the disease at the airway level in infants are not known. At cellular level, it has also been reported that the CFTR expression and localization could be related to the differentiation state of the airway epithelium. Furthermore, it has been reported that gap junctions could be involved in dysregulate inflammation process. In CF infants, many answers are still lacking. For a better understanding of the early stages of cystic fibrosis, it is of major interest to study respiratory epithelial cells obtained as early as possible. In 15 CF infants and 15 control infants, a nasal brushing will be performed by means of a soft sterile cytology brush. Samples will be used for cytological and functional studies: ciliary beating frequency, cAMP-dependent chloride efflux, potassium efflux, tight and gap junctions functionalities. These studies will be done in basal conditions and will be repeated after activation of the nasal epithelial cells by the bacteria, such as Staphylococcus aureus, which can be found very early in the course of CF disease.