View clinical trials related to Cystic Fibrosis Liver Disease.
Filter by:Portal hypertension (PHT) and its sequelae are the most clinically important manifestation in cystic fibrosis related liver disease (CFLD), with end-stage liver failure as a late and rare manifestation. The aim is to evaluate the safety and efficacy of a pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) for the prophylaxis of variceal bleeding in pediatric CFLD patients with subclinical non-cirrhotic portal hypertension (NCPH)
Cystic Fibrosis (CF) is a genetic condition which affects 1 in 2500 newborn infants and is the commonest genetic condition in the UK. 1 in 25 of the white population carry the mutation. The genetic defect prevents the movement of fluids from cells, leading to thickened secretions and injury. With improvements in treatments from the commonest organ affected, the lungs, patients born with CF now can expect to live into their 40s with more than 60% living past 16. Though better, more can be done. As treatments from lung complications have improved, the management of liver disease (second commonest organ involved) remains unchanged for a considerable time. Treatment options are limited with liver transplant the only curative option. Though potentially life-saving, it has risks and an organ shortage means alternative treatment options are desperately needed. Identifying those with or at risk of Cystic Fibrosis related liver disease is difficult due to inadequate diagnostic tools. Routine blood tests are unreliable; therefore specific blood tests to identify scarring of the liver (biomarkers) are urgently needed. Ultrasound scan, the recommended diagnostic investigation, is only accurate in identifying the late stages of liver disease. For new therapies to be most effective we need to be able to identify patients at a much earlier stage. This study will use multi-modality testing, including imaging techniques such as FibroScan, MRI scan and blood tests (biomarkers), to diagnose those with liver scarring and use this to better categorise disease.
The purpose of this study is to investigate the utility of a continuous glucose monitor device (CGM) in screening for cystic fibrosis related diabetes. The investigators will also study how fat deposition in the pancreas and liver impacts insulin production and response, as measured by a frequently sampled oral glucose tolerance test.
Pilot study to investigate the effect of choline chloride in cystic fibrosis patients with liver steatosis by comparing their status before and after the intervention
To determine if transient elastography (TE), when combined with ultrasound (US) pattern characterization can improve the prediction of progression to a nodular pattern on US. To confirm the feasibility of obtaining TE measurements in children with Cystic Fibrosis (CF) To prospectively assess whether TE data are associated with conventional laboratory markers of hepatic fibrosis To determine the variability of TE measurements taken at different sites in the same patient
Specific Aim 1. Determine if valid results of non-sedated MRI based assessments of liver stiffness and lipid content can be obtained in more than 90% of children and young adults with cystic fibrosis. Specific Aim 2. Determine hepatic lipid content using the HepaFat sequence and liver stiffness using MRE. Investigators will compare the results obtained by MRI with PUSH study grayscale ultrasounds in CF patients with normal, heterogeneous, homogeneous or nodular (cirrhotic) pattern on ultrasound. Specific Aim 3. Creation of an imaging core lab to centralize evaluation of MR imaging data, allow for remote image upload, electronic data storage, and remote image viewing/interpretation. This infrastructure will be utilized to standardize image post processing. Specific Aim 4. Using the longitudinal PUSH study, determine if MRI based imaging improves discrimination of subjects at risk for progression to advanced CF liver disease (development of cirrhosis) compared to using US imaging alone