This study aims to investigate the safety and effectiveness of the Instylla Hydrogel Embolic System (HES) compared to the standard of care TAE/cTACE to treat a tumor by reducing or completely closing off the arterial blood supply to the tumor. The Instylla HES is an experimental treatment. Subjects will be evaluated for 180 days following the embolization procedure. At each visit, subjects will be asked to complete at least one quality of life questionnaire, undergo a physical assessment (vitals only), blood test to check blood cell levels, and follow-up tumor imaging. Throughout the duration of the study, subjects will be evaluated for complications or side effects that may be related to the procedure and/or Instylla HES device. This study will be conducted at up to 20 sites in the U.S. with additional sites outside of the U.S. It is anticipated that the full investigation will take approximately 2 years.
This is a prospective cohort study of subjects with portal hypertension to examine whether increased sphingosine 1 phosphate : ceramide ratio and circulating bile acids are associated with HPS in patients with advanced liver disease. The study will consist of 400 individuals who are evaluated for liver transplantation at the Field Centers. This population has advanced liver disease and will represent the population with cirrhosis at the Centers. As is considered standard of clinical care for these patients and required for liver transplant evaluation, patients will undergo phlebotomy, interviews, pulmonary function testing, echocardiography, and arterial blood gas sampling at their initial evaluation. During the clinical phlebotomy, additional samples will be drawn for research purposes. If any of these procedures does not occur during the clinical visit, it may be conducted for research purposes. Six minute walk testing, frailty scales, SF36, and optional actigraphy, all of which are research-only assessments, will be performed at baseline. Subjects will then be followed via phone for the duration of the study period.
The purpose of this study is to find out about the safety and effectiveness of Belapectin (GR-MD-002) (an oral investigational drug - not approved by the FDA) for the prevention of esophageal varices in NASH (Non-Alcoholic Steatohepatitis) Cirrhosis. The study is designed to last 156 weeks with study visits occurring every 2 weeks.
Alpha-1 Antitrypsin (AAT) is a naturally occurring protein involved in the protection of lungs from inflammation. A mutation in the AAT gene (a change in the body's genetic instructions on how to make AAT) causes it to be made incorrectly and very little of it gets into the bloodstream. Severe AAT deficiency (lack of AAT in the blood) causes emphysema, which causes holes in the lungs. This study is being done to learn more about the safety and effectiveness of VX-864 in patients with Alpha-1 Antitrypsin Deficiency.
The treatment period is 28 days with 3 additional follow-up visits. Participants will be randomized to one of 4 groups. Randomized means there is an equal chance of being placed in any of the 4 groups. The groups are placebo (no active drug) or treatment groups 1-3 with varying doses. All groups will take 5 tablets in the morning and 5 tablets at night. The treatment group will take either 100mg, 300mg, or 500mg tablets twice a day. There is an 80% chance of being assigned to a treatment group and a 20% chance of being assigned to placebo.
This study is testing an investigational (not yet FDA approved) drug called CFZ533 compared to standard of care anti-rejection medications in patients who are having a Liver transplant. This study drug is being tested because it may have fewer long-term side effects than current standard therapy. This study is for first time liver transplant patients. Study drug will be administered every 2 weeks and participation will last for 2 years after transplant surgery.
This is a Phase 3, randomized, double-blind, placebo-controlled study
evaluating the safety and efficacy of GS-9674 in subjects with PSC
The study will consist of an 8-week Screening period,96 weeks of treatment, and a follow-up visit 4 weeks after completion of treatment. (Patients would be required to come to MUSC for 12 visits for a physical check-up, blood work, and to receive more study medication).
If the subject qualifies they will be randomized into one of two groups. The two groups include one receiving 100mg active GS-9674 or placebo. There is a 2/3 chance to be receiving active drug over placebo. The drug GS-9674 is a capsule that is taken once a day in the morning (with or without food).
The purpose of this study is to see if GS-9674 slows the scarring process in subjects with PSC.
The aim of this study is to determine if a long-acting tacrolimus product can reduce the severity and incidence of several neurotoxicities commonly seen after liver transplant. The medications being used in this study are extended release tacrolimus (Envarsus) and immediate release tacrolimus (Prograf). Participants will receive SOC treatment for up to 15 days and no longer than 12 months post-transplant, and then randomized to either Envarsus or Prograf for the 6 month duration of the study. There are 9 study visits that will involve tests, exams and procedures that are both standard of care and study purposes.
This is a two armed multicenter cluster randomized controlled trial (RCT), to assess the effectiveness of two pragmatic PC models for patients with ESLD (Consultative PC vs. Trained hepatologist led PC). To prevent bias at the level of providers, randomization will take place at the level of clinical centers; however patients will be the unit of inference. Parallel to this cluster-RCT, a qualitative study will be undertaken to evaluate the patient/caregiver experiences in the two PC models, using semi structured interviews.
To execute this project, Duke has identified 19 clinical centers to participate; 8 Veterans Health Administration (VHA) systems and 11 non-VHA, Academic Medical Centers.
1.Consultative PC led approach (Model 1): The PC model will include: 1) routine PC consults, using a standardized checklist , 2) in-person visits at initial, 1 and 3 months.
2.Trained hepatologist led PC (Model 2): The Hepatologist Led PC model will comprise: 1) Hepatologist training (through E Learning modules), and 2) in person visits utilizing the same PC checklist as utilized in Model 1. The in-person visits will occur at initial, 1 and 3 months i.e. similar to Model 1 and follow the same visit specified agenda.
MUSC has been assigned to the Model 2 approach, "Hepatologist led Palliative Care" to be lead by Dr. Don Rockey and Dr. Heather Simpson.
Adult patients 18 years of age or older will be enrolled. With 14 clinical centers in different geographic locations and diversity in race/ ethnicity, 1260 patient/ caregiver dyads will be enrolled.
This an observational study that aims to assess the outcome of patients with advanced fibrosis and cirrhosis recurrence in patients with hepatitis C virus (HCV) infection after HCV eradication. We aim to describe this unique group of patients as well as determine whether any particular variables are significant predictors of fibrosis/cirrhosis resolution. A multivariate analysis will be conducted to determine risk factors independently related to fibrosis/cirrhosis resolution and clinical outcome.
Non-Alcoholic fatty liver disease is the most common liver disease, and involves fat deposition in the liver. The fat in the liver can lead to inflammation, scarring, end stage liver disease and potential liver cancer. Some patients with fat in their liver do not see these changes, and our current understanding of why some people are not affected while others see progression of their disease is poor. We are currently in process of initiating studies to learn more about fatty liver disease, and having a database of patients at the VA medical center who are willing to participate in these studies and future studies would help both the patients learn about the new and upcoming therapies, and help the clinical investigators to quickly screen their patients and invite them to participate in their studies.