A Phase 2 Randomized, Controlled, Dose-titration, Open-Label Study Evaluating the Safety and Efficacy of BIV201 in Addition to Standard of Care Compared to Standard of Care to Reduce the Recurrence of Ascites and Complications in Patients with Refractory Ascites Secondary to Decompensated Liver Cirrhosis

Date Added
May 5th, 2021
PRO Number
Pro00109448
Researcher
David Koch

List of Studies


Keywords
Liver
Summary

The purpose of this research study is to evaluate the efficacy of BIV201 continuous infusion with SOC compared to SOC alone in adult patients with refractory ascites secondary to decompensated hepatic cirrhosis.

Cirrhotic patients with refractory ascites have a very poor prognosis and suffer from complications due to the continuous buildup of ascites fluid in their abdomens. There are currently no approved pharmacological treatments for refractory ascites and first-line standard of care is limited to repeated mechanical removal by paracentesis, with liver transplantation the only definitive treatment. The vasoconstrictor terlipressin, which is not yet approved in the United States, reduces splanchnic vasodilation associated with portal hypertension in cirrhotics and can lead to a decrease in ascites.

This exploratory dose-titration trial is designed to evaluate the therapeutic efficacy of BIV201 continuous infusion on ascites recurrence and clinical complications of decompensated cirrhosis with refractory ascites, and to explore the hypothesis that if ascites recurrence or accumulation is reduced during the intervention period, consisting of two 28-day treatment periods separated by a wash-out
interval, there will be a better clinical outcome (reduction in complications) long-term over 180 days than in those treated with SOC alone

Institution
MUSC
Recruitment Contact
Christian Conley
843-876-4273
conleyc@musc.edu

MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) MARCH-PFIC

Date Added
April 13th, 2021
PRO Number
Pro00108203
Researcher
Nagraj Kasi

List of Studies


Keywords
Liver, Pediatrics
Summary

This is a randomized Double-blind Placebo-controlled Phase 3 Study to determine whether a study drug called maralixibat is safe and effective in treating itchy skin (pruritus) in children with Progressive Familial Intrahepatic Cholestasis (PFIC). There is currently no treatment approved for PFIC and available medical approaches have limited success.

Randomized means by chance, like the flip of a coin. Subjects will be randomly assigned to one of the two arms of the study. Subjects will have a 50:50 chance of receiving maralixibat as compared to placebo.

Double blinded means a study in which neither the investigators nor the subjects know which intervention the subject is receiving.

A Phase 3 study is large scale trial to confirm and expand information on safety and usefulness of a new drug.

Subjects who qualify to participate in this study will be randomly assigned to receive the study drug or a placebo. The dose of the study drug will be gradually increased during the study upto a tolerable dose . Subjects will continue to take the medication for about 5 months. Subjects will have approximately 9 study visits over 8 months in this study. Subjects will also be asked questions about health and complete questionnaires at the study visits and as well as at home. A Physical exam, Electrocardiogram, Liver ultrasound will be performed at some study visits. A blood and urine sample will also be collected at some of the study visits.

It is possible that the maralixibat may or may not improve symptoms from PFIC. Even if there is no benefit, other children may benefit from what is learned in this study.

Institution
MUSC
Recruitment Contact
Kenreka Yeadon
843-792-7965
yeadon@musc.edu

The effect of semaglutide in subjects with non-cirrhotic non-alcoholic steatohepatitis

Date Added
April 13th, 2021
PRO Number
Pro00108809
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

The purpose of this study is to find out about the safety and effectiveness of an investigation drug called Semaglutide for the treatment of NASH. (Non-Alcoholic Steatohepatitis). NASH occurs when the fat buildup in the liver leads to inflammation (hepatitis) and scarring. NASH is associated with increased risk of morbidity (medical problem or complication) and mortality (death). Currently, treatment options are few and insufficient. There is therefore an unmet medical need for effective and safe pharmacological treatment options. The study is designed to last 257 weeks (approximately 4 years and 11 months), with study visits occurring approximately every 4 weeks. Most visits will include blood work and some will include assessments such as body weight and vital signs. Most visits will include reviewing of diary entries during the course of the study. This study also includes weekly injections of semaglutide (or placebo). Semaglutide is a self-administered injection that is given under the skin. Semaglutide has built an extensive amount of data with other trials that have focused on weight management and Type 2 diabetes. Semaglutide is FDA-approved for diabetes treatment, but is investigational for this study. In these previous trials, semaglutide was found to be safe and well-tolerated. This study is randomized, double-blinded, and placebo-controlled. This means that you may receive the study drug or a placebo. Neither the study subject or the study team members will know which each subject will be receiving. Study subjects will be randomized 2:1. This means that subjects will have a greater chance (66%) of receiving the drug versus the placebo.

Institution
MUSC
Recruitment Contact
Bridgette Blankenship
843-876-8439
blanke@musc.edu

Randomized Multi-Center, Subject and Evaluator Blinded, Parallel- Group Study to Evaluate the Safety and Effectiveness of the Instylla Hydrogel Embolic System (HES) Compared with Standard of Care Transcatheter Arterial Embolization (TAE) / Transcatheter Arterial Chemoembolization (cTACE) for Vascular Occlusion of Hypervascular Tumors; A Pivotal Study

Date Added
February 23rd, 2021
PRO Number
Pro00106547
Researcher
Marcelo Guimaraes

List of Studies


Keywords
Kidney, Liver, Surgery, Vascular
Summary

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.

Institution
MUSC
Recruitment Contact
Elizabeth Kelley
843-792-2305
kelleyel@musc.edu

Pulmonary Vascular Complications of Liver Disease 3 (PVCLD3)

Date Added
January 22nd, 2021
PRO Number
Pro00103260
Researcher
David Koch

List of Studies


Keywords
Hypertension/ High Blood Pressure, Liver, Men's Health, Pulmonary, Transplant, Vascular
Summary

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.

Institution
MUSC
Recruitment Contact
Christian Conley
(843)876-4273
conleyc@musc.edu

A Seamless, Adaptive, Phase 2b/3, Double-Blind, Randomized, Placebo-controlled, Multicenter, International Study Evaluating the Efficacy and Safety of Belapectin (GR-MD-02) for the Prevention of Esophageal Varices in NASH Cirrhosis

Date Added
October 13th, 2020
PRO Number
Pro00100506
Researcher
Don Rockey

List of Studies


Keywords
Aging, Liver
Summary

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.

Institution
MUSC
Recruitment Contact
Megan Bickford
843-876-8439
liverstudies@musc.edu

A 12-month, open-label, multicenter, randomized, safety, efficacy, pharmacokinetic (PK) and pharmacodynamic (PD) study of two regimens of anti-CD40 monoclonal antibody, CFZ533 vs. standard of care control, in adult de novo liver transplant recipients with a 12-month additional follow-up and a long-term extension (CONTRAIL I)

Date Added
February 25th, 2020
PRO Number
Pro00089195
Researcher
Derek DuBay

List of Studies


Keywords
Liver, Transplant
Summary

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 up to 6 years after transplant surgery.

Institution
MUSC
Recruitment Contact
Tamara Jenkins
843-792-1851
saundert@musc.edu

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety, Tolerability, and Efficacy of GS-9674 in Non-Cirrhotic Subjects with Primary Sclerosing Cholangitis

Date Added
June 25th, 2019
PRO Number
Pro00087383
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

This is a Phase 3, randomized, double-blind, placebo-controlled study
evaluating the safety and efficacy of GS-9674 in subjects with PSC
without cirrhosis.

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.

Institution
MUSC
Recruitment Contact
Brittiny Bowers
843-876-8439
liverstudies@musc.edu

The effect of conversion to once-daily Envarsus on the neurologic toxicity burden in liver transplant recipients

Date Added
April 9th, 2019
PRO Number
Pro00083855
Researcher
Derek DuBay

List of Studies


Keywords
Liver, Transplant
Summary

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.

Institution
MUSC
Recruitment Contact
Courtney Rowley
843-792-7082
rowle@musc.edu

Introducing Palliative Care (PC) within the Treatment of End Stage Liver Disease (ESLD): A Cluster Randomized Controlled Trial

Date Added
December 18th, 2018
PRO Number
Pro00084674
Researcher
Don Rockey

List of Studies


Keywords
Alcohol, Cancer/Gastrointestinal, Liver, Pain
Summary

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.

Comparative Approaches:
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.

Institution
MUSC
Recruitment Contact
Joshua Inman
843-876-4303
inmanj@musc.edu



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