A Randomized, Double-blind, Placebo-controlled, Phase 2b study to Evaluate Safety and Efficacy of DUR-928 in Subjects with Alcoholic Hepatitis

Date Added
September 14th, 2021
PRO Number
Pro00109938
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

There is a new investigational drug called DUR-928. This study is being done to see if this investigational drug helps in the treatment of Alcoholic Hepatitis (AH).

The hope is that the use of DUR-928 will lead to lowering the MELD (Model for end-stage liver disease) score, some other liver blood tests, and then other symptoms of AH may get better.

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

A Double-blind, Randomized, Placebo-Controlled Study and Open-label Long Term Extension to Evaluate the Efficacy and Safety of Elafibranor 80 mg in Patients with Primary Biliary Cholangitis with Inadequate Response or Intolerance to Ursodeoxycholic Acid

Date Added
July 13th, 2021
PRO Number
Pro00110190
Researcher
Don Rockey

List of Studies


Keywords
Drug Studies, Liver, Rare Diseases
Summary

This is an experimental drug trial to study a new drug, elafibranor, in patients with Primary Biliary Cholangitis (PBC). The main objective of this study is to evaluate the effect of daily oral 80mg elafibranor for 52 weeks on the treatment of cholestasis (impairment of bile formation and/or bile buildup) in patients with PBC. Elafibranor is a medicine being developed by Genfit Pharmaceuticals that is designed to work differently than any other available medications for PBC. In addition to decreasing bile acid formation, increasing bile acid uptake, and detoxifying bile acids like medications that work similarly to elafibranor, elafibranor additionally has anti-inflammatory effects that Genfit believes will provide additional health benefits to patients with PBC.

Persons interested in participation will need to complete at least 1, but no more than 3, Screening Visits at the Main Campus of the Medical University of South Carolina (MUSC) in Charleston, SC in order to determine eligibility. If enrolled, you will have up to 21 study visits with the study doctor at MUSC over a period of at least 52 weeks (1 year) and up to a maximum of 312 weeks (6 years). At study visits you will be asked to complete up to 7 study-related questionnaires and complete any study-related testing such as physical exams, medical imaging, lab work (such as blood or urine tests), etc.

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

VK2809 A PHASE 2B, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO ASSESS THE EFFICACY, SAFETY, AND TOLERABILITY OF VK2809 ADMINISTERED FOR 52 WEEKS FOLLOWED BY A 4-WEEK OFF-DRUG PHASE IN SUBJECTS WITH BIOPSY PROVEN NON-ALCOHOLIC STEATOHEPATITIS WITH FIBROSIS

Date Added
July 6th, 2021
PRO Number
Pro00109573
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

The purpose of this study is to find out about the safety and effectiveness of a study medicine (VK2809) in study subjects with non-alcoholic steatohepatitis (NASH) with fibrosis. The study will take approximately 64 weeks to complete, with study (clinic) visits approximately every 4 weeks.

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

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



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