Prospective non-interventional, phase IV, multicenter, study to assess the effectiveness, safety, and tolerability of elafibranor 80mg/day in patients with PBC receiving treatment in real-world settings

Date Added
June 3rd, 2025
PRO Number
Pro00136802
Researcher
Don Rockey

List of Studies


Keywords
Liver, Non-interventional, Rare Diseases
Summary

This is an international, multicenter, study that will not prescribe elafibranor. It is designed primarily to collect data and assess real-world effectiveness of treatment with elafibranor 80mg/day on adult patients with PBC, and to describe the safety of this treatment and its impact on their quality of life, over a period of 24 months.

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

A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY EVALUATING THE SAFETY AND EFFICACY OF EFRUXIFERMIN IN SUBJECTS WITH NON-CIRRHOTIC NONALCOHOLIC STEATOHEPATITIS (NASH)/METABOLIC DYSFUNCTION-ASSOCIATED STEATOHEPATITIS (MASH) AND FIBROSIS

Date Added
May 9th, 2025
PRO Number
Pro00141743
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

Primary objective is to evaluate the effect of EFX compared to placebo on achieving
NASH/MASH resolution AND fibrosis regression at Week 52 (in
Cohort 1 only) and to evaluate the effect of EFX compared to placebo on all-cause
mortality and liver-related clinical outcomes as measured by the
time to first occurrence of any of the predefined, adjudicated events
in subjects with NASH/MASH and fibrosis.

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

A Phase 3. Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Efruxifermin in Subjects with Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH)

Date Added
March 27th, 2025
PRO Number
Pro00138507
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with NASH/MASH cirrhosis and fibrosis stage 4.

Institution
MUSC
Recruitment Contact
Joshua Inman
843-876-8439
inmanj@musc.edu

A phase 3, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of denifanstat in patients with metabolic dysfunction-associated steatotic liver disease (MASLD)/metabolic dysfunction-associated steatohepatitis (MASH)

Date Added
December 19th, 2024
PRO Number
Pro00138889
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

To evaluate the safety and tolerability of
denifanstat 50 mg compared to placebo in patients
with MASLD/MASH after 52 weeks of treatment.

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

A phase 3, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of denifanstat in patients with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) and F2/F3 fibrosis (FASCINATE-3)

Date Added
November 25th, 2024
PRO Number
Pro00138888
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

To evaluate the effect of denifanstat 50 mg compared to placebo in reducing the risk of liver-related composite clinical outcome.
To evaluate the effect of denifanstat 50 mg compared to placebo after 52 weeks of treatment on achieving:
- MASH resolution without worsening of fibrosis,
OR
- Fibrosis regression without worsening of steatohepatitis.

Institution
MUSC
Recruitment Contact
Joshua Inman
843-876-8439
inmanj@musc.edu

A Single-blind, Phase 2, Multi-center, Randomized Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of the Relaxin Agonist R2R01 Plus Terlipressin Versus Terlipressin Alone in Patients with Hepatorenal Syndrome – Acute Kidney Injury

Date Added
November 21st, 2024
PRO Number
Pro00139091
Researcher
Don Rockey

List of Studies


Keywords
Kidney
Summary

This is a randomized, single-blind controlled, two group, multicenter trial
preceded by a safety run-in, in patients with Hepatorenal Syndrome (HRS) –
Acute Kidney Injury (HRS-AKI) to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of the Relaxin Agonist R2R01 Plus Terlipressin Versus Terlipressin Alone in Patients.

Institution
MUSC
Recruitment Contact
Jad Allam Saab
843-876-8439
allamj@musc.edu

A Phase 3 Study to Evaluate the Efficacy and Safety of Pegozafermin in Subjects with Compensated Cirrhosis due to Metabolic Dysfunction-Associated Steatohepatitis (MASH)

Date Added
July 23rd, 2024
PRO Number
Pro00136269
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

Evaluate the effect of pegozafermin compared to placebo in reducing the risk of clinical outcomes measured as a composite endpoint

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

A Phase 3 Study to Evaluate the Efficacy and Safety of Pegozafermin in Subjects with Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis

Date Added
June 24th, 2024
PRO Number
Pro00134737
Researcher
Don Rockey

List of Studies


Keywords
Drug Studies, Hepatology, Liver
Summary

The purpose of this study is to evaluate the effect of pegozafermin compared to placebo to see how well pegozafermin might improve liver fibrosis after 52 weeks.

Institution
MUSC
Recruitment Contact
Bridgette Blankenship
8438768439
blanke@musc.edu

A Phase 2a, open-label, randomized, controlled, multicenter, proof of concept study, to assess the efficacy, safety and tolerability of VS-01 on top of standard of care, compared to standard of care alone, in adult patients with acute-on-chronic liver failure (ACLF) grades 1 and 2 and ascites

Date Added
April 2nd, 2024
PRO Number
Pro00131012
Researcher
Don Rockey

List of Studies


Keywords
Liver
Summary

This is a multi-center, randomized, controlled, open-label, Phase 2a
proof of concept study of VS-01 in adult patients with ACLF grades 1
and 2 and ascites. Approximately 60 patients will be enrolled. Sample
size was calculated to meet the study objectives assuming a 10% dropout rate.

Institution
MUSC
Recruitment Contact
Jad Allam Saab
843-876-7233
allamja@musc.edu

A Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants with Primary Biliary Cholangitis (PBC)

Date Added
February 26th, 2024
PRO Number
Pro00130418
Researcher
Don Rockey

List of Studies


Keywords
Digestive System, Drug Studies, Hepatology, Liver, Rare Diseases
Summary

The main goal of this study is to evaluate how well taking oral elafibranor 80 mg daily works, compared to a placebo, in reducing or preventing the occurrence of death, liver transplant, worsening of liver disease, and liver disease-related complications in adults with PBC.

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



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