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

Investigating the Effects of an Investigational Medication on Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH) and Metabolic Dysfunction-Associated Steatohepatitis (MASH) with Fibrosis

R
Raj Vuppalanchi, MD

Primary Investigator

Enrolling By Invitation
18 years - 80 years
All
Phase 3
1 participants needed
1 Location

Brief description of study

This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with non-cirrhotic NASH/MASH and fibrosis stage 2 or 3 (F2 or F3).

Study goals:

To evaluate the effect of EFX compared to placebo on achieving NASH/MASH resolution AND fibrosis regression at Week 52 (in Cohort 1 only)

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

THIS STUDY IS ENROLLING BY INVITATION ONLY

Detailed description of study

This is a Phase 3, randomized, double-blind, placebo-controlled study
evaluating the safety and efficacy of EFX in subjects with noncirrhotic
NASH/MASH and fibrosis stage 2 or 3 (F2 or F3). The study will enroll
approximately 750 F2/F3 subjects with biopsy-proven definite
NASH/MASH into Cohort 1. An additional cohort (Cohort 2) of
approximately 900 subjects with biopsy-proven fibrosis stage 3 (F3),
will be enrolled. Primary histology efficacy evaluation at Week 52 will
occur in Cohort 1 only and all subjects will be evaluated for long-term
safety and clinical outcomes and endpoints. A biopsy will be collected
at Week 96 for Cohort 2 only.
Subjects meeting the study’s eligibility criteria will be randomly
assigned in a 1:1:1 ratio to 1 of 3 treatment groups as shown in the
figure below.

The primary histology endpoint analysis will occur after all subjects
from Cohort 1 have completed 52 weeks of treatment or permanently
discontinued from the study. For evaluation of long-term safety and
clinical outcomes, subjects will continue to receive their assigned
treatment for up to a total of 240 weeks.
 

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Metabolic Dysfunction-Associated Steatohepatitis, MASH With Fibrosis, Non-Cirrhotic Nonalcoholic Steatohepatitis,NASH With Fibrosis
  • Age: 18 years - 80 years
  • Gender: All

Inclusion Criteria:

  • Males and non-pregnant, non-lactating females between 18 - 80 years of age inclusive, based on the date of the screening visit.
  • Previous history or presence of 2 out of 4 components of metabolic syndrome (obesity, dyslipidemia, elevated blood pressure, elevated fasting glucose) or type 2 diabetes.
  • Cohort 1: Biopsy-proven NASH/MASH. Must have had a liver biopsy obtained ≤ 180 days prior to screening with fibrosis stage 2 or 3 and a non-alcoholic fatty liver disease (NAFLD) activity score (NAS) of ≥ 4 with at least a score of 1 in each of the following NAS components:
    • Steatosis (scored 0 to 3),
    • Ballooning degeneration (scored 0 to 2), and
    • Lobular inflammation (scored 0 to 3).

Exclusion Criteria:

  • Other causes of liver disease based on medical history and/or liver histology and/or central laboratory results.
  • Presence of cirrhosis on liver biopsy (fibrosis stage 4).
  • Type 1 or uncontrolled Type 2 diabetes.

Other inclusion and exclusion criteria may apply.

This study investigates the effects of an investigational medication on people with non-cirrhotic NASH (Nonalcoholic Steatohepatitis) or MASH (Metabolic Dysfunction-Associated Steatohepatitis) and fibrosis. NASH and MASH are conditions where fat builds up in the liver, causing inflammation and damage. The purpose of this study is to see if the investigational medication can resolve these conditions and reduce liver fibrosis, which is the thickening and scarring of liver tissue.

During the study, participants will be randomly assigned to receive either the investigational medication or a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine. Participants will have regular check-ups, including liver biopsies, to monitor the effects of the treatment. The study will assess the safety and effectiveness of the investigational medication over a period of time.

  • Who can participate: Adults aged 18 to 80 years with biopsy-proven NASH/MASH and fibrosis stage 2 or 3 can participate. Participants should have a history or presence of metabolic syndrome or type 2 diabetes. Individuals with other liver diseases or cirrhosis are excluded.
  • Study details: Participants will be assigned to one of three treatment arms and will receive either the investigational medication or a placebo. They will undergo regular monitoring and liver biopsies, a procedure to remove a small piece of liver tissue for examination, to assess the safety and effects of the treatment.
Updated on 03 Mar 2026. Study ID: GI-AKERO-SYNCHRONY-HISTOLOGY, 28973
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only