Efruxifermin for NASH with Cirrhosis

Not currently recruiting at 56 trial locations

Overseen By Akero Study Director

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Akero Therapeutics, Inc

Must be taking: GLP-1R agonist

Disqualifiers: Type 1 diabetes, Uncontrolled Type 2 diabetes, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 96 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether a drug called efruxifermin can treat a liver condition known as NASH with cirrhosis. NASH, or non-alcoholic steatohepatitis, is a serious liver disease often linked to obesity and diabetes. Participants will receive either efruxifermin or a placebo (a look-alike with no active drug) to evaluate the treatment's effectiveness. This trial may suit those diagnosed with cirrhosis due to NASH or those with type 2 diabetes using specific diabetes medications. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are in Cohort D, you must have been using a GLP-1R agonist (a type of diabetes medication) for at least 90 days before joining the study.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that efruxifermin is generally safe and well-tolerated. One study found that participants experienced improvements in liver health indicators, suggesting the treatment might benefit the liver without causing significant harm.

Participants reported no major side effects, indicating the treatment is manageable. While more remains to be learned, past research offers a promising view of efruxifermin's safety for those considering joining clinical trials. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for NASH?

Unlike the standard treatments for NASH with cirrhosis, such as lifestyle changes and medications like vitamin E and pioglitazone, Efruxifermin is unique because it targets the underlying causes of liver damage through a novel mechanism. Efruxifermin is a fusion protein designed to activate the fibroblast growth factor 21 (FGF21) pathway, which plays a crucial role in regulating metabolism and reducing liver fibrosis. Researchers are excited about Efruxifermin because it has the potential to not only halt disease progression but also reverse liver damage, offering hope for improved liver health and function in patients with NASH and cirrhosis.

What evidence suggests that this trial's treatments could be effective for NASH with cirrhosis?

Research has shown that efruxifermin (EFX), one of the treatments studied in this trial, may help treat non-alcoholic steatohepatitis (NASH) with liver scarring. In earlier studies, patients taking efruxifermin experienced improvements in liver health and scarring. These improvements suggest the drug might aid in liver healing and better function. Although efruxifermin did not meet some early goals in reducing scarring, its potential benefits for liver health remain promising. Overall, efruxifermin could offer hope for those with liver issues related to NASH. Participants in this trial may receive either efruxifermin or a placebo. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for adults aged 18-75 with compensated cirrhosis due to NASH, which is a type of liver disease. Participants may also have Type 2 diabetes or other metabolic syndrome components like obesity. Pregnant or breastfeeding women and those with recent significant weight loss or uncontrolled diabetes are excluded.

Inclusion Criteria

I have cirrhosis from NASH confirmed by a biopsy.

My liver fibrosis is at stage 1, 2, or 3.

I have been diagnosed with type 2 diabetes.

Exclusion Criteria

I have cirrhosis confirmed by a liver biopsy.

I have Type 1 diabetes or my Type 2 diabetes is not under control.

I have lost more than 10% of my weight in the last 3 months or since my liver biopsy.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive efruxifermin (EFX) or placebo in a randomized, double-blind, placebo-controlled study

96 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Efruxifermin
• Placebo

Trial Overview The study tests Efruxifermin (EFX), comparing it against a placebo in people with advanced fatty liver disease. It's conducted at multiple centers where participants are randomly assigned to receive either the drug or placebo without knowing which one they're getting.

How Is the Trial Designed?

Group I: EFX 50 mg (Main Study) Experimental Treatment 1 Intervention

Group II: EFX 50 mg (Cohort D) Experimental Treatment 1 Intervention

Group III: EFX 28 mg (Main Study) Experimental Treatment 1 Intervention

Group IV: Placebo (Cohort D) Placebo Group 1 Intervention

Group V: Placebo (Main Study) Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Akero Therapeutics, Inc

Lead Sponsor

Trials

6

Recruited

3,900+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36644237/

A randomized, double-blind, placebo-controlled phase IIa ... Conclusions: Efruxifermin appeared safe and well-tolerated with encouraging improvements in markers of liver injury, fibrosis, and glucose and ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06528314

NCT06528314 | A Study Evaluating Efruxifermin in ... This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with compensated cirrhosis due ...

3. jcto.weill.cornell.edu jcto.weill.cornell.edu/open_clinical_trials/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-nashmetabolic-dysfunction-associated

A Phase 3, Randomized, Double-Blind, Placebo ... The purpose of this research study is to evaluate the effect of the investigational study drug Efruxifermin (EFX) in treating compensated liver cirrhosis ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9832280/

A randomized, double-blind, placebo-controlled phase IIa trial ... Efruxifermin has shown clinical efficacy in patients with non-alcoholic steatohepatitis (NASH) and F1–F3 fibrosis. The primary objective of the BALANCED Cohort ...

5. appliedclinicaltrialsonline.com appliedclinicaltrialsonline.com/view/efruxifermin-mash-cirrhosis

Efruxifermin Shows Long-Term Promise in MASH-Related ... Missed Primary Endpoint at 36 Weeks. Efruxifermin did not significantly reduce fibrosis without worsening of MASH at week 36 compared to ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06528314

NCT06528314 | A Study Evaluating Efruxifermin in ... This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with compensated cirrhosis due to ...

7. natap.org natap.org/2025/EASL/NEJMoa2502242.pdf

Efruxifermin in Compensated Liver Cirrhosis Caused by ... Data are needed on the efficacy and safety of efruxifermin in patients with compensated cirrhosis (stage 4 fibrosis) caused by MASH. METHODS. In ...