1650 Participants Needed

Efruxifermin for NASH

Recruiting at 233 trial locations
AS
Overseen ByAkero Study Director
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: Akero Therapeutics, Inc
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called efruxifermin to determine its effectiveness for people with non-alcoholic steatohepatitis (NASH), a liver condition causing inflammation and damage from fat buildup. The study compares two doses of efruxifermin against a placebo (a substance with no active drug) to assess its effectiveness in treating NASH without cirrhosis (severe liver scarring). Suitable participants have been diagnosed with NASH through a recent liver biopsy and face daily challenges from metabolic conditions like obesity, high blood pressure, or type 2 diabetes. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for NASH.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that efruxifermin is likely to be safe for humans?

Research has shown that efruxifermin is generally safe and well-tolerated. In earlier studies, patients taking efruxifermin showed improvements in liver health. These studies did not identify any major safety concerns. Most side effects were mild and manageable. Some participants experienced minor issues like nausea or headache, but these were uncommon. Overall, efruxifermin appears to be a promising option for treating non-cirrhotic NASH, with a good safety record so far.12345

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

Efruxifermin is unique because it targets the root cause of NASH by activating the FGF21 pathway, unlike most current treatments that focus on managing symptoms like liver inflammation and fibrosis. This mechanism is promising because it could potentially reverse liver damage by improving metabolic processes and reducing fibrosis. Researchers are excited because Efruxifermin's approach offers a potential disease-modifying effect, which could significantly improve outcomes for patients with NASH, a condition with limited effective therapies.

What evidence suggests that efruxifermin might be an effective treatment for NASH?

Research shows that efruxifermin (EFX) can significantly reduce liver fat in people with NASH, a serious liver disease. Studies also indicate that it improves liver scarring in patients with moderate stages of the disease. Researchers have found EFX to be effective and generally safe for patients. Evidence suggests that EFX could be a promising treatment option for people with NASH, especially those with fibrosis stages 2 and 3. These findings make it a strong candidate for effectively treating this condition.25678

Are You a Good Fit for This Trial?

This trial is for people with a liver condition called non-cirrhotic NASH or MASH, which involves fat buildup and fibrosis (scarring) at stage 2 or 3. Participants should not have cirrhosis, a more advanced liver scarring.

Inclusion Criteria

FibroScan® measurement > 7.5 kPa
I have NASH confirmed by a recent biopsy showing specific liver damage.
ELF score ≥ 7.7
See 1 more

Exclusion Criteria

I have a liver condition not caused by other known diseases.
I have Type 1 diabetes or my Type 2 diabetes is not under control.
My liver biopsy shows I have cirrhosis.

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

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

188 weeks

Long-term follow-up

Participants are monitored for event-free survival and other long-term outcomes

240 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Efruxifermin
Trial Overview The study tests Efruxifermin (EFX), comparing it to a placebo in a controlled setting. Participants are randomly assigned to either the drug group or the placebo group without knowing which one they receive.
How Is the Trial Designed?
3Treatment groups
Experimental Treatment
Placebo Group
Group I: EFX 50 mgExperimental Treatment1 Intervention
Group II: EFX 28 mgExperimental Treatment1 Intervention
Group III: PlaceboPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Akero Therapeutics, Inc

Lead Sponsor

Trials
6
Recruited
3,900+

Published Research Related to This Trial

In a phase 2b trial involving 128 patients with non-alcoholic steatohepatitis (NASH) and moderate to severe fibrosis, efruxifermin significantly improved liver fibrosis and resolved NASH in 39% of patients receiving 28 mg and 41% of those receiving 50 mg, compared to 20% in the placebo group.
Efruxifermin was generally well-tolerated, with most adverse events being mild to moderate, such as diarrhea and nausea, indicating its potential for further evaluation in larger phase 3 trials.
Safety and efficacy of once-weekly efruxifermin versus placebo in non-alcoholic steatohepatitis (HARMONY): a multicentre, randomised, double-blind, placebo-controlled, phase 2b trial.Harrison, SA., Frias, JP., Neff, G., et al.[2023]
Efgartigimod, an FcRn inhibitor, effectively reduced total IgG and autoreactive IgG levels in patients with pemphigus vulgaris and pemphigus foliaceus, showing early signs of improved disease activity and good tolerability during a phase 2 clinical trial.
Interestingly, while total IgG levels returned to baseline after treatment, autoreactive antibody levels remained low in some participants, suggesting a potential long-lasting immunomodulatory effect of efgartigimod beyond just blocking IgG recycling.
FcRn Antagonism Leads to a Decrease of Desmoglein-Specific B Cells: Secondary Analysis of a Phase 2 Study of Efgartigimod in Pemphigus Vulgaris and Pemphigus Foliaceus.Maho-Vaillant, M., Sips, M., Golinski, ML., et al.[2023]
Efruxifermin (EFX) is a promising treatment for liver fibrosis due to nonalcoholic steatohepatitis (NASH), showing a dose-proportional pharmacokinetic profile in a phase 2a study involving patients with moderate-to-advanced fibrosis or compensated cirrhosis.
A validated electrochemiluminescent immunoassay (ECLIA) was developed to accurately measure biologically active EFX in human serum, ensuring reliable pharmacokinetic assessments and demonstrating the importance of the intact C-terminus for EFX's pharmacological activity.
Noncompetitive immunoassay optimized for pharmacokinetic assessments of biologically active efruxifermin.Kinne, AS., Tillman, EJ., Abdeen, SJ., et al.[2023]

Citations

1.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/34239138/
Efruxifermin in non-alcoholic steatohepatitis: a randomized, ...Treatment with efruxifermin significantly reduced HFF in patients with F1-F3 stage NASH, with an acceptable safety profile.
NCT06161571 | A Study Evaluating Efruxifermin in ...The aim of this study is to assess the safety and tolerability of EFX compared to placebo in subjects with non-invasively diagnosed NASH/MASH and NAFLD/MASLD.
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 ...
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 ...
A systematic review and meta-analysis of efruxifermin's ...Efruxifermin may be a potential therapeutic for MASH-related fibrosis, with the available data indicating seemingly favorable tolerability.
6.pubmed.ncbi.nlm.nih.govpubmed.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 ...
7.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40818852/
Safety and efficacy of once-weekly efruxifermin versus ...Safety and efficacy of once-weekly efruxifermin versus placebo in metabolic dysfunction-associated steatohepatitis (HARMONY): 96-week results ...
NCT03976401 | A Study of Efruxifermin in Subjects With ...This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study administered for 16 weeks in subjects with ...
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