Ireland
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Recruiting Phase 3

A Pivotal Clinical Study to Investigate Efimosfermin Alfa in Participants With Biopsy-confirmed F2- or F3-stage MASH

NCT07221227 · tracked via the Priya Life Science USA tracker
Phase
Phase 3
Started
2025-10-24
Last updated
2026-07-27

Condition(s) studied

Non-alcoholic Fatty Liver Disease

Investigational drug(s) / intervention(s)

Efimosfermin alfa →Efimosfermin alfa →Placebo

Efimosfermin alfa: Efimosfermin alfa will be administered

Efimosfermin alfa: Efimosfermin alfa will be administered

Placebo: Placebo will be administered

Study summary

The purpose of this study is to assess the safety and efficacy of efimosfermin alfa in the resolution of steatohepatitis and improvement of liver-related clinical outcome compared to placebo in individuals with MASH and biopsy-confirmed F2- or F3-stage fibrosis.

Eligibility

Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
Inclusion Criteria: 1. Able and willing to understand and sign a written informed consent form that must be obtained prior to the initiation of study procedures 2. Age \>=18 and \<=75 years at enrollment 3. History or presence of 2 or more of the 5 components of metabolic syndrome per American Heart Association definition 4. Liver biopsy confirmation of MASH consistent with stage F2 or F3 fibrosis and a NAS score \>=4 confirmed by a central pathologist Exclusion Criteria: 1. Contraindication or ineligibility for percutaneous liver biopsy 2. ALT or AST \>=5 x upper limit of normal (ULN) 3. Total bilirubin \>=1.3 milligram per deciliter (mg/dL). Individuals with documented Gilbert's syndrome may be enrolled if they experienced an isolated increase in total bilirubin of \>=1.3 mg/dL and direct bilirubin is \<=20% of total bilirubin; otherwise, the individual will be excluded. 4. Serum albumin \<=3.5 grams per deciliter (g/dL) 5. International normalized ratio (INR) \>=1.3 not due to therapeutic anticoagulation. Individuals receiving chronic anticoagulant treatment with higher INR values may be enrolled at the discretion of the Investigator and Study Medical Monitor. 6. Alkaline phosphatase (ALP) \>=2\*ULN 7. Platelet (PLT) count \<140,000 per (/) cubic millimeter (mm\^3); individuals with a PLT count between 110,000/mm\^3 and 140,000/mm\^3 may be enrolled after discussion with the Study Medical Monitor. 8. Serum creatinine \>=1.5 mg/dL or creatinine clearance \<=60 milliliter (mL)/minute (min)/1.73 square meter by Chronic Kidney Disease Epidemiology Collaboration equation 9. Alpha-fetoprotein \>=20 nanogram per milliliter (ng/mL) 10. Glycated hemoglobin \>=9.0% 11. Model for End-Stage Liver Disease score \>=12 unless the score is elevated in the absence of liver dysfunction (e.g., Gilbert's syndrome) 12. Phosphatidyl ethanol (PEth) \>=80 ng/mL at Screening 13. Evidence of infection with any of the following: 1. Human immunodeficiency virus; 2. Hepatitis B virus (detectable HBsAg at Screening); 3. Hepatitis C virus (HCV); 14. Chronic liver disease from any other cause including, but not limited to, alcoholic liver disease; evidence of portal hypertension; viral hepatitis or any history or evidence of cirrhosis on screening liver biopsy; or decompensated liver disease such as clinical ascites, bleeding gastroesophageal varices, hepatorenal syndrome, or hepatic encephalopathy prior to Screening or Day 1. 15. Current or history of excessive alcohol intake for \>=3 months within the 12-month period prior to Screening

Primary outcome measure(s)

  • Proportion of participants experiencing improvement in fibrosis by >=1 stage and no worsening of steatohepatitis at Week 52 — At Week 52
    Proportion of participants experiencing improvement in fibrosis of greater than or equal to (\>=) 1 stage by MASH clinical research network (CRN) fibrosis scores and no worsening of steatohepatitis (defined as no increase in nonalcoholic fatty liver disease activity score \[NAS\] for ballooning, inflammation, or steatosis) at 52 weeks will be assessed. MASH CRN fibrosis score ranges from 0 to 4, higher score indicates greater severity. NAS score ranges from 0 to 8, higher score indicates worse disease activity.
  • Proportion of participants experiencing resolution of steatohepatitis reading and no worsening of MASH CRN fibrosis score at Week 52 — At Week 52
    Resolution of steatohepatitis is defined as absence of fatty liver disease or isolated or simple steatosis without steatohepatitis and a NAS of 0 or 1 for inflammation, 0 for ballooning, and any value for steatosis. NAS score ranges from 0 to 8, higher score indicates worse disease activity. MASH CRN fibrosis score ranges from 0 to 4, higher score indicates greater severity.
  • Time from randomization to an adjudicated composite liver-related clinical outcome — From Randomization (Day 1) to 48 months
    Liver-related outcome will comprised of all-cause mortality; transplantation; occurrence of significant hepatic events.

Trial sites (91)

FacilityCityRegionStatus
GSK Investigational Site Chandler Arizona Recruiting
GSK Investigational Site Tucson Arizona Recruiting
GSK Investigational Site Little Rock Arkansas Recruiting
GSK Investigational Site Arcadia California Recruiting
GSK Investigational Site Covina California Recruiting
GSK Investigational Site Folsom California Recruiting
GSK Investigational Site Los Angeles California Recruiting
GSK Investigational Site Montclair California Recruiting
GSK Investigational Site Riverdale California Recruiting
GSK Investigational Site San Diego California Recruiting
GSK Investigational Site Santa Maria California Recruiting
GSK Investigational Site Van Nuys California Recruiting
GSK Investigational Site Colorado Springs Colorado Recruiting
GSK Investigational Site Boynton Beach Florida Recruiting
GSK Investigational Site Cape Coral Florida Recruiting
GSK Investigational Site Clermont Florida Recruiting
GSK Investigational Site Fort Myers Florida Recruiting
GSK Investigational Site Hialeah Florida Recruiting
GSK Investigational Site Hialeah Florida Recruiting
GSK Investigational Site Homestead Florida Recruiting
GSK Investigational Site Inverness Florida Recruiting
GSK Investigational Site Jacksonville Florida Recruiting
GSK Investigational Site Kissimmee Florida Recruiting
GSK Investigational Site Lakeland Florida Recruiting
GSK Investigational Site Maitland Florida Recruiting
GSK Investigational Site Miami Florida Recruiting
GSK Investigational Site Miami Florida Recruiting
GSK Investigational Site Miami Florida Recruiting
GSK Investigational Site Miami Florida Recruiting
GSK Investigational Site Miami Florida Recruiting
GSK Investigational Site Miami Florida Recruiting
GSK Investigational Site Miami Florida Recruiting
GSK Investigational Site Miami Lakes Florida Recruiting
GSK Investigational Site Ocala Florida Recruiting
GSK Investigational Site Palmetto Bay Florida Recruiting
GSK Investigational Site Sarasota Florida Recruiting
GSK Investigational Site Marietta Georgia Recruiting
GSK Investigational Site Topeka Kansas Recruiting
GSK Investigational Site Bastrop Louisiana Recruiting
GSK Investigational Site Marrero Louisiana Recruiting

+ 51 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07221227 on ClinicalTrials.gov ↗ ← All trials in the USA