A Study to Investigate the Safety and Efficacy of Efimosfermin Alfa Compared With Placebo in Adult Participants With Alcohol-related Liver Disease (ALD)

NCT07791043 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 3068372026-526391-23
• Study Type: interventional
• Target: 274
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a dose-ranging study to evaluate safety of efimosfermin alfa and to establish proof-of-concept that efimosfermin alfa therapy provides benefit in participants with ALD.

Conditions

Liver Diseases, Alcoholic

Keywords

Efimosfermin alfa; Alcohol-related liver disease; Liver stiffness measurement; Advanced chronic liver disease

Outcome Measures

Primary Outcomes (2)

• Change from Baseline in vibration-controlled transient elastography-liver stiffness measurement (VCTE-LSM)

  VCTE-LSM is a non-invasive test that uses vibration-controlled transient elastography to measure the stiffness of the liver in kilopascals (kPa)

  Baseline (Day 1) and up to Week 60

• Change from Baseline in model for end-stage liver disease (MELD) score

  MELD is a scoring system for assessing the severity of chronic liver disease. MELD scores range between 6 and 40, with 40 being the most severe.

  Baseline (Day 1) and up to Week 60

Secondary Outcomes (11)

• Change from Baseline in serum aspartate aminotransferase (AST) (International units per liter)

  Baseline (Day 1) and up to Week 60

• Change from Baseline in a blood test that helps predict the risk of disease progression in liver disease

  Baseline (Day 1) and up to Week 52

• Trough Concentration at steady state (Ctrough,ss) following administration of efimosfermin alfa

  Up to Week 56

• Area Under the concentration-time Curve from Time 0 (pre-dose) to the last quantifiable concentration (AUC[0-t]) of efimosfermin alfa in the subset of participants with optional intensive pharmacokinetics (PK) sampling

  Up to Week 4

• Maximum observed drug concentration (Cmax) of efimosfermin alfa in the subset of participants with optional intensive PK sampling

  Up to Week 4

Study Arms (4)

Efimosfermin alfa Dose Level 1

EXPERIMENTAL

Participants will receive efimosfermin alfa dose level 1.

Drug: Efimosfermin alfa

Efimosfermin alfa Dose Level 2

EXPERIMENTAL

Participants will receive efimosfermin alfa dose level 2.

Drug: Efimosfermin alfa

Efimosfermin alfa Dose Level 3

EXPERIMENTAL

Participants will receive efimosfermin alfa dose level 3.

Drug: Efimosfermin alfa

Placebo

PLACEBO COMPARATOR

Participants will receive placebo.

Drug: Placebo

Interventions

Efimosfermin alfa DRUG

Efimosfermin alfa will be administered.

Efimosfermin alfa Dose Level 1; Efimosfermin alfa Dose Level 2; Efimosfermin alfa Dose Level 3

Placebo DRUG

Placebo will be administered.

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant must be 18 to 75 years of age inclusive, at the time of Screening.
• Capable of giving signed informed consent prior to the performance of any study-specific procedures.
• Able and willing to comply with all study assessments and adhere to the protocol schedule of activities.
• History of heavy alcohol consumption for greater than 6 months at any time prior to Screening.
• A female participant is eligible to participate if they are not pregnant or breastfeeding, and one of the following conditions applies:
• Is a Participant of non-childbearing potential (PONCBP) OR
• Is a Participant of childbearing potential (POCBP) and using a contraceptive method that is highly effective, with a failure rate of less than (\<)1 percentage (%), 30 days prior to and during the study intervention period and for at least 16 weeks after the last dose of study intervention.

You may not qualify if:

• Other primary causes of liver disease (e.g., viral hepatitis, primary biliary cholangitis, primary sclerosing cholangitis, autoimmune hepatitis, drug-induced hepatotoxicity, Wilson disease, hemochromatosis, alpha-1-antitryspin deficiency, etc.). Alcohol must be the primary cause of liver disease.
• Co-infection with Hepatitis B virus (HBV), Hepatitis C virus (HCV), or Human immunodeficiency virus (HIV) as indicated from the central lab
• History of diabetes mellitus (DM), either:
• Type 1 DM
• Type 2 DM with unstable glycemic control or with major complications.
• History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer), treated or untreated, within the 3 years prior to Screening 1, regardless of any evidence of local recurrence or metastasis.
• Current, or history of known hepatocellular carcinoma (HCC).
• Any major surgery within 3 months prior to Screening 1 or planned during the study
• Any surgical or medical condition that might jeopardize the individual's safety or compliance with study procedures in the opinion of the investigator.
• All organ transplant recipients, except for history of corneal transplants, or current listing or active consideration for liver transplant during the Screening period.
• Poorly controlled hypertension.
• Evidence of Wernicke-Korsakoff syndrome or alcohol-related dementia.
• Prior use of a Fibroblast growth factor 21 (FGF21) analogue, including efimosfermin, within the 6 months prior to Screening 1.
• Individuals with a history of resmetirom use within 3 months prior to Day 1 are to be excluded.
• Concomitant use of investigational drugs for Metabolic dysfunction-associated steatohepatitis (MASH) or ALD.

Sponsors & Collaborators

• GlaxoSmithKline: lead

MeSH Terms

Conditions

Liver Diseases, Alcoholic

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Alcohol-Induced Disorders; Alcohol-Related Disorders; Substance-Related Disorders; Chemically-Induced Disorders

Study Officials

• GSK Clinical Trials

  GlaxoSmithKline

  STUDY DIRECTOR

Central Study Contacts

US GSK Clinical Trials Call Center

CONTACT

877-379-3718; GSKClinicalSupportHD@gsk.com

EU GSK Clinical Trials Call Center

CONTACT

+44 (0) 20 89904466; GSKClinicalSupportHD@gsk.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 21, 2026
• First Posted: August 27, 2026
• Study Start (Estimated): September 23, 2026
• Primary Completion (Estimated): September 5, 2028
• Study Completion (Estimated): February 6, 2030
• Last Updated: August 27, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Anonymized IPD will be made available within 6 months of publication of primary, key secondary and safety results for studies in product with approved indication(s) or asset(s) with development terminated across all indications.
• Access Criteria: Anonymized IPD is shared with researchers whose proposals are approved by an Independent Review Panel and after a Data Sharing Agreement is in place. Access is provided for an initial period of 12 months, but an extension may be granted, when justified, for up to 6 months.