A Pivotal Clinical Study to Investigate the Safety and Efficacy of Efimosfermin Compared With Placebo in Adult Participants With Compensated Cirrhosis Due to Metabolic Dysfunction-associated Steatohepatitis (MASH)

NCT07701993 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: 3063002026-525755-82
• Study Type: interventional
• Target: 1,740
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study will investigate the safety and efficacy of efimosfermin alfa in participants with compensated cirrhosis due to MASH.

Conditions

Metabolic Dysfunction-associated Steatohepatitis

Keywords

Efimosfermin alfa; Metabolic dysfunction-associated; steatohepatitis; Fibrosis; Cirrhosis

Outcome Measures

Primary Outcomes (1)

• Time from randomization to an adjudicated composite liver-related clinical outcome

  Liver-related outcome comprises all-cause mortality; liver transplantation; occurrence of significant hepatic decompensation events.

  From Randomization (Day 1) to Week 356 (end of treatment)

Secondary Outcomes (18)

• Proportion of participants achieving change from Baseline in vibration-controlled transient elastography- liver stiffness measurement (VCTE-LSM) and in enhanced liver fibrosis (ELF) score

  Baseline (Day 1), Week 96, and Week 260

• Proportion of participants achieving change from Baseline in VCTE-LSM

  Baseline (Day 1), Week 96, and Week 260

• Proportion of participants with treatment-emergent adverse events (TEAEs) and TEAEs by severity

  Week 96, Week 260 and Week 356 (end of treatment)

• Proportion of participants with TEAEs leading to discontinuation and TEAEs leading to discontinuation by severity

  Week 96, Week 260 and Week 356 (end of treatment)

• Proportion of participants with Grade 3 and Grade 4 laboratory abnormalities

  Week 96, Week 260 and Week 356 (end of treatment)

Study Arms (2)

Participants receiving efimosfermin alfa

EXPERIMENTAL

Drug: Efimosfermin alfa

Participants receiving placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Efimosfermin alfa DRUG

Efimosfermin alfa (subcutaneous injection) will be administered.

Participants receiving efimosfermin alfa

Placebo DRUG

Placebo (subcutaneous injection) will be administered.

Participants receiving 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:

• Participants aged between 18 and 75 years at enrollment.
• Participants with compensated cirrhosis due to MASH, confirmed by non-invasive assessments.
• Participants with history or presence of at least two components of metabolic syndrome.

You may not qualify if:

• Participants with other chronic liver diseases.
• Participants with evidence or history of decompensated liver disease or hepatocellular carcinoma.
• Participants with history of Type 1 diabetes mellitus or major Type 2 diabetes complications.
• Participants with history or evidence of chronic pancreatic disease; pancreatic injury or acute pancreatitis within 6 months before screening.
• Participants with a recent history or planned surgical procedures or medications intended to produce significant weight loss.
• Participants with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>=5 times upper limit normal (ULN).
• Participants with current or history of excessive alcohol intake.

Sponsors & Collaborators

• GlaxoSmithKline: lead

MeSH Terms

Conditions

Fatty Liver; Fibrosis

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

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 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: This is a double-blind study.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2026
• First Posted: July 14, 2026
• Study Start: July 20, 2026
• Primary Completion (Estimated): July 25, 2033
• Study Completion (Estimated): August 22, 2033
• Last Updated: July 14, 2026

Record last verified: 2026-06

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 terminated asset(s) 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.