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Recruiting NCT07701993

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)

Phase III Interventional Metabolic Dysfunction-associated Steatohepatitis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Efimosfermin alfa, Placebo.
Who it may be relevant to
Registry conditions: Metabolic Dysfunction-associated Steatohepatitis. Basic parameters: 18 years — 75 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 3, Double-blind, Randomized, Placebo Controlled, 2-arm Study to Investigate the Safety and Efficacy of Efimosfermin Alfa Injection in Adult Participants With Compensated Cirrhosis Due to Metabolic Dysfunction-associated Steatohepatitis (NEBULA-1)

Overview

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

Interventions

  • Drug Efimosfermin alfa
    Efimosfermin alfa (subcutaneous injection) will be administered.
  • Drug Placebo
    Placebo (subcutaneous injection) will be administered.

Primary outcome measures

  • Time from randomization to an adjudicated composite liver-related clinical outcome [Time frame: From Randomization (Day 1) to Week 356 (end of treatment)]
Secondary outcome measures (12)
  • Proportion of participants achieving change from Baseline in vibration-controlled transient elastography- liver stiffness measurement (VCTE-LSM) and in enhanced liver fibrosis (ELF) score [Time frame: Baseline (Day 1), Week 96, and Week 260]
  • Proportion of participants achieving change from Baseline in VCTE-LSM [Time frame: Baseline (Day 1), Week 96, and Week 260]
  • Proportion of participants with treatment-emergent adverse events (TEAEs) and TEAEs by severity [Time frame: 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 [Time frame: Week 96, Week 260 and Week 356 (end of treatment)]
  • Proportion of participants with Grade 3 and Grade 4 laboratory abnormalities [Time frame: Week 96, Week 260 and Week 356 (end of treatment)]
  • Absolute change from Baseline in VCTE-LSM [Time frame: Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment)]
  • Relative change from Baseline in VCTE-LSM [Time frame: Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment)]
  • Absolute change from Baseline in Magnetic resonance elastography (MRE) scores [Time frame: Baseline (Day 1), Week 96, and Week 260]
  • Relative change from Baseline in MRE scores [Time frame: Baseline (Day 1), Week 96, and Week 260]
  • Absolute change from Baseline in ELF scores [Time frame: Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment)]
  • Relative change from Baseline in ELF scores [Time frame: Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment)]
  • Proportion of participants experiencing improvement in ELF score [Time frame: Week 96, Week 260 and Week 356 (end of treatment)]

Eligibility criteria

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 1 center
  • GSK Investigational Site — Miami

Identifiers

NCT: NCT07701993 · 306300 · 2026-525755-82

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗