A Study to Investigate Safety and Efficacy of Efimosfermin Compared With Placebo in Adult Participants With Compensated Cirrhosis Due to Metabolic Dysfunction-associated Steatohepatitis (MASH)
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 →
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Official title
A Phase 3, Two-part, Double-blind, Randomized, Placebo-controlled Study to Investigate the Safety and Efficacy of Efimosfermin Alfa Injection in Adult Participants With Compensated Cirrhosis (Stage F4 Fibrosis) Due to Metabolic Dysfunction-associated Steatohepatitis (NEBULA-2)
Overview
This is a multi-center, randomized, two-part (Part A and Part B) study investigating the safety and efficacy of efimosfermin alfa in adult participants with compensated cirrhosis due to MASH. Participants who complete the treatment during Part A of the study and meet the inclusion criteria will have the option to enroll in Part B (open label) of the study.
Interventions
- Drug Efimosfermin alfa
Efimosfermin alfa (subcutaneous injection) will be administered. - Drug Placebo
Placebo (subcutaneous injection) will be administered.
Primary outcome measures
- Part A: Proportion of participants achieving improvement in liver fibrosis by >=1 stage and no worsening of MASH [Time frame: At Week 96]
Secondary outcome measures (5)
- Part A: Proportion of participants achieving change from Baseline in vibration-controlled transient elastography (VCTE)- liver stiffness measurement (LSM) and in enhanced liver fibrosis (ELF) score [Time frame: Baseline (Day 1) and Week 96]
- Part A Change from Baseline in VCTE-LSM [Time frame: Baseline (Day 1) and Week 96]
- Part A: Proportion of participants with Treatment-Emergent Adverse Events (TEAEs) and TEAEs by severity [Time frame: Week 96]
- Part A: Proportion of participants with TEAEs leading to discontinuation and TEAEs leading to discontinuation by severity [Time frame: Week 96]
- Part A: Proportion of participants with Grade 3 and Grade 4 laboratory abnormalities [Time frame: Week 96]
Eligibility criteria
Inclusion criteria
- Participants aged between 18 and 75 years at enrolment.
- Participants with history or presence of at least two components of metabolic syndrome.
- Liver biopsy consistent with cirrhosis (fibrosis stage 4).
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 mellitus complications.
- History or evidence of chronic pancreatic disease; pancreatic injury or acute pancreatitis within 6 months before Screening.
- A recent history or planned surgical procedures or medications intended to produce significant weight loss.
- Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) >= 5 times upper limit normal (ULN).
- 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
- Triple blind
- Primary purpose
- Treatment
Study locations
United States · 2 centers
- GSK Investigational Site — Miami
- GSK Investigational Site — Houston
Identifiers
NCT: NCT07704892 · 308623 · 2026-525757-37