NCT07701993RecruitingPhase 3
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)
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- GlaxoSmithKline
- Enrollment
- 1,740
- Locations
- 9
- Primary Endpoint
- Time from randomization to an adjudicated composite liver-related clinical outcome
Study Overview
Brief Summary
This study will investigate the safety and efficacy of efimosfermin alfa in participants with compensated cirrhosis due to MASH.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
This is a double-blind study.
Eligibility Criteria
- Ages
- 18 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
Participants receiving placebo
Placebo Comparator
Intervention: Placebo (Drug)
Participants receiving efimosfermin alfa
Experimental
Intervention: Efimosfermin alfa (Drug)
Outcomes
Primary Outcomes
Time from randomization to an adjudicated composite liver-related clinical outcome
Time Frame: From Randomization (Day 1) to Week 356 (end of treatment)
Liver-related outcome comprises all-cause mortality; liver transplantation; occurrence of significant hepatic decompensation events.
Secondary Outcomes
- 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))
- Absolute change from Baseline in VCTE-LSM(Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment))
- Relative change from Baseline in VCTE-LSM(Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment))
- Absolute change from Baseline in Magnetic resonance elastography (MRE) scores(Baseline (Day 1), Week 96, and Week 260)
- Relative change from Baseline in MRE scores(Baseline (Day 1), Week 96, and Week 260)
- Absolute change from Baseline in ELF scores(Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment))
- Relative change from Baseline in ELF scores(Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment))
- Proportion of participants experiencing improvement in ELF score(Week 96, Week 260 and Week 356 (end of treatment))
- Change from Baseline in glycated hemoglobin (HbA1c) (Percentage of HbA1c) in participants with Type 2 Diabetes Mellitus (T2DM)(Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment))
- Change from Baseline in fasting glucose (Millimole per Liter) in participants with T2DM(Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment))
- Change from Baseline in fasting total cholesterol, low-density lipoprotein (LDL)-cholesterol, high-density lipoprotein (HDL)- cholesterol, and fasting triglycerides (Millimoles per liter)(Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment))
- Change from Baseline in Patient-reported outcomes measurement information system (PROMIS)-Fatigue score(Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment))
- Change from Baseline in Chronic Liver Disease Questionnaire-Nonalcoholic Steatohepatitis (CLDQ-NASH) domain and total score(Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment))
- Change from Baseline in Short Form-36 (SF-36) component and domain scores(Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment))
Investigators
Study Sites (9)
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