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)

Condition studied

Metabolic dysfunction-associated steatohepatitis

Investigational medicinal product(s)

Eligibility

Primary endpoint

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

Endpoint detail

Proportion of participants achieving change from Baseline in vibration-controlled transient elastography- liver stiffness measurement (VCTE-LSM) and in enhanced liver fibrosis (ELF) score, Proportion of participants achieving change from Baseline in VCTE-LSM, Proportion of participants with treatment-emergent adverse events (TEAEs) and TEAEs by severity, Proportion of participants with TEAEs leading to discontinuation and TEAEs leading to discontinuation by severity, Proportion of participants with Grade 3 and Grade 4 laboratory abnormalities, Absolute change from Baseline in VCTE-LSM, Relative change from Baseline in VCTE-LSM, Absolute change from Baseline in Magnetic resonance elastography (MRE) scores, Relative change from Baseline in MRE scores, Absolute change from Baseline in ELF scores, Relative change from Baseline in ELF scores, Proportion of participants experiencing improvement in ELF score, Change from Baseline in glycated hemoglobin (HbA1c) (Percentage of HbA1c) in participants with Type 2 Diabetes Mellitus (T2DM), Change from Baseline in fasting glucose (Millimole per Liter) in participants with T2DM, Change from Baseline in fasting total cholesterol, low-density lipoprotein (LDL)cholesterol, high-density lipoprotein (HDL)- cholesterol, and fasting triglycerides (Millimoles per liter), Change from Baseline in Patient-reported outcomes measurement information system (PROMIS)-Fatigue score, Change from Baseline in Chronic Liver Disease Questionnaire-Nonalcoholic Steatohepatitis (CLDQ-NASH) domain and total score, Change from Baseline in Short Form-36 (SF36) component and domain scores