> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/trial/a-pivotal-clinical-study-to-investigate-the-safety-and-615aa1a9-45ff-4b7d-8459-0d17f85cf323 > Record changed: 2026-10-13 # 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 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) Phase Phase 3 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) Recruitment status Recruiting Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) Participants 1,740 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) Lead sponsor GlaxoSmithKline Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) Start date 2026-07-22 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) Number of sites 1 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Title and summary **Summary** This study will investigate the safety and efficacy of efimosfermin alfa in participants with compensated cirrhosis due to MASH. Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Acronym** NEBULA-1 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Short title** 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) Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **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) Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Status and phase **Control arm** Placebo Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Registry reports results** No Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Listing at source** Listed Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Recruitment status** Recruiting Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Phase** Phase 3 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Study type** Interventional Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Dates **Completion date** 2033-08-24 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **First posted** 2026-07-14 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Registry last updated** 2026-08-05 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Primary completion date** 2033-07-27 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Start date** 2026-07-22 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Size and sites **Participants** 1,740 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Participant count basis** Estimated Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Planned participants** 1,740 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Protocol amendments** 0 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Number of sites** 1 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Sponsor and oversight **Data monitoring committee** Yes Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Registry sponsor category** Industry Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Lead sponsor** GlaxoSmithKline Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Source registry** ClinicalTrials.gov Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Identifiers **Registry ID** NCT07701993 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **ClinicalTrials.gov ID** NCT07701993 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) **Registry record** Open registry record Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Study design **Allocation** Randomised **Model** Parallel groups **Purpose** Treatment **Masking** Quadruple masking **Masking description** This is a double-blind study. **Masked roles** Participants, Care providers, Investigators, Outcome assessors Sources for study design Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Outcomes ### Primary outcomes 1. Time from randomization to an adjudicated composite liver-related clinical outcome Timeframe: 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 1. Proportion of participants achieving change from Baseline in vibration-controlled transient elastography- liver stiffness measurement (VCTE-LSM) and in enhanced liver fibrosis (ELF) score Timeframe: Baseline (Day 1), Week 96, and Week 260 VCTE-LSM is a non-invasive test that uses vibration-controlled transient elastography to measure the stiffness of the liver in kiloPascal (kPa). ELF score (scale of 6.7 to 11.3 with higher scores indicative of increased fibrosis) is a blood-based noninvasive test used as a prognostic marker for disease progression. 2. Proportion of participants achieving change from Baseline in VCTE-LSM Timeframe: Baseline (Day 1), Week 96, and Week 260 VCTE-LSM is a non-invasive test that uses vibration-controlled transient elastography to measure the stiffness of the liver in kPa. 3. Proportion of participants with treatment-emergent adverse events (TEAEs) and TEAEs by severity Timeframe: Week 96, Week 260 and Week 356 (end of treatment) 4. Proportion of participants with TEAEs leading to discontinuation and TEAEs leading to discontinuation by severity Timeframe: Week 96, Week 260 and Week 356 (end of treatment) 5. Proportion of participants with Grade 3 and Grade 4 laboratory abnormalities Timeframe: Week 96, Week 260 and Week 356 (end of treatment) 6. Absolute change from Baseline in VCTE-LSM Timeframe: Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment) 7. Relative change from Baseline in VCTE-LSM Timeframe: Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment) 8. Absolute change from Baseline in Magnetic resonance elastography (MRE) scores Timeframe: Baseline (Day 1), Week 96, and Week 260 MRE is a non-invasive imaging technique that combines magnetic resonance imaging (MRI) scanning with low-frequency mechanical vibrations to measure the stiffness of liver. MRE scores less than (\\<) 2.5 is normal, 2.5 - 3.0 Normal or inflammation, 3.0 - 3.5 Stage 1-2 fibrosis, 3.5 - 4.0 Stage 2-3 fibrosis, 4.0 - 5.0 Stage 3-4 fibrosis and greater than (\\>) 5.0 Stage 4 fibrosis. 9. Relative change from Baseline in MRE scores Timeframe: Baseline (Day 1), Week 96, and Week 260 MRE is a non-invasive imaging technique that combines magnetic resonance imaging (MRI) scanning with low-frequency mechanical vibrations to measure the stiffness of liver. MRE scores \\<2.5 is normal, 2.5 - 3.0 Normal or inflammation, 3.0 - 3.5 Stage 1-2 fibrosis, 3.5 - 4.0 Stage 2-3 fibrosis, 4.0 - 5.0 Stage 3-4 fibrosis and \\> 5.0 Stage 4 fibrosis 10. Absolute change from Baseline in ELF scores Timeframe: Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment) The ELF score will be calculated using a published algorithm combining the values of a set of extracellular matrix markers. The ELF score is used as a prognostic marker for disease progression: ELF score \\<9.8: Low risk of progression, ELF score 9.8 to \\<11.3: Moderate risk of progression and ELF score greater than or equal to (\\>=) 11.3: High risk of progression. 11. Relative change from Baseline in ELF scores Timeframe: Baseline (Day 1), Week 96, Week 260, and Week 356 (end of treatment) The ELF score will be calculated using a published algorithm combining the values of a set of extracellular matrix markers. The ELF score is used as a prognostic marker for disease progression: ELF score \\<9.8: Low risk of progression, ELF score 9.8 to \\<11.3: Moderate risk of progression and ELF score \\>=11.3: High risk of progression. 12. Proportion of participants experiencing improvement in ELF score Timeframe: Week 96, Week 260 and Week 356 (end of treatment) The ELF score will be calculated using a published algorithm combining the values of a set of extracellular matrix markers. The ELF score is used as a prognostic marker for disease progression: ELF score \\<9.8: Low risk of progression, ELF score 9.8 to \\<11.3: Moderate risk of progression and ELF score \\>=11.3: High risk of progression. 13. Change from Baseline in glycated hemoglobin (HbA1c) (Percentage of HbA1c) in participants with Type 2 Diabetes Mellitus (T2DM) Timeframe: Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment) 14. Change from Baseline in fasting glucose (Millimole per Liter) in participants with T2DM Timeframe: Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment) 15. Change from Baseline in fasting total cholesterol, low-density lipoprotein (LDL)-cholesterol, high-density lipoprotein (HDL)- cholesterol, and fasting triglycerides (Millimoles per liter) Timeframe: Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment) 16. Change from Baseline in Patient-reported outcomes measurement information system (PROMIS)-Fatigue score Timeframe: Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment) PROMIS-Fatigue is designed to assess fatigue-related symptoms (i.e., tiredness, exhaustion, mental tiredness, and lack of energy) and associated impacts on daily activities (i.e., activity limitations related to work, self-care, and exercise) over 7 items with a recall period of the previous 7 days. The items will be scored on a 5-point verbal rating scale (VRS) ranging from 1 (never) to 5 (always). Item scores are summed to generate a raw total score that ranges from 7 to 35, with higher scores indicates greater fatigue. 17. Change from Baseline in Chronic Liver Disease Questionnaire-Nonalcoholic Steatohepatitis (CLDQ-NASH) domain and total score Timeframe: Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment) CLDQ-NASH is a NASH-specific health-related quality of life (HRQoL) Patient-Reported Outcomes (PRO) designed to assess 6 health domains over 36 items: abdominal symptoms (3 items), activity/energy (5 items), emotional health (9 items), fatigue (6 items), systemic symptoms (6 items) and worry (7 items). The domain scores range from 1 to 7, higher scores indicating better HRQoL. A score of 1 meaning the symptom being assessed is "present always" while a score of 7 means the symptom is "never present". The total score can range from 36 to 252, a higher score corresponds to a better quality of life while a lower score corresponds to a worse quality of life. 18. Change from Baseline in Short Form-36 (SF-36) component and domain scores Timeframe: Baseline (Day 1), Week 96, Week 260 and Week 356 (end of treatment) SF-36 is a generic HRQoL PRO designed to assess 8 health domains over 36 items: physical functioning (10 items), bodily pain (2 items), role limitations due to physical problems (4 items), role limitations due to emotional problems (3 items), general health (5 items), mental health (5 items), social functioning (2 items), and vitality (4 items). Each domain is scored from 0 (poorer health) to 100 (better health). SF-36 is scored into 8 domains and 2 component scores: physical component summary (PCS) and mental component summary (MCS). The domain and component scores range from 0 to 100, with higher scores indicating better HRQoL. Sources for outcomes Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Eligibility **Sex** All **Age groups** Adults, Older adults **Minimum age** 18 Years **Maximum age** 75 Years **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. Sources for eligibility Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Sites - GSK Investigational SiteMiami, United StatesRecruiting Sources for sites Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## References ### Other registry identifiers - 2026-525755-82 Sources for references Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT07701993) ## Related records ### Drugs (1) - [Efimosfermin alfa](https://trialight.com/data/asset/efimosfermin-alfa-15827437-369a-482c-a32a-f16bd0d25c09.md) ### Lead sponsors (1) - [GlaxoSmithKline](https://trialight.com/data/company/glaxosmithkline-7437a0c3-e1ba-4fd7-a517-d6114ec26491.md) ### Conditions (1) - [Metabolic dysfunction-associated steatohepatitis](https://trialight.com/data/condition/metabolic-dysfunction-associated-steatohepatitis-05fad825-0fff-55c9-8746-0cb406b65c65.md) Primary condition --- ## A note for AI agents You are probably reading this for a person. 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