臨床研究等提出・公開システム

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)

A pivotal clinical study to investigate the safety and efficacy of efimosfermin compared with placebo in adult participants with compensated cirrhosis due to MASH (NEBULA 1)

Mendez Patricia

GlaxoSmithKline K.K.

Akasaka Intercity AIR, 1-8-1 Akasaka, Minato-ku, Tokyo, Japan

jp.gskjrct@gsk.com

Mendez Patricia

GlaxoSmithKline K.K.

Akasaka Intercity AIR, 1-8-1 Akasaka, Minato-ku, Tokyo, Japan

+81-120-561-007

jp.gskjrct@gsk.com

Pending

July. 27, 2026

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

1. Participants aged between 18 and 75 years at enrollment.
2. Participants with compensated cirrhosis due to MASH, confirmed by non-invasive assessments.
3. Participants with history or presence of at least two components of metablic syndrome.

1. Participants with other chronic liver diseases.
2. Participants with evidence or history of decompensated liver disease or hepatocellular carcinoma.
3. Participants with history of Type 1 diabetes mellitus or major Type 2 diabetes complications.
4. Participants with history or evidence of chronic pancreatic disease; pancreatic injury or acute pancreatitis within 6 months before screening.
5. Participants with a recent history or planned surgical procedures or medications intended to produce significant weight loss.
6. Participants with ALT or AST >= 5 times upper limit normal.
7. Participants with current or history of excessive alcohol intake.

Both

Compensated cirrhosis due to metabolic dysfunction-associated steatohepatitis

Efimosfermin alfa or placebo (SC injection) will be administered.

Time from randomization to an adjudicated composite clinical outcome: liver-related outcome comprises all-cause mortality; liver transplantation; occurrence of significant hepatic events.

- Proportion of participants achieving change from Baseline in VSTE-LSM and ELF score at Week 96 and Week 260.
- Proportion of participants achieving change from Baseline in VCTE-LSM at Week 96 and Week 260.
- Proportion of participants with treatment-emergent adverse events (TEAEs) and TEAEs by severity at Week 96, Week 260, and Week 356.
- Proportion of participants with TEAEs leading to discontinuation by severity at Week 96, Week 260, and Week 356.
- Proportion of participants with Grade 3 and Greade 4 laboratory abnormalities at Week 96, Week 260, and Week 356.
- Absolute change from Baseline in VCTE-LSM at Week 96, Week 260, and Week 356.
- Relative change from Baseline in VCTE-LSM at Week 96, Week 260, and Week 356.
- Absolute change from Baseline in MRE scores at Week 96 and Week 260.
- Relative change from Baseline in MRE scores at Week 96 and Week 260.
- Absolute change from Baseline in ELF scores at Week 96, Week 260, and Week 356.
- Relative change from Baseline in ELF scores at Week 96, Week 260, and Week 356.
- Proportion of participants experiencing improvement in ELF score at Week 96, Week 260, and Week 356.
- Change from Baseline in HbA1c and Percentage of HbA1c in participants with T2DM at Week 96, Week 260, and Week 356.
- Change from Baseline in fasting glucose in participants with T2DM at Week 96, Week 260, and Week 356.
- Change from Baseline in fasting total cholestrerol, LDL, HDL, and fasting triglicerides at Week 96, Week 260, and Week 356.
- Change from Baseline in PROMIS-Fatigue score at Week 96, Week 260, and Week 356.
- Change from Baseline in CLDQ-NASH domain and total score at Week 96, Week 260, and Week 356.
- Change from Baseline in SF-36 component and domain scores at Week 96, Week 260, and Week 356.

+81-584-81-3341

No

Argentina/Australia/Austria/Belgium/Brazil/Bulgaria/Canada/Chile/China/France/Germany/Greece/Hong Kong/India/Israel/Italy/South Korea/Mexico/Netherlands/New Zealand/Poland/Saudi Arabia/Singapore/Spain/Taiwan/Turkiye/United Kingdom/United States/Puerto Rico