A clinical study to investigate the safety and tolerability of efimosfermin alfa injection in participants with known or suspected F2- or F3-stage MASH (BOS-580-302)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Glaxosmithkline Research & Development Limited

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Decision date (initial)

2026-06-14

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

GlaxoSmithKline Research & Development Limited

External identifiers

EU CT number

2025-523674-16-00

ClinicalTrials.gov

NCT07221188

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety

To assess the effects of efimosfermin on safety and tolerability.

Secondary objectives 8

1. To assess the effects of efimosfermin on ELF score
2. To assess the effects of efimosfermin on VCTE-LSM score.
3. To assess the effects of efimosfermin on MRE score
4. To assess the effects of efimosfermin on HFF and ALT normalization.
5. To assess the effects of efimosfermin on glycemic and metabolic biomarkers.
6. To assess the effects of efimosfermin on lipids.
7. To assess the immunogenicity of efimosfermin.
8. To assess the steady-state PK of efimosfermin.

Conditions and MedDRA coding

Metabolic Dysfunction-Associated Steatohepatitis (MASH)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

Yes

IPD plan description

Qualified researchers may request access to anonymized individual patient-level data (IPD) and related study documents of the eligible studies via the Data Sharing Portal. Details on GSK's data sharing criteria can be found at: https://www.gsk.com/en-gb/innovation/trials/data-transparency/

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Able and willing to understand and sign a written informed consent form (ICF) that must be obtained prior to the initiation of study procedures
2. Age >=18 through <=75 years at enrolment
3. History or presence of 2 or more of the 5 components of metabolic syndrome per American Heart Association definition
4. History or presence of known or suspected MASH

Exclusion criteria 13

1. ALT or AST >=5 × upper limit of normal (ULN)
2. Total bilirubin (BILI) >=1.3 milligram per deciliter (mg/dL). Individuals with documented Gilbert's syndrome may be enrolled if they experienced an isolated increase in total BILI of >=1.3 mg/dL and direct BILI is <=20% of total BILI; otherwise, the individual will be excluded.
3. Serum albumin <=3.5 grams per deciliter (g/dL)
4. International normalized ratio (INR) >=1.3 not due to therapeutic anticoagulation. Individuals receiving chronic anticoagulant treatment with higher INR values may be enrolled at the discretion of the Investigator and Study Medical Monitor.
5. Alkaline phosphatase (ALP) >=2 × ULN
6. Platelet (PLT) count <140 000 per (/) cubic millimeter (mm^3); individuals with a PLT count between 110,000/mm^3 and 140,000/mm^3 may be enrolled after discussion with the Study Medical Monitor
7. Serum creatinine >=1.5 mg/dL or creatinine clearance <=60 milliliter (mL)/minute (min)/1.73 square meter by Chronic Kidney Disease Epidemiology Collaboration equation.
8. HbA1c >=9.0%
9. Model for End-Stage Liver Disease (MELD) 3.0 score >=12 unless the score is elevated in the absence of liver dysfunction (eg, Gilbert's syndrome)
10. Phosphatidylethanol (PEth) >=80 nanogram per milliliter (ng/mL) at Screening
11. Evidence of infection with any of the following: a. Human immunodeficiency virus; b. Hepatitis B virus (detectable HBsAg at Screening); c. Hepatitis C virus (HCV)
12. Chronic liver disease from any other cause including, but not limited to, alcoholic liver disease; evidence of portal hypertension; viral hepatitis, or any history or evidence of cirrhosis; or decompensated liver disease such as clinical ascites, bleeding gastroesophageal varices, hepatorenal syndrome, or hepatic encephalopathy prior to Screening or Day 1.
13. Current or history of excessive alcohol intake for >=3 months within the 12-month period prior to Screening

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. For all participants at Week 52, incidence and severity of TEAEs.
2. For all participants at Week 52, incidence and severity of TEAEs leading to discontinuation.
3. For all participants at Week 52, incidence of Grade 3 and Grade 4 laboratory abnormalities.

Secondary endpoints 14

1. Absolute and relative change from baseline to Week 52 in ELF score for all participants.
2. Achieving an improvement in ELF score of ≥ 0.5.
3. Absolute and relative change from baseline to Week 52 in VCTE-LSM scores for all participants.
4. Achieving a change from baseline in VCTE-LSM ≥ 30% at Week 52.
5. Absolute and relative change from baseline to Week 52 in the subset of participants with MRE scores.
6. Absolute and relative change from baseline to Week 52 in HFF by MRI-PDFF for all participants.
7. Absolute and relative change from baseline to Week 52 in ALT, AST, and ALT/AST ratio for all participants.
8. Achieving ALT and HFF normalization at Week 52.
9. Achieving HFF ≤ 5% at Week 52.
10. Change from baseline to Week 52 in HbA1c for participants with T2DM.
11. Change from baseline to Week 52 in body weight for all participants.
12. Change from baseline in fasting total cholesterol, LDL-C, HDL-C, and fasting triglycerides at Week 52 for all participants.
13. Incidence of ADAs at Week 52.
14. Efimosfermin serum concentrations in participants with PK data following multiple doses.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Glaxosmithkline Research & Development Limited

Sponsor organisation

Glaxosmithkline Research & Development Limited

Address

79 New Oxford Street

City

London

Postcode

WC1A 1DG

Country

United Kingdom

Scientific contact point

Organisation

Glaxosmithkline Research & Development Limited

Contact name

EU GSK Clinical Trials Call Center

Public contact point

Organisation

Glaxosmithkline Research & Development Limited

Contact name

EU GSK Clinical Trials Call Center

Third parties 15

Locations

9 EU/EEA countries · 88 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 167 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications