A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Efruxifermin in Subjects With Non-invasively Diagnosed Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Nonalcoholic Fatty Liver Disease (NAFLD)/Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 700
- 试验地点
- 1
- 主要终点
- Number of participants with adverse events
研究概览
简要总结
The aim of this study is to assess the safety and tolerability of EFX compared to placebo in subjects with non-invasively diagnosed NASH/MASH and NAFLD/MASLD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Main Study Only:
- •Males and non-pregnant, non-lactating females between 18 - 80 (between 19-80 in the Republic of Korea) years of age inclusive, on the day of signing informed consent
- •Previous history or presence of 2 out of 4 components of metabolic syndrome (obesity, dyslipidemia, elevated blood pressure, elevated fasting glucose) or type 2 diabetes
- •Suspected or confirmed diagnosis of NASH/MASH or NAFLD/MASLD or non-invasively diagnosed NASH/MASH or NAFLD/MASLD
- •Open-Label Rollover
- •Prior participation in a previous Akero Phase 2 study
排除标准
- •Other causes of liver disease based on medical history and/or liver histology and/or central laboratory results, including but not limited to: alcoholic liver disease, autoimmune disorders (e.g., primary biliary cholangitis [PBC], primary sclerosing cholangitis [PSC], autoimmune hepatitis), drug induced hepatotoxicity, Wilson disease, clinically significant iron overload, or alpha-1-antitrypsin deficiency
- •Type 1 or unstable Type 2 diabetes
- •A reduced list of inclusion and exclusion criteria apply to participants in the open-label rollover extension.
- •Other inclusion and exclusion criteria may apply.
研究组 & 干预措施
EFX 50 mg
干预措施: Efruxifermin (Drug)
Placebo
干预措施: Placebo (Drug)
EFX 50 mg (Open-Label Rollover)
干预措施: Efruxifermin (Drug)
结局指标
主要结局
Number of participants with adverse events
时间窗: 52 Weeks
An adverse event (AE) is any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study drug, whether or not related to the study drug.
Number of participants with adverse events by severity
时间窗: 52 Weeks
All AEs, both serious and non-serious, will be assessed for severity using the Common Terminology Criteria for Adverse Events v5.0.
Number of participants with clinically significant changes in clinical assessments
时间窗: 52 Weeks
Clinical assessments include clinical laboratory tests, electrocardiogram, ultrasounds, vital sign assessments, and concomitant medication usage.
Extent of exposure
时间窗: 52 Weeks
A participant's extent of exposure to study drug (weeks) will be generated from the data recorded in the study drug administration eCRF.
次要结局
- Percentage of participants with a reduction in ELF score by ≥ 0.5(52 Weeks)
- Percentage of participants with a reduction in LSM by ≥ 30%(52 Weeks)
- Change from baseline in lipoproteins(52 Weeks)
- Change from baseline in markers of glycemic control: HbA1c (%)(52 Weeks)
- Change from baseline in markers of glycemic control: adiponectin (mg/L)(52 Weeks)
- Change from baseline in markers of liver injury(52 Weeks)
- Change from baseline in markers of liver injury: uric acid (mg/dL)(52 Weeks)
- Change from baseline in body weight (kg)(52 Weeks)
- Percentage of participants with reduction in enhanced liver fibrosis (ELF) score by ≥ 0.5 and reduction in liver stiffness measurement (LSM) by ≥ 30%(52 Weeks)
- Change from baseline in non-invasive marker ELF score(52 Weeks)
- Change from baseline in non-invasive marker pro-peptide of type 3 procollagen (Pro-C3)(52 Weeks)
- Change from baseline in non-invasive marker liver stiffness assessed by transient elastography (kPa, CAP)(52 Weeks)