NCT07791043招募中2 期
A Dose-finding, Double-blind, Randomized, Placebo-controlled Phase 2 Study to Evaluate the Efficacy and Safety of Treatment With Efimosfermin Alfa in Adult Participants With Alcohol-related Liver Disease (ALD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 274
- 试验地点
- 3
- 主要终点
- Change from Baseline in vibration-controlled transient elastography-liver stiffness measurement (VCTE-LSM)
研究概览
简要总结
This is a dose-ranging study to evaluate safety of efimosfermin alfa and to establish proof-of-concept that efimosfermin alfa therapy provides benefit in participants with ALD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be 18 to 75 years of age inclusive, at the time of Screening.
- •Capable of giving signed informed consent prior to the performance of any study-specific procedures.
- •Able and willing to comply with all study assessments and adhere to the protocol schedule of activities.
- •History of heavy alcohol consumption for greater than 6 months at any time prior to Screening.
- •A female participant is eligible to participate if they are not pregnant or breastfeeding, and one of the following conditions applies:
- •Is a Participant of non-childbearing potential (PONCBP) OR
- •Is a Participant of childbearing potential (POCBP) and using a contraceptive method that is highly effective, with a failure rate of less than (<)1 percentage (%), 30 days prior to and during the study intervention period and for at least 16 weeks after the last dose of study intervention.
排除标准
- •Other primary causes of liver disease (e.g., viral hepatitis, primary biliary cholangitis, primary sclerosing cholangitis, autoimmune hepatitis, drug-induced hepatotoxicity, Wilson disease, hemochromatosis, alpha-1-antitryspin deficiency, etc.). Alcohol must be the primary cause of liver disease.
- •Co-infection with Hepatitis B virus (HBV), Hepatitis C virus (HCV), or Human immunodeficiency virus (HIV) as indicated from the central lab
- •History of diabetes mellitus (DM), either:
- •Type 1 DM
- •Type 2 DM with unstable glycemic control or with major complications.
- •History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer), treated or untreated, within the 3 years prior to Screening 1, regardless of any evidence of local recurrence or metastasis.
- •Current, or history of known hepatocellular carcinoma (HCC).
- •Any major surgery within 3 months prior to Screening 1 or planned during the study
- •Any surgical or medical condition that might jeopardize the individual's safety or compliance with study procedures in the opinion of the investigator.
- •All organ transplant recipients, except for history of corneal transplants, or current listing or active consideration for liver transplant during the Screening period.
- •Poorly controlled hypertension.
- •Evidence of Wernicke-Korsakoff syndrome or alcohol-related dementia.
- •Prior use of a Fibroblast growth factor 21 (FGF21) analogue, including efimosfermin, within the 6 months prior to Screening
- •Individuals with a history of resmetirom use within 3 months prior to Day 1 are to be excluded.
- •Concomitant use of investigational drugs for Metabolic dysfunction-associated steatohepatitis (MASH) or ALD.
- •Current or planned participation in any clinical trial of investigational therapies or medical devices.
- •Exceeding pre-defined laboratory parameters for Triglycerides, Alanine Aminotransferase (ALT), Alkaline Phosphatase (ALP), International normalised ratio (INR), Albumin, Urine albumin-creatinine ratio (uACR) or Glycosylated Hemoglobin (HbA1c).
- •History of drug abuse within the 12 months prior to Day 1 or evidence of such abuse as indicated by laboratory assays conducted at Screening.
- •History of hypersensitivity (such as anaphylaxis or hepatotoxicity) to study intervention and/or its excipients, drugs of similar biological class, FGF21 protein analogs, Fc-fusion proteins, or other protein-based therapeutics.
- •Overt hepatic encephalopathy (West Haven grade >=2).
研究组 & 干预措施
Placebo
Placebo Comparator
Participants will receive placebo.
干预措施: Placebo (Drug)
Efimosfermin alfa Dose Level 2
Experimental
Participants will receive efimosfermin alfa dose level 2.
干预措施: Efimosfermin alfa (Drug)
Efimosfermin alfa Dose Level 1
Experimental
Participants will receive efimosfermin alfa dose level 1.
干预措施: Efimosfermin alfa (Drug)
Efimosfermin alfa Dose Level 3
Experimental
Participants will receive efimosfermin alfa dose level 3.
干预措施: Efimosfermin alfa (Drug)
结局指标
主要结局
Change from Baseline in vibration-controlled transient elastography-liver stiffness measurement (VCTE-LSM)
时间窗: Baseline (Day 1) and up to Week 60
VCTE-LSM is a non-invasive test that uses vibration-controlled transient elastography to measure the stiffness of the liver in kilopascals (kPa)
Change from Baseline in model for end-stage liver disease (MELD) score
时间窗: Baseline (Day 1) and up to Week 60
MELD is a scoring system for assessing the severity of chronic liver disease. MELD scores range between 6 and 40, with 40 being the most severe.
次要结局
- Number of Participants with Anti-drug antibodies (ADA) against efimosfermin alfa(Up to Week 56)
- Number of participants with treatment-emergent adverse events (TEAEs) and TEAEs by severity(Up to Week 60)
- Number of participants with TEAEs leading to discontinuation and TEAEs leading to discontinuation by severity(Up to Week 60)
- Number of participants with Grade 3 and Grade 4 laboratory abnormalities(Up to Week 60)
- Change from Baseline in serum aspartate aminotransferase (AST) (International units per liter)(Baseline (Day 1) and up to Week 60)
- Change from Baseline in a blood test that helps predict the risk of disease progression in liver disease(Baseline (Day 1) and up to Week 52)
- Trough Concentration at steady state (Ctrough,ss) following administration of efimosfermin alfa(Up to Week 56)
- Area Under the concentration-time Curve from Time 0 (pre-dose) to the last quantifiable concentration (AUC[0-t]) of efimosfermin alfa in the subset of participants with optional intensive pharmacokinetics (PK) sampling(Up to Week 4)
- Maximum observed drug concentration (Cmax) of efimosfermin alfa in the subset of participants with optional intensive PK sampling(Up to Week 4)
- Number of participants with clinically significant changes in Vital signs(Up to Week 60)
- Number of participants with clinically significant changes in 12-lead electrocardiogram (ECG) findings(Up to Week 60)
研究者
研究点 (3)
Loading locations...
相似试验
招募中
3 期
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)Metabolic Dysfunction-associated SteatohepatitisNCT07701993GlaxoSmithKline1,740
招募中
3 期
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)Metabolic Dysfunction-Associated Steatohepatitis (MASH)2025-523674-16-00Glaxosmithkline Research & Development Limited210
招募中
3 期
A pivotal clinical study to investigate the safety and efficacy of efimosfermin alfa injection in participants with biopsyconfirmed F2- or F3-stage MASH (BOS-580-301)2025-523675-39-00Glaxosmithkline Research & Development Limited173
招募中
不适用
An Open-label Prospective Study to Evaluate the Efficacy and Safety of Pegfilgrastim in Triple-Negative Breast Cancer Patients Receiving AC Regimen Following Paclitaxel and Carboplatin as Neoadjuvant TherapyTriple-Negative Breast CancerNCT07525869Yonsei University40
进行中（未招募）
4 期
A clinical trial to study the safety and efficacy of Efonidipine + Telmisartan in the treatment of hypertension.CTRI/2024/03/064100Zuventus Healthcare Limited200