NCT06836609招募中1 期
A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Two-Part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of a Single Dose of ALN-CIDEB in Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) and Two Doses of ALN-CIDEB in Adult Participants With Metabolic Dysfunction-Associated Steatohepatitis (MASH)
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 132
- 试验地点
- 4
- 主要终点
- Severity of TEAEs
研究概览
简要总结
This study is researching an experimental drug called ALN-CIDEB, also referred to as "study drug". The study is focused on participants with metabolic dysfunction-associated steatotic liver disease (MASLD) (Part A) and metabolic dysfunction-associated steatohepatitis (MASH) (Part B). MASLD and MASH are long-lasting liver conditions caused by having too much fat in the liver.
The aim of the study is to see how safe and tolerable the study drug is.
The study is looking at several other research questions, including:
- What side effects may happen from taking the study drug
- How the study drug works to change liver fat content
- How much study drug and study drug metabolites (byproducts of the body breaking down the study drug) are in the blood at different times
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Part A: 18 to 55 years at Screening Visit 1 with MASLD, at Screening Visit 1 Part B: 18 to 65 years at Screening Visit 1 with a diagnosis of MASH, at Screening Visit 1
- •Body Mass Index (BMI) ≥30 kg/m2 and ≤40 kg/m2 at Screening Visit 1
- •Controlled-Attenuation Parameter (CAP) ≥285 dB/m by FibroScan during screening as described in the protocol
- •Liver fat content ≥8.5% by MRI-PDFF during screening
- •If on anti-hypertensive and/or lipid lowering medications and/or glucose lowering medications, must be on generally stable dose(s) for at least 12 weeks prior to screening and no changes to the dose(s) are anticipated during the study
- •Part B: A diagnosis of MASH documented in the participant's medical history, or a clinical suspicion of MASH based on non-invasive biomarkers (eg, evidence of fatty liver on imaging and elevated liver enzymes) and clinical risk factors, including having a history of 2 or more elements of metabolic syndrome, as defined in the protocol
- •Part B: Screening percutaneous liver biopsy NAFLD Activity Score (NAS) ≥3 and fibrosis stage, as defined in the protocol
排除标准
- •Known historical or current diagnosis of portal hypertension or cirrhosis based on clinical assessment, imaging, and/or liver biopsy
- •Known historical or current diagnosis of other forms of chronic liver disease, as defined in the protocol
- •Prior or current suspected or known drug-induced liver injury within 1 year prior to screening
- •History of liver transplant, current placement on a liver transplant list, or Model for End-stage Liver Disease (MELD) score >12
- •Contraindication to MRI examinations, such as persons with cardiac pacemaker and implants made of metal, severe claustrophobia, size restrictions, or other contraindications for MRI
- •Liver stiffness measurement, laboratory parameter assessment, estimated Glomerular Filtration Rate (GFR), and evidence of uncontrolled hypertension, as defined in the protocol
- •Evidence of Human Immunodeficiency Virus (HIV) infection, Hepatitis B Virus (HBV) infection, or Hepatitis C Virus (HCV) infection during screening, as described in the protocol
- •History of Type 1 Diabetes
- •Bariatric surgery, including any procedures to revise, reverse, or remove any previous bariatric surgery interventions, within approximately 5 years prior to randomization or planned during the study period
- •NOTE: Other protocol-defined inclusion/exclusion criteria apply.
研究组 & 干预措施
Part A
Experimental
Randomized per the protocol
干预措施: Placebo (Drug)
Part B
Experimental
Randomized per the protocol
干预措施: Placebo (Drug)
Part A
Experimental
Randomized per the protocol
干预措施: ALN-CIDEB (Drug)
Part B
Experimental
Randomized per the protocol
干预措施: ALN-CIDEB (Drug)
结局指标
主要结局
Severity of TEAEs
时间窗: Up to 48 Weeks
Incidence of Treatment-Emergent Adverse Events (TEAEs)
时间窗: Up to 48 Weeks
次要结局
- Total concentration of potential major metabolite(s) in plasma(Up to 48 Weeks)
- Urinary recovery of ALN-CIDEB as a proportion of the dose(Up to 36 Weeks)
- Total concentration of ALN-CIDEB in plasma(Up to 48 Weeks)
- Urinary recovery of potential major metabolite(s) as a proportion of the dose(Up to 36 Weeks)
- Change in liver fat fraction by Magnetic Resonance Imaging-Proton Density Fat Fraction (MRI-PDFF)(Baseline up to 48 Weeks)
- Change in Aspartate Aminotransferase (AST)(Baseline up to 48 Weeks)
- Change in Alanine Aminotransferase (ALT)(Baseline up to 48 Weeks)
- Change in hepatic Cell death-Inducing DNA fragmentation factor alpha-like Effector B (CIDEB) messenger RiboNucleic Acid (mRNA) level(Baseline up to 36 Weeks)
研究者
研究点 (4)
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