NCT04565717终止1 期
A Phase 1, Randomized, Double-Blind, Placebo-Controlled, 3-Part Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Dose ALN-HSD in Healthy Adult Subjects and Multiple Dose ALN-HSD in Adult Patients With Nonalcoholic Steatohepatitis (NASH)
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 6 人开始时间: 2020年10月9日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Part C: Change from Baseline of Liver Hydroxysteroid 17β Dehydrogenase 13 (HSD17B13) Messenger Ribonucleic Acid (mRNA)
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of single ascending doses of ALN-HSD in healthy participants (Part A) and multiple doses of ALN-HSD in patients with NASH (Parts B and C).
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)
盲法说明
Parts A&B: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Part C: Open label
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
- 是
入选标准
- •Part A Only
- •Has body mass index (BMI) ≥18 kg/m^2 and ≤28 kg/m^2
- •Has normal 12-lead electrocardiogram (ECG)
- •Parts B and C Only:
- •Has BMI ≥18 kg/m^2 and ≤40 kg/m^2
- •Has a diagnosis of NASH documented in the patient's medical history or a clinical suspicion of NASH based on defined study criteria
- •Has screening liver biopsy with NASH activity score (NAS) score of ≥3 per NASH Clinical Research Network (CRN) criteria
排除标准
- •Parts A, B and C:
- •Has any clinical safety laboratory result considered clinically significant and unacceptable by the Investigator
- •Has known active human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV) infection
- •Has known history or evidence of drug abuse, within 12 months prior to screening
- •Has evidence of other forms of known chronic liver disease
- •Has recently received an investigational agent
- •Has any uncontrolled or serious disease, medical or surgical condition that my interfere with participation or data interpretation
- •Has excessive alcohol intake for ≥ 3 months during past year
- •Has history of intolerance to SC injection(s)
- •Has international normalized ratio (INR) >1.2
- •Has platelet count <140x10^9/L
- •Part A Only
- •Has systolic blood pressure (BP) >140 mmHg and diastolic >90 mmHg;
- •Has used certain prescription drugs within last 14 days prior to screening
- •Has used certain over the counter (OTC) medication within 7 days prior to screening
- •Has estimated glomerular filtration rate (GFR) <90 mL/min/1.73m^2 at screening
- •Parts B and C Only
- •Has abnormal ECG
- •Has changes in certain prescription medications defined in the protocol within the specified timeframe prior to screening
- •Has GFR<45ml/min/1.73m^2
研究组 & 干预措施
Part A: ALN-HSD
Experimental
Participants will be administered a single dose of ALN-HSD.
干预措施: ALN-HSD (Drug)
Part A: Placebo
Placebo Comparator
Participants will be administered a single dose of ALN-HSD-matching placebo.
干预措施: Placebo (Drug)
Part B: ALN-HSD
Experimental
Participants will be administered multiple doses of ALN-HSD.
干预措施: ALN-HSD (Drug)
Part B: Placebo
Placebo Comparator
Participants will be administered multiple doses of ALN-HSD-matching placebo.
干预措施: Placebo (Drug)
Part C: ALN-HSD
Experimental
Participants will be administered multiple doses of ALN-HSD.
干预措施: ALN-HSD (Drug)
结局指标
主要结局
Part C: Change from Baseline of Liver Hydroxysteroid 17β Dehydrogenase 13 (HSD17B13) Messenger Ribonucleic Acid (mRNA)
时间窗: Baseline and Month 6
Parts A and B: Frequency of Adverse Events
时间窗: Part A: Up to 3.5 months; Part B: up to 12.5 months
次要结局
- Part B: Plasma Concentrations of ALN-HSD and Potential Major Metabolite(s)(Day 1 and Month 3 predose and up to 4 hours postdose)
- Part C: Frequency of Adverse Events(Up to 6 months)
- Part A: Fraction Excreted in Urine (fe) of ALN-HSD and Potential Metabolites(Day 1 up to 24 hours postdose)
- Part A: Area Under the Plasma Concentration-time Curve (AUC) for ALN-HSD and Potential Metabolites(Day 1 predose and up to 48 hours postdose)
- Pat A: Maximum Plasma Concentration (Cmax) for ALN-HSD and Potential Metabolites(Day 1 predose and up to 48 hours postdose)
- Part B: Change from Baseline of Liver HSD17B13 mRNA(Predose and up to 9 months postdose)
研究者
研究点 (1)
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