EUCTR2018-001362-41-GB进行中（未招募）1 期
A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single-ascending and Multiple-dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT02 in Healthy Adult Subjects and Patients with ZZ Type Alpha 1 Antitrypsin Deficiency Liver Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 96
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Inclusion Criteria for All Participants in Part A and Part B
- •- Male or female, aged 18 to 65 years, inclusive
- •- Body mass index (BMI) =18.0 kg/m2 and =30 kg/m2
- •- Nonsmoker for at least 5 years before screening
- •Additional Inclusion Criteria for Subjects in Part A
- •- AAT levels within normal limits
- •- FEV1 =85% of predicted and FEV1/forced vital capacity ratio =0.7
- •Additional Inclusion Criteria for Patients in Part B
- •- Documented ZZ type AAT by genotype
- •- Liver biopsy performed within 90 days of the first dose of study drug demonstrating liver histopathology consistent with PiZZ AATD liver disease meeting the following criteria:
- •a.Presence of diastase-resistant PAS-positive globules in hepatocytes
- •b.Ishak fibrosis score of <4
- •- Post-bronchodilator FEV1 =70% of predicted and diffusing capacity of the lung for carbon monoxide =50% of predicted
- •- Prior/Concomitant Therapy: If on any maintenance medication regimen, likely, in the opinion of the Investigator, to be able to remain on a stable medication regimen for the duration of the study (no new medications within 30 days prior to first dose of study drug)
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 96
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Exclusion Criteria for All Participants in Part A and Part B
- •- Has known active human immunodeficiency virus (HIV) infection, or evidence of current or chronic hepatitis C virus (HCV) or hepatitis B virus (HBV) infection
- •- Has an estimated glomerular filtration of =45 mL/min/1.73 m2 at screening
- •- Has any clinical safety laboratory result considered clinically significant and unacceptable by the Investigator during screening or on Day 1
- •- Received an investigational agent within 30 days or within 5 elimination half-lives, whichever is longer prior to the first dose of study drug, or are in follow-up of another clinical study prior to study enrollment
- •- Is unwilling or unable to limit alcohol consumption throughout the course of the study. Alcohol intake of >2 units/day is excluded during the study
- •- Has history of alcohol abuse, within the last 12 months before screening, in the opinion of the Investigator
- •- Has known history or clinical evidence of drug abuse, within the 12 months before screening.
- •Additional Exclusion Criteria for Subjects in Part A
- •- Is positive for the rs1303 single nucleotide polymorphism in the SERPINA1 gene
- •- Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >upper limit of normal (ULN) at screening
- •- Has total bilirubin above the ULN
- •- Has history of asthma or recurrent or chronic lung disease, excluding childhood asthma that has resolved
- •- Has history of chronic liver disease from any cause
- •Additional Exclusion Criteria for Patients in Part B
- •- Has any of the following laboratory parameter assessments at screening:
- •a. Alanine aminotransferase or AST =1.5× ULN
- •b. Total bilirubin >ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible.
- •c. Serum albumin level =80% of the LLN
- •d. Platelet count =100,000 per microliter
- •e. International normalized ratio (INR) or prothrombin time (PT) >1.2 of the clinical laboratory reference range
- •- Receiving augmentation therapy for AAT deficiency or who have received augmentation therapy within 8 weeks of first dose of study drug
- •- Has history of chronic liver disease from any known cause other than ZZ type AAT deficiency
- •- Has history of hepatic encephalopathy
- •- Has history of gastrointestinal bleeding from esophageal or gastric varices complicating portal hypertension or ascites
- •- Has any history of a bleeding disorder or inability to abstain from medications that may interfere with normal blood clotting such that the risk of bleeding following liver biopsy would be increased
研究者
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