"4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals"
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease.
研究概览
简要总结
The purpose of this study is to find out whether 4-PBA will increase the level of AAT in persons with AAT deficiency whether or not they have liver disease.
详细描述
The purpose of this study is to determine whether 4-PBA will significantly increase serum Z AAT levels in AAT-deficient individuals with and without evidence of hepatocellular injury and to assess its effects on liver injury.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Age 18-65
- •Serum A1-PI levels <11uM an appropriate genetic phenotype/genotype
- •5 of 10 subjects must have documented laboratory evidence of liver disease
- •Willingness to withhold Prolastin therapy for 6 weeks prior to screening and throughout the 4-PBA dosing period (up to 3 months)
排除标准
- •Any cause of liver disease other than Alpha-1 Antitrypsin deficiency
- •Evidence of advanced liver disease
- •HIV positive
- •Use of systemic steroids, ursodeoxycholic acid (Actigall, Urso), or herbs in the prior 6 months
研究组 & 干预措施
4-PBA
The study will involve a 4-PBA dose escalation and pharmacokinetics component The study group will be comprised of a total of at least 10 AAT-deficient,(phenotype ZZ referred to as PiZZ) patients. These patients will be divided into two groups: with and without clinical evidence of mild to moderate hepatocellular injury.
干预措施: 4-PBA (Drug)
结局指标
主要结局
To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease.
时间窗: 10 days
次要结局
- To determine the pharmacokinetics of 4-PBA(10 days)