An Open-Label Study to Evaluate the Long-Term Safety and Pharmacodynamic Efficacy of AT-007 in Adult Subjects With Classic Galactosemia (CG)
试验速览
- 阶段
- 3 期
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by physical exam parameters.
研究概览
简要总结
This study is a 12-month open-label extension (OLE) study of AT-007 in adult subjects with CG who previously participated in Study AT-007-1001 Part D and/or Part D Extension.
The study is designed to assess the long-term safety of AT-007 in subjects with CG as well as the pharmacodynamics (PD) (inhibition of galactitol) and PK of AT-007. The effect of 12-month treatment with AT-007 on the levels of galactose and other galactose metabolites in subjects with CG will also be evaluated.
详细描述
Primary:
- To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with Classic Galactosemia (CG)
Secondary:
- To evaluate the long-term inhibition of galactitol, a biomarker of aldose reductase (AR) activity, induced by 12-month oral administration of AT-007 to adult subjects with CG
- To evaluate the pharmacokinetic (PK) parameters of 12-month oral administration of AT-007 in adult subjects with CG
Exploratory:
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
- •Male or non-pregnant, non-lactating female subject with a CG diagnosis
- •Previous participation in Study AT-007-1001 Part D and/or Part D Extension
- •No other significant health problems which preclude participation
排除标准
- •Concomitant use of certain medications or over-the-counter therapies
- •Discontinuation from Study AT-007-1001 Part D and/or Part D Extension due an adverse event
研究组 & 干预措施
AT-007
Open-label AT-007 20 mg/kg once daily
干预措施: AT-007 (Drug)
结局指标
主要结局
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by physical exam parameters.
时间窗: 12 months
Number of participants with clinically-significant changes in physical examination findings, vital sign abnormalities, and electrocardiogram findings.
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events.
时间窗: 12 months
Number of participants with treatment-emergent adverse events and broken down by organ class and relatedness.
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by clinical laboratory test results.
时间窗: 12 months
Number of participants with clinically-significant changes in clinical laboratory test results.
次要结局
- To evaluate the pharmacokinetic (PK) parameter maximum concentration (Cmax) of 12-month oral administration of AT-007 in adult subjects with CG(12 months)
- To evaluate the pharmacokinetic (PK) parameter Area-Under-the-Curve (AUC) of 12-month oral administration of AT-007 in adult subjects with CG (mg*h/L)(12 months)
- To evaluate the long-term change of galactitol, a biomarker of AR activity, induced by 12-month oral administration of AT-007 to adult subjects with CG(12 months)