NCT03078881已完成不适用
LUSTRO: A Clinical Assessment Study in Crigler-Najjar Syndrome
Audentes Therapeutics4 个研究点 分布在 3 个国家目标入组 8 人开始时间: 2017年3月10日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 8
- 试验地点
- 4
- 主要终点
- Assess phototherapy usage over the course of the study
研究概览
简要总结
This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has a diagnosis of Crigler-Najjar syndrome resulting from a confirmed mutation in the UGT1A1 gene
- •Subject is aged equal or greater than 1 year of age
- •Subject receives daily phototherapy for a minimum of 6 hours within a 24-hour period (daily illumination time)
排除标准
- •Subject is currently participating in an interventional study or has received gene or cell therapy
- •Subject has received a whole liver, partial liver, or hepatocyte transplant; or subject has a liver transplant scheduled within the planned participation period of this study
- •Subject has significant cholestatic disease, in the opinion of the investigator
- •Subject is receiving phenobarbital or other known inducer of UGT1A1 within 30 days of screening
- •Subject has any clinically significant underlying liver disease (other than Crigler-Najjar syndrome), in the opinion of the investigator
- •Subject has a history of, or currently has, a clinically important condition other than Crigler-Najjar syndrome, in the opinion of the investigator
结局指标
主要结局
Assess phototherapy usage over the course of the study
时间窗: Up to 2 years
Assess variation in bilirubin levels over the course of the study and the variation of bilirubin levels over a 24-hr period
时间窗: Up to 2 years
Characterize the disease course and natural history of subjects with Crigler-Najjar syndrome
时间窗: Up to 2 years
次要结局
- Assess the humanistic and clinical burden of disease in Crigler-Najjar subjects and caregivers as measured by PedsQL(Up to 2 years)
研究者
研究点 (4)
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