Investigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid Metabolism
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 85
- 试验地点
- 1
- 主要终点
- Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
研究概览
简要总结
OBJECTIVES:
I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism
II. To assess the safety and tolerability of cholic acid
详细描述
Investigational Plan:
A Phase III, open label, single arm, nonrandomized, non-comparative, compassionate treatment study of cholic acid in the treatment of defects of bile acid metabolism.
The study was begun with a single study site at Cincinnati Children's Hospital Medical Center (CCHMC), but in 2005 was expanded so that compassionate treatment could be provided to additional patients who had been identified with inborn errors of bile metabolism through the center's screening/diagnostic program.
Patients who were screened were contacted and evaluated with respect to the inclusion/exclusion criteria. Signed informed consent by the patient and/or parents/legal guardian was obtained as soon as it is confirmed that the patient met inclusion/exclusion criteria and the parents/guardian would agree for the child to participate in the study.
The primary interventions for the study were:
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Cholic Acid
干预措施: Cholic Acids (Drug)
结局指标
主要结局
Number of Participants With Excretion of Atypical Bile Acids in Urine by Category
时间窗: Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years
Patients with excretion of atypical bile acids in urine by category, from worst status before treatment (baseline, BL) to best status on treatment (OT)
次要结局
- Adverse Events(Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years)
- Liver Histology(At baseline (if no historical data were available) and between 1 and 6 months following treatment start.)
- Change in Liver Function Tests (LFTs) Measured in Serum(Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years)
- Height and Weight(Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years)