Study of Bile Acids in Patients With Peroxisomal Disorders
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 25
研究概览
简要总结
OBJECTIVES: I. Determine the effectiveness of oral bile acid therapy with cholic acid, chenodeoxycholic acid, and ursodeoxycholic acid in patients with peroxisomal disorders involving impaired primary bile acid synthesis.
II. Determine whether suppression of synthesis of atypical bile acids and enrichment of bile acid pool with this regimen is effective in treating this patient population and improving quality of life.
详细描述
PROTOCOL OUTLINE: Patients receive oral cholic acid and oral chenodeoxycholic acid on day 1. On day 4, patients receive oral cholic and ursodeoxycholic acids. Patients are assessed at 3 and 6 months for liver function response, neurologic status, and nutritional status.
Patients receive treatment until disease progression or unacceptable toxic effects are observed.
Completion date provided represents the completion date of the grant per OOPD records
研究设计
- 研究类型
- Interventional
- 主要目的
- Treatment
入排标准
- 年龄范围
- — 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Biochemically proven peroxisomal disorder, including:
- •Zellweger syndrome
- •Pseudo-Zellweger syndrome
- •Neonatal adrenoleukodystrophy
- •Bifunctional enzyme deficiency
- •Infantile Refsum's disease
排除标准
- 未提供