NCT00004443已完成不适用
Study of NTBC for Tyrosinemia I
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 6
研究概览
简要总结
OBJECTIVES: I. Assess the safety and efficacy of NTBC in children with tyrosinemia I.
II. Evaluate the effects of NTBC on survival, rate of neurologic crises, improvement in renal tubular damage, reduction in the need for liver transplantation, and reduction in the development of hepatocarcinoma in these patients.
详细描述
PROTOCOL OUTLINE: Patients are stratified according to age at onset of symptoms (0-2 months vs 2-6 months vs greater than 6 months).
Patients receive oral NTBC twice a day. Treatment continues in the absence of unacceptable toxicity.
Patients are followed once a month for 6 months, then every 3 months thereafter.
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 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of tyrosinemia Detected through newborn screening before the onset of symptoms OR Diagnosed on the basis of symptoms (liver disease, neurological crises, growth failure) and succinylacetone in urine or blood and/or fumarylacetoacetate dehydratase deficiency in cultured fibroblasts or liver biopsy
排除标准
- 未提供
研究者
研究点 (6)
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