Study of Tetrathiomolybdate in Patients With Wilson Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 90
- 试验地点
- 2
研究概览
简要总结
OBJECTIVES:
Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.
详细描述
PROTOCOL OUTLINE: This a double blind, randomized study. Patients are randomized into one of two treatment arms.
Arm I: Patients receive tetrathiomolybdate (TM) 3 times a day with meals and 3 times a day between meals for 8 weeks in the absence of neurologic deterioration or unacceptable toxicity.
Arm II: Patients receive trientine therapy for 8 weeks in the absence of neurologic deterioration and unacceptable toxicity.
Additional therapy (off study): Patients in the TM group may receive maintenance zinc, while those in the trientine group may continue on trientine or switch to zinc.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 主要目的
- Treatment
- 盲法
- Double
入排标准
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •PROTOCOL ENTRY CRITERIA:
- •-Disease Characteristics--
- •Wilson disease presenting with neurologic or psychiatric symptoms
- •No concurrent seizure activity
- •No white matter lesions on brain magnetic resonance imaging
- •-Prior/Concurrent Therapy--
- •No more than 2 weeks of prior therapy
- •No penicillamine or trientine for longer than 2 weeks
- •-Patient Characteristics--
- •Hepatic: No severe hepatic failure
- •Other: No psychiatric or medical contraindication to protocol therapy
- •Not pregnant
排除标准
- 未提供