NCT00837824终止2 期
A Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal Disease
Genzyme, a Sanofi Company9 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2002年12月1日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 20
- 试验地点
- 9
- 主要终点
- Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease
研究概览
简要总结
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •provided written informed consent prior to any study-related procedures being performed.
- •be ≥16 years old.
- •have a current diagnosis of Fabry disease (defined as abnormal α-galactosidase (α GAL) enzyme levels or Fabry genotype).
- •have one of the following clinical conditions present at enrollment: serum creatinine level greater than 3.0 mg/dL (an average of two values at least one week apart), or be currently on dialysis, or be status post kidney transplant by greater than 3 months.
- •have the ability to comply with the requirements of the protocol
- •have a negative pregnancy test, if a female patient of childbearing potential. In addition, all female patients of childbearing potential must use a medically accepted method of contraception throughout the study.
排除标准
- •if they did not meet the specific inclusion criteria.
- •if they had participated in a study employing an investigational drug within 30 days of the start of their participation in this trial.
- •had previously received enzyme replacement therapy (ERT) for their Fabry disease.
- •had diabetic nephropathy.
- •were pregnant or lactating.
- •were unwilling to comply with the requirements of the protocol.
结局指标
主要结局
Time to Clinically Significant Progression of Cardiac Disease, Cerebrovascular Disease, and/or Death Among Fabry Patients With Severe Kidney Disease
时间窗: 7 months
The trial was terminated early due to inadequate study design. During the study period of 7 months, only 1 patient had a clinical event, a stroke, in the Fabrazyme 1 mg/kg treatment arm. The time to event was determined from first dose of Fabrazyme to the date of event.
次要结局
- Plasma Globotriaosylceramide (GL-3)(Evaluated at Baseline, Month 3, and Final Visit)
研究者
研究点 (9)
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