NCT01653444已完成1 期
Multicenter and Dose Escalation Phase 1 Study to Evaluate the Safety and Exploratory Efficacy of GC1119(Recombinant Human α-galactosidase A) for Enzyme Replacement Therapy in Fabry Disease Patients
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 8
- 试验地点
- 3
- 主要终点
- Incidence of adverse events
研究概览
简要总结
The purpose of this study is to evaluate the safety and exploratory efficacy of GC1119 (recombinant human α-galactosidase A) for enzyme replacement therapy in Fabry disease patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non 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
- Male
- 接受健康志愿者
- 否
入选标准
- •Subjects with a current diagnosis of Fabry's disease
- •Plasma α-gal activity of ≤ 1.5mnol/hr/ml and have a mutation in α-galactosidase A gene
- •Males ≥ 16 years old
- •Subjects capable of performing this clinical trial in an appropriate manner
- •Informed consent form voluntarily signed by the subject(or his legally acceptable representative if the subject is under 20 years old) to participation in the study
- •Agreement to contraception during the study period
排除标准
- •Serum creatinine > 2.5mg/dl
- •Subjects have a plan to kidney transplantation
- •Subjects have undergone kidney transplantation
- •Subjects are currently on dialysis
- •Subjects have a clinically significant organic disease(cardiovascular, hepatic, pulmonary, neurologic, or renal disease)that in the opinion of the investigator would preclude participation in the trial
- •Known life-threatening hypersensitivity(anaphylactic reaction) to α-galactosidase
- •Treatment with another investigational product within 30days from the administration of study drug dosing or plans to be treated with another investigational product during the study period
- •Known hypersensitivity to any of the ingredients of study drug(including excipients)
- •Subjects need the medication of prohibited drug
- •Alcoholism or drug addiction
研究组 & 干预措施
GC1119 0.5 mg/kg
Experimental
0.5 mg/kg biweekly
干预措施: GC1119 (Drug)
GC1119 1.0 mg/kg
Experimental
1.0 mg/kg biweekly
干预措施: GC1119 (Drug)
结局指标
主要结局
Incidence of adverse events
时间窗: 10weeks
次要结局
- change and %change of Plasma GL-3 concentration(baseline and 10weeks)
- The ratio of subjects whose plasma GL-3 values are within reference range(10weeks)
- change and %change of urine GL-3 concentration(baseline and 10weeks)
- change and %change of kidney function(baseline and 10weeks)
- change and %change of kidney size(baseline and 10weeks)
- change and %change of heart size(baseline and 10weeks)
- change of results of cornial opacity examination(baseline and 10weeks)
- change of scores that are measured by pain questionnaire(baseline and 10weeks)
研究者
研究点 (3)
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