NCT00067470已完成3 期
Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 39
- 试验地点
- 1
- 主要终点
- Change From Baseline in 12-minute Walk Test at 24 Weeks
研究概览
简要总结
The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.
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
- Double
入排标准
- 年龄范围
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
rhASB
Active Comparator
干预措施: N-acetylgalactosamine 4-sulfatase (Drug)
结局指标
主要结局
Change From Baseline in 12-minute Walk Test at 24 Weeks
时间窗: baseline and 24 weeks
Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
次要结局
- Change From Baseline in 3-minute Stair Climb at 24 Weeks(baseline and 24 weeks)
- Change From Baseline in Urinary GAG (uGAG) at 24 Weeks(baseline and 24 weeks)
研究者
研究点 (1)
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