NCT00104234已完成3 期
A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis 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
- 12-Minute Walk Test
研究概览
简要总结
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Patient consent
- •Patient must have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions
- •If female of childbearing potential, patient must have a negative pregnancy test
排除标准
- •Pregnant or lactating patient
- •Patient has received an investigational drug within 30 days prior to study enrollment
- •Patient is unwilling or unable to travel to the primary site for periodic assessments
研究组 & 干预措施
rhASB/rhASB
Other
N-acetylgalactosamine 4-sulfatase
干预措施: N-acetylgalactosamine 4-sulfatase (Drug)
Placebo/rhASB
Other
干预措施: Placebo/rhASB (Drug)
结局指标
主要结局
12-Minute Walk Test
时间窗: Baseline of ASB-03-05 through week 96 of ASB-03-06
Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.
次要结局
- Change in Urinary Glycosaminoglycans (GAG) Level(72 weeks)
- 3-Minute Stair Climb(Baseline ASB-03-05 through week 96 of ASB-03-06.)
研究者
研究点 (1)
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