NCT00048711已完成2 期
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 试验地点
- 2
- 主要终点
- 12-minute walk test
研究概览
简要总结
The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS 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
- 5 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 be five years of age or older
- •Patient must have documented biochemical or genetic proof of MPS VI
- •Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 12-minute walk test
- •If female of childbearing potential, patient must have a negative pregnancy test
排除标准
- •Patient is under consideration for or has undergone a successful bone marrow transplant (BMT).
- •Pregnant or lactating patient
- •Patient has received an investigational drug within 30 days prior to study enrollment
- •Patient has been previously treated with rhASB
- •Patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly confound study results or decrease study compliance
- •Patient has a known hypersensitivity to rhASB or to components of the study drug
- •History of cancer (except low grade and fully resolved skin malignancy)
结局指标
主要结局
12-minute walk test
时间窗: weeks 6, 12, 24, 48, 96 and 144
urinary glycoaminoglycan(uGAG)levels
时间窗: weeks 1, 4, 6, 8, 12
次要结局
未报告次要终点
研究者
研究点 (2)
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