NCT00048620已完成1 期
Double-Blind,2 Dose Group Study 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
- 1 期
- 状态
- 已完成
- 试验地点
- 2
- 主要终点
- Lysosomal storage disease
研究概览
简要总结
The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.
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
- 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 diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI
- •Leukocyte ASB enzyme activity level less than 20% of the normal range
- •Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly.
- •Ability to perform all protocol tests
- •Ability to stand independently for six minutes
- •Sexually active subjects must agree to use an adequate form of contraception
排除标准
- •History of bone marrow transplantation
- •Pregnant or lactating patient
- •Use of an investigational drug or device within 30 days prior to study participation.
- •A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up
- •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
Lysosomal storage disease
次要结局
未报告次要终点
研究者
研究点 (2)
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