NCT00144768已完成4 期
A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients With Mucopolysaccharidosis I (MPS I) Being Treated With Aldurazyme® (Laronidase).
Genzyme, a Sanofi Company6 个研究点 分布在 1 个国家目标入组 25 人开始时间: 2004年7月1日最近更新:
适应症
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 25
- 试验地点
- 6
- 主要终点
- Immunogenicity Testing
研究概览
简要总结
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have a documented diagnosis of MPS I confirmed by measurable signs and symptoms and deficient α-L-iduronidase activity (<10% of the lower limit of normal).
- •For a patients receiving Aldurazyme therapy prior to study entry: Have available as baseline data the results of urinary GAG levels and IgG antibody titers collected prior to the patient's first Aldurazyme infusion.
- •Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years.
排除标准
- •Have previously received Aldurazyme without the collection of baseline samples as specified.
- •Have a suspected hypersensitivity to Aldurazyme or a know hypersensitivity to components of infusion solution.
- •Have received a Hematopoietic Stem Cell Transplantation, injection fibroblast therapy, or major organ transplant.
- •Are receiving chronic immunosuppressant therapy.
- •Have a medical condition, serious intercurrent illness, or other extenuating circumstances that may interfere with study compliance including all prescribed evaluations and follow-up activities.
- •Are pregnant or lactating
- •Have received investigational drug within 30 days prior to study enrollment
结局指标
主要结局
Immunogenicity Testing
时间窗: Up to 4 years
Urinary GAG (glycosaminoglycans)
时间窗: Up to 4 years
次要结局
- Safety(Up to 4 years)
研究者
研究点 (6)
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