NCT00144781已完成4 期
A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I
Genzyme, a Sanofi Company10 个研究点 分布在 2 个国家目标入组 34 人开始时间: 2004年12月1日最近更新:
适应症
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 34
- 试验地点
- 10
- 主要终点
- Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level
研究概览
简要总结
The main purpose of this study is to evaluate differences in the pharmacodynamic response of 4 Aldurazyme® (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I).
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
- 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 clinical signs and symptoms of MPS I, and a documented fibroblast or leukocyte α-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
- •Weigh at least 12.5 kg.
- •Have serum creatinine and blood urea nitrogen (BUN) values within age appropriate normal ranges.
- •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. If the patient is under 18 years old and can understand the consent, written informed consent will be required from both the patient and the authorized guardian(s).
排除标准
- •Have previously received Aldurazyme® (laronidase).
- •Have a suspected hypersensitivity to Aldurazyme® (laronidase) or known hypersensitivity to components of the infusion solution.
- •Have previously undergone hematopoietic stem cell transplantation (HSCT; i.e., from bone marrow [BMT], peripheral blood, or umbilical cord blood) or other major organ transplantation.
- •Have a medical condition, serious inter-current illness, or other extenuating circumstance that may interfere with study compliance including all prescribed evaluations and follow-up activities, except the 6MWT. (Note: All patients may not be capable of performing the 6MWT due to age and/or maturity level. Exemption from performing the 6MWT must be obtained in writing by the investigator from the sponsor's medical monitor prior to enrollment).
- •Have an acute illness that requires surgical intervention, and/or anticipates surgery during study participation, and/or has had surgery within 30 days prior to study enrollment.
- •Have received an investigational drug within 30 days prior to study enrollment.
- •Is pregnant or lactating. Female patients of childbearing potential must have a negative pregnancy test [urine β-human chronic gonadotropin (hCG)] at entry (prior to the first infusion). Note: All female patients of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.
结局指标
主要结局
Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level
时间窗: Baseline to 26 Weeks
Urinary GAG Level - Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response.
次要结局
- Percent Change From Baseline to Week 26 in Liver Organ Volume(Baseline to 26 Weeks)
- Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)(Baseline to 26 Weeks)
研究者
研究点 (10)
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