A Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Years Old
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 4
- 主要终点
- Safety Evaluation
研究概览
简要总结
The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme® (laronidase)] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.
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
- — 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent is required from the parent(s) or legal guardian(s) prior to any protocol-related procedures being performed. (A separate informed consent will be requested from the parent(s) for their genotyping, which is independent of the inclusion.)
- •Be less than 5 years of age at the time of enrollment.
- •Have confirmed iduronidase deficiency with a fibroblast or leukocyte alpha-L-iduronidase enzyme activity level of less than 10.0 % of the lower limit of the normal range, or below the detection range of the measuring laboratory.
- •Have a clinical diagnosis of MPS I based on genotyping.
- •Documentation in his/her medical record that the parent(s) or legal guardian(s) have had counseling or a consultation regarding HSCT in order to assure that the parent(s) or legal guardian(s) are fully informed regarding the risks and benefits of this alternative treatment for patients eligible for the trial and with the severe manifestations of MPS I with neurodegeneration.
排除标准
- •The patient is under consideration for or has undergone hematopoietic stem cell transplantation (HSCT).
- •The patient has acute hydrocephalus at the time of enrollment.
- •The patient has a clinically significant organic disease (with the exception of symptoms relating to MPS I) including: cardiovascular, hepatic, pulmonary, neurologic, or renal disease, other serious intercurrent illness, or extenuating circumstances that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival.
- •The patient has received any investigational product within 30 days prior to trial enrollment.
- •The patient has known severe hypersensitivity to Aldurazyme® (laronidase) or components of the delivery solution.
结局指标
主要结局
Safety Evaluation
时间窗: 52 weeks
Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
Pharmacokinetics - Area Under the (Plasma Concentration-time) Curve (AUC∞)
时间窗: 52 weeks
AUC∞ is a measure of the total exposure to a drug.
Pharmacokinetics - Elimination Half Life (t1/2)
时间窗: 52 weeks
Half-life is the time it takes for the concentration of drug in plasma to decline by 50%.
Pharmacokinetics - Total Plasma Clearance (CL)
时间窗: 52 weeks
CL is volume of the body fluid cleared of the drug per unit of time.
Pharmacokinetics - Volume of Distribution (Vz)
时间窗: 52 weeks
Vz is the volume that relates the amount of drug in the body after absorption is complete to the concentration of drug in the plasma.
次要结局
未报告次要终点