A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 45
- 试验地点
- 50
- 主要终点
- Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)
研究概览
简要总结
This study is being conducted to collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) patients with MPS I disease. Patients who were previously enrolled in the Phase 3 Double-Blind Study will be enrolled 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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient or patient's legal guardian must provide written informed consent prior to any protocol-related procedures being performed.
- •The patient must have successfully completed Study ALID-003-99 (who received 21 of 26 consecutive weekly infusions).
- •The patient has not experienced any safety issues that would contraindicate participation in the Extension study.
- •A female patient of childbearing potential must have a negative pregnancy test at entry
排除标准
- •The patient is pregnant or lactating.
- •The patient has received an investigational drug within 30 days prior to the study enrollment.
- •The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly interfere with study compliance including all prescribed evaluations and follow-up activities
研究组 & 干预措施
Placebo/Aldurazyme
Patients received placebo for 26 weeks in the Double-Blind Study then received 182 weeks of Aldurazyme (0.58 mg/kg every week) in this Extension Study; patients received a total of 182 weeks of Aldurazyme.
干预措施: placebo (Biological)
结局指标
主要结局
Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)
时间窗: Baseline to Week 182
Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.
Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)
时间窗: Baseline to Week 182
Percent Predicted Forced Vital Capacity: the maximal exhaled breath volume following a maximal inhaled breath. Overall change from Baseline to Week 182 in percent predicted FVC = (observed value)/(predicted value) \* 100%). A higher value indicates a greater response.
次要结局
- Change From Baseline to Week 182 in Liver Volume(Baseline to Week 182)
- Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score(Baseline to Week 182)
- Change From Baseline to Week 182 in Active Joint Range of Motion (ROM)(Baseline to Week182)
- Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI)(Baseline to Week 182)