NCT00912925已完成3 期
A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I
Genzyme, a Sanofi Company4 个研究点 分布在 3 个国家目标入组 45 人开始时间: 2000年12月1日最近更新:
适应症
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
- 4
- 主要终点
- Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
研究概览
简要总结
This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient had a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and a fibroblast or leukocyte alpha-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
- •Female patients of childbearing potential had a negative pregnancy test (urine-beta-human chorionic gonadotropin (hCG)) at baseline (all female patients of childbearing potential and sexually mature male patients were advised to use a medically accepted method of contraception throughout the study).
- •The patient was capable of standing independently for 6 minutes and walking a minimum of 5 meters within 6 minutes.
- •The patient was capable of performing a reproducible FVC maneuver.
- •The patient had a baseline FVC value that was less than or equal to 80% of the patient's predicted normal FVC value based on polgar predicted values for standing height for children 5 through 7 years of age and the Hankinson predicted values for ages 8 and above.
排除标准
- •The patient had undergone a tracheostomy.
- •The patient had previously undergone a bone marrow transplantation.
- •The patient was pregnant or lactating.
- •The patient has received an investigational drug within 30 days prior to study enrollment.
- •The patient had a medical condition, serious intercurrent illness, or other extenuating circumstance that could have significantly interfered with study compliance including all prescribing evaluations and follow-up activities.
- •The patient had a known hypersensitivity to rhIDU or to components of the active or placebo test solutions.
结局指标
主要结局
Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
时间窗: Baseline to Week 26
Six Minute Walk Test (6MWT): Distance walked (measured in meters) in 6 minutes. A longer distance indicates a greater response.
Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC)
时间窗: Baseline to Week 26
Percent Predicted Forced Vital Capacity (FVC): the maximal exhaled breathe volume following a maximal inhaled breath. Overall Change from Baseline to Week 26 in percent predicted FVC = (observed value)/(predicted value) \* 100%). A higher value indicates a greater response.
次要结局
- Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI)(Baseline to Week 26)
- Overall Percent Change From Baseline to Week 26 in Liver Volume(Baseline to Week 26)
- Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score(Baseline to week 26)
- Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM)(Baseline to Week 26)
- Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels(Baseline to Week 26)
研究者
研究点 (4)
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