EUCTR2015-000582-31-Outside-EU/EEA进行中（未招募）不适用
An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen
Genzyme Corporation Inc0 个研究点目标入组 14 人开始时间: 2015年4月15日最近更新:
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 14
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •(1) the patient or the patient’s legal guardian(s) must provide written informed
- •consent prior to any study-related procedures being performed; (2) the patient must have a clinical diagnosis of Pompe disease as defined by documented GAA deficiency (deficient endogenous GAA activity) in skin fibroblasts or blood; (3) the patient must have been compliant with the standard dosing regimen of Myozyme (20 mg/kg qow) for a minimum of 6 months prior to study entry; and (4) the patient must have clinical decline or sub-optimal improvement in at least one of the following parameters as compared to their condition prior to beginning Myozyme treatment:
- •Left Ventricular Mass (LVM) Z-score = 6 or LVM Index = 150 g/m2 after a minimum of 6 months of treatment with Myozyme, OR
- •Respiratory
- •New development of respiratory failure requiring the use of ventilatory assistance (invasive or noninvasive) after a minimum of 6 months of treatment with Myozyme. Ventilatory assistance must have been required for at least 4 weeks prior to study enrollment, OR
- •Motor Skills
- •For patients < or equal to 2 years of age at study entry, failure to acquire at least 2 new gross motor milestones after a minimum of 6 months of treatment with Myozyme, e.g.:
- •1. Turning head side to side (supine)
- •2. Grasping small objects with hands
- •3. Transferring objects from hand to hand
- •4. Holding head upright with body supported
- •5. Rolling (supine to prone or prone to supine)
- •6. Sitting (supported or unsupported)
- •7. Walking (with support, i.e., cruising, or independently)
- •8. Walking up stairs (with assistance or independently)
- •For patients > 2 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through longitudinal assessments of manual muscle testing after a minimum of 6 months of treatment with Myozyme, OR
- •For patients > 2 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through loss of functional use of the upper extremities after a minimum of 6 months of treatment with Myozyme, OR
- •Progression to use of an assistive device for ambulation due to worsening of proximal lower extremity muscle weakness after a minimum of 6 months of treatment with Myozyme.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 13
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients will be excluded from this study if they meet any of the following exclusion criteria (1) any medical condition which, in the opinion of the Investigator, could interfere with treatment or evaluation of safety and/or efficacy of Myozyme; (2) the patient has major congenital abnormality; (3) the patient has used any investigational product (other than alglucosidase alfa in those regions where the product is not commercially available) within 30 days prior to study enrollment; or (4) the patient is pregnant or lactating.
研究者
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