EUCTR2021-005552-11-Outside-EU/EEA进行中（未招募）1 期
A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa.
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
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
- •The patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures;
- •The patient must have a confirmed diagnosis of Pompe disease as determined by deficient endogenous acid alpha-glucosidase (GAA) activity or GAA mutation analysis; and
- •The patient must be <1 year of age at time of study enrollment (and receive alglucosidase alfa treatment before 1 year of age), or the patient must be between 1 year and 24 months of age and must have initiated alglucosidase alfa treatment prior to turning 1 year of age.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
- •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
- •The patient is participating in another clinical study using alglucosidase alfa or any investigational therapy.
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