Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 36
- 主要终点
- Forced vital capacity
研究概览
简要总结
This study is aimed to investigate that whether the higher dose ERT improve safety and clinical outcomes of Pompe disease patients. Also, wish to develop a new therapeutic recommendation and hope that it could improve the long-term outcomes of Pompe diesease patients.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 0 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Pompe disease
- •Currently receiving ERT with regular clinic visits
- •Agree to sign informed consent
排除标准
- •Lost follow-up for clinical visits
- •Allergy to Myozyme
- •Refuse to sign informed consent
结局指标
主要结局
Forced vital capacity
时间窗: For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Pulmonary function test (Units: percentage of predictive value)
Peak expiratory flow
时间窗: For patient older than 3-year-old, first test before study, then every six months, up to 2-years.
Pulmonary function test (Units: percentage of predictive value)
Polysomnography
时间窗: For patient older than 6-month-old, first test before study, then every six months, up to 2-years.
Comprehensive test used to diagnose sleep disorders.
次要结局
- CK(CK will be tested before the treatment, then every three months, up to 2-years.)
- Body height(The body height will be monitored before the treatment, then every two weeks, up to 2-years.)
- ALT(ALT will be tested before the treatment, then every three months, up to 2-years.)
- uGLC4(uGLC4 will be tested before the treatment, then every three months, up to 2-years.)
- Body weight(The body weight will be monitored before the treatment, then every two weeks, up to 2-years.)
- AST(AST will be tested before the treatment, then every three months, up to 2-years.)
- Antibody titers(First test will be one month later after first ERT, then every six months, up to 2-years.)