NCT02405598已完成4 期
Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- 10% decrement of creatinine kinase
研究概览
简要总结
evaluate if beta 2-adrenergic agonist can have adjuvant effect to patients with infantile-onset Pompe disease under enzyme replacement therapy
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirm diagnosis as Pompe disease
- •age 2 years or above
- •under at least 1 year regular recombinant human acid alpha glucosidase, and no dosage/frequency change in recent 3 months
- •Provide inform consent
排除标准
- •chronic heart disease such as arrhythmia, cardiomyopathy, cardiac infarction
- •history of seizure
- •history of diabetes mellitus
- •history of hyperthyroidism
- •hypokalemia
- •allergy to β-agonists
- •under medications including diuretics, digoxin, beta-blockers, etc
研究组 & 干预措施
Salbutamol
Experimental
- age 2-6 year: 0.1mg/kg tid x 2 weeks, then gradually increase to 0.2mg/kg tid (daily total maximal12mg)
- age 6-12 year: 2mg tid x 2 weeks, then gradually increase to 4mg tid (daily total maximal 24mg)
- age 12 year and above: 4mg tidx 2 weeks, then gradually increase to 8mg tid (daily total maximal 32mg)
干预措施: Salbutamol (Drug)
结局指标
主要结局
10% decrement of creatinine kinase
时间窗: 6 months
measure and compare serum creatinine kinase levels
Improvement in 6-minutes walk test
时间窗: 6 months
measure and compare 6-minutes walk test
次要结局
未报告次要终点
研究者
Hsiao Sang Chu, MD
MD, PhD
National Taiwan University Hospital
研究点 (1)
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