JPRN-jRCT2061210056招募中未知
An Expanded Access Study for Cipaglucosidase Alfa in Conjunction with Miglustat for the Treatment of Adult Subjects with Late-Onset Pompe Disease in Japan
适应症
试验速览
- 阶段
- 未知
- 状态
- 招募中
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 18age old 至 ot applicable（—）
- 性别
- All
入选标准
- •1. Male or female, 18 years old and above
- •2. A diagnosis of LOPD based on documented deficiency of acid alpha-glucosidase (GAA) enzyme activity or GAA genotyping
- •3. Female subjects of childbearing potential and male subjects if they are using appropriate, reliable contraception
排除标准
- •1. Subject has a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat
- •2. Subject has a medical condition or any other extenuating circumstance that may pose an undue
- •safety risk to the patient
- •3. Subject, if female, is pregnant or breastfeeding
- •4. Subject, whether male or female, is planning to conceive a child during the study
- •5. Subject has received treatment with prohibited medications within 30 days of screening
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