EUCTR2017-002806-10-DE进行中（未招募）1 期
An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 9
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
- •MPS IIIA patients, as confirmed by both a documented deficiency in sulfamidase enzyme activity in concordance with a diagnosis of MPS IIIA and normal enzyme activity level of at least one other sulfatase measured in leukocytes, that are =12 to =72 months of age and that have a developmental age =12 months.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 9
- •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 with at least one S298P mutation in the SGSH gene are excluded, as well as patients that have received prior stem cell or gene therapy, or enzyme replacement therapy for MPS IIIA.
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