EUCTR2013-002701-56-NL进行中（未招募）1 期
A Phase 2, multicenter, multinational, open-label, dose-escalation study to evaluate the safety and efficacy of ORGN001 (formerly ALXN1101) in pediatric patients with Molybdenum Cofactor Deficiency (MoCD) Type A currently treated with recombinant Escherichia coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 8
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
- •- Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
- •- Currently treated with rcPMP infusions
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 8
- •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
- •- Current or planned treatment with another investigational drug or device, with the exception of rcPMP treatment through Day -1
研究者
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