NL-OMON55625招募中2 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) - ALXN1101-MCD-201
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 2
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
- 0 至 17（—）
入选标准
- •1) Male of female patients with genetically confirmed diagnosis of MoCD Type A
- •(MOCS1 mutation) and who are currently treated with rcPMP infusions.
- •2) Parent or legal guardian must have signed the informed consent form (ICF)
- •prior to any study procedures.
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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