NCT02047461已完成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)
Origin Biosciences6 个研究点 分布在 5 个国家目标入组 8 人开始时间: 2014年4月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 8
- 试验地点
- 6
- 主要终点
- Safety of ORGN001 (Formerly ALXN1101)
研究概览
简要总结
This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.
详细描述
Patients will receive daily IV infusions of ORGN001 (formerly ALXN1101) starting on Day 1. After a prescribed period, dosing will increase monthly based on defined patient safety measures. After Month 6, patients will continue daily dosing at their last tolerated dose.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
- •Currently treated with rcPMP infusions
排除标准
- •Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.
研究组 & 干预措施
ORGN001 (formerly ALXN1101)
Experimental
daily IV infusions
干预措施: ORGN001 (formerly ALXN1101) (Drug)
结局指标
主要结局
Safety of ORGN001 (Formerly ALXN1101)
时间窗: Baseline to Month 24 for all patients plus additional follow-up up to Month 90
Treatment Emergent Serious Adverse Events
次要结局
- Long-term Safety of ORGN001 (Formerly ALXN1101)(Baseline to Month 24 for all patients plus additional follow up until Month 72)
- Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)(First 6 months at each dose level, where available)
- S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time(Baseline to Month 24 for all patients plus additional follow-up to Month 90)
- Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination(Baseline to Month 24 for all patients plus additional follow-up until Month 30)
研究者
研究点 (6)
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