NCT02104336终止2 期
An Open-Label Phase 2 Safety and Efficacy Study of EPI-743 (VincerinoneTM) in Children With Pearson Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 4
- 主要终点
- Occurence of episodes of sepsis, metabolic crisis or hepatic faillure
研究概览
简要总结
Treatment of Pediatric Subjects with Pearson syndrome
详细描述
If effective, treatment with EPI-743 should result in a significant reduction in occurrence of sepsis, metabolic crisis, hepatic failure and transfusion dependence.
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
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed diagnosis of Pearson syndrome
- •Age less than 18
- •Availability of medical history for 12 months prior to enrollment
- •Abstention from use of CoQ10, vitamin E, lipoic acid and Idebenone 14 days prior to treatment with EPI-743
排除标准
- •Allergy to EPI-743, sesame oil or vitamin E
- •Clinical history of bleeding/ abnormal PT/PTT
- •Concurrent inborn errors of metabolism
- •Use of anticoagulant medications
- •Participation in any interventional study within 30 days of treatment
- •Use of erythropoietin 30 days prior to trial enrollment
研究组 & 干预措施
EPI-743
Experimental
15 mg/kg EPI-743 to be administered three times per day for 1 year
干预措施: EPI-743 (Drug)
结局指标
主要结局
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure
时间窗: 1 year
次要结局
- Neuromuscular function(1 year)
- Hospitalizations(1 year)
- Hematologic function(1 year)
- Disease severity(1 year)
- Mortality(1 year)
- Hepatic function(1 year)
- Transfusion avoidance(1 year)
- Renal function(1 year)
- Weight gain(1 year)
- Pancreatic function(1 year)
研究者
研究点 (4)
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