NCT02489292已完成2 期
Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients
Cellaion SA11 个研究点 分布在 4 个国家目标入组 5 人开始时间: 2014年10月1日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 5
- 试验地点
- 11
- 主要终点
- Efficacy as determined by de novo ureagenesis (C13 tracer method)
研究概览
简要总结
The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.
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 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Main Inclusion Criteria:
- •Paediatric patients < 12 years prior to infusion
- •Patient presents with UCD
- •Patient shows patency of the portal vein and branches, with normal flow velocity as confirmed by Doppler US and accessibility of the portal vein and /or affluants.
排除标准
- •Patient has mild disease severity, easily controlled under standard of care therapy, with no recurrent metabolic crises.
- •Patient is registered on a liver transplant waiting list or is scheduled for living donor liver transplantation before the end of the study.
- •Patient presents acute liver failure.
- •Patient presents clinical or radiological evidence of liver cirrhosis.
- •Patient presents or has a history of hepatic or extrahepatic malignancy.
- •Patient has a known clinically significant cardiac malformation.
- •Patient has a personal history of venous thrombosis, or has a clinically significant abnormal value for protein S, protein C, anti-thrombin III, and /or activated Protein C Resistance (aPCR) at screening. In case of known family history, a complete coagulation work-up should be performed. In all above described cases, results need to be discussed with PB before enrolling the patient in the study.
- •Patient had or has a renal insufficiency treated by dialysis.
结局指标
主要结局
Efficacy as determined by de novo ureagenesis (C13 tracer method)
时间窗: at 6m post-first infusion day
次要结局
- Efficacy as determined by de novo ureagenesis (C13 tracer method)(at 3, 9 and 12 months post-first infusion day)
- Efficacy as determined by Ammonia (NH3) values(up to 12 months post-first infusion day)
- Efficacy as determined by amino acids in plasma(up to 12 months post-first infusion day)
- Efficacy as determined by report of metabolic decompensations(up to 12 months post-first infusion day)
- Efficacy as determined by report on actual supportive treatment, adjustment of protein restriction and amino acids supplements(up to 12 months post-first infusion day)
- Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators(up to 12 months post-first infusion day)
- To evaluate the safety during the year following HepaStem infusions (composite)(up to 12 months post-first infusion day)
研究者
研究点 (11)
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