NCT01765283已完成1 期
A Prospective, Open Label, Multicenter, Partially Randomized, Safety Study of One Cycle of Promethera HepaStem in Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) Paediatric Patients.
Cellaion SA21 个研究点 分布在 5 个国家目标入组 20 人开始时间: 2012年3月1日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 21
- 主要终点
- Safety of HepaStem in paediatric patients suffering from CN or UCD
研究概览
简要总结
The purpose of this study is to assess the safety and to appraise the efficacy of one cycle of Hepastem (Heterologous Human Adult Liver-derived Progenitor Cells, HHALPC) infusions in paediatric patients suffering from CN or UCD.
The study duration: 12 months starting from the day of treatment: 6 months active surveillance and 6 months observation post-infusion.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject shows patency of the portal vein and branches, with normal flow velocity in the main portal vein as confirmed by Doppler ultrasound and accessibility of the portal vein, or respectively, accessibility of the umbilical vein.
- •Subject (if capable of signing) and parents or legal representative have provided a written informed assent/consent.
- •Female subjects of childbearing potential need to have a negative pregnancy test and must agree to use an acceptable method of contraception throughout the study.
- •MAIN INCLUSION CRITERIA
- •Crigler-Najjar Syndrome specific:
- •Patient presents with Crigler-Najjar syndrome type
- •Patient presents with Crigler-Najjar syndrome type 2, poorly controlled under phenobarbital treatment, or experiencing serious impairment in quality of life.
- •Diagnosis must be confirmed by genetic mutation analysis if not available.
- •Urea Cycle Disorders specific:
- •Diagnosis of one of the urea cycle disorders of which the disease is of such severity to warrant liver transplantation or alternatives despite full conservative therapy,
- •subject experiencing serious impairment in quality of life despite full conservative therapy.
排除标准
- •The subject is 18 years or older at time of screening.
- •The subject presents acute liver failure, clinical or radiological evidence of liver fibrosis or cirrhosis, presents or has a history of hepatic or extrahepatic malignancy
- •The patient has a non-corrected cardiac malformation, has a known medical or family history of coagulopathy, had or has a renal insufficiency treated by dialysis.
- •The subject requires valproate therapy.
- •The subject has a thrombosis of the portal vein or persisting impairment of anterograde portal blood flow.
- •The subject has a porto systemic shunt or fistula assessed by Doppler US.
- •Patients with disease of such severity that liver transplantation is an absolute indication.
结局指标
主要结局
Safety of HepaStem in paediatric patients suffering from CN or UCD
时间窗: 6 months
Evaluation of the clinical status, portal-vein hemodynamics, morphology of the liver, de novo detection of circulating anti-HLA antibodies, and/or other immune related markers as well as Serious Adverse Events (SAEs) and clinically significant Adverse Events (AEs) related to infusion.
次要结局
- Long-term safety profile of HepaStem in both indications(From 6 to 12 months post-administration)
- Preliminary efficacy of HepaStem in both indications (CN and UCD) and for different weight cohorts(0-6 months, 6-12 months)
研究者
研究点 (21)
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