Safety Study of Sibling Cord Blood Cell Infusion to Children With Cerebral Palsy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 4
- 主要终点
- Number of participants with abnormal clinical assessment and/or laboratory values
研究概览
简要总结
This study will investigate the safety of single dose intravenous infusion of cord blood cells which were cryopreserved after the birth of a brother or sister to a child with cerebral palsy.
详细描述
Cerebral palsy (CP) is the most common physical disability of childhood, affecting 2 per 1000 live births across the world. CP describes permanent non-progressive motor disorders arising from damage to the developing brain.
Preclinical studies of different types of stem cells in models of acute brain injury similar to CP have shown significant functional improvement. The variety of stem cells available in umbilical cord blood (UCB), an ethically uncomplicated source of stem cells, has led to a focus on UCB stem cell therapy as a quick-to-clinic option. Previous studies indicate that autologous or unrelated donor UCBC infusion is safe and feasible for children with CP, and may lead to improved motor functioning, but there is no information about the safety and effects of matched sibling cord blood. Therefore, this trial will study the safety of infusing matched sibling cord blood cells to children with cerebral palsy.
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
- 1 Year 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of any type of CP
- •CP of any severity
- •A record of sibling CBU in storage at a TGA accredited private cord blood bank
- •Ability to travel to one of the trial centres
- •Ability to participate in assessments
- •Informed consent by parent/guardian
排除标准
- •presence of progressive neurological disease
- •known genetic disorder
- •known brain dysplasia
- •immune system disorder or immune deficiency syndrome
- •infectious disease markers showing up on virology screen
- •evidence of cord blood unit contamination, or fewer than 10^7 cells/kg body mass
- •ventilator support
- •ill health, or if the participant's medical condition does not allow safe travel
- •previous cell therapy
- •Botulinum toxin A within 3 months before or after infusion
- •surgery within 3 months before or after infusion
- •cannot obtain parent/guardian consent
结局指标
主要结局
Number of participants with abnormal clinical assessment and/or laboratory values
时间窗: 12 months
Safety
次要结局
- Digital PCR analysis of peripheral blood cellular DNA to determine the fraction of donor DNA in circulation(3 months)
- Preliminary analysis of change in gross motor function(Baseline, 12 months)
- Preliminary analysis of change in quality of life(Baseline,12 months)
- Preliminary analysis of change in cognitive function(Baseline, 12 months)
- Preliminary analysis of change in fine motor function(Baseline, 12 months)