Phase II, Randomized, Double-blind Clinical Trial for Efficacy of G-CSF and Autologous Cord Blood Infusion in Children With Cerebral Palsy
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 88
- 试验地点
- 1
- 主要终点
- Confirm the safety of autologous cord blood infusion and repeated injection of G-CSF in children with cerebral palsy by repeated follow-up every 3 months over 18 months with clinical and laboratory evaluations
研究概览
简要总结
This study is performed to reveal the safety and feasibility of combination therapy with autologous cord blood mononuclear cells (CB) and G-CSF as well as repeated administration of G-CSF for children with cerebral palsy. The evaluation tools are as follows: (1) Developmental tests (DDST, PEDI, GMFM, GMFCS, MACS, QUEST), (2) Neurocognitive function test (WPPSI-IV), (3) Brain MRI-DTI, (4) Peripheral blood CD34+ cell counts, (5) Neurotrophic factors/anti-inflammatory cytokines.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ages from 2 years to 10 years at the time of enrollment
- •Non-severe type of cerebral palsy
- •Willing to comply with all study procedures
排除标准
- •Previous participation within 1 year in a clinical study with stem cell therapy including cord blood, G-CSF, and erythropoietin
- •Presence of chromosomal abnormalities
- •Unwillingness to participate clinical trial
- •Presence of hypersensitivity reaction to G-CSF
- •Evidence of hepatic, renal, cardiac dysfunctions
研究组 & 干预措施
CB + G-CSF
干预措施: G-CSF (Leucostim) (Drug)
CB + G-CSF
干预措施: CB (autologous cord blood) (Biological)
CB + placebo
干预措施: CB (autologous cord blood) (Biological)
CB + placebo
干预措施: Placebo (Normal saline) (Drug)
G-CSF
干预措施: G-CSF (Leucostim) (Drug)
Placebo
干预措施: Placebo (Normal saline) (Drug)
结局指标
主要结局
Confirm the safety of autologous cord blood infusion and repeated injection of G-CSF in children with cerebral palsy by repeated follow-up every 3 months over 18 months with clinical and laboratory evaluations
时间窗: For 18 months from date of randomization
次要结局
- Confirm the efficacy of autologous cord blood infusion and/or G-CSF in children with cerebral palsy using standardized Gross Motor Function Measure evaluation.(Every 6 months from date of randomization up to 18 months)
研究者
Young-Ho Lee
Professor, Department of Pediatrics
Hanyang University Seoul Hospital