Pilot Study to Assess the Efficacy of Autologous Umbilical Cord Blood Treatment of Brain and Heart Injury in Neonates With Congenital Heart Defect
试验速览
- 阶段
- 1 期
- 入组人数
- 60
- 试验地点
- 2
- 主要终点
- Neuroimaging of the brain before and after the surgery (ref 1)
研究概览
简要总结
The aim of the study is to evaluate the feasibility and safety and efficacy of collecting and infusing autologous umbilical cord blood (UCB) in newborn infants with hypoplastic left heart syndrome (HLHS) and transposition of great arteries (TGA).
Rationale: Neonates with HLHS and TGA have significant brain injury as demonstrated by peri-surgical MRI. Moreover, there a substantial tendency to suffer from chronic cardiac condition as low cardiac output state and valvular insufficiency. Treatment of neonates after hypoxic ischemic injury at birth with autologous UCB was shown to safe and improved developmental outcome. The effect of UCB is most likely achieved by reduction of free radicals injury and pro-inflammatory and apoptotic process.
Hypothesis: Treatment with UCB immediately after the first cardiac surgey, with in the first week life will reduce the brain injury demonstrated by MRI and reduce the choronic cardiac problems
详细描述
The aim of this pilot study is to evaluate the safety and feasibility of infusion of autologous umbilical cord blood (UCB) in neonates with with hypoplastic left heart syndrome (HLHS) or transposition of great arteries (TGA).
This is a prospective, matched control study, Phase I-II trial to evaluate the safety and efficacy of autologous UCB infusion in neonates with HLHS or TGA. The study group will consist of neonate that were diagnosed by prenatal fetal ultrasound with HLHS or TGA and their UCB was collected at the time of delivery.
The study group will include the patients with UCB and the control group will be patients without UCB.
All patients will have their surgery within 2 weeks from birth. The UCB will be infused to the patients in study group as soon as the patient has stabilized after the surgery and not more than 7 days after surgery.
Both groups will be followed similarity:
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
盲法说明
The outcome assessor will not be exposed to the participant group
入排标准
- 年龄范围
- 3 Days 至 14 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At least 35 weeks of gestation
- •Prenatal diagnosis of HLHS including mitral stenosis/atresia, aortic stenosis/atresia, arch hypoplasia and the presence of hypoplastic left ventricle with or without VSD.
- •Norwood procedure will take place within 14 days from birth.
- •Treatment with cord blood should be given within 7 days after surgery.
- •Parental informed consent for collection of umbilical cord blood.
排除标准
- •Total nucleated cells (TNC) lower than 1X107 in the collected umbilical cord blood unit.
- •Infected umbilical cord blood unit.
- •Parents refusal to continue in the study at any stage.
结局指标
主要结局
Neuroimaging of the brain before and after the surgery (ref 1)
时间窗: The time frame between MRI before and after surgery is 10-13 days
Change in MRI of the brain before (between 1-3 days) and after (10 days) the surgery between the intervention and control group. The change will be quantified by MRI brain injury severity (BIS) score quantifying white matter injury, stroke and brain hemorrhage. Diffusion tensor imaging measures will also be quantified by using average diffusivity and fractional anisotropy calculated from frontal, parietal, temporal and occipital white matter.
次要结局
- Neuro-development at one month(One month)
- Neuro-development at 12 month(12months)
- Neuro-development at six month(6 months)
研究者
Dr. Omer Bar-Yosef
Principal Investigator
Sheba Medical Center