Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- The Change of Gross Motor Function Classification System(GMFCS) Score
研究概览
简要总结
To study the clinical efficacy and safety of autologous umbilical cord blood transfusion in the treatment of cerebral palsy.
详细描述
This is a Phase 1 clinical trial that constitutes two time points cohorts with participants who will receive intravenous autologous umbilical cord blood and 0.9% sodium chloride respectively. The timing of treatment is any time after the diagnosis of cerebral palsy. The investigator will proceed the groups during the same period.
- Demographic Data and Baseline Characteristics of the Studied Group were collected:
- Basic patient's information survey
- Medical history
- Physical examination
- Basic blood test result
- Children's developmental disorders evaluation before the treatment
- Brain Magnetic Resonance Imaging-Diffusion Tensor Imaging (MRI-DTI) before the treatment
- Neurocognitive function test before the treatment
- Assessment of clinical condition in the course by measurement of blood pressure, heart and respiratory rates, temperature and adverse events was recorded.
- Autologous cord blood doses is 20-30ml (total Mononuclear cells>1*10^7/kg)，the infusion speed is 1ml/min, and with same volume of 0.9% sodium chloride as placebo.
- The follow-up: clinical test until 30th month in 3 month gaps.
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)
入排标准
- 年龄范围
- 6 Months 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A patient who was diagnosed with a mild case, and severity case of cerebral palsy or developmental disability through developmental assessment from more than two specialists including a specialist in pediatrics and rehabilitation medicine..
- •In case there is a sign of the general decrease in white matter, or periventricular leukomalacia near cerebral ventricles in MRI DTI test.
- •A subject who did a written consent to participation in this clinical trial through the very person and a legal representative.
排除标准
- •In case a patient underwent a surgical procedure, or was administered autologous cord blood within one year before participating in a clinical trial.
- •Accompanied by a serious disease, such as chromosome abnormality, etc.
- •In case where a patient's medical condition is judged to be maladapted by a researcher.
- •In case a patient or his or her legal representative doesn't agree to participation in a clinical trial.
- •A patient having a predisposition to allergies.
- •A patient having serious disorders in the liver, kidney, and cardiopulmonary function.
研究组 & 干预措施
Placebo
0.9% sodium chloride infusion any time after entering the Placebo Group, doses is 20-30ml. The infusion speed is 1ml/min.
干预措施: 0.9% Sodium Chloride (Drug)
Cord Blood Mononuclear Cells(CBMNC)
Autologous Umbilical Cord Blood Mononuclear Cells Therapy any time after the diagnosis of cerebral palsy, doses is 20-30ml (total Mononuclear cells>1*10^7/kg). The infusion speed is 1ml/min.
干预措施: Autologous Umbilical Cord Blood (Biological)
结局指标
主要结局
The Change of Gross Motor Function Classification System(GMFCS) Score
时间窗: up to 30 months after therapy at a 3-month interval
GMFCS is used to assess the gross motor function of the children who were diagnosised as cerebral palsy.The results of GMFCS are divided into five levels. Grade I represents the best outcome while grade Ⅴ represents the worst.
The Change of Gesell Developmental Scale(GDS) Score
时间窗: every 3-month after therapy until 30 months
The GDS is used to measure the gross motor,fine action,adaptability，language and individual-society function of the children whose age ranges from 1-month to 6-year-old. The score less than 75 for each subscale represent the worse outcome and more than 75 represent the better.
次要结局
未报告次要终点
研究者
Xiao Huimei
Professor
Guangdong Women and Children Hospital