Efficiency Evaluation of Allogenic Umbilical Cord Blood Mononuclear Cells (UCB-MNC) Transfusion in Patients With Autism
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 2
- 主要终点
- Changes in Standardized Gross Motor Function 66 (GMFM-66) Score for all child.
研究概览
简要总结
Cerebral palsy is a disorder of movement and posture resulted from a non-progressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability.
Many experimental animal studies have revealed that umbilical cord blood is useful to repair neurological injury in brain.
On the basis of many experimental studies, umbilical cord blood is suggested as a potential therapy for cerebral palsy.
详细描述
Cerebral palsy (CP) is a group of neurodevelopmental conditions with abnormal movement and posture resulted from a non-progressive cerebral disturbance. It is the most common cause of motor disability in childhood. Most therapies are palliative rather than restorative. Umbilical cord blood (UCB) may be used as restorative approach for children with CP.
Many experimental animal studies have revealed that UCB is beneficial to improve and repair neurological injuries, this effect achieved due to immune regulation and angiogenesis as well as the neuroprotective effect.
Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for children with CP.
This study is prospective, non randomized (open label) with control group.
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
- None
入排标准
- 年龄范围
- 1 Year 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis: Cerebral Palsy.
排除标准
- •the presence of the following diseases in history: heart failure in the stage of decompensation, stroke in history less than 1 year ago, blood diseases;
- •decompensation of chronic and endocrinological diseases;
- •acute respiratory viral and bacterial infections, period less than 1 month after the acute phase.
- •HIV infection, hepatitis B and C.
- •oncological diseases, chemotherapy in the anamnesis;
- •tuberculosis.
结局指标
主要结局
Changes in Standardized Gross Motor Function 66 (GMFM-66) Score for all child.
时间窗: 1, 3, 6 month
GMFM (Gross Motor Function Measure) as a standardized measurement tool for assessing Gross Motor Function consisting of sub-scales; lying \& rolling, sitting, crawling \& kneeling, standing, walking, running \& jumping (range: 0\~100 , Higher value means better gross motor function). We reported changes of GMFM between each assessment time points. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones. This test will be acquired for all child.
Changes in The Infant Toddler Quality of Life Questionnaire for child above 3yrs.
时间窗: 1, 3, 6 month
ITQOL - the 47-item short-form (ITQOL-SF47) developed for use in infants and toddlers from 2-months-to-5 years of age. Form scores physical, mental and social well being/ For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). Changes in completed questionnaire will be assessed. This test will be acquired for child above 3yrs.
Changes in Cognitive Neurodevelopmental Outcome for child before 3yrs
时间窗: 1, 3, 6 month
Bayley Scale of Infant Development-II (K-BSID-II) Mental Scales (higher value means better mental function: 0 - worst, 178 - best). We reported changes of BSID-II Mental Scale raw score between each assessment time points. Categories of outcome data are values of subtracting the latter scores from the former ones.This test will be acquired for child before 3yrs.
Number of participants with non-serious and serious adverse events
时间窗: 6 month
Safety assessment such as adverse events will be registered. Adverse events will be monitored during all trial.
Changes in Ashworth scale score for all child.
时间窗: 1, 3, 6 month
The Ashworth scale (AS) measures resistance during passive soft-tissue stretching and is used as a simple measure of spasticity scoring, where: 0 No increase in tone; 1. Slight increase in tone giving catch when the limb is moved in flexion and extension; 2. More marked increase in tone, but limb is easily flexed; 3. Considerable increases in tone, passive movement difficult; 4. Limb rigid in flexion or extension. This test will be acquired for child above 3yrs.
次要结局
- Number of Participants with Chimerism (longevity) of infused cell(6 month)
研究者
Volchkov Stanislav
Deputy director, Quality assurance director
State-Financed Health Facility "Samara Regional Medical Center Dinasty"