Transplantation of Umbilical Cord-derived Mesenchymal Stem Cells Via Different Routes for the Treatment of Cerebral Palsy in Children
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 44
- 试验地点
- 1
- 主要终点
- The Gross Motor Function Measure (GMFM)-88 item score
研究概览
简要总结
To preliminarily evaluate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSCs), and compare the efficacy of UC-MSCs administered through the intravenous, intrathecal, and intranasal routes, in the treatment of cerebral palsy in children.
详细描述
Cerebral palsy refers to a neurological disorder caused by a non-progressive brain injury or malformation that occurs in early childhood. It can result in central motor deficits, developmental retardation, abnormal posture, abnormal muscular strength, abnormal muscle tone and/or dysreflexia. It has a high disability rate. There is currently no effective treatment for cerebral palsy. Conventional treatments for cerebral palsy are often tiresome and expensive and have a slow onset of action. Stem cells have been recently used in the treatment of cerebral palsy. This provides a novel method for the treatment of cerebral palsy. According to the existing clinical studies, neural stem cells, bone marrow-derived mesenchymal stem cells, and umbilical cord-derived mesenchymal stem cells (UC-MSCs) are mainly used as the seed cells for the treatment of cerebral palsy. UC-MSCs are the most promising seed cells for the treatment of cerebral palsy because of the advantages including rich sources, ease of harvesting, short doubling time, low immunogenicity, long-time survival post-transplantation, and no ethical issues. UC-MSCs have been widely used to treat Parkinson's disease, rheumatoid arthritis, traumatic brain injury, aplastic anemia, and decompensated liver disease. However,only two studies, and performed only in China, are reported on the treatment of cerebral palsy with UC-MSCs. One from the Hospital 463 of PLA reported 51 patients with cerebral palsy whose symptoms had not been obviously improved after 1 year of rehabilitative treatment. These patients received intrathecal injection of UC-MSCs for three times, once a week, followed by one injection of UC-MSCs via the peripheral vein in the fourth week. Four UC-MSCs injections, once per week, were considered as one course of treatment. Activities of daily living scale score was compared between before and after treatment to evaluate efficacy. Another study is a case report from China. In this report, a combined intravenous and intrathecal injection of UC-MSCs was used to treat cerebral palsy in a 5-year-old child. 28-month follow-up results revealed that the child's gross motor function, immune function, muscle strength, and language ability improved and adverse reactions were not obvious.
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
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 18 Years(Child, Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed with hypoxic/ischemic cerebral palsy, primarily including birth asphyxia and premature children
- •Patients with spastic quadriplegia
- •Patients with moderate to severe cerebral palsy, GMFM scores of 2-3
- •Age at 2-18 years
- •Provision of signed informed consent by legal representatives of the child prior to start of the study
排除标准
- •Those with systemic diseases that likely interfere with the treatment or child's compliance
- •Those complicated by life-threatening diseases of any organ
- •Those with brain deformity
- •Those with uncontrolled epilepsy
- •Those with abnormal behavior or mood disorders
- •Those with allergies especially those who are allergic to blood products
- •Those are infected with infectious diseases
- •Those who had underwent a craniocerebral surgery in other clinical trials
研究组 & 干预措施
Intranasal UC-MSCs group
Injection of UC-MSCs via the nasal route.
干预措施: UC-MSCs (Procedure)
Control group
Routine rehabilitation treatments
干预措施: Control group (Other)
Intravenous UC-MSCs group
Injection of UC-MSCs via the peripheral vein.
干预措施: UC-MSCs (Procedure)
Intrathecal UC-MSCs group
Injection of UC-MSCs via the intrathecal route.
干预措施: UC-MSCs (Procedure)
结局指标
主要结局
The Gross Motor Function Measure (GMFM)-88 item score
时间窗: 12 months after 2 courses of treatment with UC-MSCs.
a 4-point scale that is divided into five categories including lying and rolling, sitting, crawling and kneeling, standing, walking, running, and jumping. A score of 0 indicates that the child does not initiate the task; 1 indicates that the child initiates the task (completes \< 10% of the activity); 2 indicates that the child partially completes the task (completes from 10 to 99% of the activity); 3 indicates that the child completes the task (100%). The sum of score of the five categories is used as the evaluation outcome.It is used to evaluate gross motor function.
次要结局
- The Modified Ashworth scale (MAS) score(1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.)
- Fine Motor Function Measure scale (FMFM) score(1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.)
- Gesell Development Schedule score(1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.)
- Incidence of adverse reactions(1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.)
研究者
Liu Jing
Principal Investigator
The First Affiliated Hospital of Dalian Medical University
