Safety and Efficacy of Transplantation of Specific Populations of Stem Cells and Mesenchymal Stem Cells for the Treatment of Cerebral Palsy
试验速览
- 阶段
- 1 期
- 入组人数
- 50
- 主要终点
- Changes in Motor Performance.
研究概览
简要总结
This study is a single arm, single center trial to study the safety and efficacy of purified autologous bone marrow derived stem cells and mesenchymal stem cells for the treatment of children with cerebral palsy.
详细描述
Cerebral palsy (CP) is a chronic childhood disorder defined by a range of motor and cognitive impairments and results in a substantial suffering to the patient and a tremendous socio-economic burden to the individual, family, and healthcare system. With no effective treatments or interventions, therapies for CP are currently focused on supportive and management strategies. Stem cell transplantation has been suggested as a putative intervention for neural pathology, as mesenchymal and neural stem cells, as well as olfactory ensheathing glia and Schwann cells, which have shown some regenerative and functional efficacy in experimental central nervous system disorders.
In this trial, the investigators study the safety and efficacy of the intrathecal transplantation of specific populations of purified autologous bone marrow-derived stem cells and mesenchymal stem cells in patients with cerebral palsy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must be more than 2 year of age and less than 12 years of age at the time of screening for inclusion in the study.
- •Clinical evidence of a non-progressive motor disability due to brain dysfunction.
- •Willing to comply with all study procedures.
- •Gross Motor Function Classification Score level II-V
排除标准
- •Known history of:
- •Intractable seizures
- •Traumatic brain injury
- •Genetic disorder
- •Current Infection
- •Renal insufficiency
- •Hepatic disease
- •HIV+ (as demonstrated by positive blood test)
- •Immunosuppression
- •Infectious related neurological injury
- •Other etiologies such as degenerative, mitochondrial, and metabolic disorders
- •Normal brain MRI
- •Evidence of acute illness such as fever (temperature > 37.5 C), vomiting, diarrhea, wheezing or crackles
- •Progressing neurological disease (Batten Disease, Leukodystrophies, Neurotransmitter disorders)
- •Microcephaly, macrocephaly, cortical malformations, or genetic disorders of dysgenesis
- •Pulmonary disease requiring ventilator support
- •Unwillingness to return for follow-up visits
- •Contraindications to MRI
结局指标
主要结局
Changes in Motor Performance.
时间窗: 6 months
GMPM (Gross Motor Performance Measure) as a standard measurement tool for assessing quality of movement.
次要结局
- Motor function studies(1 year)
- Specific white matter tract analysis using MRI(6 months)