Transplantation of Human Allogenic Cord Blood Mononuclear Stem Cells in Autism: Safety and Efficacy of the Method
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Incidence of Treatment Emergent Adverse Events
研究概览
简要总结
Purpose. The goals of the study are to assess the safety of the intravenous infusion of umbilical cord blood (UCB) cells in patients with autism and to confirm changes in social/ communicative skills and cognitive functioning after four infusions of ABO/Rh-matched UCB stem cells.
Material and methods. The sample comprises 30 patients (27 males, 3 females) aged between 3 and 11 years with ASD under the care of the National Medical Research Center for Psychiatry and Neurology (Saint-Petersburg, Russia). Participants are randomly assigned to either the control group (14 males, 1 female) or the experimental group (13 males, 2 females). The experimental group receives intravenous injections of UCB cells four times with a two-week gap between injections. The control group receives standard therapy.
The dynamic of cognitive functions and social/communicative skills assess with Checklist for autism spectrum disorders (CASD), Autism treatment evaluation checklist (ATEC), subscales of Wechsler Intelligence Scale for Children (WISC) - "Digit Span", "Picture completion", "Block design", "Coding".
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
- None
入排标准
- 年龄范围
- 3 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosed ASD by ICD-10 criteria
排除标准
- •acute somatic diseases
- •indication for surgical (neurosurgical) therapy
- •acute or chronic infectious diseases
- •history of acute allergic reactions
结局指标
主要结局
Incidence of Treatment Emergent Adverse Events
时间窗: 24 hours after infusion
Assessed by Adverse Event Grading Scale. It categorized adverse events according to severity. Mild Adverse Event - event results in mild or transient discomfort, not requiring intervention or treatment; does not limit or interfere with daily activities (e.g., insomnia, mild headache). Moderate Adverse Event - event is sufficiently discomforting so as to limit or interfere with daily activities; may require interventional treatment (e.g., fever requiring antipyretic medication). Severe and undesirable Adverse Event - event results in significant symptoms that prevent normal daily activities; may require hospitalization or invasive intervention (e.g., anemia resulting in blood transfusion).
Changes in severity of autistic symptoms from baseline at 3d and 6th month (measured by Checklist for Autism Spectrum Disorders (CASD)
时间窗: at baseline, 3d and 6th month
The CASD rates 30 symptoms of autism, covering six domains: problems with social interaction, perseveration, somatosensory disturbance, atypical communication and development, mood, problems with attention and safety. Symptoms are scored as present or absent based on information drawn from various sources, including teacher, caregiver, parent, observation of the child, and available records. The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to 30 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD.
Changes in severity of autistic symptoms from baseline at 3d and 6th month (measured by Autism Treatment Evaluation Checklist (ATEC)
时间窗: at baseline, 3d and 6th month
The purpose of the ATEC is to measure a change in a patient due to various interventions. It consists of four subtests: Speech/Language Communication (14 items); Sociability (20 items); Sensory/ Cognitive Awareness (18 items); Health/Physical/Behavior (25 items). The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to 179 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD.
次要结局
- "Block design" subscale of Wechsler Intelligence Scale for Children (WISC)(at baseline, 6th month)
- "Digit Span" subscale of Wechsler Intelligence Scale for Children (WISC)(at baseline, 6th month)
- "Coding" subscale of Wechsler Intelligence Scale for Children (WISC)(at baseline, 6th month)
- "Picture completion" subscale of Wechsler Intelligence Scale for Children (WISC)(at baseline, 6th month)