Protocol for Evaluating the Safety and Efficacy of Transfusion of Umbilical Cord Blood in Patients With a Diagnosis of Autism Spectrum Disorder Depending on the Degree of Tissue Compatibility of the Donor and the Recipient
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 150
- 试验地点
- 2
- 主要终点
- Autism Treatment Evaluation Checklist (ATEC).
研究概览
简要总结
Autism is one of those disorders in Autism spectrum disorders (ASD), which characterized by social interaction abnormalities, impaired verbal and non-verbal communication, and repetitive, obsessive behavior, while the therapeutic effect of current treatments remains limited progress.
The possible reason for ASD is neural hypoperfusion and immune dysregulation. The Human Umbilical Cord Blood Mononuclear Cells (hUCB-MNCs) have been shown to have the ability to modulate the immune response and enhance angiogenesis, suggesting the novel and promising therapeutic strategy. In this study, the safety and efficacy of hUCB-MNCs infusion will be evaluated in patients with Autism with regarding to HLA compatibility.
详细描述
Autism Spectrum Disorders (ASD) are heterogeneous neuro-developed disorders. Autism is the most common disorder of the diseases characterized by dysfunctions in response to social interaction and communication, as well as the presence of repetitive and stereotyped behaviors. Recent reports of a sharp increase in the number of children with autism. The exact etiology of autism remains unclear. Compliance, the definition of effective treatments for autism is particularly difficult.
Although it has been understood, it can be a question of immune dysregulation. Examination of the inflammatory cytokines, dysfunction of the immune system and the immune system. The Human Umbilical Cord Blood Mononuclear Cells (hUCB-MNCs) have been shown to have the ability to modulate the immune response and enhance angiogenesis, suggesting the novel and promising therapeutic strategy. Our study suggest that infusion of cord blood mononuclear cells will affect Autism.
This protocol was developed on the basis of the results of the previously approved protocol of the center NCT03786744 (Transfusion of allogeneic cord blood samples in patients with autism spectrum disorders), which showed high efficiency in the rehabilitation of patients. The present protocol is intended for revealing the dependence of the clinical effect on the degree of tissue compatibility of umbilical cord blood samples and the recipient.
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
- 3 Years 至 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient selection criteria (indications for this type of treatment):
- •Patient's age from 4 to 14 years;
- •Diagnosis: autistic spectrum disorder;
- •The severity of the disease on the ATEC scale of at least 16 points;
- •The presence of a compatible allogeneic sample suitable for infusion;
- •Parental consent (official guardians).
排除标准
- •(contraindications for this type of treatment):
- •The patient's age under 4 years, after 14 years;
- •The presence of the following diseases in the history: heart failure at the stage of decompensation, stroke in the history of less than 1 year ago, anemia and other blood diseases;
- •Decompensation for chronic and endocrinological diseases;
- •Acute viral and bacterial infections during the acute clinical phase of the disease;
- •HIV infection, hepatitis B and C;
- •Cancer, chemotherapy, and history of cancer;
- •Tuberculosis;
- •Severe form of intellectual disability as a concomitant disease (diagnosis can be ignored, according to the decision of the Medical Committee of the Center);
- •Fragile X chromosome syndrome;
- •Epileptic seizures with or without medication in the last 6 months before inclusion in the protocol.
结局指标
主要结局
Autism Treatment Evaluation Checklist (ATEC).
时间窗: baseline, change from baseline every month (total 12 evaluations)
Will be assessed: I. Speech/Language Communication (14 items); II. Sociability (20 items); III. Sensory/ Cognitive Awareness (18 items); and IV. Health/Physical/Behavior (25 items).
Number of participants with non-serious and serious adverse events.
时间窗: baseline, change from baseline at 12 month
Safety assessment. Adverse events rate will be assessed in all patients.
The Childhood Autism Rating Scale (CARS2).
时间窗: baseline, change from baseline at 6,12 month
Rating for behavior, characteristics, and abilities against the expected developmental growth. Will be assessed fifteen items, including: relationship to people; imitation; emotional response; body; object use; adaptation to change; visual response; listening response; taste-smell-touch response and use; fear and nervousness; verbal communication; non-verbal communication; activity level; level and consistency of intellectual response; general impressions. Total score will be assessed. Scores range form 15 to 60 with 30 being the cutoff rate for a diagnosis of mild autism. Scores 30-37 indicate mild to moderate autism, while scores between 38 and 60 are characterized as severe autism.
次要结局
- Evaluation of the survival of donor cells in the host body without immune responses(baseline, change from baseline at 6 month)
- The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the sample of umbilical cord blood and the treatment effectiveness degree(At 1, at 6 months (At first and second infusion))
- The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the patient depending on the severity of the disease.(At 1, at 6 months (At first and second infusion))
- The correlation of the concentration of CD34+ cells in the cord blood sample and the treatment effectiveness degree(At 1, at 6 months (At first and second infusion))
研究者
Volchkov Stanislav
Deputy director, Quality assurance director
State-Financed Health Facility "Samara Regional Medical Center Dinasty"