Prospective Clinical Study on Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes for the Treatment of Childhood Autism
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Number of Participants with Treatment-Related Adverse Events
研究概览
简要总结
This clinical study aims to evaluate whether a nasal spray containing exosomes derived from human umbilical cord mesenchymal stem cells (hUC-MSC-EXOs) can safely and effectively improve core symptoms in children aged 3-7 years with autism spectrum disorder (ASD). It is a 24-week, non-randomized, controlled, open-label trial. Sixty pediatric patients with ASD will be non-randomly assigned at a 1:2 ratio to two groups: a no-intervention control group and an active exosome nasal spray treatment group. The treatment group will receive the nasal spray every other day, totaling 10 administrations throughout the study. The no-intervention control group will receive no experimental treatment but will undergo the same assessments and safety checks with the treatment group. This design aims to monitor the safety and efficacy of the hUC-MSC-EXOs nasal spray.
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 7 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis meets the ICD-11 ASD criteria or DSM-5 ASD clinical diagnostic standards.
- •No significant improvement in core symptoms was observed after ≥3 months of standardized behavioral intervention.
- •Score ≥30 on the CARS2, indicating mild-to-moderate or more severe autism.
- •Aged 3 (inclusive) to 7 (inclusive) years, regardless of gender
- •Voluntary participation in this clinical study, with written informed consent provided by the patient's legal guardian, and willingness to undergo examinations, treatment, and cooperate with follow-up visits.
- •In the investigator's judgment, the patient is capable of understanding and complying with study requirements.
排除标准
- •History of severe allergic reactions.
- •Any severe mental disorder or other types of autism spectrum disorders.
- •History of epileptic seizures within the past six months.
- •Autism secondary to epilepsy, cerebrovascular disease, or traumatic brain injury.
- •Disease severity rated as normal, borderline mental disorder, or mild mental disorder on the Clinical Global Impression scale.
- •Moderate or severe extrapyramidal symptoms or tardive dyskinesia.
- •Severe self-injurious behavior.
- •Active systemic or severe localized infections, including human immunodeficiency virus, syphilis, and hepatitis.
- •Autoimmune diseases.
- •Major organ impairment.
- •Severe pulmonary or hematological diseases, malignancies, or immunodeficiency.
- •Concurrent treatments that may interfere with the safety and efficacy evaluation of stem cell therapy.
- •Participation in other clinical trials within the past three months.
- •Other clinical conditions deemed by investigators as unsuitable for study inclusion.
研究组 & 干预措施
hUC-MSC-EXOs Nasal Spray Group
干预措施: hUC-MSC-Exos Nasal Spray (Biological)
No-Intervention Control Group
结局指标
主要结局
Number of Participants with Treatment-Related Adverse Events
时间窗: Week 2, Week 6, Week 24
The incidence of treatment-related adverse events will be assessed and graded according to the Common Terminology Criteria for Adverse Events version 5.0. The number of participants experiencing any treatment-related adverse event will be recorded and reported.
Childhood Autism Rating Scale, Second Edition (CARS-2) Score
时间窗: Week 6, Week 24
Scale Description: The CARS-2 is a 15-item behavior rating scale used to diagnose and assess the severity of ASD. Range of Scores: 15 to 60. Interpretation: A higher score indicates a worse outcome.
次要结局
- Serum Level of Tumor Necrosis Factor-Alpha (TNF-α)(Week 6, Week 24)
- Autism Behavior Checklist (ABC)(Week 6, Week 24)