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.
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
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.
Parallel
hUC-MSC-Exos Nasal Spray
Biological/VaccineThe total therapeutic dosage is set at 400 billion hUC-MSC-EXOs particles. Each nasal administration delivers 40 billion particles, with a total of 10 administrations completing the full course. The treatment is administered on an alternate-day schedule.
Number of Participants with Treatment-Related Adverse Events
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
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.
Autism Behavior Checklist (ABC)
Scale Description: The ABC is a 57-item checklist used to screen for and assess behaviors associated with ASD. Range of Scores: 0 to 158. Interpretation: A higher score indicates a worse outcome.
Serum Level of Tumor Necrosis Factor-Alpha (TNF-α)
Serum concentration of the inflammatory cytokine TNF-α. The unit of measure is pg/mL.
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Dongfang People's Hospital
Lead sponsor
Shanghai Tongjin Stem Cell Technology Co., Ltd.
Collaborator
