Prospective Clinical Study on Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes for the Treatment of Childhood Autism

Обзор

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 on Mondays, Wednesdays, and Fridays, 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.

Вмешательства

• Биопрепарат hUC-MSC-Exos Nasal Spray
  The 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.

Первичные конечные точки

• Number of Participants with Treatment-Related Adverse Events [Срок оценки: Week 2, Week 6, Week 24]
• Childhood Autism Rating Scale, Second Edition (CARS-2) Score [Срок оценки: Week 6, Week 24]

Вторичные конечные точки (2)

• Autism Behavior Checklist (ABC) [Срок оценки: Week 6, Week 24]
• Serum Level of Tumor Necrosis Factor-Alpha (TNF-α) [Срок оценки: Week 6, Week 24]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Dongfang People's Hospital — Dongfang

Идентификаторы

NCT: NCT07243561 · 2025-DFKYLL-002

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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