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

NCT07243561 | Recruiting Phase 1 DMC

• 1 other identifier: 2025-DFKYLL-002
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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, randomized, controlled, open-label trial. Forty pediatric patients with ASD will be randomly assigned at a 1:1 ratio to two groups: an active exosome nasal spray treatment group and a no-intervention control 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 concurrently with the treatment group. This design aims to monitor the safety and efficacy of the hUC-MSC-EXOs nasal spray.

Conditions

Autism Spectrum Disorder (ASD; Prospective Study

Keywords

Autism Spectrum Disorder; umbilical cord mesenchymal stem cells; exosome

Outcome Measures

Primary Outcomes (2)

• 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.

  Week 2, Week 6, Week 24

• 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.

  Week 6, Week 24

Secondary Outcomes (2)

• Autism Behavior Checklist (ABC)

  Week 6, Week 24

• Serum Level of Tumor Necrosis Factor-Alpha (TNF-α)

  Week 6, Week 24

Study Arms (2)

hUC-MSC-EXOs Nasal Spray Group

EXPERIMENTAL

Biological: hUC-MSC-Exos Nasal Spray

No-Intervention Control Group

NO INTERVENTION

Interventions

hUC-MSC-Exos Nasal Spray BIOLOGICAL

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.

hUC-MSC-EXOs Nasal Spray Group

Eligibility Criteria

• Age: 3 Years - 7 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Dongfang People's Hospital: lead
• Shanghai Tongjin Stem Cell Technology Co., Ltd.: collaborator

Study Sites (1)

Dongfang People's Hospital

Dongfang, Hainan, China

RECRUITING

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Central Study Contacts

Zhongmin Liu

CONTACT

+86-0898-38932608; dfsrmyykjk@126.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2025
• First Posted: November 24, 2025
• Study Start: September 1, 2025
• Primary Completion (Estimated): July 30, 2026
• Study Completion (Estimated): July 30, 2026
• Last Updated: April 14, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)