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

April 12, 2026 updated by: Dongfang People's Hospital
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

40

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
    • Hainan
      • Dongfang, Hainan, China
        • Recruiting
        • Dongfang People's Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: hUC-MSC-EXOs Nasal Spray Group
Biological: 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.
No Intervention: No-Intervention Control Group

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants with Treatment-Related Adverse Events
Time Frame: 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.
Week 2, Week 6, Week 24
Childhood Autism Rating Scale, Second Edition (CARS-2) Score
Time Frame: 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.
Week 6, Week 24

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Autism Behavior Checklist (ABC)
Time Frame: Week 6, Week 24

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.
Week 6, Week 24
Serum Level of Tumor Necrosis Factor-Alpha (TNF-α)
Time Frame: Week 6, Week 24
Serum concentration of the inflammatory cytokine TNF-α. The unit of measure is pg/mL.
Week 6, Week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Dongfang People's Hospital

Collaborators

Shanghai Tongjin Stem Cell Technology Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 1, 2025

Primary Completion (Estimated)

July 30, 2026

Study Completion (Estimated)

July 30, 2026

Study Registration Dates

First Submitted

November 13, 2025

First Submitted That Met QC Criteria

November 17, 2025

First Posted (Actual)

November 24, 2025

Study Record Updates

Last Update Posted (Actual)

April 14, 2026

Last Update Submitted That Met QC Criteria

April 12, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2025-DFKYLL-002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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