Autism Spectrum Disorder (ASDPhase 1/2EXO-ASDNCT07243561
Prospective Clinical Study on Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes for the Treatment of Childhood Autism
A Phase 1/2 study testing hUC-MSC-Exos Nasal Spray in people with Autism Spectrum Disorder (ASD and Prospective Study, run by Dongfang People's Hospital at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 3 to 7 years
- Conditions the study is for
- Autism Spectrum Disorder (ASD, Prospective Study
- What is being tested?
- hUC-MSC-Exos Nasal Spray
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- 1 Sep 2025to 30 Oct 2026
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 7 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Dongfang People's Hospital
Dongfang, Hainan
Zhongmin Liu, +86-0898-25522727, wujunCh4773@163.com
About this study
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.
- Conditions
- Autism Spectrum Disorder (ASD, Prospective Study
- Being tested
- hUC-MSC-Exos Nasal Spray
- Keywords
- Autism Spectrum Disorder, umbilical cord mesenchymal stem cells, exosome
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hUC-MSC-Exos Nasal SprayBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| hUC-MSC-EXOs Nasal Spray Group | Experimental | Not described Receives: Biological: hUC-MSC-Exos Nasal Spray |
| No-Intervention Control Group | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Week 6, Week 24
Secondary outcomes (2)
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.
Measured: Week 6, Week 24
Serum Level of Tumor Necrosis Factor-Alpha (TNF-α)
Serum concentration of the inflammatory cytokine TNF-α. The unit of measure is pg/mL.
Measured: Week 6, Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2025
- Study start1 Sep 2025
- Primary completion30 Oct 2026 (estimated)
- Study completion30 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum Disorder (ASDStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dongfang People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Tongjin Stem Cell Technology Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07243561
- Sponsor's protocol ID
- 2025-DFKYLL-002
- Acronym
- EXO-ASD
- Status last verified
- Sep 2026
- First submitted
- 13 Nov 2025
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No