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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Clinical Study on Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes for the Treatment of Childhood Autism
- ClinicalTrials.gov ID
- NCT07243561
- Acronym / sponsor ID
- EXO-ASD / 2025-DFKYLL-002
- Conditions
- Autism Spectrum Disorder (ASD, Prospective Study
- Keywords
- Autism Spectrum Disorder, umbilical cord mesenchymal stem cells, exosome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Dongfang People's HospitalUniversity, hospital or other
- Collaborators
- Shanghai Tongjin Stem Cell Technology Co., Ltd.
- Study start
- 1 Sep 2025 (actual)
- Primary completion
- 30 Oct 2026 (estimated)
- Study completion
- 30 Oct 2026 (estimated)
- First posted
- 24 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| hUC-MSC-EXOs Nasal Spray Group | Experimental | Biological: hUC-MSC-Exos Nasal Spray |
| No-Intervention Control Group | No intervention | – |
Who may qualify
- Ages
- 3 to 7 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| hUC-MSC-Exos Nasal SprayThe 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. | Biological | hUC-MSC-EXOs Nasal Spray Group |
Primary outcome
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.
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.
2 secondary outcomes
Autism Behavior Checklist (ABC)
Time frame: Week 6, Week 24
Serum Level of Tumor Necrosis Factor-Alpha (TNF-α)
Time frame: Week 6, Week 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Zhongmin Liu (+86-0898-38932608, dfsrmyykjk@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Treatment-Related Adverse Events (time frame: Week 2, Week 6, Week 24). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.