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

NCT ID: NCT07243561

Last Updated: 2025-11-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-11-30

Study Completion Date

2026-07-30

Brief Summary

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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.

Detailed Description

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Conditions

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Autism Spectrum Disorder (ASD Prospective Study

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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hUC-MSC-EXOs Nasal Spray Group

Group Type EXPERIMENTAL

hUC-MSC-Exos Nasal Spray

Intervention Type 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.

No-Intervention Control Group

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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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.

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.
Minimum Eligible Age

3 Years

Maximum Eligible Age

7 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Dongfang People's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Dongfang People's Hospital

Dongfang, Hainan, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Zhongmin Liu

Role: CONTACT

+86-0898-38932608

Facility Contacts

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Zhongmin Liu

Role: primary

+86-0898-25522727

Other Identifiers

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2025-DFKYLL-002

Identifier Type: -

Identifier Source: org_study_id

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