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

Prospective Clinical Study on Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes for the Treatment of Childhood Autism (NCT07243561) is a Phase 1 / Phase 2 interventional studying Autism Spectrum Disorder (ASD and Prospective Study, sponsored by Dongfang People's Hospital. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

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.

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Autism Spectrum Disorder (ASD, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 40 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 willing to sign a consent form 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 Should NOT Join This Trial: - 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 weakened immune system virus, syphilis, and hepatitis. - autoimmune conditions (where your immune system attacks your own body)s. - Major organ impairment. - Severe pulmonary or hematological diseases, malignancies, or weakened immune system. - 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. Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

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.

Treatments Being Tested

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.

Locations (1)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Dongfang People's Hospital

Dongfang, Hainan, China

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT07243561), the sponsor (Dongfang People's Hospital), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT07243561 clinical trial studying?

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 full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT07243561?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT07243561?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT07243561. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT07243561. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-06-07 · Data from ClinicalTrials.gov.